BridgeBio Pharma (BBIO) Receives FDA Fast Track Designation for Investigational Gene Therapy for Congenital Adrenal Hyperplasia

May 14, 2021 7:30 AM EDT

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BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to BBP-631, an investigational adeno-associated virus 5 (AAV5) gene therapy designed for the treatment of congenital adrenal hyperplasia (CAH).

Fast Track designation, granted by the FDA, is designed to facilitate the development and to expedite the review of new therapies hoping to treat or prevent serious conditions and fill an unmet medical need. Additionally, BBP-631 was granted Rare Pediatric Disease Designation by the FDA and has received Orphan Drug Designation by the FDA and European Medicines Agency (EMA).

“The standard-of-care for CAH patients has not changed significantly over the last 50 years, and a gene therapy offers for the first time the possibility that patients may be able to make their own cortisol and aldosterone, at the right times and in the right amounts. The FDA’s Fast Track designation reinforces the urgency to address the unmet needs of patients with CAH as quickly and safely as possible. We are grateful to have received Fast Track along with other key designations granted by the FDA and the EMA,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy, which is focused on developing gene therapy treatment options for patients in need. “We are eager to initiate our first-in-human Phase 1/2 study. The Investigational New Drug application has been cleared by the FDA and site activation for the study is ongoing, and is expected to begin in the coming months.”

CAH is one of the most prevalent genetic diseases with more than 75,000 cases estimated in the United States and Europe. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol and aldosterone production. This lack of production causes patients with CAH to be unable to form physiological responses to illnesses and stressors, which can be life-threatening, especially for children. BBP-631 is designed to provide a functional copy of the 21-hydroxylase-encoding gene to help patients produce their own cortisol and aldosterone.

Additionally, BridgeBio Gene Therapy presented preclinical data from its CAH program on May 13, 2021 at the American Society of Gene & Cell Therapy (ASGCT) annual meeting, being held virtually May 11-14, 2021. The presentation covered three key Investigational New Drug (IND)-enabling studies that informed the anticipated upcoming clinical trial for BBP-631. The IND application has been cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in late 2021 or early 2022. At ASGCT, BridgeBio Gene Therapy today will also be presenting preclinical data from its program designed for the treatment of Canavan disease, which is an extremely rare genetic disease starting in infancy with an incidence of approximately one in 100,000 births worldwide.

“ASGCT is an important opportunity to share the robust preclinical data package underlying our programs. We hope that by rapidly advancing our investigational therapies into the clinic, we will be one step closer to providing CAH and Canavan patients with potential therapeutic relief,” said Clayton Beard, senior vice president of research and development at BridgeBio Gene Therapy.

ASGCT Oral Presentations:

Intravenous AAV5 Gene Therapy with Human CYP21A1 Corrects Phenotypic Deficiencies of the 21-Hydroxylase Knockout Mouse Model and Demonstrates Durability and Safety in Non-Human Primates and MicePresenter: Rachel Eclov, Ph.D., associate director of preclinical gene therapy at BridgeBio Gene TherapyPresentation date/time: Thursday May 13, 2021 at 6:45 – 7:00 p.m. ET

  • The preclinical data presented shows dose-dependent efficacy, dose-dependent biodistribution and no safety concerns in the shared studies.
  • Results from all three studies shared in the presentation support the efficacy and safety for the upcoming Phase 1/2 trial in classic CAH.

Safety Evaluation of IV-Administered BBP-812, an AAV9-Based Gene Therapy for the Treatment of Canavan Disease, in Mice and Juvenile Cynomolgus MacaquesPresenter: David Scott, director of nonclinical pharmacology and toxicology at BridgeBio Gene TherapyPresentation date/time: Friday, May 14, 2021 at 2:00 – 2:15 p.m. ET

  • The preclinical findings demonstrate safety through IV administration, which will provide superior biodistribution to deep brain regions and avoid potentially invasive brain surgery.
  • The toxicology study shows safety and facilitates a transition into IND and the clinic to begin testing in patients with Canavan disease.

For more information on the upcoming clinical trial in CAH, please visit clinicaltrials.gov (Identifier: NCT04783181).



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