New clinical and real-world data from the ALPHA phase III trial evaluating danicopan, an investigational oral factor D inhibitor, used in the treatment of paroxysmal nocturnal hemoglobinuria (PNH), will be presented at the European Hematology Association 2023 Hybrid Congress that will take place from June 8 to 11, 2023, AstraZeneca announced.

The biopharmaceutical company will also showcase scientific findings across other rare hematological conditions.

“We look forward to presenting results at European Hematology Association from the ALPHA Phase III trial showing the potential for danicopan, as an add-on to Ultomiris or Soliris, to address clinically significant extravascular hemolysis, which can impact approximately 10% to 20% of PNH patients treated with C5 inhibitors, while allowing them to maintain treatment with standard of care,” said Gianluca Pirozzi, senior vice president, head of development, regulatory and safety, Alexion.

Alexion, AstraZeneca’s rare disease group, will deliver a presentation reporting the capacity of danicopan to improve signs and symptoms of clinically significant extravascular hemolysis in a small subset of patients with PNH who are affected. These patients had improved hemoglobin levels and needed fewer transfusions after taking the study drug.  

Read more about PNH experimental therapies

The data estimate the prevalence of clinically significant extravascular hemolysis in patients with PNH who are receiving C5 inhibitor therapy to range between 7% and 21% and provide insights into disease control and treatment satisfaction among patients and physicians.

In addition, the company will report positive findings on the effectiveness of acalabrutinib (Calquence) in the treatment of chronic lymphocytic leukemia, AZD0486, a CD19/CD3 T-cell engager in the treatment of relapsed or refractory follicular lymphoma, capivasertib, an AKT inhibitor, in the treatment of relapsed or refractory non-Hodgkin lymphoma, as well as AZD4573, a CDK9 inhibitor, in the treatment of peripheral T-cell lymphoma.


AstraZeneca showcases scientific advances across rare and malignant haematological conditions at EHA 2023. News release. AstraZeneca; May 16, 2023.