- Precision to present late breaker PBGENE-HBV
data at The Liver Meeting of AASLD in November 2023
- Hosted R&D Day highlighting exclusive
focus on in vivo gene editing pipeline
- Expanded portfolio with PBGENE-PMM as a
potentially curative treatment for m.3243 associated primary
mitochondrial myopathy
- Completed strategic transaction with Imugene
for azer-cel in cancer
- Extended cash runway through the end of
2025
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene
editing company utilizing its novel proprietary ARCUS® platform to
develop in vivo gene editing therapies for sophisticated gene
edits, including gene insertion, excision, and elimination, today
announced financial results for the third quarter ended September
30, 2023 and provided a business update.
“The ARCUS platform has always been foundational to Precision,
as we have harnessed its distinct properties in pursuit of life
changing medicines for patients. Our recent transaction with
Imugene allows Precision to focus the power of ARCUS on in vivo
gene editing while capturing near-term and potential long-term
value for our CAR T assets,” said Michael Amoroso, Chief Executive
Officer at Precision BioSciences. “At our recent R&D Day, we
featured data demonstrating how the differentiated cut, size, and
simplicity of ARCUS enable it to uniquely carry out sophisticated
edits that present clear advantages in the market and compelling
therapeutic opportunities versus other editing technologies. By
design, ARCUS has been shown to preferentially drive higher
efficiency gene insertion than CRISPR-derived editors, and its ease
of delivery and simplicity confer greater utility in reaching
tissues, organs, and organelles for gene insertion, excision and
elimination. Going forward, Precision will solely focus its own
efforts on programs leveraging ARCUS’ specific utility for
sophisticated edits. To that end, we are progressing wholly-owned
PBGENE-HBV and PBGENE-PMM programs towards anticipated CTA and/or
IND filings in 2024 and 2025, respectively. In addition, the ARCUS
OTC gene insertion program being developed by our partner at iECURE
is targeting a CTA and/or IND filing by the end of this year.
Importantly, as we execute against our singular focused in vivo
strategy, we believe we will have sufficient capital to advance
these wholly-owned programs into the clinic and to Phase 1 data
points over the next two years.”
Completed Strategic Transaction with
Imugene for Azer-Cel in Cancer
In August 2023, the Company completed a strategic transaction
with Imugene Limited (ASX: IMU) for the Company’s lead allogeneic
chimeric antigen receptor (CAR) T candidate for cancer,
azercabtagene zapreleucel (azer-cel). In exchange for global rights
to azer-cel for cancer, as well as Precision’s CAR T infrastructure
and experienced cell therapy teams, Precision received upfront cash
and equity consideration valued at $21 million. In addition,
Precision is eligible for a potential $8 million near-term
milestone payment, up to $198 million in additional milestone
payments and double-digit royalties on net sales of azer-cel, as
well as $145 million in milestone payments and tiered royalties for
up to three additional research programs to be potentially
developed by Imugene. Imugene has assumed ongoing clinical
execution for azer-cel in the large B-cell lymphoma population who
have relapsed following autologous CAR T treatment.
Advancing as a Single Platform Company
Focused on ARCUS In Vivo
Gene Editing
Precision is now solely focused on leveraging its proprietary
ARCUS genome editing platform to advance in vivo gene editing
programs that go beyond gene knockouts in the liver and carry out
more sophisticated edits such as gene insertions, gene excision,
and gene elimination unlocking a broader potential for ARCUS in
vivo gene editing in human therapeutics.
In support of the go-forward strategy, Precision presented two
poster presentations at the European Society of Gene & Cell
Therapy congress on October 25 and 26, 2023, in Brussels, Belgium,
“Unique features of ARCUS nucleases enable high efficiency,
targeted gene insertion in vivo” and “ARCUS-mediated excision of
the “hot spot” region of the human dystrophin gene results in
functional improvement in a mouse model of Duchenne muscular
dystrophy (DMD).”
Wholly-owned Portfolio
PBGENE-HBV (Viral Elimination Program): Precision is
developing PBGENE-HBV for the treatment of patients with chronic
hepatitis B and expects to submit a clinical trial application
(CTA) and/or investigational new drug (IND) application in
2024.
