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Trofinetide - Neuren Pharmaceuticals

Drug Profile

Trofinetide - Neuren Pharmaceuticals

Alternative Names: G-2Me-PE; Glycyl-2-methyl-L-prolyl-L-glutamic acid; Glycyl-L-2-methylprolyl-L-glutamic acid; NNZ-2566

Latest Information Update: 07 Nov 2019

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At a glance

  • Originator Neuren Pharmaceuticals
  • Developer Neuren Pharmaceuticals; Rettsyndrome.org; Walter Reed Army Institute of Research
  • Class Anti-inflammatories; Glutamates; Neuropeptides; Neuroprotectants; Oligopeptides; Pyrrolidines; Small molecules
  • Mechanism of Action Cytokine inhibitors; Free radical modulators; Glial cell modulators; Neuropeptide receptor modulators; Synaptic transmission modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Fragile X syndrome; Rett syndrome
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Rett syndrome
  • Phase II Brain injuries; Fragile X syndrome
  • No development reported Autistic disorder; Multiple sclerosis

Most Recent Events

  • 30 Oct 2019 Phase-III clinical trials in Rett syndrome (In adolescents, In children) in USA (PO)
  • 30 Oct 2019 Acadia Pharmaceuticals plans a phase III LILAC extension trial for LAVENDER trial in Rett syndrome in the US
  • 27 Mar 2019 Efficacy data from a phase II trial in Rett syndrome (In children, In adolescents) released by Neuren Pharmaceuticals
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