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FDA Clears IND for Myogenica's MyoPAXon in Duchenne Muscular Dystrophy

• Myogenica, a University of Minnesota startup, received FDA clearance for its IND application for MyoPAXon, a stem cell therapy for muscular regeneration. • The planned clinical trial will assess the safety, tolerability, and engraftment of MyoPAXon in adult patients with Duchenne muscular dystrophy (DMD). • The therapy, derived from induced pluripotent stem cells, has the potential to treat various muscular dystrophies due to its non-mutation-specific mechanism. • Duchenne UK contributed over $900,000 to fund the IND enabling studies of MyoPAXon led by Dr. Rita Perlingeiro.

Myogenica, a University of Minnesota startup, has received FDA clearance for its Investigational New Drug (IND) application for MyoPAXon, an induced pluripotent stem cell-derived muscle stem cell product aimed at regenerating skeletal muscle in patients with Duchenne muscular dystrophy (DMD). The approval paves the way for an early-stage clinical trial led by Dr. Peter Kang at the University of Minnesota Medical School.

Clinical Trial Details

The planned clinical trial will evaluate the safety, tolerability, and engraftment of MyoPAXon in non-ambulatory adult DMD patients. The study will involve intra-muscular injections of MyoPAXon, with close monitoring for site reactions, immune responses, and the presence of dystrophin-producing myofibers. Dr. Kang emphasized the potential of stem cells for muscle regeneration in muscular dystrophy, stating, "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."

MyoPAXon's Potential

MyoPAXon's mechanism is not specific to any genetic mutation, offering potential applicability across a range of muscular dystrophies and other muscle degenerative conditions. If the initial trial yields positive results, Myogenica plans to initiate a mid-stage study targeting other muscles and evaluating muscle function.

Development and Funding

The clinical translation of MyoPAXon is the result of collaborative efforts from an interdisciplinary research team at the University of Minnesota, including Dr. Rita Perlingeiro's lab, the Molecular and Cellular Therapeutics cGMP facility, the Center for Translational Medicine, and the Greg Marzolf Jr. Muscular Dystrophy Center. Duchenne UK played a crucial role, contributing over $900,000 to fund the IND-enabling studies led by Perlingeiro. Additional funding was provided by the Department of Defense, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, MyDirectives, and the Greg Marzolf Jr. Foundation.

Company Perspective

Dr. Perlingeiro, co-founder of Myogenica, expressed enthusiasm about the FDA's decision: "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."
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Reference News

[1]
FDA Clears Investigational New Drug application for muscular dystrophy treatment from University of Minnesota startup Myogenica
prnewswire.com · Jul 17, 2024

Myogenica, a University of Minnesota startup, received FDA approval for an IND application for MyoPAXon, a stem cell-der...

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