Phase II Trial of Fragile X Treatment in Europe: AFQ056

This small Phase II trial sponsored by Novartis will test a new compound which is an mGluR5 antagonist.

A new trial has been posted today at Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients.

AFQ056 is a new compound developed by Novartis; it’s their lead mGluR5 antagonist, which means it reduces mGluR5 activity in the brain. This, as many FRAXA-supported research studies have shown over the past few years – is known to be overactive in Fragile X.

This trial, conducted in Italy, France, and Switzerland, will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg twice daily. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score, a standard and widely-accepted measure of mental health. Enrollment is by invitation only.

AFQ056 is also being studied as a potential treatment for disorders such as Parkinson’s Disease, Gastroesophageal Reflux Disease, and to help cigarette smokers reduce or stop smoking. More information about AFQ056 is available at


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