Independent Study Finds Mesoblast’s Remestemcel-L Outperforms Ruxolitinib in SR-aGvHD

Independent Meta-Analysis Highlights Clinical Edge

Mesoblast Limited has announced compelling new evidence supporting its lead cellular therapy, remestemcel-L, for steroid-refractory acute graft-versus-host disease (SR-aGvHD). Presented at the 67th American Society of Hematology (ASH) Annual Meeting, an independent peer-reviewed meta-analysis compared remestemcel-L with the current standard treatment, ruxolitinib, across 2,732 patients from 11 studies.

The analysis found that remestemcel-L delivered superior clinical outcomes, with higher rates of complete and overall remission. This is a significant development in a challenging condition where treatment options are limited and patient prognosis is often poor. Both therapies improved quality of life, but remestemcel-L’s advantage in remission rates and a more favorable safety profile; particularly regarding hematology, cardiac, and hepatic adverse events; sets it apart.

Ryoncil – A Pioneering FDA-Approved Therapy

Remestemcel-L, marketed as Ryoncil, holds the distinction of being the first mesenchymal stromal cell (MSC) therapy approved by the U.S. Food and Drug Administration (FDA) for any indication. Notably, it is the only approved treatment for children under 12 with SR-aGvHD, underscoring its critical role in pediatric care. The therapy’s approval and recognition in the flagship ASH journal highlight its breakthrough status in the field of cellular medicine.

Mesoblast’s proprietary technology platform leverages allogeneic (off-the-shelf) MSCs that modulate immune responses and reduce damaging inflammation. This mechanism offers a novel approach compared to traditional immunosuppressants, potentially transforming treatment paradigms for inflammatory diseases.

Strategic Positioning and Future Prospects

Beyond SR-aGvHD, Mesoblast is advancing remestemcel-L for adult indications and other inflammatory conditions such as biologic-resistant inflammatory bowel disease. Additionally, its rexlemestrocel-L platform targets heart failure and chronic low back pain, reflecting a broad pipeline of cell therapies.

The company’s extensive intellectual property portfolio, with over 1,000 patents and applications protecting its cell compositions and manufacturing methods through at least 2044, provides a robust commercial moat. Coupled with industrial-scale manufacturing capabilities across Australia, the U.S., and Singapore, Mesoblast is well-positioned to meet global demand.

While the meta-analysis offers strong evidence of remestemcel-L’s clinical benefits, it is important to note that it is not a direct head-to-head clinical trial. Real-world effectiveness, regulatory approvals for adult patients, and market adoption will be key factors to watch as Mesoblast seeks to expand its footprint.