MYOKARDIA, INC.
The newly established mavacamten long-term safety registry will utilize the PINNACLE Registry, the largest out-patient cardiovascular registry in the
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
“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
“As we look forward to the potential approval of mavacamten in the
“We look forward to working with
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,
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MyoKardia’s mission is to change the world for people with serious cardiovascular disease through bold and innovative science.
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About Veradigm®
Veradigm is an integrated data platforms and services business unit of
© 2020
Forward-Looking Statements
Statements 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
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Contacts
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650-351-4690
ir@myokardia.com
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
W2O
628-213-3754
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