Strong REDEMPLO Launch Momentum
FDA-approved REDEMPLO launched in Nov 2025 and continues to accelerate: ~30 new prescriptions/week with >400 prescriptions written since launch; prescriptions grew nearly threefold from the start to the end of the fiscal quarter and >40% growth over the most recent 4-week period. Patient mix: ~85% APOC3-class naive; >10% of prescriptions are switches from a competitor.
Clinical Efficacy Signals for Plozasiran
2-year open-label extension data: median triglyceride reductions of 83% in SHTG (SHASTA-2) and 67% in HTG (MUIR); 96% of SHTG patients achieved TG <500 mg/dL, 63% achieved TG <150 mg/dL, and 93% of HTG patients achieved TG <150 mg/dL. No adjudicated acute pancreatitis events observed in patients receiving plozasiran during the 2-year OLE.
Multiple Near-Term Clinical Catalysts
Several important readouts expected in 2H 2026: SHASTA-3 and -4 top-line Phase III plozasiran data (Q3) to support an sNDA for SHTG; first clinical readout of ARO-DIMER-PA (dual siRNA) in Q3; initial ARO-MAPT CNS platform data end of Q3/early Q4; ongoing updates for ARO-INHBE and ARO-ALK7 across H2 2026.
Broad and Innovative Pipeline Expansion
Pipeline exceeds 20 clinical programs across liver, muscle, adipose, CNS and lung, including the first-in-human dual functional siRNA (ARO-DIMER-PA) and a proprietary CNS delivery candidate (ARO-MAPT) designed for subcutaneous delivery across the BBB; expectation of additional dual-dimer candidates in 2027.
Global Regulatory Progress
REDEMPLO approvals in Australia, China and Canada and CHMP positive opinion recommending EU approval; anticipated launches later in 2026 in Canada, select EU countries and the U.K.; Greater China commercialization via Sanofi.
Material Balance Sheet Strengthening
Completed largest fundraising in company history: concurrent public offerings of $700M 0% convertible senior notes and $230M common stock (both several times oversubscribed). Cash and investments nearly $1.8B as of March 31, 2026; brought in over $1B in the quarter including ~$850M net from financing and milestone/payments from partners.
Strategic Business Development
Exclusive license agreement with Madrigal for ARO-PNPLA3: $25M upfront, up to $975M in development/regulatory/sales milestones, plus tiered royalties up to mid-teens; additional ongoing collaborations with Sarepta and Novartis contributing revenue recognition.
Early Commercial and Payer Dynamics Positive
Updated WAC to $45,000/year aimed at simplifying pricing across indications; company reports generally positive payer interactions and emerging coverage policies that accept both genetic and clinical diagnostic pathways for FCS.