Arrowhead Pharmaceuticals(US:ARWR)2026-01-12 16:32Arrowhead Pharmaceuticals FY Conference Summary Company Overview - Company Name: Arrowhead Pharmaceuticals - Ticker Symbol: ARWR - Market Capitalization: Approximately $9 billion with 136 million shares outstanding [2] - Cash and Investments: Approximately $920 million, excluding additional funds from Sarepta and Novartis [2] Core Business and Pipeline - Focus: Development of RNA interference (RNAi) therapies targeting various diseases [3] - First Commercial Launch: Rudemplo, approved in the US, Canada, and China for lowering triglycerides in patients with familial chylomicronemia syndrome (FCS) [3] - Pipeline: 20 clinical stage programs, including 11 wholly owned and 9 partnered candidates [3] - Expected Growth: Aim to add 2-3 additional drug candidates annually [3] Key Products and Indications Rudemplo - Indication: Treatment of FCS, with ongoing studies for severe hypertriglyceridemia (SHTG) [7] - Efficacy: Achieved approximately 80% reduction in triglyceride levels in clinical studies [9] - Market Potential: Targeting approximately 6,500 FCS patients in the US and a broader SHTG population of about 3 million [11][12] Zodasiran - Indication: Treatment of homozygous familial hypercholesterolemia (HoFH) [12] - Efficacy: Demonstrated a 41% reduction in LDL cholesterol in HoFH patients [13] - Market Potential: Estimated 1,000 to 2,000 patients in the US with HoFH [13] ARO-Dimer-PA - Indication: Designed to treat mixed hyperlipidemia by silencing both ApoC3 and PCSK9 [15] - Market Potential: Approximately 20 million patients in the US with elevated LDL cholesterol and triglycerides [16] Obesity Candidates (ARO-INHBE and ARO-ALK7) - Focus: Targeting specific pathways to reduce visceral fat and improve metabolic outcomes [18] - Efficacy: ARO-INHBE showed a 9.9% reduction in visceral fat after 16 weeks [20] - Combination Therapy: ARO-INHBE combined with tirzepatide resulted in a 9.4% weight loss compared to 4.8% with tirzepatide alone [20] CNS Program (ARO-MAPT) - Indication: Targeting tau protein for Alzheimer's and tauopathies [25] - Efficacy: Early data shows potential for significant tau reduction in animal models [26] - Market Potential: Addressing a significant unmet need in neurodegenerative diseases [25] Financial Strategy and Market Position - Pricing Strategy: Rudemplo priced at $60,000 for both FCS and SHTG populations to ensure payer acceptance and long-term market access [36] - Cash Management: Strong financial position with over $900 million available to fund ongoing and future trials [47] - Regulatory Pathways: Potential for expedited approval based on LDL reduction for ARO-Dimer-PA [45] Upcoming Milestones - Phase 3 Studies: Expected readouts for SHTG in Q3 2026 and filing for SNDA in Q4 2026 [28] - Obesity Pipeline Expansion: Continued development of ARO-INHBE and ARO-ALK7 with additional data expected throughout 2026 [29] - CNS Pipeline: Initial data for ARO-MAPT anticipated in late Q3 2026 [29] Conclusion Arrowhead Pharmaceuticals is positioned to capitalize on significant market opportunities in the RNAi space, with a robust pipeline targeting high unmet medical needs across various therapeutic areas. The company’s strategic focus on pricing, cash management, and regulatory pathways aims to enhance its market presence and drive future growth.