Zynerba Pharmaceuticals stock rose nearly 53% in Thursday trading after the biotech’s marijuana-based gel treatment met key goals in a small, mid-stage study of young people with a rare autism spectrum disorder (ASD). The experimental treatment, ZYN002, was able to reduce anxiety, depression, and various other behavioral symptoms, including hyperactivity, in kids and teenagers with the genetic condition Fragile X syndrome, the company announced.
Fragile X is “the most common known single gene cause of” autism spectrum disorder, according to the National Fragile X Foundation. It can cause both physical and psychological effects including soft skin and large facial features, as well as moderate-to-severe intellectual disabilities and social interaction problems. The condition is far more common among males—about 1 in 4,000 of them have Fragile X syndrome while 1 in 8,000 females have the condition, according to the National Institutes of Health.
Zynerba’s ZYN002 improved patients’ scores on a specific scale used to assess behavioral symptoms such as anxiety and depression compared with placebo, the biotech says. However, Zynerba’s challenges could grow down the line as the mid-stage trial only involved 20 patients; larger studies may not produce the same impressive results.
Still, analysts and investors were clearly impressed by the numbers, reversing a slump Zynerba (zyne) faced in August when this same cannabidiol gel faced consecutive trial failures in treating epilepsy and knee pain from osteoarthritis.
A number of companies, including Zynerba and GW Pharmaceuticals, are racing to win the first-ever FDA approval for a marijuana-based treatment, and many of the therapies focus on neurological and behavioral conditions.