MyoKardia Shows Clinical Trial Data for HCM Treatment

Rebekah Harrison

MyoKardia, Inc., announced initial clinical data from two Phase 1 trials of MYK-461, which targets the underlying cause of hypertrophic cardiomyopathy (HCM).

What is hypertrophic cardiomyopathy (HCM)?

Hypertrophic cardiomyopathy (HCM) is a disease in which the myocardium becomes abnormally thick. In a small number of people with HCM, the thickened heart muscle can cause shortness of breath, chest pain or problems in the heart's electrical system, resulting in life-threatening arrhythmias.
In both trials, MYK-461 was well tolerated with dose-proportional pharmacokinetics. MYK-461 has demonstrated clinical proof of mechanism in reducing cardiac muscle contractility, an important biomarker of disease.
In the first-in-human, double-blind, placebo-controlled trial evaluating single oral doses in 48 healthy adult volunteers (36 active, 12 placebo), MyoKardia has completed dosing in six cohorts at doses of up to 48 mg of MYK-461 or matching placebo.
Clinical proof of mechanism was demonstrated at doses of 12 mg and above by dose-dependent pharmacodynamic activity, as assessed by three different echocardiographic biomarkers of contractility. MYK-461 was well tolerated at all dose levels and there were no serious adverse events or clinically meaningful findings in vital signs, electrocardiogram recordings or safety laboratory tests. MYK-461 demonstrated a dose-linear and dose-proportional pharmacokinetic profile, with low inter-subject variability.
MyoKardia also initiated an open label, single ascending dose trial of MYK-461 in adult patients with HCM. Observations from the first two patients dosed at 48 mg were similar to those from healthy volunteers treated at the same dose.
“I am encouraged by the data disclosed today on the demonstration of proof of mechanism for MYK-461 across a range of doses with a favorable safety profile. The approach of addressing the underlying genetic cause of the disease has great potential to make a meaningful difference for patients suffering from this condition,” said Sharlene M. Day, MD, associate professor and director, Program for Inherited Cardiomyopathies at the University of Michigan and a participant in the MYK-461 investigational program.


MyoKardia Provides Update on Two Phase 1 Trials of MYK-461 for the Treatment of Hypertrophic Cardiomyopathy [news release]. San Francisco, CA; MyoKardia, Inc.: October 6, 2015.
Printer Printing...