CStone Pharmaceuticals and Blueprint Medicines Corporation announced the dosing of the first patient in a Phase 1b/2 trial evaluating fisogatinib in combination with CS1001 for the treatment of locally advanced or metastatic hepatocellular carcinoma (HCC). The study will be conducted across multiple clinical sites in China, where according to GLOBOCAN data, nearly half of the world's new HCC cases occurred in 2018. This trial will assess the potential for two complementary treatment approaches – precision therapy and immuno-oncology therapy – to enhance anti-tumor activity in locally advanced or metastatic HCC.

Discovered by Blueprint Medicines, fisogatinib is an investigational, potent and highly selective inhibitor of fibroblast growth factor receptor 4 (FGFR4) in clinical development for the treatment of advanced FGFR4-driven HCC. CS1001 is an investigational anti-PD-L1 monoclonal antibody being developed by CStone for multiple tumor types. Preclinical studies in an HCC model have shown that treatment with fisogatinib stimulated T-cell infiltration into the tumor microenvironment, suggesting that combining fisogatinib with a PD-L1 inhibitor may enhance anti-tumor activity in patients with FGFR4-driven HCC.

Blueprint Medicines and CStone Pharmaceuticals have an exclusive collaboration and license agreement for the development and commercialization of fisogatinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for these drug candidates in the rest of the world.