Blueprint is positioning its kinase inhibitor BLU-285 to take a dominant position in gastrointestinal tumors, with preclinical and Phase I data showing the compound inhibits not only mutant forms of c-Kit and PDGFRA that underlie resistance to marketed drugs, but primary mutations too. The findings, published in Science Translational Medicine last week, provide a case study for how a company can go from looking for compounds against specific mutations to producing a therapy that may be broadly useful in the first-line setting.
Third Rock Ventures founded Blueprint Medicines Corp. in 2011 to develop new types of kinase inhibitors that go beyond those on the market by addressing mutations that lead to acquired resistance. The company went public with a $146.6 million IPO in 2015 and has since raised another $373.8 million in follow-ons.
Its co-founders list several veterans of the receptor tyrosine kinase (RTK) inhibitor field, including Nicholas Lydon, Brian Druker and Charles Sawyers, who spearheaded the development of Novartis AG’s Gleevec imatinib, an inhibitor of BCR-ABL and c-Kit that was one of the first targeted therapies in cancer created by rational drug design. Druker is director of the Oregon Health & Science University (OHSU) Knight Cancer Institute, Sawyers is director of the Human Oncology and Pathogenesis Program at Memorial Sloan Kettering Cancer Center and Lydon is co-founder and head of the consulting firm Granite Biopharma LLC.
Novartis markets Gleevec for gastrointestinal stromal tumors (GIST) and chronic myelogenous leukemia (CML).