AveXis provides more interim data for SMA gene therapy

AveXis Inc. (NASDAQ:AVXS) reported updated interim data from the cohort of patients receiving the proposed therapeutic dose of AVXS-101 in an ongoing, open-label Phase I study in infants with spinal muscular atrophy type 1. As of the Sept. 15 cutoff, all 12 patients in the cohort were event-free, defined as death or requiring at least 16 hours per day of ventilation support for greater than two weeks in the absence of an acute reversible illness. The median age at last follow-up for the cohort is 17.3 months.

AveXis said two-thirds of patients in the cohort achieved the ability to sit unassisted, 11 patients had head control or could sit with support, and seven patients could roll over completely. The company said two patients are now walking independently. Data were presented at the World Muscle Society meeting in Granada, Spain. ...