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Cardiomyopathies

Benefits of ninerafaxstat in non-obstructive hypertrophic cardiomyopathy

Nature Reviews Cardiology volume 21, page 357 (2024)Cite this article

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In patients with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM), ninerafaxstat is well tolerated and improves exercise performance among those who are most symptomatically limited. This finding from the phase II IMPROVE-HCM trial was presented at ACC.24.

Exercise impairment is potentially caused by energy depletion in the myocardium, which leads to diastolic dysfunction because myocardial relaxation is a heavily ATP-dependent process. Ninerafaxstat partially inhibits fatty acid oxidation and shifts cardiac energy metabolism to glucose oxidation, which requires less oxygen per mole of ATP generated and is therefore more efficient, potentially improving the energy-deficient state in patients with HCM. Given its mechanism of action, ninerafaxstat is categorized as a cardiac mitotrope, a novel class of drugs that target energy homeostasis in the mitochondria.

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  1. Nature Reviews Cardiology http://www.nature.com/nrcardio/

    Gregory B. Lim

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  1. Gregory B. Lim
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Correspondence to Gregory B. Lim.

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Lim, G.B. Benefits of ninerafaxstat in non-obstructive hypertrophic cardiomyopathy. Nat Rev Cardiol 21, 357 (2024). https://doi.org/10.1038/s41569-024-01032-y

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