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 or derivative
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.
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.

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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
No information available.
Summary of Clinical Use Click here for help
Trofinetide was advanced to clinical evaluation in a number of brain conditions, including Rett syndrome [3,6], fragile X syndrome [2] and traumatic brain injuries. Its first FDA approval was issued in March 2023 [4], as the first drug for the treatment of Rett syndrome (a debilitating X-linked neurodevelopmental disorder, that is most commonly caused by mutations in the methyl-CpG binding protein 2 (MECP2) gene.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04181723 Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™) Phase 3 Interventional ACADIA Pharmaceuticals Inc. 5