Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss potential registrational pathways for SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid, which is designed to increase skeletal and cardiac muscle targeting while detargeting the liver.
According to Solid, day 90 biopsy data from 10 treated participants (ages 5–10) showed:
- Mean microdystrophin expression of 58% (how much microdystrophin protein is seen in the biopsy).
- Mean microdystrophin positive fibers of 51% (how many muscle fibers in the biopsy showed expression of the microdystrophin).
In addition to the expression and distribution in the biopsies, Solid reported:
- Strong observed biological correlations of SGT-003 microdystrophin expression levels with properly localized restoration of key components of the dystrophin-associated protein complex. This can represent a way to understand functionality of the produced microdystrophin.
- Stabilization and improvement in cardiac function, with both reductions in elevated baseline serum cardiac troponin I levels and a normalization in left ventricular ejection fraction.
- Solid reported that SGT-003 has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen.
- Additionally, improvement in a number of muscle integrity biomarkers were shared that correlated strongly with microdystrophin expression.
The company plans to meet with the FDA in the first half of 2026 to discuss potential registrational pathways, including accelerated approval, for SGT-003. Solid continues to dose participants in the INSPIRE DUCHENNE trial in the interim, with additional participant safety, clinical activity and functional data expected to enable a more robust discussion with the FDA.
PPMD looks forward to future updates from Solid on SGT-003’s progress, and we will share updates with the community as information becomes available.
Read Solid’s press release here.
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