Gamida Cell presented results of a Phase 3 clinical study of omidubicel in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR). Omidubicel is an advanced cell therapy under development as an allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.
The international, multi-center, randomized Phase 3 study of omidubicel was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. The intent-to-treat analysis included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma and was conducted at more than 50 clinical centers in the United States, Latin America, Europe and Asia.
The study achieved its primary endpoint, time to neutrophil engraftment. In the intent-to-treat analysis, the median time to neutrophil engraftment was significantly shorter for patients who received omidubicel (12 days) compared to the comparator group (22 days). Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, 96 percent of patients who received omidubicel achieved successful neutrophil engraftment, compared to 88 percent of patients in the comparator group. In addition, all three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel versus the comparator group.
These results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.
Gamida Cell’s proprietary nicotinamide-based, or NAM-based, expansion technology enables donor cells to grow while maintaining functionality. Omidubicel consists of NAM-expanded hematopoietic stem cells and differentiated immune cells, including T cells. The final cell therapy product may be cryopreserved until the patient is ready to begin the transplant, when it is thawed and infused.
Omidubicel has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration and Orphan Drug Designation in the U.S. and EU.