IP Library Granted Patent US 11,685,721
Granted Patent B2
US 11,685,721 · App. 17/522,065 · Granted Jun 27, 2023

Human plasma kallikrein inhibitors

Inventors: Pravin L. Kotian (Hoover, AL); Yarlagadda S. Babu (Birmingham, AL); Minwan Wu (Vestavia Hills, AL); Venkat R. Chintareddy (Vestavia Hills, AL); V. Satish Kumar (Birmingham, AL); Weihe Zhang (Vestavia Hills, AL)
Assignee: BioCryst Pharmaceuticals, Inc.
C07D231/14C07D401/04C07D401/12C07D401/14C07D403/04C07D403/12C07D405/12C07D413/04C07D413/12C07D417/12
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Quick Facts
Patent No.
US 11,685,721
App. No.
17/522,065
Granted
Jun 27, 2023
Kind
B2
Abstract

Disclosed are compounds of formula I as described herein, and pharmaceutically acceptable salts thereof. The compounds are inhibitors of plasma kallikrein. Also provided are pharmaceutical compositions comprising at least one compound of the invention, and methods involving use of the compounds and compositions of the invention in the treatment and prevention of diseases and conditions characterized by unwanted plasma kallikrein activity.

Claims (46)

1. A compound, or a pharmaceutically acceptable salt thereof, represented by formula XXVI:

wherein:

X represents C(NR a R b );

—Y—R 4 represents —((C 1 -C 6 )alkyl)-R 4 , —CH 2 C(O)—R 4 , —CH 2 NH-R 4 , —CH 2 N((C 1 -C 6 )alkyl)-R 4 , —CR a R b —R 4 , —NH—R 4 , —NHCH 2 —R 4 , —NHC(O)—R 4 , —N((C 1 -C 6 )alkyl)-R 4 , —N((C 1 -C 6 )alkyl)CH 2 —R 4 , —N((CH 2 ) 2 OH)—R 4 , —N[(C 3 -C 8 )cycloalkyl(C 1 -C 6 )alkyl]R 4 , —heterocyclyl-R 4 , —OR 4 , —OCH 2 —R 4 , —OC(O)—R 4 , —OC(O)NR a R b , —SCH 2 R 4 , or —SR 4 , wherein the (C 1 -C 6 )alkyl moiety of —((C 1 -C 6 )alkyl)-R 4 is optionally substituted;

Z is absent or represents halo, hydroxy, (C 1 -C 6 )alkyl, —CF 3 , —OCF 3 , (C 1 -C 6 )alkoxy, aryl, aryloxy, amino, amino(C 1 -C 6 )alkyl, —C(O)NH 2 , cyano, —NHC(O)(C 1 -C 6 )alkyl, —SO 2 (C 1 -C 6 )alkyl, —SO 2 NH 2 , or (C 3 -C 8 )cycloalkyl;

R 1c represents halo, amino(C 1 -C 6 )alkyl, (C 1 -C 6 )alkoxy, cyano, —SO 2 CH 3 , formyl, acyl, —NH 2 , or optionally substituted aryl;

R 2 represents halo, (C 1 -C 6 )alkyl, (C 3 -C 8 )cycloalkyl, (C 1 -C 6 )fluoroalkyl, —OCH 3 , —Si(CH 3 ) 3 , —CONH 2 , —C(O)OH, cyano, or phenyl;

R 3 represents —NH—, —O—, optionally substituted aryl, heteroaryl, phenyl, carbocyclyl, or heterocyclyl;

R 3a is absent or represents one or more substituents independently selected from the group consisting of halo, hydroxy, (C 1 -C 6 )alkyl, —CF 3 , —OCF 3 , (C 1 -C 6 )alkoxy, aryl, aryloxy, amino, amino(C 1 -C 6 )alkyl, —C(O)NH 2 , cyano, —NHC(O)(C 1 -C 6 )alkyl, -SO 2 (C 1 -C 6 )alkyl, and —SO 2 NH 2 ; and

R 4 represents hydrogen, hydroxy, optionally substituted (C 1 -C 6 )alkyl, optionally substituted (C 3 -C 8 )cycloalkyl, heterocyclyl(C 1 -C 6 )alkyl, (C 3 -C 8 )cycloalkyl(C 1 -C 6 )alkyl, —CH 2 OH, —CH((C 1 -C 6 )alkyl)OH, —CH(NH 2 )CH((C 1 -C 6 )alkyl) 2 , optionally substituted aryl, optionally substituted aryl(C 1 -C 6 )alkyl, heteroaryl, optionally substituted heteroaryl(C 1 -C 6 )alkyl, —CH 2 S(C 1 -C 6 )alkyl, amino, or cyano; or —CH 2 -fused to the 4-position of the ring bearing Z to form a 5- to 7-membered heterocyclic ring with optional substituents; or, when R 3 is phenyl, can represent —NH— fused to the position ortho to X on that phenyl;

each R a and R b is independently H, (C 1 -C 8 )alkyl, (C 2 -C 8 )alkenyl, (C 2 -C 8 )alkynyl, aryl(C 1 -C 8 )alkyl, (C 3 -C 8 )carbocyclyl, —C(═O)R c , —C(═O)OR c , —C(═O)NR c R d , —C(═O)SR c , —S(O)R c , —S(O) 2 R c , —S(O)(OR c ), or —SO 2 NR c R d ;

each R c and R d is independently H, (C 1 -C 8 )alkyl, (C 2 -C 8 )alkenyl, (C 2 -C 8 )alkynyl, (C 4 -C 8 ) carbocyclyl, optionally substituted aryl, optionally substituted heteroaryl, —C(═O)(C 1 -C 8 )alkyl, —S(O) n (C 1 -C 8 )alkyl, or aryl(C 1 -C 8 )alkyl;

n is 2 or 3; and

the stereochemical configuration at any chiral center is R, S, or a mixture of R and S.

