Publication: Exploratory study of the effect of 1 week of orally-administered CNSA-001 (sepiapterin) on central nervous system (CNS) levels of tetrahydrobiopterin, dihydrobiopterin, and monoamine neurotransmitter metabolites in healthy volunteers

Published in Molecular Genetics and Metabolism Reportsthis research article presents the results of a study investigating the potential effects of CNSA-001 – a preparation of sepiapterin – on neurometabolites in the CNS 

  • Results from a Phase 1 exploratory study are presented in this publication. Concentrations of molecules involved in neurometabolic pathways were recorded at baseline and day 7 in 8 healthy subjects who were administered oral CNSA-001 (n=6) or placebo (n=2) over 7 days
  • Levels of metabolites of interest measured in cerebrospinal fluid and plasma in this study – BH4, BH2, homovanillic acid, and 5-hydroxyindoleacetic acid (5-HIAA) – are presented

Smith N, Longo N, Levert K, et al.Mol Genet Metab Rep. 2019;21:100500 

Sepiapterin is an investigational drug currently in development for PKU.

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