Zynerba Pharmaceuticals Initiates FAB-C Exploratory Phase 2 Clinical Trial of ZYN002 CBD Gel in Patients with Fragile X Syndrome

Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE), a clinical-stage specialty pharmaceutical company dedicated to the development of innovative transdermal synthetic cannabinoid treatments, today announced that it has initiated the FAB-C (Treatment of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD) exploratory Phase 2 clinical trial of ZYN002 cannabidiol (CBD) gel in children with Fragile X syndrome. ZYN002 CBD gel is the first and only patent-protected, synthetic CBD that is formulated as a permeation-enhanced gel for transdermal delivery.

Fragile X syndrome (FXS) is an autism spectrum disorder and the most common inherited intellectual disability in males and a significant cause of intellectual disability in females. It is caused by a mutation in the Fragile X Mental Retardation gene located on the X chromosome and leads to dysregulation of the endocannabinoid pathway including the reduction in endogenous cannabinoids (2-AG and anandamide). The disorder negatively affects synaptic function, plasticity and neuronal connections and results in a spectrum of intellectual disabilities, social anxiety and memory problems.  In the US, there are about 71,000 patients suffering with FXS.
“Fragile X syndrome is devastating to patients and their caregivers and we are very hopeful that ZYN002 CBD gel can provide relief for this underserved patient group,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We anticipate having top line results for the FAB-C trial late in the first half of 2017.  With an orphan drug designation granted by the Food and Drug Administration for Fragile X syndrome, we look forward to working with the FDA to bring a treatment to patients as fast as possible.”
The FAB-C Trial is an exploratory clinical trial designed to evaluate the safety and efficacy of ZYN002 CBD gel in approximately 16 patients between the ages of 8-17 years with Fragile X syndrome. Dosing will be initiated at 50 mg of CBD in ZYN002 4.2% gel once daily and may be titrated up to 125 mg two times per day during the six-week titration period. Between weeks six to twelve, patients will receive a maintenance dose of 50 mg, 100 mg or 250 mg daily of CBD in ZYN002 4.2% gel.  The primary outcome measures include changes in anxiety, depression and mood as measured by the ADAMS scale, a validated patient reported outcomes questionnaire. Other measurements that will be observed include the Aberrant Behavior Checklist and visual analog scale (VAS) to assess for hyperactivity/impulsivity.