MYOK - Unproven Upside Potential For Myokardia's Mavacamten

Hypertrophic cardiomyopathy ("HCM") is a genetic heart disease mostly caused by mutations in one of the many sarcomeric genes. The most prevalent mutations include genes encoding myosin heavy chain (~30% of the HCM patients), myosin-binding protein C (~20% of the HCM patients), and cardiac troponin T (~20%). The exact mechanism of how these mutations lead to HCM still remains unclear, but pathological pathways may include increased sarcomeric contractility, altered calcium cycling and sarcomeric calcium sensitivity, disturbed biomechanical stress sensing, and impaired cardiac energy homeostasis. Those impacted pathways lead to establishment of clinical manifestation including left