– Commercial launch preparations underway ahead
of January 30, 2023 PDUFA target action date –
– New data presented across multiple medical
meetings to support the potential of omidubicel as an advanced cell
therapy, including long term follow-up data from the Phase 3 study,
a real-world analysis of donor sources, and health-related quality
of life scores and the potential for omidubicel to increase access
to transplant for patients –
– Appointed Abigail Jenkins as President and
CEO as part of planned succession –
– Strengthened financial position with sale of
ordinary shares for gross proceeds of $20 million and signed a
commitment letter for $25 million senior secured convertible term
loan, both in September 2022 –
– Company to host conference call at 8:00 a.m.
ET today –
Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the
development of NAM-enabled cell therapies for patients with
hematologic and solid cancers and other serious diseases, today
provided a business update and reported financial results for the
quarter ended September 30, 2022. Net loss for the third quarter of
2022 was $17.8 million, compared to a net loss of $23.2 million in
the third quarter of 2021. As of September 30, 2022, Gamida Cell
had total cash, cash equivalents and investments of $61.3
million.
Recently, Gamida Cell:
- Advanced commercial readiness activities to
support the potential launch of omidubicel in preparation for the
target action date of January 30, 2023 under Prescription Drug User
Fee Act (PDUFA).
- Presented new data supporting the potential of
omidubicel for the treatment of patients with blood cancers in need
of allogeneic hematopoietic stem cell transplant at the Tenth
Annual Meeting of the Society of Hematologic Oncology (SOHO) and
the 2022 Cord Blood Connect Meeting (CBC). Data presented included
new three-year follow-up data from the Phase 3 study that
demonstrated that patients treated with omidubicel had an overall
and disease-free survival rate of 63% and 56% at three years,
respectively. Additional data presentations demonstrated that
treatment with omidubicel led to higher health-related quality of
life scores, and that, if approved, omidubicel is projected to
meaningfully improve patient outcomes among racial and ethnic
minorities by extending access and reducing time to transplant for
patients who may not be able to currently find a donor source.
- Appointed Abigail “Abbey” Jenkins as President
and CEO as part of a planned succession.
- Executed an underwritten public offering in
September 2022 that raised $20 million before deducting
underwriting discounts, commissions and offering expenses. The
company also announced a commitment letter for a $25 million senior
secured convertible loan in September 2022 with Highbridge Capital
Management, LLC.
- Continued pre-clinical development of the
company’s proprietary NAM-enabled NK cell pipeline, including
genetically modified product candidates GDA-301, GDA-401, GDA-501
and GDA-601, which aim to treat solid-tumor and hematological
cancers. These cell therapy candidates utilize CAR, membrane bound-
and CRISPR-mediated technologies to increase the NK cell targeting,
potency and persistence against hematologic malignancies and solid
tumors. At the Society for Immunotherapy of Cancer’s 37th Annual
Meeting (SITC), Gamida Cell announced encouraging preclinical data
on GDA-501, supporting its continued pre-clinical development.
“Gamida Cell is entering a pivotal time of growth as we prepare to
transition from being a clinical stage to commercial stage company
upon the potential approval of omidubicel. The January 30, 2023
PDUFA date for omidubicel is rapidly approaching and, if approved,
omidubicel will offer a transformative new option to patients in
need of an allogeneic stem cell transplant,” said Abbey Jenkins,
president and CEO of Gamida Cell. “The Gamida Cell team has
utilized robust market insights to understand the important role
that omidubicel can play for transplanters, if approved, in terms
of both improving patients outcomes and increasing access for
patients who cannot currently find a donor source. We have now
accelerated launch preparations across our commercial, operations
and medical affairs teams to ensure we can begin making omidubicel
available upon potential approval in January. Beyond omidubicel, we
are enrolling patients in our company-sponsored Phase 1/2 study of
GDA-201 and are continuing to develop our genetically modified NK
cell immunotherapy programs that leverage CAR- and CRISPR-mediated
technologies. We are excited about the progress across our pipeline
as we advance on our mission to deliver potentially curative
therapies to patients with cancer and other serious diseases.”
