Clinical Trials

Date: 2017-11-01

Type of information: Publication of results in a medical journal

phase: 1-2

Announcement: presentation of results at the 2017 Annual Meeting of the American Academy of Neurology (AAN)

Company: Avexis (USA - TX), now a Novartis company (Switzerland)

Product: AVXS-101

Action mechanism:

gene therapy. AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is the only clinical-stage gene therapy in development for SMA. AVXS-101 is designed to address the monogenetic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features.

Disease: spinal muscular atrophy (SMA) Type 1

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Country: USA

Trial details:

The open-label study is designed to evaluate safety and efficacy of AVXS-101 in patients suffering from SMA Type 1. The primary outcome in the study is safety and tolerability. The secondary outcome measure is an efficacy measure as defined by the time from birth to an “event” with an event defined as death or until a patient requires at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of an acute reversible illness or perioperatively. Exploratory outcome measures include motor function testing, measured by the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), a test developed to measure motor skills of patients with SMA Type 1, and other motor milestone development surveys and tests.
The clinical protocol requires that each patient receive a one-time dosage of AVXS-101, by intravenous injection over a one hour period. The patient remains at the clinical trial site for 48 hours after dosing for monitoring prior to discharge, and weekly follow-up evaluations are conducted for one month after dosing. After the first month, additional evaluations are conducted monthly for 23 months. (NCT02122952)

Latest news:

• On November 1, 2017, AveXis announced data as of August 7, 2017, from the Phase 1 trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 were published in the New England Journal of Medicine (NEJM) in a paper titled “Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.” These data demonstrate that all patients who received a one-time intravenous dose of AVXS-101 are alive and event-free at 20 months of age. Natural history indicates that only eight percent of untreated patients with SMA Type 1 will survive event-free at 20 months of age.
The NEJM publication provides detailed data as of August 7, 2017, from the Phase 1, open-label, dose-escalating study, designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. The key measures of efficacy were the time from birth to an “event,” which was defined as either 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, and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor milestone development survey and Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).
Event-free Survival and Safety: Data as of August 7, 2017, showed no new events, and 15 of 15 (100%) patients were event-free at 20 months of age. The expected event-free survival rate at 20 months of age based on the natural history of the disease is eight percent. The median age at last follow-up was 25.7 months and 30.7 months for patients in the proposed therapeutic-dose cohort (Cohort 2) and low-dose cohort (Cohort 1), respectively.
As has been previously reported, a total of five adverse events (AEs) in four patients were deemed treatment-related. Of these, two were serious adverse events (SAEs) experienced by two patients, and three were non-serious AEs experienced by two patients. All consisted of clinically asymptomatic liver enzyme elevations and were resolved with prednisolone treatment. There were no clinically significant elevations of gamma-glutamyl transferase, alkaline phosphatase or bilirubin and, as such, Hy’s Law was not met. Other non-treatment-related AEs were expected and were associated with SMA.
A cumulative total of 297 AEs (five treatment-related AEs and 292 non-treatment related AEs) were reported as of August 7, 2017, following monitoring and source verification. Of these, 56 were determined to be SAEs and 241 were non-serious AEs.AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new treatment-related safety or tolerability concerns identified.
Treatment Durability and Motor Milestone Achievement:
As of August 7, 2017, 11 of 12 patients (92%) in Cohort 2 have achieved and maintained CHOP-INTEND scores of ?40 points.
As of August 7, 2017, 11 of 12 patients (92%) in Cohort 2 achieved head control, nine of 12 patients (75%) could roll over and 11 of 12 patients (92%) could sit with assistance.
As of August 7, 2017, 11 of 12 patients (92%) in Cohort 2 could sit unassisted for at least five seconds, 10 of 12 patients (83%) could sit unassisted for at least 10 seconds and nine of 12 patients (75%) could sit unassisted for 30 seconds or more.
As of August 7, 2017, two patients in Cohort 2 could crawl, pull to a stand and stand and walk independently.
All motor milestones have been assessed and adjudicated by an independent third-party reviewer using video evidence.

