NEW YORK – Atamyo Therapeutics on Monday said it has filed an investigational new drug (IND) application with the US Food and Drug Administration in the hopes of starting a Phase Ib/IIb trial of its gene therapy for patients with limb-girdle muscular dystrophy type 2C/R5 (LGMD-2C/R5).
Investigators will assess the safety, pharmacodynamics, efficacy, and immunogenicity of the ATA-200 gene therapy within the multicenter open-label dose-escalation study, in which children with LGMD-2C/R5 will receive a single intravenous infusion of the treatment. Evry, France-based Atamyo previously received regulatory clearance to launch this clinical trial in Italy and France.
LGMD-2C/R5 is a type of muscular dystrophy caused by mutations in the SGCG gene that produces gamma-sarcoglycan, a protein that supports the connections between muscle fibers and their environment. ATA-200 aims to treat the condition by using an adeno-associated virus vector to deliver a functional copy of a SGCG transgene.
The clinical trial is being funded by the Dion Foundation for Children with Rare Diseases.
Atamyo also announced that it has enrolled the last patient into the dose-escalation phase of its multicenter Phase Ib trial studying the pharmacodynamics and efficacy of ATA-100, a gene therapy it's developing for another type of limb-girdle muscular dystrophy, LGMD-2I/R9, which is caused by mutations in the FKRP gene that normally produces fukutin-related protein.
ATA-100 is designed as a one-time gene therapy that delivers a functional copy of the FKRP gene. The first patients treated with ATA-100 have experienced promising functional results, and the gene therapy has been well tolerated in patients treated so far, Atamyo CEO and Cofounder Stephane Degove said in a statement.
Atamyo, a spinoff of Genethon, also is in the midst of IND-enabling studies for a gene therapy candidate aiming to treat LGMD-2A/R1, which stems from deficiencies in the calpain-3 protein.