TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
There are provided compounds of Formula (I) wherein three of A 1 , A 2 , A 3 , and A 4 represent CH and one of A 1 , A 2 , A 3 , and A 4 represents CR 1 ; R 1 represents SO 2 R2 or NHCOR2 wherein R2 represents C 1 -C 6 alkyl optionally substituted by one or more halogen, hydroxyl or alkoxy groups; R 9 represents C 6 -C 10 aryl; or a pharmaceutically acceptable salt thereof.
1 . A compound of formula (Ia) or (Ib):
wherein
three of A 1 , A 2 , A 3 , and A 4 represent CH and one of A 1 , A 2 , A 3 , and A 4 represents CR 1 ;
A 5 represents CH or N;
R 1 represents SO 2 R 2 or NHCOR 2 wherein R 2 represents C 1 -C 6 alkyl optionally substituted by one or more halogen, hydroxyl or alkoxy groups;
R 9 represents C 6 -C 10 aryl, optionally substituted by one or more halogen groups;
or a pharmaceutically acceptable salt thereof.
2 . The compound of claim 1 having formula Ia.
3 . The compound of claim 1 , wherein R 9 represents 2-naphthyl or 4-chlorophenyl.
4 . The compound of claim 1 , wherein A 1 , A 2 and A 4 represent CH and A 3 represents CR 1 .
5 . The compound of claim 1 , wherein R 2 is ethyl or isopropyl.
6 . The compound selected from the following table of claim 1 , wherein the compound is:
N-(1-(naphthalen-2-yl)-1H-indazol-6-yl)isobutyramide;
N-(2-(naphthalen-2-yl)-2H-indazol-6-yl)isobutyramide;
2-(4-chlorophenyl)-6-(ethylsulfonyl)-2H-indazole; or
5-(ethylsulfonyl)-2-(naphthalen-2′-yl)-2H-benzo[d][1,2,3]triazole.
7 . A pharmaceutical composition, comprising a compound of claim 1 and a pharmaceutically acceptable carrier.
8 . A method for the treatment or prophylaxis of Duchenne muscular dystrophy, Becker muscular dystrophy or cachexia, comprising administering the compound of claim 1 .
9 . (canceled)