Valbenazine Reduced Chorea Associated With Huntington Disease

In a phase 3 study (NCT04102579), participants with Huntington disease (HD) treated with valbenazine (Ingrezza; Neurocrine Biosciences, San Diego, CA) had a reduction in chorea severity. Chorea reduction was 3.2 units (P<.0001) on the Unified HD Rating Scale (UHDRS) Total Maximal Chorea (TMC) at weeks 10 and 12. Statistically significant changes on the Clinical Global Impression of Change (CGI-C) Response Status and Patient Global Impression of Change (PGI-C) Response Status in favor of valbenazine treatment vs placebo were also observed.

"The positive results of the KINECT-HD study move us closer to bringing valbenazine as a potential treatment option to patients in the US living with chorea, one of the most common symptoms of HD," said Eiry W. Roberts, MD, chief medical officer at Neurocrine Biosciences. "We are immensely grateful to our partners at the Huntington Study Group and the Clinical Trials Coordination Center at the University of Rochester, New York, who were instrumental in completing this study, as well as the study participants and the families and caregivers who supported them. We will review the complete data and begin preparing a supplemental new drug application (sNDA) for submission to the Food and Drug Administration (FDA) next year. In the meantime, we will continue dosing in the KINECT-HD2 study, which is evaluating the long-term safety and tolerability of valbenazine in this same population."

"We are incredibly grateful to the KINECT-HD participants for contributing the success of this important clinical trial," said Erin Furr-Stimming, MD, FAAN, principal investigator, Huntington Study Group and professor of Neurology at McGovern Medical, UTHealth Houston.

The treatment emergent adverse events observed in this trial were consistent with the known safety profile of valbenazine, and no suicidal behavior or worsening of suicidal ideation in the valbenazine-treated participants. Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved by the FDA to treat tardive dyskinesia. Data from this study will be presented at a medical conference in 2022.