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PIONEER-HCM: Mavacamten may benefit in obstructive hypertrophic cardiomyopathy

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Mavacamten improved left ventricular outflow tract gradient and other metrics in patients with symptomatic obstructive hypertrophic cardiomyopathy, according to data from the PIONEER-HCM study presented at the Heart Failure Society of American Annual Scientific Meeting.

In an interview with Cardiology Today, Stephen Heitner, MD, who presented data from 11 patients from the first cohort of the phase 2 study, said therapy for patients with obstructive hypertrophic cardiomyopathy is an unmet need.

“The last development for treatment of patients with obstructive hypertrophic cardiomyopathy was the licensing of a drug in 1973, and that research was done back in the ’60s,” said Heitner, director of the HCM Clinic at Knight Cardiovascular Institute, Oregon Health and Science University. “The reality is that there haven't been any FDA-approved drugs for this particular condition in more than 50 years. That's not necessarily through the fault of us not trying. There have been three clinical trials in the recent past and, unfortunately, those were not successful.”

According to a press release from MyoKardia, the results presented by Heitner showed a meaningful reduction in resting LV outflow tract, and a less pronounced reduction in resting LV ejection fraction, in the first few weeks.

At 12 weeks, resting LV outflow tract was reduced from 68 ± 34.4 mm Hg to 14 ± 24.6 mm Hg and resting LVEF was reduced from 70 ± 7% to 55 ± 13.1%, according to the release.

Heitner also reported a mean time-series plot of dyspnea numerical rating scale of 1.7 ± 1.8 at 12 weeks vs. the baseline of 4.9 ± 1.6 (P = .002).

One patient had recurrent atrial fibrillation and stopped the study drug after 4 weeks, but all other adverse events were mild or moderate, according to the release.

“This particular study represents a renewed hope that we will develop a new medical therapy for these patients suffering from hypertrophic cardiomyopathy,” Heitner told Cardiology Today. – by Dave Quaile

Reference:

Heitner S, et al. Big Trials of the Past Year. Presented at: Heart Failure Society of America Scientific Assembly; Sept. 16-19, 2017; Dallas.

Disclosure: The study was funded by MyoKardia. Heitner reports he receives grant support from Abbott Vascular, Gilead Sciences and MyoKardia.