– AveXis on track to submit potency assay data to
FDA in August –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today reported
financial results for the second quarter ended June 30, 2017,
recent corporate highlights and upcoming milestones.
“It was another eventful quarter for AveXis with
important progress made across multiple fronts, including alignment
with the FDA on our GMP manufacturing process, presentation of
closeout data from our Phase 1 study of AVXS-101 in SMA Type 1, the
execution of licensing agreements for the use of AAV9 for two rare
neurological monogenic disorders, and an increase in our cash
position with the close of a recent public offering,” said Sean
Nolan, President and Chief Executive Officer of AveXis. “Looking
ahead, we remain on track to have the data from the potency assay
work ready to submit to the FDA as planned and to conduct an
end-of-Phase 1 meeting with the FDA to further inform the
regulatory pathway for AVXS-101.”
Recent Highlights
Presented AVXS-101 research at the Annual
Meeting of the American Academy of Neurology
(AAN): On April 25, Jerry Mendell, M.D., principal
investigator in the Phase 1 study of AVXS-101 in spinal muscular
atrophy (SMA) Type 1 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, presented results from the
closeout of the study during a plenary session at AAN, including
video evidence of children achieving motor milestones.
AveXis presented other research at AAN that
furthered the scientific understanding of AVXS-101:
- Dr. Brian Kaspar, Senior Vice President and Chief
Scientific Officer of AveXis, presented data from a
dose-response study for the cerebrospinal fluid delivery of
AVXS-101 that offers insight into vector distribution and its
correlation with transgene expression, providing guidance for
future AAV9-based clinical trials in SMA, as well as other
neurodegenerative disorders.
- Dr. Douglas Sproule, Vice President of Clinical
Development and Medical Affairs of AveXis, presented data
suggesting pre-existing antibodies to AAV9 are quite uncommon in
the pediatric population and should not impact use of gene therapy
for the vast majority of SMA Type 1 patients.
- Dr. Linda Lowes, Director of Clinical Therapies
Research and a member of the Center for Gene
Therapy at the Research Institute of Nationwide Children’s
Hospital, presented research providing further evidence of the
correlation between motor function and motor milestone achievement
in patients with SMA Type 1, demonstrating that the degree of
treatment outcome appears to be influenced by age at dosing and
baseline motor function.
Announced Exclusive Worldwide License
Agreement for Two Rare Neurological Monogenic Disorders Using NAV
AAV9 Vector: On June 7, AveXis and REGENXBIO Inc.
announced an exclusive worldwide license agreement for AveXis to
develop and commercialize gene therapy treatments using REGENXBIO’s
NAV AAV9 vector to treat Rett syndrome and a genetic form of
amyotrophic lateral sclerosis (ALS) caused by mutations in the
superoxide dismutase 1 gene. AveXis intends to provide more details
on these programs in the second half of 2017.
Announced Alignment with U.S. Food and Drug
Administration (FDA) on Good Manufacturing Practice (GMP)
Commercial Manufacturing Process: On June 14, AveXis
announced alignment with the FDA regarding the process for
producing the intended commercial scale GMP-derived gene therapy
product, following the receipt of minutes from the Type B Chemistry
Manufacturing and Controls meeting held on May 1. This alignment
includes support for the proposed commercial manufacturing process,
the proposed analytical methods and corresponding qualification and
validation plans – inclusive of key release assays such as potency,
purity and identity – and the proposed comparability protocol,
which helps assess how similar the product derived from the GMP
process is to the original product used in the Phase 1 trial of
AVXS-101 in patients with SMA Type 1.
- In the meeting minutes, the FDA made a request that the company
complete implementation of its potency assay qualification plan,
presented in the meeting, prior to initiation of upcoming clinical
studies. This assay utilizes the Delta 7 mouse model, which has
been used historically to assess AVXS-101 potency but now
incorporates additional elements to make it acceptable to global
regulatory authorities. The company has already initiated the work
necessary to address this request and expects to have the data
ready to submit to the FDA in August.
