IP Library Patent Application 16049203
Patent Application
App. No. 16/049,203

Novel Compositions for Preventing and/or Treating Lysosomal Storage Disorders

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Quick Facts
Patent No.
US None
App. No.
16/049,203
Abstract

The present invention provides novel compositions as well as methods for preventing and/or treating lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.

Claims (51)

1 - 20 . (canceled)

30 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of a compound of Formula II:

wherein:

R 1 is C(R 2 )(R 3 )(R 4 );

R 2 is hydrogen, —OH or halogen;

R 3 is hydrogen, —OH, halogen or —CH 3 ;

R 4 is halogen, —CH 3 , phenyl, fluorophenyl, methylphenyl, 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 hydrogen, phenylalkyl or substituted phenylalkyl;

Z is optional, when present Z is —(CH 2 )—, —C(═O)—, —S(═O) 2 NH—, —S(═O) 2 —, —S(═O) 2 —CH 2 —, —C(═O)—NH—, —S(═O) 2 NR 9 —, —C(═S)—NH— or —C(═O)—CH 2 —;

R 9 is hydrogen or CH 3 ;

R 5 is hydrogen or aminophenylalkyl;

R 7 is —OH or halogen; and

R 8 is hydrogen, halogen or —CH 3 ,

provided that R 2 and R 3 cannot both be hydrogen when R 4 is halogen, Z is not present, R 7 is —OH, R 5 , R 6 and R 8 are hydrogen.

31 . The method of claim 30 , further comprising administering an effective amount of at least one other therapeutic agent.

32 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, 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 hydrogen, —OH or halogen;

R 3 is hydrogen, —OH, halogen or —CH 3 ;

R 4 is halogen, —CH 3 , phenyl, fluorophenyl, methylphenyl, 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 7 is —OH or halogen; and

R 8 is hydrogen, halogen or —CH 3 ,

provided that R 2 and R 3 cannot both be hydrogen when R 4 is a halogen, R 7 is —OH and R 8 is hydrogen.

33 . The method of claim 32 , further comprising administering an effective amount of at least one other therapeutic agent.

34 . The method of claim 32 , wherein,

R 2 is hydrogen or fluorine;

R 3 is fluorine;

R 4 is fluorine, C 1-4 alkyl, or phenyl;

R 7 is —OH; and

R 8 is hydrogen.

35 . The method of claim 34 , wherein R 2 is hydrogen.

36 . The method of claim 34 , wherein R 2 is fluorine.

37 . The method of claim 34 , wherein R 4 is fluorine.

38 . The method of claim 34 , wherein R 4 is C 1-4 alkyl.

39 . The method of claim 34 , wherein R 4 is phenyl.

40 . A method for treating a lysosomal storage disorder in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of one or more compound selected from the group consisting of:

or pharmaceutically acceptable salts thereof.

41 . The method of claim 40 , further comprising administering an effective amount of at least one other therapeutic agent.

42 . The method of claim 40 , wherein the compound is:

or a pharmaceutically acceptable salt thereof.

43 . The method of claim 40 , wherein the compound is:

or a pharmaceutically acceptable salt thereof.

44 . The method of claim 40 , wherein the compound is:

or a pharmaceutically acceptable salt thereof.

45 . The method of claim 40 , wherein the compound is:

or a pharmaceutically acceptable salt thereof.

46 . The method of claim 40 , wherein the compound is:

or a pharmaceutically acceptable salt thereof.

Assignments (2)
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 →