Sanofi-SOBI Hemophilia Candidate Aces Late-Stage Study In Pretreated Patients

Swedish Orphan Biovitrum AB BIOVF and Sanofi SA 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.