What was your biggest professional highlight of the past 12 months?
It’s been a really exciting and productive 12 months for our team across our entire hematology portfolio at Regeneron. Most recently, in April, we presented and simultaneously published our pivotal results for cemdisiran, a subcutaneous siRNA therapy, which, if approved, could become a first-in-class medicine treatment of generalized myasthenia gravis (gMG).
We also continue to advance our bispecific blood cancer programs: Lynozyfic (BCMAxCD3) in late line multiple myeloma and Ordspono (CD20xCD3 bispecific) in late line follicular lymphoma and DLBCL. Both are being investigated in earlier lines of disease. The data are on display at this year’s ASCO with two orals – one for Lynozyfic in a plasma cell disorder and one for Ordspono in first-line DLBCL.
Further, our team continues to drive progress across a large Phase 3 thrombosis program, including two novel Factor XI antibodies with positive Phase 2 results for the prevention of blood clotting simultaneously presented at ACC and published in The Lancet in November.
Where is the biopharma industry making real progress that isn’t getting enough attention?
Much of the attention goes to novel modalities, innovative therapies, and improvements over the standard of care – but there is little focus on how these breakthroughs are generated: through clinical trial innovation and modernization. This includes protocol streamlining and designing trials around the patient, all with the goal of getting better medicines to patients faster.
There is a lot of work being done to ground trial design decisions in data and operational insights, and our teams aim to streamline protocols, reduce non-essential procedures and focus efforts on what truly matters to generate meaningful evidence. We engage investigators, sites, patients and advocacy organizations early and often to understand what drives, or hinders, participation and execution.
Several organizations are also working to improve trial design and share best practices, including the Center for Clinical Trials (CCT) and Clinical and Translational Science Institute (CTSI) at Tufts, which collaborate with clinical investigators and sponsors (including Regeneron) from as early as grant proposal onward to ensure application of best practices and knowledge sharing, and TransCelerate BioPharma, a nonprofit collaborative focused on simplifying and accelerating drug development.
What is the biggest bottleneck slowing progress in drug development – and how do we fix it?
The biggest bottleneck slowing progress in drug development is the competition for, and successful recruitment of, patients for participation in clinical trials, while also ensuring participants reflect real-world populations.
To fix this critical issue, our approach is about removing unnecessary burden – not lowering standards. We work to optimize clinical efficiencies and reduce the burden of participation. This means designing trials that balance scientific rigor with practical considerations so that high-quality science can move forward more efficiently and responsibly. The future of clinical trials is one in which participation is easier, protocols are streamlined, and studies are brought to patients rather than patients to studies.
How can the pharmaceutical industry become more resilient in an increasingly uncertain world?
I think the approach we’ve taken to development at Regeneron is one model for how the industry can become more resilient in an increasingly uncertain world. Our approach to scientific discovery focuses on understanding disease biology and remaining adaptable/flexible in choosing the right platforms, technologies or modalities that best fit the need and allows us to bring innovation to patients.
Our hematology programs illustrate this approach well. Regeneron has a legacy in antibodies with nearly three decades of expertise, and that focus now extends to gene therapies, cell medicines and other novel modalities to best target and treat a given blood cancer or other disorder.
What will look completely different about drug development in 10 years?
In 10 years, we will be realizing and capitalizing on the promise of AI across all areas of drug development, including operations and big data. I expect AI to play an increasingly important role across drug development, from target identification and hypothesis generation to clinical trial design, patient recruitment, and data analysis.
I also expect that gene therapy approaches will be a standard part of drug development for both rare disease indications and common conditions. We’re experiencing the beginning stages of this transition in real-time as the regulatory environment evolves to match the pace of technological innovation and manufacturing capacity improves.
Do you think your younger self would be surprised to see the career you’ve had?
Definitely, yes. My younger self envisioned a career as a practicing physician. I’m very grateful I had the opportunity to practice medicine in the early part of my career, as it gives me an important perspective as we design our clinical trials and balance the need for scientific rigor with the burden on site staff and, importantly, the participants themselves. When the opportunity arose to pivot into drug development, I knew I could apply what I’d learned in practice to benefit more patients than I’d ever be able to reach in the clinic.
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