We’re excited to share news that Solid Biosciences has released initial data from its Phase 1/2 INSPIRE DUCHENNE clinical trial, assessing the safety and efficacy of SGT-003 – a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD). Although these are early findings, the results suggest good movement in the development of potential future gene therapies that look to address the underlying cause DMD.
The key findings from the first three patients, aged 5 and 7 years, demonstrated positive improvements in muscle health, microdystrophin expression, and cardiac muscle health:
- Significant Microdystrophin Expression:
- Average microdystrophin expression of 110% was observed.
- Reduction in Muscle Injury Markers:
- Notable reductions in key muscle injury biomarkers, including CK, AST, ALT, Titin, LDH, and eMHC, were recorded.
- Potential Cardiac Benefit:
- Early indications of potential cardiac benefit were observed (via left ventricular ejection fraction measurement).
- Safety and Tolerability:
- SGT-003 was well-tolerated across all patients dosed to date (6 total).
- Observed adverse events were mild and consistent with typical AAV-based gene therapies, including nausea, vomiting, fever, and transient platelet decreases.
- No serious adverse events (SAEs) were reported.
Trial Progress and Future Plans:
- Solid Biosciences intends to request a meeting with the U.S. Food and Drug Administration (FDA) in mid-2025 to discuss the potential for an accelerated approval pathway for SGT-003.
- The INSPIRE DUCHENNE trial aims to enroll approximately 20 patients by the end of 2025.
- Patient enrollment is ongoing at six clinical trial sites in the United States and Canada, with plans to expand to the U.K. and Italy.
We are extremely pleased to present our initial clinical data from the INSPIRE DUCHENNE trial. When starting this trial, we committed to comprehensively analysing the effects of SGT-003. To that end, three different measurement methodologies showed what we believe to be potential best-in-class expression of our differentiated microdystrophin transgene. Significant reductions observed in all evaluated clinical biomarkers of muscle damage associated with Duchenne provide preliminary evidence of a beneficial effect in muscle integrity, including potential early signals of a positive cardiac benefit of SGT-003 in these young boys. In mid-2025, we plan to request a meeting with the FDA to discuss the potential for an accelerated approval regulatory pathway for SGT-003.
Bo Cumbo, President and CEO, Solid Biosciences.
About the INSPIRE DUCHENNE Trial
The INSPIRE DUCHENNE trial is an early-stage, Phase 1/2 clinical trial testing the safety and effectiveness of SGT-003, an experimental one-time gene therapy for children with Duchenne muscular dystrophy. Participants receive a single dose of SGT-003 through an IV infusion, with outcomes measures consisting of
Current Progress (as of February 11, 2025):
- Six children have received the therapy so far.
- Enrolment is ongoing, with plans to treat at least 10 participants by spring 2025 and around 20 by late 2025.
Trial Locations:
- Active sites are currently running in 6 clinics across the U.S. and Canada.
- The trial has regulatory approval to expand to the UK and Italy, with additional sites expected to open by the end of 2025.
If you have any questions or enquiries about the topics discussed in this article, please do reach out to us as info@actionduchenne.org.
