Fate Therapeutics, Inc. (NASDAQ: FATE) announced today 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.
“We are pleased to clear this important milestone with the FDA, and expect to initiate the clinical investigation of ProTmune in mid-2016 for the prevention of life-threatening complications that often compromise the curative potential of HCT,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “Programming donor immune cells ex vivo to enhance therapeutic function upon adoptive transfer is a highly-differentiated therapeutic paradigm, which is easily integrated into current clinical practice and avoids costly and time-consuming measures, such as genetic engineering, cell expansion and cell separation. We believe the use of ProTmune as the donor cell source for HCT can meaningfully improve patient outcomes, decrease hospital length of stay by mitigating use of in-hospital drug treatments, and substantially reduce the overall cost of care.”
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.
According to the Center for International Blood and Marrow Transplant Research, there are approximately 30,000 allogeneic HCT procedures performed globally each year, of which approximately 65% utilize mPB as the donor cell source. 35-50% of HCT patients develop acute GvHD, and 70-80% of HCT patients experience at least one severe infection.
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