- The New England Journal of Medicine published positive final results from the Phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe hemophilia A. Sanofi (NASDAQ:SNY) and Sobi have collaborated on the development of BIVV001.
- BIVV001 is an investigational factor VIII therapy designed to provide higher bleed protection in a once-weekly prophylactic treatment regimen.
- EXTEN-A is an open-label study that evaluated the safety, tolerability and pharmacokinetics of BIVV001 in both a 25 IU/kg (n=6) and a 65 IU/kg (n=8) dose cohort of participants aged 19-63 years with severe hemophilia A.
- Primary endpoints included occurrence of adverse events and development of inhibitors. Key findings included:
- BIVV001 was generally well tolerated with no inhibitor development detected through 28 days post-dose. No adverse events were reported.
- In the 65 IU/kg dose cohort, a single dose of BIVV001 achieved a FVIII half-life of 43 hours, a greater than three-fold increase from the 13-hour half-life observed with rFVIII. Mean factor VIII activity level was ≥51% and in the normal range for four days, and 17% at seven days post BIVV001 infusion.
- In the 25 IU/kg cohort, BIVV001 achieved a FVIII half-life of 38 hours, a four-fold increase from the 9-hour half-life observed with rFVIII, with a mean factor activity level of 5% at seven days post BIVV001 infusion.
- Factor activity levels refer to the amount of factor VIII in a person’s blood and are used to determine the severity of a person’s disease.