BBP-418 Demonstrates Potential to Be Disease Modifying Therapy, Restoring Cardiac and Disease Markers to Unaffected Levels
Globe Newswire•05/10/2026•07:30 ET
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Key Highlights
- ➤100% of BBP-418-treated participants with elevated troponin normalized by Month 12
- ➤54% on BBP-418 had stable or improved LVEF versus 25% on placebo
- ➤BBP-418 increased glycosylated αDG to asymptomatic carrier levels by Month 3
- ➤BBP-418 met all primary and secondary FORTIFY endpoints at 12-month interim
- ➤FDA Priority Review targets November 27, 2026 action date
Expert Statements
Volker Straub, M.D., Ph.D., Newcastle University, UK
“Cardiac involvement is a major driver of morbidity and mortality in LGMD2I/R9, which is why it remains one of the greatest concerns for people living with the condition, families and treating physicians. Historically, we have been able to monitor the heart and manage complications, but we have not been able to address the underlying cause of the disease.”
Volker Straub, M.D., Ph.D., Newcastle University, UK
“What stands out in this analysis is how consistent it is: high-sensitivity troponin I fell and, in those who entered the study with elevated levels, it normalized; ejection fraction stabilized; and this happened alongside walking farther and breathing better. It’s encouraging to see that the therapeutic approach seems to work across the whole muscular system, including the heart.”
- In exploratory analyses at Month 12, among individuals with LGMD2I/R9 (FKRP-related) with elevated HS troponin I at baseline, 100% treated with BBP-418 returned to the normal range by Month 12 versus 40% in the placebo arm (p=0.0248); HS troponin I, a blood marker of heart muscle injury, declined more with BBP-418 than placebo (LS mean difference -17.8 ng/L; 95% CI: -35.1 to -0.5; p=0.0443)
- 54% of BBP-418-treated individuals had stable or improved LVEF at Month 12 versus 25% in the placebo arm (p=0.0262)
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