Mesoblast Ltd. said 69% of children with acute graft versus host disease were able to keep the disease at bay with the company's cell therapy MSC-100-IV, or remestemcel-L.
Graft versus host disease is a potentially fatal disease, caused by genetic incompatibility between transplanted cells and their recipient.
The phase 3 trial tested the therapy in 55 children who did not respond to steroids after the bone marrow transplant triggered the disease.
In the study, 69% of children experienced a complete or partial response, representing a statistically significant increase over historical expectations of roughly 45%.
Meanwhile, 22% of the patients in the study died. This is in contrast to day 100 mortality rates as high as 70% in patients who fail to respond to initial steroid therapy.
The treatment regimen of the therapy was well tolerated and the incidence of adverse events was consistent with that expected from the underlying disease state and in line with previous remestemcel-L use.