PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.
“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. Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their body’s hormone and steroid balance by making their own cortisol and aldosterone,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. “This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients’ lives.”
“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,” added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy. Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapy’s portfolio.
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 (IV) 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 (17-OHP) levels and androstenedione (A4) levels will also be measured. Preclinical proof-of-concept data have shown the approach provides efficient and persistent delivery of functional 21-hydroxylase (21-OH) enzyme to the adrenal gland.
“We are honored to be the first site to administer gene therapy in a patient with CAH as it is a potential game-changing treatment option that targets the disease at its source,” said Kyriakie Sarafoglou, M.D., associate professor and director of the Center for Congenital Adrenal Hyperplasia at the University of Minnesota. “We are eager to see whether gene therapy can restore endogenous cortisol production, and also look forward to exploring its effect on the physiological mechanisms that regulate the hypothalamic-pituitary-adrenal axis including circadian and ultradian hormonal profiles.”
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.
