Gamida Cell Announces Data for Omidubicel Presented at the International Society for Cellular and Gene Therapy 2019 Annual Meeting
“In the Phase 1/2 clinical study, patients who received omidubicel had a
clinically meaningful reduction in their time to neutrophil and platelet
recovery compared to a real-world cohort of patients who received a
standard umbilical cord blood transplant. The neutrophil recovery
observed with omidubicel also resulted in fewer days spent in the
hospital compared to the comparator cohort,” said
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.3 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.4 Omidubicel 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.
“At Gamida Cell, our aspiration is to bring the first
Data Presented at ISCT 2019 Annual Meeting
Phase 1/2 Clinical Data
The presentation, “NiCord, an Expanded Cord Blood Product, Accelerates
Engraftment After Myeloablative Conditioning,” described results from
the completed multicenter, Phase 1/2 clinical trial of omidubicel in 36
patients with high-risk hematologic malignancies and no readily
available matched sibling or matched unrelated adult donor. The key
primary endpoint was the cumulative incidence of neutrophil engraftment
at 42 days. Additionally, the omidubicel patient cohort was compared to
a retrospective cohort of patients who received standard cord blood
transplant using data from the
Data from the study demonstrated that patients transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets. The age-adjusted cumulative incidence of neutrophil engraftment at 42 days following transplantation was 94 percent for omidubicel recipients compared to 85 percent for the CIBMTR cohort. Among patients who engrafted, the median time to neutrophil recovery was 11.5 days (95 percent confidence interval (CI): 9-14 days) for omidubicel recipients compared to 21 days (95 percent CI: 20-23 days) for the CIBMTR cohort (p < 0.001). For patients achieving platelet recovery, the median time to platelet recovery was 34 days (95 percent CI: 32-42 days) and 46 days (95 percent CI: 42-50 days) for the omidubicel and CIBMTR cohorts, respectively (p < 0.001). Omidubicel demonstrated an acceptable safety profile, with hypertensionreported as the most common adverse event attributable to omidubicel infusion, and moderate to severe chronic graft vs. host disease reported in 9.8 percent of patients at one year following transplantation. Primary hospital discharge occurred at a median of 20 days following transplantation. Omidubicel recipients spent a median of 73 days alive and out of hospital during the first 100 days following transplantation.
Phase 1/2 Translational Data
The presentation, “Rapid and Robust CD4+ and CD8+ T-, NK-, B- and
Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood
Transplantation,” described in-depth immune reconstitution data from the
completed Phase 1/2 clinical study of omidubicel. Immune reconstitution
for 27 patients receiving omidubicel 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 omidubicel achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation. Reconstitution of T cells in the omidubicel 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 = 0.026) and NK cells (p < 0.001), was significantly faster after transplantation with omidubicel compared to the cohorts, and suggests that omidubicel reconstitutes diverse functions of the immune system.
Omidubicel (formerly known as 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).1 Omidubicel is the first bone marrow transplant
product to receive Breakthrough Therapy Designation from the
Omidubicel is an investigational therapy, and its safety and efficacy
has not been evaluated by the
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 the progress of and data reported from the
clinical trials of Gamida Cell’s product candidates, 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 clinical trials and variability, and ramifications for the
cost thereof; and clinical, scientific, regulatory and technical
developments. In light of these risks and uncertainties, and other risks
and uncertainties that are described in the Risk Factors section of
Gamida Cell’s public filing on Form 20-F, filed with the
1 Gamida Cell’s lead development candidate consists of
omidubicel (expanded hematopoietic stem cells) and differentiated immune
cells, including T cells.
2ClinicalTrials.gov identifier NCT02730299.
4 Carreras et al. The EBMT Handbook. Springer 2019.
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.