发明名称 COMPOSITIONS AND METHODS FOR TREATING FRIEDREICH'S ATAXIA
摘要 The invention provides compositions and methods useful for the treatment of Friedreich's ataxia. In some embodiments, the invention provides compositions and methods useful for inhibiting the ubiquitination of frataxin.
申请公布号 US2016083358(A1) 申请公布日期 2016.03.24
申请号 US201514862072 申请日期 2015.09.22
申请人 Fratagene Therapeutics Ltd. 发明人 Testi Roberto;Incani Ottaviano;Rufini Alessandra;DeMartino Gabriella
分类号 C07D279/20 主分类号 C07D279/20
代理机构 代理人
主权项 1. A method of treating Friedreich's ataxia in a subject in need thereof, comprising administering to a subject a therapeutically effective amount of a polycyclic compound or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof; wherein the polycyclic compound is of a formula: wherein: X is a linking group selected from the group consisting of —S(O)2— and —C(O)—; R1a and R1b are each independently selected from the group consisting of hydrogen, lower alkyl, fluoroalkyl, aryl, and heteroaryl; wherein the aryl or heteroaryl group has from 0 to 3 R4 substituents; R2 is a first cyclic group selected from the group consisting of phenyl, naphthyl, and heteroaryl; wherein the R2 group has from 0 to 3 R4 substituents; R3 is a second cyclic group selected from the group consisting of phenyl, naphthyl, and heteroaryl; wherein the R3 group has from 0 to 4 R4 substituents; each R4 substituent is independently selected from the group consisting of C1-C6 alkyl, C2-C6 alkenyl, C2-C6 alkynyl, C1-C6 alkoxy, halo, —NO2, —CF3, —CN, —OR5, —SR5, —C(O)R5, —NHC(O)R5, —C(O)OR5, —OC(O)R5, —NR5R6, —C(O)NR5R6, —NHR5C(O)NR6R7, —S(O)2NR5R6, and a cyclic R4 substituent; wherein the cyclic R4 substituent is selected from the group consisting of aryl, heteroaryl, arylalkyl, heteroarylalkyl, cycloalkyl, cycloalkylalkyl, and heterocyclyl; and wherein the cyclic R4 substituent has from 0 to 3 R8 substituents; each R5, R6, or R7 is independently H, C1-C6 alkyl, C2-C6 alkenyl, C2-C6 alkynyl, C1-C6 alkoxy, halo, or a cyclic R5 substituent; wherein the cyclic R5 substituent is selected from the group consisting of aryl, heteroaryl, arylalkyl, heteroarylalkyl, cycloalkyl, cycloalkylalkyl, and heterocyclyl; and wherein the cyclic R5 substituent has from 0 to 3 R8 substituents; and each R8 is independently C1-C6 alkyl, C2-C6 alkenyl, C2-C6 alkynyl, C1-C6 alkoxy, halo, aryl, heteroaryl, arylalkyl, heteroarylalkyl, cycloalkyl, cycloalkylalkyl, or heterocyclyl; and wherein adjacent R4, R5, R6, or R7 substituents may optionally join to form an additional fused ring selected from the group consisting of aryl, heteroaryl, cycloalkyl, and heterocyclyl; wherein the additional fused ring has from 0 to 3 R8 substituents; with the proviso that the polycyclic compound is not disclosed in U.S. Pat. No. 8,703,749.
地址 Rome IT