Arrowhead Pharmaceuticals Completes Enrollment in Global Phase 3 YOSEMITE Study of Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia
Arrowhead Pharmaceuticals (NASDAQ: ARWR) said it completed enrollment in the global Phase 3 YOSEMITE trial of zodasiran for homozygous familial hypercholesterolemia. The study enrolled 70 patients (2:1 randomization) and is expected to complete in mid-2027. The primary endpoint is percent change in fasting LDL-C at month 12.
How this was made
The 30-second read
Why it matters
Completing enrollment (70 subjects, increased from an initial 60 design) reduces recruitment uncertainty and preserves the planned mid-2027 completion timeline, which is a key gating item for any subsequent regulatory submission plans.
Market read
A late-stage clinical execution milestone for ARWR’s lead RNAi lipid program, with the next major catalyst positioned for mid-2027 completion and subsequent regulatory steps if results are positive.
What to watch
The primary endpoint is percent change in fasting LDL-C at month 12; investors may discount the milestone if prior RNAi lipid programs have shown inconsistent magnitude or durability.
Background
YOSEMITE is a global Phase 3, randomized, double-blind, placebo-controlled study of zodasiran in HoFH patients on maximally tolerated lipid-lowering therapy.
Ticker impact
Arrowhead says it has completed enrollment in the global Phase 3 YOSEMITE trial of zodasiran, targeting HoFH, with completion expected mid-2027.
Likely modest positive bias for ARWR as the timeline de-risks, but the stock reaction may be limited until efficacy/safety data are reported.
The article is a primary clinical milestone (enrollment complete) with a stated next milestone (mid-2027 completion) but no new efficacy results or financial guidance.
Market effects
Supports the RNAi therapeutic platform narrative by showing continued progress in a late-stage, mechanism-differentiated lipid program.
No specific regional market impact beyond US-listed biotech sentiment.
Global Phase 3 enrollment completion highlights international patient recruitment momentum, relevant for eventual multi-geography regulatory strategy.
Counterpoint
Enrollment completion does not address whether zodasiran will meet the primary LDL-C endpoint; efficacy risk remains the dominant driver.
Key entities
- companyArrowhead Pharmaceuticals
NASDAQ-listed developer of RNAi therapeutics; sponsor of the YOSEMITE Phase 3 zodasiran trial.
- drug_candidatezodasiran
Investigational RNAi therapeutic targeting ANGPTL3 for HoFH treatment.
- clinical_trialYOSEMITE (NCT07037771)
Global Phase 3 trial evaluating efficacy and safety of zodasiran; primary endpoint is fasting LDL-C change at month 12.
