# US 2021/0275526 A1 · Methods for treating huntington's disease

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- Number: US 2021/0275526 A1
- Document ID: US20210275526A1
- Status: Published application
- Kind: A1
- Application number: 17/093,194
- Filed: 2020-11-09
- Published: 2021-09-09
- Earliest claimed: 2015-12-10
- Assignees: PTC Therapeutics, Inc.
- Applicants: PTC Therapeutics, Inc.
- Inventors: Suresh Babu; Anuradha Bhattacharyya; Seongwoo Hwang; Minakshi Jani; Young-choon Moon; Nadiya Sydorenko
- Web page: https://patentgrep.com/patent/US20210275526A1

## CPC

- A61K 31/506: not condensed and containing further heterocyclic rings
- A61K 31/415: 1,2-Diazoles
- A61K 31/433: Thidiazoles
- A61K 31/4439: containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole (nicotine A61K31/465)
- A61K 31/4709: Non-condensed quinolines and containing further heterocyclic rings
- A61K 31/50: Pyridazines; Hydrogenated pyridazines
- A61K 31/501: not condensed and containing further heterocyclic rings
- A61K 31/517: ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
- A61K 31/5377: not condensed and containing further heterocyclic rings, e.g. timolol
- A61K 31/5513: 1,4-Benzodiazepines, e.g. diazepam {or clozapine}
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- C07D 237/20: Nitrogen atoms (nitro radicals C07D237/12)
- C07D 401/04: directly linked by a ring-member-to-ring-member bond
- C07D 401/12: linked by a chain containing hetero atoms as chain links
- C07D 401/14: containing three or more hetero rings
- C07D 403/10: linked by a carbon chain containing aromatic rings
- C07D 403/12: linked by a chain containing hetero atoms as chain links
- C07D 403/14: containing three or more hetero rings
- C07D 405/12: linked by a chain containing hetero atoms as chain links
- C07D 405/14: containing three or more hetero rings
- C07D 409/14: containing three or more hetero rings
- C07D 413/14: containing three or more hetero rings
- C07D 417/04: directly linked by a ring-member-to-ring-member bond
- C07D 417/10: linked by a carbon chain containing aromatic rings
- C07D 417/12: linked by a chain containing hetero atoms as chain links
- C07D 417/14: containing three or more hetero rings
- C07D 471/08: Bridged systems
- C07D 471/10: Spiro-condensed systems
- C07D 487/04: Ortho-condensed systems
- C07D 487/08: Bridged systems
- C07D 487/10: Spiro-condensed systems
- G01N 33/6896: {Neurological disorders, e.g. Alzheimer's disease}
- G01N 2800/2835: Movement disorders, e.g. Parkinson, Huntington, Tourette
- 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
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

## Claims

### Claim at position 1

1-3. (canceled)

### Claim 4

A compound of Formula (Ib1):
or a form thereof, wherein (when present),
A is selected from the group consisting of phenyl, thiophenyl, indazolyl, pyridinyl, pyrimidinyl, and phenoxy,
wherein phenyl and phenoxy are each optionally substituted with 1, 2 or 3 substituents each selected from R1a,
wherein thiophenyl, indazolyl, pyridinyl, pyrimidinyl are each optionally substituted with 1 or 2 substituents each selected from R1a,
R1a is selected from the group consisting of halogen, hydroxyl, C1-4alkyl, halo-C1-4alkyl, amino, C1-4alkoxy, and heteroaryl,
wherein heteroaryl is a saturated monocyclic or bicyclic ring system having 1, 2, or 3 heteroatom ring members selected from N, O, and S optionally substituted with 1 or 2 substituents each selected from R3a; and
R3a is selected from the group consisting of nitro and C1-4alkyl;
wherein a form of the compound is selected from the group consisting of a prodrug, salt, hydrate, solvate, clathrate, isotopologue, racemate, enantiomer, diastereomer, stereoisomer, polymorph and tautomer form thereof.

