US2021009519A1PendingUtilityA1

Histone deacetylase inhibitors

Assignee: BIOMARIN PHARM INCPriority: Jul 2, 2015Filed: Sep 22, 2020Published: Jan 14, 2021
Est. expiryJul 2, 2035(~8.9 yrs left)· nominal 20-yr term from priority
A61K 31/44A61P 25/28A61P 31/00C07D 451/04A61P 25/30C07D 207/14C07D 451/06C07D 223/12C07D 211/58C07D 205/04C07D 207/12C07D 223/08A61K 31/55A61K 31/40A61P 25/02C07D 211/46A61K 31/4409A61P 25/00A61P 29/00C07D 451/02C07D 211/54A61P 35/00A61P 43/00A61K 31/46A61P 25/14A61P 21/00
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Claims

Abstract

Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A compound having a structure of formula (I), or a pharmaceutically acceptable salt thereof 
       
         
           
           
               
               
           
         
       
       wherein
 ring A is a 4-7 membered heterocycloalkyl ring containing one nitrogen ring atom or a 7-9 membered bicyclic heterocycloalkyl ring containing one nitrogen ring atom; 
 Z is O, NR 3 , S, SO, or SO 2 ; 
 R 1  is H, C 1-6  alkyl, C 1-6 hydroxyalkyl, C(O)C 1-6  alkyl, C 0-3  alkylene-C 3-10 cycloalkyl, or C 0-3  alkylene-C 2-5  cycloheteroalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl); 
 R 2  is H, F, Cl, or CH 3 ; 
 R 3  is H, C 1-6  alkyl, C 1-6  haloalkyl, C 0-3  alkylene-C 3-7 cycloalkyl, C(O)C 1-6  alkyl, or C(O)C 0-3 alkylene-C 3-7  cycloalkyl; and 
 R 4  is H or C 1-3  alkyl. 
 
     
     
         2 . The compound of  claim 1 , wherein ring A is a 4-7 membered heterocycloalkyl ring containing one nitrogen ring atom or a 7-9 membered bicyclic heterocycloalkyl ring containing one nitrogen ring atom; Z is O, NR 3 , S, or SO 2 ; R 1  is C 1-6  alkyl, C 1-6 hydroxyalkyl, or C 0-3  alkylene-C 3-7 cycloalkyl; R 2  is H, F, Cl, or CH 3 ; R 3  is H, C 1-6  alkyl, C 1-6  haloalkyl, C 0-3 alkylene-C 3-7  cycloalkyl, C(O)C 1-6  alkyl, or C(O)C 0-3  alkylene-C 3-7  cycloalkyl; and R 4  is H or C 1-3 alkyl. 
     
     
         3 . The compound of  claim 1  or  2 , wherein ring A is a 7-9 membered bicyclic heterocycloalkyl ring containing one nitrogen ring atom. 
     
     
         4 . The compound of  claim 1  or  2 , wherein ring A is a 4-7 membered heterocycloalkyl ring containing one nitrogen ring atom. 
     
     
         5 . The compound of any one of  claims 1  to  4 , wherein Z is O or NR 3 . 
     
     
         6 . The compound of any one of  claims 1  to  4 , wherein R 1  is C 1-6  alkyl, C 1-6 hydroxyalkyl, C(O)C 1-6  alkyl, C 0-3  alkylene-C 3-10 cycloalkyl, or C 0-3  alkylene-C 2-5 heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         7 . The compound of any one of  claims 1  to  4 , wherein R 1  is H, C 1-6  alkyl, C 1-6 hydroxyalkyl, C 0-3  alkylene-C 3-10 cycloalkyl, or C 0-3 alkylene-C 2-5  heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         8 . The compound of  claim 7 , wherein R 1  is C 1-6  alkyl, C 1-6  hydroxyalkyl, C 0-3 alkylene-C 3-10 cycloalkyl, or C 0-3  alkylene-C 2-5  heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         9 . The compound of  claim 7 , wherein R 1  is C 1-6  alkyl, C 1-6  hydroxyalkyl, or C 0-3 alkylene-C 3-10 cycloalkyl. 
     
     
         10 . The compound of  claim 7 , wherein R 1  is H. 
     
     
         11 . The compound of  claim 7 , wherein R 1  is a C 1-6  alkyl. 
     
     
         12 . The compound of  claim 7 , wherein R 1  is methyl, isopropyl, sec-butyl, or CH 2 C(CH 3 ) 3 . 
     
     
         13 . The compound of  claim 7 , wherein R 1  is isopropyl, sec-butyl, or CH 2 C(CH 3 ) 3 . 
     
