—Omidubicel results in improved clinical
outcomes as measured by reduction in time to neutrophil
engraftment, time in hospital and infections following bone marrow
transplant—
— Company announces omidubicel commercial
readiness plan, including the creation of Gamida Cell Assist, to
support a positive patient and transplant center experience in
preparation for potential launch of omidubicel as early as Q4 2021,
subject to ongoing FDA discussions—
—Provides update on Phase 1 study of
investigational natural killer cell therapy GDA-201 and that Phase
2 study of omidubicel in patients with severe aplastic anemia has
demonstrated sustained early engraftment, as reported at ASH Annual
Meeting—
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious
hematologic diseases, today will be providing an update on the
Phase 3 clinical study of omidubicel, commercial readiness plan and
pipeline at its virtual Pipeline Deep Dive event.
“Our goal with omidubicel is to revolutionize the field of bone
marrow transplantation and bring a potentially curative cell
therapy option to thousands of patients who are in need of a bone
marrow transplant, but lack a suitable stem cell donor. These
results bring us one step closer towards that goal,” said Julian
Adams, Ph.D., chief executive officer of Gamida Cell. “What’s more,
transplantation with omidubicel has been shown to result in more
rapid neutrophil engraftment, a decrease in the amount of time
patients spend in hospital, and a reduction in infections. These
are very meaningful outcomes for patients and may also lessen the
financial costs of certain aspects of the transplant.”
Gamida Cell previously reported top-line data for omidubicel. In
October, the company reported that the omidubicel phase 3 study
achieved its secondary endpoints, analyzed in all randomized
patients (intent-to-treat). In May, Gamida Cell reported that the
study achieved its primary endpoint, demonstrating a highly
statistically significant reduction in time to neutrophil
engraftment, a key milestone in a patient’s recovery from a bone
marrow transplant.
These pivotal data form the basis of a Biologics License
Application (BLA) that Gamida Cell expects to initiate on a rolling
basis before the end of this year. Gamida Cell is preparing to be
launch ready in anticipation of potential FDA approval as early as
the fourth quarter of 2021, subject to ongoing FDA discussions on
manufacturing, quality and other matters.
The live event will be available here. More information about
the Phase 3 study of omidubicel and the other updates included in
this release can be found in the Pipeline Deep Dive presentation
on the Gamida Cell website immediately following the
event.
Details of Phase 3 Endpoints
As previously reported, Gamida Cell achieved positive topline
results from its Phase 3 clinical study evaluating the safety and
efficacy of omidubicel. 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). Neutrophil engraftment
is a measure of how quickly the stem cells a patient receives in a
transplant are established and begin to make healthy new cells, and
rapid neutrophil engraftment has been associated with fewer
infections and shorter hospitalizations.
Today, Gamida Cell announced the details of achieving all three
of the prespecified secondary endpoints of the study, analyzed in
all randomized patients (intent-to-treat). These secondary
endpoints were the proportion of patients who achieved platelet
engraftment by day 42, the proportion of patients with grade 2 or
grade 3 bacterial or invasive fungal infections in the first 100
days following transplant, and the number of days alive and out of
the hospital in the first 100 days following transplant. All three
secondary endpoints demonstrated statistical significance in an
intent-to-treat analysis.
- Platelet engraftment was significantly accelerated with
omidubicel, with 55 percent of patients randomized to omidubicel
achieving platelet engraftment at day 42, compared to 28 percent
for the comparator (p = 0.028).
- Infection rates were significantly reduced for patients
randomized to omidubicel. The cumulative incidence of first grade 2
or grade 3 bacterial or invasive fungal infection for patients
randomized to omidubicel was 37 percent, compared to 57 percent for
the comparator (p = 0.027).
- Total days in hospital were reduced in patients
randomized to omidubicel. The median number of days alive and out
of hospital for patients randomized to omidubicel was 60.5 days,
compared to 48.0 days for the comparator (p = 0.005).
Additionally, Gamida Cell reported that the exploratory
endpoints in the study demonstrated a reduction in the cumulative
incidence of viral infections.
The international, multi-center, randomized Phase 3 study for
omidubicel was designed to evaluate the safety and efficacy of
omidubicel in patients with hematologic malignancies undergoing
allogeneic bone marrow transplant compared to a comparator group of
patients who received a standard umbilical cord blood
transplant.
The company anticipates reporting the full data set in a
peer-reviewed setting in the first half of 2021.
