IP Library Granted Patent US 12,319,701
Granted Patent B2
US 12,319,701 · App. 17/766,341 · Granted Jun 3, 2025

Antagonists of the muscarinic acetylcholine receptor M4

Inventors: Craig W. Lindsley (Brentwood, TN); P. Jeffrey Conn (Nashville, TN); Aaron M. Bender (Spring Hill, TN); Matthew Spock (Nashville, TN); Changho Han (Nashville, TN)
Assignee: Vanderbilt University
C07D495/04C07B59/002C07D405/14C07B2200/05
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Quick Facts
Patent No.
US 12,319,701
App. No.
17/766,341
Granted
Jun 3, 2025
Kind
B2
Abstract

Disclosed herein are substituted hexahydro-1H-cyclopenta[c]pyrrole compounds, which may be useful as antagonists of the muscarinic acetylcholine receptor M 4 (mAChR M 4 ). Also disclosed herein are methods of making the compounds, pharmaceutical compositions comprising the compounds, and methods of treating disorders using the compounds and compositions.

Claims (67)

1. A compound of formula (I):

or a pharmaceutically acceptable salt thereof, wherein:

A is a 5- to 6-membered heteroarene, a 5- to 6-membered heterocycle, a 6-membered arene, or a 5- to 6-membered carbocycle, the heteroarene and heterocycle having 1, 2, or 3 heteroatoms independently selected from N, O, and S;

L is NR or O;

R is hydrogen, C 1-4 alkyl, C 3-4 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl;

R 1 is G 1 , —L′—G 1 , —L′—C 1-3 alkylene—G 1 , —C 1-3 alkylene—G 1 , hydrogen, C 1-6 alkyl, C 1-6 haloalkyl, —L′—C 1-6 alkyl, —L′—C 1-6 haloalkyl, —C(O)NH 2 , or halogen;

L′ is O, —N(R 1a )—, S, S(O), SO 2 , —C(O)—, or —N(R 1a )C(O)—;

G 1 is a 5- to 12-membered heteroaryl, a 6- to 12-membered aryl, a 4- to 12-membered heterocyclyl, or a C 3-12 carbocyclyl, wherein G 1 is optionally substituted with 1-5 substituents independently selected from the group consisting of halogen, cyano, C 1-4 alkyl, C 1-4 haloalkyl, —OR 10 , —N(R 10 ) 2 , —NR 10 C(O)R 10 , —CONR 10 R 10 , —NR 10 SO 2 R 11 , —C 1-3 alkylene—OR 10 , C 3-6 cycloalkyl, and —C 1-3 alkylene—C 3-6 cycloalkyl;

R 1a is hydrogen, C 1-4 alkyl, C 1-4 haloalkyl, C 3-4 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl;

R 10 , at each occurrence, is independently hydrogen, C 1-4 alkyl, C 1-4 haloalkyl, C 3-4 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl, wherein alternatively two R 10 , together with a nitrogen to which the two R 10 attach form a 4-to 6-membered heterocyclic ring optionally substituted with 1-4 substituents independently selected from the group consisting of halogen and C 1-4 alkyl;

R 11 is C 1-4 alkyl, C 1-4 haloalkyl, C 3-4 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl;

R 2 , at each occurrence, is independently halogen, cyano, C 1-4 alkyl, C 1-4 haloalkyl, C 2-4 alkenyl, C 3-6 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl;

n is 0, 1, 2, 3, or 4;

R 3 is —L 1 —G 2 , G 2 , —L 2 —G 2 , —L 2 L 1 —G 2 , —C 2-6 alkylene—R 3a , or C 3-7 alkyl;

L 1 is C 1-3 alkylene;

L 2 is 1,1-cyclopropylene;

G 2 is a 4-to 12-membered heterocyclyl a 6-to 12-membered aryl, a 5-to 12-membered heteroaryl, or a C 3-12 carbocyclyl optionally fused to a 6-membered arene, wherein G 2 is optionally substituted with 1-5 substituents independently selected from the group consisting of halogen, cyano, C 1-4 alkyl, C 1-4 haloalkyl, —OR 13 , —N(R 13 ) 2 , —C 1-3 alkylene—OR 13 or —C 1-3 alkylene—N(R 13 ) 2 ;

R 3a is —OR 14 or —N(R 14 ) 2 ; and

R 13 and R 14 , at each occurrence, are independently hydrogen, C 1-4 alkyl, C 1-4 haloalkyl, C 3-4 cycloalkyl, or —C 1-3 alkylene—C 3-4 cycloalkyl, wherein alternatively two R 13 or two R 14 , together with a nitrogen to which the two R 13 or two R 14 attach form a 4-to 6-membered heterocyclic ring optionally substituted with 1-4 substituents independently selected from the group consisting of halogen and C 1-4 alkyl.

2. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein A is a thiophene.

3. The compound of claim 1 , of formula (I-A), (I-B), (I-C), or (I-D),

or a pharmaceutically acceptable salt thereof.

4. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein G 1 is the 5-to 12-membered heteroaryl.

5. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein G 1 is the 6-to 12-membered aryl.

6. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein G 1 is the 4-to 12-membered heterocyclyl.

7. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein G 1 is

8. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein L 1 is CH 2 .

9. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein G 2 is the 4-to 12-membered heterocyclyl.

10. The compound of claim 9 , or a pharmaceutically acceptable salt thereof, wherein G 2 is a tetrahydropyranyl, an oxetanyl, a tetrahydrothiopyranyl, a 2-oxaspiro[3.3]heptanyl, or a 3-oxaspiro[5.5]undecanyl.

11. The compound of claim 10 , or a pharmaceutically acceptable salt thereof, wherein G 2 is

12. The compound of claim 11 , or a pharmaceutically acceptable salt thereof, wherein G 2 is

13. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein R is hydrogen.

14. The compound of claim 1 selected from the group consisting of

7-chloro-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(2,5-difluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(5-fluoro-2-methylphenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(2,5-dimethylphenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl methyl)octahydrocyclopenta[c]pyrrol-5-yl)-7-(o-tolyl)thieno[2,3-d]pyridazin-4-amine;

7-(2-methoxyphenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(2-fluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-phenyl-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(2-methyl-2H-indazol-5-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

4-(2,5-difluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

4-(5-fluoro-2-methylphenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

4-chloro-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

4-(2-methyl-2H-indazol-5-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

4-phenyl-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

4-(2-fluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-7-amine;

N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-7-(2,3,5-trifluorophenyl)thieno[2,3-d]pyridazin-4-amine;

7-(2,4-dimethyl-2H-indazol-5-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(2,5-difluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl-d2)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(5-fluoro-2-methylphenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl-d2)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

7-(4,4-difluoropiperidin-1-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine; and

7-((S)-3-methylpiperidin-1-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)thieno[2,3-d]pyridazin-4-amine;

4-(4,4-difluoropiperidin-1-yl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-5,6,7,8-tetrahydrophthalazin-1-amine;

4-(2,5-difluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-5,6,7,8-tetrahydrophthalazin-1-amine;

4-(2-fluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-5,6,7,8-tetrahydrophthalazin-1-amine;

4-(3-fluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-5,6,7,8-tetrahydrophthalazin-1-amine;

4-(4-fluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl)octahydrocyclopenta[c]pyrrol-5-yl)-5,6,7,8-tetrahydrophthalazin-1-amine; and

4-(2,5-difluorophenyl)-N-((3aR,5s,6aS)-2-((tetrahydro-2H-pyran-4-yl)methyl-d2)octahydrocyclopenta[c]pyrrol-5-yl)phthalazin-1-amine;

or a pharmaceutically acceptable salt thereof.

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

16. A method for antagonizing mAChR M 4 in a subject, comprising administering to the subject a therapeutically effective amount of the compound of claim 1 , or a pharmaceutically acceptable salt thereof.

17. A method for treating a disorder in a subject, wherein the subject would benefit from antagonism of mAChR M 4 , comprising administering to the subject a therapeutically effective amount of the compound of claim 1 , or a pharmaceutically acceptable salt thereof.

18. The method of claim 17 , wherein the disorder is a neurodegenerative disorder, a movement disorder, or a brain disorder.

19. The method of claim 18 , wherein the disorder is a movement disorder.

20. A method for treating motor symptoms in a subject, comprising administering to a subject in need thereof a therapeutically effective amount of the compound of claim 1 , or a pharmaceutically acceptable salt thereof.

Assignments (2)
CONFIRMATORY LICENSE Recorded Jul 7, 2022
From: VANDERBILT UNIVERSITY
To: UNITED STATES GOVERNMENT
Reel/Frame 060613/0589 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 4, 2022
From: LINDSLEY, CRAIG W.; CONN, P. JEFFREY; BENDER, AARON M.; SPOCK, MATTHEW; HAN, CHANGHO
To: VANDERBILT UNIVERSITY
Reel/Frame 059487/0892 →
Continuity (2)
Provisional Application 62910863 · Oct 4, 2019
Related Publication 20240083907A1 · Mar 14, 2024
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