Cytokinetics Announces Orphan Drug Designation for CK-2127107 for the Treatment of Spinal Muscular Atrophy
May 15 2017 - 7:30AM
Cytokinetics, Incorporated (Nasdaq:CYTK) today announced that the
Office of Orphan Products Development of the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation to
CK-2127107 for the potential treatment of spinal muscular atrophy
(SMA). In collaboration with Astellas Pharma Inc. (TSE:4503)
(“Astellas”), Cytokinetics is developing CK-2127107, a
next-generation fast skeletal muscle troponin activator (FSTA), as
a potential treatment for people living with SMA, chronic
obstructive pulmonary disease (COPD) 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 are pleased that the FDA has granted orphan
drug status to CK-2127107 for the potential treatment of patients
with SMA, one of the most common potentially fatal genetic
disorders due to progressive neuromuscular weakness resulting in
severe respiratory and ambulatory impairment,” said Fady I. Malik,
Cytokinetics’ Executive Vice President and Head of Research &
Development. “We are exploring the potential of CK-2127107 to
improve muscle function and physical performance in patients with
SMA and we look forward to seeing results from our ongoing Phase 2
clinical trial later this year.”
About CK-2127107
Skeletal muscle contractility is driven by the
sarcomere, the fundamental unit of skeletal muscle contraction. It
is 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. CK-2127107, a
novel skeletal muscle activator 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. CK-2127107 has
demonstrated pharmacological activity that may lead to new
therapeutic options for diseases associated with muscle weakness
and fatigue. In non-clinical models of SMA, a skeletal muscle
activator has demonstrated increases in submaximal skeletal muscle
force and power in response to neuronal input and delays in the
onset and reductions in the degree of muscle fatigue. CK-2127107
has been the subject of five completed Phase 1 clinical trials in
healthy volunteers, which evaluated the safety, tolerability,
bioavailability, pharmacokinetics and pharmacodynamics of the drug
candidate. In addition to the Phase 2 clinical trial in patients
with SMA, Cytokinetics is collaborating with Astellas on the
conduct of a Phase 2 clinical trial in patients with COPD. Two
additional clinical trials of CK-2127107 are planned to begin in
2017, one in patients with amyotrophic lateral sclerosis (ALS) and
one in elderly subjects with limited mobility.
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 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 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 may have a
normal life span but eventually suffer gradual weakness in the
proximal muscles of the extremities, eventually 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 and Astellas
Collaboration
In 2013, Astellas and Cytokinetics formed a
partnership focused on the research, development, and
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. Under the collaboration, Cytokinetics exclusively
licensed to Astellas rights to co-develop and potentially
co-commercialize CK-2127107, a FSTA, in non-neuromuscular
indications. In 2014, Astellas and Cytokinetics agreed to expand
the collaboration to include certain neuromuscular indications,
including SMA, and to advance CK-2127107 into Phase 2 clinical
development, initially in SMA. Under the agreement as further
amended in 2016, Astellas has exclusive rights to co-develop and
commercialize CK-2127107 and other FSTAs in non-neuromuscular
indications and certain neuromuscular indications (including SMA
and ALS) and other novel mechanism skeletal muscle activators in
all indications, subject to certain Cytokinetics’ development and
commercialization rights; Cytokinetics may co-promote and conduct
certain commercial activities in North America and Europe under
agreed scenarios.
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 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 for the potential treatment of ALS.
Cytokinetics is preparing for the potential commercialization of
tirasemtiv in North America and Europe and has granted an option to
Astellas 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 three ongoing Phase 2 clinical trials enrolling patients
with spinal muscular atrophy, chronic obstructive pulmonary disease
and ALS. 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; 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 CK-2127107; 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:
Cytokinetics
Diane Weiser
Vice President, Corporate Communications, Investor Relations
(415) 290-7757
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