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))
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].
Click here for help
2D Structure
Click here for help
Click here for structure editor
Physico-chemical Properties
Click here for help
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
Click here for help
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
No information available.
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. The future for use of Relyvrio® remains to be decided.
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.