Healthcare Business

Zynerba Cannabinoid Investors Must Now Refocus on Treating Autism

Zynerba Pharmaceuticals Inc. (NASDAQ: ZYNE) has disclosed that its cannabis-based gel did not see statistically significant results from its most recent study. As a result, Zynerba shares were practically halved on Tuesday. At this point, investors may have to shift their focus to Zynerba’s pipeline for any hope.

The company announced topline results from the 14-week pivotal CONNECT-FX trial, a clinical study of cannabidiol (CBD) in children and adolescents with fragile X syndrome, which was evaluating the safety and efficacy of Zygel. Ultimately, Zygel did not achieve statistical significance compared with the placebo for the primary endpoint, nor in three key secondary endpoints.

First, Zygel did not reach the endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist. The unreached secondary endpoints were the change from baseline to the end of the treatment period in the Irritability subscale score, the Socially Unresponsive/Lethargic subscale score, and Improvement in Clinical Global Impression.

However, Zygel was very well tolerated in CONNECT-FX, and the safety profile was consistent with previously released data from other Zygel clinical trials.

Looking ahead, Zynerba plans to meet with the FDA to discuss the study results as soon as possible to determine a path forward for Zygel.

Note that with CONNECT-FX out of the picture (for now at least), Zynerba investors will have to refocus on other aspects of the company’s pipeline. Zynerba has the BELIEVE program for developmental and epileptic encephalopathies, BRIGHT program for autism spectrum disorder and INSPIRE program for deletion syndrome.

Zynerba stock traded down about 50% to $3.28 on Tuesday, in a 52-week range of $2.55 to $15.69. The consensus price target is $17.50.