Zynerba Pharmaceuticals, Inc. (ZYNE) announced positive top-line results from the exploratory, open-label Phase 2 BRIGHT study (An Open-Label Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Autism Spectrum Disorder) trial. Autism Spectrum Disorder is a developmental disorder that affects communication and behavior in approximately one million pediatric and adolescent patients between the ages of five and 17 in the U.S.
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The trial was designed to assess the safety, tolerability, and efficacy of the Zynerba drug called Zygel in pediatric and adolescent patients with autism spectrum disorder (ASD). Zygel was administered to patients with moderate-to-severe symptoms of ASD as add-on therapy to their standard of care utilizing a variety of efficacy assessments.
“We are very encouraged by the compelling top-line results of the BRIGHT trial and we expect to meet with the FDA to discuss the clinical pathway for developing Zygel for the treatment of behavioral symptoms of ASD in the second half of this year,” said Armando Anido, Zynerba’s Chairman and Chief Executive Officer. “Our goal is to develop Zygel for patients suffering from debilitating neuropsychiatric disorders including ASD, Fragile X syndrome, 22q and DEE. I want to thank the patients, families, physicians, clinical staff, and the Zynerba team for their support of this key study in ASD.”
Key findings from the trial disclosed today include:
- All five subscales of the Aberrant Behavior Checklist – Community (ABC-C) as well as the Parent Rated Anxiety Scale – Autism Spectrum Disorder (PRAS-ASD) showed both statistically significant and clinically meaningful improvements at 14 weeks of treatment from baseline;
- The results observed in other efficacy outcome measures, including Clinical Global Impressions – Improvement scale (CGI-I), support the subscale results observed in the ABC-C;
- Zygel was well tolerated in this trial with no serious or severe adverse events reported.
“I am very impressed with the improvements my patients made over the 14-week treatment period while receiving Zygel; the reduction in irritability, communication deficits, and repetitive movements were especially noteworthy since some of these are core autistic behaviors,” said Helen Heussler, FRACP, Associate Professor at Children’s Health Queensland, Medical Director Child Development and principal investigator in the BRIGHT trial. “The magnitude of effect on autistic behaviors in this trial is significant, including hyperactivity and stereotypy, which are among the most difficult behaviors to improve with therapeutic intervention. The results of this study strongly suggest the potential of this drug as an important treatment for ASD and I look forward to participating in future clinical studies with Zygel.”