Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.
Phase 2 trials begin testing efficacy in a larger patient group (100–300). This is often where biotech binary events occur — positive Phase 2 data can significantly advance a company's pipeline narrative, while failures can be terminal for a program.
This trial was terminated before completion. Termination can indicate safety signals, futility findings, or business/regulatory decisions by the sponsor.
Arrowhead Pharmaceuticals (ARWR) is the sponsoring company for this trial. BiotechSign currently grades this company C (53/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.