BridgeBio Completes Enrollment for Phase 3 Study of BBP-418 in LGMD2I/R9

BridgeBio Pharma, Inc. has announced the completion of enrollment for its Phase 3 registrational study, FORTIFY, on Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). This milestone aligns with the 10th annual LGMD Awareness Day, a global event held on September 30th to raise awareness for individuals living with the condition. The FORTIFY study is a randomized, double-blind, placebo-controlled Phase 3 trial that evaluates the safety and efficacy of BBP-418, an investigational oral therapy aimed at treating LGMD2I/R9.

LGMD2I/R9 is a genetic disorder caused by mutations in the fukutin-related protein (FKRP) gene. These mutations impair the glycosylation of alpha-dystroglycan (αDG), a crucial protein that stabilizes muscle cells. The disease typically manifests as a progressive skeletal myopathy, initially affecting the lower limbs before progressing to the upper limbs. Advanced stages often involve respiratory muscle and cardiac muscle issues. Patients with a homozygous L276I genotype usually show symptoms in late childhood and may face severe complications like loss of independent ambulation, assisted ventilation, and cardiomyopathy in adulthood. Those with other FKRP genotypes may experience an even more severe clinical course, often leading to rapid loss of mobility and frequent cardiac involvement by age 20, and respiratory failure by age 30.

The FORTIFY study includes an interim analysis planned at 12 months, focusing on assessing glycosylated αDG as a surrogate endpoint. This could potentially support an Accelerated Approval for BBP-418 in the U.S. The primary endpoint of the study, to be evaluated at 36 months, is the North Star Assessment for limb-girdle type muscular dystrophies, aimed at providing confirmatory clinical data to support the efficacy of BBP-418. Topline data from the interim analysis is expected in 2025.

Kelly Brazzo, CEO of CureLGMD2i Foundation, highlighted the significance of this study for patients suffering from LGMD2I/R9, a condition for which there are currently no approved treatments. The rapid enrollment in the clinical trial brings hope for potential future treatments. Dr. Douglas Sproule, Chief Medical Officer of ML Bio Solutions, a BridgeBio subsidiary, emphasized that completing the enrollment is a critical milestone. He expressed optimism about pursuing Accelerated Approval in the U.S. for BBP-418 based on the interim analysis's potential biomarker surrogate endpoint. If successful, BBP-418 could become the first disease-modifying therapy approved for LGMD2I/R9 in the U.S.

BBP-418 has already received several designations from the FDA, including Orphan Drug, Fast Track, and Rare Pediatric Disease Designations. It has also received Orphan Drug Designation from the European Medicines Agency (EMA). With the FDA's Rare Pediatric Designation, BridgeBio may qualify for a Priority Review Voucher if BBP-418 gains approval.

BridgeBio Pharma, Inc. is a commercial-stage biopharmaceutical company focused on developing transformative medicines for genetic diseases. Founded in 2015, the company has a diverse pipeline ranging from early research to advanced clinical trials. BridgeBio aims to leverage advances in genetic medicine to address unmet medical needs as swiftly as possible.