BridgeBio Pharma Inc. (NASDAQ: BBIO) has announced the dosing of the first patient in the Phase 1/2 ADventure clinical study of BBP-831, an experimental adeno-associated virus 5 gene therapy for classic congenital adrenal hyperplasia (CAH) treatment. With over 75,000 cases reported in the US and the EU, CAH is among the most common genetic illnesses.
BridgeBio doses first CAH patient
CEO Eric David said, “Dosing the first patient in our CAH trial is a landmark milestone and we are grateful for the support from the medical and patient communities who helped us reach this moment. For more than 50 years people living with CAH have had the same limited standard of care – lifelong daily steroid replacement treatment.”
By producing its aldosterone and cortisol, the company’s investigational gene therapy gives patients a possible single-dose treatment to restore steroid and hormone balance in the body. David said they are pleased to progress the study and other gene therapy studies to improve patients’ lives. This is the second gene therapy study the company has started within four months.
BridgeBio Gene Therapy’s Chief Medical Officer Adam Shaywitz said, “Adults, children and families affected by CAH experience the daily burden of the disease and often, unfortunately, the side effects and morbidities associated with the current treatment regimens. As an endocrinologist, it’s incredibly exciting to reimagine a new approach to treating this disease.”
Adrenas Therapeutics part of BridgeBio’s portfolio
Notably, BridgeBio Gene Therapy’s portfolio includes Adrenas Therapeutics, its affiliate dedicated to developing BBP-631 for CAH.
The Phase 1/2 open-label trial will assess the pharmacodynamic activity, safety, and tolerability of BBP-631, BridgeBio’s AAV5 gene therapy for people with classic CAH. Each patient will get a single intravenous infusion during the study’s preliminary dose-finding phase. The study’s key goals are safety and changes in endogenous cortisol levels from baseline, that BBP-631 has a distinctive ability to restore. Researchers will also measure the change in steroid biomarkers for hydroxyprogesterone levels and androstenedione levels from baseline. Preclinical data shows that the approach offers persistent and efficient functional 21-hydroxylase enzyme to the adrenal gland.