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Precision BioSciences Reports Fourth Quarter and Fiscal Year 2025 Financial Results and Provides Business Update
- PBGENE-HBV Phase 1 data featured Late Breaker presentation at AASLD, The Liver Meeting, showing safety, tolerability and cumulative, dose-dependent antiviral activity -
- Strong Phase 1 ELIMINATE-B trial execution for PBGENE-HBV with 13 patients now dosed across first 5 cohorts; Data updates expected at medical conferences throughout 2026 -
- Received IND Clearance for PBGENE-DMD enabling IRB and site activation for Phase 1/2 FUNCTION-DMD trial; Data from multiple patients expected by year-end 2026 -
- Raised
“2025 was an exceptional year for
“Additionally, our team completed all Investigational New Drug (IND) enabling activities for PBGENE-DMD and filed an IND application by the end of 2025 after announcing prioritization of the program in
Wholly Owned Portfolio:
PBGENE-HBV (Hepatitis B 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 be a potentially curative treatment for chronic Hepatitis B infection. In patients with chronic hepatitis B, cccDNA acts as the template to make new infectious viral particles. PBGENE-HBV is the only clinical stage program that targets the elimination of cccDNA, the sole source of viral replication, leading to sustained loss of HBV DNA and other downstream viral transcripts.
On
As part of the ongoing assessment of the safety and efficacy profile of PBGENE-HBV after repeat doses in Part 1 dose finding, Precision has administered additional doses in Cohort 3 and in parallel commenced pre-planned additional cohorts to investigate a shorter dosing interval. Cohort 4 is investigating dosing at 0.4 mg/kg every 4 weeks and Cohort 5 is investigating dosing at 0.65 mg/kg every 4 weeks to evaluate the potential for an optimized therapeutic index. In addition, to mitigate acute infusion reactions common to lipid nanoparticle (LNP) delivered therapies, such as transient hypotension and transient elevated liver enzymes, Precision continues to investigate prophylactic measures per protocol. These measures include intravenous (IV) fluids, steroids, antihistamines, and infusion duration across dose levels and administrations. The goal during Part 1 of the study is to select the dose and schedule that achieves the desired therapeutic index to move to the expansion phase of the ELIMINATE-B trial.
PBGENE-HBV is the first in vivo gene editing approach to prospectively employ repeat administrations of LNP. To date, 13 participants have completed more than 30 administrations of PBGENE-HBV across five cohorts. Looking ahead, Precision expects additional clinical biomarker and biopsy data in the first half of 2026 and expects to have completed dosing in Cohorts 3, 4, and 5. This will inform selection of an optimal dosing regimen intended to support discontinuation of nucleos(t)ide analog treatment and progression into the Part 2 expansion phase of ELIMINATE-B. Precision expects to share further clinical data from the PBGENE-HBV program at hepatitis-focused medical conferences throughout 2026.
PBGENE-DMD (Muscle Targeted Gene Excision Program)
PBGENE-DMD is Precision’s development program for the treatment of Duchenne muscular dystrophy (DMD). In
PBGENE-DMD received
New preclinical study data supporting the potential long-term efficacy of PBGENE-DMD was presented in a poster session at the
On
Partnered In Vivo Gene Editing 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. Recently announced alignment with the FDA on key study elements could support a potential Biologics License Application (BLA). In addition, ECUR-506 was granted FDA Regenerative Medicine Advanced Therapy (RMAT) designation. The OTC-HOPE study is ongoing in the
PBGENE-NVS (Gene Insertion Program)
As previously reported in
Non-Core Ex Vivo Programs:
Azer-Cel (Azercabtagene Zapreleucel Allogeneic CAR T Treatment or Cancer)
Imugene Limited continues development of Azer-Cel in diffuse large B-cell lymphoma and has received written guidance from the FDA regarding the registrational pathway for azer-cel. The guidance provided clear alignment with the FDA across key elements required to support advancement into a pivotal study, including dosing regimen, patient population, endpoints, and manufacturing readiness. In
Azer-Cel (Azercabtagene Zapreleucel Allogeneic CAR T Treatment for Multiple Sclerosis)
Separately, Azer-Cel is being evaluated by
Corporate Updates:
In
New Intellectual Property Notices
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
Other Income (Expense): Total other income for the quarter ended
Net Income (Loss) from Continuing Operations: Net income from continuing operations was
Net Income (Loss): Net income was
Fiscal Year 2025 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 (Expense): Total other income was
Net (Loss) Income from Continuing Operations: Net loss from continuing operations was
Net (Loss) Income: Net loss was
Shares: Basic and diluted weighted-average common shares outstanding for the year ended
About Chronic Hepatitis B
Chronic hepatitis B virus causes inflammation and damage to the liver, leading to chronic infection and increased risk of death from liver cancer or cirrhosis. There is no cure for chronic hepatitis B, and current treatments rarely result in a functional cure, primarily due to persistence of viral DNA in the liver. In patients with chronic hepatitis B, genetic material of the virus is converted within infected liver cells into cccDNA that acts as the only template to make new infectious viral particles. Hepatitis B virus also inserts fragments of its DNA into the human genome of infected liver cells. These integrated fragments are viral replication incompetent and cannot produce new infectious virus. Both cccDNA and integrated HBV DNA produce the viral protein, hepatitis B surface antigen (“HBsAg”), which is secreted in the blood.
