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Ionis’s Subsidiary Akcea Therapeutics and Volanesorsen

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Volanesorsen

Ionis Pharmaceuticals’ (IONS) volanesorsen is a drug designed to reduce the apoC-III protein production and lower triglycerides for patients with dyslipidemia. The company formed Akcea Therapeutics, a wholly owned subsidiary, to develop and commercialize volanesorsen.

Ionis has classified volanesorsen as a drug for severe and rare therapeutic areas. It’s in Phase III clinical studies for the treatment of patients with familial chylomicronemia syndrome (or FCS) and familial partial lipodystrophy (or FPL).

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Developments for volanesorsen

Below are some of the recent developments for volanesorsen:

  • On March 6, 2017, Ionis and Akcea announced positive results for the Phase III study APPROACH that evaluated volanesorsen for the treatment of patients with FCS.
  • APPROACH is a double-blind, randomized, placebo-controlled 12-month study to evaluate the efficacy and safety of a weekly dose of 300 mg (milligrams) of volanesorsen. The sample size is about 50 patients, and the company expects to report data from this study in 2017.
  • Akcea Therapeutics, a wholly owned subsidiary of Ionis, is engaged in precommercialization activities in order to prepare for the commercialization of volanesorsen, once it is approved.

The FDA (U.S. Food & Drug Administration) and the European Medicines Agency have granted volanesorsen Orphan Drug Designation for the treatment of patients with FCS.

You can consider ETFs such as the iShares Nasdaq Biotechnology (IBB), which holds ~1.0% of its total assets in Ionis, 6.3% in Regeneron Pharmaceuticals (REGN), 4.1% in Illumina (ILMN), 7.6% in Celgene (CELG), and 3.8% in Vertex Pharmaceuticals (VRTX).

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