US enrollment starts for hepatitis B gene therapy clinical trial
Phase 1 study aims to assess PBGEN-HBV's safety
A Phase 1 clinical trial testing PBGENE-HBV, a gene-editing therapy for chronic hepatitis B, is now recruiting patients at Massachusetts General Hospital in Boston.
Opening of the first U.S. site of the Phase 1 ELIMINATE-B trial (NCT06680232) comes a few months after the U.S. Food and Drug Administration (FDA) gave Precision Biosciences, PBGENE-HBV’s developer, clearance to expand the study to the U.S.
The trial, which is open to adults with chronic hepatitis B who are already on standard-of-care treatments, is also recruiting participants at sites in Hong Kong, New Zealand, and Moldova.
“As we continue to advance our ELIMINATE-B trial through [group] 3, we are thrilled to open recruitment at Massachusetts General Hospital, a world-renowned institution with deep expertise in running clinical studies in hepatitis,” Cindy Atwell, chief development and business officer at Precision, said in a company press release. “Initiation of our first U.S. trial site represents an important step in expanding our study to patients in the United States.”
The company expects to share ELIMINATE-B data later this year.
Gene-editing therapy aims to inactivate HBV DNA
The hepatitis B virus (HBV), which causes hepatitis B, is spread by contact with bodily fluids. It infects the liver, triggering liver inflammation. In some people, particularly those infected in childhood, the virus causes a long-lasting or chronic infection that can set the stage for life-threatening complications such as liver cancer.
Antiviral therapies can control hepatitis B, but cannot cure the infection. They can neutralize HBV viral particles in the body’s fluids, but they can’t reach the viral DNA that lurks inside of infected liver cells, serving as a reservoir to make new viruses.
PBGENE-HBV is delivered via infusions into the bloodstream and is designed to delete or inactivate all forms of HBV DNA inside of liver cells. Theoretically, this approach, combined with available antiviral treatments, could cure the infection.
The FDA recently granted PGBENE-HBV fast track status, which is meant to speed the development of investigational therapies that have the potential to fill unmet needs for serious conditions.
The Phase 1 ELIMINATE-B study, which launched about a year ago, is testing PBGENE-HBV in up to 45 adults with chronic hepatitis B who are on stable antiviral treatment and who test negative for HBeAg, a protein indicating that HBV is actively replicating in the body.
In its first part, participants will receive multiple increasing doses of the therapy. The aim is to determine the optimal dose to be tested in its second part, which will include a larger number of patients.
The trial’s main goal is to assess PBGENE-HBV’s safety. Secondary goals include evaluating its pharmacological properties and antiviral activity. Preliminary data from the first three participants indicated that a single infusion of the therapy’s lowest tested dose was well tolerated and showed signs of antiviral activity.
The study is expected to finish by the end of next year.
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