Mesoblast Limited, a global leader in allogeneic cellular medicines for inflammatory diseases, has announced a partnership with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to develop a pivotal trial of its lead product candidate, Ryoncil (remestemcel-L). The trial aims to evaluate the effectiveness of Ryoncil in treating adults with steroid-refractory acute graft versus host disease (SR-aGvHD). The BMT CTN, funded by the United States National Institutes of Health (NIH), includes centers responsible for 80% of all US allogeneic bone marrow transplants. Dr. John Levine, Chair-Elect of the BMT CTN Steering Committee, expressed enthusiasm for the potential life-saving treatment provided by Ryoncil.
The US Food and Drug Administration (FDA) has previously stated that marketing approval could be supported by positive results from a single-arm trial in a population with no available therapies. The trial, to be developed and executed by the BMT CTN, will assess the effectiveness of Ryoncil in patients aged 12 and older who are refractory to both corticosteroids and a second-line agent. Prior to implementation, the trial protocol will be reviewed by two independent committees appointed by the National Heart, Lung, and Blood Institute (NHLBI). Mesoblast also plans to submit additional potency assay data for Ryoncil to the FDA, demonstrating the standardization and characterization of the product to support approval for the pediatric indication. Mesoblast CEO Silviu Itescu expressed pleasure in partnering with the BMT CTN to provide a treatment option for adults with SR-aGvHD who have no other approved therapies.