– On track to request pre-BLA meeting with FDA in
Q2 2018 –– Intends to initiate pivotal trial in SMA Type 1 in
Europe and multi-national pre-symptomatic SMA study in the first
half of 2018 – – Anticipates IND submissions for Rett syndrome and
genetic ALS in late 2018/early 2019 –
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 fourth quarter and full year ended
December 31, 2017, recent corporate highlights and upcoming
milestones.
“2017 was marked by significant progress for AveXis across
multiple fronts, including alignment with the U.S. Food and Drug
Administration (FDA) on our commercial manufacturing process for
AVXS-101, publication of Phase 1 results for spinal muscular
atrophy (SMA) Type 1 in the New England Journal of Medicine, and
the initiation of pivotal SMA Type 1 and Phase 1 SMA Type 2 trials
in the United States,” said Sean Nolan, President and Chief
Executive Officer of AveXis. “As we continue this momentum into
2018, we look forward to the pre-Biologics License Application
(BLA) meeting with the FDA for AVXS-101, as well as to completing
enrollment in our ongoing SMA Types 1 and 2 studies, and to
expanding our clinical development program into other SMA
sub-populations, and programs beyond SMA.”
Recent Corporate Highlights
- Expanded Product Development Pipeline: On June
7, 2017, AveXis obtained exclusive worldwide rights to AAV9 from
REGENXBIO for Rett syndrome (MECP2 gene) and a genetic form of
amyotrophic lateral sclerosis (ALS) caused by mutations in the
superoxide dismutase 1 (SOD1) gene. AveXis had previously licensed
the preclinical data developed by Dr. Brian Kaspar from Nationwide
Children’s Hospital. These are rare, life-threatening, neurological
monogenic diseases that have significant unmet need with either
minimally effective or no available treatment options. AveXis
expects to leverage its Good Manufacturing Practice manufacturing
platform for these programs, which AveXis believes will reduce
overall development timelines.
- Announced Alignment with the FDA on Next Steps in
Regulatory Pathway for AVXS-101 in SMA Type 1: On January
4, 2018, following the receipt of minutes from the end-of-Phase 1
meeting with the FDA, AveXis announced plans to address detailed
information requests from the agency by submitting the requested
information to the investigational new drug (IND) application on an
ongoing basis, and plans to submit the request for a pre-BLA
meeting in the second quarter of 2018.
- Acquired Exclusive Worldwide Rights from REGENXBIO to
Entire NAV Technology Platform to Develop Treatments for
SMA: On January 8, 2018, AveXis announced an amended
licensing agreement, which expands the exclusive, worldwide license
agreement to REGENXBIO’s entire NAV platform for SMA, and permits
license assignment by AveXis upon a change of control without
consent from REGENXBIO.
- Initiated Phase 1 Trial of AVXS-101 in SMA Type 2
(STRONG) Using Intrathecal Delivery and Product from the Company’s
Good Manufacturing Practice Commercial Process: On January
16, 2018, AveXis announced the first patient was dosed in STRONG.
Two patients have been dosed to date, with a four-week interval
between the dosing of the first three patients for each dose cohort
per the trial protocol.
- Completed Public Offering with Net Proceeds of $431.9
Million: On January 22, 2018, AveXis closed an
underwritten public offering with net proceeds of $431.9 million.
AveXis intends to use the net proceeds to fund its research,
manufacturing and clinical activities to support its programs in
SMA, Rett syndrome and genetic ALS; to fund pre-commercial
activities, including medical affairs, development of commercial
initiatives for the potential launch of AVXS-101; licensing
activities; and for general corporate purposes and working
capital.
- Expanded SMA Clinical Development Program: On
January 26, 2018, AveXis announced plans to initiate three studies
to further evaluate AVXS-101, including in new SMA patient
populations.
- Initiated Screening for Remaining Patients in U.S.
Pivotal Trial of AVXS-101 for SMA Type 1 (STR1VE): On
January 30, 2018, following review of safety data and early signals
of efficacy from the first three patients dosed, and with agreement
from the FDA, AveXis initiated screening for the remaining patients
in the trial. To date, five patients have been dosed.
