– The FDA confirmed that the
ongoing Phase 1/2 single arm study of PRGN-2012 in RRP will serve
as the pivotal study to support accelerated approval and no
additional randomized, placebo-controlled trial will be required to
support submission of a BLA –
– Company to prioritize portfolio
activities to accelerate PRGN-2012 and continue advancement of
other key programs, by implementing strategies to reduce clinical
costs (e.g., reducing CRO costs without internal R&D headcount
reduction) and reduce SG&A costs –
– Completely retired the
outstanding balance of convertible notes –
– Cash, cash equivalents,
short-term and long-term investments totaled $95.6 million as of June
30, 2023; cash runway projected into 2025 –
GERMANTOWN, Md., Aug. 9, 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 second quarter and first half
2023 financial results and business updates.
"Today we announced that the FDA confirmed the ongoing Phase 1/2
study of PRGN-2012 will serve as the pivotal study to support
accelerated approval, an important milestone that brings Precigen a
step closer in our transition to a commercial stage company and in
realizing our vision of bringing life-changing therapies to
patients with unmet medical need. We look forward to working with
the FDA to submit a BLA and potentially bringing the first drug
therapy for RRP patients. As a result of this exciting milestone,
we are prioritizing our portfolio activities to maximize the
potential success of PRGN-2012 while continuing to strategically
advance other key programs," said Helen
Sabzevari, PhD, President and CEO of Precigen. "We have
built a strong portfolio based on the AdenoVerse and UltraCAR-T
platforms and continue to advance important programs with a focus
on agility, efficiency and commercial viability."
"As a result of our capital raise in January, our
portfolio prioritization and other cost-saving measures, including
completely retiring our convertible notes, the Company's balance
sheet is well positioned for the future," said Harry Thomasian Jr., CFO of Precigen. "These
measures have enabled us to extend our projected cash runway into
2025, exclusive of non-dilutive strategies, including strategic
partnerships, which could extend our cash runway further."
Program Highlights
PRGN-2012
AdenoVerse™ Immunotherapy in
RRP
- The Company announced that the US Food and Drug Administration
(FDA) has agreed that the ongoing Phase 1/2 single arm study of the
first-in-class investigational PRGN-2012 AdenoVerse™
immunotherapy for the treatment of recurrent respiratory
papillomatosis (RRP) will serve as pivotal for the purpose of
filing an accelerated approval request for licensure. The FDA also
confirmed no additional randomized, placebo-controlled trial will
be required to support submission of a biologics license
application (BLA). Based on the FDA guidance, the Company also
plans to initiate a confirmatory study prior to submission of
the BLA.
- 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 RRP.
- The Company completed enrollment and dosing in the Phase 2
portion of the study (N=23) bringing the total number of enrolled
patients to 35 at the recommended Phase 2 dose. Patient follow up
is currently ongoing and data collection is anticipated to be
completed by the second quarter of 2024.
- The Company announced that the FDA granted Breakthrough Therapy
Designation for PRGN-2012 for the treatment of RRP, adding to the
existing Orphan Drug Designation. The Breakthrough designation is
based on positive Phase 1 clinical data that showed that 50% of
adult RRP patients (who had ≥3 surgeries to treat the disease
in the year prior treatment) were "surgery-free" (Complete
Response) after PRGN-2012 treatment during the 12 month follow-up.
All complete responders continue to be surgery-free with a minimum
follow-up of 18 months post-treatment.
PRGN 2009
AdenoVerse™ Immunotherapy in
HPV-associated Cancers
- PRGN-2009 is an investigational off-the-shelf AdenoVerse
immunotherapy designed to activate the immune system to recognize
and target HPV-positive solid tumors.
- The Company completed the Phase 1 study and presented
positive Phase 1 clinical data from the monotherapy (N=6) and
combination therapy (N=11) arms in patients with recurrent or
metastatic HPV-associated cancers at the 2023 American Society of
Clinical Oncology (ASCO) Annual Meeting. PRGN-2009 was safe and
well-tolerated with only Grade 1 or 2 treatment related adverse
events and resulted in a 30% objective response rate (ORR) in the
combination arm in patients with heavily pre-treated HPV-associated
cancers that were naïve or resistant to checkpoint blockade with
prolonged duration of responses.
- Enrollment was completed in the Phase 2 monotherapy arm
with 20 evaluable patients in newly diagnosed oropharyngeal
squamous cell carcinoma (OPSCC) patients.
- The Company announced that the FDA has cleared the
Investigational New Drug (IND) application to initiate a Phase
2 study of PRGN-2009 in combination with pembrolizumab in patients
with recurrent or metastatic cervical cancer. The Phase 2
randomized, open-label, two-arm study will evaluate the efficacy
and safety of PRGN-2009 in combination with pembrolizumab
versus pembrolizumab monotherapy in patients with recurrent or
metastatic cervical cancer who are pembrolizumab resistant.
