MultiStem® stem cell technology receives
Positive Opinion from European Medicines Agency
Athersys, Inc. (Nasdaq:ATHX) today announced that the design of its
Phase 3 clinical study treating ischemic stroke with its
proprietary MultiStem® cell therapy product (the “MASTERS-2” study)
has received a Final Scientific Advice positive opinion from the
European Medicines Agency (“EMA”). This represents EMA’s
opinion that, upon success, the study should be sufficient to
warrant approval for commercialization, which is in line with the
U.S. Special Protocol Assessment and Fast Track Designation.
In Japan, Athersys’ partner, HEALIOS K.K. (“Healios”) is conducting
the TREASURE study, evaluating MultiStem therapy for ischemic
stroke, and has received a priority review designation under
Sakigake from Japan’s Pharmaceutical and Medical Devices
Agency. The results from the TREASURE study, if needed, are
acceptable for supporting the applications for registration, along
with the results from the MASTERS-2 study, in both the U.S. and
Europe.
This means that, in the three major
pharmaceutical markets, Athersys and its existing and future
partners have clarity with respect to the development path to
commercialization for MultiStem therapy for moderate to severe
ischemic stroke, facilitating planning and operations and removing
regulatory uncertainty from the development program.
Additionally, the stroke program is positioned for priority review
and accelerated approval, shortening the time from successful study
results to commercialization. As a result, with successful
study outcome and registration, Athersys’ MultiStem cell therapy
could represent the first major advancement in decades for the
treatment of acute stroke, outside of limited current treatment
options.
“We are excited about receiving the positive
opinion on our pivotal, registrational stroke study, MASTERS-2, and
we thank the EMA for its continued support of our efforts to
develop an important new treatment option for patients in this area
of significant unmet medical need,” commented Dr. Gil Van Bokkelen,
Chairman & CEO at Athersys. “Recent publications and
presentations reinforce years of promising work and continue to
illustrate how we might be able to improve outcomes and
substantially extend the treatment window to 36 hours for stroke
patients with MultiStem, beyond the narrow treatment window for tPA
and mechanical reperfusion that exist today. The publications
also provide further evidence of the important biological
mechanisms that MultiStem cell therapy may convey to help patients
recover from a debilitating stroke event. Stroke remains a
leading cause of serious disability and represents a substantial
burden on patients, families and healthcare systems around the
world, and we are committed to transforming medical care in this
important area.”
About EMA Scientific Advice
Scientific Advice is a procedure offered by EMA
to stakeholders for clarification of questions arising during
development of medicinal products. The scope of Scientific Advice
spans scientific issues that may be related to quality,
non-clinical, and/or clinical aspects of the concerned medicinal
product under development. The advice is designed to facilitate the
development and availability of high-quality, effective and
acceptably safe medicines, for the benefit of patients and helps to
ensure that developers perform the appropriate tests and studies,
so that no major objections regarding the design of the tests are
likely to be raised during evaluation of the
marketing-authorization application. Such major objections can
significantly delay the marketing of a product, and, in certain
cases, may result in refusal of the marketing authorization.
Following the Agency’s advice increases the probability of a
positive outcome. For more information on the Scientific Advice
process, please visit:
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000049.jsp
About Special Protocol
Assessments
The Special Protocol Assessment process is a
procedure by which the FDA provides official evaluation and written
guidance on the design and planned analysis of proposed study that
are intended to form the basis of efficacy claims for a new drug
application or biologic license application. Final marketing
approval depends on the results of the efficacy, the adverse event
profile and an evaluation of the benefit/risk of treatment
demonstrated in the Phase 3 clinical program. The S.P.A.
agreement may only be modified through a written agreement between
the sponsor and the FDA, or if the FDA becomes aware of a
substantial scientific issue essential to product efficacy or
safety. For more information on the S.P.A. process, please
visit:
http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm080571.pdf
About Fast Track
Designation
Fast Track is a process designed by the FDA to
facilitate the development and expedite the review of drugs to
treat serious conditions and fill unmet medical needs. The purpose
is to get important new drugs to the patient earlier. Fast Track
addresses a broad range of serious conditions. For more
information on the Fast Track process, please
visit:https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm
About the SAKIGAKE
Designation
The Strategy of the Pharmaceutical and Medical
Devices Agency’s SAKIGAKE designation consists of the following two
measurements and its coverage ranges from basic research to
clinical research/trials, approval reviews, safety measures,
insurance coverage, improvement of infrastructure and the
environment for corporate activities, and global expansion.
- SAKIGAKE Designation System: promoting research and development
in Japan aiming at early practical application for innovative
pharmaceutical products, medical devices and regenerative
medicines.
- Scheme for Rapid Authorization of Unapproved Drugs:
accelerating the practical application of unapproved/off-label use
of drugs for serious and life-threatening diseases by expanding the
scope of the Council on Unapproved Drugs/Off-label Use to include
unapproved drugs in the western countries if certain conditions are
satisfied, and by improving the environment for companies to
undertake development of such drugs.
For more information on the SAKIGAKE process,
please
visit:http://www.pmda.go.jp/english/review-services/reviews/advanced-efforts/0001.html
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 that suffer a stroke globally and, on
average, someone in the United States has a stroke every 40
seconds. 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 therapies,
administration of the clot dissolving agent tPA, or “thrombolytic,”
or surgical intervention using mechanical reperfusion 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 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 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.
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
approvals 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 ongoing and planned clinical trials,
including the MASTERS-2 Phase 3 clinical trial and the Healios
TREASURE clinical 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
David Schull
Russo Partners, LLC
Tel: (212) 845-4271 or (858) 717-2310
David.schull@russopartnersllc.com
Investor Contact:
Peter Vozzo
Westwicke Partners
Tel: 443-213-0505 or 443-377-4767 (mobile)
peter.vozzo@westwicke.com
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