Isis Reports Follow-Up Data from Phase 1 ISIS-SMN Rx SMA Study

Isis Pharmaceuticals, Inc.

announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA), show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.  The Phase 1 data, including these preliminary follow-on data, will be presented at the International Congress of the World Muscle Society by Dr. Kathy Swoboda on Oct. 3, 2013.  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.  ISIS-SMNRx is an antisense drug designed to treat all types of SMA. The preliminary data reported today is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx.  Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the