Profile: Blueprint Medicines Corp (BPMC.OQ)
18 Apr 2019
Blueprint Medicines Corporation, incorporated on October 14, 2008, is a biopharmaceutical company. The Company focuses on patients with genomically defined diseases driven by abnormal kinase activation. The Company focuses on crafting drug candidates that provide clinical responses to patients without adequate treatment options. The Company has developed a small molecule drug pipeline in cancer and a genetic disease. The Company's drug candidate, BLU-285, targets KIT, including Exon 17 mutations, and targets PDGFRa, including the D842V mutation. These mutations activate receptor tyrosine kinases that are drivers of cancer and proliferative disorders, including gastrointestinal stromal tumors (GIST), and systemic mastocytosis (SM). Its drug candidate BLU-554 targets FGFR4, a kinase that is activated in a defined subset of patients with hepatocellular carcinoma (HCC), the common type of liver cancer.
The Company is focused on BLU-285 in an ongoing Phase I clinical trial for defined subsets of patients with GIST and an ongoing Phase I clinical trial for advanced SM. GIST is a rare disease that is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal tract (GI) tract and SM is a rare disorder that causes an overproduction of mast cells and the accumulation of mast cells in the bone marrow and other organs, which can lead to a range of debilitating symptoms and organ dysfunction and failure. The Company is engaged in developing BLU-667, a drug candidate that targets RET, a receptor tyrosine kinase that is activated by mutations or translocations and RET resistant mutants that it predicts will arise from treatment with first generation therapies. RET is a driver of disease in non-small cell lung cancer (NSCLC) and cancers of the thyroid, including medullary thyroid carcinoma (MTC). In addition, the Company has predicted neurotrophic tyrosine receptor kinase (NTRK) resistant mutants.
GIST is a tumor type that depends on continued signaling of a single, active kinase. Most GISTs result from primary mutations in KIT or PDGFRa. Imatinib inhibits KIT primary mutations, however over time, secondary mutations occur elsewhere in the KIT gene that lead to kinase activation despite the presence of imatinib, thereby leading to disease progression. The most common mutation in the PDGFRa gene is D842V, found in frontline unresectable or metastatic GIST patients. The inhibitory potency of BLU-285 and imatinib against PDGFRa D842V was evaluated in an in vitro enzyme activity assay and in a cellular model driven by an activated PDGFRa D842V mutant protein.
Blueprint Medicines Corp
45 Sidney St
CAMBRIDGE MA 02139-4133