15 Oct 20
New Data Describing Statistically Significant Results from the Phase 2 BRIGHT Trial in Patients with Autism Spectrum Disorder (ASD) Presented at the Virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting
Devon, PA, October 15, 2020 – Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting a poster describing data from the 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 further demonstrating the potential for Zygel™ (ZYN002) to improve the core behavioral symptoms of autism when administered in addition to stable standard of care in children and adolescents with moderate-to-severe ASD. These open label data are being presented at the virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting. These data will also be presented as an oral presentation during the “Research Pipeline: New Findings on Diagnostic and Therapeutics” session of the virtual American Academy of Child and Adolescent Psychiatry (AACAP) 2020 Annual Meeting on Friday October 23rd, 2020.
A copy of the poster entitled, “Tolerability and Efficacy of ZYN002 Cannabidiol (CBD) Transdermal Gel in Children and Adolescents With Autism Spectrum Disorder: An Open-Label Phase 2 Study [BRIGHT (ZYN2-CL-030)]” is available on the Zynerba corporate website at http://zynerba.com/publications/.
“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.”
The BRIGHT Phase 2 trial is an exploratory, single-center, open-label Phase 2 study evaluating the safety and tolerability and efficacy of Zygel in children and adolescents with ASD who are 3 to <18 years old. The study enrolled patients with Clinical Global Impression (CGI)-Severity score ≥4 (moderate or greater) and Aberrant Behavior Checklist-Community (ABC-C) Irritability score ≥18. The primary objective of the trial was to evaluate the safety and tolerability of Zygel for up to 38 weeks (14-week treatment period and a 6 month extension period). Secondary objectives comprised evaluation of the efficacy of Zygel in the treatment of symptoms of ASD, including measuring parental/caregiver stress, Autism Impact Measure (AIM), and caregiver reported behavioral problems. The results provided in the poster are after 14 weeks of treatment with Zygel.
New data presented today include that patients receiving Zygel in this study achieved statistically significant caregiver-reported improvements compared to baseline across all subscales of the AIM, which was designed to track incremental change in frequency and impact of core ASD symptoms: Atypical behavior (p<0.001), Communication (p<0.001), Peer Interaction (p<0.001), Repetitive Behavior (p<0.001), and Social Reciprocity (p=0.0053).
Figure 1. Statistically Significant Improvements in Autism Impact Measure Scores
In addition, statistically significant improvements compared to baseline were observed at week 14 of treatment with Zygel in the Autism Parenting Stress Index (p<0.0001).
Figure 2. Statistically Significant Improvements in PRAS-ASD, APSI, and CGI-I
Zynerba also measured notable improvements in behaviors utilizing the Qualitative Caregiver Behavioral Problems Survey after 14 weeks of study drug. Clinically meaningful improvements were observed by a majority of surveyed caregivers in behavioral, social and emotional behavioral problems.
Figure 3. Notable Improvements in the Qualitative Caregiver Behavioral Problems Survey at Week 14
The authors of the poster concluded that:
- 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;
- Zygel showed improvement in all ASD measures (ABC-C, AIM, PRAS-ASD, CGI and Qualitative Caregiver Assessments);
- Further controlled studies are warranted in this difficult-to-treat population.
These results supplement the topline data initially disclosed by the Company in May 2020 (Press release), including that:
- All five subscales of the ABC-C showed both statistically significant (p<0.0002) and clinically meaningful improvements at 14 weeks of treatment from baseline.
- The results of other efficacy assessments reinforce the results demonstrated in the ABC-C, including a mean improvement of 46% at week 14 from baseline as measured by the Parent Reported Anxiety Scale for ASD (PRAS-ASD; p<0.0001) and 57% of patients were assessed as “very much improved” or “much improved” at week 14 as measured by the Clinical Global Impression – Improvement scale (CGI-I).
- Zygel was very well tolerated in this trial and the safety profile was consistent with previously released data from other Zygel clinical trials. No serious or severe adverse events were reported.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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