Rare disease magnate Alexion ($ALXN) is aligning itself with biotech Blueprint Medicines, borrowing the company's drug development technology to spotlight new treatments for genetic disorders.
Under the deal, Alexion will pay $15 million up front for the rights to an untold number of drug candidates with potential in treating an undisclosed rare disease. Alexion is on the line to reimburse all of Blueprint's research expenses as the company handles discovery and preclinical research, promising as much as $250 million more tied to developmental milestones. Once a Blueprint candidate reaches the IND stage, Alexion will take over R&D and commercialization responsibilities, per the agreement.
At the heart of the deal is Blueprint's product engine, which uses genomic profiling to match compounds from a library of highly selective kinase inhibitors with the diseases they're best suited to treat, thereby crafting personalized treatments for patients. The biotech's in-house work has thus far focused on oncology, but Alexion believes its core technology can easily pivot to the genetic disease space.
|Alexion R&D chief Martin Mackay|
"Blueprint Medicines' unique discovery platform enables it to create drug candidates for extremely challenging kinase targets," Alexion R&D chief Martin Mackay said in a statement. "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."
Blueprint, a brainchild of Third Rock Ventures, recently closed a $50 million C round to bankroll its proprietary work. With the money, the Cambridge, MA, biotech plans to get its top two candidates into the clinic by the end of this year, focusing on BLU-285, designed to treat mastocytosis and stomach tumors, and BLU-554, tailored for a certain type of hepatocellular carcinoma. The goal is to start dosing patients with both drugs next year, quickly establishing proof-of-concept and then rolling into Phase II, the company said.
The company, a 2011 Fierce 15 honoree, has raised about $115 million in venture cash since its foundation. Jeffrey Albers, who ran U.S. operations at Algeta before Bayer bought it for $2.9 billion, came aboard as CEO last year, bringing the experience Blueprint's founders say will shepherd the biotech toward clinical success.
- read the statement
Special Report: FierceBiotech's 2011 Fierce 15 - Blueprint Medicines