MYOK MyoKardia Inc

MyoKardia, Inc. (NASDAQ: MYOK), and the American College of Cardiology (ACC) announced the establishment of a nationwide registry of hypertrophic cardiomyopathy (HCM), which leverages the ACC’s PINNACLE Cardiovascular Registry operated by Veradigm®, an Allscripts (NASDAQ: MDRX), business unit. The registry will support MyoKardia’s post-marketing safety and effectiveness study (DISCOVER-HCM) of mavacamten, an investigational therapeutic for the treatment of obstructive hypertrophic cardiomyopathy (HCM).

The newly established mavacamten long-term safety registry will utilize the PINNACLE Registry, the largest out-patient cardiovascular registry in the U.S. The PINNACLE Registry was established in 2008 by the ACC and now includes more than 3,600 participating clinical practices, serving to capture and share outcomes data on cardiovascular conditions, including coronary artery disease, hypertension, heart failure and atrial fibrillation.

The DISCOVER (Deliver Insights on Safety in Hypertrophic Cardiomyopathy and ObserVe Endpoints in Real-world)-HCM study will evaluate the long-term safety and effectiveness of mavacamten based on real-world usage, for the treatment of patients with symptomatic, obstructive HCM. Veradigm will be conducting outreach to existing PINNACLE Registry sites, as well as additional HCM specialty treatment centers, for participation in DISCOVER-HCM to ensure that the population of the registry is broadly representative of HCM patients in the US. The DISCOVER-HCM study is expected to begin enrolling patients in the summer of 2021. The registry will also focus on gaining the patient perspective on the benefits of HCM treatments by using patient-focused technologies to learn whether registry participants are experiencing symptomatic changes in managing this serious, chronic illness. Additionally, this observational research effort will serve as the foundation for future global assessments of the benefit-risk of mavacamten in HCM with real-world use.

“Patients with hypertrophic cardiomyopathy have been waiting for a precision therapy like mavacamten,” said James Januzzi, M.D., Hutter Family Professor, Harvard Medical School; Cardiology, Massachusetts General Hospital; Trustee, American College of Cardiology and the DISCOVER-HCM Study Steering Committee Co-Chair.

“The DISCOVER-HCM study will provide crucial long-term safety and activity information about mavacamten, which should greatly add to our understanding of obstructive HCM. We are thrilled to be partnering with MyoKardia on this important effort,” added Adrian Hernandez, M.D., Professor, Vice Dean and Executive Director of Duke Clinical Research Institute and Steering Committee Co-Chair and the Principal Investigator for DISCOVER-HCM.

“As we look forward to the potential approval of mavacamten in the U.S., the establishment of an HCM registry will enable us to collect, analyze and track long-term safety data for our patients, expand our knowledge of the natural history of disease and compare outcomes among patients who are treated with mavacamten and those who are not,” said Jay Edelberg, M.D., Ph.D., MyoKardia’s Chief Medical Officer. “Collaborating with the ACC and Veradigm to utilize the PINNACLE Registry and recruit from the ACC’s vast and diverse network of professional cardiologists was a logical choice as we thought about creating a leading repository of real-world HCM data.”

“We look forward to working with MyoKardia and ACC to hit the ground running in recruiting sites for the prospective DISCOVER-HCM safety and effectiveness study of mavacamtem,” said Tom Langan, Chief Executive Officer of Veradigm.

HCM is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction. HCM is estimated to affect one in every 500 people. The most frequent cause of HCM is mutations in the heart muscle proteins of the sarcomere. In approximately two-thirds of individuals with HCM, the path followed by blood exiting the heart, known as the left ventricular outflow tract (LVOT), becomes obstructed by the enlarged and diseased muscle, restricting the flow of blood from the heart to the rest of the body (obstructive HCM). In other patients, the thickened heart muscle does not block the LVOT, and their disease is driven by diastolic impairment due to the enlarged and stiffened heart muscle (non-obstructive HCM). In individuals with either obstructive or non-obstructive HCM, exertion can result in fatigue or shortness of breath, interfering with a patient’s ability to participate in activities of daily living. HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death.

Mavacamten is a potential first-in-class medicine developed to correct the abnormal function of the HCM heart targeting the cardiac muscle proteins with the aim of inhibiting the hypercontractility, left ventricular hypertrophy and reduced compliance that are characteristic of HCM. Earlier this year, MyoKardia published positive data from the pivotal 3 EXPLORER clinical trial of patients with symptomatic, obstructive HCM. Mavacamten has received orphan drug and breakthrough therapy designations from the U.S. Food and Drug Administration and MyoKardia plans to file a new Drug Application with the agency in the first quarter of 2021.

About MyoKardiaMyoKardia 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. MyoKardia’s 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.

MyoKardia’s mission is to change the world for people with serious cardiovascular disease through bold and innovative science.

About American College of CardiologyThe American College of Cardiology envisions a world where innovation and knowledge optimize cardiovascular care and outcomes. As the professional home for the entire cardiovascular care team, the mission of the College and its 54,000 members is to transform cardiovascular care and to improve heart health. The ACC bestows credentials upon cardiovascular professionals who meet stringent qualifications and leads in the formation of health policy, standards and guidelines. The College also provides professional medical education, disseminates cardiovascular research through its world-renowned JACC Journals, operates national registries to measure and improve care, and offers cardiovascular accreditation to hospitals and institutions. For more, visit acc.org.

About Veradigm®Veradigm is an integrated data platforms and services business unit of Allscripts that combines data-driven clinical insights with actionable tools to help healthcare stakeholders improve the quality, efficiency, and value of healthcare delivery. The company has a long-term strategic partnership with the American College of Cardiology (ACC), which includes the ownership and operation of the PINNACLE and Diabetes Collaborative patient registries. Veradigm works closely with ACC to provide innovative cardiovascular research solutions to Life Sciences companies. Veradigm’s mission is dedicated to simplifying the complicated healthcare system with next-generation healthcare solutions. To learn more, visit www.veradigm.com.

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Forward-Looking StatementsStatements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements regarding the level of support the registry will provide to MyoKardia’s post-marketing safety and effectiveness study (DISCOVER-HCM) of mavacamten, expectations regarding the long-term safety and effectiveness of mavacamten based on real-world usage, for the treatment of patients with symptomatic, obstructive HCM, the timing and ability to ensure the right level of participation, of patients or sites, in DISCOVER-HCM to ensure that the population of the registry is broadly representative of HCM patients in the US, the likelihood that this observational research effort will serve as the foundation for future global assessments of the benefit-risk of mavacamten in HCM with real-world use or the likelihood of approval of mavacamten by the FDA reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, risks associated with the development and regulation of our product candidates, as well as those set forth in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and our other filings with the SEC. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

For more information contact:

Contacts Michelle Corral Executive Director, Corporate Communications and Investor Relations MyoKardia, Inc. 650-351-4690 ir@myokardia.com

Hannah Deresiewicz (investors) Stern Investor Relations, Inc. 212-362-1200 hannah.deresiewicz@sternir.com

Julie Normant (media)W2O628-213-3754jnormart@w2ogroup.com