US2020199136A1PendingUtilityA1

Pikfyve kinase inhibitors

47
Assignee: ACURASTEM INCPriority: Aug 28, 2017Filed: Aug 28, 2018Published: Jun 25, 2020
Est. expiryAug 28, 2037(~11.1 yrs left)· nominal 20-yr term from priority
C07D 405/14C07D 413/14C07D 491/048C07D 417/14C07D 407/14C07D 403/04
47
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Claims

Abstract

The present invention relates to compounds of formula (I) (shown below) useful as inhibitors of phosphatidylinositol-3-phosphate 5-kinase (PIKfyve) as well as their use for treating diseases and disorders associated with PIKfyve.

Claims

exact text as granted — not AI-modified
1 . A compound of the formula (I) 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein
 R 1  is hydroxy, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted alkoxy, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, or substituted or unsubstituted heterocyclyl; 
 each occurrence of R 2  is independently substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, or substituted or unsubstituted heterocyclyl; 
 R 3  is a nitrogen- or oxygen-containing moiety; 
 Ring A is (i) a 5 or 6-membered heteroaryl or 5-6 or 6-5 membered bicyclic heteroaryl, each having at least one nitrogen or oxygen ring atom, or (ii) phenyl; 
 L 1  is absent, C 1 -C 2  alkylene, —NR c —, —O—, —S—, —C(O)—, —NHC(O)—, or —C(O)NH—; 
 L 2  is —O—(CR a R b ) m —, —(CR a R b ) m —, —NR c —(CR a R b ) m —, or —S—(CR a R b ) m —; 
 X 1  is CH, N, or CR c ; 
 each occurrence of R a  and R b  are independently hydrogen, hydroxy, hydroxy(C 1-4 )alkyl, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted alkoxy, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, or substituted or unsubstituted heterocyclyl, halogen, nitro, —OR d , —SR d , —NR d R e , —C(O)R d , —C(S)R d , —OC(O)R d —SC(O)R d , OC(S)R d , SC(S)R d , —NR c C(O)R d , —NR c C(S)R d , —SO 2 R c , —S(O)R c , —NR c SO 2 R d , —OS(O) 2 R d , —OP(O)R d R e , or —P(O)R d R e ; 
 R c  is a hydrogen or C 1-6  alkyl; 
 each occurrence of R d  and R e  are independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted alkoxy, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, or substituted or unsubstituted heterocyclyl; 
 m is 1-4; and 
 p is 1 or 2. 
 
     
     
         2 . The compound of  claim 1 , wherein R 1  is heterocyclyl or heteroaryl. 
     
     
         3 . The compound of  claim 1  or  2 , wherein R 1  is be selected from (the squiggly lines indicate the point of attached to the rest of the molecule) 
       
         
           
           
               
               
           
         
       
     
     
         4 . The compound of  claim 1 , wherein R 1  is hydroxy. 
     
     
         5 . The compound of any of the preceding claims, wherein each occurrence of R 2  is independently substituted or unsubstituted phenyl. 
     
     
         6 . The compound of  claim 5 , wherein R 2  is phenyl, a halogen-substituted phenyl, an alkyl-substituted phenyl, a halogenated alkyl-substituted phenyl, or an alkoxy-substituted phenyl. 
     
     
         7 . The compound of  claim 5 , wherein R 2  is selected from phenyl, 3-methoxyphenyl, 3-methylphenyl, 3-trifluoromethylphenyl, and 3-chlorophenyl. 
     
     
         8 . The compound of any of the preceding claims, wherein R 3  is a substituted or unsubstituted, saturated or unsaturated nitrogen- or oxygen-containing heterocyclyl. 
     
     
         9 . The compound of any of the preceding claims, wherein R 3  is selected from 
       
         
           
           
               
               
           
         
       
     
     
         10 . The compound of any of the preceding claims, wherein R 3  is selected from 
       
         
           
           
               
               
           
         
       
     
     
         11 . The compound of any of the preceding claims, wherein ring A is a 5-membered heteroaryl having at least one nitrogen ring atom. 
     
     
         12 . The compound of any of the preceding claims, wherein ring A is selected from 
       
         
           
           
               
               
           
         
       
     
     
         13 . The compound of any of the preceding claims, wherein ring A can be selected from 
       
         
           
           
               
               
           
         
       
     
     
         14 . The compound of any of the preceding claims, wherein L 1  is absent. 
     
     
         15 . The compound of any of the preceding claims, wherein L 1  is —NH—, —N(CH 3 )—, —O—, or —CH 2 —. 
     
     
         16 . The compound of any of the preceding claims, wherein L 2  is —O—(CR a R b ) m —. 
     
     
         17 . The compound of any of the preceding claims, wherein L 2  is —OCH 2 CH 2 —, —OCH 2 —, or —OCH 2 CH 2 CH(OH)CH 2 —. 
     
     
         18 . The compound of any of the preceding claims, wherein L 2  is —(CR a R b ) m —. 
     
     
         19 . The compound of any of the preceding claims, wherein -L 2 -R 1  is —OCH 2 CH 2 CH(OH)CH 2 OH. 
     
     
         20 . The compound of any of the preceding claims, wherein X 1  is CH. 
     
