AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society
All patients were alive and event-free at 20 months of age as of the August 7, 2017 data cut-off
Patients in Cohort 2 continue to demonstrate improvements in motor milestones
Saint Malo, France (October 3, 2017) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report top-line data as of August 7, 2017, from the Phase 1 trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 in a late-breaking breaking poster authored by Jerry Mendell, M.D., principal investigator in the trial and Curran-Peters Chair of Pediatric Research, Professor of Pediatrics and Neurology at the Research Institute at Nationwide Childrens Hospital and The Ohio State University, Columbus, Ohio, during the 22nd International Annual Congress of the World Muscle Society
(WMS) in Saint Malo, France.
The Phase 1, open-label, dose-escalating study was designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. The key measures of efficacy were the time from birth to an event, which was defined as either death or at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or perioperatively, and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor milestone development survey and Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).
Data as of August 7, 2017, showed all patients (100%) were alive and event-free at 20 months of age. Patients in Cohort 2 continued to demonstrate improvements in motor milestones, including: