MyoKardia Announces Positive Results from Low-Dose Cohort of Phase 2 PIONEER-HCM Study of Mavacamten in Symptomatic

MyoKardia (Nasdaq:MYOK), a clinical stage biopharmaceutical company pioneering a precision medicine approach for the treatment of heritable cardiovascular diseases, today announced positive results from the Phase 2 PIONEER-HCM clinical study of the investigational agent mavacamten in symptomatic, obstructive hypertrophic cardiomyopathy (oHCM) patients, including results from a low-dose patient cohort (“Cohort B”), which studied once-daily 2mg and 5mg oral doses of mavacamten.

As quoted in the press release:

The first cohort (“Cohort A”) of the PIONEER-HCM clinical trial studied 10mg, 15mg and 20mg doses and demonstrated a reduction in patients’ left ventricular outflow tract (LVOT) gradient before steady-state mavacamten concentrations were reached.  Cohort B‘s primary goal was to evaluate lower doses (2 mg and 5mg) of mavacamten in patients with oHCM.  Both cohorts of the PIONEER-HCM clinical trial met the primary endpoint of reduction in post-exercise LVOT gradient from baseline to Week 12 with statistical significance (Cohort A p=0.002; Cohort B p=0.020).  Several key secondary endpoints, including improvements in New York Heart Association (NYHA) classification and dyspnea score, also achieved statistical significance within each cohort.

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