Roche Pharmaceutical Development and Sales Overview

Hemlibra (emicizumab, RG6013) Factor VIII mimetic for treatment of hemophilia A Indication Hemophilia A patients without inhibitors to factor VIII Roche Hemophilia A patients with and without inhibitors to Factor VIII, dosing every 4 weeks Phase/study # of patients Phase III HAVEN 3 N=135 Patients on FVIII episodic treatment prior to study entry: ARM A: Hemlibra prophylaxis qw ☐ ARM B: Hemlibra prophylaxis q2w Phase III HAVEN 4 N=46 Multicenter, open-label, non-randomized study to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of Hemlibra administered every 4 weeks. ☐ Part I: Pharmacokinetic run-in part (N=6) Part II: Expansion part (N=40) Design Primary endpoint Status " ARM C: Episodic FVIII treatment; switch to Hemlibra prophylaxis possible after 24 weeks Patients on FVIII prophylaxis prior to study entry: ARM D: Hemlibra prophylaxis qw Number of bleeds over 24 weeks FPI Q3 2016, recruitment completed Q2 2017 Study met primary and key secondary endpoints Q4 2017 FDA granted Breakthrough Therapy Designation April 2018 Data presented at WFH 2018 Filed in US (priority review) and EU in Q2 2018 Data published in NEJM 2018; 379:811-822 Number of bleeds over 24 weeks FPI Q1 2017, recruitment completed Q2 2017 Pharmacokinetic run-in data at ASH 2017 Positive interim analysis outcome reported Q4 2017 Data presented at WFH 2018 Interim data filed in US and EU in Q2 2018 Data published in Lancet Haematology 2019 Jun;6(6):e295-e305 Approved in US Q4 2018 and EU Q1 2019 CT Identifier In collaboration with Chugai NCT02847637 ASH-American Society of Hematology; WFH-World Federation of Hemophilia; NEJM-New England Journal of Medicine NCT03020160 60 60 Hemophilia

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