Friedreich’s Ataxia: Drug Enters Phase 2b Clinical Trial

Edison Pharmaceuticals has launched a phase 2b clinical trial of its experimental drug EPI-743 in adults with Friedreich's ataxia (FA) to assess whether the drug has positive effects on visual function, neurological and neuromuscular function, and disease-associated biomarkers.

EPI-743 is a small-molecule, oral drug that is designed to enter the central nervous system and synchronize (improve) energy generation in mitochondria, the energy-producing subunits in cells. It is being developed by Edison, which is located in Mountain View, Calif., and Utrecht, Netherlands.

The U.S. National Institutes of Health (NIH) also is testing this molecule in a broader range of neuromuscular disorders — all metabolic diseases of muscle and mitochondrial myopathies.

Trial to be held at three sites

Investigators plan to enroll 60 adults, ages 18-45 years. Trial participants will be assigned to take either 200 or 400 milligrams of EPI-743, or a placebo, three times a day for six months. In a planned extension study, all trial participants will take EPI-743.

Prospective participants must have a genetically confirmed diagnosis of FA and meet additional trial inclusion criteria.

The trial is being conducted at three centers in the United States. (At the time this article was posted, not all study sites had started recruiting for the trial.)

Children's Hospital of Philadelphia

Philadelphia, Pa.
Contact: Lauren Sayer at

University of California, Los Angeles

Los Angeles, Calif.
Contact: Maria Casado at

University of South Florida

Tampa, Fla.
Contact: Kelly Sullivan at

For more information

For details about the new trial, see:

Or, go to, and enter NCT01728064 in the search box.

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

21 241