SEC Filings

AVEXIS, INC. filed this Form 10-K on 02/28/2018
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Biologics License Application, or BLA, meeting for AVXS-101 in SMA with the FDA in the second quarter of 2018. The EMA granted access into its PRIority MEdicines, or PRIME, program for AVXS-101 for the treatment of SMA Type 1. PRIME is intended to enhance support for the development of medicines—specifically those that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options—through early and proactive support by EMA to optimize the generation of robust data and development plans, and potentially expedite the assessment of the marketing authorization application so these medicines may reach patients sooner. In addition, we intend to seek EMA Pre-Submission Scientific Advice in the first half of 2018 to determine if the data from our Phase 1 study of AVXS‑101 may meet the evidentiary requirements for the conditional approval pathway in the European Union.

We are also currently conducting a Phase 1 clinical trial of AVXS-101 for the treatment of SMA Type 2. SMA Type 2 typically presents between six and 18 months of age, and those affected will never walk without support and most will never stand without support. SMA Type 2 results in mortality in more than 30% of patients by the age of 25. In addition to our ongoing clinical trials, we intend to expand our clinical development program of AVXS-101 for the treatment of SMA by initiating three additional clinical trials to further evaluate AVXS-101, including in new patient populations.  First, we intend to initiate a pivotal trial of AVXS-101 for the treatment of SMA Type 1 in Europe during the first half of 2018.  Second, we intend to initiate a trial for pre-symptomatic SMA patients with two, three and four copies of the SMN2 backup gene, who are less than six weeks of age at the time of gene therapy, to evaluate appropriate clinical endpoints of a one-time intravenous, or IV, dose of AVXS-101 in the first half of 2018. Finally, we intend to initiate a pediatric "all comers" trial for approximately 50 patients between approximately six months and 18 years of age who do not qualify for other AVXS-101 trials at the time of gene therapy, to evaluate a one-time intrathecal, or IT, dose of AVXS-101 in the late fourth quarter of 2018 or early 2019.

We have an exclusive, worldwide license with Nationwide Children’s Hospital, or NCH, under certain patent applications related to both the intravenous and intrathecal delivery of AVXS‑101 for the treatment of all types of SMA, and an exclusive, worldwide license, with rights to sublicense, from REGENXBIO Inc., or REGENXBIO, to any recombinant AAV vector in REGENXBIO’s intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans. In addition, we have a non‑exclusive, worldwide license agreement with Asklepios BioPharmaceutical Inc., or AskBio, under certain patents and patent applications owned by the University of North Carolina and licensed to AskBio for the use of its self‑complementary DNA technology for the treatment of SMA.

In 2013, in connection with the exclusive license agreement with NCH, we formed a collaboration with NCH to explore the use of gene therapy for the treatment of SMA and secured our first institutional investors and expanded our leadership team. Our current operations are a result of this collaboration with NCH and research conducted by our Chief Scientific Officer, Brian Kaspar, Ph.D. Dr. Kaspar has over 20 years of gene therapy experience, and until September 2016 served as a principal investigator in the Center for Gene Therapy at The Research Institute at NCH. NCH is a leading pediatric gene therapy research institute.

In addition to developing AVXS-101 to treat SMA, we plan to develop other novel treatments for two additional rare neurological monogenetic diseases, Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene, or genetic ALS.

To execute on our mission, we have assembled a management team that includes individuals with expertise in gene therapy, regulatory development, product and clinical development, manufacturing, medical affairs and commercialization, with a history of success in building and operating innovative biotechnology and healthcare companies focused on rare and life‑threatening diseases. This team is led by our President and Chief Executive Officer, Sean P. Nolan, who brings over 26 years of broad leadership and management experience in the biopharmaceutical industry to AveXis. Prior to AveXis, Mr. Nolan was the chief business officer of InterMune, Inc. where he led multiple functions across the organization, including North American commercial operations, global marketing, corporate and business development, and global manufacturing and supply chain. Our other management team members also have successful track records developing and commercializing drugs through previous experiences at companies such as Abbott Laboratories, Amgen, Centocor, InterMune, Hospira, MedImmune, Novartis, Pfizer, Daiichi Sankyo and Quest Diagnostics.



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