Alexion Vaults Ahead on Study Results

The biotech company's shares are up more than 20%.
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Alexion Pharmaceuticals

(ALXN) - Get Report

reported positive results from a late-stage trial of one of its lead drug candidates in a rare type of anemia, saying all of the prespecified primary and secondary endpoints were met and were statistically significant.

Shares of Alexion, a biotech firm based in Cheshire, Conn., surged on the news, rising $5.07, or 23.1%, to $27.02. Volume was six times the daily average for an entire session during the last three months.

The trial studied the effect of eculizumab in patients with paroxysmal nocturnal hemoglobinuria, a genetic blood disorder characterized by the destruction of red blood cells by part of the body's immune system. Currently, there is no therapy specifically available for treating the condition.

According to estimates, 8,000 to 10,000 people in North America and western Europe have it, Alexion said. The disease can cause anemia, chronic fatigue, pain, pulmonary hypertension and potentially life-threatening blood clots.

The main goals of the trial were to reduce the median transfusion rate of patients with the condition and to stabilize their hemoglobin over six months.

Alexion said the median transfusion rate was reduced from 10 units per patient getting the placebo to none for those receiving eculizumab. Hemoglobin stabilization was achieved by 49% of the eculizumab patients vs. none of the participants in the placebo arm of the study. The trial enrolled 87 patients at 45 sites in the U.S., Canada, Europe and Australia.

The company hopes to submit marketing applications for eculizumab as a treatment for paroxysmal nocturnal hemoglobinuria during the second half of the year. Alexion, which doesn't have any products on the market yet, plans to sell eculizumab as Soliris.