Benzinga | Oct 27, 2020 07:20AM EDT

REGENXBIO To Receive $80.0M Milestone Payment From Novartis AG

ROCKVILLE, Md., Oct. 27, 2020 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV(r) Technology Platform, today announced that it will receive a milestone payment of $80.0 million from Novartis AG based upon the achievement of $1.0 billion in cumulative net sales of Zolgensma. REGENXBIO expects to recognize this revenue in the third quarter of 2020.

"Zolgensma is a transformative, one-time gene therapy for the treatment of pediatric patients with SMA, a debilitating and potentially deadly disease, and the first approved gene therapy based on REGENXBIO's NAV Technology. We believe that this achievement provides further validation of the broad potential of NAV Technology to improve the lives of patients in need," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are pleased to have contributed to a therapy that has impacted the lives of over 700 patients with SMA and their families around the world."

Zolgensma uses REGENXBIO's proprietary NAV AAV9 vector and was designed and developed by AveXis, Inc., now known as Novartis Gene Therapies. In May 2019, Zolgensma was granted U.S. Food and Drug Administration (FDA) approval for the treatment of spinal muscular atrophy (SMA) in pediatric patients who are less than two years of age, and has since been approved in Europe, Japan and other countries. REGENXBIO continues to receive tiered royalties on sales of Zolgensma. Since FDA approval in 2019, REGENXBIO has earned more than $140.0 million in revenues from royalties and commercial milestone payments related to Zolgensma through the third quarter of 2020.