7 Sep 21
Zynerba Pharmaceuticals Announces Publication of Results of Phase 2 BELIEVE Open-Label Study of Zygel™ in Children and Adolescents with Developmental and Epileptic Encephalopathies (DEE) in JAMA Network Open
Devon, PA, September 7, 2021 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced the publication of results from the Company’s open-label Phase 2 BELIEVE (Open Label Study to Assess the Safety and Efficacy of Zygel™ (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) study in the Journal of the American Medical Association (JAMA) Network Open.
The article, titled “Safety and tolerability of transdermal cannabidiol gel in children with developmental and epileptic encephalopathies: A nonrandomized controlled trial” can be accessed at HERE.
The article describes the positive results from BELIEVE, the first trial of a non-oral formulation of cannabidiol in children and adolescents with DEEs, and concludes that 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.
“DEEs are the most severe group of epilepsies and are usually drug-resistant. 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,” said Ingrid E. Scheffer, MBBS, PhD, FRS, Laureate Professor, and chair, Pediatric Neurology Research, University of Melbourne, and the lead investigator in the BELIEVE study. “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.”
Forty-eight (48) patients with a mean age of 10.5 years were enrolled in BELIEVE 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.
The authors indicated that the incidence of AEs in this study, particularly related to infections, was likely due to the high baseline rate of complex morbidities and seizure severity of these patients at study onset, and to the relatively long, 6.5 month duration of the trial over which events were collected.
From an efficacy perspective, the analysis of the 33 patients with FIAS and TCS showed a 58% median monthly reduction in seizures at month 5, and 43.5% reduction over the 6.5 month study period (the primary efficacy endpoint). The percent of ≥50% responders in the FIAS and TCS group ranged from 42.4% at month 2 and peaked at 62.5% at month 5. Parents/caregivers noted improvements in social or interpersonal engagement and irritability (33/43 [77%]); alertness, energy and sleep (23/43 [53%]); and cognition or concentration (20/43 [47%]).
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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