Atamyo, Dion Foundation partner to expand LGMD study into US
Clinical trial testing ATA-200 gene therapy for LGMD type 2C/R5
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Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5.
The Phase 1b study (NCT05973630), designed to evaluate the safety, efficacy, and pharmacological and immunological properties of ATA-200, was approved this year by regulatory authorities in France and Italy.
Now, Dion, a U.S. nonprofit, is working with the France-based pharmaceutical to bring that trial to the United States.
“We are thrilled by this key partnership and grateful to the Dion Foundation for their financial support which aims to include US patients in the first-in-human trial for ATA-200,” Stéphane Degove, Atamyo’s CEO, said in a press release. “We are already engaged in the preparation of an [investigational new drug] filing in the US for ATA-200.”
Phase 1b trial is first to test ATA-200 in LGMD type 2C/R5 patients
LGMD is a group of diseases characterized by muscle wasting in the shoulders and hips. A specific subtype of the genetic condition, LMGD2C/R5 is caused by mutations in the SGCG gene, which provides instructions for producing the protein gamma-sarcoglycan. Such mutations prevent the stabilization of dystrophin, another protein that protects muscle fibers from damage during movements.
Symptoms of LGMD typically start in early childhood and include progressive muscle weakness and the loss of walking abilities before adulthood. Also, about half of LGMD2C/R5 patients have dilated cardiomyopathy, a condition of the heart muscle.
ATA-200 uses a modified, harmless adeno-associated viral vector (AAV) to deliver an engineered version of the SGCG gene to cells and restore the function of the sarcoglycan complex. In animal models, a single administration of the therapy was well tolerated, and was shown to reverse disease symptoms and normalize levels of biomarkers.
The Phase 1b trial expects to recruit six children with LGMD2C/R5, ages 6 to 11, who are able to walk 10 meters (about 33 feet) in less than 15 seconds and rise from a chair with or without arm support. The first group of three patients will receive a dose corresponding to the minimum effective dose used in preclinical studies. ATA-200 will be given by intravenous, or into-the-vein, administration.
Pending a positive review by an independent data safety monitoring board, three additional participants will be treated with a higher dose. After the evaluation period, patients will be followed up for an additional 4.5 years.
We are so grateful for the opportunity to establish a partnership with Atamyo to help facilitate bringing their groundbreaking research to a clinical site in the US for the very first clinical trial for children with LGMD2C/R5. … This is a monumental step for the entire LGMD community.
The trial’s main goal is to assess adverse events up to six months after dosing. Secondary measures include treatment efficacy, pharmacological properties, and whether the treatment triggers an immune response.
“We are so grateful for the opportunity to establish a partnership with Atamyo to help facilitate bringing their groundbreaking research to a clinical site in the US for the very first clinical trial for children with LGMD2C/R5,” said Courtney Dion, co-founder and president of the Dion Foundation, based in Massachusetts.
“This is a monumental step for the entire LGMD community,” Dion added.
ATA-200 was granted orphan medicinal product designation by the European Medicines Agency for LGMD2C/R5. This status aims to encourage the development of therapies for rare diseases, by providing pharmaceutical companies with certain incentives, including protocol assistance for clinical trials and a period of market exclusivity if the medication is ultimately approved.
The Dion Foundation was created in 2023 by Joe and Courtney Dion after two of their three children were diagnosed with LGMD type 2C.
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