AstraZeneca: Long-term ALPHA trial data shows sustained clinical improvements for PNH patients with danicopan
Long-term data from AstraZeneca's ALPHA Phase III trial has shown that danicopan, as an add-on to Ultomiris or Soliris, sustained clinical benefits in a subset of PNH patients experiencing clinically significant extravascular haemolysis.
AstraZeneca has announced new long-term data from its pivotal ALPHA Phase III trial, evaluating the efficacy and safety of danicopan as an add-on therapy. The trial focused on patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience clinically significant extravascular haemolysis (EVH).
Results presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition showed that danicopan, administered alongside Ultomiris or Soliris, demonstrated sustained clinical benefits. Improvements in mean haemoglobin levels and absolute reticulocyte count (ARC) observed earlier in the trial were maintained through 48 weeks.
PNH is a rare blood disorder characterized by red blood cell destruction. While C5 inhibitor therapies like Ultomiris and Soliris effectively manage intravascular haemolysis (IVH), an estimated 10-20% of patients experience clinically significant EVH, leading to symptoms such as anaemia and potential transfusion needs.
Danicopan is an investigational, first-in-class oral Factor D inhibitor. The ALPHA trial data suggests that dual inhibition of the complement pathway, targeting both Factor D and C5, may offer an optimal treatment approach for this specific patient subset, ensuring continued IVH control while addressing EVH symptoms.