Rare Daily Staff

Solid Biosciences said the first patient dosed in the second cohort of its IGNITE DMD phase 1/2 study of its SGT-001, its lead gene therapy candidate for Duchenne muscular dystrophy, suffered a serious adverse event related to the treatment.

The company made the announcement in a release of its first quarter of 2019 financial results.

Shortly after dosing, the patient experienced a transient elevation of transaminases, as well as a transient increase in bilirubin higher than two times the upper limit of normal, which was rapidly resolved with an increase in oral glucocorticoids. This was reported to the U.S. Food and Drug Administration as a serious adverse event related to study drug.

The patient also experienced a transient decline in platelet count that was considered a non-serious adverse event related to the study drug. And, the patient was diagnosed with a gastrointestinal infection that was classified as a serious adverse event unrelated to the study drug. Both of these events were fully resolved. The patient is doing well and has resumed normal activities.

Solid continues to enroll patients in IGNITE DMD per the study protocol and anticipates providing additional data later this year.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by mutations in the dystrophin gene that result in the absence of dystrophin, a protein that keeps muscle cells intact. It leads to progressive muscle degeneration and weakness and occurs mainly in boys, most whom do not survive past their teen years.

Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability correct mutations in the dystrophin gene. SGT-001 is systemically administered to deliver a synthetic dystrophin transgene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins. SGT-001 utilizes AAV9, which has an affinity for muscle and is currently being evaluated in multiple clinical programs in other indications.

“We have continued to advance our programs for Duchenne muscular dystrophy over the last few months, particularly our lead microdystrophin gene therapy candidate, SGT-001. Building on our findings from the preliminary phase 1/2 clinical data that we announced in February, we initiated dosing at 2E14 vg/kg in the second cohort of patients,” said Ilan Ganot, president and CEO of Solid Biosciences. “Now, we are working to progress as quickly as possible, and we look forward to providing additional data later this year.”

Photo: Ilan Ganot, president and CEO of Solid Biosciences

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