Athersys, Inc. (Nasdaq:ATHX) today announced positive results from
the analysis of one-year follow-up data from its Phase 2 clinical
study of the intravenous administration of MultiStem® cell therapy
to treat patients who have suffered an ischemic stroke. Dr. David
Hess, lead clinical investigator of this study and a stroke
specialist and Chairman of the Department of Neurology at the
Medical College of Georgia, Augusta University, presented the
summary results today at the 2016 International Stroke Conference
in Los Angeles. The one-year data demonstrates that
MultiStem-treated subjects on average continued to improve through
one year and had a significantly higher rate of “Excellent Outcome”
(defined clinically as attaining mRS 0-1, NIHSS 0-1 and BI ≥95)
compared to placebo subjects at one year when evaluating all
subjects enrolled in the study (p=0.02), i.e., the intent-to-treat
population. The relative improvement in Excellent Outcomes
was even more pronounced in the patients who received MultiStem
treatment within 36 hours of the stroke (p <0.01).
“We are particularly excited by the one-year
follow-up results because they show that MultiStem treatment can
significantly increase the number of patients who have an Excellent
Outcome, meaning complete or nearly full recovery, over the
standard of care when considering all subjects in the trial,”
commented Dr. Gil Van Bokkelen, Chairman & CEO at
Athersys. “The one-year data continues to confirm that
MultiStem treatment is well tolerated and is associated with
continued improvement of other measures of function through one
year. As we saw in the 90-day interim analysis results
announced last April, patients who received MultiStem treatment
within 36 hours of the stroke did substantially better than placebo
patients and later treatment MultiStem subjects. As a result,
we will continue to focus our ongoing clinical development on
treatment within 36 hours of the stroke.”
Data highlights from the 365-day follow-up data
analysis include:
- MultiStem treatment continued to be well tolerated through 365
days;
- Among all subjects who received MultiStem treatment (n=65),
23.1% of patients achieved an Excellent Outcome at 365 days,
compared to 8.2% of patients who received placebo (n=61), and the
14.9% difference was statistically significant (p=0.02) and
compared favorably to the 8.8% difference at 90 days;
- Among patients who received MultiStem treatment within 36 hours
following the stroke, 29.0% achieved Excellent Outcomes (n=31), and
compared to all placebo subjects (n=61), the 20.8% difference was
significant (p<0.01) and also greater than the 9.5% difference
at 90 days;
Proportion of Subjects with Excellent Outcome at Day 90 and
Over One Year |
|
Subjects |
Day 90 |
Day 365 |
All MultiStem (n=65) |
|
15.4 |
% |
|
23.1 |
% |
All Placebo (n=61) |
|
6.6 |
% |
|
8.2 |
% |
Difference with all placebo |
|
8.8 |
% |
14.9%* |
Early Treatment with MultiStem (n=31) |
|
16.1 |
% |
|
29.0 |
% |
Difference with all placebo |
|
9.5 |
% |
20.8%** |
*p = 0.02, **p<0.01
- Substantial improvements were also observed in the Barthel
Index, which is the clinical scale used to assess the ability of
patients to live independently. Among all subjects (65
MultiStem, 61 placebo), 61.5% of MultiStem patients had an
excellent outcome in the Barthel Index (≥95), compared to 44.3% of
placebo patients (p=0.05); furthermore, 67.7% of the subset of
MultiStem patients who had treatment within 36 hours (n=31)
achieved an excellent Barthel outcome, representing a 23.4%
difference with the incidence for all placebo patients (p=0.03);
and
- Among MultiStem patients who did not achieve an Excellent
Outcome at 365 days, there appears to be meaningful benefit from
the treatment relative to standard of care, with reductions in
average initial hospitalization days, mortality, life threatening
adverse events and infections. For example, comparing all
such MultiStem and placebo subjects, MultiStem-treated patients had
1.6 fewer average hospitalization days, and an 11% lower proportion
of patients with death or life threatening adverse events. In
addition, when comparing subjects receiving early treatment with
MultiStem against all placebo subjects, MultiStem patients had an
average of 2.9 fewer hospitalization days, and an 11.4% lower
incidence of death or life threatening adverse events.
Further, such MultiStem patients appear to have better functional
improvement than these placebo patients over one year, as evidenced
by a higher proportion of excellent Barthel Index outcomes (≥95),
50% for MultiStem subjects (and 55% for early treatment MultiStem),
compared to 39% for placebo subjects.
“Achievement of an Excellent Outcome is
important because it means that a patient has substantially
improved in each of the three clinical rating scales used to assess
patient improvement and has regained the ability to live and
function independently with a high quality of life,” continued Van
Bokkelen. “Furthermore, when evaluating patients that either
received no reperfusion therapy, treatment with tPA alone, or
mechanical reperfusion alone, we observed a greater than five-fold
increase in the proportion of patients that achieved an Excellent
Outcome at one year when comparing subjects that received MultiStem
treatment within 36 hours versus placebo.”
Phase 2 Clinical Study
Design
The randomized, double-blind, placebo-controlled
Phase 2 clinical trial was conducted at sites in the United States
and the United Kingdom. The study was conducted in two parts
– a small dose selection phase involving 16 patients in two
cohorts, followed by larger efficacy phase of 118 patients.
The evaluable patient population included 8 patients from cohort 2
and the cohort 3 patients, which all received a high dose of
treatment or placebo.
