Strong Phase III data for concizumab in hemophilia A or B

Phase III results of the explorer7 study, assessing the efficacy and safety of prophylactic treatment with concizumab in people living with hemophilia A or B with inhibitors were presented on Sunday at the International Society of Thrombosis and Hemostasis Annual Congress (ISTH 2022) in London, UK.

Under development at Danish diabetes care giant Novo Nordisk (NOV: N), concizumab is an anti-tissue factor pathway inhibitor (TFPI) antibody in development for once-daily prophylactic treatment (regular treatment to prevent prolonged and spontaneous bleeding) by administration under-the-skin for all types of hemophilia. The use of concizumab is investigational and not approved by regulatory authorities.

The results showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean ABR of 1.7 compared to 11.8 with no prophylaxis, fulfilling the primary objective. The overall median ABR of concizumab was zero, compared to 9.8 for no prophylaxis. 21 (63.6%) people on concizumab experienced no treated bleeds, compared to two (10.5%) on no prophylaxis. The safety and tolerability profile of concizumab in this study was within the expected range, with no thromboembolic events reported after treatment restart.