Upcoming Milestones
- Pre-BLA Meeting Request: AveXis expects to
submit the request for a pre-BLA meeting related to AVXS-101 in SMA
Type 1 in the second quarter of 2018.
- Pivotal Trial of AVXS-101 in SMA Type 1 in Europe
(STR1VE EU): The single-arm trial is expected to initiate
in the first half of 2018 and enroll approximately 30 patients who
are less than six months of age and naïve to SMA treatment. STR1VE
EU is designed to evaluate a one-time intravenous (IV) infusion of
AVXS-101, including safety, event-free survival and achievement of
the developmental milestone of sitting without support for 10
seconds, and will study children up to 18 months of age.
- Pre-Symptomatic SMA Types 1, 2, 3 (SPRINT):
The multi-cohort, multi-national trial is expected to initiate in
the first half of 2018 and enroll approximately 44 patients with
two, three and four copies of SMN2 who are less than six weeks of
age and pre-symptomatic. SPRINT is designed to evaluate appropriate
clinical endpoints, including developmental milestones, survival,
bulbar function, and safety of a one-time IV infusion of AVXS-101.
The primary efficacy endpoint by cohort is as follows:• Cohort
with two copies of SMN2: proportion of patients who achieve
functional independent sitting for at least 30 seconds, up to 18
months of age;• Cohort with three copies of SMN2: proportion
of patients who achieve the ability to stand without support for at
least three seconds, up to 24 months of age;• Cohort with four
copies of SMN2: proportion of patients who do not manifest symptoms
consistent with SMA Type 3 based on a scaled score on Bayley V.3
Gross and Fine Motor Subtests within 1.5 standard deviations of
chronological development reference standard, as assessed at 36
months of age.
- Pediatric SMA Types 1, 2, 3 (REACH): Expected
to initiate late in the fourth quarter of 2018 or early 2019.
- Rett Syndrome and Genetic ALS: Expected to
submit IND applications for AVXS-201 for Rett syndrome (MECP2) and
AVXS-301 for genetic ALS (SOD1) in late 2018/early 2019.
Fourth Quarter and Full Year 2017 Financial
Results
- Cash Position: As of December 31, 2017, AveXis
had $324.1 million in cash and cash equivalents. This
amount does not include the net proceeds from the recently
completed public offering described above.
- R&D Expenses: Research and development
expenses were $51.4 million for the fourth quarter of 2017 (which
included $4.9 million of non-cash stock-based compensation
expense), compared to $18.3 million for the same period in 2016
(which included $1.6 million of non-cash stock-based compensation
expense), an increase of $33.1 million. The increase in research
and development expenses was primarily attributable to increases in
third‑party clinical and manufacturing research and development
spending related to AveXis’ clinical trials and clinical trial
product manufacturing, salaries and personnel‑related expenses
driven by increased headcount across all research, development and
manufacturing functions, license fees and other research and
development expenses.
- G&A Expenses: General and administrative
expenses were $31.1 million for the fourth quarter of 2017 (which
included $3.0 million of non-cash stock-based compensation
expense), compared to $7.2 million for the same period in 2016
(which included $2.4 million of stock-based compensation expense),
an increase of $23.9 million. The increase in general and
administrative expenses was primarily attributable to a loss
incurred on a non-cash common stock settlement and increases in
pre-commercial marketing expenses, salaries and personnel‑related
costs driven by increased headcount across all general and
administrative functions to support our overall growth, and legal,
professional and consulting fees and other administrative
costs.
- Net Loss: Net loss was $81.7 million, or $2.55
per share, for the fourth quarter of 2017, compared to a net loss
of $25.4 million, or $0.92 per share, for the fourth quarter of
2016.