Cohort

Age at

Gene

Transfer

(mos)

Event-

Free

Survival^a

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

Brings

Hand

Mouth

Controls

Head

Rolls

Over^b

Sits with

Assistance

Sits Unassisted^c

Other Achievements

secs

?10

secs

?30

secs

Speaks

Swallows

NIV

Use

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

Natural History

8 by 20

mos^e

0**

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.
Nutritional and Respiratory Support: According to natural history of the disease, nearly all Type 1 patients require nutritional and respiratory support by 12 months of age, and are not able to swallow or speak effectively.
As of August 7, 2017, patients who were free of respiratory or feeding support on January 20, 2017, continued without the need for supportive care.
As of August 7, 2017, six of seven (86%) patients in Cohort 2 that did not require feeding support before treatment continued without feeding support after treatment; seven of 10 (70%) patients that did not require bi-level positive airway pressure (BiPAP) support before treatment did not require BiPAP support at last assessment.
As of August 7, 2017, eleven of 12 (92%) patients in Cohort 2 were fed orally, and six of 12 (50%) patients were exclusively fed orally.
Further, as of August 7, 2017, eleven of 12 (92%) patients were able to speak; three more patients than previously reported on April 25, 2017 at the American Academy of Neurology. AVXS-101 is currently being evaluated in a pivotal trial for the treatment of SMA Type 1.
• On April 25, 2017, AveXis presented results from the closeout of the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 at the 2017 Annual Meeting of the American Academy of Neurology (AAN) in Boston. These data were presented during the Clinical Trial Plenary Session by Jerry Mendell, MD, principal investigator in the trial and Curran-Peters Chair of Pediatric Research, Professor of Pediatrics and Neurology at the Research Institute at Nationwide Children’s Hospital and The Ohio State University, Columbus, Ohio.Results from the Phase 1 Trial of AVXS-101 in SMA Type 1 as Presented at AAN: Data as of January 20, 2017, showed no new events, and 15 of 15 (100%) patients were event-free at 13.6 months. The expected event-free survival rate at 13.6 months based on the natural history of the disease is 25%. The median age at last follow-up was 20.2 months and 30.8 months for patients in the proposed therapeutic-dose cohort (Cohort 2) and low-dose (Cohort 1) respectively.
AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new treatment-related safety or tolerability concerns identified.
Nutritional and Respiratory Support: According to natural history, nearly all Type 1 patients require nutritional and respiratory support by 12 months of age, and most SMA Type 1 babies are not able to swallow or speak effectively.
As of January 20, 2017, six of seven (86%) patients in Cohort 2 that did not require feeding support before treatment continued without feeding support after treatment; seven of 10 (70%) patients that did not require bi-level positive airway pressure (BiPAP) support before treatment continued without any BiPAP after treatment.
Further, as of January 20, 2017, 11 of 12 (92%) patients in Cohort 2 were fed orally, and six of 12 (50%) patients were exclusively fed orally; and eight of 12 (67%) patients were able to speak.

Motor Milestone Achievement

For the end-of-study assessment, AveXis evaluated three validated and well-established measures of sitting unassisted for periods of increasing duration. As of January 20, 2017, nine of 12 patients (75%) in Cohort 2 could sit unassisted for at least five seconds, seven of 12 patients (58%) could sit unassisted for at least 10 seconds and five of 12 patients (42%) could sit unassisted for 30 seconds or more.
Dr. Mendell today reported three patients in Cohort 2 have achieved additional sitting unassisted milestones since the January 20, 2017 evaluation date. Ten of 12 patients (83%) in Cohort 2 could sit unassisted for at least five seconds, nine of 12 (75%) patients could sit unassisted for at least 10 seconds and eight of 12 patients (67%) could sit unassisted for 30 seconds or more in the post-January 20 analysis.
As of January 20, 2017, two patients in Cohort 2 could crawl, pull to a stand, and stand and walk independently. - For the end-of-study assessment, all motor milestone achievements were assessed and adjudicated by an independent third-party reviewer using video evidence. The post-January 20 developmental milestones were confirmed internally by video. Detailed Cohort 2 motor milestone data is included in the chart below.


Cohort 2 2.0e14 vg/kg	Age at Gene Transfer (mos)	Motor Milestone Achievement as of January 20, 2017*
		Brings Hand to Mouth	Head Control	Partial Roll^a	Roll^b	Sitting with Assistance	Sitting Unassisted
		Brings Hand to Mouth	Head Control	Partial Roll^a	Roll^b	Sitting with Assistance	?5 seconds^c	?10 seconds^d	?30 seconds^e
E.04	6	X	X	X	X	X	X
E.05	4	X	X	X	X	X	X	X	X
E.06	2	X	X	X	X	X	X	X	X
E.07	4	X	X	X	X	X	X	X
E.08	8	X
E.09	5	X	X	X	X	X	X	X	X
E.10	1	X	X	X	X	X	X	X	X
E.11	2	X	X	X	X	X	X	O	O
E.12	3	X	X	X	X	X	X	X	X
E.13	1	X	X			X	X	X	O
E.14	4	X	X	X	X	X	O	O	O
E.15	2	X	X			X

