US2020331828A1PendingUtilityA1

Novel compounds which activate estrogen receptors and compositions and methods of using the same

Assignee: UNIV ILLINOISPriority: Jan 6, 2016Filed: Jul 2, 2020Published: Oct 22, 2020
Est. expiryJan 6, 2036(~9.4 yrs left)· nominal 20-yr term from priority
A61P 35/00C07C 39/17C07C 49/733C07C 39/42C07C 49/755C07C 2602/08A61P 25/00C07C 39/367C07C 39/06C07C 255/47C07C 235/34C07C 39/12C07C 2602/10C07C 39/23C07C 57/38C07C 2603/74C07C 43/196C07C 39/14C07C 2601/14
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Claims

Abstract

Provided are compounds of formulae provided herein. The compounds may include pathway-preferential estrogens (PaPEs) derivatives with tissue-selective activities. Also provided are pharmaceutical compositions comprising the compounds, as well as methods of treating a disease or condition including administering the compounds. The disease or condition may include postmenopausal symptoms, cardiovascular disease, stroke, vascular disease, bone disease, metabolic disease, arthritis, osteoporosis, obesity, vasomotor/hot flush, cognitive decline, cancer including breast cancer.

Claims

exact text as granted — not AI-modified
The invention claimed is: 
     
         1 . A compound of formula (i): 
       
         
           
           
               
               
           
         
         and stereoisomers and pharmaceutically acceptable salts thereof; 
         wherein 
         n is an integer from 0 to 4; 
         m is an integer from 0 to 4; 
         X is H, hydroxy, or C 1-4  alkoxy; 
         R 1  and R 2  are independently H, hydroxy, C 1-4  alkyl, C 1-4  alkoxy, amino, —S—C 1-4  alkyl, or halo; 
         R 3  is H, hydroxy, oxo, cyano, halo, C 1-4  alkyl, or C 1-4  alkoxy; 
         each R 4  is independently hydrogen, hydroxy, oxo, halo, C 1-4  alkyl, or C 1-4  alkoxy; 
         R 5  is H, C 1-4  alkynyl, or is absent if a double bond is present; and 
         - - - is an optional double bond. 
       
     
     
         2 . A compound according  claim 1 , wherein C 1-4  alkyl or C 1-4  alkoxy is substituted with one or more of halo, cyano, amino, hydroxy, and C 1-4  alkoxy, 
     
     
         3 . A compound according to any one of  claim 1  or  2 , wherein R 3  is hydroxyl. 
     
     
         4 . A compound according to any one of  claims 1 - 3 , wherein at least one of R 1  and R 2  is C 1-4  alkyl. 
     
     
         5 . A compound according to  claim 4 , wherein both R 1  and R 2  are C 1-4  alkyl. 
     
     
         6 . A compound according to  claim 4 , wherein at least one of R 1  and R 2  is methyl. 
     
     
         7 . A compound according to  claim 6 , wherein both R 1  and R 2  are methyl. 
     
     
         8 . A compound according to any one of  claims 1 - 7 , wherein C 1-4  alkyl is substituted. 
     
     
         9 . A compound according to  claim 8 , wherein the substituent is hydroxy. 
     
     
         10 . A compound according to any one of  claims 1 - 9 , wherein R 1  and R 2  are independently selected from H, methyl, ethyl, chloro, —CH 2 OH, and —CH 2 OMe. 
     
     
         11 . A compound according to any one of  claims 1 - 10 , wherein R 3  is selected from H, OH, oxo, chloro, cyano, or methoxy. 
     
     
         12 . A compound according to any one of  claims 1 - 11 , wherein R 4  is selected from fluoro, chloro, bromo, methoxy, hydroxy, or oxo. 
     
     
         13 . A compound according to any one of  claims 1 - 12 , wherein R 5  is H. 
     
     
         14 . A compound according to any one of  claims 1 - 12 , wherein R 5  is —CCH. 
     
