Ionis Pharmaceuticals Inc (IONS)

[surf1944]

1:23AM ISIS Pharm presents data from ISIS-SMN Rx Phase 1 study in children with spinal muscular atrophy (ISIS) 17.84 : Co announces that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy were presented. In the presentation, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children.

Co that ISIS-SMNRx was well tolerated when administered intrathecally as a single dose directly into the spinal fluid and that no safety concerns related to the drug were identified. In addition, the intrathecal injection procedure was well tolerated in children with SMA. Concentrations of ISIS-SMNRx measured in cerebral spinal fluid were consistent with levels predicted from preclinical studies, indicating that the drug half-life in nervous system tissues is very long and that dosing once every six to nine months is feasible. Although the study was not designed to provide evidence of functional activity, clinically significant, dose-dependent improvements in the Hammersmith Functional Motor Scale-Expanded, a measure of muscle function, were observed in these children.