Cytokinetics Announces Opening of BENEFIT-ALS, A Phase IIb Clinical Trial of Tirasemtiv (CK-2017357) in Patients with Amyotrophic Lateral Sclerosis
(Thomson Reuters ONE) -
BENEFIT-ALS Will Evaluate Longer Term Effects of Novel Skeletal Muscle Activator
and Represents a Key Step Forward Towards Potential Registration
South San Francisco, CA, October 29, 2012 - Cytokinetics, Incorporated (Nasdaq:
CYTK) announced that the company has opened BENEFIT-ALS (Blinded Evaluation of
Neuromusclar Effects and Functional Improvement with Tirasemtiv in ALS),
formerly known as CY 4026, to enrollment. BENEFIT-ALS is a Phase IIb, multi-
national, double-blind, randomized, placebo-controlled, clinical trial designed
to evaluate the safety, tolerability and potential efficacy of tirasemtiv
(formerly CK-2017357) in patients with amyotrophic lateral sclerosis (ALS).
Tirasemtiv selectively activates the fast skeletal muscle troponin complex by
increasing its sensitivity to calcium, which increases skeletal muscle force in
response to neuronal input and delays the onset and reduces the degree of muscle
fatigue. Tirasemtiv is the lead drug candidate that has emerged from the
company's skeletal muscle contractility program.
"Patients who suffer from this devastating disease are in critical need of a
novel therapy that addresses the functional deficits that limit their activities
of daily living," stated Jeremy M. Shefner, MD, PhD, Professor and Chair of the
Department of Neurology at the Upstate Medical University at the State
University of New York and Principal Investigator of BENEFIT-ALS. "If
successful, this novel mechanism therapy could improve the lives of many
patients living 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.
"We are pleased to open BENEFIT-ALS to enrollment to further evaluate tirasemtiv
in patients with ALS. The 12 weeks of double-blind treatment in this trial will
be the longest time that any ALS patients have been treated with tirasemtiv;
consequently, BENEFIT-ALS will be a critical step forward in our assessment of
the potential for this treatment as a chronic therapy," stated Andrew A. Wolff,
MD, FACC, Cytokinetics' Senior Vice President of Clinical Research and
Development and Chief Medical Officer. "Based on feedback from health
regulatory authorities in the United States, Canada and the European Union, we
believe that BENEFIT-ALS may support the potential registration of tirasemtiv
for the treatment of ALS."
Development Status of Tirasemtiv
Tirasemtiv (formerly CK-2017357) is currently the subject of a Phase II clinical
trials development program and has been granted orphan drug designation and fast
track status by the United States Food and Drug Administration and orphan
medicinal product designation by the European Medicines Agency for the potential
treatment of ALS.
Data from two completed randomized, double-blind, placebo-controlled, multiple-
dose, Phase II clinical trials were presented at the April 2012 American Academy
of Neurology Annual Meeting. In one of these trials, tirasemtiv appeared to be
generally safe and well-tolerated when dosed daily for two weeks at 125 mg, 250
mg, or 375 mg, first in a cohort of patients not receiving riluzole, and then in
a cohort of patients receiving riluzole at a reduced dose of 50 mg daily.
Adverse events and clinical assessments during treatment with tirasemtiv
appeared similar, with or without co-administration of riluzole. While the trial
was not designed or powered to evaluate statistically the effects of tirasemtiv
on the various outcome measures that were assessed during the study, a combined
analysis of patients from two separate cohorts suggested encouraging trends in
the ALSFRS-R and in MVV that appeared dose-related and potentially clinically
meaningful in magnitude. In the other Phase II clinical trial, a twice-daily
dose titration regimen of tirasemtiv also appeared to be generally safe and
well-tolerated. The majority of patients in this trial were titrated
successfully to a tirasemtiv dose level of 250 mg twice daily. While this trial
also was not designed or powered to evaluate statistically the effects of
tirasemtiv on the various outcome measures that were assessed during the study,
increases were observed in ALSFRS-R that were similar in direction, and in MVV
that were similar in direction and magnitude, to those observed in the
aforementioned trial. In addition, in December 2010, data from a Phase IIa
clinical trial evaluating single doses of tirasemtiv were presented at the
21(st) International Symposium on ALS and Motor Neurone Diseases. In all three
of these Phase II clinical trials, tirasemtiv appeared to be safe and well-
tolerated, and demonstrated encouraging trends to improvement in patients'
functional abilities, and in measures of respiratory and skeletal muscle
strength and endurance.
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' research and development activities,
including plans for and the timing, initiation, conduct, size, design and
results of clinical trials for tirasemtiv (CK-2017357); the significance and
utility of clinical trial results for tirasemtiv, including the ability of the
BENEFIT-ALS trial to support registration of tirasemtiv; and the properties and
potential benefits of tirasemtiv and Cytokinetics' other drug candidates and
potential drug candidates, including tirasemtiv's potential utility in the
treatment of patients with amyotrophic lateral sclerosis (ALS). 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 will require significant additional funding to conduct a
registration program for tirasemtiv for the potential treatment of ALS and 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 (FDA) or foreign regulatory agencies may delay or limit
Cytokinetics' or its partners' ability to conduct clinical trials, regulatory
authorities may not grant tirasemtiv orphan drug exclusivity in ALS even if it
is approved for marketing; Amgen's decisions with respect to the design,
initiation, conduct, timing and continuation of development activities for
omecamtiv mecarbil; Cytokinetics may be unable to obtain or maintain patent or
trade secret protection for its intellectual property; Cytokinetics may incur
unanticipated research and development and other costs; 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:
Jodi L. Goldstein
Manager, Marketing & Corporate Communications
(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
[HUG#1652939]
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Datum: 29.10.2012 - 12:30 Uhr
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News-ID 197203
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