Patient Baseline Characteristics from
VITALITY-ALS Comparable to BENEFIT-ALS
Cytokinetics, Inc. (Nasdaq:CYTK) today announced that new data was
presented at the 27th International Symposium on ALS/MND in Dublin,
Ireland, including patient baseline characteristics from
VITALITY-ALS and results of an international physician survey on
the use of noninvasive ventilation (NIV) in the treatment of ALS.
VITALITY-ALS, a multi-national, randomized,
double-blind, placebo-controlled Phase 3 clinical trial designed to
assess the effects of tirasemtiv versus placebo on slow vital
capacity (SVC) and other measures of respiratory and skeletal
muscle function in patients with ALS, completed enrollment in
August 2016. The trial screened 866 patients, enrolled 744 and
randomized 566 patients.
Baseline characteristics of patients enrolled in
VITALITY-ALS are similar to those from BENEFIT-ALS and other
recently conducted clinical trials in patients with ALS. Patients
enrolled in VITALITY-ALS are on average 57.6 years of age, 65
percent male, 7.7 months from diagnosis, 20.6 months from their
first symptom and had an average percent predicted SVC of 90.7
percent. Like other trials of tirasemtiv, the most common adverse
events (AEs) observed to date are dizziness, fatigue and nausea.
Approximately 24 percent of patients withdrew from VITALITY-ALS
during the two week open-label phase, primarily due to AEs, similar
to what was observed during the first two weeks of tirasemtiv
treatment in BENEFIT-ALS, the Phase 2 clinical trial of tirasemtiv
in patients with ALS.
“We’re pleased to provide the first look at the
patient population from VITALITY-ALS and note the consistency with
previously conducted large-scale ALS trials,” said Jeremy M.
Shefner, M.D., Ph.D., Lead Investigator of VITALITY-ALS, Professor
and Chair of Neurology at Barrow Neurological Institute, and
Professor and Executive Chair of Neurology at the University of
Arizona, Phoenix. “We look forward to sharing full results from
VITALITY-ALS in late 2017 and remain hopeful that treatment with
tirasemtiv may slow the decline of SVC and other measures of muscle
strength including measures of respiratory function.”
Vital Capacity Key Driver to Initiating
Noninvasive Ventilation
Results of an international physician survey on
the use of NIV in the treatment of ALS presented by Terry
Heiman-Patterson, M.D., Director of the Center for
Neurodegenerative Disorders, and Professor of the Department of
Neurology at the Lewis Katz School of Medicine at Temple
University, revealed similarities in best practices for initiating
NIV in North America and Europe, yet differences in the time to
initiation. Among ALS specialists including physicians, nurse
practitioners, and physician assistants, US specialists rank
upright FVC, supine FVC, and symptoms of orthopnea and/or dyspnea
as the three most important factors influencing NIV initiation,
while EU specialists prioritize symptoms of orthopnea and/or
dyspnea, sleep-related symptoms, and supine SVC.
Regional variations were observed in the timing
of NIV initiation, in part influenced by varying insurance coverage
and regulations. In the US, 70 percent of specialists indicated
that insurance regulations and national health care coverage impact
time to NIV initiation, compared to 47.5 percent of EU specialists.
The FVC/SVC value at which specialists reported initiating NIV also
differed regionally, with many US specialists citing an upright
FVC/SVC <50 percent vital capacity, and EU specialists more
often initiating NIV at a higher upright FVC/SVC of <70 percent
or <80 percent.
“Although we found geographic differences in how
and when noninvasive ventilation is used, vital capacity remains
one of the most important measures influencing the decision,” said
Terry Heiman-Patterson, M.D., Director of the Center for
Neurodegenerative Disorders, and Professor of the Department of
Neurology at the Lewis Katz School of Medicine at Temple
University. “These results help us understand what additional
research is needed to optimize NIV use in all patients, and may
help inform the design of future clinical trials in ALS.”
