SEC Filings

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

accumulated deficit of $359.6 million. We have devoted substantially all of our efforts to research and development, including clinical development of our gene therapy product candidate, AVXS‑101, investing in our manufacturing facility and manufacturing equipment, as well as to building out our management team and infrastructure. We expect that it could be several years, if ever, before we have a commercialized product candidate. We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future. The net losses we incur may fluctuate significantly from quarter to quarter. We anticipate that our expenses will increase substantially if, and as, we:

·

continue our research and the preclinical and clinical development of our product candidate, including our pivotal clinical trial for SMA Type 1, our Phase 1 clinical trial for SMA Type 2 and other planned clinical trials for AVXS‑101;

·

initiate additional clinical trials and preclinical studies for any additional product candidates that we may pursue in the future, including trials and studies related to our preclinical programs for Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene, or genetic ALS;

·

prepare our biologics license application, or BLA, and marketing authorization application for AVXS‑101;

·

manufacture current good manufacturing practices, or cGMP, material for clinical trials or potential commercial sales;

·

continue to establish and validate our commercial‑scale cGMP manufacturing facility;

·

further develop our gene therapy product candidate portfolio;

·

establish a sales, marketing and distribution infrastructure to commercialize any product candidate for which we may obtain marketing approval;

·

develop, maintain, expand and protect our intellectual property portfolio;

·

acquire or in‑license other product candidates and technologies; and

·

seek marketing approval for AVXS‑101 in the European Union and in other key geographies.

To become and remain profitable, we must develop and eventually commercialize one or more product candidates with significant market potential. This will require us to be successful in a range of challenging activities, including completing preclinical testing and clinical trials of AVXS‑101 and other future product candidates, developing and validating commercial scale manufacturing processes, obtaining marketing approval for our product candidates, manufacturing, marketing and selling any future product candidates for which we may obtain marketing approval and satisfying any post‑marketing requirements. We currently only have one product candidate, AVXS‑101, and we may never acquire, in‑license or develop additional product candidates. We may never succeed in any or all of these activities and, even if we do, we may never generate revenues that are significant or large enough to achieve profitability. If we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis. Our failure to become and remain profitable would decrease the value of our company and could impair our ability to raise capital, maintain our research and development efforts, expand our business or continue our operations. A decline in the value of our company also could cause you to lose all or part of your investment.

Because of the numerous risks and uncertainties associated with pharmaceutical product and biological product development, we are unable to accurately predict the timing or amount of increased expenses or when, or if, we will be able to achieve profitability. If we are required by the U.S. Food and Drug Administration, or FDA, or the European Medicines Agency, or EMA, or other regulatory authorities to perform studies in addition to those currently expected, or if there are any delays in completing our clinical trials or the development of AVXS‑101, our expenses could increase and revenue could be further delayed.

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