MyoKardia’s positive Phase 2 results published in Annals of Internal Medicine


MyoKardia’s (NASDAQ:MYOK) positive Phase 2 trial results of mavacamten for the treatment of obstructive hypertrophic cardiomyopathy (HCM) were recently published in the peer-reviewed journal, Annals of Internal Medicine.

The most common genetic cause of heart disease, HCM is characterized by thickening of the walls of the heart, which reduces the heart’s pumping capacity. In approximately two-thirds of HCM patients, the left ventricular outflow tract (LVOT) becomes obstructed, putting patients at increased risk of severe heart failure and death.

The Phase 2 trial demonstrated that mavacamten – an allosteric modulator of cardiac myosin – could lower the LVOT pressure gradient to levels below the guideline-based definition of obstruction, or those used to recommend invasive intervention.

“The consistency and the magnitude of the effects observed from mavacamten treatment in addressing obstructive HCM patients’ LVOT obstruction, as well as improving symptoms and functional capacity, are impressive, and we have continued to see these effects – as well as a positive safety profile – in the ongoing open-label extension study of PIONEER patients. This is highly encouraging,” Dr. Stephen Heitner, the publication’s lead author, said in a statement.

The article, entitled “Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy,” can be accessed here.

Abby Hardy