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Gamida Cell Announces Data for Omidubicel Presented at the International Society for Cellular and Gene Therapy 2019 Annual Meeting

May 31, 2019 Off By BusinessWire

BOSTON–(BUSINESS WIRE)–lt;a href="https://twitter.com/search?q=%24GMDA&src=ctag" target="_blank"gt;$GMDAlt;/agt; lt;a href="https://twitter.com/hashtag/ISCT2019?src=hash" target="_blank"gt;#ISCT2019lt;/agt;–Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune
therapeutics company, today announced that two presentations on
omidubicel¹ (formerly known as NiCord^®), an
investigational advanced cell therapy in Phase 3 clinical development
designed to enhance the life-saving benefits of hematopoietic stem cell
(bone marrow) transplant, took place at the International Society for
Cell and Gene Therapy (ISCT) 2019 Annual Meeting being held in
Melbourne, Australia. The presentations included a summary of clinical
and translational data from the completed Phase 1/2 clinical study of
omidubicel in patients with high-risk hematologic malignancies, or blood
cancers. Results from the Phase 1/2 study showed that patients
transplanted with omidubicel had rapid and durable engraftment of
neutrophils and platelets, as well as prompt immune reconstitution. An
international, randomized Phase 3 study of omidubicel in patients with
hematologic malignancies is currently ongoing.²

“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 Joanne Kurtzberg,
M.D., Director of the Marcus Center for Cellular Cures and the Carolinas
Cord Blood Bank at Duke University Medical Center. “These data suggest
an important potential step toward making stem cell transplantation
safer and more accessible to patients with lethal blood cancers, and I
am pleased to be participating in the Phase 3 study currently enrolling
patients.”

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 FDA-approved cell
therapy for bone marrow transplantation to patients,” stated Ronit
Simantov, M.D., chief medical officer at Gamida Cell. “These data
demonstrate the potential of omidubicel to give patients with high-risk
blood cancers, particularly those who would not otherwise receive a bone
marrow transplant from a matched donor, an opportunity for a cure.”

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 Center for International Blood and Marrow
Transplant Research (CIBMTR).

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 hypertension reported
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, Laboratory of Translational Immunology.

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.

About Omidubicel

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 U.S. Food
and Drug Administration and has also received Orphan Drug Designation in
the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated
rapid and durable time to engraftment and was generally well-tolerated.⁵
A Phase 3 study evaluating omidubicel in patients with leukemia and
lymphoma is ongoing in the U.S., Europe and Asia.² Omidubicel
is also being evaluated in a Phase 1/2 clinical study in patients with
severe aplastic anemia.⁶ The aplastic anemia investigational
new drug application is currently filed with the FDA under the brand
name CordIn^®, which is the same investigational development
candidate as omidubicel. For more information on clinical trials of
omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy
has not been evaluated by the U.S. Food and Drug Administration or any
other health authority.

About Gamida Cell

Gamida Cell is a clinical-stage biopharmaceutical company committed to
developing advanced cell therapies with the potential to cure blood
cancers and rare, serious hematologic diseases. We are leveraging our
proprietary nicotinamide-based, or NAM-based, cell expansion technology
to develop product candidates designed to address the limitations of
cell therapies. For additional information, please visit www.gamida-cell.com.

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 SEC on February
25, 2019, 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.

¹ Gamida Cell’s lead development candidate consists of
omidubicel (expanded hematopoietic stem cells) and differentiated immune
cells, including T cells. Gamida Cell refers to the two components
collectively as “omidubicel.”

² ClinicalTrials.gov
identifier NCT02730299.

³ U.S. 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.

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.