US2023219938A1PendingUtilityA1

Compounds and their methods of use

Assignee: PRAXIS PREC MEDICINES INCPriority: Feb 13, 2017Filed: Oct 4, 2022Published: Jul 13, 2023
Est. expiryFeb 13, 2037(~10.6 yrs left)· nominal 20-yr term from priority
A61K 31/423C07D 413/06A61K 45/06A61K 31/4184A61K 31/429A61K 31/4439A61K 31/497A61K 31/506A61K 31/5377C07D 235/26C07D 263/58C07D 498/04C07D 209/34C07D 413/10
70
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Claims

Abstract

The present invention is directed to, in part, fused heteroaryl compounds and compositions useful for preventing and/or treating a disease or condition relating to aberrant function of a voltage-gated, sodium ion channel, for example, abnormal late/persistent sodium current. Methods of treating a disease or condition relating to aberrant function of a sodium ion channel including Dravet syndrome or epilepsy are also provided herein.

Claims

exact text as granted — not AI-modified
1 . A method of treating a neurological disorder or a psychiatric disorder, wherein the method comprises administering to a subject in need thereof a compound of Formula (I): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is O, C(R 2a )(R 2b ), or N(R 2c ); 
 A is aryl or heteroaryl (e.g., 6-membered aryl or heteroaryl), wherein aryl and heteroaryl are substituted by one or more R 3 ; 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2a , R 2b , and R 2c  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, oxo, cyano, nitro, —OR c , —N(R d ) 2 , —C(O)R c , —C(O)OR c , or —C(O)N(R d ) 2 , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl, wherein each alkyl is optionally substituted by one or more R 6 ; 
 or two R d , taken together with the atoms to which they are attached, form a ring; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; and 
 each R 7  is independently alkyl, halo, or oxo. 
 
     
     
         2 . A method of treating a neurological disorder or a psychiatric disorder, wherein the method comprises administering to a subject in need thereof a compound of Formula (I-1): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is O, C(R 2a )(R 2b ), or N(R 2c ); 
 A is aryl or heteroaryl (e.g., 6-membered aryl or heteroaryl), wherein aryl and heteroaryl are substituted by one or more R 3 ; 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2a , R 2b , and R 2c  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, oxo, cyano, nitro, —OR c , —N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R e , —S(O) 2 N(R d ) 2 , or —C(O)N(R d ) 2 , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl, wherein each alkyl is optionally substituted by one or more R 6 ; 
 or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with —OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; and 
 each R 7  is independently alkyl, halo, oxo, —C(O)R c , or —C(O)OR c ; 
 wherein the compound is not one of the following: 
 
       
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable salt thereof. 
     
     
         3 - 24 . (canceled) 
     
     
         25 . The method of  claim 2 , wherein the compound of Formula (I-1) is a compound of Formula (I-2): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is O or C(R 2a )(R 2b ); 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2a  and R 2b  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3a  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, cyano, nitro, —C(O)N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R e , —S(O) 2 N(R d ) 2 , or —OR c , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl optionally substituted with one or more halogen; 
 or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with-OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; 
 each R 7  is independently alkyl, oxo, halo, —C(O)R c , or —C(O)OR c ; n is 0, 1, 2, 3, or 4. 
 
     
     
         26 . The method of  claim 25 , wherein the compound of formula I-2 is a compound of formula I-3: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         27 . The method of  claim 25 , wherein the compound of formula I-2 is a compound of formula I-4: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         28 . The method of  claim 25 , wherein the compound of formula I-2 is a compound of formula I-5: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         29 . The method of  claim 25 , wherein the compound of formula I-2 is a compound of formula I-6: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         30 - 35 . (canceled) 
     
     
         36 . The method of  claim 25 , wherein the compound of Formula (I-1) is a compound of Formula (I-7): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is N(R 2c ); 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2c  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3a  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, cyano, nitro, —C(O)N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R e , —S(O) 2 N(R d ) 2 , or —OR c , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl optionally substituted with one or more halogen; 
 or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with —OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; 
 each R 7  is independently alkyl, oxo, halo, —C(O)R c , or —C(O)OR c ; n is 0, 1, 2, 3, or 4; 
 
       wherein the compound is not one of the following: 
       
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable salt thereof. 
     
