Precision BioSciences Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides Business Update
- Initiated final IND/CTA enabling studies for lead in vivo gene editing program PBGENE-HBV, expecting to submit IND and/or CTA in 2024.
- Completed licensing agreements with Imugene, TG Therapeutics, and Caribou Biosciences, generating nearly $50 million in upfront and potential near-term payments.
- Completed a $40 million public offering to extend cash runway into the second half of 2026, enabling achievement of first-in-human Phase 1 clinical data for two wholly owned programs.
- Focused on advancing PBGENE-HBV and PBGENE-PMM towards IND and/or CTA filings in 2024 and 2025, respectively, while planning to commence a new gene insertion program.
- Anticipates presenting and publishing new data in 2024 to differentiate ARCUS as a potential best-in-class tool for high efficiency gene insertion via homology directed repair.
- None.
Insights
The recent financial results and business update from Precision BioSciences highlight a strategic pivot towards in vivo gene editing and the monetization of assets to strengthen the balance sheet. The upfront and potential near-term payments from licensing agreements, totaling nearly $50 million, are significant as they provide non-dilutive funding which is critical for biotech companies. The $40 million public offering further extends the company's cash runway into the second half of 2026, which is essential for investor confidence, as it suggests that Precision has secured the necessary funding to reach critical milestones without the immediate need for additional capital raises.
From an investment perspective, the stock purchase by TG Therapeutics at a 100% premium indicates a strong vote of confidence in Precision's valuation and future prospects. The partnerships with TG Therapeutics, Imugene and Caribou Biosciences not only bring in immediate cash but also open avenues for future milestone payments and royalties, which could provide long-term revenue streams. However, investors should be mindful of the inherent risks in biotech investments, as the success of Precision's gene editing programs is contingent on clinical outcomes and regulatory approvals that are yet to be determined.
Advancements in Precision BioSciences' in vivo gene editing programs, particularly PBGENE-HBV for chronic hepatitis B and PBGENE-PMM for primary mitochondrial myopathy, represent a significant leap in gene therapy. The successful preclinical data and the expected IND/CTA submissions are pivotal steps toward clinical trials. The proprietary ARCUS platform's differentiation through its ability to perform sophisticated gene edits without the need for a guide RNA positions Precision BioSciences as a potential leader in the space. This capability could address limitations seen with other gene editing technologies like CRISPR-Cas.
However, the scientific community and potential investors should consider the challenges of translating preclinical success to human trials. The gene editing field is highly competitive and rapidly evolving, with regulatory landscapes that are still being shaped. Precision's ability to navigate these complexities will be critical for the success of their programs and the company's valuation. Moreover, the long-term efficacy and safety of these treatments remain to be seen in clinical settings, which will be the true test of the company's innovative approach.
The gene editing market is poised for growth, driven by technological advancements and an increasing prevalence of genetic disorders. Precision BioSciences' focus on in vivo gene editing and their strategic partnerships position them well within this burgeoning field. The company's decision to monetize non-core assets and pivot towards wholly owned programs reflects a trend in the biotech industry to streamline operations and focus on areas with the highest potential for success and profitability.
Investors should note that Precision's strategy to differentiate ARCUS and their choice to pursue treatments for conditions with high unmet medical needs could capture significant market share if successful. However, the market's response to these developments will depend on continued positive data and regulatory milestones. Precision's approach to capitalizing on their proprietary technology through strategic partnerships also demonstrates a savvy understanding of market dynamics, potentially increasing their attractiveness for future collaborations or even acquisition.
- Initiated final IND/CTA enabling studies for lead in vivo gene editing program PBGENE-HBV; expect to submit IND and/or CTA in 2024
- Completed licensing agreements to monetize divested CAR T assets with Imugene, TG Therapeutics and Caribou Biosciences including nearly
- Completed
“2023 was a transformative year for Precision BioSciences as we fully transitioned to our core capability as an in vivo gene editing company rapidly advancing development of our wholly owned in vivo programs led by PBGENE-HBV and quickly followed by PBGENE-PMM. As a part of this transition, we successfully monetized our prior CAR T investments and completed a
“As we look ahead, we continue to advance our focused strategy to differentiate ARCUS within the gene editing field and progress both our wholly owned and partnered gene editing programs. In our wholly owned pipeline, we plan to continue to advance PBGENE-HBV and PBGENE-PMM towards IND and/or CTA filings in 2024 and 2025, respectively, as well as commence a new gene insertion program of our own. More broadly, we anticipate presenting and publishing new data this year that further differentiates ARCUS as a potential best-in-class tool for high efficiency gene insertion via homology directed repair. We believe that these complementary objectives will continue to build momentum and establish Precision BioSciences as a leading in vivo gene editing company,” added Mr. Amoroso.
