BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study

Core Insights - The FORTIFY Phase 3 study of BBP-418 has successfully achieved all primary and secondary interim analysis endpoints with a well-tolerated safety profile consistent with prior studies [1] - BBP-418 treatment resulted in a significant increase in glycosylated αDG by 1.8 times from baseline at 3 months, with sustained improvements at 12 months [1][4] - The average reduction in serum CK, a marker of muscle damage, was 82% from baseline at 12 months in BBP-418 treated individuals, showing a statistically significant difference versus placebo [1][4] - The company plans to file a New Drug Application (NDA) with the FDA in the first half of 2026 [1][5] Study Results - The FORTIFY study is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating BBP-418 for LGMD2I/R9 [3] - Key results at 12 months include: - Ambulatory function (100MTT): Increase in velocity of 0.14 m/s from baseline and 0.27 m/s versus placebo (p<0.0001) [3][4] - Pulmonary function (FVC): Increase of approximately 3% predicted volume from baseline and a difference of about 5% versus placebo (p=0.0071) [3][10] Safety and Regulatory Designations - BBP-418 was well-tolerated with no new or unexpected safety findings observed [10] - The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA, as well as Orphan Drug Designation from the EMA [6] Disease Background - LGMD2I/R9 is a monogenic autosomal recessive disease caused by mutations in the FKRP gene, leading to impaired glycosylation of αDG [7] - Clinical manifestations include skeletal myopathy, pulmonary muscle involvement, and cardiac issues, with significant morbidity in affected individuals [7] Company Overview - BridgeBio Pharma, Inc. is focused on discovering and delivering transformative medicines for genetic diseases, with a commitment to applying advances in genetic medicine [8]