MyoKardia, Inc. Completes $46 Million Series B Financing

Funding Supports Advancement of Precision Therapies for the Treatment of Genetic Heart Disease

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--MyoKardia, Inc., a company pioneering the development of precision therapies for genetic heart disease, today announced the successful completion of a $46 million Series B financing. Proceeds from the financing will be used to advance and expand the company's portfolio of potential therapies for hypertrophic and dilated cardiomyopathies (HCM and DCM), including its lead drug candidate, MYK-461, which is currently in Phase 1 clinical trials.

"The investment of resources and expertise by our new shareholders helps MyoKardia continue to strengthen its scientific leadership position and advance our therapeutic programs as quickly and responsibly as possible."

The Series B financing included investments from an undisclosed public investment fund, Casdin Capital, Cormorant Asset Management, Perceptive Life Sciences, an affiliate of Cowen Group, and BridgeBio LLC. Existing investor Sanofi also participated in the financing.

"The enthusiasm from the investment community is a reflection of the hard work and dedication of our scientists and employees, founders and collaborators all sharing MyoKardia's vision to transform the lives of patients suffering from these devastating diseases," said Tassos Gianakakos, chief executive officer of MyoKardia. "The investment of resources and expertise by our new shareholders helps MyoKardia continue to strengthen its scientific leadership position and advance our therapeutic programs as quickly and responsibly as possible."

MyoKardia is developing a portfolio of potential treatments for HCM and DCM, each designed to specifically target an underlying molecular mechanism causing disease. MYK-461 is the first potential treatment in MyoKardia's HCM portfolio to enter clinical development. MYK-461 targets one of the most common molecular mechanisms causing HCM, and aims to correct the overcontraction driving the disease process and restore cardiac contractility to normal levels. MyoKardia believes this approach has the potential to stop and potentially reverse disease progression. MYK-461's clinical program will evaluate HCM patients whose disease is caused by this specific mechanism, as identified by genetic screening.

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 by Third Rock Ventures, MyoKardia was founded by world-class experts in cardiovascular disease, cardiac muscle biology and genetics. For more information, please visit www.myokardia.com.

Contacts
Pure Communications
Katie Engleman, 910-509-3977
[email protected]