# US 2019/0167640 A1 · Methods for treatment of muscular dystrophies

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- Number: US 2019/0167640 A1
- Document ID: US20190167640A1
- Status: Published application
- Kind: A1
- Application number: 16/169,019
- Filed: 2018-10-24
- Published: 2019-06-06
- Earliest claimed: 2013-10-25
- Assignees: Akashi Therapeutics, Inc.
- Applicants: Akashi Therapeutics, Inc.
- Inventors: Ernest D. Bush; Francois Nique; Catherine Jagerschmidt; Florence Sylvie Namour; Roland Blanque; Jean-Michel Lefrancois; Christophe Peixoto; Pierre Deprez; Nicholas Triballeau; Piet Tom Burt Paul Wigerinck
- Web page: https://patentgrep.com/patent/US20190167640A1

## CPC

- A61K 31/4166: having oxo groups directly attached to the heterocyclic ring, e.g. phenytoin
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61P 3/02: Nutrients, e.g. vitamins, minerals
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 19/08: for bone diseases, e.g. rachitism, Paget's disease
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 21/04: for myasthenia gravis
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 29/00: Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
- 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, inter alia, treatment of muscle dystrophy (e.g., Duchenne Muscular Dystrophy), for example, using a composition, e.g., a composition comprising Compound (I), or a pharmaceutically acceptable salt, prodrug or metabolite thereof.

## Claims

### Claim 1

A method of treating muscular dystrophy in a subject, the method comprising administering to a subject suffering from muscular dystrophy a therapeutically effective amount of Compound (I) or a pharmaceutically acceptable salt thereof, or a therapeutically effective amount of Compound (II) or a pharmaceutically acceptable salt thereof.
thereby treating the subject.

### Claim 2 (depends on 1)

The method of claim 1, wherein the muscular dystrophy is selected from the group consisting of: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Emery-Dreifuss Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy, Oculopharyngeal Muscular Dystrophy, Distal Muscular Dystrophy, and congenital muscular dystrophy.

### Claim 3 (depends on 2)

The method of claim 2, wherein the muscular dystrophy is Duchenne Muscular Dystrophy.

### Claim at position 4

4-7. (canceled)

### Claim 8 (depends on 1)

The method of claim 1, wherein Compound (I) or pharmaceutically acceptable salt thereof, or Compound (II) or pharmaceutically acceptable salt thereof, is administered in multiple doses, e.g., at a predetermined interval.

### Claim at position 6

9-17. (canceled)

### Claim 18 (depends on 1)

The method of claim 1, wherein the therapeutically effective amount of Compound (I) or pharmaceutically acceptable salt thereof, or the therapeutically effective amount of Compound (II) or pharmaceutically acceptable salt thereof, is administered at a dose of about 0.2 mg to about 0.8 mg, about 0.3 mg to about 0.7 mg, or about 0.4 mg to about 0.6 mg.

### Claim 19 (depends on 1)

The method of claim 1, wherein Compound (I) or pharmaceutically acceptable salt thereof, or Compound (II) or pharmaceutically acceptable salt thereof, is administered at a dose of about 2 μg to about 1000 μg per kilogram subject weight.

### Claim at position 9

20-25. (canceled)

### Claim 26 (depends on 1)

The method of claim 1, wherein Compound (I) or pharmaceutically acceptable salt thereof, or Compound (II) or pharmaceutically acceptable salt thereof, is administered after meal consumption.

### Claim at position 11

27-29. (canceled)

### Claim 30 (depends on 1)

The method of claim 1, wherein Compound (I) or pharmaceutically acceptable salt thereof, or Compound (II) or pharmaceutically acceptable salt thereof, is administered before meal consumption.

### Claim at position 13

31-33. (canceled)

### Claim 34 (depends on 1)

The method of claim 1, comprising administering to the subject the therapeutically effective amount of Compound (II) or pharmaceutically acceptable salt thereof,

### Claim 35

(canceled)

### Claim 36 (depends on 1)

The method of claim 1, wherein Compound (I) or pharmaceutically acceptable salt thereof, or Compound (II) or pharmaceutically acceptable salt thereof, is administered via oral administration.

### Claim 37 (depends on 1)

The method of claim 1, wherein subject is human.

### Claim 38 (depends on 37)

The method of claim 37, wherein the subject is male.

### Claim 39 (depends on 38)

The method of claim 38, wherein the subject is pediatric.

### Claim 40

(canceled)

### Claim 41 (depends on 38)

The method of claim 38, wherein the subject is from the age of about 1 year to about 18 years.

### Claim at position 22

42-43. (canceled)

### Claim 44 (depends on 1)

The method of claim 1, wherein Compound (I) or Compound (II) is in at least 95% enantiomeric excess, in at least 98% enantiomeric excess, or in at least 99% enantiomeric excess.

### Claim at position 24

45-46. (canceled)

### Claim 47 (depends on 1)

The method of claim 1, wherein levels of testosterone in the subject after treatment are not substantially changed as compared to the levels of testosterone in the subject before treatment.

### Claim 48 (depends on 1)

The method of claim 1, wherein method is substantially free of side effects selected from obesity, behavior problems, thinner and/or weaker bones (osteoporosis); delayed puberty, stomach problems, cataracts, sensitivity to infections;
hypogonadism, muscle wasting and osteoporosis; cardiovascular risk; prostate cancer risks, hypogondism, and conditions pertaining to hormonal imbalances.

### Claim 49

(canceled)

### Claim 50

A pharmaceutical composition comprising Compound (I) or a pharmaceutically acceptable salt thereof, or comprising Compound (II) or a pharmaceutically acceptable salt thereof,
wherein the pharmaceutical composition comprises about 0.1 mg to about 1 mg of the Compound (I) or pharmaceutically acceptable salt thereof, or comprises about 0.1 mg to about 1 mg of the Compound (II) or pharmaceutically acceptable salt thereof.

### Claim at position 29

51-56. (canceled)

### Claim 57 (depends on 1)

The method of claim 1, further comprising administering an additional therapeutic agent.

### Claim at position 31

58-62. (canceled)

### Claim 63

A method of treating muscular dystrophy in a subject, the method comprising:
(a) determining whether a subject suffers from or is susceptible to muscular dystrophy;
(b) selecting the subject for treatment based on the determining;
(c) administering a therapeutically effective amount of Compound (I) or a pharmaceutically acceptable salt thereof, or a therapeutically effective amount of Compound (II) or a pharmaceutically acceptable salt thereof, thereby treating muscular dystrophy in the subject.

### Claim at position 33

64-66. (canceled)