BridgeBio Pharma held a conference call on Monday, October 27, 2025 at 8:00 AM ET to announce the results of the FORTIFY Phase 3 study of its oral therapy BBP‑418 for limb‑girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The call was hosted by CEO Neil Kumar and other senior executives.
The interim analysis met all primary and secondary endpoints. Glycosylated α‑DG increased 1.8‑fold at 3 months and remained elevated at 12 months (p<0.0001). Serum creatine kinase fell 82 % from baseline (p<0.0001). All key clinical endpoints showed statistically significant, clinically meaningful improvements at 12 months, and the safety profile was well tolerated.
BBP‑418 is the first‑in‑class oral therapy for LGMD2I/R9. The FORTIFY study has Orphan Drug, Fast Track, and Rare Pediatric Disease designations and may qualify for a Priority Review Voucher. The oral route and first‑in‑class status differentiate it from existing investigational or off‑label options.
The positive interim results bring the program closer to potential regulatory approval and support BridgeBio’s strategy of delivering transformative medicines for rare genetic diseases. The company will continue to discuss the data with regulators and the market in upcoming investor communications.
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