Half-Year 2022 Financial and Clinical Trials Update slide image

Half-Year 2022 Financial and Clinical Trials Update

Hemlibra Factor VIII mimetic for treatment of hemophilia A Indication Hemophilia A patients with and without inhibitors to Factor VIII Hemophilia A mild to moderate patients without inhibitors to Factor VIII Phase/study # of patients Phase III HAVEN 5 N=85 Phase III HAVEN 6 N=70 Roche • Design . Patients with Hemophilia regardless of FVIII inhibitor status on prophylactic or episodic treatment prior to study entry: . Arm A: Hemlibra prophylaxis qw Arm B: Hemlibra prophylaxis q4w • Arm C: No prophylaxis (control arm) Number of bleeds over 24 weeks Primary endpoint Status ■ FPI Q2 2018 Recruitment completed Q1 2019 Filed in China Q2 2020 ▪ Approved in China Q2 2021 Multicenter, open-label study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of Hemlibra in patients with mild or moderate Hemophilia A without FVIII inhibitors ☐ ☐ Hemlibra qw (1.5mg/kg), q2w (3.0mg/kg) or q4w (6.0mg/kg) (patients preference) Safety and efficacy ■ FPI Q1 2020 ☐ ☐ Recruitment completed Q1 2021 Interim data presented at ASH 2021 and primary data presented at ISTH 2022 ■ Filed in EU Q4 2021 CT Identifier NCT03315455 In collaboration with Chugai ASH-American Society of Hematology; ISTH=International Society on Thrombosis and Haemostasis NCT04158648 82 62 Hemophilia
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