SEC Filings

10-Q
AVEXIS, INC. filed this Form 10-Q on 11/09/2017
Entire Document
 

o

As has been previously reported, a total of five adverse events, or AEs, in four patients were deemed treatment-related. Of these, two were serious adverse events, or SAEs, experienced by two patients, and three were non-serious AEs experienced by two patients. All consisted of clinically asymptomatic liver enzyme elevations and were resolved with prednisolone treatment. There were no clinically significant elevations of gamma-glutamyl transferase, alkaline phosphatase or bilirubin and, as such, the conditions for drug induced liver injury, also known as Hy’s Law,  were not met. Other non-treatment-related AEs were expected and were associated with SMA.

o

A cumulative total of 297 AEs (five treatment-related AEs and 292 non-treatment related AEs) were reported as of August 7, 2017, following monitoring and source verification. Of these, 56 were determined to be SAEs and 241 were non-serious AEs.

 

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Treatment Durability and Motor Milestone Achievement

o

As of August 7, 2017, 11 of 12 patients (92%) in the proposed therapeutic dose-cohort have achieved and maintained CHOP-INTEND scores of ≥40 points at 20 or more months of age.

o

As of August 7, 2017, 11 of 12 patients (92%) in the proposed therapeutic dose-cohort achieved head control, 9 of 12 patients (75%) could roll over and 11 of 12 patients (92%) could sit with assistance by 20 months of age.  

o

As of August 7, 2017, 11 of 12 patients (92%) in the proposed therapeutic dose-cohort could sit unassisted for at least five seconds, 10 of 12 patients (83%) could sit unassisted for at least 10 seconds and nine of 12 patients (75%) could sit unassisted for 30 seconds or more by 20 months of age.  

o

As of August 7, 2017, two of 12 patients (17%) in the proposed therapeutic dose-cohort could crawl, pull to a stand and stand and walk independently by 20 months of age.

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