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

Key program updates and clinical data

• ARO-DIMER-PA demonstrated first-in-class dual gene knockdown using RNAi, with plans for additional dimers in the clinic by 2027.

• Early data showed strong LDL and triglyceride reductions, with ApoB lowered by about 50%, surpassing some PCSK9 inhibitors.

• Multi-dose data for ARO-DIMER-PA expected at a future medical conference; quarterly dosing appears feasible.

• Next steps include amending the current study for multi-dose safety and efficacy before moving to pivotal phase III.

• Additional dimers targeting obesity, MASH, and cardiovascular indications are in development, with more details anticipated in 2027.

Commercialization and market strategy

• Smooth launch for plozasiran in FCS, with faster-than-expected uptake and expanded sales force.

• Priority review voucher acquired to accelerate SHTG approval; payer coverage progressing well.

• SHASTA-3 and SHASTA-4 studies showed 80% triglyceride reduction, significant AP event reduction, and strong safety profile.

• Product differentiation centers on superior safety, simplicity (quarterly dosing), and stronger triglyceride reduction compared to competitors.

• Net price set at $45,000/year, justified by pancreatitis risk reduction and cost savings.

Pipeline and innovation

• ARO-MAPT targets tau protein for Alzheimer's and other tauopathies, using subcutaneous delivery for better brain distribution.

• Proof-of-concept data for ARO-MAPT expected soon; positive results could accelerate CNS pipeline expansion.

• Obesity pipeline includes ARO-INHBE (MASH-focused) and ARO-ALK7 (potential pure weight loss), with phase II studies underway.

• Company leverages AI for target discovery, ligand design, and sequence selection, supported by a large proprietary dataset.

• Maintains multi-year head start in RNAi innovation, monitoring global competition and emphasizing rapid development.