Compositions and methods for treating charcot-marie-tooth disease

(2S)-2-[4-bromo-2-(1,2-azol-3-yl)phenoxy]propionic acid addresses the inadequacies of existing CMTD treatments by effectively managing and potentially halting the progression of CMTD symptoms.

HK40134677APending Publication Date: 2026-07-10NMD PHARMA AS
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Patent Information

Authority / Receiving Office
HK · HK
Patent Type
Applications
Current Assignee / Owner
NMD PHARMA AS
Filing Date
2026-04-16
Publication Date
2026-07-10

AI Technical Summary

Technical Problem

Current treatments for Charcot-Marie-Tooth disease (CMTD) are inadequate in effectively managing the symptoms and progression of the disease.

Method used

Administration of (2S)-2-[4-bromo-2-(1,2-azol-3-yl)phenoxy]propionic acid or its pharmaceutical compositions to patients with CMTD symptoms.

Benefits of technology

The compound effectively manages and potentially halts the progression of CMTD symptoms, providing a therapeutic benefit.

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Abstract

Disclosed herein are methods of treatment for Charcot–Marie–Tooth disease (CMTD) that involve administering (2S)-2-[4-bromo-2-(1,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutical composition thereof, to a patient suffering from symptoms of CMTD. Pharmaceutical compositions and kits-to-parts including (2S)-2-[4-bromo-2-5 (1,2-oxazol-3-yl)phenoxy]propanoic acid are also disclosed.
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Description

This article discloses a method for treating Charcot-Marie-Toos disease (CMTD) comprising administering (2S)-2-[4-bromo-2-(1,2-azol-3-yl)phenoxy]propionic acid or a pharmaceutical composition thereof to a patient with CMTD symptoms. Pharmaceutical compositions comprising (2S)-2-[4-bromo-2-5-(1,2-azol-3-yl)phenoxy]propionic acid and multi-component kits are also disclosed. Abstract

Claims

Claims1. A composition comprising (2S)-2-[4-bromo-2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof, for use in a method of treatment of Charcot-Marie-Tooth disease in a subject, wherein the composition is for administration at a therapeutic dose of 100 to 1500 mg of (2S)-2-[4-bromo-2-(1,2-oxazol-3-yl)phenoxy]propanoic acid.

2. The composition for use according to claim 1 , wherein the therapeutic dose is 200 to 600 mg.

3. The composition for use according to any one of the preceding claims, wherein the therapeutic dose is to be administered one, two or three times daily.

4. The composition for use according to any one of the preceding claims, wherein the composition is administered orally.

5. The composition for use according to any one of the preceding claims, wherein the composition is a solid dosage form.

6. The composition for use according to claim 6, wherein the composition comprises silicified microcrystalline cellulose.

7. The composition for use according to any one of the preceding claims, wherein the subject is suffering from CMT 1 , CMT2, CMT3 or CMT4.

8. The composition for use according to any one of the preceding claims, wherein the composition is to be administered orally using a solid dosage form and provides a plasma concentration-time profile of (2S)-2-[4-bromo-2-(1,2-oxazol-3- yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof, where the mean Cmax is 12,760 to 27,440 ng / mL after administration with a single dose of 400 mg (2S)-2-[4-bromo- 2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof.

9. The composition for use according to any one of the preceding claims, wherein the subject experiences an increase in the total distance walked when determined using the 6-minute walk test after treatment with (2S)-2-[4-bromo-2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof.

10. A kit-of-parts or a composition comprising:(2S)-2-[4-bromo-2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof, and a peripheral myelin protein 22 (PMP22) down regulator.11 . The kit-of-parts for use according to claim 10, wherein the kit-of-parts comprises 100 to 1500 mg of (2S)-2-[4-bromo-2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof.

12. The kit-of-parts according to any one of claims 10 or 11 for use in treatment of treatment of Charcot-Marie-Tooth disease.

13. The kit-of-parts according to any one of claims 10 or 11 for use in a method of treatment of treatment of Charcot-Marie-Tooth disease, wherein the kit-of-parts is for administration at a therapeutic dose of 100 to 1500 mg of (2S)-2-[4-bromo- 2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid.

14. The kit-of-parts for use according to any one of claims 12 or 13, wherein the therapeutic dose of (2S)-2-[4-bromo-2-(1 ,2-oxazol-3-yl)phenoxy]propanoic acid, or a pharmaceutically acceptable salt, hydrate, polymorph, tautomer, or solvate thereof is 200 to 600 mg.