BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)

– If successful, BridgeBio’s investigational gene therapy BBP-631 would be the first therapy for CAH to restore the body’s hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone

-Initial Phase 1/2 data readout anticipated in the second half of 2022

-BridgeBio’s gene therapy portfolio also includes a clinical stage program for Canavan disease and preclinical programs for classic galactosemia, TMC1 hearing loss, tuberous sclerosis types 1 and 2, cystinuria and a genetic dilated cardiomyopathy

Excerpt from the Press Release:

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.

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