Athersys, Inc. (NASDAQ: ATHX) announced today summary results from
its exploratory clinical study of the intravenous administration of
MultiStem® cell therapy to treat patients who are suffering from
acute respiratory distress syndrome (ARDS). The study results
provide further confirmation of tolerability and a favorable safety
profile associated with MultiStem treatment. Importantly, MultiStem
treatment was associated with lower mortality and a greater number
of ventilator-free and intensive care unit (ICU) free days in the
first month following diagnosis relative to patients receiving
placebo. Furthermore, analysis of initial biomarker data reflects
lower levels of inflammatory markers/cytokines following MultiStem
treatment, an expected mechanism of action in this patient
population.
Patients in the exploratory study were evaluated
through 28 days for the primary clinical assessment and will be
further assessed through a one-year follow-up period. Data
highlights from the initial evaluation include the following
results from the double-blind, randomized, placebo-controlled
portion of the study:
- Lower mortality of 25% in the
MultiStem treatment group vs. 40% in the placebo group;
- 40.2% higher ventilator-free (VF)
days, (12.9 VF days in the MultiStem treatment group vs. 9.2 VF
days for the placebo group);
- 27.2% higher ICU-free days, (10.3
days in MultiStem subjects vs. 8.1 days for subjects receiving
placebo);
- In more severe ARDS patients (as
evident in a prospectively defined analysis), the difference
between MultiStem treatment and placebo was greater – 25% mortality
in MultiStem group vs. 50% in placebo group, 14.6 VF days in
MultiStem group vs. 8.0 VF days in placebo group, and 11.4 ICU-free
days in MultiStem group versus 5.9 ICU-free days in placebo group;
and
- MultiStem treatment was well
tolerated in this very sick ARDS patient population, with no
serious adverse events related to administration.
The study was designed to evaluate the impact of
MultiStem treatment in subjects with acute onset of moderate to
severe ARDS and was conducted at sites in the United States and
United Kingdom. The study included two parts – a small initial dose
confirmation phase, followed by the larger double-blinded,
placebo-controlled and randomized phase (Phase 2a portion).
Treatment was required to begin within four days of ARDS diagnosis
with an average treatment time of approximately two days from the
diagnosis. Six subjects were treated with MultiStem in the initial
portion of the study, and in the Phase 2a portion of the study, 20
subjects were treated with an intravenous (IV) administration of
900 million MultiStem cells and 10 subjects received IV placebo. As
disclosed previously, the study was not powered for the efficacy
outcomes.
Athersys will continue to evaluate the data as
the one-year follow-up period is completed for all patients in the
trial. Athersys and the study investigators plan to present more
detailed and comprehensive results at a medical science conference
after additional analyses.
“We believe this exploratory study met all of
its key objectives and view it as an important indication of the
potential relevance of MultiStem in another critical care area
where there is substantial unmet medical need,” commented Dr. Gil
Van Bokkelen, Chairman & CEO at Athersys. “The consistent
pattern of results suggesting a beneficial effect on mortality and
key clinical parameters provides us with confidence that this is
another condition where administration of MultiStem can have a
meaningful therapeutic impact.
“We also believe these results provide strong
support for the ARDS trial that our partner, HEALIOS K.K.
(Healios), is authorized to conduct in Japan under the recently
implemented regenerative medicine regulatory framework designed to
expedite development. Healios is actively working towards
initiation of this study, and we continue to support their efforts,
as well as advance our other key programs,” concluded Dr. Van
Bokkelen.
In addition to this ARDS study, Athersys is
conducting ongoing studies in ischemic stroke (MASTERS-2 Phase 3
study) and acute myocardial infarction, and is planning for a study
to treat severe trauma patients. Athersys is also supporting
studies in Japan being conducted by Healios, targeting ischemic
stroke (TREASURE study) and ARDS. Athersys continues to make
progress in building its capabilities and capacity for completing
development, gaining approval and supporting potential commercial
activities, and ended the year with approximately $51 million in
cash and cash equivalents to support these activities.
About ARDS
Acute Respiratory Distress Syndrome (ARDS) is a
serious immunological and inflammatory condition characterized by
widespread inflammation in the lungs. ARDS can be triggered by
pneumonia, sepsis, trauma or other events, and represents a major
cause of morbidity and mortality in the critical care setting. It
has significant implications, as it prolongs intensive care unit
(ICU) and hospital stays and requires convalescence in the hospital
and rehabilitation. There are limited interventions and no
effective drug treatments for ARDS, making it an area of high unmet
clinical need with high treatment costs. Given ARDS high treatment
costs, a successful cell therapy could be expected to generate
significant savings for the healthcare system by reducing days on a
ventilator and in the ICU and importantly, could reduce mortality
and improve quality of life for those suffering from the condition.
