Accelerating Progress in Pharma and Biotech: A Closer Look at Surrogate Endpoints and Promising Therapies

Pharma and BioTech Daily

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA's accelerated approval pathway, established in 1992, has allowed over 200 new drugs to come to market based on surrogate endpoints. While many products have later received full FDA approval, recent withdrawals like Pfizer's Oxbryta and Takeda's Exkivity have raised concerns about the risks of this pathway. Despite some high-profile failures, the program has a strong track record, with over half of accelerated approvals converting to full approval. The pathway has been used primarily for cancer treatments but is now expanding to other disease areas like neurological and rare diseases. Recent failures of confirmatory trials for drugs like Sarepta's Elevidys have prompted experts to call for tighter timelines and better biomarkers for surrogate endpoints in order to improve the program. The future of the accelerated approval pathway is under scrutiny, with questions arising about how often it should be used and what areas it should focus on next.AbbVie's Tavapadon showed improvement in motor and daily living complications in a Phase III trial, following the failure of another key cerevel asset, Emraclidine. Relmada Therapeutics is considering strategic alternatives, including a potential sale, after halting two Phase III trials for a major depressive disorder drug. Other news includes BioAge discontinuing a Phase II obesity study, Merck's promising anti-ROR1 ADC data for lymphoma treatment, and Novo and Catalent's $16.5 billion deal receiving EU approval. Additionally, Vertex and Beam report advances in SCD cell and gene therapies, while evidence grows for the potential of GLP-1s in Alzheimer's disease.Recent studies have shown that GLP-1 receptor agonists, commonly used for weight loss and diabetes, may have potential in treating Alzheimer's disease. Phase III results from Novo Nordisk are expected next year. Additionally, amylin analogs are emerging as a potential alternative or complement to GLP-1 receptor agonists for weight loss with a cleaner tolerability profile. Other news includes a weight loss victory claimed by Lilly over Novo in a head-to-head study, FDA action alerts, and promising cancer therapies from companies like AstraZeneca and Merus. The FDA is also investigating the safety of Bluebird's gene therapy Skysona for hematologic malignancies. Applied Therapeutics recently faced a crash after FDA rejection of a rare disease drug. Overall, the potential of GLP-1s in Alzheimer's disease and the emergence of amylin analogs in weight loss are significant developments in the biopharma industry.