SEC Filings

AVEXIS, INC. filed this Form 10-K on 02/28/2018
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Risks related to our business operations

If we are not successful in discovering, developing and commercializing additional product candidates, our ability to expand our business and achieve our strategic objectives would be impaired.

Although a substantial amount of our efforts will focus on our ongoing clinical trials and potential approval of AVXS‑101, a key element of our strategy is to discover, develop and potentially commercialize a portfolio of product candidates to treat rare and life‑threatening neurological genetic diseases, including Rett syndrome and genetic ALS, for which we are conducting preclinical studies. We intend to do so by exploring strategic partnerships for the development of new products and in‑licensing technologies leading to the development of new product candidates. Identifying new product candidates requires substantial technical, financial and human resources, whether or not any product candidates are ultimately identified. Even if we identify product candidates that initially show promise, we may fail to successfully develop and commercialize such product candidates for many reasons, including the following:


the research methodology used may not be successful in identifying potential product candidates;


competitors may develop alternatives that render our product candidates obsolete;


product candidates we develop may nevertheless be covered by third parties’ patents or other exclusive rights that we are unable to license;


a product candidate may, on further study, be shown to have harmful side effects or other characteristics that indicate it is unlikely to be effective or otherwise does not meet applicable regulatory criteria;


a product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; and


a product candidate may not be accepted as safe and effective by patients, the medical community or third‑party payors.

If we are unsuccessful in identifying and developing additional product candidates, our potential for growth may be impaired.

We may fail to capitalize on other potential product candidates that may be a greater commercial opportunity or for which there is a greater likelihood of success.

The success of our business depends upon our ability to develop and commercialize AVXS‑101. AVXS‑101 may be shown to have harmful side effects, may be commercially impracticable to manufacture or may have other characteristics that may make the product unmarketable or unlikely to receive marketing approval.

Additionally, because we have limited resources, we may forego or delay pursuit of opportunities with certain programs or product candidates or for indications that later prove to have greater commercial potential. Our spending on current and future research and development programs may not yield any commercially viable product candidates. If we do not accurately evaluate the commercial potential for a particular product candidate, we may relinquish valuable rights to that product candidate through strategic collaboration, licensing or other arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate. Alternatively, we may allocate internal resources to a product candidate in a therapeutic area in which it would have been more advantageous to enter into a partnering arrangement.

If any of these events occur, we may be forced to abandon our development efforts with respect to a particular product candidate or fail to develop a potentially successful product candidate, which could have a material adverse effect on our business, financial condition, results of operations and prospects.



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