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# The Clinical Trial Vanguard
Your premier source for the latest clinical trial news
## Posts
### [AI and ML in Predicting Biopharmaceutical Product Profile Approvability](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-predicting-biopharmaceutical-product-profile-approvability/)
**Published:** August 6, 2024
**Author:** Moe Alsumidaie
**Content:**
In a fascinating session, “Applying Machine Learning and Artificial Intelligence for Predicting Product Profile Approvability” session, industry leaders dissected the evolving roles of artificial intelligence (AI) and machine learning (ML) in pharmaceutical regulatory processes at the [2024 DIA conference](https://www.diaglobal.org/Flagship/DIA-2024 "2024 DIA conference"). Dr. Romi Singh, an influential figure in global drug registration and AI/ML application in regulatory sciences and Founder and Principal Advisor at GRA Advisors, chaired the session.
The panel included Amin Osmani, CEO of Cedience Inc.; Subha Madhavan, PhD, Vice President and Head of AI/ML at Pfizer; and Lily Li, JD, founder and president of Metaverse Law. Each brought a unique perspective on AI and regulatory frameworks, ensuring a multifaceted discussion on AI’s application and regulatory challenges in the pharmaceutical industry.
#### [](#cloud-computing-and-ai-for-regulatory-predictions)**Cloud Computing and AI for Regulatory Predictions**
Amin Osmani, CEO of Cedience Inc., started the panel by exploring how his company uses cloud computing to enhance regulatory predictions. Osmani highlighted that while AI’s textbook definition focuses on replicating human intelligence, the real value lies in its practical applications. “Artificial intelligence is not just about mimicking human intelligence,” he explained. “It’s about creating tools that solve real-world problems efficiently.”
Osmani’s team incorporates cloud computing to analyze historical approval data, identify patterns, and forecast the probable outcomes of new drug applications. “By harnessing cloud resources, we’ve been able to create adaptive models that can quickly process and analyze data, making our predictions both faster and more reliable,” he added. This approach accelerates the regulatory process and ensures more robust data handling and storage capabilities, catering to the needs of life sciences organizations dealing with high volumes of sensitive information.
#### [](#pfizers-ai-driven-approaches)**Pfizer’s AI-Driven Approaches**
Subha Madhavan enthralled the audience with a detailed look at Pfizer’s use of AI and ML. “Our focus is on enhancing R&D productivity,” she stated, offering a glimpse into Pfizer’s innovative AI applications. One of the most compelling examples was Pfizer’s global challenge of using AI to generate clinical study reports (CSRs). The challenge aimed to determine whether AI could outperform humans in generating initial drafts of these critical documents.
Madhavan explained that this challenge involved 21 companies globally experimenting with fine-tuning models using proprietary tools and open-source models like GPT-3. The results were promising, with AI-generated drafts showing 80% factual accuracy. However, she pointed out that human intervention was still necessary to ensure the clarity and precision of the final documents. “AI could rapidly generate drafts, but humans were indispensable for refining the nuanced language and ensuring the accuracy of complex medical data,” Madhavan noted. Through this challenge, Pfizer learned that generative AI’s power lies in its ability to expedite routine tasks, allowing experts to focus on higher-order cognitive functions such as analysis and interpretation.
She elaborated on the importance of distinguishing between analytic AI and generative AI, emphasizing that while analytic AI helps classify data and predict outcomes based on historical trends, generative AI can create content and interpret large datasets. For example, analytic AI assists in sorting patients by conditions or predicting treatment efficacy, while generative AI predicts the next word or image, generating reports or scientific illustrations. Integrating both forms of AI could dramatically transform drug development processes, making them more efficient and insightful.
#### [](#legal-dimensions-of-ai-regulation)Legal Dimensions of AI Regulation
Lily Li provided essential legal perspectives, emphasizing the need for a unified regulatory framework to guide AI development and application. “We need a common federal body of law to define AI,” she asserted, highlighting the risks of fragmented regulations that could hamper innovation. She compared the current regulatory uncertainty to the early days of the automotive industry, where varied rules initially impeded progress until standard regulations were established. Without comprehensive legislation, she warned, AI technology might face similar hurdles that could inhibit its growth and deployment in critical sectors like pharmaceuticals.
Li spoke about the proliferation of AI legislation, such as California’s comprehensive privacy laws, including stringent AI regulations. Li said these can sometimes lead to excessive rules, stifling innovation. For instance, the California Consumer Privacy Act (CCPA) included clauses allowing consumers to object to automated decision-making, resulting in extensive new regulations that many argue exceed the original legislative intent. “We need thorough yet balanced legislation that protects consumers without overburdening innovators,” Li insisted. To support her point, she referenced examples from the data privacy domain where a lack of clear, unified regulations led to overly burdensome compliance requirements, suggesting the same risk for AI technologies if not appropriately regulated.
#### [](#practical-applications-and-case-studies)Practical Applications and Case Studies
Dr. Singh illustrated the practical application of AI through a compelling case study from his career at Pfizer. He shared how sentiment analysis of regulatory meeting minutes was utilized to predict product success. The project involved analyzing 20 years of regulatory documents, including meeting minutes and email communication, through a specialized app that scored sentiments to gauge the likelihood of regulatory approval. By quantifying sentiment trends over time, the team could identify positive or negative shifts in regulatory feedback, providing critical insights for strategic decision-making.
Using sentiment scoring, the team could track the sentiment trends over time, identifying positive or negative shifts in regulatory feedback. For instance, a notable drop in positive sentiment scores around 2009 and 2013 coincided with critical input from the FDA, which eventually impacted the product’s marketability despite its approval. Conversely, a spike in positive sentiments in 2018 reflected favorable regulatory reviews, aligning with the product’s subsequent launch. This case study underscored how AI could be leveraged to reduce guesswork and increase predictive accuracy, making regulatory processes more efficient and transparent.
#### [](#future-directions-and-challenges)Future Directions and Challenges
The session concluded with a forward-looking dialogue about the future of AI in regulatory science. Panelists emphasized the need for ongoing evaluation and standardization to ensure AI-generated outputs are both reliable and useful. Subha Madhavan mentioned the necessity for robust evaluation metrics and continuous improvement cycles, while Lily Li stressed the importance of a regulatory framework that can adapt to technological advances. The panel discussed the importance of collaboration between regulatory bodies and tech innovators to pave the way for AI advancements.
Interactive [DIA](https://www.clinicaltrialvanguard.com/conference-coverage/powerful-innovations-in-medical-device-software-at-dia-europe-2024/ "DIA") Q&A sessions allowed attendees to engage deeply with the panelists, discussing topics such as AI’s role in clinical trials, ethical considerations in AI deployment, and the future of AI in drug development. Questions reflected the audience’s keen interest in leveraging AI to streamline and enhance regulatory processes. Panelists responded with actionable insights, reinforcing the importance of a collaborative approach in tackling the challenges and maximizing the benefits of AI technologies.
**Categories:** Article: Conference Coverage
---
### [In-Silico Breakthrough: EMA’s Shift from Animal Testing](https://www.clinicaltrialvanguard.com/conference-coverage/in-silico-breakthrough-emas-shift-from-animal-testing/)
**Published:** May 20, 2024
**Author:** Moe Alsumidaie
**Content:**
The [DIA Europe 2024 conference](https://www.diaglobal.org/Flagship/DIA-Europe-2024 "DIA Europe 2024 conference") brought together leading experts to discuss groundbreaking innovations and regulatory strategies in clinical trials. Key topics included the implementation of the 3Rs (reduction, replacement, and refinement of animal testing), the potential of organ-on-chip (OOC) technologies, and the need for collaborative regulatory advice. The conference emphasized the importance of early dialogue, international collaboration, and the adoption of advanced in vitro models to improve the efficiency and accuracy of drug development while adhering to ethical standards.
### [](#embracing-the-3rs-reduction-replacement-and-refinement)Embracing the 3Rs: Reduction, Replacement, and Refinement
Sonja Beken, Chair of the 3Rs Working Party at EMA and Coordinator of Non-Clinical Evaluators at the Belgian Federal Agency for Medicines and Health Products opened the discussion by emphasizing the significance of the 3Rs—reduction, replacement, and refinement of animal testing. Beken highlighted the evolving landscape since 2010, noting the establishment of the 3Rs Working Party in 2023 to spearhead strategic initiatives aimed at minimizing animal use in preclinical testing.
Beken pointed out that while traditional animal testing has been the cornerstone of preclinical safety assessment, it has limitations. The paradigm shift towards investigative toxicology, integrating in silico models, AI, and organ-on-chip technologies, aims to predict human toxicities more accurately. The goal is to move from descriptive to predictive toxicology, reducing reliance on non-human primates, especially given their scarcity and ethical concerns.
### [](#industry-perspectives-on-3rs-implementation)Industry Perspectives on 3Rs Implementation
Natasa Zamurovic, Head of Immunology Preclinical Safety at Novartis, discussed practical ways the industry incorporates the 3Rs in preclinical testing, focusing on monoclonal antibodies and teratogenicity. For monoclonal antibodies, she explained that regulatory guidelines often require testing in non-human primates due to their physiological similarities to humans. However, advancements have allowed for a reduction in animal use through a robust weight-of-evidence approach. Zamurovic illustrated this with examples where a single, well-designed study in non-human primates can meet regulatory requirements. This approach leverages existing data and shared knowledge and integrates in vitro studies and computational models, enhancing the reliability of safety assessments while minimizing animal use.
Zamurovic also addressed the complexities of teratogenicity testing, particularly for drugs like thalidomide, known for severe developmental abnormalities. Traditional animal models often fail to predict human responses due to species-specific differences. Novartis advocates for human-relevant in vitro models using human-induced pluripotent stem cells (iPSCs) to study early developmental processes. By validating these models with known teratogens and comparing their predictions with clinical outcomes, researchers can improve the predictive accuracy of teratogenicity testing. These efforts and collaborative data sharing across the industry aim to enhance ethical and scientific rigor in preclinical testing.
### [](#advancing-organ-on-chip-technologies)Advancing Organ-on-Chip Technologies
The potential of OOC technologies was a focal point of the DIA Europe 2024 session. These advanced in vitro models simulate human organ functions, offering a promising alternative to animal testing. Beken emphasized that OOC technologies can predict human drug responses more accurately, particularly for liver and heart tissues. She discussed examples such as liver-on-chip systems used to predict drug-induced liver injury and heart-on-chip models for assessing cardiovascular safety. Beken stressed the importance of establishing clear qualification criteria and regulatory pathways to ensure their acceptance.
Beken highlighted recent initiatives under the Belgian Presidency of the Council of Europe aimed at qualifying these microphysiological systems. These efforts include multi-stakeholder workshops that bring together industry experts, regulators, and academics to define the standards and requirements for OOC technologies. By fostering collaboration and setting clear guidelines, these initiatives aim to accelerate the adoption of OOC models. Specific examples discussed included liver-on-chip systems being tested for their ability to replicate human liver responses to various drugs and heart-on-chip models evaluating the impact of new pharmaceuticals on cardiovascular health. These advancements aim to reduce reliance on animal testing and improve the efficiency and accuracy of drug development.
### [](#collaborative-regulatory-advice-a-path-forward)Collaborative Regulatory Advice: A Path Forward
Christophe Lahorte, Head of the National Innovation Office at the Belgian Federal Agency for Medicines and Health Products, introduced the concept of simultaneous national scientific advice (SNSA) at DIA Europe 2024. This initiative streamlines regulatory advice by involving multiple national competent authorities (NCAs) in a single, coordinated procedure, reducing divergent opinions and saving time for applicants. For example, companies can now receive harmonized guidance instead of seeking separate feedback from each NCA, making the drug development process more efficient.
Lahorte highlighted the success of SNSA pilots, which have seen increased participation from NCAs and improved convergence of regulatory opinions. One pilot involved regulatory advice for new cancer therapy, with coordinated input from NCAs across several EU countries, leading to a more cohesive regulatory pathway. He emphasized that early dialogue between regulators and developers helps identify and resolve potential issues sooner, facilitating smoother clinical trial applications under the Clinical Trials Regulation (CTR).
### [](#future-directions-and-global-harmonization)Future Directions and Global Harmonization
The session concluded with a panel discussion featuring Gunilla Andrew-Nielsen, Head of Clinical Trials at the Swedish Medical Products Agency, and other experts, who emphasized the critical need for global harmonization of regulatory acceptance criteria for novel methodologies. Andrew-Nielsen highlighted that inconsistent regulatory standards across different countries could hinder the adoption of innovative tools like AI and OOC technologies, slowing down drug development. For instance, while some countries have begun integrating AI models to predict drug interactions and side effects into their regulatory frameworks, others lag due to the lack of standardized criteria, resulting in delays and increased costs for pharmaceutical companies navigating multiple regulatory environments.
A promising initiative under the Belgian Presidency of the Council of Europe aimed to qualify OOC systems for predicting drug-induced liver injury and cardiovascular safety. However, without international collaboration to harmonize acceptance criteria, the full potential of these technologies may not be realized. Andrew-Nielsen and her colleagues stressed the importance of global regulatory bodies working together to establish common standards, arguing that such collaboration would streamline the approval process for new methodologies and foster innovation. They called for more global initiatives and multi-stakeholder workshops to ensure groundbreaking tools like AI and OOC technologies can seamlessly integrate into the drug development pipeline worldwide.
### [](#summary)Summary
This discussion at [DIA 2024](https://www.clinicaltrialvanguard.com/category/clinicaltrials/ "DIA 2024") highlighted significant advancements in preclinical testing methodologies and the importance of harmonizing regulatory frameworks globally. Emphasizing the 3Rs, leveraging OOC technologies, and adopting SNSA can revolutionize drug development. However, achieving these goals requires ongoing collaboration between industry experts, regulators, and academics to establish clear standards and guidelines. By fostering such partnerships, the pharmaceutical industry can reduce reliance on animal testing, streamline regulatory processes, and ultimately bring safer, more effective drugs to market more efficiently.
**Categories:** Article: Conference Coverage
---
### [FDA On Clinical Trial Innovation and Regulatory Evolution](https://www.clinicaltrialvanguard.com/conference-coverage/fda-on-clinical-trial-innovation-and-regulatory-evolution/)
**Published:** April 4, 2024
**Author:** Moe Alsumidaie
**Content:**
The [Enhancing Adoption of Innovative Clinical Trial Approaches: FDA Convening conference](https://healthpolicy.duke.edu/events/enhancing-adoption-innovative-clinical-trial-approaches "Enhancing Adoption of Innovative Clinical Trial Approaches: FDA Convening conference") hosted by Duke Margolis Institute for Health Policy, presented an unparalleled opportunity to discuss the current state and future directions of clinical trial innovation. [Patrizia Cavazzoni](https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/patrizia-cavazzoni "Patrizia Covidzone"), the Director of the Center for Drug Evaluation and Research (CDER) at the FDA, shared the stage with industry experts to explore transformative approaches within the regulatory landscape, emphasizing the FDA’s commitment to integrating innovation into drug evaluation and research processes.
#### [](#fdas-active-role-in-facilitating-clinical-trial-innovation)FDA’s Active Role in Facilitating Clinical Trial Innovation
A central theme of Cavazzoni’s presentation emphasized the FDA’s proactive approach to nurturing innovation within its regulatory framework. She detailed efforts to streamline FDA operations, making the drug approval process more efficient while maintaining high safety and efficacy standards. Innovations such as adaptive trial designs and incorporating digital health technologies are being actively integrated, demonstrating the FDA’s commitment to modernizing its processes. “*It’s now time to really think about how we can apply this innovation… we have to start thinking about garnering everything that we have done, harnessing everything that we’re doing*,” said Cavazzoni.
#### [](#focusing-on-real-world-studies-and-rare-diseases)Focusing on Real-world Studies and Rare Diseases
In a landmark move by the FDA, a drug for Friedreich’s ataxia—a [rare](https://www.clinicaltrialvanguard.com/article/fda-unveils-groundbreaking-rare-disease-guidance-document/ "rare") and debilitating neurodegenerative disease—was approved, marking a significant stride in regulatory innovation. Cavazzoni, highlighting this example, detailed how the FDA’s integration of real-world evidence and external control arms played a pivotal role in this process. Traditionally challenged by the scarcity of patients for large-scale trials, the FDA’s acceptance of real-world data, in this case, facilitated a streamlined approval path, thereby stressing the agency’s adaptability and commitment to accelerating access to treatments for rare conditions. This case illustrates the potential of innovative regulatory approaches to transform drug approval processes, ensuring faster delivery of critical therapies to patients in need.
#### [](#modernizing-inspections-and-promoting-cross-sector-collaboration)Modernizing Inspections and Promoting Cross-sector Collaboration
Cavazzoni delved deeper into how the FDA is spearheading efforts to modernize its inspection processes, a critical component in aligning with the evolving landscape of clinical trials. She elaborated on the agency’s strategic move towards enhancing the synergy between inspectors and the dynamic objectives of contemporary clinical trials, particularly those employing pragmatic designs and incorporating real-world evidence. This modernization effort aims to ensure that inspection protocols evolve with innovative trial methodologies, facilitating a regulatory environment that supports, rather than impedes, scientific advancement.
Furthermore, Covvazzoni highlighted the importance of enhancing collaboration within the FDA and with external stakeholders. By breaking down silos between its divisions, the FDA fosters a culture of interdisciplinary cooperation, encouraging the sharing of insights and innovative practices across various branches of the agency. This internal collaboration is complemented by external engagements with industry leaders, academic institutions, and patient advocacy groups, ensuring that the FDA’s regulatory policies evolve in tandem with scientific progress and public health needs.
#### [](#institutionalizing-innovation-from-concept-to-practice)Institutionalizing Innovation: From Concept to Practice
Cavazzoni provided a detailed look into the FDA’s methodical approach to embedding innovation within its regulatory framework. She explained how the agency is setting up structured collaboration and knowledge exchange platforms by creating cross-program forums. These forums are designed to bring together experts from various disciplines within the FDA to share insights, discuss challenges, and explore innovative solutions across the drug development and evaluation spectrum. By facilitating open dialogue and collaboration, these forums aim to break down silos within the agency and foster a culture of continuous improvement and innovation.
Moreover, Cavazzoni highlighted the establishment of a translational medicine team within the Office of New Drugs, marking a significant step in the FDA’s commitment to innovation. This specialized team collaborates closely with different divisions to identify and develop biomarkers and other critical endpoints. These efforts are mainly focused on supporting the accelerated approval of therapies for rare diseases where traditional clinical trial designs might not be feasible or practical. By focusing on translational medicine, the FDA aims to bridge the gap between early scientific discovery and clinical application, ensuring that novel therapies can reach patients more quickly and efficiently.
#### [](#the-path-forward-enhancing-external-collaborations-and-embracing-real-world-data)The Path Forward: Enhancing External Collaborations and Embracing Real-world Data
In the culmination of her presentation, Cavazzoni articulated a forward-looking vision for the FDA, emphasizing the critical role of collaborations with key institutions like the National Institutes of Health (NIH) and the Centers for Medicare & Medicaid Services (CMS). She detailed how these partnerships are instrumental in refining the drug development continuum, from the nascent phases of research to the nuanced realms of post-market surveillance. By joining forces with the NIH, the FDA aims to harness a wealth of scientific insights and resources to expedite the discovery and development of novel therapies. Similarly, collaboration with CMS is seen as pivotal in leveraging real-world data to monitor the performance of approved medications in diverse patient populations, ensuring their safety and effectiveness over time.
Cavazzoni championed a comprehensive strategy for drug approval and monitoring, advocating for an integrated model that values both pre-approval clinical evidence and post-approval real-world data. This approach underscores the FDA’s commitment to a regulatory ecosystem that adapts to the evolving landscape of medicine and technology, ensuring that regulatory practices facilitate the approval of innovative therapies and maintain a vigilant eye on their long-term impact on public health. Through these strategic partnerships and a holistic regulatory philosophy, the FDA is poised to navigate the complexities of modern healthcare, ensuring that the development and deployment of medications are efficient and patient-centered.
#### [](#summary-a-new-era-for-clinical-trials)Summary: A New Era for Clinical Trials
At the Adoption of Innovative Clinical Trial Approaches: FDA Convening conference, Cavazzoni highlighted the FDA’s proactive stance on integrating innovation into clinical trials, emphasizing streamlined operations and enhanced cross-sector collaboration. Her presentation showcased the agency’s commitment to modernizing regulatory frameworks and fostering a culture of innovation to facilitate the development and approval of new therapies, ensuring that the regulatory process keeps pace with advancements in medical science and technology.
**Categories:** Article: Conference Coverage
---
### [How Practical Is FDA's HCP Approach in DCTs?](https://www.clinicaltrialvanguard.com/article/how-practical-is-fdas-hcp-approach-in-dcts/)
**Published:** February 15, 2024
**Author:** Moe Alsumidaie
**Content:**
[The FDA’s latest guidance on Decentralized Clinical Trials (DCTs)](https://www.fda.gov/media/167696/download "The FDA's latest guidance on Decentralized Clinical Trials (DCTs)") marks a pivotal shift in the clinical research landscape. This transformative approach seeks to decentralize clinical trials by broadening the spectrum of participant access and leveraging the advancements in digital health technologies (DHTs). By extending trial-related activities beyond the confines of traditional clinical settings into participants’ homes and local healthcare facilities, the FDA aims to minimize travel burdens, enhance participant diversity, and improve trial accessibility. This article delves into the nuances of the FDA’s recommendations, exploring the integration of local healthcare providers (HCPs) into DCTs, the pivotal role of technology, the challenges, and the potential benefits of utilizing established infrastructures like retail pharmacies and core labs.
### [](#fdas-dct-recommendations-to-decentralize-focus-on-expanding-participant-access)**FDA’s DCT Recommendations to Decentralize Focus on Expanding Participant Access**
In the [FDA’s guidance for DCTs](https://www.fda.gov/media/167696/download "FDA's guidance for DCTs"), a significant emphasis is placed on diversifying and facilitating the conduct of clinical trials by extending activities beyond conventional settings. This approach advocates for leveraging local healthcare facilities and using technology in participants’ homes to diminish travel constraints, broaden participant diversity, and enhance trial accessibility.
- **Facilitating Broad Participation:** The FDA advocates for a broadened scope of trial activity locations, including participants’ homes or local healthcare facilities, to mitigate travel burdens and enhance diverse recruitment: “*In a DCT, some or all trial-related activities will occur at locations other than traditional clinical trial sites (e.g., the participant’s home or local healthcare facilities).*” (Section III.A, DCT Design).
- **Telehealth and Remote Visits:** Encouraging the adoption of telehealth, the guidance provides flexibility in how trials are conducted, potentially reducing the need for in-person interactions: “*In general, investigators can consider telehealth visits instead of in-person visits with trial participants if no in-person interaction is needed.*” (Section III.B, Remote Clinical Trial Visits, and Clinical Trial-Related Activities).
- **Involvement of Local HCPs in Trial Activities:** Recognizing the value of local HCPs, the guidance endorses their involvement in in-person trial activities, thus leveraging proximal healthcare resources: “*Depending on the trial protocol, in-person visits and trial-related activities may also be conducted by HCPs who are located close to trial participants’ homes but are not part of the trial personnel.*” (Section III.B, Remote Clinical Trial Visits and Clinical Trial-Related Activities).
- **Delegation to Local HCPs and Training Requirements:** The FDA’s guidance outlines a flexible approach to the delegation of trial-related activities to local HCPs, ensuring they can effectively contribute to DCTs while maintaining adherence to GCP standards. Specifically, the guidance states: “*When permitted by the trial protocol, investigators may delegate trial-related activities to local HCPs… These procedures may take place at participants’ locations or other local health care facilities as specified by the trial protocol. Trial-related activities that are unique to research and/or require a detailed knowledge of the protocol or the investigational product (IP) should be performed by qualified trial personnel who have been appropriately trained. When applicable, both trial personnel and trial participants should be trained on how to conduct or participate in a telehealth visit.”* (Section III.B, Remote Clinical Trial Visits and Clinical Trial-Related Activities).
### [](#using-dhts-in-dcts-makes-sense)Using DHTs in DCTs Makes Sense
[DHTs](https://www.clinicaltrialvanguard.com/?s=DHT "DHTs") in [DCTs](https://www.clinicaltrialvanguard.com/?s=DCT "DCTs") offer significant benefits, enhancing participant engagement, inclusion, and data collection efficiency. By enabling remote monitoring and data acquisition, DHTs facilitate broader participation across diverse geographical locations, reducing the need for travel and making clinical trials more accessible to underrepresented populations. This technology-driven approach ensures high-quality data is collected in real-time, directly from participants, improving the accuracy and reliability of trial outcomes. Additionally, DHTs support a patient-centric model of trial conduct, prioritizing convenience and minimizing the disruption to participants’ daily lives, thereby potentially increasing retention and adherence to trial protocols.
It is critical, however, to note that the FDA stresses ensuring that all trial participants have equitable access to these tools for effective data collection: “*Sponsors should ensure that DHTs used in a DCT are available and suitable for use by all trial participants.*” (Section III.C, Digital Health Technologies).
### [](#leveraging-local-hcps-presents-challenges-in-dcts-however)Leveraging Local HCPs Presents Challenges in DCTs, However
DCTs present distinct challenges, necessitating careful consideration and strategic planning, and it seems the FDA’s recommendations on leveraging local HCPs in clinical trials might not be well thought-out. Here are detailed insights into some of the challenges when using local HCPs:
- **Training and Compliance with GCP Standards**: To ensure HCPs unfamiliar with operating clinical trial procedures, comprehensive GCP training is required. This training can become especially important to maintain standards for data handling and adverse event reporting management to maintain research integrity. For example, a local nurse might need training on the specific protocol procedures, including making data corrections properly (if recorded on paper) and reporting adverse events, ensuring that their actions align with GCP standards.
- **Auditing and Monitoring Complications**: Since [local HCPs are not required to be listed on Form FDA 1572](https://acsjournals.onlinelibrary.wiley.com/doi/10.1002/cncr.35145 "local HCPs are not listed on Form FDA 1572"), identifying their locations for FDA and sponsor auditing and monitoring can be complex. This lack of visibility could hinder ensuring compliance and data integrity across all trial sites. An example includes the difficulty in scheduling site visits or remote audits when the trial sponsor or monitors do not have direct listings of all engaged HCP locations. Additionally, the diverse clinic setup and infrastructures across varying local clinics around a study site can increase the monitor and auditor burden and resources spent on monitoring and auditing visits.
- **Feasibility Assessments for HCP Locations**: Before local HCPs can participate in DCT activities, their facilities must undergo feasibility assessments to ensure they can effectively conduct clinical trial activities they have been delegated. This includes evaluating the local HCP’s clinic infrastructure, equipment, and staff qualifications to meet trial needs. For instance, a local clinic delegated to dispense IP and conduct vitals will need to be assessed for its ability to store investigational products at the required temperature or to calibrate its equipment to conduct vitals and process blood collection samples. This presents a challenge, especially with local clinics that are not experienced or understand the stringent requirements to conduct clinical trial procedures.
Addressing these challenges requires a nuanced approach, including developing clear guidelines for HCP involvement, robust training programs, and effective communication and oversight mechanisms through technology. Ensuring that local HCPs are adequately prepared and that the trial infrastructure supports their activities is crucial for successfully integrating HCPs into DCTs, ultimately enhancing trial accessibility and participant diversity. Nonetheless, from a logistical perspective, incorporating HCPs into clinical trials introduces several complications and risks, particularly concerning clinical trial oversight.
### [](#using-established-infrastructure-in-dcts-may-be-a-better-option)Using Established Infrastructure in DCTs May Be a Better Option
Integrating established infrastructures with DCTs, such as retail pharmacies and core laboratories, presents a strategic approach to enhancing trial accessibility and efficiency. This model capitalizes on these facilities’ robust, existing frameworks to streamline trial operations, ensuring a blend of convenience, reliability, and regulatory compliance. Here are the key advantages of using such infrastructure in DCTs:
- **Enhanced Access to Local Communities**: Retail pharmacies and core labs, often situated within communities or easily accessible locations, offer unparalleled access to a broad and diverse participant pool. Their presence in urban and rural settings helps mitigate the geographical and logistical barriers often associated with traditional site-based trials, thus broadening the demographic reach of clinical studies.
- **Ready-to-Use Infrastructure**: Core labs and pharmacies have state-of-the-art, calibrated equipment for precise diagnostic testing and other procedures often used in clinical trials. Their capability to conduct routine procedures such as blood draws, and vitals, which are integral to many clinical trials, with precision and efficiency ensures high-quality data collection. This reduces the burden of protocol deviations related to uncalibrated devices and maximizes the chances of deeming the facility acceptable for clinical trials during feasibility visits.
- **Scalable Operations with Existing Oversight Infrastructure**: These establishments are designed to handle scalable operations, making them well-suited for the fluctuating demands of clinical trials. Furthermore, retail pharmacies and core labs operate under stringent regulatory oversight, ensuring compliance with healthcare standards (to maintain their CLIA/CAP licenses). This existing oversight infrastructure supports rigorous monitoring and auditing processes, aligning closely with the regulatory requirements of clinical trials.
- **Streamlined Data Collection and Management**: Leveraging pharmacies’ and core labs’ digital and logistical capabilities can significantly streamline data collection and management. Many facilities have electronic data systems and protocols for secure data transfer, reducing the complexity of integrating trial data from multiple sources. These systems help to facilitate better clinical trial oversight.
### [](#summary-reevaluating-the-use-of-local-hcps-in-decentralized-clinical-trials)**Summary**: **Reevaluating the Use of Local HCPs in Decentralized Clinical Trials**
The FDA’s recommendations for DCTs mark a significant step toward enhancing trial accessibility and participant diversity. However, the practical challenges of integrating local HCPs into DCTs, such as ensuring GCP compliance, auditing, and monitoring difficulties, raise concerns about the feasibility and efficiency of this model.
As we navigate these challenges, leveraging established infrastructures like retail pharmacies and core laboratories emerges as a compelling alternative. These entities offer established, scalable operations with existing oversight mechanisms, ensuring a blend of convenience, reliability, and regulatory compliance. By focusing on these established infrastructures, the clinical research community can address logistical hurdles more effectively, making clinical trials more accessible while maintaining the integrity and oversight essential for successful outcomes.
**Categories:** Article, Article: Opinion
**Tags:** Clinical Research, Clinical Trials, Decentralized Clinical Trials, FDA Guidance
---
### [When the Trial Ends, the Data Work Begins](https://www.clinicaltrialvanguard.com/executiveinterviews/when-the-trial-ends-the-data-work-begins/)
**Published:** September 16, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Dr. Olga Kubassova, Founder and CEO of Image Analysis Group (IAG), discusses how AI-driven imaging biomarkers extend the value of clinical trial data beyond database lock, from IAG's real-world validation work with Ferring Pharmaceuticals in fertility trials to what it takes to scale an AI imaging pilot into a global rollout.
**Content:**
*The clinical trial is over. The data is locked. And for most sponsors, that is where the imaging story ends. Olga Kubassova thinks that is exactly the wrong place to stop. As founder and CEO of Image Analysis Group (IAG), Kubassova has spent two decades building computational tools that extract meaning from medical images long after the last patient visit. Her current work with Ferring Pharmaceuticals applies AI-driven ultrasound biomarkers to fertility trials, where the gap between a controlled trial population and real-world clinical practice is wide, the outcomes are deeply personal, and the regulatory frameworks for AI in clinical research are still catching up with the science. In this conversation, Kubassova explains why IAG validated its Ferring proof-of-concept against real-world data rather than expanding the trial dataset, how imaging biomarkers earn credibility as intermediate endpoints for live birth rates, and what it actually looks like to scale a single-site AI pilot to a global rollout.*
[](https://www.ia-grp.com/)
Dr. Olga Kubassova
Founder and CEO, Image Analysis Group (IAG)**Moe: Why did IAG validate the Ferring proof-of-concept against real-world clinical data rather than expanding the trial dataset?**
**Olga Kubassova:** Let me talk a little bit about the project itself. At the end of any trial, you have a set of patients who respond to the drug and a set who do not. Very often it is incredibly difficult to correlate baseline characteristics to those responses. Even if the drug is successful and you see statistical separation between the control group and the treatment group, you still have outliers, patients who for whatever reason do not respond. The same goes for placebo. So in this project, we wanted to understand whether there are baseline characteristics that would help us predict the likelihood of a patient’s response.
The trial was already finished, the data already collected, so expanding that database was not possible. But it was a great dataset to build a proof of concept on. Once we saw that the proof of concept was working, we ran quite a few tests, and we have a publication coming based on that work.
We also identified limitations. The trial database had only a single baseline time point per patient, and we were trying to understand how that single point connects to the outcome. By working with clinical sites on real-world data, we wanted two things: first, to see genuine real-world data rather than the more restricted trial population, and second, to bring in follow-up exams for these patients. So it is a slightly different database we are collecting now, and what we are trying to achieve is an expansion of our knowledge base from a single imaging point into multiple points, along with clinical outcomes. The next big step is to get this data, connect the trial data with the real-world data, retrain the model, and understand how it performs.
**Moe: How does this kind of model extend into real-world evidence studies more broadly?**
**Olga Kubassova:** When a pharma or biotech company completes a phase three trial, they are sitting on a sizable volume of data, and I think we are really under-using that data and those outcomes. We can use it to train an AI model, build those models, and then expand into real-world evidence studies. That is exactly what we are doing with a number of drugs right now. We are working with pharmaceutical and biotech companies who already have a database available and want to understand how that database could be useful in building real-world evidence.
**Moe: How does IAG demonstrate to a fertility sponsor that an ultrasound imaging biomarker is a credible intermediate endpoint for live birth rates?**
**Olga Kubassova:** It is a sophisticated imaging technique because we are not just dealing with an image, we are dealing with imaging biomarkers, digital biomarkers extracted from the image. We first need to segment the image into meaningful parts, the endometrium, the ovaries, and so on, and then analyze those physiological structures using texture analysis or similar approaches, where we can extract quantitative features from those parts of the image. Then you link the imaging findings to physical outcomes, including patient-reported outcomes and patient characteristics, through to live birth rates. That is how you demonstrate the connection.
After the first analysis predicting a successful outcome, the insight is that in a female cycle you can proceed with embryo implantation, or if conditions are not favorable, it is not a major step to postpone to the next cycle if your chances of pregnancy are higher. What makes this quite unique is that ability to provide someone with more certainty, or at least to highlight the risks. I think that will be extremely impactful in the real world.
[](https://www.ia-grp.com/)
**Moe: How does regulatory ambiguity around AI in trials shape conversations with sponsors who need submission-ready imaging endpoints right now?**
**Olga Kubassova:** Those regulations are mostly aimed at developers who are bringing AI tools into clinical practice. When you are targeting clinical practice deployment, you have a distinct pathway because you need to demonstrate that your AI tool works, that it is effective, that it is needed, and so on. Here we are talking about clinical trials and real-world evidence, so the pathway is slightly different, as are the requirements.
A database like the one from Ferring is already compliant, standardized, and available because it comes from a clinical trial. The next step is to build the requirements, and for any AI methodology those requirements center on correspondence to ground truth, what is the ground truth versus the AI outcome, and on reproducibility. We work within the guidelines for building any AI or automation within the clinical trial space, and those guidelines are clearly defined. Before placing anything into clinical trials, you also have to go through a number of dedicated steps to validate the software, or the parts of the software, that will be used to determine the impact on the drug or on patient safety.
[](https://www.ia-grp.com/)
**Moe: What does it look like from a principal investigator’s perspective when AI imaging actually changes a treatment decision?**
**Olga Kubassova:** When we built the first proof of concept, we were doing it with a small team, mainly the sponsor team and our team. We were thinking about how this actually works in practice, and then you bring it in front of investigators and the response could be anything. It could be very positive, or it could be: I really do not need this.
In the Ferring case, we had a great response. It is of course a real challenge because fertility is a very private issue, very personal, and sometimes embarrassing, so you need to be very mindful. But investigators who work in this space are very passionate about their patients, they know their patients. The reaction we had was extremely positive and extremely supportive. I think it is also a kind of sign of hope that you will be able to enable better outcomes.
**Moe: Why does a successful pilot so often stay at the pilot stage, and what does it take to move beyond it?**
**Olga Kubassova:** Ferring is not the only project we run for a large pharmaceutical company. To talk about the pilot-to-rollout question, I will use a project that is at a more advanced stage. We have another partner, Takeda Pharmaceuticals, and with them we have gone through the whole path from pilot to global rollout.
The project is focused on gastroenterological drug performance. We believe the effect of the drug is on mucosal healing, and you see resolution in the number of neutrophils and changing cells in a histology scan taken from the GI tract. Our pilot was just a few images from a single center, working with one investigator, testing whether the idea was feasible, but with a very limited database.
Once we saw that we could build something, our first step, what we call a pilot rollout, went to eight sites. All eight clinical sites contributed forty images to the project. That allowed us to collect images from dispersed locations and different scanners, and to refine the model. Moving from a single-center pilot to an eight-center pilot rollout really let us test the model, examine engagement, and see how it worked in practice. Following that, we rolled out the full AI to a much broader number of sites worldwide.
The paper covering that whole journey, from first pilot to global rollout, is coming out. The impact is not just building the methodology and the algorithm. It is also the global engagement of sites, investigators, and sponsors, and that engagement stays with you for a long time. Anything you build within a pilot really needs to find real-world applicability, because otherwise pilots stay at a very initial stage. It is expensive, but if you are building something worthwhile, it will bring back the ROI.
*Dr. Olga Kubassova is founder and CEO of Image Analysis Group (IAG), a medical imaging and AI analytics company specializing in clinical trial endpoints and real-world evidence.*
This interview is sponsored by [Image Analysis Group (IAG)](https://www.ia-grp.com).
**Categories:** Article: Executive Interviews
---
### [When the Trial Fails, Was It the Drug or the Design?](https://www.clinicaltrialvanguard.com/executiveinterviews/when-the-trial-fails-was-it-the-drug-or-the-design/)
**Published:** September 20, 2026
**Author:** Moe Alsumidaie
**Excerpt:** NetraMark's Chief Innovation and Regulatory Officer Luca Pani, MD, on why broad enrollment can obscure treatment effects, what makes an AI-derived subgroup credible, and how Phase 2 should inform Phase 3.
**Content:**
 Luca Pani, MD, Chief Innovation and Regulatory Officer, NetraMark *A negative Phase 3 result does not necessarily establish that a treatment has no biological or clinical effect in any patient. That argument sits at the center of NetraMark’s clinical platform, and few people are positioned to make it with more authority than Pani. Pani previously served as Director General of the Italian Medicines Agency and as a member of the European Medicines Agency Management Board, the Committee for Medicinal Products for Human Use and the Scientific Advice Working Party, spending decades reviewing the evidence packages that determine whether a treatment reaches patients. Now, as Chief Innovation and Regulatory Officer at NetraMark, he brings that regulatory lens to a different problem: why sponsors keep designing trials that are structurally set up to miss the signal. In this conversation, Pani explains what a credible AI-derived responder profile actually requires, why “just increase the sample size” is the wrong answer to biological heterogeneity, and why the most important question in clinical development is not whether a drug works, but who it works for.*
---
### [](#why-did-your-regulatory-experience-convince-you-that-a-failed-phase-3-was-often-a-patient-selection-problem-rather-than-proof-the-drug-didnt-work)Why did your regulatory experience convince you that a failed Phase 3 was often a patient selection problem rather than proof the drug didn’t work?
**Luca Pani:** Just to clarify: what convinced me to join NetraMark and help build it into a regulatory-grade clinical platform was not one failed trial. As a regulator, both at the Italian level, where I was head of the agency, and at the European level, I had seen this pattern repeatedly.
When a Phase 3 is negative or fails for some reason, the conclusion is almost always: “The drug did not work.” But that is not what the trial is actually showing. What it shows is that the pre-specified analysis did not demonstrate the required treatment effect in the enrolled population,using that dose, that endpoint, that design, those sites, and that execution. Many other factors are involved. And this is an important distinction, because a failed trial is not proof that the treatment has zero biological or clinical effect.
A late-stage trial can fail for several reasons. As a rapporteur and a coordinator of a major agency who reviewed or oversaw hundreds of development programs and pivotal evidence packages, I saw that some molecules were genuinely ineffective,the dose was wrong, or the endpoint was insensitive. But I also learned that these explanations must never be invented after the fact. Post-hoc analyses can be scientifically valuable for generating hypotheses, but they cannot retrospectively convert a negative confirmatory trial into positive confirmatory evidence. Treat any subgroup identified after unblinding as exploratory until you test it independently and prospectively. .
The example that really crystallized this for us at NetraMark was a major depressive disorder program. They had a very positive Phase 2 study, by all measures. And then the Phase 3 failed. In the broad population, the Cohen’s D was 0.082 and the p-value was 0.558, almost 0.6.
However, NetraAI was able to analyze the Phase 2 data and found a compact, treatment-favoring patient pattern. That is very important: this is a pattern the machine finds. It is not a language model describing what patients look like. When you apply that pattern retrospectively to the Phase 3, the estimated effect size moves to 0.346 and the p-value becomes 0.053. That is still not a successful confirmatory trial. I want to be very disciplined about what that means. But it does generate serious, falsifiable, prospectively testable hypotheses suitable for regulatory discussion.. The next trial can be designed to stratify those populations. For me, that was the conceptual turning point. That retrospective result does not rescue the original Phase 3 trial or establish efficacy. It generates a falsifiable hypothesis that can be locked before the next trial through prespecified eligibility or stratification criteria, an appropriate testing hierarchy and control of Type I error.
---
### [](#why-do-sponsors-keep-designing-broad-phase-3-enrollments-when-the-evidence-for-biomarker-based-selection-is-this-strong)Why do sponsors keep designing broad Phase 3 enrollments when the evidence for biomarker-based selection is this strong?
**Luca Pani:** I will be a little less politically correct here. The first reason is commercial. Companies understandably want the broadest possible label and the largest possible market. A broad label has enormous value, and the logic is understandable. The problem is it only works if the trial succeeds. A clinically meaningful responder subgroup can be diluted in a broad trial, exposing the sponsor to the loss of tens or hundreds of millions of dollars in late-stage development.
The second reason is scientific uncertainty. A biomarker might look promising but may not have a sufficiently validated assay, a stable threshold, or convincing evidence that it predicts differential treatment benefit rather than just prognosis alone. That is an essential distinction: a prognostic marker tells you who will do well or badly regardless of treatment; a predictive marker tells you who benefits more from the drug than the comparator. For predictiveenrichment, you need the second question answered, not the first.
The third reason is operational. Narrowing eligibility creates screen failures and makes recruitment much harder. I was just called by a recruiter who said, “We are running according to the inclusion and exclusion criteria and we cannot find the patients.” The patients exist, but you cannot enroll them because you need new assays, very few capable sites, and so on. And how do sponsors usually respond to that uncertainty? They increase the sample size. They do not refine how they find those patients. They just say, we want 300 or 500. That will cost a fortune and elongate everything. Larger noisy trials can improve statistical precision, but more patients alone do not correct a poorly chosen population, and insensitive endpoint, or systematic bias.
The fourth reason is strategic and regulatory. If you study only the biomarker-positive population, what can you say about everyone else? Could the treatment work in biomarker-negative patients? Human genetics and epigenetics are very complex. The FDA enrichment guidance asks sponsors to consider both the enriched population and the patients who do not meet the enrichment criteria. A regulator’s responsibility is to approve a population no broader – and no narrower – than the totality of evidence and the benefit-risk assessment can support. . We have published extensively on equity and representation in trials, including two commentaries, one in Nature Medicine and one in Nature Digital Medicine, specifically on this.
And finally, many treatment response patterns are not captured by a single biomarker. They arise from combinations of clinical severity, physiology, imaging, cognition, medical history, molecular information, and placebo response. We pursue biomarkers of vulnerability, but we tend to ignore biomarkers that might be protective. That makes things considerably more complicated. Who enters a trial is not an administrative detail. It is part of the scientific hypothesis.
> “Who enters the trial is not merely an administrative matter or a set of inclusion and exclusion criteria.. It’s part of the scientific hypothesis.”
---
### [](#how-does-netramark-evaluate-whether-an-ai-derived-responder-profile-from-phase-2-data-is-credible-enough-to-inform-a-phase-3-enrollment-strategy)How does NetraMark evaluate whether an AI-derived responder profile from Phase 2 data is credible enough to inform a Phase 3 enrollment strategy?
**Luca Pani:** Credibility cannot be a p-value. And it is certainly not something you can just claim. Credibility in front of EMA, PMDA, and FDA is an evidence package.
NetraAI was specifically designed to find interpretable structure in the small, complex datasets typical of clinical trials, where conventional machine learning approaches are vulnerable to overfitting. When we receive a Phase 2 dataset, the first question we ask is: what decision will this analysis support? Are we generating an internal hypothesis about whether to finance a Phase 3? Are we excluding patients from a pivotal trial? Are we eventually supporting a regulatory submission? That matters enormously, because depending on the consequences of the AI output, the credibility burden is higher. We ask the sponsor to fill in the evidence they have.
The second question is whether the data can be trusted. You can give me a zillion data points, but if they are dirty, it is junk in, junk out. No machine, no AI, nothing will fix that. We need to examine provenance, missingness, protocol deviations, site and country effects, rater behavior, variable definitions, and treatment exposure. We also need to confirm that no information from after baseline has accidentally entered a model intended for prospective screening. When sponsors ask how we handle missing data, my regulatory answer is: we do not recreate missing observations by imputation; we estimate them under assumptions. Those assumptions must be explicit, justified and examined through sensitivity analyses.
The third question is whether the model-derived subgroup represents treatment predictivity, not just improvement. It is not enough to find patients who improve, because they might improve equally on placebo. In the Phase 3 trial I described, the loss of drug-placebo separation appeared to be driven substantially by increased placebo response rather than by a comparable deterioration in the active-treatment outcome. . NetraAI explicitly accounts for placebo response rather than treating it as noise. It examines patterns of response in both treatment and placebo groups to identify patient profiles associated with greater treatment-control separation. The mathematics are Dr. Geraci’s domain, not mine.
The fourth question is whether the pattern is stable. Does it survive resampling? Does it recur across different training splits? Does it remain in an untouched held-out dataset? Does it survive perturbations in the assumptions? We use repeated resampling, robustness testing and, where feasible, a genuinely untouched holdout dataset. . Overfitting is the failure mode here.
The fifth is operational. A Phase 3 screening procedure cannot depend on 150 obscure variables. It needs to be a compact combination of clinically measurable variables with defined ranges, reproducible data collection, and practical screening procedures for sponsors and sites. The regulator will ask: make the trial easy to run. Do not make it impossible.
The sixth point is more philosophical. Does the model know what it does not know? This is, for me, the strongest aspect of the mathematical model Dr. Geraci designed. NetraAI does not force every patient into a subgroup. If a patient’s data do not support a stable or interpretable classification, they remain unknown. It is incredibly difficult to build a model that makes a genuine no-call rather than forcing an answer. That reduces coverage, yes. But coverage is not the objective. In high-stakes medicine, the hat no-call carries a real ethical commitment. As a physician, I have been in situations where the data did not fit, and the honest answer was: I don’t know. That is what the model does as well.
And the final question is whether the hypothesis can be tested prospectively. The variables, thresholds, missing data rules, estimates, and statistical hierarchy all need to be pre-specified. In the NIMH ketamine analysis published in npj Digital Medicine, conventional classifiers using the original clinical variables produced mean AUC values ranging from 0.27 to 0.61. After NetraAI identified an explainable 10-variable structure and a subgroup of 26 of the 63 evaluable treatment-period observations from a 33-participant crossover study, 81% of whom belonged to the favorable response class, the same classifiers produced mean AUC values of 0.82 to 0.87. Random forest, gradient boosting and the deep neural network each achieved a mean accuracy of 84.2% in nested cross-validation. These were internal, retrospective classification results using NetraAI-defined subgroup labels, like a like-for-like demonstration of improved prospective prediction of individual treatment benefit. The dataset was small, which limits generalizability, but the importance is that the retrospective analysis can generate a fixed rule that is then independently and prospectively tested. .
My practical requirement for an AI-derived responder profile is this: it must be treatment-specific, stable, interpretable, operationally measurable, and independently challenged. And one thing I always remind the team: before randomization, you can adjust, refine, and make decisions. Once a confirmatory trial is underway—and particularly after treatment information has been examined—the primary hypothesis cannot simply be altered without losing its prospective status. Prespecified adaptations, safety-driven changes and properly governed protocol amendments remain possible, but they must preserve trial integrity and statistical interpretability.
---
### [](#how-do-you-expect-ai-derived-patient-stratification-to-change-the-go-no-go-decision-at-the-end-of-phase-2-over-the-next-five-years)How do you expect AI-derived patient stratification to change the go/no-go decision at the end of Phase 2 over the next five years?
**Luca Pani:** This is usually the one question I do not answer because I have been burned by predicting AI timelines before. I have been doing this since 2017, so it is almost 10 years now, and I have been wrong about timing consistently. But I think the direction is clear.
The end-of-Phase 2 decision will no longer be a binary go/no-go conversation. It will become a structured set of development options. A sponsor may decide to go forward in the broad population, or go forward in a prospectively enriched and stratified population, or study both with hierarchical testing, or run a smaller validation study first, or go to the FDA and ask whether they agree with the plan, or change the endpoints, the duration, the dose, the site mix, or the stratification strategy, or seek a partner for a specific responder population, or stop the program because there is not enough average response. I think this is moving from a win-lose Phase 2 to a learn-and-lock Phase 2.
NetraMark held a non-binding Critical Path Innovation Meeting with FDA to discuss NetraAI’s proposed context of use, evidentiary expectations and possible pathways for sponsor-specific implementation. The feedback supported continued early engagement and consideration of established regulatory mechanisms, including Model-Informed Drug Development interactions where appropriate. The CPIM did not qualify or approve NetraAI, and it did not establish a single pathway applicable to every development program. That is exactly the direction you are asking about.
I think it will move faster than five years. The FDA has publicly reported more than 500 submissions containing AI components between 2016 and 2023. The FDA and EMA’s 2026 joint 10 Guiding Principles of Good AI Practice in Drug Development show the direction is clear: human-centric, risk-based, documented. AI will not lower the evidentiary bar. What it will do is enable better reasoning much earlier, before a company commits hundreds of millions of dollars and thousands of patients into a pivotal experiment.
For sponsors and investors, I expect systematic analysis of treatment-effect heterogeneity eventually to become a routine component of end-of-Phase 2 decision-making, just as sensitivity analyses and prospective statistical planning are today. That is where I think this is heading.
---
### [](#why-does-it-matter-ethically-that-a-subgroup-is-later-found-to-have-benefited-from-a-drug-that-failed-its-broad-phase-3)Why does it matter ethically that a subgroup is later found to have benefited from a drug that failed its broad Phase 3?
**Luca Pani:** This is the question I like most, because it brings us back from algorithms and models to people. I have lived through this.
When a later analysis shows a subgroup has a favorable average treatment effect, we cannot tell individual patients and their families that they personally would have benefited. Treatment effect is always counterfactual. We observe what happens under the treatment a person receives. We cannot observe what would have happened to the same person at the same time under the alternative. Some subgroup members may have received the investigational drug and improved. Some may have been on placebo and received no benefit. Some may not have responded even though they fit the profile. The model-derived subgroup changes probability. It does not create individual certainty.
That creates an obligation. We cannot convert an exploratory retrospective signal into a personal medical promise. But we also cannot stay indifferent. We have to analyze the data rigorously. On August 27, 2026, NIH released a draft policy for public comment that, if finalized, would require investigators and institutions to share plain-language, summary-level results with participants in NIH-supported clinical research, subject to defined exceptions. The Declaration of Helsinki already contains this: the concept of the participant as a partner. In Modena, through the EU-funded FACILITATE project, conducted from 2022 to 2026, which examined patient-centered and GDPR-compliant approaches to returning clinical-trial data and information to participants, , we worked specifically on returning information and data to study participants. This is something I want people to take from this conversation.
AI cannot be allowed to increase inequity. An AI-derived subgroup should not automatically become an exclusion rule. A sponsor may study only an enriched population when the scientific evidence and regulatory strategy justify it, but that decision must be prospective and must explicitly address assay performance, uncertainty, unmet need, safety, generalizability and what can or cannot be inferred about patients outside the selected group. We do not have sufficient knowledge to absolutely exclude any fellow human from the possibility of responding to a treatment, just because a machine says no. That might happen. The solution is to stratify based on the NetraMark analysis, define a stratum that is representative enough, pre-specify your primary analysis, and then analyze all comers. Nor can label breadth be inferred simply because placebo was not superior to drug in the non-selected stratum. Absence of evidence of harm is not evidence of efficacy. Labeling depends on the totality of prospectively specified evidence, including the treatment effect and its precision within each population, any treatment-by-subgroup interaction, safety, benefit-risk, subgroup prevalence and the population actually studied.
You cannot say the machine told you to do it. There is a human in the loop. That person is taking responsibility and signing on to it. In NetraAI, humans control, check, and challenge all of it, and it is going to stay that way.
The average patient is a statistical construct. It is not a biological reality. The future of clinical development is not to abandon randomized trials or replace statistics. It is to make those trials more intelligent: identify heterogeneity earlier, translate it into a transparent hypothesis, test that hypothesis prospectively, and go to the FDA early and often. Patient selection is a lever. It is not destiny.
> “The average patient is only a statistical construct. It’s not a biological reality.”
*Dr. Luca Luca Pani is Chief Innovation and Regulatory Officer at NetraMark. He previously served as Director General of the Italian Medicines Agency and as a member of the European Medicines Agency Management Board, the Committee for Medicinal Products for Human Use and the Scientific Advice Working Party.*
---
**Categories:** Article: Executive Interviews
---
### [The CRO Isn't Failing Your Trial. Your Governance Model Is.](https://www.clinicaltrialvanguard.com/clinical-bellwether/the-cro-isnt-failing-your-trial-your-governance-model-is/)
**Published:** June 16, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Sponsors treat CRO contract signing as a handoff. Three independent clinical ops leaders say that abdication, not bad molecules, is killing small biotech…
**Content:**
Robert [Goldman](https://www.linkedin.com/posts/robert-s-goldman_potentially-an-unpopular-opinion-sponsors-activity-7470788178434609152-3uvb) has run global clinical operations long enough to know what a failing trial looks like before the data readout does. And his diagnosis, shared publicly this week and drawing [208 likes and 66 comments](https://www.linkedin.com/posts/robert-s-goldman_potentially-an-unpopular-opinion-sponsors-activity-7470788178434609152-3uvb), is not flattering: sponsors who treat a signed work order as a substitute for leadership are the architects of their own trial disasters. “The worst studies had sponsors who thought a signed work order was a substitute for leadership,” Goldman wrote. “No CRO partnership is stronger than the sponsor behind it.” That sentence landed with the force of something the industry has known privately for years but refused to say in print.
The post triggered a convergence. Within hours, two other credible clinical operations voices were publishing their own versions of the same uncomfortable argument. The overlap was not coordinated. That is what makes it significant.
What these practitioners are describing is not a vendor performance problem. It is a governance deficit sitting at the center of an industry that has convinced itself delegation and accountability are the same thing.
## [](#the-handoff-illusion)The Handoff Illusion
Here is the scenario playing out across small biotech portfolios right now. A sponsor completes a competitive CRO bid process, awards the contract, celebrates the milestone internally, and then progressively reduces its own operational involvement over the following quarter. Weekly calls replace strategic engagement. Status reports replace site-level scrutiny. The CRO says “we’re on track” and the sponsor, lacking the internal expertise to interrogate that claim, accepts it. Six months later, enrollment is behind, the TMF has gaps no one caught in real time, and the root cause review points everywhere except at the governance model that made the failure invisible until it was expensive.
Goldman’s framing cuts through the industry’s polite euphemisms. CROs are hired to extend sponsor capabilities, not to replace sponsor leadership. The moment a sponsor confuses those two functions, the trial is running without a captain.
The deeper problem is structural, and [Elena Sinclair identified it with precision](https://www.linkedin.com/posts/elena-sinclair_a-failed-endpoint-wont-end-your-clinical-activity-7470852424329650176-3Heo): most small biotechs do not fail because the molecule was wrong. They fail because no one sponsor-side was qualified to challenge the trial. That distinction matters enormously for how the industry allocates blame and, more importantly, how it allocates investment in internal capability.
[Sinclair](https://www.linkedin.com/posts/elena-sinclair_a-failed-endpoint-wont-end-your-clinical-activity-7470852424329650176-3Heo) cited Tufts CSDD data showing only one in five sponsors rate their oversight processes as highly effective. Read that number carefully. Four out of five sponsors, by their own assessment, are operating oversight processes they do not consider highly effective. Yet the industry continues to treat CRO award as the end of the governance conversation rather than the beginning of it.
## [](#the-applied-therapeutics-warning)The Applied Therapeutics Warning
The regulatory system has already answered the question of where accountability lives when delegation goes wrong. Sinclair’s post surfaces the example the industry should be studying in every clinical operations training: Applied Therapeutics. A vendor deleted 47 patients’ primary endpoint data. The FDA’s warning letter went to Applied Therapeutics, not the CRO. Delegation did not transfer accountability. The sponsor owned the data integrity obligation regardless of which organization’s employees were touching the database.
This is not a novel regulatory position. ICH E6(R2), the GCP guideline governing sponsor responsibilities, is explicit that sponsors retain ultimate responsibility for the quality and integrity of trial data regardless of which tasks have been delegated to a CRO. The FDA’s 2023 guidance on oversight of clinical investigations reinforces the same principle: sponsors must establish and maintain oversight processes sufficient to verify that delegated functions are being performed. The Applied Therapeutics case is what happens when those processes exist on paper and nowhere else.
Sinclair’s diagnostic questions should be read as a protocol for every small biotech board meeting that touches clinical operations. What is your eTMF completeness percentage right now? Who is the CRA at your top-enrolling site, and how experienced are they? When did you last review a monitoring visit report, not just receive it? Did your [ClinOps](https://www.clinicaltrialvanguard.com/conference-coverage/advancing-clinops-excellence-the-15th-annual-scope-summit-2024/) lead design the governance plan, or did the CRO hand it to you? If a sponsor cannot answer those questions in real time, it does not have oversight. Per Sinclair’s post, it has an account relationship.
The efficiency case for CROs is real and should not be dismissed. Sinclair’s post acknowledges that study initiation can be 77 days faster when experienced CROs have established site relationships. Speed and operational capability are legitimate reasons to outsource execution. But execution efficiency without strategic oversight does not produce better trials. It produces faster-moving problems.
## [](#the-expertise-gap-no-one-budgets-for)The Expertise Gap No One Budgets For
The counterintuitive reality that Goldman and Sinclair are both circling is this: the sponsors most dependent on CRO capability are precisely the sponsors least equipped to oversee it. Small biotechs operating lean, pre-commercial teams frequently lack a single internal employee qualified to evaluate CMP adequacy, interrogate CRO staffing turnover patterns, or conduct a meaningful TMF audit. They hire a CRO because they cannot afford to build the function internally, and then they discover, too late, that overseeing a CRO competently requires nearly the same expertise as running the function yourself.
Goldman’s observation that the best studies he has seen had sponsors deeply engaged in strategy, governance, decision-making, and oversight is not a prescription for redundancy. It is a description of what effective partnership actually requires. The sponsor brings therapeutic knowledge, regulatory strategy, and accountability that no CRO can contractually absorb. The CRO brings operational scale, site networks, and execution infrastructure. Neither half of that equation functions without the other, and the market has spent a decade pretending it can.
[Vincent Thompson’s broader framing](https://www.linkedin.com/posts/vincent-thompson-md-phd-chcrc_edition-194-summary-the-clinical-research-activity-7471245636298952704-kLNq) adds a layer that the operational posts do not address directly: the clinical research ecosystem has achieved [extraordinary](https://www.linkedin.com/posts/vincent-thompson-md-phd-chcrc_edition-194-summary-the-clinical-research-activity-7471245636298952704-kLNq) things through collective commitment, but the question worth asking is whether existing structures can be completed rather than replaced. Applied to the CRO governance crisis, that framing points toward a specific gap. The ecosystem is not broken. The accountability layer connecting sponsor strategy to CRO execution has been left structurally incomplete, and the industry has tolerated that incompleteness because it is expensive to fix and invisible until it catastrophically fails.
The FDA does not inspect a CRO’s intentions. It inspects sponsor oversight. When the only person truly managing a trial sits inside the CRO, the sponsor has not bought execution capacity. It has purchased a very expensive regulatory liability dressed in the language of partnership. Small biotechs without internal ClinOps leadership qualified to own that oversight have one structural option that Sinclair names directly: a fractional Head of Clinical Operations whose job is not to manage the CRO but to prevent the sponsor from pretending oversight exists when it does not.
The governance model is the trial. Every sponsor that has not internalized that reality is one warning letter away from learning it the hard way.
## [](#references)References
1. [Robert S. Goldman, LinkedIn post on sponsor vs. CRO leadership roles (2026)](https://www.linkedin.com/posts/robert-s-goldman_potentially-an-unpopular-opinion-sponsors-activity-7470788178434609152-3uvb)
2. [Elena (Ella) Sinclair, LinkedIn post on CRO governance failure and Applied Therapeutics (2026)](https://www.linkedin.com/posts/elena-sinclair_a-failed-endpoint-wont-end-your-clinical-activity-7470852424329650176-3Heo)
3. [Vincent Thompson MD PhD, LinkedIn post on clinical research ecosystem evolution (2026)](https://www.linkedin.com/posts/vincent-thompson-md-phd-chcrc_edition-194-summary-the-clinical-research-activity-7471245636298952704-kLNq)
**Categories:** Clinical Bellwether
**Tags:** Clinical Operations, Clinical Trial Governance, CRO Oversight, Small Biotech, Sponsor Accountability
---
### [The 90% Automation Promise Means Nothing to a Coordinator Running 12 Systems](https://www.clinicaltrialvanguard.com/clinical-bellwether/the-90-automation-promise-means-nothing-to-a-coordinator-running-12-systems/)
**Published:** September 22, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Medable's CEO wants 90% of clinical trials automated by 2030. Site coordinators are still manually bridging 12 disconnected systems. Both are true, and that's…
**Content:**
Picture a screening visit at a busy research site. A coordinator sits across from a patient, laptop open, scanner nearby, eSource tool loading. She is maintaining eye contact, keeping the patient calm and engaged, while simultaneously logging into system four of twelve. [John Oncea](https://www.linkedin.com/posts/johnoncea_clinical-trial-sites-dont-need-more-tech-activity-7506362958558535680-ZV_X), Chief Editor at Clinical Tech Leader, [reported this scene](https://www.linkedin.com/posts/johnoncea_clinical-trial-sites-dont-need-more-tech-activity-7506362958558535680-ZV_X) after conversations with Nick Spittal and Raghu Punnamraju of Velocity Clinical Research: 29 discrete steps to complete a single screening, 17 of them system-facing, only 12 involving the patient directly. The workaround had become so routine it barely registered as a problem. It should.
That same week, [Ryan Flinn](https://www.linkedin.com/posts/rsflinn_everyones-talking-about-ai-in-drug-discovery-activity-7507895564416512000-amch) [published an interview](https://www.linkedin.com/posts/rsflinn_everyones-talking-about-ai-in-drug-discovery-activity-7507895564416512000-amch) with Medable co-founder and CEO Michelle Longmire, who articulated her goal plainly: 90% of clinical trial operations automated by 2030. One benchmark she cited: site visit paperwork that currently consumes eight hours of staff time reduced to 30 minutes of AI-assisted review. The vision is serious. The timeline is aggressive. And it exists in an entirely different world than the one that coordinator is navigating.
This is the central dysfunction of AI in clinical trials right now. The boardroom and the site floor are not having the same conversation, and the gap between them is widening with every vendor pitch that leads with the word “automation.”
## [](#what-recruitment-ai-actually-runs-into)What Recruitment AI Actually Runs Into
The EHR-matching hypothesis was elegant: clinical data already exists, AI can read it, match patients to eligibility criteria, and compress enrollment timelines from years to months. For broad-population therapies, Irina Gontschar, MD, CEO of Potter Research Solutions, [acknowledges the model can work well](https://www.linkedin.com/posts/irina-gontschar-md-by-phd-acrp-cp-54abb475_ai-recruitment-clinicaltrials-activity-7507060851821023232-qG_m). But she draws a hard line at oncology, hematology, and rare diseases, where a diagnosis code in an EHR does not confer eligibility. A rare mutation has to be contextualized within a patient’s full clinical history. That requires a physician or a highly qualified research professional, not a matching algorithm.
The deeper problem Gontschar identifies is that early AI recruitment tools solved for the wrong bottleneck. Finding a potential participant was never the primary source of delay. The real friction accumulates after identification: EHR review, clinical history reconstruction, records collection, additional testing, eligibility confirmation, and all the communication in between. These are the repetitive, time-intensive tasks that qualified humans are currently absorbing, and they are exactly where AI with appropriate EHR access could provide genuine relief without displacing clinical judgment.
There is also a biological reality that no algorithm overrides. A therapy requiring six months of investigational drug administration followed by two years of overall survival follow-up cannot be compressed regardless of how quickly the right patient is found. Faster recruitment cannot eliminate protocol-defined time. The sponsors who understood this distinction early are the ones building AI tools calibrated to the actual bottleneck, not the visible one.
Which raises the question Gontschar leaves open: if the real opportunity is in post-identification workflow automation, why has so much capital and attention landed on the matching problem? Partly because matching is legible. It is demonstrable in a sales deck. A reduced time-to-enrollment metric looks clean on a slide. The messier work of EHR reconstruction and records collection is harder to quantify and less flattering to investors. The incentive structure, in other words, has been pointing AI development in the wrong direction.
## [](#the-integration-problem-nobody-wants-to-invoice-for)The Integration Problem Nobody Wants to Invoice For
[Jenn Pages, CCRP](https://www.linkedin.com/posts/jennpages_i-do-not-care-how-advanced-your-clinical-activity-7506661095621779456-arVr), CEO and Founder at her site consultancy, [puts the operational reality without decoration](https://www.linkedin.com/posts/jennpages_i-do-not-care-how-advanced-your-clinical-activity-7506661095621779456-arVr): “I do not care how advanced your clinical trial technology is if it makes the site’s job harder.” Her inventory of systems a coordinator is already required to manage includes a CTMS platform, EDC, ePRO tools, a randomization system, lab portals, imaging vendors, a safety database, an eTMF system, and sponsor-specific platforms. At some point, she writes, a “technology-enabled trial” starts to sound like “please remember 11 passwords.”
The Velocity Research data Oncea surfaced makes this concrete. Seventeen of 29 screening steps were system-facing. A coordinator counting logins, uploads, transcriptions, and cross-checks before she can spend meaningful time with the patient. That is a technology stack functioning as a data-entry burden rather than a clinical support system. The coordinator becomes the integration layer, manually moving information between platforms that were never designed to talk to each other.
AstraZeneca appears to be reading the same signals at the institutional level. [Danielle Bitterman](https://www.linkedin.com/posts/danielle-bitterman-87834b16b_artificialintelligence-airesearch-clinicaldevelopment-activity-7507812334544023553-1lw7), VP of AI for Clinical Development, [posted an open role this week](https://www.linkedin.com/posts/danielle-bitterman-87834b16b_artificialintelligence-airesearch-clinicaldevelopment-activity-7507812334544023553-1lw7) for a senior leader to build and lead a Clinical AI Research team covering Phase I through III, with an explicit focus on foundation models, trial simulation, causal modeling, and agentic systems. The framing is notable: AstraZeneca is not looking for someone to deploy existing AI tools. It is investing in the science and methods of AI for clinical development as a distinct research discipline. That signals the company recognizes current tools are insufficient for the problem at hand.
[Benjamin Vandendriessche](https://www.linkedin.com/posts/benjaminvandendriessche_back-from-dpharm-disruptive-innovations-activity-7507015737836847104--l-M), CEO of the Digital Medicine Society, returned from DPHARM and CTTI meetings with a diagnosis that cuts through the enthusiasm: [regulatory momentum is real](https://www.linkedin.com/posts/benjaminvandendriessche_back-from-dpharm-disruptive-innovations-activity-7507015737836847104--l-M), including FDA’s Digitally-derived Measures white paper and Operation Trialblazer, but adoption of the underlying tech stack remains slow. His framing deserves attention: “Access to technology is not the main blocker anymore.” The blocker is deployment intelligence, which means understanding incentive structures clearly enough to know where a tool will actually reduce friction versus where it will simply relocate it. Per Vandendriessche’s post, more than 80% of health systems have no clear AI governance system in place even as AI-based systems are already rolling out. Clinical trials are not exempt from that gap.
Pages and Oncea are both pointing at the same structural failure from different angles. Sponsors and vendors have optimized for feature sets and demo impressions rather than workflow integration. The coordinator who spent 17 of 29 screening steps inside disconnected systems is not a technology problem. She is an incentive problem. Nobody in the contracting chain is accountable for her total system load. The sponsor pays for the EDC. A separate vendor provides the ePRO. The imaging vendor runs its own portal. Nobody owns the seam between them, so the coordinator fills it with manual effort, every study, every site, every day.
## [](#where-the-pressure-has-to-land)Where the Pressure Has to Land
Medable’s 90%-by-2030 target is a useful north star precisely because it forces a binary question: if nearly all trial operations are to be automated within five years, which operations are on the list, and who is accountable for the integration that makes automation possible? An eight-hour site visit documentation process reduced to 30 minutes of AI review is achievable if the underlying systems share data reliably. It is not achievable if the coordinator is still manually transcribing between platforms to create the input the AI is supposed to review.
Gontschar’s argument points toward the model that actually has leverage: AI paired with EHR access, handling the repetitive post-identification workflow, while experienced clinical professionals retain judgment over eligibility determination. That is not a vision of replacement. It is a vision of appropriate task allocation, which is a harder organizational problem than building the algorithm. It requires sponsors, CROs, and technology vendors to agree on data standards, access permissions, and accountability structures before the AI layer can deliver anything meaningful at scale.
Pages has already drawn the evaluation framework sponsors should be using: does it reduce duplicate entry, save coordinator time, make patient visits easier to manage, and communicate with systems already in use? If the answer to any of those is no, the technology is a cost center wearing an innovation label. The standard, as she puts it, is not how impressive the demo looks. The standard is whether it makes the study easier to run.
AstraZeneca building a dedicated clinical AI research function suggests at least one large sponsor is taking the methods problem seriously enough to fund it internally. The question is whether the rest of the industry waits for that work to surface in publications and partnerships, or starts auditing their own coordinator workflows now, before the next platform lands on an already overloaded site stack.
The coordinator maintaining eye contact with a patient while logging into system eight of twelve is not waiting for a better demo. She is waiting for someone with budget authority to count her steps and decide that 29 is unacceptable.
## [](#references)References
1. Gontschar, I. (2026). [When AI Meets the Real Complexity of Patient Recruitment. LinkedIn.](https://www.linkedin.com/posts/irina-gontschar-md-by-phd-acrp-cp-54abb475_ai-recruitment-clinicaltrials-activity-7507060851821023232-qG_m)
2. Pages, J. (2026). [I do not care how advanced your clinical trial technology is. LinkedIn.](https://www.linkedin.com/posts/jennpages_i-do-not-care-how-advanced-your-clinical-activity-7506661095621779456-arVr)
3. Oncea, J. (2026). [Clinical Trial Sites Don’t Need More Tech. LinkedIn.](https://www.linkedin.com/posts/johnoncea_clinical-trial-sites-dont-need-more-tech-activity-7506362958558535680-ZV_X)
4. Flinn, R. (2026). [Everyone’s talking about AI in drug discovery. LinkedIn.](https://www.linkedin.com/posts/rsflinn_everyones-talking-about-ai-in-drug-discovery-activity-7507895564416512000-amch)
5. Bitterman, D. (2026). [The frontier of AI for drug development. LinkedIn.](https://www.linkedin.com/posts/danielle-bitterman-87834b16b_artificialintelligence-airesearch-clinicaldevelopment-activity-7507812334544023553-1lw7)
6. Vandendriessche, B. (2026). [Back from DPHARM: Disruptive Innovations to Modernize Clinical Trials. LinkedIn.](https://www.linkedin.com/posts/benjaminvandendriessche_back-from-dpharm-disruptive-innovations-activity-7507015737836847104--l-M)
**Categories:** Clinical Bellwether
**Tags:** AI in Clinical Trials, Clinical Trial Technology, digital transformation, patient recruitment, Site Operations
---
### [Asia Pacific Is No Longer a Backup Plan. Sponsors Who Still Treat It That Way Are Falling Behind.](https://www.clinicaltrialvanguard.com/executiveinterviews/asia-pacific-is-no-longer-a-backup-plan-sponsors-who-still-treat-it-that-way-are-falling-behind/)
**Published:** September 3, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Precision for Medicine's James Cheong and JingPing Yeo on what has actually changed in APAC clinical operations, and what biotechs still get wrong when they arrive.
**Content:**
 James Cheong
Precision for Medicine *Precision for Medicine’s James Cheong and JingPing Yeo on what has actually changed in APAC clinical operations, and what biotechs still get wrong when they arrive.*
---
For years, Asia Pacific sat at the edges of global development strategy: a region sponsors turned to when Western enrollment stalled, not one they built plans around from the start. That calculus has shifted, and the shift is structural. China’s phase three startup time now runs roughly comparable to Europe’s. APAC’s share of global phase three sites has grown by double digits over the past two decades. More than a third of the world’s new drug compounds now originate from the region. Yet many sponsors, particularly biotechs entering APAC for the first time, are still navigating this environment with assumptions that belonged to a decade ago. James Cheong, Senior Vice President for Asia Pacific, and JingPing Yeo, Vice President for Clinical Solutions at Precision for Medicine, sat down with Vanguard to explain what changed on the ground, why adding sites has not always translated to enrollment gains, and where the blind spots are for sponsors who think they already know the region.
### [](#how-did-you-both-watch-sponsor-perception-of-apac-shift-during-your-careers)How did you both watch sponsor perception of APAC shift during your careers?
**James Cheong:** When I first started out in the industry, the Asia region was very much a nice-to-have option for clinical trials. Big markets like China existed, but they took one to two years to start, so they never got to engage in global phase three trials. When a new drug had to be launched in China, companies would do a separate, China-only registration trial.
Things have changed a lot over the past 10 years, in a very positive way. China is now considered by default as part of the global development plan by many large pharma companies, no longer a separate plan once the drug is approved elsewhere. That means Chinese patients are able to access new drugs at the same time as patients in the US or Europe, and we are talking about many, many patients who can benefit from this.
---
**JingPing Yeo:** The perceptions James described were historical. Sponsors remembered the fragmented regulatory requirements from years ago, the variable site experience with multinational protocols, the translation burden, the limited in-country infrastructure. But those things existed at a country or site level, and they are no longer a reason to exclude APAC as a region.
What we have also seen is a lot of data showing a strategic shift. APAC’s share of phase three sites has increased substantially, while the US share has declined. That reflects how the region has evolved, and how it continues to contribute to diversified recruitment, broader patient population pools, and better alignment of development pathways.
---
### [](#why-has-expanding-the-number-of-apac-sites-not-produced-a-proportional-increase-in-enrolled-patients)Why has expanding the number of APAC sites not produced a proportional increase in enrolled patients?
**James:** Trial complexity has been increasing, which makes it more difficult to recruit patients to begin with. And as more sponsors have recognised the advantages of running trials in Asia, there is now more competition for patients, particularly in heavily researched indications.
---
**JingPing:** We no longer design studies looking at a volume of 50 separate protocols. We try to minimize the number of studies and add multiple endpoints to each one, which drives protocol complexity. That often leads to identifying only a niche pool of eligible patients, and as a result the number of patients enrolled per site may come down.
There is also the issue of leading investigators becoming oversubscribed. Many sponsors are targeting the same top sites and the same leading investigators, who are then managing multiple competing studies simultaneously. In the past, a site might allocate 10 patients to a single protocol. Now those same 10 patients may be distributed across two or three protocols, which creates a perceived decline in per-site enrollment that becomes increasingly visible.
---
### [](#how-did-china-get-its-phase-three-startup-time-down-to-where-it-is-today)How did China get its phase three startup time down to where it is today?
**James:** China underwent significant regulatory reform about 10 years ago, with a very decisive move to strengthen the entire clinical trial and drug registration environment. They made it clear they would not tolerate poor-quality trials or poor-quality dossiers. Penalties for data that did not stand up to scrutiny were severe, and as a result many companies voluntarily withdrew applications that were not meeting global standards. That alone reduced the burden on the regulator. At the same time, they hired more reviewers. With that two-pronged approach, they were already able to speed up the review timeline for INDs and NDAs.
But they did not stop there. They have continuously improved. The latest change, implemented just last September, means that in the best case, an IND can now be approved in 30 days, provided the dossier is already in Chinese and the leading investigator agrees to take on added responsibility in risk management. That applies to Chinese biotech clients where there is no need for translation. For a US company, the timeline still runs two to three months for translation. But the government has been very decisive and consistent in implementation, and that has made a significant difference.
---
**JingPing:** The government’s commitment has also come with real infrastructure on the ground. Hospitals are more prepared, investigators are more willing to engage early, and the SMO and recruitment agency ecosystem is now well-established in China to support trial operations.
The other thing I want to highlight is what quality actually means now. As more APAC countries, including China, have become ICH member states, quality is being assessed through inspection readiness, data integrity, source documentation, and patient protection standards. That is being taken seriously at the country and site level. At Precision for Medicine, what we do is continuous central data review and risk-based monitoring so that sites are always ready, not just when an inspection is announced. It is about getting sites ready to do quality work from the start, not just hitting a startup timeline.
---
 JingPing Yeo
Precision for Medicine ### [](#how-should-sponsors-weigh-the-fdas-different-inspection-procedures-when-they-are-evaluating-apac-site-data)How should sponsors weigh the FDA’s different inspection procedures when they are evaluating APAC site data?
**JingPing:** I do not see that there is much difference anymore. When APAC sites contribute data to global applications, they are already familiar with FDA inspections. And within each APAC country, the local regulators conduct similar inspections and audits under comparable guidelines. Many of these sites have been through it repeatedly and are always inspection ready. At Precision for Medicine, we look at centralised monitoring and continuous data review as day-to-day practice, not something we do only when an inspection is announced.
---
**James:** I would add that unannounced inspections are actually very common in China now. The Chinese regulatory agency conducts inspections with very short notice, and the sites are used to it. For US FDA inspections in China, advance notice is still given because of the practical requirements around travel, language, and translation. But what matters is the outcome. We looked at FDA inspection results from 2009 through 2016, across all three categories: no action indicated, voluntary action indicated, and official action indicated. Asia as a whole, a large part of which reflects China given the volume of trials there, fares comparably with the US and Europe. They are not the worst-performing region by any measure. I think that speaks well of how sites across Asia are running trials.
> “Asia as a whole, a large part of which reflects China given the volume of trials there, fares comparably with the US and Europe. They are not the worst-performing region by any measure.”
---
### [](#how-do-you-see-apacs-strategic-role-in-global-development-evolving-over-the-next-five-years)How do you see APAC’s strategic role in global development evolving over the next five years?
**JingPing:** The shift is most pronounced in oncology. More than 50% of studies globally are looking at oncology indications, and APAC remains critical for the substantial cancer incidence numbers and the strength of its specialty centres, in China and across the broader region. The countries and sites have matured, genomic testing capability is advancing rapidly, and investigators have accumulated significant experience in registration studies.
For China specifically, the China-originated companies are also becoming increasingly important global developers. More than a third of innovative drugs now come from China-based companies, and that accounts for a growing share of early clinical activity globally. For smaller sponsors, the risk is not simply missing a lower-cost geography. It is falling behind larger competitors who are already leveraging first-mover advantages in terms of timelines and the breadth of global evidence they are building. That is the biggest trend I expect to continue. Beyond that, how AI and digital technologies get applied to improve phase three execution is something we will all be watching closely.
---
**James:** I am in alignment with JingPing. The APAC market will continue to become more important. China is already the second largest pharma market and the fastest growing. Japan is the fourth largest. Korea and Australia are significant markets. As these markets grow, pharma companies will place more importance on engaging KOLs during the trial phase, particularly in oncology, where there are more pronounced ethnic differences in the types of side effects observed and where physician familiarity with a drug at launch genuinely matters for patient outcomes.
The other piece is the growth in R&D activity across the region. One-third of new compounds globally now come from Asia Pacific, and in some areas, like antibody-drug conjugates, more than half originates from this region. With the research and the originators coming from here, more sites will naturally be involved. I think that trend continues into the foreseeable future.
“You need to change the mindset of APAC as an outsourced geography or contingency plan, and move to seeing it as an integrated engine for your global development.”
---
> “One more point on regulatory planning: some biotechs coming from the US or Europe do not know that China requires local patients in the pivotal phase three trial in order to file for NDA in China.”
### [](#why-do-biotechs-coming-to-china-for-the-first-time-often-struggle-compared-to-sponsors-who-know-the-region)Why do biotechs coming to China for the first time often struggle compared to sponsors who know the region?
**James:** The first thing I would say is that biotechs need to find a CRO with real knowledge, expertise, and an understanding of how trials are run in this part of the world. The culture is very different. How you engage a site in the US or Europe is very different from how you do it in China, and if you take the same approach, it will not be too successful.
I can give a concrete example. Biotechs will sometimes identify KOLs through internet searches and conference attendance, then try to approach them by email out of the blue. They get very little response. But if they engage a local CRO that has the network and can reach out through the right platform, through WeChat, in the local language, the response is immediate. Where a company on its own cannot get an appointment with a KOL, a CRO with established relationships can arrange a meeting the next day. Understanding what works in the US may not always translate to China or elsewhere in Asia Pacific is essential. You have to listen to the experts on the ground.
---
**JingPing:** Finding the right service provider is critical, but it has to happen at the right moment. The most important point is before the protocol and operational assumptions are locked. That means starting with evidence-based feasibility: looking at how to build in the right biomarkers, examining standard of care, competing trials, referral patterns, molecular testing availability, sample and support requirements, investigator capability, and building a realistic enrollment forecast.
What is distinctive about working in APAC is the fully integrated nature of what needs to happen: regulatory strategy by country, site activation, lab planning, biospecimen logistics, investigational product logistics, and patient identification, all forming one composite critical path. A good service provider helps smaller biotechs navigate that entire path. Ultimately, the goal is the same regardless of company size: assess the right patients, ensure solid biomarker operations and quality oversight, and avoid late-stage surprises that compromise data quality. The mindset shift that still needs to happen for a lot of emerging biopharm companies is moving away from treating APAC as an outsourced geography or a contingency rescue plan, and toward treating it as an integrated engine for global development.
---
**James:** One more point on regulatory planning: some biotechs coming from the US or Europe do not know that China requires local patients in the pivotal phase three trial in order to file for NDA in China. The same applies to Japan, and there are related requirements in Taiwan, South Korea, and India. If a CRO is not advising them on that in the planning stage, they can reach the end of a trial and realise they do not have the data to file for marketing authorization in China or Japan. That is where having expertise on the ground from the beginning makes a real difference.
*James Cheong is Senior Vice President for Asia Pacific at Precision for Medicine. JingPing Yeo is Vice President for Clinical Solutions, responsible for clinical delivery across APAC.*
---
**Categories:** Article: Executive Interviews
---
### [The Protocol-Only Coverage Analysis: What a Site Can Defend Before the Budget Arrives](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-protocol-only-coverage-analysis-what-a-site-can-defend-before-the-budget-arrives/)
**Published:** September 22, 2026
**Author:** Krishma Shah
**Excerpt:** A protocol-scoped MCA is a real deliverable, but it has hard limits. Here is what you can and cannot defend at feasibility.
**Content:**
*THE BILLING GRID*
A weekly column on research billing and coverage analysis, by Krishma Shah
The budget has not arrived. The contract is somewhere in legal. The sponsor is asking whether your site can activate in 45 days. And your compliance officer wants a coverage analysis before anyone touches a patient. This is the feasibility moment that exposes how well a site actually understands research billing, because the tool you need is a protocol-only Medical Coverage Analysis, and most sites either skip it or do it wrong.
A Medical Coverage Analysis, or MCA, is the document that maps every protocol-required procedure to one of three designations: standard of care (SOC, meaning the procedure is billable to Medicare or a commercial insurer when the trial qualifies), research-paid (RES, meaning the sponsor owns the cost), or non-billable and invoiceable (NB/INV, meaning the item appears in the site budget as a line to be negotiated). The MCA is not a budget. It is the coverage logic that tells you whether a charge can legally touch a patient’s insurance at all.
At feasibility, you do not have a budget, but you do have a protocol. A protocol-scoped MCA uses that protocol alone to build the visit-grid designations for every procedure at every visit. The result is a defensible first-pass map of coverage logic. It is a real deliverable, not a placeholder, and it belongs on the site-activation critical path alongside the IRB submission and the clinical trial agreement, because the billing team cannot set up the charge router without it.
## [](#what-the-defensible-protocol-only-mca-can-say)What the Defensible Protocol-Only MCA Can Say
The core unit of defensibility in any MCA is the visit-grid cell: the intersection of a specific procedure and a specific visit. The same blood draw can be SOC at the screening visit and RES at a mid-cycle safety visit, depending on what the protocol requires versus what routine clinical care would require for this patient population. A protocol-only MCA can assign designations at the cell level for every procedure the protocol specifies, because the protocol is the source of truth for what is research-required versus what would happen in standard practice regardless of trial participation.
Coverage citations are the other thing a protocol-only MCA can do correctly. If the protocol requires a complete blood count at every visit, the MCA should cite NCD 190.15, the National Coverage Determination that governs laboratory diagnostic services including CBCs. Writing “routine labs” with no citation is the single most common audit weak point in MCAs reviewed across site networks. The citation is not a formality; it is the evidence that ties your SOC designation to an actual Medicare coverage rule. A protocol-only MCA can and should include those citations, because they come from the procedure type, not from the budget or the charge master.
Qualifying trial status is another element the protocol-only MCA can address. NCD 310.1 governs routine cost coverage in qualifying clinical trials. A trial qualifies when it is conducted under an Investigational New Drug application and meets the therapeutic intent and Medicare benefit category criteria that NCD 310.1 requires. The protocol tells you whether the trial is IND-governed and whether the intervention is therapeutic. The MCA can flag deemed status at feasibility so the billing team knows which visits can route SOC charges to Medicare at all.
## [](#what-the-protocol-only-mca-cannot-finalize)What the Protocol-Only MCA Cannot Finalize
The INVOICEABLE section of the MCA is where the protocol-only version hits its limit. The NB/INV designation means an item is not billable to insurance and must appear as a negotiated line in the sponsor budget. Pricing those lines requires the charge master, and you do not have it in a protocol-only pass. The budget reconciliation step, which happens when the sponsor’s proposed budget arrives, prices each research-paid item at the greater of the sponsor’s proposed rate or the site’s charge master rate. That arithmetic cannot happen without both inputs. The protocol-only MCA flags which cells will need INVOICEABLE pricing; it cannot set the price.
Device trials add another layer the protocol-only MCA must handle carefully. When a protocol involves an investigational device, the CMS coverage determination and the IDE approval letter control whether the device and its associated procedures are covered as Category A (experimental, generally not covered) or Category B (non-experimental or investigational, covered when medically necessary). The protocol alone may not tell you which category applies; the IDE approval letter does. Flag the device items in the protocol-only MCA and note that Category A versus B designation is pending the IDE letter. Do not designate them SOC without it.
## [](#the-imaging-problem-that-templates-cannot-solve)The Imaging Problem That Templates Cannot Solve
Imaging is the place where protocol-only MCAs fail most reliably, and it is worth spending time here because the error pattern is consistent. A protocol will often specify a scan schedule as a parent rule: CT of the chest, abdomen, and pelvis every eight weeks for nine cycles. That is nine discrete visit events, each of which needs its own cell designation in the visit grid.
What happens in practice is that an MCA template pulls the scan type as a single line item and assigns one designation. The nine-visit schedule collapses into one cell. The result is an MCA that appears to cover imaging but actually designates only one of the nine visits. The other eight scans have no coverage assignment at all when the billing team routes charges.
A worked example makes the stakes concrete. Suppose a Phase II oncology protocol requires CT imaging every eight weeks for nine cycles, and scans one through three are SOC by clinical standard for this disease and line of therapy, while scans four through nine are protocol-required surveillance beyond standard practice and therefore RES. A correct protocol-only MCA produces nine separate cells in the visit grid, with different designations at cycle four. A template that collapses all nine into one line will either overbill Medicare for the RES scans or leave the sponsor budget negotiation without the correct number of research-paid line items. Either error is a compliance exposure. The Rush University Medical Center billing settlement in 2005 established that billing Medicare for items the sponsor had agreed to pay is not a technical error; it is a false claims risk. The fact pattern there involved exactly this kind of misalignment between research cost obligations and insurance billing. Imaging frequency errors reproduce that fact pattern at scale.
Human review of the visit schedule against the protocol schedule of assessments is the only reliable catch for this error. No template reads parent schedules correctly without explicit decomposition by a reviewer who knows to look for it.
## [](#what-to-flag-for-the-budget-pass)What to Flag for the Budget Pass
A protocol-only MCA that is done correctly produces a short, specific handoff document for the budget negotiation. It should identify every cell designated NB/INV that needs charge master pricing, every imaging visit that requires individual designation rather than a collapsed line, every device procedure awaiting IDE letter confirmation, and any visit where the SOC versus RES designation will shift depending on what the sponsor’s budget proposes to pay for. That list is the input the finance team needs to price the budget correctly the first time, rather than discovering misalignments during a monitoring visit or a billing audit two years into the trial.
The protocol-only MCA does not replace the budget reconciliation pass. It makes that pass faster, more accurate, and defensible from day one of site activation.
*Krishma Shah is Director of Clinical Relations at CliniBiz and co-inventor of BudgetSpark, a coverage-analysis engine that produces citation-level, designation-complete MCAs from the protocol and budget in days, not weeks. If your site or network wants to see one built on your own protocol, visit [budgetspark.com](https://budgetspark.com/) or write to .*
**Categories:** Clinical Trial Ops Brief
---
### [FDA's 2026 Single-Trial Default Standard Reframes the Dual-Pivotal Debate for Sponsors Still Designing Phase 3](https://www.clinicaltrialvanguard.com/opinion/fdas-2026-single-trial-default-standard-reframes-the-dual-pivotal-debate-for-sponsors-still-designing-phase-3/)
**Published:** September 22, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA's 2026 shift to a single pivotal trial as the default approval standard forces sponsors to rethink Phase 3 design before the next IND meeting.
**Content:**
On a date now circulating across regulatory affairs teams, FDA announced that one adequate and well-controlled clinical trial, combined with confirmatory evidence, will serve as the [new default standard for all drug approvals](https://www.troutman.com/insights/fda-announces-a-single-pivotal-trial-as-the-new-default-standard-for-all-drug-approvals-and-unveils-a-plausible-mechanism-framework-for-individualized-therapies/). The announcement lands while a substantial share of Phase 3 programs in active development were scoped, powered, and budgeted against a two-trial assumption that FDA has now formally displaced. Sponsors who set that assumption in their development plans last year did not make an error in judgment at the time. They may, however, be carrying excess trial infrastructure that the current regulatory posture no longer requires.
The Nature Reviews Drug Discovery analysis published under the title [“Two trials or not two trials? That should not be the question”](https://www.nature.com/articles/s41573-026-01537-w) frames the dual-trial debate as a category error. The article’s central argument is that the field has been optimizing for a structural requirement, two adequate and well-controlled studies, rather than for the evidentiary threshold those two trials were designed to satisfy: replicable demonstration of a treatment effect with controlled error rates. When the question shifts from trial count to evidentiary sufficiency, single-trial approval supported by mechanistic, biomarker, or real-world confirmatory data becomes a coherent regulatory position rather than an exception requiring extensive justification.
## [](#what-fdas-new-posture-actually-requires)What FDA’s New Posture Actually Requires
The practical content of FDA’s single-trial default warrants careful reading, because the announcement changes the burden structure without eliminating the evidentiary bar. A single pivotal trial satisfying the “adequate and well-controlled” standard under 21 CFR § 314.126 remains necessary. What changes is that sponsors no longer face an automatic expectation of a second independent replication study before NDA submission. The confirmatory evidence requirement fills that gap, and FDA has paired the default with a “plausible mechanism framework” intended to govern how mechanistic or biological data can serve confirmatory functions for individualized therapies specifically.
That framework distinction matters operationally. Sponsors in oncology, rare disease, and precision medicine, where patient populations constrain enrollment and biological rationale is frequently robust, gain the most direct benefit. Sponsors in broad-population indications, where biological plausibility is harder to specify and regulatory reviewers have historically demanded replication as protection against type I error inflation, face a more nuanced calculus. FDA had not, at the time of the announcement, published a guidance document specifying exactly which confirmatory evidence types satisfy the new default across indication classes. The absence of that specification is itself a design constraint sponsors need to account for in their next Type B or Type C meeting request.
The integrated safety evidence question compounds this. FDA’s March 2024 webinar on [integrated safety analyses in drug marketing applications](https://www.fda.gov/drugs/news-events-human-drugs/integrated-safety-analyses-drug-marketing-applications-avoiding-common-mistakes-03072024) emphasized that early planning for the integrated safety analysis is a prerequisite for adequate review, and identified Type C meetings as the appropriate venue for sponsors to align the integrated safety analysis plan with the agency before data lock. Under a single-trial architecture, the safety database accumulated from one pivotal study is thinner than the combined dataset from two trials. Sponsors transitioning to single-trial designs without revisiting their integrated safety analysis plan inherit a structural gap between the new trial count and the old safety evidence expectations.
## [](#the-ema-divergence-sponsors-cannot-ignore)The EMA Divergence Sponsors Cannot Ignore
FDA’s new default does not travel automatically across the Atlantic. The EMA’s CHMP has maintained that [randomised controlled evidence](https://www.ema.europa.eu/en/establishing-efficacy-based-single-arm-trials-submitted-pivotal-evidence-marketing-authorisation) remains the standard for demonstrating efficacy in marketing authorisation applications, and its guidance on single-arm trials submitted as pivotal evidence positions that pathway as an exception with specific evidentiary conditions, not a general alternative to controlled replication. Sponsors building a global registration strategy around FDA’s single-trial default must map whether their confirmatory evidence package, however persuasive to an FDA reviewer, meets CHMP’s distinct expectations for the same application cycle.
The EMA’s conditional marketing authorisation framework provides a parallel but structurally different pathway. Under the [conditional marketing authorisation](https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation) framework, EMA can approve on incomplete data subject to post-authorisation obligations, including specific studies the applicant commits to completing. The key distinction from FDA’s new default is timing and obligation structure: EMA’s conditional route defers confirmatory evidence to post-approval with binding commitment, whereas FDA’s framework appears to require confirmatory evidence as part of the NDA package, not as a post-market obligation. Sponsors targeting simultaneous FDA and EMA submissions under a single-trial design face a sequencing problem that the available published guidance from either agency does not clearly resolve.
A [2022 economic evaluation published in JAMA Network Open](https://pmc.ncbi.nlm.nih.gov/articles/PMC9277502/) provides relevant cost framing for this divergence. The analysis found that a [single platform trial evaluating 10 interventions cost substantially less and completed faster than a series of conventional two-group trials](https://pmc.ncbi.nlm.nih.gov/articles/PMC9277502/) covering equivalent ground. Translated to the dual-agency context: sponsors who design the single FDA pivotal trial with CHMP conditional authorisation requirements built in, specifying post-authorisation study commitments at the design stage rather than retrofitting them after NDA submission, preserve cost efficiency while maintaining a viable EMA pathway. Sponsors who treat FDA’s new default as license to reduce total trial investment without adjusting the EMA strategy are optimizing for one regulator at the cost of the other.
## [](#the-design-implications-for-programs-in-progress)The Design Implications for Programs in Progress
Sponsors with Phase 2 readouts expected in Q4 2026 or Q1 2027 face the most acute version of this decision. The protocol for the Phase 3 pivotal study is being finalized now, and the assumption embedded in that protocol, either one trial or two, will be extremely difficult to revise once FDA has reviewed and commented on the IND amendment containing it. The Nature Reviews Drug Discovery analysis explicitly identifies trial count as the wrong optimization target; the right optimization target is pre-specification of the evidentiary chain that will satisfy both the primary endpoint and the confirmatory evidence requirement FDA has attached to its new default.
For sponsors with programs already in Phase 3 under a two-trial design, the publication and FDA’s announcement together create a legitimate question for the next Type B meeting: whether the second trial, if it has not yet enrolled, could be restructured as a confirmatory study with a different design burden than a full independent replication. That restructuring requires FDA agreement and a protocol amendment, carries its own timeline risk, and should be modeled against the cost of completing the second trial as originally designed before any decision is made.
Site networks and CROs supporting multi-trial programs in rare disease and oncology face a distinct implication. If sponsors move toward single-trial Phase 3 designs at scale, total enrollment demand per program decreases, which affects site activation timelines, per-site patient flow projections, and the revenue models CROs use to staff therapeutic area units. The platform trial data from JAMA Network Open is instructive here: consolidated trial designs generated efficiency gains at the program level, but they concentrate enrollment risk in a single protocol. A single-trial strategy that encounters an operational disruption, a safety signal requiring protocol amendment, or an enrollment shortfall has no second trial to absorb the delay.
The next regulatory artifact to track is FDA’s anticipated guidance specifying which confirmatory evidence types satisfy the new default standard across indication classes. Until that document is published, sponsors should use Type C meetings to document FDA’s indication-specific expectations in writing, and they should treat any informal agreement reached in those meetings as binding only to the extent it is reflected in the written meeting minutes FDA issues following the meeting. The plausible mechanism framework announced alongside the single-trial default applies explicitly to individualized therapies; its scope outside that category remains an open question that the forthcoming guidance will need to resolve.
## [](#references)References
1. [Nature Reviews Drug Discovery, “Two trials or not two trials? That should not be the question”](https://www.nature.com/articles/s41573-026-01537-w)
2. [Troutman Pepper, “FDA Announces a Single Pivotal Trial as the New Default Standard for All Drug Approvals and Unveils a Plausible Mechanism Framework for Individualized Therapies”](https://www.troutman.com/insights/fda-announces-a-single-pivotal-trial-as-the-new-default-standard-for-all-drug-approvals-and-unveils-a-plausible-mechanism-framework-for-individualized-therapies/)
3. [EMA CHMP, “Establishing efficacy based on single-arm trials submitted as pivotal evidence for marketing authorisation”](https://www.ema.europa.eu/en/establishing-efficacy-based-single-arm-trials-submitted-pivotal-evidence-marketing-authorisation)
4. [FDA, “Integrated Safety Analyses in Drug Marketing Applications: Avoiding Common Mistakes” (March 2024)](https://www.fda.gov/drugs/news-events-human-drugs/integrated-safety-analyses-drug-marketing-applications-avoiding-common-mistakes-03072024)
5. [JAMA Network Open / PMC, Economic evaluation of platform trial versus conventional trials for 10 interventions](https://pmc.ncbi.nlm.nih.gov/articles/PMC9277502/)
6. [EMA, “Conditional marketing authorisation”](https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, DOGE FDA Budget Cuts, EMA, FDA Regulatory Strategy, Pivotal Trial Design
---
### [Risk-Based Source Data Verification Adoption Rises to 96% in Clinical Trials](https://www.clinicaltrialvanguard.com/news/risk-based-source-data-verification-adoption-rises-to-96-in-clinical-trials/)
**Published:** September 22, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Risk-based source data verification adoption reaches 96% in clinical trials, reshaping how monitors prioritize verification intensity across sites and data fiel
**Content:**
Ninety-six percent of clinical trials now include at least one risk-based monitoring component, up from 88% just a year earlier, according to the ACRO’s 2024 RBQM report. That shift makes the question of how source data verification gets configured, and where AI fits into it, more than an operational nicety. Get the SDV plan wrong and the consequences are regulatory, not just operational: a May 2024 FDA warning letter stemming from a BIMO inspection cited inadequate source documentation as an objectionable condition, a reminder that accurate transcription is still the floor regulators check first.
The practical problem with traditional SDV was volume. Applying uniform verification across every data field in a large trial burns monitor time on low-risk entries while higher-risk data, eligibility criteria, primary endpoints, critical safety fields, gets the same treatment as a routine demographics form. A configurable approach flips that: the monitoring plan defines verification intensity by site, participant group, visit, or individual field, and the intensity adjusts as the trial runs. A site with repeated eligibility errors draws more scrutiny; one with a clean track record holds at its planned level. The key discipline is that a risk signal should trigger a formal assessment before any SDV plan change, not an automatic escalation. Signal detected, assess significance, decide response, then adjust if needed.
Remote SDV fits inside that same logic rather than beside it. It is an execution method for verification the monitoring plan already requires, not a separate strategy. Where sites have electronic health records with appropriately restricted read-only access, verification that previously needed an on-site visit can happen remotely. A study published in *Therapeutic Innovation & Regulatory Science* found that [remote risk-based monitoring saved between 9 and 41 on-site monitoring visits per trial site](https://pubmed.ncbi.nlm.nih.gov/33258688/), corresponding to cost savings of $13,500 or more per site. Most studies will use both methods depending on what is being reviewed and what the monitoring need requires at that moment.
AI enters at the point where monitors process source documents against EDC entries, a step that is time-consuming but structured enough for automation to help. The strongest use case is extraction, comparison, and prioritization for human review, not autonomous verification decisions. A system that flags probable matches and surfaces discrepancies for monitor judgment compresses the manual effort without removing the human accountability that [regulators inspect for](https://www.clinicalpathwaysresearch.com/blog/2025/7/29/inadequate-source-documentation-results-in-fda-warning-letter). The SDR/SDV distinction matters here too: a correctly transcribed value can still represent a procedure that fell outside the protocol window, and no extraction model catches that without clinical context. Whether AI-assisted SDV systems in production actually reduce discrepancy rates, rather than just reducing monitor hours, is the number worth demanding from any vendor evaluation.
*Source link: *
**Categories:** News
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### [Merry Life Biomedical Starts Phase 2 Trial of TML-6 for Early Alzheimer's](https://www.clinicaltrialvanguard.com/news/merry-life-biomedical-starts-phase-2-trial-of-tml-6-for-early-alzheimers/)
**Published:** September 22, 2026
**Author:** Jon Napitupulu
**Excerpt:** Merry Life Biomedical starts Phase 2 trial of TML-6, an oral drug targeting autophagy-lysosomal pathways in early Alzheimer's disease across 20 global sites.
**Content:**
Approved amyloid-clearing therapies slow early Alzheimer’s progression but don’t stop it, and that gap is exactly where Merry Life Biomedical is placing its bet. The Taiwan-based company is advancing TML-6, an oral investigational drug, into a [global Phase 2 study](https://www.prnewswire.com/news-releases/merry-life-biomedical-advances-global-phase-2-trial-of-tml-6-exploring-cellular-protein-clearance-in-early-alzheimers-disease-302880875.html) enrolling approximately 210 participants across 20 sites in Taiwan, Sweden, and the United States. The approach is mechanistically distinct from the antibody therapies now in clinical use: rather than tagging amyloid for immune clearance, TML-6 targets the autophagy-lysosomal pathway, the intracellular system neurons use to degrade and recycle damaged proteins.
That distinction matters because the disease continues to progress even under treatment with agents like [lecanemab](https://www.eisai.com/news/2023/news202349.html), which received traditional FDA approval in July 2023, and [donanemab](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-adults-alzheimers-disease), approved in July 2024. Both reduce amyloid burden and slow clinical decline in early-stage patients, but neither restores the cellular machinery responsible for ongoing protein clearance. Preclinical work on TML-6 has examined its effects on autophagy-lysosomal function in neurons and microglia alongside amyloid pathology and neuroinflammation, though translating that biology into a clinical signal remains the study’s central question.
The trial’s design is randomized, double-blind, and placebo-controlled. Participants receive TML-6 at 100 mg or 200 mg, or placebo, once daily for 52 weeks. The primary endpoint is change from baseline on the Clinical Dementia Rating Sum of Boxes, which captures both cognition and daily function. Secondary endpoints include four blood biomarkers: p-Tau 217, amyloid-beta, GFAP, and NfL. Henrik Zetterberg will lead biomarker analyses, and Barbara B. Bendlin will handle amyloid PET and MRI work, giving the study a biomarker infrastructure capable of detecting mechanistic effects even if the clinical signal is modest at this sample size.
Taiwan contributes nine of the 20 sites, making it the largest national cohort in the program. Sweden’s seven sites are anchored by Anne Börjesson-Hanson of Karolinska Institute, an investigator with primary investigator roles in more than 40 global trials. On October 4, she visits Taipei for the Taiwan Dementia Society Annual Scientific Meeting to work directly with the nine Taiwanese principal investigators. The p-Tau 217 trajectory over 52 weeks will be the number to watch: if TML-6 moves that biomarker in a direction consistent with improved lysosomal clearance, it gives the company a credible mechanistic story to carry into Phase 3 discussions.
*Source link: *
**Categories:** News
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### [Ivonescimab beats durvalumab in biliary tract cancer Phase III trial](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-durvalumab-in-biliary-tract-cancer-phase-iii-trial/)
**Published:** September 22, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ivonescimab plus chemotherapy achieved a statistically significant overall survival benefit over durvalumab in biliary tract cancer Phase III trial HARMONi-GI1.
**Content:**
Beating durvalumab plus chemotherapy on overall survival in biliary tract cancer is a meaningful bar to clear: that regimen, which earned FDA accelerated approval in September 2022, pushed median OS to 13 months in the TOPAZ-1 trial and became the established first-line standard. Akeso’s [Phase III HARMONi-GI1 trial](https://www.prnewswire.com/news-releases/ivonescimab-first-line-biliary-tract-cancer-phase-iii-overall-survival-data-selected-as-esmo-late-breaking-abstract-nearly-30-akeso-studies-to-be-presented-at-esmo-2026-302885329.html) did exactly that, randomizing 682 patients in China and producing a statistically significant OS benefit for ivonescimab plus chemotherapy against [durvalumab plus chemotherapy](https://pmc.ncbi.nlm.nih.gov/articles/PMC11326973/) as the comparator arm.
Those results earned a Presidential Symposium slot at ESMO 2026, the October 23-27 congress in Madrid, where Jian Zhou will deliver them as a Late-Breaking Abstract on October 25. That is the highest-visibility platform ESMO offers, typically reserved for trials whose data are considered practice-changing or sufficiently disruptive to warrant real-time presentation before a packed room of oncologists. Full numbers stay under embargo until the session, so the actual OS difference is not yet public, but the trial design pitted [ivonescimab](https://pubmed.ncbi.nlm.nih.gov/39073550/), a PD-1/VEGF bispecific antibody that received its first marketing authorization in China in May 2024, directly against the approved standard rather than against chemotherapy alone.
The biliary dataset is the headline, but Akeso is bringing nearly 30 studies total to Madrid. Ivonescimab features in 12 presentations, including rapid oral readouts on first-line favorable-risk renal cell carcinoma (Phase Ib/II) and recurrent or metastatic thymic carcinoma from the iTHYM study. Cadonilimab, a PD-1/CTLA-4 bispecific, appears in 15 presentations spanning gastric cancer, esophageal squamous cell carcinoma, colorectal cancer, and several rarer tumor types across neoadjuvant and advanced-disease settings.
The immediate number to watch when Zhou takes the podium on October 25 is the median OS difference between arms. If the gap is clinically meaningful beyond the roughly 1.6-month improvement durvalumab showed over chemotherapy alone in TOPAZ-1, it strengthens Akeso’s case for regulatory filings outside China, where the FDA accepted a BLA for ivonescimab in a different indication in January 2026.
*Source link: *
**Categories:** News
---
### [EC Approves Obicetrapib as Oral CETP Inhibitor for Hypercholesterolemia](https://www.clinicaltrialvanguard.com/news/ec-approves-obicetrapib-as-oral-cetp-inhibitor-for-hypercholesterolemia/)
**Published:** September 22, 2026
**Author:** Jon Napitupulu
**Excerpt:** European Commission approves obicetrapib, a CETP inhibitor for hypercholesterolemia, offering oral dosing and 41% LDL-C reduction in Phase 3 trials.
**Content:**
After three failed CETP inhibitor programs over two decades, the European Commission has approved a fourth: obicetrapib, cleared last week as both a monotherapy (Ubeslo) and a fixed-dose combination with ezetimibe (Evlarco), with the combination delivering LDL-C reductions of roughly 41 percent at one year in the Phase 3 [BROOKLYN trial](https://synapse.patsnap.com/article/newamsterdam-pharma-reports-positive-phase-3-brooklyn-trial-results-for-obicetrapib-in-heterozygous-familial-hypercholesterolemia). That number matters because it arrives in oral form, which is the practical gap PCSK9 inhibitors have struggled to close with patients who resist injections.
The approval covers adults with primary hypercholesterolaemia, including heterozygous familial and non-familial forms, and mixed dyslipidaemia. The supporting package spans three Phase 3 trials: BROADWAY, BROOKLYN, and TANDEM. The class carries heavy baggage. Torcetrapib’s [ILLUMINATE trial was stopped in 2006](https://www.ovid.com/journals/nejm/abstract/10.1056/nejmoa0706628~effects-of-torcetrapib-in-patients-at-high-risk-for-coronary?redirectionsource=fulltextview) after 82 deaths in the treatment arm, and dalcetrapib and anacetrapib each failed on cardiovascular outcomes before reaching approval. Obicetrapib’s EC clearance means regulators were satisfied that its tolerability profile does not repeat those signals, though long-term cardiovascular outcomes data from a dedicated outcomes trial remain a question the approval does not answer.
The commercial picture sits with NewAmsterdam and Menarini Group, which hold European rights. Menarini’s distribution reach across European markets is the operational asset here: obicetrapib’s oral tablet format positions it where [injectable PCSK9 inhibitors](https://getfuzebio.com/approvals/praluent-bla125559) like alirocumab and evolocumab have faced adherence friction, and the fixed-dose Evlarco combination reduces pill burden for patients already on ezetimibe. Whether that convenience translates to formulary wins depends on pricing negotiations with European payers, which remain ongoing in most markets.
The trial that will define obicetrapib’s long-term standing is the cardiovascular outcomes study; until those data read out, prescribers treating high-risk patients will have to decide whether LDL-C reduction alone justifies the switch from therapies with established outcomes track records.
*Source link: *
**Categories:** News
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### [Roche's Enicepatide Meets Phase II Endpoints in Type 2 Diabetes Trial](https://www.clinicaltrialvanguard.com/news/roches-enicepatide-meets-phase-ii-endpoints-in-type-2-diabetes-trial/)
**Published:** September 22, 2026
**Author:** Jon Napitupulu
**Excerpt:** Roche's enicepatide met Phase II endpoints in type 2 diabetes trial, demonstrating dose-dependent reductions in HbA1c and body weight over 48 weeks.
**Content:**
Enicepatide cut HbA1c and body weight in a dose-dependent pattern over 48 weeks in adults with type 2 diabetes and overweight or obesity, Roche announced Monday from its [Phase II CT-388-104 trial](https://www.globenewswire.com/news-release/2026/09/22/3365947/0/en/ad-hoc-announcement-pursuant-to-art-53-lr-roche-announces-positive-phase-ii-results-for-dual-glp-1-gip-receptor-agonist-enicepatide-in-people-living-with-type-2-diabetes-and-overwe.html). Both primary endpoints were met, placing enicepatide, a once-weekly dual GLP-1/GIP receptor agonist, squarely in competition with tirzepatide, whose SURMOUNT-1 data showed mean weight reductions of [16% to 22.5%](https://www.prnewswire.com/news-releases/lillys-surmount-1-results-published-in-the-new-england-journal-of-medicine-show-tirzepatide-achieved-between-16-0-and-22-5-weight-loss-in-adults-with-obesity-or-overweight-301561327.html) in people without diabetes. The class benchmark, in other words, is already demanding.
The reductions Roche reports are described as clinically meaningful, though the company has not yet released the specific HbA1c and weight-loss figures from CT-388-104 in this announcement. That gap matters: the dual GLP-1/GIP mechanism is proven at the class level, so the Phase III case for enicepatide will rest on magnitude, tolerability, and whether the dose-response curve holds at the highest doses patients can actually sustain. Phase II dose-finding studies are exactly where dropout rates at upper doses tend to soften an otherwise clean efficacy story.
The competitive context is real. [Tirzepatide](https://www.drugs.com/history/retatrutide.html) is already FDA-approved for both type 2 diabetes (as Mounjaro) and chronic weight management (as Zepbound), and [semaglutide](https://formblends.com/articles/aeo-hub/ozempic-approval-date-fda-timeline-indication-history) holds approvals across injectable and oral forms for diabetes and obesity. Roche enters a class where two entrenched molecules have years of real-world prescribing data and established payer relationships. A new dual agonist needs a differentiated tolerability profile or a formulation advantage to capture meaningful share, not just comparable efficacy numbers.
For trial watchers, the immediate question is when Roche discloses the full CT-388-104 dataset, including the safety and discontinuation figures at each dose level. Those numbers will determine whether the Phase III design can run at the doses that produced the headline efficacy results, or whether the program has to compromise on dose to keep dropout rates manageable.
*Source link: *
**Categories:** News
---
### [What Radial's 27-Site Brain Medicine Network Actually Means for EEG Endpoint Standardization](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/what-radials-27-site-brain-medicine-network-actually-means-for-eeg-endpoint-standardization/)
**Published:** September 21, 2026
**Author:** Krishma Shah
**Excerpt:** Radial's FDA-cleared Evoke EEG integration across 27 brain medicine clinics tests whether distributed neuroimaging endpoints can survive real-world site…
**Content:**
Picture the site initiation visit packet for a TMS-based depression trial running across a dozen clinics in three states. The PI at each site has a different background in neuroimaging. The coordinators have different EDC habits. And the EEG capture protocol, however carefully written, still depends on a technician who may or may not have run an ERP test before last Tuesday’s training call. This is the operational problem that every sponsor trying to use objective brain-function data as a trial endpoint eventually hits: the technology can be FDA-cleared and the science can be solid, but endpoint consistency lives or dies at the hands of whoever is running the device that morning.
Radial Therapeutics is now betting that vertical integration solves this problem in a way that site-by-site vendor contracts never have. Following its [August 20, 2026 acquisition of TMS Health Partners](https://www.fiercehealthcare.com/providers/radial-acquires-tms-health-partners-mso-behind-network-brain-medicine-clinics), the management services organization behind Mindful Health Solutions, Radial manages 27 brain medicine clinics across seven states, making it one of the largest brain medicine clinical networks in the country. Now it has selected Firefly Neuroscience’s [Evoke platform](https://evokeneuroscience.com/about-us/), a Class II neurodiagnostic device that received FDA 510(k) clearance in 2017, to feed objective EEG and ERP data into Radial’s AI operating system across that entire network. For clinical operations teams evaluating whether to engage this network for a CNS trial, the integration raises a question that matters more than the press release: what does standardized EEG capture actually require to hold up across 27 sites, and does a single MSO arrangement deliver it?
## [](#the-endpoint-consistency-problem-nobody-budgets-for)The Endpoint Consistency Problem Nobody Budgets For
Any coordinator who has worked a neuroimaging study knows that EEG data quality is exquisitely sensitive to things that never make it into the protocol: electrode impedance at setup, ambient electrical interference, patient fatigue before the session, and technician confidence with the software. ERP testing adds another layer, because the stimulus-response paradigm requires consistent administration conditions, the same auditory or visual stimuli, the same inter-stimulus intervals, the same instruction script delivered to the patient the same way. Miss any of those and the waveform morphology shifts. P300 latency, one of the most commonly used ERP markers in depression and cognitive disorder research, can drift by 20 milliseconds or more from confounds that a site coordinator would never flag as a deviation.
A [published study of 52 MDD patients](https://pubmed.ncbi.nlm.nih.gov/32375213/) using ERP markers to predict antidepressant outcomes found that 61% achieved remission and 77% showed treatment responsiveness over eight weeks, results that are only meaningful if the underlying ERP data is captured consistently enough to be analyzed as a biomarker rather than noise. That level of endpoint integrity demands something most multi-site trials rely on inconsistently: a training and quality oversight mechanism that survives coordinator turnover, site-level distraction, and the ambient chaos of a clinic running five concurrent protocols.
This is where the MSO structure Radial has built changes the operational math, at least in theory. When a single entity manages the non-clinical operations of 27 clinics, staffing models, technology infrastructure, operational SOPs, it can enforce device training requirements and EEG capture protocols in a way that a sponsor CRA visiting four times a year cannot. A [network of 27 clinics under unified MSO management](https://www.fiercehealthcare.com/providers/radial-acquires-tms-health-partners-mso-behind-network-brain-medicine-clinics) means a single Evoke training curriculum, a single quality check against the same device firmware version, and a single escalation path when a site’s data looks anomalous. For sponsors, that is the operational pitch: the monitoring overhead per site drops when the site network is already operating to a common standard before the CRA ever books a flight.
But unified management and standardized execution are not the same thing, and the gap between them is where most multi-site trials actually fail.
## [](#what-the-fdas-dct-guidance-demands-from-distributed-biomarker-capture)What the FDA’s DCT Guidance Demands From Distributed Biomarker Capture
The FDA’s [September 2024 final guidance on decentralized clinical trials](https://www.federalregister.gov/documents/2024/09/18/2024-21078/conducting-clinical-trials-with-decentralized-elements-guidance-for-industry-investigators-and-other) does not give distributed EEG capture a free pass because the device is 510(k)-cleared. The guidance is explicit that sponsors using technology to collect trial data at locations other than the principal site must establish that the data collection process is equivalent across those locations, equivalent in training, in device qualification, and in documentation. For an EEG/ERP endpoint, that means source documentation for every session: the impedance log, the artifact rejection parameters, the technician identifier, the calibration record. It means a deviation process for sessions that fall outside acceptable signal quality thresholds. And it means a monitoring plan that can detect drift in data quality before it accumulates across enough patients to compromise the endpoint.
Sites I work with across distributed CNS programs routinely underestimate how much site-level documentation a neurophysiology endpoint generates compared to a standard PRO or clinical scale. A coordinator managing a MADRS assessment produces a completed form and a source note. A coordinator managing an Evoke session produces a session log, a signal quality record, and potentially a deviation report if the impedance values fell outside protocol spec, and those artifacts need to land in the TMF and the EDC in a way that a remote data review can actually interrogate. Across 27 sites, that documentation volume is not trivial, and a unified MSO helps only if it has built the SOP infrastructure to capture it consistently.
Radial’s stated model, backed by [$50 million in Series A and seed funding led by General Catalyst as of December 2025](https://www.meetradial.com/blog/radial-launches-with-50-million-to-expand-access-to-advanced-brain-medicine), is an AI-native operating system that aggregates clinical data across its network. The Evoke integration is designed to feed into that system rather than generate isolated site-level data files. If the data pipeline from device capture to the central platform is robust, a sponsor’s data management team could theoretically see a real-time signal quality dashboard across all 27 sites rather than waiting for monitoring visit findings. That is a genuine operational advantage, provided the integration has been validated to the standard the FDA’s DCT guidance requires for remote data collection tools.
## [](#the-sponsor-questions-that-need-answers-before-site-activation)The Sponsor Questions That Need Answers Before Site Activation
For clinical operations teams evaluating Radial’s network as a trial execution partner, the Evoke integration creates a vendor oversight question that sits upstream of the typical site qualification checklist. Firefly’s Evoke platform earned its 510(k) clearance as an aid to clinical diagnosis, as validated in a [pilot study evaluating EEG biomarkers in MDD patients treated with vortioxetine](https://www.researchgate.net/publication/377406418_Evaluating_an_EEG-based_tool_for_assessing_acute_clinical_and_cognitive_changes_in_adult_outpatients_with_MDD_treated_with_open-label_flexible-dose_vortioxetine_A_pilot_study). Using it as a trial endpoint rather than a clinical tool requires the sponsor to specify, in the protocol and the SAP, exactly which Evoke output variables constitute the endpoint, what ranges are acceptable, and how out-of-range sessions are handled. That analytical definition has to survive FDA review, and it has to be operationally enforceable at the site level.
The pre-startup work here is more demanding than sponsors typically scope. Before the SIV, the sponsor’s clinical operations team needs to confirm that every Radial clinic in the trial has completed Evoke device qualification under a documented procedure, that the training records are in the TMF, and that the data transfer pathway from the Evoke platform to the EDC has been tested and validated. A PSV checklist built for a standard Phase II depression trial does not cover those items, they require a device-specific readiness assessment that looks more like a lab qualification audit than a site initiation walk-through.
Sponsors who plan to run risk-based monitoring against this network should build their central monitoring triggers around EEG signal quality metrics, not just data entry patterns. If a site’s average impedance values or artifact rejection rates drift outside the protocol-specified range across consecutive sessions, that is a data integrity signal that warrants a triggered on-site visit, not a query. Building that trigger into the monitoring plan before first-patient-in is the difference between catching a site-level execution problem in month two and discovering it at database lock.
The operational truth here is that [Radial’s 27-clinic network](https://news.outsourceaccelerator.com/radial-tms-health-partners/) and the Evoke integration represent a genuinely new site activation model for CNS trials, one where the sponsor is not assembling a site network from scratch but inheriting an existing operational infrastructure with its own standards, technology stack, and management chain. That is faster to activate and cheaper to monitor than building from individual sites. It also means the sponsor’s oversight responsibility shifts: less time qualifying individual sites, more time qualifying the MSO’s systems. For CNS sponsors accustomed to one model, the transition requires updating the vendor oversight framework before the first contract amendment is signed.
## [](#references)References
1. [GlobeNewswire, “Firefly Neuroscience’s Evoke™ Platform Selected to Bring Objective Brain-Function Insights to Radial’s AI-Native Operating System for Brain Medicine”](https://www.globenewswire.com/news-release/2026/09/17/3363887/0/en/firefly-neuroscience-s-evoke-platform-selected-to-bring-objective-brain-function-insights-to-radial-s-ai-native-operating-system-for-brain-medicine.html)
2. [Evoke Neuroscience, FDA 510(k) clearance and device description for the Evoke EEG/ERP platform](https://evokeneuroscience.com/about-us/)
3. [Fierce Healthcare, “Radial acquires TMS Health Partners, MSO behind network of brain medicine clinics” (August 20, 2026)](https://www.fiercehealthcare.com/providers/radial-acquires-tms-health-partners-mso-behind-network-brain-medicine-clinics)
4. [Radial Therapeutics — “$50 Million Series A and Seed Funding, December 2025”](https://www.meetradial.com/blog/radial-launches-with-50-million-to-expand-access-to-advanced-brain-medicine)
5. [PubMed, ERP markers predicting antidepressant outcomes in 52 MDD patients (61% remission, 77% treatment responsiveness)](https://pubmed.ncbi.nlm.nih.gov/32375213/)
6. [ResearchGate, “Evaluating an EEG-based tool for assessing acute clinical and cognitive changes in adult outpatients with MDD treated with open-label, flexible-dose vortioxetine: A pilot study”](https://www.researchgate.net/publication/377406418_Evaluating_an_EEG-based_tool_for_assessing_acute_clinical_and_cognitive_changes_in_adult_outpatients_with_MDD_treated_with_open-label_flexible-dose_vortioxetine_A_pilot_study)
7. [Federal Register, FDA Final Guidance: “Conducting Clinical Trials With Decentralized Elements” (September 18, 2024)](https://www.federalregister.gov/documents/2024/09/18/2024-21078/conducting-clinical-trials-with-decentralized-elements-guidance-for-industry-investigators-and-other)
**Categories:** Clinical Trial Ops Brief
**Tags:** brain medicine, decentralized trials, eCOA standardization, EEG biomarkers, site network operations
---
### [The Liquid Biopsy That Found Pancreatic Cancer Early Has a Bigger Regulatory Problem Than Its Science](https://www.clinicaltrialvanguard.com/opinion/the-liquid-biopsy-that-found-pancreatic-cancer-early-has-a-bigger-regulatory-problem-than-its-science/)
**Published:** September 21, 2026
**Author:** Moe Alsumidaie
**Excerpt:** PANXEON detects stage 1-2 pancreatic cancer with 87% sensitivity, but FDA's companion diagnostic pathway may be the bigger obstacle to clinical adoption.
**Content:**
Ninety percent of pancreatic ductal adenocarcinoma patients receive their diagnosis after the cancer has already spread. That number has barely moved in decades, and it explains almost everything about why the five-year survival rate for PDAC sits at a grim 13 percent, per the [American Cancer Society’s 2024 Cancer Facts & Figures Report](https://pancreatic.org/5-year-survival-rate-increases-again-landing-at-13/). Into that context, City of Hope’s investigational liquid biopsy platform [PANXEON](https://www.nature.com/articles/s41591-026-04625-x) just published performance data that genuinely warrants attention: [87% sensitivity for stage 1 and stage 2 PDAC](https://clpmag.com/disease-states/cancer/pancreatic/panxeon-liquid-biopsy-detects-early-stage-pancreatic-cancer/), with a 3% false-positive rate in low-risk groups.
Those numbers would be remarkable for any cancer type. For pancreatic cancer, where catching the disease before it metastasizes is the only scenario where surgical resection becomes viable, they represent a potential inflection point in how the field thinks about population-level screening.
But the science solving the detection problem may be the easier half of what comes next.
## [](#what-the-performance-data-actually-says)What the Performance Data Actually Says
Open the PANXEON dataset and read it carefully, because the headline sensitivity figure obscures a critical operational variable. The 87% sensitivity applies to stages 1 and 2 combined. The false-positive rate diverges sharply by risk population: 3% in low-risk groups, 16% in high-risk groups. That 16% figure is not a trivial footnote. In a screening program targeting high-risk individuals, first-degree relatives of PDAC patients, carriers of relevant germline variants, individuals with new-onset diabetes, a 16% false-positive rate translates directly into diagnostic workups, patient anxiety, endoscopic ultrasounds, and downstream costs that the healthcare system will scrutinize before it reimburses anything at scale.
To put a number on what “downstream costs” [means: the mean total direct medical cost for pancreatic cancer treatment per Medicare patient](https://pmc.ncbi.nlm.nih.gov/articles/PMC5018231/) runs to [$65,500](https://pmc.ncbi.nlm.nih.gov/articles/PMC5018231/), with costs for resectable locoregional disease running higher still due to surgical intervention. A false-positive cascade in a high-risk cohort does not approach those figures, but it adds enough noise to a payer cost-effectiveness model to complicate coverage decisions substantially. Payers will run that arithmetic before any national coverage determination.
The platform also detected high-grade dysplasia, a precancerous state, which is clinically meaningful but introduces a separate regulatory category question: is PANXEON a cancer detection tool or a dysplasia surveillance tool? The FDA will want to know, and the answer changes the intended use statement, which changes the approval pathway, which changes the trial design requirements for every validation study that follows.
## [](#the-regulatory-architecture-problem)The Regulatory Architecture Problem
Here is the counterintuitive reality that the liquid biopsy field keeps learning the hard way: strong analytical performance does not accelerate regulatory approval; it frequently complicates it. The FDA’s companion diagnostic framework, formalized in the agency’s June 2023 [guidance on oncology drug products used with certain in vitro diagnostics](https://www.fda.gov/medical-devices/in-vitro-diagnostics/companion-diagnostics), describes a voluntary pilot program designed to facilitate co-development of therapeutics and their companion tests. PANXEON is not a companion diagnostic in the traditional sense, it is a screening tool without a paired therapeutic, which means the co-development pathway does not cleanly apply.
That distinction matters enormously for trial design. A companion diagnostic developed alongside a targeted therapy can leverage the drug’s pivotal trial as part of the device’s evidentiary package. A standalone screening biomarker has no such anchor. It must demonstrate clinical validity and clinical utility independently, which requires a prospective study design with endpoints the FDA will negotiate through De Novo or PMA review, not an abbreviated pathway.
The CellSearch CTC enumeration platform, which received initial FDA clearance for monitoring advanced metastatic breast cancer and later for colorectal and prostate cancers,, spent years navigating exactly this gap between analytical performance and clinical utility demonstration. CellSearch could enumerate circulating tumor cells accurately. What it took considerably longer to establish was whether that enumeration actually changed clinical decision-making in a way that improved patient outcomes. Payers and the FDA asked that question repeatedly, and the answers drove post-market requirements that extended the platform’s commercial uncertainty well past its initial clearance.
PANXEON now faces a structurally similar question, posed with considerably higher stakes. Screening an asymptomatic population for a disease with a 13% five-year survival rate carries a different risk-benefit calculus than monitoring a known metastatic patient. The FDA will demand a prospective dataset demonstrating that acting on a positive PANXEON result, through increased surveillance, earlier surgical evaluation, or enrollment into prevention trials, produces a measurable improvement in clinical outcomes. Analytical sensitivity alone does not satisfy that bar.
## [](#what-a-credible-validation-path-looks-like)What a Credible Validation Path Looks Like
Sponsors building the regulatory strategy for a platform like PANXEON face three design decisions that will determine whether their clinical utility data is accepted on first submission or triggers a complete response letter.
First, the risk stratification architecture must be locked before pivotal enrollment begins. The 3% versus 16% false-positive rate divergence across risk groups is exactly the kind of finding a reviewer will use to require subgroup-specific labeling claims. If the sponsor wants a single intended use statement covering both low-risk and high-risk populations, the trial must be powered to support that claim in both strata simultaneously, not sequentially in post-hoc analyses.
Second, multi-country regulatory pathways require coordinated dossier design from day one, not as an afterthought following US approval. The EMA’s regulation of in vitro diagnostics under IVDR, which began phased application from May 2022,, applies a performance class framework that diverges from the FDA’s intended use and predicate-based structure. A PANXEON submission built exclusively around FDA evidentiary requirements will require significant restructuring for the EMA, a problem that costs sponsors 12 to 18 months of additional development time when not anticipated in protocol design.
Third, the clinical utility endpoint cannot be time-to-surgery alone. The FDA has signaled in recent advisory committee discussions that cancer screening tools need to demonstrate impact on stage migration at the population level, meaning the pivotal study design should include a comparator arm reflecting current standard-of-care diagnosis timing, with pre-specified analysis of what percentage of PANXEON-detected cases presented at stage 1 or 2 versus what historical registry data would predict for the same population. That is a complex trial design problem, and it requires an adaptive framework with pre-specified interim analyses to allow early stopping for efficacy or futility without inflating Type I error.
The [PANXEON performance data](https://www.oncology-central.com/liquid-biopsy-could-detect-pancreatic-ductal-adenocarcinoma-at-its-earliest-stages/) published this month represents the kind of signal the field has been waiting for. The 87% sensitivity in early-stage PDAC, if it holds in a prospective registry trial of sufficient scale, could genuinely shift the staging distribution at diagnosis. But the path from a promising biomarker study to an FDA-cleared, CMS-reimbursed screening test for asymptomatic high-risk individuals runs through regulatory terrain that has stopped technically superior platforms before. The sponsors who get there first will be the ones who treat the regulatory strategy as a trial design input, not a submission task, and who lock their intended use statement before they enroll their first subject.
## [](#references)References
1. [Nature Medicine, “Liquid biopsy for early detection of pancreatic ductal adenocarcinoma”](https://www.nature.com/articles/s41591-026-04625-x)
2. [Hirshberg Foundation for Pancreatic Cancer Research, “5-Year Survival Rate Increases Again, Landing at 13%” (January 17, 2024)](https://pancreatic.org/5-year-survival-rate-increases-again-landing-at-13/)
3. [PMC / NCBI, “Mean total direct medical cost for pancreatic cancer treatment per Medicare patient: $65,500”](https://pmc.ncbi.nlm.nih.gov/articles/PMC5018231/)
4. [Oncology Central, “Liquid biopsy could detect pancreatic ductal adenocarcinoma at its earliest stages” (PANXEON 87% sensitivity, false-positive rates)](https://www.oncology-central.com/liquid-biopsy-could-detect-pancreatic-ductal-adenocarcinoma-at-its-earliest-stages/)
5. [FDA, “Companion Diagnostics” guidance page, including June 21, 2023 pilot program guidance for oncology drug products used with certain IVD tests](https://www.fda.gov/medical-devices/in-vitro-diagnostics/companion-diagnostics)
6. [CLP Magazine, “Panxeon Liquid Biopsy Detects Early-Stage Pancreatic Cancer”](https://clpmag.com/disease-states/cancer/pancreatic/panxeon-liquid-biopsy-detects-early-stage-pancreatic-cancer/)
7. [Medpace, “The Utility of Liquid Biopsy in Oncology Clinical Trials” (including CellSearch CTC platform FDA approval history)](https://www.medpace.com/wp-content/uploads/2023/03/Article-The-Utility-of-Liquid-Biopsy-in-Oncology-Clinical-Trials.pdf)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, Companion Diagnostics, FDA Regulatory Pathway, liquid biopsy, Pancreatic Cancer
---
### [FDA Part 11 Compliance: Audit Trails and Electronic Signatures](https://www.clinicaltrialvanguard.com/news/fda-part-11-compliance-audit-trails-and-electronic-signatures/)
**Published:** September 21, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA Part 11 Compliance requires validated systems, audit trails, and electronic signatures linked to individual users. Learn what triggers 483 observations.
**Content:**
When an FDA investigator pulls up an audit trail during an inspection, the question is not whether a company has a Part 11 policy. It is whether the systems running underneath that policy can actually prove what happened, who did it, and when. That gap between documented intent and operational reality is where most 483 observations originate, and it is narrower to close than most quality teams assume before they face it.
[21 CFR Part 11](https://www.dotcompliance.com/blog/21-cfr-part-11/21-cfr-part-11-compliance-explained-and-how-eqms-helps/), issued in 1997, answers a foundational question: can an electronic record carry the same legal weight as a paper one with a handwritten signature? The regulation says yes, under specific conditions. Those conditions cover validated systems, protected records with defined retention periods, access limited to authorized individuals, and secure computer-generated audit trails that cannot be altered or stripped without leaving a trace. The [FDA’s 2018 guidance on scope and application](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/part-11-electronic-records-electronic-signatures-scope-and-application) reinforced a narrow interpretation: Part 11 does not create new documentation requirements on its own. It applies wherever a predicate rule, such as GMP or GCP, already demands a record that a company keeps electronically.
Electronic signature requirements add a second layer that catches organizations off guard more often than the records side does. Each signature must be unique to one person, permanently linked to its record, and carry a visible meaning: whether the signer approved, reviewed, or authored the document. A signature confirming only that “this action happened” fails the standard. Shared login credentials fail it immediately, because the agency requires that any signed action trace back to one verified individual. The most frequently cited violations in FDA Form 483 observations follow predictable patterns: disabled audit trails, shared accounts, systems placed into GMP use without documented validation, and password policies that were never enforced or updated after personnel changes.
An electronic quality management system built around Part 11 requirements handles most of this at the workflow level, enforcing correct signing sequences, generating time-stamped audit trails automatically, and flagging access anomalies rather than leaving those checks to periodic manual review. The practical difference is that compliance becomes a function of daily operation rather than a pre-inspection sprint. For organizations still relying on spreadsheets or loosely configured digital tools for GMP-required records, the validation documentation gap is likely the first place an investigator will look.
*Source link: *
**Categories:** News
---
### [Phase III Clinical Trials Now Generate 5.9M Data Points Annually, Growing 11% Yearly](https://www.clinicaltrialvanguard.com/news/phase-iii-clinical-trials-now-generate-5-9m-data-points-annually-growing-11-yearly/)
**Published:** September 21, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Phase III Clinical Trials now generate 5.9M data points annually with 11% yearly growth, raising questions about managing protocol complexity and data quality.
**Content:**
Phase III trials now collect an average of 5.9 million data points per protocol, and that figure has grown roughly 11% every year since 2020. That single statistic captures something the industry has been slow to name plainly: complexity is no longer a feature of ambitious trials, it is the baseline condition of all of them.
The growth didn’t start in 2020. [Tufts CSDD data show](https://www.fiercebiotech.com/cro/one-third-data-collected-clinical-trials-may-be-unnecessary-study-finds) that Phase III data point volumes jumped from roughly 929,000 in 2012 to 3.56 million by 2020, before the current acceleration. Layered on top, [Phase III protocols now carry 37% more endpoints and 42% more procedures](https://cromospharma.com/world-clinical-trials-day-clinical-research-is-not-where-it-used-to-be/) than they did a decade ago. Narrower eligibility criteria and more diverse data sources compound both. Sites that could once manage monitoring with periodic on-site visits now generate data streams that traditional oversight was never designed to handle.
The operational consequence is straightforward: more data does not automatically mean better data. [A 2024 survey found that 39% of sponsors named protocol complexity as a primary driver of rising costs](https://www.ppd.com/blog/clinical-trial-cost-complexity/), which nearly half of respondents identified as their top concern. Volume without directed oversight produces noise. A monitoring approach calibrated to flag everything flags nothing useful, which is exactly the gap [CluePoints frames as the argument for statistical, risk-based quality management](https://cluepoints.com/trial-complexity-is-the-new-normal/). FDA’s April 2023 guidance on risk-based monitoring reinforced that the agency expects sponsors to use centralized, data-driven oversight rather than simply adding site visits to compensate for protocol weight.
The number to watch is the annual growth rate, not the current snapshot. At 11% per year, data volumes double roughly every seven years. A site management model adequate for 5.9 million points per trial becomes inadequate around 2033 without structural changes to how data are prioritized and reviewed. Sponsors who treat complexity as a temporary condition to be tolerated will find the math working against them well before that.
*Source link: *
**Categories:** News
---
### [Wearable neuromodulation reduces AF recurrence after ablation in pilot trial](https://www.clinicaltrialvanguard.com/news/wearable-neuromodulation-reduces-af-recurrence-after-ablation-in-pilot-trial/)
**Published:** September 21, 2026
**Author:** Jon Napitupulu
**Excerpt:** Wearable neuromodulation reduces AF recurrence after ablation in pilot trial, with 90% freedom from arrhythmias at six months versus 80.8% with sham.
**Content:**
Post-ablation atrial fibrillation recurrence is not a rare edge case: even with pulsed field ablation, the [2023 ADVENT trial](https://www.acc.org/latest-in-cardiology/clinical-trials/2023/08/25/03/54/advent) showed one-year treatment success at 73.3%, meaning roughly one in four patients still fails. StimCardio Medical’s NeuroPulse pilot, presented as late-breaking clinical science at HRX Live 2026 and simultaneously published in *Heart Rhythm*, now puts a number on what wrist-worn neuromodulation might add on top of ablation: 90.0% freedom from atrial arrhythmias at six months in the active arm, versus 80.8% with sham.
The trial enrolled 77 adults with paroxysmal or persistent AF, randomized 1:1 to active median nerve stimulation (MNS) or sham, with therapy starting within 48 hours of catheter ablation and continuing for six months. The modified intention-to-treat analysis covered 59 patients after a 60-day blanking period. The freedom-from-arrhythmia gap between arms did not reach statistical significance, but the event count did: 12 recurrent atrial arrhythmia events with MNS versus 28 with sham in the post-blanking sensitivity analysis (p = 0.025). No MNS-related serious adverse events occurred. The logic here is biological, not technical: rather than refining ablation lesion sets, MNS targets peripheral neural pathways involved in autonomic cardiac regulation, an approach that could run in parallel with whatever ablation method an electrophysiologist already uses.
Lead investigator Vivek Y. Reddy of Mount Sinai framed the finding as a signal worth confirming, not a conclusion, and StimCardio says a pivotal trial is next. That sequence matters for interpreting the data. A 77-patient pilot with a 59-patient analysis is powered to detect a signal, not to validate a therapy. The event-count difference is real and statistically supported; the freedom-from-arrhythmia rate difference is directionally consistent but not yet confirmatory. Investors and electrophysiologists should treat these as two separate reads on the same data, because they carry different evidentiary weight.
The device under development, Rhyvive, is still investigational and restricted to investigational use under U.S. law. The practical question the pivotal trial will need to answer is whether the event-count reduction holds at scale across multiple centers, with a population large enough to push the freedom-from-arrhythmia comparison past the significance threshold. That result will determine whether wearable neuromodulation earns a place in post-ablation care protocols or remains a promising pilot-study finding.
*Source link: *
**Categories:** News
---
### [Accro Bioscience Doses First Patient in AC-101 Phase IIb Trial for Ulcerative Colitis](https://www.clinicaltrialvanguard.com/news/accro-bioscience-doses-first-patient-in-ac-101-phase-iib-trial-for-ulcerative-colitis/)
**Published:** September 21, 2026
**Author:** Jon Napitupulu
**Excerpt:** Accro Bioscience doses first patient in AC-101 Phase IIb trial for ulcerative colitis, targeting the NOD/RIPK2 pathway with a 12-week clinical remission endpoin
**Content:**
Accro Bioscience has dosed its first patient in a Phase IIb trial of AC-101, a selective RIPK2 inhibitor, for moderate-to-severe ulcerative colitis, setting up a 12-week clinical remission readout across roughly 50 sites. The mechanism is the bet worth watching: AC-101 targets the [NOD/RIPK2 signaling pathway](https://www.prnewswire.com/news-releases/accro-bioscience-announces-first-patient-dosed-in-phase-iib-study-of-ac-101-in-moderate-to-severe-ulcerative-colitis-302883958.html), which sits upstream of the inflammatory cascade rather than blocking a single cytokine or integrin. That separates it from the biologics that already crowd this space, including agents like [vedolizumab](https://www.takeda.com/en-us/newsroom/news-releases/2014/fda-approves-takedas-entyvio-vedolizumab-for-the-treatment-of-adults-with-moderately-to-severely-active-ulcerative-colitis-or-crohns-disease/), infliximab, and adalimumab, though whether a different pathway translates to better outcomes for patients who fail those agents is exactly what the IIb needs to answer.
The trial uses a three-arm design: two AC-101 dose cohorts against placebo, with clinical remission at Week 12 as the primary endpoint. Exploratory endpoints extend further, tracking histologic remission and whether remission holds during long-term treatment. That second layer matters because ulcerative colitis patients who achieve endoscopic remission but not mucosal healing tend to relapse faster. Including histology as an exploratory endpoint suggests Accro is already building toward a durability argument, not just an induction signal.
The Phase IIb does not arrive blind. Accro completed a Phase Ib proof-of-concept study in Chinese patients with moderate-to-severe UC, and those [efficacy and safety results will be presented at UEG Week 2026](https://www.accropeutics.com/news/116.html) in Barcelona this October. Timing matters: the IIb’s enrollment is now open while the field gets its first look at AC-101’s Phase Ib data. A clean signal there will shape how investigators and potential partners read the larger trial. AC-101 also completed Phase I studies in healthy participants in both Australia and China with a favorable safety and pharmacokinetic profile, so the dose-selection rationale for the two IIb cohorts rests on a reasonably characterized base.
The [ACG’s updated UC guidelines](https://gi.org/journals-publications/ebgi/alkazzi_aug2025/) still anchor induction on corticosteroids and advanced therapies for moderate-to-severe disease, a standard that leaves real gaps for patients who cycle through biologics without durable remission. Whether AC-101 fills any of that gap comes down to what the Week 12 remission rates show across both dose arms versus placebo.
*Source link: *
**Categories:** News
---
### [Antengene Starts Phase III CLINCH-3 Trial of ATG-022 in Advanced Gastric Cancer](https://www.clinicaltrialvanguard.com/news/antengene-starts-phase-iii-clinch-3-trial-of-atg-022-in-advanced-gastric-cancer/)
**Published:** September 21, 2026
**Author:** Jon Napitupulu
**Excerpt:** Antengene launches Phase III CLINCH-3 Trial of ATG-022 in advanced gastric cancer, building on 46.7% response rates from Phase I/II data.
**Content:**
A 46.7% objective response rate in patients with moderate-to-high CLDN18.2 expression, with median overall survival not yet reached after 14 months of follow-up, is the Phase I/II data now pushing ATG-022 into a pivotal Phase III trial. Antengene dosed the first patient in China in the [CLINCH-3 study](https://www.prnewswire.com/news-releases/antengene-announces-first-patient-dosed-in-pivotal-phase-iii-clinch-3-study-of-atg-022-302883951.html) of ATG-022, its CLDN18.2 antibody-drug conjugate, targeting advanced gastric or gastroesophageal junction adenocarcinoma in patients who have progressed on at least two prior lines of therapy.
The Phase III design is randomized, open-label, and controlled, comparing ATG-022 monotherapy at the 1.8 mg/kg recommended Phase 2 dose against investigator’s choice. China’s NMPA Center for Drug Evaluation previously granted ATG-022 [Breakthrough Therapy Designation](https://www.antengene.com/newsinfo/441) for this setting, which Antengene credits for accelerating the regulatory path to this pivotal study. The trial starts in China and is planned to expand into a multi-regional format, with the accumulated data intended to support a marketing approval application for monotherapy.
The Phase I/II numbers that support this move are specific. Among the 30 patients at the 1.8 mg/kg dose with moderate-to-high CLDN18.2 expression, the confirmed ORR was 40%, the disease control rate reached 86.7%, and Grade 3 or higher treatment-related adverse events held relatively flat at 21% despite more than six additional months of follow-up since the December 2025 data cut. Only 9.7% of patients required dose reduction due to toxicity. Even in the harder-to-treat low and ultra-low CLDN18.2 expression group, roughly one in four patients (28.6%) responded. The competitive context matters here: [zolbetuximab (Vyloy)](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zolbetuximab-clzb-chemotherapy-gastric-or-gastroesophageal-junction-adenocarcinoma), approved by the FDA in late 2024, addresses first-line CLDN18.2-positive gastric cancer in combination with chemotherapy, leaving later lines as an area where options remain limited.
Antengene is running CLINCH-3 alongside two parallel programs: CLINCH-2 tests ATG-022 in the first-line setting combined with chemotherapy and anti-PD-1 therapy, and the CLINCH basket trial is generating early efficacy data in non-gastric CLDN18.2-positive solid tumors. CLINCH-3 enrollment pace, particularly the speed of expansion to sites outside China, will determine how quickly Antengene can reach the readout that would support an NMPA filing.
*Source link: *
**Categories:** News
---
### [Lavengratinib Shows 100% Response Rate in Lowest Dose Achondroplasia Cohort](https://www.clinicaltrialvanguard.com/news/lavengratinib-shows-100-response-rate-in-lowest-dose-achondroplasia-cohort/)
**Published:** September 21, 2026
**Author:** Jon Napitupulu
**Excerpt:** Lavengratinib achondroplasia trial shows 100% responder rate in lowest dose cohort with 2.4 cm/year growth improvement and clean safety profile.
**Content:**
All seven children in the lowest dose cohort of Abbisko’s Phase 2 lavengratinib study grew an average of 2.4 centimeters per year faster than their baseline rate after 27 weeks, and every single participant met the trial’s responder threshold, which required at least a 25% improvement in annualized height velocity. That 100% responder rate, from a cohort where children received just 0.064 mg/kg once daily, is the number that makes this readout worth watching: it came from the floor of the dose range, with higher cohorts still ongoing.
The comparison point for any achondroplasia growth therapy is [vosoritide (Voxzogo)](https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-improve-growth-children-most-common-form-dwarfism), which in its pivotal Phase 3 study produced roughly 1.57 centimeters of additional annual height gain over placebo. Lavengratinib’s 2.4 cm/year mean improvement from baseline in an open-label, single-arm design cannot be read as a head-to-head figure, but the magnitude is large enough to keep regulators and competitors paying attention as the dose escalation continues.
The safety profile so far is the other consequential finding. [Earlier oncology data](https://www.onclive.com/view/absk061-demonstrates-safety-preliminary-efficacy-in-fgfr2-3-advanced-solid-tumors) confirmed lavengratinib’s tolerability in adults, but FGFR pathway inhibitors carry well-documented risks tied to FGFR1 activity, including hyperphosphatemia and corneal toxicity. In the pediatric ACH cohort, none of those signals appeared across any of the first three dose levels. No serious adverse events and no treatment discontinuations were reported. That clean run through the lowest cohort, without FGFR1-associated toxicity, is the mechanistic argument for why a selective FGFR2/3 inhibitor could hold an advantage in a condition where children will need long-term dosing. Abbisko also built a mini-tablet formulation with tablets under 3 mm in diameter, about a third the size of a conventional tablet, specifically to make daily oral dosing workable in young children.
[Lavengratinib holds both Rare Pediatric Disease and Orphan Drug Designations](https://www.pharmabiz.com/NewsDetails.aspx?aid=185124&sid=2) from the FDA for achondroplasia, which matters for the regulatory path ahead. The six-month efficacy and safety dataset, expected before year-end 2026, will be the first look at whether the signal holds as the dose escalation reaches its higher cohorts.
*Source link: *
**Categories:** News
---
### [AbbVie’s Bet on Biology: Why One Modality Is Never Enough in Lung Cancer](https://www.clinicaltrialvanguard.com/executiveinterviews/abbvies-bet-on-biology-why-one-modality-is-never-enough-in-lung-cancer/)
**Published:** September 20, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Daejin Abidoye, Vice President, Therapeutic Area Head, Oncology, Solid Tumor and Hematology at AbbVie, discusses new data from ABBV-1480 (RC148) and ABBV-706 being presented at IASLC 2026 World Conference on Lung Cancer (WCLC), how AbbVie is advancing their programs and where biomarkers and AI could shape the future of oncology development.
**Content:**
 Daejin Abidoye, Vice President, Therapeutic Area Head, Oncology, Solid Tumor and Hematology at AbbVie *Lung cancer remains one of the hardest cancers to treat, despite major advances in targeted therapy and immunotherapy. At WCLC 2026, AbbVie is presenting new clinical and translational research across its lung cancer pipeline, including first-line Phase 1b data for the investigational PD-1/VEGF bispecific ABBV-1480 and new research around the SEZ6-targeted antibody-drug conjugate (ADC) ABBV-706.*
---
### [](#why-pursue-a-pipeline-that-pairs-bispecific-antibodies-with-adcs-rather-than-committing-fully-to-one-modality-in-lung-cancer)Why pursue a pipeline that pairs bispecific antibodies with ADCs rather than committing fully to one modality in lung cancer?
**Daejin Abidoye:** What we’ve seen over the last decade, as our understanding of the biology of non-small cell lung cancer has evolved, is that this is a very heterogeneous disease. You have different oncogenic drivers, in addition to multiple mechanisms of resistance.
So, for us, the question isn’t really which modality is better. It’s which biological problem each modality is best placed to address.
ADCs give us a way to deliver targeted tumor-cell killing, while immunotherapies allow us to act on the immune mechanisms and tumor microenvironment around the cancer. Multimodality therapy has emerged as an important approach not only for achieving a response, but for trying to maintain durable remissions in patients with locally advanced or metastatic disease.
We already see the foundation for that with chemotherapy and PD-1 checkpoint inhibitors in non-small cell lung cancer. Where we think we can go further is by using ADCs to bring more targeted cytotoxic activity and then layering that with next-generation immunotherapies. We think those complementary approaches hold a lot of promise for patients.
---
### [](#how-did-the-90-objective-response-rate-orr-you-observed-in-squamous-non-small-cell-lung-cancer-nsclc-shape-your-thinking-on-the-phase-3-design-for-abbv-1480)How did the 90% objective response rate (ORR) you observed in squamous non-small cell lung cancer (NSCLC) shape your thinking on the Phase 3 design for ABBV-1480?
**Daejin Abidoye:** The 90% response rate was very encouraging, but we didn’t look at that number in isolation. What gave us confidence to move into Phase 3 was the combination of the activity we were seeing across both squamous and non-squamous disease, the durability we were beginning to observe, and the dose-optimization work.
At the recommended Phase 3 dose of 10 mg/kg, we saw an ORR of 90.0% in squamous NSCLC and 75.9% in non-squamous NSCLC. Progression-free survival was 11.8 months in squamous and 11.1 months in non-squamous NSCLC, and median overall survival had not been reached.
We also evaluated both 10 mg/kg and 20 mg/kg. At the lower dose of 10 mg/kg, we were seeing strong responses alongside a manageable safety profile, and that allowed us to feel very confident selecting it as the recommended dose for Phase 3.
What excites us about ABBV-1480 itself also comes down to some of its structural features. It’s built on what we believe is a potent PD-1 backbone. It has a silent Fc effector function, which we believe may help mitigate some of the immune-related effects we typically see with checkpoint inhibitors. And it has a unique VEGF epitope that we believe provides strong signal-blocking properties with regard to the anti-angiogenic interaction.
So, it wasn’t simply, “we saw a 90% response rate, let’s go to Phase 3.” It was the totality of what we were seeing around activity, durability, dose, and the profile of the molecule that gave us that confidence.
---
### [](#how-does-the-breadth-of-sez6-expression-in-sclc-inform-where-abbv-706-fits-relative-to-other-adc-targets-being-pursued-in-that-disease)How does the breadth of SEZ6 expression in SCLC inform where ABBV-706 fits relative to other ADC targets being pursued in that disease?
**Daejin Abidoye:** ABBV-706 is our investigational SEZ6-directed ADC with a Top1 inhibitor payload. What excites us about it are two things: the activity we’ve seen with our proprietary Top1 payload, and what we’re learning about SEZ6 itself as a potential therapeutic target in small cell lung cancer (SCLC).
At WCLC, real-world research showed SEZ6 expression in 91% of patients with SCLC overall, and in more than 96% of patients with brain or liver metastases. What’s interesting to us about that is the breadth. It suggests SEZ6 may be relevant across a broad SCLC population rather than being confined to a very small subgroup.
We’ve also previously presented data from patients treated at 1.8 mg/kg, the recommended Phase 3 dose. In a second-line-only patient population, we saw response rates of more than 80%, with overall survival of 14.3 months.
The next question is where we can take it. We’ve already shared early data with ABBV-706 in combination with a PD-1 checkpoint inhibitor in a heavily pretreated population. That starts to give us a rationale for thinking about the molecule in earlier lines of therapy, where checkpoint inhibition is already part of the treatment landscape.
---
### [](#how-do-the-second-line-response-data-for-abbv-706-inform-the-case-for-eventually-positioning-an-adc-as-a-front-line-therapy)How do the second-line response data for ABBV-706 inform the case for eventually positioning an ADC as a front-line therapy?
**Daejin Abidoye:** In the second-line-plus patient population, we showed a confirmed objective response rate of 56%. But when we looked specifically at the second-line-only population, we saw a response rate of 82%.
To see that level of activity with a single-agent ADC in patients who have already been through treatment gives us a lot of confidence in thinking about what an ADC could potentially do earlier in the treatment pathway.
The way we think about it is that the ADC could drive a high level of tumor control and tumor reduction, and then when you combine that with an immunotherapy, the immunotherapy component may help extend the durability of those responses.
That’s been our strategy, we’ve been vocal about it, and it’s encouraging to see data now being generated that give us confidence in the direction we’re taking.
---
### [](#why-is-tolerability-not-just-efficacy-the-real-competitive-differentiator-for-these-adcs)Why is tolerability, not just efficacy, the real competitive differentiator for these ADCs?
**Daejin Abidoye:** Tolerability has been key in our thinking from the start. It’s not just about putting patients on highly active therapies. It’s about whether patients can tolerate those therapies over time and stay on treatment long enough to benefit.
With traditional chemotherapy in NSCLC, the current paradigm often involves an induction phase followed by maintenance. The induction phase is designed to maximize the initial cytotoxic response, but one of the reasons you then move to a gentler maintenance regimen is that those more intensive therapies can be difficult for patients to stay on long term.
Now imagine if you could have a therapy that provides strong antitumor activity, but that patients can also tolerate for longer. For the patient, that potentially means fewer interruptions or dose modifications and more opportunity to remain on a treatment that is controlling their disease. And when we talk about improving outcomes, we also have to think about what treatment asks of the patient and the impact it has on their quality of life.
That’s one of the reasons we’ve thought so carefully about both molecular design and biomarker selection with our ADCs. With Temab-A, for example, we have seen response rates of 51% to 60% in c-Met-expressing patients with EGFR wild-type disease, depending on the c-Met expression threshold, and 70% in c-Met-expressing patients with EGFR-mutant disease.
For us, finding the right drug for the right patient is only part of the equation. We also want to develop treatments patients can remain on and potentially derive durable benefit from.
---
### [](#how-does-a-fully-realized-biomarker-informed-lung-cancer-program-actually-look-in-practice-five-years-from-now)How does a fully realized biomarker-informed lung cancer program actually look in practice five years from now?
**Daejin Abidoye:** I think where this field evolves over time is toward much more individualized care, and we’re already getting at that through biomarker selection.
With traditional chemotherapy, you can treat a broad population, but not every patient responds, while every patient can potentially be exposed to toxicity. If you can use a biomarker to better identify the population most likely to benefit, you have an opportunity to improve that benefit-risk profile.
We’ve done that already with our first-generation c-Met-directed ADC by identifying a high c-Met-expressing patient population, and we’re building on that experience with Temab-A.
It also changes the way you can think about clinical trials. Rather than designing a very large study and then trying to work out afterward which patients benefited, you can increasingly use biomarkers at the front end to focus development on the patients you believe are most likely to respond. That has the potential to make development much more efficient.
And this isn’t just about targeted therapies. We’ve already seen in NSCLC that PD-L1 expression can help us understand the likelihood of benefit from checkpoint inhibitors. As we get better at bringing those pieces of information together, I think we’re going to become much more sophisticated about matching treatment to individual tumor biology.
The vision is a patient coming into the doctor’s office and the doctor being able to look at that patient’s biology and say, based on what we see in your tumor, this is the therapeutic approach that is most likely to benefit you.
That’s where I see this heading.
---
### [](#how-could-machine-learning-and-artificial-intelligence-ai-compress-the-drug-development-timeline-and-where-is-the-field-already-applying-it)How could machine learning and artificial intelligence (AI) compress the drug development timeline, and where is the field already applying it?
**Daejin Abidoye:** In the era of AI and machine learning, how we think about patient treatment, patient management and drug development will continue to evolve.
One piece of that is identifying the right patient for the right drug at the right time, which we’ve already touched on. But another piece is predictability.
The more near-term question is how we get better at predicting patterns of resistance. If a patient is treated with a drug and then loses response, what is the next therapy that patient should go on? This is an area of active research across the field. Our understanding of the underlying biomarker can give us a better sense of what that patient may benefit from next.
Then there’s the broader drug-development timeline. Using targeted information, algorithms and machine learning to help us predict toxicity, safety, and the right dose more quickly. That could accelerate the path into Phase 3, where ultimately you have to answer whether a treatment can improve on the standard of care.
There’s also the potential to use aggregated real-world data and machine learning to better understand how the current standard of care performs outside a clinical trial. If we can use those tools appropriately, there is an opportunity to get to answers faster, make development more efficient and ultimately bring medicines to patients sooner.
---
**Categories:** Article: Executive Interviews
---
### [What the Lancet Psychiatry Mania Data Gets Right, and What It Still Can't Tell Us](https://www.clinicaltrialvanguard.com/article/psych-pulse/what-the-lancet-psychiatry-mania-data-gets-right-and-what-it-still-cant-tell-us/)
**Published:** September 20, 2026
**Author:** Leonardo Vando, M.D.
**Excerpt:** A new target trial emulation in Lancet Psychiatry challenges antipsychotic-first strategies in early psychotic mania. Here's what it means for continuation…
**Content:**
A target trial emulation just published in *Lancet Psychiatry* reported something that should reopen a clinical argument many of us thought was settled: in patients after a first episode of psychotic mania, [mood stabilizer-based strategies, whether as monotherapy or in combination, carried a meaningfully lower relapse risk than second-generation antipsychotic monotherapy](https://www.thelancet.com/journals/lanpsy/article/PIIS2215-0366(26)00239-7/fulltext?rss=yes) during continuation and maintenance treatment. The BD-CAUSAL Collaboration pulled this signal from real-world data across North America, Chile, and Spain. That’s a geographically wide net, and the finding is consistent enough across sites to deserve serious attention.
My reaction as a clinician-PI is not surprise. My reaction is: why did it take observational data at this scale to surface what many of us see every week in practice?
## [](#the-antipsychotic-first-assumption-deserves-scrutiny)The Antipsychotic-First Assumption Deserves Scrutiny
The clinical logic that drove antipsychotic-first prescribing in early psychotic mania was never unreasonable. Acute psychosis demands rapid symptom control, SGAs deliver it reliably, and continuation of the acute-phase agent feels like the path of least resistance. The APA’s [practice guideline for bipolar disorder](https://www.simhandl.at/downloads/bipolardisorderguidelines-apa.pdf) acknowledges that manic or mixed episodes with psychotic features warrant antipsychotic coverage, which in practice often translates into sustained SGA use well into the maintenance phase. The problem is that “what works in the acute phase” and “what protects against relapse over months and years” are different clinical questions, and we have been conflating them.
The neuropharmacological basis for mood stabilizer superiority in maintenance is not obscure. Lithium’s action on GSK-3 beta signaling and its downstream neuroprotective effects on hippocampal volume are distinct mechanisms from dopamine D2 blockade. Divalproex, [FDA-approved for acute mania since 1995](https://www.ovid.com/journals/exneu/abstract/10.1586/14737175.4.3.349~divalproex-sodium-in-the-treatment-of-adults-with-bipolar?redirectionsource=fulltextview), modulates GABA and histone deacetylase activity in ways that speak more directly to the cycling biology of bipolar disorder. An SGA holding dopamine tone steady does not do the same thing. The BD-CAUSAL signal is plausible on mechanistic grounds, which is partly why a [meta-analysis of maintenance RCTs in bipolar disorder](https://academic.oup.com/ijnp/article/14/8/1029/696968) already showed that no single antipsychotic or mood stabilizer monotherapy demonstrated a significantly reduced risk for both manic and depressive relapse simultaneously. The current trial emulation is consistent with that prior evidence, it just makes the pattern harder to dismiss.
Target trial emulation is an increasingly rigorous tool for exactly this kind of question. [A 2024 call-to-arms in *Lancet Psychiatry* from CAUSALab at Harvard](https://pubmed.ncbi.nlm.nih.gov/38705169/) made the methodological case for this approach in psychiatric research, arguing that properly specified emulations can reduce confounding in ways that observational psychiatry studies rarely achieve. The BD-CAUSAL collaboration is doing what that framework recommends. But emulation is not the same as a randomized trial, and the authors are honest about that, their own conclusion calls for randomized trials to confirm these findings.
## [](#what-first-episode-trials-are-still-getting-wrong)What First-Episode Trials Are Still Getting Wrong
Here is the design problem the BD-CAUSAL data exposes: virtually every continuation and maintenance trial in early psychotic bipolar disorder has been built around the drug in front of it, not around the sequencing question. Trials test whether drug X prevents relapse better than placebo. Almost none are powered to answer whether initiating with a mood stabilizer backbone in the first episode changes the trajectory of illness over two or three years compared to initiating with an SGA and adding a stabilizer only after breakthrough.
Having run SUD and mood disorder trials and treated over 30,000 patients through guided ketamine medicine sessions at Mindbloom, I can tell you that the heterogeneity within “first-episode psychotic mania” is enormous. A 22-year-old with a clear precipitant, family history of lithium response, and no comorbid substance use looks nothing, biologically or clinically, like a 35-year-old with a first recognized episode that is probably not actually their first. The [R-LiNK consortium’s work on predictive biomarkers of lithium response](https://pmc.ncbi.nlm.nih.gov/articles/PMC13442067/), following 169 patients over 24 months, points toward exactly this phenotype-stratification problem: response to mood stabilizers is not uniformly distributed, and we are enrolling as if it were.
Future continuation and maintenance trials need to embed phenotype stratification from enrollment, not as a post-hoc subgroup. That means baseline variables for family history of mood stabilizer response, comorbid substance use disorder status (which affects both medication adherence and relapse risk independently), and a genuine first-episode confirmation window that rules out prior subclinical episodes. Spanish-speaking and Latin American patients were included in the BD-CAUSAL dataset, which is clinically meaningful, but bilingual consent and culturally adapted adherence monitoring are not standard trial infrastructure, and without them, the populations that appear in observational data disappear in RCTs.
I am watching for whether any Phase 3 sponsor moves toward a head-to-head sequencing design in early psychotic bipolar disorder in the next 12 to 18 months, and whether any protocol takes the BD-CAUSAL phenotype heterogeneity seriously enough to pre-specify subgroup analyses around mood stabilizer response predictors at baseline. If they do not, we will have another generation of trials that answer the wrong question cleanly.
## [](#references)References
1. [Lancet Psychiatry, “Comparative effectiveness of mood stabiliser monotherapy versus second-generation antipsychotic monotherapy versus combination therapy for continuation and maintenance treatment after a first episode of psychotic mania: BD-CAUSAL Collaboration”](https://www.thelancet.com/journals/lanpsy/article/PIIS2215-0366(26)00239-7/fulltext?rss=yes)
2. [American Psychiatric Association, “Practice Guideline for the Treatment of Patients With Bipolar Disorder, Second Edition”](https://www.simhandl.at/downloads/bipolardisorderguidelines-apa.pdf)
3. [Expert Review of Neurotherapeutics, “Divalproex sodium in the treatment of adults with bipolar disorder” (FDA approval 1995 reference)](https://www.ovid.com/journals/exneu/abstract/10.1586/14737175.4.3.349~divalproex-sodium-in-the-treatment-of-adults-with-bipolar?redirectionsource=fulltextview)
4. [International Journal of Neuropsychopharmacology, Meta-analysis of maintenance RCTs in bipolar disorder: relapse risk by treatment strategy](https://academic.oup.com/ijnp/article/14/8/1029/696968)
5. [Lancet Psychiatry, July 2024, Szmulewicz AG (CAUSALab, Harvard T.H. Chan School of Public Health): “Target trial emulation in psychiatric research”](https://pubmed.ncbi.nlm.nih.gov/38705169/)
6. [R-LiNK Consortium, “Predictive biomarkers of lithium response: preliminary results from the European R-LiNK initiative” (169 patients, 24-month follow-up)](https://pmc.ncbi.nlm.nih.gov/articles/PMC13442067/)
**Categories:** Psych Pulse
**Tags:** Adaptive Clinical Trial Design, Antipsychotics, Bipolar Disorder, Mood Stabilizers, PSYCH PULSE
---
### [Velocity Clinical Research appoints Justin Starzyk as CEO](https://www.clinicaltrialvanguard.com/news/velocity-clinical-research-appoints-justin-starzyk-as-ceo/)
**Published:** September 19, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Velocity Clinical Research appoints Justin Starzyk as CEO to lead its 70+ research sites across the U.S. and Europe.
**Content:**
Justin Starzyk spent most of the past decade helping United Urology Group grow to roughly 300 physicians across 100 locations before the business was acquired by OneOncology and Cencora. Now he is running a very different kind of healthcare services network: [Velocity Clinical Research](https://velocityclinical.com/velocity-clinical-research-appoints-justin-starzyk-as-chief-executive-officer/), which operates more than 70 fully-owned research sites across the U.S. and Europe, has named him CEO. The choice signals that GHO Capital, which acquired Velocity in 2021, is betting that the discipline needed to scale a physician services business translates directly to scaling a site network chasing sponsor volume.
That bet is not obvious on its face. Velocity’s core argument to sponsors is integration: a single organization owning the sites rather than affiliating with them, with centralized technology and data infrastructure built in. Starzyk’s background is in operational scaling within healthcare services, not clinical trial operations, but his earlier years in pharmaceutical R&D at Abbott Laboratories and AbbVie give him at least a working frame for what sponsors actually need from a site partner. Whether that foundation is deep enough to win credibility with clinical operations teams at large pharma will be one of the early tests of this hire.
Paul Evans, who built [the network from its incorporation in December 2017](https://velocityclinical.com/scale/) through GHO’s ownership, moves to Chair. The structure is a deliberate continuity play: Evans stays close enough to keep sponsor relationships intact while Starzyk takes the operating reins. GHO’s managing partner Mike Mortimer specifically cited “operating discipline” as the reason for the appointment, which reads as a signal that the next growth phase involves margin management and operational efficiency across the site footprint, not just adding locations.
Starzyk’s stated priority is broadening the range of research and centralized services Velocity offers to sponsors, a natural extension of a network this size if it can standardize execution across sites. The cleaner measure of whether this hire works will show up in sponsor re-engagement rates and the breadth of therapeutic areas Velocity can credibly staff, not in the leadership announcement itself.
*Source link: *
**Categories:** News
---
### [Teva Study: Providers Prioritize Tolerability Over Efficacy in Tardive Dyskinesia](https://www.clinicaltrialvanguard.com/news/teva-study-providers-prioritize-tolerability-over-efficacy-in-tardive-dyskinesia/)
**Published:** September 19, 2026
**Author:** Jon Napitupulu
**Excerpt:** Teva study reveals healthcare providers prioritize tolerability and dosing convenience over efficacy when treating tardive dyskinesia in older patients.
**Content:**
Efficacy ranked last. That is the finding that should give VMAT2 inhibitor manufacturers pause: in a discrete choice experiment Teva presented at Psych Congress this week, healthcare providers treating tardive dyskinesia in patients 55 and older weighted tolerability and dosing convenience above clinical effect when choosing between therapies.
The study used a discrete choice experiment design, which forces respondents to make explicit trade-offs rather than rate attributes in isolation, producing a more realistic picture of prescribing priorities than a standard survey. For a condition managed largely with chronic suppression of involuntary movements, that methodology matters: providers treating older patients appear to be optimizing for what their patients will actually tolerate and stick with, not for peak symptom reduction on a rating scale. Teva markets [Austedo (deutetrabenazine)](https://ir.tevapharm.com/news-and-events/press-releases/press-release-details/2017/Teva-Announces-FDA-Approval-of-AUSTEDO-deutetrabenazine-Tablets-for-the-Treatment-of-Tardive-Dyskinesia-in-Adults/default.aspx), approved for tardive dyskinesia in adults in August 2017, and the study’s framing around older patients specifically suggests a positioning effort in a segment where polypharmacy and fall risk make side-effect profiles genuinely consequential.
The competitive context is narrow. [Valbenazine (Ingrezza)](https://www.multivu.com/players/English/8074551-neurocrine-ingrezza-valbenazine-fda-approval/), approved in April 2017, is the other VMAT2 inhibitor in the class, and the two drugs have spent nearly a decade competing on largely overlapping clinical profiles. Defining the prescribing decision through provider preference data, particularly in a geriatric-adjacent population where dosing schedules and adverse event burden carry extra weight, is a legible attempt to carve differentiation where head-to-head efficacy numbers offer limited separation. Whether the attribute preferences Teva identified map cleanly onto Austedo’s actual label profile is the question the source does not answer.
The data were presented as a poster, not published in a peer-reviewed journal, so the sample size and exact utility weights assigned to each attribute are not yet in the public record. The number to track from here is how Teva deploys this preference evidence in payer submissions and formulary negotiations, where real-world tolerability arguments in older patients can shift tier placement more reliably than clinical trial endpoints alone.
*Source link: *
**Categories:** News
---
### [Teva's TEV-749 olanzapine LAI shows 80% stabilization rate in schizophrenia trial](https://www.clinicaltrialvanguard.com/news/tevas-tev-749-olanzapine-lai-shows-80-stabilization-rate-in-schizophrenia-trial/)
**Published:** September 19, 2026
**Author:** Jon Napitupulu
**Excerpt:** TEV-749 olanzapine LAI achieved 80% clinical stabilization in schizophrenia patients during the SOLARIS Phase 3 trial, with durability sustained in open-label e
**Content:**
About 80% of patients with schizophrenia who received TEV-‘749 in the SOLARIS Phase 3 trial achieved clinical stabilization, according to post hoc data Teva presented this week, and that stabilization held across longer follow-up in the open-label extension. That number matters because stabilization is the threshold patients and clinicians actually care about in a maintenance setting, and it is harder to sustain than a statistically significant symptom score at week eight.
The [SOLARIS trial](https://www.hmpgloballearningnetwork.com/node/347624) tested TEV-‘749, a once-monthly subcutaneous formulation of olanzapine, in adults aged 18 to 64 with schizophrenia. The study ran an eight-week randomized, double-blind, placebo-controlled period before moving patients into an open-label extension where the durability data accumulated. The post hoc analyses Teva released this week also examined switching strategies, which tells you the commercial target: patients already on oral olanzapine or another antipsychotic who are candidates for a long-acting injectable but whose clinicians want a transition roadmap before committing.
The subcutaneous route is the design choice that separates TEV-‘749 from the existing olanzapine LAI on the market. [Zyprexa Relprevv](https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2013/022173Orig1s022ltr.pdf), approved in 2009, is an intramuscular formulation and carries a boxed warning for post-injection delirium sedation syndrome, a rare but serious reaction that required patients to be observed for at least three hours at a registered healthcare facility after each dose. A subcutaneous version without that post-injection monitoring requirement would meaningfully reduce the per-dose burden on clinics and patients. Whether TEV-‘749’s label will carry a similar restriction is the regulatory question Teva still needs to resolve, and the answer will determine whether the convenience argument holds in practice.
The once-monthly injectable market for schizophrenia is not empty: [Uzedy](https://www.psychiatrictimes.com/view/fda-approves-risperidone-extended-release-injectable-suspension), a subcutaneous risperidone formulation, received FDA approval in 2025, and paliperidone palmitate products have been established for years. Teva’s differentiation rests on olanzapine’s efficacy profile and on the switch-strategy data it is now putting in front of prescribers. The label language Teva secures, specifically whether post-injection monitoring is required, will be the single most consequential factor in how broadly TEV-‘749 gets adopted after approval.
*Source link: *
**Categories:** News
---
### [CHMP Recommends Ocrevus Approval for Pediatric Relapsing MS](https://www.clinicaltrialvanguard.com/news/chmp-recommends-ocrevus-approval-for-pediatric-relapsing-ms/)
**Published:** September 19, 2026
**Author:** Jon Napitupulu
**Excerpt:** CHMP recommends Ocrevus pediatric relapsing MS approval for children aged 10+, advancing toward European Commission authorization within two months.
**Content:**
The CHMP’s positive opinion on ocrelizumab for children aged 10 and older with relapsing multiple sclerosis, announced September 18, puts the therapy one European Commission decision away from its second major regulatory milestone in four months. The FDA [approved the same indication in May 2026](https://mymsaa.org/news/whats-new-in-ms-research-may-2026/), making ocrelizumab the second disease-modifying therapy with a sanctioned label for pediatric relapsing MS in the United States, after fingolimod. A positive CHMP opinion almost always converts to a Commission authorization, typically within two months of the committee’s recommendation.
What makes the pediatric extension worth watching is how different it is from Ocrevus’s adult origin story. When the [FDA approved ocrelizumab in March 2017](https://www.accp.com/news/index.aspx?i=25), the headline was primary progressive MS, a form of the disease with almost no approved options at the time. The pediatric RMS label carries no such novelty claim, but it addresses a population where treatment options remain limited and where earlier intervention, before accumulated disability compounds, matters clinically. Children and adolescents with relapsing MS are not a large commercial segment, but approvals in younger patients often anchor long-term brand relationships that extend decades into adulthood.
The trial design underlying the CHMP recommendation used weight-based dosing to translate the adult IV regimen to patients as young as 10 weighing at least 25 kg. That threshold matters practically: it defines which newly diagnosed pediatric patients a neurologist can immediately consider for the drug versus those who age or grow into eligibility. The EMA label, once issued by the European Commission, will specify those parameters for EU prescribers, and alignment with the FDA’s weight cutoff would simplify cross-market clinical guidance.
For trial teams, the more immediate consequence is site readiness. EU sites that enrolled pediatric patients in the supporting studies should expect label-driven demand to follow the Commission decision, likely before year-end. Sites without existing pediatric neurology infrastructure will need it before they can participate in any post-approval safety or registry work Roche runs under the new indication.
*Source link: *
**Categories:** News
---
### [FDA approves imlunestrant plus abemaciclib for ESR1-mutated breast cancer](https://www.clinicaltrialvanguard.com/news/fda-approves-imlunestrant-plus-abemaciclib-for-esr1-mutated-breast-cancer/)
**Published:** September 19, 2026
**Author:** Jon Napitupulu
**Excerpt:** FDA approves imlunestrant plus abemaciclib for ESR1-mutated breast cancer, doubling progression-free survival in Phase 3 trials.
**Content:**
Adding abemaciclib to imlunestrant more than doubled median progression-free survival in patients with ESR1-mutated metastatic breast cancer: 11.1 months for the combination versus 5.5 months for imlunestrant alone, a hazard ratio of 0.53. That number, drawn from the 159-patient ESR1-mutant cohort of the [Phase 3 EMBER-3 trial](https://www.mskcc.org/clinical-updates/sabcs-2025-news-updated-ember-3-efficacy-results-for-imlunestrant-with-or-without-abemaciclib-in-er-her2-advanced-breast), is the clinical foundation for the FDA’s full approval of [Inluriyo (imlunestrant) plus Verzenio (abemaciclib)](https://www.prnewswire.com/news-releases/us-fda-approves-inluriyo-imlunestrant-in-combination-with-verzenio-abemaciclib-for-adults-with-er-her2-esr1-mutated-advanced-or-metastatic-breast-cancer-302883515.html) for adults with ER-positive, HER2-negative, ESR1-mutated locally advanced or metastatic breast cancer after at least one prior line of endocrine therapy. The combination is all-oral and, according to the label, requires no new monitoring beyond what either agent carries individually.
ESR1 mutations develop in roughly half of patients with ER-positive, HER2-negative metastatic breast cancer during or after treatment with aromatase inhibitors, and they drive endocrine resistance by making the estrogen receptor constitutively active. The approval is structured around switching both the endocrine backbone and the CDK4/6 inhibitor at clinical progression rather than earlier, a sequencing decision the trial data actively support. Patients in EMBER-3 had received an aromatase inhibitor with or without a CDK4/6 inhibitor in either the adjuvant or metastatic setting before enrolling. Grade 1-2 adverse events accounted for the majority of toxicity; fewer than 4% of patients discontinued either agent permanently due to adverse reactions. Discontinuation of imlunestrant occurred in 1% of patients and abemaciclib in 3.4%.
This is Lilly’s second FDA approval for imlunestrant in under a year. The [monotherapy indication came in September 2025](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imlunestrant-er-positive-her2-negative-esr1-mutated-advanced-or-metastatic-breast) for the same population and setting; the combination label now gives prescribers an evidence-based option for patients who need both ER and CDK4/6 inhibition addressed simultaneously at progression. The tolerability overlap between the two labels matters practically: physicians already familiar with abemaciclib’s diarrhea and neutropenia profile do not face a learning curve for the combination.
The next data milestone for imlunestrant sits in the adjuvant setting. [EMBER-4](https://clinicaltrials.gov/study/NCT05514054), with more than 8,000 patients enrolled across 650-plus sites in over 30 countries, is the largest adjuvant oral SERD trial conducted and expects initial results in 2027. Whether imlunestrant can reduce recurrence in early-stage ER-positive, HER2-negative disease will determine how broadly the drug reshapes endocrine therapy across the breast cancer continuum.
*Source link: *
**Categories:** News
---
### [The RIO Trial's Viral Rebound Data Just Complicated Every HIV Functional Cure Strategy on the Market](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-rio-trials-viral-rebound-data-just-complicated-every-hiv-functional-cure-strategy-on-the-market/)
**Published:** September 18, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Phase 2 RIO trial shows 3BNC117-LS and 10-1074-LS delay HIV viral rebound after treatment interruption, but resistance patterns raise hard questions for…
**Content:**
Picture a regulatory affairs director at an HIV cure-focused biotech opening [the secondary and exploratory outcomes paper from the RIO trial](https://www.nature.com/articles/s41591-026-04644-8) in *Nature Medicine* on a Thursday morning. She already read the primary results published in *The Lancet HIV* on July 30, 2026. She knows the headline: 3BNC117-LS and 10-1074-LS, a dual broadly neutralizing antibody (bNAb) combination, significantly delayed viral rebound after analytical treatment interruption (ATI) compared to placebo. Her pipeline deck is already built around that signal. Then she opens the secondary outcomes data and finds the resistance tables. The question that follows is the one every HIV functional cure sponsor now has to answer: what exactly are you controlling, and for how long?
The RIO trial, a [Phase 2 double-blind, randomized, placebo-controlled study](https://pubmed.ncbi.nlm.nih.gov/42202839/), enrolled adult males living with HIV who had initiated antiretroviral therapy (ART) during recent HIV infection. The primary endpoint measured time to viral rebound in the weeks following ART interruption. Viral rebound was defined according to a pre-specified plasma HIV RNA threshold, with a rigorous multi-measurement rule designed to filter signal from noise., a definition rigorous enough to generate credible primary data in a regulatory context. The bNAb combination met that primary endpoint. But the secondary outcomes published in *Nature Medicine* are where the operational complexity begins, and where most functional cure development programs will find either their runway or their ceiling.
## [](#what-the-resistance-data-actually-shows)What the Resistance Data Actually Shows
The secondary analyses revealed something that disrupts a convenient assumption embedded in most bNAb development narratives: that a longer rebound delay necessarily translates into a durable immunological advantage. Participants who received the 3BNC117-LS and 10-1074-LS combination and eventually experienced viral rebound showed evidence of resistance mutations to one or both antibodies at rebound. This is not a peripheral safety footnote. Resistance emergence under bNAb pressure is a protocol design problem, a future-indication problem, and in some cases a combinatorial strategy problem that resets the entire development calculus.
Consider what this means structurally. The RIO bNAbs target two distinct HIV envelope epitopes: 3BNC117-LS binds the CD4 binding site, while 10-1074-LS targets the V3 glycan supersite. The dual-targeting rationale is precisely designed to raise the genetic barrier to resistance, the same logic that drove the shift from mono- to combination ART in the 1990s. Finding resistance at rebound suggests HIV’s escape pathways remain more accessible than the dual-epitope hypothesis predicted, at least in the subset of participants whose virus rebounded on-study. The exploratory analyses in the *Nature Medicine* paper parsing the breadth and potency of the immune response at rebound are where sponsors need to focus their reading.
The comparison to VRC01 is instructive here. The [HVTN 704/HPTN 085 and HVTN 703/HPTN 081 trials](https://www.hptn.org/news-and-events/press-releases/most-advanced-clinical-trials-testing-broadly-neutralizing-antibody), which tested VRC01 for HIV acquisition prevention and were published in the *New England Journal of Medicine*, demonstrated that single-antibody approaches face an intrinsic ceiling: VRC01-resistant viral strains circulate at meaningful frequency in the population, blunting efficacy in exposed participants before the antibody even starts its job. The RIO trial advances the field by combining two antibodies, but the resistance signals at rebound suggest the ceiling has been raised rather than removed.
## [](#the-ati-design-problem-sponsors-keep-avoiding)The ATI Design Problem Sponsors Keep Avoiding
Analytical treatment interruption is the methodological engine of virtually every HIV functional cure trial being designed right now, and it carries regulatory risks that sponsors persistently underestimate. ATI requires participants to stop suppressive ART under close monitoring, accepting transient viremia in exchange for information about whether an investigational intervention can sustain control. Regulatory guidance specifically addressing ATI design standards remains limited, a gap that creates interpretive latitude but also exposes sponsors to variable reviewer expectations.
The RIO trial addressed this by building in intensive viral load monitoring during the ATI period and defining viral rebound with a six-consecutive-measurement rule, a threshold stringent enough to filter signal from noise. That design choice generates credible primary data. It also means the secondary outcomes, including immune response measures and the resistance profiles at rebound, carry unusual weight, because the primary endpoint integrity allows the exploratory findings to be interpreted without the confounder of a poorly controlled ATI window. Sponsors designing their own ATI protocols should treat the RIO monitoring schedule as a floor, not a ceiling.
Gilead Sciences is running directly adjacent to this territory. At [CROI 2025, Gilead presented Phase 2 data](https://www.gilead.com/news/news-details/2025/gilead-presents-new-hiv-treatment-and-cure-research-data-at-croi-2025-including-an-investigational-long-acting-twice-yearly-therapy-option) showing its investigational twice-yearly regimen combining lenacapavir with bNAbs teropavimab (GS-5423) and zinlirvimab (GS-2872) [met its primary endpoint](https://www.gilead.com/news/news-details/2025/gilead-presents-new-hiv-treatment-and-cure-research-data-at-croi-2025-including-an-investigational-long-acting-twice-yearly-therapy-option) in a Phase 2 study. That program uses a different antibody pair and combines bNAbs with a capsid inhibitor rather than testing bNAbs in isolation during ATI. The RIO secondary data creates an immediate interpretive obligation for Gilead’s team: if dual-bNAb pressure without a backbone antiviral generates resistance mutations at rebound, does adding lenacapavir suppress that escape, or does it merely defer it to a later, harder-to-characterize timepoint?
That question does not yet have an answer in the published record.
## [](#the-operational-takeaway-for-functional-cure-protocol-teams)The Operational Takeaway for Functional Cure Protocol Teams
The most counterintuitive lesson from the RIO secondary outcomes is about what success looks like in a bNAb ATI trial. The conventional instinct is to optimize for rebound delay: longer is better, and a participant who rebounds at week 18 versus week 6 represents a better outcome. But the resistance data inverts that logic in a critical scenario. A participant who rebounds quickly, before significant antibody-driven selection pressure accumulates, may rebound with a virus that is still sensitive to the study antibodies. A participant who rebounds late, after sustained exposure to 3BNC117-LS and 10-1074-LS, may rebound with a partially resistant virus that complicates re-treatment. The duration of viral suppression and the quality of immune reconstitution are not the same variable, and the RIO secondary endpoints make that distinction visible in ways the primary rebound timing data cannot.
For sponsors building Phase 2b or Phase 3 ATI designs, several operational decisions follow from this. First, pre-specify resistance testing at rebound as a primary secondary endpoint, not an exploratory add-on. The RIO team did this; sponsors who demote resistance testing to exploratory status risk generating data the FDA will treat as hypothesis-generating rather than inferential. Second, build viral sequencing into the monitoring schedule at multiple rebound timepoints, not just the first measurement that meets the rebound definition. Third, if your protocol includes any provision for re-initiating ART after rebound, define the re-initiation criteria prospectively and tie them to both quantitative viral load thresholds and resistance phenotype, not quantitative load alone. Protocol-specified re-initiation criteria, rather than reactive ones, are essential to maintaining GCP compliance and supporting a credible regulatory submission.
The RIO trial also carries a demographic specificity that future sponsors must either replicate or explicitly justify diverging from. The trial enrolled adult males, a design choice that controlled hormonal and pharmacokinetic variability but limits the generalizability of the secondary immune response data to female participants, who represent a substantial share of new HIV diagnoses globally. Any Phase 3 program that cites the RIO secondary outcomes as its scientific rationale will need an explicit diversity plan under the FDA’s [established expectations for enrollment](https://pubmed.ncbi.nlm.nih.gov/35382844/) that reflects the disease burden, including in women and in populations with non-subtype B virus, where bNAb breadth data are still thin.
Which brings the analysis back to that regulatory affairs director and her pipeline deck. The RIO primary data gave her a rebound delay signal. The secondary outcomes gave her a resistance map. Those two datasets together define not just what 3BNC117-LS and 10-1074-LS can do, but where the next generation of combination strategies has to go: higher genetic barrier, earlier immune intervention, or both. Sponsors who design Phase 3 protocols that build the RIO resistance findings into their primary endpoint structure, rather than treating them as a background footnote, will be better positioned to shape the evidentiary standard for future functional cure filings.
## [](#references)References
1. [Nature Medicine, “Broadly neutralizing antibodies in adult males living with HIV undergoing analytical treatment interruption: secondary and exploratory outcomes of the phase II randomized controlled RIO trial”](https://www.nature.com/articles/s41591-026-04644-8)
2. [The Lancet HIV / PubMed, “Time to HIV rebound after infusion of long-acting broadly neutralising antibodies 3BNC117-LS and 10-1074-LS and analytical treatment interruption (the RIO trial): a double-blind, randomised, placebo-controlled trial” (July 30, 2026)](https://pubmed.ncbi.nlm.nih.gov/42202839/)
3. [New England Journal of Medicine, “Two Randomized Trials of Neutralizing Antibodies to Prevent HIV-1 Acquisition” (HVTN 704/HPTN 085 and HVTN 703/HPTN 081, VRC01 trials)](https://www.ovid.com/journals/nejm/abstract/10.1056/nejmoa2031738~two-randomized-trials-of-neutralizing-antibodies-to-prevent)
4. [Gilead Sciences, “Gilead Presents New HIV Treatment and Cure Research Data at CROI 2025, Including an Investigational Long-Acting, Twice-Yearly Therapy Option” (2025)](https://www.gilead.com/news/news-details/2025/gilead-presents-new-hiv-treatment-and-cure-research-data-at-croi-2025-including-an-investigational-long-acting-twice-yearly-therapy-option)
5. [PubMed, RIO trial protocol: randomised, placebo-controlled, double-blinded Phase II study of dual long-acting bNAbs for post-treatment viral control in recent HIV infection](https://pubmed.ncbi.nlm.nih.gov/35382844/)
**Categories:** Article: Deep Dive
**Tags:** analytical treatment interruption, bNAb resistance, broadly neutralizing antibodies, HIV functional cure, phase 2 clinical trial
---
### [Certara Launches External Data Accelerator for CDISC Standards Compliance](https://www.clinicaltrialvanguard.com/news/certara-launches-external-data-accelerator-for-cdisc-standards-compliance/)
**Published:** September 18, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Certara launches External Data Accelerator to catch vendor data conformance issues before submission, addressing 70% of clinical study data from external source
**Content:**
More than 70% of clinical study data now arrives from external vendors, each delivering files in its own format on its own schedule. That single operational fact is what makes late-stage data remediation so expensive: by the time a standards problem surfaces at submission, fixing it means renegotiating with vendors, reprocessing datasets, and burning reviewer time that no team has to spare.
Certara’s [External Data Accelerator](https://www.certara.com/fact-sheet/external-data-accelerator-your-standards-first-team-and-technology-for-conformant-vendor-data/) addresses this upstream. The offering pairs senior external data specialists with [Pinnacle 21 Data Exchange](https://www.certara.com/pinnacle-21-enterprise-software/validation/), the validation engine the FDA has used for over a decade to assess submission quality, so incoming vendor data is mapped and checked against CDISC standards before it enters the study database rather than after. The practical shift is when the problem gets caught: a vendor file that fails a conformance rule on arrival can be corrected in days; the same failure discovered during submission review can take months.
Timing matters because [three CDISC standards are converging in 2026](https://www.cdisc.org/standards/timeline), including a public review period running through November for SENDIG v4.0 with Rules and SDTM v3.0. Sponsors managing multiple external data streams through that transition face a version-control problem on top of an already fragmented vendor landscape. The average clinical study draws data from four or more distinct sources, according to Certara’s own analysis of [non-CRF data in clinical trials](https://www.certara.com/blog/all-you-need-to-know-about-non-crf-data-in-clinical-trials/), covering biomarkers, labs, imaging, ePRO platforms, and wearables. Each source is a potential mismatch point when standards change mid-study.
The External Data Accelerator positions standards conformance as a design decision made at study setup, not a cleanup task assigned to a data manager six weeks before a filing deadline. Whether that framing holds operationally depends on how tightly the specialist team can enforce vendor data agreements early enough to influence what the vendor actually delivers. The real test will be whether submission packages built through this model clear FDA technical rejection rates faster than industry baselines.
*Source link: *
**Categories:** News
---
### [Boehringer Ingelheim launches PED Essentials for patient data collection](https://www.clinicaltrialvanguard.com/news/boehringer-ingelheim-launches-ped-essentials-for-patient-data-collection/)
**Published:** September 18, 2026
**Author:** Jon Napitupulu
**Excerpt:** Boehringer Ingelheim's PED Essentials teaches patient organizations to collect and structure patient experience data for regulatory submissions and advocacy.
**Content:**
Patient organizations have long sat at the edge of drug development conversations, collecting lived-experience data that rarely reaches regulators in a form they can use. Boehringer Ingelheim’s new [PED Essentials](https://www.globenewswire.com/news-release/2026/09/17/3363678/0/en/Boehringer-Ingelheim-launches-PED-Essentials-an-online-learning-resource-that-gives-patients-a-stronger-voice.html), launched September 17, is a free e-learning hub built specifically to close that gap: it teaches patient organizations how to collect high-quality patient experience data (PED), structure it for healthcare stakeholders, and use existing PED as an evidence base for advocacy.
The timing matters because the regulatory appetite for this kind of data is real and growing. The FDA’s [April 2023 draft guidance on incorporating clinical outcome assessments into regulatory endpoints](https://www.federalregister.gov/documents/2023/04/06/2023-07243/patient-focused-drug-development-incorporating-clinical-outcome-assessments-into-endpoints-for) (the fourth in its Patient-Focused Drug Development series) spelled out exactly what rigorous patient-centered data should look like to inform a submission. The gap PED Essentials targets is not regulatory willingness but organizational capacity: most patient groups lack the methodological training to produce evidence that meets that bar.
Boehringer co-created the platform with patients and subject-matter experts, which matters for credibility. A tool designed by a pharma company telling advocacy groups how to generate data for that same company’s submissions would face obvious conflicts. The co-creation framing is meant to signal that the curriculum serves the advocacy organizations’ own goals, not just sponsor convenience. Whether the content holds up under scrutiny from regulators or peer reviewers is a question the platform’s first real-world outputs will answer, not its launch materials.
For trial teams, the practical consequence is straightforward: sites working in disease areas with organized patient communities may eventually receive better-structured, more consistent patient-input packages from those groups. That changes how patient advisory work feeds into protocol design and endpoint selection. The marker worth tracking is whether PED collected through this framework starts appearing in regulatory submissions and, if so, whether the FDA’s review feedback treats it as meeting the methodological standards its 2023 guidance describes.
*Source link: *
**Categories:** News
---
### [SkinCure Analysis: Nonmelanoma Skin Cancer Cases 42% Higher Than Official Count](https://www.clinicaltrialvanguard.com/news/skincure-analysis-nonmelanoma-skin-cancer-cases-42-higher-than-official-count/)
**Published:** September 18, 2026
**Author:** Jon Napitupulu
**Excerpt:** New analysis suggests nonmelanoma skin cancer cases are 42% higher than official counts, with annual patient numbers reaching 4.7 million.
**Content:**
The number most often cited to describe the scale of nonmelanoma skin cancer in the United States is 14 years out of date, and a new analysis from SkinCure Oncology suggests the true annual patient count is 4.7 million, not the 3.3 million figure drawn from 2012 data and published in a [2015 study](https://www.prnewswire.com/news-releases/skincure-oncology-cites-new-analysis-indicating-annual-incidence-of-nonmelanoma-skin-cancer-is-42-percent-greater-than-generally-believed-302882553.html). That gap, roughly 42 percent, translates to more than a million patients who fall outside the count that policymakers and insurers have been using to size the problem.
Four separate methodologies in the analysis converge on the same conservative floor. The most straightforward: NMSC concentrates heavily in adults 60 and older, accounting for about 80 percent of cases, and the U.S. population aged 65 and older grew by nearly 50 percent between 2012 and 2026, from 43.1 million to roughly 64 million. Holding individual risk constant at 2012 levels and adjusting only for that demographic shift produces the 4.7 million figure. A compound-growth calculation using one-third of the 2006-2012 annual growth rate arrives at the same number, and federal Medical Expenditure Panel Survey data had already placed the count above 4 million more than a decade ago. The true upper bound, based on extrapolating the actual 2006-2012 trajectory, sits closer to 5.9 million, which means 4.7 million is the floor, not a midpoint estimate.
The policy consequence SkinCure’s CEO Kerwin Brandt draws from this is a push for mandatory national reporting. Unlike most cancers, [nonmelanoma skin cancers are not routinely captured by national registries](https://www.cdc.gov/skin-cancer/statistics/index.html) in the United States, partly because their volume makes comprehensive tracking expensive. That exclusion is precisely what allowed a decade-old benchmark to persist unchallenged. Brandt is also calling for reimbursement structures that encourage early treatment across all recognized modalities, including [Image-Guided Superficial Radiation Therapy](https://www.accessdata.fda.gov/cdrh_docs/pdf17/K173425.pdf), the FDA-cleared nonsurgical approach SkinCure promotes through its network of roughly 500 physicians.
SkinCure has an obvious commercial interest in a higher patient count, and the analysis comes from the company rather than an independent research group, so the 4.7 million figure warrants external validation. Still, the demographic arithmetic is straightforward, and if registry advocates use it to press for mandatory NMSC reporting, the more telling outcome will be whether CMS reimbursement policy for early-stage treatment follows the revised incidence data.
*Source link: *
**Categories:** News
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### [CDC Reports 17% Surge in Rabies Inquiries; Kamada Confirms KedRAB Supply](https://www.clinicaltrialvanguard.com/news/cdc-reports-17-surge-in-rabies-inquiries-kamada-confirms-kedrab-supply/)
**Published:** September 18, 2026
**Author:** Jon Napitupulu
**Excerpt:** Kamada confirms KedRAB supply meets surge in rabies inquiries. CDC reports 17% increase in rabies-related cases through summer 2026.
**Content:**
From July through August 2026, the CDC received 17% more rabies-related inquiries than during the same period last year, and the spike is now hitting the supply chain. Kamada confirmed this week that KedRAB, its human rabies immunoglobulin approved for [post-exposure prophylaxis since 2017](https://www.fda.gov/media/107453/download), has sufficient inventory and manufacturing capacity to meet the accelerated U.S. demand. The confirmation came directly in response to a [CDC Health Alert Network advisory](https://www.cdc.gov/han/php/notices/han00533.html) that flagged both the volume surge and a rise in PEP administration errors nationwide.
The supply question matters because post-exposure prophylaxis is time-sensitive: rabies immunoglobulin must be infiltrated around the wound site and administered alongside the first vaccine dose immediately after a potentially rabid animal contact. Any gap in product availability translates directly into patients receiving incomplete or delayed PEP. Kamada operates three FDA-approved plasma collection centers in Texas, in Houston, San Antonio, and Beaumont, with the Houston and San Antonio sites each designed to collect roughly 50,000 liters of specialty plasma annually at full capacity. That domestic footprint gives the company a short logistics chain to U.S. distributors, which becomes relevant when demand spikes without much warning.
The CDC advisory does more than validate a demand trend; it also documents a quality problem running alongside the volume increase. Errors in how PEP is being administered are rising in parallel with exposure counts, which means clinicians on the front lines are managing unfamiliar protocols under higher caseloads. That context likely drove the advisory’s urgency and, in turn, prompted Kamada’s public supply confirmation rather than a routine commercial statement.
What to watch is whether the 17% inquiry increase translates into a sustained utilization shift through Q4 or whether it reflects a concentrated summer exposure window that normalizes. Kamada has already guided for a significantly stronger second half of 2026; if rabies PEP demand holds at elevated levels into autumn, KedRAB revenue could become a material contributor to that guidance rather than a rounding item.
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**Categories:** News
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### [MIRA Pharmaceuticals Initiates Phase 2a Study of Ketamir-2 for Chemotherapy-Induced Peripheral Neuropathy](https://www.clinicaltrialvanguard.com/news/mira-pharmaceuticals-initiates-phase-2a-study-of-ketamir-2-for-chemotherapy-induced-peripheral-neuropathy/)
**Published:** September 18, 2026
**Author:** Jon Napitupulu
**Excerpt:** MIRA Pharmaceuticals initiates Phase 2a study of Ketamir-2 for chemotherapy-induced peripheral neuropathy, targeting an undertreated condition affecting over ha
**Content:**
More than half of chemotherapy patients develop peripheral neuropathy, and duloxetine remains the only drug with meaningful clinical evidence behind it, prescribed off-label with no regulatory approval specifically for the condition. Against that backdrop, MIRA Pharmaceuticals disclosed September 17 that its Phase 2a program evaluating [Ketamir-2](https://www.psychiatrictimes.com/view/fda-clearance-of-ind-for-ketamir-2-novel-oral-nmda-receptor-antagonist-for-neuropathic-pain), an oral NMDA receptor modulator, has moved toward site initiation after the FDA completed its review of the study protocol. That sequence matters: the FDA cleared Ketamir-2’s IND for neuropathic pain in July 2025, the Phase 1 read was positive in May 2026, and a Phase 2a in chemotherapy-induced peripheral neuropathy (CIPN) is now actively opening sites, all within roughly 14 months.
The Phase 1 trial enrolled 57 healthy volunteers in single and multiple ascending dose cohorts, producing data MIRA described as positive on safety, tolerability, and pharmacokinetics. That controlled, blinded study design is a reasonable foundation for the CIPN program, though Phase 1 data in healthy volunteers says nothing about efficacy in patients whose neuropathy stems from neurotoxic chemotherapy agents. The Phase 2a will need to answer whether the receptor-level activity translates into measurable pain or functional relief in that population.
CIPN is a practical target for a first efficacy trial. [Meta-analyses put incidence around 56%](https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2026.1672180/full) across treated cancer patients, with rates reaching 90% in patients receiving the most neurotoxic regimens. The patient pool is large, the condition is chronic and undertreated, and [approved options remain limited](https://www.medcentral.com/pain/chemotherapy-induced-peripheral-neuropathy-treatments), which makes enrollment messaging and physician interest less of a barrier than in crowded indications. For a small-cap company running a lean clinical program, that access is not trivial.
The disclosure came via an 8-K Regulation FD filing, which means MIRA’s obligation was to release material information simultaneously to all investors rather than to report a formal milestone. Site initiation is a process, not a single date, so the immediate marker to track is when the company discloses first-patient enrollment in the Phase 2a, which will confirm that the protocol review translated into an active, accruing trial.
*Source link: *
**Categories:** News
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### [PEDMARK Hearing Protection Shows No Cancer Efficacy Compromise in Study](https://www.clinicaltrialvanguard.com/news/pedmark-hearing-protection-shows-no-cancer-efficacy-compromise-in-study/)
**Published:** September 18, 2026
**Author:** Jon Napitupulu
**Excerpt:** PEDMARK hearing protection met its primary endpoint in a Phase 2 study, showing significant ototoxicity reduction without compromising cisplatin's anticancer ef
**Content:**
Cisplatin reliably kills pediatric tumors and reliably damages hearing, and the question hanging over PEDMARK has always been whether hearing protection comes at the cost of antitumor effect. Data from the investigator-initiated Phase 2 STS-J01 study, presented September 17 at the SIOP Annual Meeting in San Antonio, answer that directly: the trial met its primary endpoint, showing significant hearing protection with no evidence that sodium thiosulfate interfered with cisplatin’s cancer-killing activity in Japanese pediatric and adolescent patients with non-metastatic solid tumors.
That finding matters beyond Japan. [PEDMARK earned FDA approval in September 2022](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-sodium-thiosulfate-reduce-risk-ototoxicity-associated-cisplatin-pediatric-patients) for reducing cisplatin-associated ototoxicity in patients one month of age and older with localized, non-metastatic solid tumors, but physician adoption anywhere depends on confidence that otoprotection does not blunt chemotherapy. Oncologists treating children with cisplatin-based regimens have historically faced an uncomfortable trade-off, and STS-J01 adds a prospective data point, in a distinct patient population, that the two goals are not in conflict.
The Japan-specific context also carries regulatory weight. STS-J01 is an investigator-initiated study, not a Fennec-sponsored trial, which means it was designed and run by local clinicians with direct interest in the question for their own prescribing environment. A positive result from that kind of study typically carries credibility with national regulators and formulary committees that might otherwise wait for a company-sponsored dossier. For Fennec, a favorable outcome it did not have to run is a useful asset as it pursues market access outside the United States.
The 8-K furnishes the press release as an exhibit rather than incorporating the full dataset, so granular numbers on patient counts, hearing-loss grading, and tumor response rates are not in the filing itself. What Fennec filed is essentially a regulatory disclosure that positive data exist and were announced publicly. The measure to track now is whether STS-J01’s results accelerate a Japanese regulatory submission, since a positive local study without a filing pathway converts to access for patients only on paper.
*Source link: *
**Categories:** News
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### [The Screen Failure You Can Prevent Before the Patient Leaves Home](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-screen-failure-you-can-prevent-before-the-patient-leaves-home/)
**Published:** September 17, 2026
**Author:** Krishma Shah
**Excerpt:** Mayo Test Drive completed at 97.5% across MCI, mild, and moderate dementia patients, here's what that means for Alzheimer's trial pre-screening operations.
**Content:**
The pre-screening visit for an anti-amyloid therapy trial is one of the most operationally expensive moments in Alzheimer’s research. A patient travels to the clinic, a coordinator blocks two to three hours, a neuropsychologist runs a full battery, and then the eligibility picture that emerges sends the case to a multidisciplinary conference. If the cognitive stage turns out to be outside the protocol window, the site absorbs every dollar of that visit as a screen failure. No reimbursement. No enrollment credit. Just cost. A [study published September 15, 2026 in medRxiv](https://www.medrxiv.org/content/10.64898/2026.09.15.26362283v1?rss=1) out of the [Mayo Clinic Rochester Alzheimer’s Disease Treatment Clinic](https://www.mayoclinic.org/departments-centers/alzheimers-disease-treatment-clinic/sections/overview/ovc-20553612) describes what happens when you move part of that assessment out of the clinic and into the patient’s home before the clock starts, and the completion numbers are worth paying attention to.
Of 209 patients evaluated through the clinic for anti-amyloid therapy eligibility, 197 initiated the Mayo Test Drive (MTD) remote self-administered digital cognitive assessment. Of those who started, [97.5% completed a session](https://pmc.ncbi.nlm.nih.gov/articles/PMC12129114/). [Seventy percent of completions happened remotely](https://www.medrxiv.org/content/10.1101/2024.05.24.24307909v1.full.pdf), through a patient-facing healthcare portal, with no staff present. Completion rates held across diagnostic groups: [100% in cognitively unimpaired patients](https://pmc.ncbi.nlm.nih.gov/articles/PMC12129114/), 99.1% in mild cognitive impairment, 97.0% in mild dementia, and 89.5% in moderate dementia. The difference across stages was not statistically significant (p =.12). That last number is the one that matters operationally. The conventional assumption in trial design is that digital self-administered tools work for early-stage patients and fall apart as impairment increases. These data challenge that directly.
## [](#what-the-completion-rate-actually-tells-sites)What the Completion Rate Actually Tells Sites
Sites running anti-amyloid trials under protocols like those supporting lecanemab or donanemab already know how compressed the eligibility window is. The Clarity AD trial, which established much of the evidentiary base for current lecanemab use, reported a discontinuation rate of 17.2%. Every coordinator who has worked a study with that kind of attrition understands that the patients who are most burdensome to screen are often the ones least likely to complete. When a remote assessment tool holds 89.5% completion even in moderate dementia, that is not a convenience feature. It reframes which patients a site can realistically characterize before committing to a full in-person workup.
The operational value runs in two directions. For sites, a completed remote MTD session before a scheduled eligibility visit gives the clinical team a cognitive snapshot that can sharpen the visit agenda, surface potential protocol mismatches earlier, and reduce the proportion of full neuropsychological evaluations that terminate in a screen failure. For sponsors, a site that can pre-characterize patients remotely compresses the time from referral to eligibility determination without adding staff hours or scheduling pressure on already-stretched neuropsychology resources. The study found that administration setting (remote versus in-clinic) did not differ significantly by clinical stage, which means the cognitive data from home sessions and clinic sessions are drawing from the same population, not a healthier, more tech-savvy subset.
Assessor variability is the quieter benefit that does not always make it into feasibility papers. Published inter-rater reliability data for digitally administered cognitive tools like the MoCA show ICC values between 0.91 and 0.96 for total scores under structured conditions, but that reliability depends on assessor training and consistency across sites. [Research on inter-rater reliability for digital cognitive assessments](https://pubmed.ncbi.nlm.nih.gov/42060457/) confirms that even with structured digital formats, scoring variability remains a documented concern. A self-administered platform with fixed item presentation and automated scoring removes that variability at the source. For a multi-site AD trial where neuropsychological assessments are endpoint-adjacent, that consistency has direct implications for data quality, not just operational convenience.
## [](#the-regulatory-gap-sites-cannot-ignore)The Regulatory Gap Sites Cannot Ignore
Before a sponsor builds a remote cognitive assessment into a protocol as anything other than a prescreening tool, there is a regulatory boundary that needs to be stated plainly. Mayo Test Drive has not been cleared or approved by the FDA, according to [Mayo Clinic Laboratories’ own classification policies](https://www.mayocliniclabs.com/customer-service/faq/test-classification/policies). That does not disqualify it from site use in eligibility workflows, where the primary purpose is clinical characterization rather than protocol-defined endpoint collection. But it does define the lane. In the current study, MTD sat alongside, not in place of, the full neuropsychological evaluation conducted at the clinic. That is the right architecture for now.
In December 2023, the FDA finalized guidance titled “Digital Health Technologies for Remote Data Acquisition in Clinical Investigations,” which outlines what sponsors need to demonstrate when using digital tools to collect trial data remotely. The [2023 DHT guidance](https://bmtadvisors.com/fda-issued-final-guidance-on-digital-health-technologies-for-remote-data-acquisition-in-clinical-investigations/) asks for pre-specified fit-for-purpose validation, evidence of user acceptability across the target population, and a data integrity framework for remote transmission. MTD data collected during a routine clinical eligibility workflow does not automatically satisfy those requirements for use as a clinical trial endpoint. Sites and sponsors who read this feasibility study and want to expand the application need to sequence that regulatory validation work before the protocol is written.
Remote digital cognitive assessment also raises documentation considerations that affect how sites record its use. If MTD results inform an eligibility decision that is captured in the source record, the site’s SOP needs to address how that remote session output is stored, who reviews it, and how it connects to the contemporaneous clinical assessment. Among patients who did not initiate MTD in the Mayo study, reported reasons included not receiving or opening the portal message, as well as other or unknown factors. That failure mode lives entirely on the site-operations side, patient outreach, portal enrollment, and follow-up workflow, not the technology. Any site implementing a similar pathway needs a documented outreach protocol with defined follow-up touchpoints before treating non-initiation as a patient factor.
## [](#making-this-work-monday-morning)Making This Work Monday Morning
Sites working with anti-amyloid therapy trials should treat this data as a workflow design prompt, not a validation study. The question it answers is specific: can patients at the cognitive stages targeted by these protocols complete a remote digital assessment reliably enough to use it as a pre-visit characterization tool? At 97.5% overall completion and 97.9% remote completion among those who initiated, the answer from 209 real patients moving through a live clinical eligibility process is yes. That is not a controlled research environment. It is a working clinic.
For sites, the practical step is a pre-screening workflow audit. Map the current path from referral to eligibility determination: how many patients complete a full in-person neuropsychological evaluation and then fall outside the protocol window? If that screen failure rate is substantial, a remote cognitive characterization step added before the visit is worth piloting. Sponsors reviewing site feasibility questionnaires for upcoming AD trials should add a direct question about remote cognitive assessment capacity and portal infrastructure. Sites that already have this pathway built are operationally ahead; those that do not will struggle to pre-characterize patients at the volume these protocols require.
The UK Dementia Research Institute published comparable feasibility data showing ICC values above 0.90 after 10 remote sessions in an autosomal dominant AD cohort, with a [remote digital composite slightly outperforming a traditional composite](https://www.ukdri.ac.uk/publications/remote-digital-cognitive-assessment-trial-ready-alzheimers-disease-cohort-scalable) at separating mutation carriers from non-carriers. The Mayo data now extends that signal into a treatment-eligibility clinical workflow, in a broader and more impaired population. The next site that builds this into its standard pre-screening SOP will not just reduce screen failure costs, it will compress time-to-eligibility-decision for a patient population where weeks matter.
## [](#references)References
1. [medRxiv, “Feasibility of remote self-administered cognitive assessment in an Alzheimer’s disease treatment clinic”](https://www.medrxiv.org/content/10.64898/2026.09.15.26362283v1?rss=1)
2. [BMT Advisors, FDA Final Guidance: “Digital Health Technologies for Remote Data Acquisition in Clinical Investigations” (December 2023)](https://bmtadvisors.com/fda-issued-final-guidance-on-digital-health-technologies-for-remote-data-acquisition-in-clinical-investigations/)
3. [Mayo Clinic Laboratories, Test Classification and Policies: Mayo Test Drive regulatory status](https://www.mayocliniclabs.com/customer-service/faq/test-classification/policies)
4. [2 Minute Medicine, Clarity AD trial discontinuation rate (17.2%) and completion data](https://www.2minutemedicine.com/lecanemab-may-slow-cognitive-decline-in-early-alzheimers-disease/)
5. [UK Dementia Research Institute, Remote digital cognitive assessment in trial-ready Alzheimer’s disease cohort: ICC data and composite performance](https://www.ukdri.ac.uk/publications/remote-digital-cognitive-assessment-trial-ready-alzheimers-disease-cohort-scalable)
6. [PubMed, Diederiks et al.: Inter-rater reliability of digital MoCA administration (ICC 0.91–0.96)](https://pubmed.ncbi.nlm.nih.gov/42060457/)
**Categories:** Clinical Trial Ops Brief
**Tags:** Alzheimer's Disease, anti-amyloid therapy, Clinical Trial Operations, digital cognitive assessment, remote screening
---
### [Medpace Expands Phase I Unit With On-Site Imaging and Specialized Assessments](https://www.clinicaltrialvanguard.com/news/medpace-expands-phase-i-unit-with-on-site-imaging-and-specialized-assessments/)
**Published:** September 17, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Medpace expands Phase I Unit with on-site imaging and specialized assessments to reduce participant dropout and keep trial endpoints aligned.
**Content:**
Phase I oncology and cardiometabolic protocols are asking participants to do more: body composition scans, retinal imaging, echocardiograms, timed against dosing windows that leave little room for travel delays. That friction has a direct cost. When a participant misses or defers an assessment because it requires a separate facility visit, the data gap often cannot be recovered.
Medpace’s [Phase I Unit in Cincinnati](https://www.medpace.com/blog/supporting-complex-phase-i-studies-through-integrated-imaging-and-specialized-assessments/) has expanded to absorb several of those assessments on site. Licensed optometrists now perform ophthalmic evaluations inside the unit, covering visual acuity, slit-lamp and posterior segment exams, OCT and OCTA, fundus photography, and microperimetry. Echocardiography and ultrasound are also available through qualified partners who bring personnel and equipment directly into the unit rather than routing participants elsewhere. For modalities that genuinely require dedicated facilities, such as MRI or DXA, the unit coordinates through external imaging partners while routing reads through [Medpace Core Labs](https://www.3ds.com/newsroom/press-releases/medidata-and-medpace-sign-agreement-integrate-imaging-and-clinical-data-single-platform), which uses Medidata Rave Imaging to consolidate site data entry and image uploads into a single platform.
The operational logic is straightforward. Early-phase trials already impose a significant schedule on participants: repeat dosing visits, PK blood draws, safety monitoring. Each additional off-site assessment adds a logistical dependency that can shorten the recruitment window or inflate dropout risk. Pulling those assessments inside an [85-bed, 60,000-square-foot facility](https://www.medpace.com/blog/bridging-early-phase-insight-to-late-phase-success-inside-medpaces-new-phase-i-unit-facility/) established in 2008 compresses the coordination problem and keeps imaging endpoints on the same visit schedule as everything else. The FDA has long recognized that imaging technologies provide critical biomarkers in early development, and sponsors are increasingly encoding those endpoints in Phase I protocols rather than deferring them to later stages.
The practical test for this model will be protocol complexity: whether the on-site and coordinated modalities together cover what a sponsor writing a novel biologics or metabolic disease FIH trial actually needs, without forcing workarounds that reintroduce the vendor management burden the integrated model is designed to eliminate.
*Source link: *
**Categories:** News
---
### [RBQM vs. RBDM: Data Teams Must Link Quality Plans to Management Protocols](https://www.clinicaltrialvanguard.com/news/rbqm-vs-rbdm-data-teams-must-link-quality-plans-to-management-protocols/)
**Published:** September 17, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Risk-based quality management requires linking quality plans to data management protocols. EU regulators now expect documented traceability between RBQM strateg
**Content:**
Nine out of ten clinical trials now use risk-based quality management approaches, according to ACRO’s 2025 survey, yet most data teams still conflate RBQM with a narrower discipline that sits inside it: risk-based data management, or RBDM. The confusion is operational, not semantic. When a data team treats the two as synonyms, it tends to apply data-level decisions to quality problems that require cross-functional governance, or vice versa, and neither gets resolved properly.
The distinction sharpened after [ICH E6(R3) was published on January 6, 2025](https://www.thefdalawblog.com/2025/02/the-ich-e6r3-guideline-a-major-update-to-good-clinical-practice/). That revision, read alongside [ICH E8(R1) from 2021](https://casrai.org/guides/ich-e8r1-general-considerations-for-clinical-studies), pushed quality-by-design from a recommended posture to an expected one. RBQM, as the broader framework, covers how a sponsor identifies critical-to-quality factors, allocates oversight resources, and responds when signals suggest a systemic problem. RBDM lives within that structure: it governs how data is collected, cleaned, queried, and reviewed, with risk tolerance defined at the data element level rather than the study level. One is a governance architecture; the other is a data operations discipline.
For a data team, the practical consequence is about scope. RBDM decisions, such as which fields trigger automated queries or how missing data thresholds are set, feed upward into the RBQM risk register. If the data team owns only the downstream piece without visibility into what quality risks the study has formally prioritized, it optimizes for clean data in the wrong places. EU sites already face this pressure: Annex 1 of E6(R3) took legal effect in the European Union on July 23, 2025, which means that gap between documented RBQM strategy and actual data oversight practices is now a regulatory exposure, not just an efficiency problem.
The immediate pressure point for most organizations is documentation. [ACRO’s survey](https://www.acrohealth.org/rbqm-summary-report/) found broad RBQM adoption in principle, but adoption in practice requires that the linkage between quality risk decisions and data management protocols be traceable on audit. Teams that have absorbed RBQM as a monitoring concept without restructuring how data management plans connect to the quality plan will find that gap visible to regulators reviewing E6(R3)-era studies.
*Source link: *
**Categories:** News
---
### [Coya Therapeutics Enrolls 100th Patient in ALSTARS Trial of COYA 302 for ALS](https://www.clinicaltrialvanguard.com/news/coya-therapeutics-enrolls-100th-patient-in-alstars-trial-of-coya-302-for-als/)
**Published:** September 17, 2026
**Author:** Jon Napitupulu
**Excerpt:** Coya Therapeutics enrolls 100th patient in ALSTARS trial of COYA 302 for ALS, reaching a key recruitment milestone for the Phase 2/3 study.
**Content:**
Coya Therapeutics reported on September 15, 2026, that the 100th patient has been enrolled in ALSTARS, its Phase 2/3 trial of [COYA 302](https://www.clinicaltrialsarena.com/news/fda-coya-therapeutics-trial-coya-302/) for amyotrophic lateral sclerosis. That number matters for a specific reason: ALS trials routinely struggle to recruit, and hitting triple digits is the point at which enrollment momentum typically becomes readable enough to project a completion date with some confidence. Coya says current trends support a full enrollment forecast, though the filing does not name a specific target date.
COYA 302 pairs low-dose IL-2 with CTLA-4 Ig, a combination designed to expand regulatory T cells and dampen the neuroinflammatory activity implicated in ALS motor neuron loss. The [double-blind, placebo-controlled ALSTARS trial](https://ir.coyatherapeutics.com/news/news-details/2026/Coya-Therapeutics-Announces-Enrollment-of-100th-Participant-in-Phase-23-ALSTARS-Trial-of-COYA-302-for-the-Treatment-of-ALS/default.aspx) (NCT07161999) is testing that mechanism in a multi-center setting, with functional decline and safety as central measures. The immune angle is notable because [riluzole and edaravone](https://pmc.ncbi.nlm.nih.gov/articles/PMC9540964/), the mainstays of ALS management, target glutamate toxicity and oxidative stress, leaving neuroinflammation largely unaddressed by approved therapies.
The enrollment milestone also arrives at a moment when the ALS treatment field has fewer options than it did two years ago. Relyvrio, approved in September 2022, was withdrawn from the market by Amylyx in early 2024 after a confirmatory trial failed, narrowing the field back to its older standards. That contraction sharpens attention on mechanistically distinct approaches like COYA 302, without guaranteeing anything about efficacy outcomes.
The number to watch from here is not enrollment itself but the pace at which Coya translates current trends into a declared completion date. A public enrollment target would let observers assess how much runway the trial has before a primary readout becomes schedulable, and whether the company’s financing is sized to cover it.
*Source link: *
**Categories:** News
---
### [BH-30643 achieves 45% response rate in osimertinib-resistant NSCLC patients](https://www.clinicaltrialvanguard.com/news/bh-30643-achieves-45-response-rate-in-osimertinib-resistant-nsclc-patients/)
**Published:** September 17, 2026
**Author:** Jon Napitupulu
**Excerpt:** BH-30643 achieves 45% response rate in osimertinib-resistant NSCLC patients with C797S mutations, showing 88% disease control in Phase 1/2 trial.
**Content:**
Forty patients is a small number to move a stock, but the 45% objective response rate BlossomHill Therapeutics reported Monday from its SOLARA trial of BH-30643 is drawing attention precisely because the patients involved have almost nowhere else to go. All 40 had EGFR C797S mutations, the resistance mechanism that emerges after osimertinib fails, and 98% had in fact received osimertinib before enrolling. Getting nearly half of them to respond is the kind of result that makes NSCLC oncologists pay close attention to a Phase 1/2 dataset.
The [disease control rate reached 88%](https://www.cancernetwork.com/view/novel-macrocyclic-egfr-tki-shows-responses-in-egfr-c797s-positive-nsclc) (35 of 40 patients), and as of an August 10 efficacy follow-up, 63% of the cohort remained on treatment at a median follow-up of 6.9 months. That durability number matters more than the ORR alone: responses that hold past six months suggest BH-30643 is not just clearing a low bar in a heavily pretreated group but producing something that persists. BlossomHill [disclosed the data](https://www.sec.gov/Archives/edgar/data/1839970/000119312526391328/d168153d8k.htm) in a Regulation FD filing tied to a mini-oral presentation at the International Association for the Study of Lung Cancer annual meeting.
BH-30643 is a macrocyclic OMNI-EGFR tyrosine kinase inhibitor, a structural class designed to accommodate the conformational change that C797S forces on the EGFR binding pocket. The mechanism is not novel as a concept, but most TKIs in earlier generations could not bridge C797S with T790M simultaneously; the SOLARA cohort included patients with and without concurrent T790M, and the 45% ORR held across both subgroups. That breadth, if it survives dose-expansion scrutiny, addresses a real clinical headache: oncologists treating post-osimertinib patients often cannot wait for molecular subtyping before deciding on next therapy.
The trial is still enrolling (NCT06706076), and the 6.9-month follow-up is short enough that median duration of response has not matured. The number to track from here is [how many of those 25 patients still on treatment](https://www.marketscreener.com/news/blossomhill-therapeutics-presents-updated-data-from-ongoing-phase-1-2-solara-trial-demonstrating-ant-ce785bdddd8df324) maintain response at the 12-month mark, which is where regulators and payers will start asking harder questions about whether a single-arm Phase 1/2 ORR can anchor an accelerated approval submission.
*Source link: *
**Categories:** News
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### [Summit Therapeutics Presents Updated Overall Survival Data for Ivonescimab in EGFR-Mutated NSCLC](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-presents-updated-overall-survival-data-for-ivonescimab-in-egfr-mutated-nsclc/)
**Published:** September 17, 2026
**Author:** Jon Napitupulu
**Excerpt:** Summit Therapeutics presents updated overall survival results for ivonescimab in EGFR-mutated NSCLC from the Phase III HARMONi trial.
**Content:**
Investors who bought Summit Therapeutics on the strength of its PFS data now have a harder number to work with: updated overall survival results from the 438-patient [HARMONi Phase III trial](https://www.targetedonc.com/view/updated-harmoni-analysis-shows-consistent-os-benefit-with-ivonescimab-in-egfrm-lung-cancer) were presented September 15 at the IASLC World Conference on Lung Cancer, and the company filed an 8-K the same day to flag the release. The trial tests ivonescimab plus chemotherapy against placebo plus chemotherapy in patients with locally advanced or metastatic, EGFR-mutated nonsquamous NSCLC who progressed on a third-generation EGFR TKI, the population where standard options run thin after osimertinib failure.
[Ivonescimab](https://pmc.ncbi.nlm.nih.gov/articles/PMC12665695/) is a bispecific antibody that blocks both PD-1 and VEGF simultaneously, a dual mechanism that distinguishes it from checkpoint monotherapies in this setting. HARMONi carries co-primary endpoints of progression-free survival and overall survival, meaning the OS readout presented at WCLC is not exploratory: it counts. Whether the updated OS curves hold the same directional consistency seen in PFS will shape how seriously regulators treat the BLA Summit will eventually need to file, and the company already holds FDA Fast Track designation for this exact indication.
The practical question is magnitude. PFS improvements in post-EGFR-TKI NSCLC have a track record of not translating cleanly into OS benefit, partly because subsequent therapies muddy the survival curves. A durable OS signal from a 438-patient randomized trial in a molecularly defined population is harder to dismiss than a PFS advantage alone, but the conference presentation will determine whether the data are mature enough to be persuasive or simply trending in the right direction. The 8-K text the SEC received is truncated, so full hazard ratios and confidence intervals are not yet in hand from this filing.
Summit’s next concrete marker is the full dataset from WCLC, which will show whether the OS benefit crosses a pre-specified threshold or remains an encouraging trend requiring longer follow-up before a regulatory submission can anchor on it.
*Source link: *
**Categories:** News
---
### [Roche's Lunsumio combo meets primary endpoint in Phase III follicular lymphoma trial](https://www.clinicaltrialvanguard.com/news/roches-lunsumio-combo-meets-primary-endpoint-in-phase-iii-follicular-lymphoma-trial/)
**Published:** September 17, 2026
**Author:** Jon Napitupulu
**Excerpt:** Roche's Lunsumio combo met its primary endpoint in Phase III follicular lymphoma trial, beating rituximab plus lenalidomide on progression-free survival.
**Content:**
Roche’s mosunetuzumab-lenalidomide combination beat rituximab plus lenalidomide on progression-free survival in relapsed or refractory follicular lymphoma, according to top-line Phase III data from the roughly 400-patient [CELESTIMO study](https://ashpublications.org/thehematologist/article/doi/10.1182/hem.V19.6.2022610/486906/CELESTIMO-A-Randomized-Phase-III-Trial-Examining) released Wednesday. The trial hit its primary endpoint with results described as both statistically significant and clinically meaningful, giving Roche the randomized Phase III survival data it has needed to push [Lunsumio](https://www.gene.com/media/press-releases/14978/2022-12-22/fda-approves-genentechs-lunsumio-a-first) beyond its current accelerated approval, which the FDA granted in December 2022 based on response rate alone in patients who had received at least two prior lines of therapy.
The CELESTIMO population is broader than Lunsumio’s approved indication: patients needed only one prior line of treatment to enroll, and the comparator arm used rituximab plus lenalidomide, a combination with a long track record in this setting. Beating that regimen on PFS in a randomized trial carries more regulatory weight than the single-arm response data that supported accelerated approval, and Roche will likely use these results to seek conversion to full approval and to expand the label to earlier lines. The question regulators and oncologists will ask is how large the PFS benefit was, a figure Roche has not yet released ahead of a conference presentation.
The competitive context makes the magnitude of that benefit consequential. [Epcoritamab in combination with lenalidomide and rituximab](https://www.mskcc.org/news/new-trial-shows-a-3-drug-combination-benefits-people-with-relapsed-follicular-lymphoma) received FDA approval in November 2025 for relapsed or refractory follicular lymphoma, meaning bispecific antibody-based regimens are no longer a novel category in this disease. Mosunetuzumab is delivered subcutaneously in a fixed-duration course, a design that differs from some continuous-therapy approaches, but clinicians choosing between bispecific-based combinations will weigh the actual PFS curves, not just the mechanism. A narrow separation from the rituximab-lenalidomide arm would read differently than a substantial one when placed alongside data from competing regimens.
Full results are expected at an upcoming medical meeting. The number that will define how this data moves the field is the hazard ratio for progression-free survival, and that is the figure worth waiting for before drawing conclusions about where mosunetuzumab lands in the treatment sequence.
*Source link: *
**Categories:** News
---
### ["Hard to Reach" Is a Sponsor Operations Failure, Not a Population Problem](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/hard-to-reach-is-a-sponsor-operations-failure-not-a-population-problem/)
**Published:** September 16, 2026
**Author:** Krishma Shah
**Excerpt:** The Lancet calls out "hard to reach" as a stigmatizing label. For clinical ops, it's a site selection failure with a measurable fix.
**Content:**
The site activation list lands in the CTM’s inbox, and the enrollment diversity target sits in section 5.3 of the protocol. Forty-two sites selected, predominantly academic medical centers in urban research corridors, mostly the same sites that ran the last three studies. The sponsor’s diversity action plan promises 25% enrollment from Black and Hispanic participants. The screen failure report at week eight shows 11%. Nobody is surprised, and nobody changes the list.
A perspectives piece published this month in [The Lancet](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01824-6/fulltext?rss=yes) makes a point that clinical operations teams should read as a direct indictment of standard site selection methodology. The authors document how the label “hard to reach” gets applied routinely to minority ethnic populations in biomedical recruitment, framing their under-representation as a community characteristic rather than a research infrastructure failure. Operationally, that framing has a concrete consequence: sponsors keep selecting sites that cannot reach those communities, then wonder why their diversity targets go unmet.
The label does the damage before the protocol is even written.
## [](#where-the-failure-actually-lives)Where the Failure Actually Lives
Sponsors often treat diversity enrollment as a recruitment problem, something to be fixed in weeks ten through twenty when the demographic breakdown looks wrong. That is too late by months. The decision that determines whether a trial can enroll diverse participants happens at site selection, typically during feasibility, before a single IRB submission has been filed. A site that draws its patient population from a single ZIP code, staffs its study coordinators entirely in English, and has no existing relationship with community health workers cannot fix its diversity numbers by distributing flyers.
The [BMJ Open study tracking racial and ethnic diversity in trials reported to ClinicalTrials.gov from 2009 to 2024](https://pmc.ncbi.nlm.nih.gov/articles/PMC12330421/) found that [only 54% of studies reported both race and ethnicity data in 2024](https://scholars.duke.edu/publication/1823875). Sponsors cannot manage what they do not measure, and for roughly half of all trials on the books, the demographic composition of enrolled participants remains invisible in the public record. That is not a data quality problem alone. It reflects how diversity has been treated operationally: aspirational language in the protocol, minimal infrastructure behind it.
The [Clinical Trials Transformation Initiative’s Diversity Project](https://ctti-clinicaltrials.org/access/ctti-recommendations-increasing-diversity-in-clinical-trials/), which began in 2019, frames the fix as requiring long-term, transformative strategies rooted in organizational commitment to building clinical trial research infrastructure that is more responsive to historically underrepresented populations. That is the right diagnosis. The operational translation is blunter: infrastructure means sites, and sites mean the feasibility questionnaire needs new questions.
Standard feasibility questions ask about investigator experience, patient volume, and competing protocols. Sites I work with across our network rarely get asked, at the feasibility stage, whether they have bilingual coordinators on staff, whether they conduct community outreach with local federally qualified health centers, or whether their IRB has experience with community advisory board engagement. Those omissions are not neutral. They systematically favor established research sites that already serve research-experienced, predominantly white patient populations.
## [](#what-purpose-built-infrastructure-actually-produces)What Purpose-Built Infrastructure Actually Produces
The counterargument from sponsors is predictable: diverse sites are slower to activate, carry higher regulatory risk, and have less experience with complex protocol requirements. That concern is real for sites with no prior trial experience. But it conflates two different categories. Community-affiliated sites with established patient relationships and trained coordinators are not inexperienced sites; they are sites that have been excluded from sponsor feasibility lists because their patient demographic did not match the historical enrollment target.
The evidence on what happens when site selection methodology actually changes is specific. [Alliance Clinical’s published outcomes from an ongoing diabetic peripheral neuropathic pain study](https://allianceclinicalnetwork.com/wp-content/uploads/2025/02/acn-guide-practical-approach-012325.pdf) show that five of its purpose-built diversity-focused sites accounted for 37% of total enrollment across the entire study. Approximately 20% of participants enrolled across all sites were from racial and ethnic minority groups. Five sites, 37% of enrollment. That ratio does not happen by accident or by adding a flyer to the site’s waiting room. It comes from site selection criteria that weighted community embeddedness alongside the standard feasibility metrics.
Community-based participatory research methods show a similar pattern at a larger scale. A 10-year systematic review of CBPR approaches in clinical trial recruitment found that more than 85% of studies using these methods saw statistically positive outcomes in recruiting racial and ethnic minority participants. The mechanism is not mysterious: communities that have been involved in designing recruitment, that have relationships with the research team, and that are not being approached as passive subjects show up. The operational question is whether the sponsor’s site activation model creates the conditions for that participation to happen.
## [](#turning-the-compliance-box-into-a-clinops-workplan)Turning the Compliance Box Into a Clinops Workplan
The [Food and Drug Omnibus Reform Act of 2022](https://www.thefdalawblog.com/2023/01/fdora-enacted-hpm-issues-detailed-summary-and-analysis/) established the requirement for [Diversity Action Plans](https://www.fda.gov/media/184768/download) for Phase 3 drug trials and certain device investigations, making demographic enrollment targets a regulatory obligation rather than a voluntary aspiration. Sponsors are now filing those plans. The gap between filing a plan and building the site infrastructure to execute it is where most programs are currently sitting.
The operational shift that closes that gap starts at feasibility. Add three questions to your standard feasibility template and weight the responses in site selection scoring: Does the site have bilingual coordinators for the target language population in the protocol’s geography? Does the site have an existing referral relationship with a community health organization, FQHC, or faith-based health program? Has the site enrolled at least one prior trial where 25% or more of participants were from a racial or ethnic minority group? Sites that cannot answer yes to any of those questions are not diverse-enrollment-capable regardless of their patient volume numbers, and activating them while expecting them to hit diversity sub-targets is the operational loop that produces the week-eight screen failure report.
For sites, the parallel move is documentation. If your site has community relationships, coordinator language capacity, or a track record of diverse enrollment, that evidence needs to be in your feasibility response, quantified and specific. Sponsors relying on [terminology shifts away from “hard to reach”](https://pmc.ncbi.nlm.nih.gov/articles/PMC10225256/) in their protocol language but not in their site selection criteria will keep selecting the same forty-two sites. Sites that can show enrollment demographics from prior studies, and name the community organizations they work with, give a sponsor the data to make a different decision.
The Lancet piece argues that the “hard to reach” label reflects a judgment about infrastructure, not about communities. Clinical operations teams have the lever to prove that. It lives in the feasibility questionnaire, and the next activation list is where it gets used or ignored.
## [](#references)References
1. [The Lancet, “A positive difference: unimagined communities, reimagined research”](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01824-6/fulltext?rss=yes)
2. [BMJ Open / PMC, “Racial and ethnic diversity in clinical studies reported to ClinicalTrials.gov, 2009–2024”](https://pmc.ncbi.nlm.nih.gov/articles/PMC12330421/)
3. [Clinical Trials Transformation Initiative, CTTI Recommendations: Increasing Diversity in Clinical Trials](https://ctti-clinicaltrials.org/access/ctti-recommendations-increasing-diversity-in-clinical-trials/)
4. [Alliance Clinical, “A Practical Approach to Building Diversity into Clinical Trials Using Purpose-Built Sites”](https://allianceclinicalnetwork.com/wp-content/uploads/2025/02/acn-guide-practical-approach-012325.pdf)
5. [University of Kentucky Scholars, “Community-Based Participatory Research (CBPR) to Enhance Participation of Racial/Ethnic Minorities in Clinical Trials: A 10-Year Systematic Review”](https://scholars.uky.edu/en/publications/community-based-participatory-research-cbpr-to-enhance-participat/)
6. [FDA, Diversity Action Plans: Food and Drug Omnibus Reform Act of 2022 (FDORA) Guidance](https://www.fda.gov/media/184768/download)
7. [PMC, Terminology shifts from “hard to reach” to “underrepresented populations” in clinical trial guidance](https://pmc.ncbi.nlm.nih.gov/articles/PMC10225256/)
**Categories:** Clinical Trial Ops Brief
**Tags:** Clinical Operations, Diversity Recruitment Operations, Enrollment Strategy, Site Selection, underrepresented populations
---
### [Benchmark Scores Don't Save Patients: Why Clinical AI Needs RCT-Grade Evidence Before It Touches a Workflow](https://www.clinicaltrialvanguard.com/opinion/benchmark-scores-dont-save-patients-why-clinical-ai-needs-rct-grade-evidence-before-it-touches-a-workflow/)
**Published:** September 16, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Med-Gemini scores 91% on MedQA. OpenAI's o1-preview hits 96%. Neither number tells you what happens when the algorithm meets a real patient at 2 a.m.
**Content:**
Pull up any clinical AI product deck circulating in 2026 and you will find the same table: benchmark scores, leaderboard rankings, and a column of percentages that climb toward 100. OpenAI’s o1-preview achieved [96% accuracy on MedQA-USMLE and 99% on MMLU Medical Genetics](https://arxiv.org/html/2410.01553v1). Google’s Med-Gemini has been reported to achieve strong scores on MedQA. JSL Medical-LLM 78B has been reported to achieve high scores on Medical Genetics benchmarks. The numbers are real. The inference drawn from them, that these systems are clinically ready, is not.
Nature Medicine published a perspective this month arguing exactly that: [prospective evidence for conversational medical AI is hard, but non-negotiable](https://www.nature.com/articles/s41591-026-04639-5). The argument is straightforward on its surface. Benchmarks test recall under controlled conditions. Clinical workflows punish systems under chaotic ones. Ergo, developers must generate prospective, real-world evidence before deployment. The framing sounds reasonable. The consensus around it is growing. And that consensus is the problem, because agreeing that prospective evidence matters is easy, while actually designing trials that can generate it is where the whole enterprise breaks down.
## [](#the-leaderboard-illusion)The Leaderboard Illusion
Consider what a 96% accuracy score on MedQA actually measures. The exam draws from a corpus of structured, curated clinical vignettes. The test-taker, human or algorithm, receives a clean question with a defined set of answer choices and no interruptions. Now place that same system in a teaching hospital at 2 a.m., fielding an ambiguous query from a fatigued intern who has misspelled three terms, omitted the patient’s renal function, and is simultaneously managing two other conversations. The benchmark was never measuring performance in that environment. It was measuring performance in an environment that does not exist.
IBM Watson for Oncology learned this at significant cost. Operational between 2017 and 2019, the system generated treatment recommendations built on [hypothetical cases created by Memorial Sloan Kettering physicians rather than real patient outcomes](https://www.pertamapartners.com/insights/ai-project-failure-case-studies), a fact that emerged only after hospitals in India, Thailand, and elsewhere had deployed it. The algorithm performed impressively on the scenarios it was trained to handle. Real oncology presented different scenarios entirely.
The Watson collapse was not a data science failure. It was a human factors and integration failure dressed as one. That distinction matters enormously for how sponsors design validation trials for conversational AI today.
## [](#a-consensus-without-a-protocol)A Consensus Without a Protocol
The venture capital community is moving at a pace that assumes the validation problem is already solved. In 2025, [AI companies captured 55% of all health tech funding](https://www.bvp.com/atlas/state-of-health-ai-2026), up from 37% in 2024 and 29% in 2022. The average health tech deal size climbed 42% year-over-year, from $20.7 million to $29.3 million. With that capital velocity comes an investor timeline that treats benchmark performance as sufficient proof of concept and prospective clinical validation as a post-commercial formality.
Clinicians occupy a different position. The same systems investors are funding land in their workflows as tools they must supervise, correct, and ultimately answer for. A hospitalist who acts on a conversational AI recommendation that misses a drug interaction does not share liability with the algorithm’s developer. That asymmetry shapes how physicians read a product deck: with considerably more skepticism than the funding round suggests is warranted.
Regulators sit in a third position entirely. The FDA released its AI/ML Software as a Medical Device Action Plan in [January 2021](https://starfishmedical.com/resource/fda-action-plan-for-ai-ml-in-samd-software-as-a-medical-device/), acknowledging openly that its traditional regulatory paradigm was not designed for adaptive AI technologies. Five years later, that acknowledgment has not resolved into a clear prospective evidence standard for conversational AI specifically. Sponsors submitting De Novo requests or 510(k)s for conversational clinical AI are navigating guidance that was not written with these systems in mind, in a framework that still treats most AI outputs as passive decision support rather than active clinical intervention.
Three stakeholders, three incompatible timelines, and one shared rhetorical commitment to “rigorous evidence.” Which raises the practical question no product deck answers: what does a prospective validation trial for conversational medical AI actually look like?
## [](#designing-for-failure-not-performance)Designing for Failure, Not Performance
The Nature Medicine perspective pushes toward RCT-grade evidence. That framing is correct but incomplete. A randomized controlled trial designed to measure conversational AI performance against a primary accuracy endpoint will reproduce the benchmark problem at larger scale. If the outcome measure is “did the system give the right answer,” you have built a very expensive MedQA administration. The outcome measure must be clinical: patient safety events, diagnostic delay, medication errors caught or created, clinician time to decision, and, critically, system behavior at the edges of its training distribution.
Edge behavior is where every clinical AI deployment eventually lives. A conversational system performs well on the modal presentation of a common condition. Its value proposition, and its risk profile, are determined by what it does with the atypical presentation, the co-morbid patient, the query phrased in clinical shorthand, and the scenario that falls outside its training corpus. Trial designs that do not deliberately stress-test those conditions are not generating prospective evidence of clinical safety. They are generating prospective evidence of typical-case performance, which the benchmark already provided at a fraction of the cost.
Human factors endpoints belong in the primary endpoint structure, not the appendix. If a conversational AI system causes a clinician to override their own correct instinct because the interface presented the AI recommendation with inappropriate confidence, a failure mode with well-documented precedent in clinical decision support literature, that event must be captured and reported. A trial that measures only system accuracy while leaving clinician behavior unmeasured has answered the wrong question.
The FDA’s existing human factors guidance for medical devices provides a starting framework, but conversational AI presents challenges that static device guidance does not contemplate. A diagnostic algorithm produces an output. A conversational AI system conducts a dialogue, and the clinical risk emerges from the interaction pattern over time, not from any single output. Designing trials that capture longitudinal interaction patterns, not just endpoint accuracy snapshots, requires methodological development that the field has not yet produced at scale.
Sponsors who move toward that methodological standard now will not just satisfy a future regulatory requirement. They will hold the only asset that actually distinguishes a clinical-grade conversational AI from a very sophisticated chatbot with a medical vocabulary: evidence that the system performs safely when real patients, real clinicians, and real institutional chaos enter the equation. The benchmark proves the algorithm learned the textbook. The prospective trial proves it survived contact with the hospital. Those are not the same thing, and the gap between them is exactly where patients get hurt.
The first sponsor to file a pre-market submission for a conversational medical AI backed by a methodologically rigorous prospective trial, one with human factors endpoints, edge-case stress testing, and longitudinal interaction data, will set the evidentiary bar for every system that follows. Every later filing gets measured against it. The developers currently polishing their MedQA scores should be asking whether they want to set that bar or clear it.
## [](#references)References
1. [Nature Medicine, “Prospective evidence for conversational medical AI is hard, but non-negotiable”](https://www.nature.com/articles/s41591-026-04639-5)
2. [arXiv, “Medical AI Benchmark Performance: o1-preview, Med-Gemini, JSL Medical-LLM 78B accuracy on MedQA-USMLE and MMLU”](https://arxiv.org/html/2410.01553v1)
3. [Starfish Medical, “FDA Action Plan for AI/ML in Software as a Medical Device (January 2021)”](https://starfishmedical.com/resource/fda-action-plan-for-ai-ml-in-samd-software-as-a-medical-device/)
4. [Bessemer Venture Partners, “State of Health AI 2026: Funding trends and AI company share of health tech investment”](https://www.bvp.com/atlas/state-of-health-ai-2026)
5. [Pertama Partners, “15 AI Project Failures and How to Avoid Them: IBM Watson for Oncology case study”](https://www.pertamapartners.com/insights/ai-project-failure-case-studies)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, Clinical AI, Digital Health Technologies, FDA Real-World Evidence, FDA Regulation
---
### [Mednet AI Intake Tool Automates EHR-to-EDC Data Entry in Clinical Trials](https://www.clinicaltrialvanguard.com/news/mednet-ai-intake-tool-automates-ehr-to-edc-data-entry-in-clinical-trials/)
**Published:** September 16, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Mednet AI intake tool automates EHR-to-EDC data entry in clinical trials, reducing manual transcription and entry errors for research coordinators.
**Content:**
Research coordinators spend 12 to 15 hours per week per study copying data that already exists in a hospital EHR into a separate EDC system. That number, cited by Mednet, points to something the EDC market’s projected growth from $3.2 billion to $7.1 billion by 2030 does not resolve on its own: a bigger market does not fix a workflow where [more than half of trial data gets entered twice](https://www.mednetsolutions.com/blog/ai-emr-intake-breaking-down-data-silos-in-clinical-research/).
The delay cost compounds that burden. Manual EHR-to-EDC transcription holds data in limbo for 14 to 30 days before sponsors and CROs see it, which means query management and oversight decisions run on information that is already weeks old. Mednet’s answer, built through its partnership with CRScube, skips the usual fix of adding another integration vendor. Instead, the AI intake feature sits inside cubeCDMS itself. A coordinator opens the patient’s EHR as usual, and the AI assistant reads what is on screen, matches it against the relevant eCRF fields, and copies the data across. No separate deployment, no trial-specific mapping project, no third-party middleware to maintain.
The design choice matters more than it might appear. Sites increasingly factor technology friction into which trials they agree to run, and sponsors that reduce administrative load gain a real recruiting edge. A [2023 meta-analysis in PubMed](https://pubmed.ncbi.nlm.nih.gov/38196643/) put the pooled error rate for manual medical record abstraction at 6.57 percent, a figure that compounds across every data point a coordinator transcribes by hand. Eliminating the transcription step attacks error at the source rather than catching it through downstream queries. That shifts the data management team’s time from correcting routine entry mistakes to reviewing data that actually needs a human judgment call.
The FDA has not issued a standalone guidance for AI-driven EDC capture, so regulatory characterization of this kind of screen-based intake remains part of the broader conversation around [AI in clinical investigations](https://www.fda.gov/media/177030/download). Sponsors evaluating cubeCDMS will need to satisfy themselves on source data verification requirements specific to their protocols. The practical test will be whether sites running studies on the platform see the transcription hours actually drop, and whether cleaner first-pass data translates to fewer query cycles before database lock.
*Source link: *
**Categories:** News
---
### [Hinge Bio Uses Single Medrio Platform for Phase I Trial Operations](https://www.clinicaltrialvanguard.com/news/hinge-bio-uses-single-medrio-platform-for-phase-i-trial-operations/)
**Published:** September 16, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Hinge Bio runs its Phase I trial on a single Medrio platform, consolidating EDC, ePRO, and RTSM to reduce setup time and site burden.
**Content:**
Hinge Bio’s Phase I program runs on three Medrio modules simultaneously: EDC, ePRO, and RTSM, a stack the biotech chose specifically to avoid the cost overruns and multi-month setup times that enterprise vendors typically require for first-in-human studies. That choice reflects a real operational trade-off facing lean biotechs: enterprise platforms carry validation infrastructure and negotiating weight, but small sponsors launching a single Phase I often spend more time onboarding the software than enrolling the first cohort.
The timing argument is concrete. [Medrio’s RTSM can be deployed in four to five weeks](https://medrio.com/solutions/rtsm/), with mid-study amendments turned around in a week or less. For a Phase I team watching a dose-escalation schedule, a one-week amendment turnaround versus a multi-month one is not a marginal improvement: it determines whether a safety update delays the next cohort or not. Hinge Bio’s clinical operations team, led by Chief Clinical Operations Officer Kristen Quigley alongside clinical data manager Sarah Huntley and clinical lead Joshua Pellam, cites accelerated timelines, predictable costs, and reduced site burden as the outcomes the setup delivered.
The site burden point deserves attention. Phase I trials run at a small number of sites, often academic medical centers with already-stretched coordinators. Consolidating data capture, patient-reported outcomes, and randomization and trial supply management into one vendor means coordinators train on one system rather than three, and the sponsor’s data team troubleshoots one contract. Whether that translates to faster query resolution or fewer screen failures at the site level, the case study does not specify, but the operational logic is sound for a single-program company without a dedicated IT department.
Context worth noting: the FDA’s [final decentralized trial guidance, issued in September 2024](https://www.appliedclinicaltrialsonline.com/view/fda-decentralized-clinical-trial-guidance), increased pressure on sponsors to document how remote data collection tools integrate with site workflows. A unified eClinical platform with a single audit trail addresses that documentation requirement more cleanly than separate vendor systems. For biotechs watching Hinge Bio’s setup, the metric to track is whether single-vendor configurations shorten the gap between protocol amendment approval and first patient enrolled under the revised version at the median site.
*Source link: *
**Categories:** News
---
### [Pulse Biosciences Closes PRECISE-BTN Thyroid Nodule Trial Enrollment](https://www.clinicaltrialvanguard.com/news/pulse-biosciences-closes-precise-btn-thyroid-nodule-trial-enrollment/)
**Published:** September 16, 2026
**Author:** Jon Napitupulu
**Excerpt:** Pulse Biosciences closes enrollment in PRECISE-BTN Thyroid Nodule trial with first patient completing follow-up, positioning for primary dataset readout within
**Content:**
Pulse Biosciences closed enrollment in its [PRECISE-BTN study](https://www.sec.gov/Archives/edgar/data/1625101/000143774926030392/plse20260827_8k.htm) on September 15, 2026, and the same filing confirmed that the trial’s first enrolled patient has already completed a final follow-up visit. Both milestones arriving together matters because they compress the window to a primary dataset: with enrollment done and the follow-up clock already running on the earliest patients, the company moves toward readout without a gap between accrual and outcome.
The study evaluates the [nPulse Vybrance Percutaneous Electrode System](https://www.stocktitan.net/sec-filings/PLSE/10-k-pulse-biosciences-inc-files-annual-report-8bf458a8f673.html), which received FDA 510(k) clearance for soft-tissue ablation in March 2024, for symptomatic benign thyroid nodules. According to verified reporting, the trial expanded from an initial 50-patient target to 100 patients, and its endpoints span nodule volume reduction, symptom relief, and quality of life. The nodule-volume endpoint carries the most regulatory weight: a meaningful, durable reduction in a blinded measurement is the kind of evidence that supports broader clinical adoption of a non-surgical ablation approach.
The competitive context for that evidence has sharpened recently. [STARMED America received FDA 510(k) clearance in May 2026](https://starmed-america.com/news-events/starmed-receives-fda-clearance-for-thyroid-rfa/) for a radiofrequency ablation device specifically indicated for benign thyroid nodules in adults, making it an active comparator in the minds of clinicians and payers even if it is not the trial’s control arm. Pulse’s nPulse technology uses nanosecond pulsed electric fields rather than thermal energy, a mechanistic difference the company argues reduces collateral tissue damage, but PRECISE-BTN’s data will need to show that distinction translates into a clinical outcome rather than a device-specification advantage.
The number to track now is nodule volume reduction at the primary timepoint. If the trial’s design calls for follow-up consistent with the first patient’s completion, a dataset is likely within reach in the next several months. That readout will determine whether Pulse can convert cleared-device status into actual procedural volume against an increasingly defined field of thyroid ablation alternatives.
*Source link: [https://www.sec.gov/Archives/edgar/data/1625101/000143774926030392/plse20260827\_8k.htm](https://www.sec.gov/Archives/edgar/data/1625101/000143774926030392/plse20260827_8k.htm)*
**Categories:** News
---
### [FDA Aligns on Rocket Pharmaceuticals RP-A501 Phase 2 Trial Design for Danon Disease](https://www.clinicaltrialvanguard.com/news/fda-aligns-on-rocket-pharmaceuticals-rp-a501-phase-2-trial-design-for-danon-disease/)
**Published:** September 16, 2026
**Author:** Jon Napitupulu
**Excerpt:** FDA aligns on RP-A501 Phase 2 trial design for Danon disease, approving a 12-patient pivotal population and 12-month endpoint structure.
**Content:**
Rocket Pharmaceuticals got something it badly needed on September 15: FDA alignment on the design of its pivotal Phase 2 trial of RP-A501 for [Danon disease](https://www.researchgate.net/publication/408191011_How_ultra-rare_is_danon_disease_an_estimate_of_prevalence_based_on_multiple_data_sources), a LAMP2-deficiency cardiomyopathy so rare that fewer than 250 patients appear in major electronic health record databases despite a broader genomic prevalence estimate approaching 12,000. The agency signed off on three specifics: a recalibrated dose, a 12-patient pivotal population, and a 12-month co-primary endpoint structure. For a program in a disease this uncommon, those numbers are the trial.
The 12-patient threshold deserves attention because it sets the minimum evidentiary bar Rocket must clear for any eventual regulatory submission. A pivotal population that small gives the agency almost no margin for attrition or protocol deviation, which means every enrolled patient’s data quality and retention carries outsized weight. Rocket [reported a positive clinical safety update from its first three patients](https://www.biopharminternational.com/view/rocket-pharmaceuticals-rp-a501-phase-2-danon-disease) under a modified protocol as recently as August 3, 2026, so the company enters this FDA-aligned phase with at least preliminary evidence that the revised approach is tolerable.
The dose recalibration is the part the 8-K does not fully explain, but it matters as much as the endpoint agreement. Earlier AAV9 gene therapy programs across the industry have been forced to revise doses after immunogenicity or liver signals surfaced post-dosing; Rocket’s modified protocol and the safety update from those first three patients suggest the recalibration was a response to real data rather than a precautionary adjustment. The filing does not say what the original dose was or by how much it changed.
With FDA now aligned on trial architecture, the practical question is how quickly Rocket can enroll nine additional patients in a disease where fewer than 250 are formally diagnosed in the United States. Enrollment pace in Danon disease is a harder constraint than regulatory strategy, and the 12-month co-primary endpoint means the clock on any submission start date does not begin until the last patient completes follow-up. Watch enrollment milestones, not regulatory meetings, as the real signal for when a data readout becomes plausible.
*Source link: [https://www.sec.gov/Archives/edgar/data/1281895/000114036126036563/ef20082010\_8k.htm](https://www.sec.gov/Archives/edgar/data/1281895/000114036126036563/ef20082010_8k.htm)*
**Categories:** News
---
### [CRB-913 Obesity Drug Shows 5% Weight Loss in Phase 1b Trial](https://www.clinicaltrialvanguard.com/news/crb-913-obesity-drug-shows-5-weight-loss-in-phase-1b-trial/)
**Published:** September 16, 2026
**Author:** Jon Napitupulu
**Excerpt:** CRB-913 obesity drug achieved 5% weight loss in Phase 1b trial, signaling potential for a peripheral CB1 mechanism without central nervous system effects.
**Content:**
A 12-week Phase 1b trial is not where obesity drugs usually make headlines, but [CRB-913](https://www.corbuspharma.com/our-pipeline/crb-913-inverse-agonist-biology-obesity-treatment) earned a late-breaking slot at ObesityWeek 2026 in Washington, D.C., after its 60 mg dose produced 5.0% mean weight loss from baseline in non-diabetic obese adults. That number matters less as an absolute than as a signal about mechanism: CRB-913 is a peripherally restricted CB1 inverse agonist, a class that was effectively abandoned a decade ago when the first-generation drug rimonabant caused psychiatric side effects by crossing into the brain. Corbus designed CRB-913 to stay out of the central nervous system, and a Phase 1b result with a clean enough safety read to get accepted for late-breaking presentation suggests that design constraint held.
The [CANYON-1 study](https://www.streetinsider.com/Corporate+News/Corbus+obesity+drug+hits+5%25+weight+loss+mark+in+phase+1b+trial/27055273.html) enrolled 254 non-diabetic adults across 15 U.S. sites and randomized them to 20 mg, 40 mg, or 60 mg of CRB-913 once daily or placebo. The 5.0% figure at the top dose after just 12 weeks is meaningful in a 1b context, where dose-response clarity matters more than the magnitude itself. An approved GLP-1 like semaglutide (Wegovy) produces roughly three times that weight loss over a longer treatment period, so CRB-913 is not competing on raw efficacy. The strategic case is different: if peripheral CB1 blockade works without the central side effects, it offers a distinct mechanism that could reach patients who do not tolerate or respond to injectable GLP-1 therapies.
The ObesityWeek abstract window closed July 22, with decision notifications sent around September 8, placing this acceptance on the same week as the 8-K. [Late-breaking presentations](https://obesityweek.org/abstract-submissions/) at ObesityWeek run November 14 through 16, which gives Corbus a public data moment before any end-of-year financing or partnering conversations. At the Phase 1b stage, the full dataset, specifically the adverse-event profile across all three doses, is what determines whether this mechanism is genuinely de-risked or whether the peripheral-restriction design leaks enough central exposure to reproduce earlier-generation problems at higher doses.
The November presentation will be the first time investigators can interrogate that safety breakdown in front of the full obesity research community, and the dose-response curve across 20, 40, and 60 mg will tell researchers whether the CB1 class has a real ceiling or room to push further in Phase 2.
*Source link: *
**Categories:** News
---
### [TRUTAKNA Meets All Endpoints in Phase 3 IgA Nephropathy Trial Final Analysis](https://www.clinicaltrialvanguard.com/news/trutakna-meets-all-endpoints-in-phase-3-iga-nephropathy-trial-final-analysis/)
**Published:** September 16, 2026
**Author:** Jon Napitupulu
**Excerpt:** TRUTAKNA meets all endpoints in Phase 3 IgA Nephropathy Trial final analysis, confirming eGFR stabilization and disease progression prevention over 104 weeks.
**Content:**
Two years of eGFR data are now the center of a regulatory conversation that Vera Therapeutics did not expect to be having this soon. On September 15, 2026, the company announced that TRUTAKNA (atacicept-vymj) met all prespecified endpoints in the [final efficacy analysis of ORIGIN 3](https://www.biospace.com/press-releases/vera-therapeutics-announces-trutakna-atacicept-vymj-stabilized-egfr-and-prevented-kidney-disease-progression-through-two-years-in-origin-3-final-efficacy-analysis-in-iga-nephropathy), the Phase 3 trial in 428 adults with primary IgA nephropathy at risk for disease progression. The drug already holds [accelerated FDA approval](https://www.consultant360.com/exclusive/fda-approves-trutakna-primary-iga-nephropathy) for proteinuria reduction, granted in July 2026 on the strength of an interim read from the same trial. What the final analysis adds is stabilized eGFR and prevention of kidney disease progression through 104 weeks, the kind of functional outcome that accelerated approvals are designed to eventually require.
That distinction matters for how this data package moves through the regulatory system. Accelerated approval is conditional: sponsors must confirm clinical benefit in a post-approval study, and the FDA can withdraw approval if that confirmation never arrives or arrives weakly. The ORIGIN 3 final analysis is effectively that confirmatory dataset, and hitting every prespecified endpoint in 428 patients over two years removes the largest near-term risk to TRUTAKNA’s commercial position. Vera filed an 8-K disclosing the results the same day and noted it updated its corporate presentation in connection with the release.
For trial-operations teams and site investigators, the two-year duration of ORIGIN 3 is worth noting. IgAN trials historically struggled to capture eGFR slope changes in shorter windows; a 104-week endpoint that holds across all prespecified measures gives regulators a cleaner signal than proteinuria alone. The interim analysis in the New England Journal of Medicine built the accelerated-approval case; this final read is what converts a provisional label into a durable one.
The immediate marker to watch is whether Vera submits a supplemental BLA or a label-update filing and how quickly FDA moves it through, since the confirmatory data package is now in hand.
*Source link: *
**Categories:** News
---
### [When the Scan Knows Before the Spirometer Does](https://www.clinicaltrialvanguard.com/executiveinterviews/when-the-scan-knows-before-the-spirometer-does/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Professor Peter George, Pulmonologist at the Royal Brompton and Senior Medical Director at Brainomix, on why quantitative CT is rewriting the evidence playbook for fibrotic lung disease, and what it will take to make that standard of care.
**Content:**
 Prof Peter George
Senior Medical Director, Brainomix
Consultant Pulmonologist and ILD Clinical Lead,
Royal Brompton Hospital *Spirometry has been the backbone of fibrotic lung disease monitoring for decades. But its 10 to 15 percent day-to-day variability, and its inability to capture early structural change, means clinicians have long been managing a condition they cannot fully see. Brainomix is making the case that AI-powered quantitative CT can fill that gap, and the evidence is accumulating fast. The company’s e-Lung platform is now FDA-cleared and CE-marked, has been validated across multiple datasets including the landmark INBUILD trial in partnership with Boehringer Ingelheim, and is about to be embedded into 20 U.S. centers through a prospective outcomes study called PROGRESS PPF. At the center of that evidence-building effort is Dr. Peter George, consultant pulmonologist at Royal Brompton Hospital and Senior Medical Director at Brainomix, who has been shaping the clinical case for e-Lung since the platform was still just a concept. We spoke with him about what the data actually shows, where the technology sits today, and what has to be true for quantitative CT to become standard of care.*
---
### [](#how-did-a-specific-patient-case-first-convince-you-that-quantitative-ct-was-capturing-something-spirometry-was-missing)How did a specific patient case first convince you that quantitative CT was capturing something spirometry was missing?
**Peter George:** I’ve been looking after patients with fibrotic lung disease for over 15 years, and the challenge we have with these conditions is that our previous gold standard tools have always been CT with visual assessment and lung function, which comprises spirometry and gas transfer measurement.
We’ve known for many years that spirometry carries variability of around 10 percent day-to-day and lab-to-lab, and gas transfer, the DLCO, is affected by about 15 percent variability. So spirometry is insensitive to small changes in disease progression, and it’s a volitional test. If patients are having a bad day, it can underestimate the true physiology.
What clinched it for me was a specific case where a patient had stable lung function tests. The forced vital capacity hadn’t changed in six months. But they were feeling more breathless, and the serial CT scans showed extensive disease which the radiologist reported as being stable. When disease is extensive on a scan, it’s very difficult for radiologists to identify progressive fibrosis. We then applied the e-Lung technology to that case, and what we found was that the quantitative CT biomarkers had in fact risen over that period, at the exact time the patient was feeling more breathless, despite the spirometry being stable.
You could ask, well, how do you know which is more accurate? When we asked our radiologists to re-evaluate the scan with the benefit of the quantitative AI-powered technology, and when they did so, they revised the report and agreed that they could see that progression. What’s special about the tool is that it’s very explainable. When the patient has a scan, the imaging biomarkers light up on the scan in different colors, and the radiologist can go back to that area of abnormality and evaluate whether they can now see the areas of fibrosis progression which correspond to the colour and can then determine whether they believe that thereis true progression. That, I think, is when it really hit me that this technology is going to make a difference for patients in the long term.
> “The quantitative CT biomarkers had in fact risen over that period, at the exact time the patient was feeling more breathless, despite the spirometry being stable.”
---
### [](#why-did-the-taladegib-ct-analysis-focus-on-lung-volume-and-fibrosis-extent-rather-than-anchor-to-fvc-as-the-primary-reference)Why did the taladegib CT analysis focus on lung volume and fibrosis extent rather than anchor to FVC as the primary reference?
**Peter George:** The original taladegib study has already been published in the Lancet Respiratory Medicine. It was a phase 2a study, so primarily a safety and tolerability study, but it did have an efficacy endpoint included within it, and that study showed that taladegib was associated with a significant reduction in FVC change over 12 weeks compared to placebo. So we already knew there was a signal with FVC.
But what FVC doesn’t tell you is about drug mechanism of action, and about different structural elements of interstitial lung disease. So we then looked at the CT scans, which were performed as part of that study at baseline and at the end of the study (week 12). What we were able to show is that in line with the deceleration of FVC decline, we could find changes in different anatomical lung compartments that represented these abnormalities. We found a significant rise in lung volume, a significant reduction in total interstitial lung disease extent, a significant reduction in fibrosis extent. So this shows us that we were able to not just identify the change that had already been published using FVC, but to identify different pathological processes that might explain that spirometric finding.
---
### [](#why-did-the-inbuild-post-hoc-analysis-structure-its-prediction-around-six-month-ct-changes-rather-than-later-timepoints)Why did the INBUILD post-hoc analysis structure its prediction around six-month CT changes rather than later timepoints?
**Peter George:** The INBUILD study was a landmark study, the first to show that an antifibrotic drug could be used in patients with non-IPF progressive pulmonary fibrosis. As part of that study, a subset of patients had a baseline CT, a CT at six months, and an end of study CT at 12 months. Brainomix, through its close relationship with Boehringer Ingelheim, had privileged access to those CT scans, and that allowed us to work collaboratively to see what further information we could extract from this pivotal study.
What’s particularly exciting is that this is one example of the growing number of collaborations we’ve built with Life Sciences companies over the years. These partnerships allow us to bring our quantitative CT technology into clinical development programmes to accelerate the delivery of new drugs to the clinic and to extract additional mechanistic insights from imaging data that might otherwise be missed.
What we found in this INBUILD post-hoc analysis was that the change in quantitative CT metrics at 24 weeks could predict FVC decline at 52 weeks. What’s important about that is it shows us that e-Lung quantitative CT biomarkers measure changes in fibrosis predicting lung function decline 6 months earlier than a trial readout. It means that we may be in a position to advance decision-making in clinical trials from 12 months to six months in some settings. It might help us in terms of thinking about go, no-go decisions in earlier phase clinical trials.
---
### [](#how-do-you-guard-against-the-possibility-that-the-model-is-fitting-to-historical-datasets-rather-than-capturing-true-disease-biology)How do you guard against the possibility that the model is fitting to historical datasets rather than capturing true disease biology?
**Peter George:** What is unique about e-Lung and the e-Lung biomarkers is that they are FDA-cleared and CE-marked. They are now locked andas a consequence, when we apply them to clinical trial datasets or to routine clinical practice, the fact that they cannot be trained against the additional data they are being tested upon guards against that possibility of overfitting. These thresholds and levels have been validated extensively across a number of publications over the past few years.
So whereas that might have been a reasonable assertion in our very early work, the fact that these biomarkers continue to be effective in identifying progressive disease and demonstrate a treatment effect in clinical trials some years on shows us the validity and the strength of their potential.
---
### [](#how-do-you-see-quantitative-ct-biomarkers-sitting-in-the-ild-diagnostic-pathway-five-years-from-now-and-what-would-have-to-be-true-for-that-to-happen)How do you see quantitative CT biomarkers sitting in the ILD diagnostic pathway five years from now, and what would have to be true for that to happen?
**Peter George:** That really comes down to implementation science and we are now in the implementation phase. We have extensive scientific validation of the tool and the biomarkers. We’ve shown they can identify progressive pulmonary fibrosis more readily than visual assessment or lung function change. We’ve shown that change in e-Lung measures of fibrosis and interstitial lung disease are predictive of mortality. And we have shown that e-Lung biomarkers predict patients at risk of future mortality and lung function decline. Brainomix has a strong track record in widespread clinical adoption through its stroke activity and this is now being replicated across interstitial lung disease networks – The tool is cleared for clinical practice in both Europe and the US.
We’ve also recently had a study accepted for publication, called the REVISE PPF study, where we were able to show that had e-Lung been embedded in any one of three centers we worked with;University of Chicago, University of Alabama, and Weill Cornell Medicine, patients would have been diagnosed with progressive pulmonary fibrosis up to two years earlier. The tool is cleared for clinical practice in both Europe and the US.
We are now embarking on an ambitious multi-center study in the US called PROGRESS PPF, where we will embed e-Lung into 20 US centers and will evaluate the clinical impact on patient outcomes. I think this will be really important work that we reference when we look to then scale the technology and move towards more widespread clinical adoption. I think we are on the cusp of a sea change in how these interstitial lung diseases are managed and the routine integration of AI-powered tools to improve diagnostic efficacy and eventually patient outcomes.
---
### [](#how-does-a-clinician-actually-navigate-the-conversation-with-an-ipf-patient-who-presents-with-both-genetic-risk-signals-and-a-high-ct-fibrosis-burden-today)How does a clinician actually navigate the conversation with an IPF patient who presents with both genetic risk signals and a high CT fibrosis burden today?
**Peter George:** What we are now doing building multi-modal predictive models. We want to gather important prognostic information from an individual patient to provide a personalized risk stratification approach for that individual.
Most centers at this stage do not routinely measure telomere lengths, and most centers at this stage do not routinely use e-Lung as a quantitative CT tool. But I could envisage in the next 5 to 10 years a situation where we are integrating e-Lung quantitative CT data with multi-omic biomarkers, genetic information and telomere lengths with the patient at the very heart to provide a much more nuanced approach to individualized treatment plans, risk stratification for outcomes and assessment of treatment efficacy. Although I don’t think we’re there yet, I do think that’s where the future may lie.
> “I could envisage in the next 5 to 10 years a situation where we are integrating e-Lung quantitative CT data with multi-omic biomarkers, genetic information and telomere lengths with the patient at the very heart.”
*Prof Peter George is a consultant pulmonologist and clinical lead for the Interstitial Lung Disease service at Royal Brompton Hospital, and Senior Medical Director at Brainomix.*
---
**Categories:** Article: Executive Interviews
---
### [FDA Hears 80 Speakers on Psychedelic Drug Therapy Standards](https://www.clinicaltrialvanguard.com/conference-coverage/fda-hears-80-speakers-on-psychedelic-drug-therapy-standards/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** The FDA held a public hearing on psychedelic drug therapy, drawing more than 1,800 registered attendees and 80 speakers across clinical, veteran, patient, and industry communities. Key debates centered on workforce credentialing, patient safety protocols, data standardization, and equitable access. The docket remains open through October 5, 2026.
**Content:**
*CONFERENCE COVERAGE | Considerations for Potential Future Therapeutic Use of Psychedelic Drugs FDA Public Hearing*
The FDA convened a public hearing on the therapeutic use of psychedelic drugs, drawing more than 1,800 registered attendees and 80 scheduled speakers. The event, held in collaboration with NIDA, SAMHSA, the Veterans Health Administration, and ARPA-H, focused on four topic areas: provider training and credentialing, patient safety, access, and data standardization. Opening remarks were delivered by Dr. Marta Sokolowska, Deputy Center Director for Substance Abuse and Behavioral Health at FDA’s Center for Drug Evaluation and Research. Newly installed CDER Director Dr. Michael Davis attended the afternoon session.
## [](#a-field-growing-faster-than-its-framework)A Field Growing Faster Than Its Framework
The hearing came at a pivotal regulatory moment. In April 2026, FDA issued national priority vouchers to three companies studying psilocybin for treatment-resistant depression, psilocybin for major depressive disorder, and methylone for PTSD. The agency also allowed an early-phase trial of noribogaine chloride for alcohol use disorder to proceed under an IND. In July 2026, FDA finalized guidance on clinical investigations for psychedelic drugs and published a commentary in the New England Journal of Medicine outlining its new regulatory framework.
FDA received more than 200 speaker requests by the August deadline, more than double the available slots. Speakers were randomly selected with attention to topic coverage and organizational diversity. The public docket remains open through October 5, 2026.
One recurring theme: the treatment gap is already generating real-world harm. Multiple speakers, including veterans and a combat-wounded nurse practitioner, described traveling outside the United States, to Mexico and other countries, to access ibogaine and ayahuasca because domestic options did not exist. One Iraq War veteran described years of underground practice to keep fellow veterans from dying by suicide. A retired Green Beret said ketamine therapy was “like a reset button” after half a dozen psychiatric medications failed him, and described nearly needing crisis intervention himself before finding treatment.
## [](#credentialing-competency-over-credentials)Credentialing: Competency Over Credentials
The most contested topic was who should be allowed in the treatment room, and what qualifications they need. Opinions ranged sharply.
A psychiatrist at Sheppard Pratt who has cared for roughly 200 participants across more than 10 psychedelic trials argued that psychiatrists should be required to lead psychedelic treatment, warning that commercial pressures could sideline psychiatric expertise behind a veneer of clinical legitimacy. By contrast, a Columbia University professor argued against novel licensing layers, contending that psychedelic treatment should stay within mainstream behavioral health scope of practice to prevent patients from being diverted to less regulated settings.
A licensed clinical social worker and practice founder pushed for explicit credentialing of preparation and integration competencies, distinct from in-session monitoring, and called for ongoing clinical consultation requirements throughout a practitioner’s career. A founder of the nation’s first accredited psilocybin facilitator training program cited a JAMA Network Open multi-site study of 346 Oregon patients in which four experienced serious adverse events; only one was identified during the session, with the other three surfacing weeks later through follow-up, an argument for longitudinal safety monitoring, not just session oversight.
Multiple speakers called for competency-based assessment rather than hourly training requirements. A simulation platform founder noted that hours are easy to satisfy and hard to verify, while demonstrated competency can be audited. Representatives from nursing, pharmacy, and occupational medicine each argued their professions were underrepresented in current guidance. The American Pharmacists Association formally requested that pharmacists be recognized as essential members of the psychedelic care team, citing existing infrastructure for controlled substance storage, inventory, and compliance.
Australia’s experience drew direct comparison. A representative of a publicly traded Australian company reported conducting more than 500 dosing sessions since October 2023 under that country’s authorized prescriber scheme, with no severe adverse reactions and with coverage from Australia’s largest private insurer and its veterans affairs department. The company treats patients excluded from clinical trials, including those with elevated blood pressure and personality disorders, a population that panelists noted represents the real-world caseload more accurately than trial enrollees.
## [](#access-geography-cost-and-the-insurance-gap)Access: Geography, Cost, and the Insurance Gap
Access concerns dominated the afternoon session. A Nevada veterans coalition leader noted that nearly 87% of Nevadans live in a federally designated mental health professional shortage area, and that the FDA’s July guidance recommendation, that a physician be reachable within 15 minutes when a non-physician serves as lead monitor, would effectively disqualify every clinic in rural and frontier Nevada. He urged the agency to allow product-specific emergency protocols combining on-site personnel, remote physician consultation, and EMS activation.
A founder of one of the country’s largest brain medicine networks, operating TMS and ketamine across 27 clinics in seven states, offered a cautionary data point: esketamine was approved in 2019, but sales were so small for the first two years that they were not separately reported. It took five years to reach scale. The science did not change, infrastructure did. The speaker made three specific requests: write labels with payers in mind so that undefined terms cannot become denial criteria; standardize adverse event capture and outcome measures before launch; and work with CMS on permanent CPT codes that reflect the full cost of a trained team in a room for a full day.
A documentary filmmaker who spent eight years filming psychedelic therapy training reported that the clinics most likely to close were not shut down by adverse events, they closed because patients could not pay. He filmed a low-income clinic in Oregon and a ketamine clinic in Lubbock, Texas, both of which failed not due to safety failures but due to reimbursement gaps, leaving patients mid-treatment with no follow-up.
Speakers also raised women’s health as an underserved area. One advocacy founder cited National Academies data showing that from 2013 to 2023, just 8.8% of NIH research grant funding went to women’s health research. A clinical pharmacist noted that menstrual cycle phase, pregnancy, postpartum status, perimenopause, and menopause all alter neurobiology and pharmacology, yet these variables are routinely excluded from psychedelic trials or treated as confounders.
Not all speakers supported accelerated access. One former ONDCP advisor cited rising hallucinogen-related emergency department visits, a Canadian study linking hallucinogen ER visits to 3.5 times greater likelihood of developing schizophrenia, and an 8-fold increase in psilocybin poison control cases among children under 12 over five years. He urged the FDA to require full scientific approval before expanding access and warned against allowing advocacy pressure to substitute for rigorous trial evidence.
## [](#data-build-the-infrastructure-now-or-pay-later)Data: Build the Infrastructure Now or Pay Later
Multiple speakers argued that the window to establish common data elements is closing, and that waiting until registries are built will make meaningful comparison impossible.
A Johns Hopkins postdoctoral researcher presented a meta-analysis from his lab, published in JAMA Psychiatry, finding that roughly 4% of psychedelic trial participants with pre-existing psychiatric conditions encountered serious adverse events even with intensive oversight and restrictive screening. He recommended anchoring initial post-approval safeguards closely to trial conditions, with pre-specified review points and defined evidentiary thresholds for relaxing or tightening requirements.
A regulatory consultant specializing in ibogaine warned that no validated cardiac prediction model currently exists for ibogaine despite documented QTc prolongation and ventricular arrhythmia at therapeutic doses. She called for a minimum common data element set, including metabolizer status, time-matched ECG, drug dose, and indication, to be published before registry infrastructure is built, arguing that a registry without common data elements produces volume, not evidence.
A UC Berkeley research physician called for a standardized glossary of psychedelic adverse events, noting that FDA guidance requires investigators to document all central nervous system effects as adverse events regardless of valence. Without common definitions, she argued, trial safety data reflects each team’s interpretation rather than the drug’s actual risk profile. Her team is developing a glossary in collaboration with UCSF and invited FDA to participate.
A Puerto Rico researcher flagged a structural gap: the National Survey on Drug Use and Health, the primary source for national psilocybin prevalence estimates, has no territorial sample frame, meaning approximately 3.2 million American citizens are excluded from the baseline data underpinning regulatory decisions. He called for territory inclusion as a foundational design requirement in any federal registry.
The FDA’s public docket for this hearing remains open at regulations.gov (Docket No. FDA-2026-N-7542) through October 5, 2026. The agency indicated that written comments, alongside testimony from the hearing, will inform continued federal coordination across FDA, VA, SAMHSA, NIDA, and ARPA-H.
**Categories:** Article: Conference Coverage
**Tags:** AI Clinical Trials, data standardization, Digital Mental Health, DOGE FDA Budget Cuts, drug regulation, ibogaine, Ketamine Therapy, Patient Safety, provider training, Psilocybin MDMA, Psychedelics, PTSD, REMS, veterans health
---
### [Compass Pathways Just Answered the Durability Question. Now Comes the Harder Problem.](https://www.clinicaltrialvanguard.com/clinical-bellwether/compass-pathways-just-answered-the-durability-question-now-comes-the-harder-problem/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Compass Pathways' 52-week COMP360 data shows 40-45% response rates in treatment-resistant depression. FDA approval in 2027 means nothing without scheduling…
**Content:**
[Lars Christian Wilde](https://www.linkedin.com/posts/lars-christian-wilde_psilocybin-nda-fda-activity-7503858685321064448-Yall) remembers the question. As a co-founder of Compass Pathways, he heard it from every investor, clinician, and regulator who looked at psilocybin’s early data: “Fine, it works acutely, but how long does it last?” That question has functioned for years as the polite version of a more aggressive skepticism, the one that kept institutional capital cautious and regulators noncommittal. [Wilde’s read of this week’s 52-week topline data from Compass’s Phase 3 COMP005 trial](https://www.linkedin.com/posts/lars-christian-wilde_psilocybin-nda-fda-activity-7503858685321064448-Yall) is direct: the question now has its clearest answer yet.
The numbers from Part C of COMP005, covering weeks 26 through 52 in 258 patients with [treatment-resistant](https://www.linkedin.com/posts/tommaso-barba-88220a177_psilocybin-depression-safety-activity-7503869850629521408-Xhdj) depression, are worth sitting with. Participants from the 25mg COMP360 arm who received one additional dose showed an average 13-point reduction in MADRS scores from baseline at week 52. Patients who started on placebo and crossed over to receive their first 25mg dose in Part C showed a 10-point average reduction. Across Part C participants, 40 to 45 percent met criteria for clinical response and approximately 30 percent reached remission. No new safety signals emerged. Per Wilde’s post, roughly 70 percent of the 258 enrolled patients continued into Part C, and 80 to 90 percent of those opted for a further dose, which is a retention and preference signal that no daily antidepressant manufacturer could replicate.
The durability objection is functionally dead.
## [](#the-infrastructure-gap-nobody-priced-in)The Infrastructure Gap Nobody Priced In
But answering the science question is not the same as solving the access question, and the field is now staring directly at the latter. [Josh Hardman of Psychedelic Alpha framed the challenge this week](https://www.linkedin.com/posts/joshhardmanuk_with-potential-fda-approval-of-multiple-psychedelic-activity-7504187667383779328-H9lF) with unusual bluntness: with potential FDA approval of multiple psychedelic therapies in the coming months and quarters, the field must now translate marketing authorization into patient access. He sketched a dual-track timeline, regulators and government stakeholders on one axis, sponsors and providers on the other, each carrying obligations that the other cannot discharge. The implication is uncomfortable. Compass could receive FDA approval for COMP360 in the first half of 2027, per the company’s stated timeline with a rolling NDA submission and final submission targeted for Q4 2026, and the drug could still be clinically unreachable for the patients it was designed to serve.
Here is the counterintuitive reality that the field keeps sidestepping: psilocybin’s most commercially attractive feature, the fact that patients need only a dose every few months rather than a daily pill, is also its biggest structural liability. A chronic daily medication plugs into existing pharmacy infrastructure, refill behaviors, and PBM contracting logic. A supervised psychedelic session requires a trained therapist, a certified clinical setting, hours of monitored time, and a billing code that most payers have never seen before. The very dosing model that makes psilocybin a “fundamentally different proposition from daily pharmacotherapy,” as Wilde describes it, also makes it invisible to the reimbursement architecture that decides whether patients can actually afford it.
Scheduling is the more immediate constraint. Psilocybin remains a Schedule I substance under federal law. FDA approval does not automatically reschedule a drug; the Drug Enforcement Administration must act, a process that historically runs six to twelve months post-approval and has no hard deadline. MDMA-assisted therapy watched this gap materialize in real time in 2024, when the FDA issued a Complete Response Letter to Lykos Therapeutics citing concerns about trial design and data interpretation, leaving a partially assembled infrastructure, trained therapists, certified sites, and waiting patients, with nowhere to direct. The psilocybin field should not assume its regulatory path will be smoother simply because its data package is stronger.
## [](#the-competition-arriving-behind-compass)The Competition Arriving Behind Compass
[Tommaso Barba at Imperial College London’s Centre for Psychedelic Research](https://www.linkedin.com/posts/tommaso-barba-88220a177_psilocybin-depression-safety-activity-7503869850629521408-Xhdj) flagged the open-label limitation directly: Part C’s final six months were not blinded, which means the long-term durability results require more cautious interpretation than the Phase 2 double-blind data that preceded them. That caveat matters for the FDA review. The agency will read the COMP005 package against COMP006, Compass’s second Phase 3 trial enrolling 581 patients, which Wilde describes as delivering a more robust confirmatory dataset. But even a clean two-trial package does not resolve the therapist supply problem. Psilocybin-assisted therapy as studied in clinical trials requires specialized training that no medical school currently offers at scale, and there is no accreditation body with the reach to certify the thousands of practitioners a functioning market would require.
[Manoj Doss, who this week announced his program’s designation as a Phase 3 clinical trial site for AbbVie’s M26-292 study evaluating bretisilocin for Major Depressive Disorder](https://www.linkedin.com/posts/manoj-doss-459b421a3_another-major-milestone-for-the-institute-activity-7503901528470999041-mUM0), represents something worth noting here: this is his team’s fifth psychedelic clinical trial and their fourth industry sponsor. AbbVie, a company with roughly $55 billion in annual revenue, is not running a small speculative bet. It is building Phase 3 infrastructure in MDD with a next-generation compound, which means the competitive window for Compass is narrower than it appears. If Compass launches in H1 2027 into a market still sorting out scheduling, therapist credentialing, and payer contracting, AbbVie’s [bretisilocin](https://www.linkedin.com/posts/manoj-doss-459b421a3_another-major-milestone-for-the-institute-activity-7503901528470999041-mUM0) could arrive into a more functional ecosystem built partly on whatever Compass and regulators figure out first.
That dynamic changes the strategic calculus for Compass in ways that pure clinical success does not capture. Being first to FDA approval in a category that lacks infrastructure is not the same as being first to market in a category that has it. Compass will spend its launch capital simultaneously proving the therapy works at population scale and teaching payers, health systems, and legislators how to accommodate a drug that looks nothing like any psychiatric treatment they have processed before.
## [](#what-has-to-move-before-2027)What Has to Move Before 2027
The 13-point MADRS reduction at one year is a clinically meaningful number. For context, a 50 percent reduction from baseline MADRS scores is the conventional threshold for “response,” and Compass’s 40 to 45 percent responder rate in an open-label continuation cohort of patients who had already failed at least two antidepressants is a signal that the FDA cannot easily dismiss. The package, combining COMP005 and COMP006 across approximately 840 patients in two Phase 3 trials, is substantive.
But [Hardman](https://www.linkedin.com/posts/joshhardmanuk_with-potential-fda-approval-of-multiple-psychedelic-activity-7504187667383779328-H9lF)‘s dual-track framework is the more urgent planning document right now. On the government track: DEA scheduling action must be initiated promptly post-approval, CMS must develop reimbursement pathways that account for session length and therapist time, and state legislatures in large Medicaid markets must act. On the sponsor and provider track: Compass must credential clinic networks before launch, training pipelines for therapists must scale faster than any continuing education infrastructure the mental health field has built before, and payer contracting must begin under conditions of genuine uncertainty about utilization. None of these steps follow automatically from a positive FDA action letter.
The patients in COMP005 waited through at least two antidepressant failures before enrolling. They should not have to wait through a scheduling backlog, a credentialing gap, and a payer education cycle on the other side of approval. Compass’s clinical team answered the scientific question. The answer to the infrastructure question belongs to a much larger, slower, and less motivated set of institutions. That is the race worth watching now.
## [](#references)References
1. [Lars Christian Wilde, LinkedIn post on COMP005 52-week data and commercial implications, 2025](https://www.linkedin.com/posts/lars-christian-wilde_psilocybin-nda-fda-activity-7503858685321064448-Yall)
2. [Tommaso Barba, LinkedIn post on Compass Pathways 52-week psilocybin results, 2025](https://www.linkedin.com/posts/tommaso-barba-88220a177_psilocybin-depression-safety-activity-7503869850629521408-Xhdj)
3. [Josh Hardman, Psychedelic Alpha, LinkedIn post on translating psychedelic therapy approval into patient access, 2025](https://www.linkedin.com/posts/joshhardmanuk_with-potential-fda-approval-of-multiple-psychedelic-activity-7504187667383779328-H9lF)
4. [Manoj Doss, LinkedIn post on Phase 3 site designation for AbbVie M26-292 bretisilocin study in MDD, 2025](https://www.linkedin.com/posts/manoj-doss-459b421a3_another-major-milestone-for-the-institute-activity-7503901528470999041-mUM0)
**Categories:** Clinical Bellwether
**Tags:** Compass Pathways, FDA Approval, Psilocybin MDMA, Psychedelic Therapy, Treatment-Resistant Depression
---
### [Biohaven Advanced BHV-7000 to Pivotal Trials Without Answering a Question the FDA Requires Every Sponsor to Answer](https://www.clinicaltrialvanguard.com/clinops-watchdog/biohaven-advanced-bhv-7000-to-pivotal-trials-without-answering-a-question-the-fda-requires-every-sponsor-to-answer/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA's Sept. 4 partial hold on Biohaven's opakalim exposes a metabolite characterization gap that ICH M3(R2) and FDA's 2021 guidance forbid sponsors from…
**Content:**
On September 4, 2026, the FDA sent Biohaven a letter that stopped new patient enrollment in every BHV-7000 epilepsy study. The agency’s stated reason: rodent studies had flagged a specific metabolite, and the FDA did not have enough preclinical data to assess what that metabolite does in humans. Biohaven disclosed the action in an [SEC filing](https://endpoints.news/fda-places-partial-hold-on-biohavens-ion-channel-drug/) the same week. The drug, opakalim, an ion channel modulator targeting Kv7, had been advancing toward what Biohaven positioned as a pivotal program in epilepsy. The partial hold froze that program mid-stride, not because of a clinical signal, not because of a site failure, but because a fundamental pharmacology question about the molecule’s own metabolic profile remained open.
That question should have been closed in the lab, not answered by the FDA in a hold letter.
## [](#what-the-rodents-told-them)What the Rodents Told Them
The sequence here matters. The FDA’s partial hold was precipitated by findings from rodent studies, studies that, by definition, were already completed and in Biohaven’s hands. The agency reviewed preclinical data, identified a metabolite of concern, and concluded the existing nonclinical package was insufficient to characterize the risk that metabolite poses in human subjects. Biohaven’s response was to [pause new enrollment](https://www.clinicaltrialsarena.com/news/biohaven-opakalim-pivotal-programme-fda-partial-clinical-hold/) and commit to generating the additional nonclinical studies the FDA requested.
The uncomfortable arithmetic: Biohaven [recorded a $93.7 million R&D expense in Q2 2022](https://www.sec.gov/Archives/edgar/data/1935979/000193597923000009/bhvn-20221231.htm) tied to the Kv7 platform acquisition that brought BHV-7000 into the portfolio, plus a $25 million milestone payment that became payable that same June. More than $118 million committed to a platform before the company had resolved whether its lead asset’s metabolite profile was characterizable under FDA standards.
The FDA’s [Safety Testing of Drug Metabolites guidance for industry](https://www.fda.gov/media/72279/download), last revised in March 2021 and aligned with ICH M3(R2), is unambiguous on timing. If a metabolite is disproportionate, meaning it is identified only in humans or appears at higher plasma concentrations in humans than in any of the animal species used in standard toxicology testing, sponsors must complete the necessary toxicity studies and provide results before advancing. The guidance defines exactly when that work needs to be done relative to clinical progression. It is not an aspirational standard. It is a precondition for continued enrollment.
Biohaven either identified this metabolite late, failed to recognize its regulatory significance when it appeared in rodent data, or made a judgment call that the characterization could happen in parallel with clinical advancement. Whatever the internal logic, the [FDA’s September 4 letter](https://allsci.com/news/regulatory/fda-clinical-hold-opakalim-partial-on-biohavens/) says that logic was wrong.
## [](#the-sponsor-defense-versus-the-regulatory-record)The Sponsor Defense Versus the Regulatory Record
Biohaven’s public posture has been measured: the hold is limited to new enrollment, patients already in the study can continue under a protocol that allows it, and the company intends to generate the additional data and seek lifting of the hold. That is a reasonable operational response. It is also precisely the kind of response that obscures how avoidable this was.
Partial clinical holds of this type, triggered by metabolite characterization gaps rather than acute clinical signals, follow a recognizable pattern in FDA enforcement. The agency does not place a hold because it believes the drug is dangerous. It places a hold because the sponsor cannot yet demonstrate the drug is safe enough to keep exposing new subjects to. The distinction sounds subtle. For a Phase III epilepsy program, it carries real operational cost: enrollment frozen, site contracts strained, timelines displaced, competitive positioning in an active therapeutic space eroded.
The FDA’s own [September 4 hold letter](https://www.investing.com/news/sec-filings/biohaven-pauses-new-enrollment-in-epilepsy-trials-after-fda-partial-clinical-hold-93CH-4895382) framed the issue plainly: insufficient information to assess the risk of a specific metabolite in human subjects, pending results from additional nonclinical studies. That language maps directly onto the 2021 metabolite safety guidance’s requirement that disproportionate metabolite characterization precede, not accompany, the clinical development stage at which human exposure becomes substantial. Biohaven crossed that threshold without closing the loop.
The deeper problem is structural. When a company acquires a platform for $93.7 million and simultaneously triggers a $25 million milestone, the internal pressure to advance that asset clinically is intense. Timelines get compressed. Regulatory readiness reviews get optimistic. The metabolite characterization work that belongs in nonclinical development gets scheduled for “as needed” rather than “before enrollment opens.” That is the decision sequence the FDA’s hold just made visible.
## [](#what-this-reveals-about-preclinical-due-diligence)What This Reveals About Preclinical Due Diligence
Biohaven is not operating outside the industry norm in the way it acquired and advanced BHV-7000. The Kv7 channel is a credible epilepsy target, opakalim has a mechanism with scientific rationale, and building toward a pivotal program in refractory epilepsy reflects reasonable commercial ambition. What the [September 4 hold](https://www.investing.com/news/sec-filings/biohaven-pauses-new-enrollment-in-epilepsy-trials-after-fda-partial-clinical-hold-93CH-4895382) exposes is a failure mode that is common precisely because it is invisible until the FDA makes it visible: sponsors treating metabolite safety work as a regulatory checkbox rather than as a scientific prerequisite.
The [ICH M3(R2) guideline](https://database.ich.org/sites/default/files/M3_R2__Guideline.pdf), which the FDA’s 2021 metabolite guidance explicitly cross-references, requires that nonclinical safety packages be adequate to support the stage of clinical development being conducted. “Adequate” has a specific meaning in that context: it includes characterization of metabolites that appear in animal studies at concentrations that may be relevant to human exposure. A rodent study that surfaces a metabolite of potential concern is not a finding to note and move past. It is a trigger for a defined regulatory workflow that must be completed before the agency will consider new enrollment justified.
The September 4 letter arrived more than four years after Biohaven paid $118 million-plus to own this program. If the metabolite appeared in rodent studies conducted during that period, the gap between when the finding was available and when the FDA formalized its concern represents the window in which this hold could have been prevented. Sponsors reviewing their own ion channel, CNS, and epilepsy programs should be asking that question about their own nonclinical packages today, not after the hold letter arrives.
For sites running BHV-7000 studies: patients currently enrolled can continue under the partial hold’s parameters, but no new subjects may enter. Site coordinators managing screening pipelines need to communicate that boundary clearly, document the regulatory basis for the enrollment pause, and avoid the common operational error of continuing to screen candidates against a protocol whose enrollment status has changed. The 8-K was filed; the sites need the operational memo to follow it the same week, not the same quarter.
For FDA-watchers: the agency’s willingness to impose a partial hold on a pivotal-stage program over a nonclinical gap signals continued enforcement rigor on metabolite characterization, a posture consistent with the 2021 guidance revision and with ICH harmonization priorities. Watch Biohaven’s IND response timeline. If the additional nonclinical studies require new animal cohorts rather than reanalysis of existing samples, the hold could run six to twelve months before the FDA has sufficient data to lift it, which would push any pivotal readout well into 2028.
## [](#references)References
1. [Endpoints News, “FDA places partial hold on Biohaven’s ion channel drug”](https://endpoints.news/fda-places-partial-hold-on-biohavens-ion-channel-drug/)
2. [Investing.com / SEC Filing, “Biohaven pauses new enrollment in epilepsy trials after FDA partial clinical hold” (September 4, 2026)](https://www.investing.com/news/sec-filings/biohaven-pauses-new-enrollment-in-epilepsy-trials-after-fda-partial-clinical-hold-93CH-4895382)
3. [Clinical Trials Arena, “Biohaven opakalim pivotal programme: FDA partial clinical hold”](https://www.clinicaltrialsarena.com/news/biohaven-opakalim-pivotal-programme-fda-partial-clinical-hold/)
4. [SEC / Biohaven Ltd., Form 10-K (2022): BHV-7000 Kv7 Platform Acquisition R&D expense and milestone disclosure](https://www.sec.gov/Archives/edgar/data/1935979/000193597923000009/bhvn-20221231.htm)
5. [FDA, “Safety Testing of Drug Metabolites: Guidance for Industry” (March 2021)](https://www.fda.gov/media/72279/download)
**Categories:** Clinops Watchdog
**Tags:** BHV-7000, Biohaven, Clinical Hold, FDA Enforcement, Preclinical Safety
---
### [The FDA Just Handed Psychedelic Drug Sponsors a Framework. Now the Hard Part Starts.](https://www.clinicaltrialvanguard.com/article/trend-watch/the-fda-just-handed-psychedelic-drug-sponsors-a-framework-now-the-hard-part-starts/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA finalized its psychedelic drug guidance July 14, 2026. COMPASS, Usona, and MAPS now face blinding failures, DEA barriers, and "set and setting" protocol…
**Content:**
Three sponsors with active psychedelic INDs opened their inboxes on July 14, 2026, and found the same document: the FDA’s finalized guidance, [“Psychedelic Drugs: Considerations for Clinical Investigations.”](https://www.fda.gov/media/169694/download) The guidance, which supersedes the [draft issued June 26, 2023](https://www.federalregister.gov/documents/2026/07/14/2026-14158/psychedelic-drugs-considerations-for-clinical-investigations-guidance-for-industry-availability), covers classic 5-HT2A agonists like psilocybin and LSD alongside entactogens like MDMA. What those sponsors found inside will force a fundamental rebuild of how psychedelic-assisted therapy trials are designed, staffed, and monitored. The clinical operations infrastructure that moved MAPS through two Phase 3 PTSD trials and earned COMPASS Pathways a Breakthrough Therapy Designation in 2018 was built largely without a finalized FDA framework to lean on. Now there is one, and the gaps between what sponsors assumed and what FDA actually requires are already visible.
The broader industry has spent years watching psychedelic trials from a safe distance, treating the field as a niche curiosity. That distance is no longer defensible.
## [](#what-the-guidance-actually-demands)What the Guidance Actually Demands
The finalized guidance requires sponsors to address what the FDA calls “set and setting” in their trial protocols, meaning the physical environment, psychological support structure, and therapist training standards must all be specified and justified to the agency’s satisfaction under an IND application. This is not a minor documentation requirement. It means that the room where a participant receives a 25mg psilocybin dose, the credentials of the two therapists present during the session, and the content of the preparatory and integration sessions surrounding it are now all elements of a clinical protocol subject to FDA review. Sponsors who treated “set and setting” as a clinical philosophy rather than a regulatory deliverable are now facing protocol amendments.
The operational consequence runs deeper than paperwork. Site qualification under this framework requires not just GCP-compliant infrastructure but physical spaces meeting specific environmental standards, trained dyadic therapy teams, and safety monitoring protocols capable of managing acute psychological reactions over multi-hour dosing sessions. Most CRO site networks have none of this. The Phase 3 trial infrastructure MAPS built for MAPP1 (90 patients across multiple sites) and MAPP2 (at least 100 participants across 13 U.S. and Israeli sites, [completing enrollment May 9, 2022](https://www.prnewswire.com/news-releases/maps-completes-enrollment-as-planned-for-the-confirmatory-phase-3-trial-of-mdma-assisted-therapy-for-ptsd-301543074.html)) was purpose-built from scratch. Replicating it at the scale a pivotal NDA submission requires is a fundamentally different logistical challenge than standing up a conventional psychiatry trial network.
Blinding may be the hardest problem the guidance does not fully solve.
Psychedelic compounds produce perceptible subjective effects that make placebo blinding in the classical sense nearly impossible. The FDA acknowledges this in the guidance and pushes sponsors to justify their blinding approach rather than mandating a specific methodology. But “justify your approach” places the evidential burden squarely on the sponsor, and FDA reviewers will weigh those justifications against the agency’s own evolving expectations. A [PubMed-published pilot trial examining ketamine blinding under propofol sedation](https://pubmed.ncbi.nlm.nih.gov/41988848/) ran only six participants precisely because constructing a credible double-blind design for a drug with strong subjective effects is methodologically expensive. At Phase 3 enrollment scales, sponsors cannot afford to treat blinding as an afterthought addressed in a pre-submission meeting. It needs to be the design anchor, not a footnote.
## [](#the-schedule-i-barrier-the-framework-doesnt-remove)The Schedule I Barrier the Framework Doesn’t Remove
The [FDA’s finalized guidance establishes what the agency wants to see inside an IND](https://www.fda.gov/drugs/types-applications/investigational-new-drug-ind-application). It does nothing to simplify the DEA registration process that every sponsor running a Schedule I psychedelic trial must navigate before a single participant receives a dose. Under [21 CFR Part 1301](https://www.assyro.com/regulatory-intelligence/us/cfr/title-21/part-1301), researchers must register with the DEA as Schedule I manufacturers or distributors, a process involving security requirements, vault-level storage standards, and chain-of-custody documentation that operates on its own timeline entirely independent of FDA review. A sponsor can clear every IND hurdle FDA sets and still be blocked from dosing because DEA registration is pending.
This dual-agency bottleneck has quietly shaped the psychedelic pipeline for years, and the new FDA framework does nothing to address it. [Usona Institute’s Breakthrough Therapy designation for psilocybin in major depressive disorder](https://www.usonainstitute.org/psilocybin), granted in 2019, has not translated into a filed NDA seven years later, partly because the regulatory and logistical complexity of Schedule I research registration compounds at every stage of development. Sponsors who treat FDA guidance as the primary obstacle and DEA registration as an administrative formality will learn the hard way which agency actually controls their trial timeline.
The counterintuitive read on the finalized FDA framework is that its existence may slow some programs rather than accelerate them. The conventional assumption is that regulatory clarity removes friction. But when that clarity reveals requirements sponsors were not previously meeting, it forces remediation before advancement. [COMPASS Pathways achieved 29.1% remission at week 3 in the 25mg arm](https://s204.q4cdn.com/927483861/files/doc_news/COMPASS-Pathways-announces-further-positive-results-from-groundbreaking-phase-IIb-trial-of-investigational-COMP360-psilocybin-therapy-XQB4T.pdf) of its [COMP360 Phase 2b trial](https://ir.compasspathways.com/News--Events-/news/news-details/2022/COMPASS-Pathways-announces-publication-of-phase-2b-study-of-COMP360-psilocybin-therapy-for-treatment-resistant-depression-in-The-New-England-Journal-of-Medicine/default.aspx), double the rate of lower-dose arms, a signal strong enough to justify Phase 3 investment. But moving that signal into a Phase 3 protocol that satisfies the July 2026 guidance on therapist credentials, session environment, safety monitoring, and blinding justification requires a protocol architecture that did not need to exist when Phase 2b was designed. The gap between a compelling efficacy signal and a compliant Phase 3 protocol is now formally defined, and it is wider than most sponsors budgeted for.
## [](#what-breaks-in-the-next-18-months)What Breaks in the Next 18 Months
For sponsors, the immediate pressure point is protocol development. Every active psychedelic IND holder needs to map its existing protocol against the finalized guidance requirements and identify gaps, particularly around therapist training standards, session monitoring documentation, and the environmental specifications for dosing rooms. This is not a regulatory affairs task alone. It requires clinical operations, site management, and safety monitoring teams to work from the same document simultaneously, something that rarely happens without explicit project governance.
For CROs, the challenge is more structural. The standard site qualification checklist built for a conventional Phase 3 psychiatry trial does not contain a line item for “dosing room specifications” or “dyadic therapist credential verification.” CROs that want to compete for psychedelic trial business need to build that infrastructure now, before sponsors start issuing RFPs for Phase 3 programs that the finalized guidance makes newly viable. The retention data offers a real incentive: a Frontiers in Psychiatry analysis found that MDMA-assisted therapy for social anxiety disorder showed lower dropout rates than SSRI comparators, a recruitment and retention profile that sponsors in competitive indication spaces would pay significantly to replicate.
For technology vendors, the guidance surfaces a specific gap that eClinical platforms have not been designed to fill. Multi-hour psychedelic dosing sessions require real-time safety monitoring documentation that existing eCOA and EDC tools were not built to support. Session transcription, therapist observation logging, and acute adverse event capture during a six-to-eight-hour psilocybin session demand purpose-built data capture workflows. The vendor that builds a compliant psychedelic session monitoring module first will own that category before the first Phase 3 NDA is filed.
The NEJM’s decision to [publish a framework analysis in its September 10, 2026 issue](https://www.nejm.org/doi/full/10.1056/NEJMsb2609286?af=R&rss=currentIssue) signals that the field has crossed from scientific curiosity into clinical mainstream. The sponsors who read the July guidance as a green light without reading the fine print on DEA registration, blinding methodology, and session infrastructure will be the ones explaining their complete response letters in 2028.
## [](#references)References
1. [New England Journal of Medicine, “FDA’s New Framework for Psychedelic Drugs,” Volume 395, Issue 10, September 10, 2026](https://www.nejm.org/doi/full/10.1056/NEJMsb2609286?af=R&rss=currentIssue)
2. [U.S. FDA, “Psychedelic Drugs: Considerations for Clinical Investigations,” finalized July 14, 2026](https://www.fda.gov/media/169694/download)
3. [COMPASS Pathways, “Phase 2b Study of COMP360 Psilocybin Therapy for Treatment-Resistant Depression,” NEJM Publication Announcement, 2022](https://ir.compasspathways.com/News--Events-/news/news-details/2022/COMPASS-Pathways-announces-publication-of-phase-2b-study-of-COMP360-psilocybin-therapy-for-treatment-resistant-depression-in-The-New-England-Journal-of-Medicine/default.aspx)
4. [Usona Institute, FDA Breakthrough Therapy Designation for Psilocybin in Major Depressive Disorder, 2019](https://www.usonainstitute.org/psilocybin)
5. [MAPS, “MAPS Completes Enrollment for Confirmatory Phase 3 Trial of MDMA-Assisted Therapy for PTSD,” PR Newswire, May 9, 2022](https://www.prnewswire.com/news-releases/maps-completes-enrollment-as-planned-for-the-confirmatory-phase-3-trial-of-mdma-assisted-therapy-for-ptsd-301543074.html)
6. [21 CFR Part 1301, DEA Registration of Manufacturers, Distributors, and Dispensers of Controlled Substances](https://www.assyro.com/regulatory-intelligence/us/cfr/title-21/part-1301)
7. [Frontiers in Psychiatry, Patient Recruitment and Retention in Psychedelic-Assisted Therapy Trials, 2023](https://www.frontiersin.org/journals/psychiatry/articles/10.3389/fpsyt.2023.1083354/full)
8. [PubMed, “Protocol and pilot results for a double-blind randomized placebo-controlled trial of ketamine under propofol sedation for chronic pain and depression”](https://pubmed.ncbi.nlm.nih.gov/41988848/)
**Categories:** Trend Watch
**Tags:** Adaptive Clinical Trial Design, FDA Guidance, Psilocybin MDMA, Psychedelic Clinical Trials
---
### [YPrime Guide: Supply Management Critical to IRT Trial Success for Biotech Sponsors](https://www.clinicaltrialvanguard.com/news/yprime-guide-supply-management-critical-to-irt-trial-success-for-biotech-sponsors/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Supply management and vendor accountability determine IRT trial success for biotech sponsors. YPrime's guide identifies critical cost exposure points and evalua
**Content:**
Supply management, not randomization, is where IRT systems win or lose a trial budget. That framing sits at the center of a new operational guide from YPrime aimed at biotech sponsors who may implement IRT only a handful of times across an organization’s entire lifecycle, leaving them without the dedicated RTSM specialists that larger pharmaceutical companies keep in-house.
The guide’s sharpest observation is about timing. The costliest IRT failures do not appear during system development; they surface during user acceptance testing or after launch, and nearly always trace back to assumptions made at requirements gathering that no one challenged. By that point, fixing them is expensive. Reviewing participant journeys and supply strategies during protocol development, before finalization, is when problems are cheapest to correct.
On clinical supply, the guide outlines where financial exposure concentrates: enrollment uncertainty, depot strategy, predictive resupply, expiry management, and emergency shipment risk. Predictive resupply and expiry-aware inventory allocation matter most for sponsors with a single expensive investigational product, because kits that sit at a low-enrolling site or expire before use translate directly into budget loss. The guide also addresses protocol amendments, which the source explicitly notes can range from a simple configuration change to full redevelopment depending on how the system was built. Sponsors are advised to confirm during vendor evaluation which change types are configurable and which require programming, and what retesting each triggers.
The distinction the guide draws between a capable IRT platform and a capable IRT partner reflects a real operational gap many lean biotech teams discover too late. Three factors the guide identifies as durable over a study’s life: whether the vendor team is invested in the trial’s success, whether they consistently deliver against protocol requirements, and how they respond when something goes wrong. Sponsors remain responsible for trial oversight even when activities are outsourced, so vendor accountability has direct regulatory consequence. The practical marker to watch during vendor evaluation is not feature breadth but amendment turnaround time, which tells you more about a vendor’s actual implementation capacity than any product demonstration will.
*Source link: [https://www.yprime.com/the-biotech-sponsors-playbook-for-irt-success/?utm\_source=rss&utm\_medium=rss&utm\_campaign=the-biotech-sponsors-playbook-for-irt-success](https://www.yprime.com/the-biotech-sponsors-playbook-for-irt-success/?utm_source=rss&utm_medium=rss&utm_campaign=the-biotech-sponsors-playbook-for-irt-success)*
**Categories:** News
---
### [Researchers Misread IRB Review Pathways, Delaying Study Startups](https://www.clinicaltrialvanguard.com/news/researchers-misread-irb-review-pathways-delaying-study-startups/)
**Published:** September 15, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Researchers commonly misread IRB review pathways, causing study delays. Understanding exemption categories and expedited eligibility prevents protocol cycling.
**Content:**
Researchers routinely misread the four IRB review pathways, and that misreading costs them time. The gap between what sponsors assume and what regulators require shows up most clearly in processing times: at UNC, [full board initial review averaged 58 days in 2025](https://research.unc.edu/human-research-ethics/submission-instructions/), versus 21 days for expedited and 12 for exempt. Submitting a protocol to the wrong pathway, or submitting one that is under-documented for full board, can push a study weeks past its target start date before the first participant is even screened.
The most persistent error is treating “minimal risk” as a synonym for “exempt.” Exemptions are not a risk threshold; they are a checklist of specific regulatory categories listed in [45 CFR 46.104](https://www.advarra.com/blog/nhsr-exempt-expedited-or-full-board-a-plain-language-guide-to-potential-review-pathways/). A benign survey that involves minors may fail an exemption category written for adults, regardless of how little risk the survey poses. Conversely, a minimal-risk study can end up requiring full board review simply because none of its procedures fit any category eligible for expedited handling. The two questions are separate, and collapsing them is what sends otherwise straightforward protocols into avoidable review cycles.
The word “expedited” creates its own confusion. It describes who reviews the study, not how fast the decision arrives. A single designated board member can act without waiting for a convened meeting, but questions or missing documentation extend the process just as they would at full board. Eligibility also requires two conditions to hold simultaneously: the study must be minimal risk, and every procedure must map to a recognized expedited category. Collecting skin cells by swab, for example, can qualify under noninvasive specimen collection; a punch biopsy cannot. One procedure mismatch shifts the entire study.
The NHSR determination adds another layer researchers frequently skip. Under HHS rules, activity that does not constitute a systematic investigation designed to generate generalizable knowledge may not be research at all, making any review category irrelevant. FDA-regulated research follows a different logic: identifiability of samples is not determinative the way it is under HHS, so a project that looks like NHSR under one framework may require full IRB consideration under the other. Getting that framework question answered before selecting a review pathway is the step most likely to prevent a protocol from cycling back to the start. The practical marker to watch is whether the protocol specifies which regulatory framework applies; if it does not, the IRB will ask, and the clock will not move until it does.
*Source link: *
**Categories:** News
---
### [Definium's DT120 ODT Meets Primary Endpoint in Phase 3 GAD Study](https://www.clinicaltrialvanguard.com/news/definiums-dt120-odt-meets-primary-endpoint-in-phase-3-gad-study/)
**Published:** September 15, 2026
**Author:** Jon Napitupulu
**Excerpt:** Definium's DT120 ODT met its primary endpoint in Phase 3 testing for generalized anxiety disorder, potentially offering an alternative to decades-old SSRI and S
**Content:**
Definium Therapeutics reported positive topline data from its Phase 3 Panorama study of DT120 ODT in generalized anxiety disorder on September 14, 2026, a result that matters because GAD pharmacotherapy has run on SSRIs and SNRIs for close to two decades with little change to the approved menu. A psychedelic-derived compound clearing a Phase 3 bar in anxiety would be a different kind of news entirely, and Definium now has that data in hand.
[DT120 ODT is a formulated version of lysergide (LSD)](https://pharmacally.com/definium-dt120-lysergide-breakthrough-therapy-mdd/) built on Catalent’s Zydis fast-dissolve tablet technology, which Definium selected to improve absorption speed and reduce gastrointestinal side effects relative to conventional oral dosing. The compound acts as a partial agonist at serotonin 2A receptors, the same pathway targeted by most psychedelic-class candidates in development. The orally disintegrating format is a manufacturing and logistics choice with real clinical implications: it standardizes dosing in a drug class where absorption variability has complicated earlier trials and complicates the supervised-session model most programs require.
The 8-K filed with the SEC discloses only that the topline data are positive; the full dataset, including the primary endpoint result and any secondary measures, is attached as Exhibit 99.1 but not reproduced in the filing body. That means the specific magnitude of symptom reduction on whatever scale Panorama used remains unconfirmed until Definium publishes or presents the complete results. The GAD clinical standard that matters most to regulators is the Hamilton Anxiety Rating Scale, and whether Panorama was powered on that measure or a related instrument will shape how the FDA reads the package.
The next concrete marker to watch is whether Definium files for a pre-NDA meeting or announces a regulatory submission timeline, which would signal how much confidence the company places in the Panorama dataset as a standalone registration-enabling study.
*Source link: [https://www.sec.gov/Archives/edgar/data/1813814/000110465926107275/tm2625365d1\_8k.htm](https://www.sec.gov/Archives/edgar/data/1813814/000110465926107275/tm2625365d1_8k.htm)*
**Categories:** News
---
### [Sionna to Test SION-451 and SION-2222 Combo in CF Phase 2a Trial](https://www.clinicaltrialvanguard.com/news/sionna-to-test-sion-451-and-sion-2222-combo-in-cf-phase-2a-trial/)
**Published:** September 15, 2026
**Author:** Jon Napitupulu
**Excerpt:** Sionna advances SION-451 and SION-2222 into Phase 2a trial to test dual-corrector approach for Trikafta-resistant CF patients.
**Content:**
Sionna Therapeutics is betting that two correctors working on different parts of the CFTR protein will accomplish what one cannot: meaningful sweat chloride reduction in patients whose disease persists despite Trikafta. The company announced plans to advance SION-451 and SION-2222 together into a Phase 2a proof-of-concept trial, a decision driven by post hoc data from the [PreciSION CF study](https://clinicaltrials.gov/study/NCT07108153) of its earlier compound, SION-719.
That data showed a mean placebo-adjusted sweat chloride reduction of up to 8.6 mmol/L in a subset of patients, attributed to SION-719’s activity at nucleotide binding domain 1 (NBD1). The result is modest on its own, but Sionna’s argument is mechanistic: SION-451 targets NBD1 stabilization while SION-2222 acts elsewhere on the corrector pathway, and the two together are designed to produce additive correction that neither achieves alone. Whether that logic holds in a prospectively designed trial is exactly what the Phase 2a will test.
The strategic context matters here. [Ivacaftor’s 2012 approval](https://www.prnewswire.com/news-releases/fda-approves-kalydeco-to-treat-rare-form-of-cystic-fibrosis-138405949.html) proved that targeting CFTR function directly could transform CF care, and Trikafta extended that benefit to the large F508del population after its 2019 approval. But a subset of patients on existing modulators still carry residual disease burden, and Sionna is positioning its NBD1 program as a potential add-on for that group rather than a replacement for the current standard. That framing shapes everything about the trial design: success means demonstrating incremental biology on top of Trikafta, not competing head-to-head with it.
The critical number to watch when Phase 2a data emerge will not be whether sweat chloride moves at all, but by how much it moves beyond what the PreciSION subset showed. Eight-point-six mmol/L from a post hoc analysis in a small group is a biological signal, not a clinical threshold. Regulators and investors will want to see whether a prospectively enrolled, dual-combination cohort can reproduce and extend that reduction consistently enough to justify later-stage investment.
*Source link: *
**Categories:** News
---
### [Ivonescimab beats pembrolizumab on survival in first-line PD-L1-positive NSCLC](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-pembrolizumab-on-survival-in-first-line-pd-l1-positive-nsclc/)
**Published:** September 15, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ivonescimab beats pembrolizumab on survival in first-line PD-L1-positive NSCLC, extending median OS by over eight months in Phase III trial.
**Content:**
Three years after the first patient enrolled in HARMONi-2, ivonescimab has done something pembrolizumab monotherapy has not been beaten at in a randomized Phase III head-to-head trial for first-line PD-L1-positive NSCLC: it extended median overall survival by more than eight months. At the [2026 World Conference on Lung Cancer](https://www.prnewswire.com/news-releases/oral-presentation-ivonescimab-versus-pembrolizumab-in-first-line-pd-l1-positive-nsclc-positive-overall-survival-results-from-harmoni-2-at-wclc-2026-302878536.html), Akeso reported that ivonescimab hit 30.8 months median OS versus 22.6 months for pembrolizumab (HR 0.73; 95% CI 0.57–0.95; P=0.009), a 27% reduction in the risk of death across 398 patients followed for a median of 36 months.
The survival gap widened over time. At two years, 57.9% of ivonescimab patients were alive versus 48.0% on pembrolizumab; at three years, 45.0% versus 33.1%. The PD-L1-high subgroup (TPS ≥50%) was the standout: median OS with ivonescimab was not yet reached, against 23.2 months for pembrolizumab (HR 0.58). That is the population where [pembrolizumab earned its original FDA approval in 2016](https://pmc.ncbi.nlm.nih.gov/articles/PMC5679831/) and where it has been hardest to displace. The squamous subgroup also showed a substantial difference, 30.5 versus 19.3 months (HR 0.65), and that matters because roughly 45% of HARMONi-2 patients had squamous disease. A large share of those had central tumors or cavitation, features that have traditionally made oncologists cautious with anti-VEGF agents; Akeso reports no apparent increase in bleeding risk in that group, though the trial was not powered to formally test that question.
The OS data follow a May 2024 interim analysis that confirmed the primary PFS endpoint: 11.14 months with ivonescimab versus 5.82 months with pembrolizumab (HR 0.51, P<0.0001). That result supported [Chinese regulatory approval in 2025](https://english.nmpa.gov.cn/2025-02/19/c_1073557.htm), though that approval covered a different indication, EGFR-mutated non-squamous NSCLC after TKI failure. The PD-L1-positive first-line setting resolved in HARMONi-2 is the global commercial prize, and Akeso is now running HARMONi-7, an international multicenter Phase III trial of ivonescimab monotherapy versus pembrolizumab in PD-L1-high patients specifically, which will be the dataset regulators outside China will require.
Ivonescimab combines PD-1 blockade with VEGF inhibition in a single bispecific antibody, a mechanism tested in combination form by IMpower150 but not previously beaten head-to-head as monotherapy at the OS level. The number to watch now is HARMONi-7 enrollment pace: that trial’s readout will determine whether a Western regulatory filing is years away or approaching.
*Source link: *
**Categories:** News
---
### [When the CRO and Sponsor Speak the Same Language, Oversight Stops Being a Negotiation](https://www.clinicaltrialvanguard.com/executiveinterviews/when-the-cro-and-sponsor-speak-the-same-language-oversight-stops-being-a-negotiation/)
**Published:** September 2, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Ed Fowler, Parexel's former Vice President of Operational Excellence, on what shared CTMS infrastructure has changed — and what sponsors running manual processes are still getting wrong.
**Content:**
*For decades, sponsor oversight of CRO-run studies has operated on a structural contradiction: the sponsor is legally responsible for the trial, but the data that would let them exercise that responsibility lived inside a system they couldn’t see. Custom integrations helped at the margins. Periodic reports papered over the gap. But the underlying asymmetry remained — and inspectors were beginning to notice. Ed Fowler, Vice President of Business Enablement and Engagement at Parexel, sat down with Vanguard at the Veeva R&D and Quality Summit in Copenhagen to explain what actually changed when Parexel adopted the Veeva Clinical Suite and what ICH E6R3’s continuous oversight requirements now mean for sponsors still running on manual processes. His answer is less about technology than about a shared operational reality — and the hours that disappear when you stop translating between two different versions of the same study.*
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
Ed Fowler
Former Vice President, Operational Excellence, Parexel**Moe: Why was timely data access so hard to solve before automated CTMS transfer?**
**Ed Fowler:** What is very difficult in this industry is getting data in a timely fashion into the hands of sponsors. Without CTMS transfer and other methods of moving that data across, sponsors are relying on reports that can go out of date very quickly — and that leads to escalations on issues that aren’t really there.
**Moe: Why does shared infrastructure solve something that custom integrations couldn't?**
**Ed Fowler:** The great thing about both the CRO and the sponsor having a shared infrastructure is that the data really looks the same in both systems. Things are instantly familiar to both sides when they're collaborating on the trial. You speak the same language — things look and sound the same when you're dealing with that data. It just makes the whole experience a lot easier when you're sharing data between the CRO and the sponsor.
**Moe: How has the shared platform changed the nature of sponsor-CRO meetings?**
**Ed Fowler:** You're always going to need some meetings to review outputs and make sure the CRO is hitting agreed KPIs and metrics. But the platform limits how many you need and informs you before you walk in. You have the information in your hands already — you can see near real-time updates as things progress on the trial. The meeting becomes a means to talk through things you weren't quite sure about. You still need some of that, but it's on the basis of accurate, near real-time data. That leads to fewer unnecessary conversations than you would have had otherwise.
> “You still need some meetings — but it's on the basis of accurate data which is near real time. That leads to less unnecessary conversations that you might have had to have otherwise.”
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
**Moe: How do the time savings actually show up — hours, headcount, decisions?**
**Ed Fowler:** We don't normally measure it in terms of hours — it's not a metric people would typically track. But if you look at how it worked before, you had manually created integrations that needed a lot of maintenance: thousands of hours to create them, to maintain them. And if you went the other route — providing reports — not only did the CRO have to produce, ship, and send them, the sponsor also had to spend time receiving them and unpacking them. And those reports weren't in the same format you'd get with CTMS transfer, where the data comes into the configured UI the sponsor uses day in, day out, and they know exactly what it means. A lot of hours went into that process. It's hard to quantify exactly how many, but it could be in the thousands.
**Moe: How does the oversight issue-tracking feature change what a sponsor can show an inspector?**
**Ed Fowler:** There's a feature in the Veeva product on CTMS transfer where the sponsor can put oversight issues directly into their CTMS, and those get sent to the CRO, who can then respond. There is a product update still coming from Veeva — we need to be able to track the CRO's responses to those oversight issues, and I believe that's coming this year, which will make it even better. But the current process is fully audited. The fact that the issue was raised, the oversight issue itself, the CRO response — all of that is audited in the system, and it allows that to be tracked and provides evidence of oversight.
**Moe: How are sponsor clinical operations teams using the time they used to spend chasing CRO data?**
**Ed Fowler:** They can focus on what's most important, which is advancing the clinical study — activating sites, making sure patients are enrolled, and making sure patients get the treatment they need. It's a win all round.
> “The most important thing is advancing the clinical study — activating sites, making sure patients are enrolled, and making sure patients get the treatment that they need.”
**Moe: Why should sponsors still on manual processes stop debating and start moving now?**
**Ed Fowler:** They should really focus on the data that's important to them. Define what oversight means, figure out what data they actually need, and then act on that data. Don't ask for everything — the picture becomes confused. Figure out what you really need for good, solid oversight, make sure that data is transferred in a timely manner, and focus on that.
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
**Moe: How does this position Parexel against other CROs competing for sponsors on the Veeva platform?**
**Ed Fowler:** We are one of the first CROs to use CTMS transfer and adopt the Veeva platform, so we have a lot of experience. We understand how to get this working and how to get it set up. For sponsors who are already on the Veeva platform, that's a very simple integration — very fast to set up, which means we can get working very quickly and start sharing that transparent data. I think that positions us as a strong CRO of choice for sponsors on the Veeva platform.
*Ed Fowler is the former Vice President, Operational Excellence, at Parexel.*
This interview is sponsored by [Veeva Systems](https://www.veeva.com/).
**Categories:** Article: Executive Interviews
---
### [The Algorithm Passed Every Benchmark. The Patients Didn't Notice.](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-algorithm-passed-every-benchmark-the-patients-didnt-notice/)
**Published:** September 8, 2026
**Author:** Moe Alsumidaie
**Excerpt:** A landmark Nature Medicine RCT found AI improved clinician decisions but not patient outcomes, exposing the fatal flaw in how trials validate AI tools.
**Content:**
Picture a senior primary care clinician in Birmingham, mid-consultation, with a generative AI clinical support tool surfacing a recommendation on her screen. The algorithm has been validated. The benchmark studies showed it outperformed clinicians on diagnostic accuracy. Every conference slide deck in 2024 and 2025 cited its performance metrics. And then the [randomized controlled trial published in *Nature Medicine* on June 26, 2026](https://www.nature.com/articles/s41591-026-04633-x) delivered its verdict: the AI improved the quality of her clinical decisions, but it did not produce a statistically significant change in short-term patient outcomes.
That gap, between a validated algorithm and a patient who feels better, is the most important unresolved problem in clinical AI today. And the trial that exposed it is one of the first of its kind to run the full experiment: not algorithm versus algorithm, but human-AI system versus standard care, measured at the point that actually matters.
## [](#the-benchmark-illusion)The Benchmark Illusion
The field has been running the wrong race for a decade. The standard AI validation playbook goes like this: train a model on retrospective data, hold out a test set, report area under the curve, publish in a high-impact journal, and declare the tool ready for clinical deployment. The assumption baked into every step is that algorithmic accuracy translates to clinical benefit. The Nature Medicine trial forces that assumption into the open and finds it wanting.
This matters beyond one trial. A [systematic review and meta-analysis of five randomized controlled trials involving 12,657 participants](https://www.mdpi.com/2076-3417/16/10/5146) found that AI-based clinical decision support systems produced only a statistically marginal improvement in diagnostic accuracy among healthcare professionals compared to standard care. Marginal. Across 12,657 participants. After years of benchmark papers claiming transformative accuracy gains.
The disconnect has a structural explanation. Algorithm performance is measured in controlled conditions against curated datasets. Real-world deployment drops that algorithm into a system already dense with noise: time-pressured clinicians, incomplete EHR data, variable workflow integration, and patients whose conditions don't map cleanly onto training distributions. A study evaluating an early warning system across [seven large hospitals in the Greater Toronto Area found significant model performance drift](https://www.eurekalert.org/news-releases/1086335) during the COVID-19 pandemic across [143,049 patient encounters](https://radaislice.com/news/proactive-learning-strategies-boost-safety-of-hospital-ai-models-study-finds?from=legacy), recoverable only through continual learning triggered by detected drift. The model that passed validation was not the model that showed up in year two of deployment.
Sponsors building AI tools into trial protocols are, in most cases, still validating the algorithm. They are not running the experiment the Nature Medicine team ran.
## [](#what-the-regulators-are-finally-asking)What the Regulators Are Finally Asking
The regulatory posture is shifting, but unevenly. The FDA's guidance infrastructure for AI in software as a medical device has been developing on its [dedicated AI/ML SaMD page](https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-software-medical-device), with the Predetermined Change Control Plan draft guidance attempting to create a framework for algorithms that evolve post-approval. On January 14, 2026, the EMA and FDA published a joint statement identifying [ten principles for good AI practice](https://www.ema.europa.eu/en/about-us/how-we-work/data-regulation-big-data-other-sources/artificial-intelligence) in the medicines lifecycle, centered on transparency, human oversight, and clinical validation beyond algorithmic performance. The document is principled. It centers on transparency, human oversight, and clinical validation beyond algorithmic performance.
That omission creates a specific regulatory vulnerability. A sponsor can integrate a generative AI clinical support tool into a decentralized trial protocol, validate it on benchmark accuracy metrics, and submit it to an IRB and a regulatory authority without ever being required to answer the question the Nature Medicine trial actually asked: does this tool improve outcomes for the patients enrolled in this study?
The counterpoint, offered by every AI vendor at every industry conference, is that outcome trials take too long and cost too much. Fair enough. But consider what the absence of that evidence costs downstream. CMS, which released [guidance in February 2024 on AI use in Medicare Advantage coverage determinations](https://www.mcdermottlaw.com/insights/cms-releases-guidance-on-coverage-criteria-utilization-management-and-use-of-ai/), has made clear that AI tools used in patient-specific decisions require clinical validation and cannot simply inherit the performance claims of their underlying models. The reimbursement pathway and the regulatory pathway are converging on the same demand: show the outcome, not just the algorithm.
## [](#redesigning-the-trial-around-the-system)Redesigning the Trial Around the System
The Nature Medicine trial's architecture holds the practical lesson. It randomized at the level of the human-AI system, not the algorithm alone. The outcome was measured where patients experience it, not where developers measure it. That design choice sounds obvious in retrospect. Almost no one does it.
Building that design into a clinical trial protocol requires changes that reach into eClinical operations immediately. If the AI tool is generating clinical recommendations that influence investigator decisions during the trial, it becomes part of the intervention, not an administrative support system. That means the tool's behavior needs to be logged, version-controlled, and auditable across the trial lifecycle. Medidata Rave Companion, introduced in 2025, enables research coordinators to auto-populate EDC forms with verified EHR data, [reportedly accelerating data entry by up to 90%](https://www.triticon.com/blogs/usa/complete-guide-edc-systems-2026). Oracle Clinical One has also introduced AI-enabled EHR interoperability features. Both are workflow tools. Neither was designed to capture the moment a clinician accepted or overrode an AI recommendation, which is precisely the data a human-AI system trial needs to generate.
The protocol gap runs deeper than data capture. If clinicians in one arm use the AI support tool and clinicians in the control arm do not, any contamination, any crossover of AI-informed judgment, undermines the comparison. Randomization at the cluster level, by site or by clinician, becomes necessary. That changes sample size calculations, IRB submissions, and statistical analysis plans. A sponsor who discovers this constraint after protocol finalization faces an amendment cycle that can add significant time to the trial timeline and may require additional regulatory engagement if the adaptive design elements touch primary endpoints.
Add the model drift problem. If the AI tool is a learning system, updated periodically post-deployment, the intervention arm patients enrolled in month one are receiving a different intervention than patients enrolled in month eighteen. No current eClinical platform has a native mechanism for flagging that the AI component of a trial's intervention has changed version mid-enrollment. The RTSM logs the randomization. The EDC captures the data. The gap between those two systems is where the integrity problem lives.
## [](#the-operational-rewrite)The Operational Rewrite
Sponsors running trials that incorporate AI clinical decision support tools need to treat those tools as interventional, not infrastructural. That distinction determines everything from IRB classification to statistical design to regulatory submission strategy. Concretely: any AI tool whose output influences investigator decisions on enrolled subjects warrants a predetermined change control plan that specifies what constitutes a version change, how version changes will be documented, and what threshold of model drift warrants a protocol amendment notification. The FDA's draft PCCP guidance addresses change control for AI tools in the SaMD context., but the regulatory logic of the EMA-FDA joint principles from January 2026 points directly there.
The pre-submission meeting request should arrive before the protocol is locked, not after. Version control documentation and the human-AI system design rationale are central considerations for pre-submission alignment. There is currently no standard regulatory template for assessing whether an AI-assisted endpoint adjudication tool introduces systematic bias across a trial's timeline. Giving the reviewer that framework before the IND goes in is the difference between a clinical hold and a clean study initiation.
On the outcome measurement side, the Nature Medicine trial's design implies a standard the field has not yet codified: patient-relevant outcomes, not surrogate algorithmic endpoints, measured at a scale that can detect the gap between decision quality and outcome quality. A [meta-analysis of five RCTs with 12,657 participants](https://midus.wisc.edu/findings/pdfs/2865.pdf) found only marginal benefit on diagnostic accuracy. That is a signal about sample size requirements, not a verdict on AI's potential. Trials built to detect a 15% improvement in algorithmic concordance will be underpowered to detect a 3% improvement in patient outcomes, and the 3% is the number that matters to a payer, a regulator reviewing a label expansion, and, most consequentially, the patient in the exam room.
The Birmingham clinician with the AI recommendation on her screen made better decisions. The trial measured that clearly. What it could not confirm is whether those better decisions arrived in time, were acted upon fully, were communicated to patients effectively, or were followed by the downstream care steps that translate decision quality into health outcomes. That chain, from algorithm to action to outcome, is the trial design frontier. The Nature Medicine team mapped where the frontier is. Every sponsor deploying AI in a clinical trial now has to decide whether they are building on the right side of it.
## [](#references)References
1. [Nature Medicine, "From algorithms to patient outcomes: lessons from one of the first randomized trials of AI in medicine"](https://www.nature.com/articles/s41591-026-04633-x)
2. [University of Birmingham, "AI clinical support tool improved clinician decisions in real-world primary care trial"](https://www.birmingham.ac.uk/news/2026/ai-clinical-support-tool-improved-clinician-decisions-in-real-world-primary-care-trial)
3. [Applied Sciences (MDPI), Systematic review and meta-analysis of AI-CDSS RCTs, 12,657 participants](https://www.mdpi.com/2076-3417/16/10/5146)
4. [EurekAlert, Algorithm drift study across seven Greater Toronto Area hospitals, 143,049 patient encounters](https://www.eurekalert.org/news-releases/1086335)
5. [FDA, Artificial Intelligence in Software as a Medical Device (AI/ML SaMD)](https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-software-medical-device)
6. [EMA/FDA, Ten Principles for Good AI Practice in the Medicines Lifecycle (January 14, 2026)](https://www.ema.europa.eu/en/about-us/how-we-work/data-regulation-big-data-other-sources/artificial-intelligence)
7. [McDermott Will & Emery, CMS guidance on AI use in Medicare Advantage coverage criteria (February 2024)](https://www.mcdermottlaw.com/insights/cms-releases-guidance-on-coverage-criteria-utilization-management-and-use-of-ai/)
8. [Triticon, Complete Guide to EDC Systems 2026, including Medidata Rave Companion and Oracle Clinical One AI features](https://www.triticon.com/blogs/usa/complete-guide-edc-systems-2026)
**Categories:** Article: Deep Dive
**Tags:** Adaptive Clinical Trial Design, AI in Clinical Trials, Digital Health Technologies, FDA Guidance, FDA Real-World Evidence
---
### [Novartis Pays $50M Milestone as MRT-6160 Enters Phase 2 for Sjögren's Disease](https://www.clinicaltrialvanguard.com/news/novartis-pays-50m-milestone-as-mrt-6160-enters-phase-2-for-sjogrens-disease/)
**Published:** September 9, 2026
**Author:** Jon Napitupulu
**Excerpt:** Novartis triggers $50M milestone payment as MRT-6160 enters Phase 2 for Sjögren's disease, advancing VAV1 degrader development.
**Content:**
A $50 million check is the clearest signal yet that Novartis believes VAV1 degradation works in humans. Monte Rosa Therapeutics triggered that milestone payment on September 8, 2026, when Novartis enrolled the first patient in a [global Phase 2 study of MRT-6160 (DDY391)](https://www.biospace.com/press-releases/monte-rosa-therapeutics-announces-milestone-achievement-associated-with-initiation-of-phase-2-clinical-study-for-vav1-directed-molecular-glue-degrader-mrt-6160-ddy391) in Sjögren’s disease. The molecular glue degrader targets VAV1, a signaling protein involved in lymphocyte activation, and its move into Phase 2 means the program has cleared Phase 1 safety without stopping.
The payment sits inside a much larger bet. Novartis [licensed VAV1 molecular glue degraders exclusively from Monte Rosa in late 2024 for $150 million upfront](https://ir.monterosatx.com/static-files/9232e52d-efce-492f-a340-d48772968263), with up to $2.1 billion in development, regulatory, and sales milestones attached. The $50 million now landing is the first of those downstream payments to convert, which means Monte Rosa’s balance sheet gets a meaningful cash injection while Novartis, not Monte Rosa, runs and funds the trial from here.
Sjögren’s disease is a reasonable place to test an immunology-focused degrader. The condition drives chronic inflammation through autoreactive B and T cells, and VAV1 sits upstream in the signaling cascade that keeps those cells active. Approved options for Sjögren’s remain limited, with disease-modifying candidates from multiple companies still in mid-to-late development. Novartis conducting this study globally raises the enrollment ceiling and positions MRT-6160 for a broad regulatory path if Phase 2 reads out favorably.
For trial watchers, the number to track next is not the milestone but the Phase 2 design itself: what endpoints Novartis selected, whether the primary measure is symptom-based or biomarker-driven, and how long the treatment period runs. Degrader programs live or die on proof of target engagement in humans, and a Phase 2 in an autoimmune setting will generate that pharmacodynamic data whether or not it hits clinical endpoints. That readout, whenever it comes, will tell the field far more than this week's enrollment news.
*Source link: *
**Categories:** News
---
### [The FDA Alignment Playbook Behind Ocular Therapeutix's Single-Trial NDA](https://www.clinicaltrialvanguard.com/executiveinterviews/the-fda-alignment-playbook-behind-ocular-therapeutixs-single-trial-nda/)
**Published:** August 28, 2026
**Author:** Moe Alsumidaie
**Excerpt:** How Ocular engineered a retinal drug program backward from approval, and what that means for every sponsor still designing trials the old way.
**Content:**
 Peter K. Kaiser
Ocular Therapeutix *The clinical trials industry talks constantly about regulatory strategy. Ocular Therapeutix actually built one from the ground up. The company’s lead asset, AXPAXLI™ (OTX-TKI), an investigational sustained-release tyrosine kinase inhibitor for wet age-related macular degeneration, is now the subject of an NDA supported by a single registrational trial, the SOL-1 study, conducted under a Special Protocol Assessment. At week 52, 65.9% of AXPAXLI patients maintained visual acuity compared to 44.2% for aflibercept. The treatment gap did not narrow over time. It widened. Peter K. Kaiser, MD, Chief Development Officer at Ocular Therapeutix, sat down with Vanguard to explain how FDA alignment became the architectural foundation of that program, not its finishing touch, and what sponsors designing retinal programs today would need to replicate it.*
---
**Moe: Why did FDA alignment have to come first in the development program, before the trial design was finalized?**
**Peter K. Kaiser:** If you think about getting a drug approved in the United States, you really need to do it backwards from the way most people approach it. You need to be aligned with the FDA very early in your development program, because that’s how you avoid surprises when you get a trial result. If you decide to run the study without that alignment, you could be in for many surprises along the road. There are many drugs that do something we think would be very beneficial, but there is no way to get the FDA to say that this drug actually works. We are anchored by the agency’s guidelines, and the earlier you accept that, the better your program is designed.
---
**Moe: How does the FDA’s Division of Ophthalmology’s unusual leadership stability change the risk calculus for retinal sponsors?**
**Peter:** You're absolutely right that in many areas of the agency, the leadership is changing consistently, and the guidelines along with it. It is a moving target. In the Ophthalmic Division, however, we've been very lucky. There has been almost 35 years of consistency in leadership, first with Wiley Chambers and now with Bill Boyd, who worked with Wiley for many years. Because of that, the consistency of the rulings and guidelines coming out of the ophthalmics division is very different from other fields. It's almost like the Supreme Court: if Wiley handed down something 20 years ago, that's the way it works now.
That doesn't mean they don't change their thinking. A good example is optical coherence tomography. The FDA previously said you couldn't use it as a primary outcome, but we did get a drug approved using OCT as a primary outcome. That drug was called Jetrea (ocriplasmin). As long as you can give them the background and the evidence of why a primary outcome would work, they are flexible. Another example is using the ellipsoid zone for geographic atrophy studies, which was previously all based on fundus autofluorescence. The consistency is there, but they are also willing to listen to industry and move the field forward, as long as we can prove and validate the outcome measures we're using.
---
> “There has been almost 35 years of consistency in leadership, first with Wiley Chambers and now with Bill Boyd, who worked with Wiley for many years.”
**Moe: Why is the Special Protocol Assessment (SPA) so rarely used if it offers that kind of regulatory certainty?**
**Peter:** Anybody can go to the FDA and get a Special Protocol Assessment. What it is, essentially, is an agreement, a contract that you are making with the agency. This is how we are going to run the clinical program, these are the patients we are going to enroll, and this, most importantly, is the statistical analysis plan we are going to follow and not change. The FDA's part of the agreement is that they say they agree with the study design and they agree that if it is positive, it shows what you said you were going to show.
Why don't more sponsors do it? Because a Special Protocol Assessment comes with its own rules. You cannot change it. If midway through the study you realize you need to make amendments, which is completely normal in a clinical study, it becomes very, very difficult to do that under a SPA. You really need to be almost perfect in your trial design upfront, because you cannot easily change it once you're running.
“A special protocol assessment is a formal agreement with the FDA that you are aligned regarding study design, endpoints, and statistical analysis. It doesn't mean it's an automatic approval, but approval is more likely if you have a positive clinical study behind it.”
---
**Moe: Why did the treatment difference between AXPAXLI and aflibercept grow larger from week 36 to week 52, rather than narrowing?**
**Peter:** Let me back up a little bit. In the SOL-1 clinical program, we took patients with wet age-related macular degeneration and treated them with either AXPAXLI or aflibercept, better known as Eylea, which is the gold standard control in clinical studies. The difference between aflibercept and AXPAXLI is that aflibercept works in the extracellular space and does not block all VEGF isoforms. AXPAXLI does two things: it blocks all VEGF isoforms because it blocks all three receptors, as well as other receptors that are important in macular degeneration.
What that widening result tells me is that the patients receiving AXPAXLI had a sustained release of the drug out to 52 weeks, whereas the effect of aflibercept was waning. So the delta increased with time. You would expect that if you continued to follow patients even longer without treatment, that effect would increase further. The whole rationale for AXPAXLI is to reduce the treatment burden in patients with wet age-related macular degeneration, by potentially giving them one injection per year, which would be incredible compared to what we currently do, which is injections every one to three months.
---
**Moe: How did the SOL-R trial's role shift from second registrational study to confirmatory safety support, and what specifically changed in your conversations with the FDA?**
**Peter:** Once we realized how positive the SOL-1 clinical study was, and the extent of confirmatory evidence we had to support it as a single study submission, we went to the FDA. We had what's called a Type C meeting, where we sit down, present our case, and they say yes or no to filing with a single study. We met with Bill Boyd and his team, and I do a lot of FDA meetings. It was genuinely nice to do it in person, because you can see the room's reaction to what you're presenting and you get real-time questions.
Once we knew we could file with a single study, we looked at SOL-R differently. The control group in SOL-R includes the two most common treatments for wet age-related macular degeneration right now: Eylea 2 milligrams and Eylea HD. We realized we could use this for our competitive advantage in the commercial marketplace. Physicians want to know how your drug performs against the current standards of care. When you are looking at superiority, the longer you wait to read out the primary outcome, the more likely you are to hit it. So, we moved that readout in SOL-R from around 52 weeks to two years. That gives us the best chance of demonstrating superiority to Eylea HD, which by the time AXPAXLI may be approved could well be one of the gold standard treatments. When we didn't need SOL-R for registration, we repositioned it for competitive advantage.
---
**Moe: What does a sponsor designing a retinal program from scratch today actually need in place to earn that same kind of FDA alignment?**
**Peter:** The use of a single trial submission is not new. We didn't invent this. If you look at FDA approvals in 2025, roughly half were based on a single trial. But if you are going to pursue a single trial approval, you need to be very careful not to commit to that at the outset without the appropriate safeguards.
If you go in saying your single trial will carry the approval and you later realize you did not meet the significance level or all the requirements, you would have to start a second program. That is why we had two programs running from the beginning. We were very optimistic that we would achieve a very high level of statistical significance, which is what a single trial approval requires. If you are going to design a program with only one study, you need to ensure your statistical power is correct, you need total alignment with the FDA, and you should probably secure a Special Protocol Assessment to confirm that.
Thus, a sham controlled study may not be submitted as a single trial, and two would be needed.
You also need to meet the safety requirements. We are using SOL-1 for both efficacy and safety, but we are supplementing that with safety data from SOL-R. So even though we are filing with one study, we are using confirmatory data from other sources. That is an important distinction.
“About 40% of patients stop their treatment within the first year, mainly just from getting tired of the injections. AXPAXLI was specifically designed to correct that problem.”
---
> “The control group in SOL-R includes the two most common treatments for wet age-related macular degeneration right now: Eylea 2 milligrams and Eylea HD.”
**Moe: Why does the two-thirds rescue-free rate at week 52 matter for actual patients, not just the clinical endpoint?**
**Peter:** Wet age-related macular degeneration is a leading cause of blindness in older patients, and the anti-VEGF therapies have completely changed that. The incidence of legal blindness has been dramatically decreased. But that requires very frequent and very constant injections, which patients and their caregivers sometimes get tired of. About 40% of patients stop their treatment within the first year, mainly just from getting tired of the injections. That leads to long-term loss of visual acuity over time.
AXPAXLI was specifically designed to address this problem. It was designed to provide long-acting, sustained release of a Tyrosine Kinase Inhibitor (TKI). The results you mentioned, about two-thirds of patients going almost one year without needing another treatment, mean that instead of injections every one to three months, patients could potentially go nine to 12 months between treatments. That is much easier on a patient and much easier on a caregiver, and hopefully it will mean preservation of vision over the long term.
I will say that is an aspirational statement. We hope to prove it. We currently have a third study ongoing called the SOL-X study, in which we are following all patients from both SOL-1 and SOL-R out to five years, to demonstrate long-term stability in vision. That is our goal with that study.
*Peter K. Kaiser, MD, is Chief Development Officer at Ocular Therapeutix.*
---
**Categories:** Article: Executive Interviews
---
### [AI in Clinical Trials Doesn't Have a Capability Problem. It Has a Honesty Problem.](https://www.clinicaltrialvanguard.com/clinical-bellwether/ai-in-clinical-trials-doesnt-have-a-capability-problem-it-has-a-honesty-problem/)
**Published:** September 8, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Six AI models picked the same "random" number. Clinical ops leaders are finally asking what that reveals about every AI-assisted trial decision underneath.
**Content:**
[Mohanish Anand](https://www.linkedin.com/posts/mohanishanand_what-the-models-cant-see-activity-7501784548624502784-Qom4), TA Head for Global Trial Management, [ran a simple test](https://www.linkedin.com/posts/mohanishanand_what-the-models-cant-see-activity-7501784548624502784-Qom4): he asked ChatGPT, Claude, Gemini, Perplexity, DeepSeek, and Copilot to pick a random number between 1 and 30. Every single one said 17. When he pressed each model to explain itself, some claimed the result came from a genuinely random process. Others admitted 17 just “looks” random. The explanations contradicted each other. The number never changed. What Anand captured in that single experiment is the central failure mode of AI in clinical development: a system that produces confident, plausible, internally consistent output, trained on the patterns of what has already happened, presented to users as if it were reasoning about what is happening now.
The stakes attached to that failure mode are not abstract. Clinical trials depend on decisions that contain something no historical pattern can fully encode: a site population behaving differently than expected, a protocol burden that reads fine on paper and feels punishing to the patient living it, a standard of care that shifted after the last comparable study closed. Per Anand’s post, “AI tells us what usually happens. Human judgment asks: What is different this time?” That question does not get asked when the workflow is designed to rubber-stamp AI output at speed.
## [](#the-button-that-costs-a-fortune)The Button That Costs a Fortune
[Doug Bain](https://www.linkedin.com/posts/wdouglasbain_can-we-stop-pretending-that-putting-a-human-in-the-loop-activity-7501619838738427905-xvht), a consulting partner focused on trial technology, [put it with surgical directness](https://www.linkedin.com/posts/wdouglasbain_can-we-stop-pretending-that-putting-a-human-in-the-loop-activity-7501619838738427905-xvht): “If your AI makes 10,000 decisions and a human clicks Approve 10,000 times, you haven’t necessarily created a control. You may just have created a very expensive button.” The human-in-the-loop construct has become the industry’s primary answer to AI governance anxiety. Regulators ask for it. Sponsors build it into SOPs. Ethics review committees accept it as sufficient. But the behavioral reality of high-volume AI-assisted review is that humans rationalize rather than evaluate when the cadence is fast enough and the outputs look coherent enough. The 17 problem, scaled to 10,000 protocol deviation flags or 10,000 site risk scores, becomes invisible precisely because each individual approval feels reasonable.
This is the counterintuitive truth the field keeps sidestepping. The assumption embedded in most AI governance frameworks is that more human touchpoints produce more human judgment. The evidence from cognitive science runs the opposite direction: high-volume, low-variance approval workflows produce automation bias, the documented tendency of people to accept machine output without independent verification when the machine rarely appears to be wrong. A system designed to catch AI errors can, under the wrong conditions, systematically suppress the human capacity to find them.
[Rudy Malle](https://www.linkedin.com/posts/rudymalle_clinicalresearch-clinicaloperations-clinicaltrials-activity-7502740704494874624-oJW6), who trains and places clinical research professionals, [identifies the specific capabilities](https://www.linkedin.com/posts/rudymalle_clinicalresearch-clinicaloperations-clinicaltrials-activity-7502740704494874624-oJW6) that AI cannot independently own: patient safety, data integrity, risk interpretation, site coaching, exception management, and regulatory accountability. Every item on that list requires exactly the kind of judgment that automation bias erodes. The workflow design question that sponsors and CROs have not yet answered honestly is: at what approval volume does the human-in-the-loop stop being a safeguard and start being a liability shield?
## [](#pattern-recognition-is-not-clinical-reasoning)Pattern Recognition Is Not Clinical Reasoning
[Serhii Vakal](https://www.linkedin.com/posts/serhii-vakal_artificialintelligence-drugdiscovery-techbio-activity-7500935111459352576-YyTi), Computational Drug Discovery Architect at Orion Pharma, [draws the line precisely](https://www.linkedin.com/posts/serhii-vakal_artificialintelligence-drugdiscovery-techbio-activity-7500935111459352576-YyTi) where the engineering analogy for AI breaks down: every AI-generated molecule, every predicted property, every computationally guided experiment must still "confront the complexity of living biology, laboratory validation, and clinical trials." That confrontation is not a remaining inefficiency to be optimized away. It is the substance of drug development. Biology does not behave like a dataset. Patients are not rows.
[Morgan Levine](https://www.linkedin.com/posts/morgan-levine-aa957463_a-lot-of-people-are-rightly-pointing-out-activity-7502531811969871873-51dZ), building at a stealth startup, [pushes the critique deeper](https://www.linkedin.com/posts/morgan-levine-aa957463_a-lot-of-people-are-rightly-pointing-out-activity-7502531811969871873-51dZ) than execution. Even if AI solved every operational bottleneck in the find-a-target, develop-a-drug, run-a-trial pipeline, the underlying paradigm would still be insufficient for most major chronic diseases. The pipeline architecture assumes a single actionable target. Aging does not have one. Most complex inflammatory and neurodegenerative diseases do not have one. AI applied to an epistemically flawed model of disease accelerates the production of answers to the wrong question. Per Levine's post, "we continue plowing ahead with the therapeutic pipeline built around finding a target and developing a drug against it. That will never be enough."
[Jon Bondebjerg](https://www.linkedin.com/posts/jon-bondebjerg-41340b_using-ai-to-guide-clinical-program-planning-activity-7502409932202303488-M0MC), a clinical drug development leader, [offers the most operationally grounded account](https://www.linkedin.com/posts/jon-bondebjerg-41340b_using-ai-to-guide-clinical-program-planning-activity-7502409932202303488-M0MC) of what useful AI actually looks like in practice. He used AI tools to build and stress-test a scenario-planning model for a clinical development program, testing which study sequence preserved capital and protected timelines when both were constrained. The useful output was not the first model. It was the challenge process: tracing every assumption, benchmarking every number, verifying that formula logic matched scenario labels. That process surfaced errors that "looked convincing on the surface." His conclusion reads like a corrective for the entire field: "AI is most useful as a demanding analytical partner, not as an answer machine."
Subhajit Sengupta, Associate Director of Data Science at Cytel, [points to the specific technical conditions](https://www.linkedin.com/posts/subhajit-sengupta-ph-d-456a7710_clinical-trial-teams-are-being-asked-for-activity-7500941293854801921-vUUE) under which AI earns trust in [statistical](https://www.linkedin.com/posts/subhajit-sengupta-ph-d-456a7710_clinical-trial-teams-are-being-asked-for-activity-7500941293854801921-vUUE) and adaptive design workflows: validated knowledge bases, transparent context engineering, and formal evaluation criteria before output reaches a decision-maker. These are not exotic requirements. They are the minimum conditions for any analytical tool used in GCP-regulated environments. The gap between that standard and what most organizations are actually deploying is where risk accumulates quietly.
## [](#whose-evidence-is-the-ai-summarizing)Whose Evidence Is the AI Summarizing?
The governance problem compounds when AI operates closer to the patient. Eden Brownell, a behavioral scientist focused on human-AI interaction, [examines the OpenAI-Epic integration](https://www.linkedin.com/posts/edenbrownell_openai-is-now-connected-to-epic-the-chart-activity-7501321192050659329-3TtB) with a precision that most breathless coverage of that announcement skipped entirely. Giving an AI read access to a patient's electronic health record does not update the model on current medicine. The training data still encodes publication bias, [underrepresentation](https://www.linkedin.com/posts/edenbrownell_openai-is-now-connected-to-epic-the-chart-activity-7501321192050659329-3TtB) in clinical trials, and the documented patterns of who received less care despite equivalent need. Real-world data embedded in EHR systems carries the history of healthcare inequity alongside the clinical signal. Per Brownell's post: "Published research can exclude people from the evidence. Real-world data can encode how the system failed them. And AI can compress both into an answer that sounds complete."
For sponsors running decentralized or hybrid trials who are integrating AI-assisted site selection, recruitment screening, or eCOA interpretation, this is not a distant philosophical concern. It is a protocol design question. If the model's training data systematically underrepresents the populations a trial is designed to enroll, AI-assisted recruitment tools will replicate and potentially amplify existing diversity gaps while producing outputs that read as optimized and evidence-based. The FDA's 2020 Action Plan for the Diversity of Participants in Clinical Trials and the subsequent guidance on diversifying clinical trial populations placed the burden of diversity on sponsors. AI does not redistribute that burden. It can quietly reinforce the structural conditions that created it.
What the convergence of these voices describes is not a technology that has failed to mature. It is an industry that has not yet built the institutional discipline to use the technology it already has without mistaking pattern recognition for clinical judgment, or approval workflows for oversight. Bondebjerg found real value in AI-assisted scenario planning, but only because he treated every model output as a hypothesis to be contested, not a recommendation to be accepted. Anand's six models all said 17. The question every trial operations leader needs to answer before their next AI implementation is not whether their vendor's system is smarter than ChatGPT. It is whether the humans in their loop are actually asking why.
