Zynerba Pharmaceuticals Inc.'s cannabis-derived drug Zygel improved symptoms among children and adolescents with autism spectrum disorder in a mid-stage clinical trial.

Autism spectrum disorder, or ASD, is a condition related to brain development that affects communication and behavior. This condition starts in early childhood and affects patients' functioning in society as they may have trouble with social interaction.

In the U.S., approximately 1 million children and adolescents between five and 17 years old are diagnosed with ASD.

Zygel is a gel formulation of a chemical compound called cannabinoid, which is derived from the plant Cannabis sativa, designed to be delivered through the skin and into the bloodstream.

The trial, named Bright, involved 37 patients with ASD who were given one of two different doses of Zygel depending on their weight. Only 28 patients completed the trial, which ran for 14 weeks.

At week 14, the patients experienced a mean improvement of 39.1% in irritability, 42.5% for inappropriate speech and 39.1% for stereotypy — repetitive movements or utterance — compared to the start of the trial. Scores for social withdrawal and hyperactivity also saw a mean improvement of 36.4% and 35.6%, respectively.

Devon, Pa.-based Zynerba said the trial also determined that Zygel was very well tolerated and safe to use, with no severe or serious adverse events reported.

Zygel has also been investigated for the treatment of epilepsy, showing benefits in terms of reducing seizure episodes, but with some adverse events arising from the treatment.

The cannabinoid gel received fast-track designation in the U.S. in May 2019 to treat behavioral symptoms associated with a rare genetic disorder called fragile X syndrome.

Zynerba is a specialty pharmaceutical company focused on providing transdermal cannabinoid therapies for people with rare and near-rare neuropsychiatric disorders such as ASD, fragile X syndrome and 22q11.2 deletion syndrome.