– Pivotal Phase 2 study data of
PRGN-2012 for the treatment of patients with recurrent respiratory
papillomatosis to be presented at the 2024 ASCO Annual Meeting as a
late-breaking oral presentation on June
3rd –
– Company to host a conference
call on June 3rd following
the PRGN-2012 ASCO presentation to discuss in detail the pivotal
study results and provide business updates –
– PRGN-2012 rolling BLA
submission, under an accelerated approval pathway, is anticipated
in the second half of 2024; commercial readiness activities
underway for a potential launch in 2025 –
– Two trial-in-progress
presentations for PRGN-2009 in combination with pembrolizumab for
the treatment of recurrent/metastatic cervical cancer
and oropharyngeal cancer to be presented at ASCO –
– Company and the Recurrent
Respiratory Papillomatosis Foundation to co-sponsor the inaugural
RRP Awareness Day on June
11th to raise awareness and bring together
individuals living with RRP, caregivers, clinicians, and government
officials –
– Cash, cash equivalents, and
short-term investments totaled $44.8
million as of March 31, 2024
–
GERMANTOWN, Md., May 14, 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 first
quarter 2024 financial results and business updates.
"We are excited to share the pivotal Phase 2 data for our
PRGN-2012 study in patients with RRP at ASCO and look forward to
providing additional details regarding the results at our planned
conference call following the presentation. We remain on track for
a PRGN-2012 rolling BLA submission in the second half of 2024 and
we are actively moving ahead with our commercial readiness efforts
in anticipation of a potential launch of PRGN-2012 in 2025," said
Helen Sabzevari, PhD, President and
CEO of Precigen. "Based on the competitive advantages of PRGN-2012,
including a favorable route of administration, safety profile and
the efficacy demonstrated in the clinical trial results so far, we
believe PRGN-2012 has the potential to be the first-in-class and
best-in-class treatment for RRP patients. We anticipate
PRGN-2012 to overwhelmingly be the treatment of choice for RRP
patients, if approved, as indicated by our commissioned research of
healthcare providers and key opinion leaders which found
PRGN-2012's competitive advantages highly compelling."
"With multiple milestones anticipated in 2024 and 2025, 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®
- PRGN-2012 in RRP: PRGN-2012 is an investigational
off-the-shelf AdenoVerse gene therapy 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 was the first to receive
Breakthrough Therapy Designation and an accelerated approval
pathway for RRP from the FDA. PRGN-2012 received 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).
- Results from 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).
- PRGN-2012 demonstrated overall safety and clinically meaningful
benefit with 50% of patients (N=12) in Complete Response, which is
defined as no surgeries needed during the 12-month period following
PRGN-2012 treatment completion. All Complete Responses were durable
and ongoing more than two years after PRGN-2012 treatment.
- 83% of patients had a reduction in RRP surgeries in the
12-month period after PRGN-2012 treatment compared to 12 months
pre-treatment.
- Correlative data support expansion of peripheral HPV 6 and HPV
11–specific T cell immunological responses as the underlying
mechanism of action for PRGN-2012.
- PRGN-2012 is built using the Company's differentiated gorilla
adenovector that allows for repeat dosing. The redosing potential
of AdenoVerse has been highlighted in clinical studies where repeat
administrations of PRGN-2009 and PRGN-2012 gene therapies led to
enhancement of antigen-specific T cell immune responses
without generation of significant neutralizing antibodies in
contrast to other viral vectors.
- Results from the pivotal Phase 2 study of PRGN-2012 for the
treatment of RRP, including immunological responses, will be
presented at the 2024 American Society of Clinical Oncology (ASCO)
Annual Meeting in a late-breaking oral presentation titled,
"PRGN-2012, a novel gorilla adenovirus-based immunotherapy,
provides the first treatment that leads to complete and durable
responses in recurrent respiratory papillomatosis patients."
