Roche Pharmaceutical Development and Sales Overview
Tominersen (RG6042, HTT ASO)
Antisense oligonucleotide (ASO) targeting human HTT mRNA
Indication
Phase/study
# of patients
Design
Huntington's disease
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
Safety, tolerability and PK/PD
Primary endpoint
"
Longer term safety, tolerability and PK/PD
FPI Q3 2015
"
FPI Q1 2018
Status
■
Data presented at CHDI 2018 and AAN 2018
PRIME designation granted 2018
☐
PK/PD data presented at AAN 2019
☐
Update presented at CHDI 2020
Published in NEJM 2019; 380:2307-2316
Study completed, patients moved to GEN-EXTEND OLE
CT Identifier
In collaboration with lonis Pharmaceuticals
NCT02519036
NCT03342053
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
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Roche
NeuroscienceView entire presentation