US 7,465,738 B2
Compounds useful as promoters of SMN2
Jill Jarecki, San Diego, Calif. (US); Xiaocun Chen, San Diego, Calif. (US); Dennis James Hurley, San Marcos, Calif. (US); Lewis R. Makings, Encinitas, Calif. (US); Mark T. Miller, San Diego, Calif. (US); Brian Pollok, Middleton, Wis. (US); Jeffrey H. Stack, San Diego, Calif. (US); and Michael A. Whitney, San Diego, Calif. (US)
Assigned to Vertex Pharmaceuticals Incorporated, Cambridge, Mass. (US)
Filed on Jun. 16, 2004, as Appl. No. 10/869,498.
Claims priority of provisional application 60/479064, filed on Jun. 16, 2003.
Claims priority of provisional application 60/479065, filed on Jun. 16, 2003.
Claims priority of provisional application 60/479062, filed on Jun. 16, 2003.
Claims priority of provisional application 60/479063, filed on Jun. 16, 2003.
Claims priority of provisional application 60/479024, filed on Jun. 16, 2003.
Prior Publication US 2005/0065173 A1, Mar. 24, 2005
Int. Cl. A61K 31/517 (2006.01); A61K 31/55 (2006.01); C07D 239/72 (2006.01)
U.S. Cl. 514—266.2  [514/266.4; 544/284; 544/291] 10 Claims
 
1. A method of treating or ameliorating one or more symptoms produced by Spinal Muscular Atrophy in a patient, comprising the step of administering to said patient a therapeutically effective amount of a compound of formula I:

OG Complex Work Unit Drawing
or a pharmaceutically acceptable salt thereof, wherein:
X is O or S;
R1 is a C1-6 aliphatic group, C6-10 aryl ring, heteroaryl ring having 5-10 ring atoms, or heterocyclyl ring having 3-10 ring atoms, each optionally substituted with 0-5 TR3; wherein each TR3 is independently halogen, CN, NO2, or an optionally substituted C1-4 alkyl, aryl, aralkyl, —N(R′)2, —CH2N(R′)—OR′, —Ch2OR′, —SR′, —CH2SR′,—COOR′,—NRCOR′,—CON(R′)2, or —S(O)2N(R′)2;
each R2 is independently halogen, CN, NO2, or an optionally substituted C1-4 alkyl, aryl, aralkyl, —N(R′)3, —CH2N(R′)2, —OR′, CH2OR′, —SR′, CH2SR′, —COOR′, NRCOR′, —CON(R′)2, or —S(O)2N(R′)2;
R′ is hydrogen or an optionally substituted C1-6 aliphatic group, an optionally substituted C6-10 aryl ring, an optionally substituted heteroaryl ring having 5-10 ring atoms, or an optionally substituted heterocyclyl ring having 3-10 ring atoms; and
y is 0, 1, 2 or 3.