AveXis Reports Third Quarter 2016 Financial and Operating Results
November 10 2016 - 4:01PM
– AVXS-101 demonstrated continued motor function
improvement in ongoing study; majority of patients on proposed
therapeutic dose achieved key developmental milestones – –
Confirmed single-arm design for pivotal study of SMA Type 1 in U.S.
–
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, 2016,
recent corporate highlights and upcoming milestones.
“This has been another eventful quarter for AveXis, with
continued positive data from the ongoing Phase 1 study of AVXS-101
in SMA Type 1, including, for the first time, reporting of motor
milestone achievements,” said Sean Nolan, President and Chief
Executive Officer of AveXis. “We look forward to progressing
AVXS-101 into pivotal studies in the U.S. and EU in the first half
of next year, and are eager to initiate a Type 2 study as we
continue to explore gene therapy for all patients suffering from
SMA.”
Recent Highlights
- Interim Phase 1 Data: On October 10, 2016,
AveXis reported interim data as of September 15, 2016 from the
ongoing Phase 1 trial of AVXS-101 in SMA Type 1, as 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.
- AVXS-101 appears to have a favorable safety profile and to be
generally well tolerated in patients studied as of September 15,
2016. There had been a cumulative total of 118 adverse events (AEs)
reported, 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 2 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) were
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 was 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) had a pulmonary event in
the third quarter. 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.
- 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 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 were 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.
Pivotal Trial Design
- On November 1, 2016, AveXis announced the pivotal study of
AVXS-101 in SMA Type 1 will reflect a single-arm design, using
natural history of the disease as a comparator, and will enroll
approximately 20 patients. This update was based on the receipt of
the minutes following a Type B meeting with the U.S. Food and Drug
Administration (FDA).
- In addition to evaluating safety, the planned program is
expected to evaluate achievement of motor milestones, specifically
patients’ ability to sit unassisted, as well as an efficacy measure
defined by the time from birth to an “event,” 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 FDA also indicated its preference for a design with
co-primary endpoints consisting of a measure of developmental
milestone achievement (such as sitting unassisted) along with a
clinically meaningful measure of survival (such as time to an
“event” as described above). Based on FDA’s suggestions as well as
other expert input, AveXis continues to evaluate a number of the
details of the trial design. More specific information will be made
available at the time the study is initiated, which is expected in
the first half of 2017.
- The Company’s strategy with the SMA Type 1 program is to
complete the ongoing Phase 1 trial and, in parallel, execute on the
single-arm pivotal trial, while continuing collaborative
discussions with the FDA regarding the most expeditious pathways
for FDA approval of AVXS-101.
Common Stock Offering
- On September 13, 2016, the Company completed an underwritten
public offering of 4,887,500 shares of its common stock, 4,597,645
shares of which were issued and sold by the Company, including the
exercise in full by the underwriters of their option to purchase
637,500 shares from the Company, and 289,855 shares of which were
sold by PBM Capital Investments, LLC (PBM), an existing stockholder
of the Company, each at a public offering price of $34.50 per
share. After deducting the underwriting discounts and
commissions and other offering expenses payable by the Company, the
net proceeds to the Company were approximately $149.1
million. The Company did not receive proceeds from the sale
of the common stock by PBM.
Clinical Development Milestones
- Provide quarterly updates on the ongoing Phase 1 trial of
AVXS-101 in SMA Type 1.
- Initiate a Phase 1 safety and dosing study of AVXS-101 via
intrathecal (IT) delivery in patients with SMA Type 2 in the second
half of 2016.
- Report 13.6 months of data for all patients in the ongoing SMA
Type 1 Phase 1 trial in the first quarter of 2017.
- Initiate pivotal trials of AVXS-101 in patients with SMA Type 1
in the United States and Europe in the first half of 2017.
Third Quarter 2016 Financial Results
- Cash Position: As of September 30, 2016,
AveXis had $263.6 million in cash and cash equivalents.
