BridgeBio Pharma, Inc. (BBIO) on Monday reported positive topline results from its Phase 3 FORTIFY study evaluating BBP-418 in individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a progressive muscular dystrophy.
The study met all primary and secondary interim analysis endpoints, with BBP-418 demonstrating a well-tolerated safety profile consistent with prior studies, the company said.
The primary interim analysis endpoint, glycosylated DG, increased by 1.8 times from baseline at three months, with improvements sustained through 12 months versus placebo. Treatment with BBP-418 also resulted in an average 82% reduction from baseline in serum creatine kinase (CK), a marker of muscle damage, showing a statistically significant difference versus placebo at 12 months.
Participants treated with BBP-418 also showed statistically significant and clinically meaningful improvements in key functional measures at 12 months. Ambulatory function, measured by the 100-meter timed test (100MTT), improved by 0.14 meters per second from baseline and 0.27 meters per second versus placebo, while pulmonary function, assessed by forced vital capacity (FVC), rose about 3% from baseline and 5% relative to placebo.
BridgeBio said it intends to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in the first half of 2026.
BridgeBio shares gained more than 8% in pre-market trading. The stock closed Friday at $54.26, up 0.84%.
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