Chronic infection with hepatitis B virus (HBV) is due to
persistence of the viral genome, in the form of covalently closed
circular DNA (cccDNA) and viral sequences integrated into the human
genome. Current treatments for chronic hepatitis B rarely achieve a
functional cure, defined as sustained undetectable levels of
circulating hepatitis B surface antigen (HBsAg) and HBV DNA after a
finite course of treatment, as they do not eliminate the root cause
of viral persistence. In September 2023, at its R&D Day, the
Company shared data highlighting the potential of PBGENE-HBV to
drive sustained reductions of circulating HBsAg and HBV DNA. Unlike
other therapies in development, PBGENE-HBV directly eliminates
cccDNA and inactivates integrated viral sequences, representing a
potentially curative approach.
A late-breaking abstract featuring preclinical data on the
PGBENE-HBV program has been accepted for a poster presentation,
“Preclinical efficacy and safety of ARCUS-POL nucleases for chronic
hepatitis B: a potentially curative strategy,” at the American
Association for the Study of Liver Diseases (AASLD) Annual Meeting
being held in Boston on November 10-14, 2023.
PBGENE-PMM (Mutant Mitochondrial Elimination Program): At
its R&D Day, Precision announced that it will pursue
development of PBGENE-PMM as a potential first-in-class opportunity
for treatment of m.3243-associated primary mitochondrial myopathy
(PMM). Mitochondrial diseases are the most common hereditary
metabolic disorder, affecting 1 in 4,300 people. PMM currently
lacks a curative treatment and impacts approximately 50% of
patients with mitochondrial disease. The high specificity and
simplistic, single component nature of Precision’s mitoARCUS
nucleases are designed to enable specific editing to eliminate
mutant mitochondrial DNA while allowing normal (wild-type)
mitochondrial DNA to repopulate in the mitochondria and restore
normal function. Precision is targeting to submit a CTA and/or IND
in 2025 for PBGENE-PMM.
In September 2023, Precision received a Notice of Allowance from
the U.S. Patent and Trademark Office (USPTO) for U.S. Patent
Application No. 18/161,560, titled “Engineered Meganucleases That
Target Human Mitochondrial Genomes.” The allowed composition of
matter claims encompass a mitochondria-targeted ARCUS nuclease
(mitoARCUS) that is designed to specifically target, cleave, and
eliminate mutant mitochondrial DNA comprising an m.3243A>G
mutation.
Partnered Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, an
ARCUS-mediated gene insertion approach is being pursued as a
potential treatment for neonatal onset ornithine transcarbamylase
(OTC) deficiency. Non-human primate (NHP) data presented in October
2022 by researchers from the University of Pennsylvania’s Gene
Therapy Program demonstrated sustained gene insertion of a
therapeutic OTC transgene one-year post-dosing in newborn and
infant NHPs with high efficiency. iECURE expects to submit a CTA
and/or IND in the second half of 2023.
PBGENE-NVS (Gene Insertion Program): Precision continues
to advance its in vivo gene editing program with Novartis to
develop a custom ARCUS nuclease for patients with
hemoglobinopathies, such as sickle cell disease and beta
thalassemia. The collaborative intent is to insert, in vivo, a
therapeutic transgene into hematopoietic stem cells as a potential
one-time transformative treatment administered directly to the
patient that would overcome many of the hurdles present today with
other therapeutic technologies, including ex vivo gene editing
approaches.
PBGENE-DMD (Gene Excision Program): Precision continues
its in vivo gene editing collaboration with Prevail Therapeutics, a
wholly-owned subsidiary of Eli Lilly and Company, in applying ARCUS
nucleases to three initial targets, including DMD in muscle, a
liver-directed target, and a central nervous system-directed
target. The goal of the PBGENE-DMD program is to utilize a pair of
ARCUS nucleases, delivered by a single AAV, that are designed to
excise an approximately 500,000 base pair mutation “hot spot”
region from the dystrophin gene to generate a variant of the
dystrophin protein that is functionally competent. During its
September 2023 R&D Day, the Company highlighted preclinical
data demonstrating the potential of ARCUS in vivo gene editing for
large gene excisions and that the edited dystrophin variant was
observed in multiple tissue types frequently involved in
progression of DMD, including skeletal muscle, heart, and
diaphragm, thereby enabling significantly improved muscle
function.
