Precision BioSciences Reports First Quarter 2026 Financial Results and Provides Business Update
Key Terms
phase 1/2a medical
cccDNA medical
lipid nanoparticle (lnp) medical
investigational new drug (ind) regulatory
fast track designation regulatory
clinical trial application regulatory
biologics license application (bla) regulatory
at-the-market (atm) facility financial
- Continued advancement of the Phase 1/2a ELIMINATE-B trial of PBGENE-HBV across multiple dosing cohorts; Late-Breaking poster presentation for PBGENE-HBV accepted at European Association for the Study of the Liver Congress 2026 -
- Advanced PBGENE-DMD toward clinical evaluation in the Phase 1/2 FUNCTION-DMD trial following
- Cash balance of
“During the first quarter, we continued to execute on our two clinical stage in vivo gene editing programs. We advanced PBGENE-HBV into new cohorts in the ELIMINATE-B trial and drove PBGENE-DMD through Investigational New Drug (IND) approval and activated our first clinical site as we prepare to dose the first patient in the FUNCTION-DMD trial,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “We remain focused on disciplined execution in 2026 as we generate additional clinical data from the ELIMINATE-B trial and enroll more sites and patients in the FUNCTION-DMD trial. We believe our ARCUS platform and targeted development strategy position us to deliver multiple important clinical and operational milestones this year.”
Wholly Owned Portfolio:
PBGENE-HBV (Hepatitis B Viral Elimination Program)
PBGENE-HBV is Precision’s wholly owned in vivo gene editing program being evaluated in a global first-in-human clinical trial as a potential curative treatment for chronic hepatitis B. 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. PBGENE-HBV is the first in vivo gene editing approach to prospectively employ repeat administrations of lipid nanoparticle (LNP) in chronic hepatitis B.
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 treated 16 patients with 38 administrations of PBGENE-HBV across five cohorts evaluating the impact of escalating dose levels as well as 8-week and 4-week dosing intervals. 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.
Looking ahead, Precision expects to share further clinical data from the PBGENE-HBV program at hepatitis-focused medical conferences throughout 2026, starting with the European Association for the Study of the Liver (EASL).
On April 22nd, Precision BioSciences announced that a late-breaking poster for PBGENE-HBV was accepted for presentation at the EASL Congress 2026 taking place on May 27-30 in
In addition, in April the Company announced that it received Clinical Trial Application approval to expand ELIMINATE-B into
In March, Precision BioSciences received two Notices of Allowance from the
PBGENE-DMD (Muscle Targeted Gene Excision Program)
PBGENE-DMD is Precision’s development program for Duchenne muscular dystrophy (DMD), designed to restore a near full-length functional dystrophin protein through a gene excision approach using two ARCUS nucleases delivered in a single AAV.
Following
Arkansas Children’s is a Parent Project Muscular Dystrophy (PPMD)-certified Duchenne Care Center, recognized for delivering specialized, multidisciplinary care for patients with DMD. PPMD’s Certified Duchenne Care Center Program is intended to help ensure that participating centers maintain high standards in clinical and sub-specialty services, rapidly incorporate evidence-based knowledge, and provide standardized multidisciplinary Duchenne care. Additionally, Arkansas Children’s is a designated Muscular Dystrophy Association (MDA) Care Center, providing specialized, multidisciplinary care for neuromuscular diseases including diagnosis, personalized treatment plans, and comprehensive support for patients.
In March 2026, PBGENE-DMD also received FDA Fast Track designation and hosted a DMD investor event, the replay is accessible here.
In addition, Precision presented preclinical PBGENE-DMD data highlighting durable dystrophin expression and functional benefit at the Muscular Dystrophy Association Clinical & Scientific Conference 2026 in March.
