Edison and Dainippon Initiate EPI-743 Phase 2B Trial For Leigh Syndrome
Edison Pharmaceuticals announced the initiation of a Phase II-B/III study to evaluate EPI-743 in children with Leigh Syndrome. The trial will be conducted with collaborator Dainippon Sumitomo Pharma Co, Ltd.
The open-label trial entitled “A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome” will study children with clinical and radiographic evidence of Leigh syndrome with or without genetic confirmation and who meet specific disease severity criteria. A minimum of five subjects will be enrolled in the study.
Leigh Syndrome is an inherited fatal, progressive, predominantly pediatric neuromuscular disease which currently has no approved treatments. The disease was first described in 1951 and is characterized by bilateral necrosis of central nervous system regions which control breathing and other critical neurological functions. The disease belongs to a large category of disorders known as mitochondrial disease and share a common biochemical feature defect in cellular energy metabolism. Leigh syndrome is also known as juvenile subacute necrotizing encephalomyelopathy and occurs in at least one in 40,000 live births.
EPI-743 is an orally bioavailable small molecule under development by Edison Pharmaceuticals for treatment of Leigh syndrome and other inherited mitochondrial diseases. The investigational molecule belongs to the para-benzoquinone class of drugs. EPI-743 augments endogenous glutathione biosynthesis, critical in the control of oxidative stress, through a redox-based mechanism. The molecule is undergoing Phase II clinical development for treatment of inherited respiratory chain disorders. The drug is also being evaluated in double-blind, placebo-controlled trials for several indications including Leigh syndrome, Undiagnosed Disorders of Oxidation-Reduction, Friedreich's ataxia, and Cobalamin C defect.
The Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and Dainippon Sumitomo agreed on the study design based on positive Phase II-A data previously reported in Molecular Genetics and Metabolism EPI-743 and on the phase 2B clinical trial of EPI-743 in Leigh syndrome patients currently ongoing in the United States.