SEC Filings

10-K
AVEXIS, INC. filed this Form 10-K on 02/28/2018
Entire Document
 

·

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 when an approved product is available, which may be exacerbated given the rare conditions of SMA, Rett syndrome and genetic ALS;

·

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 or other product candidates 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.

Any 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 or other product candidates, 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 other product candidates or allow our competitors to bring products to market before we do, which could impair our ability to successfully commercialize AVXS‑101 or other product candidates and may harm our business, financial condition, results of operations and prospects.

Additionally, if the results of our clinical trials are inconclusive or if there are safety concerns or serious adverse events associated with AVXS‑101 or other product candidates, we may:

·

be delayed in obtaining marketing approval for such product candidate, if at all;

·

obtain approval for indications or patient populations that are not as broad as intended or desired;

·

obtain approval with labeling that includes significant use or distribution restrictions or safety warnings;

·

be subject to additional post‑marketing testing requirements;

·

be subject to changes in the way the product is administered;

49


 


© AveXis, Inc. All Rights Reserved.