MyoKardia Announces Receipt of Milestone Payment from Sanofi for DCM Candidate MYK-491
December 02 2016 - 8:00AM
$25 Million Milestone Reflects Continued
Momentum in Ongoing Collaboration
MyoKardia, Inc. (Nasdaq:MYOK), a clinical stage biopharmaceutical
company pioneering a precision medicine approach for the treatment
of heritable cardiovascular diseases, announced today that the
Company has received a $25 million milestone payment in accordance
with the research agreement between MyoKardia and Sanofi
established in August 2014 for the filing of an Investigational New
Drug (IND) application with the U.S. Food and Drug Administration
(FDA) for MYK-491 in dilated cardiomyopathy (DCM).
“We are pleased to be moving into the clinic in the first half
of 2017 with the second product candidate from MyoKardia’s
precision medicine platform,” said Robert McDowell, Ph.D., senior
vice president, drug discovery. “Both the Company’s initial product
candidate for HCM, now in Phase 2, and our investigational therapy
for DCM may correct the inappropriate power output caused by
sarcomere mutations that is an underlying driver of disease
progression.”
“Our therapeutic hypothesis is that MYK-491 may increase muscle
contraction to restore cardiac output,” added Dr. McDowell.
“Further, based on preclinical research across multiple animal
models, we believe it holds potential for controlled increases in
the heart’s contractility with minimal impact on diastole or
relaxation.”
Topline results from the planned Phase 1 single ascending dose
study of MYK-491 in healthy volunteers are expected in the third
quarter of 2017.
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 the two most
prevalent forms of heritable cardiomyopathy—hypertrophic
cardiomyopathy, or HCM, and dilated cardiomyopathy, or DCM.
MyoKardia’s most advanced product candidate, MYK-461, is an
orally-administered small molecule designed to reduce excessive
cardiac muscle contractility leading to HCM and has been evaluated
in three Phase 1 clinical trials. MyoKardia is now studying MYK-461
in the Phase 2 PIONEER-HCM trial in symptomatic, obstructive HCM
(oHCM), a subset of HCM. In April 2016, the U.S. Food and Drug
Administration (FDA) granted MYK-461 Orphan Drug Designation for
the treatment of symptomatic oHCM. MYK-491, the second clinical
candidate generated by MyoKardia’s product engine, is designed to
increase the overall force of the heart’s contraction in DCM
patients by increasing cardiac contractility. MyoKardia intends to
initiate a Phase 1 study of MYK-491 in healthy volunteers in the
first half of 2017. A cornerstone of the MyoKardia platform is the
Sarcomeric Human Cardiomyopathy Registry, or 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 believes that SHaRe,
currently consisting of data from approximately 10,000 individuals,
is the world’s largest registry of patients with heritable
cardiomyopathies. MyoKardia’s mission is to change the world for
patients with serious cardiovascular disease through bold and
innovative science. For more information, please visit
www.myokardia.com.
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 clinical and therapeutic
potential of MYK-491, the Company's ability to initiate Phase 1
clinical development of MYK-491 and generate topline data from its
planned Phase 1 single ascending dose study of MYK-491 in healthy
volunteers, and the timing of these events, 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 Annual Report on Form 10-K for the fiscal
year ended December 31, 2015, our Quarterly Report on Form 10-Q for
the quarter ended September 30, 2016, 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.
Investor Contact:
Beth DelGiacco
Stern Investor Relations, Inc.
212-362-1200
beth@sternir.com
Media Contact:
Steven Cooper
Edelman
415-486-3264
steven.cooper@edelman.com
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