Arrowhead Pharmaceuticals Receives $50 Million Milestone from Royalty Pharma

PASADENA, Calif.-- Arrowhead Pharmaceuticals, Inc. announced the receipt of a $50 million milestone payment from Royalty Pharma plc. This payment was triggered by the completion of participant enrollment in the Phase 3 OCEAN(a) - Outcomes Trial for olpasiran, which is being conducted by Amgen. Arrowhead has significant agreements with both Amgen and Royalty Pharma, making it eligible to receive up to an additional $375 million from Amgen and $110 million from Royalty Pharma based on various developmental, regulatory, and sales milestones related to olpasiran.

Christopher Anzalone, Ph.D., President and CEO of Arrowhead, expressed satisfaction with the rapid enrollment in the OCEAN(a) - Outcomes Trial, highlighting the strong interest in olpasiran. This drug was developed using Arrowhead’s proprietary TRiMTM technology and was licensed to Amgen in 2016. He emphasized the importance of partnerships in Arrowhead’s strategy and acknowledged the efforts made to bring this potential new therapy closer to patient availability.

Arrowhead's pipeline now includes three TRiMTM-enabled candidates in Phase 3 trials: olpasiran, fazirsiran, and plozasiran. Notably, plozasiran, a leading investigational RNA interference (RNAi) therapeutic aimed at reducing the production of Apolipoprotein C-III (APOC3), is set to complete its first pivotal Phase 3 study this quarter, with results expected shortly thereafter.

Olpasiran is a small interfering RNA (siRNA) created using Arrowhead's Targeted RNAi Molecule (TRiMTM) platform, designed to lower levels of lipoprotein(a) (Lp(a)), a genetic risk factor for cardiovascular disease. The primary goal of the Phase 3 OCEAN(a) - Outcomes Trial is to assess the effect of olpasiran treatment compared to a placebo on the risk of coronary heart disease death, myocardial infarction, or urgent coronary revascularization in patients with atherosclerotic cardiovascular disease and high levels of lipoprotein(a).

Lipoprotein(a), or Lp(a), is predominantly genetically determined and is considered an independent risk factor for cardiovascular disease (CVD). While a definitive threshold for elevated Lp(a) levels is yet to be established, about 20% of adults have Lp(a) levels higher than 125 nmol/L (or around 50 mg/dL). Emerging evidence from various studies indicates that high Lp(a) levels may contribute to myocardial infarction, stroke, and peripheral arterial disease.

Arrowhead Pharmaceuticals is focused on developing medicines to treat difficult diseases by silencing the genes that cause them. The company utilizes a wide portfolio of RNA chemistries and efficient delivery methods to trigger the RNA interference mechanism, leading to rapid, deep, and durable knockdown of target genes. RNA interference (RNAi) is a natural cellular mechanism that inhibits the expression of specific genes, thereby affecting the production of particular proteins. Arrowhead’s RNAi-based therapeutics take advantage of this natural gene-silencing pathway.

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