Elamipretide
SS-31, MTP-131, Bendavia.
Greater than 97.0% as determined by analysis by RP-HPLC.
Description
Elamipretide Synthetic is a single, non-glycosylated polypeptide chain containing 4 amino acids, having a molecular mass of 640 Dalton and a Molecular formula of C32H49N9O5 .
Product Specs
SS-31, MTP-131, Bendavia.
H-D-Arg-(2',6'-dimethyl-Tyr)-Lys-Phe-NH2.
Product Science Overview
Elamipretide targets the inner mitochondrial membrane, where it binds reversibly to cardiolipin . Cardiolipin is an essential lipid component of the inner mitochondrial membrane, playing a crucial role in maintaining mitochondrial function and integrity. By binding to cardiolipin, elamipretide stabilizes the mitochondrial membrane, reduces the production of toxic reactive oxygen species (ROS), and enhances mitochondrial respiration and ATP production .
Elamipretide has been investigated for its potential therapeutic benefits in various conditions associated with mitochondrial dysfunction. Some of the key areas of research include:
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Mitochondrial Myopathy: Elamipretide has been studied in patients with primary mitochondrial myopathy, a condition characterized by muscle weakness and exercise intolerance due to mitochondrial dysfunction. Although a Phase III trial did not meet its primary endpoints, there were indications of potential benefits in certain subgroups .
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Barth Syndrome: This rare genetic disorder affects mitochondrial function, leading to skeletal muscle weakness, cardiomyopathy, and other symptoms. Elamipretide has received Fast Track and Orphan Drug designation from the FDA for the treatment of Barth syndrome .
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Ophthalmic Diseases: Elamipretide is being evaluated in clinical trials for dry age-related macular degeneration (AMD), a condition that leads to vision loss due to mitochondrial dysfunction in retinal cells .
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Cardiomyopathies: Research has also explored the potential of elamipretide in treating various forms of cardiomyopathy, including those associated with mitochondrial dysfunction .
Despite its promising mechanism of action, elamipretide has faced challenges in clinical development. Some trials have not met their primary endpoints, highlighting the complexity of translating mitochondrial-targeted therapies into clinical success . However, ongoing research continues to explore its potential in various indications, and future studies may provide further insights into its therapeutic benefits.
