Irish biopharmaceutical company Heart Metabolics has begun its Phase IIb trial of perhexiline to treat hypertrophic cardiomyopathy (HCM) and moderate-to-severe heart failure with preserved left ventricular function.

Perhexiline acts as a carnitine palmitoyl transferase (CPT) inhibitor and is indicated to treat HCM symptoms.

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It prevents the uptake and metabolism of fatty acids by the heart, leading the heart to resort to glucose as a fuel source and resulting in an improved myocardial energetic, reducing or normalising the energy imbalance induced by HCM.

The open label, two-period, ascending dose Phase IIb trial is designed to evaluate the efficacy of perhexiline in achieving a baseline change of a functional status measure (VO2 max) in patients with HCM and moderate-to-severe heart failure with preserved left ventricular function.

"Perhexiline is intended to reduce or even normalise the energy imbalance in heart metabolism that is created by the genetic defect found in patients with HCM."

It will enrol approximately 33 subjects who will be administered with repeated doses of perhexiline for 112 days, and enable the selection of a dose regimen to be used in future studies.

The secondary endpoints of the trial are to determine a baseline change on the basis of the Six Minute Walk Test.

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The Ohio State University Wexner Medical Center cardiovascular medicine division director and perhexiline study principal investigator William Abraham said: "Perhexiline is intended to reduce or even normalise the energy imbalance in heart metabolism that is created by the genetic defect found in patients with HCM.

“With advancements in genetic testing and this greater understanding of perhexiline based on recent clinical studies, we can now identify patients likely to positively respond to treatment and exclude patients at risk of adverse side effects."

The previously held Phase IIa trial demonstrated the efficacy of perhexiline, resulting in improved exercise capacity, energetics and quality of life.