MyoKardia, Inc. (Nasdaq:MYOK), a clinical-stage biopharmaceutical
company pioneering a precision medicine approach for the treatment
of heritable cardiovascular diseases, today announced that the
first patient has been dosed in the company’s Phase 3 EXPLORER-HCM
clinical trial. The pivotal study is intended to evaluate the
efficacy and safety of mavacamten for the treatment of symptomatic
obstructive hypertrophic cardiomyopathy (oHCM). MyoKardia
expects to report data from the Phase 3 trial in the second half of
2020.
“The dosing of the first patient in our Phase 3 EXPLORER-HCM
clinical trial is a significant step towards achieving our mission
of bringing innovative medicines to patients with serious
cardiovascular diseases,” said Tassos Gianakakos, MyoKardia’s Chief
Executive Officer. “Based on the data generated to date, and
our thoughtfully designed study measuring improvements in both
symptoms and function, we are hopeful EXPLORER will demonstrate
mavacamten’s potential to dramatically alter the course of oHCM and
transform the lives of people affected by this condition.”
EXPLORER-HCM is a multi-national randomized double-blind study
that will enroll approximately 220 patients with symptomatic
oHCM. Patients will be randomized on a 1:1 basis to receive
either mavacamten or placebo for a 30-week treatment period.
The EXPLORER-HCM trial design incorporates individualized dosing,
including two dose adjustments during the 30-week treatment period
based on measurements of provoked left ventricular outflow tract
(LVOT) gradient. All assessments and dose adjustments will be
conducted in a double-blinded fashion. Patients will be
allowed to maintain their HCM-related background medications for
the duration of the EXPLORER-HCM Phase 3 trial, including beta
blockers or calcium channel blockers. An independent data
monitoring committee (IDMC) has been established to monitor safety
throughout the study.
The primary endpoint is clinical response, defined as either 1)
an improvement of at least 1.5 mL/kg/min in peak oxygen consumption
(VO2) accompanied by an improvement from baseline of at least one
New York Heart Association (NYHA) functional class or 2) an
improvement from baseline of 3.0 mL/kg/min or greater in peak VO2
without worsening in NYHA functional class. Secondary
endpoints in the Phase 3 EXPLORER-HCM trial will include the
average changes from baseline in post-exercise peak LVOT gradient,
NYHA functional class, and peak VO2. Exploratory endpoints
will include changes in echocardiographic indices of cardiac
structure and function, N-terminal pro b-type natriuretic peptide
(NT-proBNP) concentrations, quality of life questionnaire scores
and daily physical activity assessed using a wearable
accelerometer. Following the 30-week treatment period and
eight-week post-treatment wash-out period, patients will be able to
participate in a long-term extension study of mavacamten.
About Obstructive HCMHypertrophic
cardiomyopathy is the most common genetic cause of heart disease in
which the walls of the heart thicken and prevent the left ventricle
from expanding, resulting in a reduced pumping capacity. HCM
is a chronic disease and for the majority of patients, the disease
progresses slowly and can be extremely disabling. In approximately
two-thirds of HCM patients, or an estimated 410,000 people in the
U.S., 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. Mild exertion can quickly
result in fatigue or shortness of breath, and a patient’s ability
to participate in normal work, family or recreational activities
can be substantially curtailed. Patients with oHCM are at an
increased risk of severe heart failure and death. HCM can
also cause stroke or sudden cardiac death.
About Mavacamten (MYK-461)Mavacamten is a
novel, oral, allosteric modulator of cardiac myosin being developed
for the treatment of hypertrophic cardiomyopathy (HCM).
MyoKardia is currently advancing mavacamten into a pivotal Phase 3
clinical trial, known as the EXPLORER-HCM study, in patients with
symptomatic, obstructive HCM and is currently dosing patients in a
Phase 2 clinical trial, the MAVERICK-HCM study, in patients with
symptomatic non-obstructive HCM. Mavacamten is intended to
reduce cardiac muscle contractility by inhibiting the excessive
myosin-actin crossbridge formation that underlies the excessive
contractility, left ventricular hypertrophy and reduced compliance
characteristic of HCM.
In MyoKardia’s Phase 2 PIONEER-HCM clinical trial of patients
with symptomatic oHCM, primary and secondary endpoints were
achieved across key signs and symptoms of disease, such as
elimination of LVOT gradient post-exercise and at rest, increased
exercise capacity as measured by peak VO2, improved NYHA
classification and reduced dyspnea over time. Mavacamten has
been generally well tolerated in multiple clinical trials. In
April 2016, the U.S. FDA granted Orphan Drug Designation for
mavacamten for the treatment of symptomatic oHCM, a subset of HCM.
Mavacamten is being developed in an ongoing collaboration between
MyoKardia and Sanofi.
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 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 intended to reduce
hypercontractility. Mavacamten is being studied in a pivotal
Phase 3 clinical trial, known as EXPLORER-HCM, in patients with
symptomatic, obstructive HCM. MyoKardia is also developing
mavacamten in a second indication, non-obstructive HCM, in the
Phase 2 MAVERICK-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 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 mavacamten, the initiation of patient dosing in the
Phase 3 EXPLORER-HCM trial, mavacamten’s ability to achieve
applicable endpoints in the Phase 3 EXPLORER-HCM trial, the ability
for patients who participate in the Phase 3 EXPLORER-HCM trial to
participate in a long-term extension study, the availability of
data from the Phase 3 EXPLORER-HCM trial, the potential for data
from the Company’s clinical trials of mavacamten to support a
marketing application, 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 March 31, 2018, 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:
Michelle Corral
Senior Director, Corporate Communications and Investor Relations
MyoKardia, Inc.
650-351-4690
ir@myokardia.com
Hannah Dershowitz (investors)
Stern Investor Relations, Inc.
212-362-1200
hannahd@sternir.com
Steven Cooper (media)
Edelman
415-486-3264
steven.cooper@edelman.com
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