Mesoblast Announces Successful Interim Futility Analysis In Phase 3 Trial For Acute Graft Versus Host Disease

NEW YORK and MELBOURNE, Australia, Nov. 14, 2016 (GLOBE NEWSWIRE) -- Mesoblast Limited (ASX:MSB) (Nasdaq:MESO) today announced that the Phase 3 trial of its intravenous product candidate MSC-100-IV used as front-line therapy in children with steroid-resistant acute graft versus host disease (aGVHD) had been successful in a pre-specified interim futility analysis.

Enrollment in the 60-patient open label Phase 3 trial is ongoing across multiple sites in the United States, trial completion is expected in the first half of 2017, and commercial launch activities are underway.

The independent Data Safety Monitoring Board (DSMB) notified Mesoblast that an interim analysis showed that the predefined Bayesian futility rule used to determine the probability of the trial's success using the trial's primary endpoint of Day 28 overall response had been passed. The analysis method determined the likelihood of obtaining a statistically significant treatment effect at study completion, based on the data observed at this interim time point.

There are currently no products approved in the United States for this disease. Japan is the only jurisdiction where this therapy is available, through Mesoblast's licensee JCR Pharmaceuticals Co. Ltd.  Based on guidance from the United States Food and Drug Administration (FDA), Mesoblast believes that positive data from this Phase 3 trial may be sufficient for filing for accelerated approval of MSC-100-IV in the United States. Mesoblast plans to broaden its use in adult patients with high-risk steroid-refractory aGVHD.

"We are pleased that Mesoblast has attained such an important milestone in making its product available for the potential treatment of steroid-refractory acute graft versus host disease, a serious and life threatening condition that has a very urgent need for effective therapies," said Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center and the lead investigator on the ongoing Phase 3 trial.