Abstract Details

Title Sustained Improvements with Once-daily Valbenazine in Chorea Associated with Huntington’s Disease: Interim Results from a Long-term Open-label Study

Topic Movement Disorders

Presentation(s) S30 - Movement Disorders: Clinical Trials in Movement Disorders (1:24 PM-1:36 PM)

Poster/Presentation
Number 003

Objective

To present interim analyses from KINECT®-HD2 (NCT04400331), an ongoing open-label study of once-daily valbenazine for chorea associated with Huntington's disease (HD).

Background Valbenazine significantly improved chorea versus placebo in KINECT®-HD (NCT04102579). However, more "real-world" data are needed on maintenance of efficacy, long-term tolerability, and effects on chorea when taken with other HD medications (e.g., antipsychotics).

Design/Methods KINECT-HD2 participants (including KINECT-HD completers) receive once-daily valbenazine for up to 156 weeks (starting dose: 40mg; target maintenance dose: 80mg). Interim analyses with results up to Wk50 include mean changes from baseline in Unified Huntington’s Disease Rating Scale Total Maximal Chorea (TMC) score and response status (“much improved” or better) for Clinical Global Impression of Change (CGI-C) and Patient Global Impression of Change (PGI-C). A preliminary analysis of TMC data from participants already receiving stable antipsychotic treatment at enrollment is also presented, along with treatment-emergent adverse events (TEAEs) for all participants who took ≥1 valbenazine dose. All outcomes were analyzed descriptively.

Results 127 participants were enrolled at time of analysis. Mean TMC score reductions (±SEM) were observed with valbenazine 40mg by Wk2 (-3.4±0.3, n=118) and sustained with valbenazine ≤80mg from Wk8 (-5.6±0.3, n=110) to Wk50 (-5.8±0.5, n=66), the last visit retaining >50% of participants. In participants on stable antipsychotic treatment at enrollment, similar improvements were found from Wk4 (-6.0±1.3, n=5) to Wk20 (-5.2±2.2, n=5), the last visit with >2 participants taking concomitant antipsychotics. Response status at Wk50 was 76.9% (50/65) for CGI-C and 74.2% (49/66) for PGI-C. Among 125 participants receiving treatment, 17 (13.6%) reported serious TEAEs, and 17 (13.6%) discontinued due to a TEAE. The most common TEAEs were fall (30.4%, n=38), fatigue (24.0%, n=30), and somnolence (24.0%, n=30).

Conclusions

In these interim analyses of KINECT-HD2, long-term treatment with once-daily valbenazine was well tolerated and provided clinically meaningful improvement in chorea severity, regardless of concomitant antipsychotic therapy.

Authors/Disclosures
Erin Furr-Stimming, MD, FAAN (University of Texas Health Science Center-Houston) PRESENTER	Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Michael J. Fox Foundation. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medscape. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedPage. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedPage. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Wave Life Sciences. Dr. Furr-Stimming has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Teva Pharmaceuticals. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Wave Life Sciences. Dr. Furr-Stimming has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Latus Bio. The institution of Dr. Furr-Stimming has received research support from Roche/Genetech. The institution of Dr. Furr-Stimming has received research support from Uniqure. The institution of Dr. Furr-Stimming has received research support from CHDI. The institution of Dr. Furr-Stimming has received research support from Huntington Study Group/Neurocrine Bioscienes. The institution of Dr. Furr-Stimming has received research support from NIH/University of Iowa. The institution of Dr. Furr-Stimming has received research support from Sage Therapeutics. The institution of Dr. Furr-Stimming has received research support from HDSA. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has received publishing royalties from a publication relating to health care. Dr. Furr-Stimming has a non-compensated relationship as a Committee member with AAN UES Committee that is relevant to AAN interests or activities.
Daniel O. Claassen, MD, FAAN (Vanderbilt University Medical Center)	Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark . The institution of Dr. Claassen has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Alterity. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for University of Michigan. Dr. Claassen has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for HD Insights. The institution of Dr. Claassen has received research support from NIH. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from HDSA. The institution of Dr. Claassen has received research support from Department of Defense. The institution of Dr. Claassen has received research support from CHDI. The institution of Dr. Claassen has received research support from Genentech/ Roche.
Elise P. Kayson, RN	Elise Paulin Kayson, RN has nothing to disclose.
Jody Goldstein (Huntington Study Group)	No disclosure on file
Sean Hinton, PhD (Neurocrine Biosciences)	Dr. Hinton has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Hinton has stock in Neurocrine Biosciences, Inc..
Olga Klepitskaya, MD, FAAN (Neurocrine Biosciences, Inc)	Dr. Klepitskaya has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Klepitskaya has stock in Neurocrine Biosciences.
Hui Zhang (Neurocrine Bioscience)	Hui Zhang has received personal compensation for serving as an employee of Neurocrine Bioscience Inc..
Grace L. Liang, MD	Dr. Liang has received personal compensation for serving as an employee of Neurocrine Biosciences. Dr. Liang has stock in Neurocrine Biosciences.
Dietrich Haubenberger, MD, FAAN (Neurocrine Biosciences)	Dr. Haubenberger has received personal compensation for serving as an employee of Neurocrine Biosciences, Inc. Dr. Haubenberger has stock in Neurocrine Biosciences. Dr. Haubenberger has a non-compensated relationship as a Member of the Board with American Society for Experimental Neurotherapeutics that is relevant to AAN interests or activities.

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