# US 11,312,689 B2 · Compounds and their use for the treatment of alpha1-antitrypsin deficiency

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- Number: US 11,312,689 B2
- Document ID: US11312689B2
- Status: Granted
- Kind: B2
- Application number: 17/345,928
- Filed: 2021-06-11
- Published: 2021-10-28
- Granted: 2022-04-26
- Earliest claimed: 2018-12-14
- Assignees: Z Factor Limited
- Applicants: Z Factor Limited
- Inventors: Nigel Ramsden; David John Fox; James Andrew Huntington
- Web page: https://patentgrep.com/patent/US11312689B2

## CPC

- C07D 239/36: as doubly bound oxygen atom or as unsubstituted hydroxy radical
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61K 31/513: having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- A61P 11/06: Antiasthmatics
- A61P 11/08: Bronchodilators
- C07B 2200/05: Isotopically modified compounds, e.g. labelled
- 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
- C07B: GENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR (preparation of carboxylic acid esters by telomerisation C07C67/47; telomerisation C08F)

## Abstract

The invention relates to oxopyrimidinyl-methyl-benzamide derivatives, for example in a pharmaceutically acceptable salt form or crystal form, pharmaceutical compositions comprising the derivatives, and their medical use, in particular for use in the treatment of α1-antitrypsin deficiency (A1AD or AATD).

## Claims

### Claim 1

A compound represented by the structure of:
or a pharmaceutically acceptable salt of any one thereof.

### Claim 2 (depends on 1)

The compound of claim 1, wherein the structure is represented by:
or pharmaceutically acceptable salt thereof.

### Claim 3 (depends on 1)

The compound of claim 1, wherein the structure is represented by:
or pharmaceutically acceptable salt thereof.

### Claim 4 (depends on 1)

The compound of claim 1, wherein the structure is represented by:
or pharmaceutically acceptable salt thereof.

### Claim 5 (depends on 1)

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

### Claim 6 (depends on 2)

A pharmaceutical composition comprising a compound or pharmaceutically acceptable salt of claim 2 and a pharmaceutically acceptable excipient.

### Claim 7 (depends on 3)

A pharmaceutical composition comprising a compound or pharmaceutically acceptable salt of claim 3 and a pharmaceutically acceptable excipient.

### Claim 8 (depends on 4)

A pharmaceutical composition comprising a compound or pharmaceutically acceptable salt of claim 4 and a pharmaceutically acceptable excipient.