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MyoKardia Announces Initiation of Phase 1 Clinical Study of MYK-461 for Hypertrophic Cardiomyopathy

SOUTH SAN FRANCISCO, Calif. – March 3, 2015 – MyoKardia, Inc., a company pioneering the development of precision therapies for genetic heart disease, today announced it has initiated dosing in a Phase 1 clinical trial of MYK-461. MYK-461 is the first ever therapy designed to target the underlying cause of hypertrophic cardiomyopathy (HCM), the most common heritable cardiovascular disease. Patients with HCM are born with a mutation that causes over-contraction of the heart muscle cells, leading to thickening and stiffening of the heart muscle. Patients with HCM live with often debilitating disease symptoms including shortness of breath, chest pain and fainting spells. Complications of HCM include progressive heart failure, abnormal heart rhythms, stroke and sudden cardiac death.

MyoKardia is developing a portfolio of potential treatments for HCM, each of which is designed to specifically target one of the underlying molecular mechanisms causing the disease. MYK- 461 is the first potential treatment in MyoKardia’s HCM portfolio to enter clinical development. MYK-461 has been designed to correct one of the most common molecular mechanisms causing HCM, and the clinical program will evaluate patients whose disease is caused by this specific mechanism, as identified by genetic screening.

“The initiation of MyoKardia’s first clinical program in HCM is an important milestone for patients with this devastating disease and for physicians who care for them,” said Tassos Gianakakos, chief executive officer of MyoKardia. “Our hope is that by targeting the underlying molecular defect causing hyper-contractility, MYK-461 will restore normal heart muscle contraction and relaxation, and reduce or potentially even prevent the downstream consequences of disease. We expect MyoKardia’s targeted approach will allow an earlier assessment of safety and efficacy, and with far fewer patients, as compared to traditional cardiovascular clinical studies.”

In conjunction with the initiation of MYK-461’s Phase 1 study, the company also announced the expansion of its management team with the appointment of Ingrid Boyes to senior vice president, human resources and Steven Chan to vice president, corporate controller.

“Attracting and developing driven leaders to strengthen our culture of pioneering innovation and teamwork is essential to advancing our mission of advancing a pipeline of targeted therapies while strengthening our leadership in precision cardiovascular medicine,” said Mr. Gianakakos. “Ingrid and Steven both share MyoKardia’s passion for helping patients and their proven track records will be instrumental during MyoKardia’s next phase of growth.”

About the MYK-461 Clinical Trial Program

MYK-461 is a first-in-class, oral, selective, small molecule allosteric modulator of cardiac myosin designed to treat patients with HCM caused by one of the most common molecular mechanisms of disease. MyoKardia will use genetic testing to identify patients with this specific disease mechanism.

The Phase 1 clinical trial will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of escalating single oral doses of MYK-461 in healthy volunteers. In parallel, MyoKardia has initiated a supplemental Phase 1 study of MYK-461 in patients with HCM. These studies will assess MYK-461’s therapeutic mechanism by demonstrating reduction in cardiac muscle contractility via echocardiography as evidence of target engagement.

Management Team Appointments

Ingrid Boyes brings to MyoKardia more than 20 years of human resources and senior leadership experience across the insurance, banking and biotech industries. Most recently, Ms. Boyes was vice president and head of human resources for global product development at Roche. In this role, she was responsible for leading the human resources strategy globally for that function. Previously, she was at Genentech, where she held positions of increasing responsibility, including director of corporate staffing and diversity. She was also senior director of human resources for technical operations and prior to that, held a similar role leading human resources for North America commercial operations. Ms. Boyes holds a B.S. from the University of San Francisco and an MBA from Notre Dame de Namur University.

Steven Chan comes to MyoKardia with more than 20 years of financial leadership experience. Before joining MyoKardia, he held senior management positions at Solta Medical, a global medical device company, where he was a vice president and corporate controller, responsible for leading day-to-day company-wide accounting and finance operations. Previously, Mr. Chan was vice president, finance at Moody’s Analytics, worldwide revenue director at Polycom and a regional controller at Logitech. He started his career at KPMG and has experience managing all aspects of the accounting and finance functions at companies in varying stages of growth, including a successful IPO. Mr. Chan is a certified public accountant in California and holds a B.S. in business administration from the Haas School of Business at the University of California, Berkeley.

About Hypertrophic Cardiomyopathy (HCM)

HCM is the most common heritable heart disease, affecting one in 500 people, and is the leading cause of sudden cardiac death in young adults and adolescents. HCM is characterized by hyper- contraction and an abnormal thickening of the left ventricle, the heart’s main pumping chamber. The condition also causes the heart muscle to become stiff, thereby making it difficult for the heart to relax normally and refill with fresh blood between beats, causing shortness of breath.

Available medical treatments for patients suffering from HCM were designed and approved for other illnesses and only treat the symptoms of disease. As their disease progresses, patients with HCM may require invasive procedures including open heart surgery, catheter-based interventions, or heart transplantation. HCM patients are also at risk for progressive heart failure, and rhythm disturbances that can lead to stroke or sudden cardiac death. Approximately one third of patients with HCM suffer from obstruction to the outward flow of blood from the heart, which may be associated with dizziness, fainting or chest pain

About MyoKardia

MyoKardia, Inc. is pioneering a precision medicine approach to treat patients with genetic heart disease. By combining leading-edge cardiovascular genetics with recent advances in heart muscle biochemistry, MyoKardia seeks to make a meaningful difference in the lives of people suffering from these diseases. By leveraging powerful genetic insights MyoKardia intends to classify these heritable cardiovascular diseases into a series of underlying rare genetic disorders, enabling physicians to treat patients based on their unique genetic and clinical profiles. MyoKardia has helped organize an international patient registry, the Sarcomeric Human Cardiomyopathy Registry (SHaRe) to help in this effort. As a result, MyoKardia’s therapeutic candidates have the potential to be developed far more efficiently than traditional cardiovascular drugs – matching the appropriate therapy to each patient. Launched in 2012, MyoKardia was founded by world-class experts in cardiovascular disease, cardiac muscle biology and genetics, and is funded by leading healthcare investor Third Rock Ventures. For more information, please visit www.myokardia.com.

Media Contacts:
Katie Engleman
Pure Communications, Inc.
910-509-3977
katie@purecommunicationsinc.com