Is also known as
- Alpha-Tocotrienol Quinone.
EPI-743 is a small quinone molecule developed by Edison Pharmeceuticals, Mountain View, California and Sumitomo Dainippon Pharma Co Ltd, Tokyo, Japan.
It is being tested for treating several mitochondrial disorders, including LHON.
Unlike Idebenone, EPI-743 acts in the cytosol of cells to target NAD(P)H Quinone Oxidoreductase 1, which is a powerful “scavenger” of Reactive Oxygen Species. (15102952)
Like Idebenone, EPI-743 is a tablet taken by mouth.
Some work has been done on the safety and effectiveness of EPI-743 on LHON patients. (24093206)
A trial of EPI-743 on 5 patients found that it seemed to show that EPI-743 stopped the progress of LHON, and two patients showed a lot of visual recovery. This was measurable by their improved visual actuity (reading a Snellen chart) as well as improved RNFL thickness. (22410442)
A clinical trial was running in 2014 on one patient for treating LHON with EPI-743 but the clinical trial was stopped by the company. No reason or further information has been issued by the company.
EPI-743 is no longer available under FDA Expanded Access.
A study of EPI-743 was done on siblings with LHON mutation 14484 (24093206)
The Edison Pharmaceuticals Clinical Development page shows that EPI-743 is being developed for Friedrich’s Ataxia, Leigh Syndrome, MELAS, Parkinson’s Disease, Cobalamin C Defect and “Undiagnosed Diseases of Redox and Metabolism” but not for LHON as of September 2015.
In November 2014 EPI-743 was administered to one LHON patient by the Doherty Eye Institute under FDA Expanded Access. See Clinical Trial NCT02300753 Emergency Administration of EPI-743 to a Single Patient With Leber’s Hereditary Optic Neuropathy [LHON]
At the LHON Conference in June 2015 Dr Sadum was unable to comment on the status of EPI-743 or Edison Pharmaceuticals.
Edison announced in 2014 that the US FDA had granted EPI-743 Orphan Drug status for the treatment of Leigh Syndrome.(23010433)
This page was last updated September 2 2015