Company reports on productive interactions
with FDA and preparations for launch of omidubicel, if approved, in
advance of May 1 PDUFA date
Company outlines strategic restructuring,
plans to prioritize omidubicel launch and reduce operating expenses
to extend cash runway
Company to explore strategic options to
support a broader launch of omidubicel, including potential US and
global partnerships with pharmaceutical companies
Company intends to continue GDA-201 Phase 1
study, discontinue development of natural killer (NK) cell
preclinical candidates and consolidate its operations in Israel in
Kiryat Gat
Company to host conference call at 8:00 am
ET today
Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working
to turn cells into powerful therapeutics, today provided a business
update and reported financial results for the year ended December
31, 2022. Net loss for 2022 was $79.4 million, compared to a net
loss of $89.8 million in 2021. As of December 31, 2022, Gamida Cell
had total cash and cash equivalents of $64.7 million.
The company highlighted positive data, productive regulatory
interactions and progress on commercial readiness activities
supporting its lead product candidate, omidubicel, an advanced cell
therapy candidate for allogeneic stem cell transplant, as it
advances toward its May 1, 2023 target Prescription Drug User Fee
Act (PDUFA) action date. The company also announced a strategic
restructuring of its operations to prioritize launch of omidubicel
to ensure that, if approved, patients who may potentially benefit
will have access to therapy. To reduce expenses, the company will
discontinue development of its preclinical NK cell therapy
candidates while continuing to enroll patients in the GDA-201 Phase
1 clinical trial.
“Our mission is to bring potentially curative therapies to
patients,” said Abbey Jenkins, President and Chief Executive
Officer of Gamida Cell. “We believe we have a clear path to
approval and are preparing for the commercial launch of omidubicel,
if approved. Given the challenging economic environment, to date,
we have not been able to raise adequate funding to support our full
pipeline and enable a more robust launch of omidubicel, if
approved. As a result, we are taking decisive actions to do three
things 1) prioritize resources toward the launch 2) reduce expenses
across the board 3) seek potential commercial or strategic
partnerships to maximize patient access to omidubicel, a
potentially life-saving therapy. Today’s actions are difficult.
Especially since our engineered NK cell therapy candidates, which
are derived from healthy donors, have demonstrated encouraging
pre-clinical data that differentiate them from other NK cell
therapy approaches. The science is promising, but these changes are
economically necessary to ensure omidubicel reaches as many
patients as possible.”
Today Gamida Cell announced it would:
- Implement a strategic restructuring to focus on omidubicel
launch, if approved: The company intends to allocate the vast
majority of its resources to executing a launch of omidubicel, if
approved, although with a more limited investment and slower ramp
than previously planned in order to manage its financial resources.
The company reported productive interactions with the FDA,
including a recently completed Late Cycle Meeting and a previously
reported Pre-Licensing Inspection of the company’s Kiryat Gat,
Israel, manufacturing facility, with no 483 observations received
to date. Recently presented data continue to support the clinical
benefits and safety of omidubicel, which, if approved, may be a
valuable new donor source for patients in need of allogeneic stem
cell transplant. Commercial readiness activities have made progress
as the company prepares to onboard approximately 10-15 of the top
70 transplant centers in the United States in 2023. Omidubicel has
received positive feedback from leading transplant centers,
including ones that did not participate in the company’s clinical
trials. The company has met with U.S. payers, including payers that
cover more than 90% of commercially covered lives, and reported
that payers indicate they anticipate covering a one-time therapy
with curative intent.
- Continue its GDA-201 Phase 1 study: The company will
continue to enroll patients in its GDA-201 Phase 1 dose escalation
study.
- Reduce operating expenses in order to extend its cash
runway: The company will discontinue the development of its
engineered NK cell therapy preclinical pipeline, including GDA-301,
GDA-501 and GDA-601, while maintaining the IP to these candidates.
It will implement a headcount reduction of 17%, with the majority
of impacted headcount tied to the discontinuation of the
pre-clinical NK cell therapy candidates. The company will also
close its operations in Jerusalem and consolidate Israel operations
at its state-of-the-art manufacturing facility in Kiryat Gat. These
changes are expected to extend the company’s cash runway through Q3
2023.
- Explore strategic options: The company intends to seek
potential commercial or strategic partnerships to maximize patient
access to omidubicel, if approved.
