-- Interim data through July 1 showed no “events”
--
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, 2016,
recent corporate highlights and upcoming milestones, and provided
an update on interim data from the ongoing Phase 1 trial of
AVXS-101 in spinal muscular atrophy (SMA) Type 1.
Data as of July 1, 2016 showed AVXS-101 continued to demonstrate
a favorable safety profile, with no new treatment-related safety or
tolerability concerns identified. All patients in both Cohort 1
(low dose) and Cohort 2 (proposed therapeutic dose) remained
without an “event,” defined as death or until a patient requires 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. Since July 1, one patient in the
low-dose cohort has reached a ventilation endpoint, described in
further detail below. As of July 1, the mean motor function scores
in both dosing cohorts continued to increase, with 25% of patients
in Cohort 2 having achieved motor function in a range considered to
be normal.
“We continue to be encouraged with this interim data for
AVXS-101 and are eager to collaborate with the FDA via the
breakthrough designation process to determine next steps in the
development pathway for SMA Type 1, and we have submitted a Type B
meeting request with the agency to facilitate this discussion,”
said Sean Nolan, President and Chief Executive Officer of AveXis.
“We expect several key developments in the second half of the year
including the presentation of developmental milestone data from our
ongoing SMA Type 1 study, providing additional clarity on the
development path for the Type 1 program and we expect to initiate
our initial safety and dosing study in SMA Type 2 patients.”
Interim Phase 1 Data as of July 1, 2016
- AVXS-101 appeared to have a favorable safety profile and to be
generally well tolerated in patients studied, with no new
treatment-related adverse events reported.
- There have been a cumulative total of 107 adverse events (AEs)
reported as of July 1, 2016, 31 of which were determined to be
serious adverse events (SAEs). As previously reported, two of the
31 were deemed treatment-related and consisted of clinically
asymptomatic liver enzyme elevations.
- Of the cumulative total of 76 non-serious AEs reported as of
July 1, 2016, three were the previously reported treatment-related
elevations in liver enzymes experienced by two patients.
- All of the elevated liver enzyme AEs and SAEs were clinically
asymptomatic and resolved with prednisolone treatment. There were
no elevations of GGT, alkaline phosphatase or bilirubin, and Hy’s
Law was not met.
- Other non-treatment-related AEs were expected and were
associated with SMA.
- Mean increases in motor function compared to baseline of 9.0
points in Cohort 1 and 23.3 points in Cohort 2 in CHOP-INTEND
scores were observed. 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.
- Eleven out of 12 patients (92 percent) and 8 out of 12 patients
(67 percent) in Cohort 2 had achieved CHOP-INTEND scores of at
least 40 or 50 points, respectively.
- Three out of 12 patients (25 percent) in Cohort 2 achieved a
score of 60, which is in a range considered to be normal.
“These data demonstrate rapid, sustained and marked improvements
in motor function as measured by CHOP-INTEND,” said Suku Nagendran,
MD, Senior Vice President and Chief Medical Officer, AveXis.
“Compared with the data presented as of April 1, 2016, the positive
impact on average motor function scores increased from 8.7 to 9.0
points and from 19.2 to 23.3 points in Cohorts 1 and 2,
respectively. It appears that both dose and baseline motor function
are key factors in determining the degree of treatment response.
Based on our data to date, early diagnosis and treatment appear to
be critical for optimal outcomes in this population.”
Protocol-Specified Ventilation Event Occurring After
July 1
- The protocol defines the “permanent ventilation endpoint” as
the use of at least 16 hours per day of respiratory assistance
(includes non-invasive ventilatory support) continuously for
greater than 2 weeks in the absence of an acute reversible illness,
excluding perioperative ventilation.
- In the course of reviewing the data as of July 1, 2016, AveXis
learned that one patient in Cohort 1, the low-dose group, had begun
to increase use of bi-level positive airway pressure (BiPAP) in
advance of surgery related to hypersalivation, a condition
experienced by some SMA patients. This patient did not meet the
definition of a permanent ventilation endpoint until after July 1,
2016. As a result, the independent Data Safety Monitoring Board
(DSMB) determined that this development was not a “permanent
ventilation endpoint” for the data as of July 1, 2016, but would be
classified as an “event” for the next data period ending September
30, 2016. This event was determined by the DSMB to represent
progression of disease and not an adverse event related to the use
of AVXS-101.
Recent Highlights
- Breakthrough Therapy Designation: On July 20,
2016, AveXis reported that the U.S. Food and Drug Administration
(FDA) had granted Breakthrough Therapy Designation for AVXS-101 for
the treatment of SMA Type 1 in pediatric patients based on
preliminary clinical results from the ongoing trial of AVXS-101. In
granting the designation, the FDA indicated that as a next step
that AveXis should submit a Type B meeting request to
facilitate a comprehensive discussion of the development pathway.
AveXis has subsequently submitted the meeting request.
- Pulmonary Support Data: On May 15, 2016,
Richard Shell, MD, member of the Section of Pulmonary Medicine at
Nationwide Children’s Hospital and an Associate Professor of
Clinical Pediatrics at The Ohio State University College of
Medicine, presented additional pulmonary support data at the
International Conference of the American Thoracic Society in San
Francisco.
- The interim analysis presented at the meeting demonstrated gene
therapy appeared to reduce the need for ventilation support and
allowed patients to successfully recover from respiratory illnesses
that are often lethal to SMA Type 1 patients. None of the patients
in either dosing cohort required permanent ventilation as of April
1, 2016.
