Zodasiran (AROANG3-2001) - (YOSEMITE)

July 27, 2026: Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced it has completed enrollment in the global Phase 3 YOSEMITE trial (NCT07037771) evaluating zodasiran (previously ARO-ANG3), an investigational RNA interference therapy targeting ANGPTL3, for homozygous familial hypercholesterolemia (HoFH). The study increased planned enrollment from 60 to 70 participants due to patient/physician interest.

YOSEMITE is a randomized, double-blind, placebo-controlled trial in adolescents and adults (age >12) with genetically or clinically diagnosed HoFH on maximally tolerated lipid-lowering therapy. Participants are randomized 2:1 to receive 200 mg zodasiran or placebo once every three months for four doses. The primary endpoint is percent change from baseline to month 12 in fasting LDL-C; an optional open-label extension is available after month 12.

Arrowhead expects study completion in mid-2027 and, contingent on successful results, plans to seek regulatory approvals in multiple geographies thereafter. Zodasiran has FDA Orphan Drug Designation for HoFH.