a.	Bayley Scales of Infant and Toddler Development, item #20, rolls a minimum 180^o from back in only one direction.
b.	Bayley Scales of Infant and Toddler Development, item #20, rolls a minimum 180^o from back to both left and right.
c.	Sitting unassisted for ?5 seconds is in accordance with the criteria of item 22 in the Bayley Scales of Infant and Toddler Development – gross motor subtest and surpasses the three-second count used as a basis for sitting (test item 1) in the Hammersmith Functional Motor Scale – Expanded for SMA (HFMSE).
d.	Sitting unassisted for ?10 seconds is in accordance with the criteria in the World Health Organization – MultiCentre Growth Reference Study.
e.	Sitting unassisted for ?30 seconds defines functional independent sitting and is in accordance with the criteria of item 26 in the Bayley Scales of Infant and Toddler Development – gross motor subtest.
	*Milestone achievements as of January 20, 2017, except those indicated by an O, which were achieved after the January 20, 2017 cutoff.

“Babies born with SMA Type 1 that are untreated will never reach or maintain developmental milestones, which is why the videos presented today at AAN showing the achievement of motor milestones after a one-time infusion of AVXS-101 – including the majority of patients in Cohort 2, the proposed therapeutic-dose cohort, who were able to roll and sit unassisted – is incredibly remarkable,” said Dr. Mendell. “Importantly, AVXS-101 appears to demonstrate continued improvements past 13.6 months of age, with three new patients reaching sitting thresholds since the January 20, 2017, evaluation date.” Research Presented at AAN Further Scientific Understanding of AVXS-101 Brian Kaspar, PhD, Senior Vice President and Chief Scientific Officer of AveXis, will present his poster today, “CSF Delivery of AAV9-mediated Gene Therapy for SMA, a Lethal Neuromuscular Disease in Children: A Dose-response Study in Mice and Nonhuman Primates -- data regarding a dose-response study for the CSF delivery of AVXS-101,” data from which support the use of intrathecal delivery of gene therapy for neurological diseases, such as SMA Types 2 and 3. The study offers insight into vector distribution and its correlation with transgene expression and provides guidance for future AAV9-based clinical trials in SMA, as well as other neurodegenerative disorders.

• On March 16, 2017, AveXis reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The Phase 1, open-label, dose-escalating study was designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. As of January 20, 2017, AVXS-101 appeared to have a favorable safety profile and to be generally well tolerated, with no new safety or tolerability concerns identified. As has been previously reported, a total of five adverse events (AEs) in four patients were deemed treatment-related. Of these, two were serious adverse events (SAEs) experienced by two patients, and three were non-serious AEs experienced by two patients. All consisted of clinically asymptomatic liver enzyme elevations and were resolved with prednisolone treatment. There were no clinically significant elevations of gamma-glutamyl transferase, alkaline phosphatase or bilirubin and, as such, Hy’s Law was not met. Other non-treatment-related AEs were expected and were associated with SMA.
As of January 20, 2017, 11 of 12 patients (92%) in Cohort 2 achieved head control, nine of 12 patients (75%) could roll a minimum of 180 degrees from back to both left and right, and 11 of 12 patients (92%) could sit with assistance.
A cumulative total of 256 AEs (five treatment-related AEs and 251 non-treatment related AEs) were reported as of January 20, 2017, following monitoring and source verification. Of these, 52 were determined to be SAEs and 204 were non-serious AEs. As previously noted, two of the 52 SAEs were deemed treatment-related.
There were 65 new AEs reported after September 15, 2016, of which 10 were SAEs in three patients and were associated with SMA and were not deemed treatment-related.
No New Events and 15 of 15 Patients Event-Free at 13.6 Months, including 12 of 12 Patients in Proposed Therapeutic-Dose Cohort: As of January 20, 2017, 12 of 12 patients (100%) in the cohort of patients who received the proposed one-time therapeutic dose of AVXS-101 (Cohort 2) had reached 13.6 months of age event-free, where the expected event-free survival rate based on natural history of the disease is 25%. The median age at last follow-up for Cohort 2 was 20.2 months, with the oldest patient at 31.1 months of age.
As of January 20, 2017, 9 of 9 patients -- 3 in the low-dose cohort (Cohort 1) and 6 in Cohort 2 -- reached 20 months of age event free, where the expected event-free rate based on natural history of the disease is 8%.
As of January 20, 2017, three patients in Cohort 1 reached 13.6 months of age event-free. As has been previously reported, one patient in Cohort 1 had a pulmonary event in the third quarter of 2016. The patient had increased use of bi-level positive airway pressure (BiPAP) in advance of surgery related to hypersalivation, a condition experienced by some SMA patients; the event was determined upon independent review to represent progression of disease and not to be related to the use of AVXS-101. This patient completed the final trial visit in September 2016, and as of that time BiPAP use was below the event threshold.
Rapid and Sustained CHOP INTEND Improvements Above Baseline: As of January 20, 2017, mean increases from baseline in CHOP INTEND scores of 7.7 points in Cohort 1 and 24.7 points in Cohort 2 were observed, reflecting improvement in motor function. In Cohort 2 there were mean increases in CHOP INTEND of 9.8 points one month after gene therapy and 15.4 points three months following gene therapy.
11 out of 12 patients (92%) in Cohort 2 achieved CHOP INTEND scores of at least 40 points.
10 out of 12 patients (83%) in Cohort 2 achieved CHOP INTEND scores of at least 50 points.
2 out of 12 patients (17%) in Cohort 2 achieved CHOP INTEND scores of at least 60, which is in a range considered to be normal. These two patients achieved the maximum CHOP INTEND score of 64.
Cohort 2 Patients Consistently Achieved and Maintained Key Developmental Motor Milestones: As of January 20, 2017, 11 of 12 patients (92%) in Cohort 2 achieved head control, nine of 12 patients (75%) could roll a minimum of 180 degrees from back to both left and right, and 11 of 12 patients (92%) could sit with assistance. For the end-of-study assessment, AveXis evaluated three validated and well-established measures of sitting unassisted for periods of increasing duration. Nine of 12 patients (75%) could sit unassisted for at least five seconds, seven of 12 patients (58%) could sit unassisted for at least 10 seconds and five of 12 patients (42%) could sit unassisted for 30 seconds or more. Two patients could walk independently, and each had achieved earlier and important developmental milestones such as standing with support, standing alone and walking with support.
Detailed proposed therapeutic-dose cohort motor milestone data is included in the chart below:

