Solid Biosciences shares fall after gene therapy trial data
Investing.com -- Solid Biosciences Inc. shares dropped more than 8% on Wednesday after the company released data from its Duchenne gene therapy trial. Trading in the stock was briefly halted earlier in the day.
The company reported interim results from its Phase 1/2 INSPIRE DUCHENNE trial, which is testing SGT-003, a gene therapy for Duchenne muscular dystrophy. As of Wednesday, 40 participants have received the treatment.
The trial showed microdystrophin expression with restoration of dystrophin associated protein complex components. The company also reported improvements in muscle integrity markers and cardiac function stabilization measured by left ventricular ejection fraction.
Muscle biopsy results from 20 participants at day 90 showed mean microdystrophin expression levels of 60% by western blot analysis. Three participants at day 360 showed 91% expression. Beta-sarcoglycan-positive fibers showed 60% restoration at day 90 and 69% at day 360.
The company reported a 38% reduction in serum creatine kinase and a 44% reduction in embryonic myosin heavy chain positive fibers at day 90. Solid Biosciences stated these results suggest disruption of the chronic degeneration-regeneration cycle characteristic of Duchenne.