     
         15 . A compound according any one of  claims 1 - 11 ,  13  or  14 , wherein m is 0. 
     
     
         16 . A compound according to any one of  claims 1 - 15 , wherein n is 1. 
     
     
         17 . A compound according to any one of  claims 1 - 15 , wherein n is 2. 
     
     
         18 . A compound according to any one of  claims 1 ,  3 - 7 ,  10 ,  11 ,  13 ,  15 - 17  wherein the compound is selected from the group consisting of: 
       
         
           
           
               
               
           
         
       
     
     
         19 . A compound according to  claim 1  of formula (ii): 
       
         
           
           
               
               
           
         
         and stereoisomers and salts thereof 
         wherein 
         R is H or methyl. 
       
     
     
         20 . A compound according to formula (iii): 
       
         
           
           
               
               
           
         
         and stereoisomers and salts thereof; 
         wherein 
         m is an integer from 0 to 3; 
         R 1  and R 2  are independently H, hydroxy, C 1-4  alkyl, C 1-4  alkoxy, amino, —S—C 1-4  alkyl, or halo; 
         R 5  is H, hydroxy, or C 1-4  alkyl; and 
         each R 6  is independently H, hydroxy, halo, or C 1-4  alkyl. 
       
     
     
         21 . A compound according to  claim 20 , wherein C 1-4  alkyl is substituted with one or more of halo, cyano, amino, hydroxy, and C 1-4  alkoxy. 
     
     
         22 . A compound according to any one of  claim 20  or  21 , wherein at least one of R 1  and R 2  is C 1-4  alkyl. 
     
     
         23 . A compound according to  claim 22 , wherein both R 1  and R 2  are C 1-4  alkyl. 
     
     
         24 . A compound according to  claim 22 , wherein at least one of R 1  and R 2  is methyl. 
     
     
         25 . A compound according to  claim 22 , wherein both R 1  and R 2  are methyl. 
     
     
         26 . A compound according to any one of  claims 20 - 25 , wherein C 1-4  alkyl is substituted. 
     
     
         27 . A compound according to  claim 26 , wherein the substituent is hydroxy. 
     
     
         28 . A compound according to any one of  claims 20 - 27 , wherein R 5  is hydroxy. 
     
     
         29 . A compound according to any one of  claims 20 - 27 , wherein R 5  is C 1-4  alkyl. 
     
     
         30 . A compound according to  claim 29 , wherein R 5  is substituted C 1-4  alkyl. 
     
     
         31 . A compound according to  claim 30 , wherein R 5  is —CH(OH)(CH 3 ). 
     
     
         32 . A compound according to any one of  claims 20 - 31 , wherein R 6  is C 1-4  alkyl. 
     
     
         33 . A compound according to  claim 32 , wherein R 6  is substituted C 1-4  alkyl. 
     
     
         34 . A compound according to  claim 33 , wherein R 6  is —CH 2 OH. 
     
     
         35 . A compound according to any one of  claims 18 - 32 , wherein m is 1. 
     
     
         36 . A compound according to any one of  claims 20 - 35  selected from the group consisting of: 
       
         
           
           
               
               
           
         
       
     
     
         37 . A compound having the formula (iv): 
       
         
           
           
               
               
           
         
         wherein R 1  and R 2  is selected from H, C 1-4  alkyl group, haloalkyl, hydroxyalkyl, alkyloxyalkyl, cycloalkyloxyalkyl, alkylthio, alkylthioalkyl, cycloalkylthioalkyl, R′R″N-alkyl where R′ and R″ are independently alkyl, alkylcarbonyl, or cyclic alkyl, and the parenthesis represents the presence or absence of hydroxyl, and 
         wherein R 3  and R 4  are independently selected from H, hydroxy, C 1-4  alkyl, hydroxy-C 1-4 alkyl, cyano, cyanoalkyl, nitro, nitroalkyl, —C(O)-aryl, —C(O)H, alkyl aldehyde, carboxyl, and carboxyalkyl, or wherein 
         R 3  and R 4  form a ring of from 4 to 8 member atoms, wherein the ring is substituted with cyano or hydroxy. 
       
