IP Library Granted Patent US 12698266
Granted Patent B2
US 12698266 · App. 17/786,607 · Granted Aug 4, 2026

TRPML modulators

Inventors: Jorge Garcia Fortanet (Wilmington, MA); Jeffrey Owen Saunders (Lincoln, MA); Harit Umesh Vora (North Andover, MA); Jian Lin (Acton, MA); Andrew Thomas Maynard (Winchester, MA); Erik Lee Meredith (Hudson, MA)
Assignee: Casma Therapeutics, Inc.
C07D401/04C07D211/14C07D213/83
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Quick Facts
Patent No.
US 12698266
App. No.
17/786,607
Granted
Aug 4, 2026
Kind
B2
Abstract

The present invention provides compounds, pharmaceutically acceptable compositions thereof, and methods of using the same.

Claims (102)

1 . A compound of Formula IIb-1 or IId:

or a pharmaceutically acceptable salt thereof, wherein

L 1 is —C(O)—NR 3 —, —NR 3 —C(O)—, or an optionally substituted bivalent moiety selected from C 1-6 alkylenyl, —NR 3 -C 0-6 alkylenyl, —C(O)—C 0-6 alkylenyl, —C 1-6 alkylenyl-C(O)—, and —C 1-6 alkylenyl-O—C(O)—;

Z is 4- to 16-membered monocyclic or polycyclic heterocyclyl comprising 1 to 4 heteroatoms selected from N, O, and S, wherein Z is substituted with (R 2 ) q ;

each R a is independently H, halo, —CN, or an optionally substituted group selected from C 1-6 aliphatic, —C 3-6 cycloaliphatic, and —O—C 1-6 aliphatic;

each R 2 is independently selected from halo, oxo, —CN, —OH, —O—R 2a , —C(O)—R 2a , —C(O)O—R 2ª , and

an optionally substituted group selected from —C 1-6 aliphatic, —C 6-12 aryl, and 5- to 12-membered monocyclic or bicyclic heteroaryl comprising 1 to 4 heteroatoms selected from N, O, and S;

each R 2a is independently H or an optionally substituted group selected from C 1-6 aliphatic, C 3-12 cycloaliphatic, and 4- to 12-membered monocyclic or bicyclic heterocyclyl comprising 1 to 4 heteroatoms selected from N, O, and S;

each R 3 is independently selected from H and optionally substituted-C 1-6 aliphatic;

each R 5 is —N(R 3 ) 2 or an optionally substituted group selected from —C 1-6 aliphatic, —P(O)(C 1-6 aliphatic) 2 , —C 3-12 cycloaliphatic, and 5- to 12-membered monocyclic or polycyclic heterocyclyl comprising 1 to 4 heteroatoms selected from N, O, and S;

each R 6 is independently selected from halo, —SF 5 , —S(O)—R 5 , S(O) 2 —R 5 , —S(O)(NH)—R 5 , —S(O) 2 —(NH)—R 5 , —S(O)—N(R 5 ) 2 , —S(O) 2 —N(R 5 ) 2 , —CN, —C(O)—NH(R 5 ), —C(O)—N(R 5 ) 2 , —P(O)(R 5 ) 2 , —O—R 5 , or an optionally substituted group selected from —C 1-6 aliphatic, −S—C 1-6 aliphatic, —C 0-6 alkylenyl —C(O)—R 5 , —C 0-6 alkylenyl-C(O)O—R 5 , —C 6-12 aryl, —C 3-12 cycloaliphatic, and 4- to 7-membered monocyclic heterocyclyl comprising 1 to 4 heteroatoms selected from N, O, and S;

m is 0, 1, 2, 3, or 4; and

q is 2.

2 . The compound of claim 1 , wherein each R 6 is independently halo, —S(O)—R 5 , —S(O) 2 —R 5 , —S(O)(NH)—R 5 , or an optionally substituted —O—C 1-6 aliphatic.

3 . The compound of claim 1 , wherein R 6 is —S(O)—R 5 , —S(O) 2 —R 5 , or optionally substituted —C 1-6 aliphatic.

4 . A pharmaceutical composition comprising a compound of claim 1 , and a pharmaceutically acceptable carrier, adjuvant, or vehicle.

5 . The compound of claim 1 , wherein the compound is selected from:

Structure

Compound No.

A-544

A-748

A-750

A-754

A-755

A-758

A-763

A-766

A-769

A-771

A-773

A-774

A-776

A-779

A-782

A-783

A-788

A-789

A-800

A-801

A-802

A-803

A-804

A-805

A-806

A-807

A-808

A-810

A-811

A-814

A-818

A-824

A-826

A-831

A-833

A-834

A-835

A-836

A-838

A-840

A-841

A-842

A-843

A-845

A-846

A-847

A-854

A-859

A-862

A-863

A-864

A-865

A-866

A-867

A-871

A-871A

A-871B

A-872

A-872A

A-872B

A-878

A-883

A-885

A-902

A-903

A-931

A-985

A-997

A-998

A-1001

or a pharmaceutically acceptable salt thereof.

6 . The compound of claim 1 , wherein Z is selected from:

7 . The compound of claim 1 , wherein m is 1.

8 . The compound of claim 3 , wherein R 6 is —S(O) 2 —R 5 .

9 . The compound of claim 1 , wherein L 1 is C 1-6 alkylenyl.

10 . The compound of claim 1 , wherein Z is 4- to 6-membered monocyclic heterocyclyl comprising 1 to 4 heteroatoms selected from N, O, and S.

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

12 . The compound of claim 1 , wherein each R 2 is —C 1-6 aliphatic.

13 . The compound of claim 1 , wherein R 5 is —N(R 3 ) 2 .

14 . The compound of claim 1 , wherein each R 3 is —C 1-6 aliphatic.

15 . A compound having the structure:

or a pharmaceutically acceptable salt thereof.

16 . A compound having the structure: