AstraZeneca PlcAZN said its Danicopan add-on anti-blood-clotting drug met a primary endpoint in the ALPHA phase 3 trial in patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH).
A prespecified interim analysis of the Phase 3 trial evaluating danicopan (ALXN2040) as an add-on to C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) showed positive results.
Danicopan plus Ultomiris or Soliris demonstrated superiority compared to placebo plus Ultomiris or Soliris, with statistically significant and clinically meaningful improvements in hemoglobin levels, transfusion avoidance, and fatigue scores from baseline.
PNH is a rare blood disorder characterized by the destruction of red blood cells and white blood cells & platelet activation, causing blood clots and resulting in organ damage and potentially premature death.
Danicopan was generally well tolerated, and no clinically meaningful differences in safety results were observed between the danicopan plus C5 inhibitor group and the control group.
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