Results show therapy was well-tolerated and
support potential benefits in mortality, ventilator-free days,
ICU-free days and quality of life outcomes
Separately, data pooled from two independent
studies provide further evidence of potential clinically meaningful
benefit of MultiStem as a treatment for ARDS patients
Athersys, Inc. (NASDAQ: ATHX) announced today that a manuscript
reporting data from the Company’s MUST-ARDS clinical trial have
been published in the peer-reviewed journal Intensive Care
Medicine. MUST-ARDS was a randomized, double-blind
placebo-controlled Phase 1/2 trial evaluating the safety and
efficacy of MultiStem® (invimestrocel) cell therapy in patients
with acute respiratory distress syndrome (ARDS). Enrolling patients
at 12 intensive care units in the United Kingdom and the U.S., the
study confirmed that intravenous administration of MultiStem cell
therapy was well-tolerated among critically ill patients early in
the course of moderate to severe ARDS. The trial also assessed
multiple efficacy endpoints, including mortality, liberation from
mechanical ventilation, discharge from intensive care, and quality
of life (QoL) among survivors over one full year of recovery.
Mechanisms of action were further explored by comparing acute
changes in plasma inflammatory and lung injury biomarkers in
MultiStem-treated and placebo-treated study participants. The
results of this study provided the basis for the Food and Drug
Administration (FDA) to grant the Company’s ARDS program
Regenerative Medicine Advanced Therapy (RMAT) and Fast Track
designation. The open access publication can be found online at the
following link:
https://link.springer.com/content/pdf/10.1007/s00134-021-06570-4.pdf.
Highlights from the publication, including new and previously
disclosed data:
- MultiStem treatment was well-tolerated in this clinical trial,
with no allergic or serious adverse reactions associated with the
cell therapy in any cohort through one year of follow-up;
- Notably higher median ICU-free and ventilator-free days (VFDs)
among MultiStem cell recipients compared to the placebo group at
the 28-day benchmark;
- Lower all-cause mortality in the MultiStem-treatment group
compared to the placebo group;
- QoL outcomes, assessed by the EQ-5D-3L at days 28, 90, and 365,
showed greater recovery among survivors who received MultiStem
treatment compared to those who received placebo;
- Decreases in several pro-inflammatory plasma biomarkers were
observed in the cell treatment group through Day 7 compared with
increases among placebo recipients; and
- Similar acute plasma biomarker responses to MultiStem,
including decreases in pro-inflammatory cytokines IL-1beta, TNFα,
IL-6, IFN-gamma and IL-2, have been observed following treatment
for ischemic stroke.
In addition (not reported in the published manuscript),
preliminary analyses of the data pooled from the MUST-ARDS study
and the recently completed ONE-BRIDGE study, which was conducted by
Athersys’ partner, HEALIOS K.K. (Healios), in Japan, further
supports potential clinically meaningful benefit of MultiStem as a
treatment for ARDS patients. These pooled randomized data sets
include 40 non-COVID ARDS patients treated with MultiStem cell
therapy and 20 ARDS patients treated with placebo or receiving
standard of care. Analysis of covariance, adjusting for baseline
severity, age, and study participation, revealed an estimate of 5.4
greater (90% confidence interval 0.48-10.32; two-sided p=0.07) VFDs
among MultiStem recipients compared to the control group. Similar
analyses supported lower mortality in the MultiStem treatment group
as well. The pooled data analyses can be found on the Company’s
corporate presentation at this link:
https://s23.q4cdn.com/674737627/files/11-02-2021-ATHX-Corp-Investor-Presentation.pdf.
“The MUST-ARDS study, completed before the emergence of COVID-19
as a human disease, was an important contribution to the field of
cell therapy in the treatment of ARDS. The trial confirmed
tolerability of MultiStem infusion in critically ill adults with
severe hypoxemic respiratory failure, and often with other
coincident organ failure syndromes, and revealed very encouraging
signs of improved outcomes that matter to patients and their
families,” commented Dr. Eric Jenkins, study co-author, Senior
Medical Director and Head of Clinical Programs at Athersys.
