CYSTEAMINE PRODRUGS, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE

摘要

Cysteamine prodrugs, pharmaceutical compositions comprising the cysteamine prodrugs, and methods of using cysteamine prodrugs and pharmaceutical compositions thereof for treating eye, kidney, liver, metabolic, and neurological disorders such as cystinosis, cystinuria, non-alcoholic steatohepatitis, Batten disease, Leigh syndrome, and Huntington's disease.

主权项

1. A compound according to Formula (I): or a pharmaceutically acceptable salt thereof, wherein:
X1 is ethane-1,2-diyl;W1 is chosen from a substituent of Formula (I-a) or Formula (I-b); andW2 is chosen from hydrogen or the same substitutent as W1: wherein
R1, R2, R3, and R4 are independently chosen from hydrogen, C1-6 alkyl, C3-12 cycloalkyl, C3-12 heterocycloalkyl, C5-10 aryl, and C5-10 heteroaryl; andY1 is chosen from a bond and —O—.