- In addition, 8 of 10 (80 percent) patients on the proposed
therapeutic dose of 2.0x1014 vg/kg who did not need
biphasic/bi-level ventilation (BiPAP) support prior to gene therapy
continued with no ventilation support. The two exceptions were
children hospitalized in the first quarter of 2016 for severe
respiratory illness and placed on BiPAP to aid recovery.
- Presented Interim Data at ASGCT: On May 6,
2016, Jerry Mendell, MD, director of the Center for Gene Therapy at
The Research Institute at Nationwide Children’s Hospital, presented
interim data as of April 1, 2016 from the ongoing Phase 1 trial of
AVXS-101 for the treatment of SMA Type 1 at the 19th Annual Meeting
of the American Society of Gene & Cell Therapy in Washington,
D.C.
- Appointed Michael B. Johannesen to Executive Management
Team: On July 5, 2016, Michael B. Johannesen was appointed
Senior Vice President, General Counsel and Chief Compliance
Officer. Mr. Johannesen brings to AveXis nearly 20 years of legal
experience within the global pharmaceutical, medical device and
healthcare products industries, and is responsible for all aspects
of the company’s legal and compliance functions, as well as human
resources.
Targeted Clinical Development Milestones and
Presentations
- 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.
- Disclose motor development milestones at a medical meeting in
the second half of 2016.
- Provide an update on the SMA Type 1 development path in the
second half of 2016 after meeting with FDA.
- 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.
Second Quarter 2016 Financial Results
- Cash Position: As of June 30, 2016, AveXis had
$131.4 million in cash and cash equivalents.
- R&D Expenses: Research and development
expenses were $10.4 million for the second quarter of 2016 (which
included $1.3 million of stock-based compensation), compared to
$7.7 million for the same period in 2015 (which included $5.8
million of stock-based compensation), an increase of $2.7 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 $4.5 million.
- G&A Expenses: General and administrative
expenses were $5.4 million for the second quarter of 2016 (which
included $2.6 million of stock-based compensation), compared to
$2.3 million for the same period in 2015 (which included $0.8
million of stock-based compensation), an increase of $3.1 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 $15.7 million, or $0.68
per share, for the second quarter of 2016, compared to a net loss
of $9.9 million, or $1.41 per share, for the second quarter of
2015.
Conference Call InformationAveXis will host a
conference call and webcast at 4:30 p.m. EDT today, August 11,
2016, to discuss second quarter 2016 financial results and business
updates.
Analysts and investors can participate in the conference call by
dialing (877) 508-0547 for domestic callers and (281) 973-6085 for
international callers, using the conference ID 57726673. 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 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 57726673.
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 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 expected timing for initiating and
reporting results from ongoing and planned clinical trials, the
expected timing for reporting motor development milestones and the
expected timing for the company’s planned Type B meeting with the
FDA. 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 ability to obtain and maintain regulatory approval
of AveXis’ product candidates; the scope, progress, expansion, and
costs of developing and commercializing AveXis’ product candidates;
AveXis’ ability to obtain and maintain intellectual property
protection for our product candidates; AveXis’ ability to establish
and maintain development partnerships; AveXis’ expectations
regarding federal, state and foreign regulatory requirements;
regulatory developments in the United States and foreign countries,
as well as other factors discussed in the "Risk Factors" and
"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, 2015, filed with the SEC on
March 18, 2016, the company’s Quarterly Report on Form 10-Q for the
quarter ended June 30, 2016, and other reports AveXis files with
the SEC. 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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Selected Financial
Information |
|
Operating Results: |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30 |
|
Six Months Ended June 30 |
|
|
|
|
|
|
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
|
|
|
|
|
Revenue |
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
Total
revenue |
|
|
|
|
|
|
|
|
Operating
Expenses: |
|
|
|
|
|
|
|
|
General
and administrative |
|
|
5,418,266 |
|
|
|
2,268,521 |
|
|
|
10,242,180 |
|
|
|
2,911,156 |
|
Research
and development |
|
|
10,379,521 |
|
|
|
7,654,839 |
|
|
|
26,444,610 |
|
|
|
12,466,864 |
|
Total
Operating Expenses |
|
|
15,797,787 |
|
|
|
9,923,360 |
|
|
|
36,686,790 |
|
|
|
15,378,020 |
|
Interest
income |
|
|
78,829 |
|
|
|
4,185 |
|
|
|
131,725 |
|
|
|
5,641 |
|
Net loss and
comprehensive loss |
|
$ |
(15,718,958 |
) |
|
$ |
(9,919,175 |
) |
|
$ |
(36,555,065 |
) |
|
$ |
(15,372,379 |
) |
|
|
|
|
|
|
|
|
|
Weighted-average basic
and diluted common shares outstanding |
|
|
23,013,838 |
|
|
|
7,030,976 |
|
|
|
19,876,850 |
|
|
|
5,045,211 |
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per common share |
|
$ |
(0.68 |
) |
|
$ |
(1.41 |
) |
|
$ |
(1.84 |
) |
|
$ |
(3.05 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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Balance Sheet Information: |
|
|
|
|
|
|
June 30 |
|
December 31 |
|
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
Cash and
cash equivalents |
|
|
|
|
|
$ |
131,432,653 |
|
|
$ |
62,251,860 |
|
Total
assets |
|
|
|
|
|
|
143,112,011 |
|
|
|
65,084,291 |
|
Total
Liabilities |
|
|
|
|
|
|
8,683,173 |
|
|
|
6,877,304 |
|
Total
stockholders' deficit |
|
|
|
|
|
$ |
(95,105,586 |
) |
|
$ |
(58,550,520 |
) |
|
|
|
|
|
|
|
|
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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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