Antihemophilic factor
GLASGOW—Preliminary data suggest an investigational therapy can extend the half-life of factor VIII (FVIII) in patients with severe hemophilia A.
Researchers are testing the therapy, BIVV001, in a phase 1/2a trial and have reported results in 4 patients.
BIVV001 extended the half-life of FVIII to 37 hours, with an average FVIII activity of 5.6% at 7 days post-infusion.
“For decades, scientists have been trying to overcome the von Willebrand factor ceiling, which imposes a limit on the half-life of FVIII, and these data demonstrate that BIVV001 has finally broken through that ceiling,” said Joachim Fruebis, PhD, senior vice president of development at Bioverativ Inc.
Dr Fruebis presented these data at the World Federation of Hemophilia (WFH) 2018 World Congress during the late-breaking abstract session on Monday.
The research was sponsored by Bioverativ, the company developing BIVV001.
BIVV001 (rFVIIIFc-VWF-XTEN) is a recombinant FVIII therapy that builds on Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to potentially extend its time in circulation.
In the phase 1/2a EXTEN-A trial, researchers are evaluating the safety and pharmacokinetics of BIVV001 in a low-dose and high-dose cohort of subjects, ages 18 to 65, who have severe hemophilia A.
In the data presented at the WFH World Congress, 4 adult males received a single dose of recombinant FVIII therapy (25 IU/kg) followed, after a washout period, by a single, low dose of BIVV001 (25 IU/kg).
Primary endpoints of this study include the occurrence of adverse events and the development of inhibitors.
No inhibitors have been detected, and BIBV001 was “generally well-tolerated,” according to Bioverativ. The company did not provide additional safety information.
BIVV001 extended the half-life of FVIII to 37 hours, which is an increase over the 13 hours seen with recombinant FVIII.
The average FVIII activity for the 4 subjects was 13.0% at 5 days and 5.6% at 7 days post-infusion.
“Importantly for the hemophilia community, the factor levels seen in this study are unparalleled in hemophilia A,” Dr Fruebis said, “and we are excited about the potential for BIVV001 to transform the treatment paradigm for patients and physicians.”