     
     
         38 . A compound according to  claim 37 , of formula (v): 
       
         
           
           
               
               
           
         
         wherein R 5  and R 6  are independently selected from hydroxyl, cyano, hydroxylalkyl, cyanoalkyl, halogenated hydroxylalkyl, and halogenated cyanoalkyl. 
       
     
     
         39 . A compound according to formula (vi): 
       
         
           
           
               
               
           
         
         and stereoisomers and pharmaceutically acceptable salts thereof; 
         wherein 
         n is an integer from 0 to 4; 
         m is an integer from 0 to 4; 
         X is H, hydroxy, or C 1-4  alkoxy; 
         R 1  and R 2  are independently H, hydroxy, C 1-4  alkyl, C 1-4  alkoxy, amino, —S—C 1-4  alkyl, or halo; 
         R 3  is H, hydroxy, oxo, cyano, halo, C 1-4  alkyl, or C 1-4  alkoxy; 
         each R 4  is independently hydrogen, hydroxy, oxo, halo, C 1-4  alkyl, or C 1-4  alkoxy; 
         R 5  is H, alkynyl, or is absent if a double bond is present; 
         R 6  and R 7  are independently selected from C 1-4  alkyl and H; and 
         - - - is an optional double bond. 
       
     
     
         40 . A compound according  claim 39 , wherein C 1-4  alkyl or C 1-4  alkoxy is substituted with one or more of halo, cyano, amino, hydroxy, and C 1-4  alkoxy, 
     
     
         41 . A compound according to any one of  claim 39  or  40 , wherein R 3  is hydroxyl. 
     
     
         42 . A compound according to any one of  claims 39 - 41 , wherein at least one of R 1  and R 2  is C 1-4  alkyl. 
     
     
         43 . A compound according to  claim 42 , wherein both R 1  and R 2  are C 1-4  alkyl. 
     
     
         44 . A compound according to  claim 42 , wherein at least one of R 1  and R 2  is methyl. 
     
     
         45 . A compound according to  claim 44 , wherein both R 1  and R 2  are methyl. 
     
     
         46 . A compound according to any one of  claims 39 - 45 , wherein C 1-4  alkyl is substituted. 
     
     
         47 . A compound according to  claim 46 , wherein the substituent is hydroxy. 
     
     
         48 . A compound according to any one of  claims 39 - 47 , wherein R 1  and R 2  are independently selected from H, methyl, ethyl, chloro, —CH 2 OH, and —CH 2 OMe. 
     
     
         49 . A compound according to any one of  claims 39 - 48 , wherein R 3  is selected from H, OH, oxo, chloro, cyano, or methoxy. 
     
     
         50 . A compound according to any one of  claims 39 - 49 , wherein R 4  is selected from fluoro, chloro, bromo, methoxy, hydroxy, or oxo. 
     
     
         51 . A compound according to any one of  claims 39 - 50 , wherein R 5  is H. 
     
     
         52 . A compound according to any one of  claims 39 - 50 , wherein R 5  is —CCH. 
     
     
         53 . A compound according any one of  claims 39 - 49 ,  51 , or  52 , wherein m is 0. 
     
     
         54 . A compound according to any one of  claims 39 - 53 , wherein n is 1. 
     
     
         55 . A compound according to any one of  claims 39 - 54 , wherein n is 2. 
     
     
         56 . A compound according to any one of  claims 39 - 55 , wherein R 6  is methyl. 
     
     
         57 . A compound according to any one of  claims 39 - 56 , wherein R 7  is H. 
     
