Biogen-Ionis Partnered Drug Shows Benefit In Children With Spinal Muscular Atrophy Previously Treated With Novartis' Zolgensma

Biogen Inc. (NASDAQ:BIIB) on Wednesday announced in a press release the topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for spinal muscular atrophy (SMA).

Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, Inc. (NASDAQ:IONS). Salanersen was discovered by Ionis.

Leveraging the same mechanism of action as Spinraza (nusinersen) but designed to achieve greater potency, salanersen can achieve high efficacy and enable once-a-year dose.

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An interim analysis of the phase one study in participants with SMA who were previously treated with Novartis AG’s (NYSE:NVS) gene therapy, Zolgensma (onasemnogene abeparvovec), was conducted to inform the decision on whether to move salanersen forward into registrational studies.

Both dose levels tested, ...