Ionis Pharmaceuticals, Inc. announces the publication of positive Phase 2 data for Donidalorsen (formerly IONIS-PKK-LRx) in the New England Journal of Medicine. Donidalorsen is an investigational antisense medicine Ionis is evaluating for treating patients with HAE. In the Phase 2 study, Donidalorsen demonstrated a 90% reduction in angioedema attacks compared with placebo at the 80 mg monthly dose. There was significant improvement in quality of life as assessed by the Angioedema Quality of Life Questionnaire (AE-QoL) in the patients treated with Donidalorsen.
Donidalorsen is designed to reduce the production of prekallikrein, which plays a key role in the activation of inflammatory mediators associated with acute attacks of HAE. Donidalorsen uses Ionis’ advanced LIgand-Conjugated Antisense (LICA) technology platform.
“Positive Phase 2 data demonstrate that treatment with Donidalorsen reduced attack frequency and disease burden of HAE,” says Kenneth Newman, M.D., M.B.A., Ionis’ Vice-President of Clinical Development: “The promising findings from this study are particularly encouraging as we continue to advance the Phase 3 clinical study for Donidalorsen and underscore our commitment to deliver transformative treatments for patients with unmet therapeutic needs.”
Patients reported higher overall health-related quality of life (HRQoL) over 17 weeks with Donidalorsen. There were improvements observed across all individual domains of the AE-QoL compared with placebo.
The majority of adverse events during the study were mild with a frequency that was similar between patients receiving Donidalorsen and placebo groups.
(Source: Ionis)
