-Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has completed enrollment in the global Phase 3 YOSEMITE clinical trial of zodasiran, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated low density lipoprotein-cholesterol (LDL-C) and early onset cardiovascular disease. Arrowhead anticipates that YOSEMITE will be completed in mid-2027 and, pending successful clinical results, intends to seek regulatory approval in multiple geographies thereafter.

“Completing enrollment in the global YOSEMITE Phase 3 study represents an important milestone in the development of zodasiran for people living with HoFH, a rare disease with limited effective treatment options which carries a very high risk of developing atherosclerotic cardiovascular disease. By harnessing RNA interference to reduce ANGPTL3 expression, zodasiran is designed to lower atherogenic lipoproteins through a mechanism distinct from conventional LDL-C–lowering therapies,” said James Hamilton, M.D., Chief Medical Officer and head of R&D at Arrowhead. “The YOSEMITE Phase 3 study was initially designed to enroll 60 participants; however, strong global HoFH patient and physician interest led to an increased total of 70 patients enrolled. We believe this speaks to the remaining global unmet need in this population of patients with exceptionally high cardiovascular risk.”