BridgeBio doses first patient in Phase 1/2 trial of BBP-631

BridgeBio Pharma announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus 5 gene therapy for the treatment of classic congenital adrenal hyperplasia. CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union. The Phase 1/2 open-label study is designed to evaluate the safety, tolerability and pharmacodynamic activity of the company's AAV5 gene therapy, BBP-631, in adults with classic CAH. In the initial dose-finding phase of the study, each subject will receive a single intravenous infusion. The primary outcomes of the study are safety, as well as change from baseline in endogenous cortisol levels which BBP-631 has the unique potential to restore. Change from baseline in steroid biomarkers for hydroxyprogesterone levels and androstenedione levels will also be measured. Preclinical proof-of-concept data have shown the approach provides efficient and persistent delivery of functional 21-hydroxylase enzyme to the adrenal gland. BridgeBio's investigational AAV5 gene therapy for CAH is one of the Company's 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and cancers. Initial Phase 1/2 data readouts of the Company's AAV5 gene therapy for CAH and the Company's AAV9 gene therapy for Canavan disease are expected in the second half of 2022.