Athersys, Inc. (Nasdaq:ATHX) today announced interim results from
its exploratory Phase 2 clinical study of the intravenous
administration of MultiStem® cell therapy to treat patients who
have suffered an ischemic stroke. The study results demonstrate
favorable safety and tolerability for MultiStem, consistent with
prior studies. With respect to the primary and secondary endpoints,
the cell therapy did not show a difference at 90 days compared to
placebo. However, MultiStem treatment was associated with lower
rates of mortality and life threatening adverse events (AEs),
infections and pulmonary events. Furthermore, post-hoc analysis
shows that patients who received MultiStem treatment earlier in the
treatment window had more robust recovery rates in comparison to
placebo and relative to patients who received later MultiStem
treatment.
Dr. David Hess, lead clinical investigator in the study, stroke
specialist and Chairman of the Department of Neurology at the
Medical College of Georgia at Georgia Regents University, will
present the summary results at the European Stroke Organization
conference on Sunday, April 19th at 8:50 AM in Glasgow, Scotland,
United Kingdom.
Data highlights from the 90-day interim analysis include:
- MultiStem cell therapy demonstrated favorable tolerability and
safety profile through the evaluation date, which was at least 90
days for all patients;
- Patients who received intravenous administration of MultiStem
did not show a significant difference from placebo-treated patients
for the primary endpoint (Global Stroke Recovery Assessment) and
the related secondary endpoints – which were defined as the
proportion of patients achieving a modified Rankin Scale (mRS)
value of 0-2, improvement in the NIH Stroke Scale (NIHSS) by ≥ 75%
and achieving Barthel Index (BI) ≥ 95 at day 90, however;
- Among all subjects who received MultiStem treatment, 15.4% of
patients achieved an Excellent Outcome, defined clinically as
attaining mRS 0-1, NIHSS 0-1 and BI ≥95, compared to 6.6% of
patients that received placebo, (p=0.10);
- As described in the table below, patients who received
MultiStem treatment earlier in the treatment window (24-36 hours
post-stroke) exhibited more favorable recovery on the primary and
key secondary endpoints than patients who received placebo or
patients who received MultiStem treatment later (e.g. Excellent
Outcome, p = 0.03), and this treatment effect was even more
pronounced the earlier the MultiStem administration within the
24-36 hour timeframe;
- A higher proportion of patients who received treatment with
MultiStem achieved a good clinical outcome by day 7 after
treatment, defined as achieving mRS 0-2, which was 12.9% for
patients receiving MultiStem, compared to 5.2% of patients who
received placebo, and a similar pattern was seen for improvement in
NIHSS ≥ 75%, which was achieved by 10.2% of MultiStem treated
patients vs. 3.9% of patients who were treated with placebo;
- Mortality was lower among patients who received treatment with
MultiStem in comparison with placebo. There were 9 subject deaths
(14.8%) among those receiving treatment with placebo, and only 4
patient deaths (6.2%) among patients receiving treatment with
MultiStem;
- The MultiStem treatment group had a lower rate of life
threatening adverse events and death (p=0.04), and also exhibited
lower rates of pulmonary events (p=0.08) and infections. The
MultiStem treated group also had a significantly lower level of
circulating CD-3+ T-cells at two days following dosing (p<0.01),
suggesting a reduction in the inflammatory response post-stroke,
consistent with the therapeutic hypothesis.