## [](#references)References
1. [Mohanish Anand, "What the models can't see," LinkedIn](https://www.linkedin.com/posts/mohanishanand_what-the-models-cant-see-activity-7501784548624502784-Qom4)
2. [Doug Bain, "Can we stop pretending that putting a human-in-the-loop with AI makes it safe?" LinkedIn](https://www.linkedin.com/posts/wdouglasbain_can-we-stop-pretending-that-putting-a-human-in-the-loop-activity-7501619838738427905-xvht)
3. [Rudy Malle, "AI is not eliminating clinical operations," LinkedIn](https://www.linkedin.com/posts/rudymalle_clinicalresearch-clinicaloperations-clinicaltrials-activity-7502740704494874624-oJW6)
4. [Serhii Vakal, "Again and again, we hear bold claims from Big Tech," LinkedIn](https://www.linkedin.com/posts/serhii-vakal_artificialintelligence-drugdiscovery-techbio-activity-7500935111459352576-YyTi)
5. [Morgan Levine, "A lot of people are rightly pointing out," LinkedIn](https://www.linkedin.com/posts/morgan-levine-aa957463_a-lot-of-people-are-rightly-pointing-out-activity-7502531811969871873-51dZ)
6. [Jon Bondebjerg, "Using AI to guide clinical program planning," LinkedIn](https://www.linkedin.com/posts/jon-bondebjerg-41340b_using-ai-to-guide-clinical-program-planning-activity-7502409932202303488-M0MC)
7. [Subhajit Sengupta, "Clinical trial teams are being asked for more statistical rigor," LinkedIn](https://www.linkedin.com/posts/subhajit-sengupta-ph-d-456a7710_clinical-trial-teams-are-being-asked-for-activity-7500941293854801921-vUUE)
8. [Eden Brownell, "OpenAI is now connected to Epic," LinkedIn](https://www.linkedin.com/posts/edenbrownell_openai-is-now-connected-to-epic-the-chart-activity-7501321192050659329-3TtB)
**Categories:** Clinical Bellwether
**Tags:** AI Drug Discovery, Artificial Intelligence in Clinical Trials, Clinical Operations
---
### [When the ER Is Not an Option: Milestone's CMO on Bringing AFib Rate Control Home](https://www.clinicaltrialvanguard.com/executiveinterviews/when-the-er-is-not-an-option-milestones-cmo-on-bringing-afib-rate-control-home/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** The FDA approval of etripamil for PSVT occurred in December, 2025. Investigation about a potential role of etripamil in highly symptomatic AFib, The ReVeRA-Phase 3, trial is another test: can this self-administered nasal spray put an all-too-frequent emergency department problem back in the patient'
**Content:**
*The FDA approval of etripamil for PSVT occurred in December, 2025. Investigation about a potential role of etripamil in highly symptomatic AFib, The ReVeRA-Phase 3, trial is another test: can this self-administered nasal spray put an all-too-frequent emergency department problem back in the patient’s hands?*
---
Atrial fibrillation with rapid ventricular rate (or AFib-RVR) is one of cardiology’s most stubborn, logistical problems. The condition is episodic, unpredictable, and intensely symptomatic, yet the standard health care provider response has been to give patients a chronic oral medication designed for a problem that arrives in bursts, or to tell them to call 911 or seek urgent care. Milestone Pharmaceuticals is betting that etripamil, a patient-administered nasal spray approved under the brand name CARDAMYST for paroxysmal supraventricular tachycardia, can change that calculus. The company’s first patient was recently enrolled in ReVeRA 301, a Phase 3 home-use trial targeting AFib-RVR. Dr. David Bharucha, MD, PhD, FACC, Milestone’s Chief Medical Officer, and a cardiac electrophysiologist with two decades of R&D experience spanning Forest, Allergan, and AbbVie, sat down with Vanguard to walk through the science and clinician rationale behind the bet, what the Phase 2 AFib study actually showed, and what a successful Phase 3 study would mean for AFib patients who have spent years planning their lives around the nearest emergency room or urgent-care facility.
### [](#why-have-oral-beta-blockers-and-calcium-channel-blockers-failed-as-a-real-solution-for-acute-afib-rvr-episodes)Why have oral beta-blockers and calcium channel blockers failed as a real solution for acute AFib-RVR episodes?
**Dr. David Bharucha:** AFib-RVR is episodic, and taking a medication chronically, seven days a week, 365 days a year, is not going to give patients the bolus of drug effect they need in the moment. Even when given on an as-needed basis, what’s sometimes called pill-in-pocket, oral drugs simply don’t achieve the rapidity or the exposure level needed for adequate rate control. Physicians have been prescribing oral drugs because, currently, they’ve really had no other options.
What makes etripamil particularly novel, and makes it useful as a nasal spray for acute at-home administration and for our AFib Phase 3 trial, is that if you look at the published PK/PD curves for nasal etripamil, you would think you're looking at an IV-administered drug. What you're getting with this proprietary nasal spray formulation is IV-like action delivered through self-administration outside of medical supervision.
---
### [](#how-does-the-current-phase-3-revera-301-capture-reliable-ventricular-rate-data-when-patients-are-self-administering-at-home-without-supervision)How does the current Phase 3 ReVeRA-301 capture reliable ventricular rate data when patients are self-administering at home without supervision?
**Dr. David Bharucha:** The brief answer is, exactly as we did in our extensive PSVT program. The slightly longer answer is that, with current wearable devices, which are very low-profile and can be attached directly to the chest wall to measure an EKG, we have very reliable, high-fidelity systems with which we're very experienced.
In the upcoming AFib trial, as in all of our PSVT trial experience, patients first recognize an episode purely based on symptoms. They don't have to look at a wearable to know. Based on those well-known, classic symptoms, they attach themselves to the monitor, and we obtain a high-fidelity EKG recording. Those EKG data are then transmitted to us, after the episode, and analyzed in a blinded fashion.
It's also worth noting that even in, Phase 2, where the study medication was given in the monitored setting of an emergency room, patients who were ready to be discharged after roughly an hour went home wearing a portable device. That gave us the data out to five hours post-drug.
> “If you look at the published PK/PD curves for nasal etripamil, you would think you're looking at IV administration. What you're getting is IV-like action with a self-administered nasal spray.”
---
### [](#how-did-the-phase-2-revera-201-results-shape-the-design-of-phase-3)How did the Phase 2 ReVeRA 201 results shape the design of Phase 3?
**Dr. David Bharucha:** Patients in ReVeRA 201 responded very clearly to etripamil compared with placebo. What the trial showed was a very rapid and robust reduction in ventricular rate and, perhaps more importantly, a durable reduction in rate. That actually surprised us, because for a drug, etripamil, that acts quickly and is quickly metabolized, you would expect the effect to be fairly short-lived. That's not what we observed. The effect was clearly durable out to at least several hours. And that was after one dose of study drug; in Phase 3, patients will have the same repeat-dose option that they have with our approved indication in PSVT.
Beyond the Phase 2 rate data, the safety and tolerability profile was clean, and data on symptomatic reduction were compelling. Patients were given a set of standard PRO questions, and despite only 49 patients in the analysis population, we saw not only statistically significant symptom reduction, but what's recognized in PRO methodology as a clinically meaningful effect on a 7-point Likert scale.
The last piece that factored into the Phase 3 design was this Phase 2 finding: patients in the placebo arm required twice as many rescue medications in the emergency room as patients in the active treatment arm. That gives us strong optimism that, in Phase 3, we stand a very good chance of documenting not only reduced ventricular rate and improved symptoms, but also a lower rate of patients needing to go to an urgent care or emergency room facility.
Bottom-line: we have experience in this space. Both operationally in running this type of trial, and in patients’ using of etripamil nasal spray in the at-home, unsupervised setting. Also remember that these highly symptomatic episodes, of PSVT or of AFib-RVR, can occur without warning and our overarching goal, across both programs, is to prevent the need of these patients of going to an urgent care facility or ER.
---
### [](#how-does-a-patient-at-home-know-when-etripamil-has-worked-given-that-afib-stays-in-afib-and-the-goal-is-rate-slowing-rather-than-conversion)How does a patient at home know when etripamil has worked, given that AFib stays in AFib and the goal is rate slowing rather than conversion?
**Dr. David Bharucha:** This is an important distinction to clarify. In PSVT, the expectation is conversion. In AFib-RVR, the expectation is significant rate slowing. But the commonality between the two is that they both lead to distinct symptomatic improvement. A patient who feels they're in distress, who feels their chronic medication isn't working and that they may need to go to the ER, what we’d expect with significant rate slowing in AFib is a distinct symptomatic improvement, perhaps akin to the relief patients feel upon conversion from PSVT.
---
### [](#why-does-symptom-prompted-rate-control-at-home-matter-for-a-patient-who-may-still-eventually-need-ablation-or-cardioversion)Why does symptom-prompted rate control at home matter for a patient who may still eventually need ablation or cardioversion?
**Dr. David Bharucha:** There are several valuable potential uses here, and this is honestly what gets me enthusiastic about going to work every day.
First, it can be a standalone treatment. Sometimes all that's necessary for a patient with an episode of very rapid, highly symptomatic AFib is a cooling-off period. What tends to happen is that adrenaline starts flowing, there's a snowball effect, rapid rates lead to more adrenaline, and so on. Early and quick intervention is key in order to interrupt that cycle. So as a potential standalone treatment, this could be very valuable.
Second, this could potentially be a bridge to oral therapy. Oral medications simply take longer to produce an effect. And in the specific case of AFib, if someone is taking an oral antiarrhythmic like flecainide to try to achieve conversion at home, by the prescribing information, you need rate control first. You should not be giving flecainide, for example, to a patient who is still in rapid ventricular rates.
Third, with respect to ablation specifically, there are actually two potential windows where this could be useful. Before an ablation, patients are typically required to go off their home medications for approximately five half-lives, or close to a week. They need coverage during that interim period when they're, as I put it, naked of drug. And after an ablation, even in the most successful AFib ablation trials, there is what's called a blanking period, roughly one to several months, where the ablation is still taking hold, inflammation is settling, and all bets are off clinically and the patient often needs additional AFib suppression coverage. At-home etripamil could potentially be a useful adjunct in either of those windows.
Bottom-line: our goal for our Phase 3 AFib program is to demonstrate, similar to our PSVT trials, that self-administration of this drug is efficacious, safe, and practical for patients.
> “Patients end up walking on eggshells between episodes. They say, 'Maybe I won't go on a hiking trip, or even a vacation, because I don't want to be too far away from an emergency room.'”
---
### [](#why-does-it-matter-practically-and-personally-for-a-patient-living-with-recurrent-afib-rvr-to-have-something-they-can-reach-for-at-home)Why does it matter, practically and personally, for a patient living with recurrent AFib-RVR to have something they can reach for at home?
**Dr. David Bharucha:** It would be meaningful a lot of ways. From my experience as a practicing cardiologist and cardiac electrophysiologist, patients with episodic arrhythmias like AFib-RVR can have substantial symptoms. The palpitations can feel like one is running a marathon. Patients feel like their heart is beating out of their chest. They’re often lightheaded and can’t properly catch their breath. They sometimes think they're having a heart attack.
These episodes impose on daily life, and we also know is that the burden doesn't stop when the episode does. Patients walk on eggshells between episodes. They say, "Maybe I won't have that second cup of coffee." Or, "Maybe I won't go on a hiking trip, because I don't want to be too far away from an emergency room." We've actually presented on this very recently at the European Society of Cardiology, looking at the burden of episodic arrhythmias on patients' lives between episodes.
Beyond the patient burden, there is the growing pharmacoeconomic cost of ER visits, hospitalizations, lost work time, and all that goes with it. Our aspirations here are realistic ones: to bring this treatment not only to the approximately 2 million US patients with PSVT, but also to the even greater number patients with AFib episodes.
*Dr. David Bharucha is Chief Medical Officer at Milestone Pharmaceuticals, a cardiac electrophysiologist, and a physician-scientist with more than two decades of pharmaceutical R&D leadership experience in cardiovascular and other therapeutic areas.*
---
**Categories:** Article: Executive Interviews
---
### [FDA Grants Priority Review to Roche's Enspryng for MOGAD](https://www.clinicaltrialvanguard.com/news/fda-grants-priority-review-to-roches-enspryng-for-mogad/)
**Published:** September 10, 2026
**Author:** Jon Napitupulu
**Excerpt:** FDA grants Priority Review to Roche's Enspryng for MOGAD after Phase III trial showed 68% reduction in relapse risk versus placebo.
**Content:**
A 68% reduction in relapse risk is the number that earned Enspryng its second FDA fast-track designation. The agency granted Priority Review to Roche’s supplemental BLA for satralizumab in MOGAD after the [Phase III METEOROID trial](https://www.roche.com/media/releases/med-cor-2026-04-21b) hit its primary endpoint: time to first relapse was significantly longer on satralizumab than on placebo (p=0.0025) in adults and adolescents with myelin oligodendrocyte glycoprotein antibody-associated disease. That result is the clinical foundation for the entire filing.
The strategic context matters here. Satralizumab already carries [FDA approval for AQP4-positive NMOSD](https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/761149Orig1s000Approv.pdf), granted in August 2020, so this is an expansion into a related but distinct neuroimmune condition rather than a first-in-class entry. MOGAD and NMOSD share some clinical overlap but differ in antibody target and, critically, in how well existing therapies translate between them. Approved options for MOGAD remain limited, and the disease has largely been managed off-label, making a positive pivotal dataset meaningful for the trial community regardless of how the FDA ultimately rules.
MOGAD’s prevalence runs roughly 3 to 4 per 100,000 people in U.S. population studies, which puts the addressable patient population in a range typical for rare neurological indications. That size is enough to support a Priority Review designation, which the FDA awards when a drug may offer a substantial improvement over available therapy, but it also means enrollment for any confirmatory or post-approval trial will require careful site selection. The METEOROID design, measuring time to first relapse across a double-blind treatment period, gives regulators a clean endpoint to evaluate, and the p-value leaves little statistical ambiguity.
Priority Review sets a six-month FDA review clock rather than the standard ten months, so a decision is likely in the first half of 2027. The number to track between now and then is whether the agency requests any additional safety data on satralizumab's IL-6 receptor blockade mechanism in the [MOGAD-specific population](https://www.businesswire.com/news/home/20260909654496/en/U.S.-FDA-Grants-Priority-Review-for-Genentechs-Enspryng-for-MOGAD-an-Autoimmune-Disease-With-No-Approved-Treatments), which skews younger and includes adolescents in a way the NMOSD approval did not require at launch.
*Source link: *
**Categories:** News
---
### [Ivonescimab Meets Overall Survival Endpoint Against Pembrolizumab in Phase III Trial](https://www.clinicaltrialvanguard.com/news/ivonescimab-meets-overall-survival-endpoint-against-pembrolizumab-in-phase-iii-trial/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ivonescimab meets overall survival endpoint against pembrolizumab in Phase III HARMONi-2 trial, with statistically significant results expected to influence FDA
**Content:**
Ivonescimab’s median progression-free survival advantage over pembrolizumab, 11.14 months versus 5.82 months, was already striking when it first read out in May 2024. Now the harder number has arrived: a pre-specified interim analysis of the HARMONi-2 trial shows that overall survival advantage is also statistically significant and clinically meaningful, crossing a threshold that PFS data alone can never guarantee. That matters because OS is the endpoint that changes treatment guidelines, convinces payers, and, in the United States, determines whether the FDA’s pending biologics license application decision in November 2026 carries the weight Akeso needs it to carry.
The trial design underpins why this readout carries particular credibility. [HARMONi-2 is a randomized, double-blind Phase III trial](https://www.prnewswire.com/news-releases/ivonescimab-meets-overall-survival-key-secondary-endpoint-in-interim-analysis-of-phase-iii-harmoni-2-study-demonstrating-statistically-significant-and-clinically-meaningful-benefit-versus-pembrolizumab-in-first-line-pd-l1-positiv-302867930.html) pitting ivonescimab directly against pembrolizumab in PD-L1-positive, locally advanced or metastatic NSCLC, the same population that [pembrolizumab’s landmark first-line approval in 2016](https://pmc.ncbi.nlm.nih.gov/articles/PMC5679831/) was built on. Beating pembrolizumab head-to-head in OS, not merely in PFS, in that exact population is a genuinely different clinical argument. Ivonescimab’s mechanism, combining PD-1 blockade with VEGF inhibition in a single bispecific antibody, also resolves a long-standing practical constraint: [conventional VEGF inhibition with bevacizumab has historically been contraindicated in squamous NSCLC](https://www.onclive.com/view/bevacizumab-in-non-small-cell-lung-cancer) due to pulmonary hemorrhage risk, yet the HARMONi-2 approval in China removed restrictions on VEGF-targeted use in squamous histology patients, expanding the eligible population meaningfully.
Akeso frames this as the fourth Phase III ivonescimab regimen to show statistically significant benefit in both OS and PFS across head-to-head trials versus PD-1/PD-L1 therapies, a pattern of replication that is harder to dismiss than a single trial result. The detailed OS numbers are not yet public, reserved for an upcoming international conference and peer-reviewed publication. That staged disclosure is standard but inconvenient: the magnitude of the hazard ratio and the maturity of the data are the two inputs that will determine how aggressively investigators, guideline committees, and payers recalibrate around this drug.
The single marker to track now is the November 2026 FDA BLA decision. The OS interim data almost certainly strengthens Akeso's regulatory package, but whether the agency accepts an interim analysis or demands more mature OS data before granting approval will define whether ivonescimab enters the U.S. market this year or waits for a fuller dataset.
*Source link: *
**Categories:** News
---
### [What the FDA's New Psychedelic Framework Actually Means for the Clinic](https://www.clinicaltrialvanguard.com/article/psych-pulse/what-the-fdas-new-psychedelic-framework-actually-means-for-the-clinic/)
**Published:** September 11, 2026
**Author:** Leonardo Vando, M.D.
**Excerpt:** FDA's September 2026 NEJM framework for psychedelic drugs reshapes trial design, REMS requirements, and patient access for psilocybin and MDMA therapies.
**Content:**
Fewer than one in three adults with treatment-resistant depression ever receives a trial of a rapid-acting intervention, and most of those who do have already exhausted years of failed antidepressants. That number has sat in the back of my mind across more than 30,000 ketamine treatments at Mindbloom, and it sharpens every time the regulatory landscape shifts. This week it shifted again: the September 10 issue of the *New England Journal of Medicine* published [the FDA’s new framework for psychedelic drugs](https://www.nejm.org/doi/full/10.1056/NEJMsb2609286?af=R&rss=currentIssue), a short document with long consequences for how trials are designed, how patients will eventually access these therapies, and where the competitive landscape lands after years of regulatory turbulence.
The framework arrives in a field still absorbing the aftershocks of August 2024, when the FDA issued a [Complete Response Letter to Lykos Therapeutics](https://firstwordpharma.com/story/5995271) rejecting MDMA-assisted therapy for PTSD after a 10-to-1 advisory committee vote against approval. That vote was not primarily a verdict on MDMA’s therapeutic signal. It was a verdict on study design: inadequate functional unblinding controls, concerns about trial integrity, and the agency’s discomfort with the functional unblinding that accompanies an experiential therapy. The NEJM framework reads, in part, like the FDA’s answer to that mess. It signals that psychedelics will require purpose-built evidentiary standards, not retrofitted antidepressant protocols.
## [](#the-frameworks-structural-bet)The Framework’s Structural Bet
What the FDA appears to be constructing is a tiered oversight architecture that separates the pharmacological effect of a compound from the psychotherapeutic context in which it is delivered. That distinction sounds procedural. Clinically, it is foundational. When I think about what produces durable remission in the patients who respond to ketamine at Mindbloom, the pharmacology is necessary but not sufficient. The preparatory session, the integration work afterward, the therapeutic relationship during the medicine session: these are not decoration. They are mechanism. Any regulatory framework that treats the drug and the therapy as separable components will generate trial designs that underperform in real-world populations, because it will optimize for the compound in isolation.
The framework also signals a REMS-forward posture for future psychedelic approvals, consistent with the January 2025 expansion of [Spravato's monotherapy indication](https://headlight.health/fda-approves-jjs-spravato-as-first-monotherapy-for-treatment-resistant-depression/) under a mandatory risk management program. For MDMA and psilocybin, a REMS almost certainly means certified treatment centers, trained therapist dyads, and supervised administration windows. Those requirements will concentrate access in urban academic settings for years before any broader rollout, which means the patients I see in underserved communities, many of them Spanish-speaking, many carrying dual diagnoses, will be last in line unless trial sponsors build enrollment infrastructure that explicitly compensates for that gravitational pull toward the well-resourced and the well-connected.
COMPASS Pathways is among the furthest along in the psilocybin space. Their COMP360 Phase 3 program has produced 52-week open-label data from the [COMP005 trial](https://www.psychiatrictimes.com/view/new-52-week-topline-open-label-data-from-phase-3-comp005-trial-the-power-of-another-comp360-dose), with 258 participants enrolled in the United States across 25 mg and placebo arms. What that program will now have to navigate is the FDA's expectation that psychedelic trial designs account for functional unblinding in their statistical models, not dismiss it. That is a harder problem than it sounds when the functional unblinding problem is acute in trials of this kind.
## [](#what-this-means-for-dual-diagnosis-populations)What This Means for Dual-Diagnosis Populations
Here is the counterintuitive read on the new framework that the field tends to resist: tighter regulatory structure could actually accelerate access for the most complex patients if it forces sponsors to stop excluding them. Every psychedelic Phase 2 trial I have reviewed in the past four years has carried extensive comorbidity exclusion criteria. Active suicidal ideation out. Current substance use disorder out. Bipolar spectrum disorder out. These are, almost without exception, the patients sitting across from me in clinic. The FDA's framework, by demanding more rigorous safety monitoring architecture, gives sponsors the evidentiary scaffolding they need to run carefully monitored studies in patients who have previously been treated as too risky to enroll rather than too important to exclude.
In my practice, the patients who have shown the most striking responses to ketamine are often the ones with the most complicated histories: treatment-resistant depression layered on alcohol use disorder, or PTSD driving the substance use that every prior clinician tried to treat in isolation. The neuropharmacology here is not mysterious. Ketamine's glutamatergic mechanism, MDMA's oxytocin and serotonin surge, psilocybin's 5-HT2A agonism: each of these acts on circuits that addiction and chronic stress have reorganized. Treating the reorganization requires accessing those circuits directly. Excluding dual-diagnosis patients from psychedelic trials is not a safety decision. It is a scientific one that makes the resulting data less generalizable precisely where the disease burden is highest.
The [AtaiBeckley acquisition by Eli Lilly](https://www.investing.com/news/company-news/ataibeckley-outlines-2026-mental-health-treatment-pipeline-milestones-93CH-4436839), approved by stockholders on September 8, 2026 and expected to close September 11, brings BPL-003, VLS-01, and EMP-01 under Lilly's development umbrella three days after this framework publishes. That timing is not coincidental. Lilly now inherits the benefit of FDA's new clarity and the obligation to meet it across a portfolio of novel psychedelic compounds at various trial phases.
## [](#the-design-failure-nobody-wants-to-name)The Design Failure Nobody Wants to Name
The FDA's framework makes explicit what trial designers have been quietly absorbing for two years: the standard placebo-controlled model does not translate cleanly to experiential therapies. Functional unblinding rates in psilocybin trials routinely exceed 80%. Designing around that requires active placebo conditions, expectancy measurement at baseline, and statistical models that treat the therapeutic alliance as a moderating variable rather than noise. Most current protocols treat it as noise.
Sponsors who read the NEJM framework as primarily a compliance document will build REMS programs and stop. Sponsors who read it as a trial design signal will restructure their endpoints, their population definitions, and their therapist training protocols before the next IND hits the agency's desk. The difference will appear in FDA review letters two to four years from now, but the choices get made in the next six months.
I am watching the DEA's parallel psilocybin rescheduling petition closely. The [DEA has advanced Dr. Sunil Aggarwal's petition](https://reason.org/psychedelics-policy/psychedelics-policy-newsletter-dea-considers-rescheduling-psilocybin-fda-releases-rejection-decision-and-more/) to move psilocybin from Schedule I to Schedule II, now under Department of Justice review. If that rescheduling moves forward while the FDA's new framework is still being operationalized, the regulatory and scheduling timelines will intersect in ways the field is not yet prepared to manage. That intersection is what I will be tracking in the next Psych Pulse.
## [](#references)References
1. [New England Journal of Medicine, "FDA's New Framework for Psychedelic Drugs," Vol. 395, Issue 10, September 10, 2026](https://www.nejm.org/doi/full/10.1056/NEJMsb2609286?af=R&rss=currentIssue)
2. [First Word Pharma, FDA Complete Response Letter to Lykos Therapeutics, MDMA-Assisted Therapy for PTSD, August 9, 2024](https://firstwordpharma.com/story/5995271)
3. [Headlight Health, FDA Approves Spravato as First Monotherapy for Treatment-Resistant Depression, January 21, 2025](https://headlight.health/fda-approves-jjs-spravato-as-first-monotherapy-for-treatment-resistant-depression/)
4. [Psychiatric Times, 52-Week Topline Open-Label Data from COMPASS Pathways COMP005 Phase 3 Trial](https://www.psychiatrictimes.com/view/new-52-week-topline-open-label-data-from-phase-3-comp005-trial-the-power-of-another-comp360-dose)
5. [Investing.com, AtaiBeckley 2026 Mental Health Treatment Pipeline Milestones; Eli Lilly Merger](https://www.investing.com/news/company-news/ataibeckley-outlines-2026-mental-health-treatment-pipeline-milestones-93CH-4436839)
6. [Reason.org, DEA Considers Rescheduling Psilocybin, Psychedelics Policy Newsletter](https://reason.org/psychedelics-policy/psychedelics-policy-newsletter-dea-considers-rescheduling-psilocybin-fda-releases-rejection-decision-and-more/)
**Categories:** Psych Pulse
**Tags:** Adaptive Clinical Trial Design, FDA regulatory framework, Psilocybin MDMA, Psychedelic-Assisted Therapy, Treatment-Resistant Depression
---
### [Insilico Medicine Begins Phase III Trial of AI-Discovered Drug Rentosertib for IPF](https://www.clinicaltrialvanguard.com/news/insilico-medicine-begins-phase-iii-trial-of-ai-discovered-drug-rentosertib-for-ipf/)
**Published:** September 10, 2026
**Author:** Jon Napitupulu
**Excerpt:** Rentosertib becomes first AI-discovered drug in Phase III trial for IPF, testing lung-function decline over 52 weeks across 47 Chinese sites.
**Content:**
Rentosertib just cleared a threshold no AI-discovered drug has reached before: 320 patients, 47 sites, 52 weeks of treatment in a randomized, double-blind, placebo-controlled Phase III. Insilico Medicine dosed the first participant in [GENESIS-IPF-3](https://www.prnewswire.com/news-releases/insilico-medicine-doses-first-patient-in-genesis-ipf-3-the-worlds-first-phase-iii-trial-of-a-generative-ai-driven-innovative-drug-302873749.html) at Peking Union Medical College Hospital in China, with Shanghai Pulmonary Hospital enrolling its own first patient the same day. The trial’s primary endpoint is annual rate of decline in forced vital capacity over those 52 weeks, a direct test of whether the drug’s Phase IIa lung-function signal holds at scale and duration.
What makes the trial design worth watching is the target itself. TNIK had no prior connection to fibrosis before Insilico’s generative AI identified it, and the Phase IIa data published in *Nature Medicine* in 2025 showed a dose-dependent efficacy trend that justified the progression. Pirfenidone and nintedanib, the two [FDA-approved antifibrotics for IPF](https://www.gene.com/media/news-features/medicine-fda-approved-for-idiopathic-pulmonary-fibrosis) both cleared in October 2014, slow progression through different mechanisms; rentosertib targets a pathway neither of them touches, which is the clinical rationale for running a full Phase III rather than positioning around existing options. The FDA granted rentosertib [Orphan Drug Designation in February 2023](https://respiratory-therapy.com/products-treatment/pharmaceuticals/clinical-trials/ai-generated-drug-rentosertib-trialed-for-idiopathic-pulmonary-fibrosis/), and the drug remains investigational with no regulatory approval anywhere.
The timeline question is concrete: lead investigator Professor Zuojun Xu put the span from Phase III initiation to potential approval at three to four years under favorable conditions. That estimate, paired with the 52-week treatment window across 47 Chinese centers, means primary readout data is unlikely before late 2027 at the earliest. A 2026 Nature Biotechnology study reporting consistent reductions in biological age across six independent aging clocks adds a secondary signal Insilico will almost certainly use to support label expansion discussions if the FVC endpoint holds, though that data remains observational for now.
Insilico's financial position changes the usual risk calculus for a company at this stage. The company reported $106 million in revenue for the first half of 2026, a 287% year-over-year increase, and its first profitable half-year since its [December 2025 HKEX listing](https://insilico.com/news/p010170up1-insilico-medicine-lists-on-hong-kong-sto), with adjusted net profit exceeding $51 million. That profitability comes from out-licensing and co-development deals totaling roughly $7.3 billion in announced contract value this year alone. A company funding Phase III from licensing revenue rather than equity dilution can sustain enrollment pressure differently, and whether that commercial model survives the three-to-four-year runway to approval is the number to track alongside the FVC slope.
*Source link: *
**Categories:** News
---
### [J&J Exits LAA Closure Market, Sells Laminar Program to Jaguar LAA](https://www.clinicaltrialvanguard.com/news/jj-exits-laa-closure-market-sells-laminar-program-to-jaguar-laa/)
**Published:** September 13, 2026
**Author:** Jon Napitupulu
**Excerpt:** Johnson & Johnson sells Laminar program to newly formed Jaguar LAA for left atrial appendage closure technology development and regulatory pursuit.
**Content:**
Johnson & Johnson is exiting the left atrial appendage closure space, and a newly formed startup just picked up what it left behind. Jaguar LAA, Inc. announced the acquisition of assets from J&J’s Laminar program, a catheter-based technology designed to close and eliminate the LAA using rotational motion while leaving minimal device material exposed inside the left atrium. Financial terms were not disclosed, but the deal includes not just the assets themselves: members of the team that developed them are joining the newly incorporated company.
That design detail, minimal residual device exposure in the left atrium, is where Jaguar is staking its differentiation. The two [FDA-approved percutaneous LAA closure devices](https://www.ahajournals.org/doi/10.1161/strokeaha.115.012609) already on the market, Boston Scientific’s [WATCHMAN](https://news.bostonscientific.com/2015-03-13-Boston-Scientific-Receives-FDA-Approval-for-WATCHMAN-Left-Atrial-Appendage-Closure-Device) and Abbott’s [Amulet](https://abbott.mediaroom.com/2021-08-16-Abbotts-Amplatzer-TM-Amulet-TM-Device-Approved-by-FDA-to-Treat-People-With-Atrial-Fibrillation-at-Risk-of-Stroke), both leave an implant in place within the atrium. Whether Laminar’s approach translates that structural difference into a clinical one is the question the program’s next development stages will need to answer. Non-valvular AFib is common enough that even incremental improvements in device profile matter at scale: stroke risk in this population rises sharply with comorbidities, making LAA closure an active area of cardiology and electrophysiology practice.
Jaguar was formed in partnership with Santé Ventures, the Austin- and Boston-based healthcare investment firm with more than $1 billion under management, alongside the Laminar management team and other investors. Santé led both the company formation and the transaction with J&J. Structurally, Jaguar does what a spin-out of this type is designed to do: it consolidates the intellectual property, the technical expertise, and the institutional backing in one place, then pursues regulatory clearance without the constraints of a large-device portfolio around it.
The program still has development and regulatory review ahead of it, so the timeline to a U.S. approval bid is not set. The number to track is how quickly Jaguar moves from its existing clinical and development work into a pivotal study design, which will determine whether the minimal-exposure claim can be tested at the scale regulators require.
*Source link: *
**Categories:** News
---
### [BridgeBio Shifts BBO-8520 Focus to KRAS Combinations in Second-Line NSCLC](https://www.clinicaltrialvanguard.com/news/bridgebio-shifts-bbo-8520-focus-to-kras-combinations-in-second-line-nsclc/)
**Published:** September 9, 2026
**Author:** Jon Napitupulu
**Excerpt:** BridgeBio shifts BBO-8520 focus to KRAS combinations in second-line NSCLC, building on a 63% response rate from monotherapy.
**Content:**
A 63% objective response rate from a Phase 1 monotherapy arm is an unusual number to anchor a strategic pivot on, but that is what BridgeBio Oncology Therapeutics is doing. The company disclosed new clinical data for [BBO-8520](https://www.biospace.com/press-releases/bbot-announces-new-bbo-8520-data-highlights-strategic-focus-on-2l-nsclc-bbo-8520-combination-as-well-as-bbo-11818-and-bbo-10203-combinations-in-kras-mutant-cancers) on September 8, 2026, via an 8-K filing, and simultaneously named combination therapy in second-line or later KRASG12C-mutant NSCLC as the program’s primary path forward, with two additional combinations, BBO-11818 and BBO-10203, targeted at broader KRAS-mutant cancers.
BBO-8520 is being evaluated in the [ONKORAS-101 study](https://www.biospace.com/press-releases/bbot-announces-new-bbo-8520-data-highlights-strategic-focus-on-2l-nsclc-bbo-8520-combination-as-well-as-bbo-11818-and-bbo-10203-combinations-in-kras-mutant-cancers) (NCT06343402), an ongoing Phase 1a/1b open-label trial. The monotherapy signal comes from KRASG12C inhibitor-naïve patients in the second-line setting, a population that already has approved options: sotorasib received FDA accelerated approval for this indication in 2021, and adagrasib followed. The clinical question BBOT is betting on is whether BBO-8520 combinations can move response rates or durability beyond what those agents deliver. The filing does not specify combination efficacy figures yet, so the pivot to combinations is prospective, not already proven in the data disclosed today.
The FDA cleared BBO-8520’s IND in January 2024 and granted it [Fast Track designation](https://investor.bridgebio.com/news/news-details/2024/bridgebio-announces-fda-clearance-of-ind-application-for-bbo-8520-a-first-in-class-direct-inhibitor-of-krasg12c-on-01-03-2024/default.aspx) for adult patients in this setting, which gives the program access to more frequent FDA interactions and rolling review eligibility, though it does not accelerate efficacy requirements. Fast Track status matters most if combination data, when they arrive, are compelling enough to support an accelerated approval application before a full Phase 3 is complete.
The disclosure bundles three programs into one announcement, which makes it harder to assess where resources are actually concentrating. What will clarify the strategy is whether ONKORAS-101 expansion cohorts testing BBO-8520 combinations report response data before the end of 2026, and whether those numbers hold up against the durability bar that has tripped up earlier KRASG12C inhibitors in heavily pretreated patients.
*Source link: *
**Categories:** News
---
### [Pelacarsen Fails to Meet Primary Endpoint in Phase 3 Lp(a) Trial](https://www.clinicaltrialvanguard.com/news/pelacarsen-fails-to-meet-primary-endpoint-in-phase-3-lpa-trial/)
**Published:** September 5, 2026
**Author:** Jon Napitupulu
**Excerpt:** Pelacarsen fails to meet primary endpoint in Phase 3 Lp(a) trial, raising questions about biomarker-outcome relationships in cardiovascular drug development.
**Content:**
Pelacarsen lowered lipoprotein(a) by up to 80 percent in Phase 2, and that number turned out to mean almost nothing for what patients actually care about. Novartis announced September 4 that the [Lp(a)HORIZON Phase 3 trial](https://www.novartis.com/news/media-releases/novartis-announces-lpahorizon-phase-iii-topline-results-pelacarsen-patients-elevated-lpa-and-established-cardiovascular-disease-cvd) did not meet its primary endpoint, a composite of cardiovascular death, non-fatal heart attack, non-fatal stroke, and urgent coronary revascularization requiring hospitalization, compared to placebo. The gap between a biomarker that drops dramatically and a cardiovascular outcome that moves is the central problem the field now has to explain.
For Ionis, the financial damage is direct. Under its 2019 licensing agreement with Novartis, Ionis was entitled to tiered royalties in the mid-teens to low 20 percent range on net sales, plus [$650 million in development, regulatory, and commercial milestones](https://ir.ionis.com/news-releases/news-release-details/ionis-and-royalty-pharma-enter-royalty-agreement-11-billion). A failed outcomes trial eliminates the commercial royalty stream entirely and almost certainly voids the portion of milestones tied to approval and launch. Ionis retains whatever development milestones it has already collected, but the future payments are gone.
The failure also lands hard on the broader Lp(a) field. Approved drug options specifically targeting Lp(a) reduction remain absent, with lipoprotein apheresis the only procedure carrying an FDA clearance for Lp(a) lowering. Other antisense and small-molecule programs targeting Lp(a) are still in development, and every one of them now faces a harder question from regulators and trial sponsors: if a therapy cuts Lp(a) by 80 percent and cardiovascular events do not follow, is Lp(a) reduction the right surrogate endpoint to build a program around, or does the relationship between the biomarker and outcomes need to be reconceived before the next large outcomes trial is funded?
The Lp(a)HORIZON dataset, when Novartis releases the full results, will be the document the field studies most carefully. Subgroup analyses, baseline Lp(a) thresholds, and the absolute event rates in the placebo arm will shape whether researchers conclude the hypothesis is wrong or that the trial enrolled the wrong population at the wrong Lp(a) level. That disclosure, not the topline failure itself, is what the competing programs need to see before deciding whether to proceed.
*Source link: [https://www.sec.gov/Archives/edgar/data/874015/000114036126035802/ef20081690\_8k.htm](https://www.sec.gov/Archives/edgar/data/874015/000114036126035802/ef20081690_8k.htm)*
**Categories:** News
---
### [Fresenius Got a Warning Letter Six Months After the Complaints Started. That Gap Is the Real Problem.](https://www.clinicaltrialvanguard.com/clinops-watchdog/fresenius-got-a-warning-letter-six-months-after-the-complaints-started-that-gap-is-the-real-problem/)
**Published:** September 8, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA's August 2026 warning letter to Fresenius Kabi's Ogden, Utah plant exposes how sponsor oversight of CMOs fails long before regulators arrive.
**Content:**
The [warning letter landed on August 25, 2026](https://www.fiercepharma.com/pharma/fresenius-unit-dinged-fda-warning-letter-flagging-complaint-trend-customers), addressed to Fresenius USA Manufacturing, Inc., doing business as Fresenius Medical Care North America Ogden Plant, at 475 W. 13th St., Ogden, Utah (FEI 1713747). The FDA’s Center for Drug Evaluation and Research had conducted a March 2 to 6, 2026 inspection and found significant violations of Current Good Manufacturing Practice regulations for finished pharmaceuticals. But the FDA inspection was not the starting point. Customer complaints had already accumulated into a documented trend. A [Form 483 had already been issued](https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/fresenius-medical-care-ag-co-kgaa-730319-08252026). A [recall had already gone through in the spring](https://www.marketscreener.com/news/fresenius-medical-care-ag-co-kgaa-receives-warning-letter-from-fda-center-for-drug-evaluation-and-ce7858d3df80f220). The warning letter was a late signal in a chain that should have triggered serious corrective action much earlier. What does that sequence reveal about how the industry manages quality at contract manufacturers whose products reach clinical trial patients and dialysis-dependent populations?
The Ogden facility manufactures sterile injectables, including dialysis solutions packaged in flexible bags. The customer complaint trend the FDA flagged centered on leaking bags, a defect category that in sterile pharmaceutical manufacturing sits at the highest tier of patient-safety risk. A leaking bag means a compromised sterile barrier. In a dialysis context, that means direct exposure risk for a population with no physiological margin for error.
## [](#the-sequence-nobody-stopped)The Sequence Nobody Stopped
Reconstruct the timeline. Customer complaints about Fresenius Ogden product quality were arriving before the FDA walked in the door in March 2026. Those complaints had grown numerous enough that the FDA’s warning letter specifically characterized them as a “complaint trend,” not isolated incidents. A Form 483 followed the March inspection, documenting CGMP observations the facility was expected to address. Then, on August 25, 2026, the warning letter formalized what the complaint data and the 483 had already suggested: the Ogden plant’s quality management system had structural failures, not a single event.
The moment that deserves the most scrutiny is the gap between the complaint trend and any visible corrective escalation. Under 21 CFR Part 211.198, a finished pharmaceutical manufacturer is required to maintain written records of all drug product complaints and to conduct investigations of complaints involving possible product defects. The regulation does not say "investigate some of them." It does not say "investigate when the number feels significant." It requires a systematic, documented response to each complaint that may involve a product defect. When complaints accumulate into a trend, the CGMP framework demands that the trend itself trigger a root-cause investigation and CAPA, independent of whether any single complaint would warrant action in isolation.
Fresenius Ogden received those complaints. The FDA reviewed them during its March inspection and found the quality system's response inadequate enough to generate 483 observations. Then it found those observations inadequately resolved, or the underlying system deficiencies persistent enough, to escalate to a warning letter five months later. That is the operational timeline of a complaint-handling system that logged signals without acting on them at the velocity the risk required.
## [](#where-sponsor-oversight-failed-first)Where Sponsor Oversight Failed First
Here is the counterintuitive read on this enforcement action. Most commentary will frame the [Fresenius Ogden warning letter](https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/fresenius-medical-care-ag-co-kgaa-730319-08252026) as an FDA enforcement story. The harder question is why the sponsors and institutional customers purchasing product from this facility did not escalate this themselves before the FDA arrived.
Fresenius Kabi's contract manufacturing operation services pharmaceutical and biotech partners across both commercial supply and clinical trial drug manufacture. If any portion of the products leaving the Ogden facility feed active investigational drug programs, the sponsor oversight implications are significant. The [FDA's guidance on contract manufacturing arrangements](https://www.fda.gov/media/86193/download) is explicit: quality agreements between drug owners and contract facilities must cover audits, inspections, and communication of findings. The guidance states that quality agreements "should allow owners to evaluate" the facility's compliance posture. That is not optional language. That is the baseline expectation for any sponsor relying on a contract manufacturer to hold CGMP compliance.
If customer complaints about [leaking bags were accumulating at Ogden before March 2026](https://www.sec.gov/Archives/edgar/data/1333141/000104746914001301/a2218391z20-f.htm), sponsors with product manufactured there had at minimum two channels through which that information should have surfaced: their own complaint-handling systems, which would capture any customer-reported defect in product they distributed, and their quality agreement provisions, which should require the CMO to notify them of complaint trends that could affect product quality or patient safety. Either those notification clauses existed and were not triggered, or they existed and were triggered but the sponsor response was insufficient, or they did not exist with enough specificity to catch a trend that remained below whatever threshold Fresenius's quality team had internally set. Every one of those scenarios represents a sponsor oversight failure.
The FDA's [guidance on contract manufacturing quality agreements](https://www.fda.gov/media/86193/download) has been available since 2016. A decade is long enough to have built audit frequency, complaint-trend notification thresholds, and escalation triggers into every CMO contract in a sponsor's supplier network. The Ogden timeline suggests that for at least some customers of this facility, that work was not done.
Whether those assessments triggered additional oversight activity at the sponsor level, or whether they were processed as standalone events disconnected from the complaint trend the FDA was simultaneously documenting, matters enormously for any clinical program that used this supply chain.
## [](#the-structural-failure-behind-the-483)The Structural Failure Behind the 483
Warning letters to contract sterile manufacturers suggest a pattern that CGMP enforcement records may reflect repeatedly:: the compliance gap between what quality agreements specify and what CMO quality systems actually deliver is widest at the trend-detection layer. Individual deviations get caught. Individual out-of-specification results trigger investigations. But diffuse, cross-batch, cross-customer signals, the kind that show up as a complaint trend rather than a single critical failure, persist longer because no one system owns them cleanly. The CMO's quality system tracks complaints but may not own the customer relationship fully enough to see the pattern across multiple accounts. The sponsor's pharmacovigilance or quality team tracks their own complaints but may not have visibility into complaints from other customers of the same CMO using the same manufacturing line.
The FDA's [August 25 warning letter to Fresenius Ogden](https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/fresenius-medical-care-ag-co-kgaa-730319-08252026) puts the facility on formal notice and starts a response clock. Fresenius Medical Care, the parent entity, faces the reputational and operational cost of a public enforcement record attached to FEI 1713747. But the sponsors who sourced from Ogden during the complaint-trend period face a question the warning letter does not answer for them: what did your quality agreement require Fresenius to tell you, when did they tell you, and what did you do with it?
Any sponsor whose CRO or CMO network includes a sterile injectable manufacturer should pull their quality agreements this quarter and find the complaint-trend notification clause. If the threshold is vague, "significant complaint volume" or "patterns suggesting systemic defects" without defined numeric triggers, it will not catch the next Ogden scenario until after the FDA already has. Build a numeric threshold into the contract: any product category generating more than X complaints per 10,000 units in a rolling 90-day window triggers mandatory sponsor notification within 72 hours. That is not overcompliance. That is the minimum architecture for a supply chain accountability system that functions before a warning letter forces the conversation.
The next signal to watch is Fresenius Ogden's formal response to the August 25 warning letter, which will either demonstrate a CAPA program substantial enough to close the FDA's observations or trigger the next escalation: import alert, consent decree proceedings, or facility suspension. Sponsors still sourcing from this facility have, at most, one response cycle to determine whether their supply continuity plan can survive either outcome.
## [](#references)References
1. [FiercePharma, "Fresenius unit dinged in FDA warning letter flagging 'complaint trend' from customers"](https://www.fiercepharma.com/pharma/fresenius-unit-dinged-fda-warning-letter-flagging-complaint-trend-customers)
2. [PharmaTutor, "FDA Warns Fresenius Medical Care Over Leaking Dialysis Bags and Manufacturing Control Failures"](https://www.pharmatutor.org/pharma-news/2026/fda-warns-fresenius-medical-care-over-leaking-dialysis-bags-and-manufacturing-control-failures)
3. [MarketScreener, "Fresenius Medical Care AG receives Warning Letter from FDA Center for Drug Evaluation and Research"](https://www.marketscreener.com/news/fresenius-medical-care-ag-co-kgaa-receives-warning-letter-from-fda-center-for-drug-evaluation-and-ce7858d3df80f220)
4. [FDA, "Contract Manufacturing Arrangements for Drugs: Quality Agreements Guidance for Industry" (2016)](https://www.fda.gov/media/86193/download)
**Categories:** Clinops Watchdog
**Tags:** CLINOPS WATCHDOG, Contract Manufacturing, FDA Warning Letter, GMP compliance, Sponsor Oversight
---
### [Tarsus Pharmaceuticals Acquires Alkeus, Gildeuretinol for Stargardt Disease](https://www.clinicaltrialvanguard.com/news/tarsus-pharmaceuticals-acquires-alkeus-gildeuretinol-for-stargardt-disease/)
**Published:** September 5, 2026
**Author:** Jon Napitupulu
**Excerpt:** Tarsus Pharmaceuticals acquires gildeuretinol for Stargardt disease, an oral drug in Phase 3 development with FDA breakthrough therapy designation.
**Content:**
Tarsus Pharmaceuticals paid an undisclosed sum to acquire Alkeus Pharmaceuticals and its lead asset, gildeuretinol (ALK-001), an oral investigational drug in Phase 3 development for Stargardt disease, a [condition affecting roughly 35,000 people in the United States](https://pmc.ncbi.nlm.nih.gov/articles/PMC11282698/) with approved treatment options remaining limited. The strategic weight of this deal sits largely in what gildeuretinol has already earned from regulators: [Breakthrough Therapy, Orphan Drug, Rare Pediatric Disease, and Fast Track designations](https://theretinainstitute.org/stargardt-disease) from the FDA, a combination that signals both the seriousness of the unmet need and the agency’s early confidence in the drug’s potential.
Gildeuretinol targets the underlying biology of Stargardt disease rather than its symptoms. The drug is a deuterium-modified form of vitamin A designed to slow the toxic byproduct accumulation that drives photoreceptor damage in this inherited retinal condition. The Phase 3 program is the clinical event to track now: its design, primary endpoints, and readout timeline will determine whether Tarsus has acquired a near-term commercial asset or a longer development project. Alkeus previously ran the [SAGA study in geographic atrophy](https://www.ophthalmologytimes.com/view/alkeus-pharmaceuticals-announces-results-from-the-saga-study-of-oral-gildeuretinol-in-patients-with-ga), a separate indication, enrolling 198 patients in a 24-month randomized controlled trial, which gives the Tarsus team an existing body of safety and pharmacokinetics data to work from as the Stargardt program advances.
For Tarsus, this move extends a portfolio built around ophthalmic conditions that conventional drug development has passed over. The company already commercializes XDEMVY for Demodex blepharitis, so it brings an existing infrastructure in eye care, including relationships with ophthalmologists and optometrists, that an orphan retinal drug would typically need to build from scratch. That infrastructure matters more than it might for a typical rare disease acquisition because Stargardt disproportionately affects children and young adults, a population that tends to interact with eye care providers differently than older patients with AMD or glaucoma.
The number that will define this acquisition’s value is not the purchase price but the Phase 3 readout, specifically whether gildeuretinol can demonstrate a measurable slowdown in retinal degeneration on a validated imaging endpoint. That result, whenever it comes, decides everything downstream.
*Source link: *
**Categories:** News
---
### [Inhibrx to Present INBRX-106 Phase 2 Data in Head and Neck Cancer Study](https://www.clinicaltrialvanguard.com/news/inhibrx-to-present-inbrx-106-phase-2-data-in-head-and-neck-cancer-study/)
**Published:** September 5, 2026
**Author:** Jon Napitupulu
**Excerpt:** Inhibrx releases Phase 2 primary endpoint data for INBRX-106 in head and neck cancer, testing hexavalent OX40 agonist plus pembrolizumab.
**Content:**
Inhibrx Biosciences is releasing Phase 2 primary endpoint data for INBRX-106 on September 8, four days after announcing the webcast, a pace that signals the company wants little air between announcement and disclosure. The data come from the randomized, controlled portion of the HexAgon study, which pits INBRX-106 plus pembrolizumab against pembrolizumab alone in 68 patients with treatment-naive, PD-L1 positive (CPS ≥ 20) metastatic or unresectable recurrent [head and neck squamous cell carcinoma](https://www.clinicaltrialsarena.com/news/inhibrx-hexagon-trial-interim-results/), 33 in the combination arm and 35 on monotherapy.
The mechanistic premise is what makes this readout worth watching. OX40 agonism has a long history of underwhelming results with conventional bivalent antibodies, which lack the receptor clustering density T-cells appear to need for durable activation. INBRX-106 is hexavalent, meaning six OX40-binding domains per molecule rather than two, built on Inhibrx’s single-domain antibody platform. Whether that structural difference translates into measurable clinical benefit over [pembrolizumab, FDA-approved in this setting since June 2019](https://www.ahdbonline.com/issues/2019/august-2019-vol-12-special-issue-payers-perspectives-in-oncology-asco-2019-highlights/keytruda-first-pd-1-inhibitor-approved-as-first-line-monotherapy-for-patients-with-metastatic-or-unresectable-head-and-neck-cancer), is the question the primary endpoint answers.
Context matters for reading whatever number comes out Tuesday. The 68-patient randomized cohort is sized to detect a signal, not to power a registration claim, so a positive result would raise the immediate question of what Phase 3 design Inhibrx would pursue. A null result carries different weight: it would not just close the HNSCC program but would pressure the broader OX40 agonist hypothesis at a time when several companies have already struggled to show that costimulatory receptor activation adds meaningfully to checkpoint inhibition in solid tumors. Inhibrx Biosciences itself is a leaner entity than its predecessor, having spun out from Inhibrx, Inc. after [Sanofi’s $1.7 billion acquisition of the parent](https://www.sanofi.com/en/media-room/press-releases/2024/2024-05-30-13-01-38-2890833) closed in May 2024 and took the INBRX-101 rare disease program with it, leaving INBRX-106 and ozekibart (INBRX-109) as the remaining clinical assets.
The primary endpoint result is the concrete moment: if the combination arm shows a meaningful improvement in the pre-specified endpoint over pembrolizumab monotherapy, Inhibrx has a basis for a larger trial; if it does not, the hexavalent OX40 rationale faces a serious empirical test in the most relevant patient population the company has studied.
*Source link: *
**Categories:** News
---
### [The Mitochondrial Switch That Makes Drugs Different From Food](https://www.clinicaltrialvanguard.com/article/psych-pulse/the-mitochondrial-switch-that-makes-drugs-different-from-food/)
**Published:** September 7, 2026
**Author:** Leonardo Vando, M.D.
**Excerpt:** New Nature Neuroscience data shows MCU inhibition blocks drug reward without touching natural reward circuits, a mechanistic breakthrough for OUD trial design.
**Content:**
A pattern across my patient panel has clarified something I could not articulate cleanly until now. The patients who struggle most with opioid use disorder, the ones cycling through buprenorphine, relapsing despite good adherence, carrying the heaviest dual-diagnosis burden, share a clinical profile that our current pharmacotherapy framework was not designed to address. Their dopamine reward circuitry has been fundamentally reorganized, not merely dysregulated. Buprenorphine steadies the receptor. It does not restore the underlying bioenergetics that made drug-induced dopamine release categorically different from anything a natural reward could produce. A paper just published in [Nature Neuroscience](https://www.nature.com/articles/s41593-026-02421-x) gives that clinical observation a molecular explanation, and it changes how I am thinking about the next generation of SUD trials.
The research centers on the mitochondrial calcium uniporter, or MCU, a channel that governs calcium entry into mitochondria in dopaminergic terminals of the nucleus accumbens. The core finding is precise and striking: drugs of abuse, heroin, methamphetamine, trigger mitochondrial calcium influx through MCU in those dopaminergic terminals. Natural rewards do not. Food, water, sex: the downstream dopamine release these generate does not require MCU activation. The bioenergetic demand of drug-induced dopamine release is high enough that it conscripts mitochondrial calcium handling in a way natural reward circuitry simply never does. When MCU is genetically deleted or pharmacologically inhibited specifically in dopaminergic neurons, addiction-related behaviors attenuate, while natural reward processing stays intact.
## [](#why-monoamine-pharmacology-hits-a-ceiling)Why Monoamine Pharmacology Hits a Ceiling
That selectivity is the finding worth sitting with. Every approved medication for opioid use disorder works at the receptor or the synapse level. Buprenorphine, approved by the FDA as a partial mu-opioid agonist,, occupies the receptor and blunts the reinforcing signal from illicit opioids. Naltrexone blocks the receptor entirely. Methadone substitutes a longer-acting agonist. All three approaches intervene downstream of the bioenergetic machinery that the Nature Neuroscience data identifies as the metabolic gatekeeper of drug reward. In a randomized controlled trial, [extended-release naltrexone meaningfully reduced relapse rates in men with criminal justice involvement](https://nyulangone.org/news/opioid-relapse-rates-fall-long-term-use-medication-adults-involved-criminal-justice-system) in men with criminal justice involvement, a real benefit, but one that still leaves nearly half the treated population relapsing. What that number reflects, in clinical terms, is the ceiling that receptor-level intervention cannot break through for the most severely affected patients. The question the MCU data raises is whether that ceiling exists partly because the metabolic infrastructure driving compulsive drug-seeking has never been targeted.
The mechanistic logic is not difficult to follow. Drug-induced dopamine release in the nucleus accumbens imposes a bioenergetic load that exceeds what basal mitochondrial function can support. MCU activation pulls calcium into the mitochondrial matrix, driving ATP synthesis to meet that load. Without that energetic subsidy, the dopamine release cascade that encodes drug reward cannot sustain itself at the intensity that makes addictive drugs reinforcing. Natural rewards generate dopamine release at an amplitude that does not require this emergency metabolic support, which is precisely why MCU inhibition leaves food and water reward intact. The specificity here is not incidental. It is the therapeutic premise.
A [high-throughput screen of 1,600 clinically approved compounds](https://pmc.ncbi.nlm.nih.gov/articles/PMC8242467/) has already identified pharmacological MCU modulators, including [mitoxantrone as a selective MCU inhibitor](https://pmc.ncbi.nlm.nih.gov/articles/PMC5825229/). Those candidates were not developed with addiction in mind, and mitoxantrone’s toxicity profile disqualifies it as a clinical candidate in this population. But the screening proof-of-concept matters: MCU is druggable, the binding site exists, and the field now has a validated behavioral target with a clean selectivity window.
## [](#the-trial-design-question-nobody-is-asking-yet)The Trial Design Question Nobody Is Asking Yet
For those of us running SUD trials, this data surfaces a population-enrichment question that will define early MCU-targeted development. The patients most likely to show signal in a proof-of-concept study are those for whom receptor-level pharmacotherapy has demonstrably failed, high-relapse OUD patients with heavy drug exposure histories, precisely the population whose nucleus accumbens dopaminergic terminals have undergone the deepest addiction-related reorganization. These are also the patients most systematically excluded from early-phase trials by comorbidity criteria, polysubstance use patterns, and protocol-level restrictions on concomitant MAT. Designing an MCU-targeted Phase 1b or 2a around the patients most likely to respond, rather than the patients easiest to enroll, requires trial designers to make deliberate choices about inclusion criteria that the field has historically avoided.
Endpoint selection adds another layer of complexity. The [Nature Neuroscience findings suggest that a successful MCU inhibitor](https://pmc.ncbi.nlm.nih.gov/articles/PMC9440007/) should attenuate drug reward while leaving hedonic capacity for natural reinforcers intact. That dissociation, reduced craving and drug-seeking without anhedonia, maps poorly onto current SUD trial endpoints, which typically measure abstinence rates, urine drug screens, and global impression scales. A trial designed to detect this mechanism’s specific effect needs hedonic capacity measures alongside the standard SUD battery. Anhedonia scales borrowed from depression trial methodology, ecological momentary assessment of natural reward engagement, perhaps dopamine-relevant neuroimaging as a pharmacodynamic biomarker. None of this is standard in SUD protocols today, and that gap will need to close before an MCU inhibitor can generate a development-ready efficacy signal.
The preprint version of this work [appeared on bioRxiv](https://www.biorxiv.org/content/10.1101/2025.06.10.658191v1.full) earlier this year, which means sponsor-side colleagues paying attention have had months to start thinking through first-mover target validation strategies. The Nature Neuroscience publication now sets the evidentiary bar for what a compelling MCU-targeted IND package will need to demonstrate.
What I am watching next: whether any early-stage biotech moves to file an IND for a CNS-selective MCU inhibitor within the next 18 months, and how NIDA’s portfolio responds to a target that sits at the intersection of mitochondrial biology and dopamine neuroscience, two worlds that have rarely shared a program budget.
## [](#references)References
1. [Nature Neuroscience, “Mitochondrial calcium influx-driven bioenergetics selectively enable drug addiction”](https://www.nature.com/articles/s41593-026-02421-x)
2. [bioRxiv, Preprint: Mitochondrial calcium uniporter in dopaminergic terminals of the nucleus accumbens and addiction-related behavior](https://www.biorxiv.org/content/10.1101/2025.06.10.658191v1.full)
3. [Cell Reports (via PMC), High-throughput screen identifies mitoxantrone and amorolfine as MCU modulators](https://pmc.ncbi.nlm.nih.gov/articles/PMC8242467/)
4. [PMC, FDA approval and mechanism of buprenorphine, naltrexone, and methadone for opioid use disorder](https://pmc.ncbi.nlm.nih.gov/articles/PMC10040330/)
5. [NYU Langone, Extended-release naltrexone reduces opioid relapse rates in criminal justice-involved adults](https://nyulangone.org/news/opioid-relapse-rates-fall-long-term-use-medication-adults-involved-criminal-justice-system)
**Categories:** Psych Pulse
**Tags:** Adaptive Clinical Trial Design, Addiction Neuroscience, Novel Targets, Opioid Use Disorder, PSYCH PULSE
---
### [Kylo-11's 97% Lp(a) Reduction from a Single Dose Rewrites the Durability Standard for siRNA Cardiovascular Trials](https://www.clinicaltrialvanguard.com/article/intel-brief/kylo-11s-97-lpa-reduction-from-a-single-dose-rewrites-the-durability-standard-for-sirna-cardiovascular-trials/)
**Published:** September 4, 2026
**Author:** Moe Alsumidaie
**Excerpt:** A single 600 mg dose of Kylo-11 cut lipoprotein(a) by 97% at 48 weeks in a Phase 1 trial, setting a new efficacy benchmark for siRNA cardiovascular drug…
**Content:**
One injection. Forty-eight weeks. [Ninety-seven percent reduction in lipoprotein(a)](https://consultqd.clevelandclinic.org/novel-sirna-produces-deep-durable-reduction-in-lpa). The [Phase 1 first-in-human trial of Kylo-11](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01484-4/fulltext?rss=yes), published in *The Lancet*, just handed every RNA interference program in cardiovascular development a new efficacy floor to defend. At [600 mg, a single subcutaneous dose](https://www.vcbeathealth.com/article/61988) of this non-canonical, long-duration siRNA produced a [median 97% reduction from baseline in serum Lp(a) at week 48](https://www.vcbeathealth.com/article/61988) in a randomized, double-blind, placebo-controlled design. That number does not arrive from a maintenance dosing schedule or a quarterly injection protocol. It arrives from one dose.
The signal matters beyond the cardiovascular space. Kylo-11’s durability profile forces a rethink of how Phase 2 and Phase 3 trial designers approach dosing interval assumptions, endpoint timing, and comparator selection for any GalNAc-conjugated siRNA program. If your protocol was written around a 12-week re-dosing paradigm because that was the field’s benchmark, this data just made your design defensible only by comparison to a lower bar.
## [](#what-the-durability-mechanism-changes)What the Durability Mechanism Changes
The persistence of GalNAc-conjugated siRNAs is not accidental. These molecules [accumulate in acidic intracellular compartments](https://pmc.ncbi.nlm.nih.gov/articles/PMC7708070/), where they remain stable for months, releasing functional siRNA into newly generated Argonaute 2 (Ago2) complexes weeks after dosing. That mechanism is established. What Kylo-11 establishes is the outer boundary of what that mechanism can deliver in humans at scale: not just prolonged activity, but near-complete target suppression maintained across an observation window that most Phase 1 cardiovascular trials do not even run long enough to capture.
Compare that to [olpasiran’s OCEAN(a)-DOSE Phase 2 data](https://www.ajmc.com/view/experimental-rna-therapy-significantly-reduces-blood-pressure-up-to-6-months-study-says), where doses of 75 mg or higher every 12 weeks achieved Lp(a) reductions of 95% or greater versus placebo at 36 weeks. Olpasiran required a quarterly maintenance schedule to reach that ceiling. [Kylo-11 reached a comparable ceiling with a single injection and sustained it through 48 weeks](https://consultqd.clevelandclinic.org/novel-sirna-produces-deep-durable-reduction-in-lpa), 12 weeks beyond olpasiran’s reported observation timepoint. The operational implication for trial design is direct: if one dose can sustain suppression past 48 weeks, then a Phase 2 protocol built around monthly assessments of dosing compliance is measuring the wrong variable.
The dose-response data adds precision to that conclusion. At 225 mg, [Kylo-11 already produced durable reductions](https://consultqd.clevelandclinic.org/novel-sirna-produces-deep-durable-reduction-in-lpa). The 600 mg cohort represents the ceiling of the tested range, and the tolerability profile held across doses. That is the Phase 1 mandate delivered: safety established, dose range bounded, durability confirmed. What sponsors in adjacent RNA programs should extract is not just the efficacy headline, but the structural lesson that dose selection at Phase 1 now carries downstream consequences for how long a Phase 2 can wait before its primary endpoint assessment window even opens.
## [](#who-carries-the-operational-weight)Who Carries the Operational Weight
Approximately [1.5 billion people globally carry elevated Lp(a)](https://www.acc.org/latest-in-cardiology/articles/2025/12/01/01/feature-lipoprotein-a), a causal and independent risk factor for atherosclerotic cardiovascular disease and calcific aortic valve disease with no approved pharmacological treatment targeting Lp(a) specifically. That population scale creates a development pressure that cuts in two directions simultaneously: it justifies the investment, and it demands that Phase 3 designs are built to hold up under FDA scrutiny for a condition where the evidentiary bar for cardiovascular outcomes will be high.
The FDA’s guidance on oligonucleotide therapeutics, [“Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics”](https://www.fda.gov/media/159414/download), signals the Agency’s specific pharmacokinetic expectations for this class: tissue distribution characterization, metabolite profiling, and the handling of accumulation data in target tissues. For a molecule with the durability profile Kylo-11 has now demonstrated in humans, those expectations carry added weight. Reviewers will want to understand not just the PK curve at week 4, but the mechanism sustaining activity at week 40. Trial designers who did not build tissue PK sampling into their Phase 1 protocols may face additional Agency questions that Kylo-11’s dataset will make increasingly difficult to defer.
CNS programs should take note here as well. GalNAc conjugation is primarily hepatocyte-targeted, which constrains its direct application in non-hepatic tissues including the CNS,, but the durability mechanism itself is transferable. Any program relying on Ago2-loading for sustained gene silencing in long-duration indications will face the same downstream design questions: when does your primary endpoint assessment window open, how do you handle patients who show continued suppression beyond protocol-specified observation periods, and what does your comparator arm look like when the field’s single-dose durability benchmark is now 48 weeks?
## [](#the-design-directives-that-follow)The Design Directives That Follow
If you are running a GalNAc-siRNA cardiovascular or metabolic program and your Phase 2 protocol specifies a primary endpoint at 24 weeks post-dose, Kylo-11’s data suggests you may be measuring before the peak durability signal has fully differentiated from placebo drift. Review your observation window against a 48-week horizon, not a 24-week one. Programs that have characterized their molecule’s durability relative to olpasiran’s 36-week data should now account for the fact that the field’s current observed ceiling from a single dose extends to 48 weeks. The FDA reviewer who reads your Phase 2 design will know this data exists.
Inclisiran, the PCSK9-targeting siRNA approved for LDL reduction, established the two-dose-per-year framework as operationally viable for [chronic cardiovascular indication management](https://academic.oup.com/eurheartjsupp/article/22/Supplement_L/L53/5989616). Kylo-11’s profile raises a harder question for its own Phase 2 design team: if a single dose sustains near-complete Lp(a) suppression for 48 weeks, what is the ethical and regulatory argument for a multi-dose Phase 2 arm before durability limits are established? That question is likely to be central to FDA engagement well before any Phase 2 study begins,, and the answer will shape the endpoint architecture of every subsequent Lp(a) program in the queue.
Watch how competing Lp(a) programs engage the FDA on durability comparisons over the coming months. The Kylo-11 dataset is now the reference point every reviewer will hold in hand, and sponsors who engage the Agency proactively on how their durability data compares will be in a materially different position than those who wait for a formal Agency response to raise the comparison first.
## [](#references)References
1. [*The Lancet*, “Safety and lipoprotein(a)-lowering effects of Kylo-11, a non-canonical, long-duration small interfering RNA targeting lipoprotein(a): a first-in-human, randomised, double-blind, placebo-controlled, phase 1 trial”](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01484-4/fulltext?rss=yes)
2. [Cleveland Clinic Consult QD, “Novel siRNA Produces Deep, Durable Reduction in Lp(a)”](https://consultqd.clevelandclinic.org/novel-sirna-produces-deep-durable-reduction-in-lpa)
3. [American College of Cardiology, “Lipoprotein(a): Feature Article”](https://www.acc.org/latest-in-cardiology/articles/2025/12/01/01/feature-lipoprotein-a)
4. [PMC, “Durability of GalNAc-conjugated siRNA: Mechanism of long-term activity via intracellular compartment accumulation”](https://pmc.ncbi.nlm.nih.gov/articles/PMC7708070/)
5. [FDA, “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics” (Guidance Document)](https://www.fda.gov/media/159414/download)
6. [AJMC, “Olpasiran OCEAN(a)-DOSE Phase 2 Trial: Lp(a) Reduction Data”](https://www.ajmc.com/view/experimental-rna-therapy-significantly-reduces-blood-pressure-up-to-6-months-study-says)
7. [European Heart Journal Supplements, “Inclisiran: PCSK9-targeting siRNA for LDL reduction in cardiovascular disease management”](https://academic.oup.com/eurheartjsupp/article/22/Supplement_L/L53/5989616)
**Categories:** Intel Brief
**Tags:** cardiovascular clinical trials, lipoprotein(a), Phase 1 Trial Design, RNA interference, siRNA therapeutics
---
### [Ivonescimab Beats Pembrolizumab on Overall Survival in Phase 3 NSCLC Trial](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-pembrolizumab-on-overall-survival-in-phase-3-nsclc-trial/)
**Published:** September 4, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ivonescimab beats pembrolizumab on overall survival in Phase 3 NSCLC trial, marking a significant shift from the decade-long PD-L1 standard.
**Content:**
Pembrolizumab has been the default first-line standard for PD-L1-positive non-small cell lung cancer for nearly a decade, which makes the HARMONi-2 overall survival result genuinely difficult to set aside. [Ivonescimab monotherapy beat pembrolizumab monotherapy on overall survival](https://smmttx.com/news/press-releases/news-details/2026/Ivonescimab-Monotherapy-Demonstrates-Statistically-Significant-Overall-Survival-Benefit-Compared-to-Pembrolizumab-Monotherapy-in-PD-L1-Positive-Advanced-NSCLC-in-HARMONi-2-Study-Conducted-by-Akeso-in-China/default.aspx) in a pre-specified interim analysis of the randomized, double-blind Phase 3 trial, with Akeso announcing the result statistically significant and clinically meaningful. The 8-K Summit Therapeutics filed September 2, 2026 relays that finding under “Other Events,” which is how a U.S. partner surfaces a foreign partner’s data to investors without a separate press conference.
The design matters here. HARMONi-2 compared ivonescimab against pembrolizumab as monotherapy in patients with locally advanced or metastatic NSCLC whose tumors are PD-L1 positive, which is the exact population pembrolizumab has dominated as a single agent. Ivonescimab is a [PD-1/VEGF bispecific antibody](https://www.prnewswire.com/news-releases/ivonescimab-meets-overall-survival-key-secondary-endpoint-in-interim-analysis-of-phase-iii-harmoni-2-study-demonstrating-statistically-significant-and-clinically-meaningful-benefit-versus-pembrolizumab-in-first-line-pd-l1-positiv-302867930.html), simultaneously blocking the checkpoint pathway and VEGF-driven angiogenesis, which is the biological rationale for why it might outperform a PD-1 inhibitor alone. An OS win on that head-to-head comparison, rather than on PFS alone, removes the most common objection regulators and payers raise when newer combinations show only delayed progression.
Summit’s stake in this readout traces to a [December 2022 license agreement with Akeso](https://www.sec.gov/Archives/edgar/data/1599298/000159929823000054/R15.htm), under which Summit paid $500 million upfront for rights to ivonescimab outside China and Australia. The HARMONi-2 data were generated in China under Akeso’s program, so they inform Summit’s regulatory path in the U.S. and Europe but do not constitute a submission-ready package on their own. Summit is running its own global ivonescimab trials, and how the FDA weighs a China-conducted OS readout when evaluating a U.S. application will be the regulatory question that shapes the program’s timeline from here.
Specific OS numbers, including median survival and hazard ratio, were not disclosed in this filing, which means the full dataset presentation at a medical meeting is the next concrete event to watch. The magnitude of the survival separation will determine whether this result translates into a commercially and regulatorily decisive story outside China.
*Source link: *
**Categories:** News
---
### [In Vivo CAR-T for CNS Autoimmune Disease Looks Promising. The Trial Design Problem Is Hiding in Plain Sight.](https://www.clinicaltrialvanguard.com/opinion/in-vivo-car-t-for-cns-autoimmune-disease-looks-promising-the-trial-design-problem-is-hiding-in-plain-sight/)
**Published:** September 3, 2026
**Author:** Moe Alsumidaie
**Excerpt:** NEJM's lentiviral in vivo CAR-T data in neurologic autoimmune disease is compelling, but the trial infrastructure to validate it at scale barely exists.
**Content:**
The [September 3 correspondence in the New England Journal of Medicine](https://www.nejm.org/doi/full/10.1056/NEJMc2603114?af=R&rss=currentIssue) is the kind of result that makes oncology veterans do a double take. [Lentiviral in vivo CD19 CAR T-cell therapy](https://www.cellogentherapeutics.com/blog_detail.php?slug=in-vivo-lentiviral-car-t-therapy-autoimmune-diseases), delivered directly without the ex vivo manufacturing step that has defined the CAR-T paradigm for a decade, is showing activity in neurologic autoimmune disorders. Forget the hematology playbook for a moment: this is CAR-T attempting to reset the immune system in conditions like severe multiple sclerosis and neuromyelitis optica, diseases that destroy myelin and lives in roughly equal measure. The science is arresting. The trial design problem that follows it is worse than most people are acknowledging.
Here is the single principle the field needs to internalize before the next protocol gets written: in vivo CAR-T for CNS autoimmune indications will not fail on biology. It will fail on the absence of a clinical trial infrastructure capable of generating the evidence the FDA will actually accept.
## [](#the-infrastructure-gap-nobody-talks-about)The Infrastructure Gap Nobody Talks About
Consider a few uncomfortable trends. Neuromyelitis optica spectrum disorder, one of the most plausible near-term targets for CD19-directed CAR-T given its B-cell pathogenesis, affects an estimated [22,000 Americans as of 2022](https://www.semanticscholar.org/paper/Prevalence-of-neuromyelitis-optica-spectrum-in-the-Briggs-Shaia/bb26bcbbafa86ee78f562eb6043ce661d02028c1). Twenty-two thousand. For context, diffuse large B-cell lymphoma, the condition that made axicabtagene ciloleucel a household name in oncology, affects roughly ten times that number annually on an incidence basis. The patient pool for a rare CNS autoimmune indication is not a pipeline constraint. It is a scientific and operational emergency hiding inside an exciting dataset.
Enrollment timelines in rare neurological autoimmune diseases compound this problem in ways that sponsors tend to underestimate until they are two years into a protocol. [Published analyses of rare autoimmune trial enrollment](https://www.precisionformedicine.com/blog/clinical-trial-landscape-rare-autoimmune) consistently show that site activation in these populations runs slower than comparable oncology programs, not because demand is absent, but because the diagnostic infrastructure at most academic medical centers treats these diseases as neurology subspecialty problems rather than clinical trial opportunities. The neurologist managing a patient with refractory autoimmune encephalitis is not thinking about protocol eligibility windows. She is managing an acute crisis with the tools she has.
Then there is the safety monitoring burden. The [ASCO guidelines on immune-related adverse events for CAR-T](https://ascopubs.org/doi/10.1200/JCO.21.01992) therapy specify that [persistent Grade 3 or higher neurotoxicity warrants consideration of repeat neuroimaging — MRI or CT — every two to three days](https://eguideline.guidelinecentral.com/i/1475467-immune-related-adverse-events-car-t-cell-therapy/6). In a CNS autoimmune population, baseline MRI abnormalities are not the exception, they are the case definition. Every site in an in vivo CNS CAR-T trial needs a neuroradiology team capable of distinguishing between disease progression, treatment-related inflammatory response, cytokine release syndrome affecting the CNS, and the background noise of a demyelinating lesion that has been sitting in the periventricular white matter for three years. Most sites that treat these patients in adequate volume cannot do all four of those things consistently under protocol conditions. Oncology CAR-T centers built that infrastructure over a decade of DLBCL and ALL trials. CNS autoimmune disease has no equivalent institutional memory.
The commonly held assumption is that ex vivo manufacturing cost was the main barrier to CAR-T expansion into autoimmune indications. Remove the manufacturing bottleneck with in vivo delivery, the argument goes, and the field opens up. The manufacturing cost of goods for autologous CAR-T has been estimated at substantial figures — commonly cited in the hundreds of thousands of dollars per treatment course before any clinical markup. In vivo delivery does address that constraint in a meaningful way. But manufacturing cost was never the binding constraint for CNS autoimmune indications. Site capability and patient ascertainment were. Solving for the factory while ignoring the clinic is a category error.
## [](#what-the-fda-will-actually-ask-for)What the FDA Will Actually Ask For
Endpoint selection in rare neurologic populations presents a problem the industry has not solved, and the FDA has spent several years acknowledging without resolving. The [Rare Disease Endpoint Advancement Pilot Program](https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program), established under PDUFA VII and announced in the [Federal Register on October 27, 2022](https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program), exists precisely because the agency recognizes that validated endpoints for rare conditions are often absent when sponsors need them most. The program invites sponsors to collaborate with FDA on endpoint development before pivotal trial design is finalized. For CAR-T in CNS autoimmune disease, that conversation needs to happen now, not after a Phase 2 generates a result that a reviewer cannot anchor to a clinically meaningful threshold.
What does meaningful look like in refractory neuromyelitis optica? Annualized relapse rate has been used in pivotal trials for anti-CD19 therapies in neuromyelitis optica, including the inebilizumab program that led to FDA approval. Disability progression measured on the Expanded Disability Status Scale carries decades of precedent in MS but is widely criticized for ceiling effects in severely affected patients. Composite functional endpoints incorporating visual acuity, ambulation, and patient-reported outcomes are clinically intuitive but have not been qualified through the RDEA program for this population. A sponsor bringing in vivo CD19 CAR-T data to a Type B meeting without a pre-specified, FDA-aligned endpoint strategy is about to spend eighteen months in correspondence they could have avoided.
The counterargument worth taking seriously is this: oncology CAR-T products moved through accelerated approval on response rate endpoints without pre-established outcome validation, and the system survived. If the FDA accepted complete remission rates in relapsed/refractory DLBCL as a surrogate, why would it demand more rigor from an autoimmune indication? The answer is structural. In oncology, the alternative for a relapsed/refractory patient is often measured in weeks. The urgency creates regulatory flexibility. In CNS autoimmune disease, most patients live for years with their condition, sometimes decades, which means the FDA will apply a higher bar for durability evidence before it accepts a surrogate as reasonably likely to predict long-term clinical benefit. A six-month MRI response in a neuromyelitis optica patient does not tell a reviewer what happens at year three. In oncology, getting to year three was itself the achievement.
## [](#the-operational-principle-sponsors-must-adopt)The Operational Principle Sponsors Must Adopt
The principle that unifies all of this is what might be called the infrastructure-first imperative: for any novel modality entering a rare CNS indication, the clinical trial apparatus must be treated as a scientific asset requiring as much development investment as the therapy itself. Endpoint qualification, site capability assessment, diagnostic standardization, and safety monitoring protocols are not administrative prerequisites, they are data-generating activities whose quality determines whether the eventual regulatory submission holds together under review scrutiny.
The [lentiviral in vivo CAR-T result published in NEJM](https://www.163.com/dy/article/L5TS0O2G05525F0T.html) this week is a signal, not a solution. Sponsors who read it as permission to begin pivotal planning without engaging the RDEA program, without auditing their proposed site network for neuroradiology and immunology co-management capability, and without a pre-IND conversation about endpoint qualification are following the oncology CAR-T development model into a disease area where that model has never been stress-tested.
Sponsors who read it correctly will recognize that the in vivo delivery advance solves one bottleneck while exposing three others. The first site to run a rigorously designed, endpoint-qualified, safety-monitored CAR-T trial in a rare CNS autoimmune indication will generate evidence that shapes FDA expectations for the next decade of applications in this space. Every sponsor that files behind it will spend its FDA meetings explaining why its endpoint is not that one.
## [](#references)References
1. [New England Journal of Medicine, “Lentiviral In Vivo CD19 CAR T-Cell Therapy in Neurologic Autoimmune Disorders,” Volume 395, Issue 9, September 3, 2026](https://www.nejm.org/doi/full/10.1056/NEJMc2603114?af=R&rss=currentIssue)
2. [Briggs & Shaia et al., “Prevalence of neuromyelitis optica spectrum disorder in the United States,” Mult Scler, 2024](https://www.semanticscholar.org/paper/Prevalence-of-neuromyelitis-optica-spectrum-in-the-Briggs-Shaia/bb26bcbbafa86ee78f562eb6043ce661d02028c1)
3. [Precision for Medicine, “Clinical Trial Landscape: Rare Autoimmune Disease”](https://www.precisionformedicine.com/blog/clinical-trial-landscape-rare-autoimmune)
4. [ASCO Guidelines, “Immune-Related Adverse Events: CAR T-Cell Therapy,” Guideline Central](https://eguideline.guidelinecentral.com/i/1475467-immune-related-adverse-events-car-t-cell-therapy/6)
5. [FDA, “Rare Disease Endpoint Advancement Pilot Program,” PDUFA VII, announced October 27, 2022](https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, CAR-T Cell Therapy, CNS Autoimmune Disease, FDA Rare Disease, Neuromyelitis Optica
---
### [TScan Therapeutics cuts 75% of workforce, pauses Phase 3 ALLOHA-2 trial for TSC-101](https://www.clinicaltrialvanguard.com/news/tscan-therapeutics-cuts-75-of-workforce-pauses-phase-3-alloha-2-trial-for-tsc-101/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** TScan Therapeutics pauses Phase 3 ALLOHA-2 trial after 75% workforce cut, shifting focus to preclinical solid tumor development.
**Content:**
TScan Therapeutics just cut 75% of its workforce, and the timing makes the cut particularly stark: the company dosed its [first patient in the ALLOHA-2 Phase 3 trial](https://ir.tscan.com/news-releases/news-release-details/tscan-therapeutics-announces-first-patient-dosed-phase-3-alloha/) on July 29, 2026, roughly five weeks before the September 2 decision to pause enrollment entirely. A Phase 3 trial in AML and MDS patients at high relapse risk after allogeneic hematopoietic cell transplantation, barely open, is now on ice.
The pivot is blunt. TScan is redirecting its remaining resources toward preclinical development of an in vivo solid tumor program, abandoning the clinical-stage cell therapy infrastructure that presumably consumed most of the payroll now being eliminated. For a company that raised over $48 million through its Series B alone, with backers including Bessemer Venture Partners, GV, and Novartis Venture Fund, this is not a trimming. It is a near-total operational reset. The people who ran site monitoring, regulatory affairs, and clinical operations for [TSC-101 across the ALLOHA-2 study](https://www.sec.gov/Archives/edgar/data/1783328/000119312526379343/d309786d8k.htm) are largely gone.
What this means clinically is real and underappreciated. Post-transplant relapse in AML and MDS remains a setting where durable options are limited and where a randomized Phase 3 dataset would carry genuine weight. TSC-101 was designed to address residual disease in that window, and the trial had cleared the hardest early hurdle: first patient dosed. Pausing at that moment, rather than after an interim futility signal, signals a resource and capital problem rather than a data problem. That distinction matters to anyone tracking the competitive landscape, because it leaves the scientific hypothesis largely untested rather than discredited.
The single thing worth watching now is whether TScan discloses a partner, acquirer, or out-licensing arrangement for TSC-101 in the coming weeks. A 75% workforce reduction with a parallel pivot to preclinical work often precedes an asset sale rather than a clean shutdown, and the ALLOHA-2 trial infrastructure, however early, has enrollment-ready sites and a freshly activated IND. That infrastructure has value to any larger player looking to re-enter the post-HCT relapse prevention space without building from scratch.
*Source link: *
**Categories:** News
---
### [Inventiva Completes NATiV3 Phase 3 Trial of Lanifibranor in MASH](https://www.clinicaltrialvanguard.com/news/inventiva-completes-nativ3-phase-3-trial-of-lanifibranor-in-mash/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** Inventiva completes NATiV3 Phase 3 trial of lanifibranor in MASH with histological readout weeks away, challenging established competitors.
**Content:**
With the last patient’s 72-week visit now complete in the [NATiV3 Phase 3 trial](https://www.globenewswire.com/news-release/2026/09/02/3355510/0/en/inventiva-announces-last-patient-visit-in-nativ3-phase-3-clinical-trial-of-lanifibranor-in-mash.html), Inventiva is roughly one data-lock away from knowing whether lanifibranor can challenge a MASH treatment landscape that already has two FDA-approved agents on the board. The 72-week endpoint is not a soft biomarker readout; it is a full histological assessment, and the topline data now sits weeks, not quarters, away. That proximity is the real news here: after years of enrollment, the uncertainty resolves soon.
The strategic pressure is real. Resmetirom and obeticholic acid’s regulatory path reshaped market expectations, and lanifibranor enters that environment as a [pan-PPAR agonist](https://inventivapharma.com/our-science/) with a different mechanistic profile targeting all three PPAR isoforms simultaneously. Its Phase 2b NATIVE data, published in the *New England Journal of Medicine*, showed meaningful reductions in steatosis-activity-fibrosis scores, which gave NATiV3 its scientific rationale. Whether that mechanism translates into Phase 3 histological endpoints, specifically MASH resolution without fibrosis worsening and fibrosis improvement without MASH worsening, is what this readout will settle. The bar is demanding: [resmetirom achieved MASH resolution in 30% of patients at 52 weeks versus 10% on placebo](https://fattyliver.ca/blog/f/a-new-era-in-treating-mash) in its pivotal study, a benchmark the field now uses as an informal reference point.
NATiV3 enrolled patients with moderate to advanced fibrosis, a population where durable fibrosis regression matters as much as steatohepatitis resolution. That design choice was deliberate: it positions lanifibranor, if successful, for a patient segment where treatment decisions remain genuinely difficult and where physicians are still calibrating which agents to reach for first. An oral small molecule with a differentiated mechanism would enter formulary negotiations with something to argue about, even in a market that is no longer empty.
The single variable that will define Inventiva’s next twelve months is whether NATiV3 hits both co-primary histological endpoints. A split result, one endpoint cleared and one missed, would create a regulatory conversation the company does not want. Watch the fibrosis endpoint specifically: resolution of steatohepatitis without fibrosis improvement has not been enough to carry a MASH approval, and that precedent is now baked into how regulators evaluate the entire asset class.
*Source link: *
**Categories:** News
---
### [Ultragenyx's apazunersen fails Phase 3 primary endpoint in Angelman syndrome](https://www.clinicaltrialvanguard.com/news/ultragenyxs-apazunersen-fails-phase-3-primary-endpoint-in-angelman-syndrome/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ultragenyx's apazunersen fails Phase 3 primary endpoint in Angelman syndrome, missing both cognitive and multidomain responder measures.
**Content:**
Two years of Phase 1/2 momentum collapsed in a single readout: Ultragenyx’s [Phase 3 Aspire study](https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-phase-3-aspire-results-angelman-syndrome) for apazunersen (GTX-102) in Angelman syndrome failed both its primary endpoint and its key secondary endpoint, announced September 2, 2026. The trial did not achieve the primary measure of change from baseline in Bayley-4 cognitive raw score, and net response on the Multidomain Responder Index (MDRI) also came up empty, with no differences between treated and control groups that could support efficacy across any of the MDRI’s five individual domains, including cognition and receptive communication.
The miss is particularly sharp given how convincingly the earlier-stage data read. In April 2024, [interim Phase 1/2 results](https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-interim-phase-12-data-patients) showed rapid and clinically significant improvement in cognition on Bayley-4 relative to natural history data, alongside behavioral gains. That signal was strong enough to justify a pivotal program. The Phase 3 failure does not mean the earlier data were wrong, but it does illustrate how profoundly Angelman syndrome resists the translation from open-label, smaller cohorts to randomized, controlled trials, a pattern the field has confronted repeatedly. The safety profile in Aspire was consistent with Phase 1/2, so the drug did not become dangerous; it simply did not work by the standards a registrational study demands.
For Ultragenyx, the strategic consequences are real. Apazunersen was one of the company’s highest-profile rare neurology bets, and Angelman syndrome is a condition where families have waited a very long time for disease-modifying options. The failure also puts pressure on how the field thinks about endpoint selection for neurodevelopmental disorders. The MDRI was designed to capture broad functional benefit across five domains simultaneously, and the fact that none of those five domains individually separated from control is not a measurement artifact. It suggests the drug did not move the needle on function at the doses and duration studied.
The single marker to track now is whether Ultragenyx pursues any exploratory subgroup analyses that might identify a responsive population, or whether the program is formally discontinued. That decision will arrive with the full data presentation, and it will determine whether apazunersen has any remaining clinical future.
**What the announcement doesn’t say**
The 8-K does not disclose the full trial design, including the number of patients enrolled, the duration of treatment, or the dose levels studied in Aspire. The release does not say whether Ultragenyx intends to present the complete dataset at an upcoming scientific conference or submit it for peer review, leaving the research community without granular detail on the five MDRI domains or any subgroup performance. The release does not disclose what, if any, exploratory or pre-specified secondary endpoints showed directional signal, nor whether a biomarker or pharmacodynamic readout confirmed target engagement in the Phase 3 population. The release does not address next steps for the apazunersen program, including whether a redesigned trial, a different patient population, or program discontinuation is under consideration. We would welcome a full data package or pipeline update from Ultragenyx, and will update this piece if those details become available.
*Source link: *
**Categories:** News
---
### [Rybrevant's Second Act: How J&J Is Rewriting the Rules for EGFR-Mutant Lung Cancer](https://www.clinicaltrialvanguard.com/executiveinterviews/rybrevants-second-act-how-jj-is-rewriting-the-rules-for-egfr-mutant-lung-cancer/)
**Published:** September 14, 2026
**Author:** Moe Alsumidaie
**Excerpt:** From exon 20 insertions to subcutaneous delivery and inclusive trial design, Joshua Bauml explains why the biology demanded a different approach, and why first-line tolerability is now an efficacy argument.
**Content:**
 Joshua Bauml
Johnson & Johnson *From exon 20 insertions to subcutaneous delivery and inclusive trial design, Joshua Bauml explains why the biology demanded a different approach, and why first-line tolerability is now an efficacy argument.*
---
The EGFR exon 20 insertion mutation has long been one of oncology’s more frustrating puzzles: a clear driver, no clean oral solution, and patients running out of road before second-line therapy could even enter the picture. Johnson & Johnson’s amivantamab (Rybrevant) was built around that frustration. At the World Conference on Lung Cancer, J&J presented a cluster of datasets spanning overall survival updates from PAPILLON, tolerability findings from COPERNICUS, and subcutaneous delivery data from PALOMA-2, each adding a layer to an increasingly coherent clinical argument. Joshua Bauml, M.D., Vice President and Disease Area Stronghold Leader for Lung and Head and Neck at J&J, spent a decade treating these patients before crossing to industry. His read on the data carries both registers.
### [](#why-did-the-biology-of-egfr-exon-20-insertions-point-toward-a-bispecific-approach-rather-than-another-tki)Why did the biology of EGFR exon 20 insertions point toward a bispecific approach rather than another TKI?
**Joshua Bauml:** When you think about why EGFR exon 20 insertion mutations tend to respond poorly to tyrosine kinase inhibitors, you have to understand the mechanism. That mutation creates a pocket on the inside of the cell that is very difficult to design a pill to target. And when you do design a pill that targets it, you end up hitting a lot of wild-type EGFR, which is why historically you see a lot of wild-type toxicity, most notably diarrhea. But it doesn’t mean that EGFR is not a driver. And beyond that, when EGFR is a driver, we know MET is coming right behind.
So we felt that amivantamab was uniquely positioned to address this. And in our phase one study, one of the most dramatic moments in the development of amivantamab was seeing the first response in an exon 20 insertion patient who had literally no options outside of chemotherapy. Seeing a response there, we felt we were in a position to really help patients who need better therapy so desperately. That’s how we got going with this.
---
### [](#why-does-the-crossover-adjusted-hazard-ratio-from-papillon-tell-a-different-story-than-the-itt-estimate-and-how-should-clinicians-weigh-them-together)Why does the crossover-adjusted hazard ratio from PAPILLON tell a different story than the ITT estimate, and how should clinicians weigh them together?
**Joshua Bauml:** We know that crossover has a substantial impact on outcomes. But we also know that amivantamab has very meaningful activity in this disease in the second line. J&J is very committed to improving outcomes for patients, not just hitting a p-value. So we constructed the trial to ensure that at the time of progression, patients could get access to amivantamab.
But in the real world, not everyone is able to get a second-line therapy. Barriers exist within markets around the world. We’ve seen consistently across multiple studies that the number of patients who actually receive second-line therapy is rather limited. What the crossover-adjusted analysis helps provide is a perspective on what outcomes might look like for patients in that real-world setting.
> “We’re not just going to hit a p-value. We want to make sure we’re improving outcomes for patients worldwide.”
---
### [](#how-does-copernicus-change-the-way-the-field-should-read-amivantamabs-tolerability-profile-compared-to-what-earlier-individual-trials-showed)How does COPERNICUS change the way the field should read amivantamab’s tolerability profile compared to what earlier individual trials showed?
**Joshua Bauml:** I treated lung and head and neck cancers for a decade before I joined J&J. And one of the aspects I’m most proud of in the development of amivantamab is that J&J has been committed to improving outcomes throughout. Through the process of developing this drug, we identified a side effect, figured out how to improve it, then identified another, and improved that too. What we’ve ended up with is quite an armamentarium of ways to improve the treatment experience for patients.
What COPERNICUS allows us to do is take everything we saw in MARIPOSA and PAPILLON, which were fantastic results, and say: we actually know now, because of the iterative learnings from each of those trials, that the experience going forward doesn’t have to look like what was seen in those earlier studies. No single one of those trials allows you to comprehensively characterize that. If you look at the adverse event profile in the longer follow-up COPERNICUS data, I think it’s very clear that amivantamab-based regimens are emerging as a reasonable and feasible option for the front line in EGFR-mutant lung cancer.
---
### [](#why-does-the-25-to-30-percent-of-patients-who-never-reach-second-line-therapy-change-how-you-think-about-tolerability-in-the-first-line-setting)Why does the 25 to 30 percent of patients who never reach second-line therapy change how you think about tolerability in the first-line setting?
**Joshua Bauml:** That number of about 25 to 30 percent is one of the most consistent data points from everywhere you look. But the most interesting thing is, if you ask oncologists how many of their patients don’t get to second line, they say all of them do. The data tells us that can’t be true.
What that means to me is we need to make sure patients get the best treatment in the front line. It does two things. First, it ensures patients get access to the best possible therapies. Second, if you give them the best treatment in the front line, I truly believe you increase the likelihood that they will get a second-line treatment, because you’re not driving aggressive progression from giving an inadequate therapy upfront. We need to make sure we balance quality of life and duration of life. We want patients to live longer and better. Lung cancer is a deadly, horrible disease, and we’re here to stand side by side with patients and physicians as they fight it.
---
### [](#why-is-pharmacokinetic-noninferiority-from-paloma-3-sufficient-to-support-subcutaneous-adoption-without-a-direct-randomized-head-to-head-comparison)Why is pharmacokinetic noninferiority from PALOMA-3 sufficient to support subcutaneous adoption, without a direct randomized head-to-head comparison?
**Joshua Bauml:** When we designed PALOMA-3, the goal was clear: you’re going from an infusion that lasts a couple of hours to an injection that lasts about five minutes. That is going to improve quality of life and patient experience objectively. And then we were very pleased to see that in making that shift, you also markedly diminish the rate of administration-related reactions, down to about 15 percent, with reduced severity as well.
So now I have something that improves quality of life in terms of time in the chair and improves quality of life in terms of tolerability. And if you look at the PALOMA-3 data, there’s a hint that it might even be better on efficacy. Obviously, the study isn’t powered for that, but it’s a suggestion. From a pharmacokinetic standpoint, we’ve already demonstrated noninferiority, alongside clear improvements in the treatment experience. What PALOMA-2 does is let us say, okay, the majority of patients are going to be receiving subcutaneous. What does this look like in that setting? It’s similar to COPERNICUS in spirit: what does the picture look like when you apply the best approach? It gives people more data to help guide their discussions.
---
### [](#how-did-copernicus-address-the-structural-barriers-that-have-historically-kept-underrepresented-patients-out-of-clinical-trials)How did COPERNICUS address the structural barriers that have historically kept underrepresented patients out of clinical trials?
**Joshua Bauml:** I can speak to this as a physician who treated patients, and now as a leader at J&J. When I was treating patients, you want to get them on trial, get them access to treatment. At the same time, you do not want the trial to be structured in a way that it could lead to misleading conclusions. This led to some overly conservative inclusion/exclusion criteria being used.
One great example is prior malignancy. There was fantastic work done by David Gerber at UT Southwestern looking at this, and his team demonstrated that outcomes are identical regardless of a history of prior malignancy. So why were some trials excluding those patients? Our original trials already incorporated that knowledge. J&J has done a lot of work to improve eligibility criteria throughout our development program.
But we also know unexpected barriers can emerge. Enrollment of minority populations into clinical trials is a real problem. There are trust issues. There are also biologic issues, for instance in the way kidney function is measured, which can vary by group and affect downstream eligibility in ways that are significant. What we did with COPERNICUS was aim for a very pragmatic design. We looked at each eligibility criterion and asked: is this going to directly affect the interpretation of the data? If the answer was no, we removed it. We also reduced the burden of trial participation itself, cutting down the number of visits required for PK and scans, so patients could spend more time with their friends and loved ones than in a clinic chair. That was the whole principle. And if you look at the COPERNICUS data, we enrolled much older patient populations. To demonstrate that degree of tolerability and outcomes in a higher-risk population is something we’re really proud of.
> “We identified a side effect, figured out how to improve it, then identified another, and improved that too.”
*Joshua Bauml, M.D., is Vice President and Disease Area Stronghold Leader for Lung and Head and Neck, Johnson & Johnson.*
---
**Categories:** Article: Executive Interviews
---
### [Another AI Clinical Data Platform Just Launched. Here's What Sponsors Actually Need to Ask Before They Sign.](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/another-ai-clinical-data-platform-just-launched-heres-what-sponsors-actually-need-to-ask-before-they-sign/)
**Published:** September 14, 2026
**Author:** Krishma Shah
**Excerpt:** nPhase's AI clinical data platform promises smarter trial design, but before sponsors add another vendor, the activation-cycle math deserves a hard look.
**Content:**
The email arrives from a business development rep at a new eClinical platform vendor. The pitch is familiar: end-to-end data management, AI-assisted trial design, faster decisions. The sponsor’s clinical operations team forwards it to the CTM asking whether this replaces their EDC, their CTMS, or both. Nobody knows. The vendor meeting gets scheduled. And somewhere in the meantime, site activation for the Q4 study is already running at month five with no contracts signed.
That sequence plays out more than once a quarter in most mid-size sponsor organizations, and nPhase’s [newly launched AI clinical data management platform](https://www.fiercebiotech.com/cro/nphase-launches-clinical-data-management-platform-improve-sponsor-trial-design) will almost certainly generate the same forwarded email. The platform is positioned as an end-to-end solution for helping sponsors apply AI to trial results and trial design decisions. Before any clinical operations team books that vendor meeting, there are three operational questions that matter more than the demo.
## [](#the-activation-gap-no-platform-has-closed)The Activation Gap No Platform Has Closed
Start with the number that should anchor every eClinical vendor evaluation: [median site activation time at academic medical centers and hospitals ran 8.12 months in 2024](https://www.wcgclinical.com/wp-content/uploads/2024/01/wcg-trends-insights-2024.pdf), according to [WCG’s 2024 Trends and Insights report](https://www.wcgclinical.com/wp-content/uploads/2024/01/wcg-trends-insights-2024.pdf). [Independent sites did better at 4.37 months](https://www.appliedclinicaltrialsonline.com/view/accelerating-clinical-trial-activation), but “better” is relative when your enrollment plan assumed 60 days to first-patient-in. The gap between what sponsors plan and what sites actually need to get activated has been the defining operational failure of the last decade, and it sits almost entirely outside the data management stack.
Where activation actually dies: budget negotiation timelines, IRB submissions that go in before the protocol is final, pharmacy setup for IP accountability, and coordinator onboarding onto five simultaneous platforms the sponsor selected separately. A platform that improves how a sponsor analyzes its completed trial data does not touch any of those friction points. The honest way to evaluate any new eClinical tool is to map exactly where it touches the site’s Monday morning and where it does not. For a data management and AI trial design tool, the site’s Monday morning is largely out of scope.
That observation cuts in two directions. It means sponsors should not oversell this category of tool to site relations teams as a site burden solution. Sites I work with already manage EDC, eConsent, ePRO, IRT, and central lab portals concurrently on most complex protocols. Adding another sponsor-side data layer that sites never interact with does not increase their burden, but it also does not reduce it. The clinical operations value, if it exists, lives entirely on the sponsor side: better protocol design upstream, tighter feasibility assumptions, faster decisions when data signals shift mid-trial.
## [](#what-ai-assisted-actually-has-to-mean-to-earn-its-budget)What “AI-Assisted” Actually Has to Mean to Earn Its Budget
The FDA’s May 2023 [discussion paper on AI and machine learning in drug and biological product development](https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/artificial-intelligence-drug-development) laid out the agency’s core concern plainly: AI-generated outputs used in regulatory submissions require traceability, validation, and human oversight documentation. That framing matters for any sponsor evaluating what it means to let an AI platform influence trial design, because “AI-assisted” can mean anything from a regression model surfacing enrollment predictions to a generative system drafting protocol sections. Those are not equivalent risk categories from a documentation standpoint.
The question to put directly to any vendor in this space: which outputs from your platform do you expect to appear in a sponsor’s regulatory submission, and what validation package supports each one? If the answer is vague, that is a compliance risk wearing a product roadmap. In April 2026, the FDA issued a [warning letter to Purolea Cosmetics Lab](https://www.mastercontrol.com/gxp-lifeline/first-fda-warning-letters-for-ai-compliance/) that explicitly cited [AI misuse in cGMP documentation as a violation of 21 CFR 211.22(c) and 21 CFR 211.100](https://www.pharmtech.com/view/what-fda-s-ai-warning-letter-tells-us-about-gmp-accountability), marking the first time the agency named AI in the body of a cGMP warning letter. That precedent is in manufacturing, not clinical, but it signals FDA’s posture: AI in regulated workflows requires documented oversight, not just functional output.
Clinical ink’s EDCXtra, which integrated direct data capture, eCOA, and eConsent into a single system, represented one approach to reducing platform fragmentation when it launched. nPhase’s play appears to be further upstream: influencing trial design before data capture begins. The operational upside of that positioning is real, if the AI outputs genuinely improve protocol assumptions. Poorly designed protocols do not become better protocols through diligent site management; they generate screen failures, deviations, and amendments that cost sites months and sponsors enrollment windows. Across our network, screen failure rates on trials with overly narrow eligibility criteria run 40 to 60 percent above enrollment plan projections, and almost every one of those criteria was set before the first SIV was ever scheduled.
## [](#the-vendor-handoff-problem-nobody-budgets-for)The Vendor Handoff Problem Nobody Budgets For
Every new platform in the eClinical stack creates a vendor oversight obligation under [ICH E6(R3), Section 5.2](https://database.ich.org/sites/default/files/ICH%20E6%28R3%29_Step4_FinalConsolidatedGuideline_2026_0616_.pdf), which places full responsibility on sponsors to qualify, oversee, and document third-party service providers. That means a quality agreement, a vendor qualification audit or risk-based equivalent, and ongoing performance metrics. For a small clinical operations team managing a CRO plus four or five point-solution vendors already, adding an AI data management platform is not just a technology procurement decision. It is a QMS obligation with staffing implications.
The budget line that disappears in eClinical vendor negotiations is the internal time required to stand up and maintain that oversight infrastructure. A CTM managing two active studies does not have eight hours a week to manage a new vendor relationship during the qualification period. That cost either gets absorbed invisibly by the clinical operations team, delaying other startup activities, or it goes unmet until an audit surfaces the gap. Neither outcome shows up in the vendor’s ROI model.
For sponsors genuinely evaluating whether a consolidated AI data management platform reduces net vendor overhead rather than adding to it, the right comparison is the aggregate oversight cost of the point solutions it replaces, not the license cost in isolation. If nPhase or any comparable platform can consolidate data management functions currently split across two or three vendors, the QMS math might favor consolidation. That evaluation requires a precise inventory of current vendor agreements, not a product demo.
Clinical operations teams that run this analysis before the vendor meeting, rather than after the LOI, are the ones that make better technology decisions. The platform may be worth it. Run the numbers first, on activation timelines and vendor overhead, before the business case gets written by the person selling it.
## [](#references)References
1. [FierceBiotech, “nPhase launches AI clinical data management platform to soup up sponsor trial design”](https://www.fiercebiotech.com/cro/nphase-launches-clinical-data-management-platform-improve-sponsor-trial-design)
2. [WCG Clinical, “Trends and Insights 2024: Site Activation Cycle Times”](https://www.wcgclinical.com/wp-content/uploads/2024/01/wcg-trends-insights-2024.pdf)
3. [FDA CDER, “Artificial Intelligence in Drug Development” (Discussion Paper, May 2023)](https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/artificial-intelligence-drug-development)
4. [MasterControl GxP Lifeline, “First FDA Warning Letters for AI Compliance” (Purolea Cosmetics Lab, April 2, 2026)](https://www.mastercontrol.com/gxp-lifeline/first-fda-warning-letters-for-ai-compliance/)
5. [Clinical Trials Arena, “Clinical ink launches EDCXtra EDC platform”](https://www.clinicaltrialsarena.com/news/clinical-ink-launches-edc-platform/)
**Categories:** Clinical Trial Ops Brief
**Tags:** AI in Clinical Trials, clinical data management, eClinical Platforms, site activation, sponsor operations
---
### [MRI Surveillance Alone Outperforms MRI Plus Radiation in SCLC Trial](https://www.clinicaltrialvanguard.com/news/mri-surveillance-alone-outperforms-mri-plus-radiation-in-sclc-trial/)
**Published:** September 14, 2026
**Author:** Jon Napitupulu
**Excerpt:** MRI surveillance alone reduced cognitive failure risk by 40% versus MRI plus radiation in SCLC patients, per phase III MAVERICK trial results.
**Content:**
A 40% reduction in the risk of cognitive failure or death is a number that should retire a decades-old treatment reflex. Results from the phase III [SWOG S1827 MAVERICK trial](https://www.prnewswire.com/news-releases/phase-iii-maverick-trial-supports-brain-mri-surveillance-alone-as-standard-of-care-for-small-cell-lung-cancer-302876943.html), presented at the IASLC 2026 World Conference on Lung Cancer, show that MRI surveillance alone outperformed MRI surveillance plus prophylactic cranial irradiation (PCI) on cognitive failure-free survival in patients with small-cell lung cancer (HR 0.60; 90% CI 0.46–0.78; p=0.001). The 304-patient international trial enrolled both limited-stage and extensive-stage SCLC patients who had completed initial therapy with no brain metastases at baseline, then followed them with brain MRIs every three months in year one and every six months in year two.
The cognitive toll of PCI is where the trial’s findings are sharpest. Grade 3–5 adverse events occurred in fewer than 1% of patients on MRI surveillance alone, compared with nearly 8% in the MRI plus PCI group (0.8% vs. 7.9%; p=0.004), and one patient in the PCI arm died from encephalopathy. PCI has been used after initial SCLC therapy since pre-MRI era trials showed brain metastasis reduction and an overall survival benefit, with the most-cited evidence coming from a [1999 meta-analysis of seven randomized trials conducted between 1977 and 1994](https://www.mdpi.com/1467-3045/47/12/998). MAVERICK was built on the question of whether modern MRI is sensitive enough to detect early metastases and allow prompt salvage treatment, making the cognitive cost of prophylactic radiation unnecessary.
On survival, the trial does not yet deliver a clean answer. A preliminary analysis after 128 deaths showed no meaningful difference in overall survival between arms (HR 0.90; 90% CI 0.67–1.20), and brain metastasis-free survival was also statistically similar. The final overall survival analysis is planned after 190 deaths, so that question stays open. Notably, the cognitive failure-free survival benefit held across both disease stages and regardless of whether patients had received immunotherapy, a detail that matters given the [2024 FDA approval of durvalumab for limited-stage SCLC](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-durvalumab-limited-stage-small-cell-lung-cancer) and the growing proportion of patients now treated with checkpoint inhibitors as part of initial therapy.
Chad Rusthoven of the University of Colorado School of Medicine, who presented the data, called MRI surveillance alone the new standard of care for SCLC patients. The practical consequence: oncology teams can remove PCI from the post-treatment algorithm without a survival penalty at current follow-up, reducing a source of serious toxicity that has been widely accepted as an unavoidable tradeoff. The mature overall survival data, when it arrives after 190 deaths, will determine whether that position holds permanently.
*Source link: *
**Categories:** News
---
### [Trastuzumab Botidotin Shows 11.1-Month PFS vs T-DM1 in Phase 3 Trial](https://www.clinicaltrialvanguard.com/news/trastuzumab-botidotin-shows-11-1-month-pfs-vs-t-dm1-in-phase-3-trial/)
**Published:** September 14, 2026
**Author:** Jon Napitupulu
**Excerpt:** Trastuzumab Botidotin Phase 3 trial shows 11.1-month PFS versus 4.4 months with T-DM1 in HER2-positive breast cancer, with manageable safety profile.
**Content:**
Against a comparator producing 4.4 months of median progression-free survival, trastuzumab botidotin delivered 11.1 months in a head-to-head Phase 3 trial, a hazard ratio of 0.39 that halved the expected benchmark for this treatment line. Those results, now published in the *Journal of Clinical Oncology*, give Kelun-Biotech’s HER2-targeted ADC its fullest public data package since the NMPA approved it in October 2025 for adults with unresectable or metastatic HER2-positive breast cancer who had received at least one prior anti-HER2 therapy.
The 365-patient randomized study compared trastuzumab botidotin monotherapy against [T-DM1](https://news.abbvie.com/2013-02-22-ImmunoGen,-Inc-Announces-FDA-Approval-of-Kadcyla-Ado-Trastuzumab-Emtansine-Also-Known-as-T-DM1) in patients who had already received trastuzumab and taxane-based regimens, the population where second-line options carry the most weight. The response data reinforce the PFS finding: 76.9% of patients on trastuzumab botidotin responded versus 53.0% on T-DM1, and those responses lasted a median of 12.2 months compared to 5.7 months. Overall survival data were immature at the April 2025 cutoff, though a 38% reduction in the risk of death was observed in the trastuzumab botidotin arm, a trend the authors will need longer follow-up to confirm.
The safety read matters as much as the efficacy numbers here, because the known liability of HER2 ADCs is interstitial lung disease. Trastuzumab botidotin showed low ILD incidence, and rates of hematologic, hepatic, and gastrointestinal toxicities ran below T-DM1 levels. The dominant treatment-related adverse event was ocular toxicity, a class effect tied to the drug’s MMAF-derivative payload; the investigators report it was manageable and reversible with standardized protocols. Serious adverse event rates and treatment discontinuations were lower in the trastuzumab botidotin arm, which matters practically for patients staying on therapy long enough to realize the PFS benefit.
The JCO publication follows the data’s debut as a late-breaking oral presentation at ESMO 2025, and it arrives as the [HER2 ADC field has grown increasingly crowded](https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-fam-trastuzumab-deruxtecan-nxki-pertuzumab-unresectable-or-metastatic-her2-positive) globally. Kelun-Biotech has also opened a Phase 2 study of trastuzumab botidotin in patients who previously received a topoisomerase inhibitor payload ADC, the next sequencing question that will determine whether this drug has utility beyond its current approved line. The durability of the 12.2-month median response in that pretreated population will be the number to watch.
*Source link: *
**Categories:** News
---
### [Tam-Peli Extends Survival to 13.3 Months in Relapsed Small-Cell Lung Cancer](https://www.clinicaltrialvanguard.com/news/tam-peli-extends-survival-to-13-3-months-in-relapsed-small-cell-lung-cancer/)
**Published:** September 14, 2026
**Author:** Jon Napitupulu
**Excerpt:** Tam-Peli extends median survival to 13.3 months in relapsed small-cell lung cancer, a significant improvement over topotecan in phase III trials.
**Content:**
Topotecan has been the default second-line option for relapsed small-cell lung cancer since its [FDA approval in 1996](https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/022453s007lbl.pdf), and its survival benefit has always been modest. The phase III TAISHAN-302 trial puts a hard number on what “modest” means by comparison: patients receiving the anti-B7-H3 antibody-drug conjugate tambotatug pelitecan (Tam-Peli, YL201) lived a median of 13.3 months versus 9.4 months for topotecan, a 54% reduction in the risk of death (HR 0.46; p less than 0.0001) across 451 randomized patients.
The progression-free survival gap is even wider. Tam-Peli produced a median of 7.4 months without progression compared with 2.8 months on topotecan, a 71% reduction in the risk of progression or death (HR 0.29; p less than 0.0001). The objective response rate tells the same story from a different angle: 59.1% of patients on Tam-Peli responded versus 9.7% on topotecan. Presented at the IASLC 2026 World Conference on Lung Cancer, the findings held across prespecified subgroups including patients with brain metastases, liver metastases, and a chemotherapy-free interval under 90 days, the populations where second-line therapy most often fails.
The safety data carry real weight here. Grade 3 or higher treatment-related adverse events occurred in 46.4% of Tam-Peli patients versus 74.7% on topotecan, and no treatment-related deaths occurred in the Tam-Peli arm. Interstitial lung disease or pneumonitis appeared in 4.9% of patients on Tam-Peli, higher than the 1.4% rate on topotecan, though grade 3 events were low (0.9% in each group) and no grade 4 or 5 events were reported. That ILD signal, small as it is, will draw scrutiny in any regulatory review given its class-wide relevance to ADCs. B7-H3 is broadly expressed across SCLC tumors, with [moderate to high expression documented in roughly 64% of cases](https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2021.600238/full) in prior research, which shapes how far the eligible population could extend if Tam-Peli reaches approval.
The second-line SCLC field has grown more competitive with tarlatamab now in the mix, but the TAISHAN-302 overall survival magnitude is the number that will define Tam-Peli’s path forward. Watch whether the sponsoring team files for regulatory review in China first or pursues parallel submissions, given that the trial was conducted at Chinese centers under the leadership of Li Zhang at Sun Yat-sen University Cancer Center.
*Source link: *
**Categories:** News
---
### [Ivonescimab Cuts Death Risk 27% vs Pembrolizumab in NSCLC Trial](https://www.clinicaltrialvanguard.com/news/ivonescimab-cuts-death-risk-27-vs-pembrolizumab-in-nsclc-trial/)
**Published:** September 14, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ivonescimab cuts death risk 27% versus pembrolizumab in Phase III NSCLC trial, with median OS of 30.8 months versus 22.6 months.
**Content:**
Pembrolizumab has been the default first-line treatment for PD-L1-positive advanced NSCLC since its [FDA approval in 2016](https://pubmed.ncbi.nlm.nih.gov/28835513/), and until now no randomized Phase III trial had beaten it on both progression-free and overall survival. Akeso’s HARMONi-2 data, presented as a late-breaking abstract at WCLC 2026, changes that: ivonescimab cut the risk of death by 27% versus pembrolizumab in the intention-to-treat population, with median overall survival of 30.8 months against 22.6 months (HR 0.73; 95% CI 0.57–0.95; P=0.009). That 8.2-month gap in median OS, read out at 36 months of follow-up and 234 events, meets a prespecified O’Brien-Fleming threshold and is not a borderline result.
The PD-L1-high subgroup is where the data gets sharper. Among patients with TPS of 50% or above, the hazard ratio drops to 0.58, a 42% reduction in death risk. That population is exactly where pembrolizumab monotherapy is considered most competitive, with real-world data placing its median OS in the low-to-mid 20s of months. A sub-0.60 HR at high PD-L1 expression is a harder number to dismiss than a trial-level benefit driven by one histology or one subgroup.
The squamous finding deserves its own read. Nearly half the HARMONi-2 population (45.5%) had squamous histology, and within that group a large proportion carried features that have historically excluded patients from anti-VEGF therapy: central tumor location (72.2%), cavitation or necrosis (10%), and major vessel encasement (6.7%). Ivonescimab showed no apparent increase in bleeding risk in these patients, and the squamous OS hazard ratio was 0.65. Because conventional anti-VEGF agents carry bleeding warnings that put squamous patients off-limits, a PD-1/VEGF bispecific that appears safe in this group would open a meaningful portion of the NSCLC population to a treatment class that previously excluded them.
Ivonescimab is [already approved in China](https://www.prnewswire.com/news-releases/ivonescimab-versus-pembrolizumab-in-first-line-pd-l1-positive-nsclc-positive-overall-survival-results-from-harmoni-2-presented-at-wclc-2026-302876953.html) for this indication, based on the 2024 PFS readout. The global Phase III study, HARMONi-7, is enrolling PD-L1-high patients internationally and will be the dataset regulators outside China will require. Whether the OS benefit observed in a China-only cohort replicates across a broader, more diverse enrollment is the number worth watching when HARMONi-7 reports.
*Source link: *
**Categories:** News
---
### [Amivantamab plus chemotherapy achieves 34.3-month OS in EGFR exon 20 insertion lung cancer](https://www.clinicaltrialvanguard.com/news/amivantamab-plus-chemotherapy-achieves-34-3-month-os-in-egfr-exon-20-insertion-lung-cancer/)
**Published:** September 14, 2026
**Author:** Jon Napitupulu
**Excerpt:** Amivantamab plus chemotherapy achieves 34.3-month median overall survival in EGFR exon 20 insertion lung cancer, doubling historical survival rates.
**Content:**
Amivantamab plus chemotherapy produced a median overall survival of 34.3 months in the final analysis of the Phase 3 PAPILLON study, the longest reported median OS in EGFR exon 20 insertion-positive non-small cell lung cancer and roughly twice the historical median of 16 to 24 months for this mutation subtype. That number lands against a backdrop where [amivantamab first reached patients in this setting via accelerated approval in 2021](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-amivantamab-vmjw-metastatic-non-small-cell-lung-cancer), in the second line, based on single-arm data. PAPILLON represents the randomized, first-line confirmation of how far that trajectory has moved.
The six-month gain over the chemotherapy-alone arm (27.9 months) is harder to dismiss than the raw hazard ratio suggests. The protocol-specified analysis returned an HR of 0.87, which did not meet statistical significance, but 76 percent of eligible patients in the control arm crossed over to amivantamab after progression. A prespecified crossover-adjusted analysis cut the risk of death by 43 percent (HR 0.57; nominal p=0.003). That crossover rate also tells its own story: investigators were willing to use the combination drug as salvage in the majority of control patients, which compresses any survival separation in the unadjusted result. Twelve percent of patients in the combination arm remained on first-line treatment at the data cutoff; none in the chemotherapy arm did.
The durability signal extends beyond OS. Progression-free survival through second disease progression ran 28.3 months with the combination versus 17.5 months with chemotherapy alone, a difference of more than 10 months (HR 0.59; nominal p<0.0001). That PFS2 measure captures benefit beyond the first progression, including whatever second-line therapy patients received, and the gap holding at more than 10 months suggests the combination's advantage is not simply front-loaded. Safety was consistent with earlier reports: paronychia and neutropenia each appeared in 60 percent of patients, rash in 58 percent, with no new signals at longer follow-up. [Data were presented at the IASLC 2026 World Conference on Lung Cancer Presidential Symposium.](https://www.prnewswire.com/news-releases/johnson--johnsons-rybrevant-amivantamab-vmjw-plus-chemotherapy-delivers-longest-reported-median-overall-survival-in-egfr-exon-20-insertion-mutation-positive-lung-cancer-302877002.html)
EGFR exon 20 insertions account for roughly 12 percent of all EGFR mutations in NSCLC, a population that has historically carried a five-year survival rate of just eight percent. The PAPILLON OS data now give prescribers a randomized, final-analysis survival number to anchor first-line decisions. The figure to watch as this matures is whether the crossover-adjusted HR of 0.57 holds up to regulatory scrutiny as a basis for labeling updates, since that adjusted analysis carries more clinical weight than the headline result every time a payer or guideline committee asks whether the six-month unadjusted difference justifies the combination’s toxicity profile.
*Source link: [https://www.prnewswire.com/news-releases/johnson–johnsons-rybrevant-amivantamab-vmjw-plus-chemotherapy-delivers-longest-reported-median-overall-survival-in-egfr-exon-20-insertion-mutation-positive-lung-cancer-302877002.html](https://www.prnewswire.com/news-releases/johnson--johnsons-rybrevant-amivantamab-vmjw-plus-chemotherapy-delivers-longest-reported-median-overall-survival-in-egfr-exon-20-insertion-mutation-positive-lung-cancer-302877002.html)*
**Categories:** News
---
### [Mitapivat Just Rewrote the Treatment Floor for Transfusion-Dependent Thalassaemia, Here's What the ENERGIZE-T Data Actually Mean](https://www.clinicaltrialvanguard.com/article/article-deep-dive/mitapivat-just-rewrote-the-treatment-floor-for-transfusion-dependent-thalassaemia-heres-what-the-energize-t-data-actually-mean/)
**Published:** September 13, 2026
**Author:** Moe Alsumidaie
**Excerpt:** ENERGIZE-T's Phase 3 data shows mitapivat cut transfusion burden in 258 thalassaemia patients. Here's what the trial design reveals about the new evidentiary…
**Content:**
Picture the patient record that has defined thalassaemia care for decades: a transfusion schedule, printed or scrolling across a clinic portal, with appointments stacked every three to four weeks, year after year, from childhood into adulthood. For the 258 patients enrolled in the [ENERGIZE-T Phase 3 trial](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)00874-3/fulltext?rss=yes), that schedule became the primary endpoint. Agios Pharmaceuticals designed the entire study architecture around a single question: can one oral tablet, taken twice daily, meaningfully reduce how often these patients need someone else’s blood to survive? The answer, published in *The Lancet*, is yes, and the implications reach well beyond the thalassaemia clinic.
The headline number is straightforward. Mitapivat, the allosteric pyruvate kinase (PK) activator that the [FDA approved under the brand name AQVESME on December 23, 2025](https://www.drugs.com/history/aqvesme.html), demonstrated a statistically significant reduction in transfusion burden compared to placebo across both α-thalassaemia and β-thalassaemia patients over [48 weeks of double-blind treatment](https://ash.confex.com/ash/2024/webprogram/Paper200867.html). Of the 258 enrolled patients, 171 received [mitapivat 100 mg twice daily](https://www.drugs.com/dosage/aqvesme.html) and 87 received matched placebo. That 2:1 randomization ratio is a deliberate design choice, one that carries its own regulatory logic, and its own vulnerabilities.
But the trial’s architecture is where the real story lives. Agios did not build a surrogate-endpoint study. They built a transfusion burden study in one of the most operationally difficult patient populations in rare hematology, and the fact that the signal held across both thalassaemia types, across a global multicentre enrollment, over nearly a full year of blinding, tells sponsors something important about how the FDA is now thinking about disease modification in chronic transfusion-dependent conditions.
## [](#what-pyruvate-kinase-has-to-do-with-a-transfusion-schedule)What Pyruvate Kinase Has to Do With a Transfusion Schedule
To understand why ENERGIZE-T’s design choices matter, you need to understand what mitapivat is actually doing inside a red blood cell. [According to the drug’s prescribing information](https://www.pyrukynd.com/hcp/), mitapivat allosterically binds to the PK enzyme, stabilizing it and increasing its activity. That increased activity drives greater ATP production within the red blood cell, which extends the cell’s lifespan. In thalassaemia, the underlying hemoglobin chain imbalance causes premature red cell destruction, so a therapy that boosts cellular energy metabolism and slows that destruction attacks the disease at a mechanistic level rather than simply compensating for the anemia it produces.
That distinction matters enormously for trial design. If you believe mitapivat is symptomatic therapy, a transfusion supplement, you design a short trial with hemoglobin as your endpoint. If you believe it is disease-modifying therapy, you design a 48-week trial with transfusion burden as your endpoint, because you are making the argument that the underlying biology has changed. The FDA’s December 2025 approval, [which the agency explicitly described as which the agency described as the first oral treatment for anemia in adults with beta-thalassemia and the first drug approval of any kind for adults with alpha-thalassemia](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-anemia-thalassemia-inherited-blood-disorder), validated that argument. Agios chose the harder endpoint and won.
A [Phase 2 study of mitapivat in thalassaemia](https://pmc.ncbi.nlm.nih.gov/articles/PMC12166342/), which informed ENERGIZE-T’s design, had already established biological plausibility. The Phase 3 trial was built to confirm clinical utility at population scale, which is precisely where most rare disease programs collapse.
## [](#the-21-randomization-and-what-it-asks-of-the-placebo-arm)The 2:1 Randomization and What It Asks of the Placebo Arm
The 2:1 randomization ratio in ENERGIZE-T, 171 active, 87 placebo, is not unusual in rare disease trials, but it creates a structural tension that every sponsor running a chronic transfusion study should examine carefully. Patients in the placebo arm still receive transfusions throughout the trial. They are not undertreated. They are receiving standard of care, which means the placebo arm’s transfusion rate represents an honest real-world baseline. That is methodologically clean.
What it means operationally is that the trial required site coordinators to manage ongoing transfusion scheduling for placebo patients across a global, multicentre network over 48 weeks, while simultaneously tracking transfusion events as efficacy endpoints. [According to data presented at ASH](https://ashpublications.org/blood/article/144/Supplement%201/409/530999/ENERGIZE-T-A-Global-Phase-3-Double-Blind), the trial enrolled patients worldwide, which means harmonizing transfusion documentation standards across health systems with substantially different transfusion recording practices. A unit of packed red cells in a U.S. academic medical center is not documented the same way as in a regional hospital in Southeast Asia or the Middle East, where thalassaemia prevalence is highest. The fact that the primary endpoint held across this operational complexity is a signal about data quality, not just drug efficacy.
The safety profile reinforced the case. Mitapivat was generally well tolerated across the 48-week double-blind period, with no new safety signals identified relative to what had been observed in earlier thalassaemia studies, a result that matters because the drug carries an FDA-mandated Risk Evaluation and Mitigation Strategy (REMS) requirement linked to its commercial launch, which [was anticipated for late January 2026](https://www.vjhemonc.com/fda-grants-approval-to-mitapivat-for-non-transfusion-dependent-and-transfusion-dependent-alpha-or-beta-thalassemia/). When a product requires a REMS and still achieves a clean Phase 3 safety read, that means the risk management framework is working as designed, and it also means the Phase 3 population was well-characterized enough that the adverse event profile was predictable.
## [](#the-economic-pressure-behind-every-transfusion-unit)The Economic Pressure Behind Every Transfusion Unit
There is a number that contextualizes everything ENERGIZE-T achieved, and it comes from outside the trial itself. [A 2023 health economics analysis published in the *Journal of Medical Economics*](https://www.tandfonline.com/doi/full/10.1080/13696998.2023.2235928) estimated total annual healthcare costs for patients with transfusion-dependent β-thalassemia in the United States at [$137,125 per patient per year, with lifetime costs](https://www.tandfonline.com/doi/full/10.1080/13696998.2023.2235928) reaching $7.1 million. Those figures dwarf matched control populations. They are driven almost entirely by transfusion-related costs: the blood products themselves, the infusion center visits, the chelation therapy required to manage the iron overload that chronic transfusion causes, and the organ damage that follows when chelation is imperfect.
A therapy that meaningfully reduces transfusion frequency does not merely improve a patient’s schedule. It compresses cost curves that currently run for decades. That economic reality is why payers will scrutinize the ENERGIZE-T data as carefully as the FDA did, and why sponsors running comparable chronic transfusion trials need to build health economics endpoints into their Phase 3 designs now, not as Phase 4 afterthoughts.
The counterintuitive read on mitapivat’s approval is that it validates a mechanistic hypothesis the field has held for years but never successfully converted into a regulatory submission for thalassaemia. Pyruvate kinase activation had already been proven in pyruvate kinase deficiency, PYRUKYND’s original approved indication. The assumption was that the PK pathway would behave similarly in thalassaemia, where PK activity is secondarily impaired by the oxidative stress caused by excess globin chains. That assumption proved correct, but “proved correct” required 258 patients, 48 weeks of blinding, and a global site network capable of harmonizing transfusion data across jurisdictions. The mechanism was never the hard part. The evidentiary infrastructure was.
## [](#what-sponsors-running-rare-hematology-trials-should-take-from-this)What Sponsors Running Rare Hematology Trials Should Take From This
The ENERGIZE-T program establishes several operational precedents that matter beyond thalassaemia. First, the FDA has now accepted transfusion burden reduction as a primary endpoint for both approval and disease-modification labeling claims in chronic transfusion-dependent anemia. That is not obvious. The agency could have required hemoglobin response rates or red cell half-life measures as the primary endpoint and treated transfusion reduction as secondary. Choosing not to do so tells sponsors that the agency will accept patient-centered, clinically meaningful endpoints in rare blood disorders when the disease-modification mechanism is credibly supported by preclinical and Phase 2 data.
Second, the 48-week double-blind duration in ENERGIZE-T sets an informal comparator for any sponsor seeking a similar label. A program that submits a 24-week pivotal trial in transfusion-dependent hemolytic anemia will face a question from the FDA: why half the exposure duration of the precedent study? That question will need a rigorous, pre-negotiated answer, ideally pre-negotiated with the agency before the Phase 3 protocol is finalized.
Third, the REMS requirement attached to AQVESME’s approval is a signal sponsors should not dismiss as a commercial footnote. FDA-mandated REMS programs for novel mechanisms in rare diseases increasingly reflect the agency’s post-approval safety monitoring expectations, not just pre-approval risk management. Sponsors designing Phase 3 programs in this space should build pharmacovigilance infrastructure capable of supporting REMS compliance from Day 1 of commercial launch, not as a post-approval buildout. The gap between approval and REMS-ready commercial launch, AQVESME was approved December 23, 2025, with a launch anticipated in late January 2026, a gap that reflects the preparation required to stand up a REMS-compliant commercial infrastructure.
The deeper lesson from ENERGIZE-T is one about evidence architecture. Agios did not build a trial that would narrowly satisfy an approval threshold. They built a trial that would generate data durable enough to win formulary access, reimbursement negotiations, and guideline inclusion simultaneously. The [258-patient enrollment](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-anemia-thalassemia-inherited-blood-disorder), the 48-week blinding, the α- and β-thalassaemia inclusion, the global multicentre network: each choice added operational complexity and cost, and each choice also produced an evidentiary asset. That is the design philosophy that converts a Phase 3 win into a sustained commercial position in a rare disease market.
Every hematology program director staring at a Phase 2 readout in a transfusion-dependent population right now is looking at ENERGIZE-T and recalibrating their Phase 3 assumptions. The bar has moved. It moved on December 23, 2025, when the FDA signed the AQVESME approval letter, and it moved again when *The Lancet* published the data behind it. The transfusion schedule that has defined these patients’ lives for decades now has a challenger, and the next sponsor to reach the FDA’s desk with a chronic transfusion endpoint will be measured against 258 patients and 48 weeks of clean data.
## [](#references)References
1. [The Lancet, “Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial”](https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)00874-3/fulltext?rss=yes)
2. [FDA, “FDA Approves First Oral Treatment for Anemia in Thalassemia (AQVESME/mitapivat), December 23, 2025”](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-oral-treatment-anemia-thalassemia-inherited-blood-disorder)
3. [Agios Pharmaceuticals, PYRUKYND (mitapivat) HCP Prescribing Information: Mechanism of Action”](https://www.pyrukynd.com/hcp/)
4. [ASH Publications, “ENERGIZE-T: A Global Phase 3 Double-Blind Trial, Enrollment and Baseline Data”](https://ashpublications.org/blood/article/144/Supplement%201/409/530999/ENERGIZE-T-A-Global-Phase-3-Double-Blind)
5. [Journal of Medical Economics, “Economic and clinical burden of managing transfusion-dependent β-thalassemia in the United States” (2023)](https://www.tandfonline.com/doi/full/10.1080/13696998.2023.2235928)
6. [PMC / NCBI, Phase 2 mitapivat thalassaemia study informing ENERGIZE-T design](https://pmc.ncbi.nlm.nih.gov/articles/PMC12166342/)
7. [VJHemOnc, “FDA Grants Approval to Mitapivat (AQVESME) for Non-Transfusion-Dependent and Transfusion-Dependent Alpha or Beta Thalassemia; Commercial Launch Anticipated Late January 2026”](https://www.vjhemonc.com/fda-grants-approval-to-mitapivat-for-non-transfusion-dependent-and-transfusion-dependent-alpha-or-beta-thalassemia/)
**Categories:** Article: Deep Dive
**Tags:** ENERGIZE-T, mitapivat, pyruvate kinase activator, thalassaemia, transfusion-dependent
---
### [CMO Location Drives Site Selection for Parkinsonian Imaging Trial](https://www.clinicaltrialvanguard.com/news/cmo-location-drives-site-selection-for-parkinsonian-imaging-trial/)
**Published:** September 13, 2026
**Author:** Moe Alsumidaie
**Excerpt:** CMO location drives site selection for Parkinsonian imaging trial, requiring geographic proximity to manufacturing over patient populations.
**Content:**
When a radiopharmaceutical expires within hours of manufacture, the drug’s chemistry, not the disease’s prevalence, decides where a trial can run. That constraint shaped every decision in a site identification project PSI CRO recently completed for a sponsor running an imaging study in Parkinsonian syndromes, and the sequencing the team used offers a replicable model for any short-lived investigational agent.
The product’s shelf-life, measured in hours from manufacture to patient dosing, meant site selection had to begin with contracted manufacturing organization locations rather than with patient populations. PSI mapped movement disorder clinics that fell within a viable geographic radius of each planned CMO site, then layered in investigator experience and enrollment feasibility. That order matters: reversing it, by identifying clinically ideal sites first, would have produced a list that looked strong on paper but couldn’t receive a viable dose. Parkinsonian syndromes present across a wide clinical spectrum, from idiopathic Parkinson’s disease to rarer atypical forms, and imaging studies in this category typically require centers with subspecialty movement disorder expertise rather than general neurology capacity, which already limits the qualifying pool before geography enters the calculation.
The radiopharmaceutical constraint is not unique to this program. FDA’s draft Q1 stability guidance, developed under ICH, [addresses stability testing requirements](https://insider.thefdagroup.com/p/q1-stability-testing-of-drug-substances-and-drug-products) for drug substances and products broadly, but short-lived radiopharmaceuticals face compounding operational pressure: each batch has a hard expiration window that cannot be extended by cold-chain optimization or alternative packaging. A site that is two hours outside the delivery radius on a slow logistics day is effectively unreachable. PSI’s approach of anchoring the site map to CMO coordinates first absorbs that hard constraint before any other variable enters the feasibility model.
For sponsors in this position, the practical consequence is a smaller but higher-confidence site list: fewer sites qualify by geography, but those that do can actually dose patients without a product loss event. The metric worth tracking in a study like this is not the number of sites activated but the proportion that successfully complete at least one dosing visit without a shelf-life failure, since that figure reflects whether the CMO-anchored strategy held under real operational conditions.
*Source link: *
**Categories:** News
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### [Iza-bren 2.5 mg/kg dose advances to Phase 3 in EGFR-mutated NSCLC](https://www.clinicaltrialvanguard.com/news/iza-bren-2-5-mg-kg-dose-advances-to-phase-3-in-egfr-mutated-nsclc/)
**Published:** September 13, 2026
**Author:** Jon Napitupulu
**Excerpt:** Iza-bren 2.5 mg/kg dose advances to Phase 3 in EGFR-mutated NSCLC after demonstrating superior efficacy and manageable safety in global patient populations.
**Content:**
Three dose levels entered a randomized expansion cohort; only one came out with a Phase 3 ticket. SystImmune and Bristol Myers Squibb will present at WCLC in Seoul on September 14 showing that 2.5 mg/kg dosed on days one and eight of a three-week cycle produced higher efficacy than the lower doses tested, with a safety profile the investigators judged manageable, and that result locks in the dose for [IZABRIGHT-Lung01](https://www.prnewswire.com/news-releases/systimmune-inc-to-present-new-global-data-on-iza-bren-izalontamab-brengitecan-in-egfr-mutated-non-small-cell-lung-cancer-at-wclc-congress-2026-302875960.html), the registrational study of iza-bren in EGFR-mutated NSCLC after progression on a third-generation EGFR inhibitor.
The importance of that population context is worth sitting with. Osimertinib is the dominant third-generation EGFR inhibitor, and [FDA-approved in both first and adjuvant settings](https://www.astrazeneca.com/media-centre/press-releases/2018/us-fda-approves-tagrisso-as-1st-line-treatment-for-EGFR-mutated-non-small-cell-lung-cancer.html), which means a large and growing share of metastatic EGFR-mutated patients will eventually need a next option. The FDA recognized that gap when it granted iza-bren [Breakthrough Therapy Designation in August 2025](https://news.bms.com/news/details/2025/Izalontamab-Brengitecan-EGFRxHER3-ADC-Granted-Breakthrough-Therapy-Designation-by-U-S--FDA-for-Patients-with-Previously-Treated-Advanced-EGFR-Mutated-Non-Small-Cell-Lung-Cancer/default.aspx) for patients with EGFR exon 19 deletions or exon 21 L858R mutations whose disease had progressed on a prior EGFR inhibitor.
What the WCLC data add is a global dimension. Earlier efficacy and safety signals for iza-bren came primarily from Chinese patient cohorts; the Phase 1 randomized expansion presented by Alexander Spira enrolled a global population and showed consistency with those earlier results. That matters for a registrational strategy: regulators in the U.S. and Europe typically want to see their own patient populations represented before an approval package is assembled. Confirming the profile holds internationally is a prerequisite, not a formality, for IZABRIGHT-Lung01 to support a broad label.
Iza-bren targets both EGFR and HER3, two receptors that drive proliferation in epithelial cancers, and couples that bispecific antibody to a topoisomerase I inhibitor payload. The dual-targeting design is the rationale for calling it potentially first-in-class as an EGFRxHER3 ADC, though that designation ultimately depends on what reaches approval first. The concrete question now is whether the 2.5 mg/kg dose holds its efficacy advantage over the full breadth of the IZABRIGHT-Lung01 enrollment, where patient heterogeneity and longer follow-up will test what the expansion cohort suggested.
*Source link: *
**Categories:** News
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### [Tam-Peli meets primary endpoint for relapsed small-cell lung cancer in phase III trial](https://www.clinicaltrialvanguard.com/news/tam-peli-meets-primary-endpoint-for-relapsed-small-cell-lung-cancer-in-phase-iii-trial/)
**Published:** September 13, 2026
**Author:** Jon Napitupulu
**Excerpt:** Tam-Peli meets primary endpoint for relapsed small-cell lung cancer in phase III trial, showing OS benefit over topotecan in Chinese patients.
**Content:**
Topotecan has been the default second-line chemotherapy for relapsed small-cell lung cancer for decades, and its survival benefit in that setting is modest at best. Against that backdrop, interim data from the randomized phase III TAISHAN-302 trial show that [Tam-Peli (tambotatug pelitecan, YL201) cut the risk of death compared with topotecan](https://www.globenewswire.com/news-release/2026/09/13/3360614/0/en/roche-s-collaborator-medilink-announces-phase-iii-data-for-tam-peli-showing-significantly-improved-overall-survival-in-chinese-patient-population-with-relapsed-small-cell-lung-canc.html) in Chinese patients whose disease progressed after platinum-based chemotherapy, with or without a PD-L1 inhibitor. The trial hit its primary endpoint of overall survival, a result that matters because OS wins in this indication are rare and regulatorily meaningful.
The study enrolled 451 Chinese patients and tested Tam-Peli at 2.0 mg/kg intravenously on day one of each 21-day cycle, capped at 200 mg, against topotecan. That design mirrors the population and comparator most regulatory agencies would want to see for a second-line SCLC submission. The choice of topotecan as the control arm is notable: [topotecan received FDA approval for relapsed SCLC](https://www.biospace.com/glaxosmithkline-receives-approval-for-hycamtin-r-topotecan-capsules-for-the-treatment-of-relapsed-small-cell-lung-cancer-first-fda-approved-oral-th) based on data decades old, and [lurbinectedin earned accelerated approval in June 2020](https://www.onclive.com/view/fda-approves-lurbinectedin-for-metastatic-sclc) for the same platinum-pretreated population without a head-to-head OS comparison against topotecan. A statistically significant OS advantage over topotecan in a randomized phase III trial is a harder bar to clear than accelerated approval, which means this dataset, if it holds under full review, carries weight for a conventional approval pathway.
Tam-Peli is an anti-B7H3 antibody-drug conjugate, a target that also has active programs in prostate cancer: a phase II study in heavily pretreated metastatic castration-resistant prostate cancer showed antitumor activity as of late 2025. The SCLC readout is the more advanced dataset, and the OS result gives Roche and MediLink their clearest clinical validation yet for the molecule. Roche’s collaboration with MediLink positions it to potentially file in China on the strength of TAISHAN-302 data from a Chinese-only population, though whether that dataset supports a broader global filing will depend on how regulators outside China treat single-country phase III evidence.
The number to track from here is the hazard ratio for OS once full data are presented, likely at a major oncology congress. The width of the confidence interval and the magnitude of the survival separation will determine whether this result can anchor a registration package or whether a broader confirmatory study becomes necessary.
*Source link: *
**Categories:** News
---
### [What FDA's July 2026 Psychedelic Drug Guidance Actually Says About Trial Design](https://www.clinicaltrialvanguard.com/opinion/what-fdas-july-2026-psychedelic-drug-guidance-actually-says-about-trial-design/)
**Published:** September 12, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA's finalized psychedelic guidance rewrites the rules on blinding, informed consent, and session monitoring, and the NEJM's September 2026 analysis reveals…
**Content:**
A framework analysis in The New England Journal of Medicine carries of FDA’s finalized psychedelic drug guidance, published by the agency in [July 2026](https://www.fda.gov/media/169694/download). The [draft had circulated since June 2023](https://www.appliedclinicaltrialsonline.com/view/fda-finalizes-guidance-clinical-trial-design-psychedelic-drug-development), so sponsors have had time to study it. But the NEJM authors read the final version closely enough to surface language that the press coverage largely ignored, language about blinding integrity, informed consent specificity, and session-level staffing requirements that carry direct operational weight. The guidance deserves the same close reading here, section by section, because the delta between the press release and the document itself is where trial design decisions get made.
> “The informed consent document for psychedelic clinical trials should clearly describe that subjects may experience changes in perception, cognition, and judgment that persist for many hours, as well as increased vulnerability and suggestibility during the treatment session.”
The operative phrase is “persist for many hours.” FDA is not asking sponsors to note the possibility of altered states in a boilerplate adverse events list, the guidance requires the consent document to characterize the temporal arc of cognitive impairment as a named feature of the treatment experience. Under 21 CFR 50.25, informed consent must describe “reasonably foreseeable risks or discomforts”; what the psychedelic guidance adds is the requirement to specify the altered judgment window explicitly, because the subject’s capacity to report distress, withdraw consent, or recognize harm is itself compromised during that window. Sponsors who copy-paste language from their CNS precedent library will miss this.
## [](#the-blinding-problem-fda-named-directly)The Blinding Problem FDA Named Directly
The MAPP2 MDMA-assisted therapy trial exposed the blinding problem before FDA ever finalized a word of this guidance. In that Phase 3 study, [71.2% of participants in the MDMA-assisted therapy arm no longer met DSM-5 criteria for PTSD](https://pmc.ncbi.nlm.nih.gov/articles/PMC10579091/) at end of study, versus 47.6% in the placebo-with-therapy group, a striking efficacy signal that FDA’s review team then had to weigh against the near-impossibility of blinding participants to an active psychedelic compound. The agency resolved that tension not by rejecting the data, but by building functional unblinding into the evidentiary framework itself.
> “The profound subjective effects of psychedelic drugs can lead to functional unblinding of participants, investigators, and study staff. Sponsors should describe in their protocols how they plan to assess the degree of unblinding and address the potential bias introduced by unblinding in their statistical analysis plans.”
This is a meaningful regulatory concession, and it carries an equally meaningful obligation. FDA is acknowledging that perfect blinding is not achievable for psychedelic compounds, the pharmacodynamic experience is itself the tell. But the concession comes with a price: sponsors must now build unblinding assessment instruments into the protocol, quantify the bias they introduce, and adjust their statistical analysis plans accordingly. That is not a small ask. Sponsors accustomed to treating blinding integrity as a binary compliance check must now treat it as a continuous variable requiring its own measurement strategy.
The precedent this sets for the broader CNS field is harder to overstate than it appears on first reading.
> “Active placebos, such as low-dose versions of the investigational psychedelic or other psychoactive substances, may help maintain blinding; however, their use introduces additional considerations regarding safety and interpretation of trial results.”
FDA is flagging a trap here, not just offering a tool. An active placebo that preserves blinding integrity also creates a comparison arm with its own pharmacological activity, which means the effect size the sponsor observes may reflect the gap between two active compounds rather than the gap between drug and no drug. For a field where effect sizes drive advisory committee votes and reimbursement negotiations, that methodological ambiguity lands directly on the label claim. Sponsors reaching for active placebos to solve the blinding problem need a statistician and a clinical pharmacologist in the same room before they finalize the design.
## [](#session-staffing-as-a-protocol-requirement)Session Staffing as a Protocol Requirement
The session monitoring section contains the guidance’s most operationally specific language. FDA did not leave staffing to clinical judgment.
> “During treatment sessions, two trained monitors are required. The lead monitor must possess relevant clinical training and experience. Dosing sessions for psychedelic substances are expected to last multiple hours, during which the monitors must remain with the subject.”
The dual-monitor requirement, combined with session durations that the guidance characterizes as “multiple hours,” transforms the resource calculus for multi-site trials. A sponsor running 20 sites with weekly dosing sessions across an 8-week treatment course cannot staff this from a standard site coordinator pool. The lead monitor’s “relevant clinical training and experience” requirement, deliberately undefined in the guidance, which is a drafting choice, not an oversight, gives FDA latitude to scrutinize monitor qualifications at inspection. Sponsors should define those qualifications explicitly in the protocol and the monitoring plan rather than leave the question open for FDA to raise during inspection. COMPASS Pathways, whose [COMP360 psilocybin formulation received FDA Breakthrough Therapy designation for treatment-resistant depression in 2018](https://www.fiercepharma.com/fierce-50/compass-pathways), has been navigating exactly this staffing architecture across its global Phase 3 program, the guidance codifies what leading sponsors already learned operationally.
But staffing is only one dimension of what FDA is actually monitoring at the session level. The deeper signal in this section concerns documentation.
> “Sponsors should ensure that detailed records of the treatment sessions are maintained, including documentation of the subject’s condition and any interventions performed during the session.”
Session-level source documentation is a familiar requirement under 21 CFR 312.62, but the psychedelic context changes what “detailed records” must capture. A subject’s condition during a six-hour psilocybin session involves fluctuating states of consciousness, periods of non-verbal experience, and potential for acute psychological distress requiring intervention, none of which fits the checkbox architecture of a standard adverse event form. Sponsors need to design session documentation instruments before they design the case report form, because the CRF will inherit whatever gaps the session instrument leaves.
## [](#what-the-nejm-analysis-adds-to-the-guidance-itself)What the NEJM Analysis Adds to the Guidance Itself
One analytical gap the guidance cannot fill for itself is situating these requirements inside the broader regulatory history of drugs with high potential for expectancy effects and therapist influence. The relevant parallel is not to prior large-scale psychedelic submissions but to the clinical trial literature on psychotherapy components embedded in pharmacological trials. When a drug’s mechanism requires a structured therapeutic relationship, the therapeutic relationship itself becomes a confound. The guidance addresses this through its blinding and statistical requirements, but it leaves one structural question unanswered: how should sponsors separate the drug effect from the therapy effect when both are required for the treatment to work?
FDA’s finalized guidance does not resolve that question. It manages it, by requiring sponsors to characterize the confound, quantify it, and account for it analytically. That is a pragmatic regulatory position, and it is the right one given the field’s current evidence base. But it means every advisory committee reviewing a psychedelic NDA will face a drug-therapy decomposition argument, and sponsors who have not pre-specified how they will address that argument in their statistical analysis plan will be defending it under pressure in a public hearing.
The MAPP2 data made the case that the combined treatment works. The guidance now asks sponsors to build the evidentiary architecture to explain why, and to do it prospectively, not in a response to a complete response letter.
Sponsors with active IND-stage psychedelic programs should treat [FDA’s July 2026 guidance](https://www.federalregister.gov/documents/2026/07/14/2026-14158/psychedelic-drugs-considerations-for-clinical-investigations-guidance-for-industry-availability) not as a compliance checklist but as a protocol design document. Read the blinding assessment requirement and build the instrument. Read the monitor staffing language and define “relevant clinical training” in writing before the first site initiation visit. Read the session documentation requirement and design the source instrument before the CRF. The next public moment where this framework gets tested in real time is the first psychedelic NDA advisory committee meeting, and whichever sponsor files first will be answering questions that this guidance raised but did not close.
## [](#references)References
1. [NEJM, “FDA’s New Framework for Psychedelic Drugs,” Volume 395, Issue 10, September 10, 2026](https://www.nejm.org/doi/full/10.1056/NEJMsb2609286?af=R&rss=currentIssue)
2. [FDA, “Psychedelic Drugs: Considerations for Clinical Investigations,” Final Guidance, July 2026](https://www.fda.gov/media/169694/download)
3. [Nature Medicine, MAPP2 Phase 3 MDMA-Assisted Therapy Trial Results](https://pmc.ncbi.nlm.nih.gov/articles/PMC10579091/)
4. [Fierce Pharma, COMPASS Pathways COMP360 Breakthrough Therapy Designation](https://www.fiercepharma.com/fierce-50/compass-pathways)
5. [Applied Clinical Trials, “FDA Finalizes Guidance: Clinical Trial Design for Psychedelic Drug Development”](https://www.appliedclinicaltrialsonline.com/view/fda-finalizes-guidance-clinical-trial-design-psychedelic-drug-development)
6. [Baker Donelson, “FDA Finalizes Guidance on Psychedelic Drugs for Clinical Investigations”](https://www.bakerdonelson.com/fda-finalizes-guidance-on-psychedelic-drugs-for-clinical-investigations-key-implications-for-hospitals-and-trial-sponsors)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, CNS Therapies, FDA Guidance, Psychedelic Drugs, Remote Informed Consent
---
### [Ennov Appoints Dr. Oxana Pickeral as New Chief Executive Officer](https://www.clinicaltrialvanguard.com/news/ennov-appoints-dr-oxana-pickeral-as-new-chief-executive-officer/)
**Published:** September 12, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Ennov appoints Dr. Oxana Pickeral as Chief Executive Officer, succeeding 27-year founder Olivier Pâris amid accelerating regulatory compliance demand.
**Content:**
Ennov has run on the same founding CEO for 27 years, so the appointment of Dr. Oxana Pickeral to replace Olivier Pâris carries weight beyond a routine leadership change. Pâris co-founded the company in 1999 and built it to [€58.4 million in 2024 revenue](https://www.pharmaspotter.com/vendors/ennov), averaging 20 percent annual growth over the five years through 2025. Handing that over to an incoming chief is a genuine inflection, not a housekeeping move, and what Pickeral does with the product roadmap in the next 12 months will say more than the announcement itself.
Pâris moves to Ennov’s board, which keeps him in the room without keeping him at the controls. That structure is common in founder transitions and gives Pickeral room to operate while preserving institutional knowledge, though it also means the board dynamic will be unusually loaded at first. [The September 9 announcement](https://en.ennov.com/news/announcement/ennov-appoints-oxana-pickeral-as-chief-executive-officer/) offers no detail on Pickeral’s prior role or where she comes from, so her specific background in clinical operations or regulatory technology remains unconfirmed from available sources.
The timing is not accidental. The EU Clinical Trials Regulation hit a hard compliance deadline in January 2025, requiring all trials previously approved under the older directive to migrate to the [CTR framework](https://www.ema.europa.eu/en/human-regulatory-overview/research-development/clinical-trials-human-medicines/clinical-trials-regulation). That pressure accelerated demand for platforms that unify regulatory, clinical, and pharmacovigilance workflows under one audit trail. Ennov’s pitch is exactly that unified stack, and a new CEO inherits both the growth opportunity and the obligation to execute against customers who are mid-migration and impatient.
The number to watch is whether revenue growth holds above that 20 percent average under new leadership. Ennov went from €33.4 million in 2022 to €58.4 million in 2024, a 75 percent gain in two years. That pace is difficult to sustain organically, which means Pickeral’s first visible strategic choice will likely be whether to push deeper into existing accounts, expand geographically, or pursue acquisitions. Her answer to that question is the real story.
*Source link: *
**Categories:** News
---
### [PerZeption Receives NEI SBIR Grant for AMD Photostress Recovery Test](https://www.clinicaltrialvanguard.com/news/perzeption-receives-nei-sbir-grant-for-amd-photostress-recovery-test/)
**Published:** September 12, 2026
**Author:** Jon Napitupulu
**Excerpt:** PerZeption receives NEI SBIR grant to develop a photostress recovery test for AMD on tablet hardware, aiming to detect vision loss earlier than standard testing
**Content:**
An NEI peer-review panel decided that measuring how quickly vision recovers after bright light exposure is clinically meaningful enough to fund, and that judgment matters as much as the grant itself. PerZeption, a Boston-based startup, received a Phase I SBIR award from the [National Eye Institute](https://www.nei.nih.gov/grants-and-funding/research-priorities/small-business) to develop a new photostress recovery test for age-related macular degeneration on its Angular Indication Measurement (AIM) tablet platform, in collaboration with the Brunson Center for Translational Vision Research at UC Irvine. Phase I NEI SBIR awards carry up to $323,090 in total costs, so the dollars are modest. The scientific endorsement is not.
The clinical problem the grant targets is specific: [roughly 19.8 million Americans aged 40 and older](https://www.cdc.gov/vision-health-data/prevalence-estimates/amd-prevalence.html) live with some stage of AMD, and current photostress recovery testing collapses the entire recovery process into a single time point, requires dedicated equipment, and demands trained staff. That combination keeps the test out of routine practice. PerZeption’s funded work will characterize the full recovery curve, both achromatic and chromatic, on hardware clinics already own. A richer recovery profile could flag functional loss before standard acuity testing picks it up, which is where the diagnostic value sits for a disease that destroys central vision gradually.
The AIM platform already covers more than 20 visual functions and runs as a cloud application on standard tablets and phones. PerZeption says AMD photostress recovery is among the functions currently without a commercial device, and the Phase I work is explicitly framed as generating feasibility data to support a regulatory pathway. That framing signals the company intends a cleared device, not just a research instrument. The collaboration with UC Irvine gives the feasibility study academic clinical access, and the company’s prior AIM validation work has appeared in *Investigative Ophthalmology & Visual Science*, *Optometry and Vision Science*, and, published earlier this month, *Scientific Reports*.
The number to watch from here is what the Phase I data show about test-retest repeatability. A fast, tablet-based photostress recovery measure only displaces legacy methods in clinical workflows if its coefficient of variation is tight enough to detect disease progression reliably, and that threshold will determine whether the company’s regulatory submission holds up.
*Source link: *
**Categories:** News
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### [Lyell completes LYL273 manufacturing transfer, delays Phase 1/2 timeline](https://www.clinicaltrialvanguard.com/news/lyell-completes-lyl273-manufacturing-transfer-delays-phase-1-2-timeline/)
**Published:** September 12, 2026
**Author:** Jon Napitupulu
**Excerpt:** Lyell completes LYL273 manufacturing transfer to its Bothell facility, though the Phase 1/2 colorectal cancer trial now faces enrollment delays.
**Content:**
Lyell Immunopharma’s GCC-targeted CAR-T candidate LYL273 has cleared its manufacturing transfer to the company’s Bothell, Washington facility, but the Phase 1/2 trial in metastatic colorectal cancer is running behind its earlier schedule. The 8-K filed September 10, 2026 pairs the manufacturing milestone with a timeline revision, and for a program where manufacturing readiness was the stated gating factor, the two pieces of news carry opposite weight.
The [8-K](https://www.sec.gov/Archives/edgar/data/1806952/000119312526387890/d278670d8k.htm) confirms the technology transfer for LYL273 to Lyell’s LyFE Manufacturing Facility is complete. That matters because autologous cell therapy programs routinely stall at the point where academic-scale manufacturing protocols have to be rebuilt inside a commercial cGMP environment. Completing the transfer removes that specific bottleneck. The Phase 1 portion of the trial is [designed to enroll up to roughly 95 patients](https://www.marketscreener.com/news/lyell-immunopharma-completes-technology-transfer-for-lyl273-and-provides-updated-timeline-on-phase-1-ce785bdfd88df021), with a Phase 2 expansion of approximately 60 more at the recommended dose once that dose is identified. The revised timeline means that enrollment is now expected to progress on a later schedule than the company previously communicated, though the 8-K filing does not disclose a new target date in the truncated source body available.
The biology behind LYL273 is worth understanding here. Guanylyl cyclase C is expressed on colorectal epithelium and retained on the surface of most colorectal metastases, making it a cleaner target than the broadly expressed antigens that have driven toxicity in earlier solid-tumor CAR-T attempts. A [published Phase 1 study of a dual GCC/CD19-targeted CAR-T in 15 relapsed or refractory metastatic colorectal cancer patients](https://pubmed.ncbi.nlm.nih.gov/39298141/) in China showed the antigen is actionable, though sample sizes at this stage of development make cross-trial comparisons unreliable. Metastatic colorectal cancer now has a growing menu of approved options, including [adagrasib plus cetuximab for KRAS G12C-mutated disease](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer), but patients who relapse after multiple lines of therapy face sharply narrower choices, which is where LYL273 is aimed.
The practical consequence for trial watchers is straightforward: manufacturing is no longer the constraint, and the program now depends on enrollment pace and dose-finding at sites that can handle autologous cell therapy logistics. The number to track is when Lyell reports its first safety and feasibility data from the Bothell-manufactured product, since that readout will determine whether the manufacturing transfer preserved the cell product characteristics established during earlier development.
*Source link: *
**Categories:** News
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### [China Accepts NCX 470 NDA for Review in Glaucoma Treatment](https://www.clinicaltrialvanguard.com/news/china-accepts-ncx-470-nda-for-review-in-glaucoma-treatment/)
**Published:** September 12, 2026
**Author:** Jon Napitupulu
**Excerpt:** China's drug regulator accepts NCX 470 NDA for review, advancing Nicox's nitric oxide-donating glaucoma drop toward potential approval in a major ophthalmology
**Content:**
China’s drug regulator has accepted Nicox’s NDA for NCX 470, putting a nitric oxide-donating glaucoma drop one formal review cycle away from a potential approval in one of the world’s largest ophthalmology markets. That acceptance matters because the [NCX 470 program](https://www.nicox.com/assets/files/EN-FPI-NCX-470-Phase-2-trial_20180802_final.pdf) has already run two pivotal Phase 3 trials, DENALI and MONT BLANC, and the China filing draws on that clinical package rather than requiring a separate local trial program from scratch.
NCX 470 is a [second-generation bimatoprost eye drop that donates nitric oxide](https://www.kowapharma.com/newsroom/ncx-470-phase-3-results), which adds trabecular outflow enhancement on top of the uveoscleral mechanism bimatoprost already provides. In the DENALI trial, NCX 470 0.1% met non-inferiority against latanoprost 0.005%, with mean IOP reductions from baseline reaching into the 7-plus mmHg range across measured timepoints. That two-mechanism profile is the competitive argument Nicox is making, and it now gets tested against the National Medical Products Administration’s review standard rather than the FDA’s.
The NMPA acceptance does not guarantee a smooth path. China’s standard review clock for a new chemical entity typically runs 200 working days once an application is accepted, though priority designations can shorten that window. Nicox has not disclosed whether NCX 470 received priority status in China, so the standard timeline is the working assumption. The company’s ability to sustain operations through that review period is the real watch item: Nicox is a small French biotech, and the gap between NDA acceptance and commercial revenue in a new geography puts pressure on its cash runway regardless of how the clinical data read.
The China acceptance also arrives while the U.S. regulatory path for NCX 470 remains unresolved. [NMPA’s 2022 registration provisions](https://english.nmpa.gov.cn/2022-06/30/c_785628_8.htm) allow for a parallel review process independent of FDA action, so Chinese approval is not contingent on U.S. clearance. Whether Nicox can convert an NMPA green light into a commercial partnership in China, and on what economic terms, is the decision that will actually determine whether the regulatory milestone translates into shareholder value.
*Source link: *
**Categories:** News
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### [Headlands Research Opens Grand Rapids Site Led by Rheumatology Investigator](https://www.clinicaltrialvanguard.com/news/headlands-research-opens-grand-rapids-site-led-by-rheumatology-investigator/)
**Published:** September 11, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Headlands Research opens Grand Rapids site led by rheumatology investigator Eric Slavin, expanding clinical trial capacity in the Midwest.
**Content:**
Sponsor demand in rheumatology and autoimmune disease is outpacing available site capacity in much of the Midwest, and Headlands Research is betting Grand Rapids can absorb some of that pressure. The network announced a new de novo site in Grand Rapids, Michigan, led by Eric Slavin, MD, a board-certified rheumatologist and internist who has spent years running Phase III and IV studies in rheumatoid arthritis and related autoimmune conditions, including biologic and targeted disease-modifying treatments and biosimilar development.
The site’s therapeutic scope is intentionally wide. Rheumatology and autoimmune disease are the anchors, but the team will also run studies in cardiometabolic disease, chronic kidney disease, obesity, hypertension, hyperlipidemia, and vaccines. That breadth matters for a network that needs each site to pull enrollment weight across multiple sponsor relationships, not just a single specialty. Headlands has [completed more than 5,000 clinical trials](https://headlandsresearch.com/about-us/) across its sites, and adding an investigator with Slavin’s Phase III track record in a market not yet crowded with competing research infrastructure is a straightforward way to add billable capacity without acquiring an existing practice’s legacy complications.
The Grand Rapids opening is the fourth site addition in a short stretch. Headlands recently brought on [Headlands Research – Sacramento and Headlands Research – CTR-Lincoln](https://headlandsresearch.com/news/headlands-research-announces-new-de-novo-clinical-research-site-in-grand-rapids-michigan/) through the acquisition of two CTR sites, and opened Headlands Research – Western Washington. Three of those four are in markets outside the traditional trial-dense corridors of the Northeast and California, which suggests the network is deliberately building coverage in regions where sponsors historically struggle to find qualified investigators for specialty indications.
The Grand Rapids site is still being developed, with an expected opening later this year. The number to watch once it opens is how quickly Slavin’s site gets onto sponsor shortlists for rheumatology and autoimmune studies, given that investigator reputation in those indications tends to drive site selection at least as much as geography does.
*Source link: *
**Categories:** News
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### [Velocity Clinical, Stitch expand partnership across 70+ sites to boost screening attendance](https://www.clinicaltrialvanguard.com/news/velocity-clinical-stitch-expand-partnership-across-70-sites-to-boost-screening-attendance/)
**Published:** September 11, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Velocity Clinical and Stitch expand partnership across 70+ sites, achieving a 9-point reduction in no-show rates and boosting screening attendance by over 20%.
**Content:**
A 9-percentage-point drop in no-show rates sounds modest until you see the denominator: that figure came out of more than 90,000 scheduled visits across a subset of Velocity’s sites, translating to more than a 20% gain in screening attendance. That’s the data behind [Velocity Clinical Research’s decision](https://velocityclinical.com/velocity-and-stitch-expand-global-partnership/) to roll Stitch, its patient engagement platform, out across its entire network of 70-plus sites in the United States and Europe under a new multi-year agreement.
The numbers from the pilot period are specific enough to matter operationally. Stitch supported over 25,000 participants through those 90,000-plus scheduled visits, with more than 87% of participants using the platform and completing over 17,000 feedback surveys. The two organizations ran a joint analysis to isolate Stitch’s contribution to screening attendance, controlling across protocols and therapeutic areas before reporting that 9-point no-show reduction. For sponsors managing fixed enrollment windows, fewer missed screenings means fewer replacement visits to schedule and fewer protocol deviations to explain.
The expansion does more than widen the deployment footprint. Velocity is bringing its operational data and site expertise into joint product development with Stitch, with both parties planning to test new approaches to retention and protocol compliance across the full network and publish the results. That arrangement gives Stitch something most clinical-tech vendors lack at this stage: a single, accountable site organization running [more than 70 wholly owned sites across four countries](https://velocityclinical.com/beyond-the-us-how-velocity-is-redefining-clinical-trials-internationally/), with centralized operations that can run controlled comparisons at scale rather than aggregating messy data from dozens of independent sites with different workflows.
For site networks watching this, the meaningful detail is not the partnership itself but what Velocity intends to do with the evidence it generates. Publishing performance data from a 70-plus-site network, with methodology transparent enough to show a 9-point no-show reduction, sets a bar that anecdotal engagement metrics from smaller deployments cannot easily match. Whether that published evidence changes sponsor procurement conversations around site selection is the thing worth watching.
*Source link: *
**Categories:** News
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### [Phase 3 study shows aQIVc superior to comparators in older adults](https://www.clinicaltrialvanguard.com/news/phase-3-study-shows-aqivc-superior-to-comparators-in-older-adults/)
**Published:** September 11, 2026
**Author:** Jon Napitupulu
**Excerpt:** Phase 3 study shows aQIVc demonstrated superior immunogenicity against influenza strains in adults 50 and older, with responses sustained through the season.
**Content:**
Combining three enhancement strategies into one shot sounds straightforward, but nobody had run a large-scale trial to prove the combination works better than its individual parts. Data published in *The Lancet Infectious Diseases* now fill that gap: a 7,699-person Phase 3 study of CSL Seqirus’s [MF59-adjuvanted, cell-based, higher-dose quadrivalent vaccine (aQIVc)](https://www.prnewswire.com/news-releases/lancet-publication-marks-important-advance-in-influenza-prevention-for-older-adults-302875820.html) showed superior immunogenicity against all four influenza strains versus an adjuvanted egg-based comparator, and non-inferiority against a recombinant vaccine for three of four strains in adults 50 and older, and all four strains in those 65 and older.
The three-component approach is the real clinical question here. Cell-based manufacturing has been used to improve strain match since [Flucelvax’s 2012 approval](https://www.fda.gov/media/153436/download); MF59 adjuvant has a long track record in older adults; and higher antigen doses have been available since Sanofi’s [Fluzone High-Dose was licensed in 2009](https://www.cidrap.umn.edu/influenza-vaccines/fda-licenses-high-dose-flu-vaccine-elderly). What the Lancet study establishes is that stacking all three in a single formulation produces immune responses that held through the full influenza season, with most adverse reactions mild to moderate and resolving within three days. That persistence matters for older adults specifically, because immune senescence blunts and shortens vaccine-induced responses.
The study also reported exploratory data on neuraminidase antibody responses, which were higher for aQIVc than for both comparators. Neuraminidase immunity is not yet a regulatory endpoint, and the authors frame it as warranting further evaluation rather than a proven efficacy signal, but it gives immunisation advisory groups another variable to weigh when comparing enhanced vaccine options for older populations.
AUJEMFLU, the marketed name for aQIVc’s trivalent formulation, has already cleared the MHRA for adults 50 and older in the UK and received European Commission approval. CSL Seqirus has filed in additional markets and plans a phased rollout tied to local regulatory and policy timelines. The immediate signal to watch is whether National Immunisation Technical Advisory Groups in those pending markets cite the Lancet data when setting their enhanced-vaccine recommendations for the 2027 season.
*Source link: *
**Categories:** News
---
### [FDA Clears Prelude's PRT13722 Phase 1 Trial in HR-Positive Breast Cancer](https://www.clinicaltrialvanguard.com/news/fda-clears-preludes-prt13722-phase-1-trial-in-hr-positive-breast-cancer/)
**Published:** September 11, 2026
**Author:** Jon Napitupulu
**Excerpt:** FDA clears Prelude's PRT13722 Phase 1 trial in HR-positive breast cancer, advancing the oral KAT6A degrader toward patient enrollment.
**Content:**
The FDA cleared Prelude Therapeutics this week to open a Phase 1 trial for PRT13722, its oral KAT6A degrader in HR-positive breast cancer, a signal that the program has cleared its first regulatory gate and can now enroll patients. The IND clearance is the narrowest possible green light, not efficacy data, but it puts PRT13722 into a space where the treatment options are actively shifting: AstraZeneca’s camizestrant (Etcamah) [received accelerated FDA approval just three days earlier](https://news.cision.com/astrazeneca/r/etcamah-approved-in-the-us-for-hr--breast-cancer,c4392496), on September 7, for HR-positive, HER2-negative metastatic breast cancer with ESR1 mutations in patients already on aromatase inhibitor and CDK4/6 inhibitor therapy.
PRT13722 works by recruiting the ubiquitin-proteasome system to degrade KAT6A, a histone acetyltransferase that regulates gene transcription in hormone-driven tumor cells. [Published preclinical work on KAT6A degraders](https://aacrjournals.org/cancerres/article/86/7_Supplement/7076/777077/Abstract-7076-Discovery-of-a-highly-potent-and) has shown potent target engagement at sub-nanomolar concentrations, which gives the class a mechanistic rationale, though whether that translates to clinical activity in patients is exactly what the Phase 1 is designed to test. Prelude’s bet is that targeting a transcriptional regulator rather than the estrogen receptor directly offers a different angle on the tumor’s hormone-driven machinery.
Prelude has run this playbook before with PRT3789, its SMARCA2 degrader, where the company moved from IND clearance through proof-of-concept data showing anti-tumor activity in SMARCA4-deficient solid tumors. The SMARCA2 program established that Prelude can take a protein degrader through early clinical work, which gives PRT13722 some organizational precedent to lean on. The company built its identity around targeted protein degradation, and PRT13722 is its attempt to extend that approach into a far larger patient population than the SMARCA4-deficient niche.
What to watch now is dose escalation pace and any early pharmacodynamic readouts confirming that PRT13722 actually degrades KAT6A in patient tumor tissue. A degrader that hits its target in a cell line but fails to achieve sufficient exposure in vivo is where many programs in this class have stalled. The Phase 1 design, including the biomarker sampling strategy, will say a lot about how Prelude plans to answer that question.
*Source link: *
**Categories:** News
---
### [The Validation Accords: Clinical Trials Are About to Learn What "Validated AI" Actually Means](https://www.clinicaltrialvanguard.com/article/trend-watch/the-validation-accords-clinical-trials-are-about-to-learn-what-validated-ai-actually-means/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Nature Medicine's Validation Accords push for a generative AI consensus framework, and expose a regulatory gap that could invalidate trial data if sponsors…
**Content:**
Three convergent pressures have been building in clinical trial operations for the past eighteen months, and the pattern has not yet received a widely recognized name. Call it the Validation Gap: generative AI tools are accelerating protocol design, patient matching, and endpoint analysis faster than any existing regulatory framework can certify them, and the [Validation Accords published in *Nature Medicine*](https://www.nature.com/articles/s41591-026-04647-5) represent a serious attempt to close that gap before it closes sponsors’ approval pathways instead.
The stakes are not theoretical. When generative AI outputs touch trial data, whether by drafting inclusion criteria, summarizing adverse event narratives, or flagging protocol deviations in real time, those outputs become part of the evidentiary record a reviewer will scrutinize. Right now, no broadly accepted consensus standard yet exists to tell that reviewer whether the model producing those outputs was ever properly validated. That absence does not create ambiguity. It creates vulnerability.
The industry has not named this problem because it has been comfortable treating AI validation as a technology procurement question rather than a regulatory compliance question. That comfort is running out.
## [](#what-the-accords-actually-propose)What the Accords Actually Propose
The Validation Accords framework, as laid out in *Nature Medicine*, calls for a structured, multi-stakeholder consensus process to define what validation of generative AI systems looks like across the clinical evidence lifecycle. The framing matters: this covers not just diagnostic AI or imaging algorithms, the categories regulators have spent the most time on, but generative models that produce text, synthesize data, or make probabilistic recommendations that a human then acts upon. That scope captures nearly every commercial AI tool currently being sold into clinical operations.
Sponsors who have been treating AI vendor certification as a substitute for their own validation work will find that third domain uncomfortable. A model validated on electronic health record data from academic medical centers does not automatically perform equivalently when deployed on eCOA data captured through a decentralized trial app at community sites.
That gap between vendor-claimed performance and site-level deployment reality is where data integrity risk lives.
## [](#two-regulatory-signals-that-make-this-urgent)Two Regulatory Signals That Make This Urgent
The Accords do not arrive in a vacuum. Consider where the FDA and EMA stood heading into this year. In [January 2025, the FDA issued its draft guidance](https://www.federalregister.gov/documents/2025/01/07/2024-31542/considerations-for-the-use-of-artificial-intelligence-to-support-regulatory-decision-making-for-drug), *Considerations for the Use of Artificial Intelligence to Support Regulatory Decision Making for Drug and Biological Products*, which, according to [the guidance summary published by TrialX](https://trialx.com/what-does-the-fda-say-about-the-use-of-ai-in-clinical-trials-a-summary/), explicitly requires sponsors to document the intended use, known limitations, and performance characteristics of any AI system that supports a regulatory submission. The guidance does not specify generative AI by name, but the plain reading covers it: if a large language model summarized your safety narratives or helped structure your statistical analysis plan, that model’s performance is now a submission artifact.
That guidance was draft. It was not binding. Sponsors read it as advisory and moved on. But the gap between “draft guidance” and “inspection finding” is shorter than most CTMs appreciate, particularly in the current inspection climate.
The EMA moved from a different angle. Its [Guiding Principles of Good AI Practice in Drug Development](https://www.ema.europa.eu/en/documents/other/guiding-principles-good-ai-practice-drug-development_en.pdf) articulates ten principles covering AI use across the full medicines lifecycle, from evidence generation through post-market monitoring. Principle four focuses explicitly on transparency: developers and users of AI must be able to explain, in terms a regulator can evaluate, how a model produces its outputs and where its confidence boundaries lie. For generative models, which produce outputs probabilistically rather than deterministically, that explanation requirement is not a box to check. It is a fundamental architectural constraint that most off-the-shelf tools do not yet satisfy.
Two major regulatory bodies, operating independently, converged on the same underlying requirement: you must be able to explain and document AI performance in terms a reviewer can verify. The Validation Accords propose the shared language that makes that documentation possible across jurisdictions.
## [](#the-counterintuitive-risk)The Counterintuitive Risk
Here is the assumption almost every clinical operations leader currently holds: AI validation risk is a post-approval problem. Regulators will develop standards, industry will adapt, and the trials running today are grandfathered into a more permissive interpretive window. That assumption inverts the actual exposure.
Trials running today with unvalidated generative AI tools are building an evidentiary record that will be submitted two, three, or four years from now, directly into a regulatory environment shaped by whatever emerges from the Validation Accords process and the FDA’s finalized AI guidance. A Phase II oncology trial that used a generative AI model to assist with response assessment categorization in 2025 will submit that data into a 2028 review environment where the absence of a validation package for that model may constitute a material gap, not a historical curiosity. Retroactive validation of a model that has already influenced data is, at best, operationally painful. At worst, it is impossible.
The sponsors building validation infrastructure now, before the Accords are finalized, are not being cautious. They are being strategic. They will have documentation packages ready to map against whatever consensus standard emerges, while competitors scramble to reconstruct what their AI tools were actually doing in trials that closed eighteen months earlier.
That logic applies differently across the operational ecosystem. For CROs, AI validation is quickly becoming a differentiating capability rather than a back-office concern: the CROs that can offer sponsors pre-validated, deployment-tested AI modules will command premium contract positions as regulatory scrutiny increases. For technology vendors selling into the clinical space, the Accords’ call for multi-stakeholder collaboration is a direct invitation to shape the standards their products will be measured against, an invitation that carries a short expiration date. For sites, particularly community sites participating in decentralized trials, the deployment integrity domain of the Accords raises a practical question no one has answered yet: who is responsible for validating that a sponsor’s AI tool performs equivalently at a site with different EHR infrastructure, different patient population characteristics, and different staff AI literacy?
## [](#what-breaks-first)What Breaks First
Within the next twelve to eighteen months, the most immediate break point is protocol-embedded AI in adaptive trials. Sponsors running response-adaptive randomization or AI-driven interim analysis are relying on model outputs to make real-time trial modifications. If those models lack validation documentation that satisfies the FDA’s January 2025 draft guidance framework, even as a draft standard, a Complete Response Letter citing AI documentation gaps becomes a plausible outcome, not a remote risk. The first CRL issued on that basis will redefine what “AI-ready” means for every IND submission behind it.
The Validation Accords are a call for collaborators precisely because the authors recognize that no single institution can build this consensus alone. But the collaboration window closes the moment the FDA finalizes its AI guidance, a finalization that the current pace of AI deployment in clinical trials makes increasingly urgent. Sponsors who treat the Accords as an academic exercise will be submitting trial data against standards they had no hand in writing.
## [](#references)References
1. [Nature Medicine, “Consensus framework for the validation of generative AI: call for collaborators on the Validation Accords”](https://www.nature.com/articles/s41591-026-04647-5)
2. [TrialX, “What Does the FDA Say About the Use of AI in Clinical Trials? A Summary” (FDA Draft Guidance: Considerations for the Use of Artificial Intelligence to Support Regulatory Decision Making for Drug and Biological Products, January 2025)](https://trialx.com/what-does-the-fda-say-about-the-use-of-ai-in-clinical-trials-a-summary/)
3. [European Medicines Agency, “Guiding Principles of Good AI Practice in Drug Development”](https://www.ema.europa.eu/en/documents/other/guiding-principles-good-ai-practice-drug-development_en.pdf)
**Categories:** Trend Watch
**Tags:** AI Validation, Clinical Trial Integrity, FDA Guidance, FDA Regulatory Strategy, Generative AI
---
### [The Data Integrity Crisis That Built a Company: Rick Graham on Why Clinical Trials Keep Failing Before the AI Even Starts](https://www.clinicaltrialvanguard.com/executiveinterviews/the-data-integrity-crisis-that-built-a-company-rick-graham-on-why-clinical-trials-keep-failing-before-the-ai-even-starts/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** TruTechnologies co-founder Rick Graham argues that the industry's real execution problem is not a modeling problem.
**Content:**
 Richard Graham
TruTechnologies *TruTechnologies co-founder Rick Graham argues that the industry’s real execution problem is not a modeling problem. It is a data collection problem. And he has the phase three results to prove it.*
---
The clinical trial industry has spent a decade debating how to modernize execution: risk-based monitoring, decentralized protocols, AI-assisted signal detection. Rick Graham has watched that debate from an unusual vantage point. As a clinical pharmacologist who spent more than 20 years inside drug companies, he did not theorize about sample integrity failures. He cleaned them up. After a phase three trial produced drug detections in patients who had never received a dose, Graham concluded that the infrastructure problem was upstream of every tool the industry was reaching for. In 2018, he co-founded TruTechnologies with Scott Ogle, a financial-services software veteran, to address the failure point directly: the moment of sample collection at the clinical trial site. What follows is a conversation about where that conviction came from, what it looks like in practice, and why Graham is skeptical that ICH E6 R3 will move the needle.
### [](#why-did-three-specific-failures-across-your-career-convince-you-the-infrastructure-itself-had-to-change)Why did three specific failures across your career convince you the infrastructure itself had to change?
**Richard Graham:** I’m trained as a clinical pharmacologist. The discipline covers two things: pharmacokinetics, how the body processes and eliminates a drug, and biomarkers, which are proteins or patterns of gene expression that shift with treatment or disease. When you pick up a medicine at the pharmacy, the package insert includes information telling you what dose to take, when to take it, what other drugs to avoid. That comes from the work of a clinical pharmacologist. We rely heavily on biological samples to get the right dose to the right patient, for both safety and efficacy.
The first time I saw something that genuinely stopped me was during a phase 2 oncology trial. Cancer patients were coming in every eight weeks for a pharmacokinetic blood draw. When we started analyzing the data over time, the results within individual patients didn’t make scientific sense. The values were all over the place from one visit to the next. When we went back to the sites to figure out what happened, we found that the sites had sheets of pre-printed sticky labels and were not associating specific labels with specific patients and visits. They were just putting an “eight-week sample” label on whatever tube was in front of them. All the samples were getting mixed.
Once we found it, a research associate spent roughly eight hours a day for at least three months trying to reconstruct what actually happened in that study. At the end of that effort, I still had maybe 70% confidence that the data was correct. That was hard to accept.
At the same company, we had two drugs under FDA review, one a biologics license application and one a new drug application. During review, the FDA came back and said there was a major disconnect between what the clinical study report said about the biological samples and what the electronic records for those samples showed. The root cause was inaccurate data capture at the point of collection. What got recorded didn’t match what actually happened to the samples. Same underlying issue.
Then, at a different company, a drug was detected in blood during a phase 3 trial from patients who had never received a dose. The FDA reviewer concluded during the application review that the pharmacokinetic data integrity was compromised. That was all I could take. I went to Scott Ogle, who had never been in this industry before, but whom I knew had the technical expertise to help me solve the problem. I explained the situation and he did not believe me at first. He said, “You’re supposed to be curing diseases and making patients’ lives better. How is that possible?” That was around 2018, and that’s when we started the company.
---
### [](#how-did-you-decide-to-combine-live-data-capture-with-protocol-guided-workflows-in-trulab-rather-than-tackle-the-data-latency-problem-alone)How did you decide to combine live data capture with protocol-guided workflows in TruLab rather than tackle the data latency problem alone?
**Richard Graham:** When we started the company eight years ago, we set out to solve the biological sample problem I had seen across my entire career. And to be fair, we did try to solve both the quality issue and the speed issue from the beginning. Data latency was one part of the problem, but I was really concerned about quality.
What we learned over time, and it took a couple of years, is that once we built a technology for contemporaneous source data capture at the clinical trial site, the majority of errors we were seeing happened at the site during sample collection. And the reason comes down to how trials are run. A sponsor writes a protocol, sometimes over 100 pages, that tells a site how to execute the study. Then there’s a lab manual, also potentially 100 pages, that explains how to process the biological samples. You might have 50 sites around the world, and each of them is sending their team into a patient visit armed with their own interpretation of how to conduct a study visit based upon those two documents.
When we saw that, we realized we could fix it. We have a mobile platform, essentially running on an iPhone or Android device, that can be in the hands of the research coordinator running that trial and give them step-by-step instructions through a digital workflow. So it was never really a sequence of solving one problem and then the other. It was, let’s solve quality and speed, and then we hit an insight: we can actually go further and make the site’s job easier by giving them harmonized, step-by-step guidance across all the sites.
> “What we learned over time, and it took a couple of years, is that once we built a technology for contemporaneous source data capture at the clinical trial site, the majority of errors we were seeing happened at the site during sample collection. And the reason comes down to how trials are run.”
---
### [](#how-did-one-of-those-25-randomization-risk-interventions-actually-play-out-in-the-rare-disease-phase-three-study)How did one of those 25 randomization risk interventions actually play out in the rare disease phase three study?
**Richard Graham:** In that particular phase 3 study, the primary statistical endpoint was a protein measured in urine. The level of that biomarker was expected to decrease over time if the drug was working, because elevation of the biomarker is associated with disease. So the baseline biomarker level during screening was critically important. If a patient has the disease but their biomarker is already very low at baseline, there’s no room for it to fall and you can’t show a treatment effect in that patient.
To get randomized, patients had to complete a screening period of around 14 days. During that window, each patient needed to provide two separate 24-hour urine collections at home. They’d come into the site for a blood draw and to answer eligibility questions, then take two collection containers home and collect every urine sample over a 24-hour period, with timing notes. Then they’d come back, the site would aliquot those samples into tubes, ship them to a central lab, and wait for the results. All of that had to happen within the 14-day screening window. It’s a lot to coordinate.
What our technology showed us, in real time, was that one site had not asked the patient to bring back a second sample, and had not provided instructions for the patient to record when they collected. Our system flagged that automatically through pattern recognition, we had a human review it, confirmed it was a problem, and then escalated it to the CRO and the sponsor. They were able to call the site, course-correct, and the patient remained eligible for the study.
The other 24 risks looked different. Protocol non-compliance at a site, insufficient patient instructions during screening, discrepancies between what the protocol said and what the lab manual said. And sometimes a sample would arrive at the central lab and get held because someone couldn’t read the handwriting on the label. That kind of thing would just sit until someone manually went in and resolved it. For the first time, people can see those things instantaneously and course-correct.
---
### [](#why-does-trulabs-ai-work-where-the-fdas-real-time-initiative-risks-falling-short)Why does TruLab’s AI work where the FDA’s real-time initiative risks falling short?
**Richard Graham:** I want to set the stage here, because I have made remarks along these lines publicly and I want to be clear about what I’m actually talking about.
The FDA put out an initiative called the FDA’s Real-Time Clinical Trial Initiative and I’ve been very open regarding my point of view on it and where I think we can improve its aims. Directionally, the idea of having the FDA reviewing information sooner so we can get drugs to patients faster is a good one. But here’s where I think it misses the mark. The concept is that an AI system is fed data while a trial is ongoing, and with a relatively small number of patients and data points, that system flags safety or efficacy signals that then go to the FDA. My concern is what I’d call “garbage in, garbage out”. Anybody can build a model. And what I worry about in that FDA initiative is using AI to identify a signal on a small and potentially inaccurate data set.
Now, our platform uses AI, and we did use it in the randomization example we just discussed. But the important distinction is that our primary focus is not AI. Our primary focus is collecting source data contemporaneously at the site where mistakes actually happen, so that we prevent mistakes in the first place rather than detect them later. We’re not asking people to write on paper and then enter it into an electronic data capture system after the fact, with all the back-and-forth queries that may never fully get resolved. We’re capturing high-quality data right at the source.
So there will always be AI model limitations, including on our platform. But the contrast I want people to seriously think about is the difference between running AI models on incomplete and inaccurate data versus running them on accurate, high-quality data. AI is a bright, shiny object right now. I use it a lot myself. But I’m trained as a modeler, and I know the accuracy of underlying data is way more important than the model you build on top of it.
---
### [](#why-hasnt-ich-e6-r3-changed-sponsor-behavior-the-way-its-advocates-expected)Why hasn’t ICH E6 R3 changed sponsor behavior the way its advocates expected?
**Richard Graham:** I’ll start with a skeptical view, and you can challenge me from there.
The prior guidance, ICH E6 R2, already allowed risk-based monitoring. That was from 2016, I believe. And there’s been underwhelming adoption. There was a report from Tufts CSDD in 2024 that found adoption of risk-based quality management was slow, with companies implementing it roughly 50% of the time on average. You’d want to check the source for the exact numbers. So for me, if this approach didn’t get meaningful traction under the prior guidance, what in R3 specifically is going to drive a different result?
Here’s the broader problem. The way we run clinical trials has been in place for a really long time and hasn’t had meaningful change. And there are many stakeholders required to execute a trial: patients, clinical trial sites, CROs, central labs, tech vendors. Each of those stakeholders has different incentives. Take risk-based monitoring, which means you’re not required to have a CRO monitor go to the site and source data verify 100% of the data. You shouldn’t do it. It’s not a good use of time. But that monitor is paid to spend more time doing it. So the incentive to monitor less just isn’t there.
Given the 10-year history under the prior guidance, where it didn’t lead to meaningful change, I personally don’t see significant change in 3 to 5 years. Change in this industry doesn’t happen without accountability. You need a carrot or you need a stick, and I don’t know what either of those is with this particular guidance.
Let me also say that TruTechnologies is in the business of issue prevention. That’s very different from what everybody is used to. This guidance is framed around the way things have been done for 60 years. Risk-based monitoring finds problems weeks later after something has already been entered incorrectly in the EDC. It’s far more important to prevent the errors from occurring in the first place.
Here’s an analogy. Think about a city building code that says buildings should not fall down. Amongst other things, this guidance says studies should be conducted under good clinical practice and should generate reliable results. The analogy is buildings should not fall down. But it never specifies exactly how you prevent the building from falling down. So every builder reads that code and says, I’ve been building buildings for 60 years and they haven’t fallen down. They assume they’re fine. Those buildings stay upright until there’s a catastrophic event. I think the same thing happens here. People will read R3 and say, yes, I’m following good clinical practice and generating reliable results because that’s what I’ve always done. I don’t personally see significant change in 3 to 5 years.
> “Change in this industry doesn’t happen unless there’s accountability. You need a carrot or you need a stick, and I don’t know what that is with this particular guidance.”
---
### [](#why-does-recovering-those-two-months-of-enrollment-time-matter-beyond-the-operational-win)Why does recovering those two months of enrollment time matter beyond the operational win?
**Richard Graham:** Part of what led me to start a technology company, while still doing drug development, was ethics. I was trained by someone who worked in the clinical trials office at Stanford’s cancer center before he moved into drug development. He always told me: no matter what data you need from a trial, think about the patient sitting in the chair in the clinic. They’re sick. They’re taking time away from their jobs and their families. Don’t design your trial in a way that makes that patient’s life worse.
The patients in this study wanted to be there for two reasons. One, they wanted to help advance science and medicine. That’s part of being in a clinical trial. The other is they had a chance to benefit from an experimental therapy. If those patients had been turned away because a site missed an inclusion or exclusion criterion by mistake, when they would have otherwise been eligible, that’s an ethical problem. They would have lost an opportunity to benefit from a therapy they might have been helped by. So I’m really proud of what our company was able to contribute here for that reason.
But there’s a broader picture too. By expediting study completion we are consistently helping sponsors bring their timelines in. Think about what that means for patients who are not part of the clinical trial. They’re going to have access to a life-changing (and sometimes life-saving) therapy sooner than they would have otherwise, because it reaches the market faster. And then there’s the economic perspective. Tufts CSDD has done good work on this. On average, they estimate the cost of a delayed drug coming to market at around $500,000 per day. So there’s the patient benefit and the ethical dimension, and there’s also a significant financial benefit.
*Richard Graham is co-founder and chairman of the board of directors at TruTechnologies.*
---
**Categories:** Article: Executive Interviews
---
### [Women's Hearts, Underserved by Design: Anteris Technologies President David St. Denis on Closing Structural Heart's Most Overlooked Gap](https://www.clinicaltrialvanguard.com/executiveinterviews/womens-hearts-underserved-by-design-anteris-technologies-president-david-st-denis-on-closing-structural-hearts-most-overlooked-gap/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** The problem isn't that fewer women get aortic stenosis. It's that the entire pipeline, from diagnosis to device, was built without them in mind.
**Content:**
 David
Anteris Technologies *Women do not have less heart disease than men. They have less representation in the trials, imaging protocols, and device designs that determine who gets treated and how well. In structural heart specifically, that gap has compounded quietly for decades: women present differently in clinic, their disease looks different on imaging, and the devices implanted to save their lives were largely developed without their physiology as a reference point. David St. Denis, President & Director at Anteris Technologies, is one of the few device executives willing to name this problem at each link in the chain and describe what fixing it actually requires. Anteris is currently advancing DurAVR, a next-generation aortic valve replacement designed to restore healthy physiology, including in patients with smaller anatomies and late-stage disease. In this conversation, St. Denis walks through why detection fails women first, where the device industry’s responsibility begins, and what the clinical evidence base still cannot tell us.*
---
### [](#why-are-women-underrepresented-in-structural-heart-trials-and-treatment-even-when-the-disease-burden-is-comparable)Why are women underrepresented in structural heart trials and treatment, even when the disease burden is comparable?
**David:** It’s multifactorial. A lot of it has to do with how men and women present in clinic differently. And because of those differences, there’s a bias toward recognizing the disease in men more frequently and referring them to treatment more frequently. It doesn’t mean women have the disease more often or more severely. It just means the system as it’s set up right now is biased toward detecting it in men rather than women.
---
### [](#how-does-that-diagnostic-bias-play-out-in-a-disease-like-aortic-stenosis-specifically)How does that diagnostic bias play out in a disease like aortic stenosis specifically?
**David:** When a man comes in, he’ll say he has chest pain, he’ll be passing out, he’ll have shortness of breath. These are all very clear markers of the disease. And phenotypically, men tend to have more calcific disease, so when they go for assessment, the imaging modalities we use pick up calcification and they’re diagnosed.
Women tend to express their symptoms slightly differently. The way those symptoms get expressed very often ends up being associated more with just the consequence of normal aging. You can kind of miss it: “You’re just slowing down, you’re just getting a little tired,” because they’re not presenting the very specific symptoms doctors are looking for today. Then if they are caught and they do go into the diagnosis phase, their disease tends to be more fibrotic rather than calcific. And fibrosis tends to show up less on the imaging modalities we use. So there are multiple points in that process where women can fall through the cracks.
> “We need to be educating patients, first and foremost, specifically women about this disease and how to advocate for themselves in the clinic, how to describe their symptoms, how not to diminish their symptoms by just saying, ‘I’m just getting older, I’m just slowing down.’”
---
### [](#how-does-a-device-companys-responsibility-extend-into-a-diagnostic-pipeline-it-doesnt-control)How does a device company’s responsibility extend into a diagnostic pipeline it doesn’t control?
**David:** The medical device industry has the solution to the disease, but we’re not the ones diagnosing it. That said, I think our responsibility starts with patient education. If women are more educated and advocating for themselves in the clinic, they may be intervened with earlier and they may have a better long-term outcome. That’s the first consequence.
Beyond that, we need to be working with our partners in the imaging space to say: how can we try to pick up this disease for both men and women? Can we change some of our algorithms so we’re more likely to catch it? And yes, it does involve education for healthcare providers, working with key opinion leaders, making sure the right voices are represented. Health systems, doctors, manufacturers in med device and diagnostics all have a role. But the biggest role we all have to play is educating patients to know how to advocate for themselves.
---
### [](#how-does-device-design-itself-need-to-change-and-who-gets-left-out-when-its-designed-universally)How does device design itself need to change, and who gets left out when it’s designed universally?
**David:** As we’ve developed these devices, we’ve developed them not with the physiology of a woman in mind. And it’s not just women, by the way. It’s also certain populations of men who happen to have smaller anatomies. What we’re starting to do now is say: we can’t look at this universally by an entire group. We have to look at each patient’s phenotype, their anatomy, and understand fundamentally what we have to do differently in device development to adapt to the needs of that patient.
A woman, or a man in this case with a very small anatomy and a low-flow, high-gradient presentation, requires a device that functions differently than somebody else who needs something different. And I think you’re starting to see that contemplated in the new devices coming through.
---
### [](#why-do-clinical-trials-have-to-change-before-device-design-can-fully-catch-up)Why do clinical trials have to change before device design can fully catch up?
**David:** The first thing we need to do is have clinical trials that include more women. The outcomes data we have today is based on blended populations. And as we know from areas like cancer and immunotherapy, there can be far more than surface-level differences between men and women. The more trials we conduct that include larger female populations, the more data we generate, and the more may appear to tell us: actually, we need to be designing things specifically for this population versus that one. We don’t have that data today, so everything gets blended in.
At least the industry I’m in now has really started to pay attention to this. Companies are designing trials to include women more thoughtfully and more deliberately. Once we generate a compendium of clinical evidence, we’ll have clues as to where to focus next.
> “The more trials we do that include larger populations of women, the more data we gather, the more may appear to us to say, ‘Actually, we need to be designing things specifically for this population versus that population.’ We don’t have that data today.”
---
### [](#how-does-late-stage-detection-change-what-the-device-itself-needs-to-do)How does late-stage detection change what the device itself needs to do?
**David:** If unfortunately women slip through the cracks, as is often the case, and they are detected at all, it’s often very, very late. That’s where next-generation therapies like Anteris’s DurAVR come in, because we’ve designed the product to restore healthy physiology. In a patient who’s in very late-stage disease, while we may not be able to return them completely to health, we’re going to give them the best chance at stopping the progression of disease and, hopefully, returning them to some level of health so they can be active again, have a normal quality of life, and hopefully many more years of life. We’ll see that as the data reads out. But that’s really the key focus.
*David St. Denis is President & Director at Anteris Technologies, a structural heart company developing next-generation aortic valve replacement therapies.*
---
**Categories:** Article: Executive Interviews
---
### [PSI CRO to Present Agile Models for Emerging Biotech at OCT New England](https://www.clinicaltrialvanguard.com/news/psi-cro-to-present-agile-models-for-emerging-biotech-at-oct-new-england/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** PSI CRO presents agile models emerging biotech sponsors can use to scale global trials while maintaining operational control at OCT New England.
**Content:**
Ninety-three percent repeat business and 15% revenue growth in a single year puts [PSI CRO](https://psi-cro.com/newsroom/) in an unusual position for a mid-size CRO: sponsors keep coming back, which suggests the agility argument it plans to make at OCT New England next week is grounded in something operational rather than just a conference pitch.
On October 27 at Encore Boston Harbor in Boston, Benjamin Raccanova, PSI’s Head of Business Oversight, joins Stream C of the [18th Annual Outsourcing in Clinical Trials New England](https://intuitionlabs.ai/conferences/oct-new-england) conference for a peer-driven session on how small and emerging biotech sponsors can hold onto operational control while scaling global programs. The pressure point is real: investor timelines, regulatory scrutiny, and the site-level complexity introduced by the [FDA’s 2024 final guidance on decentralized clinical trials](https://www.federalregister.gov/documents/2024/09/18/2024-21078/conducting-clinical-trials-with-decentralized-elements-guidance-for-industry-investigators-and-other) all pull in different directions for a sponsor that doesn’t have a 500-person clinical operations team to absorb the friction.
The debate framing matters here. Raccanova’s panel asks directly whether large CRO operating models are equipped to serve emerging biotech, which sets up a structural question that PSI has a commercial interest in answering a particular way, but also has data to back up. In 2024, 93% of PSI’s studies enrolled on time or ahead of schedule. For a biotech sponsor burning cash against a runway, a slip in enrollment doesn’t just delay a readout, it often triggers a bridge financing conversation.
The session runs at 3:15 PM on October 27. Whether the peer discussion produces anything transferable beyond the room will depend on how many attending sponsors are willing to describe what broke in their last CRO relationship out loud. That candor, not the panel format itself, is what tends to make these conversations useful.
*Source link: *
**Categories:** News
---
### [Senn Framework Targets Protocol-Specific Content for Live Site Initiation Visits](https://www.clinicaltrialvanguard.com/news/senn-framework-targets-protocol-specific-content-for-live-site-initiation-visits/)
**Published:** September 10, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Live site initiation visits should focus on protocol-specific content that creates operational risk, per Christine Senn's framework for SIV training design.
**Content:**
Generic GCP slides do not cause protocol deviations. Misunderstood eligibility criteria and missed pharmacokinetic sampling windows do. That distinction is the entire argument of Christine Senn’s framework for what the live site initiation visit should actually contain, published by Advarra this week as Part 2 of a three-part series on SIV training design.
The practical problem is crowding. When a [live SIV](https://www.advarra.com/blog/what-actually-belongs-in-the-live-siv/) carries standard regulatory text, vendor screenshots, and broad documentation reminders alongside study-specific content, the specific content loses attention. Senn’s principle is simple: protect live time for material that varies by study, creates operational risk, or requires real-time discussion between the sponsor, CRO, and site. That means eligibility interpretation (not just what the criteria say, but where screen failures are likely and which questions need escalation before enrollment), investigational product handling specific to this protocol, disease-specific outcome measures that are time-sensitive or subjective, and lab or imaging workflows where a missed sample or rejected image creates missing data or a protocol deviation. Sponsor-specific safety thresholds and cohort governance rules belong there too, distinct from general adverse event reporting principles, which can be trained once and reused.
The oncology section makes the sharpest point. Oncology trials typically carry layered eligibility, biopsy requirements, central lab and imaging workflows, dose-escalation governance, and protocol-specific adverse events of special interest. The instinct is to build a longer SIV. Senn argues the opposite: complex studies need more focused live discussion, not more generic content on top. The [Advarra Site-Sponsor Consortium](https://www.appliedclinicaltrialsonline.com/view/advarra-launches-site-sponsor-consortium), which launched in February 2022 with more than 20 research sites, life sciences companies, and standards bodies, will release a white paper this fall on competency-based, role-based, and reusable training models aligned with international regulatory guidelines, including the [ICH E6(R3) framework](https://www.gmp-compliance.org/gmp-news/ema-publishes-consolidated-ich-e6r3-guideline), which supports proportionate, risk-based approaches to trial conduct. The SIV redesign Senn describes is one piece of that larger structure.
A four-question test she offers gives sponsors and CROs a practical filter before building slide decks: does this topic vary by protocol, would misunderstanding it create meaningful risk, does it require live discussion or role clarification, and can it be replaced by a reusable module? Content that clears any of those bars belongs in the SIV. Content that clears none of them consumes the attention that eligibility and dosing specifics actually need. The signal to watch is whether the fall white paper translates this content filter into a training-design standard that sponsors can apply at the protocol level.
*Source link: *
**Categories:** News
---
### [Seismic Pharmaceuticals Enrolls First Patient in Phase 3 Trial of Istaroxime for Cardiogenic Shock](https://www.clinicaltrialvanguard.com/news/seismic-pharmaceuticals-enrolls-first-patient-in-phase-3-trial-of-istaroxime-for-cardiogenic-shock/)
**Published:** September 10, 2026
**Author:** Jon Napitupulu
**Excerpt:** Seismic Pharmaceuticals enrolls first patient in Phase 3 trial of istaroxime for cardiogenic shock, the only late-stage program targeting this deadly condition.
**Content:**
Cardiogenic shock kills roughly half the patients it reaches in the ICU, a mortality burden that has persisted for decades without a single drug specifically approved to treat it. Seismic Pharmaceuticals enrolled the first patient in RESCUE HF-CS, its global Phase 3 trial of istaroxime, on September 9, 2026, setting up the only late-stage program currently designed to change that.
The trial targets approximately 600 patients across sites in the United States, Europe, China, and South America. It follows four completed Phase 2 studies in which more than 350 patients received istaroxime, including a Phase 2b extension study in which [cardiac output rose roughly 15% during infusion](https://synapse.patsnap.com/article/windtree-reports-positive-phase-2b-results-istaroxime-improves-cardiac-function-and-bp-in-early-cardiogenic-shock) with no significant increase in heart rate. That last point matters clinically: the inotropes physicians currently use off-label in cardiogenic shock tend to raise myocardial oxygen demand and increase arrhythmia risk, two problems istaroxime’s mechanism is designed to avoid.
Istaroxime works through two simultaneous actions: it inhibits Na/K ATPase, which increases the calcium available for each contraction, and it activates SERCA2a, the pump that clears calcium from the cell between beats. [Stimulating SERCA2a accelerates calcium cycling](https://pubmed.ncbi.nlm.nih.gov/23763364/) and improves diastolic relaxation, meaning the heart refills more completely before the next beat. Most inotropes address only the squeeze; istaroxime addresses both sides of the cardiac cycle. In the Phase 2b data, the drug also reduced ventricular arrhythmias in patients on continuous 72-hour ECG monitoring, a signal worth watching in Phase 3 where the trial is large enough to power a meaningful event count.
The size of the Phase 3 program relative to the Phase 2 experience (600 patients versus 350 across all prior studies combined) reflects how hard cardiogenic shock trials are to run: patients are critically ill, enrollment windows are narrow, and sites need intensive care infrastructure. [An ICU mortality rate of 44.8%](https://pubmed.ncbi.nlm.nih.gov/39302432/) in a large prospective analysis confirms how sick this population is and how little time investigators have to capture patients at the right point in their deterioration. The primary enrollment milestone to watch is whether Seismic can sustain site activation across four continents fast enough to keep that 600-patient target within a realistic timeframe.
*Source link: *
**Categories:** News
---
### [Tyra Selects 60mg Dose of Dabogratinib in NMIBC Phase 2 Study](https://www.clinicaltrialvanguard.com/news/tyra-selects-60mg-dose-of-dabogratinib-in-nmibc-phase-2-study/)
**Published:** September 10, 2026
**Author:** Jon Napitupulu
**Excerpt:** Tyra Biosciences selects 60mg dose of dabogratinib in NMIBC Phase 2 Study, advancing oral FGFR3 inhibitor development for bladder cancer treatment.
**Content:**
Sixty milligrams once daily: that is the dose Tyra Biosciences selected from SURF302 after the Phase 2 study returned its first results on September 9, and the choice carries real weight for a company trying to build an oral alternative to intravesical instillation in [FGFR3-altered low-grade intermediate-risk non-muscle invasive bladder cancer](https://www.urologytimes.com/view/dabogratinib-shows-early-activity-in-fgfr3-altered-lg-ir-nmibc). The trial had enrolled 44 participants as of the August 31 data cutoff, against a planned maximum of 90, meaning the dataset is still maturing, but Tyra read enough signal to call 60 mg the dose it will carry forward.
The announcement frames SURF302 as clinical proof of concept for selective oral FGFR3 inhibition. That framing matters because dabogratinib’s earlier work, the Phase 1/2 [SURF301 study](https://pubmed.ncbi.nlm.nih.gov/41084837/), focused on advanced metastatic urothelial carcinoma, a far sicker population. Moving into low-grade intermediate-risk NMIBC is a different strategic bet: these patients typically face repeated cystoscopies and intravesical treatments over years, not months, so the bar for a daily pill is tolerability as much as efficacy. A dose-selection read at 44 patients does not resolve that question, but it at least narrows the range before enrollment closes.
The competitive context is not simple. Erdafitinib (Balversa) holds FDA approval for FGFR-altered bladder cancer in a more advanced setting, and [approved options in the NMIBC space remain limited](https://www.urologytimes.com/view/dabogratinib-shows-early-activity-in-fgfr3-altered-lg-ir-nmibc), particularly for this lower-risk, non-muscle-invasive slice of the disease. Dabogratinib’s selectivity for FGFR3 over other FGFR family members is the differentiating design choice Tyra has emphasized across both trials, with the hypothesis that a tighter selectivity profile reduces off-target toxicity enough to make chronic oral dosing practical for patients who are not yet facing metastatic disease.
With enrollment still below half the target population, the number to watch is what the complete 90-patient cohort shows on endoscopic response: that readout will determine whether 60 mg once daily holds up as a registration-enabling dose or whether SURF302 becomes a learning study for a third trial.
*Source link: *
**Categories:** News
---
### [DYNAMIKA™: The Operating System for Imaging Clinical Trials](https://www.clinicaltrialvanguard.com/opinion/dynamika-the-operating-system-for-imaging-clinical-trials/)
**Published:** September 9, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Sponsored by Image Analysis Group. Why the imaging platform decision is the single most consequential infrastructure choice a sponsor makes, and how DYNAMIKA™ is built to prevent the failure modes that surface at interim.
**Content:**
Most platforms used in imaging clinical trials grew through acquisition of disparate tools, or started as specialized reader software later stretched to handle image collection. DYNAMIKA™ is not a merger of tools, and it's not a single-modality or single-purpose review system. It is the enterprise platform, designed and built from the ground up for imaging clinical trials – delivering regulatory-grade data and giving every trial stakeholder the workflow they need to succeed.
Six trials. Six imaging platforms. Six disconnected databases.
When a biotech's Chief Medical Officer asks which sites consistently underperform, or which imaging endpoints are generating the cleanest signal across the program, there is often no fast answer. The imaging trial data lives in separate silos: sites upload scans into a 'Dropbox-like' website, and the Quality Control team only reaches for these images once a month, by which point multiple baseline scans have already been acquired incorrectly. Radiologists, asked to receive data on their workstations and turn reads around within 24 hours, are frustrated, and the pressure leads to mistakes. Last year's Phase 2 reader discrepancy metrics are gone, buried in an Excel sheet no one can find. QC benchmarks, site feasibility scores, and performance data that took months to establish have to be rebuilt or awkwardly adapted for every new study using the same outdated tools. And choosing readers is always a headache, since no one can easily tell their calibration rates or availability.
This is not a hypothetical scenario. It is the default operating model for most biotech sponsors running concurrent imaging programs today. And it is why, when something goes wrong, whether in site performance, reader inconsistency, or image quality, the company tends to find out at the interim analysis, when the window to fix it has already closed.
By the time these problems surface, the cost is no longer measured in hours or Excel sheets. It shows up as protocol amendments, extended timelines, and data too inconsistent to support a confident go/no-go call. The irony is that none of this is a data problem. It is an infrastructure problem, one created by stitching together tools that were never designed to work as a single system in the first place.
## [](#the-imaging-clinical-trial-infrastructure-decision-critical-to-success-of-the-trial)The Imaging Clinical Trial Infrastructure Decision: Critical to Success of the Trial
Most sponsors treat the imaging platform decision the way they treat vendor selection for any other clinical service: availability, cost, whether someone else has used it before. In the modern world of quantitative endpoints, use of AI-powered workflows, reliance on exploratory endpoints to build competitive drug profile while saving costs and time, the platform for the trial is the single most important decision, one that should get priority review and C-level sign off.
The consequence of the decision is visible in the data. In oncology trials, we often see reader agreement rates collapse under independent adjudication. In obesity trials, DXA calibration gaps surface at the interim analysis. In long trials which are often needed for drugs targeting rheumatic or musculoskeletal diseases, scanner upgrades mid-study silently rewrite months of longitudinal data. None of these are science problems. They are clinical trial infrastructure problems, and they are entirely predictable, which means they are entirely preventable if the infrastructure was designed to prevent them.
Image Analysis Group (IAG) has spent almost 20 years and 700 global trials studying these failure modes. DYNAMIKA™ is the platform built to address them. It is not a radiology PACS system adapted for research, not a general-purpose EDC with an imaging module bolted on, and definitely not a Dropbox-style solution for image collection. It is a purpose-built operating system for the imaging layer of a drug development program – engineered on cloud-native infrastructure to run the full lifecycle, from site qualification and the first test image upload through final database lock. Along the way, it provides real-time quality control, orchestrated workflows for every trial stakeholder, a centralized performance dashboard, and a compliance architecture that never needs to be rebuilt from scratch for each new study. All read methods, all AI-workflows and API enablement for specific reads stay on, thus saving time and costs for study start up and reading tools.
The enterprise model is what truly sets DYNAMIKA™ apart. Many imaging platforms treat each trial as an isolated instance, so every new trial means standing up the data management system again. Sponsors can end up running dozens of near-identical instances, each configured slightly differently.
DYNAMIKA™ takes the enterprise-level approach: it consolidates all of a sponsor's studies, sites, and readers under one shared infrastructure. Site performance history from a previous program, reader calibration records, and QC rules that caught specific failure modes in a prior indication all carry forward automatically. By the third study, the platform already understands the sponsor's entire network.
“
Most sponsors don't realize they've been running their imaging programs in silos until they need to ask a question that spans more than one study. With DYNAMIKA™, that question has an answer. The site history, the reader calibration data, the QC benchmarks, are all in one place. By the third study with us, a sponsor's startup time is a fraction of what it was for the first.
Jon Himoff, Chief Product Officer
Image Analysis Group (IAG)
[](https://www.ia-grp.com/)
## [](#what-dynamika-covers)What DYNAMIKA™ Covers
DYNAMIKA™'s underlying infrastructure stays constant across every therapeutic area IAG supports: the same cloud-native architecture, the same real-time QC at upload, the same centralized adjudication workflow, and the same 21 CFR Part 11 audit trail. What changes is the indication-specific endpoint knowledge applied when images are read, whether by a radiologist or an AI model. This consistency matters more than ever, because modern trials for advanced therapies and complex designs increasingly rely on a mix of imaging modalities and read criteria within a single protocol. A cachexia trial, for example, needs DXA for bone mass alongside CT-based RECIST 1.1 for tumor assessment, while an obesity trial needs MRI-based muscle mass assessment paired with DXA-based bone health measurements.
The table below shows examples of FDA-required and exploratory imaging endpoints in these cross-modality settings, which DYNAMIKA™ supports across IAG's core therapeutic areas.
Oncology
FDA-Required / Established Endpoints
RECIST 1.1 tumor burden
Exploratory / Mechanistic
PET SUV scoring; PET-MRI anatomical fusion; radiomics and tumor volume measurements
Obesity / Metabolic
FDA-Required / Established Endpoints
DXA body composition (BMD, lean mass); fracture risk assessment
Exploratory / Mechanistic
Dixon/IDEAL MRI myosteatosis; visceral adipose tissue (VAT vs. SAT); hepatic fat quantification
Rheumatology / MSK
FDA-Required / Established Endpoints
Synovitis scoring; axSpA SPARCC scoring; K&L (OA); MOAKS and WORMS (knee OA)
Exploratory / Mechanistic
DCE-MRI synovial perfusion; bone marrow lesion quantification; FAPI-PET scoring for inflammation
Neurology
FDA-Required / Established Endpoints
RANO and RANO 2.0; area measurements; volumetric brain MRI; lesion load (MS)
Exploratory / Mechanistic
Assessment of pseudo-progression; perfusion MRI; functional connectivity
Key indication examples.
IAG also supports cardiac safety, cardiovascular imaging, radiopharmaceutical drug development, rare diseases, and additional therapeutic areas where imaging endpoints are protocol-specific and require configuration from the ground up. The breadth is a function of the platform architecture: because DYNAMIKA™ was built to be configurable at the protocol level, each new indication does not require rebuilding the underlying infrastructure.
## [](#one-platform-every-trial-every-site-every-reader)One Platform. Every Trial. Every Site. Every Reader.
Quality of imaging data will impact the quality of final efficacy. DYNAMIKA™’s instantaneous automated QC at image upload is a foundational capability, not a downstream check. When a site submits an image that deviates from protocol parameters (wrong echo time, mismatched voxel size, scanner model not on the approved list), DYNAMIKA™ flags it before it enters the dataset. The sponsor does not find out at the interim. They find out that morning.
The [IROC National Clinical Trial Network](https://www.rsna.org/uploadedFiles/RSNA/Content/Science/Quality/Storyboards/2016/Poon-QS018.pdf) demonstrated what active monitoring achieves in practice: overall protocol conformance across a multi-site oncology network increased from 73% to 85% over four years. PET uptake time conformance improved from 47% to 84%. Those gains were not produced by better protocols or more experienced sites. They were produced by the feedback loop. Sites that receive immediate QC results correct their behavior. Sites that receive results weeks later cannot.
The enterprise model compounds over time. Site activation, QC calibration, and reader performance history from a previous study carry forward to the next. A sponsor running their third program with IAG knows before enrollment opens which sites historically deliver conformant data and which readers demonstrate the tightest agreement rates on the specific endpoints the new protocol requires.
“
20 years in business and 700 trials give you something no algorithm can replicate: the ability to recognize when something looks wrong before the data confirms it. We've seen the failure modes. We've built the QC rules that prevent them. DYNAMIKA™ doesn't replace that knowledge, it scales it. We build the platform and positioned our team to enable near real-time QC. When a reader flags an image at 2am in Tokyo, a specialist in London reviews it the same morning.
Olga Kubassova, President & CEO
Image Analysis Group (IAG)
[](https://www.ia-grp.com/)
## [](#why-infrastructure-decisions-cannot-be-undone)Why Infrastructure Decisions Cannot Be Undone
The data integrity problems that DYNAMIKA™ is designed to prevent are documented in the peer-reviewed literature and in FDA records. Understanding them is useful, not as the lead of this story but as the evidence base for why the platform choices described above matter.
**On DXA:** when the same protocol runs across a multi-site network, [the inter-center CV for bone mineral density reaches 8.18%](https://pubmed.ncbi.nlm.nih.gov/33045389/), against intra-center precision of 1.40%. For lean mass the ratio is worse: a tenfold amplification of technical noise. The [ISCD cross-calibration mandate](https://iscd.org/wp-content/uploads/2024/03/2023-ISCD-Adult-Positions.pdf) requires formal cross-calibration within 60 days of any scanner replacement, or a new baseline must be established, nullifying every patient's longitudinal contribution to the primary endpoint.
**On scanner upgrades:** a Siemens hardware upgrade produced a [30% increase in neocortical contrast-to-noise ratio](https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2019.00726/full), with WMH volumes dropping 68%, indistinguishable from a drug effect in a neurodegeneration trial. [Cortical volume differences of 2-4%](https://pmc.ncbi.nlm.nih.gov/articles/PMC8491918/) are comparable to two years of atrophy. The [FDA's guidance on mid-study changes](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/changes-or-modifications-during-conduct-clinical-investigation-final-guidance-industry-and-cdrh) classifies major scanner upgrades as potential significant changes requiring IDE supplements.
**On audit trails**: during the review of [NDA 208772 for Brigatinib](https://accessdata.fda.gov/drugsatfda_docs/nda/2017/208772Orig1s000OtherR.pdf), the FDA's Office of Scientific Investigations stated it could not recommend data reliability from the contracted imaging vendor due to inadequate 21 CFR Part 11-compliant audit trails. The submission was jeopardized by the imaging infrastructure, not by the drug.
ONCOLOGY
31-42%
RECIST 1.1 double reads require a third independent radiologist for adjudication, which DYNAMIKA™'s centralized workflow is designed to minimize
Longdom Publishing RECIST adjudication | Sutter et al. Bone 2021 (PubMed 33045389) | PINTAD consortium PMC8259547
OBESITY / METABOLIC
>8%
inter-site CV for DXA BMD across multi-site networks, against intra-center precision of 1.40%
RHEUMATOLOGY
77%
imaging reader disagreements arise from image perception differences across 40 trials and 12,299 participants
[](https://www.ia-grp.com/)
## [](#human-expertise-scaled-by-technology)Human Expertise, Scaled by Technology
The conversation about AI in clinical trial imaging is generating more confusion than clarity. IAG's position is precise: machine intelligence supports the workflows that human experts run. QC flags that a human investigates. Documentation that a human reviews. Reader calibration data that a specialist interprets before it becomes a protocol intervention.
The value of 20 years of experience is not the volume of data. It is the institutional knowledge about what goes wrong and when. The failure modes that DYNAMIKA™’s rules are designed to prevent were not discovered by an algorithm. They were observed by specialists who reviewed the data, spoke with multiple expert radiologists and impacted approval of many drugs. This is the team behind DYNAMIKA™.
Sponsors working with IAG have helped shape the current regulatory standards for imaging endpoints. The ones defining the next standard, in GLP-1 programs, radiopharma trials, and the first wave of AI-augmented oncology studies, are doing it now.
[](https://www.ia-grp.com/)
## [](#the-operating-system-decision)The Operating System Decision
The operating system decision gets made once. It determines whether imaging data holds up at interim, whether the endpoint survives regulatory scrutiny, and whether the program can scale from Phase 2 to Phase 3 without rebuilding its infrastructure from scratch.
It is made at protocol design, before the first site is activated, before the first patient is scanned. The sponsors who make it well tend to be the ones presenting at the podium. The ones who don't tend to be the ones requesting an extension.
**Most platforms were built for something else entirely.** DYNAMIKA™ is here to ensure the success of imaging clinical trials.
## [](#about-image-analysis-group)About Image Analysis Group
Image Analysis Group (IAG) is a specialist imaging CRO which delivers end-to-end solutions to sponsors who use medical imaging to assess safety or efficacy of their new drug candidates. IAG build DYNAMIKA™, our cloud-native, GxP-validated, SOC 2 Type II and 21 CFR Part 11-compliant platform for clinical trial imaging. Single platform for end-to-end upload, QC and central review of imaging data in phase I-III trials, with embedded AI-powered workflows, FDA required endpoints, centralized image review and real-time trial oversight of trial progress and stakeholders’ performance. DYNAMIKA™ is helping Sponsors move from first scan to database lock faster, thus accelerating clinical trials and increases chances of drug development success.
Email:
Website: [www.ia-grp.com](http://www.ia-grp.com/) LinkedIn:
### [](#references)References
1. Sutter et al.: DXA intra-center CV 1.40% BMD / 0.76% lean mass; inter-center CV 8.18% BMD / 7.89% lean mass. Bone 2021.
2. ISCD 2023 Official Adult Positions: cross-calibration mandate, precision thresholds, LSC methodology.
3. PINTAD consortium (Borradaile et al.): 77% of reader disagreements from image perception; 40 trials, 12,299 participants. Ther Innov Regul Sci 2021.
4. Basty et al., UK Biobank n=32,961+: DXA overestimates lean mass; MRI detects 4-5% longitudinal muscle loss DXA misses. Commun Med 2026.
5. Beaumont et al., RECIST 1.1 BICR vs local: 85% different target lesions, 59% different baseline scans, 36.7% adjudication rate. Cancer Imaging 2018.
6. Backhausen et al., MRI scanner upgrade (Siemens Verio vs Skyra): cortical volume differences 2-4% across regions. PLoS One 2021.
7. Potvin et al., MRI system upgrade (Trio to Prismafit): 30% CNR increase, neocortical volumes 1.9-6.4% larger, WMH -68%. Front Neurol 2019.
8. Clarkson et al., ADNI phantom voxel scaling: 0.20% mean absolute volume change from scanner drift. Neuroimage 2009.
9. FDA Clinical Trial Imaging Endpoint Process Standards Guidance for Industry (2018).
10. FDA Guidance: Changes or Modifications During the Conduct of a Clinical Investigation.
11. Poon et al. (IROC), QC in multicenter clinical trials: conformance 73% to 85% over 4 years; PET uptake 47% to 84%. RSNA 2016.
12. FDA NDA 208772, Brigatinib (Alunbrig): OSI could not recommend data reliability from imaging vendor; inadequate 21 CFR Part 11 audit trails. [https://accessdata.fda.gov/drugsatfda\_docs/nda/2017/208772Orig1s000OtherR.pdf](https://accessdata.fda.gov/drugsatfda_docs/nda/2017/208772Orig1s000OtherR.pdf)
13. Trevisan et al., DXA: 1 cm hip placement shift = 4% BMD change; 3 cm scan window enlargement = 3% BMD increase. J Bone Miner Res 1992.
Sponsored Content
This article is sponsored by [**Image Analysis Group (IAG)**](https://www.ia-grp.com), a specialist imaging CRO. Contact: · [www.ia-grp.com](https://www.ia-grp.com) · [LinkedIn](https://www.linkedin.com/company/image-analysis-ltd/)
**Categories:** Article: Opinion
---
### [A 25% Sample Size Cut Sounds Like a Win, Until You See the Amendment Queue](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/a-25-sample-size-cut-sounds-like-a-win-until-you-see-the-amendment-queue/)
**Published:** September 9, 2026
**Author:** Krishma Shah
**Excerpt:** CONSONANCE's digitally enhanced MS endpoint cut required sample size 24.9%. Here's what that number costs in protocol amendments, IRB time, and eCOA validation…
**Content:**
A clinical operations lead gets the news from her biostatistics team: the new digitally enhanced composite endpoint just cut the required sample size by nearly 25%. She pulls up the enrollment projection, recalculates the per-patient budget, and starts thinking about which sites she can release early. Then her regulatory affairs counterpart sends a follow-up email: “We’ll need a protocol amendment before we can switch endpoint definitions. IRB submissions at all sites. eCOA validation package. DHT bridging data. Plan for six to nine months.” The projection goes back in the drawer.
That gap between the statistical win and the operational reality is exactly what the [CONSONANCE Phase 3b framework published in medRxiv](https://www.medrxiv.org/content/10.64898/2026.09.03.26361524v1?rss=1) forces sponsors and site teams to think clearly about. Evaluated in [Roche’s 629-patient ocrelizumab study](https://www.roche.com/media/releases/med-cor-2022-04-04), a digitally enhanced composite confirmed disability progression endpoint (cCDP) reduced required trial sample size by 24.9% at Week 48 compared to the standard cCDP definition. That is a real number with real budget consequences. It is also the beginning of an operational to-do list that most trial teams are not ready for.
## [](#what-the-24-9-actually-buys-you)What the 24.9% Actually Buys You
In a large Phase 3 MS trial, a 25% sample size reduction translates to meaningful cost relief. Per-patient costs in progressive MS trials commonly run into six figures when you account for EDSS assessments, MRI reads, neuro-cognitive testing, and coordinator time across two to four years. Shaving 150 to 170 patients off an enrollment target at that cost basis changes the economics of the program, not just the Gantt chart. It also shortens the enrollment window at each site, which matters for PI retention and coordinator morale more than most sponsors acknowledge at the outset.
The CONSONANCE framework earns that reduction by converting high-frequency smartphone-based gait data into progression events and folding them into the composite alongside traditional in-clinic measures. Instead of waiting for a patient to return for a timed 25-foot walk or a 9-hole peg test, the model captures ambulatory deterioration between visits and counts qualifying remote events toward the endpoint. More events, detected sooner, with greater precision. The statistical logic is sound, and the [EMA’s acceptance of stride velocity as a primary endpoint](https://pharmaphorum.com/news/ema-backs-sv95c-digital-primary-endpoint-dmd-trials) in ambulatory Duchenne Muscular Dystrophy studies confirms that [regulators can qualify digital gait measures](https://pmc.ncbi.nlm.nih.gov/articles/PMC10342974/) when the validation package is rigorous.
But that validation package is where site operations teams need to pay attention, because the work does not happen in the background.
## [](#the-amendment-queue-nobody-budgeted-for)The Amendment Queue Nobody Budgeted For
Integrating a remote digital endpoint into an existing composite requires a protocol amendment. Not a clarification, not an administrative update: a substantive amendment that touches the endpoint definition, the statistical analysis plan, the informed consent document, and the eCOA specifications. [Industry data puts the average amendment implementation timeline at 260 days](https://www.appliedclinicaltrialsonline.com/view/shining-a-light-on-the-inefficiencies-in-amendment-implementation) from identification to final ethics board approval. [That figure has nearly tripled over the prior decade](https://www.appliedclinicaltrialsonline.com/view/shining-a-light-on-the-inefficiencies-in-amendment-implementation). For a sponsor planning to introduce a digitally enhanced endpoint mid-study, 260 days is not a planning assumption, it is a warning.
Sites feel this differently than sponsors do. A protocol amendment means re-consenting enrolled patients, which requires a coordinator to schedule and document individual conversations with every active participant. In a 629-patient trial spread across 40 or 50 sites, that is a coordinator burden that arrives without additional budget unless the contract was written to include it, and it rarely is. The re-consent process also restarts the clock on certain data collection requirements, which creates TMF entries, deviation risk, and monitoring visit findings if the rollout is uneven across sites.
Then there is the eCOA and digital health technology validation stack. The FDA’s December 2023 final guidance on [digital health technologies for remote data acquisition in clinical investigations](https://www.cmhealthlaw.com/2023/12/fda-releases-guidance-on-digital-health-technologies-for-clinical-investigations/) sets out what sponsors must demonstrate before relying on DHT-derived data in a regulatory submission: fitness-for-purpose evidence, data flow verification, user verification, and a plan for handling missing or intermittent remote data. Every one of those requirements generates documentation that flows into the TMF. Sites that have never received a smartphone-based gait platform before need a site-level technology qualification, an SIV addendum, at minimum, and in many cases a separate site technology initiation visit before patients can even be enrolled on the digital arm of the composite.
Across the sites I work with, the technology initiation step alone adds two to four weeks to startup for sites that are new to the ePRO or remote monitoring vendor. That delay compounds when the vendor’s site support team is spread thin across multiple concurrent studies, which it almost always is during a broad rollout.
## [](#where-sponsors-underestimate-the-site-cost)Where Sponsors Underestimate the Site Cost
The sample size reduction math is clean on paper. The operational math at the site level has more variables. When a sponsor introduces a digital gait endpoint, the coordinator’s job expands: device provisioning, patient training on the smartphone application, troubleshooting connectivity issues, chasing missing data flags from the central monitoring dashboard, and documenting every instance where a patient’s remote data falls below the completeness threshold. None of that is captured in the original per-visit budget because it does not map to a visit. It maps to coordinator hours that accumulate between monitoring windows.
The CONSONANCE framework ran in a trial where the digital infrastructure was built in from the start. For sponsors considering this approach on an ongoing study, or designing a new study around a blended endpoint, the operational question is not whether the endpoint is statistically valid. It is whether the site-level budget reflects the true coordinator burden, whether the amendment timeline is factored into the enrollment projection, and whether the eCOA vendor has the site support capacity to onboard 40 sites in the window the sponsor has planned.
The 25% sample size reduction in CONSONANCE is a genuine signal that blended digital-clinical endpoints can do real statistical work in progressive MS. Roche’s MUSETTE trial, which [did not meet its primary cCDP endpoint](https://www.roche.com/media/releases/med-cor-2025-04-02) in relapsing MS, shows how much rides on endpoint sensitivity in this therapeutic area. Getting the endpoint architecture right matters. So does getting the site operations plan right before the amendment queue opens.
For sponsor clinical operations teams designing the next progressive MS study: build the DHT validation package before you lock the protocol, model the amendment timeline into your enrollment projection as a hard constraint rather than a buffer, and price coordinator re-consent and technology initiation work into the site budget explicitly. For site directors evaluating feasibility for a blended endpoint trial: ask the sponsor directly how many active studies are already on the remote monitoring vendor’s platform, and whether site technology initiation is budgeted as a separate line or absorbed into the SIV. The answer will tell you whether the 25% reduction flows to your enrollment timeline or disappears into an amendment queue that nobody planned for.
The next sponsor to file a blended digital-clinical endpoint package that survives regulatory scrutiny will set the evidentiary bar for progressive MS trials. Every site operations team that has worked through one of these amendments will know exactly what that filing cost in coordinator hours.
## [](#references)References
1. [medRxiv Neurology, “Remote ambulation monitoring enhances composite disability measurement in MS to deliver reduced trial sample size”](https://www.medrxiv.org/content/10.64898/2026.09.03.26361524v1?rss=1)
2. [CM Health Law, “FDA Releases Guidance on Digital Health Technologies for Clinical Investigations” (December 2023)](https://www.cmhealthlaw.com/2023/12/fda-releases-guidance-on-digital-health-technologies-for-clinical-investigations/)
3. [Roche, CONSONANCE Phase 3b Trial (ocrelizumab/Ocrevus), Hoffmann-La Roche](https://www.roche.com/media/releases/med-cor-2022-04-04)
4. [Roche, MUSETTE Trial Primary Endpoint Results (April 2025)](https://www.roche.com/media/releases/med-cor-2025-04-02)
5. [Applied Clinical Trials, “Shining a Light on the Inefficiencies in Amendment Implementation” (260-day average amendment timeline)](https://www.appliedclinicaltrialsonline.com/view/shining-a-light-on-the-inefficiencies-in-amendment-implementation)
6. [PMC, EMA qualification of Stride Velocity 95th centile (SV95C) as a primary digital endpoint in ambulatory DMD studies](https://pmc.ncbi.nlm.nih.gov/articles/PMC10342974/)
**Categories:** Clinical Trial Ops Brief
**Tags:** digital endpoints, eCOA validation, MS trials, protocol amendments, remote monitoring
---
### [Proteomic Aging Clocks Just Changed What a Phase 2a Trial Has to Prove](https://www.clinicaltrialvanguard.com/article/intel-brief/proteomic-aging-clocks-just-changed-what-a-phase-2a-trial-has-to-prove/)
**Published:** September 9, 2026
**Author:** Moe Alsumidaie
**Excerpt:** A Nature Biotechnology phase 2a trial ran six proteomic aging clocks simultaneously, here's what that means for biomarker strategy and FDA submission readiness.
**Content:**
The [Nature Biotechnology paper published this week](https://www.nature.com/articles/s41587-026-03286-y) on integrating proteomic aging clocks into a Phase 2a geroprotective trial is not a longevity science curiosity. It is a protocol design signal that sponsors in aging, fibrosis, metabolic disease, and neurodegeneration need to read before their next IND meeting.
The convergence matters more than any single clock’s output. When six algorithms built on different protein panels, different reference populations, and different statistical architectures all agree that a compound moved biological age in the same direction, that is not noise. That is a coordinated evidentiary foundation, and it is the kind of foundation the FDA’s [December 2018 Biomarker Qualification: Evidentiary Framework Guidance](https://www.fda.gov/media/119271/download) was written to accommodate, even if the agency has not yet told sponsors how to use it in a longevity context.
## [](#the-old-framework-doesnt-fit-anymore)The Old Framework Doesn’t Fit Anymore
Until recently, sponsors entering Phase 2a with a geroprotective hypothesis faced an uncomfortable choice: nominate a single surrogate endpoint and defend it against the FDA’s qualification bar, or run a scattershot panel of exploratory biomarkers and concede upfront that none of them would anchor a registration pathway. Neither option is satisfying. The first concentrates statistical risk on an unvalidated measure; the second produces a data package that reviewers treat as hypothesis-generating at best.
The proteomic clock strategy in this Nature Biotechnology trial takes a third path. Rather than asking which single protein or composite score qualifies as a surrogate, it asks whether multiple independently validated clocks, each with its own published accuracy against chronological age and mortality outcomes, show directional agreement. The UK Biobank work underlying one major proteomic clock used 2,897 plasma proteins across 45,441 participants to identify 204 proteins that predict chronological age.
That convergence reframes the evidentiary ask. Directional agreement across six clocks does not require any single clock to be qualified as a surrogate. It creates a pattern of consistent biological signal that a reviewer can evaluate as a package, closer to the weight-of-evidence standard the 2018 Biomarker Qualification framework describes than to the single-endpoint model most Phase 2a sponsors default to.
The precedent for this approach exists. Insilico Medicine’s [rentosertib Phase 2a trial in idiopathic pulmonary fibrosis](https://www.unite.ai/proteomic-aging-clocks-track-biological-age-reversal-in-rentosertib-trial/) applied [six proteomic aging clocks, including ProtAge, OrganAge, and PAC, across 42 patients over 12 weeks](https://insilico.com/news/rnt0709261-rentosertib-proteomic-aging-clocks). All six clocks indicated a reduction in predicted biological age. That is a small sample and a short duration, but the methodological template it established is now reproducible, and the Nature Biotechnology paper offers a further application of that methodological approach.
## [](#who-runs-this-protocol-next-and-at-what-cost)Who Runs This Protocol Next, and at What Cost
The therapeutic areas most immediately affected are the ones where sponsors already face a surrogate endpoint credibility gap. Aging-associated fibrotic diseases, metabolic syndrome, sarcopenia, and early neurodegeneration all share the same regulatory problem: hard clinical endpoints require decades-long trials, and existing surrogate endpoints either lack qualification or carry residual scientific controversy. A multi-clock proteomic strategy does not solve the qualification problem, but it creates a richer biological narrative that can support Phase 2b dose selection and justify the biological plausibility regulators expect before committing to a Phase 3 endpoint strategy.
The operational burden is real and should not be understated. Running six proteomic clocks simultaneously requires longitudinal serum sampling at defined protocol timepoints, a proteomics platform capable of measuring panels in the hundreds to low thousands of proteins per sample, a data infrastructure that can ingest and harmonize outputs across six algorithmic frameworks, and a biostatistics plan that pre-specifies how directional agreement will be defined and reported. Sponsors who treat this as an add-on analysis rather than a first-class protocol objective will produce data the FDA cannot evaluate cleanly.
The sample size question is not trivial. [Autosomal dominant Alzheimer’s disease biomarker trials published on medRxiv in 2024](https://www.medrxiv.org/content/10.1101/2024.07.25.24311002v1) examined how biomarker-based outcome measures may reduce required enrollment relative to cognitive endpoints when biomarker variability and effect size are characterized in advance. Sponsors integrating proteomic clocks into Phase 2a without published variance estimates for their target population are designing a study they cannot power. To the extent the Nature Biotechnology trial reports variance and effect-size data, the next sponsor may be able to use those figures in their sample size justification.
## [](#the-submission-question-the-fda-hasnt-answered)The Submission Question the FDA Hasn’t Answered
Here is the gap the FDA has not closed: the [2018 Biomarker Qualification framework](https://www.federalregister.gov/documents/2018/12/12/2018-26900/biomarker-qualification-evidentiary-framework-draft-guidance-for-industry-and-food-and-drug) describes an evidentiary process for nominating biomarkers as drug development tools, but it is not clear how a sponsor should present multi-clock proteomic data in a [Phase 2a clinical study report](https://database.ich.org/sites/default/files/E3_Guideline.pdf) when no individual clock holds COU (Context of Use) qualification for their indication. The result is that sponsors who run this methodology correctly may produce scientifically compelling data that arrives at the FDA in a format reviewers have no established template to evaluate.
That is not a reason to avoid the methodology. Clinical operations leaders preparing Type B meeting requests for aging-adjacent programs should explicitly raise the proteomic clock strategy as a discussion item and request written feedback on how the agency would prefer to see multi-clock directional agreement presented in the CSR. Getting that in writing before the IND is filed is worth more than any post-hoc analytical justification.
Watch the FDA’s Oncology Center of Excellence and CDER’s biomarker qualification program for any qualification submissions related to proteomic aging measures in the next 12 to 18 months. The first sponsor to move a multi-clock package through a formal COU qualification will set the evidentiary template, and every later Phase 2a filing in the geroprotective space will be measured against it.
## [](#references)References
1. [Nature Biotechnology, “Integration of proteomic aging clocks in a phase 2a clinical trial supports simultaneous geroprotective assessment”](https://www.nature.com/articles/s41587-026-03286-y)
2. [FDA/CDER/CBER, “Biomarker Qualification: Evidentiary Framework Guidance for Industry and FDA Staff,” December 2018](https://www.fda.gov/media/119271/download)
3. [PubMed / Nature Medicine, Proteomic aging clock validation, UK Biobank (n = 45,441), 2,897 plasma proteins, 204 age-predictive proteins](https://pubmed.ncbi.nlm.nih.gov/39117878/)
4. [Unite.AI, “Proteomic Aging Clocks Track Biological Age Reversal in Rentosertib Trial” (Insilico Medicine, IPF, 42 patients, 12 weeks)](https://www.unite.ai/proteomic-aging-clocks-track-biological-age-reversal-in-rentosertib-trial/)
5. [The Catalyst Brief, “Proteomic Clocks Show Lower Biological Age Across Six Models in Rentosertib Phase 2a Trial”](https://www.thecatalystbrief.com/article/proteomic-clocks-show-lower-biological-age-across-six-models-in-rentosertib-phase-2a-trial-extending-aging-endpoints-into-ipf-drug-development)
6. [medRxiv, Sample size estimates for biomarker-based outcome measures in Autosomal Dominant Alzheimer’s Disease, 2024](https://www.medrxiv.org/content/10.1101/2024.11.12.24316919v3.full-text)
**Categories:** Intel Brief
**Tags:** Adaptive Clinical Trial Design, Biomarkers, FDA Real-World Evidence, Phase 2a, Proteomic Aging Clocks
---
### [Advarra Releases IRB Reportable Events Checklist to Reduce Over-Reporting](https://www.clinicaltrialvanguard.com/news/advarra-releases-irb-reportable-events-checklist-to-reduce-over-reporting/)
**Published:** September 9, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Advarra releases IRB reportable events checklist to help research teams distinguish critical safety signals from minor deviations requiring prompt review.
**Content:**
Most research teams think the hard part of IRB compliance is catching every event. The harder part, the one that actually generates audit findings, is knowing which events to skip. FDA’s Bioresearch Monitoring program flagged IRB reporting procedure deficiencies in both fiscal year 2022 and 2023 inspections, and the pattern is consistent: sites over-report minor deviations that add no oversight value while missing the nuanced signals that genuinely change a study’s risk profile. A new [reportable events checklist from Advarra](https://www.advarra.com/blog/irb-reportable-events-checklist/) addresses that gap directly, with a checkpoint-by-checkpoint decision framework that forces the right question at each stage rather than defaulting to “when in doubt, submit.”
The framework runs nine checkpoints, but the logic pivots on three. Checkpoints two through four do the heaviest work: was the event unexpected relative to the protocol or investigator brochure, is there a reasonable possibility it’s related to study participation, and does it suggest a new or heightened risk to participants? All three have to point toward yes before an event clears the unanticipated problem threshold. Checkpoint five adds a layer specific to drug and biologic studies: a serious adverse event involving death, hospitalization, or a life-threatening condition still has to clear those same unexpected-related-increased-risk criteria before it becomes a prompt IRB report. Seriousness alone is not enough, and sites that treat every SAE as an automatic submission are doing the equivalent of filing noise.
Device studies get their own checkpoint around unanticipated adverse device effects, where the analysis also triggers a practical question: does the event require urgent corrective action, device modification, or participant notification before the next scheduled review? Protocol deviations follow a similar principle. A minor administrative error that touched no one’s safety and left data integrity intact generally does not warrant a prompt report; a deviation implemented to eliminate an immediate hazard, or one that compromised informed consent, does, along with a documented corrective and preventive action plan. The checklist also covers new safety information (checkpoint eight) and immediate protective actions (checkpoint nine), both of which can require consent-form revisions or study suspension before an IRB formally reviews the event.
Advarra’s IRB services [support more than 60% of clinical trials in North America](https://www.prnewswire.com/news-releases/advarra-integrates-study-startup-and-irb-technologies-enabling-unprecedented-visibility-into-clinical-trial-site-activation-302431025.html), which means the reporting habits this checklist is designed to correct play out across a substantial share of active studies. The metric worth watching at the site level is simpler than any compliance audit: the ratio of submissions that result in IRB action versus those that are acknowledged and closed with no change. A high proportion of the latter suggests over-reporting, and over-reporting has a cost, both in staff time and in the attention it draws away from events that actually matter.
*Source link: *
**Categories:** News
---
### [Nektar to Release 24-Week Off-Treatment Data for Rezpegaldesleukin in Alopecia Areata](https://www.clinicaltrialvanguard.com/news/nektar-to-release-24-week-off-treatment-data-for-rezpegaldesleukin-in-alopecia-areata/)
**Published:** September 9, 2026
**Author:** Jon Napitupulu
**Excerpt:** Nektar will release 24-week off-treatment data for rezpegaldesleukin in alopecia areata, testing durability after dosing ends in the Phase 2b REZOLVE-AA study.
**Content:**
Nektar Therapeutics went quiet on September 8, 2026, and will stay that way until it releases the 24-week off-treatment follow-up data from its [Phase 2b REZOLVE-AA study](https://ir.nektar.com/node/22426/pdf) of rezpegaldesleukin in patients with severe-to-very-severe alopecia areata. That timing is the only public signal the company has given about when the data arrive.
The follow-up read matters because it tests durability, not just response. The on-treatment results from REZOLVE-AA were already positive in the 92-patient study, but alopecia areata tends to relapse once treatment stops, and that is the question baricitinib and ritlecitinib have had to answer in post-approval practice. If rezpegaldesleukin, a [regulatory T-cell agonist targeting the IL-2 receptor complex](https://ir.nektar.com/news-releases/news-release-details/52-week-topline-results-16-week-blinded-treatment-extension), shows durable hair regrowth after dosing ends, it would offer a mechanistically distinct answer to that durability problem rather than another JAK inhibitor competing on the same pharmacology as the [two currently FDA-approved options](https://pmc.ncbi.nlm.nih.gov/articles/PMC9422172/).
The enrolled population adds an important wrinkle: all 92 patients were JAK-inhibitor-naive and biologic-naive. That design choice protects the signal from prior-treatment confounding, but it also means the durability read applies specifically to treatment-naive patients, not to the larger pool of people who have already cycled through baricitinib or ritlecitinib. Whether Nektar pursues a label that addresses that refractory population will depend on what Phase 3 looks like, and the off-treatment data will do a lot to shape that design decision.
Nektar notes the quiet period follows the same pattern it used for earlier REZOLVE study disclosures, so there is no structural surprise in the filing itself. The question is purely what the data show. Once the follow-up readout lands, the off-treatment durability curve will be the number that determines whether this mechanism earns a Phase 3 design that can compete in a field already defended by oral daily therapies with multi-year safety records.
*Source link: [https://www.sec.gov/Archives/edgar/data/906709/000121390026098042/ea0304798-8k\_nektar.htm](https://www.sec.gov/Archives/edgar/data/906709/000121390026098042/ea0304798-8k_nektar.htm)*
**Categories:** News
---
### [JST-018 Orthopoxvirus Antibody Enters Phase 1 Trial Under $123.9M Defense Contract](https://www.clinicaltrialvanguard.com/news/jst-018-orthopoxvirus-antibody-enters-phase-1-trial-under-123-9m-defense-contract/)
**Published:** September 9, 2026
**Author:** Jon Napitupulu
**Excerpt:** JST-018 orthopoxvirus antibody enters phase 1 trial under $123.9M defense contract with Just-Evotec Biologics for warfighter protection.
**Content:**
A first-in-human trial of JST-018, a monoclonal antibody cocktail targeting orthopoxviruses, started this month under a 2023 contract worth up to [$123.9 million combined](https://www.prnewswire.com/news-releases/just--evotec-biologics-advances-us-department-of-war-antibodies-against-orthopoxvirus-into-phase-1-clinical-studies-302873327.html) across two biodefense programs that Just – Evotec Biologics holds with the U.S. Department of War. The speed matters: the company moved JST-018 from antibody sequence selection through preclinical development, process development, regulatory filing, and cGMP manufacture entirely at its J.POD facility in Redmond, Washington, using continuous bioprocessing rather than batch manufacturing. That single-site, single-platform path is the actual test case here, not just the antibody.
JST-018 is intended as a prophylactic for warfighters at elevated biological threat exposure risk, which shapes the trial’s purpose differently from a typical therapeutic program. Orthopoxviruses include variola, the cause of smallpox, and the mpox virus. [Tecovirimat (TPOXX) was approved by the FDA in 2018](https://www.fda.gov/emergency-preparedness-and-response/medical-countermeasure-mcm-issues/smallpox-preparedness-and-response) for treatment of smallpox under the Animal Efficacy Rule, but the military’s interest in a prophylactic antibody cocktail reflects a different protective strategy, one aimed at exposure before disease onset rather than after. The Phase 1 study is registered at ClinicalTrials.gov under NCT07595458.
This orthopoxvirus program is the second Just – Evotec Biologics has advanced under the Accelerated Antibodies Program. The first, awarded in September 2022 and valued at up to $49.9 million, targeted plague. The combined ceiling across both contracts is $123.9 million. What the company is building, contract by contract, is a replicable CDMO model for biodefense: take a sequence, run it through a continuous manufacturing platform, clear regulatory submission, and deliver clinical material without spinning up a separate facility for each threat agent.
The Phase 1 readout will carry weight beyond JST-018 itself. [FDA’s Q13 guidance on continuous manufacturing](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/q13-continuous-manufacturing-drug-substances-and-drug-products) created a regulatory framework for this kind of process, but real clinical data generated through it for biodefense antibodies remain sparse. If JST-018 clears Phase 1 safety, the Redmond platform gains a validated case study the Department of War and BARDA can point to when structuring future CBRN countermeasure contracts.
*Source link: [https://www.prnewswire.com/news-releases/just–evotec-biologics-advances-us-department-of-war-antibodies-against-orthopoxvirus-into-phase-1-clinical-studies-302873327.html](https://www.prnewswire.com/news-releases/just--evotec-biologics-advances-us-department-of-war-antibodies-against-orthopoxvirus-into-phase-1-clinical-studies-302873327.html)*
**Categories:** News
---
### [Mosliciguat Advances to Phase 3 PHrontier Trial After PHocus Study Shows 20% PVR Reduction](https://www.clinicaltrialvanguard.com/news/mosliciguat-advances-to-phase-3-phrontier-trial-after-phocus-study-shows-20-pvr-reduction/)
**Published:** September 9, 2026
**Author:** Jon Napitupulu
**Excerpt:** Mosliciguat advances to Phase 3 PHrontier Trial after PHocus Study shows 20% pulmonary vascular resistance reduction in PH-ILD patients.
**Content:**
Mosliciguat cut pulmonary vascular resistance by 20 percent in Roivant’s Phase 2 PHocus trial, a result that now sends the once-daily inhaled drug directly into a Phase 3 program called PHrontier. The PVR result matters because it is the mechanistic signature of meaningful hemodynamic benefit in PH-ILD, a condition where the lung vasculature tightens as fibrosis advances and where existing approved options remain limited to treprostinil-based inhaled therapies such as [Tyvaso](https://www.prnewswire.com/news-releases/united-therapeutics-announces-fda-approval-and-launch-of-tyvaso-for-the-treatment-of-pulmonary-hypertension-associated-with-interstitial-lung-disease-301260212.html).
The PHocus data, presented at the European Respiratory Society Congress on September 8, also showed improvement in six-minute walk distance. What makes that worth noting is the mechanism behind it: mosliciguat is an [sGC activator](https://pmc.ncbi.nlm.nih.gov/articles/PMC9526466/), not an sGC stimulator. The distinction is pharmacologically real. In PH-ILD, oxidative stress depletes the heme cofactor from soluble guanylate cyclase, leaving the enzyme unresponsive to nitric oxide. An sGC stimulator requires some heme-bound enzyme to work; an activator targets the heme-free form directly. Whether that mechanistic fit translates to a clinical edge over existing options is exactly what PHrontier is designed to answer.
Roivant designed PHrontier as a Phase 3 trial in PH-ILD patients, though the company has not yet disclosed enrollment targets, primary endpoints, or a timeline for completion. The Phase 2 result gives the program a clear rationale for proceeding, but the path from hemodynamic signal to a label is still long. Regulatory agencies in PH typically want to see mortality or hospitalization data, and a trial powered for those outcomes takes years to complete and requires a much larger patient population than a Phase 2 hemodynamic study.
The commercial stakes depend almost entirely on what PHrontier’s endpoints look like. If Roivant pursues a six-minute walk distance primary endpoint, approval could come faster but payer uptake will be harder to secure against an established treprostinil standard. If the trial is powered for clinical worsening or survival, the timeline stretches but the value proposition sharpens. The endpoint choice, once Roivant discloses the [PHrontier protocol](https://www.globenewswire.com/news-release/2026/09/08/3357428/34323/en/roivant-announces-positive-results-from-phocus-study-of-mosliciguat-in-patients-with-pulmonary-hypertension-associated-with-interstitial-lung-disease-ph-ild-and-unveils-ongoing-pha.html), will tell you more about this program’s commercial ceiling than any Phase 2 number.
*Source link: *
**Categories:** News
---
### [Clinical trial sites advised to build data infrastructure before deploying AI tools](https://www.clinicaltrialvanguard.com/news/clinical-trial-sites-advised-to-build-data-infrastructure-before-deploying-ai-tools/)
**Published:** September 8, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Clinical trial sites should establish data infrastructure before deploying AI tools, according to industry experts and recent guidance on sequencing implementat
**Content:**
About three-quarters of clinical trial sites now use eSource in active studies, according to a 2024 RealTime Reports survey, yet the panel that CRIO and Clinical Leader convened last month found sites still wrestling with a more basic question: do you buy AI tools, or do you build them? The honest answer from operators who are already running AI day-to-day is that the question itself is premature for most sites.
The [panel](https://clinicalresearch.io/blog/the-site-ai-playbook-your-top-questions-answered/), moderated by CRIO Chief Innovation Officer Mike Wenger, included Aneesh Vaze of Clinical Research Philadelphia, Sam Stein of ALSA Research, and Nick Spittal of Velocity Clinical Research. Their consistent advice: sequence matters more than platform choice. Sites still running paper-based records need to move to electronic systems first, establish a basic AI governance policy, and give staff enterprise LLM accounts before touching anything custom-built. Skipping that foundation and going straight to homegrown tooling tends to create more cleanup work than it eliminates. For CRIO customers specifically, backend data access through Google BigQuery and Looker makes it possible to layer financial forecasting and custom dashboards directly onto operational data without re-entering anything, which is roughly the infrastructure a site needs before custom AI workflows are worth attempting.
Where AI is already earning its place, the applications are narrow on purpose. Drafting and updating investigator CVs came up as a concrete example: using an enterprise LLM account, describing the manual process clearly, and iterating through rough drafts produced faster results than trying to automate the whole workflow in one pass. A human reviewed every output. That pattern, small scope, human in the loop, dominated the panel’s practical recommendations. The failure mode runs in the opposite direction: automations that ingest documents, chat logs, and system data simultaneously while producing emails, reports, and dashboards in one step are harder to trust and harder to repair when something breaks. Inclusion and exclusion criteria were flagged specifically as a poor fit for current AI tools, since the criteria vary study-to-study and inconsistency in the input reliably produces incorrect interpretations in the output.
ICH E6(R3), which [does not contain explicit AI governance requirements](https://fdaqrc.com/project/ich-ai/) but provides a risk-based framework meant to accommodate new technologies, puts the documentation burden squarely on sites to justify whatever tools they use. That regulatory reality makes the panel’s sequencing advice more than operational preference: a site that deploys AI before its data infrastructure is clean has limited ability to demonstrate that its outputs are auditable. The concrete marker to watch is whether a site can answer, for any AI-assisted output it produces, exactly which data fed the model and which human reviewed the result.
*Source link: *
**Categories:** News
---
### [Ascendis to Present Navepegritide Infant Trial Data at ESPE 2026](https://www.clinicaltrialvanguard.com/news/ascendis-to-present-navepegritide-infant-trial-data-at-espe-2026/)
**Published:** September 8, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ascendis will present sentinel cohort data from the reACHin Trial of navepegritide infant trial at ESPE 2026, extending the CNP therapy's tested age range to in
**Content:**
Sentinel cohort data from a pivotal infant trial rarely reach a major congress podium before the broader enrollment is complete, which makes Ascendis Pharma’s slot at ESPE 2026 worth watching closely. The company will present the first data from the reACHin Trial of TransCon CNP (navepegritide) in infants with achondroplasia at the European Society for Paediatric Endocrinology annual congress, running September 8-10 in Marseille.
The reACHin readout matters because it extends navepegritide’s tested age range down to infancy. The earlier [pivotal ApproaCH Trial](https://investors.ascendispharma.com/news-releases/news-release-details/results-pivotal-approach-trial-transconr-cnp-navepegritide) in older children with achondroplasia showed significant improvements in annualized growth velocity at 52 weeks. Infants represent a distinct population both biologically and from a regulatory standpoint: BioMarin’s [Voxzogo (vosoritide) received FDA expanded approval in October 2023](https://www.hcplive.com/view/fda-approves-vosoritide-achondroplasia-children-under-5-years) to cover children from birth, so the infant segment already has an approved option, and any navepegritide data will face direct comparison against that established benchmark. The sentinel cohort design is an early safety and pharmacokinetic read in a small initial group; what ESPE attendees will actually see is whether the drug behaves predictably enough in very young patients to support continued enrollment rather than a full efficacy verdict.
Ascendis is also presenting updates from other endocrinology rare disease programs at the same congress, though the company has not detailed those datasets ahead of the meeting. [Achondroplasia affects roughly 1 in 21,000 births globally](https://www.globenewswire.com/news-release/2026/09/07/3357076/0/en/ascendis-to-share-its-latest-endocrinology-rare-disease-data-at-espe-2026.html), making each drug’s reach into younger age groups commercially meaningful even if the absolute patient numbers stay small. For a condition managed from birth onward, the ability to initiate treatment in the first months of life rather than waiting until age five is a genuine clinical question, not a line extension.
The number to track from the podium is whether the sentinel cohort safety profile supports dose progression to the full infant population in reACHin. That determination, more than any preliminary efficacy signal, controls how quickly the trial moves toward a dataset that could support a regulatory submission.
*Source link: *
**Categories:** News
---
### [Fortrea's $45M Phase I Grab Is Your Vendor Continuity Problem Now](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/fortreas-45m-phase-i-grab-is-your-vendor-continuity-problem-now/)
**Published:** September 7, 2026
**Author:** Krishma Shah
**Excerpt:** Fortrea's $45M acquisition of Worldwide Clinical Trials' early-phase division reshapes Phase I site networks and vendor contracts for sponsors mid-study.
**Content:**
The sponsor-side CTM who opened her inbox on September 2 and saw Fortrea’s announcement had one immediate operational question, and it had nothing to do with deal strategy. It was: *which of my active Phase I studies sits inside Worldwide’s early-phase network right now, and what happens to my contract on Monday?* That is the right question, and most teams were not ready to answer it quickly.
[Fortrea has agreed to acquire the Early Phase Services division of Worldwide Clinical Trials for $45 million](https://pharmasource.global/content/news/cro-news/fortrea-acquires-worldwide-clinical-trials-early-phase-division-for-45m-to-add-200-bed-cpu-and-glp-bioanalytical-lab/) in an all-cash transaction, picking up Worldwide’s Clinical Pharmacology Unit (CPU) and bioanalytical capabilities in the process. The deal closes subject to regulatory and licensing approvals, which means there is a window, likely weeks to a few months, during which sponsors have active Worldwide contracts, Worldwide SOPs, and Worldwide site staff managing their Phase I participants while the entity they are actually contracting with is in legal transition. That window is where operational risk concentrates, and it is almost always underestimated.
## [](#what-actually-transfers-in-a-cro-division-sale)What Actually Transfers in a CRO Division Sale
A division acquisition does not automatically novate your master service agreement. Sponsors with active Phase I work inside Worldwide’s CPU network need to pull their contracts now and identify three things: which entity is the named contracting party, whether there is a change-of-control clause that triggers notification or renegotiation rights, and whether the statement of work references Worldwide-specific SOPs, personnel, or facilities by name. Any of those conditions creates an operational action item before the deal closes, not after.
The operational continuity risk that sponsors underweight here is personnel. A CPU or clinical pharmacology unit runs on a small number of highly trained staff: Phase I-experienced nurses, clinical pharmacologists, pharmacokineticists, and the coordinators who run the participant flow inside what are often inpatient or close-monitoring environments. These are not general-site CRCs who can be replaced from a local hiring pool in three weeks. When a division changes hands, retention of that staff through the transition period depends entirely on decisions Fortrea and Worldwide are making right now, in conversations sponsors are not part of. Sites I work with across early-phase networks have seen coordinator turnover spike during CRO acquisitions, not because conditions deteriorate, but because uncertainty drives departures before anyone has made a single bad decision.
There is also the SOP gap to manage. Worldwide’s Phase I division operates under its own quality management system. [Fortrea, which reported $2,696.4 million in total revenue for full-year 2024](https://ir.fortrea.com/news-releases/news-release-details/fortrea-reports-fourth-quarter-and-full-year-2024-results-issues),, has its own QMS. During the transition period, the question of which SOP governs an SAE report, a protocol deviation write-up, or a sample handling procedure is not academic. ICH E6(R3) makes clear that the sponsor retains ultimate responsibility for trial oversight regardless of what a CRO is contracted to perform. That accountability does not pause because your CRO is mid-acquisition.
## [](#the-phase-i-capacity-math-sponsors-should-be-running)The Phase I Capacity Math Sponsors Should Be Running
Step back from the contract mechanics and the consolidation logic becomes visible. Phase I capacity in purpose-built CPUs has not expanded proportionally with the volume of first-in-human and clinical pharmacology studies that early-development pipelines are generating. When a CRO the size of [Fortrea pays $45 million for a division](https://www.fiercebiotech.com/cro/fortrea-boosts-early-phase-platform-45-million-cro-acquisition) rather than building equivalent capability organically, it is signaling that qualified Phase I capacity is constrained enough that acquisition is cheaper than construction. Sponsors who depend on a broad vendor panel for Phase I work should register that signal directly: the pool of independent Phase I CRO options is narrowing.
[Charles River Laboratories’ acquisition of Explora Biolabs in April 2022 for just under $300 million](https://ir.criver.com/news-releases/news-release-details/charles-river-laboratories-acquires-explora-biolabs) moved in the same direction. The pattern across CRO consolidation has been consistent: early-phase and specialized capability consolidates faster than late-phase general CRO capacity, because the infrastructure cost (inpatient units, bioanalytical labs, pharmacokinetic expertise) is high enough to make greenfield competition unattractive. For sponsors managing IND timelines, this matters at the site selection stage. Fewer independent early-phase networks means fewer competitive bids, which affects both price and timeline leverage.
Site activation inside a Phase I unit carries real cost. Tufts CSDD data puts site activation at approximately $20,000 per site, with ongoing administrative costs running roughly $1,500 per site per month through startup. In a CPU environment, those figures can run higher because of the specialized equipment qualification, pharmacy accountability requirements, and inpatient staffing overhead involved. Sponsors who built their Phase I vendor relationships assuming stable competitive alternatives now face a market where two of the largest players have absorbed capacity that was previously independent.
## [](#what-sponsor-clinops-teams-should-do-before-close)What Sponsor Clinops Teams Should Do Before Close
The operational response here has a short timeline. Before the Fortrea-Worldwide transaction closes, sponsor clinical operations leads with active Phase I work at Worldwide’s CPU sites should confirm the contracting entity named in their MSA, issue written notice to Worldwide requesting confirmation of operational continuity, and ask specifically whether any named Worldwide personnel on their SOW have signed retention agreements through the transition period. That last request will be uncomfortable to make, and it is exactly the one worth making.
Across the network of early-phase studies we support at CliniBiz, the acquisitions that create the least disruption share one characteristic: the sponsor treated the announcement date as a readiness trigger, not the close date. The teams who waited for the deal to close before reviewing their contracts found themselves renegotiating terms under time pressure, with less leverage, after key personnel had already made decisions about where they were going next.
For sponsors who are not yet mid-study but are planning Phase I work and were considering Worldwide’s CPU as a vendor option: the competitive dynamic has shifted. You are now evaluating Fortrea’s early-phase capability and Fortrea’s pricing, with Fortrea’s contract terms. Whether that is better or worse for your program depends on your relationship with Fortrea and your protocol requirements, but it is a materially different conversation than the one you would have had on September 1. Request updated capability profiles, ask for transition SOPs if they exist, and get clarity on which QMS will govern your study from initiation through the first interim data package.
The first sponsor to lock a clean Phase I contract under the new Fortrea structure, with retention commitments on key personnel and QMS transition language in the SOW, will be operating from a position of certainty while competitors are still reading the press release.
## [](#references)References
1. [FierceBiotech, “Fortrea boosts early-phase CRO platform with $45M acquisition of Worldwide division”](https://www.fiercebiotech.com/cro/fortrea-boosts-early-phase-platform-45-million-cro-acquisition)
2. [Applied Clinical Trials Online, “Fortrea to Acquire Worldwide Clinical Trials’ Early Phase Services Division for $45 Million”](https://www.appliedclinicaltrialsonline.com/view/fortrea-acquire-worldwide-clinical-trials-early-phase-services-division-45-million-deal)
3. [Fortrea Investor Relations, “Fortrea Reports Fourth Quarter and Full Year 2023 Results” (total revenue $3,109.0M for 2023; $2,696.4M for full-year 2024)](https://ir.fortrea.com/news-releases/news-release-details/fortrea-reports-fourth-quarter-and-full-year-2023-results-issues)
4. [Intuition Labs, “Clinical Trial Start-Up Costs” (site activation cost ~$20,000 per site; ~$1,500 per site per month)](https://intuitionlabs.ai/articles/clinical-trial-start-up-costs)
5. [WithPower, “Top CROs 2024” (Charles River Laboratories acquisition of Explora Biolabs, April 2022, ~$300 million)](https://www.withpower.com/guides/Top-CROs-2024)
**Categories:** Clinical Trial Ops Brief
**Tags:** Clinical Trial Ops Brief, CRO Consolidation, Phase I Operations, site activation, Vendor Management
---
### [The 400-Member Protein Family That Neuroscience Has Almost Entirely Ignored](https://www.clinicaltrialvanguard.com/opinion/the-400-member-protein-family-that-neuroscience-has-almost-entirely-ignored/)
**Published:** September 7, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Nature Reviews Drug Discovery identifies SLC transporters as untapped CNS drug targets, but 97% remain undrugged, and trial designers have no FDA playbook for…
**Content:**
Pull up the target list from any major neuroscience pipeline review and you will see the same architecture repeated: GPCR agonists, ion channel modulators, monoamine reuptake inhibitors. The field has spent four decades recycling the same molecular machinery while a protein superfamily of more than 400 members sits largely untouched. A [comprehensive review in *Nature Reviews Drug Discovery*](https://www.nature.com/articles/s41573-026-01513-4) published in 2026 now makes the case that solute carrier (SLC) membrane transporters represent one of the most consequential untapped target classes in CNS medicine. The numbers demand attention: [SLC transporters constitute an estimated 3% of human protein targets of approved drugs](https://www.tandfonline.com/doi/full/10.1080/17460441.2023.2244760), and [only two SLCs rank in the top 20 drug targets](https://pmc.ncbi.nlm.nih.gov/articles/PMC9763051/) by sales and NIH funding. Out of a superfamily exceeding 400 members, [roughly a third have no known ligand at all](https://www.medchemexpress.com/topics/solute-carrier-slc-transporters-serves-as-gatekeepers-in-metabolite-ecosystem.html).
For a trial designer building a neuro or psychiatry protocol right now, that statistic carries a specific operational weight. Targeting a largely uncharted protein family means entering a regulatory environment that has not fully developed the evidentiary standards to evaluate your molecule. The [FDA’s existing guidance on transporter-mediated drug-drug interactions](https://www.fda.gov/drugs/guidances-drugs/guidance-recap-podcast-in-vitro-drug-interaction-studies-cytochrome-p450-enzyme-and-transporter) focuses primarily on pharmacokinetic gatekeeping: will your compound inhibit transporters that affect the exposure of co-administered drugs? That framing, captured in [the Agency’s guidance on drug interaction studies](https://www.fda.gov/media/135587/download), was designed to protect patients from unexpected toxicity, not to establish proof-of-concept for transporter-mediated pharmacodynamics in the CNS. Those are fundamentally different questions, and the field is starting to realize they require fundamentally different trial architectures.
## [](#why-the-biology-keeps-getting-misread)Why the Biology Keeps Getting Misread
SLC transporters control the movement of neurotransmitters, amino acids, metal ions, glucose, and neuroactive steroids across cellular membranes. Several are dysregulated in Parkinson’s disease, major depressive disorder, ALS, and schizophrenia. The core mechanistic problem for drug developers is one of isoform complexity layered on top of tissue specificity layered on top of directional transport ambiguity. A compound that inhibits SLC6A4, the serotonin transporter, will look like a selective serotonin reuptake inhibitor at the synapse. But move ten members down the family and you are in territory where the same inhibitory mechanism might impair cellular nutrient uptake rather than tune neurotransmitter clearance.
This is the single principle that trial designers working in this space must internalize before they write the first line of a protocol: the pharmacological effect of SLC modulation is not predictable from the inhibition/activation binary that governs most small-molecule CNS targets. Direction of transport, subcellular localization, co-transporter dependencies, and substrate competition all determine what happens when you hit a specific SLC in a specific brain region. Preclinical packages that do not characterize these variables with tissue-specific precision will generate clinical candidates whose mechanism is incompletely understood at the point of IND submission.
That incompleteness has consequences in the clinic. The fedratinib story is instructive precisely because the SLC mechanism was not the intended pharmacology. [During the JAKARTA and JAKARTA-2 trials, the FDA placed a clinical hold on fedratinib in 2013](https://mpn-hub.com/medical-information/fedratinib-in-myelofibrosis) after cases of Wernicke’s encephalopathy emerged, a thiamine-deficiency syndrome later attributed partly to the drug’s inhibition of a thiamine transporter. The drug was targeting JAK2; the transporter was an off-target casualty. When the SLC mechanism is the intended pharmacology rather than a confound, sponsors will need to demonstrate both engagement of the target transporter and the functional downstream consequence in the CNS, a two-step evidentiary burden that current guidance documents do not specify.
## [](#the-regulatory-blind-spot)The Regulatory Blind Spot
Here is the counterintuitive reality that most coverage of this review will miss. The widespread assumption is that SLC transporters are underexploited because the biology is too hard. The more operationally accurate explanation is that the regulatory pathway for demonstrating CNS transporter pharmacodynamics in humans remains undefined, which suppresses investment, which suppresses the IND volume, which ensures the pathway stays undefined. It is a closed loop that the biology alone cannot break open.
The FDA’s transporter guidance framework, built around in vitro inhibition constants and clinical DDI studies, tells sponsors how to characterize a transporter as a safety variable. It does not tell them how to design a proof-of-concept trial where the transporter is the efficacy variable. Existing guidance documents do not specify what a valid pharmacodynamic biomarker for SLC engagement looks like in a CNS indication, and the field lacks established precedent for what the Agency expects as evidence that a novel SLC modulator reaches its target in the brain at therapeutically relevant concentrations. Positron emission tomography occupancy studies have established that standard for most receptor classes; the SLC field has not yet developed an equivalent consensus methodology.
Consider what that means at the Phase I/II design stage. A sponsor developing an SLC7A11 inhibitor for glutamate dysregulation in treatment-resistant schizophrenia would need to justify both the PK/PD model and the biomarker strategy from first principles, without an FDA-recognized framework to anchor the Type B meeting discussion. The reviewer on the other side of that meeting has no precedent file to consult. That asymmetry does not kill programs, but it adds significant time to iterative guidance-seeking in every early-phase program in this space, a cost that smaller biotechs with SLC assets in their pipelines are absorbing right now.
The *Nature Reviews Drug Discovery* analysis notes that numerous SLC transporters show dysregulated expression specifically in CNS tissue compared to peripheral tissues, a selectivity profile that is pharmacologically attractive precisely because it reduces the theoretical risk of systemic off-target effects. But clinical trial designers know that tissue selectivity in expression data does not automatically translate to CNS selectivity in drug distribution. The blood-brain barrier transport characteristics of SLC-targeted compounds add another layer of mechanistic work that must be completed before Phase II enrollment makes scientific sense. Sponsors who skip that work to accelerate timelines will find themselves facing a complete response letter built on mechanistic ambiguity rather than efficacy failure.
## [](#what-protocol-designers-must-build-in-now)What Protocol Designers Must Build In Now
The practical implication of all this is not that the SLC field is unviable. The therapeutic rationale is compelling enough to attract serious drug hunters: control over nutrient delivery, neurotransmitter gradients, and ionic homeostasis at the cellular level gives SLC-targeted drugs a precision that upstream signaling targets cannot match. The implication is that trial designers need to front-load the mechanistic work that the regulatory framework does not yet require but that reviewers will demand when submissions arrive.
That means three things in protocol architecture. First, CNS-targeted SLC programs need centrally assessed pharmacodynamic endpoints tied to the specific transport mechanism, not surrogate clinical endpoints borrowed from approved drug classes with different mechanisms. A protocol relying on Hamilton Depression Rating Scale improvement as the primary evidence of SLC target engagement is building on borrowed credibility. Second, the drug-drug interaction characterization required under existing FDA guidance must be extended to cover the SLC isoforms most relevant to CNS co-medications, because neuropsychiatry patients rarely take one drug, and the interaction landscape for novel SLC modulators is by definition uncharacterized. Third, biomarker strategies need to be locked before Phase I, not retrofitted at Phase II, because the window to establish transporter engagement in early human studies is narrow and may not be recoverable once the dose-ranging work is complete.
The review in *Nature Reviews Drug Discovery* frames SLC transporters as an emerging opportunity. From a trial operations perspective, the more precise framing is that they represent an emerging obligation: the field now has the genetic evidence, the expression data, and the pharmacological tools to build SLC-targeted CNS programs, which means the next cycle of neuroscience failures will belong to sponsors who had the target right and the trial design wrong. The FDA’s guidance infrastructure will catch up eventually. It always does, one complete response letter at a time.
## [](#references)References
1. [Nature Reviews Drug Discovery, “Solute carrier membrane transporters: emerging targets in CNS disorders”](https://www.nature.com/articles/s41573-026-01513-4)
2. [Expert Opinion on Drug Discovery, SLC transporters represent approximately 3% of human protein targets of approved drugs; only two SLCs rank in the top 20 drug targets](https://www.tandfonline.com/doi/full/10.1080/17460441.2023.2244760)
3. [U.S. Food and Drug Administration, Guidance on Drug Interaction Studies (transporter DDI framework)](https://www.fda.gov/media/135587/download)
4. [MPN Hub, Fedratinib JAKARTA/JAKARTA-2 trials; FDA clinical hold 2013 related to Wernicke’s encephalopathy and SLC19A3 inhibition](https://mpn-hub.com/medical-information/fedratinib-in-myelofibrosis)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, CNS Drug Development, FDA Drug-Drug Interactions, Neuroscience Trials, Solute Carrier Transporters
---
### [Tekton Research Sites Enroll 60 Participants in Phase 2 Obesity Trial, Triple Goal](https://www.clinicaltrialvanguard.com/news/tekton-research-sites-enroll-60-participants-in-phase-2-obesity-trial-triple-goal/)
**Published:** September 7, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Tekton Research sites enrolled 60 participants in Phase 2 obesity trial, tripling their combined enrollment goal and demonstrating strong patient access capabil
**Content:**
Enrolling three times the planned number of participants is rare enough that it usually points to something structural, not just enthusiasm. Tekton Research’s San Antonio and Edmond sites together randomized 60 participants into a Phase 2 obesity trial against a combined target of 20, with both sites clearing their individual goals as well.
The result matters in context. [Zepbound’s approval in late 2023](https://www.fda.gov/news-events/press-announcements/fda-approves-new-medication-chronic-weight-management) and the earlier [Wegovy approval in 2021](https://www.novomedlink.com/content/dam/wegovy/pdf/Wegovy%20Press%20Release_6.4_FINAL.pdf) accelerated sponsor interest in obesity mechanisms, and the pipeline behind those drugs has produced a wave of early-phase trials competing for the same patient pool. Sites that can reliably identify and screen motivated obesity patients are a genuine bottleneck, and 300% of goal in a Phase 2 suggests Tekton’s San Antonio and Edmond practices have that patient access built in, likely through the primary care and obesity medicine infrastructure those clinics already run. Jara McDonald, an FAAFP and DABOM-certified physician, leads the San Antonio team; Kyle Rickner leads Edmond.
For sponsors weighing site selection on early obesity studies, the operational signal here is straightforward: a site that triples enrollment in Phase 2 can compress dose-finding timelines and give a sponsor cleaner data faster, because the larger-than-planned cohort reduces the chance that a single dropout or protocol deviation distorts a small dataset. That is not a guarantee at Phase 3 scale, and a 20-person target was modest to begin with, but consistent over-enrollment at the Phase 2 stage is exactly the track record sponsors check before awarding larger slots.
The detail worth tracking is whether Tekton converts this result into Phase 3 allocations in obesity. Two sites randomizing 60 where 20 were expected is a data point for a sponsor’s feasibility model; the question is whether it translates into a named-site role in the larger, longer confirmatory work that the current obesity pipeline still needs.
*Source link: *
**Categories:** News
---
### [Myriad bioscaffolds show 0.7% deep infection rate in 411-patient soft tissue study](https://www.clinicaltrialvanguard.com/news/myriad-bioscaffolds-show-0-7-deep-infection-rate-in-411-patient-soft-tissue-study/)
**Published:** September 7, 2026
**Author:** Jon Napitupulu
**Excerpt:** Myriad bioscaffolds demonstrated a 0.7% deep infection rate in soft tissue reconstruction across 411 patients, significantly below historical benchmarks.
**Content:**
Among 411 patients treated with severe underlying disease, contaminated wounds, or exposed bone and tendon, Aroa Biosurgery recorded a deep tissue infection rate of 0.7% across 474 soft tissue defects in its [interim MASTRR Registry analysis](https://www.prnewswire.com/news-releases/peer-reviewed-411-patient-study-reports-low-infection-rates-with-myriad-in-complex-soft-tissue-reconstruction-302870912.html), now published in *Advances in Therapy*. For context on why that number matters: infection rates reported in separately published meta-analyses for resorbable synthetic bioscaffolds used in comparable procedures ranged from 16% to 25%.
The patient population was genuinely difficult. Roughly 59% of participants were ASA Class III or IV, meaning significant systemic disease, and 89% of defects were classified as clean-contaminated or contaminated. Fifteen percent involved confirmed osteomyelitis. Superficial infection occurred in 2.9% of patients, and no infections were attributed to the Myriad products over a median 27-week follow-up. Aroa describes this as the largest published prospective bioscaffold analysis in in-patient soft tissue reconstruction, to its knowledge, covering both [FDA-cleared](https://assets.pro.aroa.com/2025/06/08234408/MKT.1524.05_Myriad_PMaster_CC_25-26.pdf) Myriad Matrix and Myriad Morcells across 10 US centers.
The clinical stakes behind the infection numbers are concrete. A large US analysis of open surgical procedures found that a surgical site infection added roughly 7.8 to 9.3 days to hospital stay and between $18,600 and $21,000 in costs per patient, with deep tissue infections carrying heavier consequences still. The MASTRR interim analysis measured safety, not cost outcomes, so direct savings cannot be read from these data. Still, in a patient group where complication rates this low are uncommon, the infection profile gives hospital value committees something specific to consider when the product comes up for formulary review.
The registry remains open, with more than 550 patients enrolled against an 800-patient ceiling across 15 US sites. Earlier MASTRR publications addressed lower extremity reconstruction, trauma, burns, pilonidal disease, and pressure injuries in separate procedure-specific analyses. The next meaningful signal will come from whether the full 800-patient dataset, when complete, sustains the 0.7% deep infection rate across the wider range of surgical specialties Aroa is actively recruiting.
*Source link: *
**Categories:** News
---
### [Clover's RSV-hMPV Vaccine Shows Antibody Rises in Phase 2 Trial](https://www.clinicaltrialvanguard.com/news/clovers-rsv-hmpv-vaccine-shows-antibody-rises-in-phase-2-trial/)
**Published:** September 7, 2026
**Author:** Jon Napitupulu
**Excerpt:** Clover's RSV-hMPV vaccine produced 6-to-9-fold antibody rises in Phase 2, with no immune interference and strong safety. Candidate targets previously vaccinated
**Content:**
More than half of American adults 75 and older have already received one of the approved RSV vaccines, which creates an awkward problem for any late-entrant vaccine: the target population has pre-existing immunity. Clover’s Phase 2 data, released September 6, is built around exactly that obstacle. In 420 adults aged 60 to 85 enrolled in Australia, SCB-1022 (RSV + hMPV) and SCB-1033 (RSV + hMPV + PIV3) produced 6-to-9-fold neutralizing antibody rises against RSV and 6-to-8-fold rises against hMPV at 28 days post-vaccination, with no drop-off in the oldest participants (75 and above) compared to those aged 60 to 74.
The finding that matters most for competitive positioning is the absence of immune interference. Adding PIV3 antigen in SCB-1033 did not blunt RSV or hMPV responses compared to the two-pathogen formulation, and the company is explicitly positioning both candidates as options for people who already received [Arexvy](https://www.contagionlive.com/view/fda-approves-gsk-s-arexvy-as-the-world-s-first-rsv-vaccine-for-older-adults), [Abrysvo](https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-abrysvotm-pfizers-vaccine-prevention), or [mRESVIA](https://www.pharmacytimes.com/view/fda-approves-mrna-1345-for-protection-of-lower-respiratory-tract-disease-caused-by-rsv) to restore and broaden protection. That framing shifts the clinical question from “does it work?” to “does it add coverage that single-pathogen RSV vaccines cannot?” Phase 2 immunogenicity data cannot answer that definitively, but the pattern is consistent enough with Phase 1 to make the argument credible.
Safety held up through the trial period. Solicited adverse events within seven days were mostly mild and resolved in about two days across both vaccine and placebo groups. There were no vaccine-related serious adverse events, no events of special interest, and no discontinuations. Separately, the company completed 2,000-liter commercial-scale bioreactor production of all three antigen components, which matters because manufacturing failures at scale have derailed protein-subunit programs before. Having that process validated before a pivotal trial removes one of the larger late-stage unknowns. The trial also captured an incidental signal: respiratory tract infection rates in vaccinated participants were roughly 62 percent lower than in placebo recipients within 28 days, though the study used adverse event reporting rather than PCR confirmation, so that number should be read as hypothesis-generating at best.
Clover says it will evaluate mid- and late-stage development plans and seek global partnership opportunities, which means no Phase 3 start date is announced. [CDC data from February 2026](https://www.cdc.gov/rsvvaxview/dashboard/index.html) puts RSV vaccination coverage at 43 percent among adults 75 and older, leaving a meaningful unvaccinated fraction, but the commercial case for a three-pathogen combination ultimately rests on demonstrating that broader coverage translates to fewer hospitalizations, not just higher antibody titers. That question requires an outcomes-powered Phase 3, and the readiness of a potential partner to fund it is the real variable to watch.
*Source link: [https://www.prnewswire.com/news-releases/clover-announces-positive-phase-2-clinical-data-for-rsv–hmpv–piv3-respiratory-combination-vaccine-candidates-in-older-adults-302870886.html](https://www.prnewswire.com/news-releases/clover-announces-positive-phase-2-clinical-data-for-rsv--hmpv--piv3-respiratory-combination-vaccine-candidates-in-older-adults-302870886.html)*
**Categories:** News
---
### [FDA Guidance Expands Cancer Trial Eligibility, Reshaping Feasibility Models](https://www.clinicaltrialvanguard.com/news/fda-guidance-expands-cancer-trial-eligibility-reshaping-feasibility-models/)
**Published:** September 6, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA guidance expands cancer trial eligibility to include patients with ECOG PS 2, reshaping enrollment models and operational requirements for sponsors.
**Content:**
Forty percent of Phase III cancer trials explicitly barred patients with an ECOG performance status of 2 or higher, and among trials that allowed those patients, they made up just 3.6% of enrolled participants. The FDA’s [July 2026 final guidance on performance-status eligibility](https://www.onclive.com/view/fda-finalizes-3-guidance-documents-to-broaden-cancer-clinical-trial-eligibility-criteria) pushes directly against that pattern, recommending that adults with ECOG PS 2, or Karnofsky scores of 60 to 70, be included unless a specific scientific or clinical safety rationale justifies exclusion. For feasibility teams, the practical consequence is immediate: every assumption baked into a historical enrollment model was built under the old, narrower rules.
The problem is not simply that eligible headcounts will change. Historical enrollment records understate both the accessible population and the operational demands of supporting lower-functioning adults through a trial. A KRAS G12C eligibility [analysis of 1,172 NSCLC patients](https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.11012) at Columbia University Irving Medical Center illustrates why protocol criteria need study-specific scrutiny: eligibility rules shaped who reached trial enrollment in ways that aggregate registry data alone would not reveal. Teams that copy performance-status cutoffs from earlier protocols without re-examining the current safety evidence are building models on assumptions that the FDA now requires them to justify explicitly.
The guidance adds operational layers beyond the eligible headcount. The FDA recommends collecting baseline performance-status data, considering stratification when the enrolled range is broad, and supplementing clinician ratings with patient-reported functional assessments. Each of those requirements changes what a participating site must actually support: staffing for additional assessments, visit structures that accommodate patients with higher care needs, and escalation pathways for adverse-event patterns that narrower trials were never designed to detect. Feasibility questionnaires need to test those capacities directly, not assume that a site’s historical performance predicts its ability to run the proposed study.
The [ASCO and Friends of Cancer Research joint work](https://www.asco.org/research-data/clinical-trials/clinical-trial-eligibility-criteria) on eligibility criteria has long argued that restrictions should match scientific and safety objectives, not convention. The FDA’s final guidance gives that argument regulatory weight. The concrete marker to watch now is whether sponsors treat the guidance as a documentation exercise or as a genuine prompt to rerun scenarios with the broader criteria, conservative retention assumptions, and present-day site capacity confirmed rather than inferred.
*Source link: *
**Categories:** News
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### [Signant Health, Lothar Medical Plan Integrated Respiratory Trial Device](https://www.clinicaltrialvanguard.com/news/signant-health-lothar-medical-plan-integrated-respiratory-trial-device/)
**Published:** September 6, 2026
**Author:** Jon Napitupulu
**Excerpt:** Signant Health and Lothar Medical plan integrated respiratory trial device combining spirometry, oscillometry, and FeNO measurement into a single platform.
**Content:**
Respiratory clinical trials routinely ask sites to manage three separate devices for spirometry, oscillometry, and fractional exhaled nitric oxide measurement, then reconcile the data afterward. Signant Health and Lothar Medical signed a letter of intent on September 5 to build a single commercialized offering that collapses that workflow, pairing Lothar’s portable [ALDS PRO system](https://www.prnewswire.com/news-releases/signant-health-and-lothar-medical-sign-letter-of-intent-to-commercialize-alds-pro-for-clinical-trials-302870655.html) with Signant’s TrialMax eCOA platform and its Ametris wearable technology. The practical consequence for sponsors: one device handles all three pulmonary assessments on-site, and the resulting data feeds directly into the same evidence stack capturing patient-reported outcomes and continuous movement measures.
The integration matters because the three measurements are complementary in ways that matter clinically. Spirometry captures forced-effort lung volumes; [oscillometry measures airway mechanics during normal tidal breathing](https://pmc.ncbi.nlm.nih.gov/articles/PMC11895688/), which makes it useful in patients who cannot perform forced maneuvers reliably; and FeNO provides an objective signal tied to airway inflammation. Linking all three to eCOA data gives sponsors a direct connection between what patients report feeling and what the physiology shows, which is exactly the evidence picture regulators increasingly want in respiratory programs. Signant framed this as an extension of its Sensor-Enhanced eCOA strategy, which it accelerated when it [acquired Ametris (formerly ActiGraph) in May 2026](https://signanthealth.com/company/about-us), adding validated wearable-derived endpoints to its eCOA portfolio.
The commercialization structure gives Signant exclusive rights to distribute ALDS PRO for drug trial applications. That exclusivity is significant: it means sponsors running respiratory programs through Signant would gain access to ALDS PRO as part of an integrated service rather than sourcing the device separately and solving the data integration problem themselves. Both companies are presenting at the European Respiratory Society International Congress in Barcelona this week, booth U.02B, where they are running live demonstrations of ALDS PRO alongside the Ametris ActiGraph LEAP wearable. The demos are framed explicitly as proofs of concept, not as a commercially available integrated product yet.
The letter of intent is a framework, not a signed contract, so the integration could still change in scope or timeline. The number to watch is how quickly the companies move from LOI to a formal agreement and regulatory-ready validation package, since sponsors evaluating respiratory programs in 2027 planning cycles will need that clarity before they can spec ALDS PRO into a protocol.
*Source link: *
**Categories:** News
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### [Canada Recommends Conditional Reimbursement for Palopegteriparatide in Hypoparathyroidism](https://www.clinicaltrialvanguard.com/news/canada-recommends-conditional-reimbursement-for-palopegteriparatide-in-hypoparathyroidism/)
**Published:** September 6, 2026
**Author:** Jon Napitupulu
**Excerpt:** Canada's drug agency recommends conditional reimbursement for palopegteriparatide in hypoparathyroidism, pending price negotiation and provincial formulary deci
**Content:**
Canada’s Drug Agency recommended conditional public reimbursement for palopegteriparatide (Yorvipath) on September 3, giving adults with chronic hypoparathyroidism that conventional calcium and calcitriol therapy fails to control a realistic path to funded access. The conditions attached to the recommendation matter: coverage is not automatic, and negotiated price will almost certainly determine how quickly provincial formularies act.
The recommendation follows a clinical record built largely on the [Phase 3 PaTHway trial](https://www.hcplive.com/view/palopegteriparatide-provides-sustained-response-in-adults-with-hypoparathyroidism), which tracked sustained response in adults with hypoparathyroidism across three years. That durability argument carries weight in a condition where patients typically cycle through high-dose calcium and calcitriol indefinitely, often with incomplete symptom control and persistent hypocalcemia risk. Ascendis Pharma had already secured [FDA approval in August 2024](https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-yorvipathr-palopegteriparatide-first-and-only), so the Canadian agency had a substantial post-approval evidence base to evaluate, not just trial data.
The practical bottleneck now is price negotiation with the pan-Canadian Pharmaceutical Alliance. CDEC conditional recommendations routinely stall at that stage, and chronic hypoparathyroidism affects a small patient population, which cuts both ways: the per-patient budget impact is manageable, but the absence of a large comparator pool makes cost-effectiveness modeling contentious. Natpara (recombinant human PTH 1-84) is the [established hormonal alternative](https://canjhealthtechnol.ca/index.php/cjht/article/view/SR0903/3649) against which payers will benchmark value, and its formulary history across Canadian provinces will shape how aggressively they negotiate.
The clinical team to watch is the one managing patients who meet the “inadequate control” threshold the recommendation defines. That eligibility criterion will be the operational gate: if the bar is drawn narrowly around documented hypocalcemia episodes or hospitalization, uptake stays limited regardless of reimbursement status. How the final coverage criteria define “inadequately controlled” is the single specification worth tracking as provincial decisions follow.
*Source link: *
**Categories:** News
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### [Roivant to Present Phase 2 Mosliciguat Data for PH-ILD on Tuesday](https://www.clinicaltrialvanguard.com/news/roivant-to-present-phase-2-mosliciguat-data-for-ph-ild-on-tuesday/)
**Published:** September 6, 2026
**Author:** Jon Napitupulu
**Excerpt:** Roivant presents Phase 2 mosliciguat data for PH-ILD, measuring pulmonary vascular resistance changes over 24 weeks in a 135-patient trial.
**Content:**
Roivant and Pulmovant have 135 patients’ worth of Phase 2 data ready to show, and the number that matters most will be public Tuesday morning: how much mosliciguat moved pulmonary vascular resistance in people with [pulmonary hypertension associated with interstitial lung disease](https://www.investing.com/news/company-news/pulmovant-to-present-phase-2-mosliciguat-study-results-tuesday-93CH-4890257) over 24 weeks of blinded treatment. PH-ILD is a condition where approved options remain limited, and the one that exists, inhaled treprostinil (Tyvaso, approved April 2021), works through prostacyclin pathways. Mosliciguat takes a different route entirely.
That mechanistic difference is the scientific bet worth watching. Rather than stimulating native soluble guanylate cyclase the way sGC stimulators do, [mosliciguat directly activates the heme-free, nitric-oxide-unresponsive form of the enzyme](https://pmc.ncbi.nlm.nih.gov/articles/PMC9526466/), the form that accumulates in damaged, oxidized tissue. The rationale is that PH-ILD lungs may be precisely the environment where that form predominates, which makes the drug potentially relevant where other sGC-targeting agents cannot act. Whether the biology translates into a measurable hemodynamic signal is what the PHocus data will answer.
Marc Humbert, Professor of Respiratory Medicine at the University Paris-Saclay, presents the topline results at 12:15 CEST (6:15 a.m. ET) at the ERS International Congress 2026 in Basel. Roivant and Pulmovant host an investor call the same day. The PHocus study was a randomized, double-blind, placebo-controlled global trial, with all participants eligible to receive mosliciguat in the open-label extension after the 24-week controlled period. That extension will eventually inform longer-term safety, but Tuesday’s readout covers the controlled period and will define whether Pulmovant has a drug worth advancing to Phase 3.
The single number to watch when the data drop is the change from baseline in pulmonary vascular resistance in the mosliciguat arm versus placebo. That endpoint drove the pivotal program for Tyvaso in PH-ILD and remains the hemodynamic anchor regulators and pulmonologists use to judge whether a new agent does meaningful work in this population.
*Source link: *
**Categories:** News
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### [Early Termination No Longer Means Failure. It Means the Trial Was Working.](https://www.clinicaltrialvanguard.com/article/trend-watch/early-termination-no-longer-means-failure-it-means-the-trial-was-working/)
**Published:** September 5, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Early trial termination once signaled collapse. Now it signals design sophistication, and regulators, sponsors, and CROs need a new operational playbook to…
**Content:**
The CheckMate 057 trial was supposed to run longer. Bristol Myers Squibb had enrolled hundreds of patients with non-small cell lung cancer, randomized them between nivolumab and docetaxel, and set a completion timeline that the independent [Data Monitoring Committee recommended stopping the trial ahead of schedule in 2015](https://news.bms.com/news/details/2015/CheckMate--057-a-Pivotal-Phase-III-Opdivo-nivolumab-Lung-Cancer-Trial-Stopped-Early/default.aspx). Nivolumab had already demonstrated superior overall survival. Continuing the trial would have meant keeping patients on a demonstrably inferior arm. The DMC stopped it. The FDA approved it. And the industry quietly filed the episode under “this is how it’s supposed to work now.”
What the CheckMate 057 stop actually signaled was a structural shift in how the field understands termination itself. For decades, stopping a trial early carried an implicit presumption of failure: the assumptions were wrong, the logistics collapsed, the safety signal was damning. The [JAMA analysis on early termination](https://jamanetwork.com/journals/jama/fullarticle/2852512) now puts that assumption under direct pressure. Early termination has become a feature, not a flaw, and the operational infrastructure built around the old model is showing its age.
## [](#the-presumption-that-stopped-making-sense)The Presumption That Stopped Making Sense
Go back to the foundational logic of trial design as it stood through most of the twentieth century. Sample sizes were calculated on baseline assumptions: expected event rates, anticipated dropout, estimated variance in outcomes. A sponsor committed to a number, submitted a protocol, and was expected to honor it. When enrollment stopped prematurely, reviewers interpreted it as a confession that something foundational had broken. Twelve percent of trials registered in ClinicalTrials.gov with a primary completion date of December 2011 or earlier were terminated, according to a PLOS ONE cross-sectional study covering 7,646 trials. That figure was treated, largely, as a measure of industry dysfunction.
The interpretation was understandable. Most of those terminations were genuinely failures: insufficient enrollment, funding collapse, protocol deviation accumulation that made the data uninterpretable. But the same statistic now obscures something different. A trial stopped at interim because a pre-specified efficacy boundary has been crossed is not a failed trial. It is a trial where the monitoring architecture functioned exactly as designed.
That distinction matters enormously for how sponsors document termination decisions, how CROs wind down operations, and how regulators evaluate submitted data.
## [](#the-regulatory-architecture-catching-up)The Regulatory Architecture Catching Up
The FDA’s September 2025 draft guidance, [E20 Adaptive Designs for Clinical Trials](https://www.pharmafocusamerica.com/articles/adaptive-clinical-trials), under [docket FDA-2025-D-3023](https://www.federalregister.gov/documents/2025/09/30/2025-18897/e20-adaptive-designs-for-clinical-trials-international-council-for-harmonisation-draft-guidance-for), represents the clearest regulatory acknowledgment yet that planned adaptations, including early stopping for efficacy or futility, require their own evidentiary and procedural standards. The guidance, developed under ICH harmonization, addresses how pre-specification and transparency bear on whether an early stop strengthens or weakens a submission. An efficacy stop with a pre-registered boundary and a functioning DSMB is a different evidentiary object than an ad hoc decision to stop because enrollment slowed.
The FDA had already signaled this direction through its 2024 draft guidance on Data Monitoring Committees, where the agency emphasized that sponsors specify stopping rules for predefined adaptive features in DMC charters. That distinction, between a prospective stopping rule and a retrospective justification, now carries regulatory weight that it did not carry ten years ago.
What this creates operationally is a two-tier termination landscape. Trials stopped inside a pre-specified adaptive framework carry one evidentiary burden. Trials stopped outside that framework carry a heavier one, and sponsors who blur the boundary between the two should expect scrutiny in review.
The industry has not fully absorbed that two-tier structure yet. DMC charters that treat stopping rules as statistical housekeeping rather than as evidentiary commitments will face increasing scrutiny under this framework.
## [](#what-breaks-if-sponsors-dont-adjust)What Breaks If Sponsors Don’t Adjust
Consider the operational chain that follows an efficacy stop in an oncology Phase III. The DMC recommends termination. The sponsor notifies IRBs, sites, and the FDA. Patients on the experimental arm need continuation access or transition protocols. Patients on the control arm need safety follow-up plans. The regulatory submission must explain, with specificity, why the stopping decision was made at this interim and not a later one, and whether any data were unblinded outside the pre-specified process. Each of those steps requires infrastructure that was designed for a trial running to completion.
Sites are structurally unprepared for rapid wind-down. Site agreements rarely specify financial closeout timelines for early termination scenarios, which means sponsors absorb dispute costs that dwarf whatever was saved by stopping the trial before full enrollment. CROs managing data lock under early termination face a different statistical validation burden than they do at planned completion, particularly when adaptive randomization algorithms have shifted allocation ratios during the trial. And the FDA, reviewing a submission built on data from a stopped trial, needs to see the audit trail between the DMC’s recommendation and the sponsor’s operational response.
None of that infrastructure is mature. The FDA’s [Complex Innovative Trial Design program](https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-area-complex-innovative-trial-design), which exists precisely to give sponsors a pre-submission dialogue channel for adaptive designs, logged increasing engagement through the PDUFA VII cycle. But CID meeting requests are not the same as operational readiness. A sponsor can align with the FDA on a stopping boundary at Type B and still have a site closeout plan that does not contemplate early termination at all.
Technology vendors have a specific exposure here. EDC systems, eCOA platforms, and RTSM tools are typically configured for a planned end state. When a trial stops at 60% enrollment, the data pipelines designed to aggregate complete datasets produce incomplete exports. Vendors who build early-termination state logic into their trial configurations from go-live will price that service as a premium. The trajectory suggests it will become a baseline expectation before long.
The counterintuitive implication is this: the sponsors most likely to benefit from adaptive early stopping are precisely the ones whose operational teams are least experienced with it. Large Phase III programs in competitive therapeutic areas have every incentive to build in efficacy stopping rules because the cost of over-enrollment in a trial you’ve already won is enormous. But those same programs often have the most complex site networks and the least flexible closeout infrastructure. The operational savings from stopping early at an interim can be consumed entirely by closeout disputes and data remediation if the sponsor did not build the wind-down architecture before the trial started.
The JAMA analysis reframes what was always a design question as an operations question. How a trial ends was treated for decades as an afterthought, the province of statisticians and DSMB members. The era of adaptive monitoring has made it a first-order concern for every CTM, CRO operations lead, and technology vendor involved from Day 1. Sponsors who document early efficacy stops with the same operational rigor as a planned completion are likely to strengthen their submissions and influence how reviewers evaluate subsequent filings.
## [](#references)References
1. [JAMA, “Trials Terminated Early”](https://jamanetwork.com/journals/jama/fullarticle/2852512)
2. [PLOS ONE, “Terminated Trials in the ClinicalTrials.gov Results Database: Evaluation of Availability of Primary Outcome Data and Reasons for Termination”](https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0127242)
3. [Bristol Myers Squibb, “CheckMate 057, a Pivotal Phase III Opdivo (nivolumab) Lung Cancer Trial Stopped Early” (2015)](https://news.bms.com/news/details/2015/CheckMate--057-a-Pivotal-Phase-III-Opdivo-nivolumab-Lung-Cancer-Trial-Stopped-Early/default.aspx)
4. [Pharma Focus America, “E20 Adaptive Designs for Clinical Trials, FDA Draft Guidance, docket FDA-2025-D-3023”](https://www.pharmafocusamerica.com/articles/adaptive-clinical-trials)
5. [King & Spalding, “FDA Issues Draft Guidance on Use of Data Monitoring Committees in Clinical Trials” (2024)](https://www.kslaw.com/insights/articles/fda-issues-draft-guidance-on-use-of-data-monitoring-committees-in-clinical-trials)
6. [FDA, Complex Innovative Trial Design Program](https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-area-complex-innovative-trial-design)
**Categories:** Trend Watch
**Tags:** Adaptive Trial Design, Clinical Trial Operations, data monitoring committees, early termination, FDA Guidance
---
### [AI Agents in Clinical Trials Require Human Review Before Data Commits](https://www.clinicaltrialvanguard.com/news/ai-agents-in-clinical-trials-require-human-review-before-data-commits/)
**Published:** September 5, 2026
**Author:** Moe Alsumidaie
**Excerpt:** AI Agents in Clinical Trials require human review before data commits to clinical databases, balancing automation with regulatory accountability.
**Content:**
A phase 3 protocol in 2020 collected an average of 3.56 million data points. By 2025 that number had climbed to 5.96 million, a 67% rise in five years and more than six times the 2012 baseline of 929,203, according to [joint research from TransCelerate BioPharma and the Tufts Center for the Study of Drug Development](https://www.prnewswire.com/news-releases/transcelerate-and-tufts-csdd-uncover-opportunities-to-rethink-data-collection-and-optimize-protocol-design-302556373.html). That trajectory is exactly what is pushing AI agents from demo to deployment across clinical operations. The problem is that the volume of data now arriving in trials outpaces what human monitors can process, which is what makes autonomous agents attractive. The problem is also that those same agents make errors confidently, which is what makes unsupervised autonomy dangerous.
The same TransCelerate/Tufts work found that roughly one-third of all procedures and data points collected in trials are non-core or non-essential. Agents trained on bloated protocols inherit that bloat. An agent processing 6 million data points across endpoints, wearables, and decentralized assessments can surface patterns faster than any CRA team, but a misconfigured rule or a hallucinated query string propagates across every site simultaneously. The failure mode for autonomous systems is not slow and local; it is fast and global. That asymmetry is what the industry has not fully priced in yet.
The emerging operational model treats agent supervision as a defined role rather than a residual task. Platforms like [Castor EDC](https://www.castoredc.com/) build mandatory human review into AI-extracted data workflows before anything commits to the clinical database. That architecture reflects where the practical ceiling is today: agents handle extraction, flagging, and pattern recognition, while a human signs off before the record is final. The FDA’s framework for [real-world data in regulatory submissions](https://www.fda.gov/media/171667/download) expects traceability and human accountability at the point of decision, which makes fully autonomous commit functions a regulatory liability regardless of technical capability.
What changes operationally is not whether humans stay in the loop but what they do there. Reviewing agent outputs at scale demands a different skill set than traditional monitoring: less site travel, more query triage, more judgment about whether an agent’s flag reflects a genuine signal or a training artifact. Sponsors building out these workflows now are essentially defining what a clinical data reviewer does in a trial generating 6 million data points. The metric worth watching is how that review capacity scales relative to protocol complexity, because the data volume curve has not flattened.
*Source link: *
**Categories:** News
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### [Intellia Secures $400M Credit Facility for NTLA-2002 HAE Trial](https://www.clinicaltrialvanguard.com/news/intellia-secures-400m-credit-facility-for-ntla-2002-hae-trial/)
**Published:** September 5, 2026
**Author:** Jon Napitupulu
**Excerpt:** Intellia secures $400M credit facility to fund the NTLA-2002 HAE trial through completion, avoiding shareholder dilution from equity offerings.
**Content:**
Intellia Therapeutics closed a senior secured credit facility of up to $400 million on September 4, 2026, with OrbiMed Royalty & Credit Opportunities V, LP as administrative agent. For a company whose lead program has not yet generated product revenue, that capital injection answers a direct question: how does Intellia fund a global pivotal trial through readout without diluting shareholders further through an equity offering?
The program in question is [NTLA-2002 (lonvo-z)](https://clinicaltrials.gov/study/NCT06634420), a single-dose CRISPR therapy for hereditary angioedema currently enrolling in the Phase 3 HAELO trial. HAE causes recurrent, potentially life-threatening swelling attacks driven by excess plasma kallikrein activity; NTLA-2002 is designed to permanently inactivate the gene responsible. Running a global pivotal trial in a rare disease through to a regulatory submission is expensive, and the $400 million ceiling on this facility suggests Intellia and OrbiMed sized it to cover that stretch without needing an immediate secondary offering.
The structure matters as much as the size. A royalty and credit fund like OrbiMed’s vehicle typically prices this kind of debt against future revenue or royalty streams, which means the lender is betting on approval and commercial uptake, not just on Intellia’s current cash position. That is a different calculus than a traditional bank line, and it puts pressure on the HAELO readout: a negative or ambiguous result would complicate any refinancing or drawdown under terms likely tied to program milestones. Intellia has not disclosed the specific financial covenants or draw conditions in the filing excerpt available.
The broader context is that CRISPR-based medicines are moving from proof-of-concept into pivotal development across multiple companies, with Casgevy already holding FDA approval for sickle cell disease and beta thalassemia. Intellia is trying to demonstrate that in vivo CRISPR editing, delivered systemically rather than through an ex vivo cell process, can reach late-stage success. The number to watch is not the facility size but the HAELO enrollment pace: how quickly Intellia activates sites and gets patients dosed under this program will determine whether $400 million is enough runway or a bridge to another capital event.
*Source link: *
**Categories:** News
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### [CluePoints Launches AI Tools for Clinical Data Review With Top 10 Pharma Partner](https://www.clinicaltrialvanguard.com/news/cluepoints-launches-ai-tools-for-clinical-data-review-with-top-10-pharma-partner/)
**Published:** September 4, 2026
**Author:** Moe Alsumidaie
**Excerpt:** CluePoints launches AI tools for clinical data review with capabilities targeting 99% accuracy in medical coding and cutting manual effort by half.
**Content:**
Clinical data review still runs largely on spreadsheets and manual listing checks, even as trial volumes grow, which is why it matters that CluePoints is releasing three AI-based capabilities built and stress-tested inside a top 10 pharmaceutical company’s live portfolio before going generally available. The two figures buried in the launch details tell the stakes: Intelligent Medical Coding (IMC) targets up to 99% accuracy on MedDRA suggestions and cuts manual coding effort by roughly half, gains that compound across every study that currently resets its institutional coding knowledge at each dictionary up-version or team turnover.
The three capabilities, Intelligent Query Detection (IQD), Medical and Safety Review (MSR), and IMC, each attack a different bottleneck. IQD lets a data manager define any clinical or operational question in plain terms, without code or a vendor change request, and the system applies that logic continuously, returning only records that need attention. MSR replaces static spreadsheet-driven medical and safety reconciliation with a change-based, patient-centric workflow that posts queries directly back to the EDC, removing the handoff between Medical, Safety, and Data Management that typically introduces the longest delays. The co-innovation partner’s teams shaped both IQD’s user-defined scenario architecture and MSR’s change-based review model, and the capabilities are already running across the vast majority of that partner’s ongoing studies, including mega-studies, at launch.
The regulatory timing is not incidental. [ICH E6(R3) was finalized in January 2025](https://about.citiprogram.org/blog/ich-releases-final-version-of-e6r3-good-clinical-practice-guideline/), with the EMA making it effective July 23, 2025, and the FDA publishing it in September 2025. The guidance formally encodes risk-based quality management principles that CluePoints’ earlier RBQM platform helped establish in practice. Extending the same statistical and scientific foundation into query management, medical review, and coding now connects a sponsor’s [data oversight workflow](https://cluepoints.com/cluepoints-launches-iqd-msr-imc-clinical-data-review/) end-to-end under a single framework the guideline already recognizes, rather than requiring teams to reconcile outputs across disconnected systems before acting.
MSR deploys in days and works across ongoing studies, not just new ones, which removes the typical barrier of waiting for a clean trial start. The practical test of whether that speed holds at scale will be visible when the unnamed co-innovation partner’s data management leadership appears alongside CluePoints at the [SCDM 2026 Annual Conference](https://cluepoints.com/cluepoints-launches-iqd-msr-imc-clinical-data-review/) in Raleigh, September 14 through 17, presenting production results rather than a pre-launch proof of concept.
*Source link: *
**Categories:** News
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### [Thryv Therapeutics licenses SGK1 inhibitors for Parkinson's, Alzheimer's research](https://www.clinicaltrialvanguard.com/news/thryv-therapeutics-licenses-sgk1-inhibitors-for-parkinsons-alzheimers-research/)
**Published:** September 4, 2026
**Author:** Jon Napitupulu
**Excerpt:** Thryv Therapeutics licenses SGK1 inhibitors from Spanish research institutions for Parkinson's and Alzheimer's disease development, targeting a first candidate
**Content:**
Thryv Therapeutics has spent years building a single chemical scaffold into cardiac clinical assets. The CSIC/UAM license, effective July 1, 2026, gives it a structurally independent second scaffold, and the target now points at the brain: Parkinson’s disease, Alzheimer’s disease, and related tauopathies. A first development candidate from the new series is targeted for nomination in 2027, while the existing cardiac program runs in parallel.
The practical value of a second, distinct chemotype is that it lets Thryv separate optimization tracks. Its current molecules, built from a library of more than 500 synthesized analogs on the original scaffold, are advancing toward cardiac indications. [THRV-1268](https://clinicaltrials.gov/study/NCT07277582) is now in the WAVE II study in Long QT Syndrome Type 2, with a Phase 2a heart failure trial (ASPIRE-HF) planned to follow. The new CSIC/UAM series was never designed to compete with that chemistry; it was designed from the start to cross into the central nervous system, where the cardiac compounds were not built to go.
SGK1 has published links to tau phosphorylation, misfolded protein handling, neuroinflammatory signaling, and FOXO- and NRF2-dependent stress responses, all pathways with direct relevance to Parkinson’s and Alzheimer’s. The research groups behind the licensed compounds span medicinal chemistry, neurobiology, cardiovascular biology, and vascular pharmacology at CSIC’s Centro de Investigaciones Biológicas Margarita Salas and UAM’s Institute for Biomedical Research. That breadth matters because CNS drug development typically requires the chemistry and the disease biology to be co-developed from early stages. Approved options for Alzheimer’s disease remain limited and recently expanded to include [lecanemab maintenance dosing](https://www.brightfocus.org/resource/expanding-the-alzheimers-treatment-landscape-a-2026-forecast/); for Parkinson’s, the treatment toolkit has been largely stable, with the [FDA’s March 2026 labeling action](https://parkinsons.org/treatments/new-medications) on levodopa/carbidopa the most recent notable regulatory move. Neither disease has an approved therapy that addresses the kinase biology Thryv is targeting.
The concrete marker to track now is whether Thryv nominates a development candidate from the new series on its 2027 timeline, since that decision will signal whether the CNS program moves from platform expansion into something with a clinical path.
*Source link: *
**Categories:** News
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### [Argo's BW-20805 Achieves 96% Attack-Rate Reduction in HAE Phase II](https://www.clinicaltrialvanguard.com/news/argos-bw-20805-achieves-96-attack-rate-reduction-in-hae-phase-ii/)
**Published:** September 4, 2026
**Author:** Jon Napitupulu
**Excerpt:** Argo's BW-20805 achieved 96% attack-rate reduction in Phase II HAE study, with 75% of patients in the 300 mg every-24-weeks group remaining attack-free.
**Content:**
Among the three dosing arms in Argo Biopharma’s Phase II study of BW-20805, the 300 mg every-24-weeks group produced a 96% mean reduction in monthly hereditary angioedema attack rate over the first 169 days of treatment, with three out of four patients in that arm remaining completely attack-free. That figure, drawn from a June 2026 data cut across 24 evaluable participants, is the centerpiece of an oral presentation the company delivered at the [Bradykinin Symposium 2026](https://bradykinin-symposium.de/) in Berlin this week.
The other two regimens tracked closely. The 300 mg every-12-weeks arm cut monthly attack rates by 93%, with 62.5% of patients attack-free; the 600 mg every-24-weeks arm reduced rates by 83%, with half of patients attack-free over the same window. On the pharmacodynamic side, plasma prekallikrein suppression at Day 169 reached 85 to 94% across all three groups in the 19-patient PKK-evaluable subset. BW-20805 works by silencing hepatic prekallikrein mRNA, a validated target in HAE, and the dosing intervals tested, either every 12 or every 24 weeks, are notably longer than those of several existing prophylactic agents. The [open-label, multicenter study](https://www.prnewswire.com/news-releases/argo-biopharma-to-present-positive-phase-ii-updated-results-of-sirna-therapeutic-bw-20805-for-hae-at-bradykinin-symposium-2026-302869556.html) enrolled adults with HAE type 1 or 2 at sites across multiple countries.
Safety across all 25 dosed participants was unremarkable in the ways that matter most for a prophylactic drug: no treatment discontinuations, no withdrawals, no deaths, and no participant met protocol hepatotoxicity thresholds. Injection-site reactions were the most frequently flagged adverse event of special interest, and most events were mild. That hepatic safety signal, or rather the absence of one, will draw attention given that RNA-silencing therapies targeting the liver have historically been watched closely on that dimension. The HAE prophylaxis market has expanded in recent years, with the FDA approving garadacimab ([Andembry](https://dermsquared.com/news-research/fda-approves-monthly-andembry-hereditary-angioedema)) for adults and adolescents aged 12 and older in 2025, so Argo will need Phase III data to differentiate on both efficacy durability and dosing convenience.
The number to track next is whether the 300 mg every-24-weeks arm holds its attack-free rate past Day 169. A single six-month dosing interval with three quarters of patients attack-free is a commercially relevant claim, but it rests on eight patients. A Phase III enrollment decision will clarify how much confidence the company places in that dose over the higher one.
*Source link: *
**Categories:** News
---
### [WCG releases checklist to speed protocol amendment implementation at clinical trial sites](https://www.clinicaltrialvanguard.com/eclinical-vendor-watch/wcg-releases-checklist-to-speed-protocol-amendment-implementation/)
**Published:** September 3, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Protocol amendments add three months to clinical trials on average. WCG's checklist helps sponsors streamline implementation and reduce site-level delays.
**Content:**
Three-point-three amendments per protocol, on average, across trials that get amended at all, and [76% of trials now require at least one](https://www.precisionformedicine.com/blog/the-amendment-trap-why-76-of-clinical-trials-face-six-figure-protocol-changes), up from 57% in 2015. That arithmetic compounds fast: each change triggers a cascade of IRB submissions, site retraining, informed consent revisions, and vendor updates, none of which happen in parallel by default. WCG published a [protocol amendment implementation checklist](https://www.wcgclinical.com/insights/protocol-amendment-checklist-for-sponsors/) this week aimed at shortening the gap between an approved amendment and a site actually enrolling patients under the new version, and the gap it’s addressing is real enough that it deserves attention beyond the press-release cycle.
The operational problem is sequencing. Sponsors often treat amendment approval as the finish line when it’s closer to the starting gun for site-level work. Under [21 CFR 312.30](https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-312/subpart-B/section-312.30), a sponsor must submit a protocol amendment to the IND before implementing any change that affects subject safety or trial conduct, and sites cannot proceed until the relevant IRB has also approved. In practice, that dual-track requirement means a site receiving an amendment package still faces local review, new training attestations, and revised consent forms before a single patient can be enrolled under the new protocol. Without a coordinated task list, each step waits for the previous one to close rather than running concurrently where regulations permit.
WCG’s checklist structures those tasks by phase: pre-submission planning, regulatory filing, site notification, training, and consent re-execution. The value isn’t novelty; it’s codification. Most experienced CROs have internal versions of this workflow, but smaller sponsors and first-time trial teams often reconstruct it from scratch with each amendment. Phase III protocols average 3.5 substantial amendments per the [Tufts CSDD 2024 analysis](https://intuitionlabs.ai/articles/clinical-trial-protocol-amendments-cost-data), which means a late-stage program can run this gauntlet multiple times in a single development cycle. Each iteration where implementation takes weeks longer than it should delays enrollment windows, pushes interim analyses, and in competitive indications can matter to a program’s commercial timing.
The metric worth watching in any trial affected by a substantial amendment is how long it actually takes each site to enroll a first patient under the revised protocol after IRB approval lands. That site-level lag, not the sponsor-to-IND submission time, is where most of the delay accumulates, and it’s the number a checklist like this one is designed to compress.
The checklist itself is a [free two-page download](https://www.wcgclinical.com/wp-content/uploads/2026/09/Sponsor-Site-Protocol-Amendment-Checklist-A-1.pdf), aimed at sponsors rather than sites, and organized around five readiness areas: amendment announcement, the amendment package, budget, communication, and implementation. What WCG has not published is outcome data. There is nothing on whether sites whose sponsors follow the checklist actually reach first-patient-enrolled faster under an amended protocol, or by how much. Those numbers would show whether the checklist closes the gap it targets; WCG has an open invitation to share them.
*Source link: *
**Categories:** eClinical Vendor Watch
---
### [Fortrea's $45M Early Phase Deal Sets a 90-Day Test for Sponsors With Active Studies](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/fortreas-45m-early-phase-deal-sets-a-90-day-test-for-sponsors/)
**Published:** September 3, 2026
**Author:** Krishma Shah
**Excerpt:** Fortrea's $45M buy of Worldwide Clinical Trials' Early Phase Services unit means active sponsors face contract novation, lab method transfer, and timeline risk now.
**Content:**
Picture a clinical pharmacology study mid-enrollment. The CPU beds are occupied, the bioanalytical lab has validated methods running on a platform the team spent four months qualifying, and the sponsor’s project manager has a CRA report due Friday. Then the announcement drops: [Fortrea is acquiring Worldwide Clinical Trials’ Early Phase Services division for approximately $45 million in cash](https://www.fiercebiotech.com/cro/fortrea-boosts-early-phase-platform-45-million-cro-acquisition), including Worldwide’s Clinical Pharmacology Unit and its bioanalytical laboratory operations. That Friday CRA report just became the least urgent document in the queue.
For sponsors with active programs running through Worldwide’s early phase infrastructure, the [deal announced September 2, 2026](https://ir.fortrea.com/news-releases/news-release-details/fortrea-acquire-clinical-pharmacology-unit-and-bioanalytical) is not background news. Contract obligations, study timelines, bioanalytical method ownership, and operational counterpart relationships are all in motion simultaneously. The window to get ahead of each one is measured in weeks, not quarters.
## [](#the-contract-layer-nobody-moves-fast-enough-on)The Contract Layer Nobody Moves Fast Enough On
The first operational question is deceptively simple: who is the legal counterparty to your Master Service Agreement and work orders after close? CRO acquisitions transfer operational assets, but they do not automatically novate contract obligations to the acquiring entity unless the original agreement contains assignment provisions permitting it. Sponsors who signed MSAs with Worldwide Clinical Trials need to pull those agreements now and read the assignment clause. If the clause requires sponsor consent to assignment, that consent is a negotiating moment, not a formality.
Work order novation is where the operational drag actually lives. In acquisitions structured as asset purchases rather than stock purchases, individual work orders may need to be terminated and reissued under the acquiring entity’s legal name to maintain enforceability. Any sponsor team that has navigated a mid-study CRO transition knows the documentation cascade this triggers: updated signature pages, budget amendments reflecting the new entity, revised wire instructions, and updated IRB and ethics committee correspondence naming the new service provider. Phase I studies running at Worldwide’s CPU need that checklist started before close, not after.
Fortrea already operates Phase I clinical research units in Dallas, [Daytona Beach (an 88-bed purpose-built facility conducting early-phase trials since 1993](https://www.fortrea.com/sites/default/files/2026-08/daytona-beach-research-unit.pdf)), and Madison, according to [Fortrea’s published locations](https://www.fortrea.com/about-us/locations). Adding Worldwide’s Clinical Pharmacology Unit expands that footprint, but expansion does not eliminate the integration period during which staff, SOPs, and quality systems are harmonized. That period is exactly when active studies absorb the most operational friction.
## [](#the-bioanalytical-lab-is-the-highest-risk-transfer)The Bioanalytical Lab Is the Highest-Risk Transfer
GLP bioanalytical laboratory capacity is moving from Worldwide to Fortrea. For sponsors whose samples are currently in Worldwide’s bioanalytical pipeline, this is the most technically consequential element of the deal, and the one most likely to be underestimated by anyone who has not lived through a lab-side facility transition.
The FDA’s [Bioanalytical Method Validation guidance, published May 2018](https://www.federalregister.gov/documents/2018/05/22/2018-10926/bioanalytical-method-validation-guidance-for-industry-availability), governs what happens when a validated method moves between laboratories or analytical systems. Full revalidation is not always required, but the guidance is explicit that method transfers must demonstrate comparability between sending and receiving sites using pre-defined acceptance criteria. In practice, this means the sponsor’s regulatory strategy team needs to understand which of their validated assays are affected, whether Fortrea’s integrated lab will run on the same analytical platform, and what a cross-validation package for each method would require if the platforms differ.
None of that work is fast. Bioanalytical method transfer timelines can extend significantly when instruments match, and longer when they do not, depending on the complexity of the assay and the degree of platform overlap between sites. A Phase I PK study cannot report primary endpoints until the bioanalytical data are validated and the method is confirmed transferable. If a sponsor’s study timeline does not account for that window, the clinical report delivery date is already slipping.
There is regulatory history worth knowing here. An FDA Form 483 was issued to Worldwide Clinical Trials Early Phase Services, LLC following an [October 2018 inspection](https://www.redica.com/document-store/documents/view/100092673/worldwide-clinical-trials-early-phase-services-llc-form-483-2018-10-17). That inspection predates current ownership: Worldwide has subsequently been recapitalized through [Kohlberg and Company](https://www.kohlberg.com/worldwide-clinical-trials-completes-recapitalization-with-kohlberg-company/). But any sponsor conducting due diligence on the integration should request Fortrea’s plan for harmonizing Worldwide’s quality management system into Fortrea’s existing QMS, including how open CAPAs and prior inspection findings will be addressed and closed under the new organizational structure. That question belongs in the next governance call, not deferred until Fortrea’s first post-acquisition audit.
## [](#what-sponsors-should-verify-before-close)What Sponsors Should Verify Before Close
The practical move for any sponsor clinical operations team with an active Worldwide early phase contract is a rapid internal audit of three things: the legal assignment terms in the MSA, the list of validated bioanalytical methods currently in use and the platform they run on, and the named project team contacts on the Worldwide side who will need operational continuity during Fortrea’s integration.
That last point matters more than it sounds. Phase I studies depend on tight communication loops between the sponsor clinical pharmacology team and the CPU’s operational staff. If key Worldwide personnel exit during integration, the institutional knowledge about study-specific deviations, subject safety signals, and database query resolutions walks out with them. Sites I work with in Phase II and III contexts lose enormous ground when coordinator turnover breaks those loops. The risk is identical at a CPU when a CRO acquisition reshapes the team. Sponsors should ask Fortrea directly, and soon, which Worldwide staff are being retained and in what roles.
For sponsors whose studies are far enough along that close is unlikely to disrupt enrollment, the near-term focus should narrow to three deliverables: a confirmed contract novation plan with a signed timeline, a written bioanalytical method transfer and comparability protocol accepted by both parties, and a named operational lead at Fortrea responsible for continuity. Any of those three missing at close is a protocol deviation waiting to be written.
Fortrea’s integration execution in the next ninety days will tell sponsors whether this [$45 million acquisition](https://www.investing.com/news/company-news/fortrea-to-acquire-worldwides-early-phase-unit-for-45-million-93CH-4885852) adds reliable early phase capacity to the CRO market or exports a year of operational turbulence to the studies already running inside it. The CPU beds do not care which company owns them. The samples in the bioanalytical queue do.
## [](#references)References
1. [Fierce Biotech, “Fortrea boosts early phase platform with $45 million CRO acquisition”](https://www.fiercebiotech.com/cro/fortrea-boosts-early-phase-platform-45-million-cro-acquisition)
2. [Fortrea, “About Us: Locations”](https://www.fortrea.com/about-us/locations)
3. [Federal Register, “Bioanalytical Method Validation Guidance for Industry: Availability” (May 22, 2018)](https://www.federalregister.gov/documents/2018/05/22/2018-10926/bioanalytical-method-validation-guidance-for-industry-availability)
4. [Kohlberg & Company, “Worldwide Clinical Trials Completes Recapitalization with Kohlberg & Company”](https://www.kohlberg.com/worldwide-clinical-trials-completes-recapitalization-with-kohlberg-company/)
5. [Redica Systems, Worldwide Clinical Trials Early Phase Services LLC, FDA Form 483, October 17, 2018](https://www.redica.com/document-store/documents/view/100092673/worldwide-clinical-trials-early-phase-services-llc-form-483-2018-10-17)
**Categories:** Clinical Trial Ops Brief
**Tags:** Clinical Trial Ops Brief, CRO Consolidation, Phase I Operations, site activation, study startup
---
### [NSABP B-59 Failed. The Real Question Is Why We Keep Designing Trials That Can't Detect the Signal.](https://www.clinicaltrialvanguard.com/article/article-deep-dive/nsabp-b-59-failed-the-real-question-is-why-we-keep-designing-trials-that-cant-detect-the-signal/)
**Published:** September 3, 2026
**Author:** Moe Alsumidaie
**Excerpt:** The NSABP B-59/GeparDouze trial enrolled 1,550 TNBC patients and missed its primary endpoint. Atezolizumab's Phase 3 failure reveals a biomarker stratification…
**Content:**
Picture the moment a data safety monitoring board releases its primary endpoint analysis on a trial that enrolled [1,550 patients across 353 sites](https://communities.springernature.com/posts/immunotherapy-for-early-stage-triple-negative-breast-cancer-the-nsabp-b-59-gepardouze-trial) in the United States, Germany, Canada, and Spain. The investigators behind [NSABP B-59/GeparDouze](https://www.nature.com/articles/s41591-026-04565-6) had every structural reason for optimism: a Phase 3 randomized design co-led by two of the most rigorous cooperative groups in oncology, a PD-L1 checkpoint inhibitor that had already shown pCR improvement in an earlier trial, and a patient population with a disease so aggressive that even incremental gains matter enormously. The board reads the numbers. [Atezolizumab does not meet its primary endpoint](https://communities.springernature.com/posts/immunotherapy-for-early-stage-triple-negative-breast-cancer-the-nsabp-b-59-gepardouze-trial) in early triple-negative breast cancer. The trial fails.
That failure carries weight far beyond the TNBC space. Every immuno-oncology sponsor running a Phase 3 program with a PD-L1 inhibitor, in any indication, should be reading the B-59 readout and asking a harder question than “did it work?” The harder question is: did we design a trial that was even capable of detecting the population where it works?
## [](#the-biomarker-problem-nobody-wants-to-name)The Biomarker Problem Nobody Wants to Name
To understand what went wrong, you have to go back to the predecessor data. The IMpassion031 trial, published in *The Lancet*, showed that atezolizumab added to nab-paclitaxel and anthracycline-based chemotherapy significantly improved pathological complete response rates in early-stage TNBC. That result was real enough to generate serious enthusiasm. But pCR improvement in a Phase 2 or earlier Phase 3 population does not automatically translate to event-free survival or overall survival benefit in a broader, larger, more heterogeneous cohort — and that gap is precisely where B-59 fell apart.
The structural issue is PD-L1 as a stratification instrument. Research published in *Frontiers in Immunology* confirms that PD-L1 expression can predict objective response rate and two-year overall survival in PD-L1-positive metastatic TNBC patients receiving checkpoint inhibitors. But “can predict” and “reliably stratifies a 1,550-patient trial with sufficient power” are two different claims. TNBC is not immunologically uniform. Researchers have identified at least three distinct TNBC immune subtypes — IS1, IS2, and IS3 — [with significant differences in prognosis, gene mutation profiles, immune infiltration levels, and drug sensitivity](https://pmc.ncbi.nlm.nih.gov/articles/PMC8593253/). A trial that enrolls all three subtypes and treats PD-L1 positivity as the primary biomarker stratifier is not enrolling one immunological population. It is enrolling three, then averaging the result.
Averaging is how you miss signals.
A single-cell RNA sequencing analysis of 9,683 tumor-infiltrated immune cells isolated from 14 treatment-naive TNBC tumors identified 22 distinct immune cell subsets — including novel T cell sub-populations not previously characterized in standard PD-L1 assays. That level of microenvironmental complexity is invisible to a binary PD-L1 positive/negative stratification scheme. Sponsors who design trials around PD-L1 expression status alone are drawing a map that leaves most of the territory blank.
## [](#keynote-522-won-b-59-lost-the-difference-is-instructive)KEYNOTE-522 Won. B-59 Lost. The Difference Is Instructive.
The contrast with pembrolizumab’s trajectory in the same indication is unavoidable, and it is uncomfortable for anyone who wants to attribute B-59’s failure purely to mechanism-of-action differences between atezolizumab and pembrolizumab. The [Phase 3 KEYNOTE-522 study of neoadjuvant pembrolizumab](https://www.jhoponline.com/issue-archive/2022-issues/april-2022-vol-12-no-2/pembrolizumab-plus-chemotherapy-for-neoadjuvant-and-adjuvant-therapy-in-early-triple-negative-breast-cancer-keynote-522) plus chemotherapy followed by adjuvant pembrolizumab in high-risk early-stage TNBC [reported results at ASCO 2026 confirming its long-term event-free survival benefit](https://ascopubs.org/doi/10.1200/JCO.2026.44.16_suppl.507). Pembrolizumab targets PD-1. Atezolizumab targets PD-L1. Both are checkpoint inhibitors working on the same axis. Both were tested in early TNBC. One succeeded as a registrational trial; one did not.
The mechanistic argument is real — PD-1 blockade and PD-L1 blockade have different immunological profiles in the tumor microenvironment — but the more operationally important difference may be trial design architecture. KEYNOTE-522 included adjuvant pembrolizumab continuation after surgery. B-59’s design choices, enrollment scope, and stratification strategy produced a diluted signal across an immunologically mixed population. Blaming the molecule before interrogating the trial architecture is the kind of convenient shortcut that sends the next generation of immuno-oncology programs down the same path.
Which raises the question every sponsor should be sitting with right now: if we ran B-59 again with IS3-enriched enrollment, tumor-infiltrating lymphocyte counts as co-stratification variables, and an adaptive interim that allowed biomarker-guided subgroup enrichment, would we get a different answer?
## [](#what-the-protocol-should-have-done-differently)What the Protocol Should Have Done Differently
The [FDA finalized its guidance on adaptive designs for clinical trials of drugs and biologics in December 2019](https://www.federalregister.gov/documents/2019/12/02/2019-25986/adaptive-designs-for-clinical-trials-of-drugs-and-biologics-guidance-for-industry-availability). That guidance explicitly defines adaptive design as a trial that “allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial.” Seven years later, large cooperative group oncology trials are still being designed as rigid, fixed-enrollment studies with a single biomarker stratification layer — and then failing in ways that adaptive interim analyses might have caught or corrected mid-course.
For a trial like B-59, with 1,550 patients enrolled across four countries and a known biomarker heterogeneity problem in TNBC, the protocol should have specified a prospectively planned biomarker-enrichment interim. At 50% enrollment, with emerging immune subtype data, the trial design could have triggered a population refinement — not a subgroup fishing expedition, but a pre-specified adaptive strategy that narrows the treatment population to the IS2 and IS3 subtypes where checkpoint inhibitor activity concentrates. That is not a post-hoc rationalization. That is what the FDA’s 2019 adaptive design guidance was written to enable.
Instead, the trial enrolled broadly, stratified on PD-L1 alone, and diluted the treatment effect across three immune subtypes with meaningfully different tumor microenvironments. The result is a negative primary endpoint that tells us less about atezolizumab’s actual ceiling in TNBC than it does about the limits of trial architecture that was state-of-the-art in 2015.
Sponsors building immuno-oncology programs today need to make two concrete changes to their protocol development process. First, PD-L1 expression status should be treated as a necessary but insufficient stratification variable — tumor-infiltrating lymphocyte density, immune subtype classification (IS1/IS2/IS3), and pre-treatment stromal TIL counts should be included as co-stratification factors with prospective power calculations that account for subgroup heterogeneity. Second, any Phase 3 immunotherapy trial enrolling more than 800 patients in an indication with known immune subtype variability should include a prospectively planned, FDA-aligned adaptive biomarker enrichment interim — documented in the IND before first patient in, not added as a protocol amendment after disappointing interim signals.
Those are not theoretical improvements. The infrastructure for both exists. The willingness to write them into protocols before enrollment starts is the variable that separates programs that generate regulatory knowledge from programs that generate large, expensive negative datasets.
## [](#references)References
1. [Nature Medicine — “Atezolizumab in early triple-negative breast cancer: the randomized phase 3 NSABP B-59/GeparDouze trial”](https://www.nature.com/articles/s41591-026-04565-6)
2. [Springer Nature Communities — “Immunotherapy for early-stage triple-negative breast cancer: the NSABP B-59/GeparDouze trial” (enrollment and site demographics)](https://communities.springernature.com/amp/posts/immunotherapy-for-early-stage-triple-negative-breast-cancer-the-nsabp-b-59-gepardouze-trial)
3. [PubMed/The Lancet — IMpassion031 trial: atezolizumab in early-stage TNBC, pathological complete response findings](https://pubmed.ncbi.nlm.nih.gov/32966830/)
4. [Frontiers in Immunology — PD-L1 as predictive biomarker for checkpoint inhibitors in metastatic TNBC](https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2023.1060308/full)
5. [ASCO Publications — KEYNOTE-522 final analysis, neoadjuvant pembrolizumab plus chemotherapy in high-risk early-stage TNBC](https://ascopubs.org/doi/10.1200/JCO.2026.44.16_suppl.507)
6. [bioRxiv — “A Single-Cell Immune Atlas of Triple Negative Breast Cancer Reveals Novel Immune Cell Subsets” (9,683 tumor-infiltrated immune cells, 14 TNBC tumors)](https://www.biorxiv.org/content/10.1101/566968v1.full)
7. [PMC/NIH — “Immune Classification and Immune Landscape Analysis of Triple-Negative Breast Cancer” (IS1, IS2, IS3 subtypes)](https://pmc.ncbi.nlm.nih.gov/articles/PMC8593253/)
8. [FDA via CASRAI — “Adaptive Designs for Clinical Trials of Drugs and Biologics: Guidance for Industry,” finalized December 2019](https://casrai.org/guides/adaptive-design-clinical-trials)
**Categories:** Article: Deep Dive
**Tags:** Adaptive Trial Design, Addiction Immunotherapy, Atezolizumab, Biomarker Stratification, Triple-Negative Breast Cancer
---
### [PMV Pharmaceuticals Structures Warrant Offering Around Rezatapopt NDA Acceptance](https://www.clinicaltrialvanguard.com/news/pmv-pharmaceuticals-structures-warrant-offering-around-rezatapopt-nda-acceptance/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** PMV Pharmaceuticals ties warrant pricing to rezatapopt NDA acceptance, betting on FDA review of 46% response rate data in TP53 Y220C ovarian cancer.
**Content:**
A 46% overall response rate in a notoriously difficult-to-treat population is the number driving PMV Pharmaceuticals’ boldest regulatory bet yet. Among 76 evaluable patients with platinum-resistant or refractory ovarian cancer harboring a TP53 Y220C mutation, [rezatapopt produced 35 responders](https://ir.pmvpharma.com/news-releases/news-release-details/pmv-pharmaceuticals-announces-updated-promising-rezatapopt), including four confirmed complete responses, assessed by RECIST 1.1 criteria. That efficacy profile is now the engine behind an NDA the company is preparing to submit, and the FDA’s decision on whether to accept that application for review is consequential enough that PMV has written it directly into the financial mechanics of an ongoing warrant offering.
The SEC filing is a 424B5 prospectus supplement tied to a common stock warrant raise, and buried in the pricing terms is a structural tell: the exercise price of the warrants drops to the greater of 33% of the initial exercise price or a defined floor, automatically, on the date PMV publicly announces FDA acceptance of the rezatapopt NDA. That is not routine warrant language. It is a built-in dilution event contingent on a single regulatory milestone, which means the company is essentially pricing investor risk around whether FDA accepts the filing at all. Acceptance is not approval, but it is the gate that confirms the agency finds the package sufficiently complete to begin a substantive review.
The clinical context matters here. Platinum-resistant ovarian cancer is a setting where options remain limited and heavily mutation-specific. [Mirvetuximab soravtansine (Elahere) received full FDA approval in March 2024](https://www.cancer.gov/news-events/cancer-currents-blog/2024/fda-elahere-platinum-resistant-ovarian-cancer) for patients with FRα-positive disease, demonstrating that the agency will move on biomarker-selected populations with compelling single-arm data. Rezatapopt targets a completely different molecular subset, TP53 Y220C, a mutation that destabilizes the p53 protein’s folding rather than simply disabling its transcriptional function. PMV’s drug works by stabilizing the mutant conformation, a mechanism with no close approved analogue. Rezatapopt already carries [Fast Track designation](https://www.marketbeat.com/stocks/NASDAQ/PMVP/fda-events/) for this indication, which accelerates agency interactions but does not guarantee a favorable review clock.
The one number worth tracking now is the NDA acceptance date itself. A Refuse to File letter would collapse the warrant price trigger, freeze the program’s momentum, and force PMV to refile, likely costing six months or more. Acceptance, by contrast, flips the dilution mechanism live and signals that the 46% ORR dataset, whatever its limitations in a single-arm design, passed FDA’s threshold for substantive engagement. That decision, expected within 60 days of submission, is the closest thing this program has to a binary event before any advisory committee or approval action.
*Source link: *
**Categories:** News
---
### [Nanochon Enrolls First Patient in Chondrograft Knee Cartilage Study](https://www.clinicaltrialvanguard.com/news/nanochon-enrolls-first-patient-in-chondrograft-knee-cartilage-study/)
**Published:** September 3, 2026
**Author:** Jon Napitupulu
**Excerpt:** Nanochon enrolls first patient in Chondrograft knee cartilage study, a 3D-printed implant designed for focal chondral defects with immediate mechanical support.
**Content:**
Cartilage regeneration has resisted durable solutions for decades, and the global knee cartilage repair market sits at roughly $2.06 billion in 2025 precisely because nothing on it fully solves the problem. Against that backdrop, Nanochon has enrolled and treated its first patient in the First-in-Human study of [Chondrograft, its 3D-printed patented implant for focal chondral defects of the knee](https://www.prnewswire.com/news-releases/nanochon-performs-first-case-in-the-chondrograft-first-in-human-clinical-study-302867243.html). The procedure was performed at The Panama Clinic by regenerative sports medicine specialists Dr. Juan Osorio and Dr. Emilio Tufiño, and the speed of that first enrollment signals something substantive: patient demand in this category is not theoretical.
The study targets patients aged 22 to 60 who have exhausted conservative treatment and present with one or two femoral condyle and/or trochlear cartilage lesions. Approved options in this space exist, including the [Agili-C implant, which received FDA Premarket Approval in March 2022](https://www.prnewswire.com/news-releases/fda-approves-cartiheals-implant-for-the-treatment-of-cartilage-and-osteochondral-defects-301513667.html), but the clinical literature has long documented that current approaches rarely restore cartilage with the mechanical properties of native tissue and often require prolonged rehabilitation. Chondrograft is designed to provide immediate mechanical support through a minimally invasive, bone-sparing technique, positioning it explicitly as a “pre-replacement” option for patients who need more than temporary relief but are not yet joint-replacement candidates.
That positioning matters strategically for the trial design as much as for the market. The FIH study is prospective and safety-focused, built to generate the data foundation for a subsequent Level 1 multi-center, randomized, controlled pivotal trial. Nanochon already holds FDA Breakthrough Device Designation, which shortens the feedback loop with regulators and strengthens the case for the accelerated path to that pivotal study. The designation does not guarantee approval or shorten clinical timelines, but it does signal that FDA has recognized the unmet need the device addresses, which carries real weight when negotiating trial design and endpoints.
The number to track from this point forward is lesion fill and patient-reported outcome data from this FIH cohort. If Chondrograft demonstrates durable tissue integration at follow-up, that single dataset determines whether the pivotal randomized trial launches on a credible mechanistic story or has to rebuild one. The [knee cartilage repair market is projected to reach $3.50 billion by 2034](https://www.fortunebusinessinsights.com/industry-reports/knee-cartilage-repair-market-100260), and the companies that capture meaningful share will be those whose pivotal data arrive first with structural endpoints regulators trust. Nanochon’s FIH clock is now running.
*Source link: *
**Categories:** News
---
### [Veeva Launches Falcon Safety AI for Adverse Event Processing Across E2B Systems](https://www.clinicaltrialvanguard.com/news/veeva-launches-falcon-safety-ai-for-adverse-event-processing-across-e2b-systems/)
**Published:** September 2, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Veeva Falcon Safety will streamline adverse event processing across E2B-compliant safety systems, including Oracle Argus and ArisGlobal LifeSphere.
**Content:**
Pharmacovigilance teams have long been forced to choose: adopt a vendor’s end-to-end stack or stitch together brittle integrations between safety systems. Veeva’s bet with [Falcon Safety](https://www.unite.ai/veeva-falcon-safety-automates-adverse-event-intake-across-e2b-systems/), announced September 1, is that the second path doesn’t have to be painful, and that position is the strategic provocation buried inside an otherwise tidy product launch. Falcon Safety is an agentic safety operations product for running adverse event intake, case processing, and follow-up tracking across any E2B-compliant safety system, including Oracle Argus and ArisGlobal LifeSphere MultiVigilance, not just Veeva Safety. That interoperability design is a direct signal to the installed base: you don’t have to rip out your existing platform to access the automation layer.
The timing matters. The [pharmacovigilance software market, valued at roughly $207 million in 2023, is projected to reach $372 million by 2032](https://www.snsinsider.com/reports/pharmacovigilance-and-drug-safety-software-market-2379) at a 6.76% CAGR, a growth rate that reflects steady regulatory pressure rather than explosive adoption. That pressure is intensifying: regulators, including the FDA through its draft AI guidance framework, are actively shaping how autonomous agents can be used to support drug safety reporting decisions. Veeva is inserting Falcon Safety into that environment before the compliance requirements around agentic AI in pharmacovigilance fully crystallize, which is either smart positioning or a race ahead of regulatory clarity, depending on your risk appetite.
Falcon Safety is part of Veeva AI, the company’s industry-specific AI solutions, and early adopter availability is scheduled for November 2026. The product’s agentic architecture is the operationally interesting part: rather than AI-assisted workflows where humans still drive each case step, Falcon Safety can operate autonomously across intake channels, reducing the manual coordination burden that traditionally scales poorly as case volumes grow. For pharmacovigilance operations teams at small-to-mid-size biopharma companies, where headcount constraints make per-case processing costs acute, the automation argument is straightforward. For larger organizations with deeply embedded legacy systems, the E2B compatibility claim is the real test, and that proof will arrive in early customer deployments.
Watch the November early adopter cohort closely. Whether Veeva can demonstrate clean E2B interoperability at production scale, specifically against Argus deployments, will determine whether Falcon Safety expands Veeva’s footprint inside existing safety infrastructures or remains a feature benefit primarily for Veeva Safety customers.
*Source link: [https://www.prnewswire.com/news-releases/veeva-falcon-safety-to-work-with-all-e2b-compliant-safety-systems-across-any-intake-channels-302865792.html ](https://www.prnewswire.com/news-releases/veeva-falcon-safety-to-work-with-all-e2b-compliant-safety-systems-across-any-intake-channels-302865792.html)*
**Categories:** News
---
### [The EMA Just Opened a Door That Animal Testing Has Kept Closed for Decades](https://www.clinicaltrialvanguard.com/opinion/the-ema-just-opened-a-door-that-animal-testing-has-kept-closed-for-decades/)
**Published:** September 2, 2026
**Author:** Moe Alsumidaie
**Excerpt:** EMA's September 2026 NAM data pilot lets sponsors share organ-chip and computational data directly with regulators, reshaping preclinical drug approval…
**Content:**
Picture a toxicologist at a mid-size European biotech, staring at a dataset from a human Liver-Chip that correctly flagged drug-induced liver injury in [87% of cases where animal models had given the all-clear](https://euroocs.eu/emulate-liver-chip-able-to-correctly-identify-87-of-drugs-that-caused-drug-induced-liver-injury-to-patients/). The data is compelling. The science is published in *Nature Communications Medicine*. And until September 2026, she had no formal channel to put that data in front of the EMA in a way that could actually count. She could run the animal studies in parallel, generate the required package, and file the NAM data as a footnote, or she could simply leave it in a drawer. The EMA’s new [voluntary data submission (VDS) pilot for New Approach Methodologies](https://www.ema.europa.eu/en/news/voluntary-data-submission-pilot-advance-innovative-alternatives-animal-testing) changes that calculus entirely.
The [pilot, launched this month](https://www.ema.europa.eu/en/news/voluntary-data-submission-pilot-advance-innovative-alternatives-animal-testing), creates a structured pathway for sponsors to submit data generated using NAMs, including organs-on-chip, human tissue models, and computational toxicology tools, directly to EMA reviewers outside of any specific marketing authorization application. The purpose is explicit: build a regulatory evidence base for methods that “have the potential to provide information comparable to, or better than, current testing approaches.” That is not the language of accommodation. That is the language of replacement.
## [](#the-machine-nobody-explained)The Machine Nobody Explained
To understand why this pilot matters, you have to understand the trap that has paralyzed NAM adoption for the better part of two decades. Regulators require validated methods. Validation requires data. Data requires regulatory use cases. And regulatory use cases require, in turn, validated methods. The loop closes on itself, and sponsors caught inside it have had two rational choices: run the animal studies and move on, or spend years in scientific advice meetings trying to establish precedent one molecule at a time.
The EMA’s existing framework under [Directive 2010/63/EU](https://www.ema.europa.eu/en/human-regulatory-overview/research-development/ethical-use-animals-medicine-testing) commits the agency to the 3Rs principles: replace, reduce, and refine animal use in medicine testing. The directive’s stated ambition is to replace all animal research with non-animal methods where scientifically justified. But ambition without infrastructure is a policy document, not a regulatory pathway. The VDS pilot is the infrastructure.
Here is how the mechanism actually works. A sponsor generates NAM data using, say, an organ-on-chip system or a virtual control arm built from historical patient data. Under the old paradigm, that data could inform internal decisions but carried almost no weight in a submission unless the specific methodology had been formally qualified. Qualification is a multi-year process through the CHMP’s Joint Expert Group on 3Rs, and as of March 31, 2026, the [CHMP had only just issued a draft qualification opinion](https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-advice-protocol-assistance/qualification-novel-methodologies-medicine-development) for a NAM using virtual control arms. One methodology. One opinion. Draft status.
The VDS pilot breaks the sequence. Sponsors submit NAM datasets voluntarily, outside any live application, and EMA reviewers assess them against existing guidelines, including the agency’s own [2017 “Guideline on the principles of regulatory acceptance of 3Rs](https://www.ema.europa.eu/en/regulatory-acceptance-3r-replacement-reduction-refinement-testing-approaches-scientific-guideline) testing approaches” (EMA/CHMP/CVMP/JEG-3Rs/450091/2012). The reviewers document what the data demonstrates, where it falls short, and what additional evidence would be needed to support regulatory acceptance. That feedback becomes part of a growing institutional knowledge base, and each submission incrementally de-risks the methodology for the next sponsor who uses it.
Call this the principle of accumulated regulatory memory. The VDS pilot treats NAM evidence not as a binary pass/fail qualification event but as an iterative learning process, where each data package narrows the uncertainty that makes reviewers hesitant to accept novel methods in live applications.
The Emulate human Liver-Chip validation published in December 2022 illustrates exactly what is at stake. That study showed [87% sensitivity for drug-induced liver injury](https://www.thebts.org/wp-content/uploads/2023/09/Opportunities-for-the-application-of-the-3Rs-in-toxicology-studies.pdf), detecting compounds that had passed conventional animal testing. The specificity figure matters too: the chip correctly cleared compounds that animals had flagged as concerning. Animals are not a gold standard against which NAMs are measured. In many toxicological domains, they are the baseline that NAMs are outperforming.
## [](#where-the-precedent-breaks-down)Where the Precedent Breaks Down
Consider a concrete scenario. A sponsor developing a hepatotoxic compound in Phase I has Liver-Chip data showing a clear safety signal at a dose where rat studies showed no effect. Under current GCP and ICH S7A guidelines, the rat data is what goes into the IND. The chip data is, at best, a supporting exhibit. At worst, it creates a disclosure problem: the sponsor knows something the regulatory framework cannot yet process.
That asymmetry has real consequences for trial design. If sponsors cannot trust that NAM signals will be weighted appropriately by regulators, they have no financial incentive to generate them systematically. The cost of running both a Liver-Chip study and the required animal battery is additive, not substitutive. The VDS pilot, by creating a formal feedback loop, begins building the regulatory confidence that eventually makes the chip study substitutive rather than supplementary.
The CHMP’s draft qualification opinion from March 2026 on [virtual control arms](https://intuitionlabs.ai/articles/digital-twins-clinical-trials-virtual-control-arms) offers a parallel preview of that process in action. Virtual control arms use historical patient-level data to construct a synthetic comparator, potentially eliminating or shrinking the placebo arm in randomized trials. The draft opinion does not grant blanket acceptance. It specifies the conditions under which the methodology is considered fit for purpose, the disease areas where the evidence is strongest, and the data quality thresholds required. That is exactly the kind of granular, conditional guidance that sponsors need to build compliant protocols, and it took years of accumulated scientific advice interactions to produce it.
The VDS pilot accelerates that process across multiple NAM types simultaneously, rather than qualifying one methodology at a time through the bottleneck of formal opinion procedures.
## [](#the-skeptics-have-a-point)The Skeptics Have a Point
Not everyone reads the pilot as unambiguously progressive. The [Society of Toxicology, in a May 2025 statement](https://www.ema.europa.eu/en/news/voluntary-data-submission-pilot-advance-innovative-alternatives-animal-testing), urged government agencies to “maintain flexibility” in safety and risk assessment policies as the field shifts toward non-animal methods. The SOT’s position is not anti-NAM. It is a warning against the kind of regulatory overreach that mandates replacement before the evidentiary base is deep enough to support it. The concern is legitimate: a voluntary pilot that generates positive NAM data from well-resourced sponsors using cutting-edge platforms could create implicit pressure on regulators to accept methods that have not yet been validated across the full diversity of compounds, disease areas, and laboratory conditions that drug development demands.
Sponsors running trials in rare diseases with small patient populations, where animal models are already poorly predictive and NAMs have even less validation data, could find themselves caught between a regulatory expectation of non-animal evidence and a scientific reality that cannot yet deliver it at the required confidence level. The [VDS pilot’s voluntary structure](https://www.ema.europa.eu/en/news/voluntary-data-submission-pilot-advance-innovative-alternatives-animal-testing) is the right architecture for this moment. The risk arrives if voluntary becomes normative before the science catches up.
What the EMA has built, carefully and deliberately, is a mechanism for learning without commitment. The VDS pilot does not promise that NAM data will replace animal testing in any specific application. It promises that NAM data submitted now will shape what the agency knows and expects five years from now. For sponsors who are already generating this data internally, the choice to participate is obvious: submit it, get feedback, and own the precedent. The sponsors who sit out the pilot will be reading its conclusions in someone else’s regulatory briefing document.
## [](#references)References
1. [European Medicines Agency — “Voluntary data submission pilot to advance innovative alternatives to animal testing” (September 2026)](https://www.ema.europa.eu/en/news/voluntary-data-submission-pilot-advance-innovative-alternatives-animal-testing)
2. [European Medicines Agency — “Ethical use of animals in medicine testing” (Directive 2010/63/EU and 3Rs framework)](https://www.ema.europa.eu/en/human-regulatory-overview/research-development/ethical-use-animals-medicine-testing)
3. [PMC / CHMP — “Guideline on the principles of regulatory acceptance of 3Rs testing approaches” (EMA/CHMP/CVMP/JEG-3Rs/450091/2012, adopted February 24, 2017)](https://pmc.ncbi.nlm.nih.gov/articles/PMC12988525/)
4. [Emulate Bio / Nature Communications Medicine — “Organ-Chip Validation Study: 87% Sensitivity for Drug-Induced Liver Injury” (December 2022)](https://emulatebio.com/press/nature-publication-validates-organ-chips-for-predictive-toxicology-announcement/)
5. [Society of Toxicology — “SOT Statement on the Use of Animals in Research and Testing” (May 1, 2025)](https://toxchange.toxicology.org/blogs/cynthia-rider1/2025/05/01/sot-statement-on-the-use-of-animals-in-research-an)
**Categories:** Article: Opinion
**Tags:** Animal Testing Alternatives, EMA, FDA Regulatory Science, New Approach Methodologies, Preclinical Development
---
### [Medtronic acquires distribution rights to Cornerstone Robotics' Sentire surgical system for $700M](https://www.clinicaltrialvanguard.com/news/medtronic-acquires-distribution-rights-to-cornerstone-robotics-sentire-surgical-system-for-700m/)
**Published:** September 2, 2026
**Author:** Jon Napitupulu
**Excerpt:** Medtronic acquires distribution rights to Cornerstone Robotics' Sentire surgical system for $700M to expand robotic surgery access in non-U.S. markets.
**Content:**
Medtronic is writing a $700 million check for a Hong Kong robotics startup most of its surgeons have never heard of, and the logic only makes sense when you read the fine print on what it already owns. The investment in Cornerstone Robotics comes bundled with distribution rights for the Sentire surgical system across select non-U.S. markets where the device holds clearance, positioning Medtronic to accelerate the commercial rollout of a second robotic platform even as its own Hugo system is still gaining regulatory footing in key geographies. Hugo received [FDA clearance for urologic procedures only in December 2025](https://news.medtronic.com/2025-12-03-Medtronic-announces-FDA-clearance-of-Hugo-TM-robotic-assisted-surgery-system-for-urologic-surgical-procedures), leaving a broad multispecialty gap that Sentire, cleared in China in 2024 and granted CE mark and Singapore Health Sciences Authority approval in May 2026 across general, gynecologic, thoracic, and urologic indications, can fill immediately.
Medtronic’s own framing is telling: it described Sentire as something that “complements” Hugo rather than competes with it, which is either precise positioning or a convenient way to avoid admitting it is carrying two robotic platforms into the same operating theaters. Either way, the strategic arithmetic works. Cornerstone brings a vertically integrated architecture, developing its own hardware, control software, algorithms, and imaging and energy platforms in-house, a design philosophy that gives Medtronic a more resilient supply chain than a purely licensed technology would. [Published retrospective data on Sentire](https://pmc.ncbi.nlm.nih.gov/articles/PMC12236983/) showed comparable clinical outcomes to the da Vinci system in robot-assisted radical prostatectomy alongside improved cost-effectiveness, which is the combination that moves hospital procurement committees in cost-sensitive markets.
The geography here is the real signal. Cornerstone’s regulatory clearances in the EU and Singapore arrived just three months ago, meaning commercial traction is essentially zero and distribution infrastructure is the bottleneck. Medtronic’s network across more than 150 countries solves that problem faster than Cornerstone could on its own, and the $700 million gives Cornerstone capital to continue its three-center global R&D operation and expand beyond its existing 30,000-square-meter manufacturing facility in China. Robotic surgery adoption remains low worldwide relative to total surgical volume, and the unaddressed demand is concentrated precisely in the markets where Sentire is now cleared.
The single variable worth tracking closely is the pace of Sentire’s CE-mark commercial launches in Europe, specifically whether Medtronic converts its distribution rights into active sales agreements with health systems before Hugo’s own European multispecialty clearance path catches up. If Sentire generates meaningful procedure volume in EU markets within the next 12 months, it validates Medtronic’s dual-platform bet; if it stalls, the $700 million looks more like a defensive land grab than a growth strategy.
*Source link: *
**Categories:** News
---
### [Girl with Griscelli Syndrome Type II undergoes first haploidentical transplant at Miami hospital](https://www.clinicaltrialvanguard.com/news/girl-with-griscelli-syndrome-type-ii-undergoes-first-haploidentical-transplant-at-miami-hospital/)
**Published:** September 2, 2026
**Author:** Jon Napitupulu
**Excerpt:** Girl with Griscelli Syndrome Type II receives first haploidentical transplant at Miami hospital, offering new hope for ultra-rare genetic disease.
**Content:**
One in a million is not a figure of speech here: [Griscelli Syndrome Type II affects roughly one in every million children](https://www.prnewswire.com/news-releases/father-to-daughter-bone-marrow-transplant-gives-daughter-a-second-chance-at-life-302866842.html), and without a bone marrow transplant most do not survive past age five. Victoria was diagnosed at two months old, received a haploidentical transplant from her father at twelve months, and returned home in July 2026 at two years old, considered cured of the hemophagocytic lymphohistiocytosis (HLH) that had compounded her already lethal underlying disorder. The transplant, performed at Nicklaus Children’s Hospital in Miami, is the first of its kind at that institution and among only a few documented cases in the United States.
The clinical complexity here deserves attention. Victoria faced two simultaneous life-threatening processes: the primary genetic defect in RAB27A-driven immune regulation that defines GS2, and a secondary HLH cascade in which the immune system turns destructive against the body’s own organs. HLH carries high acute mortality, and pharmacological options, while they exist ([emapalumab gained FDA approval in 2018 for refractory primary HLH](https://www.fda.gov/drugs/fda-approves-emapalumab-hemophagocytic-lymphohistiocytosis)), do not address the underlying genetic defect driving recurrence. Only transplant does. The haploidentical approach, using a half-matched donor rather than waiting for a fully matched unrelated donor, is particularly consequential for ultra-rare diseases where the urgency of HLH leaves almost no time to search registries. Her father was immediately available; the transplant team at Nicklaus acted on that window.
The broader clinical signal is in the transplant design itself. [Published outcomes data from a 35-patient GS2 cohort](https://pubmed.ncbi.nlm.nih.gov/32286505/) represent essentially the entire published experience with HSCT in this disease, illustrating just how thin the evidence base is. Haploidentical transplantation expands the donor pool for children who cannot wait, but it introduces graft-versus-host risk and requires sophisticated conditioning, which is why the Nicklaus team’s depth in complex pediatric transplants was the operative variable. The program describes itself as South Florida’s most experienced pediatric blood and marrow transplant provider, and this case will add a rare data point to a literature that desperately needs more of them.
The marker to watch is long-term engraftment durability. Victoria’s HLH is considered resolved, but multi-year follow-up reporting from cases like hers, added to the existing GS2 transplant literature, will be what ultimately shapes haploidentical protocols for children who present with compounded rare immune disorders and no time to spare.
*Source link: *
**Categories:** News
---
### [Elevar Presents Age-Stratified Camrelizumab-Rivoceranib Data After FDA Rejection](https://www.clinicaltrialvanguard.com/news/elevar-presents-age-stratified-camrelizumab-rivoceranib-data-after-fda-rejection/)
**Published:** September 2, 2026
**Author:** Jon Napitupulu
**Excerpt:** Elevar presents age-stratified camrelizumab-rivoceranib data from Phase 3 CARES-310 after two FDA rejections, signaling potential resubmission strategy.
**Content:**
Two Complete Response Letters from the FDA in roughly two years should have quieted the camrelizumab-rivoceranib story, but Elevar Therapeutics is betting that a granular post hoc cut of its [Phase 3 CARES-310 data](https://pubmed.ncbi.nlm.nih.gov/41308676/) can reopen that conversation. The analysis heading to the International Liver Cancer Association conference this week focuses on younger patients with unresectable hepatocellular carcinoma, an age-stratified subgroup breakdown drawn from a trial that already showed the combination extended median progression-free survival to 5.6 months against 3.7 months for sorafenib in the overall population. That 1.9-month delta was not enough to carry an NDA across the finish line: [the FDA issued its most recent CRL in July 2026](https://www.targetedonc.com/view/fda-passes-again-on-approving-rivoceranib-plus-camrelizumab-in-hcc), a second rejection for this filing.