Fourth Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
- New data presented at ASH and TCT: The company presented
new data characterizing peripheral blood lymphocytes measured in
correlation with time to neutrophil and platelet engraftment in
omidubicel-transplanted and standard cord blood-transplanted
patients at the 2023 Tandem Meetings, Transplantation &
Cellular Therapy (TCT) Meetings of the American Society for
Transplantation & Cellular Therapy and the Center for
International Blood and Marrow Transplant Research in February.
Seven days post-transplant, omidubicel-transplanted patients showed
a statistically significant correlation between CD3+/CD4+ T cell
counts and time to neutrophil engraftment. Similar correlations
were noted between CD3+/CD8+/CD19+ cell counts and time to platelet
engraftment. Patients transplanted with standard cord blood showed
no such correlations at Day 7 post-transplant, and only began to
show correlations starting at 14 days post-transplant. Data support
past findings that omidubicel stimulates a faster immune response
than standard cord blood, which may be a contributing mechanism
resulting in the lower incidence of serious bacterial, fungal and
viral infections for omidubicel-transplanted patients.
- New publication in press: The company reported a
publication in press in Transplantation and Cellular Therapy, now
available online, reporting on long-term follow-up of patients
transplanted with omidubicel across five clinical trials. The
analysis showed a three-year overall survival of 62.5% and
disease-free survival of 54%. With up to 10 years of follow-up,
omidubicel showed durable hematopoiesis.
- Manufacturing readiness: The company’s state-of-the-art
manufacturing facility in Kiryat Gat, Israel, is ready for
commercial launch if omidubicel is approved and is currently
producing omidubicel for the company’s Extended Access Program
(EAP) and its ongoing omidubicel aplastic anemia study. The
facility, which has completed its Israeli Ministry of Health and
FDA pre-licensure inspections with no 483 observations to date, has
the ability to deliver omidubicel back to the transplant center
within approximately 30 days from the start of manufacturing.
- Commercial readiness: The company continues to advance
efforts throughout the organization to prepare for the launch of
omidubicel, if approved.
GDA-201: Intrinsic NK Cell Therapy
- New data presented at Tandem Meetings: The company
presented a poster at the 2023 Tandem Meetings, Transplantation
& Cellular Therapy (TCT) Meetings of the American Society for
Transplantation & Cellular Therapy and the Center for
International Blood and Marrow Transplant Research reporting new
preclinical data on the cryopreserved formulation of GDA-201, which
showed increased potency and enhanced cytotoxicity. GDA-201 cells
were tested for viability, phenotyping, function and potency.
Previous characterization of GDA-201 showed high levels of CD56,
CD16, CD49a and CD62L expression, low levels of CD57, and low
levels of immune checkpoints such as LAG3 and CD200R. The new
analyses showed that cryopreserved GDA-201 exhibited high viability
(>90%) and high purity up to 12 months post-manufacturing and
preserved the ability to proliferate post-thaw. GDA-201 maintained
high levels of expression of CD16, which mediates
antibody-dependent cellular toxicity, and CD62L, which is a homing
and retention marker. GDA-201 also demonstrated high potency, based
on the intracellular secretion of TNF-alpha & IFN-gamma and
extracellular degranulation marker CD107a. In addition, external
investigator Veronika Bachanova, M.D., Ph.D., Professor at the
University of Minnesota Medical School, gave an oral presentation
highlighting novel observations of “on treatment” tumor biopsies
from eight patients treated with GDA-201 in a Phase 1 study. The
microscopic spatial analysis demonstrated that while GDA-201 cells
were virtually undetectable in tumors after 14 days, T cells were
observed in 50-95% of tumor site cellularity. Most biopsies
obtained as early as three to seven days post-infusion showed
strong indications of widespread tumor death. These observations
suggest that GDA-201 infusions trigger profound immune
microenvironment changes, supporting the influx of host T cells
early post-GDA-201 infusion. These findings further suggest the
engagement of the adaptive immune system and effective tumor
elimination.
Corporate Developments
- On March 20, the company announced that Shawn Cline Tomasello
was elected Chairwoman of the Board of Directors, succeeding
Chairman Robert I. Blum, who resigned. Ms. Tomasello joined the
Gamida Cell Board of Directors in June 2019. She has extensive
experience in leading successful commercial activities at several
pharmaceutical companies and providing key strategic guidance on
company boards. Dr. Anat Cohen-Dayag and Dr. Naama Halevi Davidov
also resigned from the company’s Board of Directors.
