Exhibit 99.1


MyoKardia Reports Fourth Quarter and Full Year 2018 Financial Results


Company to Host Conference Call and Webcast on March 4, 2019 at 8:30 a.m. ET (5:30 a.m. PT)


SOUTH SAN FRANCISCO, Calif., February 28, 2019 – MyoKardia, Inc. (Nasdaq: MYOK), a clinical-stage biopharmaceutical company pioneering a precision medicine approach for the treatment of serious cardiovascular diseases, today reported financial results for the fourth quarter and full year ended December 31, 2018.  


“2018 was MyoKardia’s most significant year of progress towards building a leading cardiovascular company addressing the number one global health burden with novel precision medicines,” said Tassos Gianakakos, Chief Executive Officer.  “Our commitment to a mission-driven, values-based culture continues to be a key differentiator as we build a growing community of employees motivated to change the world for people with serious cardiovascular diseases.  We’ve added incredible people at all levels of the company and strengthened our HCM disease-area leadership.  In 2018, we initiated six clinical trials for mavacamten and MYK-491, including the pivotal Phase 3 EXPLORER-HCM trial, shared important clinical data from both, and unveiled three new therapeutic programs, including MYK-224 which will enter the clinic this year.”  


Gianakakos continued, “As we move into 2019, our pipeline is set up to generate meaningful clinical data across multiple programs, including six-month mavacamten data from our open-label PIONEER-OLE study to be presented at the upcoming American College of Cardiology annual meeting. Over the course of the year, we will report additional 12-month clinical data from PIONEER-OLE and complete enrollment for our Phase 3 EXPLORER-HCM trial, bringing us closer to delivering much-needed new therapies to people with HCM.  Phase 2 data from our MAVERICK study of mavacamten in non-obstructed HCM is expected later this year and will generate important insights towards addressing diastolic heart disease.  We also expect to share Phase 2 data from our MYK-491 program designed to improve systolic function in people with impaired cardiac contractility.”


MyoKardia’s management will conduct a conference call on Monday, March 4 at 8:30 a.m. ET/5:30 a.m. PT to discuss fourth quarter and year-end 2018 financial results.  The call and webcast will also review six-month safety, efficacy and biomarker data from the PIONEER open-label extension (OLE) trial of mavacamten in patients with obstructive HCM.  These data will become available electronically on March 4th and will be presented at the American College of Cardiology’s 68th Annual Scientific Session being held March 16-18 in New Orleans.


Therapeutic Program Highlights


Mavacamten for Hypertrophic Cardiomyopathy (HCM)


In 2018, MyoKardia initiated four new clinical studies of mavacamten for the treatment of hypertrophic cardiomyopathy, including:  the pivotal Phase 3 EXPLORER-HCM trial in patients with obstructive hypertrophic cardiomyopathy (oHCM); the MAVERICK-HCM Phase 2 trial in non-obstructive HCM (nHCM); and two long-term safety studies, PIONEER-OLE and MAVA-LTE.  Data from the MAVA-LTE clinical trial, along with results of the pivotal Phase 3 EXPLORER-HCM trial, are intended to support the registration submission for mavacamten for the treatment of obstructive HCM (oHCM).



In October 2018, MyoKardia reported 12-week data from the PIONEER-OLE study. Interim data for seven patients demonstrated statistically significant reductions in left ventricular outflow tract (LVOT) obstruction compared to baseline following treatment with mavacamten. Each patient’s rate of ejection fraction remained well in the normal range of greater than 50 percent, and there were no significant adverse events reported.  



In November 2018, MyoKardia presented preclinical and clinical evidence of mavacamten’s potential to improve diastolic compliance.  The ability for the heart to fill with blood in between beats, or diastolic relaxation, is typically impaired in HCM.  In a genetic mini-pig model of non-obstructive HCM, mavacamten was shown to decrease filling pressure, while increasing the end diastolic volume of the left ventricle.  An analysis of echocardiographic measures of left ventricular relaxation, pressure and filling from MyoKardia’s Phase 2 PIONEER-HCM clinical trial in oHCM patients also demonstrated a decrease in filling pressure and an

The following information was filed by Myokardia Inc (MYOK) on Thursday, February 28, 2019 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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