INVESTORS
Press Releases
Precision BioSciences Reports Fourth Quarter and Fiscal Year 2024 Financial Results and Provides Business Update
- Announced first in vivo clinical validation for ARCUS gene editing across two distinct programs including wholly owned PBGENE-HBV and partnered program ECUR-506 -
- Commenced Phase 1 ELIMINATE-B clinical trial for PBGENE-HBV, the first gene editing technology studied for Hepatitis B, in fourth quarter of 2024 -
- Demonstrated PGENE-HBV was safe and well tolerated with substantial antiviral activity measured by reduction of Hepatitis B surface antigen after only one administration at lowest dose level; Subsequent doses of PBGENE-HBV to follow with data expected throughout 2025 -
- Announced complete clinical response in first infant dosed by partner iECURE in ongoing Phase 1/2 clinical trial in Ornithine Transcarbamylase (OTC) Deficiency -
- Anticipated cash runway into the second half of 2026 is expected to enable achievement of key Phase 1 clinical inflection points across both wholly owned in vivo gene editing programs -
“2024 was a transformational year for
“In 2025, our steadfast focus remains on continued clinical execution as we work to deliver on the promise of our ARCUS gene editing technology through robust clinical data,” continued
Wholly Owned Portfolio
PBGENE-HBV (Viral Elimination Program): PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to potentially cure chronic Hepatitis B infection. Currently, it is estimated that approximately 300 million people worldwide are afflicted with chronic Hepatitis B. Unlike all other downstream targeting modalities, which offer low likelihood of achieving a cure, PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA. The ELIMINATE-B trial is designed to investigate PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients afflicted with chronic Hepatitis B who are HBeAg-negative.
Precision received IND and CTA approvals for its Phase 1 ELIMINATE-B trial in
The Company dosed the first patient in
The study is primarily designed to test the safety of three dose administrations of PBGENE-HBV. In the first cohort, all three patients dosed with the first dose administration of PBGENE-HBV have completed the initial safety evaluation period. PBGENE-HBV was well-tolerated and none of the patients experienced a Grade ≥2 treatment-related adverse event or serious adverse event.
In addition to safety, the ELIMINATE-B protocol is designed to assess the efficacy for three dose administrations at each dose level, with the goal to maximize cumulative viral editing to achieve undetectable levels of HBsAg ultimately enabling patients to stop taking lifelong nucleos(t)ide analog therapy. PBGENE-HBV demonstrated a substantial reduction in HBsAg in two of the three participants following the first administration at the lowest dose. Initial clinical data in the first cohort of patients was consistent with the HBsAg reductions observed in preclinical non-human primate models.
“Given PBGENE-HBV's novel modality, these data suggest that PBGENE-HBV appears to be working by its intended mechanism of eliminating the source of viral replication in cccDNA while inactivating integrated disease,” said
With a well-tolerated safety profile and early antiviral activity established after the first administration at dose level 1, Precision expects to complete subsequent administrations in all cohort 1 patients while in parallel expanding to the next higher dose cohorts. The Company plans to provide ongoing updates on the full low-dose cohort, including multiple dose administrations, and data at higher dose levels throughout 2025.
Supporting the ELIMINATE-B clinical study design, Precision presented new preclinical safety and efficacy data at the Global Hepatitis Summit (GHS) on
PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program): PBGENE-3243 is a first-of-its-kind potential treatment for m.3243-associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA, thereby eliminating the root cause of the disease. Currently, there are no cures for m.3243-associated mitochondrial disease, which affects approximately 20,000 people in the
The high specificity of ARCUS nucleases enables editing and elimination of mutant mitochondrial DNA while allowing wild-type (normal) mitochondrial DNA to repopulate, thus improving cellular function. Unlike CRISPR/Cas, base editors, and prime editors that require a guide RNA, ARCUS single-component nucleases are able to penetrate the mitochondrial membranes and target mutant mitochondrial DNA. In 2025, Precision plans to present new data for PBGENE-3243 while advancing the program toward a CTA and/or IND.
