Edison Pharmaceuticals initiates Phase 2B/3 study of EPI-743 in children with Leigh syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2B/3 study entitled, "A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome" to be conducted in conjunction with Dainippon Sumitomo Pharma Co, Ltd. 

The trial is an open-label study lasting six months. To be eligible for the trial, children must have clinical and radiographic evidence of Leigh syndrome with or without genetic confirmation, and meet certain disease severity criteria. At least five subjects will be enrolled. The clinical trial primary endpoint is the Newcastle Paediatric Mitochondrial Disease Scale. Secondary endpoints include neurologic, muscular, and biomarker indices.

The study design agreed to by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and Dainippon Sumitomo Pharma is based upon positive phase 2A data previously reported in Molecular Genetics and Metabolism EPI-743 reverses the progression of the pediatric mitochondrial disease—Genetically defined Leigh Syndrome 107 (2012) 383-388, and on the ongoing phase 2B clinical trial of EPI-743 in Leigh syndrome patients being conducted in the United States.

Source: Edison Pharmaceuticals, Inc.