Analyst Conference Summary

biotechnology

conference date: November 25, 2025 @ 1:30 PM Pacific Time
for quarter ending: September 30, 2025 (fiscal Q4, fourth quarter fiscal 2025)

Forward-looking statements

Overview: Redemplo (plozasiran) approved by FDA to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

Basic data (GAAP):

Did not give figures for the quarter.

Full fiscal year 2025 revenue was $829 million, up from $4 million year-earlier. Revenue was from milestone and other partner payments, not sales.

Full fiscal year 2025 net income was negative $2 million, up from negative $599 million year-earlier.

Full fiscal year 2025 Diluted EPS was negative $0.01, up from negative $5.00 year-earlier.

Guidance:

Does not expect substantial Redemplo revenue in fiscal 2026, but expects cash to last into 2028.

Conference Highlights:

CEO Christopher Anzalone said "The recent FDA approval of Redemplo, indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), is a transformational milestone for Arrowhead. Redemplo is the first and only FDA-approved siRNA medicine for people living with FCS and represents the first FDA-approval for a medicine leveraging Arrowhead's proprietary and differentiated Targeted RNAi Molecule (TRiM) platform. However, Arrowhead is truly just getting started and we are well positioned to execute on our aggressive goals in discovery, clinical development, business development, and commercial to enable multiple potential new launches, both independently and with partners, over the coming years." [WM: Note that Ionis had a similar FCS therapy approved in Decembers 2024.]

In November 2025 the FDA approved Redemplo (plozasiran) for FCS (familial chylomicronemia syndrome). This is Arrowhead's first approved medicine. Has built out the sales and marketing teams for plozasiran. Has started working with payers and providers. Feedback has been favorable. Helps to prevent pancreatitis from FCS. $60,000 annual price, dosing every 3 months. Priced for the larger severe hypertriblyceridemia market, where it is in a Phase 3 trial. Believes is best in class. [WM: This price is far less than the price of Tryngolza for FCS]

In the quarter announced a global licensing agreement with Novartis for ARO-SNCA, a preclinial asset for Parkinson's Disease and possble other targets. Received $200 million upfront. Future payments up to $2 billion, plus tiered low double digit royalties.

In fiscal Q4 2025 Arrowhead filed a request for regulatory clearance for a Phase 1/2a clinical trial of ARO-MAPT as a potential treatment for tauopathies including Alzheimer's disease.

In fiscal Q4 Arrowhead dosed the initial subject in a Phase 3 clinical trial of zodasiran, for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease.

In fiscal Q4 riled a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-DIMER-PA for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia. ARO-DIMER-PA is a dual functional RNAi therapeutic designed to silence expression of the proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes. It is the first clinical candidate to target two genes simultaneously in one molecule.

In fiscal Q3 2025 Arrowhead earned a $100 million milestone payment from Sarepta upon dose escalation target for ARO-DM1. In February 2025 completed a major licensing and collaboration deal with Sarepta Therapeutics. Arrowhead received $835 million upon closing (in 2025): $500 million cash and a $325 million equity investment priced at a 35% premium ($27.30). Will also receive $250 million over five years. Also possible $300 million in the next year as a current Phase 1/2 study, ARO-DM1, continues enrollment. Possible $10 billion in milestone payments, plus tiered royalties on sales up to low double digits. Licensing out ARO-DUX4, ARO-DM1, ARO-MMP7, ARO-ATXN2, all clinical stage, plus the preclinical ARO-HTT, ARO-ATXN1 and ARO-ATXN3. Also allows Sarepta to select six new targets for discover. All using the TRiM platform for RNAi. If Sarepta should not be able to pay milestones, the potential therapies will be returned to Arrowhead, but no repayment would be due to Sarepta. So, that is fine. Believes Sarepta will be able to pay. On November 24, 2025 announced had qualified for another $200 million milestone, to be recorded in fiscal Q1 2025. Elected to receive approximately $50 million worth of Arrowhead common stock and approximately $50 million in cash from Sarepta Therapeutics to satisfy the payment of the first $100 million milestone owed to Arrowhead. These shares were subsequently placed into treasury to reduce the number of Arrowhead shares outstanding.

In Q3 2025 Arrowhead dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, an investigational RNAi therapeutic being developed as a potential treatment for obesity.

In August 2025 announced Visirna Therapeutics, majority owned by Arrowhead, will sell the rights to plozasiran in China to Sanofi. Upfront payment $130 million. Possible milestones up to $265 million. Plus royalties. Signed the agreement in Q4.

Fazirsiran (licensed to Takeda) is in Phase 3.

Olpasiran, licensed to Amgen, had fully enrolled its Phase 3 trial.

See also the Arrowhead Pharmaceuticals pipeline page.

Cash and equivalents (including investments) ended at $782 million, down sequentially from $0.9 billion. For full fiscal year 2025 $180 million cash from operations.

Operating expenses for full fiscal year 2025 of $731 million included $607 million for R&D and $124 million for G&A. Leaving operating income of $98 million. Other expense $47 million. Tax $21 million. Non-contolling interest income $32 million.

Q&A selective summary:

Acute pancreatitis sufficent data? Shasta 3 and 4 were powered for triglyceride reduction. They were designed to be pooled v. placebo for reduction of pancreatitis. So the power is reasonably good. Shasta 5 is specifically designed to have pancreatitis as a primary endpoint. We did revise the design recently to be more representative of the SHTG patient population.

Obesity data plans? First study is nearly fully enrolled. INHBE first cohort data and combo cohort should be come abailable in 2026. ALK7 also.

MAPT program, competitor failed program? The JnJ monoclonal antibodies do not pass the blood brain barier well, then attach to Tau outside the cell. Our program goes inside the neuron to try to reduce the tau.

Outlined likely data to be released in 2026.

The RAGE program is being verified in steps, so data not likely in 2026. With data we can decide if we want to build out a pulmonary franchise.

There are a lot of therapies for LDL, but there are a lot of patients (20 million) with mixed hypertriglyceridemia, our potential drug could address both issues, combined with a statin. Dimer platform did well in animal studies.

Redemplo pediatric pathway? We plan pediatric work in the U.S. and Europe, requires weight-based dosing for studies.

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