– Patients who received a single dose of
AVXS-101 resulted in longer survival, superior achievement of motor
milestones, and better motor function than in historical cohorts
–
– As of August 7, 2017, all patients are alive,
event-free and have reached at least 20 months of age –
– As of August 7, 2017, all patients who
received the proposed therapeutic dose of AVXS-101 demonstrated
continued treatment durability and achievement of motor milestones,
including 75 percent of these patients now sitting for 30 seconds
or longer –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy
company developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, 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 today 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 publication may be found online at
www.nejm.org/doi/full/10.1056/NEJMoa1706198.
“It is incredibly encouraging to see that all children who have
received AVXS-101 remain event-free and demonstrate a durable
treatment effect at 20 months of age and older, including in many
cases achievement of new developmental milestones,” Sean Nolan,
President and Chief Executive Officer of AveXis. "To have these
Phase 1 data published in the prestigious New England Journal of
Medicine highlights the groundbreaking work of Dr. Jerry Mendell
and his team and further validates the clinically transformative
nature of gene therapy in children with SMA Type 1.”
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
2
Age at
Gene
Transfer
(mos)
Event-
Free
Survivala
Event-free Survival and Motor and Other Milestones Among
the 12 Patients in Cohort 2 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 + +
+ + + + + +
Patient with
outcome (%)
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 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.
“AveXis and my team at Nationwide Children’s Hospital have
worked tirelessly to alter the inevitable course of SMA Type 1,”
Jerry Mendell, MD, principal investigator in the Center for Gene
Therapy at Nationwide Children’s Hospital. “We are proud to see
these data published in New England Journal of Medicine,
representing the culmination of decades of dedication and
commitment to making a better life possible for the children
diagnosed with this unforgiving condition.”
AVXS-101 is currently being evaluated in a pivotal trial for the
treatment of SMA Type 1.
About SMA
SMA is a severe neuromuscular disease characterized by the loss
of motor neurons leading to progressive muscle weakness and
paralysis. SMA is caused by a genetic defect in the SMN1 gene that
codes SMN, a protein necessary for survival of motor neurons. The
incidence of SMA is approximately one in 10,000 live births and is
the leading genetic cause of infant mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder
characterized by motor neuron loss and associated muscle
deterioration, which results in mortality or the need for permanent
ventilation support before the age of two for greater than 90
percent of patients. SMA Type 2 typically presents between six and
18 months of age and affected patients can sit unassisted but never
walk or stand without support.
About AVXS-101
AVXS-101 is a proprietary gene therapy candidate of a one-time
treatment for SMA Types 1 and 2, designed to address the monogenic
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 crossing the blood brain barrier to allow effective
targeting of both central and systemic features.
About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing
treatments for patients suffering from rare and life-threatening
neurological genetic diseases. The company’s initial proprietary
gene therapy candidate, AVXS-101, is in the pivotal phase of study
for the treatment of SMA Type 1, and a Phase 1/2a study for SMA
Type 2. The company also intends to expand the study of gene
therapy into two additional rare neurological monogenic disorders:
Rett syndrome (RTT) and a genetic form of amyotrophic lateral
sclerosis (ALS) caused by mutations in the superoxide dismutase 1
(SOD1) gene.
For additional information, please visit www.avexis.com.
Forward-Looking Statements
This press release contains "forward-looking statements," within
the meaning of the Private Securities Litigation Reform Act of
1995, regarding, among other things, AveXis’ research, development
and regulatory plans for AVXS-101, including the potential of
AVXS-101 to alter the course of disease in patients with SMA Type
1, AveXis’ ongoing clinical trial of AVXS-101 in SMA Type 1 and
plans to expand AveXis’ research, development efforts to explore
potential treatments of RTT and genetic ALS. Such forward-looking
statements are based on current expectations and involve inherent
risks and uncertainties, including factors that could delay, divert
or change any of them, and could cause actual results to differ
materially from those projected in its forward-looking statements.
Meaningful factors which could cause actual results to differ
include, but are not limited to, the scope, progress, expansion,
and costs of developing and commercializing AveXis’ product
candidates; regulatory developments in the U.S. and EU, as well as
other factors discussed in the "Risk Factors" and the "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of AveXis’ Annual Report on Form 10-K for the
year ended December 31, 2016, filed with the SEC on March 16, 2017,
and AveXis’ Quarterly Report on Form 10-Q for the quarter ended
June 30, 2017, filed with the SEC on August 10, 2017. In addition
to the risks described above and in the Annual Reports on Form
10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K
and other filings with the SEC, other unknown or unpredictable
factors also could affect AveXis’ results. There can be no
assurance that the actual results or developments anticipated by
AveXis will be realized or, even if substantially realized, that
they will have the expected consequences to, or effects on, AveXis.
Therefore, no assurance can be given that the outcomes stated in
such forward-looking statements and estimates will be achieved.
All forward-looking statements contained in this press release
are expressly qualified by the cautionary statements contained or
referred to herein. AveXis cautions investors not to rely too
heavily on the forward-looking statements AveXis makes or that are
made on its behalf. These forward-looking statements speak only as
of the date of this press release (unless another date is
indicated). AveXis undertakes no obligation, and specifically
declines any obligation, to publicly update or revise any such
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by law.
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Media Inquiries:W2O GroupLauren Barbiero,
646-564-2156lbarbiero@w2ogroup.comorInvestor Inquiries:AveXis,
Inc.Jim Goff, 650-862-4134jgoff@avexis.com
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