Half-Year 2022 Financial and Clinical Trials Update
Evrysdi (risdiplam, RG7916)
Oral SMN2 splicing modifier
Indication
Phase/study
Phase II
RAINBOWFISH
Spinal muscular atrophy (SMA)
Phase II/III
MANATEE
Roche
# of patients
Design
Primary endpoint
Status
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
▪ 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 and MDA 2022
ARM A:
N=180
Part 1: GYM329 plus Evrysdi for 24 weeks, followed by GYM329 plus Evrysdi for
72 weeks
Part 2: 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
• Safety, PK/PD and muscle biomarkers
FPI Part 1 Q2 2022
Orphan Drug Designation granted by FDA in Q4 2021 for GYM329
☐
Filed in US and EU Q4 2021
▪ Approved in US Q2 2022
CT Identifier
In collaboration with PTC Therapeutics and SMA Foundation
NCT03779334
NCT05115110
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
110
NeuroscienceView entire presentation