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BLU-654 is an investigational small molecule drug designed as a potent and selective inhibitor of mutant KIT, with particular selectivity for KIT V654A, a mutation associated with resistance to first-generation tyrosine kinase inhibitors in gastrointestinal stromal tumor (GIST)[7]. BLU-654 was originally developed by Blueprint Medicines and subsequently licensed to IDRx as IDRX-73 for further development[7]. The drug aims to overcome resistance mechanisms in patients with GIST who have failed prior treatments by targeting KIT mutations that drive tumor progression[7]. Its mechanism of action involves inhibition of the KIT receptor tyrosine kinase, with a focus on the V654A mutant[7].
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