Positive Phase III data for valbenazine for chorea associated with Huntington disease

Neurocrine Biosciences (Nasdaq: NBIX) rose 3.3% to $85.00 after-hours yesterday, when the US biotech announced positive top-line data from its Phase III KINECT-HD study evaluating the efficacy, safety and tolerability of Ingrezza (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).

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 III KINECT-HD study will be presented at a medical conference in 2022.