Analyst Conference Summary

biotechnology

conference date: August 7, 2025 @ 1:30 PM Pacific Time
for quarter ending: June 30, 2025 (fiscal Q3, third quarter fiscal 2025)

Forward-looking statements

Overview: Focus was on partnership with Sarepta.

Basic data (GAAP):

Revenue was $28 million, down sequentially from $543 million, and up from $0 million year-earlier.

Net income was negative $175 million, down sequentially from $370 million, and down from negative $171 million year-earlier.

Diluted EPS was negative $1.26, down sequentially from $2.75, and up from negative $1.38 year-earlier (on higher share count).

Guidance:

none

Conference Highlights:

CEO Christopher Anzalone said "Arrowhead continues to achieve strong execution in discovery, clinical and regulatory, and business development. Our pipeline has become quite mature, with four Arrowhead discovered candidates currently in pivotal Phase 3 studies. In addition, our commercial buildout is designed to make us launch ready on day one, should plozasiran receive regulatory approval on the November 18, 2025, PDUFA date. We continue to maintain a strong balance sheet, which we believe gives us the financial resources to move multiple innovative new medicines through the clinical and regulatory process and ultimately get them to the patients who need them " Believes has potential for more capital inflows from business development. Also hopes to introduce 3 or 4 new products to the clinic each year.

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.

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.

In Q2 2025 Arrowhead announced topline results from Part 2 of a Phase 1/2 Study of ARO-C3 in patients with IgA nephropathy. ARO-C3 achieved deep and sustained reductions in alternative pathway complement activity and proteinuria. Mean sustained reductions in C3 of greater than 87%, AH50 of greater than 76%, and Wieslab AP of greater than 89% through week 24. Mean reduction in spot urine protein-to-creatinine ratio (UPCR) of 41% by week 24.

As of August 2025 had built out the sales and marketing teams for plozasiran. Has started working with payers and providers. Feedback has been favorable.

The revenue in the June quarter was related to the Sarepta deal.

In Q1 2025 the FDA accepted the NDA for plozasiran for FCS (familial chylomicronemia syndrome) and assigned a PDUFA data of November 18, 2025. There is no plan for an advisory committee meeting. SHTG (severe hypertriglyceridemia) Phase 3 trials begun in fiscal Q2 2024 and fully enrolled in May 2025; potential data and FDA filing in late 2026. Plozasiran marketing teams are already in place and field force will be hired over the next few months. [competing with the Ionis product, Tryngolza (Olezarsen), approved by FDA in December 2024. Believes differentiated and superior.]

In 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.

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.

Zodasiran (formerly ARO-ANG3) dosed first patient in Phase 3 in Q3 2025. In Q2 read out Phase 2 data at AHA showing reduced levels of triglicerides and LDL cholesterol for mixed dyslipidemia.

Fazirsiran (licensed to Takeda) is in Phase 3.

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

Footprint expansion is complete, plans little capital expense in 2025.

See also the Arrowhead Pharmaceuticals pipeline page.

Cash and equivalents (including investments) ended at $0.9 billion, down sequentially from $1.1 million. $155 million cash used in operations.

Operating expenses of $193 million included $162 million for R&D and $31 million for G&A. Leaving operating loss of $179 million. Other expense $14 million. Tax benefit $0.4 million.

Q&A selective summary:

Ionis competition? Studies are hard to compare. Our data is very good. We believe we have a best in class triglyceride reducer. The patient populations look similar, so it will be interesting to see the data they report out in September.

SHTG pancreatitis event rate? We count true adjudicated cases of pancreatitis, but Ionis is including cases of abdominal pain. It is an important metric.

Pricing FCS, SHTG? Still looking at pricing, but SHTG would be lower than FCS.

Sarepta potential share sales? The do own some ARWR shares. We don't know their plans, but there are other parties who have expressed interest in those shares.

Visirna cash? A large portion of that will go to shareholders, which includes Arrowhead.

Annual payments from Sarepta? $50 million per year, first quarter each year.

Plozasiran auto-injector? We will initially use a prefilled syringe, but we should have the auto-injector around time of SHTG launch.

DM1 program dosing? We still can go up to 12 in the current study.

Obesity, size of trial, muscle sparing color? Phase 1 study is hypothesis generating, the powering would be in later studies. Probably quarterly to half-yearly dosing.

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