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FDA Clears Investigational New Drug application for muscular dystrophy treatment

MINNEAPOLIS and ST. PAUL, Minn., July 17, 2024. Today, Myogenica, a University of Minnesota startup company, announced U.S. Food and Drug Administration approval for an Investigational New Drug (IND) 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.

The clinical translation of MyoPAXon is the culmination of years of work by an interdisciplinary research team at the University of Minnesota, encompassing the lab of Dr. Rita Perlingeiro, the Molecular and Cellular Therapeutics cGMP facility, the Center for Translational Medicine, and the Greg Marzolf Jr. Muscular Dystrophy Center. In addition to co-funding the proposed clinical trial, Duchenne UK was instrumental in contributing over $900,000 to fund the IND enabling studies of MyoPAXon led by Perlingeiro. This project also received funding from the Department of Defense, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, MyDirectives, and the Greg Marzolf Jr. Foundation.

Myogenica, co-founded by Perlingeiro, is the exclusive licensee of the intellectual property that underpins MyoPAXon. "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.

The University of Minnesota is an equity holder and is entitled to receive royalties under a license agreement with Myogenica. These interests have been reviewed and managed by the University of Minnesota in accordance with its conflict of interest policies.

SOURCE Myogenica Inc.

Posted: July 2024

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