Arrowhead Pharmaceuticals (ARWR) Morgan Stanley 24th Annual Global Healthcare Conference summary

Key clinical updates and pipeline progress

• ARO-DIMER-PA demonstrated first-in-class dual gene knockdown via RNAi, with strong reductions in LDL and triglycerides, and plans for additional dimers to enter the clinic in 2027.

• Early data showed ~50% ApoB reduction, outperforming some PCSK9 inhibitors, with multi-dose data expected at a future medical conference.

• Plans include a dual regulatory pathway: a pivotal program based on LDL reduction for faster market entry, followed by a cardiovascular outcomes trial.

• Quarterly dosing is likely, with potential for six-month intervals pending further data.

• Additional dimer programs are in development for obesity, MASH, and cardiovascular indications, with more details anticipated in 2027.

Commercialization and market strategy

• The plozasiran launch in FCS has been smooth, with faster-than-expected uptake and expanded sales force to compete with new entrants.

• Payer coverage is strong, and a priority review voucher was acquired to accelerate SHTG approval.

• SHASTA-3 and SHASTA-4 data showed 80% triglyceride reduction, significant AP event reduction, and a favorable safety profile.

• The therapy is differentiated by quarterly dosing, minimal monitoring, and superior triglyceride lowering compared to competitors.

• Net price is set at $45,000/year, justified by the drug’s impact on pancreatitis risk and healthcare cost savings.

Physician feedback and competitive landscape

• Physicians value the favorable safety profile and simplicity, reducing concerns about monitoring and dose adjustments.

• Strong risk reduction in pancreatitis was observed even in patients with moderate triglyceride elevations.

• Market entry is expected to be gradual, focusing on high-risk patients, with education needed to drive broader adoption.

• Competition is seen as beneficial for patient education and market growth.