Ann: Remestemcel-L Improves Survival in High-Risk Acute GvHD, page-115

Summary of the study outcomes
The study compared outcomes in 25 children from Mesoblast’s Phase 3 trial of remestemcel-L in SRaGVHD with 27 closely matched children from the Mount Sinai Acute GVHD International Consortium
(MAGIC)2 who participated in a prospective natural history study and were matched for the Phase 3
trial entry criteria. The objective of the study was to evaluate whether outcomes differed according to
treatment with remestemcel-L vs other therapies in children at highest risk of death, namely those
with baseline MAGIC Algorithm Probability (MAP) biomarker levels ≥0.291, a level predictive of very
high mortality and poor responses to therapy in SR-aGVHD. MAP combines the serum concentrations
of two biomarkers, Reg3α and ST2, into a single value that predicts long-term outcomes and
significant GI tract damage.
MAP levels ≥0.291 were present in 48% of remestemcel-L treated children (12/25) and 37% of the
MAGIC cohort (10/27). Treatment with remestemcel-L resulted in 67% Day 28 Overall Response and
64% Day 180 overall survival compared with 10% Day 28 Overall Response and 10% Day 180
survival in the MAGIC cohort (both p=0.01) when treated with various biologics, including ruxolitinib.
These results extend previous observations showing that children who achieved clinically meaningful
responses and survival after treatment with remestemcel-L had significant reductions in the ST2
biomarker of inflammation, consistent with healing of the GI tract.