Blueprint Medicines Corporation develops drugs of small molecule kinase inhibitors that target genomic drivers in various cancers and a rare genetic disease. Its lead drug candidates include avapritinib, which completed Phase I clinical trials that targets PDGFRA Exon 18 mutant GIST and KIT-driven GIST; and BLU-554, which is in Phase I clinical trials an orally available, potent, and irreversible inhibitor of the kinase FGFR4 that is activated in a defined subset of patients with hepatocellular carcinoma. It is also developing BLU-667, an orally available and potent inhibitor that targets RET, a receptor tyrosine kinase that is abnormally activated by mutations or translocations; and RET resistant mutants that would arise from treatment with first generation therapies. In addition, the company is developing BLU-782, an inhibitor targeting the kinase ALK2 for the treatment of fibrodysplasia ossificans progressiva, a rare genetic disease caused by mutations in the ALK2 gene, ACVR1. The company has collaboration and license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. to discover, develop, and commercialize small molecule therapeutics targeting kinases. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was founded in 2008 and is headquartered in Cambridge, Massachusetts.
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