US2017051013A1PendingUtilityA1

Hepcidin mimetic peptides and uses thereof

Assignee: MERGANSER BIOTECH INCPriority: Apr 7, 2014Filed: Apr 14, 2016Published: Feb 23, 2017
Est. expiryApr 7, 2034(~7.7 yrs left)· nominal 20-yr term from priority
Inventors:Gene Merutka
A61P 7/06A61P 7/00A61P 3/00A61K 38/08A61K 38/00C07K 7/02A61P 1/16C07K 7/06
40
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Claims

Abstract

Compounds and methods are described herein that can be used to treat subjects for conditions related to hepcidin activity, such as but not limited diseases of iron metabolism, beta thalassemia, hemochromatosis, iron-loading anemias, alcoholic liver disease, or chronic hepatitis C.

Claims

exact text as granted — not AI-modified
1 - 15 . (canceled) 
     
     
         16 . A compound, or a pharmaceutically acceptable salt thereof, of Formula II: 
       
         
           
           
               
               
           
         
         wherein: 
         R 1  is, H, —S—Z 1 ; —Z 2 , —SH, —C(═O)—Z 3 , or —S—C(═O)—Z 3 , 
         R 2  and R 3  are each, independently, optionally substituted C 4 -C 7  alkyl, 
       
       
         
           
           
               
               
           
         
       
       D-Arg, D-Ile, Leu, D-Leu, Thr, D-Thr, Lys, D-Lys, Val, D-Val, D-Nω,ω-dimethyl-arginine, L-Nω,ω-dimethyl-arginine, D-homoarginine, L-homoarginine, D-norarginine, L-norarginine, citrulline, a modified Arg wherein the guanidinium group is modified or substituted, norleucine, norvaline, beta homo-Ile, Ach, N-Me-Arg, N-Me-Ile;
 R 4  is Ida, Asp, Acetyl-Asp, N-MeAsp, Acetyl-Gly-Ida, or Acetyl-Gly-Asp or a derivative thereof to remove its negative charge above pH 4; 
 R 5  is CR 6 R 7 , aryl or heteroaryl; 
 B is absent or forms a 5-7 membered ring; and 
 q is 0-6, wherein when R 5  aryl or heteroaryl q is 1 and B is absent;
 Z 1  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 
 Z 2  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 Z 3  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 R 6  and R 7  are each, independently, H, halo, optionally substituted C 1 -C 3  alkyl, or haloalkyl, provided that when R 1  is H, the compound is not Compound 1. 
 
     
     
         17 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein the compound is a compound of Formula II-A, II-B, or II-C: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         18 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein R 1  is, H, —S—Z 1  or —C(═O)—Z 3 . 
     
     
         19 - 20 . (canceled) 
     
     
         21 . A compound, or a pharmaceutically acceptable salt thereof, of Formula III: 
       
         
           
           
               
               
           
         
         wherein: 
         R 1  is H, —S—Z 1 , —Z 2 , —SH, —S—C(═O)—Z 3 , or —C(═O)—Z 3    
         R 2  and R 3  are each, independently, optionally substituted C 4 -C 7  alkyl, 
       
       
         
           
           
               
               
           
         
       
       D-Arg, D-Ile, Leu, D-Leu, Thr, D-Thr, Lys, D-Lys, Val, D-Val, D-Nω,ω-dimethyl-arginine, L-Nω,ω-dimethyl-arginine, D-homoarginine, L-homoarginine, D-norarginine, L-norarginine, citrulline, a modified Arg wherein the guanidinium group is modified or substituted, norleucine, norvaline, beta homo-Ile, Ach, N-Me-Arg, N-Me-Ile;
 R 4  is Ida, Asp, Acetyl-Asp, N-MeAsp, Acetyl-Gly-Ida, or Acetyl-Gly-Asp or a derivative thereof to remove its negative charge above pH 4; 
 B is absent or forms a 5-7 membered ring; and
 Z 1  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 Z 2  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 Z 3  is substituted or unsubstituted C 1 -C 18  alkyl, wherein the C 1 -C 18  alkyl is branched or unbranched; 
 
 provided that when R 1  is H, the compound is not Compound 1. 
 
     
     
         22 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 21 , wherein the compound, or a pharmaceutically acceptable salt thereof, has a formula of Formula III-A. 
       
         
           
           
               
               
           
         
       
     
     
         23 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein R 5  is CR 6 R 7 . 
     
     
         24 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 23 , wherein R 6  and R 7  are H. 
     
     
         25 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 23 , wherein R 6  is H and R 7  is halo, optionally substituted C 1 -C 3  alkyl, or haloalkyl. 
     
