Patent ID: 11858941
Assignee: PTC THERAPEUTICS, INC.
Field: Organic fine chemistry (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A compound of Formula (Ibbl):, or a form thereof, wherein:
Ra is selected from the group consisting of hydrogen, cyano, halogen, hydroxy, C1-6 alkyl, deutero-C1-4 alkyl, halo-C1-6 alkyl, C1-6 alkoxy, halo-C1-6 alkoxy, C1-6alkoxy-C1-6 alkyl, amino, C1-6 alkyl-amino, (C1-6 alkyl)2-amino, amino-C1-6 alkyl, and hydroxy-C1-6alkyl;
R1 is selected from the group consisting of C3-10 cycloalkyl and heterocyclyl,
wherein heterocyclyl in R1 is a saturated or partially unsaturated 3-7 membered monocyclic, 6-10 membered bicyclic or 13-16 membered polycyclic ring system having 1, 2, or 3 heteroatom ring members independently selected from N, O, or S, and
wherein each instance of C3-10 cycloalkyl and heterocyclyl in R1 is optionally substituted with one, two or three R3 substituents and optionally, with one additional R4 substituent, or,
wherein, alternatively, each instance of C3-10 cycloalkyl and heterocyclyl in R1 is optionally substituted with one, two, three, or four R3 substituents;

R2 is selected from the group consisting of phenyl, heterocyclyl, and heteroaryl,
wherein heterocyclyl in R2 is a saturated or partially unsaturated 3-7 membered monocyclic, 6-10 membered bicyclic or 13-16 membered polycyclic ring system having 1, 2, or 3 heteroatom ring members independently selected from N, O, or S,
wherein heteroaryl in R2 is a 3-7 membered monocyclic or 6-10 membered bicyclic ring system having 1, 2, 3, or 4 heteroatom ring members independently selected from N, O, or S, and
wherein each instance of phenyl, heterocyclyl, and heteroaryl in R2 is optionally substituted with one, two, or three R5 substituents, and optionally, with one additional R6 substituent;

R3 is, in each instance, independently selected from the group consisting of cyano, halogen, hydroxy, C1-6 alkyl, deutero-C1-4alkyl, halo-C1-6alkyl, C1-6 alkoxy, halo-C1-6 alkoxy, C1-6alkoxy-C1-6 alkyl, amino, C1-6 alkyl-amino, (C1-6 alkyl)2-amino, amino-C1-6alkyl, and hydroxy-C1-6alkyl;
R4 is selected from the group consisting of C340cycloalkyl, phenyl, heteroaryl, and heterocyclyl,
wherein heterocyclyl in R4 is a saturated or partially unsaturated 3-7 membered monocyclic, 6-10 membered bicyclic or 13-16 membered polycyclic ring system having 1, 2, or 3 heteroatom ring members independently selected from N, O, or S,
wherein heteroaryl in R4 is a 3-7 membered monocyclic or 6-10 membered bicyclic ring system having 1, 2, 3, or 4 heteroatom ring members independently selected from N, O, or S, and
wherein, each instance of C3-10cycloalkyl, phenyl, heterocyclyl, and heteroaryl in R4 is optionally substituted with one, two, or three R7 substituents;

R5 is, in each instance, independently selected from the group consisting of halogen, hydroxy, cyano, nitro, C1-6 alkyl, deutero-C1-4 alkyl, halo-C1-6 alkyl, C1-6alkoxy, halo-C1-6 alkoxy, oxime, amino, C1-6alkyl-amino, (C1-6 alkyl)2-amino, and C1-6alkyl-thio;
R6 is selected from the group consisting of phenyl and heteroaryl,
wherein heteroaryl in R6 is a 3-7 membered monocyclic or 6-10 membered bicyclic ring system having 1, 2, 3, or 4 heteroatom ring members independently selected from N, O, or S, and
wherein each instance of phenyl and heteroaryl in R6 is optionally substituted with one, two, three or four R8 substituents;

R7 is, in each instance, independently selected from the group consisting of cyano, halogen, hydroxy, C1-6 alkyl, deutero-C1-4alkyl, halo-C1-6alkyl, C1-6 alkoxy, halo-C1-6 alkoxy, C1-6alkoxy-C1-6 alkyl, amino, C1-6 alkyl-amino, (C1-6 alkyl)2-amino, amino-C1-6alkyl, and C3-10 cycloalkyl; and
R8 is, in each instance, independently selected from the group consisting of cyano, halogen, hydroxy, C1-6 alkyl, deutero-C1-4alkyl, halo-C1-6alkyl, C1-6 alkoxy, halo-C1_6 alkoxy, C1-6alkoxy-C1-6 alkyl, amino, C1-6 alkyl-amino, (C1-6 alkyl)2-amino, amino-C1-6 alkyl, and C3-10 cycloalkyl;

wherein a form of the compound is selected from the group consisting of a salt, hydrate, solvate, racemate, enantiomer, diastereomer, stereoisomer, and tautomer form thereof.