It’s been a year of transition for Blueprint Medicines. Since early 2014, the Cambridge, MA-based company moved on from its founding CEO, named two lead programs, added a whole bunch of new investors, and raised a total of $75 million.
Now it’s getting its first stamp of approval from a large drugmaker—Alexion Pharmaceuticals.
Blueprint and Cheshire, CT-based Alexion (NASDAQ: ALXN), the rare disease drug maker, cut a deal today to co-develop and sell drug candidates aimed at an unspecified kinase target that causes an unnamed genetic disease. Blueprint will get $15 million up front in the deal, with $250 million in downstream payments tied to various clinical and commercial milestones, as well as royalties on any sales.
Blueprint will handle the research work and be reimbursed for the costs, and then hand things over to Alexion when it’s time to begin the clinical trial work.
The deal marks Blueprint’s first strategic collaboration since its inception in 2011, when Third Rock Ventures and Fidelity Biosciences poured a $40 million Series A into the company, formed around a scientific trio (Nick Lydon, Brian Druker, and Charles Sawyers) who helped develop the landmark targeted cancer therapy imatinib (Gleevec). Including the $75 million it raised last year, Blueprint has hauled in a total of $115 million to date.
Blueprint uses high-speed DNA sequencing instruments to help develop precisely targeted cancer drugs that hit specific kinase enzymes while avoiding others. The company has built an in-house library of kinase inhibitors. The idea is that Blueprint scientists find new genetic drivers of cancer, and can then quickly test how these various drug prospects fare against them.
“[Blueprint’s] unique discovery platform enables it to create drug candidates for extremely challenging kinase targets,” Alexion research chief Martin Mackay said in a statement. “Even in these early stages, Blueprint’s compounds show impressive selectivity toward the mutant kinase, thereby sparing other kinases and delivering drug to the specified target.”
Blueprint hasn’t tested any of its drug candidates in human patients as of yet; CEO Jeffrey Albers told me a few months ago that the company intends to file papers with the FDA to begin its first trials in the first half of this year. The company’s lead programs target the D816v mutation of the c-Kit gene, which plays a role in systemic mastocytosis and some gastrointestinal stromal tumors; and what’s known as fibroblast growth receptor 4 (FGFR4), which is implicated in a subset of patients with liver cancer.
The Alexion deal comes at a time when Blueprint seems headed for an IPO. Last year, the company switched CEOs twice: founding CEO Chris Varma was replaced on an interim basis by Third Rock Ventures partner Alexis Borisy, who was in charge for several months before Blueprint brought in Albers, a former Algeta executive, in July. A few months later, in November, Blueprint raised $50 million in a round that had a group of “crossover” investors—hedge funds or mutual funds that typically invest in public companies. That’s typically a sign that a company is prepping for public offering.
“We’re obviously thinking of the next step,” Albers told me in an interview a few months ago.