Arrowhead Pharmaceuticals announced today that enrollment has been completed for the global Phase III YOSEMITE clinical trial evaluating Zodasiran.

The study is designed to assess the efficacy and safety of Zodasiran in treating homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder that causes extremely high levels of low-density lipoprotein cholesterol and early-onset cardiovascular disease.

Where to begin

The YOSEMITE trial initially planned to enroll 60 participants, but based on strong global demand from HoFH patients and physicians, the final enrollment number was increased to 70.

Dr. James Hamilton, Chief Medical Officer and Head of Research and Development at Arrowhead, stated that this reflects the significant unmet medical needs still present within this patient population.

Why just 70 patients?

The study is a multicenter, randomized, double-blind, placebo-controlled trial involving patients aged 12 and older. Participants are randomized in a 2:1 ratio to receive either 200 mg of Zodasiran or a placebo via injection every three months for a total of four doses.

The primary endpoint is the percentage change in fasting LDL-C from baseline to month 12. After this period, participants may choose to enter an open-label extension phase.

Underlying reasons for the expansion

Previous Phase II GATEWAY study results showed that Zodasiran led to clinically meaningful reductions in LDL-C levels in HoFH patients, with a favorable safety profile and no drug-related serious adverse events or deaths.

Based on these positive outcomes, the advancement of the Phase III YOSEMITE study aims to further validate its efficacy and safety.

Arrowhead expects the study to be completed by mid-2027. If results are positive, the company plans to submit regulatory approval applications in multiple regions thereafter.

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Editor: Zhang Jun SF065