US2021260211A1PendingUtilityA1

Antibody Drug Conjugates Having Derivatives of Amatoxin As The Drug

63
Assignee: SORRENTO THERAPEUTICS INCPriority: May 31, 2016Filed: Apr 26, 2021Published: Aug 26, 2021
Est. expiryMay 31, 2036(~9.9 yrs left)· nominal 20-yr term from priority
C07K 14/415C07K 2319/00A61K 47/6889A61K 47/6863C07K 16/32A61K 2039/505C07K 7/64A61K 47/6855A61K 47/6857A61P 43/00A61K 47/6831A61K 47/6851A61K 47/6883C07K 19/00A61P 35/00
63
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Claims

Abstract

There is disclosed derivatives of amanitin conjugated to a targeting antibody to form an ADC (antibody drug conjugate).

Claims

exact text as granted — not AI-modified
1 - 3 . (canceled) 
     
     
         4 . A compound having a structure of the following formula:
   L 1 -L 2 -X-D   or a pharmaceutically acceptable salt thereof,   wherein:
 L 1 -L 2  is a linker selected from 
   
       
         
           
           
               
               
           
         
         
           L 2 -X has a structure of 
         
       
       
         
           
           
               
               
           
         
         wherein R 4  is hydrogen or C 1-6  alkyl; and the wavy line indicates the point of attachment to D;
 L 2  is a linker comprising (a) —R 6 OC(O)NR 5 —, and (b) —(CH 2 ) p —, —(CH 2 CH 2 O) m —, —C(O)NH—, —NHC(O)—, or a combination of two or more thereof, wherein: 
 
         R 5  is hydrogen, C 1-6  alkyl, —(CH 2 ) p —, —(CH 2 CH 2 O) m —, or a combination of two or more thereof; 
         R 6  is Val-Cit-PAB or Ala-Ala-Asn-PAB; 
         wherein —R 6 OC(O)NR 5 — is connected to L 1  through R 5  or R 6 ;
 D is a drug moiety active agent derived from amatoxin having a structure of Formula (II): 
 
       
       
         
           
           
               
               
           
         
         wherein R 1  is NH 2  or OR 2 ; R 2  is H or C 1-10  alkyl; R 3  is H or OH; and the wavy line indicates the point of attachment to X;
 m is an integer from 1-24; and 
 p is an integer from 1-6. 
 
       
     
     
         5 . The compound of  claim 4 , wherein R 4  is C 1-6  alkyl. 
     
     
         6 . The compound of  claim 5 , wherein R 4  is isopropyl. 
     
     
         7 . The compound of  claim 5 , wherein R 4  is methyl. 
     
     
         8 . The compound of  claim 5 , wherein L 1 -L 2  is 
       
         
           
           
               
               
           
         
       
     
     
         9 . The compound of  claim 5 , wherein L 2  comprises (a) —R 6 OC(O)NR 5 — and (b) —(CH 2 ) p —. 
     
     
         10 . The compound of  claim 4 , wherein R 6  is Ala-Ala-Asn-PAB. 
     
     
         11 . The compound of  claim 4 , wherein R 6  is Val-Cit-PAB. 
     
     
         12 . The compound of  claim 4 , wherein R 5  is —(CH 2 ) p —. 
     
     
         13 . The compound of  claim 4 , wherein R 5  is C 1-6  alkyl. 
     
     
         14 . The compound of  claim 13 , wherein R 5  is methyl. 
     
     
         15 . The compound of  claim 4 , wherein D has a structure of Formula (II): 
       
         
           
           
               
               
           
         
         wherein R 1  is NH 2  or OH; R 3  is H or OH; and the wavy line indicates the point of attachment to X. 
       
     
     
         16 . The compound of  claim 4 , which is selected from: 
       
         
           
           
               
               
           
         
         
           
           
               
               
           
         
       
     
     
         17 . The compound of  claim 16 , having the structure: 
       
         
           
           
               
               
           
         
       
     
     
         18 . An antibody drug conjugate (ADC) having the structure of Formula (I):
   Ab L 1 -L 2 -X-D) n    (I)
   or a pharmaceutically acceptable salt thereof,   wherein:
 Ab is a monoclonal antibody; 
 L 1 -L 2  is a linker selected from 
   
       
         
           
           
               
               
           
         
         wherein the wavy line indicates the point of attachment to Ab;
 L 2 -X has a structure of 
 
       
       
         
           
           
               
               
           
         
         wherein R 4  is hydrogen or C 1-6  alkyl; and the wavy line indicates the point of attachment to D;
 L 2  is a linker comprising —R 8 —S—S—R 7 —, wherein: 
 
         R 7  is C 2-6  alkylene or —(CH 2 CH 2 O) m —; 
         R 8  is selected from C 1-6  alkyl, C 1-6  alkylene, substituted C 1-6  alkylene, —C(O)NH—, —C(O)—NH—CHR 9 —CR 10 R 11 —, —NHC(O)—CHR 9 —CR 10 R 11 —, —(CH 2 CH 2 O) m —, and combinations thereof; 
         R 9  is selected from hydrogen, C 1-6  alkyl, C 1-6  alkylene, —(CH 2 CH 2 O) m —, —C(O)NH—, —NHC(O)—, —C(O)NH—(CH 2 ) p —SO 3 H, C(O)NH—(CH 2 ) p —CO 2 H, —NHC(O)—(CH 2 ) p —SO 3 H, —NHC(O)—(CH 2 ) p —CO 2 H, and combinations thereof; 
         R 10  and R 11  are each independently selected from hydrogen, C 1-6  alkyl, and combinations thereof; 
         wherein —R 8 —S—S—R 7 — is connected to L 1  through R 8 ;
 D is a drug moiety active agent derived from amatoxin having a structure of Formula (II): 
 
       
       
         
           
           
               
               
           
         
         wherein R 1  is NH 2  or OR 2 ; R 2  is H or C 1-10  alkyl; R 3  is H or OH; and the wavy line indicates the point of attachment to X;
 n is an integer from 1-10; 
 m is an integer from 1-24; and 
 p is an integer from 1-6. 
 