Cohort 2 2.0e14 vg/kg	Age at Gene Transfer (mos)	Motor Milestone Achievement as of January 20, 2017
		Brings Hand to Mouth	Head Control	Partial Roll^a	Roll^b	Sitting with Assistance	Sitting Unassisted
		Brings Hand to Mouth	Head Control	Partial Roll^a	Roll^b	Sitting with Assistance	?5 seconds^c	?10 seconds^d	?30 seconds^e
E.04	6	X	X	X	X	X	X
E.05	4	X	X	X	X	X	X	X	X
E.06	2	X	X	X	X	X	X	X	X
E.07	4	X	X	X	X	X	X	X
E.08	8	X
E.09	5	X	X	X	X	X	X	X	X
E.10	1	X	X	X	X	X	X	X	X
E.11	2	X	X	X	X	X	X
E.12	3	X	X	X	X	X	X	X	X
E.13	1	X	X			X	X	X
E.14	4	X	X	X	X	X
E.15	2	X	X			X

a. Bayley Scales of Infant and Toddler Development, item #20, rolls a minimum 180^o from back in only one direction.
b. Bayley Scales of Infant and Toddler Development, item #20, rolls a minimum 180^o from back to both left and right.
c. Sitting unassisted for ?5 seconds is in accordance with the criteria of item 22 in the Bayley Scales of Infant and Toddler Development – gross motor subtest and surpasses the three second count used as a basis for sitting (test item 1) in the Hammersmith Functional Motor Scale – Expanded for SMA (HFMSE).
d. Sitting unassisted for ?10 seconds is in accordance with the criteria in the World Health Organization – MultiCentre Growth Reference Study.
e. Sitting unassisted for ?30 seconds defines functional independent sitting and is in accordance with the criteria of item 26 in the Bayley Scales of Infant and Toddler Development – gross motor subtest.