Third Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy Candidate
- Data presentations at the 64th American Society of
Hematology (ASH) Annual Meeting: In November, Gamida Cell
announced it will present new real-world analysis data comparing
the safety and efficacy of omidubicel to alloHCT donor sources used
in clinical practice from the Center for International Blood and
Marrow Transplant Research (CIBMTR) database at the December ASH
annual meeting. The analysis demonstrated that omidubicel was
associated with more rapid neutrophil recovery (median: 10 days)
than all other donor sources (median 15-20 days; p-value <0.001)
and evaluated other outcomes comparing omidubicel Phase 3 results
and those from the CIBMTR database. Overall survival (OS) was
comparable across donor sources.
- Presented new long-term data from Phase 3 trial at SOHO:
In September, at SOHO, Gamida Cell presented new long term
follow-up data and health-related quality of life scores of
patients treated with omidubicel. In an analysis of 105 patients
transplanted with omidubicel between 2006 and 2020 (median
follow-up of 22 months), the data demonstrated an overall and
disease-free survival rate of 63% (95% CI, 53%-73%) and 56% (95%
CI, 47%-67%) at three years, respectively. A second presentation
featured an analysis of 108 patients that completed validated
health-related quality of life (HRQL) surveys on screening and days
42, 100, 180, and 365 post-transplant. Measures of physical and
functional well-being and other HRQL scores were more favorable
with omidubicel and suggest clinically meaningful and sustained
improvements in physical, functional and overall well-being
compared to umbilical cord blood (UCB) transplantation.
- Presented data demonstrating the impact of transplantation
with omidubicel for patients with hematologic malignancies at
CBC: In September, at CBC, Gamida Cell presented data
supporting the potential of omidubicel for the treatment of
patients with blood cancers in need of an allogeneic hematopoietic
stem cell transplant. The data suggest during the first-year
post-transplant, patients receiving omidubicel had meaningfully
greater preservation or improvement of important HRQL domains
compared to UCB recipients. A second poster featured an analysis of
the projected impact of allogeneic hematopoietic cell transplant on
clinical outcomes and potential access for patients who currently
cannot find a donor source, with the greatest improvements among
the racial and ethnic groups underserved by the current standard of
care. In a third poster, results of a translational sub-study from
the Phase 3 trial showed that patients transplanted with omidubicel
had more rapid and robust immune reconstitution than controls,
including higher numbers of Recent Thymic Emigrants in peripheral
blood at one year post transplant compared to transplantation with
UCB, which suggest faster thymopoiesis and provide a mechanistic
rational for the lower infection rates in these patients.
- Continued launch preparations in preparation for January 30,
2023 PDUFA date: The company’s Biologics License Application
(BLA) for omidubicel is under review with the U.S. Food and Drug
Administration (FDA) with a target action PDUFA date of January 30,
2023. Gamida Cell continues to advance readiness activities
throughout the organization to support the potential launch of
omidubicel.
GDA-201: NAM-Enabled NK Cell Therapy
- Continued advancement of Phase 1/2 study of cryopreserved
formulation of GDA-201: Gamida Cell continues to advance its
company-sponsored Phase 1/2 study evaluating a cryopreserved
formulation of GDA-201 for the treatment of follicular and diffuse
B-cell lymphomas.
NAM-Enabled NK Cell Pipeline Development
- Presented preclinical data on GDA-501: At the SITC
Annual Meeting, Gamida Cell announced new preclinical data on
GDA-501, a genetically modified HER2-CAR NAM-NK cell therapy
candidate, that provide support for its continued preclinical
development. GDA-501 displayed significantly enhanced in vitro
cytotoxicity when cultured with HER2+ targeted cancer cells, as
well as increased potency based on elevated levels of
proinflammatory cytokines and biomarkers compared with control
cells. Importantly, increased cytotoxicity and potency were
persistent. These preclinical data demonstrate potent antitumor
activity.
