ProTmune is a programmed cellular immunotherapy being developed as a next-generation allogeneic graft for the thousands of patients each year with hematologic malignancies and rare genetic disorders that seek curative outcomes through hematopoietic cell transplantation (HCT). All too often the curative potential of HCT is compromised though by the occurrence of graft-versus-host disease (GvHD) and severe infections. Approximately 50% of patients undergoing HCT die or experience relapse within the first two years following HCT, with the leading causes of non-relapse mortality being GVHD and severe infections. By optimizing the therapeutic properties of the graft itself prior to its administration to a patient, we believe the three leading causes of morbidity and mortality associated with allogeneic HCT – namely, graft-versus-host disease, severe infections and disease relapse – can be significantly reduced and overall patient survival can be improved.
We are conducting a multi-center Phase 1/2 clinical trial of ProTmune in adult subjects with hematologic malignancies undergoing matched unrelated donor mobilized peripheral blood HCT. The PROTECT study is intended to evaluate the safety and tolerability of ProTmune, and to assess the potential of ProTmune to prevent acute GvHD. There are currently no approved therapies for the prevention of GvHD in patients undergoing allogeneic HCT, giving rise to a significant unmet medical need.
The PROTECT Phase 1 stage is intended to assess the safety of ProTmune in up to ten subjects, and does so by evaluating neutrophil engraftment and survival at Day 30 following HCT. Following a satisfactory assessment of the PROTECT Phase 1 safety data by the study’s independent data monitoring committee, the PROTECT Phase 2 stage is expected to be initiated. The Phase 2 stage is randomized, controlled and blinded, where 30 subjects are to receive ProTmune and 30 subjects are to receive control, a standard-of-care conventional hematopoietic cell graft.
The U.S. Food and Drug Administration granted Fast Track and Orphan Drug Designations, and the European Medicines Agency granted Orphan Medicinal Product Designation, for ProTmune. The orphan designation granted in each jurisdiction broadly covers subjects undergoing allogeneic HCT across diseases for which the procedure is performed, including blood cancers and genetic disorders.