MINNEAPOLIS and ST.
PAUL, Minn., July 17,
2024 /PRNewswire/ -- 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.
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content:https://www.prnewswire.com/news-releases/fda-clears-investigational-new-drug-application-for-muscular-dystrophy-treatment-from-university-of-minnesota-startup-myogenica-302199393.html
SOURCE Myogenica Inc.