First Regulatory Approval and Commercial Launch of REDEMPLO
FDA approval on Nov 18, 2025 for REDEMPLO in adults with familial chylomicronemia syndrome (FCS); launched independently in the U.S.; product available in channel within days of approval. Over 100 prescriptions received in ~10 weeks of launch across a geographically balanced prescriber base; self-administered 25 mg every 3 months (4 injections/year).
Clinical Efficacy from PALISADE (REDEMPLO)
Phase III PALISADE demonstrated deep and durable TG reductions with a median ~80% reduction from baseline; reductions largely maintained below guideline threshold of 500 mg/dL through 12 months and a lower numerical incidence of adjudicated acute pancreatitis versus placebo.
Multiple International Approvals and Partnerships
REDEMPLO approvals obtained in the U.S., Health Canada and China (NMPA); Arrowhead to market in U.S. and Canada; Sanofi to market in Greater China; potential EU/U.K. launches pending review.
Material Balance Sheet Strengthening — $1.33B Transactions
Completed transactions with gross proceeds of $1.33 billion including $200M Novartis upfront, $200M Sarepta milestone and concurrent public offerings (convertible notes $700M and common stock $230M); convertible notes carry 0% coupon and ~35% initial conversion premium.
Q1 Financial Turnaround — Net Income and Revenue
Net income of $30.8M ($0.22/share) for the quarter ended Dec 31, 2025 vs. net loss of $173.1M prior-year quarter (swing of ~$203.9M). Quarterly revenue of $264M driven primarily by Sarepta and Novartis collaborations (≈$229M Sarepta, $34M Novartis) and first commercial sale of plozasiran.
Robust Cash and Funding Position
Reported cash and investments of $917M as of Dec 31, 2025 (does not include subsequent $200M Sarepta milestone received in January and $50M anniversary payment expected), plus proceeds from early January financings — providing runway to support development and commercialization.
Encouraging Early Obesity and RNAi Delivery Data
ARO-INHBE + tirzepatide produced ~2x greater weight loss at week 16 vs tirzepatide alone in obese T2D patients and ~3x reductions in total, visceral and liver fat (MRI week 12). ARO-ALK7 showed dose-dependent adipose ALK7 mRNA knockdown (mean −88% at 200 mg at week 8, max −94%).
Pipeline Progress — Multiple Clinical Milestones Expected in 2026
Phase III plozasiran (SHASTA-3/4, MUIR-3) blinded portion on schedule to complete mid-2026 with topline data in Q3 2026 and planned sNDA submission for SHTG before year-end. ARO-DIMER-PA first patients dosed (interim data expected H2 2026). ARO-MAPT (CNS TRiM delivery) dosed first subjects with healthy volunteer interim data expected in 2026.
Targeted Enrollment and High-Risk Focus in SHASTA Studies
SHASTA-3/4 enrollment includes substantial high-risk population: 37% of enrolled patients reported TGs >880 mg/dL and 20% had prior history of pancreatitis, supporting relevance for pancreatitis risk assessment and regulatory endpoints.