– Positive Phase 1 clinical data
presented for PRGN-2012
AdenoVerse™ immunotherapy in
RRP demonstrated favorable safety profile and significant
reduction in surgeries with 50% of the patients in Complete
Response following treatment with PRGN-2012 –
– Enrollment completed in the Phase 2
study of PRGN-2012 in RRP –
– First patient dosed in Phase
1/1b dose escalation/dose expansion
study of PRGN-3007, a next generation UltraCAR-T®
incorporating PD-1 checkpoint inhibition, in advanced
ROR1+ hematological and solid tumors –
– Regained exclusive rights to validated
CAR-T targets, CD19 and BCMA, to enable unencumbered development
and commercialization of UltraCAR-T® –
– Latest clinical advancements for
PRGN-3005 UltraCAR-T® and PRGN-2009 off-the-shelf
AdenoVerse™ immunotherapy to be presented at the
2023 ASCO annual meeting in June –
– Significantly strengthened balance
sheet, raising $72.8 million, net of
offering costs via a public offering of common stock –
– Retired an additional $29.5 million of outstanding convertible notes,
leaving $13.8 million maturing on
July 1, 2023, which will be
retired using the Company's restricted cash balance –
– Cash, cash equivalents, short-term and
long-term investments and restricted cash totaled $125.4 million as of March
31, 2023 –
GERMANTOWN, Md., May 10, 2023
/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 2023 financial results and business updates.
"Precigen continues to execute on our strategy to maintain
corporate strength while advancing our most promising programs.
This quarter, we successfully closed a public offering and more
recently announced that Precigen has regained rights to two
validated targets (CD19 and BCMA) that will further bolster our
already robust UltraCAR-T portfolio and provides an opportunity to
advance potential best-in-class UltraCAR-T drug candidates. We
continue to advance our vision to transform the personalized cell
therapy landscape using Precigen's library approach to build the
most comprehensive clinical and preclinical CAR-T portfolios with
antigen-specific targets spanning both hematological and solid
tumors where there is high unmet medical need for cancer patients,
including CD33, MUC16, ROR1, CD19, BCMA and MSLN," said
Helen Sabzevari, PhD, President and
CEO of Precigen. "We are pleased with the progress of our programs
so far this year. We successfully dosed the first patient with
PRGN-3007 and showcased preclinical data for our MSLN next
generation UltraCAR-T at the 2023 AACR annual meeting. We also
presented positive Phase 1 data for our PRGN-2012 AdenoVerse
immunotherapy in RRP at our R&D day and completed enrollment in
the Phase 2 study. Finally, we look forward to sharing additional
data at the 2023 ASCO annual meeting for our PRGN-3005 UltraCAR-T
and PRGN-2009 AdenoVerse immunotherapy."
"We remain focused on strengthening our financial footing while
containing costs to support our business objectives," said
Harry Thomasian Jr., CFO of
Precigen. "Our program of financial discipline, combined with a
public offering and early retirement of most of our debt, has
provided a solid cash runway to support priorities into late
2024."
Program Highlights
Exclusive Rights to UltraCAR-T® Targets, CD19 and
BCMA, and IL-12 Gene Therapy
- The Company amended its exclusive license agreement with
Alaunos Therapeutics to bolster its portfolio and broaden strategic
opportunities.
- The Company regained exclusive rights to CD19 and B-cell
maturation antigen (BCMA) targets to enable unencumbered
development and commercialization of two validated targets
utilizing the UltraCAR-T® platform.
- The Company also regained exclusive rights to its interleukin
(IL)-12 gene therapy, including application through the
off-the-shelf AdenoVerse immunotherapy platform, paving the
way for potential future treatments in oncology given the important
role of IL-12 cytokines in targeting many types of tumors such as
human papillomavirus (HPV)-associated cancers.
PRGN-2012
AdenoVerse™ Immunotherapy in
RRP
- PRGN-2012 is an investigational off-the-shelf (OTS) AdenoVerse
immunotherapy designed to elicit immune responses directed against
cells infected with HPV 6 or HPV 11 for the treatment of RRP. The
US Food and Drug Administration (FDA) granted orphan drug
designation for PRGN-2012 for patients with recurrent respiratory
papillomatosis (RRP).
