What's The Role Of The Endocannabinoid System In Fragile X Syndrome? How Can Cannabidiol Treatment Help? New Paper

Zynerba Pharmaceuticals, Inc. ZYNE, announced the publication of a paper entitled, “Role of the endocannabinoid system in fragile X syndrome: potential mechanisms for benefit from cannabidiol treatment,” in the Journal of Neurodevelopmental Disorders. The review of evidence suggests a central role for the endocannabinoid system (ECS) in neuronal development and cognitive function and in the pathogenesis of fragile X syndrome (FXS), and the potential role of CBD as a treatment for FXS.

“This publication describes the potential of Zygel as a treatment for fragile X syndrome based on the central role the endocannabinoid system plays in neuronal development and function,” stated Armando Anido, chairman and CEO of Zynerba. “The proposed mechanisms of action of cannabidiol are supported by the results from our phase 3 CONNECT-FX trial in patients with fragile X syndrome with greater than or equal to 90% methylation of the FMR1 gene, benefited from treatment with Zygel. These findings bolster our confidence in the design of the ongoing, confirmatory, pivotal phase 3 RECONNECT trial.”

FXS is caused by deficiency or absence of the FMR1 protein, FMRP. The absence of FMRP disrupts ECS signaling, which has been implicated in FXS pathogenesis. The ECS helps maintain neuronal function and signaling and is thought to be disrupted in FXS. CBD may help restore the function of the ECS in FXS according to a review of data to date. CBD may also act as an agonist on serotonin 5HT_1A and other receptors which may contribute to beneficial effects in people with FXS. Consistent with these proposed mechanisms of action of CBD in FXS, in the phase 3 CONNECT-FX trial, Zygel was associated with improvements in measures of social avoidance, irritability and social interaction, particularly in patients showing ≥90% methylation of the FMR1 gene.

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