US2023041584A1PendingUtilityA1

Novel prodrugs and conjugates of dimethyltryptamine

72
Assignee: ATAI Life Sciences AGPriority: Jun 9, 2021Filed: Jun 9, 2022Published: Feb 9, 2023
Est. expiryJun 9, 2041(~14.9 yrs left)· nominal 20-yr term from priority
C07D 209/12C07D 209/08C07D 209/14C07D 209/16C07D 401/06A61P 25/24C07F 9/5728C07D 403/06A61K 31/4045A61K 31/675A61P 25/00A61P 25/22
72
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Claims

Abstract

Provided herein are compounds of Formula (I), or pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R8, R9, X, Y, Z, W, and m are defined herein. Also provided herein are pharmaceutical compositions comprising a compound of Formula (I) or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable excipient.

Claims

exact text as granted — not AI-modified
What is claimed: 
     
         1 . A compound of Formula (I): 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof; 
         wherein, 
         R 1  and R 2  are independently hydrogen, alkyl, cycloalkyl, or R 1  and R 2  together with the atoms to which they are attached form a heterocycle; 
         X, Y and Z are independently hydrogen, halogen, —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl; 
         R′, R 3 , R 4 , R 5  and R 6  are independently absent, hydrogen, alkyl, —(C═O)-alkyl, —(C═O)CHR 7 , —(C═O)(CH 2 ) n (NH 2 ), —(C)CH(NH 2 )(CH 3 ), —(C═O)CH(NH 2 ), (CH 2 ) n (NH)(C═NH), or —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2 ; 
         R 7  and R 8  are independently hydrogen or alkyl; 
         R 9  is hydrogen, alkyl, or halogen; 
         L and M are independently alkyl or aryl; 
         W is oxygen, sulfur or NR 6 ; 
         n is an integer from 2-7; and 
         m is an integer from 1-4, 
         wherein at least one of R′, R 3 , R 4 , and R 5  and R 6  is —(C═O)-alkyl, —(C═O)CHR 7 (NH 2 ), —(C═O)(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  or 
       
       
         
           
           
               
               
           
         
       
     
     
         2 . The compound of  claim 1 , wherein R 1  and R 2  are independently C 1 -C 6  alkyl. 
     
     
         3 . The compound of  claim 1  or  2 , wherein R 1  and R 2  are independently methyl, ethyl, propyl, and isopropyl. 
     
     
         4 . The compound of any one of  claims 1 - 3 , wherein R 1  and R 2  are methyl. 
     
     
         5 . The compound of any one of  claims 1 - 4 , wherein X, Y and Z are H. 
     
     
         6 . The compound of any one of  claims 1 - 5 , wherein R 8  is hydrogen. 
     
     
         7 . The compound of any one of  claims 1 - 6 , wherein R 8  is alkyl. 
     
     
         8 . The compound of any one of  claims 1 - 7 , wherein R 8  is C 1 -C 6  alkyl. 
     
     
         9 . The compound of any one of  claims 1 - 8 , wherein R 9  is a halogen. 
     
     
         10 . The compound of any one of  claims 1 - 9 , wherein R 9  is Br. 
     
     
         11 . The compound of any one of  claims 1 - 8 , wherein R 9  is alkyl. 
     
     
         12 . The compound of any one of  claims 1 - 8  or  11 , wherein R 9  is C 1 -C 6  alkyl. 
     
     
         13 . The compound of any one of  claims 1 - 8 , wherein R 7  and R 8  are independently hydrogen or C 1 -C 8  alkyl and R 9  is hydrogen, halogen, or C 1 -C 8  alkyl. 
     
     
         14 . The compound of  claim 13 , wherein the halogen is bromine. 
     
     
         15 . The compound of  claim 13 , wherein said C 1 -C 8  alkyl is substituted with at least one of hydroxyl, thiol and halogen. 
     
     
         16 . The compound of any one of  claims 1 - 15 , wherein m is 1. 
     
     
         17 . The compound of any one of  claims 1 - 15 , wherein m is 2. 
     
     
         18 . The compound of  claim 1 , wherein X, Y and Z are hydrogen; W is NR 6 ; and R 6  is —(C═O)— alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         19 . The compound of  claim 1 , wherein two of X, Y and Z are hydrogen. 
     
     
         20 . The compound of  claim 19 , wherein the non-hydrogen X, Y or Z is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         21 . The compound of  claim 1 , wherein X and Y are hydrogen; Z is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         22 . The compound of  claim 1 , wherein X and Z are hydrogen; Y is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         23 . The compound of  claim 1 , wherein Y and Z are hydrogen; X is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         24 . The compound of any one of  claims 1 - 23 , wherein R 5  is —(C═O)-alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         25 . The compound of any one of  claims 1 - 24 , wherein R 4  is —(C═O)-alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         26 . The compound of any one of  claims 1 - 25 , wherein R 3  is —(C═O)-alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         27 . The compound of any one of  claims 1 - 26 , wherein R 6  is —(C═O)-alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         28 . The compound of any one of  claims 1 - 23 , wherein at least one of R′, R 3 , R 4 , R 5  and R 6  is —(C═O)(CH 2 ) n (NH 2 ) and n is 3. 
     
     
         29 . The compound of any one of  claims 1 - 23 , wherein at least one of R′, R 3 , R 4 , R 5  and R 6  is —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), and n is 4. 
     
     
         30 . The compound of any one of  claims 1 - 23 , wherein at least one of R′, R 3 , R 4 , R 5  and R 6  is —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  and n is 3. 
     
     
         31 . The compound of any one of  claims 1 - 23 , wherein at least one of R′, R 3 , R 4 , R 5  and R 6  is —(C═O)CH(NH)(CH 2 ) n (C═NH)—NH 2  and n is 2. 
     
     
         32 . The compound of any one of  claims 1 - 31 , wherein L is methyl or isopropyl and M is alkyl or aryl. 
     
     
         33 . The compound of any one of  claims 1 - 32 , wherein W is NR 6  and R 6  is hydrogen or alkyl. 
     
     
         34 . A compound of  claim 1 , which is a salt of the Formula (IV): 
       
         
           
           
               
               
           
         
         wherein R 10  is —(C═O)-alkyl, —(C═O)CHR 7 (NH 2 ), —(C═O)(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  or 
       
       
         
           
           
               
               
           
         
         A is a pharmaceutically acceptable anion. 
       
     
     
         35 . The compound of any one of  claims 1 - 34 , wherein the compound is a compound shown in Table 1, Table 2, Table 3, or Table 4. 
     
     
         36 . A compound of Formula (III): 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof; 
         wherein, 
         R 1  and R 2  are independently hydrogen, alkyl, cycloalkyl, or R 1  and R 2  together with the atoms to which they are attached form a heterocycle; 
         X, Y and Z are independently hydrogen, halogen, —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl, 
         R′, R 3 , R 4 , and R 5  are independently absent, hydrogen, alkyl, —(C═O)-alkyl, —(C═O)CHR 7 (NH 2 ), —(C═O)(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 ) (CH 2 ) n (NH)(C═NH)—NH 2 , —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  or 
       
       
         
           
           
               
               
           
         
         R 7  and R 8  are independently hydrogen or alkyl; 
         R 9  is hydrogen, alkyl or halogen; 
         L and M are independently alkyl or aryl; 
         n is an integer from 2-7; and 
         m is an integer from 1-4, 
         wherein at least one of R′, R 3 , R 4 , and R 5  is —(C═O)-alkyl, —(C═O)CHR 7 (NH 2 ), —(C═O)(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  or 
       
       
         
           
           
               
               
           
         
       
     
     
         37 . The compound of  claim 36 , wherein R 1  and R 2  are independently C 1 -C 6  alkyl. 
     
     
         38 . The compound of  claim 36  or  37 , wherein R 1  and R 2  are independently methyl, ethyl, propyl, and isopropyl. 
     
     
         39 . The compound of any one of  claims 36 - 38 , wherein R 1  and R 2  are methyl. 
     
     
         40 . The compound of any one of  claims 36 - 39 , wherein X, Y and Z are H. 
     