PRGN-3006 UltraCAR-T® in
AML
- PRGN-3006 is an investigational multigenic, autologous
chimeric antigen receptor T (CAR-T) cell therapy engineered to
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 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. PRGN-3006 was
well-tolerated with no dose-limiting toxicities. A single infusion
of autologous PRGN-3006 cells resulted in a 27% ORR in heavily
pre-treated relapsed or refractory AML patients infused following
lymphodepletion. A single infusion of UltraCAR-T cells with or
without lymphodepletion demonstrated robust expansion and
persistence in blood and bone marrow and PRGN-3006 infusion with
lymphodepletion resulted in a decrease in bone marrow blasts in 60%
of heavily pre-treated patients.
- The Phase 1b dose expansion study
of PRGN-3006 is ongoing and an interim 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 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 and presented positive Phase 1 clinical data in patients
with advanced platinum resistant ovarian cancer at the 2023 ASCO
Annual Meeting. PRGN-3005 was well-tolerated with no dose-limiting
toxicities, no cytokine release syndrome (CRS) greater than Grade
2, and no neurotoxicity. PRGN-3005 cells demonstrated expansion and
persistence when delivered via either IP or IV infusion without
lymphodepletion or via IV infusion after lymphodepletion. A single
IV infusion following lymphodepletion decreased tumor burden in 67%
of the heavily pretreated patients (median of 8 or more prior
therapies).
- The Phase 1b dose expansion study
of PRGN-3005 is ongoing.
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 Phase 1 dose escalation part of the Phase 1/1b study is ongoing. 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)).
Financial Highlights
- Completely retired the outstanding balance of convertible notes
in the second quarter.
- Cash, cash equivalents, short-term and long-term investments
totaled $95.6 million as of
June 30, 2023.
- Selling, general, and administrative (SG&A) costs decreased
versus the prior year, by 27% and 21% for the three and six months
ended June 30, 2023,
respectively.
Second Quarter 2023 Financial Results Compared to Prior Year
Period
Research and development expenses decreased
$0.1 million, or 0.7%, compared to
the three months ended June 30, 2022.
This decrease was primarily driven by reduced spending on
preclinical research programs.
SG&A expenses decreased $3.4
million, or 27%, compared to the three months ended
June 30, 2022. This decrease was
primarily driven by a reduction in professional fees of
$2.2 million, due to decreased legal
fees associated with certain litigation matters, as well as a
$1.1 million reduction in salaries,
benefits, and other personnel costs due to reduced head count.
Revenues decreased $1.1 million,
or 39%, compared to the three months ended June 30, 2022. This decrease was related to
reductions in services performed at Exemplar.
Total other income, net, increased $2.7
million compared to the three months ended June 30, 2023. This was primarily due to reduced
interest expense associated with the Company's convertible notes as
they were retired in the second quarter of 2023, and increased
interest income due to higher interest rates on investments.
Loss from continuing operations was $20.3
million, or $(0.08) per basic
and diluted share, compared to loss from continuing operations of
$26.1 million, or $(0.13) per basic and diluted share, in Q2
2022.
First Half 2023 Financial Results Compared to Prior Year
Period
Research and development expenses increased
$0.3 million, or 1.2%, compared to
the six months ended June 30, 2022.
This increase was primarily driven by a continued prioritization of
clinical product candidates, offset by reduced spending on
preclinical research programs.
SG&A expenses decreased $5.4
million, or 21%, compared to the six months ended
June 30, 2022. This decrease was
primarily driven by a reduction in professional fees of
$4.2 million, due to decreased legal
fees associated with certain litigation matters, as well as a
$1.1 million reduction in salaries,
benefits, and other personnel costs due to reduced head count.
Revenues decreased $4.8 million,
or 57.1%, compared to the six months ended June 30, 2022. This decrease was primarily
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 of $1.0 million at
Exemplar.
Total other income, net, increased $5.2
million compared to the six months ended June 30, 2022. This was primarily due to reduced
interest expense associated with the Company's convertible notes as
they were retired in the second quarter of 2023, and increased
interest income due to higher interest rates on investments.
Loss from continuing operations was $43.1
million, or $(0.18) per basic
and diluted share, compared to loss from continuing operations of
$50.0 million, or $(0.25) per basic and diluted share, in the six
months ended June 30, 2022. The 2023
second quarter loss was lower than the 2023 first quarter loss
primarily due to continued reductions in SG&A expenses.
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.
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.
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
anti-PDL1/TGF-Beta Trap (bintrafusp alfa) in patients with
HPV-associated cancers (NCT04432597), including oropharyngeal
squamous cell carcinoma (OPSCC), and a Phase 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. Additionally, the
FDA has cleared the IND to initiate a Phase 2 study of PRGN-2009
AdenoVerse immunotherapy in combination with pembrolizumab in
patients with recurrent or metastatic cervical cancer.
For patients interested in enrolling in NCI-led clinical
studies, please call NCI's toll-free number 1-800-4-Cancer
(1-800-422-6237) (TTY: 1-800-332-8615), email
NCIMO_Referrals@mail.nih.gov, and/or visit the website:
https://trials.cancer.gov.
