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Orphan Drug Designation among Latest Happenings at Ionis

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Nusinersen

As discussed earlier, Ionis’s (IONS) Nusinersen is a drug designed for the treatment of patients with spinal muscular atrophy (or SMA). The company entered a collaborative agreement with Biogen (BIIB) for the development and commercialization of Nusinersen in January 2012.

The company has classified Nusinersen as a drug for severe and rare therapeutic areas. It’s under phase III clinical studies for the treatment of both infants and children with spinal muscular atrophy.

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Recent developments for Nusinersen

Some of the developments for the drug Nusinersen are as follows:

  • Ionis received $2 million in 2Q16 from Biogen (BIIB) for the progression in Nusinersen’s phase III study as well as Ionis-BIIB4Rx.
  • The company announced the achievement of primary end points for the ENDEAR study in August 2016.
  • Presently, the company is evaluating Nusinersen in the phase III studies named Cherish and Endear in collaboration with Biogen. The company is also evaluating Nusinersen in two phase III studies for infants and children with SMA.
  • Cherish is a double-blind, randomized, sham-procedure controlled 15-month phase III study to evaluate the efficacy and safety of Nusinersen in children. The sample size is around 117 children.
  • Endear is a double-blind, randomized, sham-procedure controlled 13-month phase III study to evaluate the efficacy and safety of 12-mg doses of Nusinersen in infants. The sample size is around 110 infants, and the company expects to report data from this study in the first half of 2017.

The US Food and Drug Administration and the European Medicines Agency also granted Orphan Drug Designation for Nusinersen for the treatment of patients with SMA.

Investors can consider ETFs like the iShares Nasdaq Biotech ETF (IBB), which holds ~0.6% of its total assets in Ionis, in order to divest the risk. Apart from Ionis, IBB also holds 7.1% of its total assets in Regeneron Pharmaceuticals (REGN), 8.1% in Celgene (CELG), and 4% in Vertex (VRTX).

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