CBD-Infused Gel by Zynerba Shows Positive Results In Treating Symptoms of Fragile-X Syndrome
Results are in from a Phase 2 clinical trial of a CBD-based gel formulated to treat a congenital condition known as Fragile-X syndrome, a condition which causes mild to severe intellectual disability. The experimental drug known as ZYN002 is being developed by Zynerba Pharmaceuticals.
Data collected in the study suggests that one year of treatment with the topically applied drug can provide sustained benefit to young patients suffering from the condition.
Fragile X syndrome, also sometimes referred to as FXS, Martin-Bell syndrome, causes delays in talking, anxiety disorders, hyperactive behavior, aggression, impulsivity, sleep disorder, nonsense word repetition, repetitive movements, self-harm, tremors, flaccid muscles, problems with coordination, and in some cases seizures. Visual indications of the condition include large ears, a long face, a prominent jaw and forehead, and flat feet, and sunken chest.
Traditionally, therapy and prescription medications are used to treat the anxiety and mood disorders that come with the condition.
In this study, ZYN002 gel was applied topically to twelve children and adolescents once daily for 12 months. According to the study’s authors, patients experienced significant improvements in emotional and behavioral symptoms.
The details of the study entitled, “Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X Syndrome,” were presented at the 57th Annual Meeting of the American College of Neuropsychopharmacology (ACNP) in Hollywood, Florida.
The Phase 2 trial, called FAB-C (which stands for “Treatment of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD”; an NCT number was not evident), which has so far enrolled 20 FXS patients ages 6 to 17 and is ongoing.
A total of 18 of the 20 patients completed the 12 weeks of treatment, and thirteen patients have progressed to a 24-month extension study. At this time, 11 patients remain in that study.
According to the authors, significant improvements were seen in trial subjects including positive changes in social avoidance and manic/hyperactive behavior, depressive mood, general anxiety, compulsive behavior, irritability, and inappropriate speech.
Interestingly the effects increased over time. A 77.2 percent improvement in social avoidance behaviors was seen at 12 months of treatment compared to 57.9 percent improvement at three months.
“I am encouraged to see that improvements in FXS-associated emotional and behavioral symptoms after 12 months of treatment with ZYN002 are consistent with those seen at three and nine months; these data continue to suggest the potential for sustained responses that may be conserved over extended use of the drug. Improvements in these behaviors may enhance the child’s capacity for interaction and engagement with their peers, families, teachers and caregivers.” — Steven Siegel, MD, PhD, professor and chair of psychiatry and behavioral sciences at Keck School of Medicine
Researchers claim that the CBD-infused gel was well-tolerated by patients and no serious side effects were reported. The most common side effects reported in the study were mild to moderate gastroenteritis (inflammation of the intestines) and upper respiratory tract infection.
Zynerba is now exploring ZYN002’s efficacy in a larger trial and is currently recruiting FXS patients, age 3-18, for clinical trials in Australia, New Zealand, and the United States.
“Our aspirations and expectations are clear: To work closely with the U.S. Food and Drug Administration to expand the opportunity for pharmaceutically-developed CBD treatments that meet their rigorous medical and manufacturing standards, and in doing so, continue toward our goal of addressing significant unmet medical needs in neuropsychiatric disorders.” — Armando Anido, chairman and CEO of Zynerba
Zynerba expects to announce results from its next phase of studies sometime in the second half of 2019. Plans include extending the clinical development of ZYN002 to include patients with autism spectrum disorder and 22q deletion syndrome as well as developmental and epileptic encephalopathy programs.