Phase IIa of the clinical trial of Lu AG06466 has recently concluded. This drug is meant for the treatment of Tourette syndrome, but has not met the primary endpoints in its study. When compared to a placebo, Lu AG06466 was not significantly favored.

About Tourette Syndrome

Tourette syndrome is a nervous system disorder that is characterized by tics, which are involuntary movements or sounds. About 100,000 to 300,000 people are affected by this disorder in the United States.

Symptoms of Tourette syndrome vary greatly between individuals, as everybody experiences different tics. These tics can be brought on by a variety of triggers, such as stress, fatigue, illness, or excitement. Symptoms of this syndrome include:

• Jerking of the arms and head
• Repeated eye blinking
• Nose twitching
• Bending or twisting
• Shrugging
• Obscene gestures or words
• Grunting or barking
• Coughing and clearing the throat
• Repeating words
• Attention deficit disorder
• Obsessive compulsive disorder

The exact cause of this condition is unknown, although it is suspected to be a combination of inherited and environmental factors. Others believe that chemical imbalances within the brain result in Tourette syndrome.

No cure exists for Tourette syndrome. Treatment consists of medications that block dopamine, Botox injections, seizure medications, anti-depressants, and behavioral therapy. If one is affected by an extremely severe case, doctors may use a device that sends electrical impulses to the brain.

The Study

Lu AG06466 was evaluated in a double blind, randomized, placebo controlled study. 48 participants were enrolled in the study, all from various locations in Europe. The primary endpoint was based off of the Yale Global Tic Severity Scale.

Results showed that Lu AG06466 was not significantly favored over the placebo. While this result is unfortunate, there were no major adverse effects or issues in the safety profile. This means that this medication can be indicated for other conditions.

This drug, which is an inhibitor of monoacylglycerol lipase, did not meet endpoints in this trial, but researchers hope that it can be indicated in the treatment of other disorders. They will continue to develop and investigate this compound in order to better the lives of those with rare diseases.

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