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). The curative potential of HCT is compromised, however, 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 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 have reported Day 100 clinical data from the PROTECT Phase 1 Study of ProTmune in patients with hematologic malignancies undergoing allogeneic HCT. The initial data suggest that ProTmune can provide the critical immunological balance necessary to reduce the incidence and severity of GvHD and maintain graft-versus-leukemia activity, and supported the initiation of the Phase 2 stage of the PROTECT study.
The ongoing Phase 2 stage of PROTECT is a randomized, controlled and double-blinded clinical trial of ProTmune in up to 60 adult subjects with hematologic malignancies undergoing matched unrelated donor HCT. The primary efficacy endpoint of PROTECT is cumulative incidence of Grades 2-4 acute GvHD by Day 100 following HCT, where prospective clinical studies have shown that 40% to 80% of patients undergoing matched unrelated donor HCT experience Grades 2-4 acute GvHD. Additional key endpoints, including rates of chronic GvHD, cancer relapse, and disease-free and overall survival, are also being assessed.
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 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.