Precision BioSciences Reports Third Quarter 2023 Financial Results and Provides Business Update
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- Precision to present late breaker PBGENE-HBV data at The Liver Meeting of AASLD in November 2023
- Hosted R&D Day highlighting exclusive focus on in vivo gene editing pipeline
- Expanded portfolio with PBGENE-PMM as a potentially curative treatment for m.3243 associated primary mitochondrial myopathy
- Completed strategic transaction with Imugene for azer-cel in cancer
- Extended cash runway through the end of 2025
“The ARCUS platform has always been foundational to Precision, as we have harnessed its distinct properties in pursuit of life changing medicines for patients. Our recent transaction with Imugene allows Precision to focus the power of ARCUS on in vivo gene editing while capturing near-term and potential long-term value for our CAR T assets,” said Michael Amoroso, Chief Executive Officer at Precision BioSciences. “At our recent R&D Day, we featured data demonstrating how the differentiated cut, size, and simplicity of ARCUS enable it to uniquely carry out sophisticated edits that present clear advantages in the market and compelling therapeutic opportunities versus other editing technologies. By design, ARCUS has been shown to preferentially drive higher efficiency gene insertion than CRISPR-derived editors, and its ease of delivery and simplicity confer greater utility in reaching tissues, organs, and organelles for gene insertion, excision and elimination. Going forward, Precision will solely focus its own efforts on programs leveraging ARCUS’ specific utility for sophisticated edits. To that end, we are progressing wholly-owned PBGENE-HBV and PBGENE-PMM programs towards anticipated CTA and/or IND filings in 2024 and 2025, respectively. In addition, the ARCUS OTC gene insertion program being developed by our partner at iECURE is targeting a CTA and/or IND filing by the end of this year. Importantly, as we execute against our singular focused in vivo strategy, we believe we will have sufficient capital to advance these wholly-owned programs into the clinic and to Phase 1 data points over the next two years.”
Completed Strategic Transaction with Imugene for Azer-Cel in Cancer
In August 2023, the Company completed a strategic transaction with Imugene Limited (ASX: IMU) for the Company’s lead allogeneic chimeric antigen receptor (CAR) T candidate for cancer, azercabtagene zapreleucel (azer-cel). In exchange for global rights to azer-cel for cancer, as well as Precision’s CAR T infrastructure and experienced cell therapy teams, Precision received upfront cash and equity consideration valued at
Advancing as a Single Platform Company Focused on ARCUS In Vivo Gene Editing
Precision is now solely focused on leveraging its proprietary ARCUS genome editing platform to advance in vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination unlocking a broader potential for ARCUS in vivo gene editing in human therapeutics.
In support of the go-forward strategy, Precision presented two poster presentations at the European Society of Gene & Cell Therapy congress on October 25 and 26, 2023, in
Wholly-owned Portfolio
PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B and expects to submit a clinical trial application (CTA) and/or investigational new drug (IND) application in 2024.
Chronic infection with hepatitis B virus (HBV) is due to persistence of the viral genome, in the form of covalently closed circular DNA (cccDNA) and viral sequences integrated into the human genome. Current treatments for chronic hepatitis B rarely achieve a functional cure, defined as sustained undetectable levels of circulating hepatitis B surface antigen (HBsAg) and HBV DNA after a finite course of treatment, as they do not eliminate the root cause of viral persistence. In September 2023, at its R&D Day, the Company shared data highlighting the potential of PBGENE-HBV to drive sustained reductions of circulating HBsAg and HBV DNA. Unlike other therapies in development, PBGENE-HBV directly eliminates cccDNA and inactivates integrated viral sequences, representing a potentially curative approach.
A late-breaking abstract featuring preclinical data on the PGBENE-HBV program has been accepted for a poster presentation, “Preclinical efficacy and safety of ARCUS-POL nucleases for chronic hepatitis B: a potentially curative strategy,” at the American Association for the Study of Liver Diseases (AASLD) Annual Meeting being held in
PBGENE-PMM (Mutant Mitochondrial Elimination Program): At its R&D Day, Precision announced that it will pursue development of PBGENE-PMM as a potential first-in-class opportunity for treatment of m.3243-associated primary mitochondrial myopathy (PMM). Mitochondrial diseases are the most common hereditary metabolic disorder, affecting 1 in 4,300 people. PMM currently lacks a curative treatment and impacts approximately
In September 2023, Precision received a Notice of Allowance from the
Partnered Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, an ARCUS-mediated gene insertion approach is being pursued as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Non-human primate (NHP) data presented in October 2022 by researchers from the University of Pennsylvania’s Gene Therapy Program demonstrated sustained gene insertion of a therapeutic OTC transgene one-year post-dosing in newborn and infant NHPs with high efficiency. iECURE expects to submit a CTA and/or IND in the second half of 2023.
PBGENE-NVS (Gene Insertion Program): Precision continues to advance its in vivo gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic transgene into hematopoietic stem cells as a potential one-time transformative treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, including ex vivo gene editing approaches.
