Hematopoietic stem cell transplantation (HSCT), commonly known as bone marrow transplantation, is the most frequently used cell therapy to treat a variety of hematologic malignancies and other serious conditions.1 In this procedure, patients are treated with chemotherapy, radiation or a combination of both to destroy residual cancerous or defective cells in the bone marrow. As this process also destroys the hematopoietic stem cells that are responsible for forming red blood cells, platelets and white blood cells, patients must then receive new bone marrow stem cells from a healthy donor, which is intended to fully reconstitute the bone marrow and immune system, with the intent to cure patients of their disease. Despite the curative potential of a bone marrow transplant, it is estimated that up to 40 percent of eligible patients do not receive one for various reasons, including the ability to find a matched donor.2,3
NiCord® is a NAM-expanded cell therapy with the potential to serve as a universal, curative bone marrow transplant for patients who do not have an available matched donor. NiCord maximizes the benefits of umbilical cord blood, a readily available source of stem cells. Cord blood, unlike bone marrow, does not require a fully matched donor for successful transplantation, and is suitable for use in approximately 95% of patients. In addition, the donor T cells in cord blood are naïve, meaning that they have not matured and may more readily adapt to the recipient. This results in greater immunologic compatibility, or the matching of the donor cells with the recipient’s cells, reducing the frequency and severity of graft-vs-host disease (GvHD).
In a Phase 1/2 study, patients undergoing transplantation with NiCord had rapid and long-lasting engraftment of neutrophils and platelets, two types of blood cells, compared to a historical evaluation of patients who received cord blood alone. Engraftment refers to when the cells received during transplant begin to grow and make healthy blood cells, which is an important milestone in transplant recovery. Additionally, NiCord treatment was associated with a significant reduction in the rate and severity of infections as well as significantly shorter hospital stays.
We are evaluating NiCord in an international, multicenter, randomized, pivotal Phase 3 clinical study. The study is currently enrolling 120 patients with high-risk leukemias, myelodysplastic syndrome or lymphomas. We anticipate completing enrollment in 2019 and reporting topline data in the first half of 2020. Learn more about the Phase 3 study (NCT02730299) at www.clinicaltrials.gov.
NiCord has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) as a novel graft modality for enhancement of cell engraftment and immune reconstitution in patients receiving a bone marrow transplant. In addition, NiCord has received Orphan Drug Designation from both the FDA and the European Medicines Agency.
NiCord is also being investigated in a Phase 1/2 open-label study in patients with severe aplastic anemia, a rare, life-threatening bone marrow failure disease.* This study is designed to evaluate the safety and effectiveness of transplantation with NiCord to overcome the high incidence of graft rejection associated with conventional cord blood for severe aplastic anemia. The trial is currently enrolling and preliminary data from the study is anticipated in 2019. Learn more about the Phase 1/2 study (NCT03173937) at www.clinicaltrials.gov.
1 D’Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): Center for International Blood and Marrow Transplant Research (CIBMTR) 2017 Summary Slides (the “CIBMTR Slides”) http://www.cibmtr.org.
2 Besse K, et al. Estimating Demand and Unmet Need for Allogeneic Hematopoietic Cell Transplantation in the United States Using Geographic Information Systems. J Oncol Pract. 2015 Mar;11(2):e120-30.
3 Gamida Cell market research data on file.
*The aplastic anemia investigational new drug (IND) application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as NiCord.