Alexion Pharmaceuticals Inc. said the phase 3 study of ALXN1210 to treat a rare blood disorder met its main and secondary goals.
Results from the trial showed the New Haven, Conn.-based biopharmaceutical company's investigational long-acting C5 complement inhibitor was as effective as Soliris, or eculizumab, in patients with paroxysmal nocturnal hemoglobinuria.
The C5 gene encodes a component of the complement system, a part of the immune system that plays a role in inflammation, host homeostasis and host defense against pathogens.
Paroxysmal nocturnal hemoglobinuria, or PNH, is a rare, acquired, life-threatening blood disease in which uncontrolled activation of the complement system, a part of the body's normal immune system, leads to the destruction of red blood cells.
The study showed that ALXN1210 helped patients avoid blood transfusion and normalized levels of lactate dehydrogenase, an enzyme that is involved in energy production in cells.
In addition, ALXN1210 was as effective as Soliris on all four secondary goals — reducing lactate dehydrogenase levels, improving quality of life, preventing hemolysis or the destruction of red blood cells and stabilizing hemoglobin levels.
ALXN1210 was found to be as safe as Soliris and was generally well-received, with the most frequent side effects being headache and heightened body temperature. A patient in the Soliris group died from lung cancer, although it was unrelated to the Soliris treatment, while two patients withdrew from the same group for unrelated reasons. No cases of meningococcal infection, a known risk with terminal complement inhibition, were reported in either ALXN1210 or Soliris groups.
Alexion intends to seek regulatory approval for ALXN1210 in the U.S., EU and Japan in the second half of 2018.
