Item 2.02 Results of Operations and Financial Condition.
On
The information in this Item 2.02 (including Exhibit 99.1) of this Current Report on Form 8-K is being furnished and shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended (the "Securities Act"), or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 7.01 Regulation FD Disclosure.
The Company has also updated its corporate deck, which is available in the "Investors & Media" portion of the Company's website at https://investor.precisionbiosciences.com.
The information in this Item 7.01 of this Current Report on Form 8-K is being furnished and shall not be deemed "filed" for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On
On
In 2022, the Company entered into a new in vivo gene editing collaboration with
Novartis focused on gene insertion for sickle cell disease and beta thalassemia,
added
The Company's management believes that, as of
Key priorities and planned upcoming milestones for 2023 include the following.
•
The Company intends to progress azer-cel to a decision point for a Phase 2 trial in NHL subjects who have relapsed following autologous CAR T treatment following planned completion of the Phase 1b cohort for azer-cel to determine final dosing schedule, requesting FDA clinical meeting pending data, and completing Phase 1 dose escalation for PBCAR19B in the earlier line NHL setting. The Company expects to present a CAR T clinical update in the first quarter of 2023, based on patient accrual and follow-up.
•
The Company intends to advance its wholly owned PBGENE-HBV in vivo program to final clinical candidate enabling a target Clinical Trial Application ("CTA") and/or Investigational New Drug ("IND") filing in 2024. Data published in 2022 demonstrates that ARCUS efficiently targeted and degraded hepatitis B virus ("HBV") cccDNA by 85% and durably reduced expression of HBV S-antigen by 77% in HBV-infected primary human hepatocytes ("PHH") and optimized specificity of the ARCUS nuclease completely prevented detectable chromosomal translocations in the PHH model. -------------------------------------------------------------------------------- Using lipid nanoparticle ("LNP") delivery, ARCUS nucleases showed high on-target editing and a robust decrease in viral DNA in both mouse and non-human primate models, along with 96% sustained reduction of HBV S-antigen in mice. The Company plans to present additional data at a scientific conference in 2023.
•
The Company intends to advance the first ARCUS in vivo gene editing program to
clinical readiness and pursue, in partnership with iECURE, an ARCUS-mediated
gene insertion approach as a potential treatment for neonatal onset ornithine
transcarbamylase ("OTC") deficiency. Non-human primate data presented by
researchers from the
•
The Company intends to progress its key partnered programs with Novartis and Lilly toward IND, including Duchenne muscular dystrophy ("DMD") and sickle cell disease.
As part of the previously announced ongoing strategic prioritization exercise for the Company's in vivo research pipeline, it has made the decision to cease pursuit of PBGENE-PCSK9 for familial hypercholesterolemia ("FH") with iECURE as its partner and, while the Company expects to continue to pursue gene knock-out programs opportunistically, the Company intends to prioritize programs involving complex edits, as with the HBV and DMD programs, and gene insertion (adding a functional copy of a gene) as exemplified by the partnered OTC program. PCSK9 for FH remains a wholly-owned program, and the Company is monitoring the regulatory landscape as it considers FH as well as several potential cardiovascular disease indications in its pipeline prioritization exercise. Additionally, work on the PBGENE-PH1 program progressed as planned in 2022. The Company has clinical candidates ready to proceed to the next stage of IND enabling studies. Based on the Company's new prioritized focus as well as the evolving treatment paradigm for primary hyperoxaluria type 1 ("PH1"), the Company has made the choice to look for a partner in the kidney disease arena for further development of PBGENE-PH1 and will no longer develop the program on its own.
Forward-Looking Statements
This Current Report on Form 8-K 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 and Section 21E of the Exchange Act. All statements contained in this Current Report on Form 8-K that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the research advancement, clinical development and regulatory review of our product candidates, the expected timing of updates regarding our CAR T and in vivo gene editing programs, the expected timing of our communications with regulators, expected efficacy and benefit of our product candidates and programs, expectations about our operational initiatives and our business strategy, achieving key milestones and additional collaborations, and expectations regarding our cash balance and ability to fund operating expenses and capital expenditure requirements. The words "aim," "anticipate," "approach," "believe," "contemplate," "could," "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 and requirements
under our current debt instruments and effects of restrictions thereunder; risks
associated with raising additional capital; 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; pending and potential liability lawsuits and
penalties against us or our collaborators related to our technology and our
product candidates; the
All forward-looking statements speak only as of the date of this Current Report on Form 8-K 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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Item 9.01. Financial Statements and Exhibits.
(d) Exhibits Exhibit No. Description 99.1 Press Release ofPrecision BioSciences, Inc. , datedJanuary 9, 2023 . 104 Cover Page Interactive Data File (embedded within the Inline XBRL document).
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