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Mesoblast Limited's First Patients In Bone Marrow Transplant Trial Show Earlier Engraftment

April 09, 2017

Australian regenerative medicine company Mesoblast Limited has announced successful results from the first five patients who underwent bone marrow transplantation with haematopoietic stem and progenitor cells expanded by the patented allogeneic, or "off-the-shelf", Mesenchymal Precursor Cells (MPCs).

The Phase I/II trial in up to 30 patients is being conducted by Mesoblast's US-based associated company Angioblast Systems Inc. at the University of Texas M. D. Anderson Cancer Center, Department of Stem Cell Transplantation and Cellular Therapy. The trial is funded through a grant awarded by the United States National Institutes of Health (NIH).

Successful bone marrow reconstitution and engraftment was achieved in all five patients with haematologic malignancies who received MP! C-expanded haematopoietic stem and progenitor cells from cord ! blood, w ith no cell-related adverse events. The median time to engraftment was 15 days, approximately two weeks faster than expected without MPC expansion.

The MPC product used in this trial is being developed under a United States Food and Drug Administration (FDA) orphan drug designation recently granted to Angioblast Systems Inc. for expanding haematopoietic stem and progenitor cell numbers in patients with haematologic malignancies.

Executive Director Professor Silviu Itescu said these initial results achieved with the Angioblast allogeneic MPCs were extremely encouraging.

"By significantly reducing the time to engraftment and increasing the overall success rate of an allogeneic bone marrow transplant, this technology has the potential to lower the risk of infections, bleeding, and death in critically ill patients with haematologic malignancies following chemotherapy," he said.

In view of the important nature of the unmet medical need,! the Company will seek to obtain US FDA clearance to commence an accelerated Phase 3 program if subsequent patients in the trial continue to show enhanced bone marrow engraftment potential.

"This would represent a significantly shortened timetable to product commercialisation", added Professor Itescu.

About Orphan Drug Designation

Orphan drug designation is reserved for therapies which are being developed for conditions affecting up to 200,000 patients annually in the US, and allows for an accelerated review process by the FDA, seven-year market exclusivity in the US upon obtaining marketing authorisation, tax benefits, and exemption from user fees.

Mesoblast Limited