The multi-national, randomized, double-blind, placebo-controlled trial assessed the efficacy and safety of the Zygel CBD gel as a treatment for behavioral symptoms of the syndrome (FXS) in 212 patients.
In a statement, Zynerba said Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS).
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The company also said Zygel did not demonstrate statistical significance versus placebo in the three key secondary endpoints, which were the change from baseline to the end of the treatment period in the Irritability subscale score of the ABC-CFXS, the Socially Unresponsive/Lethargic subscale score of the ABC-CFXS and Improvement in Clinical Global Impression (CGI-I).
Zynerba said a pre-planned ad hoc analysis of the most severely impacted patients in the trial -- as defined by patients having at least 90% methylation (full methylation) of the impacted FMR1 gene -- demonstrated that patients receiving Zygel achieved statistical significance in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo.
This group comprised 80% of the patients enrolled in the CONNECT-FX study, and the company said it believes that full methylation occurs in about 60% of the overall FXS patient population.
Meeting with FDA
Based on this analysis, Devon, Pennsylvania-based Zynerba said it intends to meet with the US Food and Drug Administration regarding a regulatory path forward for Zygel.
Fragile X is a genetic condition caused by a mutation in the FMR1 gene. The mutation causes the X chromosome to appear constricted, or fragile, under a microscope, hence its name. Patients with Fragile X often have learning disabilities and other cognitive impairments.
“This study identified a key population of patients who appear to benefit from treatment of their behavioral symptoms of FXS with Zygel,” said Dr Randi Hagerman, an investigator in the clinical trial and medical director and endowed chair in Fragile X research at UC Davis. “Zygel has the potential to be an important therapeutic option for the most severely impacted patients with Fragile X.”
Zynerba CEO Armando Anido said the test results may offer the company a “pathway towards licensure” and that it will seek to discuss the result with the FDA “as soon as possible.”
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