– Announces USAN selection of omidubicel as nonproprietary name for
company’s investigational NAM-expanded hematopoietic stem cells
(formerly known as NiCord®) –
– Patient enrollment in Phase 3 study of omidubicel expected
to be completed in second half of 2019; Topline results expected in
first half of 2020 –
– Phase 1 clinical study of GDA-201 (formerly known as NAM-NK)
continues to progress, with additional data expected in 2H19 –
BOSTON–(BUSINESS WIRE)–lt;a href="https://twitter.com/search?q=%24GMDA&src=ctag" target="_blank"gt;$GMDAlt;/agt;–Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune
therapeutics company, today reported financial results for the quarter
ended March 31, 2019. The company also highlighted continued progress in
advancing its clinical development candidates: omidubicel1
(formerly known as NiCord®), an investigational advanced cell
therapy in Phase 3 clinical development designed to enhance the
life-saving benefits of hematopoietic stem cell (bone marrow)
transplant, and GDA-201 (formerly known as NAM-NK), an investigational,
natural killer (NK) cell-based cancer immunotherapy in Phase 1
development in patients with non-Hodgkin lymphoma and multiple myeloma.
“Gamida Cell is focused on transforming the treatment landscape for
patients with blood cancers and rare, serious hematologic diseases. We
are pleased that omidubicel has been selected as the nonproprietary name
for NiCord, highlighting our progress toward bringing this important
cell therapy to patients in need of a bone marrow transplant,” stated
Julian Adams, Ph.D., chief executive officer of Gamida Cell. “We have
also made several key personnel appointments this year that reflect our
strategic focus on commercial preparedness, including hiring our first
chief commercial officer and nominating new board members who bring
commercial, operational and financial experience to Gamida Cell’s board
of directors.”
Dr. Adams continued, “We are pleased that the multi-center, randomized
Phase 3 study of omidubicel is progressing, with patient enrollment
expected to be complete by the end of this year and topline data
anticipated in the first half of 2020. Positive data from the study
would enable the submission of our first biologics license application
next year, which would be a significant achievement.”
“Earlier this year, we also reported encouraging data from the Phase 1
clinical study of our natural killer cell product candidate, GDA-201,
previously known as NAM-NK. The multiple complete responses observed
emboldened us to begin scaling up our manufacturing process to enable
the evaluation of a cryopreserved formulation of GDA-201 in a
multi-center, multi-dose Phase 1/2 clinical study in patients with
non-Hodgkin lymphoma next year,” Dr. Adams concluded.
Company Highlights
-
Omidubicel selected as nonproprietary name for NiCord: Today
Gamida Cell announced that the United States Adopted Names (USAN)
Council selected omidubicel as the nonproprietary name for Gamida
Cell’s investigational hematopoietic stem cell expanded through the
company’s proprietary nicotinamide-based, or NAM, technology. The USAN
Council aims for global standardization and unification of drug
nomenclature to ensure that drug information is communicated
accurately and unambiguously. Gamida Cell’s lead investigational
product has two components: omidubicel (hematopoietic stem cells
expanded through the company’s proprietary nicotinamide-based, or NAM,
technology) and differentiated immune cells, including T cells. Gamida
Cell refers to the two components collectively as “omidubicel.” Going
forward, Gamida Cell will use the name “omidubicel” in publications
and public statements, at conferences and other forums, and in medical
and commercial-related materials. -
Reported encouraging data for omidubicel and GDA-201 at TCT Annual
Meeting: In February, data from the omidubicel and GDA-201
clinical programs were reported 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 from the completed Phase 1/2 clinical study of
omidubicel demonstrated that recipients who received omidubicel had
rapid and robust reconstitution of key immune cells. Successful immune
reconstitution is an important factor in the recovery of patients
undergoing bone marrow transplant.Data were also reported
from the ongoing Phase 1/2 study of omidubicel in patients with severe
aplastic anemia. In the initial cohort of three patients, all
successfully underwent a bone marrow transplant consisting of
omidubicel plus a haploidentical stem cell graft. The results enable
the initiation of a second cohort of patients to be treated with
omidubicel as a stand-alone graft. Patient enrollment in the
second cohort is expected to begin in the first half of 2019.Additionally,
data reported from the ongoing Phase 1 study of GDA-201 in patients
with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM) demonstrated
that GDA-201 was clinically active, with three complete responses
observed in patients with NHL and one complete response in a patient
with MM. These data, along with safety data showing that GDA-201 was
generally well tolerated, support continued clinical development.