For the end-of-study assessment, all motor milestone achievements above were assessed and adjudicated by an independent third-party reviewer using video evidence.
• On October 8, 2016, AveXis provided an update on interim data from the ongoing Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 as of September 15, 2016. The data were presented by Jerry Mendell, M.D., director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, at the 21st International Annual Congress of the World Muscle Society in Granada, Spain. For the first time, interim data from the trial were presented that highlighted patient achievement of key motor development milestones as of September 15, 2016. Two-thirds of patients in Cohort 2 (the proposed therapeutic dose) had achieved the ability to sit unassisted, including one patient whose achievement of this milestone was confirmed after September 15. In Cohort 2, 11 of 12 patients achieved head control, 7 of 12 patients could roll over completely and 11 of 12 patients could sit with support. Two patients are now walking independently, including one whose achievement of this milestone was confirmed after September 15. These two patients each achieved earlier and important developmental milestones such as crawling, standing with support, standing alone and walking with support.
Data as of September 15, 2016 showed AVXS-101 continued to demonstrate a favorable safety profile and was generally well tolerated, with no new treatment-related safety or tolerability concerns identified. There has been a cumulative total of 118 adverse events (AEs) reported as of September 15, 2016, 34 of which were determined to be serious adverse events (SAEs) and 84 were determined to be non-serious AEs. As previously reported, a total of 5 AEs in 4 patients were treatment-related. Two were deemed treatment-related SAEs (experienced by 2 patients) and three were deemed non-serious AEs (experienced by 3 patients). All consisted of clinically asymptomatic liver enzyme elevations. All of the elevated liver enzyme AEs and SAEs were clinically asymptomatic and resolved with prednisolone treatment. There were no clinically significant elevations of gamma-glutamyl transferase (GGT), alkaline phosphatase or bilirubin, and as such Hy’s Law was not met. Other non-treatment-related AEs were expected and were associated with SMA. All patients in Cohort 2 (proposed therapeutic dose) are event-free, defined as death or requiring at least 16 hours per day of ventilation support for breathing for greater than two weeks in the absence of an acute reversible illness, or perioperatively. The median age at last follow-up for Cohort 2 is 17.3 months, with the oldest patient at 27.4 months of age. As previously reported, one patient in Cohort 1 (the low-dose cohort) did have a pulmonary event after July 1, 2016. The patient had increased use of bi-level positive airway pressure (BiPAP) in advance of surgery related to hypersalivation, a condition experienced by some SMA patients; the event was determined by independent review to represent progression of disease and not to be related to the use of AVXS-101. Mean increases in CHOP-INTEND scores of 9.0 points in Cohort 1 and 24.8 points in Cohort 2 were observed, reflecting improvement in motor function. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) is a test developed to measure motor skills of patients with SMA Type 1. 11 out of 12 patients in Cohort 2 achieved CHOP-INTEND scores of at least 40 points. 9 out of 12 patients in Cohort 2 achieved CHOP-INTEND scores of at least 50 points. 3 out of 12 patients in Cohort 2 achieved CHOP-INTEND scores of at least 60, which is in a range considered to be normal. Patients on the proposed therapeutic dose of AVXS-101 consistently achieved and maintained key developmental motor milestones. As of September 15, 2016, 11 out of 12 patients achieved head control; 7 out of 12 patients could roll over (completely); 11 out of 12 patients could sit with support; and 8 out of 12 patients could sit unassisted, including one patient whose achievement of this milestone was confirmed after September 15. In addition, 7 patients are able to feed themselves, including one patient whose achievement of this milestone was confirmed after September 15, and 5 patients are speaking (1 bilingual). 4 patients are now standing with support, including two whose achievements of this milestone were confirmed after September 15. 2 patients are now walking independently, including one whose achievement of this milestone was confirmed after September 15. These two patients each achieved earlier and important developmental milestones such as crawling, standing with support, standing alone and walking with support. Detailed Cohort 2 motor milestone data is included in the chart below:

Motor Milestone Achievement as of September 15, 2016

Cohort 2

2.0e14 vg/kg

Age at Gene Transfer (mos)

Brings Hand to Mouth

Head Control

Rolls Over (partial)

Rolls Over (complete)

Sitting with

Assistance

Sitting

Unassisted

E.04

E.05

E.06

E.07

E.08

E.09

E.10

E.11

E.12

E.13

E.14

E.15

* Achievement confirmed after Sept 15, 2016

• On May 16, 2016, AveXis reported pulmonary results from an interim analysis of data as of April 1, 2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. The data were presented by Richard Shell, MD, member of the Section of Pulmonary Medicine at Nationwide Children’s Hospital, at the International Conference of the American Thoracic Society 2016 in San Francisco. In the interim analysis presented at the meeting, gene therapy appeared to reduce the need for ventilation support and allowed patients to successfully recover from respiratory illnesses that are often lethal to SMA Type 1 patients. The interim analysis found that none of the patients in either dosing cohort required permanent ventilation as of April 1, 2016. In addition, 8 of 10 (80 percent) patients on the proposed therapeutic dose of 2.0x1014 vg/kg who did not need biphasic/bi-level ventilation (BiPAP)

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