How Ionis’s Spinraza Performed in 4Q17

Ionis Pharmaceuticals’ (IONS) Spinraza (nusinersen) is a drug designed for the treatment of spinal muscular atrophy (or SMA), a severe motor-neuron disease. Biogen (BIIB) has held the worldwide development and commercialization rights for nusinersen since January 2012, while Ionis receives royalties for Spinraza sales from Biogen.

The above chart shows recent developments for Spinraza. The company has classified nusinersen under the severe and rare therapeutic areas. For US markets, the US Food & Drug Administration (or FDA) has approved Spinraza for the treatment of both pediatric and adult patients with spinal muscular atrophy. Also, Biogen holds the future developing, manufacturing, and commercializing rights for nusinersen.

Some of the developments for Spinraza (nusinersen) are as follows:

• For 4Q17, Ionis received $52.1 million from Biogen in Spinraza royalties, while for the full-year 2017, Ionis received $112.5 million in Spinraza royalties.
• On February 14, 2018, Ionis and Biogen announced the results from the CHERISH Study, a phase three study that evaluated Spinraza for the treatment of later-onset spinal muscular atrophy. The New England Journal of Medicine published the end of study results. 
• On November 1, 2017, Ionis and Biogen announced the end of study results for the ENDEAR study, and the results were published in The New England Journal of Medicine.
• On October 27, 2017, Ionis announced that Spinraza was awarded the Prix Galien USA Award for the Best Biotechnology Product in 2017.
• On October 5, 2017, Biogen released new data from the ENDEAR study, a phase III study. The new data shows that infants with SMA who initiated Spinraza treatment earlier showed greater benefits and improvement in motor function outcomes.

The PowerShares Dynamic Biotechnology & Genome ETF (PBE) holds 2.8% of its total investments in Ionis Pharmaceuticals (IONS), 4.8% in Biogen (BIIB), 4.9% in Amgen (AMGN), and 5.2% in Regeneron Pharmaceuticals (REGN).