- The Company announced positive Phase 1 dose escalation and
expansion cohort data (N=15) in January
2023 at its R&D Day virtual event.
- The Company completed enrollment in the Phase 2 portion of the
study (N=23) bringing the total number of enrolled patients to 35
at Dose Level 2. Patient follow up is ongoing.
- The Company plans to outline the regulatory strategy as FDA
discussions advance.
PRGN 2009
AdenoVerse™ Immunotherapy in
HPV-associated Cancers
- PRGN-2009 is an OTS investigational immunotherapy utilizing the
AdenoVerse platform designed to activate the immune system to
recognize and target HPV-positive (HPV+) solid
tumors.
- The Company completed enrollment in the Phase 1 monotherapy
(N=6) and combination therapy (N=11) arms in patients with
recurrent or metastatic HPV-associated cancers. An abstract for the
clinical data of the PRGN-2009 Phase 1 study (Abstract # 2628)
titled, "Phase I evaluation of PRGN-2009 alone and in combination
with bintrafusp alfa in patients (pts) with recurrent/metastatic
(R/M) HPV-associated cancers (HPV-C)" has been selected for
presentation at the 2023 American Society of Clinical Oncology
(ASCO) Annual Meeting on June 3, 2023
from 8:00 to 11:00 AM CT.
- Enrollment was completed in the Phase 2 monotherapy arm with 20
evaluable patients in newly diagnosed oropharyngeal squamous cell
carcinoma (OPSCC) patients. An interim clinical data presentation
from the Phase 2 monotherapy arm is expected in the second half of
2023.
PRGN-3006 UltraCAR-T® in AML
- 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 kill switch. The FDA granted orphan
drug designation and fast track designation for PRGN-3006
UltraCAR-T for patients with relapsed or refractory (r/r) acute
myeloid leukemia (AML).
- The Company completed the Phase 1 dose escalation study and
announced positive data at the 64th American Society of Hematology
(ASH) Annual Meeting and Exposition. Subsequently, the Company
initiated a multicenter Phase 1b dose
expansion study of PRGN-3006. The Company received FDA clearance to
incorporate repeat dosing in the Phase 1b trial. A Phase 1b clinical data presentation is expected in
2024.
PRGN-3005 UltraCAR-T® in Ovarian
Cancer
- PRGN-3005 UltraCAR-T is an investigational multigenic,
autologous CAR-T cell therapy engineered to express a CAR
specifically targeting the unshed portion of MUC16, mbIL15, and a
kill switch.
- The Company completed enrollment in the Phase 1 dose escalation
cohorts of the intraperitoneal (IP) and intravenous (IV) arms
without lymphodepletion as well as in the lymphodepletion cohort in
the IV arm. An abstract for the clinical data of the PRGN-3005
Phase 1 dose escalation study (Abstract # 5590) titled, "Phase
1/1b study of PRGN-3005 autologous
UltraCAR-T cells manufactured overnight for infusion next day to
advanced stage platinum resistant ovarian cancer patients" has been
selected for presentation at the 2023 ASCO Annual Meeting on
June 5, 2023 from 1:15 to 4:15 PM CT.
- The Company initiated a Phase 1b
dose expansion trial of PRGN-3005. The Company received FDA
clearance to incorporate repeat dosing in the Phase 1b study. A Phase 1b clinical data presentation is expected in
2024.
PRGN-3007 UltraCAR-T® in Advanced ROR1+
Hematological and Solid Tumors
- PRGN-3007, based on the next generation of the 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 kill switch, and a
novel mechanism for the intrinsic blockade of PD-1 gene
expression.
- The Company announced dosing of the first patient in the Phase
1/1b dose escalation/dose expansion
study of PRGN-3007 in advanced ROR1-positive (ROR1+)
hematological and solid tumors. The target patient population for
the Phase 1/1b study includes
hematological cancers (chronic lymphocytic leukemia (CLL), mantle
cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), and
diffuse large B-cell lymphoma (DLBCL)) and solid tumors (triple
negative breast cancer (TNBC)).
Next Generation UltraCAR-T® Platform
- The Company showcased advances in the UltraCAR-T platform with
a preclinical data presentation for the next generation UltraCAR-T
platform utilizing mesothelin (MSLN) CAR from Precigen's library of
non-viral plasmids at the American Association for Cancer Research
(AACR) Annual Meeting 2023. Enhancement of efficacy due to
incorporation of a novel mechanism for PD-1 blockade in MSLN
UltraCAR-T in preclinical models was presented in the abstract
titled, "Next Generation UltraCAR-T® Cells with
Intrinsic Checkpoint Inhibition and Overnight Manufacturing
Overcome Suppressive Tumor Microenvironment Leading to Sustained
Antitumor Activity."
Financial Highlights
- In January 2023, the Company
completed an underwritten public offering of approximately 44
million shares of common stock, including a partial exercise of the
underwriters' option to purchase additional shares, at a price to
the public of $1.75 per share, which
resulted in net proceeds to the Company of $72.8 million (after deducting underwriting
discounts, fees and other expenses).
- During the three months ended March 31,
2023, the Company successfully retired, through open market
purchases, $29.5 million of
outstanding convertible notes due in July
2023 at a discount to par bringing the total outstanding
balance to $13.8 million. Any
remaining outstanding convertible notes will be retired using the
Company's restricted cash balance. Early retirements have saved the
Company close to $7 million through
retirements at discounts to par and reduced interest costs.
- Cash, cash equivalents, short-term and long-term investments
and restricted cash totaled $125.4
million as of March 31,
2023.
- Selling, general and administrative (SG&A) costs decreased
by 15% for the three months ended March 31,
2023 compared to the prior year period.
First Quarter 2023 Financial Results Compared to Prior Year
Period
Research and development expenses increased
$0.4 million, or 3%, from the three
months ended March 31, 2022. This
increase was primarily driven by a continued prioritization of
clinical product candidates.
Total other income, net, increased $2.5
million over the three months ended March 31, 2022. This increase was primarily due
to reduced interest expense associated with the Company's
Convertible Notes as a significant portion of the original
$200 million face value of the
Convertible Notes has been retired. In addition, interest income
increased due to higher interest rates on the Company's
investments.
SG&A expenses decreased $2.1
million, or 15%, from the three months ended March 31, 2022. This decrease was primarily
driven by a reduction in professional fees of $2 million, primarily due to decreased legal fees
associated with certain litigation matters.
Total revenues decreased $3.7
million, or 66%, from the three months ended March 31, 2022. This decrease related to the
recognition of revenue in the first quarter of 2022 related to
agreements for which revenue was previously deferred that did not
occur in the first quarter of 2023 of $1.0
million, as well as declines in services performed at
Exemplar.
Loss from continuing operations was $22.7
million, or $(0.10) per basic
and diluted share, compared to loss from continuing operations of
$23.9 million, or $(0.12) per basic and diluted share, in Q1
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 Twitter @Precigen,
LinkedIn or YouTube.
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 neutralizing antibodies and
effector T cell immune responses as well as an ability to boost
these antibody and T cell responses via repeat administration.
Superior performance characteristics and high yield manufacturing
of AdenoVerse vectors combined with 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 anti-PDL1/TGF-Beta Trap
(M7824) in patients with HPV-associated cancers (NCT04432597) and a
Phase 2 study of PRGN-2012 AdenoVerse immunotherapy in patients
with recurrent respiratory papillomatosis (NCT04724980). PRGN-2012
has been granted Orphan Drug Designation in patients with RRP by
the FDA.
