Legal status
Legal status
  • Investigational New Drug
CAS Number 853400-76-7 YesY
PubChem (CID) 11318905
Chemical and physical data
Formula C13H21N3O6
Molar mass 315.322
3D model (Jmol) Interactive image

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 formed 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 in Phase II clinical trials against Fragile X syndrome and Rett syndrome.[3][4][5]


Neuren Pharmaceuticals was created in 2004 via a merger of two companies that were formed around the University of Auckland in Auckland, New Zealand. The company now has registered offices in Australia and New Zealand. In 2005, Neuren Pharmaceuticals was listed on the Australian Securities Exchange (ASX)[6] As a company that is publicly traded, Neuren Pharmaceuticals receives a large portion of its current funding from shareholders, since it currently has no drug products on the market. It is also largely funded by grants from companies or agencies with an interest in their project.[7] In 2007, acquired venture-backed Hamilton Pharmaceuticals for $4.4 million in stock. Since the acquisition was stock-based, the three venture funds that had previously backed Hamilton Pharmaceuticals, Vivo Ventures, CNF Investments, and Index Ventures, became stockholders in Neuren Pharmaceuticals.[8] As a result of the acquisition, Neuren pharmaceuticals gained control of the drug Motiva, a drug to aid treatment of neurological disorders such as Parkinson's and Alzheimer's disease. Motiva did not pass phase 2 clinical trials.[8]

Neuren Pharmaceuticals received several grants from various sources for research on Trofinetide, including $26 million in funding from the United States army, who was interested in the drug for its use on TBI. This grant did include one point stating that an army scientist had to be used as a principal investigator.[6] They also received funding from International Rett Syndrome Foundation, FRAXA, and National Fragile X Foundation at significantly lesser value.[9]


Neuren's analyst through Bell Porter reported that the company's estimated earnings before interest, taxation, depreciation, and amortization will be 84$ million at the end of 2016. This amount is expected to decrease by the end of 2017 to $27 million.[10] The promising results include estimates from upcoming licensing deals for Trofinetide and payment milestones from those deals. The product is expected to be available for commercial sale in 2019 for Rett Syndrome and in 2020 for Fragile X Syndrome.[10]


  1. Bickerdike MJ, Thomas GB, Batchelor DC, Sirimanne ES, Leong W, Lin H, Sieg F, Wen J, Brimble MA, Harris PW, Gluckman PD. NNZ-2566: a Gly-Pro-Glu analogue with neuroprotective efficacy in a rat model of acute focal stroke. J Neurol Sci. 2009 Mar 15;278(1-2):85-90. doi: 10.1016/j.jns.2008.12.003. PMID 19157421
  2. Cartagena CM, Phillips KL, Williams GL, Konopko M, Tortella FC, Dave JR, Schmid KE. Mechanism of action for NNZ-2566 anti-inflammatory effects following PBBI involves upregulation of immunomodulator ATF3. Neuromolecular Med. 2013 Sep;15(3):504-14. doi: 10.1007/s12017-013-8236-z. PMID 23765588
  3. Deacon RM, Glass L, Snape M, Hurley MJ, Altimiras FJ, Biekofsky RR, Cogram P. NNZ-2566, a novel analog of (1-3) IGF-1, as a potential therapeutic agent for fragile X syndrome. Neuromolecular Med. 2015 Mar;17(1):71-82. doi: 10.1007/s12017-015-8341-2. PMID 25613838
  4. Study Details - Rett Syndrome Study
  5. Neuren’s trofinetide successful in Phase 2 clinical trial in Fragile X
  6. 1 2 "Neuren Pharmaceuticals Investor Presentation". Neuren Pharmaceuticals. May, 2013. Retrieved 2016-11-13. Check date values in: |date= (help)
  7. "About Us | Neuren Pharmaceuticals". Retrieved 2016-11-14.
  8. 1 2 "New Zealand's Neuren Pharma snaps up Hamilton Pharma for $4.4M". VentureBeat. Retrieved 2016-11-14.
  9. "Investor Presentation". Neuren Pharmaceuticals. January 2016. Retrieved 2016-11-19.
  10. 1 2 Jain, Tanushree (April 27, 2016). "Neuren". Bell Porter. Bell Porter. Retrieved 2016-11-19.
This article is issued from Wikipedia - version of the 12/3/2016. The text is available under the Creative Commons Attribution/Share Alike but additional terms may apply for the media files.