Isis Pharmaceuticals Reports Interim Results from ISIS-SMN Rx Phase 2 Study in Infants with Spinal Muscular Atrophy
Isis Pharmaceuticals, Inc reports Interim Results from its ongoing open label, multiple dose ISIS-SMN Rx Phase 2 Study in Infants with Spinal Muscular Atrophy I type. 4 patients with spinal muscular atrophy are involved. After 6 months of treatment infants are alive and can breath without respiratory support. To date, all four infants are now approximately nine and a half to 16 months in age with an average age of approximately 12 and a half months. Isis announced late in 2013 that the Phase 2 infant study was expanded to enroll up to 20 infants and that the first infant was dosed in the 12 mg dose cohort. Infants from the 12 mg dose cohort will also be eligible to receive an additional 12 mg dose six months after they have completed the initial three scheduled doses. Clinical trial is registered at NCT. ISIS-SMNRx is also currently being evaluated in an open-label, multiple-dose, dose-escalation Phase 1b/2a study in children with SMA with Type II and Type III SMA. SIS-SMNRx is an antisense drug designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein for treatment of all three types of SMA