Historically, the focus for drug development and regulatory approval of drugs for chronic hepatitis B has relied on the temporary suppression of HBsAg. Achieving undetectable HBsAg may lead to a functional cure if there is no rebound in HBV DNA or HBsAg after drug treatment has been discontinued for at least six months, but this is achieved in less than three out of 100 patients treated with the current standard of care. Since cccDNA is the only source of infectious particles (HBV DNA), elimination of cccDNA results in a cure of chronic hepatitis B. Sustained loss of HBV DNA alone as a result of cccDNA elimination is an approvable endpoint for the FDA and highly relevant for PBGENE-HBV.
About PBGENE-HBV, A 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 be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic Hepatitis B, cccDNA, while inactivating integrated HBV DNA. Elimination of cccDNA results in HBV cure as cccDNA is the only source of infectious replication (HBV DNA). The ELIMINATE-B trial is investigating PBGENE-HBV at multiple dose levels across a number of administrations per dose level in patients with chronic Hepatitis B. PBGENE-HBV has been granted
PBGENE-HBV is the only clinical stage program targeting the elimination of cccDNA leading to sustained loss of HBV DNA. The FDA has previously provided guidance that sustained loss of HBV DNA is an approvable endpoint for chronic hepatitis B.
Further details on the trial can be found on Precision’s website and on clinicaltrials.gov identifier NCT06680232.
About PBGENE-DMD, A Muscle-Targeted Excision Program
PBGENE-DMD is Precision’s development program for the treatment of DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the
PBGENE-DMD was granted Orphan Drug Designation by the FDA in July, 2025. The PBGENE-DMD program is eligible for a Priority Review Voucher (PRV) via the Rare Pediatric Disease Priority Review Voucher (PRV) program, which was signed into law on
Further details on the trial can be found on Precision’s website and on clinicaltrials.gov identifier NCT07429240.
About
The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination (removing a genome e.g. viral DNA such as in the Company’s PBGENE-HBV program), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV such as in the Company’s PBGENE-DMD program) and gene insertion (inserting DNA into gene to cause expression/add function).
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 key advantages of ARCUS and its key capabilities and differentiating characteristics; expectations about operational initiatives, strategies, further development, or timing of additional updates or data releases of PBGENE-HBV and PBGENE-DMD, timing and progress of IRB processes and site activations following IND clearance for the PBGENE-DMD program and FUNCTION-DMD trial; administration of additional doses in Cohort 3 and in parallel commencement of pre-planned additional cohorts to investigate a shorter dosing interval as part of the ongoing assessment of the safety and efficacy profile of PBGENE-HBV after repeat doses in Part 1 dose finding; investigation of dosing at 0.4 mg/kg every 4 weeks and dosing at 0.65 mg/kg every 4 weeks in Cohort 5 to evaluate the potential for an optimized therapeutic index; continued investigation of prophylactic measures per protocol to mitigate acute infusion reactions common to lipid nanoparticle (LNP) delivered therapies, such as transient hypotension and transient elevated liver enzymes, measures including IV fluids, steroids, antihistamines and infusion duration across dose levels and administrations; the goal during Part 1 of the study is to select the dose and schedule that achieves the desired therapeutic index to move to the expansion phase of the ELIMINATE-B trial; expectations of additional clinical biomarker and biopsy data in the first half of 2026 in the ELIMINATE-B trial and expectations to have completed dosing in Cohorts 3, 4, and 5, selection of an optimal dosing regimen intended to support discontinuation of nucleos(t)ide analog treatment and progression into the Part 2 expansion phase; expectations to share further clinical data from the PBGENE-HBV programs at hepatitis-focused medical conferences throughout 2026; the design of PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to cure; the suitability of PBGENE-HBV for the treatment of hepatitis B and the targeting of the root cause of the disease; expectations concerning the receipt of initial