Arrowhead Pharmaceuticals (ARWR) Jefferies Global Healthcare Conference summary

Platform and pipeline overview

• Proprietary TRiM platform enables targeted RNAi delivery to multiple cell types, supporting a diverse pipeline of 14 programs across cardiometabolic, pulmonary, muscle, and CNS indications.

• Ten programs are wholly owned, while four are partnered, with a range of stages from early clinical to late-stage development.

• Three to four programs are in phase 3, with the first phase 3 readout for plozasiran showing success on primary and key secondary endpoints.

Cardiometabolic program progress

• Plozasiran phase 3 data showed over 90% APOC3 reduction and 80% triglyceride reduction, with no significant difference between genetically and clinically confirmed FCS patients.

• Statistically significant reduction in acute pancreatitis events was achieved, a key motivator for treatment and a hoped-for endpoint.

• Market opportunity spans rare FCS, phenotypic FCS, severe hypertriglyceridemia (SHTG), and potentially ASCVD, with SHTG alone representing a multibillion-dollar opportunity.

• Pricing strategy will reflect orphan disease value, but will be influenced by competitor launches and payer negotiations.

Clinical development and regulatory strategy

• SHASTA-3, SHASTA-4, and SHASTA-9 studies are enrolling, aiming for completion in 2026 and potential approvals by 2028.

• SHASTA-5 pancreatitis study is more relevant for payers than regulators, especially in Europe, and will be included in filings if completed in time.

• Cardiovascular outcome study planning is underway, with regulatory interactions ongoing and capital allocation considerations influencing timing and design.