Blueprint Medicines Corporation (NASDAQ: BPMC) is a leader in discovering and developing targeted kinase medicines.
Physicians use Blueprint and other medicines to treat patients with genomically defined diseases.
Blueprint announced updated data from its ongoing Phase 1 clinical trial of what they call BLU-554. In particular, this is a potent and highly selective inhibitor of fibroblast growth factor receptor 4 (FGFR4). For example, oncologists use BLUE-554 in the treatment of patients with advanced hepatocellular carcinoma (HCC).
Especially relevant, BLU-554 demonstrated a 16 percent objective response rate (ORR) in patients with FGFR4-driven HCC. In addition, 49 percent of patients with FGFR4-driven HCC had radiographic tumor reduction.
“Patients with hepatocellular carcinoma face a very poor prognosis with few therapeutic options.” This according to Richard Kim, M.D., Associate Professor, Moffit Cancer Center. He served as an investigator for the study. Most noteworthy, “the new BLU-554 data show that in heavily pre-treated patients, BLU-554 demonstrated encouraging clinical activity. And approximately half of patients with FGFR4-driven HCC had tumor shrinkage.