Company: Stealth BioTherapeutics
Drug Type: Small Molecule
Conditions: Barth syndrome, Mitochondrial myopathies
Mechanism Type: Mitochondrial bioenergetics
Mechanism: Elamipretide targets cardiolipin, a lipid residing in the mitochondrial inner membrane, which is critical to maintaining mitochondrial function and cellular energy supply. Elamipretide, formerly known as Bendavia, has been shown to promote energy production and reduce the production of excess of reactive oxygen species. This drug could have potential applications in neurodegenerative diseases, including ALS.
U.S. Status for ALS: Preclinical
 A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension. ClinicalTrials.gov, 27 Oct 2017. Accessed 8 Feb 2018 from https://www.clinicaltrials.gov/ct2/show/NCT03323749.
Last updated March 11th, 2016