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  1. Missolapola 8 juli 2025 14:57

    • Missolapola

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    Arrowhead Pharmaceuticals Initiates Phase 3 YOSEMITE Study of Investigational Zodasiran for the Treatment of Homozygous Familial Hypercholesterolemia
    July 8, 2025

    - Zodasiran targets ANGPLT3, an emerging therapeutic target to treat HoFH and other dyslipidemias

    - YOSEMITE Phase 3 study further enhances Arrowhead’s late-stage pipeline of RNAi-based cardiometabolic candidates

    PASADENA, Calif.--(BUSINESS WIRE)--Jul. 8, 2025-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease. Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies, after investigational drugs plozasiran, fazirsiran (licensed to Takeda) and olpasiran (licensed to Amgen).

    “Patients living with HoFH are difficult to adequately treat and have a very high risk of developing atherosclerotic cardiovascular disease due to severely elevated LDL-C, often exceeding 500 mg/dL. As an RNAi-based therapeutic targeting ANGPTL3, investigational zodasiran has the potential to treat HoFH in a fundamentally different manner from traditional LDL-C–lowering therapies,” said James Hamilton, M.D., Chief Medical Officer and head of R&D at Arrowhead. “In Phase 2 clinical studies, patients with HoFH receiving zodasiran achieved reductions from baseline in LDL-C, ApoB, non-HDL-C, and triglycerides, supporting its potential therapeutic role for the treatment of HoFH patients.”
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