Arrowhead Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in the development of medicines that treat intractable diseases by silencing the genes that cause them. The firms preclinical stage drug candidates include ARO-ANG3, ARO-AAT, ARO-APOC3, ARO-HIF2, ARO-HSD, ARO-Lung2, ARO-COV, and ARO-ENaC. The company was founded by Robert Bruce Stewart in 1989 and is headquartered in Pasadena, CA.
Clinical Trials
78
31 active
Approvals
2
Total approvals
Agencies
2
Regulatory bodies
Founded
1989
Active, not recruiting
30
38.5%
Available
1
1.3%
Completed
21
26.9%
Not yet recruiting
1
1.3%
Recruiting
11
14.1%
Terminated
11
14.1%
Withdrawn
3
3.9%
- The FDA approved Curium's Bexlutry, the first generic radioligand therapy, a copycat of Novartis' Lutathera that delivers radioactive lutetium to SSTR-positive neuroendocrine tumors. - The approval covers foregut, midgut and hindgut SSTR-positive neuroendocrine cancers and followed Curium's victory in a patent dispute with Novartis. - Arrowhead's dual-acting RNA interference therapy Aro-Dimer-Pa cut PCSK9 and APOC3 protein levels by 72% and 88% after a single dose in an early-stage trial. - Corbus reported CRB-913 produced up to 5 percentage points more weight loss than placebo at 12 weeks, but psychiatric side effects drove treatment discontinuations.
- Michael S. Perry, a Director at Arrowhead Pharmaceuticals, sold 11,600 shares of common stock on Aug. 18, 2026, according to an SEC Form 4 filing. - The transaction was valued at approximately $1 million, with shares disposed of at prices ranging from $87.20 to $87.44. - The sale liquidated 9% of Perry's direct holdings, leaving him with 111,459 directly held shares and 0.0791% of the company's equity. - Arrowhead Pharmaceuticals reported trailing twelve-month revenue of $669.5 million and a net loss of $320 million as it advances its RNA interference therapeutic pipeline.
- The ANGPTL3 inhibitors market was valued at USD 0.21 billion in 2025 and is projected to reach USD 3.48 billion by 2035, at a 32.4% CAGR. - Monoclonal antibodies led the market with 71% share in 2025, driven by Regeneron's EVKEEZA, the sole FDA-approved ANGPTL3 inhibitor for homozygous familial hypercholesterolemia. - Arrowhead's zodasiran is advancing into Phase III development following positive Phase IIb ARCHES-2 results, positioning RNAi therapeutics as the fastest-growing drug class. - Verve Therapeutics' VERV-201, an in vivo base-editing therapy, is advancing toward potentially one-time, permanent ANGPTL3 reduction for refractory hypercholesterolemia and HoFH.
- RNA interference (RNAi) therapies using small interfering RNA (siRNA) are already approved by the FDA, with at least seven treatments available for genetic diseases and more in clinical trials. - The global RNAi therapeutics market is projected to grow from about $1.5 billion in 2024 to roughly $5.1 billion in 2034, reflecting rapid commercial expansion. - Delivery beyond the liver is a key frontier, with researchers developing siRNAs targeting muscles, nerves, the placenta, and other organs. - Korean firms OliX and rznomics signed technology transfer deals with Eli Lilly worth 900 billion won and 1.9 trillion won, respectively, underscoring global interest in RNA-based platforms.
- The novel hypercholesterolemia drugs market is projected to grow from USD 17.6 billion in 2026 to USD 42.8 billion by 2036, a 9.3% CAGR. - PCSK9 inhibitors are forecast to hold a 42.0% drug-class share in 2026, spanning monoclonal antibodies, inclisiran-based siRNA, and the new oral enlicitide. - Merck's Lipfendra (enlicitide) gained FDA approval in July 2026 as the first once-daily oral PCSK9 inhibitor for adults with hypercholesterolemia. - Injectable therapies lead with a 57.0% route-of-administration share, while gene-editing programs from Verve, CRISPR Therapeutics, and Eli Lilly advance toward one-time treatment.
- Two recent Nature portfolio publications highlight the growing role of machine learning and deep learning in predicting drug–target binding affinities and drug–drug interactions, addressing critical challenges in pharmaceutical R&D. - Sequence-based computational models are demonstrating improved accuracy in forecasting how drug compounds interact with biological targets, potentially accelerating early-stage drug discovery. - Deep learning frameworks for drug–drug interaction prediction offer new tools to identify adverse combination effects before clinical exposure, enhancing patient safety. - These computational approaches represent a shift toward data-driven, predictive pharmacology that may reduce reliance on costly and time-intensive experimental screening.
- A comprehensive analysis reveals over 40 companies are developing more than 90 RNA interference therapeutics, highlighting the sector's rapid expansion and therapeutic potential. - Recent strategic partnerships include SanegeneBio's global licensing agreement with Genentech and Arrowhead Pharmaceuticals' collaboration with Novartis for Parkinson's disease treatment. - RNAi therapies offer promising alternatives to conventional drugs, particularly for infectious diseases and genetic disorders where traditional treatments face limitations. - Key marketed therapies like Lumasiran (OXLUMO) for primary hyperoxaluria and investigational drugs like Cemdisiran in Phase III trials demonstrate the technology's clinical advancement.
- China's National Medical Products Administration has approved Sanofi's Myqorzo (aficamten) for obstructive hypertrophic cardiomyopathy and Redemplo (plozasiran) for familial chylomicronemia syndrome. - Myqorzo represents a novel cardiac myosin inhibitor that addresses the most common inherited cardiovascular disorder, while Redemplo offers a siRNA-based approach to managing extremely high triglyceride levels. - Both therapies received breakthrough therapy designations and target severe conditions with significant unmet medical needs, potentially transforming treatment options for Chinese patients. - The approvals strengthen Sanofi's commitment to the Chinese market and expand access to innovative treatments for rare genetic disorders.
- Arrowhead Pharmaceuticals reported that its gene-silencing candidate ARO-INHBE, when combined with Eli Lilly's Zepbound, helped obese patients with diabetes lose 9.4% of their weight after 16 weeks compared to 4.8% with Zepbound alone. - The combination therapy demonstrated superior fat reduction, with patients losing 23% of visceral fat, 15% of total fat, and 77% of liver fat, significantly outperforming Zepbound monotherapy. - ARO-INHBE represents the first clinical demonstration that targeting the Activin E/ALK7 pathway can therapeutically improve body composition in obese patients with type 2 diabetes, a population that typically responds less well to incretin-based therapies. - Both ARO-INHBE and ARO-ALK7 candidates have shown generally good tolerability profiles in early-stage trials, with most adverse events being mild in severity.
- Arrowhead Pharmaceuticals announced dual public offerings totaling $700 million, including $500 million in convertible senior notes and $200 million in common stock to fund late-stage product development. - The company plans to use proceeds for research and development, clinical trials, commercialization activities, and preparation for potential commercial launches of late-stage RNAi therapeutics. - Arrowhead develops RNA interference-based medicines that silence disease-causing genes, representing a novel therapeutic approach for treating intractable diseases. - J.P. Morgan and Jefferies are serving as joint book-running managers for both offerings, with the convertible notes maturing in January 2032.
