Half-Year 2022 Financial and Clinical Trials Update
Tominersen (RG6042, HTT ASO)
Antisense oligonucleotide (ASO) targeting human HTT mRNA
Indication
Huntington's disease
Phase/study
Phase III
Generation HD1
N=791
# of patients
ARM A: Tominersen 120mg q2w
ARM B: Tominersen 120mg q4m
ARM C: Placebo q2w
Design
▪ CUHDRS globally
Primary endpoint
TFC USA only
Status
CT Identifier
Phase III
GEN-EXTEND
N=1,050
Roche
OLE study in patients participating in prior Roche and Genentech sponsored
studies
ARM A: Tominersen 120mg q2w
• ARM B: Tominersen 120mg q4m
■ Long term safety, tolerability
FPI Jan 2019
▪ Q1 2019 protocol modified to allow for bi-monthly vs four-monthly dosing,
FPI for new protocol July 2019
■ Recruitment completed Q2 2020
■ Dosing stopped in Q1 2021 based on IDMC recommendation regarding the
potential benefit/risk profile for study participants. No new safety signals
identified.
Data presented at EHDN and CHDI 2022
•
⚫ FPI Q2 2019
Dosing stopped in Q1 2021
NCT03761849
NCT03842969
In collaboration with lonis Pharmaceuticals
CUHDRS=composite Unified Huntington's Disease Rating Scale; TFC=total function capacity; HTT=Huntingtin; OLE=Open Label Extension; IDMC-Independent Data Monitoring Committee; CHDI-Huntington's Disease Association of
Ireland; EHDN-European Huntington's Disease Network
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