– First patient has been dosed in pivotal trial
of AVXS-101 for SMA Type 1 –
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 third quarter ended September 30, 2017,
recent corporate highlights and upcoming milestones.
“We had several significant accomplishments this quarter, and
the recent dosing of our first patient in the pivotal study for SMA
Type 1 using AVXS-101 product derived from our GMP process is a
very exciting milestone for the company and the patients we serve,”
said Sean Nolan, President and Chief Executive Officer of AveXis.
“The anticipated initiation of the Type 2 trial, as well as our
upcoming end-of-Phase 1 meeting later this quarter to further
inform the regulatory pathway for AVXS-101, will propel us into an
eventful 2018.”
Recent Highlights
Initiated Pivotal Trial of AVXS-101 in SMA Type 1 Using
Product from New Good Manufacturing Practice (GMP) Commercial
Process: On September 29, AveXis announced the U.S. Food
and Drug Administration (FDA) notified the company it may initiate
its planned pivotal trial of AVXS-101 for patients with spinal
muscular atrophy (SMA) Type 1 using the intravenous (IV)
formulation produced by the company’s GMP commercial manufacturing
process.
- The open-label, single-arm, single-dose, multi-center trial –
known as STR1VE – is designed to evaluate the efficacy and safety
of a one-time IV infusion of AVXS-101 of 1.1 x 1014 vg/kg, which is
equivalent to the proposed therapeutic dose (Cohort 2) in the Phase
1 trial, in patients with SMA Type 1.
- The trial will enroll a minimum of 15 patients with SMA Type 1
who are less than six months of age at the time of gene therapy,
and who have one or two copies of the SMN2 backup gene as
determined by genetic testing and bi-allelic SMN1 gene deletion or
point mutations. The intent to treat (ITT) population defined in
the protocol includes symptomatic infants less than six months of
age with the bi-allelic deletion of the SMN1 gene, only two copies
of the SMN2 backup gene and no exon 7 modifier. There will be at
least a four-week dosing interval between dosing of the first three
patients to allow review of the safety analysis as well as early
signals of efficacy, prior to dosing of the next patient.
First Patient Dosed in Pivotal Trial of AVXS-101 in SMA
Type 1: Today, AveXis announced the first patient has been
dosed in the pivotal trial. The patient met the ITT criteria of
being symptomatic, less than six months of age and was confirmed by
genetic testing to have a bi-allelic deletion of the SMN1 gene, two
copies of the SMN2 backup gene, and no exon 7 modifier.
Data from Phase 1 Trial of AVXS-101 in SMA Type 1
Presented at the International Annual Congress of the World Muscle
Society (WMS): On October 3, AveXis reported top-line data
as of August 7, 2017, from the Phase 1 trial of AVXS-101 in
patients with SMA Type 1 in a late-breaking poster authored by
Jerry Mendell, M.D., principal investigator in the trial 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, Columbus, Ohio, during the
22nd International Annual Congress of the World Muscle Society.
Analysis Presented at WMS of Seven Patients in Cohort 2
of the Phase 1 Trial of AVXS-101 in SMA Type 1 Demonstrated
Cognitive, Fine Motor and Language Abilities Within the Normal
Range: Linda Lowes, PT, PhD, Director of Clinical
Therapies Research and a member of the Center for Gene Therapy at
the Research Institute of Nationwide Children’s Hospital, analyzed
seven of the 12 patients in Cohort 2 of the Phase 1 Trial of
AVXS-101 in SMA Type 1 using subsets of the Bayley Scales of Infant
and Toddler Development® Third Edition, a norm-referenced,
well-validated tool for infant and toddler development. Selection
of patients for this evaluation was based on availability and
ability to complete the testing.
- All patients evaluated demonstrated fine motor, cognition and
language ability within the normal range. Natural history indicates
patients will rarely achieve the ability to speak or retain any
significant motor ability.
- In normal children, average composite scores for language and
cognition abilities are 90-109, and fine motor skills are reported
as a scaled score in which 10 is the mean.• Seven of 7 (100%)
patients demonstrated age-appropriate language (94-106), cognition
(90-105) and fine motor skills (9-12) when compared to healthy
age-matched peers.
Data from Phase 1 Trial of AVXS-101 in SMA Type 1
Published in The New England Journal of Medicine
(NEJM): On November 1, AveXis reported the full data
from the Phase 1 study was published in NEJM. Data as of August 7,
2017, showed:
- All 15 patients (100%) were alive and event-free at 20 months
of age, in contrast to the eight percent event-free rate
demonstrated in an independent, peer-reviewed natural history study
for patients with SMA Type 1. The median age at last follow-up was
25.7 months and 30.8 months for 12 patients in Cohort 2 and the
three patients in the low-dose cohort (Cohort 1), respectively.
Patients in Cohort 2 continued to demonstrate improvements in motor
milestones: the majority of patients (92%) achieved head control,
rolling over (75%), sitting with assistance (92%) and sitting
without assistance for at least 5 seconds (92%). Two patients in
the cohort could crawl, pull to a stand, as well as stand and walk
independently.
- All patients who were free of respiratory or feeding support on
January 20, 2017, continued without the need for these elements of
supportive care.
- 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 Initiated Additional Formal Clinical and Quality
Scientific Advice Proceedings with the European Medicines Agency
(EMA) and Initiated the Process to Discuss the Potential for a
Conditional Registration Pathway for the European Union
(EU): AveXis also held detailed Clinical and Quality
Scientific Advice with a number of European National Authorities
focused on the Clinical Development Plan, as well as the
Manufacturing/Quality process, which will serve to facilitate
review and approval of upcoming Clinical Trial Applications.