     
         37 - 41 . (canceled) 
     
     
         41 . The method of  claim 1 , wherein the compound is selected from: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         42 . A compound of Formula (I-1): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is O, C(R 2a )(R 2b ), or N(R 2c ); A is aryl or heteroaryl (e.g., 6-membered aryl or heteroaryl), wherein aryl and heteroaryl are substituted by one or more R 3 ; 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2a , R 2b , and R 2c  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, oxo, cyano, nitro, —OR c , —N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R e , —S(O) 2 N(R d ) 2 , or —C(O)N(R d ) 2 , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl, wherein each alkyl is optionally substituted by one or more R 6 ; or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with -OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; and 
 each R 7  is independently alkyl, halo, oxo, —C(O)R c , or —C(O)OR c ; 
 wherein the compound is not one of the following: 
 
       
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable salt thereof. 
     
     
         43 - 59 . (canceled) 
     
     
         60 . The compound of  claim 42 , wherein the compound of Formula (I-1) is a compound of Formula (I-2): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is O or C(R 2a )(R 2b ); 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2a  and R 2b  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3a  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, cyano, nitro, —C(O)N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R c , —S(O) 2 N(R d ) 2 , or —OR c , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl optionally substituted with one or more halogen; 
 or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with —OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; 
 each R 7  is independently alkyl, oxo, halo, —C(O)R c , or —C(O)OR c ; n is 0, 1, 2, 3, or 4, wherein the compound is not one of the following: 
 
       
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable salt thereof. 
     
     
         61 . The compound of  claim 60 , wherein the compound of formula I-2 is a compound of formula I-3: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         62 . The compound of  claim 60 , wherein the compound of formula I-2 is a compound of formula I-4: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         63 . The compound of  claim 60 , wherein the compound of formula I-2 is a compound of formula I-5: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         64 . The compound of  claim 60 , wherein the compound of formula I-2 is a compound of formula I-6: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         65 - 72 . (canceled) 
     
     
         73 . The compound of  claim 42 , wherein the compound of Formula (I-1) is a compound of Formula (I-7): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein:
 each of X, Y, and Z is independently N or CR′; 
 M is N(R 2c ); 
 R′ is hydrogen, alkyl, —OR c , or halogen; 
 R 1  is hydrogen, alkyl, carbocyclyl, or heterocyclyl, wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 4 ; 
 each of R 2c  is independently hydrogen or alkyl, wherein alkyl is optionally substituted by one or more R 4 ; 
 each R 3a  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OR c , wherein alkyl, carbocyclyl, and heterocyclyl are optionally substituted with one or more R 5 ; 
 each of R 4  and R 5  is independently deuterium, alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, halo, cyano, nitro, —C(O)N(R d ) 2 , —C(O)R c , —C(O)OR c , —S(O) 2 —R c , —S(O) 2 N(R d ) 2 , or —OR c , wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 7 ; 
 each R c  is independently hydrogen, alkyl, carbocyclyl, heterocyclyl, aryl, or heteroaryl, wherein alkyl, carbocyclyl, heterocyclyl, aryl, and heteroaryl is optionally substituted by one or more R 6 ; 
 each R d  is independently hydrogen or alkyl optionally substituted with one or more halogen; 
 or two R d , taken together with the atoms to which they are attached, form a heterocyclyl optionally substituted with-OH, alkoxy, or alkyl optionally substituted with alkoxy; 
 each R e  is alkyl; 
 each R 6  is independently alkyl, carbocyclyl, heterocyclyl, halo, cyano, nitro, or —OH; 
 each R 7  is independently alkyl, oxo, halo, —C(O)R c , or —C(O)OR c ; n is 0, 1, 2, 3, or 4; 
 wherein the compound is not one of the following: 
 
       
         
           
           
               
               
           
         
       
       and a pharmaceutically acceptable salt thereof 
     
     
         74 - 78 . (canceled) 
     
     
         79 . The compound of  claim 42 , wherein the compound is selected from: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         80 . A pharmaceutical composition comprising a compound of  claim 42 , or pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier. 
     
     
         81 . The method of  claim 1 , wherein the method comprises administering the pharmaceutical composition of  claim 80 . 
     
     
         82 . A method of treating a neurological disorder or a psychiatric disorder, wherein the method comprises administering to a subject in need thereof the compound of  claim 42 .

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