       
     
     
         19 . The ADC of  claim 18 , wherein L 1 -L 2  is: 
       
         
           
           
               
               
           
         
       
     
     
         20 . The ADC of  claim 18 , wherein R 4  is C 1-6  alkyl. 
     
     
         21 . The ADC of  claim 20 , wherein R 4  is methyl. 
     
     
         22 . The ADC of  claim 18 , wherein R 7  is C 2-6  alkylene. 
     
     
         23 . The ADC of  claim 18 , where Ab is an anti-HER2 antibody. 
     
     
         24 . The ADC of  claim 18 , wherein the ADC is: 
       
         
           
           
               
               
           
         
       
     
     
         25 . A compound having the structure of the following formula:
   L 1 -L 2 -X-D   or a pharmaceutically acceptable salt thereof,   wherein:
 L 1 -L 2  is a linker selected from 
   
       
         
           
           
               
               
           
         
         
           L 2 -X has a structure of 
         
       
       
         
           
           
               
               
           
         
         wherein R 4  is hydrogen or C 1-6  alkyl; and the wavy line indicates the point of attachment to D;
 L 2  is a linker comprising —R 8 —S—S—R 7 —, wherein: 
 
         R 7  is C 2-6  alkylene or —(CH 2 CH 2 O) m —; 
         R 8  is selected from C 1-6  alkyl, C 1-6  alkylene, substituted C 1-6  alkylene, —C(O)NH—, —C(O)—NH—CHR 9 —CR 10 R 11 —, —NHC(O)—CHR 9 —CR 10 R 11 —, —(CH 2 CH 2 O) m —, and combinations thereof; 
         R 9  is selected from hydrogen, C 1-6  alkyl, C 1-6  alkylene, —(CH 2 CH 2 O) m —, —C(O)NH—, —NHC(O)—, —C(O)NH—(CH 2 ) p —SO 3 H, C(O)NH—(CH 2 ) p —CO 2 H, —NHC(O)—(CH 2 ) p —SO 3 H, —NHC(O)—(CH 2 ) p —CO 2 H, and combinations thereof; 
         R 10  and R 11  are each independently selected from hydrogen, C 1-6  alkyl, and combinations thereof; 
         wherein —R 8 —S—S—R 7 — is connected to L 1  through R 8 ;
 D is a drug moiety active agent derived from amatoxin having a structure of Formula (II): 
 
       
       
         
           
           
               
               
           
         
         wherein R 1  is NH 2  or OR 2 ; R 2  is H or C 1-10  alkyl; R 3  is H or OH; and the wavy line indicates the point of attachment to X;
 m is an integer from 1-24; and 
 p is an integer from 1-6. 
 
       
     
     
         26 . The compound of  claim 25 , wherein L 1 -L 2  is: 
       
         
           
           
               
               
           
         
       
     
     
         27 . The compound of  claim 25 , wherein R 4  is C 1-6  alkyl. 
     
     
         28 . The compound of  claim 27 , wherein R 4  is methyl. 
     
     
         29 . The compound of  claim 25 , wherein R 7  is C 2-6  alkylene. 
     
     
         30 . The compound of  claim 25 , wherein the compound is: 
       
         
           
           
               
               
           
         
       
     
     
         31 . A method of treating a cancer in an individual in need thereof, comprising administering an effective amount of the ADC of  claim 18  to the individual. 
     
     
         32 . A method of treating a cancer in an individual in need thereof, comprising administering an effective amount of an antibody drug conjugate (ADC) to the individual, wherein the ADC has the structure of Formula (I):
   Ab L 1 -L 2 -X-D) n    (I)
   or a pharmaceutically acceptable salt thereof,   wherein:
 Ab is a monoclonal antibody; 
 L 1 -L 2  is a linker selected from 
   
       
         
           
           
               
               
           
         
         wherein the wavy line indicates the point of attachment to Ab;
 L 2 -X has a structure of 
 
       
       
         
           
           
               
               
           
         
         wherein R 4  is hydrogen or C 1-6  alkyl, and the wavy line indicates the point of attachment to D;
 L 2  is a linker comprising (a) —R 6 OC(O)NR 5 —, and (b) —(CH 2 ) p —, —(CH 2 CH 2 O) m —, —C(O)NH—, —NHC(O)—, or a combination of two or more thereof, wherein: 
 R 5  is hydrogen, C 1-6  alkyl, —(CH 2 ) p —, —(CH 2 CH 2 O) m —, or a combination of two or more thereof; 
 R 6  is Val-Cit-PAB or Ala-Ala-Asn-PAB; 
 wherein —R 6 OC(O)NR 5 — is connected to L 1  through R 5  or R 6 ; 
 D is a drug moiety active agent derived from amatoxin having a structure of Formula (II): 
 
       
       
         
           
           
               
               
           
         
         
           wherein R 1  is NH 2  or OR 2 , R 2  is H or C 1-10  alkyl, and R 3  is H or OH; and 
           the wavy line indicates the point of attachment to X; 
           n is an integer from 1-10; 
           m is an integer from 1-24; and 
           p is an integer from 1-6.

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