Zynerba Pharmaceuticals Initiates Open-Label Phase 2 Trial of ZYN002 in Developmental and Epileptic Encephalopathies (DEE)
- Category: Small Molecules
- Published on Tuesday, 10 April 2018 16:28
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DEVON, PA, USA I April 10, 2018 I Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today announced that it has initiated the Phase 2 BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) clinical trial. The study will assess the safety and efficacy of ZYN002 in DEE, including Lennox-Gastaut and Dravet syndromes. ZYN002 is the first and only pharmaceutically-produced cannabidiol (CBD) formulated as a patent-protected permeation-enhanced transdermal gel.
“DEE is a heterogeneous group of epilepsy syndromes that involve significant developmental impairment or regression of developmental progress, and are highly resistant to treatment,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer. “Little attention is paid to DEE outside of Lennox-Gastaut and Dravet syndromes despite the high unmet need. The BELIEVE 1 study will assess children and adolescents with a variety of epilepsy syndromes that are characterized as DEE. Our goal is to deliver an effective, well tolerated, and easy to administer therapeutic option for individuals living with DEE and their families.”
The six-month BELIEVE 1 clinical trial is an open label multi-dose Phase 2 clinical trial designed to evaluate the efficacy and safety of ZYN002 in children and adolescents (three to <18 years) with DEE as classified by the International League Against Epilepsy (ILAE) (Scheffer et al. 2017). Approximately 50 patients with confirmed DEE will be enrolled in the clinical trial, approximately half of whom may have either Dravet or Lennox-Gastaut syndrome. Enrolled patients will receive weight-based initial doses of 125 mg BID (250 mg daily) or 250 mg BID (500 mg daily) of ZYN002 CBD gel. The primary efficacy assessment is change in seizure frequency. The Company expects to report topline results in 2019.
About Developmental and Epileptic Encephalopathies (DEE)
DEE is a heterogeneous group of epilepsy syndromes that may be associated with severe cognitive impairment and behavioral disturbances. These disorders are often progressive, and are highly resistant to treatment. DEE includes a number of rare and ultra-rare epilepsy syndromes including early myoclonic encephalopathy, epileptic encephalopathy with continuous spike and wave during sleep, and certain syndromes including Ohtahara, West, Landau-Kleffner, Lennox-Gastaut, Doose and Dravet. Improved seizure control may have a positive impact on development and quality of life.
Zynerba’s ZYN002 CBD gel is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced transdermal gel and is being studied in children and adolescents with Fragile X syndrome and developmental and epileptic encephalopathies, and in adult epilepsy patients with focal seizures. ZYN002 is a clear, permeation-enhanced gel that is designed to provide controlled drug delivery transdermally with once- or twice-daily dosing.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric diseases with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.
SOURCE: Zynerba Pharmaceuticals