Heart Metabolics Announces Initiation Of Phase 2b Study Of Perhexiline For Hypertrophic Cardiomyopathy
DUBLIN, Ireland and SAN FRANCISCO, 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.
Source: Heart Metabolics
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