12:00 AM
Oct 08, 2012
 |  BioCentury  |  Product Development

Boosting mitochondria

Edison throwing itself behind EPI-743 on early positive results in Leigh disease

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.

While no drug is approved to treat Leigh disease, patients have reported anecdotal success taking the dietary supplement Coenzyme Q10 to enhance mitochondrial activity by restoring redox homeostasis. Coenzyme Q10 is a natural component of the electron transport chain.

Based on this information, Edison designed and screened a library of 36,000 small molecules that could more potently enhance redox activity. The company identified the vitamin E...

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