In a Phase 1/2 study of MultiStem therapy for
the treatment of ARDS, favorable tolerability data and meaningful
potential benefits in mortality, ventilator-free days and ICU-free
days observed
Company plans to initiate a Phase 2/3 pivotal
study in patients with COVID-19 induced ARDS this quarter
Advancement of key Athersys program with FDA
Fast Track designation for the treatment of ARDS
Athersys, Inc. (NASDAQ: ATHX) announced today that the U.S. Food
and Drug Administration (FDA) has authorized the Company to
initiate a Phase 2/3 pivotal study to assess the safety and
efficacy of MultiStem® therapy in subjects with moderate to severe
acute respiratory distress syndrome (ARDS) induced by the novel
coronavirus disease (COVID-19). This program falls under the
current Investigational New Drug (IND) application for the
Company’s completed MUST-ARDS study and, therefore, a new IND does
not need to be filed. The Company plans to open the first clinical
sites for recruitment of this MACOVIA (MultiStem Administration for
COVID-19 Induced ARDS) study this quarter.
According to the World Health Organization (WHO) and other
recent clinical and epidemiological data, ARDS is the leading cause
of death among COVID-19 infected patients. There are no
FDA-approved medicines for the treatment of ARDS. Athersys recently
completed a Phase 1/2 study evaluating administration of MultiStem
to patients with ARDS and based on the promising data from the
study, the program was recently granted Fast Track designation by
the FDA.
This trial will be a multicenter study featuring an open-label
lead-in followed by a double-blinded, randomized,
placebo-controlled Phase 2/3 portion. The primary objectives of the
MACOVIA study are to evaluate the safety and efficacy of MultiStem
therapy as a treatment for subjects with moderate to severe ARDS
due to COVID-19. The primary efficacy endpoint will be number of
ventilator-free days through day 28 as compared to placebo, a
well-established endpoint for ARDS trials that evaluates an
intervention’s combined impact on survival and liberation from
invasive mechanical ventilation. The secondary objectives of this
study are to evaluate pulmonary function, all-cause mortality,
tolerability and quality of life (QoL) among survivors associated
with MultiStem therapy as a treatment for subjects with moderate to
severe ARDS due to COVID-19. The study is designed to enroll
approximately 400 subjects and will be conducted at leading
pulmonary critical care centers throughout the United States. The
first cohort of the study will be open-label, with a single active
treatment arm to evaluate the safety of the MultiStem product
candidate at two dose levels. The second cohort will be a
double-blind, randomized, placebo-controlled run-in phase to
evaluate the efficacy of MultiStem. The design of the third planned
cohort will be based on analysis of the results of the second
cohort. The intent-to-treat population will include all randomized
subjects (i.e., subjects from the second and third cohorts).
“We are grateful for the FDA’s timely review and feedback during
our design of this pivotal Phase 2/3 study,” commented Dr. Eric
Jenkins, MD, Senior Medical Director and Head of Clinical
Operations at Athersys. “With encouraging non-clinical and clinical
data, affirmed by the FDA’s Fast Track designation for ARDS,
Athersys and its collaborating clinical investigators are highly
motivated by the FDA’s authorization that we may proceed with
enrollment of the first open-label cohort to evaluate safety. We
are in communication with FDA regarding the enrollment of further
cohorts to conduct a scientifically rigorous evaluation of
MultiStem’s safety and efficacy in the treatment of ARDS due to
COVID-19. We believe that MultiStem treatment, by modulating
patients’ hyperinflammatory response to highly pathogenic
respiratory viruses, including SARS-CoV-2 which causes COVID-19,
represents a very promising approach to improving outcomes in
patients who suffer the most severe manifestations of these
illnesses.”
With the spread of COVID-19 and the resulting ARDS cases, there
is an immediate need for therapies for the treatment of ARDS. The
data from the Company’s Phase 1/2 MUST-ARDS study met its primary
endpoint of tolerability and participants were evaluated through 28
days on key clinical parameters as well as improvement in pulmonary
function. As previously reported, study subjects receiving
MultiStem experienced less mortality, more ventilator-free days,
and more intensive care unit (ICU)-free days during the 28-day
clinical evaluation period than the subjects who received placebo.
