Cytokinetics Announces Presentation of Phase IIA Clinical Trial Data of Tirasemtiv in Patients with Myasthenia Gravis
(Thomson Reuters ONE) -
Positive Results from Evidence of Effect Trial Inform Translation of Novel
Mechanism of Action
South San Francisco, CA, March 21, 2013 - Cytokinetics, Incorporated (Nasdaq:
CYTK) announced the presentation yesterday of positive data from a completed
Phase IIa "Evidence of Effect" clinical trial of tirasemtiv in patients with
generalized myasthenia gravis (MG) during the Emerging Science Program at the
65(th) Annual Meeting of the American Academy of Neurology. Tirasemtiv, the
lead drug candidate from the company's skeletal muscle contractility program,
selectively activates the fast skeletal muscle troponin complex by increasing
its sensitivity to calcium, thereby increasing skeletal muscle force in response
to neuronal input and delaying the onset and reducing the degree of muscle
fatigue. Tirasemtiv is being evaluated as a potential treatment for amyotrophic
lateral sclerosis (ALS) in BENEFIT-ALS (Blinded Evaluation of Neuromuscular
Effects and Functional Improvement with Tirasemtiv in ALS), an international
Phase IIb clinical trial that is now enrolling patients.
Emerging Science Presentation at the 65(th) Annual Meeting of the American
Academy of Neurology
A presentation titled "A Study to Evaluate Efficacy, Safety and Tolerability of
Single Doses of Tirasemtiv in Patients with Myasthenia Gravis" was made by
Donald B. Sanders, MD, FAAN, Professor, Division of Neurology and Founder of the
Duke Myasthenia Gravis Clinic and Chair. The presentation summarized the
results of a double-blind, randomized, three-period crossover, placebo-
controlled, pharmacokinetic and pharmacodynamic study of tirasemtiv in patients
with generalized MG. In this trial, also known as CY 4023, patients received
single, oral, double-blind doses of placebo, 250 mg of tirasemtiv, and 500 mg of
tirasemtiv in random order and approximately one week apart. The main objectives
of this trial were to assess the effects of tirasemtiv on various measures of
skeletal muscle strength and fatigue including measures of pulmonary function.
Since CY 4023 was a hypothesis-generating trial, no single primary efficacy
endpoint was pre-specified.
At six hours after dosing in CY 4023, improvements (i.e., decreases) in the
Quantitative MG score (QMG) were related to the dose of tirasemtiv. Decreases
in the QMG score were dose related (-0.49 QMG points per 250 mg; p = 0.02). At
six hours after the 500 mg dose of tirasemtiv, the QMG score decreased by 0.99
points (p = 0.02). In a responder analysis, twice as many patients (n = 12)
improved by 3 points or more six hours after dosing with 500 mg of tirasemtiv
than six hours after placebo (p = 0.098). The QMG is a validated index of
disease severity that is often employed as a primary endpoint in clinical trials
of patients with MG. In addition, the percent predicted forced vital capacity
(FVC) increased relative to placebo at six hours after both the 250 mg and 500
mg doses of tirasemtiv (7.0 ± 2.1%, p = 0.0012 and 4.5 ± 2.1%, p = 0.034,
respectively). Increases in percent predicted FVC were also dose-related (2.2%
per 250mg, p = 0.043).
The authors concluded that both the 250 mg and 500 mg single oral doses of
tirasemtiv studied in this Phase IIa clinical trial were well-tolerated by the
32 patients enrolled. No premature terminations and no serious adverse events
were reported in this trial. The most commonly reported adverse event in this
trial was dizziness, which was mild in all but one case that was classified as
moderate.
Cytokinetics has been awarded $3.5 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. Through December 31, 2012,
Cytokinetics has incurred $4.6 million in research and development expenses
associated with its MG program, and has received $3.2 million or 70% of the
program's funding from 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.
Development Status of Tirasemtiv
Tirasemtiv (formerly CK-2017357) is currently being evaluated in BENEFIT-ALS, an
international, double-blind, randomized, placebo-controlled, Phase IIb clinical
trial designed to evaluate the safety, tolerability and potential efficacy of
this novel drug candidate in patients with ALS. BENEFIT-ALS is designed to
enroll approximately 400 patients who will first complete one week of treatment
with open-label tirasemtiv at 125 mg twice daily. Following completion of the
open-label period, patients will be randomized to receive 12 weeks of double-
blind treatment with twice-daily oral ascending doses of tirasemtiv beginning at
125 mg twice daily and increasing weekly up to 250 mg twice daily or a dummy
dose titration with placebo. Clinical assessments will take place monthly during
the course of treatment; patients will also participate in follow-up evaluations
one and four weeks after their final dose. The primary efficacy analysis of
BENEFIT-ALS will compare the mean change from baseline in the ALS Functional
Rating Scale in its revised form (ALSFRS-R) on tirasemtiv versus placebo.
Secondary endpoints will include Maximum Voluntary Ventilation (MVV) and other
measures of respiratory and skeletal muscle function. Patients taking riluzole
at the time of enrollment and who are randomized to receive tirasemtiv will
receive riluzole at a reduced dose of 50 mg daily. Cytokinetics plans to conduct
this trial at over 70 sites across the United States, Canada, and several
European countries.
Data from prior Phase IIa clinical trials of tirasemtiv in patients with ALS
were presented at the 2012 American Academy of Neurology Annual Meeting and the
2010 International Symposium on ALS and Motor Neurone Diseases.
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' lead drug candidate from its cardiac muscle
contractility program, omecamtiv mecarbil, is in Phase II clinical development
for the potential treatment of heart failure. Amgen Inc. holds an exclusive
license worldwide (excluding Japan) to develop and commercialize omecamtiv
mecarbil and related compounds, subject to Cytokinetics' specified development
and commercialization participation rights. Cytokinetics is independently
developing tirasemtiv, a skeletal muscle activator, as a potential treatment for
diseases and conditions associated with aging, muscle wasting or neuromuscular
dysfunction. Tirasemtiv is currently the subject of a Phase II clinical trials
program and 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 amyotrophic
lateral sclerosis, a debilitating disease of neuromuscular impairment in which
treatment with tirasemtiv produced potentially clinically relevant
pharmacodynamic effects in Phase II trials. 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 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 conduct, design and results of clinical trials, the
significance and utility of clinical trial results, 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, Cytokinetics anticipates that it will be required to conduct at
least one confirmatory Phase III clinical trial of tirasemtiv in ALS patients
which will require significant additional funding, and it may be unable to
obtain such additional funding on acceptable terms, if at all; 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 decisions with respect to the design, initiation, conduct, timing and
continuation of development activities for omecamtiv mecarbil; 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.
Contact:
Joanna (Jodi) L. Goldstein
Manager, Corporate Communications & Marketing
(650) 624-3000
This announcement is distributed by Thomson Reuters on behalf of
Thomson Reuters clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Cytokinetics, Inc. via Thomson Reuters ONE
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Datum: 21.03.2013 - 12:30 Uhr
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