Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) shared positive top-line results Wednesday from BELIEVE 1, an open label multidose Phase 2 clinical trial evaluating the efficacy and safety of Zygel, a CBD gel treatment for children and adolescents with a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE).
In a statement, the company said the trial also assessed the safety and efficacy of Zygel in various DEEs, including Dravet syndrome and Lennox-Gastaut syndrome that may be associated with severe cognitive impairment and behavioral disturbances.
The company said the BELIEVE 1 trial enrolled 48 patients between the ages of three and 16. The enrolled patients received weight-based initial doses of 250 mg, or 500 mg daily of Zygel.
READ: Zynerba shares evidence that CBD may be effective in managing constellation of sociobehavioral symptoms
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,” reported the company.
The Devon, Pennsylvania-based company also revealed that qualitative assessments by caregivers in the study demonstrate that use of Zygel may result in improved socio-behavioral and cognitive symptoms of DEE. Zygel was also well-tolerated in this study.
Clinically meaningful reductions in seizures
Professor Ingrid Scheffer, who is the chair of Paediatric Neurology Research, at the University of Melbourne, and an investigator in the BELIEVE 1 trial described the data from the clinical trial as “promising.”
“It suggests that Zygel may reduce seizure frequency in many types of difficult to treat DEEs and improve important behavior deficits, alertness, social interactions, and enable the child to be well enough to attend school more consistently,” said Scheffer in a statement.
“DEEs 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,” she added.
Scheffer, an Australian paediatric neurologist has made several major advances in the field of epilepsy research and is credited with finding the first gene implicated in epilepsy.
“We are encouraged by the positive topline results of the BELIEVE 1 trial of Zygel in children and adolescents with DEE, and we believe these data represent an important step forward for these patients and their families,” said Zynerba CEO Armando Anido.
“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.”
Anido said that after the company completed its analyses of the data, it intended to seek a meeting with the FDA, likely in the “first half of next year, to discuss the clinical pathway to approval.”
The US Patent and Trademark Office recently granted Zynerba a patent for treating Autism Spectrum Disorder, characterized by challenges with social skills and speech, with CBD.
— Adds details on study design, patient demographics —
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