– Significant progress made in the
development of the PRGN-2012 AdenoVerse immunotherapy for the
treatment of RRP; Precigen plans to submit a BLA under an
accelerated approval pathway in the second half of 2024; ramping up
commercial readiness activities for a potential launch in 2025
–
– Precigen's PRGN-2012 received
the first Breakthrough Therapy Designation and accelerated approval
pathway from the FDA for the treatment of RRP –
– Precigen received IND clearance
for a randomized Phase 2 study of PRGN-2009 AdenoVerse
immunotherapy in combination with pembrolizumab in HPV-associated
recurrent/metastatic cervical cancer; study now active and
recruiting patients –
– Interim data from the ongoing
Phase 1b study of PRGN-3006
UltraCAR-T in relapsed/refractory AML anticipated in the second
half of 2024 –
– Preliminary data from the Phase
1 study of PRGN-3007 next generation UltraCAR-T in ROR1+ advanced
cancers anticipated in the second half of 2024 –
– Cash, cash equivalents,
short-term and long-term investments totaled $62.9 million as of December 31, 2023 –
– Continued focus on cost
containment resulted in a reduction in SG&A costs of 16% for
the twelve months ended December 31,
2023, compared to the prior year period –
GERMANTOWN, Md., March 19,
2024 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a
biopharmaceutical company specializing in the development of
innovative gene and cell therapies to improve the lives of
patients, today announced full year 2023 financial results and
business updates.
"2024 is poised to be a transformative year for Precigen, as we
are on track to present pivotal Phase 2 data in the second
quarter and submit a BLA for PRGN-2012 in the second half, a
milestone bolstered by the Breakthrough Therapy Designation and
accelerated approval pathway granted by the FDA," said
Helen Sabzevari, PhD, President and CEO of Precigen. "We are
preparing our manufacturing facility and commercial operations in
anticipation of the launch of PRGN-2012 in 2025. We are also
looking forward to exciting updates from our UltraCAR-T programs,
which offer a novel approach compared to traditional CAR-T
therapies and have garnered significant interest from potential
partners due to the safety, preliminary efficacy, and manufacturing
advantages."
"With multiple value inflection points anticipated in
2024, we remain steadfastly committed to a strategy of sound
financial management," said Harry Thomasian
Jr., CFO of Precigen. "We are evaluating various
financing opportunities to strengthen our balance sheet as we
prepare our lead asset PRGN-2012 for potential commercial launch in
2025."
Key Program Highlights
AdenoVerse™ Immunotherapies
- PRGN-2012 in RRP: PRGN-2012 is an investigational
off-the-shelf AdenoVerse immunotherapy designed to elicit immune
responses directed against cells infected with human papillomavirus
(HPV) 6 or HPV 11 for the treatment of recurrent respiratory
papillomatosis (RRP). PRGN-2012 has received Breakthrough Therapy
Designation and Orphan Drug Designation from the US Food and Drug
Administration (FDA) and Orphan Drug Designation from the European
Commission.
- PRGN-2012 is currently under investigation in a Phase 1/2
pivotal single-arm study in adult patients with RRP (clinical trial
identifier: NCT04724980).
- PRGN-2012 demonstrated strong efficacy and a favorable safety
profile in the Phase 1 portion of the study with 50% of
patients (N=12) in durable and ongoing Complete Response more than
two years after PRGN-2012 treatment. Results of the Phase 1 portion
of the Phase 1/2 study were published in the peer-reviewed journal,
Science Translational Medicine, a leading
publication from the American Association for the Advancement of
Science (AAAS).
- Enrollment and dosing in the Phase 2 portion of the study is
complete and a Phase 2 data presentation is anticipated in the
second quarter of 2024.
- The Company has received agreement from the FDA that PRGN-2012
is eligible for consideration of a rolling Biologics License
Application (BLA) review. A planned BLA submission under an
accelerated approval pathway is anticipated in the second half of
2024.
- Commercial readiness preparations are underway for a potential
launch in 2025.
- PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is
an investigational off-the-shelf AdenoVerse immunotherapy
designed to activate the immune system to recognize and target
HPV-associated cancers.
