Solid Biosciences Inc. announced positive interim data from its Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 as of February 23, 2026, showing robust microdystrophin expression, restored beta-sarcoglycan and nNOS activity, biomarker improvements, and stabilization in cardiac function across 40 dosed participants. The company aligned with FDA on Phase 3 IMPACT DUCHENNE trial design, with first dosing anticipated in Q1 2026.
Key takeaways
40 participants dosed with SGT-003 as of March 11, 2026; generally well tolerated with steroid-only regimen
Day 90 biopsy (n=20): mean 11 vector copies/nucleus, 60% microdystrophin by western blot, 60% beta-sarcoglycan-positive fibers, 35% nNOS-positive fibers
Day 360 biopsy (n=3): mean 12 vector copies/nucleus, 91% microdystrophin by western blot, 69% beta-sarcoglycan-positive fibers
Day 90 serum biomarkers (n=24): 38% CK reduction, 43% ALT reduction, 46% LDH reduction; sustained at Day 360
FDA alignment on Phase 3 IMPACT DUCHENNE design for ages 7-<12, primary endpoint TTR velocity at 18 months; screening underway, first dose Q1 2026