Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics
company, today announced the publication of data from the previously
reported, multi-center Phase 1/2 clinical study evaluating the safety
and efficacy of NiCord® as a stand-alone, hematopoietic stem
cell (bone marrow) transplant in the Journal
of Clinical Oncology1,2. NiCord is an investigational
product candidate in Phase 3 development as a universal bone marrow
transplant solution for patients with high-risk hematologic
malignancies, or blood cancers.
Results from the Phase 1/2 study showed that patients transplanted with
NiCord had rapid and durable engraftment of neutrophils and platelets,
as well as prompt immune reconstitution. The median time to neutrophil
recovery was shortened by nearly 50 percent for patients who received
NiCord compared to a retrospective cohort of patients who received
standard umbilical cord blood. NiCord also demonstrated an acceptable
safety profile for patients undergoing bone marrow transplant.
“In this study, patients who received NiCord had a clinically meaningful
reduction in their time to neutrophil and platelet recovery compared to
a retrospective cohort of patients who received a standard umbilical
cord blood transplant. The neutrophil recovery observed with NiCord also
resulted in fewer days spent in the hospital compared to the comparator
cohort,” said Mitchell Horwitz, M.D., principal investigator and
professor of medicine at the Duke Cancer Institute. “These data suggest
a potential step toward making stem cell transplantation safer and more
accessible to patients with lethal blood cancers.”
Despite the curative potential of bone marrow transplants, it is
estimated that more than 40 percent of eligible patients do not receive
one for various reasons, including finding a matched donor.3
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.
NiCord Phase 1/2 Study Design and Results
“Phase I/II study of stem cell transplantation using a single cord blood
unit expanded ex vivo with nicotinamide,” described results from the
completed multicenter, Phase 1/2 clinical trial of NiCord 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 NiCord patient cohort was compared to a retrospective
cohort of patients who received standard cord blood transplant using
data from the Center for International Blood and Marrow Transplant
Key findings included the following:
Patients transplanted with NiCord had rapid and durable engraftment of
neutrophils and platelets, as well as prompt immune reconstitution.
The age-adjusted cumulative incidence of neutrophil engraftment at 42
days following transplantation was 94 percent for NiCord recipients
compared to 85 percent for the CIBMTR cohort.
Among patients who engrafted, the median time to neutrophil recovery
was 11.5 days (95% CI: 9-14 days) for NiCord recipients compared to 21
days (95% CI: 20-23 days) for the CIBMTR cohort (p0.001).
For patients achieving platelet recovery, the median time to platelet
recovery was 34 days (95% CI:32-42 days) and 46 days (95% CI:42-50
days) for the NiCord and CIBMTR cohorts, respectively (p0.001).
NiCord demonstrated an acceptable safety profile, with hypertension reported
as the most common adverse event attributable to NiCord 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. NiCord recipients spent a median of 73 days alive and
out of hospital during the first 100 days following UCB
“These data demonstrate the potential of NiCord to give patients with
high-risk blood cancers an opportunity for a cure, particularly patients
who would otherwise not be able to receive a bone marrow transplant
using a matched donor source,” stated Ronit Simantov, M.D., chief
medical officer at Gamida Cell. “We are actively enrolling patients in
our Phase 3 study, which is designed to confirm the potential of NiCord
to be an effective transplantation solution. We look forward to
completing patient enrollment expected in the second half of 2019.”
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 U.S. Food and Drug Administration,
making it the first bone marrow transplant alternative to receive this
designation. It has also received U.S. and EU orphan drug designation. A
Phase 3 study evaluating NiCord in patients with leukemia and lymphoma
is ongoing in the United States, Europe and Asia.4 For more
information on NiCord clinical trials, please visit www.clinicaltrials.gov.
About Gamida Cell
Gamida Cell is a clinical stage
biopharmaceutical company leveraging its proprietary technology to
develop cell therapies that are designed to cure cancer and rare,
serious hematologic diseases. The company is leveraging its
nicotinamide-, or NAM-, based cell expansion technology to develop a
pipeline of products designed to address the limitations of cell
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 completion of enrollment of the ongoing Phase 3 clinical study
of NiCord and the results of clinical development of NiCord, which
statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the timing and success of
clinical trials and potential complications thereof and the scope,
progress and expansion of developing and commercializing NiCord. 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 September 28,
2018, and other filings that Gamida Cell makes with the SEC from time to
time (which are available at http://www.sec.gov),
the events and circumstances discussed in such forward-looking
statements may not occur, and Gamida Cell’s actual results could differ
materially and adversely from those anticipated or implied thereby. Any
forward-looking statements speak only as of the date of this press
release and are based on information available to Gamida Cell as of the
date of this release.
2 Horwitz M.E., Wease S., Blackwell B.,
Valcarcel D. et al. 2018. Phase I/II study of stem-cell transplantation
using a single cord blood unit expanded ex vivo with nicotinamide. Journal
of Clinical Oncology. DOI: 10.1200/JCO.18.00053
Department of Health and Human Services: Health Resources and Services
Administration. Bone Marrow and Cord Blood Donation and Transplantation. https://bloodcell.transplant.hrsa.gov/about/general_faqs/index.html.
Last accessed November 27, 2018.