AstraZeneca Plc AZN released data from the Phase 3 ALPHA trial of danicopan as an add-on to the standard of care for paroxysmal nocturnal hemoglobinuria (PNH).
The data showed that danicopan as an add-on to the standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) demonstrated a statistically significant and clinically meaningful increase in hemoglobin levels and maintained disease control in PNH patients, compared to placebo plus established C5 inhibitor therapy.
PNH 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).
In patients managing PNH with Ultomiris or Soliris, add-on treatment with danicopan was superior to placebo plus Ultomiris or Soliris based on the change in hemoglobin from baseline to week 12.
Further, significant improvements in hemoglobin were observed with danicopan by week two and maintained through week 12.
All key secondary endpoints also met statistical superiority in favor of danicopan plus Ultomiris or Soliris, compared to placebo plus C5 inhibition.
Results showed significantly more patients treated with danicopan (59.5%) versus placebo (0%) experienced an improvement in hemoglobin of ≥2 g/dL at week 12 in the absence of transfusion.
Additionally, significantly more patients treated with danicopan avoided transfusion through week 12 versus the comparator arm.
Results from this Phase 3 trial showed danicopan is generally well tolerated, and no new safety concerns were identified.
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