- 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
- Commercial readiness activities underway to support potential
launch in 2022
- Phase 1/2 clinical trial of GDA-201 in patients with follicular
and diffuse large B-cell lymphomas expected to start by the end of
the year
- Four new development programs announced leveraging
next-generation, NAM-enabled, genetically-modified NK cells in
solid tumor and hematological cancers
- 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 cancer and other serious diseases,
today reported financial results for the quarter ended June 30,
2021. Net loss for the second quarter of 2021 was $21.3 million,
compared to a net loss of $15.1 million for the same period in
2020. As of June 30, 2021, Gamida Cell had total cash and cash
equivalents of $150.2 million.
During the quarter, the company continued to execute on plans to
submit a Biologic License Application (BLA) for omidubicel, a
potentially life-saving treatment for patients with blood cancers
in need of stem cell transplant. This submission is expected to
occur by the end of the year, subject to a pre-BLA meeting with the
U.S. Food and Drug Administration (FDA) planned for the fourth
quarter. In addition, Gamida prepared to begin a Phase 1/2 trial of
GDA-201 in non-Hodgkin lymphoma (NHL), expected to occur by the end
of 2021. Also, the company expanded its NAM-enabled natural killer
(NK) cell pipeline targeting solid-tumor and hematological cancers,
including genetically modified variants of proprietary NK therapies
using both CRISPR/Cas9 and CAR methodologies.
“Our progress this quarter represents a major step forward for
Gamida Cell and our mission to bring cancer patients potentially
curative cell therapies,” said Julian Adams, Ph.D., chief executive
officer of Gamida Cell. “We are delivering against key process
development, quality and manufacturing milestones in preparation
for a BLA submission for omidubicel while also advancing our
go-to-market strategy for our planned commercial launch. In
parallel, we bolstered our NAM-enabled NK pipeline both by readying
to advance GDA-201 into the clinic based on its encouraging
clinical data in patients with hematological cancers and by
expanding our NK cell pipeline to address solid and liquid
tumors.”
Q2 and Recent Developments
Omidubicel: Advanced Cell Therapy
- Continued advancement toward planned BLA submission for
omidubicel to the FDA in the fourth quarter of this year. The
company’s activities included CMC qualification requirements at
both the Gamida–owned facility in Israel and at Lonza, a contract
manufacturing organization that will be supplying commercial
material upon FDA approval. Advancements were made in analytical
methods validation, analytical comparability and clinical
manufacturing for Expanded Access Program patients, which are also
planned to be used for clinical comparability.
- Advanced launch planning activities by expanding Gamida’s
commercial, operational and medical affairs teams. Conducted
further market research and health economic and outcomes research
(HEOR) to support planned market entry and market access
activities. Readied Gamida Cell Assist, supply chain and logistics
programs to facilitate positive patient and transplant center
experiences at time of launch.
- Announced that results of the international, multi-center,
randomized Phase 3 clinical study of omidubicel were published in
Blood, the official journal of the American Society of Hematology.
This pivotal trial compared the safety and efficacy of omidubicel
to standard umbilical cord blood transplant in patients with
high-risk hematologic malignancies undergoing a bone marrow
transplant. The results demonstrate that transplantation with
omidubicel leads to faster neutrophil and platelet recovery, and
results in fewer bacterial, viral and fungal infections and less
time in the hospital, compared to a standard umbilical cord blood
graft.
GDA-201: NAM-Enabled NK Immunotherapy
- Prepared for filing of an Investigational New Drug (IND)
application with the FDA.
- Finalized clinical study protocol and statistical plan for a
planned Phase 1/2 clinical trial of allogeneic, cryopreserved
GDA-201 in patients with follicular and diffuse large B-cell
lymphoma.
- Conducted study start-up activities, including contract
research organization (CRO) and clinical site selections.
NAM-Enabled NK Cell Pipeline Expansion
- Advanced four new development programs that involve
modifications intended to direct NK cells against specific tumor
markers to improve their cancer killing capabilities against both
hematological and solid tumors. Newly designated product candidates
include:
- GDA-301: Knockout of CISH (cytokine inducible SH2 containing
protein) in NK cells using CRISPR/Cas9 in combination with a
membrane-bound IL-15/IL-15Ra. Designed to improve tumor killing by
promoting activation and inhibiting negative feedback signals.
Potential applications exist across a range of solid tumors and
lymphoma.
- GDA-401: Undisclosed target genetically engineered to enhance
NK cell survival in the solid tumor microenvironment for potential
application across a broad range of solid tumors.
