AT845 is designed to deliver a healthy copy of the gene that encodes the enzyme acid alpha-glucosidase (GAA) directly to muscle cells.
FORTIS (NCT04174105), which is expected to enrol eight participants, will be conducted at eight sites across the U.S. and in Germany and the U.K, to LOPD patients aged 18 to 80.
The trial is being conducted by Astellas Gene Therapies, the result of the acquisition of Audentes Therapeutics by Astellas Pharma.
“The only approved treatment for LOPD is to replace the enzyme through chronic, repeated injections, and lack of other therapeutic options represents a significant unmet need for these patients,” Nate Bachtell, MD, vice president of clinical development at Astellas Gene Therapies, said in a short video posted on Twitter.
In contrast, AT845 is given as a single dose. Participants in the FORTIS study will receive one IV infusion of either a lower or higher dose of AT845, followed by a 48-week observation period and a five-year follow-up. The trial’s primary outcomes are the safety and tolerability of AT845, as well as the change in GAA protein expression levels and activity from the study’s start to 12 weeks post-treatment.
“We’re grateful for the courageous Pompe patient community, the dedication of the study sites, and the years of commitment by the FORTIS study team, without whom this milestone wouldn’t be possible,” Bachtell added.
Astellas Gene Therapies is now a Gene Therapy Center of Excellence — created following the Astellas acquisition. It aims to advance the development of gene therapies for rare neuromuscular diseases, including Pompe.