|
Basic Characteristics of Mutations
|
|
Mutation Site
|
N236T |
|
Mutation Site Sentence
|
RESULTS: E-CFCP potently blocked HBVWTD1 production (IC50qPCR_cell=1.8 nM) in HepG2.2.15 cells and HBVWTC2 (IC50SB_cell=0.7 nM), entecavir (ETV)-resistant HBVETV-RL180M/S202G/M204V (IC50SB_cell=77.5 nM), and adefovir-resistant HBVADV-RA181T/N236T production (IC50SB_cell=14.1 nM) in Huh7 cells. |
|
Mutation Level
|
Amino acid level |
|
Mutation Type
|
Nonsynonymous substitution |
|
Gene/Protein/Region
|
RT |
|
Standardized Encoding Gene
|
P
|
|
Genotype/Subtype
|
C2;D1 |
|
Viral Reference
|
-
|
|
Functional Impact and Mechanisms
|
|
Disease
|
Cell line
|
|
Immune
|
- |
|
Target Gene
|
-
|
|
Clinical and Epidemiological Correlations
|
|
Clinical Information
|
- |
|
Treatment
|
Abacavir(ADV) |
|
Location
|
- |
|
Literature Information
|
|
PMID
|
33333207
|
|
Title
|
Identification of a novel long-acting 4'-modified nucleoside reverse transcriptase inhibitor against HBV
|
|
Author
|
Higashi-Kuwata N,Hayashi S,Kumamoto H,Ogata-Aoki H,Das D,Venzon D,Hattori SI,Bulut H,Hashimoto M,Otagiri M,Takamune N,Kishimoto N,Davis DA,Misumi S,Kakuni M,Tanaka Y,Mitsuya H
|
|
Journal
|
Journal of hepatology
|
|
Journal Info
|
2021 May;74(5):1075-1086
|
|
Abstract
|
BACKGROUND & AIMS: While certain nucleos(t)ide reverse transcriptase inhibitors (NRTIs) are efficacious in treating HBV infection, their effects are yet to be optimized and the emergence of NRTI-resistant HBV variants is an issue because of the requirement for lifelong treatment. The development of agents that more profoundly suppress wild-type and drug-resistant HBVs, and that have a long-acting effect, are crucial to improve patient outcomes. METHODS: Herein, we synthesized a novel long-acting 4'-modified NRTI termed E-CFCP. We tested its anti-HBV activity in vitro, before evaluating its anti-HBV activity in HBV-infected human-liver-chimeric mice (PXB-mice). E-CFCP's long-acting features and E-CFCP-triphosphate's interactions with the HBV reverse transcriptase (HBV-RT) were examined. RESULTS: E-CFCP potently blocked HBV(WT)(D1) production (IC(50)(qPCR_cell)(=)1.8 nM) in HepG2.2.15 cells and HBV(WT)(C2) (IC(50)(SB_cell)=0.7 nM), entecavir (ETV)-resistant HBV(ETV-R)(L180M/S202G/M204V) (IC(50)(SB_cell)=77.5 nM), and adefovir-resistant HBV(ADV-R)(A181T/N236T) production (IC(50)(SB_cell)=14.1 nM) in Huh7 cells. E-CFCP profoundly inhibited intracellular HBV DNA production to below the detection limit, but ETV and tenofovir alafenamide (TAF) failed to do so. E-CFCP also showed less toxicity than ETV and TAF. E-CFCP better penetrated hepatocytes and was better tri-phosphorylated; E-CFCP-triphosphate persisted intracellularly for longer than ETV-triphosphate. Once-daily peroral E-CFCP administration over 2 weeks (0.02~0.2 mg/kg/day) reduced HBV(WT)(C2)-viremia by 2-3 logs in PXB-mice without significant toxicities and the reduction persisted over 1-3 weeks following treatment cessation, suggesting once-weekly dosing capabilities. E-CFCP also reduced HBV(ETV-R)(L180M/S202G/M204V)-viremia by 2 logs over 2 weeks, while ETV completely failed to reduce HBV(ETV-R)(L180M/S202G/M204V)-viremia. E-CFCP's 4'-cyano and fluorine interact with both HBV(WT)-RT and HBV(ETV-R)(L180M/S202G-M204) -RT via Van der Waals and polar forces, being important for E-CFCP-triphosphate's interactions and anti-HBV potency. CONCLUSION: E-CFCP represents the first reported potential long-acting NRTI with potent activity against wild-type and treatment-resistant HBV. LAY SUMMARY: Although there are currently effective treatment options for HBV, treatment-resistant variants and the need for lifelong therapy pose a significant challenge. Therefore, the development of new treatment options is crucial to improve outcomes and quality of life. Herein, we report preclinical evidence showing that the anti-HBV agent, E-CFCP, has potent activity against wild-type and treatment-resistant variants. In addition, once-weekly oral dosing may be possible, which is preferrable to the current daily dosing regimens.
|
|
Sequence Data
|
-
|
|
|