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electrical
impedance myography testing (EIM) and "ability captured through interactive video evaluation-mini" testing (ACTIVE-mini).
Once
a patient meets the screening criteria for the clinical trial, the patient receives a one-time dosage of AVXS-101, by intravenous injection over a one hour period. The patient
remains at NCH for 48 hours after dosing for monitoring, and then is discharged. For one month after dosing, weekly follow-up evaluations are conducted. After the first month, additional
monthly evaluations are conducted for 23 months.
Preliminary Clinical Results
As of December 31, 2015, we had fully enrolled our Phase 1 trial, having dosed a total of 15 patients in
the trial. The first cohort, which completed dosing in September 2014, consisted of three infants who received a dose of AVXS-101 administered at the low dose, based on the patient's weight. The
second cohort consisted of 12 infants who received the proposed therapeutic dose of AVXS-101.
Based
on the preliminary results of the trial as of December 31, 2015, none of the 15 patients receiving AVXS-101 has experienced an "event," defined as death or at least
16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or perioperatively. Of the 13 patients for whom data was
available because they had at least one follow-up appointment as of December 31, 2015, all patients experienced improvement or stabilization from baseline in motor skills measured by their CHOP
INTEND scores and such improvement appeared to be dose-dependent. Based on the 15 patients dosed as of December 31, 2015, we have observed AVXS-101 to be generally well-tolerated. We expect to
continue to report quarterly findings throughout this Phase 1 clinical trial.
As
of December 31, 2015, all nine patients who have reached 8.1 months of age were event-free, all nine patients who have reached 10.5 months of age were
event-free, all six patients who have reached 13.6 months of age were event-free and the three patients who have reached 20 months of
age were event-free. We expect to have collected 13.6 months of data for all 15 patients enrolled in our Phase 1 clinical trial in the first quarter of 2017. Although the results of the Finkel
2014 Study were not pre-specified as a comparator for our trial, we believe that this compilation of data from patients with SMA Type 1 provides a useful context to consider the results of our
trial to date. This peer-reviewed publication reported that 75%, 50%, 25% and 8% of patients with SMA Type 1 were event-free at 8.1, 10.5, 13.6 and 20.0 months of age, respectively. As
of December 31, 2015, the median event-free age of the three patients in the first, low-dose cohort is 22.7 months, and the mean event-free age is 23.6 months, with the oldest
patient at 25.8 months of age. All three patients in the first, low-dose cohort are over 20 months of age and event-free as of December 31, 2015. The median event-free age of the
12 patients in the second, proposed therapeutic-dose cohort is 8.7 months, and the mean event-free age is 9.3 months, with the oldest patient at 18.7 months of age as of
December 31, 2015. The first six patients enrolled in the second, proposed therapeutic-dose cohort are over 10.5 months of age and event-free as of December 31, 2015. The median
event-free age of all 15 patients enrolled in the clinical trial is
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