Zynerba Pharmaceuticals Presents Long-Term Safety and Efficacy Data of Zygel in Children and Adolescents with Fragile X Syndrome (FXS) at the International Society for Autism Research Annual Meeting

• Data support the long-term safety and effectiveness of ZYGEL
• Greatest improvements seen in those with complete methylation of their fmr1 gene

Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting an oral podium presentation at the International Society for Autism Research Annual Meeting (INSAR 2022), being held both in-person and virtually May 11-14, 2022, from Austin, TX. A copy of the presentation will be made available on the Zynerba corporate website following the presentation on May 12, 2022, at 4:30 p.m. ET at http://zynerba.com/publications/.

The presentation titled, “Long-Term Safety and Sustained Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Fragile X Syndrome (ZYN2-CL-017),” 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-C_FXS 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-C_FXS Social Avoidance, and demonstrated similar improvements in ABC-C_FXS 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 (range 21 days to 38 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.

“The interim results from this open-label extension trial support the long-term safety and effectiveness of Zygel in children and adolescents with Fragile X syndrome, with the greatest improvements seen in those with complete methylation of their FMR1 gene,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We are excited by the potential that Zygel may offer for patients with FXS.”

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), autism spectrum disorder (ASD), and 22q11.2 deletion syndrome (22q). 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 rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, and 22q11.2 deletion syndrome.