Solid Biosciences(US:SLDB)2025-11-03 21:00SGT-003 Clinical Progress - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial[12, 23] - The company anticipates an FDA meeting in the first half of 2026 to discuss registrational pathways for SGT-003[13] - A Phase 3 randomized, placebo-controlled, ex-US trial of SGT-003, IMPACT DUCHENNE, has its first clinical trial site activated and participant screening underway as of October 2025[18] Microdystrophin Transduction and Localization - At Day 90, microdystrophin transduction was observed at 13 vector copies/nucleus (N=10), decreasing slightly to 12 vector copies/nucleus at Day 360 (N=2)[14] - At Day 90, microdystrophin protein expression was 58% by Western blot (WB), 58% by mass spectrometry (MS), and 51% by immunofluorescence (IF) (N=10)[16] - At Day 360, microdystrophin protein expression was 107% by WB, 100% by MS, and 67% by IF (N=2)[16] DAPC Restoration and Muscle Integrity - At Day 90, mean microdystrophin positive fibers were 51%, nNOS activity was 26%, and β-sarcoglycan positive fibers were 50% (N=10)[20] - At Day 90, there was a 34% mean reduction from baseline in CK levels, a 25% mean reduction in AST levels, a 42% mean reduction in LDH levels, a 22% mean reduction in Titin levels, and a 41% mean reduction in ALT levels[65] - At Day 90, eMHC positive fibers showed a 49% reduction[62] Cardiac Function - Early data suggests potential cardiac benefit through observed troponin I reductions & improvements in systolic function (LVEF)[18] - At Day 90, troponin levels showed a 31% mean reduction from baseline, increasing to a 70% mean reduction at Day 360[37]