Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

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Novo Biosciences Inc., has achieved several major milestones in its mission of bringing its breakthrough drug candidate, trodusquemine (aka MSI-1436), to market as a potential regenerative medicine treatment for heart disease and Duchenne muscular dystrophy (DMD). Trodusquemine is a repurposed drug candidate that has already been shown to be well tolerated by patients.
"Trodusquemine holds significant potential for the treatment of some of our most devastating diseases," said Kevin Strange, Ph.D., Novo's CEO. "Our recent progress on the path toward clinical trials gives hope to the millions of patients who face limited treatment options."
Novo has demonstrated that trodusquemine stimulates the regeneration of heart muscle tissue in mice after an artificially induced heart attack. Trodusquemine is the only small molecule known to stimulate regeneration of the adult mammalian heart. Heart disease is the nation's leading cause of death, but treatment options are limited to efforts to prevent a secondary heart attack and to organ transplantation for patients who have suffered heart failure.
Voot Yin, Ph.D., Novo's chief scientific officer, was awarded a two-year, $1.5 million Small Business Innovation Research grant in 2017 from the National Heart, Lung and Blood Institute, an institute of the National Institutes of Health (NIH), to study trodusquemine in the pig, the animal model whose heart most closely resembles that of a human. The pig study is the critical next step in moving trodusquemine into clinical trials in patients who have suffered an acute heart attack. Early results from these studies are "very encouraging," according to Strange.
Novo has also demonstrated that trodusquemine strikingly slows heart and skeletal muscle degeneration in a mouse DMD model. DMD is a neuromuscular disease caused by mutations in the dystrophin gene. It is characterized by rapidly progressing muscle weakness and wasting due to degeneration of skeletal, smooth and cardiac muscle. DMD is irreversible and patients typically die in early adulthood. Because DMD is designated an orphan, or rare, disease, the U.S. Food and Drug Administration (FDA) offers fast-tracked approvals and other incentives to drug developers.
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