NEW YORK – Atamyo Therapeutics and the Dion Foundation for Children with Rare Diseases on Wednesday said they will work together to launch a clinical trial in the US and test Atamyo's investigational gene therapy ATA-200 in certain muscular dystrophy patients.
In the partnership, the Dion Foundation, a nonprofit that supports research into treatments for rare genetic diseases, will fund the US-based trial of ATA-200 in children with γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). In the multicenter Phase Ib dose-escalation trial, which already has regulatory clearance to proceed in Italy and France, researchers are evaluating ATA-200's safety, pharmacodynamics, efficacy, and immunogenicity.
According to Atamyo CEO Stéphane Degove, the Genethon spinoff is already preparing to file an investigational new drug application seeking permission from the US Food and Drug Administration to start this Phase Ib trial of ATA-200 in the US.
LGMD2C/R5 is due to mutations in the gene that produces the transmembrane protein γ-sarcoglycan, which plays a role in connecting muscle fibers and their environment. Patients with LGMD2C/R5 experience symptoms in early childhood including muscle weakness, loss of movement, and cardiac issues. There is no cure for the disease, only supportive treatments.
ATA-200, developed by Evry, France-based Atamyo, comprises an adeno-associated virus vector carrying a normal copy of the gene that produces γ-sarcoglycan. In preclinical models, an injection of ATA-200 appeared to correct symptoms and biomarkers associated with LGMD2C/R5. According to Atamyo, ATA-200 has been deemed an orphan medicinal product by regulators in Europe, where around 2,000 people have this condition.