newsfilter
← Latest News
GlobeNewswire·

Mesoblast and BMT CTN to Initiate Pivotal Trial of Ryoncil® as Part of First-Line Regimen in Adults with Severe Acute GVHD Refractory to Steroids

MESO
  • Mesoblast Limited & the NIH-funded Blood and Marrow Transplant Clinical Trials Network (BMT CTN) will collaborate on a pivotal Phase 3 trial of Ryoncil (remestemcel-L-rknd) for adults with severe steroid-refractory acute graft versus host disease (SR-aGvHD).
  • The trial aims to evaluate Ryoncil as part of a first-line regimen for patients failing corticosteroids, addressing high mortality rates & poor response to existing therapies like ruxolitinib.
  • Dr. John Levine of the BMT CTN Steering Committee stated the partnership seeks to extend Ryoncil's FDA-approved use from pediatric patients to adults with severe SR-aGvHD.
  • Existing data shows 44-58% of adults treated with ruxolitinib did not achieve response at Day 28, with survival rates as low as 20-30% by 100 days in patients failing ruxolitinib.
  • Expanded Access program data indicates 76% survival at Day 100 for patients aged 12+ with SR-aGvHD who failed ruxolitinib or other second-line agents.
  • The trial protocol, developed following FDA discussions, will randomize patients early after corticosteroid refractoriness to receive ruxolitinib alone or combined with Ryoncil.
  • Mesoblast plans to submit the trial protocol to the FDA to initiate enrollment in the first quarter of 2026.
  • CEO Silviu Itescu noted the trial addresses a major unmet need, with the adult SR-aGvHD market opportunity estimated to be 3-4 times larger than the pediatric market.
  • Ryoncil is currently the first FDA-approved mesenchymal stromal cell (MSC) therapy for steroid-refractory acute graft versus host disease in pediatric patients aged 2 months and older.
  • Mesoblast holds over 1,000 granted patents or patent applications protecting its technology, with commercial protection extending through at least 2044.
  • The BMT CTN represents U.S. centers performing approximately 80% of all U.S. allogeneic blood and marrow transplants.