SOBI (SOBI) Status Update summary

Franchise and treatment profile

• Haemophilia revenue, including royalties, rose from SEK 1.1B in 2016 to SEK 7.7B in 2022; haemophilia represented 36% of 2022 revenue.

• Development and commercialisation rights for Alprolix, Elocta and efanesoctocog alfa are shared with Sanofi; rights cover Europe, North Africa, Russia and most Middle Eastern markets, with Sanofi holding rights elsewhere.

• Efanesoctocog alfa is a VWF-decoupled FVIII therapy designed for once-weekly prophylaxis; a single dose showed a 3–4x longer half-life than other FVIII products.

Clinical efficacy

• In XTEND-1, weekly prophylaxis reduced mean ABR 77% versus prior FVIII prophylaxis; model-based mean ABR was 0.71 and median ABR was 0.00.

• In XTEND-KIDS, estimated mean ABR was 0.89; 64% had no bleeds, 82% no joint bleeds and 88% no spontaneous bleeds.

• FVIII levels reached the non-haemophilia range for up to 4 days in adults and adolescents and up to 3 days in children.

• On-demand treatment resolved 97% of bleeding episodes with one injection; perioperative responses were rated excellent in 100% of cases.

Safety and patient outcomes

• No FVIII inhibitor development was detected; there were no reports of serious allergic reactions, anaphylaxis or vascular thrombotic events.

• Weekly prophylaxis improved joint health; pain intensity declined significantly by Week 52, and 81.5% of participants reported improved or maintained pain intensity.

• HRQoL and physical functioning improved versus prior FVIII prophylaxis; 71.4% maintained or improved their physical health score by Week 52.

• All 29 participants answering preference interviews selected efanesoctocog alfa over their previous FVIII prophylaxis.