Ionis Pharma Announces Publication Of Positive Donidalorsen Data For Angioedema Attacks

  • Ionis Pharmaceuticals Inc IONS announced the publication of Phase 2 data for donidalorsen in the New England Journal of Medicine (NEJM). 
  • Donidalorsen is an investigational antisense medicine under development for hereditary angioedema (HAE), a disorder characterized by recurrent episodes of severe swelling.
  • In the Phase 2 study, donidalorsen demonstrated a 90% reduction in angioedema attacks compared with placebo at the 80 mg monthly dose. 
  • There was a significant improvement in quality of life as assessed by the Angioedema Quality of Life Questionnaire (AE-QoL) in the patients treated with donidalorsen.
  • Also See: Pfizer, Ionis Discontinue Vupanorsen Clinical Development Program.
  • Donidalorsen is designed to reduce the production of prekallikrein, which plays a crucial role in the activation of inflammatory mediators associated with acute attacks of HAE. 
  • The data showed that the mean monthly rate of angioedema attacks was 0.23 and 2.21 in patients receiving donidalorsen and placebo, respectively.
  • The mean attack rate between Weeks 5 and 17 was 0.07 and 2.06.
  • Most adverse events during the study were mild, with a similar frequency between patients receiving donidalorsen and placebo groups. 
  • Price Action: IONS shares traded 1.03% higher at $34.33 premarket on the last check Thursday.
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