Gamida Cell Announces Publication in Blood, the Journal of the American Society of Hematology, of the First Pivotal Trial to ...
June 23 2021 - 7:00AM
Business Wire
- Omidubicel is a first-in-class, NAM-enabled, advanced cell
therapy being evaluated as a potential life-saving treatment for
patients with blood cancers in need of an allogeneic hematopoietic
stem cell (bone marrow) transplant
- The Phase 3 clinical trial achieved both primary and secondary
endpoints
- Gamida Cell remains on track to submit a Biologics License
Application for omidubicel in the fourth quarter of this year
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious
hematologic diseases, today announced that the results of a Phase 3
clinical study of omidubicel have been published in Blood, the
official journal of the American Society of Hematology. Omidubicel
is an advanced cell therapy under development as a potential
life-saving allogeneic hematopoietic stem cell transplant solution
for patients with hematologic malignancies.
The results demonstrate that transplantation with omidubicel
leads to faster neutrophil and platelet recovery compared to a
standard umbilical cord blood graft, and results in fewer early
bacterial and viral infections and less time in the hospital.
“We are pleased that the data from this well-conducted
international Phase 3 trial have been published in Blood, the
highly respected, peer-reviewed journal of the American Society of
Hematology,” said Ronit Simantov, M.D., chief medical officer of
Gamida Cell. “The robust results of this clinical trial have
demonstrated that omidubicel could provide an important new option
for patients with hematologic malignancies in need of a bone marrow
transplant.”
Data from this study were previously presented at the
Transplantation & Cellular Therapy Meetings of the American
Society of Transplantation and Cellular Therapy and Center for
International Blood & Marrow Transplant Research, and most
recently during the Presidential Symposium at the 47th Annual
Meeting of the European Society for Blood and Marrow
Transplantation. The pivotal study was an international,
multi-center, randomized Phase 3 trial designed to compare the
safety and efficacy of omidubicel to standard umbilical cord blood
transplant in patients with high-risk hematologic malignancies
undergoing a bone marrow transplant.
“Previous studies have shown that engraftment with omidubicel is
durable, with some patients in the Phase 1/2 study now a decade
past their transplant. The Phase 3 data reinforce omidubicel’s
potential to be a new standard of care for patients who are in need
of stem cell transplantation but do not have access to an
appropriate matched donor,” said Mitchell Horwitz, M.D., lead
author of the paper and a professor of medicine at the Duke Cancer
Institute.
The full Blood manuscript is available here:
https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.
Details of Phase 3 Efficacy and Safety Results Shared in
Blood
The intent-to-treat analysis included 125 patients aged 13–65
years with a median age of 41. Forty-four percent of the patients
treated on study were non-Caucasian, a population known to be
underrepresented in adult bone marrow donor registries. Patient
demographics and baseline characteristics were well-balanced across
the two study groups. Patients with acute lymphoblastic leukemia,
acute myelogenous leukemia, chronic myelogenous leukemia,
myelodysplastic syndrome or lymphoma 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, details of which were first
reported in December 2020, demonstrated a statistically
significant improvement among patients who were randomized to
omidubicel compared to patients randomized to standard cord blood
graft. 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). 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 61 days, compared to 48
days for the comparator (p=0.005). 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).
Additional data reported in the manuscript included a comparison of
infection density, or the number of infections during the first
year following transplantation, which showed that the risk for
grade 2 and grade 3 infections was significantly lower among
recipients of omidubicel compared to control (risk ratio 0.5,
p<0.001).
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 in incidence of 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 form the basis of a Biologics
License Application (BLA) that Gamida Cell plans to submit to the
U.S. Food and Drug Administration (FDA) in the fourth quarter of
2021.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a
potential life-saving allogeneic hematopoietic stem cell (bone
marrow) transplants for patients with hematologic malignancies
(blood cancers), for which it has been granted Breakthrough Status
by the FDA. 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 the potential for omidubicel to
become a new standard of care and the anticipated submission of a
BLA for omidubicel, which statements are subject to a number of
risks, uncertainties and assumptions, including, but not limited to
Gamida Cell’s ability to prepare regulatory filings and the review
process therefor; complications in Gamida Cell’s plans to
manufacture its products for commercial distribution; 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 March 9, 2021, as
amended on March 22, 2021, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210623005336/en/
For investors: Stephanie Ascher Stern Investor Relations,
Inc. stephanie.ascher@sternir.com 1-212-362-1200
For media: Rhiannon Jeselonis Ten Bridge Communications
rhiannon@tenbridgecommunications.com 1-978-417-1946
Gamida Cell (NASDAQ:GMDA)
Historical Stock Chart
From Mar 2024 to Apr 2024
Gamida Cell (NASDAQ:GMDA)
Historical Stock Chart
From Apr 2023 to Apr 2024