MINNEAPOLIS and ST. PAUL, MN — Myogenica, a University of Minnesota startup company, announced U.S. Food and Drug Administration approval for an Investigational New Drug application for MyoPAXon — an induced pluripotent stem cell-derived muscle stem cell product to regenerate skeletal muscle. A pending clinical trial would evaluate the safety, tolerability and engraftment of MyoPAXon in patients with Duchenne muscular dystrophy (DMD).

Dr. Peter Kang, a pediatric neuromuscular neurologist and director of the Greg Marzolf Jr. Muscular Dystrophy Center at the University of Minnesota Medical School, would lead the proposed early-stage clinical trial. Kang and the clinical research team plan to administer intra-muscular injections of MyoPAXon in non-ambulatory adult DMD patients and monitor any potential site reactions or immune response and assess for the presence of dystrophin-producing myofibers.

"We believe stem cells have enormous potential as a tool for muscle regeneration in muscular dystrophy and the timing is perfect to move toward a human clinical trial of this type of therapy in Duchenne muscular dystrophy," said Kang.

If the proposed trial provides positive outcomes, the team will plan on initiating a mid-stage study targeting other muscles and evaluating muscle function. Because MyoPAXon is not specific for any genetic mutation, it has potential use as a treatment for a range of other muscular dystrophies, as well as other muscle degenerative conditions.

"After many years of basic and preclinical work, we are thrilled to finally have the FDA approve an IND application for an iPSC-derived myogenic cell product. This has been a tour de force and we are excited with what the future holds," said Perlingeiro.