Boosting mitochondria

Edison Pharmaceuticals Inc. plans to bring EPI-743 into Phase IIb testing this quarter after leveraging insights gained from an expanded access protocol to score a positive Phase IIa result in Leigh disease. The company is refocusing its R&D strategy around the compound and expects six-month data in 3Q13.

Leigh is a fatal pediatric condition caused by mutations that affect mitochondrial activity, resulting in a cellular energy imbalance that leads to a loss of neurological function. The disease is characterized by necrotizing lesions in the brain, but is caused by many different mutations that manifest with the same phenotype. Edison estimates its incidence is about one in 30,000...