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Pending positive results from an early-stage trial, a mid-stage study evaluating MyoPAXon’s effect on other targeted muscles and muscle function will be initiated.
Peter Kang, MD, FAAN, FAAP
(Credit: University of Minnesota)
According to a recent announcement, the FDA has granted the approval of Myogenica’s investigational new drug (IND) application to study MyoPAXon, an induced pluripotent stem cell-derived muscle stem cell product to regenerate skeletal muscle. The company noted that a proposed early-stage clinical trial would assess the safety, tolerability, and engraftment of the therapy among patients with Duchenne muscular dystrophy (DMD).1
The pending trial, led by Peter Kang, MD, FAAN, FAAP, a pediatric neuromuscular neurologist and director of the Greg Marzolf Jr. Muscular Dystrophy Center at the University of Minnesota Medical School, plans to administer intra-muscular injections of MyoPAXon in adult patients with nonambulatory DMD. Kang and colleagues in the trial will then monitor any potential site reactions or immune response and investigate the presence of dystrophin-producing myofibers.
"I was thrilled to see this milestone in testing cell therapies for muscular dystrophy in humans. There is enormous potential to provide lasting relief for patients with muscular dystrophy. Our planned initial study will focus on safety considerations, and we will be injecting MyoPAXon into a small foot muscle to assess safety and tolerability of various doses," Kang told NeurologyLive®. "What makes MyoPAXon distinct is that it has the exciting potential for muscle regeneration. Once injected, the MyoPAXon cells can survive indefinitely and thus yield long term beneficial effects."
Myogenica noted in the announcement that Kang and colleagues will initiate mid-stage study targeting other muscles and evaluating muscle function if the proposed trial produces positive findings. Since MyoPAXon is not specific for any genetic mutation, it may have the potential use as a treatment for a range of other muscular dystrophies as well as other muscle degenerative conditions, according to the company.
READ MORE: Gaps in Follow Up and Sustained Clinical Care Observed in Pediatric Muscular Dystrophy
Clinical translation of MyoPAXon is a culmination of years of interdisciplinary teamwork at the University of Minnesota, involving the lab of Rita Perlingeiro, PhD, the Molecular and Cellular Therapeutics cGMP facility, the Center for Translational Medicine, and the Greg Marzolf Jr. Muscular Dystrophy Center. Duchenne UK contributed over $900,000 to fund the IND-enabling studies of MyoPAXon in addition to cofunding the proposed clinical trial. The project also had funding provided by the Department of Defense, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, MyDirectives, and the Greg Marzolf Jr. Foundation.
"Pure excitement and joy. We have worked intensely on this project for the last 7 years so we were overjoyed with the news from the FDA," Perlingeiro, the cofounder of Myogenica and the Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota, told NeurologyLive when asked about her reaction to the news. "We hope MyoPAXon will contribute to new and healthy muscle in DMD, and other types of MD in the near future. The main goal of the current trial is safety, but a secondary outcome is detection of dystrophin to confirm production of new donor-derived healthy myofibers."
"MyoPAXon is an universal allogeneic stem cell product. Therefore, MyoPAXon's application is not restricted to a subset of DMD mutations, which is a major differential from other current investigational therapies for patients with DMD," Perlingeiro added. "Accordingly, MyoPAXon's therapeutic application goes beyond DMD, as it has the potential to contribute to healthy muscle regeneration in other types of muscular dystrophies and muscle degenerative conditions. In addition, differently from gene therapy, MyoPAXon is not limited to one single time injection."
At the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 19-22, in Dallas, Texas, Perlingeiro presented a talk on the potential of using iPS cells in the treatment of muscular dystrophy. In addition to her talk, the session also focused on the use of patient-specific iPS cells to model neuromuscular diseases, with the goal of understanding disease pathogenesis and treatment development. Also, the session emphasized the expansion of bioengineering approaches for skeletal muscle modeling.2 In an interview with CGTLive®, a sister publication to NeurologyLive®, Perlingeiro provided an overview of some of the main highlights addressed. She also spoke about the challenges and unmet needs of this patient population that have started to gain attention, as well as the potential of iPS in regenerative medicine.