     
         26 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein R 5  is aryl or heteroaryl. 
     
     
         27 . (canceled) 
     
     
         28 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein R 2  and R 3  are 
       
         
           
           
               
               
           
         
       
     
     
         29 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein R 2  and R 3  are 
       
         
           
           
               
               
           
         
       
     
     
         30 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein one of R 2  and R 3  is 
       
         
           
           
               
               
           
         
       
       and the other is 
       
         
           
           
               
               
           
         
       
     
     
         31 - 33 . (canceled) 
     
     
         34 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein 
       
         
           
           
               
               
           
         
       
       is modified to reduce the charge. 
     
     
         35 - 51 . (canceled) 
     
     
         52 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein the compound is a compound of Formula IV, or a pharmaceutically acceptable salt thereof: 
       
         
           
           
               
               
           
         
         wherein the carbonyl forms a bond with the 6-membered ring at C a , C b , or C c . 
       
     
     
         53 - 55 . (canceled) 
     
     
         56 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein the compound is a compound of Formula V, or a pharmaceutically acceptable salt thereof, 
       
         
           
           
               
               
           
         
         wherein the carbonyl forms a bond with the 5-membered ring at C d  or C e . 
       
     
     
         57 . The compound, or a pharmaceutically acceptable salt thereof, of  claim 16 , wherein the compound is a compound of Formula VI, or a pharmaceutically acceptable salt thereof, 
       
         
           
           
               
               
           
         
         wherein the bond from the carbonyl forms a bond with the 7-membered ring at C f , C g , C h , or C i . 
       
     
     
         58 - 60 . (canceled) 
     
     
         61 . A compound, or a pharmaceutically acceptable salt thereof, selected from the group consisting of: Compound 2, Compound 3, Compound 4, Compound 5, Compound 6, Compound 7, Compound 8, Compound 9, and Compound 10, or a pharmaceutically acceptable salt thereof. 
     
     
         62 . A compound, or a pharmaceutically acceptable salt thereof, of the formula:
 P 1 -P 2 -P 3 -P 4 -P 5 -P 6 -P 7 -P 8 -P 9 -P 10  or P 10 -P 9 -P 8 -P 7 -P 6 -P 5 -P 4 -P 3 -P 2 -P 1 , wherein P 1  to P 10  are as defined in the following table:   
       
         
           
                 
                 
                 
                 
                 
                 
                 
                 
                 
                 
                 
               
                     
                 
                   Compound # 
                   P 1   
                   P 2   
                   P 3   
                   P 4   
                   P 5   
                   P 6   
                   P 7   
                   P 8   
                   P 9   
                   P 10   
                 
                     
                 
                     
                 
                 
                 
                 
                 
                 
                 
                 
                 
                 
                 
                 
               
                   2 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   bhPro 
                   Arg 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   3 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   bhPro 
                   Arg 
                   Cys-C(═O)CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   4 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   bhPro 
                   Arg 
                   Cys-CH 2 —CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   5 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   Npc 
                   Arg 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   6 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   Npc 
                   Arg 
                   Cys 
                   Arg 
                   Trp 
                   X 3   
                 
                   7 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   D-Npc 
                   Arg 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   8 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   isoNpc 
                   Arg 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   9 
                   Acetyl-Gly-Ida 
                   Thr 
                   His 
                   Dpa 
                   bhPro 
                   Arg 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                   10 
                   Ida 
                   Thr 
                   His 
                   Dpa 
                   bAla 
                   Ara 
                   Cys-S—CH 3   
                   Arg 
                   Trp 
                   X 3   
                 
                     
                 
             
                
                
                
               
               
                
               
            
             
                
                
                
                
                
                
                
                
                
                
               
            
           
         
       
       wherein: X 3  is Ahx-Ida(NH-PAL)-NH 2 , Ida is Iminodiacetic acid; bhPro is beta-homoproline, Npc is L-nipecotic acid; isoNpc is isonipecotic acid and bAla is beta-alanine. 
     
     
         63 . A pharmaceutical composition comprising the compound, or a pharmaceutically acceptable salt thereof, of  claim 16  and a pharmaceutically acceptable carrier. 
     
     
         64 . A method of treating a subject or a subject in need thereof a disease of iron metabolism, beta thalassemia, hemochromatosis, iron-loading anemias, alcoholic liver disease, or chronic hepatitis C, comprising administering to the subject a compound, or a pharmaceutically acceptable salt thereof, of  claim 16 . 
     
     
         65 - 82 . (canceled)

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