SEC Filings

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

§

All motor milestones have been assessed and adjudicated by an independent third-party reviewer using video evidence.

 

The proposed therapeutic dose-cohort

Age at Gene Transfer (mos)

Event-Free Survivala

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

Brings Hand to Mouth

Controls Head

Rolls Overb

Sits with Assistance

Sits Unassistedc

Other Achievements

≥5 secs

≥10 secs

≥30 secs

Speaks

Swallows

No NIV Use

No Nutritional Supportd

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

+

+

 

+

+

+

+

+

+

 

 

This Study

100

100

92

75

92

92

83

75

92

92

58

50

Natural History

8 by 20 mose

NA

0

0**

0**

0**

0**

0**

NA

NA

NA

8 by 20 mose

 

*At baseline, none of the patients in the proposed therapeutic dose-cohort 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.

20


 


© AveXis, Inc. All Rights Reserved.