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Blueprint Medicines to Present Preclinical Data on BLU-554 and on Novel Drug Target at International Liver Cancer Association 9th Annual Conference

September 1, 2015 at 8:01 AM EDT

CAMBRIDGE, Mass., Sept. 1, 2015 /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC) today announced that it will present preclinical data on its drug candidate BLU-554 in hepatocellular carcinoma (HCC), as well as on a novel drug target for a rare form of liver cancer known as fibrolamellar hepatocellular carcinoma (FL-HCC), at the International Liver Cancer Association 9th Annual Conference (ILCA) taking place Sept. 4-6 in Paris.

Blueprint Medicines

BLU-554 as a Molecularly Targeted Therapy for HCC
BLU-554 is an exquisitely selective inhibitor of fibroblast growth factor receptor 4 (FGFR4) and represents a promising new approach for molecularly targeted therapy in HCC. Liver cancer is the second leading cause of cancer-related deaths worldwide, with HCC accounting for most liver cancers. The oral presentation at ILCA details the prevalence of aberrant FGFR4 signaling, triggered by overexpression of the FGF19 ligand, in HCC, as well as preclinical data demonstrating BLU-554's anti-tumor activity in preclinical models of HCC dependent on aberrant FGFR4 signaling. BLU-554 is currently being studied in a Phase 1 trial in HCC and cholangiocarcinoma, cancer of the bile duct in which aberrant FGFR4 signaling may play a key role.

  • Date & Time: Sunday, Sept. 6 from 10:45 a.m.-12:15 p.m. (CEST)
  • Presentation Title: FGF19 Aberrations and Selective Targeting with FGFR4 Inhibitors for Hepatocellular Carcinoma
  • Session Title: General Session 5: From Drivers to Clinical Trials
  • Presenter: Ben Wolf, M.D., Ph.D., Vice President of Clinical Development, Blueprint Medicines
  • Abstract Number: 0-030
  • Location: Scene AB Ground Level

Novel Drug Target for FL-HCC
Using a proprietary computational approach to identify kinase fusions as drivers of cancer, Blueprint Medicines scientists discovered a recurrent DNAJB1-PRKACA fusion in FL-HCC tumors that causes increased signaling of the PRKACA kinase. FL-HCC is a rare, deadly and understudied cancer that arises in the otherwise normal liver of adolescents and young adults. The oral presentation at ILCA sheds light on the role of DNAJB1-PRKACA in FL-HCC and highlights its potential as a novel target for therapeutic intervention.

  • Date & Time: Friday, Sept. 4 from 11:15 a.m.-12:45 p.m. (CEST)
  • Presentation Title: Interrogation of DNAJB1-PRKACA Kinase Fusion as a Therapeutic Target in Fibromellar Hepatocelllular Carcinoma
  • Presenter: Stefanie Schalm, Ph.D., Senior Scientist, Blueprint Medicines
  • Session Title: General Session 1: Molecular Pathogenesis of Liver Cancer I
  • Abstract Number: 0-006
  • Location: Scene AB Ground Level

About Blueprint Medicines
Blueprint Medicines makes kinase drugs to treat patients with genomically defined diseases. Led by a team of industry innovators, Blueprint Medicines integrates a novel target discovery engine and a proprietary compound library to understand the blueprint of cancer and craft highly selective therapies. This empowers the Blueprint Medicines team to develop patient-defined medicines aimed at eradicating cancer.

Forward-Looking Statements
Various statements in this release concerning Blueprint Medicines' future expectations, plans and prospects, including without limitation, statements regarding Blueprint Medicines' ability to craft highly selective therapies and deliver patient-defined medicines for patients with genomically defined diseases, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995.  Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, the risk of delay of any planned clinical trials and/or development of Blueprint Medicines' drug candidates, including BLU-554, Blueprint Medicines' ability to successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its drug candidates, which may not support further development of such drug candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Blueprint Medicines' ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, competition from others developing products for similar uses, Blueprint Medicines' ability to manage operating expenses, Blueprint Medicines' ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Blueprint Medicines' dependence on third parties for various functions, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Blueprint Medicines' Quarterly Report on Form 10-Q for the most recent fiscal quarter, as well as discussions of potential risks, uncertainties and other important factors in Blueprint Medicines' subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Blueprint Medicines' views only as of today and should not be relied upon as representing its views as of any subsequent date. Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

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SOURCE Blueprint Medicines

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