FDA Clears IND for ATA-200 Gene Therapy for LGMD2C/R5

EVRY, France-- Atamyo Therapeutics, a biotechnology company at the clinical stage, has announced that the U.S. Food & Drug Administration (FDA) has approved its Investigational New Drug (IND) application for ATA-200. This approval allows the company to proceed with a Phase 1b/2b clinical trial. ATA-200 is a gene therapy designed as a one-time treatment for γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a severe condition that impacts children, causing loss of mobility before they reach adulthood.

The Phase 1b, open-label, dose-escalation study (NCT05973630) will be conducted across multiple centers and will assess various parameters such as safety, pharmacodynamics, efficacy, and immunogenicity in children administered a single-dose intravenous ATA-200. This treatment uses an Adeno-Associated Virus (AAV) vector to carry the human γ-sarcoglycan transgene. The clinical trial in the United States receives funding from The Dion Foundation for Children with Rare Diseases and has already gained regulatory approval in France and Italy.

Dr. Sophie Olivier, Chief Medical Officer at Atamyo, emphasized the importance of this IND clearance for bringing ATA-200 to affected children in the US. Stéphane Degove, Atamyo’s Chief Executive Officer, highlighted that this would be the first treatment for LGMD-2C/R5 to enter clinical development in the United States, with plans to open the first US center by the end of the year.

Additionally, the US FDA has granted Orphan Drug Designation to ATA-200, which provides seven years of market exclusivity in the United States. This designation came shortly after the FDA gave ATA-200 the Rare Pediatric Disease Designation.

LGMD-2C/R5 is a rare genetic disorder caused by mutations in the gene responsible for producing γ-sarcoglycan, a protein crucial for the interaction between muscle fibers and their environment. The condition affects about 2,000 individuals in Europe and the US. Symptoms usually manifest in early childhood, leading to progressive muscle weakness and loss of mobility by adulthood. Around half of the patients may also develop dilated cardiomyopathy, affecting life expectancy. Currently, there is no cure for LGMD-2C/R5, and treatment is primarily supportive.

ATA-200 aims to address this by delivering a normal copy of the gene responsible for γ-sarcoglycan production. Preclinical studies in mice have shown that ATA-200 is both tolerable and effective in correcting symptoms and biomarkers of the disease. Atamyo plans to begin enrolling patients for the clinical trial in the fourth quarter of 2024.

The gene therapy is rooted in the research of Dr. Isabelle Richard, Chief Scientific Officer at Atamyo and Research Director at CNRS. Dr. Richard leads the Progressive Muscular Dystrophies Laboratory at Genethon, where the foundational research for this therapy was conducted.

Atamyo Therapeutics, spun off from gene therapy pioneer Genethon, specializes in developing advanced gene therapies for neuromuscular diseases. The company leverages expertise in AAV-based gene therapy and muscular dystrophies from Genethon’s Progressive Muscular Dystrophies Laboratory. Atamyo’s leading programs focus on various forms of limb-girdle muscular dystrophies (LGMD), with clinical-stage programs targeting LGMD-R9 and LGMD-R5. The company’s name combines the Celtic word "Atao," meaning "Always" or "Forever," and the Greek root "Myo," meaning muscle, signifying its commitment to providing life-long, effective treatments for patients with neuromuscular diseases.