Zynerba Pharmaceuticals Presents Positive Data from Phase 2 INSPIRE Trial in 22q11.2 Deletion Syndrome at the National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit
Zynerba Pharmaceuticals (Nasdaq: ZYNE) showcased a poster titled “An Open-Label Tolerability and Efficacy Study of ZYN002” at the NORD Rare Diseases Summit. The study, involving 20 patients with 22q11.2 deletion syndrome, demonstrated significant improvements in anxiety and behavioral symptoms during a 14-week period. Key outcomes included notable enhancements in the Pediatric Anxiety Rating Scale and other assessment measures. Zygel, the transdermal gel used in the trial, was well tolerated, with mild adverse events reported. Zynerba aims to discuss regulatory pathways with the FDA based on the promising Phase 2 data.
- Significant improvements observed in anxiety-related and behavioral symptoms in children with 22q11.2 deletion syndrome.
- Zygel demonstrated a positive risk-benefit profile and was well tolerated by participants.
- Clinical data supports potential regulatory discussions with the FDA.
- Three patients reported treatment-related adverse events, although all were mild.
- One patient discontinued treatment due to unrelated adverse events.
DEVON, Pa., Oct. 17, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, 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, D.C. A copy of the poster is available on the Zynerba corporate website at www.zynerba.com/publications.
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 Zygel™ in improving anxiety-related and behavioral symptoms in children and adolescents with 22q11.2 deletion syndrome (22q). This study enrolled 20 patients in the U.S. 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). In addition, the majority of patients showed clinically meaningful improvements as demonstrated by the Clinical Global Impression – Improvement (CGI-I). 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.
“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. “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.”
About Zygel
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 transdermally (i.e. through the skin). Recent studies suggest that cannabidiol may modulate the endocannabinoid system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), 22q11.2 deletion syndrome (22q) and autism spectrum disorder (ASD). The Company has received orphan drug designation for cannabidiol, the active ingredient in Zygel, from the FDA and the European Commission in the treatment of FXS and by the FDA for the treatment of 22q. Additionally, Zygel has been designated a Fast Track development program for treatment of behavioral symptoms of FXS.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, 22q11.2 deletion syndrome and autism spectrum disorder. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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Zynerba Contact
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com
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