Wholly Owned Portfolio
PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B. Currently, it is estimated that approximately 300 million people worldwide are afflicted with chronic Hepatitis B. In November 2023, Precision presented preclinical efficacy and safety data at the 2023 American Association for the Study of Liver Diseases Annual Meeting. The data demonstrated strong proof of concept efficacy and safety for the final clinical candidate including no detectable off-target editing at maximal on-target editing dose.
In February 2024, Precision announced that the company had received pre-IND regulatory feedback from the
PBGENE-PMM (Mutant Mitochondrial DNA Elimination Program): PBGENE-PMM is a first of its kind potential treatment for m.3243-associated primary mitochondrial myopathy (PMM). Mitochondrial diseases are the most common hereditary metabolic disorder in the world, affecting 15,000 to 25,000 people in the
Partnered Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is the first ARCUS-mediated gene editing program to advance into the clinic following approval from the Australian Therapeutic Goods Administration and the
PBGENE-NVS (Gene Insertion Program): Precision continues to advance its 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 as a potential one-time transformative treatment administered directly to the patient to overcome disparities in patient access to treatment with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach.
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 (Lilly), in applying ARCUS nucleases to three initial targets, including Duchenne muscular dystrophy (DMD) in muscle, a central nervous system directed target, and a liver directed target. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single adeno-associated virus (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 the Company’s September 2023 R&D Day, Precision 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, enabling significantly improved muscle function.
PBGENE-LLY2 (Gene Insertion Program): During the Precision 2023 Gene Editing R&D Day, Precision highlighted new data demonstrating that ARCUS is capable of high efficiency gene insertion in nondividing cells in adult nonhuman primates, the most challenging context for gene insertion. In the pre-clinical study involving coadministration of AAV and lipid nanoparticle, Precision scientists observed
Business Updates – Monetization of CAR T Investments:
Completed Licensing Deal with TG Therapeutics for Cell Therapy Azer-Cel in Autoimmune Diseases:
In January 2024, Precision announced the completion of a transaction with TG Therapeutics (Nasdaq: TGTX) for certain exclusive and non-exclusive license rights to develop Azercabtagene Zapreleucel (azer-cel) for autoimmune diseases and other indications outside of cancer. In exchange for these rights, Precision received upfront and potential near-term economics valued at
Completed Licensing Deal with Imugene Limited for Azer-Cel in Cancer:
The agreement with TG Therapeutics followed an agreement with Imugene Limited (ASX: IMU) in August 2023 for an exclusive license for azer-cel in cancer. In exchange for global rights to azer-cel for cancer Precision received upfront economics valued at
Completed Non-Exclusive Patent License Agreement with Caribou Biosciences:
In February 2024, Precision announced that it had granted Caribou Biosciences (Nasdaq: CRBU) a non-exclusive, worldwide license, with the right to sublicense, to one of Precision’s foundational cell therapy patent families for use with CRISPR-based therapies in the field of human therapeutics. Under the terms of the agreement, Precision received an upfront payment and, upon commercialization by Caribou, will receive royalties on net sales of licensed products. In addition, for each occurrence of certain strategic transactions involving Caribou, Precision is entitled to receive a specific tiered milestone payment.
In total, these three transactions provide Precision BioSciences with nearly
Business Updates –
On March 1, 2024, Precision completed a
Quarter Ended December 31, 2023 Financial Results:
Cash and Cash Equivalents: As of December 31, 2023, Precision had
Revenues: Total revenues for the quarter ended December 31, 2023 were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Other Income and Expense: Total other income was
Continuing Operations: Loss from continuing operations was
Net Loss: Net loss was
Fiscal Year 2023 Financial Results:
Revenues: Total revenues for the year ended December 31, 2023 were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Other Income and Expense: Total other income was
Continuing Operations: Loss from continuing 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.
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), excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV), and elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA).
