Heart Metabolics and FDA Reach Agreement on SPA for Single Phase 3 Trial


Posted: 2015-04-15 in Press Releases

Download PDF

Heart Metabolics and FDA Reach Agreement on Special Protocol Assessment for Single Phase 3 Trial of Perhexiline in Hypertrophic Cardiomyopathy


Company Appoints William Daly as Chief Executive Officer and Gregory M. Ayers, M.D., Ph.D., as Chief Medical Officer


Dublin – April 15, 2015 – Heart Metabolics Limited, a biotechnology company focused on the development of drugs for orphan diseases including hypertrophic cardiomyopathy (HCM), today announced that it has received an agreement letter from the U.S. Food and Drug Administration (FDA) related to the company’s proposed Phase 3 trial Special Protocol Assessment (SPA) request. The FDA has agreed to a single, randomized, pivotal Phase 3 trial of 350 patients evaluating perhexiline for the treatment of moderate-to-severe HCM.


In conjunction with this achievement, William Daly has joined Heart Metabolics as President and Chief Executive Officer and member of the board of directors, and Gregory Ayers, M.D., Ph.D., has joined as Chief Medical Officer.


“Securing this SPA agreement is a key piece of our strategic plan to rapidly advance perhexiline through the final stages of clinical development,” said Corey Goodman, Ph.D., Chair of Heart Metabolics and Managing Partner at venBio. “With a clear path for completing clinical development in the U.S., we have added two talented and experienced leaders to our executive team. I look forward to working with Mr. Daly and Dr. Ayers to advance perhexiline for this orphan disease.”


“Heart Metabolics has successfully reached a critical milestone in which we have secured an agreement with the FDA regarding the SPA for perhexiline; we now plan to start this Phase 3 pivotal trial in the first half of 2016,” said Mr. Daly, Chief Executive Officer of Heart Metabolics and formerly Senior Vice President of Operations and Business Development at Puma Biotechnology. “I look forward to working with the board and management team to initiate this pivotal study and continue to grow Heart Metabolics into one of the premier orphan disease companies.”  


Mr. Daly has more than 20 years of experience in the biotech industry as an industry executive and an investment banker. In addition to his most recent role at Puma Biotechnology, he has worked in business development and operations roles at several companies, including Johnson & Johnson (Ortho Biotech), Allergan and Akebia Therapeutics.


“Perhexiline has the potential to be the first FDA-approved treatment for the 120,000 patients in the United States suffering from the symptoms of heart failure associated with HCM, the most common of all hereditary heart diseases and a leading cause of sudden cardiac death in young adults,” said Dr. Ayers, Chief Medical Officer of Heart Metabolics. “Based on previous clinical data, I believe this drug will be an effective therapy for this rare and debilitating disease.”


Dr. Ayers, a cardiac electrophysiologist, has over 25 years of experience working with biotech start-up companies.  Dr. Ayers is a former venture partner of MPM Capital and has served as a senior executive or board member of several companies focused on cardiovascular diseases, including Hemosense and CryoCor.


About Special Protocol Assessments

A Special Protocol Assessment (SPA) is a process by which the FDA provides an official evaluation and written guidance on a proposed Phase 3 clinical trial design, endpoints and statistical analyses (including trial sample size) that are intended to form the basis for a new drug application.  Final marketing approval depends on the results of efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in the Phase 3 clinical program.


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-acyltransferase (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.  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 cardiac energetic status (myocardial phosphocreatine:ATP ratio), LV diastolic filling at rest and during exercise, peak oxygen consumption, symptoms, and quality of life.


About Heart Metabolics

Heart Metabolics Limited is a biopharmaceutical company developing new treatments for orphan disease including cardio-metabolic diseases. Headquartered in Dublin, Ireland, and founded in 2014, Heart Metabolics Limited is funded by venBio Partners, Seroba Kernel Life Sciences, Brandon Capital Partners (on behalf of AustralianSuper) and AshHill. More information is available at www.heartmetabolics.com.


# # #


For Further Information, Please Contact:


Corporate Contact

Sherry Toney 

+1 (707) 664-9590



Media Contact

Canale Communications

Monica May

(619) 849-5383