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news.Alexion Pharmaceuticals' investigational long-acting C5 complement inhibitor, ALXN1210, has succeeded in the pivotal phase 3 study of patients with rare blood disorder, paroxysmal nocturnal hemoglobinuria (PNH).
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The study showed that ALXN1210 achieved non-inferiority to Soliris (eculizumab) in complement inhibitor treatment-naïve patients with PNH based on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase (LDH) levels.
ALXN1210, which is developed by Alexion, will inhibit the C5 protein in the terminal complement cascade, a part of the body’s immune syste, when activated in an uncontrolled manner.
Soliris is a first-in-class complement inhibitor, which works by inhibiting the C5 protein in the terminal part of the complement cascade.
Alexion’s trial also showed non-inferiority on all four secondary endpoints, including percentage change from baseline in LDH levels, change from baseline in quality of life, proportion of patients with breakthrough hemolysis and proportion of patients with stabilized hemoglobin levels
The randomized, open-label, active-controlled, multinational, and multicenter trial assessed the efficacy and safety of ALXN1210 compared to Soliris administered by intravenous (IV) infusion to adult patients who are naïve to complement inhibitor treatment.
Alexion recruited 246 adult patients with a confirmed diagnosis of PNH who had never been treated with a complement inhibitor.
The drug is expected to play a crucial role in the treatment of severe ultra-rare disorders such as atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG), in addition to PNH.
ALXN1210 secured orphan drug designation to treat of patients with PNH in the US and EU, as well as for the subcutaneous treatment of patients with aHUS in the US.
Alexion executive vice president and R&D head Dr John Orloff said: “We are very pleased with these positive data for ALXN1210 in the first and only head-to-head study versus Soliris, and the results reinforce our ambition to establish ALXN1210 as the new standard of care for patients with PNH.”