MyoKardia's mavacamten meets in Phase II for HCM

MyoKardia Inc. (NASDAQ:MYOK) reported top-line data from 10 evaluable patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) in the first cohort of the Phase II PIONEER-HCM trial showing that once-daily 10 or 15 mg mavacamten met the primary endpoint of reducing mean post-exercise peak left ventricular outflow tract (LVOT) gradient at week 12 compared to baseline. Specifically, mavacamten reduced the endpoint to 19 mmHg at week 12 from 125 mmHg at baseline (p=0.002).

Mavacamten also met the secondary endpoint of increasing mean peak oxygen consumption at week 12 compared to baseline (24.6 vs. 20.7 mL/kg/min, p=0.004). On an exploratory endpoint, mavacamten improved New York Heart Association (NYHA) functional class by ≥1 class in 7 patients and by ≥2 classes in 2 patients. Mavacamten was generally well tolerated...