- R&D Expenses: Research and development
expenses were $14.1 million for the third quarter of 2016 (which
included $2.7 million of non-cash stock-based compensation
expense), compared to $6.3 million for the same period in 2015
(which included $5.3 million of non-cash stock-based compensation
expense), an increase of $7.8 million. The increase in research and
development expenses was primarily attributable to an increase in
expenses necessary to support the advancement of the Company’s
manufacturing product development efforts, clinical and
pre-clinical programs, primarily the ongoing trial of AVXS-101 in
SMA Type 1, and increases in employee compensation. Partially
offsetting the increase in R&D spending was lower stock-based
compensation expense of $2.6 million.
- G&A Expenses: General and administrative
expenses were $7.1 million for the third quarter of 2016 (which
included $2.7 million of non-cash stock-based compensation
expense), compared to $3.7 million for the same period in 2015
(which included $1.5 million of stock-based compensation expense),
an increase of $3.4 million. The increase in general and
administrative expenses was primarily attributable to an increase
in employee compensation, legal and professional fees and other
infrastructure costs to support the company’s overall growth, and
higher stock-based compensation expense.
- Net Loss: Net loss was $21.1 million, or $0.87
per share, for the third quarter of 2016, compared to a net loss of
$9.9 million, or $1.32 per share, for the third quarter of
2015.
Selected Financial Information
Operating Results:
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
|
|
|
|
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
|
|
|
|
|
Revenue |
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
Total revenue |
|
|
|
|
|
|
|
|
Operating
Expenses: |
|
|
|
|
|
|
|
|
General and administrative |
|
|
7,082,683 |
|
|
|
3,740,077 |
|
|
|
17,324,863 |
|
|
|
6,651,233 |
|
Research and development |
|
|
14,097,713 |
|
|
|
6,289,350 |
|
|
|
40,542,323 |
|
|
|
18,756,214 |
|
Total Operating Expenses |
|
|
21,180,396 |
|
|
|
10,029,427 |
|
|
|
57,867,186 |
|
|
|
25,407,447 |
|
Interest income |
|
|
98,588 |
|
|
|
88,769 |
|
|
|
230,313 |
|
|
|
94,410 |
|
Net loss and
comprehensive loss |
|
$ |
(21,081,808 |
) |
|
$ |
(9,940,658 |
) |
|
$ |
(57,636,873 |
) |
|
$ |
(25,313,037 |
) |
|
|
|
|
|
|
|
|
|
Weighted-average basic
and diluted common shares outstanding |
|
|
24,166,113 |
|
|
|
7,504,148 |
|
|
|
20,958,421 |
|
|
|
7,042,977 |
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per common share |
|
$ |
(0.87 |
) |
|
$ |
(1.32 |
) |
|
$ |
(2.75 |
) |
|
$ |
(3.59 |
) |
|
|
|
|
|
|
|
|
|
Balance Sheet Information:
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
|
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
Cash and
cash equivalents |
|
|
|
|
|
$ |
263,642,069 |
|
|
$ |
62,251,860 |
|
|
Total
assets |
|
|
|
|
|
|
|
284,803,706 |
|
|
|
65,084,291 |
|
|
Total
liabilities |
|
|
|
|
|
|
|
17,581,342 |
|
|
|
6,877,304 |
|
|
Accumulated
deficit |
|
|
|
|
|
$ |
(116,187,393 |
) |
|
$ |
(58,550,520 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
Conference Call InformationThe AveXis’
conference call and webcast of November 1, 2016, was conducted in
lieu of the previously announced conference call scheduled for
Thursday, November 10, 2016 at 4:30 p.m. EST. AveXis will not host
a conference call and webcast related to third quarter 2016
financial and operating results. The November 1 webcast is archived
through November 30, 2016, on the Events and Presentations page of
the company’s website, and is available for telephonic replay
through November 14, 2016, by dialing (855) 859-2056 (Domestic) or
(404) 537-3406 (International), conference ID
12184733.
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 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, expectations regarding design and timing of the
pivotal trial of AVXS-101 as well as the overall clinical
development 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 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.
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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