- GDA-501: CAR-engineered NK cells to target HER2+ solid tumors
with the potential to enhance homing and activation against cancers
with HER2 overexpression, including breast, ovarian, lung, bladder,
gastric and others.
- GDA-601: Knockout of CD38 on NK cells to avoid fratricide by
CD38 targeted antibodies in combination treatment of multiple
myeloma, combined with a CD38 CAR designed to enhance killing of
cancerous cells.
- Advanced additional NAM-enabled research programs targeting
immunosuppressive pathways using both CRISPR/Cas9 and CAR, with
potential to treat solid tumor and blood cancers.
Corporate
- Hired Vladimir Melnikov as Senior Vice President, Global
Operations and Manufacturing. Vladimir has over 25 years of
experience in the biopharmaceutical industry. He previously served
as general manager at Omrix Biopharmaceuticals and biologic
technical operations lead at Ethicon Biosurgery, both part of a
Johnson & Johnson Company. In those roles he supervised three
Israeli biotech manufacturing sites and technology transfer to
external partners. Vladimir will have responsibility for the
company’s Israeli manufacturing site and manufacturing partnership
with Lonza.
- Hired Josh Patterson as General Counsel, effective August 30,
2021. Josh has over 20 years of experience as in-house legal
counsel for biopharmaceutical companies. Josh will be joining
Gamida Cell from Akcea Therapeutics, a wholly owned subsidiary of
Ionis Pharmaceuticals, where he is currently General Counsel. Josh
will be responsible for building, leading and managing the legal
function for Gamida Cell.
Second Quarter 2021 Financial Results
- Research and development expenses in the second quarter of 2021
were $13.5 million, compared to $9.3 million for the same period in
2020. The increase was mainly due to omidubicel commercial
manufacturing readiness activities, and the advancement of the
GDA-201 program, including broadening scientific capabilities and
talent.
- Commercial expenses in the second quarter of 2021 were $5.2
million, compared to $1.0 million for the second quarter of 2020.
The increase was mainly attributed to progress with omidubicel
commercial readiness activities.
- General and administrative expenses were $3.8 million for the
second quarter of 2021, compared to $2.5 million for the same
period in 2020. The increase was mainly due to the hiring of key
management positions to support business growth.
- Finance income, net, was $1.2 million for the second quarter of
2021, compared to $2.2 million for the second quarter of 2020. The
increase was primarily due to non-cash income, resulting from
revaluation of warrants offset by interest expenses that resulted
from the $75 million convertible note financing in February
2021.
Net loss for the second quarter of 2021 was $21.3 million,
compared to a net loss of $15.1 million for the same period in
2020.
2021 Financial Guidance
Gamida Cell reiterates its prior financial guidance and expects
cash used for ongoing operating activities in 2021 to range from
$110 million to $120 million. The company believes that its current
cash and cash equivalents will support the 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 Developments and Milestones
Gamida Cell plans to achieve the following key milestones during
the second half of 2021:
Omidubicel
- Pre-BLA meeting with FDA in the fourth quarter of 2021
- BLA submission to the FDA in the fourth quarter of 2021
- Commercial readiness activities ongoing for potential launch
following approval
GDA-201
- IND submission to FDA in third quarter 2021
- Initiation of a company-sponsored Phase 1/2 clinical study in
NHL before year-end 2021
NK cell pipeline expansion
- Advance pipeline of NAM-enabled, genetically-modified NK cells
in solid tumor and blood cancers
Conference Call Information
Gamida Cell will host a conference call today, August 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
potentially life-saving1 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.2,3 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.gamida-cell.com.
