- Treatment with ECUR-506 resulted in a
complete clinical response from three months post exposure to the
end of study (six months post exposure)
- ECUR-506 was generally well tolerated with no
significant clinical safety concerns
- Insertion of a functional OTC gene through
ARCUS in vivo gene editing may provide lasting clinical benefit for
children with OTC deficiency who are in dire need of effective
treatments
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, including gene insertion, excision, and
elimination, today announced that its partner iECURE has reported
clinical efficacy and safety data in the first patient dosed with
ECUR-506 in the Phase 1/2 OTC-HOPE study. ECUR-506 is iECURE’s in
vivo gene insertion program designed to treat neonatal onset
Ornithine Transcarbamylase (OTC) deficiency utilizing a
PCSK9-specific ARCUS nuclease, licensed from Precision, that
enables insertion of a functional copy of the OTC gene.
“Congratulations to iECURE for these exciting results and
initial clinical validation in the first infant dosed in their
OTC-HOPE trial. These results showcase that a complete clinical
response can be achieved through in vivo gene editing for children
born with this devastating genetic disease,” said Michael Amoroso,
Chief Executive Officer of Precision BioSciences. “The data
increases our confidence in the therapeutic potential of ARCUS as a
novel in vivo gene editing approach for patients who have been and
plan to be treated in clinical trials by Precision and
partners.”
Treatment with ECUR-506, which employs an ARCUS® nuclease
licensed from Precision BioSciences, was generally well tolerated
in this infant with no significant clinical safety concerns apart
from asymptomatic transaminitis at four weeks. The asymptomatic
transaminitis was managed with immunosuppressive therapy and
resolved within four weeks. Twelve weeks after a single dose of
ECUR-506, ammonia scavenger medication was discontinued and mean
daily protein intake was increased to age-appropriate levels.
Protein liberalization was well tolerated, and the subsequent mean
ammonia level remained within normal limits and was reduced
compared to the mean pretreatment level. The response was
maintained through six months and indicates that a complete
response was achieved in this patient.
The OTC-HOPE study is ongoing in the United Kingdom, the United
States, Australia, and Spain, and iECURE expects to finish
enrollment in 2025 and provide complete data for the program in the
first half of 2026.
Precision’s ELIMINATE-B trial for PBGENE-HBV is ongoing in
chronic hepatitis B in Moldova, Hong Kong and New Zealand with
Phase 1 data expected in 2025.
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, Precision’s pipeline is comprised of in vivo
gene editing candidates designed to deliver lasting cures for the
broadest range of genetic and infectious diseases such as chronic
hepatitis B where no adequate treatments exist.
About ECUR-506
iECURE’s approach to gene editing for its initial programs,
including OTC deficiency, relies on the delivery of two
adeno-associated virus (AAV) vectors, each carrying different
payloads. ECUR-506 comprises two vectors, an ARCUS® nuclease vector
targeting gene editing in the well-characterized PCSK9 gene locus
and a donor vector that inserts the desired functional OTC gene.
iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision
BioSciences. The cut in the PCSK9 site serves as the insertion site
for the OTC gene, providing a potential path to permanent
expression of a functional gene. ECUR-506 is being studied in the
OTC-HOPE study, the first clinical ARCUS-based in vivo gene
insertion program.
About the OTC-HOPE Study
The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial
of ECUR-506 in baby boys with genetically confirmed neonatal onset
OTC deficiency and will test multiple dose levels of ECUR-506. The
study is enrolling newborn males up to seven months of age at
screening who are diagnosed with severe neonatal onset OTC
deficiency and meet certain other criteria. The primary objective
is to assess the safety and tolerability of intravenous
administration of a single dose of ECUR-506. It will also assess
the pharmacokinetics and efficacy of ECUR-506 administration and
the potential effects of ECUR-506 on disease-specific biologic
markers, developmental milestones and quality of life. The main
study will occur in a series of stages over a 10-month period,
including screening, stabilization, dosing eligibility, study drug
administration, and six-month follow-up. Upon completion of the
OTC-HOPE study, participants transition to the 14.5 year long term
follow up study (ECUR-LTFU). For more information, visit
https://OTC-HOPE.com.
About OTC Deficiency
OTC deficiency, the most common urea cycle disorder, is an
inherited metabolic disorder caused by a genetic defect in a liver
enzyme responsible for the detoxification of ammonia. Individuals
with OTC deficiency can build up excessive levels of ammonia in
their blood potentially resulting in devastating consequences,
including irreversible neurological damage, coma and death. The
severe form of the condition emerges shortly after birth and is
more common in boys than girls. The only corrective treatment for
early onset severe OTC deficiency is a liver transplant. Currently
available medical therapies do not correct the disease and do not
eliminate the risk of life-threatening symptoms or crises.
About iECURE
iECURE is a clinical-stage gene editing company focused on
developing therapies that utilize mutation-agnostic in vivo gene
insertion for the treatment of liver disorders with significant
unmet need. iECURE believes their approach has the potential to
restore the function of a dysfunctional gene, regardless of
mutation, by knocking-in a functional copy of that gene to offer
durable gene expression and long-term, potentially curative,
therapeutic benefit. iECURE’s management team has extensive
experience in executing global orphan drug and gene therapy
clinical trials and successfully commercializing multiple products.
iECURE intends to leverage their team’s core strength in research
and development strategy to identify what they believe to be the
most suitable target and modality for their product candidates to
address particular liver diseases. For more information, visit
https://iecure.com and follow on LinkedIn.
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, benefit, and therapeutic potential of our and our
partners’ product candidates and gene editing approaches (including
PBGENE-HBV); the suitability of ARCUS nucleases for gene insertion
and/or restoration of function, gene elimination other gene editing
approaches; the expected timing of regulatory processes;
expectations about our and our partners’ operational initiatives,
strategies, further development of our and our partners’ programs;
expectations about achievement of key milestones; and anticipated
timing of our and our partners’ clinical data. In some cases, you
can identify forward-looking statements by terms such as “aim,”
“anticipate,” “approach,” “believe,” “contemplate,” “could,”
“designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,”
“mission,” “plan,” “possible,” “potential,” “predict,” “project,”
“pursue,” “should,” “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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250109651360/en/
Investor and Media Contact: Naresh Tanna Vice President
of Investor Relations Naresh.Tanna@precisionbiosciences.com
Grafico Azioni Precision BioSciences (NASDAQ:DTIL)
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Da Dic 2024 a Gen 2025
Grafico Azioni Precision BioSciences (NASDAQ:DTIL)
Storico
Da Gen 2024 a Gen 2025
