- Received FDA acceptance of BLA for omidubicel
with Priority Review; PDUFA target action date set for January 30,
2023 -
- Dosed first patient in company-sponsored
Phase 1/2 study of cryopreserved formulation of GDA-201 for the
treatment of follicular and diffuse large B-cell lymphomas -
- Finished second quarter of 2022 with $55.1
million in cash;
sufficient funding for the company’s operations
into mid-2023, excluding the cost of commercializing omidubicel
-
- 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 June
30, 2022. Net loss for the second quarter of 2022 was $18.6
million, compared to a net loss of $23.6 million in the second
quarter of 2021. As of June 30, 2022, Gamida Cell had total cash,
cash equivalents and investments of $55.1 million.
Recently, Gamida Cell:
- Received acceptance for filing from the U.S. Food and Drug
Administration (FDA) with priority review for its Biologics License
Application (BLA) for omidubicel. The BLA 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.
- Dosed the first patient in a company-sponsored Phase 1/2 study
evaluating a cryopreserved formulation of GDA-201, a readily
available cell therapy candidate for the treatment of follicular
and diffuse large B-cell lymphomas.
- Continued 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.
Promising new pre-clinical data on GDA-301 and GDA-601 were
presented at the International Society for Cell & Gene Therapy
Meeting. The data demonstrated that both NAM-enabled cell therapy
candidates represented a novel potent and cytotoxic approach in
fighting cancer.
- Advanced strategic evaluation for omidubicel commercialization,
including assessing whether to commercialize omidubicel ourselves
or to pursue strategic alternatives to commercialize omidubicel,
upon receipt of regulatory approval. The company currently has
sufficient cash to fund the company’s operations into mid-2023,
excluding the cost of commercializing omidubicel.
“2022 is a potentially transformative year for Gamida Cell as we
continue to execute against our clinical and regulatory milestones.
We were excited that the FDA accepted our BLA submission with
priority review, and 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. We believe
that omidubicel has the potential to change the outlook for
patients suffering from blood cancers through improved outcomes,
quality of life and increased access for patients who are currently
eligible for transplant, but cannot find a match,” said Julian
Adams, Ph.D., chief executive officer of Gamida Cell. “In addition,
the development of our NAM-enabled NK cell therapy candidate,
GDA-201, creates an opportunity to potentially bring a new
treatment option to tens of thousands of patients with
relapsed/refractory lymphoma worldwide. We continue to execute our
mission of advancing our broad pipeline of NAM-enabled cell
therapies with a curative approach for patients with solid tumors
and blood cancers and other serious blood diseases.”
Second Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
- BLA accepted by FDA with Priority Review: In August
2022, the FDA accepted for filing Gamida Cell’s BLA for omidubicel
for the treatment of patients with blood cancers in need of an
allogeneic hematopoietic stem cell transplant. The FDA granted
Priority Review for the BLA and has set a PDUFA target action date
of January 30, 2023. In parallel, Gamida Cell is preparing for the
commercialization of omidubicel in the U.S.
GDA-201: NAM-Enabled NK Cell Therapy
- Dosed the first patient in Phase 1/2 study of cryopreserved
formulation of GDA-201: In August 2022, Gamida Cell completed
the dosing of the first patient in a company-sponsored Phase 1/2
study evaluating a cryopreserved formulation of GDA-201 for the
treatment of follicular and diffuse B-cell lymphomas.
- The Phase 1 portion of the study is designed as a dose
escalation phase to evaluate the safety of GDA-201, and will
include patients with follicular lymphoma (FL), diffuse large
B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL),
marginal zone lymphoma or mantle cell lymphoma. The Phase 2
expansion phase is designed to evaluate the safety and efficacy of
GDA-201 in 63 patients comprised of two patient cohorts, FL and
DLBCL. The study will include patients who have relapsed or
refractory lymphoma after at least two prior treatments, which may
include CAR-T or stem cell transplant.
