IP Library Granted Patent US 9,493,509
Granted Patent B2
US 9,493,509 · App. 14/677,679 · Granted Nov 15, 2016

Triazole macrocycle systems

Inventor: Huw M. Nash (Concord, MA)
Assignee: AILERON THERAPEUTICS, INC.
C07K7/06C07C271/22C07K1/006C07K1/1075C07K7/08C07K7/56C07K14/4748C07C2103/18
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Quick Facts
Patent No.
US 9,493,509
App. No.
14/677,679
Granted
Nov 15, 2016
Kind
B2
Abstract

The present invention provides novel peptidomimetic macrocycles and methods for their preparation and use, as well as amino acid analogs and macrocycle-forming linkers, and kits useful in their production. Macrocycles of the invention include triazole moieties that crosslink amino acid side chains. The cross links can stabilize a secondary structure of a peptidomimetic macrocycle, such as an α-helix.

Claims (41)

1. A peptidomimetic macrocycle of Formula (I):

wherein:

each A, C, D, and E is independently a natural or non-natural amino acid;

each B is independently a natural or non-natural amino acid, amino acid analog,

[—NH-L 3 -CO—], [—NH-L 3 -SO 2 —], or [—NH-L 3 -];

R 1 and R 2 are independently —H, alkyl, alkenyl, alkynyl, arylalkyl, cycloalkyl, cycloalkylalkyl, heteroalkyl, or heterocycloalkyl, each non-H group being unsubstituted or substituted with halo-;

each R 3 is independently hydrogen, alkyl, alkenyl, alkynyl, arylalkyl, heteroalkyl, cycloalkyl, heterocycloalkyl, cycloalkylalkyl, cycloaryl, or heterocycloaryl, each non-H group being optionally substituted with R 5 ;

L is a triazole-containing macrocycle-forming linker of the formula

each L 1 , L 2 , and L 3 is independently alkylene, alkenylene, alkynylene, heteroalkylene, cycloalkylene, heterocycloalkylene, cycloarylene, heterocycloarylene, or [—R 4 —K—R 4 -] n , each being optionally substituted with R 5 ;

each R 4 is alkylene, alkenylene, alkynylene, heteroalkylene, cycloalkylene, heterocycloalkylene, arylene, or heteroarylene;

each K is O, S, SO, SO 2 , CO, C(═O)—O—[[CO 2 ]], or CONR 3 ;

each R 5 is independently halogen, alkyl, —OR 6 , —N(R 6 ) 2 , —SR 6 , —SOR 6 , —SO 2 R 6 , —CO 2 R 6 , a fluorescent moiety, a radioisotope or a therapeutic agent;

each R 6 is independently —H, alkyl, alkenyl, alkynyl, arylalkyl, cycloalkylalkyl, heterocycloalkyl, a fluorescent moiety, a radioisotope or a therapeutic agent;

R 7 is —H, alkyl, alkenyl, alkynyl, arylalkyl, cycloalkyl, heteroalkyl, cycloalkylalkyl, heterocycloalkyl, cycloaryl, or heterocycloaryl, each non-H group being optionally substituted with R 5 , or part of a cyclic structure with a D residue;

R 8 is —H, alkyl, alkenyl, alkynyl, arylalkyl, cycloalkyl, heteroalkyl, cycloalkylalkyl, heterocycloalkyl, cycloaryl, or heterocycloaryl, each non-H group being optionally substituted with R 5 , or part of a cyclic structure with an E residue;

v is an integer from 1-1000;

w is an integer from 1-1000;

x+y+z is 3, 4, 5, 6, 7, 8, 9, or 10; and

n is an integer from 1-5.

