Glyceryl Tribenzoate Inhalation Forms for Sustained Huntington’s Therapy

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Solution Overview

Problem

Sodium benzoate, despite its therapeutic benefits for neurodegenerative disorders, is quickly metabolized and excreted from the body, necessitating frequent administration, which is inconvenient for patients.

Innovation Solution

Development of pharmaceutical compositions comprising glyceryl tribenzoate and/or glyceryl dibenzoate in inhalation dosage forms, which are administered less frequently, allowing for sustained therapeutic effects by slow conversion to sodium benzoate in the body.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If sodium benzoate is administered frequently to maintain therapeutic levels, then therapeutic effectiveness is improved, but patient convenience and compliance deteriorate

Engineering Contradiction:
Improvetherapeutic effectivenessVSAvoidpatient convenience
Core Design Contradiction:
ReliabilityVSEase of operation

Solution Approach 1:

The prodrug glyceryl tribenzoate is administered in advance and converted to sodium benzoate in the body, creating a reservoir that slowly releases the active compound. This preliminary action of converting the stable prodrug form to the active form ensures sustained therapeutic levels without frequent dosing.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

Glyceryl tribenzoate serves as an intermediary substance that bridges the gap between infrequent administration and sustained therapeutic effect. It is converted by lipases to sodium benzoate, acting as a mediator that extends the duration of action while maintaining therapeutic effectiveness.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Reliability

If high doses of sodium benzoate are administered to overcome rapid excretion, then therapeutic effectiveness is improved, but loss of substance increases

Engineering Contradiction:
Improvetherapeutic effectivenessVSAvoiddrug excretion
Core Design Contradiction:
ReliabilityVSLoss of substance

Solution Approach 1:

The conversion of glyceryl tribenzoate to sodium benzoate occurs continuously in the body through lipase action, maintaining a steady supply of the active compound. This continuous conversion ensures sustained therapeutic effect while reducing the need for high repeated doses and minimizing total substance loss through excretion.

Inventive Principle:
Principle #20Continuity of useful action

3Reliability

If frequent administration of sodium benzoate is required, then therapeutic effectiveness is maintained, but loss of time increases

Engineering Contradiction:
Improvetherapeutic effectivenessVSAvoidadministration frequency
Core Design Contradiction:
ReliabilityVSLoss of time

Solution Approach 1:

Instead of administering small frequent doses, the invention uses a single or reduced frequency administration of the prodrug that provides excessive or sufficient action through continuous conversion. This partial reduction in administration frequency maintains therapeutic effectiveness while reducing the time loss associated with frequent dosing.

Inventive Principle:
Principle #16Partial or excessive action

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The compositions provide prolonged therapeutic benefits with reduced administration frequency, effectively inhibiting neurodegenerative disorder progression and improving locomotor activities, while reducing cell activation and treating peripheral tissues.

Implementation Method 1

Glycerol tribenzoate is cleaved by different lipases in the body to produce three benzoate molecules. Typically, pancreatic lipase hydrolyzes the benzoic acid esterified to the first and third carbon atoms of glycerol

Methodology Applied
Scientific EffectHydrolysis: Hydrolysis

Implementation Method 2

After intake, these three active compounds are converted into cinnamic acid by oxidation and hydrolysis, respectively. Then, cinnamic acid is β-oxidized to benzoate in the liver

Methodology Applied
Scientific EffectOxidation: Oxidation

Data Source

PatentEP3999051B1Inhalation dosage forms of glycerol tribenzoate and/or glycerol dibenzoate for use in the treatment of huntington's disease
Publication Date: 2025.07.09 RUSH UNIV MEDICAL CENT
  • EP3999051B1 patent drawingFigure 1A~1E
  • EP3999051B1 patent drawingFigure 2A~2F
  • EP3999051B1 patent drawingFigure 3A~3G

AI summary

The present disclosure generally relates to improved pharmaceutical compositions useful for the treatment of diseases and disorders. More particularly, the disclosure relates to pharmaceutical compositions comprising glyceryl tribenzoate and/or glyceryl dibenzoate for the treatment of neurodegenerative disorders and peripheral diseases.