-- Jerry Mendell, MD, Presented Data as of April
1, 2016 at the American Society of Gene & Cell Therapy 19th
Annual Meeting --
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing novel treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today presented 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. Jerry Mendell, MD, director of the Center for Gene
Therapy at The Research Institute at Nationwide Children’s
Hospital, presented the data at the 19th Annual Meeting of the
American Society of Gene & Cell Therapy in Washington, D.C.
The data reported at the meeting show that AVXS-101 continues to
demonstrate a favorable safety profile in patients studied, with no
new treatment-related safety or tolerability concerns identified;
all patients in both the low-dose and proposed therapeutic-dose
cohorts remain without an “event,” defined as death or until a
patient requires at least 16 hours per day of ventilation support
for breathing for 14 consecutive days in the absence of an acute
reversible illness, or perioperatively; and the mean motor function
score continues to increase, with two patients having achieved
motor function in a range considered to be normal.
Dr. Mendell said, “Given the rapid and devastating disease
course of SMA Type 1, it is very encouraging to see that all
patients in both dosing cohorts have remained event free, and all
patients have demonstrated sustained improvements above baseline in
motor function since receiving AVXS-101. Additionally, new
data presented today appear to indicate that AVXS-101 may have a
positive impact on both the pulmonary and nutritional support of
patients in the trial suffering from SMA Type 1, which may be
impacting the overall survival benefit.”
The ongoing Phase 1 study is designed to evaluate safety and
preliminary indications of efficacy of AVXS-101 in patients
suffering from SMA Type 1. Data as of April 1, 2016 has shown:
- AVXS-101 appears to have a favorable safety profile and to be
generally well tolerated in patients studied. There has been a
total of 74 adverse events (AEs) reported, 22 of which were
determined to be serious adverse events (SAEs). Two of the 22 were
deemed treatment-related and, as previously reported, those SAEs
were clinically asymptomatic liver enzyme elevations. Of the 52
non-serious AEs, 3 were treatment-related elevations in liver
enzymes experienced by two patients. All elevated liver enzyme AEs
and SAEs were resolved with prednisolone treatment. Other
non-treatment-related AEs were expected and were associated with
SMA.
- No patients in either dosing cohort have experienced an
“event.” The median event-free age of all 15 patients was 14.9
months, and the median event-free age of patients in Cohort 1 was
25.7 months and in Cohort 2 was 11.7 months. The natural history of
the disease indicates that 25 percent of untreated SMA Type 1
patients survive event-free at 13.6 months of age and that 8
percent survive event-free at 20 months of age1.
- Mean increases of 8.7 points and 19.2 points in CHOP-INTEND
scores were observed in Cohort 1 and Cohort 2, respectively. Nine
out of 12 patients (75 percent) and 7 out of 12 patients (58
percent) in Cohort 2 have achieved CHOP-INTEND scores of at least
40 points or 50 points, respectively. Two patients achieved the
maximum score of 64. A score of 60 is considered normal. 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.
- The natural history of SMA Type 1 indicates that bulbar
weakness leads to impaired swallowing, malnutrition and growth
failure. The median age to growth failure is 7 months of age2. The
median age to nutritional support is 8 months of age (IQR 6-13
months)1. AVXS reported today that 7 of 7 (100%) of AVXS-101
patients that did not require feeding support before treatment
continued without feeding support as of April 1, 2016 in the
ongoing Phase 1 trial. Five patients had gastric feeding tube
placement prior to gene therapy. One patient in Cohort 2 who had
gastric feeding tube placement prior to gene transfer is also
feeding orally.
- The natural history of SMA Type 1 indicates that bulbar muscle
weakness, skeletal muscle weakness in the neck and intercostal
muscle weakness lead to respiratory impairment, poor clearance of
airway secretions, risk of aspiration and recurrent infections
leading to death or permanent ventilation. The median age to
permanent ventilation or death is 10.5 months (IQR 8.1-13.6
months), and by 13.6 months only 25 percent of SMA Type 1 patients
are alive and free of permanent ventilation1. In the ongoing trial,
8 of 10 (80%) of AVXS-101 patients that did not use
biphasic/bi-level ventilation (BiPAP) support before gene transfer
continue without any ventilation support as of April 1, 2016 (the 2
exceptions being after severe illness/hospitalizations to assist
recovery).
“We are encouraged by these interim results as we work
diligently to bring AVXS -101 to patients who suffer from SMA Type
1, a devastating disease for which there are currently no
FDA-approved therapies,” said Suku Nagendran, MD, Senior Vice
President and Chief Medical Officer, AveXis. “We look forward to
reviewing the ongoing data from this study over the coming year as
we continue the development of AVXS-101.”
Webcast at 4:30 p.m. EDTAveXis will host a live
audio webcast of a conference call to discuss the interim Phase 1
trial data today at 4:30 p.m. EDT. Analysts and investors can
participate in the conference call by dialing (877) 508-0547 for
domestic callers and (615) 247-5963 for international callers,
using the conference ID 95576133. The webcast can be accessed live
on the Events and Presentations page in the Investors and Media
section of the AveXis website, www.AveXis.com. The webcast will be
archived on the company’s website for 30 days and will be available
for telephonic replay for 14 days following the call by dialing
(855) 859-2056 (Domestic) or (404) 537-3406 (International),
conference ID 95576133.
Phase 1 Trial DesignThe Phase 1 open-label,
dose-escalation study is designed to evaluate safety and
preliminary indications of 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 efficacy
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.
The trial has fully-enrolled with a total of 15 patients who met
enrollment criteria of diagnosis of SMA Type 1 before six months of
age, with two copies of the SMN2 backup gene, as determined by
genetic testing. The trial includes two dosing cohorts:
- Cohort 1 (low dose) includes three patients dosed at (6.7 X1013
vg/kg), aged 5.9 to 7.2 months at time of dosing;
- Cohort 2 (proposed therapeutic dose) includes 12 patients dosed
at (2.0 X1014 vg/kg), aged 0.9 to 7.9 months at time of
dosing.
About SMASMA 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.
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 1 is the leading genetic cause of
infant mortality.
About AVXS-101 AVXS-101 is a proprietary gene
therapy candidate of a one-time, intravenous 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 intravenous (IV) dosing route
and 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 an ongoing Phase 1 clinical trial for the treatment of SMA Type
1. For additional information, please visit www.avexis.com.
1. Finkel, R. et al. Observational study of spinal
muscular atrophy type I and implications for clinical
trials. Neurology 83,810–817 (2014).2.
Sproule, D. et al. Age at Disease Onset Predicts Likelihood and
Rapidity of Growth Failure Among Infants and Young Children with
Spinal Muscular Atrophy Types 1 and 2. J Child Neurol 27, 845-851
(2012).
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 expected timing for reporting results
from the ongoing Phase 1 clinical trial and the potential for
AVXS-101 to positively impact the pulmonary and nutritional support
of patients suffering from SMA Type 1. 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 United States and
foreign countries, as well as other factors discussed in the "Risk
Factors" and "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, 2015, filed
with the SEC on March 18, 2016. 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.
Media Inquiries:
Lauren Barbiero
W2O Group
646-564-2156
lbarbiero@w2ogroup.com
Investor Inquiries:
Jim Goff
AveXis, Inc.
650-862-4134
jgoff@avexis.com
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