Biogen Idec drugs could advance treatment of hemophilia, clinical trials find

Results of phase-3 hemophilia trials published in Blood, NEJM

NEW YORK — Two experimental drugs under development by Biogen Idec are effective in reducing bleeding episodes in patients with two forms of hemophilia when administered as a preventive treatment, while also reducing the need for frequent injections, according to late-stage clinical trial data.

The two phase-3 studies were published in the November issue of the journal Blood and the December issue of the New England Journal of Medicine. The Blood trial, titled "A-LONG," tested Biogen Idec's recombinant factor VIII Fc fusion protein in patients with hemophilia A, while the NEJM trial, titled "B-LONG", tested recombinant factor IX Fc fusion protein in patients with hemophilia B.

"Results from these largest global phase-3 pivotal trials demonstrate the potential of revolutionizing the hemophilia treatment paradigm by offering longer protection from bleeding episodes while reducing the frequency of intravenous injections," clinical lead Alvin Luk told DSN.

The hemophilia A trial divided 165 patients into three groups, one of which received a dose of the drug every 3-5 days per week on a preventive basis, known as prophylaxis; another prophylaxis group that received a dose every week; and another that received the drug on an as-needed basis as bleeding episodes were occurring. Any patients in any group could use the drug for major surgeries. Patients who received prophylaxis had significantly reduced bleeding rates compared with those who received it on an episodic basis.

The 123 hemophilia B patients were divided into a group that received the weekly prophylaxis; a second group that received interval-adjusted prophylaxis every 10 days or longer; a third group that received treatment as needed; and a fourth that received it for surgical procedures. In the first, second and third groups, 90.4% of bleeding episodes were resolved after one injection, and the responses were good or excellent among patients undergoing surgery.


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