Elamipretide

Description

Elamipretide, also known as MTP 131, is a mitochondria-targeted peptide antioxidant. Elamipretide decreases inflammation and protect against a variety of neurological illnesses. Elamipretide (SS-31) improves mitochondrial dysfunction, synaptic and memory impairment induced by lipopolysaccharide in mice. Elamipretide Promotes Mitophagosome Formation and Prevents Its Reduction Induced by Nutrient Excess in INS1 β-cells.

The Uses of Elamipretide

D-Arginyl-2,6-dimethyl-L-tyrosyl-L-lysyl-L-phenylalaninamide-d5 Trifluoroacetic Acid Salt is labelled D-Arginyl-2,6-dimethyl-L-tyrosyl-L-lysyl-L-phenylalaninamide (A769700) which is a mitochondrial-targeted compound that re-energizes ischemic mitochondria by interacting with cardiolipin. It is a cell-permeable antioxidant peptide.

Mechanism of action

Elamipretide can inhibit the production of pathogenic ROS (reactive oxygen species) and restore mitochondrial ATP synthesis. Currently, the most accepted mechanism for these two actions is the direct interaction between Elamipretide and cardiolipin.

Clinical Use

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has an Orphan Drug, Fast Track and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for treating Barth syndrome.

Side Effects

Side effects of elamipretide include:
Mild redness or itching at the injection site
Headache
Dizziness