BridgeBio Cuts Gene Therapy Budget After Disappointing Early-Phase Data

BridgeBio Pharma has decided to significantly reduce its investment in gene therapy, a strategy shift influenced by the outcomes of a recent phase 1/2 clinical trial. CEO Neil Kumar, Ph.D., explained that the results were not yet transformative, prompting the biotech firm to redirect its resources towards other drug candidates and treatment methods. This decision impacts BBP-631, BridgeBio's gene therapy aimed at treating congenital adrenal hyperplasia (CAH).

Earlier this year, at the 2024 J.P. Morgan Healthcare Conference, Kumar laid out the criteria for continuing with BBP-631. The therapy is intended to provide a functional copy of a gene responsible for an enzyme that enables cortisol production. However, Kumar emphasized that the therapy would only progress if it proved to be more effective, not merely more convenient, than existing treatments.

In the phase 1/2 trial, BBP-631 did not meet these stringent criteria. The goal was to achieve cortisol levels of 10 μg/dL or higher. Although the trial did see cortisol levels reach up to 11 μg/dL, the data showed a maximum change from baseline levels of 4.7 μg/dL and 6.6 μg/dL at the two highest doses tested. Normal cortisol levels can fluctuate throughout the day, typically ranging from 5 μg/dL to 25 μg/dL when measured in the morning.

The current standard treatment for CAH involves glucocorticoids, which replace deficient cortisol and suppress a specific hormone. Neurocrine Biosciences' near-approval CRF1 antagonist can reduce the glucocorticoid dosage but did not increase cortisol levels in its phase 2 trial. Despite generating evidence of sustained transgene activity, BridgeBio found the clinical data insufficient to justify further investment in BBP-631 for CAH. Consequently, the company is now seeking partnerships to advance the development of BBP-631 and next-generation gene therapies for this condition.

This discontinuation is part of a larger strategic reassessment of BridgeBio's investment in gene therapy. According to Brian Stephenson, Ph.D., the company's Chief Financial Officer, BridgeBio will cut its gene therapy budget by over $50 million. The company will focus on gene therapies only for targets that cannot be treated through other means. Last year, BridgeBio allocated $458 million to research and development.

Another gene therapy in BridgeBio's clinical pipeline targets Canavan disease, a much rarer condition compared to CAH. The therapy is currently in phase 1/2 of development. Stephenson affirmed that BridgeBio would collaborate closely with the FDA and the Canavan disease community to expedite bringing this therapy to patients. Preliminary results from the trial indicated improvements in functional outcomes such as head control and the ability to sit upright among treated patients.

BridgeBio's pivot away from gene therapy, except for highly prioritized targets, underscores the challenges and financial risks involved in developing these advanced therapeutics. As the company recalibrates its focus, it aims to maximize the impact of its R&D investments by pursuing the most promising treatment avenues.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!