- Allogeneic CAR T Program Updates Planned for
Late Q4 2022 or Early Q1 2023
- Abstract Showcasing Functional Attributes of
Azer-cel (PBCAR1091) Accepted for Presentation at the 64th American
Society of Hematology (ASH) Annual Meeting
- Preclinical Research on In Vivo Gene Editing
Programs Presented at the European Society of Gene & Cell
Therapy (ESGCT) 29th Congress
- Strong Cash Position Provides More than Two
Years of Expected Runway
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS®-based ex vivo allogeneic CAR
T and in vivo gene editing therapies, today announced financial
results for the third quarter ended September 30, 2022 and provided
a business update.
“Over the last year, Precision has made considerable progress
against our corporate and development objectives. We executed a
disciplined portfolio strategy to focus on human therapeutics,
advanced our lead clinical stage CAR T programs, leveraged platform
partnerships, and made data-informed decisions designed to maximize
patient impact and extend our expected cash runway to the end of
2024,” said Michael Amoroso, Chief Executive Officer of Precision
BioSciences. “We have refined the scope of our CAR T portfolio,
focusing on PBCAR0191, known as azer-cel, in the growing CAR
T-relapsed population, which has shown high complete response
rates, long-term responses, and peak CAR T expansion on par with
autologous CAR T in durable responders. We are also exploring the
potential of our immune-evading stealth cell technology with
PBCAR19B in earlier line DLBCL patients with the goal of displacing
autologous CAR T treatment options. Looking ahead, our team is
focused on executing and advancing our clinical stage CD19 targeted
CAR T programs and providing a clinical update in late Q4 2022 or
early Q1 2023, depending on patient accrual and follow-up.”
Mr. Amoroso continued, “We also advanced multiple in vivo gene
editing programs, independently and with partners, highlighted by
our research collaboration with Novartis in June and recent
preclinical data shared at ESGCT. As a result of ARCUS’
versatility, recent high efficiency in vivo gene insertion data,
and scientific progress with wholly-owned and partnered programs,
we have commenced a portfolio review of our in vivo gene editing
programs. We are exploring ways to expedite key programs, advance
new indications, and maximize ARCUS’ core features which have the
potential to enable high efficiency gene insertion and complex
edits aimed at restoring genomic function and treating the
underlying root cause of specific genetic diseases. Our aim is to
focus on programs in which ARCUS is most differentiated and where
we see potential for a clear and rapid path to market, while
providing a potentially curative therapeutic solution to patients
with the highest unmet need. We look forward to providing
meaningful progress updates along the way.”
Recent Developments and Upcoming Milestones:
Ex Vivo Allogeneic CAR T Portfolio:
PBCAR0191: PBCAR0191, azercabtagene zapreleucel
(azer-cel), is Precision’s lead investigational anti-CD19
allogeneic CAR T candidate in a Phase 1/2a clinical trial of adult
subjects with relapsed or refractory (R/R) non-Hodgkin lymphoma
(NHL). An abstract on the cell dose and functional attributes of
azer-cel that may be associated with positive safety and efficacy
results for CAR T therapy in R/R B-cell lymphoma was accepted for
poster presentation at the 64th ASH Annual Meeting taking place
December 10-13, 2022. The poster presentation will highlight the
first analysis of an allogeneic CD19 CAR T product composition to
demonstrate that strategies intended to maximize stem central
memory T-cell fraction (CCR7+) while limiting CD4+ CCR7+
differentiated fraction may improve safety and efficacy of CAR T
therapy.
Precision continues dosing subjects with optimized azer-cel CAR
T cells in the CAR T relapsed population in which it has shown high
and durable complete response rates, while further reducing the
dose of lymphodepletion to standard levels in pursuit of best
therapeutic index for this patient population.
