Stock Price Drop#
Solid Biosciences Inc. experienced a significant decline in its stock price, dropping more than 8% on Wednesday. This decrease followed the company's announcement of interim results from its gene therapy trial aimed at treating Duchenne muscular dystrophy. Trading of the stock was temporarily halted earlier in the day due to the volatility.
Trial Overview#
The data released pertains to the Phase 1/2 INSPIRE DUCHENNE trial, which is evaluating SGT-003, a gene therapy designed to address Duchenne muscular dystrophy. So far, 40 participants have received the treatment, and the trial aims to assess its safety and effectiveness.
Key Findings#
The trial results indicated that the treatment led to the expression of microdystrophin, a shortened version of the dystrophin protein that is crucial for muscle function. Notably, improvements were observed in muscle integrity and cardiac function, measured by the left ventricular ejection fraction, which indicates how well the heart pumps blood.
Detailed Results#
Muscle biopsy analyses from 20 participants at 90 days post-treatment showed an average microdystrophin expression level of 60%. By 360 days, three participants exhibited a remarkable 91% expression level. Additionally, there was a 38% reduction in serum creatine kinase, an enzyme that indicates muscle damage, and a 44% reduction in embryonic myosin heavy chain positive fibers at the 90-day mark. Solid Biosciences suggested that these findings indicate a potential disruption of the ongoing muscle degeneration and regeneration cycle typical in Duchenne muscular dystrophy.