— Study shows younger donor age is associated
with improved outcomes following bone marrow transplant —
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious blood
diseases, today announced data from an observational study
demonstrating that younger donor age is associated with more rapid
time to neutrophil engraftment and increased overall survival in
patients who received hematopoietic stem cell transplant (HSCT, or
bone marrow transplant) for the treatment of hematological
malignancies. These data are being presented at the Cord Blood
Connect Meeting, which is taking place virtually today and on
September 17.
The study is the result of a research agreement between Gamida
Cell and the CIBMTR® (Center for International Blood and Marrow
Transplant Research®) designed to collect and analyze health
outcomes data in patients with hematologic malignancies who receive
a hematopoietic stem cell transplant or cellular therapy infusion,
including bone marrow transplant graft from various donor sources.
The study evaluated clinical outcomes for 660 patients in the
CIBMTR registry who underwent a bone marrow transplant with a
matched unrelated, mismatched unrelated or haploidentical graft
source contemporaneous to the Phase 3 study of omidubicel, Gamida
Cell’s investigational advanced cell therapy in development as a
treatment option for patients in need of a bone marrow transplant.
Key clinical outcomes, including time to neutrophil engraftment and
overall survival, were improved for patients with donors under the
age of 30.
“As new graft options evolve for bone marrow transplant, and we
as a field learn more about the long-term patient outcomes of these
graft options, the selection algorithms that are used to match
patients with donors must also evolve to take into account the most
current clinical data,” said Ronit Simantov, M.D., chief medical
officer at Gamida Cell. “These data indicate that donor age is a
factor in clinical outcomes and that donor age should be considered
when matching patients with a graft source.”
In May, Gamida Cell reported that its Phase 3 study of
omidubicel achieved its primary endpoint, demonstrating a highly
statistically significant reduction (p < 0.001) in time to
neutrophil engraftment, a key milestone in recovery from a bone
marrow transplant. Omidubicel is the first bone marrow transplant
product to receive Breakthrough Therapy Designation from the U.S.
Food and Drug Administration (FDA). Gamida Cell expects to begin
submitting the biologics license application for omidubicel to the
FDA on a rolling basis in the fourth quarter of 2020.
“This analysis reinforces the importance of considering donor
age when selecting a bone marrow graft source for patients, as
donors aged 30 or younger resulted in improved overall survival,”
said Julian Adams, Ph.D., chief executive officer at Gamida Cell.
“As we look toward our anticipated regulatory submission of
omidubicel to the FDA and potential approval, given that omidubicel
is derived from cord blood, we believe these findings could
potentially have future implications for considering omidubicel for
any patient who does not have an available related or unrelated
donor of suitable age.”
More Details About the Study
This observational study utilizes data from the CIBMTR® registry
to analyze long‐term safety and efficacy data for patients with
hematologic malignancies who underwent a bone marrow transplant
with a matched unrelated, mismatched unrelated or haploidentical
graft source. The criteria for inclusion of patients and the
outcomes evaluated in the analyses were consistent with those in
the Phase 3 study of omidubicel. The median donor age was 30 (range
2-74) years.
Statistical analyses, performed by Gamida Cell, compared
important clinical outcomes for patients with donors ≤ 30 years old
(n=334) to patients with donors > 30 years of age (n=326).
Patient demographics were well-balanced across the two donor age
groups. The study demonstrated that neutrophil recovery was more
rapid in patients with donors ≤ 30 years old (p = 0.013).
Additionally, patients with donors ≤ 30 years of age showed
improved overall survival at one year (p = 0.016).
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 and 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 evaluated by the U.S. Food and Drug
Administration 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.
About the CIBMTR
The CIBMTR® (Center for International Blood and Marrow
Transplant Research®) is a research collaboration between the
National Marrow Donor Program® (NMDP)/Be The Match® and the Medical
College of Wisconsin (MCW). The CIBMTR collaborates with the global
scientific community to advance hematopoietic cell transplantation
(HCT) and cellular therapy worldwide to increase survival and
enrich quality of life for patients. The CIBMTR facilitates
critical observational and interventional research through
scientific and statistical expertise, a network of more than 300
transplant centers, and one of the largest databases worldwide for
clinical outcomes of cellular therapy, and a biorepository with
tissue samples.
For more information on the CIBMTR, please visit www.cibmtr.org
or follow the CIBMTR on Facebook, LinkedIn, or Twitter at
@CIBMTR.
About the National Marrow Donor Program/Be The Match
The National Marrow Donor Program® (NMDP)/Be The Match® is the
global leader in providing a cure to patients with life-threatening
blood and marrow cancers like leukemia and lymphoma, as well as
other diseases. The NMDP/Be The Match manages the world’s largest
registry of potential blood stem cell donors and cord blood units,
connects patients to their donor match for a life-saving marrow or
umbilical cord blood transplant and educates health care
professionals and patients. In 2016, the NMDP/Be The Match
established Be The Match BioTherapies® to accelerate patient access
to life-saving therapies, by providing proven services and support
to companies developing and delivering cell and gene therapies.
Learn more at BeTheMatchClinical.org.
About the Medical College of Wisconsin
With a history dating back to 1893, The Medical College of
Wisconsin (MCW) is dedicated to leadership and excellence in
education, patient care, research and community engagement. More
than 1,200 students are enrolled in MCW’s medical school and
graduate school programs in Milwaukee, Green Bay, and Central
Wisconsin in 2016. MCW’s School of Pharmacy opened in 2017. A major
national research center, MCW is the largest research institution
in the Milwaukee metro area and second largest in Wisconsin. In
FY2016, faculty received more than $184 million in external support
for research, teaching, training and related purposes. This total
includes highly competitive research and training awards from the
National Institutes of Health (NIH). Annually, MCW faculty direct
or collaborate on more than 3,100 research studies, including
clinical trials. Additionally, more than 1,500 physicians provide
care in virtually every specialty of medicine for more than 525,000
patients annually.
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 anticipated timing of
regulatory filing submissions, which statements are subject to a
number of risks, uncertainties and assumptions, including, but not
limited to the ongoing global COVID-19 pandemic 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, 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.
References
1Horwitz 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. 2Gamida 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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Investors: Jaren Irene Madden jaren@gamida-cell.com
1-617-286-6264 Media Inquiries: Matthew Corcoran
mcorcoran@tenbridgecommunications.com 1-617-866-7350
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