- The Phase 2 study of PRGN-2009 in combination with
pembrolizumab in newly diagnosed patients with HPV-associated
oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients
(clinical trial identifier: NCT05996523).
- The Phase 2 randomized, open-label study of PRGN-2009 in
combination with pembrolizumab in patients with HPV-associated
recurrent/metastatic cervical cancer is active and recruiting
patients (clinical trial identifier: NCT06157151).
UltraCAR-T® Cell Therapies
- PRGN-3006 in AML/MDS: PRGN-3006 is an
investigational multigenic, autologous chimeric antigen receptor T
cell (CAR-T) therapy engineered to simultaneously express a CAR
specifically targeting CD33, membrane bound IL-15 (mbIL15), and a
safety/kill switch. PRGN-3006 has been granted Orphan Drug
Designation in patients with acute myeloid leukemia (AML) and
Fast Track Designation in patients with relapsed/refractory
(r/r) AML by the FDA.
- PRGN-3006 is currently under investigation in a Phase
1b dose expansion clinical trial
(clinical trial identifier: NCT03927261) for the treatment of
patients with r/r AML or higher-risk myelodysplastic syndromes
(MDS).
- The first-in-human Phase 1 dose escalation study data show that
PRGN-3006 was well-tolerated with no dose-limiting toxicities
(DLTs) and a 27% objective response rate (ORR) in heavily
pre-treated r/r AML patients infused following
lymphodepletion.
- An interim Phase 1b dose
expansion data presentation is anticipated in the second half of
2024.
- PRGN-3005 in Ovarian Cancer: PRGN-3005 is an
investigational multigenic, autologous CAR-T cell therapy
engineered to express a CAR specifically targeting the unshed
portion of MUC16, mbIL15, and a safety/kill switch.
- The Phase 1b dose expansion
portion of the Phase 1/1b study is
ongoing (clinical trial identifier: NCT03907527).
- PRGN-3007 in Advanced ROR1+ Hematological and Solid
Tumors: PRGN-3007, based on the next generation UltraCAR-T
platform, is an investigational multigenic, autologous CAR-T cell
therapy engineered to express a CAR targeting receptor tyrosine
kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch,
and a novel mechanism for the intrinsic blockade of PD-1 gene
expression.
- The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier:
NCT05694364).
- A preliminary Phase 1 dose escalation data presentation is
anticipated by the end of 2024.
Financial Highlights
- Cash, cash equivalents, short-term and long-term investments
totaled $62.9 million as of
December 31, 2023.
- Selling, general, and administrative (SG&A) costs decreased
versus the prior year, 16% for the twelve months ended December 31, 2023.
Full Year 2023 Financial Results Compared to Prior Year
Period
Research and development expenses increased $1.4 million, or 3.1%, compared to year ended
December 31, 2022. Salaries,
benefits, and other personnel costs increased $2.8 million due to an increase in the hiring of
employees to support the growth in the Company's development
activities, and to a lesser extent, increases in salaries of our
continuing employees. These increases were offset by less expenses
incurred related to preclinical research programs for the
comparable period.
SG&A expenses decreased $7.6
million, or 15.8%, compared to the year ended December 31, 2022. This decrease was primarily
driven by a reduction in professional fees of $6.5 million, due to decreased legal fees
associated with certain litigation matters, and $0.7 million decreased insurance-related
premiums.
Total revenues decreased $20.7
million, or 76.9%, compared to the year ended December 31, 2022. Collaboration and licensing
revenues decreased $14.6 million, or
99.5%, compared to the year ended December
31, 2022, primarily due to the prior year period non-cash
recognition of revenue related to historical collaboration
agreements for which revenue was previously deferred. Product and
services revenues decreased $5.9
million, or 48.8%, compared to the year ended December 31, 2022. This decrease is related to
reductions in services performed at Exemplar as well as the
recognition of revenue in the first quarter of 2022 related to
agreements for which revenue was previously deferred that did not
occur in 2023.
Total other income, net, increased $8.5
million, compared to the year ended December 31, 2022. This was primarily due to
$6.3 million in reduced interest
expense associated with the Convertible Notes as they were fully
retired in the second quarter of 2023, and $3.1 million increased interest income due to
higher interest rates on investments. This increase was partially
offset by a $0.9 million decrease in
gain recorded on the early retirement of a portion of our
Convertible Notes compared to the year ended December 31, 2022.
