Written By: Pharmacally Medical News Desk
Neurocrine Biosciences, Inc. has reported topline results from its Phase 3 KINECT-DCP study evaluating valbenazine in pediatric and adult patients with dyskinetic cerebral palsy (DCP). The study did not meet its primary endpoint or key secondary endpoints, marking a setback in efforts to develop the first approved therapy for this condition.
KINECT-DCP (NCT05206513) was the largest double-blind, placebo-controlled clinical trial ever completed in dyskinetic cerebral palsy. The study assessed whether 14 weeks of treatment with valbenazine could improve chorea, a form of involuntary, irregular movement that is a major contributor to disability in DCP. Participants aged 6 to 70 years with dyskinesia due to cerebral palsy and prominent choreiform movements were randomized to receive either valbenazine or placebo.
Despite the robust study design and broad age range, valbenazine did not demonstrate a statistically meaningful improvement over placebo on the primary endpoint of chorea reduction, nor on key secondary efficacy measures.
The safety and tolerability profile observed in KINECT-DCP was generally consistent with the established safety experience of valbenazine from prior indications. No new or unexpected safety signals were reported, an important consideration given the pediatric population included in the trial.
Commenting on the results, Neurocrine’s Chief Medical Officer, Sanjay Keswani, M.D., acknowledged the unmet need in this population and thanked patients, families, investigators, and site staff for their participation and commitment. The company emphasized that the negative outcome is particularly disappointing given the absence of approved treatments for dyskinetic cerebral palsy.
Neurocrine plans to present the full dataset from KINECT-DCP at an upcoming scientific meeting, where additional details on efficacy measures and subgroup analyses may be shared.
Cerebral palsy is a nonprogressive neurodevelopmental disorder affecting movement and posture, beginning in early childhood. Dyskinetic cerebral palsy accounts for roughly 15 percent of all CP cases and is characterized by mixed hyperkinetic movements, including dystonia and choreoathetosis. These involuntary movements often result in severe functional impairment, and there are currently no approved pharmacologic therapies to specifically treat dystonia or choreoathetosis in CP.
Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor. It was first approved by the US FDA in 2017 for tardive dyskinesia and later, in 2023, for chorea associated with Huntington’s disease. While the KINECT-DCP results limit its role in dyskinetic cerebral palsy, Neurocrine continues to invest in movement disorder research, including the development of next-generation VMAT2 inhibitors such as NBI-1065890, which is expected to enter Phase 2 testing for tardive dyskinesia in 2026. Neurocrine remains committed to movement disorders amid this setback
References
Neurocrine Biosciences Provides Update on Phase 3 Study of Valbenazine in Dyskinetic Cerebral Palsy, 22 December 2025, https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-provides-update-phase-3-study-valbenazine
Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy, ClinicalTrials.gov ID NCT05206513, https://clinicaltrials.gov/study/NCT05206513