Manufacturer of transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), completed enrollment for the 14-week Phase 2 INSPIRE trial of Zygel (Transdermal CBD Gel) in the treatment of behavioral symptoms of 22q11.2 deletion syndrome in children and adolescents. The company expects top-line data from this trial mid-year 2022.
“The completion of enrollment in our INSPIRE trial is an important step forward in advancing Zygel as a potential new treatment option for patients suffering from 22q,” stated Armando Anido, chairman and CEO of Zynerba. “Pending results from the INSPIRE trial and subsequent discussion with the FDA on the regulatory path forward, we plan to advance our clinical development program in 22q with Zygel. We are thankful for the combined efforts of our researchers, investigators and the patient and caregiver community.”
The 14-week INSPIRE trial is an open-label, multi-dose, Phase 2 clinical trial designed to evaluate the efficacy and safety of Zygel in children and adolescents with genetically-confirmed 22q. Enrolled patients receive weight-based doses of 250 mg daily or 500 mg daily of Zygel. A total of 20 patients have been enrolled in the INSPIRE trial.
According to Zynerba, 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.
Recent studies suggest that cannabidiol may modulate the endocannabinoid system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel has been granted orphan drug designation by the FDA in the treatment of Fragile X syndrome and 22q. Additionally, the FDA has designated Zygel a Fast Track development program for the treatment of behavioral symptoms of FXS.
22q is caused by a small missing piece of the 22nd chromosome. Anxiety is among the most common neuropsychiatric symptoms of 22q. Children with 22q also experience withdrawn behavior, ADHD, cognitive impairment, and autism spectrum disorder that affect communication and social interaction. Later in life, they are at an increased risk of developing mental illnesses such as schizophrenia.
It is estimated that 22q has a prevalence rate of approximately one in 4,000 people in the U.S.1,2, suggesting that there are approximately 83,000 people living with 22q in the U.S.
