KCNT1 inhibitors and methods of use
AssigneePraxis Precision Medicines, Inc.
InventorsAndrew Mark Griffin, Gabriel Martinez Botella, Brian Edward Marron, Paul S. Charifson
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Abstract
The present invention is directed to, in part, compounds and compositions useful for preventing and/or treating a neurological disease or disorder, a disease or condition relating to excessive neuronal excitability, and/or a gain-of-function mutation in a gene (e.g., KCNT1). Methods of treating a neurological disease or disorder, a disease or condition relating to excessive neuronal excitability, and/or a gain-of-function mutation in a gene such as KCNT1 are also provided herein.
Claims14
A pharmaceutical composition comprising a compound of Formula I-I:
- or a pharmaceutically acceptable salt thereof, wherein
- L is a bond or C1-6alkyl;
- X is CH or N, wherein, if X is CH, the hydrogen of CH may be substituted by R5;
- G is selected from the group consisting of phenyl, C3-10cycloalkyl, 5-10 membered heterocyclyl, and 5-10 membered heteroaryl, wherein G is not pyrazolyl;
- R2 is hydrogen;
- R3 is C1-6alkyl optionally substituted with C1-6alkoxy;
- R4 is hydrogen;
- R5 is each independently selected from the group consisting of halogen, C1-6alkyl, C1-6haloalkyl, C1-6alkoxy, C1-6haloalkoxy, and C3-10cycloalkyl;
- R6 is each independently selected from hydrogen or C1-6alkyl;
- R12 is each independently selected from the group consisting of halogen, cyano, hydroxyl, oxo, —S(O)2(C1-6alkyl), —S(O)2(C3-6cycloalkyl), —C(O)C1-6alkyl, —C(O)N(R6)2, C1-6alkyl, C1-6haloalkyl, C1-6alkoxy, C1-6haloalkoxy, C3-10cycloalkyl and phenyl;
- and
- z is 0, 1, 2, 3, or 4;
- and a pharmaceutically acceptable excipient.
A compound of Formula I-I:
- or a pharmaceutically acceptable salt thereof, wherein
- L is a bond or C1-6alkyl;
- X is CH or N, wherein, if X is CH, the hydrogen of CH may be substituted by R5;
- G is selected from the group consisting of phenyl, C3-10cycloalkyl, 5-10 membered heterocyclyl, and 5-10 membered heteroaryl, wherein G is not pyrazolyl;
- R2 is hydrogen;
- R3 is C1-6alkyl optionally substituted with C1-6alkoxy;
- R4 is hydrogen;
- R5 is each independently selected from the group consisting of halogen, C1-6alkyl, C1-6haloalkyl, C1-6alkoxy, C1-6haloalkoxy, and C3-10cycloalkyl;
- R6 is each independently selected from hydrogen or C1-6alkyl;
- R12 is each independently selected from the group consisting of halogen, cyano, hydroxyl, oxo, —S(O)2(C1-6alkyl), —S(O)2(C3-6cycloalkyl), —C(O) C1-6alkyl, —C(O) N(R6) 2, C1-6alkyl, C1-6haloalkyl, C1-6alkoxy, C1-6haloalkoxy, C3-10cycloalkyl and phenyl;
- and
- z is 0, 1, 2, 3, or 4.
Dependencies are inferred from the claim text.