IP Library Granted Patent US 10,632,103
Granted Patent B2
US 10,632,103 · App. 15/881,323 · Granted Apr 28, 2020

Arginine methyltransferase inhibitors and uses thereof

Inventors: Richard Chesworth (Concord, MA); Lorna Helen Mitchell (Cambridge, MA); Gideon Shapiro (Gainesville, FL)
Assignee: Epizyme, Inc.
A61K31/415A61K31/4155A61K31/454A61K31/5377C07D231/10C07D231/12C07D401/02C07D401/04C07D405/04C07D413/02C07D413/08C12N9/1007C12Y201/01125
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Quick Facts
Patent No.
US 10,632,103
App. No.
15/881,323
Granted
Apr 28, 2020
Kind
B2
Abstract

Described herein are compounds of Formula (I), pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof. Compounds described herein are useful for inhibiting arginine methyltransferase activity. Methods of using the compounds for treating arginine methyltransferase-mediated disorders are also described.

Claims (37)

1. A compound of Formula (I):

or a pharmaceutically acceptable salt thereof,

wherein

X is N, Z is NR 4 , and Y is CR 5 ; or

X is NR 4 , Z is N, and Y is CR 5 ; or

X is CR 5 , Z is NR 4 , and Y is N; or

X is CR 5 , Z is N, and Y is NR 4 ;

L 1 is a bond, —O—, —N(R B )—, —S—, —C(O)—, —C(O)O—, —C(O)S—, —C(O)N(R B )—, —C(O)N(R B )N(R B )—, —OC(O)—, —OC(O)N(R B )—, —NR B C(O)—, —NR B C(O)N(R B )—, —NR B C(O)N(R B )N(R B )—, —NR B C(O)O—, —SC(O)—, —C(═NR B )—, —C(═NNR B )—, —C(═NOR A )—, —C(═NR B )N(R B )—, —NR B C(═NR B )—, —C(S)—, —C(S)N(R B )—, —NR B C(S)—, —S(O)—, —OS(O) 2 —, —S(O) 2 —, —SO 2 —, —N(R B )SO 2 —, —SO 2 N(R B )—, or an optionally substituted C 1-6 saturated or unsaturated hydrocarbon chain, wherein one or more methylene units of the hydrocarbon chain is optionally and independently replaced with —O—, —N(R B )—, —S—, —C(O)—, —C(O)O—, —C(O)S—, —C(O)N(R B )—, —C(O)N(R B )N(R B )—, —OC(O)—, —OC(O)N(R B )—, —NR B C(O)—, —NR B C(O)N(R B )—, —NR B C(O)N(R B )N(R B )—, —NR B C(O)O—, —SC(O)—, —C(═NR B )—, —C(═NNR B )—, —C(═NOR A )—, —C(═NR B )N(R B )—, —NR B C(═NR B )—, —C(S)—, —C(S)N(R B )—, —NR B C(S)—, —S(O)—, —OS(O) 2 —, —S(O) 2 —, —SO 2 —, —N(R B )SO 2 —, or —SO 2 N(R B )—;

each R A is independently selected from the group consisting of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted carbocyclyl, optionally substituted heterocyclyl, optionally substituted aryl, optionally substituted heteroaryl, an oxygen protecting group when attached to an oxygen atom, and a sulfur protecting group when attached to a sulfur atom;

each R B is independently selected from the group consisting of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted carbocyclyl, optionally substituted heterocyclyl, optionally substituted aryl, optionally substituted heteroaryl, and a nitrogen protecting group, or an R B and R W on the same nitrogen atom may be taken together with the intervening nitrogen to form an optionally substituted heterocyclic ring;

R x is optionally substituted C 1-4 alkyl or optionally substituted C 3-4 cycloalkyl;

R W is hydrogen, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; provided that when L 1 is a bond, R W is not hydrogen;

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

R 4 is hydrogen, substituted C 1 alkyl, optionally substituted C 2-6 alkyl, optionally substituted C 2-6 alkenyl, optionally substituted C 2-6 alkynyl, optionally substituted C 3-7 cycloalkyl, optionally substituted 4- to 7-membered heterocyclyl; or optionally substituted C 1-4 alkyl-Cy;

Cy is optionally substituted C 3-7 cycloalkyl, optionally substituted 4- to 7-membered heterocyclyl, optionally substituted aryl, or optionally substituted heteroaryl; and

R 5 is hydrogen, halo, —CN, optionally substituted C 1-4 alkyl, or optionally substituted C 3-4 cycloalkyl.

