Atamyo Therapeutics has received FDA clearance for its Investigational New Drug (IND) application for ATA-200, a gene therapy designed to treat Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5). This clearance allows Atamyo to expand its Phase 1b dose-escalation clinical trial (NCT05973630) to the United States, marking a significant step in the development of a potential treatment for this rare and debilitating disease.
This expansion is supported by funding from The Dion Foundation for Children with Rare Diseases. ATA-200, an adeno-associated virus (AAV) vector-based gene therapy, delivers a functional copy of the human SGCG gene. Preclinical studies in mice have demonstrated that ATA-200 was well-tolerated and effectively corrected disease symptoms and biomarkers.
Clinical Trial Expansion and Design
The Phase 1b trial, already cleared in Italy and France, will now include sites in the US. The therapy will be administered intravenously. The trial aims to evaluate the safety and efficacy of ATA-200 in children suffering from LGMD2C/R5.
"This IND clearance is an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease," said Sophie Olivier, MD, Chief Medical Officer of Atamyo Therapeutics.
Regulatory Milestones and Orphan Drug Designation
Atamyo submitted the IND application to the FDA in September 2024, after which the agency granted rare pediatric disease designation to ATA-200. In addition to the IND clearance, the FDA has also granted ATA-200 orphan drug designation, further emphasizing the critical need for effective treatments for this rare condition.
"We are proud to be the first treatment for LGMD-2C/R5 to enter into clinical development in the US and we are committed to opening the first US center before year-end," stated Stéphane Degove, CEO of Atamyo Therapeutics.
ATA-200's Novel Design
ATA-200 incorporates a novel promoter designed to enhance liver and cardiac safety, a crucial aspect of gene therapy development. "ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy," said Isabelle Richard, PhD, cofounder and Chief Scientific Officer of Atamyo Therapeutics. "This first-in-class experimental treatment represents a new hope for the patients."
Atamyo's Broader Pipeline
In addition to ATA-200, Atamyo is also developing ATA-100, another AAV vector-based gene therapy targeting fukutin-related protein limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). ATA-100 is currently in a Phase 1/2 clinical trial (EudraCT 2021-004276-33, NCT05224505) across multiple European countries and the US. Atamyo recently completed recruitment for the dose escalation phase of the ATA-100 Phase 1b trial and plans to present preliminary results at upcoming conferences.
