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Edison Pharmaceuticals Announces Phase 2 Positive Clinical Results for EPI-743 in Rett Syndrome
Date:9/2/2014

MOUNTAIN VIEW, Calif., Sept. 3, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced results of a recently completed phase 2 placebo-controlled trial in pediatric patients. Rett syndrome patients treated with EPI-743 demonstrated a statistically significant increase in head circumference.  Decreased brain growth and head circumference are hallmarks of the disease.

Rett syndrome is a genetic neurodevelopmental disorder predominately affecting girls, and is usually diagnosed by 24 months of age. It is characterized by a constellation of neurological, behavioral, functional, and structural features. These include delayed development, characteristic hand wringing, seizures, impaired respiratory function, slowing of brain growth, and resultant diminished head size, as well as other neurological disorders.

The double-blind, placebo-controlled clinical trial was conducted at the General University Hospital (Azienda Ospedaliera Universitaria Senese) of Siena, Italy, a leading international Rett syndrome clinical care and research center. A total of 24 subjects, 2.5 to 8 years of age, were enrolled in the six-month trial. While the study did not meet the primary endpoint of improving the subjective Rett syndrome disease severity score, subjects treated with EPI-743 demonstrated an objective improvement in head growth. Specifically, head growth in the treatment group was 0.75 cm from baseline, compared to 0.29 cm in the placebo group (p=0.05).  In a subgroup analysis of the children with the largest head growth, improvements in oxygenation, hand function, and disease-relevant biomarkers were also recorded. These data will be submitted for publication in the near future.

The natural history of decreased head growth in girls with Rett syndrome has been quantified and is an established metric of disease. Improvement in head growth was recorded in nine of the 12 subjects
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SOURCE Edison Pharmaceuticals, Inc.
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