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Dive Brief:
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An experimental Duchenne muscular dystrophy gene therapy from Solid Biosciences showed potential in a small clinical trial, leading the biotechnology company to quickly raise funds on the findings.
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Three months after treatment with Solid’s therapy, SGT-003, the first three participants in an early-stage clinical trial produced higher-than-normal levels of a tiny protein linked to muscle function, Solid said Tuesday. No serious side effects were observed so far, the company added.
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Solid claims the results, while early, suggest SGT-003 could be more potent than Sarepta Therapeutics’ Elevidys, the only approved Duchenne gene therapy. The company intends to discuss an accelerated approval pathway with U.S. regulators later this year. Solid’s share price rose by as much as 79% Tuesday morning before settling back to trade up 40%. It announced a $200 million stock offering alongside the study results.
Dive Insight:
Solid’s results are a step in the company’s yearslong turnaround.
Solid was one of the first developers of so-called microdystrophin gene therapy for Duchenne, a progressive and fatal muscle-wasting condition. As their name suggests, these treatments help people produce a miniature or “micro” version of a protein — dystrophin — that stabilizes muscles. Developers have hoped doing so might halt or even reverse Duchenne’s inexorable onslaught.
Yet, while other gene therapies advanced, Solid’s initial program was stalled multiple times due to safety concerns. Those issues led to a depressed share price and corporate restructuring. In 2022, the company overhauled its management team and strategy upon merging with a gene therapy startup. Since then, Elevidys won approval and a Regenxbio treatment reached late-stage testing, raising the bar for would-be competitors.
Solid has claimed SGT-003 could be more potent than its predecessor and potentially other programs, too. There’s room to improve upon Elevidys, which in multiple placebo-controlled trials produced mixed results that have led to persistent questions about its benefit.
While Solid’s results are from only three study participants between five and seven years old, they point to a “highly differentiated, potential class-leading” medicine, said CEO Bo Cumbo, a former Sarepta executive, on a Tuesday conference call.
Notably, the average microdystrophin levels after three months were higher than what’s been reported at similar time points in testing of Elevidys and Regenxbio’s therapy. A treatment Pfizer brought to late-stage testing but later shelved was also associated with lower levels, albeit at a later time point.