SEC Filings

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

meet commercial demand in the event that AVXS‑101 receives marketing approval and for our other programs. We continue to utilize our internal process science group and work with third parties in order to evaluate and develop manufacturing process improvements that may increase the productivity and efficiency of our manufacturing process.


The biotechnology and pharmaceutical industries are highly competitive. In particular, the field of gene therapy is characterized by rapidly advancing technologies, intense competition and a strong emphasis on proprietary products. We face substantial competition from many different sources, such as large and specialty pharmaceutical and biotechnology companies, academic research institutions, government agencies and public and private research institutions.

At this time, there is one FDA‑approved, and one EMA‑approved, treatment for SMA. There are currently no treatments approved by the FDA for SMA that specifically address the underlying cause of the disease, the genetic defect in the SMN1 gene. The standard of care for patients with SMA Type 1 has been generally limited to palliative therapies, including life‑long respiratory care, ventilator support, nutritional care, orthopedic care and physical therapy; however, in December 2016, an alternative approach for the treatment of SMA called alternative splicing, which seeks to achieve more efficient production of full‑length SMN protein from the SMN2 gene, SPINRAZATM (nusinersen) received FDA approval and was subsequently launched in the United States. In addition, a number of companies are developing drug candidates for SMA. We are aware of other companies exploring gene therapy treatments in preclinical development, including Biogen, Voyager Therapeutics, Inc. and Généthon. In addition to a gene therapy‑based solution, alternative approaches in clinical development for the treatment of SMA include alternative splicing, neuroprotection and muscle enhancer technologies:


Alternative splicing seeks to increase production of full‑length SMN protein from the SMN2 backup gene. Ionis Pharmaceuticals, Inc., together with its licensee Biogen, received FDA approval in December 2016 for SPINRAZA™ (nusinersen) under Priority Review, and EMA approval in April 2017 and European Commission approval in June 2017, for the treatment of SMA in pediatric and adult patients and subsequently launched the product in the United States and Europe. Additionally, Roche Holding Ltd is conducting Phase 2 clinical trials in SMA Types 1, 2 and 3 and Novartis Corporation is conducting Phase 2 clinical trials for SMA Type 1 patients in the EU.


Neuroprotectants seek to mitigate the loss of motor neurons. Trophos SA, which has been acquired by Roche Holding Ltd, has completed its Phase 2 clinical trial of its lead neuroprotectant product candidate, Olesoxime (TRO19622), in patients between the ages of three and 25 with SMA Types 2 and 3.


Other approaches seek to improve muscle function and performance in SMA via additional mechanisms. Cytokinetics, in collaboration with Astellas, is conducting a Phase 2 trial of CK‑2127107 (CK‑107), a skeletal troponin activator, in patients with SMA Types 2, 3 or 4 who are 12 years and older. In November 2017, Catalyst announced it initiated a Phase 2 trial for Firdapse (amifampridine), a small molecule that enhances neuromuscular junction transmission, in ambulatory SMA Type 3 patients.

Intellectual Property

We strive to protect and enhance the proprietary technology, inventions, and improvements that are commercially important to the development of our business. We intend to seek, maintain and defend our patent rights, whether developed internally or licensed from third parties. We also rely on trade secrets relating to our proprietary technology and on know‑how, continuing technological innovation and in‑licensing opportunities to develop, strengthen and maintain our proprietary position in the field of gene therapy. Additionally, we intend to rely on regulatory protection afforded through orphan drug designations, data exclusivity and market exclusivity as well as patent term extensions, where available.

Our future commercial success depends, in part, on our ability to: obtain and maintain patent and other proprietary protection for commercially important technology, inventions and know‑how related to our business; defend



© AveXis, Inc. All Rights Reserved.