In this interview, I spoke with Jonathan Rigby, CEO of Sernova, and Francis Shannon, SVP of Clinical Development and Regulatory Affairs. We discussed Sernova’s groundbreaking advancements in cell therapies for type one diabetes. Their recent data demonstrates the long-term survival and function of islets in their cell pouch over five years, a development that could significantly alter disease management. Our conversation explored the implications of this achievement, the technological innovations behind the cell pouch, and the future potential of Sernova’s therapies.
Moe Alsumidaie: How does Sernova’s islet survival data change diabetes therapy?
Jonathan Rigby: The perception that type one diabetes lacks unmet needs is misleading. Despite insulin, CGMs and pumps, better treatment is crucial. As a type one diabetic, I joined Sernova to help develop a functional cure. Our recent data offers hope for long-term therapy, potentially allowing insulin independence. This could significantly change disease management and has applications beyond diabetes, like hypothyroidism and hemophilia A. For instance, data presented by Dr. Witkowski in Madrid showed abundant, functional surviving islets in the cell pouch for over five years with no necrosis or fibrosis. This is unprecedented and offers a future where patients might not need multiple
daily insulin injections. The implications extend beyond diabetes, as our platform could address other diseases caused by dysfunctional cell types, offering a new paradigm in disease management.
Moe Alsumidaie: What innovations in the cell pouch support islet survival?
Francis Shannon: The innovation lies in the cell pouch’s simple design. It uses an open mesh structure that integrates with the patient’s tissue without triggering a foreign body response. This allows for well-vascularized tissue chambers, providing an optimal environment for cell transplantation. We’ve shown its effectiveness in multiple applications, including thyroid and hemophilia programs. Unlike other devices that might cause encapsulation and rejection, the body accepts the cell pouch, functioning as if it were a natural bio simulated organ. This approach supports islet survival and opens the door for other cell-based therapies. By avoiding complex designs, we’ve created a conducive environment for cells, enhancing their longevity and function.
Moe Alsumidaie: What regulatory pathway did you take for the cell pouch?
Francis Shannon: The FDA recognizes our therapy as a combination product, and we anticipate a biologics license application (BLA) for market authorization. This classification acknowledges the therapy’s dual nature, combining biological and device components. The regulatory pathway ensures the therapy meets all safety and efficacy standards before reaching the market. By working closely with regulatory bodies, we ensure our innovative approach is thoroughly vetted, paving the way for a successful launch. This pathway validates our technology and sets a precedent for future combination therapies in the field.
Moe Alsumidaie: What are the next milestones in your phase 1/2 study?
Jonathan Rigby: We’re about two-thirds through our current phase 1/2 study focusing on optimizing the immune suppression regime. Our long-term goal is to transition to using induced pluripotent stem cell-derived islet-like clusters, providing an unlimited supply of consistent quality cells. This will be the next phase of our clinical trials. The ongoing study has shown promising results, with six out of six patients in Cohort A achieving insulin independence. As we refine our approach, we aim to bridge trials with these stem cell-derived clusters, which could revolutionize treatment by providing a sustainable and scalable solution. This transition marks a significant step towards a more accessible and effective therapy for type one diabetes.
Moe Alsumidaie: How does patient input shape your trial design and goals?
Jonathan Rigby: Patient input is crucial. Understanding how the disease affects patients and their families helps us articulate the need for better treatments. It also motivates us and informs our discussions with potential investors. Personal experiences, like my own with type one diabetes, emphasizes the importance of developing a functional cure. For example, the story of a patient who achieved insulin independence for over four years highlights the profound impact our therapy can have. Such testimonials drive home the real-world benefits and guide our development priorities. By staying close to patients, we ensure our therapies address their most pressing needs, enhancing their quality of life.
Moe Alsumidaie: What challenges have emerged during the cell pouch’s development?
Francis Shannon: Determining the optimal dose and distribution of islets was challenging, but we’ve solved that. We’ve also optimized the immune suppression regimen, which differs from traditional hepatic transplants. We’ve made significant progress in reducing adverse events and enhancing tolerability. For instance, we’ve learned that the immune suppression required for subcutaneous placement in the cell pouch differs from that needed for hepatic transplants. This insight has allowed us to tailor our approach, improving patient outcomes and minimizing side effects. By addressing these challenges head-on, we’ve strengthened our clinical program and set the stage for future success.
Moe Alsumidaie: What is the growth potential for this medical product?
Francis Shannon: The cell pouch broadly applies to glandular disorders and other conditions requiring cell or tissue supplementation. Its full containment and retrievability offer significant advantages, especially as we explore gene editing technologies. This ensures safety and quality control, even with potential recalls. Beyond diabetes, we’re looking at conditions like hypothyroidism and hemophilia, where the cell pouch could provide a stable and effective delivery system for therapeutic cells. The ability to retrieve the pouch if needed adds an extra layer of safety, making it an attractive option for a wide range of applications. This versatility positions the cell pouch as a transformative tool in regenerative medicine.
Moe Alsumidaie: How are you managing adverse events to ensure trial safety?
Francis Shannon: We have a robust system involving a data and safety monitoring board comprised of top experts. They provide guidance and oversight, ensuring we address any unexpected adverse events promptly and effectively. This board plays a critical role in maintaining the trial’s integrity, offering an external perspective that helps us navigate complex safety issues. By closely monitoring adverse events and adjusting our protocols as needed, we ensure that patient safety remains our top priority. This proactive approach not only safeguards participants but also strengthens the credibility of our clinical data, supporting our path to regulatory approval.
Moe Alsumidaie is Chief Editor of The Clinical Trial Vanguard. Moe holds decades of experience in the clinical trials industry. Moe also serves as Head of Research at CliniBiz and Chief Data Scientist at Annex Clinical Corporation.
