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Blueprint Medicines Inks Deal with Alexion Targeting Rare Genetic Disease

NEW YORK (GenomeWeb) – Blueprint Medicines today announced a collaboration with Alexion to discover, develop, and commercialize novel drug candidates for an unnamed, kinase-related, rare genetic disease. An activated kinase target, which was also not named, is the cause of the disease.

Under the terms of the agreement, Blueprint Medicines will receive an upfront payment of $15 million and will be reimbursed for all research expenses. The firm is also eligible to receive over $250 million in payments upon reaching pre-specified preclinical, clinical, regulatory, and commercial milestones. In addition, it will be eligible to receive royalty payments following commercialization of the product.

In addition to using its kinase-focused drug discovery platform to identify and optimize drug candidates, Blueprint will perform all research activities prior to the filing of an investigational new drug application with the US Food and Drug Administration, it said. Alexion will develop and commercialize drug candidates identified by Blueprint.

"Even in these early stages, Blueprint Medicines' compounds show impressive selectivity toward the mutant kinase, thereby sparing other kinases and delivering drug to the specified target," Alexion Executive Vice President and Global Head of R&D Martin Mackay said in a statement.

Blueprint's proprietary platform combines genomics expertise with a library of kinase inhibitors for the development of potent and highly selective compounds, according to the company's website. It said it has raised $115 million in financing since its founding in 2011.

In 2013, the Cambridge, Mass.-based firm teamed with Personal Genome to find and develop kinase-inhibiting cancer treatments.