ARROWHEAD PHARMACEUTICALS, INC. Q3 FY2025 earnings call

• Addressed partnership with Sarepta, stating Sarepta's strategic restructuring prioritizes licensed programs from Arrowhead, and collaboration continues as expected. • Progress in development: plozasiran has productive interactions with regulators, full enrollment in Phase III studies for SHTG, YOSEMITE Phase III study for zodasiran initiated. Other late-stage candidates like fazirsiran and olpasiran also highlighted. • Commercial build-out: team nearly assembled for plozasiran launch. • Visirna deal: Sanofi acquiring rights to plozasiran in Greater China, Visirna gets upfront and milestone payments, Arrowhead to own ~56% of Visirna. • Sarepta milestones: Reaching enrollment targets in ARO-DM1 study triggers $100M and potential $200M payments. • Pipeline progress: obesity candidates ARO-INHBE and ARO-ALK7 in Phase I/II, first RNAi dimer to enter clinic, CNS franchise with ARO-MAPT filing CTA expected.

No detailed segment performance with revenue contribution percentages provided in the transcript. The focus is on various drug candidates and partnerships.

• Visirna deal will result in $130M upfront payment recognized in Q4. • Expect to trigger $200M Sarepta milestone at year-end. • Plozasiran Phase III studies for SHTG on track for completion mid-2026. • YOSEMITE Phase III study for zodasiran could support regulatory filings as early as 2028/2029.

• Risk that Sarepta fails to meet obligations under the agreement, though termination provisions exist. • Regulatory uncertainties related to approvals and study outcomes. • Broader biotech market sentiment impacts on stock price.

Q: When thinking about SHTG pivotals with ongoing SHASTA-3 and 4, how are prospects of competitor Ionis' programs reading out in September considered?

A: Chris Anzalone and Bruce Given state they focus on their own studies, have good data in Phase II and III, and will follow Ionis' data but can't compare directly.

Q: On SHTG program, thoughts on baseline demographics similar to Ionis' published data and event rate?

A: Bruce Given says it's hard to compare across studies, but patient populations are similar, and they'll follow Ionis' data release in September.

Q: On SHTG program, payer access for genetically confirmed and clinically defined FCS, differentiation in coverage path and pricing expectations?

A: Chris Anzalone says not prepared to opine on SHTG pricing, expects it to be lower than FCS.

Q: On Sarepta situation with their Arrowhead shares and commercial activities for SHTG education?

A: Chris Anzalone mentions Sarepta's lockup period, and Andy Davis discusses medical education via liaisons and focus on disease burden and goal attainment.

Q: On HoFH addressable patient population for zodasiran?

A: Bruce Given states prevalence of HoFH is ~1 in 500,000 to 1 in 1 million, and accessible population is similar to estimates.

Q: On Visirna deal cash and R&D from Sarepta?

A: Chris Anzalone explains cash goes to Visirna, portion distributed to shareholders, and Sarepta pays $50M annually for 5 years.

Q: On initial presentation and auto-injector for plozasiran?

A: Andy Davis says initial is prefilled syringe, Bruce Given states auto-injector for SHTG will be available at launch or soon after.

Q: On INHBE readout for obesity program?

A: Chris Anzalone says data from all cohorts (SAD/MAD, combination) will be shared, measuring biomarkers like PD, body composition, lipids.

Q: On obesity program sample size and dosing frequency?

A: Chris Anzalone states Phase I is hypothesis generating, looking at quarterly dosing frequency.

Q: On MUIR-3 study patient adherence and trial readouts?

A: Bruce Given says they pay attention to trial execution, adherence is good, and trials will read out close together but not on same day.

Q: On DM1 program max dose?

A: Chris Anzalone states max dose in DM1 study is still up to 12 mg/kg.

Q: On obesity program safety and dosing frequency confidence?

A: Chris Anzalone mentions loss of function data in ALK7 carriers, ligand-driven approach for adipocyte delivery, and safety from non-GLP and GLP-tox studies, with quarterly dosing considered favorable.