- Continued the development of NAM-enabled genetically
modified NK pipeline: Gamida Cell continues to progress its
NAM-enabled genetically modified NK pipeline, which utilizes CAR,
membrane bound- and CRISPR-mediated technologies to increase
targeting, potency and persistence against hematologic malignancies
and solid tumors. In order to both prioritize resources to the
commercialization of omidubicel and advancement of GDA-201, as well
as enable development activities to progress further, the company
will hold on selecting an IND candidate and will continue to
conduct in vitro and in vivo preclinical proof-of-concept studies
for these genetically modified NK therapeutic targets.
Corporate Updates
- Appointed Abigail Jenkins as President and CEO: As part
of a CEO succession plan, Julian Adams retired as CEO and Abigail
“Abbey” Jenkins was appointed as President and CEO. Ms. Jenkins has
also been appointed to Gamida Cell’s Board of Directors. Ms.
Jenkins brings over 25 years of leadership experience in the
biopharmaceutical industry developing life-enhancing therapies from
research to commercialization. Julian Adams will remain on the
company’s Board of Directors.
- Strengthened financial position: In September 2022,
Gamida Cell executed an underwritten public offering raising
approximately $20 million, before deducting underwriting discounts,
commissions and offering expenses. Also, in September 2022, the
company announced it entered into a commitment letter with
Highbridge Capital Management, LLC for a $25 million senior
secured, convertible term loan. These capital commitments will be
used to fund the commercial readiness activities to support the
potential launch of omidubicel, if approved, and to further the
clinical development of its NK product candidates, including
GDA-201.
Third Quarter 2022 Financial Results
- Research and development expenses were $9.9 million in the
third quarter of 2022, compared to $11.7 million in the same
quarter in 2021. The decrease was attributable mainly to a $1.6
million decrease in clinical activities relating to the conclusion
of the Phase 3 clinical trial and a decrease of $0.2 million in
GDA-201 clinical program.
- Commercial expenses were $2.8 million in the third quarter of
2022, compared to $5.8 million in the third quarter of 2021. The
decrease was attributable mainly to an $2.5 million decrease in
launch readiness activities, and $0.6 million decrease in headcount
related expenses.
- General and administrative expenses were $4.4 million in the
third quarter of 2022, compared to $5.0 million in the same period
in 2021. The decrease was mainly due to a $0.3 million decrease in
professional services expenses and $0.3 million decrease in
headcount related expenses.
- Finance expenses, net, were $0.7 million in the third quarters
of 2022 and 2021, with no material changes.
- Net loss was $17.8 million in the third quarter of 2022,
compared to a net loss of $23.2 million in the third quarter of
2021.
2022 Financial Guidance
Gamida Cell expects that its current total cash position,
together with recent financing, will support the company’s ongoing
operating activities into mid-2023, excluding the cost of
commercializing omidubicel beyond the initial launch. This cash
runaway guidance is based on the company’s current operational
plans and excludes any additional funding and any business
development activities that may be undertaken. Gamida Cell
continues to assess all financing options that support its
corporate strategy.
Expected Milestones in 2023
Omidubicel
- PDUFA target action date of January 30, 2023.
Conference Call Information
Gamida Cell will host a conference call today, November 14,
2022, at 8:00 a.m. ET to discuss these financial results and
company updates. To access the conference call, please register
here and be advised to do so at least 10 minutes prior to joining
the call. 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. A replay of the webcast will be available
approximately two hours after the event, for approximately 30
days.
About NAM Technology Our NAM-enabling technology is
designed to enhance the number and functionality of targeted cells,
enabling us to pursue a curative approach that moves beyond what is
possible with existing therapies. Leveraging the unique properties
of NAM (nicotinamide), we can expand and metabolically modulate
multiple cell types — including stem cells and natural killer cells
— with appropriate growth factors to maintain the cells’ active
phenotype and enhance potency. Additionally, our NAM technology
improves the metabolic fitness of cells, allowing for continued
activity throughout the expansion process.
