· The open-label, single-arm, single-dose, multi-center trial known as STR1VE is designed to evaluate the efficacy and safety of a one-time IV infusion of AVXS-101 of 1.1 x 1014 vg/kg, which is equivalent to the proposed therapeutic dose (Cohort 2) in the Phase 1 trial, in patients with SMA Type 1.
· The trial will enroll a minimum of 15 patients with SMA Type 1 who are less than six months of age at the time of gene therapy, and who have one or two copies of the SMN2 backup gene as determined by genetic testing and bi-allelic SMN1 gene deletion or point mutations. The intent to treat (ITT) population defined in the protocol includes symptomatic infants less than six months of age with the bi-allelic deletion of the SMN1 gene, only two copies of the SMN2 backup gene and no exon 7 modifier. There will be at least a four-week dosing interval between dosing of the first three patients to allow review of the safety analysis as well as early signals of efficacy, prior to dosing of the next patient.
First Patient Dosed in Pivotal Trial of AVXS-101 in SMA Type 1: Today, AveXis announced the first patient has been dosed in the pivotal trial. The patient met the ITT criteria of being symptomatic, less than six months of age and was confirmed by genetic testing to have a bi-allelic deletion of the SMN1 gene, two copies of the SMN2 backup gene, and no exon 7 modifier.
Data from Phase 1 Trial of AVXS-101 in SMA Type 1 Presented at the International Annual Congress of the World Muscle Society (WMS): On October 3, AveXis reported top-line data as of August 7, 2017, from the Phase 1 trial of AVXS-101 in patients with SMA Type 1 in a late-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.
Analysis Presented at WMS of Seven Patients in Cohort 2 of the Phase 1 Trial of AVXS-101 in SMA Type 1 Demonstrated Cognitive, Fine Motor and Language Abilities Within the Normal Range: Linda Lowes, PT, PhD, Director of Clinical Therapies Research and a member of the Center for Gene Therapy at the Research Institute of Nationwide Childrens Hospital, analyzed seven of the 12 patients in Cohort 2 of the Phase 1 Trial of AVXS-101 in SMA Type 1 using subsets of the Bayley Scales of Infant and Toddler Development® Third Edition, a norm-referenced, well-validated tool for infant and toddler development. Selection of patients for this evaluation was based on availability and ability to complete the testing.
· All patients evaluated demonstrated fine motor, cognition and language ability within the normal range. Natural history indicates patients will rarely achieve the ability to speak or retain any significant motor ability.
· In normal children, average composite scores for language and cognition abilities are 90-109, and fine motor skills are reported as a scaled score in which 10 is the mean.
· Seven of 7 (100%) patients demonstrated age-appropriate language (94-106), cognition (90-105) and fine motor skills (9-12) when compared to healthy age-matched peers.
Data from Phase 1 Trial of AVXS-101 in SMA Type 1 Published in The New England Journal of Medicine (NEJM): On November 1, AveXis reported the full data from the Phase 1 study was published in NEJM. Data as of August 7, 2017, showed:
· All 15 patients (100%) were alive and event-free at 20 months of age, in contrast to the eight percent event-free rate demonstrated in an independent, peer-reviewed natural history study for patients with SMA Type 1. The median age at last follow-up was 25.7 months and 30.8 months for 12 patients in Cohort 2 and the three patients in the low-dose cohort (Cohort 1),