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elamipretide   Click here for help

GtoPdb Ligand ID: 14276

Synonyms: bendavia | Forzinity® | MTP-131 | MTP131 | SS-31 | SS31
Approved drug
elamipretide is an approved drug
Compound class: Peptide
Comment: Elamipretide (MTP-131; SS-31) is a cell-penetrating tetrapeptide that binds to the mitochondrial phospholipid cardiolipin [4,6] via electrostatic and hydrophobic interactions [1,5]. This action stabilises mitochondrial cristae structure, improves ATP synthesis (bioenergetics) and reduces reactive oxygen species production.
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES CC1=C(C[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(=CC(=C1)O)C
Isomeric 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
InChI InChI=1S/C32H49N9O5/c1-19-15-22(42)16-20(2)23(19)18-27(41-29(44)24(34)11-8-14-38-32(36)37)31(46)39-25(12-6-7-13-33)30(45)40-26(28(35)43)17-21-9-4-3-5-10-21/h3-5,9-10,15-16,24-27,42H,6-8,11-14,17-18,33-34H2,1-2H3,(H2,35,43)(H,39,46)(H,40,45)(H,41,44)(H4,36,37,38)/t24-,25+,26+,27+/m1/s1
InChI Key SFVLTCAESLKEHH-WKAQUBQDSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
References
1. Birk AV, Liu S, Soong Y, Mills W, Singh P, Warren JD, Seshan SV, Pardee JD, Szeto HH. (2013)
The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin.
J Am Soc Nephrol, 24 (8): 1250-61. [PMID:23813215]
2. Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C. (2025)
Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study.
Orphanet J Rare Dis, 20 (1): 199. [PMID:40281531]
3. Kim AY, Vernon H, Manuel R, Almuqbil M, Hornby B. (2022)
Quality of life in Barth syndrome.
Ther Adv Rare Dis, 3: 26330040221093743. [PMID:37180415]
4. Li M, Kong D, Meng L, Wang Z, Bai Z, Wu G. (2024)
Discovery of novel SS-31 (d-Arg-dimethylTyr-Lys-Phe-NH2) derivatives as potent agents to ameliorate inflammation and increase mitochondrial ATP synthesis.
RSC Adv, 14 (41): 29789-29799. [PMID:39301232]
5. Szeto HH. (2014)
First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics.
Br J Pharmacol, 171 (8): 2029-50. [PMID:24117165]
6. Tung C, Varzideh F, Farroni E, Mone P, Kansakar U, Jankauskas SS, Santulli G. (2025)
Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential.
Int J Mol Sci, 26 (3). [PMID:39940712]