Zynerba Pharmaceuticals Announces Positive Mid-Stage Results of Fragile X Therapy

Rare Daily Staff

Zynerba Pharmaceuticals, a developer of transdermal cannabinoid treatments, reported positive mid-stage results of ZYN002, an experimental therapy for the treatment of fragile X syndrome anxiety and behavioral challenges in pediatric and adolescent patients.

The study successfully met its primary endpoint, achieving a 46 percent improvement in the total score of Anxiety, Depression, and Mood Scale at week twelve compared to baseline.

ZYN002 also achieved clinically meaningful improvements in all measures of the Aberrant Behavior Checklist for Fragile X, which address the key symptoms of condition including social avoidance, temper tantrums, repetitive movements, and hyperactivity.

Fragile X syndrome is an autism spectrum disorder affecting 1 in 4,000 males and 1 in 8,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females.

“Fragile X is a challenging genetic autism spectrum disorder, with complex symptomatology that significantly impacts patients and their families,” said Steven Siegel, professor and chair of psychiatry and behavior sciences at the Keck School of Medicine of USC. “Fragile X is a challenging genetic autism spectrum disorder, with complex symptomatology that significantly impacts patients and their families. Many children with fragile X and their families struggle with the lack of approved drugs to safely treat their symptoms.”

Zynerba said it expects that it will meet with the U.S. Food and Drug Administration in the first half of 2018 with the goal of moving quickly into a pivotal Phase 2/3 program in pediatric and adolescent patients with fragile X syndrome in 2018.

Zynerba’s ZYN002 CBD gel is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced gel and is being studied in children with Fragile X Syndrome, osteoarthritis and in adult epilepsy patients with focal seizures. ZYN002 is a clear, permeation-enhanced gel that is designed to provide controlled drug delivery transdermally with once- or twice-daily dosing.

Cannabinoids are a class of chemical compounds found in the Cannabis plant. The two primary cannabinoids contained in Cannabis are cannabidiol, or CBD, and 9-tetrahydrocannabinol, or THC. The company said transdermal delivery of cannabinoids may have benefits over oral dosing because it allows the drug to be absorbed through the skin directly into the bloodstream.

This avoids first-pass liver metabolism, potentially enabling lower dosage levels of active pharmaceutical ingredients with a higher bioavailability and improved safety profile. Transdermal delivery also avoids the gastrointestinal tract, lessening the opportunity for GI related adverse events and the potential degradation of CBD by gastric acid into THC, which may be associated with unwanted psychoactive effects.

The FDA has granted Zynerba Orphan Drug designation for the use of CBD as treatment of patients with fragile X syndrome.

September 28, 2017