Blueprint Medicines Corp (NASDAQ:BPMC) 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.

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 (Original Source)

Shares of Blueprint Medicines Corp. closed yesterday at $27.36. BPMC has a 1-year high of $37.17 and a 1-year low of $19.96. The stock’s 50-day moving average is $28.17 and its 200-day moving average is $28.10.

On the ratings front, Blueprint Medicines has been the subject of a number of recent research reports. In a report issued on July 14, Wedbush analyst David Nierengarten reiterated a Buy rating on BPMC, with a price target of $41, which implies an upside of 49.9% from current levels. Separately, on July 13, Cowen’s Eric Schmidt reiterated a Buy rating on the stock .

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, David Nierengarten and Eric Schmidt have a total average return of 59.1% and 36.5% respectively. Nierengarten has a success rate of 54.4% and is ranked #4 out of 3742 analysts, while Schmidt has a success rate of 58.5% and is ranked #33.

Blueprint Medicines Corpis a biopharmaceutical company. It is focused on improving the lives of patients with genomically defined diseases driven by abnormal kinase activation.