Discontinued
A0001
Edison Pharmaceuticals
Discontinued
A0001 is alpha-tocopherolquinone, a drug which functions in the mitochondria.
HOW DOES A DRUG GET DEVELOPED?
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.
A double-blind placebo-controlled trial phase II study with three arms: placebo, low dose, and high dose A0001 in 31 adults with FA. The drug had previously been developed by PenWest and later by Edison Pharmaceuticals, also known as BioElectron.
June 2011:
- 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.
- Follow-on compounds of A0001 (e.g. PTC-743/vatiquinone) were developed by BioElectron, which was acquired by PTC Therapeutics, and are being evaluated for FA.