Zynerba Pharmaceuticals Presents Data Supporting FMR1 Methylation Status as a Correlate to Fragile X Syndrome Severity at the Virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting
DEVON, Pa., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and
near-rare neuropsychiatric disorders, is presenting a poster describing data from the CONNECT-FX (Clinical study Of CaNNabidiol (CBD) in
ChildrEn and AdolesCenTs with Fragile X) trial describing the role of FMR1 methylation status in
children and adolescents with Fragile X syndrome (FXS) as a correlate to disease severity and as a prognostic biomarker. These data are being presented at the virtual Joint 16th
International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting. These data will also be presented as 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 Friday, October 23rd,
A copy of the poster entitled, “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 on the Zynerba corporate website 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,” said Zynerba’s Chief Medical Officer, Joseph M. Palumbo, MD, FAPA, MACPsych. “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.”
CONNECT-FX is 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. Although the CONNECT-FX full analysis set did not achieve statistical significance in its endpoints, building on current scientific evidence, a pre-planned ad hoc analysis of patients having at least 90% methylation (“full methylation” or FMet) of the impacted FMR1 gene, representing 80% of the overall study population, was performed. The results, including the achievement of statistical significance (p=0.020) in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo, suggest that Zygel may have benefit in patients with full methylation of the FMR1 gene.
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