US2023047589A1PendingUtilityA1

Inhibitors of integrated stress response pathway

Assignee: Praxis Biotech LLCPriority: Dec 13, 2017Filed: Aug 10, 2021Published: Feb 16, 2023
Est. expiryDec 13, 2037(~11.4 yrs left)· nominal 20-yr term from priority
C07D 401/12C07D 491/048C07D 211/58A61P 21/00C07D 265/36C07C 235/36A61K 31/428A61P 35/00C07D 413/12C12N 5/0018A61P 25/28C07C 231/12C07D 263/24C07D 215/18C07D 307/85C07D 277/68A61P 29/00C07D 405/12A61K 31/4525A61K 31/445C07C 231/14C12N 2500/32A61K 31/343
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Claims

Abstract

The present disclosure relates generally to therapeutic agents that may be useful as inhibitors of Integrated Stress Response (ISR) pathway.

Claims

exact text as granted — not AI-modified
1 - 9 . (canceled) 
     
     
         10 . A compound of formula (XX-I-2): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof; 
       wherein:
 R Y5  is hydrogen or C 1 -C 6  alkyl; 
 R N  is hydrogen or C 1 -C 6  alkyl; 
 A 13  is selected from the group consisting of:
 C 6 -C 10  aryl optionally substituted with one or more R 95  substituents; and 
 5-10 membered heteroaryl optionally substituted with one or more R 95  substituents; 
 
 R 95  is selected, independently at each occurrence, from the group consisting of halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 95-a R 95-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl), —C(O)NR 95-a R 95-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 95-a R 95-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl);
 wherein R 95-a  and R 95-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 
 R 88  is selected from the group consisting of hydrogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —C(O)(C 1 -C 6  alkyl), —C(O)(C 1 -C 6  haloalkyl), —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 88-a R 88-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl), and —S(O) 2 NR 88-a R 88-b ; 
 wherein R 88-a  and R 88-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 R 89  is selected, independently at each occurrence, from the group consisting of hydrogen, halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 89-a R 89-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 89-a R 89-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 89-a R 89-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl), 
 wherein R 89-a  and R 89-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle. 
 
     
     
         11 - 15 . (canceled) 
     
     
         16 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein the compound is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         17 . A pharmaceutical composition comprising a compound of  claim 10 , or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier. 
     
     
         18 . A method of treating a disease or disorder mediated by an integrated stress response (ISR) pathway in an individual in need thereof comprising administering to the individual a therapeutically effective amount of a compound of  claim 10 , or a pharmaceutically acceptable salt thereof. 
     
     
         19 . A method of producing a protein, comprising contacting a eukaryotic cell comprising a nucleic acid encoding the protein with the compound of  claim 10 , or a salt thereof. 
     
     
         20 . A method of culturing a eukaryotic cell comprising a nucleic acid encoding a protein, comprising contacting the eukaryotic cell with an in vitro culture medium comprising the compound of  claim 10 , or a salt thereof. 
     
     
         21 . A method of producing a protein, comprising contacting a cell-free protein synthesis (CFPS) system comprising eukaryotic initiation factor 2 (eIF2) and a nucleic acid encoding a protein with the compound of  claim 10 , or a salt thereof. 
     
     
         22 . An in vitro cell culture medium, comprising the compound of  claim 10 , or a salt thereof and nutrients for cellular growth. 
     
     
         23 . A cell-free protein synthesis (CFPS) system comprising eukaryotic initiation factor 2 (eIF2) and a nucleic acid encoding a protein with the compound of  claim 10 , or a salt thereof. 
     
     
         24 . The method of  claim 18 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a neurodegenerative disease, an inflammatory disease, an autoimmune disease, a metabolic syndrome, a cancer, a vascular disease, a musculoskeletal disease, an ocular disease, or a genetic disorder. 
     
     
         25 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a neurodegenerative disease. 
     
     
         26 . The method of  claim 25 , wherein the neurodegenerative disease is vanishing white matter disease. 
     
     
         27 . The method of  claim 25 , wherein the neurodegenerative disease is amyotrophic lateral sclerosis (ALS). 
     