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 nicotinamide
(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, the lead candidate in the NAM-enabled NK cell pipeline,
has demonstrated promising initial clinical trial results, as
reported at the 2020 American Society of Hematology (ASH) Annual
Meeting & Exposition. 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 has been in development through an
investigator-sponsored study in patients with refractory NHL and
multiple myeloma. 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 pioneering a diverse immunotherapy pipeline of
potentially curative cell therapies for patients with solid tumor
and blood cancers and other serious blood diseases. We apply a
proprietary expansion platform leveraging the properties of NAM to
allogeneic cell sources — including umbilical cord blood-derived
cells and NK cells — to create therapies with potential to redefine
standards of care. These include omidubicel, an investigational
product with potential as a life-saving alternative for patients in
need of bone marrow transplant, and a line of modified and
unmodified NAM-enabled NK cells targeted at solid tumor and
hematological malignancies. For additional information, please
visit www.gamida-cell.com or follow Gamida Cell on LinkedIn,
Twitter, Facebook, Instagram, or YouTube 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 (including the
submission of the BLA for omidubicel to the FDA), commercialization
planning efforts, the potentially life-saving or curative
therapeutic and commercial potential of omidubicel, and Gamida
Cell’s expectations regarding its projected cash to be used for
operating activities and cash runway. Any statement describing
Gamida Cell’s goals, expectations, financial or other projections,
intentions or beliefs is a forward-looking statement and should be
considered an at-risk statement. Such statements are subject to a
number of risks, uncertainties and assumptions, including those
related to the impact that the COVID-19 pandemic could have on our
business, and including the scope, progress and expansion of Gamida
Cell’s clinical trials and ramifications for the cost thereof;
clinical, scientific, regulatory and technical developments; and
those inherent in the process of developing and commercializing
product candidates that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around
such product candidates. 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. Although Gamida Cell’s
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Gamida Cell. As a result, you are cautioned not
to rely on these forward-looking statements.
___________________ 1 Gragert et al. HLA Match Likelihoods for
Hematopoietic Stem-Cell Grafts in the U.S. Registry. N Engl J Med
2014;371:339-48. Bejanyan et al. Myeloablative Conditioning for
Allogeneic Transplantation Results in Superior Disease-Free
Survival for Acute Myelogenous Leukemia and Myelodysplastic
Syndromes with Low/Intermediate but not High Disease Risk Index: A
Center for International Blood and Marrow Transplant Research
Study. Biol Blood Marrow Transplant 00 (2020) 1-9. 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 Horwitz M.E., et al. Blood. 2021 Jun 22;blood.2021011719. doi:
10.1182/blood.2021011719. Online ahead of print. 4 Bachanova et al.
ASH 2020 abstract
INTERIM CONSOLIDATED
STATEMENTS OF FINANCIAL POSITION
U.S. dollars in
thousands
June 30,
December 31,
2021
2020
2020
Unaudited
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
100,490
$
88,638
$
127,170
Marketable securities
49,702
-
-
Prepaid expenses and other current
assets
3,730
2,241
2,815
Total current
assets
153,922
90,879
129,985
NON-CURRENT ASSETS:
Property, plant and equipment, net
25,607
14,204
18,238
Right-of-use assets
5,404
7,490
6,474
Other assets
1,787
642
786
Total
non-current assets
32,798
22,336
25,498
Total
assets
$
186,720
$
113,215
$
155,483
LIABILITIES AND EQUITY
CURRENT LIABILITIES:
Trade payables
$
5,435
$
2,738
$
6,329
Employees and payroll accruals
4,796
3,187
4,705
Current maturities of lease
liabilities
1,937
2,145
2,532
Accrued interest
1,618
-
-
Accrued expenses and other payables
8,839
5,509
7,988
Total current
liabilities
22,625
13,579
21,554
NON-CURRENT LIABILITIES:
Liabilities presented at fair value
6,233
4,551
12,043
Employee benefit liabilities, net
768
773
768
Other long-term liabilities
4,839
5,946
5,378
Liability to Israel Innovation
Authority
19,146
13,816
17,003
Convertible senior notes, net
69,025
-
-
Total
non-current liabilities
100,011
25,086
