BridgeBio Cuts Gene Therapy Budget After Adrenal Gland Medicine Data Review

BridgeBio Pharma has announced plans to cut its gene therapy research budget by over $50 million following disappointing early-stage study results for one of its experimental therapies. The company's BBP-631 therapy, designed to treat congenital adrenal hyperplasia (CAH), did not meet the expected benchmarks for further investment. The results, disclosed by BridgeBio, showed that BBP-631 led to increased production of cortisol, a crucial hormone for the body's stress response, in patients with CAH. Although the therapy did not cause any serious side effects, the data were not considered transformative enough for continued funding, according to CEO Neil Kumar. Consequently, BridgeBio is now seeking a partner to assist in the development of BBP-631.

Despite this setback, BridgeBio remains committed to the field of gene therapy. The company is advancing another experimental treatment aimed at a neuromuscular disease known as Canavan disease. Chief Financial Officer Brian Stephenson emphasized the significant unmet needs that gene therapies could address and expressed eagerness to collaborate with the FDA and the Canavan community to expedite the development of their therapy.

Moving forward, BridgeBio plans to concentrate its gene therapy efforts on priority targets that cannot be treated through other means. This strategic shift will result in a more focused pipeline, emphasizing a handful of late-stage assets. Among these, acoramidis, a treatment for transthyretin amyloidosis with cardiomyopathy, stands out as it is currently under regulatory review in the United States. Additionally, BridgeBio has several medicines in Phase 3 trials for various conditions, including a rare calcium disorder, a type of muscular dystrophy, and a bone growth disorder.

The results for BBP-631 were gathered from the second half of a Phase 1/2 study involving adults with classic CAH. The study demonstrated that higher doses of BBP-631 led to increased endogenous cortisol production in all participants. Researchers also observed significant and lasting increases in the product and substrate of the enzyme responsible for cortisol synthesis, indicating that the therapy had successfully delivered its genetic payload. BBP-631 employs a benign adeno-associated virus to carry a functional copy of the gene encoding the enzyme to the adrenal gland. BridgeBio initially hoped this would enable the body to produce cortisol autonomously, potentially reducing or eliminating the need for steroid treatments currently used by CAH patients.

By refocusing its gene therapy efforts and prioritizing late-stage assets, BridgeBio aims to optimize its resources and bring effective treatments to market more efficiently. The company's strategic realignment underscores its commitment to addressing unmet medical needs through innovative therapies, even as it navigates the challenges inherent in developing groundbreaking treatments.

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!