Arrowhead Pharmaceuticals (ARWR) H.C. Wainwright 28th Annual Global Investment Conference summary

Platform and pipeline overview

• Developed a broad RNAi platform targeting multiple cell types, with five represented in clinical candidates.

• Transitioned to a commercial stage with 21–22 drug candidates in clinical studies.

• Plans to access a new cell type every 18–24 months, expanding beyond liver to CNS and other tissues.

• Focused on building a broad-based company addressing diverse indications.

REDEMPLO (plozasiran) launch and expansion

• REDEMPLO approved for FCS, targeting both genetic and clinically defined FCS populations.

• Contracting with payers established; sales force expanded to address larger clinical FCS population.

• Preparing for SHTG launch in Q2 2027, with further sales force growth planned in early 2027.

SHASTA-3 and SHASTA-4 trial results

• Achieved ~80% reduction in triglycerides in severe hypertriglyceridemia patients.

• Statistically significant reduction in acute pancreatitis risk, with 78–100% risk reduction in high-risk subgroups.

• Clean safety profile: no significant liver, hypersensitivity, or thrombocytopenia issues; no increase in liver fat.