Core Insights - Mesoblast Limited is collaborating with the NIH-funded Blood and Marrow Transplant Clinical Trials Network to conduct a pivotal trial of Ryoncil for adults with severe steroid-refractory acute graft versus host disease (SR-aGvHD) [1][2][3] - The trial aims to address the high mortality rates in patients who do not respond to corticosteroids, with survival rates as low as 20-30% by Day 100 after failing ruxolitinib [2] - Ryoncil has shown promising results in pediatric patients, with a 76% survival rate at Day 100 in adults who failed other treatments [2][5] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory diseases, utilizing a proprietary mesenchymal lineage cell therapy technology platform [4][6] - The company has a strong intellectual property portfolio with over 1,000 patents or applications, providing commercial protection until at least 2044 [7] - Mesoblast is expanding its product offerings, with Ryoncil being developed for additional indications beyond SR-aGvHD, including biologic-resistant inflammatory bowel disease and heart failure [6] Clinical Development - The pivotal trial for Ryoncil will randomize patients as early as possible after corticosteroid refractoriness, comparing ruxolitinib alone versus ruxolitinib combined with Ryoncil [3] - The trial protocol is set to be submitted to the FDA to initiate enrollment in the first quarter of 2026 [3] - The collaboration with BMT CTN is significant as it represents U.S. centers performing approximately 80% of all U.S. allogeneic bone marrow transplants [1][10]