Solid Biosciences announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial. The Company also provided an update to the planned timing of its meeting with the U.S. Food and Drug Administration (FDA) to discuss potential registrational pathways, including accelerated approval pathways, for SGT-003.
Bo Cumbo, President and CEO of Solid Biosciences commented, “The interim INSPIRE DUCHENNE data reported today strengthens our confidence in SGT-003’s therapeutic potential. From strong observed biological correlations of SGT-003 microdystrophin expression levels with properly localized restoration of key components of the dystrophin-associated protein complex to early evidence of cardiac function normalization, we are observing a clear and cascading effect in the human body, suggesting a coordinated, systemic response to treatment. We believe these interim data represent one of the most thorough early analyses of any Duchenne gene therapy to date. The quality and concurrence of these interim findings reinforce our conviction in SGT-003’s potential to translate molecular impact into meaningful clinical outcomes.”
Mr. Cumbo continued, “In light of the evolving regulatory landscape and the rapid pace of enrollment in INSPIRE DUCHENNE, we have made the proactive decision to move our planned meeting with the FDA to the first half of 2026. The additional time will enable us to 1) generate a more fulsome data set for discussion with the FDA, 2) work towards a comprehensive external comparator based on high-quality, well-matched natural history data, 3) begin our process performance qualification (PPQ) manufacturing runs with our CDMO partner in preparation for a potential biologics license application (BLA) submission, and 4) initiate dosing in IMPACT DUCHENNE, our Phase 3 randomized, double-blind, placebo-controlled clinical trial that will be conducted outside of the United States. We believe these activities put Solid in the best position to deliver the most compelling package to regulators.
“Our priority now is to rapidly build a robust data set to support a potential accelerated regulatory pathway for SGT-003, and we are committed to executing with urgency to deliver on SGT-003’s potential as quickly as possible. Beyond SGT-003, our lead pipeline programs continue to progress.” Mr. Cumbo concluded.
SGT-003 – INSPIRE DUCHENNE Clinical Trial
- 23 participants have been dosed in the INSPIRE DUCHENNE trial as of October 31, 2025; Solid expects to have dosed 30 participants in total by early 2026, then plans to meet with the FDA to discuss potential registrational pathways in H1 2026 –
- SGT-003 has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen; cardiac safety monitoring continued to show reduction in cardiac injury and early signals of cardiac function normalization –
- Day 90 biopsy data from 10 treated participants (ages 5-10) showed all participants responded to treatment with mean microdystrophin expression of 58% by western blot, 58% by mass spectrometry, mean microdystrophin positive fibers of 51% by immunofluorescence, and mean beta-sarcoglycan positive fibers of 50% by immunofluorescence –
- Strong statistical correlations observed between Day 90 microdystrophin expression levels and key components of the dystrophin-associated protein complex (DAPC), beta-sarcoglycan (r = 0.95) and nNOS (r = 0.95), demonstrated evidence of SGT-003-mediated DAPC restoration and concordant signals of muscle protection via reductions in CK levels (r = -0.78) –
- Solid has activated the first clinical trial site and is currently screening participants for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled ex-U.S. clinical trial of SGT-003 –
Favorable reductions across a range of biomarkers of muscle injury and breakdown were observed through both Day 90 and Day 360:
| Serum Biomarkers | Day 90 Mean Reductions (N=14 unless noted) | Day 360 Mean Reductions (N=3 unless noted) |
| Serum creatine kinase (CK) | 34% | 42% |
| Serum alanine transaminase (ALT) | 41% | 29% |
| Serum aspartate aminotransferase (AST) | 25% | 40% |
| Serum lactate dehydrogenase (LDH)* | 42% | 46% |
| Serum titin** | 22% | 25% |
| *N=12 participant samples available at Day 90 for LDH (two samples hemolyzed); **N=11 participant samples available at Day 90 and N=2 samples available at Day 360 for titin, which was batch-analyzed at an earlier cutoff date. | ||
IMPACT DUCHENNE Phase 3
In October 2025, Solid activated the first clinical trial site and began participant screening for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled clinical trial assessing SGT-003. IMPACT DUCHENNE will be conducted in pediatric participants outside of the United States (U.S.) and was designed to support potential ex-U.S. regulatory authorizations. We have received regulatory approvals to conduct IMPACT DUCHENNE in both Canada and Australia, and we plan to expand the trial into additional countries, subject to receipt of regulatory approvals. (IMPACT DUCHENNE)
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