Arrowhead Pharmaceuticals Q1 Earnings Call Highlights

Key Points

• REDEMPLO received FDA approval (Nov 18, 2025) for familial chylomicronemia syndrome and Arrowhead launched it in the U.S., reporting >100 prescriptions in the first ~10 weeks with geographically balanced uptake, favorable payer feedback and a unified pricing model; approvals were also secured in Canada and China (Sanofi to commercialize in Greater China).
• Plozasiran was granted FDA Breakthrough Therapy designation for severe hypertriglyceridemia, with SHASTA-3/4 and MUIR-3 blinded portions on track for mid‑2026 completion and top-line SHASTA readouts expected in Q3 2026 ahead of a planned sNDA submission, while Arrowhead also flagged promising early data and near-term readouts for obesity candidates (ARO-INHBE, ARO-ALK7), the dual-target ARO-DimerPA, and CNS program ARO‑MAPT.
• $1.33 billion of recent transactions—including a Novartis collaboration, a $200M Sarepta milestone and $930M of convertible-note and equity offerings—significantly strengthened the balance sheet; Arrowhead reported $917M cash & investments and turned a quarterly net income of $30.8M (Q1 revenue $264M) driven largely by licensing revenue despite rising R&D and commercialization expenses.

Arrowhead Pharmaceuticals NASDAQ: ARWR highlighted a series of regulatory, commercial, clinical, and financing milestones in its fiscal 2026 first-quarter earnings call, covering the period ended December 31, 2025. Management emphasized the U.S. approval and launch of its first commercial product, updates across its cardiometabolic and central nervous system (CNS) pipeline, and transactions that the company said significantly strengthened its balance sheet.

REDEMPLO approval and early U.S. launch indicators

CEO Dr. Chris Anzalone said the company received its first regulatory approval on November 18, 2025, when the FDA approved REDEMPLO as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). Arrowhead described FCS as a severe rare disease with an estimated 6,500 people in the U.S. living with genetic or clinical FCS and triglyceride levels that can be 10–100 times higher than normal, increasing the risk of acute pancreatitis.

The approval was supported by phase III PALISADE data, which management said showed a median 80% reduction in triglycerides from baseline, described as “deep and durable,” along with a lower numerical incidence of acute pancreatitis events versus placebo. The company noted it launched REDEMPLO independently in the U.S. using a “one REDEMPLO pricing model” intended to keep a consistent price across current and potential future indications.

Commercial lead Andy Davis said Arrowhead is targeting about 5,000 healthcare professionals through personal promotion alongside broader omni-channel efforts. He said early prescribing has been led by preventive cardiologists and endocrinologists, who together accounted for about 70% of prescriptions, with the remainder coming from internal medicine physicians focused on lipid disorders. Davis also said advanced practice providers are playing a meaningful role in patient identification and treatment decisions.

Management reported receiving more than 100 prescriptions in the first roughly 10 weeks in market, a period that included major holidays. Anzalone and Davis described initial uptake as geographically balanced across the U.S. Davis said the “vast majority” of patient starts were in the APOC3-class-naïve segment, with the remainder split roughly evenly between patients transitioning from expanded access and those switching from olezarsen. On switching, Davis said physicians have cited both efficacy and safety as the principal drivers.

On access and payer dynamics, Davis said Arrowhead is encouraged by payer feedback on the product’s clinical profile and its unified pricing approach. He added that discussions with payers have reflected willingness to cover REDEMPLO to label, including access based on either genetic or clinical diagnosis of FCS. In response to a question about timing from prescription to treatment, Davis said conversion varies by patient and insurance but is generally achieved “within just a couple of weeks” from prescription to patients receiving drug.

Chief Medical Officer Dr. James Hamilton reviewed key label information, stating REDEMPLO is administered as a 25 mg subcutaneous injection once every three months and can be self-administered at home. He said the U.S. label includes no contraindications, warnings, or precautions, and the most common adverse reactions include hyperglycemia, headache, nausea, and injection site reactions. Hamilton also said PALISADE included both genetic and clinically diagnosed FCS patients and that triglyceride reductions were similar between the two populations.

