Cytokinetics trial: New drug for rare, symptomless heart condition shows positive results

James Powel, USA TODAY
Updated ·2 min read
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A new drug to treat a rare heart condition may be set to go before the Food and Drug Administration.

Pharmaceutical company Cytokinetics announced Wednesday that its drug aficamten produced, "statistically significant and clinically meaningful" results in the phase three trial of the drug.

Aficamten is intended to treat hypertrophic cardiomyopathy, a condition that thickens the heart muscle and is present in .2% of the world's population, according to the National Institute of Health.

The company said in its press release that full results of the study would be presented at, "an upcoming medical conference." If the drug does make it to market it would compete against Bristol Meyer's drug Camzyos.

"A therapy like aficamten that improves exercise capacity in a clinically meaningful manner, absent low (left ventricular ejection fraction) events that interrupt treatment, should be a welcome addition for HCM patients as well as the clinicians who treat them," Martin Maron, M.D., Director of the Hypertrophic Cardiomyopathy Center at Lahey Hospital and Medical Center in Burlington, Massachusetts and principal investigator for the trial said.

Cytokinetics stock closed down a quarter of a percent at $83.24 per share Thursday but is still up significantly from Tuesday's close of $45.71.

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This is a picture of the Cytokinetics headquarters in South San Francisco, CA, Sep 21, 2020. Cytokinetics' new trial on a heart drug has had promising results in a late-stage trial.
This is a picture of the Cytokinetics headquarters in South San Francisco, CA, Sep 21, 2020. Cytokinetics' new trial on a heart drug has had promising results in a late-stage trial.

What did the phase three trial find?

Patients in the trial arm saw improvement in exercise tolerance, peak oxygen uptake and "clinically meaningful improvements" in the study's secondary endpoints including participant's Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, according to the release.

The Kansas City Cardiomyopathy Questionnaire Clinical Summary Score is a test that measures, "symptoms, physical and social limitations, and quality of life in patients with heart failure," according to the Journal of American College of Cardiology.

Serious adverse effects occurred in 5.6% of people in the trial arm and 9.3% of people in the placebo arm.

What is hypertrophic cardiomyopathy?

Hypertrophic cardiomyopathy is a condition that causes the heart muscle to thicken and makes it more difficult to pump blood, according to the Mayo Clinic.

The condition is usually caused by genetics and does not cause symptoms in many cases. Symptoms, if they are experienced, can include:

  • Chest pain

  • Fainting

  • Heart murmur

  • Sensation of fast, fluttering or pounding heartbeats (palpitations)

  • Shortness of breath

The Mayo Clinic recommends seeking immediate medical attention if a person is experiencing rapid or irregular heartbeat, difficulty breathing or chest pain.

Loyola Marymount basketball star Hank Gathers was killed by a cardiac arrest during a game in 1990 and his autopsy found that he had hypertrophic cardiomyopathy.

Oklahoma City Thunder draft pick and former Florida Gator and Kansas State Wildcat star Keyonte Johnson was diagnosed with the condition after collapsing in a game in 2020.

This article originally appeared on USA TODAY: Cytokinetics heart drug trial has positive results, could go to FDA