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)
|
June 30,
2023
|
December 31,
2022
|
Assets
|
|
|
Current
assets
|
|
|
Cash
and cash equivalents
|
$
16,546
|
$
4,858
|
Restricted cash
|
-
|
43,339
|
Short-term investments
|
71,888
|
51,092
|
Receivables
|
|
|
Trade,
net
|
1,354
|
978
|
Other
|
13,052
|
12,826
|
Prepaid expenses and other
|
2,792
|
5,066
|
Total current assets
|
105,632
|
118,159
|
Long-term in investments
|
7,127
|
-
|
Property, plant and equipment, net
|
6,574
|
7,329
|
Intangible assets, net
|
42,656
|
44,455
|
Goodwill
|
36,966
|
36,923
|
Right-of-use assets
|
7,623
|
8,086
|
Other assets
|
949
|
1,025
|
Total assets
|
$ 207,527
|
$ 215,977
|
Liabilities and
Shareholders' Equity
|
|
|
Current
liabilities
|
|
|
Accounts payable
|
$
2,510
|
$
4,068
|
Accrued compensation and benefits
|
4,820
|
6,377
|
Other accrued liabilities
|
3,257
|
4,997
|
Settlement and
Indemnification Accrual
|
18,750
|
18,750
|
Deferred revenue
|
15
|
25
|
Current portion of long-term debt
|
-
|
43,219
|
Current portion of lease liabilities
|
1,421
|
1,209
|
Total current liabilities
|
30,773
|
78,645
|
Deferred revenue, net of current portion
|
1,818
|
1,818
|
Lease liabilities, net of current portion
|
6,545
|
6,992
|
Deferred tax liabilities
|
2,181
|
2,263
|
Total liabilities
|
41,317
|
89,718
|
Shareholders'
equity
|
|
|
Common stock
|
-
|
-
|
Additional paid-in capital
|
2,080,348
|
1,998,314
|
Accumulated deficit
|
(1,911,620)
|
(1,868,567)
|
Accumulated other comprehensive loss
|
(2,518)
|
(3,488)
|
Total shareholders' equity
|
166,210
|
126,259
|
Total liabilities and shareholders' equity
|
$
207,527
|
$
215,977
|
Precigen, Inc. and
Subsidiaries
Consolidated
Statement of Operations
|
(Unaudited)
|
|
Three months
ended
|
Six months
ended
|
(Amounts in
thousands, except share and per share data)
|
June 30,
2023
|
June 30,
2022
|
June 30,
2023
|
June 30,
2022
|
Revenues
|
|
|
|
|
Product
revenues
|
$
324
|
$
621
|
$
648
|
$
1,113
|
Service
revenues
|
1,438
|
2,213
|
2,965
|
7,146
|
Other
revenues
|
5
|
77
|
5
|
165
|
Total
revenues
|
1,767
|
2,911
|
3,618
|
8,424
|
Operating
Expenses
|
|
|
|
|
Cost of products and
services
|
1,697
|
1,811
|
3,224
|
3,505
|
Research and
development
|
11,874
|
11,954
|
24,037
|
23,755
|
Selling, general and
administrative
|
9,316
|
12,670
|
20,954
|
26,359
|
Impairment of
goodwill
|
-
|
-
|
-
|
482
|
Impairment of other
noncurrent assets
|
-
|
638
|
-
|
638
|
Total operating
expenses
|
22,887
|
27,073
|
48,215
|
54,739
|
Operating
loss
|
(21,120)
|
(24,162)
|
(44,597)
|
(46,315)
|
Other Expense,
Net
|
|
|
|
|
Interest
expense
|
(136)
|
(2,063)
|
(460)
|
(4,101)
|
Interest
income
|
828
|
37
|
1,460
|
75
|
Other income,
net
|
44
|
40
|
424
|
238
|
Total other income
(expense), net
|
736
|
(1,986)
|
1,424
|
(3,788)
|
Equity in net loss of
affiliates
|
-
|
-
|
-
|
(1)
|
Loss from continuing
operations before
income
taxes
|
(20,384)
|
(26,148)
|
(43,173)
|
(50,104)
|
Income tax
benefit
|
65
|
89
|
120
|
147
|
Loss from continuing
operations
|
$
(20,319)
|
$
(26,059)
|
$
(43,053)
|
$
(49,957)
|
Income from
discontinued operations, net of income taxes
|
-
|
8,424
|
-
|
13,071
|
Net loss
|
$
(20,319)
|
$
(17,635)
|
$
(43,053)
|
$
(36,886)
|
Net Loss per
share
|
|
|
|
|
Net loss from
continuing operations per share, basic and diluted
|
$
(0.08)
|
$
(0.13)
|
$
(0.18)
|
$
(0.25)
|
Net income from
discontinued operations per share, basic and diluted
|
-
|
0.04
|
-
|
0.07
|
Net loss per share,
basic and diluted
|
$
(0.08)
|
$
(0.09)
|
$
(0.18)
|
$
(0.18)
|
Weighted average shares
outstanding, basic and diluted
|
248,003,322
|
200,461,441
|
240,307,403
|
200,047,629
|
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SOURCE Precigen, Inc.