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
# of patients
Design
Phase I/lla
Phase II
OLE
N=46
N=46
"
▪ Multiple ascending doses of tominersen administered intrathecally to adult
patients with early manifest Huntington's Disease
"
▪ Patients from phase I are enrolled into OLE
Primary endpoint
▪ Safety, tolerability and PK/PD
■ FPI Q3 2015
Status
CT Identifier
Data presented at CHDI 2018 and AAN 2018
PRIME designation granted 2018
Published in NEJM 2019; 380:2307-2316
NCT02519036
■ Longer term safety, tolerability and PK/PD
▪ FPI Q1 2018
▪ PK/PD data presented at AAN 2019
Update presented at CHDI 2020
• Study completed, patients moved to GEN-EXTEND OLE
NCT03342053
In collaboration with lonis Pharmaceuticals
HTT=Huntingtin; PK/PD=Pharmacokinetics/Pharmacodynamics; PRIME=Priority medicines; OLE=Open Label Extension; AAN-American Academy of Neurology; NEJM-New England Journal of Medicine; CHDI-Huntington's Disease
Association of Ireland
146
Roche
NeuroscienceView entire presentation