Trofinetide

Summary

Trofinetide (CHEMBL197084) is an approved small-molecule neuroprotective agent (ATC N07XX24); indicated across 5 conditions including rett syndrome and brain injury.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N07XX24
• Indications: 5 conditions
• Clinical trials: 12
• Chemistry: 315.32 Da · C13H21N3O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL197084
Name	Trofinetide
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11318905
ChEBI	CHEBI:229599
ATC	N07XX24
Molecular formula	C13H21N3O6
Molecular weight	315.32
InChIKey	BUSXWGRAOZQTEY-SDBXPKJASA-N

SMILES: C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)O

IUPAC name: (2S)-2-[[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino]pentanedioic acid

ChEBI definition: A tripeptide comprising of glycine, 2-methyl-L-proline, and L-glutamic acid joined in sequence by peptide linkages. It is the first and only treatment approved by the FDA specifically indicated for Rett syndrome in adults and pediatric patients two years of age and older.

Pharmacological roles (ChEBI): neuroprotective agent, anti-inflammatory agent.

Also known as: ACP-2566, Daybue, NNZ-2566, Trofinetida, Trofinetide, Gly-Pro-Glu, H-Gly-Pro-Glu-OH, H-Gly-PMe-Glu-OH, TROFINETIDE, Gly-L-Pro-L-Glu, trofinetide

Patent coverage: 65 distinct patent families (166 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Glutamate NMDA receptor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 5 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P35439	5.1	Ki	7960	nM	CHEMBL_ACT_1520848
P35439	5.1	Ki	7960	nM	CHEMBL_ACT_1743885

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Rett syndrome	4	MONDO:0010726	MONDO:0010726
brain injury	2	MONDO:0043510	MONDO:0043510
fragile X syndrome	2	MONDO:0010383	MONDO:0010383
injury	1	MONDO:0021178	EFO:0000546

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE2	6
PHASE3	3
PHASE1	2
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04181723	PHASE3	COMPLETED	Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
NCT04279314	PHASE3	COMPLETED	Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
NCT04776746	PHASE3	TERMINATED	Open-Label Extension Study of Trofinetide for Rett Syndrome
NCT04988867	PHASE2/PHASE3	TERMINATED	An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
NCT00805818	PHASE2	COMPLETED	Study of NNZ-2566 in Patients With Traumatic Brain Injury
NCT01366820	PHASE2	COMPLETED	Study of NNZ-2566 in Patients With Traumatic Brain Injury Under EFIC
NCT01703533	PHASE2	COMPLETED	A Safety Study of NNZ-2566 in Patients With Rett Syndrome
NCT01894958	PHASE2	COMPLETED	A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
NCT02100150	PHASE2	TERMINATED	A Safety and Efficacy Study of NNZ-2566 in Patients With Mild Traumatic Brain Injury (mTBI)
NCT02715115	PHASE2	COMPLETED	A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
NCT00961779	PHASE1	COMPLETED	Safety Study of NNZ-2566 in Healthy Female Subjects
NCT01420042	PHASE1	COMPLETED	Safety Study of NNZ-2566 in Healthy Subjects, Following Oral Administration

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).