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 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
The UltraCAR-T
platform has shifted the autologous CAR-T manufacturing paradigm
using an advanced non-viral multigene delivery system and an
overnight, decentralized manufacturing process for administration
of autologous CAR-T cells one day after gene transfer to reduce
vein-to-vein time. 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+) hematologic chronic lymphocytic leukemia (CLL),
mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL),
diffuse large B-cell lymphoma (DLBCL) and solid tumor triple
negative breast cancer (TNBC) malignancies (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 cancer 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, AdenoVerse,
UltraVector, UltraPorator, 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, including the
possibility that the timeline for the Company's clinical trials
might be impacted by the COVID-19 pandemic, 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, 2023
|
December 31,
2022
|
Assets
|
|
|
Current
assets
|
|
|
Cash
and cash equivalents
|
$
9,740
|
$
4,858
|
Restricted cash
|
13,800
|
43,339
|
Short-term investments
|
94,351
|
51,092
|
Receivables
|
|
|
Trade,
net
|
1,771
|
978
|
Other
|
13,751
|
12,826
|
Prepaid expenses and other
|
4,330
|
5,066
|
Total current assets
|
137,743
|
118,159
|
Long-term in investments
|
7,460
|
-
|
Property, plant and equipment, net
|
6,908
|
7,329
|
Intangible assets, net
|
43,848
|
44,455
|
Goodwill
|
36,966
|
36,923
|
Right-of-use assets
|
7,617
|
8,086
|
Other assets
|
1,004
|
1,025
|
Total assets
|
$
241,546
|
$
215,977
|
Liabilities and
Shareholders' Equity
|
|
|
Current
liabilities
|
|
|
Accounts payable
|
$
3,809
|
$
4,068
|
Accrued compensation and benefits
|
4,959
|
6,377
|
Other accrued liabilities
|
22,887
|
23,747
|
Deferred revenue
|
15
|
25
|
Current portion of long-term debt
|
13,819
|
43,219
|
Current portion of lease liabilities
|
1,244
|
1,209
|
Total current liabilities
|
46,733
|
78,645
|
Deferred revenue, net of current portion
|
1,818
|
1,818
|
Lease liabilities, net of current portion
|
6,623
|
6,992
|
Deferred tax liabilities
|
2,239
|
2,263
|
Total liabilities
|
57,413
|
89,718
|
Commitments and
contingencies (Note 14)
|
|
|
Shareholders'
equity
|
|
|
Common stock
|
-
|
-
|
Additional paid-in capital
|
2,078,133
|
1,998,314
|
Accumulated deficit
|
(1,891,301)
|
(1,868,567)
|
Accumulated other comprehensive loss
|
(2,699)
|
(3,488)
|
Total shareholders' equity
|
184,133
|
126,259
|
Total liabilities and shareholders' equity
|
$
241,546
|
$
215,977
|
Precigen, Inc. and
Subsidiaries
|
Consolidated
Statements of Operations
|
(Unaudited)
|
|
Three months
ended
|
(Amounts in
thousands, except share and per share data)
|
March 31, 2023
|
March 31, 2022
|
Revenues
|
|
|
Product
revenues
|
$
324
|
$
492
|
Service
revenues
|
1,527
|
4,933
|
Other
revenues
|
-
|
88
|
Total
revenues
|
1,851
|
5,513
|
|
|
|
Operating
Expenses
|
|
|
Cost of products and
services
|
1,527
|
1,694
|
Research and
development
|
12,163
|
11,801
|
Selling, general and
administrative
|
11,639
|
13,689
|
Impairment of
goodwill
|
-
|
482
|
Total operating
expenses
|
25,329
|
27,666
|
Operating
loss
|
(23,478)
|
(22,153)
|
Other Income
(Expense), Net
|
|
|
Interest
expense
|
(324)
|
(2,038)
|
Interest
income
|
633
|
38
|
Other income,
net
|
380
|
198
|
Total other
income (expense), net
|
689
|
(1,802)
|
Equity in net loss of
affiliates
|
-
|
(1)
|
Loss from continuing
operations before income taxes
|
(22,789)
|
(23,956)
|
Income tax
benefit
|
55
|
58
|
Loss from continuing operations
|
(22,734)
|
(23,898)
|
Income from discontinued operations, net of income taxes
|
-
|
4,647
|
Net loss
|
$
(22,734)
|
$
(19,251)
|
Net Loss per
share
|
|
|
Net loss from
continuing operations per share, basic and diluted
|
$
(0.10)
|
$
(0.12)
|
Net income from
discontinued operations per share, basic and diluted
|
-
|
0.02
|
Net loss per share,
basic and diluted
|
$
(0.10)
|
$
(0.10)
|
Weighted average shares
outstanding, basic and diluted
|
229,770,381
|
199,629,218
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/precigen-reports-first-quarter-2023-financial-results-and-business-updates-301820418.html
SOURCE Precigen, Inc.