Today Osiris Therapeutics announced a $750,000 milestone payment from the Juvenile Diabetes Research Foundation (JDRF) for progress in its Phase II clinical trial for the treatment of Type I diabetes. The clinical trial is testing the company’s proprietary product, Prochymal, which is formulated from adult mesenchymal stem cells (MSCs). The milestone payment was triggered when one-half of the patients were enrolled in the study, which has a target enrollment of 60 participants. Additionally, the clinical trial is now being expanded to include pediatric patients and is also conducting enrollment for patients between 12 and 35 years of age.
According to C. Randal Mills, Ph.D., president and CEO of Osiris, "We are proud to be working alongside the JDRF in this landmark trial to evaluate the role of mesenchymal stem cells in patients with Type I diabetes. Caring for kids with limited medical options is a very special part of life at Osiris and we take that responsibility seriously. The progress we are making in this clinical program is remarkable and would not be possible without our patients, clinical teams, and the FDA’s thoughtful and responsive assistance."
Osiris and the JDRF originally entered into the partnership in 2007, for the development of Prochymal as a therapy for Type I diabetes, especially in the early stages of the disease when it has been newly diagnosed.
One of the many autoimmune diseases, Type I diabetes is characterized by the body’s destruction of its own beta islet cells – the cells of the pancreas which produce insulin. Conventional medical therapies currently offer no known effective treatment for the disease, but adult stem cell therapy may now offer the first therapy which not only reverses the course of diabetes but also regenerates lost beta cells. MSCs were shown in a preclinical trial conducted by Genzyme to preserve beta cell function, and a number of other studies have also demonstrated that MSCs have the ability to generate new cells that resemble beta islet cells in morphology and function.
In addition to this Phase II clinical trial for Type I diabetes, Prochymal is also currently in Phase III clinical trials for acute steroid-refractory graft-versus-host-disease (GvHD), as well as Phase II clinical trials for the treatment of acute myocardial infarction, Crohn’s disease, and chronic obstructive pulmonary disease (COPD). Prochymal is the only stem cell product currently on the market which has been granted both Fast Track and Orphan status by both the U.S. FDA (Food and Drug Administration) and the European Medicines Agency. The MSCs contained in Prochymal are obtained from the bone marrow of healthy adult donors and are specially formulated according to a proprietary process for intravenous infusion. Osiris is also developing another adult stem cell product, Chondrogen, which is currently in clinical trials for the treatment of osteoarthritis of the knee.
A leader in adult stem cell therapies, Osiris Therapeutics is focused on the development of products for the treatment of inflammatory, orthopedic and cardiovascular diseases. In November of last year, Osiris formed a strategic alliance with the biotech company Genzyme that was valued at over $1.3 billion. In 2007, the two companies were awarded a $224.7 million contract from the U.S. Department of Defense for the development of Prochymal in the treatment of radiation sickness.
(Please see a number of related news articles on this website pertaining to Osiris Therapeutics, including, "Adult Stem Cells Treat Diabetes", dated June 29, 2009).