NAM-Enabled NK Cell Pipeline Expansion
- Progressed 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. The
company continues to conduct in vitro and in vivo preclinical
proof-of-concept studies for these genetically modified NK
therapeutic targets which are already showing encouraging results
and plans to select the next NK pipeline product candidate for IND
enabling studies by the end of 2022. These therapeutic targets
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;
- GDA-401: A development candidate with an undisclosed
target;
- GDA-501: Anti HER2 CAR-engineered NK cells to target solid
tumors expressing HER2, based on a single-chain variable fragment
of the widely used humanized monoclonal antibody trastuzumab;
and
- GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti
CD38 CAR. CD38 is an established immunotherapeutic target in
multiple myeloma, but its expression on NK cells and its further
induction during ex vivo NK cell expansion represents a barrier to
the development of an anti CD38 CAR-NK cell therapy. Gamida Cell is
advancing this program in collaboration with the Dana-Farber Cancer
Institute to study the in vitro cytotoxicity of GDA-601 in fresh
tumor tissue samples from multiple myeloma patients.
Corporate Updates
- Appointed Ivan M. Borrello, M.D. to Board of Directors:
Dr. Borrello is an Associate Professor of Oncology at the Sydney
Kimmel Comprehensive Cancer Center at Johns Hopkins and a renowned
physician and author who has made major contributions to better the
understanding of immunotherapies and the treatment of hematologic
malignancies as well as bone marrow transplant. The Company also
announced the resignation of Ofer Gonen from its Board of
Directors.
Second Quarter 2022 Financial Results
- Research and development expenses were $10.6 million in the
second quarter of 2022, compared to $13.4 million in the same
quarter in 2021. The decrease was attributable mainly to a $2.4
million decrease in clinical activities relating to the conclusion
of our Phase 3 clinical trial and a decrease of $0.4 million in the
GDA-201 clinical program.
- Commercial expenses were $3.2 million in the second quarter of
2022, compared to $5.0 million in the second quarter of 2021. The
decrease was attributable mainly to reducing near-term commercial
readiness expenses, as we continued to assess strategic approaches
for the commercialization of omidubicel.
- General and administrative expenses were $4.3 million in the
second quarter of 2022, compared to $3.9 million in the same period
in 2021. The increase was mainly due to a $0.9 million increase in
professional services expenses, offset by a decrease of $0.5
million in headcount related expenses.
- Finance expenses, net, were $0.5 million in the second quarter
of 2022, compared to $1.3 million in the same period in 2021. The
decrease was primarily due to $0.6 million in non-cash expenses and
an increase of $0.2 million in interest income from cash
management.
- Net loss was $18.6 million in the second quarter of 2022,
compared to a net loss of $23.6 million in the second quarter of
2021.
2022 Financial Guidance
Gamida Cell expects that its current cash, cash equivalents and
investments will support the company’s ongoing operating activities
into mid 2023, excluding the cost of commercializing omidubicel. If
we decide to market omidubicel ourselves, we will require
substantial additional funding. 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 2022 and Early 2023
Omidubicel
- PDUFA target action date of January 30, 2023.
NK cell pipeline expansion
- Conduct preclinical proof of concept studies of the
NAM-enabled, genetically modified NK therapeutic targets
- Select pipeline candidate for IND-enabling studies
Conference Call Information
Gamida Cell will host a conference call today, August 15, 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 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.
