Arrowhead Pharmaceuticals (ARWR) investor relations material

Commercial transition and product launch

• Preparing for first commercial launch with Plozasiran, targeting a November 18th PDUFA date and label negotiations underway.

• Full commercial team is hired and trained, with launch readiness for both drug and distribution channels.

• Plozasiran targets familial chylomicronemia syndrome (FCS) and potentially a broader clinically defined FCS population, with both groups responding similarly in trials.

• Market for FCS is ultra-orphan, estimated at 1,000 genetically defined patients in the U.S., with additional potential in clinically defined cases.

• Expansion into severe hypertriglyceridemia (SHTG) represents a much larger market opportunity, with multiple phase 3 studies ongoing.

Clinical development and pipeline progress

• Three phase 3 studies (Shasta-3, Shasta-4, NEER-3) for SHTG are fully enrolled, with data expected in Q3 next year.

• Shasta-5 study focuses on high-risk patients with extremely high triglycerides and history of pancreatitis, using a time-to-event design.

• Acute pancreatitis event rates are a key secondary endpoint, with data pooled from Shasta-3 and Shasta-4.

• Plozasiran has shown around 80% triglyceride reduction in FCS, outperforming competitor's 40% reduction; quarterly dosing offers differentiation.

• Zodasiran in phase 3 for HoFH, aiming for quarterly subcutaneous dosing and minimal incremental commercial costs.

Obesity and metabolic programs

• Two obesity assets (INHBE and ALK7) in clinical trials, targeting non-GLP-1 pathways for high-quality weight loss and muscle sparing.

• Initial data from INHBE expected by year-end, with ALK7 slightly behind; combination cohorts with tirzepatide are being studied.

• Proof of concept will focus on translation of animal data to humans, with potential for monotherapy or combination strategies.

• Additional obesity candidates and targets expected next year, including possible CNS-targeted therapies.