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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 [4,7] via electrostatic and hydrophobic interactions [1,6]. 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/)
No information available.
Summary of Clinical Use Click here for help
Elamipretide was progressed to clinical studies to determine safety and protective and restorative efficacy in diseases involving mitochondrial dysfunction. The US FDA issued accelerated approval for use of elamipretide to treat Barth syndrome in September 2025 [5].
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT05162768 Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) Phase 3 Interventional Stealth BioTherapeutics Inc.
NCT06373731 ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) Phase 3 Interventional Stealth BioTherapeutics Inc.
NCT02245620 A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly Phase 2 Interventional Stealth BioTherapeutics Inc.
NCT03098797 A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome Phase 2/Phase 3 Interventional Stealth BioTherapeutics Inc. The TAZPOWER clinical trial 2-3