     
         41 . The compound of any one of  claims 36 - 40 , wherein R 8  is hydrogen. 
     
     
         42 . The compound of any one of  claims 36 - 41 , wherein R 8  is alkyl. 
     
     
         43 . The compound of any one of  claims 36 - 42 , wherein R 8  is C 1 -C 6  alkyl. 
     
     
         44 . The compound of any one of  claims 36 - 43 , wherein R 9  is hydrogen. 
     
     
         45 . The compound of any one of  claims 36 - 43 , wherein R 9  is alkyl. 
     
     
         46 . The compound of  claim 45 , R 9  is C 1 -C 6  alkyl. 
     
     
         47 . The compound of any one of  claims 36 - 46 , wherein R 7  and R 8  are independently hydrogen or C 1 -C 8  alkyl and R 9  is hydrogen, halogen or C 1 -C 8  alkyl. 
     
     
         48 . The compound of  claim 47 , wherein the C 1 -C 8  alkyl is substituted with at 1-3 groups including hydroxyl, thiol or halogen. 
     
     
         49 . The compound of any one of  claims 36 - 48 , wherein m is 1. 
     
     
         50 . The compound of any one of  claims 36 - 48 , wherein m is 2. 
     
     
         51 . The compound of any one of  claims 1 - 39  or  41 - 50 , wherein two of X, Y and Z are hydrogen. 
     
     
         52 . The compound of  claim 51 , wherein the non-hydrogen X, Y or Z is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         53 . The compound of  claim 36 , wherein X and Y are hydrogen; Z is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         54 . The compound of  claim 36 , wherein X and Z are hydrogen; Y is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         55 . The compound of  claim 36 , wherein Y and Z are hydrogen; X is —O—, —S—, —NR A —, or —(P═O)—OR A (—OR′), wherein R A  is hydrogen or alkyl. 
     
     
         56 . The compound of any one of  claims 36 - 55 , wherein R 4  is —(C═O)-alkyl, or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         57 . The compound of any one of  claims 36 - 56 , wherein R 5  is —(C═O)-alkyl or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         58 . The compound of any one of  claims 36 - 57 , wherein R 3  is —(C═O)-alkyl, or —(C═O)(CH 2 ) n (NH 2 ). 
     
     
         59 . The compound of any one of  claims 36 - 55 , wherein at least one of R′, R 3 , R 4 , and R 5  is —(C═O)(CH 2 ) n (NH 2 ) and n is 3. 
     
     
         60 . The compound of any one of  claims 36 - 55 , wherein at least one of R′, R 3 , R 4 , and R 5  is —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ) and n is 4. 
     
     
         61 . The compound of any one of  claims 36 - 55 , wherein at least one of R′, R 3 , R 4 , and R 5  is —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  and n is 3. 
     
     
         62 . The compound of any one of  claims 36 - 55 , wherein at least one of R′, R 3 , R 4 , and R 5  is —(C═O)CH(NH 2 )(CH 2 ) n (NH)(C═NH)—NH 2  and n is 2. 
     
     
         63 . The compound of any one of  claims 36 - 55 , wherein at least one of R′, R 3 , R 4 , and R 5  is 
       
         
           
           
               
               
           
         
       
     
     
         64 . The compound of any one of  claims 36 - 63 , in salt form, of the Formula (V): 
       
         
           
           
               
               
           
         
         wherein R 10  is —(C═O)-alkyl, —(C═O)CHR 7 (NH 2 ), —(C═O)(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH 2 ), —(C═O)CH(NH 2 )(CH 2 ) n (NH)(C═NH)—NH 2 , —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2  or 
       
       
         
           
           
               
               
           
         
       
       and
 wherein A is a pharmaceutically acceptable anion. 
 
     
     
         65 . The compound  claim 64 , wherein L is methyl or isopropyl and M is alkyl or aryl. 
     
     
         66 . A pharmaceutical composition comprising the compound of any one of  claims 1 - 65  and a pharmaceutically acceptable excipient. 
     
     
         67 . A compound as shown in Table 2, wherein X and Y are hydrogen and R 3  and R 4  are absent. 
     
