Experimental Mitochondrial Myopathy Treatment Shows Encouraging Results in Phase 2 Trial

stealth bioStealth Bio Therapeutics has reported encouraging results from a phase 2 clinical trial to evaluate elamipretide for the treatment of muscle weakness caused by mitochondrial disease.

Elamipretide is an experimental drug designed to modify disease by helping to restore normal energy production in mitochondria and decrease oxidative stress.

In the completed MMPOWER trial, investigators treated 36 patients with genetically confirmed mitochondrial disease, ages 16-65 years, with one of three doses of elamipretide or placebo. The trial was designed to evaluate the safety, tolerability and efficacy of the drug to improve function in primary mitochondrial myopathy.

Results showed that treatment with elamipretide was safe and well-tolerated. In addition, trial participants treated with the drug showed improvements on the six-minute walk test (6MWT), which measures the distance a person can walk on a flat, hard surface in a period of six minutes.

Although MDA didn’t fund this study, MDA has supported research investigating the role of oxidative stress and mitochondrial energy production in mitochondrial myopathies, as well as other potential therapies targeting these mechanisms.

If proven safe and effective in late-stage trials, elamipretide could someday become an approved therapy for the treatment of mitochondrial myopathy. The FDA granted fast track status for the treatment of primary mitochondrial myopathy in patients with genetic mitochondrial diseases earlier this year.