Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc. has presented data from the Phase 2 BRIGHT trial describing the tolerability and efficacy of its Zygel transdermal cannabinoid therapy in children and adolescents with autism spectrum disorder (ASD) over a longer-term, 38-week treatment period.
The company said the data were presented as an oral presentation at The Society for the Study of Behavioural Phenotypes (SSBP) Conference 2021 which is being held virtually from September 9-10, 2021. A copy of the presentation is available on the Zynerba corporate website.
Helen (Honey) Heussler, associate professor, faculty of medicine, Child Health Research Centre, University of Queensland, Australia, delivered the oral presentation titled: “Longer Term Tolerability and Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Autism Spectrum Disorder (ASD): An Open-Label Phase 2 Study (BRIGHT [ZYN2-CL-030]).”
READ: Zynerba Pharmaceuticals says results of Phase 2 BELIEVE open-label study of Zygel described as positive in medical journal
Zynerba said the presentation shows that through 38 weeks of treatment, the BRIGHT trial has provided initial evidence suggesting a positive benefit-risk profile for Zygel when administered in addition to stable standard of care in children and adolescents with moderate-to-severe ASD.
Furthermore, in patients who completed the 38-week treatment period, statistically significant improvements compared to baseline were sustained in all efficacy measures of ASD.
“Current management options for ASD are restricted to behavioral therapies and a limited number of approved pharmacologic treatments, highlighting the substantial unmet need for novel therapies in this difficult-to-treat population,” said Dr. Joseph Palumbo, Zynerba’s chief medical officer, in a statement. “These data are encouraging, and if confirmed, suggest meaningful therapeutic utility in ASD."
The company said the open-label Phase 2 study evaluated the safety, tolerability, and efficacy of Zygel in children and adolescents aged 3-17 years with ASD in addition to a stable standard of care. Eighteen of the 27 patients who completed week 14 (Period 1) demonstrated a 35% improvement in the Aberrant Behavior Checklist-Community (ABC-C) irritability subscale at week 14 and were allowed to continue treatment for an additional 24 weeks (Period 2).
Zynerba said Zygel was generally well-tolerated, and the safety profile was consistent with data from previous Zygel clinical trials. Adverse Events (AEs) were mild (80%) or moderate (20%). Treatment-related AEs were reported in 19% of patients; most were mild and transient. One patient was discontinued due to application-site reaction. No serious or severe AEs or clinically significant changes in vital signs, laboratory tests, or ECG parameters were reported during the study.
Meanwhile, Dr. Randi Hagerman, medical director, and endowed chair in Fragile X Research at UC Davis MIND Institute and distinguished professor at the Department of Pediatrics at UC Davis School of Medicine, will present previously announced data from the CONNECT-FX trial, a randomized, double-blind, placebo-controlled trial which assessed the efficacy and safety of Zygel as a treatment for the behavioral symptoms of Fragile X Syndrome, on September 10 at 2:15 pm at SSBP.
A copy of the presentation, titled “A Pivotal Study of ZYN002 Cannabidiol (CBD) Transdermal Gel in Children and Adolescents With Fragile X Syndrome [CONNECT-FX (ZYN2-CL-016)],” will be available on the Zynerba corporate website following the presentation.
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