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 neglected rare cardiovascular diseases. Their 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. Their lead program, mavacamten, is being developed for the potential treatment of hypertrophic cardiomyopathy, or HCM. In HCM, the walls of the heart thicken due to excessive contraction and prevent the left ventricle from expanding, resulting in a reduced pumping capacity. Mavacamten is being studied in four clinical trials in patients with HCM, including a 220-patient pivotal Phase 3 clinical trial known as EXPLORER-HCM in patients with obstructive HCM (oHCM). In 2016, mavacamten was granted Orphan Drug Designation by the U.S. Food and Drug Administration, or the FDA, for the treatment of symptomatic oHCM. They are also studying mavacamten for a second potential indication, symptomatic, non-obstructive HCM (nHCM), in a Phase 2 clinical trial, known as MAVERICK-HCM.