Cytokinetics Joins Global Initiative To Recognize International Rare Disease Day
February 28 2019 - 7:30AM
Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that
it is standing with the European Organisation for Rare Diseases
(EURORDIS) and the National Organization for Rare Disorders (NORD)
to recognize Rare Disease Day®, an international campaign elevating
the public understanding of rare diseases. This year’s theme for
Rare Disease Day, “Bridging health and social care,” calls
attention to the coordination between medical, social, and support
services, and underscores the challenges people with rare diseases
and their families face while balancing care and the demands of
everyday life.
“It’s our honor to once again join EURORDIS and
NORD to bring awareness to Rare Disease Day. This year’s theme is
an important reminder of what people living with rare diseases, as
well as their families and caregivers, confront on a daily basis.”
said Robert I. Blum, Cytokinetics’ President and Chief
Executive Officer. “We look forward to sharing results in the next
few months from our Phase 2 clinical trial of reldesemtiv in
patients living with ALS. This trial is further testament to our
longstanding commitment to the courageous patients who battle rare
diseases and who inspire us to develop our potential medicines for
devastating diseases of muscle dysfunction.”
Cytokinetics is collaborating with Astellas to
develop reldesemtiv, a fast skeletal muscle troponin muscle
activator. Reldesemtiv was studied in a Phase 2 clinical study in
patients with spinal muscular atrophy (SMA) from which data were
reported in 2018. Reldesemtiv is also being studied in
FORTITUDE-ALS
(Functional Outcomes in
a Randomized Trial
of Investigational Treatment
with CK-2127107
to Understand Decline
in Endpoints – in ALS),
a Phase 2 clinical trial designed to assess the change from
baseline in percent predicted slow vital capacity and other
measures of skeletal muscle function after treatment
with reldesemtiv (formerly CK-2127107). Cytokinetics
recently announced that results from FORTITUDE-ALS are expected to
be reported in Q2 2019.
About Rare Disease Day
Rare Disease Day, which takes place every year
on the last day in February, was established in Europe in
2008 by the European Organisation for Rare
Diseases (EURORDIS) and is now observed in more than 80
nations. In the United States, Rare Disease Day is sponsored
by the National Organization for Rare Disorders (NORD), a
leading independent, non-profit organization committed to the
identification, treatment, and cure of rare diseases. According to
the National Institutes of Health (NIH), in the US, a
rare disease is defined as one that affects fewer than 200,000
people. With over 6,000 rare diseases, 25 million Americans are
living with a rare disease, but only 5 percent of these diseases
have a treatment.
About ALS
Amyotrophic lateral sclerosis (ALS) is a
progressive neurodegenerative disease that afflicts approximately
20,000 people in the United States and a comparable
number of patients in Europe. Approximately 5,000 new cases of
ALS are diagnosed each year in the United States. The average
life expectancy of an ALS patient is approximately three to five
years after diagnosis and only approximately 10 percent of patients
survive for more than 10 years. Death is usually due to respiratory
failure because of diminished strength in the skeletal muscles
responsible for breathing. Few treatment options exist for these
patients, resulting in a high unmet need for new therapies to
address functional deficits and disease progression.
About SMA
SMA is a severe, genetic neuromuscular disease
that leads to debilitating muscle function and progressive, often
fatal, muscle weakness. It occurs in 1 in 6,000 to 10,000 live
births each year and is one of the most common potentially fatal
genetic disorders. Spinal muscular atrophy manifests in various
degrees of severity as progressive muscle weakness resulting in
respiratory and mobility impairment. There are four types of SMA,
named for age of initial onset of muscle weakness and related
symptoms: Type 1 (Infantile), Type 2 (Intermediate), Type 3
(Juvenile) and Type 4 (Adult onset). Of the prevalent
population, approximately 80% of the patients are characterized as
Type 2 and Type 3. Life expectancy and disease severity vary
by type of SMA. Type I patients have the worst prognosis, with a
life expectancy of no more than two years; Type 2 patients have
delayed motor milestones with the most advanced milestone normally
achieved being sitting unsupported; Type 3 patients can
usually stand and walk but have increasingly limited mobility as
their abilities regress as they age; Type 4 patients may have a
normal life span but eventually suffer gradual weakness in the
proximal muscles of the extremities, eventually resulting in
mobility issues. With the recent introduction of gene modifying
therapies, it is expected that patients may live longer but will
still have a significant need to address ongoing disabilities
related to respiration and mobility. Approximately 50% of
Type 3 patients with SMA are believed to maintain ambulatory
function today and an increasing number of Type 2 patients with SMA
are expected to remain ambulatory with the advent of complementary
new therapies that can enable motor milestones. Over the next
5 years, the prevalence of ambulatory adolescents and adults with
SMA may exceed 5-10,000 patients in the United
States alone.
