Mar. 11 at 7:45 PM
$SLDB Solid Biosciences says interim results reinforce confidence in SGT-003 potential
Solid Biosciences reported updated "positive" interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. The new data, including additional muscle biopsy, serum biomarker, cardiac function, and safety analyses, "add to a growing dataset that suggests the potential of the biological activity of SGT-003 microdystrophin therapy," the company stated. The interim update was delivered in an oral presentation at the 2026 MDA Annual Meeting on March 11. "SGT-003 has been generally well tolerated in the 40 participants dosed as of March 11, 2026. The safety and tolerability profile observed in the INSPIRE DUCHENNE trial continued to be promising; SGT-003 is administered using a low-burden, steroid-only prophylactic immunomodulation regimen.
The trial is being conducted at 15 clinical sites across the US, Canada, Italy and the United Kingdom and participant dosing remains ongoing. Microdystrophin transduction and expression levels, beta-sarcoglycan localization and nNOS activity were evaluated by biopsy in 20 participants (ages 1-10 years) at Day 90 and in 3 participants at Day 360. Results demonstrated robust mean vector copies per nucleus and microdystrophin expression as well as properly localized and restored beta-sarcoglycan-positive fibers and nNOS activity-positive fibers," the company stated.
Bo Cumbo, President and CEO of Solid Biosciences commented, "These data continue to suggest that SGT-003 may be having a disease-relevant treatment effect, with observed robust microdystrophin expression, restoration of key components of the dystrophin-associated protein complex suggesting a direct biologic correlate of dystrophin activity, and improvements across multiple biomarkers of muscle integrity.
Together with the promising safety and tolerability profile, these interim results reinforce our confidence in the potential of SGT-003 to meaningfully impact the disease course of Duchenne. Duchenne continues to have an unmet medical need. We are pursuing guidance on a potential accelerated approval pathway for SGT-003 and look forward to continued engagement with the FDA as we work toward that goal. Our focus remains on urgently advancing SGT-003 to provide the Duchenne community with an additional therapeutic choice."
