Ionis registers Phase 3 pediatric study of olezarsen for FCS

This week's update centers on a newly posted pediatric trial for Ionis's olezarsen program.

• Ionis Pharmaceuticals is recruiting for a Phase 3 open-label study of olezarsen (ISIS 678354) in pediatric patients with Familial Chylomicronemia Syndrome. The study is a multi-center open-label design evaluating safety, pharmacokinetics, efficacy, and pharmacodynamics of olezarsen in participants aged 2 to less than 18 years with FCS.¹ The trial has an estimated enrollment of 12 participants¹ and its primary outcome is percent change from baseline in fasting triglycerides at 6 months¹. Dosing is split by age cohort, with participants aged 12 to less than 18 years receiving multiple monthly subcutaneous doses of olezarsen for up to 1 year¹ in Cohort 1, and participants aged 2 to less than 12 years receiving monthly doses based on data from Cohort 1¹ in Cohort 2.