Table of Contents
The development of product candidates for our Rett syndrome and ALS programs will be subject to many risks.
If we do not successfully develop and commercialize product candidates in these programs, our business prospects may be adversely affected.
Even if the results of IND-enabling studies for our gene therapy product candidates for the treatment of Rett syndrome and ALS substantiate
advancing these programs into clinical trials, the development of these product candidates will be subject to many risks. There is a high failure rate for drugs and biologic products proceeding
through clinical trials. Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials even after achieving promising results in
preclinical testing and earlier-stage clinical
trials. Data obtained from preclinical and clinical activities are subject to varying interpretations, which may delay, limit or prevent regulatory approval. In addition, we may experience regulatory
delays or rejections as a result of many factors, including due to changes in regulatory policy during the period of our product candidate development. Success in preclinical testing and early
clinical trials does not ensure that later clinical trials will generate the same results or otherwise provide adequate data to demonstrate the efficacy and safety of a product candidate. Frequently,
product candidates that have shown promising results in early clinical trials have subsequently suffered significant setbacks in later clinical trials. In addition, the design of a clinical trial can
determine whether its results will support approval of a product and flaws in the design of a clinical trial may not become apparent until the clinical trial is well advanced.
ability to successfully develop gene therapy treatments for Rett syndrome and ALS will be subject to many of the same development risks as our AVXS-101 product candidate for SMA,
including those discussed under the headings "Risk Factors Risks related to the development of our current product candidate" and "Risk Factors Risks related
to the commercialization of AVXS-101" in our Annual Report on Form 10-K for the year ended December 31, 2016, which is incorporated by reference into this prospectus supplement. These
risks include the risks of:
- completing preclinical studies and clinical trials with positive results;
- receiving marketing approvals from applicable regulatory authorities;
- obtaining, maintaining, defending and enforcing intellectual property rights important to the product candidates;
- establishing commercial manufacturing capabilities, or contracting with third-party manufacturers with such capabilities;
- establishing and maintaining collaborations, licensing or other arrangements;
- launching commercial sales, if and when approved;
- achieving acceptance, if and when approved, by patients and the medical community;
- effectively competing with other therapies; and
- maintaining an acceptable safety profile following approval.
we are unable to successfully develop and commercialize product candidates for Rett syndrome or ALS, we will not be able to obtain future revenues from these programs, which would
have a material adverse effect on our future business prospects, financial condition and results of operations.