Data from Evidence
of Effect Trial Provide Support for Novel Mechanism of Action
in Neuromuscular Diseases
South San Francisco, CA, March 25, 2015 - Cytokinetics, Incorporated (Nasdaq: CYTK) announced
the publication of a manuscript relating to its fast skeletal
muscle troponin activator tirasemtiv in the
journal Neurotherapeutics. This
publication summarizes results from a Phase IIa "Evidence of
Effect" or hypothesis-generating clinical trial which evaluated
tirasemtiv in patients with generalized
myasthenia gravis (MG). Tirasemtiv is
the lead drug candidate from Cytokinetics' skeletal muscle
contractility program and is being developed as a potential
treatment for amyotrophic lateral sclerosis (ALS).
"We are pleased to share
additional clinical data relating to tirasemtiv in patients with generalized myasthenia
gravis," stated Andrew A. Wolff, MD, FACC, Cytokinetics' Senior
Vice President and Chief Medical Officer. "We believe that
effects observed on the Quantitative Myasthenia Gravis score and on
vital capacity following administration of a single dose of
tirasemtiv support the evaluation of skeletal
muscle activation in patients with neuromuscular disorders
including ALS. We are preparing to initiate a Phase III
clinical development program to evaluate the effects of tirasemtiv on measures of respiratory function and
other measures of skeletal muscle performance in patients with
ALS."
The publication, titled "A
Double-Blinded, Randomized, Placebo-Controlled Trial to Evaluate
Efficacy, Safety, and Tolerability of Single Doses of Tirasemtiv in Patients with Acetylcholine
Receptor-Binding Antibody-Positive Myasthenia Gravis," appeared
online in the March edition of the journal Neurotherapeutics. The primary objective of this
early-stage clinical study was to evaluate the effects of single
250 mg and 500 mg doses of tirasemtiv versus
placebo on measures of skeletal muscle function and fatigability in
patients with generalized MG and persistent muscle weakness.
The secondary objectives of the study were to evaluate and
characterize the relationship, if any, between the doses and plasma
concentrations of tirasemtiv and its
pharmacodynamic effects, and to evaluate the safety and
tolerability of tirasemtiv administered as
single doses to patients with MG. The authors concluded that
6 hours after dosing, tirasemtiv produced
dose-related improvements from baseline in the Quantitative MG
(QMG) score (slope: -0.49 QMG point per 250 mg administered;
p=0.02; lower scores indicate better function) and in percent
predicted forced vital capacity (slope: 2.2 % increase per 250 mg
administered; p=0.04). The QMG improved by >3 points in twice as
many patients after 500 mg tirasemtiv than
after placebo. Both doses of tirasemtiv were
well tolerated; there were no premature terminations or serious
adverse events. The results of this study suggest that tirasemtiv may improve muscle function in patients with
MG and support further development of tirasemtiv in neuromuscular diseases.
About Tirasemtiv
Tirasemtiv, a
novel skeletal muscle activator, is the lead drug candidate from
Cytokinetics' skeletal muscle contractility program. Tirasemtiv selectively activates the fast skeletal
muscle troponin complex by increasing its sensitivity to calcium
and, in preclinical studies and early clinical trials, demonstrated
increases in skeletal muscle force in response to neuronal input
and delays in the onset and reductions in the degree of muscle
fatigue. Cytokinetics is developing tirasemtiv, a fast skeletal muscle activator, as a
potential treatment for ALS.
Cytokinetics conducted BENEFIT-ALS, a Phase IIb, multi-national,
double-blind, randomized, placebo-controlled, clinical trial
designed to evaluate the safety, tolerability and efficacy
of tirasemtiv in patients with
ALS. BENEFIT-ALS enrolled 711 patients from 73 centers
in 8 countries. Following review of results from BENEFIT-ALS and
initial regulatory interactions in both the United States and
Europe, the company is preparing to advance tirasemtiv to a Phase III clinical development
program that is designed to potentially confirm and extend results
from BENEFIT-ALS. Objectives of the Phase III program will include
measures of respiratory function after longer duration treatment in
patients with ALS, including effects on Slow Vital
Capacity.
About Myasthenia
Gravis
Myasthenia gravis is a
progressive, chronic neuromuscular disease that commonly strikes
people between the ages of 40 and 70 and afflicts between 50,000
and 85,000 people in the United States. Approximately 13,600 new
cases of myasthenia gravis are diagnosed each year. Myasthenia
gravis is an autoimmune disease in which the immune system attacks
the junction between nerve and muscle, targeting either the muscle
cell's acetylcholine receptor (which receives signals from the
associated nerve cell) or the muscle-specific kinase, a protein
that helps to organize acetylcholine receptors on the muscle cell.
The cause of myasthenia gravis is unclear. Researchers suspect
viruses or bacteria may trigger the autoimmune response; the thymus
gland also may play a role in the disease. Symptoms include fatigue
and weakness of voluntary muscles, including partial paralysis of
eye movements, double vision, droopy eyelids, and weakness and
fatigue in neck and jaw regions. This weakness fluctuates daily but
tends to progress over the course of a few years, especially as may
be untreated.
About
Cytokinetics
Cytokinetics is a clinical-stage
biopharmaceutical company focused on the discovery and development
of novel small molecule therapeutics that modulate muscle function
for the potential treatment of serious diseases and medical
conditions. Cytokinetics is developing tirasemtiv, a fast skeletal muscle activator, as a
potential treatment for amyotrophic lateral sclerosis (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 collaborating with Amgen Inc. to develop omecamtiv mecarbil, a 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. All of
these drug candidates have arisen from Cytokinetics' muscle biology
focused research activities and are directed towards the
cytoskeleton. The cytoskeleton is a complex biological
infrastructure that plays a fundamental role within every human
cell. Additional information about Cytokinetics can be obtained at
http://www.cytokinetics.com/.
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 initiation, conduct, design, and results
of clinical trials, the significance and utility of clinical trial
results, the expected availability of clinical trial results, and
the use of effects on respiratory function, including slow vital
capacity, as a Phase III clinical trial endpoint for tirasemtiv;
potential markets for tirasemtiv; and the properties and potential
benefits of tirasemtiv 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 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.
Funding: Cytokinetics received $3.4 million in
grants (Award Number RC3NS070670) from the National Institute of
Neurological Disorders and Stroke (NINDS) to fund research and
development of tirasemtiv in MG.
Cytokinetics incurred $4.7 million in research and
development expenses associated with its MG program; 73% of the
program's funding was from the NINDS. The content of this press
release is solely the responsibility of Cytokinetics and does not
necessarily represent the official views of the NINDS or the
National Institutes of Health.
Contact:
Cytokinetics, Inc.
Joanna L. Goldstein (Investors &
Media)
(650) 624-3000
This
announcement is distributed by NASDAQ OMX Corporate Solutions on
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The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Cytokinetics, Inc. via Globenewswire
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