About Cytokinetics
Cytokinetics is a late-stage
biopharmaceutical company focused on discovering, developing and
commercializing first-in-class muscle activators and best-in-class
muscle inhibitors as potential treatments for debilitating diseases
in which muscle performance is compromised and/or declining. As a
leader in muscle biology and the mechanics of muscle performance,
the company is developing small molecule drug candidates
specifically engineered to impact muscle function and
contractility. Cytokinetics is collaborating
with Amgen Inc. (Amgen) to develop omecamtiv
mecarbil, a novel cardiac muscle activator. Omecamtiv
mecarbil is the subject of an international clinical trials
program in patients with heart failure including GALACTIC-HF and
METEORIC-HF. Amgen holds an exclusive worldwide license
to develop and commercialize omecamtiv mecarbil with a
sublicense held by Servier for commercialization
in Europe and certain other
countries. Cytokinetics is also collaborating
with Amgen to develop AMG 594, a cardiac troponin
activator, discovered under the companies’ joint research program.
Further development of AMG 594 is subject to the collaboration
agreement
between Amgen and Cytokinetics. Cytokinetics is
collaborating with Astellas Pharma Inc. (Astellas) to
develop reldesemtiv, a fast skeletal muscle troponin activator
(FSTA). Reldesemtiv has been granted orphan drug
designation by the FDA for the potential treatment of
spinal muscular atrophy. Reldesemtiv was the subject of a
Phase 2 clinical study in patients with spinal muscular atrophy
which showed increases in measures of endurance and stamina
consistent with the mechanism of action. Reldesemtiv is
currently the subject of a Phase 2 clinical trial in patients with
amyotrophic lateral sclerosis. Cytokinetics is also
advancing CK-601, a next-generation FSTA, under the collaboration
with Astellas. Astellas holds an exclusive worldwide license to
develop and commercialize reldesemtiv. Licenses held
by Amgen and Astellas are subject to specified
co-development and co-commercialization rights
of Cytokinetics. Cytokinetics is also developing
CK-274, a novel cardiac myosin inhibitor that company scientists
discovered independent of its collaborations, for the potential
treatment of hypertrophic cardiomyopathies.
Cytokinetics continues its over 20-year history of pioneering
innovation in muscle biology and related pharmacology focused to
diseases of muscle dysfunction and conditions of muscle
weakness.
For additional information
about Cytokinetics, visit www.cytokinetics.com and follow
us on Twitter, LinkedIn, Facebook and YouTube.
Forward-Looking Statements
This press release contains forward-looking
statements for purposes of the Private Securities Litigation Reform
Act of 1995 (the “Act”). Cytokinetics disclaims any intent or
obligation to update these forward-looking statements and claims
the protection of the Act's Safe Harbor for forward-looking
statements. Examples of such statements include, but are not
limited to, statements relating to Cytokinetics’ and its partners’
research and development activities; the design, results,
significance and utility of preclinical study results; and the
properties and potential benefits of Cytokinetics’ drug candidates.
Such statements are based on management's current expectations, but
actual results may differ materially due to various risks and
uncertainties, including, but not limited to, potential
difficulties or delays in the development, testing, regulatory
approvals for trial commencement, progression or product sale or
manufacturing, or production of Cytokinetics’ drug candidates that
could slow or prevent clinical development or product approval,
including risks that current and past results of clinical trials or
preclinical studies may not be indicative of future clinical trial
results, patient enrollment for or conduct of clinical trials may
be difficult or delayed, Cytokinetics’ drug candidates may have
adverse side effects or inadequate therapeutic efficacy, the FDA or
foreign regulatory agencies may delay or limit Cytokinetics’ or its
partners’ ability to conduct clinical trials, and Cytokinetics may
be unable to obtain or maintain patent or trade secret protection
for its intellectual property; Astellas’ decisions with respect to
the design, initiation, conduct, timing and continuation of
development activities for reldesemtiv; Cytokinetics may incur
unanticipated research and development and other costs or be unable
to obtain additional financing necessary to conduct development of
its products; standards of care may change, rendering Cytokinetics’
drug candidates obsolete; competitive products or alternative
therapies may be developed by others for the treatment of
indications Cytokinetics’ drug candidates and potential drug
candidates may target; and risks and uncertainties relating to the
timing and receipt of payments from its partners, including
milestones and royalties on future potential product sales under
Cytokinetics’ collaboration agreements with such partners. For
further information regarding these and other risks related to
Cytokinetics’ business, investors should consult Cytokinetics’
filings with the Securities and Exchange Commission.
Contact:CytokineticsDiane
WeiserVice President, Corporate Communications, Investor
Relations(415) 290-7757
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