US2021284645A1PendingUtilityA1

Compositions useful for treating disorders related to kit

74
Assignee: BLUEPRINT MEDICINES CORPPriority: Oct 17, 2013Filed: Oct 14, 2020Published: Sep 16, 2021
Est. expiryOct 17, 2033(~7.3 yrs left)· nominal 20-yr term from priority
A61K 31/53C07D 403/14A61K 31/496A61K 45/06A61P 35/00C07D 487/04A61K 31/5377C07D 413/14C07D 417/14C07D 403/04A61P 1/04C07D 401/14A61P 43/00A61K 31/497A61P 35/02A61P 3/04
74
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Claims

Abstract

Compounds and compositions useful for treating disorders related to mutant KIT are described herein.

Claims

exact text as granted — not AI-modified
1 . A method of treating melanoma in a patient in need thereof, wherein the method comprises administering to the patient a therapeutically effective amount of a compound of Formula I: 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein
 W is selected from hydrogen and 
 
       
         
           
           
               
               
           
         
       
       wherein Ring A is selected from monocyclic or bicyclic aryl, monocyclic or bicyclic heteroaryl, cycloalkyl and heterocyclyl;
 each X and Y is independently selected from CR 1  and N; 
 Z is selected from C 1 -C 6  alkyl, cycloalkyl, monocyclic or bicyclic aryl, monocyclic or bicyclic aralkyl, monocyclic or bicyclic heteroaryl, monocyclic or bicyclic heterocyclyl, and monocyclic or bicyclic heterocyclylalkyl; wherein each of C 1 -C 6  alkyl, cycloalkyl, monocyclic or bicyclic aryl, monocyclic or bicyclic aralkyl, monocyclic or bicyclic heteroaryl, monocyclic or bicyclic heterocyclyl, monocyclic and bicyclic heterocyclylalkyl is independently substituted with 0-5 occurrences of R C ; 
 L is selected from a bond, —(C(R 2 )(R 2 )) m —, —(C 2 -C 6  alkynylene)-, —(C 2 -C 6  alkenylene)-, —(C 1 -C 6  haloalkylene)-, —(C 1 -C 6  heteroalkylene)-, —(C 1 -C 6  hydroxyalkylene)-, —C(O)—, —O—, —S—, —S(O), —S(O) 2 —, —N(R 2 )—, —O—(C 1 -C 6  alkylene)-, —(C 1 -C 6  alkylene)-O—, —N(R 2 )—C(O)—, —C(O)—N(R 2 )—, —(C 1 -C 6  alkylene)-N(R 2 )—, —N(R 2 )—(C 1 -C 6  alkylene)-, —N(R 2 )—C(O)—(C 1 -C 6  alkylene)-, —C(O)—N(R 2 )—(C 1 -C 6  alkylene)-, —N(R 2 )—S(O) 2 —, —S(O) 2 —N(R 2 )—, —N(R 2 )—S(O) 2 —(C 1 -C 6  alkylene)-, and —S(O) 2 —N(R 2 )—(C 1 -C 6  alkylene)-; 
 each R A  and R B  is independently selected from C 1 -C 6  alkyl, C 1 -C 6  cycloalkyl, C 1 -C 6  heterocyclyl, halo, C 1 -C 6  haloalkyl, C 1 -C 6  hydroxyalkyl, C 1 -C 6  heteroalkyl, monocyclic or bicyclic aralkyl, —N(R 2 )(R 2 ), cyano, and —OR 2 ; 
 each R C  is independently selected from C 1 -C 6  alkyl, C 1 -C 6  alkynyl, halo, C 1 -C 6  heteroalkyl, C 1 -C 6  haloalkyl, C 1 -C 6  haloalkoxy, C 1 -C 6  hydroxyalkyl, cycloalkyl, monocyclic or bicyclic aryl, monocyclic or bicyclic aryloxy, monocyclic or bicyclic aralkyl, monocyclic or bicyclic heterocyclyl, monocyclic or bicyclic heterocyclylalkyl, nitro, cyano, —C(O)R 2 , —OC(O)R 2 , —C(O)OR 2 , —SR 2 , —S(O) 2 R 2 , —S(O) 2 —N(R 2 )(R 2 ), —(C 1 -C 6  alkylene)-S(O) 2 —N(R 2 )(R 2 ), —N(R 2 )(R 2 ), —C(O)—N(R 2 )(R 2 ), —N(R 2 )(R 2 )—C(O)R 2 , —(C 1 -C 6  alkylene)-N(R 2 )—C(O)R 2 , —NR 2 S(O) 2 R 2 , —P(O)(R 2 )(R 2 ), and —OR 2 ; wherein each of heteroalkyl, haloalkyl, haloalkoxy, alkyl, alkynyl, cycloalkyl, aryl, aryloxy, aralkyl, heterocyclyl, heterocyclylalkyl is independently substituted with 0-5 occurrences of R a ; or 2 R C  together with the carbon atom(s) to which they are attached form a cycloalkyl or heterocyclyl ring substituted with 0-5 occurrences of R a ; 
 each of R D  and R F  is independently selected from hydrogen, C 1 -C 6  alkyl, C 1 -C 6  cycloalkyl, hydroxyl, halo, C 1 -C 6  alkoxy, C 1 -C 6  haloalkyl, —N(R 2 )(R 2 ), and cyano; 
 each R 1  is independently selected from hydrogen, C 1 -C 6  alkyl, monocyclic aralkyl, C 1 -C 6  hydroxyalkyl, halo, C 1 -C 6  haloalkyl, —N(R 2 )(R 2 ), and —OR 2 ; 
 each R 2  is independently selected from hydrogen, hydroxyl, halo, thiol, C 1 -C 6  thioalkyl, —NR″R″, C 1 -C 6  alkyl, C 1 -C 6  alkoxy, C 1 -C 6  haloalkyl, C 1 -C 6  hydroxyalkyl, cycloalkyl, cycloalkylalkyl, heterocyclyl, and heterocyclylalkyl, wherein each of C 1 -C 6  alkyl, cycloalkyl and heterocyclyl is independently substituted with 0-5 occurrences of R b , or 2 R 2  together with the atoms to which they are attached form a cycloalkyl or heterocyclyl ring; 
 each R a  and R b  is independently selected from hydrogen, halo, cyano, hydroxyl, C 1 -C 6  alkoxyl, —C(O)R′, C(O)OR′, C 1 -C 6  alkyl, C 1 -C 6  haloalkyl, C 1 -C 6  heteroalkyl, C 1 -C 6  hydroxyalkyl, —NR′R′, and cycloalkyl, wherein cycloalkyl is substituted with 0-5 occurrences of R′; 
 each R′ is hydrogen, hydroxyl, or C 1 -C 6  alkyl; 
 each R″ is hydrogen, C 1 -C 6  alkyl, —C(O)—C 1 -C 6  alkyl, —C(O)—NR′R′; or —C(S)—NR′R′; and 
 m, p, and q are each independently 0, 1, 2, 3, or 4. 
 