PBCAR19B: PBCAR19B is Precision’s second generation,
anti-CD19 targeting allogeneic CAR T candidate designed to evade
immune rejection by host T cell and natural killer (NK) cells with
a single-gene edit to knock-down beta-2 microglobulin and insert an
HLA-E transgene. Precision continues to recruit patients at Dose
Level 2 (flat dose of 540 million cells) with the intent to
complete the Phase 1 dose escalation.
Precision expects to provide an update in late Q4 2022 or early
Q1 2023, depending on patient accrual and follow-up, on its two
distinct CD19 targeted products, including azer-cel which is aiming
to achieve a potential first-in-class allogeneic CAR T therapy in
the CAR T relapsed population, and PBCAR19B which is seeking to
displace autologous CAR T in the second/third line DLBCL
population.
PBCAR269A + GSI: PBCAR269A is Precision’s investigational
allogeneic CAR T cell candidate targeting B-cell maturation antigen
(BCMA) for R/R multiple myeloma in combination with nirogacestat, a
gamma secretase inhibitor (GSI) developed by SpringWorks
Therapeutics, Inc. The combination therapy and increased dose of
PBCAR269A resulted in improved cell expansion, which correlated
with increased clinical activity when compared to dose-matched
PBCAR269A monotherapy treatment. However, in light of the
competitive landscape of BCMA targeted therapies in multiple
myeloma, Precision has made the strategic decision not to continue
the PBCAR269A clinical program. All subjects enrolled in the study
and evaluated for treatment with PBCAR269A and nirogacestat had
acceptable tolerability results. While no clinical spending is
planned, Precision researchers will evaluate further modifications
to the BCMA construct aimed at enabling an allogeneic approach
similar to that of autologous CAR T in multiple myeloma. Precision
thanks the patients and clinicians for their participation in the
PBCAR269A clinical program.
In Vivo Gene Editing Portfolio:
Precision believes that in vivo applications are particularly
well suited to ARCUS because they require extremely low levels of
off-target editing and efficient delivery. As a gene editing tool,
ARCUS can be differentiated by unique attributes which are designed
for precise, specific and versatile gene editing. By nature of its
origin from a homing endonuclease, ARCUS can be particularly
applicable to gene insertion and complex edits designed for gene
repair aimed at restoring function, as well as more simple gene
knock outs. ARCUS is also unique in its relatively small size which
allows delivery to a wider range of cells and tissues using viral
and non-viral gene delivery methods.
Novartis In Vivo Gene Editing Collaboration Precision is
advancing its gene editing research and development collaboration
and license agreement with Novartis to develop a single, custom
ARCUS nuclease designed to insert a therapeutic transgene, in vivo,
at a “safe harbor” location in the genome. This has the potential
to be a one-time transformative treatment option for diseases
including certain hemoglobinopathies such as sickle cell disease
and beta thalassemia. In conjunction with the close of the
agreement, Novartis made a $25 million equity investment in
Precision in the second quarter of 2022 and Precision received $50
million in cash in the third quarter of 2022.
Lilly In Vivo Gene Editing Collaboration: Precision
continues its in vivo gene editing collaboration with Lilly and is
applying ARCUS nucleases for three initial targets, including
Duchenne muscular dystrophy in muscle, a central nervous system
directed target and a liver directed target.
PBGENE-HBV: Precision’s gene editing program for chronic
Hepatitis B applies ARCUS to knock out persistent covalently closed
circular DNA (cccDNA) and inactivate integrated hepatitis B
genomes, potentially achieving durable HBV S-antigen (HBsAg) loss
and reducing viral persistence. Preclinical data from this program
were presented during ESGCT in October 2022. Data presented
demonstrated that ARCUS efficiently targeted and degraded hepatitis
B virus (HBV) cccDNA by 85% and reduced expression of HBsAg by 77%
in HBV-infected primary human hepatocytes (PHH). Importantly, the
optimized specificity of the ARCUS nuclease completely prevented
detectable chromosomal translocations in the PHH model.
