Sunday, December 8, 2013

Cytokinetics Announces Presentation Providing Update on BENEFIT-ALS at International Symposium on ALS/MND

Press Release: Cytokinetics Announces Presentation
Providing Update on BENEFIT-ALS at International
Symposium on ALS/MND
Dec 8 at 09:31
Profile hits: NONE

   Interim, Blinded, Aggregate Data on Patient Enrollment, Baseline
Demographics, Dose Escalation and Tolerability Presented; Final Results
Expected in Second Quarter of 2014

   South San Francisco, CA, December 8, 2013 - Cytokinetics, Incorporated
(Nasdaq: CYTK) announced today that a platform presentation of data from
BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional
Improvement with Tirasemtiv in ALS) was given at the 24(th)
International Symposium on ALS/MND on December 7, 2013 in Milan, Italy.
The clinical trial design was explained and interim, double-blind,
aggregate data on patient enrollment, baseline demographics, dose
escalation, and tolerability were presented.  BENEFIT-ALS is evaluating
tirasemtiv, a novel mechanism skeletal muscle activator, as a potential
treatment for patients with amyotrophic lateral sclerosis (ALS).

   Presentation at the 24(th) International Symposium of ALS/MND

   A presentation titled, "The Effect of Tirasemtiv on Functional Status in
Patients with ALS" was given by Jeremy M. Shefner, M.D., Ph.D.,
Professor and Chair, Department of Neurology at the Upstate Medical
University, State University of New York and Lead Investigator for
BENEFIT-ALS.  Dr. Shefner provided an update on BENEFIT-ALS and shared
interim, double-blind, aggregate data (i.e., data from the two treatment
groups, tirasemtiv versus placebo, were not separated) from the ongoing
clinical trial.  He reported that BENEFIT-ALS recently completed
enrollment with a total of 711 patients.  He also concluded that the
open-label, lead-in phase with tirasemtiv appears to maintain the study
blind and to reduce dropouts following randomization.  Dr. Shefner
reported that, to date, the majority of patients randomized in
BENEFIT-ALS are titrated to the target dose of tirasemtiv (250 mg twice
daily) or matching placebo and complete the trial at that dose.  He
reported that dizziness, the most commonly reported adverse effect
associated with tirasemtiv in prior Phase IIa clinical trials, has been
the most frequently reported adverse event in BENEFIT-ALS to date and
has generally resolved during continuous treatment with median duration
of six to seven days.

   Dr. Shefner described the study drug assignment error that had been
reported in July, and explained the response to that error, which is
intended to maintain the original statistical power of the trial and
preserve the statistical integrity of the primary analysis of efficacy.
He concluded that changes in the baseline demographics of patients
observed over time during the conduct of BENEFIT-ALS support the
decision to exclude from the primary efficacy analysis all of the 156
patients randomized concurrently (to tirasemtiv or placebo) in blocks
that included any of the 58 patients subjected to the study drug
assignment error.  Dr. Shefner also shared interim, double-blind,
aggregate data from novel functional assessments employed in BENEFIT-ALS
and remarked on the potential of this clinical trial to provide useful
information regarding the possible effects of tirasemtiv on parameters
of skeletal muscle strength and fatigue, including specific measures of
pulmonary function, that may be relevant to activities of daily living
in patients with ALS.  Dr. Shefner also reported that the recent
completion of patient enrollment in BENEFIT-ALS may enable the results
of this clinical trial to be reported during the second quarter of 2014.

      "I am pleased to have an opportunity to provide an update on BENEFIT-ALS
at the largest international gathering of physicians, patients and caregivers
representing the ALS community," stated Dr. Shefner. "This clinical trial
appears to be proceeding well in line with our objectives. I expect that it will
provide important information regarding this novel drug candidate that offers
potential to impact measures of skeletal muscle and overall patient function
that we know are relevant to the natural history of this progressive disease."


   BENEFIT-ALS is a Phase IIb, multinational, double-blind, randomized,
placebo-controlled clinical trial designed to evaluate the safety,
tolerability and potential efficacy of tirasemtiv, a fast skeletal
muscle troponin activator, in patients with ALS.  Patients enrolled in
BENEFIT-ALS began treatment with open-label dosing of tirasemtiv at 125
mg twice daily.  Patients who tolerated this open-label treatment for
one week were randomized to receive 12 weeks of double-blind treatment
with twice-daily oral ascending doses of tirasemtiv or placebo,
beginning at 125 mg twice daily and increasing weekly up to 250 mg twice
daily (or a dummy dose titration with placebo).  Clinical assessments
occur monthly during double-blind treatment; patients also return for
follow-up evaluations at one and four weeks after their final dose of
double-blind study medication. 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 skeletal muscle function and

   Development Status of Tirasemtiv

   Tirasemtiv (formerly CK-2017357) 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, which increases skeletal muscle
force in response to neuronal input and delays the onset and reduces the
degree of muscle fatigue. Tirasemtiv is 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.  In three
previously completed Phase IIa clinical trials, tirasemtiv appeared to
be generally well-tolerated; in addition, encouraging trends toward
improvement in patients' functional status and increases in respiratory
function parameters and other measures of skeletal muscle strength and
endurance were observed.

   Background on Amyotrophic Lateral Sclerosis

   Amyotrophic lateral sclerosis is a progressive neurodegenerative disease
that afflicts approximately 25,000 people in the United States and a
comparable number of patients in Europe.  Approximately 5,600 new cases
of ALS are diagnosed each year in the United States.  The average life
expectancy of an ALS patient is approximately three to five years after
diagnosis and only 10% of patients survive for more than 10 years.
Death is usually due to respiratory failure because of diminished
strength in the skeletal muscles responsible for breathing.  Few
treatment options exist for these patients, resulting in a high unmet
need for new therapeutic options to address the symptoms and modify the
disease progression of this grievous illness.

   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 to develop and commercialize omecamtiv mecarbil and related
compounds, subject to Cytokinetics' specified development and commercialization
participation rights. Cytokinetics is independently developing tirasemtiv, a
fast skeletal muscle activator, as a potential treatment for diseases and
medical conditions associated with 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
(ALS). Cytokinetics is collaborating with Astellas Pharma Inc. to develop
CK-2127107, a skeletal muscle activator structurally distinct from tirasemtiv,
for non-neuromuscular indications. 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

   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 planned
presentations; Cytokinetics' and its partners' research and development
activities, including the conduct, design, progress and results of
clinical trials; the expected timing for the availability of, and the
significance and utility of, clinical trial results; the objectives of
the protocol amendment for BENEFIT-ALS in response to the drug
assignment error to maintain the originally intended statistical power
of the trial and preserve the statistical integrity of the primary
analysis of efficacy; the properties and potential benefits of
tirasemtiv and Cytokinetics' other drug candidates, including the
potential benefits of tirasemtiv in treating patients with ALS; and the
potential market for tirasemtiv. Such statements are based on
management's current expectations, but actual results may differ
materially due to various risks and uncertainties, including, but not

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