The strategic logic of presenting an age-subgroup analysis after a regulatory setback is not subtle. Elevar is signaling that the CARES-310 dataset contains signal the agency has not fully weighed, and that a clinically meaningful PFS benefit holds across all age subgroups is the specific claim being put on the record at a peer audience. Whether that matters to regulators depends entirely on what the CRL cited: if the FDA’s objection was manufacturing, trial conduct, or safety data rather than efficacy, an age-stratified PFS poster does essentially nothing to move the ball. If the objection touched on population-level generalizability, this data could anchor a resubmission argument.
The competitive backdrop makes the timing more complicated. First-line unresectable HCC is no longer a thin field. Atezolizumab plus bevacizumab [has been standard of care since 2020](https://www.asco.org/practice-policy/policy-issues-statements/asco-in-action/fda-approves-atezolizumab-plus-bevacizumab), and [nivolumab plus ipilimumab earned a first-line approval in April 2025](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-ipilimumab-unresectable-or-metastatic-hepatocellular-carcinoma). Each new approved regimen raises the implicit bar for differentiation that a resubmission would need to clear, not on efficacy alone but on the question of where this combination fits in an increasingly crowded sequence.
The single number worth tracking now is the timeline and stated basis of any resubmission to the FDA. If Elevar files and specifies which CRL deficiency the new data package addresses, that narrows the probability of a third rejection considerably. If no resubmission timeline emerges after the ILCA presentation, this conference poster reads less as a regulatory prelude and more as a program in search of a path forward.
*Source link: *
**Categories:** News
---
### [PMV Pharma's Rezatapopt Achieves 44% Response Rate in TP53 Y220C Ovarian Cancer](https://www.clinicaltrialvanguard.com/news/pmv-pharmas-rezatapopt-achieves-44-response-rate-in-tp53-y220c-ovarian-cancer/)
**Published:** September 2, 2026
**Author:** Jon Napitupulu
**Excerpt:** PMV Pharma's rezatapopt achieves 44% response rate in TP53 Y220C ovarian cancer patients, positioning the drug for potential FDA accelerated approval discussion
**Content:**
A 44% objective response rate in heavily pretreated ovarian cancer patients is the number PMV Pharmaceuticals is betting its regulatory future on. Updated interim data from the Phase 2 pivotal portion of the [PYNNACLE trial](https://ir.pmvpharma.com/news-releases/news-release-details/pmv-pharmaceuticals-announces-updated-promising-rezatapopt), with a May 14, 2026 data cutoff, show rezatapopt monotherapy delivering that response rate in patients whose tumors carry a TP53 Y220C mutation, a genetically defined subset that has historically had few targeted options and is typically managed with chemotherapy until progression. That figure, in a single-arm registrational cohort, is the direct evidence base PMV will carry into an FDA discussion about whether this data package supports accelerated approval.
The clinical logic here is unusually clean for an oncology program. [Rezatapopt](https://www.pmvpharma.com/pipeline/) is an orally available small molecule that binds a hydrophobic pocket created specifically by the Y220C mutation, refolding misshapen p53 protein back toward functional conformation. Because the drug’s target is the mutation itself rather than a downstream pathway, patient selection is binary: tumors either carry Y220C or they do not. That precision cuts enrollment noise and, critically, gives regulators a biomarker-defined population in which to interpret single-arm response data without a randomized comparator arm. The registrational design across five tumor-type cohorts in PYNNACLE reflects PMV’s read that the FDA is receptive to this framework in genomically selected populations.
What the interim data do not yet resolve is durability. Response rate in a snapshot dataset tells you tumors shrank; it does not tell you for how long. The ovarian cancer cohort in a single-arm trial will face scrutiny on progression-free and overall survival curves before any label claim about clinical benefit can be made definitive. PMV is betting that the Y220C selection is tight enough, and the unmet need deep enough in this line of therapy, to clear an accelerated pathway on response alone while confirmatory data mature. That is a reasonable bet, but it is still a bet.
The number to track from here is not the response rate itself but the duration of response in the May cutoff dataset. If the median duration of response in the ovarian cohort holds above six months in the full data package submitted to FDA, the accelerated approval argument becomes substantially harder to dismiss.
*Source link: *
**Categories:** News
---
### [Benchmarks Don't Fail Silently. Agentic AI Does.](https://www.clinicaltrialvanguard.com/clinical-bellwether/benchmarks-dont-fail-silently-agentic-ai-does/)
**Published:** September 1, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Scale AI's CliniCARE-Bench found 34.7% error rates and right-answer-wrong-process failures. The clinical trials field has no governance framework to catch…
**Content:**
[Francis deSouza](https://www.linkedin.com/posts/francisdesouza_clinicare-bench-clinical-ai-agents-can-be-activity-7498576516369342464-zhcm) published a number this week that deserves more than a LinkedIn like. [Scale AI’s CliniCARE-Bench](https://www.linkedin.com/posts/francisdesouza_clinicare-bench-clinical-ai-agents-can-be-activity-7498576516369342464-zhcm), built on 750 real patient cases and 25 clinical care scenarios, found that the underlying models in agentic clinical systems reached error rates of 34.7%. When Scale required those same systems to be both correct and free of incorrect shortcuts, scores dropped by as much as 14.8 percentage points. The agents were arriving at the right answer without reading the chart. They were skipping the longitudinal record, bypassing conflicting evidence, and presenting a conclusion that looked earned but wasn’t. In a benchmark environment, that’s a finding. In a live trial, it’s a protocol deviation no one detected.
That gap, between a system that scores well and a system a clinician can operationally rely on, is precisely what the clinical trials field has not solved. And right now, sponsors, CROs, and health systems are deploying agentic AI anyway.
## [](#the-governance-deficit-is-structural)The Governance Deficit Is Structural
The benchmark problem would be containable if governance frameworks were keeping pace. They aren’t. [Mo Johnson](https://www.linkedin.com/posts/mo-johnson_in-a-senior-leadership-meeting-an-executive-activity-7500509021863309312-JweM), a cardiothoracic surgeon tracking AI accountability, [cited a February survey of 120 health systems](https://www.linkedin.com/posts/mo-johnson_in-a-senior-leadership-meeting-an-executive-activity-7500509021863309312-JweM) that should stop every AI deployment committee in its tracks: 75% have deployed AI or plan to, 18% have mature governance with a documented strategy and a formal enforcement group, and 42% have neither. Not a lean framework. Nothing structural underneath them at all.
Johnson draws a distinction the industry has been treating as semantic when it is actually operational. Healthcare AI, the administrative layer managing scheduling, billing, and bed flow, carries operational failure costs. Clinical AI, the decision layer covering diagnostic imaging, treatment planning, sepsis prediction, and trial endpoint adjudication, carries patient outcome failure costs. Most governance frameworks treat them identically. That means a health system can deploy an agentic tool that touches protocol eligibility criteria or safety signal detection under the same oversight structure it uses for a revenue cycle automation. The accountability stakes are not remotely comparable.
[Cassandra Chuljian](https://www.linkedin.com/posts/activity-7500276529189490689-2Csm), writing from the regulated industry perspective, made the failure mode explicit: [“the scariest AI agents aren’t necessarily the ones that fail. They’re the ones that seem to work.”](https://www.linkedin.com/posts/activity-7500276529189490689-2Csm) An agent producing reasonable-looking outputs while running on stale data or flawed logic won’t trigger an alert. It won’t generate a deviation report. It will produce a clean-looking record that satisfies an auditor until a patient outcome forces the retrospective audit that uncovers the process failure buried underneath the correct-looking answer. That is silent failure in a regulated environment, and it is the specific failure mode that no current FDA guidance on AI-enabled devices, including the agency’s 2021 action plan for AI and machine learning-based software as a medical device, has operationally resolved for agentic systems operating inside trial workflows.
## [](#process-fidelity-over-benchmark-performance)Process Fidelity Over Benchmark Performance
[Sachin Bajpai](https://www.linkedin.com/posts/sachin-bajpai-379ba11_designing-responsible-ai-agent-systems-for-activity-7499216201810821120-1jHb) published research in the International Journal of Computer this week arguing that the architecture conversation needs to happen before deployment at scale, not during it. [His framework](https://www.linkedin.com/posts/sachin-bajpai-379ba11_designing-responsible-ai-agent-systems-for-activity-7499216201810821120-1jHb), validated against a sepsis-deterioration scenario, separates the thinking layer from the acting layer, matches human oversight to the level of decision risk, and builds the audit trail into the architecture from the start rather than retrofitting it as a compliance checkbox. The third element is the one most clinical operations teams skip. An audit trail added post-deployment is a documentation artifact. An audit trail built into the agent’s decision architecture is an accountability mechanism. Those are not the same thing.
[Sumant Ranji](https://www.linkedin.com/posts/activity-7498445455551287297-KyLh), Director of UCSF’s Coordinating Center for Diagnostic Excellence, framed the deeper epistemological problem by referencing a randomized trial of an LLM-based decision-support system in Kenyan primary care clinics, published in Nature Medicine. [The AI improved documentation of diagnoses and treatment plans](https://www.linkedin.com/posts/activity-7498445455551287297-KyLh), a measurable process outcome, but did not affect the primary clinical outcome of treatment failure. The process improved. The outcome did not move. Ranji connects this to Donabedian’s quality triad and to decades of quality improvement work demonstrating that measurable process gains do not automatically translate into outcome gains. The clinical AI field is at risk of repeating exactly that lesson, this time at the speed of agentic deployment.
This is the counterintuitive claim the industry needs to sit with. Better benchmark performance may actually increase deployment confidence in systems that fail on the dimensions that matter most for patients. A system that scores well on CliniCARE-Bench while bypassing the longitudinal record 14.8% of the time will be approved for broader use faster than a system that scores lower but documents its reasoning at every decision node. The benchmark rewards the outcome. The trial rewards the process. Scale AI is to be credited for building a benchmark that surfaces this tension explicitly, but surfacing it and solving for it in deployment governance are different problems entirely.
## [](#who-answers-when-the-agent-is-wrong)Who Answers When the Agent Is Wrong
[Rubén Lozano Aguilera](https://www.linkedin.com/posts/lozanoaguilera_ai2-the-best-ai-tools-arent-built-for-scientists-activity-7499284110176202753-RKwx), writing from Ai2, made the accountability argument most precisely: [“AI is not a moral agent; it can be reliable or unreliable, but it cannot be trustworthy.”](https://www.linkedin.com/posts/lozanoaguilera_ai2-the-best-ai-tools-arent-built-for-scientists-activity-7499284110176202753-RKwx) Calling an agentic clinical system trustworthy, as vendors routinely do in their deployment materials, displaces the accountability question onto the technology and away from the humans who built it, validated it, and authorized its use in a trial workflow. The Ai2 partnership with Providence Swedish and the Earle A. Chiles Research Institute in Portland offers a more honest model: Asta AutoDiscovery flagged that invasive lobular carcinoma, a breast cancer type historically excluded from immunotherapy trials as immunologically cold, showed more immune activity than previously characterized in the TCGA dataset. The scientists were skeptical, per Lozano Aguilera’s account. They confirmed the signal in an independent patient dataset and in real tumor tissue before committing to a clinical trial. The AI located the signal. The humans defended the inference. That division of accountability is not a limitation of the system; it is the design.
[Ingrid O’Dwyer](https://www.linkedin.com/posts/ingrid-odwyer_clinical-stage-ai-enabled-therapeutic-assets-activity-7498801572764794880-VZfw) at Sanofi noted this week that approximately 117 AI-discovered drugs have now entered clinical trials, with Insilico Medicine’s rentosertib as the most prominent example. [Per O’Dwyer’s read of the data](https://www.linkedin.com/posts/ingrid-odwyer_clinical-stage-ai-enabled-therapeutic-assets-activity-7498801572764794880-VZfw), AI drugs are not outperforming traditional molecules on clinical success rates, but several startups have compressed discovery-to-Phase 1 timelines from the typical 4 to 4.5 years down to 1.5 to 2 years. Speed is real. Superiority has not arrived. That distinction matters because the governance frameworks being built now, while AI drugs are fast but not demonstrably better, will be the same frameworks in place when and if the performance gap closes and the deployment stakes increase further.
Michelle Longmire at Medable pointed to Tufts Center for the Study of Drug Development data showing ROI from agentic AI in oncology trials, [describing the moment as a watershed](https://www.linkedin.com/posts/michellelongmire_as-the-dream-of-the-autonomous-clinical-trial-activity-7499119819682934785-PGSb). It may be. But watersheds are also the moment when downstream infrastructure either holds the load or reveals it was never designed for it. The accountability infrastructure for agentic AI in clinical trials, who owns the failure, what the audit trail must contain, how silent process errors get detected before they compound, has not been designed for the volume of deployment the field is now executing. CliniCARE-Bench gave the industry a precise vocabulary for the failure modes. The 42% of health systems running clinical AI with no governance structure underneath it suggests the vocabulary hasn’t reached the room where deployment decisions are made.
The next regulatory action on AI-enabled clinical tools will not cite benchmark scores. It will cite a patient outcome, a missing audit trail, and a sponsor who could not explain how the agent reached its conclusion. Build the architecture that can answer that question before the question gets asked in a Form 483.
## [](#references)References
1. [Francis deSouza, LinkedIn post on CliniCARE-Bench, Scale AI Labs](https://www.linkedin.com/posts/francisdesouza_clinicare-bench-clinical-ai-agents-can-be-activity-7498576516369342464-zhcm)
2. [Mo Johnson, LinkedIn post on clinical vs. healthcare AI governance, Eliciting Insights survey of 120 health systems](https://www.linkedin.com/posts/mo-johnson_in-a-senior-leadership-meeting-an-executive-activity-7500509021863309312-JweM)
3. [Sachin Bajpai, LinkedIn post on agentic AI architecture, International Journal of Computer](https://www.linkedin.com/posts/sachin-bajpai-379ba11_designing-responsible-ai-agent-systems-for-activity-7499216201810821120-1jHb)
4. [Cassandra Chuljian, LinkedIn post on silent AI failure modes in regulated industries](https://www.linkedin.com/posts/activity-7500276529189490689-2Csm)
5. [Sumant Ranji, LinkedIn post on AI quality measurement and Nature Medicine LLM trial in Kenyan primary care](https://www.linkedin.com/posts/activity-7498445455551287297-KyLh)
6. [Rubén Lozano Aguilera, LinkedIn post on AI trustworthiness and Ai2/Providence Swedish partnership, Asta AutoDiscovery and TCGA dataset](https://www.linkedin.com/posts/lozanoaguilera_ai2-the-best-ai-tools-arent-built-for-scientists-activity-7499284110176202753-RKwx)
7. [Ingrid O’Dwyer, LinkedIn post on AI drug performance in clinical trials, Insilico Medicine rentosertib](https://www.linkedin.com/posts/ingrid-odwyer_clinical-stage-ai-enabled-therapeutic-assets-activity-7498801572764794880-VZfw)
8. [Michelle Longmire, LinkedIn post on agentic AI in oncology trials, Tufts Center for the Study of Drug Development](https://www.linkedin.com/posts/michellelongmire_as-the-dream-of-the-autonomous-clinical-trial-activity-7499119819682934785-PGSb)
**Categories:** Clinical Bellwether
**Tags:** agentic AI, AI Accountability, AI in Clinical Trials, Clinical AI Governance, Digital Health Technology
---
### [SRD-002 Gene Therapy Shows Sustained HFpEF Improvement at 12 Months](https://www.clinicaltrialvanguard.com/news/srd-002-gene-therapy-shows-sustained-hfpef-improvement-at-12-months/)
**Published:** September 1, 2026
**Author:** Jon Napitupulu
**Excerpt:** SRD-002 gene therapy shows sustained HFpEF improvement at 12 months with 80% PCWP normalization from a single intracoronary dose.
**Content:**
A 30% reduction in peak exercise pulmonary capillary wedge pressure at 12 months, sustained from a single intracoronary dose, is not the kind of number a gene therapy program produces in Phase 1 and quietly files away. Medera presented that result for SRD-002 at ESC Congress 2026, alongside a finding that 8 of 10 treated patients met a prespecified threshold for normalization of cardiac filling pressures at the same timepoint. For a disease where the dominant pharmacologic options, including [empagliflozin approved in February 2022](https://www.tctmd.com/news/fda-approves-empagliflozin-treatment-hfpef) for HFpEF, reduce hospitalization risk but do not address the underlying myocardial stiffness driving the condition, a durable hemodynamic signal from a one-time intervention reframes what “treatment” could mean in this space.
The mechanism matters here. [SRD-002 uses an AAV1 vector to deliver SERCA2a](https://www.medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/updated-results-first-in-human-gene-therapy-trial-for-heart-failureb19ebbea), the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump, directly targeting the calcium-handling dysfunction that produces impaired myocardial relaxation. That is not a symptomatic or neurohormonal workaround; it is an attempt to restore the specific molecular machinery that fails in HFpEF. The PCWP normalization rate of 80% at 12 months is clinically notable precisely because elevated exercise PCWP is among the most direct hemodynamic signatures of the disease, and the durability over a year from a single dose is what separates this readout from acute catheterization data.
The statistical caution appropriate here is real. Ten patients is not a powered efficacy trial, and [the relationship between PCWP reduction and hard clinical outcomes in HFpEF remains incompletely characterized](https://academic.oup.com/eurheartj/article/35/44/3103/2293271) in the literature. Whether normalized filling pressures at 12 months translate into reduced cardiovascular death or hospitalization at scale is the question a larger trial must answer. Medera is presenting a signal, not a verdict, and the field has watched promising HFpEF mechanistic hypotheses fail to survive rigorous outcome trials before.
What makes this moment consequential for trial watchers is the endpoint selection precedent. If regulators engage with PCWP normalization as a meaningful intermediate endpoint in HFpEF gene therapy, it shapes the Phase 2 design Medera will need to run next. The single number to track from here is the duration of the PCWP effect: if the 12-month normalization rate holds at 18 or 24 months in any extended follow-up, the durability argument for a one-time gene therapy versus daily oral therapy in HFpEF becomes materially harder to dismiss.
*Source link: *
**Categories:** News
---
### [Stop Writing 'Routine Labs.' Every Lab Line Needs Its Own Coverage Citation](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/stop-writing-routine-labs-every-lab-line-needs-its-own-coverage-citation/)
**Published:** September 1, 2026
**Author:** Krishma Shah
**Excerpt:** A CBC isn't covered because it's routine. It's covered because NCD 190.15 says when. Here's how citation-level MCAs survive audits.
**Content:**
*THE BILLING GRID*
A weekly column on research billing and coverage analysis, by Krishma Shah
A billing compliance officer at a mid-sized academic medical center once pulled a coverage analysis for a Phase II oncology trial and found a single line in the lab section: *Routine labs, SOC.* Twelve procedures. One designation. Zero citations. That MCA (medical coverage analysis, the document that assigns each protocol procedure a payer designation and legal basis) would not survive a contractor audit, and it would not survive an OIG review either. The site had been billing Medicare for those procedures for eight months.
This is not a hypothetical failure mode. It is the most common structural weakness in site-level MCAs, and it persists because “routine” feels like a complete thought. It is not. Routine describes clinical familiarity. It does not describe Medicare coverage. Those are different questions, and conflating them is where research billing liability begins.
## [](#the-unit-of-defense-is-the-cell-not-the-category)The Unit of Defense Is the Cell, Not the Category
A properly constructed MCA assigns a designation, SOC (standard of care, billable to Medicare or insurer when the trial qualifies), RES (research-paid, sponsor cost), or NB/INV (non-billable or invoiceable per the site budget), to each individual procedure at each individual visit. The defensible unit is the visit-grid cell: the same CBC can be SOC at the baseline visit and RES at an unscheduled safety visit driven entirely by protocol, depending on clinical context and what the protocol requires versus what would occur in routine oncology care outside the trial.
When a lab section says “routine labs, SOC” across all visits, the MCA has collapsed dozens of cells into one assertion. An auditor working from the protocol visit schedule and the site’s charge master will reconstruct those cells individually. If your MCA does not show the citation that justifies SOC for each one, the site cannot demonstrate that the billing decision was made deliberately. That is the evidentiary gap that turns a documentation problem into a compliance problem.
## [](#the-lab-ncd-family-is-specific-and-searchable)The Lab NCD Family Is Specific and Searchable
Medicare coverage for laboratory procedures is governed by a family of National Coverage Determinations (NCDs, the national-level Medicare coverage rules issued by CMS). These are not catch-all authorizations. Each NCD specifies which diagnoses or clinical conditions support coverage, what frequency is covered, and in some cases what clinical setting applies.
For a complete blood count, the operative citation is NCD 190.15. It does not cover a CBC because a protocol schedules one. It covers a CBC when the clinical indication aligns with the conditions the NCD enumerates. That distinction matters at every visit. A baseline CBC in an oncology trial where the patient has a qualifying diagnosis and the CBC is not required solely by the protocol may be SOC under NCD 190.15. A CBC scheduled at week 16 only because the protocol requires it for safety monitoring is a different analysis, and the MCA needs to show that analysis was done.
The same logic applies across the lab panel. Comprehensive metabolic panels, liver function tests, coagulation studies, urinalyses, lipid panels, and tumor markers each have their own NCD or LCD (local coverage determination, the regional-level Medicare coverage rule issued by a Medicare Administrative Contractor) that governs when billing is appropriate. Writing a single line for “chemistry panel, SOC” when that panel contains a half-dozen individually covered tests is not a shortcut. It is an undocumented billing decision waiting to be questioned.
## [](#a-worked-example-oncology-trial-baseline-visit)A Worked Example: Oncology Trial, Baseline Visit
Consider a protocol that schedules the following at the baseline visit: CBC with differential, comprehensive metabolic panel (CMP), LDH, and a CA-125 tumor marker. The trial qualifies under NCD 310.1 (the national coverage determination for routine costs in qualifying clinical trials, which applies to trials under IND with therapeutic intent and Medicare-benefit-category items). The site is treating patients with ovarian cancer.
A citation-level MCA for that single visit would work through each test independently. The CBC gets NCD 190.15 and a notation of the qualifying diagnosis. The CMP is analyzed under the applicable LCD for the MAC jurisdiction, because metabolic panels do not have a single national NCD the way CBCs do, coverage is regional, and the analyst needs to pull the right contractor document. LDH in an oncology context is assessed against the relevant LCD for tumor marker testing. CA-125 has specific coverage criteria tied to diagnosis and treatment context that require explicit documentation.
Four tests. Four citations or citation attempts. If one of those tests does not meet SOC criteria at the baseline visit, perhaps the protocol is collecting LDH for pharmacodynamic reasons that have no correlate in standard oncology practice for this indication, it goes to RES, sponsor-paid, and the budget pass later reconciles that against the sponsor’s grant. That is the analysis. It takes longer than writing “routine labs, SOC.” It is also the only version that holds up.
## [](#why-summary-level-mcas-fail-audits-that-citation-level-mcas-survive)Why Summary-Level MCAs Fail Audits That Citation-Level MCAs Survive
The Rush University Medical Center case from 2005 is the reference point that billing compliance officers in academic medicine still cite. The core fact pattern was straightforward: procedures that should have been charged to the research sponsor were billed to Medicare. The MCA structure at the visit level is the primary control that prevents that error from happening systematically, because it forces the coverage question to be answered before the patient is enrolled, not after the claim is filed.
When a contractor auditor reviews research billing, they are not accepting the MCA’s conclusions on faith. They are checking whether the MCA’s stated basis is sufficient to support the designation. A summary-level MCA that says “routine labs, SOC” gives the auditor nothing to evaluate. A citation-level MCA that says “CBC, NCD 190.15, qualifying diagnosis documented, SOC at baseline and cycle 1 day 1 visits; RES at unscheduled safety visits” gives the auditor a position to either accept or rebut. That is the difference between a defensible document and an absent one.
Sites that adopt citation-level MCA construction also find a secondary benefit: the budget reconciliation pass is faster. When the invoiceable section of the MCA is built from the same cell-level analysis, the finance team can price research-paid items against the charge master with the designations already resolved. The budget does not have to re-litigate coverage questions that the MCA should have answered at feasibility.
## [](#what-to-fix-before-the-next-protocol-lands)What to Fix Before the Next Protocol Lands
If your current MCA template has a lab section that accepts free-text category entries without requiring a citation field, the template is the problem. The fix is structural: every lab row in the procedure grid needs a citation column that cannot be left blank. Acceptable entries are an NCD number, an LCD identifier with the MAC jurisdiction, or an explicit RES or NB designation with the rationale. “Routine” is not an entry. It is a flag that the analysis was not done.
The MCA sits on the site-activation critical path alongside the contract and the IRB. Getting it right at feasibility, before the budget pass, before first-patient-in, is the only sequence that protects the site. Fixing it after a claim is filed is compliance remediation. Fixing it before is operations.
*Krishma Shah is Director of Clinical Relations at CliniBiz and co-inventor of BudgetSpark, a coverage-analysis engine that produces citation-level, designation-complete MCAs from the protocol and budget in days, not weeks. If your site or network wants to see one built on your own protocol, visit [budgetspark.com](https://budgetspark.com/) or write to .*
**Categories:** Clinical Trial Ops Brief
---
### [Orexo's $4 Million DOJ Settlement Is Deliberately Vague — and That Vagueness Is the Story](https://www.clinicaltrialvanguard.com/clinops-watchdog/orexos-4-million-doj-settlement-is-deliberately-vague-and-that-vagueness-is-the-story/)
**Published:** September 1, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Orexo AB settled a 6-year DOJ opioid-marketing investigation for $4M, but the press release names no conduct, no victims, no violated statute. That silence…
**Content:**
On [August 31, 2026, Orexo AB issued a press release](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice) announcing a non-binding agreement in principle to settle a [Department of Justice investigation that has been running since 2020](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice). The company would pay [USD 4 million in installments over several years](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice), plus a capped [contingent payment of five percent of net sales](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice) tied to sales thresholds in 2030 and 2031. The release names no specific statute violated. It describes no conduct. It identifies no patient harmed, no prescriber improperly influenced, no marketing material that crossed a legal line. Six years of federal investigation, and the public record is a number and a phrase: “opioid marketing.” That is not transparency. That is its own story.
The DOJ has been watching Orexo since 2020. Six years is a long time. For context, the DOJ’s criminal and civil resolution against Endo Health Solutions, which involved the opioid Opana ER with INTAC, [resulted in $1.536 billion in criminal fines and civil settlements](https://www.justice.gov/archives/opa/pr/opioid-manufacturer-endo-health-solutions-inc-ordered-pay-1536b-criminal-fines-and) and came with a detailed factual recitation of exactly how Endo’s sales force was trained to minimize the drug’s abuse potential in physician conversations. Orexo’s press release contains none of that. Which raises a specific operational question: what did Orexo’s marketing apparatus for Zubsolv, its buprenorphine/naloxone sublingual tablet for opioid use disorder, actually do between 2020 and now that warranted six years of federal scrutiny?
## [](#what-six-years-of-silence-conceals)What Six Years of Silence Conceals
The DOJ investigation opened in 2020, the same year Zubsolv was Orexo’s primary commercial product in the U.S. market. Buprenorphine products occupy a peculiar regulatory position: they are Schedule III controlled substances prescribed to treat opioid dependence, meaning the line between legitimate promotion and off-label or misleading marketing carries direct public-health consequences. An aggressive sales representative overstating Zubsolv’s superiority to Suboxone, or understating diversion risk, is not a minor compliance incident. It is a clinical harm vector.
Orexo’s own regulatory record during this period is not clean. [The FDA issued a second Complete Response Letter for OX124](https://www.fiercepharma.com/pharma/fda-gives-another-thumbs-down-orexos-nasal-spray-opioid-overdose), Orexo’s high-dose naloxone nasal spray for opioid overdose reversal, marking the second rejection in 15 months for the same application. Two CRLs on the same product in 15 months signals a sponsor that is either submitting inadequate data packages or failing to execute FDA’s feedback from the first rejection, sometimes both. Neither reflects an operation with exceptional regulatory discipline. And a company navigating simultaneous FDA rejections and a DOJ opioid-marketing investigation is, by definition, an operation under stress at multiple compliance nodes at once.
Stress at multiple nodes is exactly when the rot in standard operating procedures becomes visible. A sales compliance function that is stretched, or underfunded, or culturally subordinated to revenue targets, will not catch a field representative making a claim that goes three sentences past the approved label. A medical affairs function that is siloed from legal review will approve promotional slide decks with efficacy language that does not survive a 21 CFR Part 202 analysis. None of this requires malice. It requires a sponsor that decided, at some point, that commercial urgency outweighed the overhead of rigorous promotional review. The DOJ investigation suggests that calculus was made.
The non-binding nature of this agreement deserves its own scrutiny. “Non-binding agreement in principle” is legal language for: we have agreed on the shape of a deal but nothing is final. Settlements structured this way sometimes collapse. They sometimes expand when discovery surfaces new conduct during the finalization period. They almost never result in a smaller payment than the one announced. For Orexo, the contingent payment structure tied to 2030 and 2031 net sales thresholds is particularly telling: the DOJ accepted a deal where part of the penalty is conditional on commercial success that has not yet occurred. That is a structurally unusual accommodation, and it suggests Orexo’s negotiators successfully argued that a fixed penalty above $4 million would threaten the company’s viability. Whether the DOJ’s acceptance of that argument was appropriate requires knowing the magnitude of the underlying conduct, which the press release does not tell you.
## [](#the-indictment-the-press-release-refuses-to-write)The Indictment the Press Release Refuses to Write
Orexo was founded in 1995 in Uppsala, Sweden, and built its U.S. commercial presence substantially on Zubsolv. The opioid use disorder market is not a market where promotional misconduct is a technical violation. It is a market where a prescriber who is misled about a buprenorphine product’s risk profile makes a prescribing decision that affects a patient in active addiction recovery. The downstream harm of that decision is not an abstract compliance cost. It is a relapse, a lost job, a family crisis, or worse.
The DOJ knows this. The agency has been aggressive about naming specific conduct in opioid settlements precisely because the public-health stakes justify transparency. The Endo resolution named the training scripts. The Purdue Pharma resolution named the sales territories and the physician targets. The [Mallinckrodt resolution, which involved $1.6 billion](https://oag.ca.gov/news/press-releases/attorney-general-becerra-16-billion-global-settlement-opioid-manufacturer), named the specific claims made about abuse-deterrent formulations. Orexo’s settlement announcement names nothing. That is not because nothing happened. Investigations do not run for six years on a hunch. The absence of named conduct in a voluntary press release means Orexo chose not to disclose it, and no final court order yet compels them to.
That gap is where FOIA requests and court-docket monitoring become mandatory tools. Clinical operations executives at any company that partnered with Orexo in a commercial or co-promotion capacity between 2020 and 2026 should be pulling those records now. If the DOJ’s eventual formal settlement agreement, once finalized, identifies specific promotional practices, those practices may have touched shared commercial infrastructure. The compliance liability does not stop at Orexo’s organizational boundary.
## [](#what-sponsors-must-do-before-the-final-order-lands)What Sponsors Must Do Before the Final Order Lands
The structural read here is not complicated, but it is uncomfortable. The [opioid-marketing enforcement wave that consumed Purdue, Endo, Mallinckrodt, and AmerisourceBergen](https://www.attorneygeneral.gov/blocked/) did not end because regulators ran out of targets. It decelerated because the largest actors were resolved. Orexo, as a smaller Swedish specialty pharma with a single major U.S. opioid-class asset, represents exactly the type of secondary actor that enforcement attention reaches after the headline cases close. There are likely others. Any sponsor running a buprenorphine, methadone, or extended-release naltrexone commercial program in the U.S. during the early 2020s should treat this settlement as a signal to conduct a proactive promotional-practices audit before a CID arrives.
For sponsors in or adjacent to the opioid-class commercial space: commission an independent review of your promotional review committee records from 2019 through 2024. Not a legal-privilege review designed to protect the company. An operational audit designed to find what a DOJ attorney would find. The difference between a $4 million settlement and a $1.5 billion settlement is almost never the underlying conduct. It is how early a company found the problem and how credibly it could demonstrate corrective action before the government came in the door.
For FDA-watchers: the moment the Orexo settlement agreement is finalized and filed, the conduct description in that document will be the most important opioid-marketing compliance data point of 2026. If the DOJ buries it in a consent decree with minimal public docketing, file the FOIA request the same day. The public deserves to know what six years of investigation found, not just what Orexo’s communications team chose to include in a press release timed to a Monday news cycle.
The next test of the system is the finalized settlement document itself, which must survive DOJ approval and, if structured as a False Claims Act resolution, requires court filing that creates a public record. Watch the docket. The [contingent payment clause tied to 2030 and 2031 sales thresholds](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice) means this case will not be fully closed for another five years, and every Zubsolv sales quarter between now and then is a data point in a federally supervised commercial compliance story that Orexo has not yet fully told.
## [](#references)References
1. [PR Newswire / Orexo AB — “Orexo reaches non-binding agreement in principle to settle investigation by US Department of Justice” (August 31, 2026)](https://www.prnewswire.com/news-releases/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice-302865649.html)
2. [Orexo AB — Official press release, DOJ settlement agreement in principle, terms and contingent payment structure](https://www.orexo.com/media/pressrelease/orexo-reaches-non-binding-agreement-in-principle-to-settle-investigation-by-us-department-of-justice)
3. [U.S. Department of Justice — “Opioid Manufacturer Endo Health Solutions Inc. Ordered to Pay $1.536B in Criminal Fines and Civil Settlements”](https://www.justice.gov/archives/opa/pr/opioid-manufacturer-endo-health-solutions-inc-ordered-pay-1536b-criminal-fines-and)
4. [Fierce Pharma — “FDA gives another thumbs down to Orexo’s nasal spray for opioid overdose” (second CRL for OX124)](https://www.fiercepharma.com/pharma/fda-gives-another-thumbs-down-orexos-nasal-spray-opioid-overdose)
5. [Orexo AB — Corporate history and product platform overview](https://orexo.com/about-orexo/)
**Categories:** Clinops Watchdog
**Tags:** CLINOPS WATCHDOG, DOJ Settlement, Opioid Marketing, Pharma Compliance, Regulatory Accountability
---
### [Nature Medicine's 38,753-Patient HF Synthesis Signals a Replicable Model for RWE-Driven Label Expansion Without New RCTs](https://www.clinicaltrialvanguard.com/opinion/nature-medicines-38753-patient-hf-synthesis-signals-a-replicable-model-for-rwe-driven-label-expansion-without-new-rcts/)
**Published:** September 1, 2026
**Author:** Moe Alsumidaie
**Excerpt:** A 38,753-patient cross-trial synthesis in Nature Medicine maps short-term safety of HF combination therapies—signaling how sponsors can use pooled RCT data to…
**Content:**
On July 14, 2026, *Nature Medicine* published a [cross-trial synthesis examining short-term effects of combination heart failure therapies on blood pressure, kidney function, and serum potassium](https://www.nature.com/articles/s41591-026-04623-z) across [38,753 participants enrolled in nine landmark heart failure trials](https://www.researchgate.net/journal/Nature-Medicine-1546-170X). The study’s primary regulatory value is methodological: it demonstrates how individual participant data pooled from completed randomized controlled trials can generate safety signal resolution at a precision level that no single Phase 3 program achieves alone.
This matters operationally because the FDA’s 2023 guidance framework on real-world evidence (“Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products,” December 2023, available at fda.gov/media/171667/download) explicitly endorses the use of pre-specified, transparently reported observational and data-integration methods to reduce uncertainty in regulatory submissions. The *Nature Medicine* synthesis, while built on trial data rather than claims or registry data, fits the evidentiary architecture FDA has described as acceptable for supplementary submissions and label revisions.
## [](#what-the-synthesis-actually-demonstrates)What the Synthesis Actually Demonstrates
The study pooled individual participant data from nine heart failure trials to characterize the short-term hemodynamic and metabolic consequences of initiating and combining guideline-directed medical therapies, specifically ARNIs, beta-blockers, mineralocorticoid receptor antagonists (MRAs), and SGLT2 inhibitors. The central safety question was whether specific two-, three-, or four-drug combinations produce acute reductions in systolic blood pressure, acute kidney injury signals, or hyperkalemia that would contraindicate initiation in patients with borderline baseline values.
Across the [38,753-participant dataset](https://www.eureflect.com/new-tool-predicts-heart-failure-treatment), the synthesis found that SGLT2 inhibitor combinations with ACE inhibitor or ARNI-based regimens produced a measurable but clinically bounded reduction in estimated glomerular filtration rate in the first four to twelve weeks, after which kidney function stabilized or improved relative to non-SGLT2 arms. This is consistent with findings from the [EMPEROR-Reduced trial, where empagliflozin’s early eGFR dip](https://pubmed.ncbi.nlm.nih.gov/35711093/) was reversible and not associated with downstream acute kidney injury events at rates that exceeded placebo, a result corroborated by [post-hoc analyses cited by *Pharmacy Times*](https://www.pharmacytimes.com/view/sglt-2-inhibitors-associated-with-reduced-hyperkalemia-in-patients-with-heart-failure) confirming SGLT2 inhibitors’ net protective profile on potassium when used alongside MRAs.
On blood pressure, the synthesis generated subgroup-level precision that individual trials could not. In patients with a baseline systolic blood pressure below 100 mmHg, triple-therapy initiation (ARNI plus beta-blocker plus MRA) produced a mean systolic reduction that the authors flagged as a clinically significant signal meriting protocol-level attention. Notably, [FDA’s February 2022 draft guidance on blood pressure monitoring](https://www.clinicalleader.com/doc/fda-draft-guidance-blood-pressure-monitoring-in-clinical-trials-0001) in clinical trials, as reported by *Clinical Leader*, specifies that study designs should be powered to exclude a 3-mmHg increase in 24-hour average systolic blood pressure using the upper bound of a two-sided 95% confidence interval. The *Nature Medicine* analysis applied directionally analogous precision thresholds to the downward blood pressure risk, giving sponsors a methodological precedent for framing hypotension risk in future submissions.
## [](#implications-for-sponsors-with-active-hfref-programs)Implications for Sponsors With Active HFrEF Programs
Sponsors currently holding INDs for heart failure combination regimens, or those planning label expansion submissions for existing HFrEF-approved agents, face a specific evidentiary gap: guideline bodies recommend quadruple therapy (ARNI, beta-blocker, MRA, SGLT2 inhibitor), but individual trial protocols were not designed to characterize the safety of simultaneous initiation across all four drug classes in patients with marginal hemodynamic or renal tolerance. A December 2025 systematic review and network meta-analysis published in *JACC* and cited by the American College of Cardiology confirmed that quadruple therapy delivers an all-cause mortality hazard ratio of 0.39 (95% CI: 0.32–0.49) versus placebo, but that mortality benefit was derived from trials run sequentially rather than as a simultaneous initiation protocol. The short-term safety profile of simultaneous initiation remained under-characterized.
The *Nature Medicine* synthesis directly addresses that gap, and sponsors should read it as a template rather than a standalone result. The methodology, pre-specified pooling of individual participant data from nine trials with harmonized covariate definitions, transparent handling of differential follow-up windows, and subgroup stratification by baseline renal function and blood pressure, maps onto the FDA’s stated requirements for individual patient data meta-analyses used to support labeling language. Specifically, FDA’s real-world evidence guidance framework (December 2023) cites pre-registration, transparent methodology, and adequate sample size as the three conditions under which integrated analyses can reduce confounding bias sufficiently to inform regulatory language.
For sponsors with Phase 3 HFrEF programs that did not enroll patients with eGFR below 30 mL/min/1.73 m² or systolic blood pressure below 95 mmHg, the synthesis provides external, peer-reviewed quantification of risk in those excluded subpopulations. That quantification could support a labeling negotiation with FDA’s Division of Cardiology and Nephrology rather than a new dedicated safety study, which would require three to four additional years and a patient population that is, by definition, difficult to recruit.
Site networks and CROs managing heart failure combination studies should note that the synthesis’s blood pressure signal in the sub-100 mmHg baseline subgroup creates a de facto floor for future protocol inclusion criteria. Investigators running open-label extension studies or registry sub-studies using quadruple-therapy initiation protocols will face increased IRB scrutiny if they enroll patients below that threshold without pre-specified safety monitoring rules aligned with the synthesis’s identified risk window, which the authors place at weeks two through eight post-initiation.
## [](#a-replicable-methodology-not-a-one-time-dataset)A Replicable Methodology, Not a One-Time Dataset
The broader regulatory signal here is that this synthesis belongs to an emerging category of regulatory evidence: trial-derived, participant-level, cross-program safety analyses that occupy the space between a single-study submission and a full RCT. FDA has not yet published guidance that explicitly defines this category’s evidentiary weight for label revision purposes, but the December 2023 RWE framework’s inclusion of “pre-specified analyses of existing trial data” as an acceptable real-world data input implies sponsors who structure these analyses with FDA’s pre-submission engagement process can use them offensively in Type B meeting requests.
Two recent regulatory precedents reinforce this reading. The FDA’s accelerated approval of finerenone for chronic kidney disease with type 2 diabetes (Kerendia, approved July 2021) relied partly on pre-specified subgroup analyses from the FIDELIO-DKD trial to support labeling language on eGFR thresholds, not a second dedicated trial. Similarly, the [2023 label expansion for dapagliflozin (Farxiga)](https://www.fda.gov/media/117976/download) into a broader heart failure population with preserved ejection fraction drew on pooled safety data from the DELIVER and DAPA-HF trials, allowing a single supplemental NDA rather than a new Phase 3 program. The *Nature Medicine* synthesis scales that methodology to nine trials and 38,753 participants, demonstrating the sample size and analytic precision now achievable through participant-level data harmonization.
For investors monitoring pipeline risk in the heart failure space, the synthesis reduces one category of uncertainty: sponsors with approved SGLT2 inhibitors or ARNIs who wish to pursue label language specifically covering combination initiation in borderline-hemodynamic patients now have a credible external evidence base to cite. That reduces the binary risk of a Complete Response Letter citing insufficient safety characterization in that subpopulation, which has been a recurring concern in CDER Division of Cardiology and Nephrology reviews over the past three years.
The directive for sponsors this quarter is concrete. Any program targeting quadruple-therapy label language or combination-initiation guidance in HFrEF should commission a formal gap analysis against the *Nature Medicine* synthesis’s subgroup definitions. Where the sponsor’s own trial data overlap with the synthesis’s nine contributing datasets, a pre-submission meeting request to FDA’s Division of Cardiology and Nephrology should be filed to determine whether a participant-level data contribution to an analogous synthesis could satisfy the supplemental safety data requirement for a label revision, before committing resources to a new Phase 4 safety study.
The next artifact to watch is FDA’s anticipated guidance on the use of individual participant data meta-analyses in regulatory submissions, which the agency signaled in its 2025 Evidence Action Plan as a priority document for fiscal year 2026. When that guidance publishes, the evidentiary standard the *Nature Medicine* synthesis used will either be formally codified or explicitly constrained, and sponsors who have already built their data packages around this methodology will be positioned to respond within a comment period rather than restructuring their submission strategy from the beginning.
## [](#references)References
1. [Nature Medicine — “Short-term effects of combinations of heart failure therapies on blood pressure, kidney function and serum potassium”](https://www.nature.com/articles/s41591-026-04623-z)
2. [FDA — “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products” (December 2023)](https://www.fda.gov/media/171667/download)
3. [Pharmacy Times — “SGLT-2 Inhibitors Associated with Reduced Hyperkalemia in Patients with Heart Failure”](https://www.pharmacytimes.com/view/sglt-2-inhibitors-associated-with-reduced-hyperkalemia-in-patients-with-heart-failure)
4. [Clinical Leader — “FDA Draft Guidance: Blood Pressure Monitoring in Clinical Trials” (February 2022)](https://www.clinicalleader.com/doc/fda-draft-guidance-blood-pressure-monitoring-in-clinical-trials-0001)
**Categories:** Article: Opinion
**Tags:** Clinical Data Synthesis, FDA Guidance, FDA Real-World Evidence, Heart Failure Trials, Label Expansion
---
### [Astellas donates tacrolimus to U.S. API reserve, strikes pricing deal](https://www.clinicaltrialvanguard.com/news/astellas-donates-tacrolimus-to-u-s-api-reserve-strikes-pricing-deal/)
**Published:** September 1, 2026
**Author:** Jon Napitupulu
**Excerpt:** Astellas donates tacrolimus active pharmaceutical ingredient to U.S. Strategic API Reserve and strikes voluntary Medicaid pricing agreement with government.
**Content:**
Astellas Pharma is donating 25 kilograms of tacrolimus active pharmaceutical ingredient to the U.S. Strategic Active Pharmaceutical Ingredients Reserve, a stockpile formally bolstered by executive order in August 2025, as part of a voluntary pricing agreement with the U.S. government announced August 31. The Medicaid price concessions and the API donation arrive together, and that pairing is the tell: this is not a routine rebate negotiation. It is a coordinated signal to Washington that a major Japanese pharmaceutical company is willing to trade measurable supply-chain assets for political goodwill in an environment where import tariffs and domestic manufacturing mandates are live threats.
The tacrolimus commitment carries real weight. [Tacrolimus has been the cornerstone immunosuppressant for solid organ transplant recipients since its initial FDA approval in 1994](https://www.drugs.com/history/prograf.html), and concentrated API stockpiles are exactly what the Trump administration’s [SAPIR program](https://en.wikipedia.org/wiki/Strategic_Active_Pharmaceutical_Ingredients_Reserve) was designed to accumulate after pandemic shortages exposed generic API dependence on overseas suppliers. Donating 25 kg is not symbolic. It is a quantity with clinical consequence for patients whose supply continuity depends on domestic reserves in a disruption scenario.
The Medicaid pricing component follows an emerging pattern: companies avoiding the formal Medicare drug price negotiation machinery by striking voluntary deals that let both sides claim a win without a mandated ceiling on the books. Astellas did not disclose which specific products face price reductions or the magnitude of those reductions, which limits any precise assessment of the financial hit. What it does confirm is that the company is pricing future medicines with reference to international benchmarks, a concession that has downstream implications for how its pipeline assets, spanning oncology, ophthalmology, and immunology, will be positioned at launch in the U.S. market.
The agreement also highlights Astellas’ U.S. manufacturing footprint, including its cell therapy facility in Westborough, Massachusetts, and its gene therapy plant in Sanford, North Carolina, as relevant context for the deal’s political logic. The single metric worth tracking from here is whether Astellas discloses which Medicaid products are repriced and at what percentage reduction. That number will determine whether this agreement functions as a genuine affordability lever or primarily as a reputational buffer against harder regulatory action.
*Source link: *
**Categories:** News
---
### [Canada's Drug Agency Issues Positive Reimbursement Recommendation for Lecanemab](https://www.clinicaltrialvanguard.com/news/canadas-drug-agency-issues-positive-reimbursement-recommendation-for-lecanemab/)
**Published:** September 1, 2026
**Author:** Jon Napitupulu
**Excerpt:** Canada's drug agency issues positive reimbursement recommendation for lecanemab after reversing its February rejection, opening access for patients with mild co
**Content:**
Canada’s Drug Agency reversed course on lecanemab in roughly five months, flipping from a February 2026 recommendation against public reimbursement to a final positive recommendation issued September 1 — and that reversal now puts Eisai and BioArctic in front of a potential patient population the Alzheimer Society of Canada projects will nearly double, from 770,000 people living with dementia today to 1.7 million by 2050. The speed and completeness of the about-face matters clinically: [CDA-AMC’s initial draft in February was a flat rejection despite Health Canada having approved lecanemab in October 2025](https://guelph.ctvnews.ca/london/article/new-alzheimers-drug-recommendation-offers-hope-patients-and-families/), making this reconsideration outcome a meaningful regulatory correction rather than a routine step forward.
The positive recommendation is not reimbursement. Lecanemab must still pass through pan-Canadian Pharmaceutical Alliance price negotiations before individual provincial and territorial public drug plans make their own coverage decisions, a process that routinely takes a year or longer and often produces uneven access across Canada’s fragmented payer landscape. Patients with mild cognitive impairment or mild dementia due to Alzheimer’s disease — the eligible population named in the recommendation — are the ones who benefit from early intervention, so every month of negotiation delay translates directly into disease progression for people at the precise window where the drug has shown effect.
The global context sharpens the stakes. Lecanemab is now approved in 53 countries, and [the FDA approved a subcutaneous autoinjector formulation in July 2026](https://investors.biogen.com/news-releases/news-release-details/fda-approves-leqembi-iqlikr-lecanemab-irmb-subcutaneous) that allows patients or caregivers to administer the drug at home, a delivery shift that could substantially reduce the infusion-center burden that has constrained real-world uptake elsewhere. Canada’s reimbursement pathway currently points only toward the intravenous formulation, which means the administration complexity that has slowed patient access in other markets will remain a live friction point during negotiations. The AHEAD 3-45 Phase 3 study in preclinical Alzheimer’s, fully recruited as of October 2024, also looms: a positive readout there would expand the eligible population well beyond what today’s recommendation covers, giving payers reason to negotiate hard on price now.
The single variable to track is the pace and geographic breadth of provincial coverage decisions once pan-Canadian negotiations conclude: whether large provinces like Ontario and Quebec move in lockstep or fragment into a patchwork will determine whether Canada’s positive recommendation translates into meaningful population-level access or remains a regulatory milestone without clinical reach.
*Source link: *
**Categories:** News
---
### [When Early Efficacy Data Forces a Site Ramp You Weren't Budgeted For](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/when-early-efficacy-data-forces-a-site-ramp-you-werent-budgeted-for/)
**Published:** August 31, 2026
**Author:** Krishma Shah
**Excerpt:** Aprea's APR-1051 early signals are forcing a rapid site expansion—here's what the enrollment acceleration actually costs at the operational level.
**Content:**
The email lands mid-monitoring visit. Early efficacy signals from the ACESOT-1051 first-in-human study. The sponsor wants to accelerate. New sites, faster enrollment, aggressive timelines. The CRA finishes the monitoring visit report, closes the laptop, and starts doing math that nobody in the budget meeting has done yet.
That is the operational moment Aprea Therapeutics created when it [announced expansion of its WEE1 inhibitor program](https://www.genengnews.com/topics/cancer/aprea-expands-development-of-wee1-inhibitor-after-seeing-early-clinical-activity-signs-vs-cancer/) after early clinical activity signs from [APR-1051. As of May 2026, 28 patients with advanced solid tumors had been enrolled](https://ir.aprea.com/news-releases/news-release-details/aprea-therapeutics-presents-updated-phase-1-data-wee1-inhibitor/) in the Phase 1 trial. The science is moving. The operational infrastructure has to move with it, and that is the part nobody announces in a press release.
## [](#the-gap-between-a-go-decision-and-a-ready-site)The Gap Between a Go Decision and a Ready Site
WEE1 kinase sits at a compelling mechanistic intersection: it regulates the G2/M cell cycle checkpoint, and inhibiting it forces cancer cells, particularly those with p53 mutations and compromised DNA repair, into premature mitosis. The biology rewards urgency. The operational reality does not.
When a sponsor decides to expand site count in oncology, the internal assumption is usually that activated sites follow a predictable ramp curve. In practice, according to a [2024 AACI survey published in JCO Oncology Practice](https://ascopubs.org/doi/10.1200/OP.24.00164), Cancer Center Clinical Trial Offices are already operating under capacity pressure. The survey of North American cancer centers found that most CCTOs are running concurrent protocol loads with limited dedicated startup staff. Adding a new oncology protocol, especially one with biomarker eligibility requirements tied to specific cancer-associated gene alterations, does not slot into existing infrastructure. It competes with it.
The eligibility profile of APR-1051 sharpens the problem. Trials targeting patients with specific molecular alterations in advanced or metastatic solid tumors require sites with active next-generation sequencing workflows, molecular tumor boards, and coordinators who understand how to document biomarker-confirmed eligibility in source records. That narrows the qualifying site universe considerably. A sponsor team planning to add seven sites in one quarter is, operationally, planning to identify seven sites that can do all of this simultaneously, execute a contract and budget cycle from scratch, complete IRB submission and approval, run an SIV, and enroll their first patient, all within 90 days. Sites I work with in oncology routinely see 60 to 90 days from executed contract to first-patient-in under favorable conditions. Unfavorable conditions are the norm.
The data from Servier’s published benchmarks on clinical recruitment makes the stakes tangible: among more than 10,000 recruiting oncologic trials worldwide, 60% enroll fewer than five participants per site, and more than 20% enroll no one at all. Roughly 37% of sites under-enroll, and 30% of principal investigators have never enrolled a single patient in a clinical trial. Expansion plans that assume new sites will hit enrollment velocity within weeks are working against the documented baseline for the entire field.
## [](#where-the-budget-math-breaks-down)Where the Budget Math Breaks Down
The Tufts Center for the Study of Drug Development estimates site activation costs at approximately $1,500 per site per month, with total per-site startup costs running around $25,000 before a single patient is enrolled. Multiply that by seven new sites and you are looking at roughly $175,000 in startup costs alone, none of which appears in a per-patient budget line. Sponsor finance teams building the expansion budget from per-visit fee schedules routinely miss this number entirely.
That structural budget gap matters because expansion decisions triggered by early efficacy signals move faster than budget amendment cycles. The clinops team gets the green light. Contracts are still running on the original study budget assumptions. The new sites need startup funding, screen failure coverage, coordinator onboarding time, and central IRB fees, and those line items either appear in a budget amendment that takes six to eight weeks to execute, or the site absorbs the gap and flags it to the sponsor three months later when enrollment is already behind.
Screen failure economics compound the problem specifically in biomarker-selected oncology trials. When eligibility hinges on confirmed molecular alterations, screen failure rates can run 40 to 60 percent before a single eligible patient is identified and consented. Sites do not get paid for screen failures under most oncology budgets. The coordinator hours, the pharmacy review, the pre-screening lab work: that is real cost that sites carry while the sponsor counts enrolled patients. A 3x or 4x enrollment velocity target does not compress the screen failure rate. It inflates the absolute number of uncompensated site touches required to hit the target.
## [](#what-changes-monday-morning)What Changes Monday Morning
For site directors and clinical operations leads receiving a site activation letter for an expanded oncology trial: do not start the SIV clock until you have a signed budget that explicitly covers screen failure reimbursement, central IRB fees, and coordinator training time for biomarker eligibility review. The pressure to move fast is real, but a site that activates without those line items in the contract will spend the next six months in budget dispute while trying to enroll.
For sponsor clinops teams managing the ramp: the investigator readiness assessment conducted during PSV needs to go beyond the standard PI CV and delegation log review. If the site cannot confirm active NGS turnaround times, documented molecular tumor board access, and coordinator availability specifically for this protocol (not borrowed from an adjacent study), that site will not contribute meaningfully in the first quarter regardless of how fast the contract executes. Adding sites to a site list and activating sites to enrollment capacity are different deliverables, and conflating them is where 3x enrollment projections become 1x reality.
In the trials our network has managed through rapid expansion phases, the sites that ramp fastest are almost always the ones that completed a genuine feasibility reconciliation before the SIV, not the ones that said yes fastest. The distinction matters most in exactly the kind of biomarker-driven, molecularly-selected oncology study that APR-1051 represents.
The early efficacy signal that triggered Aprea’s expansion is the kind of data point every sponsor wants to see in Phase 1. What happens operationally in the next 90 days will determine whether that signal translates into a Phase 2 with clean enrollment data and a functional site network, or a timeline extension and a TMF full of deviation reports. The sites know which outcome they are building toward. The question is whether the expansion plan does.
## [](#references)References
1. [Genetic Engineering News — “Aprea Expands Development of WEE1 Inhibitor After Seeing Early Clinical Activity Signs vs. Cancer”](https://www.genengnews.com/topics/cancer/aprea-expands-development-of-wee1-inhibitor-after-seeing-early-clinical-activity-signs-vs-cancer/)
2. [Aprea Therapeutics — ACESOT-1051 Phase 1 Trial Enrollment Data, May 2026](https://aprea.com/aprea-therapeutics-clinical-trials/)
3. [JCO Oncology Practice / AACI — “North American Cancer Center Clinical Research Capacity and Benchmarking in the Postpandemic Era,” July 2024](https://ascopubs.org/doi/10.1200/OP.24.00164)
4. [Servier — Clinical Studies Patient Recruitment Benchmarks](https://servier.com/en/newsroom/clinical-studies-patient-recruitment/)
**Categories:** Clinical Trial Ops Brief
**Tags:** enrollment acceleration, investigator readiness, Oncology Trials, site activation, WEE1 inhibitor
---
### [When a Failed Primary Endpoint Teaches More Than a Win: What CARDIOTTRansform's Secondary Analysis Reveals About Stratification in Complex Trials](https://www.clinicaltrialvanguard.com/article/trend-watch/when-a-failed-primary-endpoint-teaches-more-than-a-win-what-cardiottransforms-secondary-analysis-reveals-about-stratification-in-complex-trials/)
**Published:** August 31, 2026
**Author:** Moe Alsumidaie
**Excerpt:** CARDIOTTRansform missed its primary endpoint, but its secondary analysis of eplontersen in ATTR-CM patients on background stabilizers is reshaping trial…
**Content:**
The CARDIOTTRansform investigators already knew their Phase 3 trial had not met its primary endpoint when they opened the subgroup data. What they found inside that dataset is quietly forcing a rethink of how cardiomyopathy trials handle concomitant therapy from the moment of randomization, not as an afterthought buried in a sensitivity analysis, but as a foundational design variable. Call it the Stratification Debt Problem: the industry’s habit of treating background therapy as background noise, rather than as a treatment modifier that reshapes the entire efficacy surface of the drug being studied.
The signal is specific. [This secondary analysis of the CARDIOTTRansform Phase 3 trial](https://www.nature.com/articles/s41591-026-04670-6), published in Nature Medicine, examined patients receiving [eplontersen 45 mg subcutaneously every four weeks](https://ir.ionis.com/news-releases/news-release-details/update-cardio-ttransform-phase-3-trial-eplontersen-adults) with or without background transthyretin stabilizers, primarily tafamidis. Across [1,432 randomized participants spanning 130 sites](https://ir.ionis.com/news-releases/news-release-details/update-cardio-ttransform-phase-3-trial-eplontersen-adults) in 20 countries, the trial was powered to detect a composite outcome. The primary endpoint fell short. But the stratified picture that emerged from this secondary analysis tells a structurally different story about who responds, under what conditions, and why the trial’s aggregate signal was almost certainly diluted by a failure to adequately pre-stratify on background therapy status.
## [](#the-concomitant-therapy-blind-spot)The Concomitant Therapy Blind Spot
Transthyretin amyloid cardiomyopathy has a prevalence of [95.8 per 100,000 individuals aged 65](https://www.ahajournals.org/doi/full/10.1161/JAHA.125.047135) and older, [according to data from the US Merative MarketScan Medicare Database](https://www.ahajournals.org/doi/full/10.1161/JAHA.125.047135). That patient population is not treatment-naive. By the time a patient reaches enrollment eligibility in a Phase 3 trial like CARDIOTTRansform, a large share will already be on tafamidis, diflunisal, or another stabilizer, because those agents are standard of care. The question a protocol must answer before randomization is not whether those patients will be in the trial. They will be. The question is whether background stabilizer use will be treated as a stratification variable, a covariate, or a footnote.
CARDIOTTRansform treated it as a stratification variable, which is why the secondary analysis is possible at all. But the operational insight buried here is that pre-specifying a subgroup of this kind in the statistical analysis plan is not the same as designing the trial to be powered within that subgroup. The study enrolled across a 1:1 randomization ratio, eplontersen versus placebo, but the resulting subgroup of patients on background stabilizers was not independently powered to detect the treatment effect that the secondary analysis ultimately found. That gap, between pre-specification and statistical powering, is the core design vulnerability this paper exposes.
The FDA’s own guidance on covariate adjustment, [“Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products,”](https://www.fda.gov/media/148910/download) is clear that stratification variables should be incorporated into the primary analysis model. What the guidance does not resolve is the harder operational question: when a stratification variable like background therapy status is known to be a strong treatment modifier, at what point does it graduate from covariate to design driver, requiring independent powering of the resulting subgroups? CARDIOTTRansform, with its missed primary endpoint and its informative secondary analysis, sits squarely on that unresolved boundary.
## [](#what-the-subgroup-data-actually-shows)What the Subgroup Data Actually Shows
Read the secondary analysis carefully and you find not a rescue narrative, but a precision signal. Patients receiving eplontersen without background transthyretin stabilizers showed a treatment effect pattern meaningfully different from those on concurrent stabilizer therapy. The mechanistic logic is not obscure: tafamidis stabilizes tetrameric transthyretin, while eplontersen reduces the production of transthyretin protein via an antisense oligonucleotide mechanism. These are not redundant mechanisms. In combination, the disease biology is altered in ways that affect the measurable endpoints, particularly cardiac biomarkers and functional capacity outcomes, differently than either agent alone.
That biological interaction is exactly what a trial design has to anticipate, not discover post hoc.
The [Nature Medicine publication](https://www.tandfonline.com/doi/full/10.1080/17582024.2025.2554385) does not position itself as a recovery document. It positions itself as a methodological contribution, and that framing is correct. What this analysis demonstrates is that when you sort the [CARDIOTTRansform population](https://academic.oup.com/eurjhf/advance-article/doi/10.1093/ejhf/xuag168/8698746) by background therapy status, the aggregate null result at the primary endpoint level was carrying two heterogeneous treatment effects that cancelled each other’s signal strength in the pooled analysis. Sponsors designing trials in a crowded standard-of-care environment, where patients arrive pre-treated, need to internalize that lesson before they write their first protocol draft.
The broader pattern here extends well beyond ATTR-CM. In oncology, background checkpoint inhibitor use is now routinely used as a stratification variable. In heart failure, background sacubitril-valsartan status is increasingly treated as a design driver. ATTR-CM trials are arriving at the same inflection point later than they should, given that [tafamidis received FDA approval in 2019](https://www.ahajournals.org/doi/10.1161/CIRCHEARTFAILURE.126.014205) and has been standard of care for nearly seven years. Every ATTR-CM trial launched after 2020 was enrolling into a landscape where background stabilizer use was the norm, not the exception.
## [](#rebuilding-the-protocol-from-the-stratification-up)Rebuilding the Protocol from the Stratification Up
For sponsors currently in the design phase of ATTR-CM trials, the CARDIOTTRansform secondary analysis functions as a retroactive protocol audit. The key operational question it raises: are you enrolling a mixed population and planning to adjust statistically, or are you stratifying at randomization, powering within strata, and pre-specifying stratum-specific primary hypotheses? Those are fundamentally different trial designs, and regulators will evaluate them as such.
The [FDA’s covariate adjustment guidance](https://www.fda.gov/media/148910/download) explicitly states that covariate adjustment models should include stratification variables, but also that the choice between stratified randomization and covariate-only adjustment carries implications for how the primary analysis is interpreted. In a disease like ATTR-CM, where background therapy is both prevalent and mechanistically interactive, relying on post-hoc adjustment without stratified powering creates exactly the kind of diluted primary endpoint result that CARDIOTTRansform produced. The secondary analysis did not save the trial. It explained why the primary analysis structure was insufficient for the population that actually enrolled.
For CROs managing complex cardiomyopathy protocols, the operational implication is harder to fix than it looks. Stratified randomization on background therapy status requires real-time verification of concomitant medications at the point of randomization, a process that demands tighter integration between the RTSM/IRT system, the site’s electronic medical records, and the data management team’s eligibility verification workflow. A randomization system that cannot lock background therapy status at the moment of allocation creates exactly the kind of stratification error the FDA guidance specifically addresses, noting that if incorrect stratification occurs, the covariate adjustment model should use actual baseline strata variables. That correction is possible analytically, but it introduces uncertainty that reviewers will scrutinize.
Technology vendors selling RTSM platforms to oncology and cardiology sponsors should understand that CARDIOTTRansform’s experience represents a commercial pressure point. The next generation of complex cardiomyopathy trials will require randomization platforms capable of handling multi-variable stratification in near real-time, with background therapy status captured from source documentation, not site-reported fields that may lag by days.
The counterintuitive read of this situation is that a failed primary endpoint in a [1,432-patient Phase 3 trial](https://ir.ionis.com/news-releases/news-release-details/update-cardio-ttransform-phase-3-trial-eplontersen-adults) may ultimately do more to advance ATTR-CM trial methodology than a clean positive result would have. A trial that wins with a diluted design teaches no one anything. A trial that misses its primary endpoint and then produces a rigorously pre-specified secondary analysis revealing differential effects by background therapy status forces the next sponsor into a harder, better protocol conversation before the IND goes in, not after the database locks.
Watch for the next ATTR-CM Phase 3 to either power independently within the stabilizer-naive stratum or to restrict enrollment to that stratum entirely. If it does neither, the CARDIOTTRansform secondary analysis will have been read by the wrong people.
## [](#references)References
1. [Nature Medicine — “Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial”](https://www.nature.com/articles/s41591-026-04670-6)
2. [AstraZeneca — “Update: CARDIO-TTRansform Phase III Trial” (enrollment and design details, 1,432 participants, 130 sites, 20 countries)](https://www.astrazeneca.com/media-centre/press-releases/2026/update-cardio-ttransform-phase-iii-trial.html)
3. [FDA — “Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products” (guidance on stratification variable inclusion in primary analysis models)](https://www.fda.gov/media/148910/download)
4. [Journal of the American Heart Association — ATTR-CM prevalence: 95.8 per 100,000 individuals aged 65 and older (US Merative MarketScan Medicare Database, 2022)](https://www.ahajournals.org/doi/full/10.1161/JAHA.125.047135)
5. [European Society of Cardiology — “Eplontersen trial did not meet its primary endpoint in transthyretin-mediated amyloid cardiomyopathy”](https://www.escardio.org/news/press/press-releases/eplontersen-trial-did-not-meet-its-primary-endpoint-in-transthyretin-mediated-amyloid-cardiomyopathy)
**Categories:** Trend Watch
**Tags:** Adaptive Trial Design, ATTR-CM, CARDIOTTRansform, eplontersen, trial stratification
---
### [Ascletis Doses First Patient in ASC30 Phase III Obesity Trial](https://www.clinicaltrialvanguard.com/news/ascletis-doses-first-patient-in-asc30-phase-iii-obesity-trial/)
**Published:** August 31, 2026
**Author:** Jon Napitupulu
**Excerpt:** Ascletis doses first patient in ASC30 Phase III obesity trial, advancing oral GLP-1 agonist against established semaglutide competition through 2028.
**Content:**
With roughly 4,600 participants, two pivotal trials, and a regulatory clock that runs through late 2028, Ascletis has placed a substantial bet that the obesity pill market will be far more crowded and competitive by the time ASC30 reaches an FDA reviewer’s desk. The company announced this week that the first participant has been dosed in AURORA-1 and AURORA-2, its global Phase III program for the once-daily oral small molecule GLP-1 receptor agonist ASC30. That timing matters because [oral semaglutide for weight management already cleared the FDA in December 2025](https://www.ajmc.com/view/fda-approves-oral-semaglutide-as-first-glp-1-pill-for-weight-loss), and Ascletis itself acknowledges ASC30 is positioned to become the second oral small molecule GLP-1 receptor agonist available in the U.S. and Europe, if approved. Entering a market against an established oral incumbent is a meaningfully different competitive posture than entering against injectables alone.
The trial architecture is straightforward but demanding. AURORA-1 enrolls adults with obesity or overweight without type 2 diabetes; AURORA-2 targets the same BMI thresholds in patients who have type 2 diabetes. Both are 72-week, randomized, double-blind, placebo-controlled studies testing three maintenance doses (20 mg, 40 mg, and 60 mg), with percentage change in body weight from baseline as the primary endpoint in each. Sites span the U.S., Europe, and Canada. Topline data are expected in the third quarter of 2028, with an NDA submission targeted by year-end 2028 and an EMA marketing authorization application to follow in early 2029. Ascletis states its current cash position funds operations into 2029, covering both planned regulatory submissions without an intervening financing round.
The Phase II profile Ascletis is carrying into Phase III includes what the company describes as a competitive weight loss signal, favorable gastrointestinal tolerability, and no observed hepatic safety signals. That last point deserves attention: hepatotoxicity concerns have shadowed some small molecule GLP-1 programs, and a clean liver signal through Phase II removes one plausible obstacle to enrollment and regulatory acceptance. The [STEP UP trial showed oral semaglutide 7.2 mg achieving 20.7% mean weight loss at 72 weeks in adherent patients](https://www.appliedclinicaltrialsonline.com/view/step-up-trial-semaglutide-superior-weight-loss), setting a concrete efficacy benchmark that ASC30’s dose-ranging design will need to address credibly at the 60 mg ceiling.
Beyond the monotherapy program, Ascletis is also advancing oral fixed-dose combinations targeting GLP-1/GIP and GLP-1/GIP/amylin, positioning ASC30 as a platform anchor rather than a standalone asset. That pipeline logic only pays off if AURORA delivers on weight loss magnitude. The single number to track between now and Q3 2028 is the 60 mg arm’s percentage body weight reduction: it is the dose most likely to define whether ASC30 can differentiate from a now-entrenched oral competitor or arrive as a follower competing on convenience and tolerability alone.
*Source link: *
**Categories:** News
---
### [Lecanemab's At-Home Start Dose Just Rewrote the Rules for Alzheimer's Trial Design](https://www.clinicaltrialvanguard.com/article/intel-brief/lecanemabs-at-home-start-dose-just-rewrote-the-rules-for-alzheimers-trial-design/)
**Published:** August 30, 2026
**Author:** Moe Alsumidaie
**Excerpt:** FDA's July 2026 approval of at-home subcutaneous lecanemab initiation eliminates the 18-month IV requirement, reshaping CNS trial recruitment and safety…
**Content:**
On July 13, 2026, the FDA did something that Alzheimer’s trial designers have been quietly lobbying for since lecanemab’s first infusion center bottleneck emerged: it [approved a subcutaneous starting dosage regimen for Leqembi (lecanemab-irmb) that patients can initiate at home](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-home-starting-dose-alzheimers-disease-treatment), administered by themselves or a caregiver, without ever sitting in an infusion chair first. That sentence alone dismantles the operational architecture that every anti-amyloid trial has been built around for the past three years.
The consequence for sponsors running or designing lecanemab-adjacent programs is immediate. The [old regimen required 10 mg/kg intravenous infusion every two weeks](https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=9d1ff786-e577-410a-a273-c4d7d0e4e975&type=display), each dose diluted in 250 mL of 0.9% sodium chloride and delivered over approximately one hour, for 18 months before patients could transition to a maintenance schedule. Every one of those visits was a site resource event: a chair, a nurse, a monitoring window, a billing encounter. Multiply that by 1,795 patients enrolled in the CLARITY AD Phase 3 confirmatory trial and the site burden becomes a structural argument against broad commercial rollout. The July approval collapses that model at the front end, where dropout pressure is highest.
## [](#the-operational-fault-line-this-exposes)The Operational Fault Line This Exposes
What the approval actually signals is a regulatory willingness to accept home-initiated biologics in a CNS population that carries one of the most consequential safety monitoring obligations in the drug development portfolio. Lecanemab’s ARIA (amyloid-related imaging abnormality) profile is not theoretical. [The FDA has issued a dedicated Drug Safety Communication recommending baseline MRI within one year before treatment initiation, plus additional earlier MRI monitoring](https://www.fda.gov/drugs/drug-safety-communications/fda-recommend-additional-earlier-mri-monitoring-patients-alzheimers-disease-taking-leqembi-lecanemab) for patients on lecanemab. That requirement does not go away because the injection is now subcutaneous and administered in a living room.
Here is where the approval creates genuine operational friction for trial teams. The FDA’s [September 2024 final guidance on decentralized clinical trials](https://www.duanemorris.com/alerts/fda_issues_guidance_remote_clinical_trial_activities_1024.html) established a framework for conducting trial activities at locations other than traditional clinical sites, including patient homes. But that guidance was built around the assumption that sponsors would design DCT elements into a protocol from the start, with pre-specified remote monitoring procedures, local lab networks, and caregiver training plans. The lecanemab at-home start dose lands as a post-approval commercial change, not a purpose-built DCT protocol. Sponsors running real-world registry studies or observational programs around lecanemab now face a protocol amendment question they did not plan for: how do you validate home-initiation adherence when ARIA surveillance MRI still requires the patient to show up somewhere?
The counterintuitive read here matters. The conventional assumption is that at-home dosing reduces monitoring burden. For a molecule with ARIA risk, the opposite logic applies: home initiation actually increases the complexity of safety signal detection, because the first weeks of treatment, when ARIA events are most likely to emerge, are now happening outside the site’s direct observation window.
## [](#who-gets-recalibrated-first)Who Gets Recalibrated First
Academic medical centers running lecanemab-adjacent investigator-initiated trials feel this most acutely. Their IRB-approved protocols likely specify IV initiation as a visit-tied event with concurrent safety assessments. A protocol amendment to accommodate subcutaneous home initiation requires not just a dosing change, but a renegotiation of the visit schedule, caregiver training documentation, and the remote symptom reporting pathway. None of that is fast.
Sponsors designing de novo Alzheimer’s trials with lecanemab as an active comparator arm face a cleaner but equally urgent problem. The July 13 approval changes the standard-of-care assumption for the comparator arm’s delivery model. A protocol written before July 2026 that specifies IV initiation for the comparator is now operationally out of step with what neurologists will actually do in practice, which erodes the external validity argument during regulatory review.
The recruitment calculus also shifts in ways that matter for enrollment projections. CLARITY AD completed enrollment of 1,795 early Alzheimer’s patients in March 2021, a period when IV infusion center access was the only pathway. Community neurologists without infusion infrastructure were effectively excluded as referring sites. Home-initiated subcutaneous dosing opens the referral network to outpatient practices that never had a chair to offer. For sponsors modeling site feasibility for new CNS programs, the assumption that anti-amyloid therapy requires infusion-capable sites is no longer defensible.
## [](#the-protocol-action-this-requires)The Protocol Action This Requires
If you are running a lecanemab observational registry, a real-world evidence program, or any investigator-initiated trial that references Leqembi’s dosing regimen, your protocol language needs to be reviewed against the July 13 approval before your next IRB submission. Specifically, audit three things: how the protocol defines treatment initiation as a site-based versus home-based event, whether caregiver training is specified as a protocol-required activity with documented competency assessment, and whether your ARIA surveillance MRI schedule is decoupled from the dosing visit schedule. If your MRI windows are anchored to infusion visit dates, they no longer map to a home-injection calendar without a formal amendment.
The forward signal to watch is whether Eisai and Biogen file for a supplemental indication that explicitly incorporates home initiation into a supported DCT protocol framework, or whether the agency issues any follow-on guidance clarifying ARIA monitoring expectations for patients who begin subcutaneous therapy outside a clinical setting. The [FDA’s DCT guidance from September 2024](https://www.appliedclinicaltrialsonline.com/view/fda-decentralized-clinical-trial-guidance) is the closest operative document right now, but it predates this specific approval by nearly two years. An agency that approved home initiation of an amyloid antibody without a companion monitoring guidance update has created a compliance gap that sponsors are currently navigating with no map. The first ARIA event reported from a home-initiated patient will determine how quickly that gap gets addressed.
## [](#references)References
1. [JAMA — “FDA Approves at-Home Starting Dose for Anti-Amyloid Therapy”](https://jamanetwork.com/journals/jama/fullarticle/2852497)
2. [FDA — “FDA Approves First Home Starting Dose for Alzheimer’s Disease Treatment” (July 13, 2026)](https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-home-starting-dose-alzheimers-disease-treatment)
3. [DailyMed — Leqembi (lecanemab-irmb) Prescribing Information, Dosing Regimen](https://dailymed.nlm.nih.gov/dailymed/getFile.cfm?setid=9d1ff786-e577-410a-a273-c4d7d0e4e975&type=pdf)
4. [FDA Drug Safety Communication — MRI Monitoring for Leqembi (lecanemab-irmb)](https://www.fda.gov/drugs/drug-safety-communications/fda-recommend-additional-earlier-mri-monitoring-patients-alzheimers-disease-taking-leqembi-lecanemab)
5. [Duane Morris — FDA Final Guidance on Decentralized Clinical Trials (September 18, 2024)](https://www.duanemorris.com/alerts/fda_issues_guidance_remote_clinical_trial_activities_1024.html)
6. [Biogen Investor Relations — CLARITY AD Trial Enrollment Completion, 1,795 Patients (March 2021)](https://investors.biogen.com/news-releases/news-release-details/eisai-and-biogen-inc-announce-us-fda-grants-breakthrough-therapy)
**Categories:** Intel Brief
**Tags:** Alzheimer's Disease, CNS trial design, Decentralized Clinical Trials, FDA Approval
---
### [enVast Mechanical Thrombectomy Meets Primary Endpoint in Large-Thrombus STEMI Trial](https://www.clinicaltrialvanguard.com/news/envast-mechanical-thrombectomy-meets-primary-endpoint-in-large-thrombus-stemi-trial/)
**Published:** August 30, 2026
**Author:** Jon Napitupulu
**Excerpt:** Mechanical thrombectomy for STEMI with large thrombus burden reduced infarct size by 26% in the NATURE trial, meeting its primary endpoint.
**Content:**
A 26% reduction in infarct size, measured by CK-MB area-under-the-curve, is not an incremental improvement in a crowded field. It is a statistically significant signal (p < 0.001) from a 154-patient randomized controlled trial that may reopen one of cardiology’s more contentious debates: whether mechanical thrombectomy belongs in the standard STEMI workflow. The [NATURE trial](https://www.prnewswire.com/news-releases/nature-randomized-controlled-trial-meets-primary-endpoint-evaluating-envast-assisted-mechanical-thrombectomy-in-large-thrombus-stemi-302863297.html), presented as late-breaking science at ESC Congress 2026 in Munich, met its primary endpoint evaluating Vesalio’s enVast Coronary Clot Retriever against conventional primary PCI alone in STEMI patients with large thrombus burden.
The design matters as much as the headline number. NATURE enrolled 154 patients across 11 centers in a prospective, two-arm randomization, targeting a precisely defined population: STEMI with large thrombus burden, a subgroup that has historically driven the worst reperfusion outcomes and the highest procedural complication rates. The primary endpoint was infarct size by CK-MB AUC, not a composite of softer clinical events. A secondary cardiac MRI endpoint at day three showed a 25% relative reduction in infarct size as a percentage of left ventricular mass, directionally consistent with the primary read. On the safety side, the enVast arm recorded zero strokes and zero deaths at 30 days, against 1.3% and 2.6% respectively in the control group, and total MACE came in at 1.3% versus 3.8%. These are small absolute numbers from a small trial, and they should not be over-interpreted, but they are not a red flag either.
The strategic context requires care. Routine mechanical thrombectomy in STEMI was largely abandoned after large trials showed no benefit in unselected patients. Vesalio’s thesis is that the failure was one of patient selection, not of the concept itself, and NATURE was built to test that thesis in the large thrombus burden subset specifically. The [enVast device holds FDA 510(k) clearance](https://www.vesalio.com/vesalio-receives-fda-510k-clearance-of-envast-the-first-stent-based-coronary-thrombectomy-technology/) for mechanical thrombectomy in the cardiac circulation and [CE marking dating to December 2021](https://www.vesalio.com/vesalio-initiates-clinical-study-evaluating-innovative-thrombectomy-technique-for-patients-with-stemi/), so regulatory access is not the bottleneck. Physician adoption and guideline incorporation are.
The single marker worth tracking now is whether the NATURE dataset is sufficient for a formal guideline consideration process at ESC or ACC/AHA, specifically in the large thrombus burden subgroup. A trial of 154 patients, however clean its signal, sits below the threshold most guideline committees require for a Class IIa or higher recommendation. Vesalio’s next move almost certainly involves a larger confirmatory study or a patient-level meta-analysis, and how quickly that design is registered will determine whether NATURE becomes a landmark or a proof-of-concept footnote.
*Source link: *
**Categories:** News
---
### [Dubai's rWGS Scale-Up Rewrites the Diagnostic Playbook — But the Data Infrastructure Problem Stays Unsolved](https://www.clinicaltrialvanguard.com/article/article-deep-dive/dubais-rwgs-scale-up-rewrites-the-diagnostic-playbook-but-the-data-infrastructure-problem-stays-unsolved/)
**Published:** August 29, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Dubai Health's rapid whole-genome sequencing rollout for critically ill children delivers diagnostic yields that dwarf standard testing, but exposes a data…
**Content:**
Picture a neonatologist at Dubai Health standing over a critically ill infant with no confirmed diagnosis, a deteriorating trajectory, and a family waiting for an answer. Six months ago, the clinical pathway from that bedside moment ran through targeted gene panels, weeks of turnaround time, and a [diagnostic yield that — in comparable ICU populations — hovered around 10 to 15 percent](https://pmc.ncbi.nlm.nih.gov/articles/PMC12025730/). Today, that same moment triggers a [rapid whole-genome sequencing (rWGS) workflow](https://www.nature.com/articles/s41591-026-04627-9) that returns a result in under 72 hours. The question worth asking is not whether the technology works. The question is what happens to all that data once the diagnosis lands.
Dubai Health has done something genuinely difficult: it moved rWGS from pilot program to universal first-line diagnostic tool for rare genetic diseases across its hospital network, replacing targeted gene testing and whole-exome sequencing entirely. That decision, now documented in *Nature Medicine*, represents one of the most operationally significant precision medicine deployments in a Middle Eastern healthcare system. But embedded inside that success story is a structural warning for any sponsor or health system trying to build trial-adjacent clinical infrastructure on top of genomic data pipelines that were never designed for interoperability.
## [](#the-yield-gap-no-one-wants-to-quantify)The Yield Gap No One Wants to Quantify
Start with the numbers that matter. The NICUSeq randomized time-delayed trial, published in *JAMA Pediatrics* in 2021, enrolled [354 critically ill infants suspected of having a genetic disease](https://pmc.ncbi.nlm.nih.gov/articles/PMC8477301/) and remains the most rigorous head-to-head comparison of rWGS against conventional genetic testing in the ICU setting. The diagnostic yield differential is not marginal. Whole-genome sequencing consistently outperforms panels and exome sequencing in undiagnosed pediatric cases by a factor that makes traditional testing look like a coin flip.
That gap is now operational reality in the Gulf.
King Faisal Specialist Hospital and Research Centre in Saudi Arabia has reported a [67 percent reduction in WGS costs](https://www.kfshrc.edu.sa/en/news/2024/08/kfshrc-achieves-67-reduction-in-whole-genome-sequencing-costs) compared to previous technology, a figure that dismantles one of the oldest objections to broad deployment. When cost per test drops to a level comparable with extended gene panels, the economic argument for sequencing everything at admission becomes straightforward. Dubai Health appears to have reached a similar cost threshold, because the system’s switch from targeted testing to universal rWGS as the first-line tool is not a research decision. It is a procurement and clinical policy decision — which means the volume of genomic data now flowing through Middle Eastern hospital networks is growing at a rate that the region’s data governance frameworks were not designed to absorb.
The UAE passed [Federal Law by Decree No. 49 of 2023 Regulating the Use of the Human Genome](https://uaelegislation.gov.ae/en/legislations/2195/download), a comprehensive statute governing all genomic applications across the country, including Free Zones. The law is notable for its scope: it applies to research, clinical use, and commercial applications simultaneously. What it does not specify, with the precision that clinical implementation requires, is how genomic findings flow between institutions, how they integrate with electronic health record systems running on heterogeneous platforms, and what interoperability standard governs machine-readable variant reporting across hospital networks. That gap matters enormously for anyone treating this data as a future asset — for trials, for longitudinal research, for population-level pharmacogenomic analysis.
## [](#infrastructure-built-for-diagnosis-not-for-science)Infrastructure Built for Diagnosis, Not for Science
Here is the counterintuitive reality that the Dubai Health announcement obscures: scaling rWGS clinically and building a research-grade genomic data infrastructure are not the same project. Most health systems that have achieved the former assume they are simultaneously building the latter. They are not.
The [HL7 FHIR Genomics Reporting Implementation Guide](https://build.fhir.org/ig/HL7/genomics-reporting/) exists precisely because raw variant output from sequencers is not interoperable by default. The guide defines how genomic findings should be structured, coded, and communicated so they are “consistent, computable, and shareable” across clinical systems. Adoption of this standard in Gulf health systems remains uneven. When Dubai Health runs rWGS on a critically ill neonate and returns a diagnosis in 72 hours, the clinical workflow is optimized. Whether that variant call file, the interpreted finding, and the longitudinal outcome data are structured in a way that feeds a FHIR-compliant genomic data repository — queryable for future research, linkable to phenotypic outcomes, usable as real-world evidence in a regulatory submission — is a separate question entirely. One that the *Nature Medicine* paper, commendably honest about what it is reporting, does not fully answer.
Sponsors designing rare disease trials with any Middle Eastern site strategy need to confront this directly. The patient population is there. The diagnostic infrastructure is arriving. But a site’s ability to sequence a patient rapidly does not automatically translate into that site’s ability to contribute structured genomic data to a multi-site trial database, generate CDISC-compliant genomic datasets, or produce variant annotations that meet [FDA’s 2023 expectations for real-world genomic data submissions](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pharmacogenomic-data-submissions-0).
The Kyrgyz Republic’s implementation of whole-genome sequencing for tuberculosis diagnostics — deploying the Illumina MiSeq platform at a throughput of up to [55 WGS tests per week](https://pubmed.ncbi.nlm.nih.gov/34321545/) in a low-resource setting — demonstrated that sequencing infrastructure can be stood up in resource-constrained environments with focused investment. What that implementation also revealed is that local sequencing capacity and nationally harmonized data reporting are entirely separate infrastructure problems, each requiring its own solution timeline and governance framework. Dubai operates at a different resource level than Kyrgyzstan, but the structural lesson transfers: you can sequence before you can report, and the gap between those two capabilities is where research value evaporates.
## [](#what-sponsors-operating-in-this-space-must-do-now)What Sponsors Operating in This Space Must Do Now
For any sponsor considering Middle Eastern pediatric sites in a rare disease or precision medicine trial, the Dubai Health rWGS scale-up creates an opportunity with a specific operational precondition attached. Before executing a site agreement, the due diligence question is not “can this site sequence?” The answer is yes, increasingly, and at falling cost. The question is “can this site export structured genomic data in a format that satisfies your data management plan, your CDISC submission package, and any FDA or EMA request for real-world genomic evidence?”
Add a pre-site-qualification genomic data audit to the qualification visit checklist. Specifically: ask whether the site’s laboratory information management system outputs variant data in a format compatible with the [HL7 FHIR Genomics Reporting standard](https://www.hl7.org/fhir/genomics.html), whether the site has an existing data transfer agreement framework for genomic data that satisfies [UAE Federal Law No. 49 of 2023](https://uaelegislation.gov.ae/en/legislations/2195/download), and whether the site has contributed genomic data to any prior multi-site research database. If the answer to all three is no, the site is a diagnostic asset — not yet a research asset. That distinction determines your timeline, your data cleaning budget, and your submission risk.
The [Dubai Health rWGS deployment](https://sapac.illumina.com/company/news-center/feature-articles/dubai-health-embraces-genomics-to-advance-care-and-research.html) is a genuine milestone: a health system in a region historically underrepresented in genomic research is generating diagnostic-grade whole-genome data at population scale, on critically ill children, with turnaround times that rival the fastest programs anywhere in the world. The infrastructure to translate that diagnostic capacity into research-grade evidence infrastructure is the next construction project. Sponsors who wait for it to be complete before engaging will find that someone else has already enrolled the patients.
## [](#references)References
1. [Nature Medicine — “Scaling rapid whole-genome sequencing for critical pediatric care in the Middle East”](https://www.nature.com/articles/s41591-026-04627-9)
2. [King Faisal Specialist Hospital and Research Centre — “KFSHRC Achieves 67% Reduction in Whole Genome Sequencing Costs”](https://www.kfshrc.edu.sa/en/news/2024/08/kfshrc-achieves-67-reduction-in-whole-genome-sequencing-costs)
3. [UAE Legislation — Federal Law by Decree No. 49 of 2023 Regulating the Use of the Human Genome](https://uaelegislation.gov.ae/en/legislations/2195/download)
4. [HL7 International — Genomics Reporting Implementation Guide](https://build.fhir.org/ig/HL7/genomics-reporting/)
5. [PubMed — WGS Implementation for TB Diagnostics in the Kyrgyz Republic](https://pubmed.ncbi.nlm.nih.gov/34321545/)
6. [Illumina / JAMA Pediatrics — NICUSeq Randomized Time-Delayed Trial, 2021](https://www.illumina.com/company/news-center/press-releases/2021/22054f20-61c4-4b1c-8427-e30d8bbacf05.html)
7. [Illumina — “Dubai Health Embraces Genomics to Advance Care and Research”](https://sapac.illumina.com/company/news-center/feature-articles/dubai-health-embraces-genomics-to-advance-care-and-research.html)
**Categories:** Article: Deep Dive
**Tags:** clinical data integration, FDA Real-World Evidence, genomic diagnostics, pediatric clinical trials, precision medicine
---
### [Cumberland Seeks FDA Action on Ifetroban DMD Data Before Extension Study Complete](https://www.clinicaltrialvanguard.com/news/cumberland-seeks-fda-action-on-ifetroban-dmd-data-before-extension-study-complete/)
**Published:** August 29, 2026
**Author:** Jon Napitupulu
**Excerpt:** Cumberland Pharmaceuticals seeks FDA action on ifetroban DMD data from an ongoing extension study, positioning the drug for cardiomyopathy treatment.
**Content:**
Cumberland Pharmaceuticals is asking the FDA to act on data it has not yet fully made public. The 8-K filed this week discloses that a slide presented at the [PPMD 2026 Annual Conference in Orlando](https://www.prnewswire.com/news-releases/parent-project-muscular-dystrophy-hosts-2026-annual-conference-in-orlando-florida-302809445.html) indicated that long-term safety and efficacy data from the ongoing open-label extension of the Phase 2 FIGHT DMD trial are expected to support a regulatory submission for ifetroban in DMD-associated cardiomyopathy. The company is surfacing that expectation publicly before the extension data are in hand, which is either a signal of internal confidence or a move to manage investor expectations around a program that has been running for years without a clear filing timeline.
The underlying Phase 2 trial enrolled 41 male DMD patients aged seven and older, randomizing them to high-dose ifetroban (300 mg/day), low-dose (150 mg/day), or placebo across 12 months. Ifetroban carries [Orphan Drug, Rare Pediatric Disease, and Fast Track designations](https://investor.cumberlandpharma.com/news-releases/news-release-details/cumberland-pharmaceuticals-receives-fda-orphan-drug-and-rare) from the FDA for cardiomyopathy associated with DMD, which means a priority review voucher is potentially in play upon approval. For a small-cap company like Cumberland, that voucher alone could be worth hundreds of millions of dollars, making the regulatory pathway as financially consequential as the drug itself.
The strategic tension here is real. DMD cardiac disease is a serious and progressive complication, and current management has relied heavily on off-label approaches. Cumberland is positioning ifetroban as an oral thromboxane receptor antagonist with a differentiated mechanism, but the Phase 2 study was a 41-patient trial, and the extension cohort will carry enormous weight with regulators evaluating durability of effect. The PPMD conference presentation served as a controlled preview, giving the patient community and potential partners a reason to stay engaged while the open-label data mature. That is a legitimate communications tactic, but it compresses the distance between clinical signal and regulatory narrative in a way that raises the stakes for what the extension actually shows.
The number to track from here is the cardiac endpoint readout from the open-label extension: specifically, whether functional measures hold or improve beyond the 12-month randomized period, because that durability argument will determine whether the FDA accepts a Phase 2-based submission or requires a larger confirmatory study before granting full approval.
*Source link: *
**Categories:** News
---
### [Reading the COMPARE Phase 1 Data in Nature Medicine Closely](https://www.clinicaltrialvanguard.com/opinion/reading-the-compare-phase-1-data-in-nature-medicine-closely/)
**Published:** August 28, 2026
**Author:** Moe Alsumidaie
**Excerpt:** Nature Medicine's COMPARE phase 1 data on CD19 CAR T in refractory RA shows zero severe CRS in 6 patients — a stark contrast to the 18.5% rate in oncology.…
**Content:**
On August 28, 2026, *Nature Medicine* published the Phase 1 results from the [COMPARE trial (NCT06475495)](https://www.nature.com/articles/s41591-026-04603-3), a Phase 1/2 study comparing B-cell depletion by rituximab and anti-CD19 CAR T cell therapy in patients with active, ACPA-positive, treatment-refractory rheumatoid arthritis. Six patients received a single infusion of mivocabtagene autoleucel (KYV-101), a fully human anti-CD19 CAR T cell construct. The press release emphasized the headline result. What deserves closer reading is the protocol architecture — the eligibility criteria, the safety monitoring logic, and the dose justification — because those design choices will determine whether Phase 2 generates data the FDA can actually act on.
> “Phase 1 enrolled six patients — three women and three men, aged 31 to 69 — who received a single infusion of mivocabtagene autoleucel (KYV-101). All six patients had seropositive rheumatoid arthritis and had failed multiple prior lines of therapy, including conventional synthetic DMARDs and at least one biologic DMARD, before receiving the investigational treatment.”
The enrollment window — 31 to 69 years old, balanced by sex — reflects deliberate population breadth for a first-in-indication study. Sponsors running CD19 CAR T in hematologic malignancies typically skew younger and more heavily pre-treated; the inclusion of patients up to age 69 signals that the investigators were modeling the actual RA treatment-refractory population, not a convenient one. The [EULAR definition of difficult-to-treat RA requires failure](https://ard.bmj.com/content/80/1/31) of at least two conventional DMARDs plus at least one biologic or targeted synthetic DMARD after adequate dosing, and this cohort satisfies that threshold. That matters because [EULAR’s D2T RA criteria](https://ard.bmj.com/content/80/1/31) are the closest thing the field has to a regulatory consensus definition of “refractory” — and anchoring eligibility there gives the FDA a recognized reference frame when reviewing the IND data package.
## [](#the-safety-architecture-the-press-release-skipped)The Safety Architecture the Press Release Skipped
> “The COMPARE trial investigates the safety of anti-CD19 CAR T cell therapy in Phase 1, and the safety of both CAR T cell therapy and rituximab in Phase 2. The study also seeks to assess ACPA seroconversion — whether treatment can drive ACPA negativity — as a key biomarker endpoint.”
The two-stage safety mandate embedded in this language is not standard. Most Phase 1 cell therapy designs establish a single agent’s safety, then move to efficacy in Phase 2. The COMPARE protocol layers a head-to-head safety comparison with rituximab into Phase 2, which means the investigators are running a concurrent active comparator arm at a stage where most sponsors are still characterizing dose-response. Under [FDA’s guidance on early-phase clinical trials of cellular and gene therapy products](https://www.fda.gov/media/106369/download), early-phase designs must justify dose selection and escalation logic explicitly, and the addition of a randomized comparator arm in Phase 2 substantially increases the evidentiary demand on the Phase 1 safety database. Six patients are adequate to characterize acute toxicity signals; they are not adequate to power a safety comparison. That tension — between Phase 1 sample size and Phase 2 comparator ambition — is the structural design question the publication does not fully resolve.
> “The Phase 1 cohort received a single infusion of KYV-101. B-cell depletion was observed following treatment, consistent with the mechanism of action of anti-CD19 CAR T cell therapy targeting CD19-expressing B cells.”
The single-infusion design is consequential. In oncology CAR T protocols, dose escalation across cohorts is the norm precisely because early-phase cell therapy guidance requires sponsors to identify a maximum tolerated dose or a recommended Phase 2 dose. A single fixed dose in all six Phase 1 patients means the investigators made a prior determination that one dose level was clinically justified — likely informed by the oncology safety database for KYV-101 — rather than running a classic 3+3 escalation. Sponsors reading this protocol should note that FDA’s cellular therapy guidance does permit single-dose Phase 1 designs when a biologically active dose can be established from prior human data, but the justification must appear explicitly in the IND. If the Phase 2 randomization proceeds at a dose that was never escalated in Phase 1, that justification becomes a primary inspection target.
The confirmation of B-cell depletion as a pharmacodynamic outcome is important for a different reason: it anchors ACPA seroconversion as a mechanistically plausible endpoint, not simply a correlative one. ACPA-producing plasmablasts are CD19-positive; depleting them is the mechanism by which the investigators hypothesize seroconversion occurs. That chain of reasoning — depletion confirmed, seroconversion measurable, clinical remission correlated — is exactly the evidentiary architecture FDA needs to grant accelerated approval on a biomarker endpoint under 21 CFR 314.510 or its biologics equivalent. The protocol is building that chain deliberately.
## [](#cytokine-release-syndrome-what-zero-events-in-six-patients-actually-means)Cytokine Release Syndrome: What Zero Events in Six Patients Actually Means
> “The trial assessed safety outcomes including cytokine release syndrome and neurotoxicity, consistent with standard monitoring for CD19-directed CAR T cell therapies across indications.”
The absence of severe cytokine release syndrome events in this cohort deserves calibration against the oncology baseline. A [pooled analysis of 982 patients treated with CD19 CAR T for hematologic malignancies](https://pubmed.ncbi.nlm.nih.gov/32305113/) found a [severe CRS rate of 18.5%](https://pubmed.ncbi.nlm.nih.gov/29766234/) (95% CI: 12.8–25.9%). In adult patients specifically, that rate was 16.1%. The COMPARE Phase 1 cohort reported no severe CRS events across all six patients. That is encouraging, but six patients is not a sample from which you can draw a rate estimate with any statistical confidence — the 95% confidence interval around zero events in six patients runs from 0% to roughly 46%. The investigators and the press coverage are right to call this a safety signal worth pursuing. They would be wrong to call it a safety conclusion.
What the clean safety profile does accomplish is satisfy the Phase 1 stopping rules. Under the [FDA’s early-phase cell therapy guidance](https://www.fda.gov/media/106369/download), Phase 1 designs must specify dose-limiting toxicity criteria and decision rules for proceeding to Phase 2. No severe CRS and no grade 3+ neurotoxicity in six patients clears the typical DLT threshold and formally enables Phase 2 enrollment under most IND structures. The protocol has done its job: it has established that the dose is tolerable enough to randomize. Whether the dose is optimal is a separate question that Phase 2 will need to answer.
> “Long-term immunoglobulin monitoring is incorporated into the follow-up protocol, given the established relationship between CD19-directed B-cell depletion and hypogammaglobulinemia in treated patients.”
This clause is brief but represents one of the most operationally demanding commitments in the entire protocol. Data from [a study of 579 patients treated with CD19-directed CAR T cell therapy](https://pmc.ncbi.nlm.nih.gov/articles/PMC11805655/) found that 90.6% developed hypogammaglobulinemia (IgG at or below 600 mg/dL) after treatment, with [mean IgG levels falling from 587 mg/dL pre-treatment to 362 mg/dL post-treatment](https://pmc.ncbi.nlm.nih.gov/articles/PMC11805655/). For oncology patients with short expected survival horizons, that immunocompromise is a manageable trade-off. For an RA patient in their thirties or forties who achieves remission, long-term hypogammaglobulinemia and its associated infection risk becomes a dominant safety narrative that will define the therapy’s benefit-risk profile for regulators and payers alike. The protocol’s commitment to longitudinal immunoglobulin monitoring is the right call; the question for Phase 2 is whether the follow-up duration and the immunoglobulin replacement intervention criteria are pre-specified with enough granularity to generate data that actually resolves the question.
## [](#what-phase-2-must-deliver)What Phase 2 Must Deliver
Read end-to-end, the COMPARE Phase 1 publication is a competent execution of a genuinely ambitious protocol. The investigators chose a population that matches the real-world RA refractory burden. They anchored eligibility in the EULAR D2T RA framework. They selected a mechanistically coherent biomarker endpoint in ACPA seroconversion. They committed to the long-term safety monitoring that CD19 depletion demands. And they cleared the acute toxicity bar that justifies proceeding to Phase 2. What the Phase 1 data cannot tell us — and what the press coverage conflates with what it can — is whether KYV-101 produces durable remission at a rate that exceeds rituximab in a properly powered comparison, whether hypogammaglobulinemia becomes a treatment-limiting problem over a three- to five-year follow-up horizon, and whether ACPA negativity correlates with clinical remission tightly enough to serve as a regulatory endpoint rather than a biomarker curiosity.
The directive for sponsors running analogous cell therapy programs in autoimmune indications is specific: the Phase 1-to-Phase 2 transition logic must be written into the IND before Phase 2 begins, not reconstructed afterward. [FDA’s cellular therapy guidance requires explicit documentation](https://www.usdm.com/resources/blogs/cellular-therapy-regulations) of the dose-selection rationale, the DLT evaluation period, and the criteria for proceeding. In a single-dose Phase 1 with six patients, the absence of a traditional escalation design means the justification for the Phase 2 dose carries extra weight during review. Sponsors should also pre-specify the immunoglobulin monitoring intervals and the threshold for initiating replacement therapy in the Phase 2 protocol — because a post-hoc immunoglobulin dataset is not the same as a pre-specified safety endpoint, and FDA will draw that distinction when the BLA lands.
The next document to watch for is the Phase 2 protocol amendment, which will either pre-specify ACPA seroconversion as a primary endpoint or relegate it to exploratory status. That choice will determine whether this program has a path to accelerated approval or is building toward a full efficacy trial measured in years.
## [](#references)References
1. [Nature Medicine — “CD19 CAR T cell therapy for treatment-refractory seropositive rheumatoid arthritis: a phase 1 trial”](https://www.nature.com/articles/s41591-026-04603-3)
2. [Veeva CTV — COMPARE Trial (NCT06475495): Phase 1/2 enrollment and patient demographics](https://ctv.veeva.com/study/comparison-of-b-cell-depletion-by-rituximab-and-anti-cd-19-car-t-therapy-in-patients-with-rheumatoid)
3. [PubMed — Pooled severe CRS rate of 18.5% in 982 patients treated with CD19 CAR T for hematologic malignancies](https://pubmed.ncbi.nlm.nih.gov/32305113/)
4. [Annals of the Rheumatic Diseases — EULAR definition of difficult-to-treat rheumatoid arthritis](https://ard.bmj.com/content/80/1/31)
5. [FDA — “Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry”](https://www.fda.gov/media/106369/download)
6. [PMC — Hypogammaglobulinemia incidence (90.6%) and IgG decline in 579 patients after CD19-directed CAR T cell therapy](https://pmc.ncbi.nlm.nih.gov/articles/PMC11805655/)
**Categories:** Article: Opinion
**Tags:** Adaptive Clinical Trial Design, Autoimmune Disease, CAR T cell therapy, rheumatoid arthritis
---
### [Akeso's ivonescimab meets OS endpoint in EGFR-TKI-resistant NSCLC Phase III](https://www.clinicaltrialvanguard.com/news/akesos-ivonescimab-meets-os-endpoint-in-egfr-tki-resistant-nsclc-phase-iii/)
**Published:** August 28, 2026
**Author:** Jon Napitupulu
**Excerpt:** Akeso's ivonescimab met overall survival endpoint in Phase III trial for EGFR-TKI-resistant NSCLC, with consistent results across Western and Chinese patient co
**Content:**
Three Phase III wins in a single August, across three distinct tumor types, is not a pipeline story anymore — it is a clinical infrastructure story. Akeso’s ivonescimab, a PD-1/VEGF bispecific antibody, posted an overall survival hazard ratio of 0.76 in EGFR-TKI-resistant NSCLC (with Western patients matching the China cohort almost exactly at HR 0.76 versus 0.74), beat durvalumab-based chemotherapy on OS in [first-line biliary tract cancer](https://www.prnewswire.com/news-releases/akeso-2026-interim-results-strong-commercialization-momentum-io2-0-global-strategy-redefining-clinical-standards-bispecific-advancement-in-adc-ii-and-alzheimers-302862455.html), and received NMPA approval for squamous NSCLC on the back of dual positive OS and PFS data versus a PD-1 monoclonal antibody plus chemotherapy in a randomized, double-blind Phase III — a bar that comparator arms built around pembrolizumab, [approved for first-line PD-L1-positive NSCLC since 2016](https://pmc.ncbi.nlm.nih.gov/articles/PMC5679831/), had not previously crossed in a head-to-head setting.
The commercial read is just as important as the clinical one. First-half 2026 drug sales hit RMB 1,803.2 million, up 28.7% year-over-year, with all 12 approved indications now inside China’s National Reimbursement Drug List. That reimbursement sweep matters because NRDL inclusion is what converts Phase III wins into volume, and Akeso is sitting on RMB 9,160 million in cash to fund what comes next. The FDA BLA for ivonescimab in EGFR-TKI-resistant NSCLC is already under review, making the HR 0.76 OS data the number regulators are now evaluating against a setting where [approved checkpoint options carry EGFR/ALK resistance restrictions](https://en.wikipedia.org/wiki/Atezolizumab) and sequential therapy after TKI failure remains genuinely difficult to optimize.
The strategic depth behind these readouts is what separates this from a routine interim update. Ivonescimab is running in more than 17 registrational Phase II/III studies, including seven global trials and eight using standard-of-care active comparators. Cadonilimab, the PD-1/CTLA-4 bispecific, is enrolled in more than 13 registrational or Phase III studies and is being tested against nivolumab-based chemotherapy in gastric cancer — a direct challenge to an entrenched first-line combination. Partnership trials span Revolution Medicines, ARCUS Biosciences, and collaborations with Dana-Farber and Mass General Brigham, meaning the combination data generating over the next 18 months will come from externally validated academic sites, not only Akeso-controlled studies.
The single marker to track now is the FDA’s action on the EGFR-TKI-resistant NSCLC BLA. A U.S. approval would transform ivonescimab from a China-dominant commercial asset into a global one, and the consistency between the Western-patient HR (0.76) and the [China-conducted HARMONi-A result (HR 0.74)](https://www.prnewswire.com/news-releases/akeso-2026-interim-results-strong-commercialization-momentum-io2-0-global-strategy-redefining-clinical-standards-bispecific-advancement-in-adc-ii-and-alzheimers-302862455.html) removes the usual concern about ethnic subgroup heterogeneity that slows cross-border extrapolation. That decision date is the one clinical calendar event that changes the scale of this story.
*Source link: *
**Categories:** News
---
### [AusperBio Closes $120M Series C for AHB-137 Hepatitis B Program](https://www.clinicaltrialvanguard.com/news/ausperbio-closes-120m-series-c-for-ahb-137-hepatitis-b-program/)
**Published:** August 28, 2026
**Author:** Jon Napitupulu
**Excerpt:** AusperBio closes $120M Series C for AHB-137 Hepatitis B program showing 70% functional cure rate in Phase 2 trial data.
**Content:**
A 70% functional cure rate in treatment-naive patients, reported at EASL 2026, is the number that explains why AusperBio just closed a $120 million Series C, bringing its total raise to $360 million since 2024. That figure reframes what this financing actually is: not a bet on early science, but a commercial runway build for a drug that has already produced Phase 2 signal strong enough to justify a Phase 3 registrational program now underway in China.
The strategic logic is straightforward but the execution challenge is not. [Eight FDA-approved drugs](https://www.hepb.org/treatment-and-management/drug-watch/) already exist for chronic hepatitis B, nearly all nucleos(t)ide analogues and interferons that suppress viral replication effectively but achieve functional cure, defined by the FDA as sustained HBsAg loss below 0.05 IU/mL plus durable HBV DNA suppression, in only a small fraction of patients. An estimated [254 million people worldwide](https://www.ajmc.com/view/ahb-137-demonstrates-high-cure-rates-sustained-viral-suppression-in-chronic-hepatitis-b) live with chronic hepatitis B, and the overwhelming majority require indefinite suppressive therapy rather than finishing treatment. That structural gap is where AusperBio is positioning AHB-137, its unconjugated antisense oligonucleotide built on the Med-Oligo platform, which suppresses HBsAg production, inhibits viral DNA replication, and is designed to promote immune reactivation rather than simply hold viral load in check.
The $120 million does two things simultaneously. It funds the Phase 3 registrational program for AHB-137 and builds out commercialization infrastructure, meaning regulatory, manufacturing, and launch readiness are being developed in parallel rather than sequentially. That dual spend is a deliberate structural choice: companies that wait for Phase 3 data before standing up commercial capabilities routinely lose 12 to 18 months of potential launch time. The round also accelerates AHB-171, an siRNA candidate using the company’s Au-HALO hepatocyte-targeted delivery platform, explicitly positioned as the second component of a combination backbone strategy for deeper and more durable antiviral responses. RA Capital’s entry as a new investor alongside the existing syndicate of HanKang Capital, Qiming Venture Partners, and CDH Investments signals external validation of that two-asset combination thesis, not just the lead program.
The single marker worth tracking is the [FDA’s functional cure endpoint](https://www.natap.org/2022/HBV/042622_01.htm), specifically whether AusperBio pursues a U.S. registrational path for AHB-137 alongside its China Phase 3, or sequences the two. That regulatory geography decision will determine whether the $360 million raised is sized correctly for a regional launch or a global one.
*Source link: *
**Categories:** News
---
### [MBX Biosciences Doses First Patient in Phase 3 Canvuparatide Trial for Hypoparathyroidism](https://www.clinicaltrialvanguard.com/news/mbx-biosciences-doses-first-patient-in-phase-3-canvuparatide-trial-for-hypoparathyroidism/)
**Published:** August 28, 2026
**Author:** Jon Napitupulu
**Excerpt:** MBX Biosciences has dosed the first patient in the Phase 3 canvuparatide trial for hypoparathyroidism, a 160-patient pivotal study evaluating once-weekly PTH re
**Content:**
A 3:1 randomization ratio and a 160-patient enrollment target are modest numbers, but they carry real weight for MBX Biosciences: the company dosed its first patient in the pivotal Phase 3 [oPTimize trial](https://www.biospace.com/press-releases/mbx-biosciences-announces-first-patient-dosed-in-pivotal-phase-3-optimize-trial-of-once-weekly-canvuparatide-in-adults-with-hypoparathyroidism) of once-weekly canvuparatide on August 27, 2026, setting a clock that now runs through a 26-week double-blind period and a subsequent 78-week open-label extension. That design is lean enough to generate a registrational dataset relatively quickly, but the open-label tail means full program readout is still years away, which matters for a company whose current valuation rests almost entirely on this single asset.
The strategic bet here is on dosing convenience. Chronic hypoparathyroidism requires lifelong hormone replacement, and patients have historically cycled through calcium supplementation and active vitamin D with inconsistent results. Canvuparatide is a once-weekly subcutaneous PTH analog, and the [Phase 2 Avail trial](https://investors.mbxbio.com/news-releases/news-release-details/mbx-biosciences-announces-once-weekly-canvuparatide-achieved) across 64 randomized patients produced efficacy results MBX characterized as positive across the 400, 600, and 800 microgram dose arms tested against placebo. Phase 3 will need to translate that signal into a clean, durable normalization of serum calcium with a safety profile that holds up over a longer exposure window than Phase 2 allowed.
The addressable population is real but constrained. Estimates place U.S. [hypoparathyroidism prevalence at roughly 115,000 patients](https://pmc.ncbi.nlm.nih.gov/articles/PMC5393595/), with a narrower diagnosis-based count of approximately 59,000 insured adults carrying chronic disease. That ceiling shapes what a commercial launch could realistically look like, and it puts pressure on MBX to demonstrate superiority on quality-of-life endpoints, not just biochemical normalization, to justify premium pricing in a market where payers will scrutinize every line of the dossier.
The single number to track as enrollment opens: what proportion of the 160-patient target is enrolled within the first two quarters. Slow accrual in rare-disease trials is often the earliest visible signal of site readiness problems or patient-identification gaps, and either one compresses a timeline that the market has already priced as aggressive.
*Source link: *
**Categories:** News
---
### [Genentech, DualityBio Partner on Next-Generation ADC Payloads](https://www.clinicaltrialvanguard.com/news/genentech-dualitybio-partner-on-next-generation-adc-payloads/)
**Published:** August 28, 2026
**Author:** Jon Napitupulu
**Excerpt:** Genentech partners with DualityBio on next-generation ADC payloads designed to overcome topoisomerase inhibitor resistance in cancer patients.
**Content:**
Resistance to topoisomerase inhibitor payloads is already measurable in the clinic: a 2025 study in *Clinical Cancer Research* found [TOP1 mutations at disease progression in 12.9% of metastatic breast cancer patients](https://aacrjournals.org/clincancerres/article/31/10/1966/762210/TOP1-Mutations-and-Cross-Resistance-to-Antibody) who had received ADCs, compared with 0.7% at baseline. That signal is exactly what Genentech is paying $45 million upfront to get ahead of, through a new collaboration with DualityBio anchored on the company’s DUPAC payload platform, with milestone eligibility exceeding $1 billion across all programs.
The strategic logic is tight. Approved topoisomerase inhibitor ADCs, including [sacituzumab govitecan and fam-trastuzumab deruxtecan](https://pmc.ncbi.nlm.nih.gov/articles/PMC10182892/), are moving into earlier lines of therapy across major solid tumor indications. That success is also creating a downstream problem: a larger, earlier-emerging population of patients whose tumors are resistant or less responsive to that payload class. DUPAC is built specifically for that population, with payloads DUP5, DUP9, and DUP10 each operating through distinct antitumor mechanisms, paired with linker technologies engineered for systemic stability and tumor-specific release. Preclinical data, presented across AACR 2025 and 2026, show activity in tumor models that are relatively insensitive to topoisomerase inhibitor payloads, plus non-human primate tolerability data.
The deal structure reflects a clean division of labor. DualityBio leads discovery and early global clinical development through Phase 1a, applying its platform against Genentech-defined oncology targets. At the Phase 1a handoff, Genentech takes exclusive worldwide responsibility for further development and commercialization. That arrangement is deliberate: DualityBio retains the asset-generation engine while Genentech absorbs the capital-intensive late-stage and commercial burden. For DualityBio, which is already running multicenter trials across nearly 20 countries with more than 3,500 patients enrolled, this is not a capacity question. It is a reach question, and Roche’s global commercialization infrastructure answers it.
The number to watch as these programs advance is Phase 1a tumor-response rates in patients previously treated with topoisomerase inhibitor ADCs. That subgroup readout will be the first real-world test of whether DUPAC’s preclinical resistance-bypass profile translates into clinical differentiation, and it will set the valuation floor for the milestone payments that follow.
*Source link: *
**Categories:** News
---
### [How to Make Clinical Trials for Elderly with Disabilities More Inclusive](https://www.clinicaltrialvanguard.com/analysis/how-to-make-clinical-trials-for-elderly-with-disabilities-more-inclusive/)
**Published:** August 28, 2023
**Author:** Moe Alsumidaie
**Content:**
The global demographic is undergoing a significant shift, with an increasing number of elderly patients. This aging population brings a host of health challenges, many of which result in disabilities that profoundly affect quality of life, especially when including elderly patients in clinical trials. Richie Kahn’s recent article on the importance of inclusivity in clinical trials, especially for those with disabilities, serves as a timely reminder of the gaps in our current medical research landscape. But it’s also important to consider designing better clinical trials for elderly with disabilities. This article will discuss these topics.
# [](#richie-kahns-insightful-perspective-on-disabled-patients-in-clinical-trials)**Richie Kahn’s Insightful Perspective on Disabled Patients in Clinical Trials:**
[Kahn’s article](https://globalforum.diaglobal.org/issue/august-2023/thoughtful-consideration-of-disabilities-in-clinical-trials/ "Khan's article") provides a comprehensive look into the disparities in healthcare accessibility for adults with disabilities across different countries. He emphasizes that those with disabilities are four times more likely to report unmet healthcare needs, a statistic that is both alarming and revealing. Kahn’s perspective is deeply personal, as he himself is a clinical trialist with a rare disease (Wolfram syndrome) that leads to both blindness and sensorineural hearing loss. Drawing from this personal experience, as well as the United Nations’ definition of disabilities, Kahn categorizes disabilities into various segments. He not only highlights the unique challenges each group faces when participating in clinical trials but also underscores the importance of thoughtful consideration in designing these trials. For instance, he presents a case example of a trial designed for patients with severe vision loss, emphasizing the myriad of considerations that might not be immediately apparent to those unfamiliar with the disability.
# [](#a-broader-perspective-on-aging-and-disabilities-in-the-u-s)**A Broader Perspective on Aging and Disabilities in the U.S**.
In the United States, the burden of disability among the aging population is evident. Disabilities, including cognitive and physical, pose significant challenges[1](https://chat.openai.com/#user-content-fn-3%5E). Mild cognitive impairment, a precursor to [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease, is a growing area of concern, with sensory integration training emerging as a potential intervention to improve mental mobility in affected elderly individuals[2](https://chat.openai.com/#user-content-fn-4%5E). Additionally, sensory impairments, particularly in vision and hearing, are prevalent and can significantly impact the quality of life and societal participation of the elderly[3](https://chat.openai.com/#user-content-fn-1%5E). Furthermore, [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions, especially depression, can be debilitating, affecting cognitive functions and social interactions[3](https://chat.openai.com/#user-content-fn-1%5E).
# [](#recommendations-for-study-and-protocol-design-for-inclusivity-in-clinical-trials)**Recommendations for Study and Protocol Design for Inclusivity in Clinical Trials:**
1. **Holistic Patient Assessment:** Beyond the specific condition being studied, a comprehensive health assessment of elderly participants can provide insights into co-existing conditions and disabilities.
2. **Flexible Trial Protocols:** Recognize that elderly patients might have multiple health issues. Design trials that can accommodate variations in health profiles.
3. **Accessibility and Comfort:** Ensure study sites are easily accessible, with provisions for those with mobility issues. Consider the comfort of participants, especially when designing study visits, to not put the patients through too many procedures. Focus on attaining the study’s endpoints instead.
4. **Clear Communication:** Use simple language, visual aids, and repetition to ensure that elderly participants fully understand the trial procedures and their roles. Also use eConsent and include rich media, like videos, to easily explain the study to the participants.
5. **Engage Caregivers:** Often, elderly individuals rely on caregivers. Engaging them in the trial process can provide additional support to participants.
# [](#summary)Summary
The increasing prevalence of age-related disabilities in the U.S. necessitates a shift in how clinical trials are designed and conducted. Richie Kahn’s article is a stepping stone, urging us to think deeper and act more inclusively. As we design clinical trials for elderly with disabilities, let’s ensure they truly represent the diverse and complex needs of our aging population.
References:
1. [Common Physical Health Issues in Persons with Intellectual Disability](https://dx.doi.org/10.33591/sfp.48.6.u3)
2. [Influence of sensory integration training on mental mobility of elderly people with MCI](https://dx.doi.org/10.1051/e3sconf/202018503049)
3. [Prevalence of sensory impairments, physical and intellectual disabilities, and mental health in children and young people with self/proxy-reported autism: Observational study of a whole country population](https://dx.doi.org/10.1177/1362361318791279)
**Categories:** Analysis, Clinical Trials
---
### [Decentralized Clinical Trials: Big Pharma's New Frontier](https://www.clinicaltrialvanguard.com/conference-coverage/decentralized-clinical-trials-big-pharmas-new-frontier/)
**Published:** October 2, 2023
**Author:** Moe Alsumidaie
**Content:**
In a recent webinar, industry luminaries including Harpreet Gill from ICON plc, Jane Twitchen from Biogen, Angela May from Bayer, and Stephanie Manson Brown from [AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/) convened to unravel the intricate world of Decentralized Clinical Trials (DCTs). The event, hosted by the Financial Times, delved deep into the critical themes and challenges surrounding DCTs, offering a treasure trove of insights and potential solutions in this dynamic and evolving field.
### [](#navigating-the-technological-labyrinth-in-decentralized-clinical-trials)**Navigating the Technological Labyrinth in Decentralized Clinical Trials**
The discussion brought the pressing technological challenges in Decentralized Clinical Trials to the forefront. Panelists emphasized the importance of a comprehensive understanding of technology’s role and impact on trial sites and patients. The conversation explored using various [digital health technologies](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/), such as watches or sensors, as tools for data collection and patient monitoring in Decentralized Clinical Trials. While these technologies offer unprecedented opportunities for real-time data collection and patient engagement, the speakers highlighted the crucial need to ensure their reliability, accuracy, and ease of use. Concerns were raised about the potential for technology to overburden trial sites and patients, underscoring the need for a balanced and thoughtful approach to technology integration in DCTs.
### [](#operational-delivery-ensuring-seamless-and-effective-execution-of-dcts)**Operational Delivery: Ensuring Seamless and Effective Execution** of DCTs
Operational delivery in Decentralized Clinical Trials emerged as a significant theme, with experts highlighting the importance of effectively engaging and supporting trial sites. The panelists discussed potential challenges related to site contracts, reimbursement, and the overall burden on trial sites involved in DCTs. They emphasized the importance of clear communication, adequate support structures, and fair reimbursement practices to ensure the smooth and effective operation of DCTs. The discussion underscored the importance of minimizing the administrative and operational burden on trial sites to ensure their active and sustained participation in Decentralized Clinical Trials, ultimately contributing to the success and integrity of decentralized trials.
### [](#prioritizing-patient-and-investigator-insights-in-dcts)**Prioritizing Patient and Investigator Insights in DCTs**
The discussion underscored the importance of prioritizing patient and investigator insights in DCTs. The experts collectively emphasized the need for a patient-centric approach in designing and implementing Decentralized Clinical Trials. The conversation explored the significance of real-time access to data and metrics for subjects and patients, highlighting the potential of such access to enhance patient engagement, satisfaction, and retention in clinical trials. The panelists discussed the importance of understanding and addressing patients’ needs, preferences, and concerns, advocating for a proactive and responsive approach to patient engagement in Decentralized Clinical Trials. They stressed the critical role of patients as active stakeholders in clinical trials, whose insights and feedback are invaluable in shaping effective, efficient, and patient-friendly Decentralized Clinical Trials.
### [](#delving-into-study-phase-relevance-in-decentralized-clinical-trials)**Delving into Study Phase Relevance in Decentralized Clinical Trials**
The conversation also delved into the domain of phase relevance in DCTs, shedding light on the diverse opinions regarding the most suitable phases for conducting decentralized trials. The panelists offered varied perspectives, with some suggesting the particular relevance of phases 2 and 4 for Decentralized Clinical Trials. The discussion underscored the importance of a strategic and thoughtful approach to phase selection in DCTs, considering each phase’s unique characteristics, requirements, and challenges. The panelists highlighted the need for careful consideration and planning to ensure the successful and effective conduct of DCTs across different phases, ensuring that each trial is optimally designed and executed for maximum impact and benefit.
### [](#summary)**Summary**
As the extensive discussion unfolded, it painted a rich and multifaceted picture of the world of Decentralized Clinical Trials. While the promise and potential of DCTs are immense, the conversation highlighted the significant challenges that must be effectively navigated to realize this potential fully. The discussion’s emphasis on a thoughtful and balanced approach to technology integration, operational delivery, patient and investigator engagement, and phase relevance resonated, offering a comprehensive and insightful roadmap for the future of Decentralized Clinical Trials. As reflected in the conversation, the unwavering commitment of industry experts to addressing these challenges head-on augurs well for the future of Decentralized Clinical Trials, promising a new era of more inclusive, innovative, and effective clinical trials.
**Categories:** Article: Conference Coverage, Decentralized Clinical Trials
**Tags:** Article, Clinical Trials, Conference Coverage, Decentralized Clinical Trials
---
### [DCTs: Washington's Jubilant Journey To Revolutionize Trials](https://www.clinicaltrialvanguard.com/conference-coverage/dcts-washingtons-jubilant-journey-to-revolutionize-trials/)
**Published:** November 28, 2023
**Author:** Moe Alsumidaie
**Content:**
The 2023 [Decentralized Trials & Research Alliance (DTRA) Annual Meeting](https://www.dtra.org/ "Decentralized Trials & Research Alliance Summit (DTRA)") featured a pivotal moment in clinical research with a focused panel discussion on Decentralized Clinical Trials (DCTs), featuring prominent U.S. government representatives including Gina Conenello, Program Officer at [BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/), Christopher Hartshorn, Chief, Digital & Mobile Technologies Section at NIH, Stephen Konya, Senior Advisor and Innovation Portfolio Lead at Office of the National Coordinator for Health IT, Timil Patel, Medical Oncologist at FDA, and Anindita Saha, Assistant Director, Digital Health Center of Excellence at FDA, moderated by Craig Lipset, Co-Founder of DTRA. This dialogue offered deep insights into the evolving landscape of DCTs, underscoring their potential to revolutionize healthcare.
#### [](#governments-endorsement-and-vision-for-dcts)**Government’s Endorsement and Vision for DCTs**
The discussion began with an affirmation of the government’s endorsement of DCTs. Panelists elucidated the pivotal role DCTs play in expanding the scope and inclusivity of clinical research. They emphasized that DCTs are not just a response to immediate needs, such as the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, but a long-term strategy to democratize clinical research, making it more accessible to diverse patient populations nationwide.
DTRA Government Panel
#### [](#highlighting-government-supported-dct-initiatives)**Highlighting Government-Supported DCT Initiatives**
1. [**Clinical Translational Science Awards Program**:](https://ncats.nih.gov/research/research-activities/ctsa#:~:text=Under%20NCATS'%20leadership%2C%20the%20Clinical,research%20discoveries%20into%20improved%20care.&text=Currently%2C%20more%20than%2060%20leading,nation%20receive%20CTSA%20Program%20funding. "Clinical Translational Science Awards Program:") Panelists shed light on the CTSA program. This initiative illustrates the government’s commitment to fostering a national network of medical centers dedicated to translational science, thus enhancing the reach and efficacy of clinical research in the U.S.
2. **[ACTIV-6 Trial](https://activ6study.org/ "ACTIV-6 Trial") and [N3C Initiative](https://covid.cd2h.org/ "N3C Initiative")**: The ACTIV-6 trial for COVID-19 therapeutics and the National COVID Cohort Collaborative (N3C) served as prime examples of the government’s proactive role in utilizing DCTs to address public health emergencies. These initiatives demonstrated how DCTs could be rapidly deployed to gather essential data across a wide demographic, showcasing the flexibility and scalability of DCTs in urgent situations.
3. **[All of Us Research Program](https://allofus.nih.gov/ "All of Us Research Program")**: The discussion also highlighted the “All of Us” research program as an exemplary model of a large-scale, decentralized research platform. This ambitious program’s study on nutrition precision health has been instrumental in leveraging DCTs for diverse data collection, moving beyond traditional clinical settings to gather comprehensive health data from across the U.S.
#### [](#navigating-challenges-and-regulatory-frameworks)**Navigating Challenges and Regulatory Frameworks**
Panelists delved into the challenges surrounding DCTs, particularly in the context of regulatory compliance and ethical considerations. They discussed the necessity of developing regulatory frameworks that can adapt to the rapid evolution of DCTs, ensuring patient safety and data integrity. Ethical concerns, especially around patient privacy and data security, were discussed as crucial elements in establishing trust and confidence in DCT methodologies.
#### [](#revolutionizing-patient-engagement-and-data-accuracy)**Revolutionizing Patient Engagement and Data Accuracy**
Much of the conversation was dedicated to how DCTs transform patient engagement and data collection methods. By enabling more personalized patient care and real-time data monitoring, DCTs are improving patient experience and the accuracy and comprehensiveness of data collection. This leads to improved clinical trial outcomes and a deeper understanding of diverse patient responses.
#### [](#the-future-trajectory-of-dcts-in-healthcare)**The Future Trajectory of DCTs in Healthcare**
The roundtable concluded with a forward-looking discussion on the role of DCTs in shaping the future of healthcare. Panelists expressed optimism about the potential of DCTs to redefine clinical research, emphasizing the need for patient-centric, efficient, and adaptable trial frameworks. This approach aligns with a broader vision of transforming healthcare delivery and treatment modalities, making healthcare more personalized and accessible.
#### [](#summary)**Summary**
The DTRA panel offered a rich and enlightening perspective on the transformative role of Decentralized Clinical Trials in modern healthcare, underpinned by substantial government-led examples and initiatives. With in-depth insights from key U.S. government agencies, the discussion underscored the importance of embracing the opportunities and challenges presented by DCTs. The panelists’ expertise highlighted DCTs’ capacity to enhance clinical research, ensure regulatory compliance, and prioritize patient-centered care. These discussions and examples underscore the promising future of DCTs in revolutionizing clinical trials and advancing toward more effective, efficient, and patient-focused healthcare solutions.
**Categories:** Article: Conference Coverage
**Tags:** Clinical Trials, Conference Coverage, Decentralized Clinical Trials
---
### [AstraZeneca's ASH 2023: Surging Hematology Innovations](https://www.clinicaltrialvanguard.com/news/astrazenecas-ash-2023-surging-hematology-innovations/)
**Published:** November 30, 2023
**Author:** Jon Napitupulu
**Content:**
At the upcoming 65th American Society of Hematology (ASH) Annual Meeting, [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) will present a wealth of new clinical and real-world data spanning multiple hematological disorders. This presentation will comprise a substantial 63 abstracts highlighting 14 approved and innovative medications within the company’s extensive portfolio, including their group focusing on rare diseases, Alexion.
Significant among the data presented will be six-year follow-up results from the ELEVATE-TN Phase III trial, which is anticipated to reinforce the long-term efficacy and safety of CALQUENCE ([acalabrutinib](https://www.clinicaltrialvanguard.com/news/ascentage-pharma-presents-promising-cancer-research-at-aacr/)) in patients with chronic lymphocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (CLL) who have not undergone previous treatment. Moreover, in the realm of paroxysmal nocturnal hemoglobinuria (PNH), the ALPHA Phase III trial is set to reveal the potential of danicopan to manage clinically significant extravascular hemolysis effectively.
Anas Younes, Senior Vice President of Hematology R&D at AstraZeneca, and Gianluca Pirozzi, Senior Vice President at Alexion, emphasized the company’s commitment to advancing care in hematology through innovative therapies like T-cell engagers, antibody-drug conjugates, and other novel scientific approaches.
These updates highlight AstraZeneca’s strategic efforts to drive advancements across various stages of drug development and spotlight their leadership role in addressing hematological malignancies and other blood disorders.
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**Categories:** News
---
### [Gilead Great Trodelvy Focus: New Insights](https://www.clinicaltrialvanguard.com/news/gilead-great-trodelvy-focus-new-insights/)
**Published:** November 30, 2023
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences, Inc. is set to present new data at the San Antonio [Breast Cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) Symposium ([SABCS](https://www.clinicaltrialvanguard.com/news/celcuity-to-present-updated-viktoria-1-data-at-sabcs-2025/)) 2023, highlighting the benefits of Trodelvy® ([sacituzumab](https://www.clinicaltrialvanguard.com/news/sacituzumab-tirumotecan-plus-pembrolizumab-meets-primary-endpoint-in-lung-cancer-trial/) govitecan-hziy) for certain patients with metastatic triple-negative breast cancer (mTNBC) and HR+/HER2- metastatic breast cancer (mBC) who have received prior treatments. The presentations will include results from a clinical outcomes analysis based on age from the Phase 3 TROPiCS-02 study, as well as a qualitative analysis on the clinical meaningfulness of mBC treatments from the perspectives of patients, caregivers, and clinicians.
The SABCS 2023 will feature eight abstracts associated with Trodelvy, offering insights into its significant survival benefits for patients with second-line metastatic TNBC and pre-treated HR+/HER2- metastatic breast cancer. Bill Grossman, MD, PhD, from Gilead Oncology, emphasized the importance of Trodelvy as the first approved Trop-2-directed ADC that has shown survival improvement in these breast cancer populations. Additionally, Gilead will share real-world data highlighting quality of life and other health measures that are vital for treatment decision-making.
Trodelvy has been recommended as a category 1 preferred treatment for second-line mTNBC and metastatic HR+/HER2- breast cancer by the NCCN. The data presented at the symposium will enhance the understanding of Trodelvy’s role in treating difficult breast cancer types and provide deeper context for its use in clinical practice.
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**Categories:** News
**Tags:** Clinical Trial News, Clinical Trials
---
### [PsychoGenics Revolutionary Brain Drug Set to Combat Mental Ills](https://www.clinicaltrialvanguard.com/news/psychogenics-revolutionary-brain-drug-set-to-combat-mental-ills/)
**Published:** December 4, 2023
**Author:** Jon Napitupulu
**Content:**
PsychoGenics Inc., with its drug discovery platform PGI Drug Discovery LLC, has announced an exclusive license agreement with Roche for the global rights to RO7117997, an Equilibrative Nucleoside Transporter 1 (ENT1) inhibitor. Discovered through their collaboration, PsychoGenics aims to advance RO7117997 as a treatment for a variety of psychiatric and neurological conditions, with an initial focus on sleep and seizure disorders.
RO7117997 represents a novel approach to addressing neuropsychiatric disorders and has already shown a promising safety profile in preclinical studies. ENT1 inhibitors work by increasing extracellular adenosine levels, which can improve sleep quality and reduce seizure frequency via adenosine receptor activation. Potential applications for ENT1 inhibition extend to [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/), addiction, neurodegenerative diseases, pain, and inflammation.
PsychoGenics is well-known for its expertise in translating rodent behavioral and physiological responses into high-throughput phenotyping, aiding in discovering several compounds now in clinical and preclinical development. The company is preparing to submit an IND application and begin clinical trials for RO7117997 in 2024.
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**Categories:** News
---
### [BMS Opdivo Wins FDA Fast-Track: Remarkable Cancer Hope](https://www.clinicaltrialvanguard.com/news/bms-opdivo-wins-fda-fast-track-remarkable-cancer-hope/)
**Published:** December 6, 2023
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) has announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review a supplemental [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (sBLA) for Opdivo ([nivolumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/)) in combination with cisplatin-based chemotherapy. This is intended as a first-line treatment for adult patients with unresectable or metastatic urothelial carcinoma. The FDA has set a target action date of April 5, 2024.
The application is based on the results of the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -901 trial, which demonstrated a statistically significant and clinically meaningful survival benefit of the Opdivo-based regimen over the standard-of-care gemcitabine plus cisplatin treatment in this patient population. The trial’s data showcased improvements in both overall survival (OS) and progression-free survival (PFS), while maintaining a tolerable safety profile with no new concerns.
If approved, this combination would mark the first immunotherapy-chemotherapy regimen authorized for these patients in the U.S., providing a potential new treatment option for a condition with few efficacious first-line therapies. Bristol Myers Squibb (BMS) acknowledges the patients and investigators who contributed to the CheckMate -901 trial and foresees working closely with the FDA during the review process. Opdivo-based combinations have made noteworthy strides in improving overall survival in various tumors, signifying a pivotal advancement for individuals battling metastatic urothelial carcinoma.
Source link: [http://www.businesswire.com/news/home/20231204596253/en/U.S.-Food-and-Drug-Administration-Accepts-for-Priority-Review-Bristol-Myers-Squibb%E2%80%99s-Application-for-Opdivo-nivolumab-in-Combination-with-Cisplatin-Based-Chemotherapy-for-the-First-Line-Treatment-of-Adult-Patients-with-Unresectable-or-Metastatic…](http://www.businesswire.com/news/home/20231204596253/en/U.S.-Food-and-Drug-Administration-Accepts-for-Priority-Review-Bristol-Myers-Squibb%E2%80%99s-Application-for-Opdivo-nivolumab-in-Combination-with-Cisplatin-Based-Chemotherapy-for-the-First-Line-Treatment-of-Adult-Patients-with-Unresectable-or-Metastatic...)
**Categories:** News
---
### [Merck Evobrutinib Fails in Trials: A Devastating Outcome for MS](https://www.clinicaltrialvanguard.com/news/merck-evobrutinib-fails-in-trials-a-devastating-outcome-for-ms/)
**Published:** December 6, 2023
**Author:** Jon Napitupulu
**Content:**
Merck KGaA, a renowned science and technology company, has announced the results of its two Phase III EVOLUTION clinical trials (evolutionRMS 1 and evolutionRMS 2), which investigated the efficacy and safety of evobrutinib, a promising oral medication for relapsing [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (RMS). Unfortunately, the trials did not meet their primary endpoints as the annualized relapse rates (ARR) in patients treated with evobrutinib were equivalent to those treated with the comparator drug, teriflunomide.
Despite the ARR results, the safety and tolerability profile of evobrutinib matched the positive outcomes seen in earlier Phase II trials. Danny Bar-Zohar, the Global Head of R&D and Chief Medical Officer at Merck’s healthcare sector, expressed disappointment but reiterated the company’s dedication to continuing to improve its healthcare offerings through both its current portfolio and new innovations. Comprehensive data analysis from the EVOLUTION trials will be undertaken and shared in the future.
Evobrutinib, designed to modulate B cell responses and macrophage/microglia activation, is part of Merck’s commitment to addressing unmet needs in the treatment of MS, a debilitating neurological disease affecting millions worldwide. The EVOLUTION program was comprehensive, involving a sizable number of patients in studies comparing evobrutinib with teriflunomide over a considerable time frame. The results from this research will inform Merck’s continued efforts in the neurology and immunology therapeutic areas.
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**Categories:** News
---
### [Sanofi Venetoclax: Breakthrough NK Cell Therapy Stuns at ASH 2023](https://www.clinicaltrialvanguard.com/news/sanofi-venetoclax-breakthrough-nk-cell-therapy-stuns-at-ash-2023/)
**Published:** December 12, 2023
**Author:** Jon Napitupulu
**Content:**
Innate Pharma SA, in collaboration with Sanofi, shared updated efficacy and safety results from a Phase 1/2 dose-escalation study of SAR443579 / [IPH6101](https://www.clinicaltrialvanguard.com/news/unlock-the-latest-in-cancer-treatment-sanofi-and-innate-pharmas-sar443579-iph6101-breakthrough/) ( venetoclax ), an investigational Natural Killer Cell Engager (NKCE). The results were presented at the American Society of Hematology 2023 Annual Meeting in San Diego, California.
SAR443579 is a [CD123](https://www.clinicaltrialvanguard.com/news/unveiling-the-promising-advancements-in-blood-cancer-treatment-innate-pharmas-groundbreaking-trial-findings/) targeting NKp46/CD16-based NKCE being tested as monotherapy for blood cancers that lack sufficient treatment options, specifically relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), and high-risk myelodysplasia (HR-MDS). The therapy has received FDA Fast Track Designation for treating AML.
As of July 5, 2023, the study analyzed 43 patients who had received a median of 2 lines of prior treatments. Many had also been exposed to venetoclax, a therapy for chronic lymphocytic leukemia. At the highest dose level utilized in the study (1000 μg/kg QW), about one-third of AML patients achieved complete remission (CR), with a well-tolerated safety profile observed up to 6000 μg/kg QW.
These emerging results show the durable clinical efficacy and favorable safety of SAR443579, offering hope for the continuation of development in blood cancers. Sonia Quaratino, Chief Medical Officer of Innate Pharma, expressed enthusiasm for advancing the development of multi-specific NK Cell Engagers leveraging Innate’s [ANKET](https://www.clinicaltrialvanguard.com/news/innate-pharmas-next-gen-anket-iph6501-highlighted-in-science-immunology/) platform®. Peter Adamson from Sanofi reiterated this sentiment, emphasizing the potential importance of SAR443579 for AML patients with limited treatment options.
The ANKET® platform is Innate’s proprietary technology for developing a new class of molecules to induce synthetic immunity against cancer. Innate’s research collaboration and license agreement with Sanofi apply this technology to develop innovative, multi-specific antibody formats that engage NK cells through the NKp46 receptor.
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**Categories:** News
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### [Merck & Moderna Blaze Trail with New Cancer Vaccine Trial](https://www.clinicaltrialvanguard.com/news/merck-moderna-blaze-trail-with-new-cancer-vaccine-trial/)
**Published:** December 13, 2023
**Author:** Jon Napitupulu
**Content:**
Merck, and Moderna, Inc., have launched INTerpath-002, a pivotal Phase 3 randomized clinical trial for a cancer vaccine. The study aims to evaluate V940 (mRNA-4157), Moderna’s investigational individualized neoantigen therapy (INT), in combination with Merck’s [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/), an anti-PD-1 therapy, as adjuvant treatment for patients with resected Stage II or IIIA/IIIB [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) with nodal involvement (N2).
Global recruitment for INTerpath-002 has started, with the first patients enrolled in Australia. Dr. Marjorie Green, Merck’s Senior Vice President, emphasized the importance of new scientific developments in treating lung cancer, particularly in its earlier stages where the prognosis is more favorable. The trial combines KEYTRUDA with V940, exploring innovations for earlier stages of NSCLC.
Kyle Holen, M.D., from Moderna, highlighted the complexities involved in addressing lung cancer, with each patient’s cancer presenting unique genetic mutations. This necessitates an innovative approach to develop individualized medicines tailored to each patient’s tumor profile. Moderna believes that their individualized neoantigen therapy can be a catalyst for such innovation, potentially propelling cancer care with cancer vaccine into a new frontier.
INTerpath-002 follows INTerpath-001, another Phase 3 trial examining the combination of V940 (mRNA-4157) and KEYTRUDA in patients with resected high-risk melanoma. INTerpath-001 is actively screening across several countries and has multiple clinical sites. Plans are in place to expand the clinical development program for V940 to additional tumor types.
V940 (mRNA-4157) is an mRNA-based therapy containing a synthetic mRNA coding for up to 34 neoantigens, which are determined and produced based on each patient’s tumor DNA sequence. When administered, this therapy uses the body’s natural processes to translate and present these neoantigens, thereby training and activating an immune response specifically targeted at the patient’s unique tumor mutations. This innovative treatment aims to stimulate a potent adaptive immune response to fight cancer.
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**Categories:** News
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### [Mooresville Locals Breathe Easy with Sanofi COPD Study](https://www.clinicaltrialvanguard.com/news/mooresville-locals-breathe-easy-with-sanofi-copd-study/)
**Published:** December 13, 2023
**Author:** Jon Napitupulu
**Content:**
A new clinical trial, known as the AERIFY-2 study, is underway in Mooresville, N.C., focusing on chronic obstructive pulmonary disease (COPD). Conducted by Sanofi, this study is investigating [itepekimab](https://www.clinicaltrialvanguard.com/news/regeneron-sanofi-copd-trial-shows-mixed-results/)‘s potential to diminish the frequency and severity of COPD exacerbations in both current and former smokers with moderate-to-severe COPD.
COPD is a major health concern and the sixth leading cause of death in the United States. Patients with COPD often suffer from exacerbations, which are periods when their respiratory symptoms intensify and become more difficult to manage than usual. These exacerbations can be severe enough to necessitate hospitalization and are associated with an elevated risk of mortality. Though there are treatments available for COPD, there is an urgent need for more effective and safer therapeutic options.
In Iredell County, where the study is being conducted, COPD is responsible for over 100 deaths annually. Dr. Sever Surdulescu, the Care Access Research Investigator for this study, emphasized the critical need for new therapies to help reduce or prevent these life-threatening exacerbations, offering significant benefits to those with COPD.
The AERIFY-2 study is currently open for enrollment, seeking participants between 40 and 85 diagnosed with moderate-to-severe COPD. Interested individuals are encouraged to visit joincopdstudy.com or contact Care Access, the global research company facilitating the trial, to obtain more information about participating.
Care Access operates in Mooresville coordinating with local community leaders and healthcare providers, aiming to make clinical research more accessible to the local population. They allow residents to engage in clinical trials conveniently close to home, potentially accelerating the development of new medications and broadening participation.
Care Access operates over 150 research sites worldwide and collaborates with 14 of the top 15 biopharmaceutical companies, offering services including site network management, community screenings for enhanced study outreach, staffing, and training programs to support all aspects of clinical study operations.
For further details on active clinical trials and Care Access services or to inquire about enrolling in a study, interested parties can visit www.careaccess.com or email Joseph Ohmedia at media@careaccess.com or call 202-970-6885.
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**Categories:** News
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### [Miracle CAR T-Cell Hope from Gilead for Lymphoma](https://www.clinicaltrialvanguard.com/news/miracle-car-t-cell-hope-from-gilead-for-lymphoma/)
**Published:** December 13, 2023
**Author:** Jon Napitupulu
**Content:**
Kite, a Gilead Company (Nasdaq: GILD), has presented encouraging data from various studies on the CAR T-cell therapies Tecartus® (brexucabtagene autoleucel) and Yescarta® (axicabtagene ciloleucel), showing substantial long-term survival benefits for patients with certain types of relapsed or refractory (R/R) non-Hodgkin lymphoma.
For Tecartus®, the ZUMA-2 study, with nearly four years of follow-up, reported a median overall survival (OS) of 46.4 months in adult patients with R/R [Mantle Cell Lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL) who had previously undergone multiple lines of therapy. From the 68 patients involved, 44% were alive at the time of data cutoff, and those who achieved a complete response (CR) had a median OS of 58.7 months. Complementing this data, ZUMA-18, an expanded access study, showed an 87% investigator-assessed objective response rate (ORR) with a 57% CR rate.
In the realm of Yescarta®, three studies provided follow-ups suggesting durable treatment effects and potential curative intent. The ZUMA-1 post-hoc analysis with six years of follow-up in patients with refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (LBCL) discovered that those maintaining a CR at 12 and 24 months post-treatment had a 72-month estimated disease-specific survival (DSS) of 94.4% and 100%, respectively, signaling a possibly extended OS. The ZUMA-5 study with a four-year follow-up in relapsed/refractory follicular lymphoma demonstrated continued response and survival, while the ZUMA-7 subgroup analysis indicated that patients aged 65 and older can benefit from CAR T therapy without age being a barrier.
Real-world evidence (RWE) additionally highlighted Tecartus®’s effectiveness in adult patients with R/R MCL, with a CR rate of 81% overall and 84% for high-risk features, and a 76% complete remission rate in patients with R/R B-Cell Precursor Acute Lymphoblastic Leukemia (B-ALL), further supporting its use in aggressive blood cancers.
Dr. Frank Neumann from Kite expressed optimism in the ASH meeting presentations, affirming the pattern of extended survival outcomes and the importance of these therapies as transformative options. These results contribute to Kite’s ongoing commitment to developing treatments that can provide hope and improved quality of life for patients with challenging blood cancers.
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**Categories:** News
---
### [BMS's RELATIVITY-123 Trial Fail: A Devastating End](https://www.clinicaltrialvanguard.com/news/bmss-relativity-123-trial-fail-a-devastating-end/)
**Published:** December 18, 2023
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb announced the discontinuation of the Phase 3 RELATIVITY-123 trial, which was evaluating the fixed-dose combination of nivolumab and [relatlimab](https://www.clinicaltrialvanguard.com/news/relativity-098-trial-update-bms-provides-insights/) as a treatment for microsatellite stable (MSS) metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC) patients. This decision comes after an independent data monitoring committee determined that the trial was unlikely to meet its primary endpoints of overall survival.
The discontinuation was not due to safety concerns but rather the futility of the trial’s outcomes. The safety profile of the combination treatment was consistent with findings from previous studies. Despite this setback, Bristol Myers Squibb remains committed to developing immunotherapy options and will continue investigating nivolumab and relatlimab for other tumor types. The results won’t affect the existing approval for melanoma patients.
Jeffrey Walch, M.D., Ph.D., Vice President at Bristol Myers Squibb, expressed disappointment in the trial’s findings, acknowledging the high unmet need for effective treatments in MSS mCRC, where immunotherapies have historically shown limited efficacy. The company extends gratitude to the trial’s investigators, patients, and families and plans to share the data from the RELATIVITY-123 trial to inform the next steps and future research.
RELATIVITY-123 was designed to compare the efficacy of the fixed-dose combination of nivolumab and relatlimab to regorafenib or trifluridine plus tipiracil (TAS-102) in patients with MSS mCRC who had progressed despite prior therapies. The dual primary endpoints were overall survival and progression-free survival in patients with a PD-L1 combined positive score (CPS) ≥ 1. Secondary endpoints included objective response rate, duration of response, safety, and effects on physical function and quality of life. The trial did not include patients with MSI-H or dMMR tumors.
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**Categories:** News
---
### [FDA Nods to Padcev-Keytruda for Bladder Cancer](https://www.clinicaltrialvanguard.com/news/fda-nods-to-padcev-keytruda-for-bladder-cancer/)
**Published:** December 18, 2023
**Author:** Jon Napitupulu
**Content:**
Pfizer Inc. and Astellas Pharma Inc. have announced the FDA’s approval of PADCEV® (enfortumab vedotin-ejfv, an antibody-drug conjugate \[ADC\]) combined with [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/)® (pembrolizumab, a PD-1 inhibitor) for the treatment of adult patients with locally advanced or metastatic urothelial cancer (la/mUC). This approval marks the first combination treatment option to offer an alternative to the standard platinum-containing chemotherapy for first-line la/mUC.
The approval by the FDA was based on the groundbreaking results from the Phase 3 [EV-302](https://www.clinicaltrialvanguard.com/news/padcev-plus-keytruda-shows-long-term-efficacy-in-urothelial-cancer/) clinical trial (also known as [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-A39), which showed that the combination of PADCEV and pembrolizumab nearly doubled the median overall survival (OS) and median progression-free survival (PFS) in comparison to platinum-containing chemotherapy.
The trial’s findings, presented at the ESMO Congress 2023, also confirm the accelerated approval granted in April 2023 for using this combination in patients with la/mUC who are ineligible for cisplatin-containing chemotherapy. The label has now been expanded to include those eligible for cisplatin chemotherapy based on the results from EV-302.
Roger Dansey, M.D., Chief Development Officer, Oncology at Pfizer, noted the significant potential for the combination to transform the standard of care and extend the lives of patients with advanced bladder cancer. Ahsan Arozullah, M.D., M.P.H., Senior Vice President, Head of Oncology Development at Astellas, emphasized the pivotal change this approval represents, offering new hope to patients and marking the first regimen to surpass the efficacy of platinum chemotherapy, which has been the gold standard for decades.
The EV-302 study achieved its dual primary endpoints of OS and PFS when comparing the combination treatment with chemotherapy. The combination treatment led to a median OS of 31.5 months versus 16.1 months for chemotherapy alone, a 53% reduction in the risk of death, and a median PFS of 12.5 months compared to 6.3 months with chemotherapy, a 55% reduction in the risk of cancer progression or death. Consistency in OS and PFS results across patient subgroups reinforces the robustness of these findings.
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**Categories:** News
---
### [AstraZeneca & Daiichi Breakthrough Trial: New Breast Cancer Therapy Tested](https://www.clinicaltrialvanguard.com/news/astrazeneca-daiichi-breakthrough-trial-new-breast-cancer-therapy-tested/)
**Published:** December 20, 2023
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo and AstraZeneca have begun dosing the first patient in two global, randomized Phase 3 trials of their collaborative treatment, [datopotamab](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/) deruxtecan (Dato-DXd), in combination with [durvalumab](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-durvalumab-in-phase-iii-biliary-tract-cancer-trial/), an anti-PD-L1 therapy by AstraZeneca. These trials aim to evaluate the efficacy and safety of the combination in treating two types of [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/).
The trials, named TROPION-Breast04 and TROPION-Breast05, are part of an extensive clinical program to assess the TROP2 directed DXd antibody drug conjugate (ADC) datopotamab deruxtecan in different breast cancer settings. TROPION-Breast04 focuses on a neoadjuvant treatment with datopotamab deruxtecan plus durvalumab, followed by adjuvant durvalumab with or without chemotherapy, for patients with stage II-III triple-negative breast cancer (TNBC) or hormone receptor (HR) low, HER2 low, or negative breast cancer. TROPION-Breast05 investigates datopotamab deruxtecan alone and with durvalumab in patients with metastatic TNBC expressing PD-L1 (CPS ≥ 10).
Triple-negative breast cancer, which accounts for about 15% of all breast cancer diagnoses, is known for its aggressive nature and higher likelihood of recurrence and progression. Traditional treatments typically involve chemotherapy, sometimes in combination with immunotherapy. The trials also target patients with HR low, HER2 low, or negative breast cancer, a group often excluded from TNBC research but with poorer outcomes compared to those with hormone receptor-strongly positive tumors.
Mark Rutstein, MD, Global Head of Oncology Clinical Development at Daiichi Sankyo, pointed out the necessity for improved treatments for TNBC patients, despite recent advancements. These Phase 3 trials will determine if a combination of the TROP2 targeted ADC with durvalumab presents a more viable option for various breast cancer stages.
Cristian Massacesi, Chief Medical Officer and Oncology Chief Development Officer at AstraZeneca, shared that initial trials showed promising tumor responses with a manageable safety profile for the datopotamab deruxtecan and durvalumab combination. The initiation of these Phase 3 trials reflects a commitment to exploring the pairing’s potential across multiple TNBC settings and HR low disease.
Additionally, Daiichi Sankyo and AstraZeneca are conducting two more Phase 3 trials examining datopotamab deruxtecan in different TNBC contexts, expanding the research scope to potentially provide new treatment avenues for patients battling this aggressive cancer form.
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**Categories:** News
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### [Breaking New Ground: AstraZeneca, Roche, Acadia in GCP & AI](https://www.clinicaltrialvanguard.com/conference-coverage/breaking-new-ground-astrazeneca-roche-acadia-in-gcp-ai/)
**Published:** December 20, 2023
**Author:** Moe Alsumidaie
**Content:**
The 2nd GCP Inspection Readiness Conference, convened by Momentum Events, attracted a significant gathering of clinical experts in the pharmaceutical industry, focusing on the evolving integration of analytics and AI in quality assurance. The panel comprised Stefan Van den Akker, Executive Director of R&D Quality and Risk Management at Acadia Pharmaceuticals, Michael Torok, Ph.D., Vice President and Global Head of Quality Assurance Programs at Roche, and Kevin Richards, Director of Analytics and Insights at [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/). Their collective expertise provided invaluable insights into the future of quality assurance in clinical trials.
#### [](#realigning-quality-assurance-strategies-in-clinical-trials)Realigning Quality Assurance Strategies in Clinical Trials
The panelists discussed a significant shift in the pharmaceutical industry towards more quality-centric approaches, drawing insights from manufacturing sectors. They highlighted how stringent quality control in manufacturing enhances product reliability and plays a crucial role in business aspects like contract negotiations and operational cost management.
Applying this to clinical trials, the panel suggested that a similar emphasis on quality could lead to more efficient trial processes, reduced risk of non-compliance, and potentially lower trial costs in the long run. They pointed out that rigorous quality measures in clinical trials could mean fewer protocol deviations, reduced need for amendments, and enhanced data integrity, leading to smoother regulatory review processes and faster market access for new drugs. This comparison underscored the need for the pharmaceutical industry to adopt more advanced, quality-focused strategies, aligning with practices seen in other sectors.
#### [](#astrazenecas-study-quality-oversight-tool)AstraZeneca’s Study Quality Oversight Tool
One of the many highlights of the conference was the introduction of AstraZeneca’s ‘Study Quality Oversight’ tool. This innovative reporting suite is designed to transform how clinical trials are monitored and managed. It facilitates the transition from reactive to proactive quality management by providing comprehensive oversight throughout the study phases. The tool enables measuring and tracking various metrics, from study inception to completion, supporting a more proactive approach to quality. This represents a significant leap in leveraging technology to enhance clinical trial quality and compliance.
#### [](#predictive-analytics-and-ai-integration)Predictive Analytics and AI Integration
A significant part of the discussion delved into the expanding role of predictive analytics and AI in quality assurance. The panel underscored an expected increase in AI’s role in automating intricate tasks within clinical trials. They discussed specific examples, such as automating protocol deviation reviews, where AI could systematically identify, categorize, and predict potential deviations based on historical data patterns.
Furthermore, the panelists explored the transformative potential of AI in protocol drafting. They envisioned a future where AI tools could analyze vast historical trial data to identify common pitfalls and success factors. For instance, AI could suggest protocol amendments by referencing similar past studies, proactively mitigating risks, and improving the trial design. This approach would streamline the protocol development process and enhance the overall quality and reliability of clinical trials.
#### [](#data-driven-decision-making-and-cultural-shift)Data-Driven Decision-Making and Cultural Shift
The panelists stressed fostering a culture that values and actively utilizes data-driven decision-making. They delved into specific examples, such as using advanced data analytics tools to identify trends and patterns in clinical trial data, which could be crucial in predicting potential issues and improving trial outcomes.
They also highlighted the need for harmonizing disparate data sources. For instance, integrating data from clinical trial management systems, electronic health records, and patient-reported outcomes can provide a more holistic view of a trial’s progress and potential risks. This integration allows for more comprehensive analysis, leading to informed decision-making that proactively addresses quality issues before they escalate.
Moreover, the panelists discussed the importance of continuous learning from data insights. They illustrated this by referencing case studies where ongoing data analysis had led to real-time adjustments in trial protocols, enhancing the efficiency and effectiveness of the trials. This approach shifts from traditional, retrospective quality control measures to a more dynamic, data-informed strategy.
#### [](#addressing-accessibility-and-language-barriers)Addressing Accessibility and Language Barriers
The Q&A session brought up the challenges of smaller companies in accessing advanced AI technologies. The panelists recommended forming partnerships with service providers and utilizing open-source analytics tools, such as those offered by The Inter coMPany quALity Analytics (IMPALA) Consortium, as potential solutions. Additionally, they touched on overcoming language barriers in global studies, suggesting using AI for accurate data assessment across different languages.
#### [](#conclusion)Conclusion
The conference confirms an industry’s trend towards a more data-centric approach to quality assurance within the pharmaceutical industry. The insights shared by the panel provide a clear roadmap for integrating analytics and AI in quality assurance practices across organizations of varying sizes. This approach aims to enhance efficiency, compliance, and proactive quality management in clinical trials, marking a significant evolution in the field.
Momentum continues this important conversation at their upcoming Clinical Data Analytics virtual event on February 15th. [Click here](https://bit.ly/3TqnVey "Click here") for more details and use discount code CTA.
**Categories:** Article: Conference Coverage
**Tags:** Clinical Trial Quality, GCP
---
### [Pfizer Tivdak SLA Accepted by FDA for Priority Cervical Cancer Review](https://www.clinicaltrialvanguard.com/news/pfizer-tivdak-sla-accepted-by-fda-for-priority-cervical-cancer-review/)
**Published:** January 11, 2024
**Author:** Jon Napitupulu
**Content:**
Genmab A/S and Pfizer Inc. announced the FDA’s acceptance of the supplemental [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (sBLA) to potentially convert the accelerated approval of TIVDAK® ([tisotumab](https://www.clinicaltrialvanguard.com/news/tivdaks-full-fda-approval-a-triumphant-victory-for-metastatic-cervical-cancer-treatment/) vedotin-tftv) to full approval for patients with recurrent or metastatic cervical cancer who have progressed following first-line therapy. The sBLA has been granted Priority Review, with an action date set for May 9, 2024.
The sBLA submission is supported by data from the Phase 3 innovaTV 301 trial, where TIVDAK demonstrated superior overall survival, progression-free survival, and confirmed objective response rate compared to chemotherapy in patients with recurrent or metastatic cervical cancer. The safety profile of TIVDAK from the study was consistent with the known safety information.
Results from this trial were presented at ESMO Congress in October 2023. Genmab’s CEO, Jan van de Winkel, Ph.D., emphasized the need for therapeutic options that provide a survival advantage and represent new treatment approaches. Roger Dansey, M.D., Pfizer’s Chief Development Officer, Oncology, highlighted the FDA’s acceptance as crucial progress for providing an option that may extend the lives of adults with cervical cancer.
Cervical cancer remains a significant health concern, with over 13,960 new cases expected in the U.S. in 2023 alone, and an estimated 4,310 deaths. Despite advancements in prevention and early detection, there is a high need for better treatments for recurrent and/or metastatic cervical cancer—a devastating and mostly incurable condition.
The innovaTV 301 trial is a global, open-label Phase 3 study evaluating TIVDAK against investigator’s choice of chemotherapy, focusing on advanced cervical cancer. The goal of this trial, along with the sBLA, is to continue delivering TIVDAK as a viable treatment option for women in the U.S. who are affected by this aggressive disease.
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**Categories:** News
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### [Takeda's HYQVIA Wins FDA Approval for CIDP Therapy](https://www.clinicaltrialvanguard.com/news/takedas-hyqvia-wins-fda-approval-for-cidp-therapy/)
**Published:** January 17, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda has announced that the FDA has approved HYQVIA® for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) as a maintenance therapy to prevent relapse in adults. This approval allows HYQVIA, an FDA-approved combination of immunoglobulin (IG) and hyaluronidase, to be used in the treatment of CIDP, expanding its use beyond primary immunodeficiency (PI), for which it was first approved in the U.S. in 2014.
HYQVIA is a facilitated subcutaneous immunoglobulin (SCIG) infusion that can be administered up to once a month, facilitated by the hyaluronidase component, which aids the subcutaneous dispersion and absorption of IG. The therapy can be delivered by healthcare professionals or self-administered by trained patients or caregivers, providing flexibility and convenience.
Giles Platford, president of Takeda’s Plasma-Derived Therapies Business Unit, noted the significance of this approval, highlighting the company’s aim to offer more personalized treatment options for adults with CIDP, a debilitating neurological condition. Platford also expressed the hope that this would be the first of many global approvals for HYQVIA.
The approval is based on results from the [ADVANCE](https://www.clinicaltrialvanguard.com/news/mineralys-therapeutics-publishes-pivotal-phase-2-advance-htn-results-in-nejm/)-CIDP 1 and ADVANCE-CIDP 3 studies, which assessed the efficacy and safety of HYQVIA in adults. The primary efficacy analysis involved 122 adults with a confirmed CIDP diagnosis who had been on stable IVIG therapy. The analysis presented a significant difference in relapse rates between the HYQVIA and placebo groups, demonstrating the superiority of HYQVIA in preventing CIDP relapse.
CIDP is a rare, immune-mediated disorder that affects the peripheral nervous system, typically characterized by weakness, sensory loss, loss of reflexes, and difficulty walking. Due to overlapping symptoms with other conditions, CIDP can be challenging to diagnose and treat.
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**Categories:** News
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### [Arcus & Gilead: Quemliclustat Regimens Show Hope in Pancreatic Cancer](https://www.clinicaltrialvanguard.com/news/arcus-gilead-quemliclustat-regimens-show-hope-in-pancreatic-cancer/)
**Published:** January 18, 2024
**Author:** Jon Napitupulu
**Content:**
Arcus Biosciences, Inc. announced promising overall survival data from the ARC-8 Phase 1b trial, co-developed with Gilead Sciences. The trial studies quemliclustat, an investigational CD73 inhibitor, combined with chemotherapy—with or without zimberelimab, an anti-PD-1 antibody—in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC). The results are scheduled to be presented at the ASCO GI Symposium in 2024.
Dr. Zev A. Wainberg from the University of California Los Angeles highlighted the apparent survival benefit of quemliclustat plus standard chemotherapy, relative to chemotherapy alone, which has been the standard of care for more than 30 years. He noted CD73’s high expression on [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) cells and the potential of inhibiting CD73 to improve outcomes in mPDAC.
Data from all patients (n=122) treated with 100mg quemliclustat regimens were compared to a Synthetic Control Arm (SCA) constructed post-hoc by Medidata AI. This matched analysis, based on demographic and key baseline characteristics, indicated that ARC-8 patients experienced longer survival than the control arm. The efficacy data for quemliclustat-based regimens were also numerically greater than the historical benchmark for chemotherapy alone, with median overall survival (mOS) rates higher than the approximately nine-month survival seen historically.
The pooled dose-escalation, dose-expansion, and randomized arms data showcased a mOS greater than the SCA, with hazard ratios indicating a positive trend in survival. The ARC-8 patients exhibited a 12-month OS of 62.7% compared to 41.1% in the SCA, and median PFS was extended in the ARC-8 group. The percentages of objective response rate (ORR) between the treatment group and SCA were comparable.
This trial advancement suggests quemliclustat-based regimens may offer a significant survival benefit for mPDAC patients, potentially altering the treatment landscape for this aggressive cancer.
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**Categories:** News
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### [Opdivo & Cabometyx Show 4-Year Benefits in RCC Trial](https://www.clinicaltrialvanguard.com/news/opdivo-cabometyx-show-4-year-benefits-in-rcc-trial/)
**Published:** January 25, 2024
**Author:** Jon Napitupulu
**Content:**
Opdivo® ([nivolumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/)) in combination with CABOMETYX® (cabozantinib) has shown continued benefits in the first-line treatment of [advanced renal cell carcinoma](https://www.clinicaltrialvanguard.com/news/allogenes-allo-316-achieves-31-response-rate-in-advanced-renal-cell-carcinoma/) (RCC) versus sunitinib, including a 23% reduction in the risk of death. Bristol Myers Squibb and Exelixis, Inc. have announced four-year follow-up results from the [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -9ER trial, which demonstrated superior progression-free survival (PFS) and objective response rates (ORR), as well as improved health-related quality of life with the Opdivo plus CABOMETYX combination.
These long-term results will be presented at the ASCO 2024 Genitourinary Cancers Symposium. The trial’s updated findings support the important role of the immunotherapy and tyrosine kinase inhibitor (TKI) combination in treating RCC, which is often difficult to manage, especially for those with advanced disease or metastasis.
At a median follow-up of 55.6 months, all patients treated with Opdivo plus CABOMETYX showed continued efficacy across multiple endpoints. In patient subgroups, durable and clinically meaningful benefits were observed across various risk groups, including those with favorable and intermediate- to poor-risk classifications.
Dana Walker, M.D., M.S.C.E., Vice President at Bristol Myers Squibb, highlighted Opdivo and CABOMETYX as a new standard of care that has the potential to help patients with advanced RCC live longer, irrespective of risk classification. This further emphasizes the importance of Opdivo-based combinations in genitourinary cancer treatment.
The positive data from the CheckMate -9ER trial offers hope for patients with previously untreated advanced or metastatic RCC, marking a significant advancement in treatment options and supporting ongoing research efforts to enhance outcomes for this patient population.
Source link: [http://www.businesswire.com/news/home/20240122053621/en/Opdivo%C2%AE-nivolumab-in-Combination-with-CABOMETYX%C2%AE-cabozantinib-Demonstrates-Long-Term-Survival-Benefits-After-Four-Years-of-Follow-Up-in-the-CheckMate–9ER-Trial-in-First-Line-Advanced-Renal-Cell-Carcinoma](http://www.businesswire.com/news/home/20240122053621/en/Opdivo%C2%AE-nivolumab-in-Combination-with-CABOMETYX%C2%AE-cabozantinib-Demonstrates-Long-Term-Survival-Benefits-After-Four-Years-of-Follow-Up-in-the-CheckMate--9ER-Trial-in-First-Line-Advanced-Renal-Cell-Carcinoma)
**Categories:** News
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### [8-Year Data: BMS Opdivo & Yervoy Outperform in RCC Survival](https://www.clinicaltrialvanguard.com/news/8-year-data-bms-opdivo-yervoy-outperform-in-rcc-survival/)
**Published:** January 25, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) announced that the combination of Opdivo (nivolumab) and Yervoy ([ipilimumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/)) continues to show significant long-term survival benefits for previously untreated patients with advanced or [metastatic renal cell carcinoma](https://www.clinicaltrialvanguard.com/news/adicet-opens-adi-270-phase-1-trial-enrollment-for-metastatic-renal-cell-carcinoma/) (RCC). In the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -214 trial, the immunotherapy duo reduced the risk of death by 28% compared to sunitinib, an eight-year result that applies to patients of any risk group as defined by the International Metastatic RCC Database Consortium (IMDC).
Notably, the combination treatment not only improved survival but also provided more durable responses than sunitinib, particularly among patients with intermediate- and poor-risk prognostic factors as well as across the entire randomized patient group. These findings will be presented during the American Society of Clinical Oncology (ASCO) 2024 Genitourinary Cancers Symposium.
Dr. Nizar Tannir from The University of Texas MD Anderson Cancer Center emphasized the significance of these results as the longest follow-up in a Phase 3 trial for a checkpoint inhibitor combination therapy in advanced RCC. The data suggest that the dual immunotherapy could help patients achieve long-term positive outcomes, regardless of their IMDC risk status.
The safety profile of the combination has been manageable, and no new safety concerns have been identified with extended follow-up. This update reinforces Bristol Myers Squibb’s leadership position in immunotherapy and highlights the potential of their treatments across multiple cancer types.
The CheckMate -214 trial results contribute valuable insights into the management of advanced RCC and offer hope for better long-term survival for patients battling this disease.
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**Categories:** News
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### [Gilead Evoke-01 Phase 3 Study: Critical Update Issued](https://www.clinicaltrialvanguard.com/news/gilead-evoke-01-phase-3-study-critical-update-issued/)
**Published:** January 25, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences, Inc. announced that the Phase 3 [EVOKE-01](https://www.clinicaltrialvanguard.com/news/study-results-in-metastatic-nsclc-presented-at-asco-2024-astonishing-breakthrough/) study, which evaluated Trodelvy® (sacituzumab govitecan-hziy; SG) against docetaxel in patients with metastatic non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) that had previously progressed following platinum-based and checkpoint inhibitor therapy, did not meet its primary endpoint of overall survival (OS).
While the study did not achieve its primary goal, a numerical OS improvement with Trodelvy was observed, including within both squamous and non-squamous histology patient groups. Trodelvy was generally well tolerated, with safety profiles consistent with prior studies and no new safety signals were identified.
Notably, pre-specified analysis of a sub-group of over 60% of patients non-responsive to last prior anti-PD-(L)1 therapy showed a more than three-month median OS difference favoring Trodelvy. This finding was not formally statistically tested due to lack of alpha control. Conversely, this difference was not exhibited in the sub-group of patients who did respond to their last anti-PD-(L)1 therapy.
Given the high unmet medical need, Gilead intends to further investigate the potential role of Trodelvy for these particular patients and will discuss the trial results with regulators.
Merdad Parsey, MD, PhD, Chief Medical Officer of Gilead Sciences, expressed continued confidence in Trodelvy’s potential for metastatic NSCLC and mentioned the company’s extensive lung cancer clinical development program, including multiple ongoing Phase 3 trials.
Gilead highlighted the ongoing Phase 3 EVOKE-03 study of Trodelvy in combination with pembrolizumab for 1L metastatic PD-L1 high NSCLC, bolstered by promising preliminary data from the Phase 2 EVOKE-02 study presented at the World Conference on Lung Cancer 2023.
Gilead’s broad clinical development program also includes research into domvanalimab, the first Fc-silent investigational anti-[TIGIT](https://www.clinicaltrialvanguard.com/news/anti-tigit-antibody-the-revolutionary-cancer-treatment-you-must-know-about/) antibody. Despite available immunotherapies, many patients with metastatic NSCLC will experience cancer progression, with limited options post-progression, especially for those unresponsive to immunotherapy.
Gilead thanked the participating patients, families, investigators, and advocates for their contributions to this research. Trodelvy, as a Trop-2-directed antibody-drug conjugate (ADC), has already shown survival benefits in two different cancer types and continues to be a subject of extensive research.
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**Categories:** News
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### [ICON Survey Stresses Combo Therapies in Obesity Care](https://www.clinicaltrialvanguard.com/news/icon-survey-stresses-combo-therapies-in-obesity-care/)
**Published:** January 25, 2024
**Author:** Jon Napitupulu
**Content:**
ICON plc conducted an industry survey of over 100 professionals engaged in [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/)-related clinical research, revealing expectations for the future of obesity therapies and current research trends. The survey found that:
1\. Most respondents believe that obesity therapy trials should measure multiple outcomes due to the overlap between obesity and comorbidities such as diabetes, steatosis (fatty liver), and cardiovascular disease.
2\. A majority (64%) feel that combination therapies will be a primary focus for future research in obesity, reflecting the complex nature of the condition and its associated comorbidities.
3\. Respondents are keen on employing multi-indication studies within their current obesity clinical trials, which helps address the condition holistically.
4\. Two-thirds (66%) are confident about the prospects of their obesity-related pipeline succeeding in the current market, indicating optimism about upcoming treatment possibilities.
Despite their positive outlook, research professionals also highlighted some challenges, including:
1\. The absence of long-term follow-up studies, which are critical for observing the sustained impact of potential treatments.
2\. A need for trial designs that are specifically tailored to obesity.
3\. The difficulty of recruiting a diverse patient population into obesity-related studies.
Simon Bruce, ICON’s VP of Internal Medicine, pointed out the increasing effort to simultaneously develop assets that address obesity and its related comorbidities, aiming to create more efficacious and efficient treatments.
Jack Martin, Senior Director of Cardiovascular Therapeutics at ICON, emphasized the importance of considering obesity’s related comorbidities in clinical trial designs. The survey indicates that breakthroughs in obesity treatment are expected by both drug and device development sectors, showcasing optimism for future therapeutic advancements.
The survey also underscores a growing need for long-term clinical data in commercializing obesity treatments, which is not only crucial from a safety perspective but also for evaluating the impact of obesity drugs on long-term comorbidities.
With more than 1,000 active clinical trials involving obesity drugs ranging from pre-clinical to phase III studies, it highlights the significant ongoing research efforts aimed at combating this growing global health issue.
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**Categories:** News
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### [Advancing ClinOps Excellence: The 15th Annual SCOPE Summit 2024](https://www.clinicaltrialvanguard.com/conference-coverage/advancing-clinops-excellence-the-15th-annual-scope-summit-2024/)
**Published:** February 6, 2024
**Author:** Moe Alsumidaie
**Content:**
Join us at the [15th Annual SCOPE Summit](https://www.scopesummit.com/ "15th Annual SCOPE Summit"), scheduled for February 11-14, 2024, in Orlando, Florida, at the Rosen Shingle Creek, which offers a comprehensive program focused on advancing clinical trial and ClinOps practices. This event, a pivotal gathering for clinical research and operations professionals, will feature 29 focused conferences covering a wide range of topics such as patient-centric trial design, [digital health technologies](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/), decentralized trials, and more. The summit promises networking opportunities, keynotes, awards, and special events, including a golf tournament and an investor conference, designed to foster innovation and collaboration among over 3,500 attendees from 850 organizations across 27 countries.
**Categories:** Article: Conference Coverage
**Tags:** Conference Coverage
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### [AstraZeneca Grows US Cell Therapy Production Capacity](https://www.clinicaltrialvanguard.com/news/astrazeneca-grows-us-cell-therapy-production-capacity/)
**Published:** February 7, 2024
**Author:** Jon Napitupulu
**Content:**
[AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) has announced a $300 million investment in a new state-of-the-art facility in Rockville, Maryland, aimed at advancing its [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) platforms for cancer research and future commercial supply in the U.S. This endeavor will create over 150 new skilled jobs focused on manufacturing T-cell therapies to facilitate both global clinical trials and potential future therapeutic expansions.
Pam Cheng, Executive Vice President of Global Operations & IT and Chief Sustainability Officer at AstraZeneca, expressed enthusiasm for the project. She emphasized the critical role this investment will play in propelling AstraZeneca’s goal to make next-generation cell therapy a reality, meeting demands for patient treatment.
The facility is strategically located within a prominent [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) hub and near one of AstraZeneca’s key global R&D centers. The proximity to numerous universities and educational institutions in Maryland is expected to be conducive to talent acquisition and employment opportunities in the biotech sector.
Maryland Governor Wes Moore welcomed the partnership with AstraZeneca, acknowledging the shared dedication to innovation and the positive impact on the state’s life sciences leadership and competitive edge.
The Rockville site will be part of AstraZeneca’s extensive manufacturing network, which includes nearly 30 sites globally. In the U.S., AstraZeneca’s manufacturing focuses on small molecules and biologics, with over 2,600 full-time employees producing more than 9 billion doses of medicine each year.
AstraZeneca’s ongoing work in cell therapy explores the enhancement of immune T-cells’ cancer-fighting capabilities, with research into targeting CAR-T cells and overcoming immune-suppressive tumor environments. Current and future projects, such as the next-generation cell therapies being developed from healthy donor cells and specific programs like the Glypican 3 (GPC3) targeting CAR-T in hepatocellular carcinoma, will leverage this collaborative research and production effort in Maryland to fuel advancements in oncology treatment.
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**Categories:** News
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### [BMS's Opdivo for Lung Cancer: US & EU Filings Accepted](https://www.clinicaltrialvanguard.com/news/bmss-opdivo-for-lung-cancer-us-eu-filings-accepted/)
**Published:** February 8, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics Application (sBLA) for the use of neoadjuvant Opdivo (nivolumab) with chemotherapy, followed by surgery and adjuvant Opdivo as a perioperative treatment for patients with resectable stage IIA to IIIB [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). A target action date is set for October 8, 2024. Additionally, the European Medicines Agency (EMA) validated the type II variation application, which initiates the review process in Europe.
This acceptance is based on the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -77T trial results, the company’s second positive Phase 3 randomized immunotherapy-based combination trial for NSCLC. The trial demonstrated a statistically significant improvement in event-free survival (EFS) along with benefits in key secondary endpoints, including pathologic complete response (pCR) and major pathologic response (MPR). The safety profile was consistent with previously reported studies in NSCLC, and no new safety concerns were identified.
The EFS, pCR, and MPR results from the CheckMate -77T trial were presented at the European Society of Medical Oncology (ESMO) Congress in 2023. The ongoing study will also evaluate overall survival as another secondary endpoint.
Opdivo, including Opdivo-based combinations, has demonstrated efficacy in various settings across multiple cancers, such as lung, bladder, and esophageal/gastroesophageal junction cancers. The acceptance of these regulatory applications highlights Bristol Myers Squibb’s commitment to addressing unmet needs in NSCLC treatment, particularly in earlier stages, and reflects their progress in offering patients potential new treatment options.
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**Categories:** News
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### [Takeda's TAK-861 Narcolepsy Phase 3 Trials Set for FY2024](https://www.clinicaltrialvanguard.com/news/takedas-tak-861-narcolepsy-phase-3-trials-set-for-fy2024/)
**Published:** February 12, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda has announced promising topline results from a Phase 2b trial evaluating [TAK-861](https://www.clinicaltrialvanguard.com/news/unveiling-the-transformative-power-of-takedas-narcolepsy-treatment-clinical-trial-triumphs-at-sleep-europe-2024/), an oral orexin receptor 2 (OX2R) agonist, for patients with narcolepsy type 1 (NT1). The randomized, double-blind, placebo-controlled trial showed that TAK-861 led to significant and clinically meaningful improvements in wakefulness and reductions in cataplexy rates compared to placebo.
The NT1 trial demonstrated that TAK-861 notably improved the Maintenance of Wakefulness Test (MWT) scores, Epworth Sleepiness Scale (ESS) scores, and Weekly Cataplexy Rate (WCR), indicating its potential as an effective treatment for NT1. Based on these results, Takeda now plans to initiate global Phase 3 trials for TAK-861 rapidly within the first half of the fiscal year 2024.
Concurrently, Takeda has decided not to advance TAK-861 for narcolepsy type 2 (NT2) at this time and is analyzing data for next steps in populations with normal levels of orexin, such as in NT2 and other indications where orexin biology plays a role. Multiple orexin agonists are in progress for other patients with normal orexin levels.
The trial also found TAK-861 to be generally safe and well-tolerated, with no treatment-related serious adverse events, hepatotoxicity, or visual disturbances reported. The safety profile and positive outcomes of TAK-861 could signify a breakthrough in addressing the underlying pathophysiology of narcolepsy type 1. Results from the study will be presented at an upcoming scientific congress.
Takeda recognizes the contributions of patients, caregivers, and investigators involved in the orexin agonist trials and remains committed to leveraging a deep understanding of orexin biology to develop and deliver transformative treatments for various indications connected to this mechanism.
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**Categories:** News
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### [Takeda's EoE Drug Eohilia Gets First FDA Approval in US](https://www.clinicaltrialvanguard.com/news/takedas-eoe-drug-eohilia-gets-first-fda-approval-in-us/)
**Published:** February 13, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda has announced that the U.S. Food and Drug Administration (FDA) has approved EOHILIA (budesonide oral suspension) for the treatment of [eosinophilic esophagitis](https://www.clinicaltrialvanguard.com/news/tezspire-hits-both-primary-endpoints-in-phase-3-eosinophilic-esophagitis-trial/) (EoE) in individuals aged 11 years and older. This approval makes EOHILIA the first and only FDA-approved oral therapy for EoE and is expected to be available by the end of February in 2 mg/10 mL single-dose stick packs.
EoE is a chronic condition characterized by esophageal inflammation and symptoms such as choking, and difficulty or pain while swallowing. The approval of EOHILIA is based on efficacy and safety data from two randomized, double-blind, placebo-controlled 12-week studies. In this research, EOHILIA demonstrated the ability to alleviate esophageal inflammation and reduce dysphagia symptoms.
EOHILIA’s novel formulation allows for consistent dose delivery with thixotropic properties, which means it flows more easily when shaken and thickens upon swallowing. Dr. Ikuo Hirano, a gastroenterology professor at Northwestern University, emphasized the significance of having an FDA-approved, specifically formulated treatment that can offer a consistent dose delivery for managing EoE.
The FDA approval is supported by results showing that a significantly higher percentage of patients receiving EOHILIA achieved histologic remission compared to those on placebo in both studies. Additionally, improvements were seen in the Dysphagia Symptom Questionnaire (DSQ) scores, which assess the frequency and behavioral adaptations related to swallowing difficulties in EoE patients.
While EOHILIA is indicated for 12 weeks of treatment, it has not proven safe and effective beyond this duration. Common adverse reactions (≥2% of patients and greater than placebo) included respiratory tract infection, gastrointestinal mucosal candidiasis, headache, and other conditions. These reactions underscore the importance of monitoring patient outcomes with the new treatment.
This approval of EOHILIA presents a promising option for clinicians and patients dealing with the unmet needs of EoE and enhances Takeda’s ongoing commitment to addressing various disease areas through innovative therapeutic solutions.
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**Categories:** News
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### [Gilead Boosts Liver Portfolio by Acquiring CymaBay](https://www.clinicaltrialvanguard.com/news/gilead-boosts-liver-portfolio-by-acquiring-cymabay/)
**Published:** February 14, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences, Inc. has agreed to acquire [CymaBay](https://www.clinicaltrialvanguard.com/news/gilead-sciences-successfully-acquires-cymabay/) Therapeutics, Inc. for $4.3 billion, expanding its portfolio with seladelpar, a treatment for primary biliary cholangitis (PBC), including pruritus. The transaction, valuing CymaBay’s shares at $32.50 each in cash, will complement Gilead’s existing liver disease portfolio and reaffirm its commitment to liver health.
Seladelpar, an investigational oral PPARδ agonist, is anticipated to receive FDA approval in the third quarter of 2024, having already received priority review. Demonstrating a best-in-disease profile for second-line PBC in Phase 3 trials, seladelpar targets critical metabolic and liver disease pathways, offering potential relief for PBC symptoms like itching and fatigue.
Daniel O’Day, Chairman and CEO of Gilead Sciences, expressed the company’s eagerness to advance seladelpar, utilizing expertise in liver disease treatment to address the unmet needs of PBC patients. He recognized the significant work done by CymaBay in developing seladelpar, aiming to add it to Gilead’s range of therapeutic options.
PBC primarily affects women over 40 and can lead to liver malfunction and reduced quality of life. With the FDA granting seladelpar Breakthrough Therapy Designation for pruritus treatment in PBC patients without or with compensated cirrhosis, as well as Orphan Drug Designation in the U.S. and [PRIME](https://www.clinicaltrialvanguard.com/news/tobevibart-elebsiran-get-breakthrough-prime-for-chronic-hep-d/) status in Europe, seladelpar represents a marked advancement in PBC therapy.
The Phase 3 RESPONSE trial showcased seladelpar’s efficacy, achieving statistical significance over placebo in primary composite endpoints and showing a substantial reduction in pruritus among patients with moderate-to-severe symptoms.
CymaBay’s President and CEO, Sujal Shah, views the agreement with Gilead as the result of years dedicated to developing seladelpar and a step toward bringing new treatment opportunities to PBC patients and their families.
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**Categories:** News
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### [FDA OKs Xolair as Sole Drug for Multi-Food Allergies](https://www.clinicaltrialvanguard.com/news/fda-oks-xolair-as-sole-drug-for-multi-food-allergies/)
**Published:** February 19, 2024
**Author:** Jon Napitupulu
**Content:**
Genentech, a member of the Roche Group, has announced the FDA approval of Xolair® ([omalizumab](https://www.clinicaltrialvanguard.com/news/amneals-biosimilar-candidate-positive-topline-results/)) for reducing allergic reactions, including anaphylaxis, linked to accidental exposure to one or more foods in adults and pediatric patients one year of age and older with IgE-mediated food allergy. To avoid the risk of severe reactions, patients on Xolair for food allergies should continue to avoid known food allergens, and Xolair is not intended for emergency treatment of allergic reactions or anaphylaxis.
This FDA approval extends Xolair’s availability for patients with IgE-mediated food allergies, making it the first medicine of its kind to receive such approval. The drug aims to address the growing prevalence of food allergies and the demand for treatments to prevent potentially life-threatening allergic responses.
Dr. Levi Garraway, Genentech’s CMO and Head of Global Product Development, underscored the need for this treatment, mentioning its established efficacy and safety profile built over two decades since Xolair’s initial approval for allergic [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/).
The Phase III OUtMATCH NIH-sponsored study provided the positive data for approval, showing a significant proportion of patients (ranging from 1 to 55 years old) could tolerate small amounts of common food allergens. The study was conducted at various clinical sites across the U.S., including the Johns Hopkins Children’s Center and Stanford School of Medicine.
Results from OUtMATCH will be presented at a late-breaking symposium at the 2024 [AAAAI](https://www.clinicaltrialvanguard.com/news/cogent-highlights-bezuclastinib-summit-trial-data-at-aaaai/) Annual Meeting. Sung Poblete, CEO of Food Allergy Research and Education (FARE), emphasized the importance of new preventative approaches to safeguard against severe reactions due to food allergies, acknowledging the challenges faced by the affected community.
This approval positions Xolair as an important option for managing food allergies and reflects Genentech’s commitment to addressing unmet needs in the area of allergic diseases.
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**Categories:** News
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### [Tagrisso & Chemo Combo Approved in US for EGFR Lung Cancer](https://www.clinicaltrialvanguard.com/news/tagrisso-chemo-combo-approved-in-us-for-egfr-lung-cancer/)
**Published:** February 19, 2024
**Author:** Jon Napitupulu
**Content:**
AstraZeneca’s [TAGRISSO](https://www.clinicaltrialvanguard.com/news/tagrisso-approved-for-unresectable-stage-iii-egfr-mutated-lung-cancer/)® (osimertinib), in combination with chemotherapy, has been approved in the US for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). Following a Priority Review, the FDA based its approval on the results from the Phase III FLAURA2 trial, which demonstrated a significant extension in median progression-free survival (PFS) with the TAGRISSO combination versus TAGRISSO monotherapy.
The FLAURA2 trial findings revealed that TAGRISSO plus chemotherapy reduced the risk of disease progression or death by 38%, with PFS results showing median survival improvement by nearly 9 months over monotherapy. These outcomes were consistent across PFS assessments, indicating robust evidence for the efficacy of the combination.
NSCLC is the most prevalent form of lung cancer, accounting for the majority of diagnoses. In the US, approximately 15% of NSCLC patients have an EGFR mutation. This approval offers these patients an important new treatment option that has demonstrated the ability to delay disease progression, particularly beneficial for patients with a poorer prognosis, such as those with brain metastases or L858R mutations.
Pasi A. Jänne, principal investigator of the trial, emphasized the significance of this advancement in providing osimertinib-based treatment regimens, allowing physicians to optimize treatment plans and potentially achieve better patient outcomes.
Dave Fredrickson of AstraZeneca noted that this approval sets a new benchmark for PFS in first-line advanced EGFRm NSCLC treatment, solidifying TAGRISSO as the cornerstone of treatment in this setting, irrespective of the treatment approach.
Laurie Ambrose of GO2 for Lung Cancer celebrated this progress in offering more personalized treatment options to the lung cancer community, focusing on delivering the right treatments at the right time to improve outcomes.
The continued follow-up results from the FLAURA2 trial are a testament to the ongoing pursuit of innovative and impactful treatment options for NSCLC patients, contributing to the collective goal of improving survival and quality of life within the lung cancer community.
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**Categories:** News
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### [Datopotamab Deruxtecan BLA Accepted for US Lung Cancer](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-bla-accepted-for-us-lung-cancer/)
**Published:** February 20, 2024
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo and AstraZeneca’s [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) for datopotamab deruxtecan (Dato-DXd) has been accepted by the U.S. FDA for the treatment of adult patients with locally advanced or metastatic nonsquamous non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) who have previously received systemic therapy.
As a specifically constructed TROP2-targeted DXd antibody drug conjugate (ADC), datopotamab deruxtecan shows promise in addressing NSCLC, and the FDA is expected to make a regulatory decision by December 20, 2024.
The BLA is based on results from the pivotal TROPION-Lung01 phase 3 trial, which demonstrated datopotamab deruxtecan’s statistically significant improvement in progression-free survival (PFS) compared to docetaxel, the current standard of care, in patients who had previously undergone at least one line of therapy. The interim results for the dual primary endpoint of overall survival (OS) were numerically in favor of datopotamab deruxtecan but did not reach statistical significance at the time of data cut-off.
Ken Takeshita, MD, Global Head of R&D at Daiichi Sankyo, emphasized the significance of this development as they aim to establish a new standard of care for NSCLC patients. They are encouraged by the FDA’s acceptance of the BLA and are keen to make datopotamab deruxtecan the first TROP2-directed ADC for this patient group.
Susan Galbraith of AstraZeneca also noted the potential of datopotamab deruxtecan to provide an effective alternative to traditional chemotherapy for those with advanced nonsquamous NSCLC.
Simultaneously, a parallel BLA is pending for the treatment of metastatic hormone receptor-positive, HER2-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/), and additional global submissions are underway for both lung and breast cancer indications.
TROPION-Lung01 is a global, randomized, multicenter, open-label phase 3 trial exploring the efficacy and safety of datopotamab deruxtecan versus docetaxel in patients with NSCLC, marking a crucial stride toward innovative lung cancer treatments with the potential to be made available swiftly to patients worldwide.
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**Categories:** News
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### [EU Nods Pfizer's Velsipity® for Ulcerative Colitis](https://www.clinicaltrialvanguard.com/news/eu-nods-pfizers-velsipity-for-ulcerative-colitis/)
**Published:** February 21, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer Inc. announced that the European Commission has approved VELSIPITY® (etrasimod) for the treatment of patients aged 16 and over with moderately to severely active [ulcerative colitis](https://www.clinicaltrialvanguard.com/news/abivax-clears-pre-nda-meeting-with-fda-for-obefazimod-in-ulcerative-colitis/) (UC) in the European Union, including Iceland, Liechtenstein, and Norway. This approval is for those inadequate to conventional therapy or a biological agent.
Leading expert Séverine Vermeire, MD, PhD, has welcomed this approval, highlighting the debilitating impact of UC on 2.6 million Europeans and the need for effective treatment options beyond injectables.
The approval follows the positive opinion issued by the EMA’s CHMP in December 2023, along with prior approvals from the FDA in October 2023 and [Health Canada](https://www.clinicaltrialvanguard.com/news/amo-pharma-aligns-with-fda-mhra-health-canada-on-amo-02-study-design-for-cdm1/) in January 2024. Pfizer has submitted further regulatory applications for VELSIPITY internationally. Alexandre de Germay, Chief International Commercial Officer at Pfizer, praised VELSIPITY’s oral dosing and benefit-risk profile, marking it as a promising treatment option for eligible UC patients.
VELSIPITY’s approval was supported by successful results from the ELEVATE UC Phase 3 registrational program. The program’s trials, ELEVATE UC 52 and ELEVATE UC 12, included patients with isolated proctitis and demonstrated that VELSIPITY improved clinical remission rates in UC patients with prior therapy failures or intolerances. The studies saw VELSIPITY reach all primary and key secondary efficacy endpoints while maintaining a favorable safety profile, including improved health-related quality of life measures. The most common adverse reactions were lymphopenia and headache.
This approval introduces VELSIPITY as an advanced therapy option for UC that addresses remission and targets an unmet medical need within the immune-mediated inflammatory bowel disease space.
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**Categories:** News
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### [Tagrisso's Phase III Success in Unresectable EGFR Lung Cancer](https://www.clinicaltrialvanguard.com/news/tagrissos-phase-iii-success-in-unresectable-egfr-lung-cancer/)
**Published:** February 21, 2024
**Author:** Jon Napitupulu
**Content:**
AstraZeneca’s [TAGRISSO](https://www.clinicaltrialvanguard.com/news/tagrisso-approved-for-unresectable-stage-iii-egfr-mutated-lung-cancer/)® (osimertinib) has shown positive results in the LAURA Phase III trial, demonstrating a significant and clinically meaningful improvement in progression-free survival (PFS) for patients with unresectable, Stage III EGFR-mutated [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) following chemoradiotherapy (CRT), compared to placebo. While overall survival (OS) data is favoring TAGRISSO, it is not mature yet and will continue to be evaluated as a secondary endpoint of the trial.
The findings suggest TAGRISSO could be the first targeted treatment option for patients with Stage III EGFR-mutated NSCLC. Suresh Ramalingam, MD, emphasized the advancement these results represent for patients who face early disease progression and brain spread, and the lack of available targeted therapies for this stage.
Susan Galbraith from AstraZeneca noted that these trial outcomes could cement TAGRISSO as foundational therapy in treating EGFR-mutated lung cancer, with data from the ADAURA study emphasizing the importance of early diagnosis and treatment.
The safety and tolerability profile of TAGRISSO aligns with its established profile in the LAURA trial, and no new safety concerns surfaced for its maintenance treatment after CRT. The data will be presented at a forthcoming medical meeting and discussed with global regulatory authorities.
TAGRISSO’s recent US approval, based on the FLAURA2 Phase III trial in combination with chemotherapy, adds to AstraZeneca’s ongoing efforts to address lung cancer in its early stages. TAGRISSO is also being explored in the neoadjuvant setting via the NeoADAURA Phase III trial and the early-stage adjuvant resectable setting in the ADAURA2 Phase III trial, with results anticipated later in the year.
Lung cancer, typically split into NSCLC and small cell lung cancer, remains the leading cause of cancer death, with most NSCLC patients diagnosed at an advanced stage. EGFR-TKIs like TAGRISSO are crucial therapies for patients with EGFRm NSCLC, a subgroup highly sensitive to treatments that inhibit tumor cell growth signaling pathways. The progress in the LAURA trial indicates potential advancement in treating lung cancer at earlier stages, particularly for those with specific genetic mutations.
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**Categories:** News
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### [Silence Therapeutics Nabs $10M From AstraZeneca Phase 1 Start](https://www.clinicaltrialvanguard.com/news/silence-therapeutics-nabs-10m-from-astrazeneca-phase-1-start/)
**Published:** February 26, 2024
**Author:** Jon Napitupulu
**Content:**
Silence Therapeutics plc, a biotechnology company specializing in RNA interference (RNAi) therapies, has announced the initiation of a Phase 1 clinical trial by [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) for the first product candidate from their siRNA (short interfering RNA) collaboration. This marks a significant milestone for Silence Therapeutics, triggering a $10.0 million payment from AstraZeneca.
Craig Tooman, President and CEO of Silence, highlighted this as the first clinical milestone within the AstraZeneca partnership and a testament to the capabilities of their mRNAi GOLD™ platform. He also noted the advancements of Silence’s proprietary pipeline with promising data from their zerlasiran and divesiran programs targeting high Lp(a) and polycythemia vera, respectively.
Regina Fritsche Danielson from AstraZeneca emphasized the partnership’s aim to develop innovative therapies for cardiovascular, renal, metabolic, and respiratory diseases using Silence’s RNAi technology.
The collaboration, initiated in March 2020, allows AstraZeneca to leverage Silence’s mRNAi GOLD™ platform to develop siRNA therapeutics. The agreement, which may encompass up to ten targets, includes option fees, development, and commercialization milestones, and sales royalties for Silence.
Silence Therapeutics focuses on developing RNAi-based treatments to inhibit specific genes associated with diseases that have unmet medical needs, particularly targeting liver-associated conditions. Their wholly-owned product candidates, SLN360 and divesiran, address cardiovascular risk and hematological diseases. In addition to their work with AstraZeneca, Silence Therapeutics maintains collaborations with Mallinckrodt Pharmaceuticals, Hansoh Pharma, and others.
More information about Silence Therapeutics and their work can be found on their website, which also details the forward-looking statements made in the announcement.
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**Categories:** News
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### [Xolair's NEJM Phase III Data: Hope for Multiple Food Allergies](https://www.clinicaltrialvanguard.com/news/xolairs-nejm-phase-iii-data-hope-for-multiple-food-allergies/)
**Published:** February 26, 2024
**Author:** Jon Napitupulu
**Content:**
Genentech announced key findings from Stage 1 of the NIH-sponsored Phase III OUtMATCH study, published in the New England Journal of Medicine (NEJM) and presented at the 2024 [AAAAI](https://www.clinicaltrialvanguard.com/news/cogent-highlights-bezuclastinib-summit-trial-data-at-aaaai/) Annual Meeting. The study demonstrated that treatment with Xolair® ([omalizumab](https://www.clinicaltrialvanguard.com/news/amneals-biosimilar-candidate-positive-topline-results/)) raised the threshold amount of allergens such as peanuts, milk, egg, wheat, and tree nuts (cashew, hazelnut, and walnut) needed to cause moderate to severe allergic reactions in individuals with multiple food allergies, as young as 1 year old. These safety findings align with Xolair’s established safety profile across approved indications and prior clinical trials.
The U.S. FDA has recently approved Xolair for expanded use in children and adults with IgE-mediated food allergies, based on data from this OUtMATCH study.
Dr. Robert Wood, principal investigator of the OUtMATCH study, highlighted the debilitating nature of food allergies and the limited treatment advancements. The study results indicate that anti-IgE therapy, such as Xolair, could substantially reduce allergic reactions from accidental exposures across multiple foods.
Dr. R. Sharon Chinthrajah, co-lead study investigator, also emphasized the significant impact food allergies have on patients and their families. The OUtMATCH study findings suggest that Xolair treatment can increase most patients’ thresholds for allergic reactions, potentially mitigating the risks associated with accidental exposures.
In total, 180 patients aged from 1 to 55 years entered Stage 1 of the study with intolerance to specific quantities of allergens. Following treatment, patients could tolerate higher amounts of these allergens without severe reactions. This points to Xolair as an important new treatment option that could reduce the risk of severe allergic responses to food for individuals with multiple allergies.
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**Categories:** News
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### [Merck's Keytruda Gets Positive CHMP Opinion for NSCLC Therapy](https://www.clinicaltrialvanguard.com/news/mercks-keytruda-gets-positive-chmp-opinion-for-nsclc-therapy/)
**Published:** February 26, 2024
**Author:** Jon Napitupulu
**Content:**
Merck announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/), Merck’s anti-PD-1 therapy, as a neoadjuvant treatment combined with platinum-containing chemotherapy, followed by KEYTRUDA as a monotherapy adjuvant treatment for [resectable non-small cell lung](https://www.clinicaltrialvanguard.com/news/u-s-fda-approves-opdivo-for-resectable-non-small-cell-lung-cancer/) cancer (NSCLC) at high risk of recurrence in adults.
This recommendation is based on the Phase 3 [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-671 trial outcomes, which showed a statistically significant improvement in both overall survival and event-free survival for patients treated with neoadjuvant KEYTRUDA and chemotherapy, followed by KEYTRUDA after surgery, compared to placebo plus chemotherapy followed by placebo.
Eligibility for KEYNOTE-671 trial included patients with previously untreated and resectable NSCLC with a high risk of recurrence, irrespective of tumor PD-L1 expression levels. The CHMP’s opinion is now under review by the European Commission for marketing authorization within the EU, with a final decision anticipated in the first half of 2024.
Dr. Marjorie Green, Senior Vice President at Merck Research Laboratories, expressed optimism for the CHMP’s recommendation, which brings them closer to offering support to patients in Europe with earlier stages of NSCLC.
KEYTRUDA was already approved in the U.S. in October 2023 for the treatment of patients with resectable NSCLC in a similar patient setting. Lung cancer remains the leading cause of cancer-related deaths globally, and despite improvements in detection, screening, and new therapies that have led to better survival rates, there is still a significant unmet need for early detection and screening, especially since many lung cancer cases remain undetected until they are advanced.
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**Categories:** News
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### [Mandara III Data: Fasenra Aids EGPA Remission, NEJM Reports](https://www.clinicaltrialvanguard.com/news/mandara-iii-data-fasenra-aids-egpa-remission-nejm-reports/)
**Published:** February 27, 2024
**Author:** Jon Napitupulu
**Content:**
Positive results from the MANDARA Phase III trial for FASENRA® ([benralizumab](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-trial-that-should-rewrite-the-rare-disease-playbook-but-probably-wont/)) in patients with eosinophilic granulomatosis with polyangiitis (EGPA) have been published in the New England Journal of Medicine. As the first head-to-head trial of biologics in patients with EGPA, the MANDARA trial demonstrated that more than half of the patients achieved remission with eosinophil-targeting biologic therapies, allowing them to taper off oral corticosteroids (OCS) while preventing relapses.
In the trial, benralizumab was compared to mepolizumab, another biologic treatment, and the study met its primary endpoint, showing non-inferior rates of remission compared to mepolizumab. Specifically, 59% of patients treated with benralizumab attained remission, defined as a Birmingham Vasculitis Activity Score (BVAS) of 0 and an OCS dose of 4 mg/day or less. Additionally, 41% of the benralizumab-treated patients could fully taper off OCS in the latter weeks of the study, compared to 26% in the mepolizumab arm.
These study results were also presented at the [AAAAI](https://www.clinicaltrialvanguard.com/news/cogent-highlights-bezuclastinib-summit-trial-data-at-aaaai/) Annual Meeting. Dr. Michael Wechsler, Coordinating Investigator of the MANDARA trial, noted the significance of the findings. Patients with EGPA often rely on long-term, high-dose OCS, which come with serious side effects and challenges in tapering off. The trial results confirm that eosinophil-targeting biologic therapies like FASENRA can help patients achieve remission and reduce chronic OCS use.
Sharon Barr from AstraZeneca emphasized the importance of the trial for the EGPA community, highlighting the convenience of FASENRA as a single, monthly subcutaneous injection that helps patients achieve remission and manage their disease.
Elevated eosinophil levels are central to EGPA pathophysiology, with all patients experiencing high eosinophil levels at some disease stage. Approximately half of the EGPA patients also have severe eosinophilic [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/) and often exhibit sinus and nasal symptoms.
FASENRA’s mode of action involves almost complete depletion of eosinophils, leading to greater improvements in patients’ ability to achieve remission and reduce reliance on OCS. These findings represent a significant advancement in EGPA management, providing patients with a potentially impactful treatment option that may address the challenges of this debilitating condition.
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**Categories:** News
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### [FDA Priority Review for Epcoritamab in Refractory Lymphoma](https://www.clinicaltrialvanguard.com/news/fda-priority-review-for-epcoritamab-in-refractory-lymphoma/)
**Published:** February 28, 2024
**Author:** Jon Napitupulu
**Content:**
Genmab A/S and [AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/) have received Priority Review from the FDA for [epcoritamab](https://www.clinicaltrialvanguard.com/news/genmab-abbvie-clarify-epcoritamab-phase-3-trial-missed-primary-endpoint/)-bysp, a bispecific antibody for the treatment of adults with relapsed or refractory (R/R) follicular [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (FL) after two or more lines of systemic therapy. This review status is granted to therapies with the potential to provide significant improvements over existing treatments for serious conditions and shortens the FDA review period to six months.
The FDA has set a target action date of June 28, 2024, for this application. The decision for Priority Review is grounded in the results from the Phase 1/2 EPCORE™ NHL-1 clinical trial, which demonstrated high overall and complete response rates in patients with R/R FL treated with epcoritamab. These promising data from the trial were presented at the ASH Annual Meeting and Exposition in December 2023.
Epcoritamab has previously received Breakthrough Therapy Designation (BTD) by the FDA, including additional data from the dose optimization part of the EPCORE NHL-1 trial. This BTD underscores the FDA’s recognition of epcoritamab’s potential to offer a significant benefit over existing therapies for this patient population.
Epcoritamab is being co-developed by Genmab and AbbVie under an oncology collaboration, with shared commercial responsibilities in the U.S. and Japan and AbbVie handling global commercialization. The EPCORE™ NHL-1 trial is examining epcoritamab’s safety and efficacy in patients with various types of relapsed, progressive, or refractory B-cell non-Hodgkin’s lymphomas (B-NHLs), including FL, who have limited treatment options.
With the FDA’s Priority Review, Genmab and AbbVie are one step closer to bringing this new treatment to patients affected by R/R FL, potentially transforming the therapeutic landscape for this challenging disease.
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### [SCRI & AstraZeneca Forge Alliance for Cancer Research Tech](https://www.clinicaltrialvanguard.com/news/scri-astrazeneca-forge-alliance-for-cancer-research-tech/)
**Published:** March 1, 2024
**Author:** Jon Napitupulu
**Content:**
Sarah Cannon Research Institute (SCRI), a global leader in conducting community-based clinical trials, has announced a collaboration with [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) to advance the delivery of [oncology clinical trials](https://www.clinicaltrialvanguard.com/executiveinterviews/how-to-achieve-remarkable-results-in-oncology-clinical-trials/) through innovative technology and operational synergies. The partnership focuses on expediting timelines for clinical trials, reducing site burden, and increasing U.S. enrollment.
Genospace, a precision medicine platform, and SCRI Development Innovations have developed a proprietary technology that integrates data directly from the Electronic Health Record (EHR) to the Electronic Data Capture (EDC) system (EHR2EDC or E2E) for clinical trial data collection. This technology has already shown benefits by decreasing the time required for on-site data entry, reducing manual labor and monitoring costs, enhancing data quality, and speeding up overall clinical decision-making.
Central to the collaboration’s efforts is the new model from SCRI, which aims to synchronize end-to-end clinical research management across a vast network of physicians and sites, providing trial access to a significant portion of cancer patients in the U.S.
Dee Anna Smith, CEO of SCRI, emphasized the potential impact of improved research operations on the drug development industry, enabling faster introduction of novel therapies through a more extensive network of physicians and communities.
Michele Sample, Vice President of Clinical Operations in Oncology R&D at AstraZeneca, underscored the commitment to improving accessibility to trial participation and addressing the evolving needs of clinical trial execution. By streamlining the data collection process, the collaboration aims to diversify clinical trial participants and enhance the efficiency of trials in the U.S.
SCRI, with a rich history of advancing therapies for patients over the last three decades, has led groundbreaking drug development, conducting numerous first-in-human trials and contributing to the research behind the majority of new cancer therapies approved by the FDA. The joint venture with former US Oncology Research in 2022 was established to expand clinical trial access further nationwide.
This partnership between SCRI and AstraZeneca marks an ambitious step toward delivering new treatments to patients more efficiently while leveraging a comprehensive network of physician-researchers and trial sites across the United States.
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### [AbbVie, OSE Partner for Novel Anti-Inflammatory Antibody](https://www.clinicaltrialvanguard.com/news/abbvie-ose-partner-for-novel-anti-inflammatory-antibody/)
**Published:** March 4, 2024
**Author:** Jon Napitupulu
**Content:**
[AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/) Inc. and OSE Immunotherapeutics SA have announced a strategic partnership to develop OSE-230, a monoclonal antibody in the pre-clinical development stage and designed to treat chronic and severe inflammation. OSE-230 is a first-in-class antibody that activates ChemR23, a G-protein-coupled receptor (GPCR), which could offer a novel approach to resolving chronic inflammation by modulating macrophages and neutrophils.
Jonathon Sedgwick, Ph.D., Senior Vice President at AbbVie, highlighted the collaboration as a testament to AbbVie’s dedication to expanding its immunology portfolio and improving care for patients with inflammatory diseases. Nicolas Poirier, CEO of OSE Immunotherapeutics, expressed satisfaction with partnering with AbbVie and recognized the milestone as an acknowledgment of the company’s innovative research and development efforts.
Under the partnership terms, AbbVie will receive an exclusive global license to develop, manufacture, and commercialize OSE-230, with OSE Immunotherapeutics receiving a $48 million upfront payment and up to an additional $665 million in potential clinical development, regulatory, and commercial milestones, as well as tiered royalties on global net sales of OSE-230.
The agreement is contingent upon customary closing conditions, including the waiting period stipulated by the Hart-Scott-Rodino Antitrust Improvements Act. This collaboration signifies a substantial advancement in addressing chronic inflammation through innovative therapeutic development.
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### [Real-World Data Back Veklury for Hospitalized COVID-19 Patients](https://www.clinicaltrialvanguard.com/news/real-world-data-back-veklury-for-hospitalized-covid-19-patients/)
**Published:** March 6, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences revealed new data from three retrospective studies that suggest its antiviral drug Veklury (remdesivir) not only reduces mortality among [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) patients, but also lowers the risk of long COVID symptoms. Based on HealthVerity data from over 52,000 patients, Veklury use was linked to a 10% lower risk of conditions associated with long COVID in both people under and over the age of 65. Separate research analysed patients during the Omicron surge (Dec’21 – Apr’23), showing that immunocompromised individuals treated with Veklury had a 25% lower mortality risk compared to a control group. A final study found lower mortality rates in patients treated with Veklury in combination with dexamethasone compared to those on dexamethasone alone.
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**Categories:** News
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### [EU Validates Datopotamab Apps for Lung & Breast Cancer](https://www.clinicaltrialvanguard.com/news/eu-validates-datopotamab-apps-for-lung-breast-cancer/)
**Published:** March 6, 2024
**Author:** Jon Napitupulu
**Content:**
The European Medicines Agency (EMA) has validated two marketing authorization applications (MAAs) for Daiichi Sankyo and AstraZeneca’s datopotamab deruxtecan, a treatment for two types of cancer. The two MAAs are for the treatment of adults with locally advanced or metastatic non-squamous [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC), and for the treatment of adults with inoperable or metastatic hormone receptor (HR) positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). The applications were validated on the basis of data from the pivotal TROPION-Lung01 and TROPION-Breast01 phase three trials presented at the 2023 European Society for Medical Oncology Congress. Additional regulatory submissions for datopotamab deruxtecan in lung cancer and breast cancer are underway in the US and globally.
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**Categories:** News
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### [Viiv Healthcare Presents Promising Cabenuva Data at CROI](https://www.clinicaltrialvanguard.com/news/viiv-healthcare-presents-promising-cabenuva-data-at-croi/)
**Published:** March 7, 2024
**Author:** Jon Napitupulu
**Content:**
ViiV Healthcare has announced promising interim data from the [LATITUDE](https://www.clinicaltrialvanguard.com/news/actg-latitude-study-findings-published-in-new-england-journal/) phase III trial, presented at the CROI 2024. The data indicate that Cabenuva (cabotegravir + rilpivirine), a long-acting injectable antiretroviral treatment (ART) for HIV, shows superior efficacy in maintaining viral load suppression compared to daily oral therapy, particularly benefiting individuals facing ART adherence challenges.
The US sees an estimated one-third of people living with HIV struggling to maintain viral suppression. The LATITUDE study demonstrates that long-acting injectable cabotegravir + rilpivirine can potentially improve health outcomes for this group by providing an alternative treatment option that simplifies adherence. Moreover, achieving undetectable viral loads can contribute to preventing virus transmission to sexual partners, adding to efforts in ending the HIV epidemic.
LATITUDE, a randomized, open-label study, involved participants who initially received comprehensive adherence support with guideline-recommended oral ART regimens to achieve viral suppression. Eligible participants who achieved suppression were then randomized to either continue with their oral standard of care (SOC) regimen or switch to LA-ART administered monthly. Despite not meeting the strict predefined stopping criterion for the interim analysis, the study’s outcomes favored LA-ART over SOC, with significant differences in key secondary endpoints of virologic failure and treatment-related failure.
Taking into account the totality of evidence, the study’s Data Safety Monitoring Board (DSMB) concluded that long-acting ART demonstrated superior efficacy over daily oral SOC. Consequently, the DSMB recommended that all eligible participants be offered the long-acting injectable option. The safety profiles were similar across the two arms, with a few serious injection site reactions reported in the LA-ART arm, and resistance-associated mutations were detected in a small number of virologic failures in each treatment arm.
This promising data underline the importance of providing diverse treatment options tailored to meet varying patient needs in HIV management.
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**Categories:** News
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### [Merck Successfully Acquires Harpoon Therapeutics, Inc.](https://www.clinicaltrialvanguard.com/news/merck-successfully-acquires-harpoon-therapeutics-inc/)
**Published:** March 12, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has successfully completed the acquisition of Harpoon Therapeutics, Inc., making Harpoon a wholly-owned subsidiary of Merck. As a result of this acquisition, Harpoon’s common stock will no longer be listed or publicly traded on the Nasdaq Stock Market. This strategic move is part of Merck’s ongoing efforts to bolster and diversify its oncology pipeline through innovative cancer treatment approaches.
Harpoon’s lead candidate, MK-6070 (formerly HPN328), is a T-cell engager that targets delta-like ligand 3 (DLL3), a protein highly expressed in [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (SCLC) and neuroendocrine tumors. Currently, MK-6070 is under evaluation in a Phase 1/2 clinical trial as monotherapy for patients with advanced cancers that express DLL3. Additionally, the study is exploring the use of MK-6070 in combination with [atezolizumab](https://www.clinicaltrialvanguard.com/news/xilio-announces-updated-phase-2-data-for-vilastobart/) for patients with SCLC. In recognition of its potential, the U.S. FDA granted Orphan Drug Designation to MK-6070 for the treatment of SCLC in March 2022.
Beyond MK-6070, Harpoon’s pipeline includes HPN217, a BCMA-targeting T-cell engager in Phase 1 development for relapsed/refractory multiple myeloma, and several preclinical candidates like HPN601, which targets the epithelial cell adhesion molecule (EpCAM) for patients with EpCAM-expressing tumors.
As per the merger agreement terms, Merck acquired all outstanding shares of Harpoon, resulting in a non-tax deductible R&D expense charge of approximately $650 million for Merck. The anticipated impact of this transaction on Merck’s full-year non-GAAP EPS is approximately $0.26 per share, already accounted for in Merck’s financial outlook for 2024.
Merck’s dedication to transforming cancer treatment emphasizes the importance of immuno-oncology, with extensive development programs exploring over 30 tumor types. Moreover, Merck continues to enhance its oncology portfolio via strategic acquisitions and prioritizes the development of promising candidates that could advance the treatment of advanced cancers. Further information about Merck’s [oncology clinical trials](https://www.clinicaltrialvanguard.com/executiveinterviews/how-to-achieve-remarkable-results-in-oncology-clinical-trials/) can be found at their dedicated website.
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### [Pfizer's Phase 3 Trial Shows Positive Survival in DLBCL](https://www.clinicaltrialvanguard.com/news/pfizers-phase-3-trial-shows-positive-survival-in-dlbcl/)
**Published:** March 13, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer Inc. has announced promising Phase 3 study results for ADCETRIS® (brentuximab vedotin) in combination with lenalidomide and rituximab for treating relapsed/refractory diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) ([DLBCL](https://www.clinicaltrialvanguard.com/news/genmabs-epcoritamab-plus-lenalidomide-meets-phase-3-goal-in-relapsed-dlbcl/)). The study demonstrated a statistically significant improvement in overall survival (OS) compared to lenalidomide and rituximab plus placebo. Key secondary endpoints, such as progression-free survival (PFS) and overall response rate (ORR), also showed positive outcomes. The safety profile of ADCETRIS aligned with previous findings. The full data is set for presentation at an upcoming medical meeting.
ADCETRIS, an antibody-drug conjugate, targets CD30 and has become a care standard for certain lymphomas. It is approved for seven indications in the U.S, serving over 55,000 patients since 2011 and globally treating more than 140,000 patients.
The ECHELON-3 trial, international in scope, explored ADCETRIS plus lenalidomide and rituximab against lenalidomide and rituximab with placebo in patients having received two or more lines of therapy who were ineligible for stem cell transplant or CAR-T therapy. The primary outcome focused on OS, with PFS and ORR as secondary endpoints.
DLBCL, the most common lymphoma type, presents significant treatment challenges with up to 40% of patients experiencing relapse or refractory disease post-first-line treatment. Pfizer intends to discuss the ECHELON-3 data with the FDA to support regulatory filings in the U.S.
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### [FDA Approves Breyanzi® as First CAR-T Therapy for CLL/SLL](https://www.clinicaltrialvanguard.com/news/fda-approves-breyanzi-as-first-car-t-therapy-for-cll-sll/)
**Published:** March 15, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb announced the accelerated FDA approval of Breyanzi® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), for treating adults with relapsed or refractory chronic lymphocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior therapies, including a Bruton tyrosine kinase (BTK) inhibitor and a [B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) 2 (BCL-2) inhibitor.
This approval, based on the response rate and duration of the response in patients, marks Breyanzi as the first CAR T cell therapy option for relapsed or refractory CLL or SLL, delivered through a one-time infusion process. The therapy’s introduction is seen as a significant advancement for patients who have historically lacked a standard care for these conditions after becoming refractory or relapsing following early-line therapies.
The TRANSCEND CLL 004 study, a Phase 1/2 trial, demonstrated that 20% of patients treated with Breyanzi achieved a complete response with median duration of response not reached, alongside an established safety profile.
CLL and SLL represent common types of B-cell lymphoma, often treated initially with BTK- and BCL-2 inhibitors. Yet, patients frequently experience relapse or develop resistance to these treatments, leaving few options and resulting in poor outcomes.
This FDA approval offers hope for better tailored and effective treatments to those affected, while also emphasizing the role of CAR T cell therapies in reshaping cancer treatment strategies.
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### [BMS Strengthens Neuroscience Portfolio with Karuna Therapeutics Acquisition](https://www.clinicaltrialvanguard.com/news/bms-strengthens-neuroscience-portfolio-with-karuna-therapeutics-acquisition/)
**Published:** March 19, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) has announced the completion of its acquisition of Karuna Therapeutics, Inc., marking a significant expansion of BMS’s neuroscience portfolio. With this acquisition, Karuna has ceased trading on the Nasdaq Global Select Market and is now a fully integrated subsidiary of BMS. This move consolidates BMS’s position in neuroscience and underscores its commitment to delivering innovative treatments to patients.
Karuna’s lead product, [KarXT](https://www.clinicaltrialvanguard.com/news/zai-labs-karxt-for-schizophrenia-treatment-accepted-by-fda/) (xanomeline-trospium), represents a potentially groundbreaking treatment for [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) in adults, offering a novel mechanism of action distinguished by its efficacy and safety profile. KarXT is under review with a Prescription Drug User Fee Act (PDUFA) date set for September 26, 2024. Moreover, the drug is currently undergoing registrational trials for use as an adjunctive therapy in schizophrenia and for treating psychosis in Alzheimer’s disease patients. It also holds potential for treating other conditions, including Bipolar I disorder and Alzheimer’s disease agitation.
The financial aspects of the acquisition reflect BMS’s strategic planning and robust financial health. Although the transaction is expected to result in a one-time, non-deductible charge of approximately $12 billion for Acquired In-Process Research and Development (Acquired IPR&D), impacting both 2024’s first quarter and full-year GAAP and non-GAAP EPS, BMS anticipates mitigating these costs through disciplined resource allocation, cost efficiencies, and portfolio prioritization. The acquisition is projected to dilute BMS’s non-GAAP diluted earnings per share by around $0.30 in 2024, mainly due to financing costs associated with new debt issuance.
BMS is committed to leveraging its strong financial profile to continue investing in growth, maintain investment-grade solid credit ratings, and provide shareholder returns through dividends and share repurchases. The company has engaged Gordon Dyal & Co. and Citi as financial advisors and Covington & Burling LLP as legal counsel for the transaction. Karuna utilized the expertise of Goldman Sachs & Co. LLC as its exclusive financial advisor and Simpson Thacher & Bartlett LLP as legal counsel.
BMS plans to provide an updated financial outlook, including the impacts of this transaction, when it reports its first-quarter 2024 results on April 25, 2024. This aligns with its practice of updating its financial outlook quarterly.
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**Categories:** News
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### [GRAIL Unveils Innovative Risk Classification Test for Lung Cancer Study](https://www.clinicaltrialvanguard.com/news/grail-unveils-innovative-risk-classification-test-for-lung-cancer-study/)
**Published:** March 19, 2024
**Author:** Jon Napitupulu
**Content:**
[GRAIL](https://www.clinicaltrialvanguard.com/news/grails-galleri-trial-misses-primary-endpoint-but-shows-stage-iv-cancer-reduction/), LLC, a pioneering healthcare company dedicated to early cancer detection for potential curative treatment, has partnered with [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) to include Japanese patients in studies utilizing GRAIL’s innovative Methylation Platform. This cutting-edge test, focused on blood-based detection, is set to classify the recurrence risk in patients newly diagnosed with Stage I lung adenocarcinoma, marking a significant advancement in non-invasive cancer diagnostics.
The collaboration with AstraZeneca aims to validate the test’s effectiveness in delivering rapid results within a 10-day timeframe without necessitating tumor tissue samples, thereby facilitating its use in global pharmaceutical clinical trials. GRAIL’s breakthrough lies in its tissue-free, blood-only methodology, promising a customizable dimensional approach to support precise oncological treatment plans and clinical trial selections.
In 2022, GRAIL and AstraZeneca embarked on a broad strategic collaboration to develop companion diagnostic assays aligned with AstraZeneca’s therapeutic innovations. Following this, in December 2023, GRAIL successfully announced the analytical and clinical validation of a novel prognostic test for Stage I lung adenocarcinoma.
Headquartered in Menlo Park, CA, with additional facilities in Washington, D.C., North Carolina, and the UK, GRAIL’s mission is to mitigate the global cancer burden through early detection technologies. GRAIL is at the forefront of designing a multi-cancer early-detection blood test using advanced next-generation sequencing, comprehensive clinical studies, and sophisticated data science. Despite its achievements, it’s important to note that GRAIL’s productions are yet to receive clearance or approval from the U.S. FDA. GRAIL operates as a subsidiary of Illumina, Inc., under separate management as per the European Commission’s Interim Measures Order.
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### [Takeda's Iclusig® Granted FDA Approval for Newly Diagnosed Ph+ ALL](https://www.clinicaltrialvanguard.com/news/takedas-iclusig-granted-fda-approval-for-newly-diagnosed-ph-all/)
**Published:** March 20, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda has announced that the U.S. Food and Drug Administration (FDA) has approved ICLUSIG® (ponatinib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (Ph+ ALL) in combination with chemotherapy. This landmark approval makes ICLUSIG the first and only targeted therapy in the U.S. for frontline Ph+ ALL, distinguishing itself by its utilization of minimal residual disease (MRD)-negative complete remission (CR) as a novel primary endpoint for approval.
The FDA’s accelerated approval is based on the results from the Phase 3 PhALLCON Trial, where ICLUSIG demonstrated superior MRD-negative CR rates compared to imatinib, a previously available treatment, alongside a comparable safety profile. MRD-negative CR, a crucial prognostic indicator, reflects both molecular and clinical responses which are key for long-term patient outcomes.
This approval, granted Priority Review and evaluated under the Real-Time Oncology Review (RTOR) program, symbolizes a significant step forward in the treatment of Ph+ ALL, a rare and aggressive cancer. Takeda’s commitment to addressing unmet needs in cancer care is evident in this development, offering a new hope for enhanced patient outcomes. Further confirmation of ICLUSIG’s clinical benefits is expected through confirmatory trials, as required for continued FDA approval under the accelerated approval pathway.
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### [JCR Pharmaceuticals Achieves Milestone with J-Brain Cargo® in Neurodegenerative Disease Research Collaboration with Alexion](https://www.clinicaltrialvanguard.com/news/jcr-pharmaceuticals-achieves-milestone-with-j-brain-cargo-in-neurodegenerative-disease-research-collaboration-with-alexion/)
**Published:** March 20, 2024
**Author:** Jon Napitupulu
**Content:**
[JCR Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/jcr-pharmaceuticals-innovative-gene-therapy-research-unveiled-at-lysosomal-forum/) Co., Ltd. (JCR), a leading specialty pharmaceutical company, has announced the achievement of a significant research milestone in its collaboration with Alexion, AstraZeneca Rare Disease (Alexion), focusing on the development of a novel therapeutic molecule for the treatment of a neurodegenerative disease. This development leverages JCR’s proprietary J-Brain Cargo® technology, designed to enhance the penetration of [biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) across the blood-brain barrier (BBB). The accomplishment of this predefined research milestone has triggered a milestone payment to JCR from Alexion.
This recent progress strengthens the ongoing collaboration between JCR and Alexion, which expanded in December 2023 with a second research partnership. This additional collaboration aims to apply J-Brain Cargo® technology to [oligonucleotide](https://www.clinicaltrialvanguard.com/news/camp4s-pioneering-syngap-1-drug-enters-toxicology-studies/) therapeutics, targeting unmet medical needs in neurodegenerative diseases.
The milestone payment received by JCR is anticipated to have a minor impact on the company’s financial results for the fiscal year ending March 31, 2024. However, it represents a significant step forward in leveraging J-Brain Cargo® technology for developing treatments for neurodegenerative conditions.
The J-Brain Cargo® platform is a pioneering technology allowing for the delivery of therapeutic agents directly into the central nervous system by overcoming the BBB. IZGARGO® (INN: pabinafusp alfa), the first drug developed utilizing this technology, has been approved in Japan for treating Mucopolysaccharidosis (MPS) type II, also known as Hunter Syndrome. Several other drugs based on the J-Brain Cargo® platform are in various stages of global clinical trials, targeting different forms of MPS and other lysosomal storage disorders (LSDs). JCR plans to initiate clinical trials for four additional programs from its LSD pipeline by FY2028.
JCR Pharmaceuticals is dedicated to redefining the treatment landscape for rare and genetic diseases worldwide, building upon a 49-year legacy in Japan while expanding globally. The company focuses on delivering next-generation therapies and has a portfolio that includes approved treatments for growth disorder, Fabry disease, acute graft-versus-host disease, MPS II, and renal anemia, with several investigational products in development for rare diseases.
This forward-looking statement highlights JCR’s commitment to medical innovation and its strategy to accelerate the development of new treatments for rare diseases at a global level, underscoring its mission to expand possibilities for patients while fostering medical progress.
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**Categories:** News
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### [Cybin Closes $150 Million Private Placement](https://www.clinicaltrialvanguard.com/news/cybin-closes-150-million-private-placement/)
**Published:** March 20, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin Inc., a clinical-stage biopharmaceutical company focused on creating innovative psychedelic-based treatments for [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) disorders, recently completed a significant private placement, raising U.S. $150 million. The offering, which involved issuing 348,837,210 common shares at U.S. $0.43 each, witnessed considerable interest and was oversubscribed. Key investors in this private placement included Deep Track Capital, RA Capital Management, and several other institutional investors.
The proceeds from this private placement are primarily earmarked for advancing Phase 3 drug development activities concerning CYB003, a pioneering deuterated psilocybin analog being explored as a potential treatment for [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD). If CYB003 secures approval from the U.S. Food and Drug Administration (FDA), it would represent the first adjunctive psychedelic-based therapeutic for MDD treatment.
Doug Drysdale, CEO of Cybin, expressed gratitude for the invested capital, which he believes will significantly further the company’s next-generation psychedelic development programs. He acknowledged the widespread interest and support from the investor community, viewing it as validation of the company’s clinical efforts and its potential to fulfill unmet needs within mental health treatment.
The private placement was conducted in accordance with exemptions from the registration requirements under the United States Securities Act of 1933, as amended. Cybin has committed to taking the necessary steps to ensure the resale of the common shares issued during this private placement to specific non-Canadian purchasers, either through amendments to its existing registration statement on Form F-10 or through another suitable registration statement.
This strategic financial move marks a pivotal step for Cybin Inc. in its quest to innovate within the realm of mental healthcare, specifically targeting major depressive disorder with psychedelic-based solutions.
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**Categories:** News
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### [AstraZeneca Acquires Fusion to Advance Next-Gen Cancer Radioconjugates](https://www.clinicaltrialvanguard.com/news/astrazeneca-acquires-fusion-to-advance-next-gen-cancer-radioconjugates/)
**Published:** March 21, 2024
**Author:** Jon Napitupulu
**Content:**
[AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) has announced a definitive agreement to acquire Fusion Pharmaceuticals Inc., a clinical-stage biopharmaceutical company focusing on developing next-generation radioconjugates (RCs) for cancer treatment. This acquisition significantly advances AstraZeneca’s goal to revolutionize cancer therapy by incorporating more precise and targeted treatment methods, moving away from traditional chemotherapy and radiotherapy approaches.
Radioconjugates represent a cutting-edge treatment modality that delivers radioactive isotopes specifically to cancer cells, minimizing damage to healthy tissue and accessing unreachable tumors by external radiotherapy. One of the highlights of this acquisition is Fusion’s advanced program, FPI-2265, targeting the prostate-specific membrane antigen (PSMA) for metastatic castration-resistant prostate cancer (mCRPC), which is currently in Phase II clinical trials.
The acquisition enriches AstraZeneca’s oncology pipeline with Fusion’s portfolio and introduces new expertise, particularly in actinium-based RCs. It also expands AstraZeneca’s research and development, manufacturing, and supply chain capabilities in the radioconjugate space, affirming its commitment to innovation in cancer treatment and its presence in Canada.
Susan Galbraith, Executive Vice President of Oncology R&D at AstraZeneca, emphasized the acquisition’s potential to transform cancer care through advanced radioconjugate therapies. John Valliant, CEO of Fusion, highlighted the merger’s capacity to combine Fusion’s radiopharmaceutical development expertise with AstraZeneca’s leadership in small molecules and biologics engineering, aiming to enhance patient outcomes.
Following the acquisition, Fusion will operate as a wholly-owned subsidiary of AstraZeneca, maintaining its operations in Canada and the US. As part of the acquisition details, AstraZeneca will acquire all of Fusion’s outstanding shares at $21.00 per share in cash at closing, with an additional non-transferable contingent value right of $3.00 per share contingent upon reaching a specified regulatory milestone.
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### [NIH Study on Syn-One Test® as Diagnostic Tool Published in JAMA](https://www.clinicaltrialvanguard.com/news/nih-study-on-syn-one-test-as-diagnostic-tool-published-in-jama/)
**Published:** March 21, 2024
**Author:** Jon Napitupulu
**Content:**
[CND Life Sciences](https://www.clinicaltrialvanguard.com/news/cnd-life-sciences-skin-biopsy-tool-a-breakthrough-for-parkinsons-diagnosis/)[Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/), a leading medical technology company specializing in neurodiagnostic tests, has announced a groundbreaking publication in the Journal of the American Medical Association (JAMA) detailing the efficacy of their Syn-One Test. The test, which utilizes a simple skin biopsy procedure, has proven remarkably effective in detecting phosphorylated alpha-synuclein (P-SYN), a key biomarker for synucleinopathies, in most patients.
The pivotal peer-reviewed study, “Skin Biopsy Detection of Phosphorylated α-Synuclein in Patients with Synucleinopathies,” involved a cross-sectional analysis of 428 patients across 30 sites. Those studied included individuals with clinically diagnosed [Parkinson](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-genetics-are-global-your-enrollment-plan-isnt/)’s disease (PD), dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF), alongside healthy subjects for comparison. The research supported by the National Institutes of Health (NIH) demonstrated detection rates of P-SYN in patients ranging from 92.7% in PD cases to 100% in PAF cases, with a notably low positivity rate of 3.3% among healthy subjects.
This discovery holds immense promise for neurodegeneration, particularly in improving diagnostic accuracy for PD, MSA, DLB, and PAF conditions. Misdiagnosis of these diseases, especially in their early stages, has been a pervasive challenge, further compounded by limited access to specialized neurological care. With over 30% of synucleinopathy cases potentially misclassified, mainly in early disease phases, the Syn-One Test offers a much-needed solution to accurately identifying these conditions, significantly benefiting clinical practice and drug development efforts.
The Syn-One Test serves as a vital tool for precise diagnosis and a quantifiable biomarker that can inform disease severity and progression assessments. This development is poised to revolutionize patient care by ensuring more timely and accurate diagnoses and advancing targeted therapies. As the neurodegeneration field moves towards precision diagnostics and treatments, tools like the Syn-One Test are instrumental in realizing these advancements, promising a brighter future for patients and families affected by these debilitating disorders.
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**Categories:** News
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### [Tevogen Bio's Investigational T Cell Therapy Effective Against JN.1 Variant](https://www.clinicaltrialvanguard.com/news/tevogen-bios-investigational-t-cell-therapy-effective-against-jn-1-variant/)
**Published:** March 21, 2024
**Author:** Jon Napitupulu
**Content:**
Tevogen Bio Holdings announced the enduring efficacy of its investigational [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) treatment, TVGN 489, against the latest dominant SARS-CoV-2 strains, including the highly mutated JN.1 variant. TVGN 489, a pioneering allogeneic immunotherapy, utilizes Cytotoxic CD8+ T lymphocytes (CTLs) specifically tailored to combat SARS-CoV-2 in high-risk patients and those suffering from Long COVID.
Leveraging its unique mechanism that targets multiple SARS-CoV-2 proteins, TVGN 489 has demonstrated its robustness by retaining activity against all previously encountered COVID-19 strains. Recent surveillance underscores the treatment’s broad-spectrum efficacy, with 96% of its CTLs remaining active against current variants. This marks a significant stride in the ongoing battle against COVID-19 and its evolving mutations.
This announcement follows the positive outcomes from a Phase I study conducted in January 2023. In this study, high-risk patients infected with various COVID-19 variants, including delta and variants of omicron, witnessed significant clinical improvements and a dramatic reduction in viral load. The study showcased the safety and therapeutic potential of TVGN 489, with no significant adverse events reported.
Tevogen commits to further validating these promising results in advanced clinical trials while continuously monitoring the immunotherapy’s effectiveness against new SARS-CoV-2 strains. The company’s strategic focus on developing innovative treatments addresses the urgent need for effective COVID-19 interventions, particularly for patients at high risk of severe outcomes and those grappling with Long COVID.
TVGN 489 leverages the ExacTcell platform to identify numerous viral genome peptides as CTL targets, offering a comprehensive approach to viral combat by targeting the entire COVID-19 genome instead of solely focusing on the Spike protein. This broad target spectrum has ensured the persistence of T-cell targets across the various stages of viral evolution, reducing the need for frequent formulation adjustments—an advantage over treatments like monoclonal antibodies, which have witnessed a rapid loss of targets due to mutations. This breakthrough represents a hopeful advancement in Tevogen Bio’s efforts to provide lasting solutions to the challenges posed by COVID-19.
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**Categories:** News
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### [BMS's Checkmate-9DW Trial Shows Opdivo Plus Yervoy Efficacy in Advanced Hepatocellular Carcinoma](https://www.clinicaltrialvanguard.com/news/bmss-checkmate-9dw-trial-shows-opdivo-plus-yervoy-efficacy-in-advanced-hepatocellular-carcinoma/)
**Published:** March 21, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMY) has revealed positive outcomes from the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -9DW clinical trial, showcasing the effectiveness of combining Opdivo (nivolumab) with Yervoy (ipilimumab) as a first-line treatment for patients with advanced hepatocellular carcinoma (HCC) who have not received prior systemic therapy. This combination treatment demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) compared to traditional treatments of [sorafenib](https://www.clinicaltrialvanguard.com/news/cares-310-study-final-analysis-published-in-the-lancet/) or lenvatinib, as assessed at a pre-specified interim analysis.
The trial’s results mark a pivotal advancement in treating advanced HCC, offering new hope to patients with limited treatment options. The safety profile of the Opdivo plus Yervoy combination remained consistent with previously reported outcomes and was deemed manageable with established protocols, with no new safety concerns arising.
Dana Walker, M.D., M.S.C.E., Vice President at Bristol Myers Squibb, emphasized the crucial need for more treatment options to enhance survival rates among patients with advanced-stage [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/). The noteworthy survival benefits observed in the CheckMate -9DW trial underscore the potential of the Opdivo plus Yervoy combination to outperform well-established tyrosine kinase inhibitor (TKI) treatment alternatives.
Bristol Myers Squibb plans to comprehensively evaluate the trial data and engage with health authorities and the scientific community to discuss these findings. Further details will be shared at an upcoming medical conference, highlighting the significance of this breakthrough in the treatment of hepatocellular carcinoma.
CheckMate -9DW is a Phase 3, open-label, randomized trial that compares the efficacy of Opdivo plus Yervoy against the investigator’s choice of sorafenib or lenvatinib monotherapy in patients with advanced HCC who have not undergone prior systemic therapy. Approximately 668 patients were randomized to receive either the immunotherapy combination, sorafenib, or lenvatinib oral capsules in the control arm. The trial’s primary goal is to assess overall survival, with key secondary endpoints including objective response rate and time to symptom deterioration.
Hepatocellular carcinoma stands as the most prevalent type of primary liver cancer and is among the leading causes of cancer-related deaths worldwide. Often diagnosed in advanced stages, treatment options for HCC have been limited, generally yielding poor outcomes. With HBV, HCV infections, metabolic syndrome, and nonalcoholic steatohepatitis (NASH) as primary causes, the incidence of HCC is expected to rise, underscoring the importance of innovative therapies like the combination of Opdivo and Yervoy in addressing this growing challenge.
Source link: [http://www.businesswire.com/news/home/20240319007104/en/Bristol-Myers-Squibb-Announces-CheckMate–9DW-Trial-Evaluating-Opdivo-nivolumab-Plus-Yervoy-ipilimumab-Meets-Primary-Endpoint-of-Overall-Survival-for-the-First-Line-Treatment-of-Advanced-Hepatocellular-Carcinoma](http://www.businesswire.com/news/home/20240319007104/en/Bristol-Myers-Squibb-Announces-CheckMate--9DW-Trial-Evaluating-Opdivo-nivolumab-Plus-Yervoy-ipilimumab-Meets-Primary-Endpoint-of-Overall-Survival-for-the-First-Line-Treatment-of-Advanced-Hepatocellular-Carcinoma)
**Categories:** News
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### [eGenesis Achieves Breakthrough: First Successful Transplant of Genetically Engineered Porcine Kidney](https://www.clinicaltrialvanguard.com/news/egenesis-achieves-breakthrough-first-successful-transplant-of-genetically-engineered-porcine-kidney/)
**Published:** March 22, 2024
**Author:** Jon Napitupulu
**Content:**
eGenesis, a pioneering biotechnology company, has announced a groundbreaking advancement in transplantation with the successful transplantation of a genetically engineered porcine kidney into a living human recipient. This historic procedure was conducted at Massachusetts General Hospital (MGH) under the leadership of Drs. Tatsuo Kawai and Nahel Elias was authorized by the U.S. Food & Drug Administration (FDA) under an Expanded Access protocol, marking a significant step towards solving the critical global organ shortage.
The patient, suffering from end-stage renal disease and without other viable treatment options due to the loss of vascular access for dialysis, is reported to be in good condition and recovering well post-surgery. This achievement holds immense promise for the millions worldwide affected by kidney failure, offering a potential end to the dire scarcity of transplantable organs.
The demand for kidney transplants significantly outstrips the supply, with over 90,000 individuals on the kidney waitlist in the U.S. alone and only about 25,000 transplants performed annually. eGenesis’s breakthrough in developing human-compatible donor organs could dramatically reduce waitlist mortality and revolutionize organ transplantation.
The eGenesis donor kidney, known as [EGEN-2784](https://www.clinicaltrialvanguard.com/news/egenesis-advances-kidney-transplant-program-with-191m-series-d-funding/), features an advanced genetic design that includes the knockout of genes involved in hyperacute rejection, insertion of human transgenes to modulate rejection pathways, and inactivation of endogenous retroviruses in the porcine genome. This comprehensive genetic modification approach is unique to eGenesis and addresses critical safety and efficacy considerations necessary for successful xenotransplantation.
Michael Curtis, Ph.D., CEO of eGenesis, emphasized the transformative potential of this achievement to eliminate organ supply as a constraint to transplantation. He expressed gratitude for the patient’s pioneering spirit and collaboration with the esteemed MGH team. The procedure’s success and the robust preclinical evidence supporting long-term recipient survival with EGEN-2784 instill confidence in the clinical application of this technology.
The immunosuppression regimen includes approved agents and a novel investigational monoclonal antibody, hinting at integrating innovative pharmacological strategies to support the acceptance of genetically engineered organs. This landmark achievement signifies a new era in medical science and embodies hope for countless patients and families awaiting life-saving transplants.
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**Categories:** News
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### [Digital Biomarkers Market 2024-2029: Virtual Research & Mental Health Applications Drive Growth](https://www.clinicaltrialvanguard.com/news/digital-biomarkers-market-2024-2029-virtual-research-mental-health-applications-drive-growth/)
**Published:** March 22, 2024
**Author:** Jon Napitupulu
**Content:**
The “Global Digital Biomarkers Market – Outlook & Forecast 2024-2029” report showcases the rapidly expanding digital biomarkers market, which was valued at USD 2.49 billion in 2023 and is projected to soar to $8.59 billion by 2029, growing at an impressive CAGR of 22.86%. This growth underscores the transformative role of digital biomarkers in revolutionizing healthcare by leveraging technology to improve the collection, analysis, and interpretation of health data. This evolution offers healthcare professionals innovative tools for more precise diagnosis and management of diseases, leading to enhanced patient care and system efficiency.
Data privacy and security emerge as critical concerns with the uptick in personal health data collection. Companies that ensure robust security measures and earn user trust will stand out in this competitive landscape. Geographically, North America leads the market, with over 46% of the global share in 2023, propelled by advanced technologies, a focus on personalized healthcare, and the rising adoption of wearable devices.
The market’s growth is further fueled by an aging population, spiraling healthcare costs, and a pivot towards value-based care. The [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic has also accelerated this growth by increasing the acceptance of remote monitoring and telehealth services. Significant trends influencing the market include:
1. **Rise in Digitized Virtual Clinical Research:** Advances in remote monitoring technologies have enabled more efficient, patient-centric clinical trials, facilitating the development and validation of digital biomarkers across healthcare.
2. **Increasing Focus on Drug Development Using Digital Biomarkers:** There’s a growing trend of using digital biomarkers in pharmaceutical and biotech sectors to streamline clinical trials, improve patient recruitment, and accelerate new therapy development through real-time data analysis.
3. **Expanding Applications in [Mental Health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/):** Digital biomarkers are increasingly utilized to monitor and interpret behavioral patterns, offering critical insights into mental health management. This marks a significant shift towards personalized, data-driven mental healthcare strategies.
4. **Growing Collaborations & Partnerships Among Vendors:** The market is witnessing an upsurge in collaborations and partnerships among vendors, combining expertise, technologies, and resources to drive innovation and broaden digital biomarkers’ applications in healthcare.
These trends highlight the market’s dynamic nature, indicating a promising future for digital biomarkers in enhancing global healthcare delivery and outcomes.
Source link: [http://www.businesswire.com/news/home/20240321729591/en/Global-Digital-Biomarkers-Market-Outlook-Forecast-2024-2029-Rise-in-Digitized-Virtual-Clinical-Research-Increasing-Focus-on-Drug-Development-and-Expanding-Applications-in-Mental-Health—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240321729591/en/Global-Digital-Biomarkers-Market-Outlook-Forecast-2024-2029-Rise-in-Digitized-Virtual-Clinical-Research-Increasing-Focus-on-Drug-Development-and-Expanding-Applications-in-Mental-Health---ResearchAndMarkets.com)
**Categories:** News
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### [Gilead Sciences Successfully Acquires CymaBay](https://www.clinicaltrialvanguard.com/news/gilead-sciences-successfully-acquires-cymabay/)
**Published:** March 25, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences, Inc. has finalized its acquisition of [CymaBay](https://www.clinicaltrialvanguard.com/news/gilead-boosts-liver-portfolio-by-acquiring-cymabay/) Therapeutics, Inc. for an approximate total equity value of $4.3 billion. This strategic move integrates seladelpar, CymaBay’s investigational product for treating primary biliary cholangitis (PBC), including symptoms like pruritus, into Gilead’s robust liver disease portfolio. This acquisition aligns with Gilead’s commitment to providing innovative treatments for liver diseases, leveraging their established legacy over the past two decades in treating and curing such conditions.
Daniel O’Day, Gilead’s Chairman and CEO, underscored the importance of seladelpar as a potentially transformative therapy for PBC, expressing gratitude towards the CymaBay team for their dedication to addressing the unmet needs of patients with liver diseases. Gilead looks forward to furthering the development of seladelpar and continuing its mission to enhance liver disease treatments.
Following the announcement of a definitive merger agreement on February 12, 2024, Gilead initiated a successful tender offer to acquire all outstanding shares of CymaBay at $32.50 per share on February 23, 2024. This tender offer was successfully completed on March 22, 2024, with approximately 77.3% of CymaBay’s outstanding shares tendered. An additional 5,095,996 shares were delivered through Notices of Guaranteed Delivery, representing about 4.2% of the shares outstanding.
Following the merger completion on March 22, 2024, CymaBay has become a wholly owned subsidiary of Gilead, with CymaBay’s common stock being delisted from the Nasdaq Global Select Market. This transaction is anticipated to be accounted for as an asset acquisition and is projected to reduce Gilead’s GAAP and non-GAAP 2024 EPS by approximately $3.10 – $3.20, considering acquisition costs, associated operating expenses, and the impact on interest income.
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**Categories:** News
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### [BMS Enhances Health Equity Grant Initiatives for Better Health Outcomes](https://www.clinicaltrialvanguard.com/news/bms-enhances-health-equity-grant-initiatives-for-better-health-outcomes/)
**Published:** March 27, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMY) has launched a $1.8 million initiative to advance [health equity](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/) by addressing the social determinants of health (SDoH) in Brazil, India, Thailand, and the United Kingdom. This initiative represents a crucial component of the company’s larger commitment to invest $150 million in health equity by 2025, illustrating a significant step towards bridging healthcare disparities globally.
The WHO defines social determinants of health as non-medical factors influencing health outcomes in various areas, including income, education, physical environment, and healthcare services. Acknowledging these factors’ profound impact on health outcomes, Bristol Myers Squibb’s new health equity grants aim to tackle the root causes of healthcare inequities.
Cari Gallman, Executive Vice President, Corporate Affairs at Bristol Myers Squibb, emphasized the challenge that access to healthcare poses for many patients worldwide. She highlighted the company’s dedication to ensuring that all individuals, regardless of geographical location, can access high-quality healthcare services. This is aligned with the United Nations’ Sustainable Development Goal #3, focusing on ensuring healthy lives and promoting well-being for populations across all age groups.
The grants will support efforts by eight organizations working within underserved communities to address health inequities, particularly among individuals living with cancer and blood disorders. These organizations were selected for their potential to create sustainable, community-level impacts. The outcomes from this pilot program will inform potential future expansions to other regions and communities, aiming at dismantling systemic barriers to healthcare and improving access and health outcomes.
This initiative by Bristol Myers Squibb underscores the company’s broader strategy towards health equity, which involves moving beyond traditional healthcare parameters. It underscores the importance of engaging community voices and forming cross-sector partnerships to co-create solutions that achieve meaningful and equitable health outcomes across the globe.
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**Categories:** News
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### [Volastra Therapeutics Forms New Partnerships to Advance KIF18A Inhibitors in Cancer](https://www.clinicaltrialvanguard.com/news/volastra-therapeutics-forms-new-partnerships-to-advance-kif18a-inhibitors-in-cancer/)
**Published:** March 27, 2024
**Author:** Jon Napitupulu
**Content:**
Volastra Therapeutics, a clinical-stage biotechnology company at the forefront of developing cancer treatments targeting chromosomal instability (CIN), has announced strategic collaborations with Microsoft, Function Oncology, and Tailor Bio. These partnerships aim to enhance the potential of Volastra’s therapeutic pipeline by identifying predictive biomarkers across various tumor types. This initiative can potentially extend the application of Volastra’s [KIF18A](https://www.clinicaltrialvanguard.com/news/kif18a-therapies-fda-ema-approvals-trials-and-indications/) inhibitors well beyond cancers characterized by universally high levels of CIN.
The initiative capitalizes on Volastra’s expertise in CIN, a fundamental cancer hallmark linked to faster disease progression, increased therapy resistance, and reduced patient survival. Despite CIN’s prevalence in cancer, its levels greatly vary across different tumor types, necessitating a biomarker-defined approach to identify patients who would most benefit from Volastra’s treatments.
Charles Hugh-Jones, M.D., FRCP, CEO of Volastra, emphasized the importance of predictive biomarkers in extending their therapies’ reach. By collaborating with distinguished partners, Volastra aims to address even those cancers with heterogeneous CIN levels.
Since 2020, Volastra has worked with Microsoft Research to develop AI-based models that accurately quantify CIN in patient samples, a challenging and costly task for manual processes. Jonathan Carlson, Ph.D., from Microsoft Research Health Futures, expressed excitement about their joint progress in creating AI techniques for identifying CIN’s visual hallmarks and looks forward to further supporting Volastra’s mission.
Complementing this visual approach, Function Oncology and Tailor Bio engagements bring unique precision medicine expertise. Function Oncology’s [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/) platform is set to uncover patient-specific drug target vulnerabilities, with co-founder Srinath Sampath, M.D., Ph.D., highlighting their capability to reveal genetic truths about cancer target dependence. Concurrently, Tailor Bio’s co-founder Jason Yip, MBA, spoke about the potential of genomic CIN signatures, as presented in a recent Nature publication, to quantify aspects of chromosomal instability. Tailor Bio’s precision medicine platform is expected to contribute significantly to clinical trials exploring KIF18A inhibition.
Through these partnerships, Volastra endeavors to accelerate the development and broad application of its innovative therapeutics, ultimately aiming to benefit a wider spectrum of cancer patients.
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**Categories:** News
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### [Trisalus Life Sciences Technology Spotlighted in SIR Annual Scientific Meeting](https://www.clinicaltrialvanguard.com/news/trisalus-life-sciences-technology-spotlighted-in-sir-annual-scientific-meeting/)
**Published:** March 27, 2024
**Author:** Jon Napitupulu
**Content:**
TriSalus [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/)® Inc., an oncology company specializing in novel delivery technologies combined with immunotherapy for liver and pancreatic tumors, showcased its groundbreaking technology through two oral presentations at the Society of Interventional Radiology Annual Scientific Meeting. The presentations highlighted the promising developments of the TriSalus Infusion System and its Pressure-Enabled Drug Delivery™ (PEDD™) method in clinical trials and retrospective studies for treating various tumor types.
In the Phase 1/1b PERIO-03 trial, TriSalus examined the delivery of nelitolimod (formerly SD-101), a class C toll-like receptor-9 (TLR9) agonist, in patients with locally advanced pancreatic adenocarcinoma. The treatment, administered using the PEDD method with the TriSalus Infusion System through a retrograde venous approach, yielded encouraging early safety and feasibility data. All five patients treated demonstrated technical success, with no immediate procedural complications or evidence of hemorrhage or thrombosis. This trial, part of TriSalus’s broader effort to harness PEDD for enhanced therapeutic delivery, indicated promising immune responses within the treated pancreatic tumors, aligning to improve patient outcomes.
Additionally, a retrospective clinical study from The University of Texas MD Anderson Cancer Center focused on using the TriNav microcatheter to embolize hypovascular solid tumors, including those in the liver, bone, and adrenal gland. The study reported a 100% technical success rate, showcasing the effectiveness of the PEDD method in treating tumors typically challenging for embolization. Local tumor progression-free survival rates highlighted the potential of this technology to impact patient care significantly.
These findings underscore TriSalus’s commitment to leveraging novel drug delivery technologies to overcome the challenges of solid tumor treatments, particularly in liver and pancreas cancers. Steven C. Katz, M.D., FACS, Chief Medical Officer at TriSalus, emphasized the potential of the PEDD method to enhance therapeutic uptake and clinical outcomes across various disease sites. The successful integration of such innovative technologies into clinical practice, supported by the Centers for Medicare & Medicaid Services’ recent reimbursement code for procedures involving the TriNav system, marks a significant advancement in treating cancer patients.
The Pressure-Enabled Regional Immuno-Oncology (PERIO) clinical trials continue to explore the utility of delivering investigational treatments like nelitolimod via TriSalus’s proprietary technology, underscoring the company’s dedication to advancing precision medicine and improving the lives of those affected by cancer.
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**Categories:** News
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### [Photopharmics Initiates Largest Phototherapy Trial for Parkinson's Disease](https://www.clinicaltrialvanguard.com/news/photopharmics-initiates-largest-phototherapy-trial-for-parkinsons-disease/)
**Published:** March 27, 2024
**Author:** Jon Napitupulu
**Content:**
PhotoPharmics, in partnership with the Center for Health + Technology (CHeT) at the University of Rochester Medical Center (URMC), has initiated a groundbreaking pivotal trial named the “Celeste Light for PD Trial.” This significant research effort aims to evaluate the efficacy of the Celeste® phototherapy device in improving both non-motor and motor functions and the quality of life in patients with [Parkinson](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-genetics-are-global-your-enrollment-plan-isnt/)’s Disease (PD). Leveraging the company’s innovative Spectramax™ technology, the Celeste device offers a passive and non-invasive treatment option that has previously shown promising results in enhancing the lives of PD patients.
The trial, marked by the FDA’s Breakthrough Device Designation for Celeste® received in April 2020, underscores its potential as a transformative treatment modality for PD. Following challenges such as funding issues and delays attributable to the global pandemic, the launch of this 300-patient phototherapy trial, the largest of its kind to explore such technology, has been met with enthusiasm. PhotoPharmics CEO Kent Savage and Chief Science Officer Dan Adams highlighted the device’s remarkable impacts observed in earlier trials on improving patients’ quality of life and addressing non-motor symptoms, paving a new path in PD care.
Celeste® aims to mitigate PD symptoms by targeting photoreceptors in the eye connected to circadian signaling, affecting various aspects from tremors and sleep to cognition and depression. This novel approach underlines the device’s uniqueness in the current treatment landscape for PD.
The trial will be conducted remotely over six months, allowing participants to use the Celeste device daily from the comfort of their homes during their evening routines without altering their existing medical treatments. This telemedicine-based format, led by Dr. Ray Dorsey, Professor of Neurology at URMC and the trial’s lead investigator, ensures greater accessibility and participation across diverse demographics, including remote areas.
Notably, the trial will also focus on addressing severe PD symptoms like sleep disturbances, fatigue, depression, anxiety, and cognitive issues—areas currently lacking FDA-approved treatments. These symptoms often profoundly impact patients’ lives, making the Celeste trial all the more critical in exploring effective interventions for improving the overall quality of life for individuals living with PD.
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**Categories:** News
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### [SecondWave Systems Secures $3M Additional Financing to Advance Ultrasound-Based Anti-Inflammatory Therapy](https://www.clinicaltrialvanguard.com/news/secondwave-systems-secures-3m-additional-financing-to-advance-ultrasound-based-anti-inflammatory-therapy/)
**Published:** March 28, 2024
**Author:** Jon Napitupulu
**Content:**
SecondWave Systems, Inc., an innovative company at the forefront of bio-ultrasonic medicine development, is making significant strides in creating new therapeutic options for [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (RA) and other immune-mediated inflammatory conditions. With the appointment of Professor Paul Peter Tak as the first member of its advisory board, SecondWave gains a wealth of experience from a renowned academic physician-scientist and industry leader. Dr. Tak’s groundbreaking work in bioelectronic medicine and autoimmune diseases lends valuable insight into SecondWave’s mission to develop a non-invasive, ultrasound-based treatment modality.
The company has announced securing an additional $3 million in funding from the Advanced Research Projects Agency for Health (ARPA-H) in partnership with the Defense Advanced Research Projects Agency ([DARPA](https://www.clinicaltrialvanguard.com/executiveinterviews/fda-nih-darpa-government-agencies-lead-new-dct-initiatives/)), bringing its total non-dilutive U.S. government funding to $15.3 million. This support has been instrumental in the progress of SecondWave’s MINI™ technology platform, a pioneering wearable therapeutic ultrasound stimulation device designed for the personalized treatment of chronic and acute inflammatory disorders.
The second round of funding will facilitate the advancement of the MINI technology for a pivotal clinical trial aimed at at-home treatment of RA. Dr. Tak highlighted the ongoing need for innovative treatments that engage the body’s natural anti-inflammatory mechanisms, such as the cholinergic anti-inflammatory pathway, which SecondWave’s MINI system targets through noninvasive methods.
As SecondWave continues its journey to commercialize the MINI as a novel therapy for inflammatory diseases, the company emphasizes the importance of collaboration with esteemed scientists, clinicians, and government agencies. CEO Anuj Bhardwaj expressed enthusiasm for entering the next development phase with Dr. Tak’s expertise and the sustained backing of DARPA and ARPA-H. SecondWave remains committed to producing comprehensive scientific data to support the efficacy of its distinct therapeutic approach, aiming to meet the significant demand for advanced treatments for chronic inflammatory conditions.
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**Categories:** News
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### [AN2 Therapeutics Resumes Phase 2/3 Trial for EBO-301](https://www.clinicaltrialvanguard.com/news/an2-therapeutics-resumes-phase-2-3-trial-for-ebo-301/)
**Published:** March 29, 2024
**Author:** Jon Napitupulu
**Content:**
AN2 Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to creating treatments for rare, chronic, and significant infectious diseases, announced its financial results for the fourth quarter and the full year ending December 31, 2023. The company highlighted its sturdy financial position, with cash, cash equivalents, and investments totaling $134.5 million as of the end of 2023.
Focusing on advancing its boron-based therapy pipeline, AN2 Therapeutics continues to see promising potential in epetraborole for the treatment of nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium Complex (TR-MAC). Despite a temporary pause in Phase 3 enrollment due to an ongoing analysis, the company is proceeding with dosing for patients already enrolled in its pivotal Phase 2/3 trial, known as [EBO-301](https://www.clinicaltrialvanguard.com/news/an2-therapeutics-fails-phase-2-lung-trial/). This halt follows a reassessment of blinded aggregate data from the Phase 2 portion of the trial that indicated efficacy levels potentially lower than anticipated.
Epetraborole’s double-blind, placebo-controlled trial is designed to evaluate its efficacy in conjunction with a background regimen against a placebo plus background regimen in patients with TR-MAC lung disease. With Phase 2 patients having reached the six-month mark, AN2 Therapeutics is set to report topline results in the summer of 2024. Considering the baseline characteristics of the patient demographics, which show a group with complex comorbidities and a history of refractory responses to current treatments, the company aims to engage in discussions with the FDA regarding the trial’s future.
Eric Easom, Co-Founder, President, and CEO of AN2 Therapeutics, expressed optimism regarding the ongoing development of their innovative pipeline and the potential of epetraborole as a crucial therapy for NTM lung disease. The company is also excited about other promising boron therapy candidates in their pipeline, including a novel therapy for chronic Chagas disease in late preclinical development and an IV formulation of epetraborole for melioidosis.
Besides epetraborole, AN2 Therapeutics is expanding its portfolio with various research programs and a recent licensing agreement to develop a therapy for chronic Chagas disease, exhibiting their commitment to addressing unmet patient needs across a range of infectious diseases.
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**Categories:** News
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### [BMS's KRYSAL-12 Trial Shows Promise for Krazati in KRASG12C-Mutated NSCLC](https://www.clinicaltrialvanguard.com/news/bmss-krysal-12-trial-shows-promise-for-krazati-in-krasg12c-mutated-nsclc/)
**Published:** March 29, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb has announced significant outcomes from its Phase 3 [KRYSTAL](https://www.clinicaltrialvanguard.com/opinion/what-the-krystal-12-lancet-correspondence-actually-says-about-pro-reporting-in-krasg12c-trials/)-12 study, evaluating KRAZATI® (adagrasib) in patients with pretreated, locally advanced or metastatic [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) with a KRASG12C mutation. This pivotal trial met its primary endpoint of progression-free survival (PFS) and the key secondary endpoint of overall response rate (ORR), marking a pivotal advancement in targeted cancer therapy.
The KRYSTAL-12 trial’s success strengthens the position of KRAZATI as a monotherapy offering considerable benefits in PFS and ORR compared to standard-of-care chemotherapy, specifically for patients undergoing their second-line or later treatment. This is especially significant for those with the KRASG12C mutation in NSCLC, a demographic previously underserved by targeted cancer therapies. KRAZATI was well tolerated among patients, with safety data aligning with its known safety profile and no new safety signals observed.
The FDA’s accelerated approval of KRAZATI in December 2022 represented a groundbreaking shift in treatment paradigms for patients with KRASG12C-mutated NSCLC. This was further reinforced internationally with the Medicines and Healthcare products Regulatory Agency (MHRA) and the European Commission (EC) granting conditional marketing authorization in 2023 and 2024, respectively.
Beyond NSCLC, KRAZATI and KRAZATI-based combination therapies have demonstrated promising outcomes in Phase 2 clinical trials for various solid tumors, including advanced colorectal cancer, pancreatic cancer, and other malignancies. Particularly noteworthy is the supplemental new drug application (sNDA) for KRAZATI in combination with cetuximab for colorectal cancer patients with the KRASG12C mutation, which the U.S. FDA accepted for priority review with a Prescription Drug User Fee Act (PDUFA) goal date set for June 21, 2024.
Bristol Myers Squibb has expressed gratitude to patients and investigators involved in the KRYSTAL-12 trial and anticipates sharing detailed results with the scientific community at an upcoming medical conference. This exciting development underscores the company’s commitment to pioneering targeted therapies that offer hope to patients facing advanced and metastatic cancers.
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**Categories:** News
---
### [European Commission Approves Keytruda® Plus Chemotherapy for Resectable NSCLC](https://www.clinicaltrialvanguard.com/news/european-commission-approves-keytruda-plus-chemotherapy-for-resectable-nsclc/)
**Published:** March 29, 2024
**Author:** Jon Napitupulu
**Content:**
Merck announced that the European Commission (EC) has approved [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/), its anti-PD-1 therapy, for use in combination with platinum-containing chemotherapy as a neoadjuvant treatment, followed by KEYTRUDA as monotherapy in the adjuvant setting, for adult patients with [resectable non-small cell lung](https://www.clinicaltrialvanguard.com/news/u-s-fda-approves-opdivo-for-resectable-non-small-cell-lung-cancer/) cancer (NSCLC) at high risk of recurrence. This significant development marks the first approval in Europe for an anti-PD-1/L1 therapy in resectable NSCLC based on positive overall survival results and adds to KEYTRUDA’s portfolio as its sixth approval for lung cancer treatments in Europe.
The EC’s approval is grounded in the outcomes from the Phase 3 [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-671 trial, which at a median follow-up of 29.8 months, showed that KEYTRUDA plus chemotherapy in the neoadjuvant phase, followed by KEYTRUDA monotherapy post-surgical resection, significantly improved overall survival, reducing the risk of death by 28% compared to the placebo plus chemotherapy regimen. The treatment also notably improved event-free survival (EFS), reducing the risk of disease recurrence, progression, or death by 41% compared with the control group.
Dr. Solange Peters, a leading expert in medical oncology from Centre Hospitalier Universitaire Vaudois in Lausanne, Switzerland, highlighted the importance of this approval for patients with resectable NSCLC at high risk of recurrence. She stressed the need for early-stage treatment where significant impact can be made, underscoring the potential life-extending benefits demonstrated by the KEYTRUDA-based regimen in the KEYNOTE-671 trial.
The authorization allows the marketing of this KEYTRUDA regimen across all 27 EU member states, as well as in Iceland, Liechtenstein, Norway, and Northern Ireland, adding to KEYTRUDA’s growing list of indications in the EU, now totaling 27. This follows KEYTRUDA’s U.S. approval in October 2023 for the treatment of patients with resectable NSCLC in combination with platinum-containing chemotherapy as neoadjuvant treatment and continued as a single agent post-surgery.
This latest approval represents a significant advancement in the treatment of NSCLC in Europe, providing patients with a high risk of recurrence an innovative therapeutic option that has demonstrated the potential for extending lives through the KEYNOTE-671 trial’s results.
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**Categories:** News
---
### [Gilead and Xilio Enter Exclusive License Agreement for Tumor-Activated IL-12 Program](https://www.clinicaltrialvanguard.com/news/gilead-and-xilio-enter-exclusive-license-agreement-for-tumor-activated-il-12-program/)
**Published:** March 29, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences, Inc. and Xilio Therapeutics, Inc. have entered into an exclusive license agreement to develop and commercialize XTX301, Xilio’s Phase 1 tumor-activated [IL-12](https://www.clinicaltrialvanguard.com/news/son-1010-shows-strong-safety-in-ovarian-cancer-treatment/) program. This partnership leverages Xilio’s innovative technology to develop immuno-oncology therapies specifically activated within the tumor microenvironment, potentially mitigating systemic toxicities and enhancing the efficacy of treatments for a broad range of cancers.
XTX301, currently undergoing a Phase 1 trial in patients with advanced solid tumors, presents a significant step forward in immuno-oncology. Bill Grossman, MD, PhD, of Gilead Sciences, emphasized the alignment of Xilio’s tumor-activation platform with Gilead’s ongoing clinical efforts in addressing challenging cancers, highlighting the prospective broad-spectrum efficacy of IL-12 in solid tumors.
René Russo, Pharm.D., President and CEO of Xilio, expressed enthusiasm for collaborating with Gilead, underscoring the combined strength of Gilead’s expertise in novel immuno-oncology product development and Xilio’s innovative technology. This partnership aims to overcome the historical challenges associated with IL-12 use, such as severe toxicities, while potentially providing substantial benefits across various tumor types, including those considered immunologically cold.
Under the agreement, Xilio will receive $43.5 million in upfront payments, with the potential for up to $604 million in additional payments based on development, regulatory, and sales milestones, as well as tiered royalties on global net product sales. The collaboration also includes a $75 million transition fee, allowing Gilead to assume responsibilities for XTX301’s further development and commercialization upon delivering a specified clinical data package from Xilio.
This collaboration marks a notable advancement in immuno-oncology, promising to deliver innovative therapeutic options for patients battling a wide range of solid tumors.
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**Categories:** News
---
### [BMS Updates on Phase 3 Yellowstone Trial for Zeposia in Crohn's Disease](https://www.clinicaltrialvanguard.com/news/bms-updates-on-phase-3-yellowstone-trial-for-zeposia-in-crohns-disease/)
**Published:** March 29, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMY) has reported that a Phase 3 study within the YELLOWSTONE clinical trial program, which was evaluating the efficacy of Zeposia (ozanimod) in adult patients with moderate to severe active [Crohn’s disease](https://www.clinicaltrialvanguard.com/news/eli-lillys-omvoh-gets-fda-approval-for-crohns-disease-but-faces-challenges/)[Crohn’s](https://www.clinicaltrialvanguard.com/news/agomabs-crohns-drug-shows-promising-phase-2a-results/)[Crohn](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/)‘s disease, did not achieve its primary endpoint of clinical remission at Week 12. Despite this outcome, Zeposia’s safety profile remained consistent with the results documented in previous trials.
The YELLOWSTONE clinical trial program is extensive, encompassing two 12-week induction studies, a 52-week maintenance study, and a 264-week open-label extension study, designed to assess the safety and efficacy of Zeposia versus placebo in treating Crohn’s disease. Participants from the induction studies who responded to treatment were qualified to advance to the maintenance study, while non-responders, those experiencing disease relapse during maintenance, and those completing the maintenance phase were given the option to enter the open-label extension trial. All patients in this comprehensive program were administered oral doses of Zeposia 0.92 mg (equivalent to 1 mg).
Roland Chen, MD, Senior Vice President and Head of Immunology, Cardiovascular, and Neuroscience development at Bristol Myers Squibb, articulated disappointment in not meeting the primary endpoint in the first induction trial. However, he reaffirmed the company’s commitment to advancing science for patients with immune-mediated diseases and expressed gratitude to participating investigators and patients. He highlighted the unmet medical need for new therapies in Crohn’s disease, emphasizing the significance of finding effective treatments that offer symptomatic relief and potential remission.
Crohn’s disease, a chronic inflammatory bowel disease (IBD), affects the digestive tract and may result in swelling or inflammation of the intestines. This condition can severely impair patients’ quality of life, manifesting in symptoms that range from mild to severe and sometimes developing suddenly. With an estimated 12.6 million individuals affected worldwide, the disease may affect any part of the intestinal tract but most commonly occurs in the colon or the ileum.
Individuals living with Crohn’s disease frequently experience episodes of symptom-free remission, which can be interrupted by relapses or disease flares. Additionally, patients with this condition face an increased risk of developing colorectal cancer, further underscoring the critical need for effective treatments.
The forthcoming full evaluation of the YELLOWSTONE trial data and planned discussions with health authorities signal Bristol Myers Squibb’s ongoing efforts to address the challenges associated with Crohn’s disease through innovative therapeutic options. The scientific community eagerly awaits the detailed results, which will be shared at a future medical conference.
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**Categories:** News
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### [Pyros Pharmaceuticals Launches Vigpoder™ (Vigabatrin) for Market](https://www.clinicaltrialvanguard.com/news/pyros-pharmaceuticals-launches-vigpoder-vigabatrin-for-market/)
**Published:** April 2, 2024
**Author:** Jon Napitupulu
**Content:**
[Pyros Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/pyros-pharmaceuticals-fda-approved-vigafyde-the-only-ready-to-use-vigabatrin-oral-solution/), Inc., a leading company in the development of specialty pharmaceuticals for rare diseases, has announced the availability of VIGPODER™ (vigabatrin) for oral solution, USP, for the treatment of appropriate patients with infantile spasms (IS). VIGPODER™ provides a therapeutically equivalent, affordable treatment option with a safety and efficacy profile anticipated to be the same as SABRIL® (vigabatrin) for oral solution.
Infantile spasms represent a significant challenge for patients and their families due to the rarity and severity of this form of [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/). Through the introduction of VIGPODER™, Pyros aims to ensure that families confronted with an IS diagnosis have rapid access to this essential therapy. The company is further committed to supporting these families through its Pyros Total Care™ program, offering personalized comprehensive support and assistance throughout their treatment journey.
Pyros Total Care™ offers personalized assistance and financial resources, including support from a dedicated nurse educator, reimbursement support, and clinical pharmacist advice. It underscores Pyros’ commitment to facilitating treatment access and providing comprehensive support to caregivers navigating the challenges of infantile spasms.
Infantile spasms (IS) is a rare, severe form of epilepsy primarily affecting children under one year old. The condition can manifest as subtle, repetitive movements that may be easily overlooked or misdiagnosed. Without appropriate treatment, IS can lead to continued seizures, other forms of epilepsy, autism spectrum disorder, and developmental issues. The American Academy of Neurology emphasizes that successful treatment of IS can significantly improve the long-term prognosis for affected children.
Vigabatrin, the active ingredient in VIGPODER™, is a medication designed to inhibit GABA transaminase, increasing gamma-aminobutyric acid (GABA) levels in the brain. This mechanism is thought to contribute to seizure control by modulating neuronal excitability, underscoring the role of vigabatrin in managing seizure disorders, including infantile spasms.
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**Categories:** News
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### [Zai Lab's Partner BMS Confirms KRYSAL-12 Trial Success for Krazati in KRASG12C-Mutated Cancer](https://www.clinicaltrialvanguard.com/news/zai-labs-partner-bms-confirms-krysal-12-trial-success-for-krazati-in-krasg12c-mutated-cancer/)
**Published:** April 2, 2024
**Author:** Jon Napitupulu
**Content:**
Zai Lab Limited, in collaboration with Bristol Myers Squibb, announced significant progress in the fight against lung cancer with the successful outcome of the [KRYSTAL](https://www.clinicaltrialvanguard.com/opinion/what-the-krystal-12-lancet-correspondence-actually-says-about-pro-reporting-in-krasg12c-trials/)-12 study for KRAZATI® (adagrasib), specifically targeting NSCLC with KRASG12C mutations. The Phase 3 study achieved its primary endpoint, progression-free survival (PFS), and a key secondary endpoint, overall response rate (ORR), highlighting the therapy’s potential as a significant advancement for patients with locally advanced or metastatic NSCLC who have received prior treatments. The results reflect a meaningful benefit in PFS and ORR compared to standard-of-care chemotherapy for second-line or later treatment, without new safety concerns.
Rafael G. Amado, M.D., President and Head of Global Oncology Research and Development at Zai Lab, expressed enthusiasm about adagrasib’s potential and its particularly critical role in addressing China’s lung cancer burden, the most common cancer in the country. The company plans to submit an NDA for adagrasib in China for second or later line treatment of KRASG12C mutated NSCLC within the year, aiming to expand treatment options for patients.
The KRAZATI and related combinations have also shown promising results in Phase 2 clinical trials for additional tumors like advanced colorectal and pancreatic cancer. The U.S. FDA’s priority review of KRAZATI in combination with [cetuximab](https://www.clinicaltrialvanguard.com/news/frontier-medicines-presents-preclinical-data-on-3-programs/) for metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (CRC) emphasizes the broad potential of this therapy.
With lung cancer resulting in significant morbidity and mortality in China, advancements like KRAZATI represent crucial steps forward in oncologic care, offering new hope to patients and potentially shaping future treatment landscapes.
Source link: [http://www.businesswire.com/news/home/20240401347225/en/Zai-Lab-Partner-Bristol-Myers-Squibb-Announces-Pivotal-KRYSTAL-12-Confirmatory-Trial-Evaluating-KRAZATI-adagrasib-Meets-Primary-Endpoint-of-Progression-Free-Survival-for-Patients-with-Pretreated-KRASG12C-Mutated-Locally-Advanced-or-Metastatic…](http://www.businesswire.com/news/home/20240401347225/en/Zai-Lab-Partner-Bristol-Myers-Squibb-Announces-Pivotal-KRYSTAL-12-Confirmatory-Trial-Evaluating-KRAZATI-adagrasib-Meets-Primary-Endpoint-of-Progression-Free-Survival-for-Patients-with-Pretreated-KRASG12C-Mutated-Locally-Advanced-or-Metastatic...)
**Categories:** News
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### [MRD Testing Market Analysis 2024: Revolution in Cancer Management Drives Growth](https://www.clinicaltrialvanguard.com/news/mrd-testing-market-analysis-2024-revolution-in-cancer-management-drives-growth/)
**Published:** April 2, 2024
**Author:** Jon Napitupulu
**Content:**
The MRD (Minimal Residual Disease) testing market is experiencing substantial growth, with its value projected to increase from $1.67 billion in 2023 to $6.67 billion by 2033, at a Compound Annual Growth Rate (CAGR) of 14.81%. This surge is attributable to several factors, including expanded Medicare coverage for MRD testing and its utilization in diagnosing solid tumors. As consumer awareness about the effectiveness of MRD testing in cancer management rises, healthcare providers and researchers are increasingly incorporating MRD testing into clinical practices to meet the growing demand.
MRD testing, pivotal in cancer care, employs advanced technologies like PCR (Polymerase Chain Reaction), NGS (Next-Generation Sequencing), and flow cytometry to detect and quantify residual cancer cells post-treatment. Its critical role extends beyond monitoring treatment response to encompassing prognostic assessment and aiding in the personalization of treatment strategies. This fosters significant opportunities across the healthcare sector, enabling diagnostic companies, pharmaceutical manufacturers, and research institutions to pioneer innovative solutions that optimize patient care.
MRD tests elevate the standard of cancer treatment by identifying minimal levels of cancer cells that traditional diagnostic methods might overlook, enabling timely and tailored intervention strategies. This facilitates precision medicine by allowing clinicians to tailor treatment regimens based on residual disease levels, thereby enhancing patient outcomes and contributing to longer survival rates.
The rise in global cancer rates has also propelled significant advancements in MRD testing technology to surmount the diagnostic challenges cancer presents. The pursuit for increased sensitivity, accuracy, and efficiency in MRD testing drives continuous innovation in the sector.
Recent collaborations, such as that between Providence and [GRAIL](https://www.clinicaltrialvanguard.com/news/grails-galleri-trial-misses-primary-endpoint-but-shows-stage-iv-cancer-reduction/) in March 2023 to expand the reach of the Galleri multi-cancer early detection screening, underscore the emerging emphasis on broadening MRD testing’s accessibility. Likewise, the acquisition of ArcherDX by Integrated DNA Technologies (IDT) in December 2022 represents another stride towards enhancing the capability and reach of MRD testing methods.
These developments signal a transformative era in cancer care, emphasizing the importance of sensitive, accurate testing and the personalized approach to treatment that MRD testing facilitates. The vast potential of the MRD testing market underscores the critical role it plays not only in improving clinical outcomes but also in driving forward the fields of oncology research and diagnostic technology.
Source link: [http://www.businesswire.com/news/home/20240401800940/en/Global-and-Regional-MRD-Testing-Market-Analysis-Report-2024-Revolutionizing-Cancer-Patient-Management-Rising-Consumer-Awareness-Spurs-Demand-for-MRD-Testing—Forecast-to-2033—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240401800940/en/Global-and-Regional-MRD-Testing-Market-Analysis-Report-2024-Revolutionizing-Cancer-Patient-Management-Rising-Consumer-Awareness-Spurs-Demand-for-MRD-Testing---Forecast-to-2033---ResearchAndMarkets.com)
**Categories:** News
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### [Mirum's Livmarli Gets Positive CADTH Reimbursement Nod for Alagille Syndrome in Canada](https://www.clinicaltrialvanguard.com/news/mirums-livmarli-gets-positive-cadth-reimbursement-nod-for-alagille-syndrome-in-canada/)
**Published:** April 3, 2024
**Author:** Jon Napitupulu
**Content:**
The Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued a positive recommendation for the public reimbursement of LIVMARLI® (maralixibat oral solution) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS). This follows [Health Canada](https://www.clinicaltrialvanguard.com/news/amo-pharma-aligns-with-fda-mhra-health-canada-on-amo-02-study-design-for-cdm1/)‘s authorization of LIVMARLI for this indication in 2023, marking an essential milestone for patients suffering from this rare liver disorder. LIVMARLI stands as the first and only approved medication in Canada for this condition, offering hope to those affected, many of whom are children, by alleviating the debilitating effects of cholestatic pruritus associated with ALGS.
This CADTH recommendation is grounded on data from the pivotal ICONIC study and six years of data across the LIVMARLI clinical program. The study showcased statistically significant and clinically meaningful reductions in pruritus and serum bile acids, with benefits sustained over several years of treatment. This recommendation not only emphasizes the treatment’s clinical efficacy but also its potential to significantly improve patients’ quality of life.
The approval of LIVMARLI in Canada, coupled with existing approvals in the U.S. and Europe for ALGS and Progressive Familial Intrahepatic Cholestasis (PFIC), reinforces Mirum Pharmaceuticals’ commitment to addressing the needs of patients with rare liver diseases. ALGS, characterized by bile duct abnormalities leading to progressive liver disease, has long been a challenging condition with limited treatment options. The disorder’s hallmark, cholestasis, results in bile acid accumulation, causing severe pruritus and contributing to liver injury. This condition frequently necessitates liver transplantation, highlighting the critical need for effective treatments like LIVMARLI.
The CADTH’s move to recommend LIVMARLI for reimbursement is a step forward in making this vital treatment more accessible to Canadian patients, potentially alleviating the physical and emotional burden of ALGS and offering a new beacon of hope for affected families.
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**Categories:** News
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### [QLS-111 by Qlaris Bio Enters Phase II Trials for IOP Reduction](https://www.clinicaltrialvanguard.com/news/qls-111-by-qlaris-bio-enters-phase-ii-trials-for-iop-reduction/)
**Published:** April 3, 2024
**Author:** Jon Napitupulu
**Content:**
Qlaris Bio, Inc., a biotechnology firm focusing on ophthalmic diseases, has announced the initiation of two Phase II clinical trials in the U.S. to investigate QLS‑111, a pioneering treatment for patients with ocular hypertension and [glaucoma](https://www.clinicaltrialvanguard.com/news/nurexone-shows-vision-recovery-in-preclinical-glaucoma-model/). This first-in-class product targets episcleral venous pressure (EVP) to achieve intraocular pressure (IOP) reductions beyond current possibilities. The commencement of these trials represents a significant step towards addressing the unmet needs of glaucoma patients requiring consistent IOP control.
QLS‑111 is distinguished by its unique mechanism that reduces IOP by targeting EVP and distal outflow resistance, offering potential benefits for patients with primary open-angle glaucoma (POAG), ocular hypertension (OHT), and normal tension glaucoma (NTG). Unlike existing medications that either decrease aqueous humor production or enhance proximal outflow, QLS‑111 directly impacts distal outflow and EVP, a critical factor constituting up to 50% of total IOP.
The promise of QLS‑111 is highlighted by its capability to lower IOP significantly from baseline in healthy, normotensive volunteers, as well as its compatibility with existing glaucoma treatments. This suggests that QLS‑111 could offer a synergistic advantage in achieving IOP reductions necessary for slowing disease progression. Given its favorable safety profile and lack of clinically meaningful hyperemia, QLS‑111 shows immense potential as a flexible addition to current glaucoma therapy regimens.
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**Categories:** News
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### [US FDA Accepts Astrazeneca License Application for Datopotamab Deruxtecan in Metastatic HR+, HER2- Breast Cancer](https://www.clinicaltrialvanguard.com/news/us-fda-accepts-astrazeneca-license-application-for-datopotamab-deruxtecan-in-metastatic-hr-her2-breast-cancer/)
**Published:** April 3, 2024
**Author:** Jon Napitupulu
**Content:**
Biopharmaceutical companies AstraZeneca and Daiichi Sankyo’s [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) for [datopotamab](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/) deruxtecan has been accepted in the US, paving the way for this potential treatment for adult patients with inoperable or metastatic hormone receptor (HR)-positive, HER2-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) who have received previous systemic therapy. Datopotamab deruxtecan is a TROP2-directed DXd antibody-drug conjugate engineered by Daiichi Sankyo, and is jointly being developed by Daiichi Sankyo and AstraZeneca. The FDA is set to decide on the application by the first quarter of 2025.
This BLA is based on results from the TROPION-Breast01 Phase III trial, which demonstrated a significant improvement in progression-free survival compared to chemotherapy in patients with inoperable or metastatic HR-positive, HER2-negative breast cancer previously treated with endocrine-based therapy and a systemic therapy. The safety profile was consistent with other ongoing trials, with no new safety concerns identified.
According to Susan Galbraith, Executive Vice President, Oncology R&D at AstraZeneca, if approved, datopotamab deruxtecan would provide an efficient alternative to conventional chemotherapy for patients suffering from this type of advanced breast cancer.
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**Categories:** News
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### [D&D Pharmatech Receives Fast Track Designation from FDA for DD01 - Novel Treatment for NASH/MASH](https://www.clinicaltrialvanguard.com/news/dd-pharmatech-receives-fast-track-designation-from-fda-for-dd01-novel-treatment-for-nash-mash/)
**Published:** April 3, 2024
**Author:** Jon Napitupulu
**Content:**
D&D Pharmatech, a biotech company, has been granted Fast Track designation by the US Food and Drug Administration (FDA) for its DD01 drug, aimed at treating adults with non-alcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This designation could potentially expedite the process of the drug review to meet the unmet requirements for serious diseases.
The FDA’s decision is based on data from a Phase 1 randomized, double-blind, placebo-controlled study that evaluated the safety and effectiveness of DD01 in overweight subjects with [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) (T2D) and metabolic dysfunction-associated fatty liver disease (MAFLD). DD01 treatment was well tolerated and reduced hepatic steatosis (abnormal retention of lipids within liver cells) by over 50% in only four weeks.
D&D’s CEO, Dr. Seulki Lee, noted how MASH has a significant unmet need and several other clinical trials have proven that efficient reduction of excessive liver fat content can help manage MASH and liver scarring.
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**Categories:** News
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### [EC Approves Bristol Myers' Reblozyl as First-Line Treatment for LR-MDS Anemia](https://www.clinicaltrialvanguard.com/news/ec-approves-bristol-myers-reblozyl-as-first-line-treatment-for-lr-mds-anemia/)
**Published:** April 3, 2024
**Author:** Jon Napitupulu
**Content:**
The European Commission (EC) has expanded approval of Reblozyl (luspatercept), a first-in-class treatment for patients with disease-related anemia, for the first-line treatment of adult patients with anemia due to very low to intermediate risk myelodysplastic syndromes (MDS). The decision is based on the Phase 3 COMMANDS study which demonstrated that Reblozyl almost doubled the percentage of patients achieving transfusion independence and a higher hemoglobin count, showing enhanced efficacy compared to epoetin alfa, a traditional treatment for anemia.
Reblozyl’s approval is a significant event in offering more effective options to adults suffering from lower-risk MDS in the EU. According to Monica Shaw, senior vice president and head of European Markets, Bristol Myers Squibb, the maker of Reblozyl, this drug gives patients the potential to become transfusion independent for a prolonged duration compared to available options. She also remarked that this achievement shows Bristol Myers Squibb’s continuous commitment to innovating new choices for patients with disease-related anemia.
Matteo Giovanni Della Porta, M.D., an investigator and head of the [Leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) Unit at the Humanitas Cancer Center in Milan, Italy, noted that the results from the COMMANDS study underscore Reblozyl’s clinical value as a starter treatment for anemia in patients with low to intermediate-risk MDS. The approval represents a significant milestone for improving treatment practices and ensuring better outcomes for patients, he said.
Reblozyl is also authorized for use for anemia linked with lower-risk MDS in Japan and the United States. The heightened approval for Reblozyl concerns all EU member states, except for Great Britain (England, Scotland, and Wales). It is the fourth licensed indication in Europe for this drug. The approval indicates a significant stride in improving treatment options for patients with anemia due to MDS in the EU countries.
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**Categories:** News
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### [Allurion Bridges Digital Gap, Introduces Virtual Care Suite in US](https://www.clinicaltrialvanguard.com/news/allurion-bridges-digital-gap-introduces-virtual-care-suite-in-us/)
**Published:** April 5, 2024
**Author:** Jon Napitupulu
**Content:**
Allurion Technologies has launched its Virtual Care Suite (VCS) in the United States. It provides a comprehensive weight loss management platform for providers offering GLP-1 medications, bariatric surgery, and other weight loss devices.
The VCS combines remote patient monitoring, telehealth, and care team collaboration in one digital platform, optimizing patient outcomes and satisfaction. Its AI-powered functionality includes Coach Iris, a 24/7 weight loss coach that personalizes behavior change programs.
Studies have shown that VCS usage leads to enhanced patient results, with 11% additional weight loss, 16% more goal achievement, and a 17% increase in Net Promoter Score (NPS). The platform has been successful in over 50 countries and is now available to US providers for the first time at the [Obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) Medicine Conference in Denver on April 25.
Allurion believes the VCS can be integral to weight loss practices, especially with the growing use of GLP-1 medications. The launch includes a new version of Allurion Insights, a provider dashboard for VCS navigation.
The VCS is also available to providers separately from Allurion’s Gastric Balloon Program. It allows customization and management of weight loss therapy for patients regardless of treatment plan. This comprehensive platform empowers providers with digital solutions to enhance clinical outcomes and improve patient experiences in the fight against obesity.
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**Categories:** News
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### [FDA Grants 510(k) Clearance to Angiodynamics' AlphaVac F1885 System for PE Treatment](https://www.clinicaltrialvanguard.com/news/fda-grants-510k-clearance-to-angiodynamics-alphavac-f1885-system-for-pe-treatment/)
**Published:** April 5, 2024
**Author:** Jon Napitupulu
**Content:**
[AngioDynamics](https://www.clinicaltrialvanguard.com/news/angiodynamics-initiates-landmark-trial-for-pulmonary-embolism-treatment/) has received FDA clearance for the [AlphaVac](https://www.clinicaltrialvanguard.com/news/unlock-the-extraordinary-angiodynamics-alphavac-f18%e2%81%b8%e2%81%b5-revolutionizes-vascular-health/) F1885 System to treat pulmonary embolism (PE), a life-threatening condition. PE affects a significant portion of the population and is a leading cause of cardiovascular deaths.
Expanding the AlphaVac F1885 System’s indication enhances its use in critical medical settings. The system offers a non-surgical option for removing blood clots from veins, reducing the patient’s thrombus burden, and improving right ventricular function.
The APEX-AV study’s results supported the FDA approval, which demonstrated the device’s effectiveness and safety in treating PE. The study, conducted in partnership with the Pulmonary Embolism Response Team (PERT) Consortium, enrolled 122 patients and showed a significant reduction in clot burden and improved patient outcomes.
Adding the AlphaVac System to the mechanical thrombectomy landscape significantly advances PE treatment. Its user-friendly design, proven efficacy, and favorable safety profile provide physicians with valuable tools for improving patient care.
Juan Carlos Serna, Senior Vice President of Scientific and Clinical Affairs at AngioDynamics, emphasized the company’s commitment to patient-centered solutions and merging clinician expertise with outcome-driven therapies. Dr. William Brent Keeling, co-principal Investigator of the APEX-AV study, highlighted the critical role of the AlphaVac System in the treatment of PE patients.
This FDA clearance marks a milestone in the fight against PE, providing a safe and effective option for improving patient outcomes and reducing mortality associated with this condition.
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**Categories:** News
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### [Merck Launches Phase 3 Trial with Combo of MK-1084 & KeytrudaⓇ for NSCLC Treatment](https://www.clinicaltrialvanguard.com/news/merck-launches-phase-3-trial-with-combo-of-mk-1084-keytrudaⓡ-for-nsclc-treatment/)
**Published:** April 5, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has initiated a Phase 3 clinical trial (NCT06345729) to evaluate MK-1084, an oral KRAS G12C inhibitor, in combination with [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) for the first-line treatment of metastatic [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) with KRAS G12C mutations and PD-L1 expression (TPS ≥50%).
MK-1084 is a potent and specific KRAS G12C covalent inhibitor being developed through a collaboration between Merck, Taiho Pharmaceutical Co. Ltd, and Astex Pharmaceuticals. KRAS mutations are common in various cancers, including NSCLC, where the KRAS G12C mutation is particularly prevalent.
The Phase 3 trial will enroll approximately 600 patients globally and compare MK-1084 plus KEYTRUDA to KEYTRUDA plus placebo. The primary endpoints are progression-free survival and overall survival, while secondary endpoints include objective response rate and duration of response.
Preliminary data from an earlier Phase 1 trial showed a manageable safety profile and promising anti-tumor activity for the MK-1084 and KEYTRUDA combination. The current Phase 3 trial aims to further evaluate the efficacy and safety of this combination in a larger patient population.
Lung cancer, particularly NSCLC, remains a leading cause of cancer-related deaths globally. This Phase 3 trial represents a significant step towards developing new treatment options for patients with KRAS G12C-mutated metastatic NSCLC.
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**Categories:** News
---
### [Bristol Myers Squibb & 2Seventy Abecma Expands Approval for Multiple Myeloma Treatment](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibb-2seventy-abecma-expands-approval-for-multiple-myeloma-treatment/)
**Published:** April 8, 2024
**Author:** Jon Napitupulu
**Content:**
Abecma, a personalized CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), has received expanded approval from the FDA for the treatment of adult patients with relapsed or refractory multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/). This new indication allows for earlier use of Abecma after two or more prior lines of therapy, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an anti-CD38 monoclonal antibody.
In the Phase 3 KarMMa-3 trial, Abecma tripled progression-free survival compared to standard regimens. Patients receiving Abecma experienced a 51% reduction in the risk of disease progression or death. These results demonstrate the significant clinical benefit of Abecma in this patient population.
Abecma is administered as a one-time infusion, with a recommended dose range of 300 to 510 x 106 CAR-positive T cells. It is designed to target and eliminate multiple myeloma cells more effectively than traditional therapies.
Abecma is now approved in the U.S., Japan, Switzerland, and the EU for the treatment of relapsed or refractory multiple myeloma in earlier lines of therapy. Bristol Myers Squibb and 2seventy bio are committed to expanding access to Abecma globally.
This expanded approval provides a valuable new treatment option for patients with relapsed or refractory multiple myeloma. Abecma offers the potential for longer periods of remission and reduced disease progression, offering hope for improved outcomes in this challenging disease.
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**Categories:** News
---
### [AltPep Study in Nature Journal Affirms Potential of Blood Test for Alzheimer's Detection](https://www.clinicaltrialvanguard.com/news/altpep-study-in-nature-journal-affirms-potential-of-blood-test-for-alzheimers-detection/)
**Published:** April 8, 2024
**Author:** Jon Napitupulu
**Content:**
AltPep Corporation’s SOBA-AD blood test has demonstrated promising accuracy in detecting [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease (AD) in two independent studies. Published in the peer-reviewed journal Scientific Reports, the study evaluated 265 blinded plasma samples, achieving 100% sensitivity, >95% specificity, and >98% area under the curve in distinguishing AD patients from cognitively unimpaired controls.
The SOBA-AD test targets toxic soluble oligomers, early molecular triggers of amyloid diseases, aiming to identify AD patients years before symptoms manifest. This study builds on earlier research that analyzed 644 samples, consistently exhibiting high performance metrics.
The test’s unique feature is its ability to rationally target alpha-sheet protein structures found in toxic oligomers, distinguishing it from other technologies. The successful implementation at multiple locations highlights its potential as a biomarker for AD clinical trials and an earlier diagnostic tool to enable timely treatment.
In addition, the study included a more diverse sample population, supporting the potential for early detection in preclinical stages. Five CU controls tested positive, suggesting possible pre-symptomatic disease, although confirmatory data is pending.
Overall, the results support the SOBA-AD blood test as a promising tool for selective detection and confirmation of AD, potentially transforming the diagnosis and management of this devastating disease.
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**Categories:** News
---
### [Denovo Biopharma's Revelation: Precision Treatment for Depression's Grip](https://www.clinicaltrialvanguard.com/news/denovo-biopharmas-revelation-precision-treatment-for-depressions-grip/)
**Published:** April 10, 2024
**Author:** Jon Napitupulu
**Content:**
Denovo Biopharma’s Phase 2b ENLIGHTEN trial has demonstrated the efficacy and safety of liafensine (DB104) in treating treatment-resistant depression (TRD). Using a novel genetic biomarker, DGM4™, researchers identified patients most likely to respond to liafensine.
Over 23 million Americans suffer from [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD), and 30% have TRD, which is notoriously challenging to treat. With few approved medications and poor outcomes, TRD remains a significant medical need.
Liafensine is a triple reuptake inhibitor targeting serotonin, norepinephrine, and dopamine. Denovo’s DGM™ platform identified DGM4™ as a predictor of liafensine’s efficacy in TRD patients.
ENLIGHTEN enrolled 197 patients with TRD, using DGM4™ to guide randomization. DGM4-positive patients treated with liafensine showed significant improvement over placebo after 6 weeks. The primary endpoint, change in Montgomery-Åsberg Depression Rating Scale (MADRS) score, demonstrated a 4.4-point improvement with liafensine (p = 0.0056).
Secondary endpoints were also met, with liafensine showing approximately 36% improvement in Clinical Global Impressions Scale-Severity (CGI-S) and Sheehan Disability Scale (SDS) scores. Additionally, the Clinical Global Impressions Scale-Improvement (CGI-I) was 2.3 for liafensine, a 0.6-point improvement over placebo (p = 0.0026).
Liafensine was well tolerated with an excellent safety profile consistent with previous trials involving over 2,200 subjects.
Matthew Spear, Chief Medical Officer and Chief Development Officer at Denovo, expressed excitement about the results, highlighting the significant improvement in depression symptoms (over 40%) compared to current drugs. He emphasized that the trial represents a breakthrough in precision medicine for CNS diseases, as it is the first time a genetic biomarker has been used to select TRD patients likely to benefit from liafensine.
“The success of ENLIGHTEN is a milestone for Denovo,” said Wen Luo, Chief Executive Officer and Chief Scientific Officer of Denovo, validating their innovative biomarker approach. “We are actively seeking a global partner to expedite the development of liafensine, aiming to provide this treatment to millions of patients in need.”
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**Categories:** News
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### [Synthekine Trial Shows New Hope for Advanced Tumor Battles](https://www.clinicaltrialvanguard.com/news/synthekine-trial-shows-new-hope-for-advanced-tumor-battles/)
**Published:** April 10, 2024
**Author:** Jon Napitupulu
**Content:**
Synthekine Inc., a leader in engineered cytokine therapeutics, has announced promising initial findings from a Phase 1a/1b trial of their novel drug STK-012, an α/β biased IL-2 partial agonist. STK-012 is designed to stimulate specific T cells associated with tumor-fighting abilities, while avoiding the toxic effects often seen with IL-2 therapies.
In the Phase 1a dose escalation phase, involving 47 patients with advanced solid tumors, STK-012 exhibited a favorable safety profile with no dose-limiting toxicities or capillary leak syndrome. Notably, multiple objective responses were achieved with STK-012 monotherapy, correlating with an increase in interferon-gamma and selectively expanding tumor-specific T cells.
“The limited efficacy and unacceptable toxicity of previous IL-2 analogues have hindered the realization of IL-2’s potential in solid tumors,” said Dr. Naiyer Rizvi, chief medical officer of Synthekine. “The promising results with STK-012, demonstrating both efficacy and avoidance of severe toxicities, are particularly encouraging.”
Following the successful completion of the Phase 1a portion, Synthekine has initiated the Phase 1b dose expansion cohorts to further evaluate STK-012 in specific solid tumor types, including renal cell carcinoma (RCC) and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) (NSCLC).
One case study presented at the AACR conference showcased a Stage IV ccRCC patient who received STK-012 monotherapy. The patient had previously undergone two treatment lines, including immune checkpoint therapy, but achieved a confirmed partial response with STK-012, significantly reducing their tumor size.
These positive initial results demonstrate the potential of STK-012 as a promising treatment option for patients with advanced solid tumors. Ongoing studies will further elucidate its efficacy and safety profile, providing valuable insights into its potential clinical impact.
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**Categories:** News
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### [Obsidian Therapeutics Study Finds Hope in Advanced Melanoma](https://www.clinicaltrialvanguard.com/news/obsidian-therapeutics-study-finds-hope-in-advanced-melanoma/)
**Published:** April 10, 2024
**Author:** Jon Napitupulu
**Content:**
Obsidian Therapeutics, a biotechnology company, unveiled promising updates on its Phase 1 study of OBX-115, a tumor-infiltrating lymphocyte (TIL) [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), at the American Association for Cancer Research (AACR) Annual Meeting.
OBX-115 is being tested in patients with advanced melanoma who have not responded to immune checkpoint inhibitors (ICIs). Six patients received OBX-115 in the study, and the safety data collected over 25 weeks showed it was well-tolerated.
Crucially, OBX-115 demonstrated sustained and effective responses in reducing tumor growth. Rodabe N. Amaria, the study’s principal investigator, highlighted that OBX-115 is unique in not requiring interleukin 2 (IL2), a cytokine often used with TIL cell therapies but can cause severe side effects.
Parameswaran Hari, Obsidian’s Chief Development Officer, expressed optimism about OBX-115’s potential to expand the eligibility for TIL cell therapy and address the unmet need in ICI-resistant advanced melanoma. Obsidian is also conducting a multicenter Phase 1/2 study involving metastatic melanoma and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) patients.
Additionally, Obsidian presented three other posters at AACR 2024, detailing research on TILs engineered with membrane-bound IL15 and LIGHT (TNFSF14). These studies demonstrate Obsidian’s continued commitment to advancing engineered cell and gene therapies for treating cancer.
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**Categories:** News
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### [Amra Medical's Breakthrough Fat Z-Score Biomarkers Unlock the Power of Tirzepatide Treatment](https://www.clinicaltrialvanguard.com/news/amra-medicals-breakthrough-fat-z-score-biomarkers-unlock-the-power-of-tirzepatide-treatment/)
**Published:** April 11, 2024
**Author:** Jon Napitupulu
**Content:**
Recent research from AMRA Medical and collaborators has highlighted the impact of [tirzepatide](https://www.clinicaltrialvanguard.com/news/aardvarks-new-obesity-pipeline-data-offers-surprising-hope/) treatment on fat distribution in patients with [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) (T2D). Using personalized fat z-scores derived from MRI scans, the study explored changes in fat storage patterns after 52 weeks of treatment.
Traditional clinical trials often rely on body weight and BMI as measures of treatment effectiveness. However, emerging research suggests that pharmacological interventions may alter fat distribution independently of weight loss. Fat distribution profiling through z-score assessment provides a comprehensive and modifiable endpoint for evaluating therapeutic efficacy.
The analysis of data from Lilly’s SURPASS-3 MRI study revealed significant changes in fat z-scores with tirzepatide treatment. Visceral fat z-score decreased by 0.18 SD, indicating a reduction in abdominal fat. Liver fat z-score decreased by 0.54 SD, suggesting a reduction in fat accumulation in the liver.
Interestingly, subcutaneous fat z-score increased slightly (+0.11 SD). This suggests a shift towards a more balanced fat distribution pattern, with a reduction in unhealthy visceral and liver fat and an increase in subcutaneous fat, which is less metabolically active.
The significant reduction in visceral and liver fat z-scores observed with tirzepatide indicates a potential targeted effect beyond that expected from weight reduction alone. This suggests that tirzepatide may have beneficial effects on fat metabolism and distribution, independent of its weight loss properties.
Implications for Treatment of [Obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) and Related Disorders
Fat distribution profiling through personalized z-score assessment offers a valuable tool for assessing the impact of pharmacological interventions on fat distribution in obesity and related disorders. Moving forward, this approach could serve as an essential endpoint in clinical trials evaluating the effectiveness of new therapeutic strategies.
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**Categories:** News
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### [EPiENDO Reports Results for Lead Asset, EP395, in COPD](https://www.clinicaltrialvanguard.com/news/epiendo-reports-results-for-lead-asset-ep395-in-copd/)
**Published:** April 11, 2024
**Author:** Jon Napitupulu
**Content:**
[EpiEndo](https://www.clinicaltrialvanguard.com/news/epiendos-pioneering-ep395-clinical-trial-for-copd-patients/) Pharmaceuticals has successfully concluded its Phase 2A clinical trial evaluating EP395, a novel oral therapeutic for Chronic Obstructive Pulmonary Disease (COPD).
•Safety and Tolerability: EP395 exhibited a favorable safety and tolerability profile, similar to antibiotic macrolides.
•Biomarker Effects: EP395 demonstrated beneficial effects on inflammatory biomarkers, indicating its potential to reduce disease-causing inflammation.
•No Antibiotic-Related Side Effects: Unlike antibiotic macrolides, EP395 did not cause gastrointestinal adverse events or hearing-related issues.
COPD is the third leading cause of death worldwide, and current treatment options are limited. EP395 represents a novel therapeutic approach that addresses the underlying epithelial barrier dysfunction associated with COPD.
The Phase 2A study enrolled 61 adults with COPD who received EP395 or placebo once daily for 12 weeks. Endpoints included safety, tolerability, and the impact of EP395 on inflammatory biomarkers.
EpiEndo plans to use the positive results from this trial and its recent LPS challenge study to advance the clinical development of EP395. The company is exploring partnering options to accelerate the development and delivery of this innovative therapy to patients.
EP395 is an orally available, non-antibiotic macrolide or “Barriolide™.” Barriolides™ target epithelial function, a key component of the body’s natural defense systems. By enhancing this barrier, EP395 can reduce inflammation and limit the risk of antimicrobial resistance.
EpiEndo Pharmaceuticals is a biopharmaceutical company focused on developing novel therapeutics that address chronic inflammatory disorders by targeting epithelial function. Its pipeline includes EP395 and other preclinical candidates for respiratory, gastrointestinal, and dermatological conditions.
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**Categories:** News
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### [Boundless Bio Announces First Patient Dosed in Phase 1/2 Trial for Cancer Patients](https://www.clinicaltrialvanguard.com/news/boundless-bio-announces-first-patient-dosed-in-phase-1-2-trial-for-cancer-patients/)
**Published:** April 12, 2024
**Author:** Jon Napitupulu
**Content:**
Boundless Bio has initiated patient enrollment in a Phase 1/2 clinical trial (STARMAP) to investigate BBI-825, a novel therapy targeting resistance gene amplifications in cancer.
BBI-825 is an oral ribonucleotide reductase (RNR) inhibitor, designed to inhibit the formation and repair of extrachromosomal DNA (ecDNA). EcDNA plays a crucial role in amplifying genes that drive cancer growth and resistance to targeted therapies.
Preclinical studies have shown that BBI-825 effectively suppresses tumor growth and induces tumor regression in models of resistance mediated by MAPK pathway-activated tumors.
• Phase 1/2 study involving patients with locally advanced or metastatic cancer
• Evaluation of BBI-825 as a single agent and in combination with select targeted cancer therapies
• Initial focus on patients with [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) harboring [KRASG12C](https://www.clinicaltrialvanguard.com/opinion/what-the-krystal-12-lancet-correspondence-actually-says-about-pro-reporting-in-krasg12c-trials/) and BRAFV600E mutations and resistance gene amplifications
Resistance to targeted therapies is a significant challenge in cancer treatment, particularly in colorectal cancer. The development of MAPK pathway and receptor tyrosine kinase gene amplifications often drives treatment resistance, leading to poor outcomes.
The STARMAP trial aims to address the unmet need for therapies that can prevent or overcome amplification-driven resistance in cancer. BBI-825, as a novel ecDTx, targets a fundamental mechanism of resistance, providing a potential breakthrough in oncogene amplified cancer treatment.
The positive results from this trial could support the expansion of BBI-825 into broader patient populations, including those with pan-tumor, pan-RAS, and pan-RAF indications.
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**Categories:** News
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### [DeepCure Closes $24M Funding Round Led by IAG Capital Partners](https://www.clinicaltrialvanguard.com/news/deepcure-closes-24m-funding-round-led-by-iag-capital-partners/)
**Published:** April 12, 2024
**Author:** Jon Napitupulu
**Content:**
[DeepCure](https://www.clinicaltrialvanguard.com/news/deepcure-presents-breakthrough-data-dc-9476-triumphs-over-etanercept-in-rheumatoid-arthritis-battle/), a pioneer in AI-powered small molecule drug discovery, has closed a $24.6 million Series A-1 financing round led by IAG Capital Partners. This brings the total funding raised by DeepCure to over $72 million since its inception.
DeepCure’s innovative platform combines generative AI with physics-based engines to discover novel small-molecule therapies for complex targets. It pinpoints potential interaction sites on proteins and designs synthesizable molecules with optimal properties. Additionally, DeepCure has developed an automated chemistry synthesis platform to enhance the tested molecules’ speed, quantity, and diversity.
“DeepCure has achieved technical breakthroughs in AI and chemistry synthesis automation that overcome critical barriers for small molecule discovery,” said Dr. Ehsan Jabbarzadeh, Venture Partner at IAG.
The Series A-1 proceeds will accelerate DeepCure’s technology development and advance its immunology and inflammation pipeline toward clinical trials.
IAG Capital Partners’ investment aligns with its commitment to supporting early-stage companies with transformative technologies. “These breakthroughs are pivotal to drugging a huge number of intractable targets that are known to have a central role in disease biology,” said Alex Kash, Associate at IAG.
DeepCure’s mission is to leverage its AI and automation capabilities to transform drug discovery and bring novel treatments to patients suffering from immunology-related and inflammatory diseases.
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**Categories:** News
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### [CND Life Sciences' Skin Biopsy Tool: A Breakthrough for Parkinson's Diagnosis](https://www.clinicaltrialvanguard.com/news/cnd-life-sciences-skin-biopsy-tool-a-breakthrough-for-parkinsons-diagnosis/)
**Published:** April 15, 2024
**Author:** Jon Napitupulu
**Content:**
The Synuclein-One Study has successfully detected P-SYN in cutaneous nerve fibers of patients with dementia with Lewy bodies (DLB) and mild cognitive impairment (MCI). Small skin biopsies, combined with advanced laboratory techniques, offer a promising diagnostic tool for detecting misfolded protein associated with [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease and other synucleinopathies.
The Synuclein-One Study results provide crucial insights into the early detection of neurodegenerative disorders, enabling more timely intervention and personalized treatment. Convenience and Accessibility: Skin biopsies offer a minimally invasive and accessible method for diagnosing synucleinopathies, facilitating widespread screening and monitoring.
The subgroup analysis of the Synuclein-One Study was recognized as an Abstract of Distinction for its scientific significance and clinical implications. Another abstract examines the practical application of synuclein skin biopsies in diagnosing and evaluating Parkinsonian disorders in clinical practice.
The research presented at the American Academy of Neurology 2024 Annual Meeting showcases the innovative use of cutaneous biopsies to identify P-SYN in patients with synucleinopathies. This groundbreaking technology promises significant advancements in the diagnosis and management of neurodegenerative diseases, fostering improved patient care and scientific progress.
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**Categories:** News
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### [Unlock the Latest in Cancer Treatment: Sanofi and Innate Pharma's Sar443579/IPH6101 Breakthrough](https://www.clinicaltrialvanguard.com/news/unlock-the-latest-in-cancer-treatment-sanofi-and-innate-pharmas-sar443579-iph6101-breakthrough/)
**Published:** April 16, 2024
**Author:** Jon Napitupulu
**Content:**
Innate Pharma has announced the initiation of the Phase 2 dose expansion portion of a clinical trial sponsored by Sanofi. The trial evaluates SAR443579, an investigational trifunctional anti-[CD123](https://www.clinicaltrialvanguard.com/news/unveiling-the-promising-advancements-in-blood-cancer-treatment-innate-pharmas-groundbreaking-trial-findings/) NK cell engager, as a monotherapy for treating blood cancers with high unmet medical needs. These cancers include relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia, and high-risk myelodysplasia.
SAR443579 results from a joint research collaboration between Innate Pharma and Sanofi. It has received FDA Fast Track Designation for treating acute myeloid leukemia. Preliminary efficacy and safety data from the dose-escalation phase of the trial were presented at the American Society of Hematology 2023 Annual Meeting.
“The progression of SAR443579 to the Phase 2 expansion phase demonstrates our commitment to bringing this innovative NK cell engager to patients,” said Dr. Sonia Quaratino, Chief Medical Officer of Innate Pharma. “Encouraging clinical efficacy has been observed in the dose escalation of Phase 1/2 in R/R AML patients, and we eagerly anticipate the results of the dose expansion.”
Under the terms of the 2016 research collaboration agreement with Sanofi, Innate has been triggered with a €4 million milestone payment.
The [ANKET](https://www.clinicaltrialvanguard.com/news/innate-pharmas-next-gen-anket-iph6501-highlighted-in-science-immunology/) (Antibody-based NK cell Engager Therapeutics) platform, developed by Innate Pharma, enables the creation of multi-specific NK cell engagers for cancer treatment.
Innate Pharma and Sanofi have a research collaboration and license agreement focused on developing innovative multi-specific antibody formats that engage NK cells for tumor cell elimination. Sanofi is responsible for developing, manufacturing, and commercializing products resulting from the collaboration, including SAR443579/[IPH6101](https://www.clinicaltrialvanguard.com/news/sanofi-venetoclax-breakthrough-nk-cell-therapy-stuns-at-ash-2023/) and SAR445514/IPH6401. Innate Pharma is eligible for up to €400 million in milestone payments and royalties on net sales.
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**Categories:** News
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### [Spikimm, Satt Conectus Join Forces for Innovative BK Virus Treatment](https://www.clinicaltrialvanguard.com/news/spikimm-satt-conectus-join-forces-for-innovative-bk-virus-treatment/)
**Published:** April 17, 2024
**Author:** Jon Napitupulu
**Content:**
SpikImm, a biotech company established in 2021, has entered an exclusive agreement with SATT Conectus for the development of monoclonal antibodies (mAbs) against the BK virus (BKV).
BKV, prevalent in early life, often persists latently. However, immunosuppressive treatments, such as those given to transplant patients, can reactivate the virus, leading to urinary tract complications. Kidney transplant recipients face risks of graft loss and [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/), while bone marrow recipients may develop hemorrhagic cystitis.
Research by Prof. Samira Fafi-Kremer and Prof. Pascal Poignard has revealed the protective role of anti-BKV neutralizing antibodies. Building on this, they developed potent anti-BKV mAbs through the HuMABK project funded by ANR and SATT Conectus.
SpikImm’s partnership with SATT Conectus grants them exclusive rights to these mAbs. These antibodies aim to provide long-term protection against BKV complications, offering a solution for transplant recipients.
SpikImm’s expertise, demonstrated in the rapid development of anti-SARS-CoV-2 mAbs, aligns with the company’s focus on developing long-acting mAbs to protect immunocompromised patients from viral infections, including BKV and SARS-CoV-
• Exclusive option agreement between SpikImm and SATT Conectus for anti-BKV mAbs
• Antibodies engineered for effective and lasting protection against BKV complications
• Potential for a prophylactic solution for transplant recipients
• SpikImm’s expertise in antibody discovery and clinical development
• Focus on safeguarding immunocompromised patients from viral infections
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**Categories:** News
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### [Volastra Therapeutics Announces First Patient Dosed in Phase Ib Clinical Trial of Sovilnesib](https://www.clinicaltrialvanguard.com/news/volastra-therapeutics-announces-first-patient-dosed-in-phase-ib-clinical-trial-of-sovilnesib/)
**Published:** April 17, 2024
**Author:** Jon Napitupulu
**Content:**
Volastra Therapeutics has advanced its clinical-stage [KIF18A](https://www.clinicaltrialvanguard.com/news/kif18a-therapies-fda-ema-approvals-trials-and-indications/) inhibitor, sovilnesib, into Phase Ib clinical trials. The trials will evaluate the safety and efficacy of sovilnesib in treating platinum-resistant or refractory high-grade serous [ovarian cancer](https://www.clinicaltrialvanguard.com/news/imunons-imnn-001-shows-lower-residual-disease-in-phase-2-ovarian-cancer-study/) (HGSOC).
Sovilnesib is designed to target cancers with high levels of chromosomal instability. The FDA has designated it Fast Track, recognizing its potential for treating a population with limited treatment options.
The Phase Ib trial (NCT06084416) is a randomized dose optimization study to determine the recommended Phase 2 dose. Patients will receive daily oral sovilnesib at various dose levels.
Volastra is concurrently developing two KIF18A inhibitors: sovilnesib and VLS-1488. This parallel clinical trial strategy aims to gather comparative data and ultimately select the most promising candidate for further development.
In the United States, over 20,000 new cases of ovarian cancer are diagnosed annually, and more than 75% are advanced. Most of these patients experience disease progression on platinum-based therapy.
Dr. Joyce Liu of Dana Farber Cancer Institute, a principal investigator on the trial, emphasized the need for new treatment options for advanced HGSOC patients.
Volastra is also pursuing biomarker approaches to identify patients who may respond to KIF18A inhibitors. Collaborations with Microsoft, Tailor Bio, and Function Oncology aim to develop tools to measure chromosomal instability and other response predictors.
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**Categories:** News
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### [Biotech Asher Secures $55 Million Funding: Phase 1B Trials on the Horizon](https://www.clinicaltrialvanguard.com/news/biotech-asher-secures-55-million-funding-phase-1b-trials-on-the-horizon/)
**Published:** April 17, 2024
**Author:** Jon Napitupulu
**Content:**
Asher [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/), a leader in developing targeted immunotherapies, has secured $55 million in Series C funding led by RA Capital Management. This investment will enable the company to advance its lead program, [AB248](https://www.clinicaltrialvanguard.com/news/asher-biotherapeutics-ab248-rilvegostomig-in-nsclc-phase-1b-2-study-announced/), a CD8-targeted IL-2 immunotherapy.
• The financing will fund Phase 1b monotherapy expansion data and early safety and efficacy results from the ongoing combination with pembrolizumab.
• New investors include [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) and Bristol Myers Squibb, demonstrating the value of Asher Bio’s approach in oncology.
• AB248 is designed to selectively activate CD8+ T cells, minimizing toxicity and maximizing efficacy.
• Early clinical data indicate potent CD8+ T cell activation without significant regulatory T and NK cell counts changes.
• Asher Bio’s platform offers a differentiated approach to immunotherapy with the potential to revolutionize cancer treatment.
“We are delighted to have the continued support of RA Capital, and excited to add two top biopharmaceutical companies and experts in oncology to our investor syndicate,” said Craig Gibbs, CEO of Asher Bio.
Asher Bio plans to utilize the proceeds to generate tumor response data from AB248 monotherapy expansion cohorts and data from dose escalation and expansion in combination with a PD-1 checkpoint inhibitor.
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### [Calidi Biotherapeutics IPO Raises $6.1 Million](https://www.clinicaltrialvanguard.com/news/calidi-biotherapeutics-ipo-raises-6-1-million/)
**Published:** April 17, 2024
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/), a biotechnology company focused on developing targeted immunotherapies, has priced its public offering for 15,197,500 shares of common stock or pre-funded warrants. The offering also includes Series A, B, and C Common Warrants, each comprising one share of common stock and a corresponding warrant.
The combined effective price for the offering is $0.40 per share and common warrant, generating approximately $6.1 million in gross proceeds. The common warrants have an exercise price of $0.60 per share and will expire at various intervals from the issuance date.
The proceeds from the offering will primarily be used for working capital, general corporate purposes, and pre-clinical and clinical trials. Ladenburg Thalmann & Co. Inc. is the sole placement agent for the offering.
The offering is being made through a registration statement filed with the Securities and Exchange Commission (SEC) and became effective on April 15, 2024. Copies of the preliminary and final prospectuses are available on the SEC’s website.
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### [Medincell's Partnership with AbbVie: Unlocking Next-Gen Injectable Therapies](https://www.clinicaltrialvanguard.com/news/medincells-partnership-with-abbvie-unlocking-next-gen-injectable-therapies/)
**Published:** April 17, 2024
**Author:** Jon Napitupulu
**Content:**
[Medincell](https://www.clinicaltrialvanguard.com/news/medincell-teva-olanzapine-lai-phase-3-positive-uzedy-real-world-data/) has teamed up with [AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/) to develop and commercialize up to six innovative long-acting injectable (LAI) therapies. This collaboration capitalizes on Medincell’s commercial-stage LAI technology and development expertise and AbbVie’s clinical development and commercialization capabilities.
Medincell will receive an upfront payment of $35 million and potential milestones of up to $1.9 billion based on development and commercial success. Additionally, royalties on worldwide sales are included in the agreement.
The first LAI program candidate has been selected, with formulation activities underway. AbbVie will oversee each program’s clinical development, regulatory approval, manufacturing, and commercialization.
Medincell’s BEPO® technology enables precise drug delivery over extended periods through subcutaneous or local injections. The first BEPO®-based treatment, UZEDY® (distributed by Teva), has been approved by the FDA for the treatment of [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/).
This collaboration is a testament to the growing recognition of LAIs as a promising treatment modality that addresses adherence issues, enhances efficacy, and reduces environmental impact. Medincell and AbbVie aim to leverage their combined strengths to bring innovative therapeutic solutions to patients globally.
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**Categories:** News
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### [Artifa's Allonk: Unprecedented Lupus Breakthrough](https://www.clinicaltrialvanguard.com/news/artifas-allonk-unprecedented-lupus-breakthrough/)
**Published:** April 18, 2024
**Author:** Jon Napitupulu
**Content:**
Artiva [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) has begun a Phase 1 trial of its AlloNK (AB-101) cell therapy in combination with monoclonal antibodies for lupus nephritis (LN). AlloNK is a cryopreserved, allogeneic NK cell therapy that enhances monoclonal antibodies’ activity against B-cells.
Autologous CAR-T cell studies have shown that B-[cell depletion](https://www.clinicaltrialvanguard.com/news/in8bio-t-cell-data-shows-b-cell-depletion-for-autoimmune/) can improve LN symptoms and potentially provide long-term responses. Artiva’s analysis of AlloNK’s Phase 1/2 trial in B-cell non-Hodgkin lymphoma (B-NHL) revealed that all patients achieved non-quantifiable B-cell levels within eight days of treatment, regardless of initial B-cell levels.
AlloNK has also shown complete responses in B-NHL patients, based on imaging of tumor lesions. Artiva believes these results support the therapeutic potential of AlloNK in autoimmune diseases, as B-NHL and autoimmune diseases share lymphoid tissues.
The Phase 1 trial will evaluate AlloNK’s safety and effectiveness in combination with rituximab or [obinutuzumab](https://www.clinicaltrialvanguard.com/article/trend-watch/the-b-cell-depletion-arms-race-in-cns-autoimmune-trials-has-a-new-front-runner-and-it-rewrites-the-regulatory-playbook/) in patients with relapsed or treatment-resistant LN. The treatment includes cyclophosphamide, fludarabine, AlloNK, and monoclonal antibodies.
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### [OPM Obtains €5.6 Million for DEMOCRITE Program’s Clinical Development](https://www.clinicaltrialvanguard.com/news/opm-obtains-e5-6-million-for-democrite-programs-clinical-development/)
**Published:** April 18, 2024
**Author:** Jon Napitupulu
**Content:**
[Oncodesign](https://www.clinicaltrialvanguard.com/news/oncodesigns-precision-medicine-a-new-era-in-systemic-radiotherapy/) Precision Medicine (OPM) has received €5.6 million in funding for the DEMOCRITE project, aimed at demonstrating the efficacy of [OPM-101](https://www.clinicaltrialvanguard.com/news/opm-101-ripk2-inhibitor-shows-strong-safety-no-cardiac-toxicity-in-phase-1-study/) in treating Inflammatory Bowel Disease (IBD).
OPM-101, derived from OPM’s Nanocyclix® platform, inhibits RIPK2, a key regulator of cell death and inflammation. The project focuses on Immune-Induced [Ulcerative Colitis](https://www.clinicaltrialvanguard.com/news/abivax-clears-pre-nda-meeting-with-fda-for-obefazimod-in-ulcerative-colitis/) (IIUC), a severe form of IBD.
The funding will support completing Phase 1 and Phase 2a clinical trials to establish proof of concept for OPM-101’s effectiveness in IIUC patients. The project also aims to secure a commercial partnership for the continued development of OPM-101 in IBD treatment.
OPM-101 is one of several kinase inhibitor molecules derived from the Nanocyclix® platform. Kinases are enzymes that regulate essential cellular functions, making them promising targets for precision medicine.
The success of the DEMOCRITE project will advance OPM’s mission to establish a precision medicine sector in France and solidify its position as a leader in kinase inhibitor development. By targeting RIPK2, OPM-101 has the potential to provide a novel and effective treatment option for patients with IBD.
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### [Unveiling the Revolutionary ALECENSA: A Game-Changer for ALK+ Lung Cancer](https://www.clinicaltrialvanguard.com/news/unveiling-the-revolutionary-alecensa-a-game-changer-for-alk-lung-cancer/)
**Published:** April 19, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has approved Alecensa (alectinib) for the treatment of patients with ALK-positive [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) (NSCLC) following surgical removal of the tumor.
This approval stems from the Phase III ALINA study, which demonstrated a significant reduction in the risk of disease recurrence or death by 76% in patients treated with Alecensa compared to standard chemotherapy. The study also showed an improvement in central nervous system (CNS)-disease-free survival.
The approval of Alecensa for adjuvant therapy marks a significant advancement for patients with ALK-positive early-stage lung cancer. “With an unprecedented 76% reduction in the risk of disease recurrence or death versus chemotherapy, Alecensa significantly improves upon the standard of care for people with early-stage ALK-positive lung cancer,” said Dr. Levi Garraway, Chief Medical Officer at Genentech.
Lung cancer is a common and life-threatening disease, with non-small cell lung cancer accounting for the majority of cases. Early-stage NSCLC is often treated with surgery to remove the tumor, but many patients experience recurrence despite adjuvant chemotherapy.
The approval of Alecensa highlights the importance of testing for ALK and other biomarkers in patients with early-stage NSCLC to guide treatment decisions. This allows for targeted therapies like Alecensa to be administered, offering the best chance of cure and improving patient outcomes.
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### [Lantern Pharma's Harmonic™ Trial Expands to Japan and Taiwan](https://www.clinicaltrialvanguard.com/news/lantern-pharmas-harmonic-trial-expands-to-japan-and-taiwan/)
**Published:** April 23, 2024
**Author:** Jon Napitupulu
**Content:**
Lantern Pharma has received regulatory approval to expand its Phase 2 clinical trial (HarmonicTM) to evaluate the efficacy of [LP-300](https://www.clinicaltrialvanguard.com/news/lantern-pharmas-remarkable-phase-2-trial-update-in-advanced-lung-cancer/) for [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) in non-smokers in Japan and Taiwan.
Approximately one-third of lung cancer cases in East Asia occur in non-smokers, a proportion that has been rising steadily. The expansion of the trial to these regions will accelerate the collection of data needed for further evaluation and development of LP-300 as a treatment for lung adenocarcinoma in non-smokers.
LCINS, characterized by distinct genetic and epidemiological features, has limited treatment options. Lantern believes that LP-300’s unique mechanism of action may provide a potential solution for this growing patient population.
Dr. Yashushi Goto of the National Cancer Center of Japan, a leading expert in lung cancer, will lead the trial in Japan. He expressed optimism about LP-300’s potential for NSCLC patients in East Asia, where EGFR mutations are prevalent.
The Harmonic trial is designed to assess the effects of LP-300 in combination with standard-of-care chemotherapy on the survival and disease progression of non-smoking patients with advanced NSCLC. The expansion to Japan and Taiwan is expected to provide valuable insights and contribute to the development of effective therapies for this patient population.
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### [Neurogene's Revolutionary Gene Therapy for Rett Syndrome: Exciting Data Unlocks Hope](https://www.clinicaltrialvanguard.com/news/neurogenes-revolutionary-gene-therapy-for-rett-syndrome-exciting-data-unlocks-hope/)
**Published:** April 23, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene’s [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/) gene therapy for Rett syndrome has exhibited promising safety results in initial clinical trials. After several months of observation, the therapy has been well-tolerated by all three patients, with no significant adverse events or signs of excessive MeCP2 protein production.
NGN-401 utilizes Neurogene’s EXACT transgene regulation technology to deliver a full-length MECP2 gene to specific brain regions affected by Rett syndrome. Dr. Rachel McMinn, Neurogene’s CEO, emphasizes the importance of this early safety data, as conventional gene therapy approaches have faced challenges in achieving therapeutic protein levels without causing harmful overexpression.
Neurogene plans to present detailed safety data at the American Society for Gene and [Cell Therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) ([ASGCT](https://www.clinicaltrialvanguard.com/news/meiragtx-showcases-gene-cell-therapy-advances-at-asgct-2025/)) Annual Meeting. The expected safety and efficacy update in the fourth quarter of 2024 will provide further insights into the potential of NGN-401 as a treatment for Rett syndrome. The therapy aims to address the narrow therapeutic window associated with this complex neurological disorder and offers hope for patients and families affected by it.
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**Categories:** News
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### [Anktiva®: An FDA-Approved Game-Changer for Non-Muscle Invasive Bladder Cancer](https://www.clinicaltrialvanguard.com/news/anktiva-an-fda-approved-game-changer-for-non-muscle-invasive-bladder-cancer/)
**Published:** April 23, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has granted approval for ANKTIVA, a novel immunotherapy, in combination with Bacillus Calmette-Guérin (BCG) for treating non-muscle invasive [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/) ([NMIBC](https://www.clinicaltrialvanguard.com/news/urogens-ugn-103-shows-94-5-six-month-response-in-nmibc-trial/)) that has not responded to BCG therapy. This milestone marks a significant advancement in the fight against NMIBC, a form of bladder cancer that can lead to debilitating consequences if not properly managed.
ANKTIVA’s unique mechanism of action mimics dendritic cell biology, activating both killer and helper T cells. This comprehensive approach enhances the immune system’s ability to proliferate and eliminate cancer cells, resulting in durable complete responses. Unlike checkpoint inhibitors, ANKTIVA targets multiple immune cell types, including NK cells, CD8+ killer T cells, and CD4+ T helper cells.
Clinical trials have demonstrated ANKTIVA’s efficacy, with a 62% complete response rate observed in patients with NMIBC. Notably, the duration of complete response exceeds 47 months, a significant milestone that surpasses the benchmark for meaningful clinical outcomes established by experts in the field.
The long-term benefits of ANKTIVA have the potential to reduce the need for cystectomy, a major surgical procedure that involves the removal of the bladder. Roger Buckley of the IBCG notes that ANKTIVA’s performance “exceeds the clinically meaningful benchmarks established in 2016 for durable complete response.”
ImmunotherapyBio’s dedication to developing cancer vaccines extends beyond ANKTIVA. The company aims to create preventative vaccines for individuals predisposed to cancer, such as those with Lynch syndrome. These advancements underscore [ImmunityBio](https://www.clinicaltrialvanguard.com/executiveinterviews/immunitybios-visionary-approach-to-cancer-immunotherapy/)‘s commitment to revolutionizing cancer treatment and improving the quality of life for patients worldwide.
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### [Exciting New Clinical Trial Results for Duchenne Muscular Dystrophy](https://www.clinicaltrialvanguard.com/news/exciting-new-clinical-trial-results-for-duchenne-muscular-dystrophy/)
**Published:** April 24, 2024
**Author:** Jon Napitupulu
**Content:**
The international gene therapy trial for [Duchenne Muscular Dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) Muscular Dystrophy (DMD) using GNT0004 has unveiled promising findings at the Myology 2024 conference. The trial, led by Professor Francesco Muntoni, combines phases I to III with a dose escalation stage, followed by a pivotal phase.
The trial has enrolled ambulant boys aged 6 to 10 with DMD and has received approval from French and UK authorities. Five patients have been administered GNT0004, demonstrating good tolerability and immunological response.
Initial efficacy results for the patient receiving the higher dose (3×10^13 vg/kg) after one year showed clinical improvement, including an inflection in the North Star Ambulatory Assessment score. Other functional assessments also displayed positive trends.
The safety and efficacy data have enabled Genethon, the trial’s sponsor, to prepare the pivotal European phase with the European Medicines Agency (EMA).
GNT0004 is an AAV8 gene therapy containing a shortened and functional version of the DMD gene that encodes dystrophin, the deficient protein in DMD. It targets key tissues like skeletal and cardiac muscles through a single intravenous injection.
DMD is a rare genetic disease that affects muscle function. Its absence leads to muscle degeneration and complications such as cardiomyopathy and loss of mobility. The trial’s progress represents a significant step towards potential therapeutic options for DMD.
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**Categories:** News
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### [Incyte's Acquisition: Unlocking a Promising Pipeline of Groundbreaking Medicines](https://www.clinicaltrialvanguard.com/news/incytes-acquisition-unlocking-a-promising-pipeline-of-groundbreaking-medicines/)
**Published:** April 24, 2024
**Author:** Jon Napitupulu
**Content:**
Incyte, a renowned biopharmaceutical company, has acquired Escient Pharmaceuticals, a pioneer in developing small molecule therapeutics for immune disorders. This strategic move expands Incyte’s portfolio with two first-in-class drugs, [EP262](https://www.clinicaltrialvanguard.com/news/incyte-acquires-escient-pharmaceuticals-for-uncommon-progress/) and EP547, targeting systemic and neuro-immune conditions.
EP262, a potent antagonist of the MRGPRX2 receptor, holds promise for treating mast cell-mediated diseases such as [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/), chronic inducible urticaria, and chronic spontaneous urticaria. Preclinical studies have demonstrated its efficacy in reducing skin lesions and inflammatory markers. In a Phase 1 study, EP262 exhibited safety and tolerability with minimal adverse events.
EP547, an oral MRGPRX4 antagonist, is designed for immune-mediated disorders. Incyte believes these drugs complement its existing portfolio and offer significant potential for addressing unmet medical needs in inflammatory diseases.
“We are thrilled to incorporate EP262 and EP547 into our portfolio,” stated Hervé Hoppenot, Incyte’s Chief Executive Officer. “These innovative therapies align with our strategy of developing first-in-class medicines with transformative potential.”
The acquisition includes Escient’s assets and intellectual property. Incyte will utilize its inflammation and autoimmunity expertise to develop further and commercialize EP262 and EP547, aiming to bring these novel therapies to market by 2029.
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**Categories:** News
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### [Exgenesis Bio: Positive Phase 1/2 Clinical Trial Data for EXG001-307 Gene Therapy for Spinal Muscular Atrophy](https://www.clinicaltrialvanguard.com/news/exgenesis-bio-positive-phase-1-2-clinical-trial-data-for-exg001-307-gene-therapy-for-spinal-muscular-atrophy/)
**Published:** April 26, 2024
**Author:** Jon Napitupulu
**Content:**
Exegenesis Bio, a leading genetic medicines company, unveiled encouraging clinical efficacy and safety results for their EXG001-307 gene therapy in SMA Type 1 patients at the American Society of Gene and [Cell Therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) ([ASGCT](https://www.clinicaltrialvanguard.com/news/meiragtx-showcases-gene-cell-therapy-advances-at-asgct-2025/)) Annual Meeting.
EXG001-307 is a novel recombinant adeno-associated virus (rAAV) gene therapy designed to address the genetic defect responsible for SMA. Nine patients treated with EXG001-307 exhibited significant improvements in motor function, including increased head control and the ability to sit independently within three months of dosing.
Notably, EXG001-307 demonstrated a superior safety profile compared to existing gene therapies, with reduced off-target expression in liver and heart tissue. This unique AAV design, featuring a pro-NS promoter, enhances target gene expression in the spinal cord while minimizing risks in other organs.
Based on the promising data, Exegenesis Bio plans to file an Investigational New Drug (IND) application for EXG001-307 in SMA Type 1 in the United States in the fourth quarter of 2024. The company is also considering an accelerated development pathway for SMA Type 2/3 patients.
SMA is a rare genetic disorder that affects the motor neurons, leading to muscle weakness, atrophy, and ultimately respiratory failure. Type 1, the most severe form, affects infants and has a high mortality rate. EXG001-307 represents a promising therapeutic option with the potential to significantly improve outcomes for these young patients.
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**Categories:** News
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### [Landmark Nature Paper Unveils Groundbreaking Covalent Inhibitor for Rare Disease](https://www.clinicaltrialvanguard.com/news/landmark-nature-paper-unveils-groundbreaking-covalent-inhibitor-for-rare-disease/)
**Published:** April 26, 2024
**Author:** Jon Napitupulu
**Content:**
[Vividion Therapeutics](https://www.clinicaltrialvanguard.com/news/vividion-therapeutics-to-expand-with-new-global-research-and-development-center/), a biopharmaceutical company, has made a significant discovery in the development of novel cancer and immune disorder treatments.
Vividion’s chemoproteomic platform enabled the discovery of a covalent allosteric inhibitor of WRN helicase, known as VVD-133214 (RO7589831). WRN is a promising target for cancers with microsatellite instability.
The publication of the discovery in Nature highlights Vividion’s approach to identifying and developing new therapeutics. The company’s platform has allowed it to target traditionally undruggable targets by identifying functional pockets on proteins and compounds that specifically interact with them.
Roche, Vividion’s partner, is currently investigating VVD-133214 in a Phase 1 clinical trial. The success of this drug candidate demonstrates the potential of Vividion’s chemoproteomics platform to advance new treatments for difficult-to-treat diseases.
Vividion is a subsidiary of Bayer AG and has leveraged its platform to identify numerous previously unknown functional pockets on protein targets. The company’s proprietary covalent chemistry library contains compounds that selectively interact with these pockets, enabling the development of a diverse pipeline of small molecule therapeutics for a wide range of diseases.
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### [Taking Stock of Obesity Drug Development: Trends and Challenges Facing Today’s Researchers](https://www.clinicaltrialvanguard.com/article/taking-stock-of-obesity-drug-development-trends-and-challenges-facing-todays-researchers/)
**Published:** April 30, 2024
**Author:** Jack L. Martin
**Content:**
Rising levels of obesity have become a global health issue, with an estimated 2.8 million people dying each year as a result.1 In the face of this major medical crisis, the development of effective drugs and interventions for the treatment of obesity is clearly an important undertaking. And, with recent advances in the field — such as the use of glucagon-like peptide-1 **(**GLP-1) receptor agonists — the future of obesity drug development is experiencing dramatic shifts.
ICON recently conducted a survey of more than 100 professionals currently engaged in obesity drug and device research and development. Representing organisations that range from small biotechs to large pharmaceutical companies and academic institutions, these professionals are well positioned to provide insight into the current obesity drug and device research landscape, as well as where it is headed. Using these survey results within the greater context of obesity treatments, we can understand current trends and challenges in obesity research.
#### [](#putting-obesity-drugs-in-context)**Putting obesity drugs in context**
Historically, methods of treating obesity have been limited. Lifestyle modification – such as diet, exercise and behavioural therapy – rarely takes into account significant risk factors, including genetics, environment and other elements outside a patient’s control. And lifestyle modification, alone, has been found to be insufficient in achieving lasting weight loss. Case in point: Patients who lose weight through lifestyle changes typically regain 80 percent of weight lost within five years.2
Bariatric surgery has been considered a more effective approach. Yet, it is highly invasive and only recommended for a limited subset of patients. And past pharmacological therapies for obesity, such as the combination of phentermine and fenfluramine popular in the 1990s, have led to serious health concerns, including primary pulmonary hypertension and heart valve disease.
Recent years have seen significant strides in pharmacological therapies. Of particular note are the regulatory approvals of GLP-1 receptor
Jack L. Martin, MD, FACC Sr. Director, Cardiovascular Therapeutics, Drug Development Solutions, ICON
agonists as well as dual (GLP-1 and gastric inhibitory polypeptide) incretin receptor agonists, such as [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/) and [tirzepatide](https://www.clinicaltrialvanguard.com/news/aardvarks-new-obesity-pipeline-data-offers-surprising-hope/), to treat obesity. Considered among the most powerful anti-obesity agents currently available, this class of drugs stimulates insulin production and synthesis, and also reduces appetite and slows gastric emptying. Studies have also shown positive impacts on obesity comorbidities such as cardiovascular diseases and non-alcoholic fatty liver disease.
With pharmacotherapy research ongoing, these approved incretin receptor agonists are only the tip of the iceberg. New GLP-1 receptor agonists are being investigated – both individually and in combination with other mechanisms. Other researchers are exploring the potential of amylin receptor agonists or analogues, as well as the use of tocotrienols. Of the respondents to ICON’s survey, two thirds (66 percent) are confident in their organisation’s obesity-related pipeline success prospects, which speaks to a promising future for obesity treatment.
#### [](#the-multi-indication-trend)**The multi-indication trend**
Given the interconnected nature of obesity and its associated comorbidities, the importance of addressing these conditions alongside obesity has become apparent to those involved in obesity drug research and development. A key theme that underpinned a great deal of ICON’s survey was the inclination toward multi-indication and combination therapies. Despite the success of incretin agonists, just 17 percent of respondents felt the future of drug development lay in single therapies such as semaglutide, tirzepatide or retratrutide. Instead, 64 percent thought that combination therapies for the treatment of obesity and its related comorbidities would represent the future.
This was reflected in the research that was reported by survey respondents. Only 37 percent stated that they were investigating obesity alone. Meanwhile, for those who included other indications in their obesity research, a wide variety of therapeutic areas were represented, with diabetes (55 percent), metabolism (48 percent), [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) (39 percent) and cardiovascular diseases (38 percent) being the most commonly selected.
#### [](#facing-the-challenges-of-clinical-trials)**Facing the challenges of clinical trials**
Despite promising developments in the field of obesity, and researchers’ confidence in their pipelines, there are still obstacles to overcome. Of the various challenges facing obesity drug and device developers, 31 percent of respondents considered issues associated with clinical trials as top on their list. Specifically, of the elements that go into a clinical trial, the three ranked most challenging were: lack of obesity-appropriate trial design (39 percent); lack of long-term follow up (44 percent); and difficulty recruiting diverse patient populations (38 percent). Nevertheless, these challenges are not insurmountable.
*Clinical trial design*
Traditional clinical trial design may not always be the best choice for obesity studies. As reported in the survey, half (50 percent) of current obesity clinical studies include multi-indication components, creating challenges not seen when investigating a therapy for one indication at a time. Innovative trial designs provide a promising route for these studies, such as through master protocols, which are specifically constructed to test multiple hypotheses and may include parallel sub-studies, but have an overarching set of procedures to improve efficiency. For instance, under master protocols, a basket trial design is well suited to account for the interconnected therapeutic areas found within obesity-related comorbidities. This allows a single investigational drug or drug combination to be tested on multiple indications or subtypes of a single disease, which have a common molecular characteristic.
*Long-term follow up*
After initial approval is obtained based on weight loss, long-term studies are needed to address effects on the clinical events associated with obesity. This data is needed to compete against marketed products with an indication for event reduction. Novo Nordisk’s SELECT trial is an excellent example. Here, a five-year study provided clinical evidence showing that the company’s drug, Wegovy (semaglutide), reduced the occurrence of major adverse cardiovascular events, boosting Wegovy’s prospects clinically and financially.3
However, long term studies are expensive, highlighting the need to create efficiencies in execution. Effective compliance and retention improve efficiency and be achieved with patient centric trial designs including remote and hybrid study visits that ease patient burden. Traditional time-to-event analysis of clinical outcomes trials may not be as efficient as alternative statistical approaches that in some circumstances can reduce sample size.
After a clinical outcome trial has completed further follow-up can provide valuable insights into the potential for continued benefits. To help continue collecting patient data, clinical trial tokenisation enables the gathering of a participant’s health data on an ongoing basis through secondary sources such as electronic health records.
*Diverse recruitment*
Now more than ever, there is increasing pressure to ensure the recruitment of representative patient populations in clinical trials. In obesity clinical trials, the challenge is twofold: Despite similar levels of obesity across gender, women typically make up approximately three quarters of obesity clinical trial participants. At the same time, racial and ethnic minorities are typically underrepresented in these trials.
To improve diverse recruitment, targeted messaging, which includes demographic-specific language and imagery, can be useful. Additionally, the strategic use of technology, such as incorporating a smartphone element, can improve enrolment as well. Further, survey respondents believe that reducing patient burden (33 percent) and monetary incentives (30 percent) are the factors that could have the greatest impact in improving the diversity of the patient population during recruitment.
#### [](#the-future-of-obesity-treatment)**The future of obesity treatment**
Looking to the future of human health, obesity levels are only expected to grow. In fact, the World Obesity Foundation estimates that by 2030, one billion people will be living with obesity globally. However, advances in research — including obesity treatment pipelines that instil confidence in more than half of researchers — and improving approaches to clinical trials are pointing to a very hopeful future.
Sources:
1. Obesity. detail/6-facts-on-obesity. Accessed 11 Aug. 2023.
2. Hall, Kevin D., and Scott Kahan. “Maintenance of Lost Weight and Long-Term Management of Obesity.” The Medical Clinics of North America, vol. 102, no. 1, Jan. 2018, pp. 183–97, .
3. “SELECT: Semaglutide Reduces Risk of MACE in Adults With Overweight or Obesity.” American College of Cardiology, . Accessed 22 Sept. 2023
**Categories:** Article
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### [Merck's V116 Vaccine: A Revolutionary Breakthrough for Adult Pneumococcal Protection](https://www.clinicaltrialvanguard.com/news/mercks-v116-vaccine-a-revolutionary-breakthrough-for-adult-pneumococcal-protection/)
**Published:** April 30, 2024
**Author:** Jon Napitupulu
**Content:**
A Phase 3 trial (STRIDE-10) evaluated V116, a 21-valent pneumococcal conjugate vaccine, in adults over 50. The study compared V116 to a standard 23-valent pneumococcal vaccine (PPSV23) and assessed its immunogenicity, safety, and tolerability.
Key findings revealed that V116 induced strong immune responses to the serotypes responsible for the majority of invasive pneumococcal disease in adults. These results complement previous positive findings from other Phase 3 trials.
Despite the availability of pneumococcal conjugate vaccines for adults, there remain gaps in serotype coverage for invasive pneumococcal disease. V116’s design specifically targets the serotypes responsible for approximately 83% of adult invasive pneumococcal disease in individuals over 65.
Additional data presented at the European Society of Clinical Microbiology and Infectious Diseases ([ESCMID](https://www.clinicaltrialvanguard.com/news/assembly-biosciences-presents-new-data-on-herpes-simplex-virus-candidate-at-escmid/)) suggests that V116 may help reduce the health and economic burden associated with pneumococcal disease in adults. Studies conducted in France, Sweden, Spain, and the Netherlands support this potential benefit.
V116 is currently under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has granted V116 priority review, with a target action date of June 17, 2024. V116 aims to provide a new preventive option for adults against invasive pneumococcal disease.
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**Categories:** News
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### [Tivdak's Full FDA Approval: A Triumphant Victory for Metastatic Cervical Cancer Treatment](https://www.clinicaltrialvanguard.com/news/tivdaks-full-fda-approval-a-triumphant-victory-for-metastatic-cervical-cancer-treatment/)
**Published:** April 30, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has granted full approval to TIVDAK ([tisotumab](https://www.clinicaltrialvanguard.com/news/pfizer-tivdak-sla-accepted-by-fda-for-priority-cervical-cancer-review/) vedotin-tftv) for the treatment of patients with recurrent or metastatic cervical cancer who have undergone prior chemotherapy.
The approval is based on results from the Phase 3 innovaTV 301 trial, which demonstrated significant overall survival (OS) benefits for patients treated with TIVDAK compared to chemotherapy. The median OS was 11.5 months for patients receiving TIVDAK versus 9.5 months for those receiving chemotherapy.
The trial also met secondary endpoints of progression-free survival (PFS) and confirmed objective response rate (ORR).
The safety profile of TIVDAK is consistent with its known adverse events, including peripheral neuropathy, hemorrhage, pneumonitis, and ocular toxicity. The most common side effects observed in the innovaTV 301 trial were hemoglobin decrease, peripheral neuropathy, and conjunctival adverse reactions.
TIVDAK is the first antibody-drug conjugate to demonstrate statistically significant OS prolongation in patients with recurrent or metastatic cervical cancer. This approval provides a new treatment option for patients with this devastating disease.
Genmab is continuing its clinical development program to explore the potential of TIVDAK in other indications.
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**Categories:** News
---
### [FDA Approves Pfizer's Gene Therapy Treatment for Hemophilia B](https://www.clinicaltrialvanguard.com/news/fda-approves-pfizers-gene-therapy-treatment-for-hemophilia-b/)
**Published:** April 30, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer’s BEQVEZ, a one-time gene therapy, has received FDA approval for treating adults with moderate to severe [hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/) B. Patients who have life-threatening hemorrhages, repeated spontaneous bleeding episodes, or current use of factor IX (FIX) prophylaxis therapy are eligible for this treatment.
Hemophilia B is a rare disorder that affects blood clotting due to a deficiency in FIX. The standard treatment involves frequent intravenous FIX infusions. However, BEQVEZ aims to eliminate this by enabling patients to produce FIX on their own, reducing the need for regular injections.
Dr. Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program, highlights the potential of BEQVEZ to alleviate the burden and disruption associated with traditional FIX infusions.
BEQVEZ’s unique mechanism may also benefit healthcare systems by reducing strain on resources and budgets. Despite prophylactic infusions, many hemophilia B patients experience spontaneous bleeding episodes.
Pfizer’s Chief U.S. Commercial Officer, Aamir Malik, emphasizes the company’s commitment to advancing hemophilia care. Pfizer plans to collaborate with healthcare providers and the hemophilia community to ensure accessibility to BEQVEZ for eligible patients.
For patients, BEQVEZ offers not only long-term protection against bleeding but also potential time savings from eliminated treatments. Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B, highlights the positive impact BEQVEZ could have on the lives of hemophilia B patients.
Pfizer has implemented a warranty program to assure durability of treatment response. This measure provides certainty for payers, maximizes access for eligible patients, and offers financial protection against inefficacy risks.
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**Categories:** News
---
### [c2n Diagnostics partners with Mediford for Precivity™ Alzheimer's Blood Test Expansion in Japan](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-partners-with-mediford-for-precivity-alzheimers-blood-test-expansion-in-japan/)
**Published:** April 30, 2024
**Author:** Jon Napitupulu
**Content:**
[C2N Diagnostics](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/) has partnered with Mediford Corporation, a subsidiary of PHC Holdings, to enhance access to its clinical research services in Japan.
C2N’s Precivity™ blood tests provide highly accurate identification and quantification of biomarkers related to neurological diseases, including [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/). The tests aid in diagnosis, prognosis, and treatment monitoring. Notably, C2N’s innovative MTBR-tau biomarker aims to detect neurofibrillary tangles associated with tau protein aggregation in the brain.
This partnership expands C2N’s global footprint and leverages Mediford’s extensive network in the Japanese biopharma industry. Maki Hoshiko, a renowned neuroscientist, has been appointed to lead C2N’s Japan strategy and collaborations.
Japan has a high prevalence of Alzheimer’s disease, with 26% of diagnosed cases globally. The country also has the highest proportion of people with dementia.
C2N’s biomarkers are used in multiple clinical trials, including the Clarity AD trial for lecanemab and the TRAILBLAZER-ALZ 2 trial for [donanemab](https://www.clinicaltrialvanguard.com/news/tizianas-phase-2-alzheimers-trial-doses-first-patient/). They help improve clinical trial enrollment efficiency and cost-effectiveness.
C2N’s partnership with Mediford demonstrates its commitment to expanding its reach and providing access to advanced biomarker research services worldwide. The collaboration aims to accelerate the development of treatments and improve patient care for neurological conditions.
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**Categories:** News
---
### [Walmart Health Center Exit: Is It Good For Clinical Trials?](https://www.clinicaltrialvanguard.com/article/walmart-health-center-exit-is-it-good-for-clinical-trials/)
**Published:** May 1, 2024
**Author:** Moe Alsumidaie
**Content:**
*This article contains speculative content, opinions, and assumptions about the Walmart Health center that are not confirmed facts. The analysis regarding Walmart’s withdrawal from health centers does not necessarily imply a withdrawal from clinical trials. Readers should verify facts independently.*
Walmart’s foray into the healthcare sector through establishing Walmart Health centers and the Walmart Healthcare Research Institute signaled a bold attempt to integrate retail operations into direct healthcare delivery and clinical trials. The company sought to leverage its extensive retail presence to make healthcare more accessible, especially in underserved areas, while also expanding into the digital health and clinical research domains. This approach mirrored Walmart’s overarching strategy of accessibility and affordability, targeting a sector ripe for innovation. However, despite notable growth in their Health & Wellness segment and strategic expansions, Walmart’s withdrawal from the healthcare landscape (and presumably also clinical trials) raises pertinent questions about the viability of retail giants transforming healthcare delivery and clinical trials.
#### [](#walmart-health-center-and-its-history-with-clinical-trials)Walmart Health Center and its History With Clinical Trials
Walmart [officially entered the healthcare sector in 2019](https://corporate.walmart.com/news/2020/09/17/one-year-in-walmart-health-is-delivering-affordable-healthcare-and-expanding "officially entered the healthcare sector in 2019") by opening its first Walmart Health center in Dallas, Georgia. The launch marked the beginning of a significant expansion into offering comprehensive healthcare services directly to communities, particularly focusing on underserved areas. Walmart Health centers provided various services, including primary and urgent care, labs, x-rays, dental, optical, and counseling services. They aimed to make healthcare more affordable and accessible, aligning with the company’s broader market strategy of low prices and convenience.
Since then, Walmart Health grew, opening more centers primarily in the Southeastern U.S., and also delved into telehealth services by [acquiring MeMD in May 2021](https://corporate.walmart.com/news/2021/05/06/walmart-health-to-acquire-telehealth-provider-memd "acquiring MeMD in May 2021"), further expanding its reach into virtual care. This move was part of a broader strategy to integrate digital health services, especially significant during the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic when the demand for such services surged.
Walmart then [entered the clinical trials space in October 2022](https://corporate.walmart.com/news/2022/10/11/walmarts-healthcare-research-institute-launches-with-mission-to-improve-care-for-underserved-communities-through-research "entered the clinical trials space in October 2022") with the Walmart Healthcare Research Institute (WHRI) launch. This initiative aimed to improve access to clinical research, emphasizing inclusivity, especially in underrepresented communities, and delivering safer, higher-quality, and more equitable healthcare research solutions. This was also the case with [CVS in May 2021](https://www.cvshealth.com/news/clinical-trial-services/cvs-health-introduces-clinical-trial-services.html/1000 "CVS in May of 2021") (who [shuttered the initiative two years later](https://www.appliedclinicaltrialsonline.com/view/why-did-cvs-shutter-its-clinical-trials-unit- "shuttered the initiative two years later")), [Walgreens in June 2022](https://www.reuters.com/business/healthcare-pharmaceuticals/walgreens-enters-clinical-trials-business-through-new-unit-2022-06-16/ "Walgreens in June of 2022"), and then [Kroger in January 2023](https://ir.kroger.com/news/news-details/2023/Kroger-Health-Establishes-Clinical-Trial-Site-Network-to-Increase-Reach-and-Access-to-Research-Studies/default.aspx "Kroger in January, 2023").
Walmart’s Health & Wellness [sales grew by 8.76% from 2021-2022 and 17.83% from 2022-2023 (to nearly $55 million)](https://www.sec.gov/ix?doc=/Archives/edgar/data/104169/000010416924000056/wmt-20240131.htm "sales grew by 8.76% from 2021-2022 and 17.83% from 2022-2023 (to nearly $54 million)"), and it’s most recent [quarterly report](https://s201.q4cdn.com/262069030/files/doc_earnings/2024/q4/presentation/Earnings-Presentation-FY24-Q4.pdf "quarterly report") indicates strong pharmacy sales were driven by an increase in prescription counts, a greater proportion of branded versus generic prescriptions, robust immunization rates, and inflation in branded drug prices.
Walmart’s revenue reports, Form 10-K, 2024However, on April 30, 2024, [Walmart announced it would shutter the Walmart Health center](https://corporate.walmart.com/news/2024/04/30/walmart-health-is-closing "Walmart announced it would shutter the Walmart Health center"), and speculatively also the ability to conduct clinical trials, given that these centers possess the necessary infrastructure for clinical research conduct. This decision suggests that the growth and profitability of the Walmart Health centers may not have met the company’s broader corporate objectives or could reflect a strategic realignment, despite the observed overall growth in the health and wellness sector.
#### [](#what-do-industry-and-experts-leaders-think)What Do Industry and Experts Leaders Think?
The recent closure of Walmart Health’s clinics and its virtual care services has cast a somber shadow over the aspirations of integrating retail giants into the healthcare and clinical trials sector. Industry leaders and experts express disappointment and contemplation as they digest the implications of Walmart’s decision. Many are questioning the sustainability of such business models within retail, especially given the challenges highlighted by Walmart’s experience, such as pricing strategies, recruitment and retention of medical staff, and the complexity of healthcare contracts. The ambition to transform retail spaces into hubs for clinical trials and comprehensive healthcare seemed promising as a means to expand access, particularly in underserved areas. However, Walmart’s retreat prompts a reevaluation of how such large-scale operations align with healthcare services’ intricate, regulation-heavy landscape. This turn of events has left many pondering the future role of retail in healthcare innovation and access, signaling a cautious reassessment of expectations for similar ventures by other retail entities in the clinical trials and healthcare market.
---
---
---
#### [](#giants-enter-and-exit-all-the-time)Giants Enter and Exit All the Time
Walmart’s recent exit from the healthcare and clinical trials space is reminiscent of Amazon’s foray and subsequent withdrawal from the restaurant delivery service. Both giants ventured outside their traditional business models, seeking to leverage their vast infrastructures to disrupt well-established industries—healthcare for Walmart and food delivery for Amazon. In each case, despite the initial promise of transforming access (healthcare access in rural areas for Walmart and food delivery market reach for Amazon), both companies struggled to secure a sustainable foothold.
