Swedish Orphan Biovitrum AB (OTC:BIOVF) and Sanofi SA (NASDAQ:SNY) have announced topline results from the XTEND-1 phase 3 study of efanesoctocog alfa (BIVV001) in previously treated severe hemophilia A patients aged 12 and above.
- The study met the primary endpoint, showing clinically meaningful prevention of bleeds in patients receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks.
- The median annualized bleeding rate (ABR) was 0 with a mean ABR of 0.71.
- The key secondary endpoint was also met, showing once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy.
- The data showed a statistically significant reduction in ABR based on the intra-patient comparison.
- Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events were headache, arthralgia, fall, and back pain.
- The data will be the basis for submission to regulatory authorities expected to start in 2022.
- Submission in the EU will follow the availability of data from the ongoing XTEND-Kids pediatric study, expected in 2023.
- Price Action: SNY shares are up 2.27% at $51.34 during the premarket session on the last check Wednesday.
