– NAM-NK therapy clinically active and generally well tolerated, with
multiple complete responses observed –
– Data in severe aplastic anemia support potential of NiCord to treat
non-malignant bone marrow failure disorders –
– Gamida Cell to host conference call today at 8:00 a.m. ET –
HOUSTON--(BUSINESS WIRE)--Feb. 21, 2019--
Gamida Cell Ltd. (Nasdaq:GMDA), a leading cellular and immune
therapeutics company, today announced that new data from its NAM-NK and
NiCord® programs was presented at the 2019 Transplantation &
Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow
Transplantation and Center for International Blood and Marrow Transplant
Research taking place in Houston, Texas. Data reported from the first 14
patients in the ongoing Phase 1 study of NAM-NK, an investigational,
cell-based cancer immunotherapy, in patients with non-Hodgkin lymphoma
(NHL) and multiple myeloma (MM) demonstrated that NAM-NK was highly
active, with three complete responses observed in patients with NHL and
one complete response in a patient with MM. These data, along with
additional safety data showing that NAM-NK was generally well tolerated,
support continued clinical development. Gamida Cell is planning to
initiate a multi-center, Phase 1/2 clinical study of NAM-NK in 2020.
NAM-NK cells are natural killer cells that have been expanded using
Gamida Cell’s proprietary nicotinamide-based, or NAM, technology.
“I am encouraged by the emerging clinical profile of NAM-NK, and it is
particularly exciting to witness complete responses in this
heavily-pretreated patient population. Following treatment, two of the
patients who were in complete remission received a bone marrow
transplant, which has curative potential,” stated M Health
Hematologist/Oncologist Veronika Bachanova, M.D., Ph.D., Associate
Professor of Medicine, Section Head for Malignant Hematology in the
Division of Hematology, Oncology and Transplantation, University of
Minnesota Medical School and Masonic Cancer Center member. “I look
forward to continuing to evaluate the potential of NAM-NK as the study
progresses.”
Additionally, data were reported from the ongoing Phase 1/2 study of
NiCord, an investigational advanced cell therapy designed to enhance and
expand the life-saving benefits of hematopoietic stem cell (bone marrow)
transplant, in patients with severe aplastic anemia. In the initial
cohort of three patients, all three successfully underwent a stem cell
transplant consisting of NiCord plus a haploidentical stem cell graft.
The rapid engraftment, sustained hematopoiesis and accelerated immune
recovery observed in these patients enable the initiation of a second
cohort of patients to be treated with NiCord as a stand-alone graft.
“Data from our NiCord and NAM-NK programs continue to demonstrate the
transformative potential of our proprietary nicotinamide-, or NAM-based,
cell expansion technology. We are pleased to be at the forefront of
exploring NK-therapy, which we believe has potential to advance
treatment paradigms for patients just as CAR T therapy provided
ground-breaking treatment options for patients,” stated Julian Adams,
Ph.D., chief executive officer at Gamida Cell. “We are also encouraged
by the initial NiCord data in severe aplastic anemia, which supports
further exploring a reduced intensity regimen and highlights the
potential of NiCord as a bone marrow transplant solution not only for
patients with hematologic malignancies but also for patients with severe
bone marrow failure disorders. We look forward to continued progress
with both programs throughout 2019.”
NAM-NK Data in Patients with NHL and MM
The safety and activity of NAM-NK is currently being evaluated in a
Phase 1 dose-escalation study. Patients received rituximab (NHL
patients) or elotuzumab (MM patients) prior to and after NAM-NK
infusion. The presentation, “First-in-Human Phase I Study of
Nicotinamide-Expanded Related Donor Natural Killer Cells for the
Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma and Multiple
Myeloma” (Poster #242), included six patients with NHL and eight
patients with MM. All 14 patients were evaluable for safety, and 12 of
14 patients were evaluable for activity (all six NHL patients and six of
eight MM patients). The majority of patients were heavily pre-treated
and had advanced disease.
