- PBGENE-HBV is the first-ever investigational
in vivo gene editing therapy cleared to enter clinical trials for
the treatment of chronic hepatitis B in the United States (U.S.)
–
- IND clearance represents a significant
regulatory milestone for PBGENE-HBV –
- Company to expand its Phase 1 ELIMINATE-B
study to the U.S., joining world-class clinical sites in Moldova,
Hong Kong, and New Zealand where strong clinical execution is
currently underway –
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 high unmet
need diseases, today announced that the U.S. Food and Drug
Administration (FDA) has cleared the Investigational New Drug (IND)
application for PBGENE-HBV. 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. Precision is in final stages of U.S. site activation,
enabling the ELIMINATE-B trial to begin enrolling patients in the
U.S. where over one million people are infected with chronic
hepatitis B. The ELIMINATE-B trial is actively enrolling patients
in Moldova, Hong Kong, and New Zealand and will soon initiate Phase
1 clinical activities in the U.S at the Liver Center at
Massachusetts General Hospital. The Company recently announced
promising safety and efficacy data for PBGENE-HBV following first
dose administration at the lowest dose level in the ELIMINATE-B
clinical trial.
“IND clearance to expand the ELIMINATE-B trial for chronic
hepatitis B is a first for the gene editing space. This marks the
advancement of potentially curative gene editing modalities into
major markets for diseases where enormous global disease burden and
lack of curative options have unfortunately become the norm,” said
Michael Amoroso, President and Chief Executive Officer of Precision
BioSciences. “With four regulatory approvals in four months, this
clearance validates our parallel global development strategy backed
by our robust pre-clinical safety and efficacy package. The
ELIMINATE-B trial remains on track following the recent completion
of initial dose administration in our first cohort of patients and
we look forward to continuing to share clinical data throughout
2025.”
“The IND clearance milestone marks a significant step forward in
our mission to advance potential curative treatment options for
hepatitis B patients,” said Murray A. Abramson, MD, MPH, Head of
Clinical Development at Precision BioSciences. “We’re very excited
to extend the ELIMINATE-B trial to the U.S. and look forward to
working with the investigators in the U.S., alongside those in
Moldova, Hong Kong and New Zealand as we build on the momentum of
the ELIMINATE-B phase 1 study.”
Investigators in the ELIMINATE-B trial accrued the first cohort
of patients within the first month of open enrollment and are
currently administering additional doses to patients in Cohort 1 at
the same dose level (0.2 mg/kg). In addition, the Company expects
to escalate to a higher dose level in Cohort 2 with the goal to
define the optimal dose and number of dose administrations for
safely eliminating cccDNA and inactivating integrated HBV DNA.
Precision plans to further expand the study to the U.K. and
continue accelerating recruitment and evaluation of a genetically
diverse patient population in the Phase 1 study. Precision plans to
share detailed clinical data throughout 2025.
“Hepatitis B has a significant impact on the lives of people
living with hepatitis B in the U.S. and many countries throughout
the world. Millions of people are eager for new treatment
approaches that can help reduce the burden of hepatitis B which
includes liver damage, liver cancer, premature death and social
stigma. In the U.S. alone there up to 2.4 million people living
with chronic hepatitis B, and despite vaccinations and continued
medical developments, the numbers haven’t changed appreciably over
the past 15 years,” said Chari A. Cohen, DrPH, MPH, Hepatitis B
Foundation President.
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. Despite the availability of approved antiviral therapies,
an estimated 300 million people globally and 1-2 million people in
the US are 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 (Viral Elimination Program):
PBGENE-HBV is Precision’s wholly owned in vivo gene editing
program under investigation in a global first-in-human clinical
trial, which is designed to potentially cure chronic hepatitis B
virus (HBV) infection. Currently, it is estimated that 300 million
people worldwide are afflicted with chronic hepatitis B. PBGENE-HBV
is the first and only potentially curative gene editing program to
enter clinical investigation that is specifically designed to
eliminate cccDNA and inactivate integrated HBV DNA. Lipid
nanoparticle technology for PBGENE-HBV has been provided by Acuitas
Therapeutics Inc.
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 safety profile and substantial antiviral
activity established after the first administration at dose level 1
of PBGENE-HBV; the clinical development and demonstrated, potential
and expected safety, efficacy and benefit of PBGENE-HBV, our other
product candidates and those being developed by partners; 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 and studies 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;
plans to provide additional administrations of PBGENE-HBV at the
first dose level; plans to escalate to higher dose levels and next
cohorts in the ELIMINATE-B clinical trial to define the optimal
dose and number of dose administrations for safely eliminating
cccDNA and inactivating integrated HBV DNA; expansion of the
ELIMINATE-B clinical trial to the United States and United Kingdom;
expectations around acceleration of recruitment of the ELIMINATE-B
clinical trial and plans to evaluate a genetically diverse patient
population in the Phase 1 study; expectations about achievement of
key milestones; and anticipated timing of patient dosing and
clinical data for PBGENE-HBV. In some cases, you can identify
forward-looking statements by terms such as “aim,” “anticipate,”
“approach,” “believe,” “contemplate,” “could,” “design,”
“designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,”
“mission,” “plan,” “possible,” “potential,” “predict,” “project,”
“pursue,” “should,” “strive,” “suggest,” “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/20250317223481/en/
Investor and Media Contact: Naresh Tanna Vice President
of Investor Relations naresh.tanna@precisionbiosciences.com
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