Zynerba Pharmaceuticals Inc. (NASDAQ: ZYNE) shares saw a huge gain early on Thursday after the firm announced positive results from its mid-stage cannabinoid trial. Specifically, the firm was evaluating ZYN002 cannabidiol (CBD) gel in pediatric and adolescent patients with fragile X syndrome (FXS).

For some quick background: fragile X syndrome is an autism spectrum disorder affecting one in 4,000 males and one in 8,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females.

The study successfully met its primary endpoint, achieving a 46% improvement in the total score of Anxiety, Depression, and Mood Scale (ADAMS) at week 12 compared to the baseline. ZYN002 also achieved clinically meaningful improvements in all measures of the Aberrant Behavior Checklist for Fragile X (ABC-FXS), which address the key symptoms of FXS including social avoidance, temper tantrums, repetitive movements and hyperactivity.

Considering these results, Zynerba anticipates that it will meet with the U.S. Food and Drug Administration (FDA) in the first half of 2018 with the goal of moving quickly into a pivotal Phase 2/3 program in pediatric and adolescent patients with FXS in 2018. The FDA has granted Zynerba Orphan Drug designation for the use of CBD as treatment of patients with FXS.

Steven Siegel, M.D., Ph.D., Professor and Chair, Psychiatry and Behavior Sciences, Keck School of Medicine of USC, commented:

The data from the FAB-C trial are very exciting and demonstrate that ZYN002 may have a profound effect on improving many of the most disabling symptoms of Fragile X, such as anxiety and difficult behaviors. Fragile X is a challenging genetic autism spectrum disorder, with complex symptomatology that significantly impacts patients and their families. Many children with Fragile X and their families struggle with the lack of approved drugs to safely treat their symptoms. This study suggests that ZYN002 is ready for the next phase of development, and I believe that this drug holds great promise as a potential treatment for these very difficult-to-treat symptoms.

Shares of Zynerba closed Wednesday up 6% at $6.19, with a consensus analyst price target of $8.14 and a 52-week range of $5.42 to $25.95. Following the announcement the stock was up about 90% at $11.75 in early trading indications Thursday.

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