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FDA Clears Biotech Cancer Therapies for Early Trials

Liver illustration

(Mikael Häggström, Wikimedia Commons)

13 July 2015. Biotechnology company Blueprint Medicines says U.S. Food and Drug Administration accepted new drug applications on its two lead products, clearing the way for trials with humans. The Cambridge, Massachusetts enterprise is developing cancer therapies, with its lead products targeting liver and gastrointestinal tumors.

Blueprint develops cancer therapies that limit the actions of kinases, enzymes supporting cancer growth resulting from genomic mutations. The company’s technology is designed to generate treatments for patients that address their precise genomic alterations, with a library of kinase inhibitors referencing some 200 kinases. The company says these kinase inhibitors can be building blocks for powerful cancer-fighting medications.

FDA accepted Investigational New Drug Applications for Blueprint’s  lead drug candidates that up to now were in preclinical testing. The company’s therapy code-named BLU-554 limits activity of mutations generating the enzyme fibroblast growth factor receptor 4, associated with hepatocellular carcinoma, a common type of liver cancer. Tests of BLU-554 reported at the International Liver Congress in April 2015, show that mice induced with hepatocellular carcinoma from amplified fibroblast growth factor receptor 4 had complete tumor remission when treated with the highest doses of BLU-554.

Blueprint is planning an early-stage clinical trial of BLU-554 with 50 participants having either advanced inoperable hepatocellular carcinoma or cholangiocarcinoma, a related cancer of the biliary duct, where fibroblast growth factor receptor 4 is also implicated. The trial is expected to recruit participants at sites in the U.S., Europe, and Asia.

FDA also approved a new drug application for Blueprint’s candidate code-named BLU-285 therapy that aims to block kinases released by mutations in a gene (KIT exon 17) associated with gastrointestinal stromal tumors or GISTs. Preclinical studies, also reported in April 2015, show the highest doses of BLU-285 resulted in significant tumor regression in mice induced with gastrointestinal stromal tumors and prevented regrowth of the tumors for 28 days.

Blueprint says it plans early-stage clinical trials of BLU-285 enrolling 60 individuals with GISTs or related tumors at sites in the U.S., Europe, and Asia. In both cases, the trials will look primarily for safety and tolerability of the therapy candidates, with the goal of establishing a maximum tolerable dose or recommended dose. The studies will also look for biological activity in participants, as indicated by disease-specific biomarkers, as well as evidence of efficacy based on standard criteria for evaluating responses to solid tumor therapies.

The company was founded in 2011 by biotechnology industry veterans and entrepreneurs, with backing from venture capital company Third Rock Ventures. Blueprint went public at the end of April 2015.

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