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tauroursodeoxycholic acid   Click here for help

GtoPdb Ligand ID: 4746

Abbreviated name: TUDA
Synonyms: Relyvrio® (sodium phenylbutyrate + taurursodiol) | tauroursodeoxycholate | taurursodiol | TUDCA | UR 906 | ursodoxicoltaurine (INN)
Approved drug PDB Ligand Immunopharmacology Ligand
tauroursodeoxycholic acid is an approved drug (FDA (2022))
Compound class: Metabolite
Comment: Tauroursodeoxycholic acid (TUDCA) is a conjugated bile acid derivative. Evidence suggests that it acts as a chemical chaperone and facilitates protein folding within the endoplasmic reticulum [2].
2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 7
Hydrogen bond donors 4
Rotatable bonds 8
Topological polar surface area 132.31
Molecular weight 499.3
XLogP 4.86
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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Canonical SMILES OC1CCC2(C(C1)CC(C1C2CCC2(C1CCC2C(CCC(=O)NCCS(=O)(=O)O)C)C)O)C
Isomeric SMILES O[C@@H]1CC[C@]2([C@@H](C1)C[C@@H]([C@@H]1[C@@H]2CC[C@]2([C@H]1CC[C@@H]2[C@@H](CCC(=O)NCCS(=O)(=O)O)C)C)O)C
InChI InChI=1S/C26H45NO6S/c1-16(4-7-23(30)27-12-13-34(31,32)33)19-5-6-20-24-21(9-11-26(19,20)3)25(2)10-8-18(28)14-17(25)15-22(24)29/h16-22,24,28-29H,4-15H2,1-3H3,(H,27,30)(H,31,32,33)/t16-,17+,18-,19-,20+,21+,22+,24+,25+,26-/m1/s1
InChI Key BHTRKEVKTKCXOH-LBSADWJPSA-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/)

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Summary of Clinical Use Click here for help
An orally bioavailable fixed-dose coformulation containing tauroursodeoxycholic acid (taurursodiol) and sodium phenylbutyrate, known as AMX0035 (PB-TURSO; Amylyx Pharmaceuticals) was investigated for clinical efficacy in neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) [1,5-6]. Sodium phenylbutyrate and taurursodiol target endoplasmic reticulua and mitochondria neuronal stress, and the combined actions reduce neuronal death in experimental models, which suggests potential to treat human neurodegeneration [3,8]. The FDA approved the AMX0035 coformulation (Relyvrio®) as a treatment for ALS in September 2022, based on positive evidence from a phase 2 study. However, in March 2024 Amylyx announced that Relyvrio® failed to meet primary and secondary endpoints in a larger and longer confirmatory ALS clinical trial. In April 2024 approval was withdrawn in the US and Canada.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03127514 AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) Phase 2/Phase 3 Interventional Amylyx Pharmaceuticals Inc. 5-6
NCT05619783 Extension Study Evaluating The Safety And Tolerability of AMX0035 Phase 3 Interventional Amylyx Pharmaceuticals Inc. This phase 3 study (PHOENIX) was intended as a larger confirmatory trial of Relyvrio® in ALS patients, following the 2022 approval by the FDA. Unfortunately the therapy failed to meet primary and secondary enpoints in this trial.