Solid Biosciences(SLDB) - 2025 Q3 - Quarterly Results

Clinical Trials and Research - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial, with an expectation to dose a total of 30 participants by early 2026[1][6] - The company activated the first clinical trial site for FALCON, a Phase 1b trial for SGT-212, and expects to activate the first site for ARTEMIS, a Phase 1b trial for SGT-501, in Q4 2025[1][4] - The interim data from the INSPIRE DUCHENNE trial suggests a coordinated systemic response to treatment, reinforcing confidence in SGT-003's therapeutic potential[1][3] - Solid activated the first clinical trial site for the Phase 3 IMPACT DUCHENNE trial in October 2025, assessing SGT-003 in pediatric participants outside the U.S.[28] - The company plans to expand the IMPACT DUCHENNE trial into additional countries, having already received regulatory approvals in Canada and Australia[28] - SGT-212, the first investigational gene therapy for Friedreich's Ataxia, began participant screening in October 2025, with the trial designed to evaluate safety and tolerability[29] Financial Performance - The company ended Q3 2025 with $236.1 million in cash, cash equivalents, and available-for-sale securities, with an anticipated cash runway into H1 2027[1][2] - As of September 30, 2025, Solid had $236.1 million in cash, cash equivalents, and available-for-sale securities, an increase from $148.9 million as of December 31, 2024, enabling operational funding into the first half of 2027[35] - Research and Development (R&D) expenses for Q3 2025 were $38.9 million, up from $27.3 million in Q3 2024, primarily due to a $12.8 million increase in costs for SGT-003 related to manufacturing, regulatory, and clinical expenses[39] - General and Administrative (G&A) expenses for Q3 2025 were $9.2 million, compared to $7.9 million in Q3 2024, driven by increased personnel-related costs and legal fees[39] - The net loss for Q3 2025 was $45.8 million, compared to a net loss of $32.7 million in Q3 2024[39] - Total operating expenses for the nine months ended September 30, 2025, were $129.8 million, compared to $89.8 million for the same period in 2024[47] Treatment Outcomes - Day 90 biopsy data from 10 treated participants showed mean microdystrophin expression of 58% by western blot and mass spectrometry, with mean microdystrophin positive fibers at 51%[1][12] - Strong statistical correlations were observed between Day 90 microdystrophin expression levels and key components of the dystrophin-associated protein complex, with a Pearson correlation of 0.95 for both beta-sarcoglycan and nNOS[1][9] - Mean reductions in serum creatine kinase (CK) of 34% at Day 90 and 42% at Day 360 were observed, indicating muscle injury reduction[1][15] - Mean cardiac function trended into normal left ventricular ejection fraction (LVEF) ranges (60-69%) for all SGT-003-treated participants who reached the Day 180 follow-up[1][16] Regulatory and Development Plans - The company plans to meet with the FDA in H1 2026 to discuss potential registrational pathways for SGT-003[1][3] - The company anticipates completing PPQ manufacturing batches for SGT-003 in 2026, aligning with FDA discussions on registrational pathways[27] - Solid has executed over 30 agreements for the use of its proprietary AAV-SLB101 capsid, with more expected by year-end[34] Adverse Events - One treatment-related serious adverse event (SAE) was reported, identified as Grade 3 immune-mediated myositis, which resolved with steroid treatment[1][22]