Scott M. Norberg, DO, Associate
Research Physician, Center for Immuno-Oncology, Center for Cancer
Research, National Cancer Institute, will deliver the presentation
on June 3, 2024 at 8:30 AM CT.
- The Company plans to host a conference call on June 3, 2024 to discuss in detail the PRGN-2012
pivotal study results presented and provide business updates.
- FDA confirmed that the ongoing Phase 1/2 single arm study will
serve as pivotal and no additional randomized, placebo-controlled
trial will be required to support submission of a Biologics License
Application (BLA). A rolling BLA submission under an accelerated
approval pathway is anticipated in the second half of 2024. Based
on FDA guidance, the Company is on track to initiate a confirmatory
study prior to submission of the BLA.
- Commercial readiness preparations are underway for a potential
launch in 2025.
- The Company and the Recurrent Respiratory Papillomatosis
Foundation will co-sponsor the inaugural RRP Awareness Day on
June 11, 2024. The multi-stakeholder
event will raise awareness and bring together individuals living
with RRP, caregivers, clinicians, and government officials to
encourage new connections and build community among those
interested in and affected by RRP. The inaugural event will be
hybrid with in-person participation at the National Press Club in
Washington DC and a webcast for
virtual participation.
- PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is
an investigational off-the-shelf AdenoVerse gene therapy
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).
- An abstract titled, "Phase II trial of immunotherapeutic HPV
vaccine PRGN-2009 with pembrolizumab before standard treatment in
subjects with newly diagnosed HPV-associated oropharyngeal cancer"
will be presented as a trial-in-progress poster presentation on
June 2, 2024 from 9:00 AM to 12:00 PM CT at ASCO.
- 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).
- An abstract titled, "A Phase 2 study to evaluate efficacy and
safety of PRGN-2009, a novel gorilla adenovirus-based
immunotherapy, in combination with pembrolizumab versus
pembrolizumab monotherapy in patients with recurrent or metastatic
cervical cancer" will be presented as a trial-in-progress poster
presentation on June 3, 2024 from
9:00 AM to 12:00 PM CT at ASCO.
UltraCAR-T®
- 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).
- 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, and short-term investments totaled
$44.8 million as of March 31, 2024.
- Selling, general, and administrative (SG&A) costs decreased
13% compared to the three months ended March
31, 2023.
- Property, plant, and equipment, net, increased $5.5 million from December
31, 2023 primarily due to the build-out of our cGMP
manufacturing facility.
First Quarter 2024 Financial Results Compared to Prior Year
Period
Research and development expenses increased $2.1 million, or 17%, compared to the three
months ended March 31, 2023.
Salaries, benefits, and other personnel costs increased
$1.5 million due to an increase in
the hiring of employees throughout 2023 to support the growth in
the Company's clinical development activities as well as increased
fees paid to consultants and contract research organizations in the
first quarter of 2024 compared to the same period in 2023.
SG&A expenses decreased $1.5
million, or 13%, compared to the three months ended
March 31, 2023, primarily driven by a
reduction in stock compensation and insurance expenses in the first
quarter of 2024 compared to same period in 2023. In addition, the
costs associated with PRGN-2012 commercial readiness increased
compared to the same period in 2023.
Total revenues decreased $0.8
million, or 43%, compared to the three months ended
March 31, 2023. This decrease was due
to the reduction in products and services performed at Exemplar.
Gross margin on product and services also declined in the current
period primarily as a result of the decreased revenues at
Exemplar.
Net Loss was $23.7 million, or
$(0.10) per basic and diluted share,
compared to net loss of $22.7
million, or $(0.10) per basic
and diluted share, in period ended March 31,
2023.
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.
AdenoVerse®
Precigen's AdenoVerse 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 gene therapies 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® Clinical Programs
Precigen's
AdenoVerse platform is currently under clinical investigation in a
Phase 1/2 study of PRGN-2009 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 in combination with pembrolizumab in
patients with recurrent or metastatic cervical cancer
(NCT06157151), and a Phase 1/2 study of PRGN-2012 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.
