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Cytokinetics, Inc.: Cytokinetics Announces Top-Line Results From BENEFIT-ALS

Company Summarizes Initial Data From Phase IIb Clinical Trial of Tirasemtiv

No Effect on Primary Efficacy Endpoint; Mixed Effects on Secondary Endpoints


Cytokinetics, Incorporated (Nasdaq: CYTK) announced today the top-line
results of BENEFIT-ALS (B
linded E
valuation of N
euromuscular E
ffects and F
unctional I
mprovement with T

in AL
). Detailed results will be presented during the 66thAnnual Meeting of the
American Academy of Neurology (AAN) on Tuesday, April 29th at the
Pennsylvania Convention Center in Philadelphia, PA.

BENEFIT-ALS did not achieve its primary efficacy endpoint, the mean change
from baseline in the ALS Functional Rating Scale in its revised form
(ALSFRS-R) on tirasemtiv
versus placebo (-2.98 points in thetirasemtiv
group versus -2.40 points in the placebo group, p = 0.11). Secondary efficacy
analyses of the effect oftirasemtiv
on respiratory function and other measures of skeletal muscle function
produced mixed results.

"Patients with ALS desperately need new therapeutic alternatives to slow the
course of their disease and loss of function. We stand with the ALS community
in our disappointment that BENEFIT-ALS did not achieve its primary efficacy
endpoint," stated Robert I. Blum, Cytokinetics' President and Chief Executive
Officer. "The results from BENEFIT-ALS are just now becoming available to our
team at Cytokinetics and will be shared in more detail with the broader
scientific and medical community focused to research in ALS in the next few
days. Understanding these results will require significant further review.
Once we have fully evaluated the data from BENEFIT-ALS, we expect to
determine whether there is a potential development path forward fortirasemtiv
for the potential treatment of ALS and what may be the appropriate next


, 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


BENEFIT-ALS was a Phase IIb, multi-national, double-blind, randomized,
placebo-controlled, clinical trial which was designed to evaluate the safety,
tolerability and efficacy of tirasemtiv
in patients with amyotrophic lateral sclerosis (ALS). BENEFIT-ALS enrolled
711 patients in 73 centers in 8 countries. Patients enrolled in BENEFIT-ALS
began treatment with open-labeltirasemtiv
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
occurred monthly during double-blind treatment; patients also returned 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
compared the mean change from baseline in the ALS Functional Rating Scale in
its revised form (ALSFRS-R) on tirasemtiv
versus placebo. Secondary endpoints evaluated measures of respiratory
performance and other measures of skeletal muscle function and fatigability.

About Amyotrophic Lateral Sclerosis (ALS)

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
commercializeomecamtiv mecarbil
and related compounds, subject to Cytokinetics' specified development and
commercialization participation rights. Cytokinetics is independently
, 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 fromtirasemtiv
, 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

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' research and development
activities, including the conduct, design, and results of clinical trials,
the anticipated timing for the availability of clinical trial results and
planned presentations of such results, evaluations of clinical trial results,
and the significance and utility of clinical trial results; the further
development of 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, the results of BENEFIT-ALS may not support further clinical development
of tirasemtiv; further clinical development of tirasemtiv in ALS patients, if
supported by the BENEFIT-ALS data, will require significant additional
funding, and Cytokinetics 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 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.

Joanna L. Goldstein
Manager, Investor Relations&Corporate Communications
(650) 624-3000


This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
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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