DE MONITOR - Dé Pharming Expert schreef op 17 oktober 2023 19:50:
Weer een concurrent met goede progressie.
IONIS SHARES SIGNIFICANT BUSINESS AND PIPELINE PROGRESS, INCLUDING ON DONIDALORSEN
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.