Trials begin for experimental drug for dilated cardiomyopathy

16th February 2017

Human trials have begun of an experimental drug designed specifically to treat heart contraction problems in dilated cardiomyopathy (DCM).

The drug’s maker, American biopharmaceutical company MyoKardia, says it has begun giving the drug to a group of healthy volunteers.

The drug, called MYK-491, is designed to increase heart muscle contractility in people with DCM. Reduced heart contractility is believed to be the cause of several types of the condition.

This phase 1 trial, a randomised, placebo-controlled study, will assess the safety, tolerability, and the early effects of oral doses of the drug and how it moves in the bodies of healthy people.  Any changes in how the heart pumps will be measured.

 “There are currently no approved therapies that address the underlying biomechanical causes of DCM, which can lead to chronic heart failure that can shorten life,” said Dr Marc Semigran, the chief medical officer of MyoKardia.

There are two parts to the pumping action of the heart. The first part is called diastole, when blood collects in the lower heart chambers (the right and left ventricles) as it is pushed through the tricuspid and mitral valves.

Once the ventricles are filled with blood, the second part of the pumping action begins. The ventricles contract and blood is pushed from the right ventricle into the pulmonary artery and from the left ventricle into the aortic valves. This part is called systole.

Dr Semigan said early research had demonstrated that MYK-491 may increase the heart’s contractility with minimal adverse effects on myocardial diastolic function, which can also be abnormal in DCM patients.

The company is also working on a drug to treat the underlying causes of hypertrophic cardiomyopathy (HCM).  Early trials from this drug, MYK-461, has suggested the drug is well tolerated and has the ability to reduce excessive heart muscle contractility.

MyoKardia says it is using its precision medicine platform to generate a pipeline of therapies for the treatment of the two most common forms of heritable cardiomyopathy— HCM and DCM.

For more details, see here.