Compositions for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders

Patent metadata

Field	Value
Publication number	US-12552736-B2
Application number	US-202217838817-A
Country	US
Kind code	B2
Filing date	Jun 13, 2022
Priority date	Apr 25, 2017
Publication date	Feb 17, 2026
Grant date	Feb 17, 2026

Abstract

The present invention provides methods for treating lysosomal storage disorders such as Gaucher's disease, by using compounds of Formula III as described herein.

First claim

What is claimed is:

1 . A method for treating a Gaucher's disease, which comprises administering to the patient in need thereof an effective amount of a compound of Formula III:

wherein:
R 1 is C(R 2 )(R 3 )(R 4 );
R 2 is selected from the group consisting of hydrogen, —OH or halogen;
R 3 is selected from the group consisting of hydrogen, —OH, halogen or —CH 3 ;
R 4 is selected from the group consisting of hydrogen, halogen, —CH 3 , phenyl, fluorophenyl, methylphenyl or cyclohexylmethyl;
R 3 and R 4 may join with the carbon to which they are attached to form a cycloalkyl ring, which may be optionally substituted with one or more halogen atoms;
R 6 is selected from the group consisting of —OH or halogen;
R 8 is selected from the group consisting of —OH or halogen; and
R 10 is selected from the group consisting of —OH or halogen, provided that that at least one of R 2 , R 3 , R 4 , R 6 , R 8 or R 10 comprises a halogen.

2 . A method for treating Gaucher's disease, which comprises administering to the patient in need thereof an effective amount of a compound selected from the group consisting of the following:

or a pharmaceutically acceptable salt, or solvate, or prodrug thereof.

3 . The method of claim 2 , further comprising administering an effective amount of at least one other therapeutic agent selected from the group consisting of imiglucerase or 1,5-(butylimino)-1,5-dideoxy-D-glucitol.

4 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

5 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

6 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

7 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

8 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

9 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

10 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

11 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

12 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

13 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

14 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

15 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

16 . The method of claim 2 , wherein the compound comprises

or a pharmaceutically acceptable salt or solvate thereof.

Assignees

• Amicus Therapeutics Inc

Inventors

• Boyd Robert

Classifications

• C07C233/06

  having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a ring other than a six-membered aromatic ring · CPC title

• C07B2200/07

  Optical isomers · CPC title

• A61K45/06

  Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca · CPC title

• A61P25/28

  for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia · CPC title

• A61K2300/00

  Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00 · CPC title