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Mavacamten gives good showing in extension study
October 2019
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PARIS & SOUTH SAN FRANCISCO, Calif.—The PIONEER open-label extension study of mavacamten in symptomatic, obstructive hypertrophic cardiomyopathy (HCM) demonstrated a durable safety and efficacy profile for the compound, MyoKardia Inc. reported recently. The study enrolled 13 patients from the Phase 2 PIONEER-HCM study, 12 of which were evaluable at 36 weeks. Once-daily oral doses of mavacamten led to reductions in patients’ resting and provoked left ventricular outflow tract gradient while maintaining a left ventricular ejection fraction about 55 percent at all assessment times through week 36.
 
Dr. June Lee, chief development officer, commented in part that, “Given the chronic and progressive nature of HCM, we are particularly encouraged by the long-term durability of the safety and efficacy profile, as well as the sustained changes in multiple biomarkers of cardiac stress and diastolic filling pressures moving toward normal levels through 36 weeks of mavacamten treatment.”

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