- BLA submission for omidubicel, a potentially life-saving
treatment for patients with blood cancers in need of stem cell
transplant, expected in fourth quarter of 2021
- Pre-commercial and manufacturing activities underway to support
potential launch of omidubicel in 2022
- Phase 1/2 clinical trial of allogeneic, off-the-shelf GDA-201
in NHL planned with IND submission anticipated in the second half
of 2021
- Strengthened financial position with sale of $75M exchangeable
senior notes in February 2021; sufficient liquidity to fund the
company’s operations into the second half of 2022
- Company to host conference call at 8:00 a.m. ET today
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious blood
diseases, today reported financial results for the quarter ended
March 31, 2021. The company also highlighted progress with
omidubicel, an advanced cell therapy with positive Phase 3 clinical
data, as a potentially life-saving treatment option for patients in
need of an allogeneic hematopoietic stem cell (bone marrow)
transplant, and GDA-201, a natural killer (NK) cell immunotherapy
in Phase 1/2 development for patients with non-Hodgkin lymphoma
(NHL).
“In the first quarter of this year, we made significant progress
on key initiatives across all functions of our business, starting
with omidubicel, a potentially transformative treatment option for
patients with hematological malignancies,” said Julian Adams,
Ph.D., chief executive officer of Gamida Cell. “We are working
diligently to bring this novel therapy to patients, with submission
of a BLA to the FDA anticipated in the fourth quarter of this year.
We are progressing well with our manufacturing readiness activities
in response to the clear feedback from the FDA regarding
registration of our commercial manufacturing facilities and are
actively building our launch readiness capabilities, including
market access and support services, to ensure a positive patient
experience at transplant at the time of potential FDA
approval.”
“We also continue to expand our clinical pipeline with plans to
submit an IND for our GDA-201 natural killer cell therapy, initiate
a multi-center Phase 1/2 clinical study in NHL and continue to
advance our R&D activities to pursue the development of
genetically modified NAM-enabled NK cells in solid tumors.
Importantly, we are well positioned to deliver our 2021 corporate
goals and objectives toward improving the lives of the patients we
serve,” Dr. Adams continued.
Omidubicel, a proprietary, investigational advanced cell
therapy for allogeneic bone marrow transplant
Omidubicel is the foundational product based on Gamida Cell's
proprietary cell expansion technology. During the quarter, Gamida
Cell continued to advance omidubicel, the first cell therapy for
bone marrow transplant to receive Breakthrough Therapy Designation
from the U.S. Food and Drug Administration (FDA). The company
anticipates submitting a Biologics License Application (BLA) to the
FDA in the fourth quarter of this year, based on the results of an
international, randomized Phase 3 study of omidubicel that was
designed to evaluate the safety and efficacy of omidubicel in
patients with hematologic malignancies undergoing a bone marrow
transplant compared to patients who received a standard umbilical
cord blood transplant. The study achieved its primary endpoint, a
statistically significant reduction in time to neutrophil
engraftment, as well as all key secondary endpoints. A key
milestone in a patient’s recovery, neutrophil engraftment is a
measure of how quickly the stem cells a patient receives in a bone
marrow transplant are established and begin to make healthy new
cells. In the recently completed Phase 3 study, 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). Additionally, the study met key secondary endpoints related
to the speed of platelet engraftment, decrease in infections and
reduction in hospitalizations, all significant clinical measures in
bone marrow transplant.
In February 2021, the company presented details of the results
of the omidubicel Phase 3 study at the Transplantation &
Cellular Therapy Meetings of the American Society of
Transplantation and Cellular Therapy and Center for International
Blood & Marrow Transplant Research. The study’s intent-to-treat
analysis included 125 patients aged 13–65 years with a median age
of 41. Patients were enrolled at more than 30 clinical centers in
the United States, Europe, Asia, and Latin America. Racial and
ethnic diversity and baseline characteristics which were
well-balanced across the two study groups. Diseases included acute
lymphoblastic leukemia, acute myelogenous leukemia, chronic
myelogenous leukemia, myelodysplastic syndrome or lymphoma.
In addition to the efficacy results described above, safety
results were also presented, showing decreased incidence related to
grade III/IV acute GvHD (14 percent for omidubicel, 21 percent for
the comparator) and comparable results for all grades chronic GvHD
at one year (35 percent for omidubicel, 29 percent for the
comparator). Transplants with umbilical cord blood, the comparator,
have been historically shown to result in low incidence of GvHD in
relation to other graft sources and, in this study, omidubicel
demonstrated a similar GvHD profile.