- In December, the company and its wholly owned subsidiary, as
borrower, closed on a senior secured convertible term loan of $25
million with certain funds managed by Highbridge Capital
Management, LLC. The loan has a maturity date of December 12,
2024.
Full Year 2022 Financial Results
- Research and development expenses, net were $42.7 million in
2022, compared to $50.2 million in 2021. The decrease was primarily
due to a $9.6 million decrease in clinical and operational
activities relating to the conclusion of our Phase 3 study of
omidubicel, offset by an increase of $1.1 million in T&E and
other expenses as well as a $1.0 million decrease in Israeli
Innovation Authority grants.
- Commercial expenses in 2022 were $12.9 million, compared to
$20.0 million in 2021. The decrease was primarily due to a $8.2
million decrease in commercial launch readiness expenses, offset by
an increase of $1.1 million in headcount related expenses.
- General and administrative expenses were $19.4 million in 2022,
compared to $17.0 million in 2021. The increase was mainly driven
by an increase of $1.4 million attributed to our corporate
headquarters and headcount-related expenses as well as a $1.0
million increase in professional services expenses.
- Financial expenses, net, were $4.4 million for 2022, compared
to $2.6 million for 2021. The increase was primarily due to
expenses relating to the closing on a senior secured convertible
term loan of $25 million with certain funds managed by Highbridge
Capital Management, LLC.
- Net loss for 2022 was $79.4 million, compared to $89.8 million
in 2021.
2023 Financial Guidance
Gamida Cell expects its current cash and cash equivalents will
support the company’s ongoing operating activities through the
third quarter of 2023. 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.
Conference Call Information
Gamida Cell will host a conference call today, March 27, 2023,
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 conference call webcast can be accessed in the “Investors
& Media” section of Gamida Cell’s website at
www.gamida-cell.com. A webcast replay will be available
approximately two hours after the event for approximately 30
days.
About Omidubicel
Omidubicel is an advanced cell therapy candidate for allogeneic
hematopoietic stem cell (bone marrow) transplant that, if approved,
has the potential to expand access and improve outcomes for
patients with blood cancers. Omidubicel demonstrated a
statistically significant reduction in time to neutrophil
engraftment compared to standard umbilical cord blood in an
international, multicenter, randomized Phase 3 study (NCT02730299)
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 a 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 U.S. and E.U. Omidubicel has a PDUFA
target action date of May 1, 2023.
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
GDA-201 is an intrinsic NK cell therapy candidate being
investigated for the treatment of hematologic malignancies.
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. A multicenter Phase 1/2 study of GDA-201 for the
treatment of non-Hodgkin lymphoma is ongoing.
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 a cell therapy pioneer working to turn cells into
powerful therapeutics. The company’s research and development
efforts have produced potentially curative cell therapy candidates
for patients with blood cancers. The company applies a proprietary
expansion platform leveraging the properties of nicotinamide to
cell sources including umbilical cord blood-derived cells and NK
cells to create allogeneic cell therapy candidates with the
potential to redefine standards of care. These include omidubicel,
an advanced cell therapy candidate for allogeneic hematopoietic
stem cell transplant that, if approved, has the potential to expand
access and improve outcomes for patients with blood cancers, and
GDA-201, an intrinsic NK cell therapy candidate being investigated
for the treatment of 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 the FDA’s review of the
BLA for omidubicel, and the potentially life-saving or curative
therapeutic and commercial potential of Gamida Cell’s product
candidates (including omidubicel), and the company’s anticipated
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 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. 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 November 14, 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.