“The results of this study, previously presented only as an oral
abstract, provided the basis for the advancement of Healios’
ONE-BRIDGE ARDS trial and our own MACoVIA pivotal trial of
MultiStem for the treatment of ARDS resulting from COVID and other
severe infections. While the study was small, and therefore not
powered to provide definitive conclusions about efficacy, the trial
provided a strong foundation of safety and persuading support that
MultiStem could be an effective immunomodulatory therapy for this
previously untreatable and high morbidity inflammatory lung injury
syndrome. These results encouraged Athersys, Healios, our clinical
collaborators, and others in the field to advance cell therapy for
the treatment of ARDS into confirmatory and late-stage clinical
trials. It is important that these results are now available in the
peer-reviewed literature. I would like to thank our co-authors, the
MUST-ARDS clinical research teams, and, most importantly, the
patients and families, who on their behalf chose, in their most
desperate vulnerability, to contribute to the advancement of
medical care for future patients,” concluded Dr. Jenkins.
There remains a strong need for a safe treatment option that can
reduce the mortality of patients who develop ARDS, speed recovery,
and improve the quality of life for survivors. The MUST-ARDS study
has shown safety and tolerability of intravenous administration of
MultiStem at doses up to 900 million cells in patients with ARDS.
The study data, along with preclinical data, suggest that MultiStem
may modulate multiple host responses to tissue injury
simultaneously, including down-regulation of hyperinflammatory
responses, stimulation of tissue repair, and restoration immune
system balance, and support the rationale to conduct an additional
larger size study to evaluate efficacy. The Company’s MACoVIA study
is a multi-center, randomized, double-blind, placebo-controlled,
Phase 2/3 trial to investigate the therapeutic efficacy of
MultiStem for the treatment of pathogen-induced ARDS.
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 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. The
following risks and uncertainties 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: our ability to raise capital to fund
our operations, including but not limited to, the timing and nature
of results from MultiStem clinical trials, including the MASTERS-2
Phase 3 clinical trial evaluating the administration of MultiStem
for the treatment of ischemic stroke, and the Healios TREASURE and
ONE-BRIDGE clinical trials in Japan evaluating the treatment in
stroke and ARDS patients, respectively, including the timing of the
release of data by Healios from its clinical trials, which could be
delayed by, among other things, the regulatory process with the
PMDA; the success of our MACOVIA clinical trial evaluating the
administration of MultiStem for the treatment of COVID-19 induced
ARDS, and the MATRICS-1 clinical trial being conducted with The
University of Texas Health Science Center at Houston evaluating the
treatment of patients with serious traumatic injuries; the impact
of the COVID-19 pandemic on our ability to complete planned or
ongoing clinical trials; the possibility that the COVID-19 pandemic
could delay clinical site initiation, clinical trial enrollment,
regulatory review and the potential receipt of regulatory
approvals, payment of milestones under our license agreements and
commercialization of one or more of our product candidates, if
approved; the availability of product sufficient to meet commercial
demand shortly following any approval, such as in the case of
accelerated approval for the treatment of COVID-19 induced ARDS;
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 impact of the COVID-19 pandemic on the production
capabilities of our contract manufacturing partners and our
MultiStem trial supply chain; the possibility of delays, work
stoppages or interruptions in manufacturing by third parties or us,
such as due to material supply constraints, contamination,
operational restrictions due to COVID-19 or other public health
emergencies, labor constraints, regulatory issues or other factors
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 neurological, inflammatory and immune,
cardiovascular and other critical care 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; the success of our
competitors and the emergence of new competitors; and the risks
mentioned elsewhere in our Annual Report on Form 10-K for the year
ended December 31, 2020 under Item 1A, “Risk Factors” and our other
filings with the SEC. You should not place undue reliance on
forward-looking statements contained on our website and/or on our
accounts on Twitter, Facebook, LinkedIn or other social media
platforms, 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/20211130005361/en/
William (B.J.) Lehmann Interim CEO, President and Chief
Operating Officer Tel: (216) 431-9900 bjlehmann@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
Athersys (NASDAQ:ATHX)
Historical Stock Chart
From Apr 2024 to May 2024
Athersys (NASDAQ:ATHX)
Historical Stock Chart
From May 2023 to May 2024