Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc told investors that an article in the Journal of the American Medical Association (JAMA) Network Open had described the results of the company's open-label Phase 2 BELIEVE study as positive.
This is an open label study assessing the "safety and efficacy" of Zygel (ZYN002) as a transdermal gel for children and adolescents with developmental and epileptic encephalopathy (DEE).
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The article concludes that the cannabidiol transdermal gel was "safe, well tolerated, and was associated with reductions in focal impaired awareness seizures (FIAS) and tonic-clonic seizure (TCS) frequency and disease burden", reported Zynerba.
"DEEs are the most severe group of epilepsies and are usually drug-resistant," said Ingrid E. Scheffer, the lead investigator in the BELIEVE study, Laureate Professor and chair of Pediatric Neurology Research at the University of Melbourne.
"Antiseizure medications are usually administered orally, which can be extremely challenging in children with behavioral and cognitive problems. Non-oral therapies are needed to provide an alternative route to deliver medications to control seizures and improve developmental outcomes.
"The data from the BELIEVE study are promising and suggest that Zygel may be a safe and well-tolerated option to improve seizure control, challenging behaviors and other symptoms associated with DEEs."
Zynerba said 48 patients with a mean age of 10.5 years were enrolled in the BELIEVE study and included in the safety analysis.
Sixty-percent (60%) had at least one treatment-related adverse event (AE) over the 6.5 month trial period and 96% of these AE’s were mild or moderate. During the treatment period, 10 patients (21%) reported serious adverse events (SAEs).
Two SAEs were considered to be possibly treatment related: nonconvulsive status epilepticus and lower respiratory tract infection, in separate patients. All SAEs resolved, and none resulted in alteration of study medication, reported the company.
The article was called "Safety and tolerability of transdermal cannabidiol gel in children with developmental and epileptic encephalopathies: A nonrandomized controlled trial" and can be accessed at HERE.
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