The medical need for a safe and effective treatment of ARDS is
significant due to its high mortality rate, and it affects annually
approximately 400,000 - 500,000 patients in Europe, the United
States and Japan.
MultiStem cell therapy has demonstrated the
capacity to reduce inflammation, support tissue regeneration and
promote homeostasis in acute immunological and injury settings.
Preclinical data suggests that MultiStem cells may have a
protective effect by shifting the physiological response from
pro-inflammatory to anti-inflammatory, and through the promotion of
key reparative mechanisms. In animal models, MultiStem cells have
demonstrated an ability to reduce the severity of pulmonary
distress, reduce alveolar edema and return lung endothelial
permeability to normal. Intravenous MultiStem treatment early
following the onset of the condition may ameliorate the initial
hyper-inflammation and reduce the fibrotic activity that follows,
thereby speeding the return to and improving the likelihood of more
normal lung function and helping patient recovery.
About MultiStem®
MultiStem® cell therapy is a patented
regenerative medicine product in clinical development 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 therapy
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 clinical studies, MultiStem therapy could
provide a meaningful benefit to patients, including those suffering
from serious diseases and conditions with unmet medical need.
About Athersys
Athersys is an international biotechnology
company engaged in the development of therapeutic products 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 focused on disease
indications in the neurological, cardiovascular, inflammatory and
immune, certain pulmonary and other areas where the current
standard of care is limited or inadequate for many patients, and
has several ongoing clinical trials evaluating this potential
regenerative medicine product. Athersys has forged strategic
partnerships and a broad network of collaborations to further
advance MultiStem cell therapy toward commercialization. More
information is available at www.athersys.com. Follow Athersys on
Twitter at www.twitter.com/athersys.
This clinical trial included subjects from the
United Kingdom and the United States. The United Kingdom arm of the
trial was supported by £2M funding from Innovate UK. Cell and Gene
Therapy Catapult, working alongside Athersys, provided the United
Kingdom clinical operations support for the successful enrollment
of the trial. This research was also supported by the National
Institute On Alcohol Abuse And Alcoholism, as part of the U.S.
National Institutes of Health under Award Number R42AA024003. The
information provided here is solely the responsibility of the
authors and does not necessarily represent the official views of
the National Institutes of Health.
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
therapeutics, including 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: our ability to raise capital to fund our operations; the
timing and nature of results from our MultiStem clinical trials,
including the MASTERS-2 Phase 3 clinical trial and the Healios’
TREASURE clinical trial in Japan; the possibility of delays in,
adverse results of, and excessive costs of the development process;
our ability to successfully initiate and complete clinical trials
of our product candidates; the possibility of delays, work
stoppages or interruptions in manufacturing by third parties to us,
such as due to material supply constraints, contaminations, or
regulatory issues, which could negatively impact our trials and the
trials of our collaborators; uncertainty regarding market
acceptance of our product candidates and our ability to generate
revenues, including MultiStem cell therapy for the treatment of
stroke, acute respiratory distress syndrome, acute myocardial
infarction and trauma, and the prevention of graft-versus-host
disease and other disease indications; changes in external market
factors; changes in our industry's overall performance; changes in
our business strategy; our ability to protect and defend our
intellectual property and related business operations, including
the successful prosecution of our patent applications and
enforcement of our patent rights, and operate our business in an
environment of rapid technology and intellectual property
development; our possible inability to realize commercially
valuable discoveries in our collaborations with pharmaceutical and
other biotechnology companies; our ability to work with Healios to
reach an agreement for an option in China; our ability to meet
milestones and earn royalties under our collaboration agreements,
including the success of our collaboration with Healios; our
collaborators’ ability to continue to fulfill their obligations
under the terms of our collaboration agreements and generate sales
related to our technologies; the success of our efforts to enter
into new strategic partnerships and advance our programs,
including, without limitation, in North America, Europe and Japan;
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.
Contacts:
William (B.J.)
Lehmann
President and Chief Operating
Officer
Tel: (216) 431-9900bjlehmann@athersys.com
Karen Hunady Corporate Communications &
Investor RelationsTel: (216) 431-9900khunady@athersys.com
David SchullRusso Partners, LLCTel: (212)
845-4271 or (858) 717-2310David.schull@russopartnersllc.com
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