Precision BioSciences' Gene Editing Therapy Shows Promise in Early Hepatitis B Trial

Precision BioSciences, Inc. (DTIL) has announced encouraging initial safety and antiviral activity data from its Phase 1 dose-finding study, ELIMINATE-B, for chronic Hepatitis B. The trial is evaluating PBGENE-HBV, a novel in vivo gene editing therapy designed to directly eliminate and inactivate the root cause of Hepatitis B virus from covalently closed circular DNA (cccDNA) and integrated DNA.

PBGENE-HBV demonstrated a strong safety profile in the first cohort of patients, with no Grade ≥2 treatment-related adverse events or serious adverse events reported. This is a significant achievement, as existing hepatitis B treatments often come with side effects and potential long-term complications. The well-tolerated safety profile of PBGENE-HBV, combined with its early antiviral activity, suggests that it could potentially offer a more favorable risk-benefit profile compared to existing treatments.

In addition to safety, PBGENE-HBV demonstrated substantial reduction in Hepatitis B surface antigen (HBsAg) in two of the three participants following the first administration at the lowest dose level (0.2 mg/kg). This early antiviral activity supports the potential of the drug to eliminate the root cause of Hepatitis B virus from cccDNA and integrated DNA, which is crucial for achieving undetectable levels of HBsAg.

The planned dosing schedule in the ELIMINATE-B trial balances the need for safety and efficacy by allowing for two additional administrations at the lowest dose level while simultaneously investigating the next higher dose level. This approach enables Precision BioSciences to gather more data on the safety and antiviral activity of PBGENE-HBV at the initial dose level before escalating to higher doses.



As the ELIMINATE-B study progresses, additional administrations at higher dose levels will help define the optimal dose and number of dose administrations for safely eliminating cccDNA and inactivating integrated HBV DNA. This will further validate the drug's efficacy and potential for treating chronic Hepatitis B in a broader patient population.

The substantial reduction in HBsAg observed in two of the three participants can be attributed to several factors, including PBGENE-HBV's unique mechanism of action, the dose level and administration, patient characteristics, and consistency with preclinical data. These factors suggest that PBGENE-HBV may be effective across a range of chronic Hepatitis B patient profiles.

In conclusion, the encouraging initial safety and antiviral activity data from the ELIMINATE-B trial support the potential of PBGENE-HBV as a novel and effective treatment option for chronic Hepatitis B. As the study continues, investors will eagerly await further data to validate the drug's efficacy and potential market adoption.