ARWR | Market Cap: $10.4B (07/13/26)
Industry:
Pharma & Biotech

DESCRIPTION

Arrowhead Pharmaceuticals is an RNAi therapeutics company that develops drugs designed to silence disease-causing genes. Arrowhead's drugs use a natural cellular mechanism to degrade messenger RNA, stopping the body from producing a target protein. Arrowhead's proprietary TRiM platform delivers these silencing molecules to specific cell types — including liver, muscle, lung, brain, and fat — enabling programs across a wide range of diseases. Arrowhead's first approved product is REDEMPLO (plozasiran), an APOC3-silencing drug approved by the FDA in November 2025 for familial chylomicronemia syndrome, a rare genetic disorder causing dangerously elevated triglycerides. Arrowhead sells REDEMPLO directly to specialty physicians in the U.S. The FCS patient population is small (~6,500 patients), and the larger commercial opportunity is in severe hypertriglyceridemia (~3-4M U.S. patients), where Phase III data are expected in mid-2026. Beyond plozasiran, Arrowhead has 18 candidates in clinical trials spanning cardiometabolic disease, CNS, obesity, and rare diseases. Arrowhead operates two revenue streams: collaboration revenue from partnerships with Sarepta, Novartis, GSK, Takeda, and Amgen — which generate upfront payments, milestones, and royalties — and nascent commercial product revenue from REDEMPLO. Collaboration revenue has been the dominant funding source, allowing Arrowhead to advance wholly-owned programs without significant equity dilution. Arrowhead also operates an in-house GMP manufacturing facility in Verona, Wisconsin, which supports both internal supply and contract manufacturing for partners.

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