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Press Release

Heart Metabolics Announces Initiation Of Phase 2b Study Of Perhexiline For Hypertrophic Cardiomyopathy

Heart Metabolics
Posted on: 31 Aug 16

DUBLIN, Ireland and SAN FRANCISCO, Aug. 31, 2016 /PRNewswire/ -- Heart Metabolics Limited (Heart Metabolics), a biotechnology company focused on the development of drugs for orphan diseases including hypertrophic cardiomyopathy (HCM), today announced it has initiated dosing in patients for an open-label Phase 2b study of perhexiline in patients with HCM and moderate-to-severe heart failure with preserved left ventricular function. In a previously completed randomized, placebo-controlled, Phase 2a study, statistically significant improvements in exercise capacity, energetics and quality of life were observed.

"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."

"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," said William T. Abraham, MD, FACP, FACC, FAHA, FESC, director of the Division of Cardiovascular Medicine at The Ohio State University Wexner Medical Center and the overall Principal Investigator in the perhexiline study. "Perhexiline is intended to reduce or even normalize 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 Phase 2b is an open label, two-period, ascending dose study designed to evaluate the effect of perhexiline on the change from baseline of a functional status measure (VO2 max) in patients with HCM and moderate-to-severe heart failure with preserved left ventricular function following repeat dosing of perhexiline for 112 days, and will enable the selection of a dose regimen to be used in future studies. Secondary endpoints will include change from baseline in the Six Minute Walk Test. The study will enroll approximately 33 subjects at multiple sites in the United States.

More information is available at (Identifier: NCT02862600)

About Hypertrophic Cardiomyopathy (HCM)

HCM is one of the most common of all hereditary heart diseases and the leading cause of sudden cardiac death in young adults. HCM is a genetic defect of heart muscle that occurs due to deletions in genes that encode key contractile proteins in the heart. As a result, there is enlargement of the heart muscle that causes abnormal cardiac energetics. In later stages of the disease, patients develop debilitating and unrelenting symptoms of heart failure. There are more than 120,000 people in the U.S. with HCM who suffer from moderate-to-severe heart failure symptoms. The current treatment options for patients with this disease are limited, with no FDA-approved drugs.

About Perhexiline

Perhexiline is a carnitine palmitoyl transferase (CPT) inhibitor being developed by Heart Metabolics to treat symptoms in patients with HCM. Perhexiline blocks the uptake and metabolism of fatty acids by the heart, causing the heart to 'switch' to glucose as a fuel source. This results in improved myocardial energetic, reducing or even normalizing the energy imbalance created by the genetic defect that defines HCM. In addition, perhexiline has multiple ion channel effects that limit the potential for destabilizing ventricular arrhythmias. It is believed that perhexiline treatment of symptomatic patients with HCM may delay, prevent or even reverse progression of disease. In a Phase 2 clinical trial, perhexiline significantly improved peak oxygen consumption (VO2 max), cardiac energetic status (myocardial phosphocreatine:ATP ratio), left ventricular diastolic filling at rest and during exercise, symptoms, and quality of life.

About Heart Metabolics

Heart Metabolics is a biopharmaceutical company developing new treatments for orphan diseases including cardio-metabolic diseases. The Company has obtained an Orphan Drug Designation in the U.S. for the use of perhexiline to treat HCM patients with moderate-to-severe symptoms of heart failure. In addition, Heart Metabolics has reached agreement with FDA on a Special Protocol Assessment for a single, pivotal Phase 3 trial in about 350 HCM patients. Headquartered in Dublin, Ireland, and founded in 2014, Heart Metabolics is funded by venBio, Brandon Capital Partners (on behalf of AustralianSuper), Seroba Life Sciences and AshHill Biomedical. More information is available at

Editor's Details

Mike Wood

Last updated on: 31/08/2016

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