– Topline data from Phase 3 study of omidubicel
expected in first half of 2020 –
– GDA-201 program progressing with additional
data expected in first half of 2020 –
– Jas Uppal, Ph.D., appointed as chief
regulatory and quality officer –
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to finding cures for blood cancers and serious
blood diseases, today reported its expected milestones for
2020-2021, which highlight the company’s progress advancing its
clinical development candidates: omidubicel, an advanced cell
therapy in Phase 3 clinical development as a potential life-saving
treatment option for patients in need of bone marrow transplant,
and GDA-201, an investigational, natural killer (NK) cell-based
cancer immunotherapy in Phase 1 development in patients with
non-Hodgkin lymphoma (NHL) and multiple myeloma.
“This is an incredibly important year for Gamida Cell. With
patient enrollment completed, we are expecting topline data from
the Phase 3 study of omidubicel in the first half of this year.
Omidubicel is the first bone marrow transplant product to receive
Breakthrough Therapy Designation from the U.S. Food and Drug
Administration (FDA) and has the potential to be the first
FDA-approved bone marrow transplant graft. Positive data from our
study would represent an important step toward bringing potentially
curative medicines to patients,” stated Julian Adams, Ph.D., chief
executive officer at Gamida Cell. “We are also very excited by the
progress of GDA-201, an investigational therapy in a class that we
believe has the potential to be the next generation of cell
therapies to dramatically improve the treatment of blood cancers.
GDA-201 has shown promise for the treatment of non-Hodgkin
lymphoma, including an aggressive form of lymphoma called diffuse
large B cell lymphoma, and we anticipate announcing additional data
in the first half of 2020.”
Today Gamida Cell also announced the appointment of Jas Uppal,
Ph.D. to the newly created role of chief regulatory and quality
officer. Dr. Uppal brings more than 25 years of global experience
in the pharmaceutical industry, including expertise in hematology,
immunology and neurology. During her career, she has played key
roles in building regulatory organizations and leading multiple
successful product launches.
“We are delighted to welcome Jas to Gamida Cell. Her strategic,
global experience in building teams and securing multiple product
approvals for novel biologics will be invaluable as we move
omidubicel and GDA-201 through critical regulatory milestones,”
stated Dr. Adams.
Dr. Uppal most recently served as vice president, global head of
regulatory affairs of oncology, endocrinology and rare diseases at
Ipsen Biopharmaceuticals, where she held worldwide responsibility
for Ipsen’s oncology, endocrinology and rare diseases portfolio. In
this role, she led all areas of product development and managed a
team of regulatory professionals. Prior to Ipsen, she served as
vice president, global head of regulatory affairs at Karyopharm
Therapeutics, where she was responsible for developing a global
regulatory strategy and approach for multiple Phase 2 and Phase 3
programs that were being developed to treat hematological
malignancies and solid tumors. Earlier in her career, Dr. Uppal
held several regulatory-related positions over the course of 12
years at Biogen Idec (now Biogen) that culminated in her role as
director of global emerging markets and head of development
sciences. Dr. Uppal has participated in over 30 new drug approvals
worldwide and has more than 30 publications in peer reviewed
journals. She holds a Ph.D. in biochemistry from Kings College,
University of London.
Expected 2020-2021 Milestones
Gamida Cell targets achieving the following milestones during
2020-2021:
Omidubicel
- Report topline data from the Phase 3 study in the first half of
2020
- Present data from the Phase 3 study at a medical meeting in the
second half of 2020
- Submit the biologics license application to the FDA in the
second half of 2020, assuming positive data
- Report additional data from the Phase 1/2 study in patients
with severe aplastic anemia in the second half of 2020
- Launch omidubicel in 2021, contingent upon FDA approval
GDA-201
- Present additional data from the Phase 1 study in the first
half of 2020
- Submit company-sponsored investigational new drug application
to FDA in the second half of 2020
- Initiate a Phase 1/2 clinical study in patients with
non-Hodgkin lymphoma in 2021
2020 Financial Outlook
Gamida Cell ended 2019 with cash, cash equivalents and
available-for-sale securities of approximately $56 million
(unaudited). The company expects that its current cash, cash
equivalents and available-for-sale securities will support the
company’s ongoing operating activities into the fourth quarter of
2020. This cash runway guidance is based on the company’s current
operational plans and excludes any additional funding that may be
received or business development activities that may be
undertaken.
About Omidubicel
Omidubicel, 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). Omidubicel 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, omidubicel
demonstrated rapid and durable time to engraftment and was
generally well-tolerated.1 A Phase 3 study evaluating omidubicel in
patients with leukemia and lymphoma is ongoing in the U.S., Europe
and Asia.2 Omidubicel is also being evaluated in a Phase 1/2
clinical study in patients with severe aplastic anemia.3 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.
About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell
expansion technology to develop GDA-201 (formerly known as NAM-NK),
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.4
Omidubicel and GDA-201 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 an advanced cell therapy company committed to
finding cures for 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.
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 patient enrollment in and
timing of initiation and progress of and data reported from the
clinical trials of Gamida Cell’s product candidates, expectations
regarding regulatory filings, approval and commercialization of
Gamida Cell’s product candidates, and Gamida Cell’s expectations
regarding its projected operating expenses and cash runway, which
statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the scope, progress,
expansion and results of Gamida Cell’s clinical trials and
variability, 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 of Gamida Cell’s
public filing on Form 20-F, filed with the SEC on February 25,
2019, 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.
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):367-374.2ClinicalTrials.gov identifier
NCT02730299.3ClinicalTrials.gov identifier NCT03173937.
4ClinicalTrials.gov identifier NCT03019666.
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version on businesswire.com: https://www.businesswire.com/news/home/20200113005222/en/
Jaren Irene Madden jaren@gamida-cell.com 617-286-6264
Max Stendahl (media) max@tenbridgecommunications.com
508-277-8117
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