Arrowhead Pharmaceuticals, Inc.

What they do

Arrowhead develops RNA interference (RNAi) therapeutics using its proprietary TRiM platform, which uses ligand-mediated delivery to target specific tissues. The company has 18 clinical-stage drug candidates ranging from Phase 1 to Phase 3, and operates lab facilities in California and Wisconsin. Its lead product REDEMPLO (plozasiran) is approved for familial chylomicronemia syndrome (FCS), and it has multiple partnerships with companies like Amgen, Takeda, GSK, Sarepta, and Novartis.

Revenue drivers

• REDEMPLO (plozasiran) commercial sales — First commercial product, approved in the U.S., EU, Canada, Australia, and China (with Greater China rights out-licensed to Sanofi). Prescription volume roughly doubled in the fiscal third quarter of 2026, with over 400 unique prescribers.
• Licensing and collaboration agreements — Multiple deals with Amgen, Takeda, GSK, Sarepta, Novartis, Madrigal, and others. Include upfront payments, milestones, and royalties; e.g., Madrigal paid $25 million upfront for ARO-PNPLA3 with potential milestones up to $975 million.
• Pipeline progress and milestones — Phase 3 candidates (plozasiran for sHTG, zodasiran, fazirsiran) could generate future regulatory milestones and commercialization revenue if approved.

Recent performance

For fiscal 2025, revenue was $829.4 million and net loss was $1.6 million (diluted EPS -$0.01), a major improvement from a net loss of $599.5 million in 2024. Revenue in fiscal 2026 first three quarters was $73.7 million (March 2026) and $75.3 million (June 2026), down from $256.5 million and $264.0 million in the prior-year quarters, reflecting lower collaboration revenue. Operating cash flow was positive $179.6 million in fiscal 2025, but cash and equivalents were $54.8 million as of June 30, 2026.

Strategy

Arrowhead is focused on building commercial capabilities for REDEMPLO in FCS, then expanding to severe hypertriglyceridemia (sHTG), pending supplemental NDA filing in 2026. The company plans to progress its broad pipeline, including moving zodasiran, fazirsiran, and other candidates through Phase 3. It uses licensing deals to generate non-dilutive capital and expand reach of its TRiM platform. Key partnerships include Sarepta, Amgen, Takeda, GSK, Novartis, and Sanofi (for Greater China rights to plozasiran).

Risks

• Commercial execution risk — REDEMPLO launch is early; reimbursement coverage and physician adoption are still developing, and commercial success is unproven.
• Clinical trial failure risk — Many pipeline candidates are in Phase 1-3; failure of any Phase 3 trial (e.g., SHASTA studies for sHTG, YOSEMITE for HoFH) would delay or prevent regulatory approvals.
• Regulatory and approval delays — Supplemental NDA for sHTG is planned for 2026 but depends on successful Phase 3 completion; other candidates may face similar regulatory hurdles.
• Financial runway risk — Cash and equivalents were only $54.8 million as of June 30, 2026, which may be insufficient to fund all planned operations without additional financing or partnerships.

Outlook

Management expects to file a supplemental NDA for plozasiran in sHTG in 2026, based on positive SHASTA-3 and SHASTA-4 results. They anticipate completion of the YOSEMITE trial for zodasiran in mid-2027 and plan to seek regulatory approval thereafter. REDEMPLO launches continue to expand globally, and the company expects to build a larger commercial footprint for sHTG.