Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound

Patent metadata

Field	Value
Publication number	US-9913829-B2
Application number	US-201113813914-A
Country	US
Kind code	B2
Filing date	Jul 29, 2011
Priority date	Aug 5, 2010
Publication date	Mar 13, 2018
Grant date	Mar 13, 2018

Abstract

The present invention relates to the compound of formula (I)

for use in the treatment of a nonsense-mutation-mediated genetic disease.

First claim

The invention claimed is:

1. A method of treating a nonsense-mutation-mediated genetic disease selected from the group consisting of: nonsense-mutation-mediated Duchenne muscular dystrophy, nonsense-mutation-mediated lung cancer and nonsense-mutation-mediated dystrophic epidermolysis bullosa, which comprises administering as sole active ingredient to a subject in need thereof a compound of formula (I):

or a salt, solvate, clathrate, hydrate or polymorph thereof.

2. The method of claim 1 , wherein the compound of formula (I) is administered through injection.

3. A method of treating a nonsense-mutation-mediated genetic disease selected from the group consisting of: nonsense-mutation-mediated Duchenne muscular dystrophy, nonsense-mutation-mediated lung cancer and nonsense-mutation-mediated dystrophic epidermolysis bullosa, which comprises administering to a subject in need thereof a compound of formula (I):

or a salt, solvate, clathrate, hydrate or polymorph thereof,
in combination with an agent able to enhance readthrough of the mRNA premature stop-codon, said mRNA premature stop-codon coming from the translation of the gene comprising the nonsense-mutation.

4. The method of claim 3 , wherein said agent is 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl] benzoic acid.

5. The method of claim 3 , wherein the compound of formula (I) and the agent are administered simultaneously, sequentially or over a period of time.

Assignees

• Lejeune Fabrice
• Deprez Benoit
• Beghyn Terence
• Gonzalez Hilarion Sara Sofia
• Univ De Droit Et De Sante De Lille
• Inst Nat Sante Rech Med
• Pasteur Institut

Inventors

• Lejeune Fabrice
• Deprez Benoit
• Beghyn Terence
• Gonzalez-Hilarion Sara Sofia

Classifications

• A61P3/10

  for hyperglycaemia, e.g. antidiabetics · CPC title

• A61P7/06

  Antianaemics · CPC title

• A61P5/14

  of the thyroid hormones, e.g. T3, T4 · CPC title

• A61P37/00

  Drugs for immunological or allergic disorders · CPC title

• A61P7/04

  Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents · CPC title