About VITALITY-ALS
VITALITY-ALS is a multi-national, randomized,
double-blind, placebo-controlled clinical trial in patients with
possible, probable or definite ALS, diagnosed within 24 months, and
with percent predicted SVC at baseline ≥ 70 percent. The primary
endpoint of the trial will assess change from baseline in SVC, to
be assessed after 24 weeks of double-blind, placebo-controlled
treatment. Secondary endpoints, to be assessed at 48 weeks, include
time to decline from baseline in percent predicted SVC by ≥ 20
percentage points or the onset of respiratory insufficiency or
death; time to decline from baseline in percent predicted SVC to ≤
50 percent predicted or the onset of respiratory insufficiency or
death; time to first occurrence of any use of assisted ventilation
or death; time to decline in any of the three respiratory domains
of the ALSFRS-R or death; and change in the Mega-Score of muscle
strength. Patients enrolled in VITALITY-ALS receive two-weeks of
open-label treatment with tirasemtiv administered at 250 mg/day.
Patients are then randomized into a double-blind treatment phase to
placebo or one of three target tirasemtiv dose levels (250 mg/day,
375 mg/day, 500 mg/day) in a 3:2:2:2 ratio. After 48 weeks of
randomized, double-blind, placebo-controlled treatment, patients
who received tirasemtiv during those 48 weeks of double-blind
treatment will be randomized to continue the tirasemtiv dose at
which they completed the 48 weeks of double-blind treatment or to
placebo for a four-week double-blind, tirasemtiv withdrawal
phase. Patients who received placebo during the 48 weeks of
double-blind treatment will continue to receive placebo during the
double-blind, tirasemtiv withdrawal phase. Following their
participation in VITALITY-ALS, patients are eligible to participate
in an open-label extension study of tirasemtiv.
About ALS
Amyotrophic lateral sclerosis (ALS) is a
progressive neurodegenerative disease that afflicts approximately
30,000 people in the United States and a comparable number of
patients in Europe. Approximately 6,000 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 percent 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 therapies to address
functional deficits and disease progression.
About Tirasemtiv
Tirasemtiv, a novel skeletal muscle activator,
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. Tirasemtiv has been studied in clinical trials
that have enrolled over 1000 people
internationally. Tirasemtiv is currently the subject of
VITALITY-ALS, a Phase 3 clinical trial, designed to confirm and
extend findings on measures of respiratory function and muscle
strength from prior studies.
About Cytokinetics
Cytokinetics is a late-stage
biopharmaceutical company focused on discovering, developing and
commercializing first-in-class muscle activators as potential
treatments for debilitating diseases in which muscle performance is
compromised and/or declining. As a leader in muscle biology and the
mechanics of muscle performance, the company is developing small
molecule drug candidates specifically engineered to increase muscle
function and contractility. Cytokinetics’ lead drug candidate
is tirasemtiv, a fast skeletal muscle troponin activator, for
the potential treatment of 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 retains the right to develop and
commercialize tirasemtiv, subject to an option held
by Astellas Pharma Inc. Cytokinetics is also
collaborating with Astellas to develop CK-2127107, a fast skeletal
muscle activator, for the potential treatment of spinal muscular
atrophy, chronic obstructive pulmonary disease and
ALS. Cytokinetics is collaborating with Amgen
Inc. to develop omecamtiv mecarbil, a novel cardiac
muscle activator, for the potential treatment of heart
failure. 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. For additional information
about Cytokinetics,
visit http://www.cytokinetics.com/.
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’ and
its partners’ research and development activities, including the
conduct, design, enrollment, progress and timing of results of the
VITALITY-ALS Phase 3 clinical trial of tirasemtiv in
patients with ALS; the significance and utility of preclinical
study and clinical trial results; and the properties and potential
efficacy and safety profile 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 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 trial 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 FDA 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; Cytokinetics may incur unanticipated research
and development and other costs or be unable to obtain additional
financing necessary to conduct development of its products;
standards of care may change, rendering Cytokinetics' drug
candidates obsolete; and 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:
Cytokinetics
Diane Weiser
Vice President, Corporate Communications, Investor Relations
(415) 290-7757
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