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MyoKardia Launches Inaugural MyoSeeds™ Research Grants Program to Advance Independent Research in Heart Disease

Program Intended to Accelerate Innovation in the Biology and Underlying Mechanisms of Cardiomyopathies to help Enable Treatment Breakthroughs

Up to Four, $250,000 Grants Available in Inaugural Round

SOUTH SAN FRANCISCO, September 20, 2018 – MyoKardia, a clinical-stage biopharmaceutical company pioneering precision medicine for the treatment of cardiovascular diseases, today announced the launch of the MyoSeeds™ Research Grants Program, a new initiative to support original, independent research in the biology and underlying mechanisms of cardiomyopathies and precision heart disease treatment with the goal of improving the lives of patients.

The MyoSeeds Research Grants Program will support research focused on basic biology of the heart and cardiomyopathies. Priority research areas for funding will be announced prior to each cycle. Priorities for 2018-2019 Funding Cycle are focused on hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM):

  • Disease risk and progression of idiopathic and genetic/sarcomeric DCM
  • Therapeutic interventions involving protein and gene (dys)regulation causal of heart failure
  • Development of disease models in genetic/sarcomeric DCM
  • Mechanisms of pathogenesis in "genotype-negative" HCM and idiopathic DCM
  • Role of energetics in HCM and DCM

MyoKardia will fund up to four awards under the inaugural MyoSeeds program, with a total investment of up to $1 million in funding over 2018-2019 with maximal annual per-project funding of $250,000 which is renewable. Grant proposals will be reviewed for scientific merit, feasibility and impact of the proposed project, and alignment with the research priorities for the funding cycle.

"MyoKardia is deeply committed to advancing the research, treatment and diagnoses of cardiomyopathies. We believe by building a deep understanding of the biology of cardiomyopathies we can better design therapeutic strategies that address the underlying drivers of disease. We've created the MyoSeeds grant program to support independent research initiatives in areas that we've identified as important and complementary to our internal R&D priorities," said Robert McDowell, PhD., MyoKardia's Chief Scientific Officer. "It is our hope that through MyoSeeds we can bring much needed additional resources to academic researchers and clinical scientists who share our determination to improve the lives of cardiomyopathy patients through groundbreaking cardiovascular science."

Additional details about the program criteria, areas of research and the application may be found here. Pre-application letters of intent are due by October 14, 2018 and grant selection notification and awards will occur by no later than December 31, 2018. MyoKardia plans to host a conference call for interested researchers who wish to learn more about eligibility, potential areas of study and the applications process on September 28, 2018 at 10 a.m. PDT. For details, please email myoseeds@myokardia.com.

MyoKardia's research and drug development efforts are focused on applying a precision medicine approach to the treatment of cardiomyopathies by establishing a deep understanding of the biology of the disease, creating novel drugs that are targeted to that biology and identifying subgroups of patients with shared disease characteristics who are most likely to benefit from a targeted treatment. Currently, more than 1.5 million individuals in the U.S. are affected by HCM or DCM. There are no approved medical therapies that target the underlying defects in contractility characteristic of HCM or DCM.

About MyoKardia

MyoKardia is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and rare cardiovascular diseases. MyoKardia's initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. MyoKardia has used its precision medicine platform to generate a pipeline of therapeutic programs for the chronic treatment of two of the most prevalent forms of heritable cardiomyopathy – hypertrophic cardiomyopathy (HCM), and dilated cardiomyopathy (DCM). MyoKardia's most advanced product candidate is mavacamten (formerly MYK-461), a novel, oral, allosteric modulator of cardiac myosin intended to reduce hypercontractility. Mavacamten is being studied in a pivotal Phase 3 clinical trial, known as EXPLORER-HCM, in patients with symptomatic, obstructive HCM. MyoKardia is also developing mavacamten in a second indication, non-obstructive HCM, in the Phase 2 MAVERICK-HCM clinical trial. MYK-491, MyoKardia's second product candidate, is designed to increase the overall extent of the heart's contraction in DCM patients by increasing cardiac contractility. MyoKardia is currently evaluating MYK-491 in a Phase 1b study in DCM patients. A cornerstone of the MyoKardia platform is the Sarcomeric Human Cardiomyopathy Registry (SHaRe), a multi-center, international repository of clinical and laboratory data on individuals and families with genetic heart disease, which MyoKardia helped form in 2014. MyoKardia's mission is to change the world for patients with serious cardiovascular disease through bold and innovative science.

Contacts:

For questions about the MyoSeeds™ Grants Program, please contact: myoseeds@myokardia.com

Michelle Corral (investors)

Senior Director, Corporate Communications and Investor Relations

MyoKardia, Inc.

650-351-4690

ir@myokardia.com.

Steven Cooper (media)

Edelman

415-486-3264

steven.cooper@edelman.com .