BridgeBio halts CAH gene therapy after Phase 1/2 shows cortisol production but not transformative; cuts budget $50M+

BridgeBio Pharma, Inc.

Phase 1/2 ADventure trial of BBP-631 in classic CAH: increased endogenous cortisol in all higher-dose patients (max change 6.6 µg/dL post-ACTH); well tolerated with no treatment-related SAEs.
BBP-631 achieved durable transgene activity: 55-fold avg increase in 11-deoxycortisol at highest doses; ≥50% reduction in 17-OHP in majority of patients.
Company discontinues BBP-631 development for CAH, citing data not meeting threshold for additional capital investment; budget cut >$50M.
BridgeBio seeking partnership opportunities for BBP-631 or next-gen CAH gene therapies; est. >75,000 CAH cases in US+EU.
CFO states reserving gene therapy for priority targets like Canavan disease; will work with FDA to accelerate Canavan therapy.

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