Isis Pharmaceuticals Gets $1.5 Mln Milestone Payment From Biogen - Quick Facts

(RTTNews) - Isis Pharmaceuticals, Inc.(ISIS), Tuesday said it earned $1.5 million in milestone payments from Biogen Idec(BIIB), for the advancement of the ongoing Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy or SMA. The trial is an open-label, multiple-dose, dose-escalation pilot study in infants who have been diagnosed with Type I SMA. The study is being conducted at centers in the United States and Canada.

ISIS-SMNRx is an investigational compound designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. The compound received orphan drug status and fast track designation from the United States Food and Drug Administration or FDA.