BridgeBio Pharma reported positive topline results from the company’s phase 3 pivotal study of its small molecule oral therapy, BBP-418, in individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
According to the company, all primary and secondary interim analysis endpoints in the FORTIFY study were successfully achieved with well-tolerated safety profile consistent with prior studies.
Specifically, the primary interim analysis endpoint, glycosylated αDG, significantly increased by 1.8x change from baseline at 3 months, and improvements were sustained at 12 months in BBP-418 treated individuals versus placebo. Additionally, there was a verage reduction in serum CK, a marker of muscle damage, of 82% change from baseline and statistically significant difference versus placebo in BBP-418 treated individuals at 12 months.
"I’m very excited to see that treatment with BBP-418 was associated with clinically meaningful improvements in motor and pulmonary function, along with robust restoration of αDG glycosylation. This is such an important result for individuals living with LGMD2I/R9, which is a progressive muscular dystrophy. The resulting weakness often leads loss of ambulation, need for respiratory support, and need for heart failure medications,” said Katherine Mathews, MD, Professor of Pediatrics and Neurology at the University of Iowa’s Roy J. and Lucille A. Carver College of Medicine. “To date, there has been no specific treatment. These results bring enormous hope that BBP-418 might change the disease course.”
Analyses of the full FORTIFY interim analysis data are ongoing, and BridgeBio plans to present detailed results at a future medical meeting. BBP-418 has previously received orphan drug, fast track, and rare pediatric disease designations from the FDA. If BBP-418 is approved, BridgeBio may qualify for a priority review voucher. The company intends to engage the FDA later this year to discuss these data and plans for submission of an NDA for BBP-418 in the first half of 2026.