Gamida Cell Announces Immune Reconstitution Data from Completed Phase 1/2 Clinical Study of NiCord® Presented at 2019 TCT Annual Meeting
– Data support ongoing Phase 3 study of NiCord in patients with hematologic malignancies –
“Reconstitution of a patient’s bone marrow and immune system is a
crucial factor in recovery following allogeneic hematopoietic stem cell
Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor.2 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect quality of life.3 NiCord is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of cells while preserving the cells’ functional therapeutic characteristics.
Data Presented at TCT Annual Meeting
The oral presentation, “Rapid and Robust CD4+ and CD8+ T-, NK-, B-Cell,
Dendritic Cell, and Monocyte Reconstitution after Nicotinamide-Expanded
Cord Blood Transplantation” (Abstract 69), described in-depth immune
reconstitution data from the completed Phase 1/2, multi-center clinical
study of NiCord as a stand-alone graft after myeloablative therapy in
patients with high-risk hematologic malignancies.4 Immune
reconstitution for 27 patients receiving NiCord was compared to
retrospective cohorts of adolescent and young adults with hematologic
malignancies receiving unmanipulated cord blood transplantation (unCBT,
n=27) or unrelated bone marrow transplantation (BMT, n=20). The primary
endpoint was the probability of achieving CD4+ immune reconstitution
(>50×106/L) within the first 100 days. Secondary endpoints
included the recovery of B cells, CD4+ T cells and natural killer (NK)
cells during the first year after transplantation. Analyses were
performed at the University Medical Centre Utrecht,
The analysis showed that 91 percent of patients receiving NiCord achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation. Reconstitution of T cells in the NiCord group (median age 41.5 years) was similar to the unCBT and BMT cohorts (median age 15.4 and 14.3 years, respectively), despite the younger age of the cohorts, who would be expected to reconstitute faster. In addition, reconstitution of a number of cell types, including B cells (p = 0.02) and NK cells (p < 0.001), was significantly faster after transplantation with NiCord compared to the cohorts, and suggests that NiCord reconstitutes diverse functions of the immune system. These findings may be explained by the higher stem cell dose and proliferative capacity of NiCord.
“Our goal is to bring a potentially transformative new treatment option
to patients in need of bone marrow transplant, and these data further
reinforce our belief in the clinical potential of NiCord,” stated
During the TCT Annual Meeting, new data were also presented from Gamida Cell’s NAM-NK clinical program, as well as initial data from a Phase 1/2 study of NiCord in patients with severe aplastic anemia. More information on those presentations can be found here.
NiCord, the company’s lead clinical program, is an advanced cell therapy
under development as a potential life-saving allogeneic hematopoietic
stem cell (bone marrow) transplant solution for patients with
hematologic malignancies (blood cancers). NiCord is the first bone
marrow transplant product to receive Breakthrough Therapy Designation
NAM-NK and NiCord are investigational therapies, and their safety and
efficacy have not been evaluated by the
About Gamida Cell
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is
defined in the Private Securities Litigation Reform Act of 1995,
including with respect to translational data from the completed Phase
1/2 study of NiCord for the treatment of hematologic malignancies and
the ongoing Phase 3 study of NiCord in patients with leukemia and
lymphoma, which statements are subject to a number of risks,
uncertainties and assumptions, including, but not limited to the scope,
progress and expansion of Gamida Cell’s studies and clinical, scientific
and technical developments. In light of these risks and uncertainties,
and other risks and uncertainties that are described in the Risk Factors
section of our Registration Statement on Form F-1 filed with
the SEC on
1ClinicalTrials.gov identifier NCT02730299.
3 Carreras et al. The EBMT Handbook. Springer 2019.
4 ClinicalTrials.gov identifier NCT01816230. Last accessed
5 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.
6 ClinicalTrials.gov identifier NCT03173937.