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A blueprint for new therapies
May 2015
by Lori Lesko  |  Email the author
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CAMBRIDGE, Mass.—Targeted toward ultimately treating rare genetic diseases with an investigational new drug, the preclinical-stage biotech Blueprint Medicines has launched a strategic joint agreement with Alexion Pharmaceuticals to discover, develop and commercialize new drug candidates for an undisclosed activated kinase target—the cause of a rare genetic disease.
 
The collaboration, announced March 3, stipulates that Blueprint Medicines will receive $15 million up front from Connecticut-based Alexion, plus potentially $250 million in milestone payments, as well as royalties on any approved drugs. Blueprint received $50 million in Series C funding in November 2014, and has now raised more than $130 million in venture funding since it was launched by Third Rock in 2011.
 
The collaboration combines Blueprint Medicines’ kinase-focused drug discovery platform with Alexion’s experience in developing and commercializing therapies for severe and life-threatening disorders, and is aimed at filing an Investigational New Drug (IND) application with the U.S. Food and Drug Administration.
 
Blueprint Medicines is expected to conduct all research activities up until filing the IND application, after which Alexion will take responsibility for the development and commercialization of Blueprint’s drug candidates.
 
“Our kinase-focused platform, which integrates a novel target discovery engine and a proprietary compound library, enables us to craft highly selective kinase drugs for genomic drivers of disease across many therapeutic areas,” Jeffrey Albers, CEO of Blueprint Medicines, stated in a news release. “Alexion is the ideal partner for our rare genetic disease program with its successful track record developing and commercializing therapies for severe and life-threatening disorders.”
 
“Working with Alexion on this target will allow the team at Blueprint Medicines to focus on our primary strategic area of oncology, while we leverage our platform in additional therapeutic areas,” Albers added.
 
Blueprint Medicines’ kinase inhibitors block proteins from a type of gene alteration, a method widely used in cancer drugs. While neither Blueprint Medicines nor Alexion are revealing much about the drugs or specific diseases targeted by the collaboration, Albers says his company’s drugs can apply to “genomic drivers of disease across many therapeutic areas.”
 
Martin Mackay, executive vice president and global head of research and development at Alexion, stated, “Blueprint Medicines’ unique discovery platform enables it to create drug candidates for extremely challenging kinase targets. 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 was thrust onto the front page of pharmaceutical news in 2011 when its approved drug Soliris, a treatment for an ultra-rare, life-threatening blood disorder called paroxysmal nocturnal hemoglobinuria, received the distinction of being the world’s most expensive drug—at the cost of more than $400,000 per year.
 
Alexion is the original developer and distributor of Soliris (eculizumab), and the company notes that “Today, Soliris is approved in nearly 50 countries as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), and in nearly 40 countries as a treatment for patients with atypical hemolytic uremic syndrome (aHUS). Both PNH and aHUS are life-threatening, ultra-rare diseases that are caused by chronic uncontrolled complement activation.”
 
Alexion’s current development programs span several therapeutic areas, including hematology, nephrology, transplant, neurology, metabolic disorders and inflammatory disorders.
 
In addition to inking the collaboration with Blueprint Medicines, Alexion has opened a new laboratory in Cambridge, Mass., to support the research and development team focused on the company’s messenger RNA (mRNA) platform.
 
The new laboratory will house scientists whose capabilities include cell biology, biochemistry and molecular biology, all focused on targeted delivery of mRNA therapeutics to address rare diseases caused by protein deficiencies, Alexion reported.
 
Mackay stated, “Alexion’s growing footprint in Cambridge will facilitate the engagement of the best academic and biotech scientists and collaborators in the Boston area, enabling us to leverage innovative technologies to better serve our patients.” Blueprint Medicines is also located in Cambridge.
 
DDNews reached out to Blueprint Medicines for a comment on the latest news, but was told April 23 by Alina Tuttle-Melgar of public relations firm Chandler Chicco Companies that Blueprint Medicines “is currently in a quiet period” and, therefore, would not be commenting to the media.
 
Code: E051506

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