Gamida Cell is planning to initiate a multi-center, Phase 1/2 clinical
study of GDA-201 in patients with NHL in 2020.
-
Evolved Board of Directors to reflect company’s progress toward
commercialization: In March, the company announced the
nominations of Shawn Cline Tomasello and Stephen T. Wills to its board
of directors. These nominations require approval at the Annual
Shareholders Meeting, which will take place in June 2019. Ms.
Tomasello has extensive experience in commercializing first-in-class
medicines for the treatment of cancer, including Yescarta®
(at Kite Pharma, now part of Gilead Sciences) and Imbruvica®
(at Pharmacyclics, now part of AbbVie). Mr. Wills has extensive
operational, financial and transactional experience over nearly three
decades in the life sciences and accounting industries. He has served
as chief financial officer of Palatin Technologies, a publicly-traded
biotechnology company developing peptide therapeutics, since 1997 and
also serves as Palatin’s chief operating officer and executive vice
president.In January, the company appointed Nurit
Benjamini to Gamida Cell’s board of directors and chair of the board’s
audit committee. Ms. Benjamini has served as chief financial officer
of TabTale Ltd. since 2013. Previously, she held a number of chief
financial officer positions, including at Wix.com Ltd., Sigma Designs
Israel Ltd. and Compugen Ltd.
-
Appointed Thomas Klima as chief commercial officer: In January,
the company announced the appointment of Thomas Klima as chief
commercial officer. In this newly created role, Mr. Klima will
be responsible for building the team and executing the strategy to
potentially bring omidubicel to patients, including oversight of
reimbursement and patient services. Klima brings nearly 20
years of global experience in the pharmaceutical industry with
expertise in cellular therapy, hematology, oncology and
transplantation. During his career, he has played key roles in
building commercial organizations and leading multiple successful
product launches.
Anticipated 2019-2020 Milestones
Gamida Cell’s anticipated
program milestones in 2019-2020 are as follows:
Omidubicel
-
Initiate Cohort 2 in the Phase 1/2 study evaluating omidubicel as
stand-alone graft in severe aplastic anemia in the first half of 2019 -
Complete enrollment in Phase 3 study of omidubicel in patients with
hematologic malignancies in the second half of 2019 -
Report topline data from the Phase 3 study of omidubicel in patients
with hematologic malignancies in the first half of 2020 -
Complete BLA submission for omidubicel in hematologic malignancies in
the second half of 2020, should Phase 3 data be positive
GDA-201
-
Complete patient enrollment in the ongoing Phase 1 study in the second
half of 2019 - Present additional data at a medical meeting in the second half of 2019
-
Initiate multi-center, Phase 1/2 clinical study in patients with NHL
in 2020
First Quarter 2019 Financial Results
-
As of March 31, 2019, Gamida Cell had total cash, cash equivalents and
available-for-sale securities of $50.3 million, compared to $60.7
million as of December 31, 2018. -
Research and development expenses in the first quarter of 2019 were
$7.3 million, compared to $5.1 million in the same period in 2018. The
difference was attributable mainly to a $1.2 million increase in
clinical activities relate to the advancement of omidubicel and
GDA-201, $0.5 million reduction in grants received from the Israeli
Innovation Authority (IIA) and an increase of $0.5 million in
compensation and other R&D expenses. -
General and administrative expenses were $3.8 million for the first
quarter of 2019, compared to $1.7 million in the same period in 2018.
The increase was due mainly to a $1.0 million increase in expenses
related to hiring and establishing the U.S. headquarters, an increase
of $0.5 million in non-cash stock-based compensation expenses, and
$0.6 million in professional services, rent and other expenses. -
Finance expenses, net, were $4.4 million for the three months ended
March 31, 2019, compared to $0.7 million in income in the same period
in 2018. The increase was primarily due to noncash expenses resulting
from revaluation of warrants and the revaluation of royalty-bearing
grant IIA liability. -
Net loss for the first quarter of 2019 was $15.5 million, compared to
a net loss of $7.4 million in the same period in 2018.
