Shares of cannabinoid drug maker Zynerba Pharmaceuticals Inc 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.
Related Links:
Canaccord Genuity Bullish On Zynerba Ahead Of CBD Gel Trial Data
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
The Benzinga Cannabis Capital Conference is coming to Florida
The Benzinga Cannabis Capital Conference is returning to Florida, in a new venue in Hollywood, on April 16 and 17, 2024. The two-day event at The Diplomat Beach Resort will be a chance for entrepreneurs, both large and small, to network, learn and grow. Renowned for its trendsetting abilities and influence on the future of cannabis, mark your calendars – this conference is the go-to event of the year for the cannabis world.
Get your tickets now on bzcannabis.com – Prices will increase very soon!