Selected Financial Information
Operating
Results (In thousands, except per share data): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
Operating
Expenses: |
|
|
|
|
|
|
|
General
and administrative |
|
31,078 |
|
|
|
7,198 |
|
|
|
69,976 |
|
|
|
24,523 |
|
Research
and development |
|
51,445 |
|
|
|
18,350 |
|
|
|
150,391 |
|
|
|
58,892 |
|
Total
Operating Expenses |
|
82,523 |
|
|
|
25,548 |
|
|
|
220,367 |
|
|
|
83,415 |
|
Loss from
operations |
|
(82,523 |
) |
|
|
(25,548 |
) |
|
|
(220,367 |
) |
|
|
(83,415 |
) |
|
|
|
|
|
|
|
|
Interest
income |
|
858 |
|
|
|
173 |
|
|
|
2,316 |
|
|
|
403 |
|
|
|
|
|
|
|
|
|
Net Loss |
$ |
(81,665 |
) |
|
$ |
(25,375 |
) |
|
$ |
(218,051 |
) |
|
$ |
(83,012 |
) |
|
|
|
|
|
|
|
|
Weighted-average basic
and diluted common shares outstanding |
|
32,030 |
|
|
|
27,678 |
|
|
|
29,935 |
|
|
|
22,648 |
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per common share |
$ |
(2.55 |
) |
|
$ |
(0.92 |
) |
|
$ |
(7.28 |
) |
|
$ |
(3.67 |
) |
|
|
|
|
|
|
|
|
Balance Sheet
Information (In thousands): |
|
|
|
|
|
|
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
Cash and cash
equivalents |
|
|
|
|
$ |
324,117 |
|
|
$ |
240,430 |
|
Total assets |
|
|
|
|
|
391,578 |
|
|
|
270,575 |
|
Total liabilities |
|
|
|
|
|
58,838 |
|
|
|
24,443 |
|
Accumulated
deficit |
|
|
|
|
$ |
(359,613 |
) |
|
$ |
(141,562 |
) |
|
|
|
|
|
|
|
|
Conference Call InformationAveXis will host a
conference call and webcast at 4:30 p.m. EST today, February 27, to
discuss the fourth quarter and full year 2017 financial and
operating results, recent business highlights and upcoming
development milestones.
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 1599399. 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 1599399.
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 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 those affected will never walk without
support and most will never stand without support. SMA Type 2
results in mortality in more than 30 percent of patients by the age
of 25.About Rett Syndrome Rett syndrome is a
devastating, rare neurological disorder characterized by slowed
growth, loss of normal movement and coordination and loss of
communication skills. Rett syndrome is caused by an X-linked
dominant mutation in the methyl CpG binding protein 2 (MECP2) gene,
which results in problems with MECP2 protein production critical
for brain development. Rett syndrome occurs in approximately one of
every 10,000 female births in the U.S., and affected infants
usually begin to show signs and symptoms 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 Sclerosis
Amyotrophic lateral sclerosis (ALS) is a progressive
neurodegenerative disease that affects motor neurons in the brain
and the spinal cord. Inherited forms of ALS comprise five to 10
percent of ALS cases, or approximately 1,000 to 2,000 people in the
U.S., and can be caused by mutations in several genes known to be
associated with ALS. Approximately 15 to 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 AVXS-101AveXis’ initial product
candidate, AVXS-101, is its proprietary gene therapy currently in
development for the one-time treatment of 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, Inc. is a
clinical-stage gene therapy company, dedicated to developing and
commercializing novel treatments for patients suffering from rare
and life-threatening neurological genetic diseases. Our initial
product candidate, AVXS-101, is our proprietary gene therapy
currently in development for the treatment of spinal muscular
atrophy, or SMA, Type 1, the leading genetic cause of infant
mortality, and SMA Type 2. The U.S. Food and Drug Administration,
or FDA, has granted AVXS-101 Orphan Drug Designation for the
treatment of all types of SMA and Breakthrough Therapy Designation,
as well as Fast Track Designation for the treatment of SMA Type 1.
In addition to developing AVXS-101 to treat SMA Type 1 and Type 2,
we also plan to develop other novel treatments for rare
neurological diseases, including Rett syndrome and a genetic form
of amyotrophic lateral sclerosis 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’ clinical 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 and Type 2, the expected timing of future meetings with
the FDA, the continued enrollment of patients in the STR1VE and
STRONG clinical trials, the initiation of AveXis’ planned future
clinical trials of AVXS-101, AveXis’ clinical development and
regulatory plans for AVXS-201 and AVXS-301, and the planned
expansion of AveXis’ development of gene therapy into additional
disorders. 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 September 30, 2017, filed
with the SEC on November 9, 2017 and the information included in
AveXis’ Current Report on Form 8-K filed with the SEC on January
16, 2018. 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 BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
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