October 4, 2017 — MyoKardia Inc. recently provided a clinical update on its MYK-491 program for dilated cardiomyopathy.
Earlier this year, MyoKardia initiated a Phase 1 single ascending dose trial of MYK-491 in healthy volunteers. The objectives of this randomized, placebo-controlled trial are to assess safety, tolerability, preliminary pharmacokinetics and pharmacodynamics of MYK-491.
Preliminary safety data available from this ongoing trial show that MYK-491 has been generally safe and well-tolerated in all dose cohorts tested thus far. Based on results observed to date, MyoKardia has elected to include additional dose cohorts in the trial in order to further refine its understanding of MYK-491 pharmacokinetics and pharmacodynamics. Topline data from the 001 trial are now expected to be released by early 2018.
MyoKardia intends to initiate a single ascending dose trial of MYK-491 in symptomatic DCM patients before year-end. The objectives of this randomized, double-blind, placebo-controlled trial are to assess safety, tolerability, preliminary pharmacokinetics and pharmacodynamics of MYK-491 in patients.
MYK-491 is an oral, small molecule, allosteric activator of myosin designed to increase cardiac contractility in a DCM heart. Reduced cardiac contractility, or hypocontractility, is believed to be the underlying cause of DCM and MYK-491 is designed to increase cardiac muscle contractility. Based on preclinical research across multiple animal models, MYK-491 may hold potential for controlled increases in the heart’s contractility with minimal impact on diastole or relaxation. MYK-491 is being studied in a Phase 1 single ascending dose trial in healthy volunteers.
For more information: www.myokardia.com
May 06, 2026 
