AveXis Reports First Quarter 2016 Financial and Operating Results
May 12 2016 - 4:01PM
Encouraging interim data for lead clinical
program, AVXS-101, for Spinal Muscular Atrophy 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 first quarter ended March 31, 2016, and
provided an update on recent corporate highlights and upcoming
milestones.
“The first quarter and recent weeks have been a
period of important progress for AveXis, as we reported encouraging
interim data for our lead program for AVXS-101 in spinal muscular
atrophy (SMA) Type 1,” said Sean Nolan, President and Chief
Executive Officer of AveXis. “Our team is focused on executing our
plan to bring AVXS-101 to patients suffering from SMA.”
Recent Highlights
- Interim Phase 1 Data: On May 6, 2016, AveXis
reported interim data through the April 1, 2016 time period from
the ongoing Phase 1 trial of AVXS-101 in SMA Type 1. The data show
that AVXS-101 continued to demonstrate a favorable safety profile
in patients studied as of April 1, 2016, 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 14 consecutive days in the absence of an
acute reversible illness, or perioperatively; and the mean motor
function score continued to increase, with two patients having
achieved motor function in a range considered to be normal.
- AVXS-101 appears to have a favorable safety profile and to be
generally well tolerated in patients studied as of April 1, 2016.
There had been a total of 74 adverse events (AEs) reported, 22 of
which were determined to be serious adverse events (SAEs). Two of
the 22 were deemed treatment-related and, as previously reported,
consisted of clinically asymptomatic liver enzyme elevations. Of
the 52 non-serious AEs, 3 were treatment-related elevations in
liver enzymes experienced by two patients. All elevated liver
enzyme AEs and SAEs were resolved with prednisolone treatment.
Other non-treatment-related AEs were expected and were associated
with SMA.
- Mean increases in motor function of 8.7 points and 19.2 points
in CHOP-INTEND scores were observed in Cohort 1 and Cohort 2,
respectively. Nine out of 12 patients (75 percent) and 7 out of 12
patients (58 percent) in Cohort 2 had achieved CHOP-INTEND scores
of at least 40 points or 50 points, respectively. Two patients
achieved the maximum score of 64. A score of 60 is considered
normal. 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.
- New data appear to indicate that AVXS-101 may have a positive
impact on both the nutritional and pulmonary support of patients in
the trial.
- The natural history of SMA Type 1 indicates that bulbar
weakness leads to impaired swallowing, malnutrition and growth
failure. The median age to nutritional support is 8 months of age
(IQR 6-13 months)1. AVXS reported that 7 of 7 (100%) of AVXS-101
patients that did not require feeding support before treatment
continued without feeding support as of April 1, 2016. Five
patients had gastric feeding tube placement prior to gene therapy.
One patient in Cohort 2 who had gastric feeding tube placement
prior to gene transfer was also feeding orally.
- The natural history of SMA Type 1 indicates that bulbar muscle
weakness, skeletal muscle weakness in the neck and intercostal
muscle weakness lead to respiratory impairment, poor clearance of
airway secretions, risk of aspiration and recurrent infections
leading to death or permanent ventilation. The median age to
permanent ventilation or death is 10.5 months (IQR 8.1-13.6
months), and by 13.6 months only 25 percent of SMA Type 1 patients
are alive and free of permanent ventilation1. In the ongoing trial,
8 of 10 (80%) of AVXS-101 patients that did not use
biphasic/bi-level ventilation (BiPAP) support before gene transfer
continued without any ventilation support as of April 1, 2016 (the
2 exceptions being after severe illness/hospitalizations to assist
recovery).
Upcoming Clinical Development Milestones
and Presentations
- Additional pulmonary support data presented by Dr. Richard
Shell, 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, at the
International Conference of the American Thoracic Society (ATS) in
San Francisco. The oral presentation entitled, “Gene Therapy
for Spinal Muscular Atrophy (SMA) Type 1 Improves Survival and
Pulmonary Outcomes in a Phase I Safety Study (Abstract #480)” is
scheduled for Sunday, May 15 at 10:45 a.m. in the session,
“Genetics and Genomics of Childhood Lung Disease,” Room: 3006/3008
(West Building, Level 3), Moscone Center.