     
         58 . A compound according to formula (vii): 
       
         
           
           
               
               
           
         
         and stereoisomers and salts thereof; 
         wherein 
         R 7  and R 8  are independently H, C 1-5  alkyl, amino, hydroxyl, cyano, amido, cyclic C 3-8  alkyl, or heterocyclyl. 
       
     
     
         59 . A compound according to  claim 58 , wherein R 7  and R 8  are selected from C 1-4  alkylaminocarboxy-C 1-4  alkyl, C 1-4  alkylamino-C 1-4  alkyl, C 1-4  alkylamino-C 1-4  alkyl-amino-carboxy-C 1-4  alkyl, C 1-4  alkyloxy-C 1-4  alkylamino-carboxy-C 1-4  alkyl, C 1-4  alkylthio-C 1-4  alkylamino-carboxy-C 1-4  alkyl, or C 1-4  alkylthio-C 1-4  alkyl. 
     
     
         60 . A compound according to  claim 58  or  59 , wherein R 7  is methyl. 
     
     
         61 . A compound according to any one of  claims 58 - 60 , wherein R 8  is substituted C 1-5  alkyl. 
     
     
         62 . A compound according to  claim 61 , wherein R 8  is substituted with —C(O)—R, —C(O)NR N1 R N2 , —C(O)OR, —NR N1 C(O)R, or —OC(O)R, wherein R N1 , R N2  and R are independently selected from H or C 1-4  alkyl. 
     
     
         63 . A compound according to  claim 62 , wherein at least one of R N1 , R N2 , or R is —(CH 2 ) n —R 10 , wherein n is an integer of from 2 to 5, and wherein R 10  is —C(O)—R, —C(O)NR N1 R N2 , —C(O)OR, —NR N1 C(O)R, or —OC(O)R, wherein R N1 , R N2  and R are independently selected from H, aryl, cycloalkyl, or C 1-4  alkyl optionally substituted with OH or amino. 
     
     
         64 . A compound according to any one of  claims 58 - 63 , wherein the compound is 
       
         
           
           
               
               
           
         
       
     
     
         65 . A compound selected from the group consisting of 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         and stereoisomers and pharmaceutically acceptable salts thereof. 
       
     
     
         66 . A compound according to  claim 65 , selected from the group consisting of 
       
         
           
           
               
               
           
         
       
       and pharmaceutically acceptable salts thereof. 
     
     
         67 . A pharmaceutical composition comprising a compound of any one of  claims 1 - 66  and a pharmaceutically acceptable excipient. 
     
     
         68 . A method of treating a disease or condition in a subject, the method comprising administering a therapeutically effective amount of a compound or composition according to any one of  claims 1 - 67 . 
     
     
         69 . The method of  claim 68  wherein the disease or condition is affected by the extranuclear-initiated pathway of the estrogen receptor. 
     
     
         70 . The method of any one of  claim 68  or  69 , wherein the disease or condition is selected from postmenopausal symptoms, cardiovascular disease, stroke, vascular disease, bone disease, metabolic disease, arthritis, osteoporosis, obesity, vasomotor/hot flush, cognitive decline, and cancer. 
     
     
         71 . The method of any one of  claims 68 - 70 , wherein the disease is stroke. 
     
     
         72 . The method of any one of  claims 68 - 70 , wherein the disease is metabolic disease. 
     
     
         73 . The method of any one of  claims 68 - 70 , wherein the cancer is breast cancer. 
     
     
         74 . The method of  claim 73 , wherein the disease is obesity-related breast cancer. 
     
     
         75 . The method of  claim 73 , wherein the disease is estrogen-responsive breast cancer. 
     
     
         76 . The method of any one of  claims 68 - 70 , wherein the disease is vascular disease. 
     
     
         77 . The method of any one of  claims 68 - 70 , wherein the disease is osteoporosis. 
     
     
         78 . The method of any one of  claims 68 - 77 , wherein the subject is human. 
     
     
         79 . A kit comprising a compound or composition of any one of  claims 1 - 67  and instructions for use.

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