Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the
European Medicines Agency (EMA) has granted orphan medicinal
product designation to reldesemtiv for the potential treatment of
spinal muscular atrophy (SMA). The U.S. Food and Drug
Administration previously granted orphan drug designation to
reldesemtiv for the potential treatment of SMA in 2017. In
collaboration with Astellas, Cytokinetics is developing
reldesemtiv, a fast skeletal muscle troponin activator (FSTA), as a
potential treatment for people with SMA and certain other
debilitating diseases and conditions associated with skeletal
muscle weakness and/or fatigue.
Orphan medicinal product designation is adopted
by the European Commission based on an opinion by the EMA’s
Committee for Orphan Medicinal Products (COMP). Orphan medicinal
product designation is granted by the EMA to medicines intended for
the diagnosis, prevention or treatment of a life-threatening or
chronically debilitating disease affecting fewer than 5 in 10,000
persons in the European Union, or for which it is unlikely that the
costs associated with the development and commercialization of the
medicine would be recovered by expected sales under normal market
conditions without the incentives provided by the designation. The
designation offers potential incentives, which may include a
ten-year period of EU marketing exclusivity from the date of
marketing authorization, EU-funded research, protocol assistance
and fee reductions.
“We’re pleased that reldesemtiv received orphan
designation from the European Commission,” said Fady I. Malik,
M.D., Ph.D., Cytokinetics’ Executive Vice President of Research
& Development. “Despite advances with SMN-directed treatments,
residual muscle impairment and weakness are expected to present
continuing challenges for patients with SMA. Treatment with
reldesemtiv may represent an additive and complementary approach to
increase muscle function.”
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.
In non-clinical models of spinal muscular
atrophy, a skeletal muscle activator increased submaximal skeletal
muscle force and power in response to neuronal input, delayed the
onset of skeletal muscle fatigue and reduced the degree of skeletal
muscle fatigue. Data from preclinical studies of reldesemtiv showed
that the addition of reldesemtiv to treatment with SMN upregulators
significantly increased muscle force in a mouse model of spinal
muscular atrophy. In these studies, the addition of reldesemtiv to
SMN upregulators resulted in a leftward shift of the
force-frequency curve, indicating an increase in calcium
sensitivity of the muscle at submaximal stimulation frequencies and
confirming the efficacy of fast skeletal muscle activation in
muscle in conjunction with SMN upregulators. In a Phase 2
hypothesis-generating clinical study in patients with SMA, patients
treated with reldesemtiv demonstrated increases in measures of
endurance and stamina consistent with the mechanism of action.
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 1 patients have the worst prognosis, with a life expectancy of
no more than two years unless treated with SMN-directed therapies;
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
SMN-directed 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.i 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.ii
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.
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 collaborating with
Astellas Pharma Inc. (Astellas) to develop reldesemtiv, a fast
skeletal muscle troponin activator (FSTA) for diseases of
neuromuscular dysfunction, including SMA and ALS. 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
____________________________i Zeres er al. Journal of
Neurological Sciences 146 (1997) 67-72ii Proprietary market
research and company estimates
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