2. The compound of claim 1 , wherein Z represents halo.

3. The compound of claim 1 , wherein Z represents F.

4. The compound of claim 1 , wherein R 1c represents —NH 2 .

5. The compound of claim 1 , wherein R 2 represents (C 1 -C 6 )fluoroalkyl.

6. The compound of claim 1 , wherein R 2 represents CF 3 .

7. The compound of claim 1 , wherein R 3 represents optionally substituted aryl or heteroaryl.

8. The compound of claim 1 , wherein R 3 represents optionally substituted phenyl.

9. The compound of claim 8 , wherein R 3a is absent or represents cyano.

10. The compound of claim 1 , wherein R 3 represents pyridyl.

11. The compound of claim 10 , wherein R 3a is absent.

12. The compound of claim 1 , wherein R 3a is absent or represents cyano.

13. The compound of claim 1 , wherein —Y—R 4 represents —((C 1 -C 6 )alkyl)-R 4 .

14. The compound of claim 1 , wherein R 4 represents optionally substituted (C 3 -C 8 )cycloalkyl.

15. The compound of claim 1 , wherein —Y—R 4 represents —CH 2 CH 2 —R 4 .

16. The compound of claim 1 , wherein R 4 represents (C 3 )-cycloalkyl.

17. The compound of claim 1 , wherein R a is H; and R b is H.

18. The compound of claim 1 , wherein R a is H.

19. The compound of claim 18 , wherein R b is —S(O) 2 R c ; and R c is (C 1 -C 8 )alkyl.

20. The compound of claim 1 , wherein the pharmaceutically acceptable salt thereof is a hydrochloride salt.

21. The compound of claim 1 , wherein the pharmaceutically acceptable salt thereof is a bis(hydrochloride) salt.

22. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein said compound is selected from the group consisting of:

23. The compound of claim 22 , wherein the pharmaceutically acceptable salt thereof is a hydrochloride salt.

24. The compound of claim 22 , wherein the pharmaceutically acceptable salt thereof is a bis(hydrochloride) salt.

25. A pharmaceutical composition, comprising a compound of claim 1 , or a pharmaceutically acceptable salt thereof; and a pharmaceutically acceptable carrier.

26. A method of treating a disease or condition characterized by unwanted plasma kallikrein activity, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of claim 1 .

27. The method of claim 26 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is selected from the group consisting of stroke, inflammation, reperfusion injury, acute myocardial infarction, deep vein thrombosis, post fibrinolytic treatment condition, angina, edema, angioedema, hereditary angioedema, sepsis, arthritis, hemorrhage, blood loss during cardiopulmonary bypass, inflammatory bowel disease, diabetes mellitus, retinopathy, diabetic retinopathy, diabetic macular edema, diabetic macular degeneration, age-related macular edema, age-related macular degeneration, proliferative retinopathy, neuropathy, hypertension, brain edema, increased albumin excretion, macroalbuminuria, and nephropathy.

28. The method of claim 26 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is angioedema.

29. The method of claim 26 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is hereditary angioedema.

30. A method of treating a disease or condition characterized by unwanted plasma kallikrein activity, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of claim 22 .

31. The method of claim 30 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is selected from the group consisting of stroke, inflammation, reperfusion injury, acute myocardial infarction, deep vein thrombosis, post fibrinolytic treatment condition, angina, edema, angioedema, hereditary angioedema, sepsis, arthritis, hemorrhage, blood loss during cardiopulmonary bypass, inflammatory bowel disease, diabetes mellitus, retinopathy, diabetic retinopathy, diabetic macular edema, diabetic macular degeneration, age-related macular edema, age-related macular degeneration, proliferative retinopathy, neuropathy, hypertension, brain edema, increased albumin excretion, macroalbuminuria, and nephropathy.

32. The method of claim 30 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is angioedema.

33. The method of claim 30 , wherein the disease or condition characterized by unwanted plasma kallikrein activity is hereditary angioedema.

Assignments (4)
SECURITY INTEREST Recorded Jan 23, 2026
From: BIOCRYST PHARMACEUTICALS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 074485/0651 →
TERMINATION AND RELEASE OF SECURITY INTEREST IN PATENT COLLATERAL RECORDED AT REEL 063363/FRAME 0873 Recorded Oct 8, 2025
From: BIOPHARMA CREDIT PLC
To: BIOCRYST PHARMACEUTICALS, INC.
Reel/Frame 073056/0492 →
PATENT SECURITY AGREEMENT Recorded Apr 19, 2023
From: BIOCRYST PHARMACEUTICALS, INC.
To: BIOPHARMA CREDIT PLC
Reel/Frame 063363/0873 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 9, 2022
From: KOTIAN, PRAVIN L.; BABU, YARLAGADDA S.; WU, MINWAN; CHINTAREDDY, VENKAT R.; KUMAR, V. SATISH; ZHANG, WEIHE
To: BIOCRYST PHARMACEUTICALS, INC.
Reel/Frame 059204/0913 →