The director of the Division of Neuromuscular Diseases at UC Irvine discussed the positive data, noting that the treatment was “incredibly safe.” [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
“FORTIS is a muscle-directed approach, where the enzyme is going to be produced locally in the muscle, it will have steady levels, and hopefully, it will do a better job of clearing out glycogen and stabilizing the disease.”
Positive interim safety data from the phase 1/2 FORTIS study (NCT04174105) of AT845 in adults with late-onset Pompe disease (LOPD) were presented at the 18th Annual WORLDSymposium, February 7-11, 2022, in San Diego, California, by Astellas Pharma. The investigational adeno-associated gene replacement therapy delivers a functional GAA gene to express acid α glucosidase directly into muscle cells.1
To better understand findings, we sat down with Tahseen Mozaffar, MD, FAAN, director, UC Irvine-MDA ALS and Neuromuscular Center, director, Division of Neuromuscular Diseases, Neurology School of Medicine, and professor of neurology, pathology, and orthopaedic surgery, University of California, Irvine. In conversation with NeurologyLive®, Mozaffar outlined the importance of AT845 as a treatment in that it offers an alternative to enzyme replacement therapy, which is currently the only approved therapy for treatment of Pompe disease and requires biweekly IV infusions.
Trial investigators have dosed 4 patients thus far in the FORTIS study, with the intention to enroll approximately 8 to 12 patients, Mozaffar said. In those that have been dosed, the treatment was “very well tolerated,” he added, noting that there were no major treatment-related adverse events or treatment-emergent adverse events. One patient did report abnormally high liver function tests, specifically the transaminases, but the patient responded to steroid therapy and levels were lowered, he said.