BridgeBio Halts Gene Therapy for Adrenal Disorder

BridgeBio announced that it will halt the development of its gene therapy aimed at treating a group of genetic disorders affecting the adrenal glands and disrupting cortisol production. The California-based biotechnology firm made this decision due to Phase 1/2 data that did not meet their expectations for being "transformational," as stated by CEO Neil Kumar. The gene therapy in question, named BBP-631, was being tested by BridgeBio’s subsidiary, Adrenas Therapeutics, in adults with classic congenital adrenal hyperplasia (CAH).

This strategic shift will result in a $50 million reduction in BridgeBio’s gene therapy budget. The decision reflects broader challenges in the gene therapy field, including high-profile negative results, diminishing investor interest, and waning enthusiasm from major pharmaceutical companies. BridgeBio had partnered with biomanufacturer National Resilience for the production of BBP-631 and other gene therapies in their development pipeline.

Despite the setback, BridgeBio is "actively seeking partnership opportunities" for the gene therapy or its potential successors for treating the genetic disease, as mentioned in their Tuesday statement. CAH affects approximately 75,000 individuals in the United States and the European Union.

A spokesperson for BridgeBio did not immediately respond to inquiries regarding the potential impact of these changes on the workforce of the company or its subsidiary. The trial investigators will continue to monitor the eight patients who participated in the study, which commenced in 2021. Importantly, there have been no reports of serious adverse events related to the treatment.

Currently, much of BridgeBio's focus is on preparing for a potential approval of its ATTR-CM drug acoramidis, which faces an FDA decision deadline on November 29. In recent months, BridgeBio has divested certain parts of its pipeline, transferring oncology-related assets to a new spinout and rare disease assets to another venture named GondolaBio.

Adrenas Therapeutics expressed their disappointment in a September 10 post on their website but acknowledged that their efforts over the past six years have led to a better understanding of CAH and offered greater hope for those affected by the condition. The gene therapy aimed to deliver a functional copy of the 21-hydroxylase gene through a one-time IV infusion, with the hope that it would enable CAH patients to produce their own cortisol and aldosterone, potentially reducing or eliminating the need for daily doses of glucocorticoids and mineralocorticoids.

Moving forward, BridgeBio plans to reserve gene therapy for priority targets that cannot be treated by other means, as highlighted by CFO Brian Stephenson. The company will now focus on a different gene therapy for Canavan disease, a rare inherited disorder causing progressive destruction of nerve cells and neurodevelopmental delays. Stephenson expressed eagerness to collaborate closely with the FDA and the Canavan community to bring the treatment to patients. BridgeBio’s subsidiary, Aspa Therapeutics, is testing this therapy, codenamed BBP-812, in a Phase 1/2 open-label study expected to conclude in October 2026, according to a federal trials database.

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