PBGENE-PH1: Precision has initiated IND-enabling
activities for its PBGENE-PH1 candidate designed to knock out the
HAO1 gene as a potential one-time treatment for primary
hyperoxaluria type 1 (PH1).
PBGENE-PCSK9: In 2021, Precision initiated a
collaboration with iECURE, pursuant to which iECURE is expected to
advance Precision’s PBGENE-PCSK9 candidate through preclinical
activities as well as a Phase 1 study in familial
hypercholesterolemia. As of this date, IND enabling activities for
PBGENE-PCSK9 have not been completed. Precision is in discussions
with iECURE and will provide an update on the program when more
information is available.
Other ARCUS Research:
International Conference on Ureagenesis Defects and Allied
Conditions 20221: Preclinical data were presented by
researchers from the University of Pennsylvania’s Gene Therapy
Program in collaboration with iECURE, Precision’s partner,
highlighting an ARCUS-based gene insertion approach for the
treatment of ornithine transcarbamylase (OTC) deficiency. Non-human
primate (NHP) data demonstrated stable insertion of the therapeutic
gene one year post-dosing in newborn and infant NHPs In the follow
up data, 12-month biopsies continued to demonstrate construct
stability, with transduction efficiency up to 28.2% as measured by
in-situ hybridization (ISH). These data further demonstrate the
preclinical feasibility of using an ARCUS-mediated gene insertion
approach.
ESGCT 29th Congress: Additional abstracts on ARCUS in
vivo gene editing were presented in addition to Precision’s HBV
program, including one poster presentation each on Precision’s
Apolipoprotein C3 and mitochondrial DNA preclinical research.
- APOC3 poster presentation: ARCUS gene editing of Apolipoprotein
C3 results in substantial reduction in serum triglycerides in
vivo
- Mito DNA poster presentation: Specific elimination of
m.3243A>G mutant mitochondrial DNA using mitoARCUS in cultured
cells and a novel xenograft mouse model
Quarter Ended September 30, 2022
Financial Results:
Cash and Cash Equivalents: As of September 30, 2022,
Precision had approximately $212.1 million in cash and cash
equivalents. The Company expects that existing cash and cash
equivalents, expected operational receipts, and available credit
will be sufficient to fund its operating expenses and capital
expenditure requirements to the end of 2024.
Revenues: Total revenues for the quarter ended September
30, 2022 were $7.4 million, as compared to $24.0 million for the
same period in 2021. The decrease of $16.6 million in revenue
during the quarter ended September 30, 2022 was primarily the
result of the absence of $17.9 million in revenue recognized under
the iECURE Agreement in August 2021 subsequent to the full
satisfaction of the performance obligation. These decreases in
revenue were partially offset by an increase of $3.6 million in
revenue recognized under the Novartis Agreement.
Research and Development Expenses: Research and
development expenses were $20.0 million for the quarter ended
September 30, 2022, as compared to $25.9 million for the same
period in 2021. The decrease of $5.9 million was primarily due to a
decrease of $3.6 million in external development costs associated
with our allogeneic CAR T product candidates, a decrease of $0.9
million in employee-related and other operational costs driven by
the separation of Elo in 2021, and a decrease of $1.4 million in
clinical manufacturing organization and research costs related to
our preclinical studies.
General and Administrative Expenses: General and
administrative expenses were $10.3 million for the quarter ended
September 30, 2022, as compared to $9.6 million for the same period
in 2021. The increase of $0.7 million was primarily due to
increased share-based compensation expense.
Other Income and Expense: Total other expense was $1.0
million for the quarter ended September 30, 2022, as compared to
total other income of $0.3 million for the same period in 2021.
