Angina drug to help some people with hypertrophic cardiomyopathy?

2nd September 2016

A biotechnology company working on drugs for hypertrophic cardiomyopathy (HCM) is to begin further trials of the drug perhexiline in patients with the disease.

The drug was developed in the 1970s to treat angina but its popularity waned because of side-effects in some patients.

Heart Metabolics, which has its headquarters in Dublin, announced this week it will be using the drug to treat 33 people with HCM and moderate-to-severe heart failure with preserved left ventricular function. In an earlier study, the company reported statistically significant improvements in exercise capacity, energetics and quality of life.

"This study represents an important step in the clinical pathway we have designed for perhexiline, and we expect it will once again demonstrate the compound's ability to safely improve the functional status of patients with HCM, and help identify optimal dosing levels," said William Daly, president and chief executive officer of Heart Metabolics.

"Patients with symptomatic HCM often suffer from chronic fatigue, have a poor quality of life and have an increased risk of heart failure. There is currently no approved treatment for HCM, so there is a clear need for a new therapy."

Dr William Abraham, director of cardiovascular medicine at the Ohio State University Wexner Medical Centre and the principal investigator in the perhexiline study, said: "In recent years, clinical studies of perhexiline have shown promising results in patients with HCM, with statistically significant improvements in exercise capacity and cardiac energetics, while also providing symptom relief.

"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 new study will look at repeat dosing of perhexiline for 112 days, and will enable the selection of a dose regimen to be used in future studies. The study will enroll patients at various centres in the United States.

Cardiomyopathy support nurse Robert Hall said: “Previous studies have been undertaken to look at the effectiveness of perhexiline in adjusting the heart’s metabolism. It will be interesting to see the results of this study, particularly focussing on hypertrophic cardiomyopathy.’


More information about the study is available at (Identifier: NCT02862600)