DEVON, PA — Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, announced positive top line results from the exploratory, open label Phase 2 BRIGHT (An Open-Label Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Autism Spectrum Disorder) trial.
The trial was designed to assess the safety, tolerability and efficacy of 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. Key findings from the trial disclosed 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.
“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.”
The 14-week exploratory, open label Phase 2 BRIGHT trial enrolled 37 patients with ASD at a single clinical site. The trial was designed to evaluate the safety, tolerability and efficacy of Zygel in children and adolescents ages three to 17 with ASD as confirmed by DSM-5 diagnostic criteria. Enrolled patients received weight-based doses of 250 mg or 500 mg daily of Zygel.
Patient Disposition and Baseline Demographics
Thirty-seven (37) patients were enrolled in the trial and are included in the safety analysis. One patient was lost to follow up with no post-treatment efficacy evaluation and, as a result, thirty-six (36) patients are included in the efficacy analyses. Twenty-eight (28) patients completed the 14-week trial. The discontinuation rate is consistent with other trials in ASD.
The mean age of patients enrolled in the trial was 9.2 years and thirty-four (92%) of the patients were male. Patients weighed between 15 and 108 kilograms (mean=41.6; median=30.2). The mean time to diagnosis in this population was 5.4 years. Using the Autism Diagnostic Observation Schedule (ADOS-2), 94% of enrolled patients had moderate-to-severe symptoms of ASD. The mean baseline ABC-C Irritability subscale score of 30.3 further supports the severity of the enrolled patient population. Ninety-two percent (92%) of patients entered the trial with the use of at least one underlying medication. Sixty-five percent (65%) of patients were on at least one psychotropic medication, for example, anti-depressants, anxiolytics and antipsychotics.
Top-line Efficacy Results
The trial evaluated multiple efficacy assessments, including the ABC-C, PRAS-ASD, Autism Parenting Stress Index, Autism Impact Measure (AIM), and Clinical Global Impression – Severity (CGI-S) and Improvement (CGI-I). The ABC-C irritability subscale was used as the basis for approval for the two atypical antipsychotics indicated for ASD.
Results from each of the subscales of the ABC-C after 14 weeks of treatment with Zygel are as follows:
“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.”
Safety and Tolerability
Zygel was very well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials. Less than half (49%) of the patients experienced any adverse event (whether unrelated or related to study drug), all of which were mild (75%) or moderate (25%). Only 14% of patients experienced an adverse event that was deemed to be treatment-related, all of which were application site-related; most were mild and transient. There were no severe or serious adverse events reported during the study. Eighteen (18) patients who completed the BRIGHT trial have rolled into the open label extension.
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