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The Superplenary at Advanced Therapies Week 2024 addressed the challenges, concerns and major wins of the industry from the perspective of those who are involved as the key stakeholders. The Fireside chat featured Anthony Davies, Laurie Adami, Nicole Verdun, Pascal Touchan and Michael E. Myers. The session ignited crucial conversations facilitated by the agenda of the week.
Advanced Therapies Week 2024 commenced with a vibrant start at the Fontainebleau Miami Beach Hotel. Participants of the Women in Advanced Therapies Pre-day and Investor Pre-day engaged in the morning sessions preceding the eagerly awaited opening networking event. As attendees trickled in, drinks and nibbles in-hand, the evening began with a sunny view overlooking Miami’s South Beach.
Phacilitate’s Kim Barnes introduced the Superplenary, setting the stage for the conversations that would follow. The discussion was led by Anthony Davies, CEO of Dark Horse Consulting, featuring a fireside chat with CAR-T patient Laurie Adami, FDA’s Director of the Office of Therapeutic Products Nicole Verdun, Atara Biotherapeutics CEO Pascal Touchon, and H.C. Wainwright & Co.‘s Vice Chairman and Head of M&A and Strategic Advisory Services, Michael E. Myers. Watch this space (above) to watch the full Superplenary session.
Advanced Therapies Week not only brought together like-minded individuals but also served as a forum for collating ideas, perspectives, and future outlooks for advanced therapies. It was evident that the primary focus should be on expediting the delivery of potential treatments to patients in need and enhancing the developmental processes of these treatments.
The opening fireside chat with Pascal Touchon highlighted the crucial importance of cultivating a robust network of partners in the cell and gene therapy domain. The discussion emphasized the need to forge strong relationships with Contract Development and Manufacturing Organizations (CDMOs), recognizing their pivotal role in a field where demonstrating efficacy, quality, and safety is paramount for regulatory approval. This is particularly crucial in the context of rare diseases, where timelines for approvals are pressing. Many within the industry share a common motivation—to deliver optimal treatments for the benefit of patients.
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Next, Laurie Adami’s touching story illustrated the critical importance of delivering treatments to patients promptly. Diagnosed with stage 4 incurable follicular non-Hodgkin lymphoma in 2006, Adami’s journey led her to explore clinical trials, where she discovered R-Bendamustine, a drug approved in Europe at the time. Adami’s participation in a Phase II clinical trial of CAR-T cell therapy (Yescarta) at UCLA marked a significant moment. As the first patient to enroll at UCLA, her oncologist, Dr Sven de Vos, estimated she carried over 8 pounds of tumor burden at the start of treatment. Faced with grade 4 cytokine release syndrome and neurotoxicity, leading to a medically induced coma, Adami’s perseverance prevailed. On day 30, scans revealed her lymphoma was finally in complete remission.
Following Adami’s fireside chat, Dr Nicole Verdun shared her professional journey, transitioning from a hematologist-oncologist to the Director of the Office of Therapeutic Products at the FDA. She expressed a commitment to fostering development in the field and exploring innovative trial approaches. Recognizing the challenges of confirmatory trials, especially for rare diseases, she advocated for regulatory flexibility across all pathways, irrespective of the company’s size. She highlighted the FDA’s dynamic mechanisms, notably the establishment of six super offices, each focused on specific areas. Additionally, a revised talent recruitment process was implemented to improve efficiency and communication within the FDA.
Emphasizing the need for fairness in the application process, Verdun called for better guidance to assist companies in navigating touchpoints for successful submissions. She highlighted recent changes in the FDA’s mechanisms, aiming to enhance accessibility for companies. As the industry expands, Verdun stressed the growing imperative to respond to regulatory standards, facilitating quicker delivery of treatments to patients through improved process efficiency. Verdun also acknowledged the necessity for effective conversations with the FDA in the rapidly evolving landscape, recognizing the agency’s role in managing exponential growth.
Michael E. Myers later delved into the financial landscape of the cell and gene therapy industry, underscoring the crucial need to deploy capital with a favorable rate of return. Emphasizing the industry’s imperative to collaborate closely with regulatory agencies, Myers particularly highlighted the challenges associated with defining comparability, addressing limited long-term data, and navigating pricing and cost issues.
He also reiterated the industry’s advantageous position to actively present credit across the capital structure, especially in the context of strategic deals. With the introduction of new modalities, Myers stressed the necessity for implementing updated policies and the hands-on involvement of staff. Discussing the industry’s evolutionary journey, Myers metaphorically described the completion of a ‘circle’ as seeds sown in the past now flourish in the present and near future, with various operational factors coming to fruition.
