Prodrug CPHPC Derivative for Oral SAP Depletion

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Solution Overview

Problem

Current treatments for amyloidosis and related conditions, such as Alzheimer's disease and type 2 diabetes, face challenges in completely removing serum amyloid P component (SAP) from amyloid deposits, leading to incomplete regression of amyloid and ongoing disease progression, and existing compounds like CPHPC have low oral bioavailability and cause discomfort with long-term administration.

Innovation Solution

The compound (2R,2'R)-bis(((tetrahydro-2H-pyran-4-carbonyl)oxy)methyl) 1,1'-adipoylbis(pyrrolidine-2-carboxylate) is developed, which exhibits good physicochemical properties, including oral bioavailability and stability, capable of generating CPHPC, allowing for efficient SAP depletion without the drawbacks of existing treatments.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If existing compounds like CPHPC are used for SAP depletion, then SAP can be depleted from circulation, but oral bioavailability is low and long-term administration causes discomfort

Engineering Contradiction:
ImproveSAP depletion efficacyVSAvoidoral bioavailability and administration tolerance
Core Design Contradiction:
ReliabilityVSEase of operation

Solution Approach 1:

The patent applies preliminary action by developing a prodrug formulation of CPHPC that is prepared in advance with improved physicochemical properties. The prodrug is designed to be orally bioavailable and stable in the gastrointestinal tract, converting to active CPHPC in vivo. This preliminary preparation resolves the contradiction by enabling oral administration (improving ease of operation) while maintaining SAP depletion efficacy (maintaining reliability).

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent applies parameter changes by modifying the chemical structure of CPHPC to create a prodrug with altered physical and chemical parameters. The prodrug formulation changes solubility, stability, and bioavailability parameters, allowing oral administration while maintaining the core SAP-depleting function. This resolves the contradiction between oral bioavailability and treatment efficacy.

Inventive Principle:
Principle #35Parameter changes

2Productivity

If palindromic bivalent ligands are administered for SAP depletion, then rapid and almost complete depletion of SAP from circulation is achieved, but not all SAP bound to amyloid deposits is removed

Engineering Contradiction:
ImproveSAP depletion speed and completenessVSAvoidamyloid deposit regression completeness
Core Design Contradiction:
ProductivityVSReliability

Solution Approach 1:

The patent applies continuity of useful action by designing a treatment regimen where the prodrug formulation enables sustained oral administration. The continuous supply of active CPHPC through oral dosing maintains SAP depletion in circulation over time, which progressively leads to complete amyford deposit regression. This continuous action resolves the contradiction between rapid initial depletion and complete eventual removal.

Inventive Principle:
Principle #20Continuity of useful action

Solution Approach 2:

The prodrug formulation performs preliminary action by ensuring stable delivery and absorption of CPHPC precursors in the gastrointestinal tract. This preliminary optimized delivery system ensures continuous availability of active compound, enabling both rapid initial SAP depletion and sustained action for complete amyford deposit removal over time.

Inventive Principle:
Principle #10Preliminary action

3Reliability

If long-term administration of CPHPC is required for complete amyford regression, then treatment duration increases, but patient comfort and compliance decrease due to administration discomfort

Engineering Contradiction:
Improvecomplete amyford regressionVSAvoidpatient comfort and compliance
Core Design Contradiction:
ReliabilityVSEase of operation

Solution Approach 1:

The patent applies the intermediary principle by introducing a prodrug formulation as a mediator between the patient and active CPHPC. The prodrug serves as an intermediate vehicle that is orally bioavailable and comfortable to administer, which then converts to active CPHPC in the body. This intermediary resolves the contradiction by making long-term treatment comfortable and compliant while maintaining efficacy for complete amyford regression.

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent applies parameter changes by modifying CPHPC into a prodrug with improved physical and chemical parameters for oral administration. The prodrug formulation changes solubility, stability, and bioavailability parameters, enabling comfortable long-term oral dosing while maintaining the therapeutic effect needed for complete amyford regression over time.

Inventive Principle:
Principle #35Parameter changes

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

This compound effectively depletes SAP, facilitating the removal of amyloid deposits and improving treatment outcomes for amyloidosis, Alzheimer's disease, and type 2 diabetes, while offering a more tolerable and efficient administration method compared to existing therapies.

Implementation Method 1

The compound (2R,2'R)-bis(((tetrahydro-2H-pyran-4-carbonyl)oxy)methyl) 1,1'-adipoylbis(pyrrolidine-2-carboxylate) is developed, which exhibits good physicochemical properties, including oral bioavailability and stability, capable of generating CPHPC

Methodology Applied
Scientific EffectHydrolysis: Hydrolysis

Data Source

PatentEP3368532B1D-proline derivative as sap depleting agent
Publication Date: 2020.01.15 GLAXOSMITHKLINE INTPROP DEV LTD
  • EP3368532B1 patent drawingFigure 1
  • EP3368532B1 patent drawingFigure 2
  • EP3368532B1 patent drawingFigure 3

AI summary

The present invention relates to the compound ((2R,2'R)-bis(((tetrahydro-2H-pyran-4- carbonyl)oxy)methyl) 1,1'-adipoylbis(pyrrolidine-2-carboxylate), pharmaceutical compositions comprising the same and the use of the same for treatment of diseases or disorders wherein depletion of serum amyloid P component (SAP) would be beneficial, including amyloidosis, Alzheimer s disease, type 2 diabetes mellitus and osteoarthritis.