Alexion and Cincinnati Children’s Launch Rare Disease Innovation Fund

July 1, 2014

Alexion Pharmaceuticals, Inc. and Cincinnati Children’s Hospital Medical Center today announced the establishment of a collaboration and fund for the advancement of research in rare disease. Cincinnati Children’s is a recognized center of excellence in pediatric rare diseases. Alexion is a global leader in the development and delivery of breakthrough therapies for patients with severe and life-threatening rare diseases. This collaboration leverages the collective expertise of a leading pediatric hospital with a leading biopharmaceutical company.

“Collaborations between industry and academia are important to drive innovation for patients suffering from rare diseases and we look forward to working with Cincinnati Children’s to advance our research programs which are focused on developing life-transforming therapies for patients with devastating disorders,” said Martin Mackay, Ph.D., executive vice president and Global Head of Research and Development at Alexion. “Our research and development team is continually working to understand the pathophysiology of severe and rare diseases so that we can identify and investigate potential breakthrough therapies. Working with an institution with the research experience and capabilities of Cincinnati Children’s will enhance our ability to accomplish this important goal.”

“As an institution that not only treats and cares for patients suffering from rare diseases but is also intensely interested in discovering potential treatments for them, we look forward to the opportunity to work with a company like Alexion, which brings tremendous expertise and capabilities to the table,” said Margaret Hostetter, MD, incoming chief medical officer of Cincinnati Children’s and director, Cincinnati Children’s Research Foundation. “Our hope is that the Alexion Rare Disease Innovation Fund can serve as a model for how industry and academia can better work together to bring health innovations from the bench to the bedside.”

Under the terms of the agreement, Alexion will have the opportunity to fund selected research programs through the Alexion Rare Disease Innovation Fund. Following completion of the funded research programs, Alexion will have an exclusive option to enter into a licensing agreement for these programs.

The Alexion fund is a focused opportunity within Cincinnati Children’s broader Innovation Fund, which covers all therapeutic areas and technology types. Alexion will focus its selections based on the company’s specialty of developing new treatments for complex and rare diseases.

The company decided to collaborate with Cincinnati Children’s because of the medical center’s strength in rare- and complex-disease research. Research programs selected for the Alexion fund will receive funding commensurate with project development needs and based on projects reaching specific milestones.

Upon entering into any subsequent licensing agreement, Cincinnati Children’s will be eligible to receive certain development and commercial milestone payments, as well as royalties on product sales from Alexion. Alexion will have the primary responsibility for further clinical development and commercialization of products arising from the collaboration. The collaboration and Innovation Fund are managed by Cincinnati Children’s Center for Technology Commercialization.

About Alexion:

Alexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris(R) (eculizumab) as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in nearly 50 countries for the treatment of PNH and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates, including asfotase alfa, across multiple therapeutic areas. This press release and further information about Alexion can be found at:

About Cincinnati Children’s:

Cincinnati Children’s Hospital Medical Center ranks third in the nation among all Honor Roll hospitals in U.S.News and World Report’s 2014 Best Children’s Hospitals. It is also ranked in the top 10 for all 10 pediatric specialties. Cincinnati Children’s, a non-profit organization, is one of the top three recipients of pediatric research grants from the National Institutes of Health, and a research and teaching affiliate of the University of Cincinnati College of Medicine. The medical center is internationally recognized for improving child health and transforming delivery of care through fully integrated, globally recognized research, education and innovation. Additional information can be found at Connect on the Cincinnati Children’s blog, via Facebook and on Twitter.

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