The data from the study relating to exploratory endpoints also
supported the clinical benefit demonstrated by the study’s primary
and secondary endpoints. The rate of infection was significantly
reduced for patients randomized to omidubicel, with the cumulative
incidence of first grade II or grade III bacterial or invasive
fungal infection for patients randomized to omidubicel of 37
percent, compared to 57 percent for the comparator (p = 0.027).
Additionally, the study demonstrated a reduction in the incidence
of viral infections. Non-relapse mortality was 11 percent for
patients randomized to omidubicel and 24 percent for patients
randomized to the comparator (p=0.09). Overall survival at 15
months following randomization was 73 percent for patients
randomized to omidubicel and 62 percent for patients randomized to
control (p=0.16), median overall survival was not yet reached.
Non-relapse mortality and overall survival were exploratory
endpoints that were not powered for statistical significance. When
considering the patient experience following transplant, faster
hematopoietic recovery, fewer bacterial and viral infections and
fewer days in hospital are all meaningful results and represent
potentially important advancements in care. Learn more.
Gamida Cell also reported data from the Phase 3 study in March
2021, in an oral session at the Presidential Symposium of the 47th
Annual Meeting of the European Society for Blood and Marrow
Transplantation (EBMT 2021). Additionally, the session was featured
in a panel discussion, “EBMT Talks: Live with the Best
Abstracts.”
Additional omidubicel highlights:
- Progress with commercial manufacturing readiness: Gamida
Cell is making important progress to address the clear feedback
received during a Type B meeting with the FDA in December 2020 for
commercial manufacturing facilities to be ready for BLA submission.
These facilities include the Gamida Cell facility in Israel and a
commercial facility for which the company has a contractual
relationship with Lonza. Both of these facilities are currently on
track to meet the FDA requirements that will be required for BLA
submission.
- Continued launch readiness: The company continues to
progress commercial launch readiness activities for the potential
launch of omidubicel in 2022, pending FDA approval. Based on market
research insights, there is a clear opportunity to improve outcomes
based on clinical needs with current donor sources, increase access
for patients who are eligible and not matched for transplant, and
increase patient eligibility based on the encouraging clinical
profile of omidubicel.
- Gamida Cell announced the Gamida Cell Assist program.
The transplant process can be challenging and complex for patients,
caregivers and the entire transplant care team. Gamida Cell Assist
is designed to focus on patient access and support at each step of
the process. Once the program is launched, the Gamida Cell Assist
case management team will provide a consistent, single point of
contact for patients and health care professionals, work with the
transplant center to track production of omidubicel for each
individual patient, and provide real-time updates on the status of
the therapy. The services provided will include coverage and
reimbursement support, which may include financial, travel and
lodging assistance. Gamida Cell is committed to supporting a
positive journey for patients and their transplant teams so they
can focus on what matters most, the patient experience and
successful clinical outcomes. Learn more.
- Phase 1/2 study of omidubicel in patients with severe
aplastic anemia: Gamida Cell is actively evaluating omidubicel
in an investigator-sponsored Phase 1/2 study in patients with
severe aplastic anemia (SAA). Results to date have shown 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.
GDA-201, a proprietary innate NK cell immunotherapy
- Continued advancement of Phase 1/2 study of GDA-201:
Gamida Cell is preparing for the submission of an investigational
new drug (IND) application for cryopreserved, off-the-shelf GDA-201
to enable a multi-center, Phase 1/2 clinical study in patients with
NHL in the second half of this year. Gamida Cell is pioneering a
potentially curative, novel approach that harnesses the power of
its cell expansion technology, which improves antibody-dependent
cellular cytotoxicity and tumor targeting of NK cells. Learn
more.
- Advancing NK cell R&D activities: The company
continues to advance R&D activities to support pipeline growth,
including the development of genetically modified NK cells.
Corporate Highlights
- Strengthened financial position: In February 2021, the
company completed a $75 million financing with Highbridge Capital
Management, LLC, before deducting offering expenses. This financing
will be used to support manufacturing, regulatory and potential
commercial development activities for omidubicel and to further the
preclinical and clinical development of GDA-201.
First Quarter 2021 Financial Results
- Research and development expenses in the first quarter of 2021
were $11.4 million, compared to $7.9 million for the same period in
2020. The increase was mainly due to omidubicel commercial
manufacturing readiness activities and advancing the GDA-201
program, including broadening the company’s scientific capabilities
and talent.
