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, in the treatment of adults with steroid-refractory acute graft versus host disease (SR-aGvHD). The BMT CTN, funded by the United States National Institutes of Health (NIH), is a body comprising centers responsible for approximately 80% of all US allogeneic BMTs. The trial, which will evaluate Ryoncil in patients aged 12 and older who are refractory to corticosteroids and a second line agent, aims to address the lack of approved therapies for this condition. Mesoblast also plans to submit additional potency assay data for Ryoncil to the FDA to support its approval for the pediatric indication.
Dr. John Levine, Chair-Elect of the BMT CTN Steering Committee, expressed excitement about the partnership and praised the clinical data from children treated with Ryoncil. He referred to the treatment as potentially life-saving for individuals with the most severe form of aGVHD. Mesoblast CEO Silviu Itescu also expressed satisfaction with the collaboration and emphasized the company’s goal of providing a treatment option for adults suffering from aGVHD who have no other approved therapies. Mesoblast, a world leader in allogeneic cellular medicines, utilizes proprietary technology to develop product candidates for inflammatory diseases, such as aGVHD, inflammatory bowel disease, and acute respiratory distress syndrome. The company has a strong global intellectual property portfolio and commercial partnerships for Phase 3 assets in Europe and China.
This partnership with the BMT CTN represents a significant step forward for Mesoblast in advancing their lead product candidate, Ryoncil, in the treatment of adults with SR-aGvHD. This trial will provide crucial data to support potential FDA approval and address the unmet medical need for patients who do not respond to existing therapies. Additionally, the company’s commitment to providing additional potency assay data for Ryoncil manufactured using the FDA-inspected process demonstrates their dedication to ensuring the standardized and well-characterized nature of the product. With their expertise and global presence, Mesoblast continues to be at the forefront of developing innovative cellular therapies for severe and life-threatening inflammatory conditions.