MyoKardia Announces Appointment of Marc Semigran, M.D., as Chief Medical Officer

Former Medical Director of the Heart Failure and Cardiac Transplant Program at Massachusetts General Hospital to Lead Clinical Development

SOUTH SAN FRANCISCO, Calif., Dec. 01, 2016 -- MyoKardia, Inc. (Nasdaq:MYOK), a clinical stage biopharmaceutical company pioneering a precision medicine approach for the treatment of heritable cardiovascular diseases, today announced that Marc Semigran, M.D., has joined the Company as chief medical officer. In his new position, Dr. Semigran will oversee clinical development and lead MyoKardia’s clinical trial strategy.

“We are excited to welcome Marc to MyoKardia as chief medical officer,” said Tassos Gianakakos, chief executive officer. “His extraordinary medical and scientific expertise and strong relationships in the cardiovascular and clinical communities will be invaluable as MyoKardia pursues its mission to change the world for patients with serious cardiovascular disease through bold and innovative science.”

“Dr. Semigran shares MyoKardia’s values, particularly with respect to Patients First and Passion for Science, and I am confident his leadership will enable MyoKardia to deliver first-ever, potentially transformative therapies for patients with heritable cardiomyopathies,” said Mr. Gianakakos.

Most recently, Dr. Semigran led the Massachusetts General Hospital Heart Failure and Cardiac Transplant Program as section head and medical director, from 2004 to 2016. In addition, he was principal investigator of the Harvard Regional Clinical Center of the National Heart, Lung, and Blood Institute Heart Failure Network. Dr. Semigran has been a member of the internal medicine and cardiology staff of Massachusetts General for more than 25 years and is an associate professor at Harvard Medical School.

“MyoKardia is a great innovator in cardiovascular therapy, and the Company is emblematic of the science-driven, precision medicine approach that will bring about much-needed therapies for patients,” said Dr. Semigran. “I am delighted to have the opportunity to lead MyoKardia’s clinical programs at such a pivotal time for the Company.”

Dr. Semigran has published more than 140 peer-reviewed papers in cardiomyopathy, heart failure and cardiac transplantation, and served as editor for a leading textbook on heart failure. He is a recipient of numerous National Institutes of Health and industry research awards, and has served as principal or co-investigator in several major clinical trials across various therapeutic areas. In addition, he has served in scientific and medical advisory capacities to such companies as GSK, Medtronic plc and Bayer.

Dr. Semigran earned A.B., A.M. and M.D. degrees from Harvard University. He completed his internal medicine residency and cardiology fellowship training at Massachusetts General.

About MyoKardia

MyoKardia is a clinical stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and rare cardiovascular diseases. MyoKardia’s initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. MyoKardia has used its precision medicine platform to generate a pipeline of therapeutic programs for the chronic treatment of the two most prevalent forms of heritable cardiomyopathy—hypertrophic cardiomyopathy, or HCM, and dilated cardiomyopathy, or DCM. MyoKardia’s most advanced product candidate, MYK-461, is an orally-administered small molecule designed to reduce excessive cardiac muscle contractility leading to HCM and has been evaluated in three Phase 1 clinical trials. MyoKardia is now studying MYK-461 in the Phase 2 PIONEER-HCM trial in symptomatic, obstructive HCM, for which the FDA has granted MYK-461 Orphan Drug Designation. MYK-491, the second clinical candidate generated by MyoKardia’s product engine, is designed to increase the overall force of the heart’s contraction in DCM patients by increasing cardiac contractility. MyoKardia intends to initiate a Phase 1 study of MYK-491 in healthy volunteers in the first half of 2017. A cornerstone of the MyoKardia platform is the Sarcomeric Human Cardiomyopathy Registry, or SHaRe, a multi-center, international repository of clinical and laboratory data on individuals and families with genetic heart disease, which MyoKardia helped form in 2014. MyoKardia believes that SHaRe, currently consisting of data from approximately 10,000 individuals, is the world’s largest registry of patients with heritable cardiomyopathies. MyoKardia’s mission is to change the world for patients with serious cardiovascular disease through bold and innovative science. For more information, please visit www.myokardia.com.

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