About Omidubicel Omidubicel is an advanced cell therapy
candidate developed as a potential life-saving allogeneic
hematopoietic stem cell (bone marrow) transplant for patients with
blood cancers. Omidubicel demonstrated a statistically significant
reduction in time to neutrophil engraftment in comparison to
standard umbilical cord blood in an international, multi-center,
randomized Phase 3 study (NCT0273029) in patients with hematologic
malignancies undergoing allogeneic bone marrow transplant. The
Phase 3 study also showed reduced time to platelet engraftment,
reduced infections and fewer days of hospitalization. One-year
post-transplant data showed sustained clinical benefits with
omidubicel as demonstrated by significant reduction in infectious
complications as well as reduced non-relapse mortality and no
significant increase in relapse rates nor increases in
graft-versus-host-disease (GvHD) rates. Omidubicel is the first
stem cell transplant donor source to receive Breakthrough Therapy
Designation from the FDA and has also received Orphan Drug
Designation in the US and EU.
The BLA for omidubicel has been assigned a Prescription Drug
User Fee Act (PDUFA) target action date of January 30, 2023. If
approved, omidubicel will be the first allogeneic advanced stem
cell therapy donor source for patients with blood cancers in need
of a stem cell transplant.
Omidubicel is an investigational stem cell therapy candidate,
and its safety and efficacy have not been established by the FDA or
any other health authority. For more information about omidubicel,
please visit https://www.gamida-cell.com.
About GDA-201 Gamida Cell applied the capabilities of its
nicotinamide (NAM)-enabled cell expansion technology to develop
GDA-201, an innate NK cell immunotherapy candidate for the
potential 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 study data. Preclinical studies have
shown that GDA-201 may address key limitations of NK cells by
increasing the cytotoxicity and in vivo retention and proliferation
in the bone marrow and lymphoid organs. Furthermore, these data
suggest GDA-201 may improve antibody-dependent cellular
cytotoxicity (ADCC) and tumor targeting of NK cells. There are
approximately 40,000 patients with relapsed/refractory lymphoma in
the US and EU, which is the patient population that will be studied
in the currently ongoing GDA-201 Phase 1/2 clinical trial.
For more information about GDA-201, please visit
https://www.gamida-cell.com. For more information on the Phase 1/2
clinical trial of GDA-201, please visit www.clinicaltrials.gov.
GDA-201 is an investigational cell therapy candidate, 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 therapy
candidates 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 therapy candidates 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 or Instagram 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 (including omidubicel and GDA-201), regulatory filings
submitted to the FDA (including the potential timing of the FDA’s
review of the BLA for omidubicel), commercialization planning
efforts and launch activities, the potentially life-saving or
curative therapeutic and commercial potential of Gamida Cell’s
product candidates (including omidubicel and GDA-201), progress on
the preclinical NAM-enabled NK cell pipeline, 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 Quarterly
Report on Form 10-Q, filed with the Securities and Exchange
Commission (SEC) on August 15, 2022, 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.
