The BioPharm Brief: Rare Disease, Trial Setbacks, and Oral Obesity

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today, we’re looking at new FDA plans for rare disease drug development, a setback for BioNTech’s cancer immunotherapy, and Viking Therapeutics’ next step in oral obesity treatment.

The FDA is launching its Rare Disease Framework Initiative to make drug development and regulatory review more consistent for therapies targeting rare conditions. The agency plans to gather public input beginning October 28 and examine evidence requirements, reviewer coordination, patient input, and manufacturing quality review. No regulatory changes have been proposed yet, but the initiative could affect how sponsors plan development for small patient populations.

BioNTech’s BNT113 missed its progression-free survival goal in the Phase II/III AHEAD-MERIT trial. The mRNA cancer immunotherapy, combined with pembrolizumab, did not significantly improve progression-free survival compared with pembrolizumab alone in patients with HPV16-positive head and neck cancer. The trial will continue to assess overall survival, its second primary endpoint, which could still determine whether the study ultimately meets its objectives.

Viking Therapeutics has started Phase III testing of oral VK2735 in two SUNRISE trials involving adults with obesity, including people with type 2 diabetes. The once-daily tablet targets both GLP-1 and GIP receptors, and each study will evaluate weight change over 68 weeks. Earlier Phase II results showed average weight loss of up to 12.2% after 13 weeks. The phase 3 program will test whether those results hold up over a longer treatment period.

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Key takeaways

• FDA targets rare disease development. A new initiative will examine how to make drug development and regulatory review more consistent for rare disease therapies.
  
• BioNTech’s BNT113 misses its primary endpoint. The combination with pembrolizumab failed to significantly improve progression-free survival in HPV16-positive head and neck cancer. Overall survival data are still pending.
  
• Viking advances its oral obesity drug. Phase III SUNRISE trials are underway for VK2735, a once-daily GLP-1/GIP receptor agonist, following promising Phase II weight-loss results.FYB206 advances to FDA review without a comparative phase 3 efficacy trial.