35,192
SHAREHOLDERS' EQUITY:
Share capital
167
137
166
Share premium
379,981
304,175
375,280
Capital reserve
(441
)
(541
)
(441
)
Reserve from financial assets measured at
FVOCI
(25
)
-
-
Accumulated deficit
(315,598
)
(229,221
)
(276,268
)
Total shareholders' equity
64,084
74,550
98,737
Total liabilities and shareholders' equity
$
186,720
$
113,215
$
155,483
INTERIM CONSOLIDATED
STATEMENTS OF COMPREHENSIVE LOSS
U.S. dollars in thousands
(except share and per share data)
Six months ended
June 30,
Three months ended
June 30,
Year ended
December 31,
2021
2020
2021
2020
2020
Unaudited
Unaudited
Operating expenses:
Research and development, net
$
24,817
$
17,198
$
13,451
$
9,319
$
41,385
Commercial activities
9,660
2,497
5,230
1,029
8,748
General and administrative
7,230
5,490
3,817
2,496
12,167
Operating loss
41,707
25,185
22,498
12,844
62,300
Finance expense
4,150
1,366
2,594
2,320
10,640
Finance income
(6,080
)
(894
)
(3,801
)
(109
)
(236
)
Loss before tax benefit
39,777
25,657
21,291
15,055
72,704
Tax benefit
(447
)
-
-
-
-
Net loss
39,330
25,657
21,291
15,055
72,704
Net loss
per share:
Basic loss per share
$
0.66
$
0.69
$
0.36
$
0.37
$
1.66
Diluted loss per share
$
0.76
$
0.69
$
0.42
$
0.37
$
1.66
Weighted average share count
59,725,076
37,141,582
59,336,633
49,589,719
43,725,584
INTERIM CONSOLIDATED STATEMENTS
OF CASH FLOWS
U.S. dollars in thousands
Six months ended
June 30,
Three months ended
June 30,
Year ended
December 31,
2021
2020
2021
2020
2020
Unaudited
Unaudited
Cash flows from
operating activities:
Net loss
$
(39,330
)
$
(25,657
)
$
(21,291
)
$
(15,055
)
$
(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
1,277
1,106
642
556
2,397
Financial (income) expense,
net
1,007
(260
)
522
(128
)
483
Share-based compensation
2,463
1,221
1,449
322
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 derivatives
(5,810
)
(670
)
(3,525
)
1,778
6,822
Revaluation of liability to
IIA
1,858
1,315
832
593
4,302
Deferred income taxes
(447
)
-
-
-
348
2,716
(80
)
3,121
16,966
Changes in asset and liability items:
Decrease (increase) in prepaid
expenses, other current assets, and other assets
68
(1,065
)
591
(607
)
(1,626
)
Increase (decrease) in trade
payables
(893
)
1,574
(1,768
)
(360
)
5,083
Increase (decrease) in accrued
expenses and other payables
(201
)
(624
)
2,523
2,472
3,454
(1,026
)
(115
)
1,346
1,505
6,911
Cash received during
the period for:
Interest received
268
357
268
9
361
Interest paid
(85
)
(80
)
(34
)
(33
)
(161
)
Net cash used in operating
activities
(39,825
)
(22,779
)
(19,791
)
(10,453
)
(48,627
)
Cash flows from
investing activities:
Purchase of property, plant and
equipment
(5,390
)
(7,109
)
(2,584
)
(4,990
)
(11,804
)
Investment in long term
deposit
(1,000
)
-
(1,000
)
-
-
Purchase of marketable
securities
(68,151
)
-
(68,151
)
-
-
Proceeds from maturity of
marketable securities
17,824
-
17,824
-
(158
)
Proceeds from sale of marketable
securities
-
13,551
-
-
13,551
Net cash provided by (used in)
investing activities
$
(56,717
)
$
6,442
$
(53,911
)
$
(4,990
)
$
1,589
INTERIM CONSOLIDATED
STATEMENTS OF CASH FLOWS
U.S. dollars in
thousands
Six months ended
June 30
Three months ended
June 30
Year ended
December 31,
2021
2020
2021
2020
2020
Unaudited
Cash flows from
financing activities:
Proceeds from secondary offering,
net
-
-
-
-
133,316
Receipt of grants from the
IIA
52
200
-
147
399
Proceeds from secondary offering,
net
-
63,860
-
63,860
-
Proceeds from issuance of
convertible senior notes, net of issuance costs
70,777
-
(235
)
-
Payment of lease liabilities
(1,129
)
(1,122
)
(465
)
(335
)
(1,985
)
Exercise of options
556
147
54
141
650
Payment of issuance costs related
to public offering
(468
)
-
-
-
-
Net cash (used in) provided by
financing activities
69,788
63,085
(646
)
63,813
132,380
Exchange differences on balances
of cash and cash equivalents
74
52
40
(24
)
(10
)
Increase (decrease) in cash and
cash equivalents
(26,680
)
46,800
(74,308
)
48,346
85,332
Cash and cash equivalents at
beginning of period
127,170
41,838
174,798
40,292
41,838
Cash and cash equivalents at end
of period
$
100,490
$
88,638
$
100,490
$
88,638
$
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
$
656
$
-
$
607
$
-
$
103
Issuance expenses on credit
$
-
$
-
$
-
$
-
$
468
Purchase of property, plant and
equipment on credit
$
1,563
$
960
$
1,563
$
960
$
415
Borrowing costs
capitalization
$
574
$
-
$
574
$
-
$
-
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210811005275/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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