Ionis shares significant business and pipeline progress at Innovation Day, including on donidalorsen

Ionis Pharmaceuticals today held an investor and analyst event, Ionis Innovation Day: Discovering, Developing and Delivering Transformational Medicines. Presentations from Ionis leaders and external physicians highlight key pipeline and technology highlights.

Brett P. Monia, Ph.D., Ionis’ Chief Executive Officer, said: “Our leadership in RNA-targeted medicine includes multiple modalities empowering us to advance the right medicines for the right targets. Our powerful and prolific research and development engine has discovered ground-breaking medicines for people with devastating diseases.”

As part of the Innovation Day, an update on the potential HAE treatment, donidalorsen was provided:

  • Donidalorsen was recently granted U.S. Orphan Drug Designation
  • Ionis plans to report Phase 3 results with donidalorsen for prophylactic treatment of hereditary angioedema (HAE) in the first half of 2024. Ionis also plans to report topline data from a second Phase 3 study evaluating switching from currently approved HAE treatments in mid-2024.
  • Two-year results from the Phase 2 open-label-extension trial showed that donidalorsen treatment resulted in a 96% overall sustained mean reduction from baseline in HAE attack rates. These data will be presented at a medical congress later this year.
  • Raffi Tachdjian, M.D., MPH, FAAAAI, FACAAI, Associate Clinical Professor of Medicine and Pediatrics UCLA School of Medicine; Chief, Division of Allergy and Immunology Providence St. John Medical Center will discuss the need for new prophylactic HAE treatments as more than one-third of patients on prophylactic treatment report more than two attacks per month.

(Source: Ionis Pharmaceuticals)