Positive top-line results from Phase III study of Ultomiris in aHUS

US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) announced that the Phase III study of Ultomiris (ravulizumab-cwvz), the company’s long-acting C5 complement inhibitor, met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (aHUS).

In the initial 26 week treatment period, 53.6% of patients (95% CI [39.6%, 67.5%]) demonstrated complete thrombotic microangiopathy (TMA) response. Ultomiris provided immediate and complete inhibition of the complement C5 protein that was sustained over the entire eight-week dosing interval.

If eventually approved for the aHUS indication, Ultomiris will compete with Alexion’s own drug Soliris (eculizumab) estimated sales of almost $3.5 billion last year.