Athersys, Inc. (NASDAQ:ATHX) announced today that its partner,
HEALIOS K.K. (“Healios”), has enrolled the first patient in its
study in Japan evaluating MultiStem® cell therapy treatment of
patients who have suffered an ischemic stroke (the “TREASURE”
study).
The TREASURE study is designed to enroll a total
of 220 patients with moderate to severe strokes and will be
randomized 1:1, with patients receiving a single intravenous
infusion of MultiStem or placebo within 18 to 36 hours of the onset
of the stroke. The primary efficacy outcome is the proportion of
subjects achieving an Excellent Outcome at three months following
treatment. Excellent Outcome is a commonly used measure of
efficacy in stroke treatment, evaluated using three standard
clinical rating scales of functional and neurological deficit and
recovery following the stroke, including the NIH Stroke Scale,
modified Rankin Scale and Barthel Index.
“We are pleased that the Healios TREASURE trial
in Japan is now enrolling patients,” stated Dr. Gil Van Bokkelen,
Chairman and Chief Executive Officer of Athersys. “With a
successful trial outcome, Japan’s progressive regulatory framework
for regenerative medicine products provides the potential for
either conditional or full marketing approval. Additionally,
the priority review designation obtained under the Sakigake
framework is designed to speed the review by Japan’s Pharmaceutical
and Medical Devices Agency, allowing advancement of this program in
a highly efficient manner.”
“This is the largest stem cell trial initiated
under the new regenerative medicine regulatory framework
established in Japan, and it represents an important milestone,”
added Dr. Van Bokkelen. “We believe that MultiStem has the
potential to meaningfully enhance recovery following a stroke and
substantially extend the treatment window from several hours under
current standard of care, which is only relevant to a small
percentage of stroke patients, out to 36 hours, enabling treatment
of a much larger number of people,” concluded Dr. Van Bokkelen.
In parallel with the TREASURE trial, Athersys
has been planning for its Phase 3 study entitled, MultiStem
Administration for Stroke Treatment and Enhanced Recovery Study-2
(“MASTERS-2”), to be conducted in North America and Europe.
The MASTERS-2 study has received authorization from the U.S. Food
and Drug Administration (“FDA”) under a Special Protocol Assessment
(“SPA”) for the design and planned analysis of this pivotal
clinical trial. The SPA provides formal agreement from the
FDA that the protocol design, clinical endpoints, planned conduct
and statistical analyses in this Phase 3 study are acceptable to
support a regulatory submission for marketing approval of MultiStem
cell therapy as a product for treating ischemic stroke patients.
Additionally, the MultiStem stroke program has been awarded Fast
Track designation by the FDA, meaning that the program is eligible
for accelerated approval, priority review and rolling submission of
the biologics license application, which facilitates a timely
regulatory review.
Recently, in addition to the SPA and Fast Track
designations from the FDA, the MASTERS-2 trial also obtained a
Final Scientific Advice positive opinion from the European
Medicines Agency, and received the Regenerative Medicine Advanced
Therapy (RMAT) designation from the FDA, which was established this
year under the 21st Century Cures Legislation. The RMAT
designation may be obtained for eligible cell therapy and other
regenerative medicine and advanced therapies when the FDA agrees
that preliminary clinical evidence indicates that the therapy has
demonstrated the potential to address unmet medical needs for a
serious or life threatening disease or condition. The designation
enables sponsors to discuss with the FDA multidisciplinary
strategic development plans, including expediting manufacturing
development plans for commercialization to support priority review
and accelerated approval.
The MASTERS-2 clinical trial is planned to be a
randomized, double-blind, placebo-controlled clinical trial
designed to enroll 300 patients in North America and Europe who
have suffered moderate to moderate-severe ischemic stroke.
The enrolled subjects will receive either a single intravenous dose
of MultiStem cell therapy or placebo, administered within 18 to 36
hours of the occurrence of the stroke, in addition to the standard
of care. The primary endpoint will evaluate disability using
modified Rankin Scale (mRS) scores at three months, comparing the
distribution, or the “shift,” between the MultiStem treatment and
placebo groups. The mRS shift analysis considers disability across
the full spectrum, enabling recognition of large and small
improvements in disability and differences in mortality and other
serious outcomes, among strokes of different severities. The study
will also assess Excellent Outcome (mRS ≤1, NIHSS ≤1, and Barthel
Index ≥95) at three months and one year as key secondary
endpoints. Additionally, the study will consider other
measures of functional recovery, biomarker data and clinical
outcomes, including hospitalization, mortality, life-threatening
adverse events and post-stroke complications, such as
infection.
About Ischemic Stroke
Stroke represents an area where the clinical
need is particularly significant, since it is a leading cause of
death and serious disability worldwide, with a substantially
impaired quality of life for many stroke victims. Currently, there
are nearly 17 million people who suffer a stroke globally and more
than two million stroke victims each year in the United States,
Europe and Japan, combined. Ischemic strokes, which represent the
most common form of stroke, are caused by a blockage of blood flow
in the brain that cuts off the supply of oxygen and nutrients and
can result in long-term or permanent disability due to neurological
damage. Unfortunately, current therapeutic options for ischemic
stroke victims are limited, since the only available treatments,
administration of the clot dissolving agent tPA, or “thrombolytic,”
or surgical intervention to remove the clot, must be conducted
within several hours of the occurrence of the stroke. As a
consequence of this limited time window, only a small percentage of
stroke victims are treated with the currently available
therapy—most simply receive supportive or “palliative” care. The
long-term costs of stroke are substantial, with many patients
requiring extended hospitalization, extended physical therapy or
rehabilitation (for those patients that are capable of entering
such programs), and many require long-term institutional or family
care.
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. 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
human therapeutics, such as the uncertainty regarding regulatory
approval and market acceptance of our product candidates and our
ability to generate revenues, including MultiStem for the treatment
of ischemic stroke, acute myocardial infarction, spinal cord injury
and acute respiratory distress syndrome and other disease
indications, including graft-versus-host disease. 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 Healios and others, 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 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 or collaborations
and advance our programs; our ability to raise additional capital;
results from our MultiStem clinical trials, including the MASTERS-2
Phase 3 clinical trial and the TREASURE 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 within the expected time frame or at
all; 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 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.
ATHX-STR
Contact:
William (B.J.)
Lehmann
President and Chief Operating
Officer
Tel: (216)
431-9900bjlehmann@athersys.com
Karen Hunady Corporate CommunicationsTel: (216)
431-9900khunady@athersys.com
David Schull Russo Partners, LLCTel:
(212) 845-4271 or (858)
717-2310David.schull@russopartnersllc.com
Athersys (NASDAQ:ATHX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Athersys (NASDAQ:ATHX)
Historical Stock Chart
From Apr 2023 to Apr 2024