Roche Pharmaceutical Development and Sales Overview

Hemophilia A Unique gene therapy platform Molecule Indication Phase/study # of patients Design SPK-8011 (RG6357) Spark THERAPEUTICS TM SPK-8016 (RG6358) Roche Phase I N=100 Hemophilia A Phase I/II N=30 Long term follow up study of patients who have received SPK-8011 in any prior Spark- sponsored SPK-8011 study Gene transfer, dose-finding safety, tolerability, and efficacy study of SPK-8011 Hemophilia A with inhibitors to Factor VIII Phase I/II N=30 Gene transfer, dose-finding safety, tolerability, and efficacy study of SPK-8016 in individuals with FVIII inhibitors Safety Primary endpoint Ongoing Status CT Identifier NCT03432520 ☐ ■ Safety and changes from baseline in FVIII activity levels at week 52 FPI Q1 2017 Updated data presented at ISTH 2020 and 2021 Recruitment completed Q1 2021 Data published in NEJM 2021; 385:1961-1973 NCT03003533 Safety; peak and steady state FVIII activity levels at week 52 FPI Q1 2019 NCT03734588 ISTH=International Society on Thrombosis and Haemostasis; NEJM-New England Journal of Medicine 142 Hemophilia

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