Among the six NHL patients, three patients achieved a complete response,
one patient achieved a partial response, and two patients experienced
progressive disease. Two of the patients who achieved a complete
response subsequently received a bone marrow transplant. Among the six
MM patients evaluable for activity, one patient achieved a complete
response, two patients experienced stable disease, and three patients
experienced progressive disease. Activity was observed at all three dose
levels evaluated.
NAM-NK was generally well tolerated, with no graft vs. host disease
(GvHD), no tumor lysis syndrome and no neurotoxicity syndrome observed.
Grade 3 (n = 3) and Grade 4 (n = 1) hematologic adverse events were
observed. Non-hematologic adverse events were mostly Grade 1 and Grade
2. There was one case of Grade 3 cytokine release syndrome and one death
due to sepsis.
NiCord Data in Patients with Severe Aplastic Anemia
The safety and activity of NiCord in patients with severe aplastic
anemia is being evaluated in an ongoing Phase 1/2 study. The
presentation, “Ex Vivo Nicotinamide-Expanded (NAM-Expanded) Unrelated
Cord Blood Transplantation (UCB) for Refractory Severe Aplastic Anemia
Results in Rapid Engraftment and Expedites Immune Recovery” (Poster
#295), included data from three severe aplastic anemia patients with
severe neutropenia who failed immunosuppressive therapy.
All three patients enrolled in the first cohort were successfully
treated with reduced intensity conditioning regimens and underwent a
bone marrow transplant consisting of NiCord plus a haploidentical stem
cell graft. Engraftment occurred rapidly, with a median neutrophil
recovery of 6 days (range: 6-7 days), which was sustained at day 100,
and was superior to that observed in a retrospective cohort of 16
patients who received a single unexpanded umbilical cord blood
transplant and haploidentical cells using the same conditioning regimen
(P = 0.006). At median follow-up of 11 months (range 4-18 months), all
three patients who received NiCord were alive and GvHD-free.
Conference Call Information
Gamida Cell will host a conference call and webcast today, Thursday,
February 21, 2019, at 8:00 a.m. ET to review the data from its NAM-NK
and NiCord programs that are being presented at the 2019 TCT Annual
Meeting. A live webcast of the conference call can be accessed in the
Investors section of Gamida Cell’s website at https://investors.gamida-cell.com.
To participate in the conference call, please dial 1-866-930-5560
(domestic) or 1-409-216-0605 (international) five minutes prior to start
time. The conference ID number is 9462948. An archived version of the
webcast will be available on Gamida Cell’s website for 30 days.
About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion
technology to highly functional NK cells to develop NAM-NK, an innate
immunotherapy for the treatment of hematologic and solid tumors in
combination with standard of care antibody therapies. NAM-NK 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. NAM-NK is in Phase 1 development through an
investigator-sponsored study in patients with refractory non-Hodgkin
lymphoma and multiple myeloma.1
About NiCord
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). NiCord 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,
NiCord demonstrated rapid and durable time to engraftment and was
generally well-tolerated.2 A Phase 3 study evaluating NiCord
in patients with leukemia and lymphoma is ongoing in the U.S., Europe
and Asia.3 NiCord is also being evaluated in a Phase 1/2
clinical study in patients with severe aplastic anemia.4 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 NiCord. For more information on
clinical trials of NiCord, please visit www.clinicaltrials.gov.
NAM-NK and NiCord are investigational therapies, and their 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 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 initiation and timing of a Phase 1/2 study
of NAM-NK and expansion of the Phase 1/2 study of NiCord for the
treatment of severe aplastic anemia, which statements are subject to a
number of risks, uncertainties and assumptions, including, but not
limited to the scope and progress of Gamida Cell’s studies. In light of
these risks and uncertainties, and other risks and uncertainties that
are described in the Risk Factors section of our Registration Statement
on Form F-1 filed with the SEC on September 28, 2018, 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 ClinicalTrials.gov
identifier NCT03019666.
2 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.
3
ClinicalTrials.gov identifier NCT02730299.
4
ClinicalTrials.gov identifier NCT03173937.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190221005284/en/
Source: Gamida Cell Ltd.
Jaren Irene Madden
jaren@gamida-cell.com
1-617-286-6264
Media Inquiries
Krystle Gibbs
krystle@tenbridgecommunications.com
508-479-6358
