Date: 2015-05-11
Type of information: Publication of results in a medical journal
phase:
Announcement: publication of results in the Journal of Clinical Investigation (JCI)
Company: Blueprint Medicines (USA - MA)
Product: selective kinase inhibitors
Action
mechanism: kinase inhibitor
Disease:
Therapeutic area: Cancer - Oncology
Country:
Trial details:
Latest
news: * On May 11, 2015, Blueprint Medicines announced that the Journal of Clinical Investigation (JCI) published an overview of the Company\'s kinase drug discovery and development strategy. The publication underscores Blueprint Medicines\' focus on identifying novel genomically defined disease drivers and the use of novel chemistry from its proprietary compound library to craft highly selective kinase inhibitors for new and difficult-to-drug kinase targets. \"Targeting cancer with kinase inhibitors,\" authored by Blueprint Medicines\' scientists, was published in the May 2015 issue of JCI, a leading journal focused on science and clinical research to advance medicine. The JCI review article describes how the abnormal activation of kinases drives many hallmarks of tumor biology. Kinases are proven cancer drug targets, but progress in recent years has been largely incremental. In the article, Blueprint Medicines scientists call attention to important opportunities for advancing the field that serve as the foundation for the Company\'s drug discovery and development strategy: Uncovering novel disease drivers and drug targets: New genomics and sequencing approaches are needed to elucidate the biology of the full kinome, which is comprised of 518 kinases, and to uncover the function of kinases of unknown biology (or KUBs) and their potential for drug discovery. Crafting highly selective kinase inhibitors: Novel chemical matter and structure-informed design are needed to create more selective and potent therapies that can inhibit new and difficult-to-drug kinase targets. Complete target inhibition with minimal off-target effects is also needed to provide more durable responses with few toxicities, improving outcomes for patients. Predicting resistance mutations: Novel approaches in structural biology and computational chemistry are needed to predict future resistance mutations which arise from targeted therapies. Novel chemistry is needed to design a single therapy that can target both the original primary disease driver and the subsequent mutations that cause patients to become refractory to treatment. Developing novel combination therapies: Combinations of highly targeted kinase inhibitors acting within a single kinase pathway or between parallel kinase pathways are needed to mount a multi-pronged attack that improves efficacy and delays the onset of treatment resistance. Blueprint Medicines expects to advance its lead programs into Phase 1 clinical trials in mid-2015: BLU-554, a selective inhibitor of fibroblast growth factor receptor 4 (FGFR4), in hepatocellular carcinoma, and BLU-285, a selective inhibitor of PDGFRα D842V and KIT Exon 17 mutants, in gastrointestinal stromal tumors and systemic mastocytosis. The company also has a drug discovery program targeting RET kinase fusions and predicted resistance mutations.