Quarter Ended September 30, 2023
Financial Results:
In August 2023, the Company announced its final stages of
execution to operate as a single platform company focused
exclusively on developing in vivo gene editing therapies with the
completion of the strategic transaction with Imugene for azer-cel
for cancer. Precision determined that the decision to only pursue
development of CAR T cell therapies through partnerships qualified
for discontinued operations accounting treatment. Accordingly, the
accompanying financial statements for all periods presented reflect
Precision’s CAR T cell therapy business as a discontinued
operation.
Cash and Cash Equivalents: As of September 30, 2023,
Precision had approximately $122.2 million in cash and cash
equivalents. The Company expects that existing cash and cash
equivalents, expected operational receipts, including upfront
consideration received from Imugene, operational efficiencies
gained from divestment of the CAR T business, availability of the
ATM facility, and available credit will be sufficient to fund its
operating expenses and capital expenditure requirements through the
end of 2025. The Company expects its cash runway to be sufficient
to achieve first-in-human Phase 1 clinical data for its lead in
vivo gene editing programs.
Revenues: Total revenues for the quarter ended September
30, 2023 were $13.1 million, as compared to $7.4 million for the
same period in 2022. The increase of $5.7 million in revenue during
the quarter ended September 30, 2023 was primarily the result of an
increase of $4.0 million in revenue recognized under the Novartis
Agreement and an increase of $1.7 million in revenue recognized
under the Prevail Agreement.
Research and Development Expenses: Research and
development expenses were $15.9 million for the quarter ended
September 30, 2023, as compared to $11.8 million for the same
period in 2022. The increase of $4.1 million was primarily due to
$5.1 million in expenses related to initiating CTA/IND-enabling
studies for the PBGENE-HBV development program planned for CTA/IND
submission in 2024, offset by decreases in laboratory supplies and
services and share-based compensation expense. R&D expenses in
the quarter ended September 30, 2023 are not necessarily indicative
of future spend as work may fluctuate quarter to quarter in the
pre-CTA/IND phase.
General and Administrative Expenses: General and
administrative expenses were $9.6 million for the quarter ended
September 30, 2023, as compared to $10.3 million for the same
period in 2022. The decrease of $0.7 million was primarily due to a
decrease in share-based compensation expense offset by an increase
in information technology expenses.
Interest Income/Expense: Interest expense was $0.6
million for the quarter ended September 30, 2023 compared to $0.4
million for the same period in 2022, primarily due to higher
interest rates on our debt.
Interest income was $1.9 million during the quarter ended
September 30, 2023 compared to $1.2 million for the same period in
2022. The increase in interest income was primarily the result of
higher interest rates on our cash and cash equivalents compared to
the three months ended September 30, 2022.
Discontinued Operations: Income from discontinued
operations was $4.0 million during the three months ended September
30, 2023 compared to an $8.3 million loss during the three months
ended September 30, 2022. The $12.3 million increase was the result
of the $8.4 million gain on sale of our CAR T infrastructure to
Imugene and a $3.9 million decrease in cell therapy expenses during
the three months ended September 30, 2023 as compared to the three
months ended September 30, 2022.
Net Loss: Net loss was $8.1 million, or $(0.07) per share
(basic and diluted), including $4.0 million income from
discontinued operations for the quarter ended September 30, 2023.
Net loss was $23.9 million, or $(0.22) per share (basic and
diluted), including a $8.3 million loss from discontinued
operations for the same period in 2022.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company
dedicated to improving life (DTIL) with its novel and proprietary
ARCUS® genome editing platform that differs from other technologies
in the way it cuts, its smaller size, and its simpler structure.
