NEW YORK – BridgeBio Pharma said Tuesday that it is discontinuing development of its investigational gene therapy BBP-631 for congenital adrenal hyperplasia (CAH).
In new data from the Phase I/II open-label ADventure study examining the safety, tolerability, and pharmacodynamic activity of BBP-631 in adults with classic CAH, BridgeBio said the therapy led to increased endogenous cortisol production in all patients who received a high dose of the treatment and was well tolerated with no treatment-related serious adverse events.
However, BridgeBio CFO Brian Stephenson said in a statement that "the results of the trial did not meet the threshold to warrant additional capital investment at this time."
BBP-631, an adeno-associated vector-based gene therapy, is designed to deliver a functional copy of the 21-hydroxylase gene that is deficient in CAH. This gene is needed to make the key hormones cortisol and aldosterone, and people who lack these hormones cannot mount the typical physiological responses to stressors, which can be life-threatening.
Palo Alto, California-based BridgeBio will also decrease its gene therapy budget by more than $50 million. The company added that it is seeking partnership opportunities for future development of BBP-631 or other gene therapies for CAH.
The company announced separately that its investigational gene therapy BBP-812 to treat Canavan disease, a potentially fatal neurodevelopmental disease, has received regenerative medicine advanced therapy designation from the US Food and Drug Administration. BBP-812 already has orphan drug, rare pediatric disease, and fast-track designations from the FDA and orphan drug designation from the European Medicines Agency.