Apellis Pharmaceuticals revealed data from a late-stage clinical trial on Tuesday showing its experimental drug candidate appears to treat a rare blood condition better than a blockbuster Alexion drug.
The drug candidate, known as pegcetacoplan, takes aim at a genetic disease known as paroxysmal nocturnal hemoglobinuria, or PNH. People with the condition are missing certain protective proteins on red blood cells that would otherwise prevent their immune systems from attacking the cells. About 5,000 Americans have the condition.
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