ISIS Pharm Begins Clinical Study of ISIS-SMN Rx in Infants With Spinal Muscular Atrophy

Isis Pharmaceuticals, Inc.

announced the initiation of a Phase 2 study of ISIS-SMN[Rx] in infants with spinal muscular atrophy (SMA).   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.  The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study, which initiates the Phase 2/3 program for ISIS-SMN[Rx] in infants, is designed to provide data to allow Isis to define the optimal dose for the larger planned Phase 2/3 study in infants and to provide safety and tolerability data.  Isis will earn a $3.5 million milestone payment from Biogen Idec when the first infant is dosed in the study, which is projected for May 2013.   The Phase 2 study of ISIS-SMN[Rx] is an open-label, multiple-dose, dose-escalation pilot study, which will include eight infants who have been diagnosed with SMA.  To meet enrollment criteria, infants must be between the ages of three weeks and seven months, live in close proximity to a study site and pass screening evaluations conducted at study sites.  The study will be conducted at centers in the United States and Canada.  For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMN[Rx].