- FDA Advice Provides Clarity on Future
Development for Lead CAR T Program Candidate Azer-Cel; CAR T
Partnering Discussions Ongoing
- Presented Preclinical Data Demonstrating
Potential of ARCUS for Treatment of Duchenne Muscular Dystrophy and
Chronic Hepatitis B
- Company plans to host In Vivo Gene Editing
R&D Day on September 12, 2023
- Cash Balance Provides Expected Runway Through
Q1 2025
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS®-based in vivo gene editing
and ex vivo allogeneic CAR T therapies, today announced financial
results for the second quarter ended June 30, 2023 and provided a
business update.
“The first half of 2023 has been a busy time at Precision. Key
updates have continued to elucidate the development and potential
regulatory pathway for our ex vivo portfolio as well as demonstrate
the potential of our proprietary ARCUS® genome editing platform as
a differentiated technology for high efficiency in vivo gene
insertion and excision,” said Michael Amoroso, Chief Executive
Officer at Precision BioSciences. “Following our recent CAR T data
update in May 2023 and subsequent Type B End of Phase 1 meeting
with the U.S. Food and Drug Administration (FDA), we are actively
engaging with potential partners to advance azer-cel and PBCAR19B
to the next stage of development. As we prioritize organic
development of our in vivo portfolio, we continue to generate
supportive preclinical data to leverage the core features of ARCUS
and advance differentiated programs to the clinic. We look forward
to providing further updates across our in vivo programs at our
upcoming gene editing R&D event in September.”
Ex Vivo Allogeneic CAR T Platform
In July 2023, Precision received final meeting minutes from its
June 2023 Type B meeting with the FDA for azer-cel. The objective
of the meeting was to gain further clarity on the potential
registration path for azer‑cel including study design, endpoints,
and the recommended phase 2 dose in the CAR T relapsed patient
setting. The discussion with the FDA provided clarity on azer-cel
development, including a potential pathway toward registration.
Based on the advice received from the FDA and clinical data shared
during the May 2023 CAR T update, Precision is currently advancing
discussions with multiple potential strategic partners for its cell
therapy assets, including hematologic and non-hematologic
applications.
In Vivo Gene Editing Platform
ARCUS may have broad utility in many diseases and the Company
believes ARCUS is uniquely suited for in vivo gene editing,
including the potential to produce a profound impact on diseases
that are best treated by therapeutic gene insertion or excision of
large defective gene sequences.
Chronic Hepatitis B: Precision is developing PBGENE-HBV
for the treatment of patients with chronic hepatitis B with the
goal of submitting a clinical trial application (CTA) and/or
investigational new drug (IND) application in 2024. Hepatitis B
virus (HBV) causes inflammation and damage to the liver, which can
lead to chronic infection and increased risk of death from liver
cancer or cirrhosis. There is no cure for chronic hepatitis B and
current treatments rarely result in a functional cure, primarily
due to persistence of viral DNA in the liver. In patients with
chronic HBV infection, genetic material of the virus is converted
within infected liver cells into covalently closed circular DNA
(cccDNA) that acts as a template to make HBV copies. HBV also
inserts its DNA into the human genome of infected liver cells. This
integrated HBV DNA is a primary source of the viral protein,
hepatitis B surface antigen (HBsAg), which is secreted in the
blood. The presence of HBsAg is associated with poorer outcomes,
and elimination of HBsAg, along with loss of circulating HBV DNA,
is necessary for achieving a functional cure of chronic hepatitis
B. Using ARCUS, Precision scientists have generated a highly
specific nuclease designed to eradicate chronic HBV infection. The
Company believes PBGENE-HBV is the only approach designed to
inactivate and immediately eliminate cccDNA with direct edits as
well as to inactivate integrated HBV DNA with the goal of
long-lasting reductions in HBsAg and HBV DNA.
In June 2023, the Company presented data at the European
Association for Study of the Liver (EASL) Congress. In an episomal
adeno-associated virus (AAV) mouse model, Company researchers
demonstrated that administration of lipid nanoparticles containing
mRNA encoding an HBV-targeted ARCUS nuclease resulted in a 96%
reduction in serum HBsAg. In a follow-on experiment, treatment of
HBV-infected primary human hepatocytes with the HBV-targeted ARCUS
nuclease resulted in a 90% reduction of cccDNA and high
specificity. The Company plans to present additional data during
its in vivo gene editing R&D event in September and at
subsequent scientific conferences in 2023.
Novartis Partnered Program: Precision continues to
advance an in vivo gene insertion 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 a therapeutic transgene in vivo as a potential
one-time transformative treatment administered directly to the
patient that, if successful, would overcome many of the hurdles
present today with other therapeutic technologies, including those
that are utilizing an ex vivo gene editing approach.
Prevail Partnered Programs: Precision continues to
progress 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
Duchenne muscular dystrophy (DMD) in muscle, a central nervous
system directed target, and a liver 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. In May 2023, the Company presented
in vivo proof-of-concept data in preclinical models at the American
Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting
demonstrating the therapeutic potential of PBGENE-DMD, including
excision and repair of large sections of DNA. Precision scientists
observed the edited dystrophin variant in multiple tissue types
frequently involved in progression of DMD, including skeletal
muscle, heart, and diaphragm, enabling significant functional
muscle improvement.
