NiCord improves efficacy of bone marrow transplant for hematologic malignancies
A phase 3 trial of omidubicel has shown the graft modality superior to standard umbilical cord blood for bone marrow transplantation in patients with hematologic malignancies, according to topline data released by the agent’s manufacturer.
The investigational cell therapy met all three secondary study endpoints, including measures related to platelet engraftment, infections and hospitalizations. All are considered key clinical measures in bone marrow transplantation.
Omidubicel (NiCord, Gamida Cell) is an ex vivo-expanded, cryopreserved graft derived from an entire cord blood unit. The therapy uses Gamida Cell’s proprietary nicotinamide-based expansion technology that enables donor cells to grow while maintaining functionality. The final cryopreserved product is given via IV and includes nicotinamide-expanded hematopoietic stem cells and differentiated immune cells, including T cells.
The results come from an international, multicenter, randomized phase 3 study (NCT02730299) to evaluate the efficacy and safety of omidubicel among patients with leukemia, lymphoma and myelodysplastic syndrome. The study evaluated patients undergoing bone marrow transplant who received omidubicel compared with a group of patients who received a standard umbilical cord blood transplant.
The group who received omidubicel showed statistically significant improvement in all three secondary endpoints compared with the standard umbilical cord blood group. The endpoints included the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infections in the first 100 days after transplant, and the number of days alive and out of the hospital in the first 100 days after transplant.
Gamida Cell previously reported that omidubicel achieved the trial’s primary endpoint by showing a statistically significant reduction in time to neutrophil engraftment compared with standard umbilical cord blood transplantation.
“These data, obtained in a global, randomized, multi-institutional setting, could represent an important step forward in the field. In addition to more rapid platelet engraftment, a key step toward recovery, reducing infections and hospitalizations are considered meaningful patient outcomes and have the potential to provide substantial value for patients, their families and the health care system,” Mitchell Horwitz, MD, principal investigator and professor of medicine at Duke Cancer Institute, said in a press release. “The totality of these data strengthen my belief that omidubicel has the potential to be a graft source for any patient who does not have access to a matched related donor and could help make stem cell transplantation more accessible and more successful for patients with lethal blood cancers.”
Omidubicel was previously granted breakthrough therapy designation by the FDA for bone marrow transplantation in patients with hematologic malignancies. The therapy was the first bone marrow transplant product to receive this designation and, if approved by the FDA, would become the first commercially available engineered bone marrow transplant graft.
Gamida Cell anticipates reporting the full data set at a major medical meeting during the fourth quarter of 2020, according to the press release. The company also anticipates submitting a biologics license application to the FDA for omidubicel during the same time frame.