Alexion partners with GNS, Sema4

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) is rebuilding its early stage pipeline with two new deals focused on driving rare disease discovery with big genomic data. The company partnered with Sema4 (New York, N.Y.) for genomic sequencing and licensed the Reverse Engineering and Forward Simulation (REFS) platform from GNS Healthcare Inc. (Cambridge, Mass.) to accelerate development of new rare disease therapeutics.

At the end of July, Alexion refocused its pipeline on the complement cascade, dropping 3 early stage partnerships and at least 6 early stage candidates. The moves left Alexion with a top-heavy portfolio, including its marketed complement 5 (C5) inhibitor Soliris eculizumab; and ALXN1210, a long-acting humanized mAb against C5 in Phase III testing for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) (see BioCentury, July 31)...