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Zynerba to present data on its Zygel CBD Gel to treat autism spectrum disorder and Fragile X Syndrome

Published: 13:00 03 Mar 2020 EST

Zynerba Pharmaceuticals - Zynerba to present data on its Zygel CBD Gel to treat autism spectrum disorder and Fragile X Syndrome
Zygel is designed to provide controlled drug delivery into the bloodstream through the skin

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) will present data this week on use of its flagship Zygel CBD Gel to treat both autism spectrum disorder and the behavioral symptoms of Fragile X Syndrome. 

On Tuesday and Wednesday, the company will present at the American Society for Experimental Neurotherapeutics (ASENT) 2020 Meeting in Bethesda, Maryland.

Zynerba will describe the baseline characteristics of the pediatric and adolescent patients in the fully-enrolled Phase 2 BRIGHT trial of Zygel in children and adolescents with autism spectrum disorder (ASD), indicating that the trial enrolled a broad population of patients with moderate-to-severe ASD.

READ: Zynerba fills enrollment roster for Fragile X trial of Zygel CBD Gel

“ASD is a complex neurodevelopmental disorder characterized by difficulties with behaviors, communication, and social interaction,” said Dr Joseph Palumbo, who will present the data, in statement.

“Pediatric and adolescent patients with ASD may also present with profound clinical anxiety, above the rate seen in neurotypical children, further complicating their condition and treatment regimen. Unfortunately, current ASD management options are restricted to cognitive behavioral therapy and a small number of approved pharmacologic treatments, highlighting the substantial unmet need for novel therapies in this population. We believe that we have enrolled an appropriate population of patients into our well-designed exploratory BRIGHT trial to enable a robust analysis of outcomes to help inform the design of future double-blind, placebo-controlled studies.”

Zynerba also will present two posters on the health burden and diagnostic challenges of Fragile X syndrome (FXS).

The first poster presents health state utility indices that estimate the severity of pediatric disorders including FXS, and the potential benefit of Zygel in children and adolescents with FX. 

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. 

The second poster speaks to the initial family experience in FXS, expanding upon the existing knowledge of patient presentation, diagnosis and understanding of FXS; the protracted journey to diagnosis; and the high prevalence of comorbid conditions.

“The mean health state utility index score for FXS in this seminal analysis was calculated to be 0.57, estimating a significant disease-related impact on HRQoL in FXS which may be as robust as, or perhaps even more impactful, than that described in the published literature for other debilitating pediatric conditions in measures of HUI”, said Dr Palumbo. “We will work to confirm our initial observations in future analyses.”

Zygel, a patented CBD-formulated clear gel, is designed to provide controlled drug delivery into the bloodstream through the skin. Some recent studies have suggested that neuropsychiatric conditions such as Fragile X Syndrome are associated with a disruption in the endocannabinoid system, and CBD may improve certain core social and behavioral symptoms by modulating that system.

Contact the author: patrick@proactiveinvestors.com

Follow him on Twitter @PatrickMGraham




 

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