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 Programs
Precigen's
UltraCAR-T platform is currently under clinical investigation for
hematological and solid tumors, including a Phase 1/1b study of PRGN-3005 in patients with advanced,
recurrent platinum resistant ovarian, fallopian tube or primary
peritoneal cancer (NCT03907527), a Phase 1/1b study of PRGN-3006 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 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 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.
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)
|
March 31,
2024
|
December 31,
2023
|
Assets
|
|
|
Current
assets
|
|
|
Cash
and cash equivalents
|
$
17,478
|
$
7,578
|
Short-term investments
|
27,280
|
55,277
|
Receivables
|
|
|
Trade,
net
|
872
|
902
|
Other
|
290
|
673
|
Prepaid expenses and other
|
3,626
|
4,325
|
Total current assets
|
49,546
|
68,755
|
Property, plant and equipment, net
|
12,620
|
7,111
|
Intangible assets, net
|
38,717
|
40,701
|
Goodwill
|
26,555
|
26,612
|
Right-of-use assets
|
6,658
|
7,097
|
Other assets
|
751
|
767
|
Total assets
|
$
134,847
|
$
151,043
|
Liabilities and
Shareholders' Equity
|
|
|
Current
liabilities
|
|
|
Accounts payable
|
$
4,716
|
$
1,726
|
Accrued compensation and benefits
|
9,962
|
8,250
|
Other accrued liabilities
|
7,296
|
6,223
|
Settlement and
Indemnification Accrual
|
5,075
|
5,075
|
Deferred revenue
|
407
|
509
|
Current portion of lease liabilities
|
1,318
|
1,202
|
Total current liabilities
|
28,774
|
22,985
|
Deferred revenue, net of current portion
|
1,888
|
1,818
|
Lease liabilities, net of current portion
|
5,387
|
5,895
|
Deferred tax liabilities
|
1,779
|
1,847
|
Total liabilities
|
37,828
|
32,545
|
Shareholders'
equity
|
|
|
Common stock
|
-
|
-
|
Additional paid-in capital
|
2,088,025
|
2,084,916
|
Accumulated deficit
|
(1,988,209)
|
(1,964,471)
|
Accumulated other comprehensive loss
|
(2,797)
|
(1,947)
|
Total shareholders' equity
|
97,019
|
118,498
|
Total liabilities and shareholders' equity
|
$
134,847
|
$
151,043
|
Precigen, Inc. and
Subsidiaries
Consolidated
Statements of Operations
(Unaudited)
|
|
Three Months
Ended
|
(Amounts in
thousands, except share and per share data)
|
March 31,
2024
|
March 31,
2023
|
Revenues
|
|
|
Product
revenues
|
$
138
|
$
324
|
Service
revenues
|
919
|
1,527
|
Other
revenues
|
8
|
-
|
Total
revenues
|
1,065
|
1,851
|
Operating
Expenses
|
|
|
Cost of products and
services
|
1,075
|
1,527
|
Research and
development
|
14,249
|
12,163
|
Selling, general and
administrative
|
10,151
|
11,639
|
Total operating
expenses
|
25,475
|
25,329
|
Operating
loss
|
(24,410)
|
(23,478)
|
Other Income
(Expense), Net
|
|
|
Interest
expense
|
(2)
|
(324)
|
Interest
income
|
608
|
633
|
Other income,
net
|
37
|
380
|
Total other income,
net
|
643
|
689
|
Loss before income
taxes
|
(23,767)
|
(22,789)
|
Income tax
benefit
|
29
|
55
|
Net loss
|
$
(23,738)
|
$
(22,734)
|
Net Loss per
share
|
|
|
Net loss per share,
basic and diluted
|
$
(0.10)
|
$
(0.10)
|
Weighted average shares
outstanding, basic and diluted
|
249,220,335
|
229,770,381
|
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SOURCE Precigen, Inc.