BridgeBio Cuts Gene Therapy Budget by Over $50 Million Following Disappointing Early-Phase Data

Budget Reduction:
BridgeBio Pharma has significantly reduced its gene therapy budget by over $50 million due to early-phase data from the BBP-631 trial for congenital adrenal hyperplasia (CAH) not meeting the "transformational" target.

Trial Results:
The Phase 1/2 ADventure study showed that all patients in higher dose groups of BBP-631 had an increase in endogenous cortisol production, a first for CAH patients, but the data were not deemed groundbreaking enough to warrant further investment.

Safety and Tolerability:
The gene therapy was well-tolerated with no serious adverse events linked to the treatment reported.

Future Plans:
BridgeBio will discontinue the development of BBP-631 for CAH and is actively seeking partnership opportunities to support future development of gene therapies for CAH, which affects over 75,000 individuals in the United States and European Union.

Strategic Focus:
The company is prioritizing gene therapy for high-need targets that cannot be addressed through other means, such as Canavan disease, and aims to work closely with the FDA and the Canavan community to expedite therapy development.