     
         21 . The compound of any of the preceding claims, wherein each occurrence of R a  and R b  are independently hydrogen, hydroxy, or hydroxy(C 1-4 )alkyl. 
     
     
         22 . The compound of any of the preceding claims, wherein each occurrence of R a  and R b  are independently hydrogen or hydroxy. 
     
     
         23 . The compound of any of the preceding claims, wherein m is 1. 
     
     
         24 . The compound of any of the preceding claims, wherein m is 2. 
     
     
         25 . The compound of any of the preceding claims, wherein p is 1. 
     
     
         26 . The compound of any of the preceding claims, wherein p is 2. 
     
     
         27 . The compound of any of the preceding claims, wherein the moiety 
       
         
           
           
               
               
           
         
       
       is selected from 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         28 . A compound of the formula (II) 
       
         
           
           
               
               
           
         
       
       or a pharmaceutically acceptable salt thereof, wherein
 R 1  is hydroxy, substituted or unsubstituted heteroaryl, or substituted or unsubstituted heterocyclyl; 
 R 2  is substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted cycloalkyl, or substituted or unsubstituted heterocyclyl; 
 R 3  is a substituted or unsubstituted oxygen-containing heterocyclyl; 
 Ring A is a 5-membered heteroaryl having at least one nitrogen ring atom; 
 L 2  is —O—(CR a R b ) m —; 
 each occurrence of R a  and R b  are independently hydrogen, hydroxy, or hydroxy(C 1-4 )alkyl; and 
 m is 1-4. 
 
     
     
         29 . The compound of  claim 28 , wherein R 1  is heterocyclyl or heteroaryl. 
     
     
         30 . The compound of  claim 28  or  29 , wherein R 1  is selected from 
       
         
           
           
               
               
           
         
       
     
     
         31 . The compound of any one of  claims 28 - 30 , wherein R 2  is substituted phenyl. 
     
     
         32 . The compound of  claim 31 , wherein R 2  is 3-methoxyphenyl or 3-methylphenyl. 
     
     
         33 . The compound of any one of  claims 28 - 32 , wherein R 3  is selected from 
       
         
           
           
               
               
           
         
       
     
     
         34 . The compound of  claim 33 , wherein R 3  is 
       
         
           
           
               
               
           
         
       
     
     
         35 . The compound of  claim 33 , wherein R 3  is 
       
         
           
           
               
               
           
         
       
     
     
         36 . The compound of any one of  claims 28 - 35 , wherein ring A is a 5-membered heteroaryl having (i) two nitrogen ring atoms or (ii) one nitrogen ring atom and one sulfur ring atom. 
     
     
         37 . The compound of any one of  claims 28 - 36 , wherein ring A is selected from 
       
         
           
           
               
               
           
         
       
     
     
         38 . The compound of any one of  claims 28 - 37 , wherein L 2  is —OCH 2 —, —OCH 2 CH 2 —, —OCH 2 CH 2 CH(OH)CH 2 —, or —CH 2 CH 2 —. 
     
     
         39 . The compound of  claim 38 , wherein L 2  is —OCH 2 —, —OCH 2 CH 2 —, or —OCH 2 CH 2 CH(OH)CH 2 —. 
     
     
         40 . A compound selected from 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
       and pharmaceutically acceptable salts thereof. 
     
     
         41 . A method of inhibiting PIKfyve in a subject in need thereof comprising administering an effective amount of a compound of any one of  claims 1 - 40 . 
     
     
         42 . A method for treating a disease or disorder associated with PIKfyve in a human subject in need thereof comprising administering an effective amount of a compound of any one of  claims 1 - 40 . 
     
     
         43 . A method of treating a subject having a neurological disease comprising administering to the subject an effective amount of a compound of any one of  claims 1 - 40 . 
     
     
         44 . The method of  claim 43 , wherein the neurological disease is amyotrophic lateral sclerosis (ALS). 
     
     
         45 . The method of  claim 43 , wherein the neurological disease is frontotemporal dementia (FTD). 
     
     
         46 . The method of  claim 43 , wherein the neurological disease is Alzheimer's disease. 
     
     
         47 . The method of  claim 43 , wherein the neurological disease is Parkinson's disease. 
     
     
         48 . The method of  claim 43 , wherein the neurological disease is Huntington's disease. 
     
     
         49 . The method of  claim 43 , wherein the neurological disease is Charcot-Marie-Tooth disease (CMT). 
     
     
         50 . A method of treating a viral infection in a subject in need thereof comprising administering an effective amount of a compound of any one of  claims 1 - 40 . 
     
     
         51 . The method of  claim 50  wherein the virus is Ebola virus. 
     
     
         52 . The method of  claim 50  wherein the virus is middle east respiratory syndrome virus (MERS). 
     
     
         53 . The method of  claim 50  wherein the virus is JC polyomavirus (JC). 
     
     
         54 . The method of  claim 50  wherein the virus is BK polyomavirus (BK). 
     
     
         55 . The method of  claim 50  wherein the virus is Herpes Simplex Virus (HSV). 
     
     
         56 . The method of  claim 50  wherein the virus is Marburg virus (MarV). 
     
     
         57 . The method of  claim 50  wherein the virus is Venezuelan equine encephalitis virus (VEEV). 
     
     
         58 . The method of  claim 50  wherein the virus is Lymphocytic choriomeningitis virus (LCMV).

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