From WikiProjectMed
Jump to navigation Jump to search
Trofinetide structure.png
Legal status
Legal status
  • (2S)-2-{[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino}pentanedioic acid
CAS Number
PubChem CID
Chemical and physical data
Molar mass315.326 g·mol−1
3D model (JSmol)
  • C[C@]1(CCCN1C(=O)CN)C(=O)N[C@@H](CCC(=O)O)C(=O)O
  • 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

Trofinetide (NNZ-2566) is a drug developed by Neuren Pharmaceuticals that acts as an analogue of the neuropeptide (1-3) IGF-1, which is a simple tripeptide with sequence Gly-Pro-Glu obtained by enzymatic cleavage of the growth factor IGF-1 within the brain. Trofinetide has anti-inflammatory properties and was originally developed as a potential treatment for stroke,[1][2] but has subsequently been developed for other applications and is now at the FDA in the New Drug Application phase, after a successful Phase III clinical trial against Rett syndrome.[3] Trofinedite has also had a successful Phase II trial againast Fragile X syndrome.[4][5][6]


  1. ^ Bickerdike MJ, Thomas GB, Batchelor DC, Sirimanne ES, Leong W, Lin H, et al. (March 2009). "NNZ-2566: a Gly-Pro-Glu analogue with neuroprotective efficacy in a rat model of acute focal stroke". Journal of the Neurological Sciences. 278 (1–2): 85–90. doi:10.1016/j.jns.2008.12.003. PMID 19157421. S2CID 7789415.
  2. ^ Cartagena CM, Phillips KL, Williams GL, Konopko M, Tortella FC, Dave JR, Schmid KE (September 2013). "Mechanism of action for NNZ-2566 anti-inflammatory effects following PBBI involves upregulation of immunomodulator ATF3". Neuromolecular Medicine. 15 (3): 504–14. doi:10.1007/s12017-013-8236-z. PMID 23765588. S2CID 12522580.
  3. ^ "Positive top-line results from pivotal Phase 3 trial in Rett syndrome" (PDF). 7 December 2021. Retrieved 21 July 2022.
  4. ^ Deacon RM, Glass L, Snape M, Hurley MJ, Altimiras FJ, Biekofsky RR, Cogram P (March 2015). "NNZ-2566, a novel analog of (1-3) IGF-1, as a potential therapeutic agent for fragile X syndrome". Neuromolecular Medicine. 17 (1): 71–82. doi:10.1007/s12017-015-8341-2. PMID 25613838. S2CID 11964380.
  5. ^ "Study Details - Rett Syndrome Study". Retrieved 21 July 2022.
  6. ^ "Neuren's Tofinetide Successful in Phase 2 Clinical Trial in Fragile X". 7 December 2015. Retrieved 21 July 2022.