- AveXis plans to initiate a pivotal study trial of AVXS-101 in
SMA Type 1 in the U.S. and a Phase 1/2a trial of AVXS-101 in SMA
Type 2 in the U.S. later in the third quarter of 2017, pending
agreement from the FDA that these data are sufficient. AveXis
expects to provide an update once the company has received FDA
agreement that these data are sufficient to initiate the studies.
The company also expects to provide design details for these
studies at that time.
- Additionally, FDA is aligned with the company’s proposed
comparability protocol to assess the similarity of key
characteristics of the Nationwide Children’s Hospital product, used
in the Phase 1 SMA Type 1 study, with the product derived from the
new GMP manufacturing process. Data from this comparability work is
ongoing and will include the above-mentioned potency qualification
data, which will be incorporated into the data package along with
the full Phase 1 clinical data that will be reviewed and discussed
at the upcoming end-of-Phase 1 meeting. This meeting will help
further inform the regulatory pathway options for AVXS-101. The
company anticipates providing an update on the outcome of that
meeting once the official minutes are available, which is
anticipated to be in the fourth quarter of 2017.
Announced Raise of $269.7 Million in Public
Offering: On June 26, 2017, AveXis closed an underwritten
public offering of 4,111,250 shares of its common stock at a public
offering price of $70.00 per share before underwriting discounts
and commissions, including 536,250 shares sold pursuant to the
underwriters’ full exercise of their option to purchase additional
shares. The net proceeds to AveXis from the offering, after
deducting the underwriting discounts and commissions and other
offering expenses payable by AveXis, were $269.7 million.
Second Quarter 2017 Financial
Results
- Cash Position: As of June 30, 2017, AveXis had
$417.6 million in cash and cash equivalents.
- R&D Expenses: Research and development
expenses were $45.2 million for the second quarter of 2017 (which
included $3.7 million of non-cash stock-based compensation
expense), compared to $10.4 million for the same period in 2016
(which included $1.3 million of non-cash stock-based compensation
expense), resulting in an increase of $34.8 million. The increase
was primarily attributable to product manufacturing expenses and
associated accelerated spending, including increased headcount, in
the company’s product manufacturing facility, as well as expenses
related to the conclusion of the Phase 1 clinical trial of AVXS-101
in SMA Type 1, licensing fees related to the company’s planned new
programs in Rett syndrome and genetic ALS and an increase in
non-cash stock-based compensation expense.
- G&A Expenses: General and administrative
expenses were $13.2 million for the second quarter of 2017 (which
included $4.5 million of non-cash stock-based compensation
expense), compared to $5.4 million for the same period in 2016
(which included $2.6 million of stock-based compensation expense),
resulting in an increase of $7.8 million. The increase was
primarily attributable to increases in salaries and
personnel-related costs; legal, professional and consulting fees;
other administrative costs driven by increased headcount across all
general and administrative functions to support the company’s
overall growth, and non-cash stock-based compensation expense.
- Net Loss: Net loss was $58.0 million, or $2.07
per share, for the second quarter of 2017, compared to a net loss
of $15.7 million, or $0.68 per share, for the second quarter of
2016.