- Commercial expenses in the first quarter of 2021 were $4.4
million compared to $1.5 million for the first quarter of 2020. The
increase was mainly attributed to progress with commercial
readiness activities, including the hiring of an experienced
commercial leadership team.
- General and administrative expenses were $3.4 million for the
first quarter of 2021 compared to $3.0 million for the same period
in 2020. The increase was mainly due to the hiring of key
management positions to support the growth of the business.
- Finance income, net, was $0.7 million for the first quarter of
2021, compared to finance income, net, of $1.7 million for the
first quarter of 2020. The decrease was primarily due to interest
expenses following the recent $75M financing with Highbridge
Capital Management, and non-cash expense resulting from revaluation
of warrants, and Israeli Innovation Authority royalty-bearing grant
liability.
- Net loss for the first quarter of 2021 was $18.0 million,
compared to a net loss of $10.6 million for the same period in
2020.
- As of March 31, 2021, Gamida Cell had total cash and cash
equivalents of $174.8 million, compared to $127.2 million as of
December 31, 2020.
2021 Financial Guidance
Gamida Cell expects cash used for ongoing operating activities
in 2021 to range from $110 million to $120 million.
Gamida Cell expects that its current cash and cash equivalents
will support the company’s ongoing operating activities into the
second half of 2022. This cash runway guidance is based on the
company’s current operational plans and excludes any additional
funding and any business development activities that may be
undertaken.
Expected 2021-2022 Milestones and Key Events
Gamida Cell expects the following milestones and key events
through 2022:
Omidubicel
- BLA submission to the FDA in the fourth quarter of 2021
- Manufacturing and launch readiness activities ongoing for
potential FDA approval in 2022
GDA-201
- Submit company-sponsored IND application to the FDA and
initiate a Phase 1/2 clinical study in NHL patients in the second
half of 2021
Conference Call Information
Gamida Cell will host a conference call today, May 11, 2021, at
8:00 a.m. ET to discuss these financial results and company
updates. A live webcast of the conference call can be accessed in
the “Investors & Media” 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 5258448. A recording of the webcast will be
available approximately two hours after the event, for
approximately 30 days.
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.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 established by the FDA 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 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 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 have not been established by the FDA 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.
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,
commercialization efforts and Gamida Cell’s expectations regarding
its projected ongoing operating activities 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 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 March 9, 2021, as amended, 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
INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION
U.S. dollars in thousands March 31,
December 31,
2021
2020
2020
Unaudited Audited ASSETS CURRENT
ASSETS: Cash and cash equivalents
$
174,798
$
40,292
$
127,170
Prepaid expenses and other current assets
3,352
1,637
2,815
Total current assets
178,150
41,929
129,985
NON-CURRENT ASSETS: Property, plant and equipment, net
21,162
8,543
18,238
Right-of-use assets
5,920
5,820
6,474
Other assets
772
637
786
Total non-current assets
28,854
15,000
25,498
Total assets
$
206,004
$
56,929
$
155,483
March 31, December 31,
2021
2020
2020
Unaudited Audited LIABILITIES AND EQUITY
CURRENT LIABILITIES: Trade payables
$
7,204
$
3,098
$
6,329
Employees and payroll accruals
3,854
2,549
4,705
Current maturities of lease liabilities
2,076
1,535
2,532
Accrued interest
634
-
-
Accrued expenses and other payables
6,099
2,922
7,988
Total current liabilities
19,867
10,104
21,554
NON-CURRENT LIABILITIES: Liabilities presented at fair value
9,758
2,773
12,043
Employee benefit liabilities, net
768
773
768
Other long-term liabilities
4,988
4,920
5,378
Liability to Israel Innovation Authority (IIA)
18,080
13,077
17,003
Convertible senior notes, net
68,646
-
-
Total non-current liabilities
102,240
21,543
35,192
SHAREHOLDERS' EQUITY: Share capital - Ordinary shares of NIS
0.01 par value -
167
92
166
Authorized: 100,000,000 shares at March 31, 2021 and 2020
(unaudited) and December 31, 2020; Issued and outstanding:
59,247,838 and 33,696,582 shares at March 31, 2021 and 2020
(unaudited), respectively and 59,000,153 shares at December 31,
2020. Share premium
378,478
239,897
375,280
Capital reserve
-441
-541
-441
Accumulated deficit
-294,307
-214,166
-276,268
Total shareholders' equity
83,897
25,282
98,737
Total liabilities and
shareholders' equity
$
206,004
$56,929
$
155,483
INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
U.S. dollars in thousands Three months
endedMarch 31, Year endedDecember 31,
2021
2020
2020
Unaudited Audited Operating expenses: Research
and development, net
$ 11,366
$ 7,879
$ 41,385
Commercial activities
4,430
1,468
8,748
General and administrative
3,413
2,994
12,167
Operating loss
19,209
12,341
62,300
Finance expense
1,690
919
10,640
Finance income
-2,413
-2,658
-236
Loss before tax benefit
18,486
10,602
72,704
Tax benefit
-447
-
-
Net loss
18,039
10,602
72,704
Other comprehensive loss: Items that will be
reclassified subsequently to profit or loss: Actuarial net gain of
defined benefit plans
-
-
-100
Changes in the fair value of marketable securities
-
4
4
Total comprehensive loss
$ 18,039
10,606
$ 72,608
Net loss per share: Basic loss per share
$ 0.31
$ 0.31
$ 1.66
Diluted loss per share
$ 0.34
$ 0.31
$ 1.66
Weighted average share count
60,011,038
33,674,506
43,725,584
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS U.S.