The Company recorded a $10.4
million impairment charge in the fourth quarter of 2023
related to its Exemplar subsidiary as a result of the Company's
annual goodwill impairment test.
Loss from continuing operations was $95.9
million, or $(0.39) per basic
and diluted share, compared to loss from continuing operations of
$79.8 million, or $(0.40) per basic and diluted share, in year
ended December 31, 2022.
Precigen: Advancing Medicine with
Precision™
Precigen (Nasdaq: PGEN) is a dedicated
discovery and clinical stage biopharmaceutical company advancing
the next generation of gene and cell therapies using precision
technology to target the most urgent and intractable diseases in
our core therapeutic areas of immuno-oncology, autoimmune
disorders, and infectious diseases. Our technologies enable us to
find innovative solutions for affordable biotherapeutics in a
controlled manner. Precigen operates as an innovation engine
progressing a preclinical and clinical pipeline of
well-differentiated therapies toward clinical proof-of-concept and
commercialization. For more information about Precigen, visit
www.precigen.com or follow us on X @Precigen, LinkedIn or
YouTube.
UltraCAR-T®
UltraCAR-T is a multigenic
autologous CAR-T platform that utilizes Precigen's advanced
non-viral Sleeping Beauty system to simultaneously express
an antigen-specific CAR to specifically target tumor cells, mbIL15
for enhanced in vivo expansion and persistence, and a kill
switch to conditionally eliminate CAR-T cells for a potentially
improved safety profile. Precigen has advanced the UltraCAR-T
platform to address the inhibitory tumor microenvironment by
incorporating a novel mechanism for intrinsic checkpoint blockade
without the need for complex and expensive gene editing techniques.
UltraCAR-T investigational therapies are manufactured via
Precigen's overnight manufacturing process using the proprietary
UltraPorator® electroporation system at the patient's
medical center and administered to patients only one day following
gene transfer. The overnight UltraCAR-T manufacturing process does
not use viral vectors and does not require ex vivo
activation and expansion of T cells, potentially addressing major
limitations of current T cell therapies.
UltraCAR-T® Clinical Program
Precigen's
UltraCAR-T platform is currently under clinical investigation for
hematological and solid tumors, including a Phase 1/1b study of PRGN-3005 UltraCAR-T in patients with
advanced, recurrent platinum resistant ovarian, fallopian tube or
primary peritoneal cancer (NCT03907527), a Phase 1/1b study of PRGN-3006 UltraCAR-T in patients with
relapsed or refractory acute myeloid leukemia (AML) or higher risk
myelodysplastic syndrome (MDS) (NCT03927261) and a Phase
1/1b study of PRGN-3007 UltraCAR-T
incorporating PD-1 checkpoint inhibition in patients with
ROR1-positive (ROR1+) chronic lymphocytic leukemia
(CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia
(ALL), diffuse large B-cell lymphoma (DLBCL) and triple negative
breast cancer (TNBC) (NCT05694364). PRGN-3006 UltraCAR-T has been
granted Orphan Drug Designation and Fast Track Designation in
patients with AML by the US Food and Drug Administration (FDA).
UltraCAR-T® Library Approach
Precigen's
UltraCAR-T library approach is designed to transform the
personalized cell therapy landscape for cancer patients. Precigen's
goal is to develop and validate a library of non-viral plasmids to
target tumor-associated antigens. Enabled by design and
manufacturing advantages of UltraCAR-T, coupled with the
capabilities of the UltraPorator® system, Precigen is working
to empower medical centers to deliver personalized, autologous
UltraCAR-T treatment with overnight manufacturing to any cancer
patient. Based on the patient's cancer indication and biomarker
profile, one or more non-viral plasmids would be selected from the
library to build a personalized UltraCAR-T treatment. After initial
treatment, this approach has the potential to allow for redosing of
UltraCAR-T targeting the same or new tumor-associated antigen(s)
based on the treatment response and the changes in antigen
expression of the patient's tumor. Precigen believes that the
combination of the advanced UltraVector® DNA
construction platform and the ease of overnight manufacturing gives
this library approach a proprietary advantage over traditional
T-cell therapies.