     
         14 . The compound of  claim 7 , wherein R 1  is C 1-6  hydroxyalkyl. 
     
     
         15 . The compound of  claim 7 , wherein R 1  is 
       
         
           
           
               
               
           
         
       
     
     
         16 . The compound of  claim 7 , wherein R 1  is C 3-10 cycloalkyl or C 1-3  alkylene-C 3-10 cycloalkyl. 
     
     
         17 . The compound of  claim 7 , wherein R 1  is C 3-10 cycloalkyl or CH 2 alkylene-C 3-10 cycloalkyl. 
     
     
         18 . The compound of  claim 7 , wherein R 1  is C 3-6  cycloalkyl or C 1-3  alkylene-C 3-6 cycloalkyl. 
     
     
         19 . The compound of  claim 7 , wherein R 1  is C 3-6  cycloalkyl or CH 2 C 3-6  cycloalkyl. 
     
     
         20 . The compound of  claim 7 , wherein R 1  is C 3-6  cycloalkyl. 
     
     
         21 . The compound of  claim 7 , wherein R 1  is CH 2 C 3-6  cycloalkyl. 
     
     
         22 . The compound of any one of  claims 16  to  22 , wherein the cycloalkyl group is cyclopropyl, cyclobutyl, or cyclohexyl. 
     
     
         23 . The compound of  claim 22 , wherein the cycloalkyl group is cyclopropyl. 
     
     
         24 . The compound of  claim 7 , wherein R 1  is C 0-3  alkylene-C 10 cycloalkyl. 
     
     
         25 . The compound of  claim 24 , wherein R 1  is C 10 cycloalkyl or C 1-3  alkylene-C 10 cycloalkyl. 
     
     
         26 . The compound of  claim 25 , wherein R 1  is C 10 cycloalkyl or CH 2 —C 10 cycloalkyl. 
     
     
         27 . The compound of  claim 26 , wherein R 1  is adamantanyl or CH 2 -adamantanyl. 
     
     
         28 . The compound of  claim 27 , wherein R 1  is CH 2 -adamantanyl. 
     
     
         29 . The compound of  claim 7 , wherein R 1  is cyclopropyl, cyclobutyl, cyclohexyl, adamantanyl, CH 2 cyclopropyl, (1-methylcyclopropyl)methyl, CH 2 cyclobutyl, CH 2 cyclohexyl or CH 2 -adamantanyl. 
     
     
         30 . The compound of  claim 7 , wherein R 1  is cyclopropyl, cyclobutyl, or cyclohexyl. 
     
     
         31 . The compound of  claim 7 , wherein R 1  is cyclopropyl. 
     
     
         32 . The compound of  claim 7 , wherein R 1  is CH 2 cyclopropyl, (1-methylcyclopropyl)methyl, CH 2 cyclobutyl, CH 2 cyclohexyl, or CH 2 -adamantanyl. 
     
     
         33 . The compound of  claim 7 , wherein R 1  is cyclopropyl, CH 2 cyclopropyl, (1-methylcyclopropyl)methyl, CH 2 cyclohexyl or CH 2 -adamantanyl. 
     
     
         34 . The compound of  claim 7 , wherein R 1  is C 0-3  alkylene-C 2-5  heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         35 . The compound of  claim 7 , wherein R 1  is C 2-5  heterocycloalkyl or C 1-3  alkylene-C 2-5  heterocycloalkyl, with each heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         36 . The compound of  claim 7 , wherein R 1  is C 2-5  heterocycloalkyl or CH 2 —C 2-5 heterocycloalkyl, with each heterocycloalkyl having 1 or 2 heteroatoms selected from O, S, N, and N(C 1-4 alkyl). 
     
     
         37 . The compound of any one of  claims 34  to  36 , wherein the C 2-5 heterocycloalkyl is azetidinyl, pyrrolidinyl, piperidinyl, piperazinyl, oxetanyl, tetrahydrofuranyl, tetrahydropyranyl, or morpholinyl. 
     
     
         38 . The compound of  claim 37 , wherein the C 2-5  heterocycloalkyl is azetidinyl, pyrrolidinyl, piperidinyl, or piperazinyl. 
     
     
         39 . The compound of  claim 37 , wherein the C 2-5  heterocycloalkyl is oxetanyl, tetrahydrofuranyl, or tetrahydropyranyl. 
     
     
         40 . The compound of  claim 37 , wherein the C 2-5  heterocycloalkyl is oxetanyl. 
     
