Arrowhead Pharmaceuticals (ARWR) Conference Transcript

Arrowhead Pharmaceuticals Conference Summary Company Overview - Company: Arrowhead Pharmaceuticals (ARWR) - Industry: RNA interference (RNAi) therapeutics - Current Status: Transitioning from R&D to commercial operations with 20 drug candidates in clinical studies or at market by the end of 2025 [3][70] Key Drug Candidate: Posaziran - Indication: Designed to reduce expression of APOC3 for treating familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG) - PDUFA Date: November 18, 2025, for FCS population [3][4] - Clinical Data: - Phase III study showed an 80% reduction in triglycerides and an 83% improvement in risk of acute pancreatitis [5][6] - 75% of patients achieved triglyceride levels below 880 mg/dL, and 50% below 500 mg/dL [11][12] - Dosing: Administered quarterly via subcutaneous injection [5] Market Potential and Competitive Landscape - Patient Population: - Genetic FCS patients estimated at 1,000 in the U.S. - Phenotypic FCS patients could number 5,000 to 10,000 [13][15] - Comparison with Competitors: - Posaziran shows superior efficacy compared to a competitor's drug, which demonstrated a 40% reduction in triglycerides [23][24] - Posaziran's quarterly dosing is more favorable compared to competitor's monthly dosing [23] Pricing and Payer Considerations - Pricing Strategy: - Positioned as a pancreatitis drug rather than a cardiovascular drug, potentially allowing for higher pricing [28][29] - Discussions ongoing with payers regarding the value proposition of reducing pancreatitis risk [32][33] - Market Size: - Broad market estimated at 3 to 4 million patients with triglycerides above 500 mg/dL, with a narrower focus on those above 880 mg/dL [48] Future Developments - Expansion Plans: - Plans to file a supplemental NDA (sNDA) for broader SHTG population by the end of 2026 [43] - Development of a dimer approach targeting both PCSK9 and APOC3 for broader cardiovascular applications [50][51] - Other Drug Candidates: - Zodasiran, targeting ANGPTL3, is in Phase III for HoFH (homozygous familial hypercholesterolemia) [55] - MAPT for Alzheimer's disease expected to enter clinical trials soon [58][71] Partnership and Collaborations - Novartis Partnership: - Collaboration focused on CNS targets, including alpha-synuclein for neurodegenerative diseases [57][62] - Upfront payment of $200 million with potential milestones exceeding $2 billion [63] Conclusion - Arrowhead Pharmaceuticals is poised for significant growth with its lead candidate, posaziran, targeting severe hypertriglyceridemia and pancreatitis. The company is strategically positioning itself in the market while preparing for a transition to commercial operations, with multiple drug candidates in the pipeline and ongoing partnerships to enhance its therapeutic offerings [70][76]