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AstraZeneca's Phase III Trial Of Danicopan In Paroxysmal Nocturnal Haemoglobinuria Meets Goals

AstraZeneca Plc (AZN.L,AZN) said on Friday that its ALPHA phase III trial has met the primary endpoint and key secondary endpoints.

The ALPHA phase III trial is evaluating the company's investigational drug Danicopan as an add-on to C5 inhibitor therapy Ultomiris or Soliris in patients with paroxysmal nocturnal haemoglobinuria who experience clinically significant extravascular haemolysis.

The primary endpoint was the change in haemoglobin from baseline at 12 weeks and key secondary endpoints included transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score.

According to the prespecified interim analysis, Danicopan plus Ultomiris or Soliris demonstrated superiority compared to placebo plus Ultomiris or Soliris, with statistically significant and clinically meaningful improvements in haemoglobin levels, transfusion avoidance and FACIT Fatigue scores from baseline.

Paroxysmal nocturnal haemoglobinuria is a rare and severe blood disorder with the destruction of red blood cells, known as intravascular haemolysis, and white blood cell and platelet activation that can cause thrombosis. This may result in organ damage, leading to a potentially premature death.

Jong-Wook Lee, investigator at the ALPHA trial, said: "C5 inhibitors are a proven treatment option for patients living with PNH, yet a small percentage may continue to experience anaemia and burden of transfusion due to clinically significant EVH, however it is not life-threatening. These data show that danicopan has the potential to resolve clinically significant EVH while allowing patients to remain on standard of care treatment with Ultomiris or Soliris."

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