FDA grants orphan drug designation for MultiStem stem cell therapy

Athersys, Inc. (Nasdaq:ATHX) announced today that MultiStem®, 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.

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