Omaveloxolone CAS# 1474034-05-3 奥马索龙

Omaveloxolone is a semi-synthetic oleanane triterpenoid that activates Nrf2 by disrupting Keap1–Nrf2 binding. In biochemical and cellular assays, it increased Nrf2-driven antioxidant response element signaling with EC₅₀ values of ~20–50 nM and reduced pro-inflammatory mediators (e.g., NO, TNF-α) in macrophages. It improved mitochondrial bioenergetics in neuronal cells, increasing ATP production and reducing ROS. In the KIKO and YG8R Friedreich’s ataxia mouse models, oral omaveloxolone improved rotarod performance, reduced dorsal root ganglia pathology, and restored mitochondrial enzyme activities. Pharmacokinetic studies showed good oral exposure and CNS penetration, supporting further development for mitochondrial and oxidative stress–driven disorders.