Regenxbio (RGNX) Expands Relationship with AveXix (AVXS) Through Amended License Agreement for Development and Commercialization of Treatments for Spinal Muscular Atrophy

January 8, 2018 8:05 AM EST

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REGENXBIO Inc. (NASDAQ: RGNX) and AveXis, Inc. (NASDAQ: AVXS) today announced that they have entered into an amendment which expands upon the exclusive, worldwide license agreement they entered into in March 2014 (2014 License Agreement) 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 in addition to adding and amending certain terms of the 2014 License Agreement, including the modification of the assignment provision to now permit assignment in the event of a change of control by AveXis without REGENXBIO’s consent. 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 REGENXBIO is 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.

"We have been very encouraged by the clinical data demonstrated to date by AVXS-101 utilizing the NAV Technology Platform from REGENXBIO in infants with SMA Type 1 and are optimistic that we may also see robust results in our recently initiated Phase 1 trial in SMA Type 2," said Sean Nolan, President and Chief Executive Officer of AveXis. "We believe this expanded partnership with REGENXBIO, which provides exclusive rights to the entire REGENXBIO NAV Technology Platform, will further strengthen our leadership position in gene therapy treatments for this devastating disease.”

About SMA

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births.

The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality.

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