Arrowhead Completes Enrollment in Phase 3 Zodasiran Trial for Rare Cholesterol Disorder — Arrowhead Pharmaceuticals, Inc.

Event summary

Arrowhead Pharmaceuticals completed enrollment in the global Phase 3 YOSEMITE study for zodasiran, an RNA interference therapeutic targeting homozygous familial hypercholesterolemia (HoFH).
The trial enrolled 70 patients, exceeding the initial target of 60 due to high interest from physicians and patients.
Arrowhead anticipates completing the study by mid-2027, with regulatory approval filings expected thereafter if results are positive.

The big picture

Arrowhead’s completion of the YOSEMITE trial marks a critical step in developing a first-in-class treatment for HoFH, a condition with limited therapeutic options. The success of zodasiran could validate RNA interference as a viable mechanism for addressing rare genetic disorders, potentially opening new avenues in lipid management and cardiovascular disease prevention.

What we're watching

Regulatory Pathway: Whether the Phase 3 data will support regulatory approval filings in multiple geographies and the timeline for potential approvals.
Market Potential: The commercial viability of zodasiran given the ultra-rare nature of HoFH and the high unmet need for effective treatments.
Competitive Landscape: How Arrowhead’s RNAi approach will differentiate zodasiran from existing LDL-C lowering therapies in a highly specialized patient population.