An oral, liver-restricted LXR inverse agonist for dyslipidemia: preclinical development and phase 1 trial.

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Title: An oral, liver-restricted LXR inverse agonist for dyslipidemia: preclinical development and phase 1 trial.
Authors: Li X; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Benegiamo G; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Vijayakumar A; OrsoBio, Menlo Park, CA, USA., Sroda N; OrsoBio, Menlo Park, CA, USA., Kimura M; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Huss RS; OrsoBio, Menlo Park, CA, USA., Weng S; OrsoBio, Menlo Park, CA, USA., Murakami E; OrsoBio, Menlo Park, CA, USA., Kirby BJ; OrsoBio, Menlo Park, CA, USA., von Alvensleben GVG; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Kremoser C; WM Therapeutics, Heidelberg, Germany., Gane EJ; New Zealand Clinical Research, University of Auckland, Auckland, New Zealand., Takebe T; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Premium Research Institute for Human Metaverse Medicine (WPI-PRIMe), and Division of Stem Cell and Organoid Medicine, The University of Osaka, Suita, Japan., Myers RP; OrsoBio, Menlo Park, CA, USA., Subramanian GM; OrsoBio, Menlo Park, CA, USA. mani@orsobio.com., Auwerx J; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland. admin.auwerx@epfl.ch.
Source: Nature medicine [Nat Med] 2026 Mar; Vol. 32 (3), pp. 883-893. Date of Electronic Publication: 2026 Jan 16.
Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1546-170X
DOI:10.1038/s41591-025-04169-6