Athersys Announces Enrollment of First Patient in Masters-2 Phase 3 Study of Multistem® Treatment for Ischemic Stroke
Pivotal
study has received a Special Protocol Assessment (SPA), Fast Track
Designation, and Regenerative Medicine Advanced Therapy (RMAT)
designation from the U.S. Food and Drug Administration (FDA)
CLEVELAND,
July 31, 2018 (GLOBE NEWSWIRE) -- Athersys, Inc. (NASDAQ: ATHX)
announced today that the first patient has been enrolled its Phase 3
study entitled, MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2 (“MASTERS-2”) to evaluate MultiStem® cell therapy treatment of patients who have suffered an ischemic stroke.
The
MASTERS-2 clinical trial is 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.
“We are pleased to have initiated the
MASTERS-2 trial, and to have reached this important milestone regarding
enrollment of the first patient into the trial,” commented Dr. Robert
William Mays, Vice President of Regenerative Medicine and Head of
Neuroscience programs at Athersys. “We believe that MultiStem cell
therapy has the potential to improve standard of care for patients that
have suffered a debilitating stroke and can also meaningfully extend the
treatment window, providing a potential treatment option for many more
patients. The commencement of this trial is an important goal that we
have been working toward while we continue to support the ongoing
TREASURE trial in Japan that is being conducted by our partner,
Healios.”
“Over the past few years we have
conducted studies evaluating the potential therapeutic relevance of
MultiStem in multiple areas, working in collaboration with leading
independent research teams around the world. A growing body of data
suggests that MultiStem could represent an important advance for
critical care indications across several therapeutic areas, including in
acute neurological indications and other indications where there is
substantial unmet medical need. We are committed to conducting robustly
designed clinical trials that will allow us to rigorously evaluate the
potential of this important technology, and we believe this trial
represents a significant step toward achieving that goal,” concluded Dr.
Mays.
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 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.
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 under the 21st
Century Cures Legislation passed by Congress in December 2016. 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.
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 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, 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 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 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: our ability to work with Healios to reach an
agreement on an option for China; 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) 431-9900
bjlehmann@athersys.com
President and Chief Operating Officer
Tel: (216) 431-9900
bjlehmann@athersys.com
Karen Hunady
Corporate Communications & Investor Relations
Tel: (216) 431-9900
khunady@athersys.com
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
Russo Partners, LLC
Tel: (212) 845-4271 or (858) 717-2310
David.schull@russopartnersllc.com
No comments:
Post a Comment