2019 Financial Guidance
Gamida Cell continues to expect cash
used for ongoing operating activities in 2019 to range from $35-$40
million, reflecting anticipated expenditures to advance the company’s
clinical programs.
Gamida Cell expects that its cash, cash equivalents, available-for-sale
securities and short-term debt will support the company’s capital needs
through the data readout for the Phase 3 clinical study of omidubicel,
which is expected in the first half 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.
Conference Call Information
Gamida Cell will host a
conference call today, May 7, 2019, at 8:30 a.m. ET to discuss these
financial results and company updates. A live webcast of the conference
call can be accessed in the “Investors” section of Gamida Cell’s website
at www.gamida-cell.com.
To participate in the live call, please dial 866-930-5560 (domestic) or
409-216-0605 (international) and refer to conference ID number 2277888.
A replay of the webcast will be available for approximately 30 days.
About Omidubicel
Omidubicel (formerly known as 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).1 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.2 A Phase 3 study evaluating
omidubicel in patients with leukemia and lymphoma is ongoing in the
U.S., Europe and Asia.3 Omidubicel 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 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.5
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 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 patient enrollment in and timing of
initiation and progress of and data reported from the clinical trials 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 and
expansion 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.
| INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION | ||||||||||
| U.S. dollars in thousands | ||||||||||
| March 31, | December 31, | |||||||||
| 2019 | 2018 | |||||||||
|
ASSETS |
||||||||||
|
CURRENT ASSETS: |
||||||||||
| Cash and cash equivalents | $ | 43,749 | $ | 40,272 | ||||||
| Available-for-sale financial assets | 6,507 | 20,417 | ||||||||
| Prepaid expenses and other current assets | 684 | 1,502 | ||||||||
|
Total current assets |
50,940 | 62,191 | ||||||||
|
NON-CURRENT ASSETS: |
||||||||||
| Property and equipment, net | 2,782 | 2,311 | ||||||||
| Right-of-use assets | 6,668 | – | ||||||||
| Other assets | 657 | 662 | ||||||||
|
Total non-current assets |
10,107 | 2,973 | ||||||||
|
Total assets |
$ | 61,047 | $ | 65,164 | ||||||
|
LIABILITIES AND EQUITY |
||||||||||
|
CURRENT LIABILITIES: |
||||||||||
| Trade payables | $ | 1,341 | $ | 1,985 | ||||||
| Employees and payroll accruals | 2,580 | 2,888 | ||||||||
| Current maturities of lease liabilities | 2,156 | – | ||||||||
| Accrued expenses and other payables | 1,739 | 1,832 | ||||||||
|
Total current liabilities |
7,816 | 6,705 | ||||||||
|
NON-CURRENT LIABILITIES: |
||||||||||
| Liabilities presented at fair value | 25,031 | 24,049 | ||||||||
| Employee benefit liabilities, net | 276 | 183 | ||||||||
| Lease Liabilities | 4,671 | – | ||||||||
| Liability to Israel Innovation Authority (IIA) | 10,108 | 9,540 | ||||||||
|
Total non-current |
40,086 | 33,772 | ||||||||
|
SHAREHOLDERS’ EQUITY: |
||||||||||
| Share capital | 68 | 67 | ||||||||
| Share premium | 197,967 | 193,953 | ||||||||
| Capital reserve due to actuarial gains | (160) | (77) | ||||||||
| Available-for-sale reserve | (10) | (43) | ||||||||
| Accumulated deficit | (184,720) | (169,213) | ||||||||
|
Total shareholders’ equity |
13,145 | 24,687 | ||||||||
|
Total liabilities and |
$ | 61,047 | $ | 65,164 | ||||||
1 Gamida Cell’s lead development candidate consists of
omidubicel (expanded hematopoietic stem cells) and differentiated immune
cells, including T cells. Gamida Cell refers to the two components
collectively as “omidubicel.”
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.
5 ClinicalTrials.gov identifier NCT03019666.
Contacts
Jaren Irene Madden
jaren@gamida-cell.com
617-892-9084
Krystle Gibbs (media)
krystle@tenbridgecommunications.com
508-479-6358