Healthcare Industry News:  cell therapy 

Regenerative Medicine FDA

 News Release - September 21, 2010

Athersys Receives Orphan Drug Designation for MultiStem in Graft vs. Host Disease

Stem cell therapy in Clinical Development for Cancer Treatment Support in Patients With Leukemia or Related Blood Cancers

CLEVELAND, Sept. 21, 2010 -- (Healthcare Sales & Marketing Network) -- Athersys, Inc. (Nasdaq:ATHX ) announced today that MultiStem(R), Athersys' multipotent adult progenitor stem cell therapy, has been granted orphan drug designation by the U. S. Food and Drug Administration for the prevention of graft vs. host disease (GvHD). GvHD is a common condition associated with a bone marrow transplant in which immune cells in the transplanted marrow recognize the transplant recipient as foreign and mount an immunologic attack.

"We are extremely pleased to have been granted the benefits of orphan drug designation as we continue to advance this program in our ongoing clinical studies," said Deborah Ladenheim, Ph.D., Vice President of Regulatory Affairs of Athersys. "Complications, such as GvHD, associated with current treatment approaches to leukemia and other blood born cancers represent a significant unmet medical need. Because of MultiStem's consistent safety profile, potential for off-the-shelf administration, and multiple mechanisms of action, we feel this program could help effectively address the needs of this patient community."

MultiStem is currently in a phase 1 clinical trial to evaluate the safety of administration in support of bone marrow transplantation for the treatment of certain cancers of the blood and immune system, where GvHD is a common complication. Although partnered for select indications in other areas, such as with Pfizer for the treatment of inflammatory bowel disease, and Angiotech for the treatment of cardiovascular disease, Athersys maintains exclusive development rights to MultiStem for the oncology area, as well as a broad range of other indications.

Athersys is working with leading experts in the stem cell and bone marrow transplantation field to study MultiStem in preclinical models of radiation therapy and GvHD. In multiple preclinical models, MultiStem has been shown to be non-immunogenic, even when administered without the genetic matching that is typically required for conventional bone marrow or stem cell transplantation. Preclinical data have also shown that MultiStem suppresses the undesired T-cell-mediated immune responses that are an important factor in causing GvHD and that MultiStem treatment leads to a significant increase in survival. MultiStem administration also improved overall health and may have the potential to reduce the incidence or severity of complications associated with bone marrow transplant, including severe anemia, immunodeficiency and a substantial reduction in digestive capacity.

Orphan drug designation, which is intended to facilitate drug development, 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

MultiStem is a patented and proprietary cell therapy product consisting of a special class of stem cells that are obtained from the bone marrow or other tissue sources of healthy, consenting adult donors, which have demonstrated the ability to produce a range of factors, as well as form multiple cell types. MultiStem appears to promote tissue repair and healing in multiple ways, such as through the production of multiple therapeutic factors produced in response to signals of inflammation and tissue damage. Athersys believes that MultiStem represents a unique "off-the-shelf" stem cell product based on work that demonstrates the ability to deliver multiple mechanisms of therapeutic benefit, administration of the product without tissue matching or immunosuppression, and its capacity for large-scale production.

About Athersys, Inc.

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, a patented, adult-derived "off-the-shelf" stem cell product platform for multiple disease indications, including damage caused by myocardial infarction, bone marrow transplantation and oncology treatment support, ischemic stroke, and inflammatory bowel disease. The Company is also developing 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 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

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," "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 market acceptance of our product candidates and our ability to generate revenues, including MultiStem for the treatment of inflammatory bowel disease, acute myocardial infarction and other disease indications, and the prevention of GvHD. 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 possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete a phase II clinical trial of MultiStem for AMI; 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 realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; our ability to meet milestones under our collaboration agreements, 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.

Source: Athersys

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