Solid Biosciences’ gene therapy candidate for patients with Duchenne muscular dystrophy (DMD) has elicited a 110% expression of the microdystrophin gene.
The US-based company’s stock soared by 78.66% following the announcement, from a 14 February close of $4.03 to a high of $7.20 after the markets opened on 18 February.
The company announced 90-day biopsy data from the Phase I/II Inspire DMD trial (NCT06138639) of its gene therapy candidate SGT-003. The first three patients, out of six, saw a 110% expression of microdystrophin, which can slow disease progression and potentially preserve muscle health.
At the same time, the company reported improvements in multiple biomarkers used as indicators of muscle health and resilience, including a 78% increase in dystrophin-positive fibres. There was also a reduction in biomarkers associated with muscle stress and degeneration.
It also added that SGT-003 was well-tolerated in the first six patients dosed, with all patients reaching at least 20 days post-SGT-003 treatment.
Now, the company says it plans to hold a meeting with the US Food and Drug Administration (FDA) seeking an accelerated approval regulatory pathway for SGT-003.
Solid Biosciences CEO Bo Cumbo said: “Three different measurement methodologies showed what we believe to be a potential best-in-class expression of our differentiated microdystrophin transgene.
"Significant reductions observed in all evaluated clinical biomarkers of muscle damage associated with DMD provide preliminary evidence of a beneficial effect in muscle integrity, including potential early signals of a positive cardiac benefit of SGT-003 in these young boys.”
SGT-003 is an intravenous, adeno-associated virus (AAV) that delivers nNOS-containing microdystrophin. It activates dystrophin, a structural protein found in small amounts in normal muscle.
DMD is a rare genetic form of muscular dystrophy that typically occurs in young boys in early life. It is characterised by progressive muscle degeneration as a result of alterations in dystrophin. Microdystrophin is a shortened version of the dystrophin gene.
The first three patients in the first-in-human, open-label study are two five-year-old boys and one seven-year-old boy at the time of dosing.
Solid Biosciences' chief medical officer Gabriel Brooks said: “While the loss of normal dystrophin is the defining molecular hallmark of DMD, there is growing understanding within the community that the success of microdystrophin gene therapy extends beyond expression, and will also depend on signals of restoration and preservation of muscle health, which were observed in these early clinical data.”
Elsewhere in the field of DMD, Cumberland Pharmaceuticals has reported positive top-line outcomes from a Phase II trial of ifetroban as a potential treatment for the condition. Meanwhile, Entrada Therapeutics has secured authorisation from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) to begin its two-part Phase I/II trial ELEVATE-44-201.
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