TerminatedPhase 3Results postedNCT04776746
Open-Label Extension Study of Trofinetide for Rett Syndrome
ACADIA Pharmaceuticals Inc.n = 778 Nov 2020 to 30 Jun 2023
Rett Syndrome
Trofinetide / Studies
Synthetic analog · 3 amino acids · 315.3 Da
Trofinetide studies and research data
PepBank has 31 trofinetide studies with results. All of them gave trofinetide, with 204 results, grouped by what the studies measured. In each group, results from studies in people come first. Data as of 2026-10-30.
Searches 204 results and 58 papers. Newer studies from Europe PMC
Results in people
168
82% of 204 results
Results in animals
33
16% of 204 results
Results in lab cells
2
under 1% of 204 results
From computer models
1
under 1% of 204 results
The molecule
Trofinetide identity
Identifiers, parent molecule, targets and development stage, from curated data.
Trofinetide is an analog of the IGF-1 N-terminal tripeptide Gly-Pro-Glu. Its therapeutic mechanism in Rett syndrome is unknown.
- Kind
- Synthetic analog · 3 residues
- Identifiers
- ChEMBLCHEMBL197084 (opens in a new tab)PubChem CID11318905 (opens in a new tab)UNIIZ2ME8F52QL (opens in a new tab)DrugBankDB06045 (opens in a new tab)CAS853400-76-7
- Parent
- Analog of Insulin-like growth factor 1Trofinetide is an analog of Gly-Pro-Glu, the N-terminal tripeptide of mature IGF-1. It contains 2-methyl-L-proline at residue 2. P05019 identifies the parent protein, not trofinetide. Parent coordinates remain unset.
- Development
- Approved in the US and EU (Daybue, 2023)Trofinetide has approved US and EU medicinal products. The therapeutic mechanism in Rett syndrome remains unknown. Approval does not extend to every indication. Source (opens in a new tab)
Targets
No molecular target is recorded for this peptide.
Sequence and structure
Residues, differences from the parent, calculated properties and 3D structure. Source values and calculated values are shown separately.
Sequence
3 amino acids · 1 modification
H-
Residues 1 to 3:
-OHGE
Alignment to Insulin-like growth factor 1
Calculated from the two sequences. Curated notes are shown as given.
2 of 3 aligned residues identical (67%) · 1 non-canonical residue · Insulin-like growth factor 1 1–3
- Top row: Insulin-like growth factor 1 (parent)
- Bottom row: Trofinetide
GE
- Same as parent
- Substituted
- Non-canonical or D-form
- Added
- Gap
- Modified side chain or end
- Parent context
Differences from the parent
- 1Parent 2 · analog 2Pro → Xaa · Non-canonical residue2-methyl-L-Pro
Basis: Trofinetide is an analog of Gly-Pro-Glu, the N-terminal tripeptide of mature IGF-1. It contains 2-methyl-L-proline at residue 2. P05019 identifies the parent protein, not trofinetide. Parent coordinates remain unset.
Calculated from the two sequences with @pepdb/chem alignToParent (ungapped unless a gap scores higher). The record’s curated notes are shown as given.
Properties
From identity sources
- Formula
- C13H21N3O6
- Molecular weight
- 315.32 g/mol
- Monoisotopic mass
- 315.143 Da
- GSRS UNII Z2ME8F52QL(opens in a new tab)retrieved 2026-10-27
- PubChem CID 11318905(opens in a new tab)retrieved 2026-10-27
- Curated Daybue US label(opens in a new tab)retrieved 2026-10-27
Calculated from the sequence
Some values cannot be calculated. The sequence has a non-canonical residue (X). Residue tables cover only the 20 standard amino acids.
- Average mass
- Not calculated
- Monoisotopic mass
- Not calculated
- Isoelectric point (pI)
- Not calculated
- Net charge at pH 7.4
- Not calculated
- GRAVY (hydropathy)
- Not calculated
- Extinction coefficient, 280 nm (Cys reduced)
- 0 M⁻¹ cm⁻¹
- 5500 x Trp + 1490 x Tyr · Pace et al. 1995 (ExPASy ProtParam)
- No Trp or Tyr: absorbance at 280 nm cannot measure this peptide.
- Instability index
- Not calculated
- Aliphatic index
- Not calculated
- Boman index
- Not calculated
- Hydrophobic moment (μH)
- Not calculated
- 1 non-canonical residue (X) is not included in calculated values.
- Defined and validated for proteins; use with care for short peptides.
- Assumes an ideal alpha helix. Many short peptides are not helical.
@pepdb/chem 0.2.0 · EMBOSS pKa set · ExPASy residue masses
Net charge by pH
No charge curve
The sequence has a non-canonical residue (X). Residue tables cover only the 20 standard amino acids.
Composition
- Negative33% (1)E
- Special33% (1)G
- Non-canonical33% (1)X
3 residues. Each residue counts in one class; F and W count as aromatic.
A Ala0 · 0%
I Ile0 · 0%
L Leu0 · 0%
M Met0 · 0%
V Val0 · 0%
F Phe0 · 0%
W Trp0 · 0%
Y Tyr0 · 0%
H His0 · 0%
K Lys0 · 0%
R Arg0 · 0%
D Asp0 · 0%
E Glu1 · 33%
N Asn0 · 0%
Q Gln0 · 0%
S Ser0 · 0%
T Thr0 · 0%
C Cys0 · 0%
G Gly1 · 33%
P Pro0 · 0%
X Xaa1 · 33%
3D structure
PredictedAlphaFold DB · P05019
Loads the 3D viewer (3Dmol.js, about 150 KB) and the model file from AlphaFold DB (EMBL-EBI).
2D structure
Draws the structure in your browser with RDKit (about 2.3 MB, loaded once).
SMILES fromPubChem CID 11318905(opens in a new tab)
C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)OResults by area
What the studies measured
People first in each area, then animals, then lab cells. Counts are exact and come from studies that gave the peptide.
Trials and papers
Newer studies
PepBank reads each paper before it shows the results. This search looks in Europe PMC for newer papers that PepBank has not read yet.
Status and safety
Data