     
     
         2 . The method of  claim 1 , wherein the compound is a compound of Formula II: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         3 . The method of  claim 1 , wherein the compound is a compound of Formula III: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof. 
       
     
     
         4 . The method of  claim 1 , wherein at least one of X and Y is N. 
     
     
         5 . (canceled) 
     
     
         6 . The method of  claim 1 , wherein L is —(C(R 2 )(R 2 )) m —. 
     
     
         7 . The method of  claim 1 , wherein A is monocyclic or bicyclic aryl. 
     
     
         8 . The method of  claim 1 , wherein Z is monocyclic or bicyclic aryl. 
     
     
         9 . The method of  claim 1 , wherein Z is monocyclic or bicyclic heteroaryl. 
     
     
         10 . The method of  claim 1 , wherein Z is monocyclic heteroaryl. 
     
     
         11 . The method of  claim 1 , wherein Z is selected from pyrazolyl, isoxazolyl, thiophenyl, thiazolyl, and pyridyl. 
     
     
         12 . The method of  claim 1 , wherein Z is phenyl. 
     
     
         13 . The method of  claim 1 , wherein Z is monocyclic or bicyclic heterocyclyl. 
     
     
         14 . The method of  claim 1 , wherein R A  is fluoro and q is 1. 
     
     
         15 . A method of treating melanoma in a patient in need thereof, wherein the method comprises administering to the patient a therapeutically effective amount of a compound selected from the group consisting of the compounds below, or a pharmaceutically acceptable salt thereof: 
       
         
           
                 
                 
               
                     
                 
                   Compound 
                     
                 
                   Number 
                   Structure 
                 
                     
                 
                     
                 
                 
                 
               
                   1 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   2 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   4 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   6 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   7 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   8 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   9 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   10 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   11 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   12 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   13 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   14 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   15 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   16 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   17 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   18 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   19 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   20 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   21 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   22 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   23 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   24 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   25 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   26 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   27 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   28 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   29 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   30 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   31 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   32 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   33 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   34 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   35 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   36 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   37 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   38 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   39 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   40 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   41 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   42 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   43 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   44 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   45 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   46 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   47 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   48 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   49 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   50 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   51 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   52 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   53 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   54 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   55 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   56 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   57 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   58 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   59 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   60 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   61 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   62 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   63 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   64 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   65 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   66 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   67 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   68 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   69 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   70 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   71 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   72 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   73 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   74 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   75 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   76 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   77 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   78 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   79 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   80 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   81 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   82 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   83 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   84 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   85 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   86 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   87 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   88 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   89 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   90 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   91 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   92 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   93 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   95 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   96 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   97 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   94 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   98 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   99 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   100 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   101 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   102 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   103 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   104 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   105 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   106 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   107 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   108 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   109 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   110 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   111 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   112 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   113 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   114 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   115 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   116 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   117 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   118 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   119 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   120 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   121 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   122 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   123 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
                   124 
                   
                     
                       
                       
                           
                           
                       
                     
                   
                 
                     
                 
             
                
                
                
                
               
               
                
               
            
             
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
                
               
            
           
         
       
     
     
         16 .- 19 . (canceled) 
     
     
         20 . The method of  claim 1 , wherein the patient has a mutation in Exon 17 in KIT. 
     
     
         21 . The method of  claim 20 , wherein the patient has a D816 mutation in KIT in Exon 17. 
     
     
         22 . The method of  claim 21 , wherein the D816 mutation is D816V. 
     
     
         23 .- 24 . (canceled) 
     
     
         25 . A method of treating systemic mastocytosis in a patient in need thereof, wherein the systemic mastocytosis is selected from aggressive systemic mastocytosis (ASM), SM with associated hematologic non-mast cell lineage disease (SM-AHNMD), and mast cell leukemia, wherein the method comprises administering to the patient once daily
 i) 200-300 mg of a compound which is:   
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, or
 ii) a pharmaceutical composition comprising a pharmaceutically acceptable carrier and 200-300 mg of the compound or a pharmaceutically acceptable salt thereof. 
 
     
     
         26 .- 31 . (canceled) 
     
     
         32 . The method of  claim 25 , wherein the patient is administered once daily
 i) 200 mg of the compound or a pharmaceutically acceptable salt thereof; or   ii) a pharmaceutical composition comprising a pharmaceutically acceptable carrier and 200 mg of the compound or a pharmaceutically acceptable salt thereof.

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