- Productive collaboration advanced three
programs and demonstrated proof of concept of ARCUS for gene
excision and gene insertion
- Precision exercised option to regain control
of programs and intends to bring collaboration programs back to
develop independently or with new partners
- Precision’s most important near-term clinical
priorities in Hepatitis B (HBV) and Primary Mitochondrial Myopathy
(PMM) and the expected cash runway into the second half of 2026 are
not impacted
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene
editing company, today announced the anticipated return of three
programs from Prevail Therapeutics Inc., a wholly owned subsidiary
of Eli Lilly and Company. Precision exercised its option to regain
rights for the programs following Prevail Therapeutics’ decision to
conclude the collaboration. Precision uses its novel proprietary
ARCUS® platform to develop in vivo gene editing therapies for
sophisticated gene edits, including gene elimination, insertion,
and excision. The collaboration began in January 2021 and was
amended in June 2023 to transfer certain preclinical research,
manufacturing, and investigational new drug (IND)-enabling
activities from Precision BioSciences to Prevail Therapeutics.
“We enjoyed a productive gene editing collaboration with Prevail
Therapeutics and appreciate their contributions to the success of
these programs. Together, we advanced three programs from concept
toward clinical candidates, and Precision completed its workplan
for these programs to the next stage gate, taking us to an
important development decision point,” said Michael Amoroso,
President and Chief Executive Officer of Precision BioSciences.
“Our decision to regain control of the programs brings exciting
development opportunities to Precision’s pipeline with a focus on
benefiting people born with incurable genetic diseases.”
“These in vivo gene editing programs are designed to take
advantage of unique attributes of ARCUS, namely its cut, size, and
simplicity. Our next steps will be to prepare for GLP toxicology
studies followed by potential IND and clinical trial application
(CTA) submissions,” said Jeff Smith, PhD, Co-Founder and Chief
Research Officer. “We are excited about the compelling in vivo
proof-of-concept data generated for ARCUS gene excision of a “hot
spot” region of the dystrophin gene in the DMD program.
Additionally, in vivo data for the gene insertion program
demonstrated up to 45% high efficiency gene insertion in
non-dividing cells of non-human primates measured by total liver
tissue. This is important and differentiating proof-of-concept data
for ARCUS compared to CRISPR, base editors and prime editors, which
have not demonstrated such high levels of gene insertion efficiency
in dividing or non-dividing cells in vivo, potentially enabling
broader therapeutic applicability for ARCUS.”
As a result of the strong proof of concept data generated to
date, Precision is exploring opportunities to develop the returned
programs independently or in partnership with others. Importantly,
the return of these programs does not impact the Company’s
near-term clinical priorities in ornithine transcarbamylase (OTC)
deficiency, HBV, and PMM or its expected cash runway to achieve
these clinical data milestones.
“Turning to our fundamental story, Precision continues to make
progress with our wholly owned programs for HBV and PMM as well as
through partnerships with Novartis and iECURE. Most recently,
iECURE has commenced regulatory and clinical activities in major
markets around the world to use the ARCUS platform for gene
insertion to address OTC deficiency using an ARCUS nuclease,” added
Mr. Amoroso.
Precision BioSciences remains focused on its most important
near-term priorities and clinical data milestones with several
opportunities to validate ARCUS for both wholly owned and the lead
partnered program in 2024 and 2025.
- The OTC deficiency program partnered with iECURE is the most
advanced ARCUS in vivo gene editing program with first-in-human
clinical dosing expected to commence in 2024. IND and CTAs have
been approved in the United States, United Kingdom, and Australia
for the Phase 1/2 OTC-HOPE study.
- Following receipt of regulatory guidance in and outside of the
United States, Precision’s wholly owned PBGENE-HBV viral
elimination program has commenced final IND and CTA enabling
studies and is rapidly progressing toward the clinic with
submissions planned in 2024.
- The PBGENE-PMM mutant mitochondrial DNA elimination program is
on track for IND and/or CTA submission in 2025.
The cash received from our recent public offering, upfront and
potential near-term cash from cell therapy transactions, along with
existing cash and cash equivalents, expected operational receipts,
continued fiscal and operating discipline, availability of our
at-the-market facility, and available credit, are expected to
provide Precision with a cash runway into the second half of 2026.
The completion of the collaboration does not impact Precision’s
expected cash runway as no milestones from Prevail Therapeutics
were assumed in our cash runway through 2026.
Company-Hosted Webcast and Conference Call
Information
Precision will host a conference call and webcast on Tuesday,
April 16, 2024, at 5:00pm EDT to discuss its in vivo gene editing
business. The dial-in conference call number is (800) 715-9871 and
the conference ID number for the call is 2110172. Participants may
access the live webcast, and accompanying presentation materials,
as well as the archived webcast on Precision’s website in the
Investors section under Events & Presentations:
https://investor.precisionbiosciences.com/events-and-presentations.
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.
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 and our partners’ product candidates
and gene editing approaches including editing efficiency; the
suitability of ARCUS nucleases for gene insertion, large gene
excision and other gene editing approaches; the expected timing of
regulatory processes, including filings and studies for PBGENE-HBV
and PBGENE-PMM; expectations of further presentations and
publications further differentiating ARCUS; expectations and
updates around partnership and collaboration opportunities; our
expected cash runway and available credit; the sufficiency of our
cash runway and available credit extending through phase 1 clinical
readouts for our HBV and PMM programs; expectations about
achievement of key milestones and receipt of any milestone,
royalty, or other payments; expectations regarding our liquidity
and capital resources; expectations about our and our partners’
operational initiatives and business strategy; and anticipated
timing of 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,”, “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 or other partnership opportunities to
advance our programs on terms that are acceptable to us, or at all;
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; our ability to
obtain an adequate supply of T cells from qualified donors; 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 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; 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
Annual Report on Form 10-K for the fiscal year ended December 31,
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240416117356/en/
Investor and Media Contact: Naresh Tanna Vice President
of Investor Relations Naresh.Tanna@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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