MyoKardia, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is mavacamten, an orally administered small molecule, which is in Phase III clinical trial that is designed to reduce left ventricular contractility to alleviate the functional
…More consequences and symptoms of obstructive hypertrophic cardiomyopathy (HCM) and prevent or reverse HCM progression, as well as in Phase II clinical trial for non-obstructive HCM. The company also develops MYK-491, an orally-administered small molecule, which is in Phase IIa clinical trial that is designed to restore normal cardiac muscle contractility in the diseased dilated cardiomyopathy (DCM) heart. Its preclinical programs include MYK-224, a HCM-targeting candidate that is designed to reduce excess cardiac contractility and enhance diastolic function; LUS-1, which is used to counteract a muscle abnormality that results in impaired relaxation of the left ventricle; and ACT-1 targeting genetic DCM due to sarcomeric mutations and impaired calcium regulation. The company has a collaboration with 23andMe, Inc., a consumer genetics and research company. Myokardia, Inc. was founded in 2012 and is headquartered in South San Francisco, California.