Zynerba presents long-term safety, efficacy data of Zygel

Zynerba Pharmaceuticals is presenting an oral podium presentation at the International Society for Autism Research Annual Meeting, being held both in-person and virtually May 11-14, 2022, from Austin, TX. The presentation titled, "Long-Term Safety and Sustained Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Fragile X Syndrome," includes data from 240 patients and demonstrates the long-term safety and efficacy of Zygel in children and adolescents with FXS. The study includes patients who entered the trial from two completed trials with Zygel: CONNECT-FX, a randomized, double-blind, placebo-controlled Phase 3 trial, and FAB-C, the initial open-label Phase 2 trial in FXS. The efficacy data were evaluated through 15 months of treatment and demonstrated improvements in both ABC-CFXS Social Avoidance and Irritability subscales. Patients with complete methylation of their FMR1 gene who completed CONNECT-FX and matched the primary efficacy population in the ongoing confirmatory RECONNECT trial achieved and maintained clinically meaningful change in ABC-CFXS Social Avoidance, and demonstrated similar improvements in ABC-CFXS Irritability subscale scores. These finding support the design enhancements that were made for RECONNECT. Zygel was well tolerated in patients, with a median duration of exposure of 16 months. Adverse events potentially related to Zygel were reported in 12.9% of patients; the most common reported in 6.7% of patients was application site pain which resolved quickly and was mild in the majority of cases.