Alexion Pharmaceuticals Inc., headquartered in New Haven, Connecticut, announced positive results from its pivotal Phase III clinical trial of ALXN1210 in paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare, acquired, life-threatening blood disease where the red blood cells are destroyed by part of the body’s immune system known as the complement system. It is also considered the only hemolytic anemia that is acquired, as opposed to inherited, due to a specific defect in the cell membrane.

ALXN1210 is a long-acting C5 complement inhibitor. In the trial, it was compared to Alexion’s Soliris (eculizumab) in treatment-naïve PNH patients. The co-primary endpoints were avoidance of transfusions and normalization of lactate dehydrogenase (LDH) levels, which is a direct marker of complemented-mediated hemolysis in the disease. ALXN1210 also showed non-inferiority for all four key secondary endpoints.

The safety profiles of the two drugs were reported to be basically the same. The most common side effects were headache and pyrexia, which is a fever. During the trial, one patient in the Soliris arm died from lung cancer, unrelated to the treatment. Two patients dropped out of the Soliris arm of the trial for unrelated reasons. Isolated cases, one for each arm of the trial, of anti-drug antibody were observed.

"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," said John Orloff, Alexion’s executive vice president and head of Research & Development, in a statement. "The data are also consistent with our hypothesis that immediate, complete, and sustained C5 inhibition is critical for patients with this potentially life-threatening disease. Soliris has established a high bar for efficacy. Achieving non-inferiority on both co-primary and all key secondary endpoints, as well as seeing numeric results in favor of ALXN1210, in such a rigorous study met a very high hurdle. We look forward to regulatory submissions of ALXN1210 in PNH in the U.S., EU, and Japan in the second half of 2018."

Investors responded accordingly, and shares climbed 11 percent in premarket trading today.

Proactive Investors writes, "Essentially, the company wanted to prove that its new drug is just as effective and safe as its existing one, which it did. PNH patients currently receiving Soliris need 26 infusions a year, but one of the key benefits of ALXN1210 is that it only needs to be administered six times a year."

"Having a new treatment option that achieves transfusion avoidance, and provides rapid and sustained normalization of LDH levels when administered six times a year could be a meaningful improvement for patients with PNH who currently need 26 infusions per year," said Jong Wook Lee, professor of Internal Medicine, Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, Korea, and an investigator in the study, in a statement

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