#United States #San Diego #Neurocrine Biosciences #Valbenazine #Dyskinetic CP

Neurocrine Biosciences' Phase 3 Study Update on Valbenazine for Dyskinetic Cerebral Palsy

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) has provided an essential update regarding its Phase 3 KINECT®-DCP study, which aimed to evaluate the effectiveness, safety, and tolerability of valbenazine in treating dyskinetic cerebral palsy (DCP). Unfortunately, the study results were disappointing as neither the primary objectives nor critical secondary endpoints were met.

The primary goal of the KINECT®-DCP study was focused on improving understanding and treatment outcomes for chorea, an involuntary movement disorder commonly affecting individuals with DCP. Dr. Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, expressed regret over the outcome, given that approved treatment options for patients suffering from dyskinetic cerebral palsy are currently nonexistent. He extended gratitude to everyone who participated in the trial—patients, their families, investigators, and staff—whose dedication made the study possible.

This clinical trial, recognized as the largest double-blind placebo-controlled study conducted in the context of dyskinetic cerebral palsy, assessed the efficacy of valbenazine over a treatment period of 14 weeks. Participants involved ranged from pediatric to adult subjects, all of whom experienced choreiform movements linked to cerebral palsy. Notably, adverse events reported during the trial were generally in line with the previously established safety profile of valbenazine.

The data will be comprehensively presented at an upcoming scientific meeting, allowing for a better understanding of the research findings and their implications for future treatments.

Understanding Dyskinetic Cerebral Palsy

Dyskinetic cerebral palsy (DCP) is one of the main forms of cerebral palsy, characterized by fluctuating muscle tone, which can significantly impair movement. It primarily affects coordination and can manifest through symptoms such as dystonia and choreoathetosis. These involuntary movements often lead to marked motor impairments, having a profound impact on the quality of life for those affected. Estimates suggest that DCP accounts for approximately 15% of all cerebral palsy cases, with about three in every 1,000 children in the U.S. being affected by some form of this disorder.

About the Phase 3 KINECT®-DCP Study

The KINECT®-DCP study was a randomized, double-blind, placebo-controlled trial designed to scrutinize the effectiveness of valbenazine for patients dealing with dyskinesia resulting from cerebral palsy. Participants, aged 6 to 70 years, were randomly assigned to receive valbenazine or a placebo for the specified duration. Following this double-blind treatment phase, participants had the opportunity to join an open-label extension phase, where they were administered valbenazine.

The main objective of this extensive study—encompassing the lives of several individuals afflicted with DCP—was to evaluate the potential efficacy of valbenazine as a therapeutic option and improve outcomes related to chorea.

An Overview of Valbenazine

Valbenazine operates as a selective vesicular monoamine transporter 2 (VMAT2) inhibitor. Initially approved by the U.S. Food and Drug Administration (FDA) in 2017, valbenazine was recognized as the first treatment explicitly targeted for tardive dyskinesia. In 2023, the FDA granted approval for its use to manage chorea associated with Huntington's disease. As part of its development strategy, Neurocrine is advancing two next-generation VMAT2 inhibitors, with one, NBI-1065890, expected to commence Phase 2 trials for tardive dyskinesia in 2026.

Neurocrine Biosciences: A Leader in Neuroscience

Neurocrine Biosciences is committed to addressing complex neurological disorders through innovative treatments. Their portfolio includes FDA-approved medications for conditions ranging from tardive dyskinesia to endometriosis and congenital adrenal hyperplasia. With a strong focus on developing therapies that cater to the urgent needs faced by patients, the company strives to alleviate suffering for those battling neurological and neuropsychiatric disorders. As research continues, Neurocrine aims to refine and expand its capabilities in delivering meaningful solutions.

While today’s news regarding the KINECT®-DCP study poses challenges, Neurocrine's tenacity in finding impactful treatments for debilitating diseases remains unwavering. The ongoing battle against DCP and similar conditions underscores the vital need for continued research and development.