Wholly Owned Portfolio – Under Assessment
In
-
PBGENE-DMD (Muscle Targeted Excision Program): New preclinical, in vivo efficacy data using the clinical construct, PBGENE-DMD, was presented at the 2025
Muscular Dystrophy Association Clinical and Scientific Conference onMarch 19, 2025 . The oral presentation highlighted significant functional dystrophin protein production across heart, diaphragm and skeletal muscles at levels expected to provide therapeutic benefit. In a humanized DMD-diseased mouse model, PBGENE-DMD demonstrated long-term functional improvement over multiple time points, including achieving 93% of the maximal force output observed in healthy control mice. Importantly, PBGENE-DMD demonstrated the ability to edit Pax7+ cells, a marker for satellite muscle stem cells which are the precursor cells to new muscle cell formation and a potential predictor of durable functional benefit. These in vivo efficacy results further support the therapeutic potential of an ARCUS gene editing approach for the treatment of DMD and ongoing development in clinical trials. - PBGENE-CNS (CNS Targeted Excision Program): Precision expects to publicly present preclinical data on PBGENE-CNS for the first time at a scientific conference in 2025.
ARCUS Platform
At the ESGCT 31st Annual Congress in
Partnered Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediated in vivo gene editing program currently in a first-in-human Phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. In
The OTC-HOPE study is ongoing in 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.
Corporate Updates
Strengthened Senior Leadership Team: In
Business Updates – Monetization of CAR T Investments:
In
In
Quarter Ended
Cash, Cash Equivalents, and Restricted Cash: 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
Net Loss from Continuing Operations: Net loss from continuing operations was
Net Loss: Net loss was
Fiscal Year 2024 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
Net Income from Continuing Operations: Net income from continuing operations was
Net Income: Net income was
Shares: Basic and diluted weighted-average common shares outstanding the year ended
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), 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’ and licensees’ product candidates and gene editing approaches including editing efficiency, and the suitability of ARCUS nucleases for gene insertion, gene elimination and gene excision and differentiation from other gene editing approaches; the expected timing of regulatory processes and clinical operations (including filings, studies, enrollment and clinical data for PBGENE-HBV, PBGENE-3243 and iECURE OTC); the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; clinical data suggesting that PBGENE-HBV appears to be working by its intended mechanism of eliminating the source of viral replication in cccDNA while inactivating integrated disease; plans to provide ongoing updates on the full low-dose cohort for the PBGENE-HBV study, including multiple dose administrations, and data at higher dose levels throughout 2025; the ability of ARCUS single-component nucleases to penetrate the mitochondrial membranes; the high specificity of ARCUS nucleases enables editing and elimination of mutant mitochondrial DNA while allowing wild-type (normal) mitochondrial DNA to repopulate, thus improving cellular function; plans to present new data for PBGENE-3243 in 2025 while advancing the program toward a CTA and/or IND; the ability of PBGENE-DMD to provide significant functional dystrophin protein production across heart, diaphragm and skeletal muscles at levels expected to provide therapeutic benefit in a humanized DMD-diseased mouse model; the ability of PBGENE to edit satellite muscle stem cells, a potential predictor of durable functional benefit; the translation of results