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Danicopan Phase III trial met primary endpoint
Danicopan Phase III trial met primary endpoint.
About this update from Astrazeneca Plc
[{"type":"text","content":"\n \n \n 16 September 2022 07:10 BST\n \n \n \n \n \n \n \n \n Danicopan (ALXN2040) add-on to Ultomiris or Soliris met primary endpoint in ALPHA Phase III trial for patients with paroxysmal nocturnal haemoglobinuria who experience clinically significant extravascular haemolysis\n \n \n \n \n \n \n \n \n \n Interim results demonstrate statistically significant improvement compared to placebo in haemoglobin levels from baseline to week 12\n \n \n \n \n \n \n \n \n A prespecified interim analysis of the ALPHA Phase III trial evaluating danicopan (ALXN2040),\n an investigational, oral factor D inhibitor, as an add-on to C5 inhibitor therapyUltomiris(ravulizumab) or Soliris (eculizumab) showed positive high-level results \n in patients with paroxysmal nocturnal haemoglobinuria (PNH) who experience clinically significant extravascular haemolysis (EVH).\n \n \n \n \n \n The trial met its primary endpoint of change in haemoglobin from baseline at 12 weeks and key secondary endpoints, including transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score. Danicopan plus Ultomiris or Soliris demonstrated superiority compared to placebo plus Ultomiris or Soliris for this specific patient population, with statistically significant and clinically meaningful improvements in haemoglobin levels, transfusion avoidance and FACIT Fatigue scores from baseline. \n \n \n \n \n \n \n \n PNH is a rare and severe blood disorder characterised by the destruction of red blood cells, known as intravascular haemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.1-3\n \n \n \n \n \n Marc Dunoyer, Chief Executive Officer, Alexion, said\n : \"Alexion has relentlessly innovated for the PNH community, pioneering with Soliris, the first treatment for PNH, and establishing Ultomiris as a standard of care. We are proud of our continued innovation to advance new ways of targeting the complement cascade to help address the needs of patients living with this debilitating disease. These are the first positive Phase III results for an oral factor D inhibitor and demonstrate the potential for danicopan add-on therapy to improve signs and symptoms and reduce the need for trans...