Additional regulatory actions and expansion efforts in hypertriglyceridemia

Anzalone said REDEMPLO also received approvals for FCS from Health Canada and China’s National Medical Products Administration, with availability in Canada expected later in 2026 and potential launches in select EU countries and the U.K. pending regulatory review. He added that in Greater China, REDEMPLO will be marketed by Sanofi.

Beyond FCS, Hamilton discussed ongoing development of plozasiran in severe hypertriglyceridemia (SHTG). He said the FDA granted breakthrough therapy designation for investigational plozasiran as an adjunct to diet to reduce triglycerides in adults with SHTG. Hamilton outlined phase III studies intended to support a supplemental NDA: SHASTA-3 and SHASTA-4 (about 750 patients combined) and MUIR-3 (1,400 patients). Arrowhead is also enrolling SHASTA-5 to directly assess reduction in acute pancreatitis risk as the primary endpoint.

Hamilton said the company remains on schedule to complete the blinded portion of SHASTA-3, SHASTA-4, and MUIR-3 in mid-2026, with top-line data expected in the third quarter of 2026 and a planned sNDA submission for SHTG before year-end. He noted the primary endpoint in SHASTA is triglyceride lowering versus placebo and said the studies are “overpowered” to show triglyceride lowering. He added that acute pancreatitis rate assessment is a key secondary endpoint in SHASTA-3 and SHASTA-4, while clarifying the studies were not prospectively powered to demonstrate acute pancreatitis reduction after one year of treatment.

Hamilton highlighted baseline risk characteristics in pooled SHASTA-3/4: 37% of enrolled patients reported triglycerides greater than 880 mg/dL and 20% had a prior history of pancreatitis. He said the studies are still blinded but that the company is seeing acute pancreatitis events occur, without providing further detail.

Pipeline updates: obesity programs, dual-target lipid candidate, and CNS delivery platform

Arrowhead also discussed multiple earlier-stage cardiometabolic programs. Anzalone said ARO-INHBE and ARO-ALK7 are in phase I/II development as potential obesity treatments, and that Arrowhead recently initiated a phase I/II study of ARO-DimerPA in mixed hyperlipidemia.

For ARO-INHBE, management cited interim data in obese patients with type 2 diabetes receiving tirzepatide. Anzalone and Hamilton said two administrations of ARO-INHBE at the 400 mg dose in combination with tirzepatide achieved roughly twofold better weight loss at week 16 compared with tirzepatide alone, alongside approximately threefold reductions in total fat, visceral fat, and liver fat measures based on week 12 MRI versus tirzepatide alone. Hamilton said the diabetic signal in combination with tirzepatide appeared clearest in early data, and he characterized ongoing studies as hypothesis-generating. He said Arrowhead is increasing patient numbers in diabetic cohorts, extending follow-up to better understand durability out to one year, and initiating monotherapy cohorts in obese diabetic patients, with additional data expected later in 2026.

For ARO-ALK7, Anzalone said early data showed dose-dependent reductions in adipose ALK7 mRNA, with a mean reduction of 88% at the 200 mg dose at week eight and a maximum reduction of 94%. He said the ARO-ALK7 study is about two quarters behind ARO-INHBE and called the dataset early and incomplete, with additional results expected later in 2026. In Q&A, management said it is “way too early” to discuss potential pricing for the obesity programs.

On ARO-DimerPA, Anzalone described it as a dual functional RNAi candidate designed to silence PCSK9 and APOC3 simultaneously to reduce both LDL cholesterol and triglycerides in mixed hyperlipidemia, a population Arrowhead estimated at about 20 million people in the U.S. He said Arrowhead believes it is the first clinical candidate targeting two genes simultaneously in one molecule. Anzalone said interim data are expected in the second half of 2026. In response to an analyst question on what reductions would be encouraging, management referenced prior dyslipidemic monkey data showing roughly 40%–50% reductions in LDL and triglycerides as a benchmark that “would be really encouraging,” while noting results will ultimately depend on human data.