2. The compound of claim 1 , wherein the compound is of Formula (II), (III), (IV), or (V):

or a pharmaceutically acceptable salt thereof.

3. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein -L 1 -R W is optionally substituted alkyl.

4. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein R 3 is hydrogen, C 1-4 alkyl, cyclopropyl, or cyclobutyl.

5. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein R 4 is hydrogen.

6. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein R 5 is hydrogen or optionally substituted C 1-4 alkyl.

7. The compound of claim 1 , or a pharmaceutically acceptable salt thereof, wherein R 4 is hydrogen, optionally substituted C 2-6 alkenyl, optionally substituted C 2-6 alkynyl, optionally substituted C 3-7 cycloalkyl, optionally substituted 4- to 7-membered heterocyclyl; or optionally substituted C 1-4 alkyl-Cy.

8. The compound of claim 1 , wherein the compound is selected from the group consisting of:

or pharmaceutically acceptable salts thereof.

9. A pharmaceutical composition comprising a compound of claim 1 or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient.

10. A kit or packaged pharmaceutical comprising a compound of claim 1 or a pharmaceutically acceptable salt thereof, and instructions for use thereof.

11. A method of inhibiting an arginine methyl transferase (RMT) comprising contacting a cell with an effective amount of a compound of claim 1 or a pharmaceutically acceptable salt thereof.

12. The method of claim 11 , wherein the arginine methyl transferase is PRMT1, PRMT6, PRMT3, PRMT8, or CARM1.

13. A method of modulating gene expression comprising contacting a cell with an effective amount of a compound of claim 1 or a pharmaceutically acceptable salt thereof.

14. A method of modulating transcription comprising contacting a cell with an effective amount of a compound claim 1 or a pharmaceutically acceptable salt thereof.

15. A method of treating a RMT-mediated disorder, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of claim 1 , or a pharmaceutically acceptable salt thereof.

16. The method of claim 15 , wherein the RMT-mediated disorder is a PRMT1-mediated disorder, a PRMT6-mediated disorder, a PRMT3-mediated disorder, a PRMT8-mediated disorder, or a CARM1-mediated disorder.

17. The method of claim 15 , wherein the disorder is a proliferative disorder, a neurological disorder, a muscular dystrophy, an autoimmune disorder, a vascular disorder, or a metabolic disorder.

18. The method of claim 17 , wherein the disorder is cancer.

19. The method of claim 17 , wherein the disorder is amyotrophic lateral sclerosis.

20. The method of claim 17 , wherein the disorder is selected from the group consisting of breast cancer, prostate cancer, lung cancer, colon cancer, bladder cancer, lymphoma, leukemia, diabetes mellitus, kidney failure, coronary heart disease, oculopharyngeal muscular dystrophy, and amyotrophic lateral sclerosis.

Assignments (3)
TERMINATION AND RELEASE OF SECURITY INTEREST IN PATENTS AT REEL/FRAME: 051057/0848 Recorded Aug 13, 2022
From: BIOPHARMA CREDIT PLC
To: EPIZYME, INC.
Reel/Frame 061165/0501 →
SECURITY INTEREST Recorded Nov 19, 2019
From: EPIZYME, INC.
To: BIOPHARMA CREDIT PLC
Reel/Frame 051057/0848 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 5, 2019
From: CHESWORTH, RICHARD; MITCHELL, LORNA HELEN; SHAPIRO, GIDEON
To: EPIZYME, INC.
Reel/Frame 048501/0052 →
Continuity (5)
Continuation 15622213 · Jun 14, 2017
Continuation 14808290 · Jul 24, 2015
Continuation 14212543 · Mar 14, 2014
Provisional Application 61781051 · Mar 14, 2013
Related Publication 20180289671A1 · Oct 11, 2018