SYS-CON MEDIA Authors: Adine Deford, Cynthia Dunlop, Harry Trott, Xenia von Wedel, Peter Silva

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MyoKardia Announces Launch of SHaRe Patient Registry for Genetic Heart Disease in Collaboration with World-Leading Cardiovascular Patient Care Centers

MyoKardia, Inc., a company pioneering the development of precision therapies for genetic heart disease, today announced the launch of the Sarcomeric Human Cardiomyopathy Registry (SHaRe), a multi-center, international repository of clinical data on individuals with genetic heart disease. The registry was developed in collaboration with investigators from seven world-leading cardiovascular centers and currently contains de-identified data on over 5,800 subjects. SHaRe aims to grow into the world’s most robust database of clinical information on genetic heart disease through relationships with additional clinical investigators and cardiovascular centers of excellence.

SHaRe is a first-of-its-kind effort that promises to yield a deeper understanding of genetic heart disease and pave the way for more targeted and effective approaches to treating the disease. Two types of genetic heart disease, hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM), together affect nearly 800,000 people in the United States, but treatment to halt the progression of disease has remained elusive due to the complex and varied nature of these conditions. Mutations in the molecular motor of the heart, the sarcomere, are an important cause of these inherited heart muscle disorders. Patients with HCM and DCM do not all share the same disease-causing genetic mutations, or genotype. There are many different clinical characteristics, or phenotypes, in part driven by their individual genetic makeup. SHaRe will play an important role in helping researchers and clinicians better understand how these genotypes and phenotypes interact, which will ultimately lead to the development of targeted therapies based on the specific disease-causing genetic mutations in individual patients.

“Genetic heart disease can present itself in both children and adults. Moreover, the burden of symptoms and the severity of disease are highly variable,” said Carolyn Ho, M.D., assistant professor of medicine at Harvard Medical School, medical director of the Cardiovascular Genetics Center at Brigham and Women’s Hospital and lead SHaRe investigator. “Our goal in creating SHaRe is to critically advance the understanding of HCM and DCM. By amassing these detailed longitudinal and centralized data, we hope to generate better predictors of risk and disease progression. With this information, we can better understand why some patients do poorly, and as importantly, why some patients do well. By redefining and refining phenotypes and modifiers of outcome in genetic heart disease, we ultimately hope to improve the care of our patients and families.”

The centers of excellence currently participating in SHaRe include Brigham and Women’s Hospital, Boston; University of Michigan Medical Center; Stanford University Medical Center; Boston Children’s Hospital; The Heart Hospital, University College London, U.K.; Florence Centre for Cardiomyopathies, Italy; and Erasmus University Medical Center, Netherlands.

Despite the often devastating effect on families, public awareness and understanding of genetic heart disease remains low. As part of SHaRe, a companion website has been launched to provide patients, families and communities with information about heritable cardiomyopathies, SHaRe centers and investigators, related research activities and other genetic heart disease information.

“Together with our SHaRe collaborators, MyoKardia is committed to helping patients with genetic heart disease and their families,” said Jonathan C. Fox, M.D., Ph.D., chief medical officer of MyoKardia and consulting professor at the Stanford Cardiovascular Institute. “Currently available treatment options for patients with heritable cardiomyopathies are limited and do not target the primary cause of the disease. For these patients, a ‘one-size-fits-all’ approach to therapy simply won’t work. The insights gleaned from this large database of clinical information will allow us to better tailor our programs in HCM and DCM and advance our goal of enabling precision medicines for patients with genetic heart disease.”

MyoKardia is developing a pipeline of novel, small-molecule targeted therapeutics to treat genetic heart disease based on mutations in heart muscle that are known to cause the disease. The company’s current programs are focused on both HCM and DCM, for which no therapeutics have ever been approved.

About Genetic Heart Disease

Hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM) are types of genetic heart disease that are caused by mutations in the protein genes of the sarcomere, the fundamental contractile unit of heart muscle, and passed on in families in an autosomal dominant pattern of inheritance. HCM impacts an estimated one in 500 people in the general population by producing thickening of the heart walls and is recognized as a cause of sudden cardiac death, the most common cause of non-traumatic death in young adults. DCM produces weakening of the heart walls and enlargement of the heart chambers and is estimated to occur in one in 2,500 people in the general population. The prevalence of HCM and DCM, taken together, is estimated at nearly 800,000 people in the United States, affecting both children and adults.

About MyoKardia

MyoKardia, Inc. is dedicated to revolutionizing the treatment of genetic heart diseases through the development of novel, small-molecule targeted therapeutics that address the underlying cause of disease. By combining leading-edge cardiovascular genetics with recent advances in heart muscle biochemistry, MyoKardia seeks to usher in an era of precision medicine that will dramatically improve the treatment of cardiomyopathies and make a meaningful difference in the lives of people with genetic heart disease. Launched in 2012 by a group of researchers widely acknowledged as the world leaders in their fields, MyoKardia is funded by leading life sciences investor Third Rock Ventures. For more information, please visit www.myokardia.com.

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