Mesoblast advancing towards Phase III trials

Australian regenerative medicine company Mesoblast has formally begun perusing FDA approval for Phase III clinical trials of its bone marrow transplant allogeneic following positive results from a trial at the University of Texas Md Anderson Cancer Centre.

The company said that the Phase III program would see the patented allogeneic, or "off-the-shelf," Mesenchymal Precursor Cells (MPCs) used under a FDA Orphan Drug Designation to expand haematopoietic stem and progenitor cell numbers in patients with haematologic malignancies.

"By increasing the overall success rate of an allogeneic bone marrow transplant while reducing the risk of graft-versus-host disease, our platform technology has the potential to lower the risk of infections, bleeding, and death in critically ill patients with haematologic malignancies following chemotherapy," said Mesoblast Chief Executive Professor Silviu Itescu.

Graft-versus-host disease afflicts around 60 percent of patients receiving bone marrow transplants from adult donors outside of their own families.

Mesoblast said that in the first 25 patients transplanted with MPC-expanded haematopoietic progenitors from cord blood, 80 percent successfully achieved the key composite endpoint at 100 days of survival with sustained engraftment of both neutrophils and platelets. By contrast only 38 percent of patients treated with non-cordal blood reach this stage, according to data from the U.S Center for International Blood and Marrow Transplant Research. Further, only four patients (16 percent ) receiving expanded cord blood during the University of Texas trial developed severe graft-versus-host-disease.