ARROWHEAD PHARMACEUTICALS, INC. Q1 FY2025 earnings call

• Sarepta deal: Arrowhead received $500M upfront, $325M from stock purchase, $250M in annual installments, and potential $300M near-term payments. Total potential value exceeds $11B. Sarepta takes clinical development responsibilities, reducing R&D expenses and providing downstream capital. • Plozasiran: First commercial launch pending FDA review/approval late 2024. Phase III studies SHASTA-3, 4, 5 on track for enrollment completion in 2024, study completion in 2026, and sNDA filing. Potential $2B-$3B per year drug in SHTG market. U.S. FDA accepted NDA for plozasiran for FCS with PDUFA date Nov 18, 2025. • Obesity programs: ARO-IoN-HPE and ARO-ALK7 early-stage programs with near/midterm data readouts. Phase I/II trial of ARO-INHBE initiated, ARO-ALK7 received regulatory clearance in NZ. • CNS pipeline: Emerging CNS pipeline with TRiM platform, preclinical data in nonhuman primates, aiming for IND/CTA filings in 2024-2026. • Other programs: Potential partnering opportunities for noncore programs like ARO PNPLA3, pulmonary platform, ARO-RAGE, ARO-C3, ARO-CFB.

Revenue for the quarter ended December 31, 2024, was $2.5 million compared to $3.6 million for the same period in 2023. Revenue in 2024 related to collaboration agreements with GSK, while 2023 revenue included recognition from license and collaboration agreements with Takeda and GSK. Cash and investments totaled $552.9 million at December 31, 2024, and pro forma cash and investments with Sarepta agreements would be $1.4 billion. Expected cash and investments balance at end of 2025 is approximately $1 billion with runway into 2028.

• 2025 catalyst calendar: Initiate SHASTA-5, fully enroll SHASTA-3, 4, MUIR-3, make global regulatory submissions, commercial launch of plozasiran in FCS, initiate Phase III HoFH study, initiate dosing in ARO-ALK7 trial, potential first data for ARO-INHBE, full enrollment of REDWOOD study, initial data in Sarepta Phase I/II studies, data readouts for complement programs, and filing CTAs for CNS pipeline.

• Uncertainty in clinical trial outcomes for obesity and CNS programs. • Delays in regulatory approvals for plozasiran and other candidates. • Competition in the cardiometabolic and CNS drug development spaces. • Dependence on milestone payments and royalties from partnerships for financial stability.

Q: How thinking about zodasiran in HoFH and heterozygous FH.

A: No regulatory path for heterozygous FH, focusing on HoFH as current program.