AstraZeneca : Phase III Trial Data Show Danicopan Sustained Clinical Improvements In PNH

(RTTNews) - AstraZeneca (AZN, AZN.L) said that positive results from the 24-week and long-term extension period of the pivotal ALPHA Phase III trial showed danicopan as add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab-cwvz) or Soliris (eculizumab) continued to demonstrate clinical benefit for patients with paroxysmal nocturnal hemoglobinuria or PNH who experience clinically significant extravascular hemolysis or EVH.

Data showed that improvements in mean hemoglobin levels and absolute reticulocyte count (ARC) levels, which were demonstrated at 12 weeks, were maintained through 48 weeks.

Secondary endpoints measured at 24 weeks include change from baseline in hemoglobin, ARC, and lactate dehydrogenase (LDH) levels; the percentage of patients with hemoglobin increase of =2 g/dL in the absence of transfusion; and the percentage of patients with transfusion avoidance.

All key secondary endpoints met superiority in favor of danicopan plus ULTOMIRIS or SOLIRIS compared to placebo plus ULTOMIRIS or SOLIRIS at 12 weeks, and data showed benefits were maintained at 24 weeks in the danicopan-danicopan arm.

Paroxysmal nocturnal hemoglobinuria is a rare and severe blood disorder characterized by the destruction of red blood cells within blood vessels, known as intravascular hemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.

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