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US 12,661,342 B2Granted
Heteroaryl compounds and therapeutic uses thereof in conditions associated with the alteration of the activity of beta-glucocerebrosidase
AssigneeGt Gain Therapeutics SA
InventorsAna Maria García Collazo, Elena Cubero Jordà, Manolo Bellotto, Enrique Fernández Inglesias
Life of this patent
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Abstract
The application is directed to compounds of formulae (IA) and (IB) and their salts and solvates, wherein R1a, R2a, R3a, A1, A2, A3, R1b, R2b, R3b, B1, and B2 are as set forth in the specification, as well as to methods for their preparation, pharmaceutical compositions comprising the same, and use thereof for the treatment and/or prevention of, e.g., lysosomal storage diseases, such as Gaucher's disease, and α-synucleinopathies, such as Parkinson's disease.
Claims31
A compound of formula (IA):
- or a pharmaceutically acceptable salt or solvate thereof, wherein
- A1, A2, and A3 are each independently selected from the group consisting of N, CH and C(R4a), provided that at least one of A1, A2, or A3 is N and no more than two of A1, A2, or A3 are N;
- each R4a is independently selected from the group consisting of halogen, —C1-4 alkyl, —C1-4 alkoxy, and —CN;
- R1a is selected from the group consisting of C5-7 cycloalkyl, phenyl, and —C1-3 alkyl-phenyl, wherein said C5-7 cycloalkyl, phenyl and —C1-3 alkyl-phenyl groups are optionally substituted with 1, 2 or 3 substituents each independently selected from the group consisting of halogen, hydroxy, —CN, —ORba, —SRba, —N(Rba)2, and —C1-4 alkyl optionally substituted with 1, 2, or 3 halogen atoms;
- and wherein said C5-7 cycloalkyl, phenyl, and —C1-3 alkyl-phenyl is optionally fused to a further (second) ring;
- and
- R2a is selected from the group consisting of hydrogen and —C1-4 alkyl;
- or
- R1a and R2a together with the nitrogen atom to which they are attached form an optionally substituted 5- to 10-membered heterocyclic ring, wherein said heterocyclic ring optionally contains 1, 2, or 3 additional heteroatoms selected from the group consisting of N, S, or O, and wherein said heterocyclic ring is optionally fused to a phenyl ring;
- each Rba is independently hydrogen or —C1-4 alkyl, wherein said alkyl group is optionally substituted by 1, 2 or 3 fluorine atoms;
- and
- R3a is selected from the group consisting of cyclohexyl, phenyl, and pyridinyl, wherein said cyclohexyl, phenyl, and pyridinyl groups are optionally substituted with 1, 2 or 3 substituents each independently selected from the group consisting of halogen, hydroxy, —CN, —ORba, —SRba, —N(Rba)2, and C1-4 alkyl optionally substituted with 1, 2, or 3 substituents each independently selected from the group consisting of halogen, —CN, —ORba, and —N(Rba)2, and wherein said phenyl is optionally fused to a 5- or 6-membered heterocyclic.
9 more elements
A method of treating Gaucher's disease, comprising administering to a patient in need thereof an effective amount of a compound of formula (IA):
- or a pharmaceutically acceptable salt or solvate thereof, wherein
- A1, A2, and A3 are each independently selected from the group consisting of N, CH and C(R4a), provided that at least one of A1, A2, or A3 is N and no more than two of A1, A2, or A3 are N;
- each R4a is independently selected from the group consisting of halogen, —C1-4 alkyl, —C1-4 alkoxy, and —CN;
- R1a is selected from the group consisting of C5-7 cycloalkyl, phenyl, and —C1-3 alkyl-phenyl, wherein said C5-7 cycloalkyl, phenyl and —C1-3 alkyl-phenyl groups are optionally substituted with 1, 2 or 3 substituents each independently selected from the group consisting of halogen, hydroxy, —CN, —ORba, —SRba, —N(Rba)2, and —C1-4 alkyl optionally substituted with 1, 2, or 3 halogen atoms;
- and wherein said C5-7 cycloalkyl, phenyl, and —C1-3 alkyl-phenyl is optionally fused to a further (second) ring;
- and
- R2a is selected from the group consisting of hydrogen and —C1-4 alkyl;
- or
- R1a and R2a together with the nitrogen atom to which they are attached form an optionally substituted 5- to 10-membered heterocyclic ring, wherein said heterocyclic ring optionally contains 1, 2, or 3 additional heteroatoms selected from the group consisting of N, S, or O, and wherein said heterocyclic ring is optionally fused to a phenyl ring;
- each Rba is independently hydrogen or —C1-4 alkyl, wherein said alkyl group is optionally substituted by 1, 2 or 3 fluorine atoms;
- and
- R3a is selected from the group consisting of cyclohexyl, phenyl, and pyridinyl, wherein said cyclohexyl, phenyl, and pyridinyl groups are optionally substituted with 1, 2 or 3 substituents each independently selected from the group consisting of halogen, hydroxy, —CN, —ORba, —SRba, —N(Rba)2, and C1-4 alkyl optionally substituted with 1, 2, or 3 substituents each independently selected from the group consisting of halogen, —CN, —ORba, and —N(Rba)2, and wherein said phenyl is optionally fused to a 5- or 6-membered heterocyclic ring.
9 more elements
Dependencies are inferred from the claim text.