     
         68 . A compound as shown in Table 2, wherein X is hydrogen, R 3  is absent, Y is O, and R 4  is hydrogen. 
     
     
         69 . A compound as shown in Table 2, wherein X is hydrogen, R 3  is absent, Y is O, and R 4  is Me. 
     
     
         70 . A compound as shown in Table 2, wherein X is hydrogen, R 3  is absent, Y is O, and R 4  is Ac. 
     
     
         71 . A compound as shown in Table 2, wherein X is O, R 3  is hydrogen, Y is hydrogen, and R 4  is absent. 
     
     
         72 . A compound as shown in Table 2, wherein X is O, R 3  is Me, Y is hydrogen, and R 4  is absent. 
     
     
         73 . A compound as shown in Table 2, wherein X is O, R 3  is Ac, Y is hydrogen, and R 4  is absent. 
     
     
         74 . A compound of the formula: 
       
         
           
           
               
               
           
         
       
     
     
         75 . A compound of the formula: 
       
         
           
           
               
               
           
         
       
     
     
         76 . A compound of the formula 
       
         
           
           
               
               
           
         
       
     
     
         77 . A compound of the formula: 
       
         
           
           
               
               
           
         
       
     
     
         78 . A compound of Formula (VI): 
       
         
           
           
               
               
           
         
         R 1  and R 2  are independently hydrogen, alkyl, cycloalkyl, or R 1  and R 2  together with the atoms to which they are attached form a heterocycle; 
         R 3 , R 4 , R 5  and R 6  are independently absent, hydrogen, alkyl, —(C═O)-alkyl, —(C═O)CHR 7 , —(C═O)(CH 2 ) n (NH 2 ), —(C)CH(NH 2 )(CH 3 ), —(C═O)CH(NH 2 ), (CH 2 ) n (NH)(C═NH), or —(C═O)(CH 2 ) n (NH)(C═NH)—NH 2 ; 
         X is carbon or nitrogen; 
         W is carbon, oxygen, or nitrogen; and 
         Z is hydrogen, deuterium, or oxygen. 
       
     
     
         79 . The compound of  claim 78 , having the following chemical formula: 
       
         
           
           
               
               
           
         
       
     
     
         80 . A compound selected from the formulae: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         81 . A phosphonooxymethyl prodrug according to  claim 1  of the Formula (VIII): 
       
         
           
           
               
               
           
         
       
       wherein
 Ru and R 12  are pharmaceutically acceptable salt and are independently H, D, Na + , K + , NH 4   + ; or 
 R 1  and R 12  are independently a pharmaceutically acceptable salt, hydrogen, deuterium, alkyl, substituted alkyl, cycloalkyl, or R 11  and R 12  together with the atoms to which they are attached form an alkyl, cycloalkyl, heterocycles and are ether, hemiaminal ketal and amino acid, wherein the pharmaceutically acceptable salt is Na + , K + , NH 4   + . 
 
     
     
         82 . A pharmaceutical composition comprising a compound of any one of  claims 1 - 65  or  67 - 81  and a pharmaceutically acceptable excipient. 
     
     
         83 . An oral dosage form comprising the pharmaceutical composition of  claim 66  or  82 . 
     
     
         84 . A method of treating post-traumatic stress disorder (PTSD) in a subject in need thereof, comprising administering a therapeutically effective amount of a compound of any one of  claims 1 - 65  or  67 - 81  or a pharmaceutical composition of  claims 66 ,  82  or  83  to the subject. 
     
     
         85 . A method of treating depression in a subject in need thereof, comprising administering a therapeutically effective amount of a compound of any one of  claims 1 - 65  or  67 - 81  or a pharmaceutical composition of  claims 66 ,  82  or  83  to the subject. 
     
     
         86 . The method of  claim 85 , wherein the depression is major depressive disorder (MDD) or treatment-resistant depression (TRD). 
     
     
         87 . A method of treating an anxiety disorder in a subject in need thereof, comprising administering a therapeutically effective amount of a compound of any one of  claims 1 - 65  or  67 - 81  or a pharmaceutical composition of  claims 66 ,  82  or  83  to the subject.

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