in preclinical studies of ARCUS nucleases to clinical studies in humans; expectations about our and our partners’ operational initiatives, strategies, and further development of our programs; expectations and updates around our partnerships and collaborations and our ability to enter into new collaborations, license agreements or other arrangements; our expected cash runway and available credit; the sufficiency of our cash runway extending into the second half of 2026 and realizing Phase 1 clinical data for multiple in vivo gene editing 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 clinical data . In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “appear,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “suggest,” “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 |
||||||||
|
|
2024 |
|
|
2023 |
|
|||
| Revenue |
$ |
638 |
|
$ |
7,038 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
15,907 |
|
|
13,389 |
|
||
| General and administrative |
|
9,577 |
|
|
8,539 |
|
||
| Total operating expenses |
|
25,484 |
|
|
21,928 |
|
||
| Operating loss |
|
(24,846 |
) |
|
(14,890 |
) |
||
| Other income (expense): | ||||||||
| Loss from equity method investment |
|
(972 |
) |
|
(871 |
) |
||
| (Loss) gain on changes in fair value |
|
(659 |
) |
|
1,603 |
|
||
| Gain on change in fair value of warrant liability |
|
7,812 |
|
|
— |
|
||
| Interest expense |
|
(392 |
) |
|
(579 |
) |
||
| Interest income |
|
1,494 |
|
|
1,827 |
|
||
| Loss on disposal of assets |
|
(182 |
) |
|
(524 |
) |
||
| Total other income |
|
7,101 |
|
|
1,456 |
|
||
| Loss from continuing operations |
$ |
(17,745 |
) |
$ |
(13,434 |
) |
||
| Loss from discontinued operations |
|
— |
|
|
(2,855 |
) |
||
| Net loss |
$ |
(17,745 |
) |
$ |
(16,289 |
) |
||
| Net loss per share-basic and diluted |
$ |
(2.22 |
) |
$ |
(4.06 |
) |
||
| Weighted average shares of common stock outstanding-basic and diluted |
|
7,999,288 |
|
|
4,010,067 |
|
||
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Years Ended |
||||||||
|
|
2024 |
|
|
2023 |
|
|||
| Revenue |
$ |
68,696 |
|
$ |
48,727 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
59,559 |
|
|
53,375 |
|
||
| General and administrative |
|
35,299 |
|
|
39,088 |
|
||
| Total operating expenses |
|
94,858 |
|
|
92,463 |
|
||
| Operating loss |
|
(26,162 |
) |
|
(43,736 |
) |
||
| Other income (expense): | ||||||||
| Loss from equity method investment |
|
(1,084 |
) |
|
(4,931 |
) |
||
| Gain on changes in fair value |
|
258 |
|
|
1,145 |
|
||
| Gain on change in fair value of warrant liability |
|
29,610 |
|
|
— |
|
||
| Interest expense |
|
(1,782 |
) |
|
(2,230 |
) |
||
| Interest income |
|
6,763 |
|
|
7,686 |
|
||
| Loss on disposal of assets |
|
(436 |
) |
|
(461 |
) |
||
| Total other income |
|
33,329 |
|
|
1,209 |
|
||
| Income (loss) from continuing operations |
$ |
7,167 |
|
$ |
(42,527 |
) |
||
| Loss from discontinued operations (including gain on disposal of |
|
— |
|
|
(18,792 |
) |
||
| Net income (loss) |
$ |
7,167 |
|
$ |
(61,319 |
) |
||
| Net income (loss) per share | ||||||||
| Basic |
$ |
1.05 |
|
$ |
(15.96 |
) |
||
| Diluted |
$ |
1.04 |
|
$ |
(15.96 |
) |
||
| Weighted-average shares of common stock outstanding | ||||||||
| Basic |
|
6,832,982 |
|
|
3,841,405 |
|
||
| Diluted |
|
6,883,911 |
|
|
3,841,405 |
|
||
| Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| Cash, cash equivalents, and restricted cash |
$ |
108,468 |
$ |
116,678 |
||
| Working capital |
|
80,009 |
|
86,372 |
||
| Total assets |
|
136,388 |
|
159,781 |
||
| Total liabilities |
|
79,995 |
|
140,920 |
||
| Total stockholders' equity |
$ |
56,393 |
$ |
18,861 |
||
| Common stock outstanding |
|
8,202,715 |
|
4,164,038 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20250326544666/en/
Investor and Media Contact:
Vice President,
Naresh.Tanna@precisionbiosciences.com
Source: