Precision's gene editing therapy shows promising results in HBV trial

DURHAM, N.C. - Precision BioSciences (NASDAQ:DTIL) presented data from its Phase 1 ELIMINATE-B study evaluating PBGENE-HBV, a gene editing therapy targeting chronic Hepatitis B, at the American Association for the Study of Liver Diseases meeting Monday. The clinical-stage biotech company, currently valued at approximately $80 million, has seen its stock price drop 16.6% over the past week, though it remains up 61% year-to-date according to InvestingPro data. Analysts have set price targets significantly above current levels, suggesting potential upside despite the company's weak financial health rating.

The therapy, designed to eliminate cccDNA and inactivate integrated HBV DNA, showed dose-dependent antiviral activity across all nine patients treated in the first three study cohorts, according to data presented.

In the highest dose cohort (0.8 mg/kg), all three patients demonstrated steep declines in hepatitis B surface antigen (HBsAg) at day 14, with evidence of cumulative declines after repeat administration in one patient.

The company reported that PBGENE-HBV was well-tolerated across all tested doses with no dose-limiting toxicities observed. Adverse events were generally transient and resolved within 12 hours.

Notably, paired liver biopsy data from one patient in the second cohort (0.4 mg/kg) provided evidence of viral DNA editing after two administrations, which the company states directly correlates with observed HBsAg reductions.

"For the first time, we have clinical biopsy evidence that a gene editing therapy aimed at elimination of cccDNA can directly modify HBV DNA in infected human liver tissue," said Dr. Man-Fung Yuen, lead investigator, in the press release.

The company plans to complete all administrations in the third cohort by the first quarter of 2026 and is considering testing for cure by stopping nucleos(t)ide analogues when HBsAg approaches undetectable levels on a sustained basis. While Precision BioSciences holds more cash than debt on its balance sheet and maintains a current ratio of 3.45, InvestingPro analysis indicates the company is quickly burning through cash. With analysts forecasting a sales decline and continued unprofitability this year, investors should note the company reported a negative $83.6 million in net income over the last twelve months.

Chronic Hepatitis B affects an estimated 300 million people globally and 1-2 million in the United States, with current treatments requiring lifelong administration and rarely leading to functional cure.

The information in this article is based on a press release from Precision BioSciences.

In other recent news, Precision BioSciences announced the publication of research showcasing the capabilities of its ARCUS gene editing platform in the journal Nucleic Acids Research. The study highlights the platform's ability to achieve high-efficiency gene insertions and various DNA edits, with transgene insertion rates exceeding 85% in T lymphocytes and up to 40% in non-dividing primary human hepatocytes. Additionally, Precision BioSciences presented clinical data from its Phase 1 ELIMINATE-B trial of PBGENE-HBV in patients with chronic Hepatitis B, revealing that the therapy has been well-tolerated and shown activity in patients. The company also secured a U.S. patent for its PBGENE-HBV gene editing therapy, which extends protection until March 2042. This patent covers the ARCUS nuclease used in their lead in vivo gene editing program. Furthermore, Precision BioSciences updated executive contracts with new severance terms for its key officers, including President and CEO Michael Amoroso and other senior executives. The new agreements reflect current annual base salaries and target bonuses, with cash severance paid as a lump sum. These developments indicate ongoing advancements in the company's gene editing initiatives and corporate governance.

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