Arrowhead Pharma Stock Price, News & Analysis

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) is a biotechnology company focused on developing medicines that treat intractable diseases by silencing the genes that cause them. According to multiple company disclosures, Arrowhead uses a broad portfolio of RNA chemistries and efficient modes of delivery to trigger the natural mechanism of RNA interference (RNAi), leading to rapid, deep, and durable knockdown of target genes and corresponding proteins.

The company’s RNAi-based therapeutics leverage this endogenous pathway of gene silencing in living cells. By inhibiting expression of specific genes, Arrowhead aims to reduce production of disease-driving proteins across a range of serious conditions, particularly those characterized by abnormal lipid metabolism, obesity, and neurodegenerative processes. Arrowhead’s common stock is listed on The Nasdaq Global Select Market under the ticker symbol ARWR, as noted in its SEC filings.

Core Technology and TRiM™ Platform

Across its public communications, Arrowhead highlights its proprietary Targeted RNAi Molecule (TRiM™) platform. This platform is designed to deliver small interfering RNA (siRNA) molecules to specific tissues and cell types throughout the body. Company materials describe the TRiM platform as capable of delivering siRNA to multiple cell types, including liver and adipose tissue, and, in newer programs, to the central nervous system (CNS) via systemic administration.

Arrowhead reports that its therapies are engineered to induce deep and durable reductions in target mRNA and protein levels. This durability has the potential to support infrequent dosing schedules in several programs, such as once every three months for certain investigational or approved products described in the company’s news releases.

Therapeutic Focus Areas

Based on recent press releases and regulatory disclosures, Arrowhead is active in multiple therapeutic areas where gene silencing may address high unmet medical need:

• Cardiometabolic and lipid disorders: Arrowhead has developed plozasiran, marketed as REDEMPLO in certain jurisdictions, a small interfering RNA medicine targeting apolipoprotein C-III (apoC-III), a key regulator of triglyceride metabolism. REDEMPLO is described as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a severe and rare form of hypertriglyceridemia. Company communications also describe ongoing Phase 3 programs (SHASTA-3, SHASTA-4, SHASTA-5 and MUIR) evaluating plozasiran in patients with severe hypertriglyceridemia and mixed hyperlipidemia.
• Obesity and metabolic disease: Arrowhead is advancing investigational RNAi therapeutics ARO-INHBE and ARO-ALK7 as potential treatments for obesity and related metabolic complications. Interim Phase 1/2a data released by the company describe reductions in visceral fat, total fat, liver fat, and body weight, including in obese patients with type 2 diabetes mellitus when ARO-INHBE is combined with tirzepatide. These programs target components of the Activin E/ALK7 pathway, which the company characterizes as a genetically validated pathway regulating adipose fat storage and energy homeostasis.
• Neurodegenerative disease and tauopathies: Arrowhead has initiated a Phase 1/2a study of ARO-MAPT, an investigational RNAi therapeutic designed to silence CNS expression of the microtubule associated protein tau (MAPT) gene. The company states that ARO-MAPT uses a TRiM-based delivery system intended for subcutaneous administration and systemic delivery across the blood-brain barrier to achieve gene knockdown throughout the CNS, including deep brain regions. ARO-MAPT is being studied in healthy volunteers and subjects with early Alzheimer’s disease and other tauopathies.
• Liver and cardiometabolic indications in Greater China: Through its majority-owned subsidiary Visirna Therapeutics, Arrowhead has structured regional development and commercialization for certain cardiometabolic candidates in Greater China. Company news indicates that rights to commercialize REDEMPLO (plozasiran) in Greater China were acquired by Sanofi from Visirna, and that regulatory approval by the Chinese National Medical Products Administration (NMPA) for REDEMPLO in FCS triggers a milestone payment to Visirna.