Outside cardiometabolic, Anzalone and Hamilton described progress in a proprietary CNS delivery system designed to achieve blood-brain barrier penetration via subcutaneous administration. Arrowhead’s first wholly owned program using the platform is ARO-MAPT, targeting tau protein in tauopathies including Alzheimer’s disease. Hamilton said the phase I/II study includes healthy volunteers and Alzheimer’s patients, with interim data from the healthy volunteer portion anticipated in 2026 and patient data expected in 2027. Hamilton said key confidence-building healthy volunteer readouts would include safety and cerebrospinal fluid (CSF) knockdown, while patient cohorts could enable additional biomarkers, including phospho-tau measures and tau PET over time.

Financings and collaboration milestones highlighted

Anzalone said Arrowhead completed transactions totaling $1.33 billion in gross proceeds since its last reporting period. These included:

• Novartis collaboration: A global licensing and collaboration agreement for ARO-SNCA, a preclinical siRNA therapy against alpha-synuclein for synucleinopathies such as Parkinson’s disease. Arrowhead received $200 million upfront and said it is eligible for up to $2 billion in development, regulatory, and sales milestones, plus tiered royalties up to low double digits.
• Sarepta milestone: A $200 million milestone payment earned following a drug safety committee review and authorization to dose escalate, alongside achievement of a second pre-specified patient enrollment target for ARO-DM1.
• Capital markets offerings: Concurrent public offerings of $700 million aggregate principal amount of 0% coupon convertible senior notes and $230 million of common stock. Management said both were oversubscribed and described the terms as favorable.

Quarterly financial results and near-term catalysts

CFO Dan Apel reported net income of $30.8 million, or $0.22 per share, for the quarter ended December 31, 2025, compared with a net loss of $173.1 million, or $1.39 per share, in the prior-year quarter. Quarterly revenue totaled $264 million, driven primarily by licensing collaboration agreements with Sarepta and Novartis. Apel said about $229 million of revenue related to the Sarepta collaboration, including $181 million tied to the second DM1 milestone, $32 million from ongoing recognition of initial consideration, and $17 million in reimbursement of incurred collaboration program costs. He also said Arrowhead “re-recognized” $34 million of the Novartis $200 million upfront payment, with the remainder to be deferred over time as preclinical collaboration obligations are met.

Apel said Arrowhead recorded its first commercial sale of plozasiran in FCS but is not disclosing specific sales figures yet because they are not considered a meaningful driver of financials at this time.

Total operating expenses were approximately $223 million, up from $164 million a year earlier, driven by higher R&D and SG&A. Apel attributed increased R&D expense primarily to higher clinical costs associated with phase III registrational studies for plozasiran in SHTG and increased clinical supply chain costs, noting that nearly half of clinical trial spend in the quarter was associated with SHASTA-3, SHASTA-4, and MUIR. SG&A increased primarily due to commercialization investments for REDEMPLO, which management said were designed to be “highly leverageable” for potential future approvals in SHTG and HoFH.

Cash and investments totaled $917 million as of December 31, 2025, with 137.4 million common shares outstanding. Apel noted the cash balance did not include a $200 million ARO-DM1 milestone received in January, an expected $50 million Sarepta anniversary payment due on or before February 10, or the January financing proceeds from the convertible notes and equity offerings.

Looking ahead, Anzalone pointed to several anticipated 2026 events, including continued REDEMPLO commercial progress, a third-quarter 2026 readout of SHASTA-3 and SHASTA-4, second-half 2026 interim data from ARO-DimerPA, additional ARO-INHBE and ARO-ALK7 data in 2026, and early ARO-MAPT data from healthy volunteers in 2026.

About Arrowhead Pharmaceuticals NASDAQ: ARWR

Arrowhead Pharmaceuticals, Inc is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Since its founding in 2008, Arrowhead has leveraged its proprietary delivery platform—known internally as the Advanced RNAi Compound (ARC) technology—to silence disease-causing genes in patients suffering from genetically defined diseases. The company's approach aims to offer durable, targeted treatments across a range of therapeutic areas.

The company's pipeline includes multiple candidates in various stages of development.

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