The study enrolled subjects who received
intravenously either MultiStem treatment or placebo one to two days
following the stroke. Functional and neurological deficit and
recovery following the ischemic stroke were evaluated using three
standard methods: the modified Rankin Score (mRS), a scale from 0-6
directed to assessing disability; the NIH Stroke Scale (NIHSS), a
scale from 0-42 for evaluating neurological deficit; and the
Barthel Index, assessing performance related to activities of daily
living on a 100 point scale. See
www.strokecenter.org/professionals/stroke-diagnosis/stroke-assessment-scales/
for additional information on these assessment scales.
Additionally, other clinical, safety and biomarker data was
collected over the assessment period. Of the patients
evaluated in the study, 65 patients were in the MultiStem treatment
group and 61 patients were in the placebo group, and among the
MultiStem subjects, 31 received MultiStem treatment within 36 hours
following the stroke.
About the Disease Condition
Ischemic stroke is caused by a blockage of blood
flow to the brain. A leading cause of death and disability
globally, each year more than 15 million people are estimated to
suffer a stroke, including more than two million people in the
United States, Japan and European Union, combined. According
to the American Heart Association, ischemic strokes comprise more
than 85% of all strokes. Current standard of care for
ischemic stroke involves the administration of a thrombolytic (clot
dissolving) agent within three to four hours after a stroke has
occurred, a narrow window that results in only a small percentage
of patients receiving such treatment.
About MultiStem
MultiStem cell therapy is a patented
regenerative medicine product that has shown the ability to promote
tissue repair and healing in a variety of ways, such as through the
production of therapeutic factors produced in response to signals
of inflammation and tissue damage. MultiStem therapy’s
potential for multidimensional therapeutic impact distinguishes it
from traditional biopharmaceutical therapies focused on a single
mechanism of benefit. The product represents a unique
"off-the-shelf" stem cell product that can be manufactured in a
scalable manner, may be stored for years in frozen form, and is
administered without tissue matching or the need for immune
suppression. Based upon its efficacy profile, its novel mechanisms
of action, and a favorable and consistent safety profile
demonstrated in both preclinical and clinical settings, MultiStem
therapy could provide a meaningful benefit to patients, including
those suffering from serious diseases and conditions with unmet
medical need. Athersys has forged strategic partnerships and a
broad network of collaborations to develop MultiStem cell therapy
for a variety of indications, with an initial focus in the
neurological, cardiovascular and inflammatory and immune disorder
areas.
About Athersys
Athersys is an international biotechnology
company engaged in the discovery and development of therapeutic
product candidates designed to extend and enhance the quality of
human life. The Company is developing its MultiStem® cell therapy
product, a patented, adult-derived "off-the-shelf" stem cell
product, initially for disease indications in the cardiovascular,
neurological, inflammatory and immune disease areas, and has
several ongoing clinical trials evaluating this potential
regenerative medicine product. Athersys has forged strategic
partnerships and collaborations with leading pharmaceutical and
biotechnology companies, as well as world-renowned research
institutions to further develop its platform and products. More
information is available at www.athersys.com.
Athersys Forward Looking
Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that involve risks and uncertainties. These
forward-looking statements relate to, among other things, the
expected timetable for development of our product candidates, our
growth strategy, and our future financial performance, including
our operations, economic performance, financial condition,
prospects, and other future events. We have attempted to identify
forward-looking statements by using such words as "anticipates,"
"believes," "can," "continue," "could," "estimates," "expects,"
"intends," "may," "plans," "potential," "should," “suggest,”
"will," or other similar expressions. These forward-looking
statements are only predictions and are largely based on our
current expectations. A number of known and unknown risks,
uncertainties, and other factors could affect the accuracy of these
statements. Some of the more significant known risks that we face
that could cause actual results to differ materially from those
implied by forward-looking statements are the risks and
uncertainties inherent in the process of discovering, developing,
and commercializing products that are safe and effective for use as
human therapeutics, such as the uncertainty regarding market
acceptance of our product candidates and our ability to generate
revenues, including MultiStem for the treatment of ischemic stroke,
acute myocardial infarction, spinal cord injury and acute
respiratory distress syndrome and other disease indications,
including graft-versus-host disease. These risks may cause our
actual results, levels of activity, performance, or achievements to
differ materially from any future results, levels of activity,
performance, or achievements expressed or implied by these
forward-looking statements. Other important factors to consider in
evaluating our forward-looking statements include: the success of
our collaboration with Healios, our possible inability to realize
commercially valuable discoveries in our collaborations with
pharmaceutical and other biotechnology companies; the success of
our collaborations, including our ability to reach milestones and
receive milestone payments, and whether any products are
successfully developed and sold so that we earn royalty payments;
our collaborators' ability to continue to fulfill their obligations
under the terms of our collaboration agreements; the success of our
efforts to enter into new strategic partnerships or collaborations
and advance our programs; our ability to raise additional capital;
results from our MultiStem clinical trials; the possibility of
delays in, adverse results of, and excessive costs of the
development process; our ability to successfully initiate and
complete clinical trials; changes in external market factors;
changes in our industry's overall performance; changes in our
business strategy; our ability to protect our intellectual property
portfolio; our possible inability to execute our strategy due to
changes in our industry or the economy generally; changes in
productivity and reliability of suppliers; and the success of our
competitors and the emergence of new competitors. You should not
place undue reliance on forward-looking statements contained in
this press release, and we undertake no obligation to publicly
update forward-looking statements, whether as a result of new
information, future events or otherwise.
Contact:
William (B.J.) Lehmann, J.D.
President and Chief Operating Officer
Tel: (216) 431-9900
bjlehmann@athersys.com
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