MyoKardia is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and rare cardiovascular diseases. MyoKardia’s initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction.
MyoKardia has used its precision medicine platform to generate a pipeline of therapeutic programs for the chronic treatment of the two most prevalent forms of heritable cardiomyopathy – hypertrophic cardiomyopathy (HCM), and dilated cardiomyopathy (DCM). MyoKardia’s most advanced product candidate is mavacamten (MYK-461), a novel, oral, allosteric modulator of cardiac myosin that has been shown to reduce hypercontractility in early clinical studies and is currently being studied in the PIONEER-HCM and EXPLORER-HCM clinical trials. MYK-491, MyoKardia’s second product candidate, is designed to increase the overall extent of the heart’s contraction in DCM patients by increasing cardiac contractility.
MyoKardia estimates that out of 6 million heart failure patients in the U.S., 630,000 of those patients suffer from hypertrophic cardiomyopathy and 410,000 of those patients experience the obstructive form of the disease, oHCM. Foresite Capital believes that mavacamten has the potential to capture a large portion of the oHCM market.