BRISBANE - MyoKardia, Inc. (Nasdaq: MYOK) today presented clinical and non-clinical data related to mavacamten, MyoKardia's investigative therapeutic in late-stage development for the potential treatment of hypertrophic cardiomyopathy (HCM), at the American Heart Association's Scientific Sessions 2020.

Three poster presentations were made available detailing exploratory analyses from Myokardia's Phase 3 EXPLORER-HCM study of mavacamten for the treatment of obstructive HCM and from the Phase 2 MAVERICK-HCM study of mavacamten in patients with non-obstructive HCM, as well as non-clinical results of a mavacamten surrogate compound in a large animal model.

'These data add detail to the emerging picture of mavacamten's beneficial impact on the HCM heart, including improvements in cardiac pathophysiology, diastolic function and biomarkers of disease progression,' said Jay Edelberg, M.D., Ph.D., MyoKardia's Chief Medical Officer. 'HCM is characterized by the thickening of the heart muscle and constraints on diastolic filling. Having repeatedly demonstrated that mavacamten can have a profound effect on reducing the obstruction of the left ventricular outflow tract in HCM, echocardiography data from our EXPLORER-HCM trial show that in just 30 weeks of treatment, mavacamten is gradually bringing measures of cardiac structure closer to a normal state, improving parameters of diastolic function and reducing biomarkers of disease. We are optimistic that these changes may ultimately point to the benefits of mavacamten treatment in the progression of HCM.'

Mavacamten Favorably Impacts Key Pathophysiologic Processes in Obstructive Hypertrophic Cardiomyopathy: Results From the EXPLORER-HCM Study

Treatment with mavacamten led to statistically significant reductions in left ventricular mass (LVMI), indicating that mavacamten is having an effect on cardiac structure. LVMI has been shown to be a predictor of HCM-related mortality.

Mavacamten treatment improved left ventricular relaxation (which in turn led to improved cardiac filling pressures). Statistically significant improvements (p

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