Shares of AstraZeneca (NASDAQ: AZN) were on an upswing in pre-market trading on Friday as its investigational drug danicopan (ALXN2040) showed positive results in an ALPHA Phase III trial.
Danicopan (ALXN2040) is an investigational, oral factor D inhibitor that showed positive high-level results in patients with paroxysmal nocturnal haemoglobinuria (PNH). PNH is an acquired, rare life-threatening disease of the blood which causes the destruction of red blood cells.
The interim Phase 3 ALPHA trial indicated that Danicopan plus Ultomiris or Soliris met its primary endpoint with “statistically significant and clinically meaningful improvements in haemoglobin levels, transfusion avoidance and FACIT [ Functional Assessment of Chronic Illness Therapy] Fatigue scores from baseline.”
Marc Dunoyer, CEO, Alexion commented, “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 transfusions for the limited proportion of people living with PNH who experience clinically significant EVH [extravascular haemolysis].”
In other news, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommended AZN’s Evusheld, a long-acting antibody cocktail for the treatment of COVID-19 in adults and adolescents “who do not require supplemental oxygen and who are at increased risk of progressing to severe COVID‑19.”
The European Union (EU) also gave a marketing authorization for AstraZeneca’s Beyfortus (nirsevimab) for the prevention of lower respiratory tract disease.
