Osiris Therapeutics, Inc has achieved a $750,000 milestone payment from the Juvenile Diabetes Research Foundation (JDRF) for progress made on 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 payment was triggered after one-half of the patients in the 60-participant study were enrolled, a milestone in the funding agreement with the JDRF. The trial is also being expanded to include paediatric patients, opening enrolment to patients between 12 and 35 years of age. Osiris and the JDRF entered into a partnership in 2007 for the development of Prochymal as a therapy for newly diagnosed type-1 diabetes mellitus.
The change to the entry criteria will help ensure that the study includes the population most likely to benefit from a therapy designed to intervene in the early stages of the disease. To support expanding the age range in the trial, early, blinded data from the ongoing type-1 diabetes trial was submitted to the Food and Drug Administration (FDA) for review. Additionally, updated safety information on Prochymal from experience with paediatric patients in other indications, including the paediatric expanded access program for graft vs. host disease (GvHD), was submitted.
"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-1 diabetes," said C Randal Mills, president and chief executive officer of Osiris Therapeutics. "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 programme is remarkable and would not be possible without our patients, clinical teams, and the FDA's thoughtful and responsive assistance."
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 have the ability to delay the progression of type-1 diabetes by preserving beta cell function. In clinical trials, Prochymal has already shown promise for treating GvHD, a severe immune-mediated disease. Prochymal is currently in phase-III trials for acute and steroid-refractory GvHD, indications that have been granted Fast Track status by the FDA.
Prochymal is a preparation of mesenchymal stem cells specially formulated for intravenous infusion.
JDRF is a leader in setting the agenda for diabetes research worldwide, and is the largest charitable funder and advocate of type 1 research.
Osiris Therapeutics is the leading stem cell therapeutic company focused on developing products to treat serious medical conditions in the inflammatory, orthopedic and cardiovascular areas.