Key capabilities and differentiating characteristics may enable
ARCUS nucleases to drive more intended, defined therapeutic
outcomes. Using ARCUS, the Company’s pipeline is comprised of in
vivo gene editing candidates designed to deliver lasting cures for
the broadest range of 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 safety,
efficacy and benefit of our product candidates and gene editing
approaches including ARCUS’s potential editing efficiency and
differentiating aspects and the ability for the ARCUS genome
editing platform to develop differentiated programs; the
suitability of ARCUS nucleases for gene or viral elimination, large
excision, gene insertion, and other complex gene editing approaches
to drive defined outcomes; the potential of PBGENE-HBV to drive
sustained reductions of circulating HBsAg and HBV DNA; the expected
timing of regulatory processes, including timing of expected CTA
and/or IND filings; expectations about our operational initiatives
and business strategy; expectations around the Company’s
partnerships; expectations about achievement of key milestones; and
expectations regarding the Company’s cash runway, including the
assumptions underlying the Company’s calculations of its cash
runway and whether its cash runway will be sufficient to provide
first-in-human clinical data for the Company’s research and
development programs. The words “aim,” “anticipate,” “approach,”
“believe,” “contemplate,” “could,” “designed”, “estimate,”
“expect,” “explore,” “goal,” “intend,” “look,” “may,” “mission,”
“plan,” “possible,” “potential,” “predict,” “project,” “promise,”
“pursue,” “seek” “should,” “target,” “will,” “would,” and other
similar words or expressions, or the negative of these words or
similar words or expressions, are intended to identify
forward-looking statements, though not all forward-looking
statements use these words or expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. These statements are neither promises nor
guarantees, but involve 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 to advance our programs; risks
associated with our capital requirements, anticipated cash runway,
requirements under our current debt instruments and effects of
restrictions thereunder, including our ability to raise additional
capital due to market conditions and/or our market capitalization;
our operating expenses and our ability to predict what those
expenses will be; our limited operating history; the progression
and 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; the risk that
other genome-editing technologies may provide significant
advantages over our ARCUS technology; our dependence on our ARCUS
technology; the initiation, cost, timing, progress, achievement of
milestones and results of research and development activities and
preclinical and clinical studies, including clinical trial and
investigational new drug applications; 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 product 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 advance product candidates into, and successfully
design, implement and complete, clinical or field trials; 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; our ability to obtain
orphan drug designation or fast track designation for our product
candidates or to realize the expected benefits of these
designations; 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; the rate and degree of market
acceptance of any of our product candidates; our ability to
effectively manage the growth of our operations; our ability to
attract, retain, and motivate executives and personnel; effects of
system failures and security breaches; insurance expenses and
exposure to uninsured liabilities; effects of tax rules; effects of
any pandemic, epidemic, or outbreak of an infectious disease; 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; effects of
natural and manmade disasters, public health emergencies and other
natural catastrophic events; effects of sustained inflation, supply
chain disruptions and major central bank policy actions; market and
economic conditions; risks related to ownership of our common
stock, including fluctuations in our stock price, 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, 2023, as any such factors may be updated from time to time in
our other filings with the Securities and Exchange Commission
(“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 Statements of
Operations (In thousands, except share and per share amounts)
(unaudited)
For the Three Months Ended September 30,
2023
2022
Revenue
$
13,120
$
7,363
Operating expenses Research and development
15,850
11,758
General and administrative
9,633
10,281
Total operating expenses
25,483
22,039
Operating loss
(12,363
)
(14,676
)
Other income (expense): Loss from equity method investment
(1,350
)
(1,782
)
Gain on changes in fair value
311
—
Interest expense
(576
)
(405
)
Interest income
1,870
1,172
Loss on disposal of assets
(2
)
—
Total other income (expense)
253
(1,015
)
Loss from continuing operations
$
(12,110
)
$
(15,691
)
Income (loss) from discontinued operations (including gain on
disposal of $8,446 during the three and nine months ended September
30, 2023)
4,031
(8,255
)
Net Loss
$
(8,079
)
$
(23,946
)
Net loss per share attributable to common stockholders-basic
and diluted
$
(0.07
)
$
(0.22
)
Weighted average shares of common stock outstanding-basic and
diluted
115,174,752
110,849,196
Precision Biosciences, Inc. Condensed Balance Sheets
Data (In thousands, except share amounts) (Unaudited)
September 30, 2023 December 31, 2022 Cash and cash
equivalents
$
122,231
$
189,576
Working capital
95,235
139,441
Total assets
164,344
238,169
Total liabilities
135,281
177,736
Total stockholders' equity
$
29,063
$
60,433
Common stock outstanding
115,680,008
110,964,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231107837870/en/
Investor and Media Contact: Mei Burris Senior Director of
Finance and Corporate Controller
Mei.Burris@precisionbiosciences.com
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