A new drug called Elamipretide (formerly Bendavia) is currently being developed by a US based pharmaceutical company called Stealth Biotherapeutics.
The drug was initially developed to protect against a special type of tissue damage which is caused when there is a sudden return of blood to an organ after there has been a lack of blood. (This commonly happens on recovery from heart attacks or following organ injury.)
This damage is called ‘reperfusion injury’ and is caused by mitochondria suddenly over-working to restore the balance in the cell. This over-working can generate an overflow of oxygen species (called free radicals) which can trigger wide scale tissue damage and cell death.
Elamipretide focuses on preventing the mitochondria from wide scale release of these oxygen species, which in turn is thought to reduce cell damage and death.
Developing drugs that target mitochondria is challenging because it is difficult to penetrate cell and outer mitochondrial membranes however in multiple pre-clinical studies, the systemic delivery of elamipretide has been shown to reach the inner mitochondrial membrane and target a lipid critical to maintaining mitochondrial function and cellular energy supply.
Animal models have shown the drug to be effective and Phase II clinical trials are currently underway to test the safety and efficacy of this drug for primary mitochondrial disease and LHON.
In June 2016 initial results from a Phase ii clinical trial on elamipretide (MMPOWER) showed positive results. The findings demonstrated statistically significant improvements in 'distance walked in six minutes' which is an accepted measurement of functional exercise capacity.
Dr. Amel Karaa, the trial investigator and clinical geneticist at Massachusetts General Hospital said “These findings demonstrate the potential for elamipretide to help improve their ability to perform everyday activities. We look forward to further study of this compound in upcoming trials of primary mitochondrial disease.”
You can read the [Press Release] for more details.
Follow on trials to evaluate additional efficacy and longer dosing schedules for patients who participated in MMPOWER began in mid 2016 and are ongoing. Results are expected in mid 2017.