Solid Biosciences Reports Third Quarter 2025 Financial Results and Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions

Core Insights - Solid Biosciences reported positive interim data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, indicating its therapeutic potential for Duchenne muscular dystrophy [1][2][26] - The company plans to meet with the FDA in the first half of 2026 to discuss potential registrational pathways for SGT-003, including accelerated approval options [26][27] - Solid has also activated clinical trial sites for other gene therapy candidates, SGT-212 for Friedreich's ataxia and SGT-501 for catecholaminergic polymorphic ventricular tachycardia [29][31] Clinical Trial Updates - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial, with plans to dose a total of 30 participants by early 2026 [1][6] - The trial has shown strong statistical correlations between microdystrophin expression levels and key components of the dystrophin-associated protein complex, indicating effective treatment [7][11] - The first clinical trial site for IMPACT DUCHENNE, a Phase 3 trial for SGT-003, has been activated, with plans to expand into additional countries [28] Safety and Efficacy Data - SGT-003 has been generally well tolerated, with a steroid-only prophylactic immunomodulation regimen showing minimal adverse effects [21][22] - Day 90 biopsy data indicated a mean microdystrophin expression of 58% and significant reductions in biomarkers of muscle injury, such as serum creatine kinase (CK) [11][14] - Cardiac function improvements were observed, with mean reductions in serum cardiac troponin I levels and normalization of left ventricular ejection fraction [17][18] Financial Position - As of September 30, 2025, Solid Biosciences reported $236.1 million in cash and equivalents, providing a cash runway into the first half of 2027 [35] - Research and development expenses for Q3 2025 were $38.9 million, reflecting increased costs associated with SGT-003 [35][36] - The net loss for Q3 2025 was $45.8 million, compared to $32.7 million for the same period in 2024 [44]