New data add to the body of evidence supporting
efficacy of omidubicel, Gamida Cell’s lead product candidate, which
has a target PDUFA action date with U.S. Food and Drug
Administration (FDA) of May 1, 2023
New data for GDA-201, Gamida Cell’s natural
killer (NK) cell therapy candidate in ongoing Phase 1/2 study for
non-Hodgkin lymphoma, show robust immune system engagement and high
cytotoxicity levels
Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working
to turn cells into powerful therapeutics, today announced that two
oral presentations and a poster presentation highlighting Gamida
Cell’s investigational product candidates, omidubicel and GDA-201,
were shared at the 2023 Tandem Meetings, Transplantation &
Cellular Therapy (TCT) Meetings of the American Society for
Transplantation and Cellular Therapy (ASTCT), and the Center for
International Blood and Marrow Transplant Research (CIBMTR). The
meetings took place February 15-19 virtually and in person in
Orlando, Florida.
“The data presented at Tandem demonstrate Gamida Cell’s
expertise in developing potent, potentially curative cell therapies
for patients with hematologic malignancies,” said Ronit Simantov,
M.D., chief medical and scientific officer of Gamida Cell. “The
translational data from our Phase 3 study of omidubicel, our lead
product candidate, show faster, multi-faceted immune response – as
quickly as seven days—in omidubicel-treated patients compared to
standard cord blood. These data add to the substantial body of
knowledge that supports omidubicel’s potential, if approved, as a
new advanced cell therapy donor source for patients in need of an
allogeneic stem cell transplant. The data on GDA-201, our NK cell
therapy candidate in Phase 1/2 development for non-Hodgkin
lymphoma, provide further characterization of its phenotype and
functionality, and demonstrate its activity in triggering an
adaptive immune response. These data advance our understanding of
GDA-201’s potential as a well-differentiated NK cell therapy.”
Reporting on translational data from a Phase 1 study of a fresh
formulation of GDA-201, external investigator Veronika Bachanova,
M.D., Ph.D., professor at the University of Minnesota Medical
School, said, “The GDA-201 treatment results in engagement with the
adaptive immune system, which is what we would hope for from an NK
cell therapy. The high T cell infiltration to the tumor site three
to 16 days post GDA-201 administration points to the recruitment of
the adaptive immune system, and signals the potential for durable
effectiveness. These data elaborate on previous results that show
the safety and preliminary evidence of activity of GDA-201 in
patients with relapsed refractory non-Hodgkin lymphoma.”
Additional details about the presentations are as follows:
Title: GDA-201: A Cryopreserved, Readily Available
Formulation of Nicotinamide-Enabled Natural Killer Cells, Shows
Increased Potency and Enhanced Cytotoxicity Abstract Number:
204 Lead Author: Yael Yoffe-Mizrahi, Ph.D.; Associate
Director and Head of Natural Killer Pipeline at Gamida Cell
- Highlights: GDA-201 cells were tested for viability,
phenotyping, function and potency. Previous characterization of
GDA-201 showed high levels of CD56, CD16, CD49a and CD62L
expression, and low levels of CD57, as well as low levels of immune
checkpoints such as LAG3 and CD200R. The new analyses showed that
cryopreserved GDA-201 exhibited high viability (>90%), and high
purity up to 12 months post-manufacturing, and preserved the
ability to proliferate post-thaw. GDA-201 maintained high levels of
expression of CD16, which mediates antibody-dependent cellular
toxicity, and CD62L, which is a homing and retention marker.
GDA-201 also demonstrated high potency, based on intracellular
secretion of TNF-alpha & IFN-gamma and extracellular
degranulation marker CD107a.
Title: Tumor Microenvironment Spatial Analysis after
Adoptive NK Cell Therapy for Lymphoma Revealed Cross-Talk with
Adaptive T Cell Immunity Abstract Number: 81 Lead
Author: Veronika Bachanova, M.D., Ph.D.; Professor at the
University of Minnesota Medical School
- Highlights: This presentation highlights the novel
observations of “on treatment” tumor biopsies from eight patients
treated with GDA-201 in a Phase 1 study. Spatial analysis
demonstrated that NK cells infiltrated the lymph nodes at low
frequencies (<1% of all cells in the tumor microenvironment).
GDA-201 cells were undetectable after 14 days. Remarkably, T cells
were observed in 50-95% of tumor site cellularity. Most biopsies
obtained as early as three to seven days post-infusion showed
strong indications of widespread tumor death. These observations
suggest that GDA-201 infusions trigger profound immune
microenvironment changes, supporting the influx of host T cells
early post GDA-201 infusion. This further suggests the engagement
of the adaptive immune system, and effective tumor
elimination.
Title: Longitudinal Immune Reconstitution Profiling
Suggests Anti-Viral Protection after Transplantation with
Omidubicel: A Phase 3 Substudy Abstract Number: 84 Lead
Author: Roei Mazor, M.D., Ph.D.; Head of the Clinic of
Histiocytic Neoplasms at Assuta Medical Center and Medical Director
at Gamida Cell
- Highlights: New data on peripheral blood lymphocyte
counts measured in correlation with time to neutrophil and platelet
engraftment in omidubicel transplanted and standard cord blood
transplanted patients. Seven days post-transplant, omidubicel
transplanted patients showed a statistically significant
correlation between CD3+/CD4+ T cell counts and time to neutrophil
engraftment. Similar correlations were noted between
CD3+/CD8+/CD19+ cell counts and time to platelet engraftment.
