LML419

LML419 is an orally bioavailable, small-molecule inhibitor of the Bromodomain and Extra-Terminal (BET) family of proteins, which includes BRD2, BRD3, BRD4, and BRDT. Developed by Novartis, LML419 was primarily investigated for the treatment of myeloproliferative neoplasms (MPNs), including myelofibrosis, essential thrombocythemia, and polycythemia vera. The drug functions as an epigenetic modulator by competitively binding to the acetyl-lysine recognition pockets (bromodomains) of BET proteins, thereby disrupting their recruitment to chromatin and inhibiting the transcription of key oncogenic and pro-inflammatory genes, such as MYC. Although it reached Phase 2 clinical trials, LML419 was subsequently removed from the Novartis development pipeline in late 2022.

Other names

2-((4s)-6-(4-chlorophenyl)-1-methyl-4h-benzo[c]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate2-[(4S)-6-(4-chlorophenyl)-1-methyl-4H-isoxazolo[4,5-e][1,4]benzodiazepin-4-yl]acetamideCAS 1801215-52-4CAS1801215-52-4CAS-1801215-52-4