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sepiapterin   Click here for help

GtoPdb Ligand ID: 14278

Synonyms: CNSA-001 [1] | L-sepiapterin | PTC-923 | PTC923 | Sephience®
Approved drug
sepiapterin is an approved drug
Compound class: Synthetic organic
Comment: Sepiapterin (PTC923) is an orally bioavailable phenylalanine hydroxylase (PAH) activator. It is a precursor of the PAH cofactor BH4 (tetrahydrobiopterin/sapropterin).
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Click here for structure editor
Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 8
Hydrogen bond donors 4
Rotatable bonds 2
Topological polar surface area 129.17
Molecular weight 237.22
XLogP -0.67
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES C[C@@H](C(=O)C1=NC2=C(NC1)NC(=NC2=O)N)O
Isomeric SMILES O=C1C2=C(NCC(C([C@H](C)O)=O)=N2)NC(N)=N1
InChI InChI=1S/C9H11N5O3/c1-3(15)6(16)4-2-11-7-5(12-4)8(17)14-9(10)13-7/h3,15H,2H2,1H3,(H4,10,11,13,14,17)/t3-/m0/s1
InChI Key VPVOXUSPXFPWBN-VKHMYHEASA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
References
1. Abell TL, Garcia LM, Wiener GJ, Wo JM, Bulat RS, Smith N. (2021)
Effect of Oral CNSA-001 (sepiapterin, PTC923) on gastric accommodation in women with diabetic gastroparesis: A randomized, placebo-controlled, Phase 2 trial.
J Diabetes Complications, 35 (9): 107961. [PMID:34176722]
2. Bratkovic D, Margvelashvili L, Tchan MC, Nisbet J, Smith N. (2022)
PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: a phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study.
Metabolism, 128: 155116. [PMID:34973284]
3. Lamb YN. (2025)
Sepiapterin: First Approval.
Drugs, [Epub ahead of print]. [PMID:41091365]
4. Muntau AC, Longo N, Ezgu F, Schwartz IVD, Lah M, Bratkovic D, Margvelashvili L, Kiykim E, Zori R, Campistol Plana J et al.. (2024)
Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial.
Lancet, 404 (10460): 1333-1345. [PMID:39368841]
5. Smith N, Longo N, Levert K, Hyland K, Blau N. (2019)
Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers.
Mol Genet Metab, 126 (4): 406-412. [PMID:30922814]