There are approximately 60,000 patients with hematologic malignancies that undergo stem cell transplantation on a worldwide annual basis. Of those patients, approximately 20-25% of patients receive a sub-optimal cell dose, and the cell dose received by the remainder of the patients may be effectively enhanceable to potentially improve clinical outcomes. Moreover, each year there are approximately 5,000 patients with hematologic malignancies that are candidates for stem cell transplantation but don’t qualify due to their inability to mobilize a sufficient number of stem cells.

ProHema is a proprietary biologic that is currently undergoing clinical testing in patients requiring hematopoietic stem cell transplantation (HSCT) for the treatment of hematologic malignancies.

Fate recently completed a twenty-one patient, Phase 1b clinical study of ProHema in patients requiring dual umbilical cord blood unit transplantation. Based on the results of the Phase 1b study, Fate is advancing ProHema into additional clinical studies. Fate intends to explore the following settings: allogeneic transplantation using umbilical cord blood; allogeneic transplantation using other sources of hematopoietic stem cells (HSCs) (e.g., bone marrow; peripheral blood); autologous transplantation in patients that have failed to or that have poorly mobilized; and allogeneic transplantation in patients with non-malignant, but incurable, diseases (e.g., inborn errors of metabolism and certain autoimmune diseases).

ProHema is produced through the pharmacologic modulation of hematopoietic stem cells HSCs using a small molecule (FT1050), resulting in rapid and supra-physiologic changes in key stem cell properties. Such changes are believed to enhance hematopoietic reconstitution in patients by improving HSC homing to the bone marrow niche and by increasing HSC proliferation and survival; our studies suggest that, based on these changes, ProHema enables an estimated 10-fold increase in the effective cell dose administered to patients in need of reconstitution. The foundation of Fate’s ProHema program is based on the esteemed research of one of our scientific founders, Dr. Leonard Zon, whose seminal work formed the subject of a publication in the journal Nature (“Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis,” North et al., Nature 2007).