CONSOLIDATED BALANCE SHEETS
U.S. dollars in thousands
(except share and per share data)
December 31,
2022
2021
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
64,657
$
55,892
Marketable securities
-
40,034
Prepaid expenses and other
current assets
1,889
2,688
Total current assets
66,546
98,614
NON-CURRENT ASSETS:
Restricted deposits
3,668
3,961
Property, plant and equipment,
net
44,319
35,180
Operating lease right-of-use
assets
7,024
7,236
Severance pay fund
1,703
2,148
Other long-term assets
1,513
1,647
Total non-current assets
58,227
50,172
Total assets
$
124,773
$
148,786
CONSOLIDATED BALANCE SHEETS
U.S. dollars in thousands
(except share and per share data)
December 31,
2022
2021
LIABILITIES AND SHAREHOLDERS’
EQUITY
CURRENT LIABILITIES:
Trade payables
$
6,384
$
8,272
Employees and payroll
accruals
5,300
4,957
Operating lease liabilities
2,648
2,699
Accrued interest of convertible
senior notes
1,652
1,640
Accrued expenses and current
liabilities
8,891
7,865
Total current liabilities
24,875
25,433
NON-CURRENT LIABILITIES:
Convertible senior notes, net
96,450
71,417
Accrued severance pay
1,914
2,396
Long-term operating lease
liabilities
4,867
5,603
Other long-term liabilities
4,690
-
Total non-current liabilities
107,921
79,416
CONTINGENT LIABILITIES AND
COMMITMENTS
SHAREHOLDERS’ EQUITY
(DEFICIT):
Ordinary shares of NIS 0.01 par
value -
Authorized: 150,000,000 shares at
December 31, 2022 and 2021; Issued: 74,703,030 and 59,970,389
shares at December 31, 2022 and 2021, respectively; Outstanding:
74,583,026 and 59,970,389 shares at December 31, 2022 and 2021,
respectively
211
169
Treasury ordinary shares of NIS
0.01 par value; 120,004 and 0 shares at December 31, 2022 and 2021,
respectively
*
-
Additional paid-in capital
408,598
381,225
Accumulated deficit
(416,832
)
(337,457
)
Total shareholders’ equity (deficit)
(8,023
)
43,937
Total liabilities and shareholders’ equity
$
124,773
$
148,786
* Represents an amount lower than $1.
CONSOLIDATED STATEMENTS OF
OPERATIONS
U.S. dollars in thousands (except share
and per share data)
Year ended
December 31,
2022
2021
Research and development
expenses, net
$
42,692
$
50,177
Commercial expenses
12,900
20,013
General and administrative
expenses
19,401
16,977
Total operating loss
74,993
87,167
Financial expenses, net
4,382
2,626
Loss
$
79,375
$
89,793
Net loss per share attributable
to ordinary shareholders, basic and diluted
$
1.24
$
1.52
Weighted average number of shares
used in computing net loss per share attributable to ordinary
shareholders, basic and diluted
63,826,295
59,246,803
CONSOLIDATED STATEMENTS OF CASH
FLOWS
U.S. dollars in thousands (except share
and per share data)
Year ended
December 31,
2022
2021
Cash flows
from operating activities:
Loss
$
(79,375
)
$
(89,793
)
Adjustments to reconcile loss to
net cash used in operating activities:
Depreciation of property, plant
and equipment
440
431
Financing expense (income),
net
(375
)
359
Share-based compensation
5,041
4,233
Amortization of debt discount and
issuance costs
783
638
Operating lease right-of-use
assets
2,494
2,109
Operating lease liabilities
(3,069
)
(2,193
)
Decrease (increase) accrued
severance pay, net
(37
)
12
Decrease in prepaid expenses and
other assets
224
1,008
Increase (decrease) in trade
payables
(1,888
)
1,941
Increase (decrease) in accrued
expenses and current liabilities
5,339
(505
)
Net cash used in operating
activities
(70,423
)
(81,760
)
Cash flows
from investing activities:
Purchase of property, plant and
equipment
(6,354
)
(15,054
)
Purchase of marketable
securities
(5,037
)
(102,179
)
Proceeds from maturity of
marketable securities
45,029
61,534
Investment in restricted
deposits
-
(5,222
)
Proceeds from restricted
deposits
406
-
Net cash provided by (used in)
investing activities
$
34,044
$
(60,921
)
Cash flows
from financing activities:
Proceeds from exercise of
options
$
76
$
626
Proceeds from share issuance,
net
22,298
-
Proceeds from issuance of
convertible senior notes, net
22,770
70,777
Net cash provided by financing
activities
45,144
71,403
Increase (decrease) in cash and
cash equivalents
8,765
(71,278
)
Cash and cash equivalents at
beginning of year
55,892
127,170
Cash and cash equivalents at end
of year
$
64,657
$
55,892
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Investor and media contact: Dan Boyle Orangefiery
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