- Regulatory feedback and alignment from both
U.S. and ex-U.S. regulators supports clinical development path for
PBGENE-HBV
- IND/CTA enabling studies with final clinical
candidate underway for PBGENE-HBV program; expect to file IND
and/or CTA in 2024
- Expected to be the first and only potentially
curative gene editing program to enter the clinic that is
specifically designed to eliminate cccDNA and inactivate integrated
HBV DNA
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 that the company has received pre-IND
regulatory feedback from the U.S. Food and Drug Administration
(FDA) and ex-U.S. agencies. Receipt of this regulatory feedback
provides alignment and clarity on Precision’s final
IND/CTA-enabling preclinical plans and clinical strategy for
PBGENE-HBV prior to advancement into Phase 1 clinical studies.
“We believe the regulatory feedback we have received to date
informs and strengthens our clinical strategy and provides a clear
pathway towards an IND and/or CTA filing for our lead gene editing
program PBGENE-HBV,” said Michael Amoroso, President and Chief
Executive Officer at Precision BioSciences. “Based on this
feedback, we have initiated the final preclinical studies as well
as site selection efforts as we move towards clinical readiness. We
look forward to filing regulatory applications in 2024 and continue
to believe that PBGENE-HBV has the potential to achieve a
functional cure for patients suffering with Chronic Hepatitis
B."
The pre-IND meeting with FDA provided feedback on overall design
for the proposed first-in-human clinical study as well as feedback
on the toxicology and specificity assessments. This interaction
builds on the engagement and alignment achieved through early
scientific advice during the FDA INTERACT meeting in July 2023.
Similar, productive consultations have been held with regulators in
ex-U.S. countries.
Collectively, the feedback and overall alignment obtained from
multiple regulatory authorities validates Precision’s path towards
a planned IND and/or CTA filing for PBGENE-HBV in 2024. Precision
is committed to rapidly advancing PBGENE-HBV into a global clinical
Phase 1 study and is currently expanding site selection efforts
with specialized centers focused on the treatment of Chronic
Hepatitis B in several countries. Simultaneously, manufacturing is
proceeding to supply drug for all planned nonclinical and clinical
activities.
PBGENE-HBV is expected to be the first and only potentially
curative gene editing program to enter the clinic that is
specifically designed to eliminate cccDNA and inactivate integrated
HBV DNA.
“Current treatment options for patients with HBV are unable to
both eliminate cccDNA and inactivate HBsAg expression from
integrated HBV DNA genomes in the hepatocyte, which is essential to
drive a durable loss of viral markers and potentially achieve a
functional cure for patients with HBV,” said Dr. Geoffrey Dusheiko,
MD, FCP(SA), FRCS. Emeritus Professor of Medicine, King’s College
Hospital & University College London. “Given the constructive
feedback from the FDA and other regulatory agencies worldwide, I
look forward to seeing this novel modality progressing to further
assessment in the clinic.”
About Hepatitis B and PBGENE-HBV:
Hepatitis B is a leading cause of morbidity in the US and death
globally, with no curative options currently available for
patients. In 2019, despite the availability of approved antiviral
therapies, an estimated 300 million people globally and more than 1
million people in the US were estimated to have chronic hepatitis B
infection. An estimated 15% to 40% of patients with HBV infections
may develop complications, such as cirrhosis, liver failure, or
liver cancer (hepatocellular carcinoma), which account for the
majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence
of HBV cccDNA and integration of HBV DNA into the human genome in
liver cells, the primary source of HBsAg in late-stage disease.
Current treatments for patients with HBV infection include agents
that result in long-term viral suppression as indicated by
reduction of circulating HBV DNA, but these therapies do not
eradicate HBV cccDNA, rarely lead to functional cure, and require
lifelong administration. PBGENE-HBV is a highly specific, novel
therapeutic approach to treating patients with chronic HBV
infection. It’s designed to directly eliminate cccDNA and
inactivate integrated HBV DNA with high specificity, resulting in
degraded cccDNA and a reduction in HBsAg.
About ARCUS
ARCUS is a proprietary genome editing technology discovered and
developed by scientists at Precision BioSciences. It uses
sequence-specific DNA-cutting enzymes, or nucleases, that are
designed to either insert, excise, or eliminate DNA of living cells
and organisms. ARCUS is based on a naturally occurring genome
editing enzyme, I-CreI, that evolved in the algae Chlamydomonas
reinhardtii to make highly specific cuts in cellular DNA and
stimulate gene insertion at the cut site by homologous
recombination. Precision's platform and products are protected by a
comprehensive portfolio including more than 130 granted patents to
date.
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. 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 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 durably
achieve a near-complete reduction across viral markers including
HBsAg, HBeAg, HBV DNA and HBV RNA and as a potentially curative
treatment for HBV with no off-target editing at maximal on-target
editing dose; and the expected timing of manufacturing activities
and regulatory processes, including timing of expected CTA and/or
IND filings. The words “aim,” “anticipate,” “approach,” “believe,”
“contemplate,” “could,” “designed”, “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; our dependence on our ARCUS
technology; the risk that other genome-editing technologies may
provide significant advantages over 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; 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’ or other licensees’
ability to advance product candidates into, and successfully
design, implement and complete, clinical or field trials; our or
our collaborators’ other licensees’ 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; 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 sustained
inflation, supply chain disruptions and major central bank policy
actions; insurance expenses and exposure to uninsured liabilities;
effects of tax rules; risks related to ownership of our common
stock; our ability to meet the requirements of and maintain listing
of our common stock on NASDAQ or other public stock exchanges and
other important factors discussed under the caption “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarterly period ended
September 30, 2023, as any such factors may be updated from time to
time in our other filings 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240214220478/en/
Investor and Media Contact: Naresh Tanna Vice President,
Investor Relations naresh.tanna@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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