Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update.
Bo Cumbo, President and CEO of Solid, commented: “The diligent execution of our Duchenne and FA development programs over the last 18 months has transformed Solid and given us tremendous momentum into 2025 and beyond. The initial 90-day data from the first three participants in the Phase 1/2 INSPIRE DUCHENNE trial of our next-generation Duchenne muscular dystrophy (Duchenne) program, SGT-003, while early, have shown a reassuring safety experience and promising improvements in biomarkers of muscle integrity and health. We believe these early indicators support the potential of SGT-003 to be a best-in-class gene therapy candidate to treat Duchenne. We understand the excitement around these data in the Duchenne patient communities and we are committed to moving with purpose and urgency to bring SGT-003 through the clinic as quickly as possible.” (Read More)
Positive Initial Clinical Data from SGT-003
Observed high vector genome copies per nucleus, robust microdystrophin expression and early improvements in additional measures of muscle integrity, including:
- Mean vector copies per nucleus: 18.7 (N=3),
- Mean microdystrophin expression: 110% (N=3), as measured by western blot,
- Mean microdystrophin expression: 108% (N=3), as measured by mass spectrometry,
- Mean percent dystrophin positive fibers: 78% (N=3), as measured by immunofluorescence,
- Mean beta sarcoglycan percent positive fibers: 70% (N=3),
- Mean nNOS (neuronal nitric oxide synthase) percent positive fibers: 42% (N=3),
- Improvements in 7 additional muscle integrity biomarkers (N=3), and
- Early mean improvement in left ventricular ejection fraction (LVEF) of 8% from baseline at Day 180 (N=2).
Learn More: Frequently Asked Questions About Solid Biosciences SGT-003 Gene Therapy
Meeting with the FDA to discuss potential accelerated approval pathways for SGT-003
- SGT-003 has been well tolerated in the 6 participants dosed as of the data cutoff date of February 11, 2025, with no serious adverse events (SAEs), suspected unexpected serious adverse reactions (SUSARs), or AEs of acute liver injury observed.
- Enrollment in the trial is ongoing, and the Company expects to dose more than 10 total participants by early second quarter 2025, and approximately 20 total participants by the fourth quarter of 2025.
- In mid-2025, the Company plans to request a meeting with the FDA to discuss potential accelerated approval pathways for SGT-003.
Learn More: Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
