Half-Year 2022 Financial and Clinical Trials Update
Evrysdi (risdiplam, RG7916)
Oral SMN2 splicing modifier
Indication
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 1 (dose-finding): At least 4 weeks
Part 2 (confirmatory): 24 months
■ Safety, tolerability, PK/PD and efficacy
12-month data from Part 1 presented at AAN, CureSMA
and EAN 2019; 16-month data presented at WMS 2019
▪ Study met primary endpoint in part 2 Q1 2020
Part 2 1-year data presented at AAN 2020, part 12-
year data at WMS 2020
■ Part 1 data published in NEJM 2021;384:915-923
▪ Part 2 2-year data presented at AAN 2021
▪ Part 2 1-year data published in NEJM 2021;385:427-
435
■ 3-year data presented at EPNS 2022
Spinal muscular atrophy (SMA)
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 1 (dose-finding): At least 12 weeks
· Part 2 (confirmatory): 24 months
Safety, tolerability, PK/PD and efficacy
▪ Recruitment completed for part 2 Q3 2018
▪ 12-month data from Part 1 presented at AAN,
CureSMA and EAN 2019; 16-month data
presented at WMS 2019
▪ Study met primary endpoint in part 2 Q4 2019
· Part 2 1-year data presented at SMA Europe
2020, 2-year data at MDA 2021 and 3-year
data at MDA 2022
▪ Part 2 data 1-year published in Lancet
Neurology, Dec 2021
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
CT Identifier
NCT02913482
In collaboration with PTC Therapeutics and SMA Foundation
■ Approved in US Q3 2020 and EU Q1 2021
NCT02908685
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
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Roche
NeuroscienceView entire presentation