Bayer HealthCare announced positive results from the Phase 3 PROTECT VIII trial evaluating BAY 94-9027 in previously treated adults and adolescents with severe hemophilia A. BAY 94-9027 is an investigational long-acting site-specific PEGylated recombinant human factor VIII compound.
PROTECT VIII (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII) is a multicenter, multinational, partially randomized, open-label trial with four treatment arms evaluating the safety and efficacy of BAY 94-9027 in previously treated adults and adolescents (n=134) with severe hemophilia A. All patients in the three prophylaxis arms began treatment with the site-specific PEGylated recombinant factor VIII twice per week. After a ten-week period, patients experiencing >1 bleed during this assessment period stayed on two infusions per week at a higher dose and all other patients were randomized to either every five- or seven-day treatment for six months.
The study met its primary objective of protection from bleeds with fewer infusions. Eighty eight percent of patients met the pre-defined criterion of bleeding control in the ten-week initial assessment period and qualified for randomization. All patients receiving infusion every five days (n=43) remained in this treatment arm.
In the every five-day treatment arm, 44% of patients experienced no bleeds. A median annualized bleeding rate (ABR) of 1.9 was observed in this treatment arm. Seventy four percent of the patients receiving infusion every seven days (n=43) remained in their treatment arm of which 37% experienced no bleeds. A median ABR of 3.9 (including non-completers) was observed in this treatment arm. The 13 patients who remained in the two times per week treatment arm, reduced their median ABR from 17.4 to 4.1. Patients who received on-demand treatment (n=20) had a median ABR of 23.
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