Pluristem PLX cell therapy granted FDA Orphan Drug Designation
Pluristem Therapeutics announced that the FDA has granted Orphan Drug Designation to the Company's PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery following hematopoietic cell transplantation, or HCT. Pluristem's product PLX-R18 is currently being evaluated for the treatment of insufficient hematopoietic recovery following bone marrow transplantation in an ongoing Phase 1 clinical trial in the U.S. and Israel. The trial is designed to evaluate the safety of intramuscular injections of PLX-R18 cells in 24 subjects with incomplete hematopoietic recovery persisting for at least 4 months after HCT, with a 12-month follow-up period. The primary endpoint is safety. Exploratory endpoints include changes in platelet and hemoglobin levels, changes in transfusion frequency, a shift from transfusion dependence to transfusion independence, changes in quality of life, and changes in the serum immunological parameters.