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Wednesday, October 30, 2019

Regenxbio down on partial hold on Novartis’ Zolgensma

REGENXBIO (NASDAQ:RGNX) slips 4% premarket on light volume in reaction to Novartis’ disclosure of an FDA-imposed partial clinical hold on studies evaluating intrathecal (into the spinal canal) administration of gene therapy Zolgensma (anasemnogene abeparvovec-xioi) in patients with spinal muscular atrophy Type 2 (SMA2) after a potential safety signal was observed in an animal study.
RGNX receives Zolgensma-related milestones and royalties from licensor AveXis which Novartis acquired for $8.7B last year.
The action does not affect the commercial product since it is administered intravenously.
SMA competitors Ionis Pharmaceuticals (NASDAQ:IONS) and Biogen (NASDAQ:BIIB) are up 4% and 3%, respectively, premarket.

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