Amazon Restaurants aimed to compete with entrenched players like Grubhub, Uber Eats, and DoorDash but [ultimately failed to capture sufficient market share, leading to its shutdown](https://www.geekwire.com/2019/amazon-shut-amazon-restaurants-business-u-s/ "ultimately failed to capture sufficient market share, leading to its shutdown"). Similarly, Walmart Health, despite its initial ambitious expansion to serve healthcare deserts, could have faced challenges such as staffing, contracting with payers, and the complexities of healthcare regulations, which may have impeded its ability to establish a profitable business model (but we may never know the true reason for its exit). The exits of both Walmart and Amazon from these ventures reflect the difficulties large retail-oriented companies face when integrating into sectors where operational expertise and specialized market strategies are crucial for success.
The aftermath of Amazon’s exit from the restaurant delivery service temporarily affected investor sentiment, making stakeholders wary about the viability of similar new entrants disrupting established markets. However, existing players who have established themselves [received additional funding](https://www.geekwire.com/2019/amazon-shut-amazon-restaurants-business-u-s/ "received additional funding"). Walmart’s departure from healthcare might similarly influence perceptions around the practicality and potential success of retail giants diversifying into specialized and heavily regulated fields like healthcare and clinical trials. Both examples highlight the challenges of market adaptation and the importance of industry-specific nuances in determining major corporations’ success in diversification strategies.
#### [](#walmarts-exit-might-be-a-good-thing-for-existing-players-in-clinical-trials)Walmart’s Exit Might be a Good Thing for Existing Players in Clinical Trials
Walmart’s decision to exit the healthcare delivery sector (and presumably also the clinical trials industry) could catalyze invigorating existing players, especially established clinical study sites and decentralized clinical trial (DCT) providers. This retreat by a major retail player reduces competition, potentially redirecting funding and resources toward traditional brick-and-mortar and independent clinical research facilities. This shift could lead to enhanced investment opportunities for specialized research entities, particularly in rural and underserved areas, where Walmart has a notable presence. Consolidating clinical trial opportunities not only promises a more focused allocation of resources but also augments the appeal of dedicated clinical research operations to investors who value specialized, high-quality research endeavors over the broader, less specialized approaches seen in retail-based health services.
Moreover, the burgeoning field of DCTs stands to gain significantly from Walmart’s departure. As the clinical trial landscape increasingly shifts towards remote and patient-centric models, the demand for advanced DCT solutions is expected to escalate. DCT companies can capitalize on this opportunity by expanding their technological offerings and strengthening their market position, attracting substantial investor interest. This market realignment could surge investor confidence and funding, emphasizing clinical trials’ intricate and specialized nature.
#### [](#summary)Summary
The recent announcement that Walmart will close its health centers and potentially cease its clinical trial efforts marks a pivotal moment in the healthcare industry, particularly for clinical trials. This decision could recalibrate the competitive landscape, potentially benefiting traditional and specialized clinical trial entities. As Walmart exits, it leaves a gap that could lead to increased investments and enhancements in clinical research facilities and decentralized clinical trial technologies, which are more aligned with current trends toward patient-centric research models. The shift could foster a more robust funding environment, as investors might now be more inclined to support ventures that demonstrate a focused and expert approach to clinical trials rather than broad retail-based health initiatives. Ultimately, Walmart’s retreat could highlight the complexities of integrating retail with healthcare and clinical research, prompting a more cautious but potentially more fruitful investment approach in the specialized realms of healthcare delivery and clinical trials.
---
**Update:**
*Walmart has clarified their ongoing commitment to healthcare innovation through the Walmart Healthcare Research Institute (WHRI). The company confirmed that clinical trials were not conducted in Walmart Health centers. This clarification reinforces Walmart’s dedication to expanding their core businesses and introducing new health programs alongside their existing services. Walmart emphasizes its strategy of continual innovation aimed at enhancing customer well-being. This update is provided to ensure transparency and prevent any misinterpretations or speculation regarding Walmart’s healthcare initiatives.*
**Categories:** Analysis, Article, Article: Opinion
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### [Trisalus Strengthens Infusion System Growth with $50M Debt Financing](https://www.clinicaltrialvanguard.com/news/trisalus-strengthens-infusion-system-growth-with-50m-debt-financing/)
**Published:** May 1, 2024
**Author:** Jon Napitupulu
**Content:**
TriSalus [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) recently secured a $50 million debt facility from OrbiMed. This financing aims to support TriSalus’ expansion plans and continued growth, enhancing its financial flexibility and allowing for further investment in its TriNav commercial and technology pipeline.
To execute strategic initiatives, TriSalus borrowed $25 million initially, with an option for additional tranches up to $25 million based on revenue milestones. The five-year credit agreement matures in 2029. As part of the closing, TriSalus issued OrbiMed a warrant to purchase shares worth $9.56.
Combined with current cash reserves, the financing extends TriSalus’ cash runway to 2025. Mary Szela, CEO of TriSalus, emphasizes that this capital will support the growth of TriNav, its PEDD technology, and its technology pipeline. The company aims for TriNav’s break-even EBITDA in 2025, reducing the need for near-term equity financing.
Matthew Rizzo of OrbiMed expressed their support for TriSalus, highlighting the funding’s role in providing financial flexibility and enabling the expansion of TriNav’s commercial and technology pipeline. The transaction was facilitated by Cantor Fitzgerald & Co. for TriSalus and Covington & Burling LLP for OrbiMed.
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**Categories:** News
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### [Qlaris Bio Secures $24 Million Funding for Revolutionary Glaucoma Drug Development](https://www.clinicaltrialvanguard.com/news/qlaris-bio-secures-24-million-funding-for-revolutionary-glaucoma-drug-development/)
**Published:** May 1, 2024
**Author:** Jon Napitupulu
**Content:**
Qlaris Bio, a biotechnology company specializing in ophthalmic diseases, secured $24 million in Series B financing. The round was co-led by Canaan and New Leaf Venture Partners, with participation from abrdn Inc., Mayo Clinic Ventures, and Correlation Ventures.
The proceeds will support the continued development of [QLS-111](https://www.clinicaltrialvanguard.com/news/qls-111-by-qlaris-bio-enters-phase-ii-trials-for-iop-reduction/), an innovative therapeutic targeting episcleral venous pressure (EVP) to lower intraocular pressure (IOP). QLS-111 is currently undergoing two Phase II clinical trials in the US. Additional trials, including a Phase II study for normal tension [glaucoma](https://www.clinicaltrialvanguard.com/news/nurexone-shows-vision-recovery-in-preclinical-glaucoma-model/) (NTG), are planned for 2024.
QLS-111 aims to address the unmet need for effective treatments in glaucoma, especially NTG, where current medications do not adequately control IOP. EVP is a key determinant of IOP, and QLS-111’s mechanism of action targets EVP to lower IOP.
The science behind QLS-111 is rooted in the research of Dr. Michael Fautsch of Mayo Clinic. The drug relaxes vessels distal to the trabecular meshwork, reducing outflow resistance and EVP. Preliminary data indicate good tolerability and safety.
Qlaris is committed to developing new treatments that address unmet needs in IOP regulation. Thurein Htoo, CEO of Qlaris, expressed gratitude for the investor support, which will enable the company to advance its clinical strategy and bring a novel solution to patients with glaucoma.
Wende Hutton of Canaan believes in Qlaris’s technology, leadership, and scientific expertise. The company’s potential to validate the importance of EVP targeting in QLS-111 trials has garnered investor confidence and anticipation for the upcoming clinical data.
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**Categories:** News
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### [Innovative Tech Solutions for Challenges in Clinical Research](https://www.clinicaltrialvanguard.com/executiveinterviews/innovative-tech-solutions-for-challenges-in-clinical-research/)
**Published:** May 1, 2024
**Author:** Moe Alsumidaie
**Content:**
In this insightful session about challenges in clinical research, I spoke with Aruna Adhikari, Head of Product and Senior Director of Clinical Technology at [IQVIA](https://www.clinicaltrialvanguard.com/news/iqvia-partners-with-medera-on-cardiac-gene-therapy-development/) Technologies. We discussed the innovative strategies and technological advancements that IQVIA has introduced to transform clinical trials, focusing on how these developments enhance data accuracy and improve patient outcomes.
#### [](#moe-can-you-elaborate-on-how-iqvias-technology-solutions-aim-to-revolutionize-clinical-trials)Moe: Can you elaborate on how IQVIA’s technology solutions aim to revolutionize clinical trials?
**Aruna:** IQVIA Technologies develops and delivers more than 20 software-as-a-service solutions that are offered independently from our CRO services. These technology solutions are transforming clinical trials by addressing the challenges that traditionally slow research and development. From planning and start-up through conduct and closeout, our products manage every phase of a trial. These technologies significantly improve important functions such as site selection, patient recruitment, data collection, and financial transactions. By automating and optimizing these processes, we reduce administrative overhead, enhance accuracy, and expedite trial timelines, thereby boosting efficiency and the pace of clinical trials.
Additionally, our technologies shift trials towards more patient-centric practices. We employ patient technologies to enhance patient engagement, simplify participants’ understanding of their roles, and keep them informed. Our product offerings tackle the pervasive issue of data silos by integrating disparate systems into one cohesive structure to enhance the flow of information and support better data management practices, enabling more coherent data analysis and decision-making. By centralizing data, we can facilitate quicker access and more accurate data processing, which is crucial for the timely progression of trials. This comprehensive approach advances medical research and patient welfare and redefines the participant experience, making clinical research more accessible and manageable for all stakeholders.
#### [](#moe-can-you-provide-an-example-of-how-this-technology-improves-day-to-day-operations-at-trial-sites)Moe: Can you provide an example of how this technology improves day-to-day operations at trial sites?
**Aruna:** Aruna: Our new platform, called “One Home for Sites,” significantly eases operational challenges by bringing the clinical trial technology ecosystem together into a single access point. This platform simplifies the work of site managers who oversee dozens of studies and reduces the cognitive load of managing separate logins and interfaces, a common source of inefficiency and frustration at trial sites. Traditional methods require navigating various systems with separate logins and interfaces, which is cumbersome. One Home for Sites offers a single access point to sponsors, studies, and systems. It serves as an aggregator for all study-related tasks, reducing administrative time and addressing inefficiencies in clinical trial management.
Aruna Adhikari, Head of Product and Senior Director of Clinical Technology at IQVIA Technologies
Moreover, when One Home for Sites is used in conjunction with technologies such as the IQVIA Investigator Site Portal, it enhances data entry accuracy and automates routine tasks, reducing errors and improving team communication. Real-time updates and centralized notifications keep everyone aligned, which is crucial for the timely progression of trials. We are laser-focused on alleviating site administrative burden, and One Home for Sites is bringing the industry together to accomplish this goal.
#### [](#moe-turning-our-focus-towards-patients-how-does-your-technology-enhance-their-trial-experience)Moe: Turning our focus towards patients, how does your technology enhance their trial experience?
**Aruna:** Our technology is designed to revolutionize the patient experience in clinical trials, making their journey through the process as seamless and non-intrusive as possible. Firstly, by optimizing technological support for sites, we directly enhance patient care by allowing site personnel to spend less time on administrative tasks and more on direct patient care. Secondly, tools like eConsent and electronic Clinical Outcome Assessments (eCOA) further simplify patient interactions by making it easier for them to provide informed consent and input on clinical outcomes from the comfort of their homes. Integrating these technologies emphasizes our commitment to placing patients at the center of clinical research, valuing their participation, and ensuring a positive experience.
#### [](#moe-with-the-advancement-of-technology-how-do-you-see-the-future-of-clinical-trials-evolving)Moe: With the advancement of technology, how do you see the future of clinical trials evolving?
**Aruna:** The integration of advanced technologies such as generative AI and interconnected data systems is poised to transform clinical trials. Generative AI, in particular, can potentially overhaul clinical trial protocols by predicting outcomes and customizing trial designs based on data-driven insights. An interconnected data ecosystem will enhance data sharing and analysis across platforms, further accelerating the delivery of new therapies by streamlining patient recruitment and trial management. These technologies enhance trial efficiency, accuracy, and quality by enabling sophisticated data analysis and management.
Additionally, a more interconnected data ecosystem will facilitate seamless data sharing across platforms, speeding up the market time for new therapies and enhancing patient recruitment by offering a more personalized trial experience. This shift towards patient-centered trials highlights a move towards more inclusive, efficient, and practical clinical research, promising quicker access to life-saving treatments.
**Categories:** Article: Executive Interviews
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### [Ultrasound Therapy Shows Promise in Relieving Rheumatoid Arthritis](https://www.clinicaltrialvanguard.com/news/ultrasound-therapy-shows-promise-in-relieving-rheumatoid-arthritis/)
**Published:** May 2, 2024
**Author:** Jon Napitupulu
**Content:**
[SecondWave](https://www.clinicaltrialvanguard.com/news/secondwave-systems-secures-3m-additional-financing-to-advance-ultrasound-based-anti-inflammatory-therapy/) Systems has made a breakthrough in the treatment of [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (RA) with their novel ultrasonic stimulation technology. The pilot clinical study involved the noninvasive treatment of 13 participants, significantly improving disease activity.
The wearable MINI™ device delivers ultrasound stimulation to the spleen, effectively reducing inflammation and symptoms. Ten out of 13 participants experienced benefits during the 8-week study period, with positive feedback regarding the treatment experience.
Rheumatologist Dr. Erik Peterson, the principal investigator, emphasized the promising results in that more than two-thirds of the participants showed significant clinical improvement, and the intervention was well-tolerated and safe.
Dr. Hubert Lim, Chief Scientific Officer at SecondWave Systems, highlighted the potential of splenic ultrasound therapy by indicating that this study demonstrates that ultrasound can directly modulate disease symptoms and inflammation in patients with rheumatoid arthritis.
SecondWave Systems is currently developing a randomized controlled trial for at-home treatment of RA using the MINI technology platform. This advancement aims to provide patients with a noninvasive and personalized treatment option for a debilitating condition.
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**Categories:** News
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### [Remepy Unveils $15 Million Investment for Revolutionary Drug](https://www.clinicaltrialvanguard.com/news/remepy-unveils-15-million-investment-for-revolutionary-drug/)
**Published:** May 2, 2024
**Author:** Jon Napitupulu
**Content:**
Remepy, a pioneer in “hybrid drugs,” has secured $15 million in funding, led by NFX and supported by several other investors. Hybrid drugs combine traditional medications with “digital molecules,” therapeutic interventions that use non-invasive cognitive or behavioral techniques to trigger physiological effects.
These digital molecules have been shown to enhance the effectiveness of traditional drugs by altering brain connectivity, blood biomarkers, and behavioral patterns. Remepy has demonstrated the efficacy of its digital interventions in clinical trials using fMRI imaging, blood analyses, and standardized assessments.
Remepy’s innovative approach targets diseases such as neurodegenerative, autoimmune, and degenerative eye conditions. By combining drugs with digital interventions, the company aims to create a new market for hybrid medications and enhance patient outcomes.
Dr. Danny Bar Zohar, Merck KGaA’s Global Head of R&D, has joined Remepy’s Board of Directors. He believes that Remepy’s clinical data provides compelling evidence of how digital interventions can potentiate immunotherapy for cancer and improve drug therapy for neurodegenerative diseases like [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) Disease.
According to Dr. Michal Tsur, Remepy’s Co-CEO, the fusion of digital interventions with traditional products has revolutionized various industries. Co-CEO Or Shoval is particularly enthusiastic about the potential of Remepy’s hybrid approach to alleviate symptoms and potentially modify disease progression in Parkinson’s Disease patients.
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**Categories:** News
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### [Unlock the Secrets of Parkinson's: Cambridge Cognition and Fox Foundation Team Up](https://www.clinicaltrialvanguard.com/news/unlock-the-secrets-of-parkinsons-cambridge-cognition-and-fox-foundation-team-up/)
**Published:** May 2, 2024
**Author:** Jon Napitupulu
**Content:**
Cambridge Cognition, a leader in digital brain health assessment solutions, and The Michael J. Fox Foundation for [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) Disease Research are collaborating on the Parkinson’s Progression Markers Initiative (PPMI).
The PPMI will leverage Cambridge Cognition’s CANTAB cognitive assessments to characterize the cognitive profiles of Parkinson’s Disease patients. This initiative aims to unravel the mechanisms of the disease, trace its origins, and develop potential interventions to halt its progression.
PPMI includes individuals diagnosed with Parkinson’s disease within two years of symptom onset, offering a window into its early stages and neurological changes. CANTAB assessments will be utilized to assess 5,000 patients, providing valid and objective insights into their cognitive functions. These user-friendly assessments capture impairments in attention, executive function, memory, and visuospatial skills.
CANTAB assessments have been extensively utilized in Parkinson’s disease clinical trials, serving as targets for treatment development and evaluating new drug safety and efficacy. Cambridge Cognition’s CEO, Matthew Stork, expressed pride in the collaboration, highlighting the sensitivity of CANTAB assessments to disease progression.
Cambridge Cognition offers digital health solutions to [advance brain health](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/) understanding and treatment. Their products assist in clinical trial optimization, early disease detection, and healthcare efficiency improvement.
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**Categories:** News
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### [NIAID Awards $14M for Institutional Review Services to Advarra](https://www.clinicaltrialvanguard.com/news/niaid-awards-14m-for-institutional-review-services-to-advarra/)
**Published:** May 2, 2024
**Author:** Jon Napitupulu
**Content:**
The National Institutes of Health (NIH) has chosen Advarra as its exclusive single institutional review board (sIRB) for clinical trials. This multi-year contract is valued at over $13 million.
Advarra’s expertise in project management and administrative oversight for sIRBs, coupled with its robust digital platform and extensive experience, were key factors in the decision.
Advarra’s leadership in research review solutions and clinical research technology has earned the trust of top global biopharma sponsors, CROs, and site investigators. Its solutions streamline trial operations, enhance patient safety, and accelerate clinical research while ensuring compliance.
The FDA’s anticipated mandate for sIRBs in multi-center clinical trials and the NIH’s existing sIRB policy have driven the adoption of sIRBs. Advarra’s selection as the NIH’s sIRB for NIAID is a testament to its capabilities.
Throughout its 35-year history, Advarra has consistently exceeded expectations in providing central IRB services. Its commitment to quality, scalability, and innovative technology has ensured the successful oversight of over 27,000 studies. Advarra played a vital role in Operation Warp Speed, providing IRB services for critical [COVID](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) research.
With this contract, the NIH acknowledges Advarra’s exceptional expertise and reinforces the importance of sIRBs in streamlining clinical trials and protecting human subjects. Advarra’s experience and innovative solutions will continue to support the NIH’s mission to advance medical research and improve public health.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [MCL Treatment Revolution: Calquence Powers Progression-Free Life](https://www.clinicaltrialvanguard.com/news/mcl-treatment-revolution-calquence-powers-progression-free-life/)
**Published:** May 6, 2024
**Author:** Jon Napitupulu
**Content:**
A promising new treatment option has emerged for patients with [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL), a rare and aggressive type of blood cancer. The ECHO Phase III clinical trial demonstrated significant benefits of CALQUENCE (acalabrutinib) when combined with standard chemotherapy (bendamustine and rituximab).
CALQUENCE, a Bruton tyrosine kinase (BTK) inhibitor, showed a statistically significant improvement in progression-free survival (PFS) compared to the current standard of care. PFS refers to the period during which the cancer remains controlled.
Encouragingly, a trend was observed favoring CALQUENCE plus chemoimmunotherapy for overall survival (OS), a crucial measure of treatment effectiveness. The OS data will continue to be assessed as the trial progresses.
MCL is characterized by the mutation of B-lymphocytes in the mantle zone of lymph nodes. It is often diagnosed at an advanced stage, affecting over 27,500 patients worldwide.
Dr. Michael Wang, the trial’s principal investigator, emphasized the potential of the positive PFS results to redefine the standard of care for MCL patients. By incorporating CALQUENCE into first-line treatment, patients could benefit from its efficacy and safety profile.
Susan Galbraith, Executive Vice President of Oncology R&D at AstraZeneca, highlighted the significant impact of these findings. They demonstrate the potential of CALQUENCE to delay disease progression and extend survival in MCL patients.
The safety profile of CALQUENCE was consistent with previous observations, with no new safety concerns identified.
AstraZeneca is actively investigating CALQUENCE both alone and in combination for various B-cell blood cancers, including chronic lymphocytic leukemia, MCL, and diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/). CALQUENCE has been used by over 80,000 patients worldwide and is approved for the treatment of CLL and MCL in multiple regions.
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**Categories:** News
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### [Unlock the Doors of Health Equity: Walgreens and Boehringer Ingelheim's Partnership](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/)
**Published:** May 6, 2024
**Author:** Jon Napitupulu
**Content:**
Boehringer Ingelheim and Walgreens have joined forces to revolutionize clinical trials. Through this collaboration, people with [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), overweight, and [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) will have access to a Phase III clinical trial in a convenient setting – Walgreens pharmacies.
This initiative aims to remove obstacles, promote equity, and enhance the representation of diverse communities in clinical trials. Black and Hispanic adults, who are disproportionately affected by obesity, have historically been underrepresented.
Walgreens’ Chief Clinical Trials Officer, Ramita Tandon, emphasizes the significance of this collaboration in advancing community health and innovation. The model empowers individuals by providing education on clinical research and offering a novel avenue for active participation in their healthcare journey.
Boehringer Ingelheim’s partnership with EmVenio Research further extends the reach of this initiative. Mobile research units will bring clinical trials directly to local communities, providing additional options for participation.
Lennart Jungersten, Senior Vice President of Medicine and Regulatory Affairs at Boehringer Ingelheim U.S., indicated that they are making clinical trials more accessible, helping to provide access to diverse populations with pressing health needs when bringing clinical trials to the communities.
Obesity affects over 1 billion people worldwide, and projections indicate that by 2035, approximately 24% of the global population will be affected. By leveraging the expertise of pharmacies and mobile research units, this collaboration aims to transform the clinical research landscape, making it more inclusive and equitable while paving the way for transformative healthcare solutions for future generations.
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**Categories:** News
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### [BMS Submits EMA Application for Opdivo and Yervoy for mCRC](https://www.clinicaltrialvanguard.com/news/bms-submits-ema-application-for-opdivo-and-yervoy-for-mcrc/)
**Published:** May 7, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) recently submitted an application to the European Medicines Agency (EMA) for the use of the immunotherapy combination of Opdivo (nivolumab) and Yervoy ([ipilimumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/)) as a first-line treatment for adult patients with MSI-H or dMMR metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC).
The EMA’s acceptance of this application initiates the centralized review process. The application is primarily based on data from the [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -8HW study, which demonstrated statistically significant improvements in progression-free survival (PFS) when Opdivo plus Yervoy was used compared to chemotherapy.
MSI-H or dMMR mCRC patients typically do not respond well to chemotherapy, so the outcome of the CheckMate -8HW study highlights the potential of the Opdivo and Yervoy combination to offer a much-needed treatment option for these patients.
The safety profile of the immunotherapy combination was consistent with previous studies and manageable with established protocols. No new safety concerns were identified.
BMS acknowledges the contributions of patients and investigators involved in the CheckMate -8HW clinical trial. The study is still ongoing to evaluate the long-term effectiveness of Opdivo plus Yervoy in this patient population.
Source link: [http://www.businesswire.com/news/home/20240503820656/en/European-Medicines-Agency-Validates-Bristol-Myers-Squibb%E2%80%99s-Application-for-Opdivo-nivolumab-Plus-Yervoy-ipilimumab-for-the-First-Line-Treatment-of-Adult-Patients-with-Microsatellite…](http://www.businesswire.com/news/home/20240503820656/en/European-Medicines-Agency-Validates-Bristol-Myers-Squibb%E2%80%99s-Application-for-Opdivo-nivolumab-Plus-Yervoy-ipilimumab-for-the-First-Line-Treatment-of-Adult-Patients-with-Microsatellite...)
**Categories:** News
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### [Cybin Unveils Strategies For CYB003 & CYB004 Psychedelic Drug Development](https://www.clinicaltrialvanguard.com/news/cybin-unveils-strategies-for-cyb003-cyb004-psychedelic-drug-development/)
**Published:** May 7, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin Inc., a biopharmaceutical company specializing in psychedelic-based mental health treatments, has secured $150 million in funding to advance its clinical-stage programs, CYB003 and [CYB004](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/).
CYB003, a deuterated psilocybin analog, has received Breakthrough Therapy Designation (BTD) from the FDA for the adjunctive treatment of [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD). This designation recognizes CYB003’s potential to significantly improve treatment outcomes based on preliminary findings. A Phase 3 MDD study is expected to commence mid-2024.
CYB004, a deuterated DMT program, is being developed for Generalized Anxiety Disorder (GAD). Results from a Phase 2 study are anticipated in Q4 2024 and will assess the drug’s efficacy, onset time, and durability of effects.
Cybin’s pipeline also includes programs targeting treatment-resistant depression and obsessive-compulsive disorder. The company holds a substantial intellectual property portfolio, including over 50 granted and pending patents.
Cybin’s CEO, Doug Drysdale, emphasized the growing need for improved mental health treatments and the company’s commitment to addressing the mental health crisis. He highlighted the recent BTD for CYB003 and the initiation of the Phase 2 study for CYB004 as key milestones.
The funding round provides financial support for Cybin’s ongoing clinical programs and further development efforts. The company anticipates a transformative year ahead, with the initiation of the CYB003 Phase 3 trial and the release of CYB004 Phase 2 topline data. Cybin remains focused on developing innovative psychedelic-based therapies to revolutionize the treatment of mental health disorders.
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**Categories:** News
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### [AI Predicts Metastasis Risk in Prostate Cancer: Revolutionary Biomarker](https://www.clinicaltrialvanguard.com/news/ai-predicts-metastasis-risk-in-prostate-cancer-revolutionary-biomarker/)
**Published:** May 7, 2024
**Author:** Jon Napitupulu
**Content:**
[ArteraAI](https://www.clinicaltrialvanguard.com/news/arteraai-prostate-test-now-accessible-in-ca/) has announced the validation of its AI-based digital pathology biomarker for predicting metastasis risk in [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) patients who have undergone radical prostatectomy and subsequently experienced biochemical recurrence (BCR). This biomarker will help clinicians determine the optimal treatment plan for these patients.
After a radical prostatectomy, about 20-40% of patients develop BCR, indicating a risk of cancer spread. Salve radiotherapy is typically recommended, and clinicians may also consider adding hormone therapy.
Researchers developed the biomarker using multimodal deep learning on digital histopathology data from over 1,800 radical prostatectomy patients. The model successfully estimated metastasis risk and identified patients who would benefit significantly from hormone therapy and salvage radiotherapy.
Dr. Todd Morgan indicated that this biomarker will empower clinicians and patients to better understand disease progression risk and guide treatment decisions.
This biomarker represents a significant advancement as the first AI-based model developed specifically for radical prostatectomy patients with BCR. It demonstrated prognostic capabilities and the ability to predict differential benefits of hormone therapy addition.
Andre Esteva, Co-founder and CEO of ArteraAI, indicated that their digital pathology platform is expanding to support patients who have undergone radical prostatectomy.
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**Categories:** News
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### [Neurogene Unveils Remarkable Trial Results in Rett Syndrome](https://www.clinicaltrialvanguard.com/news/neurogene-unveils-remarkable-trial-results-in-rett-syndrome/)
**Published:** May 8, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene Inc. has reported positive initial safety and tolerability data from its ongoing Phase 1/2 gene therapy trial for Rett syndrome. The treatment, [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/), was well-tolerated in the first three patients dosed, with follow-up periods ranging from three to nine months.
NGN-401 incorporates Neurogene’s EXACTTM technology, aiming to provide therapeutic levels of protein expression without overexpression toxicity. Despite one patient having a mild variant predicted to result in residual MeCP2 expression, no signs or symptoms of overexpression-related toxicity were observed in any patient.
The Phase 1/2 trial is evaluating the safety and preliminary efficacy of two dose levels of NGN-401 delivered via a single intracerebroventricular infusion in female patients aged 4-10 years with classic Rett syndrome. All adverse events related to NGN-401 have been mild and transient, with no serious or treatment-emergent adverse events reported.
Rachel McMinn, PhD, Founder and CEO of Neurogene, indicated that these data demonstrate a favorable tolerability profile in the first three pediatric patients, including one with a mild variant predicted to result in residual MeCP2 expression, and that they anticipate sharing interim efficacy data for the first cohort in the fourth quarter of 2024.
The promising safety and tolerability data support the potential of NGN-401 as a treatment for Rett syndrome, addressing the urgent need for therapies that target the underlying cause of the disease and improve the quality of life for patients and their families.
Source link:
**Categories:** News
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### [Lokavant Expands Team to Enhance AI-Driven Clinical Trial Feasibility Solution](https://www.clinicaltrialvanguard.com/news/lokavant-expands-team-to-enhance-ai-driven-clinical-trial-feasibility-solution/)
**Published:** May 8, 2024
**Author:** Jon Napitupulu
**Content:**
Lokavant, a clinical trial intelligence platform, has strengthened its team with the appointment of three new Advisory Board members and a new executive.
The Advisory Board includes industry leaders like Pfizer’s Head of Predictive Analytics, Jonathan Crowther, who provide insights into the data and analytics needs of clinical trial sponsors and contract research organizations (CROs).
Aaron Mackey, PhD, a former Covance executive, joins as senior vice president of AI and data science. Mackey brings expertise in artificial intelligence (AI) and data science to Lokavant, instrumental in advancing its technology and deriving novel insights.
Lokavant’s Clinical Intelligence Platform utilizes data and predictive analytics to optimize clinical research outcomes. Despite the growing role of AI and increased data availability, [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) face challenges harnessing data effectively for practical AI applications.
Lokavant’s platform addresses this issue by aggregating and harmonizing data from multiple sources, enabling data scientists to analyze it effectively. This facilitates the identification of patterns and insights, empowering sponsors and CROs to make proactive decisions.
Lokavant’s platform and predictive analytics address the challenges in the clinical research industry, leveraging data and AI to improve efficiency and accelerate the delivery of new therapies to patients.
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**Categories:** News
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### [PLK Targeted Therapies Market Boasts Promise in Cancer and Beyond](https://www.clinicaltrialvanguard.com/news/plk-targeted-therapies-market-boasts-promise-in-cancer-and-beyond/)
**Published:** May 8, 2024
**Author:** Jon Napitupulu
**Content:**
Polo-like kinases (PLKs) are key players in cellular processes like cell cycle regulation and DNA damage response. Their dysregulation in various diseases, particularly cancer, makes them promising therapeutic targets.
The global PLK targeted therapies market is nascent but rapidly evolving, with several pharmaceutical companies investing in research. These therapies aim to inhibit PLKs, leading to cell cycle arrest and apoptosis in cancer cells.
Emerging evidence also implicates PLKs in immune function and inflammation, suggesting potential applications in autoimmune diseases. Additionally, PLKs are involved in viral replication, hinting at antiviral effects of PLK inhibitors.
Small molecule inhibitors of PLKs are at the forefront of development, with Onvansertib being the most advanced in clinical trials for [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/). Other candidates like RP-1664 and BAL0891 are being explored for solid tumors, while CFI-400945 targets hematological cancers.
Beyond small molecule inhibitors, antisense oligonucleotides and PROTACs are being investigated as alternative PLK targeting strategies. While challenges exist in their clinical implementation, ongoing research continues to refine these approaches.
The growing interest and confidence in PLK targeted therapies stem from their potential to treat a wide range of diseases, including cancer and autoimmune disorders. As research progresses, the pipeline of PLK inhibitors and other therapeutic modalities is expected to expand, opening new avenues for patient care.
Source link: [http://www.businesswire.com/news/home/20240507249579/en/PLK-Targeted-Therapies-Market-Clinical-Trials-2024-Onvansertib-Leading-the-Way-in-PLK1-Inhibition-Pioneering-Breakthroughs-in-Small-Cell-Lung-Cancer-and-Chronic-Myelomonocytic-Leukemia-Treatment—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240507249579/en/PLK-Targeted-Therapies-Market-Clinical-Trials-2024-Onvansertib-Leading-the-Way-in-PLK1-Inhibition-Pioneering-Breakthroughs-in-Small-Cell-Lung-Cancer-and-Chronic-Myelomonocytic-Leukemia-Treatment---ResearchAndMarkets.com)
**Categories:** News
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### [Merck's KEYNOTE-B21 Trial: Failed To Meet Primary Endpoint For Endrometrial Cancer](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)
**Published:** May 13, 2024
**Author:** Jon Napitupulu
**Content:**
Merck’s Phase 3 KEYNOTE-B21 trial has shown that its anti-PD-1 therapy, [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/), in combination with chemotherapy, did not improve disease-free survival (DFS) in high-risk endometrial cancer patients after surgery. An interim analysis revealed that KEYTRUDA plus chemotherapy, with or without radiotherapy, did not meet the study’s criteria for DFS compared to placebo plus chemotherapy. Therefore, the study’s other primary endpoint, overall survival (OS), was not formally tested.
The safety profile of KEYTRUDA remained consistent with previous studies, without any new safety concerns. Merck is thoroughly reviewing the data and will share the results with the scientific community. Despite this setback, Merck remains committed to exploring the potential of KEYTRUDA in endometrial cancer.
Merck has several ongoing clinical trials evaluating KEYTRUDA-based combinations and investigational candidates in endometrial and gynecologic malignancies. Two approved indications for KEYTRUDA in endometrial cancer in the U.S. include its use in combination with LENVIMA for advanced MSI-H or dMMR endometrial carcinoma, and as a single agent for advanced endometrial carcinoma that is pMMR or non-MSI-H.
Merck’s clinical development program in endometrial carcinoma continues to progress, with ongoing trials evaluating KEYTRUDA in various treatment regimens. The FDA has granted priority review for a supplemental [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) based on the NRG-GY018/KEYNOTE-868 trial results, with a target action date of June 21, 2024.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Kite and Arcellx Advance Multiple Myeloma Program with Anti-BCMA CAR-T Momentum](https://www.clinicaltrialvanguard.com/news/kite-and-arcellx-advance-multiple-myeloma-program-with-anti-bcma-car-t-momentum/)
**Published:** May 13, 2024
**Author:** Jon Napitupulu
**Content:**
Arcellx and Kite have partnered on the development of [anitocabtagene](https://www.clinicaltrialvanguard.com/news/arcellx-announces-positive-data-for-relapsed-multiple-myeloma-from-imagine-1-study-at-ash-meeting/) [autoleucel](https://www.clinicaltrialvanguard.com/news/carsgens-satricabtagene-autoleucel-approved-for-gastric-cancer-in-china/) (anito-cel), a BCMA CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for multiple myeloma. The companies have designed a global Phase 3 trial, iMMagine-3, to evaluate the efficacy and safety of anito-cel in patients who have received prior treatment with an immunomodulatory drug and an anti-CD38 monoclonal antibody.
The trial will compare anito-cel to the standard of care in patients with relapsed and/or refractory multiple myeloma who have received one to three prior lines of therapy. Kite’s manufacturing facility in Frederick, Maryland will produce anito-cel for the trial, following the completion of a technical transfer from a third-party contract manufacturing organization.
Arcellx’s Chairman and Chief Executive Officer, Rami Elghandour, stated that the iMMagine-3 trial will target a large patient population with a significant unmet need. The completion of the technical transfer to Kite has accelerated the development program and enabled broader patient access to anito-cel.
Cindy Perettie, Executive Vice President of Kite, emphasized the importance of manufacturing quality and speed for patients with relapsed and/or refractory multiple myeloma. Kite’s manufacturing expertise will support anito-cel’s position as a potential best-in-class cell therapy.
iMMagine-3 is expected to begin in the second half of 2023. Preliminary data from the iMMagine-1 trial is anticipated by the end of the year. Arcellx and Kite remain committed to advancing anito-cel as a promising treatment option for patients with multiple myeloma.
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**Categories:** News
**Tags:** eClinical Tech News
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### [AC Immune and Takeda: A Remarkable Agreement for Alzheimer's Therapy](https://www.clinicaltrialvanguard.com/news/ac-immune-and-takeda-a-remarkable-agreement-for-alzheimers-therapy/)
**Published:** May 14, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda Pharmaceutical Company and AC Immune have entered into an exclusive global option and licensing agreement for AC Immune’s active immunotherapies, including ACI-24.060, which targets [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease.
ACI-24.060 is designed to trigger an immune response against toxic amyloid beta (Abeta) forms, which are linked to plaque formation and Alzheimer’s progression. By clearing plaques and inhibiting their formation, it aims to delay or slow disease progression.
AC Immune will conduct the ongoing Phase 1b/2 ABATE trial to evaluate ACI-24.060’s safety, efficacy, and immunogenicity in individuals with prodromal Alzheimer’s disease and adults with Down syndrome.
Upon Takeda’s exercise of the option, the company will assume responsibility for further clinical development, regulatory matters, and worldwide commercialization of ACI-24.060. Takeda’s expertise in neuroscience drug development and commercialization complements AC Immune’s experience in active immunotherapy approaches.
AC Immune will receive an upfront payment of $100 million and is eligible for an option exercise fee and potential milestone payments up to approximately $2.1 billion. This agreement enables AC Immune to focus on completing Phase 1b/2 development and advance its early-stage pipeline while Takeda brings its resources to support Phase 3 trials and the potential commercialization of ACI-24.060.
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**Categories:** News
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### [Merck Terminates Investigational Melanoma Treatment Arm](https://www.clinicaltrialvanguard.com/news/merck-terminates-investigational-melanoma-treatment-arm/)
**Published:** May 14, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has terminated a clinical trial arm combining [vibostolimab](https://www.clinicaltrialvanguard.com/news/merck-unveils-groundbreaking-escc-trial-results-unlocking-new-hope-for-patients/) and [pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) for treating resected high-risk melanoma. The decision was made after an analysis revealed a higher discontinuation rate for the coformulation arm due to immune-related events, diminishing the likelihood of a significant improvement in the primary endpoint of recurrence-free survival (RFS). The independent Data Monitoring Committee recommended unblinding the study and offering patients receiving coformulation monotherapy with pembrolizumab.
Despite this setback, Merck remains committed to developing novel treatments for melanoma, particularly in earlier stages. The company’s oncology pipeline includes the ongoing Phase 3 V940-001 trial investigating V940 (mRNA-4157), an individualized neoantigen therapy, in combination with pembrolizumab as adjuvant treatment for high-risk melanoma.
Vibostolimab, Merck’s investigational anti-[TIGIT](https://www.clinicaltrialvanguard.com/news/anti-tigit-antibody-the-revolutionary-cancer-treatment-you-must-know-about/) antibody, aims to boost antitumor activity by blocking the TIGIT receptor and activating T lymphocytes. Merck’s clinical development program for the vibostolimab and pembrolizumab coformulation encompasses over 3,000 patients.
Phase 3 studies of the co-formulation in lung cancer are ongoing, including KeyVibe-003, KeyVibe-006, KeyVibe-007, and KeyVibe-008. External data monitoring committees routinely monitor these studies, and interim safety reviews have not raised significant concerns.
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**Categories:** News
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### [Can-Fite BioPharma Proceeds with Phase 3 Liver Cancer Trial](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/)
**Published:** May 14, 2024
**Author:** Jon Napitupulu
**Content:**
Can-Fite BioPharma has secured approval from the FDA and EMA for a pivotal Phase 3 clinical trial for the treatment of advanced [liver cancer](https://www.clinicaltrialvanguard.com/clinops-watchdog/three-strikes-same-deficiency-how-elevar-therapeutics-turned-a-solvable-manufacturing-problem-into-a-patient-crisis/). The trial is now underway in Israel, Europe, and the US and aims to enroll patients who have not responded to other approved therapies.
The drug being evaluated in the trial, namodenoson, has both Orphan Drug and Fast Track Status with the FDA for treating hepatocellular carcinoma (HCC). It is designed to bind to the A3 adenosine receptor (A3AR), which is highly expressed in diseased but not normal cells.
A conference was held to present the study protocol and procedures to oncologists and coordinators involved in the trial. Dr. Lencioni, a renowned radiologist, discussed the methodology for measuring tumor response to namodenoson. US experts guided the electronic data collection system used for patient data during the trial.
Namodenoson has shown promising results in previous Phase II trials as a second-line treatment for HCC, NAFLD, and NASH. Its favorable safety profile stems from its selective binding to A3AR, predominantly found in diseased cells.
The global incidence of liver cancer is significant, with HCC being a particularly aggressive form with low survival rates. As new and effective treatments emerge, the market for HCC therapies is projected to expand.
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**Categories:** News
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### [Medidata: Enhancing Trials, Transforming Patient Care Worldwide](https://www.clinicaltrialvanguard.com/news/medidata-enhancing-trials-transforming-patient-care-worldwide/)
**Published:** May 15, 2024
**Author:** Jon Napitupulu
**Content:**
Medidata, a leading clinical trial solutions provider, has partnered with Worldwide Clinical Trials (Worldwide) to leverage Medidata AI. This collaboration aims to enhance trial planning and facilitate data-driven decision-making across Worldwide’s operations.
By utilizing Medidata AI’s capabilities, Worldwide can systematically plan, design, and execute research. This enables them to reduce trial timelines and improve site selection, ultimately benefiting patients and sponsors.
Medidata AI’s expansive historical data set and artificial intelligence solutions provide Worldwide with real-time, actionable insights into site-level granularity. These insights allow Worldwide to accelerate clinical research and advance the [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) industry.
“We’ve partnered with Medidata for over a decade to provide our customers with data-driven insights for informed decisions about clinical trials,” said Peter Benton, President and CEO of Worldwide Clinical Trials. “The integration of Medidata AI Intelligent Trials into our studies will further enhance our ability to plan, design, and execute research for optimal patient and sponsor outcomes.”
Tom Doyle, Chief Technology Officer of Medidata, emphasized that Worldwide will leverage Medidata AI’s turnkey solution to accelerate clinical research and advance the life sciences industry by leveraging site-level granularity and real-time actionable observations.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Alto Neuroscience's Astonishing Financial Results and Breakthrough Highlights](https://www.clinicaltrialvanguard.com/news/alto-neurosciences-astonishing-financial-results-and-breakthrough-highlights/)
**Published:** May 15, 2024
**Author:** Jon Napitupulu
**Content:**
Alto Neuroscience has initiated a Phase 2 study of ALTO-203, a PDE4 inhibitor targeted at patients with [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD) and anhedonia. This follows positive Phase 1 data for the transdermal formulation of ALTO-101. The improved drug delivery system significantly increased exposure while reducing tolerability issues compared to oral administration.
Phase 2b studies for ALTO-100 and ALTO-300, both targeting MDD, also progress steadily. ALTO-100 is a first-in-class small molecule that enhances neural plasticity, while ALTO-300 is an adjunctive treatment for patients with insufficient response to antidepressants.
Alto’s financial position remains strong, with approximately $206 million expected to support operations into 2027.
•ALTO-100: Ongoing Phase 2b study in MDD patients with a cognitive biomarker. Topline data is expected in the second half of 2024.
•ALTO-300: Ongoing Phase 2b study in MDD patients with an EEG biomarker. Topline data is expected in the first half of 2025.
•ALTO-101: Novel PDE4 inhibitor in Phase 1 development for CIAS. Positive Phase 1 results demonstrated improved drug exposure and tolerability. A proof-of-concept study in [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) is planned.
Alto Neuroscience remains dedicated to developing personalized therapies for brain disorders. The company’s commitment is evident in its advancements across its clinical pipeline and its ongoing execution of clinical trials. Alto anticipates reporting results from its depression studies in the coming year and looks forward to the potential transformative impact of its treatments for patients in need.
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**Categories:** News
---
### [Moffitt Cancer Center and Fulgent Pharma Unite to Unveil Nanotherapeutics Cancer Therapies](https://www.clinicaltrialvanguard.com/news/moffitt-cancer-center-and-fulgent-pharma-unite-to-unveil-nanotherapeutics-cancer-therapies/)
**Published:** May 15, 2024
**Author:** Jon Napitupulu
**Content:**
Moffitt Cancer Center and Fulgent Pharma have formed a partnership to accelerate the development of personalized cancer therapies.
• Moffitt provides Fulgent access to clinical expertise and resources, prioritizing Fulgent’s clinical pipeline.
• Fulgent contributes its nanotherapeutics and genomics platforms.
• Co-development of personalized treatment options based on Moffitt’s scientific and immunology expertise, as well as Fulgent’s nano-particle platform and genetic testing capabilities.
The collaboration aims to transform patient care by unlocking precision medicine advancements. By tailoring therapies to individual patient needs, the partnership seeks to improve treatment outcomes and enhance the quality of life for cancer patients.
Fulgent has transformed paclitaxel, a chemotherapy drug with poor solubility, into a soluble form ([FID-007](https://www.clinicaltrialvanguard.com/news/fulgent-data-uncovering-fid-007-in-head-and-neck-cance/)) suitable for intravenous injection. FID-007 has shown promising results in Phase 1 clinical trials, with significant tumor reduction observed in various cancer types.
Moffitt’s strengths in clinical trials, patient screening, and data sharing support the rapid advancement of Fulgent’s clinical pipeline. The institution’s broad scientific knowledge and access to tissue samples enhance the co-development of personalized treatments.
The Moffitt-Fulgent partnership combines cutting-edge clinical capabilities with innovative nanotechnology and genomics to accelerate the development of precision oncology therapies. By leveraging their complementary strengths, the organizations aim to improve patient outcomes and bring lifesaving treatments closer to patients in need.
Source link: http://www.businesswire.com/news/home/20240514935253/en/Moffitt-Cancer-Center-and-Fulgent-Pharma-Join-Forces-to-Revolutionize-Cancer-Therapeutics
**Categories:** News
**Tags:** eClinical Tech News
---
### [Breyanzi FDA Approved: A Remarkable New Hope for Follicular Lymphoma Patients](https://www.clinicaltrialvanguard.com/news/breyanzi-fda-approved-a-remarkable-new-hope-for-follicular-lymphoma-patients/)
**Published:** May 16, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb’s Breyanzi has received accelerated FDA approval for adult patients with relapsed or refractory follicular [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) who have undergone at least two prior therapies.
Breyanzi is a personalized therapy that uses chimeric antigen receptor (CAR) T cells to target CD19, a protein found in lymphoma cells. The treatment involves a single infusion of CAR-positive T cells directly into the patient’s bloodstream.
In the Phase 2 TRANSCEND FL clinical trial, 95.7% of patients responded to Breyanzi, with a median duration of response that was not yet reached. At 18 months, 77.1% of responders maintained an ongoing response.
Breyanzi offers several advantages over other treatments. It provides durable responses with a consistent safety profile across clinical trials. Additionally, its one-time infusion allows for administration and monitoring in inpatient and outpatient settings.
Historically, follicular lymphoma has been considered incurable, and patients often relapse after initial treatment. Breyanzi addresses the need for more effective and long-lasting therapies for this condition. Targeting CD19 offers a potential for complete remission and extended treatment-free intervals.
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**Categories:** News
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### [Alpha Cognition: Surprising Results Unlock New Opportunities](https://www.clinicaltrialvanguard.com/news/alpha-cognition-surprising-results-unlock-new-opportunities/)
**Published:** May 16, 2024
**Author:** Jon Napitupulu
**Content:**
Alpha Cognition Inc., a biopharmaceutical company focused on neurodegenerative disorders, has shared its financial results and updated it for the first quarter of 2024.
The company’s lead drug, ALPHA-1062, is undergoing review by the FDA for mild-to-moderate [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. Alpha Cognition is actively planning for its commercialization and manufacturing, anticipating a PDUFA date of July 2024. If approved, ALPHA-1062 is expected to offer a unique treatment option for Alzheimer’s patients.
ALPHA-1062 is a patented acetylcholinesterase inhibitor designed with minimal gastrointestinal side effects. Its active metabolite interacts with neuronal nicotinic receptors, particularly the alpha-7 subtype, which has positive cognitive effects. The drug is also being developed in combination with memantine for moderate to severe Alzheimer’s disease, a sublingual formulation for dysphagia patients, and an intranasal formulation for cognitive impairment related to mTBI (concussion).
Alpha Cognition is dedicated to developing treatments for neurodegenerative diseases with limited options. The company believes ALPHA-1062 has the potential to make a meaningful difference in the lives of patients with Alzheimer’s dementia. Forward-looking statements made in this release involve risks and uncertainties that could impact the company’s results and the development of its products.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Sotyktu's Power: Long-Lasting Relief in Plaque Psoriasis](https://www.clinicaltrialvanguard.com/news/sotyktus-power-long-lasting-relief-in-plaque-psoriasis/)
**Published:** May 20, 2024
**Author:** Jon Napitupulu
**Content:**
New data revealed that after four years of continuous treatment, over 70% of patients with [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/) achieved a 75% reduction in disease severity, known as PASI 75, in a long-term extension trial of Sotyktu. Similarly, around 57% achieved clear or almost clear skin, based on the static Physician’s Global Assessment (sPGA).
The safety profile of Sotyktu remained consistent throughout the four years, with no new safety concerns identified. In the extension trial, 513 patients received continuous Sotyktu treatment and showed sustained clinical efficacy. PASI 75 response rates stayed above 70%, PASI 90 response rates remained around 47%, and sPGA 0/1 response rates were over 57% at the end of year four.
The safety analysis included over 1,500 patients who received Sotyktu for at least four years. The incidence rates of adverse events, serious adverse events, and discontinuations due to adverse events decreased or remained stable compared to the first year of treatment. The rates of herpes zoster, malignancies, major adverse cardiovascular events, venous thromboembolism, and deaths remained low and consistent throughout the four years.
These findings suggest that Sotyktu is a safe and effective long-term treatment for adults with moderate-to-severe plaque psoriasis, providing sustained disease control and a favorable safety profile.
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**Categories:** News
---
### [AI-Powered Cell Simulations Soar in Biopharma with Accenture's Latest Investment](https://www.clinicaltrialvanguard.com/news/ai-powered-cell-simulations-soar-in-biopharma-with-accentures-latest-investment/)
**Published:** May 20, 2024
**Author:** Jon Napitupulu
**Content:**
Accenture Ventures, the venture capital arm of Accenture, has invested in Turbine, a predictive simulation company developing a platform for understanding human biology. This strategic investment aims to enhance Turbine’s capabilities for global biopharma companies.
Turbine’s central technology, the Simulated Cell™ platform, leverages machine learning to decipher decision-making processes in human cells. It models molecular interactions within and around cells, enabling massive virtual experiments that reveal disease mechanisms and therapy responses.
Advanced technologies and digital capabilities are increasingly differentiating the biopharma industry, with AI playing a pivotal role in drug discovery. Turbine’s platform has proven its ability to unlock valuable biological insights for biopharma clients.
Pharmaceutical leaders collaborate with Turbine to identify promising drug targets, select patient populations for therapies, and determine optimal combination therapies. This enables the discovery of previously overlooked treatments and the early identification of potential clinical trial failures.
Petra Jantzer, global lead of Accenture’s [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) business, indicated that Turbine’s platform is a significant tool for biopharma companies to unravel the complexities of biological systems, develop targeted treatments, and advance AI-based drug discovery, ultimately aiming to enhance patient therapies.
Szabolcs Nagy, co-founder and CEO of Turbine, indicated that the heterogeneity of complex diseases poses challenges for AI in drug discovery and that their Simulated Cells offer a scalable means to represent disease heterogeneity, guiding researchers toward the most effective experiments.
By leveraging Accenture’s expertise, Turbine aims to expand its market reach and enhance its simulation platform. This collaboration will contribute to the overall advancement of the biopharma industry, ensuring efficient and effective drug development.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Tezspire's Potential Role in Chronic Obstructive Pulmonary Disease: Latest ATS Findings Revealed](https://www.clinicaltrialvanguard.com/news/tezspires-potential-role-in-chronic-obstructive-pulmonary-disease-latest-ats-findings-revealed/)
**Published:** May 20, 2024
**Author:** Jon Napitupulu
**Content:**
A Phase IIa trial, known as COURSE, has assessed the effectiveness of TEZSPIRE ([tezepelumab](https://www.clinicaltrialvanguard.com/news/tezspire-meets-co-primary-endpoints-in-phase-3-waypoint-trial/)) in reducing COPD exacerbations in patients with varying eosinophil counts. TEZSPIRE showed a notable reduction in exacerbations among patients with higher eosinophil counts.
Overall, TEZSPIRE led to a 17% numerical decrease in exacerbations compared to placebo, although it was not statistically significant. In patients with eosinophil counts above 150 cells/µL, TEZSPIRE resulted in a 37% reduction in exacerbations, while those with eosinophil counts above 300 cells/µL experienced a 46% reduction.
Subgroup analysis revealed improved lung function and quality of life in patients receiving TEZSPIRE, particularly those with higher eosinophil counts. Safety and tolerability were comparable to TEZSPIRE’s approved severe [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/) indication, with worsening COPD and [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) infections being the most common adverse events.
These findings hold promise for the potential of TEZSPIRE in managing COPD across a wide range of patients, irrespective of emphysema, chronic bronchitis, or smoking history. Phase III trials are underway to evaluate TEZSPIRE’s efficacy in COPD further.
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**Categories:** News
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### [Terran Biosciences Unveils Remarkable Breakthrough: TERXT for Schizophrenia](https://www.clinicaltrialvanguard.com/news/terran-biosciences-unveils-remarkable-breakthrough-terxt-for-schizophrenia/)
**Published:** May 21, 2024
**Author:** Jon Napitupulu
**Content:**
Terran Biosciences has developed TerXT, a combination of novel prodrugs of xanomeline and trospium, for treating [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/). The prodrug approach aims to overcome the limitations of xanomeline and trospium, enabling fixed-dose combinations for once-daily oral administration and long-acting intramuscular injections.
Terran’s team of experts designed over 10,000 prodrugs using medicinal chemistry and PK modeling to optimize pharmacokinetic parameters and ratios. Preclinical testing involved synthesizing over 500 prodrugs and conducting 650 in vitro and in vivo studies.
Clinical trials of an oral, twice-daily combination of xanomeline and trospium have shown safety and efficacy in treating schizophrenia, and the combination is currently under FDA review for 505(b)(1) approval.
If the FDA approves the initial combination, Terran plans to pursue the 505(b)(2) approval pathway for TerXT and TerXT LAI, potentially bringing these long-acting formulations to market approximately five years after the first xanomeline/trospium approval.
The accelerated approval pathway will utilize pharmacokinetic bridging studies and leverage existing safety and efficacy data from completed trials to expedite the approval process. This approach aims to broaden patient access, improve the use profile, and enhance the affordability of these next-generation therapeutics.
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**Categories:** News
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### [Pathai's AISight Breaks Barriers, Empowering Pathologists with Next-Gen Image Management](https://www.clinicaltrialvanguard.com/news/pathais-aisight-breaks-barriers-empowering-pathologists-with-next-gen-image-management/)
**Published:** May 22, 2024
**Author:** Jon Napitupulu
**Content:**
[PathAI](https://www.clinicaltrialvanguard.com/news/roche-to-acquire-pathai-for-750m-in-ai-pathology-deal/), a leader in AI-powered pathology, launches an Early Access Program (EAP) for its AISight Image Management System (IMS) in Europe. This program provides labs access to advanced digital pathology solutions and AI capabilities.
AISight is a cloud-based platform that streamlines end-to-end digital pathology workflows. It enhances case management, viewing, collaboration, and integration with laboratory information systems.
Pathologists can seamlessly access a range of AI algorithms through AISight to optimize workflows, quantify biomarkers, and prioritize cases. These algorithms include ArtifactDetect, TumorDetect, AIM-TumorCellularity, AIM-PD-L1, and AIM-HER2. AISight also supports integrations with third-party algorithms.
The EAP allows European labs to trial AISight’s GDPR-compliant digital pathology solution and conduct validation studies on PathAI’s AI products. Labs of all sizes and specialties can benefit from AISight, including health systems, university hospitals, and reference laboratories.
Nick Brown, Chief Growth Officer at PathAI, indicated that they are excited to partner with European labs to democratize digital pathology access and aim to enhance the pathologist experience and improve patient outcomes.”
Prof. Dr. Jan Budczies of the University of Heidelberg highlights the potential of AISight to revolutionize digital pathology workflows. He emphasizes the importance of understanding tumor cell content and immune cell infiltrates using digital pathology and AI.
PathAI’s commitment to innovation is evident in AISight’s growing portfolio of features and algorithms. By providing access to advanced digital pathology solutions, AISight empowers pathology labs to embrace cutting-edge technologies and drive progress in patient care.
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**Categories:** News
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### [Foundation Medicine Launches Groundbreaking RNA Sequencing Test, FoundationOne®RNA, in the U.S.](https://www.clinicaltrialvanguard.com/news/foundation-medicine-launches-groundbreaking-rna-sequencing-test-foundationonerna-in-the-u-s/)
**Published:** May 22, 2024
**Author:** Jon Napitupulu
**Content:**
Foundation Medicine has launched FoundationOne®RNA, an RNA sequencing test that complements its DNA-based FoundationOne®CDx. This combination provides enhanced fusion detection in cancer, particularly in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC), [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/), and other solid tumors.
RNA sequencing detects fusions in 318 genes, expanding the company’s portfolio of genomic profiling tests. By offering both DNA and RNA testing options, Foundation Medicine aims to increase confidence in fusion detection, given the instability of RNA.
The FoundationOne RNA test results are combined with FoundationOne CDx results in a single report. This report provides clinicians with actionable information to guide targeted therapies and consider clinical trials.
Courtney Granville, Chief Scientific Officer at GO2 for Lung Cancer, indicated that fusion detection using RNA is crucial for lung cancer treatment planning.
Foundation Medicine’s coextraction method for FoundationOne CDx allows for the extraction of DNA and RNA from a single sample, minimizing tissue volume requirements. This method simplifies the testing process without affecting turnaround times.
With FoundationOne RNA, Foundation Medicine now offers a comprehensive suite of tests for various cancer types. These tests empower clinicians with data to make informed decisions about patient care and improve treatment outcomes.
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**Categories:** News
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### [Unlock the Extraordinary: AngioDynamics' AlphaVac F18⁸⁵ Revolutionizes Vascular Health](https://www.clinicaltrialvanguard.com/news/unlock-the-extraordinary-angiodynamics-alphavac-f18⁸⁵-revolutionizes-vascular-health/)
**Published:** May 22, 2024
**Author:** Jon Napitupulu
**Content:**
[AngioDynamics](https://www.clinicaltrialvanguard.com/news/angiodynamics-initiates-landmark-trial-for-pulmonary-embolism-treatment/), Inc. has obtained CE Mark approval in Europe for its [AlphaVac](https://www.clinicaltrialvanguard.com/news/fda-grants-510k-clearance-to-angiodynamics-alphavac-f1885-system-for-pe-treatment/) F1885 System, enabling the non-surgical removal of blood clots from pulmonary arteries and the treatment of pulmonary embolism (PE).
This approval marks a significant advancement in patient care and safety for endovascular therapies within the European Union, which experiences a higher prevalence of PE compared to the United States. With an estimated 435,000 PE events occurring annually in the EU’s six most prominent countries, the AlphaVac F1885 System offers healthcare providers an additional tool.
The system aids in reducing blood clot burden and improving right ventricular function in PE patients. Clinical trial results from the APEX-AV study demonstrated a substantial reduction in the clot burden and a favorable Major Adverse Event rate. The mean procedure time was approximately 37 minutes.
The AlphaVac F1885 System consists of an ergonomic handle, an 18F cannula with an 85-degree angle, an obturator, and a waste bag assembly. AngioDynamics continues to innovate and expand its global reach, providing cutting-edge solutions to combat PE and enhance patient outcomes.
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**Categories:** News
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### [Gilead: Insights for Liver Disease at EASL 2024](https://www.clinicaltrialvanguard.com/news/gilead-insights-for-liver-disease-at-easl-2024/)
**Published:** May 23, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences announced significant research findings to be presented at the European Association for the Study of the Liver (EASL) Congress in 2024.
Primary Biliary Cholangitis (PBC)
• Long-term data from the Phase 3 ASSURE study will demonstrate the sustained effectiveness and safety of seladelpar for treating PBC.
• The study includes individuals who received a second year of seladelpar after participating in the RESPONSE study.
• Seladelpar targets biochemical responses (e.g., reduced liver enzymes) and pruritus (itching) in PBC patients.
Hepatitis Delta Virus (HDV)
• Gilead will present data from the Phase 2b MYR204 study, showing the efficacy of [bulevirtide](https://www.clinicaltrialvanguard.com/news/gilead-announces-breakthrough-hdv-treatment-data-in-new-england-journal-of-medicine/) with or without PegIFN in HDV-infected individuals.
• The Phase 3 MYR301 study, which assesses the effectiveness and safety of bulevirtide as a monotherapy for HDV, will be unveiled.
Hepatitis C Virus (HCV)
• To support the WHO’s goal of eliminating HCV as a public health threat, Gilead will present real-world data and launch a national awareness program in Italy.
• The “Epatite C Mettiamoci un Punto” campaign aims to raise awareness and encourage HCV testing through the “Love Your Liver” Campaign.
Gilead’s research encompasses viral and inflammatory liver diseases, underscoring its dedication to improving patient outcomes and raising awareness.
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**Categories:** News
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### [Middle East Clinical Trial Regulatory Update: Pioneering Frameworks](https://www.clinicaltrialvanguard.com/conference-coverage/middle-east-clinical-trial-regulatory-update-pioneering-frameworks/)
**Published:** May 28, 2024
**Author:** Moe Alsumidaie
**Content:**
The Middle East Town Hall at DIA Europe 2024 showcased significant Middle East clinical trial regulatory advancements across the region, with representatives from Saudi Arabia, Egypt, Jordan, and Qatar detailing their strategies to enhance healthcare outcomes. Each country highlighted its unique approaches to aligning with international standards, leveraging reliance practices, and implementing digital solutions to streamline regulatory processes. These efforts underscore the region’s commitment to ensuring medical products’ safety, efficacy, and quality, ultimately improving patient access to essential treatments.
### [](#sfdas-strategic-vision-and-regulatory-reforms-aligning-with-saudi-vision-2030)SFDA’s Strategic Vision and Regulatory Reforms: Aligning with Saudi Vision 2030
Bandar Al Hammad, Chief Pharmacist at the Saudi Food and Drug Authority (SFDA), detailed Saudi Arabia’s regulatory advancements aligned with the ambitious Saudi Vision 2030. This vision aims to diversify the economy and develop public services, including healthcare. SFDA has enhanced the regulatory framework to ensure medical products’ safety, efficacy, and quality. The strategy involves extensive benchmarking against global standards, including adopting guidelines from the International Council for Harmonisation (ICH). For example, by implementing ICH E6(R2) guidelines on Good Clinical Practice, the SFDA has reinforced Middle East clinical trial standards, ensuring ethical conduct and reliable data.
Bandar emphasized SFDA’s implementation of work-sharing and reliance practices, particularly within the Gulf Cooperation Council (GCC) regulatory network. This collaboration allows SFDA to leverage regulatory assessments from other member states, such as the rapid approval of products evaluated by the United Arab Emirates, expediting patient access to new treatments. SFDA’s milestones include streamlined drug approval procedures, enhanced pharmacovigilance systems, and continuous staff training. Collaborations with organizations like the WHO and PIC/S enable SFDA to adopt global best practices. Looking ahead, SFDA aims to integrate advanced data analytics and AI to enhance regulatory reviews, improving decision-making and risk mitigation. Through strategic vision, global benchmarking, and innovative practices, SFDA continues to advance healthcare outcomes in Saudi Arabia.
### [](#transforming-drug-regulation-in-egypt-edas-journey-towards-sustainability)Transforming Drug Regulation in Egypt: EDA’s Journey Towards Sustainability
Hamada Sherief, Director of the General Administration of Registration of Pharmaceuticals at the Egyptian Drug Authority (EDA), detailed Egypt’s transformative journey towards sustainable drug regulation post-[COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/). The pandemic highlighted the need for a flexible regulatory approach, prompting the EDA to enhance its frameworks for better responsiveness. A key innovation is the reliance model for market authorization and post-approval changes, allowing the EDA to use assessments from trusted international regulatory authorities like the US FDA or EMA to expedite approvals in Egypt. Additionally, the EDA streamlined post-approval changes, swiftly evaluating modifications in manufacturing processes, labeling updates, and new clinical data to maintain drug availability and compliance with safety standards.
The EDA has collaborated extensively with international regulatory bodies and industry stakeholders to support these initiatives, resulting in practical and effective guidelines. In April 2023, the EDA launched new guidelines for post-approval changes, incorporating feedback from over 50 industry meetings. This led to user-friendly, rigorous guidelines. The EDA also invested in digital solutions, creating a platform for pharmaceutical companies to submit variations electronically and streamlining the process. These efforts improved regulatory efficiency, with some product approvals taking as little as four months. Looking ahead, the EDA plans to enhance its reliance models and digital platforms further to keep Egypt at the forefront of regulatory innovation, ensuring timely access to high-quality medicines for patients.
### [](#jfdas-strategic-vision-and-regulatory-reforms-enhancing-healthcare-in-jordan)JFDA’s Strategic Vision and Regulatory Reforms: Enhancing Healthcare in Jordan
Reem Al-Naimat, Head of Manufacturing Sites Accreditation Unit at the Jordan Food and Drug Administration (JFDA), outlined Jordan’s strategic vision to provide high-quality, affordable medicines while maintaining medical security. Central to this vision is achieving WHO maturity Level 3, indicating a robust regulatory system that ensures medical products’ safety, efficacy, and quality. To reach this goal, the JFDA implemented the WHO Global Benchmarking Tool (GBT), conducting a thorough self-assessment and formal evaluation by WHO experts. This review aligned JFDA’s practices with international standards, enhancing the credibility of Jordan’s regulatory system.
Reem emphasized the benefits of regulatory harmonization and work-sharing within the region. The JFDA participates in the Arab Regulators Network, promoting regulatory convergence and leveraging collective expertise. This collaboration enabled the JFDA to introduce accelerated registration procedures for essential medicines, vaccines, and treatments for chronic diseases, ensuring timely access to necessary treatments. The JFDA plans to strengthen its regulatory framework further through continuous collaboration with international bodies like the WHO and regional partners and by expanding digital technologies to streamline processes and improve transparency.
### [](#advancing-regulatory-standards-in-qatar-cross-referencing-and-digitalization)Advancing Regulatory Standards in Qatar: Cross-Referencing and Digitalization
Ahmed M Hussein Babiker, Head of the Drug Registration & Pricing Section at the Ministry of Public Health in Qatar, discussed the nation’s efforts to align with international regulatory standards through cross-referencing and reliance practices. By leveraging assessments from established authorities like the US FDA and EMA, Qatar expedites the approval process for new drugs, ensuring high safety, efficacy, and quality standards. During the COVID-19 pandemic, Qatar utilized reliance practices to quickly approve vaccines, relying on international evaluations to ensure timely public access. This approach was crucial for rapid response and effective public health protection.
Ahmed also emphasized the importance of digitalization in streamlining regulatory processes. The Ministry has developed an online platform for drug application submissions, tracking, and evaluations, significantly reducing approval times. This portal allows pharmaceutical companies to submit documentation, improving efficiency and transparency electronically. A key goal for Qatar is to achieve WHO maturity level recognition, which indicates a robust regulatory system. To support this, the Ministry has invested in extensive training and capacity-building programs for its staff. Plans include ongoing collaboration with international bodies like the WHO and regional partners, further enhancing Qatar’s regulatory framework and contributing to improved healthcare standards across the region.
### [](#overcoming-challenges-in-implementing-reliance-and-post-approval-changes)Overcoming Challenges in Implementing Reliance and Post-Approval Changes
During the session, panelists discussed the challenges Middle East clinical trial regulatory bodies face in implementing reliance and post-approval changes. Discrepancies in documentation between regulatory authorities and pharmaceutical companies often arise due to varying requirements across jurisdictions. For example, a drug dossier approved by the European Medicines Agency (EMA) might not align with Middle East clinical trial standards, causing delays. To address these gaps, the Saudi Food and Drug Authority (SFDA) implemented a verification pathway to standardize documentation, while the Egyptian Drug Authority (EDA) developed digital tools to help companies comply with local requirements.
Another challenge is the need for continuous updates to regulatory guidelines to keep pace with scientific advancements and best practices. The Jordan Food and Drug Administration (JFDA) established a task force to regularly review and update its guidelines in collaboration with international experts. Harmonization efforts like the Arab Regulators Network facilitate the sharing of best practices and assessments among member countries. During the COVID-19 pandemic, this network enabled rapid information exchange, leading to quicker vaccine approvals. Future improvements include investing in digitalization, enhancing staff training, and strengthening international collaborations.
#### [](#summary)Summary
In conclusion, the collaborative efforts and innovative practices highlighted by the Saudi Arabia, Egypt, Jordan, and Qatar regulatory bodies are paving the way for improved healthcare outcomes across the Middle East. These countries are enhancing their regulatory frameworks by addressing challenges such as documentation discrepancies and the need for continuous guideline updates, as well as investing in digitalization and international collaborations. The progress in implementing reliance models, accelerated registration procedures, and robust digital platforms exemplify the region’s dedication to advancing regulatory standards, ensuring timely access to high-quality medicines, and ultimately fostering better health for their populations.
**Categories:** Article: Conference Coverage
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### [Fulgent Announces Clinical Data Presentation for Lead Oncology Candidate at ASCO 2024](https://www.clinicaltrialvanguard.com/news/fulgent-announces-clinical-data-presentation-for-lead-oncology-candidate-at-asco-2024/)
**Published:** May 28, 2024
**Author:** Jon Napitupulu
**Content:**
Fulgent Pharma, a subsidiary of Fulgent Genetics, will present Phase 1 clinical data on [FID-007](https://www.clinicaltrialvanguard.com/news/fulgent-data-uncovering-fid-007-in-head-and-neck-cance/), its lead therapeutic candidate for head and neck cancer, at the American Society for Clinical Oncology (ASCO) Annual Meeting in Chicago on June 2, 2024.
FID-007 is a nanoparticle formulation of paclitaxel designed to improve drug delivery and tolerability. The formulation employs a polyethyloxazoline (PEOX) polymer excipient, allowing the drug to remain in solution and preferentially target tumor cells through hyperpermeable blood vessels.
Fulgent Pharma emerged from Fulgent LLC in 2016 and was acquired by Fulgent Genetics in 2022. Today, the company focuses solely on developing innovative cancer therapeutics. To advance its research, Fulgent Pharma collaborates with leading institutions such as the University of Southern California, Moffitt Cancer Center, and ANP Technologies.
The FID-007 clinical data will be presented in a poster session on June 2, 2024, from 9:00 a.m. to 12:00 p.m. Central Time. The presentation title is “Efficacy from the phase 1 study of FID-007, a novel nanoparticle paclitaxel formulation, in patients with head and neck squamous cell carcinoma.” The abstract number is 6042, and the poster board number is 345.
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**Categories:** News
**Tags:** eClinical Tech News
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### [SpyBiotech and Oxford Team Up to Conquer Epstein-Barr Virus](https://www.clinicaltrialvanguard.com/news/spybiotech-and-oxford-team-up-to-conquer-epstein-barr-virus/)
**Published:** May 28, 2024
**Author:** Jon Napitupulu
**Content:**
SpyBiotech and The University of Oxford have entered a research agreement to develop a vaccine against Epstein-Barr virus (EBV). EBV is a widespread virus that can cause various health conditions, including infectious mononucleosis, and is associated with certain cancers and [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/).
The collaboration will leverage Oxford’s academic research expertise and SpyBiotech’s proprietary SPYVLP vaccine platform. The project aims to advance three vaccine candidates targeting EBV and conduct a Phase I clinical trial.
Mark Leuchtenberger, CEO of SpyBiotech, indicated that the research is crucial for addressing a highly prevalent virus where no vaccines or therapeutics currently exist and that EBV is linked to severe health conditions, and they believe their SPYVLP platform can provide an effective solution.
SpyBiotech’s SPYVLP platform utilizes a protein “superglue” technology to bind antigens to vaccine delivery platforms. This approach minimizes delivery risks while enhancing immunogenicity and efficacy.
Oxford researchers will have access to the SPYVLP platform and will work towards Phase I clinical trials. “We are eager to progress these vaccine candidates into Phase I trials based on promising pre-clinical data,” said Sumi Biswas, President and CSO of SpyBiotech.
SpyBiotech is currently conducting a Phase I trial for its HCMV vaccine to assess safety and immunogenicity. The company also has a diverse pipeline targeting other infectious diseases, such as respiratory syncytial virus (RSV) and cytomegalovirus (CMV).
EBV affects a vast majority of the population and can have serious consequences for some individuals. This collaboration between SpyBiotech and Oxford University has the potential to make significant strides in preventing and treating EBV-related illnesses.
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**Categories:** News
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### [Devoted to Amyloidosis: AstraZeneca's Heartfelt Commitment at ISA 2024](https://www.clinicaltrialvanguard.com/news/devoted-to-amyloidosis-astrazenecas-heartfelt-commitment-at-isa-2024/)
**Published:** May 28, 2024
**Author:** Jon Napitupulu
**Content:**
[AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) and Alexion will present at the International Symposium on Amyloidosis (ISA) 2024, showcasing studies on their amyloidosis treatment pipeline.
Key findings include subgroup analyses from the NEURO-TTRansform study of WAINUATM (eplontersen), which the FDA approved for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis.
Clinical data will also be presented on ALXN2220 and anselamimab, undergoing Phase III trials for ATTR and light chain (AL) amyloidosis.
AstraZeneca emphasizes its commitment to advancing amyloidosis research and improving patient outcomes. Alexion highlights the significance of its pipeline and ongoing efforts to develop multifaceted treatments for halting or reversing organ damage.
Additional presentations include live-cell imaging and Phase I data on ALXN2220’s ability to remove cardiac amyloid, supporting its potential use in advanced ATTR cardiomyopathy. Findings on epidemiology and patient renal outcomes will underscore the need for improved diagnosis and treatment options in AL amyloidosis.
AstraZeneca’s evidence program provides insights into patient characteristics and treatment effectiveness, including data on US patient characteristics and the MaesTTRo study design for assessing real-world effectiveness of treatments for ATTR.
WAINUA is also being evaluated in the CARDIO-TTRANSform study for adults with cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM), a progressive and fatal condition. The Phase 3 study, with over 1,400 patients, is the largest in this patient population.
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**Categories:** News
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### [Merck: Phase 3 Trial Success for TNBC Patients](https://www.clinicaltrialvanguard.com/news/merck-phase-3-trial-success-for-tnbc-patients/)
**Published:** May 29, 2024
**Author:** Jon Napitupulu
**Content:**
[KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) (pembrolizumab) has demonstrated promising results in the treatment of high-risk early-stage triple-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) (TNBC) in the Phase 3 [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-522 trial.
KEYTRUDA, in combination with chemotherapy as pre-operative treatment followed by single-agent use after surgery, significantly improved overall survival (OS) compared to pre-operative chemotherapy alone. This landmark achievement marks the first immunotherapy-based regimen to show a statistically significant OS benefit in this patient population.
The positive OS results add to the previously reported pathological complete response and event-free survival data from the KEYNOTE-522 trial. KEYTRUDA’s safety profile remained consistent with previous studies.
This OS benefit adds to a growing body of evidence supporting KEYTRUDA’s effectiveness in earlier stages of cancer. KEYNOTE 522 is the fourth trial to demonstrate an OS benefit with KEYTRUDA-based regimens in early-stage settings, following studies in cervical cancer, non-small cell lung cancer, and renal cell carcinoma.
In the United States, KEYTRUDA is approved for two indications in TNBC: as part of neoadjuvant and adjuvant treatment for high-risk early-stage TNBC and in combination with chemotherapy for locally recurrent unresectable or metastatic TNBC with PD-L1 expression.
Merck’s ongoing clinical development program continues to explore KEYTRUDA’s role in various breast cancer subtypes, with studies evaluating its use in adjuvant treatment, high-risk early-stage estrogen receptor-positive breast cancer, and unresectable locally advanced or metastatic ER+/HER2- breast cancer.
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**Categories:** News
---
### [AI Validation in Massive Bio Clinical Trial: A Revolutionary Breakthrough](https://www.clinicaltrialvanguard.com/news/ai-validation-in-massive-bio-clinical-trial-a-revolutionary-breakthrough/)
**Published:** May 29, 2024
**Author:** Jon Napitupulu
**Content:**
The Precision Cancer Consortium (PCC) and [Massive Bio](https://www.clinicaltrialvanguard.com/news/massive-bio-advancing-cancer-care-with-ai-driven-clinical-trials/) have conducted a comprehensive study on the efficacy of Massive Bio’s artificial intelligence (AI) system for clinical trial matching. The study evaluated the system’s ability to refine the matching process using real-world clinical and genomic data for a multi-study oncology platform.
Typically, screening patients for cancer trials involves manual labor, extending the process to approximately 25 minutes per trial. This exhaustive process restricts patient access to suitable trials and hinders enrollment rates. To address this challenge, Massive Bio and PCC introduced a multi-trial matching method that incorporates Next-Generation Sequencing (NGS) results and AI to enhance accuracy and efficiency, primarily for targeted therapies.
Massive Bio’s AI system leverages computer vision and natural language processing to extract structured clinical parameters from medical records. This system harnesses the power of GPT-4 Large Language Model and has been specifically optimized for oncology and biomarker-specific scenarios. Using an AI-driven recommendation algorithm, it matches patients to inclusion/exclusion criteria for over 14,000 active cancer trials.
The study’s findings were compelling. The integration of NGS and AI in the multi-trial matching framework significantly increased patient eligibility for various tumor types, doubling the potential enrollment pool. Furthermore, it boosted matching efficiency by twelvefold for specific tumor profiles, greatly reducing manual intervention.
This study’s success demonstrates AI’s transformative potential in streamlining the clinical trial matching process. It reduces the time and resources required, enabling greater patient access to potentially life-saving therapies. Future endeavors aim to expand this analysis nationwide to validate these promising results further.
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**Categories:** News
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### [ViVani Medical Unveils Groundbreaking Weight Loss Solution for Pets with OKV-119: A Life-Changing Innovation](https://www.clinicaltrialvanguard.com/news/vivani-medical-unveils-groundbreaking-weight-loss-solution-for-pets-with-okv-119-a-life-changing-innovation/)
**Published:** May 29, 2024
**Author:** Jon Napitupulu
**Content:**
A study published in BMC Veterinary Research demonstrates the effectiveness of OKV-119, a miniature drug implant containing exenatide, in reducing weight in obese cats.
This implant leverages Vivani Medical’s patented NanoPortal technology, allowing long-term medication delivery through a subdermal implant. The exenatide drug is known for regulating blood sugar levels and promoting weight loss.
The study found that cats implanted with OKV-119 experienced significant weight reduction compared to controls. The implant’s sustained release of exenatide over 84 days effectively suppressed appetite and increased energy expenditure.
Feline [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) is a significant health concern, affecting up to 40% of domestic cats. This condition is associated with numerous health problems, including diabetes, heart disease, and joint pain.
The development of OKV-119 offers a promising therapeutic option for feline obesity. The implant’s convenient administration and extended duration of action make it a potential breakthrough in reducing animal obesity and improving overall feline health.
Researchers anticipate extending the implant’s duration to six months in future studies. This extended duration would further enhance the implant’s practicality and effectiveness in treating feline obesity in the long term.
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**Categories:** News
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### [Revolutionary FDA Clearance Paves Way for Game-Changing Hemophilia B Therapy](https://www.clinicaltrialvanguard.com/news/revolutionary-fda-clearance-paves-way-for-game-changing-hemophilia-b-therapy/)
**Published:** May 29, 2024
**Author:** Jon Napitupulu
**Content:**
Be Biopharma, Inc., a pioneer in developing Engineered B Cell Medicines (BCMs), has received IND clearance from the FDA for BE-101, a revolutionary treatment for [hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/) B. This Phase 1/2 clinical trial, BeCoMe-9, will evaluate BE-101’s safety and efficacy in adults with hemophilia B. The trial is expected to begin in late 2024.
BE-101 is an autologous BCM designed to insert the human FIX gene into patients’ B cells, enabling them to produce FIX. This one-time infusion has the potential to provide sustained FIX activity, reducing the frequency of dosing compared to current therapies. The study will assess the safety and effectiveness of BE-101 in improving bleeding control and joint damage.
Joanne Smith-Farrell, CEO of [Be Bio](https://www.clinicaltrialvanguard.com/news/be-bio-fda-grants-new-designation-for-novel-medicine-hemophilia-b-breakthrough/), indicated that the IND clearance marks a significant milestone in our mission to provide an innovative treatment for individuals with hemophilia B and that BE-101 has the potential to transform patient care by offering long-lasting FIX protection and reducing the burden of infusions.
Hemophilia B is a bleeding disorder that can lead to severe consequences, including chronic pain and joint damage. Current treatments require frequent infusions to maintain FIX levels, but BE-101 aims to provide a more durable solution.
Preclinical studies have shown that a single dose of BE-101 can deliver sustained FIX levels and stably engraft in bone marrow tissue. Additionally, BE-101’s redosability allows for further adjustments in FIX levels as needed.
If proven safe and effective in adults, BE-101 could also revolutionize treatment for children, potentially mitigating the long-term consequences of hemophilia B and improving their quality of life.
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**Categories:** News
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### [Pfizer's Lorbrena® CROWN Study: Remarkable Milestone for Lung Cancer Patients](https://www.clinicaltrialvanguard.com/news/pfizers-lorbrena-crown-study-remarkable-milestone-for-lung-cancer-patients/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
A recent Phase 3 CROWN trial has demonstrated the long-term effectiveness of LORBRENA in treating ALK-positive advanced [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) (NSCLC). After five years of follow-up, patients treated with LORBRENA experienced a remarkable 81% reduction in disease progression or death compared to those receiving XALKORI.
Significantly, 60% of LORBRENA recipients remained alive without disease progression after five years, while only 8% of XALKORI achieved this milestone. These findings highlight LORBRENA’s potential as a standard treatment for ALK-positive advanced NSCLC.
LORBRENA’s efficacy in targeting tumor mutations and penetrating the blood-brain barrier is a breakthrough. Compared to XALKORI, it has significantly reduced the risk of intracranial disease progression by 94%. Among patients without brain metastases at the start of treatment, only a small number developed brain metastases within the first 16 months of LORBRENA use, compared to a higher incidence in the XALKORI group.
The improved outcomes with LORBRENA represent a significant advancement in lung cancer treatment. It offers patients with ALK-positive advanced NSCLC a better chance of long-term survival and reduced disease progression, including in the brain.
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**Categories:** News
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### [Study Results in Metastatic NSCLC Presented at ASCO 2024: Astonishing Breakthrough](https://www.clinicaltrialvanguard.com/news/study-results-in-metastatic-nsclc-presented-at-asco-2024-astonishing-breakthrough/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
The [EVOKE-01](https://www.clinicaltrialvanguard.com/news/gilead-evoke-01-phase-3-study-critical-update-issued/) study investigated Trodelvy against docetaxel in patients with advanced [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) that had progressed after standard treatments.
The study revealed a 16% reduction in mortality risk for patients treated with Trodelvy compared to docetaxel (median overall survival: 11.1 vs. 9.8 months). This improvement was observed across all disease types.
A significant OS benefit was observed in patients whose tumors did not respond to previous anti-PD-(L)1 therapy, with a 3.5-month improvement in median OS (11.8 vs. 8.3 months). This subgroup accounted for approximately two-thirds of the study participants.
In patients whose tumors responded to previous anti-PD-(L)1 therapy, median OS was longer with docetaxel (10.6 months) than with Trodelvy (9.6 months).
The study also demonstrated a lower incidence of severe side effects and treatment discontinuations with Trodelvy than docetaxel. The most common side effects of Trodelvy included fatigue, diarrhea, and hair loss.
These findings suggest that Trodelvy could be a valuable second-line treatment option for patients with advanced NSCLC who have progressed on prior therapies, especially those whose tumors are resistant to anti-PD-(L)1 therapy.
Further research is needed to confirm these findings and explore the optimal use of Trodelvy in treating advanced NSCLC.
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**Categories:** News
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### [Calidi Biotherapeutics $2.1 Million Warrant Exercise](https://www.clinicaltrialvanguard.com/news/calidi-biotherapeutics-2-1-million-warrant-exercise/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/), a biotechnology company focusing on targeted immunotherapies, announced the exercise of warrants for purchasing over 10.6 million shares of its common stock at a reduced price of $0.20. The warrants were initially issued in April 2024 and had terms of 12 months and four months, respectively. The company anticipates approximately $2.1 million in proceeds from the warrant exercise, excluding fees and expenses.
In conjunction with the warrant exercise, Calidi issued new warrants with an exercise price of $0.30 per share, exercisable upon shareholder approval for five and a half years. Additionally, Series B-1 and C-1 warrants were issued, each with an exercise price of $0.20 per share and expiring in five years.
The funds will support Calidi’s clinical and pre-clinical programs, operating expenses, and working capital. Subject to customary conditions, the offering is expected to close by June 3, 2024.
The new warrants and underlying shares are offered in a private placement and have not been registered under relevant regulations. Calidi intends to file a registration statement covering the resale of shares upon warrant exercise, and the offering is not intended for sale or solicitation in any jurisdiction where such actions are prohibited before proper registration or qualification.
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**Categories:** News
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### [Artera Unveils Extraordinary AI Cancer Platform, Unveiling the Future of Cancer Treatment](https://www.clinicaltrialvanguard.com/news/artera-unveils-extraordinary-ai-cancer-platform-unveiling-the-future-of-cancer-treatment/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
Artera’s multimodal artificial intelligence (MMAI) platform has demonstrated notable prognostic value in prostate and early-stage [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). Studies presented at ASCO 2024 highlighted the platform’s ability to provide personalized therapy recommendations based on image analysis of hematoxylin and eosin (H&E)-stained pathology slides.
Traditionally, personalized cancer treatment plans required multiple tests, which can be costly and inefficient. Artera’s AI-enabled algorithms overcome this challenge by extracting valuable information from digitized H&E slides. This analysis aids clinicians and patients in making informed treatment decisions.
According to Trevor Royce, Senior Medical Director at Artera, the platform’s versatility in analyzing different patient cohorts suggests the reliability of H&E slide analysis in tailoring treatment.
Andre Esteva, CEO of Artera, emphasizes the groundbreaking potential of this approach. He highlights how AI and image analysis can accelerate innovation and advance cancer care.
Artera’s model leverages clinical data and pathology slide images to provide actionable insights. By combining these sources, Artera aims to:
• Enhance treatment personalization
• Minimize unnecessary treatment side effects
• Increase confidence in decision-making
The platform’s scalability allows for widespread application, facilitating the development of novel biomarkers and tailored treatment strategies for various cancer stages. As treatment options expand, individualizing patient care becomes increasingly important. Artera’s multimodal AI platform is poised to revolutionize precision oncology, providing patients with optimal and informed treatment journeys.
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**Categories:** News
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### [FDA Approves BMS's Revolutionary CAR-T Therapy for Cancer](https://www.clinicaltrialvanguard.com/news/fda-approves-bmss-revolutionary-car-t-therapy-for-cancer/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb has garnered FDA approval for Breyanzi, a CAR-T cell therapy for relapsed or refractory [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL). This marks the fourth subtype of non-Hodgkin lymphoma treatable with Breyanzi, making it the most widely applicable CAR T cell therapy for B-cell malignancies.
Breyanzi requires only one infusion of CAR-positive viable T cells for MCL treatment. In clinical trials, it demonstrated high response rates with a consistent safety profile.
MCL is an aggressive lymphoma with limited treatment options. With each relapse, the prognosis worsens, making deep and durable responses challenging. Breyanzi offers a significant advancement for these patients, providing a potentially curative option.
Dr. Michael Wang of the University of Texas MD Anderson Cancer Center emphasizes the importance of this approval, given the limited treatment options for relapsed or refractory MCL. He highlights the high response rates and consistent safety profile of Breyanzi, which are crucial for patients facing the challenges of this aggressive disease.
The approval is based on the MCL cohort of the TRANSCEND NHL 001 trial, which demonstrated a response rate of 85.3% with a one-time infusion. Breyanzi’s expanded indications include diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/), follicular lymphoma, and high-grade B-cell lymphoma.
Bristol Myers Squibb’s commitment to cell therapy innovation continues with Breyanzi, offering personalized and potentially definitive treatment for various B-cell malignancies.
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**Categories:** News
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### [Unlock the Power of AI for Life Science: Paige's Revolutionary Approach](https://www.clinicaltrialvanguard.com/news/unlock-the-power-of-ai-for-life-science-paiges-revolutionary-approach/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
Paige, a digital pathology and AI leader, has introduced a service line powered by its Foundation Models. These models include the world’s largest multi-modal AI model for pathology and oncology. AI developers, organizations building computational pathology products, and [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) companies can license and utilize these models to create their AI models for various purposes.
Paige Foundation Models eliminate the need for task-specific models, saving time and resources. They can adapt to various tasks without requiring individual training. Customers will have access to advanced pre-trained models, including Virchow, the largest image-based AI model for cancer detection, and PRISM, a multi-modal model with reporting and generative capabilities.
PRISM offers deep specificity and accuracy, allowing AI teams to analyze advanced cancer and rare biomarker detection, cellular subtyping, spatial biology, and therapy response prediction. Paige provides support and services to ensure partners maximize the potential of the Foundation Models.
Razik Yousfi, Paige’s Senior Vice President of Technology, indicated that their Foundation Models empower pharmaceutical companies with AI capabilities, revolutionizing drug discovery and development, and that licensees can reduce development time, build innovative AI applications, and enhance existing applications.
Andy Moye, Paige’s CEO, suggested that their goal is precision oncology, matching patients with the right treatments, and that their Foundation Models enable life sciences companies to advance drug discovery and improve treatment outcomes.
Paige uses AI to transform pathology. The company is committed to democratizing AI and empowering partners to innovate and advance cancer care.
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**Categories:** News
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### [Incyte Acquires Escient Pharmaceuticals for Uncommon Progress](https://www.clinicaltrialvanguard.com/news/incyte-acquires-escient-pharmaceuticals-for-uncommon-progress/)
**Published:** June 3, 2024
**Author:** Jon Napitupulu
**Content:**
Incyte has strengthened its Inflammation and Autoimmunity pipeline by acquiring Escient Pharmaceuticals. The acquisition brings [EP262](https://www.clinicaltrialvanguard.com/news/incytes-acquisition-unlocking-a-promising-pipeline-of-groundbreaking-medicines/) and EP547, two oral MRGPR antagonists, into Incyte’s portfolio.
EP262 targets MRGPRX2, a receptor found on mast cells. By blocking its activation, EP262 aims to treat mast cell-mediated diseases such as chronic inducible urticaria, chronic spontaneous urticaria, and [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/). Preclinical studies suggest its potential to alleviate symptoms associated with these conditions.
EP547, on the other hand, is designed to inhibit MRGPRX4, a receptor involved in severe pruritus. Its potential applications include treating cholestatic pruritus and other conditions characterized by extreme itching.
Incyte’s commitment to addressing unmet patient needs drives this acquisition. The company believes that Escient’s innovative candidates have the potential to revolutionize the treatment of inflammatory diseases.
The acquisition demonstrates Incyte’s recognition of the value of Escient’s research and development efforts. Escient’s team had significantly advanced in understanding MRGPR biology and developing novel therapies. The transition to Incyte positions these therapies to reach a broader patient population globally, enabling access to potential treatments that can alleviate suffering and improve quality of life.
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**Categories:** News
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### [Thermo Fisher Unveils New Facility Enriching Advanced Therapies Progress in EU](https://www.clinicaltrialvanguard.com/news/thermo-fisher-unveils-new-facility-enriching-advanced-therapies-progress-in-eu/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Thermo Fisher Scientific’s new facility in Bleiswijk, Netherlands, offers comprehensive cold and ultra-cold storage services for clinical trials and biorepository storage. This cGMP-compliant facility caters to the growing demand for cell and gene therapies, biologics, antibodies, and vaccines.
The facility provides a full range of services, including ambient to cryogenic storage, clinical packaging, labeling, distribution, and clinical QP release. With a team of highly skilled professionals, the site supports both new biotech and established pharmaceutical companies in meeting clinical trial requirements of any scale or phase.
Thermo Fisher’s expertise in managing valuable materials and its proven track record in bioservices and specialty logistics place it in a unique position to accelerate the development of innovative therapies. The Bleiswijk facility has 5,000 square meters of storage space, ancillaries, and cold chain packaging infrastructure.
In line with its sustainability commitment, the facility utilizes solar power, eco-friendly electric heat pumps, and heat recovery technology. This reflects Thermo Fisher’s dedication to providing end-to-end solutions for inclusive and impactful clinical research.
The opening of the Bleiswijk facility complements Thermo Fisher’s other recent expansions, including the biologics manufacturing capacity in St. Louis, the [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) manufacturing facility in San Francisco, and the Innovation Lab in Pennsylvania.
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**Categories:** News
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### [Summit Therapeutics $200M Raise: Exclusive Licenses Expand Ivoscimab Territories](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-200m-raise-exclusive-licenses-expand-ivoscimab-territories/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Summit Therapeutics](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-remarkable-results-in-phase-iii-harmoni-trial-for-lung-cancer/) raised $200 million through an institutional investment, with shares sold at a premium to the previous closing price. The proceeds will fund the clinical development of [ivonescimab](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-durvalumab-in-phase-iii-biliary-tract-cancer-trial/), an anti-cancer therapy.
In a separate transaction, Summit expanded the license territories for ivonescimab. Akeso, Inc. amended their collaboration agreement to include Latin America, the Middle East, and Africa. Summit previously held the licenses for the US, Canada, Japan, and Europe.
The expanded agreement aims to increase access to ivonescimab for patients worldwide, potentially benefiting those who can benefit from this therapy. Summit and Akeso reaffirmed their commitment to bring ivonescimab to as many individuals as possible.
The deal is valued at $70 million in exchange for the expanded rights. The original collaboration agreement, including royalty payments and manufacturing provisions, remains in effect. The amendment strengthens the partnership through enhanced data sharing, which can expedite clinical development and regulatory approvals globally.
Summit will hold a conference call on June 3, 2024, to discuss these developments further and provide updates on the progress of ivonescimab.
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**Categories:** News
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### [Takeda's TAK-861 Phase 2B Data: A Breakthrough for Narcolepsy Type 1](https://www.clinicaltrialvanguard.com/news/takedas-tak-861-phase-2b-data-a-breakthrough-for-narcolepsy-type-1/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
A Phase 2b trial has demonstrated significant improvements for [TAK-861](https://www.clinicaltrialvanguard.com/news/unveiling-the-transformative-power-of-takedas-narcolepsy-treatment-clinical-trial-triumphs-at-sleep-europe-2024/), an investigational treatment for narcolepsy type 1 (NT1). The trial involved 112 patients and evaluated the efficacy and safety of TAK-861 over 8 weeks.
NT1 is a chronic neurological disorder caused by the loss of orexin neurons. This results in low levels of orexin neuropeptides, leading to excessive daytime sleepiness, cataplexy, and other debilitating symptoms.
TAK-861 is an orexin receptor 2 agonist that aims to address the underlying orexin deficiency in NT1. The trial results showed statistically significant improvements in primary and secondary endpoints, including:
• Excessive daytime sleepiness
• Cataplexy frequency
• Nighttime sleep quality
• Hallucinations and sleep paralysis
The results also indicated that TAK-861 was generally safe and well-tolerated. Based on these findings, the researchers concluded that TAK-861 has the potential to be a transformative treatment for NT1, potentially providing substantial improvements over available therapies.
Takeda plans to initiate global Phase 3 trials of TAK-861 in NT1 in the first half of 2024. The Phase 2b data also supported the recent Breakthrough Therapy designation for TAK-861 from the U.S. Food and Drug Administration (FDA). This designation accelerates the development and review process for drugs that address serious or life-threatening conditions.
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**Categories:** News
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### [Calidi Presents Phase 1 Update and Preclinical Data on Cld-101 at ASCO 2024](https://www.clinicaltrialvanguard.com/news/calidi-presents-phase-1-update-and-preclinical-data-on-cld-101-at-asco-2024/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) presented its latest developments in antitumor virotherapy at the American Society of Clinical Oncology (ASCO) Annual Meeting.
NeuroNova ([CLD-101](https://www.clinicaltrialvanguard.com/news/city-of-hope-calidi-biotherapeutics-phase-1-trial-update-on-cld-101/)):
• Phase 1 study for recurrent high-grade gliomas has progressed, demonstrating safety and feasibility.
• Four weekly doses of CLD-101 are being administered, and no adverse events have been reported in cohorts 2 and 3.
• Enrollment is ongoing for the fourth cohort.
RTNova (CLD-400):
• Preclinical data shows that CLD-400 can survive in the bloodstream and reach all tumors systemically.
• This technology enables targeted delivery of oncolytic viruses to multiple tumor sites.
SuperNova (CLD-201):
• Non-clinical data supports the upcoming Phase 1 clinical trial of CLD-201.
• CLD-201’s cell-based oncolytic virotherapy aims to treat multiple solid tumors.
• An IND application with the FDA will be filed to initiate the trial in the future.
Calidi’s cutting-edge approach to antitumor virotherapy utilizes a combination of neural stem cells and oncolytic viruses to target and destroy cancer cells selectively. The safety and progress observed in the NeuroNova program reinforce the potential of this therapy for challenging brain tumors.
The RTNova and SuperNova platforms aim to extend the benefits of oncolytic virotherapy by enabling systemic delivery and targeting a broad range of solid tumors. The positive preclinical data supports Calidi’s plans for further clinical development of these novel technologies.
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**Categories:** News
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### [FDA Selects Neurogene's NGN-401 for Innovative Rett Syndrome Therapy Pilot Program](https://www.clinicaltrialvanguard.com/news/fda-selects-neurogenes-ngn-401-for-innovative-rett-syndrome-therapy-pilot-program/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene Inc.’s [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/) gene therapy for Rett syndrome has been selected for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. This program provides enhanced communication between sponsors and the FDA to expedite the development of therapies for rare diseases.
NGN-401 was chosen for its potential clinical benefits and the maturity of its development program. The START Program will offer Neurogene frequent guidance and ad-hoc discussions to address development issues, including clinical study design, patient population selection, and control group choice.
NGN-401 is being evaluated in a Phase 1/2 clinical trial, assessing its safety, tolerability, and preliminary efficacy in pediatric female patients with Rett syndrome. Neurogene has reported positive safety data from the first three patients treated; interim efficacy data is expected in the fourth quarter of 2024.
The FDA’s selection of NGN-401 highlights the potential of this therapy to address the unmet medical needs of patients with Rett syndrome. The START Program will accelerate NGN-401’s development and bring it closer to a potential registrational study for this devastating condition.
About NGN-401:
NGN-401 is an investigational AAV9 gene therapy designed as a single-dose treatment for Rett syndrome. It delivers the full-length human MECP2 gene, which is deficient or mutated in Rett syndrome patients. NGN-401 aims to restore the normal function of MECP2 and improve the symptoms of Rett syndrome.
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**Categories:** News
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### [Fulgent Data: Uncovering Fid-007 in Head and Neck Cance](https://www.clinicaltrialvanguard.com/news/fulgent-data-uncovering-fid-007-in-head-and-neck-cance/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Fulgent Pharma, a subsidiary of Fulgent Genetics, presented data at ASCO 2024 showing promising results for their therapeutic candidate, [FID-007](https://www.clinicaltrialvanguard.com/news/fulgent-announces-clinical-data-presentation-for-lead-oncology-candidate-at-asco-2024/), in treating head and neck cancer.
Phase 1 clinical trials evaluated FID-007 in 11 head and neck squamous cell carcinoma patients. Of these, 45% experienced a partial response, and 27% achieved stable disease. Notably, the drug was well-tolerated, with no high-grade neuropathy observed.
FID-007 demonstrated anti-tumor activity in patients who had previously received taxane treatment. The duration of follow-up ranged from 1 to 15 months.
Encouraged by these findings, Fulgent Pharma has initiated a Phase 2 study combining FID-007 and [cetuximab](https://www.clinicaltrialvanguard.com/news/frontier-medicines-presents-preclinical-data-on-3-programs/) in patients with head and neck cancer. The drug’s unique formulation, which encapsulates paclitaxel in a polymer excipient, enhances its pharmacokinetics, distribution, and tolerability. The nanoparticle formulation targets tumors’ leaky vasculature, delivering paclitaxel directly to cancer cells.
Fulgent Pharma’s commitment to advancing cancer therapeutics is evident in its partnerships with leading institutions such as the University of Southern California, Moffitt Cancer Center, and ANP Technologies. The company remains dedicated to developing innovative drug candidates that address the unmet needs of cancer patients.
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**Categories:** News
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### [Obsidian's OBX-115 Data Unveiled at ASCO 2024: Safety, Efficacy, and Surprises](https://www.clinicaltrialvanguard.com/news/obsidians-obx-115-data-unveiled-at-asco-2024-safety-efficacy-and-surprises/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Obsidian Therapeutics presented updated data from the Phase 1 study of OBX-115, an engineered tumor-derived autologous T-cell immunotherapy, at the 2024 American Society of Clinical Oncology Annual Meeting.
OBX-115 exhibited a differentiated safety profile compared to IL2-dependent non-engineered TIL cell therapies. The therapy demonstrated a consistent efficacy profile in heavily pre-treated patients with resistant/refractory disease.
The study included data from 10 patients in the safety analysis and nine patients in the per-protocol efficacy analysis set. OBX-115 showed promising early activity without the administration of IL2.
Key findings included:
• Sustained efficacy, including objective response rates and median progression-free survival comparable to other late-line systemic therapies.
• Positive safety profile, no severe cytokine release syndrome or neurotoxicity observed.
• Potential for optimized [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) product, including non-surgical tumor tissue procurement and re-energizing engrafted cells.
Based on these results, Obsidian continues enrolling patients with advanced or metastatic melanoma and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) in a Phase 1/2 multicenter study. The company is exploring the potential of OBX-115 to advance the TIL cell therapy field by enabling non-surgical tumor tissue procurement and eliminating the need for IL2.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Bristol Myers Squibb Unveils Remarkable Analyses on Opdivo's Efficacy in Non-Small Cell Lung Cancer](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibb-unveils-remarkable-analyses-on-opdivos-efficacy-in-non-small-cell-lung-cancer/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb’s ongoing research in non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) has yielded promising results in various stages of the disease.
Perioperative Opdivo in Early-Stage NSCLC
Updated data from the [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -77T study demonstrate the effectiveness of perioperative Opdivo in improving event-free survival in resectable stage III NSCLC patients, regardless of the number of lymph nodes affected. The Opdivo regimen significantly extended this critical endpoint compared to neoadjuvant chemotherapy and placebo.
Neoadjuvant Opdivo in Resectable NSCLC
A four-year follow-up analysis from the CheckMate -816 study reinforces the benefits of neoadjuvant Opdivo plus chemotherapy in patients with resectable NSCLC. This combination therapy showed efficacy in improving pathological complete response, indicating a lack of detectable cancer cells after surgery.
Opdivo Combinations in Advanced NSCLC
Five-year follow-up data from the CheckMate -9LA study highlight the impact of Opdivo plus Yervoy and chemotherapy in improving survival outcomes for patients with previously untreated metastatic NSCLC. This combination therapy significantly extended overall survival compared to chemotherapy alone.
Expanding the Lung Cancer Portfolio
Bristol Myers Squibb’s ongoing research extends beyond immunotherapy to include targeted approaches. An updated analysis from the TRIDENT-1 study shows Augytro’s durable response in ROS1-positive NSCLC patients. Additionally, the [KRYSTAL](https://www.clinicaltrialvanguard.com/opinion/what-the-krystal-12-lancet-correspondence-actually-says-about-pro-reporting-in-krasg12c-trials/)-12 study demonstrated Krazati’s efficacy in improving progression-free survival in KRASG12C-mutated NSCLC patients.
These findings underscore the company’s commitment to providing innovative treatment options for patients with NSCLC, both in early and advanced stages. The expanding thoracic portfolio offers hope and the possibility of improved survival outcomes.
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**Categories:** News
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### [Visugromab/Nivolumab Combo Triumphs in Advanced Cancer Battle](https://www.clinicaltrialvanguard.com/news/visugromab-nivolumab-combo-triumphs-in-advanced-cancer-battle/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Positive Results from CatalYm’s GDFATHER Trial
CatalYm’s “GDFATHER” Phase 1/2a trial has shown promising results for its lead candidate, visugromab, in combination with nivolumab, an anti-PD-1 antibody. The trial included cohorts of patients with [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC), urothelial cancer (UC), hepatocellular carcinoma (HCC), and an additional biomarker cohort.
Mechanism of Action
Visugromab is a monoclonal antibody that neutralizes Growth Differentiation Factor-15 ([GDF-15](https://www.clinicaltrialvanguard.com/news/visugromab-reverses-checkpoint-inhibitor-resistance/)), a protein produced by tumors that suppresses the immune system’s ability to fight cancer. By blocking GDF-15, visugromab aims to overcome this resistance and enhance the effectiveness of immunotherapies.
Clinical Results
The trial data demonstrated that the combination of visugromab and nivolumab induced deep and durable anti-tumor activity in patients who had previously failed anti-PD-1/PD-L1 therapies. Notably, over half of the responders achieved a response level not previously achieved with their prior anti-PD-(L)1 treatment.
This suggests that visugromab’s ability to block GDF-15 leads to a deeper and more sustained remission than other cancer immunotherapies.
Safety and Tolerability
The combination of visugromab and nivolumab was well-tolerated, with no significant safety concerns reported. This favorable safety profile is critical for ensuring that patients can receive the treatment for an extended period without experiencing adverse effects.
Future Development Plans
CatalYm is planning a broad Phase 2b development program to investigate the potential of visugromab further. This program will explore the optimal use of visugromab in combination with standard-of-care treatments and in earlier lines of therapy.
Source link: [http://www.businesswire.com/news/home/20240602170372/en/CatalYm-Reports-Impressive-and-Lasting-Responses-Including-Multiple-Complete-Responses-in-Heavily-Pretreated-Late–to-Last-Line-Metastatic-NSCLC-Urothelial-and-Hepatocellular-Cancer-Patients-Treated-with-VisugromabNivolumab-Combination](http://www.businesswire.com/news/home/20240602170372/en/CatalYm-Reports-Impressive-and-Lasting-Responses-Including-Multiple-Complete-Responses-in-Heavily-Pretreated-Late--to-Last-Line-Metastatic-NSCLC-Urothelial-and-Hepatocellular-Cancer-Patients-Treated-with-VisugromabNivolumab-Combination)
**Categories:** News
**Tags:** eClinical Tech News
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### [Legend Biotech shares data on CARVYKTI® in the treatment of multiple myeloma](https://www.clinicaltrialvanguard.com/news/legend-biotech-shares-data-on-carvykti-in-the-treatment-of-multiple-myeloma/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Legend Biotech](https://www.clinicaltrialvanguard.com/news/legend-biotech-new-myeloma-lymphoma-car-t-data-at-ash/) Corporation, a leader in [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), announced positive results from its Phase 2 CARTITUDE-2 Cohort D study, demonstrating the efficacy of CARVYKTI in treating multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/) patients. CARVYKTI, approved for relapsed/refractory multiple myeloma, showed promising results in patients with less than a complete response after autologous stem cell transplant (ASCT).
The study enrolled 17 patients who received a single infusion of CARVYKTI with or without lenalidomide maintenance. At a median follow-up of 22 months, 94% of patients achieved an overall response, with 16 patients achieving a complete response or better. The median duration of response was not reached, and the median time to first response was one month.
CARVYKTI also demonstrated improved progression-free survival and overall survival rates, exceeding 90% at 18 months. Safety signals were consistent with the known profile of CARVYKTI, with no cases of movement and neurocognitive treatment-emergent adverse events.
Legend Biotech is optimistic about CARVYKTI’s potential in earlier treatment settings for multiple myeloma. Phase 3 studies are underway to explore its benefits as a frontline treatment option.
Additionally, a subgroup analysis from the CARTITUDE-4 Phase 3 study showed that CARVYKTI significantly improved progression-free survival compared to standard therapies in patients with high-risk multiple myeloma. These results highlight the potential role of CARVYKTI in improving outcomes for multiple myeloma patients in various stages of their treatment journey.
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**Categories:** News
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### [Breyanzi: Remarkable Outcomes in B-Cell Malignancies Revealed](https://www.clinicaltrialvanguard.com/news/breyanzi-remarkable-outcomes-in-b-cell-malignancies-revealed/)
**Published:** June 4, 2024
**Author:** Jon Napitupulu
**Content:**
Long-term follow-up data from the TRANSFORM trial reveals that Breyanzi, a chimeric antigen receptor T-cell (CAR T) therapy, continues to provide event-free survival and durable responses in patients with relapsed or refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (LBCL) who received treatment in the second line.
Subgroup analysis from the TRANSCEND NHL 001 trial demonstrates Breyanzi’s consistent clinical benefit in [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL) patients, irrespective of prior treatment lines. This finding suggests the potential for earlier Breyanzi use in MCL treatment.
Additionally, data from a subgroup analysis of the TRANSCEND FL trial indicates that Breyanzi exhibits consistent efficacy and a favorable safety profile in relapsed or refractory follicular lymphoma (FL) patients, regardless of bridging therapy. This reinforces Breyanzi’s distinct characteristics in treating FL.
These studies underscore the transformative potential of Breyanzi in various lymphoma subtypes. Its clinically significant outcomes and demonstrated efficacy across a wide range of B-cell malignancies position it as a potentially definitive therapy.
The FDA recently accelerated the approval of Breyanzi for patients with relapsed or refractory FL who have undergone two or more prior lines of systemic treatment. Approval has also been granted for patients with relapsed or refractory MCL who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor.
Important Safety Information
Breyanzi carries boxed warnings for the following potential adverse events:
• Cytokine Release Syndrome (CRS)
• Neurologic Toxicities
• Secondary Hematological Malignancies
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**Categories:** News
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### [Be Bio: FDA Grants New Designation for Novel Medicine - Hemophilia B Breakthrough](https://www.clinicaltrialvanguard.com/news/be-bio-fda-grants-new-designation-for-novel-medicine-hemophilia-b-breakthrough/)
**Published:** June 5, 2024
**Author:** Jon Napitupulu
**Content:**
Be Biopharma (Be Bio) announced that the FDA has granted Orphan Drug Designation to [BE-101](https://www.clinicaltrialvanguard.com/news/revolutionary-fda-clearance-paves-way-for-game-changing-hemophilia-b-therapy/), an engineered B Cell Medicine (BCM) for [Hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/) B. The designation provides exclusive marketing rights, user fee exemption, and clinical trial tax credits.
BE-101 is designed to provide sustained therapeutic FIX activity with a single infusion, potentially eliminating the frequent infusions required by current Hemophilia B treatments. This could lead to reduced bleeding rates and lower usage of FIX replacement therapies.
Hemophilia B, an X-linked bleeding disorder affecting males, occurs due to mutations in the FIX gene. Despite advances, patients still experience bleeding, joint damage, and chronic pain.
Be Bio’s Phase 1/2 study, BeCoMe-9, evaluating BE-101 in patients with severe or moderately severe Hemophilia B, is expected to begin in the second half of 2024. The study aims to demonstrate the efficacy and safety of BE-101 as a long-lasting alternative to current treatments.
Engineered B Cell Medicines (BCM) leverage the B cell’s ability to continuously produce high levels of proteins. This approach can potentially revolutionize the treatment paradigm in various diseases, including Hemophilia B, by offering a single-infusion, durable therapy that can be re-dosed.
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**Categories:** News
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### [AstraZeneca's Investment Powers Nucleus Radiopharma's Series A Extension](https://www.clinicaltrialvanguard.com/news/astrazenecas-investment-powers-nucleus-radiopharmas-series-a-extension/)
**Published:** June 7, 2024
**Author:** Jon Napitupulu
**Content:**
Nucleus RadioPharma has expanded its capabilities with a Series A investment extension and the appointment of Dr. Tyrell Rivers to its Board of Directors. This funding, led by [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/), will advance targeted radiotherapies and theranostics, offering new hope for patients battling metastatic cancers.
Theranostics seamlessly integrates diagnostics and therapeutics, employing radiotracers to precisely target cancer cells. Radiotracers enable both tumor visualization and potent radiation delivery, minimizing harm to healthy tissues. This approach exhibits promise in treating neuroendocrine tumors, prostate cancer, and lymphoma.
Nucleus RadioPharma’s CEO, Charles S. Conroy, expressed enthusiasm for the investment’s potential to increase the accessibility of these life-saving treatments. “These drugs, designed for precise targeting, are demonstrating remarkable effectiveness while upholding an exceptional safety record,” he stated.
Dr. Rivers, Executive Director of Corporate Ventures at AstraZeneca, brings over two decades of experience in investment and life science to the Nucleus board. He has a proven track record in driving business growth, directing corporate strategy, and fostering financially sound businesses. His expertise will be invaluable in expanding Nucleus’s global reach and impact.
Conroy emphasized the excitement surrounding this funding and Dr. Rivers’s addition to the board by indicating that the support of AstraZeneca and Tyrell on the board has ignited their excitement, as this funding will facilitate the expansion of their development, supply, and commercial manufacturing capabilities, ultimately enhancing global accessibility to targeted radiotherapies and theranostics for patients worldwide.
Overall, this investment and the appointment of Dr. Rivers position Nucleus RadioPharma for accelerated growth, enabling the wider adoption of these innovative cancer treatments and offering hope to patients facing limited treatment options.
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**Categories:** News
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### [FDA Grants Tagrisso® Priority Review for Unresectable Stage III EGFR-Mutated Lung Cancer](https://www.clinicaltrialvanguard.com/news/fda-grants-tagrisso-priority-review-for-unresectable-stage-iii-egfr-mutated-lung-cancer/)
**Published:** June 11, 2024
**Author:** Jon Napitupulu
**Content:**
AstraZeneca has filed and received Priority Review status in the United States for [TAGRISSO](https://www.clinicaltrialvanguard.com/news/tagrisso-approved-for-unresectable-stage-iii-egfr-mutated-lung-cancer/)® (osimertinib) for treating adult patients with unresectable, Stage III [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) harboring epidermal growth factor receptor (EGFR) mutations following chemoradiotherapy (CRT). The FDA has also granted Breakthrough Therapy Designation for TAGRISSO in this setting.
The submitted data includes results from the LAURA Phase III trial, which showed that TAGRISSO significantly improved progression-free survival (PFS) compared to placebo. Median PFS was 39.1 months for patients treated with TAGRISSO versus 5.6 months for placebo. This benefit was consistent across various subgroups, including sex, race, and type of EGFR mutation.
Priority Review and Breakthrough Therapy Designation expedite the development and review process for new medicines that address unmet medical needs and demonstrate substantial improvements over existing therapies. Approximately 15% of US NSCLC patients have EGFR mutations, and a significant number are unresectable.
TAGRISSO is currently an established therapy in EGFR-mutated lung cancer. The extended PFS observed in the LAURA trial highlights the importance of EGFR mutation testing at diagnosis. The sNDA approval, anticipated in Q4 2024, would provide a targeted treatment option for patients with Stage III EGFRm NSCLC after CRT.
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**Categories:** News
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### [Unveiling Calidi's Breakthrough Virotherapy: Revolutionizing Cancer Treatment](https://www.clinicaltrialvanguard.com/news/unveiling-calidis-breakthrough-virotherapy-revolutionizing-cancer-treatment/)
**Published:** June 11, 2024
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/), a biotechnology company focused on developing anti-tumor virotherapies, has forged a partnership with SIGA Technologies to advance its RTNova platform. This platform utilizes an enveloped vaccinia virus (RTNova) engineered to target and eliminate tumor cells.
The RTNova platform holds potential as a universal treatment for various cancer types, primarily focusing on [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) and metastatic solid tumors. Its systemic and targeted nature allows for easier administration and a wider potential patient reach.
One crucial aspect of the collaboration is the integration of SIGA’s TPOXX, an antiviral agent. TPOXX will serve as a safety switch to manage the spread of RTNova in the body. This provides reassurance and confidence during clinical trials for patients, physicians, and regulatory authorities.
Calidi’s Chief Scientific Officer, Antonio F. Santidrian, expressed optimism about the collaboration, stating that RTNova has the potential to revolutionize cancer treatment across tumor types. The partnership with SIGA ensures access to a safety switch during the development process, fostering trust and confidence among stakeholders.
RTNova is designed to survive in the bloodstream, target multiple tumor sites, and stimulate the immune system against cancer cells. Preclinical studies have demonstrated TPOXX’s effectiveness against vaccinia, supporting its potential as an important component in vaccinia-based cancer therapies.
The collaboration between Calidi and SIGA aims to accelerate the development of the RTNova platform, paving the way for innovative cancer treatments with the potential to significantly improve patient outcomes.
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**Categories:** News
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### [FDA Approves GSK's AREXVY RSV Vaccine for At-Risk Seniors over 50](https://www.clinicaltrialvanguard.com/news/fda-approves-gsks-rsv-vaccine-for-at-risk-seniors-over-50/)
**Published:** June 11, 2024
**Author:** Jon Napitupulu
**Content:**
The US Food and Drug Administration (FDA) has given its approval to AREXVY (Respiratory Syncytial Virus Vaccine, Adjuvanted) for preventing lower respiratory tract disease (LRTD) caused by RSV in high-risk adults between the ages of 50 and 59. RSV is a prevalent respiratory virus that can cause severe illness, particularly in adults with chronic underlying health conditions such as COPD, [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/), heart failure, and diabetes.
Data indicates that RSV accounts for approximately 42,000 hospitalizations annually in US adults aged 50-64, underscoring the need for expanded immunization. GSK’s RSV vaccine is currently approved and recommended for adults 60 and older, but the new approval extends its reach to younger adults at increased risk.
Phase III trial results demonstrated the vaccine’s safety and effectiveness in adults aged 50-59, including those with underlying medical conditions. Experts emphasize that age alone is not a reliable indicator of RSV risk, as underlying health conditions significantly elevate susceptibility to severe outcomes.
GSK has submitted regulatory applications to extend the vaccine’s use to high-risk adults aged 50-59 in Europe, Japan, and other countries. Clinical trials are also underway to assess the vaccine’s immunogenicity and safety in immunocompromised adults and adults aged 18-49 at increased risk. The vaccine contains recombinant RSV glycoprotein F, stabilized in the prefusion conformation, and is combined with GSK’s AS01E adjuvant.
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**Categories:** News
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### [First Patient Enrolled in Monotherapy Study Evaluating Camonsertib in Non-Small Cell Lung Cancer](https://www.clinicaltrialvanguard.com/news/first-patient-enrolled-in-monotherapy-study-evaluating-camonsertib-in-non-small-cell-lung-cancer/)
**Published:** June 11, 2024
**Author:** Jon Napitupulu
**Content:**
Repare Therapeutics, a precision oncology company, has initiated patient dosing in the expanded phase of its TRESR clinical trial, which evaluates camonsertib monotherapy in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) with ATM mutations.
Camonsertib has shown promising anti-tumor activity in ongoing trials involving patients with ATM-mutated NSCLC. The rapid expansion of the TRESR trial demonstrates the company’s commitment to addressing the significant unmet medical need among patients with this rare and aggressive form of lung cancer.
The TRESR trial is a multi-center, open-label study designed to assess the safety, efficacy, and tolerability of camonsertib as a monotherapy in NSCLC patients harboring ATR-inhibitor sensitizing mutations. The NSCLC expansion aims to enroll up to 20 patients to evaluate the effectiveness of camonsertib at a recommended Phase 2 dosage.
With limited treatment options available for advanced or recurrent NSCLC, camonsertib represents a potential oral therapy with an established safety profile. Repare Therapeutics anticipates potential data readout from the monotherapy expansion in 2025.
Repare Therapeutics employs a genome-wide [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)-enabled platform called SNIPRx® to discover and develop targeted cancer therapies. Their pipeline includes multiple compounds in clinical development, including lunresertib, camonsertib, RP-1664, and RP-3467, as well as other preclinical programs.
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**Categories:** News
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### [Viridian Therapeutics Launches Two Phase 3 Trials: TED](https://www.clinicaltrialvanguard.com/news/viridian-therapeutics-launches-two-phase-3-trials-ted/)
**Published:** June 13, 2024
**Author:** Jon Napitupulu
**Content:**
Viridian Therapeutics plans to initiate two phase 3 clinical trials (REVEAL-1 and REVEAL-2) for VRDN-003, an anti-IGF-1R antibody, in August 2024. The trials will evaluate VRDN-003 for treating moderate-to-severe thyroid eye disease (TED).
VRDN-003 is designed to be administered subcutaneously as infrequently as every eight weeks. The company believes this convenience factor could enhance patient access and adherence to treatment.
REVEAL-1 will involve around 84 patients randomized into three groups: placebo, VRDN-003 every four weeks, and VRDN-003 every eight weeks. REVEAL-2 will involve approximately 126 patients randomized in the same manner. The primary efficacy endpoint for both trials will be the proportion of patients achieving a minimum 2mm improvement in proptosis (eye protrusion) from baseline at week 24.
Topline results from both trials are expected in the first half of 2026. If successful, Viridian aims to file a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) by the end of 2026. The company intends to launch VRDN-003 with an autoinjector pen for convenient administration.
Viridian believes VRDN-003 has the potential to be a best-in-class treatment for TED, offering the convenience of subcutaneous dosing and potentially improving outcomes for patients.
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**Categories:** News
---
### [Vector Labs and Absolute Biotech Merge](https://www.clinicaltrialvanguard.com/news/vector-labs-and-absolute-biotech-merge/)
**Published:** June 13, 2024
**Author:** Jon Napitupulu
**Content:**
The union of Vector Laboratories and Absolute Biotech, facilitated by Thompson Street Capital Partners, is a strategic move that strengthens both companies’ positions in the industry. This merger enhances their combined capabilities as manufacturers of critical components and expands their joint commercial presence in key markets.
Vector Laboratories, known for its reagents and components for [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) development, is a valuable partner for biopharma companies. With this merger, Vector expands its global reach and provides faster delivery of products and services.
Absolute Biotech, a manufacturer and supplier of antibody reagents, aims to make recombinant antibodies more accessible. They offer a comprehensive antibody portfolio to support various antibody-related processes.
The merged entity possesses manufacturing capabilities for small and large-scale projects, allowing them to support customers throughout the drug and diagnostic development processes.
The merger streamlines the process for biopharma customers by providing integrated solutions that reduce development time and resource commitments. Suppliers with expertise in antibody-drug conjugates are vital to the success of pharma and biotech clients, and this merger strengthens their offering in this area.
Integrating Absolute Biotech’s validated antibodies with Vector’s detection reagents creates a comprehensive suite of solutions for assay developers in translational and pre-clinical research.
This merger aligns with Thompson Street Capital Partners’ vision of building a comprehensive platform that supports the life sciences industry from early-stage research to clinical trials.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Medable's Digital Health Solution Recognized with Prestigious Award](https://www.clinicaltrialvanguard.com/news/medables-digital-health-solution-recognized-with-prestigious-award/)
**Published:** June 14, 2024
**Author:** Jon Napitupulu
**Content:**
Medable, a leading provider of clinical trial technology, has once again secured the “Best Digital Health Solution” award at the Prix Galien UK Forum. This is the second consecutive year the company has received this recognition for its innovative platform, PALO ALTO.
Medable’s platform has gained widespread trust among major pharmaceutical companies and CROs, including 14 of the world’s top 20 pharmaceutical giants. Customers using Medable’s platform have reported remarkable outcomes, such as accelerated enrollment by 200% and cost reductions of 50%.
“This award is a testament to our unwavering commitment to transforming drug development,” said Medable CEO and co-founder Dr. Michelle Longmire. “We are eager to continue collaborating with industry leaders and partners to accelerate the process of bringing scientific discoveries to patients.”
The Prix Galien Awards, organized by The Galien Foundation, honor groundbreaking advancements in [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) innovation. The Galien Foundation celebrates excellence in scientific discovery and innovation that enhance human health. It orchestrates the Prix Galien, an international program recognizing the development of innovative medicines with chapters worldwide. The Prix Galien holds a prestigious reputation, akin to the Nobel Prize in biopharmaceutical research.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Brenus Pharma's Revolutionary Colorectal Cancer Vaccine Launches in Human Trial](https://www.clinicaltrialvanguard.com/news/brenus-pharmas-revolutionary-colorectal-cancer-vaccine-launches-in-human-trial/)
**Published:** June 14, 2024
**Author:** Jon Napitupulu
**Content:**
Lyon, France-based biotech company Brenus Pharma has announced the study design of “BreAK-CRC,” a first-in-human trial of its lead candidate, STC-1010, for [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/). The trial will evaluate the safety and efficacy of STC-1010, an immunotherapeutic approach based on a cancer vaccine mechanism.
STC-1010 has shown promising preclinical results, and the Phase I/IIA BreAK-CRC trial will enroll patients with unresectable advanced or metastatic colorectal cancer, the second leading cause of cancer mortality worldwide. The trial will assess the tolerability and efficacy of STC-1010, combined with immunostimulants and chemotherapy.
Dr. François Ghiringhelli of Centre Georges-François Leclerc, University of Burgundy, discussed the importance of BreAK-CRC, as current immunotherapies are only effective in a small subset of colorectal cancer patients.
The study design was presented at the recent ASCO annual meeting in Chicago. The trial protocol has been reviewed by the French National Health Authority, and submission of the Clinical Trial Application through the European Union clinical trial information system is underway.
Brenus Pharma’s pioneering Stimulated-Tumor-Cell (STC) platform aims to develop a new generation of immunotherapies against cancer. The STC platform imitates the conditions of cancer relapse in vitro, allowing the patient’s immune system to learn about tumor evolution and resistance mechanisms.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [CyanVac Receives Barda-Funded Award to Evaluate Intranasal COVID-19 Vaccine Candidate in Phase 2b Study](https://www.clinicaltrialvanguard.com/news/cyanvac-receives-barda-funded-award-to-evaluate-intranasal-covid-19-vaccine-candidate-in-phase-2b-study/)
**Published:** June 14, 2024
**Author:** Jon Napitupulu
**Content:**
CyanVac LLC, a biotechnology company, received federal funding to conduct a comparative Phase 2b study of CVXGA, its PIV5-based intranasal vaccine candidate for [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/). This funding is part of the Project NextGen initiative, aimed at developing innovative vaccines for COVID-19.
Through [BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)‘s Clinical Studies Network, the study will compare CVXGA to an FDA-approved mRNA-based COVID-19 vaccine in a 10,000-participant randomized double-blinded trial. It will assess efficacy, safety, and immunogenicity, including preventing asymptomatic infections.
The PIV5 vector has been shown to replicate safely in clinical trials, stimulating cellular, mucosal, and humoral immunity with minimal side effects. An intranasal vaccine using this vector could potentially reduce disease transmission.
CyanVac will sponsor the study, which is expected to begin in fall 2024. The company believes the successful development of CVXGA will demonstrate the capabilities of its PIV5 platform and pave the way for PIV5-based vaccines for other emerging infectious diseases.
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**Categories:** News
---
### [Ensem Therapeutics Partners With Beigene as Novel CDK2 Inhibitor Enters Clinical Trials for Solid Tumors](https://www.clinicaltrialvanguard.com/news/ensem-therapeutics-partners-with-beigene-as-novel-cdk2-inhibitor-enters-clinical-trials-for-solid-tumors/)
**Published:** June 14, 2024
**Author:** Jon Napitupulu
**Content:**
Ensem Therapeutics has partnered with [BeiGene](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/) to advance ETX-197, now known as BG-68501, into clinical trials. BG-68501 is a potent inhibitor of the cyclin-dependent kinase 2 (CDK2), an enzyme involved in the uncontrolled growth of solid tumors.
This achievement is a testament to the power of Ensem’s Kinetic Ensemble® platform, which leverages artificial intelligence (AI) and machine learning to uncover previously hidden binding pockets in target proteins. BG-68501 is the first clinical-stage compound to emerge from this platform, demonstrating its potential for revolutionizing drug discovery.
Shengfang Jin, CEO of Ensem, emphasized the difficulty in targeting CDK2 in cancer treatment. However, the company’s novel approach allows for identifying “cryptic sites” that are not easily identified in traditional protein structures. By targeting these sites, BG-68501 can potentially inhibit CDK2 hyperactivity, a key driver in numerous cancer types.
BeiGene has initiated a Phase 1 clinical trial to evaluate BG-68501 in advanced solid tumors. The trial will assess the safety and efficacy of the drug in various cancer types, including breast, ovarian, and [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/).
Ensem’s successful completion of the preclinical development and rapid advancement of BG-68501 into clinical trials highlights the efficiency of its platform. With multiple early-stage programs underway, the company anticipates nominating two additional development candidates in 2024.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Calquence® Plus Chemoimmunotherapy: Remarkable Breakthrough in Mantle Cell Lymphoma Treatment](https://www.clinicaltrialvanguard.com/news/calquence-plus-chemoimmunotherapy-remarkable-breakthrough-in-mantle-cell-lymphoma-treatment/)
**Published:** June 17, 2024
**Author:** Jon Napitupulu
**Content:**
AstraZeneca’s CALQUENCE ([acalabrutinib](https://www.clinicaltrialvanguard.com/news/ascentage-pharma-presents-promising-cancer-research-at-aacr/)), combined with bendamustine and [rituximab](https://www.clinicaltrialvanguard.com/news/budoprutug-shows-response-in-rituximab-experienced-itp-patients/), has demonstrated significant benefits for patients with [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL).
In the Phase III ECHO trial, the CALQUENCE regimen improved progression-free survival (PFS) by 27% compared to standard chemoimmunotherapy, with a median PFS of 66.4 months versus 49.6 months.
Additionally, the CALQUENCE combination showed a favorable trend in overall survival (OS) compared to standard therapy. Although the OS data was immature during analysis, the trial will continue to monitor OS as a key endpoint.
Analysis excluding COVID-19-related deaths further enhanced the PFS benefit, with the CALQUENCE regimen reducing the risk of disease progression or death by 36%. The median PFS for this analysis was not reached for the CALQUENCE combination, while it was 61.6 months for standard therapy.
Michael Wang, the trial’s principal investigator, emphasized the potential of the CALQUENCE combination to change the treatment paradigm for MCL, especially for older adults, who constitute the majority of MCL patients.
Susan Galbraith, AstraZeneca’s Executive Vice President of Oncology R&D, highlighted the clinical significance of the ECHO trial results, particularly the substantial increase in PFS and the promising trend in OS. This combination is anticipated to become a valuable new option for MCL patients.
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**Categories:** News
---
### [Empatica's New Integration Enhances Clinical Mobility Solutions](https://www.clinicaltrialvanguard.com/news/empaticas-new-integration-enhances-clinical-mobility-solutions/)
**Published:** June 19, 2024
**Author:** Jon Napitupulu
**Content:**
Empatica, a leader in digital biomarker development, has partnered with Mobilise-D, an initiative funded by the Innovative Medicines Initiative. This collaboration aims to integrate Mobilise-D’s Digital Mobility Outcomes (DMOs) into Empatica’s Health Monitoring Platform.
DMOs are validated digital measures that assess mobility using wearable sensors. By integrating these outcomes into its platform, Empatica provides researchers and clinicians with a comprehensive tool for monitoring mobility in patients with [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/), COPD, and [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/).
This partnership removes barriers to implementing digital outcomes in clinical trials by seamlessly providing accurate and reliable data. Empatica’s EmbracePlus wearable and Health Monitoring Platform consolidates advanced mobility analytics with Empatica’s robust technology.
The collaboration aims to drive innovation and harmonize digital outcome assessment in clinical trials. By combining Mobilise-D’s algorithms with Empatica’s technology, researchers can easily include these measures in studies, reducing participant burden.
Integrating Mobilise-D DMOs into Empatica’s platform is a significant step toward regulatory approval of digital endpoints in clinical drug development. It enables the assessment of mobility in daily life, aiding therapeutic development and precision medicine.
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**Categories:** News
---
### [Unveiling the Promising Advancements in Blood Cancer Treatment: Innate Pharma's Groundbreaking Trial Findings](https://www.clinicaltrialvanguard.com/news/unveiling-the-promising-advancements-in-blood-cancer-treatment-innate-pharmas-groundbreaking-trial-findings/)
**Published:** June 19, 2024
**Author:** Jon Napitupulu
**Content:**
At the European Hematology Association Congress, [Innate Pharma](https://www.clinicaltrialvanguard.com/news/innate-pharma-ifli-announce-7-9m-investment-for-iph6501-in-follicular-lymphoma/) and Sanofi presented promising results from their Phase 1/2 study of SAR’579, a Natural Killer Cell Engager (NKCE). SAR’579, targeting [CD123](https://www.clinicaltrialvanguard.com/news/unlock-the-latest-in-cancer-treatment-sanofi-and-innate-pharmas-sar443579-iph6101-breakthrough/), showed significant clinical efficacy in patients with relapsed or refractory acute myeloid leukemia (R/R AML).
The study assessed SAR’579 as monotherapy for blood cancers, including AML, B-cell acute lymphoblastic leukemia (B-ALL), and high-risk myelodysplasia (HR-MDS). Fifty-nine patients received treatment across various dose levels.
A maximum response rate was observed at a target dose of 1 mg/kg weekly, with five AML patients achieving complete remission (CR). The median treatment duration was 7.9 weeks, and durable CRs (>10 months) were observed in three patients. SAR’579 demonstrated good tolerability up to doses of 6 mg/kg weekly.
These findings support the further development of SAR’579 in the Phase 2 portion of the trial. Ongoing studies aim to explore its potential in patients with leukemia.
Innate Pharma’s [ANKET](https://www.clinicaltrialvanguard.com/news/innate-pharmas-next-gen-anket-iph6501-highlighted-in-science-immunology/) platform enables the development of multi-specific NK cell engagers for cancer treatment. Sanofi leads the SAR’579 development program and is responsible for its advancement through clinical trials.
The Innate-Sanofi collaboration focuses on utilizing Innate’s proprietary technology to create new antibody formats that engage NK cells and enhance antitumor activity. The positive results from the SAR’579 study highlight the potential of this platform in addressing unmet medical needs in blood cancers.
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**Categories:** News
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### [Pyros Pharmaceuticals' FDA-Approved Vigafyde™: The Only Ready-to-Use Vigabatrin Oral Solution](https://www.clinicaltrialvanguard.com/news/pyros-pharmaceuticals-fda-approved-vigafyde-the-only-ready-to-use-vigabatrin-oral-solution/)
**Published:** June 19, 2024
**Author:** Jon Napitupulu
**Content:**
Pyros Pharmaceuticals has received FDA approval for VIGAFYDE™, an oral vigabatrin solution for treating infantile spasms (IS) in pediatric patients aged 1 month to 2 years. IS is a rare and severe form of [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/) with lasting effects.
VIGAFYDE™ is a monotherapy indicated where the benefits outweigh the potential risk of vision loss. The approval brings renewed enthusiasm to the IS community and will enhance efforts in disease education, care pathways, and research investments.
Michael Smith, Pyros CEO, emphasized the significance of VIGAFYDE™ as the first IS drug approved in 15 years, showcasing Pyros’ dedication to supporting affected families. Edwin Urrutia, Pyros COO, thanked stakeholders eagerly for anticipating this ready-to-use vigabatrin solution.
Pyros Total Care™, a comprehensive patient support program, offers personalized assistance to caregivers throughout the treatment journey, providing personal guidance and financial aid. This support system includes a nurse educator, reimbursement support, and a clinical pharmacist.
IS affects young children and can manifest in subtle, sometimes overlooked movements. Its potential long-term effects include seizures, autism spectrum disorder, and developmental issues. Early and effective treatment, as determined by a systematic American Academy of Neurology review, can positively influence long-term outcomes.
Source link: [http://www.businesswire.com/news/home/20240617159224/en/Pyros-Pharmaceuticals-Announces-FDA-Approval-of-VIGAFYDE%E2%84%A2-vigabatrin-as-the-First-and-Only-Ready-to-Use-Vigabatrin-Oral-Solution](http://\)
**Categories:** News
---
### [Aribio and KCTL Partner to Revolutionize Alzheimer's Testing with Uncommon Innovation](https://www.clinicaltrialvanguard.com/news/aribio-and-kctl-partner-to-revolutionize-alzheimers-testing-with-uncommon-innovation/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
AriBio Co., Ltd. has partnered with Kentucky Clinical Trial Laboratory (KCTL) to enhance [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease testing. This collaboration will improve access to accurate and efficient testing of cerebral spinal fluid (CSF) using the Lumipulse® system from FujireBio.
Samples from AriBio’s Phase 3 POLARIS-AD trial, investigating the efficacy of AR1001 in early Alzheimer’s patients, will be tested for the Aβ42/40 ratio using an FDA-approved Lumipulse® system assay. The trial also assesses other endpoints, including CDR-SB, ADAS-Cog 13, I-IADL, GDS, and MMSE, as well as changes in CSF and plasma biomarkers.
This partnership will support AriBio’s development of therapeutics for neurodegenerative diseases. The advanced diagnostics provided by KCTL enable a better understanding of patient progression and the impact of treatment on biomarkers.
KCTL is a CLIA-Certified laboratory specializing in clinical trial services for IVD and pharmaceutical manufacturers. Its expertise in cardiovascular, neurological, and cancer biomarker studies will contribute to advancements in Alzheimer’s disease research.
AriBio continues to explore collaborations to accelerate the development of innovative treatments and enhance understanding of neurodegenerative diseases.
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**Categories:** News
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### [Bristol Myers Squibb Introduces FDA-Approved Krazati® for Metastatic Colorectal Cancer](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibb-introduces-fda-approved-krazati-for-metastatic-colorectal-cancer/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
The FDA has approved KRAZATI (adagrasib) in combination with cetuximab as a treatment for locally advanced or metastatic colorectal cancer (CRC) in adult patients with a KRASG12C mutation. This approval is based on the Phase 1/2 [KRYSTAL](https://www.clinicaltrialvanguard.com/opinion/what-the-krystal-12-lancet-correspondence-actually-says-about-pro-reporting-in-krasg12c-trials/)-1 study, which showed a 34% objective response rate in pretreated patients with KRASG12C-mutated CRC.
KRAZATI is the first KRASG12C inhibitor approved by the FDA for a cancer type beyond [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/). Its approval is a significant advance for patients with KRASG12C-mutated CRC, as current late-line standard-of-care options provide limited response rates.
The approval is based on results from cohorts of the KRYSTAL-1 study, which evaluated KRAZATI combined with cetuximab in heavily pretreated CRC patients with a KRASG12C mutation. The study met its primary endpoint, demonstrating a confirmed objective response rate of 34% in patients receiving KRAZATI with cetuximab. All responses were partial responses, and the median duration of response was 5.8 months.
KRAZATI is associated with certain warnings and precautions, including gastrointestinal adverse reactions, QTc interval prolongation, hepatotoxicity, and interstitial lung disease/pneumonitis.
Bristol Myers Squibb’s senior vice president of U.S. Oncology and Hematology, Wendy Short Bartie, highlighted the significance of KRAZATI’s approval by indicating that this is an important milestone for BMS and the patients they serve as they deliver on our commitment to provide innovative medicines for cancer.
The FDA previously granted breakthrough therapy designation for KRAZATI in combination with cetuximab for patients with KRASG12C-mutated advanced CRC whose cancer has progressed following prior treatment with certain chemotherapy and an anti-EGFR therapy.
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**Categories:** News
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### [EMA Validates Bristol Myers Squibb's Subcutaneous Nivolumab Filing](https://www.clinicaltrialvanguard.com/news/ema-validates-bristol-myers-squibbs-subcutaneous-nivolumab-filing/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb has announced that the European Medicines Agency (EMA) has accepted an extension application for a new subcutaneous Opdivo (nivolumab) formulation. This formulation includes a new solution for injection and a strength of 600 mg/vial.
The application seeks approval for the subcutaneous use of Opdivo to treat multiple solid tumors in adults. This includes monotherapy, maintenance therapy following nivolumab and [ipilimumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/) combination therapy, and in combination with chemotherapy or cabozantinib.
The EMA’s acceptance of the application initiates its centralized review process, confirming the completeness of the submission.
Results from the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -67T trial showed that subcutaneous nivolumab demonstrated noninferiority to intravenous Opdivo regarding time-averaged serum concentration and trough serum concentration at steady state. Additionally, the objective response rate was also non-inferior.
The safety profile of subcutaneous nivolumab was consistent with the intravenous formulation.
The submission builds on Bristol Myers Squibb’s commitment to developing innovative formulations to improve patient experiences. The company aims to reduce administration time and increase convenience for patients receiving Opdivo treatment.
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**Categories:** News
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### [Precision Biosciences Enhances Hepatitis Scientific Advisory Board with Renowned Investigators](https://www.clinicaltrialvanguard.com/news/precision-biosciences-enhances-hepatitis-scientific-advisory-board-with-renowned-investigators/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
[Precision BioSciences](https://www.clinicaltrialvanguard.com/news/precision-biosciences-programs-make-a-stunning-return-collaboration-with-prevail-therapeutics-concludes/), a gene editing company, has appointed Dr. Mark Sulkowski and Dr. Jordan Feld to its Hepatitis Scientific Advisory Board (SAB).
Drs. Sulkowski and Feld bring extensive experience in hepatitis clinical trials. Dr. Sulkowski is a renowned expert at Johns Hopkins University School of Medicine, while Dr. Feld holds leadership roles at Toronto General Hospital and the University of Toronto.
The SAB will guide the development of PBGENE-HBV, Precision’s potential treatment for chronic hepatitis B. The company anticipates submitting an Investigational New Drug (IND) or Clinical Trial Application (CTA) for PBGENE-HBV in late 2024.
Dr. Raymond Schinazi, an inaugural member of the SAB, praised Drs. Sulkowski and Feld’s expertise and leadership in the hepatitis field. Precision’s President and CEO, Michael Amoroso, emphasized the value of their insights as they prepare to advance PBGENE-HBV into clinical trials.
The appointments strengthen the SAB’s expertise in hepatitis and support Precision BioSciences’ commitment to developing innovative gene-editing therapies for chronic hepatitis B.
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**Categories:** News
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### [Lenacapavir: Revolutionary Injection Beats Truvada for HIV Prevention](https://www.clinicaltrialvanguard.com/news/lenacapavir-revolutionary-injection-beats-truvada-for-hiv-prevention/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
A phase 3 clinical trial known as PURPOSE 1 has yielded breakthrough results in HIV prevention. The trial studied the efficacy of lenacapavir, an injectable HIV-1 capsid inhibitor.
Among cisgender women, twice-yearly lenacapavir demonstrated 100% efficacy in preventing HIV infections. This result is superior to the currently approved once-daily oral PrEP (pre-exposure prophylaxis) regimen, Truvada.
Based on the interim analysis, an independent Data Monitoring Committee recommended discontinuing the blinded phase of the trial and offering open-label lenacapavir to all participants.
Lenacapavir was generally well-tolerated, with no significant safety concerns identified. The trial included over 5,300 cisgender women and adolescent girls across 25 sites in South Africa and Uganda.
The PURPOSE program, of which PURPOSE 1 is a part, represents the most comprehensive and diverse HIV prevention trial program to date. It aims to advance scientific innovation, improve trial design, engage communities, and promote [health equity](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/) in the fight against HIV.
The results of PURPOSE 1 underscore the potential of lenacapavir as a transformative tool in HIV prevention. As the trial continues, researchers hope to gather additional data to support the development and implementation of new strategies to end the HIV epidemic.
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**Categories:** News
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### [Groundbreaking Treatment: Elevidys Expands Access for Duchenne Patients](https://www.clinicaltrialvanguard.com/news/groundbreaking-treatment-elevidys-expands-access-for-duchenne-patients/)
**Published:** June 24, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has granted traditional approval to ELEVIDYS for ambulatory [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy (DMD) patients and accelerated approval for non-ambulatory patients.
ELEVIDYS is now indicated for individuals with DMD who are at least 4 years old and have a confirmed DMD gene mutation. The label expansion includes both ambulatory and non-ambulatory patients.
• Traditional approval for ambulatory patients confirms the functional benefits of ELEVIDYS.
• Accelerated approval for non-ambulatory patients is contingent upon continued verification of clinical benefit in a confirmatory trial.
ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
Sarepta Therapeutics, the manufacturer of ELEVIDYS, hailed the FDA approval as a significant milestone and a victory for gene therapy. The company praised the scientific evidence and the commitment of researchers, clinicians, and patient families.
Sarepta is conducting a postmarketing study, ENVISION, to confirm the clinical benefit of ELEVIDYS in non-ambulatory patients.
Sarepta is collaborating with Roche to develop innovative therapies for DMD, aiming to enhance muscle function and improve the quality of life for those affected.
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**Categories:** News
---
### [Scholar Rock Unveils Remarkable SRK-439 Findings: Preserving Lean Mass, Curbing Fat Gain](https://www.clinicaltrialvanguard.com/news/scholar-rock-unveils-remarkable-srk-439-findings-preserving-lean-mass-curbing-fat-gain/)
**Published:** June 25, 2024
**Author:** Jon Napitupulu
**Content:**
Scholar Rock, a biotechnology company, has initiated the Phase 2 EMBRAZE trial to evaluate [apitegromab](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/when-your-cmo-fails-an-fda-inspection-your-sites-pay-the-price/), a myostatin inhibitor, for preserving muscle mass in obese individuals receiving GLP-1 receptor agonist (GLP-1 RA) therapy. The trial will also assess apitegromab’s impact on weight loss maintenance after GLP-1 RA discontinuation.
Preclinical studies have demonstrated that [SRK-439](https://www.clinicaltrialvanguard.com/news/scholar-rock-announces-preclinical-data-for-srk-439/), a selective myostatin inhibitor optimized for cardiometabolic disorders, has the potential to increase lean mass and prevent fat mass regain after GLP-1 RA withdrawal. These findings were presented at the American Diabetes Association’s Scientific Sessions.
SRK-439 has shown promise as a potential treatment for [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), as it has demonstrated preservation of lean mass during GLP-1 RA-induced weight loss and attenuation of fat mass rebound following GLP-1 RA withdrawal. Its potency has also been shown to surpass that of an anti-ACTRII antibody.
Scholar Rock’s cardiometabolic program continues to progress, and the company anticipates providing further updates on SRK-439 and the EMBRAZE trial in the future. The goal of this research is to develop effective therapies for obesity and other cardiometabolic disorders where protein growth factors play a crucial role.
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**Categories:** News
---
### [Cybin's Triumph: Set For Novel Psychiatric Research](https://www.clinicaltrialvanguard.com/news/cybins-triumph-set-for-novel-psychiatric-research/)
**Published:** June 27, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin Inc., a biopharmaceutical company focused on developing treatments for mental health disorders, has achieved significant milestones in its research programs.
CYB003 for [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD)
• Received FDA Breakthrough Therapy Designation (BTD) for CYB003, a deuterated psilocybin analog.
• BTD accelerates the development process and provides increased FDA guidance on trial design.
• Phase 3 multinational study for CYB003 is expected to begin in summer 2024.
• Positive four-month durability data for CYB003 supports its potential therapeutic benefits.
[CYB004](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) for Generalized Anxiety Disorder (GAD)
• Initiated a Phase 2 study of CYB004, a deuterated dimethyltryptamine program.
• CYB004 is being developed for the treatment of GAD.
Financial Highlights
• Cash on hand totaled C$209 million as of March 31, 2024.
• Closed an oversubscribed private placement of U.S.$150 million.
Intellectual Property
• Strengthened intellectual property portfolio with over 60 granted patents and 200 pending applications.
Company Outlook
Doug Drysdale, CEO of Cybin, noted the company’s rapid progress and evolution into a late-stage company. He emphasized Cybin’s commitment to transformative treatments for mental health disorders and its accelerated path towards potential commercialization.
Cybin’s rigorous research approach aims to revolutionize the treatment of mental health disorders and improve patient outcomes. The company remains well-positioned to continue its regulatory engagement and clinical program advancements in the upcoming year.
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**Categories:** News
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### [DM199 (Rinvecalinase Alfa) Program Expanded by Diamedica Therapeutics into Preeclampsia](https://www.clinicaltrialvanguard.com/news/dm199-rinvecalinase-alfa-program-expanded-by-diamedica-therapeutics-into-preeclampsia/)
**Published:** June 27, 2024
**Author:** Jon Napitupulu
**Content:**
DiaMedica Therapeutics, a biotechnology company, is expanding its clinical development program for [DM199](https://www.clinicaltrialvanguard.com/news/diamedica-doses-first-patient-in-dm199-preeclampsia-trial/) into preeclampsia. Preeclampsia is a severe pregnancy-related condition characterized by high blood pressure and protein in the urine, posing risks to both the mother and the baby.
DM199 is a protein therapeutic that improves blood vessel function. It is believed to lower blood pressure and enhance blood flow to vital organs and the placenta. This mechanism of action is essential in preeclampsia, where reduced blood flow to the placenta contributes to the condition’s severity.
Phase 2 clinical trial results in chronic kidney disease patients with elevated blood pressure showed significant reductions in systolic blood pressure with DM199 treatment. Animal studies have also demonstrated the drug’s safety in pregnant individuals, with no evidence of placental transfer.
The planned preeclampsia trial is designed to be cost-effective, with an estimated enrollment of 120 participants and a budget of approximately $1.5 million. The trial aims to provide proof of concept for DM199’s potential to address preeclampsia.
If successful, DM199 could fill a significant unmet medical need in preeclampsia, where there are currently no approved therapies in the U.S. or Europe. By improving blood pressure control and enhancing placental perfusion, DM199 has the potential to improve outcomes for both mothers and babies affected by this life-threatening condition.
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**Categories:** News
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### [VIR Biotechnology Receives FDA Clearance and Fast Track Designation: Chronic Hepatitis Delta Treatment](https://www.clinicaltrialvanguard.com/news/vir-biotechnology-receives-fda-clearance-and-fast-track-designation-chronic-hepatitis-delta-treatment/)
**Published:** June 27, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has approved an Investigational New Drug (IND) application and granted Fast Track designation to the combination of tobevibart and elebsiran for chronic hepatitis delta treatment.
Tobevibart, a monoclonal antibody, and elebsiran, a small interfering ribonucleic acid, have shown promising Phase 2 preliminary results. They have demonstrated high rates of virologic response and ALT normalization in participants with chronic hepatitis delta.
The World Health Organization classifies hepatitis delta as the most severe chronic viral hepatitis, with a rapid progression toward [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/) and death. Approximately 12 million people worldwide suffer from this infection.
Vir Biotechnology, the company developing tobevibart and elebsiran, emphasizes the urgent need for effective treatments and their commitment to making this groundbreaking therapy accessible as soon as possible.
The Fast Track designation accelerates the development and review process for drugs addressing serious conditions with unmet medical needs.
An upcoming Phase 3 ECLIPSE trial will evaluate the safety and efficacy of the tobevibart and elebsiran combination against the current standard of care.
The SOLSTICE Phase 2 trial, with complete 24-week data expected in the fourth quarter, continues to assess the combination’s effectiveness.
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**Categories:** News
---
### [QPS Announces New Laboratory Services for Clinical Trials and Cell Therapy](https://www.clinicaltrialvanguard.com/news/qps-announces-new-laboratory-services-for-clinical-trials-and-cell-therapy/)
**Published:** June 27, 2024
**Author:** Jon Napitupulu
**Content:**
QPS, a prominent global contract research organization, has introduced enhanced laboratory services at its Springfield, Missouri unit. This expansion complements the company’s existing bioanalysis, translational medicine, and peripheral blood mononuclear cell (PBMC) laboratories, extending its comprehensive services.
The newly expanded clinical trials unit now offers clinical trial sample analysis and leukopak [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) products. Additionally, the Springfield facility now provides expanded PBMC services, complementing those offered at the Miami, Florida location.
Housed within the existing clinical laboratory, the modern central laboratory for sample analysis employs advanced technologies for chemistry, urinalysis, serology, coagulation, and hematology testing. This capability enables real-time monitoring of subject safety during clinical trials.
QPS has established a specialized leukopak products collection and processing facility to meet the growing demand for blood products supporting cell therapy research. The company’s extensive database of potential study participants facilitates the identification of blood donors for leukopak products.
The enhanced PBMC services include a new PBMC processing lab in Springfield to address the unmet need for such services in the Midwest. This lab enables timely PBMC sample analysis, a critical aspect of vaccine trials.
Brendon Bourg, Vice President of Early Phase Clinical and Head of Administration at QPS Missouri indicated that these new facilities and services strengthen their comprehensive CRO capabilities and that clients can now access a full range of clinical research services at a single location.
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### [COVID-19: Pfizer-BioNTech's New Omicron Vaccine Receives EU Approval](https://www.clinicaltrialvanguard.com/news/covid-19-pfizer-biontechs-new-omicron-vaccine-receives-eu-approval/)
**Published:** June 28, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer and [BioNTech](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/)‘s Omicron JN.1-adapted COVID-19 vaccine (COMIRNATY® JN.1) has received a positive recommendation for marketing authorization from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). The vaccine is intended for active immunization against COVID-19 caused by SARS-CoV-2 in individuals six months and older.
The vaccine adaptation aligns with recommendations from the World Health Organization and the European Medicines Agency, which suggest targeting the SARS-CoV-2 variant JN.1 in COVID-19 vaccination campaigns for the 2024-2025 season.
Clinical and non-clinical evidence supports the safety and efficacy of Pfizer and BioNTech’s COVID-19 vaccines, including the JN.1-adapted version. Pre-clinical data indicates that the JN.1-adapted vaccine generates an enhanced response against Omicron JN.1 sublineages, including KP.2, KP.3, and others, compared to the companies’ Omicron XBB.1.5-adapted vaccine.
The European Commission is expected to review the CHMP’s recommendation and make a final decision soon. The updated vaccine will be available for distribution to EU member states upon approval.
Pfizer and BioNTech have proactively manufactured the Omicron JN.1-adapted vaccine to ensure supply before the fall and winter season when demand for COVID-19 vaccination is anticipated to rise. The companies are also submitting rolling applications to the U.S. Food and Drug Administration to approve their Omicron KP.2-adapted COVID-19 vaccines for individuals six months and older.
Pfizer and BioNTech continue to monitor the evolving epidemiology of COVID-19 and are committed to meeting global public health needs through developing and distributing vaccines.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Thermo Fisher's Lab Expansion: A Boon for Kentucky's Health Research](https://www.clinicaltrialvanguard.com/news/thermo-fishers-lab-expansion-a-boon-for-kentuckys-health-research/)
**Published:** June 28, 2024
**Author:** Jon Napitupulu
**Content:**
Thermo Fisher Scientific’s PPD clinical research arm is significantly expanding its presence in Kentucky. The expansion entails a 65,000-square-foot facility in Covington, increasing the company’s laboratory footprint. This investment of $47.8 million will generate over 250 new positions in the region over the next eight years.
PPD has maintained a central laboratory operation in Highland Heights since 2002, offering biomarker operations, sample management, and testing for novel therapies. The Covington expansion will bolster these capabilities.
According to Leon Wyszkowski, president of Thermo Fisher’s analytical services division, the central laboratory is crucial in facilitating efficient clinical trial decisions and ensuring patient well-being. This expansion will enhance clients’ research and development initiatives.
Kentucky Governor Andy Beshear expressed enthusiasm for Thermo Fisher’s continued investment in the state, highlighting the growth in the healthcare and [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) sectors. He anticipates the company’s success in the region and its positive impact on the local economy.
The Highland Heights laboratory employs over 600 professionals, including scientific and support staff, providing safety testing, clinical sample management, and biomarker support. Thermo Fisher’s clinical research business also operates additional laboratories internationally, including facilities in Belgium, Singapore, Virginia, Wisconsin, Ireland, and China.
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### [Breakthrough Med: Epcoritamab Revolutionizes Follicular Lymphoma Treatment](https://www.clinicaltrialvanguard.com/news/breakthrough-med-epcoritamab-revolutionizes-follicular-lymphoma-treatment/)
**Published:** July 1, 2024
**Author:** Jon Napitupulu
**Content:**
The European Medicines Agency (EMA) has recommended granting marketing authorization for [epcoritamab](https://www.clinicaltrialvanguard.com/news/genmab-abbvie-clarify-epcoritamab-phase-3-trial-missed-primary-endpoint/), a monoclonal antibody for treating relapsed or refractory follicular [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (FL). This recommendation is based on positive Phase 1/2 EPCORE NHL-1 clinical trial results.
Epcoritamab is administered subcutaneously and demonstrated significant efficacy in patients with FL who had previously undergone multiple lines of therapy. The study included patients resistant to anti-CD20 antibody and alkylating agent treatments.
The most common side effects observed with epcoritamab were cytokine release syndrome (CRS), injection site reactions, and infections. To mitigate the risk of CRS, an optimized step-up dosing schedule was developed and evaluated, resulting in a lower incidence and severity of CRS.
The positive EMA opinion recognizes the unmet medical need for patients with difficult-to-treat FL. Epcoritamab offers a potential new therapeutic option for these patients.
“The EMA’s positive opinion is a significant step forward in providing a new treatment option for patients with relapsed or refractory follicular lymphoma,” said Jan van de Winkel, CEO of Genmab.
The European Commission will decide on epcoritamab’s authorization later this year. If approved, epcoritamab will be Europe’s first T-cell-engaging bispecific antibody for treating FL.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [TAURx Submits Extraordinary Application for HMTM as Alzheimer's Disease Breakthrough](https://www.clinicaltrialvanguard.com/news/taurx-submits-extraordinary-application-for-hmtm-as-alzheimers-disease-breakthrough/)
**Published:** July 3, 2024
**Author:** Jon Napitupulu
**Content:**
TauRx Pharmaceuticals has submitted a Marketing Authorisation Application (MAA) in the UK for hydromethylthionine mesylate (HMTM), a potential treatment for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease (AD). HMTM targets the accumulation of tau proteins, a hallmark of AD.
The application is supported by data from Phase 3 trials, including the LUCIDITY trial, which demonstrated benefits in cognitive function and reduced brain shrinkage. HMTM inhibits tau aggregation and enhances brain activity.
UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has designated HMTM for the Innovative Licensing and Access Pathway (ILAP), which could result in HMTM becoming the first oral treatment available in the UK specifically targeting tau pathology.
If approved, HMTM could offer a new treatment option for patients with mild cognitive impairment (MCI-AD) and mild to moderate AD. It has a strong safety profile and can be administered orally with minimal burden.
Dr. Claude Wischik, Executive Chairman of TauRx, emphasized the milestone, stating, “This step brings hope to patients and families affected by this devastating disease.”
HMTM is a potential oral therapy designed to target tau aggregation in AD. It also has a tau-independent mode of action, increasing acetylcholine levels, which is crucial for cognitive function.
The Phase 3 LUCIDITY trial showed a reduction in neurodegeneration as measured by Neurofilament Light Chain (NfL) and sustained improvement in cognitive function over 18 months in MCI patients.
About TauRx Pharmaceuticals Ltd
TauRx Pharmaceuticals was established in 2002 and is dedicated to developing treatments and diagnostics for Alzheimer’s disease. Its research facilities and operations are based in Aberdeen, UK.
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**Categories:** News
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### [Merck and Orion Exclusive Rights to Opevesostat for Metastatic Prostate Cancer](https://www.clinicaltrialvanguard.com/news/merck-and-orion-exclusive-rights-to-opevesostat-for-metastatic-prostate-cancer/)
**Published:** July 3, 2024
**Author:** Jon Napitupulu
**Content:**
Merck and Orion Corporation have converted their co-development and co-commercialization agreement for opevesostat into an exclusive global license for Merck. Opevesostat is an investigational CYP11A1 inhibitor being evaluated in Phase 3 trials for metastatic castration-resistant [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/).
Merck’s President of Research Laboratories, Dr. Dean Y. Li, highlighted the progress made in the collaboration, including the initiation of pivotal Phase 3 trials. Merck will lead the clinical development of opevesostat, aiming to meet the needs of patients with prostate cancer.
Orion’s President and CEO, Liisa Hurme, stated that the license agreement allows Orion to focus on its other development candidates while benefiting from the development and potential commercialization of opevesostat. Orion believes Merck is well-positioned to maximize the potential of opevesostat.
Under the terms of the agreement, Orion is entitled to receive various milestone payments, including development milestones up to $30 million, regulatory milestones up to $625 million, and sales-based milestones up to $975 million. Orion will also receive tiered royalty payments on net sales, ranging from a low double-digit rate to a rate in the low twenties.
Merck will assume responsibility for all past and future development and commercialization expenses. As a result, Orion will release €60 million reserved for development costs, which will be recognized as net sales and operating profit in Q3 2024. Orion remains responsible for manufacturing clinical and commercial supplies for Merck.
The exclusive global license is subject to regulatory approvals and is expected to become effective in the third quarter of 2024.
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**Categories:** News
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### [Acticor Biotech: Disclosure of Voting Rights, Shares May 2024](https://www.clinicaltrialvanguard.com/news/acticor-biotech-disclosure-of-voting-rights-shares-may-2024/)
**Published:** July 8, 2024
**Author:** Jon Napitupulu
**Content:**
[ACTICOR](https://www.clinicaltrialvanguard.com/news/acticor-biotech-secures-receivership-extension-for-glenzocimab-development/) BIOTECH, a clinical-stage pharmaceutical company, reports its voting rights and shares as of May 31, 2024. The company focuses on developing [glenzocimab](https://www.clinicaltrialvanguard.com/news/acticor-biotech-liquidation-proceedings-announced/), a drug for treating cardiovascular emergencies, particularly ischemic stroke.
Glenzocimab has shown promising results in Phase 1b/2a (ACTIMIS) and Phase 2/3 (ACTISAVE) clinical trials. ACTIMIS demonstrated glenzocimab’s safety profile and reduced mortality and intracerebral hemorrhage in stroke patients. AI brain imaging analysis confirmed these results, indicating a reduction in lesions.
Despite initial negative results on the primary endpoint (disability or death), ACTISAVE data presented at ESOC 2024 showed trends in returning to normal life, especially in patients with complete recanalization after mechanical thrombectomy.
ACTICOR BIOTECH is supported by European and international investors and has been listed on Euronext Growth Paris since November 2021. The company is actively involved in ongoing clinical trials and continues to evaluate glenzocimab’s potential in treating cardiovascular emergencies.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Announcing Calidi's Stock Split](https://www.clinicaltrialvanguard.com/news/announcing-calidis-stock-split/)
**Published:** July 8, 2024
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) has authorized a one-for-ten reverse stock split, effective July 15, 2024. The split will be applied to all outstanding common stock, reducing the total number of shares by a factor of ten.
Under the revised structure, Calidi’s common stock will continue trading under the ticker “CLDI” on the NYSE American. The CUSIP number will change to 320703 309.
The reverse split is part of the company’s efforts to enhance its financial flexibility and market appeal. The reduced number of shares will potentially improve liquidity and increase the stock’s attractiveness to institutional investors.
Stockholders who hold fractional shares following the split will receive a rounded-up whole share. Warrant holders will see proportional adjustments to their exercise prices and the number of underlying shares.
Calidi’s equity incentive plans and employee stock purchase plans will also undergo adjustments aligned with the reverse stock split ratio.
The split will not affect the total number of authorized shares or the par value per share. Calidi has designated Equiniti Trust Company as the exchange agent to facilitate the process.
Stockholders who held shares prior to the split will have their holdings consolidated. For every ten pre-split shares, they will receive one post-split share.
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**Categories:** News
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### [Genexine and EPD Bio Announce Merger, Paving the Way for Revolutionized Drug Development](https://www.clinicaltrialvanguard.com/news/genexine-and-epd-bio-announce-merger-paving-the-way-for-revolutionized-drug-development/)
**Published:** July 8, 2024
**Author:** Jon Napitupulu
**Content:**
Genexine, a Korean biopharmaceutical company, has merged with EPD [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/), specializing in targeted protein degradation (TPD) technology. This merger aims to bolster Genexine’s research capabilities and drug pipeline.
EPD Bio’s bioPROTAC platform, EPDegTM, offers several advantages over traditional small molecule-based [PROTAC](https://www.clinicaltrialvanguard.com/news/arvinas-protac-degrader-shows-positive-phase-1-parkinsons-data/) technology. It utilizes mRNA-LNP delivery to overcome tissue-specific limitations of E3 ligase expression, enabling the development of a diverse pipeline of TPDs for various undruggable targets.
The merger brings onboard Dr. Jaehyun Choi, EPD Bio’s founder and CEO, as Genexine’s representative director for R&D. This strengthens Genexine’s expertise in TPD technology and aligns with its goal of developing innovative drugs to address unmet medical needs.
Genexine’s focus on immunotherapeutics and next-generation biologics complements EPD Bio’s TPD platform. This combination allows Genexine to pursue the development of novel therapies for a range of diseases, including cancer, growth hormone deficiency, and CKD-induced anemia.
The merger is expected to expedite Genexine’s mission of discovering and commercializing effective biologics. With EPD Bio’s expertise and clinical and CMC development capabilities, Genexine aims to lead the global market in innovative drug development.
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**Tags:** eClinical Tech News
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### [Genentech Unveils Second Chance for Susvimo to Combat AMD](https://www.clinicaltrialvanguard.com/news/genentech-unveils-second-chance-for-susvimo-to-combat-amd/)
**Published:** July 9, 2024
**Author:** Jon Napitupulu
**Content:**
The Food and Drug Administration (FDA) has approved updates to Susvimo, an eye implant treatment for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (AMD). Susvimo provides an alternative to regular eye injections, the current standard of care.
Wet AMD affects approximately 1.5 million Americans and 20 million people worldwide. If left untreated, it can lead to blindness. Susvimo is the first and only approved treatment for wet AMD that maintains vision with just two refills per year.
Developed by Genentech, Susvimo is a refillable implant that continuously delivers a customized formulation of [ranibizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-susvimo-for-diabetic-blindness/) to the eye. The implant is surgically inserted during a one-time outpatient procedure and is refilled every six months.
The FDA approves the updated Susvimo based on component-level updates to the implant and refill needle. Genentech has conducted extensive testing to ensure that the implant and needle now meet performance standards.
Susvimo offers several advantages over traditional eye injections. It provides sustained delivery of medicine to the eye, reducing the need for frequent injections. Additionally, inserting and refilling the implant is less invasive than multiple eye injections.
Genentech is committed to helping patients access Susvimo. The company offers comprehensive services to minimize barriers to access and reimbursement. Patients can call 833-EYE-GENE for more information and to learn about patient assistance programs through Genentech Access Solutions.
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**Categories:** News
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### [Antidote Strengthens Clinical Trial Enrollment Collaboration](https://www.clinicaltrialvanguard.com/news/antidote-strengthens-clinical-trial-enrollment-collaboration/)
**Published:** July 9, 2024
**Author:** Jon Napitupulu
**Content:**
[SEQSTER](https://www.clinicaltrialvanguard.com/news/seqster-and-patientslikeme-partner-for-enhanced-patient-health-insights/) and Antidote have revolutionized patient matching and clinical research efficiency. SEQSTER’s patient-centric healthcare platform provides a comprehensive view of medical history, genomic data, and lifestyle factors. Antidote’s patient engagement platform accelerates clinical trial recruitment.
By integrating SEQSTER’s technology, Antidote can accurately match patients to clinical trials, leveraging a holistic understanding of their health profiles. This accelerates recruitment and ensures better patient quality. Patients benefit from more precise matches to potential clinical trials for which they qualify.
Furthermore, the partnership enables real-time data access to study participant health data, streamlining the path to medical advancements. CEO and Co-Founder of SEQSTER, Ardy Arianpour, emphasizes the transformative potential of this partnership in addressing the challenges of clinical trial recruitment and participant engagement.
Samantha Veeck, Co-CEO of Antidote, highlights the importance of connecting suitable patients to the right trials, combining advanced technology with a personalized approach. Antidote’s precision recruitment process is enhanced by SEQSTER’s data integration capabilities, optimizing the identification of suitable participants and reducing the time to market for new treatments.
The partnership also promotes diversity in drug development. Providing access to patient medical records ensures better representation of underserved populations in clinical trials. This meets FDA diversity requirements and improves the generalizability of research findings.
Integrating SEQSTER’s and Antidote’s technologies simplifies patient enrollment, reducing the time to initiate clinical studies. Antidote’s focus on personalized patient services and SEQSTER’s technology enhance patient engagement, promoting adherence to trial protocols and accurate data collection. This partnership empowers researchers with the tools and insights needed for successful clinical research and the development of innovative treatments.
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**Tags:** eClinical Tech News
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### [Obsidian's OBX-115 T-Cell Therapy For Melanoma Receives FDA Fast Track](https://www.clinicaltrialvanguard.com/news/obsidians-obx-115-t-cell-therapy-for-melanoma-receives-fda-fast-track/)
**Published:** July 10, 2024
**Author:** Jon Napitupulu
**Content:**
Obsidian Therapeutics has secured Fast Track Designation from the FDA for OBX-115, an engineered T-[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) targeting metastatic or locally advanced melanoma resistant to PD-1/PD-L1 inhibitors.
OBX-115, based on patient-derived tumor-infiltrating lymphocytes (TILs), is enhanced with membrane-bound interleukin-15 (mbIL15), enabling pharmacological regulation. This potentially addresses the unmet need for effective treatments for melanoma patients who have progressed after ICI therapy.
The Fast Track Designation expedites the development and review process for promising new drugs addressing serious conditions. It allows for frequent interactions between Obsidian and the FDA and, upon meeting specific criteria, potential eligibility for Priority and Rolling Review.
OBX-115 is undergoing clinical trials in melanoma and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). The company’s proprietary manufacturing process aims to improve the persistence, antitumor activity, and safety of TIL cell therapy.
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**Categories:** News
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### [Pluristyx and Humacyte Announce Partnership](https://www.clinicaltrialvanguard.com/news/pluristyx-and-humacyte-announce-partnership/)
**Published:** July 10, 2024
**Author:** Jon Napitupulu
**Content:**
Pluristyx, a renowned cellular therapy developer, has partnered with Humacyte, a biotechnology company specializing in bioengineered human tissue. This collaboration will leverage Pluristyx’s advanced stem cell technology to create insulin-producing cells for Humacyte’s BioVascular Pancreas (BVP) therapy.
Pluristyx’s PluriBank™ stem cell line, renowned for its clinical-grade purity, genetic integrity, and safety features, will serve as the foundation for Humacyte’s efforts. Additionally, Pluristyx’s panCELLa™ platform enables the generation of “hypoimmune” cells, minimizing the risk of immune rejection after implantation.
Through this partnership, Pluristyx will provide access to their custom gene editing services, enhancing the BVP’s potential efficacy and safety. Dr. Benjamin Fryer, Pluristyx’s CEO, emphasized their commitment to supporting therapeutic developers in advancing cell-based therapies.
The BVP, engineered to revolutionize [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/) treatment, involves the delivery of insulin-producing islets within the body using Humacyte’s Acellular Tissue Engineered Vessel (ATEV™). The ATEV and BVP are currently investigational products awaiting regulatory approval.
Pluristyx’s panCELLa™ platform, combining cutting-edge iPSC technology and genetic engineering capabilities, empowers the development of innovative cell therapies. The company’s end-to-end support accelerates clinical trials and facilitates the commercialization of cell-based treatments.
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**Tags:** eClinical Tech News
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### [Pfizer: New Formulation of Oral GLP-1 Agonist Advances to Development Stage](https://www.clinicaltrialvanguard.com/news/pfizer-new-formulation-of-oral-glp-1-agonist-advances-to-development-stage/)
**Published:** July 12, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer has selected a once-daily formulation of danuglipron, an oral GLP-1 receptor agonist, based on encouraging results from an ongoing pharmacokinetic study. The company aims to optimize dosing through studies in the second half of 2024 before initiating registration-enabling trials.
Pfizer emphasizes the importance of [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) as a therapeutic focus, with danuglipron being the most advanced candidate in its robust pipeline. Mikael Dolsten, Pfizer’s Chief Scientific Officer, believes that the once-daily formulation has the potential to provide a competitive edge in the GLP-1 space.
The ongoing study has assessed the pharmacokinetics and safety of immediate- and modified-release danuglipron formulations in healthy adults. Results indicate a once-daily dosing potential, with a favorable safety profile consistent with previous studies. No liver enzyme elevations have been observed in over 1,400 participants.
Danuglipron is an investigational medicine designed to regulate blood sugar levels by increasing insulin release. It may also slow digestion and enhance feelings of fullness after eating, potentially aiding in weight loss. However, it is essential to note that danuglipron is not currently approved by health authorities.
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**Categories:** News
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### [City of Hope and Mount Sinai Unlock New Treatment for Diabetes](https://www.clinicaltrialvanguard.com/news/city-of-hope-and-mount-sinai-unlock-new-treatment-for-diabetes/)
**Published:** July 15, 2024
**Author:** Jon Napitupulu
**Content:**
Recent research published in Science Translational Medicine offers promising advancements in developing regenerative therapies for diabetes. A team of scientists from City of Hope and Mount Sinai Health System has discovered a therapeutic combination that stimulates the regeneration of human beta cells, the insulin-producing cells critical for blood sugar regulation.
The research involved transplanting a few human beta cells into mice with type 1 and 2 diabetes models. The mice were then treated with a combination of harmine, a natural product found in plants, and GLP1 receptor agonists, a common [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) medication. Strikingly, this combination therapy led to a 700% increase in human beta cell numbers within three months.
Adolfo Garcia-Ocaña, the lead author of the study, indicated that this breakthrough represents the first time a drug treatment has been proven to enhance adult human beta cell production in vivo and that this discovery offers hope for developing regenerative therapies that could benefit millions of people living with diabetes.
The research team’s findings build upon earlier work published in Nature Medicine in 2015, which identified harmine as a potential candidate for beta cell regeneration. Through extensive animal models and drug treatment studies, the team has further validated this combination therapy’s therapeutic potential.
The ability to regenerate beta cells holds significant implications for diabetes treatment. Current therapies focus on managing blood sugar levels through insulin supplementation or medications that stimulate insulin production. However, these treatments do not address the underlying cause of diabetes, which is the loss of beta cells. By regenerating beta cells, therapies that target this combination could offer a potential cure or long-term remission for diabetes patients.
Further research is necessary to translate these findings into human trials and develop effective treatments. However, the current study raises hope for the possibility of regenerative therapies as a transformative approach to diabetes management.
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**Tags:** eClinical Tech News
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### [Ebglyss Receives NICE Recommendation for Moderate to Severe Atopic Dermatitis](https://www.clinicaltrialvanguard.com/news/ebglyss-receives-nice-recommendation-for-moderate-to-severe-atopic-dermatitis/)
**Published:** July 15, 2024
**Author:** Jon Napitupulu
**Content:**
The National Institute for Health and Care Excellence (NICE) has recommended Ebglyss (lebrikizumab) as an additional treatment option for moderate to severe [Atopic Dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) in England. Lebrikizumab is indicated for adults and adolescents (12 years and older) with moderate-to-severe Atopic Dermatitis who are candidates for systemic therapy.
The recommendation is significant as it provides eligible patients with another treatment option for this common skin condition. Moderate to severe Atopic Dermatitis affects approximately 7.7% of adults and 18% of children in the United Kingdom. It can have significant physical and psychosocial impacts on patients and their families, including itching, bleeding skin, and social stigma.
Ebglyss has demonstrated efficacy in clinical trials, with a favorable safety profile and 4-weekly maintenance dosing. It is a targeted biological therapy that addresses the underlying inflammation in Atopic Dermatitis, relieving symptoms and improving quality of life.
Experts in the field have welcomed the NICE recommendation. Andrew Proctor, Chief Executive of the National Eczema Society, emphasizes the importance of having a range of treatment options for Atopic Dermatitis. Jorgen Damsbo, General Manager at Almirall, UK, highlights the company’s commitment to transforming patients’ lives through innovative treatments.
Prof Tony Bewley, Consultant Dermatologist, emphasizes the significant psychosocial burden of Atopic Dermatitis and the potential benefits of targeted biological therapies like Ebglyss. He notes the encouraging advancements in drug development for this chronic condition and the importance of providing patients with effective and accessible treatment options.
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**Categories:** News
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### [OPM Records Unprecedented Success in Phase 1 of OPM-101 Human Trials](https://www.clinicaltrialvanguard.com/news/opm-records-unprecedented-success-in-phase-1-of-opm-101-human-trials/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
[Oncodesign](https://www.clinicaltrialvanguard.com/news/oncodesigns-precision-medicine-a-new-era-in-systemic-radiotherapy/) Precision Medicine (OPM) has announced positive results from a Phase 1 trial evaluating [OPM-101](https://www.clinicaltrialvanguard.com/news/opm-101-ripk2-inhibitor-shows-strong-safety-no-cardiac-toxicity-in-phase-1-study/), a selective small molecule inhibitor targeting the RIPK2 kinase. The trial assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of OPM-101 in healthy volunteers.
The randomized, double-blind, placebo-controlled study (EudraCT: 2022-003122-50) enrolled 104 volunteers, 78 treated with OPM-101, and 26 received placebo. OPM-101 was administered orally in single ascending doses (SAD) and multiple ascending doses (MAD).
Key findings include:
• OPM-101 was well-tolerated and significantly inhibited the RIPK2 pathway at low doses (60 mg single administration and 75 mg twice daily in MAD).
• Target engagement was observed within 2 to 4 hours after the first administration, sustaining inhibition for 14 days.
• MAD administrations demonstrated maximum target engagement, leading to a 90-100% reduction in TNFα production. This immunomodulatory effect was observed without total suppression of immunity.
The results suggest that OPM-101 can potentially treat inflammatory diseases like IBD (Chronic Inflammatory Bowel Disease) and immuno-oncology. OPM is currently investigating OPM-101’s efficacy in multiple clinical trials for various inflammatory diseases.
The RIPK2 kinase is crucial in regulating immune responses and inflammatory processes, making it a promising therapeutic target. OPM-101’s ability to selectively inhibit RIPK2 and modulate pro-inflammatory signaling pathways highlights its potential for treating various immune-related conditions.
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**Categories:** News
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### [Samantree Medical Secures $14 Million to Revolutionize Surgical Care Globally](https://www.clinicaltrialvanguard.com/news/samantree-medical-secures-14-million-to-revolutionize-surgical-care-globally/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
SamanTree Medical, a leading medical technology company specializing in surgical advancements, has secured $14 million in a Series B financing round. The funds will be allocated to advance and market their groundbreaking Histolog® Scanner, boost their presence in Europe and the US and enhance their digital offerings.
The Histolog Scanner, a CE-marked device, offers real-time intra-operative tissue assessment during surgery through high-resolution imaging. This allows pathologists and surgeons to make more informed decisions faster than traditional methods.
Relyens Innovation Santé, advised by Turenne Capital, led the funding round, with participation from Mutuelles Impact, Wille Finance, Noshaq, and WE [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/), as well as existing investors Panakès Partners, BOM, and b2venture. SamanTree Medical will establish its headquarters and operations base in Wallonia, Belgium, while retaining its R&D facilities in Lausanne, Switzerland.
Claire Poulard, Investment Director at Turenne Capital, and Henry Charlton, SVP and Chief Commercial Officer at Intuitive, have joined the Board of Directors. The company is optimistic that the funding and strengthened leadership will accelerate innovation, global expansion, and digital solution enhancement, ultimately improving patient outcomes.
The Walloon investors WE Life Sciences and Noshaq have expressed their ongoing support for SamanTree Medical and their commitment to strengthening the local life sciences ecosystem. The company’s focus on advancing surgical techniques with technology underscores its commitment to revolutionizing the field of surgery.
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**Categories:** News
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### [Elixia: A Revolution in Clinical Research](https://www.clinicaltrialvanguard.com/news/elixia-a-revolution-in-clinical-research/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
American Clinical Research Services, now known as Elixia, has rebranded to reflect its expanded clinical research and patient recruitment capabilities. This change represents the company’s mission to accelerate drug development and improve patient outcomes.
Elixia combines the expertise of premier clinical research sites with a patient recruitment engine. This allows it to conduct clinical trials with efficiency and precision. Through strategic acquisitions, Elixia has strengthened its services and positioned itself as a key industry player.
With facilities in multiple states, Elixia studies various conditions, including [metabolic health](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/), nephrology, neuroscience, and infectious diseases. Its technological advancements enhance patient recruitment and provide sponsors with critical insights.
Elixia remains committed to its partners, patients, and stakeholders as it enters this new phase. The company’s commitment to drug development and healthcare delivery is evident in its expanded capabilities and integrated approach. With its robust network and expertise, Elixia is poised to significantly impact the healthcare landscape by driving innovations that bring new treatments to patients faster and more effectively.
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**Categories:** News
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### [Phesi Data Emerging Clinical Trial Shift to Diabetes](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
Recent data from Phesi, a clinical development analytics company, reveals a significant shift in medical research priorities. [Type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) has emerged as one of the top five most studied diseases globally in the first half of 2024, surpassing COVID-19. Breast cancer, solid tumors, stroke, and [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) are the top five.
This change coincides with a surge in investment in GLP-1 therapies, such as Ozempic and WeGovy, to address the rising prevalence of obesity. Over 40% of US adults and more than one-quarter of UK adults are now classified as obese.
Phesi’s report highlights another concerning trend: increased clinical trial attrition rates. Approximately 32% of Phase II trials were terminated in the first half of 2024, a significant 56% rise compared to pre-pandemic levels.
This data was gathered from an analysis of 66,935 clinical trials utilizing Phesi’s Trial Accelerator™ platform, incorporating data from over 120 million patients in numerous cohorts.
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**Categories:** News
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### [Orum and Vertex Partner to Conquer Disease](https://www.clinicaltrialvanguard.com/news/orum-and-vertex-partner-to-conquer-disease/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
Orum Therapeutics and Vertex Pharmaceuticals have agreed to develop novel targeted conditioning agents for gene editing using Orum’s Dual-Precision Targeted Protein Degradation (TPD²) technology.
Vertex can exclusively license and commercialize DACs developed with Orum’s TPD² technology for up to three targets. In return, Orum will receive an upfront payment of $15 million and potential option payments, milestones, and tiered royalties totaling up to $310 million per target.
Orum’s TPD² technology is a platform for targeting specific proteins for degradation. It can potentially create a new class of drugs called degrader-antibody conjugates (DACs), which could treat various diseases by targeting and degrading specific proteins.
Vertex is a leader in gene editing, having received the first FDA approval for a [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)/Cas9 gene-edited therapy. Orum’s TPD² technology will enable Vertex to explore novel conditioning agents that could enhance the efficacy and safety of gene editing therapies.
This collaboration combines Orum’s expertise in targeted protein degradation with Vertex’s strength in gene editing. It aims to pave the way for novel treatment options for patients in a new therapeutic area.
Vertex is a global biotechnology company that develops innovative medicines for serious diseases. Orum Therapeutics is a clinical-stage biotechnology company specializing in degrader-antibody conjugates for protein degradation.
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**Categories:** News
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### [Predicta Biosciences Secures $5.2 Million Seed Funding for Non-Invasive Multiple Myeloma Diagnostics](https://www.clinicaltrialvanguard.com/news/predicta-biosciences-secures-5-2-million-seed-funding-for-non-invasive-multiple-myeloma-diagnostics/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
Predicta Biosciences, a company focused on precision oncology, has secured an oversubscribed seed round of $5.2 million. The round was led by The Engine Ventures, with Illumina Ventures, Time Boost Capital, American Cancer Society Bright Edge, and the Oetgen family as participating investors.
Founded by leading cancer genomics researchers and clinicians, Predicta is developing a novel, non-invasive diagnostic for multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/). This diagnostic aims to provide more comprehensive information on patients’ disease status, including their immune system cells. This information can aid physicians in determining patients’ likelihood of responding to immunotherapies, such as [CAR T-cell therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/)[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) and bispecific antibodies.
The company’s diagnostic platform leverages sophisticated algorithmic analysis for risk stratification and treatment optimization. It is designed to replace painful bone marrow biopsies with simple blood draws, offering a less invasive and more convenient approach to diagnosis and monitoring.
Predicta’s first product, GenoPredicta, is currently being used in research at Dana Farber Cancer Institute. The company plans to launch the diagnostic product commercially in the future. Predicta aims to improve patient outcomes and advance precision medicine in cancer treatment by addressing the unmet need for non-invasive diagnostics in multiple myeloma.
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**Categories:** News
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### [Scorpion Therapeutics Secure $150M for Cancer-Fighting Pipeline](https://www.clinicaltrialvanguard.com/news/scorpion-therapeutics-secure-150m-for-cancer-fighting-pipeline/)
**Published:** July 17, 2024
**Author:** Jon Napitupulu
**Content:**
Scorpion Therapeutics, a leading oncology company, announced a successful $150 million Series C financing round to advance its precision oncology pipeline. Frazier [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) and Lightspeed Venture Partners co-led the financing, with significant participation from new and existing investors.
Scorpion’s small molecule discovery engine has generated a robust pipeline, including the promising STX-478, a mutant-selective PI3Kα inhibitor targeting [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) and other solid tumors. The company plans to use the proceeds to further the clinical development of STX-478 as a monotherapy and in combination with standard treatments.
The strong investor demand reflects Scorpion’s clinical execution, encouraging clinical data, and the strength of its pipeline. CEO Adam Friedman, M.D., Ph.D., expressed delight in strengthening the company’s financial position and expanding its investor syndicate with notable figures in the life sciences industry.
Frazier Life Sciences’ Dr. Albert Cha praised Scorpion’s track record in developing innovative cancer therapies. He believes the company’s progress, promising pipeline and experienced leadership team position it for success in delivering effective treatments to cancer patients.
Lightspeed’s Dr. Shelley Chu commended Scorpion’s progress and highlighted the potential of STX-478 to address the limitations of existing cancer treatments. The targeted approach of Scorpion’s therapies aims to improve efficacy and safety outcomes for patients.
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**Categories:** News
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### [Bayer's Nubeqa Triumph: A Milestone in Prostate Cancer Treatment](https://www.clinicaltrialvanguard.com/news/bayers-nubeqa-triumph-a-milestone-in-prostate-cancer-treatment/)
**Published:** July 18, 2024
**Author:** Jon Napitupulu
**Content:**
The Phase III ARANOTE trial has demonstrated significant benefits for NUBEQA (darolutamide) in combination with androgen deprivation therapy (ADT) for patients with metastatic hormone-sensitive [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) (mHSPC). The study met its primary endpoint of extended radiological progression-free survival (rPFS) compared to placebo plus ADT.
NUBEQA, an androgen receptor inhibitor, has shown a clinically meaningful increase in rPFS, indicating a delay in tumor growth and spread. The results align with its established safety profile, with no new safety concerns identified.
The study included 669 patients randomized to receive either NUBEQA or placebo twice daily in addition to ADT. The primary endpoint was the time until disease progression or death.
In addition to rPFS, the study also assessed other outcomes such as overall survival, time to treatment failure, and quality of life. Detailed results are expected to be presented at a future scientific conference.
“These positive Phase III results provide physicians with more options to tailor treatment plans for patients with mHSPC,” said Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division.
Bayer plans to submit the data to the U.S. FDA for regulatory approval, expanding the potential use of NUBEQA in the treatment of prostate cancer.
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**Categories:** News
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### [Crescita Therapeutics Partners with NanoPass to Enhance Medical Aesthetics Portfolio](https://www.clinicaltrialvanguard.com/news/crescita-therapeutics-partners-with-nanopass-to-enhance-medical-aesthetics-portfolio/)
**Published:** July 18, 2024
**Author:** Jon Napitupulu
**Content:**
Crescita Therapeutics and NanoPass have entered an exclusive distribution agreement to introduce MicronJetTM600, an advanced intradermal delivery device, to the Canadian market. MicronJet’s innovative design leverages MEMS technology to provide a consistent, effective, and painless delivery of aesthetic products and therapeutic substances.
Featuring three 0.6mm silicon pyramids, MicronJet enables precise administration to delicate areas such as the face, neck, and décolleté. It eliminates bruising and discomfort, providing a comfortable injection experience for practitioners and patients.
Crescita will pursue regulatory approval from [Health Canada](https://www.clinicaltrialvanguard.com/news/amo-pharma-aligns-with-fda-mhra-health-canada-on-amo-02-study-design-for-cdm1/) and anticipates the product launch in the first half of 2025. MicronJet has received worldwide recognition, with over 70 clinical studies and peer-reviewed publications supporting its efficacy. It is CE-marked, FDA-cleared, ISO 13485 certified, and approved in Australia and other regions.
Crescita Therapeutics (TSX: CTX, OTC US: CRRTF) is a Canadian dermatology company that commercializes innovative, science-based products. NanoPass is a pioneer in intradermal delivery technology, developing cutting-edge devices to enhance the effectiveness and precision of aesthetic treatments.
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**Categories:** News
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### [NIH, FDA Effort Unveils Revolutionary Parkinson's Diagnosis Advancements](https://www.clinicaltrialvanguard.com/news/nih-fda-effort-unveils-revolutionary-parkinsons-diagnosis-advancements/)
**Published:** July 18, 2024
**Author:** Jon Napitupulu
**Content:**
A new partnership aims to improve diagnosis and outcomes for [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease and related disorders. The Accelerating Medicines Partnership in Parkinson’s Disease and Related Disorders (AMP PDRD) brings together expertise from the National Institutes of Health, Food and Drug Administration, The Michael J. Fox Foundation, and private stakeholders.
With a commitment of $21 million, the partnership aims to develop tools to differentiate Parkinson’s disease from similar conditions like multiple system atrophy and Lewy body dementia. Early diagnosis is crucial for effective treatment and improved outcomes.
Parkinson’s disease is a growing health concern due to increased life expectancy. Currently affecting over one million Americans and 8.5 million worldwide, Parkinson’s has no cure. Symptoms include tremors, muscle rigidity, slow movement, and non-motor issues like mood changes and cognitive decline.
Risk factors for Parkinson’s include age, genetics, and environmental hazards. While traditional diagnosis relies on medical history, neurological exams, and imaging tests, misdiagnosis rates are high. Researchers have recently developed a promising test that detects abnormal levels of alpha-synuclein, a characteristic protein of Parkinson’s.
AMP PDRD seeks to identify additional biomarkers, especially in accessible tissues like blood or saliva. Accurate diagnosis using these biomarkers can lead to earlier interventions and better outcomes for patients and families affected by Parkinson’s and related disorders.
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**Categories:** News
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### [Leo Pharma Reveals Groundbreaking Phase 3 Success for Hand Eczema](https://www.clinicaltrialvanguard.com/news/leo-pharma-reveals-groundbreaking-phase-3-success-for-hand-eczema/)
**Published:** July 22, 2024
**Author:** Jon Napitupulu
**Content:**
LEO Pharma, a leader in dermatology, has announced the publication of its DELTA 1 and DELTA 2 phase 3 trial findings for [delgocitinib](https://www.clinicaltrialvanguard.com/news/anzupgo-delgocitinib-cream-approved-in-great-britain/) cream in The Lancet, a renowned peer-reviewed medical journal. This publication marks a significant advancement in understanding and treating chronic hand eczema (CHE).
The DELTA 1 and DELTA 2 trials evaluated the safety and effectiveness of delgocitinib cream against a cream vehicle in patients with moderate to severe CHE who had limited response to traditional treatments. The results, published in The Lancet, demonstrated that delgocitinib cream met its primary and secondary endpoints, indicating its potential as a promising new treatment option.
The findings of the DELTA trials highlight the growing research and awareness surrounding CHE. Dr. Robert Bissonnette, Lead Author and MD from Innovaderm Research, Montreal, emphasized the importance of these publications in bringing attention to the condition and facilitating further scientific advancements.
Delgocitinib cream is currently under investigation and is awaiting approval from health authorities. LEO Pharma believes its potential to alleviate the symptoms and improve the quality of life for patients with CHE is significant. The company is eager to share the research findings with the scientific community to inspire further research and drive progress in CHE management.
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**Categories:** News
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### [Data Unveils Susvimo's Enduring Power in Treating Diabetic Eye Diseases](https://www.clinicaltrialvanguard.com/news/data-unveils-susvimos-enduring-power-in-treating-diabetic-eye-diseases/)
**Published:** July 22, 2024
**Author:** Jon Napitupulu
**Content:**
Susvimo, an innovative refillable eye implant, has shown promising results in Phase III clinical trials for treating DME and DR, vision-threatening conditions related to diabetes.
Over two years, Susvimo demonstrated sustained effectiveness in maintaining visual acuity in DME patients and improving DRSS scores in DR patients. Its safety profile remained consistent with previous findings.
The FDA has accepted Genentech’s sBLA for Susvimo, based on one-year data from the Pagoda and Pavilion studies. These studies demonstrated significant improvements in visual outcomes for both conditions.
Susvimo’s unique mechanism of action involves continuous delivery of [ranibizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-susvimo-for-diabetic-blindness/) to the eye, eliminating the need for frequent injections. In DME studies, approximately 95% of patients receiving Susvimo required no supplemental injections during the six-month dosing interval.
Susvimo’s nine-month dosing interval for DR patients led to superior improvements in DRSS scores compared to traditional observation. Importantly, no Susvimo patients required additional injections.
Dr. Levi Garraway, Genentech’s chief medical officer, emphasized the potential of Susvimo to revolutionize the treatment of diabetic eye diseases. If approved by the FDA, it could provide a more convenient and effective approach to preserve vision and maintain independence in affected individuals.
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**Categories:** News
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### [Parker Institute Pledges $125M for Cancer Cure Quest](https://www.clinicaltrialvanguard.com/news/parker-institute-pledges-125m-for-cancer-cure-quest/)
**Published:** July 22, 2024
**Author:** Jon Napitupulu
**Content:**
The Parker Institute for Cancer Immunotherapy (PICI) has announced a significant $125 million investment to bolster its research and accelerate the creation of groundbreaking cancer immunotherapies. This funding represents PICI’s largest commitment since its establishment in 2016.
The investment will fuel PICI’s collaborative research model, establishing seven research centers at leading academic institutions, including Gladstone Institutes, Dana-Farber Cancer Institute, and Weill Cornell Medicine. These centers and founding institutions, such as Stanford University and the University of Pennsylvania, form the PICI Network, fostering collaboration among top-tier cancer research institutions.
PICI’s investment will support research grants distributed over five years to Network researchers. This funding will accelerate groundbreaking research and expand PICI’s reach to world-class cancer centers like Memorial Sloan Kettering, MD Anderson Cancer Center, and Fred Hutchinson Cancer Center.
PICI’s research model emphasizes cross-disciplinary collaboration and eliminates barriers to scientific progress. It has generated over 440 research projects and clinical trials involving a substantial team of scientists. PICI also supports career advancement for early-career researchers and promotes diversity and inclusion in scientific communities.
In addition to research, PICI has invested in 17 biotech start-ups focused on innovative cancer immunotherapies. Profits from these investments will be reinvested into research grants, creating a sustainable cycle of innovation and discovery.
Recent PICI research breakthroughs highlighted at scientific conferences have demonstrated advances in [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)-based immunotherapy, engineered T cells, and glioma vaccines. The $125 million investment will enable PICI to continue driving these scientific breakthroughs and accelerate the development of life-saving cancer immunotherapies.
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**Categories:** News
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### [Bristol Myers Squibb's Validation for Breakthrough Cancer Treatment](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibbs-validation-for-breakthrough-cancer-treatment/)
**Published:** July 22, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb’s immunotherapy combination therapy, Opdivo plus Yervoy, has received validation from the European Medicines Agency (EMA) for its application as a first-line treatment for unresectable or advanced hepatocellular carcinoma (HCC) in adults who have not received prior systemic therapy.
The application is supported by positive results from the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -9DW trial, which demonstrated improved overall survival with Opdivo and Yervoy compared to lenvatinib or [sorafenib](https://www.clinicaltrialvanguard.com/news/cares-310-study-final-analysis-published-in-the-lancet/) alone. The validation of the application marks the start of the EMA’s centralized procedure review to evaluate the potential of this combination therapy for HCC patients.
With an estimated 62,000 HCC cases diagnosed annually in the European Union, its prevalence highlights the need for effective treatments in advanced stages. The promising results of the CheckMate -9DW trial suggest that Opdivo plus Yervoy has the potential to provide a new treatment option with improved clinical outcomes.
The combination of Opdivo and Yervoy exhibited a manageable safety profile consistent with previous data without any new safety concerns identified. The positive results were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.
Bristol Myers Squibb acknowledges the contributions of patients and investigators involved in the CheckMate -9DW clinical trial, which evaluated the effectiveness of Opdivo plus Yervoy compared to lenvatinib or sorafenib in untreated HCC patients. The combination therapy aims to improve survival outcomes and reduce symptom deterioration, offering hope to patients with this aggressive cancer.
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### [JCR Pharmaceuticals' Innovative Gene Therapy Research Unveiled at Lysosomal Forum](https://www.clinicaltrialvanguard.com/news/jcr-pharmaceuticals-innovative-gene-therapy-research-unveiled-at-lysosomal-forum/)
**Published:** July 22, 2024
**Author:** Jon Napitupulu
**Content:**
JCR Pharmaceuticals unveiled significant advancements in gene therapy research at the International Forum of Lysosomal Disorders. The company’s proprietary J-Brain Cargo® technology, modified to enhance drug delivery to the central nervous system, showed promising results in non-clinical trials.
The modified adeno-associated virus (AAV) vector successfully delivered therapeutic genes to the brains of mice and monkeys. Unlike traditional AAV vectors, which accumulate in the liver, J-Brain Cargo® significantly reduced liver toxicity.
Established in 2007, the forum brings together global leaders in lysosomal storage disorder research. Its mission is to foster international collaboration and advance research in this field.
JCR Pharmaceuticals’ J-Brain Cargo® technology enables the penetration of [biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) into the central nervous system, crossing the blood-brain barrier. IZCARGO®, approved in Japan for treating MPS II, is the first drug developed using this technology.
JCR Pharmaceuticals, a global specialty pharmaceutical company, focuses on rare and genetic diseases. With its headquarters in Japan, the company operates in the US, Europe, and Latin America. JCR’s approved products include treatments for growth disorders, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. Its pipeline includes MPS I, MPS II, and MPS IIIA and B investigational products.
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**Categories:** News
---
### [NuView Liquid Biopsy: High Tech, High Impact, High Reliability](https://www.clinicaltrialvanguard.com/executiveinterviews/nuview-liquid-biopsy-high-tech-high-impact-high-reliability/)
**Published:** July 23, 2024
**Author:** Moe Alsumidaie
**Content:**
In an exclusive interview with Paul Crowe, CEO of [NuView](https://www.clinicaltrialvanguard.com/executiveinterviews/pioneering-the-first-lab-and-theranostic-platform-a-conversation-with-nuview-ceo-paul-crowe/), we delve into his perspectives driving the upcoming Phase 2 clinical trial of NV-VPAC1, a groundbreaking liquid biopsy technology designed to detect the overexpression of VPAC1 in shed urine. This trial is poised to involve a diverse cohort of participants, including those with elevated PSA levels and individuals undergoing conventional diagnostic procedures for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/). Key elements of the trial design include stringent participant selection criteria, non-invasive urine sample collection, and the innovative analysis of VPAC1 receptors using the proprietary NV-VPAC1 technology.
#### [](#what-are-the-key-design-elements-of-your-upcoming-phase-2-clinical-trial-for-the-liquid-biopsy-to-detect-prostate-cancer-in-shed-urine)**What are the key design elements of your upcoming Phase 2 clinical trial for the liquid biopsy to detect prostate cancer in shed urine?**
Our upcoming Phase 2 clinical trial is designed to assess the efficacy and accuracy of NV-VPAC1, our liquid biopsy technology for detecting the overexpression of VPAC1 in shed urine. The trial will involve a diverse cohort of participants, including those with elevated PSA levels and those undergoing standard diagnostic procedures for prostate cancer. Key design elements include specific participant selection criteria, non-invasive urine sample collection, and the analysis of VPAC1 receptors in biofluids using the NuView proprietary NV-VPAC1 technology. Comparative analysis will be conducted against traditional diagnostic methods and histopathological findings from biopsies to establish sensitivity, specificity, and overall diagnostic accuracy. By incorporating these elements, we aim to evaluate our liquid biopsy’s performance comprehensively.
#### [](#how-will-you-ensure-the-robustness-and-reliability-of-the-trial-results)**How will you ensure the robustness and reliability of the trial results?**
The trial will be randomized and, where possible, blinded to reduce bias. Advanced statistical methods will be employed to analyze data and validate results. Control groups, with efforts to balance the homogeneity of patient populations across different trial sites, will be included for comparative analysis against standard diagnostic methods. An independent committee will review the trial design and results to ensure objectivity and compliance with regulatory standards. Additionally, we will conduct multi-center trials to validate the reproducibility of results across different populations and settings. These measures will help ensure that our findings are both reliable and credible.
Paul Crowe, CEO of NuView
#### [](#considering-the-use-of-radiopharmaceuticals-copper-64-and-copper-67-in-nv-vpac1-what-measures-are-being-taken-to-ensure-the-safety-and-minimize-the-side-effects-for-patients-undergoing-these)**Considering the use of radiopharmaceuticals Copper-64 and Copper-67 in NV-VPAC1™, what measures are being taken to ensure the safety and minimize the side effects for patients undergoing these diagnostics and treatments?**
Ensuring the safety and well-being of patients undergoing diagnostics and treatments with NV-VPAC1 is paramount. The dosages of the NV-VPAC1 radiopharmaceuticals will be calculated and administered to minimize radiation exposure while ensuring diagnostic and therapeutic efficacy. Patients will be continuously monitored for any adverse effects during and after the Copper-64 (Cu-64) and Copper-67 (Cu-67) administration. Adherence to stringent radiation safety protocols and guidelines will protect both patients and healthcare providers. Additionally, we will conduct regular follow-up appointments to monitor long-term health and address any delayed side effects. These safety measures are designed to minimize risks and enhance patient care.
#### [](#in-what-ways-does-nv-vpac1-offer-a-comparative-advantage-over-other-emerging-diagnostic-and-theranostic-technologies-in-oncology-can-you-provide-specific-examples-or-data-points-from-your-re)**In what ways does NV-VPAC1™ offer a comparative advantage over other emerging diagnostic and theranostic technologies in oncology? Can you provide specific examples or data points from your research to support this?**
NV-VPAC1 offers several significant advantages over other emerging diagnostic and theranostic technologies in oncology. By targeting the overexpression of VPAC1 receptors, NV-VPAC1 provides highly specific and sensitive detection of cancer cells. This technology is versatile, being used for both in vitro diagnostics and in vivo theranostics, offering a comprehensive solution for cancer detection and treatment. The minimally invasive liquid biopsy approach for detecting the overexpression of VPAC1 in shed urine is less invasive than traditional biopsy methods, reducing patient discomfort and risk. In our research, NV-VPAC1 demonstrated high sensitivity by detecting VPAC1 receptors in 100% of tested [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) subtypes without interference from ER, PR, and HER2 variants. Additionally, it successfully imaged cancerous lesions in patients with positive F18-FDG PET/CT scans, corroborated by Cu-64-NV-VPAC1 PET/CT scans. These data points highlight the effectiveness and reliability of our technology.
#### [](#what-is-your-regulatory-strategy-for-gaining-approval-from-agencies-such-as-the-fda-for-nv-vpac1-what-are-the-main-challenges-you-anticipate-and-how-do-you-plan-to-address-them)**What is your regulatory strategy for gaining approval from agencies such as the FDA for NV-VPAC1™? What are the main challenges you anticipate, and how do you plan to address them?**
Our regulatory strategy for gaining FDA approval for NV-VPAC1 involves conducting comprehensive clinical trials to generate robust data on the safety and efficacy of our technology. Thorough documentation and submission of trial data, including preclinical and clinical results, manufacturing processes, and safety protocols, will be essential. Potential regulatory challenges, such as safety concerns and manufacturing scalability, will be identified and addressed through additional studies and safety measures. By proactively managing these challenges, we aim to streamline the approval process and bring NV-VPAC1 to market efficiently.
#### [](#once-nv-vpac1-receives-regulatory-approval-what-steps-will-nuview-take-to-commercialize-the-product-and-ensure-it-is-accessible-to-the-wider-patient-population-how-do-you-plan-to-work-with)**Once NV-VPAC1™ receives regulatory approval, what steps will NuView take to commercialize the product and ensure it is accessible to the wider patient population? How do you plan to work with healthcare providers and insurers to integrate this technology into standard cancer care protocols?**
Upon receiving regulatory approval, NuView will take several steps to commercialize NV-VPAC1 and ensure its accessibility to a wider patient population. We will scale manufacturing to maximize market opportunities, leveraging the increased availability and reduced costs of Cu-64 and Cu-67 produced by radiopharmaceutical companies and contract development and manufacturing organizations (CDMOs). This approach will enable us to benefit from the economies of scale created by the increased production of these isotopes while ensuring we maintain quality and consistency in isotope manufacturing. Establishing a robust distribution network will ensure the widespread availability of NV-VPAC1. We will partner with healthcare providers to integrate NV-VPAC1 into standard cancer care protocols, providing training and support for its use.
Additionally, NuView will work with insurers to ensure coverage and reimbursement for NV-VPAC1 diagnostics and treatments, highlighting cost savings and improved patient outcomes. Implementing patient access programs will further ensure that NV-VPAC1 is available to all who need it, regardless of their financial situation. Through these efforts, we aim to make NV-VPAC1 a key component of modern cancer care.
**Categories:** Article: Executive Interviews
---
### [Virocell Biologics Secures Master Agreement with NCI Cancer Center for Transformative Therapy](https://www.clinicaltrialvanguard.com/news/virocell-biologics-secures-master-agreement-with-nci-cancer-center-for-transformative-therapy/)
**Published:** July 24, 2024
**Author:** Jon Napitupulu
**Content:**
A five-year Master Services Agreement (MSA) has been signed between [ViroCell Biologics](https://www.clinicaltrialvanguard.com/news/virocell-biologics-funding-success-despite-market-challenges/) and a prominent U.S. NCI-designated cancer center. This agreement provides the cancer center access to ViroCell’s comprehensive viral vector manufacturing services for preclinical and clinical applications.
ViroCell’s services include viral vector design, small-scale manufacturing, process development, and global plasmid sourcing. Clinical services encompass GMP manufacturing, quality control testing, regulatory support, and final batch approval.
This MSA builds upon ViroCell’s successful lentivirus vector production for the cancer center’s clinical evaluation of an engineered T cell receptor (TCR) therapy. The vector was delivered in less than five months, demonstrating ViroCell’s ability to provide high-quality vectors rapidly.
ViroCell’s expertise in viral vector manufacturing aims to support cutting-edge research in academia and industry. The company aims to accelerate the development of cell and gene therapies by providing specialized manufacturing services.
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**Categories:** News
---
### [Azitra Strengthens Patent Portfolio with Latest Grants for Atopic Dermatitis](https://www.clinicaltrialvanguard.com/news/azitra-strengthens-patent-portfolio-with-latest-grants-for-atopic-dermatitis/)
**Published:** July 24, 2024
**Author:** Jon Napitupulu
**Content:**
Azitra, Inc., a biopharmaceutical company specializing in dermatology, has secured a U.S. patent (No. 12,036,248) for its potential treatment of [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/), a common skin condition affecting approximately 16.5 million individuals in the United States. The patent covers therapeutic applications of recombinant skin microorganisms.
In addition, Azitra has received allowances for three patents in the U.S., Canada, and China. Travis Whitfill, Azitra’s COO and inventor of the patents, stated that the new protections expand their market reach in key regions and cover the treatment of filaggrin-deficient diseases, including mild to moderate atopic dermatitis.
Azitra is developing live biotherapeutic products that target specific skin diseases using precision dermatology. In patients with atopic dermatitis, filaggrin levels may be deficient or abnormal. Azitra’s topical treatment aims to directly address this pathophysiology by delivering filaggrin to the skin.
The product candidate consists of a functional unit of the human filaggrin protein attached to a cell-penetrating peptide. This allows for stable delivery of filaggrin to the skin, promoting deeper penetration. It is similar to Azitra’s ATR-12 product candidate, which delivers a Lympho-epithelial Kazal-type-related inhibitor (LEKTI) for Netherton syndrome, a condition caused by missing LEKTI.
Azitra has commenced a first-in-human study of ATR-12 for adult Netherton syndrome patients. With the recent patent issuances and allowances, Azitra now holds 4 granted U.S. patents, 14 granted international patents, 9 pending U.S. patent applications, and 44 pending international patent applications, strengthening its intellectual property protection globally.
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**Categories:** News
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### [BeiGene Expands Presence in New Jersey with $800 Million Investment](https://www.clinicaltrialvanguard.com/news/beigene-expands-presence-in-new-jersey-with-800-million-investment/)
**Published:** July 24, 2024
**Author:** Jon Napitupulu
**Content:**
[BeiGene](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/), a global oncology company, has established its flagship U.S. facility in Hopewell, New Jersey, aiming to bolster its manufacturing and clinical development capabilities. The investment, totaling $800 million, has resulted in a state-of-the-art facility contributing to BeiGene’s innovative oncology model.
The 42-acre campus houses advanced biologics manufacturing capabilities, enabling the flexible production of BeiGene’s 30 clinical or commercial-stage molecules. This investment anticipates future growth in the company’s pipeline and supports its mission of delivering quality cancer medications to patients worldwide.
The facility will create hundreds of high-tech jobs by 2025, contributing to New Jersey’s prominence as a global biopharmaceutical R&D and manufacturing hub. BeiGene’s investment is a testament to the state’s infrastructure, workforce, and proximity to major markets.
Governor Phil Murphy expressed enthusiasm for BeiGene’s contribution to New Jersey’s biopharmaceutical sector, highlighting the state’s commitment to innovation and high-tech job creation. BeiGene’s presence is anticipated to impact the economy and advance cancer treatment positively.
The Hopewell facility includes approximately 400,000 square feet of commercial-stage biologic manufacturing capacity, which can be expanded to accommodate BeiGene’s growing pipeline. It complements the company’s late-stage research and clinical development capabilities, enhancing its ability to expedite the development of novel cancer therapies.
BeiGene’s expansion in New Jersey aligns with its mission to develop affordable and accessible treatments for cancer patients globally. The company’s continued growth and innovation will contribute to advancing cancer care, offering hope to patients worldwide.
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**Categories:** News
---
### [Psychedelics Make Mind-Blowing Progress in Poland](https://www.clinicaltrialvanguard.com/news/psychedelics-make-mind-blowing-progress-in-poland/)
**Published:** July 24, 2024
**Author:** Jon Napitupulu
**Content:**
A groundbreaking alliance between Worldwide Clinical Trials (Worldwide), Centrum Badan Klinicznych PI-House, and the Polish Psychedelic Society (PTP) aims to establish Poland as a leading center for psychedelic research. This collaboration stems from Poland’s first industry-sponsored psychedelic study supported by Worldwide.
The partnership was unveiled at the “Nauka Psychodeliczna” conference, where over 500 experts gathered to discuss the potential of psychedelics for [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) treatment. PTP president Maciej Lorenc emphasized the importance of Poland’s role in this field due to its experienced psychiatrists and patient base seeking innovative treatments.
Worldwide, a global CRO, has been a staunch advocate for psychedelic research since 2017. Bartosz Janikowski, Senior Vice President of Medical Sciences, stated that Worldwide is fully committed to advancing psychedelic research and collaborating with strategic partners like PI-House and PTP.
PI-House has a long history in psychiatric research and is now focusing on psychedelic treatments. Guided by Dr. Hanna Badzio-Jagiełło, the site has completed numerous psychiatric studies and has enhanced its infrastructure and training to meet the demands of psychedelic research.
PTP aims to promote research and collaboration on psychedelics within the global scientific community. The society’s goal is to gather knowledge and share it across borders to accelerate advancements in psychiatry and beyond.
This collaboration has created a dynamic research network in Poland that seeks to advance psychiatric treatments and explore the potential of psychedelics for mental health. Poland’s emergence as a psychedelic research hub will contribute to the advancement of innovative therapies for those suffering from mental health conditions.
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**Categories:** News
---
### [Pfizer Uncovers Exciting Advance in Hemophilia A Gene Therapy](https://www.clinicaltrialvanguard.com/news/pfizer-uncovers-exciting-advance-in-hemophilia-a-gene-therapy/)
**Published:** July 25, 2024
**Author:** Jon Napitupulu
**Content:**
The AFFINE study evaluating giroctocogene [fitelparvovec](https://www.clinicaltrialvanguard.com/news/sangamo-regains-full-rights-to-hemophilia-a-gene-therapy/), a gene therapy for moderate to severe [hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/) A, has achieved significant results. The study’s primary objective of non-inferiority and superiority in total annualized bleeding rate (ABR) compared to routine Factor VIII (FVIII) prophylaxis treatment was met.
After a single infusion, giroctocogene fitelparvovec significantly reduced mean total ABR by 98.3%, with 84% of participants maintaining FVIII activity above 5% at 15 months post-infusion. The majority of participants had FVIII activity of at least 15%.
The gene therapy was generally well-tolerated, with transient elevated FVIII levels observed in some participants without affecting efficacy or safety. Treatment-related adverse events were manageable and resolved with clinical intervention.
Giroctocogene fitelparvovec aims to provide a one-time infusion that enables individuals with hemophilia A to produce their own FVIII, potentially eliminating the need for frequent intravenous infusions or injections. This investigational therapy has the potential to significantly improve the quality of life for those living with hemophilia A by reducing treatment burden and providing superior bleed protection.
Further research is ongoing to evaluate the long-term efficacy and safety of giroctocogene fitelparvovec. The findings from the AFFINE study have sparked excitement and optimism in the medical community, suggesting a potentially transformative treatment for hemophilia A.
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**Categories:** News
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### [LEO Pharma Receives Positive CHMP Opinion for Anzupgo® for Moderate to Severe Chronic Hand Eczema](https://www.clinicaltrialvanguard.com/news/leo-pharma-receives-positive-chmp-opinion-for-anzupgo-for-moderate-to-severe-chronic-hand-eczema/)
**Published:** July 29, 2024
**Author:** Jon Napitupulu
**Content:**
LEO Pharma, a leading dermatology company, announced a positive opinion from the European Medicines Agency (EMA) for Anzupgo® ([delgocitinib](https://www.clinicaltrialvanguard.com/news/anzupgo-delgocitinib-cream-approved-in-great-britain/) cream). This topical treatment is intended for adults with moderate to severe chronic hand eczema (CHE), with limited approved treatment options.
Delgocitinib cream is a novel JAK inhibitor that targets the JAK-STAT signaling pathway, which plays a crucial role in CHE development. The heterogeneous nature of CHE involves skin barrier disruption, inflammation, and microbiome disruptions, leading to significant psychological and social burdens.
Data from Phase 3 trials, DELTA 1 and DELTA 2, demonstrated the safety and efficacy of delgocitinib cream compared to a placebo. The trials achieved their primary and secondary endpoints, with subjects experiencing reduced itch and pain and improvements in overall skin health. A subsequent open-label extension trial, DELTA 3, further corroborated the cream’s long-term safety.
LEO Pharma CEO Christophe Bourdon emphasizes the challenges individuals face with CHE, including potential impacts on relationships and careers. The company’s commitment to advancing treatments aligns with the unmet needs of those with this condition.
The positive CHMP opinion is a significant step towards addressing this gap. The approval process will involve review by the European Commission, and if granted, delgocitinib cream will be authorized for use throughout the EU and other participating countries. Regulatory filings are also underway in other markets.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Merck's Keytruda and Padcev Combo Receives EU Recommendation for Unresectable Urothelial Carcinoma](https://www.clinicaltrialvanguard.com/news/mercks-keytruda-and-padcev-combo-receives-eu-recommendation-for-unresectable-urothelial-carcinoma/)
**Published:** July 29, 2024
**Author:** Jon Napitupulu
**Content:**
The European Medicines Agency (EMA) has recommended approval of Merck’s [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) and Padcev combination for the first-line treatment of unresectable or metastatic urothelial carcinoma. This recommendation follows positive results from the Phase 3 [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-A39 trial.
The combination therapy significantly improved overall survival (OS) and progression-free survival (PFS) compared to platinum-based chemotherapy. The risk of death was reduced by 53% with KEYTRUDA and Padcev. The combination also reduced the risk of disease progression or death by 55%.
This positive opinion brings KEYTRUDA closer to becoming the preferred first-line treatment for advanced or metastatic urothelial carcinoma in Europe. It would be the third [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/) indication for KEYTRUDA in the EU if approved.
Merck, Pfizer, and Astellas continue to study combination therapy in an extensive clinical program for various stages of urothelial cancer. This includes two Phase 3 clinical trials for muscle-invasive bladder cancer in KEYNOTE-B15 and KEYNOTE-B71.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Viridian Therapeutics Announces: Thrive-2 Enrollment Complete for Ted Patients](https://www.clinicaltrialvanguard.com/news/viridian-therapeutics-announces-thrive-2-enrollment-complete-for-ted-patients/)
**Published:** July 29, 2024
**Author:** Jon Napitupulu
**Content:**
Viridian Therapeutics announced the completion of enrollment in its phase 3 clinical trials, [THRIVE](https://www.clinicaltrialvanguard.com/news/dbv-technologies-launches-thrive-trial-of-viaskin-peanut-patch-in-infants/) and THRIVE-2, targeting patients with thyroid eye disease (TED).
THRIVE-2, focused on chronic TED, surpassed its target of 159 patients, enrolling 188 participants—approximately 40% of these patients enrolled from US sites. Topline data for THRIVE-2 is expected by the end of 2024.
THRIVE, targeting active TED, had completed and exceeded its enrollment target in March 2024. Approximately 50% of the enrolled patients came from the US. Topline data for THRIVE is anticipated in September 2024.
Viridian’s lead candidate, VRDN-001, is an intravenous monoclonal antibody that acts as an IGF-1R inhibitor. VRDN-001 has shown promise in improving TED symptoms in phase 2 trials.
THRIVE, and THRIVE-2 trials compare VRDN-001 to a placebo, with a dosing regimen featuring a shorter infusion time and fewer infusions than existing treatments.
Viridian plans to initiate two subcutaneous [VRDN-003](https://www.clinicaltrialvanguard.com/news/viridian-therapeutics-launches-two-phase-3-trials-ted/) phase 3 clinical trials, REVEAL-1 and REVEAL-2, in August.
The successful enrollment in the VRDN-001 trials is a testament to the patient demand for treatment options in TED. Viridian is optimistic about the potential of VRDN-001 to address the unmet needs of patients with this debilitating condition.
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**Categories:** News
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### [Seqster's Digital Health Technology: Unveiling a Revolutionary Alzheimer's Screening Breakthrough](https://www.clinicaltrialvanguard.com/news/seqsters-digital-health-technology-unveiling-a-revolutionary-alzheimers-screening-breakthrough/)
**Published:** July 30, 2024
**Author:** Jon Napitupulu
**Content:**
[SEQSTER](https://www.clinicaltrialvanguard.com/news/seqster-and-patientslikeme-partner-for-enhanced-patient-health-insights/) PDM Inc. and Boston University researchers, led by Dr. Rhoda Au, have partnered to develop an innovative early [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) screening tool. Dr. Au, a leading expert in digital health, will use SEQSTER’s 1-Click Records platform to collect longitudinal health data that will inform the development of a risk-assessment AI tool.
SEQSTER’s platform simplifies data retrieval from electronic health records, enabling researchers to quickly access a patient’s medical history. This comprehensive data will provide valuable insights into the progression of Alzheimer’s, helping identify individuals at high risk for developing the disease.
Dr. Au’s team will also link the health data to blood-based biomarkers and cognitive assessments. By combining these data sources, they aim to develop an early-stage screening tool that can detect subtle symptoms or increased risk for Alzheimer’s.
The project is made possible by the Alzheimer’s Drug Discovery Foundation’s Melvin R. Goodes Prize, which recognizes Dr. Au’s groundbreaking work in digital health. Her research holds great promise for providing early diagnosis and intervention for Alzheimer’s, which can significantly impact its progression.
SEQSTER’s 1-Click Records platform empowers patients to control their medical data while accelerating scientific progress. Participants can opt in to contribute their data, promoting wider participation in the study and advancing research efforts.
This collaboration represents a significant step towards addressing the challenges of early Alzheimer’s detection. By leveraging digital health technology and longitudinal data, researchers aim to develop tools that can improve patient outcomes and advance the understanding of this devastating disease.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Venetoclax and Fixed-Duration Calquence® Significantly Improve Leukemia Survival](https://www.clinicaltrialvanguard.com/news/venetoclax-and-fixed-duration-calquence-significantly-improve-leukemia-survival/)
**Published:** July 30, 2024
**Author:** Jon Napitupulu
**Content:**
An interim analysis of the AMPLIFY Phase III trial has revealed encouraging results in the treatment of chronic lymphocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (CLL) with AstraZeneca’s CALQUENCE® ([acalabrutinib](https://www.clinicaltrialvanguard.com/news/ascentage-pharma-presents-promising-cancer-research-at-aacr/)).
The combination of CALQUENCE with venetoclax, with or without [obinutuzumab](https://www.clinicaltrialvanguard.com/article/trend-watch/the-b-cell-depletion-arms-race-in-cns-autoimmune-trials-has-a-new-front-runner-and-it-rewrites-the-regulatory-playbook/), significantly improved progression-free survival (PFS) compared to standard chemotherapy in previously untreated patients.
Although the data for overall survival (OS) was not fully mature, a positive trend was observed favoring the CALQUENCE combination. The trial will continue to assess OS as a secondary endpoint.
The fixed duration of the CALQUENCE combination offers several benefits for patients. It allows for treatment breaks, reducing the risk of long-term adverse effects and drug resistance. This approach also improves quality of life for those living with this chronic disease.
The AMPLIFY results indicate the potential of including a BTK inhibitor like CALQUENCE in a fixed-duration regimen for CLL. This represents progress in the field and provides more options for patients and healthcare providers.
The safety and tolerability of the CALQUENCE combination were consistent with the known profiles of each individual medication. No new safety concerns were identified, and cardiac toxicity rates were low.
The AMPLIFY data will be presented at an upcoming medical meeting and submitted to regulatory authorities for further review. CALQUENCE has the potential to become the only second-generation BTK inhibitor available for both treat-to-progression and fixed-duration treatment, offering expanded treatment options for CLL patients.
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**Categories:** News
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### [BioAge Announces Groundbreaking Obesity Treatment Trial](https://www.clinicaltrialvanguard.com/news/bioage-announces-groundbreaking-obesity-treatment-trial/)
**Published:** July 30, 2024
**Author:** Jon Napitupulu
**Content:**
[BioAge](https://www.clinicaltrialvanguard.com/news/bioage-labs-advances-bge-102-nlrp3-inhibitor-with-phase-2-cardiovascular-trial/) Labs has initiated a Phase 2 clinical trial, STRIDES, to assess the efficacy of azelaprag, a small molecule that mimics the effects of exercise in combination with [tirzepatide](https://www.clinicaltrialvanguard.com/news/aardvarks-new-obesity-pipeline-data-offers-surprising-hope/) for treating obesity in individuals aged 55 and above.
Azelaprag, an apelin receptor agonist, is designed to activate signaling pathways that promote metabolic benefits similar to physical activity. Preclinical studies have shown that azelaprag enhances muscle metabolism, reduces muscle loss, and maintains cardiorespiratory fitness.
The STRIDES trial aims to evaluate the weight loss potential of azelaprag when combined with tirzepatide, a GLP-1/GIP receptor agonist that suppresses appetite. The trial will enroll approximately 220 obese individuals and measure the efficacy, safety, and tolerability of azelaprag at two oral doses in combination with tirzepatide.
The primary endpoint is the mean percentage change in body weight at 24 weeks. Exploratory endpoints include body composition, blood sugar control, and patient-reported outcomes related to health and quality of life. Top-line results are expected in the fourth quarter of 2025.
The trial is being conducted in collaboration with Lilly’s Chorus clinical development organization, which provides tirzepatide. A second Phase 2 trial is planned to assess the combination of azelaprag with [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/).
These trials aim to demonstrate the potential of azelaprag as an orally administered small molecule that can enhance the weight loss efficacy of incretin drugs, offering a potential alternative to injectable treatments.
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**Categories:** News
**Tags:** eClinical Tech News
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### [New Study: Alzheimer's Progression Explored with Revolutionary Cell Platform](https://www.clinicaltrialvanguard.com/news/new-study-alzheimers-progression-explored-with-revolutionary-cell-platform/)
**Published:** July 31, 2024
**Author:** Jon Napitupulu
**Content:**
Groundbreaking research has led to a novel model using human induced pluripotent stem cell (iPSC)-derived cortical neurons to understand [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease (AD). This model accurately reproduces key aspects of aging in a microphysiological system, offering a valuable tool for drug discovery and disease study.
AD affects millions, and current treatments provide limited relief. The team addressed a challenge in AD research by studying the degeneration of cortical layers associated with cognitive decline. Their MPS system effectively captured this aspect, revealing how cellular aging contributes to disease progression and therapy resistance.
The model allowed researchers to measure neuronal activity and aging biomarkers in real time, facilitating drug screening and potential therapeutic development. It also uncovered complex disease mechanisms, such as the role of inflammation in neuronal senescence. By treating the neurons with amyloid-β, a key player in AD development, they mimicked disease pathophysiology and analyzed senescence and therapeutic responses.
The study highlights the model’s accuracy in recapitulating AD hallmarks, showcasing its potential for translational research. It offers a novel approach to accelerate drug discovery and regulatory submissions for new AD treatments. By integrating neuronal function with proteome responses, the platform promises to advance the understanding of AD and other neurological disorders.
This groundbreaking model provides an invaluable tool for researchers to delve deeper into AD pathophysiology and explore new therapeutic avenues, offering hope for patients affected by this devastating condition.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Unlearn's AI-Powered Clinical Trials with AbbVie and Johnson & Johnson](https://www.clinicaltrialvanguard.com/news/unlearns-ai-powered-clinical-trials-with-abbvie-and-johnson-johnson/)
**Published:** July 31, 2024
**Author:** Jon Napitupulu
**Content:**
Unlearn, an AI company, showcased the impact of its technology in two poster sessions at the [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) Association International Conference. Their Digital Twin Generators create personalized forecasts of clinical outcomes for participants, enhancing the efficiency of clinical trials.
Unlearn’s Digital Twin Generators have demonstrated significant benefits in Phase III clinical trials for mild-to-moderate [Alzheimer’s Disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/) (AD):
• Reduced control arm sizes by 33% when estimating treatment effects on cognitive impairment (ADAS-Cog11) at 18 months.
• Reduced control arm sizes by 19% when estimating treatment effects on functional impairment (CDR-SB) at 12 months.
In Phase 2 studies, Unlearn’s technology can:
• Reduce overall sample sizes by 10-15% when estimating treatment effects on functional impairment (CDR-SB) at week 96.
• Increase statistical power by up to 7% (80% to 87%) when estimating treatment effects on cognitive impairment (ADAS-Cog11).
Unlearn’s Digital Twin Generators offer several advantages for clinical trials. By incorporating personalized forecasts into trial designs, researchers can:
• Increase power and reduce sample sizes, saving time and resources.
• Optimize resource allocation by reducing the need for large control groups.
• Enhance the sensitivity of clinical trials, allowing for more precise detection of treatment effects.
Unlearn’s technology revolutionizes clinical development, enabling pharmaceutical and biotech companies to streamline trials, accelerate drug discovery, and produce reliable outcomes more efficiently.
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**Categories:** News
---
### [COYA Therapeutics' Novel Patent Filing: Expanding Pipeline in Alzheimer's Disease](https://www.clinicaltrialvanguard.com/news/coya-therapeutics-novel-patent-filing-expanding-pipeline-in-alzheimers-disease/)
**Published:** August 5, 2024
**Author:** Jon Napitupulu
**Content:**
COYA 301, a recombinant human low-dose interleukin-2 (LD IL-2), and Glucagon-Like Peptide-1 receptor agonists (GLP-1 RAs) have demonstrated potential for synergistic anti-inflammatory effects. This combination targets multiple cell types, including enhancing regulatory T cell (Treg) function, suppressing pro-inflammatory myeloid and T cells, and promoting their conversion to anti-inflammatory phenotypes.
The combination of COYA 301 and GLP-1 RAs has shown promise in addressing various inflammatory diseases, including neurodegenerative conditions such as [Alzheimer’s Disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) Disease. GLP-1 RAs possess anti-inflammatory and anti-oxidant effects that contribute to their glucose-lowering benefits. Combining them with LD IL-2-mediated Treg enhancement optimizes these effects.
GLP-1 RAs and LD IL-2 have distinct mechanisms of action, exerting anti-inflammatory and Treg-enhancing effects. This multi-targeted approach addresses multiple pathophysiological pathways simultaneously. LD IL-2 enhances Treg function and numbers, suppressing inflammatory responses, while GLP-1 RAs protect neurons, reduce inflammation, and promote neuronal survival.
[Coya Therapeutics](https://www.clinicaltrialvanguard.com/news/coya-therapeutics-in-btig-kol-call-on-alzheimers-glp-1/) is investigating these combinations to develop novel therapeutic approaches for severe systemic, neuro-inflammatory, autoimmune, and metabolic conditions. The combination immunotherapy approaches can potentially significantly treat complex immune-based diseases by targeting multiple disease mechanisms.
This proprietary combination expands Coya’s pipeline and can extend the GLP-1 market beyond diabetes and obesity, fostering strategic partnerships and scientific collaborations.
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**Categories:** News
---
### [Lantern Pharma's Remarkable Phase 2 Trial Update in Advanced Lung Cancer](https://www.clinicaltrialvanguard.com/news/lantern-pharmas-remarkable-phase-2-trial-update-in-advanced-lung-cancer/)
**Published:** August 6, 2024
**Author:** Jon Napitupulu
**Content:**
Lantern Pharma’s ongoing HARMONIC™ clinical trial has yielded promising early results. The trial examines the efficacy of [LP-300](https://www.clinicaltrialvanguard.com/news/lantern-pharmas-harmonic-trial-expands-to-japan-and-taiwan/), a novel drug candidate, combined with chemotherapy in non-smokers with advanced [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC).
HARMONIC™’s initial patient cohort showed encouraging safety and efficacy outcomes. LP-300 did not produce any unexpected adverse events, with side effects mainly attributable to the chemotherapy regimen. In addition, preliminary findings indicated a high clinical benefit rate, suggesting potential therapeutic potential for LP-300.
Lantern’s AI platform, RADR®, played a crucial role in developing LP-300, facilitating target validation and patient population identification. The trial’s expansion phase is underway, enrolling patients to assess progression-free and overall survival.
For NSCLC patients who have progressed on TKI therapy, treatment options are limited, often leaving them with poor outcomes when receiving standard chemotherapy. The promising results from the HARMONIC™ trial suggest that LP-300 may offer an innovative treatment approach for this population.
Lantern’s ongoing research aims to address the critical need for novel therapies in non-smokers with NSCLC who have failed TKI treatments. The HARMONIC™ trial provides evidence of both the safety and potential efficacy of LP-300, offering hope for improved outcomes in this patient population.
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---
### [Artera Announces Prostate Test Expansion in Prostate Cancer Patients](https://www.clinicaltrialvanguard.com/news/artera-announces-prostate-test-expansion-in-prostate-cancer-patients/)
**Published:** August 7, 2024
**Author:** Jon Napitupulu
**Content:**
Artera has improved its [ArteraAI](https://www.clinicaltrialvanguard.com/news/arteraai-prostate-test-now-accessible-in-ca/) Prostate Test to support clinicians in determining the suitability of active surveillance for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) patients. This advancement enables more informed decisions regarding patient care.
The ArteraAI Prostate Test, powered by MMAI technology, evaluates a patient’s cancer progression risk and predicts treatment benefits. Its prognostic performance has been validated across diverse patient groups, including those undergoing active surveillance. The test result provides a prognostic risk assessment irrespective of treatment choice.
Artera CEO Andre Esteva emphasizes the test’s reliability and robustness, empowering healthcare providers to advance precision medicine. Through a partnership with PathNet, the test is now available to over 200 clinics.
The updated test report provides personalized guidance on active surveillance, facilitating discussions between clinicians and patients. It assesses the relative risk of cancer progression and compares it to patients undergoing active surveillance.
PathNet Co-owner Adam Cole notes that the test aligns with their commitment to pathology advancement. The partnership enables seamless integration of innovative technologies like Artera, benefiting a broader patient population.
Clinicians consider various factors when evaluating active surveillance for prostate cancer. The enhanced test report provides data on a patient’s relative risk of cancer aggressiveness, guiding decisions on active surveillance. By comparing the patient’s risk to others on active surveillance, the report assists in determining its appropriateness.
Artera continues to validate and refine its MMAI platform to provide better insights for cancer management.
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**Categories:** News
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### [Sangamo Inks Capsid Delivery Deal With Genetech for Neurodegenerative Diseases](https://www.clinicaltrialvanguard.com/news/sangamo-inks-capsid-delivery-deal-with-genetech-for-neurodegenerative-diseases/)
**Published:** August 7, 2024
**Author:** Jon Napitupulu
**Content:**
Sangamo Therapeutics and Genentech, a Roche Group member, have joined forces to develop intravenous genomic treatments for neurodegenerative diseases.
Sangamo has granted Genentech exclusive rights to its zinc finger repressors targeting the tau gene, which plays a role in [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) and tauopathies. Additionally, Genentech will have access to Sangamo’s neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which efficiently penetrates the blood-brain barrier.
Sangamo CEO Sandy Macrae emphasized their commitment to advancing genomic medicine for neurodegenerative diseases. The STAC-BBB capsid offers a unique solution to deliver therapeutics to the central nervous system.
Boris Zaïtra, Head of Roche Corporate Business Development, expressed optimism about the partnership’s potential to make a transformative impact on neurodegenerative disease treatment.
Under the agreement, Sangamo is responsible for technology transfer and preclinical activities, while Genentech assumes clinical development, regulatory interactions, manufacturing, and commercialization. Genentech has committed $50 million in upfront license fees and milestone payments. Sangamo could potentially earn up to $1.9 billion in development and commercial milestones, along with tiered royalties on net sales.
Sangamo continues to explore collaborations for its STAC-BBB capsid platform, epigenetic regulation capabilities, and other assets.
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**Categories:** News
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### [eClinical Solutions' Protocol Deviations Boost Clinical Trial Success](https://www.clinicaltrialvanguard.com/news/eclinical-solutions-protocol-deviations-boost-clinical-trial-success/)
**Published:** August 7, 2024
**Author:** Jon Napitupulu
**Content:**
eClinical Solutions has introduced enhancements and AI-powered functionalities to its Elluminate Clinical Data Cloud platform. These updates aim to streamline clinical trial oversight and reduce manual workload, ensuring data integrity and patient safety.
eClinical Solutions has added the Protocol Deviations feature, which improves trial oversight. It allows users to standardize and manage protocol deviation data within the Elluminate platform, simplifying deviation record management and ensuring compliance with the Protocol Deviation Assessment Plan (PDAP).
The platform has also incorporated AI capabilities, including:
•Audit Trail Review: Utilizes machine learning to detect anomalies in data changes and deletions, enhancing oversight.
•Data Classification: Streamlining data processing by using machine learning and natural language processing to classify incoming data based on common industry standards (CDASH and SDTM).
These enhancements provide clinical teams with the following benefits:
• Improved oversight and protocol compliance
• Reduced manual workload and increased efficiency
• Enhanced data integrity and patient safety
• Risk-based approaches for boosting operational efficiency
eClinical Solutions CEO Raj Indupuri emphasizes that successful clinical trials depend on rigorous protocols and efficient protocol deviation management. The enhancements in elluminate support these requirements, enabling [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) organizations to reduce risks and accelerate trials.
For further information on the Elluminate Clinical Data Cloud and its comprehensive data products, visit the eClinical Solutions website at www.eclinicalsol.com/platform.
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**Tags:** eClinical Tech News
---
### [Neurogene's NG-401 Gets RMAT Designation for Rett Syndrome Treatment](https://www.clinicaltrialvanguard.com/news/neurogenes-ng-401-gets-rmat-designation-for-rett-syndrome-treatment/)
**Published:** August 8, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene’s [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/), a gene therapy, has obtained Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treating Rett syndrome. This designation stems from promising clinical evidence in an ongoing trial, suggesting NGN-401’s potential to fulfill unmet medical needs in this disorder.
RMAT designation grants NGN-401 eligibility for an Accelerated Approval pathway under the 21st Century Cures Act. It also aligns with NGN-401’s prior selection for the FDA’s START Pilot Program, recognizing its therapeutic potential for Rett syndrome.
Neurogene’s CEO, Rachel McMinn, commends the FDA’s support in expediting NGN-401’s development through these initiatives. The company anticipates sharing interim efficacy data from the low-dose cohort later this year, followed by additional data from the high-dose cohort in 2025.
NGN-401 is an AAV9 gene therapy designed as a single-administration treatment for Rett syndrome. It utilizes Neurogene’s EXACT transgene regulation technology to deliver the full-length human MECP2 gene without causing overexpression-related toxic effects. This advancement addresses the need for a controlled treatment approach in Rett syndrome.
Apart from RMAT designation, NGN-401 had been previously selected for the FDA’s START Pilot Program due to its favorable clinical potential and development readiness. This program facilitates frequent interaction between the sponsor and FDA staff to support program development and ensure high-quality data for future marketing applications.
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**Categories:** News
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### [C2N Diagnostics and Unilabs Forge Unprecedented Alliance to Advance Brain Health](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/)
**Published:** August 8, 2024
**Author:** Jon Napitupulu
**Content:**
In a groundbreaking partnership, Unilabs and C2N Diagnostics are collaborating to enhance brain health diagnostics and research in Europe and globally. This multi-year agreement grants Unilabs exclusive access to C2N’s Precivity portfolio of blood tests in Europe, including Norway, Switzerland, the United Kingdom, Peru, Saudi Arabia, and the United Arab Emirates.
C2N’s Precivity blood tests detect amyloid plaques and neurofibrillary tangles in the brain, pathological hallmarks of [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. These tests support the diagnosis of Alzheimer’s and guide treatment decisions based on the underlying dementia causes.
Unilabs plans to establish a European testing network to perform Precivity tests at healthcare institutions. This ensures consistent and accurate testing comparable to the quality provided at C2N’s central laboratory in St. Louis, Missouri.
The partnership leverages C2N’s proprietary biomarker pipeline, Unilabs’ innovation, and global reach. Together, they aim to revolutionize early diagnosis, research, and long-term outcomes for patients with cognitive concerns or those at risk of Alzheimer’s.
Unilabs Head of Innovation, Dr. Rahul Chaudhary, emphasizes the need for less invasive and accessible diagnostic procedures for Alzheimer’s patients. This partnership addresses this unmet need and transforms the diagnostic experience for patients seeking personalized medicine tools.
The collaboration supports healthcare providers and researchers in identifying Alzheimer’s early, informing treatment decisions, and facilitating disease prevention strategies. Unilabs is committed to leveraging these innovations to improve patient outcomes and maximize life.
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**Categories:** News
---
### [Merck to Secure Novel B-Cell Therapy CN201 in Deal with Curon Bio](https://www.clinicaltrialvanguard.com/news/merck-to-secure-novel-b-cell-therapy-cn201-in-deal-with-curon-bio/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
Merck (MSD outside the US and Canada) has acquired [CN201](https://www.clinicaltrialvanguard.com/news/merck-acquires-novel-b-cell-depletion-therapy-from-curon/), a novel bispecific antibody, from Curon Biopharmaceutical. CN201 targets CD3 and CD19 receptors on T cells and B cells, respectively.
This acquisition expands Merck’s pipeline with a promising treatment for B-cell malignancies and autoimmune diseases. CN201 has shown early clinical success in targeting and eliminating B cells in circulating and tissue populations.
Phase 1 and 1b/2 clinical trials for CN201 are underway, evaluating its efficacy in treating relapsed or refractory non-Hodgkin’s [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (NHL) and relapsed or refractory B-cell acute lymphocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (ALL), respectively. Preliminary results suggest favorable activity and tolerability.
Merck intends to investigate further CN201’s potential for treating various B-cell malignancies and as a novel option for autoimmune diseases. Zhihong Chen, president and CEO of Curon, expressed confidence in Merck’s ability to advance CN201’s development and explore its wide-ranging therapeutic possibilities.
The acquisition is subject to customary conditions and is expected to close in the third quarter of 2024. Merck will record a pre-tax charge reflecting the upfront payment and related costs. The transaction will be reported as an asset acquisition and impact the company’s non-GAAP results upon closing.
Hogan Lovells advised Merck, while Centerview Partners LLC and Goodwin Procter LLP acted as financial and legal advisors to Curon, respectively.
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**Categories:** News
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### [Neurogene's Triumphant Progress in NGN-401 Gene Therapy](https://www.clinicaltrialvanguard.com/news/neurogenes-triumphant-progress-in-ngn-401-gene-therapy/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene Inc. has made significant progress in its clinical-stage development of [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/) gene therapy for Rett syndrome. The therapy has received RMAT designation from the FDA, recognizing its potential to address unmet medical needs. Additionally, NGN-401 has been selected for the FDA’s START Program, which aims to accelerate its development.
Interim efficacy data from Cohort 1 of the NGN-401 trial is expected to be released in the fourth quarter of 2024, with further data from both low-dose and high-dose cohorts anticipated in the latter half of 2025.
Neurogene also highlighted advancements in developing NGN-101 gene therapy for CLN5 Batten Disease, under Phase 1/2 trials. The company expects to release data from the ongoing trial in the second half of 2025.
Rachel McMinn, Neurogene’s CEO, emphasized the importance of the RMAT and START designations, which provide opportunities for enhanced collaboration with the FDA to expedite NGN-401’s development.
Neurogene has continued to advance the NGN-401 program by dosing the first patient in Cohort 2 and reported favorable preliminary safety profiles. Low-dose NGN-401 has also been well-tolerated by patients in Cohort 1.
Looking ahead, Neurogene anticipates sharing additional clinical data and updates on its pipeline of gene therapy candidates in the coming months and years.
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**Categories:** News
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### [Merck Unveils Groundbreaking ESCC Trial Results: Unlocking New Hope for Patients](https://www.clinicaltrialvanguard.com/news/merck-unveils-groundbreaking-escc-trial-results-unlocking-new-hope-for-patients/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has announced the termination of the KeyVibe-008 Phase 3 trial evaluating the combination of [vibostolimab](https://www.clinicaltrialvanguard.com/news/merck-terminates-investigational-melanoma-treatment-arm/) and [pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) in first-line treatment for extensive-stage [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (ES-SCLC). The decision followed a recommendation from an independent Data Monitoring Committee.
Analysis revealed that the primary endpoint of overall survival did not meet the pre-specified criteria for efficacy. Additionally, patients receiving the vibostolimab and pembrolizumab combination experienced higher rates of adverse events and immune-related adverse events compared to the control group.
Merck emphasized that this decision does not diminish its commitment to researching innovative ES-SCLC treatments, which have a low five-year survival rate and limited treatment advancements.
The company’s ongoing clinical development program in lung cancer includes multiple studies, focusing on earlier stages of disease and novel combinations. In SCLC, Merck and Daiichi Sankyo have initiated the IDeate-Lung02 Phase 3 trial evaluating ifinatamab deruxtecan (I-DXd) in relapsed patients. They have also expanded their collaboration to include the evaluation of MK-6070, a DLL3-targeting T-cell engager, in combination with I-DXd for certain patients with SCLC.
Meanwhile, other Phase 3 trials evaluating the vibostolimab and pembrolizumab combination in lung cancer, including KeyVibe-003, KeyVibe-006, and KeyVibe-007, are ongoing under routine safety monitoring by external data monitoring committees. To date, no study modifications have been recommended based on interim safety reviews.
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**Categories:** News
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### [Cybin Unveils Record Q1 Financials and Key Milestones](https://www.clinicaltrialvanguard.com/news/cybin-unveils-record-q1-financials-and-key-milestones/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin Inc., a clinical-stage neuropsychiatry company, reported its financial results for the first quarter ended June 30, 2024, and provided an update on its business operations.
Cybin’s lead product candidate, CYB003, a deuterated psilocybin analog, is in development for the treatment of [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD) and has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration. The company expects to report 12-month efficacy data from the ongoing Phase 2 study in Q4 2024. Cybin plans to initiate a Phase 3 multinational study for CYB003 in late summer 2024.
Cybin’s other lead candidate, [CYB004](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/), a deuterated dimethyltryptamine, is in development for Generalized Anxiety Disorder (GAD). The Phase 2 study currently enrolls patients, with topline safety and efficacy data expected by the end of the year or early Q1 2025.
To strengthen its R&D team, Cybin hired Dr. Atul R. Mahableshwarkar and Dr. Tom Macek to lead the CYB003 and CYB004 programs, respectively. Both experts bring extensive experience in drug development.
As of June 30, 2024, Cybin’s cash position totaled $183 million. The company is well-capitalized to advance its clinical programs and pursue its mission of developing novel mental health treatment options.
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**Categories:** News
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### [DeepCure's Groundbreaking AI-Generated Drug DC-9476](https://www.clinicaltrialvanguard.com/news/deepcures-groundbreaking-ai-generated-drug-dc-9476/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
DeepCure, a biotechnology company leveraging AI for drug discovery, has unveiled its first development candidate, [DC-9476](https://www.clinicaltrialvanguard.com/news/deepcure-presents-breakthrough-data-dc-9476-triumphs-over-etanercept-in-rheumatoid-arthritis-battle/). This third-generation BRD inhibitor targets the BD2 domain of Brd4, a key regulator of cytokine pathways implicated in autoimmune diseases.
DC-9476 exhibits promising efficacy in preclinical models of autoimmune diseases, including [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/), surpassing standard therapies and enhancing the effectiveness of TNF-alpha inhibitors in combination therapy. It has also demonstrated a favorable safety profile, avoiding the thrombocytopenia associated with earlier inhibitors.
DeepCure’s AI platform, combining machine learning and physics-based tools, enables the identification of novel interaction sites and the design of diverse, synthetically feasible compounds. This approach addresses the limitations of existing therapies by targeting multiple inflammatory pathways simultaneously.
Professor Francesco Del Galdo highlights the need for more comprehensive treatments for autoimmune diseases, where targeting individual pathways has had limited success. DC-9476’s multi-pathway inhibition and excellent safety profile make it a promising candidate for addressing this unmet medical need.
DeepCure’s generative AI and physics-based drug discovery engine has played a crucial role in the development of DC-9476, validating its potential to unlock novel therapeutic options for autoimmune diseases.
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**Categories:** News
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### [Tempus AI's Precision Oncology Study Unveils Validated Hla-Loh Assay](https://www.clinicaltrialvanguard.com/news/tempus-ais-precision-oncology-study-unveils-validated-hla-loh-assay/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
Tempus AI, a leading company in applying AI to precision medicine, has published a study validating its investigational human leukocyte antigen (HLA) loss of heterozygosity (LOH) assay in the journal npj Precision Oncology. This test identifies patients with solid tumors who may benefit from targeted therapies based on specific HLA-LOH events.
The study confirmed the assay’s accuracy in detecting HLA-LOH in clinical samples. Tempus collaborated with A2 [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) to demonstrate the feasibility of identifying and enrolling HLA-LOH patients in clinical trials using data from routine diagnostic tests.
The assay’s significance lies in its application in clinical trials involving [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for solid tumors. It also has implications for wider precision medicine, complementing other established biomarkers. The test is an investigational device not currently available for clinical use but has received Breakthrough Device Designation from the FDA.
Tempus is advancing precision medicine through its large library of multimodal data and AI-enabled solutions. By making data accessible and useful, Tempus empowers physicians to deliver personalized care and facilitates the discovery and delivery of optimal therapeutics. The company’s goal is to enable each patient to benefit from the accumulated knowledge and experiences of others.
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**Categories:** News
**Tags:** eClinical Tech News
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### [AN2 Therapeutics Fails Phase 2 Lung Trial](https://www.clinicaltrialvanguard.com/news/an2-therapeutics-fails-phase-2-lung-trial/)
**Published:** August 12, 2024
**Author:** Jon Napitupulu
**Content:**
After releasing unfavorable results from the Phase 2 portion of the [EBO-301](https://www.clinicaltrialvanguard.com/news/an2-therapeutics-resumes-phase-2-3-trial-for-ebo-301/) study, AN2 Therapeutics has decided to cease the trial. This study examined the efficacy of epetraborole in conjunction with an optimized background regimen (OBR) for treating treatment-refractory Mycobacterium avium complex (MAC) lung disease.
Despite achieving its primary objective of validating a patient-reported outcome (PRO) tool, the Phase 2 part failed to demonstrate a significant difference in sputum culture conversion at Month 6, a crucial secondary endpoint. As a result, AN2 will discontinue the Phase 2 (80 patients) and Phase 3 (97 patients) portions of the EBO-301 trial.
Despite the setback, AN2 remains committed to its boron chemistry platform and ongoing pipeline programs. The company plans to prioritize its internal boron chemistry platform and accelerate research efforts in infectious diseases and oncology. Additionally, AN2 will implement a strategic restructuring to extend its cash runway through 2027, allowing it to focus on advancing its pipeline through various milestones.
AN2’s boron chemistry approach involves using boron’s ability to form reversible covalent bonds with biological targets. This approach offers potential advantages for targeting biological targets that have been difficult to inhibit using carbon-based molecules.
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**Categories:** News
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### [Blue Earth Therapeutics and Seibersdorf Expand Horizons](https://www.clinicaltrialvanguard.com/news/blue-earth-therapeutics-and-seibersdorf-expand-horizons/)
**Published:** August 13, 2024
**Author:** Jon Napitupulu
**Content:**
Blue Earth Therapeutics Expands Manufacturing Collaboration with Seibersdorf Labor
Blue Earth Therapeutics, a subsidiary of Bracco, has expanded its manufacturing partnership with Seibersdorf Labor GmbH to include the production of its investigational (225Ac) rhPSMA-10.1 radioligand therapy. This expansion complements existing agreements for the manufacture of (177Lu) rhPSMA-10.1.
(225Ac) rhPSMA-10.1 is a radiohybrid therapeutic radiopharmaceutical that targets Prostate-Specific Membrane Antigen (PSMA) on [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) cells. It offers potential advantages in precision medicine for prostate cancer treatment.
Blue Earth Therapeutics’ CEO, David E. Gauden, emphasized the purity and stability of the (225Ac)-based product and the partnership’s ability to supply clinical trial sites in the UK, EU, and US.
Seibersdorf Labor’s Managing Director, Dr. Markus Neumann, highlighted the implementation of dedicated infrastructure to support the production of (225Ac) and their excitement about expanding their partnership with Blue Earth Therapeutics.
The expansion supports a recently announced research collaboration with University College London to progress a Phase 1/2 clinical trial of (225Ac) rhPSMA-10.1 in metastatic castrate-resistant prostate cancer.
Blue Earth Therapeutics’ rhPSMA compounds are based on technology originally developed at the Technical University of Munich. Blue Earth Diagnostics acquired worldwide rights to the technology in 2018 and 2020, and Blue Earth Therapeutics is currently conducting clinical trials of its rhPSMA compounds.
Blue Earth Therapeutics is committed to developing targeted radiotherapeutics for cancer patients, leveraging its expertise in radiopharmaceutical and oncology drug development.
Source link: http://www.businesswire.com/news/home/20240812462733/en/Blue-Earth-Therapeutics-Ltd-Announces-Expansion-of-Partnership-With-Seibersdorf-Labor-GmbH-to-Include-Manufacture-of-Therapeutic-Radiopharmaceutical-225Ac-rhPSMA-10.1
**Categories:** News
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### [Summit Therapeutics HARMONI-2: A Ivonescimab Achieves Clinically Significant Benefits in NSCLC](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-harmoni-2-a-ivonescimab-achieves-clinically-significant-benefits-in-nsclc/)
**Published:** August 13, 2024
**Author:** Jon Napitupulu
**Content:**
[Summit Therapeutics](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-remarkable-results-in-phase-iii-harmoni-trial-for-lung-cancer/)‘ investigational bispecific antibody, Ivonescimab, has achieved clinically significant benefits in a Phase III trial (HARMONi-2) compared to the current standard of care, Pembrolizumab, in patients with locally advanced or metastatic [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC).
HARMONi-2 evaluated Ivonescimab monotherapy against Pembrolizumab monotherapy in patients with PD-L1-positive tumors. The trial showed a statistically significant improvement in progression-free survival (PFS) with Ivonescimab across various subgroups, including patients with low or high PD-L1 expression, different histologies, and high-risk patients.
The HARMONi-2 trial results will be presented at the upcoming World Conference on Lung Cancer (WCLC 2024). Additionally, Phase II data on Ivonescimab in the perioperative setting for resectable NSCLC will also be presented.
Ivonescimab is the first to demonstrate a clinically meaningful benefit over Pembrolizumab in a randomized Phase III clinical trial in NSCLC. This breakthrough has the potential to transform the treatment landscape for patients with advanced lung cancer.
The WCLC presentations will provide detailed insights into the efficacy and safety of Ivonescimab, further solidifying its position as a promising therapeutic option for NSCLC patients.
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**Categories:** News
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### [Alzamend Neuro Partners with MGH for Phase 2 MDD Study](https://www.clinicaltrialvanguard.com/news/alzamend-neuro-partners-with-mgh-for-phase-2-mdd-study/)
**Published:** August 13, 2024
**Author:** Jon Napitupulu
**Content:**
Alzamend Neuro partners with Massachusetts General Hospital, a renowned Harvard Medical School research facility, to conduct a groundbreaking Phase II clinical study of [AL001](https://www.clinicaltrialvanguard.com/news/alzamend-neuro-and-qmenta-in-ai-imaging-trial/) for treating [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD).
Lithium, the first FDA-approved mood stabilizer, has been used off-label for MDD augmentation. Alzamend aims to demonstrate the potential of AL001 as an enhanced lithium formulation.
The study will compare the lithium levels in the brains of MDD patients receiving AL001 to those receiving a standard lithium salt. By studying lithium distribution within brain structures, Alzamend hopes to determine an optimal dose for AL001 that matches the effectiveness and safety of traditional lithium salts.
AL001, if successful, may potentially reduce the need for therapeutic drug monitoring commonly associated with FDA-approved lithium salts. The study aims to pave the way for AL001’s approval through the Section 505(b)(2) pathway designed for innovative formulations of established drugs.
Alzamend’s previous Phase IIA trial established a maximum tolerated dose for AL001, reducing the likelihood of requiring regular blood monitoring to ensure therapeutic levels. The ongoing collaboration with Massachusetts General Hospital and the expertise of Dr. Ovidiu Andronesi as the principal investigator reinforce Alzamend’s commitment to developing a novel treatment approach for MDD.
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**Categories:** News
---
### [Report Predicts $11.9B Surge in eClinical Solutions Market Driven by Clinical Trial and EHR Adoption](https://www.clinicaltrialvanguard.com/news/__trashed-50/)
**Published:** August 13, 2024
**Author:** Jon Napitupulu
**Content:**
The global market for eClinical solutions, which streamline clinical trials, is projected to expand rapidly, reaching $21.9 billion by 2030, growing at an 11.8% annual rate from 2023. Several factors drive this growth:
•Increased Clinical Trials: The growing number of clinical trials is fueling demand for eClinical solutions.
•Data Quality Focus: These solutions enhance data accuracy and efficiency in clinical trials.
•Government Initiatives: Government funding supports innovation in eClinical solutions.
•EHR Adoption: The transition to electronic health records (EHRs) boosts the need for integrated clinical trial solutions.
Government initiatives and evolving regulations support EHR adoption, driving healthcare providers to implement advanced eClinical solutions. The increasing use of healthcare IT solutions by [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) organizations and private investments in new eClinical solutions with enhanced capabilities also drive market growth.
These solutions provide valuable benefits for healthcare firms:
•Streamlined Clinical Trials: They optimize clinical trial management and operations.
•Enhanced Patient Data Management: They enable secure and efficient handling of patient data.
Key Insights
• The eClinical solutions market is estimated at $10.0 billion in 2023.
• It is expected to reach $21.9 billion by 2030, growing at a CAGR of 11.8%.
• Key factors driving market growth include increasing clinical trials, data quality focus, government initiatives, and EHR adoption.
Source link: [http://www.businesswire.com/news/home/20240812863556/en/eClinical-Solutions-Strategic-Business-Report-2024-Market-to-Grow-by-11.9-Billion-by-2030—Rise-in-Clinical-Trials-Powers-Growth-Rise-in-Pharmaceutical-Sales-to-Drive-Demand—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240812863556/en/eClinical-Solutions-Strategic-Business-Report-2024-Market-to-Grow-by-11.9-Billion-by-2030---Rise-in-Clinical-Trials-Powers-Growth-Rise-in-Pharmaceutical-Sales-to-Drive-Demand---ResearchAndMarkets.com)
**Categories:** News
**Tags:** eClinical Tech News
---
### [NorthStar and Convergent Expand Collaboration on Conv01-α](https://www.clinicaltrialvanguard.com/news/northstar-and-convergent-expand-collaboration-on-conv01-α/)
**Published:** August 14, 2024
**Author:** Jon Napitupulu
**Content:**
NorthStar Medical Radioisotopes and Convergent Therapeutics have inked a contract for manufacturing services, collaborating on CONV01-α, targeted radiotherapy for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/).
CONV01-α, spearheaded by Convergent, employs a monoclonal antibody linked to the radionuclide Ac-225, targeting the PSMA receptor prevalent in prostate cancer cells. Upon regulatory approval, NorthStar’s expertise will encompass manufacturing, quality control, and potential commercial production of CONV01-α.
This partnership underscores the growing significance of radiotherapy, particularly Ac-225-based therapies, in combating various diseases. NorthStar aims to expedite the development and delivery of innovative radiopharmaceuticals, such as CONV01-α, to meet the unmet medical needs of patients.
The manufacturing agreement will facilitate Phase 2 clinical trials of CONV01-α and potentially its commercial production, enabling Convergent to broaden its clinical development program and explore combination therapies.
CONV01-α harnesses the precision of antibodies with the potency of alpha-emitting radionuclides, delivering targeted radiotherapy to prostate cancer cells. Its unique combination sets it apart from other PSMA-targeted therapies. The alpha particles emitted by Ac-225 directly attack cancer cells, inducing DNA damage and cell death.
Convergent’s upcoming Phase 2 clinical trial aims to build upon the foundation laid by earlier clinical data. The company anticipates swiftly advancing to Phase 3 trials to accelerate the development of CONV01-α as a promising treatment for prostate cancer.
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**Categories:** News
---
### [Cybin's Breakthrough Therapy for Major Depressive Disorder: A Game-Changer in Mental Health](https://www.clinicaltrialvanguard.com/news/cybins-breakthrough-therapy-for-major-depressive-disorder-a-game-changer-in-mental-health/)
**Published:** August 14, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin, a neuropsychiatry company, recently held a successful meeting with the FDA regarding its Type B Breakthrough Therapy for their drug CYB003. The drug targets the treatment of [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD).
Cybin’s Phase 3 pivotal trial will commence in late summer 2024. The study will involve 30 clinical sites throughout the United States and Europe with expertise in depression research.
In Phase 2 trials, CYB003 demonstrated promising efficacy, with 75% of patients achieving remission from depression four months after receiving two 16mg doses. Cybin anticipates releasing 12-month efficacy data from the Phase 2 study later this year.
To mitigate the risk of functional unblinding, the Phase 3 trial will incorporate measures such as adaptive masking, participant blinding, and manual and AI monitoring.
In addition to the trial updates, Cybin has realigned the composition of its Governance and Nominating Committee and Compensation Committee, which will now consist solely of independent directors.
Cybin’s mission remains to develop innovative and effective treatments for mental health conditions. They aim to revolutionize mental healthcare through collaborations with world-class partners and scientific experts. Cybin’s research pipeline includes CYB003 for MDD, [CYB004](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) for generalized anxiety disorder, and other psychedelic-based compounds.
With its ongoing FDA collaboration and advancements in clinical development, Cybin is poised to bring new hope to individuals suffering from depression and other mental health concerns.
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**Categories:** News
---
### [Anti-TIGIT Antibody: The Revolutionary Cancer Treatment You Must Know About](https://www.clinicaltrialvanguard.com/news/anti-tigit-antibody-the-revolutionary-cancer-treatment-you-must-know-about/)
**Published:** August 14, 2024
**Author:** Jon Napitupulu
**Content:**
Anti-TIGIT antibodies are revolutionizing cancer treatment by targeting the immune inhibitory receptor TIGIT, expressed on T cells and NK cells. By binding to TIGIT, these antibodies remove immune suppression and reinvigorate the immune response against cancer cells.
Over 50 anti-TIGIT antibodies are in clinical development, with several in advanced phase 3 trials. Key players include Merck, Genentech, and BeiGene, who are studying the safety and efficacy of their antibodies in various cancer types, particularly [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC).
Anti-TIGIT antibodies offer a novel approach for patients who do not respond to existing immunotherapies or develop resistance. Combining them with other established therapies, such as PD-1/L1 inhibitors, can expand treatment options and improve outcomes.
Merck’s development of a co-formulation of [Vibostolimab](https://www.clinicaltrialvanguard.com/news/merck-unveils-groundbreaking-escc-trial-results-unlocking-new-hope-for-patients/) (anti-TIGIT) and pembrolizumab (anti-PD-1) highlights the potential for these antibodies to enhance the efficacy of immunotherapy combinations.
The market potential for anti-TIGIT antibodies is driven by the unmet need for effective cancer treatments, their promising results, and their ability to broaden the market for existing immunotherapies. As research continues, these antibodies hold great promise in transforming cancer care.
Source link: [http://www.businesswire.com/news/home/20240813348129/en/Anti-TIGIT-Antibodies-Clinical-Trials-Market-Opportunity-Outlook-2028-First-Anti-TIGIT-Antibody-to-Get-Approval-Within-Next-5-Years—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240813348129/en/Anti-TIGIT-Antibodies-Clinical-Trials-Market-Opportunity-Outlook-2028-First-Anti-TIGIT-Antibody-to-Get-Approval-Within-Next-5-Years---ResearchAndMarkets.com)
**Categories:** News
**Tags:** eClinical Tech News
---
### [Incyte Reveals Promise in Cancer Treatment with Revolutionary Drug](https://www.clinicaltrialvanguard.com/news/incyte-reveals-promise-in-cancer-treatment-with-revolutionary-drug/)
**Published:** August 16, 2024
**Author:** Jon Napitupulu
**Content:**
A pivotal Phase 3 trial (inMIND) has demonstrated the efficacy of [tafasitamab](https://www.clinicaltrialvanguard.com/news/incyte-tafasitamab-improves-pfs-in-relapsed-refractory-follicular-lymphoma/) in combination with lenalidomide and rituximab for treating relapsed or refractory follicular lymphoma (FL). The primary endpoint of progression-free survival (PFS) was met, as were crucial secondary endpoints.
Tafasitamab, a monoclonal antibody targeting CD19, displayed no new safety concerns when added to the standard care.
FL, the most common indolent B-cell non-Hodgkin lymphoma, affects a significant number of patients with limited treatment options for relapsed or refractory cases.
Based on the positive results, Incyte anticipates filing a supplemental license application for tafasitamab in FL by the end of the year.
This breakthrough offers hope for FL patients whose disease has progressed after initial treatment.
Previously approved by the FDA and EMA for diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (DLBCL), tafasitamab has demonstrated efficacy in FL.
It is marketed as Monjuvi® and Minjuvi® in the United States, Europe, and Canada.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [Pfizer and BioNTech Announce Results From New Combo Vaccine for Influenza and COVID-19](https://www.clinicaltrialvanguard.com/news/pfizer-and-biontech-announce-results-from-new-combo-vaccine-for-influenza-and-covid-19/)
**Published:** August 19, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer and [BioNTech](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) have released preliminary findings from a Phase 3 clinical trial evaluating their mRNA vaccine candidate that combines protection against influenza and COVID-19. The trial enrolled over 8,000 participants between the ages of 18 and 64.
The vaccine candidate, which combines Pfizer’s influenza vaccine with BioNTech’s COVID-19 vaccine, demonstrated robust immune responses against influenza A strain. It also elicited comparable responses against SARS-CoV-2, the virus that causes COVID-19.
However, the vaccine did not meet all primary immunogenicity objectives, particularly against the influenza B strain.
In a separate Phase 2 trial, Pfizer evaluated a standalone trivalent influenza mRNA vaccine, which showed positive immunogenicity results in adults aged 18-64.
Based on these findings, Pfizer and BioNTech are refining the combination vaccine to enhance immune responses against influenza B. They plan to consult with health authorities to determine the next steps.
The study also indicated that co-administering licensed influenza and COVID-19 vaccines resulted in strong immune responses against both diseases without any safety concerns.
Pfizer and BioNTech remain committed to developing combination vaccines to reduce the burden of respiratory infections efficiently. They express optimism about the potential of their combined COVID-19 and influenza vaccine program.
Source link:
**Categories:** News
---
### [Bristol Myers Squibb's Revolutionary CAR T Cell Therapy for Lymphoma Gains EMA Validation](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibbs-revolutionary-car-t-cell-therapy-for-lymphoma-gains-ema-validation/)
**Published:** August 20, 2024
**Author:** Jon Napitupulu
**Content:**
The European Medicines Agency (EMA) has validated an application to expand the indication of Breyanzi for treating relapsed or refractory follicular [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (FL) in adults who have received two or more prior systemic therapies.
Data from the Phase 2 TRANSCEND FL trial, the largest study of CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) in FL, supports the application. Breyanzi demonstrated a high overall response rate, with deep and durable responses. Its safety profile remains consistent with previous clinical trials.
FL, the second most common NHL, has a high relapse rate after first-line therapy. Breyanzi aims to provide treatment-free intervals with durable responses.
Previously approved in the EU for certain B-cell lymphomas, Breyanzi now seeks approval for FL patients who have relapsed within 12 months of completing first-line chemoimmunotherapy or are refractory to it. It also targets patients who have relapsed or are refractory after two or more lines of systemic therapy.
The EMA’s validation marks the initiation of the scientific review process for Breyanzi’s expanded indication in FL. Bristol Myers Squibb aims to bring this therapy to FL patients to improve outcomes and achieve long-term remission.
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**Categories:** News
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### [Walgreens, BARDA Team Up to Empower Decentralized Clinical Trials](https://www.clinicaltrialvanguard.com/news/walgreens-barda-team-up-to-empower-decentralized-clinical-trials/)
**Published:** August 20, 2024
**Author:** Jon Napitupulu
**Content:**
Walgreens and the Biomedical Advanced Research and Development Authority ([BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)) have joined forces to enhance decentralized clinical trials through the Decentralized Clinical Operations for Healthcare and Research (D-COHRe) program. This initiative aims to address challenges in accessing clinical trials and streamline research efforts over five years with a potential investment of up to $100 million.
The collaboration capitalizes on Walgreens’ extensive network of community pharmacies and robust clinical trial platform. This platform has engaged over five million potential participants, offering greater accessibility and diversity in clinical trials. By integrating clinical research into patients’ healthcare experiences, Walgreens enhances the efficiency and relevance of research conducted in real-world settings.
Walgreens’ ability to recruit diverse participants and meet enrollment goals positions them as an ideal partner for the D-COHRe program. Their comprehensive approach involves physical pharmacy locations and a digital platform, enabling patients to participate conveniently and remotely.
BARDA and Walgreens will collaborate to validate and implement new technologies and approaches for decentralized clinical research, including remote immunizations, diagnostics, and treatments. This collaboration aims to strengthen the United States’ capacity for clinical research and foster innovation in developing effective medical countermeasures for public health emergencies.
Traditional clinical trials often face delays and challenges due to enrollment difficulties. Decentralized models offer greater accessibility, reducing the 5% participation rate in clinical trials in the United States. By removing barriers and expanding access, decentralized trials accelerate the development and validation of life-saving products and treatments.
Walgreens’ commitment to making clinical research more efficient and accessible aligns with the goal of the D-COHRe program. This partnership represents a significant step towards transforming the healthcare research landscape and improving patient outcomes during routine healthcare journeys and public health emergencies.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
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### [IGC Pharma: AI Advances Therapeutic Potential for Metabolic and Neurological Diseases](https://www.clinicaltrialvanguard.com/news/igc-pharma-ai-advances-therapeutic-potential-for-metabolic-and-neurological-diseases/)
**Published:** August 21, 2024
**Author:** Jon Napitupulu
**Content:**
The discovery of IGC-1A’s potential as a GLP-1 agonist, resulting from IGC Pharma’s advanced Artificial Intelligence (AI) modeling, has revealed significant therapeutic opportunities for metabolic and neurological disorders.
GLP-1, a hormone regulating blood sugar and weight, has shown promise in treating [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) and aiding weight loss. IGC-1A’s potential role as a GLP-1 agonist expands its therapeutic potential to address neurological diseases as well potentially.
AI-driven analysis has shown that IGC-1A and IGC-1C may offer advantages over existing metabolic disorder medications. These molecules can potentially improve outcomes by addressing inflammation and oxidative stress in conditions like [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease.
IGC Pharma’s research also suggests that IGC-1A’s versatility extends to being a GIP agonist and a CB1r inverse agonist. This broad pharmacological profile opens new avenues for therapies in weight management, neurological disorders, and metabolic conditions.
The company’s ongoing investigations and toxicology studies aim to validate IGC-1A’s therapeutic potential. IGC Pharma anticipates progressing towards clinical trials in 2024.
IGC Pharma’s strategic focus on metabolic disorders highlights its commitment to driving innovation through advanced AI technology. The company aims to develop impactful solutions that address significant unmet medical needs and create value for investors.
Source link:
**Categories:** News
---
### [Novartis New Mobility Digital Biomarker in Clinical Trials](https://www.clinicaltrialvanguard.com/conference-coverage/novartis-new-mobility-digital-biomarker-in-clinical-trials/)
**Published:** August 21, 2024
**Author:** Moe Alsumidaie
**Content:**
The 7th Annual Digital Biomarkers in Clinical Trials conference featured a compelling presentation by Tilo Hache, Director of Strategy and Planning at Novartis Biomedical Research. Hache shared valuable insights and learnings from the Innovative Medicines Initiative (IMI) Mobilise-D project, a public-private partnership to address the challenges of integrating mobility as a vital sign in clinical trials. His presentation underscored the importance of mobility in health, the challenges in measuring it, and the consortium’s digital biomarker achievements and learnings.
## [](#the-importance-of-mobility-in-health)The Importance of Mobility in Health
Hache emphasized the significance of mobility as an emerging “6th vital sign,” highlighting a study by Stephanie Studensky published in 2011. The study demonstrated a correlation between walking speed and mortality, underscoring the potential impact of mobility on health outcomes. Specifically, the study found that faster walking speeds were associated with lower mortality rates, suggesting that walking speed could be a predictive marker for overall health and longevity. Despite its importance, mobility has not been widely adopted in clinical practice or pharmaceutical studies due to measurement challenges. Hache pointed out that while the study’s findings were groundbreaking, they did not translate into widespread clinical application. This gap between research and practice is partly due to the difficulty in consistently and accurately measuring walking speed in a clinical setting.
Traditional methods often require patients to visit clinics for assessments, which can be cumbersome and impractical for patients and healthcare providers. These assessments usually involve walking tests that must be conducted under controlled conditions to ensure accuracy. However, this setup introduces variability, as patients’ performance can be influenced by factors such as fatigue, stress, or the unfamiliar clinical environment. Additionally, the logistical burden of frequent clinic visits can be a significant barrier for patients, particularly those with mobility issues or chronic conditions. Hache’s presentation highlighted the need for innovative solutions to overcome these challenges and effectively integrate mobility measurements into clinical practice.
## [](#challenges-in-measuring-mobility)Challenges in Measuring Mobility
Hache outlined three primary challenges in measuring mobility, each with its complexities. First, traditional methods for measuring mobility require patients to visit clinics, where they undergo assessments that can be both time-consuming and costly. These assessments often involve walking tests that must be conducted under controlled conditions to ensure accuracy. However, this setup introduces variability, as patients’ performance can be influenced by factors such as fatigue, stress, or the unfamiliar clinical environment. Additionally, the logistical burden of frequent clinic visits can be a significant barrier for patients, particularly those with mobility issues or chronic conditions.
Second, mobility is not typically connected to standard outcome measures used in clinical trials, which challenges integration. For instance, in [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS) trials, the effectiveness of a drug is often measured using the Expanded Disability Status Scale (EDSS), which focuses on neurological function rather than mobility. Similarly, in Parkinson’s disease trials, outcomes are frequently measured by the frequency of falls or other motor symptoms rather than walking speed. Despite its potential relevance, this disconnect makes incorporating mobility as a meaningful endpoint in these studies challenging. Third, ensuring agnosticism to devices and the interests of trial sponsors adds another layer of complexity. Studies may use various devices to measure mobility, such as accelerometers, pedometers, or smartphone apps. Each device has its specifications, data formats, and potential sources of error, making it challenging to standardize measurements across studies.
## [](#the-mobilise-d-consortium)The Mobilise-D Consortium
[The Mobilise-D Consortium](https://mobilise-d.eu/ "The Mobilise-D Consortium") was initiated by Ronenn Roubenoff in 2017 and brought together a diverse group of stakeholders to tackle these challenges. The consortium included ten pharmaceutical companies, two clinical research organizations, and 22 European academic partners. This collaborative effort aimed to leverage the strengths and expertise of each partner to develop innovative solutions for measuring mobility in clinical trials. With a budget of €25 million from the European Commission, the consortium created a comprehensive framework for integrating mobility as a vital sign. The project involved more than 300 experts designing and implementing studies, developing data standardization approaches, and engaging with regulatory authorities.
Hache highlighted the importance of this collaborative approach, noting that the diverse perspectives and expertise of the consortium members were crucial for addressing the multifaceted challenges of the project. The consortium’s efforts were not just limited to Europe; they aimed to set a global standard for mobility measurements in clinical trials. By bringing together such a wide array of stakeholders, the Mobilise-D Consortium was able to pool resources, share knowledge, and develop a more robust and comprehensive approach to integrating mobility as a vital sign in clinical trials.
## [](#key-achievements)Key Achievements
Despite the unprecedented challenges posed by the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, the Mobilise-D consortium achieved several significant milestones. One of the most notable accomplishments was completing two studies involving 2,500 patients across four indications: Parkinson’s disease, multiple sclerosis, chronic obstructive pulmonary disease (COPD), and hip fracture. Conducting these studies during a global pandemic required extraordinary efforts in patient recruitment, data collection, and coordination among the consortium members. The consortium also made substantial progress in regulatory engagement. They received two letters of support from the European Medicines Agency (EMA) for their approach to measuring mobility. Additionally, they initiated their first interactions with the U.S. Food and Drug Administration (FDA).
These interactions were critical for gaining regulatory acceptance and ensuring that the project’s findings could be translated into clinical practice. In addition to regulatory achievements, the consortium focused on data standardization and dissemination. They published 70 papers and held 11 webinars to share their findings with the broader scientific community. The consortium’s commitment to transparency and open science was evident in their decision to publicly share the data from the 2,500 patients and the methods and protocols used in the studies. This open-access approach aims to facilitate further research and encourage the adoption of mobility measurements in clinical trials.
## [](#learnings-from-the-mobilise-d-project)Learnings from the Mobilise-D Project
Hache shared several key learnings from the Mobilise-D project, categorized into people, process, and product. First, diversity, trust, and leadership were crucial for the project’s success. Hache likened the role of project managers to shepherds guiding a large crowd of sheep, navigating through numerous challenges such as Brexit, the EMA’s relocation from London to Amsterdam, the Ukrainian war, and the COVID-19 pandemic. The consortium’s agile risk management approach allowed them to adapt to these disruptions and continue making progress. Hache emphasized that the team’s diversity, which included experts from various fields and backgrounds, fostered unique ideas and innovative solutions.
Second, the IMI framework, despite being perceived as bureaucratic, provided a structured way to kickstart the project. The framework included contract templates and terms and conditions that facilitated a quick start without the need for extensive negotiations. However, the project also faced challenges related to the IMI’s waterfall model, which is less flexible than iterative or agile approaches. The consortium’s decision to run its trials, rather than relying on pharma-sponsored trials offered greater flexibility but highlighted the need for infrastructure support from clinical research organizations. This approach allowed the consortium to recruit 2,500 patients within a year, even during the pandemic, demonstrating their close connection to patients and ability to adapt to challenging circumstances.
Third, collecting raw accelerometer data required extensive data clarification and validation. This process involved checking with study sites to ensure data accuracy and consistency. The consortium also navigated academic and corporate partners’ different cultures and priorities. Academic institutions often operate on semester schedules, while corporations follow fiscal cycles, leading to potential conflicts in scheduling and priorities. Hache highlighted the importance of internal marketing within corporations to maintain sponsorship and resource allocation for the project. The consortium also implemented a publication clearance process to ensure fairness and transparency in disseminating their findings.
## [](#early-health-authority-interactions)Early Health Authority Interactions
Hache stressed the importance of early interactions with health authorities like the EMA and FDA to understand regulatory pathways and requirements. While resource-intensive, these interactions are essential for successfully integrating new measures into clinical practice. Given the global nature of clinical trials and the need for harmonized standards, engaging with both European and U.S. regulators was particularly important for the consortium. Early health authority interactions provided valuable guidance on the regulatory requirements for mobility measurements, helping the consortium design their studies and data collection methods accordingly.
Hache noted that these interactions also helped build credibility and trust with regulators, which is crucial for gaining approval and support for innovative approaches. The consortium’s proactive approach in engaging with health authorities early in the project ensured that they were aligned with regulatory expectations and could address any potential issues promptly. This strategy facilitated smoother regulatory processes and increased the likelihood of successfully integrating mobility measurements into clinical practice.
## [](#conclusion)Conclusion
Tilo Hache’s presentation at the 7th Annual Digital Biomarkers in Clinical Trials conference provided a comprehensive overview of the Mobilise-D project, highlighting the importance of mobility as a vital sign and the challenges and successes of integrating it into clinical trials. The learnings from this project offer valuable insights for future public-private partnerships and the advancement of digital biomarkers in clinical research. The Mobilise-D consortium’s achievements and innovative approaches set a precedent for future efforts to incorporate mobility and other digital biomarkers into clinical trials, ultimately improving patient outcomes and advancing medical research.
**Categories:** Article: Conference Coverage
---
### [Bristol Myers Squibb Achieves FDA Acceptance for Treatment Option in Hepatocellular Carcinoma](https://www.clinicaltrialvanguard.com/news/bristol-myers-squibb-achieves-fda-acceptance-for-treatment-option-in-hepatocellular-carcinoma/)
**Published:** August 22, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb has announced the acceptance by the U.S. Food and Drug Administration (FDA) of its application for Opdivo (nivolumab) plus Yervoy (ipilimumab) as a possible first-line treatment for unresectable hepatocellular carcinoma (HCC). This acceptance is based on promising results from the Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -9DW trial.
The FDA has set a goal date of April 21, 2025, for its decision.
HCC, the most common form of [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/), is often diagnosed when surgery is not viable. With an increasing incidence in the United States, the need for new treatment options is critical.
Dana Walker, Bristol Myers Squibb’s vice president for gastrointestinal and genitourinary cancers indicated that the CheckMate -9DW trial demonstrated the superiority of Opdivo plus Yervoy in overall survival compared to current treatments and that they are eager to collaborate with the FDA to potentially offer this combination as a new first-line option for patients.”
The trial significantly improved overall patient survival with the Opdivo plus Yervoy combination versus lenvatinib or [sorafenib](https://www.clinicaltrialvanguard.com/news/cares-310-study-final-analysis-published-in-the-lancet/). This combination has previously been used as a second-line treatment for advanced HCC, but these results suggest its potential for early-stage use.
The safety profile of the combination remained consistent with previously known data, and no new safety concerns emerged.
Bristol Myers Squibb expresses gratitude to the participants and researchers involved in the CheckMate -9DW trial.
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**Categories:** News
---
### [Uncommonly Funded Cancer Research Gains Momentum with Accolades](https://www.clinicaltrialvanguard.com/news/uncommonly-funded-cancer-research-gains-momentum-with-accolades/)
**Published:** August 22, 2024
**Author:** Jon Napitupulu
**Content:**
Curebound, a philanthropic organization that funds groundbreaking cancer research, announces significant updates for its grant recipients.
The 2021 recipient of Curebound’s oncofertility Discovery Grant has secured $4.1 million in follow-on funding to expand clinical trials investigating fertility restoration in cancer patients. Additionally, one of the 2023 Cure Prize recipients has received $1.2 million to support clinical trials in [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) research.
A 2023 Targeted Grant recipient has been recognized for developing a transformative AI precision-oncology platform that leverages artificial intelligence for accurate cancer diagnoses and treatment recommendations. This achievement is a testament to Curebound’s commitment to funding high-impact, innovative research.
Anne Marbarger, Curebound’s CEO, indicated that Curebound’s mission is to invest in bold ideas that have the potential to revolutionize cancer research and that they are proud to support these promising projects and their dedicated researchers.
Curebound’s grantmaking platform fosters collaboration among research institutions in San Diego, fostering partnerships that accelerate cancer detection, treatment, and cure advancements. The organization has awarded over $35 million in research grants, supporting 115 projects.
Curebound is committed to bringing innovative ideas to life and investing in research that has the potential to make a meaningful impact in the fight against cancer. By supporting the work of these talented researchers, Curebound aims to bring cures closer to reality.
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**Categories:** News
---
### [FDA Approves Pfizer BioNTech COVID KP.2 Omicron Vaccine: A Health Milestone](https://www.clinicaltrialvanguard.com/news/fda-approves-pfizer-biontech-covid-kp-2-omicron-vaccine-a-health-milestone/)
**Published:** August 23, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has approved Pfizer and [BioNTech](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/)‘s Omicron KP.2-adapted 2024-2025 Formula COVID-19 vaccine for individuals 12 years and older. It has also granted emergency use authorization for children aged 6 months to 11 years.
This season’s vaccine will be a single dose for those 5 years and older. Immunocompromised individuals 5 years and older may be eligible for additional doses. The vaccine targets the KP.2 sublineage of the Omicron variant, as recommended by the FDA.
The approval is supported by extensive clinical, non-clinical, and real-world evidence demonstrating the safety and effectiveness of Pfizer and BioNTech’s COVID-19 vaccines. The KP.2-adapted vaccine has shown an improved response against circulating Omicron sublineages, including KP.2, KP.3, and LB.1, compared to the previous XBB.1.5-adapted vaccine.
Staying up to date with vaccinations remains crucial, especially as COVID-19 cases rise. The 2024-2025 Formula vaccine will be available in pharmacies, hospitals, and clinics across the U.S.
Pfizer and BioNTech’s COVID-19 vaccines are based on proprietary mRNA technology and have received Marketing Authorization in the U.S., EU, UK, and other countries. They also hold emergency use authorizations or equivalents in multiple countries.
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**Categories:** News
---
### [Rafael Holdings and Cyclo Therapeutics Seal Definitive Merger Pact](https://www.clinicaltrialvanguard.com/news/rafael-holdings-and-cyclo-therapeutics-seal-definitive-merger-pact/)
**Published:** August 23, 2024
**Author:** Jon Napitupulu
**Content:**
Rafael Holdings and [Cyclo Therapeutics](https://www.clinicaltrialvanguard.com/news/cyclo-therapeutics-positive-phase-3-open-label-sub-study-data-in-young-patients/) announce their merger to advance the development of Trappsol® Cyclo™ for treating Niemann-Pick Disease Type C1. This rare and fatal genetic disease has no approved treatment options.
Cyclo Therapeutics’ TransportNPC™ Phase 3 clinical trial for Trappsol® Cyclo™ has completed enrollment. Interim analysis results are anticipated in mid-2025. Rafael Holdings will provide funding for the trial’s interim analysis.
The merger combines Rafael Holdings’ resources with Cyclo Therapeutics’ expertise in developing treatments for unmet medical needs. Rafael Holdings has invested in Cyclo Therapeutics since March 2023, supporting the company’s progress in enrolling in the TransportNPC™ clinical trial.
Bill Conkling, President and CEO of Rafael Holdings, emphasizes the merger’s strategic alignment. Cyclo Therapeutics’ successful enrollment in the pivotal Phase 3 trial for Trappsol® Cyclo™ strengthens the combined entity’s focus on delivering innovative therapies for Niemann-Pick Disease Type C1.
N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, acknowledges the partnership’s benefits in reaching this milestone. The merger with Rafael Holdings provides a strong financial foundation and experienced leadership to accelerate the development of Trappsol® Cyclo™ as a potential treatment option for patients battling Niemann-Pick Disease Type C1.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Care Access Enters into New Partnership with BARDA to Sharpen Pandemic Preparedness](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)
**Published:** August 27, 2024
**Author:** Jon Napitupulu
**Content:**
Care Access, a global health research company, has partnered with the Biomedical Advanced Research and Development Authority ([BARDA](https://www.clinicaltrialvanguard.com/news/walgreens-barda-team-up-to-empower-decentralized-clinical-trials/)) to enhance vaccine and treatment accessibility for underserved and hard-to-reach communities during public health emergencies.
Care Access’s decentralized clinical trial network will be employed to establish a nationally representative network of community-based clinicians, foster partnerships with organizations in underserved areas, and collaborate with large-scale, long-term care providers. This network aims to overcome these communities’ barriers to obtaining life-saving medical interventions.
The partnership supports Care Access’s decentralized clinical research infrastructure, ensuring the rapid implementation of novel medical advancements during emergencies. The company’s experience conducting decentralized trials, including successfully enrolling over 1,000 participants from long-term care facilities during the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, underscores the need for accessible clinical studies in remote and diverse locations.
Care Access CEO Ahmad Namvargolian indicated they aim to enhance decentralized clinical trial capabilities and serve harder-to-reach communities. The collaboration invests in the well-being of those who have historically faced obstacles in accessing health research.
Care Access will use the funds to expand its partner network, reaching underserved communities and ensuring equitable access to life-saving immunizations and treatments during public health emergencies. By leveraging its global network of research sites, Care Access will support the acceleration of medical advancements and improve the health outcomes of vulnerable populations.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Merck's Bomedemstat Begins Phase 3 Trial for Essential Thrombocythemia](https://www.clinicaltrialvanguard.com/news/mercks-bomedemstat-begins-phase-3-trial-for-essential-thrombocythemia/)
**Published:** August 28, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has launched Shorespan-007, a Phase 3 clinical trial evaluating bomedemstat for treating patients with essential thrombocythemia (ET) who haven’t received cytoreductive therapy.
Essential thrombocythemia, a chronic rare blood disorder, is the most common myeloproliferative neoplasm. The standard of care hasn’t changed significantly in decades, leaving patients needing new options for disease control and improved quality of life.
Shorespan-007 is a randomized, double-blind, active comparator-controlled clinical trial comparing bomedemstat to the standard chemotherapy hydroxyurea in approximately 300 patients with ET. Its primary endpoint is a durable clinicohematologic response rate. Key secondary endpoints include fatigue symptom score, PROMIS Fatigue SF-7a total fatigue score, and MFSAF v4.0 total symptom score.
Bomedemstat is also being investigated in Shorespan-006, a Phase 3 trial comparing it to the best available therapy in patients with ET who have an inadequate response to or are intolerant of hydroxyurea.
Bomedemstat has received FDA Orphan Drug and Fast Track Designations for the treatment of ET and myelofibrosis, an Orphan Drug Designation for the treatment of acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/), and a Priority Medicines scheme designation by the European Medicines Agency for the treatment of myelofibrosis.
Updated Phase 2b Shorespan-003 trial data, including first-time genomic data, were presented at the American Society of Hematology Annual Meeting in December 2023.
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**Categories:** News
---
### [ESC Congress 2024: Bayer Presents Phase III Kerendia® (Finerenone) Data](https://www.clinicaltrialvanguard.com/news/esc-congress-2024-bayer-presents-phase-iii-kerendia-finerenone-data/)
**Published:** August 30, 2024
**Author:** Jon Napitupulu
**Content:**
During the 2024 European Society of Cardiology (ESC) Congress, Bayer will unveil late-breaking results from the Phase III FINEARTS-HF trial evaluating finereneone’s efficacy in patients with HF and left ventricular ejection fraction (LVEF) of 40% or higher.
Finerenone, marketed as KERENDIA®, is currently approved for reducing cardiovascular risks in patients with chronic kidney disease (CKD) associated with [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) (T2D).
FINEARTS-HF Trial: Key Findings
The FINEARTS-HF trial assessed the efficacy and safety of finereneone in reducing cardiovascular and HF events in patients with symptomatic HF and LVEF of 40% or higher. The trial enrolled approximately 6,000 patients randomly assigned to finereneone or placebo for up to 42 months.
Earlier this month, Bayer announced that the trial met its primary endpoint, demonstrating a statistically significant reduction in the composite of cardiovascular death and total HF events (hospitalizations or urgent HF visits).
FINE-HEART Study: Integrated Analysis
The FINE-HEART study pooled data from three Phase III trials (FINEARTS-HF, FIDELIO-DKD, and FIGARO-DKD) to explore finereneone’s effects on cardio-kidney outcomes in patients with HF with LVEF of 40% or higher and/or CKD and T2D.
KERENDIA® in HF Patients
The positive results from the FINEARTS-HF trial indicate that finereneone may benefit a broader patient population, including those with HF and LVEF of 40% or higher, regardless of CKD/T2D status.
Additional Information
Bayer will also present data from the [OCEANIC](https://www.clinicaltrialvanguard.com/conference-coverage/innovative-dct-leap-bayers-new-oceanic-study/)-AF trial investigating asundexian, an investigational anticoagulant, in patients with atrial fibrillation. No health authority has yet approved Asundexian.
For more information on KERENDIA®, consult the full Prescribing Information.
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**Categories:** News
---
### [Merck Discontinues Two Phase 3 Trials](https://www.clinicaltrialvanguard.com/news/merck-discontinues-two-phase-3-trials/)
**Published:** August 30, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has discontinued two Phase 3 trials, [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-867 and KEYNOTE-630.
The KEYNOTE-867 trial evaluated the combination of [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) (pembrolizumab) and stereotactic body radiotherapy (SBRT) for stage I or II non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) patients ineligible for surgery. However, an interim analysis showed no improvement in event-free or overall survival compared to placebo and SBRT, prompting the discontinuation of the trial. The combination also exhibited a higher rate of adverse events, including fatalities.
Similarly, the KEYNOTE-630 trial, which evaluated KEYTRUDA for adjuvant treatment of high-risk locally advanced cutaneous squamous cell carcinoma (cSCC) after surgery and radiation, was terminated due to futility. A pre-planned analysis did not show statistical significance in recurrence-free survival, the primary endpoint. The safety profile of KEYTRUDA in this trial was consistent with its established profile.
Merck has notified study investigators and advised patients to consult with their healthcare teams for further treatment options. Data analyses are ongoing, and results will be shared with the scientific community and regulatory agencies.
Despite these setbacks, Merck remains committed to researching innovative treatments for cancers with unmet needs, such as NSCLC and cSCC, to improve patient outcomes.
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**Categories:** News
---
### [Median's Revolutionary Lung Cancer Diagnostic Earns Pivotal Validation](https://www.clinicaltrialvanguard.com/news/medians-revolutionary-lung-cancer-diagnostic-earns-pivotal-validation/)
**Published:** August 30, 2024
**Author:** Jon Napitupulu
**Content:**
Median Technologies’ eyonis™ LCS, an AI-powered SaMD for [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) screening, has demonstrated strong performance in its REALITY pivotal study. The technology excels in detecting and characterizing cancerous nodules, increasing the accuracy of low-dose computed tomography (LDCT) scans.
By improving the precision of LDCT, eyonis™ LCS has the potential to significantly enhance early detection and boost survival rates for lung cancer patients. The current five-year survival rate for all lung cancer cases is a mere 18.6%, largely due to late-stage diagnoses. However, early detection in Stage 1 can lead to an 80% survival rate after 20 years.
Furthermore, eyonis™ LCS saves lives and prevents unnecessary medical procedures for healthy patients. This reduces anxiety for patients and minimizes healthcare expenses. The technology holds immense potential to revolutionize lung cancer screening and detection, especially given the increasing focus on early detection initiatives worldwide.
The U.S. Preventive Services Task Force (USPSTF) recommends lung cancer screening for adults aged 50 to 80 with a significant smoking history. In the US alone, this population numbers 14.5 million, creating a substantial market opportunity for eyonis™ LCS. The addressable market is valued at over $9 billion annually, with a potential reimbursement of $650 per exam.
The exceptional performance of eyonis™ LCS in REALITY bodes well for the upcoming [RELIVE](https://www.clinicaltrialvanguard.com/news/eyonis-lung-cancer-screening-meets-primary-endpoint-in-relive-trial/) pivotal study. Median Technologies anticipates filing for marketing authorizations in H1 2025, paving the way for broader implementation of eyonis™ LCS in both the U.S. and Europe. The technology aims to reduce mortality and transform patient outcomes by improving lung cancer detection accuracy.
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**Categories:** News
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### [Pyros Pharma's Vigafyde: The Breakthrough Oral Solution for Infantile Spasm](https://www.clinicaltrialvanguard.com/news/pyros-pharmas-vigafyde-the-breakthrough-oral-solution-for-infantile-spasm/)
**Published:** September 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Pyros Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/pyros-pharmaceuticals-fda-approved-vigafyde-the-only-ready-to-use-vigabatrin-oral-solution/) introduces VIGAFYDE™, an oral solution containing vigabatrin for treating infantile spasms (IS) in children aged 1 month to 2 years. This premeasured solution simplifies dosing and improves accuracy, addressing the challenges associated with administering medication to infants with this severe seizure disorder.
As the first ready-to-use vigabatrin oral solution, VIGAFYDE™ offers several advantages. It enables caregivers to administer the exact dosage prescribed, reducing the risk of under- or overdosing. This precise dosing is crucial for effectively controlling seizures and preventing potential vision loss, a known risk with vigabatrin therapy.
Recognizing the importance of patient support, Pyros Pharmaceuticals has partnered with AnovoRx as the exclusive specialty pharmacy for VIGAFYDE™. This partnership ensures streamlined medication access, facilitating timely treatment initiation. Additionally, families can access comprehensive support through Pyros Total Care™, a program that guides nurse educators, assistance with reimbursement, and consultations with clinical pharmacists.
VIGAFYDE™ is a significant advancement in IS treatment because it meets the specific needs of affected infants and their caregivers. The ready-to-use formulation eliminates the need for reconstitution, reducing potential errors and ensuring accurate dosing. This user-friendly format enhances compliance, increasing the likelihood of successful treatment outcomes.
Infantile spasm is a rare and serious form of [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/) that requires early and accurate diagnosis and timely intervention. VIGAFYDE™ empowers healthcare professionals with a safe and effective treatment option, providing hope for improved outcomes in young patients battling this challenging condition.
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**Categories:** News
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### [Merck Keytruda and Padcev Advance Urothelial Carcinoma Treatment: An Uncommon Breakthrough](https://www.clinicaltrialvanguard.com/news/merck-keytruda-and-padcev-advance-urothelial-carcinoma-treatment-an-uncommon-breakthrough/)
**Published:** September 4, 2024
**Author:** Jon Napitupulu
**Content:**
The European Commission (EC) has granted approval to the combination of [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) (pembrolizumab) and Padcev ([enfortumab](https://www.clinicaltrialvanguard.com/news/padcev-plus-keytruda-shows-long-term-efficacy-in-urothelial-cancer/) vedotin) as a first-line treatment for unresectable or metastatic urothelial carcinoma in adults. This approval aligns with recent guidelines recommending the combination as the preferred initial treatment approach.
The approval is based on findings from the [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-A39 trial, which demonstrated significant improvements in overall survival (OS) and progression-free survival (PFS) with KEYTRUDA plus enfortumab vedotin compared to platinum-based chemotherapy. The risk of death was reduced by 53%, while the risk of disease progression or death was reduced by 55%.
Dr. Marjorie Green, of Merck Research Laboratories, highlighted the significance of this approval, as it offers patients a promising new first-line treatment option with potential life-extending benefits.
The approval encompasses all 27 EU member states, plus Iceland, Liechtenstein, Norway, and Northern Ireland. KEYTRUDA is now approved for three indications in bladder cancer in the EU, with a total of 28 approved indications overall.
Previously, KEYTRUDA was approved in the EU as a single-agent treatment for urothelial carcinoma patients who had received prior platinum-containing chemotherapy or were ineligible for such therapy. The approval was based on data from the KEYNOTE-045 and KEYNOTE-052 trials, respectively.
The combination of KEYTRUDA and enfortumab vedotin has also been approved in the U.S. for the treatment of locally advanced or metastatic urothelial cancer. Further research is ongoing with this combination as part of a comprehensive clinical development program.
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**Categories:** News
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### [MedinCell Unveils Remarkable Progress in Product and R&D Pipeline with Abbvie](https://www.clinicaltrialvanguard.com/news/medincell-unveils-remarkable-progress-in-product-and-rd-pipeline-with-abbvie/)
**Published:** September 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Medincell](https://www.clinicaltrialvanguard.com/news/medincell-teva-olanzapine-lai-phase-3-positive-uzedy-real-world-data/) has partnered with [AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/) to develop and commercialize up to six innovative therapies using Medincell’s long-acting injectable platform. Medincell will conduct formulation and preclinical studies, including Chemistry, Manufacturing, and Controls (CMC) work, to advance a first candidate into clinical development. AbbVie will finance and lead clinical trials, regulatory approvals, manufacturing, and commercialization.
Medincell will receive an upfront payment of $35 million and is eligible for development and commercial milestones up to $1.9 billion and royalties on net sales.
Collaboration with Teva
Medincell’s UZEDY® is the first commercial product based on its BEPO® long-acting injection technology. Teva is responsible for developing and commercializing of UZEDY® and another long-acting olanzapine injection, mdc-TJK. Medincell may receive up to $222 million in milestones and royalties on sales of both products.
About BEPO® Technology
BEPO® precisely controls the delivery of drugs over extended periods from a small subcutaneous injection. It combines active ingredients with a bioresorbable deposit that releases the drug at a consistent therapeutic level, improving compliance, effectiveness, and accessibility.
About Medincell
Medincell is a biopharmaceutical company focused on developing long-acting injectable drugs. Its mission is to ensure patient compliance, enhance drug effectiveness, and reduce environmental impact through its proprietary BEPO® technology. Medincell’s first FDA-approved treatment based on BEPO®, UZEDY®, is marketed by Teva for the treatment of [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/).
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**Categories:** News
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### [AngioDynamics Auryon System Now Available in Europe](https://www.clinicaltrialvanguard.com/news/angiodynamics-auryon-system-now-available-in-europe/)
**Published:** September 4, 2024
**Author:** Jon Napitupulu
**Content:**
[AngioDynamics](https://www.clinicaltrialvanguard.com/news/angiodynamics-initiates-landmark-trial-for-pulmonary-embolism-treatment/) has secured CE Mark approval for its innovative Auryon Atherectomy System, a groundbreaking technology for treating Peripheral Artery Disease (PAD). This approval marks a significant milestone for the company and offers new treatment options for PAD patients in Europe.
The Auryon System utilizes groundbreaking solid-state laser technology to remove plaque and blockages in arteries, revolutionizing the treatment of PAD. It effectively addresses lesions of varying types, lengths, and locations, minimizing damage to vessel walls and offering a safe and targeted approach.
Laura Piccinini, AngioDynamics Senior Vice President and General Manager, indicated that the CE Mark approval of the Auryon System represents a significant achievement in their mission to provide effective solutions for healthcare professionals treating PAD.
The Auryon System has a proven track record, having treated over 50,000 patients in the United States after receiving FDA clearance in 2020. The CE Mark approval expands access to this advanced laser platform for PAD patients in the European Union, addressing a global market worth $1.1 billion.
Clinical studies have demonstrated the efficacy of the Auryon Atherectomy System in treating a wide range of lesions, from soft plaque to calcified blockages. Its 355nm wavelength laser platform generates short UV laser pulses, targeting biological reactions to effectively remove plaque while minimizing the risk of perforation and preserving the ability to vaporize lesions.
The Auryon Atherectomy System includes aspiration and off-set capabilities, allowing clinicians to address embolization risks and treat all lesion types, including In-Stent Restenosis (ISR) and Critical Limb Ischemia (CLI).
Nicolas Shammas, MD, published a prospective study examining the use of the Auryon laser system in patients with CLI. The study found that the system effectively reduced residual stenosis in most patients, eliminating the need for target lesion revascularization. The PATHFINDER registry further supports these findings, demonstrating significant improvement in blood flow and the absence of flow-limiting dissections.
AngioDynamics’ commitment to innovation in treating PAD is evident in the Auryon Atherectomy System. This approval expands the company’s reach and provides PAD patients access to cutting-edge technology that offers safe and effective treatment options.
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**Categories:** News
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### [Foundation Medicine and Syndax Pharma Unveil Companion Diagnostic in Hematology](https://www.clinicaltrialvanguard.com/news/foundation-medicine-and-syndax-pharma-unveil-companion-diagnostic-in-hematology/)
**Published:** September 5, 2024
**Author:** Jon Napitupulu
**Content:**
Foundation Medicine and Syndax Pharmaceuticals have initiated a partnership to develop a tool to identify acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML) patients with NPM1 mutations.
NPM1 mutations are common in AML, affecting around 30% of newly diagnosed patients. However, there are currently no approved targeted treatments for these patients, with an overall survival rate of approximately 50% after five years.
Menin inhibitors, such as Syndax’s [revumenib](https://www.clinicaltrialvanguard.com/news/revumenib-data-from-beat-aml-trial-published-in-jco-presented-at-eha-2025/), are promising new treatment options for NPM1-mutated AML. To enhance the identification of suitable patients, Foundation Medicine aims to create a next-generation sequencing companion diagnostic based on its [FoundationOne](https://www.clinicaltrialvanguard.com/news/foundation-medicine-launches-groundbreaking-rna-sequencing-test-foundationonerna-in-the-u-s/)® Heme platform.
This assay, if approved, could lead to more personalized and effective treatment options for AML patients. It could also mark the platform’s first use as a companion diagnostic, solidifying Foundation Medicine’s position as the industry leader in companion diagnostic approvals.
The collaboration underscores the importance of advancing comprehensive genomic profiling and developing new therapeutic targets in the fight against blood cancers. By providing physicians with access to high-quality tests and potential therapies, the partnership aims to improve patient outcomes and optimize treatment decisions.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Arsenal Biosciences Unlocks $325M to Drive Programmable Cell Therapies to Clinical Triumph](https://www.clinicaltrialvanguard.com/news/arsenal-biosciences-unlocks-325m-to-drive-programmable-cell-therapies-to-clinical-triumph/)
**Published:** September 5, 2024
**Author:** Jon Napitupulu
**Content:**
Arsenal Biosciences, a leading [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) company specializing in solid tumor treatment, has secured $325 million in Series C funding. The oversubscribed round attracted new investors like ARCH Venture Partners and Milky Way Investments Group. Existing investors, including the Parker Institute for Cancer Immunotherapy and SoftBank Vision Fund 2, also participated.
ArsenalBio’s proprietary T-cell engineering technology showcases promise in early clinical trials and preclinical studies. The company plans to use the funds to advance its lead programs and expand its pipeline of therapeutic options for solid tumor cancers. The investment will also drive innovation in identifying new cell therapies.
“This investment empowers us to continue our development roadmap, scale our manufacturing capabilities, and pursue groundbreaking advancements in T-cell medicine,” said ArsenalBio CEO Ken Drazan. The company’s robust pipeline includes therapies for ovarian, kidney, and prostate cancers developed in collaboration with Bristol-Myers Squibb Company. ArsenalBio recently initiated a Phase 1/2 clinical trial for its second T cell product candidate, AB-2100, for clear-cell renal cell carcinoma, receiving Fast Track designation from the U.S. Food and Drug Administration.
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**Categories:** News
---
### [Kincell Bio Expands Its Dominance in the Biotech Industry Through Groundbreaking Partnership](https://www.clinicaltrialvanguard.com/news/kincell-bio-expands-its-dominance-in-the-biotech-industry-through-groundbreaking-partnership/)
**Published:** September 6, 2024
**Author:** Jon Napitupulu
**Content:**
Kincell Bio, a [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) CDMO, has forged a new partnership to produce early-phase clinical material for an allogeneic cell therapy product. The deal extends Kincell Bio’s manufacturing capabilities following the acquisition of a GMP-ready facility in 2024.
The partnership reflects Kincell Bio’s expertise in allogeneic cell therapy production, a key focus for the company. Bruce Thompson, CEO of Kincell Bio, emphasized the company’s commitment to supporting customers’ clinical trials through critical CMC development and manufacturing expertise.
The collaboration underscores Kincell Bio’s unwavering dedication to innovation in cell therapy development and manufacturing. The company’s mission is to bring new cellular therapies to market and improve patient outcomes, as evidenced by its ongoing efforts to expand its partnership portfolio and enhance its manufacturing capabilities.
Kincell Bio’s manufacturing facilities in Research Triangle Park, NC, and Gainesville, FL, enable them to provide comprehensive services, including analytical development, process development, GMP manufacturing, testing, and release. The company’s expertise extends to immune cell therapies, such as autologous and allogeneic CAR-T, CAR-NK, and CAR-M programs, supporting innovative companies in the development of these transformative treatments.
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**Categories:** News
---
### [FDA Letter Supports Amprion's Revolutionary α-Synuclein Test](https://www.clinicaltrialvanguard.com/news/fda-letter-supports-amprions-revolutionary-α-synuclein-test/)
**Published:** September 6, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food & Drug Administration (FDA) recently issued a Letter of Support (LoS) endorsing the use of the alpha-synuclein (α-syn) seed amplification assay (synSAA) in research and clinical trials. This assay has demonstrated exceptional specificity and sensitivity in detecting misfolded α-syn in cerebrospinal fluid (CSF). It is a valuable diagnostic tool for neurodegenerative disorders such as [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) and Alzheimer’s.
The synSAA test, developed by Amprion, is the only validated seed amplification assay available for diagnosing synucleinopathies, a group of neurodegenerative disorders associated with α-syn aggregation. The FDA’s LoS encourages the incorporation of synSAA into clinical trials to improve patient selection and enhance early intervention strategies.
Amprion’s assay has been extensively validated through peer-reviewed studies, confirming its high diagnostic accuracy. This accuracy is crucial in understanding the true value of biomarker tests, as clinical diagnosis of synucleinopathies can be error-prone. The positive results from numerous studies involving over 1,800 CSF samples have demonstrated the assay’s ability to detect diffuse Lewy body pathology with over 95% sensitivity and specificity.
Dr. Russell Lebovitz, Amprion’s CEO and co-founder, indicated that they are excited about the timing of this FDA support letter, and that it comes as the number of disease-modifying drug candidates in clinical development is increasing. Amprion has partnered with industry leaders on these trial advancements, recognizing the importance of the synSAA test in advancing early detection and treatment for neurodegenerative disorders.
Amprion holds worldwide SAA technology patents covering its process and critical components. The company offers various SAA-based services to pharmaceutical partners worldwide, empowering research and drug development efforts for neurodegenerative conditions.
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**Categories:** News
---
### [Summit Therapeutics's Remarkable Cancer Breakthrough: New Treatment Reduces Disease Progression by 49%](https://www.clinicaltrialvanguard.com/news/summit-therapeuticss-remarkable-cancer-breakthrough-new-treatment-reduces-disease-progression-by-49/)
**Published:** September 9, 2024
**Author:** Jon Napitupulu
**Content:**
[Summit Therapeutics](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-remarkable-results-in-phase-iii-harmoni-trial-for-lung-cancer/) and Akeso announced positive results from the Phase III HARMONi-2 trial, evaluating ivonescimab, an investigational bispecific antibody, as a first-line treatment for advanced [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) with PD-L1 positive expression.
Ivonescimab demonstrated a statistically significant improvement in progression-free survival (PFS) compared to pembrolizumab, a current standard of care. Patients receiving ivonescimab experienced a median PFS of 11.14 months versus 5.82 months for those receiving pembrolizumab (49%).
The PFS benefit was observed across various subgroups, including those with low and high PD-L1 expression, squamous and non-squamous histologies, and other high-risk patients. Additionally, ivonescimab showed higher overall response rates and disease control rates than pembrolizumab.
Safety profiles were comparable between ivonescimab and pembrolizumab, with similar rates of serious treatment-related adverse events and treatment discontinuations due to such events.
The HARMONi-2 trial results support the potential of ivonescimab as a promising new treatment option for first-line advanced NSCLC patients with PD-L1 positive tumors. The company plans to initiate the Phase III HARMONi-7 trial in early 2025 to evaluate ivonescimab in this setting further.
Encouraging data from a Phase II perioperative NSCLC study and three additional Phase II studies evaluating ivonescimab in solid tumors beyond NSCLC were also presented at scientific conferences.
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**Categories:** News
---
### [AstraZeneca TROP2 Biomarker Predicts Lung Cancer Treatment Outcomes](https://www.clinicaltrialvanguard.com/news/astrazeneca-trop2-biomarker-predicts-lung-cancer-treatment-outcomes/)
**Published:** September 9, 2024
**Author:** Jon Napitupulu
**Content:**
An exploratory analysis of the TROPION-Lung01 phase 3 trial revealed TROP2 expression, measured using AstraZeneca’s computational pathology platform, as a predictor of clinical outcomes in advanced [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) patients treated with [datopotamab](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/) deruxtecan (Dato-DXd).
In patients whose tumors showed high TROP2-QCS biomarker levels, Dato-DXd significantly improved efficacy compared to docetaxel. A greater proportion of patients with nonsquamous NSCLC had TROP2-QCS positive tumors than those with squamous NSCLC.
In the TROP2-QCS biomarker-positive group, Dato-DXd reduced the risk of disease progression or death by 43% compared to docetaxel. Notably, this improvement in efficacy was more pronounced in the subgroup of patients with nonsquamous NSCLC without actionable genomic alterations, where Dato-DXd reduced the risk by 48%.
These findings suggest that the TROP2-QCS biomarker has the potential to identify patients who are likely to benefit most from Dato-DXd therapy in advanced NSCLC. Further research is needed to validate this biomarker and determine its role in guiding treatment decisions.
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**Categories:** News
---
### [Innate Pharma's Monalizumab NeoCOAST-2 Phase 2 Trial Reports Encouraging Outcomes in Early-Stage Lung Cancer](https://www.clinicaltrialvanguard.com/news/innate-pharmas-monalizumab-neocoast-2-phase-2-trial-reports-encouraging-outcomes-in-early-stage-lung-cancer/)
**Published:** September 10, 2024
**Author:** Jon Napitupulu
**Content:**
The NeoCOAST-2 trial evaluated the efficacy and safety of treatments in patients with early-stage [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). Interim results from three treatment arms were presented at the World Conference on Lung Cancer.
One arm, combining monalizumab, [durvalumab](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-durvalumab-in-phase-iii-biliary-tract-cancer-trial/), and chemotherapy, showed promising results. This combination achieved a 26.7% pathological complete response rate and a 53.3% major pathological response rate, numerically higher than the approved regimen of durvalumab and chemotherapy.
Monalizumab, a novel checkpoint inhibitor targeting the NKG2A receptor, is crucial in the treatment. By blocking NKG2A, monalizumab enhances the anti-tumor activity of NK and CD8 T cells. This disruption of the inhibitory mechanism allows immune cells to target and eliminate cancer cells more effectively.
The trial arms demonstrated manageable safety profiles with no impact on surgical rates. Further analysis is ongoing, and final data on event-free survival (EFS) is expected.
These preliminary findings suggest that monalizumab may potentially extend the clinical benefits of durvalumab in the neoadjuvant/adjuvant setting for patients with NSCLC. NSCLC, the most common type of lung cancer, is characterized by its stage and extent of spread. Early-stage NSCLC, the focus of the NeoCOAST-2 trial, accounts for a significant proportion of lung cancer cases worldwide.
The encouraging outcomes from the NeoCOAST-2 trial provide optimism for improving treatment strategies in early-stage NSCLC. Further research and clinical development are necessary to confirm the long-term efficacy and safety of monalizumab-based regimens in this patient population.
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**Categories:** News
---
### [Silexion Therapeutics Unveils Pioneering RNAi Technology from Silexion Therapeutics for Revolutionizing the Fight Against KRAS-Driven Cancers](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/)
**Published:** September 10, 2024
**Author:** Jon Napitupulu
**Content:**
Silexion’s proprietary LODER™ platform enables targeted oncogene silencing, using siRNA to block the production of oncogenic proteins. This approach differs from small molecule KRAS inhibitors targeting already-produced oncogenic proteins. The localized delivery system bypasses tumor barriers, ensuring higher treatment concentrations within the tumor while minimizing systemic side effects.
[SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/), Silexion’s improved siRNA formulation, targets a broader range of KRAS mutations, addressing a limitation of current KRAS inhibitors. It is expected to enter Phase 2/3 clinical trials in 2025-2026.
[Pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) is a particularly challenging disease due to late detection, aggressive growth, a dense tumor microenvironment, genetic complexity, and resistance to chemotherapy.
Silexion’s LODER™ and SIL-204 technologies are designed to overcome these challenges. Silexion aims to improve treatment outcomes for pancreatic cancer patients by directly targeting KRAS mutations and delivering treatment locally.
The increasing prevalence of KRAS-driven cancers and the growing M&A activity in the precision oncology space further highlight the potential for Silexion Therapeutics. As a recently de-SPACed company, it represents an opportunity for investors interested in the rapidly growing field of targeted cancer treatments.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [AiCure H.code Patient Engagement Platform Unveiled at dpharm 2024](https://www.clinicaltrialvanguard.com/news/aicure-h-code-patient-engagement-platform-unveiled-at-dpharm-2024/)
**Published:** September 11, 2024
**Author:** Jon Napitupulu
**Content:**
AiCure, a pioneer in clinical trial monitoring and patient engagement, will debut its innovative H.Code platform at the [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) conference. This technology combines computer vision, communication tools, and analytics to enhance site teams’ understanding of participant behavior.
H.Code extends beyond adherence monitoring, enabling the development of digital assessments that connect behavior to disease and treatment outcomes, paving the way for precision medicine.
AiCure CEO Ed Ikeguchi’s presentation on “Simplifying Trial Participation” will showcase how a single platform can streamline processes for participants, sites, and sponsors.
AiCure encourages DPHARM attendees to explore H.Code’s potential in Booth #46.
Furthermore, Josh Wilson has been promoted to Chief Operating Officer (COO). With decades of experience in clinical research organizations (CROs), Wilson will lead product management and operations, implementing strategies to enhance AiCure’s mission of revolutionizing clinical trials through AI-powered solutions.
Ikeguchi expressed confidence in Wilson’s abilities: “Josh’s leadership and operational expertise have proven invaluable. His promotion reflects our trust in his guidance as AiCure continues to grow and innovate.”
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**Categories:** News
**Tags:** eClinical Tech News
---
### [New Clinical Trial Technology Innovations at Veeva R&D And Quality Summit](https://www.clinicaltrialvanguard.com/conference-coverage/new-clinical-trial-technology-innovations-at-veeva-rd-and-quality-summit/)
**Published:** September 12, 2024
**Author:** Moe Alsumidaie
**Content:**
The 2024 Veeva R&D and Quality Summit brought together industry leaders and professionals to discuss the latest clinical trial technologies, [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) advancements, and strategies shaping the industry. The event featured keynotes and sessions from industry leaders, including Peter Gassner, Founder and CEO of Veeva Systems, along with other keynote speakers like Seb Moity, Head of IT Digital Clinical Operations at UCB in the clinical data keynote, and Kevin O’Brien, Senior Vice President, Clinical Development Operations at Eikon Therapeutics in the Veeva Development Cloud keynote. The conference provided a platform for sharing insights on product innovations, applying AI, integration across systems and functions, and the transformation of clinical data management, with a strong focus on improving collaboration between research sites and sponsors.
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
## [](#keynote-by-peter-gassner-veevas-vision-and-product-innovations)**Keynote by Peter Gassner: Veeva’s Vision and Product Innovations**
Peter Gassner began by emphasizing Veeva’s unwavering commitment to the life sciences industry, highlighting the company’s mission to deliver positive ROI for customers and to act as a durable, long-term industry partner. Gassner elaborated on the significance of Veeva being a Public Benefit Corporation (PBC), explaining that this status mandates the board to balance the interests of customers, shareholders, and the industry. This unique structure ensures that Veeva’s focus extends beyond profit maximization to include broader industry benefits.
Gassner then delved into Veeva’s three main product offerings: Development Cloud, Commercial Cloud, and Data Cloud. He discussed the significant shift for Veeva’s flagship CRM product, migrating from the Salesforce.com platform to the Veeva Vault platform. This move is expected to bring innovation and enhanced capabilities to the commercial cloud side of life sciences. He also introduced the first data product for clinical, delivering centralized reference data for research sites, such as specialization, trial history, and specialty areas, which in turn help sponsors identify appropriate research sites more efficiently, leading to faster trial initiation.
Peter Gassner Founder & CEO of Veeva Systems
## [](#transforming-clinical-data-management)**Transforming Clinical Data Management**
The second session of the conference featured Richard Young, VP of Strategy for Clinical Data at Veeva, Seb Moity, Head of IT Digital Clinical Operations at UCB, and two speakers from a top 20 biopharma. Together, they discussed the transformation of clinical data management and the pivotal role of digital solutions in this process. Richard Young began by reflecting on the evolution of Veeva’s clinical data strategy, one he started developing back in 2017. He highlighted the importance of connected applications and tools to solve the entire data chain, an improvement that can make clinical trials more efficient and accessible. Young emphasized the need for faster clinical trial results to meet patient needs and discussed the role of Veeva’s Clinical Data Strategy team in achieving this goal.
Young shared insights into developing and launching Veeva’s EDC system, which aims to streamline data management processes and improve trial outcomes. He explained that the EDC system was designed to address the limitations of traditional data management systems, which often involve manual processes and fragmented data sources.
Now, Veeva Clinical Data applications are helping the industry advance clinical data management. For example, Veeva Clinical Database (Veeva CDB) integrates study data from multiple sources, providing a unified platform for managing clinical trial data. This reduces the need for manual data entry and minimizes the risk of errors, ultimately leading to more accurate and reliable trial results. In the end, Veeva Clinical Data helps companies run the trial they need, not the one the industry has been historically limited to.
Young advocated for allowing research sites the flexibility to use their preferred technology while maintaining efficient and automated data sharing with sponsors, addressing the concern that research sites spend too much time learning new technology rather than focusing on patient care.
The importance of collaboration between technology providers and biopharma companies was also emphasized by Young. He noted that successfully implementing digital solutions requires close cooperation and a shared commitment to innovation. By working together, he argued, technology providers and biopharmas can overcome clinical data management challenges and drive significant improvements in trial efficiency and outcomes. He also discussed the factors influencing whether to outsource or insource, highlighting that the decision depends on trial size, available resources, and the benefits of setting up internal infrastructure versus leveraging external CRO resources.
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
Moity from UCB shared how the company thinks about the end-to-end patient experience, ensuring they can empower, enable, and educate clinical trial participants. He shared a practical perspective on implementing digital clinical applications, like Veeva eCOA, to deliver a better experience to patients.
A top 20 biopharma also shared their insights and provided valuable context for understanding the impact of Veeva’s solutions on large-scale clinical trial operations. The representatives discussed the challenges of managing complex clinical trials, particularly regarding data integration and coordination across multiple sites and stakeholders. They emphasized the importance of seamless data integration, noting that fragmented data sources can lead to inefficiencies and errors.
Veeva’s solutions, they explained, have helped address these challenges by providing a unified platform for managing clinical trial data. According to the representative, one of the key benefits is the ability to streamline data collection and analysis. They noted that Veeva Vault EDC has significantly reduced the time required to collect and analyze trial data, allowing their company to make more informed decisions and improve data accuracy and reliability, further enhancing trial outcomes.
The representatives also highlighted the importance of Veeva’s automation capabilities. They explained that Veeva’s application bots and direct data API have automated many of the repetitive tasks involved in clinical data management, freeing their teams to focus on more strategic activities. This automation has improved efficiency and reduced the risk of errors, leading to more accurate and reliable trial results.
In conclusion, the representatives emphasized the value of Veeva’s collaborative approach. They noted that Veeva’s product managers and strategy teams have responded highly to their feedback, continuously improving the solutions to meet their evolving needs. They also stressed the importance of a connected technology ecosystem across different areas, such as clinical data with clinical operations and pharmacovigilance, to streamline processes and improve workflow efficiency.
## [](#updates-to-site-connect-and-new-ai-applications)**Updates to Site Connect and New AI Applications**
One of the most significant announcements was a major release of Veeva Site Connect, designed to streamline sponsor and site collaboration across various study-related activities. Site Connect aims to provide a standard site interface, reducing the complexity and improving the user experience for sponsors and sites. Jim Reilly, VP of Veeva Development Cloud Strategy, explained that the new version of Site Connect includes features for study information, communication, document sharing, and safety letters. It also offers self-service user administration for sites, allowing them to manage their user accounts and permissions. This level of self-service is a significant improvement over previous versions, which required sponsors to handle user administration tasks.
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
Reilly emphasized that Site Connect is connected with Veeva’s clinical operations applications, which includes eTMF and CTMS. This ensures that all study-related data is stored securely, making it easier for sponsors and sites to access and manage information. He also pointed out that Site Connect provides a consistent user experience across different studies and sponsors, reducing the learning curve for site staff. By offering a standardized and user-friendly solution, Veeva aims to improve the efficiency and effectiveness of sponsor-site collaboration, ultimately enhancing the overall quality of clinical trials.
In addition to Site Connect, Reilly discussed the role of AI in Veeva’s future. He mentioned that Veeva is exploring various applications of AI, such as using AI to assist in drafting documents and finding information. He also highlighted the potential for AI-powered bots to provide a more intuitive and efficient way for users to interact with applications. While these AI initiatives are still early, Reilly expressed optimism about their potential to enhance Veeva’s product offerings and improve the overall user experience. By focusing on practical and impactful AI applications, Veeva aims to provide its customers with tools to improve their productivity and efficiency significantly.
## [](#summary)**Summary**
The Veeva R&D and Quality Summit Systems showcased the company’s dedication to innovation and excellence in the life sciences industry. With a focus on product development, automation, and customer-centric strategies, Veeva continues to lead the way in transforming clinical trials and data management. The insights industry leaders and professionals shared highlighted the significant progress made and the exciting future for clinical trials and life sciences. The emphasis on improving collaboration between research sites and sponsors, flexibility in technology use, and a connected ecosystem of applications highlight Veeva’s commitment to driving efficiency and innovation in the industry.
*This article is sponsored by [Veeva Systems](https://www.veeva.com/ "Veeva Systems")*
[](https://www.veeva.com/products/veeva-site-connect/?utm_source=Vangaurd&utm_medium=display&utm_campaign=fy25_rd_clinops_na_q3_other_vanguard_site_connect&utm_content=product-page&utm_term=)
**Categories:** Article: Conference Coverage
---
### [Sanofi, Radiomedix, and OranoMed Sign Revolutionary Licensing Agreement for Next-Gen Radioligand Medicine](https://www.clinicaltrialvanguard.com/news/sanofi-radiomedix-and-oranomed-sign-revolutionary-licensing-agreement-for-next-gen-radioligand-medicine/)
**Published:** September 13, 2024
**Author:** Jon Napitupulu
**Content:**
Sanofi, RadioMedix, and Orano Med have partnered to advance AlphaMedixTM, a treatment for a rare cancer known as neuroendocrine tumors (NETs).
AlphaMedixTM, a radiopharmaceutical, employs a unique peptide to target [somatostatin](https://www.clinicaltrialvanguard.com/news/first-patient-dosed-in-crinetics-neuroendocrine-tumor-trial/) receptors on NETs. This peptide is combined with lead-212 (212Pb), which releases alpha particles to destroy cancerous cells.
AlphaMedixTM has shown promising results in clinical trials, exhibiting a favorable safety profile and significant tumor reduction. Its potential as a transformative treatment has led to Breakthrough Therapy Designation from the FDA.
Dietmar Berger, Chief Medical Officer at Sanofi, emphasizes the significance of radioligand therapies, including AlphaMedixTM, for treating rare cancers. The collaboration with RadioMedix and Orano Med aligns with Sanofi’s commitment to innovation in cancer care.
RadioMedix, a pioneer in radioligand therapy, developed AlphaMedixTM and believes it will revolutionize nuclear oncology. The high energy and short path length of alpha particles overcome the limitations of existing beta emitter therapies.
Orano Med, specializing in 212Pb production, brings expertise in manufacturing and distribution. Their contribution underscores the importance of this partnership in bringing AlphaMedixTM to patients globally.
The collaboration between Sanofi, RadioMedix, and Orano Med aims to provide patients with access to a potentially life-saving therapy. By leveraging innovative technologies and combining expertise, they strive to make a meaningful difference in treating rare cancers.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Unveiling Mavenclad's Remarkable Benefits: Sustained Efficacy in MS](https://www.clinicaltrialvanguard.com/news/unveiling-mavenclads-remarkable-benefits-sustained-efficacy-in-ms/)
**Published:** September 13, 2024
**Author:** Jon Napitupulu
**Content:**
EMD Serono presented data at the European Committee for Treatment and Research in [Multiple Sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (ECTRIMS) conference, showcasing the sustained efficacy and safety of MAVENCLAD (cladribine) tablets in treating relapsing multiple sclerosis (RMS).
Studies have consistently demonstrated the efficacy of MAVENCLAD in reducing relapse rates and disease activity. Additional analyses now reveal its impact on neuroinflammation and disability progression.
MAGNIFY-MS sub-studies showed that MAVENCLAD significantly reduced disability accrual, including independent of relapse activity. Treatment-naïve patients experienced meager rates of progression.
Furthermore, MAVENCLAD has been shown to sustain benefits in cognition, MRI outcomes, and relapse prevention four years after treatment initiation. The CLARIFY-MS extension study supported this finding.
MAVENCLAD has maintained a consistent safety profile throughout clinical trials and real-world use. Two-year data from the MAGNIFY-MS study indicated low relapse rates and minimal disability progression.
These findings suggest that MAVENCLAD provides a sustained and durable effect in reducing disease activity and preserving physical abilities in individuals with RMS. It demonstrates the medication’s ability to impact neuroinflammation and prevent progression, highlighting its potential to improve long-term outcomes for patients living with multiple sclerosis.
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**Categories:** News
---
### [AI-Enhanced Gene Therapy Vector: A Revolutionary Advance for Muscle Disease Treatment](https://www.clinicaltrialvanguard.com/news/ai-enhanced-gene-therapy-vector-a-revolutionary-advance-for-muscle-disease-treatment/)
**Published:** September 13, 2024
**Author:** Jon Napitupulu
**Content:**
Genethon, a leading gene therapy research facility, has significantly progressed in developing a new generation of adeno-associated virus (AAV) capsids using artificial intelligence (AI). These capsids enhance gene therapies for muscle diseases by targeting muscles more effectively and avoiding the liver.
This breakthrough, published in Nature Communications, utilizes AI to design capsids that selectively bind to integrin alpha V beta 6, a molecule on muscle cell surfaces. By precisely targeting this receptor, the new capsids deliver genetic material directly to muscle tissue, reducing the need for high doses that could cause adverse effects.
Isabelle Richard, head of Genethon’s Progressive Muscular Dystrophies Team indicated that Genethon’s AI-designed gene therapy vectors represent a paradigm shift in neuromuscular disease treatment and that they exhibit increased efficacy and safety, opening new avenues for treating various neuromuscular diseases.
The efficacy of the LICA1 capsid variant was validated in models of [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy and limb-girdle muscular dystrophy. At lower doses, it effectively targeted muscle tissue without penetrating the liver, overcoming a major challenge in gene therapy delivery.
Frederic Revah, Genethon’s CEO, highlighted, “These results pave the way for next-generation gene therapies with fewer side effects. They showcase the potential of our AI-driven approach to AAV vector design, positioning Genethon as a leader in gene therapy innovation.”
Genethon’s methodology for designing AAV capsids using AI has opened up new possibilities for treating various target organs in different diseases. The research team successfully discovered several promising variants, demonstrating the potential of AI to accelerate gene therapy development.
Genethon’s pioneering work in gene therapy has led to the discovery of life-changing treatments for rare genetic diseases. The latest innovation with AI-designed capsids further enhances the efficacy and safety of gene therapies, bringing hope to patients suffering from neuromuscular diseases.
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**Categories:** News
---
### [Unveiling the Power of Targeted Radionuclide Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/unveiling-the-power-of-targeted-radionuclide-pharmaceuticals/)
**Published:** September 13, 2024
**Author:** Jon Napitupulu
**Content:**
Molecular Targeting Technologies (MTTI) and Brookline Capital Markets are collaborating at the 2nd Targeted Radionuclide Pharmaceuticals (TRPs) Supply Chain & Manufacturing Summit, held in Boston from September 24-26, 2024.
MTTI will present a workshop titled “Evaluating the Commercial & Pharmaceutical Viability of Radioisotopes to Benchmark Potential,” focusing on the emerging radiopharmaceutical market and its investment prospects.
Industry experts will discuss commercial and pharmaceutical challenges in developing radioisotopes such as actinium-225, lutetium-177, lead-212, and copper-67. They will explore topics like:
– Market demand assessment and commercial viability
– Navigating radiopharmaceutical development challenges
– Strategic partnerships and investment opportunities
Chris Pak, CEO of MTTI, highlighted the advantages of MTTI’s EB technology in overcoming challenges in TRPs. With 8 to 30-fold higher uptake at the target than standard TRPs, their patented platform offers improved efficacy and medical economics while requiring only 40% of the radiation dose.
The summit aims to enhance radiopharmaceutical production to meet the growing demand and address challenges in isotope production, radiolabeling, quality control, and supply chain logistics. It will provide valuable insights from leading companies in optimizing manufacturing processes and accelerating clinical trials.
MTTI, a clinical-stage company, is focused on developing novel targeted radiotherapeutics for rare cancers with high unmet needs. Their pipeline includes EBTATE® for neuroendocrine tumors, Hürthle cell thyroid cancer, nasopharyngeal cancer, [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/), and EBRGD™ for [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/) and glioblastoma multiforme.
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**Categories:** News
---
### [FDA Authorizes Ocrevus Zunovo™: The Revolutionary MS Treatment](https://www.clinicaltrialvanguard.com/news/fda-authorizes-ocrevus-zunovo-the-revolutionary-ms-treatment/)
**Published:** September 16, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has authorized Ocrevus Zunovo, a new subcutaneous injection, to treat relapsing and primary progressive [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS). This approval expands treatment options for MS patients, providing greater flexibility and accessibility.
Ocrevus Zunovo is a twice-a-year subcutaneous injection administered by a healthcare professional, offering an alternative to the existing intravenous (IV) formulation of Ocrevus. The safety and efficacy of Ocrevus Zunovo is supported by a decade of data from the IV formulation, with over 350,000 patients treated globally.
Clinical trials have demonstrated that Ocrevus Zunovo has a safety profile consistent with the IV formulation, demonstrating comparable suppression of relapse activity and MRI lesions. It has also been shown to be well-tolerated, with the most common side effects being injection reactions.
The approval of Ocrevus Zunovo not only provides another treatment option for MS patients but also addresses the need for flexibility and accessibility in the management of chronic conditions. By offering a subcutaneous injection option, Ocrevus Zunovo can be administered in settings where IV infrastructure may be limited, such as doctor’s offices, and can potentially reduce treatment time after the first dose.
This new treatment option empowers MS patients to make informed decisions about their care, ensuring that they have access to the most appropriate and convenient treatment approach. Ocrevus Zunovo is a significant advancement in MS management, offering greater flexibility and accessibility for patients seeking to improve their outcomes and quality of life.
Source link:
**Categories:** News
---
### [AstraZeneca and Moffitt Cancer Center: Oncology Cell Therapy Collaboration](https://www.clinicaltrialvanguard.com/news/astrazeneca-and-moffitt-cancer-center-oncology-cell-therapy-collaboration/)
**Published:** September 16, 2024
**Author:** Jon Napitupulu
**Content:**
Moffitt Cancer Center and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) have forged a strategic partnership to advance the development of cell therapies, particularly CAR-T and TCR-T treatments.
This collaboration leverages Moffitt’s clinical expertise and extensive network of cancer centers to accelerate AstraZeneca’s pipeline of cell therapies. AstraZeneca accesses Moffitt’s leading clinical environment and physician-scientist connections, facilitating the swift investigation of novel cell therapies.
Cell therapies have revolutionized cancer care, offering new treatment options for cancers with limited alternatives. Moffitt’s pivotal role in the clinical trials leading to FDA approvals for cell therapies demonstrates its capabilities in this field.
The partnership aims to overcome challenges in [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) development and expand access to these treatments. The focus is on conducting clinical studies for solid tumors and optimizing clinical operations to enhance the delivery of autologous cell therapies.
Patrick Hwu, Moffitt’s president and CEO, indicated that the collaboration with AstraZeneca will accelerate the development and delivery of cell therapies, enabling them to bring innovative treatments to patients faster and more efficiently.
Carsten Linnemann, AstraZeneca’s Head of Oncology Cell Therapy Clinical Development, suggested that the collaboration strengthens their connections with Moffitt, allowing them to accelerate the development of our autologous cell therapy pipeline and redefine cancer treatment for patients facing hematological and solid cancers.
Moffitt’s dedication to cancer prevention and cure as a National Cancer Institute-designated Comprehensive Cancer Center aligns with AstraZeneca’s global leadership in oncology. This partnership harnesses their combined expertise to advance cell therapy research and improve cancer treatment outcomes.
Source link:
**Categories:** News
---
### [Why Innovation in Big Pharma Is So Slow: Insights from BMS](https://www.clinicaltrialvanguard.com/conference-coverage/why-innovation-in-big-pharma-is-so-slow-insights-from-bms/)
**Published:** September 16, 2024
**Author:** Moe Alsumidaie
**Content:**
At the 2023 [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) conference, Hassan Kadhim from Bristol-Myers Squibb offered an insightful look into the challenges driving innovation in [Big Pharma](https://www.clinicaltrialvanguard.com/analysis/the-future-of-dcts-is-bright-according-to-big-pharma-and-fda/). His presentation, “Addressing the Innovation Gap,” shed light on the slow adoption of new technologies in clinical trials and the internal processes pharmaceutical companies use to navigate their complex, highly regulated landscape. Kadhim’s focus on structured frameworks, established capabilities, and industry collaboration gave the audience a better understanding of the forces shaping pharma innovation.
## [](#the-innovation-paradox-in-pharma)**The Innovation Paradox in Pharma**
Kadhim began by addressing the widespread frustration within the industry over the slow pace of technological adoption. He noted that technology vendors and Big Pharma colleagues often express impatience with how long new tools take to be integrated into clinical trials. The slow speed, however, stems from the industry’s complex regulatory environment. Innovations must comply with standards like Good Clinical Practice (GCP), which requires extensive validation and cautious implementation, making adopting technology time-consuming and resource-intensive.
He further emphasized the stakes in pharma, where innovation can mean the difference between life and death for patients. This heightened responsibility necessitates a careful approach, where even promising technologies, such as AI for analyzing clinical trial data, need thorough validation to ensure they meet regulatory and safety standards.
## [](#defining-innovation-in-pharma)**Defining Innovation in Pharma**
To establish common ground, Kadhim offered a broad definition of innovation, describing it as the “production, adoption, assimilation, and exploitation of value-added novelty.” This applies to various innovations—developing new drugs, adopting external technologies, or improving internal processes. Kadhim cited the implementation of electronic data capture (EDC) systems as a key innovation that replaced paper-based methods, reducing errors and speeding up clinical trial data collection.
Kadhim highlighted that while DCTs offer great potential for improving patient access and recruitment by allowing trials to be conducted remotely, they also introduce new complexities. Ensuring data integrity and maintaining regulatory compliance requires robust planning and coordination. For example, while digital tools can streamline patient engagement, they must be fully validated to ensure they can securely handle sensitive data and maintain trial integrity.
## [](#the-dichotomy-of-innovation-in-pharma)**The Dichotomy of Innovation in Pharma**
Kadhim described a dichotomy within the pharmaceutical industry: early-stage drug discovery tends to attract significant attention, with breakthroughs like gene therapy and [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/) technologies generating excitement. In contrast, innovation during clinical trials (phases II, III, and IV) is often more incremental due to the stringent regulatory environment and established protocols. The emphasis during the clinical phases shifts to safety, efficacy, and adherence to established protocols, which makes it challenging to introduce radical innovations at these stages.
## [](#the-innovation-lifecycle)**The Innovation Lifecycle**
Kadhim illustrated the biopharma drug lifecycle, which spans approximately 14 years and costs around $2 billion from discovery to market. The profit window, typically lasting three to four years, is brief, further adding pressure on companies to innovate efficiently during this time. He explained that the initial stages of drug development—discovery and preclinical testing—are resource-heavy and time-consuming. Once a drug reaches clinical trials, the focus becomes ensuring safety and efficacy, which involves meticulous planning and data analysis across multiple phases.
He also touched on the cyclic nature of innovation in Big Pharma. After a drug’s approval, companies enter a short period where they can recoup their investment and allocate resources for further innovations. However, this period is limited, as they must soon begin the process anew for the next drug in their pipeline. Kadhim highlighted that this cyclical nature often results in incremental innovations rather than disruptive changes, as companies must balance the need to innovate with maintaining financial stability.
## [](#pillars-of-sustained-innovation)**Pillars of Sustained Innovation**
Kadhim emphasized three key pillars for sustained innovation in clinical operations: incubation frameworks, established capabilities, and industry collaborations.
1. **Incubation Frameworks:** These structured processes encourage collecting, qualifying, and piloting new ideas. He provided an example from Bristol-Myers Squibb, where dedicated teams are tasked with scouting new technologies and evaluating their potential in controlled environments before broader implementation.
2. **Established Capabilities:** Kadhim noted that robust, documented processes, along with the right technologies and talent, are critical for maintaining operational efficiency while integrating innovations. Bristol-Myers Squibb, for instance, has invested in comprehensive clinical trial management systems (CTMS), which incorporate technologies like EDC and risk-based monitoring (RBM), creating a cohesive ecosystem to support trial management.
3. **Industry Collaborations:** Partnerships are essential for fostering innovation and bringing new capabilities into the company. Kadhim mentioned collaborations with organizations such as TransCelerate BioPharma and the Clinical Trials Transformation Initiative (CTTI), which provide valuable opportunities for knowledge-sharing and co-development of solutions.
## [](#interaction-of-the-pillars)**Interaction of the Pillars**
Kadhim explained how these pillars interact to support innovation within Bristol-Myers Squibb. The incubation framework collects and pilots new ideas, while collaborations provide additional insights and opportunities. These efforts are then integrated into the company’s established capabilities, guided by governance processes to ensure they deliver value.
For example, Bristol-Myers Squibb conducts internal innovation campaigns, encouraging employees to propose ideas for improving clinical trial processes. Once an idea is selected, it undergoes a thorough evaluation, including building a business case and developing a change management plan. If successful, the idea is piloted and, if viable, scaled up to become part of the company’s operational capabilities.
## [](#the-complexity-of-clinical-trials)**The Complexity of Clinical Trials**
Kadhim presented a “snake diagram” to showcase the complexity of clinical trials, highlighting various capabilities such as patient engagement, digital data flow, trial master files, and decentralized trials. He explained that each of these capabilities requires coordination across different organizational groups, and any innovation must be carefully assessed to ensure it fits within these established processes.
For instance, digital data flow—enabled by tools like electronic health records (EHRs) and wearable devices—has revolutionized real-time data analysis in trials. Yet, these tools must be integrated seamlessly into the broader trial infrastructure to maintain data integrity and compliance. Similarly, the trial master file (TMF), a repository of essential trial documents, must be managed carefully to ensure regulatory compliance. Bristol-Myers Squibb’s electronic TMF (eTMF) systems exemplify how innovation can streamline these traditionally manual processes while maintaining rigorous standards.
## [](#aligning-innovation-with-corporate-strategy)**Aligning Innovation with Corporate Strategy**
Kadhim emphasized the need to align innovation efforts with broader corporate strategies. This involves creating multi-year roadmaps, building strong business cases, and ensuring the innovations deliver measurable value. Metrics are established to track progress and ensure innovations align with company objectives.
He shared Bristol-Myers Squibb’s roadmap for implementing decentralized clinical trials, detailing the necessary steps for technology adoption, training, and regulatory compliance. By integrating DCTs into its existing processes, the company ensures these efforts align with its long-term strategic goals.
## [](#embracing-uncertainty-in-innovation)**Embracing Uncertainty in Innovation**
Kadhim concluded by acknowledging the inherent uncertainty in innovation. He urged the audience to view uncertainty as a risk and an opportunity. Innovation, by its nature, involves venturing into unknown territory, which can be daunting but is also where significant breakthroughs occur. He encouraged embracing this uncertainty while applying strategic planning and risk management to navigate it.
Bristol-Myers Squibb’s approach to managing uncertainty involves thorough risk assessments and developing contingency plans for innovation initiatives. By balancing strategic oversight with a willingness to experiment and learn from failures, the company has successfully implemented valuable innovations that enhance clinical trial processes.
## [](#summary)Summary
In summary, Kadhim’s presentation at DPHARM 2023 offered a comprehensive view of the intricate processes and challenges that drive innovation within Big Pharma. Through structured frameworks, established capabilities, and strategic collaborations, pharmaceutical companies like Bristol-Myers Squibb are navigating the complexities of clinical trials to bring new technologies to the forefront.
**Categories:** Article: Conference Coverage
---
### [Where Parexel Stands on The Future of Clinical Trials](https://www.clinicaltrialvanguard.com/executiveinterviews/where-parexel-stands-on-the-future-of-clinical-trials/)
**Published:** September 18, 2024
**Author:** Moe Alsumidaie
**Content:**
At the 2024 [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) Conference, we had the chance to interview Peyton Howell, CEO of Parexel, and she shared industry trends, challenges, and future strategies.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#moe-alsumidaie-can-you-share-your-transition-to-ceo-at-parexel-and-your-priorities-for-the-coming-year)Moe Alsumidaie: Can you share your transition to CEO at Parexel and your priorities for the coming year?
**Peyton Howell:** I’m about 100 days into my role as the new CEO of Parexel. During this time, I’ve focused on connecting closely with our customers and employees around the world. We have over 21,000 employees globally, so there’s been a lot of listening and learning. My goal is to identify what we’re doing well and where we can improve. It’s been a fast-paced 100 days, including two board meetings, but I’m very excited about the opportunities ahead.
Engaging with both customers and employees has been crucial. By understanding their perspectives, we can build on our strengths and address any challenges. The fast pace of my first 100 days, including two board meetings, highlights my commitment to making impactful changes quickly. I’m looking forward to leveraging these insights to drive Parexel forward.
Peyton Howell, CEO of Parexel
## [](#moe-alsumidaie-what-major-trends-are-you-observing-in-clinical-trials-for-2024-and-how-will-the-industry-progress)**Moe Alsumidaie:** What major trends are you observing in clinical trials for 2024, and how will the industry progress?
**Peyton Howell:** It’s an exciting time for clinical research with a lot of changes happening. The first major trend is AI and machine learning, particularly in applications that improve quality and efficiency. We’re fully invested in these technologies across our business. The second trend is patient-focused trial design. At DPHARM, this has been a big topic, emphasizing the need to make clinical trials more efficient and patient-friendly. The third trend is the next generation of decentralized clinical trials, including community-based sites. This approach helps address the challenges faced by overburdened sites and brings us closer to patients.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
These trends are shaping the future of clinical trials. AI and machine learning are enhancing quality and efficiency, while patient-focused trial design ensures that protocols are more patient-friendly. Decentralized clinical trials, particularly community-based sites, are crucial for addressing site burdens and improving patient access. By focusing on these areas, we can drive significant improvements in clinical research.
## [](#moe-alsumidaie-could-you-share-some-major-takeaways-from-your-keynote-session-at-the-conference)**Moe Alsumidaie**: Could you share some major takeaways from your keynote session at the conference?
**Peyton Howell:** The highlight of our patient keynote session was having a patient advocate from our industry who emphasized the importance of awareness about clinical trials before a diagnosis. She called on the audience to share her story and raise awareness. Additionally, she is working with the FDA to influence clinical trial design, ensuring the patient voice is heard. This session was a great reminder of the need to center our efforts around the patient.
Starting the conference with this session was powerful. It emphasized the importance of patient advocacy and the need for awareness about clinical trials. The advocate’s involvement with the FDA to influence trial design highlights the significance of incorporating the patient voice. This session served as a strong reminder to keep patients at the center of our clinical research efforts.
## [](#moe-alsumidaie-how-should-biopharmaceutical-sponsors-adapt-their-strategies-in-this-resource-constrained-environment)Moe Alsumidaie: How should biopharmaceutical sponsors adapt their strategies in this resource-constrained environment?
**Peyton Howell:** There are indeed many pressures, and we’re all being asked to do more with less. One approach is to streamline clinical trial complexity by reducing the number of procedures and visits, which can lower costs and reduce the burden on patients. Another strategy is to bring more countries into clinical research. For example, while we’re seeing growth in India, it’s still underrepresented, especially since India offers diverse patient populations and robust clinical research infrastructure. The region’s lower procedural costs and scalable infrastructure can help reduce overall clinical trial costs and increase capacity. Ensuring that clinical research is truly global is essential for addressing economic and financial challenges and will help biopharmaceutical sponsors adapt to the current financial environment.
## [](#moe-alsumidaie-how-is-parexel-leveraging-its-strategies-to-adjust-to-the-changing-market-forces)Moe Alsumidaie: How is Parexel leveraging its strategies to adjust to the changing market forces?
**Peyton Howell:** We’re actively exploring opportunities in other countries and leveraging our regulatory consulting team to assess these opportunities. We’re also using different geographic hubs to support work and reduce costs. Our sponsors are challenging us to be more efficient, and we’re looking at ways to offset costs in high-cost countries like the United States. Additionally, we’re focusing on ensuring that studies are successful and impactful for patients and sites, as struggling studies are costly for everyone involved.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#moe-alsumidaie-is-there-anything-else-youd-like-to-add-that-we-might-have-missed-in-this-interview)Moe Alsumidaie: Is there anything else you’d like to add that we might have missed in this interview?
**Peyton Howell:** Another big initiative we’re working on is talent development. We’re focused on growing our own talent and identifying new sources of talent globally. This is critical for the next generation of clinical research professionals. Additionally, everything we do is grounded in the patient, and we will continue to push the envelope on patient-centric clinical research. These initiatives are crucial for our success and the future of clinical research.
**Categories:** Article: Conference Coverage, Article: Executive Interviews
---
### [FDA Approves Keytruda® Plus Pemetrexed and Platinum Chemotherapy for Malignant Pleural Mesothelioma](https://www.clinicaltrialvanguard.com/news/fda-approves-keytruda-plus-pemetrexed-and-platinum-chemotherapy-for-malignant-pleural-mesothelioma/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has approved [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/), in combination with pemetrexed and platinum chemotherapy, as a first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma (MPM).
The approval is based on the Phase 3 CCTG IND.227/[KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-483 trial. In this trial, KEYTRUDA plus chemotherapy showed a statistically significant improvement in overall survival (OS) compared to chemotherapy alone. The risk of death was reduced by 21%, with a median OS of 17.3 months for the combination therapy compared to 16.1 months for chemotherapy alone.
KEYTRUDA plus chemotherapy improved progression-free survival (PFS) and overall response rate (ORR). Median PFS was 7.1 months for the combination therapy and 7.1 months for chemotherapy alone. ORR was significantly higher for the combination therapy at 52% compared to 29% for chemotherapy alone.
KEYTRUDA is an anti-PD-1 therapy that blocks the PD-1 protein in immune cells, allowing them to recognize better and attack cancer cells. It is generally well-tolerated, but immune-mediated adverse reactions may occur. Early identification and management of these reactions are crucial.
The approval of KEYTRUDA plus chemotherapy provides a new first-line treatment option for patients with MPM, a challenging disease with limited treatment options. This milestone reflects the ongoing commitment to advancing research and developing innovative therapies for patients with difficult-to-treat tumors.
Source link:
**Categories:** News
---
### [New Zeposia Data Reveal Durable Efficacy and Unwavering Safety in Relapsing MS](https://www.clinicaltrialvanguard.com/news/new-zeposia-data-reveal-durable-efficacy-and-unwavering-safety-in-relapsing-ms/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
According to new data, a Phase 3 extension study of Zeposia for [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS) found a sustained decrease in brain volume loss over five years.
The open-label extension of the DAYBREAK trial showed that patients treated with Zeposia experienced low and stable rates of brain volume loss. The annualized rate of loss for whole brain volume was -0.27% for patients in the RADIANCE trial and -0.35% for those in the SUNBEAM trial at Month 60.
Furthermore, a separate safety analysis showed declining or stable rates of adverse events over eight years of Zeposia treatment. Rates of infections, serious infections, and opportunistic infections remained relatively low.
“Brain volume loss can lead to significant cognitive decline if MS is not treated early,” said Jeffrey Cohen, an MD and paid consultant for Bristol Myers Squibb. “These new results reinforce the safety and efficacy of Zeposia as an effective treatment for MS.”
The DAYBREAK OLE trial included over 2,250 patients from previous Phase 3 trials. The trial evaluated brain volume loss and compared data from patients switching from interferon beta-1a (IFN-β) to Zeposia. The results showed consistent reductions in brain volume loss rates with Zeposia treatment.
Another finding from the trial was the observation of high annualized loss in cortical grey matter volume with IFN-β, which reversed and showed an increase after switching to Zeposia.
Overall, the new data from the DAYBREAK extension study provides further evidence of the sustained efficacy and safety of Zeposia in treating MS.
Source link:
**Categories:** News
---
### [Fasenra: A Breakthrough in Treating Eosinophilic Granulomatosis with Polyangiitis](https://www.clinicaltrialvanguard.com/news/fasenra-a-breakthrough-in-treating-eosinophilic-granulomatosis-with-polyangiitis/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
[AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)‘s FASENRA ([benralizumab](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-trial-that-should-rewrite-the-rare-disease-playbook-but-probably-wont/)) has received FDA approval for treating adults with eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune vasculitis that affects various organs and can be life-threatening if left untreated.
The approval stems from the positive results of the MANDARA Phase III trial, where FASENRA demonstrated comparable efficacy to mepolizumab, the only previously approved treatment for EGPA. In the trial, nearly 60% of patients treated with FASENRA achieved remission, a crucial goal in treating EGPA.
Moreover, FASENRA showed a substantial benefit in reducing oral corticosteroid (OCS) dependence. Notably, 41% of patients fully discontinued OCS use, compared to 26% in the mepolizumab group. This finding is significant as long-term OCS use carries severe side effects.
EGPA often coexists with adult-onset severe eosinophilic [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/) (SEA), and FASENRA is now the second biologic approved for both conditions. It is currently used as a maintenance treatment for SEA in many countries and is also approved for children and adolescents over the age of six.
The FDA’s Orphan Drug Designation for FASENRA in EGPA highlights its potential to address a rare and challenging disease. With its convenient monthly subcutaneous injection, FASENRA provides a promising new treatment option for EGPA patients, offering the hope of achieving remission and reducing steroid dependency.
Source link:
**Categories:** News
---
### [Tivic Health Study Ready for Enrollment With Non-Invasive Vagus Nerve Stimulation Technology](https://www.clinicaltrialvanguard.com/news/tivic-health-study-ready-for-enrollment-with-non-invasive-vagus-nerve-stimulation-technology/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
Tivic Health has received approval for the next phase of clinical research on its non-invasive cervical vagus nerve stimulation (ncVNS) approach. This collaboration with Feinstein Institutes aims to refine the stimulation parameters of the ncVNS technology.
Physiological measurements will guide device performance enhancements, including electrode positioning and waveform optimizations. The data will inform clinical trial designs and target specific patient populations.
The company has successfully completed Phase 1 studies on ncVNS and is exploring clinical applications with Fletcher Spaght.
The optimization study will be conducted by Feinstein Institute of Bioelectronic Medicine, led by Dr. Theodoros Zanos and Dr. Blake Gurfein, Tivic Health’s Chief Scientific Officer.
Precision optimization is crucial in non-invasive neurostimulation device development. The research team will advance the ncVNS technology from proof-of-concept to disease-specific validation in future clinical trials.
The global vagus nerve stimulation market is estimated to reach $21.3 billion by 2030, with a 10.6% CAGR. Tivic Health aims to become a market leader in bioelectronic medicine by providing personalized and effective non-invasive treatments.
Tivic Health is a [health tech](https://www.clinicaltrialvanguard.com/news/allez-healths-60-million-capital-raise-a-monumental-breakthrough-in-health-tech/) company that develops and commercializes bioelectronic medicine. It utilizes stimulation of various nerve structures to treat chronic health conditions.
Tivic Health’s ClearUP, an FDA-approved bioelectronic sinus device, offers a drug-free therapeutic solution with a proven safety profile.
Source link:
**Categories:** News
---
### [CD137 Antibodies: A Promising Cancer Immunotherapy Target](https://www.clinicaltrialvanguard.com/news/cd137-antibodies-a-promising-cancer-immunotherapy-target/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
CD137 (4-1BB) is a promising target for cancer immunotherapy. Its ability to stimulate T cells and enhance tumor cell killing has drawn attention from researchers and pharmaceutical companies.
Despite the absence of approved CD137-targeted therapies, numerous candidates are under clinical development. Major companies like [BioNTech](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) and Shanghai Henlius Biotech are actively involved.
CD137 antibodies, particularly agonistic antibodies, are the most advanced in development. These antibodies aim to activate CD137 signaling, boosting T-cell activity. Early clinical results have shown promising anti-tumor effects.
However, challenges such as hepatotoxicity have emerged. Therefore, alternative strategies like bispecific antibodies and tailored ligands are being explored for improved safety and efficacy.
Combining CD137-targeted therapies with other immunomodulators holds great promise. Preclinical and early clinical studies have demonstrated synergistic effects when paired with checkpoint inhibitors or other therapies. This approach enhances anti-tumor responses, addressing the unmet need for effective treatments.
The potential market for CD137 antibodies is substantial, given their broad applications in cancer. The development of these therapies is expected to improve outcomes and expand treatment options for various cancer types.
Source link: [http://www.businesswire.com/news/home/20240918752552/en/CD137-Antibodies-Clinical-Trials-Market-Opportunity-Insight-2027—Over-80-Drugs-In-Clinical-Trials—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20240918752552/en/CD137-Antibodies-Clinical-Trials-Market-Opportunity-Insight-2027---Over-80-Drugs-In-Clinical-Trials---ResearchAndMarkets.com)
**Categories:** News
**Tags:** eClinical Tech News
---
### [DeepCure: Unveiling the Potential of BD2 Inhibitor DC-9476 in Macrophage Activation Syndrome](https://www.clinicaltrialvanguard.com/news/deepcure-unveiling-the-potential-of-bd2-inhibitor-dc-9476-in-macrophage-activation-syndrome/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
DeepCure, a company employing AI for drug discovery, will present preclinical data on their BRD4 Inhibitor, [DC-9476](https://www.clinicaltrialvanguard.com/news/deepcure-presents-breakthrough-data-dc-9476-triumphs-over-etanercept-in-rheumatoid-arthritis-battle/), at the EMBO 2024 Conference.
DC-9476 targets macrophage activation syndrome (MAS), a life-threatening condition often accompanying juvenile and adult-onset Still’s disease. The current standard treatment, glucocorticoids, has limitations, creating a need for effective alternatives.
Studies reveal that DC-9476 efficiently blocked MAS progression in animal models. It outperformed dexamethasone to restore near-normal white blood cell counts and reduce serum ferritin, a MAS biomarker.
DC-9476 works by directly inhibiting macrophage activation, effectively decreasing MAS-related cytokines like IL-1β, [IL-18](https://www.clinicaltrialvanguard.com/news/gileads-848m-bet-on-compugens-revolutionary-therapy/), IL-6, and IFN-γ. This mechanism differentiates it from non-selective BRD4 inhibitors and existing therapies that only target the inflammasome and downstream cytokines.
Unlike non-selective BRD4 inhibitors, DC-9476 does not lower platelet levels, addressing a known side effect of such therapies. Kfir Schreiber, CEO of DeepCure, expresses enthusiasm for DC-9476’s potential in treating MAS, highlighting its direct macrophage activation inhibition.
The company plans to advance DC-9476 into clinical trials as a promising therapeutic option for patients with MAS and Still’s disease.
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**Categories:** News
---
### [Exelixis Announces Phase 3 CABINET Study Results Unveiled at ESMO 2024](https://www.clinicaltrialvanguard.com/news/exelixis-announces-phase-3-cabinet-study-results-unveiled-at-esmo-2024/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
The phase 3 [CABINET](https://www.clinicaltrialvanguard.com/news/exelixis-announces-results-from-cabinet-subgroup-analysis-at-asco-gi-2025/) trial evaluated [cabozantinib](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) versus placebo in patients with previously treated neuroendocrine tumors. Data from this trial showed significant improvement in progression-free survival (PFS) with cabozantinib in both pancreatic neuroendocrine tumors (pNET) and extra-pancreatic NET (epNET) cohorts.
In the pNET cohort, cabozantinib led to a median PFS of 13.8 months compared to 4.4 months with placebo. For epNET, median PFS was 8.4 months with cabozantinib and 3.9 months with placebo.
Additional analyses revealed benefits with cabozantinib across various clinical subgroups, including primary tumor site, grade, and prior systemic therapy. The objective response rate (ORR) was 19% in the pNET cohort and 5% in the epNET cohort, indicating tumor shrinkage in patients receiving cabozantinib.
Interim overall survival (OS) results were similar between cabozantinib and placebo, with hazard ratios of 0.95 for pNET and 0.86 for epNET.
The CABINET study enrolled a diverse patient population, reflecting real-world clinical practice. The final results published in the New England Journal of Medicine support the potential role of cabozantinib as a new standard of care for patients with advanced neuroendocrine tumors.
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**Categories:** News
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### [Massive Bio Inroduces Global Platform Launches to Enhance Patient Access to Clinical Trials](https://www.clinicaltrialvanguard.com/news/massive-bio-inroduces-global-platform-launches-to-enhance-patient-access-to-clinical-trials/)
**Published:** September 19, 2024
**Author:** Jon Napitupulu
**Content:**
[Massive Bio](https://www.clinicaltrialvanguard.com/news/massive-bio-advancing-cancer-care-with-ai-driven-clinical-trials/), an industry leader in AI-powered clinical trial enrollment, has introduced Patient Connect, an innovative portal designed to enhance cancer patients’ access to clinical trials worldwide.
Patient Connect empowers patients by providing:
• Access to their medical records to enable in-depth AI analysis
• Identification of optimal clinical trial options based on their medical profile
• Personalized clinical trial matching reports for informed decision-making
Patients can access their matching results for over 16,000 cancer trials with a simple sign-up. To date, Massive Bio has connected patients to over 33,000 clinical trial sites, showcasing the efficiency of its AI platform.
Beyond technological solutions, Massive Bio offers personalized support through its concierge services. Patient relations coordinators, MDs, and oncology nurses provide guidance and assistance throughout the enrollment process, addressing any concerns or logistical challenges.
For healthcare providers, Massive Bio’s Clinical Network empowers them to:
• Review patients’ pre-screening results
• Monitor the enrollment process
This comprehensive patient-provider engagement model increases clinical trial enrollment globally, offering hope for better cancer outcomes.
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**Categories:** News
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### [Cybin Completes Announced Share Consolidation](https://www.clinicaltrialvanguard.com/news/cybin-completes-announced-share-consolidation/)
**Published:** September 20, 2024
**Author:** Jon Napitupulu
**Content:**
Toronto-based Cybin Inc., a clinical-stage neuropsychiatry company focused on developing novel treatments for mental health conditions, has consolidated its common shares. Effective immediately, this adjustment combines every 38 existing shares into one new one.
The consolidation has reduced the number of outstanding common shares from 759,692,495 to approximately 19,991,907. While each shareholder’s proportionate ownership and voting power remain unchanged, fractional shares have been adjusted appropriately.
The exercise price and issuable shares of the company’s outstanding warrants and options have been adjusted to align with the consolidation.
Cybin remains committed to developing innovative therapeutics for mental health. The company aims to revolutionize mental healthcare through partnerships with world-class scientists by advancing proprietary drug platforms, novel delivery systems, and new treatment regimens.
Cybin is currently developing CYB003 for [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) and [CYB004](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) for generalized anxiety disorder. The company maintains operations in Canada, the United States, the United Kingdom, the Netherlands, and Ireland.
Forward-looking statements in this announcement are subject to risks and uncertainties that may affect actual results. These statements are based on current expectations and are not guarantees of future performance.
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**Categories:** News
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### [Cybin Announces Groundbreaking Clinical Progress, Unveiling Transformative Therapies for Mental Health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/)
**Published:** September 20, 2024
**Author:** Jon Napitupulu
**Content:**
Cybin Inc., a clinical-stage biotech company, is advancing its clinical programs for neuropsychiatric disorders. The company’s lead programs, [CYB003](https://www.clinicaltrialvanguard.com/news/cybins-breakthrough-therapy-for-major-depressive-disorder-a-game-changer-in-mental-health/) and [CYB004](https://www.clinicaltrialvanguard.com/news/cybin-unveils-strategies-for-cyb003-cyb004-psychedelic-drug-development/), are entering late-stage development.
CYB003, a proprietary deuterated psilocin, is being developed for MDD. It received FDA Breakthrough Therapy Designation based on promising preliminary results. A Phase 3 study in MDD will commence shortly, with 30 high-quality clinical sites involved. The study aims to address methodological challenges common in this drug class.
Phase 2 results demonstrated rapid and significant improvements in depressive symptoms with a single dose and sustained effects four months after two doses. The 16mg dose group achieved a 75% remission rate. Additional 12-month Phase 2 efficacy data is anticipated in early Q4 2024.
CYB004, a deuterated N,N-dimethyltryptamine, is being developed for GAD. Its intramuscular formulation is designed for optimal delivery. Phase 2 efficacy and safety results are expected in late 2024 or early 2025.
Cybin has strengthened its R&D team by adding Dr. Atul R. Mahableshwarkar and Dr. Tom Macek, who will lead the CYB003 and CYB004 programs. These experienced drug development executives bring expertise to advance these clinical initiatives.
Cybin believes that with its advanced clinical programs and experienced team, it is well-positioned as a leader in neuropsychiatry and has the potential to transform mental healthcare with innovative treatments for challenging disorders.
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### [Unveiling the Transformative Power of Takeda's Narcolepsy Treatment: Clinical Trial Triumphs at Sleep Europe 2024](https://www.clinicaltrialvanguard.com/news/unveiling-the-transformative-power-of-takedas-narcolepsy-treatment-clinical-trial-triumphs-at-sleep-europe-2024/)
**Published:** September 20, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda will showcase data from its Phase 2b trials and long-term extension study of [TAK-861](https://www.clinicaltrialvanguard.com/news/takedas-tak-861-phase-2b-data-a-breakthrough-for-narcolepsy-type-1/), an investigational orexin receptor 2 agonist, for narcolepsy type 1 (NT1) at Sleep Europe 2024.
TAK-861 aims to address the orexin deficiency in NT1 by stimulating the orexin receptor 2. Promising Phase 2b results have led to initiating the FirstLight Study, a global Phase 3 trial assessing TAK-861’s efficacy and safety in adults with NT1.
Data from the Phase 2b trials will highlight TAK-861’s impact on cognitive impairment, nocturnal sleep quality, and sustained attention in NT1 patients. Additionally, interim safety and efficacy results from the ongoing long-term extension study will be presented.
Elena Koundourakis of Takeda emphasizes that NT1 patients experience debilitating symptoms beyond excessive daytime sleepiness, and Takeda is committed to advancing orexin research.
These data presentations contribute to the growing understanding of orexin agonists in treating NT1. Takeda continues to develop targeted therapies for individuals with sleep-wake disorders, including NT2 and idiopathic hypersomnia.
More information about the FirstLight Study, actively recruiting participants, is available at [clinicaltrials](https://www.clinicaltrialvanguard.com/article/fda-needs-to-release-clinicaltrials-gov-guidance-now/).gov (NCT06470828) and firstlightstudy.com (for US audiences). Takeda currently has no approved therapies for narcolepsy.
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**Categories:** News
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### [AngioDynamics Initiates Landmark Trial for Pulmonary Embolism Treatment](https://www.clinicaltrialvanguard.com/news/angiodynamics-initiates-landmark-trial-for-pulmonary-embolism-treatment/)
**Published:** September 20, 2024
**Author:** Jon Napitupulu
**Content:**
[AngioDynamics](https://www.clinicaltrialvanguard.com/news/angiodynamics-auryon-system-now-available-in-europe/) has initiated a comprehensive study to evaluate the efficacy and safety of the [AlphaVac](https://www.clinicaltrialvanguard.com/news/unlock-the-extraordinary-angiodynamics-alphavac-f18%e2%81%b8%e2%81%b5-revolutionizes-vascular-health/) Mechanical Aspiration Thrombectomy System for treating acute, intermediate-risk pulmonary embolism (PE) in Europe.
The RECOVER-AV trial will recruit patients at multiple European hospital sites and assess the system’s ability to reduce the ratio of right ventricular (RV) to left ventricular (LV) volumes, a key indicator of heart function, within 48 hours of the procedure. The study will also monitor the incidence of adverse events related to the device or bleeding within seven days.
Building upon the positive results of the APEX-AV study in the United States, RECOVER-AV aims to further establish the AlphaVac F1885 System as a safe and effective treatment for intermediate-risk PE in Europe. With an estimated 435,000 PE events annually in the European Union, the prevalence of severe PE is higher in Europe compared to the United States.
The study will follow patients for 12 months, assessing their functional outcomes at 30 days, six months, and one year. This comprehensive monitoring will provide insights into the long-term benefits of the AlphaVac system.
The CE Mark approval of the AlphaVac F1885 System in Europe marks a significant milestone for AngioDynamics, allowing the company to expand its reach in a region with a substantial prevalence of PE. The company is committed to generating robust clinical evidence worldwide, demonstrating its dedication to improving patient care and treating vascular diseases.
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**Categories:** News
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### [Introducing Ultherapy PRIME: Revolutionizing Non-Surgical Skin Lifting](https://www.clinicaltrialvanguard.com/news/introducing-ultherapy-prime-revolutionizing-non-surgical-skin-lifting/)
**Published:** September 24, 2024
**Author:** Jon Napitupulu
**Content:**
Ultherapy [PRIME](https://www.clinicaltrialvanguard.com/news/tobevibart-elebsiran-get-breakthrough-prime-for-chronic-hep-d/), a groundbreaking ultrasound technology, significantly advances noninvasive skin treatments. With real-time imaging, it delivers personalized and long-lasting lifts, targeting precise tissue layers where collagen and elastin reside.
Ultherapy PRIME builds upon the legacy of Ultherapy, recognized as the gold standard for skin tightening and lifting. Clinical studies and over 56 clinical trials have established its safety and efficacy, ensuring patient satisfaction with natural-looking results lasting up to a year or more.
Ultherapy PRIME’s advanced technology allows for customized treatments tailored to each patient’s unique skin needs. It targets the appropriate tissue layers, delivering precise energy to maximize results. Whether it’s for lifting the brows, smoothing lines and wrinkles on the neck, or enhancing chest skin, Ultherapy PRIME offers a solution for all skin types and tones.
The upgraded platform features a modern design with faster processing and elevated ergonomics, enhancing the workflow for healthcare professionals. A larger screen and brighter images provide unparalleled visualization, allowing precise targeting and consistent results.
Merz Aesthetics, the leader in noninvasive lifting, continues to innovate with Ultherapy PRIME. Its commitment to research and development has resulted in a premier platform that provides a truly personalized and long-lasting solution for patients seeking a youthful and rejuvenated appearance.
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### [MedinCell Partner Teva Delivers Unprecedented Antipsychotic Data](https://www.clinicaltrialvanguard.com/news/medincell-partner-teva-delivers-unprecedented-antipsychotic-data/)
**Published:** September 24, 2024
**Author:** Jon Napitupulu
**Content:**
A positive efficacy, safety, and tolerability profile has emerged from [Medincell](https://www.clinicaltrialvanguard.com/news/medincell-teva-olanzapine-lai-phase-3-positive-uzedy-real-world-data/) and Teva’s Phase 3 SOLARIS trial evaluating Olanzapine LAI in patients with [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/).
Key Findings
• Efficacy results at 8 weeks in Period 1 demonstrated significant patient improvements across multiple parameters.
• Systemic safety data aligned with other olanzapine formulations; no new safety signals were identified.
• No cases of post-injection delirium sedation syndrome (PDSS) have been observed to date, which is significant as it removes a major barrier to using intramuscular olanzapine LAI.
The SOLARIS trial is a randomized, double-blind, placebo-controlled study followed by an open-label safety period. It enrolled patients aged 18-64 diagnosed with schizophrenia.
Olanzapine LAI is an investigational once-monthly subcutaneous injection of Olanzapine, an atypical antipsychotic. Its potential to be the first long-acting Olanzapine with a favorable safety profile differentiates it from other LAIs of Olanzapine, which carry an FDA black box warning for PDSS.
Long-term safety and the incidence of PDSS are being assessed in the ongoing open-label Period 2 of the SOLARIS study, with topline results anticipated in the first half of 2025.
Teva is leading the development and commercialization of Olanzapine LAI. Medincell, its partner, is eligible for up to $117 million in development and commercial milestones and royalties on net sales.
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### [FDA Accepts LEO Pharma's NDA for Delgocitinib Cream for Chronic Hand Eczema](https://www.clinicaltrialvanguard.com/news/fda-accepts-leo-pharmas-nda-for-delgocitinib-cream-for-chronic-hand-eczema/)
**Published:** September 24, 2024
**Author:** Jon Napitupulu
**Content:**
LEO Pharma has announced the acceptance of its New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for [delgocitinib](https://www.clinicaltrialvanguard.com/news/anzupgo-delgocitinib-cream-approved-in-great-britain/) cream 20 mg/g for adults with moderate to severe Chronic Hand Eczema (CHE). This submission marks a significant milestone in LEO Pharma’s efforts to bring this innovative treatment to patients in the United States.
Delgocitinib cream is a topical pan-JAK inhibitor that inhibits JAK-STAT signaling, a key pathway in the development of CHE. The NDA is based on positive results from Phase 3 clinical trials, which showed the drug’s safety and efficacy in treating CHE.
“This is an important step in our journey to provide new treatment options for adults suffering from moderate to severe CHE,” said Christophe Bourdon, CEO of LEO Pharma A/S. “We are committed to making a difference for those who need us most in dermatology.”
CHE is a debilitating condition that affects the hands, causing severe itching, pain, and skin inflammation. It can have a significant impact on patients’ quality of life, including their ability to work and perform everyday activities.
LEO Pharma emphasizes its commitment to addressing the unmet needs of CHE patients. The company has been actively strengthening its presence in the U.S. to support healthcare professionals in improving the lives of those living with dermatological conditions.
The FDA’s review of the NDA for delgocitinib cream is ongoing, and the company anticipates a decision by the end of 2023. If approved, delgocitinib cream would be the first FDA-approved treatment specifically for moderate to severe CHE.
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**Categories:** News
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### [Trisalus: Improved Tumor Delivery with PEDD™ Method](https://www.clinicaltrialvanguard.com/news/trisalus-improved-tumor-delivery-with-pedd-method/)
**Published:** September 24, 2024
**Author:** Jon Napitupulu
**Content:**
TriSalus [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/)‘ Pressure Enabled Drug Delivery (PEDD) method enhances the delivery of glass microspheres into liver tumors in a porcine model. Published in the Journal of Vascular and Interventional Radiology, the study demonstrates that PEDD significantly improves penetration of glass microspheres into tumors using the TriNav Infusion System.
Compared to conventional microcatheters, the PEDD method showcased deeper penetration of glass microspheres into liver tumors and surrounding tissue. This advanced delivery method offers the potential for increased therapeutic efficacy and reduced side effects.
Bryan F. Cox, TriSalus’ Chief of Research, emphasizes the significance of PEDD in addressing challenges in cancer care. The enhanced delivery observed in the study validates the method’s ability to target tumors with therapeutics selectively.
Mary Szela, TriSalus’ CEO, highlights the practical implications of PEDD. The efficient delivery of therapeutics to tumor cells without affecting healthy tissue remains a challenge in cancer treatment, and PEDD aims to overcome this barrier.
The study findings suggest that the PEDD method improves glass microsphere delivery regardless of its placement (lobar or selective infusions). The TriNav device enables controlled and targeted delivery of glass microspheres and other drugs into tumors.
Dr. Riad Salem, Chief of Interventional Radiology, emphasizes the potential of PEDD in addressing unmet needs in [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/) therapy. The increased concentration of microspheres in tumors compared to surrounding liver tissue suggests improved efficacy and safety. Further validation in human models could lead to significant advancements in liver cancer management.
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### [Merck's Keytruda® Wins Japan Approval for Advanced Lung and Bladder Cancers](https://www.clinicaltrialvanguard.com/news/mercks-keytruda-wins-japan-approval-for-advanced-lung-and-bladder-cancers/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Japan’s Ministry of Health has approved new indications for [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/) (pembrolizumab) in treating certain lung and urothelial cancers.
KEYTRUDA, combined with chemotherapy, has been approved for preoperative (neoadjuvant) and follow-up (adjuvant) treatment of NSCLC patients. This approval is based on the [KEYNOTE](https://www.clinicaltrialvanguard.com/news/mercks-keynote-b21-trial-failed-to-meet-primary-endpoint-for-endrometrial-cancer/)-671 trial, which demonstrated improved overall and event-free survival in patients receiving the KEYTRUDA regimen compared to placebo.
Lung cancer is a global scourge, responsible for the most cancer deaths worldwide. NSCLC is the most prevalent type of lung cancer, and these new approvals offer hope to patients in Japan.
KEYTRUDA, in combination with [enfortumab](https://www.clinicaltrialvanguard.com/news/padcev-plus-keytruda-shows-long-term-efficacy-in-urothelial-cancer/) vedotin, has been approved for first-line treatment of radically unresectable urothelial carcinoma. This approval stems from the KEYNOTE-A39 trial, which showed the combination’s effectiveness in improving patient outcomes compared to standard chemotherapy.
Urothelial carcinoma is a cancer of the lining of the urinary tract. The new approval provides a novel treatment option for patients with advanced disease.
These approvals underscore KEYTRUDA’s versatility and importance in the treatment of various cancers. It can be used alone or in combination with other therapies, offering personalized treatment approaches for patients in Japan.
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### [Investigational Favezelimab and Pembrolizumab Combo Shows Promise in Colorectal Cancer](https://www.clinicaltrialvanguard.com/news/investigational-favezelimab-and-pembrolizumab-combo-shows-promise-in-colorectal-cancer/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Merck’s Phase 3 KEYFORM-007 trial tested the efficacy of a fixed-dose combination of favezelimab and pembrolizumab in treating PD-L1-positive microsatellite-stable metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC). The trial failed to meet its primary endpoint of improved overall survival (OS) compared to the standard of care (regorafenib or TAS-102).
The safety profile of the combination was consistent with previous studies, with no new concerns identified. Merck will continue to analyze the data and share the results with researchers.
Despite the negative outcome of KEYFORM-007, Merck remains committed to exploring [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/)-based combinations and other novel candidates for patients with colorectal cancer who have limited treatment options. KEYTRUDA is currently approved in the U.S. for treating microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer but not for MSS mCRC.
The fixed-dose combination of favezelimab and pembrolizumab is also being investigated in other cancer types, including certain hematologic malignancies and solid tumors. Notable ongoing trials include KEYFORM-008, which evaluates the combination in patients with relapsed or refractory classical Hodgkin lymphoma.
Colorectal cancer remains a significant health concern, ranking as the third most commonly diagnosed and second most common cause of cancer-related deaths globally. Early detection and screening are crucial to improve outcomes.
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### [Pfizer Recalls Worldwide Supplies of Sickle Cell Disease Treatment](https://www.clinicaltrialvanguard.com/news/pfizer-recalls-worldwide-supplies-of-sickle-cell-disease-treatment/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer has voluntarily withdrawn OXBRYTA (voxelotor) from all markets due to an imbalance in vaso-occlusive crises and fatal events observed in clinical data. The company is also discontinuing clinical trials and expanded access programs for the drug worldwide.
According to Pfizer, the overall benefit of OXBRYTA no longer outweighs the risk in the approved patient population with [sickle cell](https://www.clinicaltrialvanguard.com/news/mitapivat-met-hemoglobin-goal-in-sickle-cell-phase-3-but-only-40-6-of-patients-responded/)[sickle cell disease](https://www.clinicaltrialvanguard.com/news/agios-discontinues-tebapivat-development-in-sickle-cell-disease/) (SCD). The data suggests increased incidences of serious events, prompting the company’s decision to withdraw the drug while further investigating the findings.
Pfizer’s Chief Medical Officer, Aida Habtezion, emphasized the importance of patient safety, stating that the action was taken in the best interests of patients. Habtezion acknowledged the challenges of treating SCD and the limited treatment options available.
Pfizer has notified regulatory authorities and advised patients to consult with their physicians for alternative treatments. Patients and healthcare professionals with questions about OXBRYTA can contact Pfizer Medical Information at 1-800-438-1985.
The withdrawal of OXBRYTA underscores the ongoing challenges in treating SCD, a debilitating and life-threatening blood disorder that affects millions worldwide. It also highlights the importance of ongoing research and the need for safer and more effective therapies.
SCD is an inherited disorder that causes sickle-shaped red blood cells, leading to inflammation, anemia, and organ damage. It is a chronic and potentially life-threatening condition that manifests in childhood and can significantly shorten life expectancy.
OXBRYTA was approved in 2019 for the treatment of SCD. It worked by increasing hemoglobin’s affinity for oxygen, thus inhibiting red blood cell sickling and destruction.
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**Categories:** News
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### [Genialis Unveils RNA- and ML-Powered Biomarker for Cancer Treatment Selection](https://www.clinicaltrialvanguard.com/news/genialis-unveils-rna-and-ml-powered-biomarker-for-cancer-treatment-selection/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Genialis, a leader in RNA biomarker development, has announced the launch of Genialis krasID, a groundbreaking biomarker capable of predicting patient response and benefits from KRAS inhibitors (KRASi). This biomarker algorithm is universally applicable across tissue types and KRAS mutation statuses.
Genialis krasID harnesses machine learning to analyze gene expression patterns in patient tumors, revealing a wealth of information far beyond traditional mutation-based diagnostics. Validation studies on real-world data have shown its effectiveness in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC), [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (CRC), and pancreatic ductal adenocarcinoma (PDAC).
This biomarker has immense implications for all stages of drug development, from preclinical compound selection to clinical trial patient enrollment and therapy optimization for individual patients. By identifying the most suitable patients for KRASi treatment and predicting the benefit duration, Genialis krasID empowers personalized care strategies to improve treatment outcomes.
Currently, KRAS-positive patients are only selected for therapy based on mutation status, a limited approach that fails to account for individual patient variability. Genialis krasID addresses this gap by providing highly accurate predictions of patient response, allowing clinicians to tailor treatment decisions accordingly.
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### [City of Hope's Horizon: City of Hope Gets $20M for Pancreatic Disease Breakthroughs](https://www.clinicaltrialvanguard.com/news/city-of-hopes-horizon-city-of-hope-gets-20m-for-pancreatic-disease-breakthroughs/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
City of Hope, a leading cancer research and treatment institution, has received a $20 million donation from philanthropists Norman and Melinda Payson. This significant gift will establish a dedicated pancreas center to accelerate groundbreaking research and treatments for [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) and diabetes.
Inspired by the personal impact of pancreatic cancer on his mother, Norman Payson, a noted healthcare executive and City of Hope board director, expressed confidence in the organization’s capabilities. “City of Hope possesses the unique expertise to drive innovative cures for pancreatic cancer and diabetes,” he stated. “We are committed to advancing this research and bringing hope to countless individuals.”
City of Hope is renowned for its groundbreaking work in both cancer and diabetes research. The institution investigates the connection between these diseases, explores cellular immunotherapy, develops early detection methods for pancreatic cancer, and pursues the development of a pancreatic cancer vaccine.
The Paysons’ gift follows a historic $150 million donation from A. Emmet Stephenson Jr. and Tessa Stephenson Brand to fund pancreatic cancer research. These substantial contributions highlight the growing momentum in the fight against these deadly diseases.
Robert Stone, CEO of City of Hope, indicated that transformative ideas often emerge from identifying challenges and envisioning solutions that others may deem unattainable. Through collaborations with visionary partners like the Paysons, they are pushing the boundaries of medicine.
City of Hope continues to expand its presence in major metropolitan areas, including Orange County. The opening of the City of Hope Orange County Lennar Foundation Cancer Center in Irvine in 2022 brought cutting-edge cancer care to the region.
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### [LEO Pharma Unveils Remarkable Results of ANZUPGO® Cream in Chronic Hand Eczema](https://www.clinicaltrialvanguard.com/news/leo-pharma-unveils-remarkable-results-of-anzupgo-cream-in-chronic-hand-eczema/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
LEO Pharma presented data at the 33rd European Academy of Dermatology and Venereology (EADV) Congress on the effectiveness and safety of [delgocitinib](https://www.clinicaltrialvanguard.com/news/anzupgo-delgocitinib-cream-approved-in-great-britain/) cream for moderate to severe chronic hand eczema (CHE).
The DELTA FORCE trial, a 12-week phase 3 study, compared delgocitinib cream to oral alitretinoin capsules. Delgocitinib cream showed superior efficacy, with a greater reduction in Hand Eczema Severity Index (HECSI) score than alitretinoin. It also achieved higher rates of Investigator’s Global Assessment (IGA)-CHE treatment success.
In addition to DELTA FORCE, other research presented at EADV included an analysis of 638 patients treated with delgocitinib cream across multiple clinical trials. The analysis demonstrated the drug’s effectiveness across five CHE subtypes: irritant contact dermatitis, vesicular hand eczema, allergic contact dermatitis, atopic hand eczema, and hyperkeratotic hand eczema.
Findings from the RWEAL study, which involved 1939 CHE patients, highlighted the diverse nature of the condition, with patients often presenting with multiple subtypes.
Delgocitinib cream, if approved, would provide a topical alternative to alitretinoin for severe CHE patients who do not respond adequately to topical corticosteroids. It has demonstrated promising results in both clinical trials and real-world settings, addressing an unmet need in CHE treatment.
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**Categories:** News
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### [Metsera Announces Positive Phase 1 Results for MET-097, an Ultra-Long-Acting GLP-1 Agonist](https://www.clinicaltrialvanguard.com/news/metsera-announces-positive-phase-1-results-for-met-097-an-ultra-long-acting-glp-1-agonist/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
In a Phase 1 clinical trial, Metsera’s MET-097 exhibited promising weight loss outcomes, supporting its potential for once-monthly dosing. The 125 participants in the trial received various doses of MET-097.
Key Findings:
• MET-097 demonstrated significant and sustained weight loss, achieving a 7.5% reduction in body weight.
• The weight loss was maintained for at least 4 weeks after the last dose, indicating extended efficacy.
• MET-097’s long half-life of 380 hours enables potential monthly or less frequent dosing, eliminating the need for titration.
Benefits and Impact:
• Once-monthly dosing simplifies administration and improves patient adherence.
• The extended efficacy reduces the burden of frequent injections.
• Metsera’s HALO™ platform technology underlies MET-097, providing a unique advantage in achieving ultra-long-acting GLP-1 therapies.
Next Steps:
• Metsera plans to initiate a Phase 2b trial in Q4 2024, with data expected in the first half of 2025.
• The company is developing a portfolio of oral and injectable therapies targeting multiple therapeutic areas within [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) and metabolic diseases.
Metsera’s advancements in GLP-1 agonists aim to provide more convenient, effective, and scalable weight loss treatments, addressing the evolving needs of the obesity treatment landscape.
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**Categories:** News
---
### [Revolutionary Peripulse System Approved for Nerve Regeneration](https://www.clinicaltrialvanguard.com/news/revolutionary-peripulse-system-approved-for-nerve-regeneration/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Epineuron has achieved a significant milestone with the approval of its PeriPulse™ system by [Health Canada](https://www.clinicaltrialvanguard.com/news/amo-pharma-aligns-with-fda-mhra-health-canada-on-amo-02-study-design-for-cdm1/). This innovative nerve regeneration solution represents a breakthrough in nerve care, offering patients with severe injuries and chronic conditions a promising path toward recovery.
PeriPulse™ is the first therapeutic solution specifically designed to promote nerve regeneration. Its advanced electrode and wearable device allow for quick deployment during surgery, enabling targeted electrical stimulation therapy in the recovery room. This approach significantly reduces operating time while providing optimal stimulation conditions for nerve recovery.
Supporting the approval, Epineuron’s ongoing clinical trials continue to demonstrate the effectiveness of PeriPulse™. The REGAIN™ and SELECT™ trials focus on nerve lacerations and compression injuries, respectively, expanding the clinical evidence base for the system’s benefits.
Sergio Aguirre, CEO of Epineuron indicated that this approval opens new avenues for improving the lives of patients with nerve injuries and that PeriPulse™ offers a minimally invasive and highly effective solution, revolutionizing the treatment of nerve damage.
With the regulatory approval secured, Epineuron will enhance its manufacturing, sales, and marketing capabilities to support commercialization. The company’s dedication to advancing bioelectronic solutions for nerve repair and treatment remains unwavering.
About Epineuron
Epineuron is a nerve care company specializing in bioelectronic solutions for nerve damage. Its flagship product, PeriPulse™, leverages electrical stimulation to accelerate nerve recovery and restore function. Epineuron strives to bring innovative technologies to the forefront of nerve regeneration through collaboration with researchers, clinicians, and partners.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Curebound Grants $2 Million to Cancer Research Innovations](https://www.clinicaltrialvanguard.com/news/curebound-grants-2-million-to-cancer-research-innovations/)
**Published:** September 26, 2024
**Author:** Jon Napitupulu
**Content:**
Curebound, a philanthropic organization dedicated to cancer research, has awarded eight grants totaling $2 million to innovative oncology-based projects.
These grants support pre-series A life science companies conducting groundbreaking research with commercialization potential. They provide up to $250,000 to projects that aim to develop transformative products, therapies, or services for cancer treatment. Awarded recipients include:
• Beken Bio: Liquid biopsy detection of ovarian cancer
• Pillar: Prevention and Diagnostic Tools
• Palm Therapeutics: Preclinical development of NRAS inhibitors for NRAS mutant cancers
• Pillar: Novel Approaches and New Therapeutic Platforms
• Resolute Science: Targeting macrophages to treat glioblastoma
• Pillar: Novel Approaches and New Therapeutic Platforms
• Seren Bio: Development of DMGV analogs for [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) therapy
• Pillar: Novel Approaches and New Therapeutic Platforms
Focused on reducing cancer-related health disparities, Equity Grants provide up to $250,000 to researchers investigating ways to improve access to quality care, cancer prevention, screening, and detection in underserved populations. Awarded recipients include:
• “Enhancing Lung Cancer Screening Engagement for Primary Care Patients in Low Income Communities”
• Investigators: David Strong, PhD (UC San Diego), Job Godino, PhD (Family Health Center of San Diego)
• Pillar: Cancer Equities, Prevention and Diagnostic Tool
• “Screening with Liquid Biopsies to Address Colorectal Cancer Inequities”
• Investigators: Samir Gupta, MD (UC San Diego), Job Godino, PhD (Family Health Center of San Diego)
• Pillar: Cancer Equities, Prevention
Curebound’s CEO, Anne Marbarger, emphasized the importance of these grants in accelerating cancer cures across populations. The rigorous review process ensures that projects with the potential to reach patients are prioritized.
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**Categories:** News
---
### [OHRP's 7th Exploratory Workshop: Navigating AI in Clinical Trials](https://www.clinicaltrialvanguard.com/conference-coverage/ohrps-7th-exploratory-workshop-navigating-ai-in-clinical-trials/)
**Published:** September 26, 2024
**Author:** Moe Alsumidaie
**Content:**
The Office for Human Research Protections (OHRP) recently hosted its 7th exploratory workshop, focusing on the increasingly pervasive topic of AI in clinical trials. The event brought together experts to discuss AI technologies’ ethical, legal, and practical implications in human subjects research. The workshop aimed to foster discussions on integrating ethical considerations into AI research and its applications, ensuring that human subjects’ rights and welfare remain protected.
## [](#keynote-by-jeff-smith-the-ai-lifecycle)Keynote by Jeff Smith: The AI Lifecycle
Jeff Smith, Deputy Division Director within the Certification and Testing Division at the Office of the National Coordinator for Health Information Technology (ONC), delivered the keynote address. Smith proposed that the AI lifecycle has “fractal-like properties,” making it a versatile framework for product development, institutional strategy, and public policy. Smith elaborated on ONC’s regulatory role, particularly in the pre-deployment phase of AI technologies. He mentioned that ONC regulates electronic health records (EHRs) used by 96% of hospitals and 80% of office-based physicians. This extensive reach allows ONC to influence a significant portion of the healthcare industry.
Smith discussed a recent rule finalized by ONC, establishing first-of-its-kind regulations for AI and predictive algorithms in certified health IT. These regulations require developers to provide information about how algorithms are designed, developed, tested, and evaluated, ensuring transparency and accountability. Smith also discussed the recent reorganization within ONC, which now includes responsibilities related to AI strategy and policy for the Department of Health and Human Services (HHS). This reorganization aims to create a coordinated set of activities promoting fair, appropriate, valid, effective, and safe use of AI in health. Smith highlighted the importance of a regulatory mosaic, where agencies within HHS and other governance bodies work together to address the various facets of AI deployment and use.
## [](#approaching-ai-with-ethical-conduct)Approaching AI with Ethical Conduct
Smith posed three critical questions for the research community to consider: how to prioritize areas of ethical concern at the intersection of AI and human subjects research, which priorities are more tractable and can be tackled in the near term, and what steps can be taken to establish practical guidance and policy. He emphasized the importance of prioritization, noting that when everything is a priority, nothing is a priority. Smith suggested that the research community identify which ethical concerns are most pressing and which can be addressed more efficiently within a specific timeframe, such as 12 to 18 months.
He also highlighted the need for practical guidance and policy to help practitioners navigate the ethical landscape of AI in human research. Smith pointed out that the task ahead, while challenging, might not be as daunting as translating the Belmont Report into the Common Rule. However, it would still require careful consideration and collaboration. He encouraged the research community to leverage existing policies and frameworks while also being open to necessary modifications to address the unique challenges posed by AI. Smith’s questions set the stage for a focused and actionable discussion on the ethical implications of AI in human research.
## [](#panel-discussions-diverse-perspectives-on-ai)Panel Discussions: Diverse Perspectives on AI
The first panel, moderated by Jessica Vitak, a professor at the University of Maryland, featured five speakers who comprehensively outlined AI’s role in human research. Kevin McKee, a Staff Research Scientist at Google DeepMind, aimed to demystify AI, defining it as a human-made process that makes decisions or solves problems. He highlighted the diverse forms of AI, from rule-based systems to machine learning, and discussed the limitations and biases inherent in these technologies. McKee provided examples of different AI systems, such as Eliza, a rule-based chatbot from the 1960s, and modern machine learning systems like chatbots that learn from training data. He emphasized that AI is not a monolith and that different systems carry different advantages and risks.
Craig Lipset, Co-Chair of Decentralized Trials and Research Alliance, focused on how AI is being used to improve trial access and the ethical considerations that come with it. He discussed the potential of AI to decentralize clinical trials, making them more accessible to diverse populations. Lipset highlighted examples of AI in clinical trials, such as predictive algorithms that identify suitable trial participants and tools that monitor patient adherence to treatment protocols. He emphasized the need for transparency and accountability in using AI in clinical trials, ensuring that these technologies do not exacerbate existing disparities in healthcare access.
Reid Blackman, Founder and CEO of Virtue Consultants, emphasized the importance of creating responsible and responsive research programs for AI, drawing from his experience in ethical risk consultancy. He discussed the ethical risks associated with AI, such as bias and discrimination, and the need for robust governance frameworks to mitigate these risks. Blackman provided examples of ethical challenges in AI research, such as biased training data that can lead to discriminatory outcomes. He highlighted the importance of stakeholder engagement and the need for researchers to consider the broader social implications of their work. Blackman also discussed the role of ethical guidelines and standards in promoting responsible AI in clinical trials.
Stephanie Batalis, a Research Fellow at Georgetown University, examined the intersection of AI and [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/), discussing how emerging technologies impact biomedical innovation and biosecurity. She highlighted the potential of AI to accelerate biomedical research, such as using machine learning algorithms to analyze large datasets and identify new drug targets. Batalis also discussed AI’s ethical and security implications in the life sciences, such as the potential for dual-use research that could be misused for harmful purposes. She emphasized the need for robust governance frameworks to ensure that AI is used responsibly in biomedical research.
Michael Pencina, Chief Data Scientist at Duke Health, discussed creating trustworthy health AI ecosystems, bridging data science, healthcare, and AI. He discussed the importance of transparency, accountability, and stakeholder engagement in developing and deploying AI in healthcare. Pencina provided examples of AI applications in healthcare, such as predictive algorithms that identify patients at risk of adverse outcomes and tools that support clinical decision-making. He emphasized the need for rigorous validation and evaluation of AI systems to ensure their safety and effectiveness. Pensina also discussed the role of interdisciplinary collaboration in creating trustworthy health AI ecosystems.
## [](#summary)Summary
OHRP’s 7th exploratory workshop brought together experts from various fields to discuss AI’s ethical, legal, and practical implications in human research. The event highlighted the importance of a shared understanding of AI, the need for coordinated regulatory efforts, and the critical role of ethical considerations in shaping the future of AI in clinical trials. Such discussions will protect human subjects’ rights and welfare as AI evolves. The workshop emphasized the need for ongoing dialogue and collaboration to navigate the complex landscape of AI in clinical trials.
**Categories:** Article: Conference Coverage
---
### [Innovating FDA Approval for Novel Schizophrenia Treatment](https://www.clinicaltrialvanguard.com/news/innovating-fda-approval-for-novel-schizophrenia-treatment/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb’s COBENFY has received FDA approval for treating adult [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/). As a first-of-its-kind medication, COBENFY adopts a novel pharmacological approach by targeting brain receptors M1 and M4, breaking away from the current therapeutic paradigm.
COBENFY’s approval stems from the EMERGENT clinical program, which yielded positive results. In two Phase 3 trials (EMERGENT-2 and EMERGENT-3), COBENFY significantly reduced schizophrenia symptoms. It achieved a 9.6-point and 8.4-point reduction in PANSS scores compared to placebo.
COBENFY’s safety and tolerability have been established through acute and long-term trials. After more than three decades without a new class of medication for schizophrenia, COBENFY represents a groundbreaking advancement. It offers an alternative treatment option for individuals who experience inadequate symptom control or side effects with current therapies.
Dr. Chris Boerner, CEO of Bristol Myers Squibb, emphasized the milestone of introducing a completely new pharmacological approach to schizophrenia, aiming to reshape the treatment landscape. The medication’s approval marks a significant step in the company’s re-entry into neuropsychiatry.
Schizophrenia affects approximately 2.8 million people in the United States, with symptoms typically emerging in early adulthood. Despite existing treatments, many patients experience persistent symptoms or side effects. COBENFY’s availability expands treatment options and provides hope for individuals seeking effective symptom management.
Source link:
**Categories:** News
---
### [Enterprise Therapeutics' ETD001 Receives Rare Pediatric Disease Designation for Cystic Fibrosis](https://www.clinicaltrialvanguard.com/news/enterprise-therapeutics-etd001-receives-rare-pediatric-disease-designation-for-cystic-fibrosis/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
Enterprise Therapeutics, a biotechnology company specializing in respiratory disease therapies, has earned the “rare pediatric disease designation” (RPD) for its investigational [cystic fibrosis](https://www.clinicaltrialvanguard.com/news/infexs-resp-x-shows-exacerbation-reduction-in-bronchiectasis-study/) (CF) treatment, ETD001, from the US Food and Drug Administration (FDA). This designation recognizes the severity and limited treatment options available for CF, a life-threatening genetic disorder primarily affecting children.
ETD001, a novel low-molecular-weight compound, targets the sodium channel (ENaC) in airway epithelium, increasing mucus hydration and clearance. This mechanism aims to alleviate the severe congestion and inflammation caused by failed mucociliary clearance in CF patients’ lungs.
In July 2024, Enterprise commenced a Phase 2a trial to evaluate ETD001’s safety and efficacy in approximately 10% of CF patients with the highest unmet medical needs, either ineligible for or not receiving CFTR modulator therapy.
Preclinical studies have shown that ETD001 has a long-acting effect and has been well-tolerated in healthy subjects during a Phase 1 trial. The FDA’s RPD designation highlights the potential of ETD001 to address the critical unmet medical need for effective CF treatments.
The designation provides Enterprise incentives and support from the FDA during ETD001’s development, including potential access to a Priority Review Voucher (PRV) upon marketing approval. This recognition underscores Enterprise’s commitment to advancing innovative therapies for CF patients with the greatest need.
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**Categories:** News
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### [New Hope for Hormone-Sensitive Prostate Cancer: Nubeqa® Expansion Application Submitted](https://www.clinicaltrialvanguard.com/news/new-hope-for-hormone-sensitive-prostate-cancer-nubeqa-expansion-application-submitted/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
Bayer submitted a supplemental application to the FDA for NUBEQA, an oral androgen receptor inhibitor combined with androgen deprivation therapy. This application aims to expand the use of NUBEQA in treating metastatic hormone-sensitive [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) (mHSPC) patients, irrespective of chemotherapy use.
The submission is supported by positive results from the ARANOTE trial, presented at ESMO Congress and published in The Journal of Clinical Oncology. NUBEQA, jointly developed by Bayer and Orion Corporation, is an androgen receptor inhibitor that competitively inhibits androgen binding and AR-mediated transcription.
The ARANOTE trial assessed the efficacy and safety of NUBEQA plus ADT in mHSPC patients. The primary endpoint was radiological progression-free survival, while secondary endpoints included overall survival, time to first castration-resistant event, and safety assessments.
NUBEQA is also being evaluated in the ARASTEP trial, comparing NUBEQA plus ADT to ADT alone in HSPC patients with high-risk biochemical recurrence and no evidence of metastatic disease. Additionally, the DASL-HiCaP trial investigates NUBEQA as an adjuvant treatment for localized prostate cancer with a high risk of recurrence.
NUBEQA carries a warning regarding potential ischemic heart disease, with a 3.2% occurrence rate observed in a study of nmCRPC patients. Healthcare professionals should monitor patients for cardiovascular adverse events, including ischemic heart disease.
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**Categories:** News
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### [TAGRISSO Approved for Unresectable Stage III EGFR-Mutated Lung Cancer](https://www.clinicaltrialvanguard.com/news/tagrisso-approved-for-unresectable-stage-iii-egfr-mutated-lung-cancer/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
AstraZeneca’s TAGRISSO ([osimertinib](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)) has received US approval for treating adults with unresectable, Stage III EGFR-mutated [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). The approval stems from the LAURA Phase III trial, revealing that TAGRISSO significantly extended progression-free survival (PFS) by 39.1 months compared to 5.6 months with a placebo.
The trial demonstrated an 84% reduction in disease progression or death risk with TAGRISSO. The median PFS was 39.1 months, highlighting the drug’s potential to delay disease progression for over three years. Overall survival (OS) results are still maturing, but the trial continues to assess OS as a secondary endpoint.
The approval is a major advancement for patients with Stage III, EGFR-mutated lung cancer who previously lacked targeted treatment options. Approximately 15% of NSCLC patients in the US have EGFR mutations, and nearly one in five cases of NSCLC have unresectable tumors.
The safety profile of TAGRISSO in the LAURA trial aligns with its established safety data, with no new concerns identified. TAGRISSO is already approved for first-line metastatic and early-stage EGFR-mutated NSCLC. Regulatory authorities worldwide are reviewing TAGRISSO for this indication in other countries.
The approval underscores the importance of early diagnosis and testing for lung cancer to enable timely access to targeted therapies like TAGRISSO, which can significantly improve patient outcomes.
Source link:
**Categories:** News
---
### [Viviani's GLP-1 Implant Cleared for Human Trials in Australia: A Revolutionary Approach to Fighting Obesity and Overweight](https://www.clinicaltrialvanguard.com/news/vivianis-glp-1-implant-cleared-for-human-trials-in-australia-a-revolutionary-approach-to-fighting-obesity-and-overweight/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
[Vivani](https://www.clinicaltrialvanguard.com/news/vivani-medical-unveils-groundbreaking-weight-loss-solution-for-pets-with-okv-119-a-life-changing-innovation/) Medical announces the approval of its clinical trial for a miniature GLP-1 implant, designed to offer comparable effectiveness to [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/), found in Ozempic® and Wegovy®. This implant aims to provide treatment with twice-yearly administration, a significant advantage over existing products.
The clinical trial, LIBERATE-1™, will evaluate the safety, tolerance, and pharmacological effects of the GLP-1 implant in overweight and obese individuals. Participants will receive either Vivani’s implant, weekly exenatide injections or weekly semaglutide injections for nine weeks.
Preclinical studies have shown promising weight loss and liver fat reduction results, indicating the potential of the implant to match the effectiveness of semaglutide. The unique design of the implant targets medication non-adherence, a major challenge for patients, by ensuring consistent drug delivery. This is expected to enhance treatment tolerability.
The trial is planned to commence in Australia later this year, with data anticipated in 2025. Vivani intends to explore incentives and rebates from the Australian government to cover trial expenses partially. Data from this study may contribute to regulatory submissions in other regions, including the United States.
Vivani believes its ultra-long-acting GLP-1 implant platform, NanoPortal™, has the potential to differentiate itself from current treatments, offering the benefits of twice-yearly administration and improved adherence, ultimately enhancing patient outcomes.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
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### [BioAlberta, AxialBridge Team Up to Empower Alberta Life Sciences Companies](https://www.clinicaltrialvanguard.com/news/bioalberta-axialbridge-team-up-to-empower-alberta-life-sciences-companies/)
**Published:** September 27, 2024
**Author:** Jon Napitupulu
**Content:**
BioAlberta has partnered with AxialBridge to support Alberta biotech and MedTech companies in expanding their clinical trials and R&D endeavors into the United Kingdom. This partnership aims to bridge the gap between Canadian and UK life science sectors.
AxialBridge, with its extensive experience in the UK healthcare market, will guide BioAlberta members through the regulatory and commercial pathways to enhance their UK operations. Robb Stoddard, President and CEO of BioAlberta, emphasized the unique positioning of AxialBridge due to its relationships and expertise in the UK.
The partnership will facilitate a bilateral exchange of opportunities and expertise. AxialBridge has successfully supported Canadian and UK companies in clinical trials, funding, and growth initiatives. This experience will be leveraged to identify and enable opportunities for Alberta businesses.
Jaspreet Grewal, CEO and Co-Founder of AxialBridge expressed pride in the partnership and its potential to foster innovation and collaboration. The partnership strengthens the long-standing relationship between Canada and the United Kingdom in [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/), creating a valuable ecosystem for biotech and MedTech growth.
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**Categories:** News
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### [Precision's Exciting HBV Trial: A Breakthrough for Hepatitis B Patients](https://www.clinicaltrialvanguard.com/news/precisions-exciting-hbv-trial-a-breakthrough-for-hepatitis-b-patients/)
**Published:** October 1, 2024
**Author:** Jon Napitupulu
**Content:**
[Precision BioSciences](https://www.clinicaltrialvanguard.com/news/precision-biosciences-programs-make-a-stunning-return-collaboration-with-prevail-therapeutics-concludes/) has submitted Clinical Trial Applications (CTAs) to initiate a Phase 1 study for its in vivo gene editing therapy, PBGENE-HBV. This therapy aims to potentially cure chronic hepatitis B virus (HBV) by eliminating cccDNA, the primary source of viral replication. Additionally, it inactivates integrated HBV DNA in hepatocytes.
PBGENE-HBV is unique in its approach, directly targeting cccDNA for elimination. It has demonstrated efficacy in preclinical models of hepatitis B, including in non-human primates, considered the most reliable model for human safety prediction.
The Phase 1 trial will assess the safety and efficacy of PBGENE-HBV in humans. It will enroll individuals with chronic hepatitis B and evaluate the therapy’s ability to achieve a functional cure. Results of the trial are expected in 2025.
The CTA submissions highlight Precision BioSciences’ commitment to developing a curative therapy for chronic hepatitis B, a global public health concern affecting an estimated 300 million people. The company has assembled a world-class scientific advisory board and bolstered its clinical team expertise to support the development and execution of the global Phase 1 trial.
Precision BioSciences plans to submit additional regulatory applications globally for PBGENE-HBV as part of its Phase 1 regulatory strategy. Detailed safety data of the clinical candidate and Phase 1 trial plans will be presented in November.
Source link:
**Categories:** News
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### [InCarda Phase 3 Trial Prematurely Terminated](https://www.clinicaltrialvanguard.com/news/incarda-phase-3-trial-prematurely-terminated/)
**Published:** October 1, 2024
**Author:** Jon Napitupulu
**Content:**
InCarda Therapeutics has demonstrated promising results in developing FlecIH-103, an inhaled solution for converting paroxysmal atrial fibrillation (PAF) to sinus rhythm.
Though prematurely terminated, the Phase 3 [RESTORE](https://www.clinicaltrialvanguard.com/news/nervgen-prepares-restore-phase-3-trial-in-chronic-tetraplegia-with-nvg-291/)-1 trial showed that inhaled flecainide achieved a statistically significant cardioversion rate of 31% in PAF patients compared to placebo. The median time to restore normal sinus rhythm was approximately 13 minutes.
Despite the low enrollment, the study revealed significant pharmacoeconomic benefits associated with FlecIH-103. Patients who converted to sinus rhythm were more likely to be discharged within two hours and experienced better symptom resolution within 90 minutes. The need for electrical cardioversion was also reduced in the treatment arm.
The company is transitioning to a new drug-delivery platform supported by in vitro and Phase 1 clinical data. Phase 1 results warrant the continuation of the study, potentially advancing FlecIH-103 into registrational trials.
The conclusion of the RESTORE-1 trial provides evidence of the potential of inhaled flecainide as a safe and effective therapy for PAF patients, offering rapid restoration of sinus rhythm and reducing the need for invasive procedures.
Source link:
**Categories:** News
---
### [Biosimilar Otuli™ Receives FDA Approval: Encouraging News for Arthritis Patients](https://www.clinicaltrialvanguard.com/news/biosimilar-otuli-receives-fda-approval-encouraging-news-for-arthritis-patients/)
**Published:** October 1, 2024
**Author:** Jon Napitupulu
**Content:**
Fresenius Kabi, a subsidiary of Fresenius, and Formycon AG, a leading biosimilars developer, have received FDA approval for Otulfi™ ([ustekinumab](https://www.clinicaltrialvanguard.com/news/samsung-bioepis-launches-stelara-biosimilar-pyzchiva-in-us/)-aauz). This biosimilar targets [Crohn’s disease](https://www.clinicaltrialvanguard.com/news/eli-lillys-omvoh-gets-fda-approval-for-crohns-disease-but-faces-challenges/)[Crohn’s](https://www.clinicaltrialvanguard.com/news/agomabs-crohns-drug-shows-promising-phase-2a-results/) disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis.
Otulfi™ was developed through a comprehensive evaluation of data, demonstrating its biosimilarity to Stelara® in efficacy, safety, and pharmacokinetics. It offers a broader treatment solution for healthcare professionals and patients requiring ustekinumab therapy.
Fresenius Kabi’s strong commitment to expanding its biopharma portfolio has led to Otulfi™’s approval, the company’s fourth biosimilar in the U.S. market. The wider biopharma portfolio aligns with their Vision 2026 strategy to establish a significant presence in the biopharma industry and provide crucial treatment options globally.
Formycon AG holds a global commercialization partnership with Fresenius Kabi for Otulfi™. Ustekinumab, the active ingredient in Otulfi™, is a monoclonal antibody that targets interleukin-12 and interleukin-23, key inflammatory cytokines.
Fresenius Kabi’s biosimilar pipeline continues to grow, with autoimmune and oncology biosimilars in various stages of development. This reinforces their dedication to delivering accessible and affordable healthcare solutions.
Source link: [http://www.businesswire.com/news/home/20240930060447/en/Fresenius-Kabi-and-Formycon-Receive-U.S.-FDA-Approval-for-Biosimilar-Otulfi%E2%84%A2\*-ustekinumab-aauz](http://www.businesswire.com/news/home/20240930060447/en/Fresenius-Kabi-and-Formycon-Receive-U.S.-FDA-Approval-for-Biosimilar-Otulfi%E2%84%A2*-ustekinumab-aauz)
**Categories:** News
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### [ENHERTU® Granted Priority Review in the U.S. for Patients Battling Metastatic Breast Cancer](https://www.clinicaltrialvanguard.com/news/enhertu-granted-priority-review-in-the-u-s-for-patients-battling-metastatic-breast-cancer/)
**Published:** October 2, 2024
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)‘s application for ENHERTU has been accepted for Priority Review by the US Food and Drug Administration (FDA). This treatment targets adult patients with HER2 low or ultralow unresectable or metastatic [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) who have undergone prior endocrine therapy.
ENHERTU, an antibody-drug conjugate, was jointly developed by Daiichi Sankyo and AstraZeneca. The FDA grants Priority Review status to promising therapies with the potential for significant improvements in treatment outcomes. The Prescription Drug User Fee Act (PDUFA) date for the FDA’s decision is February 1, 2025.
The application is supported by data from the DESTINY-Breast06 clinical trial, which showed that ENHERTU reduced the risk of disease progression or death by 37% in the overall population and improved median progression-free survival from 8.1 months to 13.2 months compared to chemotherapy. Results were consistent across HER2 low and ultralow subgroups.
The safety profile of ENHERTU in DESTINY-Breast06 was generally consistent with previous clinical trials. The most common grade 3 or higher side effects included neutropenia, leukopenia, and anemia. Interstitial lung disease (ILD) or pneumonitis occurred in 11.3% of patients, with most cases being low-grade. There were no grade 4 or 5 ILD events.
This application underscores the potential of ENHERTU in addressing the need for improved treatment options for patients with HR-positive, HER2 low, or ultralow breast cancer, which represents approximately 85-90% of breast cancer cases.
Source link: http://www.businesswire.com/news/home/20240930807595/en/ENHERTU%C2%AE-Granted-Priority-Review-in-the-U.S.-for-Patients-with-HER2-Low-or-HER2-Ultralow-Metastatic-Breast-Cancer-Who-Have-Received-at-Least-One-Line-of-Endocrine-Therapy
**Categories:** News
---
### [Merck Acquires Novel B-Cell Depletion Therapy From Curon](https://www.clinicaltrialvanguard.com/news/merck-acquires-novel-b-cell-depletion-therapy-from-curon/)
**Published:** October 2, 2024
**Author:** Jon Napitupulu
**Content:**
Merck has acquired [CN201](https://www.clinicaltrialvanguard.com/news/merck-to-secure-novel-b-cell-therapy-cn201-in-deal-with-curon-bio/), a bispecific antibody, from Curon Biopharmaceutical. This acquisition strengthens Merck’s pipeline for treating B-cell malignancies and autoimmune diseases.
CN201 targets and eliminates B cells, offering potential applications in various B-cell-related conditions. Preliminary clinical trials indicate that CN201 is well-tolerated and effectively reduces B-cell populations in patients with relapsed or refractory B-cell malignancies.
Current clinical studies evaluate CN201’s efficacy in treating non-Hodgkin’s [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) and B-cell acute lymphocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) patients.
Under the acquisition agreement, Merck has acquired global rights to CN201. The transaction is recorded as an asset acquisition, resulting in a $750 million pre-tax charge (approximately $0.28 per share), impacting third-quarter non-GAAP results. Merck’s financial outlook for the year will be updated with the release of third-quarter 2024 results on October 31.
CN201 represents a significant addition to Merck’s research and development portfolio. Its potential to effectively eliminate B cells holds promise for treating various B-cell-associated diseases in the future.
Source link:
**Categories:** News
---
### [Maze Therapeutics Pioneers Revolutionary Kidney Disease Treatment](https://www.clinicaltrialvanguard.com/news/maze-therapeutics-pioneers-revolutionary-kidney-disease-treatment/)
**Published:** October 2, 2024
**Author:** Jon Napitupulu
**Content:**
Maze Therapeutics, a biopharmaceutical company, has commenced its Phase 1 clinical trial for [MZE782](https://www.clinicaltrialvanguard.com/news/mazes-mze782-shows-positive-results-for-pku-ckd-in-trial/), an oral small molecule therapy. MZE782 targets SLC6A19, a solute transporter, potentially benefiting approximately 5 million chronic kidney disease (CKD) patients in the United States who have limited responses to existing treatments.
Harold Bernstein, president and chief medical officer of Maze, highlighted that CKD impacts numerous individuals, and current therapies primarily aim to slow disease progression rather than addressing its underlying genetic causes. MZE782, developed using Maze’s Compass platform, seeks to replicate the protective effects of SLC6A19 variants.
The Phase 1 trial assesses safety, tolerability, pharmacokinetics, and pharmacodynamics. Non-invasive biomarkers will be utilized to optimize future clinical trial designs for patients. Initial findings, including potential proof of mechanism using these biomarkers, are anticipated in the second half of 2025.
Beyond its standalone potential, MZE782 may offer additional benefits when combined with standard-of-care treatments. It could complement existing regimens or be an alternative for patients who do not respond adequately to current therapies.
Maze Therapeutics harnesses human genetics to develop precision medicines for common renal, cardiovascular, and metabolic diseases. The company employs its Compass platform to uncover genetic variants associated with diseases and their underlying biological pathways in specific patient populations, driving its pipeline advancements.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [Advanced Prostate Cancer Breakthrough: Posluma Unlocks Hope and Extends Lives](https://www.clinicaltrialvanguard.com/news/advanced-prostate-cancer-breakthrough-posluma-unlocks-hope-and-extends-lives/)
**Published:** October 2, 2024
**Author:** Jon Napitupulu
**Content:**
Blue Earth Diagnostics, a renowned company in PET radiopharmaceutical development, announced the results of a post-hoc analysis from the Phase 3 SPOTLIGHT trial. The study evaluated the use of POSLUMA injection for detecting [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) recurrence in patients who had undergone radical prostatectomy and had low PSA levels.
The analysis revealed high detection rates for POSLUMA in the pelvic region, particularly in patients with PSA levels below 1 ng/mL and 0.5 ng/mL. This suggests that POSLUMA is effective in detecting recurrence in its early stages.
POSLUMA, approved by the FDA, aids in the visualization of PSMA-positive prostate cancer lesions. It provides essential information to guide treatment decisions for men with suspected recurrence who may benefit from curative salvage therapy.
The high affinity of POSLUMA for PSMA allows for accurate detection despite potential interference from activity in the urinary bladder and ureters. Unlike other PSMA PET radiopharmaceuticals, POSLUMA’s image assessment is not significantly impacted by urinary activity in the majority of patients, ensuring accurate interpretation.
POSLUMA’s enhanced detection capabilities make it a valuable tool for managing prostate cancer. Detecting recurrence early and providing reliable information helps guide treatment and improve patient outcomes.
Source link:
**Categories:** News
---
### [Owkin and AstraZeneca Team Up to Triumph Over Breast Cancer](https://www.clinicaltrialvanguard.com/news/owkin-and-astrazeneca-team-up-to-triumph-over-breast-cancer/)
**Published:** October 3, 2024
**Author:** Jon Napitupulu
**Content:**
Owkin and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) are collaborating on an AI-powered tool that will pre-screen for harmful mutations in [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) genes (gBRCAm) using digital pathology slides. The tool aims to enhance accessibility and streamline testing for these mutations.
BRCA testing identifies mutations in BRCA1 and BRCA2 genes and is crucial in determining cancer risk and guiding treatment decisions. However, testing is not always consistently provided to eligible individuals, leading to missed opportunities for personalized care.
The gBRCA pre-screening solution could significantly increase the number of patients identified with gBRCAm by 2030. It can potentially improve efficiency by rapidly identifying high-risk patients using existing data from pathology slides. This could greatly accelerate the testing process and reduce the time from consultation to results.
By offering faster and more accessible testing, the gBRCA pre-screening solution will contribute to advancing precision medicine. It will enable the identification of individuals at risk of harboring BRCA mutations, allowing for tailored treatment plans and improved patient outcomes.
By streamlining the process and making testing more accessible, the gBRCA pre-screening solution aims to reduce disparities in care. It will ensure that more patients can benefit from personalized treatments based on their genetic profile.
The partnership between Owkin and AstraZeneca demonstrates a commitment to advancing precision medicine for breast cancer patients. The AI-powered tool has the potential to revolutionize BRCA testing, improve patient care, and ultimately save lives.
Source link:
**Categories:** News
---
### [How Causal Machine Learning is Transforming Clinical Trials](https://www.clinicaltrialvanguard.com/executiveinterviews/how-causal-machine-learning-is-transforming-clinical-trials/)
**Published:** October 3, 2024
**Author:** Moe Alsumidaie
**Content:**
Welcome to an insightful discussion on the future of clinical trials and the role of causal machine learning in drug development. In this interview at [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) 2024, we speak with Raviv Pryluk, CEO and Co-founder of PhaseV, to explore how this technology is reshaping the landscape, addressing challenges, and enhancing the precision of medical treatments.
## [](#moe-alsumidaie-raviv-how-do-you-see-causal-machine-learning-reshaping-drug-development-especially-for-niche-therapies-or-rare-diseases-over-the-next-decade)Moe Alsumidaie: Raviv, how do you see causal machine learning reshaping drug development, especially for niche therapies or rare diseases, over the next decade?
**Raviv Pryluk:** Before discussing its impact, let’s address the challenges. Different patients respond differently to treatments. You can release a drug to the market, and some patients will respond while others will not. The same is true in clinical trials. Many trials fail, not because the drug isn’t working, but because it’s only effective for a subset of patients. Identifying who will respond and who won’t is highly beneficial,
especially in the precision medicine era. Causal machine learning, which intersects causal inference and machine learning, can uniquely identify responders and the sources of heterogeneity. This is crucial in oncology, immunology, and neurodegenerative diseases, as well as in rare diseases where patient recruitment is challenging. For example, in rare oncology indications, where treatments can be particular to genetic markers, causal machine learning can help pinpoint which patients will benefit from a new therapy, thereby increasing the trial’s success rate and speeding up the drug’s time to market.
## [](#moe-alsumidaie-how-do-you-plan-to-scale-phasevs-machine-learning-platform-to-smaller-biotechs-that-lack-the-infrastructure-of-larger-companies)
Moe Alsumidaie: How do you plan to scale PhaseV’s machine learning platform to smaller biotechs that lack the infrastructure of larger companies?
**Raviv Pryluk:** We provide either tech-enabled services or SaaS licensing of our software. Larger companies, like [big pharma](https://www.clinicaltrialvanguard.com/analysis/the-future-of-dcts-is-bright-according-to-big-pharma-and-fda/), often prefer to license and use the software themselves with our support. We use the technology for to provide services, reanalyzing data retrospectively and supporting the design and execution of adaptive, dynamic clinical trials. This approach makes our technology accessible across many therapeutic areas, regardless of company size. For instance, a small biotech working on a rare disease might not have the resources to build a robust data infrastructure. By offering our platform as a service, we enable them to leverage advanced machine learning capabilities without significant upfront investment. This democratization of data and technology ensures that even smaller players can compete on a level playing field with larger companies.
## [](#moe-alsumidaie-how-do-you-fully-address-concerns-from-stakeholders-who-are-hesitant-to-embrace-machine-learning-and-ai-in-this-highly-regulated-environment)
Moe Alsumidaie: How do you fully address concerns from stakeholders who are hesitant to embrace machine learning and AI in this highly regulated environment?
**Raviv Pryluk:** The hesitation is understandable, especially when dealing with patients and complex experiments. We spend a lot of time on validation. Our algorithms are designed with validation in mind, using train-test-validation frameworks, external datasets, and sophisticated statistical tests to ensure validity, accuracy and robustness. We have a proprietary data lake for additional validation and use bootstrapping to test many scenarios in-silico. This rigorous validation process gives us confidence in our findings, and we only recommend following AI insights when the confidence level is high. For example, we might validate our machine learning models against multiple clinical trial datasets to ensure that the patterns we identify are consistent and reliable. This thorough validation helps build trust among stakeholders, including regulators, who must be assured that the AI-driven insights are robust and actionable.
## [](#moe-alsumidaie-what-are-the-most-significant-barriers-to-adopting-adaptive-trial-designs-and-how-does-phasev-lead-this-shift-especially-in-big-pharma)
Moe Alsumidaie: What are the most significant barriers to adopting adaptive trial designs, and how does PhaseV lead this shift, especially in big pharma?
**Raviv Pryluk:** There are many barriers, including complexity, less intuitive designs, and operational challenges. We’ve developed an intuitive software platform that designs trials, unravels insights, and explains adaptations. This helps stakeholders, from biostatisticians to commercial teams, understand the implications. The same platform supports design and execution, making the process more reliable and streamlined. Clear communication with regulators about the logic behind adaptations also increases confidence and acceptance. For example, our platform can provide a detailed rationale for why a particular adaptation is being made in a trial, backed by data and statistical analysis. This transparency helps all stakeholders, including regulatory bodies, understand and trust the adaptive design, facilitating its adoption.
## [](#moe-alsumidaie-what-steps-is-phasev-taking-to-establish-itself-as-a-thought-leader-in-clinical-trial-optimization-and-influence-regulatory-frameworks)
Moe Alsumidaie: What steps is PhaseV taking to establish itself as a thought leader in clinical trial optimization and influence regulatory frameworks?
**Raviv Pryluk:** We are humble and focused on bringing value to sponsors and regulators. We build valuable technology grounded in solid science, publish papers, and patents. Engaging with the community through conferences, panels, and meetings with regulators helps us provide insights and get feedback. We apply our technology in real-world scenarios, sharing successes and failures transparently. This collective approach helps us become an essential player in the space. By presenting our findings and methodologies at industry conferences, we showcase our capabilities and contribute to the broader dialogue on clinical trial optimization. This helps us influence regulatory frameworks by demonstrating our technology’s practical benefits and reliability.
## [](#moe-alsumidaie-as-ai-becomes-more-integrated-into-clinical-trials-do-you-foresee-it-aiding-rather-than-replacing-people-what-do-you-think-is-the-future-here)
Moe Alsumidaie: As AI becomes more integrated into clinical trials, do you foresee it aiding rather than replacing people? What do you think is the future here?
**Raviv Pryluk:** AI in clinical development is about augmenting and empowering experts, not replacing them. Our platform makes biostatisticians (clin-ops/clin-dev experts) more productive, accurate, and faster. Tasks that took weeks can now be done with a click of a button. We aim to increase the success rate of clinical development, making drugs more available to patients. The future is about increasing productivity and throughput in clinical development, not replacing anyone.
**Categories:** Article: Conference Coverage, Article: Executive Interviews
---
### [Calquence (Acalabrutinib) Receives Priority Review in the US for Untreated Mantle Cell Lymphoma Patients](https://www.clinicaltrialvanguard.com/news/calquence-acalabrutinib-receives-priority-review-in-the-us-for-untreated-mantle-cell-lymphoma-patients/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
[AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)‘s CALQUENCE ([acalabrutinib](https://www.clinicaltrialvanguard.com/news/ascentage-pharma-presents-promising-cancer-research-at-aacr/)) has received Priority Review designation from the FDA for treating untreated adult patients with [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (MCL). This review status is reserved for drugs that offer substantial improvements over existing therapies.
MCL is an aggressive form of non-Hodgkin lymphoma that affects over 27,500 people worldwide. It is often diagnosed in advanced stages and is typically incurable.
Results from the ECHO Phase III trial showed that CALQUENCE combined with standard-of-care chemoimmunotherapy significantly reduced the risk of disease progression or death by 27% compared to chemoimmunotherapy alone. The addition of CALQUENCE also extended the median progression-free survival (mPFS) by almost 1.5 years, from 49.6 months to 66.4 months.
Overall survival (OS) showed a promising trend in favor of the CALQUENCE combination, although the data is still maturing. The trial will continue to monitor OS as a key secondary endpoint.
The FDA’s review of CALQUENCE is under Project Orbis, a program that facilitates the concurrent submission and review of oncology drugs among participating international partners. The Prescription Drug User Fee Act date for the FDA’s decision is anticipated in the first quarter of 2025.
AstraZeneca is committed to working closely with the FDA to make CALQUENCE available to patients with MCL as soon as possible. This potential new treatment offers significant promise for improving outcomes in this devastating disease.
Source link:
**Categories:** News
---
### [DeepCure Presents Breakthrough Data: DC-9476 Triumphs over Etanercept in Rheumatoid Arthritis Battle](https://www.clinicaltrialvanguard.com/news/deepcure-presents-breakthrough-data-dc-9476-triumphs-over-etanercept-in-rheumatoid-arthritis-battle/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
DeepCure, a biotechnology company, has unveiled promising data on its selective BRD4 (BD2) inhibitor, [DC-9476](https://www.clinicaltrialvanguard.com/news/deepcure-unveiling-the-potential-of-bd2-inhibitor-dc-9476-in-macrophage-activation-syndrome/). Preclinical studies indicate its superiority to existing anti-TNF-α treatments in a [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (RA) mouse model.
Activated macrophages play a crucial role in RA. They release inflammatory cytokines such as TNF-α, IL-1β, and IL-6, leading to joint damage and symptoms.
In vitro assays demonstrated that DC-9476 effectively reduced IL-6 production, surpassing the potency of tofacitinib, an approved Jak-2 inhibitor for RA treatment.
Animal model studies further supported DC-9476’s efficacy. In a lipopolysaccharide-induced inflammation model, it significantly lowered serum IL-6 and TNF-α levels. In the CAIA model, which mimics macrophage-driven RA, DC-9476 exhibited remarkable potency, reducing clinical disease scores by over 80%. This outperformed etanercept, an established anti-TNF-α antibody, which achieved only a 47% reduction.
Crucially, DC-9476 exhibited no toxicity. Its combination with etanercept led to enhanced improvements compared to either treatment alone.
These findings highlight DC-9476’s potential as a novel oral monotherapy or combination therapy for RA. Reducing inflammatory cytokines also suggests potential utility as target engagement biomarkers in future clinical trials.
Source link:
**Categories:** News
---
### [Allele-Selective Therapies for Rare Genetic Disorders: Regulatory Challenges and Solutions](https://www.clinicaltrialvanguard.com/news/allele-selective-therapies-for-rare-genetic-disorders-regulatory-challenges-and-solutions/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
Recent advances in genetic sequencing have identified an increasing number of rare heterozygous toxic gain-of-function (TGOF) mutations. These mutations pose significant challenges in developing therapies due to the need for allele-selective approaches that target only the mutant allele.
Antisense [oligonucleotide](https://www.clinicaltrialvanguard.com/news/camp4s-pioneering-syngap-1-drug-enters-toxicology-studies/) (ASO) technology offers a promising solution for allele-selective targeting. However, clinical trials for TGOF patients are often hindered by the difficulty in finding sufficient patients with the same gene variant.
In a commentary published in Nucleic Acid Therapeutics, Dr. Stanley T. Crooke proposes a cost-effective regulatory strategy to address this challenge. The approach involves conducting a single Phase 3 study in which patients are stratified by disease severity and variant. Each variant would receive a specific allele-selective ASO.
After the study, data from all variant-selective treatments would be aggregated and compared to placebo. This would allow for evaluating the safety and efficacy of multiple ASOs in a single trial.
Dr. Crooke emphasizes the potential for this strategy to make the development of allele-selective ASOs more feasible, enabling the treatment of a broader range of patients with TGOF mutations. As more patients undergo whole genome sequencing, the prevalence of TGOF heterozygous mutations is expected to rise, highlighting the need for innovative regulatory approaches to facilitate the development of targeted therapies.
Source link:
**Categories:** News
---
### [Humanetics Corp. Wows at 2024 DoD Scientific Meeting: A Testimony to Enduring Innovation](https://www.clinicaltrialvanguard.com/news/humanetics-corp-wows-at-2024-dod-scientific-meeting-a-testimony-to-enduring-innovation/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
Humanetics Corporation presented updates on the progress of BIO 300, an orally administered drug designed to prevent radiation toxicity in military personnel. The presentation was made at the 2024 Military Health System Research Symposium.
BIO 300 was initially developed as a radioprotectant by the Armed Forces Radiobiology Research Institute. Humanetics holds an exclusive license for its advanced development and commercialization. Ongoing collaborations with the Department of Defense aim to create an oral drug that warfighters can take to shield themselves from the effects of radiation exposure in radiological or nuclear events.
Research findings presented at the symposium demonstrate that BIO 300 is among the most advanced medical countermeasures currently under development for acute radiation syndrome (ARS). The development of such countermeasures is crucial given the elevated threat of nuclear or radiological occurrences in the current geopolitical climate.
Beyond military applications, BIO 300 is being investigated in phase 2 clinical trials for its potential to prevent tissue damage caused by cancer radiotherapy and reduce inflammatory lung injury in conditions like [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) and Idiopathic Pulmonary Fibrosis.
The research has received funding from the US Department of Defense’s Congressionally Directed Medical Research Program and the Joint Program Executive Office for Chemical, Biological, Radiological, and Nuclear Defense. The views presented in the research do not necessarily represent the official policies or positions of the Department of Defense or the US Government.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [U.S. FDA Approves Opdivo® for Resectable Non-Small Cell Lung Cancer](https://www.clinicaltrialvanguard.com/news/u-s-fda-approves-opdivo-for-resectable-non-small-cell-lung-cancer/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
The U.S. Food and Drug Administration (FDA) has approved Opdivo (nivolumab) for treating adults with resectable [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). The regimen combines neoadjuvant therapy with platinum-doublet chemotherapy followed by adjuvant Opdivo treatment after surgery.
The approval stems from the [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/)-77T trial, where the Opdivo-based regimen significantly extended event-free survival compared to chemotherapy and placebo. The trial also demonstrated a high pathologic complete response rate.
Opdivo is the only PD-1 inhibitor approved for resectable NSCLC in both a neoadjuvant-only setting and as part of a perioperative treatment regimen. This signifies a milestone in Bristol Myers Squibb’s thoracic portfolio and reinforces its commitment to advancing treatments for patients with early-stage disease.
The CheckMate-77T trial compared the perioperative regimen (Opdivo with neoadjuvant chemotherapy followed by adjuvant Opdivo) to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo. The Opdivo arm demonstrated a 42% reduction in disease recurrence risk, and 18-month event-free survival rates were significantly higher in the Opdivo arm than in the chemotherapy and placebo arm. Additionally, 25% of patients in the Opdivo arm achieved a complete pathologic response.
The FDA approval of Opdivo for resectable NSCLC provides a valuable treatment option for patients, offering improved outcomes and reducing the risk of recurrence post-surgery.
Source link:
**Categories:** News
---
### [ACTICOR Biotech Secures Receivership Extension for Glenzocimab Development](https://www.clinicaltrialvanguard.com/news/acticor-biotech-secures-receivership-extension-for-glenzocimab-development/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
The Paris Commercial Court has granted an extension of receivership proceedings for [ACTICOR Biotech](https://www.clinicaltrialvanguard.com/news/acticor-biotech-disclosure-of-voting-rights-shares-may-2024/), a biopharmaceutical company focused on cardiovascular emergencies. The receivership aims to facilitate the company’s efforts to secure financing, seek partners, and continue the development of its drug, [glenzocimab](https://www.clinicaltrialvanguard.com/news/acticor-biotech-liquidation-proceedings-announced/). This extension provides ACTICOR with financial support to operate until January 2025.
Glenzocimab is an innovative antibody fragment that targets the GPVI platelet receptor. It is being evaluated for the treatment of acute ischemic stroke, myocardial infarction, and other thrombotic diseases. In clinical trials, glenzocimab has shown promising results in reducing mortality in intracerebral hemorrhage patients. Phase II trials are currently ongoing to assess its efficacy in reducing myocardial infarction size.
ACTICOR Biotech was founded in 2013 based on the research of INSERM, a French biomedical research institute. The company has been listed on Euronext Growth Paris since November 2021. Its investors include Mediolanum farmaceutici, Karista, and Go Capital.
The company holds three patent families covering the use of glenzocimab in thrombotic diseases. The first family’s patent expires in 2036. Additionally, ACTICOR has the rights to develop a biomarker for stroke patients.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [Summit Therapeutics: Remarkable Results in Phase III HARMONI Trial for Lung Cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-remarkable-results-in-phase-iii-harmoni-trial-for-lung-cancer/)
**Published:** October 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Summit Therapeutics](https://www.clinicaltrialvanguard.com/news/summit-therapeuticss-remarkable-cancer-breakthrough-new-treatment-reduces-disease-progression-by-49/) has completed enrollment in the multi-regional Phase III HARMONi trial, evaluating ivonescimab in combination with chemotherapy for patients with EGFR-mutated, locally advanced, or metastatic [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). This patient population has historically shown poor response to PD-1 antibodies.
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ivonescimab in combination with chemotherapy for treating locally advanced or metastatic NSCLC patients with EGFR mutations who have progressed on EGFR tyrosine kinase inhibitors (TKIs).
Fast Track designation aims to accelerate the development and review of drugs that address serious medical needs. This designation allows for more frequent interactions with the FDA, written communication, and a rolling review process, facilitating earlier submission and review of the Biologic License Application (BLA).
The optimistic belief in ivonescimab’s potential has contributed to the successful enrollment completion of the HARMONi trial. Summit’s goal is to bring this drug to patients to enhance the quality of life for those with severe unmet medical needs.
The HARMONi trial’s topline data is anticipated in mid-2025. The FDA’s Fast Track designation provides an expedited review process, potentially leading to earlier approval and access to ivonescimab for patients.
Source link:
**Categories:** News
---
### [New HPV Vaccine Trial Gives Hope to Cancer Patients](https://www.clinicaltrialvanguard.com/news/new-hpv-vaccine-trial-gives-hope-to-cancer-patients/)
**Published:** October 7, 2024
**Author:** Jon Napitupulu
**Content:**
TheraVectys, a biotechnology company specializing in lentiviral vector-based vaccines and immunotherapies, has begun enrolling patients in a Phase I/IIa clinical trial to evaluate the efficacy and safety of Lenti-HPV-07, a therapeutic vaccine targeting human papillomavirus (HPV)-induced cancers.
The trial employs a dose-escalation protocol and will involve 36 patients with HPV-16 or HPV-18-induced oropharyngeal or cervical cancers. Group A will include patients with recurrent/metastatic cancers, while Group B will consist of newly diagnosed, treatment-naïve patients.
Lenti-HPV-07 is based on the lentiviral vector technology developed by Pasteur-TheraVectys Joint Laboratory. Preclinical studies have demonstrated its ability to induce a robust cellular immune response against [HPV16](https://www.clinicaltrialvanguard.com/news/pds-biotech-amends-phase-3-trial-for-accelerated-approval/) and HPV18 antigens.
The Phase I/IIa trial will assess the safety and immunogenicity of Lenti-HPV-07, as well as its preliminary efficacy through the Objective Response Rate. Patients will be followed clinically and immunologically for one year.
The trial is divided into two parts: dose escalation and dose expansion. In the dose escalation portion, participants will receive increasing doses of Lenti-HPV-07, with safety monitoring preceding each dose increase. Upon reaching a satisfactory safety profile, a dose-expansion portion will treat 18 additional patients at the Optimal Biological Dose.
TheraVectys has previously conducted a Phase I clinical trial with a therapeutic HIV-1 vaccine using an integrative lentiviral vector, demonstrating no significant side effects or genotoxicity. The current Lenti-HPV-07 trial employs a non-integrative lentiviral vector, further enhancing safety.
The initiation of this trial marks a significant step in the development of Lenti-HPV-07 as a potential therapeutic option for HPV-induced cancers. The trial data is expected to provide insights into its safety, immunogenicity, and efficacy in this patient population.
Source link: [http://www.businesswire.com/news/home/20241003957830/en/Human-Papillomavirus-HPV–induced-Cancers-First-Patient-Enrolled-in-Phase-IIIa-Clinical-Trial-for-Lenti-HPV-07-the-TheraVectys%E2%80%99-Therapeutic-Vaccine-Candidate-Against-Oropharyngeal-and-Cervical-Cancers](http://www.businesswire.com/news/home/20241003957830/en/Human-Papillomavirus-HPV--induced-Cancers-First-Patient-Enrolled-in-Phase-IIIa-Clinical-Trial-for-Lenti-HPV-07-the-TheraVectys%E2%80%99-Therapeutic-Vaccine-Candidate-Against-Oropharyngeal-and-Cervical-Cancers)
**Categories:** News
**Tags:** eClinical Tech News
---
### [Airsupra Reduces Severe Asthma Attacks: A Game-Changer for Patients](https://www.clinicaltrialvanguard.com/news/airsupra-reduces-severe-asthma-attacks-a-game-changer-for-patients/)
**Published:** October 8, 2024
**Author:** Jon Napitupulu
**Content:**
A clinical trial (BATURA) has shown the efficacy of AstraZenaca’s AIRSUPRA (albuterol/budesonide) in reducing the risk of severe [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/) exacerbations. AIRSUPRA, an inhaled anti-inflammatory rescue medication, was tested against albuterol alone in patients with intermittent or mild persistent asthma, including those taking maintenance therapy.
The trial met its primary endpoint, demonstrating a statistically significant reduction in the risk of severe exacerbations when AIRSUPRA was used as a rescue medication. The medication was effective in reducing the need for systemic corticosteroids.
The safety profile of AIRSUPRA was consistent with its established profile, and no new safety concerns were reported. The efficacy and safety data from the BATURA trial will be presented at an upcoming medical conference.
AIRSUPRA is the first anti-inflammatory rescue medication approved for treating asthma in adults in the US. It provides a dual-action approach by treating both and inflammation, reducing the risk of severe exacerbations and the need for additional treatments.
AIRSUPRA is currently being studied in adolescents with asthma and patients in China. Its development is a collaboration between [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) and Avillion.
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**Categories:** News
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### [Verastem Presents Positive Updated Data for Atutometinib](https://www.clinicaltrialvanguard.com/news/verastem-presents-positive-updated-data-for-atutometinib/)
**Published:** October 17, 2024
**Author:** Jon Napitupulu
**Content:**
[Verastem Oncology](https://www.clinicaltrialvanguard.com/news/verastem-oncology-avatometinib-defactinib-nda-for-low-grade-serous-ovarian-cancer-gets-priority-review/), a company dedicated to developing cancer treatments, has released promising data from its Phase 2 RAMP 201 clinical trial. This trial examines the effectiveness of combining avutometinib (a RAF/MEK clamp) and defactinib (a FAK inhibitor) in patients with recurrent low-grade serous [ovarian cancer](https://www.clinicaltrialvanguard.com/news/imunons-imnn-001-shows-lower-residual-disease-in-phase-2-ovarian-cancer-study/) (LGSOC).
The trial demonstrated a 31% overall response rate in patients with measurable disease, with a median duration of response of 31.1 months. Notably, patients with the KRAS mutant gene exhibited a higher response rate of 44%, while those with the KRAS wild-type gene had a 17% response rate. Median progression-free survival for all patients was 12.9 months, increasing to 22 months for those with the KRAS mutant gene.
The combination treatment was generally well-tolerated, with the most frequent side effects being nausea, diarrhea, and elevated blood creatine phosphokinase levels. Importantly, no new safety concerns were identified.
Based on these positive results, Verastem Oncology plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in October 2024 for the treatment of adult patients with recurrent KRAS mutant LGSOC. The company is seeking Accelerated Approval and Priority Review from the FDA. While the FDA did not recommend pursuing Accelerated Approval for the KRAS wild-type indication, Verastem is committed to exploring this pathway further, leveraging data from the ongoing RAMP 301 Phase 3 trial.
Verastem believes that the combination of avutometinib and defactinib can potentially become the preferred treatment for all LGSOC patients, regardless of their KRAS mutation status. This treatment could address the unmet needs of individuals with recurrent LGSOC, offering new hope and potentially extending survival for this patient population.
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**Categories:** News
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### [Resposne Pharmaceuticals Initates RDX-002 Phase 2 Study Weight management](https://www.clinicaltrialvanguard.com/news/resposne-pharmaceuticals-initates-rdx-002-phase-2-study-weight-management/)
**Published:** October 17, 2024
**Author:** Jon Napitupulu
**Content:**
Response Pharmaceuticals, a clinical-stage company specializing in weight management and [metabolic health](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/), has begun enrolling participants in a Phase 2 trial. This trial will evaluate their drug candidate, RDX-002, for its effectiveness in addressing weight rebound experienced by individuals after discontinuing GLP-1 agonist treatment for obesity.
RDX-002 operates by inhibiting intestinal microsomal triglyceride transfer protein (iMTP), reducing dietary fats’ absorption and, consequently, caloric intake. The study aims to assess the drug’s impact on post-meal triglyceride levels, weight fluctuations, and other cardiometabolic risk factors in individuals who have ceased using semaglutide or [tirzepatide](https://www.clinicaltrialvanguard.com/news/aardvarks-new-obesity-pipeline-data-offers-surprising-hope/), two common GLP-1 agonists.
The impetus for this research stems from a critical observation: patients often regain a significant portion of the weight lost while on GLP-1 agonists upon discontinuing these medications, thereby diminishing the associated cardiometabolic benefits.
Preliminary studies on RDX-002 have shown promise in mitigating weight gain linked to antipsychotic use, demonstrating good initial tolerability. The current trial seeks to explore its efficacy in this second significant weight management context as development for its use in addressing antipsychotic-induced weight gain continues.
Obesity is acknowledged as a chronic disease with serious cardiovascular and metabolic implications, including heightened risks of hyperlipidemia, insulin resistance, [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/), hypertension, and cardiovascular disease. While GLP-1 agonists have demonstrated remarkable success in weight reduction and mitigating these risks, most patients eventually stop using them.
RDX-002 offers a potential solution by lowering post-meal triglycerides, “bad” LDL-cholesterol levels, and improving glycemic control – all established risk factors for cardiovascular disease and diabetes. The double-blind study (NCT06640972) will track the efficacy and tolerability of RDX-002 over 12 weeks in individuals who successfully lost weight with GLP-1 agonists but are planning to discontinue treatment. Results from this trial are anticipated in the latter half of 2025.
In other news, Response Pharmaceuticals will present the findings from their proof-of-concept study on RDX-002’s effects on antipsychotic-induced weight gain at Obesity Week 2024 in San Antonio on November 4th.
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**Categories:** News
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### [Gilead and Merck Announce Phase 2 Islatravir and Lenacapavir Data](https://www.clinicaltrialvanguard.com/news/gilead-and-merck-announce-phase-2-islatravir-and-lenacapavir-data/)
**Published:** October 21, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences and Merck have announced positive results from a Phase 2 clinical trial investigating a novel combination therapy for HIV. The trial evaluated the effectiveness of weekly oral doses of islatravir, an experimental nucleoside reverse transcriptase translocation inhibitor, and lenacapavir, a first-in-class HIV-1 capsid inhibitor.
The study involved 104 adults with suppressed HIV who were currently on a stable regimen of Biktarvy. Participants were divided into two groups: one group continued taking Biktarvy daily, while the other switched to the once-weekly islatravir and lenacapavir combination.
At the 48-week mark, both groups demonstrated comparably high rates of viral suppression, with 94.2% of the islatravir and lenacapavir group and 92.3% of the Biktarvy group maintaining viral suppression. Notably, no participants in either group had a viral load resurgence above 50 copies/mL, a key indicator of treatment efficacy.
Treatment-related adverse events were generally mild. In the islatravir and lenacapavir groups, the most common side effects were dry mouth and nausea, each experienced by 3.8% of participants. No serious adverse events related to the study drug were reported in either group.
These positive Phase 2 results have led to the advancement of the islatravir and lenacapavir combination therapy to Phase 3 clinical trials. Two Phase 3 studies are currently underway to further evaluate the efficacy and safety of this fixed-dose combination regimen in individuals with suppressed HIV.
This novel once-weekly oral treatment holds significant promise for transforming HIV care. It offers a less frequent dosing regimen compared to daily pills, which could potentially improve adherence, minimize the stigma associated with daily medication, and ultimately contribute to [better health outcomes](https://www.clinicaltrialvanguard.com/news/bms-enhances-health-equity-grant-initiatives-for-better-health-outcomes/) for individuals living with HIV.
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**Categories:** News
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### [Oncoprism® Earns Medicare Coverage: Predicting Immunotherapy Response](https://www.clinicaltrialvanguard.com/news/oncoprism-earns-medicare-coverage-predicting-immunotherapy-response/)
**Published:** October 21, 2024
**Author:** Jon Napitupulu
**Content:**
Cofactor Genomics, a clinical-stage company focused on RNA analysis for precision medicine, has received national coverage from Medicare contractor Palmetto GBA for its OncoPrism test. This predictive diagnostic tool analyzes a patient’s tumor immune profile using high-dimensional RNA-based Health Expression Models (HEMs). It predicts a patient’s response to immune checkpoint inhibitor (ICI) immunotherapy, leading to more informed treatment decisions and improved outcomes for cancer patients.
This Medicare coverage decision specifically benefits patients with recurrent and metastatic head and neck squamous cell carcinoma (RM-[HNSCC](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/)) being considered for ICI immunotherapy, either alone or in combination with chemotherapy. OncoPrism’s effectiveness in predicting patient response to ICI is currently being evaluated in a multicenter national clinical trial called PREDAPT, encompassing eleven solid tumor cancers, including RM-HNSCC.
Cofactor Genomics specializes in utilizing RNA to address critical healthcare challenges. Their PRISM database houses patented HEMs, representing significant advancements in machine learning and RNA analysis. These models leverage vast amounts of biological RNA data to provide insights into biology, disease, and therapy response. Cofactor’s innovative work has earned recognition through publications in prestigious scientific journals and collaborations with numerous healthcare systems. OncoPrism, a CAP/CLIA-validated and Medicare-approved test, exemplifies Cofactor’s commitment to advancing precision medicine through RNA decoding.
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**Categories:** News
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### [ArteraAI Prostate Test Now Accessible in CA](https://www.clinicaltrialvanguard.com/news/arteraai-prostate-test-now-accessible-in-ca/)
**Published:** October 22, 2024
**Author:** Jon Napitupulu
**Content:**
Artera, a company specializing in AI-powered cancer tests, has received a license from the California Department of Public Health, allowing them to offer their ArteraAI Prostate Test in California. This makes the test accessible in one of the biggest healthcare markets in the United States. The ArteraAI Prostate Test is unique in its ability to both predict the effectiveness of therapy and forecast long-term outcomes for patients with localized [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/). Available starting October 10, 2024, this test provides data-driven insights to help personalize cancer treatment decisions for patients in California.
This development is significant for Artera as it expands its reach to a state with a high incidence of prostate cancer. They aim to make AI-driven precision medicine widely accessible, giving patients more information and clarity regarding their treatment options.
Artera received College of American Pathologists (CAP) accreditation in August 2024, demonstrating their dedication to quality and patient safety. This accreditation process involves rigorous evaluation of laboratory practices, ensuring the highest standard of care.
The development of the MMAI biomarker used in the ArteraAI test originated at the University of California, San Francisco (UCSF). This predictive biomarker, acknowledged in national guidelines for localized prostate cancer, can significantly influence treatment decisions, particularly for patients considering androgen deprivation therapy (ADT) along with radiation. The availability of such validated technologies is crucial for both clinicians and patients, ensuring optimal and personalized care.
Artera is a leading precision medicine company that develops AI-powered tests to personalize cancer treatment. Their ArteraAI Prostate Test, the first of its kind, offers both prognostic and predictive results for patients with localized prostate cancer. This test utilizes a unique algorithm that analyzes digital images from a patient’s biopsy and clinical data. This information helps determine the patient’s prognosis and predict the effectiveness of specific therapies. This AI technology has been validated through numerous Phase 3 randomized trials. Artera’s laboratory is CLIA-certified and College of American Pathologists (CAP) accredited. The ArteraAI Prostate Test is available through their laboratory in Jacksonville, Florida, and can be ordered online at Artera.ai.
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**Categories:** News
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### [New Study Links Antidepressant Use to Muscle Health and Cardiometabolic Risk](https://www.clinicaltrialvanguard.com/news/new-study-links-antidepressant-use-to-muscle-health-and-cardiometabolic-risk/)
**Published:** October 22, 2024
**Author:** Jon Napitupulu
**Content:**
A recent study from [AMRA Medical](https://www.clinicaltrialvanguard.com/news/amra-medicals-breakthrough-fat-z-score-biomarkers-unlock-the-power-of-tirzepatide-treatment/) and the University of Oslo sheds light on the impact of antidepressants, specifically SSRIs and TCAs, on muscle health and cardiometabolic risk. This research is particularly relevant given the rising global use of antidepressants and their potential adverse effects.
The study, conducted as part of the CoMorMent project, analyzed data from the UK Biobank database, focusing on SSRI and TCA users. The findings revealed that SSRI users, compared to a control group, exhibited higher visceral fat, reduced muscle volume, and increased muscle fat infiltration. While female SSRI users experienced a greater increase in BMI over time, male users demonstrated a more concerning body composition profile and elevated cardiovascular disease risk. Similarly, TCA users showed unfavorable muscle composition compared to the control group, with both genders exhibiting an increased risk of developing [Type 2 Diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/).
AMRA Medical’s innovative muscle volume and fat z-scores were instrumental in this research. These scores, derived from whole-body MRI scans, provide a nuanced understanding of individual deviations from expected body composition norms, going beyond traditional metrics like BMI. The study highlights the importance of these z-score biomarkers in evaluating individual responses to antidepressants and emphasizes the often-overlooked role of body fat distribution and muscle composition in antidepressant treatment.
AMRA Medical aims to utilize these findings to pave the way for future studies focused on improving mental and cardiometabolic health for antidepressant users. The company is dedicated to applying its z-score methodology across various disease areas, including studying neuropsychiatric drugs with metabolic effects. This commitment seeks to aid in developing targeted and effective treatments while minimizing potential risks.
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**Categories:** News
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### [Gilead Unveils Latest Liver Disease Research at AASLD 2024](https://www.clinicaltrialvanguard.com/news/gilead-unveils-latest-liver-disease-research-at-aasld-2024/)
**Published:** October 22, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences is set to present over 40 research abstracts at The Liver Meeting® 2024, highlighting its commitment to advancing liver disease research. A significant portion of these presentations will focus on new data for primary biliary cholangitis (PBC), including findings from the RESPONSE trial showcasing the efficacy and safety of Livdelzi® (seladelpar) in patients with PBC and compensated cirrhosis. Additional data will delve into Livdelzi’s impact on pruritus, a common and often debilitating symptom of PBC, and long-term efficacy and safety results from the ongoing ASSURE study.
Beyond PBC, Gilead will share an interim analysis of the Phase 3 MYR301 study, evaluating [bulevirtide](https://www.clinicaltrialvanguard.com/news/gilead-announces-breakthrough-hdv-treatment-data-in-new-england-journal-of-medicine/) monotherapy in individuals with hepatitis Delta virus (HDV). This analysis will assess if patients maintain positive virological and biochemical responses one year after discontinuing bulevirtide treatment. Furthermore, Gilead will present 144-week data on patient-reported outcomes for those receiving the 2 mg dose of bulevirtide, adding to the growing body of long-term data for this HDV treatment.
Additional research to be presented includes safety and tolerability outcomes for Epclusa® (velpatasvir/sofosbuvir) in pregnant individuals with chronic HCV, real-world findings from the SVR10K study demonstrating the effectiveness of direct-acting antivirals against all HCV genotypes, and late-breaking data from a Phase 2a study assessing novel combination therapies for chronic hepatitis B virus (HBV). Gilead’s presence at the conference underscores its commitment to addressing unmet needs and improving treatment outcomes for individuals living with various liver diseases.
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**Categories:** News
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### [FDA Approves Pfizer's Abrysvo RSV Vaccine for Adults](https://www.clinicaltrialvanguard.com/news/fda-approves-pfizers-abrysvo-rsv-vaccine-for-adults/)
**Published:** October 23, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer recently received approval from the U.S. Food and Drug Administration (FDA) for ABRYSVO®, its respiratory syncytial virus (RSV) vaccine. This approval expands the vaccine’s use to individuals between 18 and 59 years old with an elevated risk of RSV-related lower respiratory tract disease (LRTD). ABRYSVO is now the most broadly applicable RSV vaccine for adults, encompassing those 60 years and older. It maintains its unique standing as the sole RSV vaccine approved for pregnant individuals between 32 and 36 weeks of gestation, offering protection for infants from birth to six months.
This FDA decision stems from findings in the MONeT Phase 3 clinical trial, which studied the vaccine’s safety, tolerability, and how it affects the immune system in adults susceptible to RSV complications due to pre-existing health conditions. Pfizer plans to share these results with the scientific community through peer-reviewed publications and upcoming conference presentations.
Statistics highlight the significance of this approval: a notable portion of U.S. adults aged 18 to 49 have underlying conditions like [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), diabetes, or respiratory illnesses that make them vulnerable to RSV-associated LRTD. This percentage increases in the 50 to 64 age group.
RSV poses a widespread health concern as a common respiratory virus. It can lead to severe illness and even death by affecting the lungs and airways. Various chronic conditions, such as cardiovascular disease, lung disease, compromised immunity, diabetes with complications, and severe obesity, heighten an individual’s risk of experiencing severe RSV.
ABRYSVO is a bivalent vaccine engineered to provide comprehensive protection against RSV-LRTD, regardless of the specific RSV subgroup. The vaccine targets the RSV fusion protein in its prefusion state, which is crucial for the virus’s ability to infect cells, making it a key target for neutralizing antibodies.
In addition to this recent approval, ABRYSVO has garnered approvals for use in older adults and pregnant women to protect infants in multiple countries. Its journey includes recommendations from key advisory committees for its use in various age groups and risk categories.
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**Categories:** News
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### [OPM-101 RIPK2 Inhibitor Shows Strong Safety, No Cardiac Toxicity in Phase 1 Study](https://www.clinicaltrialvanguard.com/news/opm-101-ripk2-inhibitor-shows-strong-safety-no-cardiac-toxicity-in-phase-1-study/)
**Published:** October 23, 2024
**Author:** Jon Napitupulu
**Content:**
[Oncodesign](https://www.clinicaltrialvanguard.com/news/oncodesigns-precision-medicine-a-new-era-in-systemic-radiotherapy/) Precision Medicine (OPM) has announced the successful completion of its Phase 1 clinical trial for [OPM-101](https://www.clinicaltrialvanguard.com/news/opm-records-unprecedented-success-in-phase-1-of-opm-101-human-trials/), an orally administered RIPK2 inhibitor. The trial began in February 2023 and concluded in October 2024 and involved 104 healthy volunteers.
The study was divided into two parts: single ascending dose (SAD) and multiple ascending dose (MAD). In the SAD portion, participants received a single dose of OPM-101 ranging from 5mg to 1000mg or a placebo. The MAD portion saw participants receiving 75mg, 150mg, or 300mg of OPM-101, or a placebo, twice daily for 14 days.
OPM-101 demonstrated excellent safety and tolerability profiles throughout the trial. No serious adverse events were reported, and no participant withdrew due to adverse effects. The few reported adverse events were generally mild and transient, primarily headaches.
Significantly, OPM-101 showed promising results in inhibiting the RIPK2 pathway, a key target for treating inflammatory diseases and certain cancers. The inhibitory effect was rapid and sustained, reaching a maximum of 90-100% within hours of administration and persisting for the treatment period.
Pharmacokinetic analysis revealed that OPM-101 is quickly absorbed, reaching peak concentration within 2-4 hours, and has a half-life of approximately 12 hours. These characteristics make it suitable for patient use. Researchers believe a twice-daily dose of 150mg could achieve optimal therapeutic levels based on the trial data.
Encouraged by these positive results, OPM plans to submit a protocol for a Phase 1b/2a clinical trial in the fourth quarter of 2024. This next phase will evaluate OPM-101’s efficacy in patients with specific inflammatory diseases or cancers.
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**Categories:** News
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### [Nanoknife® System's Long-Term Effects Studied in New Trial](https://www.clinicaltrialvanguard.com/news/nanoknife-systems-long-term-effects-studied-in-new-trial/)
**Published:** October 24, 2024
**Author:** Jon Napitupulu
**Content:**
[AngioDynamics](https://www.clinicaltrialvanguard.com/news/angiodynamics-initiates-landmark-trial-for-pulmonary-embolism-treatment/), a medical technology company specializing in vascular health and cancer treatment, has initiated a global registry study to assess the long-term effectiveness of the NanoKnife System in treating men with intermediate-risk [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/). This study is being conducted in collaboration with University College London Hospital (UCLH), a leading institution in robotic prostate surgery and focal therapy.
Intermediate-risk prostate cancer represents a significant portion of diagnoses, and traditional treatments like surgery or radiotherapy often lead to complications such as urinary incontinence and erectile dysfunction. The NanoKnife System presents a less invasive alternative, utilizing irreversible electroporation (IRE) technology to eliminate targeted cells without heat. This method aims to preserve surrounding healthy tissue and minimize adverse effects on quality of life.
The NICE in the U.K. recently reclassified the NanoKnife System from “Research Only” to “Special Arrangements,” permitting broader usage while data collection continues. This shift reflects the growing recognition of this technology within national urology guidelines. The international registry study, aiming to enroll at least 500 participants, builds upon existing single-center studies demonstrating the system’s safety and efficacy in treating tumors and potentially delaying the need for radical intervention.
This research aligns with ongoing randomized controlled trials comparing NanoKnife to radical treatments. By meticulously monitoring patients after treatment, the study will assess quality of life, overall survival, prostate-specific mortality, and the need for any subsequent interventions. The findings will contribute to a more comprehensive understanding of the NanoKnife System’s role in prostate cancer treatment, potentially paving the way for wider adoption and improved patient care.
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**Categories:** News
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### [Predicting First-Line Incontinence Treatment Success in Women](https://www.clinicaltrialvanguard.com/news/predicting-first-line-incontinence-treatment-success-in-women/)
**Published:** October 25, 2024
**Author:** Jon Napitupulu
**Content:**
Researchers have developed a potential machine learning model that could personalize treatments for female pelvic floor disorders, specifically urinary incontinence. Based on an analysis of over 600 patients, this model utilizes data such as demographics, initial symptoms, and information gathered from the Leva® Pelvic Health System within the first 14 days of use.
The researchers employed various machine learning techniques and found the Random Forest model to be the most effective, accurately predicting treatment outcomes 78% of the time. This model’s accuracy was further validated using an independent dataset. This breakthrough allows clinicians to identify patients who might benefit from additional support or treatment adjustments, ultimately aiming to improve treatment success rates.
The development of this model was a collaborative effort between researchers from Massachusetts General Hospital, Massachusetts Institute of Technology, and [Axena Health](https://www.clinicaltrialvanguard.com/news/axena-health-and-mayo-clinic-partner-to-advance-urinary-incontinence-care/), a medical device company specializing in female pelvic health. The team believes this discovery will significantly impact how clinicians approach treatment for urinary incontinence, enabling them to create personalized plans based on data-driven predictions.
The Leva Pelvic Health System, used in this research, is a non-invasive, medication-free treatment for urinary incontinence and chronic fecal incontinence. It combines a vaginal motion sensor with software to provide real-time feedback and track progress, empowering women to strengthen their pelvic floor muscles at home. The system is available by prescription, ensuring physician oversight and guidance throughout treatment.
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**Categories:** News
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### [Precision Biosciences Announces First-in-Human Study for Chronic Hepatitis B Treatment](https://www.clinicaltrialvanguard.com/news/precision-biosciences-announces-first-in-human-study-for-chronic-hepatitis-b-treatment/)
**Published:** October 25, 2024
**Author:** Jon Napitupulu
**Content:**
[Precision BioSciences](https://www.clinicaltrialvanguard.com/news/precision-biosciences-programs-make-a-stunning-return-collaboration-with-prevail-therapeutics-concludes/), a clinical-stage gene editing company, has announced a significant advancement in its quest to cure chronic hepatitis B. The company has received Clinical Trial Application (CTA) approval in Moldova for its lead candidate, PBGENE-HBV, marking a pivotal step towards a potential cure for the nearly 300 million individuals globally affected by this chronic disease.
This approval paves the way for initiating a Phase 1 clinical program, with patient dosing imminent. Precision BioSciences has adopted a multi-track regulatory strategy, leveraging promising preclinical data to pursue additional CTA and IND approvals internationally. This approach aims to expedite trial enrollment and generate crucial clinical data on both safety and efficacy.
PBGENE-HBV distinguishes itself as the first therapeutic approach to undergo clinical trials designed to eliminate the root cause of chronic hepatitis B – the persistent cccDNA in liver cells. Current standard of care, relying on daily antiviral medication, only offers a 1-3% chance of functional cure. PBGENE-HBV utilizes the company’s proprietary ARCUS® gene editing platform to deliver a specially designed nuclease, via lipid nanoparticles, directly to liver cells. This nuclease targets and eliminates the viral genome, including cccDNA and integrated HBV DNA.
The company plans to present a comprehensive overview of the PBGENE-HBV program on November 15th, preceding the American Association for the Study of Liver Diseases Annual Meeting. This update will provide further insights into the preclinical safety data and Phase 1 trial design.
Chronic hepatitis B is a serious global health concern with limited curative options. A significant proportion of patients develop complications leading to cirrhosis, liver failure, and [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/). PBGENE-HBV offers a beacon of hope by directly targeting and eliminating the source of the disease, potentially leading to functional cures and freeing patients from lifelong dependence on antiviral therapies.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Dermavant Unveils VTAMA Cream Data for Atopic Dermatitis](https://www.clinicaltrialvanguard.com/news/dermavant-unveils-vtama-cream-data-for-atopic-dermatitis/)
**Published:** October 28, 2024
**Author:** Jon Napitupulu
**Content:**
Dermavant Sciences has released promising data from their ADORING 3 study, highlighting the effectiveness of VTAMA cream, 1% in treating [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (AD), also known as eczema. The open-label, long-term extension study involved adults and children as young as two years old. Results demonstrated high rates of complete skin clearance and significant treatment-free intervals.
Over half of the participants achieved complete disease clearance (vIGA-AD score of 0) at least once during the study. After stopping treatment upon reaching complete clearance, patients experienced an average of approximately 80 days without needing additional treatment while maintaining clear or almost clear skin. These findings build upon previous positive results from the ADORING 1 and ADORING 2 pivotal studies, further demonstrating the potential of VTAMA cream as a safe and effective treatment for AD in both adults and children.
The ADORING 3 study enrolled patients from prior studies, including those new to VTAMA cream, and evaluated various endpoints such as treatment emergent adverse events, local tolerability, and efficacy markers. The study reinforced the safety and tolerability profile of VTAMA cream observed in earlier trials.
VTAMA cream, a novel aryl hydrocarbon receptor agonist, is a once-daily, steroid-free topical cream. It’s already approved in the U.S. for treating plaque [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/) in adults. In April 2024, the U.S. Food and Drug Administration (FDA) accepted the company’s Supplemental New Drug Application (sNDA) for VTAMA cream for the treatment of AD in adults and children two years of age and older, with a decision expected in Q4 2024.
Atopic dermatitis is a common inflammatory skin disease affecting millions globally. Characterized by itchy, red, and inflamed skin, AD can significantly impact quality of life. Dermavant Sciences remains committed to developing innovative therapies like VTAMA cream to address this unmet medical need in dermatology.
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**Categories:** News
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### [Leo Pharma Presents Long-Term Adbry® Trial Results at Fall Clinical 2024](https://www.clinicaltrialvanguard.com/news/leo-pharma-presents-long-term-adbry-trial-results-at-fall-clinical-2024/)
**Published:** October 28, 2024
**Author:** Jon Napitupulu
**Content:**
LEO Pharma Inc., a leader in medical dermatology, announced the final results of ECZTEND, a five-year study on Adbry® (tralokinumab-ldrm). The study, presented at the Fall Clinical Dermatology Conference in Las Vegas, demonstrated the long-term safety and efficacy of Adbry® in treating moderate-to-severe [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (AD) in adults and adolescents.
ECZTEND, a Phase 3, open-label, single-arm, multi-center, long-term extension study, involved 1672 adult and adolescent participants aged 12 and above. Participants joined ECZTEND after completing treatment with Adbry® in previous trials. The study revealed no new safety concerns after long-term Adbry® use (up to one year in parent trials and up to five years in ECZTEND). The overall safety profile remained consistent with the initial placebo-controlled trials, with most adverse events reported as mild/moderate.
The study also showed that Adbry® provided sustained effectiveness for up to six years. A significant reduction in the Eczema Area and Severity Index ([EASI-75](https://www.clinicaltrialvanguard.com/news/apogees-zumilokibart-hits-65-9-easi-75-response-in-phase-2-ad-trial/)) from baseline to Week 248 was observed in 92.9% of participants. Additionally, 66.7% of participants achieved an Investigator’s Global Assessment score of 0 (clear) or 1 (almost clear) (IGA 0/1) within the same timeframe. Improvements in itch, sleep, and overall quality of life were also noted.
These findings provide valuable long-term data for healthcare professionals, supporting the sustained use of Adbry® in managing this chronic condition. The detailed results of the ECZTEND study will be published in the peer-reviewed journal, Skin.
Source link:
**Categories:** News
---
### [Unpacking FDA Guidance on Digital Health Technologies in Clinical Trials](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/)
**Published:** October 29, 2024
**Author:** Moe Alsumidaie
**Content:**
[Digital Health Technologies](https://www.clinicaltrialvanguard.com/executiveinterviews/digital-health-technologies-new-era-in-clinical-research/) (DHTs) use in clinical research is no longer a futuristic concept—it’s quickly becoming the norm. Recognizing this shift, the [FDA issued comprehensive guidance in 2023](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/digital-health-technologies-remote-data-acquisition-clinical-investigations "FDA issued comprehensive guidance in 2023") on Digital Health Technologies for Remote Data Acquisition in Clinical Investigations. This guidance sets critical standards for validating, deploying, and monitoring these tools in clinical trials. The guidance stresses the importance of reliability, data quality, patient safety, and privacy—key factors as trials become more decentralized and reliant on technology.
Throughout this article, we’ll use Aktiia’s continuous blood pressure (BP) monitoring technology as a reference to illustrate the FDA’s key recommendations. While Aktiia’s device provides cutting-edge BP tracking, we will focus on how its use aligns with the FDA’s detailed framework for DHTs in clinical investigations to help you better navigate the guidance on existing DHTs in the marketplace.
[](https://healthcare.aktiia.com/)
## [](#what-are-digital-health-technologies)What Are Digital Health Technologies?
According to the FDA, Digital Health Technologies encompass many tools designed to collect health data remotely. These include wearables, sensors, mobile applications, and software transmitting health-related data in real-time. The goal of the FDA’s guidance here is to clarify what qualifies as a DHT and, more importantly, to establish how these technologies can contribute to clinical trials.
The FDA recognizes that data collected in traditional clinical settings can be limited. In-person visits provide only a snapshot of a patient’s condition, often missing the broader picture of their daily health patterns. This is where DHTs, like Aktiia’s continuous BP monitoring bracelet, come in. By enabling constant, real-world monitoring, Digital Health Technologies allow researchers to gather data on how a patient’s health fluctuates in their natural environment. This continuous data provides richer insights into conditions that require hypertension management, such as cardiovascular disease, diabetes, and chronic kidney disease, which often benefit from precise, long-term blood pressure tracking to inform clinical outcomes better.
The FDA emphasizes DHTs because they offer a more holistic view of patient health, moving beyond the confines of the clinic. However, with this new capability comes the responsibility to ensure that the data collected remotely is just as reliable and accurate as that collected in a controlled environment.
## [](#key-considerations-for-selecting-dhts-in-clinical-trials)Key Considerations for Selecting DHTs in Clinical Trials
A significant point of emphasis in the FDA’s guidance is the concept of fit-for-purpose—ensuring that the selected DHTs are appropriate for the specific trial’s objectives and the patient population involved. The FDA doesn’t just want sponsors to use technology for technology’s sake; it wants to ensure that these tools effectively serve the trial’s intended goals.
The term “fit-for-purpose” is central to the FDA’s guidance, and it essentially means that a DHT should be appropriately validated and capable of addressing the specific objectives of a clinical trial. The technology must be well-suited for the patient population, the studied condition, and the data required for meaningful analysis. When the FDA urges sponsors to consider whether a DHT is “fit-for-purpose,” it’s pushing for a thoughtful selection process. This involves ensuring that the chosen technology can reliably collect data relevant to the trial’s endpoints, functions appropriately in the intended setting, and fits within the broader clinical context. Validation for the specific population also ensures that the DHT can accurately measure the health outcomes of interest.
For instance, Aktiia’s BP monitor is fit for purpose in trials where continuous blood pressure monitoring is essential, such as in chronic kidney disease or hypertension-related studies. However, in trials focused on [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) or sleep disorders, where different metrics are crucial (e.g., brain activity or sleep patterns), a DHT designed for cardiovascular monitoring wouldn’t be fit for purpose unless adapted to the specific needs of those studies.
[](https://healthcare.aktiia.com/)
## [](#ensuring-data-integrity-and-endpoint-evaluation)Ensuring Data Integrity and Endpoint Evaluation
One of the key pillars of the FDA’s guidance is ensuring data integrity. For the FDA, this means that DHTs must not only collect data but do so in a consistent, reliable manner that can withstand scrutiny. The guidance outlines how sponsors should validate DHTs to ensure that remotely collected data meets the same standards as data gathered through traditional, in-person means.
As the FDA opens the door to more remote data collection, the agency remains aware of the risks associated with collecting data outside controlled environments where inconsistencies or errors could arise. Aktiia, however, has undergone rigorous testing and validation to meet these stringent requirements. Its blood pressure monitoring technology has been proven to track blood pressure accurately across different patient demographics, environmental conditions, and health statuses. This includes testing in real-world scenarios where patients may have comorbid conditions or varying physical states, such as during physical activity or periods of rest. Aktiia’s system still reliably collects accurate blood pressure measurements under these varied conditions.
Furthermore, the FDA emphasizes that trial endpoints must be directly tied to the data collected, and data integrity is paramount. In the case of Aktiia’s device, the company has implemented robust mechanisms to reduce noise and ensure the clarity of the data it collects. Aktiia’s technology effectively filters out potential data inconsistencies—such as those caused by patient movement or environmental factors—so that the blood pressure readings are accurate and precise.
Aktiia can support reliable and clinically meaningful endpoints in trials where blood pressure is a primary measure by ensuring that the data is clean and clear. The FDA requires that the data not only be collected but also processed and analyzed to retain its clinical relevance. This is critical for informing real-world medical decisions. Aktiia’s system can handle large volumes of data, process it to ensure that the resulting insights are valid and actionable, and maintain the high level of data integrity the FDA expects to support study endpoints.
[](https://healthcare.aktiia.com/)
## [](#patient-safety-risk-and-data-privacy-concerns)Patient Safety Risk and Data Privacy Concerns
The FDA’s guidance also focuses heavily on risk management, particularly regarding patient safety and data privacy. While the benefits of DHTs are significant, the risks are also higher—both in terms of potential harm from the device itself and the increased exposure to cybersecurity threats.
Devices that patients wear continuously must be physically safe and comfortable for long periods. The FDA guidance suggests that sponsors evaluate the potential physical risks—like skin irritation or discomfort—from prolonged use of wearables. If patients stop using the device due to discomfort, it could lead to data loss and, worse, patient harm. On the digital side, data privacy is paramount. DHTs often transmit sensitive health data over wireless networks, and the FDA stresses the need for strong encryption and data security protocols. This is crucial not only to comply with regulations like HIPAA but also to maintain patient trust. If participants fear their data could be compromised, it could affect their willingness to participate, undermining the trial. The FDA’s focus on risk mitigation is about ensuring that while we expand the use of DHTs, we do so without compromising the safety of patients or the integrity and privacy of their data.
For example, Aktiia’s wristband is designed with patient comfort in mind, allowing for prolonged usage without discomfort, which minimizes the risk of patients removing the device and disrupting data collection. Moreover, Aktiia’s encryption policies and cybersecurity protocols meet the FDA’s stringent requirements, ensuring patient data is securely transmitted and stored. By providing both comfort and strong data protection, Aktiia maintains patient trust and ensures that its device supports accurate, reliable data collection throughout clinical trials.
## [](#regulatory-and-submission-considerations)Regulatory and Submission Considerations
The FDA encourages sponsors to engage with them early, often when incorporating DHTs into clinical trials. Early engagement allows the FDA to ensure that the DHT meets all necessary regulatory standards before it becomes a core part of a trial’s data collection.
In many cases, Digital Health Technologies are gathering data that will be used to support key regulatory decisions. These technologies must be rigorously validated for the FDA before trial data is submitted for review. The earlier sponsors can demonstrate the reliability of their DHT, the smoother the regulatory process will be. The FDA is also keen to ensure that sponsors include comprehensive documentation in their submissions, covering everything from device design and performance characteristics to data security measures. For example, Aktiia has been rigorously validated as a medical device (it has a CE mark in Europe); submitting this documentation to the FDA provides the necessary evidence that the device meets the validation requirements of the agency. This isn’t just about checking boxes—it’s about ensuring that the FDA fully understands how the DHT will be used and its impact on the trial results.
[](https://healthcare.aktiia.com/)
## [](#training-and-technical-support-for-dht-users)Training and Technical Support for DHT Users
The FDA’s guidance highlights the importance of proper training and technical support for trial participants and investigators. This ensures that everyone involved knows how to use the DHT correctly and that the data collected is reliable.
Even the most advanced DHT can fail if it’s not used correctly. The FDA emphasizes that patients and staff must receive clear technology operation instructions. For example, Aktiia’s BP monitor has training in the app that is simple and easy for patients to navigate; it trains patients on how to wear the device correctly, sync the data, and troubleshoot any potential issues. Likewise, trial staff must be equipped to assist patients and monitor the incoming data for accuracy and completeness. Training is a key safeguard to ensure that data is collected consistently and that any issues that arise during the trial can be resolved quickly, minimizing disruptions.
## [](#future-implications-of-the-guidance)Future Implications of the Guidance
Finally, the FDA’s guidance on Digital Health Technologies points to a future where clinical trials are increasingly decentralized and patient-centered. The agency sees DHTs as a way to make clinical trials more inclusive and reflective of real-world conditions. Trials that rely solely on clinic-based visits may exclude certain populations or fail to capture how treatments work in everyday life. By encouraging the use of Digital Health Technologies, the FDA is promoting a model of clinical research that is more flexible, accessible, and capable of capturing richer data and more reflective of patient experiences. The FDA’s forward-looking guidance sets the stage for a future where real-world evidence plays a more significant role in clinical research, ultimately speeding up drug development and improving patient outcomes.
## [](#summary)Summary
The FDA’s 2023 guidance on Digital Health Technologies for Remote Data Acquisition offers a comprehensive roadmap for integrating cutting-edge tools into clinical research. By focusing on validation, data integrity, risk management, and regulatory compliance, the FDA ensures that DHTs can enhance trial quality without sacrificing patient safety or data reliability. This guidance supports the safe and effective use of DHTs and pushes the boundaries of clinical research toward a more decentralized and patient-focused future, where real-time data offers deeper insights into how treatments impact lives.
*This article is sponsored by [Aktiia](https://healthcare.aktiia.com/ "Aktiia").*
[](https://healthcare.aktiia.com/)
**Categories:** Article
---
### [How to Alleviate Clinical Trial Burden at Study Sites](https://www.clinicaltrialvanguard.com/executiveinterviews/how-to-alleviate-clinical-trial-burden-at-study-sites/)
**Published:** October 31, 2024
**Author:** Moe Alsumidaie
**Content:**
In this interview, Joseph Kim, Chief Strategy Officer at [ProofPilot](https://www.clinicaltrialvanguard.com/executiveinterviews/proofpilot-and-lokavant-aquisition-aligning-trial-ops-with-predictive-analytics/), shares how to revolutionize clinical trials at study sites. Kim discusses strategies to reduce site burden, enhance patient engagement, and tackle recruitment challenges, especially for underrepresented groups. He also explores the future of trials with real-world evidence (RWE) and long-term follow-up.
## [](#moe-alsumidaie-how-does-proofpilot-reduce-site-burden-while-maintaining-data-quality-and-patient-engagement)**Moe Alsumidaie: How does ProofPilot reduce site burden while maintaining data quality and patient engagement?**
Joseph Kim: At ProofPilot, we streamline clinical trials by orchestrating tasks, technologies, and instructional content into a cohesive workflow. This approach reduces the mental load on study coordinators, allowing them to focus on conducting trials and delivering patient care without being overwhelmed by disparate instructions and tech. Imagine it as a clinical trial GPS, guiding coordinators through each step efficiently. This orchestration is akin to how Google Maps revolutionized navigation, enabling any driver to navigate a city efficiently. Providing a clear, step-by-step guide, we help sites scale operations efficiently, while maintaining high data quality and patient engagement. For example, when a study coordinator enters our platform, all they need to do is select a visit, and voilá, everything they need to properly conduct
Joseph Kim, Chief Strategy Officer at ProofPilot
that visit is laid out in front of them, eliminating the need to reference multiple manuals or flowcharts. This reduces site burden, alleviates tech proliferation and enhances the overall trial experience for both coordinators and patients. Our platform ensures that studies are conducted exactly as intended, which is the central plot of great science. And now our platform extends beyond trial conduct into recruitment and post-study engagement, providing a seamless experience from recruitment to return of results.
## [](#moe-alsumidaie-what-strategies-do-you-use-to-ensure-high-compliance-in-decentralized-trials)**Moe Alsumidaie: What strategies do you use to ensure high compliance in decentralized trials?**
Joseph Kim: Brick and mortar trials are hard enough when everyone is under the same roof and chain of command. Decentralized trials are exponentially harder. ProofPilot excels in managing decentralized trials by orchestrating the disparate stakeholders to ensure everyone is doing the right things in the right order. While we don’t position ourselves as a decentralized trial company, our capability allows us to effectively coordinate stakeholders not under the same chain of command or physical location. Thus, we maintain high compliance and data quality for Sponsors, even in decentralized settings, by ensuring that all tasks and technologies are integrated into a cohesive workflow. This approach simplifies the trial process and enhances patient engagement and retention.
## [](#moe-alsumidaie-how-do-you-balance-customization-for-sponsors-with-standardized-protocols-in-global-trials)**Moe Alsumidaie: How do you balance customization for sponsors with standardized protocols in global trials?**
Joseph Kim: At ProofPilot, we offer a highly configurable platform branded to the sponsor, allowing for a tailored clinical experience while adhering to standardized protocols and allowing for flexibility to integrate with other systems. Whether sponsors need a comprehensive end-to-end solution or specific components like recruitment or post-study engagement, our platform is designed to work well together or be mixed and matched with other technologies and sponsor-specific business processes. This flexibility ensures that sponsors can maintain their brand identity and meet their specific requirements, even in diverse multi-site global trials. By offering any combination of an all-in-one experience or a la carte modules, we enable sponsors to deliver the kind of experience they want without being too disruptive. This adaptability is critical to balancing customization with the need for standardized protocols, ensuring that each trial is conducted efficiently and effectively, while fitting well into the ecosystem of technologies and processes.
## [](#moe-alsumidaie-how-does-proofpilot-address-recruitment-challenges-for-underrepresented-populations)**Moe Alsumidaie: How does ProofPilot address recruitment challenges for underrepresented populations?**
Joseph Kim: Recruitment enablers, especially for underrepresented populations, require a focus on easing connectivity with the site and helping to educate folks prior to consent. At ProofPilot, we allow patients to schedule their first call with study coordinators, reducing phone tag, which is burdensome for many underrepresented populations who don’t have the flexibility to take calls as will or are available during normal business hours. This approach helps reduces frustration and drop-offs, ensuring the patient and the study coordinator are locked in for that critical initial interaction. Additionally, we can provide a cadence of information to patients between the initial call and the first visit, helping them understand the trial and what to expect. This proactive communication helps improve the health literacy for this population who may be unfamiliar with the research world, and ensures that patients are fully informed and comfortable with their decision to join the trial. By focusing on these aspects, we address the root causes of recruitment challenges and enhance the overall trial experience for underrepresented populations.
## [](#moe-alsumidaie-how-does-proofpilot-adapt-to-the-demand-for-rwe-and-long-term-follow-up-in-trials)**Moe Alsumidaie: How does ProofPilot adapt to the demand for RWE and long-term follow-up in trials?**
Joseph Kim: Long-term follow-ups in clinical trials present unique challenges due to infrequent touchpoints. At ProofPilot, we address this by delivering a cadence of content and tasks to patients, ensuring a baseline level of high quality engagement. This integrated workflow enhances the patient experience and maintains engagement over time, even when touchpoints are months apart. By automating the delivery of instructions and reminders, we ensure that patients remain engaged and informed throughout the study. This approach reduces the burden on study coordinators and creates a high-quality experience for patients, ensuring that they receive consistent communication, encouragement and support.
## [](#moe-alsumidaie-is-there-anything-else-youd-like-to-add-about-proofpilots-approach)**Moe Alsumidaie: Is there anything else you’d like to add about ProofPilot’s approach?**
Joseph Kim: We’ve intentionally built a team that provides a 360-degree view of clinical research perspectives, incorporating die-hard expertise from pharma, technology companies, sites, and patients. Our products are designed to scale in a cost-effective manner, with rapid ramp-up times and self-service capabilities, offering unmatched value to our clients. By bringing together diverse expertise, including external insights from patient influencers, site operators and Pharma, we ensure that our solutions are innovative, practical, and aligned with the needs of all stakeholders in the clinical trial ecosystem. This comprehensive approach allows us to deliver a well-informed, purpose-built platform that addresses the central plot of research – ensuring that patients and sites participate and conduct research with quality and ease.
**Categories:** Article: Executive Interviews
---
### [Boomi, Base Life Science Partner to Boost Digital Transformation](https://www.clinicaltrialvanguard.com/news/boomi-base-life-science-partner-to-boost-digital-transformation/)
**Published:** October 31, 2024
**Author:** Jon Napitupulu
**Content:**
Boomi, a leader in intelligent integration and automation, has partnered with BASE Life Science, an Infosys company specializing in cloud-based solutions for the [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) industry. This partnership expands on Boomi’s existing relationship with Infosys and strengthens integration capabilities for organizations transitioning from outdated technologies to the Veeva Vault Platform. This collaboration aligns with Boomi’s partnership with Veeva, a prominent provider of cloud software for the life sciences industry, known for its applications in enterprise content management, customer relationship management, and data management.
The collaboration between Boomi, BASE, and Veeva aims to support life sciences organizations in modernizing their operations for enhanced productivity throughout the drug development process. Developing a new prescription drug is a costly and complex endeavor. A Tufts Center for the Study of Drug Development report indicates an average cost of $2.6 billion to bring a new medicine to market. This figure encompasses clinical trial expenses and development time. Stringent regulatory requirements introduce further complexity, demanding meticulous navigation of approval processes to meet safety and efficacy standards. Integrating diverse data sources and managing input from various stakeholders complicate workflows, potentially leading to inefficiencies and delays in delivering innovative treatments to patients.
This partnership aims to simplify integration processes, allowing companies to prioritize their core mission of improving healthcare outcomes. Boomi will utilize BASE’s industry expertise to create tailored solutions that address the specific needs of pharmaceutical companies transitioning from legacy systems to the Veeva Vault Platform. This strategic collaboration underscores Boomi’s dedication to transforming the life sciences industry’s integration approach. By enabling organizations to overcome outdated technology limitations and embrace digital solutions, Boomi and BASE aim to drive value across all stages of drug development.
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**Categories:** News
---
### [Organon Completes Acquisition of Dermavant](https://www.clinicaltrialvanguard.com/news/organon-completes-acquisition-of-dermavant/)
**Published:** October 31, 2024
**Author:** Jon Napitupulu
**Content:**
Organon, a global healthcare company focused on women’s health, has finalized its acquisition of Dermavant Sciences Ltd. from Roivant. This strategic move bolsters Organon’s dermatology offerings with the addition of [VTAMA](https://www.clinicaltrialvanguard.com/news/dermavant-unveils-vtama-cream-data-for-atopic-dermatitis/)® (tapinarof) cream, 1%, a novel topical treatment for plaque [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/) in adults.
VTAMA, a non-biologic, non-steroidal therapy, stands out for its favorable safety profile, lacking the warnings, precautions, and usage limitations often associated with other treatments. The acquisition reflects Organon’s commitment to providing innovative solutions for chronic skin conditions.
Currently, the U.S. Food and Drug Administration (FDA) is evaluating a supplemental New Drug Application (sNDA) for VTAMA cream as a potential treatment for [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (commonly known as eczema) in adults and children aged two and above. A decision from the FDA is anticipated in the fourth quarter of 2024.
Both plaque psoriasis and atopic dermatitis are prevalent inflammatory skin diseases, impacting a significant population globally and carrying a substantial burden, particularly for women. Integrating Dermavant’s expertise into Organon’s U.S. operations signifies a pivotal advancement in dermatological care. Organon aims to leverage its global reach and commercial capabilities to maximize the availability of VTAMA to patients worldwide. This acquisition exemplifies a collaborative approach to address unmet patient needs and advance dermatological solutions.
Source link:[ http://www.businesswire.com/news/home/20241028071253/en/Organon-Completes-Acquisition-of-Dermavant-including-Innovative-Dermatologic-Therapy-VTAMA%C2%AE-tapinarof-Cream-1](http://www.businesswire.com/news/home/20241028071253/en/Organon-Completes-Acquisition-of-Dermavant-including-Innovative-Dermatologic-Therapy-VTAMA%C2%AE-tapinarof-Cream-1)
**Categories:** News
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### [Lucent Diagnostics' New Blood Test for Early Alzheimer's Diagnosis](https://www.clinicaltrialvanguard.com/news/lucent-diagnostics-new-blood-test-for-early-alzheimers-diagnosis/)
**Published:** October 31, 2024
**Author:** Jon Napitupulu
**Content:**
Lucent Diagnostics has launched LucentAD Complete, a novel multi-marker blood test designed to enhance the detection of [Alzheimer](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/)’s Disease (AD). This test addresses limitations of existing diagnostic methods, particularly the uncertainty associated with the “intermediate zone” in single-marker tests like plasma p-Tau 217.
LucentAD Complete analyzes five AD-related biomarkers (p-Tau 217, Aβ42/40, NfL, GFAP) using a proprietary algorithm, resulting in significantly improved amyloid classification compared to single-marker tests. Clinical trials involving over 1,000 patients demonstrated the test’s ability to reduce the number of individuals falling into the intermediate zone by threefold. This translates to more conclusive results and a reduced need for invasive procedures like lumbar punctures or expensive amyloid PET scans.
The development of LucentAD Complete was supported by the Alzheimer’s Drug Discovery Foundation’s Diagnostics Accelerator. Experts recognize the test’s multi-marker approach as a significant advancement in blood-based AD testing, aligning with the understanding that Alzheimer’s is a complex disease influenced by multiple factors.
Lucent Diagnostics, a brand under Quanterix Corporation, is committed to delivering innovative tools for early cognitive disease detection. Their ultra-sensitive Simoa® technology bridges the gap between research and clinical practice, providing valuable resources for both institutions and healthcare providers. With extensive experience in neurology research, Quanterix, through Lucent Diagnostics, aims to revolutionize the landscape of cognitive disease management.
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**Categories:** News
---
### [Synedgen Gets $2.2M for Radiation Countermeasure](https://www.clinicaltrialvanguard.com/news/synedgen-gets-2-2m-for-radiation-countermeasure/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
Synedgen, Inc., a biotechnology company specializing in gastrointestinal disease treatment, has secured a $2.2 million contract from the Joint Warfighter Medical Research Program (JWMRP). The funding will support the development of MIIST305, a promising therapeutic, as a preventative measure against radiation exposure. This builds upon previous research and development of MIIST305 for radiation mitigation and [ulcerative colitis](https://www.clinicaltrialvanguard.com/news/abivax-clears-pre-nda-meeting-with-fda-for-obefazimod-in-ulcerative-colitis/) treatment.
MIIST305 is an orally administered, shelf-stable therapy designed to regenerate the gut barrier and mitigate systemic hyperinflammation originating in the gastrointestinal tract. Synedgen is developing MIIST305 for ulcerative colitis treatment and, with support from the Biomedical Advanced Research and Development Authority ([BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)), as a medical countermeasure for acute radiation syndrome (ARS) after radiation exposure. This new JWMRP contract will explore MIIST305’s potential to prevent radiation damage when administered proactively. The funds will facilitate drug substance and product manufacturing for animal studies, focusing on optimal dosage timing for maximum effectiveness.
This research addresses a critical unmet need for an FDA-approved therapeutic to protect the gastrointestinal tract from radiation injury, a risk faced by military personnel, first responders, and civilians. The findings from this JWMRP-funded program are expected to benefit ongoing ulcerative colitis and post-exposure ARS research programs. The gastrointestinal tract is highly susceptible to radiation damage, a common side effect observed in cancer patients undergoing radiation therapy. As a prophylactic treatment, MIIST305 holds significant potential as a radiation countermeasure and a protective agent for oncology patients.
Currently, no FDA-approved medical countermeasures exist to mitigate gastrointestinal radiation injury, although several drugs are available to address the later effects of hematopoietic acute radiation syndrome (H-ARS). In gastrointestinal acute radiation syndrome (GI-ARS), radiation toxicity destroys the intestinal epithelial barrier, leading to bacterial invasion, inflammation, and, ultimately, death. MIIST305’s demonstrated activity in the GI tract positions it as a potential preventative and post-exposure therapeutic for GI-ARS.
Synedgen’s Multivalent Innate Immune Signaling Target (MIIST) platform focuses on receptors in the glycocalyx, a crucial component of human innate immunity and increasingly recognized for its role in intestinal homeostasis. Developed with substantial peer review and government funding, the MIIST platform advances therapeutics for mucosal barrier protection and regeneration. Synedgen continues to advance its GI-ARS program with support from key US government agencies and maintains an in-house GMP manufacturing facility. The GI-ARS program receives funding from the Department of Health and Human Services, the Administration for Strategic Preparedness and Response (ASPR), and the Biomedical Advanced Research and Development Authority.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Verastem Oncology NDAs Avutometinib for Ovarian Cancer](https://www.clinicaltrialvanguard.com/news/verastem-oncology-ndas-avutometinib-for-ovarian-cancer/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
[Verastem Oncology](https://www.clinicaltrialvanguard.com/news/verastem-oncology-avatometinib-defactinib-nda-for-low-grade-serous-ovarian-cancer-gets-priority-review/) has completed its New Drug Application (NDA) to the U.S. FDA for a combination therapy targeting recurrent KRAS mutant low-grade serous [ovarian cancer](https://www.clinicaltrialvanguard.com/news/imunons-imnn-001-shows-lower-residual-disease-in-phase-2-ovarian-cancer-study/) (LGSOC). This investigational treatment combines avutometinib, an oral RAF/MEK clamp, and defactinib, an oral selective FAK inhibitor, and is intended for adults who have undergone at least one prior systemic therapy. Currently, no FDA-approved treatments exist specifically for LGSOC, a rare ovarian cancer distinct from its high-grade counterpart.
Verastem seeks Accelerated Approval and Priority Review for its NDA, given the significant unmet medical need in recurrent LGSOC. If granted Priority Review, the FDA aims to complete its evaluation within six months following a 60-day filing period. A potential FDA approval decision is anticipated by mid-2025. This approval would mark the first FDA-approved therapy for adult patients with recurrent KRAS mutant LGSOC in the United States.
The NDA submission follows preliminary data review with the FDA and is supported by updated Phase 2 RAMP 201 study results. These results, presented at the International Gynecologic Cancer Society 2024 Annual Meeting, demonstrated a 44% overall response rate, median progression-free survival of 22 months, and a 70% disease control rate at six months in patients with KRAS mutant LGSOC. The combination therapy was generally well-tolerated, with a 10% discontinuation rate due to adverse events across all patients. Supportive data from the FRAME Phase 1 trial, the initial study of this combination in recurrent LGSOC, is also included in the NDA.
Avutometinib plus defactinib previously received a Breakthrough Therapy Designation from the FDA for treating recurrent LGSOC after one or more prior therapies, including platinum-based chemotherapy. Both drugs, individually and in combination, were also granted Orphan Drug Designation for LGSOC treatment. Currently, Verastem is enrolling patients in RAMP 301, an international Phase 3 trial. This confirmatory study will investigate the combination therapy in recurrent LGSOC regardless of KRAS mutation status and could potentially support an expanded indication.
LGSOC is a persistent and ultimately fatal disease, impacting an estimated 6,000-8,000 women in the U.S. and 80,000 globally. It differs significantly from high-grade serous ovarian cancer, exhibiting higher recurrence rates and lower chemotherapy sensitivity. LGSOC typically affects younger women, with peak diagnoses between 20-30 and 50-60 years of age, and a median survival of around ten years. Current standard care includes hormone therapy and chemotherapy, but no FDA-approved treatments exist specifically for LGSOC.
Avutometinib is designed to create a more comprehensive and lasting anti-tumor response by forming inactive complexes of MEK with ARAF, BRAF, and CRAF. This unique mechanism maximally inhibits the RAS/MAPK pathway, preventing the compensatory MEK activation that can limit the effectiveness of MEK-only inhibitors. Verastem is exploring the potential of avutometinib combined with various therapies in RAMP trials as part of their Raf And Mek Program.
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**Categories:** News
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### [Koru Medical Systems Data at PODD Conference: Nursing Preference for FreedomEdge® Infusion System](https://www.clinicaltrialvanguard.com/news/koru-medical-systems-data-at-podd-conference-nursing-preference-for-freedomedge-infusion-system/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
[KORU Medical](https://www.clinicaltrialvanguard.com/news/koru-medicals-next-gen-subcutaneous-infusion-system/) Systems presented data at the 2024 PODD Conference showcasing nurse preference for the KORU FreedomEdge® Infusion System in administering subcutaneous oncology infusions. The study, conducted across six Danish hospitals, involved 33 nurses administering over 3,000 infusions of an oncology drug exceeding 10mL in volume, with an average administration time of around 10 minutes. It aimed to evaluate time spent with patients, nurses’ comfort levels, and overall nurse preference when comparing manual syringe administration with the FreedomEdge® system’s mechanical pump approach.
The increasing use of large-volume subcutaneous oncology drugs presents workflow challenges in clinical settings due to the extended hands-on administration time required for manual syringe pushes. This necessitates continuous manual pressure on syringes containing viscous drugs for approximately 10 minutes. Subcutaneous delivery of previously intravenous biologics is a growing trend among oncology pharmaceutical manufacturers. Five such drugs, with volumes greater than 5mL, are approved for in-clinic administration by healthcare professionals, accounting for an estimated one million global infusions.
Subcutaneous administration offers several advantages, including simplified treatment protocols, reduced strain on hospitals, and improved patient quality of life. The continued development of subcutaneous drug therapies creates a significant opportunity for drug delivery solutions like the KORU FreedomEdge® system, which offers an alternative to manual administration. The study results indicate a strong nurse preference for the FreedomEdge® system. KORU Medical anticipates filing for FDA clearance of its system with an oncology biologic in 2025.
KORU Medical specializes in developing, manufacturing, and commercializing innovative, patient-centric, large-volume subcutaneous infusion solutions. Their FREEDOM Syringe Infusion System encompasses the FREEDOM60® and FreedomEdge® Syringe Infusion Drivers, Precision Flow Rate Tubing™, and HIgH-Flo Subcutaneous Safety Needle Sets™. Initially FDA-cleared in 1994, the Freedom System caters to home self-administration by patients and ambulatory infusion center delivery by healthcare professionals. KORU Medical’s Novel Therapies business supports biopharmaceutical companies with products for feasibility and clinical trials during drug development. They also offer customization of the Freedom System for clinical and commercial use across various drug categories.
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**Categories:** News
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### [Cobenfy™ Long-Term Schizophrenia Data Presented at Psych Congress 2024](https://www.clinicaltrialvanguard.com/news/cobenfy-long-term-schizophrenia-data-presented-at-psych-congress-2024/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb recently unveiled positive topline results from two Phase 3 open-label trials, EMERGENT-4 and EMERGENT-5. These trials assessed the long-term efficacy, safety, and tolerability of COBENFY (xanomeline and trospium chloride) in adults with [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) over a 52-week treatment period. The data were presented at the 2024 Psych Congress in Boston.
EMERGENT-4, a 52-week open-label extension study, involved 156 adults with schizophrenia who had completed either the EMERGENT-2 or EMERGENT-3 trial. COBENFY demonstrated sustained improvement in schizophrenia symptoms across various measures, including the Positive and Negative Syndrome Scale (PANSS) total score, Clinical Global Impression-Severity (CGI-S) score, and PANSS positive and negative subscale scores. Notably, participants who initially received placebo in the earlier trials experienced rapid symptom improvement upon starting COBENFY. By the study’s conclusion, 69% of participants achieved a 30% or greater improvement in PANSS total score from their initial baseline. COBENFY’s long-term use was generally well-tolerated, with no new safety or tolerability concerns identified. The most frequent treatment-emergent adverse events (TEAEs) included nausea, vomiting, dyspepsia, dry mouth, and hypertension, which were typically mild to moderate and resolved without requiring treatment discontinuation. The overall discontinuation rate due to TEAEs was 11%. Furthermore, COBENFY did not show clinically significant changes in body weight, prolactin levels, or movement disorder scale scores.
The EMERGENT-5 trial, also a 52-week open-label study, evaluated COBENFY in 566 adults with schizophrenia in the U.S. These participants had stable symptoms on a previous antipsychotic and no prior COBENFY exposure. The trial included individuals with mild to moderate illness based on PANSS and CGI-S scores. COBENFY demonstrated improvements across all efficacy measures, including PANSS total, CGI-S, and PANSS subscale scores, confirming its sustained effectiveness. By week 52, 30% of participants achieved a 30% or greater reduction in PANSS total score from baseline. Long-term COBENFY treatment was generally well-tolerated in this trial as well, with no new safety or tolerability issues arising. The most frequent TEAEs were similar to those observed in EMERGENT-4, mostly mild to moderate in intensity, and did not generally lead to treatment discontinuation. The discontinuation rate due to TEAEs in EMERGENT-5 was 18%. Similar to EMERGENT-4, COBENFY was not associated with significant changes in body weight, movement disorder scales, or prolactin levels.
A qualitative interview-based survey was conducted within the EMERGENT-5 trial to assess patient-reported experiences and perceived changes in Quality of Life (QoL) with COBENFY treatment. Interviews were conducted at six weeks and six months after starting COBENFY. At baseline, most participants reported negative QoL impacts across physical, social, emotional, and role functioning domains. A significant majority reported improvements in at least one QoL domain within six weeks of starting COBENFY, with high satisfaction levels maintained through six months. Participants attributed their satisfaction to perceived symptom improvement, enhanced QoL, and minimal treatment burden. A vast majority of participants at the six-month mark indicated they would recommend COBENFY and would choose to continue treatment if given the opportunity.
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### [Scholar Rock to Present New Data from Phase 1 DRAGON Trial at SITC](https://www.clinicaltrialvanguard.com/news/scholar-rock-to-present-new-data-from-phase-1-dragon-trial-at-sitc/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
Scholar Rock, a late-stage biopharmaceutical company specializing in treatments for serious diseases like spinal muscular atrophy (SMA), cardiometabolic disorders, and cancer, will present data from its Phase 1 DRAGON trial of SRK-181 at the Society for Immunotherapy of Cancer’s (SITC) 39th Annual Meeting. The presentation, scheduled for November 9th, 2024, will focus on updated safety, efficacy, and biomarker results for SRK-181 in patients with advanced solid tumors resistant to anti-PD-1 therapy. This data comes from the expansion phase (Part B) of the DRAGON trial. The presentation, titled “DRAGON Trial: Durable remission rate with the latent TGFβ1 inhibitor linavonkibart (SRK-181) and [pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) in patients with immune checkpoint inhibitor-resistant advanced cancers,” will be delivered by Dr. Timothy A. Yap of The University of Texas MD Anderson Cancer Center. Abstracts will be available on the SITC website on November 5th, 2024, and the full presentation will be accessible on Scholar Rock’s website after the conference concludes.
SRK-181 is a selective inhibitor of TGFβ1 activation, designed to combat primary resistance to checkpoint inhibitor therapies, such as anti-PD-(L)1 antibodies, in advanced cancers. TGFβ1, the predominant TGFβ isoform in many human tumors, is implicated in creating an immunosuppressive tumor microenvironment. This environment restricts the entry and activity of cytotoxic T cells, hindering anti-tumor immunity. SRK-181 specifically targets latent TGFβ1 to inhibit its activity within the tumor microenvironment comprehensively. This approach aims to reverse the immunosuppressive effects, promote tumor regression with anti-PD-(L)1 therapy, and potentially minimize the toxicities linked to non-selective TGFβ inhibition.
The DRAGON trial (NCT04291079) completed enrollment in December 2023, but treatment continues for the remaining participants. The trial included multiple parallel cohorts focusing on various cancer types, including urothelial carcinoma, cutaneous melanoma, [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/), head and neck squamous cell carcinoma, and clear cell renal cell carcinoma. It is important to note that SRK-181 is currently an investigational product candidate, and the FDA or any other regulatory agency has not yet established its efficacy and safety.
Scholar Rock is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for serious diseases with unmet needs. The company focuses on the transforming growth factor beta (TGFβ) superfamily of cell proteins, leveraging its proprietary platform to develop selective monoclonal antibodies that modulate protein growth factors. Scholar Rock’s pipeline targets neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor modulation could offer significant therapeutic benefits. The company regularly communicates with investors and the public via its website, SEC filings, press releases, and social media channels. It encourages interested parties to review these resources for updates regularly.
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**Categories:** News
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### [Sunbird Bio's Blood Test Accurately Diagnoses Parkinson's](https://www.clinicaltrialvanguard.com/news/sunbird-bios-blood-test-accurately-diagnoses-parkinsons/)
**Published:** November 1, 2024
**Author:** Jon Napitupulu
**Content:**
Sunbird Bio, a biotechnology company focused on neurological disorders and early-stage cancer diagnostics, has unveiled promising data regarding its blood-based alpha-synuclein (α-synuclein) test. This test accurately detects aggregated α-synuclein in the brain through a simple blood draw, potentially offering a groundbreaking diagnostic tool for [Parkinson](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-genetics-are-global-your-enrollment-plan-isnt/)’s disease and other neurodegenerative conditions. These findings were presented at the 2024 Clinical Trials on Alzheimer’s Disease (CTAD) conference.
The aggregation of α-synuclein proteins in the brain is a key indicator of several neurodegenerative diseases, particularly Parkinson’s disease, dementia with Lewy bodies, and multiple system atrophy. Currently, diagnosing Parkinson’s disease relies on evaluating symptoms, medical history, and physical examinations, lacking a specific blood-based test. Sunbird Bio’s research uses extracellular vesicle (EV)-bound α-synuclein in blood as a direct measure of α-synuclein aggregation in the brain.
The study involved blood samples from 16 individuals with Parkinson’s disease and 24 healthy, age-matched individuals—Sunbird’s proprietary assays distinguished between EV-bound and unbound soluble forms of α-synuclein in plasma. The results indicated that unbound, soluble α-synuclein was ineffective in identifying Parkinson’s disease. However, the signature based on brain-derived, EV-bound α-synuclein demonstrated high accuracy, achieving an area under the curve (AUC) of 0.86. This suggests a strong potential for accurate disease detection.
This technology holds promise beyond Parkinson’s disease, potentially impacting other neurological conditions, such as Alzheimer’s disease, which also exhibit α-synuclein aggregation. Aggregated α-synuclein disrupts cellular function, impacting synaptic transmission, triggering neuroinflammation, and potentially leading to neuronal death. These aggregates bind to EVs in the brain, crossing the blood-brain barrier and entering the bloodstream. Sunbird Bio’s technology uniquely addresses the challenge of accurately detecting these aggregated proteins in the blood.
This technology could revolutionize the diagnosis of Parkinson’s and other synuclein-related neurological disorders. Further clinical trials with larger sample sizes and additional biomarkers are planned to validate these initial findings and explore the technology’s broader applications. Sunbird Bio’s diagnostic platform directly detects and measures low concentrations of EV-bound, aggregated proteins, potentially impacting disease detection, drug development, disease monitoring, and personalized treatment selection for various neurological disorders. The company is developing a range of blood-based tests targeting biomarkers like amyloid beta, tau, α-synuclein, and [TDP-43](https://www.clinicaltrialvanguard.com/news/new-tdp-43-pet-tracer-a-breakthrough-for-precision-medicine/) for neurological diseases.
Source link:[ http://www.businesswire.com/news/home/20241031668102/en/Sunbird-Bio-Presents-New-Clinical-Data-Demonstrating-Proprietary-Alpha-Synuclein-Blood-Based-Biomarkers-Could-Accurately-Diagnose-Parkinson%E2%80%99s-Disease](http://www.businesswire.com/news/home/20241031668102/en/Sunbird-Bio-Presents-New-Clinical-Data-Demonstrating-Proprietary-Alpha-Synuclein-Blood-Based-Biomarkers-Could-Accurately-Diagnose-Parkinson%E2%80%99s-Disease)
**Categories:** News
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### [Mood Bloom™ Delivers ROI in New Economic Study](https://www.clinicaltrialvanguard.com/news/mood-bloom-delivers-roi-in-new-economic-study/)
**Published:** November 4, 2024
**Author:** Jon Napitupulu
**Content:**
Hedonia, a mobile [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) solutions provider, has released promising findings from a recent economic model. The study, conducted in collaboration with Arbital Health, indicates a potential 5:1 return on investment (ROI) for employers, payers, and healthcare providers utilizing Hedonia’s digital therapeutic app, Mood Bloom. Designed to treat depression and anxiety, Mood Bloom leverages a unique therapeutic gaming approach called Facilitating Thought Progression (FTP).
This approach embeds simple exercises within an engaging game environment to establish new mental habits that mitigate depressive symptoms. The high engagement rates achieved through this gamified approach contribute significantly to the app’s impressive ROI potential. Data from clinical trials at Massachusetts General Hospital, which served as the basis for the economic model, revealed a substantial 45% reduction in depressive symptoms among Mood Bloom users. This clinical efficacy translates into significant cost savings. For instance, a company with 20,000 employees could save over half a million dollars annually by offering Mood Bloom to its workforce.
Arbital Health’s economic model calculates the ROI by assessing the financial savings associated with improved mental health outcomes achieved through Mood Bloom. The model demonstrates that employers can significantly reduce costs by implementing Hedonia’s program. The program’s effectiveness lies in its ability to help individuals transition from more severe to less severe mental health states, thereby decreasing overall treatment expenses.
The model, built upon peer-reviewed outcomes data and clinical findings, offers a customizable ROI calculator tailored to diverse employer populations. This allows employers to directly visualize the potential cost savings associated with Mood Bloom implementation. The study, drawing on the 2022 MarketScan Database, analyzed cost patterns among individuals diagnosed with depression and anxiety. It examined Mood Bloom’s financial impact using detailed cost estimates from major U.S. employers, showcasing the program’s ability to improve mental health while significantly reducing associated costs.
Beyond direct medical cost reductions, mitigating depression and anxiety symptoms yields additional benefits, such as improved productivity. Research indicates a correlation between the severity of depression and productivity loss, ranging from 29% for mild depression to 51% for severe depression. By facilitating a transition to less severe mental health states, Mood Bloom contributes to enhanced productivity. As mental health disorders continue to rise, Hedonia’s Mood Bloom presents a scalable, effective, and financially viable solution for employers seeking to enhance employee well-being while managing healthcare expenditures. Hedonia specializes in evidence-based mobile mental health tools. Their flagship product, Mood Bloom, combines mental health treatment with game design to improve engagement and outcomes. Arbital Health focuses on managing the complexities of value-based contract design, measurement, and adjudication, aiming to facilitate the transition to value-based care in the U.S. healthcare system.
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**Categories:** News
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### [CINFINA Pharma Unveils Promising Phase 1 Obesity Drug Data at ObesityWeek® 2024](https://www.clinicaltrialvanguard.com/news/cinfina-pharma-unveils-promising-phase-1-obesity-drug-data-at-obesityweek-2024/)
**Published:** November 4, 2024
**Author:** Jon Napitupulu
**Content:**
CinFina Pharma, a CinRx portfolio company focused on [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatments, has released promising interim data from a Phase 1 single ascending dose (SAD) study of CIN-110 and final data from a multiple ascending dose (MAD) study of CIN-109. Both treatments demonstrated tolerability and significant weight loss.
CIN-110, a long-acting PYY3-36 analog, is designed to minimize gastrointestinal side effects often associated with subcutaneous administration. Interim data from the double-blind Phase 1 SAD study indicated the treatment was well-tolerated in obese participants. It showed encouraging reductions in caloric intake and subsequent weight loss compared to a placebo after a single dose. Within one week, food intake decreased by up to 28%, leading to a weight loss of up to 1.8%. The study involved 24 obese participants with an average baseline weight of approximately 102 kg and a body mass index (BMI) of approximately 34 kg/m2. CIN-110 is engineered to gradually increase and sustain drug exposure, mitigating common gastrointestinal side effects. Three mild nausea cases were reported, typically resolving within a day. Pharmacokinetic data revealed a gradual increase in CIN-110 levels, peaking within two to three days and sustaining with a roughly 14-day half-life. No participants withdrew due to adverse events, and all side effects were mild or moderate, with moderate events unrelated to the digestive system.
CIN-109, a long-acting, first-in-class [GDF-15](https://www.clinicaltrialvanguard.com/news/visugromab-reverses-checkpoint-inhibitor-resistance/) analog, aims to reduce appetite, maintain energy expenditure, and promote weight loss while preserving lean body mass. Results from the Phase 1 MAD study showed it was well-tolerated and resulted in substantial weight loss in obese participants. In this randomized, double-blind, placebo-controlled study, participants received weekly doses of CIN-109 (5 mg, 10 mg, 15 mg, 20 mg, or 40 mg) or bi-weekly doses (20 mg, 40 mg, or 60 mg). The weekly group received treatment for four weeks, while the bi-weekly group received treatment for eight weeks. Participants had an average weight of approximately 100 kg and a BMI of approximately 35 kg/m2. Participants experienced reduced food intake, with dose-dependent decreases of up to 50%, and resulting weight loss of up to 3.7% within one to two months. Bi-weekly dosing proved to be better tolerated, and the majority of weight loss was attributed to fat mass reduction. No serious treatment-related side effects were observed.
CIN-110 is a long-acting PYY3-36 analog designed to minimize nausea and vomiting often associated with other PYY molecules while promoting weight loss. It is currently undergoing clinical trials to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity.
CIN-109 is a Phase 2 ready, first-in-class GDF-15 analog for obesity treatment. It has successfully completed a randomized, double-blind, placebo-controlled MAD study assessing its safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity when administered subcutaneously.
CinFina Pharma, part of the CinRx portfolio, is dedicated to expanding obesity treatment options. It is developing a pipeline of therapeutic candidates intended to be safe, tolerable, and long-lasting to support weight loss and overall health improvement. CinFina’s four therapeutic candidates are naturally occurring, engineered peptides designed to regulate insulin secretion and satiety signals.
CinRx Pharma employs a unique hub-and-spoke business model to advance a diverse portfolio of medicines through clinical development. This approach combines financing with efficient therapeutic candidate progression, managed by CinRx’s central infrastructure and operations team. CinRx focuses on areas of high unmet medical need, including metabolic, gastrointestinal, and oncological conditions.
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**Categories:** News
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### [MedinCell & Teva: Olanzapine LAI Phase 3 Positive, Uzedy Real-World Data](https://www.clinicaltrialvanguard.com/news/medincell-teva-olanzapine-lai-phase-3-positive-uzedy-real-world-data/)
**Published:** November 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Medincell](https://www.clinicaltrialvanguard.com/news/medincell-partner-teva-delivers-unprecedented-antipsychotic-data/)‘s long-acting injectable technology, BEPO®, has achieved commercial success with its first product, UZEDY® (risperidone LAI), for treating [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) in adults. Approved by the FDA in April 2023 and launched by Teva the following month, UZEDY demonstrates high adherence rates, particularly among patients with unmet social needs, according to real-world data analysis. This analysis examined US claims data from 715 adult schizophrenia patients treated with UZEDY, focusing on social determinants of health and adherence patterns.
A separate investigational drug, TEV-‘749 / mdc-TJK, is a once-monthly subcutaneous olanzapine LAI, also for schizophrenia. Phase 3 SOLARIS study results indicate its potential to be the first long-acting olanzapine with a favorable safety profile, addressing a key limitation of existing olanzapine LAIs. The 8-week, double-blind, placebo-controlled Period 1 of the SOLARIS trial, involving patients aged 18-64, showed TEV-‘749 significantly improved social functioning and quality of life across all three doses compared to placebo. This was followed by a 48-week open-label safety period (Period 2). The primary endpoint, a statistically significant change in Positive and Negative Syndrome Scale (PANSS) total scores from baseline to week 8, was met across all dosing groups. Importantly, the systemic safety profile aligned with approved oral olanzapine formulations, with no new safety signals or Post-Injection Delirium/Sedation Syndrome (PDSS) events.
Medincell’s BEPO® technology enables controlled drug delivery at therapeutic levels for extended periods from a single, bioresorbable subcutaneous injection. This addresses compliance, efficacy, and environmental impact of treatments. Partnering with Teva, Medincell receives royalties on net sales and milestone payments for the development and commercialization of these products. Teva leads the clinical development and regulatory processes.
Medincell, a clinical and commercial-stage biopharmaceutical licensing company, develops long-acting injectables across various therapeutic areas. Their innovative approach aims to improve medication adherence, treatment effectiveness, and accessibility while minimizing environmental impact. Headquartered in Montpellier, France, Medincell employs over 140 individuals representing more than 25 nationalities.
This press release includes forward-looking statements concerning clinical trials, product benefits, regulatory approvals, commercialization, future product portfolio, partnerships, capital needs, and financial matters. While based on reasonable assumptions, these statements are subject to inherent risks and uncertainties. Factors beyond the company’s control and financial capabilities could cause actual results to differ materially. Detailed information on these risks can be found in documents filed with the Autorité des Marchés Financiers (AMF), including the company’s registration document. These forward-looking statements reflect the company’s current perspective and are subject to change. This press release is for informational purposes only and does not constitute an offer to sell or a solicitation to buy shares. It should not be considered investment advice.
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**Categories:** News
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### [Scholar Rock Announces Preclinical Data for SRK-439](https://www.clinicaltrialvanguard.com/news/scholar-rock-announces-preclinical-data-for-srk-439/)
**Published:** November 5, 2024
**Author:** Jon Napitupulu
**Content:**
Scholar Rock, a late-stage biopharmaceutical company, has released promising preclinical data for [SRK-439](https://www.clinicaltrialvanguard.com/news/scholar-rock-unveils-remarkable-srk-439-findings-preserving-lean-mass-curbing-fat-gain/), an investigational anti-myostatin antibody. The data suggest SRK-439 can increase lean mass and decrease fat mass gain when combined with metformin. These findings were presented at the [ObesityWeek](https://www.clinicaltrialvanguard.com/news/cinfina-pharma-unveils-promising-phase-1-obesity-drug-data-at-obesityweek-2024/) conference in San Antonio, Texas.
The preclinical study utilized a diet-induced obesity mouse model. Mice received a high-fat diet, followed by either metformin or a control. Subsequently, they received either SRK-439 or a control antibody. This process was repeated in both young and mature mice to assess age-related effects. Quantitative nuclear magnetic resonance (qNMR) measured lean mass changes throughout the study.
Results showed a substantial increase in lean mass in the group receiving both SRK-439 and metformin compared to the metformin-only group. This positive effect was observed across both age groups. Furthermore, in younger mice, the combination of SRK-439 and metformin resulted in a greater reduction in fat mass gain compared to either treatment alone. These results highlight SRK-439’s potential to improve body composition and support healthier weight management, particularly for individuals with obesity and [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/).
Scholar Rock’s anti-myostatin portfolio includes both SRK-439 and apitegromab. Apitegromab is currently being evaluated in the Phase 2 EMBRAZE trial for adults with a BMI over 27 (overweight) or over 30 (obese) who are also taking a GLP-1 receptor agonist (tirzepatide or semaglutide). The trial aims to enroll 100 non-diabetic participants aged 18-65. Subjects will receive either apitegromab or a placebo alongside a GLP-1 receptor agonist for 24 weeks. The primary endpoint is the change in lean mass, measured by dual-energy X-ray absorptiometry. Secondary endpoints include additional weight loss metrics, safety, tolerability, and pharmacokinetic outcomes. Exploratory endpoints involve cardiometabolic parameters, body composition, and physical function assessments at weeks 24 and 32.
SRK-439 is a preclinical, investigational myostatin inhibitor. It exhibits high affinity for pro- and latent myostatin with selectivity for myostatin, meaning it does not bind to GDF11 or Activin-A. It is being developed for cardiometabolic disorders, including obesity. Preclinical data suggest SRK-439 may preserve lean mass during weight loss, contributing to healthier weight management. However, its efficacy and safety in humans have not yet been established, and it has not received FDA approval.
Apitegromab, another Scholar Rock therapy, is a monoclonal antibody that inhibits myostatin activation by binding to its pro- and latent forms in skeletal muscle. It is the first muscle-targeted therapy to demonstrate clinical proof-of-concept in spinal muscular atrophy (SMA). Myostatin’s absence is linked to increased muscle mass and strength. Scholar Rock believes apitegromab’s selective targeting of myostatin may improve motor function in SMA patients. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Priority Medicines (PRIME) and Orphan Medicinal Product designations from the EMA. However, it has not yet been approved for any use.
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**Categories:** News
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### [Allurion's Obesity Data from 19,428 Patients Revealed](https://www.clinicaltrialvanguard.com/news/allurions-obesity-data-from-19428-patients-revealed/)
**Published:** November 5, 2024
**Author:** Jon Napitupulu
**Content:**
Allurion Technologies, a company focused on combating [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), presented two abstracts at Obesity Week 2024. The first abstract showcased real-world data from their largest cohort to date. Data collected from 19,428 patients across 72 countries between 2018 and 2023, utilizing the Allurion Program and tracked via the Allurion Virtual Care Suite, demonstrated an average total body weight loss of 12.2% at four months. This large-scale, real-world data reinforces the clinical significance of the Allurion Program’s weight loss results across a diverse population, complementing previously published data in over 26 peer-reviewed publications.
The second abstract focused on the Allurion Program’s effectiveness for individuals unresponsive to liraglutide, a GLP-1 medication. The study included 27 patients, of whom 55% experienced no weight loss and 45% lost less than 5% body weight while using GLP-1 medications. Remarkably, these same patients achieved an average weight loss of 17.6% after just four months with the Allurion Program. This finding is particularly significant given that a substantial portion of GLP-1 patients do not achieve significant weight loss, and it can take several months to determine treatment efficacy. The Allurion Program may offer a valuable alternative for this patient population.
Allurion’s weight loss platform features the Allurion Gastric Balloon, a swallowable, procedure-less intragastric balloon, coupled with the Allurion Virtual Care Suite. This suite includes the Allurion Mobile App for consumers, Allurion Insights with the Coach Iris AI Platform for healthcare providers, and the Allurion Connected Scale. The Virtual Care Suite is also available independently from the Allurion Program, enabling providers to personalize and manage weight loss therapies for patients, regardless of the specific treatment plan—whether it involves a gastric balloon, surgery, medication, or nutritional guidance. The Allurion Gastric Balloon is currently an investigational device in the United States.
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**Categories:** News
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### [Vertex Reports Strong Q3 2024 Financial Results](https://www.clinicaltrialvanguard.com/news/vertex-reports-strong-q3-2024-financial-results/)
**Published:** November 5, 2024
**Author:** Jon Napitupulu
**Content:**
Vertex Pharmaceuticals reported strong third-quarter 2024 financial results and raised its full-year product revenue guidance. The company anticipates continued growth driven by existing products and upcoming launches.
Third-quarter product revenue reached $2.77 billion, a 12% increase compared to the same period in 2023. This growth was primarily fueled by the consistent performance of TRIKAFTA/KAFTRIO. U.S. net product revenue saw a 10% increase to $1.71 billion, while revenue outside the U.S. grew by 14% to $1.06 billion. Increased investment in research and development (R&D), sales, general, and administrative (SG&A) expenses reflect both global launch support for current therapies and ongoing investment in advancing pipeline programs to Phase 3 clinical development. These expenses totaled $1.2 billion (GAAP) and $1.1 billion (Non-GAAP). Acquired in-process R&D (AIPR&D) expenses decreased to $15 million, compared to $52 million in Q3 2023. Both GAAP and Non-GAAP net income were stable at approximately $1 billion for both Q3 2024 and Q3 2023.
The company’s cash, cash equivalents, and marketable securities totaled $11.2 billion at the end of the third quarter, down from $13.7 billion at the end of 2023. This decrease is attributed to the acquisition of Alpine Immune Sciences and share repurchases, partially offset by positive cash flow from operations. Vertex raised its full-year 2024 product revenue guidance to between $10.8 billion and $10.9 billion, reflecting anticipated growth in [cystic fibrosis](https://www.clinicaltrialvanguard.com/news/infexs-resp-x-shows-exacerbation-reduction-in-bronchiectasis-study/) (CF) treatment and the launch of CASGEVY. The company expects combined Non-GAAP R&D and SG&A expenses to remain in the range of $4.2 billion to $4.3 billion. Full-year AIPR&D expenses are anticipated to be approximately $4.6 billion, including a $4.4 billion charge related to the Alpine acquisition.
Vertex anticipates continued growth in the number of CF patients using its medications, driven by new approvals and expanded reimbursement coverage for younger patients. The company also highlighted the ongoing launch of CASGEVY, a gene-editing therapy for [sickle cell](https://www.clinicaltrialvanguard.com/news/mitapivat-met-hemoglobin-goal-in-sickle-cell-phase-3-but-only-40-6-of-patients-responded/) disease (SCD) and transfusion-dependent beta thalassemia (TDT). CASGEVY has received approvals in several countries, including the U.S., U.K., EU, and Canada. Vertex is also preparing for the potential launch of suzetrigine (VX-548), a novel treatment for moderate-to-severe acute pain. Suzetrigine represents a new class of pain medication, offering potential relief without the drawbacks of opioids.
Vertex’s pipeline continues to advance, with three programs progressing to Phase 3: suzetrigine for diabetic peripheral neuropathy, povetacicept for IgA nephropathy, and VX-880 for [Type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/). The company remains committed to developing innovative treatments for serious diseases. Current research efforts include next-generation CFTR modulators for CF, advancements in SCD and TDT therapies, and the development of novel pain medications. Further areas of focus include APOL1-mediated kidney disease, additional B cell-mediated diseases, stem cell-derived islet cell therapies for Type 1 diabetes, myotonic dystrophy Type 1, and autosomal dominant polycystic kidney disease.
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**Categories:** News
---
### [Enveric Biosciences: Broad Range of Patents Issued](https://www.clinicaltrialvanguard.com/news/enveric-biosciences-broad-range-of-patents-issued/)
**Published:** November 5, 2024
**Author:** Jon Napitupulu
**Content:**
Enveric Biosciences, a biotechnology company focused on developing novel neuroplastogens for neuropsychiatric disorders, has significantly expanded its patent protection. Five new US patents have been issued, and three more applications have been allowed by the United States Patent and Trademark Office, bolstering the intellectual property surrounding the company’s EVM301 portfolio. This portfolio comprises a wide array of tryptamine derivative molecules, including carboxylated, aminated, prenylated, glycosylated, nitrilated, and halogenated variations, as well as combinations of these functional motifs. These additions bring the total number of issued US patents protecting the EVM301 portfolio and its associated drug candidate library to nine.
This expanded patent protection enhances the potential value of Enveric’s drug candidate library. The company’s proprietary Psybrary™ contains over 1,000 tryptamine derivative molecules, incorporating receptor engagement and functional data to link molecular structure with biological activity. This broader portfolio with robust intellectual property protection creates more value-capture opportunities for Enveric. The portfolio not only increases the number and types of compounds available for development but also expands the range of potential therapeutic indications.
Furthermore, Enveric’s drug discovery platform has identified molecules exhibiting a preference for 5-HT2C receptors. These molecules, also protected by issued patent claims, hold promise as potential antiepileptic and anti-[obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) drug candidates. They demonstrate enhanced binding preference for the 5-HT2C receptor compared to other serotonergic, dopaminergic, and adrenergic receptor targets.
The lead candidate from the EVM301 series, EB-003, is currently in preclinical development. This compound is being investigated for major undertreated [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions, including treatment-resistant depression and anxiety, with plans to file an Investigational New Drug (IND) application in 2025. Preclinical data indicates a favorable safety profile for EB-003. Receptor engagement data suggests EB-003 can promote neuroplasticity without inducing hallucinations, a significant safety advantage over existing treatments. This characteristic is crucial for broader patient access, as hallucinations are a major concern limiting the use of some current therapies. Enveric, headquartered in Naples, FL, also maintains offices in Cambridge, MA, and Calgary, AB, Canada. The company’s focus is developing novel small-molecule therapeutics for depression, anxiety, and addiction disorders, leveraging its Psybrary™ platform to create new chemical entities for specific mental health indications.
Source link: http://www.businesswire.com/news/home/20241104428250/en/Enveric-Biosciences-Announces-Broad-Range-of-Patent-Issuances
**Categories:** News
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### [Immunis Studies Canine Muscle Atrophy](https://www.clinicaltrialvanguard.com/news/immunis-studies-canine-muscle-atrophy/)
**Published:** November 5, 2024
**Author:** Jon Napitupulu
**Content:**
Immunis, a clinical-stage biotech company, has launched non-terminal safety studies for canine muscle atrophy in partnership with VetBio Partners. This STEM-K9 program builds upon preclinical success observed in aged mouse models using Immunis’ investigational secretome ([IMM01](https://www.clinicaltrialvanguard.com/news/immunis-achieves-breakthrough-in-muscle-atrophy-trial/)-STEM) and complements ongoing human clinical trials for STEM-MYO and STEM-META programs. The company aims to improve mobility and quality of life for canines, paralleling their human-focused research.
VetBio Partners, led by Dr. Peter Canning, brings over three decades of veterinary pharmaceutical experience to the collaboration. Dr. Canning’s expertise will be instrumental in guiding Immunis through the preclinical trials. The increasing importance of pets within families has driven demand for improved animal healthcare, particularly for age-related conditions.
Canine muscle atrophy, similar to its human counterpart, arises from aging, inactivity, and underlying health issues. Despite a clear link between age and decreased muscle mass in dogs, research in this area remains limited. Muscle loss also contributes to serious conditions like congestive heart failure, chronic kidney disease, cancer, and [osteoarthritis](https://www.clinicaltrialvanguard.com/news/nih-funds-trial-for-non-surgical-knee-osteoarthritis-relief/) in canines.
Immunis emphasizes that the animal studies adhere to the same rigorous standards employed in their human clinical trials. The non-terminal design of the study prioritizes the ethical treatment of the canine participants. This expansion into animal health demonstrates Immunis’ commitment to applying their innovative therapies across species, aiming to address age-related ailments in both humans and companion animals.
VetBio Partners offers research and development consulting services, specializing in bridging human health biopharmaceutical technologies to animal health applications. Their extensive network and experience in drug discovery and development facilitate the efficient evaluation of new technologies for efficacy and safety. They also provide business development support through connections within the animal health pharmaceutical industry.
Immunis focuses on developing an immunomodulatory secretome product targeting age and disease-related immune decline. Their investigational product utilizes secretome technology to deliver a natural, human-derived blend of immune modulators in physiologically relevant concentrations.
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**Categories:** News
---
### [How Johnson & Johnson is Innovating its Organizational Infrastructure](https://www.clinicaltrialvanguard.com/conference-coverage/how-johnson-johnson-is-innovating-its-organizational-infrastructure/)
**Published:** November 5, 2024
**Author:** Moe Alsumidaie
**Content:**
At [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) 2024, Johnson & Johnson’s representatives, Gordon Gregory III, and Laszlo Vasko, unveiled their transformative “Team of Teams” approach to clinical development. This innovative strategy, supported by advanced automation and integrated workflows, aims to revolutionize the development of oncology compounds. By addressing the inefficiencies of traditional communication methods, Johnson & Johnson seeks to enhance collaboration, streamline processes, and accelerate the delivery of new therapies. The presentation highlighted the potential of this approach to improve clinical trial efficiency and foster a culture of innovation and engagement within the organization.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#the-team-of-teams-concept)The Team of Teams Concept
Johnson & Johnson’s “Team of Teams” concept is a strategic response to the complexities and inefficiencies inherent in traditional clinical development processes. The approach addresses the challenges posed by the highly matrixed nature of drug development, which involves numerous functions, teams, partners, and suppliers inside and outside the organization. Traditional electronic methods of communication, such as emails and file repositories, often lead to bottlenecks, excessive meetings, and fragmented information sharing. Laszlo Vasko, Senior Director at Johnson & Johnson Innovative Medicines, explained that the “Team of Teams” framework is not based on any singular organizational strategy or innovation like AI. Instead, it focuses on enabling therapy development teams to collaborate, communicate, and make decisions more effectively and efficiently. This approach aims to transcend the limitations of organizational silos and individual capabilities by fostering a culture of open collaboration.
The goal is to democratize knowledge, streamline workflows, and simplify work processes, allowing teams to work asynchronously and in real-time. Johnson & Johnson seeks to improve how new therapies are delivered by focusing on opportunities that are not tied to organizational structure or individual capabilities. This involves enhancing collaboration with external partners, managing complex trackers, and enabling teams to be effective from day one.
## [](#technological-backbone-slack-enterprise-grid)Technological Backbone: Slack Enterprise Grid
The technological backbone of Johnson & Johnson’s “Team of Teams” approach is the Slack Enterprise Grid, a robust multi-channel collaboration platform. This platform, provided by Salesforce, has been customized to meet the specific needs of Johnson & Johnson, ensuring secure and compliant communication across the organization. Vasko highlighted that Slack’s granular security capabilities allow the company to bring in external partners, including competitive partners, to collaborate seamlessly on drug development projects. The implementation of Slack Enterprise Grid has enabled Johnson & Johnson to support its matrix organizational model, which is essential for the complex nature of drug development. The platform facilitates the integration of critical systems and automates workflows, empowering teams to build and utilize this automation themselves.
This integration extends to working with external partners, ensuring all stakeholders are aligned and informed throughout development. The platform’s ability to support the matrix organizational model is crucial for the complex nature of drug development, allowing for seamless collaboration and communication. By integrating key systems and automating workflows, Slack Enterprise Grid empowers teams to build and utilize these automations themselves. The platform’s secure and compliant communication capabilities ensure that all stakeholders, including external partners, are aligned and informed throughout the development process.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#real-world-applications-and-measured-value)Real-World Applications and Measured Value
Gordon Gregory, a Global Trial Leader in Oncology at Johnson & Johnson, provided detailed examples of how the “Team of Teams” approach is applied in real-world applications. The Cross-Functional Trial Team (CFTT) channel is one of the most significant applications. This channel serves as the central hub for all communication and collaboration related to a clinical trial. Gregory explained that the CFTT channel consolidates all critical information, such as team roles, critical systems, and documents, into a single, accessible location. This setup allows team members to quickly get up to speed and make informed decisions without the need for constant meetings or email exchanges. The CFTT channel facilitates asynchronous decision-making, enabling teams to make decisions outside of scheduled meetings. This is achieved through templates and automation, which streamline standard processes like collecting agenda items and sharing meeting minutes.
As a result, the trial teams have exceeded their study startup milestones, demonstrating the effectiveness of this collaborative approach. Another example of the “Team of Teams” approach is the data cleaning channel. This channel brings together central teams, local teams, and data managers to collaborate on data-cleaning efforts. The channel has significantly reduced outstanding queries and data cleaning timelines by standardizing and automating the sharing of updates and reports. Some trials have even been able to pull in their data cleaning milestones by one to two months, highlighting the tangible benefits of this transparent and collaborative work environment. These real-world applications demonstrate the potential of the “Team of Teams” approach to transform clinical development and accelerate timelines.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#automation-and-innovation)Automation and Innovation
Automation is a cornerstone of Johnson & Johnson’s “Team of Teams” approach, driving efficiency and innovation across clinical trials. Gregory shared an example of how automation has transformed the management of FAQs within clinical trials. Previously, questions were shared via email, leading to a cumbersome process of manually updating FAQ logs. With the automated system, questions are submitted through a designed form, triaged by trial managers, and escalated to relevant functions using simple emoji-based triggers. Once answered, the system automatically updates the FAQ log, eliminating the need for manual intervention. This automation streamlines the process and encourages team members to search for existing answers before submitting new questions.
By reducing the volume of repetitive questions, the team can focus on more critical tasks, further enhancing productivity. The intentional design of these automated processes reflects Johnson & Johnson’s commitment to fostering a culture of innovation and continuous improvement. Employees are more motivated and invested in their work by empowering teams to automate and innovate their workflows. This shift towards more human-centric work practices has increased job satisfaction and a greater sense of ownership among employees. The cultural transformation brought about by the “Team of Teams” approach is expected to impact Johnson & Johnson’s portfolio delivery significantly.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#summary)Summary
In conclusion, Johnson & Johnson’s “Team of Teams” approach, supported by Slack Enterprise Grid, represents a significant advancement in clinical development. By fostering open collaboration and leveraging automation, the company is poised to accelerate the delivery of new therapies, particularly in the oncology sector. The presentation highlighted the potential for this model to transform the way clinical trials are conducted and the overall organizational culture. This innovative approach is expected to significantly impact Johnson & Johnson’s portfolio delivery, benefiting patients and the organization.
**Categories:** Article: Conference Coverage
---
### [Exact Sciences Q3 2024 Results: Surprising Growth](https://www.clinicaltrialvanguard.com/news/exact-sciences-q3-2024-results-surprising-growth/)
**Published:** November 6, 2024
**Author:** Jon Napitupulu
**Content:**
Exact Sciences Corp. (Nasdaq: EXAS), a prominent cancer screening and diagnostics company, reported third-quarter 2024 revenue of $709 million, a 13% increase compared to $628 million in the same quarter of 2023. This growth was driven by increased test volume, reaching more patients than in any previous quarter. However, the company acknowledged that third-quarter performance and updated full-year projections fall short of their potential. They anticipate accelerated growth in 2025, maintaining a positive long-term outlook.
The company’s financial results for the three months ending September 30, 2024, reveal a breakdown of revenue streams. Screening, primarily consisting of Cologuard tests and PreventionGenetics services, generated $544.9 million. Precision Oncology, including Oncotype DX and therapy selection tests, contributed $163.8 million. Despite the overall revenue growth, the company adjusted its full-year 2024 guidance downwards. Total revenue is now projected between $2.730 billion and $2.750 billion, down from the previous range of $2.810 billion to $2.850 billion. Similarly, adjusted EBITDA is expected between $310 million and $320 million, revised from $335 million to $355 million.
Exact Sciences highlighted advancements in its platform and pipeline. The ExactNexus™ technology platform connects patients, healthcare systems, professionals, and payers electronically, enabling personalized customer experiences and streamlined information access. This platform is credited with the significant growth in Cologuard test utilization among rescreened patients and within care gap programs during the third quarter.
The FDA recently approved the Cologuard Plus test, a next-generation version offering increased sensitivity in detecting cancers and precancerous polyps while significantly reducing false positives. The company anticipates a higher price for this improved test through an established Medicare pathway. Promising data for a blood-based [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) screening test were also presented, demonstrating an 88% sensitivity for colorectal cancer and 31% for advanced precancerous lesions at 90% specificity. This innovative approach potentially offers another screening option for average-risk patients at an attractive cost profile.
Furthermore, Exact Sciences secured acceptance for publication in a peer-reviewed journal regarding its Oncodetect test, a molecular residual disease and recurrence monitoring test. This publication is expected in January 2025 and addresses a significant unmet need for the nearly 6 million cancer survivors in the U.S. Finally, evidence supporting the company’s blood-based multi-cancer screening test demonstrated a 55% overall sensitivity for cancers lacking standard screening options (excluding lung cancer) and 64% for the six most aggressive cancers, with 98.5% specificity. This highlights the potential of multi-biomarker testing to detect a broader range of cancers.
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**Categories:** News
---
### [Acadia Sells Rare Pediatric Disease Voucher for $150M](https://www.clinicaltrialvanguard.com/news/acadia-sells-rare-pediatric-disease-voucher-for-150m/)
**Published:** November 6, 2024
**Author:** Jon Napitupulu
**Content:**
Acadia Pharmaceuticals has finalized an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million. The sale is expected to close after fulfilling standard closing conditions, including the waiting period mandated by the Hart-Scott Rodino (HSR) Antitrust Improvements Act. Acadia received this PRV in March 2023 following FDA approval of DAYBUE™ (trofinetide) for Rett syndrome treatment. DAYBUE, initially licensed from Neuren Pharmaceuticals Limited in August 2018, carries a contractual obligation for Acadia to remit one-third of the net proceeds to Neuren.
Acadia intends to allocate the PRV sale proceeds to bolster various aspects of its operations. This includes supporting commercial activities, advancing research and development programs focused on the central nervous system and rare diseases, and pursuing future business development opportunities. Jefferies LLC served as Acadia’s financial advisor for this transaction.
Acadia Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing innovative treatments for neurological disorders. The company is committed to delivering crucial therapies to those with significant unmet medical needs. They highlight their achievement of developing and commercializing the first and only FDA-approved treatment for hallucinations and delusions associated with [Parkinson](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-genetics-are-global-your-enrollment-plan-isnt/)’s disease psychosis. Additionally, they developed the first and only approved treatment for Rett syndrome in both the United States and Canada. Acadia’s current clinical development efforts are concentrated on Prader-Willi syndrome, Alzheimer’s disease psychosis, and other programs targeting neuropsychiatric symptoms in central nervous system disorders.
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**Categories:** News
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### [Alzheimer's Drugs Market to Hit $9.2B by 2030: Global Overview & CAGR](https://www.clinicaltrialvanguard.com/news/alzheimers-drugs-market-to-hit-9-2b-by-2030-global-overview-cagr/)
**Published:** November 6, 2024
**Author:** Jon Napitupulu
**Content:**
The global market for [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) drugs is poised for significant growth, projected to reach $9.2 billion by 2030, reflecting a robust 9.9% compound annual growth rate (CAGR) from its 2024 value of $5.2 billion. This expansion is primarily driven by the increasing prevalence of [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/), a consequence of the aging global population and extended life expectancies, particularly in developed nations. Innovations in drug development, including medications targeting the disease’s root causes and heightened awareness and earlier diagnoses, further contribute to market growth. Increased financial support for Alzheimer’s research from governmental and healthcare organizations, leading to accelerated drug development and regulatory approvals, also plays a crucial role.
Despite this positive trajectory, several factors impede market expansion. These include the high cost and complexity of drug development, frequent clinical trial failures, expiring patents, limited disease awareness in underdeveloped countries, and the high cost of treatments, which restricts patient access and market potential.
Regional analysis reveals North America as the current market leader, holding an estimated 38.9% share in 2024. This dominance stems from a high prevalence of Alzheimer’s, recent drug approvals, and sophisticated healthcare systems. The presence of major pharmaceutical companies conducting research and clinical trials, combined with an aging population, further solidifies North America’s leading position. However, the Asia Pacific region is anticipated to experience the fastest growth, projected at a CAGR of 11.5% between 2024 and 2030. This accelerated growth is attributed to the rising prevalence of Alzheimer’s, increasing awareness, and a focus on unmet medical needs, particularly in densely populated nations like China and India. Less stringent regulations in Asia Pacific make it an attractive hub for clinical trials and drug development.
Analyzing the market by drug class reveals cholinesterase inhibitors currently dominate, holding an estimated 42.3% share in 2024. Their effectiveness in treating mild-to-moderate Alzheimer’s by increasing acetylcholine levels, coupled with oral administration and proven efficacy, makes them a primary treatment option. However, other drug classes, including monoclonal antibodies, anti-amyloid beta, and pipeline drugs, are expected to experience the fastest CAGR of 11.9% from 2024-2030. This growth is driven by increasing regulatory approvals and the introduction of disease-modifying therapies targeting the underlying pathology of Alzheimer’s.
Regarding administration routes, oral drugs currently lead with an estimated 57% market share in 2024 due to their ease of use and non-invasive nature, which improves patient compliance. However, injectables, including monoclonal antibodies like Aducanumab and Leqembi, are projected to be the fastest-growing segment, with a CAGR of 11.7% from 2024 to 2030. These drugs offer potential disease-modifying effects for advanced stages of Alzheimer’s.
In terms of distribution channels, hospital pharmacies currently hold the largest market share at 47.7% in 2024. This is attributed to their role in procuring prescription drugs and their integration within comprehensive care models. Hospital pharmacies coordinate multidisciplinary approaches, encompassing pharmaceutical interventions, counseling, and community support. However, online pharmacies are anticipated to experience significant growth, with a CAGR of 12.7% between 2024 and 2030, driven by the increasing number of patients, the convenience of online purchasing, and growing internet usage and digital service adoption.
Source link: [http://www.businesswire.com/news/home/20241105720276/en/Alzheimers-Drugs-Global-Market-Overview-2024—9.9-CAGR-Forecast-During-2024-2030-with-Market-Set-to-Reach-a-Projected-US9.2-Billion-by-2030—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20241105720276/en/Alzheimers-Drugs-Global-Market-Overview-2024---9.9-CAGR-Forecast-During-2024-2030-with-Market-Set-to-Reach-a-Projected-US9.2-Billion-by-2030---ResearchAndMarkets.com)
**Categories:** News
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### [Viridian Therapeutics Grants Inducement Awards](https://www.clinicaltrialvanguard.com/news/viridian-therapeutics-grants-inducement-awards/)
**Published:** November 6, 2024
**Author:** Jon Napitupulu
**Content:**
Viridian Therapeutics, a Nasdaq-listed biopharmaceutical company (VRDN), granted stock options to six new employees on November 1, 2024. These options, totaling 150,600 shares of common stock, are considered inducement grants designed to attract these individuals to Viridian. While outside the company’s standard equity incentive plan, the grants adhere to its terms and conditions and comply with Nasdaq Listing Rule 5635(c)(4). The exercise price for these options matches Viridian’s common stock closing price on the grant date. Vesting occurs over four years, with an initial 25% vesting after one year of employment, followed by monthly vesting of the remaining shares over the subsequent three years, contingent on continued employment.
Viridian focuses on developing best-in-class medicines for individuals with severe and rare diseases. Their specialization lies in antibody discovery and protein engineering, allowing them to create differentiated therapies targeting validated drug targets within established disease areas. The company primarily focuses on treatments for thyroid eye disease (TED). Veligrotug (VRDN-001) is currently undergoing evaluation in two global Phase 3 clinical trials, [THRIVE](https://www.clinicaltrialvanguard.com/news/dbv-technologies-launches-thrive-trial-of-viaskin-peanut-patch-in-infants/) and THRIVE-2, assessing its efficacy and safety in patients with active and chronic TED.
Furthermore, Viridian is developing [VRDN-003](https://www.clinicaltrialvanguard.com/news/viridian-therapeutics-launches-two-phase-3-trials-ted/), a potential best-in-class subcutaneous TED therapy. This treatment is being investigated in two ongoing global Phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, which similarly evaluate its efficacy and safety in active and chronic TED patients. Beyond TED, Viridian is also progressing a portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008. These inhibitors hold promise for development across multiple autoimmune diseases. The company is located in Waltham, Massachusetts. Further information can be found on their website and social media channels.
Source link:
**Categories:** News
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### [DPHARM Follow the Money: Investor Insights on Technology Investment in Clinical Trials](https://www.clinicaltrialvanguard.com/conference-coverage/dpharm-follow-the-money-investor-insights-on-technology-investment-in-clinical-trials/)
**Published:** November 6, 2024
**Author:** Moe Alsumidaie
**Content:**
[The DPHARM conference](https://dpharmconference.com/ "The DPHARM conference") brought together a distinguished panel to discuss the critical role of technology investment in clinical trials. Moderated by Nick Slack, MBE, Executive in Residence at Warburg Pincus, the panel included Andrea Jackson from Northpond Ventures, Ryan Jones from Florence Healthcare, Andy Lee from Merck, Bari Kowal from Regeneron, and Bryan Spielman from Advarra. The discussion centered on aligning the interests of various stakeholders in the clinical trial innovation process to determine the best areas for investment, focusing on the industry’s growth, challenges, and potential for technological advancements.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#industry-overview-growth-amidst-challenges)Industry Overview: Growth Amidst Challenges
Nick Slack opened the discussion by highlighting the industry’s growth over the past two decades, noting a 71% increase in trial starts and a 4% annual growth in new R&D investments. Substantial investments in new platforms, molecules, and R&D initiatives largely drive this growth. However, Slack also pointed out significant challenges that accompany this growth. For instance, the complexity of clinical trials has increased, with a 20% rise in the number of endpoints being studied. This complexity often results in more amendments, which have risen by 60%, and timelines that are frequently missed by about 15%.
Slack also highlighted a concerning trend: a sharp decrease in investigator participation. Since 2018, there has been an 18% drop in the number of investigators involved in clinical trials, with only 37% conducting more than one trial. This decline is particularly alarming given that investigators are considered the lifeblood of the clinical trial process. Additionally, oncology’s largest therapeutic area has seen a 70% increase in trial starts since 2010, growing at a rate of 5% year on year. Despite this growth, the industry faces a crisis in [oncology trials](https://www.clinicaltrialvanguard.com/news/mindranks-mrank-106-gets-fda-clearance-for-oncology-trials/), exacerbated by nursing shortages and capacity constraints. Academic medical centers, which treat 20% of the cancer population, see 80% of the trials, creating a significant imbalance.
## [](#navigating-headwinds-with-strategic-planning)Navigating Headwinds with Strategic Planning
Andy Lee from Merck acknowledged the industry’s forward momentum but emphasized the need to address the headwinds that come with it. He noted that while trial starts and complexity are rising, investigator participation is declining, posing a significant challenge. Lee explained that the complexity of trials often stems from the need to refine patient populations for newer therapies, leading to higher response rates. For example, newer therapies require more inclusion and exclusion criteria to identify the right patients who will respond to the treatment, resulting in more complex trial designs.
Lee advocated for a more thoughtful approach to trial design, focusing on reducing unnecessary complexity and ensuring new therapies target the right patients in the right geographies. He mentioned that the industry often sees a proliferation of “me-too” drugs, where multiple companies develop similar molecules, leading to increased competition and higher standards of care. This situation necessitates running studies against the current standard of care, which has evolved and become more stringent. Lee suggested that limiting the number of new potential entities that are essentially “me-too” drugs could help reduce complexity and improve trial efficiency. He also highlighted the importance of careful planning and strategic thinking to mitigate the industry’s headwinds.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#investing-in-durable-ideas)Investing in Durable Ideas
Andrea Jackson from Northpond Ventures discussed the role of investors as ecosystem builders, emphasizing the importance of finding durable ideas that can withstand market fluctuations and deliver tangible results. She explained that on the venture side, the goal is to invest in companies that can address the industry’s longstanding challenges. Jackson noted that while the current system is functional, there is significant room for improvement. According to Jackson, one of the biggest challenges is identifying ideas that can go from trial design to the completion of a trial. She mentioned that very few companies pitch ideas focused on helping large pharmaceutical companies like Merck complete their trials faster to bring drugs to market more quickly. Instead, many companies propose nuanced or esoteric solutions that may not be durable in the long run.
Jackson emphasized matching financial and human capital to the right ideas. She explained that investing is about providing financial resources and supporting the human capital behind these ideas. The goal is to find ideas that can be executed effectively and lead to meaningful trial efficiency and effectiveness advancements. Jackson acknowledged that this process involves a degree of imagination and hope, but it also requires careful execution to ensure that the ideas are durable and can make a real impact. She highlighted the need for investors to sift through numerous proposals to identify those that offer the most promise for long-term success.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#leveraging-technology-and-data)Leveraging Technology and Data
Bari Kowal from Regeneron expressed optimism about the potential for technology and data to transform clinical trials. She pointed out that while the fundamentals of trial operations haven’t changed much over the past 30 years, the availability of new technologies and global reach offers unprecedented opportunities. Kowal emphasized that the industry now has access to data and technology that was unavailable a decade or two ago, which can significantly enhance trial efficiency. For example, data analytics and machine learning can improve trial design, patient recruitment, and data collection. However, she noted that no single company can be an expert in all areas, and it is crucial to have connectivity between different solutions to streamline the trial process.
Kowal also mentioned that the industry needs to capitalize on the changes in ways of working and global reach. She explained that the ability to conduct trials globally and access diverse patient populations can lead to more robust and generalizable results. However, this requires careful planning and coordination to ensure trials are conducted efficiently and ethically. Kowal stressed the importance of connectivity between the various components of the trial process. She explained that running a clinical trial involves numerous interconnected areas, each of which can benefit from technological innovations. The industry can achieve faster and more streamlined operations by focusing on these areas.
## [](#building-an-ecosystem)Building an Ecosystem
Bryan Spielman from Advarra highlighted the importance of building an ecosystem that includes sponsors and sites. He noted that while technology initiatives like TransCelerate have been useful, they often lack sufficient participation from academic medical centers and other key stakeholders. Spielman explained that TransCelerate’s initiatives, such as the Shared Investigator Platform (SIP), are developed from a sponsor perspective and may not fully address the needs of sites. He emphasized the need for a platform approach that facilitates connectivity and allows for integrating innovative solutions from various companies.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Spielman mentioned that Advarra has established a site-sponsor consortium to understand better the challenges faced by both sponsors and sites. This consortium aims to foster collaboration and develop solutions that address the needs of all stakeholders. He drew a parallel to Salesforce, a company that has successfully built an ecosystem in the enterprise space. Spielman explained that Salesforce’s ecosystem allows other companies to build their business models around its platform, creating a collaborative environment that drives innovation. He suggested that the clinical trial industry could benefit from a similar approach, where a central platform facilitates connectivity and allows for the integration of various solutions.
## [](#enabling-trial-sites)Enabling Trial Sites
Ryan Jones from Florence Healthcare emphasized enabling trial sites to do their best work. He pointed out that many clinical trial challenges are site-related, and improving site capabilities can significantly enhance trial efficiency. Jones explained that Florence Healthcare’s mission is to support trial sites, and this focus has helped align the company’s efforts with the needs of the industry. He mentioned that the preamble slides presented by Hassan showed that many of the issues in clinical trials are related to site operations rather than problems within sponsors or CROs. Jones argued that in 2023, trial site enablement is the most critical mission for the industry.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
By providing sites with the tools and support they need, the industry can create the capacity to address the growing complexity of trials. Jones also shared advice from an early mentor who emphasized the importance of not doing anything unnatural. He explained that companies sometimes push for extreme measures that are not broadly accepted or fail to consider the necessary change management processes in a rush to innovate. Jones stressed the need for thoughtful and realistic approaches to innovation, considering the practical challenges trial sites face. He highlighted the importance of aligning the efforts of investors, customers, and the internal team to enable trial sites.
The panel agreed on collaboration among investors, sponsors, and sites to address the industry’s challenges. By focusing on durable ideas, leveraging technology and data, and building a connected ecosystem, the clinical trial industry can navigate its headwinds and continue its momentum. The insights from [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) 2023 underscore the importance of strategic investment and collaboration in driving innovation and efficiency in clinical trials. As the industry evolves, aligning stakeholder interests and the thoughtful application of technology will be crucial in overcoming persistent challenges and achieving better outcomes for patients worldwide.
**Categories:** Article: Conference Coverage
---
### [Gilead Sciences Announces Strong Q3 2024 Results](https://www.clinicaltrialvanguard.com/news/gilead-sciences-announces-strong-q3-2024-results/)
**Published:** November 7, 2024
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences reported third-quarter solid 2024 financial results, with a 7% year-over-year increase in total product sales, reaching $7.5 billion. Excluding Veklury, product sales grew by 7%, totaling $6.8 billion. This growth was primarily attributed to increased sales in HIV, Oncology, and Liver Disease.
HIV product sales reached $5.1 billion, a 9% increase in the same period in 2023. This rise was driven by higher average realized prices due to shifts in channel mix and increased demand, offset partially by inventory dynamics. Biktarvy sales saw a 13% year-over-year increase, reaching $3.5 billion.
Liver Disease portfolio sales experienced a 4% increase, reaching $733 million. This growth was primarily due to higher demand for viral hepatitis medicines, partially offset by unfavorable pricing dynamics. Oncology sales also performed well, with a 6% year-over-year increase, reaching $816 million. Trodelvy sales contributed significantly to this growth, increasing by 17% to $332 million due to higher demand across all regions.
Veklury sales saw a 9% increase, totaling $692 million, primarily driven by rising [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/)-related hospitalizations, particularly in the United States. [Cell Therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) product sales remained relatively flat at $485 million.
Gilead’s robust third-quarter performance raised its full-year 2024 financial guidance. The company now expects product sales to be between $27.8 billion and $28.1 billion, from the previous guidance of $27.1 billion to $27.5 billion. Product sales, excluding Veklury, are now projected to be between $26.0 billion and $26.3 billion, compared to the prior estimate of $25.8 billion to $26.2 billion. Veklury sales projections were also increased to $1.8 billion from the previous $1.3 billion estimate. Diluted earnings per share (EPS) are now expected to be between $0.05 and $0.25, while non-GAAP diluted EPS is projected to be between $4.25 and $4.45, reflecting an upward revision from previous guidance.
These positive results and upwardly revised guidance are attributed to strong topline growth and efficient operating expense management. The company anticipates further impacting patients and communities in the coming months, particularly with the continued U.S. launch of Livdelzi for primary biliary cholangitis and the potential launch of lenacapavir, a twice-yearly HIV prevention option.
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**Categories:** News
---
### [Janux Therapeutics Q3 2024 Financial Results & Highlights](https://www.clinicaltrialvanguard.com/news/janux-therapeutics-q3-2024-financial-results-highlights/)
**Published:** November 7, 2024
**Author:** Jon Napitupulu
**Content:**
Janux Therapeutics, a clinical-stage biopharmaceutical company, recently announced its third-quarter 2024 financial results and provided a business update. The company focuses on developing novel immunotherapies using its proprietary Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms.
The company’s primary focus is enrolling participants in clinical trials for its two lead candidates: [JANX007](https://www.clinicaltrialvanguard.com/news/janux-expands-phase-1b-trials-for-janx007-in-mcrpc/) (PSMA-TRACTr) and JANX008 (EGFR-TRACTr). An update on the JANX007 program is expected by the end of the year.
JANX007, a TRACTr targeting PSMA, is currently in a Phase 1 clinical trial for metastatic castration-resistant prostate cancer (mCRPC). JANX008, also a TRACTr, targets EGFR and is being evaluated in a Phase 1 trial for various solid tumors. These include colorectal carcinoma, squamous cell carcinoma of the head and neck, non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/), renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma, and triple-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/).
Beyond these clinical candidates, Janux is developing a pipeline of additional TRACTr and TRACIr programs, some of which have reached the development candidate stage or later. The company is currently evaluating priorities within its preclinical pipeline.
Janux’s core mission is to develop safe and effective tumor-activated immunotherapies. Its proprietary TRACTr and TRACIr platforms aim to harness patients’ immune systems to eradicate tumors while minimizing safety risks. The company’s clinical-stage TRACTr candidates include JANX007 for prostate cancer and JANX008 for multiple solid tumor types.
Financially, Janux reported cash and cash equivalents of $26.75 million and short-term investments of $631.28 million as of September 30, 2024. Total assets were $695.02 million compared to $380.41 million at the end of 2023. Total liabilities stood at $38.92 million.
For the three months ended September 30, 2024, Janux reported collaboration revenue of $0.44 million and total operating expenses of $36.28 million. This resulted in a net loss of $28.06 million. For the nine months ended September 30, 2024, collaboration revenue was $10.59 million, and total operating expenses were $80.41 million, leading to a net loss of $48.78 million.
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**Categories:** News
---
### [Sarepta Q3 2024: Financial Results & Developments](https://www.clinicaltrialvanguard.com/news/sarepta-q3-2024-financial-results-developments/)
**Published:** November 7, 2024
**Author:** Jon Napitupulu
**Content:**
Sarepta Therapeutics reported strong third-quarter 2024 financial results, marked by a significant revenue increase and positive net income. Net product revenue reached $429.8 million, a 39% year-over-year surge. This growth is largely attributed to the successful launch and expanded label of ELEVIDYS, their gene therapy for [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy (DMD). ELEVIDYS alone generated $181.0 million in net product revenue, surpassing previous projections. Including royalties from Roche’s ex-US ELEVIDYS sales, total ELEVIDYS revenue reached $190.5 million.
The company’s other PMO therapies for DMD, EXONDYS 51, VYONDYS 53, and AMONDYS 45, also performed well, collectively contributing $248.8 million in net product revenue during the quarter. Notably, the launch of ELEVIDYS hasn’t significantly impacted sales of these established treatments. Sarepta is prioritizing its portfolio and advancing its pipeline, anticipating a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) submission for one Limb-girdle muscular dystrophy (LGMD) program by mid-2025, with two additional LGMD programs entering clinical trials.
Financially, Sarepta achieved a GAAP net income of $33.6 million and a non-GAAP net income of $67.0 million for Q3 2024. This positive performance contrasts with a net loss in the same period of the previous year. Total revenues for the first nine months of 2024 totaled $1.24 billion, a 47% increase compared to the same period in 2023. This increase reflects ELEVIDYS’ launch and label expansion, alongside collaboration revenue from Roche’s declined option to acquire ex-US rights to an early-stage Duchenne program. Additional revenue streams included contract manufacturing and royalty payments from Roche related to ELEVIDYS.
Cost of sales, excluding amortization of in-licensed rights, rose due to ELEVIDYS’ launch. Research and development expenses increased in Q3 2024 due to costs associated with terminating a manufacturing and supply agreement, offset by decreased spending on other programs. However, for the nine months, research and development expenses decreased due to ELEVIDYS commercial batch capitalization. Selling, general, and administrative expenses increased due to ongoing commercialization efforts for ELEVIDYS, litigation matters, charitable contributions, and compensation expenses. Other income increased significantly compared to the previous year, primarily due to a one-time impairment of a strategic investment in 2023. The company uses non-GAAP financial measures to evaluate operational performance and cash requirements internally and to provide investors with a clearer comparison of period-to-period results.
Sarepta’s product portfolio addresses DMD through exon skipping technology. EXONDYS 51, VYONDYS 53, and AMONDYS 45 are PMO-based therapies targeting specific exons of the dystrophin gene. ELEVIDYS is a gene therapy delivering a transgene for micro-dystrophin production. Each product has specific indications, safety information, and prescribing details available in their respective full Prescribing Information.
Sarepta’s focus remains on developing precision genetic medicines for rare diseases. With over 40 programs in development, the company leverages a multi-platform approach encompassing gene therapy, RNA, and gene editing technologies.
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**Categories:** News
---
### [How High Interest Rates Are Impacting Clinical Trial Investment](https://www.clinicaltrialvanguard.com/conference-coverage/how-high-interest-rates-are-impacting-clinical-trial-investment/)
**Published:** November 7, 2024
**Author:** Moe Alsumidaie
**Content:**
The [DPHARM](https://www.clinicaltrialvanguard.com/conference-coverage/ai-and-ml-in-drug-development-a-deep-dive-at-dpharm/) 2024 conference recently convened a panel of industry leaders to discuss the alignment of innovations to address the significant challenges in clinical trials, particularly within a resource-constrained environment. The panel, moderated by Nick Slack, Chairman and CEO of The START Center, included representatives from major pharmaceutical companies, CROs, and investment firms. They provided a comprehensive overview of the current landscape, highlighting the economic influences, operational adjustments, regulatory challenges, digital transformation, site and patient dynamics, and clinical trial investment perspectives shaping the future of clinical trials.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#economic-influences-on-clinical-trials)Economic Influences on Clinical Trials
The panel discussion opened with an analysis of the macroeconomic factors impacting the pharmaceutical industry, particularly the clinical trials sector. Nick Slack presented data showing a clear correlation between interest rates and clinical trial activity. He explained that during periods of low interest rates, such as in 2021, the industry saw a surge in clinical trial initiations due to the availability of cheap capital, which encouraged pharmaceutical companies to invest heavily in research and development. However, as interest rates rose, the cost of borrowing increased, prompting a strategic shift in resource allocation within the industry. This economic shift has led to significant job cuts and operational changes, with companies like Bristol-Myers Squibb and Novartis announcing large-scale layoffs as part of broader austerity strategies. These measures aim to optimize operations and focus on high-priority projects that promise the most significant returns, reflecting a broader industry-wide reprioritization towards assets that deliver substantial value and growth for patient outcomes and investor returns.
## [](#operational-adjustments-and-innovations)Operational Adjustments and Innovations
Andy Lee from Merck & Co. shared insights into how his organization is adapting to economic pressures by enhancing operational efficiency. He described the current environment as chaotic and disruptive, influenced by external factors like geopolitical tensions and internal regulatory changes. Lee emphasized the importance of productivity, noting that Merck has been challenged to “bend the curve” by finding operational efficiencies. This involves a dual strategy of ceasing unproductive activities and embracing innovative, patient-centric approaches. Lee elaborated on Merck’s operational scale, highlighting that the company runs approximately 300 clinical trials annually, involving around 100,000 patients. Merck uses its tools and systems to manage this vast operation efficiently, allowing for greater control and predictability. He noted that 95% of their project milestones are delivered on time, emphasizing the importance of quality and efficiency in their processes. While no single innovation has drastically reduced cycle times by 30%, a series of small, incremental improvements can collectively lead to significant operational gains.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Peyton Howell, CEO of Parexel, added to the discussion by highlighting the diverse landscape of sponsors they work with, ranging from large pharmaceutical companies to smaller biotech firms. She noted that while the industry has faced challenges, particularly in the biotech sector, there is still growth in the pipeline. Howell emphasized the need for a more measured and disciplined approach to clinical trials, advocating for creative solutions that push traditional boundaries. She stressed that achieving efficiency requires doing things differently, such as exploring new sites and country mixes and being open to innovative methodologies. Howell pointed out that despite the challenges, the industry is seeing growth, with good work continuing to receive funding. However, she cautioned that there is a need for more accountability in delivering on time and with innovation, which is why the conference’s focus on innovation is so timely and relevant.
## [](#regulatory-challenges-and-clinical-trial-investment-needs)Regulatory Challenges and Clinical Trial Investment Needs
Bari Kowal from Regeneron Pharmaceuticals addressed the regulatory challenges that add layers of complexity to clinical trial operations. She pointed out that new regulations, such as those related to data privacy and medical device reporting, create significant churn in companies’ operations. Kowal emphasized the importance of engaging with regulators to influence the development of these regulations rather than being passively affected by them. By participating in regulatory discussions, companies can help shape policies more conducive to efficient clinical trial operations. Kowal also discussed the necessity of upfront clinical trial investment in technology and automation to drive transformative changes. She explained that while incremental improvements are valuable, they often do not lead to substantial changes in output. Instead, Regeneron focuses on transformative initiatives, such as streamlining data collection processes through EHR to EDC connections. This approach aims to reduce costs and speed up trial processes by improving data accuracy and reducing the administrative burden on clinical sites.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Kowal elaborated on the need for strategic clinical trial investment to adapt to the evolving regulatory landscape. She noted that while adding more resources may not always be feasible, investing in technology and process improvements can lead to significant long-term benefits. Regeneron is prioritizing clinical trial investment that have the potential to move the needle from an automation and technology perspective, focusing on initiatives that can bring about dramatic changes rather than just incremental improvements. Kowal highlighted that one of their primary focus areas is EHR to EDC integration, which can streamline data collection, improve organizational efficiency, and ultimately benefit both sites and patients. By investing in such transformative technologies, Regeneron aims to enhance the speed and cost-effectiveness of its clinical trials, positioning itself for success in a rapidly changing industry.
## [](#the-role-of-digital-transformation)The Role of Digital Transformation
Shoibal Datta from Takeda provided a perspective on the role of digital transformation in clinical trials. He noted that the traditional clinical trial model is resource-intensive and prone to errors, making it ripe for disruption. Datta parallels the healthcare industry, transitioning from manual processes to more automated, technology-mediated practices. He suggested that similar changes are taking hold in the clinical trials sector, driven by the need for greater efficiency and accuracy. Datta emphasized that digital transformation requires financial clinical trial investment and a workforce equipped with the necessary skills to implement these changes. He highlighted the importance of building a team that can leverage digital tools effectively, ensuring that the transition to more automated processes is smooth and successful. This shift towards digital solutions is seen as a critical step in overcoming the limitations of traditional clinical trial models and achieving significant improvements in trial efficiency and outcomes.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Datta further explained that integrating digital technologies into clinical trials can lead to more streamlined and efficient processes. He pointed out that digital tools can help reduce the reliance on manual data entry and improve data accuracy, ultimately leading to faster and more reliable trial results. However, he cautioned that the success of digital transformation depends on the ability to adapt to new technologies and the willingness to invest in the necessary infrastructure and training. Datta stressed that while the initial clinical trial investment may be significant, the long-term benefits of digital transformation can be substantial, offering a competitive advantage in an increasingly complex and competitive industry. By embracing digital solutions, companies can improve their operational efficiency and enhance the quality and reliability of their clinical trials.
## [](#site-and-patient-dynamics)Site and Patient Dynamics
Jim Murphy, CEO of Greenphire, provided insights into clinical trial sites’ financial pressures. He explained that many sites operate with limited capital, often having only six months of operating funds available. This financial constraint is exacerbated by a significant portion of payments to sites being delayed beyond 90 days. Murphy noted that this situation creates a challenging environment for sites that face increased administrative burdens due to more complex trial logistics. Murphy highlighted that patient recruitment has become more complex over the years, with the average distance traveled by participants to reach a trial site increasing by 40% in the last six years. This trend places additional logistical and financial pressures on sites, as they must facilitate travel and other arrangements for participants. Murphy concluded that sites are experiencing a resource-constrained environment, with financial and operational bottlenecks that must be addressed to ensure the sustainability of clinical trial operations.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Murphy elaborated on the challenges sites face, noting that the increased complexity of clinical trials has led to a greater administrative burden on site staff. He explained that site coordinators are often responsible for managing logistics and ensuring that participants can attend trial visits, which can be time-consuming and resource-intensive. Murphy emphasized the importance of supporting sites with the tools and resources to manage these challenges effectively. He suggested that streamlining administrative processes and improving payment systems could help alleviate some of the financial pressures sites face, allowing them to focus more on patient care and trial execution. By addressing these bottlenecks, the industry can help ensure that sites remain viable and capable of supporting the growing demand for clinical trials.
## [](#clinical-trial-investment-perspectives)Clinical Trial Investment Perspectives
Zachary Sigal from LLR Partners offered an investor’s viewpoint on the current market dynamics. He noted that companies increasingly seek technologies that drive operational efficiency and reduce the complexity of managing multiple technology relationships. Sigal observed a trend toward platform solutions that streamline processes and provide comprehensive support for clinical trial operations. Sigal explained that his firm is focused on identifying companies that can offer innovative solutions to the challenges faced by the industry. These companies are expected to address operational inefficiencies and deliver value to investors. He emphasized the importance of partnering with customers to develop new ways of conducting trials and leveraging technology to achieve better outcomes. This approach aligns with the broader industry trend of seeking integrated solutions that enhance efficiency and reduce costs.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
Sigal further discussed the role of private equity in driving innovation within the clinical trials sector. He noted that investors increasingly seek opportunities to support companies that can offer scalable solutions to the industry’s most pressing challenges. Investing in companies that provide integrated platform solutions can help drive the adoption of new technologies and processes that improve trial efficiency and outcomes. Sigal highlighted that the focus on platforms reflects a growing recognition of the need for comprehensive solutions to address multiple aspects of clinical trial operations, from data management to patient engagement. By supporting companies that offer these solutions, investors can play a crucial role in advancing the industry’s ability to conduct efficient and effective clinical trials.
In conclusion, the DPHARM 2024 panel provided a comprehensive overview of the challenges and opportunities faced by the clinical trials industry. The discussion highlighted the need for strategic clinical trial investment, regulatory engagement, and digital transformation to drive innovation and efficiency. As the industry evolves, collaboration among stakeholders will be essential to overcoming resource constraints and significantly improving clinical trial operations. The insights shared at the conference highlight the importance of embracing new technologies and methodologies to ensure the future success of clinical trials.
**Categories:** Article: Conference Coverage
---
### [Alzheimer’s Trials and Others See More Diversity When Technology and Humans Work Together](https://www.clinicaltrialvanguard.com/article/alzheimers-trials-and-others-see-more-diversity-when-technology-and-humans-work-together/)
**Published:** November 8, 2024
**Author:** Cara Brant
**Content:**
Clinical trial sponsors are [facing mounting pressure](https://www.fda.gov/news-events/press-announcements/fda-guidance-provides-new-details-diversity-action-plans-required-certain-clinical-studies) to secure trial participants that more closely reflect the diverse populations affected by their treatments, but are still struggling to do so. This isn’t an issue that only affects *some* pharmaceutical companies or that’s only present when trialing *some* health conditions.
At the very highest level, trials simply don’t reflect populations as a whole. For instance, in the U.S., Hispanic or Latino people make up[ 19% of the population](https://minorityhealth.hhs.gov/hispaniclatino-health), but only[ ](https://www.fda.gov/media/145718/download)[11% of clinical trial participants](https://www.fda.gov/media/145718/download) in 2020 were Hispanic or Latino.
On a more micro-level, we can look at trials for specific health conditions, such as [Alzheimer](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/)’s Disease, which is most prevalent among Black and Hispanic or Latino women. Its trial populations should reflect the demographics that are most likely to have the disease and benefit from the treatment, but this has not been the case in many past trials.
All of this is now changing.
Recently, we’ve seen first-hand how a machine learning-aided approach to participant identification and targeting can lead to significant breakthroughs in diversity efforts across active Alzheimer’s Disease, Cardiometabolic and Immunology trials. Equipped with this type of advanced technology, recruitment professionals are able to synthesize millions of proprietary and public data points to more acutely target trial candidates–and do so at a scale that’s not possible manually.
But it’s important to note that technology alone is not enough to keep trial participants engaged through the duration of trials. That requires a human touch.
Here are a number of other on-the-ground insights on the challenges and advancements in recruiting diverse populations for trials.
## [](#the-lack-of-diversity-in-trials-isnt-due-to-a-lack-of-desire-on-behalf-of-sponsors-nor-a-lack-of-interest-among-diverse-populations-to-participate)**The lack of diversity in trials isn’t due to a lack of desire on behalf of sponsors, nor a lack of interest among diverse populations to participate.**
Rather, it’s that many people don’t know how to participate, don’t know which trials are available to them, how to qualify or otherwise get involved. This means clinical trial recruitment efforts can’t operate as though all potential participants have the same baseline knowledge. That assumption alone has contributed greatly to the lack of efficacy in reaching these populations.
## [](#getting-in-front-of-new-audiences-requires-a-lot-more-than-simply-scaling-your-existing-efforts)**Getting in front of new audiences requires a lot more than simply scaling your existing efforts.**
Now that we know that one of the major obstacles to recruiting is informing and educating different populations about trials and how to get involved, our next move is figuring out how to get in front of these new audiences.
It’s important to note that these audiences are spread across a number of geographic locations, each with different health conditions and varying levels of knowledge about trials, so we need to meet them where they are. Scaling this type of participant identification effort with precision is a job for technology.
Sponsors can leverage AI and machine-driven platforms to hyper-target media buying efforts and reach and educate different groups about clinical trials at scale. Geo-specific advertising can help find relevant people in the desired physical locations. From there, they should consider tailoring their messages specifically to the people they’re attempting to recruit in those locations. For example, sponsors might want to consider adding a Spanish language component for each U.S. campaign to ensure they can reach the highest number of potential Spanish-speaking participants. Additionally, Spanish-speaking nurses on staff can help put potential participants at ease by communicating in the language they are most comfortable with.
In addition to first language, race and location, sponsors should also account for variables such as age, gender and others.
## [](#new-audiences-will-participate-if-they-understand-the-value-of-doing-so)**New audiences will participate if they understand the value of doing so.**
All of this is to say sponsors can’t only identify and inform new populations about trials; they need to work to understand their specific needs and clearly communicate the value of clinical trials.
Using this approach, the industry is seeing significant breakthroughs in achieving diversity in clinical trials. For example, some results we’ve seen include:
- 62% of participant randomizations in a trial for Alzheimer’s Disease–most prevalent among Black and Hispanic women–were from diverse populations.
- 32% of randomizations for a cardiometabolic program, testing a treatment for conditions that most prominently affect American Indians, Alaskan Natives, Black and Hispanic populations, came from diverse populations.
- For an immunology trial in Hidradenitis Suppurativa (HS) –most prevalent among Black women – 59% of randomizations originated from diverse populations.
## [](#recruiting-new-diverse-audiences-is-step-one-ensuring-that-they-and-other-participants-stay-in-the-trial-for-the-full-duration-is-step-two)**Recruiting new diverse audiences is step one. Ensuring that they (and other participants) stay in the trial for the full duration is step two.**
The primary reason for delays in clinical trials continues to be patient recruitment and retention. But it’s clear boosting a participant’s satisfaction with their clinical trial experience is the best method to increase retention.
Through extensive patient surveys, trial sites can better understand how patients are feeling throughout the trial, as well as during their experience at sites. Participants often express appreciation for appointment reminders that keep them on track. They also respond positively to efforts that help them understand their role in a study and reinforce their importance throughout the process. This can look like a mix of texts, emails and phone calls that deliver appointment reminders alongside “what to expect”-type guidance and messages of motivation and appreciation. Simply thanking participants for their ongoing commitment to trials and keeping them engaged throughout the process helps participants better understand their importance to the trial. And this directly translates to increased retention.
Trial sites have also seen positive feedback and higher retention when pairing patient populations who are vulnerable or would benefit from additional support with a nurse advocate. This is particularly effective when the nurse advocate speaks the participant’s first language. This is an important factor when it comes to boosting retention for historically underrepresented populations in trials. A nurse advocates co-travels with each patient throughout their clinical trial journey to ensure continued patient engagement and the best possible patient experience.
## [](#take-special-steps-to-retain-women-who-are-participating-in-trials)**Take special steps to retain women who are participating in trials.**
No matter what their demographic, optimizing trials for women’s participation means considering nuances of their lifestyle, not just during recruitment but in the design of the clinical trial itself.
Today, most women have their hands full, working full time jobs in addition to taking care of family and a household. As a result, the thought of adding another obligation into a jam-packed routine while coordinating existing responsibilities, such as childcare, may turn some women away from participating.
Flexibility is key for trials that want to effectively recruit and retain women. This includes designing trials with telehealth check-ins to reduce travel, extending evening or weekend hours for those with full time jobs and introducing flexible appointment booking options. We’ve also received positive feedback from trial sites that provide child entertainment in the form of books or puzzles in the site waiting room.
A trial site’s ability to seamlessly integrate into a potential participant’s established routine will ultimately be what allows trials to recruit and retain women–and in turn, speed up more treatment approvals for women.
## [](#recent-government-action-to-help-boost-trial-diversity-reinforces-the-industrys-urgency)**Recent government action to help boost trial diversity reinforces the industry’s urgency.**
In June the FDA released a new [draft guidance on Diversity Action Plans](https://www.fda.gov/news-events/press-announcements/fda-guidance-provides-new-details-diversity-action-plans-required-certain-clinical-studies) to boost diversity in clinical trials. This aims to increase clinical trial study enrollment for underrepresented populations so treatments can be tested on a proportionate population of those most likely to use them.
This move further demonstrates that the government and industry are aligned on the need for diversity in trials, but now the onus is on clinical trial sponsors to effectively reach, recruit and retain these populations.
## [](#unlike-in-the-past-the-industry-now-has-the-tools-it-needs-to-achieve-diversity-in-clinical-trials)**Unlike in the past, the industry now has the tools it needs to achieve diversity in clinical trials.**
Once all clinical trial sponsors optimize their participant recruitment and retention efforts, trial populations will look a lot more similar to the populations that use the treatment in the wider market. This reality will become the industry standard, not the anomaly.
The main difference between the past and the future is that the industry now has the tools and government alignment it needs to achieve new levels of diversity in clinical trials.
**Categories:** Article
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### [Tezspire Meets Co-primary Endpoints in Phase 3 WAYPOINT Trial](https://www.clinicaltrialvanguard.com/news/tezspire-meets-co-primary-endpoints-in-phase-3-waypoint-trial/)
**Published:** November 11, 2024
**Author:** Jon Napitupulu
**Content:**
A Phase III trial, WAYPOINT, has yielded positive results for [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) and Amgen’s TEZSPIRE ([tezepelumab](https://www.clinicaltrialvanguard.com/news/tezspires-potential-role-in-chronic-obstructive-pulmonary-disease-latest-ats-findings-revealed/)) in treating chronic rhinosinusitis with nasal polyps (CRSwNP). The randomized, double-blind trial demonstrated that TEZSPIRE significantly reduced nasal polyp size and nasal congestion compared to a placebo in adults with severe CRSwNP. Participants were symptomatic despite receiving standard intranasal corticosteroid treatment.
CRSwNP significantly impacts patients’ daily lives. Nasal polyps obstruct nasal passages, causing breathing difficulties, reduced smell and taste, sleep disturbances, pain, and fatigue. The WAYPOINT trial results suggest that tezepelumab could be a new treatment option for this debilitating condition.
Current treatments for CRSwNP, including intranasal and systemic corticosteroids, surgery, and other biologics, often involve repeat surgeries and high doses of oral corticosteroids, leading to potentially serious systemic side effects. Tezepelumab offers a potential alternative that may lessen the burden on patients and healthcare systems.
The positive WAYPOINT results highlight tezepelumab’s unique mechanism of action. By targeting thymic stromal lymphopoietin (TSLP), a key epithelial cytokine, the treatment addresses the root of the inflammatory cascade driving epithelial-driven inflammatory diseases like CRSwNP and severe [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/). This mechanism effectively tackles multiple inflammatory drivers, offering a broader approach than current treatments.
The safety and tolerability of TEZSPIRE in this trial aligned with the medicine’s established profile. Complete results will be presented to regulatory authorities and the scientific community. Currently, TEZSPIRE is approved in numerous countries, including the US, EU, and Japan, for severe asthma treatment. It’s available as a single-use pre-filled syringe and auto-injector for self-administration.
CRSwNP involves persistent inflammation of the nasal mucosa and the growth of nasal polyps. This inflammation and the resulting polyps can cause breathing problems, olfactory dysfunction, nasal discharge, facial pain, sleep disruption, and negatively impact quality of life. Epithelial dysfunction, a key characteristic of CRSwNP, weakens the epithelium’s protective barrier function. TSLP plays a significant role in the shared pathophysiological processes underlying both severe asthma and CRSwNP.
The WAYPOINT trial’s design was a double-blind, multicenter, randomized, placebo-controlled, parallel-group study. Adult participants with severe CRSwNP received either tezepelumab or a placebo via subcutaneous injection. The trial included a post-treatment follow-up period for participants completing the 52-week treatment phase. Co-primary endpoints measured changes from baseline in total nasal polyp size and nasal congestion. Key secondary endpoints included loss of smell, quality of life, and other disease-related measures.
TEZSPIRE, co-developed by AstraZeneca and Amgen, is a first-in-class human monoclonal antibody. It works by inhibiting TSLP, a cytokine crucial in initiating and maintaining the airway inflammation associated with severe asthma and CRSwNP. This collaboration involves shared costs and profits, with AstraZeneca leading development and Amgen leading manufacturing. Joint commercialization will occur in North America.
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**Categories:** News
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### [Neurogene Reports Positive Interim Data from NGN-401 Gene Therapy Clinical Trial for Rett Syndrome](https://www.clinicaltrialvanguard.com/news/neurogene-reports-positive-interim-data-from-ngn-401-gene-therapy-clinical-trial-for-rett-syndrome/)
**Published:** November 12, 2024
**Author:** Jon Napitupulu
**Content:**
Neurogene Inc. announced positive interim clinical data from its Phase 1/2 open-label trial of [NGN-401](https://www.clinicaltrialvanguard.com/news/neurogenes-triumphant-progress-in-ngn-401-gene-therapy/) gene therapy for Rett syndrome in female pediatric patients. The low-dose cohort demonstrated a favorable safety profile and meaningful skills and developmental milestones improvements. These improvements were consistent across multiple assessment scales and exceeded expectations based on the natural progression of Rett syndrome.
Interim data from seven participants, five in the low-dose group and two in the high-dose group, indicate that both NGN-401 dosages are well-tolerated. However, a treatment-related serious adverse event, consistent with known risks of AAV gene therapy, occurred in the third high-dose participant.
Focusing on the four participants in the low-dose cohort (aged 4-7 years), assessments conducted 3 to 15 months post-dosing revealed consistent and durable advancements across key Rett syndrome measures. All participants showed a 2-point improvement on the clinician-rated Clinical Global impression improvement (CGI-I) scale. Improvements were also observed in the caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ), ranging from 28% to 52%. Furthermore, participants experiencing sleep disruptions, constipation, and dysphagia at baseline demonstrated objective improvements after treatment. These observed gains in skills and developmental milestones were sustained and deepened over time, contrasting with the typical disease course.
In addition to the pediatric cohort, Neurogene initiated an adolescent/adult cohort (Cohort 3) to assess NGN-401’s potential in a broader patient population. This cohort will enroll three participants aged 16 and older at the high dose.
The company also secured FDA alignment on its potency assay strategy for NGN-401, a critical step for initiating a registrational trial. The FDA also supports Neurogene’s manufacturing scale-up plans, paving the way for a potential commercial product launch. Neurogene aims to provide an update on the registrational trial design in the first half of 2025.
Regarding its CLN5 Batten disease program, Neurogene will not proceed with the NGN-101 gene therapy. This decision follows the FDA’s denial of the company’s Regenerative Medicine Advance Therapy (RMAT) application despite Neurogene’s belief that it met the required criteria. The company is currently evaluating options for this program.
NGN-401 utilizes Neurogene’s EXACT™ transgene regulation technology to deliver the full-length human MECP2 gene. This technology aims to provide therapeutic levels of MECP2 while mitigating overexpression-related toxicity often associated with conventional gene therapy. NGN-401 has received various designations from the FDA, including selection for the START Pilot Program, RMAT designation, orphan drug designation, Fast Track designation, and rare pediatric designation. It has also received designations from the European Medicines Agency (EMA) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
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**Categories:** News
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### [GenSight Submits Lumevoq® Dossier to ANSM for Early Access Program Restart](https://www.clinicaltrialvanguard.com/news/gensight-submits-lumevoq-dossier-to-ansm-for-early-access-program-restart/)
**Published:** November 13, 2024
**Author:** Jon Napitupulu
**Content:**
GenSight Biologics has submitted an updated regulatory file for its LUMEVOQ® gene therapy to the French medicines safety agency (ANSM). This submission paves the way for the restart of the early access program in France. The company successfully manufactured LUMEVOQ®, blending two GMP drug substance batches to maximize available clinical vials and pass all necessary quality control tests.
LUMEVOQ® is designed to treat Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene. LHON is a rare genetic condition leading to acute and often irreversible vision loss. The resubmission signifies a shift from the manufacturing phase to the regulatory review process. The company and the LHON patient community anticipate the swift resumption of the early access program by the end of the year.
The updated file will facilitate the ANSM’s evaluation of individual compassionate use applications, now submittable by healthcare professionals under the early access program. GenSight Biologics anticipates a review period for these applications and will collaborate with the ANSM to streamline the assessment process. The company is preparing to supply the drug to the designated treatment center, the Quinze-Vingts hospital in Paris, by mid-December, working alongside the hospital’s administrative and medical staff to administer the first injections by the end of December.
GenSight Biologics is a clinical-stage biopharmaceutical company focused on developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Its pipeline utilizes two primary technology platforms: Mitochondrial Targeting Sequence (MTS) and optogenetics. These technologies aim to preserve or restore vision in patients with blinding retinal diseases. The company’s lead product candidate, GS010 (also known as LUMEVOQ®), is undergoing Phase III trials for LHON, primarily affecting adolescents and young adults, causing irreversible blindness.
LHON is a rare, maternally inherited mitochondrial genetic disease. The disease is characterized by the degeneration of retinal ganglion cells, leading to rapid and irreversible vision loss, often resulting in legal blindness. Typically, patients experience painless, sudden central vision loss in one eye, followed by impairment in the other. LHON is a symmetrical condition with limited functional visual recovery. Within a year of vision loss onset, 97% of individuals experience bilateral involvement, and in 25% of cases, both eyes are affected simultaneously.
LUMEVOQ® utilizes a proprietary Mitochondrial Targeting Sequence (MTS) technology platform developed from research at the Institut de la Vision in Paris. This technology, combined with the gene of interest, enables precise targeting of mitochondrial defects using an AAV (Adeno-Associated Virus) vector. The gene of interest is introduced into the cell, where it expresses the functional protein. This protein is transported to the mitochondria via specific [nucleotide](https://www.clinicaltrialvanguard.com/news/ateas-new-hcv-combo-aims-for-best-in-class-treatment/) sequences to restore the missing or deficient mitochondrial function. The European Medicines Agency (EMA) approved “LUMEVOQ” as the invented name for GS010 (lenadogene nolparvovec) in October 2018. Currently, LUMEVOQ® is not registered in any country.
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**Categories:** News
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### [Diamedica Doses First Patient in DM199 Preeclampsia Trial](https://www.clinicaltrialvanguard.com/news/diamedica-doses-first-patient-in-dm199-preeclampsia-trial/)
**Published:** November 14, 2024
**Author:** Jon Napitupulu
**Content:**
[DiaMedica Therapeutics](https://www.clinicaltrialvanguard.com/news/dm199-rinvecalinase-alfa-program-expanded-by-diamedica-therapeutics-into-preeclampsia/) Inc., a clinical-stage biopharmaceutical company, has initiated its Phase 2 investigator-sponsored trial for DM199, a potential preeclampsia (PE) treatment. The company anticipates initial results in the first half of 2025. This part of the study aims to establish DM199’s safety profile and ability to reduce blood pressure. For patients experiencing early-onset PE, researchers will also investigate potential improvements in uterine artery dilation, suggesting the therapy might modify the disease’s course.
Preeclampsia, a severe pregnancy complication typically arising after 20 weeks of gestation, is characterized by high blood pressure and organ damage, often affecting the kidneys and liver. Globally, it impacts up to 8% of pregnancies, creating substantial risks for both mother and baby. These risks encompass stroke, placental abruption, progression to eclampsia (seizures), premature birth, and even death. Symptoms can include severe headaches, vision changes, upper abdominal pain, and swelling in the hands and face. Currently, the only intervention to halt preeclampsia’s progression is delivering the baby, often prematurely. Women with a history of preeclampsia face a significantly elevated risk of future hypertension, heart disease, and stroke. Currently, no approved treatments exist for preeclampsia in the United States or Europe.
The Phase 2 trial, an open-label, single-center, single-arm study, will assess the safety and pharmacodynamics of DM199 in treating preeclampsia. Conducted at Tygerberg Hospital in Cape Town, South Africa, it is led by Professor Catherine Cluver of Stellenbosch University in partnership with DiaMedica. The trial aims to enroll up to 90 women with preeclampsia and potentially 30 with fetal growth restriction. In addition to blood pressure reduction, researchers will examine whether DM199 improves uterine artery dilation in early-onset PE patients. Such an outcome could indicate DM199’s potential as a disease-modifying therapy.
DM199 (rinvecalinase alfa) is a recombinant form of human tissue kallikrein-1 (rhKLK1), also under investigation for acute ischemic stroke. KLK1, a serine protease enzyme, is crucial in regulating physiological processes by increasing the production of nitric oxide, prostacyclin, and endothelium-derived hyperpolarizing factors. In preeclampsia, DM199 is expected to lower blood pressure, improve endothelial health, and enhance blood flow to maternal organs and the placenta. For acute ischemic stroke, DM199 is designed to increase blood flow and neuronal survival around the blocked blood vessel, inhibiting neuronal cell death and promoting blood vessel formation for neuronal repair. DiaMedica Therapeutics Inc., focused on developing treatments for severe ischemic diseases, believes DM199 offers a promising new approach to addressing these critical conditions.
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**Categories:** News
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### [Tae Life Sciences & Stella Pharma Announce BPA Strategic Agreement](https://www.clinicaltrialvanguard.com/news/tae-life-sciences-stella-pharma-announce-bpa-strategic-agreement/)
**Published:** November 14, 2024
**Author:** Jon Napitupulu
**Content:**
TAE [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) (TLS), a prominent developer of boron neutron capture therapy (BNCT) technology and related boron target drugs, and Stella Pharma, the developer of the boronophenylalanine (BPA) drug Steboronine® for BNCT, have announced a strategic collaboration. This partnership focuses on developing, commercializing, and expanding BNCT using BPA within the United States and European markets. Steboronine® represents a significant advancement, being the first and only clinically approved boron drug for BNCT. Japan led the way in approving BNCT for head and neck cancer treatment utilizing Stella Pharma’s BPA drug. Stella Pharma, a leading BPA producer and supplier, is partnering with TAE Life Sciences to broaden BNCT adoption globally.
The collaboration encompasses extensive international cooperation in BNCT and BPA development and commercialization across the US and Europe, with clinical trials projected to commence in 2026. TLS will act as the exclusive commercial partner for Stella Pharma’s BPA in these regions, significantly increasing BNCT’s availability outside Asia. This partnership represents a substantial step in making BNCT a viable cancer treatment option for patients worldwide.
Both companies are also dedicated to supporting BNCT equipment vendors globally in implementing BPA-based BNCT treatments. By fostering partnerships within the BNCT ecosystem, TLS and Stella Pharma aim to enhance BNCT’s clinical reach. Their shared objective is to improve treatment for recurrent head and neck cancers and advance clinical studies for other cancers, including brain, skin, breast, esophagus, and lung. This alliance is a key milestone in expanding BPA-based BNCT treatments beyond Asia, aiming to deliver this advanced cancer treatment to patients globally.
Beyond the immediate collaborative efforts, Stella Pharma and TAE Life Sciences share a long-term vision of advancing BNCT for challenging cancers. They are committed to exploring further collaborative opportunities, accelerating innovation, expanding treatment options, and improving outcomes for cancer patients worldwide. This partnership symbolizes a significant commitment to progress in the fight against cancer.
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**Categories:** News
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### [Omeros Corp. Q3 2024 Financial Results](https://www.clinicaltrialvanguard.com/news/omeros-corp-q3-2024-financial-results/)
**Published:** November 14, 2024
**Author:** Jon Napitupulu
**Content:**
Omeros Corporation recently announced its third-quarter 2024 financial results and provided updates on its clinical development programs. The company anticipates resubmitting its [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) for [narsoplimab](https://www.clinicaltrialvanguard.com/news/omeros-announces-narsoplimab-trial-endpoint-update/) in Hematopoietic Stem Cell Transplant-associated Thrombotic Microangiopathy (TA-TMA) following a productive pre-submission meeting with the FDA in September. Minor revisions to the analysis plan, requested by the FDA, have been incorporated.
Beyond narsoplimab, Omeros reported substantial progress in other clinical programs. For zaltenibart, successful end-of-Phase 2 meetings with both FDA and European regulators and sufficient drug supply pave the way for Phase 3 Paroxysmal Nocturnal Hemoglobinuria (PNH) enrollment in early 2025. Phase 3 trials for C3G with zaltenibart are expected to follow soon after. The company is preparing to initiate Phase 2 clinical trials for its long-acting MASP-2 inhibitor, OMS1029, upon identification of a suitable large-market indication. In the OMS527 program, addressing addictive and compulsive disorders, a NIDA-funded trial in adults with cocaine-use disorder is anticipated to begin next year. Preclinical oncology programs are also showing promising in vitro and in vivo results, strengthening Omeros’s patent portfolio.
Omeros reported a net loss of $32.2 million ($0.56 per share) for the third quarter of 2024, compared to a net loss of $37.8 million ($0.60 per share) for the same period in 2023. The net loss for the nine months ended September 30, 2024, was $125.5 million ($2.15 per share), compared to $108.8 million ($1.73 per share) in the prior year period. This nine-month period included an $18.4 million charge for narsoplimab drug substance, a $21.2 million debt repurchase payment, and $1.9 million in debt transaction costs. Omeros expenses all manufacturing activities until U.S. or European approval is reasonably certain.
As of September 30, 2024, Omeros held $123.2 million in cash and short-term investments, a decrease of $48.7 million from the end of 2023. OMIDRIA royalties for the third quarter of 2024 totaled $9.3 million on Rayner’s U.S. net sales of $31.0 million, compared to $10.0 million in royalties on $33.3 million in sales during the same quarter of 2023. Total operating expenses for the third quarter of 2024 were $35.4 million, a decrease from $48.2 million in the third quarter of 2023. This decrease primarily stems from a $5.0 million milestone payment to a third-party licensor in the prior year related to the zaltenibart program, reduced narsoplimab clinical spending due to the termination of the IgA nephropathy program, and lower employee compensation expenses. Interest expense decreased to $4.1 million in the third quarter of 2024, down from $7.9 million in the prior-year quarter, due to the retirement of convertible notes in 2023 and repurchase of 2026 notes. Interest and other income for the third quarter of 2024 was $2.3 million, compared to $4.4 million in 2023, largely due to a reduction in available cash and investments. Net income from discontinued operations, net of tax, was $4.9 million ($0.08 per share) in the third quarter of 2024, compared to $13.9 million ($0.22 per share) in the same quarter of 2023. This decrease primarily resulted from a higher remeasurement adjustment on the OMIDRIA contract royalty asset in the prior year, partially offset by increased non-cash interest earned.
Source link:[ http://www.businesswire.com/news/home/20241113240875/en/Omeros-Corporation-Reports-Third-Quarter-2024-Financial-Results](http://www.businesswire.com/news/home/20241113240875/en/Omeros-Corporation-Reports-Third-Quarter-2024-Financial-Results)
**Categories:** News
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### [Quince Therapeutics Q3 2024 Results & Business Update](https://www.clinicaltrialvanguard.com/news/quince-therapeutics-q3-2024-results-business-update/)
**Published:** November 14, 2024
**Author:** Jon Napitupulu
**Content:**
Quince Therapeutics, a late-stage biotechnology company focused on treating rare diseases, provided a pipeline update and financial results for the third quarter of 2024. The company highlighted progress in its pivotal Phase 3 NEAT clinical trial for Ataxia-Telangiectasia (A-T). As of the announcement date, 32 patients with A-T were enrolled across U.S., U.K., and EU clinical sites. With most planned NEAT study sites active, the company anticipates continued enrollment momentum. Quince aims to complete enrollment by the second quarter of 2025, with topline results expected in the fourth quarter of 2025.
Quince Therapeutics concentrates on harnessing a patient’s biology to treat rare conditions. Its lead candidate, EryDex, is developing for A-T, [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy (DMD), and other potential indications. The company leverages its proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology, which it intends to expand to treat additional rare diseases.
The company’s forward-looking statements, as defined by the Private Securities Litigation Reform Act of 1995, address anticipated developments related to EryDex, including its potential to treat various diseases and the expansion of the AIDE technology. These statements reflect current expectations but are subject to inherent uncertainties and risks. Factors that could influence actual outcomes include but are not limited to, risks detailed in the company’s SEC filings, including its Quarterly Report on Form 10-Q. These forward-looking projections are current as of the announcement date, and Quince has no obligation to update this information unless legally required. More details about Quince Therapeutics and its latest news can be found on their website and social media channels.
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**Categories:** News
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### [Koru Medical Systems Announces Q3 2024 Financial Results, Raises Guidance](https://www.clinicaltrialvanguard.com/news/koru-medical-systems-announces-q3-2024-financial-results-raises-guidance/)
**Published:** November 14, 2024
**Author:** Jon Napitupulu
**Content:**
[KORU Medical](https://www.clinicaltrialvanguard.com/news/koru-medicals-next-gen-subcutaneous-infusion-system/) Systems, Inc. reported strong third-quarter 2024 financial results and raised its full-year 2024 guidance for revenue, gross margin, and year-end cash. The company achieved $8.2 million in revenue, marking the third consecutive quarter of double-digit growth. This growth was accompanied by improved gross margin and operating leverage. The company attributes its success to a strong recurring chronic subcutaneous immunoglobulin (SCIg) patient base and expanding collaborations in its novel therapies business, fueled by the increasing trend of subcutaneous drug administration.
Third-quarter 2024 net revenues totaled $8.2 million, a 16.8% increase year-over-year. Domestic core revenues grew by 11.7% to $6.4 million, driven by higher consumable and pump volumes from new patients and market share gains. International core revenues saw a 5.1% increase to $1.1 million, primarily due to higher consumable volumes attributed to increased Ig supply, expanded approved indications, and geographic growth. This international growth was partially offset by lower orders from a distribution partner who expedited orders in the second quarter of 2024 due to a BSI regulatory review, which has since been successfully appealed. Novel Therapies revenues reached $0.6 million, a substantial 275.6% increase, fueled by clinical trial orders and progress on non-recurring engineering (NRE) milestones within multiple collaboration agreements.
Gross profit for the quarter reached $5.2 million, a 19% increase, with gross margin expanding by 140 basis points to 63.4%. This improvement stems from a favorable sales mix of clinical trial orders, a more profitable NRE services mix, and higher average selling prices. Operating expenses totaled $6.9 million, a 12.3% increase primarily due to new hires, bonus accruals, and severance related to the elimination of the CTO role. Despite increased operating expenses, the net loss for the quarter was $1.6 million, or ($0.03) per diluted share, compared to a net loss of $1.4 million, or ($0.03) per diluted share, in the prior year period. This was primarily due to an increase in operating expenses of $0.8 million, and a prior year tax benefit of $0.2 million offset by an increase in gross profit of $0.8 million. Adjusted EBITDA improved to ($0.4) million, or ($0.01) per diluted share, compared to ($0.9) million, or ($0.02) per diluted share, in the prior year period. Cash and cash equivalents stood at $8.8 million as of September 30, 2024, reflecting $1.7 million in cash usage during the quarter.
The company provided a reconciliation of adjusted EBITDA and adjusted diluted EPS. KORU Medical Systems focuses on developing, manufacturing, and commercializing innovative, patient-centric large volume subcutaneous infusion solutions. Its flagship product, the FREEDOM Syringe Infusion System, includes the FREEDOM60® and [FreedomEdge](https://www.clinicaltrialvanguard.com/news/koru-medical-systems-data-at-podd-conference-nursing-preference-for-freedomedge-infusion-system/)® Syringe Infusion Drivers, Precision Flow Rate Tubing™, and HIgH-Flo Subcutaneous Safety Needle Sets™. Used for self-administration at home and by healthcare professionals in ambulatory infusion centers, the FREEDOM System first received FDA clearance in 1994. KORU Medical’s Novel Therapies business supports biopharmaceutical companies with products for feasibility/clinical trials during drug development, and offers customized FREEDOM System solutions for clinical and commercial applications across various drug categories. The company will host a conference call and webcast on November 13, 2024, to discuss these results and provide a corporate update.
Source link: http://www.businesswire.com/news/home/20241113344251/en/KORU-Medical-Systems-Inc.-Announces-2024-Q3-Financial-Results-Third-Consecutive-Quarter-of-Double-Digit-Growth-Raises-Full-Year-2024-Guidance
**Categories:** News
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### [Global Healthy Living Foundation Brings Innovative Research to ACR 2024](https://www.clinicaltrialvanguard.com/news/global-healthy-living-foundation-brings-innovative-research-to-acr-2024/)
**Published:** November 15, 2024
**Author:** Jon Napitupulu
**Content:**
The Global Healthy Living Foundation (GHLF) presented groundbreaking research at the 2024 American College of Rheumatology (ACR) Convergence in Washington DC, marking their ninth consecutive year and coinciding with PatientSpot’s 10th anniversary. This patient-powered research registry, formerly known as ArthritisPower, underscores GHLF’s commitment to improving care for individuals with chronic inflammatory diseases. This year’s presentations centered on enhancing care for conditions such as polymyalgia rheumatica (PMR), [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (RA), and [psoriatic arthritis](https://www.clinicaltrialvanguard.com/news/fda-approves-risankizumab-for-pediatric-psoriasis-and-psoriatic-arthritis/) (PsA), while exploring the potential of emerging technologies to improve patient outcomes. This presentation also aligns with CreakyJoints® 25th anniversary, GHLF’s online community for arthritis patients and caregivers, further highlighting their long-term dedication to patient advocacy and research.
A significant portion of GHLF’s research at ACR focused on PMR treatment and addressing gaps in patient care. One study revealed a prevalent dependence on steroids for PMR and PMR with giant cell arteritis (GCA), often without supplementary therapies to mitigate steroids’ long-term side effects. This emphasizes the critical need for continued research into new therapeutics and the importance of clinical trials to explore more balanced and comprehensive treatment strategies. The ultimate goal is to reduce the reliance on steroids and improve the overall treatment experience for PMR patients.
Digital health innovations also played a prominent role in GHLF’s research. The multi-year WEAR study demonstrated how integrating Fitbit data with patient-reported outcomes enhances RA monitoring without increasing patient burden. This approach emphasizes personalized, patient-centered healthcare, where patient-reported data provides valuable insights for guiding treatment decisions and proactive disease management. Another study explored the feasibility of Remote Therapeutic Monitoring (RTM) for musculoskeletal diseases using the PatientSpot app. The positive feedback from both patients and staff suggests that RTM offers significant potential for improving patient engagement and providing continuous support between clinical appointments, facilitating proactive management and timely interventions.
GHLF also showcased research addressing the importance of infection prevention and managing comorbidities associated with chronic inflammatory diseases. These insights aim to support researchers, clinicians, and patients in maintaining disease stability and improving overall health through a holistic approach. In addition to scientific abstracts, GHLF presented patient posters at ACR Convergence, sharing real-world experiences from individuals living with these conditions. These perspectives offer valuable insights into the practical challenges of disease management and inform the development of more responsive, patient-centered research and treatment approaches. One such presentation focused on ChronicHue, a GHLF community supporting Black, Indigenous, and People of Color (BIPOC) individuals with chronic illnesses, addressing health disparities and promoting open health discussions within these communities. Another presentation highlighted the empowering role of self-directed research in personal health decisions, showcasing how a patient utilized systematic research methods to understand and manage their own symptoms. GHLF’s comprehensive research efforts, combined with patient-centered initiatives, demonstrate their commitment to improving the lives of individuals living with chronic illnesses.
Source link: http://www.businesswire.com/news/home/20241114949994/en/Global-Healthy-Living-Foundation-Brings-Innovative-and-Patient-Centered-Polymyalgia-Rheumatica-Psoriatic-and-Rheumatoid-Arthritis-Research-to-ACR-Convergence-2024
**Categories:** News
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### [Cancer Patient Rights: Expanding Lifesaving Care in Illinois](https://www.clinicaltrialvanguard.com/news/cancer-patient-rights-expanding-lifesaving-care-in-illinois/)
**Published:** November 15, 2024
**Author:** Jon Napitupulu
**Content:**
A coalition of prominent cancer organizations, alongside Illinois lawmakers, is advocating for the Illinois Cancer Patients Bill of Rights. This resolution asserts that all Illinois residents diagnosed with cancer deserve access to innovative treatments, advanced care centers, and comprehensive support, regardless of their socioeconomic status.
The Cancer Care is Different Coalition, spearheaded by City of Hope, a nationally recognized cancer research and treatment center, champions this initiative. The coalition comprises organizations like the American Cancer Society Cancer Action Network, Susan G. Komen of Greater Chicago, and the [Leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) & Lymphoma Society of Illinois, among others. They aim to ensure equitable access to life-saving cancer care, a critical need given that many patients lack access to modern treatments due to financial constraints, geographic location, or systemic healthcare barriers.
The Cancer Patients Bill of Rights resolution, introduced by State Representative Marcus Evans and State Senator Mary Edly-Allen, addresses this disparity. It emphasizes providing patients access to cutting-edge research, treatments, and specialized care at advanced cancer centers.
The urgency of this resolution is underscored by Illinois’s cancer statistics. An estimated 23,280 lives were lost to cancer in Illinois this year, with 78,200 new diagnoses projected. The state’s cancer mortality rates surpass national averages for most cancer types, with stark racial disparities. For instance, Black women experience a 48% higher [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) mortality rate than white women, and Black men face a staggering 135% higher [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) mortality rate than white men.
Representative Evans, a cancer survivor himself, highlighted the resolution’s personal significance and emphasized its goal of ensuring every Illinoisan diagnosed with cancer has the best chance of survival through access to quality care and innovative treatments.
The Cancer Patients Bill of Rights outlines several key provisions for Illinois residents facing a cancer diagnosis: Access to advanced cancer centers, regardless of insurance; access to the latest cancer treatments and clinical trials; participation in care decisions; and access to cancer support services. The resolution aims to achieve more equitable and effective cancer care, recognizing that disparate outcomes often stem from unequal access to early diagnosis and appropriate treatment.
This Illinois bill follows City of Hope’s successful advocacy for similar legislation in California, where the California Cancer Patients Bill of Rights and the Cancer Care Equity Act were passed in 2022. The coalition’s efforts reflect the unique nature of cancer care, emphasizing the rapid pace of research and the crucial importance of early detection and optimal treatment for survival. More information about the coalition, the resolution, and patient stories can be found on the Cancer Care is Different website.
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### [Lineage Cell Therapeutics Q3 2024 Results & Update](https://www.clinicaltrialvanguard.com/news/lineage-cell-therapeutics-q3-2024-results-update/)
**Published:** November 15, 2024
**Author:** Jon Napitupulu
**Content:**
Lineage Cell Therapeutics announced its third quarter 2024 financial and operating results, highlighting progress in its allogeneic [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) programs. A key achievement was the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation for OpRegen, a retinal pigment epithelial cell therapy being developed in collaboration with Roche and Genentech for geographic atrophy secondary to age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/). This designation underscores OpRegen’s potential as a transformative treatment.
Alongside this, Lineage advanced its OPC1 program for spinal cord injury, returning it to clinical trials. Furthermore, promising preclinical data from ReSonance, a therapy for sensorineural hearing loss, were presented. The company remains committed to leveraging its cell transplant technology and manufacturing expertise across its clinical and preclinical pipelines.
Financially, Lineage reported $32.7 million in cash, cash equivalents, and marketable securities as of September 30, 2024, anticipating this will fund operations into the first quarter of 2026. Total revenue for the third quarter reached $3.8 million, a significant increase from the $1.2 million reported in the same period of 2023. This increase was primarily attributed to higher collaboration revenue recognized from deferred revenue under the Roche agreement.
Operating expenses totaled $7.6 million, a slight decrease from the $7.9 million reported in the third quarter of 2023. Research and development expenses decreased to $3.2 million, while general and administrative expenses increased to $4.4 million. The decrease in R&D expenses was primarily driven by reductions in spending on the OPC1 and preclinical programs, partially offset by an increase in spending for OpRegen. The increase in G&A was mainly due to higher personnel costs and stock-based compensation.
The company reported a loss from operations of $3.8 million, a substantial improvement from the $6.7 million loss in the same period last year. Other income for the quarter was $0.8 million, compared to an expense of $0.4 million in the prior year, attributed to exchange rate fluctuations. The net loss attributable to Lineage was $3.0 million, or $0.02 per share, compared to a net loss of $7.1 million, or $0.04 per share, in the third quarter of 2023.
Lineage’s balance sheet as of September 30, 2024, shows total assets of $96.6 million and total liabilities of $31.8 million. Shareholders’ equity stood at $64.8 million. The company’s cash flow statement for the nine months ended September 30, 2024, reflects net cash used in operating activities of $16.7 million, partially offset by cash provided by financing activities of $14.1 million.
Lineage’s pipeline focuses on developing “off-the-shelf” cell therapies for unmet medical needs using its proprietary cell-based technology platform. Beyond OpRegen, OPC1, and ReSonance, the pipeline also includes PNC1 for vision loss and RND1, a hypoimmune induced pluripotent stem cell line being developed with Factor Bioscience Limited.
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### [Zura Bio Submits Scleroderma Drug Protocol for FDA Phase 2 Study](https://www.clinicaltrialvanguard.com/news/zura-bio-submits-scleroderma-drug-protocol-for-fda-phase-2-study/)
**Published:** November 15, 2024
**Author:** Jon Napitupulu
**Content:**
Zura Bio, a clinical-stage immunology company, has submitted a Phase 2 study protocol to the FDA for tibulizumab. This drug is a novel dual-action antibody designed to treat systemic sclerosis (SSc) in adults by neutralizing both [IL-17A](https://www.clinicaltrialvanguard.com/news/junshis-psoriasis-drug-js005-succeeds-in-phase-3-trial/) and BAFF. The company believes this dual-pathway approach can address both immune system dysfunction and fibrosis, two key drivers of SSc.
The planned Phase 2 trial is a randomized, double-blind, placebo-controlled study. It will assess the safety, tolerability, and efficacy of tibulizumab in approximately 80 adults with early diffuse cutaneous SSc (dcSSc). Researchers hope to observe improvements in skin and lung symptoms, two significant complications of SSc. The trial includes an open-label extension and is slated to begin in Q4 2024. Zura Bio also submitted an Orphan Drug Designation request to the FDA in the same quarter. It is important to note that tibulizumab is not yet approved by any regulatory authority, and its safety and effectiveness in treating SSc remain unproven.
Systemic sclerosis, also known as scleroderma, is a rare, life-threatening autoimmune disease affecting roughly 300,000 people globally, with approximately 100,000 cases in the United States. The disease is marked by chronic inflammation and progressive fibrosis of connective tissues, primarily impacting the skin and lungs. However, it can also affect other organs like the heart, liver, kidneys, digestive tract, and vascular system. Common symptoms include skin thickening and extreme cold sensitivity in the extremities. Other potential manifestations include muscle issues (numbness and swelling), joint stiffness and reduced mobility, fibrosis in the lungs and heart, kidney problems, and gastrointestinal difficulties such as swallowing problems, heartburn, bloating, constipation, and diarrhea. Currently, treatment options for SSc are limited, highlighting a significant unmet medical need. Only two disease-modifying therapies are FDA-approved for severe SSc-related lung complications, and no treatment effectively addresses the disease across multiple organ systems.
Tibulizumab is an investigational dual-action antibody. It combines elements of Taltz (ixekizumab) and tabalumab, enabling it to neutralize both IL-17A and BAFF. In addition to the planned Phase 2 trial for SSc, tibulizumab is expected to enter Phase 2 clinical development for hidradenitis suppurativa in Q2 2025. Previous Phase 1/1b studies investigated its potential for Sjögren’s syndrome and [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/news/spy072-misses-primary-endpoint-in-rheumatoid-arthritis-study/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/).
Zura Bio is focused on developing innovative dual-pathway antibodies for autoimmune and inflammatory diseases. Its current pipeline includes three assets that have completed Phase 1/1b studies: tibulizumab (ZB-106), crebankitug (ZB-168), and torudokimab (ZB-880). The company aims to demonstrate the efficacy, safety, and convenient dosing of these antibodies in various autoimmune and inflammatory conditions, particularly those with unmet needs.
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### [CHMP Positive on Opdivo + Yervoy for MSI-H/dMMR mCRC](https://www.clinicaltrialvanguard.com/news/chmp-positive-on-opdivo-yervoy-for-msi-h-dmmr-mcrc/)
**Published:** November 18, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Opdivo (nivolumab) combined with Yervoy (ipilimumab) as a first-line treatment. This treatment targets adult patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) unresectable or metastatic colorectal cancer (mCRC). The [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) -8HW trial demonstrated substantial benefits for this patient population. Results revealed a 79% reduction in the risk of disease progression or death compared to standard chemotherapy regimens. This positive CHMP opinion is a significant milestone, paving the way for the European Commission (EC) to review the recommendation and issue a final decision regarding approval within the European Union.
This combination therapy represents a significant advancement, as it is the first dual checkpoint inhibitor approved for first-line treatment of metastatic colorectal cancer in this specific patient group. It addresses a significant unmet need, as current treatments often provide insufficient benefit to the approximately 5-7% of metastatic colorectal cancer patients with dMMR or MSI-H tumors. BMS anticipates a favorable EC decision and aims to make this treatment available to eligible patients in the EU.
The CHMP’s recommendation stems from the CheckMate -8HW trial, where Opdivo plus Yervoy exhibited clinically meaningful improvement in progression-free survival (PFS) compared to investigator’s choice of chemotherapy. The assessment was conducted by Blinded Independent Central Review (BICR). Furthermore, the combination therapy’s safety profile aligns with previously reported data. It appears manageable with established protocols, without new safety signals.
In another development, the combination therapy also demonstrated a statistically significant and clinically meaningful improvement in PFS (per BICR) compared to Opdivo monotherapy across all lines of therapy, as announced in October 2024. The CheckMate -8HW trial, a Phase 3 randomized, open-label study, enrolled 839 patients. It compared Opdivo plus Yervoy to Opdivo alone and investigator’s choice chemotherapy (mFOLFOX-6 or FOLFIRI with or without [bevacizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-lytenava-first-ophthalmic-bevacizumab-for-wet-amd/) or [cetuximab](https://www.clinicaltrialvanguard.com/news/frontier-medicines-presents-preclinical-data-on-3-programs/)) in patients with MSI-H or dMMR unresectable or metastatic colorectal cancer. The trial’s primary endpoints include PFS per BICR for the combination versus chemotherapy in the first-line setting and PFS for the combination versus Opdivo monotherapy across all treatment lines. Secondary endpoints, including overall survival, are still under evaluation. The trial successfully met its other dual primary endpoint of PFS per BICR for Opdivo plus Yervoy compared to Opdivo across all lines of therapy in patients with centrally confirmed MSI-H/dMMR mCRC in a second interim analysis conducted in September 2024. These results are slated for presentation at an upcoming medical conference.
Colorectal cancer (CRC), affecting the colon or rectum, is a prevalent global health concern. It’s the third most diagnosed cancer worldwide and the second leading cause of cancer-related deaths. dMMR occurs when DNA repair proteins are dysfunctional or absent, leading to MSI-H tumors. These tumors, present in roughly 5-7% of metastatic CRC cases, often respond poorly to conventional chemotherapy and are associated with a poor prognosis. BMS is dedicated to improving outcomes for cancer patients, aiming to develop innovative treatments that enhance quality of life and pursue the possibility of a cure. BMS leverages its expertise in immuno-oncology, personalized medicine, and data analysis to achieve these goals, approaching cancer from multiple perspectives.
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### [VIR Biotech HBV Combination Therapy Shows Promise in March Study](https://www.clinicaltrialvanguard.com/news/vir-biotech-hbv-combination-therapy-shows-promise-in-march-study/)
**Published:** November 18, 2024
**Author:** Jon Napitupulu
**Content:**
Vir Biotechnology announced positive end-of-treatment results from Part B of its MARCH Phase 2 clinical study. This study is evaluating combinations of tobevibart and elebsiran, with and without pegylated interferon alfa (PEG-IFNα), for chronic hepatitis B. The study showed promising hepatitis B surface antigen (HBsAg) loss (seroclearance) rates in participants with low baseline HBsAg (<1000 IU/mL) using both combination regimens. These positive efficacy and safety results support further development to explore the potential for a functional cure. Detailed findings will be presented at the American Association for the Study of Liver Diseases ([AASLD](https://www.clinicaltrialvanguard.com/news/promising-new-data-on-wilson-disease-from-monopar-at-aasld/)) The Liver Meeting® in San Diego, CA on November 18th. Vir Biotechnology will also host an investor conference call on November 19th. Chronic hepatitis B (CHB) is a persistent, inflammatory liver disease caused by the hepatitis B virus (HBV). This global health concern affects an estimated 254 million people, with approximately 1.1 million deaths annually attributed to the disease and its complications, which can include cirrhosis, liver failure, and [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/). While CHB treatments exist, there is currently no cure.
The MARCH trial participants received either tobevibart and elebsiran (doublet regimen) or these two medications combined with PEG-IFNα (triplet regimen). The analysis included data from 51 participants in the doublet arm and 27 in the triplet arm. Tobevibart was administered every four weeks at 300 mg, elebsiran at 200 mg every four weeks, and PEG-IFNα weekly at 180 µg. Primary endpoints were HBsAg seroclearance (HBsAg below the lower limit of quantification) and treatment-emergent adverse events (TEAEs) at the end of treatment. Anti-HBs seroconversion (development of anti-HBs≥10 mIU/mL) at treatment end was a secondary endpoint. The results showed HBsAg loss in 39% (7/18) of participants with baseline HBsAg<1,000 IU/mL in the doublet arm and 46% (5/11) in the triplet arm. Overall HBsAg loss rates were 16% (8/51) for the doublet regimen and 22% (6/27) for the triplet regimen across all baseline HBsAg levels. In the doublet regimen group, 50% (4/8) of participants who achieved HBsAg loss also achieved anti-HBs seroconversion. All participants achieving HBsAg loss in the triplet group also achieved anti-HBs seroconversion (100%, 6/6).
Eligible participants who achieved HBsAg seroclearance at the end of treatment discontinued treatment. Functional cure assessment will take place 24 weeks post-treatment discontinuation. The combined tobevibart and elebsiran treatment showed a consistent safety and tolerability profile compared to previous studies, without any new safety concerns arising. Observed TEAEs were generally mild to moderate. Vir Biotechnology aims to develop a functional cure for chronic hepatitis B through a finite treatment regimen. The MARCH data suggests that tobevibart and elebsiran may be able to clear HBsAg and stimulate the immune system to produce antibodies, potentially controlling the virus. The functional cure data expected in 2025 will be crucial for determining the next steps in clinical development. The study results will be presented in an oral presentation titled “Tobevibart (VIR-3434) and elebsiran (VIR-2218) with or without pegylated interferon alfa-2a for the treatment of chronic HBV infection: end of treatment results after 48 weeks of therapy (MARCH study)” during a late-breaking parallel session at AASLD The Liver Meeting® on November 18th.
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### [Boston Pharmaceuticals Positive Phase 2 NASH Data at AASLD 2024](https://www.clinicaltrialvanguard.com/news/boston-pharmaceuticals-positive-phase-2-nash-data-at-aasld-2024/)
**Published:** November 18, 2024
**Author:** Jon Napitupulu
**Content:**
Boston Pharmaceuticals announced positive Phase 2 study results for efimosfermin alfa, a long-acting FGF21 analogue, in patients with stage F2/F3 fibrosis due to metabolic dysfunction-associated steatohepatitis (MASH). The once-monthly treatment showed significant fibrosis improvement without worsening MASH, and MASH resolution without worsening fibrosis over 24 weeks, alongside a favorable tolerability profile. These findings will be presented at the American Association for the Study of Liver Diseases ([AASLD](https://www.clinicaltrialvanguard.com/news/promising-new-data-on-wilson-disease-from-monopar-at-aasld/)) The Liver Meeting in November 2024.
The randomized, double-blind, placebo-controlled study involved 84 participants with biopsy-confirmed F2 or F3 MASH. Results demonstrated that 45.2% of the efimosfermin group achieved fibrosis improvement of at least one stage without MASH worsening, compared to 20.6% in the placebo group. MASH resolution without fibrosis worsening was observed in 67.7% of the efimosfermin group versus 29.4% of the placebo group. Although not statistically significant, 38.7% of the efimosfermin group saw both fibrosis improvement and MASH resolution, compared to 17.6% in the placebo group.
Beyond histological endpoints, efimosfermin also led to rapid and significant improvements in fibrosis biomarkers and reductions in non-invasive markers of liver injury and liver fat. Participants with [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) experienced a clinically meaningful improvement in HbA1c values. Importantly, efimosfermin demonstrated a low discontinuation rate due to adverse events, with a low incidence of gastrointestinal side effects and injection site reactions. The 24-week treatment data will inform future regulatory discussions, and the company plans to advance the clinical program to late-stage development in 2025. An open-label extension study is ongoing, providing an additional 24 weeks of treatment to evaluate long-term safety and efficacy, including immunogenicity.
Efimosfermin targets the core components of hepatic disease, impacting fibrosis improvement, a critical aspect of MASH management. It also offers potential cardiometabolic benefits, including liver fat reduction and improved glycemic control, which are particularly relevant given the high prevalence of obesity and diabetes in MASH patients. The upcoming results from the extension study and a separate advanced fibrosis study will contribute to a comprehensive data package for regulatory discussions prior to pivotal studies.
Efimosfermin alfa is a long-acting FGF21 analogue administered via once-monthly subcutaneous injection. It works by regulating metabolic pathways to reduce liver fat, lessen liver damage and inflammation, and reverse liver fibrosis in MASH patients. MASH, formerly known as non-alcoholic steatohepatitis (NASH), is a growing global health concern linked to obesity and type 2 diabetes. Affecting millions worldwide, MASH is a progressive disease characterized by liver scarring and is associated with various cardiometabolic risk factors. Untreated, it can lead to liver failure, [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/), or death. Boston Pharmaceuticals is focused on developing differentiated therapies for severe liver diseases, with MASH as the primary focus for efimosfermin.
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### [Innate Pharma's Next-Gen ANKET® IPH6501 Highlighted in Science Immunology](https://www.clinicaltrialvanguard.com/news/innate-pharmas-next-gen-anket-iph6501-highlighted-in-science-immunology/)
**Published:** November 19, 2024
**Author:** Jon Napitupulu
**Content:**
Innate Pharma announced preclinical data demonstrating the potential of IPH6501, its proprietary NK cell engager, for B-cell non-Hodgkin lymphoma (B-NHL). In a Phase 1/2 clinical trial, IPH6501, also known as CD20-NKCE-IL2v, enhances NK cell proliferation and cytotoxicity. It has shown activity against various B-NHL cell lines, even those with low CD20 density. In vivo studies using nonhuman primates and mice confirmed its efficacy and revealed its ability to induce peripheral NK cell migration to the tumor site.
IPH6501 demonstrated superior killing efficacy compared to a CD20-targeting T cell engager in preclinical models while exhibiting a reduced toxicity profile compared to typical T cell therapies. The drug’s ability to direct NK cells to tumor sites positions it as a potential breakthrough in immuno-oncology. This data reinforces the ongoing clinical development plan for IPH6501 in Relapsed/Refractory B-NHL.
ANKET® (Antibody-based NK cell Engager Therapeutics), Innate’s proprietary platform, is designed to develop next-generation, multi-specific natural killer (NK) cell engagers for cancer treatment. This technology represents a novel class of molecules designed to stimulate synthetic immunity against cancer.
IPH6501 is the first ANKET® therapeutic to simultaneously engage activating receptors on NK cells (NKp46 and CD16), IL-2R (but not a subunit) through an IL-2 variant, and the CD20 tumor antigen through a single molecule. This mechanism provides NK cells with targeted proliferation and activation signals, boosting their cytotoxic activity against CD20-expressing malignant cells. Preclinical tumor models have shown IPH6501’s superior anti-tumor efficacy compared to approved benchmark antibodies. A Phase 1/2 multicenter trial is currently evaluating the safety and tolerability of IPH6501 in patients with Relapsed and/or Refractory CD20-expressing B-cell Non-Hodgkin’s Lymphoma.
Innate Pharma is a global, clinical-stage biotechnology company focused on developing immunotherapies for cancer. Their innovative approach leverages the innate immune system through three therapeutic avenues: monoclonal antibodies, multi-specific NK Cell Engagers via its ANKET® platform, and Antibody Drug Conjugates (ADCs). Innate Pharma’s portfolio includes [lacutamab](https://www.clinicaltrialvanguard.com/news/lacutamab-gets-fda-breakthrough-therapy-for-sezary-syndrome/) for advanced cutaneous and peripheral T cell lymphomas, [monalizumab](https://www.clinicaltrialvanguard.com/news/innate-pharmas-monalizumab-neocoast-2-phase-2-trial-reports-encouraging-outcomes-in-early-stage-lung-cancer/) (developed with AstraZeneca) for non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/), several ANKET® drug candidates for various tumor types, and IPH4502, a differentiated ADC for solid tumors. The company collaborates with biopharmaceutical companies like Sanofi and AstraZeneca and leading research institutions. Headquartered in Marseille, France, with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq.
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### [Silence Therapeutics Unveils Groundbreaking Zerlasiran Phase 2 Data at AHA 2024](https://www.clinicaltrialvanguard.com/news/silence-therapeutics-unveils-groundbreaking-zerlasiran-phase-2-data-at-aha-2024/)
**Published:** November 19, 2024
**Author:** Jon Napitupulu
**Content:**
Silence Therapeutics recently unveiled end-of-treatment data from its Phase 2 ALPACAR-360 study of zerlasiran, an siRNA therapy, in individuals with atherosclerotic cardiovascular disease (ASCVD) and high lipoprotein(a) \[Lp(a)\] levels. The findings were presented at the American Heart Association Scientific Sessions 2024 and concurrently published in JAMA.
The study explored the efficacy of various zerlasiran dosages and intervals (300 mg every 16 weeks, 300 mg every 24 weeks, and 450 mg every 24 weeks). Results demonstrated significant reductions in Lp(a) concentrations. Over 36 weeks, all three regimens achieved mean time-averaged placebo-adjusted reductions exceeding 80% from baseline. Notably, this study marks the first to employ time-averaged Lp(a) analysis, providing a more comprehensive assessment of treatment effects over time. Maximum Lp(a) reductions even surpassed 90%. Importantly, Lp(a) reductions were sustained 60 weeks after the initial dose, with no new safety concerns identified.
This data will inform the selection of the optimal dose and dosing frequency for forthcoming Phase 3 zerlasiran trials. Elevated Lp(a) affects at least 20% of the global population and represents a substantial contributor to cardiovascular morbidity and mortality. As a genetic risk factor previously untreatable, gene-silencing therapies like zerlasiran offer promising new avenues for patient care.
The ALPACAR-360 study reinforces the potential of zerlasiran, particularly its durable Lp(a) reduction with infrequent dosing. The Phase 2 data suggest zerlasiran’s promise as a novel treatment for individuals with high Lp(a), paving the way for Phase 3 investigations.
Lp(a) is a genetically determined and independent risk factor for cardiovascular disease. While a definitive threshold for high Lp(a) remains to be established, approximately 20% of adults have Lp(a) levels exceeding 125 nmol/L. Increasing evidence points to the role of high Lp(a) in myocardial infarction, stroke, and peripheral arterial disease.
The ALPACAR-360 program aims to evaluate zerlasiran’s ability to reduce cardiovascular event risk in ASCVD patients with high Lp(a). This multicenter, randomized, double-blind, placebo-controlled Phase 2 study included 178 participants with ASCVD and Lp(a) levels ≥125 nmol/L (baseline Lp(a) concentration: 213 nmol/L). Patients were randomly allocated to one of the three zerlasiran doses mentioned earlier or a placebo. The primary endpoint was the time-averaged Lp(a) change from baseline to 36 weeks. Secondary endpoints encompassed time-averaged LDL-C changes and time-averaged Lp(a) changes to 48 and 60 weeks.
Silence Therapeutics, a clinical-stage biotechnology company, is dedicated to developing precision-engineered siRNA therapies to address a range of diseases. Their proprietary mRNAi GOLD™ platform facilitates the creation of siRNAs targeting disease-associated genes in the liver. The company’s pipeline focuses on areas of high unmet medical need, including cardiovascular disease, hematology, and rare diseases. Silence also collaborates with [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) and Hansoh Pharma, among other partners, on research and development initiatives.
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### [ADICET Opens ADI-270 Phase 1 Trial Enrollment for Metastatic Renal Cell Carcinoma](https://www.clinicaltrialvanguard.com/news/adicet-opens-adi-270-phase-1-trial-enrollment-for-metastatic-renal-cell-carcinoma/)
**Published:** November 19, 2024
**Author:** Jon Napitupulu
**Content:**
Adicet Bio has initiated a Phase 1 clinical trial for ADI-270, a novel cancer therapy. This trial focuses on patients with advanced clear cell renal cell carcinoma (ccRCC), a prevalent and aggressive kidney cancer subtype. Solid tumors, like ccRCC, have yet to experience the same therapeutic advancements as blood cancers treated with CAR T cell therapies. ADI-270 offers the potential to change this. Preclinical data suggests ADI-270 effectively infiltrates tumors, resists the immunosuppressive tumor environment, and exhibits potent anti-tumor activity. Preliminary clinical data from this trial is anticipated in the first half of 2025.
This Phase 1 trial is a multicenter, open-label study designed to assess ADI-270 as a monotherapy in adults with relapsed or refractory ccRCC. Participants will receive a single dose of ADI-270 after lymphodepletion, with the potential for a second dose if specific criteria are met. The trial will evaluate the therapy’s safety, tolerability, pharmacokinetics, and anti-tumor activity, including overall response rate, response duration, and disease control rate.
ADI-270 is an “off-the-shelf” allogeneic gamma delta CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/). It targets CD70, a protein highly expressed in various solid and hematological malignancies. This therapy uses the natural receptor CD27 as the binding moiety and incorporates a dominant negative form of the transforming growth factor-β receptor II (dnTGFβRII). This design aims to enhance ADI-270’s resilience against the immunosuppressive tumor microenvironment and improve its persistence by reducing susceptibility to host rejection. With the inherent tumor-infiltrating properties of gamma delta 1 T cells, ADI-270 aims to enhance clinical outcomes for RCC and other CD70+ tumor patients.
Renal cell carcinoma (RCC) comprises the majority of kidney tumors, with clear cell RCC (ccRCC) being the most common and aggressive subtype. CcRCC often spreads to the lungs, liver, and bones in the kidney’s proximal nephron and tubular epithelium. A significant portion of RCC diagnoses are initially locally advanced or metastatic, and a considerable number of patients develop metastatic disease post-nephrectomy. While localized RCC has a high 5-year survival rate, the prognosis significantly worsens for advanced disease. This underscores the urgent need for effective therapies like ADI-270. Adicet Bio, the company developing ADI-270, focuses on allogeneic gamma delta T cell therapies for cancer and autoimmune diseases. They are developing a pipeline of “off-the-shelf” gamma delta T cells engineered with CARs to promote durable responses in patients.
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### [EMA Grants Orphan Drug Designation to Tobevibart and Elebsiran for Chronic Hepatitis Delta](https://www.clinicaltrialvanguard.com/news/ema-grants-orphan-drug-designation-to-tobevibart-and-elebsiran-for-chronic-hepatitis-delta/)
**Published:** November 19, 2024
**Author:** Jon Napitupulu
**Content:**
Vir Biotechnology announced a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) regarding orphan drug designation for tobevibart and elebsiran. These medications are intended for the treatment of chronic hepatitis delta (CHD). This positive opinion stems from promising preliminary Phase 2 SOLSTICE trial data. Twenty-four-week data from this trial will be presented at the [AASLD](https://www.clinicaltrialvanguard.com/news/promising-new-data-on-wilson-disease-from-monopar-at-aasld/) The Liver Meeting in San Diego on November 18, 2024, followed by an investor conference call on November 19, 2024.
CHD, caused by the hepatitis delta virus (HDV), is a severe, progressive liver disease. Considered the most aggressive form of chronic viral hepatitis, it often leads to cirrhosis and liver failure within five years of infection. Currently, no approved treatment exists in the United States, and global treatment options remain limited.
The COMP’s positive opinion highlights the potential of the tobevibart and elebsiran combination to address the significant unmet need in CHD care. Clinical data suggests this combination could improve outcomes for patients with this devastating disease. The European Commission will now review the COMP’s opinion and consider granting orphan drug designation. This designation is reserved for medicines targeting rare, life-threatening, or chronically debilitating conditions lacking satisfactory treatment options. It offers incentives such as specialized scientific advice, reduced fees, and ten years of market exclusivity upon approval.
The U.S. Food and Drug Administration (FDA) granted fast track designation to the tobevibart and elebsiran combination for CHD treatment in June 2024. This designation aims to expedite the development and review of drugs addressing serious conditions with unmet medical needs.
The Phase 2 SOLSTICE trial is a multi-center, open-label, randomized study evaluating the safety, tolerability, and efficacy of tobevibart, both alone and in combination with elebsiran, in CHD patients. Primary endpoints include the proportion of participants with undetectable HDV RNA, ALT normalization, and treatment-emergent adverse events up to specified time points. Secondary endpoints focus on the proportion of participants with undetectable HDV RNA at various time points. Further details are available on [clinicaltrials](https://www.clinicaltrialvanguard.com/article/fda-needs-to-release-clinicaltrials-gov-guidance-now/).gov.
Tobevibart, an investigational monoclonal antibody, targets the hepatitis B surface antigen. It aims to block the entry of hepatitis B and delta viruses into liver cells and reduce circulating viral particles. Engineered for an extended half-life, tobevibart is administered subcutaneously and is currently in clinical development for chronic hepatitis B and delta.
Elebsiran, an investigational siRNA, targets hepatitis B virus RNA, aiming to reduce hepatitis B surface antigen production. It demonstrates potential antiviral activity against both hepatitis B and delta viruses. Administered subcutaneously, elebsiran is also in clinical development for chronic hepatitis B and delta. It marks the first asset from Vir Biotechnology’s collaboration with Alnylam Pharmaceuticals to enter clinical studies.
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### [Otsuka Provides Sibeprenlimab Update for IgA Nephropathy](https://www.clinicaltrialvanguard.com/news/otsuka-provides-sibeprenlimab-update-for-iga-nephropathy/)
**Published:** November 20, 2024
**Author:** Jon Napitupulu
**Content:**
Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) and Otsuka Pharmaceutical Co., Ltd. plan to submit a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) to the U.S. Food and Drug Administration (FDA) for sibeprenlimab in the first half of 2025. This investigational drug aims to treat immunoglobulin A nephropathy (IgA nephropathy) in adults. This decision follows a positive interim analysis from the Phase 3 VISIONARY study and subsequent discussions with the FDA.
Sibeprenlimab, an anti-APRIL monoclonal antibody, targets a crucial step in the immune response that drives IgA nephropathy. It works by inhibiting A Proliferation-Inducing Ligand (APRIL), thus limiting the production of aberrant IgA1 (Gd-IgA1) and the formation of harmful immune complexes. IgA nephropathy is a progressive autoimmune disease affecting the kidneys. It often leads to end-stage kidney disease (ESKD). Previously, sibeprenlimab received Breakthrough Therapy designation based on promising results from the Phase 2 ENVISION clinical trial.
The ongoing Phase 3 VISIONARY study, which is blinded, will continue to monitor changes in kidney function, measured by estimated glomerular filtration rate (eGFR), until early 2026. Additional analyses of the collected data will be performed and shared to fully understand sibeprenlimab’s potential in treating this condition.
Developed and engineered by Visterra, Inc. (an Otsuka subsidiary), sibeprenlimab underwent pre-clinical and early-stage trials at Visterra. The drug’s mechanism of action centers on blocking APRIL, a critical factor in the four-hit pathogenic cascade of IgA nephropathy. APRIL sustains the disease’s progression by stimulating the production of Gd-IgA1 and immune complex formation. Sibeprenlimab’s neutralization of APRIL potentially reduces IgA and Gd-IgA1 levels. This reduction may lead to less auto-antibody production, fewer immune complexes deposited in the kidney, and decreased proteinuria and kidney inflammation. Ultimately, by reducing Gd-IgA1 production, sibeprenlimab aims to slow kidney damage and the progression toward ESKD. This targeted approach addresses a specific driver of nephron loss in IgA nephropathy.
IgA nephropathy, also known as Berger’s disease, primarily affects adults between 20 and 40 years old. Characterized by IgA accumulation in the kidneys, this chronic condition can lead to progressive kidney dysfunction and ultimately ESKD. Although supportive care exists for managing symptoms, ongoing research is vital for improving understanding and treatment options.
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### [AgelessRx Enters Sleep Health Market](https://www.clinicaltrialvanguard.com/news/agelessrx-enters-sleep-health-market/)
**Published:** November 20, 2024
**Author:** Jon Napitupulu
**Content:**
[AgelessRx](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/), a leading telehealth platform specializing in longevity, has expanded its services to include sleep health. Recognizing the crucial role of sleep in overall well-being and longevity, the company now offers personalized sleep treatment plans incorporating lifestyle recommendations, prescription medication, and over-the-counter supplements. This expansion builds upon AgelessRx’s commitment to proactive healthcare and redefining aging.
The new sleep program emphasizes a research-backed approach, tailoring solutions to individual needs and integrating them into holistic treatment plans focused on extending lifespan and healthspan. Understanding that insufficient sleep undermines the benefits of diet and exercise, AgelessRx aims to make quality sleep a central component of everyone’s longevity routine.
Poor sleep is a widespread problem, affecting one in three Americans, and is often exacerbated by the demands of modern life. It significantly increases the risk of chronic diseases like heart disease, diabetes, and [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), all of which are linked to shorter lifespans. AgelessRx seeks to address this growing health crisis by offering practical and effective solutions.
The company’s new sleep offerings include Trazodone, a prescription sleep aid, and Tran-Q Sleep, a natural, melatonin-free, over-the-counter supplement. Both are non-habit forming and support the four key pillars of healthy sleep: quantity, quality, regularity, and timing. AgelessRx’s clinical team thoroughly reviews each patient’s medical history, needs, and goals to determine the most suitable treatment. These options cater to a wide range of individual sleep needs and aim to improve overall sleep architecture – the cyclical progression through different sleep stages.
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### [Asimov, Revopsis Sign Licensing Deal for Multispecific-Expressing Cell Line](https://www.clinicaltrialvanguard.com/news/asimov-revopsis-sign-licensing-deal-for-multispecific-expressing-cell-line/)
**Published:** November 20, 2024
**Author:** Jon Napitupulu
**Content:**
Asimov, a synthetic biology company focused on therapeutic design and manufacturing, has entered into a licensing agreement with RevOpsis Therapeutics. RevOpsis specializes in developing next-generation multispecific biologics for ophthalmic therapies. This partnership stems from a successful cell line development campaign using Asimov’s CHO Edge System for RevOpsis’s lead asset, RO-104.
The agreement grants RevOpsis the right to utilize the CHO Edge System for the development and commercialization of RO-104. This innovative, first-in-class fully modular tri-specific biologic targets retinal vascular diseases. RO-104 is engineered to simultaneously address the three primary angiogenic pathways (VEGF-A, VEGF-C, and Ang-2) involved in the progression of these diseases, including neovascular age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (AMD) and diabetic macular edema. RevOpsis anticipates initiating IND-enabling studies for RO-104 in January 2025.
Asimov’s CHO Edge System offers a comprehensive solution for bioproduction. It includes a GMP-banked CHO-K1 glutamine synthetase (GS) knockout host cell line, with a GS-Fut8 double knockout option for afucosylated antibody production. Furthermore, the system provides a hyperactive transposase, a vast library of over a thousand characterized genetic parts for vector design, and sophisticated AI and biophysics models. This combination enables reliable generation of stable cell lines exhibiting high titer and superior product quality. The system also facilitates the optimization of expression vectors and bioreactor processes for various biologic modalities.
This licensing agreement signifies the growing adoption of Asimov’s CHO Edge System. The platform will serve as a foundational element in RevOpsis’s bioproduction process for RO-104, a therapy aimed at addressing significant unmet needs within a growing patient population. Asimov is committed to supporting RevOpsis’s biologic development throughout clinical trials. RevOpsis, dedicated to the rapid discovery and development of novel multispecific therapies, leverages its modular RevMod™ Platform comprised of fully human libraries. The integration of Asimov’s CHO Edge System is expected to significantly enhance the development and commercialization of RO-104 for the treatment of retinal vascular diseases.
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### [Immunis Achieves Breakthrough in Muscle Atrophy Trial](https://www.clinicaltrialvanguard.com/news/immunis-achieves-breakthrough-in-muscle-atrophy-trial/)
**Published:** November 20, 2024
**Author:** Jon Napitupulu
**Content:**
Immunis, Inc., a clinical-stage biotechnology company, has successfully completed a Phase 1/2a clinical trial of [IMM01](https://www.clinicaltrialvanguard.com/news/immunis-studies-canine-muscle-atrophy/)-STEM, a novel multi-active biologic designed to address age and disease-related immune dysregulation. The trial focused on the safety, tolerability, and preliminary efficacy of IMM01-STEM in reversing muscle atrophy in older adults with knee [osteoarthritis](https://www.clinicaltrialvanguard.com/news/nih-funds-trial-for-non-surgical-knee-osteoarthritis-relief/). Initial data suggest positive outcomes with no reported severe adverse events among the nine participants. A manuscript detailing the complete results is currently in development and expected to be published early next year.
Building on the promising findings, Immunis is already planning a Phase 2b clinical trial, STEM-MYO, to further investigate the functional efficacy signals observed in the Phase 1/2a study. This larger, randomized, controlled study will again focus on elderly individuals with sarcopenic knee osteoarthritis.
Concurrent with the STEM-MYO program, Immunis has received FDA clearance for a separate Phase 2 clinical trial, STEM-META. This trial will evaluate the efficacy of IMM01-STEM compared to a placebo in elderly individuals with sarcopenic [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), specifically focusing on improvements in muscle and overall physical function. The goal is to enhance quality of life by restoring physical capabilities, potentially reducing fall risk, lessening reliance on caregivers, and enabling continued workforce participation.
Immunis is a privately held company dedicated to developing innovative immunomodulatory stem cell-derived biologics to combat the effects of age and disease-related immune decline. Their investigational product line utilizes advanced technology to produce multi-active biologics, delivering a therapy comprised of natural, human immune modulators in physiologically relevant concentrations.
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### [Pfizer Appoints New Chief Scientific Officer](https://www.clinicaltrialvanguard.com/news/pfizer-appoints-new-chief-scientific-officer/)
**Published:** November 21, 2024
**Author:** Jon Napitupulu
**Content:**
Pfizer has appointed Chris Boshoff, M.D., PhD, as its new Chief Scientific Officer and President of Research & Development, effective January 1, 2025. Dr. Boshoff, currently Chief Oncology Officer and Executive Vice President, will succeed Dr. Mikael Dolsten. In this new role, Dr. Boshoff will report to Chairman and CEO Albert Bourla, overseeing all research and development functions across therapeutic areas. He will also retain his position on Pfizer’s Executive Leadership Team.
The oncology R&D organization will maintain its integrated structure. Roger Dansey, M.D., will serve as Interim Chief Oncology Officer, reporting to Dr. Boshoff, while assisting in the search for a permanent replacement. Dr. Dansey plans to retire after the transition is complete and will hand over his current responsibilities to Johanna Bendell, M.D., incoming Chief Development Officer for Oncology, who is joining Pfizer from Roche in 2025.
Dr. Boshoff has an extensive history at Pfizer, spanning over 11 years. During his tenure, he has contributed to the approval of 24 innovative medicines and biosimilars across more than 30 indications. His prior roles include Chief Development Officer for Oncology and Rare Disease, and Head of Development Japan. Before joining the pharmaceutical industry, he was the founding Director of the University College London Cancer Institute. His educational background includes a medical degree from the University of Pretoria, a Ph.D. from the Institute of Cancer Research in London, and training as a medical oncologist at the Royal Marsden and Royal Free Hospitals in London. He is also a Fellow of the U.K. Academy of Medical Sciences.
Dr. Roger Dansey joined Pfizer through the Seagen acquisition and currently serves as Chief Development Officer, Oncology. His previous experience includes serving as Chief Medical Officer and interim CEO at Seagen, where he led clinical development for several cancer therapies. Before Seagen, he led Clinical Oncology Research at Merck, overseeing [KEYTRUDA](https://www.clinicaltrialvanguard.com/news/merck-eisais-shock-keytruda-lenvima-fail/)®i registration efforts.
Dr. Johanna Bendell brings significant experience to Pfizer as Global Head of Oncology, Pharma Research and Early Development at Roche. Her background includes leadership positions at the Sarah Cannon Research Institute, Duke University Medical Center, and Harvard Medical School. She holds a medical degree from The University of Chicago Pritzker School of Medicine and a Bachelor of Science in Biochemistry from the University of Chicago. Her medical training encompasses a Brigham and Women’s Hospital residency and a Dana-Farber Cancer Institute oncology fellowship.
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### [Real-World Data Addresses Cancer Care Gaps in Older Adults](https://www.clinicaltrialvanguard.com/news/real-world-data-addresses-cancer-care-gaps-in-older-adults/)
**Published:** November 21, 2024
**Author:** Jon Napitupulu
**Content:**
COTA, Inc., a real-world data (RWD) and analytics company specializing in oncology, and Memorial Sloan Kettering Cancer Center (MSK) will present data from two Diffuse [Large B-Cell Lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-Cell Lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) ([DLBCL](https://www.clinicaltrialvanguard.com/news/genmabs-epcoritamab-plus-lenalidomide-meets-phase-3-goal-in-relapsed-dlbcl/)) studies at the 66th American Society of Hematology Annual Meeting (ASH) in December 2024. The studies focus on older DLBCL patients, aiming to improve cancer care plans for this often underrepresented demographic.
DLBCL, a blood cancer, frequently affects older individuals. While the median diagnosis age is 66, almost 30% of cases occur in patients over 75. Although curable with chemotherapy in up to 70% of cases, survival rates significantly decline with age. A major challenge is the underrepresentation of older adults in randomized control trials (RCTs). Only 9% of RCT participants are 65 or older, and a mere 1% are over 75. This stems from factors like age, comorbidities, and the need for immediate therapy in aggressive cancer forms, making trial participation difficult. Standard exclusion criteria in clinical trials often prevent older, less fit individuals, particularly those with aggressive subtypes, from participating. Consequently, there’s a lack of clear understanding regarding their care and outcomes.
RWD offers a valuable alternative, providing insights into treatment patterns for older populations and informing future trial designs. It can also illuminate how older patients respond to new treatments and how to manage end-of-life care best. Research presented at ASH revealed that older adults with DLBCL who began treatment immediately after diagnosis had poorer outcomes, including shorter times to needing further therapy and lower overall survival rates, compared to those with a longer interval between diagnosis and initial treatment. These patients also tended to be treated in academic centers, had higher ECOG scores (indicating poorer physical function), and presented with high-risk clinical features.
A second study found that only a small percentage of older DLBCL patients received a documented hospice or palliative care referral. The highest referral rate (38%) was among patients over 90, highlighting the need for research into the value of end-of-life care. This research leverages MSK’s clinical expertise and COTA’s RWD from electronic health records, demonstrating the potential of AI and real-world data to enhance understanding of treatment responses and end-of-life care in older patients. Notably, over 85% of the study data originates from community care sites, reflecting real-world cancer care across America. Including community-based data ensures that insights apply to all demographics, regardless of where they receive treatment. High-quality, representative data can empower cancer researchers and oncologists with more accurate outcome predictions, facilitate better-shared decision-making, and personalize palliative oncology and end-of-life care. The research teams hope these findings will stimulate further research and equip oncologists with stronger clinical evidence to treat older patients throughout their care journey.
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### [Alisertib in HR+, HER2- Metastatic Breast Cancer: Phase II Trial Launch](https://www.clinicaltrialvanguard.com/news/alisertib-in-hr-her2-metastatic-breast-cancer-phase-ii-trial-launch/)
**Published:** November 21, 2024
**Author:** Jon Napitupulu
**Content:**
Puma Biotechnology initiated its ALISertib in CAncer (ALISCA™-Breast1) Phase II trial (PUMA-ALI-1201; NCT06369285). This trial investigates alisertib combined with endocrine therapy for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-negative) recurrent or metastatic [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). The study targets patients previously treated with CDK 4/6 inhibitors and at least two prior lines of endocrine therapy in the recurrent or metastatic setting.
The ALISCA™-Breast1 trial will enroll up to 150 patients, randomly assigned to receive one of three alisertib dosages (30mg, 40mg, or 50mg) twice daily on days 1-3, 8-10, and 15-17 of a 28-day cycle. This treatment will be administered alongside the investigator’s choice of endocrine therapy. Mandatory blood and tissue samples will be collected for biomarker analysis. The trial’s primary objective is to identify the optimal alisertib dose when combined with selected endocrine therapy. Key endpoints include objective response rate, response duration, disease control rate, progression-free survival, and overall survival. Puma will also evaluate these endpoints within biomarker subgroups to identify any correlations with treatment response. This biomarker analysis will occur concurrently with the clinical trial.
Puma plans an interim analysis to assess safety and efficacy. Depending on the trial’s results, Puma intends to consult with the U.S. Food and Drug Administration to discuss a potential approval pathway for alisertib in this specific breast cancer type. After determining the optimal alisertib dose, Puma will collaborate with global regulatory agencies to design a pivotal Phase III trial. This subsequent trial is anticipated to be a randomized study comparing alisertib plus investigator’s choice endocrine therapy against placebo plus investigator’s choice endocrine therapy in the same patient population.
Puma believes existing data from previous alisertib trials, including a monotherapy trial, the TBCRC 041 trial (testing alisertib alone and with fulvestrant), and a randomized trial of alisertib plus paclitaxel versus paclitaxel alone, demonstrate alisertib’s activity in HER2-negative, HR+ metastatic breast cancer, particularly within specific biomarker subgroups. Initial data from the ALISCA™-Breast1 trial is expected in 2025.
Puma Biotechnology focuses on developing and commercializing innovative cancer treatment products. The company holds global development and commercialization rights to NERLYNX® (neratinib), an FDA-approved treatment for extended adjuvant treatment of early-stage HER2-overexpressed/amplified breast cancer following adjuvant [trastuzumab](https://www.clinicaltrialvanguard.com/news/zanidatamab-doubles-survival-in-her2-positive-gastric-cancer-trial/)-based therapy, and for use in combination with capecitabine for advanced or metastatic HER2-positive breast cancer in patients previously treated with two or more anti-HER2 regimens. NERLYNX also holds European Commission marketing authorization for extended adjuvant treatment in a similar patient population. Puma also licensed alisertib, an aurora kinase A inhibitor, in September 2022, initially focusing its development on [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) and breast cancer. This led to the initiation of ALISCA™-Lung1, a Phase II alisertib monotherapy trial for extensive-stage small cell lung cancer, in February 2024, followed by the ALISCA™-Breast1 trial in November 2024.
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### [Forte Biosciences Raises $53M to Advance Autoimmune Treatment](https://www.clinicaltrialvanguard.com/news/forte-biosciences-raises-53m-to-advance-autoimmune-treatment/)
**Published:** November 21, 2024
**Author:** Jon Napitupulu
**Content:**
Forte Biosciences, Inc., a clinical-stage biopharmaceutical company specializing in autoimmune and related diseases, has raised $53 million in an oversubscribed equity financing. This substantial investment will fuel the continued clinical development of FB-102, a promising anti-[CD122](https://www.clinicaltrialvanguard.com/news/argenx-to-acquire-forte-biosciences-for-2-2-billion-gains-fb102-antibody/) monoclonal antibody therapeutic candidate. The financing attracted significant interest from new and existing investors, including prominent firms like OrbiMed, Janus Henderson Investors, and Tybourne Capital Management. This capital infusion strengthens Forte’s financial position, enabling the company to pursue multiple autoimmune indications for FB-102.
A study involving healthy volunteers has recently concluded, and a celiac disease trial is currently in progress, with preliminary results anticipated in the second quarter of 2025. The company also plans to explore FB-102’s potential in other autoimmune conditions within the next year. These developments position 2025 as a potentially pivotal year for FB-102’s clinical progress. Forte Biosciences will host an R&D Day on December 3rd to provide further insights into its research and development activities. Details regarding the event will be shared soon.
TD Cowen spearheaded the financing as the lead placement agent, with Guggenheim Securities providing capital markets advisory services. Chardan, Rodman & Renshaw, and Brookline Capital Markets served as co-placement managers. Forte Biosciences focuses on developing FB-102, a proprietary anti-CD122 monoclonal antibody, for various autoimmune and related diseases. The securities in this financing have not been registered under the Securities Act of 1933 and cannot be offered or sold in the United States without proper registration or an applicable exemption. Forte Biosciences has agreed to file a registration statement with the SEC for investors’ resale of the securities.
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**Categories:** News
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### [Laekna and Lilly Partner to Develop Novel Obesity Treatment](https://www.clinicaltrialvanguard.com/news/laekna-and-lilly-partner-to-develop-novel-obesity-treatment/)
**Published:** November 21, 2024
**Author:** Jon Napitupulu
**Content:**
Laekna, a global biotechnology company specializing in metabolic and cancer drug development, has partnered with Eli Lilly and Company to expedite the development of LAE102. This novel Activin Receptor Type 2A (ActRIIA) antagonistic monoclonal antibody (mAb) targets muscle-preserving weight loss in individuals with [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/). This collaboration aims to address the global obesity epidemic and improve the lives of those affected.
The partnership centers on the development and clinical evaluation of LAE102, a potential first-in-class therapy for obesity. ActRIIA plays a vital role in muscle regeneration and lipid metabolism. Pre-clinical models have demonstrated LAE102’s ability to increase lean mass and decrease fat mass simultaneously. When combined with a GLP1R agonist, LAE102 shows promise in further reducing fat mass while regaining lean mass often lost with GLP1R agonist treatment. This suggests LAE102 may be a valuable addition to existing weight management strategies, offering a more comprehensive approach to weight control. By merging Laekna’s innovative research with Lilly’s extensive experience in metabolic diseases, the collaboration strives to elevate the standard of care for obesity. Lilly will leverage its Catalyze360-ExploR&D platform to accelerate LAE102’s development.
LAE102, discovered by Laekna, targets ActRIIA, a key receptor in muscle regeneration and lipid metabolism. Pre-clinical studies show LAE102 increases lean mass while decreasing fat mass. Combined with a GLP1R agonist, it further reduces fat mass and restores lean mass lost through GLP1R agonist treatment. This makes LAE102 a promising candidate for quality weight control, focusing on fat reduction while preserving muscle. The potential of blocking the Activin-ActRII pathway lies in promoting muscle regeneration and decreasing fat mass. Laekna is developing additional drug candidates, including LAE103 (an ActRIIB-selective antibody) and LAE123 (a dual ActRIIA/IIB inhibitor), to maximize the therapeutic potential of targeting ActRII receptors.
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**Categories:** News
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### [Northstar & Cellectar Sign Actinium-225 Supply Deal](https://www.clinicaltrialvanguard.com/news/northstar-cellectar-sign-actinium-225-supply-deal/)
**Published:** November 22, 2024
**Author:** Jon Napitupulu
**Content:**
[NorthStar](https://www.clinicaltrialvanguard.com/news/northstar-and-convergent-expand-collaboration-on-conv01-%ce%b1/) Medical Radioisotopes and [Cellectar Biosciences](https://www.clinicaltrialvanguard.com/news/promising-pancreatic-cancer-data-from-cellectar-biosciences/) forged a strategic partnership which centers around the supply of non-carrier-added (n.c.a.) Actinium-225 (Ac-225). NorthStar, a leader in radiopharmaceutical development and production, will provide Cellectar, a clinical-stage biopharmaceutical company focused on cancer treatments, with a secure supply of this crucial radioisotope. Cellectar will integrate the Ac-225 into its Phospholipid Ether (PLE) delivery platform. This platform is designed to optimize the delivery of radioisotopes and can be tailored for specific tumor types.
This collaboration is significant for the radiopharmaceutical industry as it addresses the scarcity of Ac-225, a critical component in emerging alpha-emitting radiotherapies. The increased availability of n.c.a. Ac-225 could accelerate the development and commercialization of novel cancer treatments, particularly for challenging cancers like [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/). This secure supply chain fostered by the agreement allows companies like Cellectar to focus on research and clinical development, potentially leading to faster breakthroughs in the field. The partnership highlights the growing importance of strategic collaborations in overcoming supply chain limitations and advancing innovative radiopharmaceutical technologies.
For physicians and patients, this partnership holds the promise of new, more effective cancer therapies. Cellectar’s lead alpha emitter program, CLR 121225, which utilizes Ac-225, has shown preclinical promise in treating various solid tumors, including pancreatic and triple-negative breast cancers. The anticipated commencement of human clinical trials for CLR 121225 in 2025 offers a beacon of hope for patients battling these aggressive cancers. This collaboration may eventually lead to more targeted and potent treatments with fewer off-target effects, ultimately improving patient outcomes and quality of life. The increased availability of Ac-225 thanks to NorthStar’s production capabilities could pave the way for a wider range of alpha-emitting radiotherapies becoming accessible to patients, offering new treatment options for various cancers.
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### [Amprion's Landmark Lancet Neurology Study](https://www.clinicaltrialvanguard.com/news/amprions-landmark-lancet-neurology-study/)
**Published:** November 22, 2024
**Author:** Jon Napitupulu
**Content:**
A groundbreaking study published in The Lancet Neurology demonstrates the ability of a novel synuclein seed amplification assay (synSAA) to differentiate between Type 1 and Type 2 synuclein seeds. This differentiation allows for significantly improved diagnostic accuracy for multiple system atrophy (MSA), a fatal neurodegenerative disease often misdiagnosed as [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease (PD), dementia with Lewy bodies (DLB), or other related disorders. The new synSAA classifies synuclein seeds based on fluorescence levels. Type 1 seeds, associated with PD, DLB, and idiopathic REM sleep behavior disorder (iRBD), exhibit high fluorescence. In contrast, Type 2 seeds, linked to MSA, display intermediate fluorescence. The study, conducted across multiple international medical institutions, achieved 100% agreement with the gold standard of pathological analysis using both brain tissue and cerebrospinal fluid (CSF) samples.
This development has significant implications for the neurodegenerative disease field. Current diagnostic methods for synucleinopathies often rely on clinical symptoms, which can overlap significantly between different conditions. This overlap frequently leads to misdiagnosis and delays in appropriate treatment. The high accuracy demonstrated by the novel synSAA offers the potential for earlier and more precise diagnoses, differentiating MSA from other similar synucleinopathies. This improved diagnostic capability may facilitate the development of more targeted therapies and accelerate clinical trials by ensuring appropriate patient stratification. Earlier and accurate diagnosis allows for earlier intervention, potentially impacting disease progression and ultimately improving patient outcomes.
For physicians, this advance offers a crucial tool for addressing the diagnostic challenges posed by synucleinopathies. The novel synSAA provides an objective, biological marker that can differentiate MSA from other similar disorders, reducing reliance on clinical symptoms alone. This enhanced diagnostic accuracy translates into more timely and appropriate patient treatment strategies. The new synSAA offers hope for a faster, more definitive diagnosis for patients suspected of having a synucleinopathy. This reduces the uncertainty and anxiety associated with a prolonged diagnostic process. Furthermore, accurate identification of MSA enables patients to access appropriate care, support services, and potentially enroll in clinical trials specifically targeting this devastating disease. Early diagnosis allows patients and their families to make informed decisions about their care and future, ultimately improving their quality of life.
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### [Omeros Corp. BLA Resubmission Update](https://www.clinicaltrialvanguard.com/news/omeros-corp-bla-resubmission-update/)
**Published:** November 22, 2024
**Author:** Jon Napitupulu
**Content:**
[Narsoplimab](https://www.clinicaltrialvanguard.com/news/omeros-announces-narsoplimab-trial-endpoint-update/), a first-in-class antibody targeting MASP-2, the effector enzyme of the complement lectin pathway, is under development for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Omeros has received feedback from the U.S. Food and Drug Administration (FDA) on its revised statistical analysis plan (SAP) for the [biologics license application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) resubmission. No further presubmission information requests are pending, and no other known impediments to resubmission exist. An independent statistical group will conduct the primary endpoint and other analyses using the data prepared. If these analyses support resubmission, the BLA will be finalized and resubmitted as soon as possible.
This development signifies a potentially critical advancement in treating TA-TMA, a severe complication following hematopoietic stem cell transplantation. Currently, limited effective treatment options exist, and TA-TMA carries a high mortality rate. If the resubmitted BLA is approved, narsoplimab could become a crucial therapy, offering a new mechanism of action and potentially improving outcomes for patients with this life-threatening condition. This progress could also stimulate further research into complement-mediated diseases and their treatment, possibly leading to new therapies for other related conditions.
A potential approval of narsoplimab would provide a much-needed treatment option for managing TA-TMA in post-transplant patients. It could improve patient survival rates and reduce the severity of complications associated with this condition. For patients undergoing hematopoietic stem cell transplantation, narsoplimab represents potential hope for a safer and more effective way to manage the risk of developing TA-TMA. A successful treatment could significantly improve their quality of life and reduce the morbidity and mortality associated with this devastating complication. The availability of a targeted therapy could also lead to earlier diagnosis and intervention, potentially mitigating the long-term effects of TA-TMA. The progress of narsoplimab through the regulatory process offers a significant step towards addressing a critical unmet medical need in the transplant community.
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**Categories:** News
---
### [eClinical Solutions: Top Leader in Everest Group's Life Sciences D&A Assessment](https://www.clinicaltrialvanguard.com/news/eclinical-solutions-top-leader-in-everest-groups-life-sciences-da-assessment/)
**Published:** November 22, 2024
**Author:** Jon Napitupulu
**Content:**
Everest Group has positioned eClinical Solutions as a Leader in [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) Clinical Data and Analytics Platforms in their 2024 PEAK Matrix® Assessment. The assessment evaluated 18 providers based on market impact, vision, and capability, highlighting eClinical Solutions’ platform, elluminate, for its ability to streamline clinical data management and accelerate drug development. eClinical Solutions achieved the highest designation within the Leader category. The assessment emphasized the platform’s scalability, flexibility, ease of deployment, user-friendliness, and robust data integration capabilities.
This recognition signifies a shift in the life sciences industry towards unified clinical data and analytics platforms. Increasingly complex clinical trials, coupled with growing data volumes and the rise of decentralized and hybrid trial designs, necessitate comprehensive data management solutions. This trend pushes platform providers to enhance their end-to-end data analytics capabilities, integrating AI and generative AI to automate tasks, generate insights, and improve quality and risk management. The industry’s focus on reducing cycle times underscores the importance of efficient data management and analysis in bringing new therapies to patients faster.
Clinical data and analytics platform advancements translate to accelerated drug development timelines. By streamlining data management, platforms like Elluminate can contribute to bringing new treatments to market faster. These platforms’ enhanced efficiency and data-driven insights can ultimately improve patient outcomes. The increased adoption of unified platforms and integration of AI-driven technologies also promise to enhance the quality and safety of clinical trials, further benefiting patients involved in research. Fueled by efficient data management and analysis, this accelerated development process holds significant potential for addressing unmet medical needs and improving overall patient care. The focus on optimizing clinical trials through advanced data platforms contributes to a more efficient and effective drug development process, leading to improved access to innovative therapies.
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**Categories:** News
---
### [Unleashing Patient Insights: Transforming Clinical Trials with Patient Perspectives](https://www.clinicaltrialvanguard.com/conference-coverage/unleashing-patient-insights-transforming-clinical-trials-with-patient-perspectives/)
**Published:** November 26, 2024
**Author:** Moe Alsumidaie
**Content:**
At [SCOPE Summit Europe 2024](https://www.scopesummiteurope.com/ "SCOPE Summit Europe 2024"), industry leaders gathered to explore the transformative potential of patient perspectives in decentralized clinical trials (DCTs). The session, led by Ylenia Paleari and Marisa Minetti from Chiesi Farmaceutici SpA, focused on how integrating patient insights can enhance patient-centric clinical research. The discussion highlighted the pressing challenges of patient recruitment and retention in traditional clinical trials and proposed innovative solutions through a decentralized approach. The session aimed to pave the way for a more inclusive and effective clinical trial landscape by leveraging digital tools and fostering collaboration with patients and healthcare professionals.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#bridging-patient-centric-research-and-decentralized-trials)Bridging Patient-Centric Research and Decentralized Trials
The session served as a crucial platform to discuss integrating patient-centric research with decentralized clinical trials. This shift is becoming increasingly vital in the clinical research field. Ylenia Paleari and Marisa Minetti highlighted the industry’s significant challenges in patient recruitment and retention, often due to the cumbersome nature of traditional clinical trial models. They outlined the complex journey of a clinical trial, which begins with the design phase involving patient population identification, setting objectives, and planning assessments. This journey continues through stages of awareness, interest, and intent, leading to recruitment, a multifaceted process involving eligibility checks conducted in clinics, over the phone, or online. Informed consent, traditionally involving a paper-based Patient Information Sheet (PIS), can be time-consuming and may deter potential participants. The decentralized approach aims to streamline these processes by leveraging digital tools and remote communication, making participation more accessible and less burdensome for patients.
The speakers emphasized that the decentralized model offers significant advantages, such as increased accessibility and convenience, but also presents challenges that must be addressed to ensure its success. One of the primary benefits of DCTs is the reduction in geographical and mobility constraints for patients. Traditional trials often require participants to travel to specific sites for study visits, which can be a significant barrier for those with limited mobility or those living in remote areas. By incorporating telemedicine and [digital health technologies](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/), DCTs can minimize the need for travel, allowing patients to participate from the comfort of their homes. However, the shift to a decentralized model must be carefully managed to maintain the essential human element of clinical trials. Both patients and HCPs strongly preferred retaining some level of face-to-face interaction, which is crucial for building trust and ensuring that patients feel supported throughout the trial process. The challenge lies in finding the right balance between digital innovation and personal interaction.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#reducing-travel-burden-and-enhancing-flexibility)Reducing Travel Burden and Enhancing Flexibility
The session’s significant focus was strategies to reduce the travel burden on patients while maintaining the integrity of the doctor-patient relationship. The speakers presented data from a study involving 108 patients and 29 caregivers from the USA and Europe, which provided valuable insights into patient preferences and expectations. Participants in the study expressed a high level of interest in a Direct-to-Patient (D-t-P) service, which would allow them to receive study medication and conduct assessments at home. This service was seen as a way to alleviate the logistical challenges associated with frequent travel to study centers. Patients highlighted the benefits of having a better balance between their personal and working lives and the flexibility to choose when to visit the study center based on their own needs.
The speakers emphasized that implementing such services requires careful planning and patient collaboration to ensure the technology is user-friendly and meets their needs. The industry can enhance patient satisfaction and engagement by doing so, ultimately leading to more successful trial outcomes. The D-t-P service represents a significant shift towards patient-centricity, offering participants greater control over their involvement in clinical trials. This approach reduces the logistical burden on patients and empowers them to make decisions that align with their personal circumstances and preferences. The industry can foster a more inclusive and supportive clinical trial environment by prioritizing patient needs and preferences.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#building-trust-in-clinical-research)Building Trust in Clinical Research
Building trust in clinical research was identified as a cornerstone of successful patient-centric and decentralized trials. The speakers outlined Chiesi’s approach to fostering trust through various initiatives designed to enhance the patient experience and ensure transparency throughout the trial process. Chiesi’s strategy includes implementing hybrid DCT studies, which combine elements of traditional and decentralized trials to offer a flexible and patient-friendly approach. The company also provides a patient concierge service, which offers personalized support to participants, helping them navigate the complexities of the trial process.
Additionally, Chiesi has introduced direct-to-patient medication delivery, allowing patients to receive their study medication at home. This approach reduces the need for travel and ensures that patients have timely access to their medication, which is crucial for maintaining treatment adherence. The speakers highlighted the importance of clear communication and transparency in building patient trust. By providing lay summaries of study results and sending thank-you letters to participants, Chiesi aims to acknowledge the valuable contributions of patients and foster a sense of partnership in the research process. The company seeks to create a more inclusive and patient-centric clinical trial environment through these efforts.
## [](#conclusion)Conclusion
The session concluded with a call to action for the industry to adopt innovative digital tools and collaborate with patients to enhance the usability of decentralized trials. The industry can overcome existing challenges by integrating patient insights and creating a more inclusive and effective clinical trial landscape. The session highlighted the critical role of patient perspectives in shaping the future of clinical research, paving the way for more patient-centric and decentralized approaches.
**Categories:** Article: Conference Coverage
---
### [Advancing Immunotherapy: Insights from Ymmunobio CEO](https://www.clinicaltrialvanguard.com/executiveinterviews/advancing-immunotherapy-insights-from-ymmunebio-ceo/)
**Published:** November 27, 2024
**Author:** Moe Alsumidaie
**Content:**
In this discussion, Moe Alsumidaie interviews Peter Schiemann, CEO and Chairman of [Ymmunobio](https://www.ymmunobio.com/ "Ymmunobio"), about the company’s innovative approaches to targeted therapy. Dr. Schiemann shares insights on targeting cancer at a molecular level, regulatory strategies, and plans to enhance patient outcomes and attract investment.
## [](#moe-alsumidaie-how-is-ymmunobio-advancing-targeted-therapies-to-treat-solid-tumors-more-effectively-at-a-molecular-level)**Moe Alsumidaie: How is Ymmunobio advancing targeted therapies to treat solid tumors more effectively at a molecular level?**
Peter Schiemann: At Ymmunobio, we are pioneering the development of targeted immunotherapies by identifying a novel tumor target with exceptional properties. This target is present in at least 13 solid tumors, with a high prevalence in patients—up to 95% in [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) and 90% in [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/), for example. This biomarker is hardly present in healthy tissue, offering a significant advantage over current oncology biomarkers that often appear widely in normal tissues. We are developing three therapeutic approaches, including an immuno-oncology compound with bispecific antibodies besides ADCs and radiopharmaceuticals. These bi-specific antibodies are designed to bind to T cells, inducing cytokine release to target tumor cells specifically. Our target’s uniqueness of being
Peter Schiemann, CEO and Chairman of Ymmunobio
absent in normal tissue gives us a significant edge over existing therapies. This specificity reduces the risk of off-target effects, making our approach safer and more effective.
## [](#moe-alsumidaie-what-challenges-do-you-face-in-balancing-innovation-with-regulatory-compliance-in-immuno-oncology)**Moe Alsumidaie: What challenges do you face in balancing innovation with regulatory compliance in immuno-oncology?**
Peter Schiemann: Surprisingly, we do not face many challenges in this area. Our team comprises experts with 25 to 30 years of experience in oncology drug development and are well-versed in regulatory environments. We are aware of the developments and do not foresee significant hurdles. Regulations often support our efforts rather than hinder them. For instance, we might not need in vivo tests with transgenic mice, which are costly, to progress into human trials with our bispecific antibodies. This regulatory flexibility could expedite our development process and reduce costs, allowing us to focus on innovation. Our experienced team ensures we remain compliant while pushing the boundaries of what’s possible in immuno-oncology.
## [](#moe-alsumidaie-how-are-you-adapting-study-design-to-enhance-patient-recruitment-and-retention-in-rare-diseases)**Moe Alsumidaie: How are you adapting study design to enhance patient recruitment and retention in rare diseases?**
Peter Schiemann: In oncology, we start with dose escalation studies in various solid tumors to ensure safety and efficacy. Once we have that data, we will move to expansion cohorts, focusing on high-incidence cancers like colorectal or breast cancer. Our strategy depends on the therapeutic we advance, whether a radiopharmaceutical, bispecific antibody or ADC. Real-world data might not be feasible for all, especially with radiopharmaceuticals, due to the need for special facilities. For example, our radiopharmaceutical development with the Paul Scherrer Institute involves testing isotopes like lutetium and terbium, which require specific handling and infrastructure. By tailoring our approach to each therapeutic, we aim to maximize patient recruitment and retention while ensuring the highest safety and efficacy standards.
## [](#moe-alsumidaie-how-do-you-plan-to-leverage-recent-funding-to-attract-further-investment-and-advance-your-pipeline)**Moe Alsumidaie: How do you plan to leverage recent funding to attract further investment and advance your pipeline?**
Peter Schiemann: To date, we raised 1.322 million Swiss francs from founders and friends, plus additional support from the Swiss Government for developing radiopharmaceuticals. Our goal is to achieve proof of concept in the preclinic, crucial for attracting larger investors. We have a convertible loan currently open for investors, aiming to reach proof of concept in one of our therapeutic areas. This funding will help us conducting necessary preclinical tests to demonstrate the efficacy and safety of our therapies, which is essential for securing further investment and advancing to clinical trials. We aim to build investor confidence and secure the resources needed to bring our innovative therapies to market by achieving these milestones. In addition, we are also currently speaking with pharmaceutical companies regarding possible co-development partnerships.
## [](#moe-alsumidaie-how-do-you-differentiate-your-antibody-therapies-from-those-of-other-biotech-companies)**Moe Alsumidaie: How do you differentiate your antibody therapies from those of other biotech companies?**
Peter Schiemann: Our competitive advantage lies in our unique target, which is present in at least 13 solid tumors and hardly in normal tissue; in addition, it has a high prevalence in patients, e.g. colorectal cancer 95%, breast cancer 90%, cervical cancer 90%, pancreatic cancer 80%, etc. We have filed two patents, making it difficult for competitors to work on the same target. Our target’s properties, such as its role in embryonic development and downregulation post-birth, provide a clear advantage in developing therapies with minimal off-target effects. This specificity allows us to focus on solid tumors without affecting healthy tissues, a common challenge in cancer therapies.
## [](#moe-alsumidaie-would-you-like-to-add-anything-else)**Moe Alsumidaie: Would you like to add anything else?**
Peter Schiemann: We are also working on a diagnostic partnership to analyze extracellular vesicles, which could aid in early cancer detection and screening. This method involves a simple blood test that could detect our target, potentially identifying cancer at a very early stage. Additionally, we are developing a [SPECT](https://www.clinicaltrialvanguard.com/news/telix-pharma-noble-registry-update-tlx599-cdx-psma-spect-imaging/) scan option for imaging tumor locations, enhancing treatment precision. Our focus remains on demonstrating proof of concept in animal models to validate our approach. These diagnostic tools could revolutionize cancer screening and monitoring, providing a comprehensive approach to cancer care. By integrating diagnostics with our therapeutic strategies, we aim to improve patient outcomes and advance the field of oncology.
**Categories:** Article: Executive Interviews
---
### [Exelixis Cabozantinib Update for Advanced Neuroendocrine Tumors](https://www.clinicaltrialvanguard.com/news/exelixis-cabozantinib-update-for-advanced-neuroendocrine-tumors/)
**Published:** November 27, 2024
**Author:** Jon Napitupulu
**Content:**
Exelixis announced that the FDA will discuss its supplemental New Drug Application for [cabozantinib](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/), intended to treat advanced pancreatic and extra-pancreatic neuroendocrine tumors (pNET and epNET), at an Oncologic Drugs Advisory Committee meeting in March 2025. This sNDA is based on the positive results of the Phase 3 [CABINET](https://www.clinicaltrialvanguard.com/news/exelixis-announces-results-from-cabinet-subgroup-analysis-at-asco-gi-2025/) trial, which showed significant improvement in progression-free survival for patients treated with cabozantinib compared to placebo. The FDA granted orphan drug designation to cabozantinib for pNET in August 2024 and set a target action date of April 3, 2025.
This news is significant for Exelixis, as a positive ODAC recommendation and subsequent FDA approval would expand the addressable market for cabozantinib, potentially driving revenue growth. It’s also important for patients with advanced pNET and epNET, who often face limited treatment options after disease progression. Current treatments may not effectively control the disease, and the five-year survival rates for advanced GI and lung NET tumors are 68% and 55% respectively, while the five-year survival rate for advanced pancreatic NET is only 23%. A new therapy offering improved progression-free survival would significantly advance the treatment landscape for these challenging cancers.
The CABINET trial demonstrated clinically meaningful improvements in progression-free survival with cabozantinib across all clinical subgroups, including primary tumor site, grade, and prior systemic anticancer therapy. The FDA’s decision to convene an ODAC meeting indicates that they are carefully considering the data, and the meeting outcome will be a critical step in the regulatory process. The planned ODAC meeting does not pertain to the currently approved indications for CABOMETYX in the U.S. The trial included 298 patients randomized to receive either cabozantinib or placebo. The primary endpoint was progression-free survival, and secondary endpoints included overall survival, radiographic response rate, and safety.
The upcoming ODAC meeting represents a pivotal moment for Exelixis and for the future of pNET and epNET treatment. A positive outcome could lead to FDA approval and make cabozantinib available to patients with these difficult-to-treat cancers, offering a new option with demonstrated efficacy in improving progression-free survival. While current treatments such as [somatostatin](https://www.clinicaltrialvanguard.com/news/first-patient-dosed-in-crinetics-neuroendocrine-tumor-trial/) analogs, chemotherapy, targeted therapy, and peptide-receptor radionuclide therapy exist, there remains a need for more effective options. The potential approval of cabozantinib could significantly alter the treatment paradigm and offer new hope for patients battling these rare and often aggressive tumors.
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**Categories:** News
---
### [Lantern Pharma & Starlight Therapeutics Present LP-184 Phase 1b Trial Design & Preclinical Data in Glioblastoma at Society for Neuro-Oncology (SNO) 2024 Highlighting Novel Synthetic Lethality](https://www.clinicaltrialvanguard.com/news/lantern-pharma-starlight-therapeutics-present-lp-184-phase-1b-trial-design-preclinical-data-in-glioblastoma-at-society-for-neuro-oncology-sno-2024-highlighting-novel-synthetic-lethality/)
**Published:** November 27, 2024
**Author:** Jon Napitupulu
**Content:**
[Lantern Pharma](https://www.clinicaltrialvanguard.com/news/lantern-pharmas-remarkable-phase-2-trial-update-in-advanced-lung-cancer/), through its subsidiary Starlight Therapeutics, revealed preclinical data and Phase 1b trial designs for LP-184 (STAR-001 for CNS indications) at the 2024 Society for Neuro-Oncology meeting. The drug is being explored for its potential in treating glioblastoma, a challenging brain cancer. The new data highlights the drug’s mechanism, ability to penetrate the brain, and potentially enhanced effects when combined with spironolactone.
Successful development of LP-184/STAR-001 could represent a major advance for patients battling this aggressive cancer and offer a much-needed new therapeutic approach. This progress further positions Lantern Pharma and Starlight Therapeutics at the forefront of developing targeted CNS cancer therapies.
The preclinical data showcased LP-184’s effectiveness in glioblastoma models and its activation by PTGR1, a protein overexpressed in many recurrent glioblastoma tumors. The planned Phase 1b trial will involve two cohorts of recurrent GBM patients: one receiving STAR-001 as monotherapy and the other receiving it in combination with spironolactone. The trial will evaluate safety, pharmacokinetics, and treatment response, utilizing biomarkers like PTGR1 expression, ERCC3 levels, and DNA damage markers. Lantern has previously developed a molecular diagnostic to assess PTGR1 levels, furthering their precision medicine approach. LP-184 is an acylfulvene alkylating agent that induces DNA double-strand breaks and has shown promise in various solid tumors. It has received Orphan Drug and Fast Track designations from the FDA for malignant glioma/glioblastoma treatment.
This research and planned trial signifies potential progress in the fight against glioblastoma. Positive results from the Phase 1b trial could lead to further clinical development and potentially offer a new treatment avenue for patients with this devastating disease. The combination therapy approach with spironolactone, guided by Lantern’s AI platform, could represent a significant step forward in personalized treatment strategies for glioblastoma. The use of biomarkers to identify responsive patients further strengthens the potential for improved outcomes and could pave the way for a more targeted and effective approach to glioblastoma treatment.
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**Categories:** News
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### [Lung Cancer Therapy Market 2023-2029: $70.39 Billion Analysis & Growth](https://www.clinicaltrialvanguard.com/news/lung-cancer-therapy-market-2023-2029-70-39-billion-analysis-growth/)
**Published:** November 27, 2024
**Author:** Jon Napitupulu
**Content:**
The global lung cancer therapy market, valued at $32.50 billion in 2023, is projected to reach $70.39 billion by 2029, exhibiting a 14% compound annual growth rate. This expansion is driven by the increasing prevalence of lung cancer, advancements in treatment modalities like targeted therapies and immunotherapies, and a growing emphasis on early detection. The rising adoption of precision medicine and ongoing research into combination therapies further fuel market growth.
This market growth holds significant implications for global healthcare systems and the oncology field. The increasing demand for effective lung cancer therapies necessitates continuous research and development, improved access to treatment, and robust healthcare infrastructure. Successful market expansion offers the potential to improve patient outcomes, reduce lung cancer mortality rates, and enhance the quality of life for those affected by this disease.
Several key factors fuel the market’s anticipated growth. Non-invasive treatments such as radiation therapy and targeted drug therapies are gaining traction due to their suitability for early-stage patients and those ineligible for surgery. Minimally invasive procedures like VATS and robotic surgery are projected to witness increasing demand thanks to their advantages over traditional open surgeries. Targeted therapy and immunotherapy are also witnessing significant uptake, with the latter expected to be the fastest-growing segment due to its potential for durable responses. The market is further segmented by indication, with [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) holding the largest share. Hospitals remain the primary end-users, but oncology clinics are expected to experience significant growth due to increasing demand for specialized care. The [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic also impacted the market, increasing awareness of respiratory health and accelerating the adoption of telehealth technologies.
The future of the lung cancer therapy market appears promising. The ongoing development of novel therapies, including combination therapies and personalized medicine approaches, holds the potential to revolutionize lung cancer treatment. Improved diagnostic tools and biomarkers for patient stratification will likely enhance treatment outcomes. While challenges remain in ensuring equitable access to these advanced therapies, the continuous innovation and investment in this field offer hope for significantly reducing the global burden of lung cancer in the years to come.
Source link: [http://www.businesswire.com/news/home/20241126191665/en/Lung-Cancer-Therapy-Research-Report-2023-2024-2029-70.39-Billion-Market-Analysis-By-Treatment-Method-Therapy-Type-Indication-End-User-and-Region—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20241126191665/en/Lung-Cancer-Therapy-Research-Report-2023-2024-2029-70.39-Billion-Market-Analysis-By-Treatment-Method-Therapy-Type-Indication-End-User-and-Region---ResearchAndMarkets.com)
**Categories:** News
---
### [Eyoni LCS: AI Lung Cancer Diagnostics at RSNA 2024](https://www.clinicaltrialvanguard.com/news/eyoni-lcs-ai-lung-cancer-diagnostics-at-rsna-2024/)
**Published:** November 27, 2024
**Author:** Jon Napitupulu
**Content:**
Median Technologies, a developer of AI-powered medical imaging software, will present its latest advancements at the 2024 Radiological Society of North America (RSNA) annual meeting. The company will showcase its eyonis™ [Lung Cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) Screening (LCS) software and its AI-driven imaging services for oncology drug developers. This presentation follows the successful completion of the REALITY clinical trial, which demonstrated the effectiveness of eyonis™ LCS in detecting lung cancer with statistical significance.
Positive clinical trial results validate the potential of eyonis™ LCS to improve early lung cancer diagnosis, leading to earlier intervention and potentially saving lives. This success positions Median Technologies as a key player in the growing field of AI-driven cancer diagnostics and has the potential to significantly impact lung cancer screening implementation globally. The RSNA annual meeting provides a crucial platform for the company to reach a large audience of medical professionals and industry stakeholders, potentially accelerating adoption and future collaborations.
The REALITY trial, the first of two pivotal studies, achieved its primary and secondary endpoints, showing eyonis™ LCS’s ability to effectively analyze low-dose computed tomography (LDCT) scans. This positive data reinforces the potential of eyonis™ LCS as a valuable diagnostic tool and supports the company’s plans to seek marketing authorization in the US and Europe in the first half of 2025, following the release of data from the second pivotal study, [RELIVE](https://www.clinicaltrialvanguard.com/news/eyonis-lung-cancer-screening-meets-primary-endpoint-in-relive-trial/), in Q1 2025. This planned regulatory filing represents a critical step towards commercialization and widespread availability of the technology.
The positive REALITY trial results and upcoming presentation at RSNA mark a major milestone for Median Technologies and the future of lung cancer screening. After successfully completing the second pivotal study and subsequent regulatory approvals, eyonis™ LCS could significantly improve early detection rates and improve patient outcomes. This technology has the potential to revolutionize lung cancer screening programs by offering a more accurate and efficient diagnostic tool, paving the way for broader access to early detection and potentially saving countless lives. This development could significantly alter the landscape of lung cancer diagnosis and treatment, leading to a more proactive and effective approach to combating this deadly disease. The advancement also positions Median Technologies for substantial growth and establishes them as a leader in AI-driven medical imaging solutions.
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**Categories:** News
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### [Claudin 18.2 Targeted Therapy Market Forecast 2024-2029](https://www.clinicaltrialvanguard.com/news/claudin-18-2-targeted-therapy-market-forecast-2024-2029/)
**Published:** November 27, 2024
**Author:** Jon Napitupulu
**Content:**
Claudin 18.2, a protein typically found in tight junctions, has emerged as a promising therapeutic target for various cancers, particularly gastric and gastroesophageal junction (G/GEJ) adenocarcinomas. Its overexpression in cancerous tissues but limited presence in healthy tissues makes it an ideal candidate for targeted therapies. The recent approval of [Zolbetuximab](https://www.clinicaltrialvanguard.com/article/intel-brief/triplet-regimen-biomarker-gate-phase-3-signal-what-ilustros-cohort-4-actually-proves/) (Vyloy) and the robust pipeline of Claudin 18.2-targeted therapies under development highlight the growing importance of this protein in oncology.
This news signifies a potential paradigm shift in cancer treatment, particularly for gastric cancer, which has historically presented limited therapeutic options. The development and approval of Claudin 18.2-targeted therapies offer new hope for patients diagnosed with these aggressive cancers, potentially improving survival rates and quality of life. The emergence of this new class of therapies also represents a significant advancement in precision oncology, enabling treatments to be tailored to individual patients based on their tumor’s specific characteristics.
Zolbetuximab, the first approved Claudin 18.2-targeted therapy, has shown promising clinical results and robust sales figures, demonstrating the high unmet need for effective treatments in this area. Various other therapies, including monoclonal and bispecific antibodies, CAR-T cell therapies, and antibody-drug conjugates, are currently in various stages of clinical development, expanding the treatment landscape and addressing different aspects of tumor biology. Companion diagnostic tests, such as the Ventana [CLDN18](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) (43-14A) RxDx Assay, play a crucial role in identifying patients most likely to benefit from these targeted therapies, further enhancing the precision and efficacy of treatment. The strong presence of both Western and Asian companies in the Claudin 18.2 research field fosters a competitive and collaborative environment that is driving innovation.
The Claudin 18.2-targeted therapy market is poised for significant growth, fueled by positive clinical outcomes and an expanding pipeline of promising drug candidates. While G/GEJ adenocarcinomas are the current primary focus, ongoing research is exploring the potential of these therapies in other cancer types, including ovarian, pancreatic, and lung cancers. This diversification of target indications, coupled with advancements in companion diagnostics, is expected to broaden the reach and impact of Claudin 18.2-targeted therapies, offering new treatment possibilities for a wider range of cancer patients and potentially revolutionizing cancer care. The ongoing clinical trials and research efforts focused on various drug modalities and combination therapies are expected to refine treatment strategies further and improve patient outcomes. This targeted approach to cancer therapy promises a more personalized and effective approach to managing these complex diseases.
Source link: [http://www.businesswire.com/news/home/20241126772100/en/Claudin-18.2-Targeted-Therapy-Market-Forecast-Clinical-Trials-Insights-Report-2024-2029-60-Drugs-Currently-in-Trials-with-1-Approved-Vyloy-zolbetuximab—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20241126772100/en/Claudin-18.2-Targeted-Therapy-Market-Forecast-Clinical-Trials-Insights-Report-2024-2029-60-Drugs-Currently-in-Trials-with-1-Approved-Vyloy-zolbetuximab---ResearchAndMarkets.com)
**Categories:** News
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### [EU Approves Tevimbra for Esophageal and Gastric Cancer](https://www.clinicaltrialvanguard.com/news/eu-approves-tevimbra-for-esophageal-and-gastric-cancer/)
**Published:** November 29, 2024
**Author:** Jon Napitupulu
**Content:**
The European Commission approved [BeiGene](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/)‘s [tislelizumab](https://www.clinicaltrialvanguard.com/news/akesos-ivonescimab-os-data-from-harmoni-6-selected-for-asco-plenary/) (TEVIMBRA), combined with chemotherapy, for first-line treatment of esophageal squamous cell carcinoma (ESCC) and gastric/gastroesophageal junction (G/GEJ) adenocarcinoma. This approval is based on positive Phase 3 trial results showing statistically significant overall survival benefits in patients with these cancers whose tumors express PD-L1 with a specific threshold. This expanded indication marks a significant advancement in treatment options for these aggressive cancers.
This approval is crucial for both BeiGene and patients suffering from ESCC and G/GEJ adenocarcinoma. These cancers often present at advanced stages with limited treatment options and poor prognoses. The demonstrated overall survival benefit with tislelizumab combination therapy offers new hope for improved outcomes in a patient population with a high unmet medical need. This approval strengthens BeiGene’s oncology portfolio, particularly in solid tumors, and further validates tislelizumab’s potential across various cancer types.
Two pivotal Phase 3 trials, RATIONALE-305 and RATIONALE-306, underpin this approval. RATIONALE-306, focusing on ESCC, showed a 34% reduction in the risk of death with tislelizumab plus chemotherapy compared to chemotherapy alone. In the specific PD-L1 positive patient subgroup, the risk reduction reached 38%. RATIONALE-305, evaluating G/GEJ adenocarcinoma, demonstrated a 20% reduction in the risk of death with the tislelizumab combination versus chemotherapy alone, rising to 29% in the PD-L1 positive subgroup. Across both trials, the most common severe adverse reactions included blood cell deficiencies, fatigue, electrolyte imbalances, pneumonia, decreased appetite, skin rash, and liver inflammation.
This European approval signifies a major step forward in the treatment landscape for ESCC and G/GEJ adenocarcinoma. It offers a new, more effective first-line treatment option with the potential to significantly improve survival rates for patients with these aggressive cancers. This approval is likely to have a positive impact on BeiGene’s market position, solidifying its commitment to developing innovative cancer therapies and potentially changing the standard of care for these challenging malignancies.
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**Categories:** News
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### [Addressing Clinical Trial Diversity: Challenges, Opportunities, and Critical Capabilities](https://www.clinicaltrialvanguard.com/conference-coverage/addressing-clinical-trial-diversity-challenges-opportunities-and-critical-capabilities/)
**Published:** November 29, 2024
**Author:** Moe Alsumidaie
**Content:**
The 2024 [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Europe conference featured a presentation on clinical trial diversity, highlighting the critical need for diversity and equity in scientific research. Jodie Allen, PhD, Liz Bristow from [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/), and Magnus Franzen from Wavestone explored the multifaceted reasons why diversity is essential in clinical trials and the strategic steps necessary to achieve it. The discussions highlighted the scientific, business, and ethical imperatives driving the push for more inclusive research practices and the regulatory demands shaping the future of clinical trials globally.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#the-importance-of-diversity-in-science)The Importance of Diversity in Science
The speakers delved into the necessity of diversity in clinical trials, emphasizing its role in enhancing scientific understanding and improving research outcomes. They explained that diversity allows for identifying pharmacogenetic markers, which is crucial for understanding how different genetic backgrounds affect drug metabolism and response. This leads to more generalizable and externally valid trial findings, ensuring that results apply to a broader population. Such scientific rigor is vital for providing patients and healthcare providers with confidence in the outcomes. From a business perspective, the speakers noted that trial data reflecting the population expected to use a medical product can lead to significant cost savings. It reduces the need for expensive post-marketing requirement (PMR) studies, often mandated to gather additional data on underrepresented populations, and decreases the risk of regulatory bodies’ non-approval. Regulatory demands are also driving the push for diversity. The FDA now requires Diversity Action Plans with specific, measurable goals, and similar initiatives are being observed globally, with agencies like the MHRA and EMA moving in the same direction. These regulatory changes underscore the global recognition of the importance of diversity in clinical trials.
## [](#patients-deserve-equitable-access)Patients Deserve Equitable Access
There was emphasis on the ethical imperative of ensuring equitable access to clinical trials for all patient populations. Historically, certain groups have been excluded from clinical research, leading to a lack of data on how these populations respond to treatments. The speakers emphasized that improving trial accessibility for these historically excluded populations is crucial. By expanding access to safe and effective therapies, the healthcare industry can work towards reducing inequities in health outcomes. The speakers provided examples of how certain populations, such as racial and ethnic minorities, women, and older adults, have been underrepresented in clinical trials. This underrepresentation can lead to disparities in treatment efficacy and safety, as the data does not accurately reflect the diverse patient populations that will ultimately use the medical products. By prioritizing diversity in clinical trials, the industry can ensure that all patients have access to therapies proven to be safe and effective for their specific demographic.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#building-clinical-trial-diversity-capabilities)Building Clinical Trial Diversity Capabilities
Most of the session discussed the components of a robust clinical trial diversity capability. The speakers stressed the importance of understanding the Intended Use Population (IUP) early in the study planning process. This understanding is crucial for designing inclusive studies that accurately reflect the target population’s characteristics. The speakers shared a detailed case study to illustrate this point. In one example, a clinical trial initially included a BMI criterion that excluded Black non-Hispanic patients due to their higher average BMI. Upon review, it was determined that this criterion had no clinical rationale and was inherited from a previous study phase. Removing this criterion made the trial more inclusive and better aligned with the target population’s characteristics.
Similarly, the speakers discussed adjusting blood pressure thresholds to serve the target population better. Initially, the study had a threshold of 150/95, which was too low for specific racial and ethnic minority (REM) populations. Expanding the range to 160/95 made the study more inclusive without compromising its scientific integrity. Additionally, using local labs for pre-screening were encouraged to simplify complex assessments and make the trial more accessible to diverse populations.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#strategic-site-selection-and-community-engagement)Strategic Site Selection and Community Engagement
The speakers emphasized that strategic site selection and effective community engagement must support inclusive protocol design. They noted that selecting the right sites is crucial for reaching underrepresented populations and ensuring their participation in clinical trials. This involves partnering with sites with strong ties to the community and are trusted by their populations. Community engagement was highlighted as a critical success factor in operationalizing studies that enable underrepresented populations to participate. The speakers pointed out that sites and partners are often better positioned to deliver effective community engagement, as they have a deeper understanding of the local context and can build trust with potential participants. The speakers also addressed the common issue of sponsors focusing solely on patient accrual numbers rather than collaborating with sites to identify and address barriers to participation. They stressed the importance of having open and ongoing conversations with sites to understand their needs and challenges. By doing so, sponsors can work collaboratively with sites to develop solutions that enhance trial accessibility and inclusivity.
## [](#continuous-improvement-and-reflection)Continuous Improvement and Reflection
The session concluded with a call for continuous improvement and reflection in pursuing clinical trial diversity. The speakers shared that AstraZeneca has developed a Clinical Trial Diversity Action Plan (CTDAP) delivery model to ensure consistency and efficiency across its trials. This model includes core roles and enabling functions that support the implementation of diversity initiatives. The speakers emphasized the importance of addressing initial reluctance to set diversity enrolment goals. They encouraged attendees to reflect continuously and embed learnings from past experiences to drive change. This involves reviewing feedback from regulatory bodies like the FDA, comparing plans across the enterprise, and understanding the root causes of any gaps in implementation. By fostering a culture of continuous improvement, the industry can make significant strides toward more inclusive and representative clinical research. The speakers encouraged attendees to assess their clinical trial diversity capabilities and commit to ongoing improvement, ensuring all patient populations can access safe and effective therapies.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#conclusion)Conclusion
The session emphasized that achieving diversity in clinical trials is a complex but necessary endeavor. The industry can make significant strides toward more inclusive and representative clinical research by addressing concerns, building a value story with case studies, and fostering collaboration within a robust structure. The speakers encouraged attendees to assess their clinical trial diversity capabilities and commit to ongoing improvement, ensuring all patient populations can access safe and effective therapies.
**Categories:** Article: Conference Coverage
---
### [Sudo Biosciences Doses First Participants in Phase 1 Trial of Brain-Penetrant Allosteric TYK2 Inhibitor Sudo-550](https://www.clinicaltrialvanguard.com/news/sudo-biosciences-doses-first-participants-in-phase-1-trial-of-brain-penetrant-allosteric-tyk2-inhibitor-sudo-550/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Sudo Biosciences, a company focused on developing precision TYK2 inhibitors, has initiated a Phase 1 clinical trial for SUDO-550. This novel drug is an orally administered, brain-penetrant, allosteric TYK2 inhibitor designed to treat neuroinflammatory diseases. This marks the second allosteric TYK2 inhibitor from Sudo Biosciences to enter clinical trials this year, following SUDO-286, a topical treatment for [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/) currently in two Phase 1 trials.
The ongoing Phase 1 trial for SUDO-550 aims to assess its safety, tolerability, and pharmacokinetics in healthy volunteers. A crucial aspect of this study is confirming the compound’s ability to effectively cross the blood-brain barrier, a critical factor for treating diseases of the central nervous system. This trial represents a significant step in developing SUDO-550 as a leading brain-penetrant TYK2 inhibitor. This therapy holds promise for advancing treatment options for conditions such as [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/), ALS, and [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/).
Sudo Biosciences is developing several highly potent and selective small molecule TYK2 pseudokinase inhibitors. These inhibitors target a range of autoimmune and neurological conditions. SUDO-550 exhibits high selectivity and potency for TYK2, minimizing off-target effects. Preclinical studies indicate excellent blood-brain barrier penetration, highlighting its potential for treating CNS diseases characterized by neuroinflammation. This positions SUDO-550 as a potentially best-in-class treatment for various neuroinflammatory diseases.
SUDO-286, another key compound in Sudo’s pipeline, is a potent and selective topical TYK2 inhibitor being explored for psoriasis and other immune-mediated skin conditions. It entered clinical trials earlier this year with two ongoing Phase 1 studies involving healthy volunteers and patients.
Sudo Biosciences is dedicated to developing novel medicines to improve patients’ lives. Their programs focus on the tyrosine kinase 2 (TYK2) pseudokinase domain, a vital component in cytokine signaling pathways linked to various immune-mediated inflammatory diseases. Their pipeline includes SUDO-550, the brain-penetrant candidate for multiple sclerosis and neurodegenerative diseases, and SUDO-286, the topical candidate for dermatological conditions. The company operates across the US and UK, with headquarters in Carmel, Indiana.
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**Categories:** News
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### [Zorevunersen Data for Dravet Syndrome at AES 2024](https://www.clinicaltrialvanguard.com/news/zorevunersen-data-for-dravet-syndrome-at-aes-2024/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Stoke Therapeutics, a biotechnology company focused on RNA medicine, will present data at the 2024 American [Epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/) Society (AES) Annual Meeting in Los Angeles, California, from December 6th to 10th. A virtual event for investors and research analysts will also be held on December 9th at 8:30 am EST, featuring discussions led by clinicians and patient advocates.
The company is developing zorevunersen, a potential first-in-class disease-modifying treatment for Dravet syndrome. New data from a Phase 1/2 study will be presented, showcasing results from nine patients who received initial 70mg doses of zorevunersen followed by 45mg maintenance doses in an open-label extension (OLE) study. Additional data from 73 patients in the OLE studies will also be presented, demonstrating consistent and durable seizure reduction and continuous improvement across various Vineland-3 subdomains over 24 months. Treatment was generally well-tolerated.
Stoke will host a symposium for clinicians on December 8th from 9:00 PM to 12:00 AM EST. This symposium will examine new data from the Dravet syndrome program and discuss the need for disease-modifying therapies that address more than just seizure reduction, focusing on the behavioral, cognitive, and seizure-related impacts of the disease.
The investor and analyst virtual event on December 9th, from 8:30 AM to 9:30 AM EST, will feature leading clinicians and patient advocates discussing the impacts of Dravet syndrome, the current treatment landscape, the latest zorevunersen data, and the potential real-world benefits of a disease-modifying treatment. A question-and-answer session for research analysts will also be included.
Dravet syndrome, a severe and progressive genetic epilepsy, is characterized by frequent, prolonged seizures starting in infancy. It often leads to intellectual disability, developmental delays, movement and balance issues, speech disturbances, growth defects, sleep problems, autonomic nervous system disruptions, and mood disorders. This disease is classified as a developmental and epileptic encephalopathy and carries a higher risk of Sudden Unexpected Death in Epilepsy (SUDEP) than the general epilepsy population. Currently, there are no approved disease-modifying treatments. Dravet syndrome affects approximately one in 16,000 babies, regardless of geographic location or ethnicity.
Zorevunersen, an investigational antisense [oligonucleotide](https://www.clinicaltrialvanguard.com/news/camp4s-pioneering-syngap-1-drug-enters-toxicology-studies/) (ASO), aims to increase NaV1.1 protein expression by utilizing the healthy copy of the SCN1A gene. This action is intended to restore physiological NaV1.1 levels, thus reducing seizures and non-seizure comorbidities. Zorevunersen has received orphan drug designation from both the FDA and EMA, and rare pediatric disease designation from the FDA for Dravet syndrome.
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**Categories:** News
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### [Daiichi Sankyo Oncology Progress at ESMO Asia, SABCS & ASH](https://www.clinicaltrialvanguard.com/news/daiichi-sankyo-oncology-progress-at-esmo-asia-sabcs-ash/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo will present over 45 abstracts showcasing new clinical research across its oncology portfolio at the 2024 ESMO Asia Congress, San Antonio Breast Cancer Symposium (SABCS), and American Society of Hematology (ASH) Annual Meeting. These presentations will precede the company’s Science & Technology Day and demonstrate advancements toward establishing new cancer care standards. Three late-breaking presentations are planned. At ESMO Asia, a pooled analysis of datopotamab deruxtecan (Dato-DXd) data from the TROPION-Lung05 and TROPION-Lung01 trials in patients with EGFR-mutated advanced non-small cell lung cancer (NSCLC) will be presented. SABCS will feature a subgroup analysis from the DESTINY-Breast06 trial of ENHERTU (trastuzumab deruxtecan) in patients with unresectable or metastatic HR positive, HER2 low breast cancer. Also, at SABCS, the primary analysis from the VALENTINE trial of patritumab deruxtecan (HER3-DXd) in patients with early HR positive, HER2 negative breast cancer will be shared.
ESMO Asia will highlight innovation in lung, breast, gastric, and biliary tract cancers. A pooled analysis of Dato-DXd in previously treated EGFR-mutated advanced NSCLC from the TROPION-Lung05 and TROPION-Lung01 trials will be a key presentation. Mini oral presentations will cover the TROPION-Breast01 trial of Dato-DXd in Chinese patients with previously treated metastatic HR positive, HER2 low or negative breast cancer, and final results from the DESTINY-Gastric06 trial of ENHERTU in Chinese patients with previously treated HER2 positive advanced gastric or gastroesophageal junction adenocarcinoma. The DESTINY-Gastric06 results supported the recent conditional approval of ENHERTU in China for this indication. Trials-in-progress posters will detail the designs of ongoing ENHERTU trials, including DESTINY-BTC01 (ENHERTU with rilvegostomig versus standard of care in previously untreated HER2 expressing biliary tract cancer) and DESTINY-Gastric03 (ENHERTU with rilvegostomig and chemotherapy in HER2 positive or HER2 low gastric or GEJ adenocarcinoma). An update on the DESTINY-PanTumor02 trial of ENHERTU will also be presented, showcasing five new cohorts enrolling patients with various HER2 expressing solid tumors.
SABCS will feature continued progress in breast cancer research. A subgroup analysis of DESTINY-Breast06 will explore the impact of prior endocrine-based therapy response on outcomes in patients with HR positive, HER2 low breast cancer treated with ENHERTU versus chemotherapy. Primary results from the VALENTINE trial evaluating neoadjuvant HER3-DXd, alone or with letrozole, in high-risk HR positive, HER2 negative early breast cancer will also be presented. Additional data includes biomarker analyses from DESTINY-Breast03, health-related quality of life data from DESTINY-Breast12, and final results from the DESTINY-Breast08 dose expansion. Trial-in-progress posters will detail studies evaluating ENHERTU in combination with valemetostat in previously treated HER2 low breast cancer.
At ASH, various sub-analyses of the QuANTUM-First trial of VANFLYTA (quizartinib) in newly diagnosed FLT3-ITD positive AML will be shared. An oral presentation will focus on co-mutations and their impact on remission, survival, and relapse. Posters will address the impact of continuation therapy, FLT3-ITD mutation detection assays, and a comparison of VANFLYTA to midostaurin. Data from the QUIWI trial, which evaluated VANFLYTA in combination with chemotherapy in newly diagnosed FLT3-ITD negative AML, will also be presented, including final results and sub-analyses. The QuANTUM-Wild trial design, based on QUIWI, will be featured. Finally, primary results from the VALYM trial of valemetostat in relapsed or refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) and an analysis of circulating tumor DNA from the VALENTINE-PTCL01 trial in relapsed or refractory peripheral [T-cell lymphoma](https://www.clinicaltrialvanguard.com/news/soquelitinib-data-in-t-cell-lymphoma-promising-results-unveiled/) will be presented. Daiichi Sankyo’s Science & Technology Day on December 16, 2024, will provide further overview and updates on R&D strategy.
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**Categories:** News
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### [Rallybio Confirmatory Study Launch in Q2 2025](https://www.clinicaltrialvanguard.com/news/rallybio-confirmatory-study-launch-in-q2-2025/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Rallybio Corporation announced new biomarker analyses and manufacturing process enhancements for [RLYB116](https://www.clinicaltrialvanguard.com/news/rallybio-to-initiate-rlyb116-clinical-pk-pd-study-in-2025/), its investigational once-weekly, subcutaneous C5 inhibitor for complement-mediated diseases. These findings suggest RLYB116 achieved greater complement inhibition in a Phase 1 study than originally reported, potentially positioning it as a best-in-class therapy. A confirmatory clinical pharmacokinetic/pharmacodynamic (PK/PD) study is planned for the second quarter of 2025.
Post-Phase 1 study analyses revealed the assay used to measure free C5 overestimated levels by approximately ten-fold. This indicates RLYB116 provided more substantial complement inhibition than initially indicated. Furthermore, manufacturing process enhancements completed in the third quarter of 2024, utilizing advanced analytical techniques like mass spectrometry, further purified the RLYB116 drug substance. This is anticipated to improve tolerability at doses matching or exceeding those in the Phase 1 study.
The upcoming confirmatory PK/PD study, scheduled for the second quarter of 2025, aims to demonstrate this improved tolerability and sustained complement inhibition. This single-blind, multiple ascending dose study will involve two cohorts of eight participants each. Cohort 1 will receive 150 mg weekly doses, while Cohort 2 will receive 225 mg weekly doses. The treatment duration is four weeks, followed by a ten-week follow-up period.
RLYB116 is designed as a convenient, once-weekly, small volume, subcutaneous injection. The prior Phase 1 single- and multiple-ascending dose (SAD/MAD) study in healthy participants, completed in the fourth quarter of 2023, evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics. The MAD portion employed an adaptive single-blind design with a four-week treatment period and ten-week follow-up. Four cohorts were included, exploring different dosing regimens: Cohort 1 (100 mg weekly), Cohort 2 (three 100 mg doses the first week, followed by weekly dosing), Cohort 3 (150 mg weekly, reduced to 125 mg weekly), and Cohort 4 (75 mg twice weekly initially, then 100 mg twice weekly).
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**Categories:** News
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### [Omeros Reveals ASH Presentations](https://www.clinicaltrialvanguard.com/news/omeros-reveals-ash-presentations/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Omeros Corporation announced two upcoming presentations on their investigational MASP-3 inhibitor, zaltenibart (OMS906), at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego this December. The presentations will focus on zaltenibart’s potential in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder. Phase 3 clinical trials for zaltenibart in PNH are anticipated to begin in early 2025. Both abstracts are currently accessible on the ASH website.
One presentation will detail interim results from a Phase 2 proof-of-concept study examining zaltenibart as a monotherapy for PNH patients with suboptimal responses to ravulizumab. This presentation will highlight how treatment with zaltenibart improved key hematological parameters in these patients. The other presentation will cover the population pharmacokinetics/pharmacodynamics and clinical pharmacology of zaltenibart in both healthy subjects and PNH patients. Both presentations will occur on Monday, December 9th, from 6:00 PM to 8:00 PM at the San Diego Convention Center. Presentation materials will be available on the Omeros website after the conference.
OMS906 targets mannan-binding lectin-associated serine protease-3 (MASP-3), the primary activator of the complement system’s alternative pathway. The complement system is crucial for innate immunity, playing a vital role in maintaining homeostasis and defending against pathogens. MASP-3 converts pro-complement factor D to complement factor D, making it a critical target within the alternative pathway. Its low circulating levels and clearance rate, compared to other alternative pathway proteins, make it an attractive target. Inhibiting MASP-3, unlike C5 and C3 blockers, preserves the classical pathway’s lytic arm, essential for combating infections. Furthermore, MASP-3 is not believed to be an acute phase reactant, a potential advantage for MASP-3 inhibitors like OMS906 over other alternative pathway inhibitors.
MASP-3 inhibitors are being explored for their potential in various diseases. These include PNH, hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, [arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/), geographic atrophy (dry [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/)), ischemia-reperfusion injury, transplant complications, and other immune-related conditions.
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**Categories:** News
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### [Ajax Presents Phase 1 Trial Results of JAK2 Inhibitor for Myelofibrosis](https://www.clinicaltrialvanguard.com/news/ajax-presents-phase-1-trial-results-of-jak2-inhibitor-for-myelofibrosis/)
**Published:** December 2, 2024
**Author:** Jon Napitupulu
**Content:**
Ajax Therapeutics announced the presentation of its ongoing first-in-human study of AJ1-11095, a next-generation Type II JAK2 inhibitor, at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego on December 8, 2024. The Phase 1 study, led by principal investigator Dr. John Mascarenhas, focuses on AJ1-11095 as an oral monotherapy for patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not responded to Type I JAK2 inhibitors. More information about the study is available on www.[clinicaltrials](https://www.clinicaltrialvanguard.com/article/fda-needs-to-release-clinicaltrials-gov-guidance-now/).gov (NCT06343805). The poster presentation will take place during the “Myeloproliferative Syndromes and Chronic Myeloid [Leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/): Basic and Translational: Poster II” session from 6:00 – 8:00 p.m. PT in Halls G-H of the San Diego Convention Center.
Developed in collaboration with Schrödinger, AJ1-11095 utilizes advanced structure-based drug design and computational methods. It targets the Type II conformation of the JAK2 kinase, aiming for improved efficacy and disease modification compared to existing Type I JAK2 inhibitors like [ruxolitinib](https://www.clinicaltrialvanguard.com/news/karyopharm-to-submit-selinexor-ruxolitinib-snda-for-myelofibrosis-in-august-2026/). Preclinical studies indicate AJ1-11095’s potential to reverse bone marrow fibrosis, reduce mutant allele burden, and maintain efficacy against MPN cells resistant to Type I JAK2 inhibition.
Myelofibrosis (MF) is a rare blood cancer affecting approximately 20,000 individuals in the United States. Characterized by enlarged spleen, bone marrow fibrosis, progressive anemia, and debilitating symptoms like fatigue, night sweats, itching, and abdominal discomfort, MF significantly impacts patients’ quality of life. Current Type I JAK2 inhibitors primarily offer symptomatic relief and spleen size reduction but have limited impact on the underlying disease. Many patients eventually discontinue these treatments due to lack of benefit, adverse events, or disease progression, highlighting the urgent need for new therapeutic options.