Patients transplanted with standard cord blood showed no such
correlations at Day 7 post-transplant, and only began to show
correlations starting at 14 days post-transplant. Data support past
findings that omidubicel stimulates a faster immune response than
standard cord blood and may also explain the lower incidence of
serious bacterial, fungal and viral infections for omidubicel
transplanted patients.
Presentations are available at gamida-cell.com/our-rd.
About Omidubicel
Omidubicel is an advanced cell therapy candidate for allogeneic
hematopoietic stem cell (bone marrow) transplant that, if approved,
has the potential to expand access and improve outcomes for
patients with blood cancers. Omidubicel demonstrated a
statistically significant reduction in time to neutrophil
engraftment in comparison to standard umbilical cord blood in an
international, multi-center, randomized Phase 3 study (NCT02730299)
in patients with hematologic malignancies undergoing allogeneic
bone marrow transplant. The Phase 3 study also showed reduced time
to platelet engraftment, reduced infections and fewer days of
hospitalization. One year post-transplant data showed sustained
clinical benefits with omidubicel as demonstrated by significant
reduction in infectious complications as well as reduced
non-relapse mortality and no significant increase in relapse rates
nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel
is the first stem cell transplant donor source to receive
Breakthrough Therapy Designation from the FDA and has also received
Orphan Drug Designation in the US and EU. Omidubicel has a PDUFA
target action date of May 1, 2023.
Omidubicel is an investigational stem cell therapy candidate,
and its safety and efficacy have not been established by the FDA or
any other health authority. For more information about omidubicel,
please visit https://www.gamida-cell.com.
About GDA-201
GDA-201 is an intrinsic NK cell therapy candidate being
investigated for the treatment of hematologic malignancies.
Preclinical studies have shown that GDA-201 may address key
limitations of NK cells by increasing the cytotoxicity and in vivo
retention and proliferation in the bone marrow and lymphoid organs.
Furthermore, these data suggest GDA-201 may improve
antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting
of NK cells. GDA-201 is the lead investigational candidate among a
pipeline of enhanced and engineered intrinsic NK cell therapy
candidates Gamida Cell is investigating as next-generation cell
therapies. A multicenter Phase 1/2 study of GDA-201 for the
treatment of non-Hodgkin lymphoma is ongoing.
GDA-201 is an investigational cell therapy candidate, and its
safety and efficacy have not been established by the FDA or any
other health authority.
About NAM Technology
Gamida Cell’s NAM based technology is designed to expand the
number and increase functionality of targeted cells, enhancing the
intrinsic properties of targeted cells as they are developed into
cell therapy candidates. In the case of omidubicel, our NAM based
technology enhances the inherent ‘stemness’ of stem cells,
expanding the number of cells and enhancing their ability to home
to the bone marrow. In the case of NK cells, our proprietary
technology reduces oxidative stress and preserves a highly
cytotoxic phenotype.
About Gamida Cell
Gamida Cell is a cell therapy pioneer working to turn cells into
powerful therapeutics. The company has a diverse pipeline of
potentially curative cell therapy candidates for patients with
blood cancers and solid tumors. We apply a proprietary expansion
platform leveraging the properties of NAM to allogeneic cell
sources including umbilical cord blood-derived cells and NK cells
to create therapy candidates with the potential to redefine
standards of care. These include omidubicel, an advanced cell
therapy candidate for allogeneic hematopoietic stem cell transplant
that, if approved, has the potential to expand access and improve
outcomes for patients with blood cancers, and a line of enhanced
and engineered NK cells targeted at solid tumors and hematologic
malignancies. For additional information, please visit
www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter,
Facebook or Instagram at @GamidaCellTx.
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 timing of the FDA’s review of the
BLA for omidubicel, and the potentially life-saving or curative
therapeutic and commercial potential of Gamida Cell’s product
candidates (including omidubicel and GDA-201). Any statement
describing Gamida Cell’s goals, expectations, financial or other
projections, intentions or beliefs is a forward-looking statement
and should be considered an at-risk statement. Such statements are
subject to a number of risks, uncertainties and assumptions,
including those related to the impact that the COVID-19 pandemic
could have on our business; clinical, scientific, regulatory and
technical developments; and those inherent in the process of
developing and commercializing product candidates that are safe and
effective for use as human therapeutics. In light of these risks
and uncertainties, and other risks and uncertainties that are
described in the Risk Factors section and other sections of Gamida
Cell’s Quarterly Report on Form 10-Q, filed with the Securities and
Exchange Commission (SEC) on November 14, 2022, 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. Although Gamida Cell’s
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Gamida Cell. As a result, you are cautioned not
to rely on these forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20230217005362/en/
Investor Contact Courtney Turiano Stern Investor
Relations, Inc. Courtney.Turiano@sternir.com 1-212-362-1200
Media Contact Dan Boyle Orangefiery dan@orangefiery.com
1-818-209-1692
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