MyoKardia Reports Positive Data from Phase 1b Single-Ascending Dose Clinical Trial of MYK-491

MyoKardia (Nasdaq:MYOK), a clinical-stage biopharmaceutical company pioneering precision medicine for the treatment of cardiovascular diseases, today announced topline results from the Phase 1b single-ascending dose study of MYK-491.  MYK-491 was generally well-tolerated and increases in cardiac contractility were observed.

As quoted in the press release:

“Patients with systolic heart failure may suffer from symptoms such as shortness of breath and fatigue that can progress to frequent hospitalizations and death.  By increasing the heart’s contractility without detracting from the heart’s ability to fill, we believe MYK-491 could potentially make a meaningful difference in patients’ lives,” said Marc Semigran, MyoKardia’s Chief Medical Officer.  “Topline data from single doses of MYK-491 are encouraging. Cardiac contractility increased across a number of standard echocardiographic parameters, with little discernible impact on measures of diastolic relaxation and MYK-491 was generally well tolerated.  Our Phase 2a multiple-ascending dose trial of MYK-491 is now underway, and we look forward to results from that study in late 2019.”

The Phase 1b clinical trial evaluated the safety, tolerability and preliminary pharmacokinetics and pharmacodynamics of single ascending doses of MYK-491.  Eight patients with stable heart failure were enrolled and randomized to receive a single dose of either MYK-491 or placebo, after which patients underwent extended observation, followed by a washout period.  This process was repeated until each patient had received at least three doses (MYK-491 or placebo).

Click here to read the full press release.