Cytokinetics Joins Global Initiative to Recognize International Rare Disease Day
February 28 2017 - 7:30AM
Cytokinetics, Inc. (Nasdaq:CYTK) today announced that it is joining
the global initiative with the European Organisation for Rare
Diseases (EURORDIS) and the National Organization for Rare
Disorders (NORD) to raise awareness of Rare Disease Day®, an
international campaign dedicated to elevating the public
understanding of rare diseases. This year’s theme, Research, calls
attention to the importance of scientific research to better
understand rare diseases and to develop innovative treatments to
better patients’ lives.
“We are proud to stand together with EURORDIS
and NORD and to honor people living with rare diseases. We are
especially inspired by this year’s theme highlighting the promise
of innovative research,” said Robert I. Blum, Cytokinetics’
President and Chief Executive Officer. “This is truly an exciting
year for us as we expect results from late-stage clinical trials of
our novel mechanism drug candidates in patients with ALS and SMA,
both rare diseases urgently in need of new therapies.”
Cytokinetics is developing drug candidates for
the potential treatment of rare diseases. Tirasemtiv, a fast
skeletal troponin activator (FSTA), is being evaluated in a Phase 3
clinical trial, VITALITY-ALS, as a potential treatment for
amyotrophic lateral sclerosis (ALS), and CK-2127107, a
next-generation FSTA, is being evaluated in a Phase 2 clinical
trial as a potential treatment for spinal muscular atrophy (SMA),
in collaboration with Astellas. Data from VITALITY-ALS are expected
in Q4 2017 and data from the Phase 2 trial of CK-2127107 are
expected in the second half of 2017.
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 nearly 7,000 rare diseases, 25 million
Americans are living with a rare disease, but only 5 percent of
these diseases have a treatment.
About ALS
ALS is a progressive neurodegenerative disease
that afflicts approximately 30,000 people in the United States and
a comparable number of patients in Europe. Approximately 6,000 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 neuromuscular disease that
occurs in 1 in every 6,000 to 10,000 live births each year and is
one of the most common fatal genetic disorders. SMA 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 I (Infantile), Type II (Intermediate), Type
III (Juvenile) and Type IV (Adult onset). 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 2 years;
Type IV patients have a normal life span but eventually suffer
gradual weakness in the proximal muscles of the extremities
resulting in mobility issues. Few treatment options exist for these
patients, resulting in a high unmet need for new therapeutic
options to address symptoms and modify disease progression.
About Cytokinetics
Cytokinetics is a late-stage
biopharmaceutical company focused on discovering, developing and
commercializing first-in-class muscle activators 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 increase muscle
function and contractility. Cytokinetics’ lead drug candidate
is tirasemtiv, a fast skeletal troponin activator
(FSTA). Tirasemtiv is the subject of
VITALITY-ALS, an international Phase 3 clinical trial in
patients with ALS. Tirasemtiv has been granted orphan
drug designation and fast track status by the U.S. Food and
Drug Administration and orphan medicinal product designation
by the European Medicines Agency. Cytokinetics is
preparing for the potential commercialization
of tirasemtiv in North
America and Europe and has granted an option
to Astellas Pharma Inc. for development and
commercialization in other countries. Cytokinetics is
collaborating with Astellas to develop CK-2127107, a
next-generation fast skeletal muscle activator. CK-2127107 is
the subject of two ongoing Phase 2 clinical trials enrolling
patients with spinal muscular atrophy and chronic obstructive
pulmonary disease. Cytokinetics is collaborating
with Amgen Inc. to develop omecamtiv mecarbil, a
novel cardiac muscle activator. Omecamtiv
mecarbil is the subject of GALACTIC-HF, an international Phase
3 clinical trial in patients with heart
failure. 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. Astellas
holds an exclusive worldwide license to develop and commercialize
CK-2127107. Licenses held by Amgen and Astellas are
subject to Cytokinetics' specified co-development and
co-commercialization rights. For additional information
about Cytokinetics,
visit http://www.cytokinetics.com/.
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, including the conduct, design,
enrollment, progress and timing of results of the VITALITY-ALS
Phase 3 clinical trial of tirasemtiv in patients with ALS and the
Phase 2 clinical trial of CK-2127107 in patients with SMA; the
significance and utility of preclinical study and clinical trial
results; and the properties and potential efficacy and safety
profile of tirasemtiv, CK-2127107 and Cytokinetics' other 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,
further clinical development of tirasemtiv in ALS patients will
require significant additional funding, and Cytokinetics may be
unable to obtain such additional funding on acceptable terms, if at
all; the FDA and/or other regulatory authorities may not accept
effects on slow vital capacity as a clinical endpoint to support
registration of tirasemtiv for the treatment of ALS; 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; 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; and 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:
Cytokinetics
Diane Weiser
Vice President, Corporate Communications, Investor Relations
(650) 624-3060
Cytokinetics (NASDAQ:CYTK)
Historical Stock Chart
From Aug 2024 to Sep 2024
Cytokinetics (NASDAQ:CYTK)
Historical Stock Chart
From Sep 2023 to Sep 2024