elamipretide

Elamipretide is a first-in-class mitochondria-targeted therapeutic tetrapeptide developed by Stealth BioTherapeutics that binds cardiolipin in the inner mitochondrial membrane and stabilizes mitochondrial cristae and electron transport chain function, thereby increasing ATP production and reducing mitochondria-derived reactive oxygen species. The drug has been studied across mitochondrial and age-related diseases, including dry age-related macular degeneration with high-risk drusen or noncentral geographic atrophy, primary mitochondrial myopathy, heart failure, and Barth syndrome. In ophthalmology, phase 1 ReCLAIM studies suggested improvements in low-luminance visual function with daily subcutaneous dosing, while the randomized phase 2 ReCLAIM-2 trial in dry AMD with geographic atrophy missed its primary endpoints but showed reduced progression of ellipsoid zone attenuation, supporting a photoreceptor-preservation hypothesis and informing phase 3 dry AMD development. Elamipretide has also reached commercialization in the United States under the brand name FORZINITY for Barth syndrome according to the provided context.

Brand names

FORZINITY

Other names

elamipretide hydrochloride