AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today announced
that the European Medicines Agency (EMA) has granted access into
its PRIority MEdicines (PRIME) program for the company’s
proprietary gene therapy, AVXS-101, for the treatment of spinal
muscular atrophy (SMA) Type 1. The PRIME application was based on
data from both preclinical evaluations and the ongoing Phase 1
clinical trial of AVXS-101 as of September 15, 2016.
PRIME is intended to enhance support for the development of
medicines – specifically those that may offer a major therapeutic
advantage over existing treatments or benefit patients without
treatment options – through early and proactive support by EMA to
optimize the generation of robust data and development plans, and
potentially expedite the assessment of the Marketing Authorization
Application (MAA) so these medicines may reach patients sooner.
“The acceptance of AVXS-101 into the PRIME program reflects the
urgent need for innovative treatment options for the patients
diagnosed with SMA in the European Union,” said James L’Italien,
Ph.D., Senior Vice President, Chief Regulatory and Quality Officer
of AveXis. “We are eager for this enhanced collaboration with the
EMA to ensure we are taking the most appropriate and expeditious
path toward the development of a robust Marketing Authorization
Application submission, and to potentially streamlining the time
needed to bring AVXS-101 to patients in the EU suffering from this
devastating disease.”
In notifying the company of the acceptance, EMA noted: “The
preliminary clinical observations following AVXS-101 administration
include positive impact on survival, pulmonary function,
nutritional support, preservation of motor function and the
attainment of development milestones, all of which are unexpected
within the framework of the natural history and disease progression
for SMA Type 1. These clinically meaningful responses in the
patients treated with AVXS-101 are sufficient preliminary clinical
evidence of treatment effect that has the potential to address an
unmet need in this devastating pediatric disease.”
In July 2016, the U.S. Food and Drug Administration (FDA)
granted Breakthrough Therapy Designation, a comparable program to
PRIME used by the FDA, for AVXS-101 for the treatment of patients
with SMA Type 1.
AveXis intends to initiate a pivotal trial of AVXS-101 in
patients with SMA Type 1 in the European Union before the end of
2017.
About PRIMEThe EMA launched the PRIME
initiative in March 2016 to foster research and development of
medicines that may offer a major therapeutic advantage over
existing treatments, or benefit patients without treatment options.
PRIME aims to strengthen clinical trial designs to facilitate the
generation of high quality data for the evaluation of an
application for marketing authorization. To be accepted for PRIME,
a medicine has to show its potential to benefit patients with unmet
medical needs based on preclinical and/or early clinical data.
These medicines are considered priority medicines within the
European Union.
After an investigational candidate has been selected for PRIME,
developers are assigned a rapporteur from the Committee for
Medicinal Products for Human Use (CHMP) to provide continuous
support and help to build knowledge ahead of a Marketing
Authorization Application (MAA). A multidisciplinary group of
experts will provide broader guidance on the overall development
plan and regulatory strategy of the product. Companies are also
eligible for accelerated assessment at the time of their regulatory
application.
For more information, please visit the research and development
section of www.ema.europa.eu.
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. 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. Approximately 30 percent of cases are expected
to be SMA Type 2. SMA Type 2 typically presents between six and 18
months of age. Affected patients will never walk without support,
and SMA Type 2 results in mortality for more than 30 percent of
patients by age 25.
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 SMN1 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 benefit
patients with SMA Type 1, the potential expediting and streamlining
of the European regulatory approval process for AVXS-101 resulting
from the acceptance of AVXS-101 into the EMA’s PRIME program,
expectations regarding timing of the planned European Union pivotal
trial of AVXS-101 in patients with SMA Type 1 and 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 European Union, 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’ Quarterly Report on Form 10-Q for
the quarter ended September 30, 2016, filed with the SEC on
November 10, 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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