Stealth BioTherapeutics Stock Surges On Elamipretide's European Orphan Drug Tag for Ultra-Rare Genetic Condition

by Vandana Singh, Benzinga Editor

June 2, 2021 7:08 AM | 30 seconds read |

The European Medicines Agency (EMA) has granted orphan drug designation to Stealth BioTherapeutics Corp's MITO elamipretide for Barth syndrome, an ultra-rare genetic condition.
Barth syndrome is characterized by cardiac abnormalities, often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness, and delayed growth.
Elamipretide, the company's lead candidate, is a peptide compound that readily penetrates cell membranes and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin.
Price Action: MITO shares are up 49% at 2.16 during the premarket session on the last check Wednesday.