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S-1
AVEXIS, INC. filed this Form S-1 on 01/15/2016
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11.8 months, with the oldest patient at 25.8 months of age. The following figure shows survival data of all 15 patients enrolled in the clinical trial through December 31, 2015.

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          The CHOP INTEND test is designed to evaluate the motor skills of patients with SMA Type 1 by testing 16 items, which measure the movement of various body segments. The Finkel 2014 Study, the only natural history data set that observed CHOP INTEND scores over a period of time, reported that patients with SMA Type 1 have an average annual decrease of 1.27 points in their CHOP INTEND score. Of the 13 patients for whom data was available in our ongoing Phase 1 clinical trial because they had at least one follow-up appointment as of December 31, 2015, all patients have had increases or stabilization in their CHOP INTEND scores relative to their baseline measurement. All three patients in the first, low-dose cohort and six of the 12 patients in the second, proposed therapeutic-dose cohort have been on treatment for at least six months as of December 31, 2015. Among these patients, the average improvement in CHOP INTEND scores after six months was 4.33 points for the first, low-dose cohort and 17.17 points for the second, proposed therapeutic-dose cohort. As of December 31, 2015, the mean CHOP INTEND score for the three patients in the first, low-dose cohort was 22.3 points, representing a mean increase from baseline of 6.0 points, and 47.1 points for the ten patients in the second, proposed therapeutic-dose cohort, representing a mean increase from baseline of 18.0 points, with a mean increase from baseline of 15.2 points for the 13 patients across the two dose cohorts for whom preliminary clinical results were available. These increases in CHOP INTEND scores have been observed as early as one month after treatment and have been generally sustained at or above baseline through the period up to December 31, 2015. We believe that there may be a dose response, based on our observation of CHOP INTEND scores of patients receiving the proposed therapeutic dose as compared to patients receiving the low dose because, as a group, the patients receiving the therapeutic dose appear to be demonstrating a larger average CHOP INTEND score increase. As of December 31, 2015, we had dosed two patients in the second, proposed therapeutic-dose cohort for whom preliminary clinical results are not yet available.

          The following table contains a summary of preliminary CHOP INTEND data from our ongoing Phase 1 clinical trial, as of December 31, 2015, for the first and second cohort of patients who received the low and proposed therapeutic dose of AVXS-101, respectively. As described above,

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