Key Products and Programs Highlighted by the Company

Arrowhead’s disclosures emphasize several named products and clinical-stage programs:

• REDEMPLO™ (plozasiran): A siRNA medicine targeting apoC-III. Company press releases state that REDEMPLO has received approvals from the U.S. Food and Drug Administration, Health Canada, and the Chinese NMPA as an adjunct to diet to reduce triglycerides in adults with FCS. Arrowhead describes REDEMPLO as the first and only siRNA treatment approved in these jurisdictions for FCS and notes that it can be self-administered at home via a subcutaneous injection once every three months.
• Plozasiran in broader triglyceride disorders: Beyond FCS, plozasiran is being evaluated in Phase 3 trials (SHASTA-3, SHASTA-4, SHASTA-5, and MUIR) in adults with severe hypertriglyceridemia or mixed hyperlipidemia. The company reports that the FDA has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglyceride levels in adults with severe hypertriglyceridemia.
• ARO-INHBE: An investigational RNAi therapeutic designed to reduce hepatic expression of the INHBE gene and its secreted product Activin E. Arrowhead cites human genetic data associating INHBE loss-of-function variants with improved fat distribution and lower risk of metabolic diseases such as type 2 diabetes. Interim clinical data reported by the company describe dose-dependent reductions in serum Activin E, visceral fat, liver fat, and total fat, with preservation or increases in lean tissue in obese volunteers.
• ARO-ALK7: An investigational RNAi therapeutic targeting adipocyte expression of ACVR1C, which encodes Activin receptor-like kinase 7 (ALK7). Company data indicate that ARO-ALK7 is the first RNAi therapeutic to show adipocyte gene target silencing in a clinical trial, with reported reductions in adipose ALK7 mRNA and early reductions in visceral fat in obese participants.
• ARO-MAPT: A CNS-directed RNAi program designed to reduce tau protein by silencing MAPT. Arrowhead describes preclinical evidence of broad CNS distribution and sustained suppression of MAPT mRNA and tau protein after subcutaneous dosing in non-human primates, and has initiated a Phase 1/2a study in healthy subjects and individuals with early Alzheimer’s disease.
• ARO-SNCA (licensed to Novartis): Under an Exclusive License and Collaboration Agreement with Novartis Pharma AG, Arrowhead has granted Novartis an exclusive license for ARO-SNCA, a preclinical RNAi therapeutic being developed as a potential treatment for Parkinson’s disease and other synucleinopathies, as well as additional collaboration targets using the TRiM platform. Arrowhead’s SEC filings describe this agreement, including an upfront payment, potential milestones, and royalties.

Regulatory and Collaborative Milestones

Arrowhead’s recent SEC filings and press releases highlight several notable regulatory and collaboration events:

• Regulatory approvals for REDEMPLO (plozasiran) in FCS: The company has announced approvals by the U.S. FDA, Health Canada (via a Notice of Compliance), and the Chinese NMPA for REDEMPLO as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome. Company materials emphasize that FCS is a severe, rare disease with extremely high triglyceride levels and limited treatment options, and that REDEMPLO has been studied in both genetically confirmed and clinically diagnosed FCS patients.
• FDA Breakthrough Therapy designation for plozasiran in severe hypertriglyceridemia: Arrowhead has reported that the FDA granted Breakthrough Therapy designation to investigational plozasiran for adults with severe hypertriglyceridemia, reflecting preliminary clinical evidence of potential improvement over existing therapies.
• Collaboration with Novartis on ARO-SNCA: An 8-K filing describes an Exclusive License and Collaboration Agreement under which Novartis receives an exclusive license to ARO-SNCA and certain additional collaboration targets. Arrowhead retains responsibility for preclinical development of ARO-SNCA, while Novartis assumes clinical development, manufacturing, and commercialization responsibilities.
• Regional partnership in Greater China: Company news indicates that Sanofi will market REDEMPLO in Greater China under an agreement involving Arrowhead’s majority-owned subsidiary Visirna Therapeutics, which was created to develop and commercialize certain investigational cardiometabolic candidates in that region.

Capital Markets Activity

Arrowhead’s SEC filings document active use of the capital markets to support its research, development, and commercialization efforts. In January 2026, the company entered into underwriting agreements for concurrent public offerings of common stock (and pre-funded warrants) and 0.00% convertible senior notes due 2032. The offerings were conducted under an automatic shelf registration statement and are expected to provide net proceeds earmarked for general corporate purposes, including working capital, capital expenditures, research and development, clinical trials, commercialization activities, preparation for potential commercial launches of late-stage products, associated supply chain activities, and potential prepayment of loans under a credit facility.

