Zynerba Pharmaceuticals Inc. (NASDAQ:ZYNE) announced on Monday that a positive meeting with the FDA would soon result in clinical trials for their CBD treatment ZYN002. ZYN002 is a transdermal CBD gel used to treat Fragile X Syndrome (FXS) in children and adolescents. Last September, ZYN002 successfully passed its open label exploratory Phase 2 Trial, and Zynerba intends to begin their double-blind clinical study in the coming months.
There are currently no FDA-approved treatments for Fragile X and its underlying defects. Zynerba is the first to introduce a CBD gel as possible treatment. With current approval, Zynerba intends to keep their focus on providing care and attention to younger patients and their families.
“We are pleased with the outcome of the discussion and the guidance on trial design,” Armando Anido, chairman and CEO of Zynerba, said in a statement. “We now have a path forward to advance the development of ZYN002 to an NDA. We look forward to…potentially providing FXS patients and their families an effective and well tolerated therapy to treat the complex behavioral symptoms of Fragile X syndrome.”
Fragile X is a genetic condition affecting the genes on the X-chromosome in patients with the disorder. There is no cure, but current treatments address the common symptoms, including hyperactivity, anxiety, and seizures. Previous clinical tests conclude that CBD can be used as an effective treatment for epilepsy and other seizure-disorders, including FXS, but pharmaceutical companies still face many limitations with CBD.
frZYN002 is Zynerba’s answer to these limitations. According to their website, ZYN002 is the first and only pharmaceutically-produced, permeation-enhanced CBD transdermal gel. Transdermal gels may provide a more controlled treatment delivery that reduces the risk of unwanted, psychoactive side effects. One study showed that stomach acids can degrade CBD into THC, creating the unwanted side effects. Applying a CBD gel directly to the skin allows the drug to be absorbed into the bloodstream without having to pass through the digestive tract.
The clinical trials, scheduled to start sometime mid-year, hope to examine these purported limitations. Approximately 200 pediatric and adolescent patients from the United States, Australia, and New Zealand will participate in the double-blind study, which has many endpoints measuring observable and relevant behaviors and reactions to treatment. According to the a statement, more details will be released when trials begin in the next few months.