SEC Filings

AVEXIS, INC. filed this Form 10-K on 02/28/2018
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Risks related to our reliance on third parties

A third party conducted the only clinical trial of AVXS‑101 to date and sponsored this trial through November 6, 2015, and our ability to influence the design and conduct of this trial was 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.

Until November 6, 2015, we had not sponsored any clinical trials relating to AVXS‑101. Prior to that, our third‑party research institution collaborator, Nationwide Children’s Hospital, or NCH, had sponsored our Phase 1 clinical trial for the treatment of SMA Type 1 relating to this product candidate, or the NCH trial, under an investigational new drug application, or IND, held by Dr. Jerry Mendell, the principal investigator at NCH. We have assumed control of the overall clinical and regulatory development of AVXS‑101 for future clinical trials. Although we sponsor the clinical trial, NCH has certain reversionary rights in the case of acts or omissions constituting negligence or willful misconduct or failure to comply with applicable law. Failure to maintain sponsorship of INDs for AVXS‑101 could negatively affect the timing of our potential future clinical trials. Such an impact on timing could increase research and development costs and could delay or prevent us from obtaining regulatory approval for AVXS‑101, either of which could have a material adverse effect on our business.

Further, we did not control the design or conduct of the NCH trial. It is possible that the FDA will not accept the NCH trial as part of a future registration package in support of a license application, for any of one or more reasons, including the safety, purity, and potency of the product candidate, the degree of product characterization, elements of the design or execution of the previous trials or safety concerns, or other trial results. For example, in 2016 the FDA completed an inspection of the NCH trial as conducted at NCH’s facilities and made observations on an FDA Form 483. We do not believe these observations will result in our inability to rely on the data from this trial, but the FDA may disagree, which could impact our development program. We may also be subject to liabilities arising from any treatment‑related injuries or adverse effects in patients enrolled in the NCH trial. As a result, we may be subject to unforeseen third‑party claims and delays in our potential future clinical trials. Any such delay or liability could have a material adverse effect on our business.

Although we have assumed control of the overall clinical and regulatory development of AVXS‑101 going forward, we have been dependent on contractual arrangements with NCH for the clinical implementation of the NCH trial. Such arrangements provide us certain information rights with respect to the NCH trial, including access to and the ability to use and reference the data, including for our own regulatory filings, resulting from the NCH trial. If these obligations are breached by NCH, or if the data prove to be inadequate compared to the first‑hand knowledge we might have gained had the completed trial been a corporate‑sponsored trial, then it may adversely affect or cause a delay in our development program. Additionally, the FDA may disagree with the sufficiency of our right to reference the preclinical, manufacturing, or clinical data generated by the NCH trial, or our interpretation of preclinical, manufacturing, or clinical data from the NCH trial. If so, the FDA may require us to obtain and submit additional preclinical, manufacturing, or clinical data before we may begin our planned trials and/or may not accept such additional data as adequate to begin our planned trials.

We may in the future enter into collaborations with third parties to develop AVXS‑101 or other product candidates. If these collaborations are not successful, our business could be adversely affected.

We may potentially enter into collaborations with third parties in the future. Any collaborations we enter into in the future may pose several risks, including the following:


collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations;


collaborators may not perform their obligations as expected;


the clinical trials conducted as part of these collaborations may not be successful;



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