ZYNERBA PHARMACEUTICALS, INC. Zynerba Pharmaceuticals, Inc. 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. 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 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.
