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Edison Pharmaceuticals Announces Initiation of EPI-743 Phase 2B Leigh Syndrome Clinical Trial
Date:11/13/2012

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 ClinicalTrials.gov.

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

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SOURCE Edison Pharmaceuticals
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