     
         28 . The method of  claim 25 , wherein the neurodegenerative disease is frontotemporal dementia (FTD). 
     
     
         29 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is an inflammatory disease. 
     
     
         30 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is an autoimmune disease. 
     
     
         31 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a metabolic syndrome. 
     
     
         32 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a cancer. 
     
     
         33 . The method of  claim 32 , wherein the cancer is prostate cancer. 
     
     
         34 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a musculoskeletal disease. 
     
     
         35 . The method of  claim 34 , wherein the musculoskeletal disease is muscular atrophy. 
     
     
         36 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a genetic disorder. 
     
     
         37 . The method of  claim 36 , wherein the genetic disorder is Down syndrome. 
     
     
         38 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is a vascular disease. 
     
     
         39 . The method of  claim 24 , wherein the disease or disorder mediated by an integrated stress response (ISR) pathway is an ocular disease. 
     
     
         40 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein R Y5  is hydrogen. 
     
     
         41 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein R N  is hydrogen. 
     
     
         42 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein A 13  is C 6 -C 10  aryl optionally substituted with one or more R 95  substituents. 
     
     
         43 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein A 13  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       wherein the * represents the attachment point to the remainder of the molecule. 
     
     
         44 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein A 13  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       wherein the * represents the attachment point to the remainder of the molecule. 
     
     
         45 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein A 13  is 
       
         
           
           
               
               
           
         
       
       wherein the * represents the attachment point to the remainder of the molecule. 
     
     
         46 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein A 13  is 
       
         
           
           
               
               
           
         
       
       wherein the * represents the attachment point to the remainder of the molecule. 
     
     
         47 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein R 88  is hydrogen. 
     
     
         48 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein R 89  is selected, independently at each occurrence, from the group consisting of hydrogen and halogen. 
     
     
         49 . The compound of  claim 10 , or a pharmaceutically acceptable salt thereof, wherein the moiety 
       
         
           
           
               
               
           
         
       
       wherein # represents the attachment point to the remainder of the molecule, is 
       
         
           
           
               
               
           
         
       
       wherein # represents the attachment point to the remainder of the molecule. 
     
     
         50 . A compound of formula (1-3): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof; 
       wherein:
 X 2  is CH or N; 
 R Y1  is hydrogen or C 1 -C 6  alkyl; 
 Y 2  is selected from the group consisting of a bond, NR Y2 , and O; provided that when X 2  is N, then Y 2  is a bond; 
 R Y2  is hydrogen or C 1 -C 6  alkyl; 
 r and s, independently of each other, are 0, 1, or 2; 
 A 1  is selected from the group consisting of:
 a substituent of formula (A 1 -a) 
 
 
       
         
           
           
               
               
           
         
         
           
             wherein
 * represents the attachment point to the remainder of the molecule; 
 Z 1  is selected from the group consisting of CR Z1-1 R Z1-2 , NR Z1-2  O, S, and —CR Z1-1 ═CR Z1-1 —; 
  wherein R Z1-1  is H or R″; and R Z1-2  is H or R 14 ; 
 Z 2  is selected from the group consisting of CR Z2-1 R Z2-2 , NR Z2-2 ; O, S, and —CR Z2-1 ═CR Z2-1 —; 
  wherein R Z2-1  is H or R 14 ; and R Z2-2  is H or R 14 ; 
 Z 3 , independently at each occurrence, is C or N, provided that at least one Z 3  is C; 
 R 13  is hydrogen or R 14 , or R 13  and R Z1-2  are taken together to form a double bond between the carbon atom bearing R 13  and Z 1 , or R 13  and R Z2-2  are taken together to form a double bond between the carbon atom bearing R 13  and Z 2 ; and 
 x1 is 1, 2, 3, or 4, and at least one R 14  is halogen; 
 
           
         
