UPMC to run drug trial

PITTSBURGH — The UPMC Heart and Vascular Institute recently enrolled its first patient in the phase 2 MAVERICK-HCM clinical trial of mavacamten, a new drug in development to treat non-obstructive hypertro­phic cardiomyopathy.

If clinical testing ultimately demonstrates safety and efficacy, mavacamten, manufactured by MyoKardia, will be the first approved drug for the condition that specifically targets an underlying disease pathway.

HCM refers to a rare heart condition that is characterized by thickening of the heart muscle, which makes it harder for the heart to pump blood.

UPMC will enroll several participants into the MAVERICK-HCM trial, all of whom must meet stringent inclusion criteria in order to allow investigators to accurately determine the safety and tolerability of the drug, as well as its impact on functional capacity and quality of life.

Affecting about 1 in 500 people, HCM may manifest in two forms. In HCM’s obstructive form, the en­larged muscle physically blocks blood flow from the left ventricle, but in its non-obstructive form, HCM leaves the heart unable to fill or pump blood effectively without an actual blockage.

Drugs such as beta blockers may provide symptom relief, and some obstructive HCM patients can undergo surgery to cut away the obstructed muscle. However, non-obstructive patients lack this option, and some eventually require a heart transplant.

“Current therapy was never designed to target the underlying cause of HCM, leaving a large symptom burden that goes untreated. An approved drug that targets a fundamental disease pathway at the molecular level would change the way we care for our patients,” Dr. Timothy Wong, MAVERICK-HCM’s primary in­vest­igator at UPMC, said in a statement.

“We’re excited to offer non-obstructive HCM patients the opportunity to participate in this important clinical trial. If mavacamten is ultimately proven safe and effective, we’ll be able to provide patients with newfound hope for more effective management of this condition,” he added.

Thirty centers from across the country will collectively enroll 60 participants, and each will be randomly assigned to receive either a daily oral dose of mavacamten or a placebo for 16 weeks. Participants will be monitored for an additional eight weeks after their dosage is complete.

Preliminary data from the MAVERICK-HCM trial are expected in late 2019. If the results of the MAVERICK-HCM study are encouraging, a larger, more definitive phase 3 clinical trial of mavacamten in patients with non-obstructive HCM would likely need to be conducted.

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