Exhibit 99.1

 

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MyoKardia Reports Fourth Quarter and 2015 Financial Results and Key Milestones

- Lead product candidate demonstrates clinical proof of mechanism in hypertrophic cardiomyopathy (HCM) patients; Phase 2 planned for second half of 2016

-Growing evidence in nonclinical studies that MYK-461 reduces left ventricular outflow tract obstruction, an important driver of clinical outcomes

-Development candidate identified for dilated cardiomyopathy (DCM) program; Phase 1 planned for first half of 2017

SOUTH SAN FRANCISCO, Calif., March 18, 2016 – 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, 2015.

“MyoKardia made tremendous progress in the fourth quarter and throughout 2015, including release of the first clinical data showing proof of mechanism for MYK-461, our most advanced product candidate, in hypertrophic cardiomyopathy (HCM) patients,” said Tassos Gianakakos, chief executive officer. “We believe that our precision medicine approach to discovery and development will continue to generate significant disease insights as we pursue our mission to improve the lives of patients with heritable cardiovascular diseases.”

“In recent months we completed enrollment of our single-ascending dose (SAD) clinical trial in HCM patients; executed our IPO; strengthened our board, executive team and scientific leadership; and delivered the first findings from the Sarcomeric Human Cardiomyopathy Registry (SHaRe), a multi-center, international repository of clinical data on individuals with genetic heart disease,” said Mr. Gianakakos.

“In 2016, we’re seeing incredible momentum in clinical progress, including data supporting the potential of MYK-461 in treatment of symptomatic obstructive hypertrophic cardiomyopathy, a subset of HCM for which there is no approved therapeutic treatment, and for which surgical interventions are often indicated,” said Mr. Gianakakos. “This is an important and promising step toward filling a critical, unmet need for patients and families.”

Key Milestones:

 

    Data from two Phase 1 trials of MYK-461 demonstrate clinical proof of mechanism in both HCM patients and healthy volunteers in reducing cardiac muscle contractility, an important biomarker of HCM. In these studies, MYK-461 was well tolerated with dose-proportional pharmacokinetics, laying the foundation for its continued development for the treatment of HCM.

 

    Publication in the February 5, 2016 issue of the basic research journal Science demonstrates ability of MYK-461 to both prevent and reverse disease in multiple genetic mouse models of HCM.


The following information was filed by Myokardia Inc (MYOK) on Friday, March 18, 2016 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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