Ionis Pharmaceuticals’ (IONS) Volanesorsen is a drug designed to reduce apoC-III protein production and lower triglycerides for patients with dyslipidemia.
Akcea Therapeutics (AKCA) is engaged in pre-commercialization activities for Volanesorsen and will take care of the commercialization of the drug Volanesorsen once it receives approval from the US Food and Drug Administration and the European Commission. The drug is classified under severe and rare therapeutic areas. Akcea Therapeutics raised ~$190 million through an initial public offering, $50 million from Novartis, and $25 million from Ionis Pharmaceuticals as strategic investments.
Present status of Volanesorsen
Ionis Pharmaceuticals and its subsidiary Akcea Therapeutics announced the positive data from APPROACH, a phase III study for evaluating Volanesorsen for the treatment of patients with familial Chylomicronemia syndrome during the first half of 2017. The study evaluated the safety and efficacy of a weekly dose of 300 mg of Volanesorsen for 12 months.
On July 27, 2017, Ionis and Akcea submitted the marketing authorization application for Volanesorsen to the European Medicines Agency for the use of Volanesorsen for the treatment of patients with familial chylomicronemia syndrome. Presently, the drug is under Phase III clinical studies for the treatment of Familial Partial Lipodystrophy.
Other drugs in Akcea’s portfolio
Other drugs in Akcea’s portfolio include AKCEA-ANGPTL3-L Rx, which is under Phase II clinical studies for the treatment of rare hyperlipidemias, non-alcoholic fatty liver disease, and metabolic complications, as well as drugs AKCEA-APO(a)-L Rx and AKCEA-APOCII-L Rx, which are under Phase II clinical studies for the treatment of cardiovascular disease.
To divest the company-specific risks, investors can consider ETFs like the VanEck Vectors Biotech ETF (BBH), which holds ~1.7% of its total assets in Ionis Pharmaceuticals (IONS). BBH also holds 7.1% of its total assets in Allergan (AGN), 10.4% in Gilead Sciences (GILD), and 6.0% in Biogen (BIIB).