Zynerba Pharmaceuticals (NASDAQ:ZYNE) was up 6% after data from a Phase 2 BRIGHT study showed CBD to have significant potential for patients with Autism Spectrum Disorder. The study looked at some of the classic behaviors associated with autism spectrum disorder (ASD), and whether or not CBD affected them. The open label data will be presented at the virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting.
In the study, researchers used the transdermal gel Zygel for all participants, then analyzed their behavior. All of the subjects are children and adolescents with moderate to severe Autism Spectrum Disorder or ASD. The data reveals that the transdermal patch created a significant improvement in behaviors, based on the Autism Impact Measure or AIM. AIM is a traditionally-used questionnaire for caregivers that assesses behaviors associated with autism. According to a resource originally published in the Journal of Autism and Developmental Disorders, the AIM questionnaire consists of 41 items on a 5-point Likert-type scale, looking at both frequency and impact. The questionnaire examines things like peer interaction, atypical behavior, communication, social reciprocity, and communication. AIM can be used to establish a baseline for people with autism, and allow researchers to better indicate if any improvements have been made and to what degree.
The study’s participants ranged from three to 18 years old, and enrolled all patients had Clinical Global Impression (CGI)-Severity score of ≥4 (moderate or greater) and Aberrant Behavior Checklist-Community (ABC-C) Irritability score ≥18. The study consisted of a 14-week treatment period and a six month extension period, totaling 38 weeks of observation at some level. Researchers looked at caregiver/parental stress, the AIM questionnaire, as well as caregiver reported behavioral problems. The authors of the study concluded that Zygel was beneficial in all of the ASD measures analyzed. The press release stated that there were “clinically meaningful improvements” at 14 weeks out from baseline, with 57% of participants assessed as “very much improved” or “much improved.” Also, the application was very well tolerated by the study’s participants. This is important because people with autism commonly have allergies and/or sensitive bodies, introducing new products may be helpful or harmful depending on what they are.
The majority of participants showed improvement across all three areas of behavioral traits: behavioral, social, and emotional. In the behavioral category, caregivers cited patterns like aggression, refusing to go to school, self-harm, and repetitive phrases as areas of concern. The study reported 69% of subjects considered these behaviors be “improved” after the 14 week treatment period. The social arena showed an improvement for 63% of patients, and 72% of participants claimed an emotional improvement. These assess areas like lack of personal space, fear of new people, minimal social engagement, easily offended, little self-regulation of emotions, and anxiety.
“The Phase 2 BRIGHT trial provides the first clinical evidence of the potential for Zygel to improve behavioral symptomology in a group of highly symptomatic pediatric and adolescent patients with ASD,” said Helen Heussler, FRACP, Associate Professor at Children’s Health Queensland, Medical Director Child Development and principal investigator in the BRIGHT trial. “In these children receiving Zygel, the observed changes from baseline are promising. In particular, the improvements seen in core symptoms of autism, as specifically assessed by the Autism Impact Measure, are of special interest. Though open label, these results are compelling and we look forward to continuing the evaluation of Zygel in ASD in future placebo-controlled clinical trials.”
Zynerba is a global pharmaceutical technology company that produces transdermal cannabinoid therapies for rare or near-rare neuropsychiatric disorders, including Fragile X Syndrome, autism spectrum disorder, different eplieptic conditions, and 22q11.2 syndrome.