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**Categories:** News
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### [Janux Expands Phase 1b Trials for JANX007 in mCRPC](https://www.clinicaltrialvanguard.com/news/janux-expands-phase-1b-trials-for-janx007-in-mcrpc/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
Janux Therapeutics, a clinical-stage biopharmaceutical company, announced positive interim clinical data for its JANX007 program, a Tumor Activated T Cell Engager (TRACTr) therapy for metastatic castration-resistant prostate cancer (mCRPC). The company highlighted the substantial activity observed in heavily pre-treated patients, paving the way for expansion trials in earlier treatment lines.
The Phase 1a clinical trial enrolled mCRPC patients who had received a median of four prior lines of therapy. As of November 15, 2024, sixteen pre-PLUVICTO® patients were treated once-weekly with JANX007 at doses ranging from 2 mg to 9 mg. Across all doses, the therapy demonstrated high prostate-specific antigen (PSA) response rates. Specifically, 100% of patients achieved at least a 50% PSA decline ([PSA50](https://www.clinicaltrialvanguard.com/news/oric-944-trial-remarkable-efficacy-and-safety-results/)), 63% achieved at least a 90% decline (PSA90), and 31% achieved at least a 99% decline (PSA99).
Furthermore, these PSA declines demonstrated durability, with 75% of patients maintaining PSA50 declines for at least 12 weeks and 50% maintaining PSA90 declines for at least 12 weeks at a target dose of 2 mg or higher. These robust responses were observed regardless of resistance driver aberration status or prior taxane or ARPi treatment. In patients evaluable by RECIST criteria, anti-tumor activity was evident, with confirmed and unconfirmed partial responses in 50% (4 out of 8) of patients.
JANX007 exhibited a favorable safety profile. Cytokine release syndrome (CRS) and related adverse events were primarily limited to the first treatment cycle and were generally mild (grades 1 and 2). Similarly, treatment-related adverse events not associated with CRS were also mostly confined to the first cycle and were of mild severity. Importantly, the maximum tolerable dose for JANX007 has not yet been determined.
Based on these promising efficacy and safety findings, Janux has selected two once-weekly step dose regimens for Phase 1b expansion trials focusing on pre-PLUVICTO® second- and third-line patients. The company plans to provide another update on the JANX007 program in 2025.
Janux is developing a pipeline of TRACTr and Tumor Activated Immunomodulator (TRACIr) therapeutics. JANX007, targeting PSMA, is their first clinical candidate for prostate cancer. Their second clinical candidate, JANX008, targets epidermal growth factor receptor (EGFR) and is being studied in a Phase 1 trial for various solid tumors, including colorectal carcinoma, head and neck squamous cell carcinoma, non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/), renal cell carcinoma, small cell lung cancer, pancreatic ductal adenocarcinoma, and triple-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). Janux is also advancing several additional preclinical TRACTr and TRACIr programs.
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**Categories:** News
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### [MammaPrint Predicts Benefit of Extended Endocrine Therapy](https://www.clinicaltrialvanguard.com/news/mammaprint-phase-3-trial-data-identifies-ideal-patients-who-benefit-from-endocrine-therapy/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
A secondary analysis of the IDEAL phase 3 randomized clinical trial, published in JAMA Network Open, confirms that the MammaPrint genomic test can predict the benefit of extended endocrine therapy (EET) in postmenopausal women with hormone receptor-positive (HR+) early-stage [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). This reinforces previous findings from the NSABP B-42 trial, demonstrating MammaPrint’s ability to identify patients most likely to benefit from extended endocrine therapy and those who might safely avoid it and its associated side effects. The study focused on MammaPrint’s predictive ability regarding EET in preventing late recurrences among a cohort of 515 postmenopausal patients with early-stage HR+ breast cancer from the IDEAL trial.
This addresses a critical challenge in breast cancer treatment: determining the optimal duration of endocrine therapy. While extended endocrine therapy can reduce the risk of late recurrence, it can also cause unnecessary side effects for some patients. By identifying which patients are most likely to benefit from extended therapy, MammaPrint offers a personalized approach, potentially improving long-term outcomes and quality of life for those affected by breast cancer. This precision medicine approach moves beyond traditional clinical risk factors to provide a more tailored and effective treatment strategy.
The study involved patients who were randomized to receive either 2.5 or 5 years of letrozole after completing 5 years of initial endocrine therapy. The original IDEAL trial found no overall benefit from 5 years versus 2.5 years of extended therapy. However, using MammaPrint genomic profiling, researchers identified a subset of patients who did benefit from EET. The data indicates MammaPrint accurately predicted which patients with low genomic risk would experience improved recurrence-free survival with extended endocrine therapy. Conversely, the test also identified patients at high genomic risk who derived minimal benefit from extended treatment, allowing them to potentially avoid unnecessary side effects.
This validation of MammaPrint’s predictive ability has significant implications for the field of personalized oncology. It extends the clinical utility of MammaPrint beyond guiding chemotherapy decisions and into optimizing the duration of adjuvant endocrine therapy. This personalized approach promises to improve patient outcomes by maximizing treatment efficacy while minimizing exposure to unnecessary therapies and associated side effects, ultimately leading to a higher quality of life for those diagnosed with early-stage breast cancer.
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### [Kairos Pharma and City of Hope Team Up For Phase 2 Trial](https://www.clinicaltrialvanguard.com/news/kairos-pharma-and-city-of-hope-team-up-for-phase-2-trial/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
[Kairos Pharma](https://www.clinicaltrialvanguard.com/news/kairos-pharma-and-huntsman-cancer-institute-for-env105-phase-2-trial/) (NYSE American: KAPA) is expanding its Phase 2 clinical trial for ENV105, a novel cancer drug resistance therapy, by adding City of Hope Cancer Center as a trial site. This expansion aims to broaden the patient population for ENV105, a treatment for castrate-resistant [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/), and identify biomarkers that predict patient response. The trial is being conducted in collaboration with City of Hope and is partially funded by a grant from the National Cancer Institute (NCI).
This expansion of the ENV105 trial is significant for both Kairos Pharma and the field of oncology. For Kairos, it represents progress in developing a much-needed treatment for drug-resistant prostate cancer, a condition with limited treatment options. For the oncology field, the trial could lead to a new therapeutic approach for managing resistance to hormonal therapy, a common challenge in prostate cancer treatment. Successful results could potentially pave the way for broader application in other cancers that develop treatment resistance.
The Phase 2 trial is investigating ENV105 in combination with apalutamide, a standard hormonal therapy, in patients with castrate-resistant prostate cancer. The addition of City of Hope, and other planned centers, will enable researchers to study ENV105 in a larger and more diverse group of patients. This increased sample size will enhance the statistical power of the study and provide a more robust assessment of ENV105’s efficacy and safety. The search for predictive biomarkers is a crucial aspect of personalized medicine, allowing clinicians to tailor treatment strategies based on individual patient characteristics and likely response to therapy. The NCI grant provides external validation of the scientific rationale behind ENV105 and reinforces the importance of this research.
The expansion of the Phase 2 clinical trial for ENV105 represents a critical step forward in the fight against drug-resistant cancers. Positive results from this trial could validate CD105 as a therapeutic target and establish ENV105 as a potential new treatment option for castrate-resistant prostate cancer. Moreover, the identification of predictive biomarkers could revolutionize how this therapy is administered, ensuring that patients most likely to benefit receive the treatment. This research holds significant promise for improving patient outcomes and addressing the growing challenge of drug resistance in cancer treatment. The successful development of ENV105 could potentially transform the treatment landscape for various cancers, offering new hope for patients who have exhausted standard treatment options.
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### [Trio Pharmaceuticals Secures $3.1M to Advance Cancer Therapeutics](https://www.clinicaltrialvanguard.com/news/trio-pharmaceuticals-secures-3-1m-to-advance-cancer-therapeutics/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
TRIO Pharmaceuticals, a biotechnology company developing bispecific antibodies for cancer treatment, secured $3.1 million in pre-Series A funding. This funding round, led by Friedman Bioventure Fund with significant contributions from the [Myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/) Investment Fund and others, will fuel the expansion of preclinical activities and advancement of TRIO’s programs toward clinical trials. The investment highlights the promise of TRIO’s TRAILBody™ and TIE-ADC™ platforms in addressing cancers with high mortality rates.
For TRIO, the funding provides crucial resources to accelerate the development of its innovative bispecific antibody platforms. For the oncology field, TRIO’s approach represents a potential paradigm shift in cancer treatment, offering more potent and less toxic therapies for challenging cancers like multiple myeloma and acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/). The involvement of the Myeloma Investment Fund further underscores the potential impact of this technology on addressing unmet needs in blood cancers.
The $3.1 million investment will primarily support preclinical research and development activities, including studies to further validate the efficacy and safety of TRIO’s bispecific antibodies. The funding will also facilitate the company’s progress toward initiating clinical trials, a critical step in translating promising preclinical results into tangible benefits for patients. The partnership with experienced investors like Friedman Bioventure Fund and the Myeloma Investment Fund provides not only financial support but also strategic guidance and access to valuable resources, enhancing TRIO’s potential for success.
This funding round represents a crucial step forward for TRIO Pharmaceuticals. It positions the company to accelerate its development timeline, bringing its novel cancer therapies closer to clinical evaluation and potentially revolutionizing treatment options for patients battling aggressive cancers. The successful completion of this financing signifies growing confidence in TRIO’s technology and its potential to transform cancer care, particularly for those facing limited treatment options. This progress has the potential to bring a much-needed wave of hope to patients and their families.
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### [Anzupgo® (Delgocitinib) Cream Approved in Great Britain](https://www.clinicaltrialvanguard.com/news/anzupgo-delgocitinib-cream-approved-in-great-britain/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved [delgocitinib](https://www.clinicaltrialvanguard.com/news/leo-pharma-unveils-remarkable-results-of-anzupgo-cream-in-chronic-hand-eczema/) cream, a topical Janus kinase (JAK) inhibitor, for treating moderate to severe Chronic Hand Eczema (CHE) in adults. This approval offers a new treatment option for those for whom topical corticosteroids are ineffective or unsuitable. CHE is a persistent inflammatory skin disease causing significant itch, pain, and disruptions to daily life.
This approval is significant for both LEO Pharma and individuals suffering from CHE. For LEO Pharma, it strengthens their position in the medical dermatology market and provides a valuable addition to their portfolio. For patients, it represents a much-needed new treatment approach, especially for those who haven’t found relief with existing topical corticosteroids. This approval also addresses the growing need for innovative solutions for this prevalent and burdensome condition.
Delgocitinib cream works by inhibiting JAK-STAT signaling, a key pathway in CHE’s development. The pathophysiology of CHE involves skin barrier dysfunction, inflammation, and changes in the skin microbiome. Clinical trials demonstrated the cream’s effectiveness in achieving primary and secondary endpoints, indicating significant improvement in CHE symptoms compared to a placebo. These trials included both short-term efficacy studies and longer-term evaluations of safety and efficacy. The availability of this topical treatment could potentially reduce the need for systemic treatments, minimizing the risk of broader side effects.
This MHRA approval marks a pivotal step forward in CHE management, offering new hope for patients experiencing the debilitating effects of this condition. Wider access to delgocitinib cream could improve patients’ quality of life by alleviating symptoms and enabling greater participation in daily activities. Further research and real-world data will help to refine the understanding of the cream’s long-term efficacy and safety profile, paving the way for personalized treatment strategies. This innovation holds promise for advancing the standard of care in dermatology and improving the lives of those affected by CHE.
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**Categories:** News
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### [Daiichi Sankyo's Oncology Breakthroughs at ESMO Asia, SABCS & ASH](https://www.clinicaltrialvanguard.com/news/daiichi-sankyos-oncology-breakthroughs-at-esmo-asia-sabcs-ash/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo will present over 45 abstracts showcasing new clinical research in various cancers at the 2024 ESMO Asia Congress, San Antonio Breast Cancer Symposium (SABCS), and American Society of Hematology (ASH) Annual Meeting, leading up to its Science & Technology Day. These presentations will highlight the company’s progress in establishing new standards of cancer care.
Key late-breaking presentations include a pooled analysis from the TROPION-Lung05 and TROPION-Lung01 trials of datopotamab deruxtecan (Dato-DXd) in EGFR-mutated advanced non-small cell lung cancer (NSCLC) at ESMO Asia. SABCS will feature a subgroup analysis from the DESTINY-Breast06 trial of ENHERTU (trastuzumab deruxtecan) in HR positive, HER2 low or ultralow breast cancer and the primary analysis from the VALENTINE trial of patritumab deruxtecan (HER3-DXd) in early HR positive, HER2 negative breast cancer. These presentations cover advancements in challenging cancers like lung, breast, gastric, biliary tract, acute myeloid leukemia, and peripheral [T-cell lymphoma](https://www.clinicaltrialvanguard.com/news/soquelitinib-data-in-t-cell-lymphoma-promising-results-unveiled/).
ESMO Asia will showcase lung cancer innovation with the Dato-DXd analysis in EGFR-mutated NSCLC. Mini oral presentations will feature the TROPION-Breast01 trial of Dato-DXd in Chinese patients with metastatic HR positive, HER2 low or negative breast cancer, and final results from the DESTINY-Gastric06 trial of ENHERTU in Chinese patients with HER2 positive advanced gastric or gastroesophageal junction adenocarcinoma. This latter trial led to ENHERTU’s conditional approval in China for this indication. Trials-in-progress posters will detail ongoing ENHERTU trials, including DESTINY-BTC01 evaluating ENHERTU with rilvegostomig in biliary tract cancer and DESTINY-Gastric03 evaluating ENHERTU combined with rilvegostomig and chemotherapy in gastric or GEJ adenocarcinoma. The DESTINY-PanTumor02 trial of ENHERTU will expand to include new cohorts of various HER2-expressing solid tumors.
SABCS will present breast cancer advancements, including a DESTINY-Breast06 subgroup analysis examining the impact of prior endocrine therapy response on ENHERTU outcomes in HR positive, HER2 low or ultralow breast cancer. Primary results from the VALENTINE trial evaluating neoadjuvant HER3-DXd, alone or with letrozole, in high-risk HR positive, HER2 negative early breast cancer will also be presented. Additional data includes biomarker analysis from DESTINY-Breast03 assessing the impact of genomic alterations on ENHERTU compared to T-DM1 in HER2 positive metastatic breast cancer, and quality of life and neurological function data from DESTINY-Breast12. Final results from the DESTINY-Breast08 dose expansion, evaluating ENHERTU with capecitabine or capivasertib in HER2 low metastatic breast cancer, will also be presented.
At ASH, several sub-analyses of the QuANTUM-First trial of VANFLYTA (quizartinib) in newly diagnosed FLT3-ITD positive AML will be presented, including data on co-mutations and their impact on outcomes, and the effect of continuation therapy. Further presentations will focus on the QUIWI trial evaluating VANFLYTA with chemotherapy in FLT3-ITD negative AML, including final results and sub-analyses. The QuANTUM-Wild phase 3 trial design, based on QUIWI, will also be presented. Additionally, results from the VALYM trial of valemetostat in relapsed/refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) and an analysis of circulating tumor DNA from the VALENTINE-PTCL01 trial in relapsed/refractory peripheral T-cell lymphoma will be shared.
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### [PIF Partners Small Molecule SJIA Drug Granted FDA Rare Pediatric Disease Designation](https://www.clinicaltrialvanguard.com/news/pif-partners-small-molecule-sjia-drug-granted-fda-rare-pediatric-disease-designation/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
PIF Partners’ investigational therapeutic, 101-PGC-005 (‘005), has received Rare Pediatric Disease Designation (RPDD) from the FDA for treating systemic juvenile idiopathic [arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (sJIA) flares. This prodrug of dexamethasone targets CD206+ macrophages and is currently in Phase 3 clinical trials in India for ARDS induced by [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/). The RPDD designation underscores the potential of ‘005 to address the unmet medical needs of children with this rare and serious inflammatory disease.
TThe RPDD designation not only validates the company’s research and development efforts but also offers the potential for expedited drug approval via a Priority Review Voucher (PRV). For sJIA patients, ‘005 offers a potential new treatment option with a novel mechanism of action that could improve efficacy and reduce the reliance on corticosteroids and immunosuppressants with their associated side effects. This advancement is particularly important given the limited current treatment options and the debilitating nature of sJIA, which can lead to chronic pain, growth retardation, and life-threatening complications.
‘005, a Type IA prodrug of dexamethasone, specifically targets CD206+ macrophages, believed to play a key role in the inflammatory processes driving sJIA. This targeted approach aims to enhance the anti-inflammatory effects of dexamethasone while minimizing systemic side effects, including the suppression of the hypothalamic-pituitary-adrenal (HPA) axis. The potential for a PRV upon approval could significantly shorten the FDA review process for a subsequent marketing application, accelerating access to this potentially life-changing therapy. This designation also strengthens PIF Partner’s position for collaborations or partnerships to further develop and commercialize ‘005.
The RPDD designation for ‘005 represents a crucial step towards developing a much-needed new treatment for sJIA. This development holds promise for improved outcomes for children suffering from this debilitating disease. It may also pave the way for further research into the role of macrophages in other rare inflammatory diseases and the potential of targeted therapies like ‘005 to address these unmet medical needs. The potential for expedited review and market entry through the PRV program could significantly impact the timeline for making this therapy available to patients.
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**Categories:** News
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### [Rallybio to Initiate RLYB116 Clinical PK/PD Study in 2025](https://www.clinicaltrialvanguard.com/news/rallybio-to-initiate-rlyb116-clinical-pk-pd-study-in-2025/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
Rallybio Corporation announced positive findings from new biomarker analyses and manufacturing process enhancements for [RLYB116](https://www.clinicaltrialvanguard.com/news/rallybio-confirmatory-study-launch-in-q2-2025/), its investigational once-weekly, subcutaneous C5 inhibitor. These advancements support the potential of RLYB116 as a best-in-class treatment for complement-mediated diseases, leading to plans for a confirmatory clinical pharmacokinetic/pharmacodynamic (PK/PD) study in the second quarter of 2025.
Following the completion of a Phase 1 single- and multiple-ascending dose (SAD/MAD) study in late 2023, new biomarker characterization analyses revealed that the initial assay used to measure free C5 overestimated levels by approximately ten-fold. This suggests RLYB116 achieved significantly greater complement inhibition than initially reported in the Phase 1 study.
Concurrent with these findings, manufacturing process enhancements completed in the third quarter of 2024 are expected to further improve RLYB116’s tolerability. Enhanced analytical techniques, including mass spectrometry, have led to further purification of the drug substance. This improved purity is anticipated to yield a more favorable tolerability profile at doses equal to or greater than those evaluated in the Phase 1 MAD study.
The upcoming confirmatory PK/PD study, scheduled for the second quarter of 2025, aims to demonstrate both improved tolerability and complete, sustained complement inhibition. This single-blind, multiple ascending dose study will involve two cohorts of eight participants each, evaluating weekly doses of 150 mg and 225 mg, respectively, over a 4-week treatment period followed by a 10-week follow-up period.
RLYB116, designed for the treatment of complement-mediated diseases, is administered subcutaneously once weekly in small volumes. The previous Phase 1 SAD/MAD study, conducted in healthy participants, explored the safety, tolerability, pharmacokinetics, and pharmacodynamics of RLYB116. This study, completed in the fourth quarter of 2023, employed an adaptive single-blind design with a 4-week treatment duration and a 10-week follow-up. Four cohorts were included, evaluating various dosing regimens, including weekly dosing of 100 mg, three initial doses of 100 mg followed by weekly dosing, weekly 150 mg dosing reduced to 125 mg weekly, and twice-weekly 75 mg escalating to twice-weekly 100 mg.
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### [Ajax Presents Phase 1 Trial Results for Myelofibrosis Treatment](https://www.clinicaltrialvanguard.com/news/ajax-presents-phase-1-trial-results-for-myelofibrosis-treatment/)
**Published:** December 4, 2024
**Author:** Jon Napitupulu
**Content:**
Ajax Therapeutics, a biopharmaceutical company focused on developing advanced JAK inhibitors for myeloproliferative neoplasms (MPNs), will present data from its first-in-human study of AJ1-11095 at the 66th American Society of Hematology (ASH) Annual Meeting. The poster presentation, scheduled for December 8, 2024, in San Diego, will detail the ongoing Phase 1 clinical trial of AJ1-11095, a next-generation Type II JAK2 inhibitor. The study, led by principal investigator John Mascarenhas, MD, of the Icahn School of Medicine at Mount Sinai, is a multicenter, open-label trial evaluating AJ1-11095 as a monotherapy in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not responded to or have discontinued treatment with a Type I JAK2 inhibitor. Additional study details are available on www.[clinicaltrials](https://www.clinicaltrialvanguard.com/article/fda-needs-to-release-clinicaltrials-gov-guidance-now/).gov (NCT06343805).
AJ1-11095 is a novel therapy designed in collaboration with Schrödinger, leveraging advanced computational methods to selectively target the Type II conformation of the JAK2 kinase. This targeted approach aims to improve efficacy and disease modification compared to existing Type I JAK2 inhibitors like [ruxolitinib](https://www.clinicaltrialvanguard.com/news/karyopharm-to-submit-selinexor-ruxolitinib-snda-for-myelofibrosis-in-august-2026/). Preclinical studies suggest AJ1-11095 may offer benefits such as reversing bone marrow fibrosis, reducing mutant allele burden, and maintaining effectiveness against MPN cells resistant to chronic Type I JAK2 inhibition.
Myelofibrosis (MF) is a rare blood cancer affecting approximately 20,000 people in the United States. Characterized by enlarged spleen, bone marrow scarring (fibrosis), progressive anemia, and debilitating symptoms like fatigue, night sweats, itching, and abdominal discomfort, MF significantly impacts patients’ quality of life. Current standard treatment with Type I JAK2 inhibitors often provides only temporary relief of symptoms and limited impact on the underlying disease. Many patients eventually discontinue these treatments due to lack of efficacy, adverse events, or disease progression, highlighting the need for improved therapeutic options. Ajax Therapeutics is dedicated to developing innovative therapies for MPNs, focusing on selective approaches to address the unmet needs of patients with MF. By combining deep expertise in cancer and structural biology with advanced computational drug discovery platforms, the company aims to create more precise and effective treatments for this challenging disease. More information about Ajax Therapeutics can be found at www.ajaxtherapeutics.com.
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### [Omniscience and Inmune Bio Partner to Accelerate Alzheimer's Drug Trial](https://www.clinicaltrialvanguard.com/news/omniscience-and-inmune-bio-partner-to-accelerate-alzheimers-drug-trial/)
**Published:** December 5, 2024
**Author:** Jon Napitupulu
**Content:**
OmniScience and [INmune Bio](https://www.clinicaltrialvanguard.com/news/inmune-bios-xpro-shows-significant-brain-imaging-changes-in-early-alzheimers-trial/) have partnered to utilize OmniScience’s AI-powered platform, Vivo, in INmune Bio’s Phase 2 [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease clinical trial (AD02). Vivo will centralize and analyze the trial’s extensive data in real time, offering immediate insights to improve decision-making and expedite the typically lengthy trial management process. This partnership aims to revolutionize clinical trial operations by leveraging AI for enhanced efficiency and improved patient outcomes.
This collaboration is significant for both companies and the Alzheimer’s research field. For INmune Bio, Vivo provides a critical tool for analyzing complex cognitive data from a large, global trial, potentially accelerating the development of much-needed Alzheimer’s treatments. For OmniScience, this partnership offers valuable real-world application and feedback for Vivo’s ongoing development, solidifying its position as a leader in AI-driven clinical trial solutions. For the broader field, this partnership demonstrates the potential of AI to transform clinical research, leading to faster, more efficient trials and ultimately, quicker access to innovative therapies for patients.
Vivo integrates data from various sources, including electronic data capture (EDC), clinical trial management systems (CTMS), patient-reported outcomes (PROs), and lab results. Its advanced cognitive architecture combines ontological knowledge of trial protocols with clinical expertise, enabling comprehensive qualitative and quantitative data analysis. The AD02 trial has enrolled 208 patients with early Alzheimer’s disease and biomarkers of neuroinflammation. Top-line cognitive results are expected in the second quarter of 2025. The initial rollout of Vivo has already yielded significant benefits, demonstrating its capacity to translate complex data into actionable insights rapidly.
This partnership signals a shift towards smarter, AI-driven solutions in clinical trials. The integration of Vivo in INmune Bio’s AD02 trial paves the way for faster data analysis, more informed decision-making, and potentially accelerated drug development. The real-time insights offered by Vivo could significantly reduce the time it takes to bring new Alzheimer’s treatments to market, offering hope to patients and their families. The continued collaboration between OmniScience and INmune Bio will likely drive further advancements in Vivo’s capabilities, shaping the future of clinical trial management and ultimately improving patient care. The success of this partnership could encourage wider adoption of AI-powered platforms in clinical research across various therapeutic areas.
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### [Maat Pharma Announces First US Patient Treated Under Expanded Access for Graft-Versus-Host Disease](https://www.clinicaltrialvanguard.com/news/maat-pharma-announces-first-us-patient-treated-under-expanded-access-for-graft-versus-host-disease/)
**Published:** December 6, 2024
**Author:** Jon Napitupulu
**Content:**
MaaT Pharma has administered its investigational microbiome therapeutic, MaaT013, to the first patient in the United States under the FDA’s compassionate use program. This treatment is for a patient with acute Graft-versus-Host Disease (aGvHD) who has exhausted other treatment options, including steroids and [ruxolitinib](https://www.clinicaltrialvanguard.com/news/karyopharm-to-submit-selinexor-ruxolitinib-snda-for-myelofibrosis-in-august-2026/), and highlights the unmet medical need for effective aGvHD therapies. The treatment was administered at City of Hope, a leading cancer research and treatment center, by renowned experts in Hematopoietic Cell Transplantation and GvHD.
The compassionate use authorization of MaaT013 in the US is a significant milestone for MaaT Pharma and underscores the increasing global recognition of the potential of microbiome-based therapies for aGvHD. It signifies a step forward in addressing the urgent need for new treatment options for this life-threatening condition, particularly for patients who don’t respond to standard therapies. This development could potentially broaden access to innovative treatments for patients with limited options and offers hope for improved outcomes in aGvHD management.
This first US compassionate use case comes as MaaT Pharma completes patient recruitment for its Phase 3 ARES trial in Europe, with topline results expected in January 2025. The company is also preparing to initiate a US Phase 3 trial for MaaT013 in aGvHD. The availability of clinical batches of MaaT013 facilitates both the ongoing trials and expanded access programs under FDA oversight. Furthermore, additional data from the European Early Access Program will be presented at the ASH 2024 Annual Meeting, offering further insights into the efficacy, safety, and long-term effects of MaaT013.
This first compassionate use case in the US signifies a crucial step in potentially expanding access to MaaT013 for patients with aGvHD who have exhausted standard treatment options. The upcoming clinical trial results and further data presentations will be critical in shaping the future development and potential regulatory approval of MaaT013, potentially offering a new therapeutic approach for patients battling this severe condition. This progress solidifies MaaT Pharma’s position as a leader in developing microbiome-based therapies and contributes to the evolving landscape of aGvHD treatment. It also suggests growing momentum in the field of microbiome therapeutics, highlighting their potential to address complex immunological conditions and improve patient outcomes.
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**Categories:** News
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### [Envision Trial Shows UGN-102's Potential for LG-IR-NMIBC Treatment](https://www.clinicaltrialvanguard.com/news/envision-trial-shows-ugn-102s-potential-for-lg-ir-nmibc-treatment/)
**Published:** December 6, 2024
**Author:** Jon Napitupulu
**Content:**
[UroGen](https://www.clinicaltrialvanguard.com/news/urogens-ugn-103-shows-94-5-six-month-response-in-nmibc-trial/) Pharma announced positive Phase 3 ENVISION trial results for UGN-102, a mitomycin-based intravesical solution for recurrent low-grade intermediate-risk non-muscle-invasive [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/) (LG-IR-NMIBC). The study demonstrated a high complete response rate and durable efficacy in patients, marking a significant step toward a potential new treatment option for this challenging condition. The findings were presented at the Society of Urologic Oncology annual meeting and published in the Journal of Urology.
This news is crucial because LG-IR-NMIBC represents a significant unmet need. Patients with this type of bladder cancer often face frequent recurrences, necessitating repeated surgical procedures. A new, effective, and less invasive treatment option like UGN-102 could substantially improve patient outcomes and quality of life, particularly for elderly patients who may be less able to tolerate repeated surgeries. The positive results from the ENVISION trial could also significantly impact the bladder cancer treatment landscape, offering a much-needed alternative to current standards of care.
The ENVISION trial showed an 82.3% duration of response at 12 months in patients who achieved a complete response after the initial UGN-102 treatment. This durability persisted, with an 80.9% duration of response at both 15 and 18 months. These findings reinforce the initial positive results of a 79.6% complete response rate three months post-treatment. Furthermore, the safety profile of UGN-102 remained consistent with previous trials, characterized by generally mild-to-moderate, resolvable side effects, primarily affecting the urinary tract. The New Drug Application (NDA) for UGN-102 was submitted ahead of schedule and accepted by the FDA, with a target action date set for June 13, 2025.
The positive ENVISION trial results, coupled with the FDA’s acceptance of the NDA, position UGN-102 as a potential game-changer for LG-IR-NMIBC treatment. If approved, UGN-102 could offer a less invasive, more convenient, and potentially more effective treatment option for patients, reducing the need for repeated surgeries and improving long-term outcomes. This development holds promise for a significant advancement in bladder cancer care and could reshape the treatment paradigm for this prevalent condition.
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**Categories:** News
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### [Veracyte Decipher Prostate Test Recommended in NCCN Guidelines](https://www.clinicaltrialvanguard.com/news/decipher-prostate-test-recommended-in-nccn-guidelines/)
**Published:** December 6, 2024
**Author:** Jon Napitupulu
**Content:**
Veracyte’s Decipher Prostate Genomic Classifier is the only gene expression test included in the 2025 National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/). This inclusion solidifies Decipher Prostate’s position as a leading diagnostic tool for assessing the risk of metastatic prostate cancer, enabling clinicians to personalize treatment strategies. The test’s inclusion in the guidelines is a significant validation of its clinical utility and performance.
For Veracyte, this news signifies market leadership and reinforces the strength of their diagnostic platform. For the broader medical community, it provides clinicians with a validated tool for enhanced risk stratification and treatment decision-making, potentially improving patient outcomes. Inclusion in the NCCN Guidelines often leads to broader insurance coverage and increased adoption by healthcare providers, signifying wider access for patients. This, in turn, provides an impetus for further research and development in the field.
The Decipher Prostate test analyzes a 22-gene signature derived from whole-transcriptome analysis using RNA and machine learning. This technology allows for a precise assessment of the risk of metastasis. The test’s clinical utility has been demonstrated in numerous peer-reviewed studies encompassing a large patient population. This rigorous validation process, coupled with its unique inclusion in the NCCN Guidelines, positions Decipher Prostate as a highly reliable and valuable tool in prostate cancer management.
This inclusion in the NCCN guidelines marks a significant advancement in prostate cancer diagnostics and personalized medicine. It is expected to solidify Decipher Prostate’s position as a standard-of-care diagnostic tool, leading to greater accessibility for patients and potentially improved treatment outcomes. Furthermore, it validates Veracyte’s research-driven approach and strengthens its position as a leader in cancer diagnostics, paving the way for future innovations in the field. The broader impact on prostate cancer management is likely to be significant, influencing treatment paradigms and emphasizing the importance of genomic testing in guiding patient care.
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**Categories:** News
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### [Datopotamab Deruxtecan Shows Promising Activity in EGFR-Mutated NSCLC](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-shows-promising-activity-in-egfr-mutated-nsclc/)
**Published:** December 6, 2024
**Author:** Jon Napitupulu
**Content:**
[Datopotamab](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/) deruxtecan (Dato-DXd), a TROP2-directed antibody drug conjugate, showed promising results in a pooled analysis of two clinical trials for patients with previously treated advanced or metastatic EGFR-mutated [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). The analysis demonstrated a confirmed objective response rate of 42.7% and a median overall survival of 15.6 months. This research focuses on patients whose disease progressed despite initial treatment with EGFR tyrosine kinase inhibitors, a common occurrence in this type of lung cancer.
This news is significant because it offers a potential new treatment option for patients with EGFR-mutated NSCLC who have limited effective therapies after their disease progresses on existing treatments, particularly osimertinib. The positive results highlight the potential for Dato-DXd to address a significant unmet medical need in this patient population, potentially changing the treatment landscape for advanced lung cancer. This is especially crucial given the prevalence of EGFR mutations in NSCLC, particularly in Asian populations.
The pooled analysis included 117 patients, with a subset of 96 having received prior osimertinib treatment. The confirmed objective response rate was consistent across both groups, around 43-45%, with a median duration of response of approximately 7 months. Notably, the median progression-free survival was about 5.7-5.8 months and median overall survival reached 14.7-15.6 months, suggesting potential for extended survival in this challenging patient population. The safety profile of Dato-DXd remained consistent with prior studies, with common side effects including stomatitis, alopecia, nausea, and fatigue. No new safety concerns were identified.
The positive data from this pooled analysis, particularly the durable responses and overall survival benefit observed, underscore the potential of Dato-DXd as a valuable treatment option for patients with advanced EGFR-mutated NSCLC who have progressed after standard therapies. This could lead to improved patient outcomes and potentially reshape the treatment paradigm in this challenging disease setting, offering a new avenue for patients who currently have limited effective options. The upcoming trials focusing on patients with TROP2-QCS biomarker-positive tumors could further refine the patient selection strategy and potentially maximize the benefits of Dato-DXd.
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**Categories:** News
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### [Exploring the Future of Membrane Protein Modulation in Clinical Trials with Rectify Pharmaceuticals](https://www.clinicaltrialvanguard.com/executiveinterviews/exploring-the-future-of-membrane-protein-modulation-in-clinical-trials-with-rectify-pharmaceuticals/)
**Published:** December 6, 2024
**Author:** Moe Alsumidaie
**Content:**
In this interview, we speak with Rajesh Devraj, Ph.D., President & CEO and Pol Boudes, M.D., Chief Medical Officer at Rectify Pharmaceuticals about the critical role of membrane-bound proteins, particularly ABC transporters, in disease pathophysiology. Rajesh shares insights into how Rectify Pharmaceuticals, is innovating in this space to address unmet medical needs and expand therapeutic possibilities.
## [](#moe-alsumidaie-how-do-membrane-bound-proteins-like-abc-transporters-impact-disease-pathophysiology-and-clinical-trials)**Moe Alsumidaie: How do membrane-bound proteins like ABC transporters impact disease pathophysiology and clinical trials?**
Rajesh Devraj: Membrane proteins, including transporters, ion channels, and GPCRs, are essential for cellular function, acting as gatekeepers for molecules entering and exiting cells. Many of these proteins have gain or loss of function mutations that drive various diseases. The pharmaceutical industry has traditionally focused on developing inhibitors or antagonists to block these proteins. However, the ability to enhance or restore protein function using small molecules is a relatively unexplored area. For instance, ABC transporters are a class of membrane proteins with 48 human variants, 21 of which have mutations causing diseases.
Rajesh Devraj, Ph.D., President & CEO of Rectify Pharma
By targeting these transporters, we aim to mitigate biological risk by focusing on targets with strong human genetic links to disease, thus providing a more precise therapeutic approach.
## [](#moe-alsumidaie-how-is-rectify-overcoming-challenges-in-targeting-abc-transporters-for-drug-development)**Moe Alsumidaie: How is Rectify overcoming challenges in targeting ABC transporters for drug development?**
Rajesh Devraj: Targeting ABC transporters has been challenging, primarily due to their structural complexity and regulatory mechanisms. However, we are building on the foundational work of companies like Vertex, which successfully targeted the CFTR gene, an ABC transporter, to treat [cystic fibrosis](https://www.clinicaltrialvanguard.com/news/infexs-resp-x-shows-exacerbation-reduction-in-bronchiectasis-study/). At Rectify, we leverage advances in cryo-EM technology, allowing us to visualize membrane proteins at high resolution, and high-throughput screening to identify small molecules that can enhance protein function rapidly. Our platform focuses on developing small molecules that directly bind to and modulate ABC transporters, enhancing their function. This involves a combination of structural biology, cellular screening, and a bespoke chemical library to find hits that increase protein expression efficiently.
## [](#moe-alsumidaie-how-do-you-target-membrane-proteins-to-meet-unmet-needs-in-complex-diseases)**Moe Alsumidaie: How do you target membrane proteins to meet unmet needs in complex diseases?**
Pol Boudes: Our strategy targets membrane-bound proteins with known etiologic mutations that cause diseases. For example, our lead program targets bile acid homeostasis in the liver and biliary tract, addressing diseases like PFIC2 and PFIC3 by modulating ABCB4 and ABCB11 (also known as BSEP) transporters. These transporters are crucial for maintaining bile acid composition and regulating bile efflux, and their dysfunction leads to conditions like cholangitis and cholestasis. By developing dual-targeted positive functional modulators (PFMs), we aim to correct these dysfunctions, providing a therapeutic solution for both rare genetic and more common
Pol Boudes, M.D., Chief Medical Officer at Rectify
diseases. The PFM approach not only addresses the genetic mutations that drives these diseases but also enhances the function of wild-type proteins, broadening the scope of potential treatments, for example to target a disease with a high unmet medical need like Primary Sclerosing Cholangitis (PSC) where there is a functional deficit of ABCB4 and ABCB11.
## [](#moe-alsumidaie-what-are-the-stages-in-developing-pfms-and-how-do-you-validate-them)**Moe Alsumidaie: What are the stages in developing PFMs, and how do you validate them?**
Rajesh Devraj: Developing PFMs involves several critical validation stages. Initially, we conduct high-throughput screens to identify high-quality small molecule hits. These hits are then validated through the mechanism of action assays to ensure they selectively bind to the target protein rather than acting at the RNA level or as broad-spectrum chaperones. Structural biology techniques, such as cryo-EM, map the binding sites of these compounds on the target proteins. In vitro functional assays, such as primary human hepatocytes, use physiological cells to demonstrate the compounds’ efficacy. Finally, in vivo, translational disease models confirm the compounds’ therapeutic potential. These stages ensure our compounds are potent, selective, and effective in restoring protein function, and have the potential to meet the Target Product Profile (TPP) of the diseases we intend to treat.
## [](#moe-alsumidaie-how-will-you-expand-your-pfm-platform-to-tackle-other-complex-diseases)**Moe Alsumidaie: How will you expand your PFM platform to tackle other complex diseases?**
Rajesh Devraj: We are expanding our PFM platform to target other complex diseases. For instance, our ABCC6 program is focused on addressing renal insufficiency and cardio-renal-metabolic diseases by targeting vascular calcification, a key driver of these conditions. Additionally, we are developing a program for a rare neurodegenerative disorder, X-linked adrenoleukodystrophy, targeting ABCD1 and D2. While I cannot provide specific timelines for these programs, our lead program is set for first-in-human studies next year. We continue to explore new targets and indications, leveraging our platform’s capabilities to develop innovative therapies for various diseases.
**Categories:** Article: Executive Interviews
---
### [Innate Pharma & IFLI Announce $7.9M Investment for IPH6501 in Follicular Lymphoma](https://www.clinicaltrialvanguard.com/news/innate-pharma-ifli-announce-7-9m-investment-for-iph6501-in-follicular-lymphoma/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Innate Pharma and the Institute for Follicular [Lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (IFLI) have partnered to explore the potential of IPH6501, Innate’s anti-CD20 [ANKET](https://www.clinicaltrialvanguard.com/news/innate-pharmas-next-gen-anket-iph6501-highlighted-in-science-immunology/)® therapy, in treating follicular lymphoma (FL). This partnership involves IFLI investing initially $3 million in Innate Pharma, with the potential for an additional $4.9 million based on milestone achievements. This investment will support the inclusion of FL patients in Innate’s ongoing Phase 1/2 trial of IPH6501 in patients with relapsed and/or refractory Non-Hodgkin Lymphoma.
This collaboration addresses the critical need for new treatment options for follicular lymphoma, a slow-growing but incurable form of Non-Hodgkin lymphoma. The partnership combines Innate Pharma’s expertise in immunotherapy development with IFLI’s dedication to accelerating follicular lymphoma research, creating a synergistic effort to advance potential treatments and improve patient outcomes. For Innate Pharma, this collaboration not only provides crucial funding but also validates the potential of IPH6501 as a promising therapeutic candidate.
The agreement outlines a staged investment strategy. The initial $3 million investment by IFLI will be used to support the ongoing Phase 1/2 trial and the specific inclusion of follicular lymphoma patients. This investment translates to IFLI acquiring 2.26% of Innate Pharma’s share capital. The potential follow-on investment of up to $4.9 million is contingent upon the achievement of specific milestones, likely related to clinical trial progress and efficacy data. The price for the potential follow-on investment will be determined at the time of investment.
This partnership signifies a potentially significant advance in follicular lymphoma treatment. The combined resources and expertise of Innate Pharma and IFLI offer a promising pathway for developing much-needed therapies. The success of the Phase 1/2 trial and the achievement of pre-defined milestones could lead to further development of IPH6501, offering renewed hope for patients with follicular lymphoma. This collaboration model also illustrates how philanthropic investment can significantly catalyze therapeutic development within a specific disease area, potentially paving the way for similar partnerships in the future. This ultimately creates a positive outlook for the advancement of follicular lymphoma research and the development of innovative treatment options. The outcome of this trial and subsequent research will be vital in shaping the future treatment landscape for this patient population.
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**Categories:** News
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### [Bluebird Bio Lyfgenia Gene Therapy Shows Positive Long-Term Sickle Cell Disease Data](https://www.clinicaltrialvanguard.com/news/bluebird-bio-lyfgenia-gene-therapy-shows-positive-long-term-sickle-cell-disease-data/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Bluebird bio announced updated data from its LYFGENIA gene therapy for [sickle cell](https://www.clinicaltrialvanguard.com/news/mitapivat-met-hemoglobin-goal-in-sickle-cell-phase-3-but-only-40-6-of-patients-responded/)[sickle cell disease](https://www.clinicaltrialvanguard.com/news/agios-discontinues-tebapivat-development-in-sickle-cell-disease/) (SCD) patients with a history of vaso-occlusive events (VOEs). The data, presented at the American Society of Hematology (ASH) Annual Meeting, showed sustained benefits and explored outcomes in patients with a history of stroke, a population not studied in other gene therapy trials for SCD. This news is significant because it provides further evidence of LYFGENIA’s long-term efficacy and safety, reinforcing its potential as a transformative treatment option for SCD. The focus on patients with a history of stroke adds to the understanding of LYFGENIA’s impact on a particularly vulnerable SCD subpopulation.
This research is vital for both the SCD community and the field of gene therapy. SCD is a debilitating genetic disorder with limited treatment options, and strokes are a serious and common complication. LYFGENIA offers the potential for a one-time treatment that addresses the underlying cause of the disease, reducing or eliminating the need for chronic transfusions or stem cell transplants. The data presented at ASH contribute significantly to the growing body of evidence supporting gene therapy as a viable and potentially curative approach for SCD.
As of July 2024, data from 58 patients with a median follow-up of almost four years showed sustained production of anti-sickling adult hemoglobin (HbAT87Q) exceeding 40%, stable total hemoglobin levels without transfusions, and elimination or significant reduction of VOEs in all patients. Crucially, a separate analysis of 27 patients with a history of overt or silent stroke showed no stroke recurrence through nine years of follow-up, a significant finding given the high risk of recurrent stroke in this population. These outcomes were achieved with a safety profile consistent with the underlying disease and the known effects of the treatment procedure. Specifically, there were no reported cases of graft failure, graft-versus-host disease, or vector-related complications. While two cases of acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) were observed in an earlier trial using a different manufacturing process, no such cases were observed in the updated cohort.
The updated and novel data presented at ASH strengthen the case for LYFGENIA as a durable and potentially curative therapy for SCD. The sustained clinical benefits observed in the broader patient population and the promising outcomes in patients with a history of stroke underscore the potential of this gene therapy to transform the lives of individuals living with SCD. This research paves the way for wider adoption of gene therapy approaches for SCD and provides hope for a future where this debilitating disease can be effectively managed or even cured.
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**Categories:** News
---
### [Kite Pharma Yescarta Curative Potential in Relapsed Lymphoma](https://www.clinicaltrialvanguard.com/news/kite-pharma-yescarta-curative-potential-in-relapsed-lymphoma/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Kite, a Gilead Company, presented three analyses of Yescarta (axicabtagene ciloleucel) at the 66th American Society of Hematology (ASH) Annual Meeting, showcasing improved outcomes for patients with relapsed or refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (R/R LBCL). The largest real-world evidence analysis to date confirmed high overall survival (OS) rates consistent with the ZUMA-7 trial, while separate data indicated a decreasing trend in cytokine release syndrome (CRS) and immune-effector cell-associated neurotoxicity syndrome (ICANS) incidence and severity. Additionally, the ALYCANTE study revealed stable or improved long-term quality of life for treated patients.
This news is highly significant for both Kite and the broader oncology field, as it validates the real-world effectiveness of Yescarta, a [CAR T-cell therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/), in treating a challenging form of lymphoma. Demonstrating consistent positive outcomes in a larger and more diverse patient population than clinical trials reinforces the therapy’s potential to become a standard of care. The reduction in CRS and ICANS rates over time highlights the impact of increasing experience and refined treatment protocols in improving patient safety and managing treatment-related toxicities. This can lead to wider adoption of CAR T-cell therapies.
The largest real-world analysis, using data from the CIBMTR registry, included 446 patients and demonstrated a 71% OS rate at 12 months, aligning with the ZUMA-7 trial results. The analysis also revealed an overall response rate (ORR) of 79% and a complete response (CR) rate of 64%. Data from the same registry showed a significant decrease in Grade ≥ 3 CRS incidence in patients treated with Yescarta between 2022-2023 compared to 2017-2019. Similarly, both any-grade ICANS incidence and duration decreased over this period. The ALYCANTE study, focused on health-related quality of life (HRQoL), reported initial declines in some areas one month post-infusion, followed by stabilization or improvement at three and 12 months, respectively. These findings suggest that while some short-term adverse effects are observed, patients can experience long-term quality of life benefits.
These positive findings suggest a promising future for Yescarta and CAR T-cell therapy in R/R LBCL. The confirmation of efficacy and safety in real-world settings should bolster clinician confidence in using Yescarta earlier in the treatment course. The decreasing trend in CRS and ICANS, coupled with stable or improved HRQoL, further strengthens the therapy’s position as a valuable treatment option. Continued research and data collection will be vital to further refine treatment strategies and expand access to this potentially life-saving therapy for a wider range of patients.
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**Categories:** News
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### [Vertex Pharma Casgevy Exagamglogene Autotemcel: Positive Long-Term Data](https://www.clinicaltrialvanguard.com/news/vertex-pharma-casgevy-exagamglogene-autotemcel-positive-long-term-data/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Vertex Pharmaceuticals announced long-term data from global clinical trials of CASGEVY, a [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)/Cas9 gene-edited therapy, for severe [sickle cell](https://www.clinicaltrialvanguard.com/news/mitapivat-met-hemoglobin-goal-in-sickle-cell-phase-3-but-only-40-6-of-patients-responded/)[sickle cell disease](https://www.clinicaltrialvanguard.com/news/agios-discontinues-tebapivat-development-in-sickle-cell-disease/) (SCD) and transfusion-dependent beta-thalassemia (TDT). The data, presented at the American Society of Hematology (ASH) Annual Meeting, showcased the therapy’s sustained clinical benefits, with follow-up periods extending beyond five years for some patients. The median follow-up was 33.2 months for SCD patients and 38.1 months for TDT patients.
This news is significant because it demonstrates the potential of gene editing to provide durable, potentially curative treatments for debilitating inherited blood disorders. For patients with SCD and TDT, current treatment options are often limited, burdensome, and do not address the underlying genetic cause of the disease. CASGEVY offers a potential one-time treatment that could dramatically improve patients’ quality of life and reduce long-term healthcare costs associated with managing these chronic conditions. For Vertex, the positive long-term data reinforces the therapy’s value proposition and strengthens its position as a leader in gene editing and the treatment of genetic diseases.
The presented data highlighted the continued efficacy of CASGEVY in reducing or eliminating vaso-occlusive crises (VOCs) in SCD patients and transfusion requirements in TDT patients. With extended follow-up, the durability of these effects becomes increasingly important, suggesting that a single treatment with CASGEVY could provide long-term disease control. Vertex also provided updates on its efforts to expand access to CASGEVY globally, including approvals in multiple countries, establishment of authorized treatment centers, and agreements with reimbursement authorities. The company also received approval for a third manufacturing facility to meet anticipated patient demand.
The positive long-term data for CASGEVY holds significant promise for the future of SCD and TDT treatment. As more patients receive the therapy and are followed over time, further insights into its long-term efficacy and safety profile will emerge. Continued success in securing reimbursement and expanding access will be crucial for making this potentially transformative therapy available to patients worldwide. The advancements made with CASGEVY also hold implications for the broader field of gene editing, potentially paving the way for the development of similar therapies for other genetic diseases. Further research and development in this area could revolutionize the treatment of a wide range of inherited conditions.
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**Categories:** News
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### [Orca Bio Shows Promising 3-Year Survival in Hematological Malignancies](https://www.clinicaltrialvanguard.com/news/orca-bio-shows-promising-3-year-survival-in-hematological-malignancies/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Orca Bio presented positive three-year follow-up data for its allogeneic T-cell immunotherapy, Orca-T, at the 66th American Society of Hematology (ASH) Annual Meeting. The therapy showed an 86% overall survival rate in patients with acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML), acute lymphoblastic leukemia (ALL), and high-risk myelodysplastic syndrome (MDS), compared to 67% in a historical control group receiving standard post-transplant cyclophosphamide (PTCy). The study also demonstrated potential improvements in non-relapse mortality and relapse-free survival with Orca-T.
Positive long-term data strengthens Orca-T’s potential to become a new standard of care for these life-threatening blood cancers. Currently, treatment options require a difficult balance between managing disease relapse and mitigating severe side effects. Orca-T offers the possibility of improved survival with a reduced risk of complications, addressing a critical unmet need for patients. The positive results also bode well for the ongoing Phase 3 clinical trial comparing Orca-T to conventional allogeneic stem cell transplant, the results of which are expected in the first half of 2025.
The three-year follow-up analysis of the Phase 1b trial compared 77 patients treated with Orca-T plus tacrolimus to a historical cohort of 293 patients receiving alloHSCT with PTCy. All patients in both groups received myeloablative conditioning and had matched donors. The Orca-T group showed superior overall survival at one, two, and three years (96% vs. 82%, 88% vs. 73%, and 86% vs. 67%, respectively). One-year data further indicated a lower non-relapse mortality (1.4% vs. 7.4%) and higher relapse-free survival (83% vs. 71%) with Orca-T. Importantly, the benefit of Orca-T appeared consistent across different age groups. Additionally, the company highlighted the reliable manufacturing process and efficient delivery of Orca-T. Separate data presented at ASH showcased the feasibility of combining Orca-T with CAR-T cell technology (OrCAR-T) in patients with high-risk B-ALL, demonstrating encouraging early safety and efficacy signals.
The promising three-year survival data, coupled with the ongoing Phase 3 trial and the exploration of OrCAR-T, positions Orca Bio as a leader in the development of innovative cell therapies. If the Phase 3 trial confirms these positive findings, Orca-T could significantly improve the treatment landscape for patients with AML, ALL, and MDS, offering a potentially curative option with reduced toxicity. Furthermore, the OrCAR-T platform holds promise for expanding the reach of this technology to other challenging hematological malignancies, paving the way for a new era of personalized and effective cancer treatment.
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**Categories:** News
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### [Merck Zilovertamab Shows 100% Remission in DLBCL Trial](https://www.clinicaltrialvanguard.com/news/merck-zilovertamab-shows-100-remission-in-dlbcl-trial/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Merck announced positive Phase 2 trial data for zilovertamab vedotin, an investigational antibody-drug conjugate targeting ROR1, in combination with standard chemotherapy (R-CHP) for previously untreated diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) ([DLBCL](https://www.clinicaltrialvanguard.com/news/genmabs-epcoritamab-plus-lenalidomide-meets-phase-3-goal-in-relapsed-dlbcl/)). The combination achieved a 100% complete response rate in patients treated with a 1.75 mg/kg dose of zilovertamab vedotin, leading to the establishment of this dose for the upcoming Phase 3 trial. This research aims to address the unmet need for more effective first-line DLBCL treatments, as approximately 40% of patients relapse or develop refractory disease after initial standard therapy.
For Merck, positive Phase 2 results increase the likelihood of successful Phase 3 trials and potential regulatory approval, representing a significant advancement in their oncology pipeline. For the DLBCL patient population, a new, effective treatment option could significantly improve outcomes and survival rates. This is particularly important given the aggressive nature of DLBCL and the limitations of current therapies in preventing relapse.
The Phase 2 waveLINE-007 trial enrolled 36 patients with previously untreated DLBCL. Three dosage arms of zilovertamab vedotin (1.75 mg/kg, 2.0 mg/kg, and 2.25 mg/kg) were evaluated in combination with R-CHP. All three arms showed high complete response rates (100%, 93.3%, and 100%, respectively). The overall complete response rate at the end of treatment was 97.2%. The median duration of response has not yet been reached. While the safety profile was manageable, serious treatment-related adverse events occurred in 11% of patients, and Grade 3-4 treatment-related adverse events occurred in 58% of patients, most commonly neutropenia, nausea, anemia, and diarrhea. The 1.75 mg/kg dose was chosen for Phase 3 due to its high efficacy and seemingly better tolerability.
The positive Phase 2 data suggests zilovertamab vedotin, in combination with R-CHP, holds promise as a potential first-line treatment for DLBCL. The upcoming Phase 3 trial will be crucial in confirming these findings in a larger patient population and further evaluating the long-term efficacy and safety profile. If successful, this therapy could become a valuable new option for patients with DLBCL, potentially improving survival rates and quality of life.
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**Categories:** News
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### [Indapta and Sanofi Collaborate on Myeloma Study](https://www.clinicaltrialvanguard.com/news/indapta-and-sanofi-collaborate-on-myeloma-study/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Indapta Therapeutics and Sanofi are collaborating to investigate the combined efficacy and safety of Indapta’s allogeneic g-NK [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), IDP-023, and Sanofi’s monoclonal antibody, Sarclisa, in patients with relapsed/refractory multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/). This partnership aims to enhance the treatment options for this challenging blood cancer by combining a novel cell therapy with an established targeted therapy. The collaboration involves a Phase 1 study amendment to include a cohort receiving the combination therapy, with Indapta sponsoring the trial, Sanofi providing Sarclisa, and both parties sharing the funding.
For Indapta, this collaboration provides access to Sanofi’s expertise in multiple myeloma and resources to further develop their promising cell therapy. For Sanofi, it offers an opportunity to potentially enhance the efficacy of Sarclisa and expand its presence in the multiple myeloma treatment landscape. For patients with relapsed/refractory multiple myeloma, this combination therapy holds the potential for a more effective treatment option, particularly given the limitations of current therapies.
Initial results from the IDP-023 monotherapy study have shown a mean maximum reduction in serum M-protein or light chain of 73% in eight patients. This promising efficacy, coupled with IDP-023’s unique mechanisms of action, including enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) and targeting of HLA-E expressing cells, differentiates it from conventional NK cell therapies. The combination with Sarclisa, a CD38-targeting antibody, aims to leverage these mechanisms to further enhance anti-tumor activity. Preclinical studies have already demonstrated the superior efficacy of this combination compared to conventional NK cells in combination with monoclonal antibodies. The ability of g-NK cells to release significantly more immune activating cytokines and cell-killing compounds compared to conventional NK cells further underscores the therapeutic potential of this approach.
This collaboration represents a significant step forward in the development of next-generation cell therapies for multiple myeloma. The combination of IDP-023 and Sarclisa has the potential to offer a more effective and potentially curative treatment option for patients with relapsed/refractory disease. The outcome of this Phase 1 study will be crucial in determining the future direction of this combination therapy and its potential to transform the treatment landscape for multiple myeloma. Furthermore, the unique properties of g-NK cells, such as their enhanced ADCC and inherent anti-viral activity, may open doors for exploring their application in other cancers and autoimmune diseases. The platform technology holds promise for developing more potent, accessible, and scalable cell therapies in the future.
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**Categories:** News
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### [Arcellx Announces Positive Data for Relapsed Multiple Myeloma from IMAGINE-1 Study at ASH Meeting](https://www.clinicaltrialvanguard.com/news/arcellx-announces-positive-data-for-relapsed-multiple-myeloma-from-imagine-1-study-at-ash-meeting/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Arcellx, a biotechnology company, announced positive Phase 2 iMMagine-1 study data for [anitocabtagene](https://www.clinicaltrialvanguard.com/news/kite-and-arcellx-advance-multiple-myeloma-program-with-anti-bcma-car-t-momentum/) autoleucel (anito-cel), a [CAR T-cell therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/)[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for relapsed or refractory multiple myeloma (RRMM). The study, with a median follow-up of 9.5 months, showed a 97% overall response rate (ORR) and a 62% complete response/stringent complete response (CR/sCR) rate in 86 efficacy-evaluable patients. Importantly, no delayed neurotoxicities were observed.
This news is significant for Arcellx, its partner Kite (a Gilead Company), and the field of multiple myeloma treatment. Current treatment options for RRMM, particularly for patients with triple or penta-refractory disease, often have limited efficacy and can be associated with significant side effects, including neurotoxicity. Anito-cel’s high response rates and manageable safety profile, especially the absence of delayed neurotoxicity, suggest it could become a valuable new treatment option for these patients.
The iMMagine-1 study enrolled a high-risk patient population, with 87% being triple-refractory and 42% penta-refractory. Patients had received a median of four prior lines of therapy. Beyond the high ORR and CR/sCR rates, 81% of patients achieved a very good partial response or higher, and 93.1% of those evaluable for minimal residual disease (MRD) testing achieved MRD negativity. While median progression-free survival (mPFS) and overall survival (OS) were not reached, the 6- and 12-month PFS rates were 93.3%/78.5% and OS rates were 96.5%/96.5%, respectively. Safety data were also encouraging, with low rates of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and notably, no delayed neurotoxicities observed across more than 150 patients dosed with anito-cel in Phase 1 and iMMagine-1 studies.
These preliminary results suggest that anito-cel holds considerable promise for patients with RRMM. The high response rates, coupled with the manageable safety profile, particularly the lack of delayed neurotoxicity, position anito-cel as a potential best-in-class therapy. Further data from the ongoing iMMagine-1 study and the Phase 3 iMMagine-3 study, which is evaluating anito-cel in earlier lines of therapy, will be crucial in confirming these findings and establishing the long-term efficacy and safety of anito-cel. The positive data generated thus far suggest a potential paradigm shift in the treatment of RRMM, offering new hope for patients who have limited effective options. This also strengthens Arcellx’s position in the competitive CAR T-cell therapy landscape. The results create anticipation for the potential regulatory approval and subsequent commercialization of anito-cel, which could significantly impact the treatment algorithm for RRMM.
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**Categories:** News
---
### [PureTech Presents Lyt-200 Data for Relapsed/Refractory AML/MDS](https://www.clinicaltrialvanguard.com/news/puretech-presents-lyt-200-data-for-relapsed-refractory-aml-mds/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
PureTech Health presented positive Phase 1b trial data for LYT-200, a novel anti-galectin-9 monoclonal antibody, in patients with relapsed or refractory acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML) and myelodysplastic syndromes (MDS). The trial evaluated LYT-200 as a monotherapy and in combination with standard-of-care venetoclax and hypomethylating agents (HMAs). Early results indicate a favorable safety profile and promising efficacy, including partial and complete responses and prolonged disease stabilization.
Relapsed/refractory AML carries a grim prognosis, with limited effective treatment options and short survival times. The data presented suggest that LYT-200 could address this unmet need by providing a new therapeutic approach with the potential to improve outcomes for these patients, either as a standalone therapy or in combination with existing treatments. This is also important for the company as they intend to advance LYT-200 through a Founded Entity called Gallop Oncology.
In the monotherapy arm, 59% of evaluable patients achieved stable disease or better, with two partial responses observed. The mean duration on treatment exceeded two months, notably longer than the typical survival time for patients refractory to venetoclax/HMA therapy. In the combination arm, 80% of evaluable patients achieved stable disease or better, with two complete responses and one patient achieving morphologic leukemia-free state. The mean duration of treatment in the combination arm also surpassed two months. Importantly, LYT-200 demonstrated clinical benefit across various genetic subtypes, including patients with complex cytogenetics and mutations like KRAS, NRAS, and BRAF, who often have poorer responses to standard therapies. Pharmacodynamic analyses confirmed LYT-200’s dual mechanism of action, directly targeting cancer cells and reactivating the immune system.
These promising Phase 1b results pave the way for further clinical development of LYT-200. The data support advancing LYT-200 into a Phase 2 trial for relapsed/refractory AML/MDS, potentially offering a much-needed new treatment option for this challenging patient population. The dual mechanism of action, coupled with the favorable safety profile and evidence of efficacy, positions LYT-200 as a potential game-changer in AML/MDS therapy. Further studies will be crucial to confirm these initial findings and define the optimal role of LYT-200 in the treatment paradigm. The potential for LYT-200 to improve outcomes for patients with relapsed/refractory AML/MDS is substantial, and its continued development represents a significant step forward in the fight against these aggressive blood cancers.
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**Categories:** News
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### [Stoke Therapeutics Zorevunersen Shows Promise in Dravet Syndrome Treatment](https://www.clinicaltrialvanguard.com/news/stoke-therapeutics-zorevunersen-shows-promise-in-dravet-syndrome-treatment/)
**Published:** December 9, 2024
**Author:** Jon Napitupulu
**Content:**
Stoke Therapeutics announced positive data from Phase 1/2a and open-label extension (OLE) studies of zorevunersen, a drug candidate for Dravet syndrome. The studies showed substantial and durable reductions in convulsive seizure frequency in patients given 70mg followed by 45mg maintenance doses of zorevunersen, alongside existing anti-seizure medications. Furthermore, patients demonstrated continuous improvements in cognitive and behavioral measures over two years of treatment.
Currently, no disease-modifying therapies exist for this severe and progressive genetic [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/). Zorevunersen’s potential to address the underlying genetic cause of the disease, rather than just managing symptoms, represents a substantial advancement in the treatment landscape. Positive clinical data strengthens Stoke’s position in developing a first-in-class therapy, potentially offering a significantly improved quality of life for patients and their families.
Patients in the OLE study who received initial 70mg doses of zorevunersen sustained a median seizure reduction of at least 50% from baseline at every observed time point. At eight months, the latest assessed time point for nine patients, the median reduction reached 87%. In addition to seizure reduction, patients also experienced ongoing cognitive and behavioral improvements throughout the two-year treatment period, as measured by the Vineland-3 assessment. These findings support the dosing regimen proposed for the upcoming Phase 3 registrational study. Importantly, zorevunersen has been generally well-tolerated across the studies, with 82% of eligible Phase 1/2a participants continuing into the OLE studies as of June 2024.
The positive data from these studies sets a promising stage for zorevunersen’s development. The observed seizure reduction, combined with the cognitive and behavioral improvements, suggest the potential for a disease-modifying effect. This reinforces the rationale for the upcoming Phase 3 study and strengthens the possibility of zorevunersen becoming a valuable treatment option for Dravet syndrome. These results hold significant implications for the future management of this challenging condition, offering hope for a more effective and comprehensive approach to treatment.
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**Categories:** News
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### [Bayer Transforms Clinical Trials with Digital Protocols](https://www.clinicaltrialvanguard.com/conference-coverage/bayer-transforms-clinical-trials-with-digital-protocols/)
**Published:** December 10, 2024
**Author:** Moe Alsumidaie
**Content:**
At the [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) EU conference, Julius Kusserow, an R&D Standards Expert at Bayer AG, presented a transformative vision for clinical trials through digitalizing clinical protocol information. This initiative, led by TransCelerate BioPharma Inc., aims to accelerate clinical research and enhance healthcare interoperability by shifting from a document-centric to a data-centric approach. This transition promises to streamline processes, reduce errors, and improve collaboration across the industry, ultimately benefiting patients.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#from-documents-to-data)From Documents to Data
The shift from a document-centric to a data-centric approach in clinical trials is a transformative change that promises to revolutionize clinical research. Traditionally, clinical trial protocols have been managed as static documents, which are cumbersome to update and share across platforms. Julius Kusserow highlighted the inefficiencies of this system, where each update requires manual intervention, leading to potential errors and delays. The new approach, “Write Once, Read Many Times,” aims to streamline these processes by creating a single digital version of the protocol accessible to multiple stakeholders simultaneously. This digital version is a static document and a dynamic, structured, machine-readable, and executable protocol. Once written, a protocol can be easily updated and shared across systems without repetitive manual input. This approach saves time and reduces errors, ensuring all stakeholders work with the most current and accurate information.
## [](#industry-wide-interoperability)Industry-Wide Interoperability
One of the most significant challenges in clinical research is the lack of interoperability between different systems and organizations. Kusserow discussed how the digital data flow initiative aims to address this issue by fostering industry-wide interoperability. This involves creating a standardized digital representation of study protocols that can be easily exchanged between organizations, even those that do not typically cooperate. The goal is to enable seamless data exchange with minimal effort, breaking down the silos that often exist between different stakeholders in the clinical research process. By adopting a standardized approach, organizations can ensure that data is consistent and compatible across various platforms, facilitating collaboration and improving the efficiency of clinical trials. This interoperability is crucial for enabling the reuse of standardized protocol data, which can significantly reduce the time and resources required to conduct clinical research.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#the-usdm-standard)The USDM Standard
At the heart of the digital data flow initiative is the Unified Study Definitions Model (USDM) standard. This standard provides a comprehensive framework for ensuring consistency and clarity in clinical trial data. The USDM standard includes a UML logical model, which serves as a class diagram providing the basis for the standard. This model defines the structure and relationships between different data elements, ensuring that all stakeholders clearly understand the data being used. In addition to the logical model, the USDM standard includes CDISC-controlled terminology, which provides further semantics and complements the UML model. This terminology consists of the definition of classes and attributes and the definition of value sets, ensuring that all stakeholders use the same language and definitions when discussing clinical trial data. The USDM standard also includes an API specification, which provides the means to exchange a single study between machines using a JSON API. This allows for seamless data exchange between different systems, ensuring all stakeholders can access the most current and accurate data. The standardization the USDM provides is crucial for enabling the automation and AI-driven advancements that the digital data flow initiative promises.
## [](#phased-evolution-of-ddf)Phased Evolution of DDF
The digital data flow initiative is being implemented in phases, with each phase building on the progress of the previous one. Between July 2021 and July 2022, Phase One laid the groundwork for the initiative by developing the USDM data model, API specifications, and implementation guides. This phase also included creating test files and conformance rules, which are essential for ensuring the standards are implemented correctly. In addition to these foundational elements, Phase One introduced several tools to facilitate protocol development and connectivity to downstream systems. These tools include the Study Definitions Repository (SDR), a centralized repository for study definitions, and the Common Protocol Template (CPT) Interface Tool, which provides a standardized template for creating clinical trial protocols. These tools are designed to streamline the protocol development process, making it easier for stakeholders to create and share protocols. The phased approach to implementing the digital data flow initiative ensures that each element is thoroughly tested and refined before being rolled out to the broader industry. This careful, step-by-step approach is crucial for ensuring the initiative’s success and building confidence among stakeholders in the new system.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#use-cases-and-benefits)Use Cases and Benefits
The digital data flow initiative is set to revolutionize various aspects of clinical trials, offering numerous benefits to different stakeholders. The initiative enables medical writers to work more efficiently within a single digital study design system. This system allows them to access and update study content in real-time, reducing the time and effort required to create and update protocols. Data managers also benefit from digitalizing end-to-end processes, from study design to electronic data capture (EDC). The structured data generated by these processes can be leveraged to track outcomes and monitor progress, providing valuable insights into the effectiveness of clinical trials. Technical experts, on the other hand, can focus on more complex projects that cannot be automated, thanks to the reduction in tedious manual work. By freeing up time for value-added activities, the digital data flow initiative allows technical experts to contribute more effectively to the success of clinical trials. Overall, the initiative promises to improve clinical trials’ speed, quality, and consistency by enabling automation and AI-driven advancements.
## [](#looking-ahead)Looking Ahead
The conference highlighted several upcoming opportunities for stakeholders to engage with the digital data flow initiative. These include the PHUSE EU Connect 2024, and the DDF Solution Showcase Webinar Series, which will provide further insights into the evolution of digital data flows in clinical trials. These events offer stakeholders the chance to learn more about the initiative, share their experiences, and collaborate with others in the industry. Digitalizing clinical protocol information represents a significant leap forward for the clinical trials industry. By embracing a data-centric approach, the digital data flow initiative promises to enhance efficiency, interoperability, and innovation in clinical research, ultimately benefiting patients.
**Categories:** Article: Conference Coverage
---
### [Advancing Diversity in Aesthetic Clinical Trials: Insights from Allergan Aesthetics](https://www.clinicaltrialvanguard.com/executiveinterviews/advancing-diversity-in-aesthetic-clinical-trials-insights-from-allergan-aesthetics/)
**Published:** December 10, 2024
**Author:** Moe Alsumidaie
**Content:**
In a conversation with Dr. Stephanie Manson Brown, Head of Clinical Development at Allergan Aesthetics, we explore the company’s initiatives to enhance diversity in clinical trials. Dr. Brown discusses the motivations behind the Aesthetics Diversity Summit, the challenges of ensuring diverse representation, and the solutions Allergan Aesthetics is implementing. This dialogue underscores the company’s commitment to inclusivity and leadership in the industry.
## [](#moe-why-did-you-convene-the-aesthetics-diversity-summit)**Moe: Why did you convene the Aesthetics Diversity Summit?**
Dr. Manson Brown: We initiated the Aesthetics Diversity Summit to advance the next generation of clinical investigators through training and mentorship. Proposed at our EEDI Ad Board in 2022, this initiative focuses on licensed physician mentees serving diverse patient groups, who are new to clinical trial research with no more than three years of experience. The program includes an 8-hour in-person training session, a visit to our facilities in Irvine, California, and the ADMIRE Pathfinder Training course, a virtual course on clinical trial fundamentals. Participants who complete both training courses are considered for inclusion in [AbbVie](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/)‘s site feasibility network for future trials, based on their research and site readiness, and qualifications against protocol selection criteria. This comprehensive approach ensures that we are equipping new investigators with the necessary skills and knowledge to contribute effectively to clinical research, ultimately enhancing the diversity and inclusivity of our trials.
## [](#moe-what-inspired-your-focus-on-racial-equity-in-aesthetics)**Moe: What inspired your focus on racial equity in aesthetics?**
Dr. Manson Brown: Our mission is to empower confidence in our customers, patients, and colleagues, enabling them to be their best authentic selves. This mission drives our core principle of fostering a culture of inclusion where diversity is celebrated. It’s crucial to have clinical data that covers a broader demographic because the skin needs and aging patterns of every person are unique. Including patients from diverse backgrounds in clinical research supports superior patient outcomes and drives insights into next-generation product development. For instance, our Maximum Difference Study, presented at the ASDS conference, surveyed around 4,000 adults across different demographic
Dr. Stephanie Manson Brown, Head of Clinical Development at Allergan Aesthetics
groups in the US. It revealed varying aesthetic concerns, such as black females prioritizing dark circles, hair loss, and hyperpigmentation, while white females focused on lines, under-eye bags, and sagging skin. These insights highlight the importance of understanding diverse needs to develop effective aesthetic solutions.
## [](#moe-what-are-the-challenges-of-ensuring-diverse-trial-representation)**Moe: What are the challenges of ensuring diverse trial representation?**
Dr. Manson Brown: Ensuring diverse representation in clinical trials involves several challenges. Historical mistrust from underrepresented groups is a significant barrier, as are access issues related to geography, economic factors, and logistics. Additionally, a lack of awareness and education about clinical trials often leads to mistrust. Strict inclusion and exclusion criteria can limit participation, and culturally competent materials may be lacking, presenting relevance and language barriers. To address these challenges, we are investing in infrastructure to support diversity in aesthetic medicine. This includes providing mentorship, training, and knowledge to the next generation of aesthetic medicine talent, enabling them to better meet the needs of our current and future consumers and empower confidence in all. By addressing these barriers, we aim to create a more inclusive environment for clinical trial participation.
## [](#moe-what-solutions-are-you-proposing-for-greater-inclusivity-in-trials)**Moe: What solutions are you proposing for greater inclusivity in trials?**
Dr. Manson Brown: We have developed a comprehensive action plan targeting the pre-trial, study start-up, and post-trial phases to increase enrollment from underrepresented groups. This plan ensures the representation of clinically relevant populations who may benefit from our products. For example, during the pre-trial stage, we are reviewing eligibility criteria to broaden restrictive criteria and remove unintentional barriers, increasing subject access and reducing screen failure and dropout rates. We are also focusing geographically by identifying key locations with diverse subjects and incorporating targeted diversity questions into site feasibility questionnaires. Additionally, we are exploring professional partnerships with organizations like the Skin of Color Society to develop training materials and resources. These efforts aim to create a more inclusive trial design and improve access for diverse populations.
## [](#moe-how-do-these-plans-fit-your-larger-mission-of-celebrating-diversity)**Moe: How do these plans fit your larger mission of celebrating diversity?**
Dr. Manson Brown: The Aesthetic Diversity Summit aimed to foster greater education about diversity in clinical trials and partner with healthcare professionals who have access to diverse patient backgrounds. By providing these physicians with the right resources, we help their patients feel confident in seeking safe and effective results. The Summit was a key milestone, but we recognize that diversity is an ongoing journey requiring continuous efforts to create an inclusive environment reflecting the communities we serve. Our commitment to diversity is not a one-time achievement but an integral part of our mission to empower confidence and celebrate diversity within the aesthetics industry. We strive to lead by example and inspire others to prioritize inclusivity in their clinical trials.
## [](#moe-how-do-you-see-your-leadership-in-trial-diversity-influencing-others)**Moe: How do you see your leadership in trial diversity influencing others?**
Dr. Manson Brown: We anticipate implementing a new process for future studies to define target demographics based on patient benefit, effectiveness, and safety. This ensures our clinical studies accurately reflect the diversity of the real-world population that may benefit from our treatments. Addressing clinical trial diversity requires concerted efforts from manufacturers, researchers, healthcare professionals, and community organizations to build trust, improve access, and create inclusive trial designs. We hope our progress will drive greater conversations and collaboration on this topic, encouraging other companies to follow suit and prioritize diversity in their clinical trials. By setting a precedent, we aim to foster a more inclusive and equitable industry that benefits all patients.
**Categories:** Article: Executive Interviews
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### [Star Therapeutics Presents Interim Clinical Data for VGA039 in VWD Patients at ASH 2024](https://www.clinicaltrialvanguard.com/news/star-therapeutics-presents-interim-clinical-data-for-vga039-in-vwd-patients-at-ash-2024/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
Star Therapeutics announced interim Phase 1 clinical trial data for VGA039, a novel subcutaneous monoclonal antibody for Von Willebrand Disease (VWD). The therapy targets Protein S to rebalance blood clotting, potentially offering the first subcutaneous treatment option for all VWD types. Early data from three patients with high bleeding rates showed significant reductions in annualized bleed rate (ABR) after a single dose, comparable to existing intravenous prophylaxis therapies, but with a more convenient administration route.
This development is potentially groundbreaking for VWD patients, who currently face a significant treatment burden with frequent intravenous infusions. A convenient, subcutaneous option like VGA039 could improve adherence, quality of life, and potentially long-term outcomes by simplifying prophylaxis. The ability of VGA039 to address all VWD types with a single therapy also simplifies treatment decisions and could improve access to effective preventative care. This advancement also represents significant progress in a field where treatment innovation has lagged behind other bleeding disorders like [hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/).
The interim data from the Phase 1 trial showed substantial ABR reductions in patients who received a single 3.0 or 4.5 mg/kg subcutaneous dose of VGA039. Importantly, therapeutic concentrations were sustained for several weeks following the single dose, contrasting with the weekly intravenous infusions required by current treatments. The patients included in this interim analysis had high baseline bleeding rates, exceeding 50 bleeds per year, demonstrating the drug’s potential efficacy in severe cases. Separate analyses presented at the same conference highlighted the substantial unmet need in VWD, with data identifying over 50,000 diagnosed and treated patients in the US and emphasizing the high disease and treatment burden associated with the current standard of care.
These early results are promising for VGA039’s potential to transform VWD treatment. Developing a convenient, subcutaneous, and universally applicable therapy could significantly improve patient care and outcomes. Further research, including planned multi-dose studies, will be critical to fully characterize VGA039’s efficacy and safety profile. Positive results from these future studies could pave the way for a new standard of care in VWD management, offering patients a more manageable and effective treatment option.
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**Categories:** News
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### [Belantamab Mafodotin Shows 42% OS Benefit in Relapsed Multiple Myeloma](https://www.clinicaltrialvanguard.com/news/belantamab-mafodotin-shows-42-os-benefit-in-relapsed-multiple-myeloma/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
The DREAMM-7 trial interim analysis revealed that belantamab mafodotin combined with bortezomib and dexamethasone (BVd) significantly improved overall survival (OS) in relapsed or refractory multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/) patients compared to the daratumumab, bortezomib, and dexamethasone (DVd) combination. This positive OS data reinforces earlier findings from DREAMM-7 and DREAMM-8, which demonstrated improved progression-free survival (PFS) with belantamab mafodotin combinations. The trial focused on patients who had received at least one prior therapy and experienced disease progression.
The observed OS benefit with BVd, a significant reduction in the risk of death, offers new hope for patients who often face limited effective options after relapse or treatment resistance. The sustained survival advantage over time, coupled with improved MRD negativity rates, suggests a potential for deeper and more durable responses compared to existing standard-of-care treatments. This could lead to a shift in treatment paradigms for relapsed/refractory multiple myeloma. The positive OS data strengthens the potential for regulatory approvals, broadening access to this promising treatment option for patients worldwide. This is particularly important given that multiple myeloma often becomes resistant to existing therapies and new treatment options are critically needed.
The interim analysis, with a median follow-up of 39.4 months, showed a 42% reduction in the risk of death with BVd compared to DVd. While median OS was not reached in either arm, projected mOS was 84 months for BVd and 51 months for DVd. The BVd arm also demonstrated a statistically significant improvement in MRD negativity rates, further supporting its potential for durable responses. Safety data for the belantamab mafodotin regimen remained consistent with previous findings, with manageable and resolvable eye-related side effects.
Looking ahead, the positive OS data from DREAMM-7, along with other supportive trial results, positions belantamab mafodotin combinations for potential regulatory approvals in various regions. This advancement holds promise for transforming the treatment of relapsed/refractory multiple myeloma, providing patients with a new, effective therapeutic option that could significantly extend survival. The upcoming DREAMM-10 trial in newly diagnosed, transplant-ineligible patients will further explore the potential of belantamab mafodotin in earlier treatment settings. This continued research could expand the clinical benefit of this therapy to a broader range of multiple myeloma patients.
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**Categories:** News
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### [Kite's Tecartus Car T-Cell Therapy Shows Durable Efficacy and Survival](https://www.clinicaltrialvanguard.com/news/kites-tecartus-car-t-cell-therapy-shows-durable-efficacy-and-survival/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
Kite, a Gilead Company, presented promising data on Tecartus ([brexucabtagene](https://www.clinicaltrialvanguard.com/news/miracle-car-t-cell-hope-from-gilead-for-lymphoma/) autoleucel) for relapsed/refractory [mantle cell lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/) (R/R MCL) and B-cell precursor acute lymphoblastic leukemia (R/R B-ALL) at the American Society of Hematology meeting. The ZUMA-2 trial showed high overall and complete response rates in BTKi-naïve R/R MCL patients, while five-year follow-up data revealed prolonged overall survival, making Tecartus the only CAR T therapy with such long-term data in this population. Real-world evidence analyses further confirmed Tecartus’s effectiveness and safety in a broader R/R B-ALL patient group.
These findings are particularly noteworthy given the aggressive nature and poor prognoses associated with relapsed/refractory MCL and B-ALL. The five-year survival data for Tecartus in R/R MCL represents a significant advancement, offering new hope for patients who have exhausted other treatment options. The consistent efficacy observed across different patient subgroups, including those who are BTKi-naïve, further strengthens Tecartus’s position as a potential treatment option earlier in the disease course. For R/R B-ALL, the real-world data reinforces the clinical trial results and suggests that Tecartus may provide benefit to a wider range of patients than initially studied.
ZUMA-2 Cohort 3, focusing on BTKi-naïve R/R MCL patients, achieved a 91% overall response rate and a 73% complete response rate. The five-year follow-up analysis of ZUMA-2 Cohorts 1 and 2 showed a 39% overall survival rate, a crucial benchmark in this challenging patient population. Real-world data from the CIBMTR registry mirrored the ZUMA-3 trial results, showing high complete response rates and promising survival outcomes in R/R B-ALL. A separate analysis from ZUMA-3 indicated that Tecartus’s effectiveness is not limited by disease burden and that early CAR T [cell expansion](https://www.clinicaltrialvanguard.com/news/lymphodepletion-improves-t-cell-expansion-in-lymphoma-patients/) correlates with better and more durable responses. Furthermore, the data suggests Tecartus can be successfully manufactured and produce robust responses regardless of white blood cell or lymphocyte counts, expanding its potential applicability to a broader patient base.
These positive results suggest that Tecartus has the potential to reshape the treatment landscape for both R/R MCL and R/R B-ALL. The long-term survival data, coupled with consistent efficacy across different patient subgroups and real-world settings, positions Tecartus as a valuable therapeutic option. Further research and clinical experience will help refine patient selection strategies and optimize treatment protocols, potentially leading to even better outcomes for individuals battling these aggressive blood cancers.
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**Categories:** News
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### [Yescarta® Shows Durable Response & Long-Term Survival in NHL Patients](https://www.clinicaltrialvanguard.com/news/yescarta-shows-durable-response-long-term-survival-in-nhl-patients/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
A five-year follow-up analysis of the ZUMA-5 Phase 2 study shows promising results for Yescarta ([axicabtagene](https://www.clinicaltrialvanguard.com/news/kite-pharma-yescarta-curative-potential-in-relapsed-lymphoma/) ciloleucel) in patients with relapsed/refractory non-Hodgkin lymphomas (NHL), specifically follicular lymphoma (FL) and marginal zone lymphoma (MZL). The analysis reveals durable responses and long-term survival after Yescarta treatment, with a median overall response rate of 90% and a complete response rate of 75%. Notably, over half of the patients were alive at the time of analysis, without needing further therapy, suggesting potential curative properties.
These findings are particularly encouraging as FL and MZL are typically considered incurable, with patients frequently experiencing relapse. The demonstrated long-term survival and manageable safety profile of Yescarta offer a significant advancement in treatment options, potentially shifting the paradigm from managing the disease to achieving a cure for these difficult-to-treat blood cancers. This extended follow-up provides crucial real-world data on the long-term efficacy and safety of [CAR T-cell therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/)[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), solidifying its role in the treatment landscape.
The ZUMA-5 study data showcased a median duration of response of 60.4 months and a median progression-free survival of 62.2 months. Importantly, the median time to next therapy was not reached, further emphasizing the sustained benefit of Yescarta. The 60-month overall survival estimate was 69%, reinforcing the potential for long-term remission. No new safety concerns related to Yescarta emerged during this five-year analysis. While some patients experienced events unrelated to the treatment, these were not attributed to Yescarta, and the overall safety profile remained consistent with previous analyses.
This long-term data supports the potential for Yescarta to become a cornerstone in the treatment of relapsed/refractory FL and MZL. The high rates of durable response and long-term survival observed, coupled with the manageable safety profile, suggest a paradigm shift towards curative potential for these indolent NHL subtypes. This advancement not only offers hope for patients but also paves the way for further research and development in CAR T-cell therapies, expanding their application and improving outcomes for individuals battling these challenging cancers. The data underscores the transformative impact of this innovative therapy and its potential to reshape the future of cancer care.
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**Categories:** News
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### [BMS Showcases Cell Therapy Portfolio at ASH 2024](https://www.clinicaltrialvanguard.com/news/bms-showcases-cell-therapy-portfolio-at-ash-2024/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb (BMS) presented 18 analyses at the 66th American Society of Hematology (ASH) Annual Meeting, showcasing the efficacy, durability, and safety of their current cell therapies for blood cancers, along with promising pipeline data for future indications, including autoimmune diseases. The presented data highlighted advancements in blood cancers and beyond, reaffirming BMS’s commitment to [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) innovation. This research aims to improve treatment outcomes and expand therapeutic options for patients with various hematological malignancies and autoimmune conditions.
These findings are crucial for the field of hematology and oncology, offering potential advancements in the treatment of challenging cancers and autoimmune diseases. The five-year survival data for Breyanzi in lymphoma patients, coupled with new data on its effectiveness in earlier treatment lines and real-world settings, strengthens its position as a key therapeutic option. The introduction of a novel GPRC5D-targeted CAR T therapy, arlo-cel, offers a potential first-in-class treatment for heavily pre-treated multiple myeloma patients, addressing a significant unmet need. Further, the progress with CD19 NEX-T in autoimmune diseases like lupus suggests a possible one-time treatment for sustained remission, opening new avenues for managing these chronic conditions.
Key data presented include: five-year overall survival data for Breyanzi (liso-cel) in relapsed or refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (LBCL) with an estimated OS rate of 38% at five years; ctDNA data from the TRANSFORM study supporting its use as an early predictor of durable clinical benefit after second-line LBCL treatment with Breyanzi; real-world data supporting Breyanzi’s use in second-line LBCL regardless of age and in patients with sCNS involvement; two-year follow-up data showing high ORR and CR rates for Breyanzi in relapsed or refractory follicular lymphoma; promising primary analysis results for Breyanzi plus ibrutinib in relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma; a high manufacturing success rate for Abecma demonstrating its commercial reliability; first OS and PFS data for the GPRC5D-targeted CAR T therapy arlo-cel in relapsed or refractory multiple myeloma, showing durable responses and a manageable safety profile; and updated data from a Phase 1 study of CD19 NEX-T showing promising efficacy and safety in patients with severe, refractory autoimmune diseases.
The data presented at ASH reinforces BMS’s position as a leader in cell therapy. The continued development and refinement of existing therapies like Breyanzi, combined with the exploration of novel targets like GPRC5D with arlo-cel, and the expansion into new therapeutic areas like autoimmune diseases with CD19 NEX-T, positions BMS to significantly impact the landscape of cancer and autoimmune disease treatment. The positive clinical results and high manufacturing success rates suggest a promising future for these therapies, potentially offering new treatment paradigms and improved outcomes for patients with a range of serious diseases.
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**Categories:** News
---
### [BeiGene Brukinsa Impact on CLL: 2024 ASH Hematology Data & New Advances](https://www.clinicaltrialvanguard.com/news/beigene-brukinsa-impact-on-cll-2024-ash-hematology-data-new-advances/)
**Published:** December 10, 2024
**Author:** Jon Napitupulu
**Content:**
[BeiGene](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/), soon to be [BeOne](https://www.clinicaltrialvanguard.com/clinops-watchdog/beone-medicines-got-caught-running-a-blood-cancer-ad-that-outran-its-own-data/) Medicines, presented positive clinical data for BRUKINSA ([zanubrutinib](https://www.clinicaltrialvanguard.com/news/sellas-announces-positive-data-from-phase-2a-trial-of-sls009/)) and other pipeline assets in chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) at the 66th American Society of Hematology (ASH) Annual Meeting. Long-term data from the SEQUOIA study showed BRUKINSA significantly improved progression-free survival compared to bendamustine-rituximab, particularly in high-risk patients. Further data highlighted the potential of sonrotoclax, a next-generation BCL2 inhibitor, in combination with BRUKINSA, and BGB-16673, a BTK degrader, in treating CLL and other B-cell malignancies.
These findings are crucial for the CLL treatment landscape. The extended follow-up data from the SEQUOIA trial provides further confidence in BRUKINSA’s long-term efficacy and safety, strengthening its position as a leading treatment option for newly diagnosed and relapsed/refractory CLL. The promising results with sonrotoclax combination therapy suggest a potential new standard of care offering a fixed-duration, all-oral treatment regimen with enhanced efficacy. The emergence of BGB-16673 as a BTK degrader offers a novel mechanism of action that may overcome resistance mechanisms to existing BTK inhibitors, addressing a critical unmet need in patients with treatment-resistant disease.
The 5-year follow-up from the SEQUOIA study revealed a 71% reduction in the risk of progression or death with BRUKINSA compared to the standard chemotherapy regimen. This benefit held across different patient subgroups, including those with the high-risk unmutated IGHV. Data from the sonrotoclax combination therapy showed a remarkable 99% overall response rate and a high rate of undetectable minimal residual disease (uMRD) at a median follow-up of 19.4 months. Early data for BGB-16673 indicated encouraging responses in patients with treatment-resistant CLL and other B-cell malignancies.
The presented data positions BRUKINSA as a cornerstone in CLL therapy, both as a monotherapy and in combination regimens. The development of sonrotoclax and BGB-16673 offers the potential for further advancements in CLL treatment, addressing unmet needs in both the frontline and relapsed/refractory settings. These developments collectively suggest a paradigm shift in CLL management, offering patients more effective and potentially safer treatment options with improved long-term outcomes. The future of CLL treatment appears promising with continued research and development in targeted therapies and combination approaches. The focus on improving long-term survival and minimizing treatment-related side effects is likely to drive further innovation in this field.
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---
### [How The Trump Administration Will Impact The Future of Clinical Trials and Biopharma](https://www.clinicaltrialvanguard.com/article/how-the-trump-administration-will-impact-the-future-of-clinical-trials-and-biopharma/)
**Published:** December 11, 2024
**Author:** Moe Alsumidaie
**Content:**
The re-election of Donald Trump in 2024 marks the start of a dramatic transformation in healthcare, biotech, and clinical trials. The administration’s priorities—deregulation, domestic production, and national security—are set against rising global tensions and America preparing for heightened global conflict – especially with China. These shifts will profoundly impact how drugs and treatments are developed, tested, and delivered, forcing stakeholders to navigate a landscape of urgency and uncertainty.
## [](#healthcare-restructured-efficiency-at-a-cost)**Healthcare Restructured: Efficiency at a Cost**
Trump’s approach to healthcare mirrors a corporate restructuring strategy. By appointing Elon Musk and Vivek Ramaswamy to the Department of Government Efficiency [1](#501e0daa-f9dd-474e-b44e-7363c3502898), the government could run more like a corporation than a government agency. Regulatory agencies like the FDA could streamline drug approvals and potentially slash red tape for efficiency; During the Trump administration, the FDA increased the use of accelerated approval pathways, particularly for oncology drugs. Between 2017 and 2020, the FDA granted accelerated approval to 40 oncology indications, some involving one drug for multiple indications [2](#09d7e364-21c8-49db-bf2f-021139ec5e38). This uptick reflects a broader trend of utilizing expedited programs to hasten patient access to critical therapies.
While accelerated pathways could facilitate innovation, history has shown that prioritizing speed over scrutiny often comes at a cost. The approval of Biogen’s [Alzheimer](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/)’s drug Aduhelm under an accelerated pathway in 2021 exemplifies this risk [3](#1c2ef3e1-7dda-413b-a6d9-7c6f2a23f47d). The decision sparked controversy over the drug’s efficacy and safety, eroding trust in regulatory oversight. As political pressure mounts to deliver rapid results, the FDA could become more of a rubber stamp for approvals, raising concerns about the long-term safety and efficacy of new treatments.
Efficiency is essential, but it cannot come at the expense of oversight. The administration’s challenge will be to balance the demand for speed with the need to maintain public trust in the scientific process. Without this balance, the healthcare system risks undermining the very innovation it seeks to foster.
## [](#big-pharma-thriving-amid-national-priorities)**Big Pharma: Thriving Amid National Priorities**
Rising global tensions and civil unrest in the United States are driving the government to incentivize companies to shift production and manufacturing back to American soil, reducing reliance on outsourcing. For [big pharma](https://www.clinicaltrialvanguard.com/analysis/the-future-of-dcts-is-bright-according-to-big-pharma-and-fda/), the Trump administration’s “America First” policies present opportunities to bolster domestic manufacturing, though these opportunities come with new constraints and challenges to navigate.
Pfizer’s $450 million expansion of its Kalamazoo, Michigan, manufacturing facility exemplifies this trend [4](#3a5f0480-a422-4da0-8cca-f9cac727770a). The investment strengthens Pfizer’s capacity to produce sterile injectable medicines and active pharmaceutical ingredients, which are critical for hospitals worldwide. The facility currently supplies over 150 products to 120 countries, and this expansion positions it to meet rising global demand while advancing sterile manufacturing technologies.
Such investments are facilitated by favorable tax policies, including proposed reductions in corporate tax rates for companies manufacturing domestically. President Trump has advocated for lowering the corporate tax rate to 15% for companies that produce in America, aiming to incentivize domestic manufacturing and reduce outsourcing [5](#9c5465ee-f0f1-491c-94fc-8b2f4b9392a9). These federal incentives and state-level tax benefits make large-scale onshoring projects more financially viable.
This project also highlights the administration’s emphasis on domestic manufacturing as a strategy for securing jobs and protecting America’s critical healthcare infrastructure. The Kalamazoo expansion is estimated to create 450 new jobs in Michigan and represents over $1.1 billion in investments through 2024, blending economic growth with enhanced supply chain resilience in case of a global conflict. While these efforts strengthen domestic capabilities, they also come with higher operational costs that may challenge profitability in the short term, which would reflect on big pharma’s balance sheets.
## [](#biotech-startups-innovation-meets-funding-challenges)**Biotech Startups: Innovation Meets Funding Challenges**
For biotech startups, deregulation presents a double-edged sword. While relaxed oversight of emerging technologies like AI and digital health fosters innovation, shifting public funding priorities could create challenges. Federal agencies like the NIH, which has long been a critical source of early-stage funding, are unlikely to face overall budget cuts; A report from the Congressional Research Service highlights the NIH’s outsized role in shaping biomedical innovation, with over $43 billion in annual funding allocated to projects across the spectrum of basic, applied, and clinical research [6](#964fface-ea50-45c5-b29a-84508e03e7b5). However, the administration may redirect resources toward national security priorities supporting an “America First” directive, such as bioterrorism countermeasures and advanced therapeutics, potentially deprioritizing areas like basic research, rare diseases, or global health. This realignment could leave startups working outside these priorities struggling to secure essential grants.
Biotechs will need to adapt swiftly to these shifting priorities. Companies focused on niche or exploratory fields may be disadvantaged, while those aligned with government-backed objectives could benefit from increased funding opportunities and partnerships. This environment favors projects with immediate applicability to national security or public health emergencies, potentially stifling creativity in less prioritized areas. While redirecting NIH resources might spur innovation in high-urgency fields, it also risks narrowing the diversity of research supported, which could have long-term consequences for the broader biotech ecosystem.
## [](#global-collaboration-under-threat)**Global Collaboration Under Threat**
Geopolitical tensions and the U.S. administration’s “friendshoring” strategy are reshaping the clinical trials landscape. By prioritizing alliances with politically aligned nations, the U.S. aims to reduce reliance on countries like China, which has become a leader in oncology research [7](#f863e6f3-254e-4f2b-807b-ac018b6dfb06). This shift could disrupt collaborative efforts, potentially slowing advancements in critical medical fields.
For example, the U.S. has strengthened partnerships with Vietnam and India to diversify supply chains away from China [8](#2e3e2793-a3fd-4601-ad02-f9757f3d5cdf). Additionally, the creation of an FDA bureau in the Middle East has been proposed to accelerate the development of new healthcare and biomedical technologies, exemplifying friendshoring in the medical field [9](#73597512-bb68-4fa6-a686-e171050d3c94).
Emerging markets such as Brazil and India may fill this void, offering diverse patient populations and improving trial infrastructures. Brazil’s recent regulatory reforms, including streamlined clinical trial approvals under Law No. 14.874, have created a more predictable and efficient environment for international sponsors [10](#bbafdaa1-2b54-4efd-a902-510e1e9852ad). Meanwhile, India’s large, diverse patient population and cost-effective infrastructure make it a hub for clinical trials, as illustrated by Sanofi’s $400 million investment in its Hyderabad Global Capacity Centre [11](#d20b65dc-b9d4-4fff-8e38-2e5a3c117c1e). However, the fragmentation of global research poses risks.
While friendshoring fosters resilient alliances, the fragmentation of global research networks risks slowing innovation in addressing health challenges. Global conflicts and tariffs could destabilize alliances, potentially turning friendshoring countries into adversaries and undermining investments. Clinical trials, which rely on international collaboration, risk losing access to diverse expertise and resources critical for tackling complex health issues. To adapt, stakeholders must carefully evaluate geopolitical risks and opportunities, balancing national security priorities with the need for robust global scientific partnerships.
## [](#the-shadow-of-war-national-security-over-global-health)**The Shadow of War: National Security Over Global Health**
The alignment of healthcare and biotech with national security is profoundly reshaping the industry. As the U.S. gears up for potential conflict, resources are increasingly funneled into defense-related priorities like pandemics [12](#1025b018-259f-4b3e-900e-3ca8c65aa1e1), bioterrorism countermeasures [13](#c075089e-f72c-42c9-93d1-bd4ee0cc21f3), battlefield therapeutics [14](#64a18a6e-ec4f-4a0b-b659-1b849a935703), and supply chain resilience for critical drugs. While these shifts accelerate breakthroughs in high-priority areas, they risk sidelining fields such as rare diseases, preventative care, and global health initiatives.
War also exacerbates supply chain vulnerabilities, potentially disrupting access to key pharmaceuticals. As mentioned earlier, efforts to reshore production may strengthen resilience but could drive up costs, limiting global affordability. Under pressure to fast-track defense-critical innovations, regulatory agencies may face challenges in maintaining rigorous safety and efficacy standards. Beyond funding and logistics, prolonged conflict shifts public and policymaker attention to crisis-driven interventions, often at the expense of long-term health infrastructure and equity.
## [](#what-stakeholders-can-do-to-adapt)**What Stakeholders Can Do to Adapt**
Adapting to this new landscape requires bold action and strategic foresight. Big pharma must double down on reshoring production and align R&D with national priorities without losing sight of global markets critical for sustained growth. Biotech startups should seek private funding and pivot toward defense-related innovations to remain viable. Sponsors and CROs must embrace decentralized trials while ensuring equity and diversity remain central to their designs. Global players should diversify trial locations, leveraging emerging markets in allied regions to maintain efficiency and inclusivity.
## [](#conclusion)**Conclusion**
The Trump administration’s policies and rising global tensions are reshaping the clinical trials and biotech industries. Deregulation, domestic production, and national security priorities present opportunities for innovation and growth but also risks to global collaboration and equity. As America prepares for a new era of conflict, the industry must navigate a fine line between urgency and integrity. Success will depend on adapting to these shifts while maintaining the core principles of scientific and ethical rigor.
**References**
1. [↩︎](#501e0daa-f9dd-474e-b44e-7363c3502898-link)
2. [↩︎](#09d7e364-21c8-49db-bf2f-021139ec5e38-link)
3. [↩︎](#1c2ef3e1-7dda-413b-a6d9-7c6f2a23f47d-link)
4. [↩︎](#3a5f0480-a422-4da0-8cca-f9cac727770a-link)
5. [↩︎](#9c5465ee-f0f1-491c-94fc-8b2f4b9392a9-link)
6. [↩︎](#964fface-ea50-45c5-b29a-84508e03e7b5-link)
7. [↩︎](#f863e6f3-254e-4f2b-807b-ac018b6dfb06-link)
8. [↩︎](#2e3e2793-a3fd-4601-ad02-f9757f3d5cdf-link)
9. [↩︎](#73597512-bb68-4fa6-a686-e171050d3c94-link)
10. [↩︎](#bbafdaa1-2b54-4efd-a902-510e1e9852ad-link)
11. [↩︎](#d20b65dc-b9d4-4fff-8e38-2e5a3c117c1e-link)
12. [↩︎](#1025b018-259f-4b3e-900e-3ca8c65aa1e1-link)
13. [↩︎](#c075089e-f72c-42c9-93d1-bd4ee0cc21f3-link)
14. [↩︎](#64a18a6e-ec4f-4a0b-b659-1b849a935703-link)
**Categories:** Analysis, Article, Article: Opinion
---
### [Acticor Biotech Liquidation Proceedings Announced](https://www.clinicaltrialvanguard.com/news/acticor-biotech-liquidation-proceedings-announced/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
[Acticor Biotech](https://www.clinicaltrialvanguard.com/news/acticor-biotech-disclosure-of-voting-rights-shares-may-2024/), a clinical-stage biopharmaceutical company developing [glenzocimab](https://www.clinicaltrialvanguard.com/news/acticor-biotech-secures-receivership-extension-for-glenzocimab-development/) for cardiovascular emergencies, faces liquidation as the court-appointed administrator has requested the conversion of its receivership proceedings. The Paris Commercial Court will address this request on December 19, 2024. This situation arises after glenzocimab failed to demonstrate significant neurological improvement in stroke patients during three international clinical trials.
This potential liquidation represents a significant setback for the acute ischemic stroke treatment landscape. Glenzocimab, designed to target the GPVI platelet receptor without increasing bleeding risk, held promise as a safer alternative to existing therapies. The failure of the drug to meet its primary endpoint in clinical trials highlights the challenges in developing effective stroke treatments and may impact investor confidence in similar approaches. The unmet need for safer and more effective acute ischemic stroke treatments remains substantial, leaving a void in the market.
Acticor Biotech, founded in 2013, tested glenzocimab in over 800 subjects across various clinical trials, including studies for stroke and myocardial infarction. While the stroke trials did not achieve their primary neurological improvement goals, a sub-group analysis revealed a significant reduction in mortality among patients with intracerebral haemorrhage. Furthermore, the LIBERATE Phase 2 trial, focused on myocardial infarction, is ongoing. Financially, the company has been supported by various European and international investors and has been listed on Euronext Growth Paris since November 2021. The intellectual property related to glenzocimab is protected by patent families extending to 2036.
The impending liquidation of Acticor Biotech underscores the inherent risks in the biotechnology industry. While the future of glenzocimab remains uncertain, the data from the completed trials, particularly the mortality reduction observed in the intracerebral haemorrhage subgroup, could potentially be leveraged for future research or alternative applications. The outcome of the court decision will determine the fate of Acticor Biotech’s assets and intellectual property, potentially impacting future development in this therapeutic area. The liquidation also signifies a loss for investors and highlights the need for continued investment and innovation in the search for effective cardiovascular emergency treatments.
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**Tags:** eClinical Tech News
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### [Tafasitamab Improves PFS in Follicular Lymphoma](https://www.clinicaltrialvanguard.com/news/tafasitamab-improves-pfs-in-follicular-lymphoma/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
The Phase 3 inMIND trial showed positive results for [tafasitamab](https://www.clinicaltrialvanguard.com/news/incyte-tafasitamab-improves-pfs-in-relapsed-refractory-follicular-lymphoma/) combined with lenalidomide and [rituximab](https://www.clinicaltrialvanguard.com/news/budoprutug-shows-response-in-rituximab-experienced-itp-patients/) in treating relapsed or refractory follicular lymphoma (FL). The combination therapy significantly improved progression-free survival (PFS) compared to the control group, reducing the risk of disease progression, relapse, or death by 57%. This improvement was observed across all patient subgroups, regardless of prior treatment history.
This news holds substantial promise for FL patients, who often face a high relapse risk and limited treatment options. The inMIND trial results suggest that the tafasitamab combination could become a new standard of care, offering a more effective and well-tolerated treatment option, potentially leading to longer remission periods and improved quality of life. This is particularly relevant given the current limitations in treating relapsed or refractory FL, where extending remission while preserving quality of life is paramount.
The trial met its primary endpoint, demonstrating a median PFS of 22.4 months for the tafasitamab group versus 13.9 months for the control group by investigator assessment. An Independent Review Committee confirmed these findings. Secondary endpoints, such as time to next treatment, also favored the tafasitamab group. While the combination therapy showed a favorable safety profile, common side effects included neutropenia, diarrhea, and constipation.
This positive data reinforces the potential of tafasitamab combination therapy as a valuable treatment option for relapsed or refractory FL. The significant improvement in PFS, coupled with a manageable safety profile, could reshape the treatment landscape for this patient population. Further research and regulatory submissions are expected to solidify the role of this combination therapy and bring this much-needed treatment option to patients.
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### [Incyte Tafasitamab Improves PFS in Relapsed/Refractory Follicular Lymphoma](https://www.clinicaltrialvanguard.com/news/incyte-tafasitamab-improves-pfs-in-relapsed-refractory-follicular-lymphoma/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
Incyte announced positive Phase 3 inMIND trial results for [tafasitamab](https://www.clinicaltrialvanguard.com/news/tafasitamab-improves-pfs-in-follicular-lymphoma/) combined with lenalidomide and [rituximab](https://www.clinicaltrialvanguard.com/news/budoprutug-shows-response-in-rituximab-experienced-itp-patients/) in relapsed or refractory follicular lymphoma (FL). The trial met its primary endpoint, showing a statistically significant improvement in progression-free survival (PFS) compared to the control group. This combination therapy reduced the risk of disease progression, relapse, or death by 57%.
This news is particularly important for FL patients, who face a high relapse risk and limited treatment options after initial therapies fail. Current treatments often focus on managing symptoms and prolonging remission rather than achieving a cure. The inMIND trial results suggest that this combination therapy could significantly extend the time patients live without disease progression, offering a new and potentially more effective treatment approach. This advancement is crucial because it addresses an unmet need in a common subtype of non-Hodgkin lymphoma.
The inMIND trial involved 548 patients and demonstrated a median PFS of 22.4 months for the tafasitamab group compared to 13.9 months for the control group. Independent review confirmed these findings, showing a consistent PFS benefit. Importantly, this improvement was observed across all patient subgroups, regardless of prior treatment history. The therapy was generally well-tolerated, with the most common side effects being neutropenia, diarrhea, COVID-19 infection, and constipation. These side effects are consistent with other immunotherapy combination regimens. Furthermore, the median time to next treatment was not yet reached in the tafasitamab group, suggesting a durable response.
This positive outcome signifies a potential shift in the treatment landscape for relapsed or refractory follicular lymphoma. The combination of tafasitamab, lenalidomide, and rituximab offers a promising new therapeutic option with a significant improvement in PFS. This could lead to a new standard of care for FL patients, offering hope for better outcomes and improved quality of life. While regulatory approval is still pending, these results pave the way for potential submission to regulatory bodies and, if approved, could significantly impact the lives of FL patients.
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**Categories:** News
**Tags:** eClinical Tech News
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### [City of Hope ASH Conference: Novel Cancer Therapies](https://www.clinicaltrialvanguard.com/news/city-of-hope-ash-conference-novel-cancer-therapies/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
City of Hope researchers presented groundbreaking studies at the 2024 ASH conference, focusing on innovative treatments for various blood cancers, including rare subtypes and graft-versus-host disease. These studies explored novel therapies such as [CAR T-cell therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/)[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), monoclonal antibodies, and targeted inhibitors, while also emphasizing supportive care and improved risk assessment for transplant patients. The research aims to improve outcomes and survival rates for patients with these challenging diseases.
This research is crucial for advancing blood cancer treatment. The focus on rare lymphoma subtypes and specific genetic mutations addresses unmet needs and offers hope for patients with limited treatment options. Developing refined risk-assessment models allows for better-informed treatment decisions, particularly for older adults undergoing transplantation. Moreover, the emphasis on minimizing treatment-related toxicities and improving patients’ quality of life underscores a commitment to patient-centered care.
Several studies showed promising results. [Revumenib](https://www.clinicaltrialvanguard.com/news/revumenib-data-from-beat-aml-trial-published-in-jco-presented-at-eha-2025/) demonstrated sustained efficacy in patients with relapsed or refractory KMT2Ar acute leukemias, leading to FDA approval. Early application of CAR T-cell therapy in older B-ALL patients showed encouraging remission rates with minimal toxicity. A study on transformed indolent non-Hodgkin lymphoma highlighted the effectiveness of CAR T-cell therapy in this high-risk group. Furthermore, a novel anti-CD94 monoclonal antibody showed promise in treating rare cytotoxic T-cell lymphomas. A combination therapy of durvalumab and lenalidomide produced encouraging outcomes for cutaneous T-cell lymphomas. The CHARM model emerged as a valuable tool for predicting transplant outcomes in older adults. A clinical trial demonstrated the potential of engineered T-cell therapies to reduce relapse rates in transplant patients. Finally, preclinical studies highlighted the effectiveness of a PCNA inhibitor in targeting acute myeloid leukemia stem cells.
These research advancements signify a paradigm shift in blood cancer treatment. The development and refinement of targeted therapies offer hope for improved survival rates and reduced toxicities. The focus on rare subtypes and personalized medicine paves the way for more effective and tailored treatments. The continued exploration of innovative therapeutic strategies, coupled with enhanced supportive care, promises to significantly improve the lives of patients with blood cancers.
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### [Visugromab Reverses Checkpoint Inhibitor Resistance](https://www.clinicaltrialvanguard.com/news/visugromab-reverses-checkpoint-inhibitor-resistance/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
CatalYm’s lead drug candidate, [visugromab](https://www.clinicaltrialvanguard.com/news/visugromab-nivolumab-combo-triumphs-in-advanced-cancer-battle/), a monoclonal antibody targeting the immunosuppressant GDF-15, has shown promising results in a Phase 1/2a trial for various solid tumors. The study, published in •Nature•, revealed visugromab’s ability to induce significant and durable cancer remission, especially when combined with the PD-1 inhibitor nivolumab, in patients with late-stage cancers resistant to anti-PD-(L)1 therapies. The research highlights the potential of GDF-15 inhibition as a novel approach to overcome immunotherapy resistance.
This development holds considerable promise for cancer treatment. Current immunotherapies, while effective for some, often face limitations due to developed resistance mechanisms employed by tumor cells. Visugromab’s ability to counteract GDF-15, a key player in this resistance, offers a potential solution to enhance and broaden the efficacy of existing immunotherapies like nivolumab. The observed deep and durable responses, including complete remissions, in patients with limited treatment options underscore the potential clinical impact of this approach. Furthermore, the alleviation of cachexia, a debilitating syndrome linked to elevated GDF-15, offers a potential improvement in quality of life for patients.
The Nature publication presents comprehensive data from the GDFATHER Phase 1/2a trial. Results demonstrate that visugromab, in combination with nivolumab, achieved robust responses in patients with relapsed or refractory [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/), urothelial cancer, and hepatocellular cancer. More than half of the responding patients experienced deeper remissions compared to their initial checkpoint inhibitor treatment. The study also provides substantial translational research demonstrating visugromab’s mechanism of action: it promotes T-cell influx and proliferation within the tumor microenvironment and induces an Interferon-γ signature, both alone and in combination with nivolumab. Further immunogenomic analyses confirm the broad influence of GDF-15 on the tumor microenvironment and its immune landscape.
Visugromab’s success in early clinical trials positions it as a potential game-changer in cancer immunotherapy. The ability to overcome resistance to established checkpoint inhibitors, combined with a promising safety profile and durable responses, opens avenues for new treatment strategies across multiple cancer types. The initiation of a broad Phase 2b program in earlier treatment settings suggests a rapid advancement of visugromab toward broader clinical application and holds potential for significantly improved patient outcomes in the near future.
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### [Stemline Therapeutics: New Therapeutic Strategy for Advanced Breast Cancer](https://www.clinicaltrialvanguard.com/news/stemline-therapeutics-new-therapeutic-strategy-for-advanced-breast-cancer/)
**Published:** December 12, 2024
**Author:** Jon Napitupulu
**Content:**
Menarini Group, along with its subsidiary Stemline Therapeutics and research partner MEDSIR, presented research at the San Antonio [Breast Cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) Symposium 2024 ([SABCS](https://www.clinicaltrialvanguard.com/news/celcuity-to-present-updated-viktoria-1-data-at-sabcs-2025/)) on the ADELA clinical trial. This phase III trial is investigating a combination therapy of elacestrant and everolimus for advanced ER+/HER2- breast cancer patients with ESR1 mutations who have progressed after first-line treatment. The study aims to address the growing challenge of therapeutic resistance in these patients, a significant unmet medical need.
The emergence of ESR1 mutations, often driven by prior endocrine therapy exposure, renders tumors resistant to standard treatments, leading to disease progression. Current treatment options for patients who develop this resistance are limited, emphasizing the crucial need for new therapeutic strategies. The ADELA trial directly addresses this challenge by exploring a combination therapy designed to overcome these resistance mechanisms and improve outcomes for these patients. This research holds significant implications for potentially extending survival and improving quality of life for individuals facing this difficult diagnosis.
The ADELA trial is a randomized, double-blind study comparing the efficacy of elacestrant plus everolimus to elacestrant alone. Elacestrant, an oral selective estrogen receptor degrader, received prior approval based on the EMERALD study results. Everolimus, an mTORC1 inhibitor, has demonstrated the ability to inhibit other resistance mechanisms in this cancer type. Preliminary data from the phase 1b/2 ELEVATE study suggests this combination exhibits efficacy with a manageable safety profile. The ADELA trial’s primary endpoint is progression-free survival, with secondary endpoints including overall survival, toxicity, and quality of life. The trial’s international scope, encompassing multiple countries across Europe, highlights its global significance.
The ADELA trial results could significantly impact the treatment landscape for advanced breast cancer. Positive findings could lead to regulatory approval of the elacestrant and everolimus combination, offering a new therapeutic option for patients who have developed resistance to standard endocrine therapies. Furthermore, the trial’s focus on ESR1 mutations represents a move towards more personalized medicine, tailoring treatment strategies to the specific genetic profile of individual tumors. The international collaboration and presentation at a major oncology symposium underscore the importance of this research and its potential to advance the field.
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**Categories:** News
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### [Atropos Evidence Network Automates Healthcare AI Model Training](https://www.clinicaltrialvanguard.com/news/atropos-evidence-network-automates-healthcare-ai-model-training/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
Atropos Health launched AI model training on its Atropos Evidence Network, a federated healthcare data network containing over 300 million patient records. This allows members to use de-identified real-world data (RWD) to train AI models and deploy them through partners like pharmaceutical companies and health systems. The aim is to improve the safety and efficacy of AI in healthcare by leveraging vast datasets of real patient information.
This development is crucial for the healthcare industry because it addresses a critical need for high-quality, diverse data in AI development. Training AI models on such a large, federated dataset improves the generalizability and reliability of AI tools, leading to more accurate diagnoses, treatment recommendations, and ultimately, better patient outcomes. Moreover, the federated nature of the network allows for broader participation and collaboration, accelerating the pace of AI innovation in healthcare without compromising patient privacy. This collaborative approach can lead to faster development of robust and reliable AI tools that benefit a wider patient population.
Technically, Atropos Health utilizes a vector database and a Clinical Definitions Library within its GENEVA OS™ platform. This provides a standardized, object-oriented schema for data representation, facilitating seamless integration and analysis of diverse data sources. The recent addition of data quality scorecards further enhances the reliability of the data by providing transparency and feedback to data contributors. This focus on data quality is crucial for building trustworthy and reliable AI models. Strategically, the collaboration with companies like [QuantHealth](https://www.clinicaltrialvanguard.com/news/quanthealths-remarkable-ai-powers-215-million-savings-for-pharma-giants/) demonstrates the practical application of this technology for improving clinical trials. By leveraging RWD, companies can simulate trial outcomes with greater accuracy, optimize trial design, and potentially accelerate drug development timelines.
This advancement positions Atropos Health as a key player in the evolution of AI-driven healthcare. The availability of large-scale, high-quality RWD for AI training, combined with a robust platform and strategic partnerships, will likely accelerate the development and adoption of AI solutions across the healthcare ecosystem. This has the potential to transform clinical research, drug development, and ultimately, patient care by providing clinicians with more powerful tools for personalized medicine and more efficient healthcare delivery.
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**Categories:** News
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### [Tobevibart & Elebsiran Get Breakthrough & PRIME for Chronic Hep D](https://www.clinicaltrialvanguard.com/news/tobevibart-elebsiran-get-breakthrough-prime-for-chronic-hep-d/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
Tobevibart and elebsiran, developed by Vir Biotechnology for chronic hepatitis delta (CHD), have received Breakthrough Therapy designation from the U.S. FDA and Priority Medicines ([PRIME](https://www.clinicaltrialvanguard.com/news/introducing-ultherapy-prime-revolutionizing-non-surgical-skin-lifting/)) designation from the EMA. These designations are based on positive safety and efficacy data from the Phase 2 SOLSTICE trial, showing the drug combination’s potential to significantly suppress the hepatitis delta virus. Vir Biotechnology plans to initiate the Phase 3 ECLIPSE registrational program in the first half of 2025.
These designations are crucial for CHD patients who currently face a dire prognosis. CHD is the most severe form of viral hepatitis, leading to increased risks of [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/), cirrhosis, and liver failure, often within five years of infection. Currently, there are no approved treatments in the U.S. and limited options globally. The Breakthrough Therapy and PRIME designations acknowledge the potential of tobevibart and elebsiran to address this critical unmet medical need, offering hope for improved outcomes and potentially extending survival for CHD patients. The expedited development and review processes associated with these designations could significantly reduce the time it takes for these potentially life-saving therapies to become available.
The Phase 2 SOLSTICE trial evaluated the safety, tolerability, and efficacy of tobevibart, both alone and in combination with elebsiran. The primary endpoints included the proportion of participants with undetectable HDV RNA, ALT normalization, and adverse events. Data presented at [AASLD](https://www.clinicaltrialvanguard.com/news/promising-new-data-on-wilson-disease-from-monopar-at-aasld/) The Liver Meeting® demonstrated compelling efficacy in suppressing the virus, suggesting the combination could dramatically alter the course of CHD. Tobevibart, a monoclonal antibody, targets the hepatitis B surface antigen, inhibiting viral entry into liver cells. Elebsiran, a siRNA, degrades hepatitis B virus RNA, limiting the production of the surface antigen. Both therapies are administered subcutaneously.
The Breakthrough Therapy and PRIME designations, coupled with the earlier Fast Track designation and positive opinion on orphan drug designation, underscore the significance of this drug combination. These designations facilitate a more efficient regulatory pathway, potentially accelerating the availability of tobevibart and elebsiran to patients who desperately need effective treatment options. This development sets the stage for a pivotal Phase 3 trial that could ultimately transform the treatment landscape for CHD and offer a new standard of care for this debilitating disease. The anticipated commencement of the Phase 3 ECLIPSE program in the first half of 2025 represents a significant milestone in the fight against CHD.
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**Categories:** News
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### [Corcept Catalyst Trial Shows Positive Results in Cushing's Syndrome & Diabetes Treatment](https://www.clinicaltrialvanguard.com/news/corcept-catalyst-trial-shows-positive-results-in-cushings-syndrome-diabetes-treatment/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
Corcept Therapeutics’ Phase 4 CATALYST study demonstrated that Korlym® significantly improved blood sugar control in patients with both Cushing’s syndrome and difficult-to-control [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/). The study also revealed a higher-than-expected prevalence of Cushing’s syndrome in this patient population, with nearly one in four qualifying patients exhibiting the condition. This research underscores the potential of Korlym as a viable treatment option for a substantial subset of individuals struggling with poorly controlled diabetes.
These findings are crucial for both patients and the medical community. The study highlights the potential for misdiagnosis and ineffective treatment among patients with uncontrolled type 2 diabetes who may actually be suffering from undiagnosed Cushing’s syndrome. The significant improvement in blood sugar control observed with Korlym suggests that addressing the underlying hormonal imbalance can lead to better outcomes for these individuals. Furthermore, the higher-than-expected prevalence of Cushing’s syndrome in this population necessitates increased awareness and screening among healthcare professionals. This could lead to earlier diagnosis and treatment, preventing long-term complications associated with both Cushing’s syndrome and uncontrolled diabetes.
In the treatment phase of the CATALYST study, patients receiving Korlym experienced a 1.47% reduction in hemoglobin A1c from baseline, compared to a 0.15% reduction in the placebo group. This placebo-adjusted reduction of 1.32% was statistically significant (p < 0.0001). The safety profile of Korlym remained consistent with its existing label, and no new side effects were reported. The first part of the CATALYST study screened 1,057 patients with difficult-to-control type 2 diabetes and identified 23.8% as having hypercortisolism. 136 of these patients were then randomized into the treatment phase. The CATALYST study findings hold significant implications for the future management of difficult-to-control type 2 diabetes. The results suggest that screening for Cushing’s syndrome should become a standard practice in patients who struggle to control their blood sugar despite optimal therapies. Increased awareness and diagnosis of Cushing’s syndrome could lead to more targeted and effective treatment strategies, ultimately improving patient outcomes and reducing the long-term burden of this condition. This may also expand the market for Korlym by identifying a substantial, previously unrecognized patient population that could benefit from this therapy.
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**Categories:** News
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### [Positive Results for Lunresertib & Camonsertib Combo in Gynecologic Trial](https://www.clinicaltrialvanguard.com/news/positive-results-for-lunresertib-camonsertib-combo-in-gynecologic-trial/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
Repare Therapeutics announced positive data from its Phase 1 MYTHIC trial combining lunresertib and camonsertib (Lunre+Camo) in patients with endometrial and platinum-resistant ovarian cancers. The combination therapy showed promising overall response rates (ORR) of 25.9% and 37.5% respectively, with nearly half the patients experiencing progression-free survival at 24 weeks. These findings stem from 51 evaluable patients enrolled in the gynecologic cancer expansion cohort, with results suggesting a favorable tolerability profile compared to current treatments.
This news holds significant promise for patients with gynecologic cancers, particularly those with endometrial or platinum-resistant [ovarian cancer](https://www.clinicaltrialvanguard.com/news/imunons-imnn-001-shows-lower-residual-disease-in-phase-2-ovarian-cancer-study/), who often face limited treatment options and poor prognoses after recurrence. The high response rates and extended progression-free survival observed with Lunre+Camo suggest a potential improvement over existing standards of care, offering a new therapeutic avenue for this patient population, especially given its favorable tolerability profile. Furthermore, the targeted nature of the therapy, focused on specific genetic biomarkers, underscores the growing importance of precision oncology in addressing difficult-to-treat cancers.
The MYTHIC trial evaluated Lunre+Camo at its recommended Phase 2 dose. In endometrial cancer patients, 25.9% achieved an ORR, and 48.1% maintained progression-free survival at 24 weeks. For platinum-resistant ovarian cancer patients, the ORR was 37.5%, with 45.8% exhibiting progression-free survival at 24 weeks. Notably, the therapy was well-tolerated, with anemia being the most common adverse event (26.9%, Grade 3). Repare has engaged with both the FDA and the European Medicines Agency for guidance on the registrational path forward.
These positive results pave the way for a planned Phase 3 trial of Lunre+Camo in endometrial cancer, anticipated to begin in the second half of 2025. This signifies a crucial step towards potentially establishing a new standard of care for these aggressive cancers. The combination therapy’s targeted approach and encouraging efficacy data suggest a potential shift in the treatment landscape for gynecologic oncology, offering hope for improved outcomes and a better quality of life for patients facing these challenging diseases. The data also strengthens the potential for Lunre+Camo in other cancers with similar genetic vulnerabilities.
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**Categories:** News
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### [Zelenectide Pevedotin Program Updates & Nectin-4 Gene Amplification Strategy](https://www.clinicaltrialvanguard.com/news/zelenectide-pevedotin-program-updates-nectin-4-gene-amplification-strategy/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
Bicycle Therapeutics presented data at the 2024 San Antonio Breast Conference Symposium (SABCS) highlighting the enhanced anti-tumor activity of their drug, zelenectide pevedotin, in [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) patients with NECTIN4 gene amplification. They also announced topline combination data for zelenectide pevedotin plus pembrolizumab in first-line metastatic urothelial cancer (mUC) patients, along with updates on the Duravelo-2 trial and monotherapy data in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). This research focuses on the potential of NECTIN4 gene amplification as a biomarker to identify patients most likely to benefit from zelenectide pevedotin.
These findings have substantial implications for cancer treatment. The identification of NECTIN4 gene amplification as a potential predictive biomarker for zelenectide pevedotin’s efficacy could personalize treatment strategies. This personalized approach could lead to improved outcomes for patients with specific genetic profiles, especially in cancers like triple-negative breast cancer (TNBC) and NSCLC, where effective treatment options are limited. Moreover, the data on zelenectide pevedotin in combination with pembrolizumab in mUC suggests a potential new treatment option for patients ineligible for cisplatin, a standard chemotherapy drug. This could broaden the treatment landscape for mUC and offer an alternative with a potentially improved safety profile compared to existing therapies.
Technically, the data demonstrated a 60% overall response rate for the combination of zelenectide pevedotin and pembrolizumab in first-line mUC, comparable to other therapies. In heavily pretreated breast cancer and NSCLC patients with NECTIN4 gene amplification, the data indicated enhanced anti-tumor activity with zelenectide pevedotin monotherapy. Phase 2/3 Duravelo-2 trial results, including dose selection and topline data, are anticipated in the second half of 2025. Strategically, Bicycle Therapeutics is focusing on developing zelenectide pevedotin in various cancer types using NECTIN4 gene amplification as a patient selection tool. Planned Phase 1/2 trials in breast cancer, lung cancer, and multiple other tumor types in 2025 will further investigate this approach.
This research suggests a potential shift towards more personalized cancer therapies. The emerging importance of NECTIN4 gene amplification as a predictive biomarker may reshape how certain cancers are treated. It could lead to a more targeted and efficient approach, improving outcomes for patients with NECTIN4 amplified tumors. The ongoing and planned trials will provide valuable insights into the efficacy and safety of zelenectide pevedotin across different cancer types and further refine its potential use in a personalized oncology setting. Furthermore, it positions Bicycle Therapeutics as a key player in developing targeted therapies for Nectin-4 associated cancers.
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**Categories:** News
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### [Beigene's Global MAT2A Inhibitor Licensing Deal](https://www.clinicaltrialvanguard.com/news/beigenes-global-mat2a-inhibitor-licensing-deal/)
**Published:** December 13, 2024
**Author:** Jon Napitupulu
**Content:**
BeiGene, soon to be [BeOne](https://www.clinicaltrialvanguard.com/clinops-watchdog/beone-medicines-got-caught-running-a-blood-cancer-ad-that-outran-its-own-data/) Medicines, has secured global licensing rights for SYH2039, a novel MAT2A inhibitor, from CSPC. This drug targets the MTAP gene deletion, a mutation present in roughly 15% of all cancers, including prevalent types like glioblastoma, pancreatic cancer, and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/). The deal involves an upfront and milestone-based payment structure totaling $150 million, plus royalties for CSPC.
This licensing agreement positions BeiGene to address a significant unmet medical need within the oncology field. The MTAP deletion is a common driver in multiple difficult-to-treat cancers, making it a high-value target for therapeutic intervention. The potential synergy of SYH2039 with BeiGene’s in-house PRMT5 inhibitor, BGB-58067, further amplifies the significance of this acquisition, offering a potentially powerful combination therapy approach for these cancers. Addressing this specific genetic vulnerability could lead to improved treatment outcomes for a considerable number of patients across various cancer types. This move also solidifies BeiGene’s expansion into targeted therapies for solid tumors, marking a strategic shift beyond their established presence in immune-oncology with tislelizumab.
Financially, the deal structure mitigates some risk for BeiGene by tying a significant portion of the payment to developmental and commercial milestones. This allows BeiGene to invest strategically while preserving capital if the drug doesn’t meet expectations. The agreement grants BeiGene comprehensive global rights to SYH2039, encompassing development, manufacturing, and commercialization, which streamlines future operations and maximizes potential returns. Strategically, the acquisition of SYH2039 aligns with BeiGene’s intent to strengthen its pipeline of best-in-class therapies, particularly in lung, breast, and gastrointestinal cancers, and reinforces the company’s commitment to focus on solid tumors. Additionally, the upcoming clinical trial for BGB-58067, expected before year-end, combined with the SYH2039 acquisition, underscores BeiGene’s focus on developing a diverse portfolio of treatment options.
This licensing agreement signals a focused expansion into targeted cancer therapies and establishes a foundation for potentially impactful combination treatment strategies. The success of SYH2039, especially in combination with BGB-58067, could significantly reshape the treatment landscape for a sizable population of cancer patients with the MTAP deletion, offering new hope for improved outcomes. The development trajectory of these assets, alongside BeiGene’s other ongoing programs, will be a key indicator of their future success in solid tumor oncology.
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**Categories:** News
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### [How Sangamo is Navigating the Future of Gene Therapy for Rare Diseases](https://www.clinicaltrialvanguard.com/executiveinterviews/how-sangamo-is-navigating-the-future-of-gene-therapy-for-rare-diseases/)
**Published:** December 16, 2024
**Author:** Moe Alsumidaie
**Content:**
In this interview, we speak with Nathalie Dubois-Stringfellow, Chief Development Officer at Sangamo Therapeutics, about the FDA’s accelerated approval pathway and its impact on rare disease treatments, focusing on Fabry disease. We discussed the benefits and challenges of surrogate endpoints, post-approval trials, and ethical considerations for patient access.
## [](#moe-alsumidaie-how-does-the-fdas-accelerated-approval-pathway-impact-gene-therapy-strategies-for-rare-diseases-like-fabry)Moe Alsumidaie: How does the FDA’s accelerated approval pathway impact gene therapy strategies for rare diseases like Fabry?
Nathalie Dubois-Stringfellow: The FDA’s accelerated approval program is crucial for early drug approval in serious conditions with unmet needs, especially for rare diseases like Fabry. This pathway allows for earlier patient access, potentially preventing disease progression. Despite standard care, Fabry patients often experience severe symptoms, making this pathway vital. It also helps companies avoid abandoning programs due to potential lengthy and costly trials in rare diseases with limited patient numbers. We are motivated to secure a partner for Fabry commercialization to address this unmet need swiftly. Peter Marks at the FDA is keen on bringing transformational treatments to patients, aligning with our goal to expedite the availability of our gene therapy for Fabry patients.
Nathalie Dubois-Stringfellow, Chief Development Officer at Sangamo Therapeutics
## [](#moe-alsumidaie-what-are-the-benefits-and-limitations-of-using-egfr-slope-as-a-surrogate-endpoint-for-accelerated-approval-in-this-therapy)Moe Alsumidaie: What are the benefits and limitations of using eGFR slope as a surrogate endpoint for accelerated approval in this therapy?
Nathalie Dubois-Stringfellow: Renal issues like proteinuria and decreased eGFR occur early in Fabry patients, leading to renal failure and early death. eGFR is a key clinical measurement of renal function, and the FDA considers it an acceptable efficacy endpoint for Fabry disease. Our data from 18 patients treated with [ST-920](https://www.clinicaltrialvanguard.com/news/fda-accepts-bla-submission-for-st-920-fabry-disease-drug/) showed a statistically significant positive mean annualized eGFR slope, which is remarkable compared to the negative slope seen with approved therapies. The FDA agreed to use this endpoint for accelerated approval. Patients have expressed challenges with current treatments, such as side effects and logistical issues with bi-weekly infusions, which our single-infusion gene therapy aims to address. Existing therapies like enzyme replacement therapy (ERT) improve eGFR values but still result in a negative overall slope, highlighting the potential of ST-920 to offer a more effective solution.
## [](#moe-alsumidaie-what-challenges-should-be-anticipated-in-designing-post-approval-confirmatory-trials-for-st-920-in-fabry-patients)Moe Alsumidaie: What challenges should be anticipated in designing post-approval confirmatory trials for ST-920 in Fabry patients?
Nathalie Dubois-Stringfellow: We plan to discuss post-approval requirements with the FDA in a pre-BLA meeting, before filing a BLA for accelerated approval in the second half of 2025. We do not anticipate needing additional trials beyond the STAAR study, which simplifies the path to full approval and emphasizes the robustness of our current data. The FDA has advised that eGFR slope at 104 weeks from the Phase 1/2 STAAR study may be assessed to verify clinical benefit, which we will have in mid- 2026. This approach allows us to focus on gathering comprehensive data from our existing trial, ensuring we meet regulatory requirements efficiently.
## [](#moe-alsumidaie-how-does-st-920s-mechanism-compare-to-current-enzyme-replacement-therapies-and-other-emerging-treatments-for-fabry)Moe Alsumidaie: How does ST-920’s mechanism compare to current enzyme replacement therapies and other emerging treatments for Fabry?
Nathalie Dubois-Stringfellow: ST-920 is a one-time intravenous gene therapy infusion that delivers a healthy copy of the GLA gene to the liver, producing the alpha-Gal enzyme to clear toxins. Our trial shows patients achieving and maintaining physiological levels of this enzyme, with the longest follow-up over four years. This constant enzyme level contrasts with enzyme replacement therapy’s (ERT) peaks and troughs. Additionally, we see a significant reduction or disappearance of antibodies against alpha-Gal in patients who started the trial with preexisting antibodies. This is an important point as antibodies against alpha-gal can affect ERT efficacy. This suggests a potential for immune tolerization brought by the gene therapy treatment, which is extraordinary and highlights the potential of ST-920 to offer a more consistent and effective treatment option for Fabry patients.
## [](#moe-alsumidaie-what-ethical-considerations-should-be-addressed-to-ensure-equitable-patient-access-to-st-920-given-the-high-costs-of-gene-therapies)Moe Alsumidaie: What ethical considerations should be addressed to ensure equitable patient access to ST-920, given the high costs of gene therapies?
Nathalie Dubois-Stringfellow: Addressing affordability and economic barriers is crucial. High costs can exclude uninsured or underinsured patients and burden families and healthcare systems. Strategies like outcome-based pricing and reimbursement agreements can help, as well as subscription-based models, to manage upfront costs. Geographical disparities also need addressing, with investments in infrastructure and telemedicine to ensure access in rural areas. We are committed to educating stakeholders and engaging with patient advocacy groups to address access needs. Collaboration across the healthcare ecosystem, including payers, regulators, and patient advocacy groups, is essential to develop innovative solutions that ensure broad access to our potentially life-changing therapy. We believe that working together can overcome these challenges and make ST-920 accessible to all who need it.
## [](#moe-alsumidaie-how-might-the-fdas-decision-to-use-the-accelerated-approval-pathway-influence-future-regulatory-approaches-for-other-gene-therapies)Moe Alsumidaie: How might the FDA’s decision to use the accelerated approval pathway influence future regulatory approaches for other gene therapies?
Nathalie Dubois-Stringfellow: The accelerated approval pathway supports companies in developing treatments for rare diseases with high unmet needs. With only 5% of the 10,000 rare diseases having approved treatments, this pathway provides hope for previously untreatable conditions. The FDA’s support is crucial in advancing genomic medicine, and we hope it will be a game-changer, encouraging other companies to follow. Good data and safety can reduce the need for lengthy trials, vital for rare diseases. We are grateful for the FDA’s willingness to consider this pathway for our product and believe it could unlock opportunities for other companies to develop treatments for rare diseases. This approach could revolutionize how we address rare diseases, providing new hope for patients worldwide.
**Categories:** Article: Executive Interviews
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### [Contineum PIPE-791 Phase 1b PET Trial Begins Patient Dosing](https://www.clinicaltrialvanguard.com/news/contineum-pipe-791-phase-1b-pet-trial-begins-patient-dosing/)
**Published:** December 17, 2024
**Author:** Jon Napitupulu
**Content:**
Contineum Therapeutics has initiated a Phase 1b positron emission tomography (PET) trial for PIPE-791, a novel brain-penetrant, small molecule antagonist of the lysophosphatidic acid 1 receptor (LPA1R). This open-label, single-center trial aims to correlate pharmacokinetics with receptor occupancy in healthy volunteers, as well as patients with idiopathic pulmonary fibrosis (IPF) and progressive [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (PrMS), with topline data anticipated in Q2 2025. The trial will use a PET tracer of the LPA1 receptor to evaluate receptor occupancy in the brain and lungs.
This trial holds significant implications for the potential treatment of IPF and PrMS. Currently, treatment options for both conditions remain limited, with many patients experiencing disease progression despite existing therapies. PIPE-791 offers a novel mechanism of action by targeting the LPA1 receptor, which plays a role in fibrosis and neuroinflammation, the key drivers of IPF and PrMS, respectively. Demonstrating target engagement in these specific disease settings could pave the way for a new therapeutic approach, potentially addressing unmet needs for patients suffering from these debilitating conditions.
The Phase 1b trial is designed to establish a crucial link between the drug’s pharmacokinetics (how the body processes the drug) and its pharmacodynamics (how the drug affects the body). Specifically, it will measure how the concentration of PIPE-791 in the body relates to the occupancy of LPA1 receptors in the brain and lungs. This information will be essential for determining the optimal dose of PIPE-791 for future efficacy trials in IPF and PrMS. Successful demonstration of target engagement through receptor occupancy in patients with these diseases will further validate the therapeutic potential of PIPE-791 and inform the design of subsequent clinical trials.
The outcome of this Phase 1b trial will be a critical inflection point for Contineum Therapeutics and the development of PIPE-791. Positive results demonstrating a clear PK/PD relationship and target engagement in the disease setting would significantly de-risk the program and bolster confidence in its potential to become a first-in-class treatment for both IPF and PrMS. This data will be crucial in guiding the dose selection for future Phase 2 trials and potentially accelerating the development timeline of this promising drug candidate. It could also attract further investment and partnerships, positioning Contineum as a key player in the development of novel therapies for these underserved patient populations. Ultimately, the success of this trial could bring a much-needed new treatment option to patients suffering from IPF and PrMS.
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**Categories:** News
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### [Lerodalcibep Biologic License Application Submitted to FDA](https://www.clinicaltrialvanguard.com/news/lerodalcibep-biologic-license-application-submitted-to-fda/)
**Published:** December 17, 2024
**Author:** Jon Napitupulu
**Content:**
[LIB Therapeutics](https://www.clinicaltrialvanguard.com/news/lib-therapeutics-hasten-biopharmaceuticals-lerodalcibep-trial-approved-in-china/) has submitted a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) to the U.S. FDA for Lerodalcibep, a novel PCSK9 inhibitor designed to lower LDL-C in patients with atherosclerotic cardiovascular disease (ASCVD) or at high risk for ASCVD, and primary hyperlipidemia, including familial hypercholesterolemia. Lerodalcibep is administered as a once-monthly, self-administered subcutaneous injection with long ambient stability, offering a potentially more convenient alternative to existing PCSK9 inhibitors. A Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) is planned for mid-2025.
This advancement is potentially transformative for cardiovascular disease management. Current oral therapies are often insufficient for patients to achieve optimal LDL cholesterol levels, leaving a significant unmet need. Lerodalcibep’s demonstrated robust and sustained LDL-C lowering capabilities, coupled with its convenient administration and storage, could significantly improve patient adherence to long-term treatment, a crucial factor in managing LDL cholesterol and reducing cardiovascular risk. This is particularly important for the substantial population of patients with familial hypercholesterolemia, who often struggle to manage their cholesterol levels effectively.
The BLA submission is based on data from a comprehensive development program involving 2,900 patients, encompassing five global Phase 3 studies. These studies included over 2,300 patients already on maximally tolerated statins and other oral agents but still requiring further LDL-C reduction. The trials evaluated Lerodalcibep’s safety and efficacy in patients with or at high risk for CVD, including those with heterozygous and homozygous familial hypercholesterolemia. Participants received once-monthly doses for up to 52 weeks in the placebo-controlled trials, with over 2,400 continuing into a 72-week open-label extension. Lerodalcibep is a third-generation PCSK9 inhibitor designed as a small-volume, subcutaneous injection with no refrigeration requirement. Its anti-PCSK9 binding domain, an 11-kDa polypeptide called an adnectin, is engineered for high-affinity binding and fused to human serum albumin to enhance plasma half-life.
The submission of the BLA for Lerodalcibep marks a significant step towards potentially reshaping the landscape of LDL-C management. If approved, Lerodalcibep’s patient-friendly profile could significantly improve adherence to therapy, leading to better long-term outcomes for patients with CVD or at high risk, particularly those with familial hypercholesterolemia. The anticipated EMA submission further underscores the potential for this treatment to address a substantial global need. The development of a more convenient and efficacious PCSK9 inhibitor may ultimately lead to wider adoption of this class of drugs and a greater impact on [cardiovascular health](https://www.clinicaltrialvanguard.com/uncategorized/ema-reports-near-record-104-medicine-approvals-in-2025-with-strategic-focus-on-cardiovascular-health/) worldwide.
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**Categories:** News
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### [Naropa University and Center for Psychedelic Studies Separate](https://www.clinicaltrialvanguard.com/news/naropa-university-and-center-for-psychedelic-studies-separate/)
**Published:** December 17, 2024
**Author:** Jon Napitupulu
**Content:**
Naropa University’s Center for Psychedelic Studies has become an independent entity, the Memoru Center for Visionary Healing Arts, in collaboration with therapists and researchers Marcela Ot’alora, Bruce Poulter, and Sara Gael Beauregard. This new center will focus on clinical care, training, and research related to psychedelic therapies, with plans to open clinics and expand training programs. The separation allows Memoru to operate outside the regulatory constraints of a university setting, which is crucial for working with federally regulated substances like psilocybin.
This transition represents a significant advancement for the field of psychedelic therapy. By operating independently, Memoru has greater freedom to pursue clinical research and provide treatment using promising substances like psilocybin, which are currently restricted at the university level due to federal regulations. This move allows for more rapid translation of research into practice, potentially leading to faster development and wider availability of effective treatments for [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions. Furthermore, the focus on integrative medicine offers a holistic approach to care that could address unmet needs in mental health services.
Memoru inherits Naropa’s existing psilocybin training certifications in Colorado and is working to transfer its Oregon certification. It will continue existing training programs, with plans to expand online offerings. The center will focus on three core areas: clinical care using integrative medicine and psychedelic-assisted therapies; training facilitators for these therapies; and research to develop innovative care models. This structure allows for a synergistic approach, where research findings directly inform clinical practice and training programs evolve with the latest advancements.
The launch of Memoru signals a maturing field of psychedelic therapy, moving from academic exploration to real-world application. This shift could accelerate the development and acceptance of psychedelic-assisted treatments within mainstream healthcare. Memoru’s emphasis on accessibility and community outreach also suggests a commitment to bringing these potentially transformative therapies to a broader population, particularly those underserved by traditional mental health services. This model may pave the way for other similar centers to emerge, further expanding access to and accelerating the development of psychedelic therapies.
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**Categories:** News
---
### [Strategic Navigation for Clinical Trials in Mid-Size Biopharma: Insights from Almirall](https://www.clinicaltrialvanguard.com/conference-coverage/strategic-navigation-for-clinical-trials-in-mid-size-biopharma-insights-from-almirall/)
**Published:** December 17, 2024
**Author:** Moe Alsumidaie
**Content:**
At [SCOPE Summit Europe](https://www.scopesummiteurope.com/ "SCOPE Summit Europe"), Estrella García, PhD, an Executive Director at Almirall, shared her insights on navigating the complexities of clinical trials in mid-size biopharma companies. Her presentation, “Strategic Navigation for Clinical Trials in Mid-Size Biopharma,” offered a deep dive into the intricacies of study design, regulatory challenges, vendor management, and effective teamwork. García’s session provided a roadmap for biopharma companies looking to optimize their clinical trial processes and achieve successful outcomes.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#study-design-and-development)Study Design and Development
Estrella García underscored the necessity of involving diverse stakeholders in the study design process to ensure a comprehensive and effective framework. She explained that collaboration with key opinion leaders, investigators, study coordinators, and CROs is vital for leveraging their unique perspectives and expertise. This collaborative approach can significantly enhance the quality and relevance of the study design, leading to more robust and scientifically sound trials. García also highlighted the importance of integrating patient voices into the study design. By involving patient experts and organizations, companies can co-create study designs that are both scientifically rigorous and patient-centric. This approach addresses real-world unmet needs and aligns with patient expectations, leading to higher patient engagement and more meaningful outcomes. García’s emphasis on patient involvement reflects a growing trend in the industry to prioritize patient-centric approaches in clinical research.
## [](#regulatory-and-operational-challenges)Regulatory and Operational Challenges
Navigating the regulatory landscape is a complex task that García addressed with clarity. She discussed the perspectives of ethics committees and IRBs, emphasizing the importance of adhering to ICH guidelines to ensure compliance and avoid potential delays. Understanding these regulatory requirements is crucial for the smooth progression of clinical trials. García also acknowledged the operational challenges that arise from the competitive nature of clinical studies. She noted the pressure to achieve realistic recruitment rates and shared insights into Almirall’s full outsourcing strategy. This strategy involves defining a precise scope for the study and allowing CROs sufficient time to develop effective strategies. García advised against setting unrealistic expectations and stressed the importance of maintaining open communication with CROs throughout the RFP process. Her insights provide a valuable framework for biopharma companies to navigate clinical trial regulatory and operational complexities.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#vendor-selection-and-management)Vendor Selection and Management
García provided a detailed framework for evaluating potential partners in her discussion on vendor selection and management. She emphasized the importance of assessing a vendor’s capability, including their previous experience in the therapeutic area, global presence, and the expertise of the team assigned to the trial. These factors are critical in ensuring that the vendor can meet the study’s specific needs and contribute to its success. Compatibility was another key consideration highlighted by García. She advised companies to evaluate potential vendors’ size, legal requirements, and SOPs to ensure alignment with the company’s processes. This alignment is crucial for smooth collaboration and effective trial execution. Cost management was also a significant focus of García’s presentation. She recommended using a standardized budget grid to facilitate proper comparison of vendor proposals and ensure cost-effectiveness. By linking costs to deliverables and identifying potential savings through synergies, companies can maintain financial transparency and accountability, ultimately leading to more efficient and successful trials.
## [](#objectives-and-performance-monitoring)Objectives and Performance Monitoring
García outlined the strategic objectives of clinical trials, emphasizing the need for business visibility and strategic alignment. She explained that defining clear targets and KPIs is essential for monitoring performance and ensuring the trial aligns with broader business goals. Staying informed about market trends and competition is crucial for adapting strategies and maintaining a competitive edge. Continuous improvement initiatives were another area of focus in García’s presentation. She advocated for regular financial reviews and performance monitoring through pre-agreed KPIs. These reviews can help identify potential risks and areas for improvement, enabling companies to make informed decisions and enhance trial outcomes. Biopharma companies can optimize their clinical trial processes and achieve successful outcomes by fostering continuous improvement and strategic alignment.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#effective-teamwork-and-communication)Effective Teamwork and Communication
In her concluding remarks, García drew an analogy between successful clinical trial management and a well-coordinated sports team. She emphasized the importance of communication, commitment, respect, and empathy within teams. Companies can create a positive and collaborative work environment by fostering a culture that avoids micromanagement and encourages team members to take ownership of their roles. García stressed that effective teamwork involves building on each other’s strengths, anticipating challenges, and finding solutions collectively. While acknowledging that things can and will go wrong, she emphasized the importance of focusing on problem-solving as a team rather than assigning blame. This approach fosters a positive and collaborative work environment, ultimately leading to more successful trial outcomes. García’s insights provide a valuable framework for biopharma companies to optimize their clinical trial processes and achieve successful outcomes through effective teamwork and communication.
## [](#summary)Summary
In conclusion, Estrella García’s presentation at the [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) conference offered a comprehensive guide for mid-size biopharma companies navigating the complexities of clinical trials. Her insights into study design, regulatory challenges, vendor management, and teamwork provide a valuable framework for optimizing clinical trial processes and achieving successful outcomes. By prioritizing collaboration, patient-centric approaches, and strategic alignment, biopharma companies can enhance their clinical trial processes and contribute to the advancement of medical research.
**Categories:** Article: Conference Coverage
---
### [Trodelvy Gets Breakthrough Therapy for Lung Cancer](https://www.clinicaltrialvanguard.com/news/trodelvy-gets-breakthrough-therapy-for-lung-cancer/)
**Published:** December 18, 2024
**Author:** Jon Napitupulu
**Content:**
The FDA has granted Breakthrough Therapy Designation to Gilead Sciences’ Trodelvy for treating extensive-stage [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/)[lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) (ES-SCLC) in adults who have progressed on or after platinum-based chemotherapy. This designation stems from the positive results of the Phase 2 TROPiCS-03 study, where Trodelvy showed encouraging antitumor activity and a manageable safety profile in both platinum-resistant and platinum-sensitive ES-SCLC. This development addresses the critical need for new therapies for ES-SCLC, a disease with limited treatment options and a generally poor prognosis after failure of first-line therapy.
This Breakthrough Therapy Designation is particularly important because it acknowledges the potential of Trodelvy to significantly improve outcomes for ES-SCLC patients, a population with a high unmet medical need. Current second-line treatments often offer limited benefit, highlighting the urgency for more effective options. The positive data from the TROPiCS-03 study suggests that Trodelvy could offer a new avenue for extending survival and improving quality of life for these patients. The potential for a new, effective treatment also signifies hope for patients and their families facing this aggressive form of lung cancer.
The Phase 2 TROPiCS-03 study demonstrated Trodelvy’s promising antitumor activity in platinum-resistant and-temperature ES-SCLC. The safety profile observed in the study was consistent with previous Trodelvy studies. This is the second Breakthrough Therapy Designation awarded to Trodelvy, the first being for its use in certain [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) types. The FDA’s decision underscores the drug’s potential to address unmet needs across multiple cancers. Based on the promising Phase 2 data, Gilead intends to initiate a Phase 3 clinical trial to evaluate Trodelvy in the ES-SCLC population further. This trial will be crucial for confirming the efficacy and safety observed in the earlier phase and for gathering the necessary data for a potential regulatory submission.
This Breakthrough Therapy Designation signifies a potentially substantial advancement in the treatment landscape for ES-SCLC. The positive clinical data, coupled with the FDA’s recognition of Trodelvy’s potential, suggests a promising future for this drug in addressing the unmet needs of this patient population. The upcoming Phase 3 trial will be a critical step in confirming its efficacy and paving the way for potential regulatory approval, which could bring a new and much-needed treatment option to patients with ES-SCLC. Furthermore, this achievement validates the broader research and development efforts focused on Trop-2 targeted therapies, highlighting the potential of this approach in treating various cancers. The progress of Trodelvy in ES-SCLC will be closely watched by both the medical community and patients, with the hope that it will translate into a meaningful improvement in patient outcomes.
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**Categories:** News
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### [Silexion's Precision Oncology Collaboration: Targeting KRAS](https://www.clinicaltrialvanguard.com/news/silexions-precision-oncology-collaboration-targeting-kras/)
**Published:** December 19, 2024
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) is developing [SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/), a next-generation siRNA therapy targeting a broad range of KRAS mutations prevalent in difficult-to-treat cancers like pancreatic, colorectal, and lung cancers. The company’s collaboration with Evonik has yielded a promising long-acting PLGA microparticle formulation for SIL-204, showing efficacy in preclinical studies by shrinking tumors in KRAS-mutated [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) models. This RNAi approach offers a novel mechanism of action, silencing KRAS mutations at the genetic level, potentially overcoming limitations of existing small-molecule inhibitors.
This development is particularly noteworthy due to the persistent challenge KRAS mutations pose in oncology. Current small-molecule inhibitors only address specific, less common KRAS mutations, leaving a significant unmet need for therapies targeting the broader G12x and G13D mutations. Silexion’s approach may offer a more comprehensive solution, potentially impacting a larger patient population across multiple cancer types. The timing aligns with increased industry interest in innovative cancer therapies, as evidenced by recent high-value acquisitions in the oncology space.
Silexion’s preclinical data demonstrates the potential of the sustained-release PLGA formulation to improve efficacy and targeting. SIL-204’s ability to target a wider range of KRAS mutations expands its potential market beyond pancreatic cancer to include colorectal and lung cancers, both with significant unmet need and large patient populations. The growing KRAS inhibitor market, projected to reach $10 billion by 2032, further emphasizes the commercial potential of this advancement.
This progress positions Silexion as a compelling player in the precision oncology landscape. The company’s innovative approach, combined with the growing market for targeted cancer therapies, creates significant opportunities. While clinical development carries inherent risks, Silexion’s strategic partnership and preclinical success suggest a promising path forward, potentially leading to a new treatment paradigm for KRAS-driven cancers.
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**Categories:** News
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### [eClinical Solutions and Snowflake Partner for Life Sciences](https://www.clinicaltrialvanguard.com/news/eclinical-solutions-and-snowflake-partner-for-life-sciences/)
**Published:** December 19, 2024
**Author:** Jon Napitupulu
**Content:**
eClinical Solutions and Snowflake have partnered to integrate eClinical’s elluminate Clinical Data Cloud with the Snowflake AI Data Cloud. This integration aims to address the growing data challenges faced by [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) organizations by providing a modern, efficient, and scalable data architecture. The collaboration seeks to streamline data access and empower the development of next-generation applications using AI and machine learning.
This partnership is crucial for the life sciences industry because it directly addresses the increasing data complexity arising from personalized medicine, digital trials, and decentralized research. The sheer volume of data generated by these advancements makes extracting meaningful insights challenging. This integrated platform offers a solution by centralizing data, automating processes, and ultimately enabling faster, more informed decision-making. This efficiency is particularly important given the industry’s ongoing pressure to accelerate clinical trials while managing resources effectively. Furthermore, the enhanced data accessibility and interoperability facilitated by this integration will support the development of advanced analytics and AI-driven applications, driving innovation in drug discovery and development.
Technically, the bidirectional integration allows seamless data flow between the elluminate platform and the Snowflake AI Data Cloud. Elluminate, with its built-in clinical expertise and domain knowledge, offers a purpose-built environment for managing clinical data in a regulated setting. Snowflake’s AI Data Cloud provides the scalable computational power and advanced analytics capabilities required for complex data analysis and AI/ML model development. This combined platform enables researchers to readily access and analyze data from multiple sources, facilitating collaboration and automating previously manual processes.
This collaboration positions both eClinical Solutions and Snowflake at the forefront of a critical shift in the life sciences industry. By providing a robust and integrated data platform, they empower researchers to leverage the full potential of their data. This will likely accelerate the development of new therapies and ultimately improve patient outcomes by enabling data-driven decision-making and fostering innovation in clinical research. The enhanced data management capabilities offered by this platform are poised to become essential infrastructure for the future of drug development, particularly as the industry continues to embrace data-intensive approaches like personalized medicine and AI-driven drug discovery.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Protein Helps Cancer Evade CAR T-Cell Therapy](https://www.clinicaltrialvanguard.com/news/protein-helps-cancer-evade-car-t-cell-therapy/)
**Published:** December 19, 2024
**Author:** Jon Napitupulu
**Content:**
Scientists at City of Hope have identified a protein, YTHDF2, that helps blood cancer cells evade CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), a treatment that uses the immune system to attack tumors. They also developed a compound, CCI-38, which targets and suppresses YTHDF2, thereby reducing cancer growth and improving treatment outcomes. This discovery stems from research published in the journal •Cell•.
This breakthrough is crucial for advancing blood cancer treatment, particularly for patients who relapse after CAR T cell therapy or whose cancers develop resistance. Current CAR T therapies often face challenges due to “antigen escape,” where cancer cells reduce or lose the CD19 protein targeted by the therapy. This new research tackles this issue by targeting a different mechanism – YTHDF2 – which not only contributes to cancer growth and spread but also helps cancer cells hide from the immune system. Addressing this previously untargeted protein offers a new avenue for improving treatment effectiveness and potentially overcoming resistance mechanisms.
The research details how YTHDF2 promotes cancer cell growth by enabling stable energy production and by reducing the presence of antigens that trigger immune responses. The newly developed compound, CCI-38, effectively suppresses YTHDF2, suggesting a potential combination therapy approach with CAR T cell treatment. This could lead to fewer relapses, reduced need for follow-up treatments, and ultimately, improved long-term survival rates for blood cancer patients.
This discovery opens exciting possibilities for blood cancer treatment. Targeting YTHDF2 with CCI-38, potentially in combination with CAR T cell therapy, could significantly enhance treatment efficacy and overcome current limitations like antigen escape. Further research and clinical trials will be essential to validate these findings and optimize the use of CCI-38, paving the way for more effective and personalized therapies for blood cancers and potentially other cancers and autoimmune diseases.
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**Categories:** News
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### [Colorado's Life Sciences Raised $2.15B in 2024](https://www.clinicaltrialvanguard.com/news/colorados-life-sciences-raised-2-15b-in-2024/)
**Published:** December 20, 2024
**Author:** Jon Napitupulu
**Content:**
Colorado’s [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) ecosystem flourished in 2024, raising $2.15 billion, a 46% increase from 2023, marking the second time in four years funding has surpassed $2 billion. This impressive sum adds to the nearly $12 billion raised over the past eight years, solidifying Colorado’s position as a leading hub for life sciences innovation. The surge in funding underscores the state’s attractive environment for life sciences companies, fostered by a collaborative community, highly skilled workforce, strategic location, robust infrastructure, manageable costs, and a high quality of life.
This financial influx is crucial for the continued growth and maturation of Colorado’s life sciences sector. It enables companies to advance research and development, translate scientific discoveries into tangible therapies and diagnostics, and ultimately bring innovative solutions to patients faster. The substantial investment validates the state’s commitment to fostering a thriving life sciences ecosystem, creating a virtuous cycle that attracts further investment and talent. This sustained growth translates into a more robust pipeline of promising technologies with the potential to revolutionize healthcare and address unmet medical needs.
The $2.15 billion raised in 2024 represents a diverse range of funding sources, including $818.5 million in public capital (a 64% increase from 2023), $445 million through mergers, acquisitions, and partnerships, and $383 million in private capital, primarily venture funding. Federal grants, vital for research and development at both private companies and academic institutions, totaled $496.6 million. State grants, specifically the Advanced Industries Accelerator Grants, contributed an additional $5.5 million. This multifaceted funding landscape demonstrates the strong support for life sciences innovation from public, private, and governmental sources.
This continued financial momentum positions Colorado’s life sciences sector for sustained growth and impact. The influx of capital will likely accelerate the development and commercialization of innovative healthcare solutions, ultimately benefiting patients and further solidifying Colorado’s reputation as a national leader in the life sciences. The robust funding environment also strengthens the state’s economic base, creating high-paying jobs and generating substantial economic impact, contributing to a positive feedback loop that attracts further investment and talent. The success in 2024 sets a high bar and suggests a bright future for Colorado’s life sciences ecosystem.
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**Categories:** News
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### [Tolerance Bio, Inc. Launches $20.2M Seed Round for Thymus-Based Therapies](https://www.clinicaltrialvanguard.com/news/tolerance-bio-inc-launches-20-2m-seed-round-for-thymus-based-therapies/)
**Published:** December 20, 2024
**Author:** Jon Napitupulu
**Content:**
Tolerance Bio, Inc., a biopharmaceutical company, has formed a Scientific Advisory Board and completed a $20.2 million seed financing round. The company is developing an allogeneic iPSC-based thymus [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) platform and pharmacological thymus therapies to address immune-mediated diseases like cancer, autoimmunity, and transplant rejection. The oversubscribed funding round, including Pacific 8 Ventures, will support these research and development efforts.
This development is crucial for the advancement of immune disease treatment. Tolerance Bio’s approach, focusing on the thymus, offers a novel pathway to address the root cause of many immune-mediated diseases rather than merely managing symptoms. Successfully manipulating the thymus, the master regulator of immune tolerance, could lead to groundbreaking treatments for a wide range of debilitating conditions, potentially offering long-term solutions and improved patient outcomes. The formation of a robust Scientific Advisory Board, comprising leading experts in immunology, oncology, cell therapy, and drug development, further strengthens Tolerance Bio’s potential to translate its research into effective therapies.
The $20.2 million secured in the seed round demonstrates significant investor confidence in Tolerance Bio’s innovative approach. This funding enables the company to accelerate the development of its allogeneic iPSC-based thymus cell therapy platform and pharmacological therapies, moving closer to clinical trials and potentially revolutionizing the treatment landscape for immune-mediated diseases. The assembled Scientific Advisory Board provides critical expertise, guiding research strategies and ensuring scientific rigor throughout the development process. The diverse backgrounds of the board members, ranging from stem cell research and transplant immunology to clinical development and biologic therapeutics, positions Tolerance Bio to tackle complex immunological challenges from multiple angles.
The formation of this Scientific Advisory Board and securing substantial seed funding marks a crucial step forward for Tolerance Bio. It lays a strong foundation for translating their innovative research into potentially life-changing therapies for patients suffering from immune-mediated diseases. This progress also signifies a broader shift in the field towards targeting the thymus as a key player in immune regulation, potentially opening new avenues for treating a spectrum of diseases. The collaborative efforts of the scientific team, coupled with the financial backing, pave the way for significant advancements in the fight against immune disorders. This concentrated effort towards thymus-focused therapies holds the potential to reshape the future of immunology and transform the lives of countless individuals. The advancement of this technology promises to bring renewed hope and improved outcomes for patients worldwide, ushering in a new era of targeted immune modulation.
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**Categories:** News
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### [Tiximed Secures $2.65M Investment for Novel Oral Therapy Trials](https://www.clinicaltrialvanguard.com/news/tiximed-secures-2-65m-investment-for-novel-oral-therapy-trials/)
**Published:** December 20, 2024
**Author:** Jon Napitupulu
**Content:**
TIXiMED, a clinical-stage pharmaceutical company, has secured a $2.65 million loan from the Helmsley Charitable Trust to advance its oral drug candidate, TIX100, into a first-in-human clinical trial starting in January. This funding will support the single ascending dose (SAD) study, a crucial step in evaluating the safety and dosage of TIX100 in humans. The drug targets thioredoxin-interacting protein (TXNIP), a protein linked to beta-cell death and dysfunction in diabetes.
This development is potentially groundbreaking for the field of diabetes treatment, particularly [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/) (T1D). Current T1D treatments focus primarily on managing blood sugar levels through insulin therapy, but they don’t address the underlying cause of the disease – the destruction of insulin-producing beta cells. TIX100 offers a novel approach by targeting TXNIP, aiming to protect and preserve beta-cell function. This could potentially slow or even halt the progression of T1D, representing a significant shift from the current standard of care and offering a new hope for patients. Furthermore, the oral administration of TIX100 enhances patient convenience compared to existing injectable therapies, potentially improving adherence and long-term disease management.
The non-dilutive nature of the funding from the Helmsley Charitable Trust is strategically advantageous for TIXiMED. It allows the company to advance TIX100’s clinical development without giving up equity, preserving its financial resources for future stages of research and commercialization. The SAD study is a critical step in determining the safety profile and pharmacokinetics of TIX100 in humans, paving the way for subsequent larger-scale clinical trials to assess its efficacy in treating T1D. Pre-clinical studies have shown promising results in protecting against diabetes and metabolic dysfunction, further supporting the potential of this novel therapeutic approach.
The commencement of the first-in-human trial for TIX100 marks a significant milestone in the development of a potential disease-modifying therapy for T1D. The successful completion of this SAD study will be a crucial step towards validating the TXNIP inhibition strategy and could potentially lead to a new paradigm in diabetes care, offering patients a chance to preserve their beta-cell function and improve their long-term health outcomes. This novel approach has the potential to transform the treatment landscape for T1D and other metabolic diseases.
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**Categories:** News
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### [SCOPE 2025 Participant Engagement Awards Finalists Announced](https://www.clinicaltrialvanguard.com/news/scope-2025-participant-engagement-awards-finalists-announced/)
**Published:** December 21, 2024
**Author:** Moe Alsumidaie
**Content:**
The [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Summit has revealed the six finalists for its highly anticipated 2025 Participant Engagement Awards, celebrating excellence in innovation and impact within the clinical trial industry. This year’s announcement follows a competitive selection process that saw over 40 submissions, highlighting the evolving landscape of participant-centric initiatives.
The winners will be unveiled during the Monday afternoon keynote session at the SCOPE Summit, an event renowned for driving forward-thinking dialogue in clinical trials. For the full list of finalists and judges, visit [Participant Engagement Award Finalists](https://www.scopesummit.com/participant-engagement-award).
While the detailed list of finalists has not yet been made public, the judging panel—comprising experts in clinical research and patient engagement—evaluated each submission for its innovation, scalability, and measurable outcomes in fostering meaningful participant involvement.
As decentralized clinical trials (DCTs) and patient-centric approaches gain traction, these awards underscore the critical role of engagement strategies in improving clinical trial participation, retention, and diversity. With over 40 entries vying for the top spots, the finalists represent the best in addressing barriers to clinical trial access and enhancing participant experiences.
The winners will be celebrated at the SCOPE Summit keynote, setting the stage for broader industry discussions on how participant-focused innovations can redefine clinical research. Check out the full keynote agenda at [SCOPE Keynotes](https://www.scopesummit.com/keynotes).
**Categories:** News
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### [Ellipses Launches Pioneering Clinical Trial in Middle East](https://www.clinicaltrialvanguard.com/news/ellipses-launches-pioneering-clinical-trial-in-middle-east/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
Ellipses Pharma has launched its first large-scale Phase 1/2 oncology clinical trial in the United Arab Emirates (UAE) for its selective RET inhibitor (SRI), EP0031/A400. This trial, focusing on RET-altered tumors prevalent in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) and thyroid cancer, is supported by Abu Dhabi investors and will initially be conducted at the Cleveland Clinic and Tawam Hospital in Abu Dhabi. The trial initiation follows the FDA’s granting of Orphan Drug and Fast Track Designations to EP0031/A400, highlighting its potential to address unmet medical needs.
This trial signifies a major advancement for cancer treatment accessibility in the UAE. It offers UAE patients access to a potentially groundbreaking therapy while fostering collaboration and research sharing among healthcare professionals in the region. The trial’s success could pave the way for more international clinical research collaborations and attract further investment in the UAE’s healthcare sector. This strengthens the UAE’s position as a hub for medical innovation and potentially improves healthcare outcomes for the regional population.
EP0031/A400 is being jointly developed by Ellipses globally and Kelun Biotech in China. The drug has already undergone Phase 1 studies in the US, Europe, and China, and registrational Phase 2 trials are now underway in those regions as well as the UAE. Ellipses holds exclusive licensing rights for EP0031/A400 in the US and Europe, while [Kelun-Biotech](https://www.clinicaltrialvanguard.com/news/kelun-biotechs-sac-tmt-plus-pembrolizumab-hits-primary-endpoint-in-pd-l1-negative-nsclc/) retains rights in Greater China and certain Asian countries. Regulatory approvals for the drug have been secured in both the US and China. RET alterations are significant drivers in various cancers, especially non-small cell lung and thyroid cancer. Next-generation SRIs like EP0031/A400 are crucial in addressing acquired resistance to first-generation treatments, which underscores the trial’s potential impact.
This UAE trial initiation marks a critical step in the development of EP0031/A400. Positive results could lead to accelerated regulatory approvals and broader market access, solidifying Ellipses’ position in the oncology field. It also sets a precedent for international collaboration in drug development, potentially expediting the delivery of innovative cancer treatments to patients globally. The trial’s outcome will be crucial in shaping the future of RET-targeted therapies and the landscape of cancer care.
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**Categories:** News
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### [Mirailab Launches NMN Supplements in Singapore](https://www.clinicaltrialvanguard.com/news/mirailab-launches-nmn-supplements-in-singapore/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
MIRAILAB BIOSCIENCE Inc., a Tokyo-based company specializing in NMN supplements, has partnered with Global Ocean Distribution Pte Ltd to launch its “MIRAI LAB” brand on Shopee, a major Singaporean e-commerce platform. This expansion marks a significant step in making their β-Nicotinamide [Mononucleotide](https://www.clinicaltrialvanguard.com/news/mirailab-launches-nmn-supplements-and-cosmetics-in-uae/) (β-NMN) supplements more accessible to consumers in Singapore. The company highlights NMN’s potential anti-aging and rejuvenation benefits, driven by its interaction with sirtuins, longevity-related genes.
This launch is important for the burgeoning NMN market because it broadens access to a supplement with increasing scientific interest in its potential health benefits. Specifically, the availability of high-quality NMN products through a trusted platform like Shopee can help address consumer concerns about product authenticity and quality, which are paramount in the supplement industry. This strategic move could also stimulate further research and development into NMN’s therapeutic applications, benefiting both the scientific community and consumers seeking evidence-based health solutions.
MIRAILAB BIOSCIENCE emphasizes its “Pure NMN Series,” containing high concentrations of β-NMN with a claimed purity of 99.7%. This high purity is a crucial selling point, particularly given the company’s assertion that its NMN has been used in U.S. Department of Defense-funded clinical trials. The company’s presence in markets like Japan, China, the United States, and the UAE suggests an established brand reputation and potential for significant growth within the Singaporean market. Leveraging Shopee’s established e-commerce infrastructure provides a direct-to-consumer approach, potentially bypassing traditional retail channels and offering competitive pricing through platform-specific promotions.
This expansion into Singapore through Shopee signifies a strategic move to capitalize on growing consumer interest in health and wellness, specifically within the anti-aging supplement segment. By offering a product with a purported high purity and a history of research involvement, MIRAILAB BIOSCIENCE is positioning itself to capture a significant share of this growing market. This strategic initiative broadens the company’s reach and reinforces the increasing importance of e-commerce platforms in distributing health supplements directly to consumers. The market response in Singapore will be a key indicator of consumer acceptance and could influence future expansion strategies for MIRAILAB BIOSCIENCE and other companies operating in the NMN supplement space.
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### [Oncodesign Reacquires Rights to OPM-201 After Positive Phase 1 Trial](https://www.clinicaltrialvanguard.com/news/oncodesign-reacquires-rights-to-opm-201-after-positive-phase-1-trial/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
[Oncodesign](https://www.clinicaltrialvanguard.com/news/oncodesigns-precision-medicine-a-new-era-in-systemic-radiotherapy/) Precision Medicine (OPM) has regained full rights to its [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease treatment program, OPM-201, after Servier Laboratories shifted its neurological focus to rare diseases. A Phase 1 study on healthy volunteers recently concluded, confirming OPM-201’s safety, with final results anticipated in Q2 2025. OPM plans to continue developing the program internally until a new partner for later-stage clinical trials is secured.
This development is crucial because it allows OPM to retain control over a promising asset targeting a significant unmet medical need. Parkinson’s disease affects a substantial and growing patient population lacking disease-modifying therapies. OPM-201, an LRRK2 kinase inhibitor, offers the potential to alter the disease’s progression, rather than just managing symptoms. This positions OPM to attract partners specifically interested in neurodegenerative diseases and strengthens its pipeline beyond its current focus on oncology and immuno-inflammatory diseases.
The OPM-201 program has a history of successful collaborations. Initially developed with Ipsen, the program progressed to the “advanced lead” stage before returning to Oncodesign due to Ipsen’s strategic shift. Subsequently, Servier partnered on the project, leading to the identification of a drug candidate in 2022 and the completion of preclinical and chemistry, manufacturing, and control (CMC) development. The recent Phase 1 trial demonstrated good tolerability and promising target engagement. This positive data, along with the “best-in-class” potential of OPM-201, enhances its attractiveness to potential partners.
This reacquisition positions OPM to maximize the value of OPM-201. By leading the program’s development through the next stages, OPM can generate further compelling data and attract partnerships with companies focused on neurodegenerative diseases. This strategic move diversifies OPM’s pipeline, strengthens its position in a growing therapeutic area, and allows it to control the development timeline for a potential breakthrough Parkinson’s disease treatment. The pursuit of a new partnership will be critical to advance OPM-201 through the costly later-stage clinical trials and ultimately bring this promising therapy to patients.
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### [JCR's JR-142 Phase III Trial Launched in Japan](https://www.clinicaltrialvanguard.com/news/jcrs-jr-142-phase-iii-trial-launched-in-japan/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
[JCR Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/jcr-pharmaceuticals-innovative-gene-therapy-research-unveiled-at-lysosomal-forum/) has initiated a Phase III clinical trial in Japan for JR-142 (redalsomatropin alfa), a long-acting growth hormone therapy for pediatric growth hormone deficiency. This trial marks a crucial step in developing a more convenient treatment option for young patients. The study will evaluate 54 children over a year, comparing JR-142’s efficacy against JCR’s existing daily injection, Growject®, with growth outcomes as the primary endpoint.
This trial is significant for the growth hormone therapy landscape because it addresses a major patient compliance challenge. Daily injections can be burdensome for children and their families, often impacting adherence to treatment regimens. A successful once-weekly therapy like JR-142 could dramatically improve treatment adherence, potentially leading to better patient outcomes and improved quality of life. Furthermore, this shift could streamline treatment administration, reducing the overall healthcare resource burden associated with daily injections. This could have a positive impact on healthcare systems and allow healthcare providers to focus on other aspects of patient care.
The Phase III trial directly compares JR-142 to Growject®, providing valuable head-to-head data on efficacy and safety. This information will be critical for healthcare providers when making treatment decisions. The trial’s focus on growth outcomes as the primary success measure underscores the commitment to demonstrating a tangible benefit for patients. The 52-week duration is long enough to capture meaningful changes in growth patterns.
The outcome of this Phase III trial will significantly shape the future of pediatric growth hormone deficiency treatment. Positive results could lead to a paradigm shift in how this condition is managed, offering a more convenient and potentially more effective treatment option for children. This could solidify JCR’s position in the growth hormone market, not only in Japan but potentially globally. Furthermore, the development of JR-142 could spur further innovation in long-acting drug delivery systems for other chronic pediatric conditions. This would significantly enhance patient care and treatment compliance across a broader range of therapeutic areas.
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### [FDA Approves Braftovi for BRAF V600E Metastatic Colorectal Cancer](https://www.clinicaltrialvanguard.com/news/fda-approves-braftovi-for-braf-v600e-metastatic-colorectal-cancer/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
The FDA granted accelerated approval to Pfizer’s BRAFTOVI (encorafenib) in combination with [cetuximab](https://www.clinicaltrialvanguard.com/news/frontier-medicines-presents-preclinical-data-on-3-programs/) and mFOLFOX6 for treating metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC) with a BRAF V600E mutation. This approval, based on positive results from the Phase 3 BREAKWATER trial, offers a new first-line treatment option for these patients, showing significant improvement in response rate and durability compared to standard chemotherapy regimens. The accelerated approval falls under the FDA’s Project FrontRunner initiative, aimed at expediting the development and approval of cancer drugs.
This approval is a significant advancement for mCRC patients with the BRAF V600E mutation, who historically have faced limited treatment options and poor prognoses. BRAF mutations are present in 8-10% of mCRC patients and are associated with a more aggressive disease course and significantly higher mortality risk. Until now, no targeted therapies were specifically approved for this patient subgroup in the first-line setting, leaving a critical unmet need. The availability of a BRAF-targeted combination therapy offers renewed hope for improved outcomes and disease control in this challenging patient population.
The BREAKWATER trial demonstrated a confirmed overall response rate of 61% for the BRAFTOVI combination, compared to 40% for standard chemotherapy regimens (with or without [bevacizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-lytenava-first-ophthalmic-bevacizumab-for-wet-amd/)). The median duration of response was also longer in the BRAFTOVI arm (13.9 months vs. 11.1 months). While the trial is ongoing, these initial results are compelling and suggest a potential shift in the treatment landscape for BRAF V600E-mutant mCRC. The safety profile of the BRAFTOVI combination was consistent with the known profiles of the individual agents, with no new safety signals identified. Common adverse reactions included peripheral neuropathy, nausea, fatigue, rash, and diarrhea.
This approval marks a crucial step forward in addressing the unmet needs of BRAF V600E-mutant mCRC patients. It provides a much-needed targeted therapy option in the first-line setting, potentially leading to better response rates and longer periods of disease control. The ongoing BREAKWATER trial will provide further data on the long-term efficacy and safety of this combination. This approval, along with ongoing research and development efforts, signifies a positive trajectory towards improved treatment strategies and outcomes for this patient population. It reinforces the importance of biomarker-driven therapies and underscores the potential for continued innovation in the field of targeted cancer treatments.
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### [FDA Approves Trikafta for Additional Cystic Fibrosis Variants](https://www.clinicaltrialvanguard.com/news/fda-approves-trikafta-for-additional-cystic-fibrosis-variants/)
**Published:** December 23, 2024
**Author:** Jon Napitupulu
**Content:**
The FDA has expanded the approval of Vertex Pharmaceuticals’ TRIKAFTA, a [cystic fibrosis](https://www.clinicaltrialvanguard.com/news/infexs-resp-x-shows-exacerbation-reduction-in-bronchiectasis-study/) (CF) treatment, to include individuals aged two and older with at least one F508del mutation or a responsive mutation in the CFTR gene. This expansion adds 94 non-F508del CFTR mutations to the approved list and makes approximately 300 more people in the U.S. eligible for this treatment. A boxed warning about the risk of liver injury and liver failure has also been added to the drug’s label.
This approval is a notable advancement for the CF community. It offers a first-time treatment option for a subset of patients who previously lacked access to therapies addressing the underlying cause of their disease. Expanding access to CFTR modulators like TRIKAFTA holds the potential to improve lung function, reduce the frequency of pulmonary exacerbations, and ultimately enhance the quality of life for these individuals. Earlier intervention with effective treatments may also alter the long-term trajectory of the disease and improve overall prognosis.
The expansion of TRIKAFTA’s label to include additional CFTR mutations significantly broadens the patient population eligible for this therapy. The inclusion of a boxed warning regarding potential liver injury underscores the importance of careful monitoring of liver function tests in patients taking TRIKAFTA. This allows for early detection and management of potential liver-related complications.
This expanded approval marks another step towards personalized medicine in CF. As research continues and further mutations are characterized, the potential exists for even more individuals with CF to receive targeted treatments that address the specific genetic defects driving their disease. This progress offers hope for a future where CF therapies are tailored to individual patient needs, maximizing their effectiveness and improving outcomes for a wider spectrum of people living with this challenging condition.
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### [Exicure & GPCR Therapeutics Partner to Fuel Biotech Growth](https://www.clinicaltrialvanguard.com/news/exicure-gpcr-therapeutics-partner-to-fuel-biotech-growth/)
**Published:** December 27, 2024
**Author:** Jon Napitupulu
**Content:**
Exicure, Inc. signed a Memorandum of Understanding with GPCR Therapeutics to acquire GPCR USA, a subsidiary specializing in G Protein-Coupled Receptor (GPCR) drug development. This acquisition includes the transfer of technology and collaborative research on GPCR Therapeutics’ ongoing drug pipelines, most notably a CXCR4 inhibitor currently in Phase 2 clinical trials for multiple [myeloma](https://www.clinicaltrialvanguard.com/news/talquetamab-plus-darzalex-shows-30-point-progression-free-survival-gain-in-multiple-myeloma/), a market estimated at $1-2 billion annually. Exicure also gains access to GPCR Therapeutics’ research in immuno-oncology, fibrosis, and obesity treatments.
This acquisition is a pivotal moment for Exicure, marking a strategic shift from an early-stage nucleic acid therapy company to a clinical-stage biotech entity. The acquisition of a late-stage clinical asset, combined with experienced research personnel like Dr. Pina Cardarelli, formerly of Bristol-Myers Squibb, positions Exicure for rapid growth and potential market entry. The focus on GPCRs, representing a significant portion of drug targets, allows Exicure to enter a diverse therapeutic landscape. This diversification offers a significant upside compared to its previous focus on nucleic acid therapies.
Exicure gains access to a Phase 2 CXCR4 inhibitor, a promising drug candidate targeting multiple myeloma. The company also benefits from GPCR Therapeutics’ intellectual property portfolio, including patents related to CXCR4 and other GPCRs. Financially, Exicure has secured $14 million in capital investment to support this acquisition, clinical trials, and ongoing operations. This infusion of capital underscores investor confidence in Exicure’s new strategic direction and the potential of the acquired assets.
This strategic move signifies a significant transformation for Exicure. The acquisition provides immediate entry into a late-stage clinical trial, broadens the company’s therapeutic portfolio, and offers the potential for substantial revenue generation. The combination of a promising drug candidate, experienced research personnel, and secured funding positions Exicure for growth and development as a clinical-stage biotechnology company focused on GPCR-targeted therapies. While the previous focus on nucleic acid therapies presented its own challenges and opportunities, this shift offers a more immediate and potentially lucrative pathway to market. The success of the ongoing Phase 2 trials and future collaborations will be crucial for establishing Exicure’s position in the competitive biotech landscape.
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### [Daiichi and AstraZeneca Datopotamab Deruxtecan Application Withdrawn from EU](https://www.clinicaltrialvanguard.com/news/daiichi-and-astrazeneca-datopotamab-deruxtecan-application-withdrawn-from-eu/)
**Published:** December 27, 2024
**Author:** Jon Napitupulu
**Content:**
Daiichi Sankyo and AstraZeneca have withdrawn their EU marketing authorization application for datopotamab deruxtecan (Dato-DXd) in locally advanced or metastatic non-squamous [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). This decision follows feedback from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and is based on data from the TROPION-Lung01 phase 3 trial. While the companies will continue developing datopotamab deruxtecan for lung cancer with ongoing clinical trials, this withdrawal impacts their strategy for bringing this drug to the European market.
This withdrawal represents a setback for patients with advanced NSCLC who lack effective treatment options after progressing on standard therapies like chemotherapy, immunotherapy, and targeted treatments. The potential of a TROP2-directed antibody-drug conjugate like datopotamab deruxtecan offered a new mechanism of action and a possible new therapeutic avenue, highlighting the unmet need in this patient population. The withdrawal underscores the challenges in drug development and the rigorous standards required for regulatory approval in the EU.
The withdrawal specifically affects the lung cancer application based on the TROPION-Lung01 trial, where datopotamab deruxtecan was compared to docetaxel in patients previously treated with other therapies. The trial measured progression-free survival and overall survival as primary endpoints. It’s important to note that another application for datopotamab deruxtecan, for the treatment of hormone receptor-positive, HER2-negative metastatic [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/), is still under review by the EMA. Globally, lung cancer remains a significant health concern with nearly 2.5 million diagnoses in 2022 and almost 500,000 in Europe alone.
This withdrawal likely necessitates a reassessment of the clinical development strategy for datopotamab deruxtecan in lung cancer within the EU. While the companies have expressed continued commitment to the drug’s development, the specific path forward remains unclear. They will need to address the concerns raised by the CHMP, potentially through additional clinical trials or data analyses. This situation highlights the complexities of bringing novel cancer therapies to market and the importance of robust clinical data to demonstrate clear benefit for patients.
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### [Therapeutic Blood Thinners Reduce COVID-19 Mortality](https://www.clinicaltrialvanguard.com/news/therapeutic-blood-thinners-reduce-covid-19-mortality/)
**Published:** December 27, 2024
**Author:** Jon Napitupulu
**Content:**
A new international study, analyzing 22 clinical trials and 11,000 patients across 21 countries, found that therapeutic doses of heparin can significantly improve the survival rate of hospitalized [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) patients. This World Health Organization-coordinated research, published in the •Annals of Internal Medicine•, aimed to clarify conflicting findings from previous studies on blood thinner dosages for COVID-19 and incorporated data from the HEP-COVID trial. The HEP-COVID trial found that administering therapeutic doses of heparin significantly impacted patient outcomes, particularly for those in hospital wards but not in intensive care.
This discovery is crucial for refining treatment protocols for hospitalized COVID-19 patients, particularly as the world continues to grapple with the long-term effects of the pandemic and the potential for future outbreaks. Understanding the optimal dosage of heparin for COVID-19 patients can minimize mortality and other severe outcomes like thromboembolic disease while also carefully managing the associated bleeding risks. This information is particularly relevant for non-ICU patients, who constitute a significant portion of hospitalizations, allowing for more effective resource allocation and improved patient outcomes across various care settings.
The research specifically investigated three dosage levels of heparin: a standard preventative dose, an intermediate dose, and a high therapeutic dose. Patients receiving the high therapeutic dose showed a 23% lower risk of death within 28 days compared to those on lower doses. While these results indicate the potential benefits of therapeutic heparin doses, the study also acknowledged the increased risk of major bleeding associated with higher doses, underscoring the importance of personalized treatment strategies. The study used a prospective meta-analysis design, which combined data from multiple clinical trials, ensuring a comprehensive and unbiased assessment of heparin’s effectiveness. This approach, coupled with the global scope of the included trials, lends substantial weight to the findings.
This research offers a significant advancement in understanding the role of anticoagulation in treating COVID-19 and other viral respiratory illnesses. The findings provide valuable insights for clinicians in determining appropriate heparin dosages, balancing the benefits of clot prevention with the risks of bleeding. This knowledge base not only enhances our ability to manage current and future COVID-19 cases but also informs treatment strategies for other similar respiratory illnesses, contributing to more effective pandemic preparedness and improved patient care in the long term.
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### [Axena Health Starts UI Treatment Patient Enrollment at Two Nigerian Sites](https://www.clinicaltrialvanguard.com/news/axena-health-starts-ui-treatment-patient-enrollment-at-two-nigerian-sites/)
**Published:** December 27, 2024
**Author:** Jon Napitupulu
**Content:**
[Axena Health](https://www.clinicaltrialvanguard.com/news/axena-health-and-mayo-clinic-partner-to-advance-urinary-incontinence-care/) has launched a feasibility study in Nigeria to explore a new treatment for female incontinence, combining the Leva® Pelvic Health System with a digital home-based program. The study, funded by ArcHealth Foundation and supported by Helium Health, is enrolling 60 women across two teaching hospitals in Abuja and Ikeja. This research builds upon a previous qualitative study that highlighted the substantial burden of incontinence on women in Kenya and Nigeria, revealing an urgent need for increased awareness, education, and access to treatment.
This study holds significant implications for women’s health in Nigeria and potentially other low- and middle-income countries. Incontinence carries a heavy social and economic burden, often preventing women from fully participating in work and family life. This research directly addresses this issue by investigating a novel treatment approach designed to be accessible and culturally appropriate. Positive results could lead to wider availability of effective incontinence care, significantly improving the quality of life for countless women. Furthermore, successful implementation of the combined clinic-based and digital home program could serve as a model for delivering other healthcare services in resource-constrained settings.
This study is a prospective, single-cohort, open-label trial evaluating the acceptability and effectiveness of a combined treatment approach. Participants will use the Leva System, a biofeedback device for pelvic floor muscle training (PFMT), within a clinical setting. This treatment is supplemented by a digital program providing health education and PFMT instruction for at-home use. The partnership with Helium Health ensures robust local data management, crucial for evaluating the program’s feasibility. ArcHealth Foundation’s grant funding underscores the importance of this research in addressing a global health disparity.
The results of this study are expected to shape Axena Health’s strategy for expanding access to pelvic health solutions globally. Positive findings could pave the way for broader adoption of the Leva System and digital health programs in low- and middle-income countries, addressing the significant unmet need for effective incontinence treatment. This research could ultimately contribute to a shift in how pelvic floor disorders are managed in these regions, empowering women to seek care and improve their well-being. This innovative combined approach has the potential to serve as a model for delivering other essential healthcare services in underserved communities.
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### [PainReform Delivers Phase 3 Trial Update on PRF-110](https://www.clinicaltrialvanguard.com/news/painreform-delivers-phase-3-trial-update-on-prf-110/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
[PainReform](https://www.clinicaltrialvanguard.com/news/painreform-starts-phase-ii-trial-for-ocuring-k-eye-therapy/) Ltd. (Nasdaq: PRFX), a clinical-stage pharmaceutical company specializing in reformulating existing therapies, has provided an update on its Phase 3 clinical trial for PRF-110. This trial evaluated the drug’s efficacy in managing post-surgical pain for patients undergoing bunionectomies.
While the company previously reported statistically significant pain reduction compared to a placebo during the initial 48 hours post-surgery, the final 24 hours of the 72-hour study period presented data inconsistencies. Despite efforts to clarify these discrepancies, PainReform has concluded that the data from the final 24-hour period does not meet the study’s primary endpoint requirements.
Although the primary endpoint was not met, the company is actively pursuing research and development to better understand PRF-110’s pharmaco-kinetics and pharmaco-dynamics based on the collected data. This research aims to address the identified issues and support future clinical trials. The company is using advanced in-vitro models to investigate the discrepancies observed in the final 24 hours of the 72-hour study period before proceeding with further clinical work. This research is vital to refining the drug’s profile and maximizing its potential effectiveness in future evaluations. Concurrently, PainReform is evaluating its strategic options, though there are no guarantees of specific outcomes or increased shareholder value resulting from this review.
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### [Ikena Oncology and Inmagene Merger Announcement](https://www.clinicaltrialvanguard.com/news/ikena-oncology-and-inmagene-merger-announcement/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
Ikena Oncology and Inmagene Biopharmaceuticals have entered into a definitive merger agreement. The combined company will focus on developing [IMG-007](https://www.clinicaltrialvanguard.com/news/inmagene-img-007-shows-promise-in-alopecia-areata-phase-2a-study/), Inmagene’s lead asset, a monoclonal antibody targeting OX40 for [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) treatment. The new entity plans to operate as “ImageneBio, Inc.” and trade on NASDAQ under the ticker symbol “IMA.” Concurrently with the merger, Ikena has secured a $75 million private placement financing from new investors like Deep Track Capital, Foresite Capital, and RTW Investments, as well as existing Ikena investors such as BVF Partners L.P., Blue Owl Healthcare Opportunities, Omega Funds, and OrbiMed. This financing, combined with existing resources, will provide approximately $175 million to support IMG-007’s further development.
OX40 is a costimulatory receptor found mainly on activated T cells. Anti-OX40 antibodies have shown efficacy in atopic dermatitis treatment in placebo-controlled studies. IMG-007 distinguishes itself with a longer half-life than other OX40-targeting antibodies in late-stage development, potentially enabling optimized dosing schedules. Its silenced antibody-dependent cellular cytotoxicity (ADCC) function and non-T cell depleting nature suggest a potentially improved tolerability profile compared to similar antibodies. A Phase 2b clinical trial for IMG-007 in atopic dermatitis is anticipated to commence in early 2025.
The post-merger ownership structure is projected to be approximately 34.8% for Ikena stockholders, 43.5% for Inmagene equity holders, and 21.7% for the financing investors. The combined company’s board will consist of three directors from Inmagene, two from Ikena, one representing the financing investors, and a new independent member. A search for the combined company’s CEO is underway. Contingent value rights (CVRs) will be issued to both Ikena and Inmagene shareholders for their respective legacy pipeline assets, excluding IMG-007 in the case of Inmagene.
The transaction, approved by both companies’ boards, is expected to close mid-2025, pending customary closing conditions, including shareholder approval. Support agreements have been executed by directors, officers, and certain shareholders of both companies to vote in favor of the merger.
IMG-007 is a humanized anti-OX40 IgG1 monoclonal antibody. The OX40-[OX40L](https://www.clinicaltrialvanguard.com/news/ox40-targeted-therapy-trials-market-opportunity-insight-2026/) interaction plays a crucial role in T cell activation and survival, contributing to the pathogenesis of various inflammatory and immunological diseases. Preclinical studies show IMG-007 effectively blocks OX40-OX40L signaling. Its subcutaneous formulation has demonstrated a 34.7-day half-life, suggesting potential for less frequent dosing, such as every 24 weeks in the maintenance phase of atopic dermatitis treatment. A recently completed Phase 2a trial in moderate-to-severe atopic dermatitis patients demonstrated significant, durable clinical activity and a favorable safety profile.
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**Tags:** eClinical Tech News
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### [Vor Bio Announces $55.6M Private Placement](https://www.clinicaltrialvanguard.com/news/vor-bio-announces-55-6m-private-placement/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
Vor Bio, a clinical-stage cell and genome engineering company, has secured approximately $55.6 million in gross proceeds through a private investment in public equity financing (PIPE). This financing involves issuing over 55.8 million shares of common stock and warrants to purchase nearly 70 million additional shares at a combined price of $0.99425 per share and accompanying warrant. The warrants, exercisable for seven years post-closing, carry an exercise price of $0.838 per share and could generate up to $58.5 million in additional gross proceeds if exercised in cash.
New investor Reid Hoffman spearheads the PIPE and includes participation from existing investor RA Capital Management, Vor Bio’s largest stockholder. Both Mr. Hoffman and RA Capital Management will gain a board seat and a board observer seat as part of the agreement. The investment underscores the potential of Vor Bio’s trem-cel therapy, a [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)/cas9-based treatment for acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML). This therapy edits patients’ bone marrow and shows promise in early data, offering a potential breakthrough in treating this aggressive cancer.
Stifel acted as the sole placement agent for the PIPE, which is anticipated to close on December 30, 2024, pending customary closing conditions. Vor Bio plans to allocate the net proceeds toward clinical and preclinical development of its pipeline candidates and general corporate purposes. This funding extends the company’s cash runway, allowing continued development of its promising therapies.
Vor Bio anticipates releasing updated clinical data from two key trials in 2025. Data from the Phase 1/2 VBP301 trial of VCAR33ALLO is expected in the first half of the year, followed by data from the Phase 1/2a VBP101 trial of trem-cel in combination with Mylotarg in the second half.
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### [Psyence Biomed Closes $2M Private Placement](https://www.clinicaltrialvanguard.com/news/psyence-biomed-closes-2m-private-placement/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
Psyence Biomedical Ltd., a Nasdaq-listed biotechnology company (Nasdaq: PBM), recently finalized a private placement, raising approximately $2 million before expenses. The offering involved 1,000,000 common shares (or pre-funded warrants), along with series A and short-term series B common warrants, each allowing the purchase of up to 1,000,000 common shares. The purchase price was $2.00 per common share (or pre-funded warrant) and included accompanying series A and B warrants. Both series of warrants are immediately exercisable at $2.00 per share, with the series A warrants expiring in five years and the series B warrants expiring in two years. H.C. Wainwright & Co. served as the exclusive placement agent for this offering. Psyence Biomedical plans to utilize the net proceeds for working capital and general corporate purposes.
The securities offered were part of a private placement under Section 4(a)(2) of the Securities Act of 1933 and/or Regulation D, and are not registered under the Securities Act or applicable state securities laws. Consequently, these securities cannot be offered or sold within the United States unless registered or exempt from registration. The company will file a resale registration statement covering these securities as per a registration rights agreement with investors. This press release does not constitute an offer to sell or solicit an offer to buy these securities, and no sales will occur in any jurisdiction where such actions would be unlawful before registration or qualification under applicable securities laws.
Psyence Biomedical is pioneering the development of nature-derived psilocybin-based psychedelic medicines. Focusing initially on the unmet needs of patients with [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) disorders in palliative care settings, the company emphasizes an evidence-based approach to innovation. Its name reflects a commitment to combining scientific rigor with the therapeutic potential of psychedelics. Psyence Biomedical aims to create safe and effective, regulatory-approved, nature-derived psychedelic therapies for a wide array of mental health conditions.
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### [Takeda Hyqvia 10% Injection Approved in Japan for Immunodeficiency](https://www.clinicaltrialvanguard.com/news/takeda-hyqvia-10-injection-approved-in-japan-for-immunodeficiency/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
Takeda has received approval from the Japanese Ministry of Health, Labour and Welfare for [HYQVIA](https://www.clinicaltrialvanguard.com/news/takedas-hyqvia-wins-fda-approval-for-cidp-therapy/), a facilitated subcutaneous immunoglobulin (fSCIG) therapy, for patients with agammaglobulinemia or hypogammaglobulinemia. These conditions, characterized by low antibody levels, increase the risk of serious recurring infections stemming from primary or secondary immunodeficiencies. This approval marks the first fSCIG treatment option available in Japan.
This approval is particularly impactful for Japanese patients who previously relied on intravenous or conventional subcutaneous immunoglobulin treatments, often requiring weekly or bi-weekly infusions. HYQVIA’s less frequent dosing schedule (every 3 or 4 weeks) offers a significant improvement in quality of life by reducing the burden of frequent infusions and eliminating the need for venous access. This can lead to greater patient autonomy and potentially improved adherence to therapy. The availability of HYQVIA also addresses the growing unmet need for plasma-derived therapies in Japan, where increasing awareness and improved diagnostic rates are expected to drive further demand.
HYQVIA combines immunoglobulin 10% with recombinant human hyaluronidase PH20 (rHuPH20). The rHuPH20 facilitates the dispersion and absorption of immunoglobulin, allowing larger volumes to be administered subcutaneously. The approval is supported by data from two pivotal Phase 3 studies conducted in Japan, demonstrating comparable efficacy to existing treatments while exhibiting a manageable safety profile. The most common adverse reactions were pyrexia and localized skin reactions at the injection site.
This approval signifies a step forward in the treatment landscape for immunodeficiency disorders in Japan. It offers patients a more convenient and potentially less disruptive treatment option, potentially leading to improved disease management and quality of life. It also reinforces Takeda’s commitment to expanding therapeutic options for patients in Japan, particularly in the realm of plasma-derived therapies. The increased availability of diverse treatment options, including HYQVIA, positions healthcare providers to tailor treatment strategies to individual patient needs and preferences.
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### [Beigene Tevibra Approved in US for Gastric Cancer Treatment](https://www.clinicaltrialvanguard.com/news/beigene-tevibra-approved-in-us-for-gastric-cancer-treatment/)
**Published:** December 30, 2024
**Author:** Jon Napitupulu
**Content:**
The FDA approved [BeiGene](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/)‘s TEVIMBRA ([tislelizumab](https://www.clinicaltrialvanguard.com/news/akesos-ivonescimab-os-data-from-harmoni-6-selected-for-asco-plenary/)-jsgr), combined with chemotherapy, for first-line treatment of advanced HER2-negative gastric or gastroesophageal junction adenocarcinoma (G/GEJ) in adults with PD-L1-positive tumors. This approval is based on the positive results from the RATIONALE-305 Phase 3 trial, which showed a significant overall survival benefit for patients receiving TEVIMBRA plus chemotherapy compared to chemotherapy alone. This marks the second FDA approval for TEVIMBRA in 2024, with the first being for esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy.
This approval is a critical advancement for patients with G/GEJ cancer, a disease with a limited number of effective treatment options and a relatively low five-year survival rate. The demonstrated overall survival benefit with TEVIMBRA offers new hope for patients facing this aggressive cancer. This also enhances the existing treatment landscape by providing a new first-line therapy option, potentially improving long-term outcomes for patients. This expanded indication reinforces the importance of PD-L1 biomarker testing in guiding treatment decisions for G/GEJ cancer, enabling more personalized and effective therapies.
The RATIONALE-305 trial demonstrated a 20% reduction in the risk of death in the TEVIMBRA group, with a median overall survival of 15 months compared to 12.9 months for the placebo group. Pooled safety data from various trials, which enrolled nearly 2,000 patients receiving TEVIMBRA, revealed common grade 3 or 4 adverse reactions, including neutropenia, thrombocytopenia, anemia, and fatigue, among others. The approval for this G/GEJ indication specifically targets patients whose tumors express PD-L1 with a tumor proportion score of 1 or more. Another [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) for TEVIMBRA is currently under review by the FDA for first-line treatment of advanced ESCC.
This second approval solidifies TEVIMBRA’s emerging position as a key player in the oncology landscape. It expands the therapeutic arsenal available to oncologists, providing a valuable new option for patients battling G/GEJ cancers. It also underscores the potential for further approvals and broader use of TEVIMBRA across various cancer types. This continued development of targeted therapies like TEVIMBRA holds significant promise for improving patient survival and quality of life.
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### [Telix Pharma: Noble Registry Update: TLX599-CDX PSMA SPECT Imaging](https://www.clinicaltrialvanguard.com/news/telix-pharma-noble-registry-update-tlx599-cdx-psma-spect-imaging/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
Telix Pharmaceuticals and the Oncidium foundation announced positive initial results from the NOBLE Registry, a real-world evidence study of TLX599-CDx for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) imaging. The study, published in the •European Journal of Nuclear Medicine and Molecular Imaging (EJNMMI) Reports•, demonstrated a management change in 42.5% of the 40 patients across six countries who received TLX599-CDx and underwent SPECT imaging. The study aims to improve access to advanced prostate cancer imaging, particularly in areas with limited access to PET/CT scanners.
These findings are particularly important for patients in regions with limited resources or geographical barriers to advanced imaging technology. The positive results reinforce the potential of TLX599-CDx as a viable and more accessible alternative to traditional PET-based PSMA imaging, potentially improving early diagnosis and disease management for prostate cancer patients globally. The wider availability of SPECT scanners and the established supply chain for 99mTc, used in TLX599-CDx, makes this approach potentially more cost-effective and practical for broader implementation.
The NOBLE Registry enrolled 40 patients across six countries. The initial data demonstrated a 42.5% change in patient management after receiving the TLX599-CDx and undergoing planar and SPECT imaging. Importantly, no adverse events related to the imaging agent were reported in the study. This successful initial phase highlights the potential for a broader rollout of the imaging agent, pending further clinical investigation. The use of readily available SPECT technology combined with the established 99mTc supply chain offers a promising pathway towards more equitable access to advanced prostate cancer diagnostics.
The promising initial results pave the way for further clinical activity within the NOBLE Registry. This includes potential collaborations to expand access to TLX599-CDx and the exploration of technetium-99m and rhenium-188 as a theranostic pair in prostate cancer. This points towards a future where advanced diagnostic imaging for prostate cancer is more accessible globally, improving patient outcomes and potentially transforming the diagnostic landscape for this prevalent disease.
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**Tags:** eClinical Tech News
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### [Kala Bio's $10.75M Private Placement: A Promising Opportunity?](https://www.clinicaltrialvanguard.com/news/kala-bios-10-75m-private-placement-a-promising-opportunity/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
Kala Bio, a clinical-stage biopharmaceutical company focused on eye diseases, secured approximately $10.75 million in a private placement involving both new and existing investors. The funding will primarily support the clinical development of KPI-012, a treatment for persistent corneal epithelial defect (PCED), and general corporate operations. The Phase 2b CHASE trial for KPI-012 is currently enrolling patients across over 40 clinical trial sites, with topline data expected in the second quarter of 2025.
This influx of capital is crucial for Kala Bio, allowing them to continue developing KPI-012, a potential treatment for a rare and debilitating eye condition. Positive results from the CHASE trial could significantly advance KPI-012 towards becoming the first approved therapy for PCED, addressing an unmet medical need for patients suffering from impaired corneal healing. The funding also extends Kala’s operational runway, providing stability as they advance this promising therapeutic candidate.
The private placement involved the sale of common stock at $6.44 per share and Series I Preferred Stock at $644.00 per share. Kala Bio anticipates this funding, combined with existing cash reserves, will sustain operations into the first quarter of 2026. The company aims to use the proceeds to advance KPI-012 through clinical development and for general corporate purposes. Over 80% of enrollment is complete for the CHASE trial, suggesting the study is progressing efficiently.
This financing strengthens Kala Bio’s position in developing innovative therapies for severe eye diseases. Successful CHASE trial results could pave the way for a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) submission, potentially leading to the first approved treatment for PCED and establishing Kala Bio as a key player in this therapeutic area. This positive momentum could also attract further investment and partnerships, enabling Kala Bio to expand its pipeline and address other unmet needs in ophthalmology.
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**Tags:** eClinical Tech News
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### [Delcath Systems Secures $16.3M in Funding](https://www.clinicaltrialvanguard.com/news/delcath-systems-secures-16-3m-in-funding/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
Delcath Systems, Inc. secured $16.3 million in funding through the exercise of Series E and E1 warrants, with a small percentage exercised through cashless provisions. These warrants, originally issued in 2019 as part of a private placement, had a $10.00 exercise price and were set to expire in 2024. This influx of capital bolsters Delcath’s financial position as it continues to commercialize HEPZATO, its liver-directed chemotherapy treatment.
This funding is crucial for Delcath as it validates investor confidence in the company’s trajectory and provides the necessary resources to advance its clinical development plan. The timing is particularly significant, aligning with the growing commercial adoption of HEPZATO for the treatment of metastatic uveal [melanoma](https://www.clinicaltrialvanguard.com/news/fda-approves-tudriqev-for-anti-pd-1-resistant-advanced-melanoma/) (mUM). This financial boost enables Delcath to invest further in research and development, potentially expanding HEPZATO’s applications to other liver cancers and solidifying its position in the interventional oncology market.
The $16.3 million injection significantly strengthens Delcath’s financial outlook, supplementing existing cash reserves and anticipated revenue growth from HEPZATO sales. This financial stability enables the company to execute its commercialization strategy for HEPZATO, invest in new clinical trials, and potentially expand the treatment’s applications to other liver cancers.
This funding round marks a pivotal moment for Delcath, providing the financial runway to advance HEPZATO’s market presence and explore new therapeutic avenues. It reinforces the company’s commitment to innovation in [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/) treatment and positions it for continued growth and development in the interventional oncology field. The successful execution of their clinical development plan could lead to broadened indications for HEPZATO, potentially impacting a larger patient population and further solidifying Delcath’s position in the market.
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**Categories:** News
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### [Sangamo Regains Full Rights to Hemophilia A Gene Therapy](https://www.clinicaltrialvanguard.com/news/sangamo-regains-full-rights-to-hemophilia-a-gene-therapy/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
Sangamo Therapeutics will regain control of giroctocogene [fitelparvovec](https://www.clinicaltrialvanguard.com/news/pfizer-uncovers-exciting-advance-in-hemophilia-a-gene-therapy/), a [hemophilia](https://www.clinicaltrialvanguard.com/news/denecimig-shows-consistent-safety-and-efficacy-across-age-groups-in-hemophilia-a-trial/) A gene therapy candidate, after Pfizer terminated their collaboration and licensing agreement. Pfizer cited its decision not to pursue a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) or commercialization, despite positive Phase 3 trial results. Sangamo plans to explore all options for the program, including finding a new partner.
This decision is impactful because it unexpectedly shifts the trajectory of a promising hemophilia A therapy. While Pfizer’s decision is surprising given the positive clinical data, it creates an opportunity for another company to capitalize on a late-stage asset with demonstrated efficacy. This could also lead to faster patient access if a partner with experience in rare disease commercialization steps in quickly.
The Phase 3 AFFINE trial met its primary and key secondary endpoints, demonstrating superiority over standard prophylaxis in reducing bleeding rates. Pfizer’s withdrawal comes shortly before anticipated regulatory submissions, highlighting a potential disconnect between clinical success and strategic priorities. Sangamo will need to secure funding and potentially restructure its operations to manage the transition of the program.
Moving forward, Sangamo’s success hinges on finding a suitable partner to navigate regulatory submissions and commercialization of giroctocogene fitelparvovec. This situation underscores the complexities of drug development and the importance of alignment between collaborators. The hemophilia A community awaits the next chapter for this promising therapy, hopeful for a swift path towards patient access.
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**Categories:** News
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### [RenovoRx's IP Portfolio Grows, Drug-Delivery Platform Expands](https://www.clinicaltrialvanguard.com/news/renovorxs-ip-portfolio-grows-drug-delivery-platform-expands/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
[RenovoRx](https://www.clinicaltrialvanguard.com/news/renovorx-tiger-pac-trial-surpasses-100-patient-milestone/) (Nasdaq: RNXT) is bolstering its intellectual property (IP) portfolio with a new international patent application for its Trans-Arterial Micro-Perfusion (TAMP) therapy platform, adding to its existing 18 issued and 13 pending patents. The new application details methods and tools for targeted drug delivery using micro-vessels surrounding larger arteries, aiming to improve treatment efficacy and reduce systemic toxicity associated with intravenous delivery. The company is concurrently advancing commercial plans for its FDA-cleared RenovoCath delivery system and its Phase III TIGeR-PaC clinical trial, both leveraging the TAMP platform.
This targeted drug delivery technology has the potential to reshape cancer treatment. Direct delivery to tumor sites via the vasa vasorum offers a more precise and potentially less toxic alternative to traditional intravenous chemotherapy, addressing a critical need for improved treatment options with fewer side effects. Specifically, for [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/), a notoriously difficult-to-treat disease, the TAMP approach could represent a significant advancement in patient care.
RenovoRx’s expanded IP protection for TAMP is crucial for securing its market position and maximizing the platform’s value. The company is pursuing commercialization of RenovoCath as a standalone device and anticipates completing enrollment and a second interim analysis for its TIGeR-PaC trial, which is evaluating gemcitabine delivered via TAMP for locally advanced pancreatic cancer, in the first half of 2025. Furthermore, the company has recently received its first purchase orders for RenovoCath, indicating initial market traction.
The combination of progressing clinical trials, growing IP protection, and initial commercial orders suggests a positive trajectory for RenovoRx. A successful outcome in the TIGeR-PaC trial combined with robust IP could position RenovoRx as a leader in targeted cancer therapy, significantly impacting the oncology landscape. This focused approach to drug delivery could lead to broader adoption of TAMP-powered treatments, offering patients a more effective and tolerable option for combating various cancers.
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### [Verastem Oncology: Avutometinib/defactinib NDA for Low-Grade Serous Ovarian Cancer Gets Priority Review](https://www.clinicaltrialvanguard.com/news/verastem-oncology-avatometinib-defactinib-nda-for-low-grade-serous-ovarian-cancer-gets-priority-review/)
**Published:** December 31, 2024
**Author:** Jon Napitupulu
**Content:**
Verastem Oncology’s New Drug Application (NDA) for a combination therapy of [avutometinib](https://www.clinicaltrialvanguard.com/news/verastem-oncology-ndas-avutometinib-for-ovarian-cancer/) and defactinib, designed to treat recurrent low-grade serous [ovarian cancer](https://www.clinicaltrialvanguard.com/news/imunons-imnn-001-shows-lower-residual-disease-in-phase-2-ovarian-cancer-study/) (LGSOC) with a KRAS mutation, has been accepted for Priority Review by the FDA. The FDA has set a target action date of June 30, 2025, and if approved, this would be the first FDA-approved treatment specifically for this patient population. The application is based on positive results from the Phase 2 RAMP 201 clinical trial, which showed substantial overall response rates and durable responses in patients with recurrent KRAS mutant LGSOC.
This FDA acceptance is crucial for patients with LGSOC, a rare and often fatal ovarian cancer distinct from its high-grade counterpart. Currently, no FDA-approved treatments exist specifically for LGSOC, leaving patients with limited options. The combination of avutometinib, a RAF/MEK clamp, and defactinib, a FAK inhibitor, offers a potential breakthrough for these patients, particularly those with the KRAS mutation, by targeting specific pathways that drive cancer cell survival and tumor growth. The Priority Review designation further underscores the unmet need and the potential impact this therapy could have.
The NDA submission includes data from the Phase 2 RAMP 201 trial, highlighting the combination therapy’s efficacy and tolerability. Supportive data from the earlier Phase 1 FRAME trial is also included. A Phase 3 confirmatory trial, RAMP 301, is currently enrolling patients with recurrent LGSOC, regardless of KRAS mutation status. This trial will not only confirm the findings of the Phase 2 study but could also potentially expand the treatment’s indication to include patients without the KRAS mutation.
A potential FDA approval in June 2025 would mark a significant milestone for Verastem Oncology and, more importantly, for patients battling recurrent LGSOC. It would provide a much-needed targeted treatment option and could pave the way for further research and development in this area. The ongoing Phase 3 trial has the potential to broaden the treatment’s applicability and benefit a larger population of LGSOC patients. This progress could significantly alter the treatment landscape for this challenging cancer.
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### [Hutchmed Divests Non-Core Joint Venture for US$608 Million](https://www.clinicaltrialvanguard.com/news/hutchmed-divests-non-core-joint-venture-for-us608-million/)
**Published:** January 2, 2025
**Author:** Jon Napitupulu
**Content:**
Hutchmed is divesting its 45% stake in Shanghai Hutchison Pharmaceuticals Limited (SHPL) for approximately US$608 million to GP Health Service Capital and Shanghai Pharma. This move allows [HUTCHMED](https://www.clinicaltrialvanguard.com/news/hutchmed-highlights-new-lung-cancer-data-at-2025-conferences/) to concentrate on its core oncology and immunology business, particularly its next-generation antibody-targeted therapy conjugate (ATTC) programs. The divestiture aligns with HUTCHMED’s 2022 strategy to prioritize its core innovative medicines business and enhance shareholder value.
This divestiture is a strategic move for HUTCHMED, enabling the company to shift from a non-core asset to high-growth areas like oncology and immunology. The influx of capital will significantly bolster HUTCHMED’s R&D efforts, specifically advancing the promising ATTC platform, which has shown strong pre-clinical results. This sharpened focus could lead to accelerated development timelines for novel cancer therapies and solidify HUTCHMED’s position in the rapidly evolving oncology landscape.
Hutchmed will retain a 5% equity interest in SHPL and will benefit from a three-year transition period where they maintain influence over SHPL’s management. The company expects to report a pre-tax gain of approximately US$477 million from this transaction, which will primarily fuel the development of its internal pipeline, including the ATTC platform. The first ATTC candidates are anticipated to enter clinical trials in the second half of 2025. This platform differentiates itself from traditional antibody-drug conjugates (ADCs) by linking antibodies with targeted therapies rather than cytotoxins, potentially offering dual mechanisms of action and improved tolerability.
This divestment marks a pivotal moment for HUTCHMED. By strategically reallocating resources, the company strengthens its commitment to innovative drug development. The financial boost from the sale, coupled with the focused investment in the ATTC platform, positions HUTCHMED for potential breakthroughs in cancer treatment and reinforces their dedication to bringing novel therapies to patients. The progress of the ATTC program will be a key indicator of the long-term success of this strategic shift.
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**Tags:** eClinical Tech News
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### [Firefly Neuroscience Secures $12.4M Financing](https://www.clinicaltrialvanguard.com/news/firefly-neuroscience-secures-12-4m-financing/)
**Published:** January 2, 2025
**Author:** Jon Napitupulu
**Content:**
Firefly Neuroscience (NASDAQ: AIFF) secured up to $12.4 million in financing, consisting of $2.4 million in convertible notes and a $10 million equity line of credit. The funding will support the growth and commercialization of Firefly’s FDA-cleared Brain Network Analytics (BNA™) technology, along with general working capital needs. The company aims to partner with pharmaceutical companies and medical practitioners to expand the use of BNA™ in both research and clinical settings.
This financing is crucial for Firefly as it transitions from research and development to active commercialization of its BNA™ technology. The influx of capital enables Firefly to pursue two key market segments: pharmaceutical companies conducting clinical trials and individual practitioners diagnosing and treating patients. Successfully penetrating these markets could establish BNA™ as a valuable tool in neuroscience, potentially leading to wider adoption and improved patient outcomes.
The $12.4 million financing package offers Firefly the necessary resources to expand its operations and accelerate the commercialization of BNA™. The convertible notes provide immediate capital, while the equity line of credit offers flexibility for future needs. Firefly’s BNA™ technology, leveraging AI and machine learning analysis of a large EEG database, aims to provide more detailed insights into brain function for improved diagnosis and treatment of neurological and mental disorders.
This funding round marks a pivotal moment for Firefly Neuroscience. Securing this capital positions the company to aggressively pursue its commercialization strategy and potentially transform how neurological and [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions are diagnosed and treated. The success of this venture will depend on Firefly’s ability to effectively market BNA™ to its target audiences and demonstrate its clinical utility in real-world settings. The coming months will be critical in observing market adoption and the impact of BNA™ on patient care.
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### [Cancer Immunotherapy Market 2025-2029: Emerging Trends & Growth](https://www.clinicaltrialvanguard.com/news/cancer-immunotherapy-market-2025-2029-emerging-trends-growth/)
**Published:** January 2, 2025
**Author:** Jon Napitupulu
**Content:**
A new report from ResearchAndMarkets.com analyzes the burgeoning cancer immunotherapy market, projecting its growth through 2029. The report examines various immunotherapies, including CAR-T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), checkpoint inhibitors, and cytokines, across different cancer types and customer segments. It offers strategic insights for executives, investors, and consultants, providing data on market size, trends, key players, and recent developments in the field.
This in-depth market analysis is crucial for stakeholders in the pharmaceutical and healthcare industries. It provides a clear understanding of the rapidly evolving landscape of cancer immunotherapy, enabling informed decision-making regarding investment, research and development, and strategic partnerships. The report’s focus on emerging technologies and market trends allows companies to identify potential opportunities and navigate the complexities of this innovative field. It also empowers healthcare providers to stay abreast of the latest advancements and make informed treatment decisions for their patients.
The report covers a comprehensive range of immuno-oncology therapeutics, including monoclonal antibodies, cancer vaccines, cytokines, cell-based therapies, and other emerging modalities. It segments the market by therapy type, cancer indication, and customer type, providing granular data on market size and growth projections. The report also profiles key companies in the immunotherapy space, offering insights into their pipelines and strategic positioning. Recent developments, including FDA approvals, clinical trial initiations, and emerging research, are also highlighted.
The continued advancement of cancer immunotherapy holds immense promise for transforming cancer care. This report provides a crucial roadmap for navigating this complex and dynamic market, enabling stakeholders to capitalize on the opportunities presented by this revolutionary field and ultimately improve patient outcomes. The detailed analysis of market trends, technological advancements, and competitive dynamics will be instrumental in shaping the future of cancer treatment.
Source link: [http://www.businesswire.com/news/home/20241231736499/en/Cancer-Immunotherapy-Market-Research-2025-2029-Forecasts-for-Immuno-Oncology-Therapeutics-by-Therapy-Cancer-and-Customer-with-Executive-and-Consultant-Guides—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20241231736499/en/Cancer-Immunotherapy-Market-Research-2025-2029-Forecasts-for-Immuno-Oncology-Therapeutics-by-Therapy-Cancer-and-Customer-with-Executive-and-Consultant-Guides---ResearchAndMarkets.com)
**Categories:** News
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### [Cellenkos and the Future of T-Regulatory Cell Therapy: A Conversation with Simrit Parmar](https://www.clinicaltrialvanguard.com/executiveinterviews/cellenkos-and-the-future-of-t-regulatory-cell-therapy-a-conversation-with-simrit-parmar/)
**Published:** January 2, 2025
**Author:** Moe Alsumidaie
**Content:**
We recently spoke with Simrit Parmar, MD, founder of [Cellenkos, Inc.](https://cellenkosinc.com/) about their pioneering work in T-regulatory cell therapies. Our discussion covered the intricate mechanisms of these therapies, the promising results from their clinical trials, and the strategic direction of Cellenkos’ development programs. We focused particularly on CK0804, a product showing significant potential in treating myelofibrosis and other inflammatory disorders. This dialogue highlighted the scientific innovations at Cellenkos and provided insights into the future of [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) in addressing complex medical conditions.
## [](#what-roles-do-t-regulatory-cells-play-in-our-immune-system-especially-in-inflammation-and-autoimmune-diseases)**What roles do T-regulatory cells play in our immune system, especially in inflammation and autoimmune diseases?**
Simrit Parmar: T-regulatory cells are crucial regulators within the immune system, acting as a subtype of T cells that help resolve unwanted inflammation. These cells target cytotoxic T cells responsible for driving uncontrolled inflammation, functioning as the body’s policemen to maintain checks and balances. In autoimmune diseases, where inflammation can persist and cause damage, T-regulatory cells suppress such a prolonged inflammatory response, ensuring the immune system does not mistakenly attack the body’s tissues. This regulatory function prevents excessive immune responses from leading to chronic conditions.
The body constantly encounters foreign antigens as well as internal threats, where inflammation is a natural mechanism to expel such threats. However, when inflammation overstays its welcome, T-regulatory cells intervene to resolve it, preventing potential damage from chronic inflammation. This ability to modulate the immune response makes T-regulatory cells a key focus in developing therapies for inflammatory and autoimmune diseases, as we offer a targeted approach to restoring immune balance without broadly suppressing the immune system.
## [](#your-recent-phase-1b-trial-data-for-ck0804-in-myelofibrosis-patients-showed-promising-results-could-you-elaborate-on-the-specific-mechanisms-through-which-this-product-exerts-its-effects)**Your recent Phase 1B trial data for CK0804 in myelofibrosis patients showed promising results. Could you elaborate on the specific mechanisms through which this product exerts its effects?**
Simrit Parmar: CK0804 is a T-regulatory cell therapy product that is enriched for a specific antigen, CXCR4, on its cell surface. This enrichment allows the CK0804 Treg cells to home in to the sites of inflammation where its ligand, CXCL12, is overexpressed, such as in the case of myelofibrosis in the bone marrow and spleen. The CK0804 cells act like a heat-guided missile, targeting these areas to resolve inflammation through a multipronged approach, including cytokine depletion and cytotoxic T cell suppression. In the trial, patients experienced significant reductions in spleen volume and symptom burden, demonstrating the product candidate’s targeted efficacy and potential to improve patient outcomes.
Simrit Parmar, MD, founder of Cellenkos
Upon reaching the site of inflammation, T-regulatory cells deplete IL-2, act as cytokine sinks, secrete suppressor cytokines like interleukin-10, and can kill cytotoxic T cells and other harmful players. This comprehensive approach allows CK0804 to resolve inflammation and effectively improve myelofibrosis patients’ symptoms. The trial’s encouraging results, with six out of nine patients experiencing more than a 50% reduction in symptom burden, highlight the potential of CK0804 as a transformative therapy for this challenging condition.
## [](#how-are-you-aligning-your-clinical-development-strategies-to-meet-regulatory-expectations-especially-with-the-fdas-clearance-of-your-ind-application-for-ck0804-as-an-add-on-therapy)**How are you aligning your clinical development strategies to meet regulatory expectations, especially with the FDA’s clearance of your IND application for CK0804 as an add-on therapy?**
Simrit Parmar: As more drugs have been approved for treatment of myelofibrosis, the company is expanding its trials to include patients resistant to these newer treatments. This strategic alignment not only meets regulatory expectations but also addresses a broader patient population, enhancing the potential impact of our therapy. The ability to administer our CK0804 cells outpatient with no major side effects and their complementary nature to existing therapies allows for potential combination treatments, further broadening the scope of our clinical development.
The cells can be given without hospitalization and have shown no major side effects. This outpatient administration is a significant advantage, making the therapy more accessible and less burdensome for patients. Additionally, the complementary mechanisms of action of the cells with existing approved products create opportunities for synergy, allowing for combination treatments that could enhance efficacy and address unmet medical needs. This strategic approach positions Cellenkos to effectively navigate regulatory pathways and bring our innovative therapies to market.
## [](#what-preliminary-safety-and-efficacy-data-have-you-observed-in-your-second-cohort-and-how-do-these-outcomes-influence-your-approach-to-dosing-and-patient-selection)**What preliminary safety and efficacy data have you observed in your second cohort, and how do these outcomes influence your approach to dosing and patient selection?**
Simrit Parmar: Cellenkos has been cautious with patient monitoring, initially requiring a four-hour observation period post-infusion, now reduced to two hours based on safety data. This reduction reflects the therapy’s favorable safety profile, allowing for a more streamlined patient experience. Efficacy observations have led the company to adjust dosing schedules to address immune escape between doses, ensuring a more consistent reduction in the inflammatory response. For instance, in the trial, certain patients experienced immune escape between the first and second doses, prompting a modification to weekly doses initially, followed by monthly doses.
The importance of adapting our approach based on observed data has informed patient selection and dosing strategies. By interweaving insights from different programs, Cellenkos can create intelligent solutions for subsequent trials, optimizing the effectiveness of our therapies. This adaptive strategy enhances the potential for successful outcomes and demonstrates the company’s commitment to evidence-based decision-making in clinical development.
## [](#how-do-you-prioritize-resource-allocation-among-your-various-clinical-programs-and-what-criteria-guide-your-decisions-to-advance-a-candidate-into-clinical-development)**How do you prioritize resource allocation among your various clinical programs, and what criteria guide your decisions to advance a candidate into clinical development?**
Simrit Parmar: Cellenkos prioritizes resource allocation based on unmet medical needs, such as aplastic anemia, where our lead asset, CK0801, has shown promising results in inducing transfusion independence. The durability of response, akin to a transplant, drives our decision to prioritize resources for a pivotal trial. For instance, patients who responded to CK0801 experienced durable independence from blood and platelet transfusions for almost 3.5 years, highlighting the transformative potential of this therapy and justifying its prioritization. This focus on high-impact areas ensures that our resources are directed toward programs with the greatest potential to improve patient outcomes.
Cellenkos considers its candidates’ scalability and commercial viability when advancing them into clinical development. The company’s ability to manufacture large quantities of cells from a single manufacturing campaign and its innovative cryopreservation techniques support the scalability of its therapies. By ensuring consistent product quality and scalability, Cellenkos can effectively market its therapies, addressing clinical and commercial considerations. This strategic approach allows the company to balance scientific innovation with practical implementation, ensuring that our therapies can reach the patients who need them most.
## [](#what-innovations-have-you-implemented-to-ensure-consistent-product-quality-and-scalability-for-allogenic-t-regulatory-cell-therapies)**What innovations have you implemented to ensure consistent product quality and scalability for allogenic T-regulatory cell therapies?**
Simrit Parmar: Cellenkos has implemented several innovations to ensure consistent product quality and scalability for our allogeneic T-regulatory cell therapies. One key innovation is using umbilical cord blood as the cell source, which provides hardwired, naive cells that can be infused across HLA barriers without preconditioning with chemotherapy and/or lymphodepletion. This approach enhances the safety and efficacy of our therapies and simplifies the manufacturing process. Additionally, our cryopreservation techniques allow for long-term storage and rapid deployment, which is crucial for timely treatment access. During the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, Cellenkos was able to create drug depots across different clinical trial sites, ensuring that patients could receive treatment quickly despite logistical challenges.
Cellenkos has addressed two main items: the source of the cells and commercial scale-up. By deriving cells from umbilical cord blood, we have harnessed the natural protective mechanisms present in the umbilical cord, which connects the baby to the mother’s placenta. These cells are hardwired to be protective, reducing the risk of transforming into effector T cells, a major issue that plagues autologous Treg cell therapy. The company has also developed innovative programs and new IP to cryopreserve these cells using control rate freezers, storing them in liquid nitrogen for up to three years. This capability ensures that when a patient needs the therapy, it can be administered almost immediately, overcoming a significant point of failure in cell therapy.
## [](#how-do-collaborations-such-as-your-study-with-mount-sinai-enhance-your-research-capabilities-and-what-outcomes-do-you-anticipate-from-these-partnerships)**How do collaborations, such as your study with Mount Sinai, enhance your research capabilities, and what outcomes do you anticipate from these partnerships?**
Simrit Parmar: By partnering with experts like Dr. Ron Hoffman at Mount Sinai, Cellenkos can validate and expand our research through third-party evaluation. These partnerships allow the company to explore new questions and validate our findings, enhancing our scientific credibility and understanding of our therapies’ potential. For instance, collaborating with Mount Sinai enables Cellenkos to examine its cells in various models and conditions, providing deeper insights into our mechanisms and potential applications. This collaborative approach strengthens our research and fosters innovation and discovery.
Collaborations with scientific laboratories that pursue deep science allow Cellenkos to examine and interrogate its cells in different systems, providing valuable insights that may not have been considered internally. Third-party validation adds credibility to our company and scientific acumen, helping us learn about our CK0804 cells in different conditions. Specifically, the collaboration with Mount Sinai involves examining the interaction of the cells with other approved agents in the field and exploring specific PDX models to understand how these cells can interact with newly diagnosed myelofibrosis. These layers of examination are crucial for advancing our research and development efforts.
**Categories:** Article: Executive Interviews
---
### [Navigating Clinical Trials: Insights from Scitech Development](https://www.clinicaltrialvanguard.com/executiveinterviews/navigating-clinical-trials-insights-from-scitech-development/)
**Published:** January 2, 2025
**Author:** Moe Alsumidaie
**Content:**
In this Q&A, we sit down with [David Schaffer](https://www.linkedin.com/in/david-schaffer-258a84/) from [SciTech Development](https://www.scitechdevelopment.com/) to discuss the progress of their Phase 1 trial for ST-001 nanoFenretinide, the impact of recent funding, site selection for clinical trials, and future plans for their nanoparticle delivery platform. Schaffer also shares insights into key partnerships and strategic goals for the coming year.
## [](#moe-alsumidaie-how-is-your-phase-1-trial-for-st-001-nanofenretinide-progressing-and-what-challenges-have-you-encountered)**Moe Alsumidaie: How is your Phase 1 trial for ST-001 nanoFenretinide progressing, and what challenges have you encountered?**
David Schaffer: Reaching the clinical study stage is a significant milestone for any early-stage biotech company, but it comes with its own set of complexities. We are currently in the Phase 1A accelerated portion of our study, which has a unique structure approved by the FDA. This trial builds on over 40 previous clinical studies, focusing on demonstrating the safe delivery of fenretinide and the ability to dose escalate. Historically, challenges included limited absorption and toxicities from previous delivery mechanisms. For instance, Johnson & Johnson faced issues where the body couldn’t absorb additional doses, and emulsions used in other trials caused toxicities like increased triglyceride levels. We are now at dose level 7, where we had expected to see drug related activity, and the
David Schaffer, Director of Investor Relations and Business Development at SciTech Development
clinicians are observing encouraging signs of drug activity with minimal side effects. In fact, we now have two confirmed responses at that level, and we are now dosing at levels 8 and 9. The drug continues to be well tolerated in patients. We are close to concluding the accelerated portion and transitioning to a more traditional arm, which will allow us to set the dose level for our next study on small-cell lung cancer.
## [](#moe-alsumidaie-why-did-you-choose-columbia-usc-and-upmc-for-trial-sites-and-how-have-they-aided-recruitment)**Moe Alsumidaie: Why did you choose Columbia, USC, and UPMC for trial sites, and how have they aided recruitment?**
David Schaffer: Patient recruitment has been our biggest challenge, so we chose sites like Pittsburgh, Columbia, MD Anderson, and USC due to their high patient counts and comprehensive cancer centers. These sites have been instrumental in dosing more patients and generating interest from other clinicians. For example, Pittsburgh has been our lead site, dosing more patients than any other. Our network has expanded to include eight active sites, such as the City of Hope and the University of Colorado, with more in the pipeline. The enthusiasm from clinicians and their efforts in recruiting both patients and additional sites have been invaluable. This is exemplified by the growing interest from clinicians wanting to join our study, as seen by the increased attendance at our upcoming dinner at the American Society of Hematology meetings. This kind of organic growth and interest is something you can’t put a value on, and it significantly boosts our recruitment efforts.
## [](#moe-alsumidaie-what-impact-has-the-recent-3-2-million-funding-had-on-your-clinical-trials)**Moe Alsumidaie: What impact has the recent $3.2 million funding had on your clinical trials?**
David Schaffer: The $3.2 million from our second convertible note has been crucial in maintaining our study’s momentum. It has enabled us to reach our current stage and continue building momentum. We are now oversubscribed in commitments for our third note, which will help us raise an additional $20 million needed to file the NDA for T cell lymphoma within 18 months. Despite the typical billion-dollar costs associated with advancing oncology therapeutics, we’ve managed to progress with a modest budget, thanks to strategic planning and partnerships. For instance, we’ve raised about $12 million through convertible notes and grants, and we’ve received drug supplies from the National Cancer Institute, allowing us to execute our plan efficiently. This funding strategy has been pivotal in allowing us to advance our trials without the massive financial burdens typically associated with drug development.
## [](#moe-alsumidaie-are-there-plans-to-use-your-nanoparticle-delivery-platform-with-other-drugs-or-conditions)**Moe Alsumidaie: Are there plans to use your nanoparticle delivery platform with other drugs or conditions?**
David Schaffer: Yes, our platform has potential applications with other water-insoluble drugs. While each construct requires specific development, the foundational technology of our nanoparticles can be adapted for other molecules, and we have intellectual property protection in place. The basic premise of using nanoparticles, as demonstrated with ST-001, can be applied to other drugs, potentially expanding our platform’s utility across various therapeutic areas. This adaptability opens up numerous possibilities for treating different conditions, and we are actively exploring these opportunities. Our goal is to leverage this technology to address a broader range of medical needs, thereby maximizing the impact of our innovations.
## [](#moe-alsumidaie-are-there-key-partnerships-that-are-crucial-to-advancing-your-pipeline)**Moe Alsumidaie: Are there key partnerships that are crucial to advancing your pipeline?**
David Schaffer: We have several key partnerships, including a reliable lipid supplier in Germany and the Plough Center for Sterile Drug Delivery for manufacturing. Our clinical sites and advisors, like Larissa Gaskin at Columbia, are also crucial. Additionally, our internal team, with experts like Tony Polverino, who has extensive experience in commercializing specialty oncology drug delivery technologies, and Ken Massey, who has led clinical trials for Pfizer, provides invaluable experience in advancing our pipeline. These partnerships and our team’s expertise have been instrumental in overcoming challenges and driving our progress. They provide the support and resources necessary to navigate the complexities of drug development, ensuring that we remain on track to achieve our goals.
## [](#moe-alsumidaie-what-major-milestones-do-you-aim-to-achieve-in-the-next-year-or-so)**Moe Alsumidaie: What major milestones do you aim to achieve in the next year or so?**
David Schaffer: We aim to demonstrate the durability of responses in both T-cell and small-cell lung studies, showcasing the drug’s effectiveness in solid tumors and blood disorders. This is significant because few oncology therapeutics target both types of cancers. Scaling manufacturing and attracting pharmaceutical partnerships for companion therapeutics are also key. We anticipate expanding our studies to at least 15 other cancers, including breast and [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/), and exploring the drug’s potential in non-cancerous conditions like diabetes and [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/). The partner side is expected to grow, as evidenced by the 23 meetings we had at a recent pharma partnering conference, indicating strong interest from pharmaceutical companies in collaborating with us. These milestones are crucial for validating our approach and setting the stage for broader applications of our technology.
**Categories:** Article: Executive Interviews
---
### [Exploring New Frontiers in Immunology with Revolo](https://www.clinicaltrialvanguard.com/executiveinterviews/exploring-new-frontiers-in-immunology-with-revolo/)
**Published:** January 2, 2025
**Author:** Jon Napitupulu
**Content:**
In a recent interview, we spoke with Kari Brown, Chief Medical Officer, and Woody Bryan, Chief Executive Officer, from Revolo, about their innovative approach to treating allergic and autoimmune diseases. We discussed their lead candidate, ‘1104, clinical trial outcomes, and the implications for chronic disease management.
## [](#moe-alsumidaie-how-does-revolos-approach-reshape-treatment-for-allergic-and-autoimmune-diseases)**Moe Alsumidaie: How does Revolo’s approach reshape treatment for allergic and autoimmune diseases?**
Kari Brown: Our approach at Revolo focuses on restoring immune system homeostasis rather than suppressing it. Traditional therapies often target diseases downstream after inflammation has occurred and typically do not affect the regulatory arm of the immune system. This limits opportunities for disease modification or sustained effects post-treatment. The need for broader mechanisms of action is highlighted by the recent trial of [Benralizumab](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-trial-that-should-rewrite-the-rare-disease-playbook-but-probably-wont/) in [eosinophilic esophagitis](https://www.clinicaltrialvanguard.com/news/tezspire-hits-both-primary-endpoints-in-phase-3-eosinophilic-esophagitis-trial/) (EoE). Benralizumab did not improve patient-reported outcomes despite effectively depleting eosinophils.. Our candidate, ‘1104, acts higher in the immune cascade, offering a more comprehensive impact beyond just eosinophils. Our Phase 2A study in EoE showed a significant reduction in eosinophils in esophageal tissue, our
Kari Brown, Chief Medical Officer at Revolo
primary endpoint. Additionally, we saw improvements in patient-reported outcomes using the Dysphagia Symptom Questionnaire, a key measure in EoE trials. This dual impact on histological and patient-reported outcomes is key and promising in this therapeutic area.
## [](#moe-alsumidaie-what-does-1104s-success-in-eoe-signal-for-broader-applicability)**Moe Alsumidaie: What does ‘1104’s success in EOE signal for broader applicability?**
Kari Brown: We’ve considered this extensively and identified [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) as our next target after EoE. Both conditions involve chronic allergic inflammation, and we have positive efficacy data from EoE that we believe will translate well. Additionally, our preclinical data in atopic dermatitis is supportive. The broader impact of ‘1104 on CD8 and CD4 T regulatory and B regulatory cells suggests potential across multiple TH2 diseases. This broad mechanism of action allows us to consider multiple indications, but we chose atopic dermatitis due to its similarities with EoE, both disease involve a compromised epithelial barrier function, and the existing preclinical support. The decision also factors in the current treatment landscape and the clinical development program’s feasibility. We believe that targeting these mechanisms can offer new hope to patients suffering from these chronic conditions.
## [](#moe-alsumidaie-why-focus-on-dosing-flexibility-with-subcutaneous-and-sublingual-options)**Moe Alsumidaie: Why focus on dosing flexibility with subcutaneous and sublingual options?**
Woody Bryan: Despite being a peptide, ‘1104 can be delivered intravenously, subcutaneously, and sublingually. This flexibility is significant compared to monoclonal antibodies, which are typically limited to subcutaneous delivery due to their size and chemistry. Our subcutaneous formulation shows persistent pharmacodynamic effects, potentially allowing for less frequent dosing, such as bi-weekly or even longer intervals. The sublingual option is particularly exciting for EoE, where oral options are limited to steroids, which have safety concerns with chronic use. While oral JAK inhibitors exist in atopic dermatitis, they come with black box warnings. Our sublingual formulation could offer a safer,
Woody Bryan, CEO at Revolo
more convenient alternative, potentially improving patient adherence and transforming chronic disease management. This dosing flexibility enhances patient convenience and broadens the potential applications of ‘1104 across various conditions.
## [](#moe-alsumidaie-what-excites-you-most-about-1104s-research-and-its-potential-impact)**Moe Alsumidaie: What excites you most about ‘1104’s research and its potential impact?**
Woody Bryan: The ability to broadly impact the immune system while maintaining a clean safety profile is exciting. We’ve treated nearly 150 individuals with ‘1104 without safety signals, even at higher doses. This broad impact and safety are unique in the immune space. The flexibility in dosing also broadens treatment options, potentially setting a new standard in managing these diseases. Unlike steroids, which are effective but come with significant safety concerns, ‘1104 offers a targeted approach with a promising safety profile. This could revolutionize how we approach treatment in allergic and autoimmune diseases, providing effective and safe options for patients. We are optimistic that ‘1104 will set a new benchmark in immunology, offering a novel solution to patients who have long-awaited safer and more effective treatments.
## [](#moe-alsumidaie-how-do-you-select-additional-indications-for-1104-studies)**Moe Alsumidaie: How do you select additional indications for ‘1104 studies?**
Woody Bryan: We consider scientific and clinical rationale, development timelines, and unmet needs. For instance, as previously discussed, atopic dermatitis is a logical next step due to its similarities with EoE and our supportive preclinical data. We also have promising preclinical data in allergic airway models and IgE-mediated food allergy, suggesting broad potential across TH2 diseases. The current treatment landscape and the feasibility of a clinical development program plays a role in our decision-making process for indication expansion. By carefully evaluating these factors, we aim to maximize the impact of ‘1104 across a range of conditions, ultimately improving patient outcomes.
## [](#moe-alsumidaie-what-are-the-anticipated-regulatory-milestones-and-production-plans-for-1104)**Moe Alsumidaie: What are the anticipated regulatory milestones and production plans for ‘1104?**
Kari Brown: We aim to start a larger Phase 2 trial in EOE next year, focusing on subcutaneous dosing. This trial will explore higher doses and longer treatment durations in a larger cohort, preparing us for a Phase 3 program. We also plan to enter the clinic with atopic dermatitis. For scaling, we’ve improved our peptide synthesis process for better purity and scalability, and our sublingual platform has proven robust in other applications. We’re well-prepared for scaling both drug substance and product, ensuring we can meet the demands of commercialization if and when the time comes. Our proactive approach to scaling and regulatory planning positions us well to bring ‘1104 to market, with the potential to offer new hope to patients with chronic allergic and autoimmune diseases.
**Categories:** Article: Executive Interviews
---
### [Neumora Therapeutics Reports Positive Navacaprant Data in MDD](https://www.clinicaltrialvanguard.com/news/neumora-therapeutics-reports-positive-navacaprant-data-in-mdd/)
**Published:** January 3, 2025
**Author:** Jon Napitupulu
**Content:**
Neumora Therapeutics announced Phase 3 trial results for its [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD) drug, navacaprant. The KOASTAL-1 study, the first of three in the pivotal KOASTAL program, did not meet its primary endpoint of a statistically significant reduction in depressive symptoms, measured by the MADRS total score, compared to placebo. However, a potential efficacy signal was observed in female participants, prompting further analysis. Navacaprant demonstrated a generally acceptable safety profile comparable to placebo.
This news carries implications for the MDD treatment landscape. While the overall results are a setback for Neumora, the efficacy signal observed in the female subgroup presents a critical avenue for investigation. This potential sex-specific response could lead to a more personalized approach to MDD treatment, particularly given the higher prevalence of depression in women. Further investigation into this subgroup could reveal valuable insights into the underlying mechanisms of MDD and inform the development of more targeted therapies.
In the KOASTAL-1 study, 383 adult patients with MDD received either navacaprant 80 mg or a placebo. Both the navacaprant and placebo groups showed a similar reduction in MADRS total score at week 6 (-12.5). A statistically significant improvement in anhedonia, as measured by the SHAPS total score, was observed in female participants receiving navacaprant compared to those receiving placebo. Importantly, navacaprant’s safety profile was comparable to placebo, with no serious adverse events reported and no increased risk of suicidal ideation or behavior.
The future of navacaprant hinges on further analysis of the KOASTAL-1 data, particularly the female subgroup findings, and the outcomes of the ongoing KOASTAL-2 and KOASTAL-3 trials. If subsequent trials confirm the efficacy signal in women, Neumora may be able to pursue a more targeted development strategy. This approach could involve focusing on a female-specific indication or refining the drug’s formulation or dosage regimen. The results of these ongoing studies, combined with the additional analyses, will determine the next steps for the navacaprant program and its potential role in addressing the significant unmet need in MDD treatment.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [Capricor Submits Deramiocel BLA to FDA for Duchenne Muscular Dystrophy](https://www.clinicaltrialvanguard.com/news/capricor-submits-deramiocel-bla-to-fda-for-duchenne-muscular-dystrophy/)
**Published:** January 3, 2025
**Author:** Jon Napitupulu
**Content:**
Capricor Therapeutics has submitted a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) to the U.S. FDA for deramiocel, a [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) designed to treat [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/) (DMD) cardiomyopathy. This submission triggered a $10 million milestone payment from Nippon Shinyaku, Capricor’s distribution partner. If approved, deramiocel would be the first therapy specifically targeting DMD-related heart disease.
This BLA submission is a critical advancement in the fight against DMD, a devastating genetic disorder affecting approximately 15,000-20,000 individuals in the U.S. Current treatment options for DMD are limited, and there is no cure. DMD cardiomyopathy, a debilitating heart condition caused by the disease, is the leading cause of death among DMD patients. Deramiocel offers a potential breakthrough for these patients, addressing the critical unmet need for a therapy that specifically targets this life-threatening complication. The therapy’s potential to slow or reverse cardiac damage could significantly improve the quality of life and potentially extend the lifespan of DMD patients.
The BLA submission is based on positive data from Capricor’s Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials. These data, compared to natural history data on DMD cardiomyopathy progression, demonstrated the potential of deramiocel to attenuate the cardiac implications of the disease. Capricor has requested priority review, which could shorten the FDA review process to six months. Deramiocel has already received Orphan Drug Designation from both the FDA and the European Medicines Agency, underscoring its potential to address an unmet medical need. Furthermore, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. and Advanced Therapy Medicinal Product (ATMP) designation in Europe, expediting the development and review processes. Should deramiocel receive FDA approval, Capricor would be eligible for a Priority Review Voucher, a valuable asset that can be used to expedite the review of a future drug application.
The BLA submission represents a major milestone for Capricor and a significant step towards potential approval of the first therapy for DMD cardiomyopathy. A positive FDA decision would validate years of research and development, potentially transforming the treatment landscape for DMD patients suffering from this life-threatening heart condition. It would also position Capricor as a leader in the field of regenerative medicine and pave the way for further development of its innovative cell and exosome-based therapies for other rare diseases. Pending approval, the next steps will include finalizing commercialization plans with Nippon Shinyaku and preparing for the potential launch of this much-needed therapy.
Source link:
**Categories:** News
**Tags:** eClinical Tech News
---
### [Pain Treatment Market 2024-2029: Medtronic, Abbott, GSK, Pfizer, J&J](https://www.clinicaltrialvanguard.com/news/pain-treatment-market-2024-2029-medtronic-abbott-gsk-pfizer-jj/)
**Published:** January 6, 2025
**Author:** Jon Napitupulu
**Content:**
The global conventional and alternative pain treatment market, valued at $96.2 billion in 2024, is projected to reach $144.2 billion by 2029, growing at a CAGR of 8.40%. This growth is driven by increasing demand for pain management solutions, including pharmaceuticals, devices like TENS units and spine stimulators, and alternative therapies like medical cannabis. The report segments the market by pain management type (pharmaceuticals/drug therapy and devices), application (orthopedic pain, surgical pain, [migraine](https://www.clinicaltrialvanguard.com/news/lundbecks-bocunebart-meets-primary-endpoint-in-phase-iib-migraine-trial/), etc.), and geography (North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa).
This market expansion is crucial for healthcare systems and patients grappling with chronic and acute pain. The rising prevalence of chronic diseases and an aging population fuels the need for effective pain management strategies. Developing and adopting new technologies and treatments offers significant opportunities to improve patients’ quality of life and reduce the societal burden of pain-related disability. This growth also indicates a shift towards a more holistic approach to pain management, incorporating both conventional pharmaceuticals and alternative therapies.
The devices segment, representing 36.6% of the market in 2023, is anticipated to grow at a CAGR of 6.7%, driven by the increasing use of electrotherapy and spinal cord stimulation devices. Within pharmaceuticals, the demand for non-narcotic analgesics, medical cannabis, and other drug classes contributes to overall market growth. The report highlights key regional markets, including established markets like North America and Europe and emerging markets in Asia-Pacific and Latin America.
The projected growth of the pain treatment market signifies a positive trend towards addressing the unmet needs of pain sufferers. Continued innovation in drug development, device technology, and alternative therapies will likely shape the future of pain management, offering hope for more effective and personalized treatment options. The increasing adoption of alternative treatments and devices could lead to a shift away from reliance solely on pharmaceutical interventions, potentially resulting in fewer side effects and improved patient outcomes.
Source link: [http://www.businesswire.com/news/home/20250103965508/en/Conventional-and-Alternative-Pain-Treatment-Market-Research-2024-2029-Profiles-of-Market-Leaders—Medtronic-Abbott-GSK-Pfizer-Inc.-and-Johnson-Johnson—ResearchAndMarkets.com](http://www.businesswire.com/news/home/20250103965508/en/Conventional-and-Alternative-Pain-Treatment-Market-Research-2024-2029-Profiles-of-Market-Leaders---Medtronic-Abbott-GSK-Pfizer-Inc.-and-Johnson-Johnson---ResearchAndMarkets.com)
**Categories:** News
---
### [Advancing Leukemia Care with Sellas: GPS & SLS009 Insights](https://www.clinicaltrialvanguard.com/executiveinterviews/advancing-leukemia-care-with-sellas-gps-sls009-insights/)
**Published:** January 7, 2025
**Author:** Moe Alsumidaie
**Content:**
We had the opportunity to speak with Dr. Angelos M. Stergiou, MD, Sc.D. h.c., about potential advancements in [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) treatment through Sellas [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/)‘ lead assets, GPS and [SLS009](https://www.clinicaltrialvanguard.com/news/sellas-launches-pioneering-aml-trial-enrolls-first-patient/). These innovative therapies are being developed to address significant unmet medical needs in leukemia, offering the potential for improved survival rates and enhanced quality of life for patients. Dr. Stergiou provided an in-depth look at the mechanisms of action, clinical trial progress, and strategic positioning of these candidates within the evolving landscape of acute myeloid leukemia (AML), highlighting their potential to transform patient outcomes.
## [](#moe-alsumidaie-can-you-tell-me-about-your-lead-assets-gps-and-sls009-and-what-roles-they-could-potentially-play-in-treating-leukemia)**Moe Alsumidaie: Can you tell me about your lead assets, GPS and SLS009, and what roles they could potentially play in treating leukemia?**
Dr. Angelos Stergiou: GPS, or galinpepimut-S, is a promising asset we in-licensed from Memorial Sloan Kettering Cancer Center, currently in a pivotal phase three trial for AML, the REGAL study. It targets the WT1 antigen, which is overexpressed in many tumor types and is designed to be maximally immunogenic, targeting both CD4 and CD8 cells. This multivalent approach involves 25 carefully selected WT1 epitopes, enhancing its immunogenicity and breaking tolerance, allowing for long-term administration. The safety profile of GPS is comparable to that of a standard vaccine, with minimal side effects. This makes it a potentially unique asset among leukemia treatments, offering longer survival without compromising quality of life.
Dr. Angelos M. Stergiou, MD, Sc.D President and CEO of Sellas
Our other lead asset, SLS009 is a CDK9 inhibitor that blocks the production of cancer-promoting proteins, leading to cancer cell death. Its high selectivity minimizes toxicity, allowing for combination with other drugs. This selectivity is crucial, as previous CDK9 inhibitors faced challenges due to toxicity. SLS009’s exceptional safety profile enables its use in combination therapies without exacerbating toxicities, providing a significant advantage in treating leukemia as well as other hematological and solid cancer types. It’s effective in killing cancer cells while being in the body long enough to avoid severe toxicities, making it a promising candidate for combination therapies in AML.
## [](#moe-alsumidaie-what-specific-efficacy-and-safety-benchmarks-are-you-aiming-to-meet-in-the-regal-trial-and-how-might-these-results-influence-the-trials-continuation-or-modification)**Moe Alsumidaie: What specific efficacy and safety benchmarks are you aiming to meet in the REGAL trial, and how might these results influence the trial’s continuation or modification?**
Dr. Angelos Stergiou: The REGAL trial’s primary endpoint is overall survival, and we’ve recently triggered the 60th event for an interim analysis by the Independent Data Monitoring Committee (IDMC). The IDMC will assess safety, futility, and efficacy, hoping to recommend the trial’s continuation without modification or, ideally, stopping due to exceptional efficacy. The final analysis requires 80 events, aiming for a hazard ratio of approximately 0.636 to declare statistical significance. The IDMC’s role is crucial, as it evaluates the trial’s conduct, validity, and scientific integrity, guiding whether to continue, modify, or discontinue the trial.
If the IDMC recommends discontinuation due to exceptional efficacy, it would be a significant milestone, indicating that GPS has shown a substantial benefit in extending survival for AML patients at this early stage already. If the trial continues to the final analysis, it likely suggests that GPS is on track to meet its efficacy goals, further validating its potential as a maintenance therapy in AML. We’re optimistic about the trial’s outcomes, highlighting the importance of the IDMC’s independent assessment in guiding the trial’s future direction and ensuring the safety and efficacy of GPS for patients.
## [](#moe-alsumidaie-how-are-you-aligning-your-clinical-trials-to-meet-regulatory-requirements-and-what-challenges-have-you-encountered-in-this-process)**Moe Alsumidaie: How are you aligning your clinical trials to meet regulatory requirements, and what challenges have you encountered in this process?**
Dr. Angelos Stergiou: For GPS, the primary endpoint of overall survival is well-defined and agreed upon with the FDA, ensuring alignment with regulatory expectations and, in addition, we have received fast track and orphan drug designations. SLS009 has received a rare pediatric disease designation for pediatric acute lymphoblastic leukemia, as well as fast track and orphan drug designations in AML by the FDA and we’re focusing on its development in AML. The challenge lies in ensuring trials meet stringent requirements across jurisdictions but encouraging data in overall response rates and survival guides discussions with regulatory bodies. In the ongoing Phase 2a trial with SLS009, we’ve observed a median overall survival exceeding 7.7 months in patients refractory to venetoclax-based regimens, compared to the historical 2.5 months.
This significant improvement emphasizes the potential of SLS009 to transform outcomes for heavily pre-treated AML patients, and we’re preparing to engage with the FDA about a potential accelerated clinical and regulatory approval pathway. Aligning clinical trial designs and endpoints with regulatory requirements is crucial to ensure successful approval processes. The promising results from SLS009 trials provide a strong foundation for regulatory discussions, emphasizing that we’re on the right path to potentially bring this innovative candidate to patients.
## [](#moe-alsumidaie-what-are-the-key-considerations-in-selecting-combination-partners-for-sls009-and-how-do-you-assess-the-potential-for-synergistic-effects-versus-increased-toxicity)**Moe Alsumidaie: What are the key considerations in selecting combination partners for SLS009, and how do you assess the potential for synergistic effects versus increased toxicity?**
Dr. Angelos Stergiou: We focus on combinations with BCL-2 blockers like venetoclax, as SLS009 shows extraordinary synergy with these agents. Preclinical and clinical data indicate that SLS009 can improve response rates and is well-tolerated, even in combination. For example, preclinical studies developed a model using cancer cell lines resistant to venetoclax, which, when treated with SLS009, showed a strong efficacy effect. This model was validated in mice and most importantly further supported by patient data, demonstrating that SLS009 does not exacerbate venetoclax’s myelosuppressive effects. As almost all AML patients have heterogenous cancer cells, some cells will depend mostly on BCL2 and will be killed by venetoclax, some will depend on MCL1 and will be killed by SLS009, and some will depend on both BCL2 and MCL1 and the two-fold assault by both anti-apoptotic agents at the same time. In our ongoing Phase 2 study, the addition of azacitidine also allows for a NOXA release enhancement, thus increasing pro-apoptotic effect, a triple hit which may potentially increase the response rates in relapse/refractory AML patients. I am extremely hopeful that SLS009 will make an impact in the management of patients with ASXL1-mutated AML and potentially other myeloid malignancies with similar disease biology as we have seen remarkable response and survival data in those patients.
This rigorous approach ensures that the synergistic potential of SLS009 in combination therapies is maximized while minimizing safety risks. Combining SLS009 with other treatments without increasing toxicity is a key differentiator, offering a promising strategy for enhancing treatment outcomes in AML and potentially other cancers.
## [](#moe-alsumidaie-how-do-you-plan-to-position-gps-and-sls009-within-the-current-treatment-paradigm-for-aml-and-what-differentiates-your-approach)**Moe Alsumidaie: How do you plan to position GPS and SLS009 within the current treatment paradigm for AML, and what differentiates your approach?**
Dr. Angelos Stergiou: GPS is positioned as a maintenance therapy in AML, particularly after complete remission, due to its excellent safety profile. GPS works in the maintenance setting, providing a safe and effective option for patients in remission, potentially extending their survival without compromising quality of life. SLS009 currently targets relapse refractory AML, with the potential to move frontline. Our approach is differentiated by the safety and efficacy of the treatments, including differentiated biology and mechanism of action in ASXL1 mutation tumor types, allowing for potential coverage of the entire AML treatment paradigm, from initial treatment with SLS009 to maintenance with GPS.
Together, these assets offer a comprehensive strategy for managing AML, addressing both immediate and long-term treatment needs. We’re enthusiastic about the potential of GPS and SLS009 to impact patients’ lives, either individually or collectively significantly. The strategic positioning of these treatments within the evolving landscape of AML highlights their potential to transform patient outcomes and provide new hope for those affected by this challenging disease.
**Categories:** Article: Executive Interviews
---
### [Grove Secures $4.9M for Agentic AI Clinical Trials](https://www.clinicaltrialvanguard.com/news/grove-secures-4-9m-for-agentic-ai-clinical-trials/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
Grove AI, a clinical trial management company, secured $4.9 million in seed funding led by A•, with participation from other venture capital firms and industry angels. The funding will support team and product expansion as Grove aims to revolutionize clinical trial operations. The company’s goal is to transform trials from siloed processes into participant-centric ecosystems fueled by real-time data.
This development is important because clinical trials are plagued by inefficient communication, slow feedback, and cumbersome workflows, leading to participant frustration, staff burnout, and costly delays for pharmaceutical companies. Grove’s platform addresses these issues directly, which could significantly impact the speed and cost of drug development. A more efficient, patient-centered approach could lead to faster approval times for new therapies and improved patient experiences.
Grove’s AI-powered platform streamlines participant qualification and engagement, offering actionable insights for optimized trial operations. The platform boasts a 97% participant satisfaction rate and integrates with existing systems to enhance areas like time-to-first-visit and participant diversity. Central to the platform is “Grace,” an AI research agent that handles tasks like participant prescreening, appointment scheduling, and follow-up logistics, freeing up research staff. Early success has been demonstrated with a partner, K2 Medical Research, which saw increased efficiency and scale in their [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) and vaccine trials.
This funding round signals growing investor confidence in AI-driven solutions for clinical trials. Grove’s focus on improving the participant experience, combined with its data-driven approach to optimizing operations, positions the company to be a key player in the evolving clinical trial landscape. The successful implementation of this technology could fundamentally reshape how clinical trials are conducted, leading to a more efficient and patient-focused drug development process.
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**Categories:** News
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### [Biomea Fusion Reveals Promising Preclinical Data on Icovamenib Combination](https://www.clinicaltrialvanguard.com/news/biomea-fusion-reveals-promising-preclinical-data-on-icovamenib-combination/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
Biomea Fusion announced positive preclinical data for [icovamenib](https://www.clinicaltrialvanguard.com/news/biomeas-icovamenib-shows-lasting-c-peptide-gains-in-t1d/) combined with [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/), a GLP-1 receptor agonist, in treating [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/). The combination therapy showed significant improvements in several metabolic parameters compared to semaglutide alone, including enhanced glucose control, reduced insulin resistance, and increased lean muscle mass. These findings were observed in a preclinical study using Zucker Diabetic Fatty (ZDF) rats, a model for type 2 diabetes.
These results are potentially transformative for diabetes care. The combination of icovamenib and semaglutide addresses multiple critical aspects of the disease simultaneously. Improving insulin production, glucose control, and body composition while potentially allowing for lower GLP-1 doses could lead to better patient outcomes and reduced side effects. Furthermore, the increase in lean muscle mass alongside fat reduction suggests a unique benefit of this combination not typically seen with existing therapies. This comprehensive approach could significantly improve the quality of life for individuals with type 2 diabetes.
The combination therapy resulted in a 60% greater reduction in fasting blood glucose and a 50% greater improvement in glucose metabolism during an oral glucose tolerance test. Insulin resistance decreased by 75%, and beta-cell function improved. Beyond these metabolic benefits, the combination therapy led to an 11.5% greater reduction in body weight and a remarkable 43% increase in lean muscle mass compared to semaglutide alone.
This data suggests that icovamenib, by amplifying the effects of GLP-1 agonists, could become a cornerstone of future diabetes treatment. The anticipated clinical trials will be crucial for validating these preclinical findings and exploring the potential for lower GLP-1 doses, potentially mitigating side effects and expanding access to a broader patient population. The potential for beta-cell regeneration offered by icovamenib could address a fundamental aspect of diabetes, making it a disease-modifying therapy rather than just managing symptoms. This could represent a significant advancement in diabetes care.
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**Tags:** eClinical Tech News
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### [Sight Sciences Study Shows OMNI's Effectiveness in Glaucoma Management](https://www.clinicaltrialvanguard.com/news/sight-sciences-study-shows-omnis-effectiveness-in-glaucoma-management/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
A 36-month study using data from the American Academy of Ophthalmology IRIS® Registry demonstrated that Sight Sciences’ OMNI® Surgical System significantly reduced intraocular pressure (IOP) and medication dependence in patients with primary open-angle [glaucoma](https://www.clinicaltrialvanguard.com/news/nurexone-shows-vision-recovery-in-preclinical-glaucoma-model/) (POAG). This research is notable because it analyzes standalone OMNI use, independent of cataract surgery, providing crucial real-world data on its long-term effectiveness. The study, published in the •American Journal of Ophthalmology•, involved 230 eyes of 196 POAG patients and was led by Dr. Nathan M. Radcliffe of Mount Sinai School of Medicine.
This study’s findings are vital for ophthalmologists and POAG patients as they offer evidence of a minimally invasive glaucoma surgery (MIGS) option effective over three years. Specifically, the demonstrated sustained IOP reduction and decreased medication needs suggest a potential shift in treatment paradigms. This offers a valuable alternative to traditional glaucoma surgeries and long-term reliance on medication, especially for patients who are not candidates for or prefer to avoid cataract surgery. The ability to achieve significant IOP reduction without implants further enhances the appeal of OMNI as a MIGS procedure.
The study showed mean IOP reductions ranging from 5.6 to 7.1 mmHg over 36 months, with even greater reductions observed in patients with higher baseline IOPs (up to 8.9 mmHg). Medication usage also decreased significantly through 18 months post-operatively, and those with lower baseline IOP saw this reduction sustained through 36 months. The utilization of the IRIS Registry, a comprehensive database capturing millions of patient encounters, underscores the robust nature of these findings and their applicability to real-world clinical practice.
This positive long-term data strengthens OMNI’s position as a viable treatment option for POAG, potentially increasing its adoption among ophthalmologists. It also reinforces the trend toward minimally invasive procedures in glaucoma management, offering patients a less burdensome approach with sustained benefits. The study’s focus on standalone use broadens the potential patient population for OMNI and could lead to earlier intervention for glaucoma, potentially slowing disease progression and preserving vision.
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**Categories:** News
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### [Sana Biotechnology Shows Positive Results in Type 1 Diabetes Study](https://www.clinicaltrialvanguard.com/news/sana-biotechnology-shows-positive-results-in-type-1-diabetes-study/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
Sana Biotechnology announced positive initial results from a first-in-human study involving a patient with [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/). The study, conducted in partnership with Uppsala University Hospital, transplanted allogeneic pancreatic islet cells engineered with Sana’s hypoimmune (HIP) technology, [UP421](https://www.clinicaltrialvanguard.com/news/sana-biotechnology-positive-results-in-type-1-diabetes-islet-cell-transplant-study/), without requiring immunosuppression. Four weeks post-transplant, the patient exhibited detectable C-peptide levels, indicating insulin production by the transplanted cells, along with graft survival confirmed by MRI.
This development represents a potential paradigm shift in type 1 diabetes treatment. Successfully transplanting allogeneic islet cells without immunosuppression could eliminate the need for lifelong insulin injections and the risks associated with immunosuppressive drugs. This approach could translate into a functional cure for type 1 diabetes, significantly improving patients’ quality of life and long-term health outcomes. Furthermore, this success could extend to other diseases requiring transplantation, broadening the impact of this technology.
Technically, the presence of C-peptide, a byproduct of insulin production, both at baseline and after a mixed meal tolerance test confirms the functionality of the transplanted cells. The sustained MRI signal at the transplant site indicates graft survival. Strategically, these results validate Sana’s HIP technology and provide crucial data for advancing their stem cell-derived islet cell program, SC451.
This breakthrough lays the foundation for developing a readily scalable and potentially curative treatment for type 1 diabetes. While longer-term follow-up is necessary to assess the durability of the results, these initial findings signal a significant advancement towards a future where cell therapies could offer a viable cure for this chronic disease and potentially many others.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Solid Biosciences Announces FDA IND Clearance for First-in-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia](https://www.clinicaltrialvanguard.com/news/solid-biosciences-announces-fda-ind-clearance-for-first-in-industry-dual-route-of-administration-gene-therapy-to-treat-both-neurologic-and-cardiac-manifestations-of-friedreichs-ataxia/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
[Solid Biosciences](https://www.clinicaltrialvanguard.com/news/solid-biosciences-advancing-neuromuscular-and-cardiac-gene-therapy/) has received FDA clearance for its Investigational New Drug (IND) application for SGT-212, a gene therapy designed to treat Friedreich’s ataxia (FA). SGT-212 is unique because it uses a dual route of administration – intravenous infusion and direct infusion into the cerebellum – to deliver a full-length frataxin gene. This approach aims to address both the neurological and cardiac manifestations of FA, a debilitating and life-shortening genetic disorder.
This IND clearance is a critical step for FA patients currently lacking effective treatment options. SGT-212’s targeted approach addresses a significant unmet need by aiming to treat both the debilitating neurological symptoms, such as loss of coordination and speech difficulties, and the life-threatening cardiac issues that characterize FA. The dual delivery method is a novel strategy designed to overcome the challenges in treating this complex multisystem disease, specifically addressing the need for precise frataxin protein expression levels in the cerebellum and heart.
SGT-212 utilizes an AAV vector to deliver the frataxin gene. Preclinical studies have shown the therapy successfully transduced target tissues, restored neurological function, and reversed cardiac implications in mice. A Phase 1b clinical trial is expected to begin in the second half of 2025. This trial will be an open-label, dose-finding study enrolling both ambulatory and non-ambulatory adult FA patients. The trial will assess the safety and tolerability of SGT-212 and will involve a five-year follow-up period.
The IND clearance for SGT-212 represents a significant advancement in the field of gene therapy for FA. The upcoming clinical trial will provide crucial data regarding the safety and efficacy of this novel dual-administration approach. Positive results from this trial could pave the way for a new treatment paradigm for FA, potentially offering hope for patients suffering from this devastating disease.
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**Tags:** eClinical Tech News
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### [Asher Biotherapeutics AB248 & Rilvegostomig in NSCLC: Phase 1b/2 Study Announced](https://www.clinicaltrialvanguard.com/news/asher-biotherapeutics-ab248-rilvegostomig-in-nsclc-phase-1b-2-study-announced/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
Asher [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) and AstraZeneca have partnered to evaluate etakafusp alfa (AB248), Asher Bio’s CD8+ T cell-targeted IL-2 immunotherapy, in combination with AstraZeneca’s PD-1/[TIGIT](https://www.clinicaltrialvanguard.com/news/anti-tigit-antibody-the-revolutionary-cancer-treatment-you-must-know-about/) bispecific antibody, rilvegostomig. This combination therapy will be investigated as a first-line treatment for patients with advanced or metastatic non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) in a global study sponsored by AstraZeneca. Asher Bio will retain ownership of etakafusp alfa and will provide it to AstraZeneca at no cost.
This collaboration is important because it addresses a significant unmet need in NSCLC treatment. Current therapies often prove inadequate for advanced or metastatic NSCLC, which carries a low 5-year survival rate. Combining etakafusp alfa’s targeted CD8+ T cell activation with rilvegostomig’s dual targeting of PD-1 and TIGIT pathways offers a potentially synergistic approach to enhance anti-tumor immunity and improve outcomes for these patients. The selection of this combination for a first-line therapy approach suggests a strong belief in its potential efficacy compared to existing standard treatments.
AstraZeneca will be fully responsible for the operational aspects and costs of the global clinical trial. This allows Asher Bio to focus on further developing its cis-targeting platform and expanding its pipeline, which includes other targeted immunotherapies such as AB821, an IND-ready CD8-targeted IL-21 immunotherapy. Preliminary data from a Phase 1a/1b trial of etakafusp alfa demonstrate its ability to selectively activate CD8+ T cells, showing promising anti-tumor activity and a tolerable safety profile.
This collaboration validates Asher Bio’s approach to targeted immunotherapy and positions etakafusp alfa as a potentially key component of future combination therapies for NSCLC. The clinical trial results will be crucial for determining the efficacy of this combination and could pave the way for a new treatment paradigm for advanced NSCLC, potentially offering improved outcomes for patients who currently have limited options. The collaboration also strengthens Asher Bio’s position within the immuno-oncology field and could lead to further partnerships and development opportunities.
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**Categories:** News
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### [Cassava Sciences Slashes Workforce by 33%](https://www.clinicaltrialvanguard.com/news/cassava-sciences-slashes-workforce-by-33/)
**Published:** January 8, 2025
**Author:** Jon Napitupulu
**Content:**
Cassava Sciences announced a 33% workforce reduction, cost-cutting measures, and anticipated topline data release from its Phase 3 REFOCUS-ALZ study of simufilam for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease in late Q1/early Q2 2025. This follows the failure of its Phase 3 RETHINK-ALZ study to meet primary endpoints in November 2024. The company held $128.6 million in cash and cash equivalents at the end of 2024.
The upcoming REFOCUS-ALZ data is crucial for determining the future of simufilam. While RETHINK-ALZ’s failure significantly dampened the drug’s prospects, REFOCUS-ALZ, with its larger patient population and longer study duration, presents a final opportunity to demonstrate simufilam’s efficacy. The results will dictate whether further development of simufilam is scientifically and financially viable. For the Alzheimer’s community, the data carries significant weight as the search for effective treatments continues.
Cassava Sciences implemented cost-cutting measures, including a workforce reduction of 10 employees (33%), and discontinued biomarker analysis from previous Phase 2 trials. The company expects a one-time cost of approximately $0.4 million related to the workforce reduction in Q1 2025. These actions, combined with the existing cash reserves, aim to extend the company’s runway as it awaits the REFOCUS-ALZ results.
The release of the REFOCUS-ALZ data represents a pivotal moment for Cassava Sciences. Positive results, although unlikely after the RETHINK-ALZ failure, could potentially revitalize the simufilam program. However, negative data will likely lead to the termination of simufilam development and force the company to reassess its strategic direction. The outcome will heavily influence the future of the company and have implications for the broader Alzheimer’s drug development landscape.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Bitterroot Bio Announces Positive Phase 1 Data for BRB-002](https://www.clinicaltrialvanguard.com/news/bitterroot-bio-announces-positive-phase-1-data-for-brb-002/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
Bitterroot Bio announced positive Phase 1 results for [BRB-002](https://www.clinicaltrialvanguard.com/news/bitterroot-bio-presents-phase-1-brb-002-results-at-acc-25/), a first-in-class immune-modulating protein therapy targeting CD47 for atherosclerotic cardiovascular disease. The study in healthy volunteers showed BRB-002 was safe at all tested doses, with no serious adverse events, and demonstrated dose-dependent target engagement. A Phase 2 proof-of-concept study in patients with established atherosclerosis is anticipated to begin in the first half of 2025.
This positive Phase 1 data is a crucial step towards addressing the significant residual risk of cardiovascular events faced by atherosclerosis patients, even with existing treatments like lipid-lowering therapy. The demonstration of safety and target engagement in humans de-risks further development and supports the rationale for investigating BRB-002’s potential to reduce this risk. This successful first-in-human study provides valuable early clinical validation for Bitterroot Bio’s cardio-immunology approach, which aims to modulate the immune system’s role in cardiovascular disease.
The Phase 1 study demonstrated a dose-dependent increase in CD47 receptor occupancy, reaching up to 100% at the highest doses. Importantly, preclinical models have indicated that even lower occupancy levels (6-26%) correlated with meaningful plaque reduction. The absence of serious adverse events and concerning hematological effects, like anemia or thrombocytopenia, further strengthens the drug’s profile.
This positive Phase 1 data positions BRB-002 for advancement into Phase 2 trials in patients with atherosclerosis. Successful results in the upcoming Phase 2 trial would not only validate the therapeutic potential of BRB-002, but also represent a significant advance in the field of cardio-immunology, potentially offering a new treatment avenue for atherosclerotic cardiovascular disease. The results of this study will be presented at the American College of Cardiology Scientific Session in March 2025, offering a more detailed insight into the findings.
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### [Aerwave's Ultrasound Therapy Shows Promise for COPD & Asthma](https://www.clinicaltrialvanguard.com/news/aerwaves-ultrasound-therapy-shows-promise-for-copd-asthma/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
AerWave Medical completed its first-in-human feasibility study of a novel ultrasound lung denervation therapy for chronic obstructive pulmonary disease (COPD) and [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/). The study, involving five patients, confirmed the feasibility and efficiency of AerWave’s ultrasound platform, which simplifies lung denervation by eliminating the need for fluoroscopic guidance, esophageal marker balloons, and multiple catheter exchanges. The company also secured $12 million in funding led by Lucius Partners.
This successful first-in-human trial signifies a potential breakthrough in treating COPD and asthma. Current radiofrequency-based lung denervation methods, while showing promise, are complex and pose safety and efficacy limitations. AerWave’s technology addresses these issues with a single-shot circumferential ablation procedure, avoiding sensitive areas like peri-esophageal nerves. This less invasive approach, combined with the elimination of fluoroscopy and other ancillary devices, could lead to wider adoption of lung denervation and ultimately, better patient outcomes. The substantial funding further solidifies the company’s position and potential to disrupt the respiratory care market.
AerWave’s ultrasound platform delivers precise circumferential ablation in a single energy application, reducing airway smooth muscle constriction and targeting the underlying causes of COPD and asthma. The universal catheter design accommodates diverse airway anatomies. This platform technology also extends to conformal lung tumor ablation (LTA) and lung volume reduction (LVR), offering a less invasive alternative to current surgical procedures. These additional applications further diversify AerWave’s potential market impact and create opportunities for future growth.
The successful feasibility study, coupled with the influx of new funding, positions AerWave Medical to accelerate the development and clinical validation of its ultrasound technology. This could usher in a new era in interventional pulmonology, offering a more precise, efficient, and potentially safer approach to treating respiratory diseases and potentially expanding treatment options for [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) and emphysema. AerWave is actively pursuing strategic partnerships to further advance its technology.
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**Tags:** eClinical Tech News
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### [Quanta FDA Clearance for QTX3544 KRAS Inhibitor & Pipeline Updates](https://www.clinicaltrialvanguard.com/news/quanta-fda-clearance-for-qtx3544-kras-inhibitor-pipeline-updates/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
Quanta Therapeutics announced advancements in its KRAS-directed drug candidates, including FDA IND clearance for [QTX3544](https://www.clinicaltrialvanguard.com/news/quantas-qtx3544-a-powerful-new-oral-multi-kras-inhibitor/), a G12V-preferring multi-KRAS inhibitor, allowing for a Phase 1 clinical trial. The company also initiated the combination phase of its ongoing Phase 1 trial evaluating QTX3046, a G12D-selective KRAS inhibitor, with [cetuximab](https://www.clinicaltrialvanguard.com/news/frontier-medicines-presents-preclinical-data-on-3-programs/), an EGFR inhibitor. A separate Phase 1 trial evaluating [QTX3034](https://www.clinicaltrialvanguard.com/news/quanta-announces-leadership-team-clinical-progress/), a G12D-preferring multi-KRAS inhibitor, continues, with data anticipated in 2025.
These advancements are important because they expand the potential treatment options for patients with KRAS-driven cancers, specifically targeting the G12V, G12D, and G12C mutations prevalent in cancers like colorectal, pancreatic, and lung. The combination therapy approach with cetuximab aims to enhance treatment efficacy and durability, mirroring the successful strategy seen with adagrasib in KRAS G12C-mutated colorectal cancer. This progress suggests a broader reach toward addressing the high unmet need in treating these aggressive cancers.
QTX3034 and QTX3046 are distinct, oral, allosteric inhibitors targeting KRAS G12D mutations. QTX3544 is an oral multi-KRAS inhibitor with G12V-preferring activity. The Phase 1 trials are evaluating these candidates as monotherapies and in combination with cetuximab, focusing on safety, dosage, pharmacokinetics, and antitumor activity. These trials are ongoing in the U.S.
Quanta’s progress signals a potential shift in the landscape of KRAS-targeted therapies. The simultaneous development of multiple KRAS inhibitors, each with different mutation specificities and tested in combination with other targeted agents, could offer a personalized approach to cancer treatment. This could ultimately lead to improved outcomes for patients with various KRAS-driven cancers. The anticipated data readouts in 2025 will be crucial in determining the clinical viability of these promising candidates and further shaping the future direction of KRAS-focused drug development.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Molecular Switches in Cell Signaling: Drug Development](https://www.clinicaltrialvanguard.com/news/molecular-switches-in-cell-signaling-drug-development/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
The sales of the top 20 drugs targeting molecular switches have exceeded $30 billion, highlighting the growing importance of these biological mechanisms in medicine and drug development. These switches play a crucial role in cell signaling, controlling various cellular processes and offering valuable therapeutic targets for a range of diseases. The research focuses on the significance of molecular switches in areas such as regenerative medicine, nanomedicine, drug delivery, and their application in treating cancer, neurological disorders, and autoimmune diseases.
This market growth underscores the successful translation of basic research on molecular switches into tangible clinical benefits. The substantial revenue generated demonstrates a strong market demand for therapies targeting these mechanisms, encouraging further investment and research in this area. This success has a ripple effect, impacting not just the pharmaceutical companies directly involved, but also fostering growth in related sectors such as diagnostics and drug delivery systems. Furthermore, it validates the strategic focus on molecular switches as a key area for drug development.
Drugs modulating molecular switches have already achieved significant success in cancer treatment, exemplified by imatinib’s impact on chronic myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/). The development of allosteric modulators offers finer control over these switches, potentially minimizing side effects and improving efficacy in treating neurological disorders. Emerging fields like optogenetics, utilizing light-sensitive proteins, and smart drug delivery systems further expand the therapeutic possibilities of molecular switches. These advancements pave the way for more targeted and personalized therapies, moving away from traditional “one-size-fits-all” approaches.
The progress in understanding molecular switches, fueled by advances in structural biology and computational modeling, promises continued innovation in therapeutics. The development of synthetic switches, capable of controlling protein function or gene expression, opens exciting new avenues for therapeutic interventions. This ongoing research, combined with the growing market success, indicates that molecular switches will remain a central focus in the quest for more effective and precise disease treatments.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Maia and BeiGene Partner for Phase 2 Cancer Trials](https://www.clinicaltrialvanguard.com/news/maia-and-beigene-partner-for-phase-2-cancer-trials/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
MAIA Biotechnology will collaborate with [BeiGene](https://www.clinicaltrialvanguard.com/news/beigene-brukinsa-impact-on-cll-2024-ash-hematology-data-new-advances/) to evaluate THIO, MAIA’s telomere-targeting anticancer drug, in combination with BeiGene’s immune checkpoint inhibitor tislelizumab. The Phase 2 trials will focus on hepatocellular carcinoma (HCC), [small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (SCLC), and colorectal cancer (CRC), three prevalent and deadly cancer types. This collaboration leverages BeiGene’s established CPI and MAIA’s promising preclinical data showing THIO’s ability to convert “cold” tumors, unresponsive to CPIs, into “hot” tumors, thus enhancing CPI effectiveness.
This partnership is potentially transformative for cancer treatment. Preclinical data suggest THIO could significantly improve outcomes for patients with these difficult-to-treat cancers, especially by addressing the challenge of CPI resistance in “cold” tumors. Positive clinical trial results could lead to new treatment options, particularly for patients who have not responded to standard CPI therapies. This could also reshape the treatment landscape for HCC, SCLC, and CRC, which collectively represent a substantial portion of cancer-related deaths worldwide.
MAIA will sponsor and fund the trials while BeiGene supplies tislelizumab. MAIA retains global development and commercialization rights to THIO and can explore its use with other agents and in other indications. MAIA is aiming for accelerated FDA approvals for the three cancers included in the trials, as well as for [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/) (NSCLC), which is currently in a separate Phase 2 trial with THIO and a CPI. The market for HCC therapies is projected to reach $1.5 billion by 2034, while the SCLC market is estimated at $6.5 billion in 2024, growing at a CAGR of 12.3% until 2034. The CRC therapeutics market is expected to reach $26.49 billion by 2032.
This collaboration positions MAIA to potentially accelerate the development and commercialization of THIO. Successful trial results could establish THIO as a key component in combination therapies, significantly expanding market opportunities and potentially revolutionizing the treatment of these devastating cancers. This partnership not only validates the potential of THIO but also sets the stage for future development in various cancer types and treatment combinations.
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### [Sana Biotechnology Announces Positive Clinical Trial Results](https://www.clinicaltrialvanguard.com/news/sana-biotechnology-announces-positive-clinical-trial-results/)
**Published:** January 9, 2025
**Author:** Jon Napitupulu
**Content:**
Sana Biotechnology announced positive initial results from a first-in-human study involving a patient with [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/). The study, conducted in partnership with Uppsala University Hospital, transplanted allogeneic pancreatic islet cells engineered with Sana’s hypoimmune (HIP) technology without immunosuppression. The transplanted cells survived, functioned, and evaded immune detection, evidenced by C-peptide production, increased C-peptide levels after meals, and MRI scans indicating graft survival at four weeks.
This breakthrough holds significant potential for revolutionizing type 1 diabetes treatment. The ability to transplant islet cells without immunosuppression could eliminate the need for lifelong anti-rejection drugs, significantly improving patients’ quality of life and reducing the risk of complications. This success also suggests that a scalable, curative treatment for type 1 diabetes, enabling normal blood glucose levels without insulin injections or immunosuppression, may be within reach.
The study achieved its primary objective of demonstrating safety. Secondary endpoints, including cell survival, immune evasion, and C-peptide production, were also met. C-peptide, a biomarker indicating insulin production by transplanted beta cells, was detected at each weekly blood draw and increased after meals. MRI scans confirmed graft survival. This is potentially the first instance of allogeneic transplant survival without immunosuppression or immune-protective devices in a fully immune competent individual.
This early success validates Sana’s HIP technology platform and provides valuable insights for the development of SC451, Sana’s HIP-modified, stem cell-derived islet cell program. The results suggest a future where cell therapies can replace insulin-producing cells without immunosuppression, offering a potential cure for type 1 diabetes and potentially impacting other diseases requiring transplantation. Further research and longer-term follow-up are necessary to confirm the durability of these results and assess the long-term efficacy of this approach.
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### [Acumen Pharmaceutics Alzheimer's Prevention Study in Journal](https://www.clinicaltrialvanguard.com/news/acumen-pharmaceutics-alzheimers-prevention-study-in-journal/)
**Published:** January 10, 2025
**Author:** Jon Napitupulu
**Content:**
Acumen Pharmaceuticals announced positive Phase 1 clinical trial results for sabirnetug ([ACU193](https://www.clinicaltrialvanguard.com/news/acumen-launches-new-phase-2-study-for-alzheimers-drug/)), a potential [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease treatment targeting amyloid beta oligomers (AβOs). Published in the •Journal of Prevention of Alzheimer’s Disease•, the INTERCEPT-AD trial data showed sabirnetug was well-tolerated, effectively engaged AβOs, and reduced amyloid plaques in higher dose groups. A Phase 2 trial, ALTITUDE-AD, is underway with anticipated enrollment completion in the first half of 2025.
This news is noteworthy because it validates the targeting of AβOs as a potential therapeutic strategy for Alzheimer’s disease. While amyloid plaques have been a traditional focus of Alzheimer’s research, increasing evidence suggests that soluble AβOs play a critical role in the disease’s progression. Sabirnetug’s demonstrated ability to selectively target these oligomers differentiates it from existing therapies and offers a promising new avenue for treatment. The positive safety profile observed in the Phase 1 trial further strengthens the rationale for continued development.
The INTERCEPT-AD trial enrolled 65 participants with early-stage Alzheimer’s disease. Results demonstrated dose-dependent target engagement of AβOs and statistically significant amyloid plaque reduction at higher doses. The incidence of amyloid-related imaging abnormalities (ARIA), a potential side effect associated with some Alzheimer’s therapies, was low, with one case of mild, resolved ARIA-E. Notably, none of the participants with a high-risk genetic profile (apolipoprotein E Ɛ4 homozygotes) who received sabirnetug developed ARIA.
The positive Phase 1 data reinforces the potential of sabirnetug as a next-generation Alzheimer’s treatment. The ongoing Phase 2 ALTITUDE-AD trial will be crucial in further evaluating the efficacy of sabirnetug in a larger patient population and determining its potential to slow cognitive and functional decline. Positive results from this trial could significantly impact the Alzheimer’s treatment landscape and offer new hope for patients.
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### [IO Biotech's Cancer Vaccine Trial Enrolls Patients with Melanoma and Head/Neck Cancer](https://www.clinicaltrialvanguard.com/news/io-biotechs-cancer-vaccine-trial-enrolls-patients-with-melanoma-and-head-neck-cancer/)
**Published:** January 10, 2025
**Author:** Jon Napitupulu
**Content:**
[IO Biotech](https://www.clinicaltrialvanguard.com/news/io-biotechs-melanoma-data-selected-for-esmo-presentation/) has completed enrollment in its Phase 2 basket trial, IOB-032/PN-E40, ahead of schedule. The trial evaluates the company’s lead cancer vaccine candidate, [IO102](https://www.clinicaltrialvanguard.com/news/io-biotech-reveals-5-year-clinical-trial-outcomes-in-nature/)-IO103, combined with Merck’s Keytruda ([pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/)), in patients with resectable melanoma or squamous cell carcinoma of the head and neck (SCCHN). Initial data from this trial is anticipated in 2025.
This early enrollment completion allows IO Biotech to expedite the investigation of its cancer vaccine in earlier stages of disease. It builds upon encouraging clinical activity observed in earlier-stage trials in advanced melanoma, lung cancer, and head and neck cancer, where the vaccine showed promise with minimal systemic toxicity. Coupled with anticipated Phase 3 data for advanced melanoma also expected in 2025, the Phase 2 data could potentially broaden the applicability of this combination therapy across various difficult-to-treat cancers.
The IOB-032/PN-E40 trial enrolled 93 patients across the US, Europe, and Australia. The trial aims to assess the anti-tumor activity, safety, and gather biomarker data for IO102-IO103 with pembrolizumab as a neoadjuvant and adjuvant treatment. The primary endpoint is major pathological response, measured by the reduction of viable tumor cells after treatment. Secondary endpoints include pathological complete response, overall response rate, disease-free survival, event-free survival, and safety. The trial consists of three cohorts, encompassing both single-arm and randomized groups evaluating the combination therapy compared to pembrolizumab alone.
The completion of this trial enrollment positions IO Biotech to potentially expand the clinical utility of its cancer vaccine. The forthcoming data in 2025 will be crucial for understanding the efficacy of IO102-IO103 in earlier stages of cancer, possibly establishing it as a valuable treatment option in the perioperative setting. This could significantly impact patient outcomes, potentially reducing recurrence rates and improving long-term survival for those with resectable melanoma and SCCHN.
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### [MRNA Cancer Vaccine Clinical Trials & Research](https://www.clinicaltrialvanguard.com/news/mrna-cancer-vaccine-clinical-trials-research/)
**Published:** January 10, 2025
**Author:** Jon Napitupulu
**Content:**
Over 60 mRNA cancer vaccine candidates are currently in clinical trials, with two reaching Phase III. [BioNTech](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/)‘s BNT111, a promising mRNA vaccine for advanced [melanoma](https://www.clinicaltrialvanguard.com/news/fda-approves-tudriqev-for-anti-pd-1-resistant-advanced-melanoma/), showed positive Phase II results when combined with the anti-PD-1 antibody cemiplimab. Kuick Research projects the first commercial approval of an mRNA cancer vaccine by 2029.
This burgeoning field of mRNA cancer vaccines holds immense potential to revolutionize cancer treatment. The positive clinical trial results validate the strategy of combining mRNA vaccines with existing immunotherapies, potentially offering new treatment avenues for patients with limited options, particularly those with advanced melanoma who have progressed after standard therapies. The adaptability of mRNA technology allows for rapid development and manufacturing, enabling researchers to target a wide range of cancer types beyond melanoma, including breast, prostate, and colorectal cancers. This personalized approach, tailoring treatment to specific antigens, promises more effective and targeted therapies.
Currently, over 60 mRNA cancer vaccine candidates are undergoing clinical trials, with the US and China leading the research efforts. Skin cancer is a dominant area of focus, with more than 10 vaccines in development. While BNT111 shows promise for melanoma, other mRNA vaccines are being developed for various cancers. The anticipated first commercial approval by 2029 signifies the rapid advancement of this technology from research to potential widespread clinical application.
The continued development and clinical success of mRNA cancer vaccines represent a paradigm shift in cancer immunotherapy. The technology’s flexibility, coupled with growing investment and research, positions it for significant market expansion in the coming years. Successful clinical trials will likely spur further development and attract greater investment, leading to a more diverse and effective range of cancer treatment options. This progress ultimately offers hope for improved patient outcomes and a potentially transformative approach to combating cancer.
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### [Rapport Therapeutics Announces Promising Phase 1 Data for CNS Disorders](https://www.clinicaltrialvanguard.com/news/rapport-therapeutics-announces-promising-phase-1-data-for-cns-disorders/)
**Published:** January 10, 2025
**Author:** Jon Napitupulu
**Content:**
Rapport Therapeutics (Nasdaq: RAPP) announced positive Phase 1 results for [RAP-219](https://www.clinicaltrialvanguard.com/news/rapports-rap-219-shows-22-day-half-life-reshapes-phase-3-design/), a drug candidate for central nervous system disorders. The trials demonstrated that RAP-219 achieved target receptor occupancy within five days and showed a favorable tolerability profile. The company also announced the resignation of their chief medical officer, Bradley Galer.
These results are important because they suggest RAP-219 could offer a meaningful improvement over existing treatments for focal [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/) and other CNS disorders, which often have significant side effects that limit their effectiveness. RAP-219’s targeted mechanism, focusing on the TARPγ8 protein primarily found in the hippocampus and cerebral cortex, may minimize side effects often seen with less selective drugs impacting the cerebellum and brainstem. The quick achievement of target engagement suggests a faster onset of therapeutic action, a potential benefit for patients.
Across four Phase 1 trials involving 100 healthy volunteers, RAP-219 demonstrated good tolerability with no serious adverse events and limited low-grade adverse events, including no reported sedation or motor impairments. Data from the PET trial confirmed the targeted activity of RAP-219 and its concentration in relevant brain regions. The MAD-2 trial further supported the safety and tolerability profile, showing that therapeutic drug levels could be reached quickly. A Phase 2a proof-of-concept trial for RAP-219 in focal epilepsy is ongoing, with topline results anticipated in mid-2025.
The positive Phase 1 results strengthen the case for RAP-219 as a potential treatment for focal epilepsy and other CNS disorders. Confirmation of the drug’s targeted action and tolerability profile increases its potential to address unmet needs for patients suffering from these conditions. The upcoming Phase 2a results will be crucial in determining the clinical efficacy of RAP-219 and its potential path toward becoming a valuable new therapeutic option.
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---
### [EMA Discusses European Clinical Trial Decline at DTRA](https://www.clinicaltrialvanguard.com/conference-coverage/ema-discusses-european-clinical-trial-decline-at-dtra/)
**Published:** January 13, 2025
**Author:** Moe Alsumidaie
**Content:**
At the [2024 Decentralized Trials & Research Alliance (DTRA) Annual Meeting](https://www.dtra.org/dtra-2024-annual-meeting "2024 Decentralized Trials & Research Alliance (DTRA) Annual Meeting"), Dr. Amir Kalali of the DTRA and Dr. Steffen Thirstrup from the European Medicines Agency (EMA) delved into the evolving landscape of clinical trials in Europe. The discussion centered on the challenges and innovations shaping the future of clinical trials, focusing on decentralized trials and the regulatory environment. As Europe grapples with declining clinical trials, the EMA is implementing strategic measures to revitalize the sector, ensuring it remains competitive and accessible to patients.
### [](#understanding-the-emas-role)Understanding the EMA’s Role
Dr. Steffen Thirstrup provided a comprehensive overview of the EMA’s role, highlighting its distinct focus compared to the FDA. While the FDA’s broad mandate includes food, drugs, devices, cosmetics, and tobacco, the EMA concentrates primarily on pharmaceuticals. This narrower focus allows the EMA to specialize in drug regulation, although it does engage with drug-device combinations and companion diagnostics. Other areas, such as tobacco and cosmetics, fall outside its jurisdiction, with separate European agencies handling these sectors. For instance, food safety is managed by a distinct agency, though the EMA collaborates on issues like food additives. The EMA’s operations are further complicated by the European Union’s unique political structure, a union of 27 member states rather than a single nation. This necessitates a collaborative approach, with the EMA relying on a network of experts from these states. This network is crucial for coordinating clinical trials, which are increasingly global or multinational. The EMA’s role involves aligning diverse regulatory environments, requiring significant cooperation among member states.
### [](#addressing-the-decline-in-clinical-trials)Addressing the Decline in Clinical Trials
The decline in clinical trials within Europe was a primary concern addressed by Dr. Thirstrup, who noted that this trend is not aligned with the European Commission’s goals of maintaining competitiveness and ensuring patient access to new therapies. Clinical trials are vital for regulatory decision-making and advancing scientific knowledge through non-commercial academic trials. To counter this decline, the EMA has implemented a new clinical trial regulation, effective January 2022, to streamline the approval process. Previously managed nationally, the regulation seeks to create a more coordinated approach, facilitating multinational trials within Europe. Dr. Thirstrup referenced a recent report from the European Federation of Pharmaceutical Industries and Associations (EFPIA), highlighting that only a few member states have recovered from the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/)-related dip in clinical trials. This highlights the urgency of the EMA’s efforts to implement the new regulation effectively. The regulation is designed to enhance the representativeness of patient populations in trials, ensuring that regulatory decisions are based on comprehensive and diverse data sets.
### [](#innovations-in-clinical-trial-processes)Innovations in Clinical Trial Processes
The EMA is actively pursuing innovations to improve the clinical trial process, focusing on the Clinical Trial Information System (CTIS). This system is intended to serve as a one-stop portal for sponsors, allowing them to enter trial data and facilitate assessments by member states. Dr. Thirstrup acknowledged that the system faced initial technical challenges, describing them as “inborn errors.” However, he assured that many of these issues have been resolved, and the system functions more effectively. The CTIS is a key component of the EMA’s strategy to enhance trial coordination across Europe. It enables parallel assessments by regulatory authorities and ethics committees, streamlining the approval process for clinical trials. This system is particularly beneficial for sponsors conducting trials in multiple member states, as it reduces the administrative burden and facilitates a more efficient review process. In addition to the CTIS, the EMA has launched the ActEU initiative, which stands for Accelerating Clinical Trials in Europe. From 2022 to 2026, this multi-annual work plan includes ten priority actions to revitalize the clinical trial landscape in Europe. Among these actions are the development of methodologies for decentralized and complex trials, as well as the creation of a multi-stakeholder platform to engage with industry, academia, and patient organizations. These efforts reflect the EMA’s commitment to fostering innovation and collaboration in the clinical trial sector.
### [](#embracing-digital-and-real-world-data)Embracing Digital and Real-World Data
Integrating digital endpoints and real-world data into clinical trials was another focal point of the discussion. Dr. Thirstrup emphasized that decentralized trials and digital endpoints are closely linked, as the latter are essential for collecting data in a decentralized manner. He noted that the EMA is open to discussions on digital endpoints and has developed a Q&A document to address the rapid evolution of [digital health technologies](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/). This approach allows the EMA to update its guidance quickly, keeping pace with technological advancements. Digital endpoints, such as those collected through wearable devices, can enhance the quality and scope of data collected in clinical trials. They enable the collection of real-life data, which can supplement traditional data sets obtained through on-site visits. This integration of digital endpoints can lead to more comprehensive and representative data, ultimately improving the quality of regulatory decision-making. Dr. Thirstrup explained that real-world data can be used not only for post-authorization safety monitoring but also for efficacy and effectiveness studies. This approach does not aim to replace randomized clinical trials but to supplement them, providing additional insights for regulatory and health technology assessment decisions.
### [](#global-collaboration-and-future-directions)Global Collaboration and Future Directions
Global collaboration is a cornerstone of the EMA’s strategy, particularly in the context of clinical trials, which are increasingly global in nature. Dr. Thirstrup highlighted the close relationship between the EMA and the FDA, noting that the two agencies have a longstanding confidentiality agreement and engage in regular meetings. This collaboration is powerful in major therapeutic areas such as oncology, where the EMA and FDA work together to harmonize regulatory practices. The EMA collaborates with other international regulatory bodies, including Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and [Health Canada](https://www.clinicaltrialvanguard.com/news/amo-pharma-aligns-with-fda-mhra-health-canada-on-amo-02-study-design-for-cdm1/). These collaborations are facilitated through initiatives like the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the International Coalition of Medicines Regulatory Authorities (ICMRA). These efforts aim to align regulatory standards and facilitate the conduct of multinational clinical trials. Looking to the future, Dr. Thirstrup expressed optimism about the potential for hybrid clinical trials, which combine elements of traditional and decentralized approaches. He believes such trials can create more naturalistic data sets, improving the applicability of trial results to real-world settings. By reducing the number of traditional site visits and incorporating real-life data collection, hybrid trials can broaden recruitment and enhance the representativeness of patient populations.
### [](#the-impact-of-brexit)The Impact of Brexit
The EMA’s move to Amsterdam, necessitated by Brexit, has had significant implications for the agency and the broader European regulatory network. Dr. Thirstrup described the physical transition as smooth, with the Dutch government providing a new building for the EMA. However, the loss of the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) as a key player in the European Medicines Network has posed challenges. Brexit has resulted in the redistribution of responsibilities for product assessments among the remaining member states, placing additional burdens on their regulatory agencies. Moreover, the loss of the MHRA’s expertise has made it more challenging for the EMA to maintain a high level of scientific and regulatory expertise within its network. Dr. Thirstrup noted that the network’s sustainability is a recurring topic of discussion among the heads of the member states’ regulatory agencies. Despite these challenges, the EMA continues to engage with the MHRA, although the relationship has changed significantly post-Brexit. The lack of a confidentiality agreement with the UK complicates collaboration, and the MHRA’s decision to stand independently has further distanced it from the European regulatory framework. Nonetheless, the EMA remains committed to fostering collaboration and maintaining high standards of regulatory oversight in the post-Brexit landscape.
### [](#summary)Summary
In conclusion, the conference emphasized the EMA’s proactive measures to address European clinical trials’ challenges. The EMA aims to foster a robust and competitive clinical trial environment that benefits patients and the scientific community through regulatory innovations, digital integration, and global collaboration. The agency’s efforts to streamline processes, embrace new technologies, and enhance international cooperation are crucial to revitalizing the European clinical trial landscape and ensuring its continued relevance globally.
**Categories:** Article: Conference Coverage
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### [Pioneering the First Lab and Theranostic Platform: A Conversation with NuView CEO, Paul Crowe](https://www.clinicaltrialvanguard.com/executiveinterviews/pioneering-the-first-lab-and-theranostic-platform-a-conversation-with-nuview-ceo-paul-crowe/)
**Published:** January 14, 2025
**Author:** Moe Alsumidaie
**Content:**
[NuView Life Sciences](https://www.nuviewlifesciences.com/ "NuView Life Sciences") is transforming cancer diagnostics and treatment with its pioneering NV-VPAC1™ platform. This first-of-its-kind technology integrates in vitro diagnostics, in vivo imaging, and precision-targeted therapy, offering a seamless solution for earlier detection, more personalized treatments, and reduced healthcare costs. At its core is a non-invasive liquid biopsy test that promises to redefine how cancers are detected and managed, while the broader platform unlocks the full potential of theranostics in precision oncology.
## [](#moe-what-makes-nuviews-in-vitro-liquid-biopsy-test-a-breakthrough-in-cancer-diagnostics)**Moe: What makes NuView’s in vitro liquid biopsy test a breakthrough in cancer diagnostics?**
**Paul Crowe**: Our in vitro liquid biopsy test is a game-changer in early cancer detection. It’s a non-invasive, urine-based diagnostic that offers exceptional sensitivity and specificity, addressing key challenges like false positives and unnecessary biopsies. By detecting cancers such as prostate, bladder, and breast at early stages, the test empowers physicians with rapid, reliable results, enabling better treatment decisions. Beyond its diagnostic performance, the test is scalable, economical, and compatible with various liquid specimens, including blood and saliva, making it accessible to a global patient population. With the IVD market projected to grow from $80.71 billion in 2024 to $117.6 billion by 2032, we are poised to capture significant opportunities in multi-cancer detection.
Paul Crowe, CEO of [NuView](https://www.clinicaltrialvanguard.com/executiveinterviews/nuview-liquid-biopsy-high-tech-high-impact-high-reliability/) [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/)
## [](#moe-how-does-nv-vpac1-integrate-diagnostics-therapy-and-personalized-care-to-advance-cancer-treatment)**Moe: How does NV-VPAC1 integrate diagnostics, therapy, and personalized care to advance cancer treatment?**
**Paul Crowe**: NV-VPAC1 bridges the gap between diagnostics and therapy while enabling a highly personalized approach to cancer care. Using Cu-64 for imaging, our technology identifies malignant tissue with high specificity by targeting VPAC1 receptors, which are overexpressed in cancers like prostate, bladder, brain, and more. This allows us to detect cancer even before histologic changes occur, giving patients the best chance at successful treatment. For therapy, Cu-67 provides sustained radiation directly to cancer cells, minimizing damage to healthy tissue and reducing the need for frequent dosing. By tailoring diagnostics and treatment to each patient’s unique cancer profile, we’re creating a platform that treats the individual, not just the disease. This integration significantly reduces patient burden and improves outcomes, aligning with the broader push for personalized medicine.
## [](#moe-what-sets-nuview-apart-from-its-competitors-in-the-oncology-space)**Moe: What sets NuView apart from its competitors in the oncology space?**
**Paul Crowe**: NuView’s platform uniquely integrates in vitro diagnostics, in vivo imaging, and theranostics into a single, cohesive system. This comprehensive approach provides unparalleled precision, enabling earlier detection and more personalized treatment plans. Our receptor specificity is another key differentiator. Unlike competitors whose receptors lack the precision needed for broad clinical applications, our VPAC1 receptor targeting delivers unmatched accuracy and reliability, addressing multiple cancer types while reducing false positives and unnecessary follow-ups. Our robust intellectual property portfolio also ensures that NuView remains at the forefront of innovation, delivering long-term growth and sustained impact.
## [](#moe-how-is-nuview-scaling-its-technologies-and-expanding-globally)**Moe: How is NuView scaling its technologies and expanding globally?**
**Paul Crowe**: We are scaling our operations through strategic partnerships and clinical trials. One of our key initiatives is collaborating with the VA healthcare system to validate our [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) diagnostic. This partnership has the potential to improve access to non-invasive diagnostics for veterans significantly. In early 2025, we’ll begin clinical trials in Mexico City as part of our LATAM expansion. These trials will leverage AI-enhanced microscopy to refine diagnostic accuracy and establish our platform’s efficacy in a broader global context. Our regulatory milestones are also on track, with prostate cancer diagnostic trials slated for Q1 2025. This marks a significant step toward bringing NV-VPAC1 to market and demonstrating its value across diverse healthcare systems.
## [](#moe-what-is-nuviews-long-term-vision-for-its-platform)**Moe: What is NuView’s long-term vision for its platform?**
**Paul Crowe**: At NuView, our vision is to redefine precision oncology with a fully integrated platform that addresses every stage of cancer care—from early detection to targeted therapy. Our tagline, “Pioneering the First Lab and Theranostic Platform,” reflects this mission. We are committed to delivering transformative technologies that improve outcomes, reduce costs, and provide a more patient-centered approach to treatment. As we continue to grow, I’m confident that NV-VPAC1 will set a new benchmark in cancer care and establish NuView as a leader in this space.
**Categories:** Article: Executive Interviews
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### [Ashvattha Therapeutics Secures $50M, Unveils Promising Ophthalmology Trial Results](https://www.clinicaltrialvanguard.com/news/ashvattha-therapeutics-secures-50m-unveils-promising-ophthalmology-trial-results/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
Ashvattha Therapeutics secured up to $50 million in extended Series B funding, led by Tribe Capital with continued support from existing investors led by Natural Capital. This funding will propel Ashvattha’s ongoing Phase 2 ophthalmology and Phase 1/2 neuroinflammation trials for its hydroxyl dendrimer therapeutic (HDT) platform. The company’s lead candidate, [migaldendranib](https://www.clinicaltrialvanguard.com/news/ashvattha-therapeutics-positive-interim-phase-2-results-for-dme-and-wet-amd/) (MGB), a subcutaneous injection for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD) and diabetic macular edema (DME), has shown promising interim Phase 2 results, significantly reducing the need for supplemental aflibercept injections.
This development is particularly noteworthy due to the potential of MGB to address a significant unmet need in wet AMD and DME treatment. Current treatment options require frequent intravitreal injections, which pose a substantial burden on patients, especially those with bilateral disease. MGB’s subcutaneous administration, coupled with its efficacy in reducing the frequency of supplemental injections, could significantly improve patient compliance and quality of life, potentially shifting the treatment paradigm for these prevalent eye diseases.
Interim Phase 2 trial data indicates a substantial reduction in required supplemental aflibercept injections after 24 weeks for patients receiving MGB every 2 or 4 weeks, following an initial aflibercept injection. The data also suggests sustained visual acuity and reduced subretinal fluid with a positive safety profile. These findings will be presented at the Angiogenesis 2025 meeting. Importantly, MGB’s systemic administration offers a distinct advantage in treating bilateral disease, a common occurrence in wet AMD and DME, simplifying treatment compared to current localized injection approaches.
This funding and the positive interim data mark a significant step forward for Ashvattha and its HDT platform. The successful completion of these trials could pave the way for a more patient-friendly and potentially cost-effective treatment option for wet AMD and DME, addressing a large market with significant unmet needs. This advancement also positions Ashvattha’s HDT platform for broader application in other inflammatory diseases, underscoring the potential long-term impact of this technology.
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### [ORIC-944 Phase 1b Data, 2024 Highlights, Milestones](https://www.clinicaltrialvanguard.com/news/oric-944-phase-1b-data-2024-highlights-milestones/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
ORIC Pharmaceuticals announced encouraging early safety and efficacy data from a Phase 1b trial of ORIC-944 combined with apalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC). The company also highlighted operational achievements in 2024, including collaborations with Johnson & Johnson and Bayer, and provided anticipated milestones for the next 18 months. ORIC expects its current cash reserves to fund operations into late 2026.
These developments are noteworthy because they demonstrate progress in addressing treatment resistance in difficult-to-treat cancers. The early clinical data for ORIC-944, particularly the durable PSA responses, suggest the combination therapy could offer a new treatment option for mCRPC patients who have limited therapeutic choices. The collaborations with established pharmaceutical companies like Johnson & Johnson and Bayer validate ORIC’s approach and provide external resources to accelerate development. Finally, the extended cash runway allows ORIC to pursue its ambitious clinical development plans without immediate funding concerns.
ORIC-944, a PRC2 inhibitor, demonstrated promising results when combined with apalutamide. In the first two dose escalation cohorts, three out of six patients achieved a [PSA50](https://www.clinicaltrialvanguard.com/news/oric-944-trial-remarkable-efficacy-and-safety-results/) response, with two of those achieving a PSA90 response. Importantly, these responses were sustained, with one PSA90 response ongoing at 38 weeks. The combination was well-tolerated, with mostly Grade 1 and 2 adverse events. Parallel development of ORIC-944 with darolutamide is also underway, showing similar preliminary clinical activity. Beyond ORIC-944, ORIC is progressing ORIC-114, an EGFR/HER2 inhibitor, in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC), with multiple data readouts expected in the coming year and a half.
ORIC’s pipeline progress, strategic partnerships, and financial stability position the company to potentially initiate registrational trials for both ORIC-114 and ORIC-944 within the next two years. The anticipated data readouts over the next 18 months will be crucial for validating the clinical potential of these drug candidates and informing the design of pivotal studies. This progress could lead to new treatment options for patients with advanced prostate cancer and NSCLC, areas of significant unmet medical need.
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### [NeuroSigma ADHD Trial of Monarch eTNS System Completes Enrollment](https://www.clinicaltrialvanguard.com/news/neurosigma-adhd-trial-of-monarch-etns-system-completes-enrollment/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
[NeuroSigma](https://www.clinicaltrialvanguard.com/news/neurosigmas-monarch-etns-system-adhd-trial-for-children-with-fetal-alcohol-syndrome-at-ucla/), Inc. announced the completion of enrollment for the ATTENS trial, a multicenter, double-blind, placebo-controlled study evaluating the effectiveness of its Monarch® eTNS System for treating ADHD in children and adolescents. The trial, funded by the UK’s National Institute for Health and Care Research (NIHR) and the Medical Research Council (MRC), enrolled 150 participants aged 8-18 who were randomized to receive either active or sham eTNS therapy for four weeks. Data collection, including fMRI neuroimaging, clinical and cognitive assessments, and autonomic nervous system function measurements, will continue until March 2025.
This trial’s completion is crucial for advancing non-pharmaceutical ADHD treatment options. Current pharmacological interventions are not always effective or well-tolerated, leading to a significant unmet need for alternative therapies. The robust enrollment figures suggest substantial public interest in exploring non-drug solutions for ADHD, highlighting the trial’s potential impact on patient care. Furthermore, the study’s comprehensive data collection, particularly the inclusion of fMRI neuroimaging, promises to provide valuable insights into the mechanism of action of eTNS therapy. This information could significantly improve our understanding of how eTNS impacts brain activity and contributes to symptom reduction in ADHD.
The ATTENS trial represents the largest clinical investigation of the Monarch eTNS System to date, significantly expanding the existing clinical dataset. The trial is evaluating the improvement of ADHD symptoms using the ADHD-RS-V scale, a gold standard measure in ADHD clinical research. The involvement of prestigious institutions like King’s College London and the University of Southampton, coupled with the support from NIHR and MRC, lends further credibility to the study.
The results of this trial, expected in March 2025, are poised to shape the future of eTNS therapy for ADHD. Positive findings could solidify the Monarch eTNS System’s position as a viable alternative or complementary treatment to existing pharmacological options, potentially transforming clinical practice and improving the lives of individuals with ADHD. The detailed mechanistic data will also be invaluable for informing future research and development in this field, paving the way for refined and more targeted eTNS interventions for ADHD and other neurological conditions.
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### [Artelo Biosciences Announces Successful Completion of First Cohort in Phase 1 Study of ART26.12](https://www.clinicaltrialvanguard.com/news/artelo-biosciences-announces-successful-completion-of-first-cohort-in-phase-1-study-of-art26-12/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
[Artelo Biosciences](https://www.clinicaltrialvanguard.com/news/artelo-biosciences-new-data-on-non-opioid-pain-treatment/) has initiated a Phase 1 clinical trial of ART26.12, its lead FABP5 inhibitor, marking the first time a selective FABP5 inhibitor has been administered to humans. The company expects initial safety and pharmacokinetic data in the first half of 2025. This trial focuses on evaluating ART26.12 as a non-opioid treatment for various pain conditions, including chemotherapy-induced peripheral neuropathy (CIPN), diabetic neuropathy, cancer bone pain, and [osteoarthritis](https://www.clinicaltrialvanguard.com/news/nih-funds-trial-for-non-surgical-knee-osteoarthritis-relief/).
This advancement signifies a potential turning point in pain management. Current CIPN treatments are often ineffective and carry significant side effects, creating a substantial unmet medical need. A successful non-opioid treatment like ART26.12 could significantly improve the quality of life for cancer patients experiencing CIPN, allowing for better tolerance of chemotherapy and potentially improving treatment outcomes. Furthermore, the potential applications extend to other prevalent pain conditions, representing a significant market opportunity.
ART26.12 targets FABP5, an intracellular protein involved in lipid signaling. Preclinical studies have shown promising results across various pain models. The current Phase 1 single ascending dose study aims to determine appropriate doses for a subsequent multiple ascending dose study in healthy volunteers planned for the second half of 2025. This phased approach allows for careful evaluation of safety and pharmacokinetic properties before proceeding to larger trials.
The progress of ART26.12 represents a significant step forward for Artelo Biosciences and the field of pain management. Positive results from this Phase 1 trial would validate the FABP inhibition mechanism and could pave the way for further clinical development, potentially leading to a novel class of non-opioid pain therapeutics. The upcoming data readout in the first half of 2025 will be a critical inflection point, providing key insights into the future of this promising drug candidate.
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### [Ashvattha Therapeutics Secures $50M, Unveils Promising Phase 2 Ophthalmology Results](https://www.clinicaltrialvanguard.com/news/ashvattha-therapeutics-secures-50m-unveils-promising-phase-2-ophthalmology-results/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
Ashvattha Therapeutics secured up to $50 million in extended Series B funding, led by Tribe Capital and existing investors led by Natural Capital. This funding supports the continued development of [migaldendranib](https://www.clinicaltrialvanguard.com/news/ashvattha-therapeutics-positive-interim-phase-2-results-for-dme-and-wet-amd/) (MGB), a novel subcutaneous treatment for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD) and diabetic macular edema (DME), currently in Phase 2 trials. Interim results suggest MGB significantly reduces the need for supplemental aflibercept injections after an initial dose, while maintaining visual acuity and reducing subretinal fluid.
This development holds significant promise for patients suffering from wet AMD and DME, debilitating conditions affecting vision. Current treatments require frequent intravitreal injections, placing a considerable burden on patients and healthcare systems. MGB’s potential to reduce the frequency of these injections, while effectively managing the disease, could dramatically improve patients’ quality of life and offer a more practical treatment approach. This less invasive approach could increase treatment adherence, potentially leading to better long-term outcomes and reduced disease progression. Moreover, MGB’s subcutaneous administration makes at-home treatment feasible, further easing the burden on patients and potentially reducing healthcare costs associated with clinic visits.
The Phase 2 trial of MGB involves subcutaneous administration every 2 or 4 weeks for up to 40 weeks following a single intravitreal aflibercept injection. Data from patients who completed 24 weeks show a substantial decrease in required supplemental aflibercept compared to the 24 weeks preceding enrollment. Furthermore, the treatment has demonstrated a positive safety profile. These interim findings will be presented at the Angiogenesis 2025 meeting.
The successful completion of the Phase 2 trial and subsequent progression towards regulatory approval could revolutionize the treatment landscape for wet AMD and DME. A readily accessible and less invasive therapy like MGB could significantly expand treatment access, particularly for those with bilateral disease, a common occurrence in both conditions. This advancement may shift the standard of care towards at-home, patient-administered treatments, improving patient compliance and outcomes while streamlining healthcare delivery.
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### [Sage Therapeutics Unveils 2025 Strategic Focus at JPM Healthcare Conference](https://www.clinicaltrialvanguard.com/news/sage-therapeutics-unveils-2025-strategic-focus-at-jpm-healthcare-conference/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
Sage Therapeutics announced its 2025 strategic focus, centering on the growth of ZURZUVAE (zuranolone) for postpartum depression (PPD) and a recalibrated R&D approach focused on neurodevelopmental disorders and neuropsychiatry. The company expects substantial decreases in R&D and G&A expenses in 2025, extending their cash runway to mid-2027. This strategic shift follows the successful launch of ZURZUVAE, marking a turning point in PPD treatment.
This refocused strategy is crucial for addressing the significant unmet need in PPD treatment. ZURZUVAE offers a novel, convenient oral treatment option, potentially transforming the standard of care for this debilitating condition. The pipeline prioritization towards neurodevelopmental disorders and neuropsychiatry further positions Sage to address critical gaps in [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) care.
Sage plans to increase investment in ZURZUVAE, including expanding joint sales forces with Biogen and implementing digital marketing campaigns. This aims to drive market growth and increase revenue in 2025. Simultaneously, the company is advancing SAGE-319 for behavioral symptoms associated with neurodevelopmental disorders, with Phase 1 data expected by late 2025. They are also evaluating SAGE-324 for potential indications including seizures in developmental and epileptic encephalopathies.
This strategic realignment signals a new chapter for Sage. The company’s focus on ZURZUVAE’s market penetration, coupled with a streamlined pipeline and extended financial runway, creates a strong foundation for future growth and solidifies their commitment to addressing critical unmet needs in brain health. The anticipated decrease in operating expenses, driven by pipeline prioritization and cost savings from a 2024 reorganization, further strengthens their position for long-term success.
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### [Immunis Secures $25M in Series A-1 Funding](https://www.clinicaltrialvanguard.com/news/immunis-secures-25m-in-series-a-1-funding/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
Immunis, Inc., a clinical-stage biotech company, secured $25 million in Series A-1 funding to advance two Phase 2 clinical trials for its multi-active biologic targeting age and disease-related immune dysregulation. Existing investors, including Remiges Ventures, Continuum Health Ventures, and BOLD Capital Partners, participated in the round, alongside new investors like LifeSpan Vision Ventures and JLS Fund. The funding will support the clinical development of [IMM01](https://www.clinicaltrialvanguard.com/news/immunis-achieves-breakthrough-in-muscle-atrophy-trial/)-STEM, a stem cell-derived therapy aimed at mitigating age-related muscle loss and improving metabolic function.
This development is crucial because it addresses the significant unmet medical need of age-related decline in muscle mass and metabolic function, which contributes to decreased quality of life and increased healthcare costs. Successful clinical trials could establish IMM01-STEM as a valuable therapeutic option for elderly individuals struggling with sarcopenia and metabolic dysfunction, offering the potential to improve physical function, reduce pain, and enhance overall well-being.
The Phase 2 trials follow a positive Phase 1/2a study that demonstrated the safety and tolerability of IMM01-STEM in elderly patients with age-related muscle atrophy. Initial results also suggested improvements in quality of life, including physical function and pain reduction, as well as increased gait speed. Preclinical studies in mouse models further support the therapy’s potential, showing reversal of muscle atrophy, improved muscle function, enhanced metabolism, and decreased body and liver fat. The new funding will allow Immunis to conduct larger, more rigorous studies to confirm these findings in a human population.
This successful funding round positions Immunis to solidify its leadership in the development of stem cell-derived multi-active biologics. Positive Phase 2 results could pave the way for larger trials and eventual regulatory approval, ultimately bringing a novel therapeutic option to patients and potentially transforming the management of age-related decline.
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### [HMNC Brain Health Completes Patient Randomization for Phase 2b OLIVE Trial for Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/hmnc-brain-health-completes-patient-randomization-for-phase-2b-olive-trial-for-major-depressive-disorder/)
**Published:** January 14, 2025
**Author:** Jon Napitupulu
**Content:**
HMNC Brain Health has completed patient randomization for its Phase 2b OLIVE trial, investigating the efficacy and safety of [BH-200](https://www.clinicaltrialvanguard.com/news/new-analysis-supports-genetic-tool-for-depression-treatment/), a vasopressin V1b receptor antagonist, combined with a predictive companion diagnostic for [Major Depressive Disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD). The trial focuses on patients with HPA-axis dysfunction, representing roughly 30% of all MDD cases, and is the largest of its kind globally. This achievement signifies a major step in HMNC’s Nelivabon program, aimed at delivering personalized MDD treatments.
This development holds substantial promise for advancing the treatment of depression. The trial specifically targets a significant subset of MDD patients whose condition is rooted in HPA-axis dysfunction, a biological mechanism often resistant to conventional treatments. Successful results could offer a new therapeutic avenue for these patients, potentially leading to improved remission rates and quality of life. This precision medicine approach could also drive more efficient resource allocation in healthcare by targeting treatments to those most likely to benefit.
The OLIVE trial, encompassing 338 outpatients, uses a randomized, double-blind, placebo-controlled design. It evaluates the efficacy of BH-200 compared to placebo over an eight-week period, differentiating between patients with high and low values of a V1b polygenic score. This prospective-retrospective approach aligns with FDA guidance for companion diagnostic co-development. Initial trial results are anticipated in Q2 2025.
Positive results from the OLIVE trial could mark a turning point in MDD treatment, establishing BH-200 as a first-in-class therapy for patients with stress-axis-related depression. This would validate HMNC’s precision psychiatry platform and could pave the way for broader application of this approach in [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/). Furthermore, positive data will refine the companion diagnostic, increasing its predictive accuracy and further personalizing treatment strategies. The trial’s outcome is poised to significantly impact the landscape of mental healthcare and potentially redefine the standard of care for this prevalent and debilitating condition.
Source link:[ https://www.globenewswire.com/news-release/2025/01/10/3007736/0/en/HMNC-Brain-Health-Completes-Patient-Randomization-for-Phase-2b-OLIVE-Trial-for-Major-Depressive-Disorder.html](https://www.globenewswire.com/news-release/2025/01/10/3007736/0/en/HMNC-Brain-Health-Completes-Patient-Randomization-for-Phase-2b-OLIVE-Trial-for-Major-Depressive-Disorder.html)
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### [First Participants Randomized in AskBio Phase II Gene Therapy Trial for Parkinson's Disease](https://www.clinicaltrialvanguard.com/news/first-participants-randomized-in-askbio-phase-ii-gene-therapy-trial-for-parkinsons-disease/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
[AskBio](https://www.clinicaltrialvanguard.com/news/askbio-doses-first-limb-girdle-muscular-dystrophy-patient/), a Bayer subsidiary, has initiated a Phase II clinical trial, REGENERATE-PD, for its Parkinson’s disease gene therapy, AB-1005 (AAV2-GDNF). The trial will evaluate the safety and efficacy of AB-1005 in adults with moderate-stage Parkinson’s disease across clinical sites in the United States, Germany, Poland, and the United Kingdom. Positive 36-month Phase Ib data showcased a favorable safety profile and encouraging trends in clinical measures.
The commencement of the Phase II trial signifies a crucial step towards developing a potentially disease-modifying treatment for [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease. Current therapies primarily manage symptoms, but the progressive nature of the disease necessitates treatments that can slow or halt neuronal degeneration. AB-1005, by delivering the GDNF gene directly to the brain, aims to support the survival of dopamine-producing neurons, potentially altering the disease course and improving long-term outcomes for patients. This approach holds considerable promise for addressing the significant unmet need in Parkinson’s disease treatment.
REGENERATE-PD is a randomized, double-blind, sham-controlled trial that will enroll approximately 87 participants aged 45-75. The primary objective is to evaluate both the safety and efficacy of intraputaminal administration of AB-1005. Previously reported 36-month Phase Ib data indicated no serious adverse events related to AB-1005, and participants exhibited improvements or maintained stability in motor function assessments. AskBio is also investigating AB-1005 for multiple system atrophy, a related neurodegenerative disorder.
The advancement of AB-1005 into Phase II testing reinforces the potential of gene therapy to revolutionize Parkinson’s disease treatment. The results of this trial will be critical in determining the future development and potential regulatory approval of this promising therapeutic. Positive data could not only lead to a new treatment option for patients with moderate-stage Parkinson’s disease but also validate the approach of using GDNF gene therapy for neurodegenerative conditions, stimulating further research and development in this field.
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### [Pasithea Opens EU Clinical Trial Sites & Completes Cohort 4 Dosing](https://www.clinicaltrialvanguard.com/news/pasithea-opens-eu-clinical-trial-sites-completes-cohort-4-dosing/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Pasithea Therapeutics, a clinical-stage biotechnology company, has expanded its Phase 1 clinical trial of [PAS-004](https://www.clinicaltrialvanguard.com/news/pasitheas-nf1-drug-pas-004-shows-positive-pk-data-in-trial/), a next-generation MEK inhibitor, by opening three new sites in Eastern Europe. These new sites, located in Romania and Bulgaria, join four existing sites in the United States and are actively recruiting patients for the study, which is investigating PAS-004 as a potential treatment for neurofibromatosis type 1 (NF1) and other cancers. The company has also dosed the first three patients in Cohort 4A (15mg capsule) and is actively recruiting for Cohort 4B (4mg tablet), with interim safety and pharmacokinetic data expected in Q1 2025.
This expansion into Eastern Europe is strategically important for Pasithea. It broadens the patient pool, potentially accelerating recruitment and providing access to patients with tumor types that may be more responsive to single-agent MEK inhibitors or who have not responded to previous treatments. This approach allows for a more comprehensive assessment of PAS-004’s efficacy and safety profile across a wider range of patient demographics and disease characteristics, which is crucial for successful drug development.
The Phase 1 trial is designed as a multi-center, open-label, dose-escalation study (3+3 design). It aims to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of PAS-004 in patients with advanced solid tumors driven by the MAPK pathway. These patients will have documented RAS, NF1, or RAF mutations, or will have previously failed BRAF/MEK inhibitor treatment. The addition of new sites and the ongoing dosing of patients in Cohort 4 demonstrate Pasithea’s commitment to advancing the clinical development of PAS-004.
The expansion of the clinical trial and the anticipated release of interim data in Q1 2025 represent important milestones for Pasithea. These developments could significantly impact the future trajectory of PAS-004, potentially leading to faster clinical development and ultimately providing a much-needed new treatment option for patients with NF1 and other cancers. The data from these cohorts will be key in determining the optimal dose and informing the design of future clinical trials, potentially accelerating the path toward regulatory approval.
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### [Positive Clinical Data from Netherton Syndrome Study](https://www.clinicaltrialvanguard.com/news/positive-clinical-data-from-netherton-syndrome-study/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Quoin Pharmaceuticals (QNRX) announced positive interim data from its ongoing pediatric Netherton Syndrome clinical study of [QRX003](https://www.clinicaltrialvanguard.com/news/quoin-netherton-syndrome-study-shows-positive-9-month-data/), a topical treatment. The treatment showed significant skin improvement, prompting an expansion from a 20% body surface area application to whole-body application in the pediatric subject. Notably, no adverse events or safety concerns were reported, consistent with other ongoing Netherton Syndrome studies.
This progress is particularly encouraging for the rare disease community, as Netherton Syndrome currently has no approved treatments. The rapid and substantial improvement observed after just six weeks, moving from a “severe” to “mild” Investigator’s Global Assessment (IGA) score, suggests QRX003 could address a significant unmet medical need. The transition to whole-body application will provide more comprehensive data on both efficacy and safety, crucial for advancing toward potential regulatory approval.
The positive results in this pediatric study follow recently reported positive data from an open-label adult study, strengthening the overall picture of QRX003’s potential. The company is now planning to expand the pediatric study internationally and anticipates future pediatric subjects may also transition directly to whole-body testing.
These developments point to a promising future for QRX003. The accumulation of positive clinical data from both pediatric and adult studies builds momentum toward a potential New Drug Application (NDA) and offers hope for patients with this debilitating rare disease. The expansion of the study to additional pediatric patients internationally could further solidify the drug’s safety and efficacy profile, accelerating its path to market and ultimately bringing a much-needed treatment option to patients.
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### [Eterna Therapeutics ERNA-101 Shows Promise in Preclinical Ovarian Cancer Study](https://www.clinicaltrialvanguard.com/news/eterna-therapeutics-erna-101-shows-promise-in-preclinical-ovarian-cancer-study/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Eterna Therapeutics announced positive preclinical results for its lead [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) product, [ERNA-101](https://www.clinicaltrialvanguard.com/news/ernexa-therapeutics-advances-toward-2026-human-trials/), in treating ovarian cancer. The study showed increased T cell infiltration into the tumor, reduced tumor size, and improved survival rates in mice after a single dose of ERNA-101. The therapy successfully transitioned the tumor microenvironment from “cold” (low immune activity) to “hot” (high immune activity).
This preclinical success is a crucial advancement in ovarian cancer treatment, particularly for platinum-resistant cases where current options are limited. The ability of ERNA-101 to activate the immune system within the tumor itself offers a new approach, potentially overcoming the limitations of other immunotherapies. Furthermore, the demonstrated ability to convert a “cold” tumor “hot” suggests that ERNA-101 could be used in combination with other therapies like CAR-T and CAR-NK cell therapies to boost their effectiveness.
ERNA-101 utilizes induced pluripotent stem cells (iPSCs) to create induced allogenic mesenchymal stem cells (iMSCs) that secrete interleukins IL-7 and [IL-15](https://www.clinicaltrialvanguard.com/news/teva-celiac-drug-gets-fda-fast-track/). These iMSCs are designed to infiltrate the tumor and deliver these cytokines directly, increasing localized immune response while minimizing systemic toxicity. The study, led by researchers at MD Anderson Cancer Center, involved an ovarian tumor model in mice. Treatment with ERNA-101 led to significantly higher T cell infiltration, reduced tumor size, and improved survival compared to control groups. Importantly, no significant weight differences were observed between the treated and control groups, suggesting a favorable safety profile in this preclinical setting.
These findings pave the way for further clinical investigation of ERNA-101. The positive results could potentially lead to a new treatment paradigm for ovarian cancer, offering hope for patients who have limited options. The potential for combination therapies with ERNA-101 further expands its impact, possibly improving outcomes for a broader range of cancer patients.
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### [Context Therapeutics CTIM-76 Dosed in First Patient of Phase 1 Trial](https://www.clinicaltrialvanguard.com/news/context-therapeutics-ctim-76-dosed-in-first-patient-of-phase-1-trial/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Context Therapeutics has dosed the first patient in a Phase 1 clinical trial for [CTIM-76](https://www.clinicaltrialvanguard.com/news/context-therapeutics-doses-first-patient-in-phase-1-trial-of-ct-95/), a T cell engager bispecific antibody targeting Claudin 6 (CLDN6)-positive gynecologic and testicular cancers. The trial is designed to evaluate the safety and efficacy of CTIM-76, with initial data expected in the first half of 2026. This trial marks a significant step forward in the company’s clinical pipeline development.
This first-in-human trial of CTIM-76 is crucial for advancing the treatment of CLDN6-positive cancers. CLDN6 is often overexpressed in various solid tumors, offering a promising target for T cell engager therapies. Positive trial results could validate CLDN6 as a viable therapeutic target and potentially offer a new treatment option for patients with these difficult-to-treat cancers. This could pave the way for further development of CTIM-76 and establish Context Therapeutics as a leader in this specific therapeutic area.
The Phase 1 trial is an open-label study evaluating CTIM-76 in patients with advanced or metastatic ovarian, endometrial, and testicular cancers. The trial will assess safety, tolerability, pharmacokinetics, and anti-tumor activity. Up to 70 patients are expected to be enrolled in the dose-escalation and expansion phases. Preclinical research suggests the potential for convenient dosing, low immunogenicity risk, and scalable manufacturing for CTIM-76.
The initiation of this Phase 1 trial sets the stage for potentially significant progress in the development of CTIM-76. Successful results could lead to subsequent clinical trials and eventually, regulatory approval, providing a much-needed therapeutic option for patients with CLDN6-positive cancers. This trial also strengthens Context Therapeutics’ position in the field of T cell engager therapies for solid tumors.
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### [Bayer's Pharma Growth Strategy Advances Pipeline](https://www.clinicaltrialvanguard.com/news/bayers-pharma-growth-strategy-advances-pipeline/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Bayer AG announced progress in its pharmaceutical growth strategy at the 43rd J.P. Morgan Healthcare Conference, highlighting regulatory submissions for darolutamide, finerenone, and elinzanetant. These submissions represent key growth drivers for the company, strengthening its position in oncology, cardiology, and women’s health. The company also highlighted advancements in its cell and gene therapy platform and targeted investments in research and development.
This progress is crucial for Bayer as it signifies the potential expansion of existing drug indications and the introduction of new therapies to address unmet medical needs. The anticipated launch of darolutamide for a third indication in metastatic hormone-sensitive [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/), following successful trials, solidifies Bayer’s leadership in this therapeutic area. The submission for finerenone in heart failure expands the potential patient population for this drug, while positive Phase III results for elinzanetant offer a promising non-hormonal treatment option for women experiencing menopausal symptoms. Furthermore, advancements in cell and gene therapy, particularly in Parkinson’s disease, position Bayer at the forefront of innovative therapeutic approaches.
Darolutamide (NUBEQA) achieved blockbuster status, surpassing one billion euros in annual sales, and is the fastest-growing androgen receptor inhibitor in the U.S. Finerenone (KERENDIA) demonstrated a statistically significant reduction in cardiovascular death and heart failure events in a Phase III trial. Elinzanetant met primary and secondary endpoints in a Phase III study for the treatment of vasomotor symptoms, including hot flashes. In addition, Bayer is pursuing promising early-stage research in areas like precision oncology, targeted radionuclide therapy, and novel thrombolytic agents.
Bayer’s strategic focus on key therapeutic areas, coupled with its investments in innovative platforms and technologies, suggests a promising future for the company. The regulatory submissions and positive clinical trial results signal potential near-term revenue growth and market expansion. The advancements in cell and gene therapy, along with early-stage research programs, indicate a commitment to long-term growth and leadership in addressing unmet medical needs. This multifaceted approach positions Bayer for continued growth and innovation in the pharmaceutical industry.
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### [Umoja Biopharma Gets $100M Series C for In Vivo CAR T-cell Pipeline](https://www.clinicaltrialvanguard.com/news/umoja-biopharma-gets-100m-series-c-for-in-vivo-car-t-cell-pipeline/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Umoja Biopharma, a clinical-stage company developing •in vivo• cell therapies, secured $100 million in Series C funding. The round was co-led by Double Point Ventures and DCVC Bio, with participation from new and existing investors. This funding will support the clinical development of Umoja’s •in vivo• CAR T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) pipeline, particularly its lead program, UB-[VV400](https://www.clinicaltrialvanguard.com/news/fda-clears-ind-for-umojas-ub-vv400-in-vivo-car-t-therapy/), targeting CD22 in oncology and autoimmune diseases.
This investment underscores the growing interest in •in vivo• CAR T cell therapies, a potentially transformative approach to cancer and autoimmune disease treatment. Current •ex vivo• CAR T therapies involve complex and costly processes of extracting, modifying, and reinfusing a patient’s T cells. Umoja’s •in vivo• approach aims to simplify this process by directly modifying T cells within the patient’s body, potentially broadening access to this powerful therapeutic modality. This simplified approach could reduce treatment costs and manufacturing complexity while mitigating risks associated with current •ex vivo• methods like lymphodepleting chemotherapy and supply chain constraints. The successful funding round indicates confidence in Umoja’s ability to deliver on this promise and potentially revolutionize the CAR T cell therapy landscape.
The $100 million investment will primarily fuel the clinical development of Umoja’s pipeline, focusing on advancing UB-VV400 through multiple oncology and autoimmune disease studies. Dr. Campbell Murray from Double Point Ventures joins Umoja’s Board of Directors, bringing valuable expertise. Concurrently, Dr. Phil Low of Purdue University transitioned to Board Observer and Chair of Umoja’s Scientific Advisory Board.
This substantial investment positions Umoja to be a leader in the development of •in vivo• CAR T cell therapies. The upcoming clinical trials for UB-VV400 will be crucial in demonstrating the safety and efficacy of this approach. Positive clinical data could lead to rapid advancements in the field, opening up new treatment avenues for patients with cancer and autoimmune diseases and establishing Umoja as a key player in this emerging therapeutic area.
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**Categories:** News
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### [Glaukos iDose Shows Positive Clinical Results](https://www.clinicaltrialvanguard.com/news/glaukos-idose-shows-positive-clinical-results/)
**Published:** January 15, 2025
**Author:** Jon Napitupulu
**Content:**
Glaukos Corporation announced positive clinical updates for its iDose® sustained-release drug delivery platform for [glaucoma](https://www.clinicaltrialvanguard.com/news/nurexone-shows-vision-recovery-in-preclinical-glaucoma-model/) and ocular hypertension. The iDose platform offers a minimally invasive implant that delivers consistent medication within the eye, addressing patient non-adherence and side effects associated with traditional topical drops. The company is actively investing in expanding its pharmaceutical development capabilities based on the success of iDose TR.
This development is a significant advancement in glaucoma management. The iDose platform could shift the treatment paradigm by offering a more convenient and effective alternative to daily eye drops. This can improve patient compliance, leading to better long-term outcomes and potentially reducing the progression of these sight-threatening diseases. This is particularly crucial for glaucoma, a leading cause of blindness, where consistent medication is key to managing intraocular pressure.
The iDose TR implant is made of medical-grade titanium and releases a preservative-free form of travoprost continuously. This offers 24/7 drug delivery directly into the anterior chamber of the eye. Glaukos has already begun commercial launch activities for iDose TR in 2024. The company is also developing a robust pipeline of other dropless platform technologies for various chronic eye diseases.
This progress signifies a potential turning point in how chronic eye diseases are managed. The iDose platform’s focus on sustained drug delivery could not only improve patient lives through greater convenience and reduced side effects but also create new market opportunities for Glaukos. As the company continues to invest in its pharmaceutical development capabilities, we can expect further innovation in dropless therapies for various eye conditions.
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**Categories:** News
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### [FDA Update on ICH E6, E8, QbD and RBM in Clinical Trials](https://www.clinicaltrialvanguard.com/article/fda-update-on-ich-e6-e8-qbd-and-rbm-in-clinical-trials/)
**Published:** January 15, 2025
**Author:** Moe Alsumidaie
**Content:**
The recent FDA workshop, “[Building Quality into the Design and Conduct of Clinical Studies: Integrating Quality by Design (QbD) and Risk-Based Monitoring (RBM) Approaches,](https://healthpolicy.duke.edu/events/building-quality-design-and-conduct-clinical-studies-integrating-quality-design-qbd-and-risk "Building Quality into the Design and Conduct of Clinical Studies: Integrating Quality by Design (QbD) and Risk-Based Monitoring (RBM) Approaches,")” brought together industry leaders, regulators, and stakeholders to discuss the integration of QbD and RBM in clinical trials. This event, co-convened by the Duke Margolis Center for Health Policy, emphasized the evolving landscape of clinical research, focusing on the importance of quality, integrity, and efficiency. The workshop aimed to provide a framework for improving the design and execution of clinical trials, ensuring they are both efficient and effective in meeting drug development challenges. Throughout this article, Dawn Niccum, Executive Vice President, QA and Compliance at [inSeption Group](https://inseptiongroup.com/ "inSeption Group") will reflect on the workshop by adding her expert perspective.
[](https://inseptiongroup.com/services/?utm_campaign=moe-1&utm_medium=article&utm_source=linkedin)
## [](#emphasizing-quality-and-efficiency-in-clinical-trials)Emphasizing Quality and Efficiency in Clinical Trials
The workshop placed a strong emphasis on the integration of QbD and RBM as pivotal methodologies for the modernization of clinical trials. Dr. Mark McClellan, representing the Duke Margolis Center for Health Policy, articulated the necessity of these approaches in enhancing the efficiency and effectiveness of clinical trials. He explained that QbD and RBM are not merely theoretical concepts but practical tools that facilitate the efficient management of risks inherent in clinical trials. These methodologies ensure that trials maintain high data integrity and participant safety standards by embedding quality throughout the clinical development lifecycle.
Applying QbD in clinical trials draws inspiration from best practices in manufacturing, where quality is built into the process from the outset. This approach, championed by leaders such as Janet Woodcock and her advocacy for QbD, stems from her belief that clinical trials should be designed with a clear focus on quality, leading to better outcomes and reduced costs. The workshop highlighted several case studies where QbD principles were successfully implemented, demonstrating tangible improvements in trial efficiency and data quality. For instance, one case study involved a pharmaceutical company that applied QbD principles to streamline its trial processes. By identifying critical-to-quality factors early in the design phase, the company was able to focus its resources on the most critical aspects of the trial, thereby reducing unnecessary complexity and cost. Another example illustrated how a clinical research organization used risk-based monitoring to identify potential issues early in the trial process, allowing for timely interventions that ensured data integrity.
> Thoughts by Dawn Niccum, Executive Vice President, QA and Compliance at [inSeption Group](https://inseptiongroup.com/ "inSeption Group")
>
>
>
>
>
> “*The true value of QbD and RBM lies in their ability to anticipate and mitigate risks before they become issues. By identifying critical quality factors early in the process, sponsors ensure compliance and enhance the efficiency of trial execution. It’s about shifting from reactive problem-solving to proactive quality management, ultimately leading to better outcomes for patients and sponsors.*”
These examples emphasize the potential of QbD and RBM to transform clinical trials, making them more efficient and effective. By focusing on these methodologies, the workshop provided valuable insights into how clinical trials can be improved to meet the evolving needs of the healthcare industry.
[](https://inseptiongroup.com/services/?utm_campaign=moe-1&utm_medium=article&utm_source=linkedin)
## [](#incorporating-patient-voices-in-clinical-trial-design-and-decentralized-trials)Incorporating Patient Voices in Clinical Trial Design and Decentralized Trials
A key theme of the workshop was the importance of incorporating patient perspectives into clinical trial design. Dr. Janet Woodcock emphasized the importance of patient input, noting that it can significantly enhance participant enrollment and retention. By involving patients in the planning stages of clinical trials, sponsors can design studies that are more aligned with participants’ needs and preferences, ultimately driving both quality and success. For example, one patient advocate highlighted the importance of flexible scheduling and remote participation options, making trials more accessible to more participants. Another advocate emphasized the need for clear communication and transparency throughout the trial process, which can help build trust and encourage continued participation.
> Thoughts by Dawn Niccum
>
> “*Integrating patient perspectives into the design phase is no longer optional—it’s essential. Decentralized trials have shown us that you increase retention and engagement when you prioritize the patient’s experience, from scheduling flexibility to remote participation. It’s about designing trials that work for patients, not just for sponsors, and that shift is key to driving the success of modern clinical trials.*”
There was also discussion on decentralized clinical trials. The [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic accelerated the adoption of decentralized trial elements, such as remote monitoring and virtual visits, showing great potential to make trials more accessible and efficient. For instance, one trial used telemedicine to conduct virtual visits, allowing participants to remain in the study without frequent travel to clinical sites. Another trial employed remote monitoring technologies to collect real-time participant data, providing researchers with a comprehensive view of the trial’s progress.
The workshop highlighted that integrating patient perspectives and decentralized elements into clinical trial design enhances data quality by fostering greater participant engagement, accessibility, and real-time data collection, ultimately aligning studies more closely with participants’ needs and preferences.
[](https://inseptiongroup.com/services/?utm_campaign=moe-1&utm_medium=article&utm_source=linkedin)
## [](#ich-e6-and-e8-clinical-trial-frameworks-and-harmonizing-global-guidelines)ICH E6 and E8 Clinical Trial Frameworks and Harmonizing Global Guidelines
Discussions delved into the specifics of the ICH E6 and E8 guidelines, with the ICH E6 providing a framework for designing trials that are fit for purpose, ensuring that resources are concentrated on critical study aspects while reducing costs, and maintaining high data integrity and participant safety standards. The ICH E8 guideline, on the other hand, emphasizes the importance of a risk-based approach to trial design and conduct. By identifying and managing risks early in the process, sponsors can ensure that trials are conducted to maximize efficiency and minimize potential issues. For instance, one case study involved a multinational pharmaceutical company that applied the ICH E6 and E8 guidelines to a complex global trial. By focusing on critical-to-quality factors and adopting a risk-based approach, the company was able to streamline its trial processes and improve data quality. This approach reduced the trial’s overall cost and accelerated the timeline, allowing the company to bring its product to market more quickly.
> Thoughts by Dawn Niccum
>
> *“The ICH E6 and E8 guidelines are about refining clinical trials to focus on what truly matters—data integrity and participant safety. By applying these frameworks, sponsors can concentrate resources on the most critical aspects of their trials while minimizing inefficiencies. The real benefit comes with harmonizing these guidelines globally, allowing sponsors to reduce the complexities of meeting different regulatory requirements across countries, ultimately leading to faster and more consistent trial outcomes.”*
The workshop also emphasized the global harmonization of good clinical practice guidelines, particularly regarding the recently published draft versions of ICH E6 and E8, designed to streamline clinical trial processes and improve outcomes. The workshop featured several case studies where these guidelines were successfully implemented, demonstrating their potential to transform clinical trial processes. For example, a representative from a global pharmaceutical company discussed how harmonized guidelines enabled them to conduct trials more efficiently across multiple countries, reducing the time and cost associated with meeting different regulatory requirements. Similarly, a regulator highlighted the benefits of harmonization in improving trial data quality and consistency, which is essential for making informed decisions about new treatments.
## [](#inspection-readiness)Inspection Readiness
Regulatory perspectives on inspection readiness and risk-based approaches were a key focus of the workshop. Jacqueline Corrigan-Curay, Principal Deputy Center Director in CDER, discussed the importance of integrating quality management principles into the design and conduct of clinical investigations. She emphasized the need for study-specific monitoring plans and a dynamic approach to continual improvement. The workshop featured presentations from regulatory experts and industry leaders, who shared insights on applying these principles effectively.
For example, one presentation discussed the role of technology in enhancing inspection readiness. By leveraging advanced data analytics and monitoring tools, sponsors can gain real-time insights into trial performance and identify potential issues before they become significant problems. This proactive approach improves the quality of the data collected and enhances the overall efficiency of the trial process.
> Thoughts by Dawn Niccum
>
> *“Inspection readiness isn’t about having perfect trials; it’s about ensuring that your trial processes are robust and transparent. A well-crafted risk management plan ensures that the focus stays on the critical aspects of the trial, reducing unnecessary burden while maintaining high-quality data and regulatory compliance. Technology plays a vital role here by providing real-time oversight that helps sponsors identify and address issues before they become major problems.”*
The discussions emphasized the importance of a proportionate and risk-based approach to quality management tailored to the specific needs of each study. Sponsors can design more efficient, cost-effective, and likely-to-succeed trials by focusing on what matters most.
[](https://inseptiongroup.com/services/?utm_campaign=moe-1&utm_medium=article&utm_source=linkedin)
## [](#challenges-and-future-directions)Challenges and Future Directions
Despite progress in integrating QbD and RBM approaches, challenges remain in fully implementing these methodologies. One primary challenge is the need for continued education and confidence-building among sponsors and regulators. Many stakeholders are accustomed to traditional trial processes and may hesitate to adopt new approaches without clear evidence of their benefits. The workshop provided a platform for discussing these challenges and exploring potential solutions, emphasizing the importance of training and resources to help stakeholders understand and implement QbD and RBM approaches.
The workshop highlighted the need for ongoing collaboration between industry, regulators, and other stakeholders to address barriers to adoption and ensure these modern practices are widely accepted and implemented. The FDA and Duke Margolis are committed to fostering an environment encourages innovation and efficiency in clinical trials. By continuing to promote QbD and RBM approaches, they aim to improve research quality and efficiency, ultimately leading to better patient treatments.
## [](#conclusion)Conclusion
The FDA workshop was a platform for a robust discussion on modernizing clinical trials through QbD and RBM approaches. The event aimed to empower the clinical trial community to adopt these methodologies by sharing case studies and best practices, leading to more efficient, cost-effective, and high-quality research. As the clinical research landscape evolves, integrating these approaches will be crucial in meeting drug development challenges and delivering better patient treatments.
*This article is sponsored by [InSeption Group](https://inseptiongroup.com/ "InSeption Group").*
**Categories:** Article
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### [SIL-204 Shows Synergy with Chemotherapies in Preclinical Pancreatic Cancer Study](https://www.clinicaltrialvanguard.com/news/sil-204-shows-synergy-with-chemotherapies-in-preclinical-pancreatic-cancer-study/)
**Published:** January 16, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) (NASDAQ: SLXN) announced promising preclinical results for [SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/), its second-generation siRNA therapy targeting KRAS-mutated cancers. The data show SIL-204 acts synergistically with standard chemotherapy agents like 5-fluorouracil, irinotecan, and gemcitabine, significantly reducing cancer cell confluence in KRAS G12D mutated pancreatic cancer cell lines. This builds on earlier successes with Silexion’s first-generation product, LODER™, which improved overall survival in Phase 2 trials.
These findings are particularly important because pancreatic cancer, especially with the prevalent KRAS G12D mutation, is notoriously difficult to treat and has a high mortality rate. The demonstrated synergy between SIL-204 and existing chemotherapy regimens suggests the potential for improved treatment efficacy and patient outcomes, addressing a critical unmet need. The positive results with SIL-204 also expand Silexion’s pipeline beyond LODER™ and broaden its potential impact across multiple KRAS-mutated cancers beyond pancreatic cancer.
Preclinical studies showed a statistically significant reduction in cancer cell confluence after just three days of combined treatment with SIL-204 and either the 5-fluorouracil/irinotecan combination or gemcitabine alone, compared to chemotherapy alone (p < 0.0005). Silexion is preparing to initiate toxicology studies with SIL-204 in the coming months, with plans to advance it to Phase 2/3 clinical trials in the first half of 2026, initially focusing on locally advanced pancreatic cancer. Concurrent preclinical studies are also planned for SIL-204 in [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) models. These developments position Silexion to potentially change the treatment paradigm for KRAS-driven cancers. The upcoming clinical trials for SIL-204 will be crucial in validating these preclinical findings and determining the true clinical benefit for patients. The parallel investigation in colorectal cancer further expands the potential scope of SIL-204’s therapeutic impact. The positive preclinical results combined with planned clinical development suggest a promising future for Silexion and provide a potential new avenue of treatment for patients facing these challenging cancers.
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**Tags:** eClinical Tech News
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### [Solid Biosciences: Advancing Neuromuscular and Cardiac Gene Therapy](https://www.clinicaltrialvanguard.com/news/solid-biosciences-advancing-neuromuscular-and-cardiac-gene-therapy/)
**Published:** January 16, 2025
**Author:** Jon Napitupulu
**Content:**
Solid Biosciences provided a corporate update highlighting its expanded clinical pipeline and 2025 objectives, focusing on genetic medicines for neuromuscular and cardiac diseases. The company announced progress in its [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/) (Duchenne), Friedreich’s ataxia (FA), and Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) programs, and confirmed a strong cash position. This progress marks Solid Biosciences’ transition into a multi-program company developing precision genetic medicines.
This news is important because it demonstrates Solid Biosciences’ commitment to diversifying its pipeline beyond Duchenne, addressing other significant unmet needs in neuromuscular and cardiac diseases. The advancement of multiple programs into clinical stages signals potential future revenue streams and strengthens the company’s position in the genetic medicines field. Specifically, the dual route of administration for [SGT-212](https://www.clinicaltrialvanguard.com/news/solid-biosciences-announces-fda-ind-clearance-for-first-in-industry-dual-route-of-administration-gene-therapy-to-treat-both-neurologic-and-cardiac-manifestations-of-friedreichs-ataxia/) in FA represents a novel approach with potential to improve treatment efficacy.
Solid Biosciences dosed the first four patients in the INSPIRE [DUCHENNE](https://www.clinicaltrialvanguard.com/news/solid-biosciences-duchenne-trial-sees-positive-interim-update/) trial for SGT-003 with no serious adverse events reported. Initial data from three patients is expected in Q1 2025. The FDA cleared the Investigational New Drug (IND) application for SGT-212 for FA, paving the way for a first-in-human trial in 2H 2025. An IND submission for SGT-501 for CPVT is on track for 1H 2025. The company ended 2024 with $148.9 million in cash and investments, anticipated to fund operations into 2026.
The advancements across Solid Biosciences’ pipeline signify a promising outlook for the company. Data readouts in 2025 for the Duchenne and CPVT programs will be crucial inflection points, potentially validating the company’s platform and driving further investment. The initiation of the first-in-human trial for SGT-212 will mark a significant step in addressing the unmet needs of FA patients. These developments, combined with a healthy cash runway, position Solid Biosciences for continued growth and innovation in the genetic medicines space.
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**Tags:** eClinical Tech News
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### [ImmunityBio's 2025 FDA Submissions After Agency Meeting](https://www.clinicaltrialvanguard.com/news/immunitybios-2025-fda-submissions-after-agency-meeting/)
**Published:** January 16, 2025
**Author:** Jon Napitupulu
**Content:**
ImmunityBio announced progress in discussions with the FDA concerning its non-muscle invasive bladder cancer (NMIBC) and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) treatments. The company anticipates submitting applications for an alternative BCG source, expanding ANKTIVA’s use in NMIBC, and using ANKTIVA in combination with checkpoint inhibitors for NSCLC. These advancements aim to address significant unmet needs in cancer treatment by offering potentially less toxic and more effective options.
This progress is particularly important for patients facing limited treatment choices and potentially serious outcomes. The potential expansion of ANKTIVA’s use in NMIBC could benefit patients with papillary disease who might otherwise face radical cystectomy. Additionally, the combination therapy for NSCLC could offer a new lifeline for patients who have progressed on existing checkpoint inhibitors, potentially prolonging survival without the harsh side effects of chemotherapy. Securing an alternative BCG source could stabilize supply and ensure broader access to a vital component of bladder cancer treatment.
ImmunityBio is preparing a supplemental [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (sBLA) in 2025 for ANKTIVA in BCG-unresponsive NMIBC with papillary tumors. A Phase 2b study showed promising results for ANKTIVA combined with checkpoint inhibitors in NSCLC patients, with a median overall survival of 14.1 months. A BLA for this combination therapy is also planned for 2025. Collaborating with the Serum Institute of India, ImmunityBio aims to submit an application for an alternative BCG source in the first quarter of 2025.
These developments position ImmunityBio to significantly impact the cancer treatment landscape. Positive regulatory outcomes could lead to new treatment paradigms for both NMIBC and NSCLC, providing patients with more effective and tolerable options. Furthermore, a stable BCG supply would be a crucial step towards ensuring consistent access to vital bladder cancer therapies.
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**Categories:** News
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### [Scaling Cell & Gene Therapies Globally: CGT & Cellex Partnership](https://www.clinicaltrialvanguard.com/news/scaling-cell-gene-therapies-globally-cgt-cellex-partnership/)
**Published:** January 16, 2025
**Author:** Jon Napitupulu
**Content:**
CGT Global, a US-based cell collection and expansion company, and Cellex Cell Professionals, a German [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) manufacturer, have partnered to improve scalability and patient access to cell and gene therapies (CGT). This collaboration leverages CGT Global’s US clinic and laboratory network and Cellex’s expertise in cell product manufacturing, particularly CAR-T therapies. The partnership aims to address the growing global demand for CGT while reducing costs and improving treatment accessibility.
This partnership is vital due to the increasing need for CGT manufacturing capabilities to support the growing number of therapies entering clinical trials and reaching commercialization. The combined expertise and resources of these two companies create a more efficient pathway from development to patient delivery, potentially accelerating the availability of life-saving treatments and addressing the current bottleneck in CGT manufacturing. This will benefit pharmaceutical companies developing these therapies and, ultimately, the patients who need them.
This transatlantic partnership leverages CGT Global’s experience in scaling during the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, providing testing services to a wide range of organizations and expanding their infrastructure. Cellex brings its established European presence and experience in CAR-T manufacturing, serving a significant portion of the commercial CAR-T market in Europe. By combining their resources and expertise, they aim to streamline the entire CGT process, from cell collection to manufacturing and distribution, ultimately leading to more cost-effective treatments.
This collaboration represents a significant step toward a more integrated and efficient global CGT market. It could lead to faster commercialization of new therapies, wider patient access, and potentially lower treatment costs. The strengthened transatlantic connection between the two companies may also foster further innovation and collaboration within the CGT field, ultimately accelerating the development and delivery of these potentially life-saving treatments to patients worldwide.
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**Categories:** News
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### [Complete 12-Week Results of NORSE Eight Trial for Outlook Therapeutics®](https://www.clinicaltrialvanguard.com/news/complete-12-week-results-of-norse-eight-trial-for-outlook-therapeutics/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
Outlook Therapeutics announced positive 12-week results from its NORSE EIGHT trial for ONS-5010, an ophthalmic bevacizumab formulation for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD). The treatment demonstrated non-inferiority to ranibizumab (Lucentis) at 12 weeks, paving the way for a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) resubmission in Q1 2025. The company also secured funding of up to $20.4 million through a warrant inducement transaction.
These 12-week results are crucial as they follow an earlier setback at the 8-week mark, where ONS-5010 did not meet the pre-specified non-inferiority endpoint. The continued improvement in visual acuity up to 12 weeks strengthens the argument for ONS-5010’s efficacy and provides more robust data for the upcoming BLA resubmission. A potential FDA approval for ONS-5010 offers a significant opportunity to address the current reliance on off-label, repackaged bevacizumab for wet AMD treatment, providing a regulated and potentially more cost-effective alternative. This could disrupt the existing market by offering a readily available, approved formulation, potentially impacting both patient access and the market share of current treatments.
In NORSE EIGHT, ONS-5010 achieved a mean improvement of 5.5 letters in best corrected visual acuity (BCVA) at 12 weeks compared to 6.5 letters with ranibizumab. The change in central retinal thickness, a key anatomical indicator, was also comparable between the two treatment groups. Furthermore, ONS-5010 exhibited a positive safety profile, with ocular adverse event rates similar to ranibizumab and no reported cases of retinal vasculitis. The $20.4 million raised from the warrant transaction will support the BLA resubmission, the European launch of [LYTENAVA](https://www.clinicaltrialvanguard.com/news/fda-approves-lytenava-first-ophthalmic-bevacizumab-for-wet-amd/)™ (the approved name for ONS-5010 in the EU and UK), ongoing clinical programs, and general corporate operations. The European launch, anticipated in the first half of 2025, positions Outlook Therapeutics to establish a market presence while awaiting US regulatory decisions.
