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Edison Pharmaceuticals Announces Initiation of EPI-743 Phase 2B Leigh Syndrome Clinical Trial

MOUNTAIN VIEW, Calif., Nov. 13, 2012 /PRNewswire/ -- Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "A Phase 2B Randomized, Placebo-Controlled, Double-Blind Clinical Trial of EPI-743 in Children with Leigh Syndrome." Four clinical trial sites have been selected in the United States: Lucile Packard Children's Hospital, Stanford University Medical Center – Palo Alto, California; Akron Children's Hospital – Akron, Ohio; Seattle Children's Hospital – Seattle, Washington; and Texas Children's Hospital, Baylor University – Houston, Texas.

The trial is a placebo-controlled study lasting six months, and then extending an additional six months, during which all subjects will receive EPI-743. Subjects must be between one and twelve years old, possess genetic confirmation of Leigh syndrome, and meet certain disease severity criteria. More information on the study specifics is available on

The primary endpoint of the clinical trial is the Newcastle Paediatric Mitochondrial Disease Scale. Secondary endpoints include neurologic, muscular, imaging, and biomarker indices. The study design was based on positive phase 2A data recently reported in the journal Molecular Genetics and Metabolism, "EPI-743 reverses the progression of the pediatric mitochondrial disease- genetically defined Leigh Syndrome" 107 (2012) 383-388.

Leigh Syndrome

Leigh syndrome is an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. Initially described in 1951, the hallmarks of the disease include death of central nervous system regions responsible for the control of breathing and coordination of movement. Leigh syndrome is a member of a larger family of diseases called mitochondrial disease, which share as a common biochemical mechanism defects in cellular energy metabolism.


EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of Leigh syndrome and other inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. It serves as a cofactor for the novel drug target– NADPH quinone oxidase 1 (NQO1). Through a redox-based mechanism, EPI-743 augments endogenous glutathione biosynthesis– essential for the control of oxidative stress.

Edison Pharmaceuticals

Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for children with orphan mitochondrial diseases.

Clinical Study Site Information

Akron Children's Hospital

Lucile Packard Children's Hospital, Stanford University Medical Center

Seattle Children's Hospital

Texas Children's Hospital, Baylor University

SOURCE Edison Pharmaceuticals
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