These differences were observed to be greatest among the
prospectively defined patients with more severe ARDS, defined as
patients with PaO2/FiO2 above or below 150mmHg. Importantly, among
all randomized subjects in the study, 45% of MultiStem treated
subjects were off the ventilator within seven days or less,
compared to only 20% of placebo recipients. Biomarker analysis
confirmed a reduction in inflammatory biomarkers among MultiStem
treated subjects. In addition, results from the one-year follow up
were consistent with the positive day-28 clinical results and an
evaluation of the subjects’ QoL, as measured by patient-reported
EQ-5D-3L self-care responses and visual analogue scale (VAS) pain
scores, more positive among patients who received MultiStem. The
data from this study were presented at the American Thoracic
Society Meeting in May 2019.
MultiStem therapy’s potential for multidimensional therapeutic
impact may distinguish it from traditional biopharmaceutical
therapies focused on a single mechanism of benefit. Since MultiStem
is not virus- or pathogen-specific, we believe it has the potential
to treat ARDS that develops from a variety of causes, including
COVID-19, as well as other pathogen-induced or non-infectious
causes of severe lung inflammation leading to ARDS. The Company is
in discussions with the Biomedical Advanced Research and
Development Authority (BARDA) to expedite the advancement of
MultiStem to treat patients with ARDS resulting from the COVID-19
epidemic and other potential pandemic outbreaks. For more detailed
information on the Company’s ARDS program, please visit the ARDS
page on the Athersys website.
About ARDS
ARDS is a serious respiratory 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. ARDS
is associated with a high mortality rate and significant long-term
complications and disability among survivors. Among survivors, the
condition prolongs ICU and hospital stays and often requires
extended convalescence in the hospital and rehabilitation care
settings. There are limited interventions and no effective drug
treatments for ARDS. There is a large unmet need for a safe
treatment that can reduced mortality and improve QoL for those
surviving ARDS. Additionally, given the high healthcare resource
burden associated with treatment of ARDS patients, a successful
therapy could be expected to generate significant savings for the
healthcare system by reducing days on a ventilator and in the ICU,
or in the setting of a widespread high pathogenicity respiratory
virus pandemic, make those resources more rapidly available to
other patients.
About COVID-19
COVID-19 is the infectious disease caused by the most recently
discovered human coronavirus, SARS-CoV-2. This new disease was
unknown before the outbreak was first discovered in Wuhan, China,
in December 2019. Older people and those with underlying medical
problems such as high blood pressure, heart problems or diabetes,
are more likely to develop serious illness, but even young,
previously healthy people can suffer severe disease and
complications such as ARDS. Data are still emerging but published
case series available today suggest mortality rates among COVID-19
patients who develop ARDS may be as high as 50% to 70%.
About MultiStem®
MultiStem® cell therapy is a patented regenerative medicine
product candidate 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 in response
to signals of inflammation and tissue damage. MultiStem therapy’s
potential for multidimensional therapeutic impact may distinguish
it from traditional biopharmaceutical therapies focused on a single
mechanism of benefit. MultiStem represents a unique "off-the-shelf"
stem cell product candidate 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 favorable efficacy data, its novel mechanisms of action, and
favorable and consistent tolerability data in clinical studies, we
believe that 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 a 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, inflammatory and immune,
cardiovascular and other critical care indications 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. Follow Athersys on Twitter at
www.twitter.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 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: the success of
our MACOVIA study; our ability to raise capital to fund our
operations; our ability to successfully finalize and implement an
alliance with BARDA, and the terms of any such alliance, including
the amount, if any, of funding that we might receive; the timing
and nature of results from MultiStem clinical trials, including our
MASTERS-2 Phase 3 clinical trial and the HEALIOS K.K. (Healios)
TREASURE and ONE-BRIDGE clinical trials in Japan; the impact on our
business, results of operations and financial condition from the
ongoing and global COVID-19 pandemic, or any other pandemic,
epidemic or outbreak of infectious disease in the United States;
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 or us, such as due to material
supply constraints 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 ischemic stroke, ARDS, 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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200413005160/en/
Contact: Ivor Macleod Chief Financial Officer Tel: (216)
431-9900 ir@athersys.com
Karen Hunady Director of Corporate Communications & Investor
Relations Tel: (216) 431-9900 khunady@athersys.com David Schull Russo Partners,
LLC Tel: (212) 845-4271 or (858) 717-2310
David.schull@russopartnersllc.com
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