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Investing.com -- Shares of Solid Biosciences Inc. (Nasdaq: NASDAQ:SLDB) surged 27% following the release of positive initial clinical data from its next-generation Duchenne gene therapy candidate, SGT-003. The company reported encouraging results from the Phase 1/2 INSPIRE DUCHENNE trial, which included average microdystrophin expression of 110% among the first three participants, and significant improvements in various muscle health biomarkers.
The trial data, which was reported on February 18, 2025, also suggested potential cardiac benefits from the therapy. The company mentioned that SGT-003 has been well-tolerated with no serious adverse events observed as of February 11, 2025. The promising results have sparked investor optimism, leading to a sharp rise in the company’s stock price.
Solid Biosciences also announced the pricing of an underwritten offering of shares and pre-funded warrants, which is expected to alleviate any near-term financing concerns. The company’s CEO, Bo Cumbo, expressed satisfaction with the trial’s findings, highlighting the comprehensive analysis and the potential best-in-class expression of the microdystrophin transgene.
Analysts have taken note of the data’s implications for the Duchenne muscular dystrophy (DMD) treatment landscape. Truist analyst Joon Lee, M.D., Ph.D., commented on the significance of the findings: "data from 3 DMD patients with at least 90 days of follow up were shared pointing to best-in-class profile of SGT-003." Lee also pointed out the impressive microdystrophin protein expression levels and other biomarkers that suggest SGT-003 could demonstrate clinical benefit in future studies.
Leerink Partners analyst Joseph P. Schwartz also weighed in, stating that the interim data makes SGT-003 "a serious contender" in DMD compared to key competitors. Schwartz believes that the DMD program is just the beginning for Solid Biosciences, with the potential for their cardiac pipeline to drive the company’s growth beyond DMD.
The INSPIRE DUCHENNE trial is ongoing, with the company planning to dose approximately 20 participants by the fourth quarter of 2025. Solid Biosciences intends to request a meeting with the FDA in mid-2025 to discuss the potential for an accelerated approval pathway for SGT-003, which could fast-track the therapy’s availability to patients if successful.
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