Danicopan (ALXN2040) Add-On to ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) Met Primary Endpoint in ALPHA Phase III Trial for Patients with Paroxysmal Nocturnal Hemoglobinuria Who Experience Clinically Significant Extravascular Hemolysis

Interim results demonstrate statistically significant improvement compared to placebo in hemoglobin levels from baseline to week 12

Excerpt from the Press Release:

WILMINGTON, Del.–(BUSINESS WIRE)–A prespecified interim analysis of the ALPHA Phase III trial evaluating danicopan (ALXN2040), an investigational, oral factor D inhibitor, as an add-on to C5 inhibitor therapy ULTOMIRIS^® (ravulizumab-cwvz) or SOLIRIS^® (eculizumab) showed positive high-level results in patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH).

The trial met its primary endpoint of change in hemoglobin 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 SOLIRISfor this specific patient population, with statistically significant and clinically meaningful improvements in hemoglobin levels, transfusion avoidance and FACIT Fatigue scores from baseline.

PNH is a rare and severe blood disorder characterized by the destruction of red blood cells, known as intravascular hemolysis (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

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