Neurocrine Biosciences (NBIX) Phase 3 KINECT-HD Study Evaluating Valbenazine for Chorea Associated with Huntington Disease Met Primary Endpoint

Neurocrine Biosciences (Nasdaq: NBIX) today announced positive top-line data from its Phase 3 KINECT-HD study evaluating the efficacy, safety and tolerability of valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor being investigated as a once-daily treatment in adults with chorea associated with Huntington disease (HD). The study met the primary endpoint of reduction in severity of chorea, the cardinal motor feature in Huntington disease, as measured by change in the Unified Huntington's Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score from baseline to the average score at weeks 10 and 12.

In the randomized, double-blind, placebo-controlled KINECT-HD study, treatment with valbenazine resulted in a placebo-adjusted mean reduction in the TMC score of 3.2 units (p < 0.0001), indicating a highly statistically significant improvement in chorea. The TMC score is part of the motor assessment of the UHDRS® and measures chorea in seven different body parts, including the face, oral-buccal-lingual region, trunk and each limb independently. The TMC score is the sum of the individual scores and ranges from 0 to 28. The secondary endpoints of Clinical Global Impression of Change (CGI-C) Response Status and Patient Global Impression of Change (PGI-C) Response Status were also statistically significant in favor of valbenazine treatment.

The treatment emergent adverse events observed in this trial were consistent with the known safety profile of valbenazine. No suicidal behavior or worsening of suicidal ideation was observed in the valbenazine-treated subjects in this study. Data from the Phase 3 KINECT-HD study will be presented at a medical conference in 2022.

"The positive results of the KINECT-HD study move us closer to bringing valbenazine as a potential treatment option to patients in the U.S. living with chorea, one of the most common symptoms of Huntington disease," said Eiry W. Roberts, M.D., 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 U.S. Food and Drug Administration 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 patient population."

HD impacts an estimated 30,000 adults in the United States. Chorea, an involuntary movement disorder characterized by irregular and unpredictable movements, is one of the most common symptoms, affecting roughly 90% of those diagnosed with Huntington disease over the course of disease progression.

"We are energized by these positive data and grateful to have been part of a study that has advanced a potential new therapy for people living with chorea associated with Huntington disease," said Andrew Feigin, M.D., Chair of the Huntington Study Group and Professor, Department of Neurology at NYU Grossman School of Medicine. "We look forward to continuing our work with Neurocrine Biosciences through the KINECT-HD2 study and working toward our goal of benefiting the lives of those living with this condition."

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

KINECT-HD2 is an open-label study to evaluate the long-term safety and tolerability of valbenazine for the treatment of chorea in Huntington disease.

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