2. The peptidomimetic macrocycle of claim 1 , wherein x+y+z is 3.

3. The peptidomimetic macrocycle of claim 1 , wherein x+y+z is 6.

4. The peptidomimetic macrocycle of claim 1 , wherein L is

5. The peptidomimetic macrocycle of claim 1 , wherein L is

6. The peptidomimetic macrocycle of claim 1 , wherein at least one of R 1 and R 2 is alkyl, alkenyl, alkynyl, arylalkyl, cycloalkyl, cycloalkylalkyl, heteroalkyl, or heterocycloalkyl, each non-H group being unsubstituted or substituted with halo-.

7. The peptidomimetic macrocycle of claim 1 , wherein R 1 and R 2 are independently alkyl, alkenyl, alkynyl, arylalkyl, cycloalkyl, cycloalkylalkyl, heteroalkyl, or heterocycloalkyl, each non-H group being unsubstituted or substituted with halo-.

8. The peptidomimetic macrocycle of claim 1 , wherein at least one of R 1 and R 2 is alkyl, each non-H group being unsubstituted or substituted with halo-.

9. The peptidomimetic macrocycle of claim 1 , wherein R 1 and R 2 are independently alkyl, each non-H group being unsubstituted or substituted with halo-.

10. The peptidomimetic macrocycle of claim 1 , wherein at least one of R 1 and R 2 is methyl.

11. The peptidomimetic macrocycle of claim 1 , wherein R 1 and R 2 are methyl.

12. The peptidomimetic macrocycle of claim 1 , wherein at least one of D and E is attached to an additional macrocycle-forming linker.

13. The peptidomimetic macrocycle of claim 1 , wherein a secondary structure of the peptidomimetic macrocycle is more stable than a corresponding secondary structure of a corresponding non-macrocyclic polypeptide.

14. The peptidomimetic macrocycle of claim 1 , wherein the peptidomimetic macrocycle exhibits increased thermal stability compared to a corresponding non-macrocyclic polypeptide.

15. The peptidomimetic macrocycle of claim 1 , wherein the peptidomimetic macrocycle exhibits increased biological activity compared to a corresponding non-macrocyclic polypeptide.

16. The peptidomimetic macrocycle of claim 1 , wherein the peptidomimetic macrocycle exhibits increased resistance to proteolytic degradation compared to a corresponding non-macrocyclic polypeptide.

17. The peptidomimetic macrocycle of claim 1 , wherein the peptidomimetic macrocycle exhibits increased ability to penetrate living cells compared to a corresponding non-macrocyclic polypeptide.

18. The peptidomimetic macrocycle of claim 1 , wherein the peptidomimetic macrocycle comprises an α-helix.

19. The peptidomimetic macrocycle of claim 18 , wherein the α-helix comprises from 1 turn to 5 turns.

20. The peptidomimetic macrocycle of claim 18 , wherein the α-helix is more stable than an α-helix of a corresponding non-macrocyclic polypeptide.

21. The peptidomimetic macrocycle of claim 18 , wherein the macrocycle-forming linker spans from 1 turn to 5 turns of the α-helix.

22. The peptidomimetic macrocycle of claim 18 , wherein the macrocycle-forming linker spans approximately 1 turn to approximately 2 turns of the α-helix.

23. The peptidomimetic macrocycle of claim 18 , wherein the length of the macrocycle-forming linker is approximately equal to the length of from about 6 carbon-carbon bonds to about 14 carbon-carbon bonds.

Assignments (2)
CHANGE OF ADDRESS Recorded Oct 7, 2015
From: AILERON THERAPEUTICS, INC.
To: AILERON THERAPEUTICS, INC.
Reel/Frame 036806/0383 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 1, 2015
From: NASH, HUW W.
To: AILERON THERAPEUTICS, INC.
Reel/Frame 036466/0862 →
Continuity (5)
Continuation 13370057 · Feb 9, 2012
Continuation 13097930 · Apr 29, 2011
Division 12037041 · Feb 25, 2008
Provisional Application 60903073 · Feb 23, 2007
Related Publication 20160031936A1 · Feb 4, 2016