Atamyo’s Limb-Girdle Muscular Dystrophy Type 2C/R5 Gene Therapy Cleared for Clinical Trial in Italy and France


In light of the CTA clearance, Atamyo plans to carry out a multicenter, open-label phase 1b clinical trial (NCT05973630).

Sophie Olivier, MD, the chief medical officer of Atamyo

Sophie Olivier, MD

Atamyo Therapeutics’ clinical trial application (CTA) for ATA-200, an investigational adeno-associated virus (AAV) vector-based gene therapy for the treatment of γ-sarcoglycan (SGCG)-related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), was subsequently cleared by the Italian Medicines Agency and then by the French Medicines Agency.1

In light of the CTA clearance, Atamyo plans to carry out a multicenter, open-label phase 1b clinical trial (NCT05973630). The trial, which will take the form of a dose escalation study, is expected to enroll 6 participants in total. According to the page, which was most recently updated on March 7, 2024, the trial is estimated to launch on May 1, 2024.

“We are thrilled to obtain our CTA approval in France and Italy for the devastating LGMD2C/R5 disease affecting primarily a pediatric population and for which there is no approved treatment,” Sophie Olivier, MD, the chief medical officer of Atamyo, said in a statement.1 “Atamyo plans to initiate dosing in patients for ATA-200 in third quarter 2024”.

ATA-200, which will be administered intravenously in the trial, is comprised of an AAV vector that delivers a functional copy of the SGCG gene.2 Atamyo stated that in preclinical mouse model research, the gene therapy was shown to be tolerated and able to correct for symptoms and disease biomarkers.

“LGMD 2C/R5 is a severe muscular dystrophy with an onset in early childhood and loss of ambulation generally occurring before adolescence” principal investigator Giacomo Comi, a full professor of neurology at the University of Milan, added to the statement.1 “It is a great motivation to know that the work we are doing has the potential to make a life-changing difference for the patients affected by this disease.”

Atamyo originally announced having submitted the CTA in September 2023.2 Alongside that announcement, the company also reported that it had received $8.6 million in nondilutive financing from France 2030, a program operated by Bpifrance, to support the program’s funding.

In addition to ATA-200, Atamyo is also developing ATA-100 (previously referred to as GNT0006), an investigational AAV vector-based gene therapy intended to treat fukutin-related protein (FKRP) limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).3 Notably, in November 2023, the phase 1/2 ATA-001-FKRP clinical trial (EudraCT 2021-004276-33, NCT05224505) for ATA-100 was cleared by its data safety monitoring board to begin enrolling patients in its second cohort based on initial positive results from the first cohort. Atamyo reported that the first 3 patients who were treated with the gene therapy in ATA-001 each experienced a “marked” decline in creatine kinase levels. Furthermore, the company noted that improved velocity, which was maintained a year after treatment, the vanishing of symptoms including cramps and myalgia, and improved quality of life were observed among the treated patients.

“The preliminary results from cohort 1 with the first dose tested already show encouraging results from a safety and efficacy perspective,” principal investigator John Vissing, MD, DMSci, a professor of Neurology at the University of Copenhagen, Denmark, and the director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, said in a November 2023 statement.3 “ATA-100 treatment has a life-changing potential in an indication where there is no approved treatment.”

1. Atamyo Therapeutics obtains regulatory authorization in Europe to initiate a clinical trial for ATA-200, its gene therapy to treat limb-girdle muscular dystrophy type 2C/R5. News release. News release. Atamyo Therapeutics. March 26, 2024. Accessed March 29, 2024.
2. ATA-200, AtamyoTherapeutics’ gene therapy to treat limb-girdle muscular dystrophy type 2C/R5, reaches key milestones with the filing of a clinical trial application in Europe and a non-dilutive financing from France 2030 program. News release. Atamyo Therapeutics. September 19, 2023. Accessed September 28, 2023.
3. Girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), presented at ESGCT. News release. Atamyo Therapeutics. October 27, 2023. Accessed November 1, 2023.
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