AveXis Opened Its Research and Development Facility in
San Diego, California: This will be the location for
analytical development, discovery research and translational
development work.
Phillip B. Donenberg Appointed as Chief Financial
Officer: On October 19, the AveXis Board of Directors
appointed Phillip B. Donenberg as Senior Vice President and Chief
Financial Officer of the company. Formerly the Vice President,
Controller at AveXis, Mr. Donenberg brings more than 25 years of
corporate finance, accounting and business development experience
at publicly traded biotech and specialty pharmaceutical companies
to the position.
Upcoming Milestones
SMA Type 2 Trial: AveXis is in ongoing
discussions with the FDA on the SMA Type 2 Trial and remains
optimistic that the trial will initiate before the end of the
year.
SMA Type 1 End-of-Phase 1 Meeting with FDA: The
end-of-Phase 1 meeting, scheduled for late in the fourth quarter,
will further inform next steps in the regulatory process for
AVXS-101. AveXis expects to provide an update in the first quarter
of 2018 upon receipt of the meeting minutes from the FDA.
SMA Type 1 EU Trial: AveXis incorporated EU
specific Scientific Advice from the EMA into the protocol design
and expects to initiate a pivotal trial of AVXS-101 in SMA Type 1
in the EU in the first half of 2018.
Third Quarter 2017 Financial Results
- Cash Position: As of September 30, 2017,
AveXis had $374.2 million in cash and cash equivalents.
- R&D Expenses: Research and development
expenses were $33.4 million for the third quarter of 2017 (which
included $5.0 million of non-cash stock-based compensation
expense), compared to $14.1 million for the same period in 2016
(which included $2.7 million of non-cash stock-based compensation
expense), resulting in an increase of $19.3 million. The increase
was primarily attributable to product manufacturing expenses and
associated accelerated spending, including increased headcount in
AveXis’ product manufacturing facility, as well as expenses related
to the conclusion of the Phase 1 clinical trial of AVXS-101 in SMA
Type 1, commencement of AveXis’ pivotal trial of AVXS-101 and an
increase in non-cash stock-based compensation expense.
- G&A Expenses: General and administrative
expenses were $16.1 million for the third quarter of 2017 (which
included $5.3 million of non-cash stock-based compensation
expense), compared to $7.1 million for the same period in 2016
(which included $2.7 million of stock-based compensation expense),
resulting in an increase of $9.0 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 $48.6 million, or $1.52
per share, for the third quarter of 2017, compared to a net loss of
$21.1 million, or $0.87 per share, for the third quarter of
2016.
Selected Financial Information (unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating Results: |
|
|
|
|
|
|
|
|
|
|
|
(In
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
Revenue |
|
|
|
|
|
|
|
|
|
|
|
|
Total revenue |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
Expenses: |
|
|
|
|
|
|
|
|
|
|
General and administrative |
|
|
16,095 |
|
|
|
7,083 |
|
|
|
38,898 |
|
|
|
17,325 |
|
|
Research and development |
|
|
33,425 |
|
|
|
14,098 |
|
|
|
98,946 |
|
|
|
40,542 |
|
|
Total Operating Expenses |
|
|
49,520 |
|
|
|
21,181 |
|
|
|
137,844 |
|
|
|
57,867 |
|
|
Loss from
operations |
|
|
|
(49,520 |
) |
|
|
(21,181 |
) |
|
|
(137,844 |
) |
|
|
(57,867 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
|
|
|
881 |
|
|
|
99 |
|
|
|
1,458 |
|
|
|
230 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
|
|
|
$ |
(48,639 |
) |
|
$ |
(21,082 |
) |
|
$ |
(136,386 |
) |
|
$ |
(57,637 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average basic and diluted common shares outstanding |
|
31,941,185 |
|
|
|
24,166,113 |
|
|
|
29,228,847 |
|
|
|
20,958,421 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted net loss per common share |
|
$ |
(1.52 |
) |
|
$ |
(0.87 |
) |
|
$ |
(4.67 |
) |
|
$ |
(2.75 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheet Information: |
|
|
|
|
|
|
|
|
|
|
(In
thousands) |
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
|
|
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
Cash and cash equivalents |
|
|
|
|
|
$ |
374,211 |
|
|
$ |
240,430 |
|
|
Total assets |
|
|
|
|
|
|
|
432,057 |
|
|
|
270,575 |
|
|
Total liabilities |
|
|
|
|
|
|
|
28,007 |
|
|
|
24,443 |
|
|
Accumulated deficit |
|
|
|
|
|
$ |
(277,948 |
) |
|
$ |
(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 3899689. 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
3899689.
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. SMA 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.
About AVXS-101AVXS-101 is a proprietary gene
therapy candidate of a one-time treatment for SMA Type 1, 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. The
company also intends to expand the study of gene therapy into SMA
Type 2 and 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 positively
impact quality of life and alter the course of disease in children
with SMA Type 1 and Type 2, the expected timing of dosing patients
in the pivotal trial for AVXS-101, the initiation of AveXis’
planned future clinical trials in SMA Type 1 and Type 2, the
expected timing of future meetings with the FDA, the overall
clinical development of AVXS-101 and expectations regarding AveXis’
research, development and regulatory plans for its programs for
treatment 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" 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 BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jun 2024 to Jul 2024
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jul 2023 to Jul 2024