# US 11,999,729 B2 · Small molecule inhibition of transcription factor SALL4 and uses thereof

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- Number: US 11,999,729 B2
- Document ID: US11999729B2
- Status: Granted
- Kind: B2
- Application number: 17/978,591
- Filed: 2022-11-01
- Published: 2023-04-13
- Granted: 2024-06-04
- Earliest claimed: 2017-10-04
- Assignees: Dana-Farber Cancer Institute, Inc.; The Brigham and Women's Hospital, Inc.
- Applicants: Dana-Farber Cancer Institute, Inc.; The Brigham and Women's Hospital, Inc.
- Inventors: Jun Qi; Anthony Varca; Li Chai
- Web page: https://patentgrep.com/patent/US11999729B2

## CPC

- C07D 471/04: Ortho-condensed systems
- A61K 31/437: the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
- A61K 31/4985: Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
- A61P 35/00: Antineoplastic agents
- C07D 487/04: Ortho-condensed systems
- C07D 519/00: Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Provided herein are compounds that interrupt the function of SALL4. Also described are pharmaceutical compositions and medical uses of these compounds.

## Claims

### Claim 1

A compound having a structure of Formula I or a pharmaceutically acceptable salt thereof:
wherein
Z is N;
R1 is optionally substituted C5 or C6-cycloalkyl, optionally substituted heterocyclyl, naphthalenyl substituted with one or more groups selected from hydroxyl and nitro,
optionally substituted oxazolyl, optionally substituted benzoisoxazolyl, optionally substituted indazolyl, optionally substituted azaindolyl, optionally substituted benzothiazolyl, optionally substituted thiazolyl, or optionally substituted carbazolyl; and
R2 is optionally substituted alkyl, optionally substituted cycloalkyl, optionally substituted heterocyclyl, optionally substituted aryl, or optionally substituted heteroaryl.

### Claim 2 (depends on 1)

The compound of claim 1, wherein R1 is naphthalenyl substituted with one or more groups selected from hydroxyl and nitro.

### Claim 3 (depends on 2)

The compound of claim 2, wherein R1 is selected from

### Claim 4 (depends on 1)

The compound of claim 1, wherein R1 is selected from

### Claim 5 (depends on 1)

The compound of claim 1, wherein R1 is optionally substituted C5 or C6-cycloalkyl or optionally substituted heterocyclyl.

### Claim 6 (depends on 5)

The compound of claim 5, wherein R1 is selected from

### Claim 7 (depends on 1)

The compound of claim 1, wherein R1 is optionally substituted oxazolyl, optionally substituted benzoisoxazolyl, optionally substituted indazolyl, optionally substituted azaindolyl, optionally substituted benzothiazolyl, optionally substituted thiazolyl, and optionally substituted carbazolyl.

### Claim 8 (depends on 7)

The compound of claim 7, wherein R1 is substituted with one or more groups selected from halo, oxy, nitro, sulfonate, and optionally substituted alkyl.

### Claim 9 (depends on 8)

The compound of claim 8, wherein R1 is selected from

### Claim 10 (depends on 1)

The compound of claim 1, wherein R2 is optionally substituted alkyl, optionally substituted cycloalkyl, or optionally substituted aryl.

### Claim 11 (depends on 10)

The compound of claim 10, wherein the alkyl is substituted with alkoxy, amino or optionally substituted aryl.

### Claim 12 (depends on 10)

The compound of claim 10, wherein R2 is selected from

### Claim 13 (depends on 10)

The compound of claim 10, wherein R2 is optionally substituted phenyl.

### Claim 14 (depends on 13)

The compound of claim 13, wherein the phenyl is substituted with one more groups selected from halo, hydroxyl, cyano, optionally substituted alkyl, optionally substituted alkoxy, carboxy, and ester.

### Claim 15 (depends on 14)

The compound of claim 14, wherein R2 is selected from

### Claim 16 (depends on 1)

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

### Claim 17 (depends on 1)

A method of treating cancer, wherein the cancer is acute myeloid leukemia, liver cancer, lung cancer, or myelodysplastic syndromes (MDS) comprising administering to a subject the compound or pharmaceutically acceptable salt thereof of claim 1.

### Claim 18

A compound which is
or a pharmaceutically acceptable salt thereof.

### Claim 19 (depends on 17)

The method of claim 17, wherein the cancer is acute myeloid leukemia.

### Claim 20 (depends on 17)

The method of claim 17, wherein the cancer is liver cancer.

### Claim 21 (depends on 17)

The method of claim 17, wherein the cancer is lung cancer.

### Claim 22 (depends on 17)

The method of claim 17, wherein the cancer is myelodysplastic syndrome (MDS).