Novo Biosciences Brings Important Milestone in New Drug to Treat Heart Disease and DMD

Novo Biosciences Inc. has achieved several milestones in its mission to market its innovative drug, trodusquamine (MSI-1436), as a potential treatment for regenerative medicine for heart disease and Duchenne muscular dystrophy. . Trodusquemine is a reused drug candidate that has already been shown to be well tolerated by patients.

"Trodusquemin has tremendous potential for treating some of our most devastating diseases," said Kevin Strange, Ph.D., Novo's CEO. "Our recent progress on clinical trials is providing hope to millions of patients facing limited treatment options."

Novo has demonstrated that trodusquamine stimulates the regeneration of cardiac muscle tissue in mice after an artificially induced heart attack. Trodusquemin is the only small molecule known to stimulate the regeneration of the adult mammalian heart. Heart disease is the leading cause of death in the country, but treatment options are limited to efforts to prevent a secondary heart attack and organ transplantation in patients with heart failure.

Voot Yin, Ph.D., senior scientist at Novo, received in 2017 a $ 1.5 million grant for research into small business innovation from the National Heart, Lung and Blood Institute, an institute of the National Institutes of Health (NIH). to study trodusquemin in the pig, an animal model whose heart resembles that of the human being. The pig study is the next crucial step in the adoption of clinical trials of trodusquamine in patients with acute heart attacks. The first results of these studies are "very encouraging", according to Strange.

Novo also demonstrated that trodusquemin dramatically slows the degeneration of skeletal and cardiac muscles 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 weight loss due to degeneration of skeletal, smooth, and cardiac muscles. DMD is irreversible and patients usually die early in adulthood. Because DMD is designated as an orphan or rare disease, the US Food and Drug Administration (FDA) provides drug suppliers with expedited approvals and other incentives.

At a recent pre-investigation meeting on the new drug (PIND) with the FDA, Novo was informed that its studies on the mouse DMD model provided sufficient evidence of the efficacy of Trodusquemine in the slowing of heart and skeletal muscle damage. Strange said, "We are extremely encouraged by this conclusion, and we still have work to do, including developing a dosing schedule for juvenile DMD patients and defining toxicity in juvenile animal models, but the path to Potential clinical trials are now clearly defined. "

Trodusquemin has recently attracted the attention of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), another NIH institution. On February 28, Yin received a $ 100,000 pilot grant to study the effectiveness of trodusquamine as a regenerative medicine treatment for the treatment of diabetic kidney failure. The one-year proof-of-principle grant is funded by the DiaComp) Pilot and Feasibility Program. DiaComp supports R & D to protect and restore the functioning of organs affected by the complications of diabetes.

Novo scientists will study trodusquamin in mice with multiple, severe renal abnormalities that are very similar to human diabetic nephropathy, a complication of diabetes leading to kidney disease. As with heart disease, treatment options for chronic kidney disease are limited. More than 200,000 patients with diabetic kidney failure undergo dialysis or a long-term kidney transplant each year. But dialysis is expensive and time consuming and the demand for donor organs for transplant far exceeds the supply.

The discovery of trodusquemine reflects Novo's innovative research and development strategy, which was the subject of a brand article "A Shot at Regeneration" in the April 2019 issue of Scientific American, the One of the most prestigious media in the world. Scientific American is published in 14 languages ​​and has a combined online and worldwide circulation of nearly 20 million copies.