In June 2023, Precision and Prevail entered into an amended and
restated development and license agreement to continue to
collaborate on developing the Company’s ARCUS nucleases for
potential in vivo therapies for genetic disorders. Precision will
continue to oversee creation, selection, in vitro development, and
optimization of ARCUS nucleases with respect to the gene targets
subject to the collaboration. Prevail will oversee and fund
preclinical research and IND-enabling activities which were
previously to be conducted by the Company at its expense. Prevail
retains responsibility for conducting clinical development and
commercialization activities for products from the collaboration.
The Company will be eligible to receive milestone payments of up to
an aggregate of $390 million to $395 million per licensed product,
a decrease from $420 million as provided in the Original Agreement.
This change reflects Prevail’s increased involvement in
pre-clinical activities.
Ornithine Transcarbamylase (OTC) Deficiency: Led by
iECURE, an ARCUS-mediated gene insertion approach is being pursued
as a potential treatment for neonatal onset OTC deficiency.
Non-human primate (NHP) data has been presented by researchers from
the University of Pennsylvania’s Gene Therapy Program demonstrating
sustained gene insertion of a therapeutic OTC transgene one-year
post-dosing in newborn and infant NHP with high efficiency. iECURE
is targeting submission of a CTA and/or IND in the second half of
2023.
Quarter Ended June 30, 2023 Financial
Results:
Cash and Cash Equivalents: As of June 30, 2023, Precision
had approximately $137.8 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
through the first quarter of 2025.
Revenues: Total revenues for the quarter ended June 30,
2023 were $19.8 million, as compared to $3.8 million for the same
period in 2022. The increase of $16.0 million was the result of a
$11.2 million increase in revenue recognized from Prevail, $10.7
million of which was the result of a cumulative catch-up adjustment
under the amended development and license agreement, and an
increase of $4.8 million in revenue recognized under the Novartis
Agreement.
Research and Development Expenses: Research and
development expenses were $21.9 million for the quarter ended June
30, 2023, as compared to $22.9 million for the same period in 2022.
The decrease of $1.0 million was primarily due to decreases in
PBCAR19B external development, outsourced research and development,
employee-related costs, and contract manufacturing organization
expenses partially offset by increases in in vivo external
development and azer-cel external development costs.
General and Administrative Expenses: General and
administrative expenses were $9.8 million for the quarter ended
June 30, 2023, as compared to $10.4 million for the same period in
2022. The decrease of $0.6 million was primarily driven by a
decrease in share-based compensation expense and expense
management, including a reduction in director and officer insurance
premiums.
Net Loss: Net loss was $11.9 million, or $(0.10) per
share (basic and diluted), for the quarter ended June 30, 2023, as
compared to a net loss of $31.0 million, or $(0.46) per share
(basic and diluted), for the same period in 2022. Weighted average
shares of common stock outstanding were approximately 114.1 million
for the quarter ended June 30, 2023, as compared to approximately
68.0 million for the quarter ended June 30, 2022. 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 2022.
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 several in vivo gene
editing candidates designed to cure genetic and infectious diseases
where no adequate treatments exist and multiple ex vivo clinical
candidates. 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. The Company intends such forward-looking statements to be
covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act of 1933,
as amended, and Section 21E of the Securities Exchange Act of 1934,
as amended. 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 editing efficiency and differentiating
aspects; the suitability of ARCUS nucleases for gene insertion,
large gene deletion, and other complex gene editing approaches and
the utilization of safe harbor site strategies for therapeutic gene
insertion; the expected timing of updates regarding our allogenic
CAR T and in vivo programs; the expected timing of regulatory
processes; expectations about our operational initiatives and
business strategy; expectations around partnership opportunities;
expectations about achievement of key milestones; and expectations
regarding our liquidity and capital resources. The words “aim,”
“anticipate,” “approach,” “believe,” “contemplate,” “could,”
“estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,”
“plan,” “possible,” “potential,” “predict,” “project,” “promise,”
“pursue,” “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 raising additional capital and requirements under
our current debt instruments and effects of restrictions
thereunder; 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; 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; 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; 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; 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 March 31, 2023,
as any such factors may be updated from time to time in our other
filings with the Securities and Exchange Commission (“SEC”),
including, but not limited to, our Quarterly Report on Form 10-Q
for the quarterly period ended June 30, 2023, 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 Statements of
Operations (In thousands, except share and per share amounts)
(unaudited)
For the Three Months Ended
June 30,
2023
2022
Revenue
$
19,789
$
3,820
Operating expenses Research and development
21,946
22,936
General and administrative
9,830
10,438
Total operating expenses
31,776
33,374
Operating loss
(11,987
)
(29,554
)
Other income (expense): Loss from equity method investment
(1,369
)
(1,448
)
Interest expense
(553
)
(178
)
Interest income
1,946
192
Gain (loss) on disposal of assets
72
(47
)
Total other income (expense)
96
(1,481
)
Net loss and net loss attributable to common stockholders
$
(11,891
)
$
(31,035
)
Net loss per share attributable to common stockholders-basic and
diluted
$
(0.10
)
$
(0.46
)
Weighted average shares of common stock outstanding-basic and
diluted
114,099,594
67,954,688
Precision Biosciences, Inc. Condensed Balance Sheets
Data (In thousands, except share amounts) (Unaudited)
June 30, 2023 December 31, 2022 Cash and cash
equivalents
$
137,794
$
189,576
Working capital
91,465
139,441
Total assets
181,697
238,169
Total liabilities
147,656
177,736
Total stockholders' equity
$
34,041
$
60,433
Common stock outstanding
115,070,517
110,964,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230804196095/en/
Mei Burris Director, Investor Relations and Finance
Mei.Burris@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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