Athersys Confirms Completion of Patient Enrollment for Its Phase I Study of MultiStem(R) for Hematopoietic Stem Cell Transplant Support

Targeting Risk for Graft Versus Host Disease

CLEVELAND, OH, USA | October 17, 2011 | Athersys, Inc. (Nasdaq:ATHX - News) today announced that it has completed patient enrollment of the repeat dose arm of its clinical study involving administration of MultiStem(R) to patients being treated for leukemia or other blood born cancers. Initial results from the repeat dose arm of this trial will be announced following the completion of patient evaluation periods and subsequent analysis of data around the end of the year.

"We look forward to evaluating the data generated from this study and expect to provide initial results from the multiple dose arm of the trial, as we did for the single ascending dose arm in May of this year," commented Dr. Robert Deans, Executive Vice President of Regenerative Medicine at Athersys. "We believe that MultiStem could have broad relevance for providing support in a clinical transplant setting. Approximately 25,000 patients annually undergo hematopoietic transplant procedures that put them at risk for life threatening graft versus host disease (GvHD). With an understanding of the safety and dose profile of MultiStem in these patients, we will be able to conduct clinical evaluation of the efficacy of the immunomodulatory and other properties of MultiStem in subsequent studies. A therapy that could meaningfully reduce the incidence and/or severity of GvHD without increasing relapse or infectious risk in HSCT patients would provide substantial clinical benefits."

The Phase I clinical trial is an open label, multi-center dose escalation trial evaluating the safety and maximum tolerated dose of a single or repeat dose administration of allogeneic MultiStem delivered intravenously following a traditional donor-derived hematopoietic stem cell transplant. Patients enrolled in the study received either a low, medium or high dose of MultiStem for both the single and repeat dose arms. The study is being conducted at bone marrow transplant centers in the United States, including Oregon Health & Science University Medical Center, Texas Transplant Institute, University Hospitals Case Medical Center, University of Pennsylvania, Mayo Clinic Arizona, as well as the Katholieke Universiteit Leuven in Belgium.

In May 2011, Athersys announced positive interim results for the single dose arm of this Phase I study. These results demonstrated that MultiStem was well tolerated at all dose levels, and also suggested that the product may reduce the incidence of GvHD as compared to historical clinical experience. Over the 100-day observation period, there was a low cumulative incidence of acute GvHD for all subjects enrolled (n=18, 28% grade II-IV, 6% grade III-IV) and, for patients receiving the high dose (n=9), there was just one case of grade II GvHD and no cases of grades III-IV GvHD. Completion of data analysis for the completed repeat dose arm for safety parameters and secondary efficacy measures including GvHD incidence is anticipated later this year.

GvHD is one of the major limitations of donor-derived hematopoietic stem cell transplants. This complication is a significant cause of morbidity and mortality and is thought to be initiated by activation of donor immune cells, such as activated T-cells, that attack host cells in the transplant recipient as foreign tissue. Acute GvHD is associated with damage to the liver, skin, gastrointestinal tract and other tissues. Moderate to severe GvHD (with grade II being moderate and grade III--IV GvHD being severe) occurs in 30-50% of matched related hematopoietic stem cell transplants (HSCT) and 50-70% of matched unrelated donor recipients, as shown in the literature describing historical clinical experience. Patients that receive a peripheral blood stem cell transplant are at higher risk for GvHD, relative to bone marrow derived hematopoietic stem cells. Severe GvHD requires treatment using intense immunosuppression with steroids and additional agents, and patients may develop serious infections as a result of such immunosuppression.

In September 2010, Athersys received orphan drug designation from the U.S. Food and Drug Administration for the prevention of GvHD. Orphan drug designation, which is intended to encourage and facilitate drug development for products designed to treat rare diseases, also provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.

About MultiStem(R)

MultiStem is a patented and proprietary allogeneic cell therapy product candidate that can be manufactured on a large scale, frozen and stored for an extended period, and subsequently thawed and administered intravenously, similar to traditional biologics. MultiStem consists of a clinical grade preparation of non-embryonic stem cells obtained from bone marrow that have the potential to produce a range of factors and form multiple cell types. MultiStem appears to work through several mechanisms that promote healing and tissue repair, but a primary mechanism appears to be the production of therapeutic proteins and other molecules produced in response to inflammation and tissue damage. Athersys believes that MultiStem may represent a unique "off-the-shelf" stem cell product based on its apparent ability to be used without tissue matching and its capacity for large scale production.

About Athersys

Athersys is a clinical stage biopharmaceutical 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 MultiStem(R), a patented, adult-derived "off-the-shelf" stem cell product platform for multiple disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The Company currently has several clinical stage programs, including for treating damage caused by myocardial infarction, bone marrow transplantation and oncology treatment support, ischemic stroke, and inflammatory bowel disease. The Company also has developed a portfolio of other therapeutic programs, including orally active pharmaceutical product candidates for the treatment of metabolic and central nervous system disorders, utilizing proprietary technologies, including Random Activation of Gene Expression (RAGE(R)). Athersys has forged several key strategic alliances and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at

SOURCE: Athersys, Inc.

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