- Program Updates Planned for Azer-cel and
PBCAR19B in May 2023
- Presented data on PBGENE-HBV program at
Global Hepatitis Summit 2023; PBGENE-DMD program data update
upcoming at ASGCT Conference
- In Vivo Gene Editing R&D Day Planned for
Mid-2023
- Cash Balance Provides Expected Runway through
Q1 2025
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 first quarter ended March 31, 2023 and provided a
business update.
“Our progress in the first quarter of 2023 makes it clear that
we are focused on the generation of meaningful clinical data from
our CAR T programs while working in parallel to advance our broad
in vivo portfolio into the clinic as soon as possible,” said
Michael Amoroso, Chief Executive Officer at Precision BioSciences.
“We continue to collect data from our latest patient cohorts for
both our azer-cel and PBCAR19B programs and anticipate providing an
update once we have sufficient follow up from these cohorts. We
expect that these data will inform key next steps for development
and potential regulatory interactions.”
“From our in vivo portfolio, we continue to leverage the core
features of ARCUS that we believe drive high efficiency gene
insertion and the ability to make large gene excisions while
advancing programs where we see a clear and rapid path to the
clinic. Preclinical data presentations at the Global Hepatitis
Summit 2023 on hepatitis B virus (HBV) and an upcoming, late
breaker oral presentation on Duchenne muscular dystrophy (DMD) at
the American Society of Gene & Cell Therapy Meeting highlight
key execution steps that have brought ARCUS closer to the clinic.
We look forward to providing further updates across our wholly
owned and partnered gene editing programs at our upcoming in vivo
gene editing R&D Day in the middle of the year,” added Mr.
Amoroso.
Ex Vivo Allogeneic CAR T Portfolio:
Azer-cel: Precision continues to progress its Phase 1/2a
clinical trial of azer-cel, the Company’s lead investigational
anti-CD19 allogeneic CAR T candidate, in adult subjects with
non-Hodgkin lymphoma (NHL) who have relapsed following CAR T
treatment. Precision plans to provide an update on the expansion
cohort as well as additional long-term follow up from the
previously presented azer-cel cohorts in May 2023. As of May 1,
2023, seven patients have been treated in the expansion/Phase 2a
cohort with a lower dose lymphodepletion regimen, in addition to
the 12 patients who were treated in previous cohorts.
PBCAR19B: Precision continues to progress the ongoing
Phase 1 study of PBCAR19B, its 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. The Company also expects to provide a program
update on seven patients treated at Dose Level 2 in May 2023.
In Vivo Gene Editing Portfolio:
Chronic Hepatitis B Virus (HBV): Precision is
prioritizing nomination of a development candidate for its wholly
owned PBGENE-HBV in vivo program, with the goal of submitting a
Clinical Trial Application (CTA) and/or Investigational New Drug
Application (IND) in 2024. An abstract titled “Targeting Hepatitis
B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate
Hepatitis B Virus In Vivo” was presented in April 2023 at the
Global Hepatitis Summit 2023. Data showed that ARCUS demonstrated a
high degree of on-target antiviral activity, as measured by
substantial reductions of both intracellular cccDNA and secretion
of HBsAg, with no detectable translocations in ARCUS-treated
primary human hepatocytes. These data support the further
development of PBGENE-HBV with the goal of developing a functional
cure for HBV. The Company plans to present additional data in
2023.
Novartis Partnered Program: Precision continues to
advance its gene editing program with Novartis to develop a custom
ARCUS nuclease for patients with sickle cell disease and beta
thalassemia. The collaborative intent is to insert, in vivo, a
therapeutic anti-sickling gene 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 those targeting an ex vivo gene
editing approach.
Prevail, a wholly-owned subsidiary of Lilly, Partnered
Programs: Precision continues its in vivo gene editing
collaboration with Prevail Therapeutics, a wholly-owned subsidiary
of Eli Lilly and Company (Lilly), in applying ARCUS nucleases to
three initial targets, including 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 April 2023, Precision announced that a late-breaking abstract
featuring preclinical data from its PBGENE-DMD program for the
potential treatment of DMD was selected for an oral presentation at
the American Society of Gene & Cell Therapy (ASGCT) 26th Annual
Meeting being held May 16-20, 2023 in Los Angeles, CA.
Ornithine Transcarbamylase (OTC) Deficiency: 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 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 NHP with high efficiency. iECURE is targeting to submit a
CTA and/or IND in the second half of 2023.
Other ARCUS Research: Preclinical data presented at the
Keystone Symposium in March 2023 demonstrated the unique potential
of ARCUS nucleases to achieve high frequency gene insertion in
nondividing primary hepatocytes. These data highlight the potential
advantage of ARCUS for use in developing therapeutics for gene
addition, for which Precision believes ARCUS is highly
differentiated given the need for extremely low levels of
off-target editing and high insertion efficiency.
