Discovery Platform

MyoKardia’s precision medicine platform comprises an integrated suite of discovery tools, bringing together extensive knowledge of the genetic drivers of cardiomyopathy, a profound understanding of the biological mechanisms of the cardiac sarcomere and deep experience in developing small molecule therapies

Table of Pipeline of First-in-Class Targeted Therapies


MyoKardia has studied MYK-461 in 86 healthy volunteers and 15 hypertrophic cardiomyopathy (HCM) patients across three Phase 1 studies.

MyoKardia plans to initiate its PIONEER-HCM study of MYK-461 in the second half of this year. The Phase 2 study will focus on symptomatic, obstructive HCM (oHCM), for which the U.S. FDA has granted MYK-461 Orphan Drug Designation.

In addition, the company is developing DCM-1, designed to treat dilated cardiomyopathy (DCM) by establishing normal contractility in a DCM heart. Other product candidates include HCM-2, which is being developed to lower cardiac muscle contractility in HCM patients; and LUS-1 to counteract muscle disruption resulting in impaired relaxation of the heart.

In 2014, MyoKardia forged a strategic worldwide collaboration with Sanofi to discover, develop and advance first-of-its-kind targeted therapeutics for HCM and DCM.