– Agency is supportive of proposed analytical
methods and comparability protocol –
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
alignment with the U.S. Food and Drug Administration (FDA) on the
company’s Good Manufacturing Practice (GMP) commercial
manufacturing process for AVXS-101 following the receipt of minutes
from the Type B Chemistry Manufacturing and Controls (CMC) meeting.
This alignment includes support for the proposed commercial
manufacturing process, the proposed analytical methods and
corresponding qualification and validation plans – inclusive of key
release assays such as potency, purity and identity – and the
proposed comparability protocol, which helps assess how similar the
product derived from the GMP process is to the original product
used in the Phase 1 trial of AVXS-101 in patients with spinal
muscular atrophy (SMA) Type 1.
Key components of the scalable, GMP commercial manufacturing
process are as follows:
- AveXis will continue to utilize HEK293 cells and an adherent
cell line.
- The commercial-scale GMP process will not include the use of
Hyperstacks for AAV9 vector production.
- The company has instead identified a novel adherent cell
culture approach that can more reliably produce product and has
greater surface area to potentially increase productivity relative
to Hyperstacks.
- Additional process development improvements, both up- and
downstream, were implemented to meet global regulatory GMP
expectations as well as to meet projected global patient
demand.
- AveXis plans to use the GMP process for all clinical and
commercial needs moving forward, including the company’s new Rett
and ALS programs.
Overall, the company believes there is alignment with the FDA on
its panel of analytical methods and the proposed assay
qualification/validation plans. Analytical methods are used to
assess how reliably and consistently the key product
characteristics can be determined in order to ensure patients
receive safe and effective product.
In the meeting minutes, the FDA made a request that the company
complete implementation of its potency assay qualification plan,
presented in the meeting, prior to initiation of upcoming clinical
studies. This assay utilizes the Delta 7 mouse model, which has
been used historically to assess AVXS-101 potency but now
incorporates additional elements to make it acceptable to global
regulatory authorities. Specifically, the agency stated, “We
believe that your potency assay development and qualification plans
have the potential to result in an acceptable potency assay, and we
are encouraged that the assay includes reference controls that
allow for calculation of relative potency and that the assay
qualification will include 3 independent runs. However, we do not
have enough information to predict the feasibility of your
plans.”
The company has already initiated the work necessary to address
this request and expects to have the data ready to submit to the
FDA in the August timeframe. AveXis plans to initiate a pivotal
study trial of AVXS-101 in SMA Type 1 in the U.S. and a Phase 1/2a
trial of AVXS-101 in SMA Type 2 in the U.S. later in the third
quarter of 2017, pending agreement from the FDA that these data are
sufficient.
“The goal of the CMC meeting was to align with FDA on our
commercial manufacturing process, analytical methods and
comparability protocol, all three of which we believe were achieved
in this collaborative and constructive discussion,” said Sean
Nolan, President and Chief Executive Officer of AveXis. “The team
has already made progress toward addressing the FDA’s request
regarding potency assay qualification, and we anticipate only a
modest impact to timelines. We are pleased with the outcomes of the
meeting and the progress we have made at the AveXis facility, and,
most importantly, believe we have a scalable GMP commercial process
in place to fulfill future patient demand and a path forward to
potentially utilize the Phase 1 data in our regulatory
pathway.”
Additionally, FDA is aligned with the company’s proposed
comparability protocol to assess the similarity of key
characteristics of the Nationwide Children’s Hospital (NCH)
product, used in the Phase 1 SMA Type 1 study, with the product
derived from the new GMP manufacturing process. Data from this
comparability work is ongoing and will include the above-mentioned
potency qualification data, which will be incorporated into the
data package along with the full Phase 1 clinical data, that will
be reviewed and discussed at the upcoming end-of-Phase 1 meeting,
likely to be requested later in August. This meeting will help
further inform the regulatory pathway options for AVXS-101. The
company anticipates providing an update on the outcome of that
meeting once the official minutes are available, which is
anticipated to be in the fourth quarter of 2017.
The company has previously stated that having its own
manufacturing facility is a key strategic capability necessary to
be successful in gene therapy. The company today reported that the
AveXis manufacturing facility is now fully operational for on-going
GMP production.
- Product for the planned SMA Type 1 pivotal trials and the Type
2 Phase 1/2a trial using intrathecal delivery has been produced at
the AveXis-owned facility, and will be used to initiate the trials,
pending FDA review of the potency assay qualification described
above and FDA agreement that designated batches of the product are
appropriate for a Phase 3 clinical study.
- The AveXis facility will be the primary production site to meet
projected commercial demand, and the company will use contract
manufacturing organizations to supplement production.
Today’s Conference Call InformationAveXis will
host a conference call and webcast at 4:30 p.m. EDT today, June 14,
2017. Analysts 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 39420674. 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 for 90 days 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 39420674.
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 Company’s commercial manufacturing
process, the Company’s potency assay development and the expected
timing of the submission of data regarding the potency assay to the
FDA, the expected timing of the initiation of the Company’s planned
future clinical trials, the expected timing of future meetings with
the FDA, the Company’s ability to meet future commercial demand for
AVXS-101 through its manufacturing facility and the possibility
that the Company may be able to utilize data from its Phase 1
clinical trial in the regulatory pathway 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
results of the Company’s ongoing potency assay development efforts
are unpredictable, uncertainties regarding the FDA’s agreement that
the data generated from such efforts will be sufficient to permit
the Company to progress in clinical trials with AVXS-101 drug
product from its new manufacturing process, potential timing delays
in the Company’s recruitment and enrollment of patients for its
planned future clinical trials, uncertainty regarding the potential
regulatory approval of AVXS-101 and 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 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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