Solid Biosciences Stock Surges On Upbeat Duchenne Muscular Dystrophy Gene Therapy Data

On Tuesday, Solid Biosciences Inc. SLDB released initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness caused by the alterations of a protein called dystrophin, which helps keep muscle cells intact.

Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110% and improvements in multiple biomarkers that indicate muscle health and resilience.

Between 70% and 88% of the participants’ muscle fibers showed dystrophin, with an average of 78%.

Also Read: Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development

The company also reported lower markers of muscle injury and stress, along with an 8% average improvement in heart function in two patients after 180 days.

SGT-003 was well-tolerated in the first six participants dosed.

Adverse events (AEs) observed after SGT-003 treatment were typical of those observed in AAV gene therapy, including nausea, vomiting, fever, and transient declines in platelets in some participants.

No serious adverse events (SAEs) or suspected unexpected serious adverse reactions were observed, and there was no evidence of thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS), or hemolysis.

None of the AEs observed required the use of additional immunomodulatory agents such as eculizumab, sirolimus, or rituximab.

Enrollment in the INSPIRE DUCHENNE trial is ongoing. At least 10 participants are anticipated to be dosed by early in the second quarter of 2025, and approximately 20 participants are anticipated to be dosed by the fourth quarter of 2025.

Price Action: Solid Biosciences stock is up 38.3% at $5.57 at last check Tuesday.

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