Company Releases Data From Open Label Trial of Experimental Fragile X Syndrome Treatment

According to a story from, the drug company Zynerba Pharmaceuticals, Inc. has announced that the data from its phase 2 open label trial has been officially published. This clinical trial was testing the company’s experimental therapeutic candidate ZYN002 as a treatment for symptoms of fragile X syndrome. ZYN002 is a cannabidiol (CBD) gel and Zynerba is focused on the development of trandermal cannabindoid therapies for rare neuropsychiatric conditions.

About Fragile X Syndrome (FXS)

Fragile X syndrome is a genetic disorder which affects men more often than women. It is characterized by intellectual disability and distinctive physical features. The disease is caused by a mutation affecting the FMR1 gene. Symptoms of fragile x syndrome include intellectual disability which can vary widely in severity, autism, social anxiety, ADHD, deficits of working memory, seizures, vision problems, and hyperactive behavior. Distinct physical characteristics become noticeable once puberty begins and may include prominent ears, poor muscle tone, a long face, enlarged testicles, soft skin, flat feet, and hyperflexible finger joints. Treatment is mostly symptomatic and may include antidepressants, antipsychotics, and stimulants. Interventions such as special education, speech therapy, and behavioral therapy can be beneficial. There is no cure for the disorder. Life expectancy is about 12 years lower than the general population. To learn more about fragile X syndrome, click here.

Trial Findings

The results from this trial have led the authors to suggest that ZYN002 could be a useful treatment for emotional and behavioral symptoms that appear with fragile X syndrome. The open label study included 20 male and female patients. These patients were permitted to continue any existing therapies that they were using. For a period of 12 weeks, patients could receive three different dose levels of the gel. These doses ranged from a minimum of 50mg once per day, 50mg twice per day, or a maximum dose of 125mg twice per day.

The treatment improved patient symptoms based on a variety of behavioral measurements. Symptoms such as mood instability, social avoidance, anxiety, hyperactivity, and impulsivity all saw improvement. These changes were all superior to placebo groups in prior studies. No severe adverse events were reported during the trial.


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