Myokardia

MyoKardia’s researchers have uncovered a range of small molecule treatments targeting the muscle proteins of the heart with the aim of rectifying the excessive contraction, impaired relaxation, or insufficient contraction that underlie systolic and diastolic dysfunction. A precision medicine approach is being employed to develop therapeutic candidates for patient groups with common traits, such as causal genetic mutations or disease subtypes. The clinical-stage pipeline comprises mavacamten (formerly 461), which is in Phase 3 and Phase 2 clinical trials for hypertrophic cardiomyopathies (HCM).