# US 2022/0033366 A1 · Compounds useful for treatment or prevention of muscular dystrophy and derivatives for treatment, amelioration or prevention of muscular dystrophy in medicinal use thereof

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- Number: US 2022/0033366 A1
- Document ID: US20220033366A1
- Status: Published application
- Kind: A1
- Application number: 17/413,608
- Filed: 2019-12-24
- Published: 2022-02-03
- Earliest claimed: 2018-12-27
- Assignees: J2H Biotech Inc.
- Applicants: J2H Biotech Inc.
- Inventors: Hyung-Chul RYU; Jae-Sun KIM; Jee-Woong LIM; Ju-Young LEE; Wenling SONG; Tae-Gon KIM
- Web page: https://patentgrep.com/patent/US20220033366A1

## CPC

- C07D 271/06: 1,2,4-Oxadiazoles; Hydrogenated 1,2,4-oxadiazoles
- A61K 31/4245: Oxadiazoles
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- 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

The present invention relates to a novel compound and a medicinal use of the novel compound. In particular, the present invention relates to a novel compound useful for the treatment and/or prevention of muscular dystrophy, including Duchenne muscular atrophy, and a medicinal use of the novel compound.

## Claims

### Claim 1

A compound represented by the following Chemical formula 1.
In Chemical Formula 1,
L is selected from the group consisting of straight or branched alkyl having 1 to 3 carbon atoms and benzyl.

### Claim 2 (depends on 1)

The compound according to claim 1, wherein the compound is
1-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1 ,2,4-oxadiazol-3-yl)benzoate;
(3-hydroxy-3-methylbutanoyloxy)methyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate;
2-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate; or
4-(3-hydroxy-3-methylbutanoyloxy)benzyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate.

### Claim 3 (depends on 1)

A pharmaceutical composition, comprising the compound of claim 1 as an active ingredient.

### Claim at position 4

-5. (canceled)

### Claim 6 (depends on 3)

The pharmaceutical composition according to claim 3, wherein the compound is
1-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate;
(3-hydroxy-3-methylbutanoyloxy)methyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate;
2-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate; or
4-(3-hydroxy-3-methylbutanoyloxy)benzyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate.

### Claim 7 (depends on 1)

A method for treating muscular dystrophy, comprising:
administering to a subject in need thereof a therapeutically effective amount of the compound of claim 1.

### Claim 8 (depends on 7)

The method according to claim 7, wherein the muscular dystrophy is progressive muscular dystrophy.

### Claim 9 (depends on 7)

The method according to claim 7, wherein the muscular dystrophy is Duchenne muscular atrophy.

### Claim 10 (depends on 7)

The method according to claim 7, wherein the compound is
1-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate;
(3-hydroxy-3-methylbutanoyloxy)methyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate;
2-(3-hydroxy-3-methylbutanoyloxy)ethyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate; or
4-(3-hydroxy-3-methylbutanoyloxy)benzyl 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoate.