Arrowhead Pharmaceuticals Inc.

ARWR

Held by 9 specialist biotech funds

High Convergence

9 funds opened new positions. Next phase3 readout (Plozasiran Injection): May 2026. Short interest: 11.6% of float.

BiotechnologyCEO: Christopher R. AnzaloneIPO: 1993Website ↗

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes ARO-AAT, a RNA interference (RNAi) therapeutic candidate that is in Phase II clinical trial for the treatment of liver diseases associated with alpha-1 antitrypsin deficiency; ARO-APOC3, which is in phase 2b and one phase 3 clinical trial to treat hypertriglyceridemia; ARO-ANG3 that is in Phase 2b clinical trial to reduce production of angiopoietin-like protein 3; ARO-HSD, which is in Phase 1/2a clinical trial to treat liver diseases; ARO-ENaC, which is in a Phase 1/2a clinical trial to reduce production of the epithelial sodium channel alpha subunit in the airways of the lung; ARO-C3 for the treatment of complement-mediated disease that is in Phase 1/2a clinical trial; ARO-Lung2 for the treatment of chronic obstructive pulmonary disorder; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-XDH to treat uncontrolled gout; ARO-COV for the treatment of COVID-19 and other pulmonary-borne pathogens; and ARO-HIF2, which is in phase 1b clinical trial to treat clear cell renal cell carcinoma. It is also involved in the development of JNJ-3989, a subcutaneously administered RNAi therapeutic candidate to treat chronic hepatitis B virus infection; Olpasiran to reduce the production of apolipoprotein A; and ARO-AMG1 for treating genetically validated cardiovascular targets. Arrowhead Pharmaceuticals, Inc. has a license and research collaboration agreement with Janssen Pharmaceuticals, Inc. to develop ARO-JNJ1, ARO-JNJ2, and ARO-JNJ3 RNAi therapeutics for liver-expressed targets; and license and research collaboration agreement with Takeda Pharmaceuticals U.S.A., Inc. to develop RNAi therapeutic candidate as a treatment for liver disease. Arrowhead Pharmaceuticals, Inc. was incorporated in 1989 and is headquartered in Pasadena, California.

Catalysts

Phase 2Q4 2015ARC-520 InjectionChronic Hepatitis BNCT02349126

Primary: Efficacy: To evaluate the depth and duration of HBsAg decline in response to a single dose of ARC 520 in combination with entecavir or tenofovir in patients with chronic HBV infection (Through Day 85 post-dosing)0Sites: 1NA, TREATMENT

Phase 2Q4 2016ARC-AAT InjectionAlpha-1 Antitrypsin DeficiencyNCT02900183

Primary: Incidence and frequency of adverse events as a measure of safety and tolerability of multiple doses of ARC-AAT Injection (Baseline through Day 287 End-of Study Visit)0Sites: 4NA, TREATMENT

Phase 2NegativeQ4 2016ARC-520Hepatitis BNCT02604212

Primary: Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) at Day 113 (Baseline, Day 113)Enrollment:32/32Sites: 16RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2NegativeQ4 2016ARC-520 InjectionHepatitis BNCT02738008

Primary: Percentage of Initial Responders to ARC-520 Therapy Achieving a 1-log Reduction in HBsAg at Week 36 Compared to Baseline (Baseline, Week 36)Enrollment:12/12Sites: 6NA, TREATMENT

Phase 2NegativeQ4 2016ARC-520Hepatitis B, Hepatitis DNCT02577029

Primary: Percentage of Participants Achieving a 1-log Reduction in Hepatitis B Surface Antigen (HBsAg) at Week 60 Compared to Baseline (Baseline, Week 60)Enrollment:79/79Sites: 33NON_RANDOMIZED, TREATMENT

Phase 2NegativeQ1 2017ARC-520Hepatitis B, ChronicNCT02065336

Primary: Change From Baseline Over Time in Quantitative Hepatitis B Surface Antigen (HBsAG) (Baseline, through Day 85 (Cohorts 1-7) and through 24 weeks post-last dose (last dose: Day 85 Cohort 9; Day 253 Cohort 10))Enrollment:58/58Sites: 2RANDOMIZED, TRIPLE, TREATMENT

Phase 2NegativeQ1 2017ARC-520 InjectionHepatitis BNCT02604199

Primary: Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) at Day 113 (Baseline, Day 113)Enrollment:58/58Sites: 16RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q2 2020ARO-HBVHepatitis BNCT03365947

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) Possibly or Probably Related to Treatment (NHV participants: up to Day 29 (± 2 days); CHB participants: Day 113 (± 2 days))Enrollment:114/114Sites: 7RANDOMIZED, DOUBLE, TREATMENT

Phase 2NegativeQ2 2021ARO-ENaCCystic Fibrosis, PulmonaryNCT04375514

Phase 2Q3 2021ARO-HSD InjectionNon-alcoholic SteatohepatitisNCT04202354

Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Possibly or Probably Related to Treatment (From first dose of study drug through Day 113 (±5 days))Enrollment:50/50Sites: 1RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q4 2021Fazisiran InjectionAlpha 1-Antitrypsin DeficiencyNCT03945292

Primary: Percent Change From Baseline in Serum Z-Alpha-1 Antitrypsin (Z-AAT) at Week 16 (Baseline, Week 16 (+/- 2 weeks))Enrollment:40/40Sites: 21RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2PositiveQ2 2022ARO-AATAlpha 1-Antitrypsin DeficiencyNCT03946449

Phase 2Q3 2022ARO-ANG3Mixed DyslipidemiaNCT04832971

Phase 2PositiveQ1 2023ARO-APOC3Mixed DyslipidemiaNCT04998201

Phase 2PositiveQ1 2023ARO-APOC3Severe HypertriglyceridemiaNCT04720534

Primary: Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG) (Baseline, Week 24)Enrollment:229/229Sites: 74RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q2 2023ARO-ANG 3 InjectionHomozygous Familial HypercholesterolemiaNCT05217667

Primary: Percent Change from Baseline in Fasting Calculated Low-Density Lipoprotein-Cholesterol (LDL-C) and LDL-C by Preparative Ultracentrifugation (LDL-C [PUC]) up to Week 24 (Baseline, up to Week 24)Enrollment:18/18Sites: 7RANDOMIZED, TREATMENT

Phase 3ApprovedQ2 2024PlozasiranFamilial ChylomicronemiaNCT05089084

Phase 3Q3 2024Tranexamic AcidHemorrhagic Shock, Trauma InjuryNCT03469947

Phase 2NegativeQ4 2024ARO-MUC5ACAsthma, Chronic Obstructive Pulmonary DiseaseNCT05292950

Phase 2Q2 2025ARO-RAGEAsthmaNCT05276570

Phase 2Q2 2025ARO-C3C3 Glomerulopathy, Ig A NephropathyNCT05083364

Phase 3Q2 2025Calcium carbonateUterine Contraction, Labor Complication, Labor Active Dilated CmNCT07056062

Phase 2Q3 2025ARO-MMP7 Inhalation SolutionIdiopathic Pulmonary FibrosisNCT05537025

Phase 2Q3 2025ARO-APOC3DyslipidemiasNCT05413135

Phase 3Q2 2026Plozasiran InjectionHypertriglyceridemiaNCT06347133

Phase 3Q2 2026Plozasiran InjectionSevere HypertriglyceridemiaNCT06347016

Phase 2Q2 2026ARO-DIMERPAHyperlipidemia; MixedNCT07223658

Phase 3Q2 2026Plozasiran InjectionSevere HypertriglyceridemiaNCT06347003

Phase 2Q4 2026ARO-ALK7Obesity, Diabetes Mellitus, Type 2NCT06937203

Phase 2Q4 2026ARO-DM1 Intravenous (IV) InfusionMyotonic Dystrophy 1NCT06138743

Phase 2Q4 2026ARO-DUX4 for InjectionFacio-Scapulo-Humeral DystrophyNCT06131983

Phase 3Jun 30, 2027StatinsIntracerebral HemorrhageNCT03936361

Phase 2Q2 2027ARO-MAPT-SCAlzheimer Disease, Alzheimer Disease, Early OnsetNCT07221344

Phase 3Q3 2027zodasiran InjectionHomozygous Familial HypercholesterolemiaNCT07037771

Phase 2Q3 2027ARO-INHBEObesityNCT06700538

Phase 2Q4 2027ARO-RAGEAsthmaNCT07241546

Phase 3Q3 2028ZodasiranHomozygous Familial HypercholesterolemiaNCT07473843

Phase 3Q3 2028Plozasiran InjectionHypertriglyceridemiaNCT06822790

Phase 3Q1 2029PlozasiranSevere HypertriglyceridemiaNCT06880770

Arrowhead Pharmaceuticals Inc.

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