BridgeBio Pharma (NASDAQ:BBIO) shares surged 15% on Monday after the company announced highly positive Phase 3 results for its small-molecule therapy BBP-418, targeting limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
The company said that its FORTIFY Phase 3 trial met all primary and secondary interim analysis endpoints, with the treatment demonstrating a favorable safety profile. The primary endpoint showed glycosylated αDG levels increased 1.8x from baseline at 3 months, while serum CK—a marker of muscle damage—fell by 82%.
Patients treated with BBP-418 also experienced statistically significant improvements at 12 months in key functional measures, including walking speed and lung function. Walking speed improved by 0.14 m/s from baseline and 0.27 m/s compared with placebo, while lung function rose by approximately 3% in predicted volume.
“We reiterate our OP on top-pick BBIO on the heels of BBP-418’s interim Ph3 data that is a blowout best-case outcome,” said Evercore ISI analyst Cory Kasimov, who raised his price target on BridgeBio to $90 from $75 while maintaining an Outperform rating.
BridgeBio plans to meet with the FDA later this year and submit a New Drug Application in the first half of 2026. The therapy has already secured Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the agency.
LGMD2I/R9 is a progressive muscular dystrophy that often leads to loss of mobility and the need for respiratory support. At present, there are no approved treatments specifically targeting this condition.