### Claim 5 (depends on 4)

The compound of claim 4 wherein (when present),
R1a is selected from the group consisting of fluoro, chloro, hydroxyl, methyl, difluoromethyl, amino, methoxy, 1H-pyrazolyl, and 1H-imidazol-1-yl,
wherein 1H-pyrazolyl is optionally substituted with 1 or 2 substituents each selected from R3a; and
R3a is selected from the group consisting of nitro, methyl, and amino;
wherein a form of the compound is selected from the group consisting of a prodrug, salt, hydrate, solvate, clathrate, isotopologue, racemate, enantiomer, diastereomer, stereoisomer, polymorph and tautomer form thereof.

### Claim 6

(canceled)

### Claim 7

A compound selected from the group consisting of:
N,2,2,6,6-pentamethyl-N-{5-[3-(1H-pyrazol-4-yl)phenoxy]-1,3,4-thiadiazol-2-yl}piperidin-4-amine, and
N,2,2,6,6-pentamethyl-N-{5-[4-(1H-pyrazol-4-yl)phenoxy]-1,3,4-thiadiazol-2-yl}piperidin-4-amine;
wherein a form of the compound is selected from the group consisting of a prodrug, salt, hydrate, solvate, clathrate, isotopologue, racemate, enantiomer, diastereomer, stereoisomer, polymorph and tautomer form thereof.

### Claim 8

(canceled)

### Claim 9

A compound salt selected from the group consisting of:
N,2,2,6,6-pentamethyl-N-{5-[3-(1H-pyrazol-4-yl)phenoxy]-1,3,4-thiadiazol-2-yl}piperidin-4-amine hydrochloride, and
N,2,2,6,6-pentamethyl-N-{5-[4-(1H-pyrazol-4-yl)phenoxy]-1,3,4-thiadiazol-2-yl}piperidin-4-amine hydrochloride;
wherein a form of the compound salt is selected from the group consisting of a prodrug, hydrate, solvate, clathrate, isotopologue, racemate, enantiomer, diastereomer, stereoisomer, polymorph and tautomer form thereof.

### Claim at position 8

10-11. (canceled)

### Claim 12 (depends on 19)

The method of claim 19, wherein the effective amount of the compound, or form thereof, is in a range of from about 0.001 mg/kg/day to about 500 mg/kg/day.

### Claim 13

(canceled)

### Claim 14 (depends on 20)

The method of claim 20, wherein the effective amount of the compound, or form thereof, is in a range of from about 0.001 mg/kg/day to about 500 mg/kg/day.

### Claim 15

(canceled)

### Claim 16 (depends on 21)

The method of claim 21, wherein the effective amount of the compound, or form thereof, is in a range of from about 0.001 mg/kg/day to about 500 mg/kg/day.

### Claim 17 (depends on 4)

A pharmaceutical composition comprising the compound of claim 4, or form thereof, in admixture with one or more pharmaceutically acceptable excipient(s).

### Claim 18

(canceled)

### Claim 19 (depends on 4)

A method for treating or ameliorating Huntington's Disease in a subject in need thereof comprising administering to the subject an effective amount of the compound, or form thereof, of claim 4.

### Claim 20 (depends on 7)

A method for treating or ameliorating Huntington's Disease in a subject in need thereof comprising administering to the subject an effective amount of the compound, or form thereof, of claim 7.

### Claim 21 (depends on 9)

A method for treating or ameliorating Huntington's Disease in a subject in need thereof comprising administering to the subject an effective amount of the compound, or form thereof, of claim 9.

### Claim 22 (depends on 7)

A pharmaceutical composition comprising the compound of claim 7, or form thereof, in admixture with one or more pharmaceutically acceptable excipient(s).

### Claim 23 (depends on 9)

A pharmaceutical composition comprising the compound of claim 9, or form thereof, in admixture with one or more pharmaceutically acceptable excipient(s).