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MyoKardia Collaborates with the American College of Cardiology and PINNACLE/Veradigm to Launch Patient Registry of Hypertrophic Cardiomyopathy

Wed 11th November, 2020 1:30pm
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BRISBANE, Calif. and WASHINGTON, Nov. 11, 2020 (GLOBE NEWSWIRE) -- 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

“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

“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 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. 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 Cardiology
The 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,

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

© 2020 Allscripts Healthcare, LLC and/or its affiliates. All Rights Reserved.
Allscripts, the Allscripts logo, and other Allscripts marks are trademarks of
Allscripts Healthcare, LLC and/or its affiliates. All other products are
trademarks of their respective holders, all rights reserved. Reference to
these products is not intended to imply affiliation with or sponsorship of
Allscripts Healthcare, LLC and/or its affiliates

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 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:

Michelle Corral 
Executive Director, Corporate Communications and Investor Relations 
MyoKardia, Inc. 

Hannah Deresiewicz (investors) 
Stern Investor Relations, Inc. 

Julie Normant (media)


GlobeNewswire, Inc. 2020
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