         R 14  is selected, independently at each occurrence, from the group consisting of halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 14-a R 14-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 14-a R 14-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 14-a R 14-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl);
 wherein R 14-a  and R 14-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 
         A 2  is C 6 -C 10  aryl substituted by at least one halogen substituent and optionally further substituted with one or more R 16  substituents, or 5-10 membered heteroaryl substituted by at least one halogen substituent and optionally further substituted with one or more R 16  substituents; 
         R 16  is selected, independently at each occurrence, from the group consisting of halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 16-a R 16-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 16-a R 16-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 16-a R 16-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl);
 wherein R 16-a  and R 16-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 
         R 1a  and R 1b  are independently selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
         R 2a  and R 2b  are independently selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
         when present, R 3a  and R 3b  are independently at each occurrence selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
         when present, R 4a  and R 4b  are independently at each occurrence selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
         or alternatively, R 1a  and R 2a  are taken together to form a C 1 -C 6  alkylene moiety; 
         or alternatively, R 1a  and an R 3a  moiety, when present, are taken together to form a C 1 -C 6  alkylene moiety, and R 1b  and the R 3b  in the geminal position to the R 3a  taken together with R 1a , are both hydrogen; 
         or alternatively, an R 3a  moiety, when present, and an R 4a  moiety, when present, are taken together to form a C 1 -C 6  alkylene moiety, and the R 3b  in the geminal position to the R 3a  taken together with the R 4a  moiety and the R 4b  in the geminal position to the R 4a  taken together with the R 3a  moiety, are both hydrogen; 
         R 9a  and R 9b  are taken together to form an oxo (═O) substituent or an imido (═NH) substituent, or alternatively, R 9a  and R 9b  are both hydrogen; 
         R 10a  is selected from the group consisting of hydrogen, —OR 10a-a , and —NR 10a-b R 10a-c ; 
         R 10b  is hydrogen; 
         R 12a  and R 12b  are taken together to form an oxo (═O) substituent, or alternatively, R 12a  and R 12b  are both hydrogen; 
         R 10a-a  is selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and C 1 -C 6  haloalkyl; 
         or R 10a-a  and R Y2  may be taken together to form a carbonyl (C═O) moiety; and 
         R 10a-b  and R 10a-c , independently of each other, are selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and C 1 -C 6  haloalkyl. 
       
     
     
         51 . A compound of formula (2-3): 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof; 
       wherein:
 X 1  and X 2 , independently of each other, are CH or N; provided that at least one of X 1  and X 2  is CH; 
 Y 1  is selected from the group consisting of a bond, NR Y1 , and O; provided that when X 1  is N, then Y 1  is a bond; 
 R Y1  is hydrogen or C 1 -C 6  alkyl; 
 Y 2  is selected from the group consisting of a bond, NR Y2 , and O; provided that when X 2  is N, then Y 2  is a bond; 
 R Y2  is hydrogen or C 1 -C 6  alkyl; 
 q1 is 1; 
 r and s, independently of each other, are 0, 1, or 2; 
 A 1  is C 6 -C 10  aryl substituted by at least one halogen substituent and optionally further substituted with one or more R 14  substituents, or 5-10 membered heteroaryl substituted by at least one halogen substituent and optionally further substituted with one or more R 14  substituents; 
 R 14  is selected, independently at each occurrence, from the group consisting of halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 14-a R 14-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 14-a R 14-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 14-a R 14-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl);
 wherein R 14-a  and R 14-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 
 A 2  is C 6 -C 10  aryl substituted by at least one halogen substituent and optionally further substituted with one or more R 16  substituents, or 5-10 membered heteroaryl substituted by at least one halogen substituent and optionally further substituted with one or more R 16  substituents; 
 R 16  is selected, independently at each occurrence, from the group consisting of halogen, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, —OH, —O(C 1 -C 6  alkyl), —O(C 1 -C 6  haloalkyl), —SH, —S(C 1 -C 6  alkyl), —S(C 1 -C 6  haloalkyl), —NH 2 , —NH(C 1 -C 6  alkyl), —NH(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl) 2 , —N(C 1 -C 6  haloalkyl) 2 , —NR 16-b , —CN, —C(O)OH, —C(O)O(C 1 -C 6  alkyl), —C(O)O(C 1 -C 6  haloalkyl), —C(O)NH 2 , —C(O)NH(C 1 -C 6  alkyl), —C(O)NH(C 1 -C 6  haloalkyl), —C(O)N(C 1 -C 6  alkyl) 2 , —C(O)N(C 1 -C 6  haloalkyl) 2 , —C(O)NR 16-a R 16-b , —S(O) 2 OH, —S(O) 2 O(C 1 -C 6  alkyl), —S(O) 2 O(C 1 -C 6  haloalkyl), —S(O) 2 NH 2 , —S(O) 2 NH(C 1 -C 6  alkyl), —S(O) 2 NH(C 1 -C 6  haloalkyl), —S(O) 2 N(C 1 -C 6  alkyl) 2 , —S(O) 2 N(C 1 -C 6  haloalkyl) 2 , —S(O) 2 NR 16-a R 16-b , —OC(O)H, —OC(O)(C 1 -C 6  alkyl), —OC(O)(C 1 -C 6  haloalkyl), —N(H)C(O)H, —N(H)C(O)(C 1 -C 6  alkyl), —N(H)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)C(O)H, —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)C(O)(C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)C(O)H, —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  alkyl), —N(C 1 -C 6  haloalkyl)C(O)(C 1 -C 6  haloalkyl), —OS(O) 2 (C 1 -C 6  alkyl), —OS(O) 2 (C 1 -C 6  haloalkyl), —N(H)S(O) 2 (C 1 -C 6  alkyl), —N(H)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  alkyl), —N(C 1 -C 6  alkyl)S(O) 2 (C 1 -C 6  haloalkyl), —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  alkyl), and —N(C 1 -C 6  haloalkyl)S(O) 2 (C 1 -C 6  haloalkyl);
 wherein R 16-a  and R 16-b  are taken together with the nitrogen atom which bears them to form a 3-10 membered heterocycle; 
 
