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Zynerba Plunges On Midstage Zygel Data In Epileptic Patients

Shanthi Rexaline

Shares of cannabinoid drug maker Zynerba Pharmaceuticals Inc (NASDAQ: ZYNE) were set to to open sharply lower Wednesday morning. 

What Happened

Zynerba, an actively-traded, micro-cap pharma company that develops synthetic cannabinoid therapies targeting rare and near-rare neuropsychiatric disorders, announced positive top-line results from the open-label Phase 2 BELIEVE 1 study that is evaluating the safety and efficacy of Zygel, codenamed ZYN002, in developmental and epileptic encephalopathies, or DEE.

Zygel is administered as a transdermal gel to children and adolescents with DEE.

DEE is heterogenous group of rare pediatric epilepsy syndromes, including Dravet syndrome, or DS, and Lennox-Gastaut syndrome, or LGS.

Top-line results showed 46 patients in the modified intention to treat, or ITT, population, who had more debilitating type of seizures, experienced a more than 44% mean reduction in seizures frrm month two onward using monthly seizure frequency normalized to 28 days.

Fifty-five percent of the patients experienced a more than 55% mean reduction in seizures at month six of treatment with Zygel.

The company also noted that 13 of the 46 patients had a variety of non-focal impaired awareness seizures and non-convulsive seizure types at baseline.

"The number of individual seizure types in these patients was too small to draw definitive conclusions and further analyses are warranted," Zynerba said.

The company said Zygel was well-tolerated, and the safety profile was consistent with previously released data from Zygel clinical trials.

About 96% of the patients experienced a treatment emergent adverse event and 60% experienced a treatment-related adverse event. The company said 10 patients reported a serious adverse event, or SAE, and they were mostly infection-related, with two SAEs possibly related to treatment.

Why It's Important

DEE is normally associated with severe cognitive impairment and behavioral disturbances, and these disorders are often progressive and highly resistant to treatment.

Improved seizure control is likely to have a positive impact on development and quality of life.

"These results suggest that Zygel may produce clinically meaningful reductions in seizures and may improve many of the difficult behaviors and symptoms, such as seizure intensity, fatigue, social isolation, poor cognition and language deficits," Zynerba CEO Armando Anido said in a statement. 

Following the complete analysis of the data, Zynerba said it intends to meet with the FDA, likely in the first half of 2020, to discuss the clinical pathway to approval. 

Zynerba shares were down 11.24% in Wednesday's premarket session. 

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