Fate Therapeutics, Inc., a biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for ProTmune for the reduction of incidence and severity of acute graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT). In addition, the company announced that its multi-center, randomized, controlled phase 1/2 clinical trial of ProTmune in adult subjects with hematologic malignancies is open for patient enrollment.

"The Fast Track designation for ProTmune underscores the significant need to address life-threatening immunological conditions which compromise the curative potential of allogeneic hematopoietic cell transplantation," said Chris Storgard, M.D., chief medical officer of Fate Therapeutics. "Acute graft-versus-host disease is a leading cause of morbidity and mortality in allogeneic HCT recipients. There is no approved preventive therapy, and current treatments suppress immune function and place high-risk immunocompromised patients at even greater risk for severe infections. We look forward to continuing to work with the FDA to rapidly advance our novel immunotherapy through the clinical development and regulatory processes, with the aim of bringing a transformative therapy to patients in an expedited time frame."

Fate Therapeutics is currently investigating ProTmune in an open-label phase 1/2 clinical trial for the prevention of acute GvHD and cytomegalovirus (CMV) infection, both of which are leading causes of morbidity and mortality in patients undergoing HCT. The multi-center clinical trial design consists of an initial 10-subject, phase 1 stage, during which all subjects undergoing allogeneic mobilized peripheral blood (mPB) HCT 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 allogeneic mPB HCT will be assigned to receive either ProTmune or a conventional mPB cell graft in a 1:1 ratio.

The Fast Track designation is designed to facilitate development and expedite review of experimental therapies that address the unmet medical needs of patients with serious conditions. Acute GvHD usually occurs within the first several months post-HCT when newly-transplanted donor immune cells recognize the patient's body as foreign and attack the patient's tissue. Despite the use of protocols to reduce the incidence of acute GvHD, up to 50 percent of HCT recipients still experience the debilitating disease. Additionally, only about half of patients with acute GVHD durably respond to its treatment. There are approximately 30,000 allogeneic HCT procedures performed globally each year according to the Center for International Blood and Marrow Transplant Research.

ProTmune is an investigational programmed cellular immunotherapy undergoing clinical development for the prevention of acute GvHD and CMV infection in patients undergoing allogeneic HCT. The cell therapy is produced by modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to enhance the biological properties and therapeutic function of the graft's immune cells. The programmed mobilized peripheral blood graft is adoptively transferred and administered to a patient as a one-time intravenous infusion.