 R 1a  and R 1b  are independently selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
 R 2a  and R 2b  are independently selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
 when present, R 3a  and R 3b  are independently at each occurrence selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
 when present, R 4a  and R 4b  are independently at each occurrence selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and halogen; 
 or alternatively, R 1a  and R 2a  are taken together to form a C 1 -C 6  alkylene moiety; 
 or alternatively, R 1a  and an R 3a  moiety, when present, are taken together to form a C 1 -C 6  alkylene moiety, and R 1b  and the R 3b  in the geminal position to the R 3a  taken together with R 1a , are both hydrogen; 
 or alternatively, an R 3a  moiety, when present, and an R 4a  moiety, when present, are taken together to form a C 1 -C 6  alkylene moiety, and the R 3b  in the geminal position to the R 3a  taken together with the R 4a  moiety and the R 4b  in the geminal position to the R 4a  taken together with the R 3a  moiety, are both hydrogen; 
 R 5a  and R 5b  are taken together to form an oxo (═O) substituent or an imido (═NH) substituent, or alternatively; 
 R 6a  is hydrogen; 
 R 6b  is hydrogen; 
 R 9a  and R 9b  are taken together to form an oxo (═O) substituent or an imido (═NH) substituent, or alternatively, R 9a  and R 9b  are both hydrogen; 
 R 10a  is selected from the group consisting of hydrogen, —OR 10a-a , and —NR 10a-b R 10a-c ; 
 R 10b  is hydrogen; 
 R 12a  and R 12b  are taken together to form an oxo (═O) substituent, or alternatively, R 12a  and R 12b  are both hydrogen; 
 R 10a-a  is selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and C 1 -C 6  haloalkyl; 
 or R 10a-a  and R Y2  may be taken together to form a carbonyl (C═O) moiety; 
 R 10a-b  and R 10a-c , independently of each other, are selected from the group consisting of hydrogen, C 1 -C 6  alkyl, and C 1 -C 6  haloalkyl; and 
 provided that when X 2  is N, then: 
 A 1  is C 6 -C 10  aryl substituted by at least two halogen substituents and optionally further substituted with one or more 10 4  substituents, or 5-10 membered heteroaryl substituted by at least two halogen substituents and optionally further substituted with one or more R 14  substituents; and 
 A 2  is C 6 -C 10  aryl substituted by at least two halogen substituents and optionally further substituted with one or more 10 6  substituents, or 5-10 membered heteroaryl substituted by at least two halogen substituents and optionally further substituted with one or more R 16  substituents.

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