Mesoblast Partners With Blood and Marrow Transplant Clinical Trials Network (BMT CTN) on Pivotal Trial in Adults With SR-aGVHD

NEW YORK, Nov. 21, 2023 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the Blood and Marrow Transplant Clinical Trials Network (BMT CTN), a body including centers responsible for approximately 80% of all US allogeneic BMTs, has entered into an agreement to develop a pivotal trial of Mesoblast’s lead product candidate Ryoncil (remestemcel-L) in the treatment of adults with steroid-refractory acute graft versus host disease (SR-aGvHD). The BMT CTN is funded by the United States National Institutes of Health (NIH).

Dr John Levine, Chair-Elect of the BMT CTN Steering Committee and Professor of Internal Medicine and Pediatrics, Icahn School of Medicine at Mount Sinai, New York said: “We are delighted to be partnering with Mesoblast in this pivotal Phase 3 trial of RYONCIL, a potentially life-saving treatment for adolescents and adults with the most severe form of aGVHD. The clinical data from children treated with RYONCIL which support this trial are very compelling.”

In its September 2023 draft guidance to industry for development of agents to treat aGVHD,¹ the US Food and Drug Administration (FDA) stated that a marketing application might be supported by positive results from a single-arm trial in a population with refractory aGVHD where there are no available therapies. The trial to be developed and executed by the BMT CTN intends to evaluate RYONCIL in patients 12 and older who are refractory to both corticosteroids and a second line agent such as ruxolitinib, for whom there are no approved therapies. Prior to implementation, the clinical trial protocol will be reviewed by two independent National Heart, Lung, and Blood Institute (NHLBI)-appointed committees. Mesoblast will then submit the final protocol to FDA, as agreed at the Type A meeting with FDA in September.

Mesoblast also intends to provide FDA with additional potency assay data for RYONCIL product manufactured using the current FDA-inspected process, linking product which was used in the pediatric Phase 3 trial, which met its primary endpoint, with product which will be used in the proposed registration Phase 3 trial in adults. Showing that the product used in the pediatric and adult trials is standardized, together with data showing that future product is well characterized for commercial release, could support approval for the pediatric indication given the absence of any approved therapies for children.