Roche Pharmaceutical Development and Sales Overview

Evrysdi (risdiplam, RG7916) Oral SMN2 splicing modifier Indication Spinal muscular atrophy (SMA) Phase/study # of patients Design Primary endpoint Status Phase II/III FIREFISH N=21 (Part 1), 41 (Part 2) Open-label study in infants with type 1 SMA Part I (dose-finding): At least 4 weeks Part II (confirmatory): 24 months " Safety, tolerability, PK/PD and efficacy 12-month data from Part I presented at AAN, CureSMA and EAN 2019; 16-month data presented at WMS 2019 Study met primary endpoint in Part II Q1 2020 Part II 1-year data presented at AAN 2020, Part I 2- year data at WMS 2020 Part I data published in NEJM 2021;384:915-923 Part II 2-year data presented at AAN 2021 Part II 1-year data published in NEJM 2021;385:427-435 3-year data presented at EPNS 2022 Phase II/III SUNFISH N=51 (Part 1), 180 (Part 2) Randomized, double-blind, placebo-controlled study in adult and pediatric patients with type 2 or type 3 SMA: " Part I (dose-finding): At least 12 weeks Part II (confirmatory): 24 months Safety, tolerability, PK/PD and efficacy Recruitment completed for part 2 Q3 2018 12-month data from Part I presented at AAN, CureSMA and EAN 2019; 16-month data presented at WMS 2019 Study met primary endpoint in Part II Q4 2019 Part II 1-year data presented at SMA Europe 2020, 2-year data at MDA 2021 and 3-year data at MDA 2022 Part II 1-year data published in Lancet Neurology, 2022; 21 (1) 42-52 ☐ Phase II JEWELFISH N=174 Open-label single arm study in adult and pediatric patients with previously treated SMA type 1, 2 and 3 " Safety, tolerability, PK/PD FPI Q1 2017 Data presented at WMS 2017, AAN 2018, WMS 2018, CureSMA 2019, WMS 2019, CureSMA 2020 and 2021 Recruitment completed Q1 2020 Orphan drug designation granted by FDA Q1 2017 and EU Q1 2019, PRIME designation in Q4 2018 Approved in US Q3 2020 and EU Q1 2021 NCT02908685 CT Identifier NCT02913482 In collaboration with PTC Therapeutics and SMA Foundation NCT03032172 SMA=Spinal muscular atrophy; SMN=survival motor neuron; PK/PD=Pharmacokinetics/Pharmacodynamics; PRIME=priority medicines; AAN=American Academy of Neurology; WMS=World Muscle Society; EAN-European Academy of Neurology; NEJM-New England Journal of Medicine; MDA-Muscular Dystrophy Association; CureSMA=Annual SMA Conference; EPNS-European Paediatric Neurology Society 87 Roche Neuroscience

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