Corporate:
Intellectual Property (IP) Update: In February 2023, the
U.S. Patent and Trademark Office issued Precision a Notice of
Allowance for an application with composition of matter claims
covering a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has
been used preclinically for both gene deletion approaches for
cardiovascular diseases such as familial hypercholesterolemia as
well as a viable and safe site to insert a functional copy of a
specific gene to restore function addressing other diseases
requiring gene addition. This patent further builds on the
Company’s IP portfolio that cover the ARCUS platform and its use
developing novel ex vivo and in vivo gene editing therapies.
Quarter Ended March 31, 2023 Financial
Results:
Cash and Cash Equivalents: As of March 31, 2023,
Precision had approximately $158 million in cash and cash
equivalents. In line with the same period for 2022, net cash used
in operating activities for the quarter ended March 31, 2023 are
expected to be higher in the first fiscal quarter than the
remainder of 2023 primarily due to the timing of the Company’s
annual compensation cycle. 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 March 31,
2023 were $8.8 million, as compared to $3.3 million for the same
period in 2022. The increase of $5.5 million in revenue during the
quarter ended March 31, 2023 was primarily driven by revenue
recognized under the Novartis agreement as work thereunder began in
the third quarter of 2022.
Research and Development Expenses: Research and
development expenses were $22.2 million for the quarter ended March
31, 2023, as compared to $20.0 million for the same period in 2022.
The increase of $2.2 million was primarily due to an increase in
direct research and development expenses related to our azer-cel
and PBCAR19B product candidates as well as consulting fees related
to our increased focus on quality management, partially offset by a
decrease in expenses related to manufacture of materials for use in
preclinical studies, PBCAR20A external development costs, and
share-based compensation expense from recent forfeitures.
General and Administrative Expenses: General and
administrative expenses were $11.1 million for the quarter ended
March 31, 2023, as compared to $10.7 million for the same period in
2022. The increase of $0.4 million was primarily related to
consulting fees related to in vivo program competitive landscape
analyses, partially offset by a decrease in insurance expense.
Net Loss: Net loss was $25.1 million, or $(0.23) per
share (basic and diluted), for the quarter ended March 31, 2023, as
compared to a net loss of $28.2 million, or $(0.46) per share
(basic and diluted), for the same period in 2022. Weighted average
shares of common stock outstanding were approximately 111.3 million
for the quarter ended March 31, 2023, as compared to approximately
61.0 million for the quarter ended March 31, 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 multiple ex vivo 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. 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 and
Section 21E of the Exchange Act. 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, clinical development and expected efficacy and benefit
of our product candidates and programs; timing and content of
program and data updates; the expected timing of regulatory
submissions and other communications; planned development
activities with our collaboration partners; expectations about our
operational initiatives, our business strategy and portfolio
review; expectations regarding our liquidity and capital resources;
the expected safety, efficacy, and benefit of our gene editing
approaches including editing efficiency and differentiating
aspects; and 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 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 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; 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 the COVID-19 pandemic and variants
thereof, or 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 Annual Report on Form 10-K for the annual period ended
December 31, 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 March 31, 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 March 31,
2023
2022
Revenue
$
8,780
$
3,317
Operating expenses Research and development
22,158
19,972
General and administrative
11,086
10,680
Total operating expenses
33,244
30,652
Operating loss
(24,464
)
(27,335
)
Other (loss) income: Loss from equity method investment
(1,341
)
(952
)
Change in fair value of investment
(769
)
—
Interest expense
(522
)
(42
)
Interest income
2,043
172
Loss on disposal of assets
(7
)
(11
)
Total other loss
(596
)
(833
)
Net loss and net loss attributable to common stockholders
$
(25,060
)
$
(28,168
)
Net loss per share attributable to common stockholders-basic and
diluted
$
(0.23
)
$
(0.46
)
Weighted average shares of common stock outstanding-basic and
diluted
111,301,409
61,031,775
Precision Biosciences, Inc. Condensed Balance
Sheets Data (In thousands, except share and per share amounts)
(Unaudited)
March 31, 2023 December 31, 2022
Cash and cash equivalents
$
158,132
$
189,576
Working capital
122,158
139,441
Total assets
204,502
238,169
Total liabilities
164,325
177,736
Total stockholders' equity
$
40,177
$
60,433
Common stock outstanding
112,339,421
110,964,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230509005380/en/
Investor and Media Contact: Mei Burris Director, Investor
Relations and Finance Mei.Burris@precisionbiosciences.com
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