CONDENSED CONSOLIDATED BALANCE
SHEETS
U.S. dollars in thousands (except share
and per share data)
September 30,
December 31,
2022
2021
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
55,071
$
55,892
Marketable securities
6,224
40,034
Prepaid expenses and other current
assets
1,408
2,688
Total current
assets
62,703
98,614
NON-CURRENT ASSETS:
Restricted deposits
3,556
3,961
Property, plant and equipment, net
39,940
35,180
Operating lease right-of-use assets
5,459
7,236
Severance pay fund
1,595
2,148
Other long-term assets
1,059
1,647
Total
non-current assets
51,609
50,172
Total
assets
$
114,312
$
148,786
September 30,
December 31,
2022
2021
LIABILITIES AND SHARHOLDERS' EQUITY
CURRENT LIABILITIES:
Trade payables
$
1,916
$
8,272
Employees and payroll accruals
5,689
4,957
Operating lease liabilities
1,880
2,699
Accrued interest of convertible senior
notes
551
1,640
Accrued expenses and other current
liabilities
12,677
7,865
Total current
liabilities
22,713
25,433
NON-CURRENT LIABILITIES:
Convertible senior notes, net
71,999
71,417
Accrued severance pay
1,865
2,396
Long-term operating lease liabilities
4,174
5,603
Total
non-current liabilities
78,038
79,416
CONTINGENT LIABILITIES AND COMMITMENTS
SHAREHOLDERS' EQUITY:
Share capital
210
169
Additional paid-in capital
407,387
381,225
Accumulated deficit
(394,036
)
(337,457
)
Total shareholders' equity
13,561
43,937
Total liabilities and
shareholders' equity
$
114,312
$
148,786
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
U.S. dollars in thousands (except
share and per share data)
Three months ended
September 30,
Nine months ended
September 30,
2022
2021
2022
2021
(unaudited)
(unaudited)
Research and development
expenses, net
$
9,864
$
11,725
$
31,732
$
36,435
Commercial expenses
2,760
5,755
9,832
14,974
General and administrative
expenses
4,437
5,001
12,866
12,388
Total operating loss
17,061
22,481
54,430
63,797
Financial expenses, net
741
692
2,149
2,119
Loss
$
17,802
$
23,173
$
56,579
$
65,916
Net loss per share attributable
to ordinary shareholders, basic and diluted
0.29
0.39
0.95
1.11
Weighted average number of shares
used in computing net loss per share attributable to ordinary
shareholders, basic and diluted
60,440,765
59,281,243
59,821,655
59,219,757
CONDENSED CONSOLIDATED STATEMENTS OF
CASH FLOWS
U.S. dollars in thousands (except share
and per share data)
Nine months ended
September 30,
2022
2021
Cash flows
from operating activities:
Loss
$
(56,579
)
$
(65,916
)
Adjustments to reconcile loss to
net cash used in operating activities:
Depreciation of property, plant
and equipment
391
317
Financing expense (income),
net
(2,461
)
89
Share-based compensation
3,829
3,122
Amortization of debt discount and
issuance costs
582
453
Operating lease right-of-use
assets
1,922
1,542
Operating lease liabilities
(2,395
)
(1,764
)
Accrued severance pay, net
23
-
Decrease in prepaid expenses and
other assets
1,719
558
Increase (decrease) in trade
payables
(6,355
)
1,533
Increase (decrease) in accrued
expenses and current liabilities
5,079
(1,361
)
Net cash used in operating
activities
(54,245
)
(61,427
)
Cash flows
from investing activities:
Purchase of property, plant and
equipment
(2,865
)
(9,577
)
Purchase of marketable
securities
(4,557
)
(97,808
)
Proceeds from maturity of
marketable securities
37,972
56,717
Proceeds (investments) from
restricted deposits
500
(5,803
)
Net cash provided by (used in)
investing activities
$
31,050
$
(56,471
)
Cash flows
from financing activities:
Proceeds from exercise of options
$
76
$
566
Proceeds from share issuance, net
22,298
-
Proceeds from issuance of
convertible senior notes, net
-
70,777
Net cash provided by financing
activities
22,374
71,343
Decrease in cash and cash equivalents
(821
)
(46,555
)
Cash and cash equivalents at beginning of
period
55,892
127,170
Cash and cash equivalents at end of
period
$
55,071
$
80,615
Significant non-cash
transactions:
Purchase of property, plant and equipment
on credit
281
1,561
Supplemental
disclosures of cash flow information:
Cash paid for interest
$
(4,406
)
$
(2,191
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221114005403/en/
For investors: Courtney Turiano Stern Investor Relations,
Inc. Courtney.Turiano@sternir.com 1-212-362-1200
For media: Heather DiVecchia Director, Investor Relations
and Corporate Communications Heather@gamida-cell.com
1-617-892-9083
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