 BRISBANE, Calif., Aug. 04, 2020 GLOBE NEWSWIRE -- MyoKardia, Inc. Nasdaq: MYOK, today reported financial results for the second quarter ended June 30, 2020. The second quarter of 2020 was one of incredible progress across our RD pipeline, including the exciting new data from our danicamtiv program highlighting its unique ability to directly activate both the left atrium and the left ventricle, and capped off by the positive results from our pivotal Phase 3 EXPLORER trial. These data, together with a successful financing, position us well in the pursuit of our mission to change the world for the tens of millions of people with serious cardiovascular disease, said Tassos Gianakakos, MyoKardias Chief Executive Officer. We are highly focused on the U.S. regulatory approval and successful launch of mavacamten and continue to develop evidence supporting its potential as a backbone therapy for people with HCM and possibly beyond. Beyond mavacamten and danicamtiv, the application of our RD platform continues to be fruitful and is generating proprietary scientific insights that could lead to several important new therapies.Clinical Program HighlightsMavacamten for Hypertrophic Cardiomyopathy Positive Phase 3 Topline Data from EXPLORER-HCM: MyoKardia announced positive topline data from its Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy HCM. Mavacamten demonstrated a robust treatment effect: the primary and all secondary endpoints were met with statistical significance p0.0006. Mavacamten was well tolerated, and meaningful improvements in symptoms, functional status and quality of life, as well as reduction or elimination in obstruction of the left ventricle, were observed among patients on treatment versus placebo. EXPLORER Data Accepted for Late-Breaker Presentation at ESC 2020: The Phase 3 data from the EXPLORER-HCM clinical trial have been accepted as a late-breaker presentation at the upcoming virtual European Society of Cardiology ESC meeting. Dr. Iacopo Olivotto, from the Careggi University Hospital, Florence, Italy, will present the data on Saturday, August 29, 2020 at 6:30 CET. Breakthrough Therapy Designation Granted by FDA: MyoKardia announced that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to mavacamten for the treatment of symptomatic, obstructive HCM. Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. MyoKardia plans to submit a New Drug Application to the FDA in the first quarter of 2021. Initiated VALOR-HCM Phase 3 Clinical Trial: The Phase 3 VALOR-HCM trial is a randomized, double-blind, placebo-controlled, multicenter study designed to provide direct clinical evidence of mavacamtens ability to improve outcomes for individuals with HCM by reducing the need for invasive septal reduction therapy SRT procedures. VALOR-HCM is being conducted in partnership with Cleveland Clinic C5Research, an academic research organization, with participation from approximately 15 HCM specialty centers with well-recognized expertise in SRT procedures in the U.S.Danicamtiv for Dilated Cardiomyopathy Presented and Published Encouraging Danicamtiv Clinical and Non-clinical Data: In a Phase 2a clinical trial, danicamtiv was generally well-tolerated in patients with stable chronic heart failure and was associated with clinically meaningful improvements in left ventricular LV contractility, including statistically significant increases in LV stroke volume, without impairing the hearts ability to relax and fill. Treatment with danicamtiv also improved left atrial LA volume and function, a new and potentially important finding given LA size is a well-established prognostic factor for atrial fibrillation. Data from nonclinical studies indicate that danicamtiv directly activates the LV and LA, providing mechanistic rationale for the robust changes observed clinically. These data were presented at the European Society of Cardiologys Heart Failure Association HFA Discoveries online event, and published in the European Journal of Heart Failure. Pending circumstances related to the coronavirus pandemic, MyoKardia expects to initiate a Phase 2 study in patients with genetic dilated cardiomyopathy DCM in the second half of 2020 and a Phase 2 study in patients with systolic heart failure and paroxysmal or persistent atrial fibrillation in the first half of 2021.Research Entered into Collaboration with Fulcrum Therapeutics: MyoKardia announced a strategic collaboration and license agreement with Fulcrum Therapeutics. Under the agreement, MyoKardia will access Fulcrums proprietary target discovery engine to identify