Fate Therapeutics has initiated enrollment in the Phase 2 stage of PROTECT, a combined, open-label Phase 1/2 clinical trial evaluating ProTmune for the prevention of acute graft-versus-host disease (GvHD) in patients with hematologic malignancies undergoing bone marrow transplants from matched, unrelated donors.
The announcement followed an independent data monitoring committee safety review recommending opening Phase 2 enrollment by unanimity. Enrollment is now open at 13 centers across the U.S.
Bone marrow transplant is the therapeutic procedure often chosen for the treatment of leukemias, lymphomas, and myelodysplastic syndromes. GvHD is a severe immunological condition that commonly arises in patients during the first weeks following bone marrow transplants from matched donors, when the donor’s immune cells attack the patient’s tissues and organs resulting in a potentially fatal immune system reaction.
ProTmune is an investigational, next-generation hematopoietic cell graft designed to prevent acute GvHD in patients undergoing bone marrow transplants. ProTmune requires a donor-sourced small blood graft to modulate with two small molecules (FT1050 and FT4145) and enhance the biological properties and therapeutic function of the transplant.
The data monitoring committee safety review concluded that all of the first seven patients enrolled in the Phase 1 part of the PROTECT study met safety objectives of neutrophil engraftment (homing the bone marrow) and survival, and reached day 28 without any events of transplant failure or serious adverse events related to ProTmune.
Of the seven patients, three were diagnosed with acute lymphoblastic leukemia (ALL), three with acute myeloid leukemia (AML), and one with myelodysplastic syndrome (MDS).
The Phase 2 stage will assess the safety and effectiveness of ProTmune in 60 participants, who will be randomized to receive either ProTmune or another unrelated donor transplant.
The primary efficacy endpoint of the Phase 2 part of PROTECT is to measure incidence of acute GvHD by day 100 following transplant.
“Acute GvHD is the leading cause of early morbidity and mortality in patients undergoing allogeneic [from matched, unrelated donors] transplant. We are excited to initiate the randomized, controlled and blinded Phase 2 stage of PROTECT and assess the potential of ProTmune to deliver transformative benefits to cancer patients,” Chris Storgard, MD, chief medical officer at Fate Therapeutics, said in a press release. “We thank the study’s independent data monitoring committee for its Phase 1 data review. Once all seven Phase 1 subjects progress to Day 100 [post-transplant], we expect to present ProTmune Day 100 efficacy data, including acute GvHD, cancer relapse and survival, at the 2017 American Society of Hematology annual meeting.”
The U.S. Food and Drug Administration (FDA) granted ProTmune fast track designation in June 2016 and orphan drug status in September 2016. Similarly, the European Medicines Agency (EMA) granted ProTmune orphan medicinal product designation in November 2016.