· Nine of 12 patients (75%) could sit unassisted for 30 seconds or more.
· Eleven of 12 (92%) patients have sustained CHOP-INTEND scores more than 40 for a mean of 18.8 months.
· All previous motor milestone achievements reported at the American Academy of Neurology (AAN) meeting in April 2017 were validated and re-confirmed at each patients last visit by the independent, external reviewer. New milestones reported at AAN that had not been validated by the external reviewer at that time were confirmed and validated during the August 2017 review.
Additionally, patients who were free of respiratory or nutritional support at January 20, 2017, continue without the need for supportive care.
There were no new treatment-related adverse events identified.
A manuscript regarding these data has been accepted for publication in a major medical journal. In keeping with the embargo policy of that journal, AveXis will not report additional trial data until after the publication.
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 and is the leading genetic cause of infant mortality.
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.
AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1, designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons, providing rapid onset of effect and crossing the blood brain barrier to allow 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 companys initial proprietary gene therapy candidate, AVXS-101, is in the pivotal phase of study for the treatment of SMA Type 1. The company also intends to expand the study of gene therapy into SMA Type 2 and two additional rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.
For additional information, please visit www.avexis.com.
This press release contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis research, development and