The BioPharm Brief: Reviews, Deals, and Delays

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Here’s what we’re talking about today.

GSK’s Jemperli is headed for an FDA priority review in a new rectal cancer indication. The agency accepted a supplemental Biologics License Application for dostarlimab in previously untreated stage II and III mismatch repair-deficient, or dMMR, microsatellite instability-high, or MSI-H, locally advanced rectal cancer. The application is supported by Phase 2 AZUR-1 data showing sustained clinical complete responses with Jemperli monotherapy. If approved, GSK says Jemperli could become the first immunotherapy option that allows some patients to avoid or delay chemotherapy, radiation, and surgery. The FDA has set a February 2027 PDUFA date, although the application is also eligible for the National Priority Voucher program, which could potentially accelerate the decision.

In obesity, Genentech is making a major bet on a treatment designed to address one of the challenges associated with current weight-loss therapies. Hanmi Pharmaceutical has licensed global rights, excluding South Korea, to HM17321 in a deal valued at up to $2.3 billion. The investigational drug is a selective CRFR2 agonist based on a urocortin-2 analog, rather than a GLP-1-based mechanism, and is designed to promote weight loss while preserving lean mass. Hanmi will complete Phase 1, with Genentech taking over from phase 2 onward. The deal includes $190 million upfront, with the remainder tied to development, regulatory, and commercial milestones, plus tiered royalties.

And Capricor Therapeutics is getting more time to make its case for deramiocel in Duchenne muscular dystrophy. The FDA extended the PDUFA date for the cell therapy from August 22 to November 22 after Capricor submitted an amendment containing 24-month open-label extension data from the HOPE-3 study, along with additional analyses. The amendment follows a July FDA advisory committee meeting in which reviewers questioned Capricor’s interpretation of the trial’s efficacy results. The company is seeking approval under a refined indication focused on upper limb function, an important measure of independence for patients with progressive DMD.

That’s The BioPharm Brief for today. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways:

• GSK’s Jemperli is headed for priority review in dMMR/MSI-H locally advanced rectal cancer.
• Genentech is betting up to $2.3 billion on Hanmi’s next-generation obesity drug HM17321.
• Capricor’s deramiocel review has been extended to November as the FDA considers additional DMD data.