Athersys Partner HEALIOS K.K. Announces Positive Topline Results of MultiStem for Acute Respiratory Distress Syndrome in the ONE-BRIDGE Study

Athersys to continue to provide close support to Healios to advance its ARDS program

Athersys, Inc. (NASDAQ: ATHX) announced today that its partner in Japan, HEALIOS K.K. (Healios), has released positive topline data from its ONE-BRIDGE clinical trial evaluating the safety and efficacy of MultiStem^® cell therapy (invimestrocel) in patients with acute respiratory distress syndrome (ARDS). The details are provided in Healios’ press release: https://ssl4.eir-parts.net/doc/4593/tdnet/2010473/00.pdf

The ONE-BRIDGE study in Japan is an open-label trial with two patient cohorts – one, with 30 pneumonia-induced ARDS subjects including treatment and registry groups, and a second, with five COVID-induced ARDS subjects. Healios reported favorable topline results for both cohorts relative to the registry and expectations for these patients, including the primary endpoint of ventilator-free days (VFD) and the secondary endpoint of 90-day mortality. These data are in line with the results from Athersys’s placebo-controlled MUST-ARDS study, conducted with a similar patient population in the United States and the United Kingdom. MultiStem cell therapy (HLCM051) has been designated in Japan as an orphan regenerative medicine product for use in the treatment of ARDS.

"We are excited by the results reported by our partner, Healios," commented Mr. William (B.J.) Lehmann, Jr., President, and Interim CEO of Athersys. "We continue to believe that MultiStem administration can have a meaningful therapeutic impact for ARDS patients. We look forward to continuing to support Healios as it moves the ARDS program forward in Japan and as we make progress elsewhere."

Athersys is currently conducting its MACoVIA study, a double-blind, placebo-controlled study evaluating MultiStem administration in patients with ARDS resulting from COVID-19 and other pathogens. Athersys’s ARDS program has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the United States Food and Drug Administration.

About ARDS

Acute respiratory distress syndrome (ARDS) is a serious immunological and inflammatory condition characterized by widespread inflammation in the lungs. ARDS can be triggered by COVID-19, pneumonia, sepsis, trauma, or other events and represents a major cause of morbidity and mortality in the critical care setting. It has significant implications, as it prolongs intensive care unit (ICU), and hospital stays and requires convalescence in the hospital and rehabilitation. There are limited interventions and no effective drug treatments for ARDS, making it an area of high unmet clinical need with high treatment costs. Given these high treatment costs, a successful cell therapy could be expected to generate significant savings for the healthcare system by reducing days on a ventilator and in the ICU and, importantly, could reduce mortality and improve quality of life for those suffering from the condition.

About MultiStem^® (invimestrocel)

MultiStem^® cell therapy (invimestrocel) is a patented regenerative medicine product candidate 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 may distinguish it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. MultiStem represents a unique "off-the-shelf" stem cell product candidate 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 favorable outcome data, its novel mechanisms of action, and favorable and consistent tolerability data in clinical studies, we believe that MultiStem therapy may 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 MultiStem cell therapy toward commercialization. More information is available at www.athersys.com. Follow Athersys on Twitter at www.twitter.com/athersys.

About Healios:

Healios is Japan’s leading clinical-stage biotechnology company harnessing the potential of stem cells for regenerative medicine. It aims to offer new therapies for patients suffering from diseases without effective treatment options. Healios is a pioneer in the development of regenerative medicines in Japan, where it leverages its proprietary, gene-edited “universal donor” induced pluripotent stem cell (iPSC) platform technology to develop next-generation regenerative treatments across several domains. Healios combines its deep iPSC and gene editing expertise to make innovative engineered cell therapeutics including HLCN061, its functionally enhanced NK cell product candidate to treat solid cancer indications. Healios’ near-term pipeline includes the somatic stem cell product HLCM051 (MultiStem), which is currently being evaluated in Japan in Phase 2/3 and Phase 2 clinical trials for ischemic stroke and acute respiratory distress syndrome, respectively. Healios was established in 2011, has over 140 people in its Tokyo and Kobe operations, and has been listed on the Tokyo Stock Exchange since 2015 (TSE Mothers: 4593). https://www.healios.co.jp/en.

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; 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 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.

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