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 (including azer-cel) and gene editing approaches including editing efficiency and differentiating aspects; the suitability of azer-cel for oncology indications and non-oncology indications including immunological diseases; the suitability of ARCUS nucleases for gene insertion, gene elimination, large gene deletion, and other gene editing approaches; the expected timing of regulatory processes (including filings and studies for PBGENE-HBV and PBGENE-PMM); expectations about our operational initiatives and business strategy; 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 clinical phase 1 readouts for our HBV and PPM programs; expectations about achievement of key milestones and receipt of any milestone, royalty, or other payments; expectations regarding our liquidity and capital resources; and anticipated timing of initial 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,” “seek,” “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
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. | ||||||||
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Three Months Ended December 31, | ||||||||
|
2023 |
|
|
2022 |
|
|||
| Revenue | $ |
7,038 |
|
$ |
10,598 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
13,389 |
|
|
12,683 |
|
||
| General and administrative |
|
8,539 |
|
|
10,024 |
|
||
| Total operating expenses |
|
21,928 |
|
|
22,707 |
|
||
| Operating loss |
|
(14,890 |
) |
|
(12,109 |
) |
||
| Other income (expense): | ||||||||
| Impairment charges |
|
— |
|
|
(10,844 |
) |
||
| Loss on disposal of assets |
|
(524 |
) |
|
(19 |
) |
||
| Gain (loss) on changes in fair value |
|
1,603 |
|
|
(510 |
) |
||
| (Loss) gain from equity method investment |
|
(871 |
) |
|
2,604 |
|
||
| Interest expense |
|
(579 |
) |
|
(486 |
) |
||
| Interest income |
|
1,827 |
|
|
1,937 |
|
||
| Total other income (expense) |
|
1,456 |
|
|
(7,318 |
) |
||
| Loss from continuing operations | $ |
(13,434 |
) |
$ |
(19,427 |
) |
||
| Loss from discontinued operations |
|
(2,855 |
) |
|
(9,061 |
) |
||
| Net Loss | $ |
(16,289 |
) |
$ |
(28,488 |
) |
||
| Net loss per share attributable to common stockholders-basic and diluted | $ |
(4.06 |
) |
$ |
(7.70 |
) |
||
| Weighted average shares of common stock outstanding-basic and diluted |
|
4,010,467 |
|
|
3,698,456 |
|
||
| Precision Biosciences, Inc. | ||||||||
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Years Ended December 31, | ||||||||
|
2023 |
|
|
2022 |
|
|||
| Revenue | $ |
48,727 |
|
$ |
25,098 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
53,375 |
|
|
46,122 |
|
||
| General and administrative |
|
39,088 |
|
|
41,284 |
|
||
| Total operating expenses |
|
92,463 |
|
|
87,406 |
|
||
| Operating loss |
|
(43,736 |
) |
|
(62,308 |
) |
||
| Other income (expense): | ||||||||
| Impairment charges |
|
— |
|
|
(10,844 |
) |
||
| Loss on disposal of assets |
|
(461 |
) |
|
(30 |
) |
||
| Gain (loss) on changes in fair value |
|
1,145 |
|
|
(510 |
) |
||
| Loss from equity method investment |
|
(4,931 |
) |
|
(1,579 |
) |
||
| Interest expense |
|
(2,230 |
) |
|
(1,111 |
) |
||
| Interest income |
|
7,686 |
|
|
3,473 |
|
||
| Total other income (expense) |
|
1,209 |
|
|
(10,601 |
) |
||
| Loss from continuing operations | $ |
(42,527 |
) |
$ |
(72,909 |
) |
||
| Loss from discontinued operations (including gain on disposal of |
|
(18,792 |
) |
|
(38,728 |
) |
||
| Net Loss | $ |
(61,319 |
) |
$ |
(111,637 |
) |
||
| Net loss per share attributable to common stockholders-basic and diluted | $ |
(15.96 |
) |
$ |
(38.10 |
) |
||
| Weighted average shares of common stock outstanding-basic and diluted |
|
3,841,405 |
|
|
2,929,873 |
|
||
| Precision Biosciences, Inc. | ||||||
| Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| December 31, 2023 | December 31, 2022 | |||||
| Cash and cash equivalents | $ |
116,678 |
$ |
189,576 |
||
| Working capital |
|
86,372 |
|
139,441 |
||
| Total assets |
|
159,781 |
|
238,169 |
||
| Total liabilities |
|
140,920 |
|
177,736 |
||
| Total stockholders' equity | $ |
18,861 |
$ |
60,433 |
||
| Common stock outstanding |
|
4,164,038 |
|
3,698,674 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20240327674339/en/
Investor and Media Contact:
Naresh Tanna
Vice President, Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What did Precision BioSciences announce regarding PBGENE-HBV?
What licensing agreements did Precision BioSciences complete?
What was the purpose of the $40 million public offering by Precision BioSciences?
What are Precision BioSciences' plans for PBGENE-HBV and PBGENE-PMM?
What objectives does Precision BioSciences aim to achieve with ARCUS in 2024?