Arrowhead’s filings also describe the use of capped call transactions in connection with the convertible notes offering, which are intended to mitigate potential dilution or cash payments upon conversion of the notes within specified price ranges.

Industry Classification and Business Context

For industry classification purposes, Arrowhead is associated with pharmaceutical preparation manufacturing within the broader manufacturing sector. Its operations, as described in public materials, center on research, development, and commercialization of RNAi-based therapeutics rather than traditional small-molecule manufacturing. The company’s programs span rare genetic lipid disorders, broader hypertriglyceridemia, obesity, and neurodegenerative diseases, with an emphasis on targets where gene silencing may modify underlying disease biology.

FAQs about Arrowhead Pharmaceuticals (ARWR)

• What does Arrowhead Pharmaceuticals do?
  Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Its therapies use RNA interference (RNAi) to reduce expression of specific genes and the proteins they encode, as described in the company’s press releases and SEC filings.
• How does Arrowhead’s RNAi technology work?
  According to company materials, Arrowhead uses small interfering RNA (siRNA) molecules and a Targeted RNAi Molecule (TRiM™) platform to trigger RNA interference in living cells. This mechanism inhibits expression of specific genes, leading to reduced production of disease-related proteins.
• What is REDEMPLO (plozasiran)?
  REDEMPLO, also referred to as plozasiran, is a siRNA therapeutic developed by Arrowhead to suppress production of apolipoprotein C-III (apoC-III), a protein that raises triglyceride levels. Company announcements state that REDEMPLO is approved in certain jurisdictions as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.
• Which diseases is Arrowhead targeting with plozasiran?
  Arrowhead’s disclosures indicate that plozasiran is approved as REDEMPLO for familial chylomicronemia syndrome and is being investigated in Phase 3 studies (SHASTA-3, SHASTA-4, SHASTA-5, and MUIR) for severe hypertriglyceridemia and mixed hyperlipidemia.
• What are ARO-INHBE and ARO-ALK7?
  ARO-INHBE and ARO-ALK7 are investigational RNAi therapeutics in Phase 1/2a clinical trials for obesity and metabolic disease. Company data describe ARO-INHBE as targeting the INHBE gene and Activin E, and ARO-ALK7 as targeting ACVR1C/ALK7 in adipose tissue, with reported reductions in visceral fat and other measures of body composition.
• What is ARO-MAPT and how is it delivered?
  ARO-MAPT is an investigational RNAi therapy designed to silence CNS expression of the MAPT gene, which encodes tau protein. Arrowhead reports that ARO-MAPT uses a TRiM-based delivery system intended for subcutaneous administration with systemic delivery across the blood-brain barrier to achieve gene knockdown throughout the central nervous system.
• Does Arrowhead have any major collaborations?
  Yes. An 8-K filing describes an Exclusive License and Collaboration Agreement with Novartis Pharma AG for ARO-SNCA and additional collaboration targets using the TRiM platform. Company news also references a regional arrangement involving Visirna Therapeutics and Sanofi for commercialization of REDEMPLO in Greater China.
• On which exchange does Arrowhead trade and under what symbol?
  Arrowhead’s common stock is registered on The Nasdaq Global Select Market under the ticker symbol ARWR, as stated in its SEC filings.
• What types of capital-raising transactions has Arrowhead undertaken?
  Recent SEC filings describe public offerings of common stock and pre-funded warrants, as well as an offering of 0.00% convertible senior notes due 2032. The company has also entered into capped call transactions related to the notes.
• How does Arrowhead describe its overall strategy?
  Across its communications, Arrowhead emphasizes using its TRiM platform and RNAi expertise to develop medicines that silence disease-causing genes in multiple tissues, with programs spanning cardiometabolic diseases, obesity, rare lipid disorders, and neurodegenerative conditions.