PBGENE-DMD (Gene Excision Program): Precision continues its in vivo gene editing collaboration with Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, in applying ARCUS nucleases to three initial targets, including DMD in muscle, a liver-directed target, and a central nervous system-directed target. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV, that are designed to excise an approximately 500,000 base pair mutation “hot spot” region from the dystrophin gene to generate a variant of the dystrophin protein that is functionally competent. During its September 2023 R&D Day, the Company highlighted preclinical data demonstrating the potential of ARCUS in vivo gene editing for large gene excisions and that the edited dystrophin variant was observed in multiple tissue types frequently involved in progression of DMD, including skeletal muscle, heart, and diaphragm, thereby enabling significantly improved muscle function.
Quarter Ended September 30, 2023 Financial Results:
In August 2023, the Company announced its final stages of execution to operate as a single platform company focused exclusively on developing in vivo gene editing therapies with the completion of the strategic transaction with Imugene for azer-cel for cancer. Precision determined that the decision to only pursue development of CAR T cell therapies through partnerships qualified for discontinued operations accounting treatment. Accordingly, the accompanying financial statements for all periods presented reflect Precision’s CAR T cell therapy business as a discontinued operation.
Cash and Cash Equivalents: As of September 30, 2023, Precision had approximately
Revenues: Total revenues for the quarter ended September 30, 2023 were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Interest Income/Expense: Interest expense was
Interest income was
Discontinued Operations: Income from discontinued operations was
Net Loss: Net loss was
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.
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 and gene editing approaches including ARCUS’s potential editing efficiency and differentiating aspects and the ability for the ARCUS genome editing platform to develop differentiated programs; the suitability of ARCUS nucleases for gene or viral elimination, large excision, gene insertion, and other complex gene editing approaches to drive defined outcomes; the potential of PBGENE-HBV to drive sustained reductions of circulating HBsAg and HBV DNA; the expected timing of regulatory processes, including timing of expected CTA and/or IND filings; expectations about our operational initiatives and business strategy; expectations around the Company’s partnerships; expectations about achievement of key milestones; and expectations regarding the Company’s cash runway, including the assumptions underlying the Company’s calculations of its cash runway and whether its cash runway will be sufficient to provide first-in-human clinical data for the Company’s research and development programs. The words “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed”, “estimate,” “expect,” “explore,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “promise,” “pursue,” “seek” “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 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’ ability to identify, develop and commercialize product candidates; pending and potential product 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 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.
| Precision Biosciences, Inc. | ||||||||
| Condensed Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| (unaudited) | ||||||||
| For the Three Months Ended September 30, | ||||||||
2023 |
2022 |
|||||||
| Revenue | $ |
13,120 |
|
$ |
7,363 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
15,850 |
|
|
11,758 |
|
||
| General and administrative |
|
9,633 |
|
|
10,281 |
|
||
| Total operating expenses |
|
25,483 |
|
|
22,039 |
|
||
| Operating loss |
|
(12,363 |
) |
|
(14,676 |
) |
||
| Other income (expense): | ||||||||
| Loss from equity method investment |
|
(1,350 |
) |
|
(1,782 |
) |
||
| Gain on changes in fair value |
|
311 |
|
|
— |
|
||
| Interest expense |
|
(576 |
) |
|
(405 |
) |
||
| Interest income |
|
1,870 |
|
|
1,172 |
|
||
| Loss on disposal of assets |
|
(2 |
) |
|
— |
|
||
| Total other income (expense) |
|
253 |
|
|
(1,015 |
) |
||
| Loss from continuing operations | $ |
(12,110 |
) |
$ |
(15,691 |
) |
||
| Income (loss) from discontinued operations (including gain on disposal of |
|
4,031 |
|
|
(8,255 |
) |
||
| Net Loss | $ |
(8,079 |
) |
$ |
(23,946 |
) |
||
| Net loss per share attributable to common stockholders- basic and diluted |
$ |
(0.07 |
) |
$ |
(0.22 |
) |
||
| Weighted average shares of common stock outstanding- basic and diluted |
|
115,174,752 |
|
|
110,849,196 |
|
||
| Precision Biosciences, Inc. | ||||||
| Condensed Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| (Unaudited) | ||||||
| September 30, 2023 | December 31, 2022 | |||||
| Cash and cash equivalents | $ |
122,231 |
$ |
189,576 |
||
| Working capital |
|
95,235 |
|
139,441 |
||
| Total assets |
|
164,344 |
|
238,169 |
||
| Total liabilities |
|
135,281 |
|
177,736 |
||
| Total stockholders' equity | $ |
29,063 |
$ |
60,433 |
||
| Common stock outstanding |
|
115,680,008 |
|
110,964,035 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20231107837870/en/
Investor and Media Contact:
Mei Burris
Senior Director of Finance and Corporate Controller
Mei.Burris@precisionbiosciences.com
Source: Precision BioSciences, Inc.
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