Osiris Completes Enrollment in Stem Cell Trial for Type 1 Diabetes

Monday, January 25, 2010 8:01 AM

Company Achieves Fourth Milestone Payment from the Juvenile DiabetesResearch Foundation

Jan. 25, 2010 (Business Wire) -- Osiris Therapeutics, Inc. (NASDAQ:OSIR) today announced that it has achieved a $750,000 milestone payment from the Juvenile Diabetes Research Foundation (JDRF) for completing enrollment in a Phase II clinical trial evaluating Prochymal, an adult mesenchymal stem cell (MSC) therapy, as a treatment for patients recently diagnosed with type 1 diabetes. The milestone is the fourth in a series of payments resulting from JDRF's partnership with Osiris for the development of a therapy for type 1 diabetes.

In total, 63 patients were treated at 20 leading centers in this double-blind, placebo controlled trial. The study is evaluating the safety and efficacy of Prochymal in preserving insulin production in patients newly diagnosed with type 1 diabetes. The trial initially included patients 18 to 30 years of age. In July 2009, after reviewing safety data, the U.S. Food and Drug Administration (FDA) allowed Osiris to expand the trial to include pediatric patients.

"Completing the enrollment phase of this landmark trial represents a significant milestone for the development of stem cell therapies for type 1 diabetes," said C. Randal Mills, Ph.D., President and CEO of Osiris Therapeutics. "Osiris is deeply committed to developing safe and effective therapies for underserved patient populations, particularly in disease states that afflict children."

JDRF funds Osiris through its Industry Discovery and Development Partnership program, in which JDRF provides funding to companies to advance promising treatments and cures for type 1 diabetes and its complications through the drug development pipeline. In type 1 diabetes, the patient's own immune system attacks and destroys insulin-producing islet cells in the pancreas, resulting in the loss of blood-sugar control. Currently, there are no approved treatments for altering the rate of destruction of these critical islet cells, called beta cells. Preclinical studies first conducted by researchers at Genzyme found that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.

About Prochymal

Prochymal is a preparation of mesenchymal stem cells (MSCs) formulated for intravenous infusion. The MSCs utilized in Prochymal are isolated from the bone marrow of healthy young adult donors, avoiding the controversy surrounding embryonic and fetal cell sources. They are grown in culture, permitting large-scale production. Because the cells can be expanded, thousands of doses can be produced from a single donation. Studies suggest MSCs are able to safely facilitate tissue repair through a number of mechanisms. Specifically, these studies indicate that MSCs are able to down-regulate severe inflammation and work at the cellular level to rebuild damaged tissue through the coordinated release of tissue specific growth factors.

Prochymal is being evaluated in Phase III programs for steroid-refractory graft versus host disease (GvHD), acute GvHD and Crohn's disease.



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