A novel recombinant of human factor VII, marzeptacog alfa (activated) (MarzAA), is a subcutaneous injection engineered to increase duration of action and increase procoagulant activity in people with Hemophilia.
SUMMARIZED RESULTS: In the 11 subjects, the mean annualized bleed rate decreased from 19.6 to 1.6.
The Study: Men with severe congenital hemophilia with an inhibitor were enrolled in a phase 2 trial where all the subjects had a baseline annualized bleeding rate of twelve or more bleeds a year. To start, subjects were injected intravenously a single 18 μg/kg dose of marzeptacog alfa (MarzAA) which purpose was to a measure 24-hour pharmacokinetics and pharmacodynamics. Subjects also received a single 30 μg/kg SC dose which purpose was to measure 48-hour pharmacokinetics and pharmacodynamics. After those two administrations, subjects received SC 30 μg/kg marzeptacog alfa (MarzAA) daily, for 50 days.
In the case of spontaneous bleeding, the dosage administered was sequentially increased to either 60, 90, or 120 μg/kg, with 50 days at the final effective dose without spontaneous bleeding to then proceed to a 30-day follow-up. The study had a primary end point of a reduction in annualized bleed rate and its secondary end points were tolerability, safety, and anti-drug antibody development.
Results (in depth): In the 11 subjects, the mean annualized bleed rate decreased from 19.6 to 1.6. This means that the mean proportion of days with bleeding decreased from 12.3% to 0.8%. A total of 517 doses were administered with reports of six injection site reactions in two patients and a fatal unrelated case was also reported. There were no ADAs detected.
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