-- Majority of patients on the proposed
therapeutic dose achieved key developmental milestones including
sitting unassisted; two patients walking independently --
-- Conference call and webcast October 10 at
8:30 a.m. EDT --
AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy
company developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today 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.
“To date, the majority of patients who received the proposed
therapeutic dose of AVXS-101 have achieved key milestones and
two-thirds of these patients can sit independently – a fact
completely inconsistent with the known disease course, as children
with untreated SMA Type 1 will never sit unassisted,” said Sean
Nolan, President and Chief Executive Officer, AveXis. “We are
encouraged by these interim data, and continue to work diligently
to bring this gene therapy to the children suffering from this
devastating condition.”
Interim Phase 1 Data as of September 15, 2016
- 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 6 X X X X X
E.05 4 X X X X X X
E.06 2 X X X X X X
E.07 4 X X X X X
E.08 8 X
E.09 5 X
X X X X X
E.10 1 X X X X X X
E.11 2 X X X X X*
E.12 3 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
* Achievement confirmed after Sept 15, 2016
“The preliminary clinical observations of extended event-free
survival, sustained increases in motor function and achievement of
developmental milestones in patients receiving a one-time infusion
of AVXS-101 have far exceeded what has been observed with natural
history,” said Suku Nagendran, MD, Senior Vice President and Chief
Medical Officer, AveXis. “These preliminary results demonstrate the
potential of AVXS-101 to positively impact quality of life and as
such alter the course of disease in these children with SMA Type
1.”
Conference Call Information
AveXis will host a conference call and webcast at 8:30 a.m.
EDT Monday, October 10, 2016, to discuss the AVXS-101 clinical
update.
Analysts and investors can participate in the conference call by
dialing (844) 889-6863 for domestic callers and (661) 378-9762 for
international callers, using the conference ID 93427499. 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 5 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 93427499.
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.
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
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.
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.
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 positively impact quality of life and alter the course
of disease in children with 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" included as Exhibit 99.1 to the Company’s Current Report
on Form 8-K filed with the Securities and Exchange Commission on
September 7, 2016 and the "Management's Discussion and Analysis of
Financial Condition and Results of Operations" section of AveXis’
Annual Report on Form 10-Q for the quarter ended June 30, 2016,
filed with the SEC on August 12, 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.
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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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