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Description

Iramipeptide, also commonly known as SS-31, MTP-131, or Bendavia, with CAS Number 736992-21-5, is a synthetic tetrapeptide compound with the molecular formula C₃₂H₄₉N₉O₅ and a molecular weight of approximately 639.79. It appears as a white to off-white solid powder and is slightly hygroscopic. Its amino acid sequence is D-Arg-Dmt-Lys-Phe-NH₂, which includes a unique D-configuration amino acid and a modified tyrosine; these structural features confer unique properties—the ability to specifically accumulate on the inner mitochondrial membrane (at concentrations thousands of times higher than in the extracellular space) and to bind to its key lipid, “cardiolipin.” Eramipeptide was originally designed by researchers at Cornell University and later advanced through clinical development by a U.S. company; it is regarded as a representative drug of the new class of mitochondria-targeted peptides. Currently, it is primarily in the clinical research phase and is widely used in scientific research and drug development for mitochondrial-related diseases.

Main Efficacy

The core mechanism of action of Iramipeptide is “mitochondrial protection.” By binding to cardiolipin on the inner mitochondrial membrane, it stabilizes the morphological structure of the mitochondrial cristae and optimizes the function of the electron transport chain. This reduces the production of harmful free radicals (reactive oxygen species) generated during mitochondrial energy production while helping damaged or ischemic mitochondria restore their function. Simply put, it acts at the source—the mitochondria, the “powerhouses” of the cell—to reduce energy loss and improve efficiency, thereby protecting organs that rely on high energy for their functioning. Based on this mechanism, it shows multifaceted potential in protecting high-energy-demand organs such as the heart, kidneys, and brain. Current evidence primarily comes from cellular studies, animal models, and some clinical trials.

1. Mitochondria-targeted antioxidant action: It specifically acts on the inner mitochondrial membrane, binds to cardiolipin, stabilizes the cristae structure, reduces ROS production by mitochondria, and minimizes oxidative damage at its source.

2. Protection of cardiac function: It improves conditions associated with myocardial ischemia, myocardial infarction, and heart failure; helps ischemically damaged myocardium recover function; and reduces myocardial injury.

3. Kidney protection: Improves renal function, alleviates kidney injury and fibrosis in renal disease models, and provides protective effects against acute kidney injury and chronic kidney disease.

4. Neuroprotection: Protects neurons from damage caused by inflammation and oxidative stress, with protective potential in neurodegenerative and ischemic injuries.

5. Improves cellular energy metabolism: Optimizes the function of the mitochondrial electron transport chain, helps restore the energy-supplying capacity of damaged mitochondria, and enhances the overall energy status of cells.

6. Anti-inflammatory: Indirectly alleviates inflammatory responses by reducing mitochondrial damage and oxidative stress, thereby protecting the tissue microenvironment.

Application Fields

At the application level, the primary use of iramipeptide is currently as a tool compound in mitochondrial biology research, helping scientists study mitochondrial function, oxidative stress, and the mechanisms underlying related diseases; it is also a candidate compound in several drug development pipelines, being developed for the treatment of mitochondrial diseases and various organ damage-related conditions. Due to its unique positioning as a “mitochondrial repair” agent, it has practical applications in scientific research, drug development, and raw material trade, making it one of the most widely recognized compounds in the niche field of mitochondria-targeted peptides.

1. Research Tool (Currently the Primary Use): As a mitochondria-targeted peptide, it is widely used in research on mitochondrial function, oxidative stress, energy metabolism, and organ protection mechanisms, serving as a vital tool compound in relevant laboratories.

2. Drug Development: As a drug candidate, it is being advanced in clinical studies for conditions such as cardiomyopathy, heart failure, ischemia-reperfusion injury, acute kidney injury, and neurodegenerative diseases; some indications have already entered late-stage clinical trials.

3. Exploration of Treatments for Rare Diseases: Clinical exploration is underway for mitochondrial-related rare diseases (such as Barth syndrome), and these compounds are regarded as promising candidates for orphan drug development.