Acoramidis CAS# 1446711-81-4 3-[3-[(3,5-二甲基-1h-吡哧唑-4-基)丙氧]-4-氟苯甲酸

Acoramidis (AG10) is a potent, selective, orally bioavailable stabilizer of transthyretin (TTR). In preclinical biochemical assays, it bound TTR with high affinity (Kd₁ ~5 nM) and prevented dissociation of both wild-type and mutant TTR tetramers, showing greater kinetic stabilization than tafamidis. Acoramidis inhibited TTR amyloid fibril formation in vitro and protected cardiomyocytes from toxicity induced by destabilized TTR variants. In rat and dog studies, it demonstrated good oral bioavailability, dose-dependent TTR occupancy, and sustained stabilization of circulating TTR, with favorable tolerability. These findings supported its development for transthyretin amyloidosis.