AveXis reports interim Phase 1 data on AVXS-101 in spinal muscular atrophy
AveXis provided an update on interim data from the ongoing Phase 1 trial of AVXS-101 in spinal muscular atrophy Type 1 as of September 15. The data were presented at the 21st International Annual Congress of the World Muscle Society. "For the first time, interim data from the trial were presented that highlighted patient achievement of key motor development milestones as of September 15. Two-thirds of patients in Cohort 2 - the proposed therapeutic dose - had achieved the ability to sit unassisted, including one patient whose achievement of this milestone was confirmed after September 15. In Cohort 2, 11 of 12 patients achieved head control, 7 of 12 patients could roll over completely and 11 of 12 patients could sit with support. Two patients are now walking independently, including one whose achievement of this milestone was confirmed after September 15. These two patients each achieved earlier and important developmental milestones such as crawling, standing with support, standing alone and walking with support," the company said. Data as of September 15 showed AVXS-101 continued to demonstrate a favorable safety profile and was generally well tolerated, with no new treatment-related safety or tolerability concerns identified.