SEC Filings

8-K
AVEXIS, INC. filed this Form 8-K on 11/02/2017
Entire Document
 

 

 

 

 

 

 

 

Event-free Survival and Motor and Other Milestones Among the 12 Patients in Cohort 2 as of August 7,
2017*

 

 

Age at

 

 

 

Brings

 

 

 

 

 

 

 

Sits Unassisted(c)

 

Other Achievements

Cohort
2

 

Gene
Transfer
(mos)

 

Event-
Free
Survival(a)

 

Hand
to
Mouth

 

Controls
Head

 

Rolls
Over(b)

 

Sits with
Assistance

 

>5
secs

 

>10
secs

 

>30
secs

 

Speaks

 

Swallows

 

No
NIV
Use

 

No
Nutritional
Support(d)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

E.04

 

5.6

 

31.1

 

+

 

+

 

+

 

+

 

+

 

 

 

 

 

+

 

+

 

 

 

 

E.05

 

4.2

 

28.5

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.06

 

1.9

 

26.1

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.07

 

3.6

 

28.1

 

+

 

+

 

+

 

+

 

+

 

+

 

 

 

+

 

+

 

+

 

 

E.08

 

7.9

 

32.4

 

+

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

E.09

 

4.9

 

28.9

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.10

 

0.9

 

25.3

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.11

 

2.3

 

23.8

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

 

 

 

E.12

 

2.6

 

23.9

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.13

 

0.9

 

22.1

 

+

 

+

 

 

 

+

 

+

 

+

 

+

 

+

 

+

 

 

 

 

E.14

 

4.1

 

22.0

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

 

+

E.15

 

2.1

 

20.6

 

+

 

+

 

 

 

+

 

+

 

+

 

+

 

+

 

+

 

 

 

 

Patient with outcome (%)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

This Study

 

100

 

100

 

92

 

75

 

92

 

92

 

83

 

75

 

92

 

92

 

58

 

50

Natural History

 

8 by 20 mos(e)

 

NA

 

0

 

0**

 

0**

 

0**

 

0**

 

0**

 

NA

 

NA

 

NA

 

8 by 20 mos(e)

 


*At baseline, none of the patients in Cohort 2 had achieved any of the listed motor milestones except for bringing a hand to the mouth. As of August 7, 2017, the majority of these patients had reached at least one major motor milestone. No patients in Cohort 1 are listed, since none attained any motor milestones. NA denotes not available, and NIV noninvasive ventilation. Plus signs indicate achievement of milestone.

 

(a)              Event-free survival (the primary efficacy outcome) was defined as the age at the end of the study at which patients were free of ventilatory support, which was defined as the need for ventilation for at least 16 hours per day for at least 14 consecutive days.

(b)             According to item 20 on the Bayley Scales of Infant and Toddler Development, rolling over is defined as movement of at least 180 degrees both left and right from a position of lying on the back.

(c)              Sitting unassisted for at least 5 seconds is in accordance with the criteria of item 22 on the Bayley Scales of Infant and Toddler Development gross motor subtest and surpasses the 3-second count that is used as a basis for sitting (test item 1) on the Hammersmith Functional Motor Scale—Expanded for Spinal Muscular Atrophy (SMA). Sitting unassisted for at least 10 seconds is in accordance with the criteria used in the World Health Organization Multicentre Growth Reference Study. Sitting unassisted for at least 30 seconds defines functional independent sitting and is in accordance with the criteria of item 26 on the Bayley Scales of Infant and Toddler Development gross motor subtest.

(d)             Nutritional support refers to the placement of either a gastrostomy tube or a nasogastric tube, as determined by the preference of the parents or the primary physician. Once enrolled in the study, all the patients who required nutritional support underwent gastrostomy-tube placement, and none were removed during the study.

(e)              Data are from Finkel et al.

** Data are from De Sanctis et al.

 

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