DEVON, Pa., Feb. 03, 2020 (GLOBE NEWSWIRE) — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that it has successfully achieved its patient screening target in its 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial assessing the efficacy and safety of Zygel™ CBD Gel in children and adolescents ages three to 17 with full mutation Fragile X syndrome (FXS). As a result, all clinical sites have been informed that screening has now closed and the Company expects to meet or exceed its target of 204 patients randomized. Topline results are expected to be announced late in the second quarter of 2020.
“This is an important milestone for patients with Fragile X syndrome and their caregivers as we move toward completion of enrollment in this pivotal trial,” commented Armando Anido, Chief Executive Officer of Zynerba. “I would like to thank all of our clinical investigators and their staff for their assistance thus far. Through strict entry criteria and overall trial design, we expect to enhance the study’s ability to demonstrate a strong signal of activity and minimize response variability. Having now achieved our screening target, we remain confident that we will announce topline results late in the second quarter of this year.”
The multi-national randomized, double-blind, placebo-controlled, 14-week CONNECT-FX trial is assessing the efficacy and safety of Zygel for the treatment of children and adolescents with FXS. At least 204 male and female patients with Fragile X syndrome, confirmed with the full mutation of the FMR1 gene, are being enrolled at clinical sites in the United States, Australia, and New Zealand. Patients are being been randomized 1:1 to either trial drug or placebo. The primary endpoint is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale. The key secondary endpoints are the change from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score, the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and improvement in Clinical Global Impression – Improvement (CGI-I) anchored to behavioral symptoms of FXS evaluated at the end of the treatment period.
The Company expects to disclose topline results of this study late in the second quarter of 2020. If the results are positive, the Company expects to meet with the U.S. Food and Drug Administration (FDA) to determine acceptability of the data as a basis to submit its New Drug Application (NDA) for Zygel in FXS in the second half of 2020, with potential approval by mid-year 2021.
Patients must complete screening prior to randomization into a clinical trial to determine if the candidate is appropriate for inclusion in the trial based on certain inclusion and exclusion criteria. These may include baseline assessment of behavior severity, any concomitant medications including over-the-counter (OTC) medications the patient is taking, the patient’s medical history including genetic confirmation of their FXS diagnosis, and results of neurological, physical, lab, and diagnostic exams.
Zygel (CBD gel) is the first and only pharmaceutically-manufactured CBD 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 Fragile X Syndrome (FXS) and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system and improve certain core social and behavioral autism-related symptoms, including social avoidance and anxiety.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in 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, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
February 03, 2020