Must-Know Developments for Ionis Pharmaceuticals in 2018


Mar. 27 2018, Updated 9:30 a.m. ET

Ionis’s recent developments

As discussed earlier, Ionis Pharmaceuticals (IONS) is focused on the development of drugs for the treatment of diseases with limited or no therapeutic options.

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Recent developments

Some of the companies’ key recent developments are as follows:

  • On March 15, 2018, Ionis and Akcea Therapeutics (AKCA) announced a worldwide license for the drug Inotersen and AKCEA-TTR-LRx (formerly referred to as IONIS-TTR-LRx). The transaction size is approximately $1.7 billion plus profit sharing.
  • On March 1, 2018, Ionis announced the results of its phase 1/2 study of the drug IONIS-HTTRx for the treatment of patients with early-stage Huntington’s disease. The top-line data show a significant reduction of disease-causing proteins in patients with Huntington’s disease.
  • On February 20, 2018, Ionis announced that the company has licensed the drug IONIS-AZ5-2.5Rx to AstraZeneca (AZN). The drug IONIS-AZ5-2.5Rx is an antisense drug designed to treat a genetic form of kidney disease, and AstraZeneca holds the development and commercialization rights for this drug. Ionis will receive an upfront payment of $30 million and may receive ~$300 million for additional developments, regulatory milestone payments, and royalties.
  • On February 14, 2018, Ionis and Biogen (BIIB) announced the results from the CHERISH Study, a phase three study that evaluated Spinraza for the treatment of later-onset spinal muscular atrophy. The end-of-study results were published in The New England Journal of Medicine.
  • On February 7, 2018, Akcea announced the completion of enrollments for the Phase 2b clinical study of the drug AKCEA-APO(a)-LRx.

The Vanguard Healthcare ETF (VHT) holds 0.2% of its total investments in Ionis Pharmaceuticals (IONS), 3.6% in Amgen (AMGN), and 1.6% in Biogen (BIIB).


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