- 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.
First Quarter 2016 Financial
Results
- Cash Position: In February 2016, AveXis
completed its initial public offering of 5,277,941 shares of common
stock at an initial public offering price of $20.00 per share,
including a partial exercise of the over-allotment option by the
underwriters of 527,941 shares, before underwriting discounts. The
total net proceeds from the initial public offering were $98.2
million, after deducting underwriting discounts and estimated
offering expenses. As of March 31, 2016, AveXis had $148.2 million
in cash, cash equivalents and marketable securities, which included
the net proceeds from its initial public offering of common
stock.
- R&D Expenses: Research and development
expenses were $16.1 million for the first quarter of 2016 (which
included $11.5 million of stock-based compensation), compared to
$4.8 million for the same period in 2015, an increase of $11.3
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 clinical and pre-clinical
programs, primarily the Phase 1 trial of AVXS-101 in SMA Type 1,
the initiation of construction of the company’s manufacturing
facility, and an increase in employee compensation and benefit
expense.
- G&A Expenses: General and administrative
expenses were $4.8 million for the first quarter of 2016 (including
$2.1 million of stock-based compensation), compared to $0.6 million
for the same period in 2015, an increase of $4.2 million. The
increase in general and administrative expenses was primarily
attributable to an increase in employee compensation and benefit
expense to support the company’s overall growth and expenses
related to preparing for the initial public offering.
- Net Loss: Net loss was $20.8 million for the
first quarter of 2016, compared to a net loss of $5.5 million for
the first quarter of 2015. Net loss was $1.24 and $0.79 per common
share for the three months ended March 31, 2016 and March 31, 2015,
respectively.
Conference Call at 4:30 p.m.
EDTAveXis will host a conference call and webcast at
4:30 p.m. EDT today, May 12, 2016, to discuss first 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 95583934. 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 95583934.
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. 1. Finkel, R. et al. Observational
study of spinal muscular atrophy type I and implications for
clinical trials. Neurology 83, 810–817
(2014). 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.
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,
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.
Selected Financial Information
AveXis, Inc. |
Condensed Consolidated Statements of Operations
and Comprehensive Loss |
(unaudited) |
|
|
|
|
|
|
Three Months Ended |
|
|
March 31, 2016 |
|
March 31, 2015 |
Revenue |
|
$ |
- |
|
|
$ |
- |
|
Operating expenses: |
|
|
|
|
Research and development |
|
|
16,065,089 |
|
|
|
4,812,025 |
|
General and administrative |
|
|
4,823,913 |
|
|
|
642,634 |
|
Total
operating expenses |
|
|
20,889,002 |
|
|
|
5,454,659 |
|
Loss from operations |
|
|
(20,889,002 |
) |
|
|
(5,454,659 |
) |
Interest expense
(income) |
|
|
(52,895 |
) |
|
|
(1,455 |
) |
|
|
|
|
|
Loss from continuing operations |
|
|
(20,836,107 |
) |
|
|
(5,453,204 |
) |
Income
tax expense (benefit) |
|
|
- |
|
|
|
- |
|
Net loss
and comprehensive loss |
|
$ |
(20,836,107 |
) |
|
$ |
(5,453,204 |
) |
|
Basic
and diluted net loss per common share |
|
$ |
(1.24 |
) |
|
$ |
(0.79 |
) |
|
|
|
|
|
Weighted-average basic and diluted common shares outstanding |
|
|
16,774,718 |
|
|
|
6,895,185 |
|
|
|
|
|
|
Condensed Consolidated Balance Sheets:
|
|
|
|
|
Three Months Ended |
Balance Sheet
Data: |
|
|
|
|
March 31, 2016 |
|
March 31, 2015 |
Cash and cash
equivalents |
|
|
|
|
$ |
148,237,433 |
|
|
$ |
7,296,434 |
|
Total assets |
|
|
|
|
|
156,043,510 |
|
|
|
7,394,762 |
|
Total Liabilities |
|
|
|
|
|
9,739,643 |
|
|
|
5,145,541 |
|
Total stockholders'
deficit |
|
|
|
|
$ |
(79,386,627 |
) |
|
$ |
(25,529,823 |
) |
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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