Blueprint Medicines Presents Positive Data on First Selective FGFR4 Inhibitor

CAMBRIDGE, Mass., April 7, 2014 (GLOBE NEWSWIRE) -- Blueprint Medicines today announced that promising preclinical data on its first-in-class selective fibroblast growth factor receptor 4 (FGFR4) inhibitor will be presented during a late-breaking minisymposium at the 105th Annual Meeting of the American Association for Cancer Research (AACR). The oral presentation by Christoph Lengauer, Ph.D., M.B.A., Chief Scientific Officer of Blueprint, will feature in vivo efficacy data including sustained tumor regression, which highlight the potential clinical benefit for patients with hepatocellular carcinoma (HCC) with amplification or overexpression of FGF19. FGF19 is the ligand of FGFR4 and a genomic driver in HCC.

"We are very encouraged by these data because they confirm the potential clinical benefit of selectively inhibiting FGFR4 as a genomic target for treating HCC patients," said Dr. Lengauer. "These data further support the unique platform Blueprint has built and is utilizing for drug discovery. Our product engine enabled us to block FGFR4 selectively with a small molecule inhibitor and to achieve the highest level of selective potency accomplished to date."

In the studies to be presented, Blueprint's FGFR4 inhibitor was evaluated in a FGF19 amplified and a FGF19 overexpressing patient-derived xenograft model. Results showed dose-dependent tumor regression in both models. Blueprint's selective FGFR4 inhibitor also proved to be well tolerated. A clear pharmacokinetic, pharmacodynamic and efficacy relationship has been established.

The presentation will also include data on prevalence estimates of HCC patients with FGF19 amplification or overexpression. In particular:

  • Blueprint scientists performed a meta-analysis of published data sets demonstrating that 7% of all HCC have a focal FGF19 amplification with a gene copy number increase greater than three-fold.
  • Blueprint scientists further expanded these findings by identifying 11% of HCC patient derived xenografts that showed similar focal FGF19 amplifications.
  • Blueprint scientists also demonstrated that more than 20% of samples show FGF19 overexpression, without genomic amplification of FGF19.
  • Overall, HCC patients with FGF19 amplification and overexpression could equal up to 30% of all HCC patients, regardless of race, geography, and etiology.

"Blueprint's early signs of anticancer activity in HCC preclinical models are very promising and generate new hope for liver cancer patients," commented Josep M. Llovet, M.D., Director of the Liver Cancer Program at the Icahn School of Medicine at Mount Sinai. "The ability to target HCC patients most likely to respond to therapy because of the genetic profile of their cancers is novel and provides the rationale for exploring proof-of-concept trials in this genomically defined patient population. Blueprint's small molecule inhibitor has potential as a single agent in enriched populations as first line therapy and also as a drug of choice in combination therapy. I am excited about the prospects of testing this compound in early clinical trials."

About Hepatocellular Carcinoma

Liver cancer is the sixth most common cancer and is the second most common cause of death from cancer worldwide. More than 780,000 cases of liver cancer are diagnosed worldwide each year.1 Hepatocellular carcinoma is the most common form of liver cancer and accounts for approximately 80 percent of the primary malignant liver tumors in adults.2

About Blueprint Medicines

Blueprint Medicines is a patient-driven oncology company developing highly selective kinase inhibitors for genomically defined cancer patients. Led by a management team and advisors with world renowned expertise in cancer genomics, drug discovery, and clinical oncology, Blueprint has developed a platform that combines genomics with a novel small molecule library of kinase inhibitors, enabling Blueprint to rapidly develop potent, highly selective compounds against clear genomic driver targets. Founded in 2011, Blueprint is privately held and initially backed by Third Rock Ventures and Fidelity BioSciences. For more information, please visit

1 Ferlay J, et al., GLOBOCAN 2012. Cancer Incidence, Mortality and Prevalence Worldwide in 2012.

2 El-Serag, Hashem, Current Concepts in Hepatocellular Carcinoma. The New England Journal of Medicine. 2011; 365: 1118-27.

CONTACT: Beth DelGiacco Stern Investor Relations, Inc. 212-362-1200 Beth@sternir.comSource: Blueprint Medicines