Arrowhead Pharmaceuticals (ARWR) investor relations material

Recent progress and regulatory milestones

• Secured four additional positive regulatory actions for lead product, including approvals in the U.S., China, Australia, and Canada, with a positive CHMP opinion in Europe and expected EC approval soon.

• Completed first full quarter of commercial sales for the lead product, showing strong market dynamics and growth potential.

• Strengthened financial position with approximately $1.8 billion in cash, supporting ambitious development and commercialization plans.

• Executed well on the first commercial product and positioned to become a leading biotech company.

Clinical development updates and trial timelines

• SHASTA-3 and SHASTA-4 phase III studies expected to complete in late June, with data readout anticipated in Q3; these are key catalysts for future growth.

• Modified adjudication criteria for acute pancreatitis (AP) events to align with regulatory and industry standards, increasing event capture and statistical power.

• SHASTA-5 trial, focused on high-risk patients and AP as the primary endpoint, is about one-third enrolled, with data expected in late 2027 or early 2028.

• Enrollment criteria for SHASTA-5 were broadened to accelerate recruitment, reflecting evolving understanding of background event rates.

Commercial outlook and market potential

• Peak revenue opportunity for the lead drug estimated at $3–$4 billion, with potential for the APOC3 class to reach $9–$10 billion annually.

• Early commercial launch in FCS has been successful, driven by strong efficacy in reducing APOC3 and triglycerides.

• Differentiated pricing strategy with a $45,000 WAC compared to competitor's $40,000.

• Payer and physician enthusiasm is high, with broad recognition of the value of this therapeutic class.