Isis Pharmaceuticals Advances Phase 2 Study of ISIS-SMN Rx in Infants with SMA

Isis Pharmaceuticals announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMN Rx in infants with Type I spinal muscular atrophy (SMA).  The Phase 2 study is designed to allow Isis to define the optimal dose for the larger Phase 2/3 study in infants and to provide safety and tolerability data.  Isis plans to complete this study and initiate the larger Phase 2/3 pivotal study early next year. 

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 Type I 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 is being conducted at centers in the United States and Canada. More info at clinicaltrials.gov.

"We continue to be very encouraged with the progress we are making with ISIS-SMNRx, and we are on track to begin our Phase 3 program early next year.   We look forward to sharing the results from our multiple dose studies in infants with Type I SMA and in children with Type II and Type III SMA later this year or early next year," said C. Frank Bennett, Ph.D., senior vice president of research at Isis.