Session Information
Date: Monday, September 23, 2019
Session Title: Huntington’s Disease
Session Time: 1:45pm-3:15pm
Location: Agora 3 West, Level 3
Objective: To present the safety, tolerability and biomarker effects of RG6042 (previously, IONIS-HTTRX) during an open-label extension (OLE) study in adults with early manifest Huntington’s disease (HD).
Background: HD is a genetic, neurodegenerative and ultimately fatal disease that has a devastating impact on families across generations. HD is characterised by behavioural symptoms, as well as progressive cognitive and motor decline, resulting in increasing disability and loss of independence. RG6042, an intrathecally administered antisense oligonucleotide (ASO) targeting huntingtin (HTT) mRNA, is the first investigational treatment to show a dose-dependent, reversible lowering of HTT protein. Preclinical models have demonstrated improved motor function and delayed disease progression with RG6042. RG6042 was well tolerated in the IONIS-HTTRx Phase I/IIa study in patients with early HD (NCT02519036) and is currently being evaluated in this ongoing OLE study (NCT03342053).
Method: This OLE study (NCT03342053) is evaluating the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of 120mg RG6042, administered intrathecally, monthly or bi-monthly, for 15 months in adults (N=46) with early manifest HD (Stage I/II) who previously participated in the Phase I/IIa study (NCT02519036). Exploratory endpoints include assessment of standard and digital clinical outcomes, as well as biomarkers.
Results: At the time of abstract submission, over 350 doses of RG6042 have been administered. OLE participants in the monthly regimen have had between 8 and 15 doses of RG6042 and participants in the bi-monthly regimen have had between 4 and 7 doses. Interim safety, tolerability and biomarker data will be presented.
Conclusion: RG6042 is the first HTT-lowering treatment to be evaluated in patients with HD. The data presented here inform on the longer-term safety, tolerability and biomarker profile of RG6042. These data complement other studies in the RG6042 Global Development Program such as the RG6042 pivotal Phase III study GENERATION HD1.
To cite this abstract in AMA style:
S. Tabrizi, B. Leavitt, P. Sanwald Ducray, E. Wild, V. Schlegel, G. Hooper, A. Nicotra, J. Chevure, A. Smith, R. Lane, F. Bennett, L. Boak, R. Doody, S. Schobel. A safety, tolerability and biomarker update from an ongoing open-label extension study of RG6042 in adults with early manifest Huntington’s disease [abstract]. Mov Disord. 2019; 34 (suppl 2). https://www.mdsabstracts.org/abstract/a-safety-tolerability-and-biomarker-update-from-an-ongoing-open-label-extension-study-of-rg6042-in-adults-with-early-manifest-huntingtons-disease/. Accessed June 14, 2025.« Back to 2019 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/a-safety-tolerability-and-biomarker-update-from-an-ongoing-open-label-extension-study-of-rg6042-in-adults-with-early-manifest-huntingtons-disease/