BBP-418 - (FORTIFY)

October 5, 2026: BridgeBio Pharma (NASDAQ: BBIO) reported new exploratory cardiac data from the 12-month interim analysis of the Phase 3 FORTIFY trial of oral BBP-418 in limb-girdle muscular dystrophy type 2I/R9 (FKRP-related), presented at the 31st World Muscle Society Congress.

In participants with elevated high-sensitivity troponin I at baseline, 100% of BBP-418-treated patients normalized by Month 12 versus 40% on placebo (p=0.0248); HS troponin I declined more with BBP-418 (LS mean difference -17.8 ng/L; 95% CI: -35.1 to -0.5; p=0.0443). At Month 12, 54% of BBP-418-treated participants had stable or improved left ventricular ejection fraction versus 25% on placebo (p=0.0262). Cardiac markers will continue to be monitored in ongoing FORTIFY.

Separate WMS 2026 data showed BBP-418 increased mean glycosylated alpha-dystroglycan by Month 3 to levels seen in asymptomatic heterozygous FKRP carriers and sustained this through Month 12; the release also states mean glycosylated alpha-dystroglycan rose above carrier levels by Month 12. The company said Phase 3 FORTIFY had previously met all primary and secondary endpoints at the pre-specified 12-month interim analysis.

BBP-418 is under FDA Priority Review with a PDUFA target action date of November 27, 2026. BridgeBio also plans pediatric LGMD2I/R9 studies in 1H 2027 and future studies in LGMD2M/R13 and LGMD2U/R20.