Partnered In Vivo Programs:
iECURE-OTC (Gene Insertion Program)
Led by iECURE, Inc. (iECURE) ECUR-506 is an ARCUS-mediated in vivo targeted gene insertion program currently in a first-in-human trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. iECURE previously announced alignment with the FDA on key study elements could support a potential Biologics License Application (BLA). The OTC-HOPE study is ongoing in the
iECURE expects to present clinical data from the ongoing OTC-HOPE clinical trial at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 11-15, 2026 in
Partnered Ex Vivo Programs:
Azer-Cel (Azercabtagene Zapreleucel Allogeneic CAR T Treatment for 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. Azer-Cel has been selected for oral presentation at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting being held on May 29-June 2, in
Azer-Cel (Azercabtagene Zapreleucel Allogeneic CAR T Treatment for Multiple Sclerosis)
Separately, Azer-Cel is being evaluated by TG Therapeutics, Inc. (Nasdaq: TGTX) in a Phase 1 trial in progressive multiple sclerosis. In April 2026, Precision received a clinical milestone cash payment under its license agreement with TG Therapeutics. The payment of
Quarter Ended March 31, 2026 Financial Results:
Cash, Cash Equivalents, and Restricted Cash: As of March 31, 2026, Precision had approximately
Revenues: Total revenues for the quarter ended March 31, 2026, were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Other (Expense) Income: Total other expense was
Additionally, in the quarter ended March 31, 2026, there was no gain or loss recognized from the Company’s equity method investment compared to
Net Loss: Net loss was
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 Fast Track designation by the FDA.
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 February 3, 2026, as part of the Consolidated Appropriations Act of 2026. PBGENE-DMD received Fast Track designation from the FDA in February 2026.
Further details on the trial can be found on Precision’s website and on clinicaltrials.gov identifier NCT07429240.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage 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. These features are intended for ARCUS nucleases to drive more defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of clinical stage 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 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; administrations of PBGENE-HBV across cohorts and the evaluation on the impact of escalating dose levels and dosing intervals; 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 to share further clinical data from the PBGENE-HBV programs at hepatitis-focused medical conferences throughout 2026; planned site initiation activities and initial patient screening expected in the second quarter of 2026 in
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 March 31, |
||||||||
|
2026 |
|
|
2025 |
|
|||
| Revenue | $ |
10,838 |
|
$ |
29 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
13,110 |
|
|
13,588 |
|
||
| General and administrative |
|
6,803 |
|
|
8,553 |
|
||
| Total operating expenses |
|
19,913 |
|
|
22,141 |
|
||
| Operating loss |
|
(9,075 |
) |
|
(22,112 |
) |
||
| Other (expense) income: | ||||||||
| Gain from equity method investment |
|
- |
|
|
1,342 |
|
||
| (Loss) gain on changes in other fair value adjustments |
|
(2,951 |
) |
|
49 |
|
||
| Loss on change in fair value of warrant liability |
|
(7,107 |
) |
|
(804 |
) |
||
| Interest expense |
|
(311 |
) |
|
(354 |
) |
||
| Interest income |
|
999 |
|
|
1,323 |
|
||
| Gain (loss) on disposal of assets |
|
4 |
|
|
(9 |
) |
||
| Total other (expense) income |
|
(9,366 |
) |
|
1,547 |
|
||
| Net loss | $ |
(18,441 |
) |
$ |
(20,565 |
) |
||
| Net loss per share | ||||||||
| Basic | $ |
(0.75 |
) |
$ |
(2.21 |
) |
||
| Diluted | $ |
(0.75 |
) |
$ |
(2.21 |
) |
||
| Weighted-average shares of common stock outstanding | ||||||||
| Basic |
|
24,634,443 |
|
|
9,292,066 |
|
||
| Diluted |
|
24,634,443 |
|
|
9,292,066 |
|
||
| Precision Biosciences, Inc. | ||||||||
| Balance Sheets Data | ||||||||
| (In thousands, except share amounts) | ||||||||
| March 31, 2026 | December 31, 2025 | |||||||
| Cash, cash equivalents, and restricted cash | $ |
125,847 |
$ |
137,153 |
||||
| Working capital |
|
100,938 |
|
|
109,827 |
|
||
| Total assets |
|
143,891 |
|
|
154,416 |
|
||
| Total liabilities |
|
67,790 |
|
|
62,168 |
|
||
| Total stockholders' equity | $ |
76,101 |
|
$ |
92,248 |
|
||
| Common stock outstanding |
|
25,805,898 |
|
|
24,088,425 |
|
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20260505850322/en/
Investor and Media Contact:
Naresh Tanna
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.