Primary Endpoint Successfully Achieved in Mesoblast’s Phase 3 Cell Therapy Trial for Acute Graft Versus Host Disease

Mesoblast (Nasdaq:MESO) announced that the Phase 3 trial of its allogeneic mesenchymal stem cell product candidate MSC-100-IV (remestemcel-L) in children with steroid refractory acute Graft versus Host Disease (aGVHD) has successfully met the primary endpoint of Day 28 overall response (OR, complete + partial response) rate.

As quoted in the press release:

In the 55 children enrolled in Mesoblast’s open-label Phase 3 trial conducted across 32 sites in the United States, the Day 28 OR rate was 69%, a statistically significant increase compared to the protocol-defined historical control rate of 45% (p=0.0003).

Among patients who received at least one treatment infusion and were followed up for 100 days (n=50), the mortality rate was 22%.  This is in contrast to Day 100 mortality rates as high as 70% in patients who fail to respond to initial steroid therapy^1,2,3.

Click here to read the full press release.