UltraPorator®
The UltraPorator system is an
exclusive device and proprietary software solution for the scale-up
of rapid and cost-effective manufacturing of UltraCAR-T therapies
and potentially represents a major advancement over current
electroporation devices by significantly reducing the processing
time and contamination risk. The UltraPorator device is a
high-throughput, semi-closed electroporation system for modifying T
cells using Precigen's proprietary non-viral gene transfer
technology. UltraPorator is being utilized for clinical
manufacturing of Precigen's investigational UltraCAR-T therapies in
compliance with current good manufacturing practices.
AdenoVerse™ Immunotherapy
Precigen's
AdenoVerse immunotherapy platform utilizes a library of proprietary
adenovectors for the efficient gene delivery of therapeutic
effectors, immunomodulators, and vaccine antigens designed to
modulate the immune system. Precigen's gorilla adenovectors, part
of the AdenoVerse library, have potentially superior performance
characteristics as compared to current competition. AdenoVerse
immunotherapies have been shown to generate high-level and durable
antigen-specific T-cell immune responses as well as an ability to
boost these responses via repeat administration. Superior
performance characteristics and high yield manufacturing of
AdenoVerse vectors leveraging UltraVector® technology
allows Precigen to engineer cutting-edge investigational gene
therapies to treat complex diseases.
AdenoVerse™ Immunotherapy Clinical
Program
Precigen's AdenoVerse immunotherapy platform is
currently under clinical investigation in a Phase 1/2 study of
PRGN-2009 AdenoVerse immunotherapy alone or in combination with an
anti-PDL1/TGF-Beta Trap in patients with HPV-associated cancers
(NCT04432597), a Phase 2 study of PRGN-2009 in combination with
pembrolizumab in newly diagnosed patients with HPV-associated
oropharyngeal squamous cell carcinoma (OPSCC) (NCT05996523), a
Phase 2 study of PRGN-2009 AdenoVerse immunotherapy in combination
with pembrolizumab in patients with recurrent or metastatic
cervical cancer (NCT06157151), and a Phase 1/2 study of
PRGN-2012 AdenoVerse immunotherapy in patients with recurrent
respiratory papillomatosis (RRP) (NCT04724980). PRGN-2012 has been
granted Orphan Drug Designation and Breakthrough Therapy
Designation in patients with RRP by the FDA and Orphan Drug
Designation by the European Commission.
Trademarks
Precigen, UltraCAR-T, UltraPorator,
AdenoVerse, UltraVector and Advancing Medicine with Precision are
trademarks of Precigen and/or its affiliates. Other names
may be trademarks of their respective owners.
Cautionary Statement Regarding Forward-Looking
Statements
Some of the statements made in this press release
are forward-looking statements. These forward-looking statements
are based upon the Company's current expectations and projections
about future events and generally relate to plans, objectives, and
expectations for the development of the Company's business,
including the timing and progress of preclinical studies, clinical
trials, discovery programs and related milestones, the promise of
the Company's portfolio of therapies, and in particular its CAR-T
and AdenoVerse therapies. Although management believes that the
plans and objectives reflected in or suggested by these
forward-looking statements are reasonable, all forward-looking
statements involve risks and uncertainties and actual future
results may be materially different from the plans, objectives and
expectations expressed in this press release. The Company has no
obligation to provide any updates to these forward-looking
statements even if its expectations change. All forward-looking
statements are expressly qualified in their entirety by this
cautionary statement. For further information on potential risks
and uncertainties, and other important factors, any of which could
cause the Company's actual results to differ from those contained
in the forward-looking statements, see the section entitled "Risk
Factors" in the Company's most recent Annual Report on Form 10-K
and subsequent reports filed with the Securities and Exchange
Commission.
Investor Contact:
Steven M.
Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
Donelle M.
