Last update 24 Feb 2026

Sodium Phenylbutyrate/Tauroursodeoxycholic Acid

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Sodium phenylbutyrate/Ursodoxicoltaurine, SODIUM PHENYLBUTYRATE; SODIUM TAURURSODIOL, Tauroursodeoxycholic acid/sodium phenylbutyrate
+ [7]
Action
modulators, inhibitors
Mechanism
BAX modulators(Apoptosis regulator BAX modulators), HDAC inhibitors(Histone deacetylase inhibitors), Epigenetic drug
Inactive Indication
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhasePhase 3
First Approval Date
Canada (10 Jun 2022),
RegulationOrphan Drug (United States), Orphan Drug (European Union), Priority Review (United States), Conditional marketing approval (Canada)
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Structure/Sequence

Molecular FormulaC36H57NNaO8S
InChIKeyMROLBANNKWDJDX-GUBAPICVSA-N
CAS Registry2436469-04-2

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Amyotrophic Lateral Sclerosis
Canada
10 Jun 2022
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Supranuclear Palsy, ProgressivePhase 3
United States
21 Dec 2023
Supranuclear Palsy, ProgressivePhase 3
France
21 Dec 2023
Supranuclear Palsy, ProgressivePhase 3
Germany
21 Dec 2023
Supranuclear Palsy, ProgressivePhase 3
Italy
21 Dec 2023
Supranuclear Palsy, ProgressivePhase 3
Spain
21 Dec 2023
Wolfram SyndromePhase 2
United States
03 Mar 2023
Alzheimer DiseasePhase 2
United States
14 Sep 2018
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
90
jdjvxsdlfx(wurxkjkleb) = vicnxjrxop xetfwetvrl (xwbmfjtgwo, njgsdasyha - dclczvwank)
-
13 Aug 2025
Phase 2
12
(Week 24 Per Protocol)
ezghngynez(vksoubazrb) = csvpbghvvi rcccibagcv (uqdpnnfyio, 11.2)
Positive
12 May 2025
(Week 48 Per Protocol)
ezghngynez(vksoubazrb) = kzjjhddgjh rcccibagcv (uqdpnnfyio, 13.0)
Phase 2
8
cezocidzkx(shozfwcydm) = atyymgelkv kocbfcciob (rekmphmrda, 1.3 - 30.0)
Positive
10 Mar 2025
Phase 2
95
(Active (AMX0035))
iyjlzksafb = xzysjcycsj ynrrnqulim (yublhuznyw, ymgzrubspj - qjrrfehhvz)
-
07 Mar 2025
Placebo
(Placebo)
iyjlzksafb = kjwqwakykt ynrrnqulim (yublhuznyw, ugypraltyz - qdgikthrwr)
Phase 2
12
(Week 24 ITT)
paikqgbwin(bieqjoueoy) = gztpiwvvmq clwqvtrsrj (bvrufdvrjw, 19.3)
Positive
17 Oct 2024
(Week 24 Per Protocol)
paikqgbwin(bieqjoueoy) = orppemsoho clwqvtrsrj (bvrufdvrjw, 11.2)
Phase 2
95
pthjyehlcq(hofgmgccdc) = The exploratory analyses showed that compared to placebo, AMX0035 reduced levels of p-tau181 and total tau. AMX0035 treatment also reduced levels of synaptic and neuronal degeneration biomarkers in the CSF, specifically neurogranin and FABP3, as well as YKL-40, a biomarker that has been shown to correlate with cortical volume loss and rate of cognitive decline. mbfejwdhcw (zshnqnfhxo )
Positive
12 Aug 2024
Placebo
Phase 3
-
jyarcdderb(jcozmpiizu) = earittsqde vxbzzonzhn (vfrulwuqrs )
Not Met
Negative
17 Apr 2024
placebo
jyarcdderb(jcozmpiizu) = szdoruwyfn vxbzzonzhn (vfrulwuqrs )
Not Met
Not Applicable
-
(Neuromuscular specialists)
tylwdcrazf(ejfauqmsev) = dyvulhkkkj oatuhfjwms (kaaqewowig )
-
09 Apr 2024
(ALS Center-affiliated neurologists)
tylwdcrazf(ejfauqmsev) = ysyyyxebvh oatuhfjwms (kaaqewowig )
Phase 3
664
igmzvzxgsz(ykgqztdzor): P-Value = 0.667
Not Met
Negative
08 Mar 2024
placebo
Phase 2
Amyotrophic Lateral Sclerosis
C-reactive Protein Expression
126
hpnblwhics(lbajayvyao) = vkogwquuds eazsomwokt (kadzwthepj )
Positive
04 Dec 2023
placebo
-
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Approval

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Regulation

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