US2016229887A1PendingUtilityA1

Neuroactive steroids, compositions, and uses thereof

Assignee: SAGE THERAPEUTICS INCPriority: Aug 23, 2013Filed: Aug 22, 2014Published: Aug 11, 2016
Est. expiryAug 23, 2033(~7.1 yrs left)· nominal 20-yr term from priority
A61P 9/00A61P 25/18A61P 29/02A61P 25/24A61P 25/28A61P 25/30A61P 25/04A61P 27/16A61P 25/08A61P 25/36A61P 25/20A61P 25/00A61P 23/00C07J 21/00A61K 45/06A61K 31/58C07J 43/003C07J 13/007C07J 7/007C07J 21/008C07J 7/002C07J 7/0085C07J 1/0059
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Claims

Abstract

Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof; wherein -------, R 1 , R 2 , R 5 , A and L are as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Claims

exact text as granted — not AI-modified
1 . A compound of the Formula (I): 
       
         
           
           
               
               
           
         
       
       a pharmaceutically acceptable salt thereof, 
       wherein:
 A is an optionally substituted nitrogen-containing heteroaryl or heterocyclyl; 
 L is —C(R 3 )(R 3 )—, —O—, —S—, or —NR 3 —; 
 R 1  is hydrogen or C 1 -C 6  alkyl, C 1 -C 6  alkenyl, C 1 -C 6  alkynyl, carbocyclyl, or heterocyclyl; 
 R 2  is hydrogen, C 1 -C 6  alkyl (e.g., C 1 -C 6  haloalkyl), or C 1 -C 6  alkoxy; 
 each R 3  is independently hydrogen or C 1 -C 6  alkyl; 
 R 5  is absent or hydrogen; and 
    represents a single or double bond, wherein
 when one of   is a double bond, the other   is a single bond; and 
 when one of the   is a double bond, R 5  is absent. 
 
 
     
     
         2 - 13 . (canceled) 
     
     
         14 . The compound of claim  13 , wherein the compound is of the Formula (Ia-3): 
       
         
           
           
               
               
           
         
         wherein 
         R 4  is cyano, nitro, hydroxy, halo, C 1 -C 6  alkyl, C 1 -C 6  alkoxy, —C(O)R a , —C(O)N(R b )(R c ), —C(O)OR a , —N(R b )(R c ), —OC(O)N(R b )(R c ), —OC(O)OR a , —OC(O)R a , —S(O) 0-2 R a , —S(O) 0-2 OR a , or —S(O) 0-2 N(R b )(R c ); 
         each R a  is hydrogen or C 1 -C 6  alkyl; 
         each R b  and R c  is independently hydrogen, C 1 -C 6  alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, or 
         R b  and R c , together with the nitrogen atom to which they are bound to form a ring (e.g., a 3-7-membered ring, e.g., a 5-7-membered ring; a ring containing at least one heteroatom, e.g., a nitrogen, oxygen, or sulfur atom); and 
         n is 0, 1, 2, or 3. 
       
     
     
         15 . (canceled) 
     
     
         16 . The compound of  claim 14 , wherein n is 0. 
     
     
         17 - 22 . (canceled) 
     
     
         23 . The compound of  claim 14 , wherein R 1  is C 1 -C 6  alkyl. 
     
     
         24 - 25 . (canceled) 
     
     
         26 . The compound of  claim 14 , wherein the compound is selected from: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         27 . A compound of the Formula (Ib): 
       
         
           
           
               
               
           
         
       
       a pharmaceutically acceptable salt thereof, 
       wherein:
 A is an optionally substituted nitrogen-containing heteroaryl or heterocyclyl; 
 L is —C(R 3 )(R 3 )—, —O—, —S—, or —NR 3 —; 
 R 1  is hydrogen or C 1 -C 6  alkyl, C 1 -C 6  alkenyl, C 1 -C 6  alkynyl, carbocyclyl, or heterocyclyl; 
 R 2  is C 1 -C 6  alkyl (e.g., C 1 -C 6  haloalkyl) or C 1 -C 6  alkoxy; 
 each R 3  is independently hydrogen or C 1 -C 6  alkyl; 
 R 5  is absent or hydrogen; and 
    represents a single or double bond, wherein
 when one of   is a double bond, the other   is a single bond; and 
 when one of the   is a double bond, R 5  is absent. 
 
