|AVEXIS, INC. filed this Form S-1/A on 02/09/2016|
Table of Contents
We may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and
efficacy to the satisfaction of applicable regulatory authorities.
Before obtaining marketing approval from regulatory authorities for the sale of AVXS-101, we must conduct extensive clinical trials to
demonstrate the safety and efficacy of that product candidate for its intended indications. Clinical testing is expensive, time-consuming and uncertain as to outcome. We cannot guarantee that any
clinical trials will be conducted as planned or completed on schedule, if at all. A failure of one or more clinical trials can occur at any stage of testing. Events that may prevent successful or
timely completion of clinical development include:
- delays in reaching a consensus with regulatory authorities on trial design;
- delays in reaching agreement on acceptable terms with prospective CROs and clinical trial sites;
- delays in opening clinical trial sites or obtaining required IRB or independent Ethics Committee approval at each clinical trial site;
- delays in recruiting suitable subjects to participate in our future clinical trials, including because such trials may be
placebo-controlled trials and patients are not guaranteed to receive treatment with our product candidate;
- imposition of a clinical hold by regulatory authorities as a result of a serious adverse event or after an inspection of our clinical
trial operations, trial sites or manufacturing facilities;
- failure by us, any CROs we engage or any other third parties to adhere to clinical trial requirements;
- failure to perform in accordance with FDA good clinical practices, or GCP, or applicable regulatory guidelines in the European Union
and other countries;
- delays in the testing, validation, manufacturing and delivery of AVXS-101 to the clinical sites, including delays by third parties
with whom we have contracted to perform certain of those functions;
- delays in having subjects complete participation in a trial or return for post-treatment follow-up;
- clinical trial sites or subjects dropping out of a trial;
- selection of clinical endpoints that require prolonged periods of clinical observation or analysis of the resulting data;
- occurrence of serious adverse events associated with the product candidate that are viewed to outweigh its potential benefits;
- occurrence of serious adverse events in trials of the same class of agents conducted by other sponsors; or
- changes in regulatory requirements and guidance that require amending or submitting new clinical protocols.
inability to successfully complete preclinical and clinical development could result in additional costs to us or impair our ability to generate revenues from product sales,
regulatory and commercialization milestones and royalties. In addition, if we make manufacturing or formulation changes to AVXS-101, we may need to conduct additional studies to bridge our modified
product candidate to earlier versions. Clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize AVXS-101 or allow our competitors to bring
products to market before we do, which could impair our ability to successfully commercialize AVXS-101 and may harm our business, financial condition, results of operations and prospects.
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