     
         41 . The compound of  claim 1 , wherein the compound of formula (I) is a compound of formula (II), or a pharmaceutically acceptable salt thereof 
       
         
           
           
               
               
           
         
       
       wherein
 ring A is selected from the group consisting of: 
 
       
         
           
           
               
               
           
         
         Z is O, NR 3 , S, SO, or SO 2 ; 
         R 1  is selected from the group consisting of 
       
       
         
           
           
               
               
           
         
         R 2  is H, F, Cl, or CH 3 ; 
         R 3  is H, C 1-6  alkyl, C 1-6  haloalkyl, C 0-3  alkylene-C 3-7 cycloalkyl, C(O)C 1-6  alkyl, or C(O)C 0-3 alkylene-C 3-7  cycloalkyl; and 
         R 4  is H or C 1-3  alkyl. 
       
     
     
         42 . The compound of  claim 41 , wherein Z is O, NR 3 , S, or SO 2 ; and R 1  is selected from the group consisting of H, CH 3 , C(O)CH 3 , 
       
         
           
           
               
               
           
         
       
     
     
         43 . The compound of  claim 1 , wherein the compound of formula (I) is a compound of formula (IIa), or a pharmaceutically acceptable salt thereof: 
       
         
           
           
               
               
           
         
       
       wherein ring A is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       Z is O, NR 3 , S, SO, or SO 2 ; R 1  is selected from the group consisting of H, CH 3 , C(O)CH 3 , 
       
         
           
           
               
               
           
         
       
       and R 3  is H, C 1-6  alkyl, C 1-6  haloalkyl, C 0-3  alkylene-C 3-7 cycloalkyl, C(O)C 1-6  alkyl, or C(O)C 0-3  alkylene-C 3-7  cycloalkyl. 
     
     
         44 . The compound of any one of  claims 1  to  43 , wherein Z is S, SO, or SO 2 . 
     
     
         45 . The compound of  claim 44 , wherein Z is S. 
     
     
         46 . The compound of  claim 44 , wherein Z is SO. 
     
     
         47 . The compound of  claim 44 , wherein Z is SO 2 . 
     
     
         48 . The compound of any one of  claims 1  to  43 , wherein Z is O or NR 3 . 
     
     
         49 . The compound of  claim 48 , wherein Z is O. 
     
     
         50 . The compound of  claim 48 , wherein Z is NR 3 . 
     
     
         51 . The compound of  claim 48  or  50 , wherein R 3  is H, C 1-6  alkyl, or C 0-3  alkylene-C 3-7  cycloalkyl. 
     
     
         52 . The compound of  claim 51 , wherein R 3  is H, C 1-6  alkyl, or C 3-7 cycloalkyl. 
     
     
         53 . The compound of  claim 51 , wherein R 3  is H, C 1-6  alkyl, or C 1-3  alkylene-C 3-7 cycloalkyl. 
     
     
         54 . The compound of  claim 51 , wherein R 3  is H, C 1-6  alkyl, or C 3-6  cycloalkyl. 
     
     
         55 . The compound of  claim 51 , wherein R 3  is H, C 1-6  alkyl, or C 1-3  alkylene-C 3-6 cycloalkyl. 
     
     
         56 . The compound of  claim 51 , wherein R 3  is H. 
     
     
         57 . The compound of  claim 51 , wherein R 3  is C 1-6  alkyl. 
     
     
         58 . The compound of  claim 51 , wherein R 3  is methyl, ethyl, or propyl. 
     
     
         59 . The compound of  claim 51 , wherein R 3  is methyl or ethyl. 
     
     
         60 . The compound of  claim 51 , wherein R 3  is methyl. 
     
     
         61 . The compound of  claim 51 , wherein R 3  is C 3-7 cycloalkyl or C 1-3  alkylene-C 3-7 cycloalkyl. 
     
     
         62 . The compound of  claim 51 , wherein R 3  is C 3-6  cycloalkyl or CH 2 C 3-6 cycloalkyl. 
     
     
         63 . The compound of  claim 61  or  62 , wherein the cycloalkyl group is cyclopropyl, cyclobutyl, or cyclohexyl. 
     
     
         64 . The compound of  claim 63 , wherein the cycloalkyl group is cyclopropyl. 
     
     
         65 . The compound of  claim 51 , wherein R 3  is H, methyl, ethyl, or CH 2 cyclopropyl. 
     
     
         66 . The compound of  claim 51 , wherein R 3  is H, methyl, or CH 2 cyclopropyl. 
     
     
         67 . The compound of  claim 48  or  50 , wherein R 3  is C(O)C 1-6  alkyl. 
     
     
         68 . The compound of  claim 67 , wherein R 3  is C(O)methyl, C(O)ethyl, or C(O)propyl. 
     
