AstraZeneca fleshes out pivotal data on $930M rare blood disorder drug it wants to pair with Soliris, Ultomiris
AstraZeneca says its add-on pill for a rare blood disorder known as paroxysmal nocturnal hemoglobinuria, or PNH, helped patients already taking Ultomiris or Soliris produce more hemoglobin than either approved drug alone.
In an interim cut of data of the ALPHA study, 63 patients with PNH who experience symptoms related to extravascular hemolysis — when blood cells are destroyed outside blood vessels — saw their hemoglobin levels increase by an average of 2.94 g/dL on danicopan and either Ultomiris or Soliris compared to 0.50 g/dL in those patients taking Ultomiris or Soliris and placebo at 12 weeks.
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