Atamyo Therapeutics has received regulatory clearance in Europe to initiate a Phase Ib clinical trial for its gene therapy, ATA-200, aimed at treating limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a rare genetic disease.
The Italian Medicines Agency (AIFA) and the French Medicines Agency (ANSM) have granted approval for the clinical trial application (CTA), marking a significant step in addressing a rare genetic disease.
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The multicentre, open-label, dose-escalation study will assess the safety, pharmacodynamics, efficacy, and immunogenicity of a single-dose intravenous administration of ATA-200 in paediatric patients.
This gene therapy involves delivering a normal copy of the gene for the production of the g-sarcoglycan protein.
In preclinical models, ATA-200 administered as a single systemic injection has shown promise with its tolerability and the ability to correct symptoms and biomarkers of the pathology.
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The European Medicines Agency (EMA) has also recognised the potential of ATA-200 by granting it orphan medicinal product designation.
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By GlobalDataAtamyo Therapeutics chief medical officer Dr Sophie Olivier said: “We are thrilled to obtain our CTA approval in France and Italy for the devastating LGMD2C/R5 disease affecting primarily a paediatric population and for which there is no approved treatment.
“Atamyo plans to initiate dosing in patients for ATA-200 in the third quarter of 2024.”
The company’s portfolio extends beyond ATA-200 and is currently progressing with a clinical trial of another gene therapy, ATA-100, to treat LGMD2I/R9, which involves deficiencies in the FKRP protein.
In September 2022, the company announced the dosing of the first subject in a Phase I/II trial of ATA-100 to treat LGMD2I/R9.
In addition, Atamyo is conducting investigational new drug application (IND)-enabling studies of this gene therapy for LGMD2A/R1, associated with deficiencies in the calpain protein.
Atamyo Therapeutics CEO Stéphane Degove said: “This is a significant milestone for LGMD-2C/R5 patients and Atamyo, as ATA-200 is the first treatment targeting LGMD-2C/R5 to enter clinical trials.
“With the ongoing clinical trial of ATA-100 in LGMD2I/R9, the initiation of the clinical program for ATA-200 confirms our unique capabilities in bringing to patients suffering from limb-girdle muscular dystrophies a new generation of safe and effective gene therapies.”
