Arrowhead Pharmaceuticals, Inc.

Arrowhead develops medicines that treat intractable diseases by silencing the genes that cause them. The company uses a broad portfolio of RNA chemistries and modes of delivery, with therapies that trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. There are currently 18 Arrowhead-discovered drug candidates in clinical trials, ranging from early stage (Phase 1) to late stage (Phase 3). The company also has a discovery stage pipeline capable of generating multiple new clinical candidates each year. Arrowhead achieved its first commercial launch in 2025, when the U.S. Food and Drug Administration ("FDA") approved REDEMPLO ® (plozasiran) as an adjunct to diet to reduce triglycerides in adults with Familial Chylomicronemia Syndrome ("FCS"). Additionally, phase 3 studies (SHASTA-3, SHASTA-4, and SHASTA-5) for severe hypertriglyceridemia ("sHTG") have been fully enrolled. The company plans to file a supplemental NDA for this indication in 2026, pending successful completion of Phase 3 clinical studies. Arrowhead has built a commercial organization to support marketing in FCS, a rare disease, and plans to progressively build its commercial capabilities to also support marketing in sHTG, a higher prevalence disease. The company has entered into multiple license and collaboration agreements with biotech and pharmaceutical companies, including Sarepta Therapeutics, Inc., Amgen Inc., Takeda Pharmaceutical Company Limited, Glaxosmithkline Intellectual Property (No. 3) Limited, and Novartis Pharma AG, for programs that Arrowhead does not intend to commercialize independently.