Fate Therapeutics, Inc, a biopharmaceutical company dedicated to the development of programmed cellular immunotherapeutics for cancer and immune disorders, announced the U.S. Food and Drug Administration (FDA) has cleared the Company's investigational new drug (IND) application for ProTmune™, a programmed cellular immunotherapy consisting of donor-sourced mobilized peripheral blood cells which have been functionally modulated using two small molecules. The IND is now active and Fate Therapeutics plans to initiate a multi-center, randomized, controlled Phase 1/2 clinical trial in adult patients with hematologic malignancies undergoing mobilized peripheral blood (mPB) hematopoietic cell transplantation (HCT) in mid-2016.
The primary objectives of the Phase 1/2 clinical trial are to evaluate safety and tolerability, and to assess the potential of ProTmune to prevent acute graft-versus-host disease (GvHD) and cytomegalovirus (CMV) infection, both of which are leading causes of morbidity and mortality in patients undergoing HCT. There are currently no approved therapies for the prevention of GvHD or CMV infection in patients undergoing allogeneic HCT, giving rise to a significant unmet medical need.
The clinical trial design consists of an initial 10-subject, Phase 1 stage, during which all subjects undergoing mPB HCT following myeloablative conditioning will receive ProTmune. Following an independent data monitoring committee safety review, a 60-subject, randomized, controlled Phase 2 stage is expected to enroll, during which subjects undergoing mPB HCT following myeloablative conditioning will be assigned in a 1:1 ratio to receive either ProTmune or unmanipulated mPB cells. Two Endpoint Adjudication Committees are expected to evaluate efficacy of ProTmune in the study, one through assessing acute GvHD and the other through assessing CMV tissue-invasive disease, viremia and additional clinical outcomes.