Net Loss: Net loss was $23.9 million, or $(0.22) per
share (basic and diluted), for the quarter ended September 30,
2022, as compared to net loss of $11.3 million, or $(0.19) per
share (basic and diluted), for the same period in 2021. Weighted
average shares of common stock outstanding were approximately 110.8
million for the quarter ended September 30, 2022, as compared to
approximately 59.7 million for the quarter ended September 30,
2021. The increase in weighted average shares of common stock
outstanding was primarily due to a $50 million underwritten
offering of common stock and Novartis’ $25 million equity
investment in the nine months ended September 30, 2022.
Corporate:
Executive Leadership: In September 2022, Cindy Atwell,
formerly Senior Vice President of Business Development and Alliance
Management, was promoted to Chief Business Officer and continues to
oversee the Business Development and Alliance functions with added
responsibility for Project and Portfolio Management. Jeff Smith,
Ph.D., co-founder and formerly Chief Technology Officer, was
promoted to Chief Research Officer and assumed responsibility for
the management and direction of the Company’s research programs,
reporting directly to the CEO. Derek Jantz, Ph.D., co-founder and
Chief Scientific Officer is focusing his time partnering with
Michael Amoroso in formulating company strategy and managing
relationships with external stakeholders, including current and
potential collaboration partners.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology
company dedicated to improving life (DTIL) with its novel and
proprietary ARCUS® genome editing platform. ARCUS is a highly
precise and versatile genome editing platform that was designed
with therapeutic safety, delivery, and control in mind. Using
ARCUS, the company’s pipeline consists of multiple ex vivo
“off-the-shelf” CAR T immunotherapy clinical candidates and several
in vivo gene editing candidates designed to cure genetic and
infectious diseases where no adequate treatments exist. For more
information about Precision BioSciences, please visit
www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including, without limitation,
statements regarding the clinical development and expected efficacy
and benefit of our product candidates and programs, the expected
timing of updates regarding programs in our allogeneic CAR T and in
vivo portfolio and ARCUS research, planned development activities
with our collaboration partners, expectations about our operational
initiatives, our business strategy and portfolio review and
expectations regarding our liquidity and capital resources. In some
cases, you can identify forward-looking statements by terms such as
“aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,”
“estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,”
“plan,” “possible,” “potential,” “predict,” “project,” “pursue,”
“should,” “target,” “will,” “would,” or the negative thereof and
similar words and expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. Such statements are subject to a number of known
and unknown risks, uncertainties and assumptions, and actual
results may differ materially from those expressed or implied in
the forward-looking statements due to various important factors,
including, but not limited to: our ability to become profitable;
our ability to procure sufficient funding and requirements under
our current debt instruments and effects of restrictions
thereunder; risks associated with raising additional capital; our
operating expenses and our ability to predict what those expenses
will be; our limited operating history; the success of our programs
and product candidates in which we expend our resources; our
limited ability or inability to assess the safety and efficacy of
our product candidates; our dependence on our ARCUS technology; the
initiation, cost, timing, progress, achievement of milestones and
results of research and development activities, preclinical studies
and clinical trials; public perception about genome editing
technology and its applications; competition in the genome editing,
biopharmaceutical, and biotechnology fields; our or our
collaborators’ ability to identify, develop and commercialize
product candidates; pending and potential liability lawsuits and
penalties against us or our collaborators related to our technology
and our product candidates; the U.S. and foreign regulatory
landscape applicable to our and our collaborators’ development of
product candidates; our or our collaborators’ ability to obtain and
maintain regulatory approval of our product candidates, and any
related restrictions, limitations and/or warnings in the label of
an approved product candidate; our or our collaborators’ ability to
