SEC Filings

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

endpoint following its review of the final results of the pivotal trial. While the FDA also indicated its preference for the pivotal trial to include the achievement of certain motor milestones, such as sitting unassisted, as a potential co‑primary endpoint, there is no assurance that the FDA will determine that the achievement of these milestones will be an acceptable efficacy endpoint following its review of the final results of the pivotal trial. The FDA may not accept either or both of these endpoints as a measure of efficacy, which could result in a delay in approval of AVXS‑101. Any such delays could materially and adversely affect our business, financial condition, results of operations and prospects.

We may find it difficult to enroll patients in our clinical trials, which could delay or prevent us from proceeding with clinical trials of AVXS‑101.

Identifying and qualifying patients to participate in clinical trials of AVXS‑101 is critical to our success. The timing of our clinical trials depends on our ability to recruit patients to participate as well as completion of required follow‑up periods. If patients are unwilling to participate in our gene therapy studies because of negative publicity from adverse events related to the biotechnology or gene therapy fields, competitive clinical trials for similar patient populations, clinical trials in products employing our vector or our platform or for other reasons, the timeline for recruiting patients, conducting studies and obtaining regulatory approval of AVXS‑101 may be delayed. These delays could result in increased costs, delays in advancing AVXS‑101, delays in testing the effectiveness of AVXS‑101 or termination of clinical trials altogether.

We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics, to complete our clinical trials in a timely manner. Patient enrollment and trial completion is affected by factors including:

·

size of the patient population and process for identifying subjects;

·

design of the trial protocol;

·

eligibility and exclusion criteria;

·

perceived risks and benefits of the product candidate under study;

·

perceived risks and benefits of gene therapy‑based approaches to treatment of diseases;

·

availability of competing therapies and clinical trials, including Ionis Pharmaceuticals, Inc. and Biogen’s SPINRAZATM (nusinersen), which was approved by the FDA in December 2016 for use in a broad range of SMA patients, including for the treatment of SMA Type 1;

·

severity of the disease under investigation;

·

proximity and availability of clinical trial sites for prospective subjects;

·

ability to obtain and maintain subject consent;

·

risk that enrolled subjects will drop out before completion of the trial;

·

patient referral practices of physicians; and

·

ability to monitor subjects adequately during and after treatment.

AVXS‑101 is being developed to treat a rare condition. We plan to seek initial marketing approval in the United States and the European Union. Subject to the results of our Phase 1 clinical trial and further discussions with the FDA, we currently anticipate that any pivotal trial of AVXS‑101 could include using natural history of the disease as a comparator. Patients may opt not to enroll in our pivotal trial because of the commercial availability of another treatment

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