Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, announced the dosing with ATA-100 of a first patient in a phase 1/2 clinical study in FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
“This is an exciting milestone for our company but most importantly, if this clinical trial is successful, it could have a life-changing impact for patients affected by LGMD-R9,” said Stephane Degove, Chief Executive Officer and Co-Founder of Atamyo Therapeutics.
This clinical trial (EudraCT 2021-004276-33, NCT05224505) is a multicenter, Phase 1/2 study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-100, a single-dose Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.
This study will consist of 2 phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2).
“LGMD-R9 is a severe progressive and debilitating disease with no approved treatment,” said Pr. John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial. “This experimental treatment represents a new hope for the patients. It is a great motivation to know that the work we are doing here has the potential to make a life-changing difference.”
“After the first patient dosed in Copenhagen, we are now expecting recruitments at the two other approved clinical sites (Paris, FR, and Newcastle, UK) to complete enrollment of the dose escalation phase (Stage 1) of the study. For Stage 2 (after dose selection), we plan to open additional clinical sites in Europe and in the United States,” said Dr. Sophie Olivier, Chief Medical Officer of Atamyo.