- Ionis Pharmaceuticals Inc (NASDAQ: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.
