Basic Information
Elamipretide (CAS No.: 736992-21-5) is a small-molecule mitochondria-targeted tetrapeptide, also known as SS-31 and MTP-131. Its molecular formula is C₃₂H₄₉N₉O₅ with a molecular weight of 639.79. Owing to its unique mechanism of action, it has become a hotspot molecule in the research of diseases related to mitochondrial dysfunction. As the "powerhouse" of cells, mitochondrial dysfunction is closely associated with aging, rare diseases and a variety of chronic diseases. The core value of Elamipretide lies in its precise targeting of the mitochondrial inner membrane to repair impaired mitochondrial function.
Mechanism of Action
Its mechanism of action mainly centers on cardiolipin, a key phospholipid in the mitochondrial inner membrane. It can specifically bind to damaged cardiolipin, stabilize the structure of the mitochondrial inner membrane, promote the assembly of respiratory chain complexes, enhance the efficiency of oxidative phosphorylation, reduce the production of reactive oxygen species (ROS), and block the vicious cycle of "mitochondrial damage-oxidative stress". Relevant studies have confirmed that Elamipretide can improve mitochondrial energy metabolism and exert protective effects in various disease models.
Clinical Progress
In clinical research, a phase Ⅱ/Ⅲ clinical trial for Bartter syndrome (a rare mitochondrial genetic disease) showed that after 36 weeks of treatment with 40 mg of Elamipretide daily, the 6-minute walking distance of patients was significantly increased and the symptom scores were remarkably improved. In a phase Ⅱ trial for age-related macular degeneration, although it did not reach the primary endpoint, it slowed down the degeneration of the ellipsoid zone associated with photoreceptor damage, providing a direction for subsequent research.
Post time: 2026-03-16