An Edison Pharmaceuticals Inc. drug focused on a neurological disorder in young girls failed a mid-stage trial, but the Mountain View company still sees promise.
EPI-743 failed to lessen the severity of Rett Syndrome, as judged by a subjective score, but the company said girls who received the drug showed a statistically significant increase in head circumference, a key objective measure of the drug’s effectiveness.
The disorder, which affects girls exclusively, is marked by slow development of the brain and head, loss of use of the hands, problems with walking and seizures. It is caused by a mutation in a protein called MeCP2 in one in every 10,000 to 15,000 girls, and patients typically die before they turn 40.
Edison’s six-month trial was conducted at the General University Hospital in Siena, Italy, with 24 girls.
Edison Chief Medical Officer Dr. Matthew Klein said in a press release that the improvement in head circumference is encouraging and Edison will speak with health authorities, patient foundations and researchers “in the next several months to define the next steps that may ultimately lead to new options for children with Rett syndrome and other mitochondrial diseases.”
EPI-743 also is being studied by Edison in Leigh Syndrome, a lethal neuromuscular disorder, and Friedreich’s ataxia, a rare, inherited disease that affects the muscles and heart.
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