The US biopharmaceutical company Stealth Biotherapeutics is running a phase 1/2 trial of Elamipretide for the treatment of vision loss and cardiac disease in Friedreich’s ataxia (FA).
The trial started in March 2022 and has enrolled 18 participants aged 16+ with advanced FA symptoms. Participants were enrolled through the Children’s Hospital of Philadelphia (CHOP) in the US. The trial is investigating the safety, tolerability, and effects of the drug Elamipretide in FA.
Participants were randomised to either a low (20-30mg) or high dose (40-60mg) of Elamipretide. They will have various measurements of their vision and cardiac function at baseline, 36 weeks and 52 weeks. Results are expected in 2025.
In preclinical studies, Elamipretide has been shown to improve the activity of cells’ energy centres, known as mitochondria, which are damaged in FA. Elamipretide has been researched in other neurodegenerative diseases, including Duchenne Muscular Dystrophy (DMD).
You can read more about Elamipretide here.
