Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design

Abstract In preclinical studies, GST-HG141, a novel hepatitis B virus (HBV) capsid assembly modulator displayed potent anti-HBV activity in vitro and strong efficacy in HBV animal models. A randomized, double-blind, ascending phase 1b trial assessed the pharmacokinetics, safety, and efficacy of GST-...

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Main Authors: Min Wu, Jiajia Mai, Hong Zhang, George Zhang, John Mao, Yanan Tang, Wenhao Yan, Wenqiang Wu, Jinlin Hou, Xieer Liang, Zhihong Liu, Yanhua Ding, Junqi Niu
Format: Article
Language:English
Published: BMC 2024-12-01
Series:Virology Journal
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Online Access:https://doi.org/10.1186/s12985-024-02584-8
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author Min Wu
Jiajia Mai
Hong Zhang
George Zhang
John Mao
Yanan Tang
Wenhao Yan
Wenqiang Wu
Jinlin Hou
Xieer Liang
Zhihong Liu
Yanhua Ding
Junqi Niu
author_facet Min Wu
Jiajia Mai
Hong Zhang
George Zhang
John Mao
Yanan Tang
Wenhao Yan
Wenqiang Wu
Jinlin Hou
Xieer Liang
Zhihong Liu
Yanhua Ding
Junqi Niu
author_sort Min Wu
collection DOAJ
description Abstract In preclinical studies, GST-HG141, a novel hepatitis B virus (HBV) capsid assembly modulator displayed potent anti-HBV activity in vitro and strong efficacy in HBV animal models. A randomized, double-blind, ascending phase 1b trial assessed the pharmacokinetics, safety, and efficacy of GST-HG141 in chronic hepatitis B (CHB) individuals. Thirty treatment-naïve CHB patients were enrolled in three cohorts (25, 50, and 100 mg twice orally after meals daily) over 28 days, with 10 subjects per cohort (8:2 ratio for GST-HG141 and placebo). Dose-related safety and tolerability, pharmacokinetic profiles, and drug responses were evaluated. GST-HG141 exhibited a generally favorable safety profile across all doses with predominantly mild adverse reactions, including three cases of grade 1 transaminase elevations. Significant reductions in HBV DNA and pregenomic RNA (pgRNA) levels were observed across all doses of (25, 50, and 100 mg of GST-HG141, twice-daily) after 28 days of treatment. Pharmacokinetic analysis showed a consistent linear trend in GST-HG141 concentrations, with mean trough concentrations ranging from 75 to 240 ng/mL. These concentrations adequately covered the protein binding-adjusted EC50 (16.89 ng/mL) by factors of 4.4, 11.1, and 14.6 for doses of 25, 50, and 100 mg, respectively. Our study demonstrated GST-HG141’s well-tolerated profile up to 100 mg over 4 weeks, alongside robust antiviral activity in CHB patients, supporting its progression into further clinical investigation for CHB management.
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spelling doaj-art-5bc7956a24714399bc46609ddbf4cb5e2025-08-20T01:59:40ZengBMCVirology Journal1743-422X2024-12-012111910.1186/s12985-024-02584-8Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled designMin Wu0Jiajia Mai1Hong Zhang2George Zhang3John Mao4Yanan Tang5Wenhao Yan6Wenqiang Wu7Jinlin Hou8Xieer Liang9Zhihong Liu10Yanhua Ding11Junqi Niu12Phase I Clinical Trial Unit, First Hospital, Jilin UniversityPhase I Clinical Trial Unit, First Hospital, Jilin UniversityPhase I Clinical Trial Unit, First Hospital, Jilin UniversityFujian Akeylink Biotechnology Co., Ltd.Fujian Akeylink Biotechnology Co., Ltd.Fujian Akeylink Biotechnology Co., Ltd.Fujian Akeylink Biotechnology Co., Ltd.Fujian Akeylink Biotechnology Co., Ltd.Department of Infectious Diseases and Hepatology Unit, Department of Infectious Diseases and Hepatology Unit, Nanfang Hospital, Southern Medical UniversityDepartment of Infectious Diseases and Hepatology Unit, Department of Infectious Diseases and Hepatology Unit, Nanfang Hospital, Southern Medical UniversityDepartment of Infectious Diseases and Hepatology Unit, Department of Infectious Diseases and Hepatology Unit, Nanfang Hospital, Southern Medical UniversityPhase I Clinical Trial Unit, First Hospital, Jilin UniversityDepartment of Hepatology, Center of Infectious Disease and Pathogen Biology, The First Hospital of Jilin UniversityAbstract In preclinical studies, GST-HG141, a novel hepatitis B virus (HBV) capsid assembly modulator displayed potent anti-HBV activity in vitro and strong efficacy in HBV animal models. A randomized, double-blind, ascending phase 1b trial assessed the pharmacokinetics, safety, and efficacy of GST-HG141 in chronic hepatitis B (CHB) individuals. Thirty treatment-naïve CHB patients were enrolled in three cohorts (25, 50, and 100 mg twice orally after meals daily) over 28 days, with 10 subjects per cohort (8:2 ratio for GST-HG141 and placebo). Dose-related safety and tolerability, pharmacokinetic profiles, and drug responses were evaluated. GST-HG141 exhibited a generally favorable safety profile across all doses with predominantly mild adverse reactions, including three cases of grade 1 transaminase elevations. Significant reductions in HBV DNA and pregenomic RNA (pgRNA) levels were observed across all doses of (25, 50, and 100 mg of GST-HG141, twice-daily) after 28 days of treatment. Pharmacokinetic analysis showed a consistent linear trend in GST-HG141 concentrations, with mean trough concentrations ranging from 75 to 240 ng/mL. These concentrations adequately covered the protein binding-adjusted EC50 (16.89 ng/mL) by factors of 4.4, 11.1, and 14.6 for doses of 25, 50, and 100 mg, respectively. Our study demonstrated GST-HG141’s well-tolerated profile up to 100 mg over 4 weeks, alongside robust antiviral activity in CHB patients, supporting its progression into further clinical investigation for CHB management.https://doi.org/10.1186/s12985-024-02584-8Antiviral therapyHepatitis BPharmacokineticsTolerabilityCapsid assembly modulator
spellingShingle Min Wu
Jiajia Mai
Hong Zhang
George Zhang
John Mao
Yanan Tang
Wenhao Yan
Wenqiang Wu
Jinlin Hou
Xieer Liang
Zhihong Liu
Yanhua Ding
Junqi Niu
Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
Virology Journal
Antiviral therapy
Hepatitis B
Pharmacokinetics
Tolerability
Capsid assembly modulator
title Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
title_full Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
title_fullStr Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
title_full_unstemmed Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
title_short Safety, pharmacokinetics, and antiviral efficacy of the novel capsid assembly modulator GST-HG141 in patients with chronic hepatitis B: a phase 1 trial with a randomized, placebo-controlled design
title_sort safety pharmacokinetics and antiviral efficacy of the novel capsid assembly modulator gst hg141 in patients with chronic hepatitis b a phase 1 trial with a randomized placebo controlled design
topic Antiviral therapy
Hepatitis B
Pharmacokinetics
Tolerability
Capsid assembly modulator
url https://doi.org/10.1186/s12985-024-02584-8
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