Blueprint Medicines Receives FDA Authorization to Proceed with Clinical Trials for Two Drug Candidates
"We are delighted to reach this important milestone in the development of our two lead drug candidates to meet the urgent needs of patients facing a poor prognosis and few, if any, viable treatment options," said
BLU-554 in Advanced HCC and Cholangiocarcinoma
BLU-554 is a potent and selective inhibitor of fibroblast growth factor receptor 4 (FGFR4). Aberrant signaling of FGFR4 is a disease driver in up to 30 percent of HCC patients and may also play a role in a subset of patients with cholangiocarcinoma, a related cancer of the biliary duct. HCC is the most prevalent form of liver cancer. Liver cancer is the second-leading cause of cancer-related deaths worldwide.
In this Phase 1 trial,
The trial will test the safety and tolerability of escalating doses of BLU-554, with the goal of establishing a maximum tolerated dose (MTD), or a recommended dose if the MTD is not achieved. Once the MTD or recommended dose has been established, HCC and cholangiocarcinoma patients with aberrant FGFR4 signaling will be enrolled in disease-specific expansion cohorts. Additional study objectives include the assessment of early signs of biological and clinical activity using disease-specific biomarkers and response rate measurement according to Response Evaluation Criteria In Solid Tumors (RECIST).
BLU-285 in Unresectable and Treatment-Resistant GIST
BLU-285 is a potent and selective inhibitor of KIT Exon 17 and PDGFR-alpha D842V mutants, which are key disease drivers of metastatic and treatment-resistant GIST. GIST is the most common form of sarcoma of the gastrointestinal tract. Patients with the KIT Exon 17 and PDGFR-alpha D842V mutants are not adequately treated with existing therapies.
In this Phase 1 trial,
Various statements in this release concerning
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