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The Speak Foundation shares great news
The Speak Foundation - Empowering the Voice of the Patient
Community Research Update: October 2025
October 25th, 2025 - The Speak Foundation (TSF) acknowledges BridgeBio’s announcement today regarding breakthrough results from the FORTIFY Phase 3 study of BBP-418, a potential treatment for individuals living with Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMD2I/R9). These results hold great promise for those who live with this form of LGMD. All primary and secondary interim analysis endpoints were successfully achieved, with a well-tolerated safety profile consistent with prior BridgeBio studies.
Key Study Findings
- Glycosylated αDG (the primary biomarker) increased by 1.8x from baseline at 3 months (p<0.0001), and improvements were sustained at 12 months (p<0.0001) in individuals treated with BBP-418 compared to placebo.
- Average reduction in serum CK, a marker of muscle damage, was 82% from baseline (p<0.0001) in the BBP-418 group at 12 months.
- Clinically meaningful improvements at 12 months were seen in:
• Ambulatory function (100-Meter Timed Test): Increase of 0.14 m/s from baseline and 0.27 m/s versus placebo (p<0.0001).
• Pulmonary function (FVC): Increase of ~3% predicted volume from baseline and ~5% versus placebo (p=0.0071).
BridgeBio plans to file a New Drug Application (NDA) for FDA approval in the first half of 2026.
Expert Insight
“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,” said Dr. Katherine Mathews, Professor of Pediatrics and Neurology at the University of Iowa. “To date, there has been no specific treatment for LGMD2I/R9. These results bring enormous hope that BBP-418 might change the disease course.”
About the FORTIFY Study
FORTIFY is a randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of BBP-418, an investigational oral therapy in development for the treatment of LGMD2I/R9.
Results showed sustained restoration of glycosylated αDG, significant reductions in CK, and meaningful improvements in both motor and pulmonary function. BBP-418 was well-tolerated, with no new or unexpected safety findings. These results support a favorable risk-benefit profile for BBP-418.
LGMD DAY ON THE HILL
At the Speak Foundation, we empower voices through education, advocacy, and support for Limb Girdle Muscular Dystrophy. Our innovative programs for our community elevate the voice of those who live with the disease daily. Our organization is led and run by those who have LGMD.