Gregory
press@precigen.com
Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com
Precigen, Inc. and
Subsidiaries
Consolidated Balance
Sheets
(Unaudited)
|
(Amounts in
thousands)
|
December 31,
2023
|
December 31,
2022
|
Assets
|
|
|
Current
assets
|
|
|
Cash
and cash equivalents
|
$
7,578
|
$
4,858
|
Restricted cash
|
-
|
43,339
|
Short-term investments
|
55,277
|
51,092
|
Receivables
|
|
|
Trade,
net
|
902
|
978
|
Other
|
673
|
12,826
|
Prepaid expenses and other
|
4,325
|
5,066
|
Total current assets
|
68,755
|
118,159
|
Property, plant and equipment, net
|
7,111
|
7,329
|
Intangible assets, net
|
40,701
|
44,455
|
Goodwill
|
26,612
|
36,923
|
Right-of-use assets
|
7,097
|
8,086
|
Other assets
|
767
|
1,025
|
Total assets
|
$
151,043
|
$
215,977
|
Liabilities and
Shareholders' Equity
|
|
|
Current
liabilities
|
|
|
Accounts payable
|
$
1,726
|
$
4,068
|
Accrued compensation and benefits
|
8,250
|
6,377
|
Other accrued liabilities
|
6,223
|
4,997
|
Settlement and
Indemnification Accrual
|
5,075
|
18,750
|
Deferred revenue
|
509
|
25
|
Current portion of long-term debt
|
-
|
43,219
|
Current portion of lease liabilities
|
1,202
|
1,209
|
Total current liabilities
|
22,985
|
78,645
|
Deferred revenue, net of current portion
|
1,818
|
1,818
|
Lease liabilities, net of current portion
|
5,895
|
6,992
|
Deferred tax liabilities
|
1,847
|
2,263
|
Total liabilities
|
32,545
|
89,718
|
Shareholders'
equity
|
|
|
Common stock
|
-
|
-
|
Additional paid-in capital
|
2,084,916
|
1,998,314
|
Accumulated deficit
|
(1,964,471)
|
(1,868,567)
|
Accumulated other comprehensive loss
|
(1,947)
|
(3,488)
|
Total shareholders' equity
|
118,498
|
126,259
|
Total liabilities and shareholders' equity
|
$
151,043
|
$
215,977
|
|
|
|
Precigen, Inc. and
Subsidiaries
Consolidated
Statements of Operations
(Unaudited)
|
|
Year
ended
|
(Amounts in
thousands, except share and per share data)
|
December 31,
2023
|
December 31,
2022
|
Revenues
|
|
|
Collaboration
and licensing revenues
|
$
75
|
$
14,661
|
Product
revenues
|
840
|
1,903
|
Service
revenues
|
5,301
|
10,094
|
Other
revenues
|
9
|
251
|
Total
revenues
|
6,225
|
26,909
|
Operating
Expenses
|
|
|
Cost of products and
services
|
6,119
|
6,339
|
Research and
development
|
48,614
|
47,170
|
Selling, general and
administrative
|
40,415
|
48,006
|
Impairment of
goodwill
|
10,390
|
482
|
Impairment of other
noncurrent assets
|
445
|
638
|
Total operating
expenses
|
105,983
|
102,635
|
Operating
loss
|
(99,758)
|
(75,726)
|
Other Expense,
Net
|
|
|
Interest
expense
|
(468)
|
(6,774)
|
Interest
income
|
3,237
|
133
|
Other income,
net
|
627
|
1,539
|
Total other income
(expense), net
|
3,396
|
(5,102)
|
Equity in net loss of
affiliates
|
-
|
862
|
Loss from continuing
operations before income taxes
|
(96,362)
|
(79,966)
|
Income tax
benefit
|
458
|
189
|
Loss from continuing
operations
|
$
(95,904)
|
$
(79,777)
|
Income from
discontinued operations, net of income taxes
|
-
|
108,094
|
Net loss
|
$
(95,904)
|
$
28,317
|
Net Loss per
share
|
|
|
Net loss from
continuing operations per share, basic and diluted
|
$
(0.39)
|
$
(0.40)
|
Net income from
discontinued operations per share, basic and diluted
|
-
|
0.54
|
Net loss per share,
basic and diluted
|
$
(0.39)
|
$
0.14
|
Weighted average shares
outstanding, basic and diluted
|
244,536,221
|
200,360,821
|
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SOURCE Precigen, Inc.