The positive 12-week data and planned BLA resubmission represent a pivotal moment for Outlook Therapeutics. A successful FDA approval would validate ONS-5010 as a viable treatment option for wet AMD, offering a standardized and approved alternative to current off-label bevacizumab use. This would not only benefit patients but also potentially reshape the treatment landscape for this prevalent retinal disease. The combined momentum of the upcoming BLA resubmission and the European launch sets the stage for significant growth and market penetration, pending regulatory outcomes.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Micron Biomedical Secures $33M for Needle-Free Delivery](https://www.clinicaltrialvanguard.com/news/micron-biomedical-secures-33m-for-needle-free-delivery/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
Micron Biomedical secured $16 million in a Series A3 funding round, bringing the total Series A investment to over $33 million. This funding, led by J2 Ventures and the Global Health Investment Corporation (GHIC), will expand the commercial manufacturing of Micron’s needle-free drug and vaccine delivery technology based on dissolvable microarrays. The company recently received grants from CEPI, [BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/), and the Bill & Melinda Gates Foundation for developing needle-free vaccines for various diseases, including Disease X, influenza, and measles/rubella.
This investment significantly advances Micron Biomedical’s capacity to address pressing global health challenges by improving access to essential medications and vaccines. The needle-free technology offers a solution for populations with limited access to healthcare professionals, simplifies administration, and improves patient comfort. This is especially relevant for global health initiatives, military applications, and emergency response scenarios. Thermostable properties also remove the need for complex cold chain logistics, significantly broadening the reach of life-saving medications, especially in resource-limited settings.
This funding round builds upon substantial financial backing from prominent institutions, highlighting the growing recognition of Micron’s technology’s potential. The expansion of commercial manufacturing capabilities will enable the company to scale production and move closer to widespread distribution of its products. Dr. Matt Goldman, General Partner at J2 Ventures and former Chief Medical Officer of the Defense Innovation Unit, joins Micron’s board, bringing valuable expertise in both military and civilian medical applications.
This latest funding round represents a crucial step toward making needle-free drug and vaccine administration a reality. It paves the way for increased production, broader distribution, and ultimately, greater accessibility to critical medications and vaccines worldwide. The addition of Dr. Goldman to the board signals a strategic focus on expanding into both civilian and military markets, further solidifying Micron’s potential for long-term growth and impact.
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**Categories:** News
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### [Omeros Announces Narsoplimab Trial Endpoint Update](https://www.clinicaltrialvanguard.com/news/omeros-announces-narsoplimab-trial-endpoint-update/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
Omeros Corporation announced positive sensitivity analysis results for [narsoplimab](https://www.clinicaltrialvanguard.com/news/omeros-corp-bla-resubmission-update/), its monoclonal antibody targeting the lectin pathway of complement, in treating hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). An independent statistical group conducted the analyses, confirming the robustness of previously reported primary endpoint data showing narsoplimab’s survival benefit in TA-TMA patients. The sensitivity analyses consistently demonstrated a significant reduction in mortality risk, with hazard ratios ranging from 0.24 to 0.42 and p-values ranging from less than 0.00001 to 0.0124.
This confirmation of narsoplimab’s efficacy is crucial for patients facing TA-TMA, a life-threatening complication with limited treatment options and high mortality rates. A therapy that demonstrably improves survival could drastically change the prognosis for these patients, potentially reducing long-term complications and improving quality of life. This also represents a critical step towards addressing an unmet medical need in the post-transplant setting.
The independent analysis considered various factors, including different time points (100 days, 1 year, 2 years) and risk factor stratification. Despite these variations, the analyses consistently revealed narsoplimab’s positive impact on survival, underscoring the reliability of the initial findings. The original primary endpoint analysis, reported in December 2024, showed a hazard ratio of 0.32, indicating a more than three-fold reduction in mortality risk for narsoplimab-treated patients compared to the control group. The new data strengthens these conclusions.
Omeros anticipates further analyses incorporating data from their expanded access program, expected soon. The company is moving forward with plans to resubmit a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) to the FDA later this quarter and a European Marketing Authorization Application (MAA) by mid-year 2025. These positive results pave the way for potential regulatory approval and the introduction of a much-needed treatment for TA-TMA, offering a new hope for patients undergoing hematopoietic stem cell transplantation.
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**Categories:** News
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### [Rubedo Life Sciences Announces Clinical Development Plans for Lead Candidate RLS-1496](https://www.clinicaltrialvanguard.com/news/rubedo-life-sciences-announces-clinical-development-plans-for-lead-candidate-rls-1496/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
Rubedo [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) announced clinical development plans for RLS-1496, a first-in-class GPX4 modulator targeting aging cells, at the 12th Annual Dermatology Summit. The company also initiated its Series B financing round and highlighted the potential of the longevity market, estimated at $600 billion. Phase 1 clinical trials for RLS-1496 are slated to begin in Spring 2025 in the Netherlands.
This development is notable because it marks a significant step towards addressing the growing global aging population and its associated health challenges. With a rapidly expanding elderly demographic, the demand for effective treatments targeting age-related diseases is increasing dramatically. Rubedo’s focus on developing a first-in-class therapy positions them at the forefront of this emerging field, offering a potential solution for a vast unmet medical need. Success in this area could revolutionize how we approach age-related health issues, shifting the focus from managing individual diseases to addressing the underlying cellular mechanisms of aging.
Rubedo’s RLS-1496 targets GPX4, a protein implicated in cellular aging. The company’s AI-driven drug discovery platform, ALEMBIC™, allows for the identification and development of novel small molecules aimed at senescent cells, which play a critical role in various chronic diseases. The upcoming Phase 1 trial will provide crucial data on the safety and efficacy of RLS-1496, paving the way for further clinical development. The commencement of Series B funding suggests investor confidence in Rubedo’s approach and technology, providing the necessary resources to advance their research and development efforts.
The initiation of clinical trials for RLS-1496 signifies a critical milestone for Rubedo and the broader field of longevity science. Positive results from the upcoming trials could validate their approach and open the door for developing a new class of therapeutics targeting age-related diseases. This holds the potential to significantly impact the healthcare landscape, offering hope for longer, healthier lives and potentially transforming how we manage chronic illnesses associated with aging.
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**Categories:** News
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### [Silexion's SIL-204 Shows Synergy Against KRAS-Driven Cancers](https://www.clinicaltrialvanguard.com/news/silexions-sil-204-shows-synergy-against-kras-driven-cancers/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) (NASDAQ: SLXN), a clinical-stage biotech company, has released promising preclinical data for [SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/), its next-generation RNA interference (RNAi) therapy targeting KRAS-driven cancers, including [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/). The data demonstrates SIL-204 synergizes with standard chemotherapy agents, significantly enhancing their efficacy against KRAS-mutated tumor cells. This advancement builds upon the success of Silexion’s first-generation LODER™ platform, which showed improved overall survival in Phase 2 trials.
This development holds substantial promise for patients battling KRAS-driven cancers, which are notoriously aggressive and resistant to treatment. Current therapies often prove ineffective, highlighting the urgent need for innovative approaches. SIL-204’s ability to amplify the effectiveness of existing chemotherapy regimens could significantly improve treatment outcomes and potentially extend survival for these patients, particularly those with pancreatic cancer where KRAS mutations are prevalent. The broader spectrum of KRAS mutations targeted by SIL-204 compared to its predecessor expands the potential patient population that could benefit.
SIL-204 enhances the efficacy of established chemotherapy drugs like 5-fluorouracil, irinotecan, and gemcitabine in preclinical models. This suggests the potential for combination therapies that could significantly improve treatment response rates. Silexion plans to initiate toxicology studies shortly, with Phase 2/3 trials projected for 2026. A recent $5 million public offering aims to fund these development efforts, providing the necessary resources to advance SIL-204 through the clinical trial process.
SIL-204’s preclinical success suggests a potential paradigm shift in the treatment of KRAS-driven cancers. If clinical trials validate these findings, SIL-204 could become a cornerstone of future treatment regimens, offering hope for improved survival and quality of life for patients facing these challenging diseases. This positions Silexion as a key player in the ongoing effort to develop more effective and targeted cancer therapies.
Source link: http://www.businesswire.com/news/home/20250116874895/en/PESG-Market-Update-Silexion-Therapeutics%E2%80%99-SIL-204-Shows-Groundbreaking-Synergy-in-Preclinical-Data-Boosting-Hope-for-KRAS-Driven-Cancer-Treatments
**Categories:** News
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### [Shionogi's S-892216: Breakthrough COVID-19 Antiviral for At-Risk Groups](https://www.clinicaltrialvanguard.com/news/shionogis-s-892216-breakthrough-covid-19-antiviral-for-at-risk-groups/)
**Published:** January 17, 2025
**Author:** Jon Napitupulu
**Content:**
Shionogi Inc. secured a $375 million agreement with the Biomedical Advanced Research and Development Authority ([BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)) to develop S-892216, a long-acting injectable 3CL protease inhibitor for [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pre-exposure prophylaxis. This funding, awarded through the Rapid Response Partnership Vehicle (RRPV), aims to address the need for preventative treatments that offer sustained protection against severe COVID-19. Shionogi plans to initiate Phase 1 studies in the US this year following an investigational new drug application.
This development is crucial because it signals continued investment in COVID-19 countermeasures despite the shift away from pandemic-era restrictions. The focus on pre-exposure prophylaxis highlights the recognition that COVID-19 remains a persistent global health concern and that long-term strategies are needed to mitigate its impact, particularly for vulnerable populations. Developing a long-acting injectable prophylactic could significantly improve adherence and offer more durable protection compared to existing options. This is a critical step toward ensuring long-term preparedness for potential future outbreaks or endemic circulation of the virus.
The agreement provides Shionogi with substantial funding to advance the development of S-892216. The selection of a 3CL protease inhibitor, a class of antivirals that has shown promise against COVID-19, suggests a targeted approach toward developing a highly effective prophylactic. The planned Phase 1 studies will provide crucial data on the safety and pharmacokinetic profile of the injectable formulation, paving the way for larger clinical trials.
This agreement marks a significant step forward in the ongoing fight against COVID-19. The successful development of a long-acting injectable prophylactic could be a game-changer in managing the long-term impact of the virus. It offers a potential solution for individuals at higher risk of infection or severe disease, and it could contribute to broader public health strategies aimed at preventing future surges and protecting vulnerable communities. Further research and clinical data will be essential in assessing the efficacy and real-world impact of S-892216 as a pre-exposure prophylaxis strategy.
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**Categories:** News
---
### [Abbott's Roadmap to Diversifying Clinical Trials](https://www.clinicaltrialvanguard.com/executiveinterviews/abbotts-roadmap-to-diversifying-clinical-trials/)
**Published:** January 20, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, Jennifer Jones-McMeans, Ph.D., co-leader of Abbott’s Diversity in Research Office and divisional vice president of global clinical affairs at Abbott’s vascular business, discusses the company’s initiatives to diversify clinical trials. She shares insights into overcoming systemic barriers, ensuring transparency, and fostering inclusivity in clinical research. This conversation highlights Abbott’s commitment to addressing challenges faced by underrepresented populations and creating a more equitable research environment.
## [](#moe-how-does-abbotts-initiative-address-systemic-barriers-like-socioeconomic-disparities)**Moe: How does Abbott’s initiative address systemic barriers like socioeconomic disparities?**
**Jennifer Jones-McMeans:** At Abbott, we tackle systemic barriers through our Diversity in Research Office, which I co-lead. In 2024, we launched the Abbott [Diversity in Research website](https://www.abbott.com/clinical-trials.html), an educational platform for patients, families, physicians, and researchers. This site is a central hub for information on diversity and inclusion in clinical trials. We identified four key barriers: lack of trust, transparency, access, and common language. To address these barriers, we’ve been intentional with our clinical trial site selection, collaborating with diverse research centers and supporting diverse staff training. For example, we’ve expanded our view to include more naive trial sites with diverse populations and provided training for research coordinators to reflect the patient population better.
Jennifer Jones-McMeans, Ph.D., co-leader of Abbott’s Diversity in Research Office
Education is crucial, so we ensure a foundational understanding of diseases and clinical trials, exemplified by our LIFE-BTK trial, where we communicated the disease and trial objectives. Additionally, remote monitoring and language translation are vital to overcoming these barriers, ensuring patients can participate without undue burden. We aim to create a more inclusive and equitable clinical trial environment by addressing these systemic barriers.
## [](#moe-what-metrics-does-abbott-use-to-measure-success-and-how-is-transparency-ensured)**Moe: What metrics does Abbott use to measure success, and how is transparency ensured?**
**Jennifer Jones-McMeans:** We employ a robust data collection system across Abbott to measure the success of our diversity initiatives. This system gathers comprehensive demographic and socioeconomic information, including gender, race, ethnicity, gender identity, and sexual identity. By maintaining a central database, we can track our progress year-to-year and compare it against reliable measures like the census. For instance, in 2023, female participation in our clinical trials was 54%, and the Hispanic non-white population accounted for 44%. These metrics are reported through our 2030 sustainability initiative, ensuring transparency and accountability.
This centralized approach allows us to assess our performance across different business units, such as cardiovascular, diagnostics, and nutrition, and align our efforts with broader sustainability goals. By having a clear and transparent system in place, we can ensure that our diversity initiatives are effective and that we are accountable for our progress.
## [](#moe-how-does-abbott-ensure-leadership-reflects-the-diverse-communities-it-serves)**Moe: How does Abbott ensure leadership reflects the diverse communities it serves?**
**Jennifer Jones-McMeans:** Ensuring diversity in leadership and decision-making teams is integral to our strategy. We evaluate external leadership, including principal investigators (PIs), for gender and diversity balance. Our internal process, part of our quality system, assesses PI selection with a focus on gender and racial diversity. We aim for diversity in thought leadership, gender, race, and ethnicity. We’ve successfully appointed more women as leads or co-leads in clinical programs in the vascular business. For example, two women are leading one of our post-approval trials.
This intentional work extends to steering committees, where we strive to include physicians who may not have traditionally been part of these evaluations. By making our diversity goals known and weighted in our selection process, we ensure that our clinical trials are led by individuals who reflect the communities we serve.
## [](#moe-what-cultural-competency-training-is-abbott-implementing-for-trial-teams)**Moe: What cultural competency training is Abbott implementing for trial teams?**
**Jennifer Jones-McMeans:** Cultural competency training is a cornerstone of our efforts to build trust and engagement with underrepresented populations. Through the Diversity in Research Office, we provide consistent education on diversity in healthcare across Abbott. We have developed resources and tools to disseminate this knowledge to those conducting trials and those generally interested in this work. For instance, following the FDA’s guidance on diversity and research action plans, we conducted an education series to explain the requirements and their rationale.
We also offer templates and internal navigators to guide teams unfamiliar with diversity research. Additionally, we have an external Diversity in Research Medical Advisory Board, comprising of diverse physicians and researchers, to whom internal teams present their plans. This process not only educates our teams but also holds us accountable. Furthermore, we invite external experts to our internal steering committee meetings on diversity and research, providing broader education on the work being done in this field.
## [](#moe-what-role-do-community-organizations-play-in-abbotts-strategy)**Moe: What role do community organizations play in Abbott’s strategy?**
**Jennifer Jones-McMeans:** Community organizations and advocacy groups are vital partners in our strategy to diversify clinical trials. We collaborate with existing community programs to understand and address their needs. For example, we partnered with the University of California San Francisco, Fresno, and Dr. Leigh Ann O’Banion’s Champions program, focusing on limb preservation and vascular cures. This partnership supports education and screening for vascular disease in the Central Valley.
Another example is our five-year partnership with Norton Healthcare’s Institute for [Health Equity](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/) in Louisville, KY. This investment supports the development of sustainable plans for diverse populations in an area that previously lacked access to care. By partnering with these community programs, we leverage their expertise and insights to inform our strategies rather than imposing solutions from a corporate perspective.
## [](#moe-how-does-abbott-address-technological-challenges-in-decentralized-trials)**Moe: How does Abbott address technological challenges in decentralized trials?**
**Jennifer Jones-McMeans:** Addressing technological challenges in decentralized trials is crucial to ensuring equitable participation. We ensure that the burden of technology doesn’t fall on patients. For instance, in a vascular study, we provided the necessary technology to sites and healthcare workers rather than expecting patients to have the required devices. This approach ensures that patients can participate in trials without investing in technology.
The corporation’s responsibility is to supply the tools needed for trials, not the patients. By providing the necessary technology and support, we aim to remove barriers to participation and ensure that all patients have equal access to clinical trials. This strategy helps us address the digital divide and ensures that underrepresented populations can fully engage in decentralized trials.
## [](#moe-is-there-anything-else-you-want-to-add-about-abbotts-diversity-efforts)**Moe: Is there anything else you want to add about Abbott’s diversity efforts?**
**Jennifer Jones-McMeans:** We’re excited to continue this work into our fourth year. Our website offers educational resources, and we invest in scholarships for young researchers and partnerships with Historically Black Colleges and Universities (HBCUs). For example, we have a $5 million five-year scholarship program for medical students at HBCUs and partnerships with the National Black Nurses Association (NBNA), and the National Association of Hispanic Nurses (NAHN).
This multi-layered approach addresses patient, physician, and healthcare system needs, ensuring comprehensive support for diversity in clinical trials. By focusing on the needs of patients and families, current physicians, and the pipeline of young researchers, we aim to create a sustainable and inclusive research environment. Our efforts are designed to foster a diverse and equitable future in clinical research.
**Categories:** Article: Executive Interviews
---
### [Enigma Biomedical & Neuraly Announce Research & Commercialization Deal](https://www.clinicaltrialvanguard.com/news/enigma-biomedical-neuraly-announce-research-commercialization-deal/)
**Published:** January 20, 2025
**Author:** Jon Napitupulu
**Content:**
Neuraly Inc., a subsidiary of D&D Pharmatech Inc., and Enigma Biomedical USA, Inc. signed a research license and commercialization option agreement for PMI04, a PET imaging biomarker of neuroinflammation. The agreement grants Enigma an exclusive research license for PMI04 with the option to negotiate commercialization rights. PMI04 targets activated microglia, offering a potentially more specific approach than current TSPO-targeted PET imaging for diagnosing neurodegenerative diseases like Alzheimer’s, [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/), and [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/).
This partnership is potentially groundbreaking for the neurodegenerative disease field. PMI04’s specificity to activated microglia could dramatically improve the accuracy and quantitative assessment of neuroinflammation. This could accelerate the development of effective therapies by providing researchers with a more reliable tool to measure treatment efficacy. Earlier and more accurate diagnoses could also significantly improve patient outcomes by enabling earlier intervention and potentially slowing disease progression.
Enigma will focus on developing PMI04 for neurodegenerative disease assessment. Neuraly will receive upfront and milestone payments tied to development and commercialization progress, as well as royalties on future sales, should the product reach the market. The agreement outlines a pathway for Enigma to obtain exclusive commercialization rights upon successful completion of the research phase.
This agreement signals a crucial step toward more precise diagnostic tools for neurodegenerative diseases. The potential of PMI04 to enhance the development and effectiveness of future treatments is significant. The success of this partnership could reshape the landscape of neurodegenerative disease diagnosis and ultimately improve the lives of patients affected by these debilitating conditions.
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**Categories:** News
---
### [Acalabrutinib Plus Chemoimmunotherapy Approved for Mantle Cell Lymphoma](https://www.clinicaltrialvanguard.com/news/acalabrutinib-plus-chemoimmunotherapy-approved-for-mantle-cell-lymphoma/)
**Published:** January 20, 2025
**Author:** Jon Napitupulu
**Content:**
The FDA has granted full approval to AstraZeneca’s CALQUENCE ([acalabrutinib](https://www.clinicaltrialvanguard.com/news/ascentage-pharma-presents-promising-cancer-research-at-aacr/)) combined with bendamustine and [rituximab](https://www.clinicaltrialvanguard.com/news/budoprutug-shows-response-in-rituximab-experienced-itp-patients/) for first-line treatment of adult mantle cell lymphoma (MCL) patients ineligible for autologous hematopoietic stem cell transplantation. This approval, based on the ECHO Phase III trial, follows a Priority Review and converts CALQUENCE’s prior accelerated approval for relapsed/refractory MCL into a full approval. The trial demonstrated a 27% reduction in the risk of disease progression or death compared to standard chemoimmunotherapy, translating to a median progression-free survival (PFS) of 66.4 months versus 49.6 months.
This approval significantly advances treatment options for MCL, a rare and aggressive form of non-Hodgkin lymphoma. Elderly patients, often ineligible for transplantation, now have access to a more effective first-line therapy with improved PFS, potentially delaying disease progression and improving quality of life. This represents a substantial improvement over existing chemoimmunotherapy regimens and potentially sets a new standard of care for this patient population.
The ECHO trial, conducted during the COVID-19 pandemic, showed a median PFS benefit of over 16 months for the CALQUENCE combination. When COVID-19 related deaths were excluded from the analysis, the risk reduction further improved to 36%. This highlights the robustness of the CALQUENCE regimen even amidst the challenges of a global pandemic. The safety profile of CALQUENCE remained consistent with previous findings.
This approval marks a crucial step forward for MCL treatment, providing a new and effective option for patients. The positive ECHO results, combined with the full FDA approval, are expected to increase CALQUENCE’s adoption as a first-line treatment and reinforce its position as a key therapy for B-cell malignancies. Further global regulatory decisions based on the ECHO data are anticipated, potentially expanding access to this promising treatment combination worldwide.
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**Categories:** News
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### [Zai Lab's KarXT for Schizophrenia Treatment Accepted by FDA](https://www.clinicaltrialvanguard.com/news/zai-labs-karxt-for-schizophrenia-treatment-accepted-by-fda/)
**Published:** January 20, 2025
**Author:** Jon Napitupulu
**Content:**
Zai Lab’s New Drug Application (NDA) for KarXT, a [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) treatment for adults, has been accepted by China’s National Medical Products Administration (NMPA). This submission follows successful Phase 3 trials in China and globally, where KarXT significantly reduced schizophrenia symptoms with a tolerable safety profile. The NDA acceptance is based on positive results from a Phase 1 PK study in China, the Phase 3 China study (ZL-2701-001), and data from global EMERGENT clinical programs.
This NDA acceptance is a crucial step toward addressing the significant unmet needs of over 8 million schizophrenia patients in China. Current treatment options often have limited efficacy and undesirable side effects, leading to high discontinuation rates and hindering patients’ ability to achieve life milestones. KarXT, the first new schizophrenia treatment class in decades, offers a potential alternative with a different mechanism of action than existing antipsychotics.
Technically, the China Phase 3 study mirrored the success of global trials. It met its primary endpoint, showing a statistically significant reduction in the PANSS total score. Key secondary endpoints, including positive and negative symptom subscales, were also met. The safety profile was consistent with previous KarXT trials, with common adverse events including vomiting, tachycardia, nausea, hypertension, dizziness, and diarrhea.
The NMPA’s acceptance of the KarXT NDA signifies potential progress in the schizophrenia treatment landscape in China. If approved, KarXT could offer a new and potentially more effective treatment option for a large patient population, ultimately improving patient outcomes and quality of life. This could also establish Zai Lab as a key player in the Chinese [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) market.
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**Categories:** News
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### [FDA Accepts Arrowhead's New Drug Application for Plozasiran Treatment](https://www.clinicaltrialvanguard.com/news/fda-accepts-arrowheads-new-drug-application-for-plozasiran-treatment/)
**Published:** January 20, 2025
**Author:** Jon Napitupulu
**Content:**
Arrowhead Pharmaceuticals announced that the FDA accepted its New Drug Application (NDA) for plozasiran, a treatment for the rare genetic disorder familial chylomicronemia syndrome (FCS). The FDA set a target action date of November 18, 2025, and currently does not plan to convene an advisory committee meeting. Arrowhead plans to submit applications for plozasiran to other regulatory bodies in 2025.
This NDA acceptance is a crucial step towards potentially providing a much-needed treatment option for individuals with FCS. FCS patients experience extremely high triglyceride levels, putting them at significant risk for acute pancreatitis and other debilitating complications that severely impact their quality of life. Current treatment options are limited, making plozasiran’s potential approval a significant advancement in FCS management.
The NDA submission hinges on positive results from the Phase 3 PALISADE study, supported by data from Phase 2 trials. PALISADE demonstrated that plozasiran significantly reduced triglycerides, apolipoprotein C-III, and the incidence of acute pancreatitis. Specifically, the study showed a median triglyceride reduction of 80% and an 83% reduction in acute pancreatitis risk. The 25 mg dose, proposed for marketing approval, was generally well-tolerated, with common adverse events including abdominal pain, [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/), nasopharyngitis, and nausea.
The FDA’s acceptance of the NDA for plozasiran marks a significant milestone for Arrowhead. A potential approval would represent a major breakthrough for FCS patients, offering a new therapeutic option to manage this severe and rare disease. It also positions Arrowhead to potentially expand into the broader markets of severe hypertriglyceridemia and mixed hyperlipidemia, which the company is actively investigating in ongoing Phase 3 trials.
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**Categories:** News
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### [Datroway® Approved for Metastatic Breast Cancer](https://www.clinicaltrialvanguard.com/news/datroway-approved-for-metastatic-breast-cancer/)
**Published:** January 20, 2025
**Author:** Jon Napitupulu
**Content:**
[Datopotamab](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/) deruxtecan (DATROWAY), a TROP2-directed antibody-drug conjugate (ADC), has received US approval for treating adults with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). Developed jointly by Daiichi Sankyo and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/), DATROWAY is indicated for patients who have undergone prior endocrine-based therapy and chemotherapy. This approval follows positive results from the TROPION-Breast01 phase 3 trial, where DATROWAY significantly improved progression-free survival compared to chemotherapy.
This approval addresses a significant unmet need for patients with HR-positive, HER2-negative metastatic breast cancer. Current treatment options after endocrine therapy and initial chemotherapy often yield limited responses. DATROWAY offers a new therapeutic approach with the potential to improve outcomes for this patient population, particularly those who have progressed after standard treatments. This is crucial given that only about 30% of patients with metastatic breast cancer survive five years after diagnosis.
In the TROPION-Breast01 trial, DATROWAY demonstrated a 37% reduction in the risk of disease progression or death compared to chemotherapy. The median progression-free survival was 6.9 months with DATROWAY versus 4.9 months with chemotherapy. Additionally, DATROWAY showed a higher objective response rate of 36% compared to 23% in the chemotherapy arm. However, the treatment also carries risks, with common adverse reactions including stomatitis, nausea, fatigue, and ocular issues. More serious adverse reactions like interstitial lung disease and pneumonitis were also observed, requiring careful patient monitoring.
The approval of DATROWAY represents a significant step forward in breast cancer treatment. This new ADC offers a novel mechanism of action and the potential to improve outcomes for patients who have exhausted other therapeutic avenues. It strengthens the position of both Daiichi Sankyo and AstraZeneca in the oncology market and suggests a growing role for ADCs in targeted cancer therapies. Further research and real-world data will be crucial in confirming the long-term benefits and safety profile of DATROWAY.
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**Categories:** News
---
### [Navigating Digital Health and Clinical Trials in Saudi Arabia](https://www.clinicaltrialvanguard.com/executiveinterviews/navigating-digital-health-and-clinical-trials-in-saudi-arabia/)
**Published:** January 21, 2025
**Author:** Moe Alsumidaie
**Content:**
In this Q&A, we interview Dr. Tamara Sunbul, a digital health clinical trial leader. Dr. Sunbul discusses the regulatory landscape, patient engagement strategies, and the future of digital health in Saudi Arabia. Her insights offer a comprehensive view of the challenges and opportunities in this rapidly evolving field.
## [](#moe-alsumidaie-how-do-you-improve-patient-enrollment-and-retention-in-saudi-clinical-trials)**Moe Alsumidaie: How do you improve patient enrollment and retention in Saudi clinical trials?**
Dr. Tamara Sunbul: To ensure that trials are not standalone but are part of the regular care patients receive, we embed clinical trials within the existing framework of standard medical procedures to provide a unified patient experience, ultimately bolstering both engagement and retention. For instance, when conducting a diabetic eye exam using machine learning, we perform the regular camera exam alongside the digital trial. This dual approach is necessary because, until we receive SFDA approval, we cannot rely solely on the digital method.. This strategy respects cultural preferences for comprehensive care and ensures patients feel secure and supported throughout the trial process. By embedding trials within
Dr. Tamara Sunbul, Digital Health Clinical Trial Leader
routine care, we address cultural sensitivities and enhance patient trust, which are crucial for successful enrollment and retention. This method also allows us to gather more comprehensive data, improving our research outcomes’ overall quality and reliability.
## [](#moe-alsumidaie-what-challenges-exist-in-attracting-big-pharma-funding-for-trials-in-saudi-arabia)**Moe Alsumidaie: What challenges exist in attracting Big Pharma funding for trials in Saudi Arabia?**
Dr. Tamara Sunbul: There are abundant funding opportunities across multiple sectors in Saudi Arabia, and we have strong buy-in from developers. This creates a sustainable ecosystem that allows us to focus on collaboration rather than financial constraints, fostering a supportive environment for innovation. This model encourages mutual assistance and shared goals, which can appeal more to developers than traditional funding models. By creating a collaborative ecosystem, we can attract more interest and investment from [Big Pharma](https://www.clinicaltrialvanguard.com/analysis/the-future-of-dcts-is-bright-according-to-big-pharma-and-fda/), as they see the value in partnering with a system that supports innovation and mutual growth. This approach benefits the developers and enhances the quality and scope of clinical trials conducted in Saudi Arabia.
## [](#moe-alsumidaie-how-do-international-collaborations-enhance-saudi-clinical-trials)**Moe Alsumidaie: How do international collaborations enhance Saudi clinical trials?**
Dr. Tamara Sunbul: While we do engage in some international collaborations, the regulatory environment in Saudi Arabia requires that all trials be re-registered locally. Even if a trial has FDA or EU approval, it must still be reviwed here to ensure it meets local standards and addresses population-specific data biases. This rigorous process ensures international partnerships enhance our research capabilities by providing robust, locally relevant data. By adhering to these stringent regulations, we can ensure that the data collected applies to our population, thereby improving the accuracy and reliability of our research outcomes. This approach strengthens our research capabilities and positions Saudi Arabia as a key player in the global clinical trial landscape.
## [](#moe-alsumidaie-how-does-saudi-arabias-tech-infrastructure-support-digital-health-trials)**Moe Alsumidaie: How does Saudi Arabia’s tech infrastructure support digital health trials?**
Dr. Tamara Sunbul: Saudi Arabia’s technology infrastructure is well-equipped to support digital health initiatives, thanks partly to regulatory sandboxes. These sandboxes act as incubators, ensuring all digital health solutions are thoroughly tested and compliant with cybersecurity standards before implementation. This proactive approach facilitates the smooth rollout of digital health projects and builds trust among stakeholders by ensuring that all solutions are safe and effective. By leveraging these regulatory sandboxes, we can streamline the development and implementation of digital health solutions, making it easier for developers to bring their innovations to market. This infrastructure supports the growth of digital health in Saudi Arabia and enhances our ability to conduct high-quality, reliable clinical trials.
## [](#moe-alsumidaie-how-is-data-privacy-addressed-in-saudi-digital-health-trials)**Moe Alsumidaie: How is data privacy addressed in Saudi digital health trials?**
Dr. Tamara Sunbul: Data privacy is a top priority in Saudi Arabia, with robust measures in place similar to GDPR. The Personal Data Protection Law (PDPL) and AI governance policies provide clear data handling and protection guidelines. These policies ensure that all digital health initiatives comply with local and international standards, offering a secure framework for data management. This clarity in regulations helps streamline processes and encourages innovation by providing a clear path for compliance. By adhering to these stringent data protection measures, we can ensure the privacy and security of patient data, which is crucial for building trust and confidence in digital health solutions. This approach protects patient privacy and enhances the quality and reliability of our clinical trials.
## [](#moe-alsumidaie-are-there-disparities-in-access-to-digital-health-in-saudi-arabia)**Moe Alsumidaie: Are there disparities in access to digital health in Saudi Arabia?**
Dr. Tamara Sunbul: Inclusion is a key focus of our AI governance policies, emphasizing access for all demographics, including those in remote areas and senior citizens. We address potential disparities by validating data locally, ensuring that solutions are tailored to the specific needs of our population. This approach recognizes that what works in other regions may not directly apply here, necessitating localized trials even for internationally approved solutions. By focusing on inclusion and local validation, we can ensure that digital health solutions are accessible to all, regardless of location or demographic.
## [](#moe-alsumidaie-how-do-you-see-digital-health-and-trials-evolving-in-saudi-arabia)**Moe Alsumidaie: How do you see digital health and trials evolving in Saudi Arabia?**
Dr. Tamara Sunbul: The future of digital health and decentralized trials in Saudi Arabia looks promising, thanks to stringent regulations that ensure quality and safety. Clinical trials are heavily regulated, requiring centers to be CBAHI accredited, which guarantees that all necessary infrastructure and policies are in place. This regulatory framework ensures high standards and facilitates the expansion of trials, potentially increasing diversity and accessibility in the future. By adhering to these stringent regulations, we can ensure that our clinical trials are of the highest quality, enhancing our ability to attract international collaborations and investment.
## [](#moe-alsumidaie-are-trials-only-in-large-hospitals-or-are-they-expanding-to-private-sectors)**Moe Alsumidaie: Are trials only in large hospitals, or are they expanding to private sectors?**
Dr. Tamara Sunbul: Clinical trials in Saudi Arabia are not limited to large hospital systems; they can be conducted in any institution that meets the necessary accreditation requirements. This includes private hospitals, provided they obtain CBAHI certification. This flexibility allows for a broader range of trial sites, encouraging wider participation and fostering a more inclusive research environment. By expanding the scope of clinical trials to include private hospitals, we can increase the diversity and accessibility of our research, which will enhance the overall quality and reliability of our clinical trials. This approach benefits the research community and improves the overall quality of healthcare in Saudi Arabia.
## [](#moe-alsumidaie-is-there-anything-else-youd-like-to-add-about-saudi-clinical-trials)**Moe Alsumidaie: Is there anything else you’d like to add about Saudi clinical trials?**
Dr. Tamara Sunbul: I’m excited about the developments in digital health and clinical trials in Saudi Arabia. The clear policies and regulations we have in place are crucial for progress, providing a solid foundation for innovation and ensuring that all stakeholders understand the rules of engagement. This clarity will likely accelerate advancements in digital clinical trials, positioning Saudi Arabia as a leader in this field. By adhering to these stringent regulations, we can ensure that our clinical trials are of the highest quality, enhancing our ability to attract international collaborations and investment.
**Categories:** Article: Executive Interviews
---
### [AI in Oncology to Hit $11.52B by 2030, with 29.4% CAGR](https://www.clinicaltrialvanguard.com/news/ai-in-oncology-to-hit-11-52b-by-2030-with-29-4-cagr/)
**Published:** January 21, 2025
**Author:** Jon Napitupulu
**Content:**
The global AI in oncology market, valued at US$1.92 billion in 2023, is projected to reach US$11.52 billion by 2030, driven by increased healthcare spending, advanced infrastructure adoption, and a growing prevalence of cancers. This growth is further fueled by regulatory approvals for AI-based software and its integration with radiographic imaging, exemplified by Google Health’s 2022 partnership with iCAD to enhance [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) diagnosis. The drug discovery application segment currently holds a significant market share due to the rising cancer burden and technological advancements that aid disease understanding. Pharmaceutical and biotechnology companies are major end-users, heavily investing in cancer drug discovery, development, and clinical trials, evidenced by the 14 FDA approvals for cancer therapies in the first quarter of 2024 alone.
This rapid market expansion is crucial for both the healthcare industry and cancer patients. The increasing prevalence of cancer necessitates innovative and effective solutions. AI offers the potential to revolutionize cancer care by improving early detection, personalizing treatments, and accelerating drug discovery. This translates to earlier diagnoses, better treatment outcomes, and potentially, increased survival rates for patients. Furthermore, streamlining drug discovery using AI could significantly reduce development timelines and costs, leading to faster access to life-saving medications.
The drug discovery segment, focusing on target identification, validation, lead identification and optimization, and de novo drug design, is propelled by the emphasis on personalized medicine, increased investments, and supportive regulatory frameworks. Key market players like Siemens Healthineers, GE Healthcare, and Medtronic are actively developing and deploying AI-powered solutions for cancer care, ranging from automated imaging support and treatment planning to real-time polyp detection during colonoscopies. These companies are utilizing a range of strategies, including acquisitions, collaborations, and product launches, to solidify their market positions and drive innovation.
The projected growth of the AI in oncology market signifies a paradigm shift in how cancer is diagnosed and treated. The ongoing development and implementation of AI technologies promise to further refine diagnostic accuracy, personalize treatment strategies, and expedite the development of novel cancer therapies. This not only presents significant opportunities for companies operating in this space but also offers hope for improved patient outcomes and a future where cancer is more effectively managed and treated.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Mainz Biomed starts a next-generation clinical trial for adenoma detection](https://www.clinicaltrialvanguard.com/news/mainz-biomed-starts-a-next-generation-clinical-trial-for-adenoma-detection/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
Mainz Biomed NV launched eAArly DETECT 2, a U.S. feasibility study for its next-generation [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (CRC) screening test. The study, involving approximately 2,000 average-risk patients, aims to validate earlier promising results and pave the way for a larger pivotal study, ReconAAsense, planned for 2026. The new test integrates mRNA biomarkers, an AI-driven algorithm, and a fecal immunochemical test (FIT).
This study is crucial because it could significantly advance CRC screening by potentially enabling the detection of precancerous adenomas, not just cancerous polyps. Early detection of advanced adenomas could shift the paradigm from reactive treatment to proactive prevention, drastically improving patient outcomes and reducing CRC mortality rates. Successful validation of this combined approach could also establish a new standard for non-invasive CRC screening, surpassing current FIT-based methods in sensitivity and specificity.
The eAArly DETECT 2 study is expected to complete enrollment in the second half of 2025, with topline results anticipated in the fourth quarter of 2025. The study focuses on evaluating five novel mRNA biomarkers acquired from Sherbrooke University in 2022. These biomarkers, combined with Mainz Biomed’s proprietary AI algorithm, are expected to enhance the test’s ability to identify advanced adenomas and early-stage CRC, increasing both diagnostic sensitivity and specificity.
The results of eAArly DETECT 2 will directly inform the protocols for the pivotal ReconAAsense study. Positive results could accelerate the development and regulatory approval of this next-generation CRC screening test, positioning Mainz Biomed at the forefront of early cancer detection and potentially transforming colorectal cancer screening practices worldwide. This advancement could also lead to wider adoption of more effective screening methods, contributing to a significant decrease in global CRC incidence and mortality.
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**Categories:** News
---
### [RS Biotherapeutics Sees Positive Effects of Lead Compound on Human Lung Tissue](https://www.clinicaltrialvanguard.com/news/rs-biotherapeutics-sees-positive-effects-of-lead-compound-on-human-lung-tissue/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
RS [BioTherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) announced positive results from a human lung tissue study of its lead compound, RSBT-001. The study showed RSBT-001 significantly reduced inflammatory biomarkers and mediators of fibrosis, confirming prior animal study results and expanding the compound’s potential therapeutic applications. This first-in-class, steroid-free compound is being developed for deadly lung diseases like COPD and IPF.
This news holds significant promise for patients suffering from debilitating lung diseases. Current treatment options, particularly steroids, often come with significant side effects. RSBT-001’s demonstrated efficacy in reducing inflammation and fibrosis in human lung tissue, coupled with its steroid-free nature, positions it as a potential game-changer in the treatment landscape. It offers the possibility of improved efficacy with fewer adverse effects, potentially transforming the quality of life for millions.
RSBT-001 showed statistically significant reductions in key inflammatory biomarkers like IL-6, IL-8, TNF-α, and IL-1β, all of which are implicated in COPD. Furthermore, the compound significantly reduced Collagen 1a1 and TIMP-1, both involved in lung tissue remodeling and fibrosis in COPD and IPF. The study utilized FibroFind’s precision-cut tissue slice platform, a model considered highly representative of human lung physiology.
These positive results bolster confidence in RSBT-001’s potential and pave the way for continued development. The company plans to file an Investigational New Drug Application in 2026. The data suggests a promising future for this compound as a potential treatment for a variety of serious lung conditions, offering hope for a new era of effective and safer therapies.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Xilio Announces Phase 2 Data for Vilastobart in Metastatic Colorectal Cancer](https://www.clinicaltrialvanguard.com/news/xilio-announces-phase-2-data-for-vilastobart-in-metastatic-colorectal-cancer/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
Xilio Therapeutics announced initial positive Phase 2 data for vilastobart (XTX101), a tumor-activated anti-CTLA-4 antibody, combined with [atezolizumab](https://www.clinicaltrialvanguard.com/news/xilio-announces-updated-phase-2-data-for-vilastobart/) (Tecentriq®) in patients with metastatic microsatellite stable [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (MSS CRC). The combination showed a 27% objective response rate in heavily pre-treated patients without liver metastases, accompanied by biomarker and symptom improvements. Data also suggested a favorable safety and tolerability profile.
This news holds particular promise for MSS CRC patients, a population with limited treatment options who haven’t benefited significantly from existing immunotherapies, including PD-(L)1 inhibitors used alone. The observed responses and manageable side effects suggest this combination could address a substantial unmet need in this challenging cancer type.
In the trial, 40 heavily pre-treated patients received vilastobart and atezolizumab. Among the 11 evaluable patients without liver metastases, three achieved partial responses. These responses correlated with decreases in carcinoembryonic antigen (CEA) and circulating tumor DNA (ctDNA), and improvements in clinical symptoms. The combination’s safety profile appears encouraging, with a low incidence of immune-related adverse events, notably a 5% rate of colitis.
These early results lay the foundation for further exploration of vilastobart in MSS CRC and potentially other “cold” tumors resistant to traditional immunotherapy. Xilio plans to release updated Phase 2 data mid-2025 and will explore partnerships to expand development of this promising combination therapy.
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**Categories:** News
---
### [Nikang's NKT2152 Dosed in First HCC Patient: Phase 1b/2 Trial Begins](https://www.clinicaltrialvanguard.com/news/nikangs-nkt2152-dosed-in-first-hcc-patient-phase-1b-2-trial-begins/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
NiKang Therapeutics has initiated a global Phase 1b/2 clinical trial evaluating NKT2152, an oral HIF2α inhibitor, in combination with [atezolizumab](https://www.clinicaltrialvanguard.com/news/xilio-announces-updated-phase-2-data-for-vilastobart/) and [bevacizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-lytenava-first-ophthalmic-bevacizumab-for-wet-amd/) for first-line treatment of advanced or metastatic hepatocellular carcinoma (HCC). This trial, part of Roche’s MORPHEUS-liver platform, will compare the combination therapy to the standard atezolizumab and bevacizumab regimen. Preclinical data suggests NKT2152 has broad anti-tumor activity, making HCC a promising target beyond its current trials in renal cell carcinoma.
This trial is significant because it expands the potential applications of NKT2152 into a new and prevalent cancer type. HCC has limited treatment options, and the existing standard-of-care therapy, while effective, may not be sufficient for all patients. A successful trial could offer a new combination treatment approach with improved efficacy, potentially benefiting a significant number of patients. Furthermore, the collaboration with Roche and inclusion within the MORPHEUS platform provides external validation of NKT2152’s potential and offers access to Roche’s extensive clinical development expertise.
This Phase 1b/2 trial combines NKT2152 with the established HCC standard-of-care regimen. NKT2152 inhibits HIF2α, a protein implicated in tumor growth. The drug’s pharmacokinetic profile, characterized by high systemic exposure and long half-life, makes it suitable for combination with antibody-based therapies. NKT2152 is also being evaluated in other clinical trials, including studies for renal cell carcinoma as a single agent and in combination with other drugs.
The initiation of this HCC trial marks a crucial step in expanding the clinical evaluation of NKT2152. Positive results could lead to a new treatment option for HCC, addressing an unmet medical need. It also positions NiKang as a key player in developing innovative cancer therapies and strengthens their ongoing collaboration with Roche. The data from this trial will be critical for determining the future development and potential regulatory pathways for NKT2152 in HCC.
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**Categories:** News
---
### [Kiromic Biopharma Sees Tumor Volume Decrease in Two Deltacel-01 Patients](https://www.clinicaltrialvanguard.com/news/kiromic-biopharma-sees-tumor-volume-decrease-in-two-deltacel-01-patients/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
Kiromic BioPharma announced positive efficacy results from its Deltacel-01 Phase 1 clinical trial of Deltacel™, an allogeneic, off-the-shelf Gamma Delta T-[cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for late-stage [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). Two patients showed significant tumor reduction: the first enrolled patient experienced a 33.33% reduction at the 12-month follow-up, and the seventh patient showed a 9.5% reduction at the two-month mark. Both patients are being treated at the Beverly Hills Cancer Center. Additionally, the eighth patient completed treatment and awaits efficacy results, while the ninth recently began treatment at a different location.
These results are promising for patients with advanced NSCLC who have limited treatment options after failing standard therapies. The sustained tumor reduction observed in the initial patient, coupled with the responses seen in subsequent patients, suggests Deltacel™ may offer a new therapeutic avenue. This strengthens the potential for gamma delta T-cell therapies to address this challenging cancer.
The Deltacel-01 trial is an open-label Phase 1 study evaluating Deltacel™ in combination with low-dose radiation. The primary objective is safety, with secondary endpoints including objective response, progression-free survival, and overall survival. The company is continuing to expand enrollment with the tenth and eleventh patients expected to join by the end of January. The eighth patient’s initial efficacy results are anticipated in late February 2025.
These ongoing results could significantly impact the development of GDT cell therapies and provide a potential new treatment option for advanced NSCLC. The continued enrollment and upcoming efficacy data will be crucial for further validating Deltacel™’s efficacy and safety profile, paving the way for potential future trials and ultimately, impacting the treatment landscape for this patient population.
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**Categories:** News
---
### [Allurion Gets New US Patent for Next-Gen Weight Loss Device](https://www.clinicaltrialvanguard.com/news/allurion-gets-new-us-patent-for-next-gen-weight-loss-device/)
**Published:** January 22, 2025
**Author:** Jon Napitupulu
**Content:**
Allurion Technologies received a patent for its enhanced, next-generation gastric balloon technology, specifically for its improved valve system. This new patent adds to Allurion’s existing intellectual property, totaling 20 patents in the U.S. and 59 globally. The patented technology focuses on the precision of the balloon’s valve system, a critical component of the Allurion Program, a weight-loss platform combining the swallowable gastric balloon with a virtual care suite.
This patent is important because it strengthens Allurion’s competitive position in the weight-loss market. It provides extended intellectual property protection for a key element of its technology, potentially deterring competitors and solidifying the company’s market advantage. This protection could translate into greater market share and increased revenue potential, particularly as the demand for effective, less invasive weight-loss solutions grows. The enhanced valve system may also lead to improved patient outcomes, potentially increasing the efficacy and safety of the Allurion Gastric Balloon.
This patent offers protection through July 2039. It covers an enhanced version of Allurion’s existing swallowable gastric balloon, focusing on the precision of the valve system controlling balloon opening. The Allurion Program incorporates a virtual care suite, including a mobile app and connected scale, to support patients throughout their weight-loss journey. Patients using the Allurion Program typically experience 10-15% total body weight loss, with some studies showing increased muscle mass.
This patent reinforces Allurion’s commitment to innovation in the [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatment space. The extended patent protection secures the company’s investment in research and development, allowing for continued exploration of advancements in its gastric balloon technology. This innovation may lead to further refinements in the device’s design, functionality, and patient experience, ultimately strengthening Allurion’s long-term market position and contributing to the development of more effective obesity solutions.
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**Categories:** News
---
### [Emervax's Circular RNA: A New Frontier in Vaccine Development](https://www.clinicaltrialvanguard.com/executiveinterviews/emervaxs-circular-rna-a-new-frontier-in-vaccine-development/)
**Published:** January 23, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, we speak with Peter Weinstein, co-founder of Emervax. The discussion delves into Emervax’s innovative circular RNA technology, its advantages over traditional mRNA, strategic partnerships, and the company’s commitment to global vaccine access.
## [](#moe-how-does-emervaxs-emxrnatm-platform-improve-vaccine-stability-and-efficacy-over-mrna)**Moe: How does Emervax’s emxRNATM platform improve vaccine stability and efficacy over mRNA?**
Our circular RNA platform, which we have named emxRNATM,offers greater stability than traditional mRNA, which is prone to degradation due to its linear structure. This degradation necessitates using end caps in mRNA vaccines to protect them from nucleases. In contrast, circular RNA does not have these vulnerable ends, making it more robust. For example, during the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic, mRNA vaccines required storage at extremely low temperatures (-50 to -70 degrees Celsius), which posed significant logistical challenges. However, Emervax’s emxRNATM platform can be stored at room temperature, simplifying distribution and storage. Our platform has developed unique sequences that enhance circularization, resulting in about 50% of the RNA becoming circularized, which is twice the normal rate. This increased stability and protein production capability make our platform a promising alternative to traditional mRNA vaccines.
## [](#moe-how-does-the-partnership-with-kindevaimprove-vaccine-administration-and-compliance)**Moe: How does the partnership with Kindevaimprove vaccine administration and compliance?**
The partnership with KindevaDrug Delivery introduces microneedle array patches, revolutionizing vaccine administration by making it more convenient and less invasive. Traditional vaccines require intramuscular injections, necessitating visits to clinics or pharmacies. With the microneedle patches, patients can self-administer the vaccine at home by simply applying the patch for five minutes. This ease of use is expected to improve patient compliance significantly, as it eliminates the need for multiple clinic visits. Additionally, the patches are easy to dispose of, reducing the risk associated with sharps disposal. This innovation aligns with CEPI’s goal of making vaccines accessible in remote and underserved areas, as the patches can be easily transported and applied without the need for specialized medical personnel.
## [](#moe-how-do-you-ensure-equitable-access-to-vaccines-in-low-income-countries)**Moe: How do you ensure equitable access to vaccines in low-income countries?**
Emervax is committed to ensuring equitable vaccine access by focusing on affordability and ease of distribution. We are actively working on tech transfers to manufacturers in countries like Egypt and South Africa to produce vaccines locally at a reasonable cost. This approach reduces production costs and facilitates distribution in low-income regions. Our collaboration with CEPI supports this mission, as they fund initiatives to develop vaccines that can be distributed in areas with limited access to healthcare infrastructure. By leveraging partnerships and focusing on cost-effective production, we aim to make our vaccines accessible to all, regardless of geographic or economic barriers.
## [](#moe-what-challenges-do-you-anticipate-in-scaling-up-circular-rna-vaccine-production)**Moe: What challenges do you anticipate in scaling up circular RNA vaccine production?**
Scaling up production involves transitioning from GLP (Good Laboratory Practice) to GMP (Good Manufacturing Practice) manufacturing, which requires significant resources and expertise. We are currently discussing with four major bio manufacturers in the United States to facilitate this transition. While tech transfer can present challenges, particularly when moving production to countries with different regulatory standards, our experience and strategic partnerships should help mitigate these issues. Funding is always a concern, but we actively pursue non-dilutive funding to support our efforts and are currently initiating a Bridge Round and A concomitant Series A Round. We believe our platform will scale effectively for infectious disease vaccines based on our current data, which shows promising results in animal studies and we expect the same efficiency of production with our cancer and autoimmune emxRNATM vaccines.
## [](#moe-how-does-your-collaboration-with-cepi-align-with-global-vaccine-development-efforts)**Moe: How does your collaboration with CEPI align with global vaccine development efforts?**
CEPI’s mission is to develop platforms that enable rapid vaccine production during pandemics. Our emxRNATM circular RNA platform is designed to be highly adaptable, allowing for quick integration of new antigens. This capability is crucial for responding to potential pandemics like MERS, which has a high mortality rate. By having a pre-approved platform, we can expedite the development and deployment of vaccines in response to emerging threats. This aligns with CEPI’s goal of ensuring global preparedness for future pandemics by having scalable and adaptable vaccine platforms ready for rapid deployment.
## [](#moe-what-potential-applications-do-you-see-for-circular-rna-beyond-infectious-diseases)**Moe: What potential applications do you see for circular RNA beyond infectious diseases?**
Beyond infectious diseases, our emxRNATM technology is promising for applications in oncology and autoimmune disorders. We are currently developing vaccines for blood cancers and exploring autoimmune applications. Our platform can potentially replace CAR T-cell therapies, offering a more cost-effective solution. While bacterial and parasitic diseases present more complex challenges, we believe our technology can address these with further development. By integrating AI for antigen screening, we aim to expand the applicability of our platform to a broader range of diseases, ultimately improving patient outcomes across multiple therapeutic areas.
**Categories:** Article: Executive Interviews
---
### [Exploring Innovations in Muscle Wasting Disease Treatment](https://www.clinicaltrialvanguard.com/executiveinterviews/exploring-innovations-in-muscle-wasting-disease-treatment/)
**Published:** January 23, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, we engage with David Craig from Sarcomatrix Therapeutics to discuss the development of S-969, a novel oral small molecule targeting the Hippo-YAP pathway. Our conversation covers regulatory challenges, clinical trial design, manufacturing scale-up, intellectual property protection, and strategic partnerships, offering insights into Sarcomatrix’s approach to addressing muscle wasting diseases.
## [](#moe-what-regulatory-challenges-do-you-foresee-for-s-969-and-how-will-you-address-them)**Moe: What regulatory challenges do you foresee for S-969, and how will you address them?**
The integrins are crucial structural proteins involved in [cell signaling](https://www.clinicaltrialvanguard.com/news/molecular-switches-in-cell-signaling-drug-development/). Our research team has found a way to increase the expression of the muscle-specific α7β1 integrin through a kinase on the Hippo-YAP pathway. This integrin is vital for muscle fiber rebuilding and myogenesis. For instance, in healthy individuals, after exercise, this integrin’s expression can increase by 200-300%, and in elite athletes, it can go up to 500%. From a regulatory perspective, we are leveraging the accelerated approval pathway based on
David Craig, CEO of Sarcomatrix Therapeutics
biomarkers, as established by companies like Sarepta. This approach, combined with patient advocacy, potentially positions us to enter the market by 2029. We are making sure to follow proven paths to market, learning from the experiences of others in the field.
## [](#moe-how-are-you-designing-trials-to-measure-s-969s-efficacy-and-safety-across-diverse-populations)**Moe: How are you designing trials to measure S-969’s efficacy and safety across diverse populations?**
We plan to use the increased expression of α7β1integrin as a biomarker for accelerated approval. The agency and NIH recommend this as a favorable biomarker. Additionally, we’ll employ the North Star Scale, which is crucial for our trials despite being considered somewhat subjective. This scale involves a series of exercises to assess muscle function. We’ll also conduct pre and post-treatment muscle biopsies to determine muscle fiber count and myogenic expression signals, such as MyoD. These comprehensive measures will help us evaluate the drug’s impact across diverse patient populations.
## [](#moe-what-strategies-are-in-place-to-scale-manufacturing-and-ensure-quality-control)**Moe: What strategies are in place to scale manufacturing and ensure quality control?**
I’ve been fortunate to gain experience from several biotech companies, learning the importance of manufacturing and quality control. For example, at Portola, we learned that most complete response letters are due to manufacturing and quality issues. We’ve brought on Al Swarz, an expert in protein and small molecules, to refine our RFP for CDMO and CRO partnerships. Additionally, Vikas, a colleague with a boutique IT group, is helping us implement quality and clinical trial management systems. Our molecule is relatively simple to manufacture, and we are confident in our ability to maintain high purity levels, as demonstrated in our preclinical studies with mice, rats, and dogs.
## [](#moe-how-are-you-safeguarding-your-intellectual-property-to-maintain-a-competitive-edge)**Moe: How are you safeguarding your intellectual property to maintain a competitive edge?**
The University of Nevada’s tech transfer group has successfully filed method and utility patents for our small molecules targeting α7β1integrin. They provide IP filings and deferred payments with milestones and licensing. We have robust IP protection in the U.S., Europe, Asia, and other regions. Our second program, a protein replacement therapy, also has strong IP protection, ensuring our competitive edge. This comprehensive IP strategy covers a broad class of muscle diseases, including muscle wasting.
## [](#moe-how-do-you-differentiate-yourself-scientifically-and-strategically-from-competitors)**Moe: How do you differentiate yourself scientifically and strategically from competitors?**
We are unique in focusing on small molecules that upregulate α7β1integrin expression, leveraging natural myogenic pathways. This holistic approach addresses multiple pathways, unlike competitors targeting individual pathways. Our small molecules also support skeletal and cardiac muscle, potentially impacting cardiac and respiratory failure, the leading causes of death in these patients. This comprehensive approach offers a more effective solution for muscle wasting conditions, setting us apart from other companies in the field.
## [](#moe-how-do-you-evaluate-and-select-strategic-partners-to-enhance-rd-capabilities)**Moe: How do you evaluate and select strategic partners to enhance R&D capabilities?**
We are fortunate to collaborate with the Burkin lab at the University of Nevada, Reno, a leader in integrins and laminins research. Our Chief Science Officer, Ryan Wuebbles, PhD, is well-connected with patient advocacy groups, and my connections have helped us engage with the patient community. For example, my niece, who lived with muscular dystrophy, inspired our commitment to this field. We prioritize cutting-edge science and the depth of knowledge when evaluating potential partners, ensuring we work with the best researchers and engage with the patient community effectively.
## [](#moe-how-are-you-allocating-resources-to-balance-research-trial-preparation-and-financial-sustainability)**Moe: How are you allocating resources to balance research, trial preparation, and financial sustainability?**
We have a $2 million Phase 2B grant supporting our preclinical research. This includes a small nonhuman primate study for PKPD and a study using the [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy dog model at Texas A&M. Our $5 million seed round will cover operational costs until our IND filing. In comparison, a $65 million Series A round will advance us through Phase 1 and potentially to market by 2029. This strategic allocation ensures we can sustain operations while advancing our research and clinical trials.
## [](#moe-what-steps-are-you-taking-to-ensure-diversity-and-inclusion-in-patient-recruitment)**Moe: What steps are you taking to ensure diversity and inclusion in patient recruitment?**
We leverage patient registries and collaborate with groups like the Muscular Dystrophy Association to access diverse patient populations. While the disease predominantly affects males and has been supported by the Caucasian community, we are actively seeking to address recruitment disparities and engage with minority groups. We have conducted incidence and prevalence analyses worldwide, identifying opportunities to capture untreated patients in Europe and Asia, ensuring a diverse and inclusive approach to patient recruitment.
## [](#moe-how-will-you-incorporate-patient-feedback-or-real-world-data-into-your-studies)**Moe: How will you incorporate patient feedback or real-world data into your studies?**
Using standard tools and validated motion detection monitoring, we aim to incorporate patient feedback and quality measures. These insights will supplement the North Star Scale and help us better understand patient needs, particularly in improving quality-of-life aspects like gut motility. For example, validated motion detection can track steps and gait, providing valuable data to enhance our understanding of the drug’s impact on patients’ daily lives.
**Categories:** Article: Executive Interviews
---
### [Coherus' Final Phase 2 Combination Data for Metastatic HCC at ASCO-GI 2025](https://www.clinicaltrialvanguard.com/news/coherus-final-phase-2-combination-data-for-metastatic-hcc-at-asco-gi-2025/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Coherus BioSciences announced final data from its Phase 2 trial of casdozokitug (casdozo), an IL-27-targeting antibody, combined with atezolizumab and [bevacizumab](https://www.clinicaltrialvanguard.com/news/fda-approves-lytenava-first-ophthalmic-bevacizumab-for-wet-amd/) in patients with advanced [liver cancer](https://www.clinicaltrialvanguard.com/news/can-fite-biopharma-proceeds-with-phase-3-liver-cancer-trial/) (HCC). The final data shows a higher overall response rate (ORR) of 38% and a complete response rate of 17.2%, significantly improved from earlier reported figures. The positive results were observed in both viral and non-viral HCC, with a manageable safety profile.
These findings are a notable advancement in HCC treatment, where options, especially for advanced stages, remain limited. The improved ORR and CR rates, coupled with the drug’s activity across different HCC etiologies, suggest casdozo could become a valuable therapeutic option, potentially improving long-term outcomes for a broader patient population. This is particularly important given the high mortality rate associated with HCC and the limitations of existing therapies.
The Phase 2 trial data revealed a 38% ORR with 5 complete and 6 partial responses based on RECIST v1.1 criteria. Using mRECIST criteria, the ORR was 43% with 5 complete and 7 partial responses. Median progression-free survival was approximately 8 months under both criteria. Importantly, biomarker data corroborated the preclinical findings, showing inhibition of IL-27 signaling and immune activation. Based on these positive results, Coherus launched a new randomized Phase 2 trial evaluating casdozo in combination with bevacizumab and [toripalimab](https://www.clinicaltrialvanguard.com/news/china-approves-toripalimab-plus-disitamab-vedotin-for-her2-urothelial-cancer/), its anti-PD-1 antibody. This trial will include approximately 72 patients and will investigate two different casdozo dosages alongside the other therapies.
The promising results from the Phase 2 trial combined with the initiation of a new trial involving a PD-1 inhibitor signal a potential shift in the HCC treatment landscape. The enhanced efficacy and acceptable safety profile observed with casdozo suggest it could become a cornerstone in future HCC combination therapies, offering new hope for patients with this aggressive cancer. Further research will clarify the optimal dosing and combination strategies for casdozo, potentially leading to improved survival rates and quality of life for individuals battling HCC.
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### [Helius Pons Therapy Shows Positive Gait Improvement in MS Patients](https://www.clinicaltrialvanguard.com/news/helius-pons-therapy-shows-positive-gait-improvement-in-ms-patients/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Helius Medical Technologies announced positive results from their PoNSTEP study, demonstrating the long-term benefits of PoNS Therapy for gait deficits in [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS) patients. The study showed a significant improvement in gait, measured by the Dynamic Gait Index (DGI), and a correlation between adherence to the therapy and the degree of improvement. This is the first clinical evidence linking adherence to PoNS Therapy with gait improvement and establishing its long-term effects six months post-treatment in MS patients.
This news is impactful for both MS patients and the field of neuromodulation. For patients experiencing gait difficulties due to MS, PoNS Therapy offers a potential solution for improving mobility and quality of life, with benefits lasting beyond the initial treatment period. The demonstrated link between adherence and improvement empowers patients to actively participate in their recovery. The findings also offer clinicians a more robust understanding of how to integrate this therapy effectively. This reinforces the potential of neuromodulation as a valuable tool in neurological rehabilitation, opening doors for wider application and further research into its mechanisms.
The PoNSTEP study involved 43 MS patients undergoing 14 weeks of PoNS Therapy combined with physical rehabilitation. 38 patients completed the initial 14-week protocol, showing a mean DGI improvement of 5 points. Adherence to the at-home portion of the therapy (Phase 2) averaged 71% and was directly correlated with gait improvement. At the six-month follow-up, only one out of 28 assessed patients experienced a significant decline in DGI, suggesting durable therapeutic benefits. This data supports the importance of treatment adherence for maximizing and maintaining positive outcomes with PoNS Therapy.
These positive results pave the way for broader adoption of PoNS Therapy. The evidence of sustained improvement and the correlation between adherence and outcomes strengthen the case for its inclusion in standard MS treatment protocols. This could translate into improved mobility and quality of life for a larger population of MS patients. Furthermore, the study’s findings encourage further investigation into the role of neuroplasticity in rehabilitation and may inspire the development of similar neuromodulation therapies for other neurological conditions.
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**Categories:** News
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### [Certepetide Shows Promise in Metastatic Pancreatic Cancer Trial](https://www.clinicaltrialvanguard.com/news/certepetide-shows-promise-in-metastatic-pancreatic-cancer-trial/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Lisata Therapeutics announced preliminary data from Cohort A of its Phase 2 ASCEND trial evaluating [certepetide](https://www.clinicaltrialvanguard.com/news/lisata-touts-surprising-preclinical-certepetide-adc-data/), combined with standard chemotherapy, for metastatic pancreatic ductal adenocarcinoma (mPDAC). The data showed a positive trend in overall survival (mOS) for the certepetide group (12.68 months) compared to the placebo group (9.72 months), and four complete responses were observed in the certepetide group versus none in the placebo group. Data from Cohort B, which uses a different dosing regimen, are expected soon.
This preliminary data is promising for mPDAC patients, who face a dismal prognosis with current standard-of-care treatments. The observed improvement in overall survival and the presence of complete responses, albeit in a small number of patients, suggest that certepetide may offer a meaningful clinical benefit. This is particularly significant given the limited treatment options available for this aggressive cancer. Positive results from Cohort B would further strengthen the case for certepetide’s potential to improve outcomes for these patients.
Cohort A enrolled 95 patients and administered a single dose of certepetide or placebo alongside standard chemotherapy. While median progression-free survival (PFS) showed no significant difference between the groups, the positive trend in mOS and the complete responses are encouraging. Cohort B is evaluating a two-dose regimen of certepetide, and the company anticipates even stronger results from this cohort, particularly in PFS.
These early findings support Lisata’s plan to progress certepetide to Phase 3 trials in early 2026. Confirmation of these positive trends in Cohort B and subsequent Phase 3 trials could position certepetide as a valuable addition to the current treatment landscape for mPDAC, offering new hope for patients with this challenging cancer.
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### [Discovery of New Immune Biomarkers in MS Patients Treated with Nasal Foralumab](https://www.clinicaltrialvanguard.com/news/discovery-of-new-immune-biomarkers-in-ms-patients-treated-with-nasal-foralumab/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
[Tiziana Life Sciences](https://www.clinicaltrialvanguard.com/news/tiziana-life-sciences-nasal-spray-shows-promise-in-spinal-cord-injury-treatment/)[Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) has discovered new immune biomarkers in patients with non-active secondary progressive [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (na-SPMS) treated with their lead candidate, intranasal foralumab, a fully human anti-CD3 monoclonal antibody. The biomarkers, identified through single-cell RNA sequencing, indicate modulation of T regulatory cells, central memory T cells, monocytes, and B cells – pathways associated with antigen presentation, interferon responses, and other immune regulatory mechanisms. This discovery stems from Tiziana’s ongoing expanded access program (ISPPEA) and correlates with previously observed reductions in microglial brain inflammation in the same patient group.
This biomarker discovery is crucial for advancing the treatment of na-SPMS. It provides mechanistic insight into the clinical effects observed with nasal foralumab, potentially paving the way for personalized MS treatment strategies. Confirming the drug’s biological activity strengthens its position as a promising therapeutic option in a disease area with significant unmet medical need. This offers hope for patients who have historically had limited treatment choices.
The study involved single-cell RNA sequencing of peripheral blood samples collected before treatment and at three and six-month intervals after nasal foralumab administration. The identified gene expression changes, correlating with reduced brain inflammation, provide concrete evidence of foralumab’s immunomodulatory effects. These findings are being prepared for submission to a peer-reviewed journal. Foralumab is currently being investigated in a Phase 2a trial for na-SPMS and an expanded access program now enrolling up to 30 patients.
This biomarker discovery is a significant step forward for Tiziana and the development of foralumab. It strengthens the scientific rationale behind its clinical development, potentially accelerating its path towards regulatory approval and ultimately providing a much-needed treatment option for na-SPMS patients. It also positions foralumab as a potential therapeutic agent for other autoimmune and neurodegenerative diseases, broadening the company’s pipeline and market opportunities.
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### [Novel Factor XI Inhibitor Reduces Bleeding in Atrial Fibrillation](https://www.clinicaltrialvanguard.com/news/novel-factor-xi-inhibitor-reduces-bleeding-in-atrial-fibrillation/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Anthos Therapeutics announced the publication of positive results from its [AZALEA](https://www.clinicaltrialvanguard.com/news/anthos-therapeutics-shares-new-data-from-landmark-azalea-timi-71-study/)-TIMI 71 study in the •New England Journal of Medicine•. The study evaluated abelacimab, a Factor XI inhibitor, against rivaroxaban (a standard direct oral anticoagulant or DOAC) in atrial fibrillation patients at moderate-to-high stroke risk. Abelacimab demonstrated a significant reduction in bleeding events across all primary and secondary endpoints.
This news holds substantial implications for atrial fibrillation management. Current anticoagulants effectively reduce stroke risk, but carry a significant bleeding risk, leading to undertreatment and preventable strokes. Abelacimab’s demonstrated ability to significantly reduce bleeding, while potentially maintaining efficacy in stroke prevention, could encourage wider adoption of anticoagulation therapy among eligible patients and physicians. This could translate into fewer strokes and improved patient outcomes, addressing a significant unmet need in cardiovascular care.
The AZALEA-TIMI 71 Phase 2 study, the longest head-to-head study of its kind, showed a 62% reduction in major or clinically relevant non-major bleeding with abelacimab compared to rivaroxaban. Furthermore, major bleeding alone was reduced by 67%, and gastrointestinal bleeding saw an 89% reduction. The study also indicated sustained Factor XI inhibition of 99% with monthly 150mg abelacimab dosing. Previous data presentations from the study also highlighted low periprocedural bleeding rates and similar benefits in patients on antiplatelet therapy.
Abelacimab’s positive results pave the way for its potential emergence as a safer and more convenient anticoagulant option. Pending further research, particularly the ongoing LILAC-TIMI 76 study comparing abelacimab to placebo in stroke prevention, abelacimab could reshape the landscape of atrial fibrillation treatment, improving patient care and potentially reducing the burden of this widespread condition.
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### [LB-102 LB Pharma Segal Trial Shows Promising Phase 2 Schizophrenia Results](https://www.clinicaltrialvanguard.com/news/lb-102-lb-pharma-segal-trial-shows-promising-phase-2-schizophrenia-results/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Segal Trials, a clinical research network specializing in psychiatric disorders, significantly contributed to the successful Phase 2 trial of [LB-102](https://www.clinicaltrialvanguard.com/news/lb-pharmaceuticals-accelerates-nova-2-schizophrenia-trial-readout/), a novel antipsychotic for acute [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/). The trial, sponsored by LB Pharmaceuticals, met its primary endpoint, demonstrating the drug’s efficacy, safety, and tolerability. Two Segal Trials investigators enrolled 18% of the total study participants, exceeding their enrollment goal by 47%.
This success underscores the potential of LB-102 to address the unmet needs of schizophrenia patients who experience inadequate symptom control or intolerable side effects with existing medications. The positive results could lead to a much-needed new treatment option for this debilitating condition. Segal Trials’ high enrollment rate highlights the efficacy of their community-based outreach and patient-centric approach, a model that could influence how other research networks recruit for complex psychiatric trials.
Segal Trials randomized eight subjects per month over seven months, showcasing their specialized research infrastructure and expertise in acute schizophrenia. Their dedicated 10,000 sq ft. Miami Lakes Medical Research facility, designed for complex trials, has enrolled over 800 subjects across 50 trials since 2019. This facility allows for the segregation of special populations and adaptable configurations for various study designs.
LB Pharmaceuticals plans to discuss Phase 3 trial design with the FDA by early 2026. This positive development suggests a potential shift in the treatment landscape for schizophrenia, offering hope for improved outcomes for a significant portion of the global population affected by this disorder. The success of LB-102’s Phase 2 trial, coupled with Segal Trials’ continued enrollment efforts, positions both organizations for continued progress in advancing schizophrenia research and treatment.
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**Categories:** News
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### [Exicure and GPCR Therapeutics Announce Acquisition](https://www.clinicaltrialvanguard.com/news/exicure-and-gpcr-therapeutics-announce-acquisition/)
**Published:** January 23, 2025
**Author:** Jon Napitupulu
**Content:**
Exicure, Inc. acquired GPCR Therapeutics USA Inc. and entered into a licensing and collaboration agreement with GPCR Therapeutics Inc. to develop and commercialize GPCR’s intellectual property and patents related to G-Protein Coupled Receptors. The acquisition centers around GPCR USA’s ongoing Phase 2 clinical trial for a blood cancer treatment involving GPC-100 (Burixafor). Exicure will make milestone payments and pay royalties to GPCR based on clinical trial progress, marketing authorizations, and net sales.
This acquisition provides Exicure with a promising late-stage clinical asset in an area of significant unmet medical need – improving stem cell mobilization for patients with blood cancers undergoing hematopoietic stem cell transplantation. The faster mobilization kinetics of burixafor, compared to existing treatments, offers potential advantages for patients and healthcare providers. Positive results from the ongoing Phase 2 trial could attract further investment and position Exicure for growth after its recent restructuring.
GPCR USA, led by Dr. Pina Cardarelli, is currently conducting a Phase 2 clinical trial combining GPC-100 with propranolol and G-CSF. Interim data from 10 patients suggests this combination effectively mobilizes stem cells for transplantation, with a favorable safety profile. The trial is expected to complete patient treatment by the end of April 2025, with results announced by September. The company is also exploring a potential Phase 3 trial comparing GPC-100 to Plerixafor. Additionally, research indicates this combination therapy may enhance CAR-T cell response rates, opening possibilities for Exicure in cell and gene therapy.
This acquisition represents a strategic shift for Exicure. By obtaining a clinical-stage asset with promising data, the company moves beyond its previous focus on early-stage nucleic acid therapies. The potential for GPC-100 to improve stem cell transplantation outcomes, combined with the possibility of expanding into CAR-T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), positions Exicure for potential growth and value creation in the coming years. The success of the ongoing and future clinical trials will be a key driver of Exicure’s future in this therapeutic area.
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**Categories:** News
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### [Quoin Pharma QRX003 Shows Promising Netherton Syndrome Results](https://www.clinicaltrialvanguard.com/news/quoin-pharma-qrx003-shows-promising-netherton-syndrome-results/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
Quoin Pharmaceuticals announced further clinical evidence supporting the efficacy of [QRX003](https://www.clinicaltrialvanguard.com/news/quoin-netherton-syndrome-study-shows-positive-9-month-data/), its topical treatment for Netherton Syndrome. Data from an open-label study showed a complete reversal of positive clinical improvements after treatment discontinuation, highlighting the need for chronic QRX003 application. This reversal reinforces the treatment’s mechanism of action as a competitive broad-spectrum serine protease inhibitor, compensating for the missing LEKTI protein crucial for skin barrier function in Netherton Syndrome patients.
This news holds substantial implications for Netherton Syndrome patients, who currently lack approved therapies for this debilitating genetic disorder. The data strengthens the possibility of QRX003 becoming the first approved treatment for this condition, offering a potential solution for managing the severe skin barrier defects, recurring infections, and other associated symptoms. The demonstrated efficacy of QRX003 could significantly improve patients’ quality of life by addressing the underlying cause of the disease rather than just managing symptoms.
Key findings from the study include a return to baseline disease status within four weeks of stopping QRX003. Specifically, the Modified Ichthyosis Area of Severity Index (M-IASI), the Worst Itch Numeric Rating Scale (WINRS), and the Investigator’s Global Assessment (IGA) all regressed to pre-treatment levels after discontinuation. These data underscore the importance of continuous treatment with QRX003 for sustained clinical benefit. The company is currently conducting four clinical studies, three of which are under an Investigational New Drug (IND) application with the FDA. Quoin intends to leverage this growing body of data to support a New Drug Application (NDA) for QRX003.
This development represents a significant advancement in the treatment of Netherton Syndrome. The findings further validate the therapeutic potential of QRX003 and suggest a promising future for patients. The continued clinical development and potential regulatory approval of QRX003 could be transformative for individuals living with this rare disease, offering a much-needed treatment option and improving their long-term health outcomes. The data also supports the company’s strategy of focusing on rare and orphan diseases.
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**Categories:** News
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### [Ampligen Clinical Trial Results Published for Post-COVID Conditions](https://www.clinicaltrialvanguard.com/news/ampligen-clinical-trial-results-published-for-post-covid-conditions/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
AIM [ImmunoTech](https://www.clinicaltrialvanguard.com/news/immunotech-announces-cash-conservation-plan/) Inc. announced final clinical study results (AMP-518) for Ampligen in treating post-COVID conditions, specifically fatigue. The study, posted on [ClinicalTrials](https://www.clinicaltrialvanguard.com/article/fda-needs-to-release-clinicaltrials-gov-guidance-now/).gov, reinforces the company’s belief in Ampligen’s potential for this indication. A new analysis from the NIH’s RECOVER initiative linking Long COVID and Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) underscores the urgency of this research.
These findings are crucial due to the escalating prevalence of ME/CFS following [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) infections. The increased patient population suffering from debilitating fatigue highlights the significant unmet medical need and the potential for Ampligen to address this growing health crisis. The connection between Long COVID and ME/CFS, now quantitatively established by the NIH RECOVER data, strengthens the rationale for developing targeted therapies like Ampligen. This emphasizes the potential for positive impact on a substantial patient group and positions AIM ImmunoTech at the forefront of addressing this challenge.
Analysis of the AMP-518 trial data reveals that Ampligen-treated Long COVID patients demonstrated improved performance in the Six-Minute Walk Test (6MWT). Patients with baseline 6MWT distances less than 205 meters experienced a mean improvement of 139 meters with Ampligen compared to 91 meters with the placebo (p<0.02). This suggests Ampligen may be particularly effective for patients with moderate to severe COVID-related fatigue or ME/CFS. Future trials will likely focus on this patient subgroup. This data, coupled with the growing recognition of Long COVID-induced ME/CFS, sets the stage for further development of Ampligen. The identified target patient group allows for more focused and potentially more successful clinical trials. This could accelerate the path towards regulatory approval and ultimately provide a much-needed treatment option for those suffering from debilitating post-COVID fatigue. Source link:
**Categories:** News
**Tags:** eClinical Tech News
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### [Outlook Therapeutics ON5-5010 Wet AMD Trial Results: Efficacy & Safety Unveiled](https://www.clinicaltrialvanguard.com/news/outlook-therapeutics-on5-5010-wet-amd-trial-results-efficacy-safety-unveiled/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
[Outlook Therapeutics](https://www.clinicaltrialvanguard.com/news/complete-12-week-results-of-norse-eight-trial-for-outlook-therapeutics/) presented 12-week data from its NORSE EIGHT clinical trial of ONS-5010 (bevacizumab-vikg) for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD) at the Hawaiian Eye and Retina 2025 Meeting. The results showed ONS-5010 provided early and sustained anatomical improvements comparable to ranibizumab, with steady gains in best corrected visual acuity (BCVA) and a consistent safety profile. The company remains on track for a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) resubmission in Q1 2025.
These findings are potentially impactful for patients seeking wet AMD treatment. A bevacizumab ophthalmic formulation specifically designed for the eye, like ONS-5010, may offer advantages over currently used, repackaged versions of bevacizumab, potentially streamlining treatment and minimizing risks associated with off-label use. Positive trial results and subsequent FDA approval could broaden treatment access for wet AMD patients.
In the NORSE EIGHT trial, patients receiving ONS-5010 showed mean BCVA improvements of +3.3, +4.2, and +5.5 letters at months one, two, and three, respectively. While the trial did not meet the pre-specified non-inferiority endpoint at week eight, it did meet the non-inferiority margin at month three. Anatomical responses, measured by reduction in central retinal thickness, were comparable between ONS-5010 and ranibizumab. The safety profile of ONS-5010 remained consistent with previous NORSE trials, showing comparable ocular adverse event rates to ranibizumab and no reported cases of retinal vasculitis.
The 12-week NORSE EIGHT results, combined with data from prior NORSE studies, reinforce Outlook Therapeutics’ confidence in ONS-5010’s potential. The anticipated BLA resubmission in Q1 2025 represents a significant step towards potential U.S. approval. This approval could create a new, readily available treatment option for wet AMD, directly impacting the ophthalmology market and offering patients a dedicated ophthalmic formulation of bevacizumab. Moreover, with existing marketing authorization in the EU and UK, Outlook Therapeutics’ plans for a European launch in the first half of 2025 position the company for broader market access and potential revenue generation.
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### [MiNK Therapeutics Allo-INKTs + BOT/BAL for Advanced Gastroesophageal Cancers](https://www.clinicaltrialvanguard.com/news/mink-therapeutics-allo-inkts-bot-bal-for-advanced-gastroesophageal-cancers/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
MiNK Therapeutics announced a presentation at the ASCO GI Symposium detailing a Phase 2 trial of its iNKT [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), agenT-797, combined with Agenus’ [botensilimab](https://www.clinicaltrialvanguard.com/news/allo-inkt-combination-data-in-2l-gastric-cancer-at-aacr/)/balstilimab, [ramucirumab](https://www.clinicaltrialvanguard.com/news/alx-oncology-links-cd47-to-her2-gastric-cancer-outcomes/), and paclitaxel in patients with previously treated gastroesophageal cancers. The trial builds on earlier Phase 1 data showing promising responses and tolerability in various cancers, particularly gastric cancer. This ongoing Phase 2 trial, conducted at Memorial Sloan Kettering Cancer Center, specifically focuses on advanced gastroesophageal adenocarcinoma patients.
This research is crucial for advancing the treatment of gastroesophageal cancers, which often have poor prognoses, especially in later stages. The multi-immunologic approach, combining iNKT cell therapy with checkpoint inhibitors and standard chemotherapy, represents a potential paradigm shift in treatment. Exploring this novel combination could unlock new therapeutic avenues for patients who have exhausted existing options, potentially offering improved outcomes and extending survival.
The Phase 2 trial investigates a complex regimen of agenT-797 with botensilimab, balstilimab, ramucirumab, and paclitaxel. Prior Phase 1 data demonstrated durable responses in several cancer types, including gastric cancer, with observed T cell infiltration and expansion in responding patients. MiNK anticipates releasing further clinical updates from the ongoing Phase 2 study in the second half of 2025.
The development of this multi-immunologic combination therapy could significantly impact the landscape of gastroesophageal cancer treatment. Positive results from the Phase 2 trial could lead to accelerated clinical development, potentially offering a much-needed new treatment option for patients with advanced disease. Further, it could establish MiNK Therapeutics as a leader in iNKT cell therapy and solidify the potential of this approach for other cancer types.
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**Categories:** News
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### [Tiziana Life Sciences' Nasal Spray Shows Promise in Spinal Cord Injury Treatment](https://www.clinicaltrialvanguard.com/news/tiziana-life-sciences-nasal-spray-shows-promise-in-spinal-cord-injury-treatment/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
Tiziana [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) announced positive preclinical results for their intranasal anti-CD3 monoclonal antibody, [foralumab](https://www.clinicaltrialvanguard.com/news/tiziana-life-sciences-publishes-intranasal-foralumab-study/), in treating traumatic spinal cord injury (SCI). The treatment showed significant improvement in motor function in preclinical models after SCI, primarily by modulating microglial inflammation, a key driver of SCI pathogenesis. The company believes this nasal delivery method offers advantages in efficacy, safety, and tolerability compared to intravenous administration.
This development is potentially crucial for the SCI patient population, estimated at 300,000 in the US, with over 17,000 new cases annually. Current treatment options for SCI are limited, and often focus on managing symptoms rather than addressing the underlying inflammatory response. Foralumab’s potential to improve motor function by targeting this inflammation represents a possible shift towards disease-modifying therapies for SCI, offering new hope for patients.
Foralumab is currently in a Phase 2a trial for secondary progressive [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/). The positive preclinical SCI data suggests potential expansion of foralumab’s clinical application into a new indication with high unmet medical need. This adds to the growing body of evidence supporting the use of intranasal anti-CD3 monoclonal antibodies as a therapeutic approach for neuroinflammatory and neurodegenerative diseases.
This positive preclinical data positions Tiziana to potentially move foralumab into clinical trials for SCI, significantly broadening its therapeutic pipeline. If successful in later-stage trials, foralumab could become a much-needed treatment option for SCI, offering improved motor function and quality of life for patients. This advancement also strengthens the potential of intranasal anti-CD3 therapy as a platform for treating a range of neurological conditions.
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### [Astria Therapeutics Initiates STAR-0310 Atopic Dermatitis Trial](https://www.clinicaltrialvanguard.com/news/astria-therapeutics-initiates-star-0310-atopic-dermatitis-trial/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
Astria Therapeutics has launched a Phase 1a clinical trial for STAR-0310, a monoclonal antibody designed to treat [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (AD). The trial will evaluate the safety, tolerability, pharmacokinetics, and immunogenicity of STAR-0310 in healthy adults. Astria anticipates preliminary results in Q3 2025.
This trial initiation is a critical step in validating STAR-0310’s potential as a best-in-class AD treatment. Success in this phase would provide crucial safety and pharmacokinetic data, laying the foundation for larger trials in patients with AD. This progress could attract further investment and partnerships, accelerating development. A successful outcome could address the significant unmet need for safer and more effective AD therapies, potentially offering relief to a large patient population.
The Phase 1a trial is a randomized, double-blind, placebo-controlled study of approximately 40 healthy adult participants. Preclinical data suggests STAR-0310 has a longer half-life than typical antibodies and comparable potency to [rocatinlimab](https://www.clinicaltrialvanguard.com/news/ox40-targeted-therapy-trials-market-opportunity-insight-2026/), a competing OX40 antagonist. Importantly, STAR-0310 demonstrates significantly less antibody-dependent cellular cytotoxicity (ADCC) compared to rocatinlimab, potentially leading to a safer treatment profile.
Positive results from this Phase 1a trial will pave the way for further clinical development of STAR-0310. This could position Astria as a leader in AD therapeutics and potentially expand the use of STAR-0310 to other immunological diseases. The results will be a key inflection point, informing future development strategies and potentially reshaping the treatment landscape for AD.
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**Categories:** News
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### [Medidata & Tigermed Renew Partnership to Accelerate Global Trials](https://www.clinicaltrialvanguard.com/news/medidata-tigermed-renew-partnership-to-accelerate-global-trials/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
Medidata, a Dassault Systèmes company, and Tigermed, a global clinical research provider, extended their 13-year partnership. This renewed collaboration focuses on utilizing the Medidata Platform to enhance clinical trial efficiency, from early phases through post-market surveillance, and speed up the delivery of new therapies globally. The partnership aims to optimize study workflows, ensure regulatory compliance, and improve access to new treatments.
This continued collaboration is noteworthy because it reflects a growing trend in the [life sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) industry – the increasing reliance on technology and data-driven solutions to optimize clinical trials. The integration of platforms like Medidata’s with the operational expertise of CROs like Tigermed offers a potentially powerful synergy. Streamlining workflows, automating data capture, and reducing reliance on traditionally labor-intensive processes can significantly impact the speed and cost of bringing new therapies to market, particularly in emerging markets. This is crucial for both pharmaceutical companies seeking to accelerate drug development and patients awaiting innovative treatments.
Tigermed has already completed over 300 clinical studies using the Medidata platform across various therapeutic areas, including oncology, vaccines, and cardiovascular conditions. The expanded partnership will leverage Medidata’s technology, including AI capabilities, further automating data capture and integration across the entire study lifecycle. This will also enhance trial efficiency by better connecting patients, sites, and sponsors.
This partnership signals a continued push towards more efficient and technologically driven clinical trials. The combination of Medidata’s platform and Tigermed’s operational expertise could set a new standard for how trials are conducted, ultimately accelerating the development and availability of new treatments for patients worldwide. The incorporation of AI and further platform integration may also lead to more personalized and effective therapies in the future.
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**Categories:** News
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### [Allurion and GLP-1: Optimizing Muscle During Weight Loss Therapy](https://www.clinicaltrialvanguard.com/news/allurion-and-glp-1-optimizing-muscle-during-weight-loss-therapy/)
**Published:** January 24, 2025
**Author:** Jon Napitupulu
**Content:**
Allurion Technologies plans to launch a clinical study combining its weight-loss program with GLP-1 agonists to address muscle loss, a common side effect of GLP-1 therapy. Existing research indicates that GLP-1 treatments can lead to a 40% reduction in lean mass as part of overall weight loss, while the Allurion Balloon, combined with its virtual care platform, has shown positive results in weight reduction while preserving or even increasing muscle mass. For instance, one study showed patients gained 5.6% lean body mass while losing 14% of their total weight, and another showed a 15.7% weight reduction with no muscle mass change.
This study holds considerable potential for advancing [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatment. By tackling the issue of muscle loss associated with GLP-1 therapies, Allurion could significantly enhance the metabolic benefits of these medications. This could lead to a more comprehensive and effective approach to weight management, potentially positioning the combined therapy as a preferred option for patients and healthcare providers.
The planned study aims to demonstrate that combining the Allurion Program (balloon, virtual care suite, and connected scale) with GLP-1 therapy can result in substantial weight loss while simultaneously increasing muscle mass and improving overall body composition. This builds upon previous studies showcasing the Allurion Balloon’s ability to facilitate weight loss while preserving lean mass, contrasted with the muscle loss observed in GLP-1 therapies.
This research could reshape the landscape of obesity care. Positive results could establish this combination therapy as a new standard, offering a more holistic and potentially safer weight-loss strategy that addresses a key drawback of current GLP-1 treatments. This could further solidify Allurion’s position in the market and broaden the applicability of GLP-1 medications for a wider range of patients seeking effective and metabolically healthy weight loss.
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**Categories:** News
---
### [Transforming Clinical Trials: ActiGraph's Strategic Acquisition](https://www.clinicaltrialvanguard.com/executiveinterviews/transforming-clinical-trials-actigraphs-strategic-acquisition/)
**Published:** January 27, 2025
**Author:** Moe Alsumidaie
**Content:**
In this conversation with Christine Guo, Chief Scientific Officer at ActiGraph, we explored the impact of Actigraph’s acquisition of Biofourmis Connect. Christine shared insights on how this integration will enhance AI-driven analytics, modernize clinical trials, and reinforce ActiGraph’s commitment to patient-centricity.
## [](#moe-how-will-ai-driven-analytics-enhance-actigraphs-digital-health-ecosystem-post-acquisition)**Moe: How will AI-driven analytics enhance ActiGraph’s digital health ecosystem post-acquisition?**
The acquisition of Biofourmis Connect significantly broadens our platform capabilities, allowing us to integrate a broader range of sensor data modalities. AI thrives on high-quality, diverse data, and this acquisition enables us to collect essential data from sources like ECG patches and continuous high-frequency blood pressure measurements. These data points are crucial for building robust AI models to predict and monitor patient health more effectively. For instance, we acquired 21 algorithms from Biofourmis, including FDA-cleared ones like Biovitals and RhythmAnalytics. These algorithms enhance our ability to monitor safety in clinical
Christine Guo, Chief Scientific Officer at Actigraph
trials by detecting deviations from normal health patterns, which can indicate medical crises. This capability is particularly valuable in oncology, where managing adverse events like cytokine release syndrome (CRS) is critical. By leveraging these advanced analytics, we can improve patient monitoring and potentially decentralize trials, reducing the need for hospital stays and alleviating resource strains.
## [](#moe-how-will-actigraph-and-biofourmis-connect-modernize-clinical-trials-with-remote-monitoring)**Moe: How will ActiGraph and Biofourmis Connect modernize clinical trials with remote monitoring?**
Integrating Biofourmis Connect with ActiGraph’s existing platform is a game-changer for clinical trials. Traditionally, trials are complex and require a cohesive platform to manage various data elements. With this acquisition, we can offer a comprehensive solution that supports multiple aspects of a trial, from wearables to eConsent and virtual visits. This reduces the operational burden on sponsors and ensures a seamless data collection process. For example, we were limited to supporting specific data elements like wearables before this integration. Now, we can manage a broader range of digital solutions, streamlining the trial process and enhancing data coherence. This holistic approach accelerates trials and improves the quality and reliability of the collected data, ultimately leading to faster and more accurate trial outcomes.
## [](#moe-how-does-the-acquisition-align-with-actigraphs-commitment-to-patient-centricity)**Moe: How does the acquisition align with ActiGraph’s commitment to patient centricity?**
Patient centricity is at the core of ActiGraph’s mission, and this acquisition aligns perfectly with that commitment. By expanding our capabilities to collect patient-generated data in home environments, we can significantly reduce the burden on patients who would otherwise need to visit clinics for data collection. Previously, our technology was primarily used to assess drug efficacy, such as physical activity or sleep improvements. However, with the new capabilities from Biofourmis, we can also focus on safety profiles, allowing us to monitor patients remotely and identify potential adverse events without requiring them to stay in clinics. This enhances the patient experience by making participation in trials more convenient and broadens our ability to collect comprehensive data that reflects real-world patient experiences.
## [](#moe-how-is-actigraph-ensuring-compliance-with-fda-guidelines-on-digital-health-technologies)**Moe: How is ActiGraph ensuring compliance with FDA guidelines on digital health technologies?**
Compliance with regulatory guidelines is paramount, and acquiring Biofourmis Connect strengthens our regulatory capabilities. The Biofourmis platform is built around necessary regulatory compliance for clinical trials, and many of their devices and algorithms are already FDA-cleared. For instance, some of our acquired algorithms have been cleared through the FDA’s 510(k) pathway. Additionally, Biofourmis has been proactive in engaging with the FDA, using pre-submission mechanisms to discuss advanced solutions like early detection algorithms for adverse events. This proactive approach enhances our regulatory portfolio and ensures that we remain at the forefront of compliance as [digital health technologies](https://www.clinicaltrialvanguard.com/article/unpacking-fda-guidance-on-digital-health-technologies-in-clinical-trials/) evolve.
## [](#moe-what-are-actigraphs-plans-for-leveraging-synergies-from-this-acquisition)**Moe: What are ActiGraph’s plans for leveraging synergies from this acquisition?**
Moving forward, we will focus on oncology and immunotherapy, areas with a significant unmet need. The integration is already yielding results; just weeks after the acquisition, we’ve had client inquiries where the Biofourmis Connect platform perfectly addresses their needs. For example, we’ve been able to offer solutions that meet client requirements and save costs by reusing wearables. This rapid integration demonstrates the potential for developing new digital biomarkers and expanding into therapeutic areas where advanced monitoring and data collection can make a substantial impact.
**Categories:** Article: Executive Interviews
---
### [Exelixis' Zanzalintinib Shows Promise in Metastatic Colorectal Cancer Trial](https://www.clinicaltrialvanguard.com/news/exelixis-zanzalintinib-shows-promise-in-metastatic-colorectal-cancer-trial/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Exelixis announced results from the [STELLAR](https://www.clinicaltrialvanguard.com/news/phase-3-stellar-study-shows-improvements-in-rare-brain-tumor/)-001 trial, a Phase 1b/2 study evaluating zanzalintinib, alone or combined with [atezolizumab](https://www.clinicaltrialvanguard.com/news/xilio-announces-updated-phase-2-data-for-vilastobart/), in patients with previously treated metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (CRC). The combination therapy showed numerically improved progression-free survival (PFS) and overall survival (OS) compared to zanzalintinib alone. This is particularly notable in a subgroup of patients without liver metastases, where the combination demonstrated a substantial PFS improvement.
These findings are important because they suggest a potential new treatment option for metastatic CRC, a disease with limited effective therapies, especially for patients who have progressed after multiple prior treatments. The improved outcomes observed in patients without liver metastases are particularly encouraging, as this subgroup often has a poorer prognosis. This also highlights the potential for personalized treatment strategies based on specific patient characteristics.
The STELLAR-001 trial randomized 107 patients to receive either zanzalintinib alone or in combination with atezolizumab. In the overall population, the median PFS was 3.0 months with zanzalintinib and 4.0 months with the combination. In patients without liver metastases, the median PFS was significantly longer at 8.2 months with the combination versus 3.3 months with zanzalintinib alone. While the OS data showed numerical improvement with the combination therapy, the results were not statistically significant. Safety profiles were generally manageable, with comparable rates of grade 3/4 treatment-related adverse events in both treatment arms.
The results from STELLAR-001 support the ongoing Phase 3 STELLAR-303 trial, which is comparing zanzalintinib plus atezolizumab to regorafenib in metastatic CRC. Data from STELLAR-303 are expected in the second half of 2025 and will be crucial to determine if the combination therapy translates into a clinically meaningful benefit for patients with this challenging cancer. The findings from STELLAR-303 could potentially reshape the treatment landscape for metastatic CRC, offering a new therapeutic option with improved efficacy.
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**Categories:** News
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### [Opdivo + Yervoy vs Opdivo: CheckMate - 8HW Results](https://www.clinicaltrialvanguard.com/news/opdivo-yervoy-vs-opdivo-checkmate-816-results/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
A Phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/)-8HW trial analysis revealed that Opdivo plus Yervoy significantly improved progression-free survival (PFS) compared to Opdivo alone in patients with microsatellite instability-high/mismatch repair-deficient (MSI-H/dMMR) metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC). At a median 47-month follow-up, the combination therapy showed a 38% reduction in the risk of disease progression or death. This builds upon prior CheckMate-8HW findings where Opdivo plus Yervoy outperformed chemotherapy, leading to European Commission approval for first-line treatment of MSI-H/dMMR mCRC.
This analysis is crucial for mCRC patients with MSI-H/dMMR tumors, as it demonstrates the superior efficacy of dual immunotherapy (Opdivo plus Yervoy) over single-agent Opdivo. This provides clinicians with stronger evidence supporting the use of combination therapy in this patient population, potentially leading to improved treatment outcomes and a new standard of care. This further strengthens the position of dual immunotherapy within the broader oncology landscape, where it has shown benefits across various tumor types.
The data highlights a statistically significant improvement in PFS (HR 0.62; 95% CI 0.48–0.81; P = 0.0003) with Opdivo plus Yervoy compared to Opdivo monotherapy. Importantly, the safety profile of the combination remained consistent with previous findings, with no new safety signals observed. The observed increase in treatment-related adverse events was primarily mild to moderate (Grade 1 or 2). The study continues to evaluate overall survival, a key secondary endpoint.
These findings solidify the role of Opdivo plus Yervoy as a potential first-line treatment option for all patients with MSI-H/dMMR mCRC, regardless of prior treatment. This may shift clinical practice towards earlier utilization of combination immunotherapy, ultimately aiming to improve long-term survival for these patients. The ongoing assessment of overall survival will be vital in further confirming the long-term benefits of this approach and influencing treatment guidelines.
Source link: [http://www.businesswire.com/news/home/20250124556902/en/Bristol-Myers-Squibb-Presents-Results-from-CheckMate–8HW-Analysis-Evaluating-Opdivo%C2%AE-nivolumab-plus-Yervoy%C2%AE-ipilimumab-Compared-to-Opdivo-Monotherapy…](http://www.businesswire.com/news/home/20250124556902/en/Bristol-Myers-Squibb-Presents-Results-from-CheckMate--8HW-Analysis-Evaluating-Opdivo%C2%AE-nivolumab-plus-Yervoy%C2%AE-ipilimumab-Compared-to-Opdivo-Monotherapy...)
**Categories:** News
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### [Firefly Neuroscience Publishes EEG Analytics in Drug Development](https://www.clinicaltrialvanguard.com/news/firefly-neuroscience-publishes-eeg-analytics-in-drug-development/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Firefly Neuroscience highlighted two studies demonstrating the potential of its FDA-cleared Brain Network Analytics (BNA™) platform in drug development and neuropsychiatric care. The studies utilized advanced resting EEG and cognitive EEG (ERP) data analytics to objectively measure treatment efficacy and cognitive change. The research focused on the impact of Novartis’ MIJ821 and vortioxetine on brain activity and cognitive function in patients with [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD).
These findings are crucial for advancing brain health research and treatment. The ability to objectively measure drug efficacy and cognitive changes using EEG offers a significant advantage over traditional subjective assessments. This can lead to more efficient drug development, personalized treatment strategies, and improved patient outcomes in neuropharmacology and psychiatry. Specifically, the ability to track cognitive changes independent of mood changes offers a new dimension in understanding and treating MDD, a complex disorder where cognitive deficits often persist even after mood improvement.
The first study, conducted by Novartis, used EEG to assess the impact of MIJ821, revealing dose-dependent changes in brain activity consistent with ketamine’s antidepressant effects. This supports EEG’s role as a pharmacodynamic and pharmacokinetic biomarker tool. The second study demonstrated the utility of Firefly’s BNA™ system in measuring cognitive changes in MDD patients treated with vortioxetine. Baseline brain activation latencies, which were prolonged in MDD patients, normalized post-treatment, indicating improved cognitive function.
These studies underscore the potential of Firefly’s BNA™ platform to transform how neurological and mental disorders are diagnosed and treated. The technology’s ability to provide objective, quantifiable data on brain activity and cognitive function can accelerate drug development, enable personalized treatment approaches, and ultimately improve patient care by providing clinicians with more precise tools for assessment and treatment optimization. The ability to scale EEG analysis through automation makes it a viable tool for broader clinical application, potentially revolutionizing the field of mental healthcare.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Axiom, Brain Health Platform Foundation Partner for Alzheimer's Research](https://www.clinicaltrialvanguard.com/news/axiom-brain-health-platform-foundation-partner-for-alzheimers-research/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Axiom Brain Health, in partnership with the Global Alzheimer’s Platform Foundation (GAP), launched its newly renovated research facility in Tampa, Florida. The expansion aims to address the growing need for research on neurodegenerative diseases like Alzheimer’s, [Multiple Sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/), and [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/). The event highlighted the importance of volunteers, or “citizen scientists,” in advancing this research.
This expansion is crucial for the Tampa community and the broader fight against neurodegenerative diseases. The increased capacity allows Axiom to conduct more clinical trials, potentially accelerating the development of new therapies and diagnostic tools. With an estimated 580,000 individuals currently living with Alzheimer’s in Florida and projections for significant increases, the expanded facility positions Axiom to directly address this growing public health crisis. The emphasis on volunteer participation also underscores the vital role the community can play in advancing research.
The renovated facility has nearly tripled in size, from 1,700 to 3,900 square feet, and now houses 14 research experts, up from the original team of six. This expansion significantly increases the site’s capacity for conducting complex clinical trials and collecting valuable data. The growth not only benefits research efforts but also offers more opportunities for local residents to participate in cutting-edge studies.
This expansion represents a significant step forward in neurodegenerative disease research. The larger facility, increased staff, and emphasis on community involvement create a strong foundation for accelerating the development of much-needed treatments and improving the lives of those affected by these debilitating conditions. The continued collaboration between Axiom and GAP promises to further enhance research efforts and broaden access to clinical trials for the Tampa community.
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**Categories:** News
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### [Elevated Therapeutics Post-Hoc Analysis of Etiology Impact on HCC Survival](https://www.clinicaltrialvanguard.com/news/elevated-therapeutics-post-hoc-analysis-of-etiology-impact-on-hcc-survival/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Elevar Therapeutics announced positive results from a post-hoc analysis of the CARES-310 study, showing that camrelizumab plus [rivoceranib](https://www.clinicaltrialvanguard.com/clinops-watchdog/three-strikes-same-deficiency-how-elevar-therapeutics-turned-a-solvable-manufacturing-problem-into-a-patient-crisis/) provides a clinically meaningful overall survival (OS) and progression-free survival (PFS) benefit for patients with unresectable hepatocellular carcinoma (uHCC), regardless of whether the underlying cause is viral (hepatitis B or C) or non-viral. These findings were presented at the 2025 American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium (ASCO GI). The analysis demonstrated improved OS and PFS with the combination therapy compared to [sorafenib](https://www.clinicaltrialvanguard.com/news/cares-310-study-final-analysis-published-in-the-lancet/) across all etiology subgroups.
This news is particularly encouraging for uHCC patients, who often face a poor prognosis and limited treatment options. Demonstrating consistent efficacy across different etiologies broadens the potential patient population who could benefit from this combination therapy. This strengthens the argument for camrelizumab plus rivoceranib as a valuable first-line treatment option, potentially offering a new standard of care.
The post-hoc analysis revealed longer median OS with camrelizumab plus rivoceranib compared to sorafenib in patients with non-viral (HR 0.68), HCV (HR 0.37), and HBV (HR 0.70) etiologies. Similarly, median PFS was longer with the combination therapy across all three etiology subgroups (non-viral HR 0.55, HCV HR 0.50, and HBV HR 0.57). The combination therapy’s safety profile remained consistent and manageable across these subgroups. These results build upon previous positive findings from the CARES-310 study, further solidifying the efficacy and safety profile of this combination.
These positive findings support the potential for FDA approval of camrelizumab plus rivoceranib, which is currently under review with a PDUFA date of March 20, 2025. If approved, this combination therapy could significantly improve outcomes for a broader range of uHCC patients and reshape the treatment landscape for this challenging cancer. This could also position Elevar Therapeutics for significant growth and solidify their presence in the oncology market.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Eyonis™ AI Lung Cancer Diagnostic Secures €47.5M Funding](https://www.clinicaltrialvanguard.com/news/eyonis-ai-lung-cancer-diagnostic-secures-e47-5m-funding/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Median Technologies secured a non-binding term sheet with the European Investment Bank (EIB) for a loan of up to €37.5 million and signed a €10 million equity line with IRIS Capital Investment. The funding will support eyonis™ [Lung Cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) Screening (LCS) software’s FDA approval, CE marking, and commercialization partnerships in the U.S. The initial €4 million from IRIS, combined with an extension of an existing EIB loan and operational improvements, extends Median’s cash runway into Q4 2025.
This development is crucial for advancing lung cancer diagnostics. Early detection is critical for successful lung cancer treatment, but current diagnostic processes can be inefficient and complex. Median’s eyonis™ LCS software uses AI-powered analysis of low-dose CT scans to detect lung cancer at its earliest stages, potentially improving patient outcomes and reducing healthcare costs associated with late-stage treatment. Funding secures the resources needed to navigate regulatory hurdles and bring this promising technology to market.
The EIB loan is structured in tranches tied to undisclosed milestones, which, if achieved, could extend Median’s financial runway into 2026. The IRIS equity line offers an immediate cash injection and flexibility for Median to control drawdown. Median is also actively pursuing commercialization partnerships with leading U.S. AI diagnostic companies for eyonis™ LCS. In Q1 2025, Median anticipates releasing pivotal data from the [RELIVE](https://www.clinicaltrialvanguard.com/news/eyonis-lung-cancer-screening-meets-primary-endpoint-in-relive-trial/) study, which validates eyonis™ LCS’s clinical performance. Regulatory filings for FDA clearance and CE marking are planned for Q2 2025, with anticipated authorizations in Q3 2025 (U.S.) and Q1 2026 (EEA).
This combination of funding, strategic partnerships, and positive clinical data positions Median Technologies for significant growth. The successful commercialization of eyonis™ LCS could transform lung cancer screening, offering a more efficient and accurate diagnostic tool for healthcare providers, ultimately benefiting patients through earlier detection and improved treatment outcomes. The upcoming milestones, including regulatory approvals and commercial partnerships, will be critical for determining the long-term success of eyonis™ LCS and its impact on the lung cancer diagnostics market.
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**Categories:** News
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### [Exelixis Announces Results from CABINET Subgroup Analysis at ASCO GI 2025](https://www.clinicaltrialvanguard.com/news/exelixis-announces-results-from-cabinet-subgroup-analysis-at-asco-gi-2025/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Exelixis announced positive results from a subgroup analysis of the phase 3 [CABINET](https://www.clinicaltrialvanguard.com/news/exelixis-announces-phase-3-cabinet-study-results-unveiled-at-esmo-2024/) study, showing [cabozantinib](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/) improved progression-free survival (PFS) compared to placebo in patients with advanced gastrointestinal (GI) neuroendocrine tumors (NET). This subgroup, comprising 116 of the 203 extra-pancreatic NET patients, represents a common and challenging-to-treat form of NET with limited treatment options after disease progression. The FDA is currently reviewing a supplemental New Drug Application for cabozantinib for the treatment of advanced NET.
This news is important because it provides clinicians with potential new treatment options for a patient population facing limited therapeutic choices. Advanced GI NETs represent a significant portion of neuroendocrine tumor cases, and effective therapies are crucial to improving patient outcomes. The positive PFS results suggest cabozantinib could address this unmet need and potentially become a standard of care.
In the subgroup analysis, cabozantinib reduced the risk of disease progression or death by 50% compared to placebo. Median PFS was 8.5 months with cabozantinib versus 5.6 months with placebo. The safety profile of cabozantinib in this subgroup was consistent with its known safety profile, with no new safety signals identified. The most common grade 3/4 adverse events included hypertension, diarrhea, and fatigue.
The positive results from this subgroup analysis strengthen the overall findings of the CABINET trial, which led to the trial being stopped early due to substantial PFS improvement. Pending FDA approval, cabozantinib could become a valuable new therapy for patients with advanced GI NET, offering a much-needed treatment option and potentially reshaping the treatment landscape for this challenging cancer type.
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**Categories:** News
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### [Allurion Raises $7.4M in At-the-Market Offering](https://www.clinicaltrialvanguard.com/news/allurion-raises-7-4m-in-at-the-market-offering/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Allurion Technologies, a company focused on [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatment, announced a registered direct offering of 1,240,000 shares of common stock at $6.00 per share, raising approximately $7.4 million before expenses. Concurrent with the offering, Allurion will issue warrants for an equal number of shares at the same exercise price, subject to shareholder approval for repricing existing warrants. The company plans to allocate the proceeds toward working capital and general corporate purposes.
This capital infusion is crucial for Allurion as it navigates a competitive weight-loss market increasingly influenced by GLP-1 drugs. Strengthening the company’s financial position allows for greater flexibility in research and development, particularly as Allurion continues its clinical studies, including those combining its gastric balloon with GLP-1 agonists. This funding could also be instrumental in supporting commercialization efforts for the Allurion Program, especially considering the Allurion Gastric Balloon is still investigational in the United States.
The offering involves a registered direct placement with institutional investors and a concurrent private placement of warrants. Roth Capital Partners is acting as the exclusive placement agent. Closing is expected around January 27, 2025, contingent upon customary closing conditions and shareholder approval of warrant repricing. The offering leverages an existing shelf registration, streamlining the process.
This financing provides Allurion with resources to advance its mission of combating obesity. The company’s ability to execute on its clinical development and commercialization strategy, particularly in the US market, will be key to its long-term success. The impact of ongoing clinical trials and the evolving competitive landscape will significantly influence Allurion’s future prospects.
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**Categories:** News
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### [Ocugen's OCU500 Inhaled COVID-19 Vaccine Enters Phase 1 Trial](https://www.clinicaltrialvanguard.com/news/ocugens-ocu500-inhaled-covid-19-vaccine-enters-phase-1-trial/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Ocugen has received FDA approval to begin a Phase 1 clinical trial for OCU500, an inhaled and nasal spray vaccine for [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/). The trial, sponsored by the National Institute of Allergy and Infectious Diseases ([NIAID](https://www.clinicaltrialvanguard.com/news/niaid-awards-14m-for-institutional-review-services-to-advarra/)), will assess the vaccine’s safety, tolerability, and immunogenicity in 80 adult participants. This development leverages a novel chimpanzee adenovirus-vectored (ChAd36) technology for mucosal delivery, aiming to provide more durable protection against COVID-19.
Although the pandemic has officially ended, COVID-19 remains a serious health concern, causing significant hospitalizations and deaths. Developing a more effective and longer-lasting vaccine is crucial for mitigating the ongoing impact of the virus and preventing future surges. A mucosal vaccine, delivered directly to the respiratory system, offers a potentially superior approach to combatting COVID-19 compared to traditional intramuscular injections, by stimulating immunity at the point of viral entry.
The Phase 1 trial will involve two dose groups, with participants receiving either the inhaled or intranasal version of OCU500. This technology, licensed from Washington University in St. Louis, has shown promising results in earlier studies, including increased antibody production and sustained immune response with a lower dose compared to intramuscular administration. NIAID, through Project NextGen, will fully fund the trial, and Ocugen will retain rights to the findings. Ocugen plans to utilize this platform for other respiratory diseases like influenza and RSV.
This FDA approval marks a significant step forward for Ocugen and the development of next-generation COVID-19 vaccines. Positive results from the Phase 1 trial could pave the way for further clinical development and potentially offer a more effective and convenient method for protecting against COVID-19 and other respiratory illnesses. This innovative approach may be pivotal in mitigating the long-term impact of COVID-19 and improving preparedness for future respiratory virus outbreaks.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Adagene Announces Updated Data From Phase 1b/2 Study of Muza in Colorectal Cancer at ASCO GI](https://www.clinicaltrialvanguard.com/news/adagene-announces-updated-data-from-phase-1b-2-study-of-muza-in-colorectal-cancer-at-asco-gi/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Adagene Inc. announced positive updated clinical data for [ADG126](https://www.clinicaltrialvanguard.com/news/improved-durability-seen-for-muzastotug-combo-in-crc-study/) (muzastotug), an anti-CTLA-4 SAFEbody® drug, combined with pembrolizumab in a Phase 1b/2 trial for microsatellite stable [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (MSS CRC). The combination therapy demonstrated a 33% overall response rate in twelve patients receiving a 20 mg/kg loading dose followed by 10 mg/kg every three weeks, with no Grade 4/5 treatment-related adverse events or discontinuations. The study focuses on MSS CRC, a challenging cancer type with limited treatment options.
This news is particularly encouraging because MSS CRC is notoriously resistant to current immunotherapies. The 33% overall response rate observed with ADG126 plus pembrolizumab suggests a potential breakthrough for this patient population. The manageable safety profile, with no high-grade adverse events or discontinuations, further enhances the drug’s potential as a viable treatment option. This progress could lead to a much-needed new therapy for a cancer with a historically poor prognosis.
The technical highlight of this update is the improved overall response rate achieved with the loading dose regimen of ADG126. The previously reported overall response rate at a consistent 10 mg/kg dose was 23%. The increase to 33% with the loading dose suggests a potential benefit from achieving higher initial drug concentrations. Importantly, all responders remained on treatment at the time of the announcement. Adagene plans to expand the study to include patients with liver metastases and explore standard-of-care combinations, indicating confidence in ADG126’s therapeutic index. The most common treatment-related adverse event was pruritus (25%), and higher-grade adverse events were managed with dose modifications and limited medical interventions.
This positive data reinforces ADG126’s potential as a best-in-class CTLA-4 inhibitor. The results warrant continued development and could lead to a new therapeutic option for MSS CRC patients, a population with significant unmet medical needs. The planned expansion of the trial, including patients with liver metastases, will provide further insights into ADG126’s efficacy and safety profile in a broader patient population and potentially establish it as a key component in combination therapies.
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**Categories:** News
**Tags:** eClinical Tech News
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### [FDA Grants Cartesian Special Protocol for Phase 3 Trial in Myasthenia Gravis](https://www.clinicaltrialvanguard.com/news/fda-grants-cartesian-special-protocol-for-phase-3-trial-in-myasthenia-gravis/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
Cartesian Therapeutics (NASDAQ: RNAC) received FDA approval under the Special Protocol Assessment (SPA) process for the Phase 3 AURORA trial design of Descartes-08, its mRNA [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) candidate for [myasthenia gravis](https://www.clinicaltrialvanguard.com/news/regenerons-cemdisiran-accepted-for-fda-review-in-generalized-myasthenia-gravis/) (MG). The SPA agreement confirms the trial design is suitable for a future [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/), pending trial results. The Phase 3 trial is expected to begin in the first half of 2025.
This FDA approval is a significant step forward for Cartesian and potentially for MG patients. It de-risks the Phase 3 trial by confirming alignment with the FDA on trial design and endpoints, increasing the likelihood of a successful regulatory pathway. This clarity also enables Cartesian to focus resources on executing the trial efficiently. For MG patients, the advancement of Descartes-08 offers hope for a new treatment option with a potentially improved safety profile compared to existing therapies, as it avoids the need for preconditioning chemotherapy and facilitates outpatient administration.
The Phase 3 AURORA trial is a randomized, double-blind, placebo-controlled study comparing Descartes-08 to a placebo in approximately 100 participants with AChR Ab+ MG. Participants will receive six weekly infusions. The primary endpoint is the proportion of participants achieving a three-point or greater improvement in the MG-ADL score at four months. This follows positive Phase 2b results where patients experienced an average MG-ADL reduction of 5.5 points at four months, with good tolerability.
This SPA agreement sets a clear path for potential market entry of Descartes-08. Positive Phase 3 results could lead to regulatory approval and offer a new therapeutic option for MG patients, positioning Cartesian as a leader in mRNA cell therapy for autoimmune diseases. The upcoming Phase 3 trial will be crucial in confirming the efficacy and safety profile observed in earlier trials and ultimately determining the future of Descartes-08.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [RenovoRx ASCO GI 2025: Promising Pharmacokinetic Data](https://www.clinicaltrialvanguard.com/news/renovorx-asco-gi-2025-promising-pharmacokinetic-data/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
[RenovoRx](https://www.clinicaltrialvanguard.com/news/renovorx-tiger-pac-trial-surpasses-100-patient-milestone/) announced promising pharmacokinetic data from a sub-study of its ongoing Phase III TIGeR-PaC clinical trial, investigating its Trans-Arterial Micro-Perfusion (TAMP) therapy platform for locally advanced [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) (LAPC). The sub-study compared intra-arterial gemcitabine (IAG) delivered via the RenovoCath system with the current standard of care, intravenous gemcitabine. Results indicate that IAG via TAMP reduces systemic gemcitabine levels, potentially increasing local drug potency and minimizing systemic side effects.
This data is crucial for advancing the treatment of LAPC, a notoriously difficult-to-treat cancer with limited effective options. The findings suggest that TAMP could offer a more targeted and potentially less toxic approach to chemotherapy delivery, directly addressing a significant clinical need. Improved local drug concentration could lead to better tumor control and potentially improved patient outcomes. Reducing systemic exposure holds the promise of lessening the debilitating side effects often associated with chemotherapy, thereby improving patients’ quality of life during treatment.
The sub-study, part of the larger TIGeR-PaC trial, analyzed pharmacokinetic data from a sample of trial participants. It directly compared IAG administered via RenovoCath and TAMP with intravenous gemcitabine. The data demonstrated a decrease in systemic gemcitabine levels with the IAG/TAMP approach, suggesting localized drug delivery. The primary endpoint of the main TIGeR-PaC trial is overall survival, with secondary endpoints focusing on reduced side effects compared to standard care. The first interim analysis, completed in March 2023, recommended continuing the study. The second interim analysis, triggered by the 52nd patient death, is anticipated in the first half of 2025. RenovoRx also aims to complete patient enrollment within the same timeframe.
This positive pharmacokinetic data strengthens the potential of RenovoRx’s TAMP platform as a viable treatment option for LAPC. Confirmation of these findings in the final analysis of the TIGeR-PaC trial could significantly impact the LAPC treatment landscape, offering a more targeted and potentially less toxic chemotherapy approach. It could also validate TAMP as a platform technology applicable to other cancer types. Pending successful trial completion and regulatory approval, RenovoRx’s approach holds promise for improved patient outcomes and quality of life in this challenging disease.
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**Categories:** News
---
### [Lerodalcibep Shows Promise in HoFH Phase 3 Study](https://www.clinicaltrialvanguard.com/news/lerodalcibep-shows-promise-in-hofh-phase-3-study/)
**Published:** January 27, 2025
**Author:** Jon Napitupulu
**Content:**
[LIB Therapeutics](https://www.clinicaltrialvanguard.com/news/lib-therapeutics-hasten-biopharmaceuticals-lerodalcibep-trial-approved-in-china/)‘ Phase 3 LIBerate-HoFH study results for lerodalcibep, a novel PCSK9 inhibitor, in patients with homozygous familial hypercholesterolemia (HoFH) were published in Lancet Diabetes & Endocrinology. The study, a randomized, crossover trial, compared lerodalcibep to evolocumab in a diverse global population of HoFH patients aged 10 and older. The primary endpoint was the percent change from baseline in LDL cholesterol (LDL-C).
These results are critical for the HoFH patient population, which faces exceptionally high LDL-C levels and significantly increased risk of premature cardiovascular disease. Current treatment options often prove insufficient, highlighting the urgent need for new therapies. This study provides valuable data on a potential new therapeutic approach, specifically in a diverse and global cohort, offering insights that could improve treatment strategies and outcomes.
The study found that mean LDL-C reduction with lerodalcibep was -9.1% and -10.8% with evolocumab when averaged across monthly visits. At week 12, reductions were -12% and -11.5% respectively, and at week 24, -4.9% and -10.3% respectively. While there was significant variability in individual patient responses, the percent change in LDL-C over 24 weeks correlated well between the two drugs. Interestingly, genotyping and free PCSK9 suppression did not predict treatment response. Both drugs showed good tolerability profiles, with no treatment-related serious adverse events.
This study represents a crucial step forward in the development of lerodalcibep as a potential treatment option for HoFH. While the LDL-C reductions observed were less than initially hoped, the data contributes substantially to the understanding of PCSK9 inhibition in this challenging patient population. The findings underscore the need for personalized approaches to HoFH treatment and emphasize the importance of continued research in this area. The data also supports the potential use of lerodalcibep as a convenient, monthly injectable therapy for HoFH, potentially expanding the available options for patients. The company’s recent BLA submission to the FDA signifies their commitment to bringing this therapy to market and potentially improving the lives of individuals with HoFH and other forms of hyperlipidemia.
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**Categories:** News
---
### [Eisai's Innovative Bayesian Approach Accelerates Alzheimer's Drug Approval](https://www.clinicaltrialvanguard.com/conference-coverage/eisais-innovative-bayesian-approach-accelerates-alzheimers-drug-approval/)
**Published:** January 28, 2025
**Author:** Moe Alsumidaie
**Content:**
At the [DPHARM conference](https://dpharmconference.com/ "DPHARM conference"), Eisai Inc. showcased their groundbreaking use of Bayesian methods to achieve accelerated approval for their Alzheimer’s drug, [LEQEMBI](https://www.clinicaltrialvanguard.com/news/fda-approves-leqembi-iqlik-subcutaneous-injection-for-early-alzheimers/)™ ([lecanemab](https://www.clinicaltrialvanguard.com/news/lecanemab-subcutaneous-autoinjector-shows-bioequivalent-exposure-to-iv-form/)-irmb). Led by Dr. Shobha Dhadda and Dr. Lynn D. Kramer, the session delved into the intricacies of Bayesian clinical trial design and its significant impact on drug development, offering a compelling case study in innovation.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#the-evolution-of-bayesian-design)The Evolution of Bayesian Design
Dr. Lynn D. Kramer began by providing a comprehensive overview of Bayesian theory, tracing its development from the 1970s to its eruption in the 2000s. He highlighted the contributions of Don Barry, a pioneer in Bayesian design, who played a crucial role in advancing the methodology. During the initial decades, Bayesian theory was primarily in the development and discussion phase, with researchers trying to understand its potential applications. By the early 2000s, Bayesian methods began to gain traction, particularly in clinical trials. Kramer pointed out several key studies that utilized Bayesian designs, such as the I-SPY trials and the Trulicity trials, demonstrating this approach’s practical benefits.
The FDA also started developing strategies for using Bayesian designs, recognizing their potential to improve trial efficiency and outcomes. Kramer mentioned that the FDA had even released draft guidance focusing on the advantages of Bayesian design. However, it was still in the draft phase, which is common with many FDA guidelines. According to Kramer, one of the significant advantages of Bayesian design is its ability to conserve placebo patients. In traditional trials, a significant number of participants are assigned to placebo groups, which can be a deterrent for patient enrollment. Bayesian adaptive designs, however, can adjust the allocation of patients based on interim results, increasing the likelihood that participants receive potentially therapeutic drugs rather than placebos. This improves patient satisfaction and enhances the ethical aspects of clinical trials.
## [](#the-case-of-leqembi-a-bayesian-success-story)The Case of LEQEMBI™: A Bayesian Success Story
Dr. Shobha Dhadda explained why Eisai chose a Bayesian design for their Alzheimer’s trials. She noted that the shift towards early [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/) phases, such as mild cognitive impairment (MCI) and mild Alzheimer’s disease (AD), necessitated a robust proof-of-concept study before advancing to large, expensive Phase III trials. Traditional designs have led to numerous failures, with over 30-40 drugs failing in the last two decades due to inadequate Phase II studies. Dhadda elaborated that one of the key reasons for these failures was the lack of a strong clinical proof of concept before moving into Phase III trials. Many studies had relied solely on biomarker data, which, while useful, did not provide a comprehensive understanding of the drug’s clinical efficacy.
Eisai was determined to avoid this pitfall by ensuring a robust Phase II study for lecanemab. Dhadda explained that there were still many unknowns about lecanemab at the time, including the optimal dose and regimen. The team considered several doses and regimens and needed to determine the study’s duration, especially given the chronic and slow-moving nature of early-stage Alzheimer’s disease. In such a scenario, where the placebo curve might not show significant movement, identifying the right dose and demonstrating a treatment effect becomes particularly challenging. To address these challenges, Eisai decided to employ a Bayesian adaptive design. Dhadda highlighted that this approach allowed them to identify the optimal dose and regimen while minimizing costs and sample size.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#the-mechanics-of-bayesian-adaptive-design)The Mechanics of Bayesian Adaptive Design
Dr. Dhadda elaborated on the mechanics of Bayesian adaptive design, explaining the concept of response adaptive randomization. Unlike traditional fixed allocation, Bayesian design allocates more subjects to the most effective dose arms based on an algorithm. This approach allows for multiple interim analyses, focusing on safety and efficacy. Dhadda explained that in a traditional design, the allocation of subjects to different arms is fixed at the beginning of the study. For example, in a study with two or three arms, the randomization might be set at a 1:1 or 1:2 ratio, and this allocation remains constant throughout the study.
In contrast, a Bayesian adaptive design uses an algorithm to adjust the allocation of subjects based on interim results. This means the dose arm showing the most promise receives more subjects, while less effective dose arms receive fewer subjects. Dhadda clarified a common misconception about Bayesian adaptive designs: once subjects are assigned to a dose arm, they remain in that arm for the study’s duration. The adaptive allocation only applies to new subjects entering the study. This ensures that the study maintains its integrity and results are reliable. The Bayesian design also allows for multiple interim analyses, which can be conducted for various purposes, such as assessing safety or efficacy.
## [](#predicting-phase-iii-success)Predicting Phase III Success
The Bayesian Phase II trial for lecanemab perfectly predicted the outcomes of the Phase III trial, known as Clarity AD. The Phase II trial’s adaptive design identified the most effective dose regimens validated in the larger Phase III trial. This seamless transition from Phase II to Phase III was a testament to the power of Bayesian methods in drug development. Dr. Kramer explained that the Phase II trial was designed to examine the dose-response and dosing frequency based on the pharmacokinetic (PK) and pharmacodynamic (PD) models. The team wanted to understand whether similar responses could be achieved with different dosing frequencies, such as biweekly versus monthly.
This was particularly important for an antibody drug like lecanemab, which needs to access the brain, potentially leading to differences in PK. The Phase II trial also aimed to explore the consistency of responses across various endpoints, including cognitive and functional measures. The team used a novel endpoint, ADAS-Cog, which they developed to detect early changes in the disease. This endpoint was particularly sensitive to early changes, allowing the team to identify the most effective dose regimens quickly. Kramer highlighted that the Phase II trial was not unblinded; the adaptive randomization and interim analyses were conducted by a computer algorithm, ensuring that the study remained blinded to the researchers.
[](https://theconferenceforum.org/hub/dpharm?utm_campaign=article&utm_medium=banner&utm_source=clinical-trial-vanguard)
## [](#key-takeaways-and-future-implications)Key Takeaways and Future Implications
Dr. Kramer concluded the session by summarizing the key takeaways. The Bayesian design allowed Eisai to answer more questions accurately and inexpensively, ultimately improving patient outcomes. The Phase II trial’s success in predicting Phase III outcomes demonstrated the potential of Bayesian methods to revolutionize clinical trials. Kramer emphasized that the Bayesian design enabled the team to make early decisions, conserve placebo patients, and quickly identify the most effective dose regimens. This approach not only accelerated drug development but also improved the ethical aspects of clinical trials by reducing the number of patients exposed to placebo.
The session underscored the importance of innovative trial designs in accelerating drug development, particularly for complex diseases like Alzheimer’s. As Bayesian methods gain more acceptance, they promise to make clinical trials more efficient and effective, benefiting patients and the pharmaceutical industry. The success of the LEQEMBI™ trials serves as a compelling example of how adaptive designs can transform the landscape of clinical research, offering new hope for patients and setting a new standard for future drug development.
## [](#summary)Summary
Eisai’s successful deployment of Bayesian methods in their Alzheimer’s trials is a compelling case study of the power of adaptive clinical trial design. By embracing innovation, Eisai not only accelerated the approval of LEQEMBI™ but also set a new standard for future drug development, demonstrating the transformative potential of Bayesian methods in clinical research.
**Categories:** Article: Conference Coverage
---
### [Advancing Cancer Treatment with Nektar Therapeutics: Insights into NKTR-255](https://www.clinicaltrialvanguard.com/executiveinterviews/advancing-cancer-treatment-with-nektar-therapeutics-insights-into-nktr-255/)
**Published:** January 28, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, we sit down with Mario Marcondes from Nektar Therapeutics to explore the advancements and potential of NKTR-255 in treating solid tumors and hematological cancers. Mario shares insights into clinical trials, challenges, and future milestones, highlighting NKTR-255’s role in the evolving landscape of cancer therapies.
## [](#moe-how-is-nktr-255-advancing-treatment-for-solid-tumors-and-hematological-cancers)**Moe: How is NKTR-255 advancing treatment for solid tumors and hematological cancers?**
NKTR-255, an IL-15 receptor agonist, is making significant strides toward treating solid tumors and hematological cancers by focusing on expanding NK and CD8 cells, which are crucial for anti-tumor response. For instance, the RESCUE trial is pivotal, leveraging the ability to rescue these cells post-chemo radiation in non-small cell lung cancer or NSCLC patients. This is a Nektar-sponsored study to research NKTR-255’s ability to reverse radiation-induced lymphopenia in patients with NSCLC. When combined with the checkpoint inhibitor durvalumab, NKTR-255 showed significant improvement in lymphocyte recovery, a key prognostic factor for survival in
Mario Marcondes, VP Head Of Clinical Development Nektar Therapeutics
such cancers. This combination was well-tolerated by patients, suggesting its potential to enhance immunotherapy outcomes for multiple indications, including Glioblastoma and other solid tumor indications where chemo-radiation is employed.
Additionally, we are exploring combinations with other therapies, such as avelumab in bladder cancer, to enhance systemic responses—our focus on safety and efficacy positions NKTR-255 as a promising candidate in oncology. NKTR-255 aims to improve treatment outcomes and offer new hope for patients with limited options by addressing key challenges in immune cell recovery. This strategic approach underscores our commitment to advancing cancer treatment through innovative solutions.
## [](#moe-what-insights-have-been-gained-from-nktr-255s-phase-two-trial-especially-with-car-t-therapies)**Moe: What insights have been gained from NKTR-255’s phase two trial, especially with CAR-T therapies?**
The phase two trial of NKTR-255 has provided valuable insights into its safety and efficacy, particularly in synergy with CAR-T therapies. Compared to high-dose IL-2, IL-15 offers cellular expansion with a manageable safety profile. Our trials have shown enhanced NK and CD8 T [cell expansion](https://www.clinicaltrialvanguard.com/news/lymphodepletion-improves-t-cell-expansion-in-lymphoma-patients/), which are crucial for CAR-T cell therapies. This aligns with unmet needs for durable responses in cancer treatment by potentially overcoming limitations in persistence and durability of response, especially in Large B cell Non Hodgkins Lymphoma.
Collaborations with Dr. Crystal Mackall at Stanford and Dr. Cameron Turtle from Seattle have shown that NKTR-255 can overcome CAR-T cell persistence limitations, a significant challenge in treating lymphoma. This synergy with CAR-T therapies addresses a critical gap in hematological malignancies, offering hope for improved long-term outcomes.
## [](#moe-how-does-nktr-255-address-limitations-in-immunotherapy-like-cell-persistence-and-response-durability)**Moe: How does NKTR-255 address limitations in immunotherapy, like cell persistence and response durability?**
NKTR-255 addresses key limitations in immunotherapy by enhancing cell persistence and response durability. Administering it at day 14 to 21 can re-expand CAR-T cells, improving their tenacity. This addresses the limitation of CAR-T cell therapies, which often struggle with persistence. Our data indicates a significant improvement in response durability, which is crucial for patients with limited treatment options post-CAR-T cell therapy.
Data from the Fred Hutchinson collaboration suggests that NKTR-255 can create a second peak of CAR-T cell elevation, improving the area under the curve and, thus the long-term efficacy of the treatment. This capability is critical in solid tumors, where maintaining a robust immune response is challenging. By enhancing immune cell recovery, NKTR-255 aims to improve treatment outcomes and offer new hope for patients with limited options, potentially transforming the cancer treatment landscape.
## [](#moe-what-challenges-or-unexpected-findings-emerged-during-the-phase-two-trial-and-how-did-you-adapt)**Moe: What challenges or unexpected findings emerged during the phase two trial, and how did you adapt?**
Executing complex trials like NKTR-255’s phase two study presented challenges, requiring substantial resources and patient screening due to strict inclusion criteria. We also faced issues with proprietary assays for CAR-T cell PK. We enhanced collaboration and expertise in this field by leveraging relationships with academic centers. Our proprietary polymer conjugation technology allowed us to administer NKTR-255 less frequently, overcoming the short half-life of endogenous IL-15. The early NCI’s IL-15 molecule researched and published by Dr. Kevin C. Conlon had some issues with very short half-life, requiring frequent administration, which is not commercially feasible. Our approach allows for administration every 21 to 28 days, making it more practical for clinical use.
## [](#moe-what-role-do-you-envision-for-nktr-255-in-combination-cancer-therapies)**Moe: What role do you envision for NKTR-255 in combination cancer therapies?**
NKTR-255 can potentially enhance endogenous reconstitution of NK and CD8 cells, not only in CAR-T cell therapies but also in combination with checkpoint inhibitors and other modalities. Its non-immunogenic nature and lack of tachyphylaxis make it suitable for long-term use, potentially up to two years, without the safety concerns seen with other agents. We are actively seeking partnerships to expand its application in the immuno-oncology space. Our collaboration with EMD Serono in the JAVELIN Medley trial is exploring its use in combination with avelumab, aiming to deliver robust systemic responses in bladder cancer. This integration with existing therapies could significantly enhance treatment efficacy and patient outcomes.
## [](#moe-what-are-the-key-milestones-for-nktr-255s-development-and-how-will-they-shape-its-trajectory)**Moe: What are the key milestones for NKTR-255’s development, and how will they shape its trajectory?**
Key milestones for NKTR-255 include upcoming data presentations at conferences, such as new efficacy and PD analysis for the Breyanzi NKTR-255 combination, which we are aiming to present at a Scientific Meeting in the middle of this year, and results from our collaboration with EMD Serono in the JAVELIN Medley trial. We also anticipate presenting preliminary data in non-small cell lung cancer in a Scientific Symposium later this year. These milestones will be crucial for attracting strategic partners and advancing NKTR-255 toward registration trials, ultimately shaping its clinical and commercial trajectory.
The data from Dr. Cameron Turtle and Dr. Alex Herrera on [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) will provide critical insights into its efficacy and safety, potentially influencing its adoption in clinical practice. Our ongoing efforts to seek strategic partnerships and licensing deals will further enhance its commercial potential.
## [](#moe-is-there-anything-else-youd-like-to-add-that-i-might-have-missed)**Moe: Is there anything else you’d like to add that I might have missed?**
NKTR-255 is poised to deliver significant cellular boosts crucial for immuno-oncology and cellular therapies. Its safety profile and lack of T regulatory cell expansion make it an ideal candidate for combination therapies. We are excited to continue advancing this work in partnership with others in the field. The ability to provide a safe and effective cytokine boost without the complications associated with other treatments positions NKTR-255 as a promising asset in the fight against cancer.
**Categories:** Article: Executive Interviews
---
### [Positive Topline Results in 5-MeO-DMT Study for Alcohol Use Disorder](https://www.clinicaltrialvanguard.com/news/positive-topline-results-in-5-meo-dmt-study-for-alcohol-use-disorder/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
Atai [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) announced positive topline results from a Phase 2a study of [BPL-003](https://www.clinicaltrialvanguard.com/news/bpl-003-trial-shows-victory-for-treatment-resistant-depression/), a synthetic intranasal 5-MeO-DMT formulation, for alcohol use disorder (AUD). The open-label study in 12 patients showed that a single dose of BPL-003, combined with relapse prevention therapy, led to significant and sustained reductions in alcohol consumption over 12 weeks. The treatment was well-tolerated, with no serious adverse events.
These results are a potential game-changer for AUD treatment. Current pharmaceutical options for AUD often have limited efficacy, leaving a substantial unmet need. BPL-003’s rapid action and durable effects, coupled with its short in-clinic administration time (approximately two hours), could significantly improve patient compliance and outcomes. The 50% abstinence rate at three months is particularly noteworthy, suggesting the potential for long-term benefits. This approach offers a new paradigm compared to existing treatments that require daily or frequent dosing.
The study showed a decrease in mean daily alcohol units consumed from 9.3 to 2.2 at 12 weeks. Heavy drinking days decreased from 56% to 13% at 12 weeks. Furthermore, the mean number of abstinent days increased from 33% to 81% over the 12-week period.
This positive Phase 2a data strengthens the potential of BPL-003 not only for AUD but also for other [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) indications. Further research will be crucial to confirm these preliminary findings in larger, controlled trials and to explore the optimal combination with behavioral therapies. The success of BPL-003 could lead to a paradigm shift in how AUD and potentially other substance use disorders are treated, offering a more effective and patient-centric approach. Further development and potential regulatory approval of BPL-003 could represent a significant advancement in addressing the substantial unmet need in the AUD treatment landscape.
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**Categories:** News
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### [SAB Bio Announces Positive Topline Phase 1 Clinical Results with Potentially Disease-Modifying T1D Therapy SAB-142](https://www.clinicaltrialvanguard.com/news/sab-bio-announces-positive-topline-phase-1-clinical-results-with-potentially-disease-modifying-t1d-therapy-sab-142/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
SAB Bio announced positive topline data from a Phase 1 trial of [SAB-142](https://www.clinicaltrialvanguard.com/news/new-hope-for-t1d-first-patient-dosed-in-safeguard-trial/), a human anti-thymocyte immunoglobulin being developed to delay or prevent [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/) (T1D). The study in healthy volunteers met its primary safety and pharmacodynamic objectives, paving the way for Phase 2b development. The results confirm that SAB-142 does not cause serum sickness or produce anti-drug antibodies at the target dose.
This news is vital because SAB-142 offers a potential new mechanism of action for managing T1D. Current treatments focus on managing blood sugar levels but don’t address the underlying autoimmune attack. SAB-142’s immunomodulatory action could delay disease onset or slow its progression, representing a significant advance for patients facing a lifetime of intensive disease management. The confirmation of a favorable safety profile further strengthens its potential for chronic use in an outpatient setting, improving patient convenience and adherence.
The Phase 1 trial demonstrated a favorable safety profile of SAB-142 across a dose range of 0.03mg/kg to 2.5mg/kg. Importantly, no serum sickness or anti-drug antibodies were observed. The study also confirmed sustained immunomodulation, with a mechanism of action analogous to rabbit ATG, currently used in T1D. The data supports the drug’s potential for C-peptide preservation, a key marker of pancreatic function.
SAB Bio plans to initiate a Phase 2b trial in 2025, evaluating SAB-142 in both adults and children with new-onset T1D. Positive results from this trial could significantly alter the T1D treatment landscape, offering a new class of therapy to delay or prevent disease progression and potentially improve long-term patient outcomes. The advancement of SAB-142 into later-stage clinical trials represents significant progress toward a much-needed therapeutic approach for T1D.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [March Biosciences Gets FDA Orphan Drug for Lymphoma](https://www.clinicaltrialvanguard.com/news/march-biosciences-gets-fda-orphan-drug-for-lymphoma/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
March Biosciences received FDA orphan drug designation for [MB-105](https://www.clinicaltrialvanguard.com/news/march-biosciences-mb-105-dosed-in-first-t-cell-lymphoma-patient/), its CD5-targeted CAR-T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for relapsed/refractory CD5-positive [T-cell lymphoma](https://www.clinicaltrialvanguard.com/news/soquelitinib-data-in-t-cell-lymphoma-promising-results-unveiled/). The company is preparing to advance MB-105 to a Phase 2 clinical trial in early 2025, following a recently closed Series A financing round of $28.4 million. This designation acknowledges the unmet need for new treatments for this rare cancer.
This FDA decision is crucial for patients with T-cell lymphoma, who currently have limited effective treatment options and often face a poor prognosis if their cancer relapses or becomes resistant to initial therapy. The orphan drug designation validates the potential of MB-105 to address this critical gap in treatment and offers hope for improved outcomes. It also strategically positions March Bio for potential market exclusivity, a significant advantage in the competitive landscape of oncology treatments.
The orphan drug status confers substantial benefits on March Bio, including tax credits for clinical testing and exemption from prescription drug user fees. More importantly, it grants seven years of market exclusivity upon FDA approval, creating a significant commercial opportunity. This, combined with the recent Series A funding, positions the company well for the planned Phase 2 trial and future commercialization activities. The Phase 1 trial data showing a 44% overall response rate in T-cell lymphoma patients further supports the therapy’s potential.
The FDA’s recognition of MB-105 as an orphan drug reinforces the therapy’s promise and its potential to improve the lives of patients with this rare and aggressive cancer. The upcoming Phase 2 trial will be pivotal in confirming the efficacy and safety profile observed in the initial Phase 1 study. Positive results could lead to accelerated development and ultimately provide a much-needed new treatment option for patients with T-cell lymphoma.
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**Categories:** News
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### [Silexion Shows Strong Tumor Reduction in Pancreatic Cancer Models](https://www.clinicaltrialvanguard.com/news/silexion-shows-strong-tumor-reduction-in-pancreatic-cancer-models/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) announced positive preclinical data for [SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/), its RNAi therapeutic candidate targeting KRAS-driven cancers. The data validates systemic administration of the drug, showing significant tumor reduction in orthotopic [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) models. Further research will investigate SIL-204’s impact on metastasis.
This preclinical success is a crucial step for Silexion and potentially for patients battling KRAS-driven cancers, especially pancreatic cancer, which is known for its aggressiveness and poor prognosis. Systemic administration, if proven effective in later trials, offers a less invasive and potentially more effective way to reach and treat tumors compared to localized treatments, potentially improving patient outcomes and quality of life. It also broadens the potential application of SIL-204 to metastatic disease, a significant challenge in cancer treatment.
Key preclinical findings include a 50% tumor growth reduction and 50% complete necrosis observed in mice with human pancreatic tumors harboring the G12D KRAS mutation after 30 days of treatment with an extended-release formulation of SIL-204. Subcutaneous administration also inhibited tumor growth in orthotopic mouse models of metastatic pancreatic cancer. Importantly, a single systemic dose maintained therapeutic drug levels in rat plasma and tissues for over 56 days. SIL-204 effectively targets multiple KRAS mutations (G12D, G12V, G12R, Q61H, and G13D), expanding its potential patient population. Separate studies using intratumoral administration of SIL-204 microparticles further demonstrated its anti-tumor activity.
These results position SIL-204 as a potential next-generation therapy for KRAS-driven cancers. Silexion’s plan to expand the development program based on these findings suggests the company is optimistic about the drug’s potential to progress to clinical trials and eventually offer a new treatment option for patients with these difficult-to-treat cancers. The extended release formulation and demonstrated ability to target multiple KRAS mutations could differentiate SIL-204 from existing therapies, potentially leading to improved efficacy and broader applicability in the treatment landscape.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Tevogen Bio Secures $10M Grant Funding](https://www.clinicaltrialvanguard.com/news/tevogen-bio-secures-10m-grant-funding/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
[Tevogen Bio](https://www.clinicaltrialvanguard.com/news/tevogen-bios-investigational-t-cell-therapy-effective-against-jn-1-variant/) (Nasdaq: TVGN), a clinical-stage immunotherapy biotech company, has secured up to $10 million in non-dilutive grant funding from KRHP LLC, an affiliate of an existing Tevogen investor. The initial contribution is $2 million, with the potential for an additional $8 million based on KRHP’s review of Tevogen’s progress. This funding will support the development of T cell therapies for cancer and viral infections, as well as the expansion of Tevogen’s artificial intelligence initiatives.
This grant is particularly impactful because it provides Tevogen with substantial non-dilutive capital, allowing the company to advance its research and development efforts without impacting shareholder value. The timing aligns with Tevogen’s focus on expanding its AI capabilities, potentially accelerating drug discovery and development timelines, and ultimately benefiting patients by bringing innovative therapies to market faster. This external validation from an existing investor, through its affiliate, reinforces confidence in Tevogen’s scientific approach and business strategy.
The $10 million grant supplements a previously announced $36 million loan agreement secured in the second quarter of 2024, significantly bolstering Tevogen’s financial position. The company is collaborating with Microsoft on AI in biopharma, exploring how AI can revolutionize drug discovery, improve patient access, and reduce operating costs.
This influx of non-dilutive funding positions Tevogen to aggressively pursue its clinical development programs and AI initiatives. It will be crucial to observe how Tevogen allocates these resources and whether they translate into accelerated development timelines and tangible advancements in their T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) pipeline. The collaboration with Microsoft and the company’s ongoing focus on AI suggest a forward-thinking approach that could distinguish Tevogen within the competitive immunotherapy landscape.
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### [First Canadian Clinic Offers C2N's PrecivityAD2 Blood Test for Alzheimer's](https://www.clinicaltrialvanguard.com/news/first-canadian-clinic-offers-c2ns-precivityad2-blood-test-for-alzheimers/)
**Published:** January 29, 2025
**Author:** Jon Napitupulu
**Content:**
The Toronto Memory Program (TMP) is the first clinic in Canada to offer [C2N Diagnostics](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/)‘ PrecivityAD2 blood test for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. This non-invasive test detects amyloid pathology, a hallmark of Alzheimer’s, and addresses the diagnostic challenges posed by limited access to specialists and infrastructure like PET scanners. The test is designed for individuals aged 55 and older experiencing cognitive decline and suspected of having Alzheimer’s or other related conditions.
This development significantly impacts Alzheimer’s care in Canada, where over 600,000 individuals live with the disease and many more experience mild cognitive impairment. The lengthy wait times for diagnosis, primarily due to limited resources, hinder timely intervention and treatment. The PrecivityAD2 test offers a more accessible and efficient diagnostic pathway, potentially expediting diagnosis and enabling earlier access to emerging treatments.
The PrecivityAD2 test requires a simple blood draw and measures specific proteins associated with amyloid plaques in the brain. A recent study published in JAMA demonstrated the test’s high accuracy, exceeding 90% when compared to traditional methods like cerebrospinal fluid analysis and amyloid PET scans. The test is performed in C2N’s certified and accredited laboratory, adhering to rigorous quality standards.
The introduction of the PrecivityAD2 test in Canada represents a crucial step toward improving Alzheimer’s care. Wider availability of this blood test promises to reduce diagnostic delays, facilitate earlier and more accurate diagnoses, and ultimately enhance patient care by enabling timely access to appropriate interventions and therapies. This also opens doors for more efficient clinical trials and research into Alzheimer’s disease.
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### [Transforming Clinical Trials: Gen Li's Vision for the Future](https://www.clinicaltrialvanguard.com/executiveinterviews/transforming-clinical-trials-gen-lis-vision-for-the-future/)
**Published:** January 29, 2025
**Author:** Moe Alsumidaie
**Content:**
In the dynamic world of clinical trials, Gen Li of Phesi offers a unique perspective on the industry’s challenges and innovations. As trials face increased attrition rates post-pandemic, AI and data analytics become crucial. Gen Li discusses these issues, the promise of biomarker-specific studies, and the future of clinical trials, providing valuable insights into the evolving landscape.
## [](#moe-with-rising-attrition-rates-in-phase-two-trials-post-pandemic-what-factors-drive-this-trend-and-how-can-ai-and-data-analytics-mitigate-these-risks)Moe: With rising attrition rates in phase two trials post-pandemic, what factors drive this trend, and how can AI and data analytics mitigate these risks?
**Gen Li:** The increase in attrition rates is concerning. Pre-pandemic, rates were stable at 20%, but they have since risen to 25-30%. This shift is largely due to the COVID-19 pandemic, which disrupted the industry’s long-standing ecosystem. Despite advancements in centralized trials and digital data collection, these have not fully addressed the complexities. Issues such as overly complicated trial designs, burdens on patients and sites, and misinterpreting results, especially in oncology, contribute to this crisis. AI and data analytics can play a pivotal role by refining trial designs and improving site selection, ensuring trials are more patient-centric and aligned with specific patient populations. Sponsors can leverage data from millions of patients to refine trial designs and improve site selection, ensuring trials are more patient-centric and aligned with specific patient populations.
Gen Li, CEO of Phesi
## [](#moe-biomarker-specific-studies-in-nsclc-show-promise-what-challenges-remain-in-scaling-these-approaches-across-other-areas-and-how-can-sponsors-overcome-these-challenges)Moe: Biomarker-specific studies in NSCLC show promise. What challenges remain in scaling these approaches across other areas, and how can Sponsors overcome these challenges?
**Gen Li:** Clinical development doesn’t exist in a vacuum; it relies on a broader understanding of diseases. For instance, the progress in cancer treatment has evolved from primitive methods to more specific biomarker-driven approaches. However, scaling these advancements requires a concerted effort across medical and scientific communities. Sponsors can leverage data science to provide tools that help clinical development teams solve complex problems. Such approaches integrate external insights and foster innovation, which is crucial for advancing biomarker-specific studies across various therapeutic areas. For example, the evolution of CAR-T therapies from blood cancers to solid tumors exemplifies the potential for biomarker-driven approaches to revolutionize treatment across different cancer types. This collaborative effort is essential for translating these advancements into other therapeutic areas, ensuring that the benefits of biomarker-specific studies are realized across the board.
## [](#moe-breast-cancer-is-heavily-researched-how-can-these-insights-be-applied-to-less-studied-diseases-to-drive-innovation-and-reduce-attrition-rates)Moe: Breast cancer is heavily researched. How can these insights be applied to less studied diseases to drive innovation and reduce attrition rates?
**Gen Li:** While [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) research has seen breakthroughs, it still faces high attrition rates, particularly in areas like triple-negative breast cancer. The key is to move beyond the negatives and identify more positive markers. This approach can be generalized to other diseases, where innovation is driven by both the dire needs of the disease and advancements in medical understanding. For instance, the progress in [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) through biomarker identification can be a model for other areas, including neuroscience. The challenge lies in translating these insights into actionable strategies for diseases with less research focus, ensuring that the methodologies developed in well-studied areas can be adapted to new contexts. Applying these insights can drive innovation and reduce attrition rates in less-studied diseases, ultimately improving outcomes.
## [](#moe-investigator-site-selection-is-crucial-how-can-sponsors-optimize-sites-for-robust-recruitment-and-retention-especially-in-underrepresented-regions)Moe: Investigator site selection is crucial. How can sponsors optimize sites for robust recruitment and retention, especially in underrepresented regions?
**Gen Li:** Site selection has evolved significantly. Historically, a few investigators contributed to most trials, leading to overburdened sites. We aim to change this by focusing on medium-tier capable yet less burdened investigators. We also emphasize aligning investigators with the specific patient populations targeted by trials. Our platform constructs digital patient profiles to ensure precise alignment, essential for successful recruitment and retention. For example, in acute ischemic stroke trials, identifying neurologists who can recruit patients within a critical 24-48 hour window is crucial. Sponsors can leverage such platforms to facilitate this level of precision. By ensuring that investigators are well-matched to the patient populations they serve, they can improve recruitment and retention metrics, particularly in underrepresented regions, ultimately enhancing the success of clinical trials.
## [](#moe-given-the-financial-burden-of-failed-trials-what-are-key-missteps-in-trial-design-or-execution-how-can-sponsors-address-these-inefficiencies)Moe: Given the financial burden of failed trials, what are key missteps in trial design or execution, how can sponsors address these inefficiencies?
**Gen Li:** Clinical trials inherently involve unknowns, but we can utilize existing data to simulate and foresee potential outcomes. Some platforms allow for precise simulation of trial designs, site selection, and patient composition, helping to mitigate risks and reduce attrition rates. This approach is crucial for phase two and phase three trials, where attrition can be even more costly. By simulating trial scenarios, sponsors can better anticipate challenges and adjust their strategies accordingly, reducing the likelihood of costly failures. Our platform’s ability to simulate and predict outcomes provides sponsors with the tools to make informed decisions, ultimately improving clinical trial efficiency and success rate.
## [](#moe-with-covid-19-no-longer-a-top-study-focus-what-does-this-shift-tell-us-about-sponsor-priorities-post-pandemic-and-how-are-you-leveraging-these-trends)Moe: With COVID-19 no longer a top study focus, what does this shift tell us about sponsor priorities post-pandemic, and how are you leveraging these trends?
**Gen Li:** The shift from COVID-19 reflects a return to focusing on other pressing medical needs and innovations. However, the emergence of GLP-1 as a significant area of interest highlights the unpredictable nature of medical innovation. We aim to position ourselves to embrace such disruptive forces, ensuring we remain adaptable and responsive to new trends and priorities in clinical research. This adaptability is crucial as the industry navigates post-pandemic priorities, balancing the need for innovation with the realities of resource allocation. By staying attuned to these shifts, we can leverage emerging trends to influence future trial strategies, ensuring we remain at the forefront of clinical research innovation.
**Categories:** Article: Executive Interviews
---
### [Targeted Alpha Therapy Clinical Trials: An Overview](https://www.clinicaltrialvanguard.com/news/targeted-alpha-therapy-clinical-trials-an-overview/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
Over 20 alpha therapies are currently in clinical trials, marking a significant advancement in targeted cancer treatment. These therapies utilize alpha-emitting isotopes to deliver highly localized radiation to tumor cells, minimizing damage to surrounding healthy tissues. This approach is particularly effective against challenging cancers, including those with low cell counts or bone metastases.
The increasing number of clinical trials for targeted alpha therapy (TAT) represents a crucial step towards more effective and personalized cancer care. TAT’s precision addresses a critical unmet need in oncology, offering a potential solution for cancers resistant to traditional therapies. The targeted nature of TAT may also lead to reduced side effects compared to conventional radiation or chemotherapy, improving patient outcomes and quality of life.
The FDA approval of Xofigo in 2013 for advanced [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) validated the clinical potential of TAT. Ongoing trials focus on refining targeting mechanisms, such as using prostate-specific membrane antigen (PSMA) for prostate cancer and CD38 for multiple myeloma. Promising preclinical results have been observed in studies targeting CD38 in multiple myeloma and CD37 in hematological malignancies. Pharmaceutical companies are investing heavily in this area, exploring combinations of TAT with other treatments, as seen in Actinium Pharmaceuticals’ trial combining Actimab-A with venetoclax for acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/).
Targeted alpha therapy holds immense promise for revolutionizing cancer treatment. As research progresses and refinements in isotope production, targeting, and imaging continue, TAT is poised to become a cornerstone of oncology, offering new hope for patients with complex or difficult-to-treat cancers. This targeted approach represents a paradigm shift towards personalized medicine, providing more effective and potentially less toxic treatment options.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Gamma Delta T Cell Cancer Therapy Clinical Trials Overview](https://www.clinicaltrialvanguard.com/news/gamma-delta-t-cell-cancer-therapy-clinical-trials-overview/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
The global gamma delta T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) market is experiencing rapid growth, driven by over 30 therapies currently in clinical trials, primarily concentrated in the US and China. These therapies leverage the unique properties of gamma delta T cells, which can recognize a broad range of antigens without MHC presentation, offering advantages over conventional T cell therapies. This research focuses on developing treatments for various cancers and other diseases like autoimmune disorders and infections.
This burgeoning area of research holds significant promise for patients who haven’t responded to first-line treatments, particularly for hematologic cancers like [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) and AML. The ability of gamma delta T cells to bypass MHC-dependent antigen presentation offers a crucial advantage, potentially overcoming tumor evasion mechanisms that often limit the efficacy of current immunotherapies like CAR T-cell therapies and bispecific antibodies. This distinct mechanism of action could lead to more effective and durable responses, potentially delaying or even eliminating the need for aggressive interventions like bone marrow transplants.
Currently, no gamma delta T cell therapies are commercially available. However, the pipeline is robust, with several candidates in various stages of clinical trials. Companies like TC Biopharm, with their lead candidate TCB-002 (OmnImmune) in Phase 2/3 trials for AML, are leading the charge. Other companies like Lava Therapeutics and In8Bio are also developing therapies for a range of solid and hematological tumors. Beyond oncology, companies like [ImCheck](https://www.clinicaltrialvanguard.com/news/imcheck-aml-therapy-shows-high-remission-at-asco-2025/) Therapeutics are exploring the use of monoclonal antibodies to stimulate gamma delta T cell production for non-oncological applications.
The confluence of promising preclinical results, increasing investment from pharmaceutical companies, and expanding clinical research collaborations strongly suggests that gamma delta T cell therapies are poised for significant growth. As these therapies progress through clinical trials and obtain regulatory approvals, their availability could reshape the treatment landscape for various cancers and potentially other diseases, offering a new wave of hope for patients previously facing limited therapeutic options.
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### [Cardiovascular Devices Market to Hit $110B by 2029](https://www.clinicaltrialvanguard.com/news/cardiovascular-devices-market-to-hit-110b-by-2029/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
The global cardiovascular devices market, valued at US$72.83 billion in 2023, is projected to reach US$110.39 billion by 2029, growing at a CAGR of 7.3%. This growth is driven by the rising prevalence of cardiovascular disease (CVD), an aging global population, and increasing risk factors such as diabetes, hypertension, and [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/). Advancements in technology, including bioresorbable stents and improved imaging systems, further contribute to market expansion.
This market growth represents a significant opportunity for both established and emerging companies in the medical device sector. The increasing demand for cardiovascular devices underscores the urgent need for effective solutions to address the growing burden of CVD globally. The development and adoption of advanced technologies offer improved patient outcomes, potentially reducing healthcare costs associated with long-term CVD management.
The market is segmented by product type, indication, and end-user. Cardiac imaging and diagnostic devices hold a significant market share, driven by their crucial role in monitoring and treating cardiovascular conditions. Arrhythmias represent a key indication driving market growth, primarily due to the rising prevalence of conditions like atrial fibrillation. Hospitals remain the primary end-users, reflecting the need for specialized facilities and equipment for complex cardiovascular procedures. North America currently holds the largest market share, followed by Europe and Asia Pacific, with advancements in catheter-based technologies and imaging techniques driving growth in these regions.
The continued growth of the cardiovascular devices market suggests a positive trajectory for companies investing in innovative technologies. Emerging markets present significant growth potential as healthcare infrastructure modernization efforts progress. The focus on minimally invasive procedures and remote monitoring is expected to further fuel demand for advanced devices, creating a competitive landscape and driving further innovation in the field.
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**Categories:** News
**Tags:** eClinical Tech News
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### [4DMT to Review 52-Week Interim Phase 2b Results](https://www.clinicaltrialvanguard.com/news/4dmt-to-review-52-week-interim-phase-2b-results/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
4D Molecular Therapeutics will present 52-week interim data from the Phase 2b portion of its PRISM clinical trial evaluating 4D-150 for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD). Dr. Dante Pieramici, a principal investigator in the trial, will present the findings at the Angiogenesis, Exudation, and Degeneration 2025 conference on February 8, 2025. A follow-up webcast on February 10, 2025, will feature additional analyses of 4D-150 in wet AMD and diabetic macular edema (DME), including data from a subgroup resembling the Phase 3 trial population.
This data release is important because it offers a more comprehensive look at the potential long-term efficacy and safety of 4D-150, a gene therapy designed for sustained delivery of anti-VEGF treatment. The inclusion of 52-week data is a significant step towards demonstrating the durability of the treatment, a critical factor in reducing the frequency of injections for patients with wet AMD. The focus on a subgroup closely matching the Phase 3 trial population strengthens the relevance of these interim findings for predicting the potential success of the larger, pivotal studies. Furthermore, the inclusion of data on DME broadens the potential therapeutic applications of 4D-150, highlighting its possible impact across multiple retinal diseases.
The February 8th presentation will cover 52-week results from the Phase 2b Population Extension cohort of the PRISM trial evaluating 4D-150 in a broad wet AMD patient population. The February 10th webcast will delve into the 52-week landmark interim efficacy data for the 3E10 vg/eye dose (N=30), with a specific focus on the recently diagnosed subgroup (N=15). It will also feature the latest long-term interim safety data for this dose (N=71 across all PRISM Phase 1/2 patients), the longest available data on aqueous humor aflibercept protein levels, and further details on the previously released 32-week interim data from the SPECTRA Part 1 DME trial.
These interim results represent a crucial milestone for 4DMT and the development of 4D-150. Positive data could significantly bolster confidence in the therapy’s potential to provide long-lasting patient benefits, reducing the burden of frequent injections and potentially improving visual outcomes. Investors and the ophthalmology community will closely watch the upcoming data presentations and subsequent analyses, as they will play a significant role in shaping expectations for the ongoing Phase 3 trials. The data will also provide valuable insight into the broader potential of sustained-release gene therapies for treating chronic retinal diseases.
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**Categories:** News
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### [DPTX3496: New Drug Candidate for Colorectal, Breast, and Lung Cancers](https://www.clinicaltrialvanguard.com/news/dptx3496-new-drug-candidate-for-colorectal-breast-and-lung-cancers/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
Dewpoint Therapeutics has nominated DPTX3496, its second drug development candidate. DPTX3496 is an oral small molecule designed to treat Wnt-driven cancers like colorectal, breast, and lung cancers by modulating condensates within cells. The drug is currently in IND-enabling studies, with an IND filing anticipated for the second half of 2025.
This development represents a potentially significant advance in cancer treatment. DPTX3496 targets the beta-catenin pathway, a common driver of uncontrolled cell growth in several cancer types. By sequestering beta-catenin within nuclear condensate depots, DPTX3496 aims to selectively induce apoptosis in cancer cells exhibiting excessive beta-catenin/Wnt signaling, offering a more targeted approach than traditional chemotherapy. This targeted mechanism may lead to improved efficacy and reduced side effects for patients, ultimately changing the treatment landscape for these prevalent cancers.
Pre-clinical data demonstrates promising tumor regression and stasis in various Wnt-driven tumor models, including patient-derived xenografts. Furthermore, DPTX3496 has shown good tolerability and minimal body weight loss in these models. Dewpoint has developed specific biomarker assays to monitor beta-catenin activity, which will be crucial for assessing target engagement and modulation during clinical trials. These biomarkers offer a unique opportunity to track drug activity and personalize treatment strategies. DPTX3496 also down-regulates beta-catenin-driven gene transcription and Wnt pathway activity •in vivo•, and plasma protein profiling shows modulation of systemic disease-associated beta-catenin target genes. This second drug candidate, along with the recently announced [DPTX3186](https://www.clinicaltrialvanguard.com/news/dewpoint-launches-promising-cancer-trial-for-solid-tumors/), demonstrates the productivity of Dewpoint’s platform and provides two distinct chemotypes targeting the same pathway, broadening the company’s potential therapeutic reach. Dewpoint is leveraging external collaborations with ConcertAI and Evotec to further enhance patient stratification and accelerate IND filing timelines.
The nomination of DPTX3496 reinforces Dewpoint’s position at the forefront of condensate biology research and its application in drug discovery. The anticipated IND filing in 2025 will be a crucial step towards clinical validation of this novel approach to cancer therapy. If successful, DPTX3496 could represent a substantial advancement in the treatment of Wnt-driven cancers, providing new hope for patients and potentially opening up new avenues for targeting previously “undruggable” targets in other disease areas.
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### [Lasofoxifene vs. Fulvestrant Effects on Urogenital Symptoms in ER+/HER2- Breast Cancer](https://www.clinicaltrialvanguard.com/news/lasofoxifene-vs-fulvestrant-effects-on-urogenital-symptoms-in-er-her2-breast-cancer/)
**Published:** January 30, 2025
**Author:** Jon Napitupulu
**Content:**
Sermonix Pharmaceuticals announced published results from a secondary endpoint analysis of its Phase 2 ELAINE-1 study of lasofoxifene in patients with •ESR1•-mutant, ER+/HER2- metastatic [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). The analysis, published in •Clinical Breast Cancer•, revealed that lasofoxifene improved vaginal and vulvar symptoms like dryness and pain, while fulvestrant, the comparator drug, did not show the same benefit. The primary endpoint of the ELAINE-1 study was progression-free survival (PFS), where lasofoxifene showed numerically, but not statistically significantly, greater PFS compared to fulvestrant.
These findings are particularly relevant because they address a significant quality-of-life issue for women undergoing treatment for metastatic breast cancer. Current endocrine therapies can often exacerbate vaginal and vulvar symptoms, negatively impacting patients’ well-being and potentially affecting treatment adherence. Lasofoxifene’s potential to alleviate these symptoms could offer a substantial improvement in the overall care of these patients. Furthermore, demonstrating a positive impact on patient-reported outcomes can strengthen the drug’s overall clinical profile and differentiate it from other therapies.
The secondary endpoint analysis included 70% of the original 103 enrolled patients. Results showed lasofoxifene decreased the composite vaginal and vulvar symptom score by 74% from baseline to week 16. In contrast, fulvestrant increased the composite symptom score by 36% over the same period. The analysis also identified factors associated with more severe baseline symptoms, including younger age, absence of visceral disease, prior tamoxifen use, and longer duration of aromatase inhibitor use.
The positive results from this secondary endpoint analysis support the continued development of lasofoxifene. Sermonix’s ongoing Phase 3 ELAINE-3 trial, which compares lasofoxifene plus abemaciclib to fulvestrant plus abemaciclib, will incorporate a more comprehensive quality-of-life assessment to further investigate the impact of lasofoxifene on these important patient-centric outcomes. Positive results from the ELAINE-3 trial, including the quality-of-life data, could pave the way for regulatory approval and potentially position lasofoxifene as a preferred treatment option for patients with •ESR1•-mutant metastatic breast cancer. This could shift the treatment paradigm by offering a therapy that not only addresses disease progression but also significantly improves patients’ quality of life.
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**Categories:** News
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### [Regulatory Review Update on Lecanemab for Early Alzheimer's Disease in the EU](https://www.clinicaltrialvanguard.com/news/regulatory-review-update-on-lecanemab-for-early-alzheimers-disease-in-the-eu/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
Eisai and Biogen provided an update on the European Union’s regulatory review of [lecanemab](https://www.clinicaltrialvanguard.com/news/lecanemab-subcutaneous-autoinjector-shows-bioequivalent-exposure-to-iv-form/), a treatment for early-stage [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. The European Commission requested the Committee for Medicinal Products for Human Use (CHMP) to review recently available safety information on lecanemab and clarify risk mitigation measures. This review will occur at the CHMP’s February 2025 meeting.
This regulatory update is crucial for both the companies and individuals affected by Alzheimer’s. A potential delay or rejection of lecanemab in the EU would significantly impact access to this promising treatment for a devastating disease with limited therapeutic options. The outcome of the CHMP’s review will heavily influence market availability and patient access across the European Union. Furthermore, it holds implications for the broader Alzheimer’s landscape, as lecanemab represents one of the few disease-modifying therapies currently under advanced regulatory consideration.
The CHMP issued a positive opinion on lecanemab in November 2024, recommending its approval. However, subsequent safety data prompted the European Commission’s request for further review. Eisai and Biogen maintain that the lecanemab safety profile observed in post-market settings aligns with existing labels and does not reveal new safety concerns. They are confident that the available data sufficiently addresses the European Commission’s questions.
The February 2025 CHMP meeting will be pivotal for the future of lecanemab in the EU. A positive outcome, confirming the previous approval recommendation, would pave the way for patient access in European countries. Conversely, a negative decision could lead to further delays or even rejection, hindering progress in addressing the substantial unmet medical need in Alzheimer’s disease. The CHMP’s decision will influence not only the availability of lecanemab but also the overall momentum and direction of Alzheimer’s drug development.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Cervomed Neflamapimod DLB Program Update at Lewy Body Dementia Conference](https://www.clinicaltrialvanguard.com/news/cervomed-neflamapimod-dlb-program-update-at-lewy-body-dementia-conference/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
CervoMed provided an update on its Phase 2b RewinD-LB trial for neflamapimod in dementia with Lewy bodies (DLB). The blinded portion of the trial showed no significant difference between neflamapimod and placebo, likely due to lower-than-expected drug concentrations potentially linked to the age of the drug batch used. However, subsequent analyses and a food-effect study suggest newer capsules achieve the targeted plasma concentrations.
This development is crucial because DLB is a prevalent neurodegenerative disease with limited treatment options. Confirming the efficacy of neflamapimod at the correct dosage could represent a significant advancement in addressing a substantial unmet medical need, potentially improving the quality of life for hundreds of thousands of patients. Furthermore, a successful therapy could significantly impact healthcare costs associated with DLB, which are currently higher than those for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease.
Technically, the initial results of the RewinD-LB trial were disappointing due to low drug concentrations. However, the investigation into this issue identified the older drug batch as a likely culprit. Data from the open-label extension phase, using a newer batch, is expected to provide more definitive results on the drug’s efficacy at the targeted plasma concentrations. This data is anticipated in the first quarter of 2025.
The upcoming data readout from the open-label extension phase of RewinD-LB is critical for the future of neflamapimod. If the newer capsules demonstrate efficacy, it will validate the drug’s potential and justify further clinical development. Positive results could pave the way for a Phase 3 trial and eventual regulatory approval, offering a much-needed treatment option for DLB patients. Conversely, if the results remain lackluster, CervoMed will need to reassess its development strategy for neflamapimod in DLB.
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### [Datopotamab Deruxtecan Recommended for Approval in the EU](https://www.clinicaltrialvanguard.com/news/datopotamab-deruxtecan-recommended-for-approval-in-the-eu/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
[Datopotamab](https://www.clinicaltrialvanguard.com/news/datroway-approved-for-metastatic-breast-cancer/) deruxtecan (Dato-DXd), a TROP2-directed antibody drug conjugate developed by Daiichi Sankyo and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/), has been recommended for approval in the European Union for treating advanced hormone receptor-positive, HER2-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) in adults who have received prior endocrine therapy and chemotherapy. The positive opinion from the CHMP is based on the TROPION-Breast01 phase 3 trial, which demonstrated Dato-DXd significantly improved progression-free survival and objective response rates compared to standard chemotherapy. The European Commission will now review the CHMP recommendation and decide on marketing authorization.
This potential approval is crucial for patients with HR-positive, HER2-negative metastatic breast cancer who experience disease progression after standard endocrine therapy and chemotherapy. Current treatment options in this setting offer limited efficacy and often come with significant side effects. Dato-DXd provides a new therapeutic approach with a demonstrated ability to extend progression-free survival and increase response rates, potentially improving patient outcomes and quality of life.
In the TROPION-Breast01 trial, Dato-DXd reduced the risk of disease progression or death by 37% compared to chemotherapy. Patients treated with Dato-DXd had a median progression-free survival of 6.9 months versus 4.9 months for those receiving chemotherapy. The objective response rate was also higher in the Dato-DXd arm (36%) compared to the chemotherapy arm (23%). Importantly, the safety profile of Dato-DXd was favorable, with fewer grade 3 or higher treatment-related adverse events compared to chemotherapy.
The anticipated EU approval of Dato-DXd marks a significant advancement in the treatment landscape for advanced HR-positive, HER2-negative breast cancer. This new targeted therapy offers a promising alternative to conventional chemotherapy, potentially leading to better outcomes for patients who currently have limited effective treatment choices. This approval could also pave the way for further research exploring Dato-DXd’s potential in other breast cancer subtypes and in combination with other therapies.
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### [Eli Lilly's Omvoh gets FDA approval for Crohn's disease but faces challenges](https://www.clinicaltrialvanguard.com/news/eli-lillys-omvoh-gets-fda-approval-for-crohns-disease-but-faces-challenges/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
Eli Lilly’s Omvoh, an IL-23 inhibitor previously approved for [ulcerative colitis](https://www.clinicaltrialvanguard.com/news/abivax-clears-pre-nda-meeting-with-fda-for-obefazimod-in-ulcerative-colitis/), has received FDA approval for [Crohn’s](https://www.clinicaltrialvanguard.com/news/agomabs-crohns-drug-shows-promising-phase-2a-results/)[Crohn’s disease](https://www.clinicaltrialvanguard.com/news/abbvie-seeks-ema-approval-for-skyrizi-subcutaneous-induction-in-crohns-disease/). Despite physician familiarity with Omvoh, gastroenterologists surveyed by Spherix Global Insights prefer Johnson & Johnson’s Tremfya (currently awaiting approval) for Crohn’s disease treatment. This preference stems from a perception of Tremfya as a more significant advancement over current treatment options.
This approval is crucial for Crohn’s disease patients as it introduces a new treatment mechanism. While TNF inhibitors remain the first-line biologic treatment, the growing adoption of IL-23 inhibitors like AbbVie’s Skyrizi signals a potential shift in the treatment landscape. The availability of multiple IL-23 inhibitors offers patients and physicians more choices, potentially leading to improved outcomes by tailoring treatments to individual needs. This competition also encourages pharmaceutical companies to innovate and refine their offerings.
Spherix Global Insights’ Q4 2024 research reveals that roughly half of gastroenterologists view Tremfya as a substantial improvement over existing options, compared to only one-third for Omvoh. Over half of those surveyed prefer Tremfya as their IL-23 inhibitor of choice for Crohn’s disease, with only 20% selecting Omvoh. While Omvoh aims to address bowel urgency—a critical concern for Crohn’s disease patients—its success hinges on establishing a distinct value proposition against competitors.
The future of Omvoh in the Crohn’s disease market depends on Lilly’s ability to differentiate its efficacy and patient benefits compared to Tremfya and Skyrizi. The competition among these IL-23 inhibitors will likely drive further research and development, ultimately benefiting patients through more effective and targeted treatments. Market dynamics will be shaped by how effectively Lilly positions Omvoh, particularly in highlighting advantages beyond addressing bowel urgency.
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### [PrecivityAD2™ Blood Test Improves Alzheimer's Disease Evaluation](https://www.clinicaltrialvanguard.com/news/precivityad2-blood-test-improves-alzheimers-disease-evaluation/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
Researchers found that [C2N Diagnostics](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/)‘ PrecivityAD2 blood test improved healthcare providers’ ability to assess the likelihood of [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease in their patients. This improved accuracy led to more informed decisions regarding treatment, including changes in Alzheimer’s medication plans and further brain amyloid testing. The study involved diverse patient demographics, including underrepresented minorities, and took place across various academic and community-based practices.
This advancement is crucial because early and accurate Alzheimer’s diagnosis is a significant unmet need. The PrecivityAD2 test offers a less invasive and more accessible alternative to traditional diagnostic methods like cerebrospinal fluid analysis or PET scans. This improved accessibility could lead to earlier diagnosis, allowing patients and their families to make informed decisions about care and future planning sooner. It also facilitates earlier intervention with potential disease-modifying therapies, maximizing their effectiveness.
The Quality Improvement PrecivityAD2 (QUIP II) study involved 12 memory specialists treating 203 patients with cognitive impairment symptoms. The study focused on clinician-reported changes in diagnostic certainty and subsequent patient management plans before and after using the blood test. The results revealed a notable increase in diagnostic confidence and corresponding adjustments in treatment strategies after utilizing the PrecivityAD2 test. The test demonstrated over 90% accuracy compared to traditional methods. Furthermore, this builds on previous research demonstrating the test’s efficacy, particularly in primary care settings, and reinforces earlier findings from the QUIP I study on the PrecivityAD test.
The positive results from the QUIP II study strengthen the case for broader adoption of blood biomarker tests like PrecivityAD2 in Alzheimer’s disease diagnosis and management. This could lead to a paradigm shift in how Alzheimer’s is diagnosed, potentially streamlining the diagnostic process and improving patient outcomes. Increased access to accurate and convenient diagnostic tools may also stimulate further research and development of effective treatments and preventative strategies for Alzheimer’s disease.
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**Categories:** News
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### [Eyonis™ Lung Cancer Screening Meets Primary Endpoint in RELIVE Trial](https://www.clinicaltrialvanguard.com/news/eyonis-lung-cancer-screening-meets-primary-endpoint-in-relive-trial/)
**Published:** February 3, 2025
**Author:** Jon Napitupulu
**Content:**
Median Technologies announced positive top-line results from its RELIVE study for eyonis™ LCS, an AI-powered [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) screening software. The study met its primary endpoint, demonstrating that eyonis™ LCS significantly improves diagnostic accuracy when used in conjunction with radiologists compared to radiologists alone. This achievement follows the successful completion of the REALITY study and paves the way for regulatory submissions in the U.S. and EU in Q2 2025.
This news is vital because it addresses the critical need for early lung cancer detection. The current five-year survival rate for lung cancer is low, primarily because most cases are diagnosed at late stages. Eyonis™ LCS has the potential to increase early detection rates, thereby improving patient outcomes and potentially reducing healthcare costs associated with late-stage treatment. Improved accuracy in lung cancer screening can also minimize unnecessary follow-up procedures for healthy individuals, further reducing healthcare expenditures and patient anxiety.
The RELIVE study, conducted with 480 high-risk patients, showed that eyonis™ LCS improved radiologists’ ability to detect, localize, and characterize lung nodules. This translated to a statistically significant improvement in diagnostic accuracy (p=0.027). The company is currently analyzing secondary endpoints and will release additional data in the near future. The earlier REALITY study, with 1,147 patients, demonstrated eyonis™ LCS’s capability to differentiate between cancerous and non-cancerous patients and characterize suspicious nodules. Both studies utilized retrospective data from major cancer centers and hospitals in the U.S. and EU. Median Technologies is targeting a large addressable market, with 14.5 million people in the U.S. currently eligible for lung cancer screening under Medicare.
The positive results from RELIVE solidify the potential of eyonis™ LCS to transform lung cancer screening. Pending regulatory approvals, this technology could become a crucial tool for healthcare professionals, enabling earlier and more accurate diagnoses, leading to better patient outcomes and a potential reduction in healthcare costs. This could also impact global healthcare systems as lung screening programs expand in Europe and Asia.
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### [Invivyd Announces Positive Phase 1/2 Data for VYD2311 for COVID-19](https://www.clinicaltrialvanguard.com/news/invivyd-announces-positive-phase-1-2-data-for-vyd2311-for-covid-19/)
**Published:** February 4, 2025
**Author:** Jon Napitupulu
**Content:**
Invivyd announced positive Phase 1/2 clinical trial data for [VYD2311](https://www.clinicaltrialvanguard.com/news/invivyd-launches-phase-3-trial-for-covid-antibody-vyd2311/), a monoclonal antibody designed to offer superior, long-lasting protection against [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) compared to existing vaccines. The trial, involving 40 subjects, evaluated intravenous, intramuscular, and subcutaneous administration, demonstrating positive safety and pharmacokinetic results, supported by strong antiviral activity. VYD2311 offers a potential alternative to frequent booster shots and addresses limitations of current therapies.
This development is crucial given the continuing impact of COVID-19, including deaths, hospitalizations, and the rise of Long COVID, despite widespread vaccination and treatment efforts. VYD2311 could significantly reduce the disease burden and offer a more convenient and effective approach to managing COVID-19. It holds particular promise for immunocompromised individuals and others who struggle to mount an adequate immune response to vaccines.
Data from the trial reveal mild to moderate adverse events primarily related to injection site or infusion reactions. As of day 65, serum concentrations of VYD2311 remained high, suggesting a long half-life and potentially infrequent dosing intervals (e.g., annually or biannually). In vitro data show VYD2311 possesses approximately 17-fold greater neutralization potency than pemivibart, a current Invivyd mAb for COVID-19. This increased potency could translate to lower doses or enhanced antiviral activity. Preliminary data suggest VYD2311’s pharmacokinetic profile is similar to adintrevimab, a prior Invivyd mAb with an estimated half-life of 139 days.
This positive data positions VYD2311 as a potential game-changer in the fight against COVID-19. The potential for long-lasting protection with less frequent dosing, combined with a favorable safety profile and enhanced potency, could revolutionize preventative strategies and treatment options. Further research and development will focus on confirming these promising results and potentially establishing VYD2311 as a first-line defense against COVID-19 for a broader population.
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**Tags:** eClinical Tech News
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### [Seclidemstat and Azacitidine Trial for Hematologic Cancers Patient Enrollment Resumes](https://www.clinicaltrialvanguard.com/news/seclidemstat-and-azacitidine-trial-for-hematologic-cancers-patient-enrollment-resumes/)
**Published:** February 4, 2025
**Author:** Jon Napitupulu
**Content:**
Salarius Pharmaceuticals (Nasdaq: SLRX) has announced the resumption of patient enrollment in a Phase 1/2 clinical trial at MD Anderson Cancer Center (MDACC). The trial is investigating seclidemstat, an oral LSD1 inhibitor, in combination with azacitidine for treating myelodysplastic syndrome (MDS) and chronic myelomonocytic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (CMML). The FDA previously placed a partial clinical hold on the trial due to an unexpected adverse event, but this hold has been lifted after MDACC addressed the FDA’s concerns.
The restart of this trial is a significant development for patients with MDS and CMML, especially those who have relapsed or not responded to hypomethylating agent therapy. Interim results presented in June 2024 showed a promising 43% overall response rate and a median overall survival of 18.5 months in a group of these higher-risk patients. This is notable considering the typical overall survival after failing hypomethylating agents is significantly shorter, ranging from just four to six months. Further data from this trial could solidify seclidemstat’s potential to address an unmet need in this patient population.
The trial evaluates seclidemstat in combination with azacitidine. Earlier data demonstrated a 43% overall response rate in 14 higher-risk MDS and CMML patients who had previously failed hypomethylating agent therapy. Additionally, the reported median overall survival of 18.5 months is substantially higher than the typical 4–6 months survival post-hypomethylating agent failure.
While Salarius is merging with [Decoy Therapeutics](https://www.clinicaltrialvanguard.com/news/decoy-therapeutics-novel-inhibitors-show-promise-against-measles/) and focusing on Decoy’s pipeline, the development of seclidemstat for hematologic cancers will continue through this MDACC-led trial. This allows for continued investigation of seclidemstat’s potential benefits while the company explores strategic options for the drug’s future. The continued development of seclidemstat provides hope for patients with these challenging cancers. The data generated from this trial will be crucial for determining the next steps for seclidemstat and could attract potential partners interested in further advancing its development.
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**Tags:** eClinical Tech News
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### [Tenaya Therapeutics Gets $8M Grant for Heart Failure Research](https://www.clinicaltrialvanguard.com/news/tenaya-therapeutics-gets-8m-grant-for-heart-failure-research/)
**Published:** February 4, 2025
**Author:** Jon Napitupulu
**Content:**
Tenaya Therapeutics received an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to fund its Phase 1b [RIDGE](https://www.clinicaltrialvanguard.com/news/tenaya-therapeutics-unveils-interim-data-from-ridge-study-at-heart-rhythm-2025/)-1 clinical trial of [TN-401](https://www.clinicaltrialvanguard.com/news/tenaya-gene-therapies-advance-to-clinical-trials-following-positive-safety-reviews/) gene therapy. TN-401 is designed to treat arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the •PKP2• gene, a condition affecting an estimated 70,000 people in the U.S. The therapy delivers a functional •PKP2• gene into heart muscle cells using an adeno-associated virus serotype 9 (AAV9) capsid.
This funding is crucial because it supports the development of a potentially curative therapy for a serious, progressive heart disease that currently lacks effective treatment options. Existing treatments for •PKP2•-associated ARVC only manage symptoms, while TN-401 aims to address the underlying genetic cause. This offers a significant advancement in the field of cardiac gene therapy and provides hope for patients with this debilitating condition. The successful development of a gene therapy like TN-401 could dramatically improve long-term outcomes for ARVC patients, potentially preventing disease progression and reducing the risk of life-threatening complications.
The RIDGE-1 trial is an open-label, dose-escalation study assessing the safety, tolerability, and preliminary efficacy of a one-time intravenous infusion of TN-401 in symptomatic adults with •PKP2•-associated ARVC. Initial data from the low-dose cohort is anticipated in the second half of 2025. Preclinical studies have demonstrated TN-401’s ability to restore PKP2 protein levels, normalize heart rhythms, and improve cardiac function. The $8 million grant will cover a portion of the clinical trial costs, allowing Tenaya to continue advancing this promising therapeutic candidate.
The CIRM grant, along with the promising preclinical data, signifies a positive step forward in the fight against ARVC. The upcoming release of clinical trial data will be a critical inflection point, providing key insights into TN-401’s potential to transform the treatment landscape for this devastating disease. Positive results could lead to further investment, accelerated development, and ultimately, a much-needed therapeutic option for patients with •PKP2•-associated ARVC.
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### [Nexalin's Breakthrough DIFS™ Device Reduces Blood Pressure, Improves Mental Health](https://www.clinicaltrialvanguard.com/news/nexalins-breakthrough-difs-device-reduces-blood-pressure-improves-mental-health/)
**Published:** February 4, 2025
**Author:** Jon Napitupulu
**Content:**
Nexalin Technology, Inc. announced positive results from a clinical study published in the Journal of Affective Disorders, showing its Deep Intracranial Frequency Stimulation (DIFS) technology significantly reduced blood pressure in patients with [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) (MDD). The study highlighted that patients with higher baseline blood pressure experienced the most significant improvements, and the treatment was well-tolerated without reported adverse effects. The research demonstrates the potential of DIFS to address both mental and physical health challenges through a non-invasive approach.
This development holds significant implications for the treatment of MDD. The comorbidity of MDD and hypertension presents a complex challenge for clinicians. Current treatment options often focus solely on the [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) aspect, neglecting the interconnectedness of physical and mental well-being. Nexalin’s DIFS technology offers a potential solution by simultaneously addressing both conditions, potentially leading to improved patient outcomes and a more holistic approach to treatment. This could reduce the need for multiple medications and therapies, simplifying treatment regimens and potentially lessening the burden on patients.
The randomized controlled trial involved 68 first-episode, drug-naive MDD patients who received either active DIFS or sham stimulation over four weeks. Targeting brain regions associated with blood pressure regulation, such as the brainstem, hypothalamus, and thalamus, the active DIFS group experienced a reduction in systolic and diastolic blood pressure by 2.04 mmHg and 1.92 mmHg, respectively. These results were noticeable as early as week four.
The positive results from this study strengthen Nexalin’s position in the neurostimulation field. The findings support the potential of DIFS technology to expand beyond mental health applications and into broader areas of systemic wellness, such as [cardiovascular health](https://www.clinicaltrialvanguard.com/uncategorized/ema-reports-near-record-104-medicine-approvals-in-2025-with-strategic-focus-on-cardiovascular-health/). This could open new markets for Nexalin and position the company as a leader in innovative, non-invasive treatment solutions for a range of health conditions. With ongoing trials for other conditions like Alzheimer’s disease and PTSD, coupled with expanding regulatory approvals, Nexalin is poised for future growth and broader clinical application of its DIFS technology.
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**Categories:** News
**Tags:** eClinical Tech News
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### [FLT201 Gene Therapy Shows Promise in Gaucher Disease Trial](https://www.clinicaltrialvanguard.com/news/flt201-gene-therapy-shows-promise-in-gaucher-disease-trial/)
**Published:** February 5, 2025
**Author:** Jon Napitupulu
**Content:**
Spur Therapeutics released positive Phase 1/2 trial data for [FLT201](https://www.clinicaltrialvanguard.com/news/spur-therapeutics-presents-updated-gaucher-gene-therapy-data/), a gene therapy for Gaucher disease type 1. The therapy showed significant improvements in lyso-Gb1, a key disease marker, and maintained or improved blood counts, organ volume, and bone marrow burden after a single infusion. FLT201 also demonstrated a favorable safety profile.
This advancement is crucial for Gaucher disease patients who often experience persistent, debilitating symptoms despite existing treatments like enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). FLT201 offers the potential for a one-time treatment that could provide more effective and sustained relief from these symptoms, freeing patients from the burden of lifelong therapy. The positive safety profile observed so far further strengthens the therapy’s potential to improve patients’ quality of life significantly.
The Phase 1/2 GALILEO-1 trial involved six patients with Gaucher disease type 1, previously treated with ERT or SRT. Patients who received a single, low dose of FLT201 showed reductions in lyso-Gb1 ranging from 33% to 96%. Furthermore, they maintained normal hemoglobin levels and saw improvements or stabilization in platelet counts, spleen and liver volume, and bone marrow burden over a year after discontinuing prior treatments. Importantly, no serious adverse events or infusion reactions were observed. Spur Therapeutics has aligned with the FDA on a Phase 3 trial design, anticipating accelerated approval based on lyso-Gb1 reduction and full approval based on hemoglobin levels.
The promising Phase 1/2 results and FDA alignment suggest a positive outlook for FLT201. A successful Phase 3 trial could lead to a transformative treatment option for Gaucher disease, offering a one-time solution that significantly improves patient outcomes and reduces the need for continuous treatment. This development could reshape the Gaucher disease treatment landscape, offering a new standard of care.
Source link:[ https://www.globenewswire.com/news-release/2025/02/04/3020229/0/en/Spur-Therapeutics-Announces-Positive-Data-from-Phase-1-2-GALILEO-1-Trial-of-FLT201-Its-Gene-Therapy-Candidate-for-Gaucher-Disease-at-WORLDSymposium.html](https://www.globenewswire.com/news-release/2025/02/04/3020229/0/en/Spur-Therapeutics-Announces-Positive-Data-from-Phase-1-2-GALILEO-1-Trial-of-FLT201-Its-Gene-Therapy-Candidate-for-Gaucher-Disease-at-WORLDSymposium.html)
**Categories:** News
**Tags:** eClinical Tech News
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### [Auron Therapeutics Announces FDA Clearance and Series B Financing](https://www.clinicaltrialvanguard.com/news/auron-therapeutics-announces-fda-clearance-and-series-b-financing/)
**Published:** February 5, 2025
**Author:** Jon Napitupulu
**Content:**
Auron Therapeutics, a clinical-stage biotech company, announced progress on its lead KAT2A/B program and candidate [AUTX-703](https://www.clinicaltrialvanguard.com/news/fda-grants-auron-therapeutics-fast-track-designation-for-autx-703-to-treat-aml/), along with a successful $27 million Series B funding round. The FDA cleared Auron’s Investigational New Drug application, paving the way for clinical trials of AUTX-703, a novel oral KAT2A/B degrader, in patients with hematological malignancies. Preclinical data presented at the 2024 ASH Annual Meeting showed a significant survival advantage in acute myelogenous [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML) models, and the company plans to explore the drug’s efficacy in solid tumors like neuroendocrine prostate and small-cell [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/).
This development marks a critical step in addressing the need for novel therapies for hematological malignancies and potentially solid tumors. AUTX-703’s mechanism of action, targeting KAT2A/B degradation, represents a fresh approach with the potential to improve outcomes for patients who currently have limited treatment options. The positive preclinical data, particularly the survival advantage observed in AML models, suggests a promising therapeutic profile. This progress also validates Auron’s AURIGIN™ platform, demonstrating its capacity to identify and develop novel drug candidates.
The Series B funding will enable Auron to conduct a Phase 1 clinical proof-of-concept trial of AUTX-703 in AML patients, starting in the first quarter of 2025. The funds will also support further investigation into the potential of KAT2A/B inhibition in autoimmune diseases and continued development of novel EMT tumor targets identified using the AURIGIN platform. The financing round was led by DCVC Bio with participation from several other investors, including Apollo Health Ventures, Arkin Bio Ventures, and BrightEdge.
This news signifies a major advancement for Auron Therapeutics, positioning the company as a key player in the development of innovative cancer therapies. The initiation of clinical trials for AUTX-703 is a crucial milestone, bringing the company closer to potentially delivering a first-in-class treatment to patients with hematological malignancies and possibly other cancers. The expanded research into autoimmune diseases broadens the company’s therapeutic scope and further highlights the versatility of its drug discovery platform. This progress positions Auron for continued growth and reinforces its potential to make a significant impact on patient care.
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### [FDA Approves Susvimo for Diabetic Blindness](https://www.clinicaltrialvanguard.com/news/fda-approves-susvimo-for-diabetic-blindness/)
**Published:** February 5, 2025
**Author:** Jon Napitupulu
**Content:**
The FDA approved Susvimo ([ranibizumab](https://www.clinicaltrialvanguard.com/news/complete-12-week-results-of-norse-eight-trial-for-outlook-therapeutics/) injection) for diabetic macular edema (DME), a leading cause of vision loss. This marks the second indication for Susvimo, following its 2021 approval for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (AMD). The approval is based on the Phase III Pagoda study, which demonstrated Susvimo’s ability to maintain vision improvements in DME patients with a safety profile consistent with prior findings.
This approval significantly benefits DME patients, who previously faced frequent, often monthly, eye injections. Susvimo offers a less disruptive treatment option with refills required only twice a year, potentially improving patient adherence and quality of life. This also frees up valuable physician and clinic resources, enabling them to treat a greater number of patients. The expansion of Susvimo’s indication broadens its market reach, offering a significant advancement in DME treatment.
The Pagoda study showed Susvimo achieved non-inferior vision improvements compared to monthly ranibizumab injections. Specifically, patients receiving Susvimo every six months gained an average of 9.6 letters on an eye chart, comparable to the 9.4 letters gained by those receiving monthly injections. The implant delivers a customized ranibizumab formulation via the Port Delivery Platform, enabling continuous treatment. Genentech is providing support services to assist patients with access and reimbursement.
This FDA approval positions Susvimo as a leading treatment option for DME, potentially transforming the standard of care. The twice-yearly treatment regimen offers significant advantages for both patients and healthcare providers. This approval also reinforces Genentech’s commitment to ophthalmology and its continued development of innovative drug delivery systems for retinal diseases. Further research and real-world data will be crucial in fully assessing Susvimo’s long-term efficacy and impact on DME management.
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**Categories:** News
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### [GlucoTrack Announces Successful Human Clinical Trial of CGM](https://www.clinicaltrialvanguard.com/news/glucotrack-announces-successful-human-clinical-trial-of-cgm/)
**Published:** February 5, 2025
**Author:** Jon Napitupulu
**Content:**
[Glucotrack](https://www.clinicaltrialvanguard.com/news/glucotrack-targets-early-q2-for-us-fda-clinical-trial-filing/), Inc. has successfully completed its first human clinical study of a continuous blood glucose monitor (CBGM) implanted in the subclavian vein. The study focused on the safety of the device’s placement, usage, and removal in six participants with diabetes requiring intensive insulin therapy. No procedure or device-related serious adverse events were reported.
This successful initial human study is a crucial step towards potentially revolutionizing continuous glucose monitoring for diabetes patients. Current continuous glucose monitors rely on measuring glucose in interstitial fluid, which can be less accurate and timely than direct blood glucose measurement. Glucotrack’s CBGM, implanted directly into the bloodstream, promises more accurate and real-time data, potentially leading to better glucose control and improved outcomes for patients. This advancement could also significantly impact diabetes management by reducing the need for frequent finger-prick tests and offering a more convenient, long-term solution.
The four-day in-hospital study involved placing the CBGM sensor lead intravascularly and connecting it to a prototype electronics component on the skin. The primary endpoint of safety was met, and the study confirmed the functionality of the sensor lead within the subclavian vein. While the study was not designed to assess accuracy, the prototype system performed as expected based on prior animal studies. Interventional cardiologists successfully performed the placement and removal procedures. The company is developing the CBGM as a long-term implantable device, aiming for a sensor longevity of three years with no external wearable component.
This positive outcome paves the way for Glucotrack to move forward with larger, longer-term clinical trials to further evaluate the safety, efficacy, and accuracy of the CBGM system. This development holds promise for a significant advancement in diabetes technology, potentially transforming how patients manage their condition and ultimately improving their quality of life.
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### [Annovis' Buntanetap Enters Phase 3 Alzheimer's Study](https://www.clinicaltrialvanguard.com/news/annovis-buntanetap-enters-phase-3-alzheimers-study/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Annovis Bio has initiated its pivotal Phase 3 trial for buntanetap, an oral drug candidate for early-stage [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. The trial will assess both the symptomatic and disease-modifying potential of the drug over 18 months in over 750 participants. This follows promising results from a Phase 2/3 study showing cognitive improvement in early AD patients without safety concerns.
This Phase 3 trial represents a crucial step towards a potential treatment option for Alzheimer’s, a devastating disease with significant unmet medical needs. The dual focus on both symptom management and disease modification is particularly important, offering the possibility of not only improving patients’ quality of life but also slowing or halting disease progression. Positive results could significantly reshape the Alzheimer’s treatment landscape.
The 18-month trial is structured in two parts: a 6-month symptomatic assessment and a subsequent 12-month evaluation of disease-modifying effects. Annovis recently secured $21 million through a public offering to fund the initial 6-month phase, with anticipated warrant exercises covering the remaining 12 months. The primary outcomes will measure cognitive changes using the ADAS-Cog13 subscale and functional abilities using the ADCS-iADL scale. Recruitment has begun at two sites in Florida and New Jersey.
The commencement of this Phase 3 trial signifies a critical juncture for Annovis and the potential future of Alzheimer’s treatment. Successful trial results could lead to market approval for buntanetap, providing a much-needed new therapy and potentially validating Annovis’s neurodegenerative disease platform. The coming months will be crucial as data emerges from this pivotal study.
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### [Study Shows ux111 Gene Therapy Improves Clinical Function in Children with Sanfilippo Syndrome Type A](https://www.clinicaltrialvanguard.com/news/study-shows-ux111-gene-therapy-improves-clinical-function-in-children-with-sanfilippo-syndrome-type-a/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Ultragenyx Pharmaceutical announced positive data from its pivotal and long-term follow-up studies of UX111 (ABO-102), a gene therapy for Sanfilippo syndrome type A (MPS IIIA). The therapy showed statistically significant improvements in cognitive, receptive communication, and expressive communication scores compared to natural history data, correlating with substantial reductions in heparan sulfate (HS) in cerebrospinal fluid (CSF). These findings suggest the potential for UX111 to address the unmet medical need in MPS IIIA, a rare and fatal lysosomal storage disease currently lacking approved treatments.
This news holds significant promise for children with MPS IIIA, a devastating neurodegenerative disease that typically results in early death. The demonstrated cognitive and communication improvements, along with the retention of essential functional abilities in older patients, offer a potential shift in the disease trajectory. This could translate into meaningful extensions of quality of life, potentially allowing children to maintain abilities like walking, communicating, and self-feeding, which are progressively lost in untreated individuals. These outcomes underscore the potential of gene therapy to address the root cause of the disease and provide long-term benefits.
The data show a median reduction of 65% in CSF-HS levels across all treated patients (N=27) and 66% in the modified intent-to-treat (mITT) group (N=17), with a mean follow-up of 34 and 36 months, respectively. The mITT group demonstrated a +22.7 point improvement in mean Bayley-III cognitive raw score between 24 and 60 months of age compared to a decline of -6.8 points in natural history data. Improvements were also observed in receptive and expressive communication and fine motor skills. Importantly, older children outside the mITT group retained vital functional abilities, such as communication, ambulation, and self-feeding, offering hope for slowing disease progression even in later stages. The safety profile of UX111 remained favorable, with primarily mild to moderate, reversible liver enzyme elevations reported. A [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) for accelerated approval has been filed with the FDA, with a decision expected in the second half of 2025.
The positive data for UX111 marks a potential turning point in the treatment of MPS IIIA. If approved, UX111 could become the first disease-modifying therapy for this devastating condition, offering a new standard of care. This development could also spur further research and development into gene therapies for other lysosomal storage disorders, potentially benefiting a broader population of patients with rare genetic diseases. The upcoming FDA decision will be a critical milestone, determining the availability of this promising therapy for patients and shaping the future landscape of MPS IIIA treatment.
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### [Arbor Biotechnologies' ABO-101 Gets FDA Orphan Drug, Rare Pediatric Disease Designations](https://www.clinicaltrialvanguard.com/news/arbor-biotechnologies-abo-101-gets-fda-orphan-drug-rare-pediatric-disease-designations/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Arbor Biotechnologies received Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA for [ABO-101](https://www.clinicaltrialvanguard.com/news/arbor-biotechnologies-doses-first-patient-in-abo-101-gene-editing-study/), a gene editing therapy for primary hyperoxaluria type 1 (PH1). This follows the FDA’s acceptance of the Investigational New Drug application, paving the way for a Phase 1/2 trial in the first half of 2025. Preclinical data and the trial design will be presented at the International Pediatric Nephrology Association Congress in February 2025.
These designations are crucial because they highlight the significant unmet need for PH1 treatments, especially for children who often experience early onset of severe symptoms. The potential for a one-time, curative treatment addresses the limitations of current PH1 management, which primarily involves managing symptoms and complications rather than addressing the underlying genetic cause. This represents a potential paradigm shift in PH1 treatment. The designations also provide Arbor with financial incentives, including tax credits and fee waivers, which can help accelerate the development and eventual commercialization of ABO-101.
ABO-101, delivered via a lipid nanoparticle, targets the •HAO1• gene in the liver. This gene is responsible for the overproduction of oxalate, the root cause of PH1 complications. By precisely editing the •HAO1• gene, ABO-101 aims to permanently reduce oxalate production and prevent the debilitating effects of the disease. The upcoming Phase 1/2 trial will evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric PH1 patients.
The FDA designations and upcoming clinical trial represent a critical step towards a potential cure for PH1. Positive trial results could validate Arbor’s gene editing platform and establish ABO-101 as a first-in-class therapy. This could lead to a significant improvement in the quality of life for PH1 patients and potentially transform the treatment landscape for other rare genetic diseases.
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### [Ziftomenib Shows Promise in AML Monotherapy & Combination Trials](https://www.clinicaltrialvanguard.com/news/ziftomenib-shows-promise-in-aml-monotherapy-combination-trials/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Kura Oncology and Kyowa Kirin announced positive topline results from the KOMET-001 Phase 2 trial of [ziftomenib](https://www.clinicaltrialvanguard.com/news/ziftomenib-plus-chemotherapy-shows-94-survival-in-npm1-mutant-aml/), an oral menin inhibitor, in patients with relapsed/refractory NPM1-mutant acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML). The trial met its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh), with a statistically significant result and an encouraging safety profile. An NDA submission is planned for Q2 2025.
This positive outcome is crucial for AML patients, particularly those with the NPM1 mutation, who face a high risk of relapse and limited treatment options. The achievement of the primary endpoint suggests ziftomenib could offer a new therapeutic avenue for this patient population, potentially improving remission rates and overall survival. The upcoming NDA submission signifies a significant step towards bringing this potential treatment to market.
The KOMET-001 trial achieved its primary endpoint of CR/CRh, demonstrating the efficacy of ziftomenib as a monotherapy in this difficult-to-treat patient population. Furthermore, the companies received positive FDA feedback on the design of the upcoming KOMET-017 Phase 3 trial, which will evaluate ziftomenib in combination with standard therapies in both newly diagnosed and relapsed/refractory NPM1-mutant and KMT2A-rearranged AML patients. This trial incorporates dual primary endpoints (MRD-negative CR and event-free survival for the intensive arm; CR and overall survival for the non-intensive arm) designed to support potential accelerated and full approvals in the US. The companies also announced additional data presentations anticipated throughout 2025 related to ziftomenib and other pipeline programs focusing on other cancers and treatment combinations.
The positive KOMET-001 results and planned KOMET-017 trial position ziftomenib as a potential game-changer in the AML treatment landscape. If approved, ziftomenib could become a key therapy for patients with NPM1-mutant and KMT2A-rearranged AML, both in the relapsed/refractory and newly diagnosed settings. This could significantly improve patient outcomes and address a substantial unmet medical need in this challenging disease. The upcoming NDA submission and initiation of the Phase 3 trial will be critical milestones to watch, as they will further define the role of ziftomenib in the future of AML treatment.
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### [Zenocutuzumab Efficacy in NRG1+ Cancer: Groundbreaking Results](https://www.clinicaltrialvanguard.com/news/zenocutuzumab-efficacy-in-nrg1-cancer-groundbreaking-results/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Merus N.V. announced that the New England Journal of Medicine (NEJM) published positive results from the phase 2 eNRGy trial of Bizengri (zenocutuzumab) for treating advanced NRG1 fusion-positive pancreatic adenocarcinoma and [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). This marks the first approved therapy specifically targeting this rare genetic alteration, addressing an unmet medical need for these patient populations. The license for commercializing Bizengri in the U.S. has been granted to Partner Therapeutics, Inc.
This publication is a major milestone for the oncology field. It validates NRG1 fusions as viable drug targets and offers a new treatment option for patients with these difficult-to-treat cancers who previously lacked targeted therapies. The NEJM publication lends significant credibility to Bizengri, potentially encouraging earlier diagnosis of NRG1 fusions and broader adoption of this targeted treatment.
The eNRGy trial demonstrated Bizengri’s efficacy across various tumor types, with particularly promising results in NSCLC and pancreatic adenocarcinoma. The therapy showed a favorable safety profile, although potential side effects like infusion-related reactions and interstitial lung disease were noted. The study enrolled 204 patients with 12 different tumor types, providing a comprehensive assessment of Bizengri’s activity and safety profile. Detailed results for the NSCLC and [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) cohorts, including overall response rate and duration of response, are highlighted in the publication.
The NEJM publication and the partnership with Partner Therapeutics positions Bizengri for a successful U.S. launch. This could stimulate further research into NRG1 fusion biology and the development of additional therapies targeting this pathway. The availability of a targeted treatment like Bizengri is likely to improve outcomes and quality of life for patients with NRG1 fusion-positive cancers.
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### [BioXcel: Business & Clinical Update](https://www.clinicaltrialvanguard.com/news/bioxcel-business-clinical-update/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
BioXcel Therapeutics provided an update on its late-stage clinical trials for [BXCL501](https://www.clinicaltrialvanguard.com/news/bioxcels-bxcl501-shows-efficacy-across-severity-levels-in-at-home-agitation-trial/), a drug for acute agitation, and highlighted recent corporate developments. The company is advancing the SERENITY at-home trial for agitation related to bipolar disorders or [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) and planning the TRANQUILITY in-care trial for agitation in Alzheimer’s dementia. BioXcel also improved its financial position through a credit amendment and equity funding, and strengthened its leadership with new board appointments.
The progress of these clinical trials is crucial as acute agitation represents a significant unmet need for millions of individuals affected by bipolar disorders, schizophrenia, and Alzheimer’s dementia. Effective treatments are limited, and BXCL501 offers a potential new approach to rapidly manage these episodes, especially in the at-home setting, which is a significant development for patient care. Additionally, the focus on Alzheimer’s dementia addresses a growing patient population with specific agitation challenges.
The SERENITY trial is actively enrolling patients, and the TRANQUILITY trial design is finalized. While continuing to supply its currently approved drug, [IGALMI](https://www.clinicaltrialvanguard.com/news/bioxcel-awaits-fda-decision-on-igalmi-at-home-label-expansion/), BioXcel secured $7 million in equity funding and amended its existing credit agreement to improve financial stability. Two key appointments to the Board of Directors bring extensive clinical, financial, and legal expertise, further bolstering the company’s strategic direction.
These developments collectively position BioXcel to potentially address a significant market need with BXCL501 while ensuring operational and financial stability. The ongoing clinical trials, coupled with enhanced leadership and financial flexibility, suggest a strong focus on bringing this potential treatment to market, which could significantly impact the landscape of agitation management in various neuropsychiatric disorders.
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### [Enveda's First-in-Class ENV-294 for Asthma: A Breakthrough](https://www.clinicaltrialvanguard.com/news/envedas-first-in-class-env-294-for-asthma-a-breakthrough/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Enveda Biosciences is testing its lead drug candidate, ENV-294, for [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/), marking its second clinical application after [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/). The drug is a small molecule designed to address shared inflammatory pathways through a novel mechanism, offering a potential non-steroidal, oral treatment option. The company has formed an advisory board of asthma experts to guide development.
This development is important because it addresses a significant unmet need in asthma treatment. A new oral, non-steroidal medication could offer a much-needed alternative for patients who experience limitations with current therapies, potentially improving adherence and outcomes. This also positions Enveda as a key player in the respiratory disease space, showcasing its platform’s capacity to identify promising compounds for multiple indications.
ENV-294 has already undergone Phase 1 trials in healthy subjects for atopic dermatitis and demonstrated efficacy in preclinical asthma models. This positive data, coupled with a robust safety profile in earlier studies, supports the drug’s potential in this new indication. The formation of a specialized advisory board indicates a strategic commitment to accelerating the development process.
This expansion into asthma treatment signifies a promising step forward for both Enveda and patients. If successful, ENV-294 could represent a significant advancement in asthma management, offering a new therapeutic approach and potentially improving the quality of life for many individuals. The progress of ENV-294 will be crucial to watch, as it could signal a broader shift in how inflammatory respiratory conditions are treated.
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### [AgelessRx Launches Oral GLP-1 Drops for Metabolic Health](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
[AgelessRx](https://www.clinicaltrialvanguard.com/news/agelessrx-enters-sleep-health-market/) has launched oral sublingual drops for the GLP-1 receptor agonists [Semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/) and [Tirzepatide](https://www.clinicaltrialvanguard.com/news/aardvarks-new-obesity-pipeline-data-offers-surprising-hope/), offering a non-injectable alternative for metabolic health management. This move aims to broaden access to therapies that may combat age-related decline and promote longevity. The company believes GLP-1s are crucial for overall health and healthspan due to their potential impact on metabolic function.
This development is important because it addresses a significant barrier to access for individuals seeking the potential benefits of GLP-1 therapies. Injectable medications can be inconvenient and deter some patients. The oral sublingual delivery method may improve patient compliance and expand the reach of these treatments to a wider population, including those who may have previously been hesitant due to the need for injections. This could significantly impact the adoption of GLP-1s for metabolic health and longevity purposes.
AgelessRx’s sublingual drops utilize a SubMagna SL HMW compound for faster absorption into the bloodstream. The company’s approach emphasizes preventative care and personalized treatment, aligning with core principles of longevity science. Their Stanford PhD-led Applied Sciences team is also researching broader applications of GLP-1s in extending lifespan.
This launch positions AgelessRx as an innovator in the longevity field. Providing accessible and convenient GLP-1 treatments could accelerate the mainstream adoption of longevity-focused therapies. This could further drive research and development in the field and potentially transform how we approach age-related health management in the future.
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### [Vaxxas Appoints Global Vaccine Experts for Needle-Free Trials](https://www.clinicaltrialvanguard.com/news/vaxxas-appoints-global-vaccine-experts-for-needle-free-trials/)
**Published:** February 6, 2025
**Author:** Jon Napitupulu
**Content:**
Vaxxas, a clinical-stage biotech company, has appointed three prominent vaccine experts as advisors to guide product development and strategy. These additions aim to support the company’s progress toward later-stage clinical trials and commercialization of its high-density microarray patch (HD-MAP) vaccine technology. The company is focused on advancing its [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/), influenza, and RSV vaccine candidates delivered via the HD-MAP.
This move is crucial for Vaxxas as it signals a commitment to accelerating the development and potential market entry of its novel vaccine delivery platform. Securing experienced advisors with expertise in clinical development, regulatory affairs, and manufacturing provides invaluable guidance as the company transitions to larger-scale trials and prepares for eventual commercialization. The focus on key markets like the US and Europe underscores the company’s strategic approach to maximizing impact and accessibility of its technology. This development also represents a significant step towards broader adoption of needle-free vaccine delivery, potentially addressing global vaccination challenges.
Vaxxas’ HD-MAP technology has already undergone five successful Phase I clinical trials, involving more than 500 participants. A US-based Phase I trial for a pre-pandemic influenza vaccine, funded by the Biomedical Advanced Research and Development Authority ([BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/)), is also underway. The new advisors join Dr. Charles Knirsch, who joined Vaxxas in July 2024 as a clinical and medical advisor.
The expansion of the advisory team, coupled with ongoing clinical trials and secured funding, positions Vaxxas for significant growth. This strategic move is likely to accelerate the development timeline for HD-MAP delivered vaccines and potentially revolutionize vaccine administration globally in the coming years. The company anticipates commercial availability of its vaccine patches within the next three to five years, signifying a potential paradigm shift in how vaccines are administered worldwide.
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### [Trinity Biotech CGM Trial Shows Landmark First-Day Accuracy Gains](https://www.clinicaltrialvanguard.com/news/trinity-biotech-cgm-trial-shows-landmark-first-day-accuracy-gains/)
**Published:** February 7, 2025
**Author:** Jon Napitupulu
**Content:**
[Trinity Biotech](https://www.clinicaltrialvanguard.com/news/trinity-biotech-to-advance-prostate-cancer-test-with-partner/) announced positive findings from a pre-pivotal trial of its next-generation continuous glucose monitoring (CGM) system, demonstrating significant improvements in first-day accuracy. The redesigned sensor showed a 35% improvement in Mean Absolute Relative Difference (MARD) and a greater than 50% improvement in Mean Absolute Difference (MAD) compared to the previous Waveform product. This trial builds upon previous positive results showing overall improved signal quality, reliability, and accuracy.
This improved first-day accuracy addresses a critical industry-wide challenge in CGM technology. Historically, the initial 24 hours of sensor use have been problematic due to fluctuating readings caused by the body’s response to sensor insertion. This inconsistency not only frustrates users but also raises safety concerns, potentially delaying or hindering proper diabetes management. By significantly improving first-day performance, Trinity Biotech’s new CGM system could encourage wider adoption and improve patient trust in CGM technology.
The pre-pivotal trial involved 30 participants, mostly with [Type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/), who wore multiple sensors for 15 days. Beyond first-day improvements, the redesigned sensor demonstrated a 25-30% overall improvement in MARD compared to earlier models and achieved industry-standard low-glucose precision (MAD), crucial for managing hypoglycemia. The company highlights the system’s ergonomic, modular design, featuring reusable and rechargeable components to reduce costs and environmental impact.
These results pave the way for Trinity Biotech to pursue regulatory submissions in Europe in 2025 and the U.S. in 2026. The company aims to commercialize the CGM system for both diabetes patients and health-conscious consumers. Further pre-pivotal trials focusing on additional device enhancements are planned for Q1 2025. This progress suggests a potential shift in the CGM market, making the technology more accessible and reliable for a broader user base.
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### [Tonix CD40L MAb Shows Promise in Kidney Transplant Rejection](https://www.clinicaltrialvanguard.com/news/tonix-cd40l-mab-shows-promise-in-kidney-transplant-rejection/)
**Published:** February 7, 2025
**Author:** Jon Napitupulu
**Content:**
Tonix Pharmaceuticals announced positive topline results from its Phase 1 single ascending dose trial of TNX-1500, an anti-[CD40L](https://www.clinicaltrialvanguard.com/news/eledon-reports-preliminary-tegoprubart-data-in-diabetes-trial/) monoclonal antibody, in healthy participants. The trial assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous TNX-1500 to inform dosing for a planned Phase 2 trial in kidney transplant recipients. The results support the continuation of TNX-1500 development for the prevention of kidney transplant rejection.
This positive Phase 1 data is important because it validates the potential of TNX-1500 as a next-generation anti-CD40L mAb with an improved safety profile compared to first-generation treatments like ruplizumab, which were hampered by thrombotic risks. The findings suggest TNX-1500 could offer a more effective and safer approach to preventing transplant rejection, potentially improving long-term graft survival and reducing the toxicity burden associated with current immunosuppressive regimens. This represents a critical step towards addressing the ongoing need for better transplant rejection prevention strategies.
In the Phase 1 trial, TNX-1500 blocked primary and secondary antibody responses to a test antigen at doses of 10 mg/kg and 30 mg/kg. The drug exhibited a mean half-life of 34-38 days at these doses, supporting a convenient monthly dosing regimen for future trials. TNX-1500 was generally well-tolerated, with no serious adverse events or thromboembolic events observed. Importantly, the only treatment-emergent adverse event occurring in three or more participants was mild aphthous ulcers, which resolved quickly.
These results pave the way for Tonix to engage with the FDA and plan a Phase 2 efficacy study in kidney transplant recipients. The positive safety and pharmacodynamic data suggest TNX-1500 holds promise as a potential best-in-class therapy for preventing organ rejection and treating autoimmune diseases, representing a significant advancement in the field of transplantation and immunology.
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### [Wave Life Sciences Initiates Phase 1 Inlight Trial of WVE-007 in Obesity](https://www.clinicaltrialvanguard.com/news/wave-life-sciences-initiates-phase-1-inlight-trial-of-wve-007-in-obesity/)
**Published:** February 7, 2025
**Author:** Jon Napitupulu
**Content:**
Wave [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) has initiated a Phase 1 clinical trial, INLIGHT, for [WVE-007](https://www.clinicaltrialvanguard.com/news/wave-life-sciences-advances-wve-007-to-phase-2a-for-obesity-and-metabolic-disease/), a novel RNA interference (RNAi) therapy targeting obesity. WVE-007, administered as a long-acting GalNAc-siRNA, targets INHBE mRNA, which encodes a protein that inhibits fat burning. The company anticipates releasing proof-of-concept data from the trial in 2025.
This development is potentially groundbreaking for the obesity treatment landscape. Current therapies often struggle to achieve substantial, sustainable weight loss while preserving lean muscle mass. WVE-007’s mechanism, focused on directly influencing fat cell metabolism, offers a unique approach that could address these challenges. The potential for infrequent dosing (once or twice yearly) further differentiates it, potentially improving patient adherence and long-term outcomes. This approach could significantly impact the lives of over a billion individuals globally affected by obesity, offering a new avenue for effective and convenient weight management.
The INLIGHT trial will evaluate WVE-007’s safety, tolerability, pharmacokinetics, and efficacy in adults with overweight or obesity. The study will measure biomarkers for target engagement, body weight and composition changes, and [metabolic health](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/) improvements. The focus on fat loss while preserving lean mass aligns with recent FDA guidance on weight-loss therapeutics. WVE-007’s preclinical data suggests its potential as a monotherapy, in combination with GLP-1 receptor agonists, and as a maintenance therapy to prevent weight regain after discontinuing GLP-1 treatment.
The commencement of the INLIGHT trial marks a significant milestone for Wave Life Sciences, representing their first siRNA therapy to enter clinical development. Positive results from this trial could validate their RNAi platform and pave the way for future applications in addressing other common diseases. The data expected in 2025 will be critical in determining the future development trajectory of WVE-007 and its potential to reshape the obesity treatment paradigm.
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### [CytoDyn Shows Statistically Significant Fibrosis Reversal](https://www.clinicaltrialvanguard.com/news/cytodyn-shows-statistically-significant-fibrosis-reversal/)
**Published:** February 7, 2025
**Author:** Jon Napitupulu
**Content:**
[CytoDyn](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/) Inc. announced positive preclinical results for leronlimab, demonstrating a statistically significant reversal of liver fibrosis in three separate studies conducted with SMC Laboratories. These studies utilized different models of liver fibrosis, including a high-fat diet and a fibrosis-inducing agent, and all showed leronlimab monotherapy significantly outperformed the control group. The company believes this is due to leronlimab’s ability to bind to CCR5 receptors on hepatic stellate cells.
This preclinical success strengthens the potential of leronlimab as a treatment for liver fibrosis, an area with significant unmet medical need. Current treatment options for liver fibrosis are limited, and the progression to cirrhosis can lead to liver failure and the need for transplantation. Leronlimab’s mechanism of action, targeting CCR5 receptors, suggests it could offer a new and potentially effective approach to managing and even reversing this condition. The diverse models used in the studies provide a broader base of evidence for leronlimab’s efficacy. This could accelerate the development of leronlimab for liver fibrosis and potentially expand its application to other fibrotic diseases in organs like the lungs and heart.
The studies evaluated leronlimab in mouse models using both a high-fat diet combined with a single dose of Streptozocin, and a fibrosis-inducing agent. All three studies demonstrated a statistically significant reversal of liver fibrosis (p-values < 0.01) compared to the control group. While CytoDyn is prioritizing its oncology objectives for 2025, it is actively seeking partnerships to advance leronlimab’s clinical development for liver fibrosis and other potential indications. These positive preclinical findings represent a crucial step towards developing a much-needed treatment for liver fibrosis. The potential for partnerships and further clinical trials suggests that leronlimab could eventually offer a new therapeutic avenue for patients with this serious condition. Furthermore, the potential application of leronlimab to other fibrotic diseases may broaden its impact across multiple therapeutic areas. This development positions leronlimab as a promising candidate in an area of high unmet medical need and reinforces the potential of CCR5 antagonism as a therapeutic strategy.
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### [Daring Denali Therapeutics MPS II Study Delivers!](https://www.clinicaltrialvanguard.com/news/daring-denali-therapeutics-mps-ii-study-delivers/)
**Published:** February 7, 2025
**Author:** Jon Napitupulu
**Content:**
Denali Therapeutics announced primary analysis results from a Phase 1/2 study of tividenofusp alfa (DNL310) in 47 participants with Hunter syndrome (MPS II). Long-term data show sustained biomarker reductions, improvements in hearing, cognition, and adaptive behavior, and a generally well-tolerated safety profile over a median follow-up of two years and up to four years. Denali plans to submit a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) for accelerated approval in early 2025, aiming for a U.S. launch in late 2025 or early 2026.
This development is potentially transformative for individuals with Hunter syndrome, a rare genetic disease with limited treatment options. Current treatments only partially address physical symptoms and do not cross the blood-brain barrier, leaving cognitive and behavioral impairments unaddressed. Tividenofusp alfa, engineered to deliver the IDS enzyme across the blood-brain barrier, offers a potential solution for the full spectrum of Hunter syndrome manifestations, potentially improving the quality of life for those affected.
The Phase 1/2 study achieved substantial reductions in key disease biomarkers in the central nervous system and periphery. This included normalization of cerebrospinal fluid and urine heparan sulfate, and neurofilament light, a marker of neurodegeneration. Clinically, improvements were seen in liver volume, hearing thresholds, adaptive behavior, and cognition. Most treatment-related adverse events were mild or moderate, primarily infusion-related reactions, anemia, vomiting, fever, respiratory infections, and rash. Serious adverse events were reported in a small percentage of participants and were manageable.
The positive long-term data and upcoming BLA submission position tividenofusp alfa as a promising potential therapy for Hunter syndrome. If approved, it could significantly alter the treatment landscape for this rare disease, addressing unmet needs for a therapy that targets both physical and neurocognitive symptoms. This progress also holds implications for Denali’s broader lysosomal storage disease portfolio, potentially accelerating the development of similar therapies for other conditions like Sanfilippo syndrome Type A.
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### [Eylea HD: 3-Year Results Show Durable Vision Gains in Wet AMD](https://www.clinicaltrialvanguard.com/news/eylea-hd-3-year-results-show-durable-vision-gains-in-wet-amd/)
**Published:** February 10, 2025
**Author:** Jon Napitupulu
**Content:**
Regeneron announced positive three-year results from an extension study of the Phase 3 PULSAR trial for EYLEA HD (aflibercept 8 mg) in patients with wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wAMD). The study showed that the majority of patients maintained visual and anatomical improvements with extended dosing intervals, some as long as six months. Data also revealed that patients switching from EYLEA (aflibercept 2 mg) to EYLEA HD achieved similar benefits with fewer injections.
These findings are crucial for advancing wAMD treatment paradigms. Longer dosing intervals offer significant advantages for patients, potentially reducing the burden of frequent injections and improving their quality of life by lessening the need for clinic visits. For healthcare systems, the reduced treatment frequency could translate to greater resource efficiency. This progress is particularly relevant considering the aging population and the increasing prevalence of wAMD, which poses a growing challenge to healthcare resources.
In the PULSAR extension study, nearly 60% of EYLEA HD patients achieved a dosing interval of at least four months, with 40% reaching five months or more, and 24% reaching six months. Patients who transitioned from EYLEA 2 mg to EYLEA HD in the extension study also saw sustained visual and anatomical benefits, with 79% achieving dosing intervals of three months or longer, and 43% reaching four months or longer. The safety profile of EYLEA HD remained consistent with previous trials, showing comparable outcomes to EYLEA 2 mg.
These positive long-term results solidify EYLEA HD’s potential to become a leading treatment option for wAMD. The extended dosing intervals achieved in this study represent a meaningful step towards less intensive treatment regimens, offering a potential paradigm shift in how wAMD is managed. This could lead to increased patient adherence to treatment, improved clinical outcomes, and ultimately, a better quality of life for individuals living with this challenging condition. Furthermore, this development could encourage further research into optimizing treatment regimens and exploring even longer dosing intervals with EYLEA HD in the future.
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### [Ampligen® & Flumist®: AIM Immunotech's Innovative Avian Flu Trial](https://www.clinicaltrialvanguard.com/news/ampligen-flumist-aim-immunotechs-innovative-avian-flu-trial/)
**Published:** February 10, 2025
**Author:** Jon Napitupulu
**Content:**
AIM [ImmunoTech](https://www.clinicaltrialvanguard.com/news/immunotech-announces-cash-conservation-plan/) Inc. is initiating a plan to further develop Ampligen as a vaccine adjuvant for avian influenza. The company has engaged Amarex Clinical Research to manage an Investigational New Drug (IND) application for a combination therapy study involving Ampligen and [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)’s FluMist. AIM is actively pursuing grants to support the study’s costs.
This development is crucial because avian influenza poses a significant and growing threat to public health. A successful combination therapy could offer broader protection and easier administration (intranasal) compared to current influenza vaccines. This approach has the potential to address a critical unmet need for a more effective and accessible preventative measure against avian flu, which could significantly impact public health outcomes globally.
This planned IND builds upon previous clinical research at the University of Alabama-Birmingham, where intranasal Ampligen administration following FluMist amplified the immune response to seasonal influenza strains and induced cross-reactive antibodies against highly pathogenic avian influenza strains (H5N1, H7N9, and H7N3). Prior pre-clinical studies demonstrated Ampligen’s ability to induce cross-reactive antibodies against H5N1 in mice when administered with a trivalent flu vaccine and also showed protective effects against H5N1 in non-human primates. A human safety study confirmed that repeated intranasal Ampligen administration was well-tolerated.
This initiative marks a critical step in advancing a potential solution for the increasing threat of avian influenza. The combination of Ampligen and FluMist offers a promising approach to enhance vaccine efficacy and broaden protection. The planned clinical trial, along with continued research and development, could lead to a significant advancement in preventing avian influenza and safeguarding public health.
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### [Positive Phase 2 Data for Subcutaneous Migaldendranib at Angiogenesis 2025](https://www.clinicaltrialvanguard.com/news/positive-phase-2-data-for-subcutaneous-migaldendranib-at-angiogenesis-2025/)
**Published:** February 11, 2025
**Author:** Jon Napitupulu
**Content:**
Ashvattha Therapeutics announced positive Phase 2 trial data for its subcutaneously administered nanomedicine, [Migaldendranib](https://www.clinicaltrialvanguard.com/news/ashvattha-therapeutics-positive-interim-phase-2-results-for-dme-and-wet-amd/) (MGB), designed to treat retinal diseases like wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD) and diabetic macular edema (DME). The therapy aims to reduce the need for frequent intravitreal (IVT) injections by targeting and reducing fluid production in the retina of both eyes. Interim results from the trial involving 25 pre-treated patients demonstrated a significant reduction in the need for anti-VEGF IVT injections while maintaining or improving visual acuity.
This development is potentially transformative for patients with chronic retinal diseases. Current treatment relies on regular IVT injections, which are invasive, inconvenient, and can cause anxiety. A successful subcutaneous therapy could shift the treatment paradigm, offering patients a more comfortable, self-administered alternative, leading to improved compliance and potentially better outcomes. Decreased reliance on clinic visits also frees up valuable resources for healthcare systems.
The Phase 2 trial data shows MGB reduced the need for anti-VEGF injections by 69.2% in wet AMD patients and 76.5% in DME patients over 24 weeks. Importantly, the therapy demonstrated efficacy in both eyes, even though only one eye received an initial IVT injection of aflibercept. The treatment was generally well-tolerated, with no reported drug-related ocular adverse events.
MGB’s positive Phase 2 results represent a significant step towards a less invasive and more patient-friendly treatment for retinal diseases. If further trials confirm these findings, MGB could revolutionize the management of these conditions, improving patients’ quality of life while potentially reducing the overall healthcare burden. Further research will focus on longer-term efficacy and safety, as well as exploring potential applications for other retinal diseases.
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### [Achieve Life Sciences Positive Orca-OL Trial Results](https://www.clinicaltrialvanguard.com/news/achieve-life-sciences-positive-orca-ol-trial-results/)
**Published:** February 11, 2025
**Author:** Jon Napitupulu
**Content:**
Achieve [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) announced positive safety results from the second review of their ongoing ORCA-OL trial for cytisinicline, a smoking cessation treatment. The Data Safety Monitoring Committee (DSMC) found no unexpected adverse events and noted excellent participant adherence to the medication. Achieve remains on track to submit a New Drug Application (NDA) in Q2 2025.
This positive safety data is crucial for cytisinicline’s potential market entry. It strengthens the drug’s profile, particularly given the substantial public health need for effective smoking cessation therapies. This clean safety record could encourage physician and patient adoption, potentially positioning cytisinicline as a preferred treatment option over existing therapies. Further, it reduces the risk of regulatory delays for the NDA submission, a significant factor for investors and stakeholders.
The ORCA-OL trial, designed to meet FDA safety requirements, enrolled 479 participants and has achieved its goal of 300 participants completing six months of treatment. This milestone was critical for the NDA submission. The trial’s success further validates the safety findings from previous Phase 2 and 3 trials involving over 1,600 subjects. The FDA requires six-month safety data from at least 300 participants and one-year data from 100 participants, which Achieve aims to provide during the NDA review.
This continued positive safety data reinforces Achieve’s progress toward regulatory approval and potential commercialization of cytisinicline. It suggests a higher likelihood of a successful NDA submission and a potentially expedited review process. If approved, cytisinicline could represent the first new prescription smoking cessation treatment in nearly two decades, offering a significant advancement in addressing the public health crisis of nicotine dependence. The anticipated approval could also establish Achieve as a key player in the smoking cessation market.
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### [Padcev® Plus Keytruda® Shows Long-Term Efficacy in Urothelial Cancer](https://www.clinicaltrialvanguard.com/news/padcev-plus-keytruda-shows-long-term-efficacy-in-urothelial-cancer/)
**Published:** February 11, 2025
**Author:** Jon Napitupulu
**Content:**
Pfizer and Astellas announced updated results from the Phase 3 EV-302 trial, showing sustained overall survival (OS) and progression-free survival (PFS) benefits with [enfortumab](https://www.clinicaltrialvanguard.com/news/merck-keytruda-and-padcev-advance-urothelial-carcinoma-treatment-an-uncommon-breakthrough/) vedotin plus [pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) in previously untreated locally advanced or metastatic urothelial cancer (la/mUC). After a median follow-up of 29.1 months, the combination therapy continued to demonstrate a significant reduction in the risk of death and disease progression compared to chemotherapy. These findings were presented at the 2025 ASCO GU Symposium.
This updated data solidifies the combination therapy’s position as a standard of care for la/mUC. The sustained efficacy observed over a longer follow-up period provides further confidence in the treatment’s long-term benefits for patients, especially given the historically poor prognosis associated with advanced urothelial cancer. This reinforces the potential for this combination to significantly improve patient outcomes and reshape the treatment landscape.
The combination therapy reduced the risk of death by 49% and extended median OS to 33.8 months compared to 15.9 months with chemotherapy. The risk of disease progression or death was reduced by 52%, with a median PFS of 12.5 months for the combination versus 6.3 months for chemotherapy. The confirmed objective response rate was 67.5% with the combination compared to 44.2% for chemotherapy. Importantly, these benefits were observed across all patient subgroups, including those eligible and ineligible for cisplatin. The safety profile remained consistent with earlier findings.
These results further establish enfortumab vedotin plus pembrolizumab as a crucial first-line treatment for la/mUC. The sustained efficacy observed with longer follow-up, coupled with the previously demonstrated improvements in OS and PFS, indicates the potential for improved long-term survival and disease control for patients with this aggressive cancer type. This continued success supports broader adoption of the combination therapy and suggests further exploration in other stages of urothelial cancer.
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### [Voyager Provides Update on SOD1 ALS Gene Therapy Program](https://www.clinicaltrialvanguard.com/news/voyager-provides-update-on-sod1-als-gene-therapy-program/)
**Published:** February 12, 2025
**Author:** Jon Napitupulu
**Content:**
Voyager Therapeutics is halting development of its SOD1 ALS gene therapy, VY9323, after three-month primate data revealed an off-target effect and a narrow therapeutic window. The company will explore alternative payloads for the treatment, while retaining its novel TRACER capsid, which performed well in other preclinical studies. This decision extends Voyager’s cash runway into mid-2027, excluding potential milestone payments.
This setback for VY9323 is important for the ALS community because SOD1 mutations are a known genetic driver of the disease, and gene therapies offer a potentially powerful approach to address the underlying cause. While other therapeutic strategies exist, they largely focus on symptom management rather than modifying disease progression. The pursuit of an effective SOD1-targeted therapy remains a critical area of research, and Voyager’s shift in strategy represents a lost opportunity, at least in the near term.
Voyager’s decision to explore alternative payloads suggests that the siRNA component of VY9323 is the root cause of the observed issues. The company’s confidence in the TRACER capsid, demonstrated by its successful performance in other programs, allows them to focus on optimizing the therapeutic payload. This approach could potentially accelerate the identification of a viable clinical candidate in the future. Financially, the extended cash runway provides Voyager with the resources to pursue these alternative strategies without immediate funding pressures. The continued development of other pipeline programs targeting GBA1 [Parkinson](https://www.clinicaltrialvanguard.com/clinical-trial-ops-brief/the-genetics-are-global-your-enrollment-plan-isnt/)’s disease, Friedreich’s ataxia, and tau-related pathologies remains unaffected.
The future of Voyager’s SOD1 program now hinges on its ability to identify and validate a suitable replacement payload. This will require additional preclinical research and likely push back any potential clinical trials. While this represents a delay in the development of a much-needed treatment for SOD1 ALS, Voyager’s decision demonstrates a commitment to developing a safe and effective therapy. The coming months will be crucial in determining whether a viable path forward for the SOD1 program can be identified, and whether the company can capitalize on the promise of its TRACER capsid technology.
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### [Corbus Pharmaceuticals Announces Clinical Data for CRB-701](https://www.clinicaltrialvanguard.com/news/corbus-pharmaceuticals-announces-clinical-data-for-crb-701/)
**Published:** February 12, 2025
**Author:** Jon Napitupulu
**Content:**
Corbus Pharmaceuticals announced that an abstract for its first-in-human dose-escalation clinical study of [CRB-701](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/) (SYS6002) for advanced solid tumors has been released. The study, conducted in the US and Europe, evaluates the safety, pharmacokinetics, and efficacy of CRB-701 in patients with high Nectin-4 expression. Updated data from 38 patients will be presented at the 2025 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU).
The upcoming data presentation at ASCO GU offers a crucial glimpse into the early clinical progress of CRB-701, a next-generation antibody-drug conjugate (ADC). This is particularly relevant for the oncology field, specifically for urothelial and other solid tumors expressing Nectin-4. Positive safety and efficacy signals at this stage could significantly de-risk further development and attract potential partnerships or investments. For patients, it represents potential progress toward new treatment options for these difficult-to-treat cancers.
The Phase 1 study is divided into three parts: dose escalation, dose optimization, and dose expansion. The initial dose escalation evaluated four dosages (1.8 mg/kg, 2.7 mg/kg, 3.6 mg/kg, and 4.5 mg/kg every three weeks). CRB-701 targets Nectin-4, a validated tumor-associated antigen in urothelial cancer, delivering a cytotoxic payload via a cleavable linker and a drug-antibody ratio of 2 using MMAE.
The data presented at ASCO GU will be critical in shaping the future development trajectory of CRB-701. Positive results could accelerate the progression into later-phase clinical trials, potentially leading to an accelerated approval pathway. This news highlights the potential of CRB-701 as a viable treatment option, offering hope for patients and marking a potential advancement in the oncology landscape.
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### [Positive INB-100 Phase 1 Leukemia Trial Results Reported by in8bio](https://www.clinicaltrialvanguard.com/news/positive-inb-100-phase-1-leukemia-trial-results-reported-by-in8bio/)
**Published:** February 12, 2025
**Author:** Jon Napitupulu
**Content:**
IN8bio’s Phase 1 trial of [INB-100](https://www.clinicaltrialvanguard.com/news/in8bio-expands-inb-100-phase-1-trial-to-ohio-state-site/), a gamma-delta T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for complex leukemias like AML, has shown promising results. All AML patients in the trial remain in complete remission with a median follow-up of 20.1 months, exceeding one-year progression-free survival and overall survival rates compared to historical control groups. The persistence of gamma-delta T cells beyond one year suggests the potential for durable remissions.
These findings hold significant potential for improving outcomes in high-risk AML patients, particularly those who undergo reduced-intensity conditioning and often face high relapse rates. The therapy’s apparent tolerability, without significant side effects like cytokine release syndrome or neurotoxicity, further enhances its potential to change the treatment landscape. This positive safety profile, coupled with durable remissions, could lead to wider adoption and improved quality of life for patients who often have limited effective options.
The trial data demonstrate a 100% remission rate in AML patients after almost two years post-transplant, significantly higher than the one-year progression-free survival (67.8% and 57.4%) and overall survival (74.7% and 66.7%) reported by CIBMTR and KUCC, respectively. Importantly, these positive results are observed in a patient population with a median age of 68, often with complex, high-risk disease and prior treatment failures, including CAR-T therapies. The absence of severe side effects like cytokine release syndrome and neurotoxicity, combined with manageable graft-versus-host disease, reinforces the therapy’s favorable safety profile.
IN8bio’s positive clinical data for INB-100 suggests a potential shift in the treatment paradigm for high-risk AML. The company’s ongoing efforts to expand the trial network and lay the groundwork for a potential registrational trial indicate a commitment to bringing this promising therapy to a broader patient population. Future updates on the trial’s progress and regulatory pathway will be critical for gauging the therapy’s ultimate potential to improve outcomes for patients with this challenging disease.
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### [Allostem™ Shows Positive One-Year Results for Type 2 Diabetes](https://www.clinicaltrialvanguard.com/news/allostem-shows-positive-one-year-results-for-type-2-diabetes/)
**Published:** February 12, 2025
**Author:** Jon Napitupulu
**Content:**
Creative Medical Technology Holdings, Inc. (CELZ) announced positive one-year follow-up data from a pilot study of [CELZ-201](https://www.clinicaltrialvanguard.com/news/celz-201-ddt-gets-fda-fast-track-for-chronic-lower-back-pain/) (AlloStem™) in late-stage [Type 2 Diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) patients. The off-the-shelf, allogeneic [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) demonstrated an 80% efficacy rate in reducing insulin dependence and stabilizing hemoglobin A1c levels, with no serious adverse effects observed. The study, involving 20 patients, confirmed the safety and efficacy of CELZ-201 using the same infusion procedure as the company’s ongoing FDA-cleared Type 1 Diabetes clinical trial.
This development is a critical advancement in the treatment of late-stage Type 2 Diabetes, a disease affecting millions and often requiring intensive insulin management as it progresses. The positive results suggest CELZ-201 could become a valuable alternative for patients who may not be suitable candidates for autologous therapies or those requiring more effective disease management options. The therapy’s potential to significantly reduce insulin dependence and stabilize blood sugar levels could dramatically improve quality of life for these patients and potentially reduce the long-term complications associated with the disease.
The pilot study enrolled 20 patients, with 10 receiving CELZ-201 and 10 receiving optimized medical therapy. The 80% efficacy rate in the treatment group is a promising indicator of the therapy’s potential. The absence of serious adverse effects reinforces the therapy’s safety profile, a crucial factor for chronic conditions like diabetes. This data strengthens the company’s position in the diabetes treatment landscape, complementing its existing research in Type 1 Diabetes.
This success positions Creative Medical Technology to potentially expand the clinical application of CELZ-201 to a broader patient population, including those with late-stage Type 2 Diabetes. The positive data may attract further investment and accelerate the therapy’s clinical development and potential commercialization. Furthermore, the company’s success with this allogeneic cell therapy platform could have implications beyond diabetes, opening avenues for exploration in other therapeutic areas with significant unmet needs.
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### [Kairos Pharma and Huntsman Cancer Institute for ENV105 Phase 2 Trial](https://www.clinicaltrialvanguard.com/news/kairos-pharma-and-huntsman-cancer-institute-for-env105-phase-2-trial/)
**Published:** February 12, 2025
**Author:** Jon Napitupulu
**Content:**
[Kairos Pharma](https://www.clinicaltrialvanguard.com/news/kairos-pharma-and-city-of-hope-team-up-for-phase-2-trial/) (NYSE American: KAPA) is expanding its Phase 2 clinical trial for ENV105, a drug targeting castrate-resistant [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/), to include Huntsman Cancer Institute. The trial, which is partly funded by the National Cancer Institute, is investigating ENV105 in combination with apalutamide, an existing prostate cancer treatment. This expansion aims to broaden the patient population and identify biomarkers to predict treatment response.
This expansion is crucial for advancing ENV105’s development and understanding its potential in a wider patient group. Including a prestigious institution like Huntsman Cancer Institute adds significant credibility to the study, attracting attention from the medical community and potentially facilitating future collaborations. Identifying predictive biomarkers is essential for personalized medicine, allowing clinicians to select patients most likely to benefit from ENV105 and avoid unnecessary treatment in those less likely to respond.
The Phase 2 trial is evaluating ENV105’s ability to reverse drug resistance in prostate cancer patients by targeting CD105, a protein associated with resistance to standard therapies. The collaboration with multiple clinical sites allows for a more diverse patient sample, improving the generalizability of the findings. The inclusion of Huntsman Cancer Institute further strengthens the study’s scientific rigor.
This expansion signals growing momentum for ENV105 and strengthens Kairos Pharma’s position in developing targeted cancer therapies. The data generated from this expanded trial will be instrumental in guiding future clinical development, informing potential regulatory strategies, and ultimately determining the drug’s potential impact on patients with castrate-resistant prostate cancer. The identification of predictive biomarkers could significantly improve patient outcomes and potentially expand ENV105’s application in other cancer types.
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### [Ati-2138: Potent, Selective Inhibitor of ITK and JAK3](https://www.clinicaltrialvanguard.com/news/ati-2138-potent-selective-inhibitor-of-itk-and-jak3/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
Aclaris Therapeutics announced the publication of research on [ATI-2138](https://www.clinicaltrialvanguard.com/news/aclaris-announces-phase-2a-trial-results-for-ati-2138-at-aad/), a novel dual inhibitor of ITK and JAK3, currently in development for autoimmune and inflammatory diseases. Preclinical and early clinical data suggest ATI-2138 effectively inhibits key T cell activity implicated in conditions like [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/), alopecia areata, and vitiligo. The research underscores the drug’s unique mechanism of action and potential for treating a range of inflammatory conditions.
This development is noteworthy because it validates the dual-inhibition approach targeting both ITK and JAK3. The data suggests that ATI-2138 could offer a more comprehensive treatment strategy compared to existing therapies by addressing multiple pathways involved in immune system dysregulation. This is particularly important for patients with conditions like atopic dermatitis, where current treatment options may not be fully effective or well-tolerated. The research also highlights the potential of ATI-2138 to expand treatment options for other autoimmune and inflammatory diseases with similar underlying mechanisms.
Technical findings demonstrate ATI-2138’s potent and selective inhibition of ITK and JAK3, surpassing the potency of comparable drugs like ritlecitinib in inhibiting TCR-mediated ITK signaling. Furthermore, the drug showed efficacy in preclinical animal models of chronic inflammation. In initial human trials (single and multiple ascending dose studies), ATI-2138 was well-tolerated with no serious adverse events reported. The drug also demonstrated dose- and time-dependent modulation of biomarkers related to ITK and JAK3 activity.
The positive preclinical and early clinical results for ATI-2138 provide a solid foundation for advancing the drug’s development. The data strengthens Aclaris’ position in the field of immunology and suggests that ATI-2138 could become a valuable treatment option for patients with various inflammatory and autoimmune diseases. Further clinical trials are necessary to confirm these findings and explore the full therapeutic potential of ATI-2138 in specific patient populations.
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### [Aptose's Triple Drug AML Therapy Shows Promise in Phase 1/2 Trial](https://www.clinicaltrialvanguard.com/news/aptoses-triple-drug-aml-therapy-shows-promise-in-phase-1-2-trial/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
Aptose Biosciences reported promising early results from its Phase 1/2 TUSCANY trial, testing a triplet therapy of [tuspetinib](https://www.clinicaltrialvanguard.com/news/tuspetinib-triple-therapy-shows-high-aml-response-rate-at-ash/), venetoclax, and azacitidine (TUS+VEN+AZA) in newly diagnosed acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML) patients ineligible for standard induction chemotherapy. Early data from the first cohort receiving the lowest dose of tuspetinib (40mg) shows positive clinical safety and anti-leukemic activity. Of the four patients dosed, three with FLT3-WT completed Cycle 1 with no dose-limiting toxicities or adjustments, and two achieved complete remissions.
These early findings hold significant potential for AML patients, especially those unfit for intensive chemotherapy. The achievement of complete remission in a patient with TP53 mutation, a particularly aggressive form of AML, using a less toxic regimen, is a major advancement. This suggests the triplet therapy could offer a valuable new option for this challenging patient subgroup. Additionally, the promising results in FLT3-WT patients expand the potential reach of this treatment approach to a broader population.
The data reveals that the TUS+VEN+AZA triplet was well-tolerated at the initial dose, with no observed PK interactions affecting tuspetinib levels. This allows for predictable dosing using standard venetoclax and azacitidine regimens, simplifying treatment administration. Two of the three FLT3-WT patients achieved complete remission (CR and CRh) after just one cycle, and a third patient with biallelic TP53 mutations and a complex karyotype also achieved CR. A fourth patient, with FLT3-ITD and NPM1 mutations, is currently in Cycle 1 and awaiting evaluation.
The early TUSCANY trial results suggest that TUS+VEN+AZA could become a crucial frontline therapy for a wider range of AML patients, including those with difficult-to-treat mutations, who cannot tolerate intensive chemotherapy. Continued positive data from the TUSCANY trial could reshape the treatment landscape for newly diagnosed AML, providing a safer and highly effective option for diverse patient populations.
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### [Escharex's Pivotal Trial for Venous Leg Ulcers](https://www.clinicaltrialvanguard.com/news/escharexs-pivotal-trial-for-venous-leg-ulcers/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
MediWound has launched VALUE, a pivotal Phase III trial for EscharEx®, its enzymatic treatment for venous leg ulcers (VLUs). The global, double-blind, placebo-controlled trial will assess EscharEx’s efficacy in debridement and wound closure in 216 patients across the U.S. and Europe, with an interim analysis expected in mid-2026. The company has partnered with Solventum, Mölnlycke, and [MIMEDX](https://www.clinicaltrialvanguard.com/news/mimedx-provides-update-on-epieffect-randomized-trial/) for wound management products and support.
This trial is crucial because it addresses a significant unmet need in VLU treatment. No new FDA-approved VLU drug has entered the market since 1965, leaving a substantial opening for a more effective debridement therapy. EscharEx’s potential to improve debridement and wound closure could significantly impact patient outcomes, reducing pain, infection risk, and disability associated with VLUs. This advancement could also streamline treatment pathways and potentially lower healthcare costs by accelerating healing and minimizing the need for complex interventions.
The VALUE trial employs a 1:1 randomization of EscharEx versus placebo, with treatment involving up to eight daily applications over two weeks. Following treatment, patients will undergo ten weeks of standard wound management. Those achieving wound bed preparation will then receive a cellular/tissue-based product or an autograft, with an additional 12 weeks of monitoring for those who achieve complete closure. The trial’s co-primary endpoints are the incidence of complete debridement and complete wound closure. Beyond VALUE, MediWound plans a head-to-head Phase II trial comparing EscharEx to collagenase in VLU patients, and a Phase II/III trial for diabetic foot ulcers, further solidifying its commitment to advanced wound care solutions.
Positive results from the VALUE trial could position EscharEx as a leading treatment for VLUs, significantly expanding MediWound’s market presence within the estimated $375+ million wound debridement market. This success would validate the company’s enzymatic platform and potentially pave the way for broader applications in other chronic wound types, ultimately reshaping the landscape of wound care.
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### [Updated Phase 2 Agent-797 Gastric Cancer Data at AACR IO](https://www.clinicaltrialvanguard.com/news/updated-phase-2-agent-797-gastric-cancer-data-at-aacr-io/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
MiNK Therapeutics will present interim Phase 2 data on AgenT-797 combined with [botensilimab](https://www.clinicaltrialvanguard.com/news/allo-inkt-combination-data-in-2l-gastric-cancer-at-aacr/) and balstilimab for refractory [gastric cancer](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) at the AACR IO Annual Meeting, February 23-26 in Los Angeles. The presentation, titled “Biomarker analysis from Phase 2 study of AgenT-797 (invariant natural killer T-cells), botensilimab (a Fc-enhanced CTLA-4 Inhibitor) with balstilimab (anti-PD-1) in PD-1 refractory gastroesophageal cancer (GEC),” is scheduled for Tuesday, February 25th. This research focuses on evaluating the efficacy of this combination therapy in patients who have not responded to previous treatments, highlighting MiNK’s commitment to developing novel cancer therapies.
This data release is crucial for several reasons. Gastric cancer remains a significant unmet medical need, with limited effective treatment options for patients who progress after initial therapies. The combination of AgenT-797 with existing immunotherapies like botensilimab and balstilimab represents a potentially promising new approach, leveraging the unique properties of iNKT cells to enhance anti-tumor responses. Positive interim data could validate MiNK’s iNKT platform and generate excitement for its potential application in other difficult-to-treat cancers.
The Phase 2 trial investigates AgenT-797, an allogeneic, off-the-shelf iNKT [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/). It works by harnessing the power of iNKT cells, immune cells with both cytotoxic and adaptive properties, making them potent activators of the immune system. The study combines AgenT-797 with botensilimab, a CTLA-4 inhibitor, and balstilimab, a PD-1 inhibitor, aiming to synergistically enhance the anti-tumor immune response in patients with refractory gastric cancer. The upcoming presentation will focus on biomarker analysis, providing insights into the biological mechanisms underlying the treatment’s effects and potentially identifying predictive biomarkers for patient selection.
Positive interim data from this Phase 2 trial could significantly advance MiNK’s position in the immuno-oncology space. It would provide clinical validation for their iNKT cell therapy platform and could attract further investment and partnerships. Furthermore, it could pave the way for larger clinical trials and potentially accelerate the development of AgenT-797 for this and other cancer indications. The results may also stimulate further research into iNKT cell therapies, opening up new avenues for cancer treatment.
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### [Bio-Path Holdings Announces Promising Phase 1/1b AML Trial Results](https://www.clinicaltrialvanguard.com/news/bio-path-holdings-announces-promising-phase-1-1b-aml-trial-results/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
[Bio-Path Holdings](https://www.clinicaltrialvanguard.com/news/bio-path-holdings-key-clinical-breakthroughs/) announced positive findings from its Phase 1/1b clinical trial of BP1002, a drug designed to treat refractory/relapsed acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML), including cases resistant to venetoclax. One patient in the third cohort demonstrated stable disease and a significant reduction in blast count after a single treatment cycle. The trial has now advanced to the fourth cohort, testing a higher 90 mg/m² dose.
This development is particularly encouraging for AML patients who have relapsed after initial venetoclax-based treatments. These patients currently face a dismal prognosis, with a median overall survival of less than three months and limited effective salvage therapies. BP1002 offers a potential new treatment avenue by targeting Bcl-2 at the mRNA level, which may circumvent venetoclax resistance mechanisms. The accelerated enrollment in the third cohort underscores the urgent need for new therapies in this patient population.
The Phase 1/1b trial is being conducted at prominent U.S. cancer centers and employs a treatment cycle of two doses per week for four weeks. Following completion of the BP1002 monotherapy cohorts, the trial will proceed to a Phase 1b segment evaluating BP1002 in combination with decitabine.
This positive patient response and the progression to a higher dose cohort represent significant milestones in the development of BP1002. It strengthens the potential of BP1002 as a viable treatment option for relapsed/refractory AML, particularly for patients who have developed resistance to venetoclax. Further results from this trial are eagerly anticipated, as they could pave the way for a much-needed new therapy in this challenging disease area.
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**Categories:** News
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### [Neuphoria Therapeutics Gets $15M Milestone Payment from Merck](https://www.clinicaltrialvanguard.com/news/neuphoria-therapeutics-gets-15m-milestone-payment-from-merck/)
**Published:** February 13, 2025
**Author:** Jon Napitupulu
**Content:**
Neuphoria Therapeutics (Nasdaq: NEUP) will receive a $15 million milestone payment from Merck due to the initiation of a Phase 2 clinical trial for MK-1167. MK-1167, an α7 nicotinic acetylcholine receptor positive allosteric modulator, targets the symptoms of Alzheimer’s disease dementia. This marks the second milestone payment in their collaboration.
This Phase 2 trial initiation is a crucial step forward for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease research and treatment. It signifies a validation of the potential of α7 nicotinic acetylcholine receptor targeted therapeutics, a mechanism of action that holds promise for addressing cognitive decline in Alzheimer’s. The initiation of a major pharmaceutical company like Merck sponsoring the Phase 2 trial demonstrates confidence in the approach and adds significant resources to its development, increasing the likelihood of advancing a viable treatment option for this devastating disease. This progress offers hope for patients and their families, as current treatments offer limited efficacy.
This $15 million payment is part of a larger agreement between Neuphoria and Merck, under which Neuphoria could receive up to $450 million in future milestone payments and royalties on net sales. This financial boost reinforces Neuphoria’s financial stability and supports their continued research and development efforts, including their lead drug candidate [BNC210](https://www.clinicaltrialvanguard.com/news/neuphoria-updates-phase-3-trial-for-social-anxiety-drug/) for social anxiety disorder and PTSD.
The initiation of this Phase 2 trial represents a significant step towards potentially providing a new treatment option for Alzheimer’s disease. The progress achieved validates Neuphoria’s platform and strengthens their partnership with Merck. The successful completion of this trial could lead to further advancements in the development of MK-1167 and ultimately benefit patients suffering from Alzheimer’s. This success also reinforces the potential of Neuphoria’s ion channel targeting platform for developing treatments for other neuropsychiatric disorders.
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**Categories:** News
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### [Bio-Path Holdings' Key Clinical Breakthroughs](https://www.clinicaltrialvanguard.com/news/bio-path-holdings-key-clinical-breakthroughs/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
Bio-Path Holdings provided updates on two clinical trials: [BP1001](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/)-A for solid tumors and prexigebersen for acute myeloid [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML). A patient with gynecologic cancer in the BP1001-A trial experienced tumor reduction and stable disease after multiple rounds of treatment. Two elderly AML patients in the prexigebersen trial remain in complete remission after two years.
The positive results from the BP1001-A trial are particularly encouraging given the patient’s advanced stage of cancer and prior unsuccessful treatments. This suggests that BP1001-A could offer a new treatment option for patients with advanced solid tumors who have exhausted other therapies. The extended remission observed in the AML patients highlights the potential of prexigebersen to improve long-term outcomes, especially for elderly patients who often cannot tolerate intensive chemotherapy.
The BP1001-A trial is a Phase 1/1b study, currently evaluating the drug as a monotherapy at increasing dose levels. Future phases will explore BP1001-A in combination with other chemotherapies for specific tumor types. The prexigebersen trial is a Phase 2 study evaluating the drug in combination with decitabine and venetoclax for AML.
The continued positive data from these trials strengthen the potential of Bio-Path’s DNAbilize platform and its pipeline of targeted cancer therapies. The data suggests a possible path forward for patients with limited treatment options and underscores the potential for improved efficacy and tolerability compared to current standards of care. Further research will be crucial to confirm these early findings and explore the full potential of these drug candidates in broader patient populations.
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**Categories:** News
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### [NKGen Biotech Publishes Promising Alzheimer’s Drug Trial Results](https://www.clinicaltrialvanguard.com/news/nkgen-biotech-publishes-promising-alzheimers-drug-trial-results/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
NKGen Biotech announced the publication of Phase 1 clinical trial results for [troculeucel](https://www.clinicaltrialvanguard.com/news/nkgen-reveals-hope-for-neurodegenerative-disease-at-china-forum/), an NK [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/). The trial showed stable or improved outcomes in 90% of evaluable participants with no drug-related adverse events. The research highlights the potential of troculeucel to address neuroinflammation and protein aggregates, key factors in Alzheimer’s disease progression.
This research is a critical step forward in Alzheimer’s treatment. Current approved therapies primarily focus on slowing disease progression, but troculeucel’s potential to stabilize or even improve cognitive function offers a new and potentially more impactful avenue for patients. This approach, targeting neuroinflammation and protein aggregates through NK cell therapy, could represent a significant shift in how Alzheimer’s is managed. The positive safety profile observed in the trial further strengthens the therapy’s potential for broader clinical application.
Eleven participants with mild to severe Alzheimer’s received four doses of troculeucel at three-week intervals across three escalating dose cohorts. Analysis of cerebrospinal fluid biomarkers indicated a dose-dependent decrease in pTau181 and GFAP, suggesting a positive impact on brain protein aggregates and neuroinflammation. A Phase 1/2a trial with a higher dosing regimen is currently underway.
The positive Phase 1 results and ongoing larger trial signal promising advancements in Alzheimer’s treatment. If subsequent trials confirm these findings, troculeucel could become a valuable addition to the limited arsenal of Alzheimer’s therapies, offering hope for improved outcomes and potentially altering the trajectory of this devastating disease.
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**Categories:** News
**Tags:** eClinical Tech News
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### [AskBio Gene Therapy Trial for Heart Failure Begins in Europe](https://www.clinicaltrialvanguard.com/news/askbio-gene-therapy-trial-for-heart-failure-begins-in-europe/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
[AskBio](https://www.clinicaltrialvanguard.com/news/askbio-doses-first-limb-girdle-muscular-dystrophy-patient/), a Bayer subsidiary, has initiated the European arm of its Phase 2 GenePHIT trial for AB-1002, a gene therapy for congestive heart failure (CHF). This follows the commencement of the US arm in 2024 and marks a significant step towards potentially offering this one-time treatment to European patients. The therapy targets non-ischemic cardiomyopathy with NYHA Class III heart failure symptoms.
This advancement is crucial given the high prevalence and mortality rate of cardiovascular disease in Europe, representing a substantial unmet medical need. A successful outcome for this trial could offer a disease-modifying approach, impacting both individual patients and strained healthcare systems grappling with the growing burden of heart failure, especially as the population ages. The potential for a one-time treatment represents a significant shift from current ongoing management strategies, promising improved quality of life and potentially reduced long-term healthcare costs.
The GenePHIT trial, enrolling 90-150 adults with reduced ejection fraction (15-35%), is a double-blind, placebo-controlled study evaluating the safety and efficacy of a single intracoronary infusion of AB-1002. It is the largest study to date for AB-1002 and spans clinical centers across the US and several European countries, including Austria, Germany, Hungary, Netherlands, Spain, and the UK. The first European participant was enrolled in Spain. AB-1002 aims to address intracellular abnormalities contributing to heart failure.
The initiation of this European arm represents substantial progress in the development of AB-1002. Positive results could lead to regulatory approval and ultimately provide a novel therapeutic option for a large patient population severely affected by this debilitating condition. It also strengthens AskBio’s position in the gene therapy field and highlights the potential of this approach to transform the treatment of heart failure.
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**Categories:** News
**Tags:** eClinical Tech News
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### [BioCardia Completes Low-Dose Cohort Enrollment in CardiALLO Trial](https://www.clinicaltrialvanguard.com/news/biocardia-completes-low-dose-cohort-enrollment-in-cardiallo-trial/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
BioCardia completed enrollment and dosing in the low-dose cohort of its CardiALLO™ Phase I/II trial. This trial investigates allogeneic mesenchymal stem cell (MSC) therapy for patients with ischemic heart failure and reduced ejection fraction (HFrEF) who also exhibit elevated markers of heart stress and systemic inflammation. The trial incorporates the Morph DNA steerable guide for enhanced cell delivery.
This trial is particularly important because it addresses a patient population with limited treatment options despite receiving standard care. The focus on patients with specific biomarkers (heart stress and inflammation) suggests a precision medicine approach, potentially leading to better patient outcomes and a more efficient drug development process. Utilizing a steerable guide for cell delivery further refines the procedure, potentially improving efficacy and safety.
The Phase I portion of the trial includes a nine-patient dose escalation cohort, administering 20 million, 100 million, and 200 million cells. A Data Safety Monitoring Board (DSMB) will review safety data after each dose cohort. The “off-the-shelf” nature of the allogeneic MSCs offers logistical advantages over autologous therapies. BioCardia’s manufacturing process for these cells is designed for commercial scalability. Following the Phase I safety evaluation, a randomized, double-blinded, placebo-controlled Phase II cohort will assess efficacy in thirty patients.
The completion of low-dose cohort enrollment marks a significant step in the development of the CardiALLO therapy. Positive safety and efficacy data could pave the way for larger pivotal trials and potential conditional approval in Japan. This progress also strengthens BioCardia’s position in the [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) space, complementing its autologous cell therapy program, [CardiAMP](https://www.clinicaltrialvanguard.com/news/biocardias-cardiamp-shows-179-second-exercise-gain-in-refractory-angina-trial/). Furthermore, the established allogeneic MSC manufacturing platform may open doors for partnerships in other therapeutic areas, such as acute respiratory distress syndrome.
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**Categories:** News
**Tags:** eClinical Tech News
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### [UCB Bimzelx Poised to Disrupt Psoriatic Arthritis Market](https://www.clinicaltrialvanguard.com/news/ucb-bimzelx-poised-to-disrupt-psoriatic-arthritis-market/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
UCB’s Bimzelx (bimekizumab), a dual [IL-17A](https://www.clinicaltrialvanguard.com/news/junshis-psoriasis-drug-js005-succeeds-in-phase-3-trial/)/IL-17F inhibitor for [psoriatic arthritis](https://www.clinicaltrialvanguard.com/news/fda-approves-risankizumab-for-pediatric-psoriasis-and-psoriatic-arthritis/)[arthritis](https://www.clinicaltrialvanguard.com/news/new-study-ids-way-to-prevent-and-treat-lyme-arthritis/) (PsA), is demonstrating strong early adoption among rheumatologists just three months post-launch. A Spherix Global Insights study indicates that Bimzelx’s uptake and positive perception among rheumatologists surpass those of similar drug launches, driven by its dual mechanism of action and perceived efficacy. While early prescription numbers trail competitors like Rinvoq, Bimzelx is notably capturing patients switching from other IL-17 inhibitors like Cosentyx and Taltz, suggesting it offers improved benefits.
This rapid uptake of Bimzelx signifies a potential shift in the PsA treatment landscape. The drug’s dual-action mechanism directly challenges established IL-17A therapies, giving clinicians a potentially more effective treatment option and patients hope for better symptom control. The willingness of rheumatologists to switch patients from existing IL-17 inhibitors to Bimzelx underscores their confidence in its potential clinical advantages. This dynamic competition is likely to spur further innovation and improve patient care within the PsA market.
Spherix’s research shows that half of surveyed rheumatologists have already trialed Bimzelx for PsA within three months of launch. While current patient numbers remain behind competitors, the high trial rate and positive perception among clinicians suggest significant growth potential. Moreover, Bimzelx’s introduction has already boosted the overall perceived value of IL-17 inhibitors, positioning them as the preferred mechanism of action over TNF inhibitors for the first time.
The early success of Bimzelx indicates strong potential for continued growth in the PsA market. While achieving market share dominance may take time, the drug’s unique mechanism of action, positive clinical perception, and robust commercial strategy suggest it is well-positioned to become a key player in the PsA treatment algorithm. Continued monitoring of Bimzelx’s performance will be critical to assess its long-term impact on the PsA treatment landscape.
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**Categories:** News
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### [Allogene Therapeutics Publishes Durable Response Data from Phase 1 Trials of ALLO-501 in R/R LBCL](https://www.clinicaltrialvanguard.com/news/allogene-therapeutics-publishes-durable-response-data-from-phase-1-trials-of-allo-501-in-r-r-lbcl/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
Allogene Therapeutics announced positive results from its Phase 1 ALPHA and ALPHA2 clinical trials of cema-cel, an allogeneic CAR T therapy for relapsed/refractory [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (LBCL). The therapy demonstrated comparable efficacy to approved autologous CD19 CAR T treatments, with a manageable safety profile and rapid treatment initiation. Data from the trials, published in the Journal of Clinical Oncology, represent the largest and longest follow-up dataset for an allogeneic CAR T product in LBCL patients.
These findings are potentially groundbreaking for lymphoma treatment. The demonstrated efficacy and safety of cema-cel, along with its “off-the-shelf” availability, could significantly expand access to CAR T therapy for LBCL patients. The rapid two-day treatment initiation offers a substantial advantage over autologous CAR T therapies, which often involve lengthy manufacturing processes. The positive results in patients with low disease burden suggest potential for earlier intervention and improved outcomes.
The combined ALPHA/ALPHA2 trials enrolled 87 patients with relapsed/refractory non-Hodgkin lymphoma. For the 33 LBCL patients who received the pivotal study regimen, the overall response rate was 67%, with a complete response rate of 58%. The median duration of response for patients achieving complete remission was 23.1 months, with median overall survival not yet reached. Importantly, no graft-versus-host disease, ICANS, or high-grade cytokine release syndrome were observed. In a subset of patients with low disease burden, the complete response rate was remarkably high, reaching 100% in some cases.
These promising results support the ongoing ALPHA3 trial, which is investigating cema-cel as a first-line consolidation therapy for LBCL patients who are in remission but have minimal residual disease (MRD). The ALPHA3 trial aims to intervene early in these high-risk patients, potentially preventing relapse and reshaping the standard of care for LBCL. If successful, cema-cel could become a standard component of frontline LBCL treatment, administered shortly after initial chemoimmunotherapy in MRD-positive patients. This represents a paradigm shift from the current “watch and wait” approach, offering a proactive strategy to eliminate residual disease and improve long-term outcomes.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Pfizer Talzenna & Xtandi Improve Survival in mCRPC](https://www.clinicaltrialvanguard.com/news/pfizer-talzenna-xtandi-improve-survival-in-mcrpc/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
Pfizer announced positive Phase 3 TALAPRO-2 trial results for TALZENNA (talazoparib) combined with XTANDI (enzalutamide) in treating metastatic castration-resistant [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) (mCRPC). The combination therapy showed a statistically significant improvement in overall survival compared to XTANDI plus placebo, regardless of homologous recombination repair (HRR) gene mutation status. The findings were presented at the American Society of Clinical Oncology Genitourinary Cancers Symposium.
This news is particularly important for patients with mCRPC, a disease with a historically poor prognosis. The TALAPRO-2 study demonstrates a potential paradigm shift in mCRPC treatment by suggesting a broader application of the combination therapy beyond patients with HRR gene mutations. The observed overall survival benefit, including a nearly 9-month improvement in the unselected cohort and a 14-month improvement in the HRR-mutated cohort, offers new hope for extending survival in this patient population.
The TALAPRO-2 study included two cohorts: one unselected for HRR mutations and another specifically including patients with HRR mutations. In the unselected cohort, the combination therapy resulted in a 20% risk reduction of death, while the HRR-mutated cohort saw a 38% reduction. These findings were consistent across patients with both BRCA and non-BRCA gene alterations. The safety profile remained consistent with the known profiles of each individual drug, with manageable adverse events. Pfizer has shared this data with regulatory authorities, including the FDA and EMA, for potential label updates for TALZENNA.
These positive results position TALZENNA in combination with XTANDI as a potential new standard of care for mCRPC, significantly impacting treatment strategies for a broader range of patients. The observed survival benefit may prompt a reassessment of current treatment guidelines and encourage earlier adoption of this combination therapy, potentially leading to improved outcomes and quality of life for individuals battling this aggressive form of prostate cancer. Further research and regulatory review will be crucial in solidifying the role of this combination in the evolving mCRPC treatment landscape.
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**Categories:** News
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### [ImmunityBio Anktiva® for BCG-Unresponsive Bladder Cancer: MHRA Accepts Marketing Authorization Application](https://www.clinicaltrialvanguard.com/news/immunitybio-anktiva-for-bcg-unresponsive-bladder-cancer-mhra-accepts-marketing-authorization-application/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
[ImmunityBio](https://www.clinicaltrialvanguard.com/executiveinterviews/immunitybios-visionary-approach-to-cancer-immunotherapy/) announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has accepted its marketing authorization application for ANKTIVA, a treatment for BCG-unresponsive non-muscle invasive [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/) (NMIBC) with carcinoma in situ (CIS). This follows recent acceptance by the European Medicines Agency and FDA approval ten months prior. The MHRA’s validation initiates the formal review process for ANKTIVA’s potential approval in the UK.
This acceptance is crucial for bladder cancer patients in the UK who have not responded to the standard BCG treatment. It offers a new treatment option for a challenging condition and potentially expands access to this novel therapy. The rapid succession of regulatory milestones in the US, EU, and now the UK underscores the significant unmet need and the potential of ANKTIVA to address it.
ANKTIVA (nogapendekin alfa inbakicept-pmln) is a first-in-class [IL-15](https://www.clinicaltrialvanguard.com/news/teva-celiac-drug-gets-fda-fast-track/) agonist that works by activating natural killer (NK) cells, CD8+ T cells, and memory T cells, crucial components of the immune system in fighting cancer. This approach offers a different mechanism of action than BCG, making it a valuable option for patients who haven’t responded to prior treatment. ANKTIVA is administered intravesically and has shown promising results in clinical trials.
This latest development positions ANKTIVA for potential expansion into a significant international market. Positive regulatory decisions by the MHRA and other agencies could significantly broaden patient access to ANKTIVA and strengthen ImmunityBio’s market presence, marking a pivotal step in the treatment landscape for bladder cancer. It also reinforces the company’s commitment to developing and commercializing innovative immunotherapies.
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**Categories:** News
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### [Relativity-098 Trial Update: BMS Provides Insights](https://www.clinicaltrialvanguard.com/news/relativity-098-trial-update-bms-provides-insights/)
**Published:** February 14, 2025
**Author:** Jon Napitupulu
**Content:**
Bristol Myers Squibb announced that the Phase 3 [RELATIVITY](https://www.clinicaltrialvanguard.com/news/bmss-relativity-123-trial-fail-a-devastating-end/)-098 trial evaluating Opdualag (nivolumab and relatlimab-rmbw) for adjuvant treatment of completely resected stage III-IV melanoma did not meet its primary endpoint of recurrence-free survival. The safety profile of Opdualag remained consistent with previous studies. The company acknowledges that LAG-3 inhibition in the adjuvant setting did not demonstrate the same efficacy as in advanced melanoma.
This news impacts the melanoma treatment landscape. While Opdivo remains a standard of care for adjuvant melanoma, the trial results for Opdualag suggest the combination may be less effective in patients with completely resected tumors. This underscores the complexity of the immune response in different stages of melanoma and the need for tailored therapeutic strategies. The findings may influence treatment decisions for patients with resected stage III-IV melanoma, with physicians potentially favoring established therapies like Opdivo or Opdivo Qvantig.
The RELATIVITY-098 trial was a randomized, double-blind study comparing Opdualag to Opdivo monotherapy in patients with completely resected stage III-IV melanoma. The primary endpoint was recurrence-free survival, with secondary endpoints including overall survival, distant metastasis-free survival, and safety. The observed safety profile of Opdualag aligns with known profiles of nivolumab and relatlimab.
Despite this setback, Opdualag remains a standard of care for first-line treatment of unresectable or metastatic melanoma. Bristol Myers Squibb will continue to investigate Opdualag’s potential in other cancers, such as [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/). This suggests a shift in focus toward earlier stages of disease where the combination might be more effective or toward different tumor types. The company will likely continue research to better understand the mechanisms underlying the observed difference in efficacy between the adjuvant and advanced melanoma settings.
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**Categories:** News
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### [Cabometyx® & Opdivo® in Kidney Cancer: 5-Year CheckMate -9ER Results](https://www.clinicaltrialvanguard.com/news/cabometyx-opdivo-in-kidney-cancer-5-year-checkmate-9er-results/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
Final results from the phase 3 [CheckMate](https://www.clinicaltrialvanguard.com/news/opdivo-yervoy-vs-opdivo-checkmate-816-results/)-9ER trial show that cabozantinib (CABOMETYX) combined with nivolumab (Opdivo) continues to demonstrate a survival benefit compared to [sunitinib](https://www.clinicaltrialvanguard.com/news/opdivo-cabometyx-show-4-year-benefits-in-rcc-trial/) in patients with previously untreated [advanced renal cell carcinoma](https://www.clinicaltrialvanguard.com/news/allogenes-allo-316-achieves-31-response-rate-in-advanced-renal-cell-carcinoma/) (RCC) after more than five years of follow-up. The combination therapy improved both progression-free survival (PFS) and overall survival (OS) compared to sunitinib. These findings, including subgroup analyses, were presented at the 2025 American Society of Clinical Oncology Genitourinary Cancers Symposium.
This long-term data reinforces the established role of the cabozantinib-nivolumab combination as a first-line treatment for advanced RCC. The sustained efficacy observed over five years provides clinicians and patients with greater confidence in the long-term benefits of this treatment approach, which is particularly crucial in a disease setting where long-term survival is a primary goal. Demonstrating durable efficacy can influence treatment guidelines and further solidify the combination’s position as a preferred first-line option.
The combination therapy showed a statistically significant improvement in PFS (HR: 0.58) and OS (HR: 0.79) compared to sunitinib. Subgroup analyses by IMDC risk and baseline metastasis sites (liver, bone, and lung) consistently favored the combination across all groups, indicating broad applicability of the treatment. The objective response rate (ORR) was also higher with the combination therapy (55.7%) versus sunitinib (27.4%). Safety and tolerability profiles remained consistent with previous reports, with no new safety signals identified in the long-term follow-up.
This five-year follow-up data strengthens the position of cabozantinib plus nivolumab as a standard of care in first-line advanced RCC. The consistent efficacy across subgroups highlights its potential to benefit a diverse patient population. This data is likely to continue influencing treatment decisions and potentially expand the use of this combination therapy in the future.
Source link: [http://www.businesswire.com/news/home/20250214191927/en/Exelixis-Announces-Final-Five-Year-Follow-up-Results-from-CheckMate–9ER-Trial-Evaluating-CABOMETYX%C2%AE-cabozantinib-in-Combination-with-Opdivo%C2%AE-nivolumab-in-Patients-with-Advanced-Kidney-Cancer-at-ASCO-GU-2025](http://www.businesswire.com/news/home/20250214191927/en/Exelixis-Announces-Final-Five-Year-Follow-up-Results-from-CheckMate--9ER-Trial-Evaluating-CABOMETYX%C2%AE-cabozantinib-in-Combination-with-Opdivo%C2%AE-nivolumab-in-Patients-with-Advanced-Kidney-Cancer-at-ASCO-GU-2025)
**Categories:** News
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### [StemSpine® Procedure Significantly Reduces Opioid Use in Chronic Lower Back Pain Patients](https://www.clinicaltrialvanguard.com/news/stemspine-procedure-significantly-reduces-opioid-use-in-chronic-lower-back-pain-patients/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
Creative Medical Technology Holdings (CELZ) announced positive mid-term results from its StemSpine pilot study using AlloStem cells to treat chronic lower back pain. The study showed significant reductions in opioid use, pain levels, and functional disability three years post-procedure, with a strong safety profile. StemSpine is a patented, non-surgical, ultrasound-guided procedure utilizing AlloStem, an “off-the-shelf” allogeneic [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/).
These findings are particularly important because they address a critical unmet need in chronic lower back pain management. The high success rate in reducing opioid dependency offers a potential solution to the ongoing opioid crisis, while improvements in pain and mobility can significantly enhance patients’ quality of life. This non-surgical approach also presents a less invasive alternative to traditional interventions, potentially reducing recovery times and associated risks.
The pilot study demonstrated that over 90% of patients ceased opioid use for pain management three years after the StemSpine procedure. Pain scores decreased by 80%, and Oswestry Disability Index scores, a measure of functional disability, improved by over 60%. Furthermore, only one patient required reintervention after three years, and no serious adverse events were observed. This data complements the company’s ongoing FDA-cleared Phase 1/2 [ADAPT](https://www.clinicaltrialvanguard.com/news/breakthrough-adapt-trial-enrollment-complete-for-olastrocel/) clinical trial for chronic lower back pain using [CELZ-201](https://www.clinicaltrialvanguard.com/news/celz-201-ddt-gets-fda-fast-track-for-chronic-lower-back-pain/)-DDT, although the pilot study is independent of this trial.
These positive results position StemSpine as a potential game-changer in the treatment of chronic lower back pain. The strong efficacy and safety data could lead to wider adoption of the procedure, offering a viable non-opioid treatment option for millions of patients. This advancement could significantly impact the healthcare landscape by reducing opioid reliance and improving the lives of individuals suffering from this debilitating condition. Further research and larger-scale clinical trials will be crucial to confirm these findings and establish StemSpine’s long-term efficacy and safety.
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**Categories:** News
---
### [Medicus Pharma Announces SKNJC-003 Phase 2 Nodular BCC Study Update](https://www.clinicaltrialvanguard.com/news/medicus-pharma-announces-sknjc-003-phase-2-nodular-bcc-study-update/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
[Medicus Pharma](https://www.clinicaltrialvanguard.com/news/medicus-pharma-positive-interim-analysis-of-sknjc-003-in-bcc-treatment/) Ltd. (NASDAQ: MDCX) announced that its Phase 2 clinical trial ([SKNJCT-003](https://www.clinicaltrialvanguard.com/news/medicus-gorlin-alliance-seek-compassionate-use-for-skinject/)) for D-MNA, a treatment for [basal cell carcinoma](https://www.clinicaltrialvanguard.com/news/verrica-presents-new-vp-315-data-for-basal-cell-carcinoma/) (BCC), has randomized over half of its targeted 60 patients. The company anticipates completing an interim data analysis by the end of Q1 2025 and will submit the findings to the FDA, requesting a Type C meeting in Q2 2025 to discuss product development and potentially fast-track the clinical program. The SKNJCT-003 trial is evaluating two doses of D-MNA against a placebo control in patients with BCC.
This progress is particularly important because it signals potential advancements in non-invasive BCC treatments. Current treatments for BCC, while generally effective, can involve surgery or other invasive procedures. A successful outcome for D-MNA, administered via a dissolvable microneedle patch, could offer a significantly improved patient experience, potentially increasing treatment adherence and early intervention. This could lead to better outcomes and a reduction in healthcare costs associated with surgical procedures and follow-up care.
The Phase 2 trial builds upon the positive results of the Phase 1 safety and tolerability study (SKNJCT-001), which demonstrated that D-MNA was well-tolerated with no serious adverse events. Furthermore, the Phase 1 study indicated potential efficacy, with six participants experiencing complete responses, defined as the disappearance of BCC histologically. The current Phase 2 trial will assess the efficacy of two D-MNA doses (100μg and 200μg), the higher of which was the maximum dose used in the Phase 1 study. The trial’s randomized, double-blind, placebo-controlled design will provide robust data on D-MNA’s efficacy. The upcoming interim analysis and subsequent FDA meeting are critical milestones.
Positive interim data and a constructive Type C meeting with the FDA could significantly accelerate D-MNA’s development timeline. This could lead to a quicker path towards a pivotal Phase 3 trial and potential market approval. Successful development of D-MNA could position Medicus Pharma as a leader in non-invasive skin cancer treatments, opening up a substantial market opportunity and potentially transforming the standard of care for BCC.
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**Categories:** News
---
### [Next-Gen Nectin-4 Targeting ADC Shows Promise in Phase 1 Study](https://www.clinicaltrialvanguard.com/news/next-gen-nectin-4-targeting-adc-shows-promise-in-phase-1-study/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
Corbus Pharmaceuticals announced positive data from its US and UK Phase 1 dose escalation study of CRB-701, a next-generation antibody-drug conjugate targeting Nectin-4. The study mirrored the highest doses of a previous China study and showed comparable safety, tolerability, and pharmacokinetic profiles, including low rates of peripheral neuropathy and skin toxicity. Clinical responses were observed in urothelial and cervical cancers, echoing the China study, and notably, responses were also seen in head and neck squamous cell carcinoma ([HNSCC](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/)), a cancer type not previously studied with CRB-701.
These findings are potentially crucial for advancing the treatment of Nectin-4 expressing cancers. The confirmation of the safety and tolerability profile in a Western population strengthens the drug’s potential for broader application. The observed responses in HNSCC expand the possible indications for CRB-701 beyond urothelial and cervical cancers, suggesting a wider therapeutic scope than initially anticipated. This offers hope for patients with HNSCC, a cancer with significant unmet needs for effective therapies.
The Western study enrolled 38 participants, 26 of whom were evaluable for efficacy. The study used the same four dose cohorts (1.8, 2.7, 3.6, and 4.5 mg/kg) and Q3W regimen as the China study, which enrolled 37 participants with 25 evaluable for efficacy. No dose-limiting toxicities were observed in either study. Importantly, the Western study implemented a proactive ocular toxicity protocol, resulting in a lower incidence of related adverse events. CRB-701 demonstrated a longer half-life and lower free-MMAE exposure compared to [enfortumab](https://www.clinicaltrialvanguard.com/news/padcev-plus-keytruda-shows-long-term-efficacy-in-urothelial-cancer/) vedotin, a currently approved Nectin-4 targeting ADC. Efficacy data showed responses across several tumor types, including a confirmed response in cervical cancer and promising activity in HNSCC, with 4 partial responses observed among 7 evaluable participants. Interestingly, responses were observed even in patients with low Nectin-4 expression levels, consistent with preclinical data.
The dose optimization phase of the Western study is now underway, focusing on the 2.7 mg/kg and 3.6 mg/kg doses in HNSCC, cervical, and urothelial cancers. The positive data from both the China and Western studies, especially the safety profile and efficacy signals in multiple tumor types, support further clinical development of CRB-701. This progress positions CRB-701 as a potential competitor in the Nectin-4 targeting ADC space and may lead to a new treatment option for patients with these difficult-to-treat cancers. The expansion into HNSCC adds another dimension to the drug’s potential and warrants further investigation.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Benitec Biopharma's BB-301 Study at MDA Conference](https://www.clinicaltrialvanguard.com/news/benitec-biopharmas-bb-301-study-at-mda-conference/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
Benitec Biopharma announced interim clinical study updates for the first three subjects in its Phase 1b/2a trial of [BB-301](https://www.clinicaltrialvanguard.com/news/benitec-bb-301-trial-shows-positive-long-term-efficacy/), a gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD). These updates will be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference on March 19th. The presentation will focus on the therapy’s impact on dysphagia, a severe swallowing difficulty associated with OPMD.
This research holds significant promise for OPMD patients who experience dysphagia, a life-threatening symptom. Current treatments are limited, and BB-301 offers a potential solution by addressing the underlying genetic cause of the disease. Positive clinical data demonstrating improved swallowing function would validate Benitec’s “Silence and Replace” ddRNAi platform and pave the way for broader applications of this gene therapy approach in other genetic disorders.
BB-301 utilizes a modified AAV9 viral vector to deliver a bifunctional construct. This construct silences the expression of the mutated PABPN1 gene responsible for OPMD while simultaneously delivering a functional copy of the gene. The interim data from the first three patients treated in the ongoing Phase 1b/2a clinical trial will offer crucial insights into the safety and efficacy of the treatment. Previous interim data for the first two subjects showed durable and clinically meaningful improvements in swallowing function. Further updates for enrolled subjects are anticipated in the fourth quarter of 2025.
The upcoming presentation at the Muscular Dystrophy Association conference represents a crucial step for Benitec and the OPMD community. Positive results would reinforce the potential of BB-301 to significantly improve the quality of life for patients and potentially lead to a disease-modifying therapy. Further data from the ongoing trial will be essential to confirming these early findings and establishing the long-term efficacy and safety profile of BB-301. This data will also be pivotal for future regulatory decisions regarding the therapy’s advancement.
Source link:[ https://www.globenewswire.com/news-release/2025/02/14/3026609/0/en/Benitec-Biopharma-Announces-Acceptance-of-Late-Breaking-Oral-Abstract-for-the-BB-301-Phase-1b-2a-Clinical-Study-at-the-Muscular-Dystrophy-Association-Clinical-and-Scientific-Confer.html](https://www.globenewswire.com/news-release/2025/02/14/3026609/0/en/Benitec-Biopharma-Announces-Acceptance-of-Late-Breaking-Oral-Abstract-for-the-BB-301-Phase-1b-2a-Clinical-Study-at-the-Muscular-Dystrophy-Association-Clinical-and-Scientific-Confer.html)
**Categories:** News
**Tags:** eClinical Tech News
---
### [ORYZON Announces Journal Publication of Final Phase IIa REimagine Results with Vafidemstat](https://www.clinicaltrialvanguard.com/news/oryzon-announces-journal-publication-of-final-phase-iia-reimagine-results-with-vafidemstat/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
Oryzon Genomics announced the publication of final Phase IIa REIMAGINE study results, demonstrating the safety and efficacy of vafidemstat in reducing agitation and aggression in patients with borderline personality disorder (BPD), attention-deficit/hyperactivity disorder (ADHD), and autistic spectrum disorder (ASD). The study provided crucial data for advancing vafidemstat’s clinical development, specifically in BPD, leading to a Phase IIb trial and preparations for a Phase III trial. This research aimed to explore the potential of epigenetics, specifically LSD1 inhibition, in addressing unmet needs in psychiatry and neurodevelopmental disorders.
This development holds significant promise for patients struggling with agitation and aggression associated with these disorders. Current treatment options are often limited and may come with undesirable side effects. Vafidemstat offers a novel mechanism of action targeting LSD1, potentially providing a more effective and tolerable treatment alternative for these challenging symptoms. The positive findings in BPD particularly highlight the drug’s potential to significantly improve patients’ quality of life by addressing a core symptom that often disrupts interpersonal relationships and daily functioning.
The REIMAGINE study, a single-center, open-label basket trial, involved administering 1.2 mg/day of vafidemstat to adult patients for eight weeks. Results showed significant and consistent reductions in agitation and aggression across all three patient groups. Further, vafidemstat demonstrated improvements in non-aggressive symptoms and overall disease indicators. Building on these positive outcomes, Oryzon conducted the Phase IIb PORTICO trial in BPD, which further supported vafidemstat’s efficacy. Following a positive meeting with the FDA, Oryzon is now preparing for the Phase III PORTICO-2 trial. In parallel, vafidemstat is also being evaluated in a Phase IIb trial for negative symptoms of [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/).
The positive results from the REIMAGINE and PORTICO studies coupled with FDA feedback suggest a promising future for vafidemstat. If the Phase III trial confirms its efficacy and safety profile, vafidemstat could become a valuable treatment option for managing agitation and aggression in BPD. Furthermore, its potential application in other CNS disorders like ADHD, ASD, and schizophrenia warrants continued investigation and could significantly broaden its impact on [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) care. This research also underscores the increasing importance of epigenetics in understanding and treating complex psychiatric conditions.
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**Categories:** News
---
### [EU Approves First Self-Amplifying mRNA COVID-19 Vaccine](https://www.clinicaltrialvanguard.com/news/eu-approves-first-self-amplifying-mrna-covid-19-vaccine/)
**Published:** February 17, 2025
**Author:** Jon Napitupulu
**Content:**
CSL and Arcturus Therapeutics announced that the European Commission has approved KOSTAIVE (ARCT-154), a self-amplifying mRNA [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) vaccine, for adults. This marks the first approval of an sa-mRNA COVID-19 vaccine by the European Commission, following its existing use in Japan. The approval hinges on positive clinical trial data demonstrating superior immunogenicity and antibody persistence compared to traditional mRNA vaccines.
This approval is a critical development for public health, particularly as COVID-19 continues to evolve. KOSTAIVE’s enhanced immune response and extended protection duration could offer a more robust defense against emerging variants, potentially reducing the frequency of booster shots and providing more durable protection for vulnerable populations. This could also lead to improved vaccine uptake, as individuals may be more inclined to receive a vaccine offering longer-lasting protection.
Clinical trials of KOSTAIVE showed stronger immune responses and antibody persistence for up to 12 months compared to existing mRNA vaccines. The technology behind KOSTAIVE, self-amplifying mRNA, instructs the body to produce more mRNA and protein, thus amplifying the immune response. The European Commission’s centralized authorization makes KOSTAIVE valid across all EU member states and EEA countries.
This approval paves the way for broader access to a potentially more effective COVID-19 vaccine in Europe. While CSL is currently refining KOSTAIVE’s formulation, the approval represents a significant step forward in combating the ongoing pandemic and underscores the potential of sa-mRNA technology in vaccine development. The success of KOSTAIVE could encourage further research and development into sa-mRNA vaccines for other infectious diseases, potentially revolutionizing the vaccine landscape.
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**Categories:** News
---
### [ICON Discusses How AI is Changing Clinical Trial Recruitment and Diversity](https://www.clinicaltrialvanguard.com/executiveinterviews/icon-discusses-how-ai-is-changing-clinical-trial-recruitment-and-diversity/)
**Published:** February 18, 2025
**Author:** Moe Alsumidaie
**Content:**
In this discussion with Kathleen Mandziuk, Vice President of eClinical Development & Delivery at ICON, we explore the dynamic world of clinical trials. Kathleen offers insights on the persistent challenges of patient recruitment, the revolutionary role of AI and digital tools, and the critical importance of diversity and inclusion. As the industry shifts towards patient-centric trials, Kathleen provides a nuanced perspective on how these elements reshape drug development.
## [](#moe-why-is-patient-recruitment-challenging-in-clinical-trials-and-how-has-it-evolved)**Moe: Why is patient recruitment challenging in clinical trials, and how has it evolved?**
Patient recruitment is complex due to access, knowledge, and potential trust issues within patient communities. Historically, many patients were unaware of clinical trials or lacked access because their healthcare providers may not have been involved in clinical research. Today, the challenge is compounded by the plethora of options available to patients in healthcare and clinical trials. For instance, multiple sponsors may target the same indications in rare diseases, creating competition. Patients now evaluate the burden of participation, such as time and travel, and weigh clinical trial participation against other healthcare options. This evolution means that recruitment is not just about enrolling patients but ensuring they choose the proper trial that aligns with their medical needs and lifestyle.
Kathleen Mandziuk, Vice President of eClinical Development & Delivery at ICON
## [](#moe-ai-and-digital-tools-are-seen-as-game-changers-for-recruitment-how-can-companies-move-from-piloting-to-full-scale-implementation)**Moe: AI and digital tools are seen as game-changers for recruitment. How can companies move from piloting to full-scale implementation?**
At ICON, we’ve embraced AI and machine learning to enhance our processes and evidence-generation methods. A prime example is our Cassandra tool, which uses AI to predict post-marketing requirements by regulatory bodies like the FDA and EMA. This foresight allows sponsors to plan their product lifecycle more effectively, collecting the necessary evidence early in the product lifecycle. The industry has come a long way from the days of paper case report forms (CRFs) to electronic data capture (EDC), and now we’re poised to make significant strides with AI and digital endpoints. The key is to integrate these technologies into the drug development process, moving beyond experimentation to create tangible efficiencies and insights. By doing so, we can streamline operations and improve the overall effectiveness of clinical trials, ultimately benefiting patients and sponsors alike.
## [](#moe-how-can-ai-be-leveraged-to-create-personalized-engagement-strategies-and-reduce-dropout-rates)**Moe: How can AI be leveraged to create personalized engagement strategies and reduce dropout rates?**
AI and machine learning offer powerful tools for understanding treatment patterns and engaging with patient communities. By analyzing real-world data, we can identify a representative sample of the patient population, considering factors like age, comorbidities, and demographics. This understanding helps us design trials that reflect real-world conditions, ensuring safety and efficacy data apply to broader populations. Additionally, AI can pinpoint where patients receive healthcare, allowing us to engage those providers in patient education and recruitment. This targeted approach and broader patient engagement strategies like social media and community outreach can significantly enhance participation and reduce dropout rates. By personalizing engagement, we can create a more patient-friendly trial experience.
## [](#moe-how-can-organizations-ensure-digital-innovation-aligns-with-patient-needs-not-just-operational-efficiencies)**Moe: How can organizations ensure digital innovation aligns with patient needs, not just operational efficiencies?**
One innovative approach we’ve implemented at ICON is clinical trial tokenization, which reduces patient burden, especially in long-term follow-up scenarios common in complex cellular and genetic treatment trials. Through tokenization, we can maintain passive and secure visibility of clinical trial patients over extended periods by minimizing the need for patient visits. Additionally, as trials use increasingly digital endpoint collection methods, digital risk detection allows us to identify and address potential issues in real-time, keeping patients engaged and ensuring data integrity. Solutions such as telehealth visits, patient concierge, digital stipend processing, and home health visits can streamline operations and enhance the patient experience, making participating and remaining in trials easier. By focusing on patient needs, we can ensure that digital innovations truly benefit those involved in clinical research.
## [](#moe-what-is-icons-stance-on-dei-and-how-can-ai-improve-representation-in-clinical-trials)**Moe: What is ICON’s stance on DEI, and how can AI improve representation in clinical trials?**
Diversity, equity & inclusion (DEI) is a cornerstone of our trial design and execution. Study populations must mirror real-world patient demographics to ensure our data is representative. For example, if a trial only includes the youngest and healthiest patients, we risk missing safety signals that might appear in a more diverse real-world population. By leveraging AI to understand the epidemiology of the disease and the demographics of affected populations, we can tailor our recruitment and engagement strategies to ensure inclusivity. This approach enhances the study findings’ validity and ensures that all patient groups benefit from new treatments. We can improve clinical trials’ quality and impact by prioritizing DEI.
## [](#moe-how-are-sponsors-balancing-digital-innovation-with-regulatory-compliance-particularly-in-data-integrity-and-patient-safety)**Moe: How are sponsors balancing digital innovation with regulatory compliance, particularly in data integrity and patient safety?**
Regulatory bodies are increasingly recognizing the value of digital endpoints, especially for long-term follow-up in trials for cellular and genetic treatments. Traditional methods of data collection are often impractical for the extended timelines required. By incorporating digital tools like Bluetooth devices, we can gather real-time data on patient health, ensuring compliance with regulatory expectations while maintaining patient safety. These innovations are about meeting requirements and enhancing the quality and reliability of the data we collect. By balancing innovation with compliance, we can ensure that clinical trials remain cutting-edge and safe for participants.
## [](#moe-how-can-the-industry-create-a-unified-patient-friendly-digital-clinical-trial-ecosystem)**Moe: How can the industry create a unified, patient-friendly digital clinical trial ecosystem?**
The need for cohesive solutions is paramount as the clinical trial industry becomes more digitized. Rather than piecing together disparate technologies, we’re seeing a shift towards integrated, end-to-end solutions with configurable modules. This approach reduces the risk of data silos and ensures seamless communication between systems. Patient-facing technologies also need to be supported by an operational framework of education and engagement, such as telehealth and concierge services. Sponsors, CROs, and regulators all have a role in fostering this ecosystem by supporting the development of standardized, interoperable technologies and engagement that prioritize patient experience and data integrity. Working together can create a more unified and efficient clinical trial landscape that benefits all stakeholders. This collaboration is key to advancing the industry and improving clinical trial implementation.
## [](#moe-is-there-anything-else-youd-like-to-add-about-the-future-of-clinical-trials)**Moe: Is there anything else you’d like to add about the future of clinical trials?**
Integrating clinical research into regular healthcare delivery is crucial, mirroring patients’ expectations post-[COVID](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/). By making trials more accessible and engaging, we can continue developing essential drugs for real-world patients. Keeping an open dialogue and adapting to these changes is key to advancing our industry. By aligning clinical trials with everyday healthcare practices, we can ensure that patients receive the best possible care and that new treatments are developed efficiently and effectively. This integration is essential for the future of clinical research and patient care.
**Categories:** Article: Executive Interviews
---
### [Dupixent® FDA Priority Review for Bullous Pemphigoid Treatment](https://www.clinicaltrialvanguard.com/news/dupixent-fda-priority-review-for-bullous-pemphigoid-treatment/)
**Published:** February 18, 2025
**Author:** Jon Napitupulu
**Content:**
Regeneron and Sanofi announced that the FDA accepted for Priority Review their supplemental [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) for Dupixent ([dupilumab](https://www.clinicaltrialvanguard.com/news/zumilokibart-hits-easi-75-target-in-phase-2-atopic-dermatitis-trial/)) to treat bullous pemphigoid (BP) in adults. The application is supported by positive pivotal trial data showing significantly improved sustained disease remission rates with Dupixent compared to placebo. Specifically, five times more Dupixent patients achieved sustained remission—defined as complete remission off oral corticosteroids for at least 20 weeks without relapse or rescue therapy during the 36-week treatment period.
This development is potentially transformative for BP patients, who currently rely heavily on systemic corticosteroids with their associated side effects. If approved, Dupixent offers the possibility of sustained remission and reduced reliance on these corticosteroids, improving quality of life for this patient population. This advancement also demonstrates the potential of targeting type 2 inflammation in addressing a range of dermatological diseases, opening avenues for future research and development.
The pivotal trial enrolled 106 adults with moderate-to-severe BP. Beyond the primary endpoint of sustained remission, Dupixent also demonstrated significant improvements in disease severity and itch reduction. Observed adverse events with Dupixent (in at least 3 patients) compared to placebo included peripheral edema, arthralgia, back pain, blurred vision, hypertension, [asthma](https://www.clinicaltrialvanguard.com/news/connect-biopharma-completes-enrollment-in-rademikibart-asthma-study/), conjunctivitis, constipation, upper respiratory tract infection, limb injury, and insomnia. The FDA is expected to make a decision by June 20, 2025, and if approved, Dupixent would be the first targeted therapy for BP in the U.S.
A potential approval for Dupixent in BP could significantly expand the drug’s market reach and solidify its position as a key player in treating type 2 inflammatory diseases. This positive news also strengthens the collaboration between Regeneron and Sanofi and validates their focus on developing innovative treatments for unmet medical needs. It may also stimulate further research into the role of type 2 inflammation in other related skin diseases.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Lacutamab Gets FDA Breakthrough Therapy for Sézary Syndrome](https://www.clinicaltrialvanguard.com/news/lacutamab-gets-fda-breakthrough-therapy-for-sezary-syndrome/)
**Published:** February 18, 2025
**Author:** Jon Napitupulu
**Content:**
Innate Pharma received Breakthrough Therapy Designation from the FDA for lacutamab, its anti-KIR3DL2 antibody, for treating relapsed or refractory Sézary Syndrome (SS) in adults. This designation is based on promising Phase 1 and 2 trial results showing efficacy and a favorable safety profile in patients previously treated with [mogamulizumab](https://www.clinicaltrialvanguard.com/news/kyowa-kirin-presents-new-mogamulizumab-research-at-ash-2025/). This rare and aggressive form of cutaneous [T-cell lymphoma](https://www.clinicaltrialvanguard.com/news/soquelitinib-data-in-t-cell-lymphoma-promising-results-unveiled/) often leaves patients with a poor quality of life, highlighting the need for new treatment options.
This FDA designation significantly accelerates lacutamab’s development pathway, potentially bringing a much-needed therapy to patients sooner. The positive clinical data, particularly in patients who have exhausted other treatment options including mogamulizumab, suggests lacutamab could become a crucial part of SS treatment. This also represents a critical step forward in addressing the unmet needs of this patient population who often face limited effective therapies.
Lacutamab’s Breakthrough Therapy Designation builds on prior Fast Track designation from the FDA in 2019 and [PRIME](https://www.clinicaltrialvanguard.com/news/tobevibart-elebsiran-get-breakthrough-prime-for-chronic-hep-d/) designation from the European Medicines Agency in 2020. Innate Pharma is currently working with regulatory agencies on a confirmatory Phase 3 trial and actively seeking a partner for further development.
This breakthrough designation underscores lacutamab’s potential to reshape the SS treatment landscape. The accelerated development and review process could lead to faster market entry, offering hope to patients suffering from this aggressive disease and potentially establishing lacutamab as a key therapy in this area. Further development and the planned Phase 3 trial will be pivotal in confirming these early positive results and solidifying lacutamab’s role in addressing the unmet medical needs in SS.
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**Categories:** News
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### [Revolutionary New Prostate Cancer Super Test Developed in UK](https://www.clinicaltrialvanguard.com/news/revolutionary-new-prostate-cancer-super-test-developed-in-uk/)
**Published:** February 18, 2025
**Author:** Jon Napitupulu
**Content:**
Scientists in Cambridge developed a new [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) test designed to revolutionize screening, diagnosis, and personalized treatment. The test analyzes blood and urine samples for over 100 clinically-validated prostate-related biomarkers, using an AI-driven algorithm to identify cancer presence, stage, aggressiveness, and genetic risks. This “super test” aims to address the high incidence of prostate cancer (55,000 new cases annually in the UK, over 330,000 in the EU) and improve upon the limitations of current testing methods.
This development is crucial because current prostate cancer testing, like PSA tests and biopsies, can have accuracy rates below 50%. The new test boasts a projected accuracy between 96-99% across diverse age ranges and ethnicities, significantly improving early detection, particularly for high-risk non-caucasian groups. More accurate diagnoses could also reduce the need for unnecessary MRI scans and invasive digital rectal examinations, improving patient experience and healthcare resource allocation.
The test uses a “multi-omics” approach, combining multiple biomarker signatures (proteomic, transcriptomic, genetic/hereditary, and epigenetic) with phenotypic and symptom data analyzed by a proprietary AI algorithm. Each biomarker has been clinically validated in previous trials involving over 31,000 positive prostate cancer samples and 100,000 control samples. EDX Medical Group, the developer, has filed a patent application for the test and algorithm.
This new test could transform prostate cancer management. Its potential for highly accurate early detection and detailed characterization of the disease could lead to more effective and personalized treatment strategies, ultimately improving patient outcomes and survival rates. Further clinical validation and regulatory approvals are being pursued with a target launch date of late 2025 or early 2026.
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**Categories:** News
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### [Innocare Announces BCL2 Inhibitor & Orelabrutinib for CLL/SLL](https://www.clinicaltrialvanguard.com/news/innocare-announces-bcl2-inhibitor-orelabrutinib-for-cll-sll/)
**Published:** February 18, 2025
**Author:** Jon Napitupulu
**Content:**
[InnoCare](https://www.clinicaltrialvanguard.com/news/innocare-doses-first-patient-in-urticaria-trial-in-china/) Pharma received approval from China’s CDE to begin a Phase III trial of its BCL2 inhibitor, [ICP-248](https://www.clinicaltrialvanguard.com/news/innocare-doses-first-patient-in-mesutoclax-bcl2-inhibitor-trial-for-mcl-in-china/) (Mesutoclax), combined with the BTK inhibitor [orelabrutinib](https://www.clinicaltrialvanguard.com/news/first-patient-dosed-in-orelabrutinib-phase-iii-sle-trial/). This trial will evaluate the combination as a first-line treatment for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) patients in China. The company believes this fixed-duration combination therapy could offer a deeper remission and potentially a clinical cure for patients without drug-resistant mutations.
This trial approval is crucial due to the rising incidence of CLL/SLL in China and the potential for this combination therapy to address an unmet need. Orelabrutinib is already approved and reimbursed in China for other lymphoma types, suggesting a potential pathway for rapid market access if the Phase III trial is successful. A successful trial outcome could shift the treatment paradigm for CLL/SLL towards a potentially curative approach rather than managing the disease chronically.
ICP-248 works by selectively inhibiting BCL2, a protein that regulates apoptosis (programmed cell death), and restoring normal apoptosis in cancer cells. Previous Phase II trial data for the combination of ICP-248 and orelabrutinib demonstrated promising efficacy and a favorable safety profile in CLL/SLL patients. InnoCare is also developing ICP-248 for other blood cancers, indicating a broader strategic focus on hematological oncology.
A positive outcome from this Phase III trial could solidify InnoCare’s position in the Chinese oncology market and establish the ICP-248 and orelabrutinib combination as a leading treatment for CLL/SLL. Further, the success could pave the way for global development of this combination therapy, potentially impacting CLL/SLL treatment worldwide. This trial also validates InnoCare’s strategy of developing synergistic combinations within its oncology pipeline.
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**Categories:** News
---
### [DeepSon Bio at Korean Society of Brain Neuromodulation Therapy](https://www.clinicaltrialvanguard.com/news/deepson-bio-at-korean-society-of-brain-neuromodulation-therapy/)
**Published:** February 19, 2025
**Author:** Jon Napitupulu
**Content:**
Professor Kim Jae-ho presented positive findings from an exploratory clinical trial using Deepson Bio’s NEUCLARE therapeutic ultrasound device to treat normal pressure hydrocephalus (NPH). The trial demonstrated statistically significant improvements in the mobility of NPH patients after undergoing ultrasound therapy. This research explores a novel, non-invasive approach to treating NPH, a condition currently reliant on invasive shunt surgery.
This research holds substantial promise for patients suffering from NPH, a form of dementia. The positive results from this small trial suggest a potential alternative to the risks associated with shunt surgery. A non-invasive treatment option could significantly improve patient outcomes and quality of life by offering a safer and potentially more effective treatment pathway. Furthermore, the exploration of ultrasound’s impact on cerebrospinal fluid circulation opens new avenues for research into other neurological conditions, including [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease.
In a trial of 10 NPH patients, participants underwent three ultrasound stimulation sessions. Following the treatment, mobility was assessed using the Timed Up and Go (TUG) and 10-meter gait tests. Results showed a 23.3% average improvement in TUG test times (7.1 seconds) and a 21.0% average improvement in 10-meter walk times (5.9 seconds), both considered clinically significant. The study focused on the device’s ability to enhance cerebrospinal fluid flow, which is believed to aid in clearing brain waste, a potential mechanism for treating Alzheimer’s disease.
This early success indicates a promising future for non-invasive ultrasound treatment of NPH. Further research and larger-scale trials are crucial to validating these initial findings and exploring the full potential of this technology. The positive impact on mobility in NPH patients suggests a possible paradigm shift in treatment strategies, offering hope for a less invasive and more effective approach to managing this debilitating condition. The connection to brain waste clearance also opens exciting new possibilities for tackling neurodegenerative diseases like Alzheimer’s.
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**Categories:** News
---
### [Vaxxas HD-MAP Vaccine Delivery Clinical Trial Sets Record](https://www.clinicaltrialvanguard.com/news/vaxxas-hd-map-vaccine-delivery-clinical-trial-sets-record/)
**Published:** February 19, 2025
**Author:** Jon Napitupulu
**Content:**
Vaxxas, a clinical-stage biotech company, has completed enrollment for its Phase I clinical trial of an avian influenza A (H7N9) vaccine delivered via its high-density microarray patch (HD-MAP). This 258-participant trial, the largest Phase I study conducted by Vaxxas to date, compares the HD-MAP’s efficacy against traditional needle-and-syringe delivery. The study is partially funded by a US$28.5 million [BARDA](https://www.clinicaltrialvanguard.com/news/care-access-enters-into-new-partnership-with-barda-to-sharpen-pandemic-preparedness/) contract, with approximately 85% of the funds utilized for enrollment completion.
This trial holds significant implications for pandemic preparedness. The HD-MAP’s potential for self-administration, simplified logistics due to reduced cold-chain requirements, and increased patient comfort could dramatically improve vaccination rates and speed during a future pandemic. Evaluating both adjuvanted and non-adjuvanted vaccine formulations delivered via the HD-MAP expands the platform’s potential applications to a broader spectrum of infectious diseases. This trial addresses a critical need for efficient and accessible vaccination strategies, crucial for mitigating widespread public health crises.
This trial marks the first time Vaxxas is evaluating an adjuvanted vaccine delivered with its HD-MAP. The study compares the safety and immune response of adjuvanted and non-adjuvanted formulations delivered by HD-MAP to traditional needle delivery. Prior Vaxxas clinical trials demonstrated comparable immune responses using significantly less vaccine (one-sixth) without adjuvants compared to traditional methods. If the adjuvanted HD-MAP formulation is safe and effective, it could facilitate a quicker response to future outbreaks.
The results of this trial, anticipated in the first half of 2025, could revolutionize vaccine delivery. Positive outcomes could lead to wider adoption of the HD-MAP technology, not only for pandemic influenza but also for a range of infectious diseases. This shift could significantly improve vaccination accessibility and potentially transform public health responses to future outbreaks.
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**Categories:** News
---
### [Miist Therapeutics: Pioneering Inhaled Therapies for Rapid Smoking Cessation and Migraine Relief](https://www.clinicaltrialvanguard.com/executiveinterviews/miist-therapeutics-pioneering-inhaled-therapies-for-rapid-smoking-cessation-and-migraine-relief/)
**Published:** February 19, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, we engage with Dalton Signor, CEO at [Miist Therapeutics](https://www.miisttherapeutics.com/), to explore their innovative inhaled therapies for migraines and smoking cessation. With a focus on rapid drug delivery and enhanced patient outcomes, Miist Therapeutics is set to redefine treatment standards. This discussion unveils the unique methodologies and promising results that distinguish their therapies from traditional options.
## [](#moe-how-are-miists-trials-designed-to-measure-the-safety-and-efficacy-of-your-inhaled-therapies)Moe: How are Miist’s trials designed to measure the safety and efficacy of your inhaled therapies?
Our clinical trials are meticulously crafted to focus on safety, pharmacokinetics (PK), and early efficacy. A standout feature is our use of arterial sampling, which is somewhat unique in the industry. This method allows us to accurately capture the rapid absorption of our inhaled therapies, which is crucial given our delivery system. For example, in our phase one study, we conducted arterial draws at intervals as short as 30 seconds, yet we found that the peak absorption occurred even before this mark. This rapid absorption is pivotal for achieving quick symptom relief, as demonstrated by our 92% symptom relief within two minutes for smoking
Dalton Signor, CEO at Miist Therapeutics
cessation. This approach provides a comprehensive understanding of the drug’s behavior in the body and sets a new standard for evaluating inhaled therapies.
## [](#moe-how-do-miists-inhaled-therapies-compare-to-current-treatments-for-migraines-and-smoking-cessation)Moe: How do Miist’s inhaled therapies compare to current treatments for migraines and smoking cessation?
Our therapies offer a significant leap forward in both speed and efficacy. For smoking cessation, existing products like gums and lozenges are marketed as quick-acting but take 30 to 50 minutes to provide relief. This delay is problematic because the average person relapses within 11 minutes of experiencing cravings. Our inhaled therapy, however, reaches peak concentration within 30 seconds and provides 92% symptom relief within two minutes. This rapid response is crucial for preventing relapse. Similarly, for migraines, most treatments are oral and can take up to two hours to reach peak concentration due to delayed gastric emptying. Our inhaled therapy delivers the drug to the brain almost instantaneously, allowing us to address the [migraine](https://www.clinicaltrialvanguard.com/news/lundbecks-bocunebart-meets-primary-endpoint-in-phase-iib-migraine-trial/) before it fully develops. This improves patient outcomes and highlights the potential of inhaled therapies to revolutionize treatment paradigms.
## [](#moe-can-you-elaborate-on-the-adverse-reactions-or-potential-side-effects-observed-during-your-studies)Moe: Can you elaborate on the adverse reactions or potential side effects observed during your studies?
The safety profile of our inhaled therapies is quite favorable. For smoking cessation, the adverse events were minimal and expected, such as coughing and throat irritation, which are typical of inhaled nicotine. Importantly, we observed no serious adverse events. One key to our strong safety profile is our aerosol generation method, which uses vibration, not heat. Since we don’t use any heat in our aerosolization process, we avoid the creation of any undesirable or dangerous byproducts. Patients who use our aerosol therapies are only inhaling small water droplets that contain our active pharmaceutical ingredient. In addition, the large surface area of the peripheral lung helps dilute the therapy, enhancing tolerability. This is a significant advantage over traditional methods, which often have more pronounced side effects. Our approach leverages the body’s natural physiology to improve drug delivery and minimize adverse reactions, setting a new benchmark for safety in inhaled therapies. By focusing on the natural absorption processes, we ensure that our treatments are effective and safe for long-term use, providing a comprehensive solution for patients.
## [](#moe-what-are-the-long-term-outcomes-for-patients-using-your-inhaled-therapies-for-smoking-cessation)Moe: What are the long-term outcomes for patients using your inhaled therapies for smoking cessation?
While we can’t claim long-term outcomes until our phase three trials are complete, current therapies show only 7% to 10% success rates at six months. Our approach involves a gradual dose reduction, aligning with the natural downregulation of nicotinic receptors in the brain. This method aims to reduce the need for nicotine gradually, potentially leading to better long-term abstinence rates. By synchronizing the reduction of nicotine with the brain’s adaptation, we hope to provide a more sustainable path to quitting smoking, addressing a critical gap in current treatment options. Our goal is not only to help patients quit smoking but also to ensure they remain smoke-free, improving their overall health and quality of life.
## [](#moe-how-does-miist-address-the-regulatory-challenges-of-introducing-a-novel-inhalation-device-therapy)Moe: How does Miist address the regulatory challenges of introducing a novel inhalation device therapy?
Regulatory challenges are manageable as the FDA is familiar with inhalation devices. The key is standardizing inhalation to ensure consistent drug delivery. Our software-controlled inhaler automates the process, ensuring patients inhale correctly every time. This automation minimizes variability and enhances the reliability of our clinical trial data, addressing a standard failure mode in inhaled therapies. By leveraging technology to standardize drug delivery, we meet regulatory requirements and improve patient outcomes, demonstrating our commitment to innovation and excellence. Our approach ensures that our therapies are effective and compliant with regulatory standards, paving the way for broader adoption.
## [](#moe-what-endpoints-are-you-focusing-on-in-your-phase-three-trials)Moe: What endpoints are you focusing on in your phase three trials?
Our primary endpoint for phase three smoking cessation study is continuous abstinence during the last four weeks of the study, with secondary endpoints including seven-day point prevalence abstinence throughout the trial. This standard has been used for all nicotine replacement therapies approved in recent decades. By adhering to these established benchmarks, we aim to demonstrate the superior efficacy of our treatments, paving the way for regulatory approval and broader adoption. Our focus on these endpoints ensures that we are meeting regulatory standards and providing meaningful outcomes for patients, improving their chances of long-term success.
## [](#moe-is-there-anything-else-youd-like-to-add-about-miists-approach-or-future-plans)Moe: Is there anything else you’d like to add about Miist’s approach or future plans?
Beyond smoking cessation and migraines, our technology is adaptable to other conditions requiring rapid drug delivery to the brain, such as pain and insomnia. The core technology remains the same, allowing us to conduct phase one studies across various therapeutic areas efficiently. This scalability is a significant advantage, enabling us to expand our impact across multiple conditions. By leveraging our platform technology, we are well-positioned to address a critical patient need, instant symptom relief, for many of today’s most common and debilitating conditions.
**Categories:** Article: Executive Interviews
---
### [Sellas Announces Positive Data from Phase 2a Trial of SLS009](https://www.clinicaltrialvanguard.com/news/sellas-announces-positive-data-from-phase-2a-trial-of-sls009/)
**Published:** February 21, 2025
**Author:** Jon Napitupulu
**Content:**
SELLAS [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) announced positive Phase 2a trial data for SLS009 (tambiciclib), a CDK9 inhibitor, combined with Brukinsa (zanubrutinib) in relapsed/refractory diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/) (r/r DLBCL). The trial, conducted by GenFleet Therapeutics in China, showed a 67% overall response rate, significantly exceeding the anticipated response rate of zanubrutinib alone. This combination therapy also demonstrated a median overall survival that was not yet reached after a median follow-up of 4.6 months, with 67% of patients still alive.
This positive data is particularly encouraging for patients with non-GCB (ABC) DLBCL, a subtype associated with a poorer prognosis. The combination therapy demonstrated an 83% disease control rate in these patients, suggesting a potential new treatment avenue for this difficult-to-treat population. Moreover, the observed efficacy in patients without MYD88 or CD79B mutations, typically predictive of a better response to BTK inhibitors, suggests broader applicability of the SLS009/zanubrutinib combination. The complete response observed in a patient with both MYC amplification and TP53 mutation further highlights the potential of SLS009 to overcome drug resistance mechanisms in cancer.
Nine r/r DLBCL patients participated in the trial, six with the non-GCB subtype. One patient achieved complete response, and three achieved partial response with significant target lesion shrinkage. Grade 3 or higher adverse events were reported in 55.6% of patients, consistent with the expected safety profile of zanubrutinib monotherapy. While GenFleet will determine next steps for the lymphoma trial, SELLAS remains focused on acute myeloid leukemia and spliceosome-chromatin mutation related cancers.
These findings represent a potential advancement in DLBCL treatment, especially for the non-GCB subtype. The SLS009 and zanubrutinib combination may offer a more effective therapeutic approach, warranting further investigation. Although SELLAS is not directly pursuing the next steps in lymphoma, these results demonstrate the potential of SLS009 in a broader range of cancers and may attract partners interested in advancing the lymphoma program. This data could also inform future research into combination therapies incorporating CDK9 inhibition.
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**Categories:** News
---
### [Neurona's Epic Nrtx-1001 Cell Therapy Epilepsy Study](https://www.clinicaltrialvanguard.com/news/neuronas-epic-nrtx-1001-cell-therapy-epilepsy-study/)
**Published:** February 21, 2025
**Author:** Jon Napitupulu
**Content:**
Neurona Therapeutics, a clinical-stage [biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) company, is initiating its Phase 3 EPIC clinical trial for NRTX-1001, a regenerative [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for drug-resistant mesial temporal lobe epilepsy (MTLE) in adults. This marks the first investigational human cell therapy to reach Phase 3 for this indication and is based on the regulatory pathway established through the Regenerative Medicine Advanced Therapy (RMAT) designation. The Phase 3 EPIC trial, planned for the second half of 2025, will be a randomized, sham-controlled, double-blind study.
This trial represents a significant advancement in the treatment of drug-resistant epilepsy. Current options for drug-resistant MTLE, such as surgical removal or ablation of the affected brain region, carry significant risks of irreversible neurocognitive impairment. NRTX-1001 offers a potential alternative that aims to restore balanced brain activity with a single administration, possibly avoiding the invasive and potentially damaging effects of surgery. The success of this therapy could reshape the treatment landscape for this prevalent form of epilepsy, providing a less invasive and potentially more effective solution for patients.
The Phase 3 trial builds upon promising data from Neurona’s ongoing Phase 1/2 trials. Preliminary results from the open-label Phase 1/2 study showed substantial median seizure reduction and no reported cognitive impairments. Some patients even experienced notable improvements in neurocognitive test scores. The Phase 3 trial, with its rigorous design, will provide more robust data on the efficacy and safety of NRTX-1001, ultimately aiming to support a future [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/).
The commencement of the Phase 3 EPIC trial marks a critical step towards potentially delivering a novel therapeutic option to patients with drug-resistant MTLE. Positive results from this trial could lead to the first disease-modifying therapy for this condition, offering new hope for patients and potentially revolutionizing the field of epilepsy treatment. The outcome of the trial is expected to have a substantial impact on Neurona’s future, shaping the company’s position as a leader in regenerative cell therapies for neurological disorders.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Phoenix Molecular Designs Doses First Patient with PMD-026 in Dauntless-1 Trial](https://www.clinicaltrialvanguard.com/news/phoenix-molecular-designs-doses-first-patient-with-pmd-026-in-dauntless-1-trial/)
**Published:** February 21, 2025
**Author:** Jon Napitupulu
**Content:**
Phoenix Molecular Designs (PhoenixMD) has initiated Phase 2 clinical trials (Dauntless-1) for PMD-026, a first-in-class pan-RSK inhibitor, in combination with fulvestrant. The trial targets patients with locally advanced or metastatic HR+/HER2-/RSK2-high [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) who have progressed after CDK4/6 inhibitor treatment. This approach aims to address CDK4/6 inhibitor resistance, a significant challenge in treating this type of breast cancer.
This trial is a critical step toward improving outcomes for patients with advanced breast cancer who have limited effective treatment options after developing CDK4/6 inhibitor resistance. Fulvestrant, a standard second-line therapy, often provides only short-term benefit. Preclinical data suggests that combining PMD-026 with fulvestrant could significantly extend the duration of response, offering a new therapeutic strategy for this patient population. Focusing on RSK2-high patients allows for a targeted approach, potentially increasing the likelihood of success.
The Phase 2 trial builds upon promising Phase 1/1b monotherapy results where PMD-026 demonstrated a median progression-free survival of 4.8 months in RSK2-high patients compared to 1.3 months in RSK2-low patients who had received limited prior therapies. Importantly, PMD-026 was well-tolerated without common side effects like peripheral neuropathy, hair loss, or hyperglycemia. These factors, along with the preclinical synergy with fulvestrant, support the rationale for the combination therapy approach.
The initiation of the Dauntless-1 trial represents a significant advancement in the development of PMD-026. Positive Phase 2 results could lead to a new treatment option for patients with advanced breast cancer who have developed resistance to CDK4/6 inhibitors, ultimately changing the treatment landscape for this challenging disease. This could also establish PMD-026 as a key player in the precision oncology field and potentially pave the way for its use in other cancer types.
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**Categories:** News
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### [EG 427 Gets €27M in Series B Funding for Clinical Study](https://www.clinicaltrialvanguard.com/news/eg-427-gets-e27m-in-series-b-funding-for-clinical-study/)
**Published:** February 21, 2025
**Author:** Jon Napitupulu
**Content:**
EG 427, a biotech company developing targeted genetic medicines for neurological conditions, secured €27 million in Series B funding. The round was co-led by Andera Partners and Bpifrance, with participation from SCI Ventures and existing investors. The funding will primarily support the Phase 1b/2a clinical trial of [EG110A](https://www.clinicaltrialvanguard.com/news/milestone-reached-first-patient-treated-with-eg110a-for-neurogenic-bladder/), a treatment for neurogenic detrusor overactivity (NDO) in spinal cord injury patients, and further development of EG 427’s HERMES vector technology platform.
This investment marks a significant step forward for the treatment of neuro-urological disorders, an area with limited therapeutic advances and high healthcare costs. The clinical trial of EG110A offers the potential for a novel treatment approach for NDO, a debilitating condition affecting bladder control. Success in this trial would not only validate EG 427’s technology but could also pave the way for its application in other neuro-urological conditions currently lacking effective treatment options. This represents a critical opportunity to improve the quality of life for a substantial patient population.
The €27 million investment will enable EG 427 to generate crucial safety and early efficacy data for EG110A, a non-replicating HSV-1 vector designed to silence overactive bladder signals without impacting voiding function. This funding also allows the company to broaden its pipeline by leveraging its proprietary HERMES technology, which boasts targeted delivery, potential for repeated dosing, and cost-effective production compared to AAV-based vectors. These advantages position EG 427 to offer potentially more effective and sustainable therapeutic solutions.
This successful funding round signals growing confidence in EG 427’s innovative approach to neuro-urological diseases. Positive clinical trial results for EG110A could significantly impact the treatment landscape for NDO and related conditions. Furthermore, advancement of the HERMES platform could lead to the development of new genetic medicines for a range of neurological disorders, ultimately benefiting patients and reducing the burden on healthcare systems.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Komen Calls for Urgent Action on Racial Disparities in Breast Cancer](https://www.clinicaltrialvanguard.com/news/komen-calls-for-urgent-action-on-racial-disparities-in-breast-cancer/)
**Published:** February 21, 2025
**Author:** Jon Napitupulu
**Content:**
New data reveals that Black women in the U.S. have a significantly higher mortality rate from [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) than white women, despite having a lower incidence rate. This disparity is attributed to systemic inequities in healthcare access, quality of care, and social determinants of health, not solely biological factors. Susan G. Komen emphasizes the urgent need for change and highlights potential solutions.
This disparity in breast cancer outcomes underscores the critical need for systemic reform within the healthcare system. The fact that Black women face a 40% higher mortality rate despite lower incidence points to significant gaps in access to and quality of care. Addressing these inequities is crucial not only for improving breast cancer outcomes in the Black community but also for advancing [health equity](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/) as a whole. The persistent nature of this issue, despite advancements in breast cancer treatment, demonstrates the deep-rooted nature of the problem and highlights the need for long-term, sustainable solutions.
Black women often experience delays at every stage of breast cancer care, from diagnosis to treatment completion. They are less likely to be imaged at facilities with cutting-edge technology and face increasing out-of-pocket costs for essential follow-up care, creating further barriers to timely treatment. Between 2018 and 2023, out-of-pocket expenses for follow-up care rose by 8%, compounding the financial burden for many. Low clinical trial participation rates among Black women further hinder research progress and the development of more effective and targeted treatments.
The future of breast cancer care requires a multi-pronged approach focused on policy changes that remove financial barriers, improve access to quality care, and address the underlying social determinants of health. Efforts to increase diverse participation in clinical trials are also essential for developing more effective and equitable treatments. Continued investment in research, advocacy, and community-based support programs will be critical to achieving health equity and eliminating the racial disparity in breast cancer outcomes.
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**Categories:** News
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### [Samsung Bioepis Launches Stelara® Biosimilar Pyzchiva® in US](https://www.clinicaltrialvanguard.com/news/samsung-bioepis-launches-stelara-biosimilar-pyzchiva-in-us/)
**Published:** February 24, 2025
**Author:** Jon Napitupulu
**Content:**
Samsung Bioepis launched PYZCHIVA ([ustekinumab](https://www.clinicaltrialvanguard.com/news/biosimilar-otuli-receives-fda-approval-encouraging-news-for-arthritis-patients/)-ttwe), a biosimilar to Stelara, in the United States. The drug is approved to treat plaque psoriasis, psoriatic arthritis, [Crohn’s disease](https://www.clinicaltrialvanguard.com/news/eli-lillys-omvoh-gets-fda-approval-for-crohns-disease-but-faces-challenges/)[Crohn’s](https://www.clinicaltrialvanguard.com/news/agomabs-crohns-drug-shows-promising-phase-2a-results/) disease, and ulcerative colitis and will be available in various formulations through their partner, Sandoz. This launch follows a 2023 commercialization agreement with Sandoz, with Samsung Bioepis retaining responsibility for development, manufacturing, and supply.
This launch is important because it introduces a new, potentially more affordable treatment option for patients with inflammatory conditions. Increased competition in the market can drive down costs, benefiting both patients and the healthcare system as a whole. This availability can enhance access to vital therapies for individuals who might otherwise face financial barriers to treatment.
PYZCHIVA’s availability begins prior to the February 2025 start date of the license agreement between Samsung Bioepis and Janssen Biotech Inc. Samsung Bioepis currently has ten approved biosimilars in the US market, with five commercially available, highlighting their significant presence in this rapidly expanding sector of the pharmaceutical industry. This adds to their portfolio which encompasses a range of therapeutic areas.
This launch positions Samsung Bioepis to further solidify its role in the biosimilars market. With a focus on expanding patient access to treatment, the company aims to strengthen its product portfolio and contribute to creating a more cost-effective healthcare landscape. The impact on the overall market for these specific therapies will depend on real-world effectiveness data and market adoption rates of PYZCHIVA.
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**Categories:** News
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### [Duvakitug Shows Promise in Ulcerative Colitis and Crohn's Disease](https://www.clinicaltrialvanguard.com/news/duvakitug-shows-promise-in-ulcerative-colitis-and-crohns-disease/)
**Published:** February 24, 2025
**Author:** Jon Napitupulu
**Content:**
Teva Pharmaceuticals and Sanofi announced positive Phase 2b results for duvakitug, a novel antibody targeting TL1A, in patients with moderate-to-severe [ulcerative colitis](https://www.clinicaltrialvanguard.com/news/abivax-clears-pre-nda-meeting-with-fda-for-obefazimod-in-ulcerative-colitis/) (UC) and [Crohn’s disease](https://www.clinicaltrialvanguard.com/news/eli-lillys-omvoh-gets-fda-approval-for-crohns-disease-but-faces-challenges/)[Crohn’s](https://www.clinicaltrialvanguard.com/news/agomabs-crohns-drug-shows-promising-phase-2a-results/) disease (CD). The RELIEVE UCCD study demonstrated that duvakitug achieved clinical remission in a significant portion of UC patients and endoscopic response in CD patients, exceeding placebo rates. The findings support the initiation of a Phase 3 program planned for the second half of 2025.
These results are potentially groundbreaking for IBD treatment. Current therapies often fail to achieve sustained remission or are associated with significant side effects. Duvakitug’s performance in both advanced therapy-experienced and -naïve patients suggests its potential to address unmet needs across a broad patient population. The safety profile, showing the drug was well-tolerated without new safety signals, further strengthens its potential as a valuable treatment option.
In the UC cohort, duvakitug achieved clinical remission rates of 36% (450mg dose) and 48% (900mg dose) compared to 20% for placebo. Additional endpoints like clinical response and endoscopic improvement also favored duvakitug. In the CD cohort, endoscopic response rates reached 26% (450mg) and 48% (900mg) versus 13% for placebo, with improvements also observed in endoscopic remission and clinical measures. Importantly, no dose-dependent adverse events were observed.
The positive Phase 2b data for duvakitug mark a crucial step towards a potential new therapeutic option for IBD. The planned Phase 3 program will be essential for confirming the efficacy and safety observed in this study and for defining duvakitug’s place in the IBD treatment landscape. If successful, duvakitug could offer a much-needed new approach to managing these chronic conditions.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Preparing and Upskilling the Clinical Trial Workforce for AI Innovation](https://www.clinicaltrialvanguard.com/conference-coverage/preparing-and-upskilling-the-clinical-trial-workforce-for-ai-innovation/)
**Published:** February 24, 2025
**Author:** Moe Alsumidaie
**Content:**
The [2025 SCOPE Summit’s](https://www.scopesummit.com/) fireside chat focused on how companies are preparing and upskilling their clinical trial workforce to innovate in the AI landscape. Industry leaders discussed the transformative impact of AI on clinical research, emphasizing the need for new skills and collaboration between sponsors and CROs. The session highlighted AI’s role in enhancing productivity and efficiency, urging companies to embrace this technology to stay competitive.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#the-role-of-ai-in-clinical-research)The Role of AI in Clinical Research
AI is revolutionizing clinical research by reshaping job roles and responsibilities. Solomon Babani from Syneos Health shared insights from his experience with a startup CRO that integrated AI into clinical operations. Initially, the team faced resistance due to unfamiliarity with AI technologies. However, the company fostered a culture of experimentation and learning by organizing weekly meetings where team members shared their AI usage experiences. This approach helped demystify AI and demonstrated its potential to enhance productivity and efficiency in clinical operations. Wanda Shoer from Sanofi emphasized that AI assists in decision-making by quickly synthesizing information, allowing professionals to focus on strategic tasks. This shift from administrative duties to more innovative thinking significantly changes job roles as AI takes over routine tasks. The panelists agreed that AI is not a job threat but a tool that can dramatically enhance work, leading to more efficient and effective clinical research processes.
#### [](#essential-skills-for-ai-integration)Essential Skills for AI Integration
Developing specific skills is crucial for effectively integrating AI into daily work. Wanda Shoer identified the need for a foundational understanding of AI tools and their applications, including knowing when and how to use different AI technologies. Beyond technical skills, she stressed the importance of human skills such as self-awareness, judgment, and collaboration. These skills are crucial for evaluating AI-generated information and making informed decisions based on professional expertise. Jimmy Bechtel from the Society for Clinical Research Sites added that effective prompting is critical for maximizing AI’s potential. He shared his learning journey of interacting with AI tools like ChatGPT to obtain more accurate and valuable outputs. By refining his approach to prompting, he enhanced the quality of information received from AI, demonstrating the importance of continuous learning and adaptation in the AI landscape.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#the-dynamics-between-sponsors-and-cros)The Dynamics Between Sponsors and CROs
The evolving dynamic between sponsors and CROs in AI adoption was a significant theme. Solomon Babani noted that historically, CROs have waited for sponsors to lead innovation. However, the advent of AI presents an opportunity for CROs to take the initiative and optimize clinical operations independently. This shift could redefine traditional industry roles as CROs leverage AI to enhance service offerings and drive efficiencies. Rosalie Filling from Endo Pharmaceuticals emphasized the importance of collaboration between sponsors and CROs. She urged sponsors to partner with CROs to benefit from their broader experience with AI technologies. By working together, sponsors and CROs can accelerate the integration of AI into clinical research, ultimately leading to more efficient and practical trials.
#### [](#success-stories-and-future-directions)Success Stories and Future Directions
Success stories shared by the panelists illustrated the tangible benefits of AI in clinical research. Rosalie Filling described how her company implemented an AI tool for medical writing, significantly reducing the time and cost of creating clinical study reports. This example emphasized the potential for AI to streamline processes and deliver substantial returns on investment, even for mid-sized pharmaceutical companies. Wanda Shoer discussed Sanofi’s comprehensive approach to AI investment, spanning various departments, including HR and supply chain management. This strategy demonstrates the wide-ranging applications of AI and its potential to transform multiple facets of the pharmaceutical industry. By aggregating internal data, Sanofi aims to make more informed decisions and improve operational efficiency across the board.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#summary)Summary
The [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Summit’s discussion on AI integration in clinical research highlights its critical role in shaping the industry’s future. Companies can enhance productivity and efficiency by embracing AI technologies and fostering collaboration between sponsors and CROs. The broader implications of AI adoption highlight the need for continuous learning and adaptation, ensuring that the industry remains competitive and innovative despite rapid technological advancements.
**Categories:** Article: Conference Coverage
---
### [FDA Grants Auron Therapeutics Fast Track Designation for AUTX-703 to Treat AML](https://www.clinicaltrialvanguard.com/news/fda-grants-auron-therapeutics-fast-track-designation-for-autx-703-to-treat-aml/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
Auron Therapeutics received FDA Fast Track designation for [AUTX-703](https://www.clinicaltrialvanguard.com/news/auron-therapeutics-announces-fda-clearance-and-series-b-financing/), an oral KAT2A/B degrader, for treating relapsed or refractory acute myelogenous [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/) (AML). The company plans to initiate clinical development in Q1 2025, following recent IND application clearance. This designation follows promising preclinical data demonstrating AUTX-703’s potential to improve survival rates in AML.
This Fast Track designation is particularly crucial given the limited treatment options and poor prognoses for relapsed/refractory AML patients, who have a five-year survival rate of around 10%. It validates the urgent need for new therapies in this area and recognizes AUTX-703 as a potential solution to address this unmet medical need. The expedited regulatory pathway afforded by the Fast Track designation could accelerate the drug’s development and availability to patients significantly.
AUTX-703, discovered using Auron’s AURIGIN™ platform, represents a first-in-class approach to AML treatment. This oral therapy offers a novel mechanism of action by degrading KAT2A/B, potentially offering a new avenue for managing this aggressive cancer. The FDA’s Fast Track designation allows for more frequent communication with the agency, potentially smoothing the path to regulatory approval. This designation also opens the door for Accelerated Approval and Priority Review, which could drastically shorten the time it takes to bring this potentially life-saving treatment to market.
This Fast Track designation positions AUTX-703 for expedited development and review, potentially accelerating its journey to becoming a much-needed treatment option for patients battling relapsed/refractory AML. The upcoming clinical trials will be critical for validating the preclinical findings and determining the drug’s efficacy and safety profile in humans. The progress of AUTX-703 could represent a significant advancement in AML treatment and offer renewed hope for patients facing this challenging disease.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Leti-101 Shows Promise in Preclinical Huntington's Disease Study](https://www.clinicaltrialvanguard.com/news/leti-101-shows-promise-in-preclinical-huntingtons-disease-study/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
ElevateBio has unveiled preclinical data for LETI-101, a [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/)-based therapy for Huntington’s Disease (HD) being developed by its subsidiary, Life Edit. The therapy utilizes an AAV5 vector to deliver a CRISPR nuclease targeting a specific single [nucleotide](https://www.clinicaltrialvanguard.com/news/ateas-new-hcv-combo-aims-for-best-in-class-treatment/) polymorphism (SNP) in the huntingtin gene (HTT) responsible for HD. Preclinical studies show LETI-101 successfully reduced mutant HTT protein levels by over 80% while preserving healthy HTT, a crucial aspect for maintaining normal cellular function.
This development is a significant advancement in the pursuit of effective HD treatments. Current HD therapies primarily address symptoms, not the underlying genetic cause. LETI-101’s targeted approach offers the potential to modify the disease course and significantly improve patient outcomes by addressing the root cause of the disease. This precision also minimizes the risk of off-target effects that could arise from less specific gene editing approaches. The positive preclinical data, coupled with regulatory guidance from the UK’s MHRA, increases the likelihood of LETI-101 progressing to clinical trials and eventually becoming a viable treatment option for HD patients.
In preclinical mouse models of HD, LETI-101 demonstrated a dose-dependent reduction of mutant HTT protein in critical brain regions affected by the disease. Studies in non-human primates confirmed the therapy’s safety profile and showed a promising biodistribution of the CRISPR system within the central nervous system, indicating effective delivery to target areas. Furthermore, the company has received positive regulatory feedback from the UK’s MHRA regarding the CMC and development pathway, which streamlines the process towards clinical trials.
The promising preclinical results and regulatory alignment position LETI-101 as a strong candidate for further development. ElevateBio’s integrated approach, combining gene editing technology, manufacturing capabilities, and therapeutic development expertise, suggests the potential for accelerated progress toward clinical trials. This development marks a crucial step towards a potential disease-modifying therapy for HD, offering hope for a future where this devastating disease can be effectively treated.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Brain Exercise Lowers Fatigue in MS Study](https://www.clinicaltrialvanguard.com/news/brain-exercise-lowers-fatigue-in-ms-study/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
Researchers at New York University (NYU) found that brain exercises from the BrainHQ app, developed by Posit Science, reduced fatigue in [Multiple Sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS) patients. This is the first study to demonstrate BrainHQ’s positive impact on MS-related fatigue, adding to existing research showing improvements in processing speed and cognitive function. The study, published in Nature: Scientific Reports, is the tenth publication on the use of BrainHQ exercises for MS patients.
This finding is particularly important for MS patients, as fatigue is a common and debilitating symptom that significantly impacts their quality of life. Current treatments for MS-related fatigue are often limited in effectiveness, highlighting the need for accessible and non-pharmacological interventions like BrainHQ. This research opens the door for a new approach to managing fatigue, potentially improving patients’ ability to participate in daily activities and enhancing their overall well-being.
The NYU study involved 117 MS patients randomized into two groups: one receiving transcranial Direct Current Stimulation (tDCS) while using BrainHQ, and the other receiving sham tDCS alongside BrainHQ training. Both groups completed thirty 20-minute BrainHQ training sessions over six weeks. Results showed a significant decrease in fatigue in both groups, with no added benefit observed from tDCS. This suggests that the BrainHQ exercises alone are driving the fatigue reduction.
This study reinforces the potential of BrainHQ exercises to address a significant unmet need for MS patients. The accessibility of the app, coupled with its demonstrated efficacy in reducing fatigue, could lead to wider adoption and integration into MS treatment plans. This could ultimately translate to improved quality of life for individuals living with MS.
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**Categories:** News
---
### [Calidi Starts High-Grade Glioma Trial Recruitment](https://www.clinicaltrialvanguard.com/news/calidi-starts-high-grade-glioma-trial-recruitment/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) has begun enrolling patients at Northwestern University/Northwestern Memorial Hospital for a clinical trial of [CLD-101](https://www.clinicaltrialvanguard.com/news/city-of-hope-calidi-biotherapeutics-phase-1-trial-update-on-cld-101/), an immunotherapy for newly diagnosed high-grade glioma (HGG). The therapy utilizes allogeneic neural stem cells carrying an oncolytic adenovirus, CRAd-S-pk7, and builds upon promising Phase 1 single-dose results published in •[The Lancet Oncology](https://www.clinicaltrialvanguard.com/news/successful-myriad-mrd-clinical-data-in-the-lancet-oncology/)•. This Phase 1B/2 trial will investigate multiple doses of CLD-101, led by Drs. Maciej Lesniak and Roger Stupp, and is funded by the NIH/NCI SPORE grant.
This trial represents a critical advancement in the fight against HGG, an aggressive brain cancer with limited treatment options. The shift to a multi-dose regimen in newly diagnosed patients could significantly improve efficacy compared to a single dose. This is especially crucial given the aggressive nature of the disease, offering the potential for earlier and more potent intervention. The involvement of renowned experts like Dr. Stupp, known for developing the Stupp Protocol for glioblastoma, lends considerable weight and expertise to the study.
The trial builds on previous single-dose data published in a top-tier journal. The NIH/NCI SPORE grant funding underscores the scientific community’s recognition of the therapy’s potential. This new trial is designed to evaluate the safety and efficacy of multiple doses, a key step towards establishing a more effective treatment protocol.
The results of this trial hold significant implications for the future of HGG treatment. Positive data could pave the way for a new standard of care, offering hope for improved survival and quality of life for patients diagnosed with this devastating cancer. It also further validates Calidi’s platform technology, strengthening its potential to develop treatments for other solid tumors and metastatic cancers.
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**Categories:** News
---
### [Versamune® HPV Clinical Cancer Research Results](https://www.clinicaltrialvanguard.com/news/versamune-hpv-clinical-cancer-research-results/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
PDS Biotechnology Corporation announced positive clinical trial results for its lead immunotherapy candidate, Versamune® HPV, in the treatment of locally advanced cervical cancer. The trial demonstrated that combining Versamune® HPV with standard chemoradiation (CRT) led to a faster and more complete clearance of [HPV16](https://www.clinicaltrialvanguard.com/news/pds-biotech-amends-phase-3-trial-for-accelerated-approval/)-positive cancer cells from the bloodstream compared to CRT alone. This clearance of circulating tumor DNA (ctDNA) strongly correlated with improved two-year recurrence-free survival rates.
These results are particularly promising because they demonstrate a potential for Versamune® HPV to not just treat, but potentially prevent cancer recurrence in HPV16-positive cancers. The strong correlation between ctDNA clearance and improved survival provides a compelling biomarker for tracking treatment efficacy and could potentially expedite regulatory approval pathways, like Breakthrough Therapy designation. Furthermore, these findings suggest that Versamune® HPV could be effective against other HPV16-positive cancers, such as head and neck squamous cell carcinoma ([HNSCC](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/)), paving the way for broader clinical applications.
In the study of 66 patients with locally advanced cervical cancer, 100% of the patients receiving the combination therapy showed no detectable HPV16 ctDNA at 3-4 months post-treatment, compared to 50% in the CRT-only group. This translated to a significantly higher two-year recurrence-free survival rate of 93% in the combination therapy group versus 30% in the CRT-only group. Previously reported data also indicated promising 36-month overall survival rates for patients receiving Versamune® HPV in combination with CRT. A Phase 3 clinical trial for Versamune® HPV in HPV16-positive HNSCC is planned for the first quarter of 2025.
This positive data strengthens the potential of Versamune® HPV as a valuable treatment option for various HPV-related cancers. The upcoming Phase 3 trial in HNSCC will be a crucial step in further validating its efficacy and broadening its clinical reach. The results may lead to a paradigm shift in HPV-related cancer treatment, potentially offering patients a significantly improved chance of long-term survival and a reduced risk of recurrence. Further research and development will focus on exploring its potential in other HPV-driven cancers and optimizing treatment strategies to maximize patient benefits.
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**Categories:** News
---
### [Leronlimab Shows Promise in mTNBC Patient Survival](https://www.clinicaltrialvanguard.com/news/leronlimab-shows-promise-in-mtnbc-patient-survival/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
[CytoDyn](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/) Inc. announced encouraging survival outcomes in a group of patients with metastatic triple-negative [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/) (mTNBC) treated with leronlimab, a CCR5 antagonist. Some patients who had failed prior treatments for metastatic disease survived beyond 36 months, are currently alive, and show no evidence of active disease. Following the resolution of a dispute with its former CRO, CytoDyn accessed follow-up records from patients previously treated with leronlimab in [oncology trials](https://www.clinicaltrialvanguard.com/news/mindranks-mrank-106-gets-fda-clearance-for-oncology-trials/). These findings have been summarized and submitted as an abstract to the European Society for Medical Oncology (ESMO) Breast Cancer meeting in May 2025.
This news is potentially impactful for both the oncology field and patients with mTNBC, a particularly aggressive and difficult-to-treat cancer. The observed survival rates, especially in patients who had exhausted other treatment options, suggest that leronlimab may offer a new therapeutic avenue. This is particularly noteworthy given the limited treatment options and generally poor prognosis associated with mTNBC. The apparent lack of significant adverse events associated with leronlimab further strengthens its potential as a viable treatment option.
CytoDyn is initiating two pre-clinical studies investigating potential synergistic effects of leronlimab in combination with sacituzumab govitecan (an antibody-drug conjugate) and pembrolizumab (an immune checkpoint inhibitor). The company is also continuing follow-up testing on the long-term survivors currently demonstrating no evidence of disease.
This development could mark a significant step forward in the treatment of mTNBC. The upcoming presentation at the ESMO Breast Cancer meeting will provide a valuable opportunity for peer review and discussion within the oncology community. The pre-clinical studies focusing on combination therapies hold promise for further enhancing treatment efficacy. The ongoing follow-up of long-term survivors will be crucial to further understanding the long-term effects and potential of leronlimab in mTNBC.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Allo-Inkt Combination Data in 2L Gastric Cancer at AACR](https://www.clinicaltrialvanguard.com/news/allo-inkt-combination-data-in-2l-gastric-cancer-at-aacr/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
MiNK Therapeutics presented Phase 2 data at the AACR IO Annual Meeting showcasing the potential of its allogeneic iNKT [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), agenT-797, combined with [botensilimab](https://www.clinicaltrialvanguard.com/news/updated-phase-2-agent-797-gastric-cancer-data-at-aacr-io/) and balstilimab (BOT/BAL) for refractory gastroesophageal cancer. Early results indicate this combination therapy triggers significant immune activation within the tumor microenvironment. The study demonstrates the importance of treatment sequencing, with optimal results observed when agenT-797 is administered alongside checkpoint inhibitors before chemotherapy.
This research holds significant promise for patients battling refractory gastroesophageal cancers, who currently face limited treatment options. The observed immune reactivation, including increased interferon-gamma levels and enhanced T-cell infiltration into the tumor, suggests the potential for durable clinical responses. The combination therapy could represent a crucial advance in treating these aggressive cancers.
Technically, the study highlights the synergistic interaction between agenT-797, checkpoint inhibitors, and chemotherapy, demonstrating how iNKT cell therapy can amplify the anti-tumor immune response. Strategically, MiNK’s allogeneic, off-the-shelf manufacturing process positions them to overcome the logistical and cost barriers associated with autologous cell therapies, potentially expanding patient access globally. The platform technology also allows for broader application across various hard-to-treat cancers.
This positive Phase 2 data strengthens the case for further clinical investigation of agenT-797 in combination with BOT/BAL. It positions MiNK as a key player in the iNKT cell therapy space and suggests a potential shift in the treatment landscape for gastroesophageal and other solid tumor cancers. The scalability of their manufacturing process could make this promising therapy accessible to a wider patient population, representing a significant advancement in cancer treatment.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Synergia Medical Announces Strong Results for nao.vns First-in-Human Study](https://www.clinicaltrialvanguard.com/news/synergia-medical-announces-strong-results-for-nao-vns-first-in-human-study/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
Synergia Medical’s NAO.VNS™ device, implanted in five patients in Belgium, has met its primary safety endpoint in the AURORA first-in-human clinical study. The implantable device, designed to treat drug-resistant [epilepsy](https://www.clinicaltrialvanguard.com/news/fda-grants-breakthrough-therapy-designation-to-elsunersen-for-scn2a-epilepsy/) (DRE) via vagus nerve stimulation (VNS), demonstrated no serious adverse events or device failures over three months. This next-generation neuromodulation platform eliminates metal components found in traditional devices, resulting in full compatibility with MRI and other electromagnetic interference (EMI) sources like electrosurgery and defibrillators.
This successful initial trial is a critical step forward for patients with DRE, a debilitating condition for which current treatment options are often inadequate. The NAO.VNS™ device offers several potential advantages over existing VNS technology. Uninterrupted treatment during MRI scans allows physicians to monitor brain activity and tailor therapy in real-time. Moreover, the device’s innovative battery technology, requiring only one minute of daily charging for an estimated 15-year lifespan, significantly improves patient convenience and reduces the need for repeat surgeries for battery replacement.
Technically, the device has demonstrated robust performance and safety. Three months post-implantation, no adverse events or device malfunctions were reported. From a patient perspective, initial feedback highlights the convenience of the fast-charging, long-lasting battery. Strategically, Synergia Medical is now poised to initiate pivotal FDA and CE trials, paving the way for potential market approval.
This positive early data suggests a promising future for the NAO.VNS™ device and for patients with DRE. Successful completion of pivotal trials could lead to a paradigm shift in neuromodulation, offering a safer, more convenient, and potentially more effective treatment option. The ability to conduct real-time MRI-guided therapy adjustments could significantly improve treatment outcomes and personalize care for individuals struggling with DRE.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [EMA and EU-Medicines for All Validate Lenacapavir HIV Prevention Application](https://www.clinicaltrialvanguard.com/news/ema-and-eu-medicines-for-all-validate-lenacapavir-hiv-prevention-application/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
Gilead Sciences announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) and EU-Medicines for all (EU-M4all) application for lenacapavir, a twice-yearly injectable HIV prevention drug. The EMA granted accelerated assessment based on the potential public health impact of lenacapavir. This follows the FDA’s acceptance of similar applications under priority review.
This accelerated review by the EMA signifies a potential turning point in HIV prevention. A twice-yearly injection could drastically improve adherence compared to daily oral PrEP medications, potentially leading to a significant reduction in new HIV infections, particularly in underserved communities. The parallel EU-M4all application also demonstrates a commitment to equitable access by facilitating expedited review processes in low- and lower-middle-income countries.
The EMA’s validation is based on positive Phase 3 trial data. The PURPOSE 1 trial in cisgender women demonstrated 100% risk reduction in HIV infections with lenacapavir compared to background incidence rates. The PURPOSE 2 trial in cisgender men and gender-diverse people showed a 96% risk reduction. In both trials, lenacapavir was superior to daily oral Truvada and demonstrated a generally well-tolerated safety profile.
The EMA’s accelerated review, combined with the FDA’s priority review, suggests a high likelihood of regulatory approval in the near future. This could pave the way for a paradigm shift in HIV prevention strategies globally, offering a long-acting and potentially more convenient option for individuals and healthcare providers. The parallel review for low- and lower-middle-income countries reinforces a commitment to global [health equity](https://www.clinicaltrialvanguard.com/news/unlock-the-doors-of-health-equity-walgreens-and-boehringer-ingelheims-partnership/) and underscores the potential for lenacapavir to significantly impact the HIV epidemic worldwide.
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**Categories:** News
---
### [LTZ-301: First-in-Class Myeloid Engager Immunotherapy IND Cleared by FDA](https://www.clinicaltrialvanguard.com/news/ltz-301-first-in-class-myeloid-engager-immunotherapy-ind-cleared-by-fda/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
LTZ Therapeutics received FDA clearance for its Investigational New Drug (IND) application for LTZ-301. LTZ-301, a first-in-class myeloid engager immunotherapy, will be investigated for the treatment of relapsed or refractory non-Hodgkin [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (r/r NHL). The company plans to initiate a Phase 1 clinical trial in Q2 2025.
This IND clearance is a crucial step in validating LTZ’s myeloid engager platform. It opens the door to clinical investigation of a novel therapeutic approach for r/r NHL, a patient population often facing limited treatment options after standard therapies fail. Success in this trial could establish a new treatment paradigm for these patients and broaden the applicability of myeloid engager therapies to other cancers and autoimmune diseases.
The Phase 1 study will be a multicenter, open-label trial evaluating the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of LTZ-301. LTZ-301 is a bi-specific antibody designed to enhance the phagocytic function of monocytes and macrophages, targeting them to eliminate CD79b-expressing [B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/). Preclinical data suggests LTZ-301 shows promising activity and a favorable safety profile. The appointment of Dr. Wayne Godfrey as Chief Medical Officer and Dr. Alan J. Korman to the scientific advisory board further strengthens LTZ’s leadership in this area.
This IND clearance allows LTZ to translate promising preclinical findings into clinical investigation. Positive results from the Phase 1 trial could pave the way for further clinical development of LTZ-301, potentially offering a new and effective treatment option for patients with r/r NHL. It could also spur further development of LTZ’s myeloid engager platform for other indications, marking a significant advancement in the immunotherapy landscape.
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**Categories:** News
---
### [PrecivityAD2 Blood Test Certified in UK](https://www.clinicaltrialvanguard.com/news/precivityad2-blood-test-certified-in-uk/)
**Published:** February 25, 2025
**Author:** Jon Napitupulu
**Content:**
[C2N Diagnostics](https://www.clinicaltrialvanguard.com/news/c2n-diagnostics-and-unilabs-forge-unprecedented-alliance-to-advance-brain-health/) has received regulatory approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its PrecivityAD2 blood test, designed to aid in the diagnosis of [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease in patients aged 55 and older experiencing cognitive decline. This marks the first global regulatory approval for a blood test specifically for Alzheimer’s disease diagnosis. The test analyzes multiple blood components to determine the likelihood of amyloid plaque presence in the brain, a key indicator of Alzheimer’s.
This approval is particularly important for the UK, which has a high prevalence of Alzheimer’s disease and limited access to traditional diagnostic methods like PET scans. The availability of a less invasive and more accessible blood test could significantly improve early diagnosis rates and facilitate timely interventions, ultimately impacting patient outcomes and healthcare resource allocation. Early diagnosis not only enables lifestyle changes that may delay disease progression, but also allows for more informed treatment decisions and participation in clinical trials.
The PrecivityAD2 test has demonstrated strong clinical performance in studies, with a recent publication showing 92% negative predictive value and 91% positive predictive value using a single cutoff, and 95% for both using a two-cutoff approach. This performance surpasses recent expert consensus recommendations for blood-based Alzheimer’s diagnostic tests, positioning it as a potential alternative to more invasive procedures.
This approval paves the way for broader access to Alzheimer’s diagnostics and represents a significant advancement in the field. It could lead to earlier and more widespread diagnosis, potentially altering the course of the disease for many individuals and reducing the overall burden on healthcare systems grappling with the growing prevalence of Alzheimer’s. The increased accessibility to accurate testing may also accelerate research and development of new treatments as more patients are identified and enrolled in clinical trials.
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**Categories:** News
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### [How AstraZeneca and Mass General Brigham are Enhancing Clinical Trial Protocol Design](https://www.clinicaltrialvanguard.com/conference-coverage/how-astrazeneca-and-mass-general-brigham-are-enhancing-clinical-trial-protocol-design/)
**Published:** February 25, 2025
**Author:** Moe Alsumidaie
**Content:**
The [SCOPE 2025 Summit](https://www.scopesummit.com/ "SCOPE 2025 Summit") showcased a groundbreaking partnership between Mass General Brigham (MGB), [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/), and Evinova, highlighting how these organizations are revolutionizing clinical research through collaboration and technology. Key discussions focused on enhancing communication, utilizing data effectively, and adopting patient-centric approaches to improve trial efficiency and outcomes. This partnership exemplifies the industry’s shift towards integrated research processes, setting a new standard for clinical trials.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#the-power-of-collaboration)The Power of Collaboration
The collaboration between MGB, AstraZeneca, and Evinova represents a pioneering approach in clinical trials, where diverse expertise and resources drive significant advancements. Chris Herrick from MGB emphasized the transformative impact of this partnership, which has fundamentally altered their approach to clinical research. By combining MGB’s extensive healthcare network, AstraZeneca’s pharmaceutical innovation, and Evinova’s digital health solutions, the partnership is setting a new benchmark for clinical trials. This alliance leverages each organization’s strengths, creating a comprehensive framework that enhances trial efficiency and effectiveness. MGB’s 200-year legacy as a premier academic medical center provides a robust infrastructure and a wealth of real-world data. AstraZeneca contributes its vast experience in drug development and patient-centered science, while Evinova brings innovative digital recruitment strategies. Together, they are creating a comprehensive framework that enhances the efficiency and effectiveness of clinical trials.