 
     
     
         28 - 29 . (canceled) 
     
     
         30 . The compound of  claim 27 , wherein the compound is of the Formula (Ib-3) 
       
         
           
           
               
               
           
         
       
       wherein
 R 4  is cyano, nitro, hydroxy, halo, C 1 -C 6  alkyl, C 1 -C 6  alkoxy, —C(O)R a , —C(O)N(R b )(R c ), —C(O)OR a , —N(R b )(R c ), —OC(O)N(R b )(R c ), —OC(O)OR a , —OC(O)R a , —S(O) 0-2 R a , —S(O) 0-2 OR a , or —S(O) 0-2 N(R b )(R c ); 
 each R a  is hydrogen or C 1 -C 6  alkyl; 
 each R b  and R c  is independently hydrogen, C 1 -C 6  alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, or 
 R b  and R c , together with the nitrogen atom to which they are bound to form a ring (e.g., a 3-7-membered ring, e.g., a 5-7-membered ring; a ring containing at least one heteroatom, e.g., a nitrogen, oxygen, or sulfur atom); and 
 n is 0, 1, 2, or 3. 
 
     
     
         31 . The compound of  claim 27 , wherein the compound is of the Formula (Ib-4) 
       
         
           
           
               
               
           
         
       
       wherein
 R 4  is cyano, nitro, hydroxy, halo, C 1 -C 6  alkyl, C 1 -C 6  alkoxy, —C(O)R a , —C(O)N(R b )(R c ), —C(O)OR a , —N(R b )(R c ), —OC(O)N(R b )(R c ), —OC(O)OR a , —OC(O)R a , —S(O) 0-2 R a , —S(O) 0-2 OR a , or —S(O) 0-2 N(R b )(R c ); 
 each R a  is hydrogen or C 1 -C 6  alkyl; 
 each R b  and R c  is independently hydrogen, C 1 -C 6  alkyl, carbocyclyl, heterocyclyl, aryl, heteroaryl, or 
 R b  and R c , together with the nitrogen atom to which they are bound to form a ring (e.g., a 3-7-membered ring, e.g., a 5-7-membered ring; a ring containing at least one heteroatom, e.g., a nitrogen, oxygen, or sulfur atom); and 
 n is 0, 1, 2, or 3. 
 
     
     
         32 . The compound of  claim 27 , wherein A is monocyclic. 
     
     
         33 . The compound of  claim 27 , wherein A is bicyclic. 
     
     
         34 - 36 . (canceled) 
     
     
         37 . The compound of  claim 27 , wherein A is a 5-membered or 6-membered heteroaryl or heterocyclyl comprising 1, 2, 3, or 4 nitrogen atoms. 
     
     
         38 . (canceled) 
     
     
         39 . The compound of  claim 37 , wherein A is morpholine or piperazine. 
     
     
         40 - 41 . (canceled) 
     
     
         42 . The compound of  claim 27 , wherein the heteroaryl is benzotriazole, azabenzotriazole, diazabenzotriazole, benzopyrazole, azabenzopyrazole, or diazabenzopyrazole. 
     
     
         43 - 45 . (canceled) 
     
     
         46 . The compound of  claim 37 , wherein A is pyrazole, triazole, or tetrazole. 
     
     
         47 . The compound of  claim 27 , wherein R 1  is C 1 -C 6  alkyl, C 1 -C 6  alkenyl, C 1 -C 6  alkynyl, carbocyclyl, or heterocyclyl. 
     
     
         48 . (canceled) 
     
     
         49 . The compound of  claim 47 , wherein R 1  is methyl, ethyl, or isopropyl. 
     
     
         50 . (canceled) 
     
     
         51 . The compound of  claim 27 , wherein R 2  is methyl. 
     
     
         52 . The compound of  claim 27 , wherein n is 0. 
     
     
         53 . The compound of  claim 27 , wherein n is 1 or 2, and R 4  is cyano, halo, C 1 -C 6  alkyl, C 1 -C 6  alkoxy, —C(O)R a , or —S(O) 0-2 R a . 
     
     
         54 - 59 . (canceled) 
     
     
         60 . The compound of  claim 53 , wherein R a  is C 1 -C 6  alkyl. 
     
     
         61 . The compound of  claim 60 , wherein R a  is methyl. 
     
     
         62 . The compound of  claim 53 , wherein R 4  is —S(O) 2 R a . 
     
     
         63 . The compound of  claim 62 , wherein R a  is methyl. 
     
     
         64 . The compound of  claim 27 , wherein R 1  is C 1 -C 6  alkyl and R 4  is —C(O)R a . 
     
     
         65 . (canceled) 
     
     
         66 . The compound of  claim 27 , wherein n is 0 or 1; R 1  is hydrogen or C 1 -C 6  alkyl; and R 2  is methyl. 
     
     
         67 . The compound of  claim 27 , wherein R 1  is methyl, ethyl, or isopropyl. 
     
     
         68 . (canceled) 
     
     
         69 . The compound of  claim 27 , wherein R 4  is C 1 -C 6  alkyl, —C(O)R a , or —S(O) 0-2 R a . 
     
     
         70 - 72 . (canceled) 
     
     
         73 . The compound of  claim 27 , wherein the compound is selected from: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         74 . A pharmaceutical composition comprising a compound of  claim 1  and a pharmaceutically acceptable excipient. 
     
     
         75 - 90 . (canceled)

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