Osiris Therapeutics, Inc. (NASDAQ: OSIR) today announced preliminary results for two phase III trials evaluating Prochymal for the treatment of acute graft versus host disease (GvHD). GvHD, the most common complication of bone marrow transplantation, is a life-threatening disease for which there is currently no approved treatment. Prochymal showed significant improvements in response rates in difficult-to-treat liver and gastrointestinal GvHD, however neither trial reached its primary endpoint.
Key findings from the GvHD trials include:
- There was no statistical difference between Prochymal and placebo on the primary endpoints for either the steroid-refractory (35% vs. 30%,>
- The primary endpoint for the steroid-refractory GvHD trial (durable complete response) for the per-protocol population approached statistical significance (40% vs. 28%,>
- In patients with steroid-refractory liver GvHD, treatment with Prochymal significantly improved response (76% vs. 47%,>
- Prochymal significantly improved response rates in patients with steroid-refractory gastrointestinal GvHD (88% vs. 64%,>
- In pediatric patients, Prochymal showed a strong trend of improvement in response rates (86% vs. 57%,>
“These data are still preliminary and further analysis is needed to gain a full appreciation of the results of these rigorous, double-blind, placebo-controlled trials,” said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris Therapeutics. “We are encouraged to see Prochymal significantly improve response rates above standard of care in GvHD patients who currently have no good treatment options. We will meet with the FDA as soon as possible to discuss the most appropriate and efficient path forward for Prochymal in this life-threatening indication.”