IP Library Granted Patent US 12,552,736
Granted Patent B2
US 12,552,736 · App. 17/838,817 · Granted Feb 17, 2026

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

Inventor: Robert Boyd (Horsham, PA)
Assignee: Amicus Therapeutics, Inc.
C07C215/44A61P25/28C07C233/06A61K45/06C07B2200/07
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Quick Facts
Patent No.
US 12,552,736
App. No.
17/838,817
Granted
Feb 17, 2026
Kind
B2
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.

Claims (39)

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.

Assignments (3)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →