Arrowhead Pharmaceuticals (ARWR) TD Cowen’s Genetic Medicines & RNA Summit summary

Cardiometabolic program updates

• Phase III PALISADE top-line data for plozasiran showed about 80% triglyceride reduction from baseline and 61–63% placebo-adjusted reduction, with statistical significance on all primary and key secondary endpoints.

• Nearly full suppression of APOC3 (90–95%) was achieved, and significant reduction in acute pancreatitis occurrence was observed.

• Additional data later this year will include more detailed efficacy, patient-reported outcomes, and risk threshold analyses for triglyceride levels.

• Ongoing investment in large phase III studies for sHTG and commercial build-out, with further updates expected at the upcoming R&D webinar.

• Decision on whether plozasiran or zodasiran will advance to cardiovascular outcome studies will be updated at the next R&D event, following extensive analysis and regulatory interactions.

Regulatory and patient population considerations

• No major differences are expected between genetically and clinically confirmed FCS populations regarding disease characteristics or placebo response.

• Labeling is anticipated to reflect the studied patient population, with ongoing discussions with regulators in the US and abroad.

Financing and business development

• Financing strategy remains focused on a mix of business development, equity, royalty-based, and potential debt financing, with over $1 billion raised in BD cash and $700 million in equity to date.

• Commercial revenue is expected to reduce reliance on external capital in the near future, with more specific guidance anticipated over the next year.

• Strengthening the balance sheet is a priority, with short- and mid-term steps planned.