A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to restore lost muscle cells in people with Duchenne muscular dystrophy (DMD).
Texas-based IPS Heart announced that it is preparing an investigational new drug application or IND — a formal request to the U.S. Food and Drug Administration (FDA) to begin testing a treatment candidate — for its cell-replacement therapy GIVI-MPC. If cleared by the FDA, the company said it hopes to begin a first-in-human trial of GIVI-MPC in people with DMD sometime in 2027. IPS Heart did not provide details about the study’s expected design.
Rauf Ashraf, CEO of IPS Heart, said the company’s goal is to address the underlying mechanisms of Duchenne.
“Our focus is genuine disease modification in the muscular dystrophies — conditions that require new, healthy muscle,” Ashraf said in a press release providing updates on the company’s experimental therapies.
DMD is a genetic type of muscular dystrophy in which the body produces virtually no dystrophin, a protein that’s needed to maintain muscle health. Without dystrophin, people with DMD experience progressive muscle damage over time, resulting in symptoms such as muscle weakness and mobility limitations.
Problems with the dystrophin protein also cause the related disease Becker muscular dystrophy (BMD), which is marked by low levels and/or abnormal versions of this protein. IPS Heart is developing two therapies for DMD and BMD using its induced pluripotent stem cell (iPSC) platform.
Pluripotent stem cells are primordial cells that can grow into other kinds of cells; they are normally present during early development in the womb, which makes them generally difficult to obtain.
iPSCs, meanwhile, are made by taking easily accessible cells, such as skin or blood cells, and reverse-engineering them to produce stem cells similar to those seen in early human development. These primordial cells can then be grown to produce various types of new, healthy cells — including the muscle cells that are lost in DMD and BMD.
DMD treatment would replace damaged muscle cells
PSC Heart’s iPSC-based therapies essentially aim to replace damaged muscle cells with healthy cells that make functional dystrophin protein. The company is developing two therapies targeting different types of muscle cells. GIVI-MPC is specifically designed to regenerate skeletal muscle, which is the muscle that moves the body around. A second therapy, called ISX9-CPC, aims to replace scar tissue in the cardiac (heart) muscle.
“With ISX9-CPC for the heart and [GIVI-MPC] planned for clinical entry next year for skeletal muscle, we are leveraging the full power of our iPSC platform to deliver meaningful treatment options,” Ashraf said.
GIVI-MPC has been awarded the FDA’s orphan drug status as a treatment for both DMD and BMD. This designation offers extra economic incentives to companies investing in treatments for rare diseases, or those that affect fewer than 200,000 people in the U.S. ISX9-CPC has also been awarded orphan drug designation as a treatment for heart disease in DMD.
Additionally, the FDA granted rare pediatric drug designation to both GIVI-MPC and ISX9-CPC as a treatment of DMD. ISX9-CPC also specifically gained a similar designation for the treatment for heart disease related to problems with the dystrophin protein.
Rare pediatric drug status is given to experimental therapies for rare diseases that manifest in childhood. Similar to orphan drug designation, the rare pediatric drug designation offers extra incentives to promote the development of treatments for rare diseases.
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