AveXis to Present Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at AAN Annual Meeting

CHICAGO, March 02, 2017 -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced results from the Phase 1 trial of AVXS-101, AveXis’ proprietary gene therapy candidate for the treatment of spinal muscular atrophy (SMA) Type 1, will be presented at the 2017 Annual Meeting of the American Academy of Neurology (AAN) taking place April 22-28 in Boston.

“AVXS‐101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Event-free Survival and Achievement of Developmental Milestones” will be presented by Jerry Mendell, MD, principal investigator in the trial and Curran-Peters Chair of Pediatric Research, Professor of Pediatrics and Neurology at the Research Institute at Nationwide Children’s Hospital and The Ohio State University, Columbus, Ohio, during the Clinical Trial Plenary Session at 10:30 a.m. EDT on April 25, and during the “Best of” Session for Neuromuscular Disease Therapeutics at 8:16 a.m. EDT on April 26.

"We are eager to present these data from the first ever clinical trial of gene therapy in spinal muscular atrophy," said Sukumar Nagendran, MD, Chief Medical Officer, AveXis. "AveXis is dedicated to continued research in spinal muscular atrophy, the most common genetic cause of infant mortality, and we look forward to presenting this and other important SMA research at the AAN Annual Meeting."

A total of five preclinical and clinical abstracts will be presented during the meeting, underscoring the company’s leadership in pioneering gene therapy to treat life-threatening neurological diseases. In addition to the presentations highlighted above, these include:

• “AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Experience with Preexisting Anti-AAV9 Antibody in the SMA1 Population;” oral presentation on April 24 at 1:00 p.m. during S13: Motor Neuron Diseases: Biomarkers, Outcome Measures and Therapeutics.
• “AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Correlation between CHOP-INTEND and Motor Milestone Achievements;” oral presentation on April 24 at 1:12 p.m. during S13: Motor Neuron Diseases: Biomarkers, Outcome Measures and Therapeutics.
• “CSF Delivery of AAV9-mediated Gene Therapy for SMA, a Lethal Neuromuscular Disease in Children: A Dose-response Study in Mice and Nonhuman Primates;” poster presentation on April 25 at 5:30 – 7:00 p.m.

All abstracts can be accessed through the AAN website at www.aan.com.

About SMA
SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.

The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.

About AVXS-101
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is currently in development for SMA. AVXS-101 is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN1 gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.

About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company’s initial proprietary gene therapy candidate, AVXS-101, is being evaluated in a Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com.

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AveXis, Inc.
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