REGENXBIO, AveXis Expand License Agreement

REGENXBIO Inc. and AveXis, Inc. have entered into an amendment which expands upon the exclusive, worldwide license agreement they entered into in March 2014 for the development and commercialization of products to treat spinal muscular atrophy (SMA). 

AveXis’ initial proprietary gene therapy candidate, AVXS-101, is in a pivotal trial for the treatment of SMA Type 1, and a Phase 1 trial for SMA Type 2. AVXS-101 uses REGENXBIO’s NAV AAV9 vector.

“An expanded relationship further aligns REGENXBIO with AveXis in their commitment to develop and commercialize novel gene therapy treatments for SMA, and serves as additional validation of our NAV Technology Platform and its potential utility in this therapeutic area,” said Kenneth T. Mills, president and chief executive officer of REGENXBIO. “We are encouraged by the innovation and progress of AVXS-101 and are pleased to broaden our relationship with AveXis by providing deeper technology access to AveXis as it advances its mission towards commercialization.”

Under the terms of the amendment, REGENXBIO granted AveXis exclusive, worldwide rights to all vectors in REGENXBIO’s NAV Technology Platform for the treatment of SMA. REGENXBIO will receive, in addition to the payments and royalties owed under the original 2014 License Agreement, an upfront payment of $80 million, an additional payment of $30 million after one year and an additional payment of $30 million after two years, and they are eligible to receive potential commercial milestone payments of up to $120 million. For any product developed for the treatment of SMA using the NAV AAV9 vector, REGENXBIO will continue to receive mid-single to low double-digit royalties on net sales as defined in the original 2014 License Agreement, and for any product developed for the treatment of SMA using a NAV vector, other than NAV AAV9, REGENXBIO will receive a low double-digit royalty on net sales.