Benzinga | Sep 9, 2021 07:16AM EDT

Zynerba Pharmaceuticals Presents Longer Term Tolerability And Efficacy Data Of Zygel In Children and Adolescents with Autism Spectrum Disorder at The Society for the Study of Behavioural Phenotypes Conference 2021

Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today presented data from the Phase 2 BRIGHT trial describing tolerability and efficacy of Zygel (cannabidiol formulated in a transdermal gel [ZYN002]) in children and adolescents with autism spectrum disorder (ASD) over a longer term, 38-week treatment period. These data were presented as an oral presentation at The Society for the Study of Behavioural Phenotypes (SSBP) Conference 2021 being held virtually from September 9-10, 2021. A copy of the presentation is available on the Zynerba corporate website at http://zynerba.com/publications/.

Helen (Honey) Heussler, MBBS FRACP MRCPCH, DM, 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])." The presentation shows that through 38 weeks of treatment, the BRIGHT trial provides 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 compared to baseline.

"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 Joseph M. Palumbo, M.D., LFAPA, MACPsych, Chief Medical Officer of Zynerba. "These data are encouraging, and if confirmed, suggest meaningful therapeutic utility in ASD."

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 stable standard of care. Eighteen of the 27 patients that completed week 14 (Period 1) demonstrated ?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). In the 18 patients who completed treatment through Period 2, Zygel showed improvement in all the Aberrant Behavior Checklist-Community (ABC-C) subscale scores (51% to 61% across domains; P<0.0001), as well as improvements in the Parent-Rated Anxiety Scale-ASD score (42%; P<0.0001), the Autism Parenting Stress Index (40%; P<0.0001) and the Autism Impact Measure (19% to 36% across domains; P?0.0008), relative to baseline.

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 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.

In addition, Randi J. Hagerman, M.D., 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 FXS, on September 10, 2021, at 2:15 p.m. 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 at http://zynerba.com/publications/.