Stealth BioTherapeutics is investigating the potential of elamipretide, a drug that has been shown to bind to the inner mitochondrial membrane, aiding in mitochondrial respiration and ATP production while protecting against reactive oxygen species (ROS). The company intends to use this drug as a treatment for multiple mitochondrial diseases.

While a Phase 3 trial for primary mitochondrial myopathy did not meet its primary endpoints, the company is still investigating the drug in Phase 2/3 crossover trials as a treatment for Barth syndrome and Leber’s hereditary optic neuropathy (LHON) along with a Phase 2 trial for dry age-related macular degeneration (AMD). Two 2021 press releases reported positive results for Barth syndrome and dry AMD, and in August 2021, the company submitted a New Drug Application (NDA) to the FDA for the treatment of Barth syndrome.

The company’s second-generation candidate, SBT-272, for amyotrophic lateral sclerosis (ALS) is currently in Phase 1 testing.