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 NAM Technology
Our NAM-enabling technology, supported by positive omidubicel
Phase 3 results, 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 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, pre-clinical and clinical trials of
Gamida Cell’s product candidates (including omidubicel and
GDA-201), anticipated regulatory filings (including the timing of
review of the BLA for omidubicel by the FDA), commercialization
planning efforts, the potentially life-saving or curative
therapeutic and commercial potential of Gamida Cell’s product
candidates (including GDA-201 and omidubicel), Gamida Cell’s
expectations for the clinical development milestones set forth
herein, and Gamida Cell’s expectations regarding its projected
cash, cash equivalents and investments to be used for operating
activities. 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,
including the scope, progress and expansion of Gamida Cell’s
clinical trials and ramifications for the cost thereof; clinical,
scientific, regulatory and technical developments; the process of
developing and commercializing product candidates that are safe and
effective for use as human therapeutics; and 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 May 12, 2022, 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)
June 30,
December 31,
2022
2021
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
37,890
$
55,892
Marketable securities
17,172
40,034
Prepaid expenses and other current
assets
2,294
2,688
Total current
assets
57,356
98,614
NON-CURRENT ASSETS:
Restricted deposits
3,591
3,961
Property, plant and equipment, net
37,967
35,180
Operating lease right-of-use assets
6,107
7,236
Severance pay fund
1,579
2,148
Other long-term assets
1,421
1,647
Total
non-current assets
50,665
50,172
Total
assets
$
108,021
$
148,786
LIABILITIES AND EQUITY
CURRENT LIABILITIES:
Trade payables
$
2,738
$
8,272
Employees and payroll accruals
4,978
4,957
Operating lease liabilities
2,517
2,699
Accrued interest of convertible senior
notes
1,652
1,640
Accrued expenses and other current
liabilities
10,412
7,865
Total current
liabilities
22,297
25,433
NON-CURRENT LIABILITIES:
Convertible senior notes, net
71,801
71,417
Accrued severance pay
1,840
2,396
Long-term operating lease liabilities
4,233
5,603
Total
non-current liabilities
77,874
79,416
CONTINGENT LIABILITIES AND COMMITMENTS
SHAREHOLDERS’ EQUITY:
Share capital -
169
169
Additional paid-in capital
383,915
381,225
Accumulated deficit
(376,234
)
(337,457
)
Total
shareholders’ equity
7,850
43,937
Total
liabilities and shareholders’ equity
$
108,021
$
148,786
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
U.S. dollars in thousands (except share
and per share data)
Three months ended June
30,
Six months ended June
30,
2022
2021
2022
2021
Unaudited
Research and development expenses, net
$
10,563
$
13,350
$
21,868
$
24,710
Commercial expenses
3,193
4,988
7,072
9,219
General and administrative expenses
4,290
3,874
8,429
7,387
Total operating loss
18,046
22,212
37,369
41,316
Financial expenses, net
508
1,345
1,408
1,427
Loss
$
18,554
$
23,557
$
38,777
$
42,743
Net loss per share, basic and diluted
0.31
0.40
0.65
0.72
Weighted average number of shares
59,546,273
59,253,315
59,510,918
59,188,504
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
U.S. dollars in thousands (except share
and per share data)
Six months ended June
30,
2022
2021
Cash flows from
operating activities:
Loss
$
(38,777
)
$
(42,743
)
Adjustments to reconcile loss to net cash
used in operating activities:
Depreciation of property, plant and
equipment
224
206
Financing expense (income), net
(273
)
1,694
Share-based compensation
2,530
2,025
Amortization of issuance costs
385
269
Operating lease right-of-use assets
1,226
1,032
Operating lease liabilities
(1,649
)
(1,187
)
Accrued severance pay, net
14
-
Increase in prepaid expenses and other
assets
(19
)
(358
)
Decrease in trade payables
(5,535
)
(884
)
Increase (decrease) in accrued expenses
and current liabilities
2,285
(622
)
Net cash used in operating activities
(39,589
)
(40,568
)
Cash flows from
investing activities:
Purchase of property, plant and
equipment
(1,540
)
(6,118
)
Purchase of marketable securities
(3,708
)
(68,151
)
Proceeds from maturity of marketable
securities
26,175
17,824
Proceeds (investments) from restricted
deposits
500
(1,000
)
Net cash provided by (used in) investing
activities
$
21,427
$
(57,445
)
Cash flows from
financing activities:
Proceeds from exercise of options
$
76
$
556
Proceeds from share issuance, net
84
-
Proceeds from issuance of convertible
senior notes, net
-
70,777
Net cash provided by financing
activities
160
71,333
Decrease in cash and cash equivalents
(18,002
)
(26,680
)
Cash and cash equivalents at beginning of
period
55,892
127,170
Cash and cash equivalents at end of
period
$
37,890
$
100,490
Significant non-cash
transactions:
Purchase of property, plant and equipment
on credit
282
1,563
Supplemental
disclosures of cash flow information:
Cash paid for interest
$
(2,203
)
$
-
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220815005184/en/
For investors: Courtney Turiano Stern Investor Relations, Inc.
Courtney.Turiano@sternir.com 1-212-362-1200
For media: Heather DiVecchia Chief of Staff
Heather@gamida-cell.com 1-617-892-9083
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