SEC Filings

AVEXIS, INC. filed this Form 8-K on 11/02/2017
Entire Document

Exhibit 99.1



Media Inquiries:

Lauren Barbiero

W2O Group



Investor Inquiries:

Jim Goff

AveXis, Inc.



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


“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



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