FX.co ★ BridgeBio Pharma Stops Development Of BBP-631 For Congenital Adrenal Hyperplasia

BridgeBio Pharma Inc. (BBIO) has announced that it will cease development of BBP-631 for Congenital Adrenal Hyperplasia (CAH). Instead, the company is actively seeking partnership opportunities to support either the future development of BBP-631 or the advancement of next-generation gene therapies for CAH—a widespread genetic disease affecting over 75,000 individuals in the United States and European Union, which still has significant unmet medical needs.

The decision follows results from the Phase 1/2 ADventure open-label study, which evaluated BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy, as a treatment for CAH.

BridgeBio Pharma noted that the trial results did not justify additional capital investment at this time. Consequently, the company will be reducing its gene therapy budget by over $50 million.

Key findings from the study included increased endogenous cortisol production in all patients at higher doses. BBP-631 was well tolerated, with only mild to moderate treatment-emergent adverse events (TEAEs) reported, and no treatment-related serious adverse events observed.