Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) has announced that it will present caregiver data from a study of its CBD-based Fragile X Syndrome (FXS) treatment Zygel at the 17th NFXF International Fragile X Conference Research Roundup.
The 14-week trial assessed the efficacy and safety of Zygel CBD gel as a treatment for the behavioral symptoms of FXS in 212 child and adolescent patients. As part of the trial, the company compiled data from caregivers on the most important behavioral challenges of the condition.
The presentation, titled, “Zygel (ZYN002) Development Program in Fragile X Syndrome” is scheduled for 3:45 pm ET, and registration can be done here.
READ: Zynerba announces top-line results from CONNECT-FX study of Zygel CBD gel in children and adolescents with Fragile X syndrome
The survey results showed that caregivers found anxiety, socially avoidant behaviors and disruptive behaviors including temper tantrums to be the most challenging behavioral symptoms of Fragile X.
Fragile X is a genetic condition caused by a mutation in the FMR1 gene, which causes the X chromosome to appear constricted, or fragile, under a microscope. Patients with Fragile X often have learning disabilities and other cognitive impairments.
“I am very pleased to participate in today’s Fragile X Research Roundup on Fragile X Awareness Day,” Zynerba's chief medical officer Joseph Palumbo said in a statement. “We believe that the caregiver data that we are presenting today further support the statistically significant improvement we achieved in the primary endpoint of social avoidance in patients with full methylation of their FMR1 gene. We look forward to discussing these and other data with the US Food and Drug Administration as soon as possible regarding a potential regulatory path forward.”
The study is known as CONNECT-FX, which stands for Clinical study of Cannabidiol in Children and Adolescents with Fragile X.
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