REGENXBIO, a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, announced that it has received an accelerated license payment of $100 million under its license agreement with AveXis for the development and commercialization of products to treat spinal muscular atrophy (SMA), due to the acquisition of AveXis by Novartis.
REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
The accelerated license payment from Novartis consisted of $60 million in annual fees and a commercial milestone fee of $40 million. Under the License Agreement, in addition to the accelerated license payment, REGENXBIO remains eligible to receive a potential commercial milestone fee of $80 million and certain royalties on net sales for any product developed for the treatment of SMA using REGENXBIO’s NAV technology.
Novartis now holds exclusive rights to the NAV Technology Platform for the development of treatments for SMA, including AVXS-101, which uses REGENXBIO’s NAV AAV9 vector. In April 2018, AveXis reported that the six SMA patients who were at least one-month post gene transfer in the pivotal trial for AVXS-101 were exhibiting motor function improvements. SMA is the leading genetic cause of infant mortality.
REGENXBIO Inc., a US clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease.