Blueprint Medicines Says Data on BLU-554 Show 16% Objective Response Rate in Hepatocellular Carcinoma

Blueprint Medicines (BPMC) said Monday an ongoing phase 1 clinical trial of BLU-554 demonstrated a 16% objective response rate in patients with FGFR4-driven advanced hepatocellular carcinoma (HCC).

In addition, 49% of patients with FGFR4-driven HCC had radiographic tumor reduction. The company said BLU-554 was well-tolerated and most adverse events reported by investigators were grade 1 or 2. Median progression free survival was 3.7 months among patients with FGFR4-driven HCC.

The company said it plans to continue to enroll and follow the cohort of patients with FGFR4-driven HCC in the ongoing phase 1 clinical trial to further evaluate the safety and clinical activity of BLU-554 in this population. In addition, the company plans to initiate an additional cohort in this clinical trial in the first quarter of 2018 to evaluate BLU-554 in TKI-naïve patients with FGFR4-driven HCC. Blueprint Medicines also plans to explore opportunities to conduct a clinical trial to evaluate BLU-554 in combination with an immune checkpoint inhibitor.

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