Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations

Lynch, David R; Perlman, Susan; Schadt, Kim

doi:10.1080/14737175.2024.2310617

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Cited by 5 publications

References 73 publications

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Friedreich Ataxia

Perlman

2024

Reference Module in Neuroscience and Biobehavioral Psychology

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Friedreich Ataxia

Perlman

2024

Reference Module in Neuroscience and Biobehavioral Psychology

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Therapies that address altered gene regulation

Smith

2025

Genetic Disease Discovery and Therapeutics

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Discovery of Propionic Acid Derivatives with a 5-THIQ Core as Potent and Orally Bioavailable Keap1–Nrf2 Protein–Protein Interaction Inhibitors for Acute Kidney Injury

Shi,

Zhang,

Wang

et al. 2024

J. Med. Chem.

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Keap1 plays a crucial role in regulating the Nrf2-mediated cytoprotective response and is increasingly targeted for oxidative stress-related diseases. Using small molecules to disrupt the Keap1–Nrf2 protein–protein interaction (PPI) has emerged as a new strategy for developing Nrf2 activators. Through extensive structure–activity relationship studies, we identified compound 56, which features a unique 5-tetrahydroisoquinoline scaffold and acts as a potent inhibitor of the Keap1–Nrf2 PPI. Compound 56 exhibited significant inhibitory activity (IC50 = 16.0 nM) and tight Keap1 binding affinity (K d = 3.07 nM), along with acceptable oral bioavailability (F = 20%). Notably, 56 enhanced antioxidant defenses in HK-2 renal tubular epithelial cells and significantly reduced plasma creatinine and blood urea nitrogen levels in acute kidney injury (AKI) mice. These findings collectively position compound 56 as a promising candidate for the treatment of AKI.

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Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations

Cited by 5 publications

References 73 publications

Friedreich Ataxia

Friedreich Ataxia

Therapies that address altered gene regulation

Discovery of Propionic Acid Derivatives with a 5-THIQ Core as Potent and Orally Bioavailable Keap1–Nrf2 Protein–Protein Interaction Inhibitors for Acute Kidney Injury

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