Zygel continued to be well-tolerated with long-term administration and maintained clinically meaningful improvements in children and adolescents with Fragile X syndrome
Zygel achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments and was generally well-tolerated through 38 weeks of treatment in INSPIRE, a Phase 2 trial with Zygel in children and adolescents with 22q
DEVON, Pa., April 12, 2023 (GLOBE NEWSWIRE) — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, is presenting two posters at the 55th Gatlinburg Conference, being held April 10-13, 2023, in Kansas City, Mo. Copies of the posters are available on the Zynerba corporate website at www.zynerba.com/publications.
“New interim results from the open-label extension trial continue to support the long-term safety and sustained effectiveness of Zygel in children and adolescents with Fragile X syndrome (FXS), with the greatest improvements seen in those with complete methylation of their FMR1 gene, the population for the primary efficacy analysis in our pivotal RECONNECT trial,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “In addition, we believe data from the Phase 2 INSPIRE trial suggest the potential of Zygel as a treatment of anxiety and other behavioral symptoms in children and adolescents with 22q.”
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 demonstrating that in the ongoing long-term safety and efficacy trial of Zygel in children and adolescents with FXS, Zygel was well-tolerated with long-term administration with up to 45 months of exposure. Patients with complete methylation, who match the primary efficacy population in the ongoing confirmatory trial, RECONNECT, achieved and maintained clinically meaningful changes in Social Avoidance over 24 months, further supporting this design enhancement for RECONNECT.
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 other behavioral symptoms in children and adolescents with 22q when added to standard of care.
RECONNECT is a Phase 3 trial of Zygel in patients with FXS ages 3 through 22 years. The trial was designed based upon learnings from the initial Phase 3 trial, CONNECT-FX, which demonstrated potential effectiveness of Zygel in patients with 100% methylation of their FMR1 gene. RECONNECT is actively enrolling participants 3 through 22 years of age. More information about the trial, including how to be contacted regarding potential participation, is available at www.fragilexhelp.com.
Zygel is the first and only pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel, designed to provide consistent 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) 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 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 and 22q11.2 deletion syndrome. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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