Table of Contents
Future SMA Type 1 Clinical Development
Based 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.
Future Applications and Opportunities
Based on preclinical data and our preliminary clinical observations to date in patients with SMA Type 1, we believe AVXS-101
may also have utility in other types of SMA, which result from the same genetic defect as SMA Type 1. We are currently evaluating preclinical data for the potential expansion of AVXS-101 into
SMA Types 2, and possibly 3, via the intrathecal route of administration. We intend to initiate a Phase 1 safety and dose-ranging clinical trial of AVXS-101 for SMA Type 2 via intrathecal
delivery in the second half of 2016, provided that the FDA allows it to proceed.
addition to our programs in SMA, we also intend to identify, acquire, develop and commercialize novel product candidates for the treatment of other rare and life-threatening
neurological genetic diseases that we believe can be treated with gene therapy.
Clinical drug supply of AVXS-101 for our ongoing Phase 1 clinical trial has been manufactured at NCH, which is approved as a
current good manufacturing practice, or cGMP, compliant facility. We manufacture AVXS-101 using adherent human embryonic kidney, or HEK, 293 cells. HEK293 cells have been used successfully to
manufacture numerous other gene therapy candidates that have been tested or are currently being tested in other clinical trials to date.
we scale our manufacturing of AVXS-101 to meet our expected needs for further clinical trials and potential U.S. commercial demand, we have initiated a technology transfer of NCH's
current manufacturing process of AVXS-101 to SAFC Carlsbad, Inc., or SAFC, another cGMP manufacturing facility. Based on an independent third-party quality and control audit, we believe that
SAFC meets cGMP and European regulatory compliance requirements. As a result, once the technology transfer to SAFC is complete, we believe SAFC will have sufficient capacity to meet demand for
AVXS-101 for our future U.S. SMA Type 1 clinical trials. Additionally, we will work with SAFC and other third parties in order to evaluate and develop manufacturing process improvements that
may increase the productivity and efficiency of our manufacturing network over the existing process.
parallel with the SAFC technology transfer, we intend to begin the process of establishing our own commercial scale cGMP-compliant manufacturing facility to enhance supply chain
control, increase supply capacity and help to ensure clinical and commercial demand is met in the event that AVXS-101 receives marketing approval.
intend to use AVXS-101 manufactured by SAFC or another third-party manufacturer in future U.S. clinical trials of AVXS-101. We expect to use a combination of internal and third-party
manufacturing sources to facilitate the production of long-term continuous supply of AVXS-101 and for clinical trials outside of the U.S.