Compositions of 1,2,4-oxadiazole benzoic acid compounds and methods for their use
AssigneePTC Therapeutics, Inc.
InventorsGary Mitchell Karp, Seongwoo Hwang, Guangming Chen, Neil Gregory Almstead, Young-Choon Moon
Life of this patent
Estimating the term…
Abstract
Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
Claims22
1-20. (canceled)
A pharmaceutical composition comprising a liquid suspension of a compound of formula (I):
- or a pharmaceutically acceptable salt thereof wherein:
- Z is substituted or unsubstituted aryl, substituted or unsubstituted cycloalkyl, substituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted arylalkyl;
- R1 is hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted aryl, —(CH2CH2O)nR6 or any biohydrolyzable group;
- R2, R3, R4, R5 and R6 are independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl;
- substituted or unsubstituted cycloalkyl, substituted or unsubstituted aryl, alkoxy, aryloxy, halogen, CF3, OCF3, OCHF2, CN, COOH, COOR7, SO2R7, NO2, NH2, or N(R7)2;
- each occurrence of R7 is independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl;
- substituted or unsubstituted cycloalkyl, substituted or unsubstituted aryl, alkoxy, aryloxy, halogen or CF3;
- and
- n is an integer from 1 to 7.
A method for treating a disease or disorder associated with a premature stop codon in a patient having a disease or disorder associated with a premature stop codon, comprising administering to the patient the pharmaceutical composition of claim 1.
Dependencies are inferred from the claim text.