MyoKardia, Inc. (Nasdaq:MYOK), a clinical-stage biopharmaceutical
company pioneering a precision medicine approach for the treatment
of heritable cardiovascular diseases, today announced that dosing
has commenced in a Phase 1b single-ascending dose patient study of
its investigational drug candidate MYK-491.
MYK-491 is being developed in an ongoing collaboration between
MyoKardia and Sanofi for the treatment of patients with dilated
cardiomyopathy (DCM). DCM is a form of heart failure in which
the left ventricle is enlarged and weakened and the heart’s
contractility is reduced, resulting in symptoms ranging from
shortness of breath and fatigue to stroke and death. MYK-491
is intended to increase contractility of the heart (systolic
function) with minimal adverse effects on myocardial relaxation
(diastolic function). The Phase 1b randomized, double-blind,
placebo-controlled, cross-over single-ascending dose trial is
enrolling DCM patients with stable heart failure. The trial
will evaluate the safety, tolerability and preliminary
pharmacokinetics and pharmacodynamics of oral doses of
MYK-491. Pharmacodynamic measures will include changes in
established echocardiographic measures of cardiac
contractility. Topline results from the Phase 1b study are
anticipated in the second half of 2018.
“Patients with DCM suffer from debilitating symptoms due to
inadequate cardiac contraction and the consequent insufficient
blood flow characteristic of their disease. There are
currently no approved therapies that directly target the depressed
contractility present in dilated cardiomyopathy,” said Marc
Semigran, M.D., Chief Medical Officer of MyoKardia. “MYK-491
was designed to address the underlying biomechanical cause of DCM.
As we advance into further clinical testing, we look forward
to gaining insights into MYK-491’s potential to increase cardiac
contractility and we are hopeful this drug candidate may restore
the heart’s contractility towards normal function in patients.”
MyoKardia also announced topline results from the company’s
Phase 1 trial of MYK-491 in healthy volunteers. Sixty-four
healthy adults (48 active, 16 placebo) were enrolled and randomized
to receive either single-ascending doses of MYK-491 or a
placebo. MYK-491 was generally well tolerated across the
range of oral doses tested. Adverse events (AE) observed were
benign and transient. The most common AE was headache.
Extra heartbeats were reported in three subjects, possibly related
to cardiac irritability. Evidence of clinical
proof-of-mechanism was observed at higher dose levels in the form
of increased contractility, as measured by echocardiographic
biomarkers. MyoKardia plans to present detailed results from
the healthy volunteer dosing study at a future medical meeting.
“This Phase 1 first-in-human study of MYK-491 in healthy
volunteers has met the core objective of helping us to determine a
starting dose for our Phase 1b trial in dilated cardiomyopathy
patients,” said Lisa Alaimo, MyoKardia’s Vice President, Project
Management and Project Team Leader. “MYK-491 was generally
well tolerated and we are encouraged by the confirmation of
expected cardiac pharmacodynamic effects. Results from our
Phase 1b patient study, plus the results shared today from our
single-ascending dose trial of MYK-491 in healthy volunteers, will
provide us with valuable data to inform future clinical plans.”
“Sanofi remains committed to driving forward
our collaboration with MyoKardia to develop new treatment
options for patients with heritable cardiomyopathies,”
said Anthony Muslin, M.D., Head of Cardiovascular Research at
Sanofi. “We’re excited about the prospects for MYK-491.”
About MYK-491MYK-491 is an oral, small
molecule, allosteric activator of myosin designed to restore the
inadequate output characteristic of a DCM heart by targeting the
biomechanical defects underlying disease and improving cardiac
contractility. Based on preclinical research across multiple
animal models, MYK-491 may hold potential for controlled increases
in the heart’s contractility with minimal impact on relaxation
(diastole). MYK-491 is currently being studied in a Phase 1a
single-ascending dose trial in healthy volunteers and a Phase 1b
single-ascending dose trial in DCM patients. MYK-491 is being
developed in an ongoing collaboration between MyoKardia and
Sanofi.
About Dilated Cardiomyopathy (DCM)DCM is a form
of heart failure that affects about one million people in the
United States. In DCM, the walls of the left ventricle are
thin and over-expanded, leading to diminished contraction and
insufficient blood being pumped by the heart. Diastolic
function, or the heart’s ability to relax and fill with blood, is
also impaired. Typical symptoms include shortness of breath,
fatigue, swelling in the extremities, or an irregular heartbeat.
As the disease progresses, patients become increasingly
debilitated, and may develop heart failure symptoms and severe
complications such as stroke, arrhythmias and death. There is
currently no approved medical therapy that targets the underlying
depressed contractility characteristic of DCM.
About MyoKardiaMyoKardia 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 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 that has been shown to
reduce hypercontractility in early clinical studies and is
currently being studied in the Phase 2 PIONEER-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.
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 clinical and therapeutic potential of MYK-491, the
availability of topline results from the Company’s Phase 1b trial
of MYK-491 in DCM patients, the availability of additional data
from the Company’s Phase 1 single-ascending dose trial of MYK-491
in healthy volunteers, as well as 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 Quarterly Report on Form 10-Q for
the quarter ended September 30, 2017, 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.
Contacts
Beth DelGiacco (Investors)
Stern Investor Relations, Inc.
212-362-1200
beth@sternir.com
Steven Cooper (Media)
Edelman
415-486-3264
steven.cooper@edelman.com
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