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Alnylam tumbles on ALN-CC5 update

May 4, 2016 1:42 AM UTC

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) fell $7.98 (12%) to $60.81 on Tuesday after reporting that it will narrow its development program for paroxysmal nocturnal hemoglobinuria (PNH) therapy ALN-CC5 to target poor responders to Soliris eculizumab from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) and PNH patients who require Soliris sparing. Alnylam had been developing the compound for a broader population of treatment-naive and Soliris-treated PNH patients.

The company expects to begin a Phase II trial by year end of the candidate to treat PNH in that population, and intends to begin trials early next year of ALN-CC5 to treat complement-mediated diseases such as atypical hemolytic uremic syndrome (aHUS) and myasthenia gravis, as monotherapy or in combination with mAbs against complement 5 (C5). ...