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-11358926-B2
Application number	US-201816608592-A
Country	US
Kind code	B2
Filing date	Apr 25, 2018
Priority date	Apr 25, 2017
Publication date	Jun 14, 2022
Grant date	Jun 14, 2022

Abstract

The present invention provides novel compounds as well as compositions and methods using the same for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Parkinson's disease, Alzheimer's disease and/or Gaucher's disease.

First claim

What is claimed:

1. 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 —OH;
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 8 or R 10 comprises a halogen.

2. A compound selected from the group consisting of the following:

or a pharmaceutically acceptable salt, thereof.

3. A kit comprising:
a container having an effective amount of a compound of claim 2 , or a pharmaceutically acceptable salt, or solvate thereof, or any combination of two or more thereof; and
instructions for using the same to treat a degenerative disorder of the central nervous system or a lysosomal storage disorder.

4. A pharmaceutical composition comprising the compound of claim 1 and at least one pharmaceutically acceptable carrier.

5. A pharmaceutical composition comprising the compound of claim 2 and at least one pharmaceutically acceptable carrier.

Assignees

• Amicus Therapeutics Inc

Inventors

• Boyd Robert

Classifications

• C07C215/44 Primary

  bound to carbon atoms of the same ring or condensed ring system · 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

• A61K31/133

  having hydroxy groups, e.g. sphingosine · 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

• 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