The investigational drug ninerafaxstat may be safe and effective in patients with nonobstructive hypertrophic cardiomyopathy.
Hypertrophic cardiomyopathy makes it more difficult for the heart to adequately pump blood and increases the risk of sudden cardiac death. Although there are currently therapies available for patients with the obstructive subtype of hypertrophic cardiomyopathy, those with the nonobstructive subtype have limited treatment options.
Ninerafaxstat works by partially inhibiting the use of fatty acids and encouraging the use of glucose as fuel.
In the phase II IMPROVE-HCM trial, results of which were presented by Maron et al at the American College of Cardiology (ACC) Annual Scientific Session 2024 and simultaneously published in the Journal of the American College of Cardiology, researchers randomly assigned 67 previously treated patients with nonobstructive hypertrophic cardiomyopathy to receive either ninerafaxstat or placebo. After a follow-up of 12 weeks, there were no statistically significant differences in the rate of adverse events between the two groups. The patients who received ninerafaxstat experienced no adverse events related to low ejection fraction, blood pressure, or heart rate. Further, the patients in the ninerafaxstat group saw reductions in the size of their left atrium as well as improvements in their disease symptoms—including fatigue and chest discomfort—and ventilatory efficiency. Ninerafaxstat was particularly effective among the subset of patients who reported significant symptoms at baseline, in which Kansas City Cardiomyopathy Questionnaire scores measuring quality of life increased by nine points compared with the scores among those in the placebo group.
A larger, phase III trial may be needed to better understand the efficacy of ninerafaxstat in this patient population.
“Our findings provide enthusiasm that a novel drug therapy...may provide [patients with] nonobstructive hypertrophic cardiomyopathy an opportunity to achieve a better quality of life by decreasing symptom burden and improving exercise capacity,” concluded lead study author Martin S. Maron, MD, Director of the Hypertrophic Cardiomyopathy Center and a cardiologist at Lahey Hospital and Medical Center.
