CARLSBAD, Calif., Oct. 3, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) 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. Isis earned a $2 million milestone payment from Biogen Idec associated with the advancement of this study.
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. For further study information, please visit www.clinicaltrials.gov and search for ISIS-SMN Rx.
"We continue to be very encouraged with the progress we are making with ISIS-SMN
Rx, 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.
ABOUT ISIS-SMN Rx ISIS-SMN Rx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. The United States Food and Drug Administration granted orphan drug status and fast track designation to ISIS-SMN Rx for the treatment of patients with SMA. Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMN Rx, to treat all types of SMA. Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study.