|AVEXIS, INC. filed this Form S-1/A on 02/01/2016|
Table of Contents
- Success in preclinical studies or early clinical trials may not be indicative of results obtained in later trials.
- AVXS-101 may cause undesirable side effects or have other properties that could delay or prevent its regulatory approval, limit the
commercial potential or result in significant negative consequences following any potential marketing approval.
- Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize
AVXS-101 and the approval may be for a more narrow indication than we seek.
- Even if we obtain and maintain approval for AVXS-101 from the FDA, we may never obtain approval for AVXS-101 outside of the United
States, which would limit our market opportunities and adversely affect our business.
- The commercial success of AVXS-101 will depend upon its degree of market acceptance by physicians, patients, third-party payors and
others in the medical community, which may be limited if, among other things, genetic screening for SMA in newborns is not approved by applicable federal and state regulatory authorities.
- A third party has conducted the only clinical trial of AVXS-101 to date and had sponsored this trial through November 6, 2015,
and our ability to influence the design and conduct of this trial has been limited. Any failure by a third party to meet its obligations with respect to the clinical and regulatory development of
AVXS-101 may delay or impair our ability to obtain regulatory approval for AVXS-101 and result in liability for us.
- We are in the process of changing our third-party manufacturer of AVXS-101 and, although we intend to establish our own AVXS-101
manufacturing facility, we expect to utilize third parties to conduct our product manufacturing for the foreseeable future. Therefore, we are subject to the risk that these third parties may not
- If we are unable to establish sales, medical affairs and marketing capabilities or enter into agreements with third parties to market
and sell AVXS-101, we may be unable to generate any product revenue.
- Delays in obtaining regulatory approval of our manufacturing process and facility or disruptions in our manufacturing process may
delay or disrupt our product development and commercialization efforts. To our knowledge, to date, no cGMP gene therapy manufacturing facility in the United States has received approval from FDA for
the manufacture of an approved gene therapy product.
- We face significant competition in an environment of rapid technological change and the possibility that our competitors may achieve
regulatory approval before us or develop therapies that are more advanced or effective than ours, which may adversely affect our financial condition and our ability to successfully market or
- Our rights to develop and commercialize AVXS-101 are subject to the terms and conditions of licenses granted to us by others.
- If we are unable to obtain and maintain patent protection for our current product candidate, any future product candidates we may
develop and our technology, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to
ours, and our ability to
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