Research and practice in thrombosis and haemostasis
Denecimig (Mim8) prophylaxis over 52 weeks in adolescents and adults with hemophilia A: FRONTIER2 extension study
By Jiménez-Yuste V, Abraham A, Tomuleasa C and 10 others.
Abstract
Background
Denecimig (Mim8) is a fully human, activated factor VIII (FVIII) mimetic bispecific antibody in development for subcutaneous prophylaxis to prevent bleeding in persons with hemophilia A (HA).
Objectives
This study aimed to report the efficacy and safety of denecimig over 52 weeks in adolescents and adults from FRONTIER2 (NCT05053139).
Methods
Males and females (aged ≥12 years) with HA (any severity), with or without inhibitors, were enrolled. In the 26-week main phase, patients previously treated on-demand were randomized to continue on-demand treatment (arm 1) or receive denecimig injections once every week (QW; arm 2a) or once every month (QM; arm 2b); patients previously receiving clotting factor concentrate prophylaxis were randomized to receive denecimig QW (arm 3) or QM (arm 4). In the 26-week extension, patients in arm 1 switched to denecimig QW or QM; other arms followed the same dosing frequency. Annualized bleeding rate (ABR) of treated bleeds was estimated using a negative binomial model.