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Monoclonal Antibody Being Tested for Huntington's Disease

AUGUST 23, 2016
James Radke, PhD
VX15, a monoclonal antibody towards semaphorin 4D, is in a phase 2 clinical trial for the treatment of Huntington’s disease. The antibody has also been given orphan drug designation by the FDA.

The Phase 2 SIGNAL Trial   

The SIGNAL trial is a phase 2, multi-center, randomized, double-blind, placebo controlled study in patients with late prodromal and early manifest Huntington’s disease. The primary outcome measure is to evaluate the safety and tolerability of monthly IV administration of a single dosage of VX15 (or placebo). Secondary outcome measures include determining the effect of VX15 on brain volumes (MRI), FDG-PET imaging, 11C-PBR28 (TSPO) PET imaging and clinical features of Huntington’s disease including cognition, motor function, behavior, functional abilities, and global function.
 
The trial is currently enrolling (by invitation only),  94 adults with late prodromal [CAG-age product score (CAP score) of greater than 200 and Diagnostic Confidence Level (DCL) of 2 or 3] or early manifest Huntington’s disease [Total Functional Capacity (TFC) greater than or equal to 11].
 
For more information about the trial, visit  clinicaltrials.gov/ct2/show/NCT02481674

Huntington’s Disease

Huntington’s disease is a fatal neurodegenerative disease characterized by uncoordinated and uncontrollable movements, cognitive deterioration and behavioral and/or psychological problems. The classic onset of Huntington’s disease symptoms typically occurs in middle age, but the disease also manifests in children and the elderly. Huntington’s disease is the most common genetic cause of abnormal involuntary writhing movements called chorea. Disease progression is characterized by a gradual decline in motor control, cognition and mental stability and generally results in death within 15‐25 years of clinical diagnosis.
 
Huntington's Disease is an inherited brain disorder that results in the progressive loss of both mental and motor control. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25-year period until the patient eventually dies. Chorea is the pronounced symptom of the condition.

VX15

VX15 is a novel monoclonal antibody that blocks the activity of Semaphorin 4D. According to the makers of VX15, Vaccinex, Semaphorin 4D plays a role in multiple cellular processes that contribute to pathophysiology of neuroinflammatory/neurodegenerative diseases, such as Huntington’s disease and multiple sclerosis. 

 


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