High Survival Rates With Ryoncil® in EIND Program Emphasize Importance of Earlier Use in Both Children and Adults With SR-aGvHD

Core Insights - Mesoblast Limited presented data on Ryoncil (remestemcel-L-rknd) at the February 2026 Tandem Meetings, highlighting its effectiveness in treating steroid-refractory acute graft-versus-host disease (SR-aGvHD) in both children and adults [1][2]. Group 1: Treatment Efficacy - Ryoncil demonstrated high survival outcomes in SR-aGvHD patients, regardless of age or treatment line, and is the first FDA-approved mesenchymal stromal cell product for this indication [2][6]. - In a study of 53 patients receiving Ryoncil as a third-line treatment, 15% died before completing the treatment, compared to only 2% in the Phase 3 trial for second-line treatment, indicating the importance of early intervention [3]. - Adult patients in the Emergency Investigational New Drug program showed survival rates comparable to children, supporting the rationale for a pivotal trial of Ryoncil in adults [4]. Group 2: Future Developments - A pivotal trial for Ryoncil's use in adults with severe SR-aGvHD is expected to begin enrollment this quarter, pending protocol approval [4]. - If successful, this trial could lead to an extension of Ryoncil's label for adult use, which represents a market approximately three times larger than that of pediatric patients [4]. Group 3: Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [5][7]. - The company has a robust intellectual property portfolio with over 1,000 patents or applications, providing commercial protection until at least 2044 [8]. - Mesoblast is also developing additional therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [7].