therapeutics that control the expression of genes known to be underlying drivers of genetic cardiomyopathies. MyoKardia will be responsible for all development and commercialization activities for, and will have global rights to, any potential therapeutics identified through this collaboration. In March 2020, in response to the coronavirus pandemic, MyoKardia paused new patient enrollment in the MAVA-LTE long-term extension study of mavacamten and in the Phase 1 healthy volunteer study of MYK-224. The company has subsequently resumed enrollment in both studies.Recent Corporate Highlights Announced New General Counsel: MyoKardia appointed Denelle J. Waynick as General Counsel and Corporate Secretary. Cynthia Ladd, MyoKardias former General Counsel, will be retiring following a transition period in the second half of 2020. Completed Successful Follow-On Offering: MyoKardia completed a follow-on offering of 6,037,500 shares of its common stock at a public offering price of 105.00 per share, raising approximately 605.0 million in net proceeds. MyoKardia anticipates using net proceeds from the offering to support the regulatory approval process and potential commercial launch of mavacamten for the treatment of obstructive HCM; to fund ongoing and potential later-stage clinical trials of mavacamten in non-obstructive HCM and HFpEF, danicamtiv in targeted segments of systolic heart failure and MYK-224; to advance ACT-1 and LUS-1 into clinical development; to fund ongoing preclinical, discovery and research programs, and for working capital, business development and other general corporate purposes.Second Quarter 2020 Financial ResultsCash Position: Cash, cash equivalents and investments as of June 30, 2020 were 918.1 million, compared to 430.3 million as of December 31, 2019. The increase in MyoKardias cash position is primarily attributable to net proceeds of approximately 605.0 million from MyoKardias follow-on offering of common stock, which priced in May 2020.RD Expenses: Research and development expenses were 44.3 million for the second quarter of 2020, compared to 27.7 million for the same period in 2019. The increase in RD expenses for the second quarter of 2020 was primarily driven by the absence of RD reimbursements from Sanofi, our previous collaboration partner, increased personnel costs including stock-based compensation, as well as increases in RD infrastructure, consulting, and contract manufacturing expenses. GA Expenses: General and administrative expenses were 20.3 million for the three months ended June 30, 2020, compared to 13.9 million for the same period in 2019. The increase in GA expenses for the second quarter of 2020 was primarily driven by an increase in personnel costs including stock-based compensation, as well as increases in professional fees and marketing expenses.Net Loss: Net loss for the second quarter of 2020 was 63.6 million 1.27 per share, compared to a net loss of 38.2 million 0.83 loss per share for the second quarter of 2019.Conference Call and Webcast MyoKardia management will host a conference call and live audio webcast on Tuesday, August 4, 2020, at 4:30 p.m. ET 1:30 p.m. PT to discuss current operations and second quarter 2020 financial results. The call may be accessed by phone by calling 844-494-0193 from the U.S. and Canada or 508-637-5584 internationally and using the conference ID 4340819. The webcast may be accessed live on the Investor Relations section of the companys website at A replay of the webcast will be available on the MyoKardia website for 90 days following the call. About MyoKardia MyoKardia is a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases. The company is pioneering a precision medicine approach to its discovery and development efforts by 1 understanding the biomechanical underpinnings of disease; 2 targeting the proteins that modulate a given condition; 3 identifying patient populations with shared disease characteristics; and 4 applying learnings from research and clinical studies to inform and guide pipeline growth and product advancement. MyoKardias initial focus is on small molecule therapeutics aimed at the proteins of the heart that modulate cardiac muscle contraction to address diseases driven by excessive contraction, impaired relaxation, or insufficient contraction. Among its discoveries are three clinical-stage therapeutics: mavacamten formerly MYK-461; danicamtiv formerly MYK-491 and MYK-224. MyoKardias mission is to change the world for people with serious cardiovascular disease through bold and innovative science. 