dollars in thousands (except share and per share data)
Three months endedMarch 31, Year endedDecember
31,
2021
2020
2020
Unaudited Audited Cash
flows from operating activities: Net loss
$ (18,039
)
$ (10,602
)
$ (72,704
)
Adjustments to reconcile net loss to net cash used in operating
activities: Adjustments to the profit or loss items:
Depreciation of property, plant and equipment and right-of-use
assets
635
550
2,397
Financial income (expense) net
485
-132
483
Share-based compensation
1,014
899
2,864
Change in employee benefit liabilities, net
-
-
94
Amortization of premium on available-for-sale financial assets
-
4
4
Revaluation of liabilities presented at fair value
-2,285
-2,448
6,822
Revaluation of liability to IIA
1,026
722
4,302
Deferred income taxes
-447
-
-
428
-405
16,966
Changes in asset and liability items: Increase in prepaid
expenses, other current assets and other assets
-523
-458
-1,626
Increase in trade payables
875
1,934
5,083
Increase (decrease) in accrued expenses and other payables
-2,724
-3,096
3,454
-2,372
-1,620
6,911
Cash received during the period
for: Interest received
-
348
361
Interest paid
-51
-47
-161
Net cash used in operating activities
-20,034
-12,326
-48,627
Cash flows from investing
activities: Purchase of property, plant and equipment
-2,806
-2,119
-11,804
Proceeds from maturity of marketable securities
-
-
-158
Proceeds from sale of marketable securities
-
13,551
13,551
Net cash provided by (used in) investing activities
$ (2,806
)
$ 11,432
$ 1,589
Three months endedMarch 31, Year
endedDecember 31,
2021
2020
2020
Unaudited Audited Cash
flows from financing activities: Proceeds from
secondary offering, net
-
-
133,316
Receipt of grants from the IIA
52
53
399
Proceeds from issuance of convertible senior notes, net of issuance
costs
71,012
-
-
Payment of lease liabilities
-664
-787
-1,985
Exercise of options
502
6
650
Payment of issuance costs related to public offering
-468
-
-
Net cash (used in) provided by financing activities
70,434
-728
132,380
Exchange differences on balances of cash and cash
equivalents
34
76
-10
Increase (decrease) in cash and cash equivalents
47,628
-1,546
85,332
Cash and cash equivalents at beginning of period
127,170
41,838
41,838
Cash and cash equivalents at end of period
$ 174,798
$ 40,292
$ 127,170
Supplemental disclosure of non-cash
financing activities: Significant non-cash transactions: Lease
liabilities arising from new right-of-use asset
$ -
$ -
$ 3,409
IIA liability for grants to be received
$ 49
$ -
$ 103
Issuance expenses on credit
$ 235
$ -
$ 468
Purchase of property, plant and equipment on credit
$ 216
$ 206
$ 415
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210511005603/en/
For investors: Stephanie Ascher Stern Investor Relations, Inc.
stephanie.ascher@sternir.com 1-212-362-1200
For media: Rhiannon Jeselonis Ten Bridge Communications
rhiannon@tenbridgecommunications.com 1-978-417-1946
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