Phase 3 Bone Marrow Transplant Trial, Mesoblast (ASX:MSB; ADR:MBLTY)
Australian, Mesoblast Limited (ASX:MSB; ADR:MBLTY), provided guidance on its Phase 3 bone marrow transplant program following a formal meeting with the US FDA.
The FDA provided guidance on Phase 3 primary endpoints and duration of patient follow-up.
- As a result of the positive meeting, and to ensure full alignment on product approval requirements, Mesoblast will seek to obtain from the FDA a binding Special Protocol Assessment (SPA) prior to commencing the Phase 3 trial,
- The SPA provides an agreement regarding the design, including size and clinical endpoints, of the pivotal trial to support an efficacy claim in a Biologic License Application (BLA).
This Phase 3, the patented allogeneic, or “off-the-shelf”, adult Mesenchymal Precursor Cells (MPCs) will be used under a United States FDA Orphan Drug Designation to expand unrelated donor haematopoietic stem and progenitor cell numbers for use in patients with haematologic malignancies.
- In the meeting with the FDA, MSB proposed a Phase 3 clinical trial whose design, size, duration, and primary endpoints were based on results from the 25 patient pilot trial performed at the University of Texas MD Anderson Cancer Center,
- Comparable control data were obtained from both MD Anderson Cancer Center and a United States registry of 300 patients collected by the Center for International Blood and Marrow Transplant Research
- MSB’s objective is to make available a source of unrelated donor bone marrow cells which can be used without full matching to effect rapid bone marrow reconstitution with a low risk of GVHD,
- This would expand the use of bone marrow transplantation to all those in need of the procedure but who currently cannot find a donor.
Less than 30% of individuals who could use an unrelated donor bone marrow transplant actually receive one because for the rest a fully matched donor cannot be found. Perfect matching is required because of the high risk of the potentially life-threatening complication of severe graft-versus-host disease (GVHD).
MSB remains on track to file an Investigational New Drug (IND) submission to the FDA to commence a Phase 3 trial for its bone marrow transplant product by the end of this year. Mesoblast has sufficient cash reserves to fund the Phase 3 trial.
