Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) said it has presented a poster at the 2022 National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit being held October 17-18, 2022, in Washington DC.
The poster, titled ‘An Open-Label Tolerability and Efficacy Study of ZYN002 (Cannabidiol) Administered as a Transdermal Gel to Children and Adolescents with 22q11.2 Deletion Syndrome (INSPIRE)’, reports data from the first 14-week treatment period that suggests a positive risk-benefit profile for Zynerba’s cannabidiol-formulated gel Zygel in improving anxiety-related and behavioral symptoms in children and adolescents with 22q11.2 deletion syndrome (22q).
"We are encouraged by the results from our Phase 2 INSPIRE study, where we saw statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments, including significant improvement in anxiety as measured by the clinician-rated PARS-R scale," said Armando Anido, chairman and chief executive officer of Zynerba in a statement.
READ: Zynerba Pharmaceuticals receives new US patent for its cannabidiol formulated gel Zygel
"We believe the Phase 2 data suggests the potential of Zygel for the treatment of anxiety and behavioral symptoms in children and adolescents with 22q, and we look forward to discussing the regulatory path forward with the FDA later this half," he added.
The study enrolled 20 patients in the US and Australia. Statistically significant improvements from baseline were seen in the Pediatric Anxiety Rating Scale (PARS-R), the total score and all five subscales of the Anxiety, Depression and Mood Scale (ADAMS) and all five subscales of the Aberrant Behavior Checklist - Community (ABC-C), Zynerba noted.
In addition, the majority of patients showed clinically meaningful improvements as demonstrated by the Clinical Global Impression - Improvement (CGI-I), the company added.
Zygel was shown to be well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials. Three patients reported treatment-related adverse events which were all mild application site adverse events. One patient discontinued treatment due to adverse events not related to Zygel.
A copy of the poster presented at the summit is available on the Zynerba corporate website
Zygel is the first and only pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream through the skin.
Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders.
Contact the author at jon.hopkins@proactiveinvestors.com