Atamyo Therapeutics Hits Milestones for Limb-Girdle Muscular Dystrophy Programs
The company pointed out that today, September 30, is LGMD Awareness Day.
Atamyo Therapeutics has finished recruiting patients for the dose escalation portion of its phase 1b ATA-001-FKRP clinical trial (NCT05224505) evaluating ATA-100, an investigational adeno-associated virus (AAV) vector-based gene therapy, for fukutin-related protein (FKRP) limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9); in addition, the company has submitted an investigational new drug (IND) application to the FDA for ATA-200, an investigational AAV vector-based gene therapy intended to treat γ-sarcoglycan (SGCG)-related LGMD Type 2C/R5 (LGMD2C/R5).1
“We are thrilled to have completed the recruitment of the dose escalation phase of our phase 1b study of ATA-100,” Sophie Olivier, MD, the chief medical officer of Atamyo, said in a statement.1 “Data of the dose escalation will serve at selecting the dose to carry over in the pivotal phase of the study. We look forward to presenting the preliminary results of the first dose cohort at the forthcoming World Muscle Society (WMS) Congress and at American Society of Gene & Cell Therapy’s Breakthroughs in Muscular Dystrophy Conference, held November 19 to 20, 2024 in Chicago.”
ATA-001-FKRP is being conducted in Denmark, France, and the United Kingdom under clinical trial applications (CTAs) cleared between 2021 and 2022.2-4 The company announced the dosing of the first patient in September 2022 and in September 2023 Atamyo received clearance of an IND application for ATA-100 from the FDA.5,6 In November 2023, ATA-001-FKRP 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.7 Atamyo reported that the first 3 patients who were treated in ATA-001-FKRP 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 first patients dosed with ATA-100 have experienced promising functional results and the gene therapy has been overall well tolerated in all treated patients with no unexpected safety signal,” Stephane Degove, the chief executive officer and cofounder of Atamyo, added to the statement.1 “Updated results of the clinical trial will be featured in an oral presentation at the 29th International Annual Congress of the WMS in Prague, October 8 to 12, 2024.”
Atamyo’s IND for ATA-200 is intended to expand the multicenter, open-label phase 1b clinical trial (NCT05973630) for the gene therapy, which has already been cleared in Italy and France, to the United States.1,8 The company noted alongside the news of the IND submission to the FDA that the agency has granted rare pediatric disease designation to ATA-200. Atamyo additionally stated that it anticipates that dosing of patients in the trial for ATA-200 will begin before the end of 2024.
Notably, the company pointed out that today, September 30, is LGMD Awareness Day. Both LGMD-2I/R9 and LGMD-2C/R5 are rare diseases that currently have no curative treatment options. Currently, treatment for LGMD-2C/R5 is only supportive in nature.
