Solid Biosciences surges on Duchenne therapy success

Boston, USA-based biotech Solid Biosciences (Nasdaq: SLDB) saw its shares rocket 36% to $5.48 yesterday, after the precision genetic medicines for neuromuscular and cardiac diseases company announced positive initial data from the Phase I/II INSPIRE DUCHENNE trial evaluating SGT-003.

Solid noted that 00SGT-003 is a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience.

“We are extremely pleased to present our initial clinical data from the INSPIRE DUCHENNE trial,” said Bo Cumbo, president and chief executive of Solid Biosciences. “When starting this trial, we committed to comprehensively analyzing 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.”