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AVEXIS, INC. filed this Form S-1/A on 02/01/2016
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the natural history data for CHOP INTEND from the Finkel 2014 Study showed that patients with SMA Type 1 experienced an average decrease of 1.27 points per year.



          Patients with SMA Type 1 enter the trial at different ages, stages of disease progression and levels of motor function that can result in significant variability in baseline CHOP INTEND scores from patient to patient. Additionally, month-to-month variability in CHOP INTEND scores can be influenced by factors that are not related to study treatment. Examples of these factors include upper respiratory tract infections and fractured limbs, which may preclude the testing of some elements of the CHOP INTEND assessment. Our observations are consistent with the Finkel 2014 Study in which tabulated CHOP INTEND scores also demonstrated significant variability. In spite of these limitations, we believe observing change in CHOP INTEND scores over time is beneficial.

          The primary endpoint for our ongoing Phase 1 clinical trial of AVXS-101 is safety. As of December 31, 2015, we have observed a total of ten serious adverse events, or SAEs, in seven patients. The following table summarizes the SAEs that have been observed thus far.


          The treatment-related SAEs consisted of two patients that experienced elevated liver function enzymes, or LFEs, which were each assessed as a Grade 4 event under the Common Terminology Criteria for Adverse Events on the basis of laboratory values. We observed the first of these treatment-related SAEs in the first patient dosed with AVXS-101 in our ongoing Phase 1 clinical trial. After the onset of elevated LFEs, this patient was given a prednisolone regimen starting at 2 mg/kg


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