— Omidubicel represents a potentially
transformative cell therapy treatment option for patients in need
of a bone marrow transplant —
—Newly presented data support clinical benefit
demonstrated by primary and secondary endpoints—
—Company anticipates BLA submission in second
half of 2021—
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious
hematologic diseases, today announced the results of a Phase 3
clinical study of omidubicel presented in an oral session at the
Transplantation & Cellular Therapy Meetings of the American
Society of Transplantation and Cellular Therapy (ASTCT) and Center
for International Blood & Marrow Transplant Research (CIBMTR),
or the TCT Meetings. Omidubicel is an advanced cell therapy under
development as a potential life-saving allogeneic hematopoietic
stem cell transplant solution for patients with hematologic
malignancies.
This clinical data set was from the international, multi-center,
randomized Phase 3 study of omidubicel that was designed to
evaluate the safety and efficacy of omidubicel in patients with
high-risk hematologic malignancies undergoing a bone marrow
transplant compared to a comparator group of patients who received
a standard umbilical cord blood transplant. This is the first
presentation of these data in a peer-reviewed conference. The full
presentation is available on the Gamida Cell website.
“The results of this global Phase 3 study of omidubicel in
patients with hematologic malignancies show that omidubicel
resulted in faster hematopoietic recovery, fewer bacterial and
viral infections and fewer days in hospital, all of which are
meaningful results and represent potentially important advancements
in care when considering the patient experience following
transplant,” said Mitchell Horwitz, M.D., principal investigator
and professor of medicine at the Duke Cancer Institute. “The
comparator, a transplant with umbilical cord blood, has been
historically shown to result in low incidence of graft versus host
disease (GvHD) in relation to other graft sources, and in this
study, omidubicel demonstrated a GvHD profile similar to the
comparator. Moreover, previous studies have shown that engraftment
with omidubicel is durable, with some patients in the Phase 1/2
study receiving their transplant more than 10 years ago. The data
presented at this meeting indicate that omidubicel has the
potential to be considered a new standard of care for patients who
are in need of stem cell transplantation but do not have access to
a matched donor.”
Details of Phase 3 Efficacy and Safety Results Shared at the
TCT Meetings
Patient demographics including racial and ethnic diversity and
baseline characteristics were well-balanced across the two study
groups. The study’s intent-to-treat analysis included 125 patients
aged 13–65 years with a median age of 41. Diseases included acute
lymphoblastic leukemia, acute myelogenous leukemia, chronic
myelogenous leukemia, myelodysplastic syndrome or lymphoma.
Patients were enrolled at more than 30 clinical centers in the
United States, Europe, Asia, and Latin America.
Gamida Cell previously reported in May 2020 that the study
achieved its primary endpoint, showing that omidubicel demonstrated
a statistically significant reduction in time to neutrophil
engraftment, a measure of how quickly the stem cells a patient
receives in a transplant are established and begin to make healthy
new cells, and a key milestone in a patient’s recovery from a bone
marrow transplant. The median time to neutrophil engraftment was 12
days for patients randomized to omidubicel compared to 22 days for
the comparator group (p<0.001).
All three secondary endpoints demonstrated a statistically
significant improvement among patients who were randomized to
omidubicel in relation to patients randomized to the comparator
group (intent-to-treat). Platelet engraftment was significantly
accelerated with omidubicel, with 55 percent of patients randomized
to omidubicel achieving platelet engraftment at day 42, compared to
35 percent for the comparator (p = 0.028). The rate of infection
was significantly reduced for patients randomized to omidubicel,
with the cumulative incidence of first grade 2 or grade 3 bacterial
or invasive fungal infection for patients randomized to omidubicel
of 37 percent, compared to 57 percent for the comparator (p =
0.027). Hospitalization in the first 100 days after transplant was
also reduced in patients randomized to omidubicel, with a median
number of days alive and out of hospital for patients randomized to
omidubicel of 60.5 days, compared to 48.0 days for the comparator
(p = 0.005). The details of these data were first reported in
December 2020.
Previously unpublished data from the study relating to
exploratory endpoints also support the clinical benefit
demonstrated by the study’s primary and secondary endpoints. There
was no statistically significant difference between the two patient
groups related to grade 3/4 acute GvHD (14 percent for omidubicel,
21 percent for the comparator) or all grades chronic GvHD at one
year (35 percent for omidubicel, 29 percent for the comparator).
Non-relapse mortality was shown to be 11 percent for patients
randomized to omidubicel and 24 percent for patients randomized to
the comparator (p=0.09).
These clinical data results will form the basis of a Biologics
License Application (BLA) that Gamida Cell expects to submit to the
U.S. Food and Drug Administration (FDA) in the second half of
2021.
“We believe that omidubicel has the potential to transform the
field of hematopoietic bone marrow transplant by expanding access
to this potentially curative cell therapy treatment for thousands
of patients who are in need of a transplant but lack access to a
matched related donor,” said Julian Adams, Ph.D., chief executive
officer of Gamida Cell. “Sharing the results of the Phase 3 study
of omidubicel with the transplant community is a major moment for
Gamida Cell, and we are forever grateful to the patients who
participated in this study, their caregivers, and the work of the
investigators and their teams.”
About Omidubicel
Omidubicel 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). In both Phase 1/2 and Phase 3
clinical studies (NCT01816230, NCT02730299), omidubicel
demonstrated rapid and durable time to engraftment and was
generally well tolerated.1,2 Omidubicel is also being evaluated in
a Phase 1/2 clinical study in patients with severe aplastic anemia
(NCT03173937). 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 have not been established by the FDA or any other health
authority.
About Gamida Cell
Gamida Cell is an advanced cell therapy company committed to
cures for patients with blood cancers and serious blood diseases.
We harness our cell expansion platform to create therapies with the
potential to redefine standards of care in areas of serious medical
need. For additional information, please visit www.gamida-cell.com
or follow Gamida Cell on LinkedIn or Twitter 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 anticipated regulatory
submissions, which statements are subject to a number of risks,
uncertainties and assumptions, including, but not limited to the
progress and expansion of Gamida Cell’s manufacturing capabilities
and other commercialization efforts 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 and other sections of Gamida Cell’s
Annual Report on Form 20-F, filed with the Securities and Exchange
Commission (SEC) on February 26, 2020, its Report on Form 6-K filed
with the SEC on August 12, 2020, 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 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.
2 Gamida Cell press release, “Gamida Cell Announces Positive
Topline Data from Phase 3 Clinical Study of Omidubicel in Patients
with High-Risk Hematologic Malignancies,” issued May 12, 2020. Last
accessed August 31, 2020.
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For investors: Stephanie Ascher Stern Investor Relations,
Inc. stephanie.ascher@sternir.com 1-212-362-1200
For media: Matthew Corcoran Ten Bridge Communications
mcorcoran@tenbridgecommunications.com 1-617-866-7350
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