According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company’s product Zygel™ (CBD gel) as a treatment for patients living with fragile X syndrome (FXS). Unfortunately, the gel failed to have a statistically meaningful impact and was unable to satisfy the primary and secondary study endpoints for the overall group. However, the data revealed that it could be effective for the most severely affected patients. Zynerba is focused on the development of transdermal CBD therapies.
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 fragile X syndrome. Life expectancy is about 12 years lower than the general population. To learn more about fragile X syndrome, click here.
About the Study
The multi-national study ran for a total of 14 weeks and involved a total of 212 patients. The primary endpoint for the trial was improvement in the results of an assessment called the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS). The three secondary endpoints were improvements in scores of the Socially Unresponsive/Lethargic subscale and the Irritability subscale, as well as Clinical Global Impression (CGI-I).
While Zygel failed to satisfy these endpoints, the study did not end in complete failure. Zygel was able to meet the primary endpoint for a certain subset of the patients. These patients, who have at least 90 percent methylation of the FMR1 gene and are the most severely affected by the disorder, did see meaningful improvement. This degree of methylation occurs in an estimated 60 percent of fragile X syndrome patients.
With these findings, Zygel will continue to be researched as a possible therapy for this group.