By David Bautz, PhD
NASDAQ:ARWR
READ THE FULL ARWR RESEARCH REPORT
Business Update
Multiple Data Readouts Ahead in 2026
Following the recent approval of REDEMPLO® for the treatment of patients with familial chylomicronemia syndrome (FCS), Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) is now a commercial-stage company and recently reported that the drug is available for patients only one week following approval, which is ahead of schedule. While the company is focused on a successful commercial launch, there are a number of important data readouts ahead in 2026:
ARO-INHBE and ARO-ALK7: We anticipate initial data for ARO-INHBE and ARO-ALK7 in January 2026 and a more complete look at the data toward the end of the second quarter of 2026. ARO-INHBE targets the INHBE gene that encodes activin E, which is a ligand for ALK7. ARO-ALK7 is designed to reduce the ALK7 receptor, which binds activin E, and is a TGF-β superfamily member that is expressed in adipocytes.
Support for targeting INHBE derives from two papers that came out in 2022 that described loss of function mutations in INHBE that were associated with favorable fat distribution. Deaton et al. reported a genome-wide association study (GWAS) from 362,679 individuals that showed a predicted loss of function variant in INHBE associated with a lower waist-to-hip ratio adjusted for BMI (WHRadjBMI), which they used as a surrogate for abdominal fat that is causally linked to type 2 diabetes and coronary heart disease (Deaton et al., 2022). Akbari et al. performed a GWAS in 618,375 individuals and identified an association with favorable fat distribution, favorable metabolic profile, and protection from type 2 diabetes for heterozygous protein-truncating mutations in INHBE (Akbari et al., 2022). Arrowhead has conducted studies of Inhbe silencing in mouse obesity models with results showing reduced weight gain compared to controls. Importantly, the difference in weight gain was primarily due to changes in fat mass with no difference in lean mass.
Similar to the data supporting targeting INHBE, a GWAS study in 2019 showed that four variants in the ACVR1C gene (which encodes ALK7) were associated with reduced percent abdominal fat in DEXA imaging, a lower WHRadjBMI, and a decreased risk of developing type 2 diabetes (Emdin et al., 2019).
ARO-DIMER-PA: We anticipate early data for ARO-DIMER-PA in summer 2026. In October 2025, Arrowhead filed a CTA to initiate a Phase 1/2a clinical trial of ARO-DIMER-PA, which is designed to prevent atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia by silencing the expression of two genes: proprotein convertase subtilisin/kexin type 9 (PCSK9) and apolipoprotein C3 (APOC3). This is the first RNAi clinical candidate to target two genes simultaneously in one molecule. Mixed hyperlipidemia is characterized by elevated low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs) and is a major risk factor for ASCVD. Preclinical data for ARO-DIMER-PA were presented at the 2025 National Lipid Association Annual Scientific Sessions in May 2025. A copy of the poster presentation can be accessed here. The results of those studies showed that ARO-DIMER-PA potently lowered serum PCSK9 and APOC3 while also decreasing levels of non-HDL cholesterol, LDL-C, and TGs in dyslipidemic nonhuman primates.
ARO-MAPT: We anticipate early data for ARO-MAPT in summer 2026. In September 2025, Arrowhead filed a CTA to initiate a Phase 1/2a trial of ARO-MAPT, which is being developed for the treatment of tauopathies, including Alzheimer’s disease. This is the first therapy developed by Arrowhead that can penetrate the blood-brain-barrier to enable knockdown of target genes in the central nervous system (CNS). Tau protein is encoded by the MAPT gene and is highly expressed in neurons, where it stabilizes microtubules in axons. Hyperphosphorylation of tau protein promotes neurofibrillary tangles, which are correlated with neurodegeneration. In Alzheimer’s disease, tau neurofibrillary tangles are predictive of cognitive decline, and currently available anti-amyloid therapies only result in minimal tau reduction. At the 2025 RNA Leaders USA Congress, Arrowhead presented preclinical data that showed deep knockdown of MAPT mRNA throughout the CNS following subcutaneous administration of ARO-MAPT in nonhuman primates. This reduction in MAPT mRNA translated into long-lasting reduction in tau protein with pharmacokinetic (PK) data showing the potential for once monthly or once quarterly dosing.
Plozasiran: We anticipate topline data from the SHASTA-3 SHASTA-4, and MUIR-3 trials of plozasiran in the third quarter of 2026. SHASTA-3 and SHASTA-4 are designed to compare reductions in TGs compared to placebo over 12 months in patients with severe hypertriglyceridemia (SHTG). MUIR-3 is being conducted in patients with mixed hyperlipidemia and is designed to supplement the safety database for when the sNDA is filed for SHTG. The company will not be filing for approval in mixed hyperlipidemia patients at this time. Arrowhead completed enrollment of those trials in June 2025. Positive results will lead to a sNDA filing for SHTG before the end of 2026.
Financial Update
On November 25, 2025, Arrowhead announced financial results for fiscal year 2025 that ended September 30, 2025. The company reported revenue of $825.9 million for fiscal year 2025 compared to approximately $3.6 million for fiscal year 2024. The revenue in 2025 was primarily related to collaboration agreements with Sarepta, Sanofi, and Amgen.
R&D expenses for the year ending September 30, 2025 were approximately $607.2 million compared to $505.9 million for the year ending September 30, 2024. The increase was primarily due to increased candidate costs, salaries, facilities-related expenses, and R&D discovery expenses. G&A expenses for fiscal year 2025 were $123.9 million compared to $98.8 million for fiscal year 2024. The increase was primarily due to increased salaries, professional services, and non-cash stock-based compensation.
Arrowhead exited fiscal year 2025 with approximately $919 million in cash, cash equivalents, and investments. In October 2024, Arrowhead announced the closing of a previously announced global licensing and collaboration agreement with Novartis for ARO-SNCA. Upon closing, Arrowhead received a $200 million upfront payment. In November 2025, Arrowhead announced the second development milestone under the Sarepta agreement was reached, which triggers a $200 million payment that will be recorded in the first fiscal quarter of 2026 and received by the company in January 2026. As of November 19, 2025, Arrowhead had approximately 135.8 million shares outstanding and, when factoring in stock options and restricted stock units, a fully diluted share count of approximately 143.0 million.
Conclusion
Arrowhead is set to have another important year of data readouts in 2026, and we look forward to updates from the company throughout the year. In addition, we will be interested in how the REDEMPLO commercial launch proceeds, however we expect minimal impact to the company’s financial statements from the sale of REDEMPLO to FCS patients. The stock reacted very favorably following the approval of REDEMPLO, however we feel there is additional upside given the expected data readouts over the next 12 months. With no changes to our model our valuation remains at $76 per share.
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