advance product candidates into, and successfully design, implement
and complete, clinical or field trials; potential manufacturing
problems associated with the development or commercialization of
any of our product candidates; our ability to obtain an adequate
supply of T cells from qualified donors; our ability to achieve our
anticipated operating efficiencies at our manufacturing facility;
delays or difficulties in our and our collaborators’ ability to
enroll patients; changes in interim “top-line” and initial data
that we announce or publish; if our product candidates do not work
as intended or cause undesirable side effects; risks associated
with applicable healthcare, data protection, privacy and security
regulations and our compliance therewith; the rate and degree of
market acceptance of any of our product candidates; the success of
our existing collaboration agreements, and our ability to enter
into new collaboration arrangements; our current and future
relationships with and reliance on third parties including
suppliers and manufacturers; our ability to obtain and maintain
intellectual property protection for our technology and any of our
product candidates; potential litigation relating to infringement
or misappropriation of intellectual property rights; our ability to
effectively manage the growth of our operations; our ability to
attract, retain, and motivate key executives and personnel; market
and economic conditions; effects of system failures and security
breaches; effects of natural and manmade disasters, public health
emergencies and other natural catastrophic events; effects of
COVID-19 pandemic and variants thereof, or any pandemic, epidemic
or outbreak of an infectious disease; insurance expenses and
exposure to uninsured liabilities; effects of tax rules; risks
related to ownership of our common stock and other important
factors discussed under the caption “Risk Factors” in our Quarterly
Report on Form 10-Q for the quarterly period ended June 30, 2022,
as any such factors may be updated from time to time in our other
filings with the SEC, including, but not limited to, our Quarterly
Report on Form 10-Q for the quarterly period ended September 30,
2022, to be filed with the SEC, which are accessible on the SEC’s
website at www.sec.gov and the Investors page of our website under
SEC Filings at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
Precision BioSciences,
Inc.
Condensed Consolidated
Statements of Operations
(In thousands, except share and
per share amounts)
(unaudited)
For the Three Months Ended
September 30,
2022
2021
Revenue
$
7,363
$
24,036
Operating expenses
Research and development
19,959
25,940
General and administrative
10,334
9,638
Total operating expenses
30,293
35,578
Operating loss
(22,930
)
(11,542
)
Other (expense) income:
Change in fair value of equity
investment
—
274
Loss from equity method investment
(1,783
)
—
Interest expense
(405
)
(55
)
Interest income
1,172
44
Total other (expense) income, net
(1,016
)
263
Net loss
$
(23,946
)
$
(11,279
)
Net loss per share - basic and diluted
$
(0.22
)
$
(0.19
)
Weighted average shares of common stock
outstanding - basic and diluted
110,849,196
59,657,677
For the Nine Months Ended
September 30,
2022
2021
Revenue
$
14,500
$
109,190
Operating expenses
Research and development
62,867
88,768
General and administrative
31,510
29,074
Total operating expenses
94,377
117,842
Operating loss
(79,877
)
(8,652
)
Other (expense) income:
Change in fair value of equity
investment
—
274
Loss from equity method investment
(4,183
)
—
Interest expense
(625
)
(79
)
Interest income
1,536
145
Total other (expense) income, net
(3,272
)
340
Net loss
$
(83,149
)
$
(8,312
)
Net loss per share - basic and diluted
$
(1.04
)
$
(0.14
)
Weighted average shares of common stock
outstanding - basic and diluted
80,127,701
58,018,550
Precision BioSciences,
Inc.
Condensed Consolidated Balance
Sheets Data
(In thousands, except share
amounts)
(Unaudited)
September 30, 2022
December 31, 2021
Cash and cash equivalents
$
212,051
$
143,663
Working capital
164,217
125,774
Total assets
271,733
211,498
Total liabilities
187,353
120,330
Total stockholders' equity
$
84,380
$
91,168
Common stock outstanding
110,934,747
60,902,105
1 University of Pennsylvania’s Gene
Therapy Program presentation sponsored by iECURE. iECURE has a
license to use of ARCUS for gene insertion for OTC.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221108005127/en/
Investor Contact: Mei Burris Director, Investor Relations
and Finance Mei.Burris@precisionbiosciences.com
Media Contact: Maurissa Messier Senior Director,
Corporate Communications
Maurissa.Messier@precisionbiosciences.com
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