Cytokinetics Granted Orphan Drug Designation for Reldesemtiv for the Treatment of Amyotrophic Lateral Sclerosis
December 18 2019 - 7:30AM
Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the
U.S. Food and Drug Administration (FDA) has granted orphan drug
designation to reldesemtiv for the treatment of amyotrophic lateral
sclerosis (ALS). Previously reldesemtiv was granted orphan drug
designation for the treatment of spinal muscular atrophy (SMA) by
the FDA and by the European Medicines Agency. In collaboration with
Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal
muscle troponin activator (FSTA), as a potential treatment for
people with ALS, SMA and certain other debilitating diseases and
conditions associated with skeletal muscle weakness and/or fatigue.
The FDA, through its Office of Orphan
Products Development (OOPD), grants orphan status to drugs and
biologic products that are intended for the safe and effective
treatment, diagnosis, or prevention of rare diseases or disorders
that affect fewer than 200,000 people in the United States.
Orphan drug designation provides a drug developer with certain
benefits and incentives, including a seven-year period of U.S.
marketing exclusivity from the date of marketing authorization,
waiver of FDA user fees, and tax credits for clinical
research.
“We’re pleased that reldesemtiv received orphan
designation from the FDA,” said Fady I. Malik, M.D., Ph.D.,
Cytokinetics’ Executive Vice President of Research &
Development. “It’s an exciting time for the development of
investigational medicines for ALS. We believe treatment with
reldesemtiv may represent a complementary approach to other
potential therapies by directly addressing impaired muscle function
and weakness that affects patients with ALS.”
About Reldesemtiv
Skeletal muscle contractility is driven by the
sarcomere, the fundamental unit of skeletal muscle contraction and
a highly ordered cytoskeletal structure composed of several key
proteins. Skeletal muscle myosin is the motor protein that converts
chemical energy into mechanical force through its interaction with
actin. A set of regulatory proteins, which includes tropomyosin and
several types of troponin, make the actin-myosin interaction
dependent on changes in intracellular calcium
levels. Reldesemtiv, a next-generation FSTA arising from
Cytokinetics’ skeletal muscle contractility program, slows the rate
of calcium release from the regulatory troponin complex of fast
skeletal muscle fibers, which sensitizes the sarcomere to calcium,
leading to an increase in skeletal muscle contractility.
Reldesemtiv has demonstrated pharmacological activity that may lead
to new therapeutic options for diseases associated with skeletal
muscle weakness and fatigue.
FORTITUDE-ALS, the Phase 2 clinical trial of
reldesemtiv in patients with ALS, showed that the trial did not
achieve statistical significance for a pre-specified dose-response
relationship in the primary endpoint of change from baseline in
slow vital capacity (SVC) after 12 weeks of dosing (p=0.11).
However, patients on all dose groups of reldesemtiv declined
numerically less than patients on placebo for SVC and ALS
Functional Rating Scale-Revised (ALSFRS-R), with larger differences
emerging over time.
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 Cytokinetics and Astellas
Collaboration
In 2013, Cytokinetics and Astellas formed a
partnership focused on the research, development, and potential
commercialization of skeletal muscle activators. The primary
objective of the collaboration is to advance novel therapies for
diseases and medical conditions associated with muscle impairment
and weakness. Cytokinetics initially exclusively licensed to
Astellas rights to co-develop and potentially co-commercialize
reldesemtiv and other FSTAs in non-neuromuscular indications and to
develop and commercialize other novel mechanism, skeletal muscle
activators in all indications. Under the agreement as subsequently
expanded and amended, Astellas also has exclusive rights to
co-develop and commercialize reldesemtiv and other FSTAs in certain
neuromuscular indications (including SMA and ALS). Cytokinetics has
certain development and commercialization rights, including the
right to co-promote FSTAs for neuromuscular indications in the
U.S., Canada and Europe and to co-promote the other collaboration
products in the U.S. and Canada. In November 2019, Cytokinetics and
Astellas agreed in principle to revise the terms of the
collaboration with respect to reldesemtiv whereby Cytokinetics
would obtain exclusive control of the product and Astellas, for
reduced collaboration funding and the provision of certain support,
would obtain low- to mid-single future royalties on reldesemtiv in
certain markets. Until the companies finalize revisions, the
collaboration remains in effect in accordance with its current
terms, the agreement in principle remains non-binding, and there
can be no assurance Cytokinetics will enter into definitive
agreements with Astellas regarding any revised terms.
About Cytokinetics
Cytokinetics is a late-stage biopharmaceutical
company focused on discovering, developing and commercializing
first-in-class muscle activators and next-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 collaborating with
Astellas Pharma Inc. (Astellas) to develop reldesemtiv, a fast
skeletal muscle troponin activator (FSTA). 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 the potential benefits of
reldesemtiv, including its ability to represent an additive and
complementary approach to increase muscle function; Cytokinetics’
and its partners’ research and development activities; the timing
of enrollment of patients in Cytokinetics’ and its partners’
clinical trials; the design, timing, results, significance and
utility of preclinical and clinical 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;
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; 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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