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Arrowhead Pharmaceuticals Completes Enrollment in Global Phase 3 YOSEMITE Study of Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia

Key Takeaway: Arrowhead Pharmaceuticals has successfully completed enrollment in its global Phase 3 YOSEMITE study for zodasiran, aimed at treating homozygous familial hypercholesterolemia (HoFH). This investigational RNA interference therapeutic targets a rare genetic disorder that causes high LDL cholesterol levels and early cardiovascular disease. The completion of enrollment marks a significant milestone in the development of this treatment.

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POSITIVE FACTORS

  • Completion of enrollment in a significant Phase 3 trial.
  • Zodasiran targets a rare but critical health condition.
  • Potential for addressing severe cholesterol issues in patients.

Full Press Release Details

PASADENA, Calif.--(BUSINESS WIRE)---- $arwr--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. Arrowhe

Frequently Asked Questions

What is the YOSEMITE study about?

The YOSEMITE study is a global Phase 3 clinical trial for zodasiran, targeting homozygous familial hypercholesterolemia.

What condition does zodasiran aim to treat?

Zodasiran is developed to treat homozygous familial hypercholesterolemia, a rare genetic disorder.

What milestone did Arrowhead Pharmaceuticals achieve?

Arrowhead Pharmaceuticals completed enrollment in the Phase 3 YOSEMITE study.

Why is homozygous familial hypercholesterolemia significant?

HoFH leads to severely elevated LDL cholesterol levels and early cardiovascular disease.

Last updated: Jul 27, 2026