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ARTICLE | Clinical News

Ionis slides after AveXis reports SMA data for Zolgensma

April 17, 2019 9:13 PM UTC

Ionis lost $10.61 (13%) to $72.67 on Wednesday after AveXis reported that Zolgensma improved event-free survival, motor function and milestone achievement in the Phase III STR1VE trial to treat the type 1 form of spinal muscular atrophy. The gene therapy, which is under FDA Priority Review for the indication, is given via a one-time IV administration, while Ionis' SMA therapy, Spinraza nusinersen, must be administered chronically for life using lumbar puncture.

Zolgensma onasemnogene abeparvovec-xioi (AVXS-101) delivers the survival of motor neuron 1 telomeric (SMN1) gene using an adeno-associated viral 9 (AAV9) vector from RegenxBio Inc. (NASDAQ:RGNX). Novartis AG (NYSE:NVS; SIX:NOVN), which obtained Zolgensma through its $8.7 billion acquisition of AveXis Inc., expects a decision from FDA next month...