Trofinetide: a pioneering treatment for Rett syndrome

STRUCTURE: Trofinetide [(2S)-2-{[(2S)-1-(2-aminoacetyl)-2-methylpyrrolidine-2-carbonyl]amino}pentanedioic acid] is derived from the tripeptide glycine-proline-glutamic acid (GPE), which is an endogenous tripeptide of the N-terminal domain of insulin-like growth factor 1 (IGF-1). The compound differs from endogenous GPE by a methyl (−CH3) substitution at the α-carbon of the proline residue. This methyl substitution assists in resistance to protease cleavage and improves oral bioavailability. The molecular formula of trofinetide is C13H21N3O6, and the molecular weight is 315.33 g/mol.