AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1
Patients who received a single dose of AVXS-101 resulted in longer survival, superior
achievement of motor milestones, and better motor function than in historical cohorts
As of August 7, 2017, all patients are alive, event-free and have reached at least 20 months of age
As of August 7, 2017, all patients who received the proposed therapeutic dose of AVXS-101 demonstrated continued treatment durability and achievement of motor milestones, including 75 percent of these patients now sitting for 30 seconds or longer
Chicago, Ill. (November 1, 2017) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced data as of August 7, 2017, from the Phase 1 trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 were published today in the New England
Journal of Medicine (NEJM) in a paper titled Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. These data demonstrate that all patients who received a one-time intravenous dose of AVXS-101 are alive and event-free at 20 months of age. Natural history indicates that only eight percent of untreated patients with SMA Type 1 will survive event-free at 20 months of age. The publication may be found online at www.nejm.org/doi/full/10.1056/NEJMoa1706198.
It is incredibly encouraging to see that all children who have received AVXS-101 remain event-free and demonstrate a durable treatment effect at 20 months of age and older, including in many cases achievement of new developmental milestones, Sean Nolan, President and Chief Executive Officer of AveXis. To have these Phase 1 data published in the prestigious New England Journal of Medicine