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Details

Stereochemistry ABSOLUTE
Molecular Formula C13H21N3O6
Molecular Weight 315.3223
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of TROFINETIDE

SMILES

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

InChI

InChIKey=BUSXWGRAOZQTEY-SDBXPKJASA-N
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

HIDE SMILES / InChI

Molecular Formula C13H21N3O6
Molecular Weight 315.3223
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 2 / 2
E/Z Centers 0
Optical Activity UNSPECIFIED

Trofinetide (NNZ 2566), a proprietary small molecule analogue of glycine-proline-glutamate [Glypromate®], is being developed by Neuren Pharmaceuticals for the treatment of brain injuries, fragile X syndrome, Rett syndrome. Trofinetide is a synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1, a growth factor produced by brain cells. In animal models, trofinetide exhibits a wide range of important effects including inhibiting neuroinflammation, normalizing the role of microglia and correcting deficits in synaptic function. Trofinetide is being developed both in intravenous and oral formulations for a range of acute and chronic conditions. The intravenous form of trofinetide is presently in a Phase 2 clinical trial in patients with moderate to severe traumatic brain injury. The oral form of trofinetide is in Phase 2 development in Rett syndrome, Fragile X syndrome and mild traumatic brain injury (concussion). Trofinetide has been granted Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in Europe for both Rett syndrome and Fragile X syndrome.

Approval Year

PubMed

PubMed

TitleDatePubMed
Progress in Rett Syndrome: from discovery to clinical trials.
2016 Sep
A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome.
2017 Nov
Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.
2019 Apr 16

Sample Use Guides

Rett syndrome: Subjects were randomly assigned in a 2:1 ratio to 35 mg/kg twice daily of trofinetide or placebo for 14 days; 35 mg/kg twice daily or placebo for 28 days; or 70 mg/kg twice daily or placebo for 28 days. Both 35 mg/kg and 70 mg/kg dose levels of trofinetide were well tolerated and generally safe. Trofinetide at 70 mg/kg demonstrated efficacy compared with placebo based on prespecified criteria.
Route of Administration: Oral
Substance Class Chemical
Created
by admin
on Fri Dec 15 17:02:32 GMT 2023
Edited
by admin
on Fri Dec 15 17:02:32 GMT 2023
Record UNII
Z2ME8F52QL
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
TROFINETIDE
INN   WHO-DD  
INN   USAN  
Official Name English
NNZ-2566
Code English
trofinetide [INN]
Common Name English
TROFINETIDE [USAN]
Common Name English
ACP-2566
Code English
glycyl-2-methyl-L-prolyl-L-glutamic acid
Systematic Name English
DAYBUE
Brand Name English
L-Glutamic acid, glycyl-2-methyl-L-prolyl-
Common Name English
Trofinetide [WHO-DD]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 409013
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
FDA ORPHAN DRUG 363111
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
EU-Orphan Drug EU/3/15/1529
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
NCI_THESAURUS C78272
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
Code System Code Type Description
INN
10076
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
NCI_THESAURUS
C152757
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
DAILYMED
Z2ME8F52QL
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
DRUG BANK
DB06045
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
EPA CompTox
DTXSID101336041
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
PUBCHEM
11318905
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
EVMPD
SUB193920
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
WIKIPEDIA
Trofinetide
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
FDA UNII
Z2ME8F52QL
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
SMS_ID
100000178278
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
CAS
853400-76-7
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
USAN
KL-85
Created by admin on Fri Dec 15 17:02:32 GMT 2023 , Edited by admin on Fri Dec 15 17:02:32 GMT 2023
PRIMARY
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