Cytokinetics, Inc. (Nasdaq:CYTK) today announced that it is joining
forces 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, “Patient Voice,” calls attention to the crucial role that
patients, caregivers and health care advocates play in emphasizing
the urgent needs of rare diseases to legislators and regulators to
effect change and ensure equitable access to life-changing
therapies, care and support.
“Patients with rare diseases, as well as their
families and caregivers, face unique challenges, and deserve our
support today and every day,” said Robert I. Blum, Cytokinetics’
President and Chief Executive Officer. “We stand in solidarity with
these patients, who inspire and motivate us as we collaborate with
the ALS and SMA research and clinical communities to advance the
development of new medicines.”
Cytokinetics is developing two drug candidates
for the potential treatment of rare diseases. Tirasemtiv, a novel
skeletal muscle troponin activator, is being evaluated in a Phase 3
clinical trial, VITALITY-ALS, as a potential treatment for people
living with amyotrophic lateral sclerosis (ALS), and CK-2127107, a
next-generation fast skeletal muscle activator, is being evaluated
in a Phase 2 clinical trial as a potential treatment for people
living with spinal muscular atrophy (SMA), in collaboration with
Astellas.
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 organization representing rare disease patients in Europe, 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), a
rare disease is defined as one that affects fewer than 200,000
people. With nearly 7,000 rare diseases, more than 25 million
Americans are living with a rare disease, but less than 5 percent
of these diseases have a treatment.
About ALS
Amyotrophic lateral sclerosis (ALS) is a
progressive neurodegenerative disease that afflicts approximately
25,000 people in the United States and a comparable number of
patients in Europe. Approximately 5,600 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 10% 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 therapeutic options to
address the symptoms and to modify the disease progression of this
grievous illness.
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. 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 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 muscle activator, for the
potential treatment of 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 for the potential treatment
of ALS. Cytokinetics retains the right to develop and
commercialize tirasemtiv. Cytokinetics is collaborating
with Amgen Inc. to develop omecamtiv mecarbil, a
novel cardiac muscle activator, for the potential treatment of
heart failure. Cytokinetics is collaborating
with Astellas Pharma Inc. to develop CK-2127107, a fast
skeletal muscle activator, for the potential treatment of spinal
muscular atrophy. Amgen holds an exclusive license
worldwide to develop and commercialize omecamtiv
mecarbil and Astellas holds an exclusive license worldwide to
develop and commercialize CK-2127107. Both licenses are subject to
Cytokinetics’ specified development and commercialization
participation rights. For additional information
about Cytokinetics, visit 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 expected revenue and
R&D and G&A expenses, the initiation, conduct, design,
enrollment, progress, continuation, completion and results of
clinical trials, the significance and utility of preclinical study
and clinical trial results, the expected availability of clinical
trial results, planned interactions with regulatory authorities and
the outcomes of such interactions; enrollment in VITALITY-ALS;
enrollment and progress of 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 CK-2127107; the expected timing of
events; 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
further clinical development of tirasemtiv in ALS patients which
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 trials
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 U.S.
Food and Drug Administration 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; Amgen's and Astellas' decisions with respect to the
design, initiation, conduct, timing and continuation of development
activities for omecamtiv mecarbil and CK-2127107, respectively;
Cytokinetics may incur unanticipated research and development and
other costs or be unable to obtain additional financing necessary
to conduct development of its products; Cytokinetics may be unable
to enter into future collaboration agreements for its drug
candidates and programs on acceptable terms, if at all; 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. Forward-looking statements are not guarantees
of future performance, and Cytokinetics' actual results of
operations, financial condition and liquidity, and the development
of the industry in which it operates, may differ materially from
the forward-looking statements contained in this press release. Any
forward-looking statements that Cytokinetics makes in this press
release speak only as of the date of this press release.
Cytokinetics assumes no obligation to update its forward-looking
statements whether as a result of new information, future events or
otherwise, after the date of this press release.
Contact:
Cytokinetics
Diane Weiser
Vice President, Corporate Communications, Investor Relations
(650) 624-3060
Cytokinetics (NASDAQ:CYTK)
Historical Stock Chart
From Mar 2024 to Apr 2024
Cytokinetics (NASDAQ:CYTK)
Historical Stock Chart
From Apr 2023 to Apr 2024