Mesoblast (ASX:MSB) has announced aggregated results from 309 children treated with RYONCIL (remestemcel-L) have been presented at the American Society for Transplantation Cellular Therapy and the Center for International Blood and Bone Marrow Transplant Research (TCT) meeting in Florida.
The company said the data showed treatment with RYONCIL across three separate trials resulted in consistent treatment responses and survival outcomes in children with steroid-refractory acute graft versus host disease (SR-aGVHD).
It said findings include: consistent safety and efficacy were observed across the continuum from first-line treatment after steroid failure through the most challenging patients who received RYONCIL as salvage after exhausting all other options; in the aggregated dataset, 204 of the 309 (66 per cent) patients achieved an overall response at Day 28 following a four-week course of RYONCIL; results were consistent across all grades of disease, including most severe (IBMTR Grade C/D or Glucksberg Grade 3/4); in the most severe patients (Grade C/D), who accounted for 82 per cent of all treated patients, Day 28 overall response was 65 per cent; overall response at Day 28 was strongly predictive of survival at Day 100 and Day 180; day 28 responders were more than twice as likely to survive as non-responders (84 per cent vs 39 per cent at Day 100, and 83 per cent vs 38 per cent at Day 180; and, RYONCIL was well tolerated with no infusion-related toxicity and no identified safety concerns.
According to Mesoblast chief medical officer Dr Fred Grossman, “These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease.
"If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.”
In January, Mesoblast filed a Biologics License Application (BLA) to the US FDA for RYONCIL for the treatment of children with steroid-refractory aGVHD. The company has requested Priority Review of the BLA by the FDA under the product candidate’s existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.