trofinetide   Click here for help

GtoPdb Ligand ID: 12482

Synonyms: ACP-2566 | analogue 4 [PMID: 15837309] | Daybue® | glycyl-alpha-methyl-L-prolyl-L-glutamic acid | NNZ-2566 | NNZ2566
Approved drug
trofinetide is an approved drug (FDA (2023))
Compound class: Peptide
Comment: Trofinetide (NNZ-2566), a synthetic analogue of the amino‐terminal tripeptide of insulin-like growth factor 1 (IGF-1; GPE). It was developed as a neuroprotective agent, for potential application in neurodegenerative diseases of the central nervous system, and traumatic brain injury.
The endogenous glycine-proline-glutamate tripeptide (GPE, Glypromate) is present in the brain [7], and it has been demonstrated to partially reverse core symptoms in Mecp2-deficient mice that are a model of human Rett syndrome [8]. Trofinetide has been modified to offer a longer half-life than GPE.

trofinetide is commercially available from our sponsors

2D Structure
Click here for help

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.

✖
Click here for structure editor
SMILES / InChI / InChIKey
Click here for help

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/)

✖
Canonical SMILES C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)O
Isomeric SMILES C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)O
InChI InChI=1S/C13H21N3O6/c1-13(5-2-6-16(13)9(17)7-14)12(22)15-8(11(20)21)3-4-10(18)19/h8H,2-7,14H2,1H3,(H,15,22)(H,18,19)(H,20,21)/t8-,13-/m0/s1
InChI Key BUSXWGRAOZQTEY-SDBXPKJASA-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/)
Bioactivity Comments
Trofinetide displaces [3H]glutamate from rat N-methyl-D-aspartate glutamate receptors (in brain synaptic membranes) with a Ki of ~8 μM [1].