Pharma Focus Europe

First Clinical Results of ATA-100, a Gene Therapy for Treating Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Shared at ESGCT

Saturday, October 28, 2023

Atamyo Therapeutics, a biotechnology company dedicated to advancing new-generation gene therapies for the treatment of muscular dystrophies and cardiomyopathies, has today unveiled the initial clinical findings from the ongoing ATA-001 Phase 1b/2b clinical trial of ATA-100. This one-time gene therapy is designed to address Fukutin-Related Protein (FKRP) Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Additionally, Atamyo has announced the approval by the Data Safety Monitoring Board (DSMB) to enroll the second dose cohort in the ATA-001 clinical trial.

ATA-100 is currently undergoing evaluation in a multi-center Phase 1b/2b trial spanning Denmark, France, and the United Kingdom, having received clearance for Investigational New Drug (IND) status from the FDA.

The initial results observed in the first cohort are as follows:

  1. No unexpected safety concerns were identified.
  2. Significant reductions in creatine kinase levels were observed in all three patients.
  3. Improved velocity was achieved and sustained for a year.
  4. Symptoms such as cramps and myalgia disappeared, resulting in an improved quality of life.
  5. Centronucleation was corrected, and evidence of transgene expression was noted in the 3-month muscle biopsy.

From the first dose cohort already demonstrate promising outcomes in terms of safety and effectiveness. ATA-100 has the potential to bring about life-altering improvements in a condition for which there are currently no approved treatments."

These initial findings will be presented at two upcoming conferences:

  1. The 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) scheduled for October 24-27, 2023, in Brussels, Belgium. Nicolai Preisler, MD, from Rigshospitalet, Copenhagen, will deliver an oral presentation titled "Gene therapy for LGMDR9: preliminary results of a dose-escalation study" during the Cardiovascular and muscular diseases session on Friday, October 27, from 11:30 AM to 1:00 PM at Le BEL, Tour & Taxis.
  2. The 2023 International Limb-Girdle Muscular Dystrophy Conference, set to take place on October 28-29, 2023, in Washington, DC. John Vissing, MD, from Rigshospitalet, Copenhagen, will present an oral talk titled "Preliminary experiences with Atamyo’s FKRP gene therapy" during the Clinical Trials Updates (Part 1) Session on October 29, from 11:00 AM to 12:10 PM at the Constitution Ballroom, Grand Hyatt.

Atamyo, expressed her excitement, stating, "These initial results, particularly those related to transgene expression in muscle fibers, are truly exhilarating. With the DSMB's clearance to commence the second cohort, we are eagerly anticipating enrolling new patients in Europe and the United States."

LGMD2I/R9 is a rare genetic condition caused by mutations in the gene responsible for producing Fukutin-Related Protein (FKRP). It affects an estimated 5,000 individuals in the United States and Europe. In its most prevalent form, symptoms emerge in late childhood or early adulthood, leading to progressive muscular weakness and eventual loss of the ability to walk. Patients are also at risk of respiratory problems. Currently, there are no curative treatments available for LGMD2I/R9.

ATA-100, a one-time gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene responsible for producing FKRP proteins. This therapy is built upon the research of Atamyo's Chief Scientific Officer, Dr. Isabelle Richard, who serves as Research Director at CNRS and heads the Progressive Muscular Dystrophies Laboratory at Genethon.



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