Solid Biosciences(US:SLDB)2025-02-18 11:55Financial Position - As of December 31, 2024, the company reported approximately $148.9 million in cash, cash equivalents, and available-for-sale securities[18]. Clinical Trials and Results - The initial data from the Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 showed an average microdystrophin expression of 110% in the first three participants[5]. - The trial reported mean reductions in muscle injury markers: serum creatine kinase (CK) decreased by 57%, serum aspartate aminotransferase (AST) by 45%, and serum alanine transaminase (ALT) by 54%[12]. - The company plans to dose at least 10 participants in the INSPIRE DUCHENNE trial by early Q2 2025 and approximately 20 by Q4 2025[10]. - The company observed a mean cardiac function increase of 8% from baseline as measured by left ventricular ejection fraction at Day 180[12]. - The first six participants in the SGT-003 trial experienced no serious adverse events, with common adverse events being nausea and vomiting[8]. Regulatory and Development Plans - The FDA has granted Fast Track designation to SGT-212 for the treatment of Friedreich's ataxia, with a Phase 1b trial expected to start in H2 2025[14]. - SGT-501 is undergoing IND-enabling GLP toxicology studies, with an IND submission anticipated in the first half of 2025[15]. - The company expects to activate additional clinical trial sites by the end of 2025, expanding its reach in the U.S. and Canada[11]. - The company plans to request a meeting with the FDA in mid-2025 to discuss the potential for an accelerated approval pathway for SGT-003[11].