DISCONTINUED: Stages of Development for EPI-A0001

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

Link to Study

A double-blind placebo-controlled trial phase II study with three arms: placebo, low dose, and high dose EPI-A0001 in 31 adults with FA

June 2011: The study was completed, and initial results were announced by Edison Pharmaceuticals.

  • The primary endpoint was Disposition Index, a measure of diabetic tendency, from a frequently sampled intravenous glucose tolerance test, evaluated 4 weeks into therapy.
  • Differences in changes in the Disposition Index between subjects treated with A0001 and placebo were not statistically significant.
  • There was significant improvement in neurological function as assessed by the Friedreich’s Ataxia Rating Scale (FARS). The improvement in the FARS was statistically significant in both the high and low dose groups in comparison to placebo. There were no differences in the rates of drug-related adverse events between the placebo group and each of the drug-treated groups.