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Zynerba Pharmaceuticals Announces Positive Long-Term Data from Phase 2 INSPIRE Trial in 22q11.2 Deletion Syndrome at the Annual Meeting of the American College of Neuropsychopharmacology
Zygel™ achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments and was generally well-tolerated through 38 weeks of treatment
DEVON, Pa., Dec. 05, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, presented positive long-term 38-week data from the Phase 2 INSPIRE trial with Zygel in children and adolescents with 22q11.2 deletion syndrome (22q). The data were presented in a poster at the 61st Annual Meeting of the American College of Neuropsychopharmacology, being held December 4-7, 2022 in Phoenix, Arizona. 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),” shows that through 38-weeks of treatment, statistically significant improvements were seen in children and adolescents treated with Zygel in the Pediatric Anxiety Rating Scale (PARS-R), all five scales of the Anxiety, Depression and Mood Scale (ADAMS), and all five subscales of the Aberrant Behavior Checklist – Community (ABC-C). These results are consistent with the previously reported 14-week treatment data suggesting a positive risk-benefit profile for Zygel in improving anxiety-related and behavioral symptoms in children and adolescents with 22q when added to a stable standard of care.
“The 38-week data from the Phase 2 INSPIRE trial suggest children and adolescents with 22q who received Zygel over a longer term continued to show improvement in anxiety-related and behavioral symptoms,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “These data provide additional support and reinforce our belief in the potential of Zygel for the treatment of anxiety and behavioral symptoms in children and adolescents with 22q.”
The INSPIRE trial enrolled 20 patients in the U.S. and Australia. Seventeen of the 20 patients completed week 14 (Period 1). Patients with a ≥35% improvement in the ABC-C irritability subscale were allowed to continue treatment for an additional 24 weeks. Thirteen patients continued treatment, with 12 patients completing week 38 (Period 2). At the end of both Period 1 and Period 2, statistically significant improvements from baseline were seen in the PARS-R, all five subscales of the ADAMS, and all five subscales of the ABC-C.
Zygel was generally well tolerated, and the safety profile was consistent with data from previous Zygel clinical trials. Over the 38-week treatment period, three patients reported treatment-related adverse events which were all mild application site adverse events. Three patients experienced serious adverse events unrelated to Zygel and one patient discontinued due to an adverse event unrelated to 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 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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