Elamipretide, also known as SS-31, MTP-131, and Bendavia, is a small mitochondrially-targeted tetrapeptide (D-Arg-dimethylTyr-Lys-Phe-NH2) that has shown promise in reducing the production of toxic reactive oxygen species (ROS) and stabilizing cardiolipin, a key component of the inner mitochondrial membrane. Developed by Stealth BioTherapeutics, elamipretide has been investigated for its potential in treating a variety of conditions related to mitochondrial dysfunction.
The peptide’s mechanism of action involves enhancing cellular ATP synthesis, improving electron transport, and reducing ROS formation, without negatively affecting healthy mitochondria. This makes it a potential therapeutic agent for diseases where mitochondrial dysfunction plays a critical role, such as heart failure, kidney injury, and neurodegenerative disorders.
In clinical trials, elamipretide has been studied for its efficacy in treating mitochondrial myopathy, with a Phase III trial initiated in 2017. However, as of January 2020, the trial did not meet its expected outcomes. Despite this setback, the peptide continues to be researched for its neuroprotective properties, particularly in the context of traumatic optic neuropathy (TON). Studies have shown that elamipretide, when administered in conjunction with TNF inhibitors like etanercept, can significantly improve retinal ganglion cell (RGC) survival following optic nerve trauma.
Elamipretide’s ability to mitigate oxidative stress and inflammation has also been demonstrated in animal models of cognitive impairment and myocardial infarction. For instance, in a mouse model of lipopolysaccharide-induced cognitive impairment, elamipretide was found to protect the hippocampus from mitochondrial dysfunction and attenuate oxidative stress and inflammatory responses.
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| Other Names | MTP-131, Elamipretide |
|---|---|
| IUPAC Name | (2S)-6-amino-2-[[(2S)-2-[[(2R)-2-amino-5-(diaminomethylideneamino)pentanoyl]amino]-3-(4-hydroxy-2, 6-dimethylphenyl)propanoyl]amino]-N-[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]hexanamide |
| CAS | 736992-21-5 |
| Molecular Weight | 639.8 |
| Molecular Formula | C32H49N9O5 |
| SMILES | CC1=CC(=CC(=C1C[C@@H](C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC2=CC=CC=C2)C(=O)N)NC(=O)[C@@H](CCCN=C(N)N)N)C)O |