Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE), a developer of transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting a poster describing its recent study of CBD in pediatric patients with Fragile X syndrome.
The study is known as CONNECT-FX, or Clinical study Of CaNNabidiol in ChildrEn and AdolesCenTs with Fragile X. It describes the role of what’s called FMR1 methylation in patients with Fragile X syndrome (FXS) as it correlates to disease severity and as a prognostic biomarker.
The data will presented at the virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting. The company will also give an oral presentation during the “Research Pipeline: New Findings on Diagnostic and Therapeutics” session of the virtual American Academy of Child and Adolescent Psychiatry (AACAP) 2020 Annual Meeting on October 23.
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A copy of the poster titled, “ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents With Fragile X Syndrome: Role of Methylation Status as a Correlate to Disease Severity and as a Prognostic Biomarker” is available at http://zynerba.com/publications/.
“We are excited to provide an update on the potential role of methylation status of the FMR1 gene as a predictive biomarker of preferential response to Zygel (ZYN002) in the treatment of the behavioral symptoms of FXS,” Chief Medical Officer Joseph Palumbo said in a statement. “These new data demonstrate that in patients diagnosed with FXS with a fully methylated FMR1 gene significantly more patients who received Zygel achieved a clinically meaningful improvement in their behavioral symptoms compared to patients who received placebo.”
Recent studies have suggested that neuropsychiatric conditions like Fragile X Syndrome — the most common form of inherited learning disability and attention deficit disorder — are associated with a disruption in the endocannabinoid system, and CBD may improve certain core social and behavioral symptoms by modulating the biological system.
CONNECT-FX was a randomized, double-blind, multinational, 14-week pivotal study to evaluate the efficacy and safety of Zygel in children/adolescents aged 3 to 17 years.
In a previous 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).
However, the company said a pre-planned ad hoc analysis of the most severely impacted patients in the trial — 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.
Zynerba said it determined that 58.2% of FMet patients receiving Zygel achieved a clinically meaningful change in their socially avoidant behavior compared to 40.6% of patients receiving placebo, and 40.3% of patients receiving Zygel achieved a clinically meaningful change in Irritability compared to 23.8% of patients receiving placebo.
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