Arrowhead Pharmaceuticals (ARWR) TD Cowen 46th Annual Health Care Conference summary

Strategic focus and pipeline overview

• Emphasis on cardiometabolic diseases, including obesity, with a growing CNS platform targeting tauopathies like Alzheimer's via subcutaneous injection.

• Over 21 drug candidates in clinical studies, with key readouts expected in 2026, including phase III data for plozasiran (SHASTA-3/4) and first clinical data for ARO-MAPT in CNS.

• Additional data from obesity programs ARO-INHBE and ARO-ALK7 anticipated late in the year, with expanded cohorts and focus on adipose-directed RNAi.

• Productive discovery engine continues to advance new candidates into the clinic.

Commercialization and product differentiation

• Waylivra launched for FCS, achieving 100 prescriptions in the first 10 weeks, with strong interest from both naive and switch patients.

• Demonstrated ~80% triglyceride reduction in FCS, outperforming competitor olezarsen, with a favorable safety profile and quarterly dosing.

• Pricing set at $60,000/year, justified by value in high-risk populations and cost savings from reduced acute pancreatitis events.

• Strategy targets high-risk SHTG patients rather than broader populations, aiming for premium pricing and payer education.

Clinical development and upcoming milestones

• SHASTA-3/4 phase III data for plozasiran in SHTG expected in Q3, aiming for 70%+ triglyceride reduction and reduction in acute pancreatitis rates.

• SHASTA-5 event-driven study serves as backup for pancreatitis outcomes.

• SNDA filing for SHTG planned post-data, with ex-U.S. registration under consideration.

• Monitoring for liver fat changes as a potential differentiator from ASO competitors.