- Approval in Hong Kong marks the first
clinical trial application clearance of an in vivo gene editing
approach for chronic hepatitis B in Hong Kong and the second CTA
approval for PBGENE-HBV in 2024
- ELIMINATE-B is a global, multi-site study now
actively recruiting patients; expected to report clinical data as
it matures throughout 2025
- U.S. investigational new drug (IND)
anticipated in 2025
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company utilizing its novel proprietary ARCUS®
platform to develop in vivo gene editing therapies for
sophisticated gene edits, today announced that it has received
Clinical Trial Application (CTA) approval in Hong Kong to study
PBGENE-HBV in the ongoing ELIMINATE-B Phase I trial. PBGENE-HBV is
Precision’s lead wholly owned in vivo gene editing program designed
to cure chronic hepatitis B by eliminating cccDNA, the key source
of replicating hepatitis B virus (HBV), and inactivating integrated
HBV DNA in hepatocytes. The company is actively recruiting patients
for the ELIMINATE-B trial in Moldova and has begun activating a top
infectious disease clinical site in Hong Kong as part of its global
regulatory and clinical operations strategy.
“We are pleased to receive our second CTA approval for
PBGENE-HBV, expanding our ELIMINATE-B trial into a world-class HBV
clinical trial site in Hong Kong,” said Dr. Murray Abramson, Senior
Vice President, Head of Clinical Development of Precision
BioSciences. “PBGENE-HBV has been designed to target a viral site
that is prevalent across all HBV genotypes, including in Asia, and
we believe that this will enable us to evaluate and provide access
to as many patients as possible. In addition to Hong Kong, our
clinical trial site in Moldova continues to execute as planned, and
we look forward to sharing clinical data as it matures throughout
2025.”
“Over 400,000 individuals in Hong Kong are currently living with
chronic hepatitis B, and many patients continue to develop liver
cancer or cirrhosis even on existing long-term treatment with
standard of care treatments,” said Dr. MF Yuen, DSc, MD, PhD, Chair
Professor of Gastroenterology and Hepatology, Li Shu Fan Medical
Foundation and Professor in Medicine, The University of Hong Kong.
“By targeting and eliminating covalently closed circular (ccc) DNA,
the root cause of the disease, PBGENE-HBV has the potential to
completely eliminate the source of viral replication from the body,
not just reduce it, which could transform the treatment landscape
for patients in Hong Kong and millions of others worldwide. Our
field has long searched for a novel way to eliminate the root cause
of chronic hepatitis B, the cccDNA, and I look forward to further
investigating PBGENE-HBV in clinic.”
Through its precision cutting, compact design, and simple
structure, PBGENE-HBV is engineered to target the HBV viral genome
and drive functional cures for patients with chronic hepatitis B.
PBGENE-HBV leverages the ARCUS® gene editing platform by delivering
an ARCUS nuclease-encoding mRNA to the liver via lipid
nanoparticles. The ARCUS nuclease specifically cuts a highly
conserved sequence in the hepatitis B viral genome and is designed
to eliminate the root cause of the disease, cccDNA, and inactivate
integrated HBV genomes. The ARCUS platform is derived from a
naturally occurring enzyme and has been optimized for over 20 years
into a ground-breaking and highly precise gene editing tool.
Precision has submitted multiple global clinical trial
applications and remains on track for a U.S. IND in 2025 as part of
its global Phase 1 regulatory strategy for PBGENE-HBV. The company
will provide updates as it receives additional regulatory approvals
to begin treating patients in those markets.
About Hepatitis B:
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 hepatitis B surface antigen
(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.
About PBGENE-HBV:
PBGENE-HBV is a potentially curative approach to treating
patients with chronic HBV infection through a highly specific,
novel therapeutic approach. PBGENE leverages the ARCUS® platform
and is designed to directly eliminate cccDNA and inactivate
integrated HBV DNA with high specificity, potentially leading to
functional cures.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage 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.
The ARCUS® platform is being used to develop in vivo gene
editing therapies for sophisticated gene edits, including gene
insertion (inserting DNA into gene to cause expression/add
function), elimination (removing a genome e.g. viral DNA or mutant
mitochondrial DNA), and excision (removing a large portion of a
defective gene by delivering two ARCUS nucleases in a single
AAV).
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 (including
PBGENE-HBV); the unique design of PBGENE-HBV to eliminate cccDNA
and inactivate integrated HBV DNA with high specificity,
potentially leading to functional cures; the expected timing of
regulatory processes (including filings such as IND’s and CTA’s for
PBGENE-HBV and the acceptance of these filings by regulatory
agencies); the suitability of PBGENE-HBV for the treatment of
hepatitis and the targeting of the root cause of the disease; the
key advantages of ARCUS and its key capabilities and
differentiating characteristics ; expectations about operational
initiatives, strategies, and further development of PBGENE-HBV; and
anticipated timing of patient dosing and clinical data. In some
cases, you can identify forward-looking statements by terms such as
“aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,”
“design”, “designed,” “endeavor,” “engineered,” “estimate,”
“expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,”
“possible,” “potential,” “predict,” “project,” “pursue,” “should,”
“strive,” “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. These statements are neither promises nor
guarantees, and involve 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 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’ or other licensees’ ability to identify, develop
and commercialize product candidates; pending and potential product
liability lawsuits and penalties against us or our collaborators or
other licensees related to our technology and our product
candidates; the U.S. and foreign regulatory landscape applicable to
our and our collaborators’ or other licensees’ development of
product candidates; our or our collaborators’ or other licensees’
ability to advance product candidates into, and successfully
design, implement and complete, clinical trials; potential
manufacturing problems associated with the development or
commercialization of any of our product candidates; delays or
difficulties in our and our collaborators’ and other licensees’
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 or our
licensees’ 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’ or other
licensees’ 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 and
other license 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; 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,
2024, 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/20241218082974/en/
Investor Contact: Naresh Tanna Vice President, Investor
Relations Naresh.Tanna@precisionbiosciences.com
Grafico Azioni Precision BioSciences (NASDAQ:DTIL)
Storico
Da Dic 2024 a Gen 2025
Grafico Azioni Precision BioSciences (NASDAQ:DTIL)
Storico
Da Gen 2024 a Gen 2025
