Gamida Cell Reports Immune Reconstitution Data from Completed Phase 1/2 Clinical Study of NiCord® Presented at ASH 2018 Annual Meeting
– NiCord Recipients Demonstrated Rapid and Robust Immune Reconstitution following Transplantation –
“Immune reconstitution following transplantation is critical for disease
and viral control, but historically cord blood transplantation has had
limitations in timely immune reconstitution in patients,” said Jaap-Jan
Boelens, M.D., Ph.D., Chief, Pediatric Stem Cell Transplantation and
Cellular Therapies Service,
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 While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.
Data Presented at ASH 2018
The poster presentation, “Rapid
and robust CD4+ and CD8+ T-, NK-, B- and monocyte cell reconstitution
after nicotinamide-expanded cord blood transplantation” (Abstract 2123),
described early, in-depth immune reconstitution data from the completed
Phase 1/2, multicenter clinical study of NiCord as a stand-alone graft
after myeloablative therapy in patients with high-risk hematologic
malignancies.3 A random subgroup of 27 patients from this
study had extensive immune monitoring evaluated throughout the first
year after transplant. The primary endpoint was the probability of
achieving CD4+ immune reconstitution (>50×106/L) within the first 100
days, and the secondary endpoints included the recovery of B cells, CD4+
T cells and natural killer (NK) cells during the first year after
transplantation. These data were compared to cohorts of adolescent and
young adults with hematologic malignancies receiving unmanipulated cord
blood transplantation (n=27) or unrelated bone marrow transplantation
(n=20). Analyses were performed at the
Key findings from the analysis include the following:
- 91 percent of patients achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation with NiCord.
- Immune reconstitution of T cells was similar in the NiCord group (median age: 41.5 years) compared to the younger cohorts receiving unmanipulated cord blood and unrelated bone marrow (median ages 15.4 and 14.3 years, respectively).
- Immune reconstitution of B cells (p = 0.02) and NK cells (p < 0.001) was significantly faster after transplantation with NiCord compared to the other groups.
- Immune reconstitution after NiCord transplantation was associated with recovery of a broad spectrum of T cell, B cell and NK cell subsets representing a range of effector functions similar to that observed with other graft sources.
“These data, combined with the clinical data from our Phase 1/2 study of
NiCord in patients with high-risk blood cancers, suggest that NiCord has
the potential to be an important treatment option for patients
undergoing bone marrow transplant,” stated
About NiCord
NiCord, the company’s lead clinical program, is
under development as a universal bone marrow transplant solution for
patients with high-risk hematologic malignancies. NiCord has been
granted breakthrough status by the
About
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
timing of patient enrollment in Gamida Cell’s ongoing Phase 3 study of
NiCord, 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 study. 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
References
1de Koning C., Horwitz M.E., Sanz G.,
Jagasia M. et al. Rapid and robust CD4+ and CD8+ T-, NK-, B- and
monocyte cell reconstitution after nicotinamide-expanded cord blood
transplantation. Presented at the
2
3ClinicalTrials.gov
identifier NCT01816230.
4ClinicalTrials.gov identifier
NCT02730299.
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Source:
Jaren Irene Madden
jaren@gamida-cell.com
1-617-286-6264