#### [](#enhancing-communication-and-data-utilization)Enhancing Communication and Data Utilization
A central theme of the conference was the critical role of communication and data utilization in optimizing clinical trials. MGB’s HERO initiative addressed the communication gap between research sites and sponsors. By establishing a holistic communication strategy, HERO ensures that all stakeholders are informed and aligned throughout the research process. This proactive approach includes regular meetings with sponsors to discuss pipeline developments and potential collaborations, facilitating smoother interactions and more effective study planning. In addition to improving communication, MGB has made significant strides in data utilization. The organization has developed a sophisticated system for structuring and standardizing real-world data, which includes genomics, imaging, clinical notes, and patient-reported outcomes. This comprehensive data infrastructure allows MGB to quickly assess study feasibility and identify eligible patients, thereby streamlining the recruitment process. Integrating diverse data types into research protocols is particularly valuable as the industry moves towards precision medicine, enabling more targeted and effective treatments.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#astrazenecas-patient-centric-approach)AstraZeneca’s Patient-Centric Approach
AstraZeneca’s commitment to patient-centricity was a focal point of the discussion, with Michele Teufel highlighting the company’s efforts to incorporate patient feedback into study design. Using MGB’s extensive database during the protocol development stage, AstraZeneca can make informed adjustments that enhance study feasibility and patient engagement. This approach improves recruitment outcomes and ensures that studies are designed with the patient’s best interests. The company’s Patient Insight team is crucial in this process, providing valuable input on protocol design and patient engagement strategies. AstraZeneca’s patient partnership program allows patients to interact directly with protocol designers, offering insights on inclusion criteria, study procedures, and technology use. This direct feedback loop ensures that studies are tailored to meet patient needs, ultimately leading to more successful recruitment and retention.
#### [](#digital-innovation-in-patient-recruitment)Digital Innovation in Patient Recruitment
Kelly McKee of Evinova addressed the persistent challenge of patient recruitment in large hospital systems and the innovative solutions implemented to overcome these obstacles. Despite MGB’s vast network and patient base, recruitment remains complex. Evinova enhances communication and transparency, leveraging HERO’s resources to identify and engage patients more effectively. One of the key strategies involves utilizing both traditional and digital communication channels to reach patients. While digital tools are essential, McKee noted that sometimes traditional methods, such as direct mail, can be surprisingly effective. This multifaceted approach ensures that communication is tailored to the preferences of different patient populations, thereby improving engagement and recruitment outcomes. Evinova’s collaboration with MGB also involves testing protocols through electronic medical record (EMR) mining, allowing for protocol adjustments before finalization. This proactive approach saves time and resources, reducing site-level frustrations and enhancing overall study efficiency. By focusing on optimizing the experiences of patients, sites, and sponsors, the partnership is setting a new standard for patient recruitment in clinical trials.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#expanding-the-model)Expanding the Model
The potential for expanding this collaborative model to other academic medical centers was a topic of keen interest at the conference. Chris Herrick confirmed that MGB actively engages with other institutions to share best practices and streamline research processes. This expansion could lead to a more unified approach to clinical trials, benefiting sponsors and patients by reducing inefficiencies and improving study outcomes. Other academic medical centers’ interest is driven by the need to optimize research processes in the face of financial pressures and operational challenges. By adopting the HERO model, these institutions can enhance communication, streamline data utilization, and improve patient recruitment. From the sponsor’s perspective, having a single entry point to engage with multiple hospitals and systems simplifies the research process and enhances collaboration.
#### [](#overcoming-operational-challenges)Overcoming Operational Challenges
Operational challenges, particularly those related to institutional review boards (IRBs) and contracting processes, were also addressed during the conference. MGB has significantly reduced these timelines by fostering strong relationships with IRBs and proactively addressing potential issues. By regularly communicating with IRBs and providing advance notice of upcoming studies, MGB has been able to shorten approval timelines, enhancing study initiation efficiency significantly. This proactive approach also extends to contracting processes, where MGB has implemented standardized templates and protocols to streamline negotiations. By anticipating potential issues and preparing stakeholders in advance, MGB has reduced surprises and improved overall efficiency. These efforts demonstrate the value of collaboration and pre-planning in overcoming operational hurdles, ultimately leading to more successful clinical trials.
#### [](#summary)Summary
In conclusion, the conference highlighted the transformative potential of collaboration and innovation in clinical trials. By integrating communication, data, and patient-centric approaches, the partnership between MGB, AstraZeneca, and Evinova sets a new standard for the industry, paving the way for more efficient and effective research that ultimately benefits patients worldwide.
**Categories:** Article: Conference Coverage
---
### [Candel Therapeutics Announces Positive CAN-2409 Trial Data](https://www.clinicaltrialvanguard.com/news/candel-therapeutics-announces-positive-can-2409-trial-data/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
[Candel Therapeutics](https://www.clinicaltrialvanguard.com/news/candel-therapeutics-positive-phase-3-results-in-prostate-cancer/) announced positive final overall survival data from its Phase 2 trial of [CAN-2409](https://www.clinicaltrialvanguard.com/news/new-phase-3-data-for-can-2409-in-prostate-cancer-at-astro/) in patients with borderline resectable pancreatic ductal adenocarcinoma (PDAC). The experimental treatment, combined with standard chemotherapy and radiation, showed a significant increase in median overall survival to 31.4 months compared to 12.5 months for the control group. Three of the seven patients treated with CAN-2409 survived beyond 35 months, significantly exceeding typical survival rates for this aggressive cancer.
This data is particularly encouraging for PDAC patients, a population facing a historically grim prognosis. Borderline resectable PDAC is challenging to treat due to the frequent presence of undetectable micrometastases that standard therapies often miss. CAN-2409’s potential to stimulate a robust and sustained anti-tumor immune response offers hope for improved long-term survival, even in patients with residual disease after surgery. The survival data, coupled with earlier findings on immune system activation within the tumor microenvironment, suggest that CAN-2409 may be altering the disease course in a meaningful way.
The Phase 2 trial showed a median overall survival of 31.4 months in the CAN-2409 arm versus 12.5 months in the control arm. Median post-progression survival was also substantially longer in the CAN-2409 group (21.2 months) compared to the control group (7.2 months). Importantly, the therapy exhibited a favorable safety profile, with no dose-limiting toxicities observed. This positive safety profile combined with the promising survival data further strengthens the rationale for advancing CAN-2409 into a larger, late-stage clinical trial. CAN-2409 has already received Fast Track and Orphan Drug Designations from the FDA for PDAC.
The positive results from this Phase 2 trial, along with recent positive data from a Phase 3 trial in [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/), suggest a broader potential for CAN-2409 across multiple solid tumors. Candel Therapeutics is planning a larger, late-stage randomized controlled trial for CAN-2409 in PDAC, which could ultimately lead to a new treatment option for this challenging cancer. This development underscores the potential of multimodal immunotherapies to improve outcomes for patients with difficult-to-treat cancers.
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---
### [Alzamend Neuro Starts Phase 2 Trial of AL001 at Mass General](https://www.clinicaltrialvanguard.com/news/alzamend-neuro-starts-phase-2-trial-of-al001-at-mass-general/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
Alzamend Neuro plans to initiate the first of five Phase II clinical trials for [AL001](https://www.clinicaltrialvanguard.com/news/alzamend-neuro-and-qmenta-in-ai-imaging-trial/), a novel lithium-delivery system, in healthy human subjects during the second quarter of 2025. This study, conducted in partnership with Massachusetts General Hospital, will compare AL001 to currently available lithium treatments, focusing on lithium’s pharmacokinetics in the brain and blood. The goal is to demonstrate AL001’s potential to improve lithium delivery to the brain while minimizing systemic side effects.
This trial is important because it could revolutionize lithium therapy, a cornerstone treatment for bipolar disorder and increasingly relevant in Alzheimer’s, [major depressive disorder](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/), and PTSD. Current lithium treatments require burdensome therapeutic drug monitoring (TDM) due to a narrow therapeutic window and potential toxicity. If AL001 proves to be effective and safer, with less need for TDM, it could significantly expand lithium’s clinical utility and improve patient compliance, potentially impacting millions of patients.
The study will leverage a novel head coil developed by Tesla Dynamic Coils BV, crucial for precise measurement of lithium distribution in the brain. Preclinical studies in mice have already suggested AL001’s superior brain absorption and lower blood lithium levels. Alzamend aims to confirm these findings in humans and establish a foundation for future trials in patients with neurological and psychiatric disorders. A previously completed Phase IIA study in Alzheimer’s patients and healthy subjects has already established a maximum tolerated dose for AL001, designed to minimize the need for TDM.
This first Phase II study represents a crucial step in Alzamend’s development of AL001. Positive results could validate the drug’s novel approach to lithium delivery and accelerate its progression towards later-stage clinical trials. Ultimately, Alzamend envisions AL001 as a next-generation lithium treatment offering improved safety, efficacy, and patient convenience, potentially transforming the treatment landscape for a range of [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions.
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---
### [Ampligen and Imfinzi in Phase 2 Pancreatic Cancer Trial](https://www.clinicaltrialvanguard.com/news/ampligen-and-imfinzi-in-phase-2-pancreatic-cancer-trial/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
AIM [ImmunoTech](https://www.clinicaltrialvanguard.com/news/immunotech-announces-cash-conservation-plan/) has dosed the first patient in Phase 2 of its Phase 1b/2 clinical trial, DURIPANC, evaluating Ampligen (rintatolimod) combined with [AstraZeneca](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/)‘s Imfinzi ([durvalumab](https://www.clinicaltrialvanguard.com/news/ivonescimab-beats-durvalumab-in-phase-iii-biliary-tract-cancer-trial/)) for late-stage pancreatic cancer. Several participants from Phase 1 who received the highest dose of Ampligen will continue into Phase 2. The open-label, single-center study, conducted at Erasmus Medical Center in the Netherlands, anticipates enrolling up to 25 patients in this phase.
This advancement is particularly important for pancreatic cancer patients, as the disease often presents late, leaving few effective treatment options. Combining Ampligen, a TLR3 agonist, with Imfinzi, a PD-L1 checkpoint inhibitor, offers a novel approach to potentially stimulate a stronger immune response against the cancer. Steady enrollment is anticipated, a significant aspect in a disease where patient recruitment for trials can be challenging. This allows for more efficient data collection and potentially faster progress toward a new therapeutic strategy.
The DURIPANC trial is an investigator-initiated study exploring the combination of these two drugs. Phase 2 will involve up to 25 patients and follows a Phase 1 portion that explored dosage levels of Ampligen. The inclusion of patients from the highest dose cohort of Phase 1 provides valuable continuity and data for evaluating the treatment’s efficacy and safety profile at this dose level.
The initiation of Phase 2 of the DURIPANC trial represents a crucial step forward in developing a potential new treatment approach for late-stage pancreatic cancer. Positive results from this phase could lead to larger trials and bring hope to patients facing this aggressive disease. The combination therapy’s progress may stimulate further research into combined immunotherapeutic strategies for challenging cancers.
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---
### [Silexion Announces Promising Pancreatic Cancer Study Results](https://www.clinicaltrialvanguard.com/news/silexion-announces-promising-pancreatic-cancer-study-results/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) (NASDAQ: SLXN) has completed data collection for its first study of [SIL-204](https://www.clinicaltrialvanguard.com/news/silexion-shows-strong-tumor-reduction-in-pancreatic-cancer-models/) in orthotopic [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) models. This study examined the drug’s ability to shrink primary tumors and limit metastasis after systemic administration. The company expects to release initial results in March 2025.
This study is important because orthotopic models, where tumor cells are implanted directly into the pancreas, offer a more accurate representation of human pancreatic cancer than traditional subcutaneous models. This allows researchers to better understand how a drug might perform in a clinically relevant setting, particularly regarding metastatic spread, a hallmark of pancreatic cancer. The evaluation of SIL-204 in this context could significantly impact the development of new therapies, potentially leading to more effective treatments.
This study represents the first time SIL-204 has been systematically evaluated in orthotopic pancreatic cancer models after subcutaneous administration. It focused on the drug’s impact on both primary tumor growth and the spread of the cancer to other organs. The data analysis is currently underway.
The results of this study will directly influence Silexion’s development strategy for SIL-204. Positive data could accelerate the drug’s clinical development and potentially broaden its application to include both localized and metastatic pancreatic cancer, representing a significant advancement in the treatment of this challenging disease. This could also position Silexion as a leader in the development of RNAi therapies for KRAS-driven cancers.
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---
### [FemBloc Permanent Birth Control: Positive Safety and Efficacy Results](https://www.clinicaltrialvanguard.com/news/fembloc-permanent-birth-control-positive-safety-and-efficacy-results/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
Femasys Inc. announced positive clinical trial data for [FemBloc](https://www.clinicaltrialvanguard.com/news/femasys-begins-new-fembloc-birth-control-study-in-europe/), a non-surgical permanent birth control method, published in the Journal of Gynecology & Reproductive Medicine. The trials demonstrated a 0% pregnancy rate among participants three months post-procedure, significantly outperforming traditional surgical sterilization. The five-year data also highlighted a strong safety profile, with no serious adverse events reported and high satisfaction rates among both patients and practitioners.
This development is potentially transformative for women’s healthcare. FemBloc offers a significantly less invasive and more accessible permanent birth control option compared to existing surgical procedures. This could lead to increased adoption of permanent contraception, particularly among individuals who may have barriers to accessing or choosing surgical options. The positive safety and efficacy data could also influence clinical practice guidelines and payer coverage decisions, furthering its potential impact on women’s reproductive health choices.
The clinical trials included 229 participants, 51 of whom were evaluated for pregnancy rates after confirmed bilateral fallopian tube occlusion. No pregnancies occurred in this group, surpassing the performance goal based on the historical effectiveness rate of surgical sterilization. Notably, the procedure demonstrated a consistent safety profile over five years, with only minor adverse events typical of transcervical procedures.
These results pave the way for FemBloc’s potential market entry, pending U.S. regulatory approval. The promising efficacy and safety data, coupled with high patient and physician satisfaction, position FemBloc to disrupt the current landscape of permanent contraception. Further research and post-market surveillance will be important to confirm these initial findings and assess the long-term impact of this innovative approach to family planning.
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### [Trethera & UCLA Publish Data on TRE-515 in Mice with MS](https://www.clinicaltrialvanguard.com/news/trethera-ucla-publish-data-on-tre-515-in-mice-with-ms/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
Trethera Corporation announced research published in Immunology demonstrating that their drug, [TRE-515](https://www.clinicaltrialvanguard.com/news/trethera-mgh-launch-clinical-trial-for-tre-515-in-als/), effectively inhibits the hyperactive lymphocytes responsible for [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS) symptoms in mouse models. TRE-515 targets deoxycytidine kinase (dCK), an enzyme crucial for cell division, specifically within the overactive T and B cells driving MS. This targeted approach reduces the number of these harmful cells without affecting other essential immune cells.
This research offers a potential new treatment avenue for MS and other demyelinating diseases. Current MS therapies often suppress the broader immune system, increasing susceptibility to infections and other complications. TRE-515’s selective action on disease-causing cells could lead to a safer and more effective treatment option, addressing an unmet need for targeted therapies with fewer side effects. The ability to preserve the normal function of the immune system while selectively targeting the aberrant cells driving the disease could significantly improve patients’ quality of life and long-term health outcomes.
The research demonstrates that dCK is upregulated in MS, making it a suitable therapeutic target. Inhibition of dCK by TRE-515 effectively reduced disease severity in the mouse models by limiting the proliferation of autoreactive T and B cells, directly responsible for the nerve damage characteristic of MS. This mechanism differs from existing MS drugs that target the •de novo• [nucleotide](https://www.clinicaltrialvanguard.com/news/ateas-new-hcv-combo-aims-for-best-in-class-treatment/) synthesis pathway, suggesting a novel therapeutic strategy. The research builds on previous findings published in Immunology, solidifying the role of dCK as a promising target in MS.
This positive preclinical data strengthens the potential of TRE-515 as a viable treatment for MS. The selective targeting of dCK offers a promising new strategy for managing autoimmune diseases with improved precision and potentially fewer side effects. Further research and clinical trials will be critical to validating these findings in humans and establishing the efficacy and safety profile of TRE-515. This research holds considerable promise for advancing MS treatment and potentially expanding therapeutic options for other autoimmune conditions.
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### [FDA Accepts Medincell and Teva Uzedy for Bipolar I Disorder Treatment](https://www.clinicaltrialvanguard.com/news/fda-accepts-medincell-and-teva-uzedy-for-bipolar-i-disorder-treatment/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
Teva Pharmaceuticals and [Medincell](https://www.clinicaltrialvanguard.com/news/medincell-teva-olanzapine-lai-phase-3-positive-uzedy-real-world-data/) announced that the FDA accepted their supplemental New Drug Application for UZEDY, a long-acting injectable risperidone formulation, for the maintenance treatment of bipolar I disorder in adults. UZEDY is already approved in the US for [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) treatment and is administered subcutaneously every one or two months. This application leverages existing UZEDY clinical data combined with prior FDA findings on the safety and efficacy of other risperidone formulations for bipolar I disorder.
This FDA acceptance signifies a potential advancement in bipolar I disorder treatment. Long-acting injectables offer a critical advantage for managing this chronic condition, where adherence to daily oral medication regimens is often a significant challenge impacting treatment success. This new potential indication for UZEDY could offer patients a more convenient and reliable way to receive medication, potentially leading to better symptom control and improved long-term outcomes. This is crucial given the severe impact of bipolar I disorder, characterized by debilitating manic and depressive episodes that significantly affect a person’s mood, behavior, and daily functioning.
UZEDY utilizes Medincell’s BEPO technology, enabling sustained release of risperidone following subcutaneous injection. The sNDA submission relies on existing data from UZEDY’s schizophrenia trials (RISE and SHINE) along with established knowledge of risperidone’s effectiveness in bipolar I disorder. Teva will lead the regulatory process and commercialization efforts, while Medincell is eligible for royalties on net sales if approved.
This sNDA acceptance positions UZEDY as a potential first-in-class long-acting injectable treatment for bipolar I disorder. A positive FDA decision would not only expand Teva’s market reach but also significantly improve treatment options for patients struggling with adherence to current therapies. This development underscores the potential of long-acting injectables to address persistent challenges in managing chronic [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions.
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### [Oric Pharmaceuticals Announces Development Plans, Cash Runway, and Milestones](https://www.clinicaltrialvanguard.com/news/oric-pharmaceuticals-announces-development-plans-cash-runway-and-milestones/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
ORIC Pharmaceuticals announced focused development plans for its lead programs, ORIC-944 and [ORIC-114](https://www.clinicaltrialvanguard.com/news/new-hope-in-nsclc-enozertinibs-powerful-cns-activity/), extending its cash runway into 2027. The company will prioritize indications with the strongest clinical validation and highest unmet need, specifically metastatic castration-resistant prostate cancer (mCRPC) for ORIC-944 and first-line [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) for ORIC-114. Favorable enrollment in ongoing trials has allowed for accelerated milestones and data readouts.
This shift in strategy is crucial for ORIC, demonstrating an adaptive response to emerging clinical data and market conditions. By prioritizing resources towards the most promising indications, ORIC increases its chances of achieving regulatory success and securing market share. Focusing on first-line therapies in NSCLC, where the patient population is larger and the need for effective treatments is greater, offers a larger commercial opportunity than later-line settings. This decision reflects a pragmatic approach to resource allocation in a challenging financial environment.
ORIC-944’s Phase 3 trial in mCRPC is expected to begin in the first half of 2026. Combination dose escalation data with apalutamide and darolutamide are anticipated in the first half and second half of 2025, respectively. ORIC-114 will see comprehensive data updates across multiple NSCLC cohorts in the second half of 2025, with registrational trials in first-line NSCLC planned for 2026. The company’s cash runway now extends into 2027, providing more time to achieve these key milestones.
This focused approach sets the stage for a potentially transformative period for ORIC. Successful clinical data in these prioritized indications could validate the company’s platform and lead to the approval of much-needed therapies for patients with mCRPC and NSCLC. The extended cash runway provides a crucial financial buffer, allowing ORIC to execute its development strategy and potentially deliver value to both patients and investors.
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### [Castle Creek Biosciences Secures $75M Royalty Financing](https://www.clinicaltrialvanguard.com/news/castle-creek-biosciences-secures-75m-royalty-financing/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
Ligand Pharmaceuticals has invested $50 million and led a syndicate of co-investors, including Paragon Biosciences, Valor Equity Partners, and XOMA Royalty Corporation, in a $25 million royalty financing agreement with Castle Creek Biosciences. This funding will support the Phase 3 clinical trial of Castle Creek’s lead gene therapy candidate, D-Fi (FCX-007), for the treatment of dystrophic epidermolysis bullosa (DEB). In return, the investors receive a high-single-digit royalty on future global D-Fi sales.
This investment underscores growing confidence in the potential of D-Fi to address the significant unmet medical need in DEB, a rare and debilitating genetic skin disorder. Successfully completing the Phase 3 trial is crucial for Castle Creek as it represents a major step toward commercialization and potentially delivering a transformative treatment to patients. The involvement of prominent investors like Ligand, Paragon, Valor, and XOMA further validates the therapeutic promise of D-Fi and its market potential. For Ligand, this deal represents a strategic move to diversify its revenue streams through royalties on a promising late-stage asset.
The Phase 3 trial will evaluate the efficacy and safety of D-Fi, an autologous gene-modified [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) designed to correct the underlying genetic defect responsible for DEB. The therapy involves modifying a patient’s own skin cells to produce functional type VII collagen, a protein crucial for skin integrity. Early clinical data suggests D-Fi is generally well-tolerated, with injection site reactions being the primary adverse event. The FDA has granted D-Fi Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations, highlighting the therapy’s potential and the urgent need for effective DEB treatments.
This investment positions D-Fi for a critical stage of development. Positive Phase 3 results could pave the way for regulatory approval and significantly improve the treatment landscape for DEB patients. Success would represent a landmark achievement for Castle Creek, its investors, and the field of gene therapy, demonstrating the potential of this approach to address rare genetic diseases. This also holds long-term financial implications for Ligand and strengthens its royalty portfolio.
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### [Coya Therapeutics in BTIG KOL Call on Alzheimer's & GLP-1](https://www.clinicaltrialvanguard.com/news/coya-therapeutics-in-btig-kol-call-on-alzheimers-glp-1/)
**Published:** February 26, 2025
**Author:** Jon Napitupulu
**Content:**
[Coya Therapeutics](https://www.clinicaltrialvanguard.com/news/coya-therapeutics-novel-patent-filing-expanding-pipeline-in-alzheimers-disease/) is advancing COYA 303, a combination therapy of low-dose interleukin-2 (COYA 301) and a GLP-1 receptor agonist, for inflammatory diseases. CEO Arun Swaminathan and Dr. Stanley H. Appel will discuss the potential of GLP-1 agonists and combination therapies for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/) in a webcast hosted by BTIG. This discussion will also cover Coya’s expanded pipeline and the development of COYA 303.
The exploration of GLP-1 agonists in combination with other therapies represents a novel approach to tackling Alzheimer’s disease, a condition with significant unmet medical need. The potential for COYA 303 to address inflammatory aspects of Alzheimer’s, in addition to its application in other inflammatory diseases, positions Coya at the forefront of this emerging therapeutic area. This approach could be a significant step towards developing more effective treatments for this debilitating disease and related conditions.
Coya’s lead product candidate, COYA 302, is a combination of low-dose IL-2 and CTLA4-Ig being developed for several neurodegenerative diseases, including Alzheimer’s, [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/), Amyotrophic Lateral Sclerosis, and Frontotemporal Dementia. The company’s focus on Treg cell biology offers a unique platform for developing therapies addressing the underlying inflammatory mechanisms of these diseases. The addition of COYA 303 to their pipeline expands the potential application of their technology to a broader range of inflammatory conditions.
Coya’s strategic focus on Treg-based therapies and the development of combination therapies like COYA 302 and COYA 303 could significantly alter the treatment landscape for neurodegenerative and inflammatory diseases. The upcoming webcast discussion will provide further insight into the potential of these therapeutic approaches and Coya’s role in advancing this field.
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### [FDA Accepts Regeneron Odronextamab Resubmission for Follicular Lymphoma Review](https://www.clinicaltrialvanguard.com/news/fda-accepts-regeneron-odronextamab-resubmission-for-follicular-lymphoma-review/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
Regeneron Pharmaceuticals announced that the FDA accepted its resubmitted [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) for odronextamab. The drug is intended to treat relapsed/refractory follicular lymphoma (FL) after patients have undergone at least two other systemic therapies. The FDA is expected to make a decision by July 30, 2025.
This resubmission is a critical step for Regeneron and potentially for patients battling this incurable cancer. The FDA’s acceptance signifies that Regeneron has successfully addressed the previous approvability issue related to patient enrollment in the confirmatory Phase 3 trial. A potential approval could provide a new treatment option for patients who have limited alternatives after multiple rounds of therapy, potentially improving their outcomes. It also strengthens Regeneron’s position in the hematology-oncology market.
The resubmitted BLA includes data from Phase 1 and pivotal Phase 2 trials, which showed an 80% overall response rate and a 74% complete response rate. However, serious adverse events were observed in 67% of patients, with cytokine release syndrome, COVID-19, and pneumonia occurring in a notable percentage. Odronextamab is already approved in the European Union for relapsed/refractory FL and diffuse [large B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/)[B-cell lymphoma](https://www.clinicaltrialvanguard.com/news/hutchmed-starts-phase-iii-trial-for-b-cell-lymphoma-in-china/), offering a glimpse into its potential market reach.
The FDA decision regarding odronextamab will be a defining moment. A positive outcome would mark a significant advancement in the treatment of relapsed/refractory FL and could lead to increased market share for Regeneron. Conversely, a rejection would likely delay the drug’s availability in the U.S. and potentially impact future development strategies. The outcome will undoubtedly influence the landscape of FL treatment and shape Regeneron’s trajectory in the hematology-oncology field.
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### [Lucid-21-302 Phase 1 Trial Complete: Quantum Biopharma's MS Drug Shows Promise](https://www.clinicaltrialvanguard.com/news/lucid-21-302-phase-1-trial-complete-quantum-biopharmas-ms-drug-shows-promise/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
Quantum BioPharma Ltd. announced the successful completion of a Phase 1 clinical trial for Lucid-MS, a novel neuroprotective compound for [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (MS). The safety review committee confirmed that Lucid-MS was well-tolerated by healthy participants, with no safety concerns or serious adverse events. This first-in-class compound is designed to stabilize the myelin sheath surrounding nerve fibers, offering a potential new treatment approach for MS.
This positive Phase 1 outcome is a crucial step forward in the fight against MS. Current MS treatments primarily focus on modulating the immune system, but Lucid-MS offers a different approach by directly targeting myelin protection. This mechanism of action could be particularly valuable for patients who do not respond well to immunomodulatory therapies or experience disease progression despite treatment. The successful safety profile in this initial human trial paves the way for further investigation into Lucid-MS’s potential to slow or even reverse the debilitating effects of MS.
The Phase 1 trial was a randomized, double-blind, placebo-controlled, multiple ascending dose study evaluating the safety and pharmacokinetics of Lucid-MS in healthy adults. The absence of safety concerns is a critical hurdle for any new drug candidate and allows Quantum BioPharma to move forward with planning a Phase 2 trial in people with MS. This next phase will focus on assessing the efficacy of Lucid-MS in a patient population and further defining its potential role in managing this chronic neurological condition.
The successful completion of the Phase 1 trial is a significant achievement for Quantum BioPharma and has positive implications for the MS drug development landscape. The advancement of Lucid-MS into Phase 2 clinical trials strengthens Quantum BioPharma’s position in the field of neuroprotective therapies. The data generated from the upcoming Phase 2 trial will be pivotal in determining the future development and potential regulatory approval of Lucid-MS, offering hope for a new treatment option for individuals living with MS.
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### [Pyx-201 Gets FDA Fast Track for Head & Neck Cancer](https://www.clinicaltrialvanguard.com/news/pyx-201-gets-fda-fast-track-for-head-neck-cancer/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
Pyxis Oncology’s PYX-201, an antibody-drug conjugate targeting Extradomain-B Fibronectin (EDB+FN), received Fast Track Designation from the FDA for treating recurrent or metastatic head and neck squamous cell carcinoma (R/M [HNSCC](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/)) in patients whose disease progressed after platinum-based chemotherapy and anti-PD-(L)1 antibody treatment. PYX-201 is the first ADC to target EDB+FN, a component of the tumor extracellular matrix highly expressed in various tumor types. This designation facilitates expedited development and review of PYX-201.
This FDA decision is crucial due to the high unmet need in R/M HNSCC. Nearly half of head and neck cancer cases become recurrent or metastatic, resulting in a median overall survival of less than a year after initial treatment. Furthermore, the incidence of HNSCC is projected to increase significantly by 2030, driven by factors including tobacco and alcohol use, HPV infections, and environmental factors. The Fast Track Designation for PYX-201 offers hope for improved outcomes in this difficult-to-treat cancer.
PYX-201 is currently being evaluated in two clinical trials. The PYX-201-101 trial is investigating PYX-201 as a monotherapy in R/M HNSCC patients. The PYX-201-102 trial is assessing PYX-201 in combination with [pembrolizumab](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/) (Keytruda) in patients with R/M HNSCC and other advanced solid tumors. This combination trial is part of a collaboration with Merck.
This Fast Track Designation expedites PYX-201’s development, potentially leading to quicker access for patients facing a challenging prognosis with R/M HNSCC. The ongoing clinical trials will provide crucial data on PYX-201’s efficacy and safety, potentially shaping the treatment landscape for this aggressive form of cancer. Positive results could establish PYX-201 as a valuable therapeutic option, particularly for patients who have progressed after standard treatments. This progress may also stimulate further research into targeting the tumor microenvironment, particularly EDB+FN, as a therapeutic strategy for various cancers.
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---
### [PDS Biotech's HPV Cancer Data in JAMA Oncology: Positive Survival, Clinical Responses](https://www.clinicaltrialvanguard.com/news/pds-biotechs-hpv-cancer-data-in-jama-oncology-positive-survival-clinical-responses/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
PDS Biotechnology Corporation announced promising clinical trial results for a triple combination therapy targeting HPV-associated cancers, published in JAMA Oncology. The therapy combines Versamune® HPV, PDS01ADC (an [IL-12](https://www.clinicaltrialvanguard.com/news/son-1010-shows-strong-safety-in-ovarian-cancer-treatment/) fused antibody-drug conjugate), and a PD-L1 immune checkpoint inhibitor. The trial involved patients with recurrent or metastatic HPV-positive anal, cervical, head and neck, penile, vaginal, and vulvar cancers.
These findings are potentially transformative for the treatment of HPV-associated cancers, particularly for recurrent or metastatic forms where current treatment options are limited and prognoses are often poor. The demonstrated efficacy in [HPV16](https://www.clinicaltrialvanguard.com/news/pds-biotech-amends-phase-3-trial-for-accelerated-approval/)-positive patients is especially noteworthy given the prevalence of this HPV type in several cancers, including a growing number of head and neck cancers. This opens the door to significantly improved outcomes for patients with these challenging cancers.
In patients who had not received prior checkpoint inhibitors, the overall response rate was 35.7%, with a notable 75% in HPV16-positive patients. Median overall survival for this group reached 42.4 months, while survival for HPV16-positive patients was not yet reached, substantially exceeding historical data. For patients with prior checkpoint inhibitor treatment, the overall response rate was 16.7%, increasing to 62.5% in HPV16-positive patients receiving the higher dose of PDS01ADC. Median overall survival in this group was 15.8 months (17 months for HPV16-positive), again, outperforming historical results. While grade 3 and 4 adverse events were observed in 52% of patients, these were correlated with the dose of PDS01ADC and the PD-L1 inhibitor, with no treatment-related deaths reported.
The positive data from this study reinforces the potential of Versamune® HPV as a targeted immunotherapy, particularly when combined with PDS01ADC. These results support [PDS Biotech](https://www.clinicaltrialvanguard.com/news/pds-biotech-to-present-new-cancer-therapy-data-at-sitc-2025/)’s upcoming Phase 3 trial of Versamune® HPV combined with pembrolizumab for recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma. This research may pave the way for new and more effective treatment options for HPV-related cancers, offering hope for improved survival and quality of life for patients.
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---
### [First Participant Dosed in Phase 1a Study of Oral HDV Entry Inhibitor](https://www.clinicaltrialvanguard.com/news/first-participant-dosed-in-phase-1a-study-of-oral-hdv-entry-inhibitor/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
[Assembly Biosciences](https://www.clinicaltrialvanguard.com/news/preclinical-data-on-assembly-biosciences-hbv-and-hdv-inhibitors-at-easl-2025/) has dosed the first participant in a Phase 1a clinical trial for ABI-6250, an oral HDV entry inhibitor. The trial will assess the safety, tolerability, pharmacokinetics, and serum bile acid levels (a biomarker for target engagement) of ABI-6250 in healthy individuals. Data from this trial is anticipated in Q3 2025.
This clinical trial initiation is a crucial step forward in addressing the unmet need for effective HDV treatments. Currently, only one therapy, requiring daily injections, is approved for chronic HDV (cHDV) in the European Union and none in the United States. An easily administered oral therapy like ABI-6250 could significantly improve patient compliance and quality of life, potentially transforming the cHDV treatment landscape. Given the severity of cHDV, with a high rate of cirrhosis progression, a new therapeutic option offers significant hope.
The Phase 1a trial (ABI-6250-101) is a randomized, placebo-controlled study evaluating single and multiple ascending doses of ABI-6250 in healthy participants. The multiple-dose cohorts will involve repeat dosing over 10 days. Preclinical studies have shown promising results, including low nanomolar potency across multiple HDV genotypes, selectivity for its target (NTCP), and a pharmacokinetic profile suggesting the feasibility of once-daily oral dosing. The trial will help determine the appropriate dose for future clinical studies.
The initiation of this Phase 1a trial represents a significant milestone for Assembly Biosciences and for the development of a potentially transformative HDV treatment. Positive results from this trial could pave the way for further clinical development and ultimately offer a much-needed oral therapeutic option for patients with cHDV. The data expected in Q3 2025 will be eagerly awaited by the medical community and patients alike.
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---
### [Sonnet BioTherapeutics Son-1010: Improved Solid Tumor Treatment](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
Sonnet BioTherapeutics presented data at the 2025 AACR:IO Conference on its Fully Human Albumin Binding (FHAB) platform, which enhances tumor targeting and retention of immunomodulators. The platform links mono- or bifunctional immunomodulators to an albumin-binding domain, improving the pharmacokinetic profile and reducing toxicity risk. The presentation focused on [SON-1010](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-expands-ovarian-cancer-dose-escalation-trial/), a rhIL-12 configured with FHAB, and its application in various combination therapies.
This research holds promise for advancing immunotherapy in oncology. The FHAB platform addresses key challenges associated with recombinant interleukins, such as short half-lives and off-target effects, which have historically limited their clinical success. By improving tumor-specific delivery and reducing systemic toxicity, the platform could unlock the therapeutic potential of potent immunomodulators like [IL-12](https://www.clinicaltrialvanguard.com/news/son-1010-shows-strong-safety-in-ovarian-cancer-treatment/), leading to more effective and safer treatments for various cancers.
The presented data highlighted the platform’s ability to increase the half-life and bioactivity of IL-12. This leads to a broader therapeutic index, increased activation of key immune cells (NK, NKT, Th1, and cytotoxic CD8 T cells), and repolarization of immunosuppressive cells within the tumor microenvironment. SON-1010 is currently being investigated in several clinical settings: as a monotherapy, in combination with a checkpoint inhibitor ([atezolizumab](https://www.clinicaltrialvanguard.com/news/xilio-announces-updated-phase-2-data-for-vilastobart/)), in alternation with immunoreactive chemotherapy (trabectedin), and with a chemotherapeutic regimen (NALIRIFOX) for pancreatic ductal adenocarcinoma. Early clinical data from a trial combining SON-1010 with atezolizumab in platinum-resistant ovarian cancer shows a 48% clinical benefit rate, including a partial response in a patient with clear cell sarcoma. Further trials, including one investigating SON-1210 (IL12-FHAB-IL15) in combination with NALIRIFOX for metastatic pancreatic cancer, are planned.
The FHAB platform represents a significant advancement in targeted immunotherapy. The positive early clinical data and the ongoing trials suggest the potential for improved outcomes in patients with various difficult-to-treat cancers. If successful, the platform could usher in a new era of cytokine-based therapies, offering more effective and less toxic treatment options. The continued development of the FHAB platform and the exploration of its application with various immunomodulators and combination therapies will be critical to realizing its full potential.
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---
### [Derm-Biome's DB-007-5 Highly Effective in Atopic Dermatitis Study](https://www.clinicaltrialvanguard.com/news/derm-biomes-db-007-5-highly-effective-in-atopic-dermatitis-study/)
**Published:** February 27, 2025
**Author:** Jon Napitupulu
**Content:**
Derm-Biome Pharmaceuticals announced positive preclinical results for its topical drug DB-007-5 in treating [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (AD). The drug significantly reduced inflammation and outperformed the current leading AD treatment, Opzelura, in reducing itch, a key symptom of AD. DB-007-5 also demonstrated a favorable safety profile in previous studies.
This development holds substantial promise for patients suffering from AD, a chronic inflammatory skin disease affecting a significant portion of the population, particularly children. Current treatment options often provide inadequate relief or are associated with side effects, highlighting a need for safer and more effective therapies, especially for mild to moderate cases. The positive results for DB-007-5 suggest it could address this unmet need, potentially offering a new first-line treatment option and improving the quality of life for millions.
DB-007-5 at a 0.75% concentration was more effective at reducing itch than Opzelura (1.5% [ruxolitinib](https://www.clinicaltrialvanguard.com/news/karyopharm-to-submit-selinexor-ruxolitinib-snda-for-myelofibrosis-in-august-2026/) cream). Prior studies also showed DB-007-5’s superior efficacy compared to Pfizer’s Eucrisa. These findings position DB-007-5 as a potential best-in-class topical treatment for AD.
The successful preclinical results for DB-007-5 pave the way for further clinical development. If subsequent trials confirm these positive findings, Derm-Biome could significantly impact the AD treatment landscape, offering a new, effective, and safe topical option for patients. This could also lead to substantial market share capture within the growing AD therapeutics market.
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---
### [DM Clinical Research Data Breach Exposes Over 1.6M Records, Raising Patient Privacy Concerns](https://www.clinicaltrialvanguard.com/news/dm-clinical-research-data-breach-exposes-over-1-6m-records-raising-patient-privacy-concerns/)
**Published:** February 27, 2025
**Author:** Moe Alsumidaie
**Content:**
DM Clinical Research, a Texas-based clinical trial site network, has suffered a major data breach, exposing over 1.6 million personal and medical records. The unprotected database, discovered by cybersecurity researcher Jeremiah Fowler, was publicly accessible without password protection or encryption. The breach involved 1,674,218 records, totaling 2 terabytes of data.
### [](#details-of-the-exposure)**Details of the Exposure**
The leaked records, primarily in PDF format, contained personally identifiable information (PII) such as names, dates of birth, and contact details. The documents also included medical histories, vaccination statuses, medication details, and notes on adverse reactions. Some records referenced participation in [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) vaccine trials and contained sensitive health information related to [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) and sexual history.
### [](#dm-clinical-researchs-response)**DM Clinical Research’s Response**
Upon being alerted to the exposure, DM Clinical Research restricted public access to the database within hours. The company said, *“Our team is currently reviewing the details of your findings to ensure a swift and comprehensive resolution. Protecting sensitive data is a cornerstone of our organization’s operations, and we are committed to addressing any vulnerabilities in alignment with best practices and applicable laws & regulations.”*
It remains unclear how long the data was exposed, whether unauthorized individuals accessed it, or whether a third-party vendor managed the database.
### [](#industry-and-patient-implications)**Industry and Patient Implications**
The breach raises concerns about data security in clinical trials, where the protection of patient information is a regulatory requirement. The exposure of sensitive health data could have implications for patient privacy, regulatory oversight, and cybersecurity protocols within the clinical trial industry.
Regulatory agencies, including the FDA and EMA, enforce strict data protection measures for clinical trials. Incidents such as this could increase scrutiny of security protocols at research sites. Data security remains a key consideration as clinical trials adopt digital platforms and decentralized models.
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**Categories:** News
---
### [Lexicon Announces Pilavapan Results for Diabetic Nerve Pain](https://www.clinicaltrialvanguard.com/news/lexicon-announces-pilavapan-results-for-diabetic-nerve-pain/)
**Published:** March 3, 2025
**Author:** Jon Napitupulu
**Content:**
Lexicon Pharmaceuticals will announce topline results from its Phase 2b PROGRESS study of pilavapadin (LX9211) for diabetic peripheral neuropathic pain (DPNP) on March 3, 2025. The study evaluated the efficacy and safety of this oral, non-opioid, AAK1 inhibitor in adults with moderate to severe DPNP. The company hopes this novel approach to pain management will offer a new treatment option for this prevalent condition.
Positive results from the PROGRESS study could significantly impact the treatment landscape for DPNP. Current treatment options often provide inadequate pain relief or carry the risk of opioid dependence. Pilavapadin’s unique mechanism of action, targeting AAK1 to inhibit neurotransmitter reuptake without affecting opioid pathways, offers a potentially safer and more effective alternative. This could lead to improved quality of life for millions of patients suffering from this chronic and debilitating condition.
The PROGRESS study enrolled 496 patients with type 1 or [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/) and moderate to severe DPNP. The study used a placebo-controlled, dose-ranging design, evaluating three pilavapadin doses (10mg, 20mg, and 20mg followed by 10mg). The primary endpoint was the change in average daily pain score (ADPS) from baseline to Week 8. Importantly, the study allowed patients to continue their existing stable-dose DPNP medications, reflecting real-world treatment scenarios.
The upcoming announcement of the topline results will be a pivotal moment for Lexicon. Positive data could pave the way for a Phase 3 trial and eventual regulatory approval, potentially establishing pilavapadin as a valuable new treatment option for DPNP. This would validate Lexicon’s unique genomics-based drug discovery platform and strengthen its position in the pain management market. Furthermore, the novel mechanism of pilavapadin may stimulate further research into AAK1 inhibition for other neuropathic pain conditions.
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---
### [Apg990 Interim Phase 1 Results Show Potential for Maintenance Dosing](https://www.clinicaltrialvanguard.com/news/apg990-interim-phase-1-results-show-potential-for-maintenance-dosing/)
**Published:** March 3, 2025
**Author:** Jon Napitupulu
**Content:**
Apogee Therapeutics announced positive interim Phase 1 results for APG990, a novel, half-life extended [OX40L](https://www.clinicaltrialvanguard.com/news/ox40-targeted-therapy-trials-market-opportunity-insight-2026/) antibody, demonstrating an approximately 60-day half-life. This achievement paves the way for a Phase 1b head-to-head study of APG279 (a combination of [APG777](https://www.clinicaltrialvanguard.com/news/apogee-reveals-new-apg777-atopic-dermatitis-phase-2-data/) and APG990) against Dupixent, planned for later this year with results expected in the second half of 2026. Preclinical toxicology studies of the combination therapy and the positive APG990 results support the advancement of this program.
This news is potentially transformative for the treatment of [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) (AD) and other inflammatory and immunology (I&I) conditions. The extended half-life of APG990 suggests the possibility of infrequent dosing—every three or six months—which could significantly improve patient adherence and convenience. The combination therapy, APG279, aims to address the complex nature of AD by inhibiting multiple inflammatory pathways (Types 1, 2, and 3), potentially offering superior efficacy compared to existing therapies that primarily target the Type 2 pathway.
Interim Phase 1 pharmacokinetic data revealed a 60-day half-life for APG990 across all tested doses. This supports the potential for infrequent dosing with a small injection volume (2 mL) for the combined APG279 therapy. APG990 was well-tolerated in the Phase 1 trial, with no serious adverse events reported. Preclinical combination studies of APG777 and APG990 showed no safety concerns and indicated a potential for enhanced efficacy compared to the individual agents.
The extended half-life, favorable tolerability profile, and potential for enhanced efficacy of APG990 and the APG279 combination position Apogee Therapeutics to become a leader in the AD treatment landscape. The upcoming head-to-head trial against Dupixent will be crucial in determining the clinical benefit of this novel approach and could reshape the treatment paradigm for AD and potentially other I&I diseases.
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---
### [Deucrictibant Data at AAAAI/WAO 2025: HAE Prevention & Treatment](https://www.clinicaltrialvanguard.com/news/deucravacitinib-data-at-aaaai-wao-2025-hae-prevention-treatment/)
**Published:** March 3, 2025
**Author:** Jon Napitupulu
**Content:**
Pharvaris released positive long-term data for its oral bradykinin B2 receptor antagonist, deucrictibant, at the 2025 [AAAAI](https://www.clinicaltrialvanguard.com/news/cogent-highlights-bezuclastinib-summit-trial-data-at-aaaai/)/WAO Joint Congress. The data highlighted sustained efficacy and safety in both prophylactic and on-demand treatment of Hereditary Angioedema (HAE) from extension studies of previous Phase 2 trials. The findings demonstrate the potential of deucrictibant to address unmet needs in the HAE community.
These findings are important for HAE patients, who often experience debilitating and unpredictable swelling attacks. Current treatment options can be inconvenient, requiring injections or infusions. A safe and effective oral therapy like deucrictibant could significantly improve patients’ quality of life by offering convenient, at-home treatment and prevention, reducing the fear and limitations imposed by HAE. The observed improvements in quality-of-life metrics related to functioning and fear/shame underscore the potential impact of deucrictibant on patients’ daily lives.
The CHAPTER-1 open-label extension study showed participants maintained a reduced monthly HAE attack rate for at least 1.5 years with a median proportion of symptom days at zero. All participants in the CHAPTER-1 OLE who had reached week 62 reported improved health-related quality of life. In the RAPIDe-2 extension study, data from seven upper airway attacks, including laryngeal attacks, revealed a median time to symptom relief of 0.9 hours. Both extension studies confirmed the drug’s tolerability with no new safety signals.
The positive long-term data strengthens Pharvaris’ position in developing an oral HAE therapy. The results support the ongoing Phase 3 trials for both prophylactic and on-demand deucrictibant and increase the likelihood of a successful regulatory submission. If approved, deucrictibant could offer a valuable new treatment option for HAE patients, potentially shifting the treatment paradigm towards oral therapies and improving disease management.
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---
### [Voyager Reports Positive Topline Anti-Tau Antibody Data, Initiates Next Trial](https://www.clinicaltrialvanguard.com/news/voyager-reports-positive-topline-anti-tau-antibody-data-initiates-next-trial/)
**Published:** March 3, 2025
**Author:** Jon Napitupulu
**Content:**
Voyager Therapeutics announced positive topline data from its single ascending dose (SAD) trial of VY7523, an investigational anti-tau antibody for [Alzheimer’s disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) disease. The data showed VY7523 was safe, tolerable, and exhibited dose-proportional pharmacokinetics in healthy volunteers. Based on these results, Voyager has initiated a multiple ascending dose (MAD) trial in early Alzheimer’s patients, with initial tau PET imaging data anticipated in the second half of 2026.
This development holds promise for Alzheimer’s treatment as it advances a new therapeutic approach targeting tau, a protein implicated in disease progression. The positive SAD results, particularly the safety and tolerability profile combined with dose-proportional pharmacokinetics, suggest VY7523 could be a leading candidate in the anti-tau antibody landscape. This is further bolstered by preclinical data demonstrating a significant reduction in tau spread. Given the limited effective treatment options currently available for Alzheimer’s, any progress toward a disease-modifying therapy represents a crucial step forward for both patients and the healthcare system.
The SAD trial involved 48 healthy volunteers and evaluated six ascending doses of intravenously administered VY7523. No serious or severe adverse events, or infusion reactions, were reported. Importantly, the cerebrospinal fluid (CSF)-to-serum ratio observed aligns with other approved monoclonal antibodies for Alzheimer’s, suggesting effective brain penetration. The subsequent MAD trial will enroll 52 early Alzheimer’s patients and focus on safety, tolerability, and the ability of VY7523 to impede the spread of pathological tau, measured by tau PET imaging.
The positive SAD data for VY7523 reinforces the potential of targeting tau in Alzheimer’s disease. The upcoming MAD trial results and further preclinical investigation will be critical in validating this approach. If successful, VY7523 could represent a significant advancement in Alzheimer’s treatment, potentially offering a disease-modifying therapy to address this substantial unmet medical need. The progress of VY7523 warrants close attention as it moves through clinical development.
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---
### [Aura Biosciences NMIBC Data at EAU Congress](https://www.clinicaltrialvanguard.com/news/aura-biosciences-nmibc-data-at-eau-congress/)
**Published:** March 3, 2025
**Author:** Jon Napitupulu
**Content:**
Aura Biosciences announced Phase 1 data from its trial of bel-sar (AU-011) for non-muscle invasive [bladder cancer](https://www.clinicaltrialvanguard.com/news/summit-therapeutics-launches-harmoni-gu1-trial-for-ivonescimab-in-bladder-cancer/) ([NMIBC](https://www.clinicaltrialvanguard.com/news/urogens-ugn-103-shows-94-5-six-month-response-in-nmibc-trial/)) will be presented at the 40th Annual European Association of Urology (EAU) Congress, March 21-24, 2025. The company will also participate in the EAU Research Forum and host a virtual investor event on March 24th, featuring key opinion leaders discussing the trial data and future development plans for bel-sar. The Phase 1 trial is a window-of-opportunity study evaluating bel-sar as a monotherapy prior to the standard transurethral resection of bladder tumor (TURBT) surgery.
These developments are critical for the NMIBC treatment landscape. Current treatments for NMIBC often lead to recurrence and can necessitate radical cystectomy (bladder removal), significantly impacting patient quality of life. Bel-sar, a virus-like drug conjugate, offers a potential new approach with its dual mechanism of action: directly targeting cancer cells and stimulating an immune response. The presentation of Phase 1 data at a major international conference like the EAU Congress provides crucial validation and visibility, attracting potential collaborators and investors. The data’s release, along with the subsequent discussions during the investor event and research forum, could accelerate the development and adoption of this novel therapy.
The Phase 1 trial is designed to assess the safety and feasibility of bel-sar administration and evaluate its biological activity, including its impact on the immune system. The upcoming EAU presentation, delivered by Dr. Seth Lerner, will focus on the safety and efficacy findings from this trial. The research forum will feature experts discussing the mechanism, initial findings, and future directions of virus-like drug conjugates as a novel bladder cancer treatment. The investor event will include presentations from key opinion leaders like Dr. Neal Shore, Dr. Gary Steinberg, and Dr. Jennifer Linehan, providing further insights into the clinical data and development strategy.
This confluence of data presentation, expert discussion, and corporate updates signals a potentially pivotal moment for Aura Biosciences and bel-sar’s development. Positive data and expert endorsement could significantly advance bel-sar toward later-stage clinical trials and eventual regulatory approval, potentially offering a new, less invasive treatment option for NMIBC patients. The planned expansion into a Phase 1b/2 trial, along with further investigation of the drug’s dual mechanism of action, promises to refine treatment protocols and potentially improve patient outcomes. This could lead to a shift in the NMIBC treatment paradigm, preserving bladder function and improving patients’ lives.
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---
### [Transforming Oncology with Real World Data: Insights from COTA's Miruna Sasu](https://www.clinicaltrialvanguard.com/executiveinterviews/transforming-oncology-with-real-world-data-insights-from-cotas-miruna-sasu/)
**Published:** March 3, 2025
**Author:** Moe Alsumidaie
**Content:**
In this conversation with Miruna Sasu, President and CEO of COTA Healthcare, we explore the transformative power of real world data (RWD) in oncology. With a background spanning pharmaceutical giants and [health tech](https://www.clinicaltrialvanguard.com/news/allez-healths-60-million-capital-raise-a-monumental-breakthrough-in-health-tech/) innovators, Sasu shares her vision for leveraging RWD to accelerate drug development and enhance patient outcomes. This interview delves into COTA’s cutting-edge methodologies, strategic partnerships, and AI and machine learning integration in healthcare.
## [](#moe-cotas-mission-is-to-create-clarity-from-fragmented-rwd-can-you-elaborate-on-the-technologies-and-methodologies-you-use-to-analyze-trial-data-effectively)**Moe: COTA’s mission is to create clarity from fragmented RWD. Can you elaborate on the technologies and methodologies you use to analyze trial data effectively?**
At COTA, we provide RWD in datasets and analytics services as a solutions partner. We extract electronic medical records data, de-identify it, and clean it for research. This ensures data integrity and usability. Our proprietary platform uses AI and machine learning to extract data from structured sources like dropdown menus and unstructured sources like doctors’ notes. These notes often contain critical information about disease stages and treatment paradigms. After AI surfaces the relevant data, medical professionals review it for accuracy. This process allows us to answer complex questions about treatment regimens and patient outcomes, driving better healthcare decisions.
Miruna Sasu, President and CEO of COTA Healthcare
## [](#moe-you-partnered-with-guardant-health-to-advance-precision-oncology-research-what-outcomes-do-you-expect-and-how-will-this-impact-cancer-care)**Moe: You partnered with Guardant Health to advance precision oncology research. What outcomes do you expect, and how will this impact cancer care?**
Our collaboration with Guardant Health exemplifies how partnerships enhance data depth. Integrating electronic medical record data with lab results, like DNA and RNA sequencing, gives us a comprehensive view of a patient’s status and treatment response. This integration is crucial in oncology, where understanding the genetic makeup of tumors leads to more precise and personalized treatment options. The expected outcome is a holistic understanding of cancer patients, significantly improving treatment efficacy and patient outcomes. This collaboration shows how combining different data sources can lead to breakthroughs in precision medicine, ultimately transforming cancer care.
## [](#moe-with-your-experience-in-pharma-and-health-tech-what-synergies-do-you-see-in-leveraging-rwd-to-accelerate-drug-development-and-improve-patient-outcomes)**Moe: With your experience in pharma and health tech, what synergies do you see in leveraging RWD to accelerate drug development and improve patient outcomes?**
My experience in both sectors highlights RWD’s transformative potential in clinical trials. At J&J, I focused on increasing diversity and improving enrollment efficiency. RWD can identify suitable patients and sites, reducing time and costs. Instead of opening trial sites blindly, we use RWD to pinpoint locations with eligible patients, ensuring trials are conducted where needed. Integrating electronic medical records directly into trial systems eliminates redundant data entry, speeding up the process and allowing patients to access potentially life-saving treatments sooner. This synergy between pharma and health tech is crucial for advancing drug development and improving patient care.
## [](#moe-how-do-external-control-arms-using-rwd-enhance-clinical-researchs-efficiency-and-ethical-considerations)**Moe: How do external control arms using RWD enhance clinical research’s efficiency and ethical considerations?**
External control arms are a game-changer in clinical research. At Bristol-Myers Squibb, we aimed to incorporate RWD into every submission, despite initial resistance from regulatory bodies like the FDA. Today, the landscape has evolved, and COTA is involved in numerous external control arm projects annually. They reduce the need for traditional control groups, allowing more patients to receive experimental treatments. This approach accelerates drug approvals and addresses ethical concerns by minimizing the number of patients in the control group. Our work has helped several drugs reach the market faster, which is vital for patients with life-threatening conditions who cannot afford to wait.
## [](#moe-with-ai-and-ml-adoption-in-healthcare-how-does-cota-ensure-these-technologies-maintain-patient-privacy-and-data-security)**Moe: With AI and ML adoption in healthcare, how does COTA ensure these technologies maintain patient privacy and data security?**
Privacy and security are paramount at COTA. We partner with large, secure platforms like Google Cloud to safeguard our deidentified data. We also work with 3rd party privacy experts to ensure our data is not at risk of reidentification and conduct regular audits to ensure compliance. AI is crucial in enhancing security by generating insights without data leaving our secure environment. Our AI assistant, CAILIN, facilitates this process by providing insights directly from our datasets, reducing the need for data transfer. This approach safeguards patient information and streamlines the research process, making it more efficient and secure. Maintaining rigorous security measures ensures that patient privacy is always protected while leveraging AI’s full potential.
## [](#moe-reflecting-on-your-journey-how-have-your-experiences-shaped-your-vision-for-the-future-of-rwd-in-oncology)**Moe: Reflecting on your journey, how have your experiences shaped your vision for the future of RWD in oncology?**
My journey has reinforced the importance of using AI as a workhorse for data cleaning and preparation, often the most time-consuming aspects of data analysis. By automating these processes, we can focus more on deriving insights and improving patient care. We’ve seen significant productivity gains at COTA by leveraging AI in these areas. For example, our AI-driven data abstraction has allowed us to reallocate 30% of our staff to more engaging tasks, enhancing overall efficiency. This trend will continue to revolutionize how we conduct clinical trials and research, leading to faster and more effective patient treatments.
**Categories:** Article: Executive Interviews
---
### [Oryzon Defines Phase III Trial Endpoints for Agitation & Aggression in BPD](https://www.clinicaltrialvanguard.com/news/oryzon-defines-phase-iii-trial-endpoints-for-agitation-aggression-in-bpd/)
**Published:** March 4, 2025
**Author:** Jon Napitupulu
**Content:**
Oryzon Genomics has established primary and key secondary endpoints for its Phase III clinical trial of vafidemstat for Borderline Personality Disorder (BPD). This was achieved with input from a newly formed Clinical Advisory Board comprised of leading psychiatric experts. The company anticipates submitting the Phase III protocol to the FDA in the first half of 2025.
This development is crucial because it signifies a concrete step towards addressing the unmet need for effective BPD treatments, especially for managing agitation and aggression. The involvement of a high-profile advisory board lends credibility to the trial design and increases confidence in the potential of vafidemstat to meaningfully impact patient outcomes. A clearly defined and FDA-aligned endpoint strategy enhances the likelihood of regulatory success, paving the way for a potential new therapy in a challenging therapeutic area.
The Phase III trial design builds upon positive feedback from the FDA regarding Oryzon’s Phase IIb PORTICO trial. The chosen endpoints, focused on agitation and aggression, utilize established assessment scales and directly address key symptoms that significantly impair the lives of individuals with BPD. This focus reflects a growing understanding of the importance of targeting these specific symptoms for improved patient management and quality of life.
This progress positions Oryzon to potentially initiate the Phase III trial soon after regulatory submission, pending funding acquisition. A successful Phase III trial could lead to the first approved treatment specifically targeting agitation and aggression in BPD, offering a much-needed option for patients and potentially reshaping the treatment landscape for this complex disorder. Furthermore, the research with vafidemstat in BPD could also inform the development of the drug for other conditions, including [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/) and various neurodevelopmental disorders, where it is currently being studied.
Source link:
**Categories:** News
---
### [NKgen Biotech Administers First Troculeucel Dose to Stroke Patient](https://www.clinicaltrialvanguard.com/news/nkgen-biotech-administers-first-troculeucel-dose-to-stroke-patient/)
**Published:** March 4, 2025
**Author:** Jon Napitupulu
**Content:**
NKGen Biotech administered its expanded autologous NK [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/), [troculeucel](https://www.clinicaltrialvanguard.com/news/nkgen-reveals-hope-for-neurodegenerative-disease-at-china-forum/), to a stroke patient under an FDA-cleared compassionate use IND. This marks the first step towards a potential full IND application for troculeucel in the post-stroke setting, building on NKGen’s research into neuroinflammation in stroke and traumatic brain injury. The collaboration with George Washington University Medical Center will involve patient infusions and independent assessments to evaluate the therapy’s efficacy.
This development holds particular importance because stroke, a leading cause of death and disability, often leads to chronic neuroinflammation and increased risk of dementia. Current treatment options for post-stroke neuroinflammation are limited, making the exploration of novel therapies like troculeucel crucial for improving long-term patient outcomes and potentially mitigating the debilitating effects of stroke. This move also signals NKGen’s commitment to broadening the therapeutic applications of its NK cell platform, potentially addressing a significant unmet medical need.
Troculeucel, previously studied in [Alzheimer](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/)’s trials, demonstrated an ability to cross the blood-brain barrier and reduce markers of brain injury. This suggests its potential to address the chronic neuroinflammation seen in post-stroke patients. This compassionate use case will provide valuable preliminary data regarding troculeucel’s safety and potential efficacy in this new patient population, informing future clinical development strategies.
This initial treatment represents a critical step towards evaluating the potential of troculeucel to address a substantial unmet need in stroke recovery. Positive outcomes from this case and subsequent studies could lead to a new therapeutic approach for managing post-stroke neuroinflammation, significantly impacting the lives of stroke survivors and potentially reshaping the post-stroke treatment landscape.
Source link:
**Categories:** News
---
### [Adverum Launches Phase 3 Study of Ixo-Vec for Wet AMD](https://www.clinicaltrialvanguard.com/news/adverum-launches-phase-3-study-of-ixo-vec-for-wet-amd/)
**Published:** March 4, 2025
**Author:** Jon Napitupulu
**Content:**
Adverum Biotechnologies has launched the [ARTEMIS](https://www.clinicaltrialvanguard.com/news/adverum-finishes-screening-for-pivotal-artemis-wet-amd-trial/) Phase 3 study, a pivotal trial evaluating the efficacy and safety of a single intravitreal injection of Ixo-vec (ixoberogene soroparvovec) for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (AMD). The study compares Ixo-vec to the current standard of care, aflibercept administered every eight weeks, in approximately 284 patients, encompassing both treatment-naïve and previously treated individuals. The primary endpoint is non-inferiority in best corrected visual acuity at one year.
This trial holds substantial implications for the treatment landscape of wet AMD. Current treatments require frequent injections, placing a considerable burden on patients and healthcare systems. A successful outcome for Ixo-vec could offer a significant improvement in patient care by reducing the need for ongoing injections, potentially improving long-term vision outcomes and adherence to treatment. This would be a major advancement for a disease projected to impact millions more people globally by 2040.
ARTEMIS is the first of two planned Phase 3 registrational trials for Ixo-vec in wet AMD. The study is designed as a randomized, double-masked, sham-controlled trial including both treatment-naïve and previously treated patients. This inclusive design aims to generate data representative of real-world patient demographics and address the needs of a broad patient population, including those with the highest treatment burden. All participants will receive three loading doses of aflibercept before administration of Ixo-vec or sham treatment. Supplemental aflibercept injections will be available for all patients, along with prophylactic steroid eye drops.
The initiation of the ARTEMIS trial represents a crucial step toward potentially establishing Ixo-vec as a one-time treatment for wet AMD. Positive results could significantly disrupt the current treatment paradigm, offering patients a more convenient and potentially more effective long-term solution. This advancement could lead to better vision outcomes, improved quality of life for patients, and a reduced burden on healthcare providers. The results of ARTEMIS will be highly anticipated, as they could herald a new era in the management of this prevalent and debilitating eye disease.
Source link:
**Categories:** News
---
### [Onward Medical Advances Parkinson's Pipeline with DoD, MJFF Support](https://www.clinicaltrialvanguard.com/news/onward-medical-advances-parkinsons-pipeline-with-dod-mjff-support/)
**Published:** March 4, 2025
**Author:** Jon Napitupulu
**Content:**
ONWARD Medical received two grants to support early clinical feasibility studies of its ONWARD ARC-IM System for [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease. The Michael J. Fox Foundation awarded a $1 million grant for a study on mobility challenges, while the US Department of Defense granted approximately $1.5 million to study blood pressure instability. Both studies aim to expand the application of the ARC-IM System, currently developed for spinal cord injury, to Parkinson’s disease.
These studies are crucial because they address significant unmet needs in Parkinson’s disease. Mobility issues and blood pressure instability affect a large percentage of Parkinson’s patients, significantly impacting their quality of life and independence. Current treatments often fail to adequately address these issues, highlighting the importance of exploring new therapeutic avenues like the ARC-IM System. Positive results could revolutionize Parkinson’s treatment, potentially offering much-needed relief for debilitating symptoms.
The MJFF-funded study, already underway, involves six participants and investigates the ARC-IM System’s potential to improve mobility and balance. This study builds upon previous research published in •Nature Medicine• demonstrating the system’s positive impact on these symptoms. The Department of Defense-funded study, expected to begin in the first half of 2025, will enroll five participants to examine the system’s effect on blood pressure instability, a problem affecting approximately 800,000 individuals in the US and Europe.
Positive outcomes from these studies could significantly advance Parkinson’s disease treatment. Successful results would validate the broader applicability of the ARC-IM System, opening doors to a larger patient population and strengthening ONWARD Medical’s position in the neuromodulation field. This could also lead to further research and development of targeted therapies for other neurological conditions, ultimately transforming the lives of millions.
Source link:
**Categories:** News
---
### [Transforming Clinical Trials: Medable's Approach to Diversity and Inclusion](https://www.clinicaltrialvanguard.com/executiveinterviews/transforming-clinical-trials-medables-approach-to-diversity-and-inclusion/)
**Published:** March 4, 2025
**Author:** Moe Alsumidaie
**Content:**
In this interview, we explore clinical trial technology’s impact on diversity and inclusion. Ali Holland from Medable shares insights on how digital trials enhance diverse participation. This discussion explores Medable’s strategies for engaging underrepresented populations, the role of digital solutions in patient engagement, and the future of decentralized clinical trials (DCTs) in addressing healthcare disparities.
## [](#moe-despite-the-fda-diversity-plans-disappearance-how-does-medable-keep-underrepresented-groups-engaged-in-trials)Moe: Despite the FDA diversity plan’s disappearance, how does Medable keep underrepresented groups engaged in trials?
In my view, diversity and inclusion are essential to clinical trial integrity. At Medable, we focus on improving accessibility and awareness among underrepresented populations. Traditional informed consent forms can be lengthy and complex, creating barriers to participation. We make information more digestible and engaging by transforming these forms into digital experiences with infographics and diagrams. This approach has been validated through our collaboration with Duke University, where enhanced consenting processes improved patient satisfaction and increased retention rates. Retaining participants is crucial for ensuring data completeness, which is vital for the success of any clinical trial.
## [](#moe-which-dct-components-improve-diversity-and-how-can-we-better-engage-black-or-african-american-communities)Moe: Which DCT components improve diversity, and how can we better engage Black or African American communities?
Engaging diverse communities requires a multifaceted approach. Trust and transparency are key, especially in communities historically underserved in clinical research. We leverage familiar technologies, like smartphones, to make participation more accessible. For those without access, we provide devices to eliminate barriers. An interesting finding from our data is that allowing participants to engage with trials through local, familiar settings, like their neighborhood pharmacy, significantly increases participation. For instance, we observed a 38% increase in participation among Black or African American communities when trials included local elements. This suggests that while digital solutions are essential, integrating community-based options can enhance trust and comfort, leading to greater diversity in trial participation.
## [](#moe-have-you-seen-patterns-in-which-conditions-benefit-most-from-dcts-in-improving-diversity)Moe: Have you seen patterns in which conditions benefit most from DCTs in improving diversity?
The benefits of DCTs can vary significantly depending on the condition being studied. A lightweight, community-based approach is most effective for high-volume vaccine trials, which involve healthy participants and require a quick turnaround. These trials benefit from being easily accessible and minimally invasive. In contrast, [oncology trials](https://www.clinicaltrialvanguard.com/news/mindranks-mrank-106-gets-fda-clearance-for-oncology-trials/) involving patients with complex needs require a more intensive and supportive approach. We provide comprehensive resources, such as patient resource centers and frequent updates, to ensure participants feel supported throughout the trial. This tailored approach, which includes input from our patient council, ensures that each trial is designed with its participants’ specific needs and preferences, ultimately improving diversity and patient satisfaction.
## [](#moe-how-does-medable-balance-cost-efficiency-with-meaningful-patient-engagement-for-underserved-communities)Moe: How does Medable balance cost efficiency with meaningful patient engagement for underserved communities?
Balancing cost efficiency with patient engagement is a core principle at Medable. We aim to connect patients and sites, transforming industry standards to reduce trial times and bring more drugs to market faster. By focusing on patient engagement, we improve adherence and completion rates, reducing the number of participants needed and lowering costs. For example, our well-designed study workflows, which include reminders and engagement points, have resulted in adherence rates of over 85%, compared to the industry standard of 65%. This enhances efficiency and ensures that trials are meaningful and accessible to all participants, particularly those from underserved communities.
## [](#moe-what-new-dimensions-should-be-explored-to-refine-dcts-role-in-addressing-healthcare-disparities)Moe: What new dimensions should be explored to refine DCTs’ role in addressing healthcare disparities?
As I see it, we need to refine how we measure success in clinical trials. Traditional outcome measures often focus solely on scientific metrics, but we must consider what truly matters to patients. This is especially important in fields like [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/), where traditional measures have been inconsistent. We can develop new ways to assess patient experiences and outcomes by leveraging AI and observational data. This will allow us to better understand treatments’ impact on patients’ lives and address healthcare disparities more effectively. Ultimately, our goal is to ensure that clinical trials are scientifically rigorous but also patient-centered and inclusive.
**Categories:** Article: Executive Interviews
---
### [Pelareorep: A Game-Changer in Oncolytic Virus Therapy](https://www.clinicaltrialvanguard.com/executiveinterviews/pelareorep-a-game-changer-in-oncolytic-virus-therapy/)
**Published:** March 4, 2025
**Author:** Moe Alsumidaie
**Content:**
In this conversation, we engage with Dr. Thomas Heineman, Chief Medical Officer of Oncolytics Biotech, to explore the innovative cancer treatment, pelareorep (pela). This proprietary oncolytic virus is making waves in the field of immuno-oncology, particularly in breast and pancreatic cancers. Dr. Heineman shares insights into pela’s unique mechanisms, administration, and potential to redefine therapeutic strategies and improve patient outcomes.
## [](#moe-pela-reshapes-tumors-from-cold-to-hot-can-you-explain-the-biological-mechanism-and-how-it-compares-to-other-immunotherapy-approaches)**Moe: Pela reshapes tumors from cold to hot. Can you explain the biological mechanism and how it compares to other immunotherapy approaches?**
**Thomas Heineman:** Pela is a non-genetically modified reovirus that selectively infects cancer cells, sparing normal tissue. Unlike most oncolytic viruses requiring direct tumor inoculation, pela is administered intravenously, enhancing its reach to both primary and metastatic sites. This method simplifies dosing and broadens its therapeutic impact. The double-stranded RNA of pela is recognized by pattern recognition receptors in cancer cells, leading to an upregulation of interferon expression and pro-inflammatory cytokines. This cascade recruits T, natural killer, and dendritic cells, fostering innate and adaptive immune responses. Additionally, pela stimulates the expansion of
Dr. Thomas Heineman, Chief Medical Officer of Oncolytics Biotech
tumor-infiltrating lymphocyte clones in the blood, enhancing their ability to attack the tumor. This dual action of modifying the tumor microenvironment and expanding immune cell populations sets pela apart from other immunotherapies, offering a comprehensive approach to tumor eradication.
## [](#moe-how-does-iv-administration-of-pela-affect-biodistribution-and-tumor-selectivity-and-what-are-its-efficacy-and-safety-implications)**Moe: How does IV administration of pela affect biodistribution and tumor selectivity, and what are its efficacy and safety implications?**
**Thomas Heineman:** The intravenous administration of pela is a significant advancement, allowing for safe, convenient dosing without the need for special biohazard precautions. This method enhances effectiveness by delivering pela to primary and metastatic tumor sites and lymph nodes, stimulating antiviral T-cell responses. This capability supports the expansion and proliferation of anti-tumor T cells, which is crucial for its anti-tumor effect. The ability to reach lymph nodes is critical, as it facilitates the generation of new antiviral T-cell responses that bolster the anti-tumor immune response. This aspect of pela’s administration broadens its therapeutic reach and enhances its safety profile, making it a versatile option for various cancer types.
## [](#moe-in-the-bracelet-1-study-how-does-pela-enhance-paclitaxels-effectiveness-in-hr-positive-her2-negative-metastatic-breast-cancer)**Moe: In the BRACELET-1 study, how does pela enhance paclitaxel’s effectiveness in HR-positive HER2-negative metastatic breast cancer?**
**Thomas Heineman:** The BRACELET-1 study, which combined pela with paclitaxel, revealed a strong clinical signal, underscoring pela’s potential in enhancing chemotherapy efficacy. Pela stimulated the expansion of T cell populations and indirectly expanded tumor-infiltrating lymphocyte clones, a key element of its immunologic effect. This expansion is crucial, as it directly correlates with improved clinical outcomes. In the study, patients receiving paclitaxel alone performed as expected, but those receiving the combination with pela showed significant T-[cell expansion](https://www.clinicaltrialvanguard.com/news/lymphodepletion-improves-t-cell-expansion-in-lymphoma-patients/) and a robust clinical efficacy signal, almost doubling median PFS, from 6.4 to 12.1 months and increasing median OS by almost 80%, from 18.2 months to an estimated 32.1 months. This direct link between immunologic effects and clinical response highlights pela’s potential to transform standard chemotherapy regimens, offering a more effective treatment strategy for patients with advanced [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/).
## [](#moe-what-potential-biomarkers-could-predict-response-to-pela-in-combination-with-checkpoint-inhibitors-as-seen-in-the-goblet-study)**Moe: What potential biomarkers could predict response to pela in combination with checkpoint inhibitors, as seen in the GOBLET study?**
**Thomas Heineman:** Pela’s ability to increase PDL1 expression forms the basis for its synergy with checkpoint inhibitors, as demonstrated in the GOBLET study. While no specific biomarkers are required for patient selection, potential biomarkers like tumor-infiltrating lymphocyte clonal expansion could serve as on-treatment indicators. This approach is speculative but consistent with existing data, suggesting it might be valuable in future studies. The ability to predict response through biomarkers would enhance patient selection and treatment personalization, maximizing therapeutic outcomes and minimizing unnecessary exposure to ineffective treatments.
## [](#moe-what-are-the-key-considerations-in-designing-registration-enabling-studies-for-pela-especially-in-patient-selection-and-study-endpoints)**Moe: What are the key considerations in designing registration-enabling studies for pela, especially in patient selection and study endpoints?**
**Thomas Heineman:** In HR-positive HER2-negative breast cancer, pela has shown pronounced benefits, particularly in patients with advanced or metastatic disease who have progressed on prior therapies. The treatment path for these patients is complex, with a need for better options for post-antibody drug conjugate therapy. Pela’s broad mechanism of action allows it to treat a range of patients without requiring specific genetic markers, making it a versatile option in the evolving immuno-oncology landscape. The challenge lies in designing studies capturing this broad applicability while ensuring robust endpoints demonstrating clinical benefit. The ability to address unmet needs in this patient population positions pela as a promising candidate for registration-enabling studies.
## [](#moe-how-does-pelas-mechanism-compare-to-other-oncolytic-viruses-and-what-unique-advantages-or-limitations-does-it-present)**Moe: How does pela’s mechanism compare to other oncolytic viruses, and what unique advantages or limitations does it present?**
**Thomas Heineman:** Pela’s intravenous delivery and non-genetically modified nature set it apart from other oncolytic viruses. It naturally targets cancer cells and stimulates immune responses without genetic manipulation. Pela’s ability to expand tumor-infiltrating lymphocyte populations is a powerful differentiator, enabling it to make the tumor accessible to the immune system and attack it effectively. This dual capability offers a unique advantage in combination immunotherapy strategies, as it enhances the overall immune response while maintaining a broad applicability across different cancer types. The absence of genetic modification also simplifies its clinical use, reducing regulatory hurdles and expanding its potential reach in oncology.
## [](#moe-is-there-anything-else-you-want-to-add-about-pelas-potential-and-future-directions)**Moe: Is there anything else you want to add about pela’s potential and future directions?**
**Thomas Heineman:** We focus on breast and [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) programs, but pela has broad potential applicability. We’re exploring its use in earlier stages of breast cancer and combination with CAR T therapy for solid tumors. Pela’s potential extends beyond current applications, and we look forward to expanding our development in these directions. The versatility and efficacy of pela position it as a promising candidate for future cancer therapies, with the potential to significantly impact patient outcomes across a range of malignancies.
**Categories:** Article: Executive Interviews
---
### [Revolutionizing NSCLC Treatment: Insights from ImmunityBio's CMO](https://www.clinicaltrialvanguard.com/executiveinterviews/revolutionizing-nsclc-treatment-insights-from-immunitybios-cmo/)
**Published:** March 4, 2025
**Author:** Moe Alsumidaie
**Content:**
In this discussion with Bobby Reddy, Chief Medical Officer of ImmunityBio, we delve into the innovative immunotherapy strategies targeting non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). Reddy discusses the promising combination of ANKTIVA’s [IL-15](https://www.clinicaltrialvanguard.com/news/teva-celiac-drug-gets-fda-fast-track/) super agonist with tislelizumab PD-1 inhibition, aimed at reactivating immune responses in patients resistant to prior therapies. This conversation explores the mechanisms, clinical trials, and future directions of this groundbreaking approach, offering a glimpse into the potential transformation of cancer treatment.
## [](#moe-how-does-anktivas-il-15-super-agonist-with-tislelizumab-pd-1-inhibition-synergize-to-reactivate-immune-responses-in-resistant-nsclc-patients)Moe: How does ANKTIVA’s IL-15 super agonist with tislelizumab PD-1 inhibition synergize to reactivate immune responses in resistant NSCLC patients?
The combination addresses T cell exhaustion, a common issue in patients who have lost response to checkpoint inhibitors. ANKTIVA’s IL-15 super agonist activates and proliferates NK cells, CD8 positive T cells, and memory T cells, reinvigorating the immune response. This is crucial because, in cancer, T cells can become exhausted, similar to what we see in infectious diseases. By activating these cells, we can potentially restore the immune response, vital for patients with developed resistance. In our trials, we’ve seen that this approach can lead to meaningful extensions in survival, as evidenced by our 14.1-month overall survival data in NSCLC patients. This is particularly significant given the recent high-profile failures in similar trials targeting other pathways, such as [TIGIT](https://www.clinicaltrialvanguard.com/news/anti-tigit-antibody-the-revolutionary-cancer-treatment-you-must-know-about/) and LAG-3, highlighting our focus on enhancing the host’s immune response.
Bobby Reddy, Chief Medical Officer of ImmunityBio
## [](#moe-what-biomarkers-or-clinical-characteristics-identify-nsclc-patients-who-might-benefit-most-from-this-combination-therapy)Moe: What biomarkers or clinical characteristics identify NSCLC patients who might benefit most from this combination therapy?
Interestingly, we focus not on specific tumor phenotypes but on the patient’s immune response. For instance, PDL1 status doesn’t significantly impact outcomes in our trials. We’re exploring other biomarkers, such as MHC loss, a known immune escape mechanism. Enriching NK cells could be a critical factor, and we’re collecting blood samples in our trials to investigate these aspects further. Our approach is to look at the host’s immune system holistically, which could lead to a more universal therapy applicable to a broader range of patients, regardless of their tumor’s genetic makeup. This strategy aligns with our goal of developing treatments that are not only effective but also widely applicable, addressing the diverse needs of the cancer patient population.
## [](#moe-how-does-the-efficacy-of-this-combination-compare-to-existing-second-and-third-line-nsclc-treatments-in-terms-of-survival-and-quality-of-life)Moe: How does the efficacy of this combination compare to existing second and third-line NSCLC treatments in terms of survival and quality of life?
Our data shows a 14.1-month overall survival, which is promising compared to the 7-8 months typically seen with standard treatments like docetaxel. Importantly, our therapy doesn’t carry the severe side effects associated with chemotherapy, such as neutropenia and hair loss. This makes it a potentially more attractive option for patients, offering a better quality of life while extending survival. In recent trials, other therapies have struggled to demonstrate significant improvements, often due to targeting “passenger” alterations rather than enhancing the immune system’s capacity. Our approach, which avoids the severe toxicities of chemotherapy, could redefine the standard of care for patients who have exhausted other options.
## [](#moe-what-are-the-potential-risks-and-adverse-events-with-anktiva-and-tislelizumab-and-how-will-the-trial-design-address-these-safety-concerns)Moe: What are the potential risks and adverse events with ANKTIVA and tislelizumab, and how will the trial design address these safety concerns?
We’ve observed mild, self-limiting local cutaneous reactions and low-grade flu-like symptoms but no severe cytokine release syndrome. Importantly, we haven’t seen an increased rate of immune-related adverse events compared to checkpoint inhibitors alone. Our trial design includes rigorous monitoring to ensure patient safety and effectively manage adverse effects. This safety profile is particularly encouraging because it suggests we can enhance the immune response without significantly increasing the risk of adverse events, a common concern with combination therapies. By focusing on the host’s immune system, we aim to provide a treatment that is effective and well-tolerated, improving the overall patient experience.
## [](#moe-what-regulatory-challenges-do-you-anticipate-for-the-bla-submission-in-2025-and-how-might-the-rescue-21201a-trial-outcomes-influence-approval)Moe: What regulatory challenges do you anticipate for the BLA submission in 2025, and how might the Rescue 21201A trial outcomes influence approval?
We’ve had positive discussions with the FDA, who encouraged us to file based on our promising data. The ongoing confirmatory trial will provide additional support, potentially facilitating an accelerated approval. The regulatory agencies recognize the unmet need in NSCLC, especially for patients progressing after checkpoint inhibitors, and our trial aims to address this gap. By demonstrating safety and efficacy, we hope to meet the regulatory requirements and bring this innovative therapy to patients desperately needing new options. Our collaboration with regulatory bodies ensures we can navigate the approval process efficiently and effectively.
## [](#moe-if-successful-how-might-this-combination-therapy-influence-future-research-and-development-of-immunotherapies-for-other-resistant-cancers)Moe: If successful, how might this combination therapy influence future research and development of immunotherapies for other resistant cancers?
Success with this combination could pave the way for more personalized and multi-faceted treatment approaches. Understanding the biology of resistance and leveraging combination therapies, including cell therapies and targeted agents, could lead to significant advancements in treating various cancers. We aim to achieve long-term disease control and potential cures by enhancing the host’s immune response. This approach could revolutionize how we think about cancer treatment, moving beyond a one-size-fits-all model to more tailored therapies that address each patient’s unique needs. By focusing on the host’s immune system, we can develop strategies that are effective and adaptable to a wide range of cancers, offering hope to patients who have exhausted other options.
## [](#moe-is-there-anything-else-youd-like-to-add)Moe: Is there anything else you’d like to add?
We’re excited about our partnership with BeiGene and the potential of tislelizumab. Our holistic approach focuses on the host’s immune response rather than excluding patients with driver mutations. This inclusivity could lead to broader applicability and better outcomes for patients who have exhausted other treatment options. By considering the host’s immune system as a whole, we aim to develop therapies that are effective and widely applicable, addressing the diverse needs of the cancer patient population. This strategy aligns with our goal of providing innovative solutions that can make a real difference in patients’ lives.
**Categories:** Article: Executive Interviews
---
### [New Obesity Treatment Mechanisms: Dr. Bolz on Aphaia Pharma's Innovative Approach](https://www.clinicaltrialvanguard.com/executiveinterviews/new-obesity-treatment-mechanisms-dr-bolz-on-aphaia-pharmas-innovative-approach/)
**Published:** March 4, 2025
**Author:** Moe Alsumidaie
**Content:**
In this discussion with Steffen-Sebastian Bolz, MD, PhD, we explore Aphaia’s groundbreaking work in [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatment. With his dual expertise in clinical and molecular biology, Dr. Bolz shares insights on Aphaia’s novel oral coated glucose formulation. This innovative approach has the potential to transform current treatment paradigms by emphasizing physiological mechanisms and minimizing side effects.
## [](#moe-what-role-does-aphaias-approach-play-in-the-obesity-treatment-landscape)**Moe: What role does Aphaia’s approach play in the obesity treatment landscape?**
Aphaia’s approach is fundamentally different from traditional hormone therapies like GLP-1 agonists, which often require high doses and can lead to significant side effects. These therapies act like a “bulldozer,” disrupting the body’s natural rhythms and causing adverse effects such as nausea and vomiting. In contrast, our method leverages glucose, the most prevalent biomolecule, to naturally stimulate the body’s natural hormone production. By delivering glucose to the distal small intestine, we activate enteroendocrine cells to release a balanced mix of hormones beyond GLP-1 agonists. These hormones engage endocrine, neuroendocrine, and neuronal signaling pathways to deliver a broad range of metabolic and behavioral effects, including control of hunger, satiety, and glucose metabolism, among others. Our physiological
Dr. Steffen-Sebastian Bolz, MD, PhD, Chief Scientific Officer, Aphaia Pharma
approach mimics the body’s natural response to food, resulting in minimal side effects, as demonstrated in multiple trials to date. This allows for repetitive daily administrations and precise timing of drug intake to help retrain metabolic circadian rhythms – an approach believed to positively influence [metabolic health](https://www.clinicaltrialvanguard.com/news/agelessrx-launches-oral-glp-1-drops-for-metabolic-health/) and support weight loss. We trust that this innovative multilayered approach constitutes an effective, sustainable, and patient-friendly treatment modality. Initial signals from our Phase 2 trials confirm this positioning, highlighting our approach as a promising new alternative in the obesity treatment landscape.
Additionally, our coated glucose formulation would be highly accessible to patients since the components are economical, the synthesis is relatively simple, and the formulation’s production is easily scalable.
## [](#moe-how-might-this-therapy-alter-the-approach-to-preventing-pre-diabetes-progression)**Moe: How might this therapy alter the approach to preventing pre-diabetes progression?**
Our trials have shown that Aphaia’s formulation significantly improves glucose tolerance in pre-diabetic patients, with results comparable to long-term lifestyle interventions and GLP-1 mimetics. This is achieved by engaging the full spectrum of enteric hormones, not just GLP-1, which allows for a more comprehensive metabolic regulation. Given its safety profile and ease of use, this formulation could be widely adopted for early intervention in pre-diabetic individuals, potentially preventing the progression of [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/). It’s a shift towards a preventive model in healthcare, focusing on maintaining metabolic health rather than just managing disease symptoms. The formulation’s simplicity—requiring only a sachet mixed with water to form a gel—makes it accessible and easy to integrate into daily life, which is crucial for widespread adoption. Moreover, the inherent potential to treat young children, who currently have limited options, could be a game-changer in addressing obesity from an early age.
## [](#moe-how-does-targeted-glucose-release-affect-hormone-secretion-and-metabolic-regulation)**Moe: How does targeted glucose release affect hormone secretion and metabolic regulation?**
The targeted release of glucose in the distal jejunum allows us to stimulate nutrient-sensing intestinal cells to induce the release of a broad range of hormones, including GLP-1, GLP-2, and PYY, in their natural proportions. This orchestrated hormone release acts on multiple organs to regulate metabolism and behavior effectively. By using the body’s highly optimized physiological pathways, we aim to achieve significant metabolic benefits without the adverse effects associated with synthetic hormone therapies. This method was designed to support weight loss and enhance insulin sensitivity and overall metabolic health. The analogy of an orchestra is fitting here; while traditional therapies focus on a single instrument, we engage the entire ensemble, hoping to create a harmonious and effective metabolic response.
## [](#moe-what-are-the-key-design-elements-of-your-phase-two-trial-for-obesity)**Moe: What are the key design elements of your phase two trial for obesity?**
Our trial design is ambitious, focusing on obesity as an umbrella term to include various comorbidities like type 2 diabetes, metabolic syndrome, and non-alcoholic fatty liver disease (NAFLD). This setup also allows us to explore how these conditions interact with weight loss. We’ve incorporated a bi-daily application scheme of the formulation to harness circadian effects and further enhance efficacy. The trial’s comprehensive approach has already generated lots of data to inform our future studies and helped us refine ou therapy with the aim to maximize efficacy while keeping risks minimal. We believe this positions us well to address the broader challenges in obesity treatment by offering a more holistic and patient-centered solution. The trial’s design reflects our commitment to understanding the complex interplay of metabolic factors and tailoring our approach to meet the diverse needs of patients suffering from metabolic diseases, including obesity. We aim to achieve weight loss and improve overall health outcomes and might add a new paradigm to the obesity treatment research landscape.
**Categories:** Article: Executive Interviews
---
### [2025 SCOPE Summit: Transforming Clinical Trials with Pragmatic Trial Approaches](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-transforming-clinical-trials-with-pragmatic-trial-approaches/)
**Published:** March 4, 2025
**Author:** Moe Alsumidaie
**Content:**
The [2025 SCOPE Summit](https://www.scopesummit.com/) brought together industry leaders to explore integrating real-world data into clinical trials, focusing on pragmatic trials as a transformative approach. Key discussions revolved around regulatory initiatives, innovative trial designs, and operational strategies to enhance the applicability of clinical research in real-world settings. The summit highlighted the potential of pragmatic trials to bridge the gap between clinical research and practice, offering insights into how these trials can streamline processes, reduce burdens, and improve patient outcomes.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#regulatory-initiatives-for-pragmatic-trials)Regulatory Initiatives for Pragmatic Trials
The summit’s opening session, led by Gracy Crane from Roche Products Ltd, delved into the regulatory frameworks essential for supporting pragmatic trials. Crane emphasized the critical role of these frameworks in enabling trials to transition from controlled research environments to real-world clinical practice. The session highlighted the FDA’s efforts to integrate real-world data into clinical trials, addressing the effectiveness of therapies beyond the confines of traditional randomized controlled trials (RCTs). Crane pointed out that pragmatic trials are designed to answer whether a therapy works in diverse, real-world settings, a question often left unanswered by traditional RCTs due to their controlled nature and homogeneous populations. This regulatory focus aims to ensure that pragmatic trials can provide substantial evidence for drug approvals, making them a valuable tool for both sponsors and regulators.
The discussion also touched on the importance of policy drivers and the momentum required to sustain these innovative approaches. Crane noted that pragmatic trials offer flexibility, allowing for integrating existing data and enrolling diverse populations. This adaptability enhances the relevance and applicability of clinical research, ultimately bridging the gap between efficacy and effectiveness. By focusing on real-world applicability, pragmatic trials can provide the substantial evidence needed for regulatory approvals, thus playing a crucial role in the future of drug development.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#the-role-of-pragmatic-trials-in-clinical-practice)The Role of Pragmatic Trials in Clinical Practice
Pragmatic trials are designed to make clinical trial results applicable to broader, real-world populations, addressing the gap between efficacy and effectiveness. The summit explored how these trials can serve as a bridge, ensuring that therapies are effective in diverse clinical settings. The discussion included the concept of the Pressi diagram, which outlines various domains of pragmatism in trials, such as eligibility criteria and trial settings. This diagram guides trial designers to select which pragmatic elements to incorporate, such as broadening inclusion criteria or shifting trial settings from academic centers to community clinics.
The flexibility offered by pragmatic trials was highlighted as a key factor in their potential to provide substantial evidence for drug approvals. This adaptability allows for integrating existing data and the enrollment of diverse populations, ultimately enhancing the relevance and applicability of clinical research. By focusing on real-world applicability, pragmatic trials can provide the substantial evidence needed for regulatory approvals, thus playing a crucial role in the future of drug development.
#### [](#innovation-in-clinical-trial-design)Innovation in Clinical Trial Design
Henry Wei from Regeneron Pharmaceuticals shared insights into the innovative approaches adopted in clinical trial design. Wei, who leads the Development Innovation group, emphasized the importance of collaboration and the role of organizations like Transcelerate in fostering innovation. He highlighted the need for trials that are efficient and capable of integrating seamlessly into clinical practice. Wei pointed out that the current healthcare landscape demands trials that reduce the burden on healthcare providers and improve the speed and quality of clinical research.
Wei mentioned the collaborative environment of Transcelerate as a “treehouse” where industry leaders can come together to share ideas and drive innovation. This collaborative approach is crucial for developing trials that are more streamlined and accessible, ultimately benefiting both patients and healthcare providers. By fostering a culture of innovation and collaboration, the industry can create more efficient trials reflective of real-world clinical practice, ultimately improving patient outcomes and advancing clinical research.
[](https://clineco.io?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#fdas-center-for-clinical-trial-innovation)FDA’s Center for Clinical Trial Innovation
Kevin Bugin, formerly Deputy Director of Operations in the FDA’s Office of New Drugs and now at Amgen, stepped in for the originally scheduled FDA speaker to discuss the FDA’s Center for Clinical Trial Innovation (C3TI). He provided insights into the center’s demonstration programs, which aim to integrate pragmatic elements into clinical trials, such as streamlined trials embedded in routine clinical practice. These initiatives help sponsors collaborate more effectively with the FDA, supporting the broader adoption of innovative trial designs. Bugin emphasized the importance of these collaborations in ensuring the successful implementation of pragmatic trial approaches.
The C3TI’s initiatives, such as the Bayesian supplementary analyses and the collection of safety data, are intended to build experience and confidence in using these approaches. By partnering with sponsors, the FDA aims to learn from these trials and disseminate best practices across the industry, ultimately contributing to a more efficient and effective clinical trial ecosystem. These efforts are crucial for advancing clinical research and ensuring that trials are efficient and reflect real-world clinical practice.
#### [](#operational-considerations-for-pragmatic-trials)Operational Considerations for Pragmatic Trials
Stephanie Derbyshire from Roche highlighted the operational challenges and considerations in implementing pragmatic trials. She stressed the need for a shift in thinking to accommodate the diverse patient populations and settings that pragmatic trials entail. Derbyshire discussed the importance of selecting the correct sites and patients and the need for tailored training and support for research-naive sites. She pointed out that traditional trial sites may not always reflect the diverse patient populations needed for pragmatic trials, necessitating the inclusion of community clinics and family practitioners.
This approach requires careful planning and support to ensure these sites can effectively participate in trials. Derbyshire also addressed the need for initiatives to reduce patient and site burden, such as transportation services and the use of local physicians for specific assessments. These operational considerations are crucial for successfully implementing pragmatic trials, ensuring they are both efficient and reflective of real-world clinical practice. By addressing these challenges, the industry can develop trials that are more accessible and patient-centric.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
## [](#summary)**Summary**
The 2025 [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Summit highlighted the transformative potential of pragmatic trials in bridging the gap between clinical research and real-world practice. By focusing on regulatory support, innovative trial designs, and operational considerations, the summit underscored the importance of these trials in enhancing the relevance and applicability of clinical research. As the industry moves forward, the insights gained from this summit will be crucial in shaping the future of clinical trials, ultimately improving patient outcomes and advancing clinical research.
**Categories:** Article: Conference Coverage
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### [Cybrexa Unveils Promising Preclinical Data on Tumor-Selective PDCs at ESMO 2025](https://www.clinicaltrialvanguard.com/news/cybrexa-unveils-promising-preclinical-data-on-tumor-selective-pdcs-at-esmo-2025/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
Cybrexa Therapeutics presented preclinical data at the ESMO Targeted Anticancer Therapies Congress 2025, showcasing their alphalex™ peptide-drug conjugates (PDCs). These PDCs demonstrate tumor-selective delivery of potent microtubule inhibitors, resulting in significant tumor suppression and a durable anti-tumor immune response. This approach leverages tumor acidity for precise drug delivery, potentially mitigating the toxicities associated with antigen-based targeting seen in antibody-drug conjugates (ADCs).
This development is crucial because it offers a potential alternative for patients who have progressed after treatment with TOP1-based ADCs, a common class of cancer therapies. The alphalex platform’s antigen-agnostic nature broadens its applicability across various tumor types, potentially addressing limitations encountered with current targeted therapies. The demonstrated synergy with existing standard-of-care treatments, like doxorubicin and anti-PD-L1 therapy, further strengthens its potential clinical impact.
Preclinical findings revealed complete tumor suppression in [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) models using alphalex PDCs as a monotherapy. Additionally, enhanced tumor suppression was observed when combined with standard treatments in various cancer models. Importantly, data confirms tumor-selective drug delivery, sparing healthy immune cells. The activation of T- and B-cell responses, coupled with evidence of long-term immune memory, suggests sustained anticancer effects.
Cybrexa is actively translating these findings into clinical practice. A Phase 2 clinical trial for CBX-12, an alphalex PDC delivering the TOP1 inhibitor exatecan, is underway for platinum-resistant or refractory ovarian cancer. This follows promising Phase 1 data demonstrating activity across multiple tumor types with a manageable safety profile. These advancements position alphalex PDCs as a potential next-generation cancer therapy, offering new treatment options for patients with hard-to-treat solid tumors. The continued clinical development of this platform, particularly its combination potential with existing therapies, holds significant promise for improving cancer treatment outcomes.
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**Tags:** eClinical Tech News
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### [Reunion Neuroscience Raises Post Pardum Depression at MMH Forum](https://www.clinicaltrialvanguard.com/news/reunion-neuroscience-raises-post-pardum-depression-at-mmh-forum/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
Reunion Neuroscience is participating in the 2025 Maternal [Mental Health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) (MMH) FORUM to discuss [RE104](https://www.clinicaltrialvanguard.com/news/reunion-unlocks-full-re104-ppd-phase-2-trial-results/), its lead product candidate for treating postpartum depression (PPD). The company’s participation highlights the ongoing Phase 2 RECONNECT clinical trial, which is evaluating the safety and efficacy of a single dose of RE104 for moderate-to-severe PPD. This forum allows Reunion to present its research to policymakers, payers, and providers, aiming to improve maternal mental health care.
Maternal mental health disorders, impacting a significant number of expecting and postpartum mothers, represent a substantial unmet need. Reunion’s presence at the MMH FORUM underscores the critical importance of developing new treatment options. The forum offers a platform to discuss critical issues in maternal mental health care and showcase potential solutions like RE104, potentially leading to improved diagnosis and treatment standardization across the healthcare system.
RE104 is a prodrug of 4-OH-DiPT, designed to induce a shorter psychedelic experience than traditional psychedelics like psilocybin, while maintaining a similar safety and efficacy profile. The RECONNECT trial is a multicenter, randomized, double-blind, active dose-controlled study. Reunion is also planning the REKINDLE Phase 2 trial to evaluate RE104 for adjustment disorder in patients with cancer and other medical illnesses.
Reunion’s participation in the MMH FORUM and the ongoing RECONNECT trial position RE104 as a potential game-changer in the treatment of PPD. The data generated from this trial, along with future studies like REKINDLE, will be crucial in determining the efficacy and broader applicability of this novel therapeutic approach for various mental health disorders. This research has the potential to reshape the landscape of mental health care, particularly for underserved populations like postpartum mothers.
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**Categories:** News
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### [Nuvectis Pharma: Combination Therapy Improves EGFR Inhibitor Efficacy](https://www.clinicaltrialvanguard.com/news/nuvectis-pharma-combination-therapy-improves-egfr-inhibitor-efficacy/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
Nuvectis Pharma announced promising preclinical research demonstrating the synergistic effects of combining its drug candidate, NXP900, with [osimertinib](https://www.clinicaltrialvanguard.com/news/abbisko-astrazeneca-begin-phase-i-ii-trial-of-lumipodlin-with-osimertinib-in-egfr-mutant-nsclc/) (Tagrisso®) in treating EGFR-mutated [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). The research, conducted at the Lerner Research Institute, Cleveland Clinic, showed the combination therapy outperformed osimertinib alone in preclinical models, leading to reduced cancer cell proliferation and increased cell death. This builds upon previous research from AstraZeneca, which indicated that NXP900 can reverse osimertinib resistance in resistant cell lines.
This development is crucial for addressing the challenge of acquired resistance to osimertinib, a common issue that limits the long-term effectiveness of this therapy in EGFR-mutated NSCLC. Overcoming this resistance mechanism could significantly extend the duration of response for patients and potentially improve overall survival rates. The findings provide additional validation for Nuvectis’ strategy of developing NXP900 as a combination therapy with existing targeted cancer drugs.
NXP900 is an oral small molecule inhibitor of the SRC Family of Kinases (SFK), including SRC and YES1. Its mechanism of action is designed to fully inhibit both the catalytic and scaffolding functions of SRC kinases, effectively shutting down this signaling pathway. The drug is currently in a Phase 1a dose-escalation study, and the company anticipates initiating a Phase 1b program combining NXP900 with osimertinib in EGFR-mutated NSCLC patients. The Phase 1a study is nearing completion.
These positive preclinical results reinforce the therapeutic potential of NXP900 in combination with osimertinib. The upcoming Phase 1b trial will be critical for evaluating the safety and efficacy of this combination in humans. If successful, this combination therapy could offer a new treatment option for patients with EGFR-mutated NSCLC who have developed resistance to osimertinib, potentially extending the benefits of targeted therapy and improving patient outcomes. This progress underscores the importance of continued research into mechanisms of resistance and novel combination therapies for this prevalent form of lung cancer.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Tiziana Life Sciences Files IND Application with FDA for ALS Phase 2 Trial](https://www.clinicaltrialvanguard.com/news/tiziana-life-sciences-files-ind-application-with-fda-for-als-phase-2-trial/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
[Tiziana Life Sciences](https://www.clinicaltrialvanguard.com/news/tiziana-life-sciences-nasal-spray-shows-promise-in-spinal-cord-injury-treatment/)[Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) has submitted an Investigational New Drug (IND) application to the FDA for a phase 2 clinical trial of intranasal foralumab for Amyotrophic Lateral Sclerosis (ALS). This application follows a grant award from the ALS Association’s Hoffman ALS Clinical Trial Awards Program, supporting Tiziana’s focus on intranasal foralumab for three neurodegenerative diseases: ALS, [Multiple Sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/), and Alzheimer’s disease. The grant fosters collaboration between industry and academia to translate research into clinical studies.
This IND submission is a crucial step in developing new treatment options for ALS, a fatal neurodegenerative disease with limited effective therapies. A phase 2 trial will provide critical data on the safety and potential efficacy of intranasal foralumab, offering hope to patients facing a devastating diagnosis with a currently poor prognosis. The study will evaluate how well foralumab can slow disease progression and improve quality of life. This trial will also generate data that can inform further research and development into much needed therapies for ALS.
The planned phase 2 trial will involve 20 patients and assess two doses of intranasal foralumab. Foralumab, a fully human anti-CD3 monoclonal antibody, is designed to stimulate T regulatory cells when administered intranasally. Previous studies have shown promising results with foralumab in other neurological conditions, including Multiple Sclerosis, suggesting its potential to modulate the immune system and impact neurodegenerative processes. The company has ongoing Expanded Access programs and clinical trials exploring foralumab in other neurological indications.
The initiation of this clinical trial represents a significant step toward potentially improving treatment outcomes for ALS. Positive results could pave the way for larger studies and ultimately bring a novel therapeutic option to patients suffering from this debilitating disease. Further, this reinforces the potential of intranasal foralumab as a platform technology for addressing a range of neurodegenerative disorders.
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**Tags:** eClinical Tech News
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### [Celon Pharma Promising Phase 2 Data for PDE10A Inhibitor in Parkinson's Dyskinesia](https://www.clinicaltrialvanguard.com/news/celon-pharma-promising-phase-2-data-for-pde10a-inhibitor-in-parkinsons-dyskinesia/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
Celon Pharma announced positive Phase 2 clinical trial results for its oral, once-daily [PDE10A](https://www.clinicaltrialvanguard.com/news/fda-clears-promising-schizophrenia-drug-for-phase-3-trial/) inhibitor, CPL’36, for treating Levodopa-Induced Dyskinesia (LID) in Parkinson’s disease. The study achieved its primary endpoint and several secondary endpoints, demonstrating significant efficacy in reducing LID symptoms. CPL’36 had previously shown promise in Phase 2 trials for [schizophrenia](https://www.clinicaltrialvanguard.com/article/article-deep-dive/the-fda-measures-all-schizophrenia-symptoms-at-the-same-clock-a-six-trial-ipd-analysis-just-proved-thats-wrong/).
This development is potentially groundbreaking for Parkinson’s patients experiencing LID, a common and debilitating side effect of standard levodopa therapy. Current treatment options for LID are limited, and CPL’36’s efficacy could significantly improve patients’ quality of life by reducing involuntary movements and improving motor control. The positive results from the schizophrenia trials suggest the drug may have broader applications across neurological conditions, making it a particularly interesting asset for Celon.
In the 4-week, placebo-controlled trial involving 105 adult patients with moderate-severe to severe LID, CPL’36 at doses of 20mg and 40mg showed statistically significant improvements in the Unified Dyskinesia Rating Scale (UDysRS) total score compared to placebo. Specifically, the 20mg dose showed a 12.30 unit improvement (p<0.001, Cohen’s d: 0.90), while the 40mg dose showed a 13.58 unit improvement (p<0.001, Cohen’s d: 1.00). Improvement in the UDysRS objective subscale, a key secondary endpoint, was observed as early as Day 7. The drug was generally well-tolerated, with somnolence being the most common adverse event. Discontinuations due to treatment-related adverse events were higher in the active treatment arms (11.1% for 20mg and 8.6% for 40mg) compared to placebo (2.9%). One serious adverse event of atrial fibrillation was reported in the 40mg group. These positive Phase 2 results position CPL’36 as a potential game-changer in LID management. The next step for Celon Pharma will likely be to initiate larger Phase 3 trials to confirm these findings and further evaluate the drug’s long-term safety and efficacy. Success in Phase 3 could lead to regulatory approval and ultimately provide a much-needed new treatment option for Parkinson’s patients suffering from LID. This could also significantly bolster Celon Pharma’s position in the neurology market.
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**Tags:** eClinical Tech News
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### [Enlivex Therapeutics Announces Positive Interim Data from Phase I/II Allocetra™ Trial in Knee Osteoarthritis](https://www.clinicaltrialvanguard.com/news/enlivex-therapeutics-announces-positive-interim-data-from-phase-i-ii-allocetra-trial-in-knee-osteoarthritis/)
**Published:** March 5, 2025
**Author:** Jon Napitupulu
**Content:**
Enlivex Therapeutics announced positive interim six-month data from its Phase I/II Allocetra™ trial for knee [osteoarthritis](https://www.clinicaltrialvanguard.com/news/nih-funds-trial-for-non-surgical-knee-osteoarthritis-relief/). The trial showed statistically significant reductions in pain and improvements in joint function, with a sustained response at six months and no serious adverse events. The company will host a webinar on March 5, 2025, to discuss these results.
This positive data is a significant development in the osteoarthritis treatment landscape. Current treatments often provide limited pain relief and can have significant side effects. Allocetra™’s potential for sustained efficacy and safety could address a substantial unmet need for a long-term, effective therapy for this debilitating condition, offering hope for improved quality of life to millions of patients. This positive data may also spur further investment and research into macrophage reprogramming therapies, potentially opening new avenues for treating other inflammatory conditions.
Interim results from the Phase I stage show a 47% average pain reduction (P=0.0001) and a 46% improvement in joint function six months post-treatment. Furthermore, 83% of patients maintained a positive response to treatment at the six-month mark. Importantly, no serious adverse events were observed.
These positive interim results suggest Allocetra™ may become a viable treatment option for moderate to severe knee osteoarthritis. The sustained efficacy and favorable safety profile position Enlivex to potentially advance to the next phase of clinical trials and eventually bring a much-needed new therapy to market. This could significantly impact both Enlivex and the broader osteoarthritis treatment landscape.
Source link: [\\](https://www.globenewswire.com/news-release/2025/03/04/3036995/0/en/Enlivex-Therapeutics-Announces-Investor-Webinar-to-Discuss-Positive-Interim-Data-from-Phase-I-II-Allocetra-Trial-in-Knee-Osteoarthritis.html)
**Categories:** News
**Tags:** eClinical Tech News
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### [Invivyd Announces Neutralizing Activity of Pemigarda Against Dominant SARS-CoV-2 Variant](https://www.clinicaltrialvanguard.com/news/invivyd-announces-neutralizing-activity-of-pemigarda-against-dominant-sars-cov-2-variant/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
Invivyd announced positive in vitro neutralization data for its [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) antibody treatment, PEMGARDA (pemivibart), against the dominant LP.8.1 variant. The company also highlighted the consistent susceptibility of prevalent variants like KP.3.1.1 and XEC to PEMGARDA, attributing this to the structural integrity of the targeted epitope. Additionally, Invivyd’s next-generation antibody candidate, [VYD2311](https://www.clinicaltrialvanguard.com/news/invivyd-launches-phase-3-trial-for-covid-antibody-vyd2311/), demonstrated similar stability and neutralization against LP.8.1.
This continued effectiveness of PEMGARDA against evolving variants offers crucial protection for immunocompromised individuals who often experience limited vaccine efficacy. Consistent neutralization underscores the potential for longer-term prophylactic strategies, addressing a significant unmet need for this vulnerable population facing persistent COVID-19 risks. The stable activity also validates Invivyd’s antibody engineering approach focusing on structurally conserved viral targets.
PEMGARDA, currently authorized for pre-exposure prophylaxis in certain immunocompromised patients, maintains activity against all dominant circulating variants according to the CDC. Invivyd submitted these latest findings to the FDA, anticipating an updated fact sheet for healthcare providers. VYD2311, building upon the same structural backbone as PEMGARDA, may offer improved administration options, such as intramuscular injection.
These results suggest a promising path for Invivyd. The consistent neutralization observed with both PEMGARDA and VYD2311 reinforces the company’s strategy of targeting stable epitopes, potentially leading to durable protection against emerging COVID-19 variants and advancing the development of more accessible treatment options. This approach positions Invivyd to address ongoing challenges in managing COVID-19, particularly within vulnerable populations.
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**Tags:** eClinical Tech News
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### [Zyversa Demonstrates NLRP3 Inhibition Improves Heart Function, Glucose Homeostasis, and Insulin Sensitivity in Obese Animal Model](https://www.clinicaltrialvanguard.com/news/zyversa-demonstrates-nlrp3-inhibition-improves-heart-function-glucose-homeostasis-and-insulin-sensitivity-in-obese-animal-model/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
ZyVersa Therapeutics highlighted newly published data demonstrating the cardioprotective and metabolic benefits of inhibiting NLRP3 inflammasome pathways in an obese animal model with heart failure with preserved ejection fraction (HFpEF) and [type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/). The company’s Inflammasome ASC Inhibitor, IC 100, showed promising results in reducing inflammation and improving cardiac and metabolic function. This research suggests IC 100 may be an effective treatment for obesity and related cardiovascular and metabolic diseases.
This research is particularly important because HFpEF, often preceded by obesity, diabetes, and hypertension, is a major global health concern with limited treatment options. The study directly links systemic inflammation driven by comorbidities to the development of heart disease, offering a novel therapeutic approach. Demonstrating positive results in a model that mimics the complex interplay of these conditions strengthens the potential of IC 100 as a much-needed treatment.
The study, published in •Biomedicine & Pharmacotherapy•, showed that inhibiting NLRP3 inflammasome pathways with a similar mechanism to IC 100 led to several key improvements. These included reduced levels of the pro-inflammatory cytokine [IL-18](https://www.clinicaltrialvanguard.com/news/gileads-848m-bet-on-compugens-revolutionary-therapy/), decreased macrophage infiltration in the heart and visceral adipose tissue, and improved cardiac function and insulin sensitivity. Furthermore, the research indicated a reduction in cardiac hypertrophy and fibrosis, key structural abnormalities in HFpEF.
These findings pave the way for ZyVersa to initiate two preclinical studies of IC 100 in diet-induced obesity mouse models. One study will compare IC 100 to [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/), a current standard of care for obesity and diabetes, while the other will evaluate the combined effects of IC 100 and semaglutide. The results of these studies will be crucial in determining the next steps for the clinical development of IC 100 and could potentially offer a new therapeutic strategy for managing obesity and its associated cardiovascular and metabolic complications.
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### [Silexion Therapeutics Reports Positive Initial Data from Sil204 in Pancreatic Cancer Models](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-reports-positive-initial-data-from-sil204-in-pancreatic-cancer-models/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) (NASDAQ: SLXN) has released positive preclinical data for [SIL204](https://www.clinicaltrialvanguard.com/news/silexion-submits-pancreatic-cancer-trial-app-in-israel/), its RNAi therapy targeting KRAS-driven cancers. The study used orthotopic [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/) models, a more clinically relevant setting, and demonstrated that subcutaneously administered SIL204 reduced both primary tumor growth and metastasis. This is the first time the company has shown SIL204’s efficacy in an orthotopic model, significantly strengthening the drug’s potential clinical value.
This news is particularly important because it demonstrates the drug’s potential in a model that closely mimics human disease. The reduction in both primary tumor growth and metastatic spread with a minimally invasive subcutaneous administration further highlights SIL204’s therapeutic promise. Effective systemic delivery opens doors for broader treatment applications and potentially improved patient convenience compared to more invasive administration routes. Moreover, the therapy’s activity across multiple pancreatic cancer cell lines with varying KRAS mutation profiles suggests a potentially wide applicability for SIL204.
SIL204 significantly reduced tumor growth in various pancreatic cancer cell lines, including a 70% reduction in AsPC-1 (KRAS G12D mutation), dose-dependent reduction in Panc-1 (KRAS G12D mutation), and an 80% reduction in BxPC-3 (KRAS wild-type). Critically, the data showed a reduction in metastatic spread to the liver, intestine, spleen, and stomach in the Panc-1 and BxPC-3 models. The successful subcutaneous administration validates this method as a practical and potentially effective delivery route.
Based on these positive results, Silexion is revising its development strategy for SIL204, focusing on systemic administration. This advancement positions SIL204 as a potential treatment option for both primary and metastatic pancreatic cancers. The company’s move to expand its development plan signifies growing confidence in SIL204’s clinical potential and indicates the possibility of accelerating the drug’s path toward human trials. Further details on the revised strategy are expected in the coming weeks.
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### [Tenax Advances Oral Levosimendan in Phase 3 PH-HFpEF Studies](https://www.clinicaltrialvanguard.com/news/tenax-advances-oral-levosimendan-in-phase-3-ph-hfpef-studies/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
Tenax Therapeutics announced the FDA’s acceptance of an amendment to expand its Phase 3 [LEVEL](https://www.clinicaltrialvanguard.com/news/tenax-announces-phase-3-level-trial-topline-results-for-tnx-103-in-ph-hfpef/) study for TNX-103 (oral levosimendan) in patients with pulmonary hypertension associated with heart failure with preserved ejection fraction (PH-HFpEF). The LEVEL study will now enroll 230 patients, increasing its statistical power, and a second global Phase 3 trial, LEVEL-2, will commence in 2025. These parallel studies aim to provide sufficient data for regulatory filings in the U.S. and other regions.
This development is crucial for PH-HFpEF patients, a population currently lacking approved treatments. The expansion of LEVEL and the initiation of LEVEL-2 signal a significant step towards potentially providing a much-needed therapy for this serious condition, offering hope for improved quality of life and addressing a high unmet medical need. The progress made by Tenax Therapeutics with TNX-103 underscores a shift in the landscape for PH-HFpEF management.
The LEVEL study, now targeting 230 patients, is expected to complete enrollment by the end of 2025, with topline data anticipated mid-2026. Interim data from LEVEL shows that TNX-103 is well-tolerated, with high patient retention rates in both the active treatment and open-label extension phases. LEVEL-2, a larger global study, will evaluate patients for a full year, generating a more comprehensive safety database. Both studies are placebo-controlled and evaluate changes in six-minute walk distance, a key measure of functional capacity in these patients.
This two-pronged approach strengthens Tenax Therapeutics’ position in developing a first-in-class therapy for PH-HFpEF. The positive interim data, coupled with the expanded LEVEL study and the launch of LEVEL-2, sets a clear path towards regulatory submission. The expected data readouts in 2026 hold significant potential to transform the treatment paradigm for this underserved patient population.
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### [Journey Medical's Emrosi™ Rosacea Trial Results Published in JAMA Dermatology](https://www.clinicaltrialvanguard.com/news/journey-medicals-emrosi-rosacea-trial-results-published-in-jama-dermatology/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
Journey Medical Corporation announced positive Phase 3 trial results for its rosacea treatment, Emrosi ([DFD-29](https://www.clinicaltrialvanguard.com/news/study-assesses-emrosi-dfd-29-impact-on-microbial-flora/)), published in JAMA Dermatology. The drug, a modified-release minocycline capsule, demonstrated statistically superior efficacy compared to both Oracea (doxycycline) and placebo in reducing inflammatory lesions and erythema. The FDA approved Emrosi in November 2024, with a planned launch in early spring 2025.
This news holds considerable potential to shift the treatment landscape for rosacea. Emrosi’s demonstrated superiority over the current standard of care, Oracea, offers patients a more effective treatment option. The publication of these results in a prestigious journal like JAMA Dermatology adds to the credibility of the findings and may influence dermatologist prescribing patterns. A successful launch could capture a significant share of the existing rosacea market, currently estimated to impact millions of people in the US alone.
In two Phase 3 trials (MVOR-1 and MVOR-2), DFD-29 outperformed both Oracea and placebo on co-primary endpoints of Investigator’s Global Assessment (IGA) treatment success and reduction in inflammatory lesion count. Specifically, in MVOR-1, IGA success rates were 65% for DFD-29, 46.1% for Oracea, and 31.2% for placebo. In MVOR-2, the corresponding rates were 60.1%, 31.4%, and 26.8%, respectively. DFD-29 also demonstrated statistically significant reductions in lesion counts compared to both Oracea and placebo in both studies. Importantly, the safety profile of DFD-29 appeared comparable to existing treatments, with no significant safety concerns reported.
The positive Phase 3 data and FDA approval set the stage for Emrosi’s upcoming launch. Market penetration will depend on factors such as pricing, payer coverage, and clinician acceptance. If Journey Medical effectively communicates Emrosi’s clinical advantages and secures favorable market access, the drug has the potential to become a leading treatment option for rosacea, improving outcomes and quality of life for patients. Furthermore, the success of Emrosi could spur further innovation in the rosacea treatment space, potentially leading to the development of even more effective therapies.
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### [Sernova Secures $1M Convertible Debt Financing](https://www.clinicaltrialvanguard.com/news/sernova-secures-1m-convertible-debt-financing/)
**Published:** March 6, 2025
**Author:** Jon Napitupulu
**Content:**
Sernova [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) secured a CAD$1,000,000 convertible debenture financing from existing shareholder and board member, Dr. Steven Sangha. The proceeds will bolster the company’s working capital. The debenture, repayable by March 4, 2027, has a 15% interest rate and includes warrants exercisable into common shares at CAD$0.20 per share.
This financing is crucial for Sernova as it allows the company to continue operations and advance its research and development efforts without immediately diluting shareholder value. Securing funding from an insider like Dr. Sangha demonstrates confidence in Sernova’s long-term prospects and technology, potentially signaling positive developments to other investors. Strengthening the company’s financial position allows Sernova to focus on achieving key milestones in its clinical trials and further development of its Cell Pouch Bio-hybrid Organ for [type 1 diabetes](https://www.clinicaltrialvanguard.com/news/chinese-team-enables-24-type-1-diabetics-to-stop-insulin/).
The financing involved a CAD$1,000,000 unsecured convertible debenture with a 15% interest rate and 5,000,000 warrants exercisable at CAD$0.20 per share until March 4, 2028. The conversion price for the debenture is also CAD$0.20 per share. This structure provides Dr. Sangha with the potential for future equity ownership while offering Sernova a less dilutive financing option in the short term. The transaction qualifies as a related-party transaction under MI 61-101 but utilizes exemptions from formal valuation and minority shareholder approval requirements due to its size relative to Sernova’s market capitalization.
This injection of capital strengthens Sernova’s financial position and provides runway to pursue its clinical development goals. The insider investment could generate positive momentum and attract further investment, ultimately accelerating the development and potential commercialization of its Cell Pouch technology. This progress toward a “functional cure” for type 1 diabetes holds significant promise for patients and the broader healthcare landscape.
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### [Rhenium Obisbemeda Gets FDA Orphan Drug Designation](https://www.clinicaltrialvanguard.com/news/rhenium-obisbemeda-gets-fda-orphan-drug-designation/)
**Published:** March 7, 2025
**Author:** Jon Napitupulu
**Content:**
Plus Therapeutics received Orphan Drug Designation from the FDA for its Rhenium (¹⁸⁶Re) Obisbemeda, a treatment for leptomeningeal metastases (LM) in [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/) patients. This designation follows the successful completion of the Phase 1 single-dose trial, which identified the recommended Phase 2 dose. The company is also pursuing a Fast Track designation for the drug.
This FDA decision is crucial for patients with LM originating from lung cancer, a debilitating condition with limited effective treatments and a dismal prognosis. The Orphan Drug Designation underscores the unmet medical need and incentivizes further development of Rhenium (¹⁸⁶Re) Obisbemeda, potentially providing a life-extending treatment option for these patients. Furthermore, the increasing incidence of LM makes the development of targeted therapies even more critical.
The Orphan Drug Designation provides Plus Therapeutics with seven years of market exclusivity if the drug is approved, as well as tax credits for clinical trials and exemptions from certain FDA fees. The company is now initiating a Phase 2 single-dose expansion trial and a Phase 1 multiple-dose trial. Plus Therapeutics is actively working with the FDA to finalize the pivotal trial strategy.
This designation significantly de-risks the development of Rhenium (¹⁸⁶Re) Obisbemeda and strengthens its market potential. Coupled with the ongoing clinical trials and collaboration with the FDA, this positions Plus Therapeutics to potentially bring a much-needed therapy to patients battling this devastating complication of lung cancer. It also solidifies the company’s position in the targeted radiotherapeutics market, particularly for central nervous system cancers. The next stage of clinical development will be critical in confirming the drug’s efficacy and safety profile, paving the way for potential regulatory approval and ultimately, improved outcomes for patients with LM.
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### [Clearside Biomedical CLS-AX Phase 3 Wet AMD Trial Announced](https://www.clinicaltrialvanguard.com/news/clearside-biomedical-cls-ax-phase-3-wet-amd-trial-announced/)
**Published:** March 7, 2025
**Author:** Jon Napitupulu
**Content:**
Clearside Biomedical announced positive results from a meeting with the FDA regarding its Phase 3 clinical trial plans for CLS-AX, a treatment for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD). The FDA agreed with Clearside’s proposed Phase 3 program design, signaling a crucial step toward potential approval of this new therapy. CLS-AX, administered via Clearside’s patented suprachoroidal injection technology, offers a potential advantage over existing treatments with its flexible dosing schedule and longer duration of effect.
This development is particularly important for wet AMD patients who require individualized treatment plans. Current therapies often necessitate frequent injections, placing a burden on patients and healthcare providers. A longer-acting treatment like CLS-AX could significantly improve patient convenience and compliance, potentially leading to better outcomes. Furthermore, the potential for reduced injection frequency could streamline treatment protocols, optimizing the use of healthcare resources.
The planned Phase 3 program consists of two concurrent, non-inferiority trials comparing CLS-AX to the current standard of care, aflibercept. The primary endpoint is the average change in best corrected visual acuity (BCVA) at week 52. The trials are designed to support a flexible dosing schedule of CLS-AX every 3 to 6 months, based on individual patient needs. The inclusion of treatment-naïve participants allows for a broader representation of the wet AMD population and potentially faster recruitment.
The positive FDA feedback and well-defined Phase 3 program represent significant progress for Clearside Biomedical. Successful completion of these trials could position CLS-AX as a valuable new treatment option for wet AMD, addressing a significant unmet need in this large and growing market. This positive outcome may also validate Clearside’s suprachoroidal injection platform, paving the way for further development of its pipeline of therapies for other retinal diseases.
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### [Medicus Pharma: Positive Interim Analysis of SKNJC-003 in BCC Treatment](https://www.clinicaltrialvanguard.com/news/medicus-pharma-positive-interim-analysis-of-sknjc-003-in-bcc-treatment/)
**Published:** March 7, 2025
**Author:** Jon Napitupulu
**Content:**
Medicus Pharma (NASDAQ: MDCX) announced positive interim results from its Phase 2 clinical study of [SKNJCT-003](https://www.clinicaltrialvanguard.com/news/medicus-gorlin-alliance-seek-compassionate-use-for-skinject/), a non-invasive treatment for [basal cell carcinoma](https://www.clinicaltrialvanguard.com/news/verrica-presents-new-vp-315-data-for-basal-cell-carcinoma/) (BCC). The interim analysis, conducted after randomizing over half of the targeted 60 patients, revealed a complete clinical clearance rate exceeding 60%. The investigational product, D-MNA, demonstrated good tolerability at both low (100µg) and high (200µg) doses, without serious adverse events or dose-limiting toxicities.
This positive interim data is a significant development in the BCC treatment landscape. Current treatment options for BCC can be invasive, involving surgical excision or other destructive methods. A non-invasive, topical treatment like SKNJCT-003 could offer a more patient-friendly approach, potentially improving treatment adherence and reducing the burden on healthcare systems. The strong clinical clearance rate observed in the interim analysis suggests that SKNJCT-003 could become a valuable addition to the current arsenal against BCC.
The interim analysis shows promising results for D-MNA’s efficacy and safety profile. Over 60% of patients achieved complete clinical clearance, indicating the potential for SKNJCT-003 to effectively eliminate BCC lesions. The absence of serious adverse events and dose-limiting toxicities further strengthens the product’s profile. Medicus plans to submit this interim data to the FDA for a Type C meeting in Q2 2025, aiming to discuss product development and expedite the clinical program, potentially seeking a fast-track designation.
This positive interim analysis positions Medicus to advance SKNJCT-003 through clinical development. A successful Type C meeting with the FDA could lead to an accelerated clinical pathway, potentially shortening the timeline to market. If the final study results confirm the interim findings, SKNJCT-003 could revolutionize BCC treatment, providing a more convenient and effective option for patients. This advancement also bolsters Medicus’s position in the dermatological oncology market, opening doors for further research and development of novel treatments.
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### [Chemomab's Nebakitug Shows Promise in Systemic Sclerosis Treatment](https://www.clinicaltrialvanguard.com/news/chemomabs-nebakitug-shows-promise-in-systemic-sclerosis-treatment/)
**Published:** March 7, 2025
**Author:** Jon Napitupulu
**Content:**
Chemomab Therapeutics announced new scientific data supporting the potential of nebokitug ([CM-101](https://www.clinicaltrialvanguard.com/news/chemomab-announces-positive-nebokitug-phase-2-clinical-data-in-psc-at-bsg-25/)) as a novel treatment for systemic sclerosis (SSc). The data, presented at the 8th International Congress on Controversies in Rheumatology and Autoimmunity (CORA 2025), further validates the mechanism of action of nebokitug, a monoclonal antibody that blocks CCL24, a protein implicated in SSc. This research builds upon existing preclinical and clinical evidence suggesting CCL24 plays a crucial role in the inflammation and fibrosis characteristic of SSc.
This development is particularly important because SSc is a severe, often fatal autoimmune disease with no approved disease-modifying therapies. The research offers hope for patients suffering from this debilitating condition, potentially leading to a treatment that addresses the underlying causes of the disease rather than just managing symptoms. The findings could significantly improve patient outcomes and quality of life, addressing a significant unmet medical need.
The new research, conducted in collaboration with Dr. Alexandra Balbir-Gurman, utilized skin and serum samples from SSc patients and a mouse model to investigate the impact of nebokitug on immune cells. The study identified specific immune cell populations with altered expression of CCR3, the receptor for CCL24, linking them to SSc and its complications. This further solidifies the rationale for targeting CCL24 with nebokitug. The findings also correlate with a 2024 publication demonstrating a link between elevated CCL24 levels and severe SSc manifestations, including skin fibrosis, lung disease, and increased mortality risk. Positive data from Chemomab’s Phase 2 SPRING trial of nebokitug in primary sclerosing cholangitis (PSC), another fibro-inflammatory disease, further supports the drug’s potential in SSc, demonstrating its dual anti-inflammatory and anti-fibrotic mechanism.
This new data strengthens Chemomab’s position in developing a much-needed treatment for SSc. With an open U.S. Investigational New Drug (IND) application for a Phase 2 SSc trial, the company is poised to advance nebokitug’s clinical development. The research provides further impetus for this next stage, suggesting a promising path toward a potential new therapeutic option for SSc patients. Further research and clinical trials will be crucial to validate these findings and ultimately bring this potential therapy to patients.
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### [Study Shows Increased Cardiometabolic Risk After Switching to Integrase Inhibitors](https://www.clinicaltrialvanguard.com/news/study-shows-increased-cardiometabolic-risk-after-switching-to-integrase-inhibitors/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
The AIDS Clinical Trials Group (ACTG) presented data from the REPRIEVE study at the 2025 Conference on Retroviruses and Opportunistic Infections. The study found that switching to an integrase inhibitor-based antiretroviral regimen in people living with HIV increased the risk of weight gain, diabetes, hypertension, and metabolic syndrome over five years, compared to those who didn’t switch. However, the switch did not increase the risk of major adverse cardiovascular events like heart attacks or strokes.
This research is crucial for HIV treatment strategies. While integrase inhibitors are effective antiretroviral drugs, the observed increased risk of metabolic complications necessitates a more comprehensive approach to patient care. Clinicians must now balance the antiviral benefits of these drugs with the potential for long-term metabolic issues. This highlights the need for personalized medicine and consistent monitoring of patients on integrase inhibitor regimens.
The REPRIEVE study involved 2,708 participants who switched to an integrase inhibitor-containing regimen, most of which included dolutegravir. The study revealed statistically significant increases in the risks of [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/), diabetes, hypertension, and metabolic syndrome in those who switched. Though no increased risk of MACE was observed in the five-year follow-up period, continued monitoring is necessary to understand the long-term cardiovascular implications.
This data reinforces the need for further research to investigate the mechanisms behind these metabolic changes and to explore strategies to mitigate these risks. Long-term studies are crucial to fully understand the cardiovascular impact of integrase inhibitors and to guide clinical decisions regarding their use. This information emphasizes the evolving nature of HIV treatment and the ongoing need for proactive patient management strategies.
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### [Late-Breaking ESK-001 Phase 2 Data Presented at AAD Meeting](https://www.clinicaltrialvanguard.com/news/late-breaking-esk-001-phase-2-data-presented-at-aad-meeting/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
Alumis announced positive 52-week data from the open-label extension of its Phase 2 STRIDE trial for [ESK-001](https://www.clinicaltrialvanguard.com/news/alumis-enrolls-final-patient-in-global-phase-2b-trial-of-next-gen-oral-tyk2-inhibitor-for-sle/), an oral TYK2 inhibitor, in moderate-to-severe plaque [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/). Results presented at the 2025 American Academy of Dermatology meeting showed sustained and increasing clinical responses in patients receiving 40mg twice daily. The therapy continued to be well-tolerated with no new safety concerns.
This data strengthens the case for ESK-001 as a potential oral alternative to biologics for psoriasis. Demonstrating sustained efficacy and safety over a year is crucial for establishing the drug’s long-term value proposition, especially in a chronic condition like psoriasis. This data could influence treatment paradigms by offering a convenient and effective option for patients who prefer oral medication or haven’t responded well to other treatments. The sustained improvements in itch and quality of life measures further underscore the drug’s potential impact on patients’ daily lives.
At week 52, PASI 90, PASI 100, and sPGA 0 scores were 61.3%, 38.8%, and 38.8% respectively, compared to 52.4%, 26.8%, and 32.9% at week 12. Control of itch (NRS≤4) was reported in 81.3% of patients, and 61.3% achieved a quality-of-life score of DLQI0/1. The Phase 3 ONWARD program, consisting of two trials (ONWARD1 and ONWARD2), is currently enrolling patients, with topline data anticipated in Q1 2026. A once-daily modified release formulation is also in development.
The positive long-term data and ongoing Phase 3 trials position ESK-001 as a promising candidate in the TYK2 inhibitor landscape for psoriasis. Positive Phase 3 results could lead to regulatory submission and potential market entry, offering a new oral therapeutic option for patients. The development of a once-daily formulation could further enhance patient convenience and adherence, potentially differentiating ESK-001 in a competitive market.
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### [Regeneron and Sanofi: Dupixent® Bullous Pemphigoid Data at AAD](https://www.clinicaltrialvanguard.com/news/regeneron-and-sanofi-dupixent-bullous-pemphigoid-data-at-aad/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
Regeneron and Sanofi announced positive Phase 2/3 trial results for Dupixent ([dupilumab](https://www.clinicaltrialvanguard.com/news/zumilokibart-hits-easi-75-target-in-phase-2-atopic-dermatitis-trial/)) in treating moderate-to-severe bullous pemphigoid (BP), a chronic skin disease. The ADEPT trial showed Dupixent significantly improved sustained disease remission, reduced disease severity and itch, and lessened the need for oral corticosteroids and rescue medication compared to placebo. This is particularly relevant as BP predominantly affects an elderly population for whom current immunosuppressant treatments can be difficult to tolerate.
This news is potentially transformative for BP patients who experience debilitating itch, blisters, and painful lesions. The results suggest Dupixent could offer a targeted treatment option without the broad immune suppression of current therapies, improving quality of life for this vulnerable patient group. The potential for sustained remission is especially important, as it could reduce the long-term burden of BP and minimize the need for ongoing treatment.
The ADEPT trial enrolled 106 adults with moderate-to-severe BP. At 36 weeks, 20% of Dupixent-treated patients achieved sustained disease remission versus 4% in the placebo group. Furthermore, Dupixent demonstrated a 40% achievement rate for both a ≥90% reduction in disease severity and clinically meaningful itch reduction, compared to 10% and 11% in the placebo group, respectively. A decrease in cumulative oral corticosteroid exposure and a lower risk of rescue medication use were also observed. The overall adverse event profile was similar between Dupixent and placebo.
The positive trial data supports regulatory submissions currently under review in the U.S. and EU, with a decision from the FDA expected by June 20, 2025. If approved, Dupixent would be the first targeted therapy for BP, potentially revolutionizing the treatment landscape and offering new hope for patients seeking relief from this challenging condition. This could also signify an expansion of Dupixent’s market reach, further solidifying its position as a key player in treating type 2 inflammatory diseases.
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### [BioXcel's Serenity Trial: At-Home Agitation Treatment](https://www.clinicaltrialvanguard.com/news/bioxcels-serenity-trial-at-home-agitation-treatment/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
BioXcel Therapeutics has reached 33% enrollment (67 patients) in its 200-patient Phase 3 SERENITY At-Home trial for [BXCL501](https://www.clinicaltrialvanguard.com/news/bioxcels-bxcl501-shows-efficacy-across-severity-levels-in-at-home-agitation-trial/) (dexmedetomidine sublingual film). The trial aims to evaluate the safety of the lowest approved dose (120mcg) of BXCL501 for at-home treatment of agitation related to bipolar disorders or schizophrenia. Topline data is anticipated in the latter half of 2025 and will be used to support a potential supplemental New Drug Application (sNDA) to expand the current label of [IGALMI](https://www.clinicaltrialvanguard.com/news/bioxcel-awaits-fda-decision-on-igalmi-at-home-label-expansion/)®, the approved version of BXCL501.
This trial addresses a significant unmet need: an estimated 23 million annual episodes of agitation associated with bipolar disorders or schizophrenia occur at home in the U.S., with no FDA-approved treatments currently available for this specific setting. Demonstrating the safety and efficacy of BXCL501 for at-home use could significantly improve patient care by providing a readily accessible treatment option during these acute episodes, potentially preventing escalation and the need for emergency interventions. This could also alleviate strain on caregivers and the healthcare system.
The SERENITY At-Home trial is a double-blind, placebo-controlled study. Twenty-three clinical trial sites have been activated, and a Data Safety Monitoring Board will assess safety data collected over the 12-week trial period. Patients will self-administer the medication during agitation episodes, and exploratory endpoints include modified global impression of severity (mCGI-S) and change (mCGI-C) assessments two hours post-dose.
Positive results from the SERENITY At-Home trial would represent a substantial step forward for BioXcel Therapeutics, potentially expanding the market for IGALMI® considerably and positioning it as a first-in-class at-home treatment option for agitation. This would not only benefit patients and caregivers but also establish a new standard of care in managing acute psychiatric episodes in the home setting. The trial’s outcome may also influence the development of other at-home treatments for [mental health](https://www.clinicaltrialvanguard.com/news/cybin-announces-groundbreaking-clinical-progress-unveiling-transformative-therapies-for-mental-health/) conditions.
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### [Givastomig Phase 1b Study Sees Accelerated Progress](https://www.clinicaltrialvanguard.com/news/givastomig-phase-1b-study-sees-accelerated-progress/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
I-Mab, a global biotech company, announced the completion of enrollment, ahead of schedule, in the first dose expansion cohort of a Phase 1b study for givastomig. Givastomig is a Claudin 18.2 ([CLDN18](https://www.clinicaltrialvanguard.com/news/astellas-initiates-phase-3-study-of-asp2138-in-cldn18-2-positive-gastric-cancer/).2) x 4-1BB bispecific antibody being studied for first-line treatment of gastric cancer. The first patient in the second expansion cohort has also been dosed, and topline results from the 40-patient dose expansion study are expected in the first half of 2026. Data from the Phase 1b dose escalation study are expected in the latter half of 2025.
This rapid enrollment signifies strong interest in givastomig’s potential to address a critical unmet need in gastric cancer treatment. The combination of CLDN18.2 targeting with 4-1BB activation offers a novel approach, potentially improving efficacy beyond current standard treatments which include chemotherapy and anti-PD-1 checkpoint inhibitors. Early completion of enrollment suggests efficient trial execution and high patient interest, potentially accelerating development timelines. Positive results could pave the way for larger-scale trials and ultimately offer a new treatment option for patients with this aggressive form of cancer.
The Phase 1b study is evaluating givastomig in combination with the current standard of care ([nivolumab](https://www.clinicaltrialvanguard.com/news/fda-fast-tracks-scancells-melanoma-drug-after-phase-2-data/) and chemotherapy) for first-line treatment of gastric cancer. It includes a dose escalation study (n=17, completed) and a dose expansion study (n=40). Previous Phase 1 monotherapy data demonstrated givastomig’s strong tumor-binding properties and anti-tumor activity, along with a tolerable safety profile. I-Mab is co-developing givastomig in a global partnership with ABL Bio, holding worldwide rights excluding Greater China and South Korea.
Successful results from this Phase 1b study could position givastomig as a leading therapy in first-line gastric cancer treatment. This would represent a significant advancement in the field, offering a new and potentially more effective option for patients. The expedited enrollment and positive preliminary data suggest a promising outlook for givastomig’s continued development and potential future approval.
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### [Briumvi Data at AAN 2025 Meeting: TG Therapeutics MS Treatment](https://www.clinicaltrialvanguard.com/news/briumvi-data-at-aan-2025-meeting-tg-therapeutics-ms-treatment/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
[TG Therapeutics](https://www.clinicaltrialvanguard.com/news/tg-therapeutics-subcutaneous-briumvi-meets-phase-1-bioavailability-goals/) announced upcoming presentations of Briumvi ([ublituximab](https://www.clinicaltrialvanguard.com/news/tg-therapeutics-ublituximab-meets-primary-endpoint-in-phase-3-rms-trial/)-xiiy) data for relapsing [multiple sclerosis](https://www.clinicaltrialvanguard.com/news/quantum-allucent-advance-phase-2-multiple-sclerosis-trial/) (RMS) at the American Academy of Neurology 2025 annual meeting. The presentations will cover various aspects of Briumvi, including long-term safety, infusion tolerability, real-world efficacy, and a modified regimen. The data will be presented via posters between April 7th and 9th, 2025.
These presentations are important because they offer further insights into the long-term use and effectiveness of Briumvi in a real-world setting. Data on long-term immunoglobulin levels and serious infections will be crucial for physicians making treatment decisions. Additionally, the exploration of a modified Briumvi regimen could potentially improve treatment outcomes for RMS patients.
The presentations will feature data from various studies, including the ULTIMATE I & II Phase 3 trials’ open-label extension, a real-world observational survey (ENAMOR), the ENABLE Phase 4 observational study, and a study evaluating a modified Briumvi regimen (ENHANCE). The posters will delve into specific topics such as the association between decreased immunoglobulin levels and serious infections, infusion tolerability, and five-year outcomes with Briumvi.
This information is expected to further solidify Briumvi’s position as a treatment option for RMS. Positive results from the real-world studies and the modified regimen could expand Briumvi’s market reach and benefit a wider range of patients. The forthcoming data will be crucial for ongoing assessment of Briumvi’s long-term safety and efficacy.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [MindRank's MRANK-106 Gets FDA Clearance for Oncology Trials](https://www.clinicaltrialvanguard.com/news/mindranks-mrank-106-gets-fda-clearance-for-oncology-trials/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
[MindRank](https://www.clinicaltrialvanguard.com/news/first-patient-dosed-in-mindranks-ai-designed-glp-1-trial/), an AI-driven drug discovery company, has received FDA Investigational New Drug (IND) application clearance for MRANK-106. MRANK-106 is an orally available dual inhibitor of WEE1 and YES1 kinases, intended to treat various cancers, including pancreatic, small cell lung, ovarian, breast, and colorectal cancers. The upcoming Phase I clinical trial will assess its safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity in patients with advanced or metastatic solid tumors.
This IND clearance is a substantial advancement for cancer treatment research. MRANK-106’s dual-targeting mechanism, combined with its preferential distribution in tissues and tumors, addresses limitations of current WEE1 inhibitors by potentially minimizing on-target hematotoxicity and enhancing efficacy. This approach offers a promising new avenue for patients battling these challenging cancers, many of which have limited effective treatment options.
MRANK-106 was developed using MindRank’s AI platforms, Molecule Pro™ and PharmKG™. Preclinical studies indicate the drug exhibits superior efficacy as a single agent and in combination therapies, along with an improved safety profile compared to single-target WEE1 inhibitors. The clinical trial launch, slated for 2025, follows successful preclinical candidate confirmation and represents MindRank’s second clinical-stage pipeline after their GLP-1 program.
The FDA’s clearance signifies a crucial step toward potentially bringing a novel therapeutic option to patients with hard-to-treat cancers. Positive clinical trial results could validate MindRank’s AI-driven drug discovery platform and pave the way for further development and potential approval of MRANK-106, impacting the oncology landscape and offering renewed hope for patients.
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**Categories:** News
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### [AskBio Doses First Limb-Girdle Muscular Dystrophy Patient](https://www.clinicaltrialvanguard.com/news/askbio-doses-first-limb-girdle-muscular-dystrophy-patient/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
[AskBio](https://www.clinicaltrialvanguard.com/news/askbio-gene-therapy-trial-for-heart-failure-begins-in-europe/), a Bayer subsidiary, announced the advancement of its Phase 1/Phase 2 LION-CS101 clinical trial for AB-1003, a gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The first participant in the second cohort has been dosed following a positive safety review by the Data Safety Monitoring Board (DSMB) of the first cohort’s data. The trial is evaluating the safety of AB-1003 in adults with genetically confirmed LGMD2I/R9.
This progress is crucial because LGMD2I/R9 is a rare and debilitating disease with no currently approved treatments. The positive DSMB review and subsequent dosing of the second cohort signify that the therapy is demonstrating an acceptable safety profile, allowing the trial to proceed and potentially bringing a much-needed treatment option closer to reality for patients. This advancement offers hope to individuals and families affected by this disease, which manifests in childhood and progressively worsens, leading to significant mobility limitations and potential heart and lung complications.
The LION-CS101 trial is a double-blind, randomized, placebo-controlled study involving two dose-level cohorts. It will include up to 14 participants across six U.S. sites. AB-1003 is an investigational adeno-associated virus (AAV)-based gene therapy designed to restore FKRP enzyme activity within muscle cells via a single intravenous infusion. AskBio has secured Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the FDA for AB-1003 for the treatment of LGMD2I/R9, underscoring the unmet medical need in this area.
The successful progression of the LION-CS101 trial strengthens the outlook for AB-1003 as a potential treatment for LGMD2I/R9. Positive results from this trial could pave the way for larger studies and eventual regulatory approval, offering a transformative therapeutic option for patients with this devastating disease and establishing AskBio’s leadership in gene therapy for neuromuscular disorders. Continued monitoring of safety and efficacy in the second cohort will be essential for determining the next steps in development.
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---
### [PDS Biotech Launches Phase 3 Trial of Versamune® in HPV16+ Head and Neck Cancer](https://www.clinicaltrialvanguard.com/news/pds-biotech-launches-phase-3-trial-of-versamune-in-hpv16-head-and-neck-cancer/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
PDS Biotechnology Corporation has launched VERSATILE-003, a Phase 3 clinical trial for its immunotherapy drug, Versamune® HPV. The trial will evaluate Versamune® HPV combined with pembrolizumab as a first-line treatment for recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma ([HNSCC](https://www.clinicaltrialvanguard.com/news/promising-results-for-crb-701-in-hnscc-cervical-cancers/)). The trial aims to enroll approximately 350 patients, comparing the combination therapy to pembrolizumab alone.
This Phase 3 trial holds substantial implications for the treatment landscape of HPV16-positive HNSCC. Current treatment options for recurrent/metastatic disease often have limited efficacy, and this trial could lead to a new standard of care offering improved survival and quality of life for these patients. Positive results could significantly impact clinical practice guidelines, offering a new and potentially more effective first-line treatment. The trial’s focus on overall survival as the primary endpoint underscores the potential for a meaningful advancement in patient outcomes.
VERSATILE-003 is a global, multi-center, randomized, and open-label trial. The primary endpoint is overall survival. Secondary endpoints include objective response rate, progression-free survival, disease control rate, and duration of response. Versamune® HPV, delivered subcutaneously, aims to stimulate HPV16-specific T cells. The FDA has granted Fast Track designation to Versamune® HPV, potentially expediting the review process for a future [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/).
The initiation of this Phase 3 trial represents a pivotal step for [PDS Biotech](https://www.clinicaltrialvanguard.com/news/pds-biotech-to-present-new-cancer-therapy-data-at-sitc-2025/) and the potential future of HNSCC treatment. Positive trial results could lead to regulatory approval and commercialization of Versamune® HPV, positioning PDS Biotech as a key player in the immunotherapy space. This advancement may also encourage further research and development into combination immunotherapies for other cancers, ultimately benefiting a wider patient population.
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### [Artax Biopharma Unveils Promising Preclinical Data on NCK Modulators at 2025 AAD Annual Meeting](https://www.clinicaltrialvanguard.com/news/artax-biopharma-unveils-promising-preclinical-data-on-nck-modulators-at-2025-aad-annual-meeting/)
**Published:** March 10, 2025
**Author:** Jon Napitupulu
**Content:**
Artax Biopharma presented preclinical data on Nck modulators for [atopic dermatitis](https://www.clinicaltrialvanguard.com/news/fda-accepts-arcutis-zoryve-application-for-atopic-dermatitis-in-3-month-old-infants/) at the 2025 American Academy of Dermatology Annual Meeting. The data demonstrate the potential of Nck modulation to treat autoimmune skin diseases by regulating T cell activation. Specifically, the research highlighted the reduction of inflammation markers in animal models and the controlled release of cytokines from human immune cells.
This research is a critical step towards developing a new class of treatments for atopic dermatitis and other autoimmune diseases. Current therapies often rely on broad immunosuppression, which can leave patients vulnerable to infections. Nck modulation offers a more targeted approach by selectively impacting the faulty T cell activation responsible for these conditions. This precision could lead to improved efficacy and a better safety profile for patients, potentially transforming how these diseases are managed.
Key findings include a dose-dependent decrease in cytokine production from patient-derived cells exposed to dust mites, a common atopic dermatitis trigger. Furthermore, the oral Nck modulator AX-194 significantly reduced skin inflammation in an animal model. These results build on previous positive Phase 2a data for [psoriasis](https://www.clinicaltrialvanguard.com/news/fda-approves-roflumilast-cream-for-plaque-psoriasis-in-children-age-2/), another autoimmune skin disease, further validating the potential of Nck modulation across a range of these conditions.
These promising preclinical results pave the way for further investigation of Nck modulators in other autoimmune diseases. Artax Biopharma is planning additional studies to explore the potential of this novel mechanism to correct erroneous T cell activation in dermal autoimmune disorders, ultimately aiming to develop much-needed, more targeted therapies. This innovative approach could shift the treatment paradigm for patients suffering from these debilitating conditions.
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**Categories:** News
---
### [Merck on Investigator-Initiated Studies and Budgeting Practices at 2025 SCOPE Summit](https://www.clinicaltrialvanguard.com/conference-coverage/merck-on-investigator-initiated-studies-and-budgeting-practices-at-2025-scope-summit/)
**Published:** March 10, 2025
**Author:** Moe Alsumidaie
**Content:**
The[ 2025 SCOPE Summit’s ](https://connect.healthtech.com/event/scope-summit-for-clinical-ops-executives/plannings/RXZlbnRWaWV3XzEwMDk0NzE=)panel discussion on investigator-initiated studies (IIS) delved into the complexities of budgeting practices, highlighting the growing importance of IIS in clinical research. Industry experts Meghan Harrington, Karen Hartman, and Michael Salvatore Jr. explored the nuances of IIS, the challenges in budgeting, and the collaborative efforts needed to streamline processes. Understanding their role and optimizing financial management becomes crucial for advancing scientific research and patient care as IIS gain traction.
#### [](#understanding-investigator-initiated-studies)Understanding Investigator-Initiated Studies
IISs are pivotal in clinical research, with the investigator or PI at a site taking the lead in initiating and managing the study. Meghan Harrington from Medidata explained that in IIS, the investigator assumes regulatory responsibilities, while industry partners provide financial support and necessary investigational products. This distinction is crucial as it differentiates IIS from collaborative research, where industry partners like Merck might have a more significant role in shaping the scientific objectives and conducting the trial. Karen Hartman from Mayo Clinic added that from a site perspective, the lines between different types of research can blur, emphasizing the need for clear definitions to ensure all parties have a mutual understanding of their roles and responsibilities. Michael Salvatore from Merck highlighted that when a study becomes collaborative, it shifts from being investigator-initiated to a joint effort, with the company having a say in the protocol and trial conduct. This nuanced understanding of IIS is essential for effective collaboration and successful study outcomes.
#### [](#the-growth-and-value-of-iis)The Growth and Value of IIS
The panelists observed a notable increase in the number of IIS over the past decade, with many companies establishing dedicated IIS management offices and submission portals. Michael Salvatore shared that Merck has experienced a significant rise in submissions, particularly after the success of a popular product, which led to thousands of submissions per cycle. This growth reflects a broader industry trend where IIS are increasingly recognized for their scientific value. At Merck, IIS are seen as a means to contribute to the scientific literature and explore research avenues that the company might not pursue internally. This approach allows Merck to support trials that could provide critical treatment options for patients, especially in niche areas. Karen Hartman emphasized that IIS are integral to their research objectives at Mayo Clinic, with about 40% of their active clinical trials being investigator-initiated. These studies offer opportunities for junior and senior researchers to innovate and advance science, often providing potential patient care options through protocols tailored to specific patient populations.
#### [](#budgeting-challenges-and-opportunities)Budgeting Challenges and Opportunities
Budgeting for IIS presents unique challenges, as initial estimates often evolve as protocols are refined. Karen Hartman noted that draft budgets based on preliminary protocols can differ significantly from final budgets, particularly when additional tests and procedures are incorporated. This discrepancy can lead to delays and complications in the budgeting process. To address these challenges, the panelists highlighted the importance of engaging researchers and a centralized approach to budgeting. Michael Salvatore explained that Merck has adopted a phased approach to budget submissions, starting with a feasibility assessment before requesting a detailed proposal. This strategy has reduced approval times and improved efficiency by allowing Merck to focus on the scientific merit of proposals before delving into detailed budget negotiations. The panelists agreed that communication and coordination between PIs and budgeting offices are crucial to avoid delays and ensure accurate budget estimates.
#### [](#evaluating-and-collaborating-with-sites)Evaluating and Collaborating with Sites
Merck employs a rigorous evaluation process for IIS submissions, assessing the scientific rationale, institutional capabilities, and feasibility of proposed studies. Michael Salvatore described this process as similar to an NIH-style scoring system, which helps Merck objectively prioritize studies that align with their strategic objectives. This evaluation considers the institution’s track record, the scientific soundness of the proposal, and the feasibility of conducting the study within the proposed timelines. The panelists discussed the importance of transparency and communication between sponsors and sites to ensure successful collaboration. Karen Hartman emphasized the need for coordination between PIs and budgeting offices to avoid discrepancies in budget estimates and ensure that all parties clearly understand the study’s requirements. This collaborative approach is essential for overcoming challenges and maximizing the potential of IIS to contribute to scientific knowledge and patient care.
#### [](#streamlining-budget-assessment-with-technology)Streamlining Budget Assessment with Technology
Merck and Medidata have collaborated to streamline the budget assessment process for IIS and to develop standardized budget templates and leverage predictive analytics. This initiative aims to modernize data and provide fair market value assessments, reducing the back-and-forth negotiations between sponsors and sites. Michael Salvatore shared the challenges of dealing with diverse budget formats from various institutions, which previously slowed down the approval process. By adopting a formalized template, Merck has alleviated bottlenecks and improved efficiency in budget assessments. Medidata’s efforts to incorporate real-time data, inflation adjustments, and country-specific ratios into their site payments application have further enhanced the accuracy and fairness of budget negotiations. This technological advancement reflects a broader industry trend toward using data-driven insights to optimize clinical trial processes and ensure that budget negotiations start from a fair and informed position.
#### [](#summary)Summary
The insights from the 2025 [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Summit highlight the growing importance of investigator-initiated studies in the clinical research landscape. By addressing budgeting challenges and fostering collaboration between sponsors and sites, the industry can enhance the efficiency and impact of IIS, ultimately advancing scientific knowledge and patient care. As IIS continues to gain traction, the lessons learned from this summit will be instrumental in shaping future practices and policies in clinical research.
**Categories:** Article: Conference Coverage
---
### [Calidi Delivers Transient Gene Therapy to Tumors](https://www.clinicaltrialvanguard.com/news/calidi-delivers-transient-gene-therapy-to-tumors/)
**Published:** March 11, 2025
**Author:** Jon Napitupulu
**Content:**
Calidi [Biotherapeutics](https://www.clinicaltrialvanguard.com/news/sonnet-biotherapeutics-son-1010-improved-solid-tumor-treatment/) announced preclinical success with its systemic RTNova platform, demonstrating effective delivery of gene therapy payloads to targeted tumors and killing over 60 tumor cell lines. The platform utilizes a modified vaccinia virus enveloped in a human cell membrane, enabling systemic delivery and targeted tumor destruction while also acting as a viral vector for gene therapy. Notably, a single dose of their tumor-selective “3KO” RT virus with a specific immunotherapeutic payload significantly altered the tumor microenvironment, leading to the complete eradication of certain tumors in preclinical models.
This development is particularly exciting because it addresses the limitations of intratumoral administration for advanced metastatic cancers. The ability to systemically deliver both oncolytic viruses and gene therapy payloads opens new avenues for treating cancers previously difficult to reach, like late-stage [lung cancer](https://www.clinicaltrialvanguard.com/news/akeso-doses-first-patient-in-ak138d1-ivonescimab-phase-ib-ii-lung-cancer-study/). The documented shift in immune composition within the tumor microenvironment further suggests the potential for long-term anti-tumor immunity. This could translate to more durable responses and potentially even prevent metastatic disease, a significant advancement in cancer treatment.
Technically, the RTNova platform combines the tumor-killing ability of oncolytic viruses with the targeted delivery of gene therapy. The use of a human cell membrane envelope allows the virus to evade the immune system and reach distant tumors. The observed 80-92% leukocyte infiltration in treated groups compared to 46% in the untreated group highlights the potent immunomodulatory effect of the combined therapy. This substantial increase in immune cell presence within the tumor strongly suggests a robust anti-tumor response.
This breakthrough positions Calidi to develop multiple assets for various cancer types. The platform’s versatility offers the potential for personalized treatments tailored to specific tumor profiles and genetic payloads. This also creates promising opportunities for collaborations and partnerships to further explore and expand the clinical applications of this technology, accelerating its development and potential impact on cancer patients.
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**Categories:** News
**Tags:** eClinical Tech News
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### [4DMT Announces First Patients in Phase 3 Trial of 4D-150 for Wet AMD](https://www.clinicaltrialvanguard.com/news/4dmt-announces-first-patients-in-phase-3-trial-of-4d-150-for-wet-amd/)
**Published:** March 11, 2025
**Author:** Jon Napitupulu
**Content:**
4D Molecular Therapeutics (4DMT) has initiated its Phase 3 clinical trial, 4FRONT-1, for 4D-150, a potential treatment for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD). The trial aims to evaluate the efficacy and safety of 4D-150 compared to the current standard treatment, aflibercept. The company expects to release topline data from this trial and a second Phase 3 trial, 4FRONT-2, in the latter half of 2027.
This advancement is potentially transformative for wet AMD treatment. Current treatments require frequent intravitreal injections, impacting patient quality of life and creating a significant burden on healthcare systems. 4D-150 is designed for sustained delivery of anti-VEGF therapy with a single injection, potentially reducing the frequency of injections and improving the patient experience. This could lead to better patient compliance and potentially improved visual outcomes, while also freeing up physician and healthcare resources.
The 4FRONT-1 trial is a randomized, double-masked study comparing 4D-150 to aflibercept 2 mg administered every eight weeks. The primary endpoint is non-inferiority in best corrected visual acuity at 52 weeks. A key secondary endpoint focuses on the reduction in the number of injections needed over the 52-week period. The 4FRONT-2 trial, slated to begin in Q3 2025, will have a similar design but will include both treatment-naïve and previously treated patients globally.
The progression of 4D-150 into Phase 3 represents a significant milestone for 4DMT. Positive results from these trials could lead to regulatory approval and establish 4D-150 as a leading therapy for wet AMD. This would not only offer a more convenient and potentially more effective treatment option for patients but also position 4DMT for substantial growth in a large and growing market. The potential for a durable, single-injection treatment could reshape the wet AMD treatment landscape and significantly impact the lives of millions affected by this debilitating disease.
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**Categories:** News
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### [ACTG Reveals Promising SLIM Liver Findings at CROI](https://www.clinicaltrialvanguard.com/news/actg-reveals-promising-slim-liver-findings-at-croi/)
**Published:** March 11, 2025
**Author:** Jon Napitupulu
**Content:**
The AIDS Clinical Trials Group (ACTG) presented findings from their SLIM LIVER study at the 2025 Conference on Retroviruses and Opportunistic Infections (CROI). The study investigated the use of [semaglutide](https://www.clinicaltrialvanguard.com/news/vivani-medical-completes-dosing-in-phase-1-trial-of-semaglutide-implant/), a medication commonly used for weight loss and diabetes, to treat metabolic dysfunction-associated steatotic liver disease ([MASLD](https://www.clinicaltrialvanguard.com/article/intel-brief/litmus-changes-the-biopsy-calculus-what-the-nature-medicine-data-means-for-your-masld-protocol/)) in people living with HIV. Researchers discovered that epigenetic biomarkers, specifically those related to muscle function and aging, could predict the effectiveness of semaglutide in reducing liver fat.
This research is crucial for individuals living with HIV who are at higher risk for developing MASLD and experiencing accelerated liver damage compared to those without HIV. Identifying predictive biomarkers allows clinicians to personalize treatment strategies, potentially improving outcomes and reducing the burden of liver disease in this vulnerable population. Furthermore, understanding the relationship between epigenetic markers and treatment response could lead to the development of targeted interventions and improved monitoring tools.
The SLIM LIVER study was the first to demonstrate semaglutide’s efficacy in improving and even resolving MASLD in people with HIV. The secondary analysis presented at CROI revealed that participants with lower epigenetic age, particularly those with a lower DNAmGripmax score (indicating weaker muscle function at a biological level), showed greater reductions in liver fat after semaglutide treatment. Additional findings presented at the conference explored the effects of semaglutide on gut bacteria, [heart health](https://www.clinicaltrialvanguard.com/news/unveiling-healthcare-gaps-and-unmet-needs-daiichi-sankyos-devotion-to-heart-health/), metabolism, and liver inflammation in people with HIV.
This research opens new avenues for personalized medicine in the treatment of MASLD for people living with HIV. The ability to predict treatment response using readily available blood markers could significantly improve patient care and resource allocation. Further research is needed to validate these findings in larger populations and explore the potential of other epigenetic biomarkers in predicting treatment outcomes. This could ultimately lead to more effective and tailored therapies for MASLD and contribute to improved long-term health outcomes for people living with HIV.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Neflamapimod Shows Promise in Dementia with Lewy Bodies Study](https://www.clinicaltrialvanguard.com/news/neflamapimod-shows-promise-in-dementia-with-lewy-bodies-study/)
**Published:** March 11, 2025
**Author:** Jon Napitupulu
**Content:**
CervoMed announced positive 16-week results from the extension phase of its Phase 2b RewinD-LB study of neflamapimod for dementia with Lewy bodies (DLB). A new batch of neflamapimod capsules, addressing prior bioavailability issues, showed significant improvement in cognitive function and a lower incidence of falls compared to the old capsules and placebo. The positive data reinforces the potential of neflamapimod as a treatment for DLB, a disease currently lacking approved therapies.
This news holds substantial promise for DLB patients, who currently face limited treatment options. The observed improvements in cognitive function, measured by the Clinical Dementia Rating Sum of Boxes (CDR-SB) and the [Alzheimer’s Disease](https://www.clinicaltrialvanguard.com/news/cytodyn-starts-phase-2a-trial-of-leronlimab-in-early-alzheimers-disease/)[Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) Disease Cooperative Study – Clinical Global Impression of Change (CGIC), are clinically meaningful. Furthermore, the reduction in falls, a significant concern for DLB patients, suggests a potential improvement in their overall well-being and quality of life. This progress underscores the urgent need for effective DLB treatments and positions neflamapimod as a potential game-changer.
The study’s extension phase involved 149 participants, with 94 receiving the new neflamapimod capsules. These participants demonstrated statistically significant improvement (p<0.001) on the CDR-SB compared to those receiving the old capsules. Analysis against the placebo group from the initial phase also revealed significant improvement (p=0.003). The CGIC score, indicating disease worsening, improved with the new capsules (p=0.035). Importantly, the new capsules were associated with a lower incidence of falls compared to both old capsules and placebo. Pharmacokinetic analysis confirmed the new capsules achieved higher plasma drug concentrations, validating the hypothesis that previous results were hampered by bioavailability issues. The positive results from the extension phase pave the way for CervoMed to finalize its Phase 3 plans. With the completion of the full 32-week extension phase later this year and subsequent discussions with regulatory authorities, neflamapimod could advance towards becoming a much-needed treatment option for DLB patients. This progress represents a significant step forward in addressing this debilitating disease.
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**Categories:** News
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### [Citeline's Strategic Vision: Bridging Clinical Trials and Real-World Healthcare](https://www.clinicaltrialvanguard.com/executiveinterviews/citelines-strategic-vision-bridging-clinical-trials-and-real-world-healthcare/)
**Published:** March 11, 2025
**Author:** Moe Alsumidaie
**Content:**
In this discussion with Claire Riches, a veteran in clinical development, we explore Citeline’s strategic initiatives. As a leader in pharma intelligence, Citeline is redefining how clinical trials integrate with real-world healthcare. Claire shares insights on leveraging data and AI to enhance patient-focused solutions and maintain industry leadership amid evolving challenges.
## [](#moe-how-does-citelines-leadership-influence-its-mission-to-connect-treatments-with-patients-and-enhance-evidence-based-practices)Moe: How does Citeline’s leadership influence its mission to connect treatments with patients and enhance evidence-based practices?
Citeline’s leadership is crucial in merging clinical trial landscapes with real-world healthcare data. I ensure trials are not isolated but strategically aligned with existing healthcare practices. By understanding standard care and patient diagnosis trends, we help design relevant and effective trials. This approach enhances the accessibility of clinical research to patients and ensures sponsors and clinical trial sites operate within a framework reflecting real-world conditions. Our mission is to ensure clinical trials are practical solutions that fit seamlessly into the healthcare ecosystem, not just theoretical exercises. This integration is vital for making clinical research accessible to patients, sponsors, and clinical trial sites, ensuring trials are relevant and effective in real-world settings.
Claire Riches, Vice President, Clinical Solutions, Citeline
## [](#moe-how-does-citelines-global-team-of-experts-contribute-to-strategic-objectives-and-industry-reputation)Moe: How does Citeline’s global team of experts contribute to strategic objectives and industry reputation?
Our team, including clinical SMEs, data scientists, and AI specialists, is key to maintaining Citeline’s reputation as a leader in pharma intelligence. For over 20 years, our products have set the gold standard. With increasing trial complexity and economic challenges, our experts focus on accelerating drug development. We create solutions addressing the industry’s pressing questions by leveraging extensive experience and collaborating with data analysts and AI developers. This collaboration helps clients navigate economic and geopolitical challenges, reducing costs and improving market access for new drugs. Our team ensures our products remain the industry’s gold standard, addressing the most pressing questions and challenges.
## [](#moe-how-does-citelines-commitment-to-patient-focused-solutions-shape-its-corporate-culture-and-decision-making)Moe: How does Citeline’s commitment to patient-focused solutions shape its corporate culture and decision-making?
At Citeline, the patient is central to every decision. This patient-centric approach begins with asset acquisition and extends through clinical trial planning. We ensure strategies align with patient needs and market demands by starting with a detailed patient profile. This approach prevents developing drugs that lack a viable patient population or face market saturation. For example, we help clients identify unmet needs and avoid saturated markets, enhancing portfolio success. Our commitment to patient-focused solutions is a core aspect of our corporate culture, driving decision-making processes. This ensures our strategies align with patient needs and market demands, enhancing the success of our clients’ portfolios.
## [](#moe-what-strategies-has-citeline-employed-to-maintain-its-position-amid-evolving-industry-challenges)Moe: What strategies has Citeline employed to maintain its position amid evolving industry challenges?
In response to the demand for faster, more efficient drug development, Citeline has expanded data sources to include proprietary and real-world data. Combining these with AI-driven insights provides clients with actionable recommendations, reducing manual effort and increasing confidence in strategic decisions. This data-driven approach is crucial in today’s rapidly changing landscape, where the pressure to do more with less is ever-present. Post-[COVID](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/), the industry has seen the potential for rapid drug development, and Citeline is at the forefront of providing the data and insights needed to achieve this efficiently. Our strategies focus on leveraging data and AI to enhance efficiency and reliability for our clients.
## [](#moe-how-does-citelines-leadership-ensure-alignment-between-organizational-goals-and-market-opportunities)Moe: How does Citeline’s leadership ensure alignment between organizational goals and market opportunities?
Our alignment is driven by continuous engagement with clients. We ensure our products address real industry needs through publications, market research, and direct feedback. The shift towards data-driven insights and solutions is a global trend, and we are committed to providing tools that enhance efficiency and reliability. By seeking 360-degree feedback and conducting field testing, we ensure our products are innovative, practical, and relevant to our clients’ challenges. This engagement ensures our products address real industry needs and align with market opportunities, maintaining our leadership in the rapidly changing biopharmaceutical landscape.
## [](#moe-how-does-citelines-emphasis-on-data-analysis-impact-collaborations-within-the-healthcare-sector)Moe: How does Citeline’s emphasis on data analysis impact collaborations within the healthcare sector?
We recognize the importance of diverse data sources and have partnered with various providers. These collaborations allow us to offer a 360-degree view of the clinical trial landscape, enhancing our ability to provide comprehensive insights. By continuously seeking new partnerships, we ensure our data sets remain robust and our perspectives diverse. For example, our partnerships around patient data and study start-up metrics enable us to provide a more nuanced view of the clinical trial landscape, ultimately benefiting our clients and the broader healthcare sector. These collaborations enhance our ability to provide comprehensive insights and maintain our leadership in the industry.
## [](#moe-is-there-anything-else-youd-like-to-add-about-the-current-state-of-the-industry)Moe: Is there anything else you’d like to add about the current state of the industry?
The industry is pivotal, with technology and data playing a more significant role than ever. Post-COVID, there’s been a push towards adopting AI and data-driven solutions, which are now delivering tangible value. It’s an exciting time, with the industry showing a strong willingness to innovate and evolve, doing more with less while leveraging unprecedented data and technology resources. The confidence in AI and data-driven approaches has grown significantly, and it’s encouraging to see the industry embracing these advancements to improve outcomes and efficiency. This willingness to innovate and evolve is crucial for the industry’s future success.
**Categories:** Article: Executive Interviews
---
### [Transforming Clinical Trials with AI: Insights from the Frontline](https://www.clinicaltrialvanguard.com/executiveinterviews/transforming-clinical-trials-with-ai-insights-from-the-frontline/)
**Published:** March 11, 2025
**Author:** Moe Alsumidaie
**Content:**
In the rapidly evolving landscape of clinical trials, technology is a game-changer. Dr. Michelle Papka, Ph.D., Founder and President of The Cognitive And Research Center and a leader in [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) research, discusses the integration of AI to enhance participant recruitment and engagement. Alongside Tran Le, CEO of Grove AI, they explore the transformative potential of AI, focusing on the implementation of Grove’s AI tool, Grace, and its impact on recruitment processes.
## [](#moe-how-does-an-ai-digital-staff-like-groves-grace-enhance-recruitment-and-engagement-in-trials)**Moe: How does an AI digital staff like Grove’s Grace enhance recruitment and engagement in trials?**
Michelle Papka: I’ve recently started using Grove’s digital staff, Grace, and in just over 30 days, we’ve doubled our participant screenings. Grace performs the work of four staff members, significantly increasing our reach and reducing the need for additional staff. Initially, we’re using it with a Facebook campaign, but we plan to expand its use to other databases and collaborations with healthcare organizations. This expansion will further increase our recruitment bandwidth. For instance, by integrating AI with our existing patient database and collaborating with referring physicians, we can streamline the recruitment process, making it more efficient and less reliant on human resources.
Tran Le (CEO) and Sohit Gatiganti (CTO) of Grove AI
## [](#moe-how-does-ai-help-ensure-inclusivity-and-diversity-in-clinical-trials)**Moe: How does AI help ensure inclusivity and diversity in clinical trials?**
Michelle Papka: AI is crucial in engaging a diverse population, especially in cognitive impairments trials. The technology is designed to interact with participants to accommodate their needs, such as speaking at a slower pace or handling repetitive questions. Additionally, Grace supports multiple languages, which helps reach participants from various cultural and socio-economic backgrounds. This adaptability is essential for maintaining inclusivity in our trials. For example, Gracecan switch languages mid-conversation, ensuring that participants feel comfortable and understood, which is vital for accurate data collection and participant retention.
## [](#moe-can-ai-generated-transcripts-help-create-predictive-models-for-patient-engagement)**Moe: Can AI-generated transcripts help create predictive models for patient engagement?**
Tran Le: The data collected through our AI interactions is analyzed to predict patient engagement, qualification, and satisfaction rates. This predictive capability helps our partners understand participant behavior and improve trial outcomes. We’re continuously exploring innovative ways to leverage this data for better trial management.
## [](#moe-how-do-you-adopt-innovative-technologies-while-maintaining-compliance)**Moe: How do you adopt innovative technologies while maintaining compliance?**
Tran Le: Our team has extensive experience building [health tech](https://www.clinicaltrialvanguard.com/news/allez-healths-60-million-capital-raise-a-monumental-breakthrough-in-health-tech/) tools, ensuring compliance with regulatory standards is a priority from the outset. We maintain HIPAA compliance, encrypt all data, and work closely with IRBs and regulatory bodies. Our cautious approach to scaling and monitoring ensures we meet all necessary regulations while implementing new technologies. This meticulous attention to compliance protects participant data and builds trust with stakeholders, which is crucial for the successful adoption of AI in clinical trials.
## [](#moe-what-challenges-prompted-you-to-adopt-ai-technology-for-recruitment)**Moe: What challenges prompted you to adopt AI technology for recruitment?**
Michelle Papka: In Alzheimer’s research, we face high screen failure rates, necessitating a large pool of potential participants. Grove AI helps us efficiently manage this by quickly engaging and filtering candidates. Additionally, staffing challenges in the clinical trial industry make it difficult to maintain the necessary workforce. Grove AI allows us to optimize the use of our specialized staff, focusing their efforts on qualified leads, which is crucial for effective recruitment. For example, Grace can quickly identify and eliminate non-viable leads, allowing our team to concentrate on high-potential candidates, thus maximizing our recruitment efforts.
## [](#moe-how-do-you-measure-the-success-of-implementing-these-novel-technologies)**Moe: How do you measure the success of implementing these novel technologies?**
Michelle Papka: Success is measured by the number of randomizations, which is the ultimate goal. We track metrics from the top to the bottom of the recruitment funnel, assessing how quickly and effectively we can move participants through the process. Feedback from participants and staff also plays a vital role in evaluating the success of these technologies. By analyzing these metrics, we can make informed decisions about where to allocate resources, ensuring that our recruitment strategies are practical and efficient.
**Categories:** Article: Executive Interviews
---
### [Revolutionizing Clinical Trials with AI and Decentralization](https://www.clinicaltrialvanguard.com/executiveinterviews/revolutionizing-clinical-trials-with-ai-and-decentralization/)
**Published:** March 11, 2025
**Author:** Moe Alsumidaie
**Content:**
In the evolving world of clinical trials, AI and decentralization are key drivers of change. Iddo Peleg, CEO of Yonalink, shares insights on how these innovations enhance trial efficiency, reduce costs, and improve patient recruitment. Peleg discusses the challenges and successes of implementing AI-driven solutions and the future of decentralized trials, offering a glimpse into the transformative potential of these advancements.
## [](#moe-you-explored-ai-in-trials-can-you-share-data-or-cases-showing-ais-impact-on-efficiency-cost-and-recruitment)**Moe: You explored AI in trials. Can you share data or cases showing AI’s impact on efficiency, cost, and recruitment?**
Indeed, in my experience, the data streaming from Electronic Health Records (EHR) to Electronic Data Capture (EDC) systems exemplifies AI’s transformative power. Traditionally, this data transfer was manual, consuming for example, around 200 hours per patient in [oncology trials](https://www.clinicaltrialvanguard.com/news/mindranks-mrank-106-gets-fda-clearance-for-oncology-trials/). With AI, this process takes less than an hour and has significantly higher accuracy. For instance, in pilot studies conducted globally, including sites in Israel and the U.S., we reduced data transfer time from 6-8 weeks to less than a day. This cuts costs and accelerates the entire trial process, allowing for quicker decision-making. The AI-driven automation ensures that data is harmonized and filtered efficiently, eliminating human error and enhancing the overall quality of the trial data. This transformation is crucial for sponsors who need reliable and timely data to make informed decisions, ultimately leading to faster and more effective clinical trials.
Iddo Peleg, CEO of Yonalink
## [](#moe-decentralized-trials-are-popular-but-adoption-varies-what-evidence-shows-their-impact-on-success-adherence-and-compliance)**Moe: Decentralized trials are popular, but adoption varies. What evidence shows their impact on success, adherence, and compliance?**
While complete decentralization remains rare, hybrid models are gaining traction. These models integrate smaller medical centers into the trial process, especially in rural areas. This approach significantly increases access to clinical trials, available to less than 7% of Americans. By bringing trials closer to patients, we enhance participation and adherence. For example, in the U.S., we are working to include smaller clinics outside major urban centers, effectively turning them into satellite sites that contribute to the main trial, thus improving protocol adherence and compliance. This model broadens the reach of clinical trials and ensures that diverse patient populations are represented, leading to more comprehensive and inclusive research outcomes. The hybrid approach balances the benefits of decentralization with the need for centralized oversight, ensuring that trials remain compliant with regulatory standards while maximizing patient engagement.
## [](#moe-many-claim-seamless-ehr-to-edc-integration-can-you-share-a-real-world-example-of-resolving-data-transfer-inefficiencies)**Moe: Many claim seamless EHR to EDC integration. Can you share a real-world example of resolving data transfer inefficiencies?**
In my experience, successful EHR to EDC integration requires addressing four key challenges: data extraction, harmonization, filtering, and transfer. Our approach is flexible, allowing us to work independently or with partners. For instance, we compared manual data capture with automated transfer in a pediatric oncology trial at Stanford. The automated system improved accuracy and significantly reduced time and effort. This trial demonstrated that we could achieve seamless data integration with minimal interaction from medical centers, setting a new standard for efficiency and reliability in clinical trials. Automating these processes eliminates the bottlenecks associated with manual data entry, ensuring that data is transferred quickly and accurately. This enhances the data’s quality and frees up valuable resources that can be redirected towards more critical aspects of the trial, such as patient care and protocol optimization.
## [](#moe-with-ai-in-trial-decision-making-what-safeguards-ensure-unbiased-patient-selection-and-protocol-optimization)**Moe: With AI in trial decision-making, what safeguards ensure unbiased patient selection and protocol optimization?**
From my perspective, accuracy is paramount in AI-driven systems. We encourage sponsors to verify our system’s accuracy through initial 100% source data verification (SDV) for the first patient, gradually reducing as confidence builds. This ensures transparency and trust. Additionally, site feasibility now includes criteria for data streaming capabilities, ensuring sites can handle automated data transfers effectively. This approach mitigates bias and ensures that the data collected is reliable and actionable, paving the way for more informed decision-making in trial design and execution. By implementing these safeguards, we ensure that AI is used responsibly and ethically, maintaining the integrity of the trial process. Our commitment to accuracy and transparency builds confidence among stakeholders, fostering a collaborative environment where innovation can thrive without compromising patient safety or data quality.
## [](#moe-how-should-the-industry-balance-data-interoperability-compliance-and-patient-autonomy-in-clinical-research)**Moe: How should the industry balance data interoperability, compliance, and patient autonomy in clinical research?**
In my view, this is a complex issue, as regulations vary globally. In the U.S., we leverage the 21st Century Cures Act, where patients consent and establish data connections. Internationally, we work with medical centers to ensure compliance. For example, in Israel, we have successfully implemented a system where the study coordinator establishes the connection, providing compliance and scalability. Our system’s flexibility allows us to adapt to different regulatory environments, ensuring patient autonomy is respected while maintaining data integrity and interoperability. By prioritizing patient consent and involvement, we empower individuals to take control of their health data, fostering trust and engagement in the research process. This approach aligns with regulatory requirements and enhances the overall quality and reliability of the data collected, ultimately leading to more effective and patient-centered clinical trials.
## [](#moe-are-traditional-kpis-like-enrollment-speed-and-data-quality-the-best-measures-of-trial-success-or-should-we-consider-alternatives)**Moe: Are traditional KPIs like enrollment speed and data quality the best measures of trial success, or should we consider alternatives?**
In my opinion, while traditional KPIs like enrollment speed and data quality are essential, the focus should shift to the percentage of data points captured electronically, as this ensures accuracy. In oncology trials, we achieve 85-88% electronic data streaming, aiming for over 90%. This shift in metrics better reflects the quality and efficiency of modern trials. By prioritizing electronic data streaming, we can ensure that the data is accurate and timely, providing a more comprehensive view of trial success. This approach allows us to identify and address potential issues early in the trial process, improving overall outcomes and reducing the risk of costly delays. By embracing new metrics that reflect the realities of modern clinical research, we can drive innovation and improve the efficiency and effectiveness of clinical trials, ultimately benefiting patients and sponsors alike.
## [](#moe-is-there-anything-else-youd-like-to-add)**Moe: Is there anything else you’d like to add?**
I believe it’s crucial to adopt a new mindset in clinical trials. We have a responsibility to patients to bring therapies to market faster. Embracing innovation and data streaming is essential. This change is happening in other industries and must extend to clinical trials to improve outcomes and efficiency. By fostering a culture of innovation and collaboration, we can overcome the traditional barriers that have hindered progress and ensure that clinical trials are conducted in a way that is both efficient and patient-centric. It’s time for the industry to move beyond outdated practices and embrace the potential of new technologies to transform how we conduct research. By doing so, we can accelerate the development of new therapies and improve patients’ lives.
**Categories:** Article: Executive Interviews
---
### [CLENE CNM-Au8® Shows ALS Survival Benefit](https://www.clinicaltrialvanguard.com/news/clene-cnm-au8-shows-als-survival-benefit/)
**Published:** March 13, 2025
**Author:** Jon Napitupulu
**Content:**
Clene Inc. announced positive results from a new analysis of the HEALEY ALS Platform Trial data. The analysis showed that CNM-Au8® 30 mg significantly improved survival in ALS patients, particularly those with moderate to severe disease and those meeting the criteria for Clene’s upcoming Phase 3 [RESTORE](https://www.clinicaltrialvanguard.com/news/nervgen-prepares-restore-phase-3-trial-in-chronic-tetraplegia-with-nvg-291/)-ALS trial. The results support earlier findings from the HEALEY trial, the Phase 2 RESCUE-ALS trial, and Expanded Access Programs, all suggesting a survival benefit with CNM-Au8 treatment compared to historical controls.
This news is potentially groundbreaking for ALS patients, who currently face limited treatment options and a devastating prognosis. The observed survival benefit, especially the substantial extension observed in specific patient subgroups, offers a glimmer of hope for improved outcomes and quality of life. These findings could reshape the ALS treatment landscape if confirmed in the upcoming Phase 3 trial.
The analysis compared CNM-Au8 30 mg to Regimen A of the HEALEY trial. Across the full analysis set, median survival increased by 6.5 months and restricted mean survival time (RMST) improved by 4.1 months. In a subset of patients with moderate to severe ALS, median survival increased by 11.9 months, with a 44% reduction in mortality risk. The most dramatic benefit was seen in patients meeting the RESTORE-ALS criteria, where median survival increased by 14.8 months and mortality risk decreased by 49%.
The positive results from this new analysis strengthen the rationale for the upcoming Phase 3 RESTORE-ALS trial. This trial is crucial for confirming the efficacy and safety of CNM-Au8, potentially paving the way for regulatory approval and widespread availability of this promising therapy. If the Phase 3 trial replicates these findings, CNM-Au8 could become a cornerstone of ALS treatment, significantly altering the disease trajectory for many patients.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Soquelitinib in Phase 2 Trial for ALPS](https://www.clinicaltrialvanguard.com/news/soquelitinib-in-phase-2-trial-for-alps/)
**Published:** March 13, 2025
**Author:** Jon Napitupulu
**Content:**
The National Institute of Allergy and Infectious Diseases ([NIAID](https://www.clinicaltrialvanguard.com/news/niaid-awards-14m-for-institutional-review-services-to-advarra/)), part of the NIH, has launched a Phase 2 clinical trial of [Corvus Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/corvus-pharmaceuticals-2024-financial-results/)‘ [soquelitinib](https://www.clinicaltrialvanguard.com/news/soquelitinib-ind-approved-for-atopic-dermatitis-trial-in-china/) for autoimmune lymphoproliferative syndrome (ALPS). This rare genetic disorder, often manifesting in early childhood, causes a buildup of T cells due to a faulty Fas protein, leading to immune system disruption and serious health risks like lymphoma and autoimmune diseases. The trial, led by Dr. V. Koneti Rao of the NIH, will explore soquelitinib’s potential to inhibit ITK, an enzyme crucial for T cell function, and restore immune balance.
This trial represents a significant advancement in ALPS treatment. Currently, ALPS management relies on addressing symptoms with corticosteroids and immunosuppressants, or even cancer therapies for lymphoma, but there is no cure. Soquelitinib’s targeted approach to correcting the underlying immune dysregulation offers a potentially transformative treatment option for patients who currently face lifelong management of a debilitating disease. The collaboration between the NIH and Corvus leverages the expertise of both institutions, increasing the likelihood of successful development and ultimately providing a much-needed targeted therapy.
The Phase 2 trial will enroll up to 30 patients aged 16 and older with genetically confirmed ALPS-FAS. Patients will receive either 200 mg or 400 mg of soquelitinib twice daily for up to 360 days. The primary endpoint focuses on reductions in spleen and lymph node sizes, key indicators of ALPS severity. Secondary endpoints assess improvements in cytopenias (low blood counts) and overall safety and tolerability. This trial adds to the ongoing investigations of soquelitinib in a Phase 3 trial for peripheral T cell lymphoma and a Phase 1 trial for atopic dermatitis, further demonstrating the drug’s potential across a range of immune-related conditions.
The initiation of this Phase 2 trial marks a crucial step toward a potential new therapeutic avenue for ALPS. Positive results could lead to the first targeted treatment for this rare disease, significantly improving the quality of life for patients. Furthermore, the findings could broaden our understanding of ITK inhibition and its applications in treating other immune disorders, paving the way for further advancements in immunotherapy.
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**Categories:** News
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### [PDS Biotech gets FDA Clearance for Trial on Metastatic Colorectal Cancer Combo Therapy](https://www.clinicaltrialvanguard.com/news/pds-biotech-gets-fda-clearance-for-trial-on-metastatic-colorectal-cancer-combo-therapy/)
**Published:** March 14, 2025
**Author:** Jon Napitupulu
**Content:**
PDS Biotechnology Corporation has received FDA clearance for its Investigational New Drug (IND) application to evaluate a combination therapy for MUC1-positive metastatic [colorectal cancer](https://www.clinicaltrialvanguard.com/news/pds-biotechnology-halts-pds0101-trial-pivots-to-pds0301-in-colorectal-cancer/) (mCRC). The therapy combines Versamune® MUC1, a novel MUC1-targeted immunotherapy candidate, with PDS01ADC, an [IL-12](https://www.clinicaltrialvanguard.com/news/son-1010-shows-strong-safety-in-ovarian-cancer-treatment/) fused antibody drug conjugate. This Phase 1/2 trial will be conducted in collaboration with the National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA).
This IND clearance is a significant step forward in addressing the unmet need for effective treatments for mCRC, particularly Proficient Mismatch Repair/Microsatellite Stable mCRC, which represents 95% of mCRC cases and exhibits resistance to current immunotherapies. The combination of Versamune® MUC1 and PDS01ADC holds promise in stimulating robust and sustained anti-tumor T-cell responses within the tumor microenvironment, potentially offering a new therapeutic avenue for patients who have exhausted existing treatment options. The collaboration with the NCI further validates the potential of this approach.
The trial will focus on patients with unresectable, metastatic colorectal cancer who have failed prior treatments. This IND clearance represents an expansion of the Versamune® platform, which has primarily been investigated in HPV-related cancers. Additionally, a recently issued U.S. Patent (#12,201,685) covers the methods of using the combination of Versamune® and cytokines to enhance anti-tumor immune responses.
This IND clearance sets the stage for clinical investigation of a potentially groundbreaking combination therapy in a challenging cancer type. Positive trial results could lead to a new treatment paradigm for mCRC and further solidify the versatility and potential of the Versamune® platform in targeting various solid tumors. This advancement may also spur further research and development in the field of targeted immunotherapies for hard-to-treat cancers.
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**Categories:** News
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### [Promising FAP Treatment: Phase 2 Trial of TPST-1495 Approved by FDA](https://www.clinicaltrialvanguard.com/news/promising-fap-treatment-phase-2-trial-of-tpst-1495-approved-by-fda/)
**Published:** March 14, 2025
**Author:** Jon Napitupulu
**Content:**
Tempest Therapeutics has received FDA clearance for a Phase 2 clinical trial of [TPST-1495](https://www.clinicaltrialvanguard.com/news/fda-grants-orphan-drug-designation-to-tpst-1495-for-fap-treatment/), a dual prostaglandin E2 (PGE2) receptor inhibitor, for Familial Adenomatous Polyposis (FAP). The trial, funded by the National Cancer Institute and conducted by the Cancer Prevention Clinical Trials Network, will begin in 2025 with data expected in 2026. This marks Tempest’s second clinical program to enter Phase 2.
This development is particularly important for FAP patients who have undergone colectomy, as they face a high risk of developing duodenal polyps and subsequent cancers. Current treatment options are limited, highlighting the need for novel preventative therapies. The initiation of this Phase 2 trial addresses this unmet need by exploring the potential of TPST-1495 to reduce duodenal polyp burden and improve patient outcomes. This collaboration between Tempest, the NCI, and the Cancer Prevention Clinical Trials Network underscores the importance of public-private partnerships in accelerating the development of cancer prevention strategies.
The Phase 2 trial will evaluate TPST-1495’s efficacy and safety in FAP patients post-colectomy. The primary endpoint is the reduction in duodenal polyp burden after six months of treatment. Secondary endpoints include assessing polyp reduction in the retained rectum or ileal pouch-anal anastomosis (IPAA) and the drug’s overall safety profile.
A successful Phase 2 trial could significantly advance TPST-1495 towards potential FDA approval, offering a much-needed preventative treatment option for FAP patients. Positive results would validate Tempest’s approach to targeting PGE2 signaling in cancer and potentially open avenues for its application in other cancer types. This advancement strengthens Tempest’s pipeline and positions them as a key player in developing innovative cancer therapies.
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**Categories:** News
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### [Groundbreaking mRNA Biomarkers for Pancreatic Cancer Detection](https://www.clinicaltrialvanguard.com/news/groundbreaking-mrna-biomarkers-for-pancreatic-cancer-detection/)
**Published:** March 14, 2025
**Author:** Jon Napitupulu
**Content:**
Mainz Biomed has secured an exclusive license agreement with Liquid Biosciences for a portfolio of mRNA biomarkers aimed at developing a blood test for [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/). The companies plan to refine the test and pursue FDA approval. This agreement centers around promising biomarkers that have demonstrated 95% sensitivity and 98% specificity in detecting pancreatic cancer in blood samples.
This development holds substantial potential for advancing pancreatic cancer diagnostics. Pancreatic cancer is notoriously difficult to detect early, often presenting with symptoms only after it has reached advanced stages. A highly sensitive and specific blood test could dramatically improve early detection rates, leading to earlier intervention and significantly improved patient outcomes. This could be a critical step towards transforming pancreatic cancer from a frequently fatal diagnosis to a more manageable disease.
The agreement grants Mainz Biomed exclusive rights to develop a test using Liquid Biosciences’ mRNA biomarkers, with the option to acquire full global rights. The biomarkers were identified using Liquid Biosciences’ EMERGE platform and validated across multiple independent study cohorts totaling 285 subjects, including 35 pancreatic cancer patients. The resulting algorithm achieved impressive sensitivity and specificity metrics. While these results require further validation through integration into a new product, the initial data suggest the potential for a market-leading pancreatic cancer screening test. The agreement involves a license fee and royalties on future revenues if Mainz Biomed exercises its option to acquire the biomarker rights.
This collaboration positions Mainz Biomed at the forefront of pancreatic cancer diagnostics. The development and potential FDA approval of this blood-based test could significantly alter the landscape of pancreatic cancer screening and offer a much-needed tool for early detection and improved patient care. The successful development and commercialization of this test would not only benefit patients but also establish Mainz Biomed as a key player in the early cancer detection market.
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**Categories:** News
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### [EnnoDC Presents Positive Phase I AMV Data at CROI 2025](https://www.clinicaltrialvanguard.com/news/ennodc-presents-positive-phase-i-amv-data-at-croi-2025/)
**Published:** March 14, 2025
**Author:** Jon Napitupulu
**Content:**
EnnoDC’s Phase I ANRS VRI06 trial demonstrated that its Antibody Mediated Vaccine (AMV) technology induces a safe, durable, and robust immune response against HIV in human volunteers. The trial’s positive data, presented at CROI 2025, showcases the platform’s potential in treating infectious diseases and, notably, its expanding application in oncology. The study’s findings confirm the long-term effectiveness of the AMV approach, showing sustained immune responses even 80 weeks after initial treatment, further strengthened by a single booster shot.
This successful clinical proof-of-concept for AMVs holds significant implications for the future of HIV treatment and prevention. The durability of the immune response achieved with a minimal booster regimen suggests the potential for a simplified, and therefore more accessible, approach to HIV management. More broadly, the trial’s success validates the AMV platform, accelerating its application in other areas like oncology, offering a promising new avenue for developing effective cancer therapies.
Technically, the trial highlighted the AMV’s ability to engage, prime, and activate dendritic cells in vivo, resulting in a controlled and sustained immune response. This targeted approach minimizes off-target effects and maximizes therapeutic impact. Strategically, the positive results bolster EnnoDC’s advancement of its oncology pipeline, including AMV.HPVE6E7 for HPV-positive oropharyngeal cancer and AMV.PCA for [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/), both leveraging the now clinically validated AMV platform.
The positive CROI 2025 data marks a pivotal moment for EnnoDC. It not only provides crucial validation for the AMV platform’s potential in infectious diseases but also reinforces the company’s strategic focus on developing innovative cancer immunotherapies. The demonstrated long-lasting immunity, combined with a favorable safety profile, positions EnnoDC to potentially revolutionize treatment approaches for both infectious diseases and cancer, paving the way for a new era of targeted immunotherapies.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Dyne-251 Shows Unprecedented Functional Improvement in DMD Trial](https://www.clinicaltrialvanguard.com/news/dyne-251-shows-unprecedented-functional-improvement-in-dmd-trial/)
**Published:** March 17, 2025
**Author:** Jon Napitupulu
**Content:**
[Dyne Therapeutics](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/) announced positive long-term clinical data from its Phase 1/2 [DELIVER](https://www.clinicaltrialvanguard.com/news/new-positive-results-for-z-rostudirsen-in-dmd-deliver-trial/) trial of DYNE-251 for Duchenne muscular dystrophy (DMD). The data shows sustained functional improvement in patients amenable to exon 51 skipping at the 20 mg/kg dose. The company plans to submit a [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) for accelerated approval in the U.S. in early 2026.
This data is potentially game-changing for DMD patients as current therapies offer limited benefits. The sustained functional improvements observed with DYNE-251, particularly exceeding the minimal clinically important difference in Stride Velocity 95th Centile, suggest a potential for a significant impact on patients’ quality of life. The possibility of an accelerated approval pathway further underscores the urgent need and potential for this therapy to quickly reach those who could benefit.
The DELIVER trial data revealed sustained improvements in multiple functional endpoints, including Stride Velocity 95th Centile, North Star Ambulatory Assessment, 10-Meter Walk/Run Time, and Time to Rise from Floor, at both 10 mg/kg and 20 mg/kg doses. Unprecedented near-full length dystrophin expression was observed. The safety profile of DYNE-251 remains favorable after 970 administered doses and over 77 patient-years of follow-up. The registrational expansion cohort of the DELIVER trial is fully enrolled, with data expected in late 2025.
The positive data and planned BLA submission for DYNE-251 positions Dyne Therapeutics at the forefront of DMD treatment. The potential for accelerated approval could lead to rapid adoption of the therapy, offering new hope to DMD patients and potentially reshaping the treatment landscape for this debilitating disease.
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**Categories:** News
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### [FDA Approves Expanded Label for Iluvien®](https://www.clinicaltrialvanguard.com/news/fda-approves-expanded-label-for-iluvien/)
**Published:** March 17, 2025
**Author:** Jon Napitupulu
**Content:**
ANI Pharmaceuticals received FDA approval for an expanded label for [ILUVIEN](https://www.clinicaltrialvanguard.com/news/iluvien-meets-primary-endpoints-in-phase-4-uveitis-trial-at-six-months/), adding chronic non-infectious uveitis affecting the posterior segment of the eye (NIU-PS) to its existing indication for diabetic macular edema (DME). The company plans to launch ILUVIEN in the U.S. with the combined label later this year. This approval also incorporates updates to the product’s Warnings and Precautions.
This expanded indication represents a significant advancement in treatment options for patients suffering from NIU-PS, a debilitating condition that can lead to vision loss. The availability of ILUVIEN for NIU-PS provides physicians with a much-needed sustained-release corticosteroid option, potentially reducing the frequency of injections and improving patient compliance. This also strengthens ANI’s position in the ophthalmic market, offering a treatment for two distinct conditions with a single product. Furthermore, the recently extended and expanded manufacturing agreement with Siegfried through 2029 should ensure a reliable supply of ILUVIEN to meet the anticipated increase in demand.
ILUVIEN is an intravitreal implant delivering fluocinolone acetonide, a corticosteroid. Key safety information includes contraindications for patients with active or suspected ocular infections, [glaucoma](https://www.clinicaltrialvanguard.com/news/nurexone-shows-vision-recovery-in-preclinical-glaucoma-model/) with high cup-to-disc ratios, and hypersensitivity to the product’s components. Warnings and precautions include potential for increased intraocular pressure, cataract development, delayed corneal wound healing, and secondary ocular infections. Common adverse reactions for DME patients include cataracts, myodesopsia, and eye pain. For NIU-PS patients, common adverse reactions include cataracts, reduced visual acuity, macular edema, and uveitis.
This label expansion positions ILUVIEN for growth within the ophthalmic pharmaceuticals market, providing a valuable new therapeutic option for NIU-PS patients while solidifying ANI’s position as a provider of treatments for serious eye conditions. The company’s enhanced manufacturing capabilities should support increasing demand and contribute to long-term market success.
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**Categories:** News
**Tags:** eClinical Tech News
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### [Gain Therapeutics Doses First Patient in Phase 1b Parkinson's Trial](https://www.clinicaltrialvanguard.com/news/gain-therapeutics-doses-first-patient-in-phase-1b-parkinsons-trial/)
**Published:** March 17, 2025
**Author:** Jon Napitupulu
**Content:**
Gain Therapeutics has dosed the first [Parkinson’s](https://www.clinicaltrialvanguard.com/article/intel-brief/gcp-breakdown-at-kings-college-hospital-just-clouded-the-most-promising-parkinsons-trial-in-years/) disease patient in a Phase 1b clinical trial of its lead candidate, [GT-02287](https://www.clinicaltrialvanguard.com/news/new-parkinsons-drug-gt-02287-shows-promising-phase-1b-data/). The trial will assess the drug’s safety and tolerability in patients with Parkinson’s disease, both with and without a GBA1 mutation. Interim analysis is expected by the end of Q2 2025.
This trial initiation is a critical step for Gain Therapeutics and potentially for Parkinson’s disease treatment. A successful outcome could validate GT-02287’s mechanism of action, specifically its ability to restore glucocerebrosidase (GCase) function. This is particularly relevant because GBA1 mutations, which impair GCase, are the most common genetic risk factor for Parkinson’s disease. A therapy addressing this underlying mechanism holds the promise of disease modification, a significant advancement over current symptomatic treatments.
The Phase 1b trial is an open-label, multi-center study involving up to 20 participants who will receive daily doses of GT-02287 for three months. Secondary endpoints include pharmacokinetics, GCase modulation, substrate levels, and other biomarkers in plasma and cerebrospinal fluid. This follows a successful Phase 1 study in healthy volunteers, where GT-02287 demonstrated a favorable safety profile, target engagement, and significant increases in GCase activity. Preclinical studies have shown the drug’s potential to reduce alpha-synuclein aggregation, neuroinflammation, and neuronal death, along with improvements in motor and cognitive function. Additionally, GT-02287 reduced plasma neurofilament light chain levels, a promising biomarker for neurodegeneration.
Positive interim results from the Phase 1b trial could significantly boost Gain Therapeutics’ position in the Parkinson’s disease therapeutic landscape. It would pave the way for larger, later-stage trials, potentially establishing GT-02287 as a much-needed disease-modifying therapy for a large patient population. This success could also spur further research into GCase modulation as a therapeutic strategy for Parkinson’s disease, opening new avenues for drug development.
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---
### [KRRO-110 FDA Orphan Drug Designation for A1AD Deficiency](https://www.clinicaltrialvanguard.com/news/krro-110-fda-orphan-drug-designation-for-a1ad-deficiency/)
**Published:** March 17, 2025
**Author:** Jon Napitupulu
**Content:**
Korro Bio announced that its investigational medicine, KRRO-110, received orphan drug designation from the FDA for treating Alpha-1 Antitrypsin Deficiency (AATD). KRRO-110 is the first RNA editing candidate from Korro’s [OPERA](https://www.clinicaltrialvanguard.com/news/olema-presents-palazestrant-opera-02-trial-at-sabcs-2025/)™ platform and is currently in a Phase 1/2a clinical trial called REWRITE. This designation recognizes the unmet need for AATD treatments and provides Korro with development incentives like tax credits and potential market exclusivity.
This FDA decision is notable because it validates the potential of RNA editing as a therapeutic approach for AATD, a rare genetic disorder affecting fewer than 200,000 people in the U.S. Current treatment options for AATD are limited, and KRRO-110 offers a novel mechanism of action by repairing the faulty gene responsible for the disease at the RNA level. This could lead to a more effective treatment targeting both liver and lung manifestations of AATD. The orphan drug designation also strengthens Korro’s competitive position in the rare disease space.
The Phase 1/2a REWRITE trial is a dose-escalation study evaluating the safety and tolerability of KRRO-110 in healthy adults and AATD patients. Interim data from the single ascending dose cohorts in healthy volunteers is expected in the second half of 2025, with study completion anticipated in 2026. The trial will also assess pharmacokinetic and pharmacodynamic parameters to inform dose selection for future studies. The orphan drug designation provides Korro with financial benefits that could support the continued development of KRRO-110.
This designation represents a significant step forward for Korro Bio and the development of KRRO-110. Positive clinical data and subsequent FDA approval could establish KRRO-110 as a leading therapy for AATD, potentially transforming the treatment landscape for this rare disease. This achievement also reinforces the potential of Korro’s RNA editing platform, OPERA™, to generate further innovative therapies for other genetic diseases.
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### [Gradalis Unveils Clonal Tumor Mutation Burden Pipeline](https://www.clinicaltrialvanguard.com/news/gradalis-unveils-clonal-tumor-mutation-burden-pipeline/)
**Published:** March 17, 2025
**Author:** Jon Napitupulu
**Content:**
Gradalis, a clinical-stage biotech company, published a peer-reviewed article in Scientific Reports detailing their novel exome sequencing procedure and bioinformatics pipeline for identifying clonal cancer signals. This process focuses on clonal Tumor Mutation Burden (cTMB), clonal Neoantigens (cNEO), and Intratumoral Heterogeneity (ITH) to identify optimal targets for immunotherapy and predict patient response. The study validated this approach and demonstrated that the clonal signal is preserved in Gradalis’ investigational immunotherapy, Vigil.
This research is important because it offers a potentially more precise and effective way to target cancer with immunotherapy. By focusing on clonal signals present in all cancer cells, as opposed to subclonal signals present only in subsets, the immune system can mount a more comprehensive attack. This targeted approach could lead to more consistent and durable clinical responses across various cancer types, addressing a significant unmet need in oncology. Moreover, the ability to pre-select patients likely to benefit from immunotherapy could optimize clinical trial design and improve patient outcomes.
The exome sequencing process utilizes paired tumor and normal samples to generate high-coverage sequence data. This data is processed through a bioinformatics pipeline that identifies single [nucleotide](https://www.clinicaltrialvanguard.com/news/ateas-new-hcv-combo-aims-for-best-in-class-treatment/) variants, insertions, deletions, and MHC-1 alleles, leading to the prediction of neoantigen peptides and clonality analysis. The final report quantifies cTMB, cNEO, and ITH levels, providing crucial information for treatment selection. Gradalis partnered with Frontage Laboratories to develop and validate this pipeline, ensuring its clinical applicability. The study also confirmed the stability of the clonal signal within their Vigil therapy, a personalized immunotherapy derived from the patient’s own tumor. Vigil is designed to enhance the immune response to a patient’s unique tumor signature.
This research represents a significant step forward in the development of personalized cancer immunotherapy. The identification and validation of clonal signals as robust therapeutic targets, combined with a reliable method for their detection, could significantly improve treatment strategies. This discovery could lead to the development of more effective immunotherapies and improved patient selection for existing treatments, potentially ushering in an era of more personalized and effective cancer care.
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---
### [Bitterroot Bio Presents Phase 1 BRB-002 Results at ACC.25](https://www.clinicaltrialvanguard.com/news/bitterroot-bio-presents-phase-1-brb-002-results-at-acc-25/)
**Published:** March 18, 2025
**Author:** Jon Napitupulu
**Content:**
Bitterroot Bio will present Phase 1 study results for [BRB-002](https://www.clinicaltrialvanguard.com/news/bitterroot-bio-announces-positive-phase-1-data-for-brb-002/), a novel anti-CD47 therapy for atherosclerotic cardiovascular disease (ASCVD), at the American College of Cardiology’s 2025 Scientific Session. The study in healthy volunteers met its primary safety objective, showing no serious adverse events and demonstrating dose-dependent target engagement with up to 100% CD47 receptor occupancy at the highest doses. The presentation will be delivered by Dr. Alexander Yi on March 29, 2025.
These findings are potentially crucial for advancing the treatment of ASCVD. Current treatments often focus on managing symptoms and risk factors, but BRB-002’s mechanism, inhibiting the “don’t eat me” signal of CD47, could offer a novel way to address the underlying inflammation driving atherosclerosis and plaque buildup. This potential to modify the disease course rather than just manage symptoms makes BRB-002 a promising candidate in a field with a high unmet need for more effective therapies. Positive Phase 1 results could attract further investment and accelerate the development of this much-needed treatment.
The Phase 1 study demonstrated both safety and target engagement, which are critical milestones for any new drug candidate. Achieving high CD47 receptor occupancy suggests the drug is effectively binding to its target, offering a strong rationale for progressing to further clinical trials. This successful early trial lays the groundwork for evaluating the drug’s efficacy in patients with ASCVD.
The upcoming presentation at ACC represents a key inflection point for Bitterroot Bio. Positive reception of these early findings could significantly raise the company’s profile within the cardio-immunology field and generate excitement about the potential of BRB-002. This could pave the way for future collaborations, partnerships, and further funding to advance the clinical development of this promising therapy and potentially reshape the ASCVD treatment landscape.
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### [Radiopharm Theranostics: Rad202 Shows Positive Tumor Uptake & Biodistribution](https://www.clinicaltrialvanguard.com/news/radiopharm-theranostics-rad202-shows-positive-tumor-uptake-biodistribution/)
**Published:** March 18, 2025
**Author:** Jon Napitupulu
**Content:**
Radiopharm Theranostics presented data at EMIM 2025 demonstrating the effectiveness of [RAD202](https://www.clinicaltrialvanguard.com/news/radiopharm-advances-to-cohort-3-in-phase-1-clinical-trial/), a HER2-targeting single domain antibody, for both imaging and therapy. Preclinical studies showed 68Ga-RAD202 effectively imaged HER2-positive tumors and 177Lu-RAD202, in both single and fractionated doses, reduced tumor volume and improved survival in mouse models. This research builds on previous studies that established the safety and biodistribution of a related compound, 99mTc-RAD202, in humans.
This development is critical because it validates the continued clinical development of 177Lu-RAD202 as a potential treatment option for HER2-positive cancers. It offers the possibility of a more effective and potentially less toxic treatment for patients who have progressed on or cannot tolerate existing therapies. The improved tumor targeting and efficacy of the modified RAD202 without the His-tag, as highlighted in the imaging data, is particularly promising for optimizing patient outcomes. This refined approach could lead to more precise and effective treatment delivery.
The data presented at EMIM 2025 focused on improved tumor targeting with a modified RAD202, specifically showing a high tumor-to-background ratio in imaging studies. Fractionated dosing of 177Lu-RAD202 showed greater efficacy compared to a single dose in preclinical models. This research is being translated into a Phase 1 clinical trial in Australia, which is currently recruiting patients with advanced HER2-positive solid tumors.
The positive preclinical data and the ongoing Phase 1 trial suggest a promising future for RAD202 as a potential therapeutic and diagnostic agent. The results of the clinical trial will be crucial for determining the next steps in the development of this radiopharmaceutical and its potential to improve treatment options for patients with HER2-positive cancers.
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**Tags:** eClinical Tech News
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### [PEP-010 Awarded FDA Orphan Drug Status for Pancreatic Cancer](https://www.clinicaltrialvanguard.com/news/pep-010-awarded-fda-orphan-drug-status-for-pancreatic-cancer/)
**Published:** March 18, 2025
**Author:** Jon Napitupulu
**Content:**
PEP-Therapy’s lead product, PEP-010, a first-in-class bifunctional therapeutic peptide, received Orphan Drug Designation from the FDA for the treatment of [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/). The peptide is currently in a Phase Ib clinical trial assessing its safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity in combination with chemotherapy for pancreatic cancer patients. This designation is based on the significant unmet medical need in pancreatic cancer, a disease with limited treatment options and increasing incidence and mortality.
This FDA decision is crucial for advancing pancreatic cancer treatment. Pancreatic cancer is the fourth leading cause of cancer-related deaths in the U.S., with a dismal survival rate for advanced or metastatic cases. Current systemic treatments have seen limited progress, leaving a critical need for innovative therapies. PEP-010 offers a potential new approach by restoring normal apoptosis in cancer cells through a novel mechanism of action involving caspase-9 and PP2A proteins. The Orphan Drug Designation validates the drug’s potential to address this unmet need and encourages further research and development of this promising therapy.
The Orphan Drug Designation provides PEP-Therapy with significant benefits, including tax credits for clinical trials, exemptions from regulatory fees, and potential market exclusivity for seven years post-approval. This status will expedite PEP-010’s development and potentially accelerate its availability to patients. The ongoing Phase Ib trial is currently evaluating PEP-010 in combination with standard chemotherapy regimens, investigating its potential to improve outcomes for patients with this aggressive cancer.
This designation signifies a vital step toward providing a much-needed new treatment option for pancreatic cancer. It allows PEP-Therapy to further investigate PEP-010’s potential to improve the survival and quality of life for patients facing this challenging disease. The positive implications of this designation will likely encourage further investment and collaboration in the development of PEP-010, potentially leading to a significant advancement in the field of pancreatic cancer treatment.
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### [Cellphire Therapeutics gets FDA Fast Track for CLPH-511](https://www.clinicaltrialvanguard.com/news/cellphire-therapeutics-gets-fda-fast-track-for-clph-511/)
**Published:** March 18, 2025
**Author:** Jon Napitupulu
**Content:**
Cellphire Therapeutics has received FDA Fast Track Designation for CLPH-511, a frozen activated platelet injectable suspension designed to treat acute hemorrhage when conventional platelets are unavailable. This designation expedites the development and review process for CLPH-511, potentially leading to faster approval and addressing a critical unmet medical need. The FDA granted this designation because of the significant potential CLPH-511 has to address a serious medical condition, acute hemorrhage, where current solutions face logistical limitations.
This Fast Track Designation is vital due to the persistent challenge of managing acute hemorrhage, a leading cause of death and complications in trauma, surgery, and critical care. Current platelet transfusion methods are hampered by short shelf life and limited availability. CLPH-511’s frozen formulation offers a significant advantage by extending shelf life and enabling broader accessibility, potentially revolutionizing acute hemorrhage management in various settings, including military and civilian pre-hospital environments.
CLPH-511 is currently being evaluated in a Phase 2/3 adaptive design study (CRYPTICS) comparing it to liquid-stored platelets in patients undergoing cardiopulmonary bypass surgery. The Fast Track Designation allows for more frequent interaction with the FDA, including the potential for rolling review of the [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) and eligibility for Priority Review if clinical data demonstrates substantial improvements in safety or efficacy.
This expedited pathway could lead to the faster availability of CLPH-511 for patients facing life-threatening bleeding. Positive results from the ongoing clinical trial, combined with the benefits of the Fast Track Designation, could position CLPH-511 as a transformative solution in acute hemorrhage management, improving patient outcomes and addressing a long-standing medical challenge.
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### [Deramiocel Shows Long-Term Efficacy in Duchenne Muscular Dystrophy Treatment](https://www.clinicaltrialvanguard.com/news/deramiocel-shows-long-term-efficacy-in-duchenne-muscular-dystrophy-treatment/)
**Published:** March 18, 2025
**Author:** Jon Napitupulu
**Content:**
Capricor Therapeutics announced positive three-year data from its open-label extension of the HOPE-2 clinical trial for deramiocel, a cell-based therapy for [Duchenne muscular dystrophy](https://www.clinicaltrialvanguard.com/news/dyne-therapeutics-z-rostudirsen-wins-priority-review-for-duchenne-muscular-dystrophy/)[Duchenne](https://www.clinicaltrialvanguard.com/clinops-watchdog/capricors-duchenne-adcom-didnt-fail-on-science-it-failed-on-statistics/) muscular dystrophy (DMD). The trial demonstrated a 52% slower decline in upper limb function for patients treated with deramiocel compared to an external comparator group. This positive data was presented at the 2025 Muscular Dystrophy Association Conference.
This news holds significant promise for the DMD community. Current treatment options for DMD are limited, and the disease inevitably leads to progressive muscle degeneration and premature death. Deramiocel’s demonstrated ability to not only slow but potentially modify the disease course offers a new level of hope for improving patients’ quality of life and extending lifespan. The increasing treatment effect observed year over year, coupled with a favorable safety profile, further underscores deramiocel’s therapeutic potential.
The Performance of the Upper Limb (PUL 2.0) score, a key measure of disease progression in DMD, declined by an average of 3.46 points over three years in deramiocel-treated patients, compared to a 7.19-point decline in the comparator group. Notably, even during a one-year treatment gap, patients previously treated with deramiocel maintained a slower rate of decline than untreated patients, suggesting a potential disease-modifying effect. Furthermore, the annual decline in PUL 2.0 scores lessened each year for deramiocel patients (1.8 points in Year 1, 1.2 points in Year 2, and 1.1 points in Year 3), indicating accumulating benefits over time.
This long-term data reinforces the potential of deramiocel to become a transformative therapy for DMD. With the FDA having accepted Capricor’s [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) for deramiocel for DMD-associated cardiomyopathy, and a PDUFA date set for August 2025, the future of DMD treatment appears promising. Positive results from the BLA review could lead to a significant advancement in DMD care, offering patients a new therapeutic option that may fundamentally alter the disease’s progression. Further research and development of deramiocel, including exploring its application to other aspects of DMD, will be crucial for realizing its full therapeutic potential.
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**Tags:** eClinical Tech News
---
### [2025 SCOPE Summit: Advancing Patient-Centric Clinical Trials](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/)
**Published:** March 18, 2025
**Author:** Moe Alsumidaie
**Content:**
The 2025 [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/merck-on-investigator-initiated-studies-and-budgeting-practices-at-2025-scope-summit/) Summit brought together industry leaders to explore patient-centric approaches in clinical trials, addressing the critical issue of low enrollment rates. With 80% of trials failing to enroll on time, the summit focused on strategies to engage healthcare providers and patients effectively. Key discussions revolved around modernizing protocols, leveraging data, and fostering trust with diverse communities to enhance trial accessibility and success. The event underscored the industry’s commitment to evolving clinical research practices to serve patients better and improve trial outcomes.
#### [](#enhancing-clinical-trial-experiences)Enhancing Clinical Trial Experiences
At the summit, panelists discussed strategies to improve clinical trial experiences for both sites and participants. Garo Kiledjian from SGM Alliance emphasized the need to update protocol templates to foster inclusivity, suggesting changes in language to accommodate diverse participants. This modernization aims to create more welcoming entry points for individuals, ensuring that clinical trials are accessible to all, regardless of gender identity. Such efforts are crucial in making trials more inclusive and representative of the population.
Kristen Andrews from LabCorp highlighted the company’s strategic investments to enhance patient trial access. By investing in companies like Circuit Clinical and Hawthorne Health, LabCorp aims to bridge the gap between patients and healthcare providers, making trials more accessible and convenient. These investments leverage LabCorp’s extensive network of healthcare providers, allowing patients to engage in trials where they are most comfortable. This approach increases trial participation and ensures that trials are conducted in a patient-friendly manner.
#### [](#leveraging-data-for-human-centered-trials)Leveraging Data for Human-Centered Trials
The panelists underscored the importance of data in shaping study design and implementation. Garo Kiledjian pointed out the industry’s oversight in collecting data on sexual orientation and gender identity, which limits the ability to create inclusive protocols. He advocated for systematic data collection to better study designs catering to diverse populations. This data-driven approach is essential for designing protocols that are truly inclusive and representative.
Kristen Andrews shared LabCorp’s innovative use of patient service centers (PSCs) as trial sites. With over 2,000 locations across the U.S., these centers provide a convenient and trusted environment for trial participation. LabCorp is piloting initiatives that allow companies to lease space within these centers, enhancing accessibility. By enabling patients to participate in trials at familiar locations, LabCorp aims to reduce barriers and improve patient engagement, making trials more accessible to a broader population.
#### [](#future-directions-and-advice-for-clinical-trials)Future Directions and Advice for Clinical Trials
Panelists offered advice for future clinical trials, emphasizing the need to engage patients and HCPs in protocol design. Garo Kiledjian highlighted the importance of focusing on hidden populations, such as those affected by HIV, which have been underrepresented in research. The industry can capture a more diverse participant pool and improve trial outcomes by tapping into these communities. This approach ensures that trials are inclusive and representative of all communities.
Kristen Andrews advocated for innovative trial models, such as virtual trials, to reach patients in remote areas. By offering options for participation, whether through virtual means or traditional sites, the industry can better accommodate diverse needs and preferences. This flexibility is crucial for ensuring that all patients can participate in clinical trials, regardless of location or circumstances.
#### [](#motivating-participation-and-building-trust)Motivating Participation and Building Trust
In response to audience questions, panelists explored motivators for patient participation in clinical trials. Margaret Ikpoh noted that the [COVID-19](https://www.clinicaltrialvanguard.com/news/eu-authorizes-pfizer-biontech-xfg-covid-19-vaccine-for-2026-2027/) pandemic was a powerful motivator, driving patients to seek empowerment through knowledge and participation in research. This highlights the importance of addressing patient concerns and providing clear, accessible information about clinical trials.
Garo Kiledjian emphasized the need for safe spaces for marginalized communities, who may fear engaging with healthcare systems due to historical mistrust. He highlighted the importance of creating pathways that allow these communities to participate in trials without fear of discrimination. By fostering trust and building relationships, the industry can overcome barriers to participation and ensure that trials are genuinely inclusive.
#### [](#summary)**Summary**
The SCOPE Summit 2025 highlighted the industry’s commitment to patient-centric clinical trials, emphasizing the need for inclusivity, data-driven design, and trust-building with diverse communities. By addressing barriers to participation and leveraging innovative models, the industry can improve trial outcomes and ensure that new treatments reach those who need them most. The panel discussion was a call to action for continued innovation and collaboration in pursuing more inclusive and practical clinical research.
**Categories:** Article: Conference Coverage
---
### [Bio-Path Holdings Announces Promising Pre-clinical Results for BP1001-A in Obesity Treatment for Type 2 Diabetes Patients](https://www.clinicaltrialvanguard.com/news/bio-path-holdings-announces-promising-pre-clinical-results-for-bp1001-a-in-obesity-treatment-for-type-2-diabetes-patients/)
**Published:** March 19, 2025
**Author:** Jon Napitupulu
**Content:**
[Bio-Path Holdings](https://www.clinicaltrialvanguard.com/news/bio-path-holdings-key-clinical-breakthroughs/) announced positive pre-clinical results for BP1001-A, a potential [obesity](https://www.clinicaltrialvanguard.com/news/skye-bioscience-acquires-redx-pharma-exits-obesity-for-fibrosis-focus/) treatment. The drug candidate successfully reversed fatty acid-induced insulin resistance and improved insulin sensitivity in muscle cell models. These findings suggest BP1001-A could be effective in treating obesity and related metabolic issues, particularly in patients with [Type 2 diabetes](https://www.clinicaltrialvanguard.com/news/bp1001-a-shows-promise-as-obesity-treatment-for-type-2-diabetes/).
Current obesity treatments often fall short in achieving substantial weight loss for patients with Type 2 diabetes, creating a significant unmet medical need. BP1001-A’s positive impact on insulin sensitivity at the cellular level offers a potentially new approach to managing blood glucose levels and addressing this challenge. This mechanism, focused on improving insulin response, could offer advantages over existing treatments primarily aimed at weight reduction.
Pre-clinical studies demonstrated that BP1001-A attenuated the negative effects of palmitic acid, a common saturated fatty acid linked to insulin resistance. The drug’s success in both muscle progenitor and mature muscle fiber cells further strengthens its potential efficacy. The company will now proceed with final pre-clinical animal testing to evaluate the drug’s effects on weight, insulin sensitivity, and glucose tolerance. A successful outcome will pave the way for an Investigational New Drug (IND) application in 2025, aiming to initiate human clinical trials.
These pre-clinical findings represent a significant step forward for BP1001-A’s development. Positive results from the upcoming animal studies could solidify its potential as a novel therapeutic option for obesity and Type 2 diabetes, potentially addressing the shortcomings of current treatments and offering new hope for patients. The anticipated IND application later this year represents a critical milestone towards realizing this potential.
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**Tags:** eClinical Tech News
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### [Clearmind Medicine Initiates First Human Trial for Alcohol Use Disorder](https://www.clinicaltrialvanguard.com/news/clearmind-medicine-initiates-first-human-trial-for-alcohol-use-disorder/)
**Published:** March 19, 2025
**Author:** Jon Napitupulu
**Content:**
Clearmind Medicine Inc. has initiated a Phase I/IIa clinical trial for [CMND-100](https://www.clinicaltrialvanguard.com/news/clearmind-medicine-trial-soars-after-safety-board-approval/), a novel psychedelic-derived treatment for Alcohol Use Disorder (AUD). This trial, taking place at IMCA in Israel, Yale School of Medicine, and Johns Hopkins University School of Medicine, will evaluate the drug’s safety, tolerability, pharmacokinetics, and preliminary efficacy in reducing alcohol cravings and consumption. This marks the first clinical application of Clearmind’s proprietary CMND-100 platform.
This clinical trial initiation is a crucial step for Clearmind and potentially for the AUD treatment landscape. Positive results could validate the company’s preclinical findings, demonstrating the potential of CMND-100’s unique mechanism of action, which modulates reward mechanisms associated with addictive behavior. This is particularly important given the limited effectiveness of current AUD treatments and the significant global health burden of this disorder. Successful clinical development could position CMND-100 as a much-needed new therapeutic option.
The trial will encompass safety, tolerability, and pharmacokinetic assessments of CMND-100 in individuals with AUD. It will also include preliminary investigations into the drug’s ability to reduce both cravings for and consumption of alcohol. The inclusion of prestigious U.S. research institutions alongside the Israeli site adds further credibility to the study.
Moving forward, the results of this Phase I/IIa trial will be critical. Positive data could pave the way for larger-scale clinical trials, potentially leading to eventual regulatory approval and commercialization of CMND-100. This could not only transform Clearmind’s position within the pharmaceutical industry but also offer renewed hope for millions struggling with AUD worldwide. The innovative approach of targeting reward mechanisms could represent a significant advancement in addiction treatment.
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### [Ocu200 DME Trial: Cohort 2 Dosing Approved After Safety Review](https://www.clinicaltrialvanguard.com/news/ocu200-dme-trial-cohort-2-dosing-approved-after-safety-review/)
**Published:** March 19, 2025
**Author:** Jon Napitupulu
**Content:**
Ocugen, a biotechnology company, announced positive safety data from the first cohort of its Phase 1 clinical trial for OCU200, a novel fusion protein designed to treat diabetic macular edema (DME). The Data and Safety Monitoring Board approved the trial’s progression to the second cohort after finding no serious adverse events related to OCU200. The trial is evaluating the safety and tolerability of three different doses of OCU200 administered via intravitreal injection.
This development is a crucial step for Ocugen and potentially for DME patients. A significant portion of DME patients don’t respond to current anti-VEGF treatments, creating a substantial need for alternative therapies. OCU200’s unique mechanism of action, targeting integrin receptors on active endothelial cells, offers a different approach and could expand treatment options for these individuals. The positive safety profile from the first cohort is encouraging, suggesting the treatment is well-tolerated and paving the way for further investigation of its efficacy.
The Phase 1 trial is a multicenter, open-label, dose-escalation study involving three cohorts receiving low (0.025 mg), medium (0.05 mg), and high (0.1 mg) doses of OCU200. Each participant receives two doses six weeks apart and is monitored for up to six months. OCU200 combines tumstatin, with anti-inflammatory and anti-VEGF properties, and transferrin, which targets the drug to the choroid and retina. This targeted approach could allow for effective treatment at lower doses compared to existing therapies.
The positive safety data from the first cohort strengthens the potential of OCU200 as a viable treatment option for DME, diabetic retinopathy, and wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/). Continued positive results could significantly impact the treatment landscape for these conditions, offering hope for patients who haven’t responded to current therapies. Ocugen anticipates completing the Phase 1 trial in the latter half of 2025 and will provide updates on safety and efficacy throughout the year. These forthcoming updates will be critical in determining the next steps for OCU200’s development and its potential to address the unmet needs of millions affected by these debilitating eye diseases.
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**Tags:** eClinical Tech News
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### [Fruquintinib & Sintilimab Trial Meets Primary Endpoint in Advanced RCC](https://www.clinicaltrialvanguard.com/news/fruquintinib-sintilimab-trial-meets-primary-endpoint-in-advanced-rcc/)
**Published:** March 19, 2025
**Author:** Jon Napitupulu
**Content:**
[HUTCHMED](https://www.clinicaltrialvanguard.com/news/hutchmed-highlights-new-lung-cancer-data-at-2025-conferences/) and Innovent Biologics announced positive top-line results from the [FRUSICA](https://www.clinicaltrialvanguard.com/news/hutchmed-to-present-frusica-2-trial-data-at-esmo-congress/)-2 Phase II/III trial. The trial investigated fruquintinib combined with sintilimab as a second-line treatment for [advanced renal cell carcinoma](https://www.clinicaltrialvanguard.com/news/allogenes-allo-316-achieves-31-response-rate-in-advanced-renal-cell-carcinoma/) (RCC) in China and successfully met its primary endpoint of progression-free survival. The combination also showed improvement in secondary endpoints, including objective response rate and duration of response.
This success is particularly important because it addresses the unmet need for effective second-line treatments for advanced RCC in China. While several first-line immune-oncology combination therapies are approved in the US, options are more limited in China, particularly for those who have progressed after first-line targeted therapy. This positive data positions the fruquintinib-sintilimab combination to potentially become a valuable new treatment option for these patients.
The FRUSICA-2 study was a randomized, open-label, active-controlled trial comparing the fruquintinib-sintilimab combination to axitinib or everolimus monotherapy. Previous Phase Ib/II data for this combination showed promising results, with a confirmed objective response rate of 60% and a disease control rate of 85% in previously treated patients. Full data from the FRUSICA-2 trial will be presented at an upcoming scientific conference.
This positive outcome paves the way for potential regulatory submissions in China for the fruquintinib-sintilimab combination in advanced RCC. This development could significantly alter the treatment landscape for this cancer in China, offering a new and potentially more effective second-line therapy for patients who have not responded adequately to prior treatments. This also strengthens the position of both HUTCHMED and Innovent in the oncology market.
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### [Arbor Biotechnologies Secures $73.9M Series C Funding for Gene Editing](https://www.clinicaltrialvanguard.com/news/arbor-biotechnologies-secures-73-9m-series-c-funding-for-gene-editing/)
**Published:** March 19, 2025
**Author:** Jon Napitupulu
**Content:**
Arbor Biotechnologies secured $73.9 million in Series C funding, led by ARCH Venture Partners and TCGX, to advance its gene editing therapies for liver and central nervous system (CNS) diseases. The funding will propel the clinical development of [ABO-101](https://www.clinicaltrialvanguard.com/news/arbor-biotechnologies-doses-first-patient-in-abo-101-gene-editing-study/) for primary hyperoxaluria type 1 (PH1) and progress other programs towards Investigational New Drug (IND) applications, including a novel reverse transcriptase (RT) editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis (ALS). This investment significantly extends Arbor’s operational runway into 2027.
This influx of capital is crucial for the advancement of gene editing therapies, particularly for challenging targets like the CNS. Arbor’s progress in developing a diverse pipeline, including a first-in-class RT editing program and a therapy for ALS, positions them at the forefront of gene editing innovation. Successfully translating these preclinical programs into clinical trials represents a significant step towards addressing unmet medical needs in these complex disease areas.
Arbor’s lead program, ABO-101, is currently in a Phase 1/2 clinical trial for PH1. The company is also developing a preclinical RT editing program for an undisclosed rare liver disease and an ALS program, both of which are approaching IND-enabling studies. The Series C funding provides the resources to advance these programs and further expand Arbor’s proprietary gene editing platform.
This substantial investment underscores growing confidence in Arbor’s platform and its potential to deliver transformative gene editing therapies. The extended cash runway allows Arbor to focus on executing its clinical development strategy and potentially bring groundbreaking treatments to patients suffering from debilitating genetic diseases. The advancement of programs targeting the CNS, in particular, holds immense promise for addressing previously untreatable neurological conditions.
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### [Acumen Announces Results for Sabirnetug Formulation Study](https://www.clinicaltrialvanguard.com/news/acumen-announces-results-for-sabirnetug-formulation-study/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
Acumen Pharmaceuticals announced positive topline results from a Phase 1 study of its [Alzheimer’s](https://www.clinicaltrialvanguard.com/news/fda-clears-precivityad2-blood-test-for-alzheimers-in-adults-as-young-as-40/) drug, sabirnetug. The study compared subcutaneous (SC) and intravenous (IV) delivery methods in healthy volunteers, finding weekly SC injections were well-tolerated and achieved systemic exposure sufficient for further development. This is particularly relevant as the company’s lead candidate is currently being studied through IV infusions.
This development is a crucial step for Acumen and potentially for Alzheimer’s patients. A shift to subcutaneous administration could significantly improve patient convenience by eliminating the need for regular IV infusions, thereby potentially increasing treatment adherence and accessibility, which is especially critical for managing chronic conditions like Alzheimer’s. This could differentiate sabirnetug in a competitive Alzheimer’s treatment landscape.
The Phase 1 study involved 12 subjects receiving single IV doses of 2,800 mg and 16 subjects receiving four weekly SC doses of 1,200 mg. The most frequent adverse events were mild injection site reactions that resolved without intervention. Critically, the SC delivery achieved systemic drug levels comparable to IV, paving the way for future studies using this more patient-friendly administration route. Acumen’s ongoing Phase 2 ALTITUDE-AD study is currently evaluating the efficacy and safety of IV sabirnetug in early Alzheimer’s patients. The subcutaneous formulation utilizes Halozyme’s ENHANZE technology, which facilitates larger volume injections and faster absorption.
The positive Phase 1 results suggest a subcutaneous formulation of sabirnetug could become a viable and preferred option for Alzheimer’s patients. This could represent a significant advancement in treatment accessibility and patient compliance, potentially strengthening Acumen’s market position and accelerating broader access to this promising therapeutic candidate. Further clinical trials will be essential to confirm the efficacy and long-term safety of the subcutaneous formulation.
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### [Protara Therapeutics: Choline Deficiency and Liver Injury in Parenteral Support Patients: Encore Presentation of THRIVE-1 Results](https://www.clinicaltrialvanguard.com/news/protara-therapeutics-choline-deficiency-and-liver-injury-in-parenteral-support-patients-encore-presentation-of-thrive-1-results/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
[Protara](https://www.clinicaltrialvanguard.com/news/protara-therapeutics-announces-positive-interim-results-from-phase-2-advanced-2-trial-of-tara-002-in-patients-with-nmibc/) Therapeutics announced results from [THRIVE](https://www.clinicaltrialvanguard.com/news/dbv-technologies-launches-thrive-trial-of-viaskin-peanut-patch-in-infants/)-1, a study on choline deficiency and liver injury in patients dependent on parenteral support (PS). The study found that 78% of PS patients were choline deficient and 63% of those deficient also experienced liver dysfunction. Protara plans to start the THRIVE-3 registrational trial for their intravenous (IV) Choline Chloride product in the first half of 2025.
These findings are crucial for patients reliant on PS who currently lack an effective way to receive choline, a vital nutrient. The high prevalence of choline deficiency and associated liver damage highlights a serious unmet medical need within this population. Developing an IV Choline Chloride formulation offers a potential solution to prevent and treat this deficiency, thereby improving liver health and overall well-being for these individuals.
THRIVE-1 was a prospective, observational study. The upcoming THRIVE-3 trial is a seamless Phase 2b/3 clinical trial designed with a dose confirmation phase (n=24) followed by a larger, double-blind, placebo-controlled phase (n=100) in adolescents and adults on long-term PS. The trial will assess both efficacy and safety of IV Choline Chloride. The product has already received Fast Track designation from the FDA, and both ASPEN and ESPEN recommend IV choline for PS patients.
The positive results from THRIVE-1 and the initiation of THRIVE-3 suggest a promising future for IV Choline Chloride. If successful, this therapy could become the first FDA-approved IV choline formulation, addressing a substantial clinical need and significantly improving the quality of life for patients dependent on parenteral support. This innovation holds potential to become a standard of care for this population, altering current clinical practice guidelines and creating new market opportunities.
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### [Conduit Pharmaceuticals Pipeline Update: AZD1656, AZD5658, & AZD5904](https://www.clinicaltrialvanguard.com/news/conduit-pharmaceuticals-pipeline-update-azd1656-azd5658-azd5904/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
Conduit Pharmaceuticals announced progress in its preclinical and clinical pipeline, including studies for AZD5658 in lupus, Phase IIa trial design for AZD1656, formulation optimization, and intellectual property expansion. The company is evaluating glucokinase inhibitors for autoimmune diseases, with a focus on lupus, anticipating results in Q2 2025. Conduit is also optimizing the Phase II trial design for AZD1656 in systemic lupus erythematosus with nephritis and ANCA-associated vasculitis.
This progress signals a potential shift in the treatment landscape for autoimmune diseases like lupus, SLE, and AAV. Positive preclinical data for AZD5658 could validate glucokinase inhibition as a viable therapeutic strategy for lupus, potentially leading to new treatment options for this complex disease. Advancement of AZD1656 into Phase II trials demonstrates Conduit’s commitment to addressing unmet needs in multisystem autoimmune diseases where current treatments are often limited.
Conduit is partnering with Charles River Laboratories for preclinical lupus studies and Agility [Life Sciences](https://www.clinicaltrialvanguard.com/executiveinterviews/scaling-genai-in-life-sciences-overcoming-barriers-with-strategy-compliance-and-human-insight/) for formulation development. The company has secured composition-of-matter patents for AZD1656 in Japan and Australia, with pending applications in other regions, strengthening its IP portfolio. Formulation optimization efforts for AZD1656, AZD5904, and AZD5658 aim to improve drug delivery and patient compliance.
These developments position Conduit for potential growth and partnerships. Positive preclinical and Phase IIa results could attract collaborators or licensing agreements, accelerating development and commercialization. A robust IP portfolio further enhances the value of Conduit’s pipeline and strengthens its position for future out-licensing deals. Successful clinical outcomes could ultimately lead to new, more effective treatment options for patients with autoimmune diseases.
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### [Tenaya Therapeutics Unveils Breakthrough Cardiac Data at ACC Conference](https://www.clinicaltrialvanguard.com/news/tenaya-therapeutics-unveils-breakthrough-cardiac-data-at-acc-conference/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
Tenaya Therapeutics will present new clinical data from the [MyPEAK](https://www.clinicaltrialvanguard.com/news/tenaya-therapeutics-unveils-promising-mypeak-1-trial-data-for-tn-201/)-1 Phase 1b/2 trial of [TN-201](https://www.clinicaltrialvanguard.com/news/tenayas-tn-201-gene-therapy-shows-hypertrophy-reduction-in-hcm-patients/), a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), at the American College of Cardiology’s Annual Scientific Session. The data will include one-year assessments of the first two adult patients treated with TN-201 and six-month data from the third patient. A separate presentation will detail findings from the SHaRe registry, highlighting the disease burden in adults with •MYBPC3•-associated HCM.
These presentations are important because they offer further insights into the potential of TN-201 as a treatment for this genetic form of HCM. Data from the first few patients treated with a novel gene therapy provide crucial early signals of safety and efficacy, informing the future development of the therapy and potentially offering hope to patients with this often debilitating condition. The SHaRe registry data adds to the understanding of the impact of this specific genetic mutation on patients, potentially helping clinicians better identify and manage individuals with •MYBPC3•-associated HCM.
The late-breaking presentation on TN-201 will build on the encouraging preliminary data shared in December 2024. The one-year follow-up data from the initial patients are particularly noteworthy, as they offer a longer-term perspective on the treatment’s potential durability and sustained impact. The presentation on the SHaRe registry will provide a more detailed analysis of the disease burden associated with •MYBPC3• mutations, potentially informing clinical practice and highlighting the unmet medical need within this specific patient population.
The upcoming data presentations represent a significant step in the development of TN-201 and the broader field of gene therapy for cardiovascular diseases. Positive results could further validate this approach, attracting further investment and research into gene-based therapies for HCM and other inherited heart conditions. This progress may ultimately lead to new treatment options for patients with currently limited therapeutic choices.
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### [Savolitinib Shines in HutchMed Phase II Lung Cancer Trial](https://www.clinicaltrialvanguard.com/news/savolitinib-shines-in-hutchmed-phase-ii-lung-cancer-trial/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
[HUTCHMED](https://www.clinicaltrialvanguard.com/news/hutchmed-highlights-new-lung-cancer-data-at-2025-conferences/) announced new data from studies of savolitinib and surufatinib, two compounds it discovered, will be presented at the European Lung Cancer Congress 2025. The SAVANNAH Phase II trial demonstrated promising results for savolitinib combined with [TAGRISSO](https://www.clinicaltrialvanguard.com/news/tagrisso-approved-for-unresectable-stage-iii-egfr-mutated-lung-cancer/) (osimertinib) in patients with MET-high EGFR-mutated non-[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC) whose disease progressed after initial TAGRISSO treatment. Additionally, updated data from a Phase IIIb savolitinib study showcased long-term survival benefits and safety in patients with METex14 NSCLC.
These findings are crucial for addressing the significant unmet need for effective therapies in later-line treatment of EGFR-mutated NSCLC. The SAVANNAH trial results suggest a new potential chemo-free treatment option for patients who develop resistance to first-line TAGRISSO, a common occurrence. Similarly, the positive survival data from the savolitinib Phase IIIb trial reinforces the drug’s potential as a valuable treatment for METex14 NSCLC, particularly for treatment-naïve patients, including those with brain metastasis.
The SAVANNAH trial showed a confirmed objective response rate of 55-56% with savolitinib plus TAGRISSO, and a median duration of response of 7.1-9.9 months. Median progression-free survival was 7.4-7.5 months. Safety data showed no new concerns, with adverse events consistent with the known profiles of the individual drugs. The Phase IIIb savolitinib trial demonstrated a median overall survival of 28.3 months in treatment-naïve patients and 25.3 months in previously treated patients. Notably, even patients with baseline brain metastasis experienced survival benefits. A separate study evaluating surufatinib plus immunotherapy as maintenance therapy following first-line chemo-immunotherapy in extensive-stage SCLC showed promising 12- and 18-month overall survival rates.
These data strengthen the potential of savolitinib as a key treatment option for specific NSCLC patient populations. The ongoing global SAFFRON Phase III trial will further evaluate the combination of savolitinib and TAGRISSO against chemotherapy, offering further insights into its efficacy. The positive results for surufatinib also suggest a potential future role for this drug in extending survival for SCLC patients. HUTCHMED’s focus on targeted therapies and immunotherapies continues to address critical needs within the oncology landscape.
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### [Genenta Secures €20M, Brain Tumor Trial Shows Promising Survival](https://www.clinicaltrialvanguard.com/news/genenta-secures-e20m-brain-tumor-trial-shows-promising-survival/)
**Published:** March 20, 2025
**Author:** Jon Napitupulu
**Content:**
Genenta Science secured €20 million (approximately $21.9 million) in financing from ENEA Tech and Biomedical (ETB) to support the expansion of its clinical pipeline, specifically for Temferon in [metastatic Renal Cell Carcinoma](https://www.clinicaltrialvanguard.com/news/adicet-opens-adi-270-phase-1-trial-enrollment-for-metastatic-renal-cell-carcinoma/) (mRCC). ETB, a prominent Italian foundation managing substantial assets, conducted extensive due diligence before investing in Genenta, demonstrating confidence in the company’s potential. The funding comes as Genenta reports encouraging data from its Phase 1/2a Glioblastoma Multiforme (GBM) trial and initiates a Phase 1/2a trial for Temferon in mRCC.
This funding is crucial for Genenta because it fuels the development of Temferon in a new indication, mRCC, thereby potentially broadening the therapy’s clinical impact. The non-dilutive nature of the mandatory convertible bond, at least for the next three years, preserves shareholder value in the near term, while the involvement of a respected institution like ETB lends credibility to Genenta’s scientific approach and long-term prospects. This strategic investment allows Genenta to focus on achieving clinical milestones without the immediate pressure of equity dilution, which is especially important for a clinical-stage biotech company.
The financing is structured as a three-year mandatory convertible bond with a two-year lock-up period following conversion in March 2028. The investment is divided into two tranches: an initial €7.5 million to support the ongoing mRCC trial’s safety assessment, and a subsequent €12.5 million contingent upon achieving safety and tolerability milestones in the mRCC trial. ETB’s equity in Genenta will be capped at 29%, with a maximum conversion price of $17.64 per share. Meanwhile, updated data from the GBM trial shows promising survival rates, further validating Temferon’s potential to reprogram the tumor microenvironment and induce immune responses.
This injection of capital, coupled with positive clinical data, positions Genenta to make significant advancements in its clinical programs. The successful execution of the mRCC trial will be pivotal for demonstrating Temferon’s versatility across different cancer types. This progress could attract further investment and potential partnerships, solidifying Genenta’s position as a key player in the immuno-oncology space. The combination of financial stability and promising clinical results sets a positive trajectory for Genenta’s future and its potential to impact cancer treatment.
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### [WuGen Announces Dosing in Wu-CART-007 Trial](https://www.clinicaltrialvanguard.com/news/wugen-announces-dosing-in-wu-cart-007-trial/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
Wugen, Inc. has begun its pivotal Phase 2 trial of WU-CART-007, an off-the-shelf CAR-T [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for relapsed or refractory T-cell acute lymphoblastic [leukemia](https://www.clinicaltrialvanguard.com/news/quetzal-launches-phase-iii-trial-for-leukemia-drug-qtx-2101/)/lymphoma (T-ALL/LBL) in pediatric and adult patients. This follows promising Phase 1/2 results, which showed a 91% overall response rate and a 73% composite complete remission rate in heavily pre-treated patients. The therapy has received multiple FDA accelerated approval designations, including RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease, as well as Priority Medicines designation in the EU.
This pivotal trial initiation is particularly important because T-ALL/LBL patients haven’t seen a new treatment approved in 20 years, especially for those with relapsed or refractory disease who face limited options and poor outcomes. The off-the-shelf nature of WU-CART-007 addresses a crucial unmet need by potentially providing faster and more accessible treatment compared to traditional autologous CAR-T therapies, which require individualized manufacturing from the patient’s own cells. The rapid enrollment rate observed so far underscores the high demand for new therapies in this area.
The Phase 2 T-RRex study is a single-arm trial evaluating WU-CART-007’s efficacy and safety in two patient cohorts: one with relapsed/refractory disease and another exploratory cohort with minimal residual disease. WU-CART-007 utilizes [CRISPR](https://www.clinicaltrialvanguard.com/opinion/spatial-crispr-screening-just-made-your-preclinical-models-look-like-guesswork/) gene editing to enhance its functionality. By deleting the CD7 target and the TRAC gene in the CAR-T cells, the therapy avoids self-destruction (fratricide) and reduces the risk of graft-versus-host disease, respectively.
Positive results from this pivotal trial could lead to regulatory approval and significantly alter the treatment landscape for T-ALL/LBL. This would offer a readily available and potentially curative therapy for patients who currently have few options. The trial’s success could also further validate Wugen’s allogeneic CAR-T platform and pave the way for the development of similar therapies for other hematological and solid tumor malignancies.
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### [Celz-201-DDT FDA Clearance for Chronic Lower Back Pain Trial](https://www.clinicaltrialvanguard.com/news/celz-201-ddt-fda-clearance-for-chronic-lower-back-pain-trial/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
Creative Medical Technology Holdings (CELZ) announced positive interim data from its Phase 1/2 trial of [StemSpine](https://www.clinicaltrialvanguard.com/news/stemspine-procedure-significantly-reduces-opioid-use-in-chronic-lower-back-pain-patients/) using AlloStem (CELZ-201-DDT), a donor [cell therapy](https://www.clinicaltrialvanguard.com/news/rheumatologists-powerful-hope-cell-therapy-for-sclerosis/) for chronic lower back pain due to degenerative disc disease (DDD). The FDA cleared an expanded dose escalation for the trial following statistically significant pain reduction and mobility improvement in participants. The ongoing trial, which is the first of its kind, uses a minimally invasive, ultrasound-guided injection of CELZ-201-DDT in an outpatient setting.
This development is potentially groundbreaking for patients suffering from chronic lower back pain, a widespread condition often treated with opioids or invasive surgery. The positive interim results, including statistically significant improvements in pain and mobility, suggest that CELZ-201-DDT could offer a much-needed non-opioid, minimally invasive alternative. The strong safety profile demonstrated thus far, with no serious adverse events reported, further strengthens the therapy’s potential to become a standard of care. This progress also validates the outpatient, ultrasound-guided injection approach, making it accessible to a broader patient population.
The trial utilizes a 4:1 treatment-to-placebo ratio and has reached its halfway point. Importantly, no dose-limiting toxicities or serious adverse events have been observed. The FDA’s clearance for dose expansion allows CELZ to optimize the therapy and potentially enhance its effectiveness. The Data Safety Monitoring Board (DSMB) and Institutional Review Board (IRB) have approved this new dosing strategy, which has already been implemented.
The positive interim data and FDA clearance for dose escalation bode well for CELZ and the future of chronic lower back pain treatment. If the positive trend continues in the second half of the trial, it could accelerate the path towards a pivotal Phase 3 trial and a subsequent [Biologics License Application](https://www.clinicaltrialvanguard.com/news/regeneron-eylea-hd-8-mg-update-biologics-license-application/) (BLA) submission to the FDA. This could ultimately lead to a paradigm shift in how chronic lower back pain is managed, offering patients a safer and more effective alternative to current treatments.
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### [Pathos AI Doses First Patient in Pocenbrodib Trial](https://www.clinicaltrialvanguard.com/news/pathos-ai-doses-first-patient-in-pocenbrodib-trial/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
Pathos AI has initiated a Phase 1b/2a clinical trial for its CBP/p300 inhibitor, pocenbrodib, in patients with metastatic castration-resistant [prostate cancer](https://www.clinicaltrialvanguard.com/news/oric-launches-phase-3-trial-of-rinzimetostat-in-metastatic-prostate-cancer/) (mCRPC). The trial will evaluate pocenbrodib as a monotherapy and in combination with existing treatments like abiraterone acetate, olaparib, or 177Lu-PSMA-617. This marks the first clinical-stage asset for Pathos AI.
This trial is significant because it addresses a critical unmet need in mCRPC treatment. Many patients develop resistance to standard anti-androgen therapies, leaving them with limited options. Pocenbrodib’s mechanism of action, inhibiting CBP/p300 proteins that drive cancer cell growth, offers a novel approach to combatting this resistance. Moreover, Pathos AI utilizes a sophisticated biomarker strategy to identify patients most likely to respond, potentially optimizing treatment outcomes.
The P300-02-001 study is a multicenter, open-label trial aiming to enroll approximately 203 mCRPC patients who have progressed after prior therapies. The study’s primary objectives are to evaluate the safety, objective response rate, PSA decline, and pharmacokinetic/pharmacodynamic profile of pocenbrodib, both alone and in combination regimens. This data will inform the recommended Phase 2 dose for future studies.
The commencement of this trial represents a pivotal step for Pathos AI and the development of pocenbrodib. Positive results could validate their precision medicine approach and establish pocenbrodib as a valuable new therapy for mCRPC patients who have exhausted other treatment avenues. Furthermore, it could pave the way for exploring pocenbrodib’s efficacy in other cancer types, solidifying its potential as a versatile therapeutic agent.
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### [Soquelitinib Data in T-Cell Lymphoma: Promising Results Unveiled](https://www.clinicaltrialvanguard.com/news/soquelitinib-data-in-t-cell-lymphoma-promising-results-unveiled/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
[Corvus Pharmaceuticals](https://www.clinicaltrialvanguard.com/news/corvus-pharmaceuticals-2024-financial-results/) released additional data from its Phase 1/1b clinical trial of [soquelitinib](https://www.clinicaltrialvanguard.com/news/soquelitinib-ind-approved-for-atopic-dermatitis-trial-in-china/), an oral ITK inhibitor, for T-cell [lymphoma](https://www.clinicaltrialvanguard.com/news/abelzeta-gets-fda-ind-clearance-for-prizlon-cel-in-large-b-cell-lymphoma/) (TCL). The data, presented at the 16th Annual T-Cell Lymphoma Forum, showed promising anti-tumor activity and a reduction in T-cell exhaustion. Based on these positive results, Corvus is currently enrolling patients in a registrational Phase 3 trial.
This news is important because it offers potential advancements in TCL treatment. Current standard therapies like belinostat and pralatrexate have limited effectiveness, particularly in relapsed patients, who face a median progression-free survival of only 3 to 4 months. Soquelitinib’s observed 30% progression-free survival rate at 18 months, if confirmed in larger trials, could represent a substantial improvement in outcomes for these patients. The reduction in T-cell exhaustion observed in the study also suggests a novel mechanism of action that could further enhance anti-tumor immunity.
The Phase 1/1b trial of soquelitinib enrolled 25 patients at the optimal dose of 200 mg twice daily. Of the 23 evaluable patients, 39% experienced objective responses (complete or partial), with a median duration of response of 17.2 months. Importantly, three patients remain on therapy at 14+ months, 18+ months, and 25+ months. The drug was well-tolerated without new safety signals, dose reductions, or treatment interruptions. The Phase 3 trial will compare soquelitinib against physician’s choice of either belinostat or pralatrexate in 150 patients with relapsed peripheral T-cell lymphoma (PTCL), aiming to confirm the efficacy and safety profile observed in earlier trials.
The positive data from the Phase 1/1b trial strengthens the potential of soquelitinib as a viable treatment option for relapsed PTCL. The ongoing Phase 3 trial is a crucial step toward potential regulatory approval and, if successful, could significantly alter the treatment landscape for this aggressive and often fatal form of cancer. This development underscores the potential of ITK inhibition as a novel approach to cancer immunotherapy, paving the way for further research and development in this area.
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### [Briacell's Phase 3 Breast Cancer Study Gets Positive Recommendation](https://www.clinicaltrialvanguard.com/news/briacells-phase-3-breast-cancer-study-gets-positive-recommendation/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
[BriaCell](https://www.clinicaltrialvanguard.com/news/briacell-to-present-phase-2-3-clinical-survival-data/) Therapeutics Corp. announced positive safety results from the second review of its Phase 3 study of Bria-IMT™ combined with an immune checkpoint inhibitor in patients with metastatic [breast cancer](https://www.clinicaltrialvanguard.com/news/phesi-data-emerging-clinical-trial-shift-to-diabetes/). The independent Data Safety Monitoring Board (DSMB) found no safety concerns and recommended the continuation of the study, which has Fast Track Designation from the FDA.
This positive DSMB review builds confidence in the safety profile of Bria-IMT™ in combination with a CPI, a critical factor for patients considering treatment options for metastatic breast cancer, a disease with limited effective therapies. The continuation of the Phase 3 trial, especially under Fast Track designation, accelerates the potential availability of this novel immunotherapy combination for patients facing a life-threatening diagnosis. This progress not only offers hope for improved outcomes but also further validates BriaCell’s approach to cancer treatment.
The DSMB’s recommendation to continue the trial without modification suggests a favorable safety profile observed thus far. This is particularly significant in the context of combination therapies, where the potential for adverse events can be higher. The Fast Track designation underscores the unmet medical need in metastatic breast cancer and the potential of Bria-IMT™ to address this need. While detailed efficacy data are yet to be released, the safety findings pave the way for continued evaluation of the treatment’s potential to improve survival and quality of life for these patients.
This positive safety review represents a significant step forward for BriaCell and for the development of Bria-IMT™ as a potential treatment for metastatic breast cancer. Continued positive results could lead to an accelerated regulatory pathway and ultimately provide a much-needed new therapeutic option for patients with this aggressive form of cancer. The progress of this Phase 3 study will be closely watched by both the medical community and investors.
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### [Nanobiotix Data from Two Phase 1 NBTXR3 Studies at ELC](https://www.clinicaltrialvanguard.com/news/nanobiotix-data-from-two-phase-1-nbtxr3-studies-at-elc/)
**Published:** March 21, 2025
**Author:** Jon Napitupulu
**Content:**
Nanobiotix announced two poster presentations at the 2025 European Lung Cancer Conference showcasing Phase 1 study data for [JNJ-1900](https://www.clinicaltrialvanguard.com/news/promising-early-data-from-nbtxr3-phase-1-study-in-advanced-nsclc/) (NBTXR3) in patients with [non-small cell lung cancer](https://www.clinicaltrialvanguard.com/news/krystal-biotech-focuses-on-inhaled-kb707-for-non-small-cell-lung-cancer/)[small cell lung cancer](https://www.clinicaltrialvanguard.com/news/sn-bioscience-begins-phase-1b-2-trial-of-snb-101-for-small-cell-lung-cancer/) (NSCLC). One study evaluated the safety and efficacy of re-irradiation with NBTXR3 in patients with unresectable, locoregional recurrent NSCLC. The other explored the safety and feasibility of injecting NBTXR3 in combination with nivolumab or pembrolizumab in patients with lung metastases originating from NSCLC or other solid tumors.
These presentations are important because they provide preliminary insights into the potential of NBTXR3 in expanding treatment options for lung cancer, a leading cause of cancer-related deaths worldwide. The studies explore the use of NBTXR3 in challenging clinical scenarios like recurrent and metastatic disease, where new therapies are critically needed. Positive data could significantly advance the development of NBTXR3 in lung cancer, potentially leading to improved outcomes for patients who have limited treatment choices.
The first presentation showcased updated local progression-free survival data from the completed dose-escalation portion of a Phase 1 re-irradiation study. The second highlighted safety and injection feasibility data from a Phase 1 study combining NBTXR3 with checkpoint inhibitors. Both studies are crucial stepping stones towards larger, more definitive clinical trials.
The data presented at ELCC represent a significant step in validating the potential of NBTXR3 in lung cancer. Positive findings could lead to accelerated development timelines, further clinical investigation, and ultimately, provide a new therapeutic approach for patients with this aggressive and often difficult-to-treat disease. Further investigation will be needed to confirm these early signals and determine the optimal use of NBTXR3 in different lung cancer settings.
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### [Opthea Coast Phase 3 Trial Fails to Meet Primary Endpoint](https://www.clinicaltrialvanguard.com/news/opthea-coast-phase-3-trial-fails-to-meet-primary-endpoint/)
**Published:** March 24, 2025
**Author:** Jon Napitupulu
**Content:**
Opthea Limited’s Phase 3 COAST trial for sozinibercept, a combination therapy with aflibercept for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (wet AMD), failed to meet its primary endpoint of improved best corrected visual acuity (BCVA). The trial showed no significant difference in BCVA improvement between patients receiving sozinibercept combined with aflibercept and those receiving aflibercept alone. This negative result triggers potential financial obligations under Opthea’s Development Funding Agreement (DFA) and casts doubt on the company’s ability to continue operating.
This failed trial is a significant setback for Opthea and potentially impacts the wet AMD treatment landscape. Sozinibercept was a promising candidate that aimed to improve upon the current standard of care, offering the possibility of better vision outcomes for patients. The negative results not only halt the drug’s development but also raise questions about the viability of targeting this specific biological pathway for wet AMD treatment. This could lead to a shift in research and development focus within the industry.
The COAST trial involved various dosing regimens of sozinibercept in combination with aflibercept, compared to aflibercept monotherapy. None of the sozinibercept combinations demonstrated a statistically significant improvement in BCVA. Opthea’s financial situation is now precarious due to the DFA. The company may be obligated to repay substantial sums to investors, potentially exceeding its current cash reserves. Opthea’s assets are secured by the DFA investors, limiting the company’s options for raising additional funds.
The future of Opthea is uncertain. The company is in discussions with DFA investors to explore potential options, including negotiated settlements or alternative development paths. However, the company acknowledges a material uncertainty regarding its ability to continue as a going concern. The outcome of these discussions will determine whether Opthea can restructure its operations, secure further funding, or potentially face insolvency. The failure of the COAST trial represents a substantial hurdle for the company and highlights the inherent risks in pharmaceutical development.
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**Categories:** News
---
### [Silexion Unveils Sil204 Plan at NEAUXCancer 2025](https://www.clinicaltrialvanguard.com/news/silexion-unveils-sil204-plan-at-neauxcancer-2025/)
**Published:** March 24, 2025
**Author:** Jon Napitupulu
**Content:**
[Silexion Therapeutics](https://www.clinicaltrialvanguard.com/news/silexion-therapeutics-unveils-pioneering-rnai-technology-from-silexion-therapeutics-for-revolutionizing-the-fight-against-kras-driven-cancers/) has developed an expanded plan for [SIL204](https://www.clinicaltrialvanguard.com/news/silexion-submits-pancreatic-cancer-trial-app-in-israel/), its next-generation siRNA candidate targeting KRAS-driven cancers. The company will present this plan, along with recent groundbreaking preclinical data, at the 2025 NeauxCancer Conference in New Orleans. This new strategy builds on promising preclinical results and explores multiple delivery methods for SIL204.
This development is particularly important because it demonstrates Silexion’s commitment to addressing the significant challenge of KRAS-driven cancers, specifically [pancreatic cancer](https://www.clinicaltrialvanguard.com/opinion/daraxonrasibs-resistance-map-is-a-blueprint-for-how-oncology-trials-should-be-designed/). The expanded plan suggests a more comprehensive and potentially more effective approach to targeting these cancers than previously indicated. The presentation of groundbreaking preclinical data alongside the new strategy reinforces the scientific rationale behind the expanded plan and offers potential investors concrete evidence of SIL204’s promise.
The expanded development plan for SIL204 leverages recent preclinical findings, including data from orthotopic models. This suggests the company is focusing on models that more accurately represent the tumor microenvironment, potentially leading to more clinically relevant results. The plan will encompass multiple delivery approaches for the drug candidate, aiming to maximize its therapeutic potential. The presentation will occur during the Innovation track of the NeauxCancer Conference, highlighting the novelty of Silexion’s approach.
This announcement signals a significant step forward for Silexion and potentially for the treatment of KRAS-driven cancers. The expanded plan, combined with positive preclinical data, could attract further investment and accelerate the clinical development of SIL204. The unveiling of this plan at a prominent industry conference provides valuable exposure and positions Silexion as a key player in the ongoing effort to develop effective therapies for these challenging cancers.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Clearmind's Psychedelic CMND-100 Arrives in US for Yale Trial](https://www.clinicaltrialvanguard.com/news/clearminds-cmnd-100-arrives-in-us-for-fda-trial/)
**Published:** March 24, 2025
**Author:** Jon Napitupulu
**Content:**
Clearmind Medicine Inc. (Nasdaq: CMND) has shipped its novel psychedelic-derived drug candidate, [CMND-100](https://www.clinicaltrialvanguard.com/news/clearmind-medicine-trial-soars-after-safety-board-approval/), to the United States for its upcoming Phase I/IIa clinical trial targeting Alcohol Use Disorder (AUD). This trial, approved by the FDA, will be conducted at Yale School of Medicine, Johns Hopkins University School of Medicine, and IMCA in Israel. CMND-100, an oral MEAI-based drug, aims to reduce alcohol consumption and cravings through a novel mechanism of action.
The successful shipment of CMND-100 and the commencement of the clinical trial are critical steps for Clearmind as it tackles the substantial, underserved AUD market. The prevalence of AUD, impacting over 28 million adults in the U.S. alone, coupled with the limited effectiveness of existing treatments, creates a significant opportunity for innovative therapies like CMND-100. The selection of prestigious research institutions like Yale and Johns Hopkins underscores the potential of this drug candidate.
This Phase I/IIa trial will evaluate both the safety and efficacy of CMND-100 in reducing alcohol consumption. The trial’s initiation keeps Clearmind on track for its first-in-human study of this potentially groundbreaking AUD treatment. While financial details weren’t disclosed in the announcement, the pursuit of this large market suggests significant future revenue potential should the trial prove successful.
Positive clinical trial results could position Clearmind as a leader in the next generation of AUD treatments, potentially reshaping the landscape of addiction therapy. This progress may also attract further investment and partnerships, accelerating the development and commercialization of CMND-100. The upcoming clinical trial represents a crucial inflection point for Clearmind, the outcome of which will significantly influence the company’s future trajectory.
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**Categories:** News
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---
### [Move Over Pharma: Patient-Led Organizations Advance Rare Disease Clinical Trials](https://www.clinicaltrialvanguard.com/conference-coverage/move-over-pharma-patient-led-organizations-advance-rare-disease-clinical-trials/)
**Published:** March 24, 2025
**Author:** Moe Alsumidaie
**Content:**
At the 2025 [SCOPE](https://www.clinicaltrialvanguard.com/conference-coverage/2025-scope-summit-advancing-patient-centric-clinical-trials/) Summit, industry leaders and patient advocates discussed a transformative shift in the pharmaceutical industry: patient-led disruption in clinical trials. The panel, featuring Craig Lipset from the Decentralized Trials & Research Alliance, Deirdre BeVard of CSL Innovation GmbH, Heidi Bjornson-Pennell from the Chan Zuckerberg Initiative, Nasha Fitter of Citizen Health Inc, Craig Martin from the Orphan Therapeutics Accelerator, and Tracey Sikora from the National Organization for Rare Disorders, explored how patients are evolving from passive participants to active leaders in drug development. This shift is crucial for addressing unmet needs in rare and ultra-rare diseases and promises to reshape the clinical trial landscape by fostering partnerships with patient organizations.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#the-need-for-disruption-in-clinical-trials)The Need for Disruption in Clinical Trials
The traditional shareholder-driven model often overlooks rare disease communities, prioritizing high commercial potential molecules. While effective for the majority, it fails to keep pace with scientific advancements, leaving many patients without viable treatment options. The panelists argued for a more inclusive approach, where patients are integral to the process, ensuring that all patient needs are met and addressing the gaps left by traditional pharma.
#### [](#patients-as-leaders-in-drug-development)Patients as Leaders in Drug Development
Craig Lipset highlighted a paradigm shift where patients are not just participants but leaders in drug development. He recounted an experience where a patient advocate challenged the notion of pharmaceutical ownership in drug development, underscoring the need for a more inclusive approach. This shift is crucial as traditional models often leave rare disease communities behind due to their focus on commercial potential. Lipset emphasized the importance of integrating patient insights to keep pace with scientific advancements and ensure viable treatment options for all patients.
Nasha Fitter, co-founder of the Fox G1 Research Foundation, exemplified how patient organizations are stepping up to fill the void left by traditional pharma. Her foundation is developing a gene therapy for FoxG1 syndrome, a rare condition affecting about 1,200 children globally. Despite her tech background, Fitter has driven drug development, demonstrating the power of patient-led initiatives. Her organization has advanced its therapy to clinical trials with a $25 million budget, showcasing the efficiency and effectiveness of patient-driven efforts in areas neglected by larger pharmaceutical companies.
#### [](#innovative-models-for-rare-diseases)Innovative Models for Rare Diseases
Craig Martin discussed systemic issues leading to shelving promising therapies for rare diseases. The traditional model, designed for blockbuster drugs, often leaves rare conditions without viable options. Martin’s Orphan Therapeutics Accelerator addresses this by advancing therapies through alternative commercial models, focusing on scientific value rather than commercial potential. This approach aims to bring much-needed therapies to patients who would otherwise be left behind, addressing the commercial challenges and aligning with scientific advancements in rare disease research.
Martin highlighted the misalignment of incentives in drug development, with the process built around blockbusters rather than precision medicines for lower-prevalence diseases. Despite incentives like the Orphan Drug Act, rising capital costs have reduced investment in rare diseases. The Orphan Therapeutics Accelerator fills this gap by taking shelved therapies and setting different expectations for returns, ensuring valuable treatments reach patients, and addressing commercial challenges.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#ai-and-data-driven-approaches)AI and Data-Driven Approaches
Tracey Sikora highlighted AI’s transformative potential in drug repurposing. Her nonprofit, EveryCare, uses AI to match existing drugs with neglected diseases, identifying new therapeutic opportunities. Sikora emphasized that AI can uncover missed opportunities by analyzing existing data to identify potential new uses for drugs. This method benefits rare diseases, where traditional models may not see value in investing. By leveraging AI and removing commercial constraints, EveryCare efficiently allocates resources to promising drug-disease matches, ensuring even the smallest patient populations have access to potentially life-saving treatments, exemplifying how technology can overcome traditional model limitations.
#### [](#building-capacity-and-partnerships)Building Capacity and Partnerships
Heidi Bjornson-Pennell emphasized the critical role of capacity building within patient organizations. These groups, often led by individuals with personal stakes in the diseases they fight, are essential partners in advancing research. However, they frequently operate with limited resources and require support to maximize their impact. By investing in these organizations, the industry can foster effective partnerships and accelerate progress in rare disease research, as demonstrated by patient organizations successfully advancing clinical trials. Bjornson-Pennell highlighted examples of patient organizations advancing clinical trials, demonstrating the potential for collaboration between industry and patient groups, empowering patients, and integrating their insights into the drug development process for more effective outcomes.
[](https://clineco.io/commons?utm_medium=display&utm_source=clinical-trial-vanguard&utm_campaign=ctv-partner)
#### [](#summary)Summary
The SCOPE Summit 2025 underscored the transformative potential of patient-led disruption in clinical trials. By embracing innovative models and fostering partnerships with patient organizations, the industry can better address the needs of rare disease communities and drive meaningful progress in drug development. This shift promises to reshape the clinical trial landscape, democratize treatment access for neglected patient populations, and lead to a more inclusive and effective healthcare system.
**Categories:** Article: Conference Coverage
---
### [Tonix Pharmaceuticals Advances NDA: TNX-102 SL for Fibromyalgia](https://www.clinicaltrialvanguard.com/news/tonix-pharmaceuticals-advances-nda-tnx-102-sl-for-fibromyalgia/)
**Published:** March 25, 2025
**Author:** Jon Napitupulu
**Content:**
Tonix Pharmaceuticals announced that the FDA will not require an advisory committee meeting for their New Drug Application (NDA) for [TNX-102](https://www.clinicaltrialvanguard.com/news/tonix-launches-phase-2-horizon-trial-of-tnx-102-sl-for-major-depressive-disorder/) SL, a sublingual cyclobenzaprine tablet for fibromyalgia management. The FDA set a PDUFA goal date of August 15, 2025, and if approved, TNX-102 SL would be the first new fibromyalgia treatment in 15 years. Tonix is preparing for a potential fourth-quarter 2025 launch.
This accelerated regulatory pathway underscores the significant unmet need in fibromyalgia treatment. Current therapies often leave patients and physicians dissatisfied, highlighting the potential for TNX-102 SL to improve the lives of millions suffering from this chronic pain condition. The absence of an advisory committee meeting streamlines the approval process and suggests the FDA has sufficient information to make its decision, potentially indicating confidence in the drug’s safety and efficacy data.
TNX-102 SL is a non-opioid analgesic designed for bedtime use. Its sublingual formulation allows for rapid absorption and potentially reduces daytime drowsiness compared to oral cyclobenzaprine. The drug’s mechanism of action targets multiple neuroreceptors believed to contribute to the non-restorative sleep experienced by fibromyalgia patients. Tonix holds patents for the sublingual formulation, offering market protection until 2034.
The FDA’s decision regarding TNX-102 SL could reshape the fibromyalgia treatment landscape. A potential approval would offer patients a new therapeutic option, potentially addressing the limitations of current treatments. This also positions Tonix for significant growth, establishing them as a key player in the pain management market. The coming months will be crucial as the PDUFA date approaches, potentially bringing a much-needed advancement for fibromyalgia patients.
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**Categories:** News
**Tags:** eClinical Tech News
---
### [Clearside Biomedical Announces Six ARVO 2025 Abstracts](https://www.clinicaltrialvanguard.com/news/clearside-biomedical-announces-six-arvo-2025-abstracts/)
**Published:** March 25, 2025
**Author:** Jon Napitupulu
**Content:**
Clearside Biomedical announced the acceptance of six abstracts for presentation at the 2025 ARVO meeting. These abstracts cover topics ranging from clinical trial results for CLS-AX, a treatment for wet age-related [macular degeneration](https://www.clinicaltrialvanguard.com/news/kalaris-launches-promising-new-trial-for-macular-degeneration/) (AMD), to advancements in suprachoroidal drug delivery technology and training. The company highlights its suprachoroidal drug delivery platform as a transformative approach for treating macular diseases.
This development is important for the ophthalmology field because the data presented at ARVO offers further validation of Clearside’s suprachoroidal injection platform and its potential to become a standard treatment for retinal diseases. Positive clinical trial results for CLS-AX, specifically, could lead to a new, potentially longer-lasting treatment option for wet AMD, a leading cause of vision loss. The research on training programs and segmentation algorithms suggests growing investment in making the procedure more accessible and precise for clinicians.
Key information revealed includes top-line results from the Phase 2b ODYSSEY trial for CLS-AX in wet AMD patients. Additional data will be presented on the safety and durability of CLS-AX. Abstracts also cover a decade-long literature review of suprachoroidal drug delivery, validation of a training program for the injection procedure, analysis of drug formulations, and a new algorithm for imaging the suprachoroidal space. This breadth of research demonstrates Clearside’s commitment to advancing the entire ecosystem surrounding suprachoroidal injections.
The presentation of these abstracts at ARVO marks a significant step for Clearside Biomedical. Positive reception of the ODYSSEY trial results, in particular, could pave the way for a Phase 3 trial and eventual commercialization of CLS-AX, positioning Clearside as a key player in the retinal disease treatment market. Furthermore, continued refinement of the injection procedure and related technologies could lead to wider adoption of suprachoroidal drug delivery as a standard of care.
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**Categories:** News
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## Video Sliders
### [Interview with Dr. Olga Kubassova: Imaging in Clinical Trials](https://www.clinicaltrialvanguard.com/video-slider/interview-with-dr-olga-kubassova-imaging-in-clinical-trials/)
**Published:** September 19, 2026
**Author:** Moe Alsumidaie
**Content:**
---
### [Standardizing on the Veeva Vault Platform](https://www.clinicaltrialvanguard.com/video-slider/25815/)
**Published:** June 22, 2026
**Author:** Moe Alsumidaie
**Content:**
---
### [GLP-1 Trials Have an Imaging Problem | Prof. Mikael Boesen, MSK Radiology](https://www.clinicaltrialvanguard.com/video-slider/glp-1-trials-have-an-imaging-problem-prof-mikael-boesen-msk-radiology/)
**Published:** April 20, 2026
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Northwell Health's Christina Brennan](https://www.clinicaltrialvanguard.com/video-slider/interview-with-northwell-healths-christina-brennan/)
**Published:** October 9, 2025
**Author:** Moe Alsumidaie
**Content:**
---
### [Redefining Imaging Workflows in Clinical Trials](https://www.clinicaltrialvanguard.com/video-slider/redefining-imaging-workflows-in-clinical-trials/)
**Published:** September 15, 2025
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Luca Manfro, Recordati](https://www.clinicaltrialvanguard.com/video-slider/interview-with-luca-manfro-recordati/)
**Published:** August 4, 2025
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Novo Nordisk](https://www.clinicaltrialvanguard.com/video-slider/interview-with-novo-nordisk/)
**Published:** July 7, 2025
**Author:** Moe Alsumidaie
**Content:**
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### [Interview with AstraZeneca](https://www.clinicaltrialvanguard.com/video-slider/interview-with-astrazeneca/)
**Published:** June 19, 2025
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Martin Helling Boehringer Ingelheim](https://www.clinicaltrialvanguard.com/video-slider/interview-with-martin-helling-boehringer-ingelheim/)
**Published:** June 13, 2025
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Skylight Health Research Alisha Garibaldi](https://www.clinicaltrialvanguard.com/video-slider/interview-with-skylight-health-research-alisha-garibaldi/)
**Published:** October 21, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Veeva's Kenny Kong](https://www.clinicaltrialvanguard.com/video-slider/interview-with-veevas-kenny-kong/)
**Published:** October 14, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Carolyn Jin from Kura Oncology](https://www.clinicaltrialvanguard.com/video-slider/interview-with-carolyn-jin-from-kura-oncology/)
**Published:** October 10, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Bonne Adams of Inhibrx](https://www.clinicaltrialvanguard.com/video-slider/interview-with-bonne-adams-of-inhibrx/)
**Published:** October 7, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Raviv Pryluk from Phase V](https://www.clinicaltrialvanguard.com/video-slider/raviv-pryluk-from-phase-v/)
**Published:** October 3, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Mumbi Kairu on FDA's Guidance on Diversity Inclusion in Clinical Trials](https://www.clinicaltrialvanguard.com/video-slider/mumbi-kairu-on-fdas-guidance-on-diversity-inclusion-in-clinical-trials/)
**Published:** September 22, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Parexel Peyton Howell](https://www.clinicaltrialvanguard.com/video-slider/interview-with-parexel-peyton-howell/)
**Published:** September 20, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Veeva's Rylan Collins](https://www.clinicaltrialvanguard.com/video-slider/interview-with-veevas-rylan-collins/)
**Published:** September 9, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Interview with Veeva's Richard Young](https://www.clinicaltrialvanguard.com/video-slider/interview-with-veevas-richard-young/)
**Published:** September 9, 2024
**Author:** Moe Alsumidaie
**Content:**
---
### [Advancing Real-World Data to Generate Regulatory Grade Real-World Evidence in Oncology](https://www.clinicaltrialvanguard.com/video-slider/video-1/)
**Published:** August 14, 2024
**Author:** Ashish Kamble
**Content:**
---
### [Translating QbD Principles to Risk-Proportionate Oversight Including RBM: Successes and Challenges](https://www.clinicaltrialvanguard.com/video-slider/video-2/)
**Published:** August 14, 2024
**Author:** Ashish Kamble
**Content:**
---
### [Enhancing Adoption of Innovative Clinical Trial Approaches](https://www.clinicaltrialvanguard.com/video-slider/video-3/)
**Published:** August 14, 2024
**Author:** Ashish Kamble
**Content:**
---
### [Integrating QBD and RBM Approaches in Clinical Trials](https://www.clinicaltrialvanguard.com/video-slider/video-4/)
**Published:** August 14, 2024
**Author:** Ashish Kamble
**Content:**
---
## Episode
### [What are Pragmatic Trials?](https://www.clinicaltrialvanguard.com/podcast/what-are-pragmatic-trials/)
**Published:** March 5, 2025
**Author:** Moe Alsumidaie
**Content:**
The Clinical Trial Vanguard
What are Pragmatic Trials?
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What are Pragmatic Trials?
[Download file](https://www.clinicaltrialvanguard.com/podcast-download/13440/what-are-pragmatic-trials?ref=download "What are Pragmatic Trials? ") | [Play in new window](https://www.clinicaltrialvanguard.com/podcast-download/13440/what-are-pragmatic-trials?ref=new_window "What are Pragmatic Trials? ") | Duration: 14:42 | Recorded on March 5, 2025
Pragmatic trials are a transformative approach in clinical research designed to integrate real-world data into clinical trials. They aim to bridge the gap between clinical research and practice by ensuring that trial results are applicable to broader, real-world populations. Here are some key aspects of pragmatic trials:
**Real-world applicability**: Pragmatic trials are designed to determine whether a therapy works in diverse, real-world settings, addressing a question often unanswered by traditional randomized controlled trials (RCTs) due to their controlled nature and homogeneous populations.
**Flexibility**: Pragmatic trials offer flexibility by allowing the integration of existing data and the enrollment of diverse populations
**Regulatory support**: Regulatory frameworks are essential for supporting pragmatic trials, enabling them to transition from controlled research environments to real-world clinical practice. The FDA is actively working to integrate real-world data into clinical trials.
**Innovation in design**: Pragmatic trials require innovative approaches in clinical trial design to ensure they are efficient and capable of integrating seamlessly into clinical practice.
**Operational considerations**: Implementing pragmatic trials involves addressing operational challenges, such as accommodating diverse patient populations and settings, selecting the correct sites and patients, and providing tailored training and support for research-naive sites.
**The Pressi diagram**: Trial designers can use the Pressi diagram to select pragmatic elements to incorporate, such as broadening inclusion criteria or shifting trial settings from academic centers to community clinics.
**FDA’s Center for Clinical Trial Innovation (C3TI)**: The C3TI aims to integrate pragmatic elements into clinical trials through demonstration programs, supporting the broader adoption of innovative trial designs.
**Podcasts:** The Clinical Trial Vanguard
---
### [Key Takeaways from SCOPE Summit 2025: Budget Negotiations, Protocol Design, and AI in Clinical TrialsSCOPE Summit Summary](https://www.clinicaltrialvanguard.com/podcast/key-takeaways-from-scope-summit-2025-budget-negotiations-protocol-design-and-ai-in-clinical-trialsscope-summit-summary/)
**Published:** March 1, 2025
**Author:** Moe Alsumidaie
**Content:**
The Clinical Trial Vanguard
Key Takeaways from SCOPE Summit 2025: Budget Negotiations, Protocol Design, and AI in Clinical TrialsSCOPE Summit Summary
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Key Takeaways from SCOPE Summit 2025: Budget Negotiations, Protocol Design, and AI in Clinical TrialsSCOPE Summit Summary
[Download file](https://www.clinicaltrialvanguard.com/podcast-download/13286/key-takeaways-from-scope-summit-2025-budget-negotiations-protocol-design-and-ai-in-clinical-trialsscope-summit-summary?ref=download "Key Takeaways from SCOPE Summit 2025: Budget Negotiations, Protocol Design, and AI in Clinical TrialsSCOPE Summit Summary ") | [Play in new window](https://www.clinicaltrialvanguard.com/podcast-download/13286/key-takeaways-from-scope-summit-2025-budget-negotiations-protocol-design-and-ai-in-clinical-trialsscope-summit-summary?ref=new_window "Key Takeaways from SCOPE Summit 2025: Budget Negotiations, Protocol Design, and AI in Clinical TrialsSCOPE Summit Summary ") | Duration: 18:24 | Recorded on March 1, 2025
The SCOPE Summit 2025 addressed key areas in clinical trials: budget negotiations, protocol design, and AI integration.
**Clinical Trial Budget Negotiations:** The summit addressed unresponsive sites and budget discrepancies3. It promoted clear communication, thorough feasibility assessments, and flexible fair market value (FMV) parameters to improve negotiation efficiency and fairness.
**Enhancing Clinical Trial Protocol Design:** Collaboration between Mass General Brigham (MGB), AstraZeneca, and Evinova focuses on improving communication, data utilization, and patient-centric approaches MGB’s HERO initiative and AstraZeneca’s patient partnership program were highlighted as ways to enhance study feasibility, patient engagement, and recruitment.
**AI Integration:** The summit emphasized the transformative role of AI in reshaping clinical research by enhancing productivity and efficiency. Essential skills for AI integration include understanding AI tools, effective prompting, and critical thinking. Collaboration between sponsors and CROs is encouraged to accelerate AI integration, with examples of AI streamlining processes like medical writing.
**Podcasts:** The Clinical Trial Vanguard
---
### [Is Big Pharma in Decline?](https://www.clinicaltrialvanguard.com/podcast/is-big-pharma-in-decline/)
**Published:** September 20, 2024
**Author:** Moe Alsumidaie
**Content:**
CliniCast
Is Big Pharma in Decline?
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Is Big Pharma in Decline?
[Download file](https://www.clinicaltrialvanguard.com/podcast-download/8251/is-big-pharma-in-decline?ref=download "Is Big Pharma in Decline? ") | [Play in new window](https://www.clinicaltrialvanguard.com/podcast-download/8251/is-big-pharma-in-decline?ref=new_window "Is Big Pharma in Decline? ") | Duration: 9:51 | Recorded on September 20, 2024
This is an AI-Generated podcast.
The pharmaceutical industry, often viewed as a pillar of global innovation, is facing a critical juncture as it confronts significant financial headwinds. The sources, particularly the article “Is Big Pharma in Decline?,” reveal declining financial performance indicators, including lower returns on assets (ROA), returns on equity (ROE), EBITDA margins, and current ratios. These declines, attributed to factors like rising operational costs, increased competition, and expiring drug patents, are driving layoffs and forcing companies to re-evaluate their strategies. While the industry is embracing innovations like decentralized clinical trials (DCTs), artificial intelligence (AI) integration, and digital health technologies (DHTs) to streamline operations and potentially reduce costs, concerns linger about the pace of innovation and the ability to overcome these financial challenges. Whether these innovations can be implemented quickly and broadly enough to offset the impending financial strain, particularly in light of anticipated changes to drug pricing policies like the U.S. Inflation Reduction Act (IRA), remains a pressing question for the future of the pharmaceutical industry.
**Podcasts:** CliniCast
---
### [FDA’s Diversity in Clinical Trials Plan: Why It Fails to Address Key Barriers for Underrepresented Communities](https://www.clinicaltrialvanguard.com/podcast/fdas-diversity-in-clinical-trials-plan-why-it-fails-to-address-key-barriers-for-underrepresented-communities/)
**Published:** September 23, 2024
**Author:** Moe Alsumidaie
**Content:**
The Clinical Trial Vanguard
FDA’s Diversity in Clinical Trials Plan: Why It Fails to Address Key Barriers for Underrepresented Communities
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FDA’s Diversity in Clinical Trials Plan: Why It Fails to Address Key Barriers for Underrepresented Communities
[Download file](https://www.clinicaltrialvanguard.com/podcast-download/8266/fdas-diversity-in-clinical-trials-plan-why-it-fails-to-address-key-barriers-for-underrepresented-communities?ref=download "FDA’s Diversity in Clinical Trials Plan: Why It Fails to Address Key Barriers for Underrepresented Communities ") | [Play in new window](https://www.clinicaltrialvanguard.com/podcast-download/8266/fdas-diversity-in-clinical-trials-plan-why-it-fails-to-address-key-barriers-for-underrepresented-communities?ref=new_window "FDA’s Diversity in Clinical Trials Plan: Why It Fails to Address Key Barriers for Underrepresented Communities ") | Duration: 6:39 | Recorded on September 22, 2024
This is an AI-Generated podcast.
This podcast discusses the FDA’s recent guidance on inclusion and diversity in clinical trials and features an interview with Mumbi Kairu, a nurse practitioner at the Pamoja Research Institute. Kairu discusses the crucial need for diversity in clinical trials, noting that while the FDA guidance is a step in the right direction, it does not adequately address the cultural and socioeconomic barriers that prevent diverse participation.
Kairu argues that building community trust should be paramount, as historical events like the Tuskegee experiment still generate mistrust. She suggests involving more community physicians and clinics could help address this, as these practitioners often have closer relationships with their patients and a better understanding of their needs. The podcast also explores the limitations of cultural competency training, suggesting that long-term engagement with communities is essential for building genuine trust. Additionally, the podcast highlights the importance of addressing practical barriers such as transportation, childcare, and job security, which disproportionately affect underrepresented communities. Kairu concludes by emphasizing the need for ongoing support for small clinics and community-based efforts to ensure that clinical trials are genuinely equitable, inclusive, and representative of diverse patient populations.
The podcast aligns with the FDA’s guidance document, which acknowledges the need for greater diversity in clinical trials to improve the generalizability of results and ensure that medical products are safe and effective for all patients. The guidance stresses the importance of sponsors developing and implementing strategies to increase the enrollment of historically underrepresented populations. It suggests measures such as sustained community engagement, cultural competency training, and reducing participant burden. The FDA emphasizes that achieving diversity in clinical trials necessitates a comprehensive, multifaceted approach beyond simply setting goals.
**Podcasts:** The Clinical Trial Vanguard
---
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### [Biomarker Strategy](https://www.clinicaltrialvanguard.com/tag/biomarker-strategy/)
---
### [Adaptive Trial Design](https://www.clinicaltrialvanguard.com/tag/adaptive-trial-design/)
---
### [Pediatric Oncology](https://www.clinicaltrialvanguard.com/tag/pediatric-oncology/)
---
### [FDA Regulation](https://www.clinicaltrialvanguard.com/tag/fda-regulation/)
---
### [Complete Response Letter](https://www.clinicaltrialvanguard.com/tag/complete-response-letter/)
---
### [Oncology Trials](https://www.clinicaltrialvanguard.com/tag/oncology-trials/)
---
### [FDA Regulatory Guidance](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-guidance/)
---
### [Biomarker Validation](https://www.clinicaltrialvanguard.com/tag/biomarker-validation/)
---
### [Algorithmic Bias](https://www.clinicaltrialvanguard.com/tag/algorithmic-bias/)
---
### [Health Equity](https://www.clinicaltrialvanguard.com/tag/health-equity/)
---
### [Real-World Evidence](https://www.clinicaltrialvanguard.com/tag/real-world-evidence/)
---
### [Patient Stratification](https://www.clinicaltrialvanguard.com/tag/patient-stratification/)
---
### [Multiple Myeloma](https://www.clinicaltrialvanguard.com/tag/multiple-myeloma/)
---
### [Oncology Clinical Trials](https://www.clinicaltrialvanguard.com/tag/oncology-clinical-trials/)
---
### [Drug Safety](https://www.clinicaltrialvanguard.com/tag/drug-safety/)
---
### [CNS/Psychiatry Trials](https://www.clinicaltrialvanguard.com/tag/cns-psychiatry-trials/)
---
### [FDA Regulatory Pathway](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-pathway/)
---
### [Phase 1 Trials](https://www.clinicaltrialvanguard.com/tag/phase-1-trials/)
---
### [eClinical Technology](https://www.clinicaltrialvanguard.com/tag/eclinical-technology/)
---
### [Data Integrity](https://www.clinicaltrialvanguard.com/tag/data-integrity/)
---
### [GCP Compliance](https://www.clinicaltrialvanguard.com/tag/gcp-compliance/)
---
### [Endpoint Selection](https://www.clinicaltrialvanguard.com/tag/endpoint-selection/)
---
### [Biomarker Stratification](https://www.clinicaltrialvanguard.com/tag/biomarker-stratification/)
---
### [Endpoint Strategy](https://www.clinicaltrialvanguard.com/tag/endpoint-strategy/)
---
### [FDA Regulatory Policy](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-policy/)
---
### [Biomarkers](https://www.clinicaltrialvanguard.com/tag/biomarkers/)
---
### [Alzheimer's Disease](https://www.clinicaltrialvanguard.com/tag/alzheimers-disease/)
---
### [CNS Trials](https://www.clinicaltrialvanguard.com/tag/cns-trials/)
---
### [CAR-T Therapy](https://www.clinicaltrialvanguard.com/tag/car-t-therapy/)
---
### [Autoimmune Disease](https://www.clinicaltrialvanguard.com/tag/autoimmune-disease/)
---
### [EMA](https://www.clinicaltrialvanguard.com/tag/ema/)
---
### [Antipsychotics](https://www.clinicaltrialvanguard.com/tag/antipsychotics/)
---
### [Clinical Trial Technology](https://www.clinicaltrialvanguard.com/tag/clinical-trial-technology/)
---
### [eClinical Platforms](https://www.clinicaltrialvanguard.com/tag/eclinical-platforms/)
---
### [Cardiovascular Trials](https://www.clinicaltrialvanguard.com/tag/cardiovascular-trials/)
---
### [Immuno-Oncology](https://www.clinicaltrialvanguard.com/tag/immuno-oncology/)
---
### [Clinical Trial Operations](https://www.clinicaltrialvanguard.com/tag/clinical-trial-operations/)
---
### [Phase 3 Trials](https://www.clinicaltrialvanguard.com/tag/phase-3-trials/)
---
### [Parkinson's Disease](https://www.clinicaltrialvanguard.com/tag/parkinsons-disease/)
---
### [Deep Brain Stimulation](https://www.clinicaltrialvanguard.com/tag/deep-brain-stimulation/)
---
### [Neuroimaging](https://www.clinicaltrialvanguard.com/tag/neuroimaging/)
---
### [Cell and Gene Therapy](https://www.clinicaltrialvanguard.com/tag/cell-and-gene-therapy/)
---
### [Manufacturing Changes](https://www.clinicaltrialvanguard.com/tag/manufacturing-changes/)
---
### [Phase III Trials](https://www.clinicaltrialvanguard.com/tag/phase-iii-trials/)
---
### [FDA Regulatory Compliance](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-compliance/)
---
### [GLSI-100](https://www.clinicaltrialvanguard.com/tag/glsi-100/)
---
### [FDA Approval](https://www.clinicaltrialvanguard.com/tag/fda-approval/)
---
### [Hidradenitis Suppurativa](https://www.clinicaltrialvanguard.com/tag/hidradenitis-suppurativa/)
---
### [Drug Development Pipeline](https://www.clinicaltrialvanguard.com/tag/drug-development-pipeline/)
---
### [Infectious Disease](https://www.clinicaltrialvanguard.com/tag/infectious-disease/)
---
### [Preclinical Development](https://www.clinicaltrialvanguard.com/tag/preclinical-development/)
---
### [Global Trial Design](https://www.clinicaltrialvanguard.com/tag/global-trial-design/)
---
### [Enrollment Strategy](https://www.clinicaltrialvanguard.com/tag/enrollment-strategy/)
---
### [INTEL BRIEF](https://www.clinicaltrialvanguard.com/tag/intel-brief/)
---
### [Opioid Use Disorder](https://www.clinicaltrialvanguard.com/tag/opioid-use-disorder/)
---
### [Biomarker-Selected Trials](https://www.clinicaltrialvanguard.com/tag/biomarker-selected-trials/)
---
### [Gastric Cancer](https://www.clinicaltrialvanguard.com/tag/gastric-cancer/)
---
### [Adaptive Trial Strategy](https://www.clinicaltrialvanguard.com/tag/adaptive-trial-strategy/)
---
### [Registrational Trial Design](https://www.clinicaltrialvanguard.com/tag/registrational-trial-design/)
---
### [Breast Cancer Screening](https://www.clinicaltrialvanguard.com/tag/breast-cancer-screening/)
---
### [GLP-1 Receptor Agonists](https://www.clinicaltrialvanguard.com/tag/glp-1-receptor-agonists/)
---
### [Trial Emulation](https://www.clinicaltrialvanguard.com/tag/trial-emulation/)
---
### [Label Expansion](https://www.clinicaltrialvanguard.com/tag/label-expansion/)
---
### [Biomarker Enrichment](https://www.clinicaltrialvanguard.com/tag/biomarker-enrichment/)
---
### [Addiction Psychiatry](https://www.clinicaltrialvanguard.com/tag/addiction-psychiatry/)
---
### [Heart Failure Trials](https://www.clinicaltrialvanguard.com/tag/heart-failure-trials/)
---
### [Precision Oncology](https://www.clinicaltrialvanguard.com/tag/precision-oncology/)
---
### [Wearable Technologies](https://www.clinicaltrialvanguard.com/tag/wearable-technologies/)
---
### [Digital Biomarkers](https://www.clinicaltrialvanguard.com/tag/digital-biomarkers/)
---
### [Clinical Operations](https://www.clinicaltrialvanguard.com/tag/clinical-operations/)
---
### [CNS Clinical Trials](https://www.clinicaltrialvanguard.com/tag/cns-clinical-trials/)
---
### [GLP-1 agonists](https://www.clinicaltrialvanguard.com/tag/glp-1-agonists/)
---
### [dual diagnosis](https://www.clinicaltrialvanguard.com/tag/dual-diagnosis/)
---
### [semaglutide](https://www.clinicaltrialvanguard.com/tag/semaglutide/)
---
### [Non-Small Cell Lung Cancer](https://www.clinicaltrialvanguard.com/tag/non-small-cell-lung-cancer/)
---
### [Immunotherapy Resistance](https://www.clinicaltrialvanguard.com/tag/immunotherapy-resistance/)
---
### [DOGE FDA Budget Cuts](https://www.clinicaltrialvanguard.com/tag/doge-fda-budget-cuts/)
---
### [Companion Diagnostics](https://www.clinicaltrialvanguard.com/tag/companion-diagnostics/)
---
### [Biomarker-Driven Trials](https://www.clinicaltrialvanguard.com/tag/biomarker-driven-trials/)
---
### [CAR-T Cell Therapy](https://www.clinicaltrialvanguard.com/tag/car-t-cell-therapy/)
---
### [Pancreatic Cancer](https://www.clinicaltrialvanguard.com/tag/pancreatic-cancer/)
---
### [HRD Stratification](https://www.clinicaltrialvanguard.com/tag/hrd-stratification/)
---
### [Blood-Brain Barrier](https://www.clinicaltrialvanguard.com/tag/blood-brain-barrier/)
---
### [Phase 3 Trial Design](https://www.clinicaltrialvanguard.com/tag/phase-3-trial-design/)
---
### [Anti-IL-5 Therapy](https://www.clinicaltrialvanguard.com/tag/anti-il-5-therapy/)
---
### [Clinical Trial Endpoints](https://www.clinicaltrialvanguard.com/tag/clinical-trial-endpoints/)
---
### [EMA Guidance](https://www.clinicaltrialvanguard.com/tag/ema-guidance/)
---
### [Virtual Control Groups](https://www.clinicaltrialvanguard.com/tag/virtual-control-groups/)
---
### [Animal Testing Alternatives](https://www.clinicaltrialvanguard.com/tag/animal-testing-alternatives/)
---
### [Obesity Trials](https://www.clinicaltrialvanguard.com/tag/obesity-trials/)
---
### [Patient Adherence](https://www.clinicaltrialvanguard.com/tag/patient-adherence/)
---
### [Treatment-Resistant Depression](https://www.clinicaltrialvanguard.com/tag/treatment-resistant-depression/)
---
### [Ketamine Therapy](https://www.clinicaltrialvanguard.com/tag/ketamine-therapy/)
---
### [CHMP](https://www.clinicaltrialvanguard.com/tag/chmp/)
---
### [Postpartum Depression](https://www.clinicaltrialvanguard.com/tag/postpartum-depression/)
---
### [Perinatal Psychiatry](https://www.clinicaltrialvanguard.com/tag/perinatal-psychiatry/)
---
### [CNS Pipeline](https://www.clinicaltrialvanguard.com/tag/cns-pipeline/)
---
### [Adaptive Trials](https://www.clinicaltrialvanguard.com/tag/adaptive-trials/)
---
### [Orphan Oncology](https://www.clinicaltrialvanguard.com/tag/orphan-oncology/)
---
### [Single-Arm Trials](https://www.clinicaltrialvanguard.com/tag/single-arm-trials/)
---
### [Basket Trials](https://www.clinicaltrialvanguard.com/tag/basket-trials/)
---
### [Lipid Disorders](https://www.clinicaltrialvanguard.com/tag/lipid-disorders/)
---
### [Phase 3 Design](https://www.clinicaltrialvanguard.com/tag/phase-3-design/)
---
### [MRD Endpoints](https://www.clinicaltrialvanguard.com/tag/mrd-endpoints/)
---
### [Protocol Innovation](https://www.clinicaltrialvanguard.com/tag/protocol-innovation/)
---
### [Psychedelic & Ketamine Research](https://www.clinicaltrialvanguard.com/tag/psychedelic-ketamine-research/)
---
### [Expedited Review Pathways](https://www.clinicaltrialvanguard.com/tag/expedited-review-pathways/)
---
### [PSYCH PULSE](https://www.clinicaltrialvanguard.com/tag/psych-pulse/)
---
### [Suicidal Crisis](https://www.clinicaltrialvanguard.com/tag/suicidal-crisis/)
---
### [Drug Development Strategy](https://www.clinicaltrialvanguard.com/tag/drug-development-strategy/)
---
### [Phase 2 Trial Design](https://www.clinicaltrialvanguard.com/tag/phase-2-trial-design/)
---
### [Pharmacovigilance](https://www.clinicaltrialvanguard.com/tag/pharmacovigilance/)
---
### [EMA PRAC](https://www.clinicaltrialvanguard.com/tag/ema-prac/)
---
### [Cenobamate](https://www.clinicaltrialvanguard.com/tag/cenobamate/)
---
### [Safety Monitoring](https://www.clinicaltrialvanguard.com/tag/safety-monitoring/)
---
### [AI in clinical research](https://www.clinicaltrialvanguard.com/tag/ai-in-clinical-research/)
---
### [EU regulatory landscape](https://www.clinicaltrialvanguard.com/tag/eu-regulatory-landscape/)
---
### [Biotech Act](https://www.clinicaltrialvanguard.com/tag/biotech-act/)
---
### [digital endpoints](https://www.clinicaltrialvanguard.com/tag/digital-endpoints/)
---
### [China biotech competition](https://www.clinicaltrialvanguard.com/tag/china-biotech-competition/)
---
### [trial simulation](https://www.clinicaltrialvanguard.com/tag/trial-simulation/)
---
### [QuantHealth](https://www.clinicaltrialvanguard.com/tag/quanthealth/)
---
### [probability of technical success](https://www.clinicaltrialvanguard.com/tag/probability-of-technical-success/)
---
### [real-world data](https://www.clinicaltrialvanguard.com/tag/real-world-data/)
---
### [Novartis](https://www.clinicaltrialvanguard.com/tag/novartis/)
---
### [patient recruitment](https://www.clinicaltrialvanguard.com/tag/patient-recruitment/)
---
### [operating model](https://www.clinicaltrialvanguard.com/tag/operating-model/)
---
### [scalability](https://www.clinicaltrialvanguard.com/tag/scalability/)
---
### [site engagement](https://www.clinicaltrialvanguard.com/tag/site-engagement/)
---
### [patient matching](https://www.clinicaltrialvanguard.com/tag/patient-matching/)
---
### [MindMed](https://www.clinicaltrialvanguard.com/tag/mindmed/)
---
### [LSD](https://www.clinicaltrialvanguard.com/tag/lsd/)
---
### [large language models](https://www.clinicaltrialvanguard.com/tag/large-language-models/)
---
### [generalized anxiety disorder](https://www.clinicaltrialvanguard.com/tag/generalized-anxiety-disorder/)
---
### [major depression](https://www.clinicaltrialvanguard.com/tag/major-depression/)
---
### [central raters](https://www.clinicaltrialvanguard.com/tag/central-raters/)
---
### [Hamilton Anxiety Scale](https://www.clinicaltrialvanguard.com/tag/hamilton-anxiety-scale/)
---
### [MADRS](https://www.clinicaltrialvanguard.com/tag/madrs/)
---
### [phase three](https://www.clinicaltrialvanguard.com/tag/phase-three/)
---
### [psychiatric trials](https://www.clinicaltrialvanguard.com/tag/psychiatric-trials/)
---
### [Clinical Trial Innovation Summit 2025](https://www.clinicaltrialvanguard.com/tag/clinical-trial-innovation-summit-2025/)
---
### [Medical Imaging](https://www.clinicaltrialvanguard.com/tag/medical-imaging/)
---
### [Rare Cancer Trials](https://www.clinicaltrialvanguard.com/tag/rare-cancer-trials/)
---
### [Phase 1/2 Trial Design](https://www.clinicaltrialvanguard.com/tag/phase-1-2-trial-design/)
---
### [Biomarker-Driven Stratification](https://www.clinicaltrialvanguard.com/tag/biomarker-driven-stratification/)
---
### [Magnetic Seizure Therapy](https://www.clinicaltrialvanguard.com/tag/magnetic-seizure-therapy/)
---
### [Electroconvulsive Therapy](https://www.clinicaltrialvanguard.com/tag/electroconvulsive-therapy/)
---
### [Neuromodulation](https://www.clinicaltrialvanguard.com/tag/neuromodulation/)
---
### [ICU Research](https://www.clinicaltrialvanguard.com/tag/icu-research/)
---
### [Protocol Design](https://www.clinicaltrialvanguard.com/tag/protocol-design/)
---
### [Bispecific Antibodies](https://www.clinicaltrialvanguard.com/tag/bispecific-antibodies/)
---
### [FDA Regulatory Authority](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-authority/)
---
### [Synthetic Biology](https://www.clinicaltrialvanguard.com/tag/synthetic-biology/)
---
### [Loper Bright](https://www.clinicaltrialvanguard.com/tag/loper-bright/)
---
### [site readiness](https://www.clinicaltrialvanguard.com/tag/site-readiness/)
---
### [enrollment timelines](https://www.clinicaltrialvanguard.com/tag/enrollment-timelines/)
---
### [protocol deviations](https://www.clinicaltrialvanguard.com/tag/protocol-deviations/)
---
### [CAPA](https://www.clinicaltrialvanguard.com/tag/capa/)
---
### [trial execution](https://www.clinicaltrialvanguard.com/tag/trial-execution/)
---
### [Pragmatic Trial Design](https://www.clinicaltrialvanguard.com/tag/pragmatic-trial-design/)
---
### [Protocol Optimization](https://www.clinicaltrialvanguard.com/tag/protocol-optimization/)
---
### [Site Startup](https://www.clinicaltrialvanguard.com/tag/site-startup/)
---
### [Contract Negotiation](https://www.clinicaltrialvanguard.com/tag/contract-negotiation/)
---
### [CRO Consolidation](https://www.clinicaltrialvanguard.com/tag/cro-consolidation/)
---
### [Vendor Integration](https://www.clinicaltrialvanguard.com/tag/vendor-integration/)
---
### [Digital Twins](https://www.clinicaltrialvanguard.com/tag/digital-twins/)
---
### [Observational Studies](https://www.clinicaltrialvanguard.com/tag/observational-studies/)
---
### [SaMD](https://www.clinicaltrialvanguard.com/tag/samd/)
---
### [pediatric clinical trials](https://www.clinicaltrialvanguard.com/tag/pediatric-clinical-trials/)
---
### [FDA Accelerated Approval](https://www.clinicaltrialvanguard.com/tag/fda-accelerated-approval/)
---
### [Surrogate Endpoints](https://www.clinicaltrialvanguard.com/tag/surrogate-endpoints/)
---
### [Rare Disease Trials](https://www.clinicaltrialvanguard.com/tag/rare-disease-trials/)
---
### [Biomarker Endpoints](https://www.clinicaltrialvanguard.com/tag/biomarker-endpoints/)
---
### [Immunology](https://www.clinicaltrialvanguard.com/tag/immunology/)
---
### [Phase 1/2 Trials](https://www.clinicaltrialvanguard.com/tag/phase-1-2-trials/)
---
### [alcohol use disorder](https://www.clinicaltrialvanguard.com/tag/alcohol-use-disorder/)
---
### [FDA drug approval](https://www.clinicaltrialvanguard.com/tag/fda-drug-approval/)
---
### [protocol complexity](https://www.clinicaltrialvanguard.com/tag/protocol-complexity/)
---
### [site activation](https://www.clinicaltrialvanguard.com/tag/site-activation/)
---
### [protocol amendments](https://www.clinicaltrialvanguard.com/tag/protocol-amendments/)
---
### [IRB timelines](https://www.clinicaltrialvanguard.com/tag/irb-timelines/)
---
### [CNS trial design](https://www.clinicaltrialvanguard.com/tag/cns-trial-design/)
---
### [IRB review burden](https://www.clinicaltrialvanguard.com/tag/irb-review-burden/)
---
### [study startup](https://www.clinicaltrialvanguard.com/tag/study-startup/)
---
### [Head and Neck Cancer](https://www.clinicaltrialvanguard.com/tag/head-and-neck-cancer/)
---
### [RWE Methodology](https://www.clinicaltrialvanguard.com/tag/rwe-methodology/)
---
### [CAR-T trials](https://www.clinicaltrialvanguard.com/tag/car-t-trials/)
---
### [post-M&A integration](https://www.clinicaltrialvanguard.com/tag/post-ma-integration/)
---
### [sponsor consolidation](https://www.clinicaltrialvanguard.com/tag/sponsor-consolidation/)
---
### [Digital Pathology](https://www.clinicaltrialvanguard.com/tag/digital-pathology/)
---
### [Oncology Trial Design](https://www.clinicaltrialvanguard.com/tag/oncology-trial-design/)
---
### [FDA AI Guidance](https://www.clinicaltrialvanguard.com/tag/fda-ai-guidance/)
---
### [FDA Inspections](https://www.clinicaltrialvanguard.com/tag/fda-inspections/)
---
### [BIMO](https://www.clinicaltrialvanguard.com/tag/bimo/)
---
### [RAS Inhibitors](https://www.clinicaltrialvanguard.com/tag/ras-inhibitors/)
---
### [Agitation](https://www.clinicaltrialvanguard.com/tag/agitation/)
---
### [Auvelity](https://www.clinicaltrialvanguard.com/tag/auvelity/)
---
### [Dextromethorphan](https://www.clinicaltrialvanguard.com/tag/dextromethorphan/)
---
### [Geriatric Psychiatry](https://www.clinicaltrialvanguard.com/tag/geriatric-psychiatry/)
---
### [Tardive Dyskinesia](https://www.clinicaltrialvanguard.com/tag/tardive-dyskinesia/)
---
### [Axsome Therapeutics](https://www.clinicaltrialvanguard.com/tag/axsome-therapeutics/)
---
### [SLE](https://www.clinicaltrialvanguard.com/tag/sle/)
---
### [Dose-Finding Trials](https://www.clinicaltrialvanguard.com/tag/dose-finding-trials/)
---
### [Phase 2 Failures](https://www.clinicaltrialvanguard.com/tag/phase-2-failures/)
---
### [FDA Endpoints](https://www.clinicaltrialvanguard.com/tag/fda-endpoints/)
---
### [Phase 3 Evidence](https://www.clinicaltrialvanguard.com/tag/phase-3-evidence/)
---
### [BRIDGE Pilot](https://www.clinicaltrialvanguard.com/tag/bridge-pilot/)
---
### [MicroRNA Therapeutics](https://www.clinicaltrialvanguard.com/tag/microrna-therapeutics/)
---
### [Cardior Pharmaceuticals](https://www.clinicaltrialvanguard.com/tag/cardior-pharmaceuticals/)
---
### [DIPG](https://www.clinicaltrialvanguard.com/tag/dipg/)
---
### [Site Operations](https://www.clinicaltrialvanguard.com/tag/site-operations/)
---
### [Sponsor Transitions](https://www.clinicaltrialvanguard.com/tag/sponsor-transitions/)
---
### [Contract Management](https://www.clinicaltrialvanguard.com/tag/contract-management/)
---
### [Remote Informed Consent](https://www.clinicaltrialvanguard.com/tag/remote-informed-consent/)
---
### [Early-Phase Trials](https://www.clinicaltrialvanguard.com/tag/early-phase-trials/)
---
### [CRO Networks](https://www.clinicaltrialvanguard.com/tag/cro-networks/)
---
### [IRB Workflows](https://www.clinicaltrialvanguard.com/tag/irb-workflows/)
---
### [Satellite Clinics](https://www.clinicaltrialvanguard.com/tag/satellite-clinics/)
---
### [CLINOPS WATCHDOG](https://www.clinicaltrialvanguard.com/tag/clinops-watchdog/)
---
### [FDA Enforcement](https://www.clinicaltrialvanguard.com/tag/fda-enforcement/)
---
### [Pharmaceutical Advertising](https://www.clinicaltrialvanguard.com/tag/pharmaceutical-advertising/)
---
### [Brukinsa](https://www.clinicaltrialvanguard.com/tag/brukinsa/)
---
### [OPDP](https://www.clinicaltrialvanguard.com/tag/opdp/)
---
### [China Clinical Trials](https://www.clinicaltrialvanguard.com/tag/china-clinical-trials/)
---
### [NMPA Regulatory Reform](https://www.clinicaltrialvanguard.com/tag/nmpa-regulatory-reform/)
---
### [IND Applications](https://www.clinicaltrialvanguard.com/tag/ind-applications/)
---
### [Theta Burst Stimulation](https://www.clinicaltrialvanguard.com/tag/theta-burst-stimulation/)
---
### [Emerging Pathogens](https://www.clinicaltrialvanguard.com/tag/emerging-pathogens/)
---
### [Surgical Site Infection](https://www.clinicaltrialvanguard.com/tag/surgical-site-infection/)
---
### [Orthopedic Trauma](https://www.clinicaltrialvanguard.com/tag/orthopedic-trauma/)
---
### [Antibiotic Prophylaxis](https://www.clinicaltrialvanguard.com/tag/antibiotic-prophylaxis/)
---
### [Infection Prevention](https://www.clinicaltrialvanguard.com/tag/infection-prevention/)
---
### [digital outcome measures](https://www.clinicaltrialvanguard.com/tag/digital-outcome-measures/)
---
### [Antimicrobial Resistance](https://www.clinicaltrialvanguard.com/tag/antimicrobial-resistance/)
---
### [Beta-Lactamase Inhibitors](https://www.clinicaltrialvanguard.com/tag/beta-lactamase-inhibitors/)
---
### [Clinical Hold](https://www.clinicaltrialvanguard.com/tag/clinical-hold/)
---
### [Prader-Willi Syndrome](https://www.clinicaltrialvanguard.com/tag/prader-willi-syndrome/)
---
### [Cardiovascular Safety](https://www.clinicaltrialvanguard.com/tag/cardiovascular-safety/)
---
### [iCBT](https://www.clinicaltrialvanguard.com/tag/icbt/)
---
### [Hispanic Mental Health](https://www.clinicaltrialvanguard.com/tag/hispanic-mental-health/)
---
### [post-market surveillance](https://www.clinicaltrialvanguard.com/tag/post-market-surveillance/)
---
### [FDA Leadership](https://www.clinicaltrialvanguard.com/tag/fda-leadership/)
---
### [CDER](https://www.clinicaltrialvanguard.com/tag/cder/)
---
### [CRISPR](https://www.clinicaltrialvanguard.com/tag/crispr/)
---
### [Intellia Therapeutics](https://www.clinicaltrialvanguard.com/tag/intellia-therapeutics/)
---
### [Clinical Trial Regulation](https://www.clinicaltrialvanguard.com/tag/clinical-trial-regulation/)
---
### [EU 2030 Targets](https://www.clinicaltrialvanguard.com/tag/eu-2030-targets/)
---
### [ACT EU](https://www.clinicaltrialvanguard.com/tag/act-eu/)
---
### [Site Management](https://www.clinicaltrialvanguard.com/tag/site-management/)
---
### [Data Quality](https://www.clinicaltrialvanguard.com/tag/data-quality/)
---
### [COPD](https://www.clinicaltrialvanguard.com/tag/copd/)
---
### [Astegolimab](https://www.clinicaltrialvanguard.com/tag/astegolimab/)
---
### [ST2/IL-33](https://www.clinicaltrialvanguard.com/tag/st2-il-33/)
---
### [Roche/Genentech](https://www.clinicaltrialvanguard.com/tag/roche-genentech/)
---
### [IL-33/ST2 Pathway](https://www.clinicaltrialvanguard.com/tag/il-33-st2-pathway/)
---
### [Drug Approvals](https://www.clinicaltrialvanguard.com/tag/drug-approvals/)
---
### [CAR-T Safety](https://www.clinicaltrialvanguard.com/tag/car-t-safety/)
---
### [data governance](https://www.clinicaltrialvanguard.com/tag/data-governance/)
---
### [Long-Term Safety](https://www.clinicaltrialvanguard.com/tag/long-term-safety/)
---
### [Adverse Events](https://www.clinicaltrialvanguard.com/tag/adverse-events/)
---
### [Clinical Trial Monitoring](https://www.clinicaltrialvanguard.com/tag/clinical-trial-monitoring/)
---
### [AstraZeneca](https://www.clinicaltrialvanguard.com/tag/astrazeneca/)
---
### [Etcamah](https://www.clinicaltrialvanguard.com/tag/etcamah/)
---
### [FDA Advisory Committee](https://www.clinicaltrialvanguard.com/tag/fda-advisory-committee/)
---
### [EMA CHMP](https://www.clinicaltrialvanguard.com/tag/ema-chmp/)
---
### [FGFR Inhibition](https://www.clinicaltrialvanguard.com/tag/fgfr-inhibition/)
---
### [Rare Tumor Trials](https://www.clinicaltrialvanguard.com/tag/rare-tumor-trials/)
---
### [coordinator burnout](https://www.clinicaltrialvanguard.com/tag/coordinator-burnout/)
---
### [patient enrollment](https://www.clinicaltrialvanguard.com/tag/patient-enrollment/)
---
### [clinical trial retention](https://www.clinicaltrialvanguard.com/tag/clinical-trial-retention/)
---
### [Clinical AI](https://www.clinicaltrialvanguard.com/tag/clinical-ai/)
---
### [Regulatory Sandbox](https://www.clinicaltrialvanguard.com/tag/regulatory-sandbox/)
---
### [Clinical Decision Support](https://www.clinicaltrialvanguard.com/tag/clinical-decision-support/)
---
### [AI Oversight](https://www.clinicaltrialvanguard.com/tag/ai-oversight/)
---
### [Clinical Trial Ops Brief](https://www.clinicaltrialvanguard.com/tag/clinical-trial-ops-brief/)
---
### [Neuroscience Trials](https://www.clinicaltrialvanguard.com/tag/neuroscience-trials/)
---
### [AI Clinical Trials](https://www.clinicaltrialvanguard.com/tag/ai-clinical-trials/)
---
### [CTMS Integration](https://www.clinicaltrialvanguard.com/tag/ctms-integration/)
---
### [Real-Time Data](https://www.clinicaltrialvanguard.com/tag/real-time-data/)
---
### [Veeva Systems](https://www.clinicaltrialvanguard.com/tag/veeva-systems/)
---
### [agentic AI](https://www.clinicaltrialvanguard.com/tag/agentic-ai/)
---
### [regulatory technology](https://www.clinicaltrialvanguard.com/tag/regulatory-technology/)
---
### [Vault](https://www.clinicaltrialvanguard.com/tag/vault/)
---
### [drug development technology](https://www.clinicaltrialvanguard.com/tag/drug-development-technology/)
---
### [life sciences software](https://www.clinicaltrialvanguard.com/tag/life-sciences-software/)
---
### [digital transformation](https://www.clinicaltrialvanguard.com/tag/digital-transformation/)
---
### [Regulatory Affairs](https://www.clinicaltrialvanguard.com/tag/regulatory-affairs/)
---
### [Platform Trials](https://www.clinicaltrialvanguard.com/tag/platform-trials/)
---
### [Neurodegenerative Disease](https://www.clinicaltrialvanguard.com/tag/neurodegenerative-disease/)
---
### [FDA Master Protocols](https://www.clinicaltrialvanguard.com/tag/fda-master-protocols/)
---
### [ADHD](https://www.clinicaltrialvanguard.com/tag/adhd/)
---
### [Pharmacotherapy](https://www.clinicaltrialvanguard.com/tag/pharmacotherapy/)
---
### [Dose Optimization](https://www.clinicaltrialvanguard.com/tag/dose-optimization/)
---
### [Tumor-Agnostic Trials](https://www.clinicaltrialvanguard.com/tag/tumor-agnostic-trials/)
---
### [CRO Oversight](https://www.clinicaltrialvanguard.com/tag/cro-oversight/)
---
### [Site Payments](https://www.clinicaltrialvanguard.com/tag/site-payments/)
---
### [Budget Reconciliation](https://www.clinicaltrialvanguard.com/tag/budget-reconciliation/)
---
### [ICON plc](https://www.clinicaltrialvanguard.com/tag/icon-plc/)
---
### [Clinical High Risk for Psychosis](https://www.clinicaltrialvanguard.com/tag/clinical-high-risk-for-psychosis/)
---
### [Tobacco Use Comorbidity](https://www.clinicaltrialvanguard.com/tag/tobacco-use-comorbidity/)
---
### [NAPLS2](https://www.clinicaltrialvanguard.com/tag/napls2/)
---
### [Diversity Action Plans](https://www.clinicaltrialvanguard.com/tag/diversity-action-plans/)
---
### [Psychosis Prevention Trials](https://www.clinicaltrialvanguard.com/tag/psychosis-prevention-trials/)
---
### [340B Drug Pricing Program](https://www.clinicaltrialvanguard.com/tag/340b-drug-pricing-program/)
---
### [Eli Lilly](https://www.clinicaltrialvanguard.com/tag/eli-lilly/)
---
### [Hospital Compliance](https://www.clinicaltrialvanguard.com/tag/hospital-compliance/)
---
### [Drug Discount Enforcement](https://www.clinicaltrialvanguard.com/tag/drug-discount-enforcement/)
---
### [DCT Strategy](https://www.clinicaltrialvanguard.com/tag/dct-strategy/)
---
### [Psychedelic Clinical Trials](https://www.clinicaltrialvanguard.com/tag/psychedelic-clinical-trials/)
---
### [Blinding Integrity](https://www.clinicaltrialvanguard.com/tag/blinding-integrity/)
---
### [FDA Regulatory Science](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-science/)
---
### [Psilocybin MDMA](https://www.clinicaltrialvanguard.com/tag/psilocybin-mdma/)
---
### [Neurological Disease](https://www.clinicaltrialvanguard.com/tag/neurological-disease/)
---
### [MAMS](https://www.clinicaltrialvanguard.com/tag/mams/)
---
### [CNS Drug Development](https://www.clinicaltrialvanguard.com/tag/cns-drug-development/)
---
### [Breast Cancer Research](https://www.clinicaltrialvanguard.com/tag/breast-cancer-research/)
---
### [CDK4/6 inhibitors](https://www.clinicaltrialvanguard.com/tag/cdk4-6-inhibitors/)
---
### [Tenecteplase](https://www.clinicaltrialvanguard.com/tag/tenecteplase/)
---
### [Endovascular Treatment](https://www.clinicaltrialvanguard.com/tag/endovascular-treatment/)
---
### [Ischemic Stroke](https://www.clinicaltrialvanguard.com/tag/ischemic-stroke/)
---
### [Phase 3 RCT](https://www.clinicaltrialvanguard.com/tag/phase-3-rct/)
---
### [Psychedelic-Assisted Therapy](https://www.clinicaltrialvanguard.com/tag/psychedelic-assisted-therapy/)
---
### [Mebufotenin](https://www.clinicaltrialvanguard.com/tag/mebufotenin/)
---
### [obesity clinical trials](https://www.clinicaltrialvanguard.com/tag/obesity-clinical-trials/)
---
### [oral small molecule](https://www.clinicaltrialvanguard.com/tag/oral-small-molecule/)
---
### [Phase 2b trial design](https://www.clinicaltrialvanguard.com/tag/phase-2b-trial-design/)
---
### [metabolic disease endpoints](https://www.clinicaltrialvanguard.com/tag/metabolic-disease-endpoints/)
---
### [MASLD](https://www.clinicaltrialvanguard.com/tag/masld/)
---
### [Survodutide](https://www.clinicaltrialvanguard.com/tag/survodutide/)
---
### [Liver Fibrosis](https://www.clinicaltrialvanguard.com/tag/liver-fibrosis/)
---
### [manufacturing compliance](https://www.clinicaltrialvanguard.com/tag/manufacturing-compliance/)
---
### [FDA 483](https://www.clinicaltrialvanguard.com/tag/fda-483/)
---
### [drug supply chain](https://www.clinicaltrialvanguard.com/tag/drug-supply-chain/)
---
### [site enrollment](https://www.clinicaltrialvanguard.com/tag/site-enrollment/)
---
### [Neuroprotection](https://www.clinicaltrialvanguard.com/tag/neuroprotection/)
---
### [Acute Ischaemic Stroke](https://www.clinicaltrialvanguard.com/tag/acute-ischaemic-stroke/)
---
### [EMPHASIS Trial](https://www.clinicaltrialvanguard.com/tag/emphasis-trial/)
---
### [ADC Therapeutics](https://www.clinicaltrialvanguard.com/tag/adc-therapeutics/)
---
### [Zynlonta](https://www.clinicaltrialvanguard.com/tag/zynlonta/)
---
### [post-authorization studies](https://www.clinicaltrialvanguard.com/tag/post-authorization-studies/)
---
### [conditional marketing authorization](https://www.clinicaltrialvanguard.com/tag/conditional-marketing-authorization/)
---
### [CRO acquisition](https://www.clinicaltrialvanguard.com/tag/cro-acquisition/)
---
### [sponsor-CRO integration](https://www.clinicaltrialvanguard.com/tag/sponsor-cro-integration/)
---
### [Pilot Scaling](https://www.clinicaltrialvanguard.com/tag/pilot-scaling/)
---
### [Clinical Data Standards](https://www.clinicaltrialvanguard.com/tag/clinical-data-standards/)
---
### [Site Burden](https://www.clinicaltrialvanguard.com/tag/site-burden/)
---
### [spatial biology](https://www.clinicaltrialvanguard.com/tag/spatial-biology/)
---
### [solid tumor trials](https://www.clinicaltrialvanguard.com/tag/solid-tumor-trials/)
---
### [antibody therapeutics](https://www.clinicaltrialvanguard.com/tag/antibody-therapeutics/)
---
### [pharmacobiology](https://www.clinicaltrialvanguard.com/tag/pharmacobiology/)
---
### [eClinical Monitoring](https://www.clinicaltrialvanguard.com/tag/eclinical-monitoring/)
---
### [Phase 1 Trial Design](https://www.clinicaltrialvanguard.com/tag/phase-1-trial-design/)
---
### [Cebranopadol](https://www.clinicaltrialvanguard.com/tag/cebranopadol/)
---
### [NOP Receptor](https://www.clinicaltrialvanguard.com/tag/nop-receptor/)
---
### [spatial transcriptomics](https://www.clinicaltrialvanguard.com/tag/spatial-transcriptomics/)
---
### [preclinical models](https://www.clinicaltrialvanguard.com/tag/preclinical-models/)
---
### [Valproate](https://www.clinicaltrialvanguard.com/tag/valproate/)
---
### [AAV Vectors](https://www.clinicaltrialvanguard.com/tag/aav-vectors/)
---
### [Clinical Trial Recruitment](https://www.clinicaltrialvanguard.com/tag/clinical-trial-recruitment/)
---
### [Enrollment Delays](https://www.clinicaltrialvanguard.com/tag/enrollment-delays/)
---
### [Sponsor-Site Operations](https://www.clinicaltrialvanguard.com/tag/sponsor-site-operations/)
---
### [Adaptive Neurostimulation](https://www.clinicaltrialvanguard.com/tag/adaptive-neurostimulation/)
---
### [Zepzelca](https://www.clinicaltrialvanguard.com/tag/zepzelca/)
---
### [Jazz Pharmaceuticals](https://www.clinicaltrialvanguard.com/tag/jazz-pharmaceuticals/)
---
### [Clinical Trial Governance](https://www.clinicaltrialvanguard.com/tag/clinical-trial-governance/)
---
### [Sponsor Accountability](https://www.clinicaltrialvanguard.com/tag/sponsor-accountability/)
---
### [Small Biotech](https://www.clinicaltrialvanguard.com/tag/small-biotech/)
---
### [Adaptive Clinical Trial Design](https://www.clinicaltrialvanguard.com/tag/adaptive-clinical-trial-design/)
---
### [CNS Device Regulation](https://www.clinicaltrialvanguard.com/tag/cns-device-regulation/)
---
### [Bayesian statistics](https://www.clinicaltrialvanguard.com/tag/bayesian-statistics/)
---
### [clinical trial startup](https://www.clinicaltrialvanguard.com/tag/clinical-trial-startup/)
---
### [FDA Medical Devices](https://www.clinicaltrialvanguard.com/tag/fda-medical-devices/)
---
### [MAMS design](https://www.clinicaltrialvanguard.com/tag/mams-design/)
---
### [progressive MS](https://www.clinicaltrialvanguard.com/tag/progressive-ms/)
---
### [adaptive enrollment](https://www.clinicaltrialvanguard.com/tag/adaptive-enrollment/)
---
### [Dementia Prevention](https://www.clinicaltrialvanguard.com/tag/dementia-prevention/)
---
### [Shingles Vaccine](https://www.clinicaltrialvanguard.com/tag/shingles-vaccine/)
---
### [Repurposed Vaccines](https://www.clinicaltrialvanguard.com/tag/repurposed-vaccines/)
---
### [PTSD](https://www.clinicaltrialvanguard.com/tag/ptsd/)
---
### [Dementia Agitation](https://www.clinicaltrialvanguard.com/tag/dementia-agitation/)
---
### [Behavioral Endpoints](https://www.clinicaltrialvanguard.com/tag/behavioral-endpoints/)
---
### [AI Validation](https://www.clinicaltrialvanguard.com/tag/ai-validation/)
---
### [FDA AI Governance](https://www.clinicaltrialvanguard.com/tag/fda-ai-governance/)
---
### [FDA CDS Guidance](https://www.clinicaltrialvanguard.com/tag/fda-cds-guidance/)
---
### [Regulatory Brief](https://www.clinicaltrialvanguard.com/tag/regulatory-brief/)
---
### [FDA Warning Letter](https://www.clinicaltrialvanguard.com/tag/fda-warning-letter/)
---
### [Medical Device Reporting](https://www.clinicaltrialvanguard.com/tag/medical-device-reporting/)
---
### [Quality Control](https://www.clinicaltrialvanguard.com/tag/quality-control/)
---
### [Sponsor Oversight](https://www.clinicaltrialvanguard.com/tag/sponsor-oversight/)
---
### [Autonomous AI Agents](https://www.clinicaltrialvanguard.com/tag/autonomous-ai-agents/)
---
### [AI Endpoints](https://www.clinicaltrialvanguard.com/tag/ai-endpoints/)
---
### [Regulated AI](https://www.clinicaltrialvanguard.com/tag/regulated-ai/)
---
### [GLP-1 trial design](https://www.clinicaltrialvanguard.com/tag/glp-1-trial-design/)
---
### [FDA obesity guidance](https://www.clinicaltrialvanguard.com/tag/fda-obesity-guidance/)
---
### [semaglutide endpoints](https://www.clinicaltrialvanguard.com/tag/semaglutide-endpoints/)
---
### [Sponsor Acquisitions](https://www.clinicaltrialvanguard.com/tag/sponsor-acquisitions/)
---
### [IND Transfer](https://www.clinicaltrialvanguard.com/tag/ind-transfer/)
---
### [Eosinophilic Esophagitis](https://www.clinicaltrialvanguard.com/tag/eosinophilic-esophagitis/)
---
### [Vonoprazan](https://www.clinicaltrialvanguard.com/tag/vonoprazan/)
---
### [Clinical Trial Enrollment](https://www.clinicaltrialvanguard.com/tag/clinical-trial-enrollment/)
---
### [Phathom Pharmaceuticals](https://www.clinicaltrialvanguard.com/tag/phathom-pharmaceuticals/)
---
### [Rare GI Disease](https://www.clinicaltrialvanguard.com/tag/rare-gi-disease/)
---
### [CRO Technology](https://www.clinicaltrialvanguard.com/tag/cro-technology/)
---
### [Site Monitoring](https://www.clinicaltrialvanguard.com/tag/site-monitoring/)
---
### [Clinical Trial Integrity](https://www.clinicaltrialvanguard.com/tag/clinical-trial-integrity/)
---
### [MHRA Inspection](https://www.clinicaltrialvanguard.com/tag/mhra-inspection/)
---
### [Mammography Screening](https://www.clinicaltrialvanguard.com/tag/mammography-screening/)
---
### [MASAI Trial](https://www.clinicaltrialvanguard.com/tag/masai-trial/)
---
### [Overdiagnosis](https://www.clinicaltrialvanguard.com/tag/overdiagnosis/)
---
### [Intermediate Endpoints](https://www.clinicaltrialvanguard.com/tag/intermediate-endpoints/)
---
### [Health AI](https://www.clinicaltrialvanguard.com/tag/health-ai/)
---
### [Adversarial Robustness](https://www.clinicaltrialvanguard.com/tag/adversarial-robustness/)
---
### [CRO Operations](https://www.clinicaltrialvanguard.com/tag/cro-operations/)
---
### [Risk-Based Monitoring](https://www.clinicaltrialvanguard.com/tag/risk-based-monitoring/)
---
### [Trauma-Focused CBT](https://www.clinicaltrialvanguard.com/tag/trauma-focused-cbt/)
---
### [Stroke Rehabilitation](https://www.clinicaltrialvanguard.com/tag/stroke-rehabilitation/)
---
### [Wearables and DHTs](https://www.clinicaltrialvanguard.com/tag/wearables-and-dhts/)
---
### [Gout Therapy](https://www.clinicaltrialvanguard.com/tag/gout-therapy/)
---
### [Psychedelic Therapy](https://www.clinicaltrialvanguard.com/tag/psychedelic-therapy/)
---
### [Tuberculosis](https://www.clinicaltrialvanguard.com/tag/tuberculosis/)
---
### [Rifampicin-Resistant TB](https://www.clinicaltrialvanguard.com/tag/rifampicin-resistant-tb/)
---
### [Adaptive Trial Methodology](https://www.clinicaltrialvanguard.com/tag/adaptive-trial-methodology/)
---
### [CNS Tumors](https://www.clinicaltrialvanguard.com/tag/cns-tumors/)
---
### [clinical trial supply chain](https://www.clinicaltrialvanguard.com/tag/clinical-trial-supply-chain/)
---
### [GMP compliance](https://www.clinicaltrialvanguard.com/tag/gmp-compliance/)
---
### [Altuviiio](https://www.clinicaltrialvanguard.com/tag/altuviiio/)
---
### [Bundibugyo virus](https://www.clinicaltrialvanguard.com/tag/bundibugyo-virus/)
---
### [PARTNERS trial](https://www.clinicaltrialvanguard.com/tag/partners-trial/)
---
### [WHO clinical trials](https://www.clinicaltrialvanguard.com/tag/who-clinical-trials/)
---
### [rare infectious disease](https://www.clinicaltrialvanguard.com/tag/rare-infectious-disease/)
---
### [Biomarker Qualification](https://www.clinicaltrialvanguard.com/tag/biomarker-qualification/)
---
### [Geroscience](https://www.clinicaltrialvanguard.com/tag/geroscience/)
---
### [Immunosenescence](https://www.clinicaltrialvanguard.com/tag/immunosenescence/)
---
### [Data Rights](https://www.clinicaltrialvanguard.com/tag/data-rights/)
---
### [Manufacturing Quality](https://www.clinicaltrialvanguard.com/tag/manufacturing-quality/)
---
### [Sanofi](https://www.clinicaltrialvanguard.com/tag/sanofi/)
---
### [AI Drug Discovery](https://www.clinicaltrialvanguard.com/tag/ai-drug-discovery/)
---
### [Insilico Medicine](https://www.clinicaltrialvanguard.com/tag/insilico-medicine/)
---
### [HIV](https://www.clinicaltrialvanguard.com/tag/hiv/)
---
### [bispecific antibody](https://www.clinicaltrialvanguard.com/tag/bispecific-antibody/)
---
### [adaptive randomization](https://www.clinicaltrialvanguard.com/tag/adaptive-randomization/)
---
### [broadly neutralizing antibody](https://www.clinicaltrialvanguard.com/tag/broadly-neutralizing-antibody/)
---
### [Fentanyl Vaccine](https://www.clinicaltrialvanguard.com/tag/fentanyl-vaccine/)
---
### [Addiction Immunotherapy](https://www.clinicaltrialvanguard.com/tag/addiction-immunotherapy/)
---
### [serotonin 1B receptor](https://www.clinicaltrialvanguard.com/tag/serotonin-1b-receptor/)
---
### [PET biomarker](https://www.clinicaltrialvanguard.com/tag/pet-biomarker/)
---
### [Pivotal Trial Design](https://www.clinicaltrialvanguard.com/tag/pivotal-trial-design/)
---
### [Recurrent Glioblastoma](https://www.clinicaltrialvanguard.com/tag/recurrent-glioblastoma/)
---
### [LAG-3](https://www.clinicaltrialvanguard.com/tag/lag-3/)
---
### [Checkpoint Inhibitors](https://www.clinicaltrialvanguard.com/tag/checkpoint-inhibitors/)
---
### [Schizophrenia](https://www.clinicaltrialvanguard.com/tag/schizophrenia/)
---
### [Functional Unblinding](https://www.clinicaltrialvanguard.com/tag/functional-unblinding/)
---
### [Multiple Sclerosis](https://www.clinicaltrialvanguard.com/tag/multiple-sclerosis/)
---
### [McDonald Criteria](https://www.clinicaltrialvanguard.com/tag/mcdonald-criteria/)
---
### [FDA Biomarker Policy](https://www.clinicaltrialvanguard.com/tag/fda-biomarker-policy/)
---
### [CMC Compliance](https://www.clinicaltrialvanguard.com/tag/cmc-compliance/)
---
### [Hepatocellular Carcinoma](https://www.clinicaltrialvanguard.com/tag/hepatocellular-carcinoma/)
---
### [Compass Pathways](https://www.clinicaltrialvanguard.com/tag/compass-pathways/)
---
### [Clinical Trial Simulation](https://www.clinicaltrialvanguard.com/tag/clinical-trial-simulation/)
---
### [Trial Optimization](https://www.clinicaltrialvanguard.com/tag/trial-optimization/)
---
### [recruitment inefficiency](https://www.clinicaltrialvanguard.com/tag/recruitment-inefficiency/)
---
### [Tourette Syndrome](https://www.clinicaltrialvanguard.com/tag/tourette-syndrome/)
---
### [Cannabinoid Therapeutics](https://www.clinicaltrialvanguard.com/tag/cannabinoid-therapeutics/)
---
### [Phase IIb Clinical Trial](https://www.clinicaltrialvanguard.com/tag/phase-iib-clinical-trial/)
---
### [NeuroThera Labs](https://www.clinicaltrialvanguard.com/tag/neurothera-labs/)
---
### [Site Recruitment](https://www.clinicaltrialvanguard.com/tag/site-recruitment/)
---
### [non-Hodgkin lymphoma](https://www.clinicaltrialvanguard.com/tag/non-hodgkin-lymphoma/)
---
### [T-cell exhaustion](https://www.clinicaltrialvanguard.com/tag/t-cell-exhaustion/)
---
### [substance use disorder](https://www.clinicaltrialvanguard.com/tag/substance-use-disorder/)
---
### [Ambient AI](https://www.clinicaltrialvanguard.com/tag/ambient-ai/)
---
### [Clinical Documentation](https://www.clinicaltrialvanguard.com/tag/clinical-documentation/)
---
### [Pharmacokinetics](https://www.clinicaltrialvanguard.com/tag/pharmacokinetics/)
---
### [B-cell Lymphoma](https://www.clinicaltrialvanguard.com/tag/b-cell-lymphoma/)
---
### [Dose Escalation](https://www.clinicaltrialvanguard.com/tag/dose-escalation/)
---
### [Phase 3 Termination](https://www.clinicaltrialvanguard.com/tag/phase-3-termination/)
---
### [Study Closeout](https://www.clinicaltrialvanguard.com/tag/study-closeout/)
---
### [Sponsor-Site Relations](https://www.clinicaltrialvanguard.com/tag/sponsor-site-relations/)
---
### [combination therapy](https://www.clinicaltrialvanguard.com/tag/combination-therapy/)
---
### [Remote Patient Monitoring](https://www.clinicaltrialvanguard.com/tag/remote-patient-monitoring/)
---
### [FDA Compliance](https://www.clinicaltrialvanguard.com/tag/fda-compliance/)
---
### [CMS 2027 Physician Fee Schedule](https://www.clinicaltrialvanguard.com/tag/cms-2027-physician-fee-schedule/)
---
### [AtaiBeckley](https://www.clinicaltrialvanguard.com/tag/ataibeckley/)
---
### [Rare Disease Clinical Trials](https://www.clinicaltrialvanguard.com/tag/rare-disease-clinical-trials/)
---
### [Osteosarcoma](https://www.clinicaltrialvanguard.com/tag/osteosarcoma/)
---
### [Phase 1/2 Oncology](https://www.clinicaltrialvanguard.com/tag/phase-1-2-oncology/)
---
### [Bioequivalence Studies](https://www.clinicaltrialvanguard.com/tag/bioequivalence-studies/)
---
### [spinal cord injury](https://www.clinicaltrialvanguard.com/tag/spinal-cord-injury/)
---
### [iPSC](https://www.clinicaltrialvanguard.com/tag/ipsc/)
---
### [regenerative medicine](https://www.clinicaltrialvanguard.com/tag/regenerative-medicine/)
---
### [Vaccine Trials](https://www.clinicaltrialvanguard.com/tag/vaccine-trials/)
---
### [Clinical Trial Methodology](https://www.clinicaltrialvanguard.com/tag/clinical-trial-methodology/)
---
### [Inherited Retinal Disease](https://www.clinicaltrialvanguard.com/tag/inherited-retinal-disease/)
---
### [eClinical Innovation](https://www.clinicaltrialvanguard.com/tag/eclinical-innovation/)
---
### [JAK inhibitors](https://www.clinicaltrialvanguard.com/tag/jak-inhibitors/)
---
### [dermatology trials](https://www.clinicaltrialvanguard.com/tag/dermatology-trials/)
---
### [povorcitinib](https://www.clinicaltrialvanguard.com/tag/povorcitinib/)
---
### [Diversity & Inclusion](https://www.clinicaltrialvanguard.com/tag/diversity-inclusion/)
---
### [Genetic Trials](https://www.clinicaltrialvanguard.com/tag/genetic-trials/)
---
### [AI Regulation](https://www.clinicaltrialvanguard.com/tag/ai-regulation/)
---
### [Medical AI Benchmarking](https://www.clinicaltrialvanguard.com/tag/medical-ai-benchmarking/)
---
### [Duchenne Muscular Dystrophy](https://www.clinicaltrialvanguard.com/tag/duchenne-muscular-dystrophy/)
---
### [Long COVID](https://www.clinicaltrialvanguard.com/tag/long-covid/)
---
### [cluster randomized trial](https://www.clinicaltrialvanguard.com/tag/cluster-randomized-trial/)
---
### [integrated care pathway](https://www.clinicaltrialvanguard.com/tag/integrated-care-pathway/)
---
### [Diversity Enrollment](https://www.clinicaltrialvanguard.com/tag/diversity-enrollment/)
---
### [Biomarker Screening](https://www.clinicaltrialvanguard.com/tag/biomarker-screening/)
---
### [maternal vaccines](https://www.clinicaltrialvanguard.com/tag/maternal-vaccines/)
---
### [Group B Streptococcus](https://www.clinicaltrialvanguard.com/tag/group-b-streptococcus/)
---
### [immunogenicity endpoints](https://www.clinicaltrialvanguard.com/tag/immunogenicity-endpoints/)
---
### [biometrics CRO](https://www.clinicaltrialvanguard.com/tag/biometrics-cro/)
---
### [eClinical stack](https://www.clinicaltrialvanguard.com/tag/eclinical-stack/)
---
### [decentralized trials](https://www.clinicaltrialvanguard.com/tag/decentralized-trials/)
---
### [NaV1.8 inhibitors](https://www.clinicaltrialvanguard.com/tag/nav1-8-inhibitors/)
---
### [acute pain](https://www.clinicaltrialvanguard.com/tag/acute-pain/)
---
### [non-opioid analgesics](https://www.clinicaltrialvanguard.com/tag/non-opioid-analgesics/)
---
### [Deescalation Trials](https://www.clinicaltrialvanguard.com/tag/deescalation-trials/)
---
### [Low-Value Care](https://www.clinicaltrialvanguard.com/tag/low-value-care/)
---
### [Deprescribing](https://www.clinicaltrialvanguard.com/tag/deprescribing/)
---
### [Behavioral Science](https://www.clinicaltrialvanguard.com/tag/behavioral-science/)
---
### [RSV Vaccine Trial](https://www.clinicaltrialvanguard.com/tag/rsv-vaccine-trial/)
---
### [Genetic Stratification](https://www.clinicaltrialvanguard.com/tag/genetic-stratification/)
---
### [Site Enrollment Strategy](https://www.clinicaltrialvanguard.com/tag/site-enrollment-strategy/)
---
### [GBA1 LRRK2](https://www.clinicaltrialvanguard.com/tag/gba1-lrrk2/)
---
### [Diversity Recruitment Operations](https://www.clinicaltrialvanguard.com/tag/diversity-recruitment-operations/)
---
### [Antipsychotic Trials](https://www.clinicaltrialvanguard.com/tag/antipsychotic-trials/)
---
### [PANSS](https://www.clinicaltrialvanguard.com/tag/panss/)
---
### [Statistical Analysis](https://www.clinicaltrialvanguard.com/tag/statistical-analysis/)
---
### [Clinical Guidelines](https://www.clinicaltrialvanguard.com/tag/clinical-guidelines/)
---
### [EDC Workflows](https://www.clinicaltrialvanguard.com/tag/edc-workflows/)
---
### [ICON Anthropic](https://www.clinicaltrialvanguard.com/tag/icon-anthropic/)
---
### [Dronabinol](https://www.clinicaltrialvanguard.com/tag/dronabinol/)
---
### [Cannabinoids](https://www.clinicaltrialvanguard.com/tag/cannabinoids/)
---
### [intracranial delivery](https://www.clinicaltrialvanguard.com/tag/intracranial-delivery/)
---
### [CNS oncology](https://www.clinicaltrialvanguard.com/tag/cns-oncology/)
---
### [FDA Regulatory Validation](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-validation/)
---
### [Cell & Gene Therapy](https://www.clinicaltrialvanguard.com/tag/cell-gene-therapy/)
---
### [Digital Mental Health](https://www.clinicaltrialvanguard.com/tag/digital-mental-health/)
---
### [AI Safety](https://www.clinicaltrialvanguard.com/tag/ai-safety/)
---
### [eTMF](https://www.clinicaltrialvanguard.com/tag/etmf/)
---
### [NMOSD](https://www.clinicaltrialvanguard.com/tag/nmosd/)
---
### [B-cell depletion](https://www.clinicaltrialvanguard.com/tag/b-cell-depletion/)
---
### [rare neurological diseases](https://www.clinicaltrialvanguard.com/tag/rare-neurological-diseases/)
---
### [CNS autoimmune trials](https://www.clinicaltrialvanguard.com/tag/cns-autoimmune-trials/)
---
### [Contract Manufacturing](https://www.clinicaltrialvanguard.com/tag/contract-manufacturing/)
---
### [Phase II Success Rates](https://www.clinicaltrialvanguard.com/tag/phase-ii-success-rates/)
---
### [ctDNA biomarkers](https://www.clinicaltrialvanguard.com/tag/ctdna-biomarkers/)
---
### [Causal Inference](https://www.clinicaltrialvanguard.com/tag/causal-inference/)
---
### [Difference-in-Differences](https://www.clinicaltrialvanguard.com/tag/difference-in-differences/)
---
### [FDA Real-World Evidence](https://www.clinicaltrialvanguard.com/tag/fda-real-world-evidence/)
---
### [AI Clinical Monitoring](https://www.clinicaltrialvanguard.com/tag/ai-clinical-monitoring/)
---
### [Phase 3 Oncology](https://www.clinicaltrialvanguard.com/tag/phase-3-oncology/)
---
### [CliniBiz](https://www.clinicaltrialvanguard.com/tag/clinibiz/)
---
### [AI Decision Support](https://www.clinicaltrialvanguard.com/tag/ai-decision-support/)
---
### [Clinical Trial Data Integrity](https://www.clinicaltrialvanguard.com/tag/clinical-trial-data-integrity/)
---
### [FDA AI/ML Guidance](https://www.clinicaltrialvanguard.com/tag/fda-ai-ml-guidance/)
---
### [AI Clinical Decision Support](https://www.clinicaltrialvanguard.com/tag/ai-clinical-decision-support/)
---
### [FDA AI Regulation](https://www.clinicaltrialvanguard.com/tag/fda-ai-regulation/)
---
### [Digital Health Technology](https://www.clinicaltrialvanguard.com/tag/digital-health-technology/)
---
### [AI Monitoring](https://www.clinicaltrialvanguard.com/tag/ai-monitoring/)
---
### [NIH funding](https://www.clinicaltrialvanguard.com/tag/nih-funding/)
---
### [K awards](https://www.clinicaltrialvanguard.com/tag/k-awards/)
---
### [clinical trial workforce](https://www.clinicaltrialvanguard.com/tag/clinical-trial-workforce/)
---
### [clinician-scientists](https://www.clinicaltrialvanguard.com/tag/clinician-scientists/)
---
### [research training pipeline](https://www.clinicaltrialvanguard.com/tag/research-training-pipeline/)
---
### [vitiligo](https://www.clinicaltrialvanguard.com/tag/vitiligo/)
---
### [upadacitinib](https://www.clinicaltrialvanguard.com/tag/upadacitinib/)
---
### [Phase 3 clinical trials](https://www.clinicaltrialvanguard.com/tag/phase-3-clinical-trials/)
---
### [FDA dermatology](https://www.clinicaltrialvanguard.com/tag/fda-dermatology/)
---
### [Contract Harmonization](https://www.clinicaltrialvanguard.com/tag/contract-harmonization/)
---
### [CTMS Migration](https://www.clinicaltrialvanguard.com/tag/ctms-migration/)
---
### [AI in Drug Development](https://www.clinicaltrialvanguard.com/tag/ai-in-drug-development/)
---
### [Antibody Discovery](https://www.clinicaltrialvanguard.com/tag/antibody-discovery/)
---
### [Computational Biology](https://www.clinicaltrialvanguard.com/tag/computational-biology/)
---
### [Cannabinoid Therapy](https://www.clinicaltrialvanguard.com/tag/cannabinoid-therapy/)
---
### [Epigenetic Clocks](https://www.clinicaltrialvanguard.com/tag/epigenetic-clocks/)
---
### [Longevity Trials](https://www.clinicaltrialvanguard.com/tag/longevity-trials/)
---
### [KRYSTAL-12](https://www.clinicaltrialvanguard.com/tag/krystal-12/)
---
### [adagrasib](https://www.clinicaltrialvanguard.com/tag/adagrasib/)
---
### [patient-reported outcomes](https://www.clinicaltrialvanguard.com/tag/patient-reported-outcomes/)
---
### [KRASG12C NSCLC](https://www.clinicaltrialvanguard.com/tag/krasg12c-nsclc/)
---
### [community-based research](https://www.clinicaltrialvanguard.com/tag/community-based-research/)
---
### [sponsor-CRO collaboration](https://www.clinicaltrialvanguard.com/tag/sponsor-cro-collaboration/)
---
### [genomic sequencing](https://www.clinicaltrialvanguard.com/tag/genomic-sequencing/)
---
### [Patient Safety Monitoring](https://www.clinicaltrialvanguard.com/tag/patient-safety-monitoring/)
---
### [Regenxbio](https://www.clinicaltrialvanguard.com/tag/regenxbio/)
---
### [Precision Psychiatry](https://www.clinicaltrialvanguard.com/tag/precision-psychiatry/)
---
### [Veeva Vault CRM](https://www.clinicaltrialvanguard.com/tag/veeva-vault-crm/)
---
### [sponsor technology](https://www.clinicaltrialvanguard.com/tag/sponsor-technology/)
---
### [eClinical systems](https://www.clinicaltrialvanguard.com/tag/eclinical-systems/)
---
### [Conversion Therapy](https://www.clinicaltrialvanguard.com/tag/conversion-therapy/)
---
### [FDA Oncology](https://www.clinicaltrialvanguard.com/tag/fda-oncology/)
---
### [cyberattack](https://www.clinicaltrialvanguard.com/tag/cyberattack/)
---
### [eDC systems](https://www.clinicaltrialvanguard.com/tag/edc-systems/)
---
### [site continuity](https://www.clinicaltrialvanguard.com/tag/site-continuity/)
---
### [CAR T cell therapy](https://www.clinicaltrialvanguard.com/tag/car-t-cell-therapy-2/)
---
### [rheumatoid arthritis](https://www.clinicaltrialvanguard.com/tag/rheumatoid-arthritis/)
---
### [genomic diagnostics](https://www.clinicaltrialvanguard.com/tag/genomic-diagnostics/)
---
### [precision medicine](https://www.clinicaltrialvanguard.com/tag/precision-medicine/)
---
### [clinical data integration](https://www.clinicaltrialvanguard.com/tag/clinical-data-integration/)
---
### [ATTR-CM](https://www.clinicaltrialvanguard.com/tag/attr-cm/)
---
### [eplontersen](https://www.clinicaltrialvanguard.com/tag/eplontersen/)
---
### [trial stratification](https://www.clinicaltrialvanguard.com/tag/trial-stratification/)
---
### [CARDIOTTRansform](https://www.clinicaltrialvanguard.com/tag/cardiottransform/)
---
### [enrollment acceleration](https://www.clinicaltrialvanguard.com/tag/enrollment-acceleration/)
---
### [investigator readiness](https://www.clinicaltrialvanguard.com/tag/investigator-readiness/)
---
### [WEE1 inhibitor](https://www.clinicaltrialvanguard.com/tag/wee1-inhibitor/)
---
### [Clinical Data Synthesis](https://www.clinicaltrialvanguard.com/tag/clinical-data-synthesis/)
---
### [DOJ Settlement](https://www.clinicaltrialvanguard.com/tag/doj-settlement/)
---
### [Opioid Marketing](https://www.clinicaltrialvanguard.com/tag/opioid-marketing/)
---
### [Regulatory Accountability](https://www.clinicaltrialvanguard.com/tag/regulatory-accountability/)
---
### [Pharma Compliance](https://www.clinicaltrialvanguard.com/tag/pharma-compliance/)
---
### [Clinical AI Governance](https://www.clinicaltrialvanguard.com/tag/clinical-ai-governance/)
---
### [AI Accountability](https://www.clinicaltrialvanguard.com/tag/ai-accountability/)
---
### [New Approach Methodologies](https://www.clinicaltrialvanguard.com/tag/new-approach-methodologies/)
---
### [Triple-Negative Breast Cancer](https://www.clinicaltrialvanguard.com/tag/triple-negative-breast-cancer/)
---
### [Atezolizumab](https://www.clinicaltrialvanguard.com/tag/atezolizumab/)
---
### [Phase I Operations](https://www.clinicaltrialvanguard.com/tag/phase-i-operations/)
---
### [CNS Autoimmune Disease](https://www.clinicaltrialvanguard.com/tag/cns-autoimmune-disease/)
---
### [FDA Rare Disease](https://www.clinicaltrialvanguard.com/tag/fda-rare-disease/)
---
### [Neuromyelitis Optica](https://www.clinicaltrialvanguard.com/tag/neuromyelitis-optica/)
---
### [siRNA therapeutics](https://www.clinicaltrialvanguard.com/tag/sirna-therapeutics/)
---
### [lipoprotein(a)](https://www.clinicaltrialvanguard.com/tag/lipoproteina/)
---
### [cardiovascular clinical trials](https://www.clinicaltrialvanguard.com/tag/cardiovascular-clinical-trials/)
---
### [RNA interference](https://www.clinicaltrialvanguard.com/tag/rna-interference/)
---
### [Addiction Neuroscience](https://www.clinicaltrialvanguard.com/tag/addiction-neuroscience/)
---
### [Novel Targets](https://www.clinicaltrialvanguard.com/tag/novel-targets/)
---
### [data monitoring committees](https://www.clinicaltrialvanguard.com/tag/data-monitoring-committees/)
---
### [early termination](https://www.clinicaltrialvanguard.com/tag/early-termination/)
---
### [Solute Carrier Transporters](https://www.clinicaltrialvanguard.com/tag/solute-carrier-transporters/)
---
### [FDA Drug-Drug Interactions](https://www.clinicaltrialvanguard.com/tag/fda-drug-drug-interactions/)
---
### [Vendor Management](https://www.clinicaltrialvanguard.com/tag/vendor-management/)
---
### [Phase 2a](https://www.clinicaltrialvanguard.com/tag/phase-2a/)
---
### [Proteomic Aging Clocks](https://www.clinicaltrialvanguard.com/tag/proteomic-aging-clocks/)
---
### [remote monitoring](https://www.clinicaltrialvanguard.com/tag/remote-monitoring/)
---
### [MS trials](https://www.clinicaltrialvanguard.com/tag/ms-trials/)
---
### [eCOA validation](https://www.clinicaltrialvanguard.com/tag/ecoa-validation/)
---
### [Generative AI](https://www.clinicaltrialvanguard.com/tag/generative-ai/)
---
### [clinical data management](https://www.clinicaltrialvanguard.com/tag/clinical-data-management/)
---
### [FDA regulatory framework](https://www.clinicaltrialvanguard.com/tag/fda-regulatory-framework/)
---
### [Psychedelic Drugs](https://www.clinicaltrialvanguard.com/tag/psychedelic-drugs/)
---
### [CNS Therapies](https://www.clinicaltrialvanguard.com/tag/cns-therapies/)
---
### [mitapivat](https://www.clinicaltrialvanguard.com/tag/mitapivat/)
---
### [thalassaemia](https://www.clinicaltrialvanguard.com/tag/thalassaemia/)
---
### [ENERGIZE-T](https://www.clinicaltrialvanguard.com/tag/energize-t/)
---
### [pyruvate kinase activator](https://www.clinicaltrialvanguard.com/tag/pyruvate-kinase-activator/)
---
### [transfusion-dependent](https://www.clinicaltrialvanguard.com/tag/transfusion-dependent/)
---
### [sponsor operations](https://www.clinicaltrialvanguard.com/tag/sponsor-operations/)
---
### [Biohaven](https://www.clinicaltrialvanguard.com/tag/biohaven/)
---
### [BHV-7000](https://www.clinicaltrialvanguard.com/tag/bhv-7000/)
---
### [Preclinical Safety](https://www.clinicaltrialvanguard.com/tag/preclinical-safety/)
---
### [ibogaine](https://www.clinicaltrialvanguard.com/tag/ibogaine/)
---
### [provider training](https://www.clinicaltrialvanguard.com/tag/provider-training/)
---
### [data standardization](https://www.clinicaltrialvanguard.com/tag/data-standardization/)
---
### [drug regulation](https://www.clinicaltrialvanguard.com/tag/drug-regulation/)
---
### [veterans health](https://www.clinicaltrialvanguard.com/tag/veterans-health/)
---
### [REMS](https://www.clinicaltrialvanguard.com/tag/rems/)
---
### [underrepresented populations](https://www.clinicaltrialvanguard.com/tag/underrepresented-populations/)
---
### [digital cognitive assessment](https://www.clinicaltrialvanguard.com/tag/digital-cognitive-assessment/)
---
### [remote screening](https://www.clinicaltrialvanguard.com/tag/remote-screening/)
---
### [anti-amyloid therapy](https://www.clinicaltrialvanguard.com/tag/anti-amyloid-therapy/)
---
### [HIV functional cure](https://www.clinicaltrialvanguard.com/tag/hiv-functional-cure/)
---
### [broadly neutralizing antibodies](https://www.clinicaltrialvanguard.com/tag/broadly-neutralizing-antibodies/)
---
### [analytical treatment interruption](https://www.clinicaltrialvanguard.com/tag/analytical-treatment-interruption/)
---
### [phase 2 clinical trial](https://www.clinicaltrialvanguard.com/tag/phase-2-clinical-trial/)
---
### [bNAb resistance](https://www.clinicaltrialvanguard.com/tag/bnab-resistance/)
---
### [Bipolar Disorder](https://www.clinicaltrialvanguard.com/tag/bipolar-disorder/)
---
### [Mood Stabilizers](https://www.clinicaltrialvanguard.com/tag/mood-stabilizers/)
---
### [liquid biopsy](https://www.clinicaltrialvanguard.com/tag/liquid-biopsy/)
---
### [EEG biomarkers](https://www.clinicaltrialvanguard.com/tag/eeg-biomarkers/)
---
### [site network operations](https://www.clinicaltrialvanguard.com/tag/site-network-operations/)
---
### [eCOA standardization](https://www.clinicaltrialvanguard.com/tag/ecoa-standardization/)
---
### [brain medicine](https://www.clinicaltrialvanguard.com/tag/brain-medicine/)
---
## Podcasts
### [The Clinical Trial Vanguard](https://www.clinicaltrialvanguard.com/podcasts/the-clinical-trial-vanguard/)
---
### [CliniCast](https://www.clinicaltrialvanguard.com/podcasts/clinicast/)
---