Ionis Pharmaceuticals, Inc. announces additional positive interim data from a Phase 2 open-label extension (OLE) study of donidalorsen, an investigational antisense medicine for the treatment of patients with HAE. Positive interim data presented in November 2022 showed that treatment with donidalorsen resulted in an overall sustained mean reduction in HAE attack rates of 95% from baseline. In the latest update, patients treated for one year with donidalorsen showed a clinically meaningful 24-point mean improvement in their Angioedema Quality of Life (AE-QoL) total score relative to baseline with improvements observed in all domains. An improvement of 6 points or more is considered clinically meaningful.

“The improvement in quality of life demonstrated in patients treated with donidalorsen for one year were clinically meaningful and further support our belief in this medicine’s potential to be a best-in-class prophylactic treatment for patients with HAE,” says Richard S. Geary, PhD, Executive Vice President and Chief Development Officer at Ionis. “We continue to be pleased with the progress of the Phase 3 OASIS study of donidalorsen, which remains on track to complete enrollment this year.”
(Source: Ionis)