MyoKardia, Inc., a clinical stage biopharmaceutical company pioneering a precision medicine approach for the treatment of heritable cardiovascular diseases, today announced the publication of an article in the leading medical journal Science. The article demonstrates the ability of MYK-461, the company’s lead drug candidate, to prevent and reverse development of disease in multiple genetic mouse models of hypertrophic cardiomyopathy (HCM). The published research represents the product of collaboration among scientists from MyoKardia, Harvard Medical School, the University of Colorado and Stanford University. These data add to a growing body of laboratory and clinical research demonstrating the potential of MYK-461 as an important and novel approach to treating HCM.
The study, titled “A Small-Molecule Inhibitor of Sarcomere Contractility Suppresses Hypertrophic Cardiomyopathy in Mice,” will be published in the Feb. 5 issue of the journal Science.
“I am encouraged by these data that illustrate MYK-461’s ability to effectively reduce the consequences of HCM mutations at the biochemical, cellular and whole animal levels,” said Jonathan Fox, M.D., Ph.D., chief medical officer of MyoKardia. “Translation of these findings from mouse to human could offer great potential to improve the lives of patients living with this devastating disease.”