# US 10,245,268 B2 · Treatment of ACVR1-mediated diseases

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- Number: US 10,245,268 B2
- Document ID: US10245268B2
- Status: Granted
- Kind: B2
- Application number: 15/671,978
- Filed: 2017-08-08
- Published: 2018-02-15
- Granted: 2019-04-02
- Earliest claimed: 2016-08-10
- Assignees: Sierra Oncology, Inc.
- Applicants: Sierra Oncology, Inc.
- Inventors: Brian Koh; Igor Theurl; Matthew Robert Warr; James Andrew Whitney
- Web page: https://patentgrep.com/patent/US10245268B2

## CPC

- A61K 31/5377: not condensed and containing further heterocyclic rings, e.g. timolol
- A61K 31/7064: containing condensed or non-condensed pyrimidines
- A61P 35/00: Antineoplastic agents
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- 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 methods, compositions, and kits for treating ACVR-1-mediated diseases using N-(cyanomethyl)-4-[2-4-morpholinoanilino)pyrimidin-4-yl]benzamide.

## Claims

### Claim 1

A method of treating an ACVR1-mediated disease comprising administering momelotinib or a pharmaceutically acceptable salt or solvate thereof to a patient in need thereof, wherein the ACVR1-mediated disease is selected from the group consisting of fibrodysplasia ossificans progressive (FOP) and diffuse intrapontine glioma (DIPG).

### Claim 2 (depends on 1)

The method of claim 1, wherein the ACVR1-mediated disease is mediated by a mutant ACVR1 polypeptide.

### Claim 3 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a substitution mutation.

### Claim 4 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises an activating mutation in the glycine-serine rich (GS) domain or in the kinase domain.

### Claim 5 (depends on 4)

The method of claim 4, wherein the activating mutation is in the GS domain.

### Claim 6 (depends on 5)

The method of claim 5, wherein the activating mutation is an R206H substitution.

### Claim 7 (depends on 4)

The method of claim 4, wherein the activating mutation is in the kinase domain.

### Claim 8 (depends on 7)

The method of claim 7, wherein the activating mutation is a R258G/S, G328E/V/W/R, or G356D substitution.

### Claim 9 (depends on 8)

The method of claim 8, wherein the activating mutation is a R258G/S substitution.

### Claim 10 (depends on 8)

The method of claim 8, wherein the activating mutation is a G328EN/W/R substitution.

### Claim 11 (depends on 8)

The method of claim 8, wherein the activating mutation is a G356D substitution.

### Claim 12 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a R206H, Q207E, R258G/S, G328E/V/W/R, or G356D mutation.

### Claim 13 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a R206H mutation.

### Claim 14 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a Q207E mutation.

### Claim 15 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a R258G/S mutation.

### Claim 16 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a G328E/V/W/R mutation.

### Claim 17 (depends on 2)

The method of claim 2, wherein the mutant ACVR1 polypeptide comprises a G356D mutation.

### Claim 18 (depends on 1)

The method of claim 1, wherein the ACVR1-mediated disease is not anemia.