Selected Financial Information (unaudited) |
|
Operating Results: |
|
|
|
|
|
|
|
|
|
|
(In
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
Revenue |
|
|
|
|
|
|
|
|
|
|
|
Total revenue |
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
Expenses: |
|
|
|
|
|
|
|
|
|
General and administrative |
|
13,154 |
|
|
|
5,418 |
|
|
|
22,803 |
|
|
|
10,242 |
|
|
Research and development |
|
45,206 |
|
|
|
10,380 |
|
|
|
65,521 |
|
|
|
26,445 |
|
|
Total Operating Expenses |
|
58,360 |
|
|
|
15,798 |
|
|
|
88,324 |
|
|
|
36,687 |
|
|
Loss from
operations |
|
|
(58,360 |
) |
|
|
(15,798 |
) |
|
|
(88,324 |
) |
|
|
(36,687 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income |
|
|
331 |
|
|
|
79 |
|
|
|
576 |
|
|
|
132 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net
loss |
|
$ |
(58,029 |
) |
|
$ |
(15,719 |
) |
|
$ |
(87,748 |
) |
|
$ |
(36,555 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average basic
and diluted common shares outstanding |
|
|
|
|
27,971,733 |
|
|
|
23,013,838 |
|
|
|
27,850,199 |
|
|
|
19,876,850 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted net loss per common share |
$ |
(2.07 |
) |
|
$ |
(0.68 |
) |
|
$ |
(3.15 |
) |
|
$ |
(1.84 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheet Information: |
|
|
|
|
|
|
|
|
|
(In
thousands) |
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
|
|
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
Cash and cash equivalents |
|
|
|
|
$ |
417,620 |
|
|
$ |
240,430 |
|
|
Total assets |
|
|
|
|
|
|
469,307 |
|
|
|
270,575 |
|
|
Total liabilities |
|
|
|
|
|
|
27,884 |
|
|
|
24,444 |
|
|
Accumulated deficit |
|
|
|
|
$ |
(229,310 |
) |
|
$ |
(141,562 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Conference Call InformationAnalysts 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 62331685. 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 until its next
quarterly earnings call, 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
62331685.
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-101AVXS-101 is a
proprietary gene therapy candidate of a one-time treatment for SMA
Type 1 and is 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 crosses the blood
brain barrier allowing an IV dosing route and effective targeting
of both central and systemic features.
About Rett SyndromeRett syndrome
(RTT) is a devastating, rare neurological disorder characterized by
slowed growth, loss of normal movement and coordination and loss of
communication skills. RTT is caused by an X-linked dominant
mutation in the methyl CpG binding protein 2 (MECP2) gene, which
results in problems with the protein production critical for brain
development. RTT occurs in approximately one of every 10,000 female
births and usually begins to show signs and symptoms in infants
between six and 18 months of age. Current treatments only offer
symptomatic relief and do not target the genetic cause of the
disease, leaving a significant unmet need.
About Genetic Amyotrophic Lateral
SclerosisAmyotrophic lateral sclerosis (ALS) is a
progressive neurodegenerative disease that affects nerve cells in
the brain and the spinal cord. Familial or inherited forms of ALS
reflect five to 10 percent of ALS cases, or approximately one to
two thousand people in the U.S., and can be caused by mutations in
several genes known to be associated with ALS. Approximately 20
percent of these cases are caused by mutations in the gene that
produces the copper zinc superoxide dismutase 1 (SOD1) enzyme,
which leads to a progressive degeneration of motor neurons
affecting movement and muscle control. ALS usually occurs in people
between the ages of 40 and 70. Current treatments only offer modest
benefits and do not target the genetic cause of the disease,
leaving a significant unmet need.
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 StatementsThis
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, expectations regarding AveXis’
research, development and regulatory plans for its programs for
treatment of RTT and genetic ALS, its expectations regarding
initiation of IND-enabling studies for these programs and timing of
providing an update on these programs, the overall clinical
development of AVXS-101, AveXis’ research, development and
regulatory plans for AVXS-101, including AveXis’ commercial
manufacturing process, AveXis’ potency assay development and the
expected timing of the submission of data regarding the potency
assay to the FDA, the expected timing of the initiation of AveXis’
planned future clinical trials, the expected timing of future
meetings with the FDA, AveXis’ ability to meet future commercial
demand for AVXS-101 through its manufacturing facility and the
possibility that AveXis may be able to utilize data from its Phase
1 clinical trial in the regulatory pathway of AVXS-101. 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" section of AveXis’ Annual
Report on Form 10-K for the year ended December 31, 2016, filed
with the SEC on March 16, 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.
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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