Arrowhead develops medicines that treat intractable diseases by silencing the genes that cause them using RNA interference (RNAi). The company's proprietary TRiM platform enables targeted delivery of RNAi therapeutics to multiple tissue types.
TRiM (Targeted RNAi Molecule)| Drug | Target | Phase | Lead Indication | Partner | Key Data | Next Catalyst |
|---|---|---|---|---|---|---|
Plozasiran (REDEMPLO) ARO-APOC3, REDEMPLO | APOC3 | Approved | Familial Chylomicronemia Syndrome (FCS) | Amgen | PALISADE: 80% TG reduction maintained at 1 year | sHTG approval decision |
ARO-APOC3 Plozasiran | APOC3 | Phase 3 | Severe Hypertriglyceridemia (sHTG) | Amgen | SHASTA-2: 77% TG reduction (vs placebo) | SHASTA-3/4 Phase 3 data |
Zodasiran ARO-ANG3 | ANGPTL3 | Phase 3 | Mixed Hyperlipidemia | Amgen | ARCHES-2: 60% LDL reduction, 73% TG reduction | ARCHES-2 pivotal data |
Fazirsiran ARO-AAT, TAK-999 | AAT (Alpha-1 Antitrypsin) | Phase 3 | Alpha-1 Antitrypsin Deficiency Liver Disease (AATD-LD) | Takeda | SEQUOIA Ph2: Sustained Z-AAT reduction; fibrosis improvement | Phase 3 data |
ARO-HSD | HSD17B13 | Phase 2 | MASH (Metabolic dysfunction-associated steatohepatitis) | Takeda | Phase 1: Well-tolerated, dose-dependent knockdown | Phase 2 interim data |
ARO-MMP7 | MMP7 | Phase 2 | Idiopathic Pulmonary Fibrosis (IPF) | - | Phase 1: MMP7 knockdown achieved; safety profile acceptable | Phase 2 data |
ARO-DUX4 | DUX4 | Phase 2 | Facioscapulohumeral Muscular Dystrophy (FSHD) | Sarepta | Phase 1: DUX4 silencing demonstrated in muscle | Phase 2 efficacy data |
ARO-C3 | Complement C3 | Phase 2 | IgA Nephropathy | - | Phase 1: Robust C3 reduction | Phase 2 data |
JNJ-3989 ARO-HBV | HBV (Hepatitis B Virus) | Phase 2 | Chronic Hepatitis B | Johnson & Johnson | Functional cure potential; HBsAg reduction | Phase 2b combination data |
ARO-INHBE | Inhibin beta E (INHBE) | Phase 1/2 | Obesity | - | Jan 2026: Weight loss demonstrated in diabetic obese patients; improved body composition | Additional cohort data |
ARO-ALK7 | ALK7 (ACVR1C) | Phase 1/2 | Obesity | - | Jan 2026: Weight loss in obese diabetic patients; muscle-sparing effect | Additional cohort data |
ARO-MAPT | MAPT (Tau) | Phase 1/2 | Alzheimer's Disease | - | CNS-targeted delivery using TRiM platform | Phase 1 safety/PK data |
ARO-DIMER-PA | PCSK9 + ANGPTL3 | Phase 1/2 | Mixed Hyperlipidemia | - | First dual-functional RNAi therapeutic | Phase 1 data |
ARO-RAGE | RAGE | Phase 1 | Diabetic Kidney Disease | - | Novel target for diabetic complications | - |
ARO-COV2 | SARS-CoV-2 | Phase 1 | COVID-19 | - | - | - |