Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE), the provider of cannabinoid therapies for neuropsychiatric disorders, has unveiled details of new clinical data for its flagship Zygel CBD gel, a treatment for Fragile X syndrome, which will be showcased at the American Society for Experimental Neurotherapeutics (ASENT) 2020 meeting in Bethesda, Maryland next month.
The biotech’s first poster presentation entitled “Phase 2 BRIGHT – An open-label tolerability and efficacy study of Zygel (ZYN002) administered as a transdermal gel to children and adolescents with autism spectrum disorder – Baseline characteristics, will take place on March 4 from 1pm to 3pm. Its second poster called “Post-hoc Analysis – An open-label study of transdermal cannabidiol (ZYN002) for the treatment of Fragile X syndrome in children and adolescents: Estimating health state utility scores” will also be unveiled.
“These new data are important and help further elucidate the significance of the conditions we are treating in our clinical trials and the potential for positive impact of Zygel, if approved by the FDA,” said Dr. Joseph Palumbo, Zynerba’s chief medical officer.
“We are committed to developing therapeutic interventions for children and adolescents with a variety of debilitating neuropsychiatric conditions including Fragile X syndrome and autism spectrum disorder,” Palumbo added.
Zynerba has already achieved its patient screening target for its upcoming trial of Zygel as a treatment for Fragile X syndrome.
The study, called CONNECT-FX, is designed to test the safety and efficacy of Zygel in children and adolescents with the genetic condition. Zynerba plans to move forward with the 14-week trial at clinical sites in the US, Australia and New Zealand, now with more than 200 patients enrolled.
Top-line results are expected later this year, and if they’re positive, Zynerba expects to meet with the US Food and Drug Administration to submit a New Drug Application. If all goes according to plan, Zygel could be approved to treat Fragile Z by mid-2021.
Zygel, a patented CBD-formulated clear gel, is designed to provide controlled drug delivery into the bloodstream through the skin. Some recent studies have suggested that neuropsychiatric conditions such as Fragile X Syndrome are associated with a disruption in the endocannabinoid system, and CBD may improve certain core social and behavioral symptoms by modulating that system.