As noted above, post-hoc analyses show that earlier MultiStem
administration appears to provide substantial benefit, as evident
in the following table:
MultiStem Administered
≤36 Hours Compared to All Placebo* |
|
Clinical parameter, at 90 days |
MultiStem** n=27 |
Placebo n=52 |
Difference |
Global Test Statistic |
Odds Ratio =2.21,
p=0.07 |
Modified Rankin Scale ≤2 |
48.1% |
32.7% |
15.4% |
NIHSS Improvement ≥75% |
51.9% |
30.8% |
21.1% |
Barthel Index ≥95 |
55.5% |
38.5% |
17.0% |
Excellent Outcome |
18.5% |
1.9% |
16.6% |
(mRS ≤1, NIHSS ≤1, and BI ≥95%) |
|
|
p=0.03 |
* Excludes confounding
data from patients that received both tPA and mechanical
reperfusion in addition to investigational product |
** Results for patients
getting MultiStem administration >36 hours not materially
different from placebo |
"This exploratory Phase 2 trial represents our first clinical
study in stroke and was designed to evaluate the safety and
efficacy of a single dose of MultiStem 24-48 hours following the
occurrence of the stroke. This treatment window extends well beyond
the limits of current standard of care, treatment with tPA, which
may only be administered within the first several hours after a
stroke," commented Dr. Gil Van Bokkelen, Chairman & CEO at
Athersys. "Going into this trial, based on extensive
preclinical work we have conducted internally and with independent
labs, we anticipated that administration of MultiStem could provide
several mechanisms of benefit, including down regulation of key
inflammatory pathways, up regulation of multiple reparative
mechanisms, and could also extend the treatment window in a
meaningful way."
"While the trial did not achieve the primary or component
secondary endpoints, we believe the evidence indicating that
patients who received MultiStem treatment early appeared to exhibit
meaningfully better recovery is very important and promising,"
continued Dr. Van Bokkelen. "The results appear to confirm
that our window of intervention with MultiStem therapy may extend
well beyond the limits of current care. Additional key
observations from the trial also appear consistent with key
elements of our initial hypothesis. In particular, we are
encouraged by the reduced mortality and lower incidence of
infections, pulmonary events and life threatening adverse events
among MultiStem-treated patients, as well as the limited biomarker
data we have seen so far. We anticipate additional data and
information from the study and will conduct further analyses to
generate more insight about the potential for MultiStem treatment
in this area."
"We also continue to advance other clinical programs in acute
myocardial infarction (AMI) and acute respiratory distress syndrome
(ARDS). In AMI, we have previously published promising data
from our Phase 1 study, testing localized delivery of MultiStem
shortly following a heart attack, and look forward to completing
this Phase 2 study as soon as possible. With respect to ARDS,
we are encouraged by the apparent impact in this study on
mortality, life threatening adverse events, infection and pulmonary
events, which together with our non-clinical study results in
pulmonary injury models, suggests the potential for benefit in this
patient population. We have a good balance sheet to support
this ongoing work and remain confident that our MultiStem therapy
and other technologies will have an important impact," concluded
Dr. Van Bokkelen.
Phase 2 Clinical Study Design
The randomized, double-blind, placebo-controlled Phase 2
clinical trial is being 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 either MultiStem
treatment or placebo one to two days following the stroke. The
primary endpoints for the study include safety over the first seven
days following treatment and global stroke recovery at day 90,
which assesses disability (modified Rankin Score ≤ 2), neurological
deficit (NIH stroke scale, delta ≥75%) and activities of daily
living (Barthel Index ≥ 95%). Additionally, there are
secondary and exploratory endpoints evaluating elements of recovery
and dysfunction, including biomarkers associated with subject
condition and recovery, and safety variables over the study
period.
Of the patients evaluated in the study, 65 patients were in the
MultiStem treatment group and 61 patients were in the placebo
group. Among the enrolled patients, the groups were generally
evenly balanced in terms of baseline stroke characteristics.
|
Characteristic |
MultiStem |
Placebo |
Age, mean (at time of admission) |
61.6 |
62.5 |
% of male Subjects |
52.2% |
54.8% |
% of female Subjects |
47.8% |
45.2% |
NIHSS at baseline, mean |
13.3 |
13.4 |
% of patients that received tPA |
43.3% |
48.4% |
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.
The Athersys, Inc. logo is available at:
http://www.globenewswire.com/newsroom/prs/?pkgid=4548
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. 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 Chugai, 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 ability to raise additional capital; final 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 realize commercially valuable discoveries in our
collaborations with pharmaceutical and other biotechnology
companies; 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 and
advance our programs; 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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