Precision BioSciences Announces Presentation of Initial Safety Data from the Phase 1 ELIMINATE-B Trial Evaluating PBGENE-HBV at the 2025 European Association for the Study of the Liver Congress (EASL)

Business Wire

07-05-2025

BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that it will present initial safety data from the Phase 1 ELIMINATE-B trial evaluating PBGENE-HBV program for the treatment of chronic hepatitis B during a late breaking poster presentation at the European Association for the Study of the Liver (EASL) Congress being held May 7-10, 2025, in Amsterdam, Netherlands.
'We are pleased to share initial safety data from the ELIMINATE-B trial for PBGENE-HBV at this year's EASL congress. Since initiating the trial, we have made rapid progress activating global clinical sites and enrolling patients as we seek to validate our ARCUS-elimination approach in the clinic for chronic hepatitis B,' said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. 'This initial safety dataset across the first three patients receiving repeat administrations (two doses) as planned in the ELIMINATE-B protocol provide early validation of the translation of preclinical pharmacokinetic and safety data in patients. We are encouraged by the signals we are seeing in the study thus far and believe these data continue to support multiple dose administrations of PBGENE-HBV as well as ongoing dose escalation. This new data builds on the update shared in February and we look forward to continuing to enroll patients in the ELIMINATE-B trial and sharing further updates, including antiviral efficacy data upon completion of each cohort, three dose administrations at each dose level, throughout 2025.'
Presentation Details:
Title: Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B
Session: Late Breaker Posters
Poster Number: LBP-038
Date: Late breaker posters will be displayed from May 7-10
PBGENE-HBV is Precision's first-in-class gene editing therapy for chronic hepatitis B being evaluated in the global Phase 1 ELIMINATE-B study. ELIMINATE-B participants are Hepatitis B e-antigen negative with chronic hepatitis B and virologically suppressed on nucleos(t)ide analog (NA) treatment. Initial study participants were enrolled at a study site in Moldova. The first cohort comprised of three participants, all of whom were male with a mean age of 41 years and range of 5.9-7.2 years on NA treatment. The participants varied in their duration of infection, from 7.5 to 39 years, and their baseline HBsAg levels, which were between 561 and 11,813 IU/mL. The three participants received dose level 1 (0.2 mg/kg) and have received 2 of the 3 dose administrations. PBGENE-HBV was well-tolerated upon repeat dosing, with no dose limiting toxicities, serious adverse events, or clinically significant laboratory abnormalities. All adverse events were mild (grade 1 or 2) and transient in nature, and there were no cumulative adverse events with a second dose administration. Initial safety data from the first cohort indicates the translation of PBGENE-HBV nonclinical pharmacokinetic and safety data from non-human primates into the clinic and supports pre-planned repeat dosing as well as dose escalation of PBGENE-HBV with the goal of moving appropriate dose and schedule into Phase 2 expansion.
In April 2025, the FDA granted Fast Track designation to PBGENE-HBV for chronic hepatitis B. Precision is approved for clinical investigation of its Phase 1 ELIMINATE-B trial in the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. The Company plans to continue sharing updates on full dose cohorts at all dose levels throughout 2025.
About Hepatitis B
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. Despite the availability of approved antiviral therapies, an estimated 300 million people globally and up to 2.4 million people in the US are estimated to have chronic hepatitis B infection. An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of hepatitis B surface antigen (HBsAg) in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration.
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. 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), 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 PBGENE-HBV and our gene editing approaches, and the suitability of ARCUS nucleases for sophisticated gene edits and diseases with high unmet need; the expected timing of regulatory processes and clinical operations, including filings, studies, enrollment and clinical data for PBGENE-HBV; the encouraging early safety signals observed in the ELIMINATE-B clinical trial that support ongoing dose escalation and multiple dose administrations of PBGENE-HBV; plans to provide ongoing updates on the full low-dose cohort for the PBGENE-HBV study, including multiple dose administrations, and data from higher dose levels throughout 2025; 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,' 'encouraged', '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.
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