October 01, 2014 9:42 AM ET


Company Overview of Blueprint Medicines Corp.

Company Overview

Blueprint Medicines Corp., an oncology company, engages in the discovery and development of highly selective kinase inhibitors for genomically defined cancer subsets. The company develops a platform that combines genomics with a novel library of kinase inhibitors to enable the development of highly selective compounds against clear genomic driver targets. Its products include KIT D816V, a validated driver mutation in aggressive systemic mastocytosis; isoform selective inhibitors of FGFR4; and programs against known and novel kinase fusions and resistance mutants. Blueprint Medicines Corp. was incorporated in 2008 and is based in Cambridge, Massachusetts.

215 First Street

Cambridge, MA 02142

United States

Founded in 2008



Key Executives for Blueprint Medicines Corp.

Chief Executive Officer
Age: 43
Executive Chairman
Age: 56
Co-Founder and Director
Age: 42
Chief Business Officer
Clinical Advisor and Member of Scientific Advisory Board
Compensation as of Fiscal Year 2014.

Blueprint Medicines Corp. Key Developments

Personal Genome Diagnostics Inc. and Blueprint Medicines Disclose First-Ever Comprehensive Genomic Study of Malignant Mixed Mullerian Tumors

Personal Genome Diagnostics Inc. and Blueprint Medicines disclosed the first-ever comprehensive genomic study of malignant mixed Mullerian tumors (MMMT), an aggressive and deadly cancer of the female reproductive system, also known as carcinosarcoma. The new study, published in the current online edition of Nature Communications, uncovers genetic alterations previously not associated with MMMT. These genetic alterations likely play a role in development of the tumors and can serve as targets for anticancer drugs. Researchers from PGDx and Blueprint Medicines, with colleagues from Johns Hopkins University and Oregon Health and Science University, discovered that MMMT/carcinosarcomas have many mutations located in clinically relevant genes, such as PIK3CA, KRAS and DNA repair pathway genes. Some of these previously unidentified genetic mutations may be addressed by existing therapies or by investigational drugs currently in clinical trials. The analysis also revealed that a high fraction of mutations were in "chromatin remodeling" genes, which regulate the structure of chromosomes and, when mutated, are thought to have dramatic effects on the biology of the cell. Chromatin remodeling genes are currently being evaluated as potential targets for epigenetic and other novel therapies.

Blueprint Medicines Corp. Presents at 2014 Morgan Stanley Global Healthcare Conference, Sep-10-2014

Blueprint Medicines Corp. Presents at 2014 Morgan Stanley Global Healthcare Conference, Sep-10-2014 . Venue: The Grand Hyatt Hotel, New York, New York, United States.

Blueprint Medicines Presents Positive Data on BLU-554 for Hepatocellular Carcinoma

Blueprint Medicines announced the presentation of preclinical data investigating BLU-554, a first-in-class selective fibroblast growth factor receptor 4 (FGFR4) inhibitor, for the treatment of patients with hepatocellular carcinoma (HCC). Results demonstrate in vivo efficacy, including sustained tumor regression and complete remissions in mice with tumors that represent a genomically defined subset of human HCC. The oral presentation was given at the International Liver Cancer Association 8th Annual Conference in Kyoto, Japan. Results showed dose-dependent tumor regression of BLU-554 in xenograft models with an abnormally activated FGFR4 signaling pathway due to overexpression or amplification of the FGFR4 ligand FGF19. 20% of the mice remained tumor free 30 days after treatment, and BLU-554 was well tolerated. Blueprint Medicines expects to initiate clinical trials with BLU-554 for the treatment of HCC patients with an abnormally activated FGFR4 signaling pathway in 2015. The targeted patient population is estimated to represent approximately one-third of all HCC patients.

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