By Chris Wack
Solid Biosciences shares were up 50% to $6.06 after the company said it saw positive initial data from a Phase 1/2 trial evaluating SGT-003, a gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy.
The life-sciences company said 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.
Duchenne muscular dystrophy is a severe, progressive genetic disorder that primarily affects boys. It causes muscle weakness and degeneration, leading to significant physical disabilities.
The company said in mid-2025, it plans to request a meeting with the Food and Drug Administration to discuss the potential for an accelerated approval regulatory pathway for SGT-003.
SGT-003 was well-tolerated in the first six participants dosed as of the data cutoff date of Feb 11. As of the cutoff date, all six participants have reached at least 20 days post SGT-003 treatment. No serious adverse events or suspected unexpected serious adverse reactions were observed.
The trial currently has a total of six active clinical sites in the U.S. and Canada, and approved clinical trial applications in the U.K. and Italy. Solid expects to activate additional trial sites by the end of 2025.
Solid Biosciences also priced an underwritten offering of 35.7 million shares at $4.03 a share, and, in lieu of common stock to certain investors, pre-funded warrants to buy 13.9 million shares at $4.029 per pre-funded warrant.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
February 18, 2025 10:31 ET (15:31 GMT)
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