Myers also highlighted the importance of leveraging CMCs (Chemistry, Manufacturing, and Controls) and maintaining a robust investment cycle, particularly concerning the intricate interplay of price and cost dynamics. Addressing concerns about the Inflation Reduction Act (IRA) and its impact on approval rates, Myers emphasized the industry’s challenge in affording curative therapy. He urged the need for a milestone-driven, value-based pricing model to create a risk pool, emphasizing the industry’s ongoing efforts to stay relevant and generate effective solutions. Myers advocated for a shift toward value-based pricing, moving away from traditional asset-based funding approaches.
The panel concluded by expressing anticipation for new data, transformative products gaining regulatory approval, and increased awareness of cell therapy among patients. They emphasized the importance of educating the public about clinical trials and acknowledged the FDA’s enhanced efficiency in the application and approval processes.
The Superplenary offered insightful perspectives, establishing an aspirational atmosphere that eased Advanced Therapies Week into full swing. As Phacilitate’s 20th-anniversary Mardi Gras celebration proceeded, conversations continued to buzz with the dynamic shifts in regulatory authorities accommodating innovative technologies. While regulatory approval remains a significant hurdle, the imminent approval of more therapies raises concerns about patient access and affordability, particularly for those with rare diseases. The industry’s persistent efforts to educate stakeholders and implement effective changes were duly acknowledged, signifying substantial progress.
The future of cell and gene therapy, as unveiled during Advanced Therapies Week, radiates with promise and innovation. The discussions and developments persist beyond the event, marking a continuous commitment to advancing the field.
The Key Takeaways from the Superplenary at Advanced Therapies Week 2024:
Collaboration and Networking:
The session emphasized the importance of collaboration and building strong networks in the cell and gene therapy space. Establishing robust relationships with partners, particularly Contract Development and Manufacturing Organizations (CDMOs), was highlighted as essential for success.
Patient-Centric Focus:
Laurie Adami’s personal journey highlighted the transformative impact of advanced therapies on patients. The patient-centric approach was emphasized, with a shared commitment among industry professionals to deliver the best possible treatments for those in need.
Regulatory Flexibility:
Dr Nicole Verdun discussed the evolving regulatory landscape, emphasizing the need for flexibility to accommodate the unique challenges posed by advanced therapies. The FDA’s efforts to streamline processes, implement super offices (currently, six), and enhance communication aim to facilitate quicker approvals.
Financial Considerations:
Michael Myers delved into the financial aspect of the industry, stressing the need for strategic capital deployment with a good rate of return. The industry’s continuous growth requires proactive adaptation to new modalities and policies.
Challenges in Rare Diseases:
The session acknowledged the urgency of delivering treatments for rare diseases. However, challenges related to pricing, access, and affordability for patients with rare diseases were recognized as areas that still need to be addressed.
Education and Awareness:
The industry’s commitment to educating stakeholders, from patients to regulatory agencies, emerged as a recurring theme. Enhancing public awareness about clinical trials and the efficacy of advanced therapies was deemed crucial for continued progress.
Optimism for the Future:
Despite challenges, the panel expressed optimism for the future of cell and gene therapy. Anticipation for new data, transformative products gaining regulatory approval, and increased awareness among patients were highlighted as positive indicators for the industry’s growth.
In Summary
The Superplenary highlighted the collaborative and patient-focused essence of the industry, showcasing ongoing initiatives to navigate regulatory challenges, address financial considerations, and mitigate challenges in delivering advanced therapies, especially for rare diseases. The future of cell and gene therapy appears promising, evident in the innovation showcased throughout Advanced Therapies Week—be it on the show floor, in the roundtables, workshops, or the innovation zone, to name a few. Importantly, it doesn’t stop here – the conversation continues and development persists.
Future Prospects
It is evident that challenges persist at every stage of the industry, yet the progress made over the years of this young therapeutic space persists to throttle on. It is often said that the future of cell and gene therapy is bright—but so is the present. We have witnessed the industry’s efforts, dedication, innovation, and resilience thrive and evolve. While we engage in candid conversations to propel the field forward, we also celebrate milestones that enable the delivery of life-changing treatments to patients with unmet needs.
There is still a long way to go, from patient advocacy, reimbursement, pricing and regulatory challenges, but the journey so far has been crucial in the monumental development that has occurred. From ideas to vials, clinical trials to approvals, innovation to execution, cell and gene therapy is making is mark. While the landscape expands and evolves for some, many of us eagerly observe and learn. For those with unmet medical needs, we persist in bridging the gap between obstacles and the delivery of essential treatments
If you did attend Advanced Therapies Week 2024, what are you celebrating and what are you pinning to the bullseye of the advanced therapies field? Take a moment to view the Top 20 Events Over 20 Years of Advanced Therapies to recap.
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