Exhibit 99.1


MyoKardia Reports Fourth Quarter and Full Year 2016 Financial Results and Operational Progress


Topline Data from Phase 2 PIONEER-HCM Trial of MYK-461 in Symptomatic Obstructive Hypertrophic Cardiomyopathy Expected in Third Quarter of 2017


MYK-491 Dilated Cardiomyopathy Candidate Phase 1 Study of Healthy Volunteers Under Way;

Topline Data Expected in Third Quarter of 2017


Sanofi Payment Received for Global Cardiomyopathy Research Collaboration


Strong Cash Position to Further Advance Programs


SOUTH SAN FRANCISCO, Calif., March 13, 2017 – MyoKardia, Inc. (Nasdaq: MYOK), a clinical stage biopharmaceutical company pioneering a precision medicine approach for the treatment of heritable cardiovascular diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2016.


“The year 2016 saw MyoKardia deepen our leadership, expand our scientific platform and advance our growing clinical pipeline,” said Tassos Gianakakos, chief executive officer. “We enter 2017 with two programs in the clinic aimed at the two most common heritable cardiomyopathies, HCM and DCM, and sufficient cash to fund operations into 2019. Both programs have data reporting out in the third-quarter of this year that we hope will bring us one step closer to helping HCM and DCM patients.”

During 2016, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for MYK-461 for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy (oHCM). Topline data from the PIONEER-HCM trial of MYK-461, and the Company’s Phase 1 study of dilated cardiomyopathy (DCM) candidate MYK-491, are expected in the third quarter of 2017.

“We are proud of the progress MyoKardia made in 2016 in reaching clinical milestones and broadening our pipeline of novel therapies that target the underlying biomechanical defects of the heart muscle,” said Marc Semigran, M.D., chief medical officer. “As these programs advance in the clinic, we continue to execute on our mission to change the world for patients with serious cardiovascular disease through bold and innovative science.”

Upcoming Clinical Milestones


Topline data from the Phase 2 PIONEER trial of MYK-461 in symptomatic oHCM are expected in the third quarter of 2017.


Topline results from the Phase 1 single ascending dose (SAD) study of DCM candidate MYK-491, currently being studied in healthy volunteers, are expected in the third quarter of 2017.


A double-blind, placebo-controlled Phase 2 trial of MYK-461 (named EXPLORER-HCM) in symptomatic oHCM, based on PIONEER data, is planned for the second half of 2017.


The Company plans to expand study of MYK-461 to patients with non-obstructive HCM in a Phase 2 trial planned to start in the second half of 2017.

The following information was filed by Myokardia Inc (MYOK) on Monday, March 13, 2017 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-K Annual Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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