Zynerba Initiates Pivotal Trial of Cannabidiol Gel in Fragile X Syndrome

July 9, 2018

Rare Daily Staff

Zynerba Pharmaceuticals said it has initiated a clinical trial of its experimental cannabidiol gel, ZYN002, in children and adolescents with Fragile X, a rare genetic condition that is the leading cause of inherited intellectual disability and autism spectrum disorder.

Fragile X is caused by a mutation in the FMR1 gene located on the X chromosome and leads to dysregulation of the endocannabinoid pathway including the reduction in endogenous cannabinoids. The disorder affects synaptic function, plasticity, and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. There are about 71,000 patients suffering with FXS in the United States.

The clinical trial will evaluate the efficacy and safety of ZYN002 in children ages 3 to 17 with full mutation Fragile X syndrome. Approximately 200 male and female patients with Fragile X syndrome, confirmed with the full mutation of the FMR1 gene, will be enrolled at approximately 20 clinical sites in the United States, Australia, and New Zealand. Top line results are expected in the second half of 2019.

The primary endpoint of the study is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific Social Avoidance subscale. Key secondary endpoints are the change from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score, the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and improvement in Clinical Global Impression – Improvement at the end of the treatment period.

“If successful, ZYN002 has the potential to become the first product indicated for the treatment of behavioral symptoms of Fragile X syndrome and help address the ongoing needs of the children and families impacted by this syndrome,” said Armando Anido, chairman and CEO of Zynerba.


July 9, 2018
Photo: Armando Anido, chairman and CEO of Zynerba

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