INVESTORS
Press Releases
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; expect to submit IND and/or CTA in 2024
- Completed licensing agreements to monetize divested CAR T assets with
- Completed
“2023 was a transformative year for
“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
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
In
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
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
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 40% to 45% overall gene insertion efficiency at 1- and 3-months. Precision scientists largely attribute this high efficiency to the unique ARCUS cut type which drives homology directed repair, even in nondividing cells.
Business Updates – Monetization of CAR T Investments:
Completed Licensing Deal with
In
Completed Licensing Deal with
The agreement with
Completed Non-Exclusive Patent License Agreement with
In
In total, these three transactions provide
Business Updates –
On
Quarter Ended
Cash and Cash Equivalents: As of
Revenues: Total revenues for the quarter ended
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
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
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.
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Three Months Ended |
||||||||
|
|
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 |
|
||
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Years Ended |
||||||||
|
|
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 |
|
||
| Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| 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:
Vice President, Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: