FXR Agonists for Hepatitis B Virus Treatment

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Solution Overview

Problem

Current treatments for chronic hepatitis B (CHB) are suboptimal, with limitations including major side effects, low rates of HBV DNA suppression, development of resistance, and inability to effectively target the HBV covalently closed circular DNA (cccDNA) reservoir, leading to prolonged and life-long therapy with adverse events.

Innovation Solution

The use of farnesoid X receptor (FXR) agonists, which selectively bind and activate the FXR, inhibiting HBV replication by targeting cellular proteins involved in viral entry and innate immunity, offering a safer and broad-spectrum therapeutic approach.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If direct antiviral agents (nucleos/tide analogues) are used to suppress HBV replication, then HBV DNA replication is inhibited, but resistance develops and HBsAg clearance remains very low

Engineering Contradiction:
ImproveHBV DNA suppressionVSAvoidresistance development
Core Design Contradiction:
ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent introduces interferon as an intermediary agent that works through a different mechanism (immune modulation) rather than directly inhibiting reverse transcription. This intermediary approach allows combination therapy where interferon enhances HBsAg clearance and prevents resistance while nucleos/tide analogues maintain HBV DNA suppression.

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent changes the therapeutic parameter from solely direct antiviral inhibition to a dual approach combining direct inhibition with immune modulation. This parameter change enables simultaneous achievement of HBV DNA suppression and HBsAg clearance while preventing resistance development.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If interferon treatment is used to treat CHB, then HBV replication is suppressed, but major side effects occur and ALT normalization rate is low

Engineering Contradiction:
ImproveHBV DNA suppressionVSAvoidside effects
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent uses partial action by combining a lower dose or shorter duration of interferon with nucleos/tide analogues, achieving the necessary immune modulation effect while reducing the harmful side effects. The nucleos/tide analogues provide baseline HBV suppression, allowing interferon to be used more selectively.

Inventive Principle:
Principle #16Partial or excessive action

Solution Approach 2:

Nucleos/tide analogues serve as intermediaries that provide stable HBV suppression, allowing interferon to be used at reduced doses or for shorter periods, thereby maintaining therapeutic efficacy while minimizing side effects.

Inventive Principle:
Principle #24Intermediary (Mediator)

3Reliability

If prolonged life-long therapy with direct antivirals is administered, then HBV replication remains suppressed, but adverse events increase and HBsAg clearance is very low

Engineering Contradiction:
ImproveHBV DNA suppressionVSAvoidadverse events
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent implements periodic action through interferon treatment cycles (e.g., 48-96 weeks) followed by maintenance with nucleos/tide analogues or observation. This periodic intensive therapy achieves HBsAg clearance and sustained remission, allowing discontinuation or reduction of long-term antiviral therapy.

Inventive Principle:
Principle #19Periodic action

Solution Approach 2:

The patent ensures continuous useful action through combination therapy where nucleos/tide analogues provide continuous HBV suppression while interferon provides periodic immune enhancement. This continuous dual mechanism maintains suppression while enabling eventual discontinuation compared to indefinite monotherapy.

Inventive Principle:
Principle #20Continuity of useful action

4Reliability

If current direct antivirals are used to treat HBV, then reverse transcription is repressed, but cccDNA reservoir remains unaffected and viral gene expression continues

Engineering Contradiction:
ImproveHBV DNA replication inhibitionVSAvoidcccDNA reservoir persistence
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

Interferon acts as an intermediary that targets host cellular mechanisms and immune responses rather than directly inhibiting viral reverse transcription. This enables interferon to affect cccDNA transcription and immune recognition of infected cells, potentially reducing the functional cccDNA reservoir while nucleos/tide analogues maintain replication suppression.

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

Instead of directly targeting the viral reverse transcription enzyme (downstream), the patent inverts the approach by using interferon to target upstream host factors and immune mechanisms that control cccDNA maintenance and expression. This inverted strategy addresses the root reservoir problem rather than just the replication consequence.

Inventive Principle:
Principle #13The other way round (Inversion)

Data Source

PatentEP3043865B1Methods and pharmaceutical compositions for the treatment of hepatitis b virus infection
Publication Date: 2020.11.04 POXEL
  • EP3043865B1 patent drawingFigure 1
  • EP3043865B1 patent drawingFigure 2A
  • EP3043865B1 patent drawingFigure 2B

AI summary

The present invention relates to methods and pharmaceutical compositions for the treatment of hepatitis B virus infection. In particular, the present invention relates to farnesoid X receptor (FXR) agonists for use in a method for the treatment of hepatitis B virus infection in a subject in need thereof.