clinical data from multiple patients for PBGENE-DMD by year-end 2026 including early efficacy assessment based on near full-length dystrophin protein expression from muscle biopsies; eligibility of PBGENE-DMD Fast Track designation for more frequent meetings and communications with the FDA, rolling review of any application for marketing approval, Priority Review if relevant criteria are met; the planned KOL event providing an overview of the FUNCTION-DMD trial; the design of PBGENE-DMD to improve function over time and address more than 60% of patients with DMD; the potential for PBGENE-DMD to provide durable functional improvement with a single dose of AAV; translation of results in preclinical studies of ARCUS nucleases to clinical studies in humans; the preclinical and clinical development and demonstrated, potential and expected safety, efficacy, durability, and benefit of PBGENE-HBV and PBGENE-DMD, as well as our other product candidates and those being developed by partners; expectations of additional data from the ongoing OTC-HOPE clinical trial in the first half of 2026; expectations of
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 |
||||||||
|
|
2025 |
|
|
2024 |
|
|||
| Revenue |
$ |
34,204 |
|
$ |
638 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
14,464 |
|
|
15,907 |
|
||
| General and administrative |
|
7,232 |
|
|
9,577 |
|
||
| Total operating expenses |
|
21,696 |
|
|
25,484 |
|
||
| Operating income (loss) |
|
12,508 |
|
|
(24,846 |
) |
||
| Other income | ||||||||
| Loss from equity method investment |
|
(5,370 |
) |
|
(972 |
) |
||
| Loss on changes in other fair value adjustments |
|
(248 |
) |
|
(659 |
) |
||
| Gain on change in fair value of warrant liability |
|
12,359 |
|
|
7,812 |
|
||
| Interest expense |
|
(348 |
) |
|
(392 |
) |
||
| Interest income |
|
773 |
|
|
1,494 |
|
||
| Loss on disposal of assets |
|
(390 |
) |
|
(182 |
) |
||
| Impairment charges |
|
(36 |
) |
|
— |
|
||
| Total other income |
|
6,740 |
|
|
7,101 |
|
||
| Income (loss) from continuing operations |
$ |
19,248 |
|
$ |
(17,745 |
) |
||
| Gain from discontinued operations |
|
885 |
|
|
— |
|
||
| Net income (loss) |
$ |
20,133 |
|
$ |
(17,745 |
) |
||
| Net income (loss) per share | ||||||||
| Basic |
$ |
1.06 |
|
$ |
(2.22 |
) |
||
| Diluted |
$ |
1.05 |
|
$ |
(2.22 |
) |
||
| Weighted-average shares of common stock outstanding | ||||||||
| Basic |
|
19,051,307 |
|
|
7,999,288 |
|
||
| Diluted |
|
19,171,669 |
|
|
7,999,288 |
|
||
| Statements of Operations | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| For the Years Ended |
||||||||
|
|
2025 |
|
|
2024 |
|
|||
| Revenue |
$ |
34,264 |
|
$ |
68,696 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
54,172 |
|
|
59,559 |
|
||
| General and administrative |
|
32,240 |
|
|
35,299 |
|
||
| Total operating expenses |
|
86,412 |
|
|
94,858 |
|
||
| Operating loss |
|
(52,148 |
) |
|
(26,162 |
) |
||
| Other income | ||||||||
| Loss from equity method investment |
|
(5,284 |
) |
|
(1,084 |
) |
||
| (Loss) gain on changes in other fair value adjustments |
|
(2,666 |
) |
|
258 |
|
||
| Gain on change in fair value of warrant liability |
|
11,129 |
|
|
29,610 |
|
||
| Interest expense |
|
(1,422 |
) |
|
(1,782 |
) |
||
| Interest income |
|
4,239 |
|
|
6,763 |
|
||
| Loss on disposal of assets |
|
(421 |
) |
|
(436 |
) |
||
| Impairment charges |
|
(36 |
) |
|
— |
|
||
| Total other income |
|
5,539 |
|
|
33,329 |
|
||
| (Loss) income from continuing operations |
$ |
(46,609 |
) |
$ |
7,167 |
|
||
| Gain from discontinued operations |
|
885 |
|
|
— |
|
||
| Net (loss) income |
$ |
(45,724 |
) |
$ |
7,167 |
|
||
| Net (loss) income per share | ||||||||
| Basic |
$ |
(3.56 |
) |
$ |
1.05 |
|
||
| Diluted |
$ |
(3.56 |
) |
$ |
1.04 |
|
||
| Weighted-average shares of common stock outstanding | ||||||||
| Basic |
|
12,826,078 |
|
|
6,832,982 |
|
||
| Diluted |
|
12,826,078 |
|
|
6,883,911 |
|
||
| Balance Sheets Data | ||||||
| (In thousands, except share amounts) | ||||||
| Cash, cash equivalents, and restricted cash |
$ |
137,153 |
$ |
108,468 |
||
| Working capital |
|
109,827 |
|
80,009 |
||
| Total assets |
|
154,416 |
|
136,388 |
||
| Total liabilities |
|
62,168 |
|
79,995 |
||
| Total stockholders' equity |
$ |
92,248 |
$ |
56,393 |
||
| Common stock outstanding |
|
24,088,425 |
|
8,202,715 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20260312974135/en/
Investor and Media Contact:
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
Source: