Zynerba Pharmaceuticals Inc. (NASDAQ: ZYNE) shares dropped on Wednesday after the firm announced results from its midstage epilepsy trial. Specifically, the top-line results are from the Phase 2 Believe 1 of Zygel (ZYN002).

The trial assessed the safety and efficacy of Zygel in developmental and epileptic encephalopathies (DEE), a heterogeneous group of rare pediatric epilepsy syndromes, including Dravet syndrome and Lennox-Gastaut syndrome. What stands out here is that Zygel is a transdermal cannabinoid therapy.

Some quick background: DEE is characterized by the presence of multiple focal and generalized seizure types and severe cognitive and behavioral impairment.

The most common and debilitating seizure types in people with epilepsy are focal impaired-awareness and convulsive seizures. Patients who experienced these seizure types achieved 44% to 58% monthly median reductions in seizures compared to baseline from month two to month six of treatment with Zygel. Further, qualitative assessments by caregivers in the study demonstrate that use of Zygel may result in improved sociobehavioral and cognitive symptoms of DEE. Zygel was also well-tolerated in this study.

Of the 46 patients, 33 (72%) had focal impaired-awareness seizures or convulsive seizures at baseline. Compared to baseline seizure frequency, these patients experienced a greater than 44% median reduction in seizures from month two onward using monthly seizure frequency normalized to 28 days. Also, 55% of patients with focal impaired-awareness or convulsive seizures experienced a greater than 50% median reduction in seizures at month six of treatment with Zygel.

Ingrid Scheffer, A.O., MBBS, Ph.D., FRACP, professor and chair of Paediatric Neurology Research, University of Melbourne, and an investigator in the Believe 1 trial, commented:

The data from the Believe 1 clinical trial are promising and suggest that Zygel may reduce seizure frequency in many types of difficult to treat developmental and epileptic encephalopathies and improve important behavior deficits, alertness, social interactions, and enable the child to be well enough to attend school more consistently. DEE are the most challenging and poorly controlled epilepsy disorders with many symptoms that adversely affect patient and family function. I believe that this drug holds promise as a potential treatment for DEE.

Shares of Zynerba traded down about 14.5% Monday morning, at $9.66 in a 52-week range of $2.75 to $16.47. The consensus price target is $22.67.

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