Athersys, Inc. (Nasdaq: ATHX) announced today that its collaborator
in Japan, HEALIOS K.K. (“Healios”) received notification this week
from the Pharmaceutical Affairs and Food Sanitation Council
(“PAFSC”) that its acute respiratory distress syndrome (“ARDS”)
clinical program using HLCM051 (MultiStem® Cell Therapy), will
receive the orphan regenerative medicine designation from the
Ministry of Health, Labour and Welfare within the next few weeks.
This designation may be given to investigational products being
evaluated for indications that have a high unmet medical need and
that occur in less than 50,000 patients in Japan. In addition to
priority consultation and review for potential approval of the
therapy, there are several financial incentives offered with the
designation, such as subsidies to reduce the financial burden of
product development, preferential tax treatment and extension of
the re-examination period for Healios.
ARDS is a serious and life-threatening pulmonary condition that
requires patients to be placed on a ventilator in the intensive
care unit (“ICU”) and is associated with a high mortality rate.
Many patients that do survive face extended stays in the ICU and
experience difficult and challenging recoveries.
In June 2018, Healios and Athersys expanded their existing
collaboration to include the rights to develop and commercialize
MultiStem (HLCM051) to treat ARDS in Japan. In May 2019, Athersys
announced positive results from its Phase 1/2 clinical study (known
as “MUST-ARDS”) at the annual American Thoracic Society meeting.
MultiStem-treated patients experienced less mortality, more
ventilator-free days and more ICU-free days compared to the placebo
patients. After submitting these results, Athersys’ ARDS program
was granted Fast Track Designation by the United States Food and
Drug Administration.
Healios is currently conducting the “ONE-BRIDGE” study to
evaluate MultiStem treatment in pneumonia-induced ARDS patients at
over 20 clinical sites in Japan. This trial began in April 2019 and
is expected to enroll 30 patients. The primary endpoint is the
amount of ventilator-free days at day 28 following treatment.
About Orphan Regenerative Medicine Designation in
Japan
In Japan, drugs and medical devices can be designated as orphan
drugs or medical devices based on the Article 77-2 of the Act on
Securing Quality, Efficacy and Safety of Pharmaceuticals, Medical
Devices, Regenerative and Cellular Therapy Products, Gene Therapy
Products, and Cosmetics if they are intended for use in less than
50,000 patients in Japan and if there is a high unmet medical need.
Programs are designated by the Minister of Health, Labour and
Welfare based on the opinion of the PAFSC. While designation does
not automatically lead to marketing approval, it provides certain
priorities and financial incentives related to potential approval
and product launch.
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. ARDS is associated with a high mortality rate and
significant sequelae 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 quality of life for those suffering with ARDS.
Additionally, given the high treatment costs associated with ARDS,
a successful therapy could be expected to generate significant
savings for the healthcare system by reducing days on a ventilator
and in the ICU.
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 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 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: 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 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
OfficerTel: (216) 431-9900 bjlehmann@athersys.com
Karen HunadyDirector of Corporate Communications and 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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