A modest production of dystrophin from 3 muscular biopsies in three participates in a phase I/II trial, IGNITE-DMD, after 3 months of treatment with SGT-001.
SGT-001, developed by the Solid Biosciences organization, a gene therapy medicine candidate that uses the microdystrophin gene. A phase I/II clinical trial (IGNITE DMD), currently ongoing in the United States, is evaluating increasing doses of SGT-001 in 16 boys between 4 and 17 years of age with Duchenne muscular dystrophy. Six participants have been included for the time being; three among them having received the smallest dose of SGT-001 by intravenous injection, while three other control patients were not treated. Solid Biosciences issued a press release announcing the preliminary muscle biopsy analyses of the first 3 patients following 3 months of therapy. In one participant, a slight production of dystrophin was observed: 5% of dystrophin viewed via western blot and 10% of muscular fibers tested positive by immunofluorescence. Furthermore, the expression of certain dystrophin activity biomarkers (nNOS and beta-sarcoglycan) eludes to the fact that the dystrophin produced is functional.
These results must be confirmed in the higher doses of SGT-001 and over the long-term. As the production is modest in GMRD dogs, a Duchenne muscular dystrophy animal model, it is necessary that at least 40% of muscle fibers express dystrophin to improve the muscle.
Access Solid Biosciences press release (7Feb19) Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy
On Clinicaltrials.gov NCT03368742
