Neurocrine's Phase 3 Valbenazine Trial for Dyskinetic Cerebral Palsy Fails to Meet Primary Endpoints
核心洞察
Neurocrine Biosciences announced that its Phase 3 KINECT-DCP study of valbenazine in dyskinetic cerebral palsy (搜索) failed to meet primary and key secondary endpoints for improving chorea (搜索) symptoms.
The study was the largest double-blind placebo-controlled clinical trial ever completed in dyskinetic cerebral palsy (搜索), evaluating 14 weeks of treatment in participants aged 6 to 70 years.
The failure represents a significant setback as no approved treatments currently exist for people living with dyskinetic cerebral palsy (搜索), which affects approximately 15% of cerebral palsy (搜索) cases.
Neurocrine Biosciences announced that its Phase 3 KINECT-DCP study evaluating valbenazine in dyskinetic cerebral palsy (搜索) (DCP) failed to meet its primary and key secondary endpoints, marking a significant setback in addressing an unmet medical need affecting thousands of patients worldwide.
The study, described as the largest double-blind placebo-controlled clinical trial ever completed in DCP, was designed to assess whether 14 weeks of valbenazine treatment could improve chorea (搜索)—a type of involuntary movement—in pediatric and adult participants with dyskinetic cerebral palsy (搜索).
"These results are disappointing, as there are no approved treatments for people living with dyskinetic cerebral palsy (搜索)," said Sanjay Keswani, M.D., Chief Medical Officer at Neurocrine Biosciences. "We are deeply grateful to the patients and their families, as well as the investigators and site staff, whose commitment, dedication, and participation made this clinical trial possible."
Study Design and Patient Population
The Phase 3, randomized, double-blind, placebo-controlled trial enrolled participants aged 6 to 70 years who experienced choreiform movements due to cerebral palsy (搜索). Participants were randomized to receive either valbenazine or placebo for 14 weeks during the double-blind treatment period, followed by an optional open-label extension phase where all participants received valbenazine.
The primary objective focused on evaluating valbenazine's efficacy versus placebo in improving chorea (搜索) in pediatric and adult subjects with DCP who had choreiform movements. Adverse events observed during the trial were generally consistent with valbenazine's established safety profile.
Unmet Medical Need in Dyskinetic Cerebral Palsy
Cerebral palsy (搜索) is a nonprogressive neurodevelopmental disorder affecting movement and posture that begins in early childhood, occurring in approximately three per 1,000 children in the United States. Dyskinetic cerebral palsy (搜索) accounts for approximately 15% of all CP cases and presents particularly challenging symptoms.
People living with DCP often experience mixed hyperkinetic movements, including dystonia (搜索) (sustained or intermittent involuntary muscle contractions) and choreoathetosis (搜索) (random or writhing involuntary movements), leading to severe motor impairment. Currently, no therapies are approved to treat dystonia or choreoathetosis in cerebral palsy (搜索).
Valbenazine's Established Therapeutic Profile
Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2 (搜索)) inhibitor that has demonstrated clinical success in other movement disorders. The U.S. Food and Drug Administration approved valbenazine in 2017 as the first drug ever developed for treating tardive dyskinesia (搜索). In 2023, the company received FDA approval for valbenazine as a treatment for chorea (搜索) associated with Huntington's disease (搜索).
Future Development Plans
Despite this setback in DCP, Neurocrine Biosciences continues advancing its VMAT2 (搜索) inhibitor pipeline. The company is developing two next-generation VMAT2 inhibitors, with NBI-1065890 scheduled to enter Phase 2 development for tardive dyskinesia (搜索) in 2026.
Neurocrine plans to present the complete study results from the KINECT-DCP trial at an upcoming scientific meeting, which may provide additional insights into the trial outcomes and inform future research directions in this challenging therapeutic area.