Arrowhead Pharmaceuticals (ARWR) investor relations material

Platform and pipeline overview

• Focused exclusively on siRNA therapeutics, leveraging the TRiM platform to advance 20 clinical candidates, with a mix of wholly owned and partnered assets, including collaborations with Amgen, Takeda, GSK, Sarepta, and Novartis.

• Wholly owned programs include REDEMPLO (approved for FCS, in phase III for SHTG), zodasiran (phase III for HoFH), and early-stage obesity and CNS programs such as ARO-ALK7, ARO-INHBE, and ARO-MAPT.

Clinical trial updates and expectations

• SHASTA-3 and SHASTA-4 pivotal data for plozasiran in SHTG expected in Q3, with last patient visits imminent and event rates tracking as anticipated.

• Confident in achieving statistical significance for acute pancreatitis endpoint, with pooled analysis planned and no changes to statistical analysis plan.

• Placebo group in triglyceride studies may show up to 10% decline due to dietary effects, but baseline values are robust at 860 mg/dL.

Safety and competitive landscape

• No hypersensitivity, thrombocytopenia, or significant liver fat increases observed with plozasiran; these are considered more ASO class effects.

• KOLs view minor liver fat increases as clinically insignificant if not accompanied by other biomarker changes.

• Positive payer feedback following price adjustment for REDEMPLO, with formulary decisions progressing smoothly.