Commercial Readiness
The company discussed the market potential for omidubicel and
launch plans. These included quantifying the market opportunity and
keys aspects for a successful launch.
As it prepares for the potential commercial launch of
omidubicel, the company also announced plans for the Gamida Cell
Assist program, which has been designed to focus on patient access
and support of every individual and their caregiver at each step of
the transplant process. Once the program is launched, the Gamida
Cell Assist case management team would provide a consistent, single
point of contact for patients and health care professionals. This
team would work with the transplant center to track each individual
patient’s omidubicel therapy and provide real-time updates on the
status of the therapy. Gamida Cell Assist is also designed to
provide additional services, including coverage and reimbursement
support, and patient and caregiver support, which may include
financial, travel, and lodging assistance.
“At Gamida Cell we are inspired to cure, with the goal of
pioneering new standards of care for patients with blood cancers
and serious blood diseases,” said Michele Korfin, chief operating
and chief commercial officer of Gamida Cell. “The transplant
process can be challenging and complex for the patient, caregivers
and the entire transplant care team. As we prepare for
commercialization, we have developed Gamida Cell Assist to serve as
a comprehensive support program to focus on assuring a positive
patient experience with omidubicel. We are committed to supporting
patients and their caregivers during every step of their journey
and enabling what matters most, a successful clinical outcome that
makes a meaningful difference for patients.”
Update on Natural Killer Cell Therapy GDA-201
In an oral presentation at the recent American Society of
Hematology (ASH) 62nd Annual Meeting, it was shown that GDA-201 was
well tolerated and no dose limiting toxicities were observed in the
Phase 1 clinical study. GDA-201 demonstrated significant clinical
activity in patients with non-Hodgkin lymphoma, with 13 complete
responses and one partial response observed in 19 patients, for a
response rate of 74 percent. Full details of the presentation can
be found in the press release.
Phase 2 Study of Omidubicel in Patients with Severe Aplastic
Anemia
In a poster presentation at ASH, it was shown that patients with
severe aplastic anemia treated with omidubicel achieved sustained
early engraftment. These data, which were presented on December 5
by Mohamed Samour, M.D., Hematology Branch, National Heart, Lung,
and Blood Institute, Bethesda, MD, are the first evidence that
omidubicel can result in rapid engraftment and can achieve
sustained hematopoiesis in patients who are at high risk for graft
failure with conventional umbilical cord blood transplant.
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.12 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 U.S. Food and Drug
Administration or any other health authority.
About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell
expansion technology to develop GDA-201, an innate natural killer
(NK) cell immunotherapy for the treatment of hematologic and solid
tumors in combination with standard of care antibody therapies.
GDA-201 addresses key limitations of NK cells by increasing the
cytotoxicity and in vivo retention and proliferation in the bone
marrow and lymphoid organs of NK cells expanded in culture. GDA-201
is in Phase 1 development through an investigator-sponsored study
in patients with refractory non-Hodgkin lymphoma and multiple
myeloma.3 For more information on the clinical study of GDA-201,
please visit www.clinicaltrials.gov.
GDA-201 is an investigational therapy, and its safety and
efficacy has not been established by the U.S. Food and Drug
Administration or any other health authority.
About the NAM Therapeutic Platform
Gamida Cell’s proprietary NAM-based cell expansion platform is
designed to enhance the number and functionality of donor cells in
culture, enabling the creation of potentially transformative
therapies that move beyond what is possible with existing
approaches. The NAM therapeutic platform leverages the unique
properties of nicotinamide to enable the expansion of multiple cell
types — including stem cells and natural killer (NK) cells — with
appropriate growth factors to maintain the cells' original
phenotype and potency. This can enable the administration of a
therapeutic dose of cells with the potential to improve patient
outcomes.
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 initiation and progress
of and data reported from the clinical trials of Gamida Cell’s
product candidates, anticipated regulatory filings, launch
readiness and FDA approval, commercialization efforts and Gamida
Cell’s expectations regarding its projected ongoing operating
activities, 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
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 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 Reports on Form 6-K
filed with the SEC on May 18, 2020, August 11, 2020 and November
10, 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.
3 Clinicaltrials.gov identifier NCT03019666
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For investors: Stephanie Ascher Stern Investor Relations,
Inc. stephanie.ascher@sternir.com 1-212-362-1200
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mcorcoran@tenbridgecommunications.com 1-617-866-7350
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