     
         69 . The compound of  claim 48  or  50 , wherein R 3  is C(O)C 0-3  alkylene-C 3-7 cycloalkyl. 
     
     
         70 . The compound of  claim 69 , wherein R 3  is C(O)CH 2 —C 3-7 cycloalkyl or C(O)C 3-7  cycloalkyl. 
     
     
         71 . The compound of  claim 69  or  70 , wherein the C 3-7 cycloalkyl is cyclopropyl. 
     
     
         72 . The compound of any one of  claims 1  to  71 , wherein ring A is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
     
     
         73 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         74 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         75 . The compound of any one of  claims 1  to  71 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         76 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         77 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         78 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         79 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         80 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         81 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         82 . The compound of  claim 72 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         83 . The compound of  claim 75 , wherein ring A is 
       
         
           
           
               
               
           
         
       
     
     
         84 . The compound of any one of  claims 1  to  83 , wherein R 2  is H. 
     
     
         85 . The compound of any one of  claims 1  to  83 , wherein R 2  is F. 
     
     
         86 . The compound of any one of  claims 1  to  83 , wherein R 2  is Cl. 
     
     
         87 . The compound of any one of  claims 1  to  83 , wherein R 2  is CH 3 . 
     
     
         88 . The compound of any one of  claims 1  to  87 , wherein R 4  is H. 
     
     
         89 . The compound of any one of  claims 1  to  87 , wherein R 4  is C 1-3  alkyl. 
     
     
         90 . The compound of  claim 89 , wherein R 4  is methyl. 
     
     
         91 . The compound of any one of  claims 1  to  90 , wherein R 1  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
     
     
         92 . The compound of  claim 1 , wherein ring A is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       Z is O or NR 3 ; and R 1  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
     
     
         93 . The compound of  claim 1 , wherein the compound of formula (I) is a compound of formula (III), or a pharmaceutically acceptable salt thereof 
       
         
           
           
               
               
           
         
       
       wherein Z is O, NR 3 , S, SO, or SO 2 ; R 1  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       R 2  is H, F, Cl, or CH 3 ; R 3  is H, C 1-6  alkyl, C 1-6  haloalkyl, C 0-3  alkylene-C 3-7 cycloalkyl, C(O)C 1-6 alkyl, or C(O)C 0-3  alkylene-C 3-7  cycloalkyl; and R 4  is H or C 1-3  alkyl. 
     
     
         94 . The compound of  claim 93 , wherein Z is O, NR 3 , S, or SO 2 ; and R 1  is selected from the group consisting of 
       
         
           
           
               
               
           
         
       
     
     
         95 . A compound having a structure selected from the group consisting of 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         96 . A compound selected from the group consisting of 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof. 
     
     
         97 . A pharmaceutical composition comprising the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier. 
     
     
         98 . A method of selectively inhibiting HDAC3 (in vitro or in vivo), the method comprising contacting a cell with an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         99 . A method of selectively inhibiting HDAC1 or HDAC2 (in vitro or in vivo), the method comprising contacting a cell with an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         100 . A method of selectively inhibiting HDAC1, HDAC2, and HDAC3 (in vitro or in vivo), the method comprising contacting a cell with an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         101 . A method of treating a disease or disorder mediated by HDAC1 or HDAC2 in a subject in need thereof, the method comprising administering an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 , to the subject. 
     
     
         102 . A method of treating a disease or disorder mediated by HDAC3 in a subject in need thereof, the method comprising administering an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 , to the subject. 
     
     
         103 . A method of treating a disease or disorder mediated by HDAC1, HDAC2, and HDAC3 in a subject in need thereof, the method comprising administering an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 , to the subject. 
     
     
         104 . A method of treating a neurological disorder such as Friedreich's ataxia, myotonic dystrophy, spinal muscular atrophy, fragile X syndrome, Huntington's disease, spinocerebellar ataxia, Kennedy's disease, amyotrophic lateral sclerosis, Niemann Pick, Pitt Hopkins, spinal and bulbar muscular atrophy, and Alzheimer's disease; a cancer; an inflammatory disease; a memory impairment condition, frontotemporal dementia, or a drug addiction in a subject in need thereof, the method comprising administering to the subject an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         105 . A method of treating Friedreich's ataxia in a subject in need thereof, the method comprising administering to the subject an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         106 . A method of treating an infection in a subject in need thereof, the method comprising administering to the subject an effective amount of the compound of any one of  claims 1  to  96 , or pharmaceutically acceptable salt thereof, or the composition of  claim 97 . 
     
     
         107 . The method of any one of  claims 98  to  106 , wherein the compound is a compound as claimed in  claim 95  or  96 , or a pharmaceutically acceptable salt thereof.

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