Roche Pharmaceutical Development and Sales Overview

Evrysdi (risdiplam, RG7916) Oral SMN2 splicing modifier Indication Spinal muscular atrophy (SMA) Phase/study # of patients Design Phase II RAINBOWFISH N=25 Open-label, single-arm, multicenter study in infants aged from birth to 6 weeks who have been genetically diagnosed with Spinal muscular atrophy but are not yet presenting with symptoms Primary endpoint Status CT Identifier ARM A: Phase II/III MANATEE N=180 Roche ☐ ☐ Part I: GYM329 plus Evrysdi for 24 weeks, followed by GYM329 plus Evrysdi for 72 weeks Part II: GYM329 plus Evrysdi for 72 weeks ARM B: Placebo plus Evrysdi comparator Change from baseline in revised hammersmith scale (RHS) score after week 72 of treatment Proportion of participants with two copies of the SMN2 gene (excluding the known SMN2 gene modifier mutation c.859G>C) and baseline CMAP>=1.5 millivolt who are sitting without support FPI Q3 2019 Recruitment completed Q1 2022 Initial data presented at CureSMA, WMS 2021, MDA and WMS 2022 Filed in US and EU Q4 2021 Approved in US Q2 2022 ■ Safety, PK/PD and muscle biomarkers ■ FPI Part I Q2 2022 Orphan Drug Designation granted by FDA in Q4 2021 for GYM329 NCT03779334 NCT05115110 In collaboration with PTC Therapeutics and SMA Foundation SMN=survival motor neuron; CMAP=compound muscle action potential; PK/PD-Pharmacokinetics/Pharmacodynamics; WMS-World Muscle Society; CureSMA=Annual SMA Conference; MDA-Muscular Dystrophy Association 88 Neuroscience

View entire presentation