Athersys, Inc. (Nasdaq: ATHX) announced additional favorable data
from its exploratory Phase 1/2 acute respiratory distress syndrome
(ARDS) clinical trial. Dr. Geoff Bellingan, Medical Director at
University College London Hospitals, described the data from the
clinical trial, referred to as the MUST-ARDS trial, during his
presentation today at the American Thoracic Society International
Conference in Dallas, Texas, the longest running, large-scale
conference in the world offering groundbreaking research in
pulmonary, critical care and sleep medicine. His talk was titled,
“Primary Analysis of a Phase 1/2 Study to Assess MultiStem® Cell
Therapy, a Regenerative Advanced Therapy Medicinal Product (ATMP),
in Acute Respiratory Distress Syndrome (MUST-ARDS)”.
As previously reported, subjects 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. The
MultiStem treatment group achieved the primary endpoint of safety
with the MultiStem treatment being well-tolerated and with no
serious adverse events related to administration.
Day 28 Results
|
MultiStem |
Placebo |
Intent to Treat Population |
n=20 |
n=10 |
Day-28 Mortality |
5 (25%) |
4 (40%) |
Ventilator-free (VF) days |
12.9 (10.7) |
9.2 (9.6) |
18.5 [0, 22] |
6.5 [0, 18.3] |
Intensive care unit (ICU)-free days |
10.3 (8.9) |
8.1 (8.9) |
12.5 [0, 18.5] |
4.5 [0, 16.8] |
Data are: n (%), mean (SD) or median [IQR]
“ARDS is more common than people realize, and
there is no cure for it because it is a heterogenous condition,”
commented Dr. Bellingan. “The data from the MUST-ARDS trial is very
encouraging, and I’m looking forward about the potential of having
a therapy to offer ARDS patients.”
In a prospectively-defined analysis examining the effects on
subjects with poorer lung function as determined by a ratio of
partial pressure arterial oxygen and fraction of inspired oxygen
(PaO2/FiO2) of <150, the difference between MultiStem treatment
and placebo was greater than that observed in the intent to treat
population. There was 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. The median values of the data set
were 18.5 VF days for the MultiStem treated patients compared to
3.5 VF days for the placebo group and 12.5 ICU-free days for
MultiStem patients compared to 1 ICU-free day for the placebo
group.
Dr. Bellingan discussed potential mechanisms by which MultiStem
may be providing benefit to ARDS patients, such as through restored
endothelial integrity of the lung, reduced lung edema, increased
alveolar fluid clearance, reduced immune cell infiltrate (including
neutrophils, macrophages and eosinophils), and the ability to shift
immune cells from a pro-inflammatory to anti-inflammatory
phenotype.
A preliminary analysis of the biomarkers reveals that, similar
to results from the Company’s MultiStem MASTERS-1 study for acute
ischemic stroke, there was an overall reduction in pro-inflammatory
cytokines in the MultiStem treatment group compared to placebo
group. Specifically, certain acute inflammatory cytokines were
lower in the ARDS patients that received MultiStem. The same
inflammatory cytokines were also downregulated in the stroke
patients that were in the MultiStem treatment group in the
MASTERS-1 study, suggesting that MultiStem may work in similar way
for both ARDS and stroke.
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.
Treatment was required to begin within four days of ARDS diagnosis
with an average treatment time of approximately two days from the
diagnosis. Initially, three subjects received 300 million MultiStem
cells and, after a safety review, an additional three subjects
received 900 million MultiStem cells. This was followed by the
larger double-blinded, placebo-controlled and randomized study of
twenty subjects treated with an intravenous (IV) administration of
900 million MultiStem cells and ten subjects receiving IV
placebo.
Additionally, last week, Athersys announced that its clinical
program evaluating MultiStem cell therapy for the treatment of ARDS
received Fast Track designation from the United States Food and
Drug Administration. This important designation is given to
qualified investigational therapies that show promise in providing
benefit to patients in areas of significant unmet medical need.
Fast Track designation allows for an expedited regulatory review
process after the clinical data is submitted to help speed
development of promising therapies to the market in order to help
patients in areas where current standard of care is limited.
In April 2019, the Company’s collaborative partner in Japan,
HEALIOS K.K. (Healios), announced the enrollment of the first
patient into its MultiStem ARDS trial, referred to as the
ONE-BRIDGE study. The study is intended to investigate the efficacy
and safety of MultiStem therapy for patients with pneumonia-induced
ARDS in Japan, and its primary endpoint will be the number of VF
days in the first 28 days following treatment.
The Company will consider Healios’ progress in its ONE-BRIDGE
study as it further develops its plans to move the ARDS program
through clinical development.
About ARDS
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 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 approximately 400,000 -
500,000 patients in Europe, the United States and Japan
annually.
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 inflammation, reduce fluid retention in the lungs
and return lung function to normal. Intravenous MultiStem treatment
early following the onset of ARDS may ameliorate the initial
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 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 neurological,
cardiovascular, and inflammatory and immune disease areas, 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 the MultiStem cell therapy toward commercialization.
More information is available at www.athersys.com.
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 Healios’ TREASURE and ONE-BRIDGE
clinical trials 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,
J.D.
President and Chief Operating
Officer
Tel: (216)
431-9900
bjlehmann@athersys.com
Karen Hunady Director of Corporate Communications and Investor
RelationsTel: (216) 431-9900khunady@athersys.com
David
Schull
Russo Partners, LLCTel: (212) 845-4271 or (858)
717-2310David.schull@russopartnersllc.com
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