level that we presently intend to evaluate in future trials. We will likely be required to conduct a pivotal trial followed by review and approval of the product candidate by the FDA before
making any claims of efficacy as to our product candidate or the appropriate dose. Although the results of the Finkel 2014 Study were not pre-specified as a comparator for our trial, we believe the
Finkel 2014 Study provides a useful context to consider the results to date of our trial. Based on preliminary results of the 15 patients dosed as of December 31, 2015, we have observed
AVXS-101 to be generally well-tolerated.
on the data observed from our ongoing Phase 1 clinical trial, we intend to engage in discussions with the FDA during the first half of 2016 to discuss the next steps in our
AVXS-101 development plan for SMA Type 1. Subject to the outcome of our discussions with the FDA and European regulatory authorities, we intend to initiate pivotal trials of AVXS-101 for SMA
Type 1 in each of the U.S. and the European Union in the first half of 2017.
have an exclusive, worldwide license with 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 from a predecessor to REGENXBIO Inc., or REGENXBIO, under certain patents and patent applications, to use the AAV9 capsid 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 for the use of its self-complementary DNA technology for the treatment of SMA.
We are building a patient-centric business with the goal of developing innovative gene therapy treatments that transform the lives of
patients and their families suffering from rare and life-threatening neurological genetic diseases. In order to accomplish this goal, we plan to execute on the following key
- rapidly advance our SMA Type 1 program through clinical trials in the United
- expand the development of AVXS-101 into SMA Types 2 and
- advance the development of AVXS-101 outside of the United
- build a pipeline of gene therapy treatments for other rare and life-threatening neurological genetic
- continue to invest in and develop robust and sustainable manufacturing processes and multiple supply sources
to ensure the supply of high-quality products;
- invest in developing and accessing intellectual property to further expand our product
- continue to develop a strong, collaborative network of key stakeholders, including patient advocacy groups,
healthcare professionals, key opinion leaders and research institutions, to inform our clinical development and commercialization strategies.
AveXis was founded by John D. Harkey, Jr., our former Chairman, in 2010. Under Mr. Harkey's leadership, 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, Dr. Brian Kaspar. Dr. Kaspar has over 20 years of gene therapy experience,