Fused-Ring Compound Solid Forms for Resistant RET Inhibition

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Solution Overview

Problem

Cancer cells develop resistance to RET inhibitors, limiting treatment options for patients with RET fusion proteins, particularly due to mutations like G810R, G810S, and G801C, which reduce drug binding to the ATP binding site.

Innovation Solution

Development of solid forms of a compound with strong RET mutation inhibitory activity, including anhydrous and solvated crystal forms, characterized by specific X-ray powder diffraction patterns and thermal analysis profiles, to enhance the effectiveness of RET inhibitors.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If RET inhibitor selpercatinib (LOXO-292) is used to treat tumor patients with RET fusion proteins, then initial treatment effectiveness is achieved, but tumor cells develop resistance through mutations like G810R, G810S, and G801C, reducing drug binding to the ATP binding site

Engineering Contradiction:
Improvetreatment effectivenessVSAvoidtreatment duration
Core Design Contradiction:
ReliabilityVSDuration of action of stationary object

Solution Approach 1:

The patent develops new compounds with modified chemical structures (formula I) that change the binding parameters to accommodate mutated RET proteins. The solid forms (crystal forms A-P) provide stable pharmaceutical formulations that maintain consistent drug delivery, ensuring reliable inhibition of both wild-type and mutated RET proteins including G810R, G810S, and G801C mutations.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates composite solid forms combining the RET inhibitor compound with specific solvents (water, alcohols, nitriles, haloalkanes, ethers) to form stable solvates and co-solvates. These composite structures enhance drug stability and bioavailability, allowing the medication to maintain effectiveness against mutated RET proteins over extended treatment periods.

Inventive Principle:
Principle #40Composite materials

2Reliability

If compounds with strong RET mutation inhibitory activity are developed, then treatment effectiveness against resistant cancer cells is improved, but the complexity of developing and characterizing multiple solid forms increases

Engineering Contradiction:
Improveinhibitory activityVSAvoidsolid form characterization
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent segments the solid form development into distinct crystal forms (A through P), each with specific X-ray powder diffraction patterns and characteristic properties. This segmentation allows systematic characterization and selection of optimal forms for different pharmaceutical applications, managing complexity through organized classification.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The patent uses X-ray powder diffraction patterns as characteristic 'signatures' for each crystal form, with specific 2θ values serving as identifiers. This provides a reliable method for distinguishing and characterizing different solid forms, simplifying quality control and formulation selection despite the multiplicity of forms.

Inventive Principle:
Principle #32Color 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

The solid forms of the compound effectively inhibit RET mutations, providing a more stable and potent treatment option for cancer cells resistant to existing RET inhibitors.

Implementation Method 1

decrease in the binding of LOXO-292 to the ATP binding site, thereby causing drug resistance

Methodology Applied
Scientific EffectATP binding site inhibition:

Implementation Method 2

an X-ray powder diffraction pattern of the crystal form A has one or two characteristic peaks at 2θ values selected from the following: 6.91°±0.20° and 10.32°±0.20°

Methodology Applied
Scientific EffectX-ray diffraction: X-Ray

Implementation Method 3

a differential scanning calorimetry (DSC) profile of the crystal form A has an endothermic peak at an onset temperature of about 175.03 °C and/or a peak temperature of about 180.61 °C

Methodology Applied
Scientific EffectDifferential scanning calorimetry: Calorimetry

Implementation Method 4

thermogravimetric analysis (TGA) of the crystal form A shows a weight loss of about 0.45% to about 0.55% (e.g., about 0.499%) upon heating from room temperature to 190 °C

Methodology Applied
Scientific EffectThermogravimetric analysis:

Data Source

PatentEP4624475A1Solid form of fused ring compound, and preparation method therefor and use thereof
Publication Date: 2025.10.01 APPLIED PHARMA SCI
  • EP4624475A1 patent drawingFigure 1A~1B
  • EP4624475A1 patent drawingFigure 1C~1D
  • EP4624475A1 patent drawingFigure 2A~2B

AI summary

The present disclosure relates to a solid form of a fused ring compound, and a preparation method therefor and a use thereof. A more advantageous pharmaceutically acceptable form is provided for a compound of formula (I). The solid form provided by the present disclosure has advantageous physicochemical and pharmacokinetic properties.