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FDA Grants Blueprint Go-Ahead for Kinase Inhibitor Trials

Xconomy Boston — 

Blueprint Medicines got the green light from the Food and Drug Administration to begin clinical trials on its two lead drug candidates for cancer, about two months after its initial public offering.

The Cambridge, MA-based maker of a type of cancer drug called kinase inhibitors is using high-speed DNA sequencing instruments to test a homemade library of potential drugs, as Xconomy’s Ben Fidler wrote in April when Blueprint completed its IPO. Blueprint wants to develop compounds that block certain kinases—a family of hundreds of enzymes that can drive the activity of cancer cells—while avoiding others.

With the go-ahead from the FDA to begin Phase 1 studies on drug candidates BLU-285 and BLU-554, Blueprint is taking its first strides toward developing an entire portfolio.

BLU-285 targets the D816v mutation of the c-Kit gene, which plays a role in certain gastrointestinal tumors and in systemic mastocytosis, an abnormal buildup of mast cells, which normally help protect the body from disease. Blueprint’s trial for BLU-285 will focus on gastrointestinal tumors, and will first test the safety and tolerability of the drug on 60 patients to determine a maximum tolerated dose. (The company is on track to seek permission to begin trials of the drug for systemic mastocytosis as well.)

BLU-554 targets fibroblast growth receptor 4, which is implicated in a subset of patients with liver cancer. The company plans to enroll 60 patients in this trial as well, though 10 will be people who suffer from advanced, unresectable cholangiocarcinoma, a related cancer of the biliary duct, Blueprint said in a statement. The trial will also seek a maximum tolerated dose. Both trials have additional goals.

Blueprint, which was first formed in 2008 by Third Rock Ventures, said it plans to use the $147 million it raised in its IPO on the trials. It also plans to use $15 million it received as an upfront payment from Alexion Pharmaceuticals, the rare disease drug maker, to co-develop drugs. The deal could pay as much as $250 million in milestones and royalties.

Third Rock and an investment fund managed by Fidelity Biosciences invested $40 million in Blueprint in 2011 in a Series A deal, when the company got its current name. It received a $25 million Series B in January 2014, and another $50 million in a November Series C.

After the IPO, the Third Rock and Fidelity’s Beacon Bioventures Fund III still own the two largest positions in the company: 27.3 percent and 8.8 percent, respectively.