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Sodium Benzoate

Phase 2Small molecule

LLiminal BioSciences Ltd.

Sodium benzoate is used to treat a rare genetic condition called urea cycle disorder, where the body cannot properly break down ammonia, which can damage the brain. It helps the body remove excess ammonia by providing an alternative pathway for ammonia elimination.[1] Sodium Benzoate is currently being investigated across 6 indications[3], with its most advanced program in Phase 2.[1]

Sodium Benzoate is one of 45 small molecule programs targeting Hyperammonemia. It is currently in Phase 2.

6

Indications

29

Total Trials

3

Active Trials

44

Competitors

Pipeline

Status by Indication[3][1]

Indication

HyperammonemiaActive, not recruiting

DiabetesNot yet recruiting

AINUnknown status

Combined D,L-2-Hydroxyglutaric AciduriaEnrolling by invitation

Endoplasmic Reticulum StressCompleted

Medium-Chain Acyl-Coa Dehydrogenase DeficiencySuspended

HyperammonemiaPreclinical

Active, not recruiting

DiabetesPhase 1

Not yet recruiting

AINPreclinical

Unknown status

Combined D,L-2-Hydroxyglutaric AciduriaPhase 1

Enrolling by invitation

Endoplasmic Reticulum StressPreclinical

Completed

Medium-Chain Acyl-Coa Dehydrogenase DeficiencyPhase 2

Suspended

Competition

Competitive Landscape

30 competitors in AIN

#DrugCompanyPhaseTrials

Reblozyl

Bristol-Myers Squibb

Jevtana

Plaquenil

Jazz Pharmaceuticals

Lynparza

Avastin

GM-CSF

YY-mAbs Therapeutics

Paraplatin

Ayvakit

BBlueprint Medicines

Brukinsa

BBeOne Medicines

Rayos

RRoxane Laboratories

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2028

Phase 2Recruiting

Medium-Chain Acyl-Coa Dehydrogenase Deficiency

NCT06773026

Q2 2029

Phase 1Not yet recruiting

Diabetes

NCT06645769

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy3 trials

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

Terminated·WWestat·Phase 1/2·NCT00439218·5 enrolled

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

Terminated·WWestat·Phase 1/2·NCT00439569·9 enrolled

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

Completed·UUniversity of Utah·Phase 1/2·NCT00528268·22 enrolled

Medium-chain Acyl-CoA Dehydrogenase Deficiency2 trials

A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation

Recruiting·JJerry Vockley, MD, PhD·Phase 2·NCT06773026·24 enrolled

A Phase 2, Open-Label, Fixed Dose Study to Evaluate the Use of Sodium Phenylbutyrate (ACER-001) in the Treatment of Pediatric and Adult Patients With MCAD Deficiency Caused by the Common ACADM c.985 A>G (K304E) Mutation

Suspended·JJerry Vockley, MD, PhD·Phase 2·NCT06069375·24 enrolled

Urea Cycle Disorders1 trial

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

Completed·—·N/A·NCT02111200·7 enrolled

Acute Myeloid Leukemia1 trial

Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00006240

Spinocerebellar ataxia type 31 trial

Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3

Withdrawn·HHospital de Clinicas de Porto Alegre·Phase 2·NCT01096095

20 more indications

Hematologic Cancer1 trial

Phenylbutyrate and Tretinoin in Treating Patients With Hematologic Cancer

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT00006239

Myelodysplastic Syndrome1 trial

Azacitidine Plus Phenylbutyrate in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT00004871

Colorectal Cancer1 trial

Phase I-II Study of Fluorouracil in Combination With Phenylbutyrate in Advanced Colorectal Cancer

Terminated·NNational Cancer Institute (NCI)·Phase 1/2·NCT00002796·46 enrolled

Huntington's Disease1 trial

Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD)

Completed·UUniversity of Rochester·Phase 2·NCT00212316·60 enrolled

Relapsed or Refractory1 trial

Phenylbutyrate and Valganciclovir in Treating Patients With Relapsed or Refractory Epstein-Barr Virus-Positive Cancer

Withdrawn·UUniversity of California, San Diego·Phase 2·NCT00387530

Insulin Secretion1 trial

Mechanism of Fatty Acid-induced Impairment of Glucose-simulated Insulin Secretion - Effect of Buphenyl

Completed·UUniversity Health Network, Toronto·Phase 4·NCT00533559·10 enrolled

Non-Small Cell Lung Cancer1 trial

Phenylbutyrate Plus Azacitidine in Treating Patients With Acute Myeloid Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Multiple Myeloma, Non-small Cell Lung Cancer, or Prostate Cancer

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00006019

Age1 trial

A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate

Terminated·LLiminal BioSciences Ltd.·Phase 1·NCT05349435·8 enrolled

Lymphoma1 trial

Phenylbutyrate in Treating Patients With Refractory Solid Tumors or Lymphoma

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT00002909·24 enrolled

Endoplasmic Reticulum Stress1 trial

Effect of Endoplasmic Reticulum Stress on Metabolic Function

Completed·WWashington University School of Medicine·N/A·NCT00771901·101 enrolled

AIN1 trial

The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino Acids

Unknown status·UUniversity of Nottingham·N/A·NCT05028803·6 enrolled

Argininosuccinic Aciduria1 trial

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

Completed·BBrendan Lee·Phase 2·NCT00345605·12 enrolled

Psychosis1 trial

Sodium Benzoate for Treatment of Attenuated/Transient Psychosis. A Randomized Placebo-controlled Trial.

Withdrawn·—·Phase 2·NCT02026947

Hyperammonemia1 trial

Efficacy and Safety of Sodium Benzoate in the Management of Hyperammonemia in Infants, Children and Adolescent With Chronic Liver Disease.

Completed·IInstitute of Liver and Biliary Sciences, India·N/A·NCT04022941·108 enrolled

Schizophrenia1 trial

Sodium Benzoate and/or N-Acetylcysteine Added to TAU in Patients With Early Schizophrenia Spectrum Disorder.

Completed·PPakistan Institute of Living and Learning·Phase 2·NCT03510741·64 enrolled

Amyotrophic Lateral Sclerosis1 trial

Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)

Completed·UUS Department of Veterans Affairs·Phase 1/2·NCT00107770·40 enrolled

Advanced or Metastatic Solid Tumors1 trial

Azacitidine Plus Phenylbutyrate in Treating Patients With Advanced or Metastatic Solid Tumors That Have Not Responded to Previous Treatment

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT00005639·34 enrolled

Dietary Intervention1 trial

Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders

Completed·NNational Center for Research Resources (NCRR)·Phase 2·NCT00004767·20 enrolled

Diabetes1 trial

Na-Phenylbutyrate VAscular Trial

Not yet recruiting·UUniversity of Pennsylvania·Phase 1·NCT06645769·30 enrolled

Unspecified2 trials

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

Enrolling by invitation·—·Phase 1·NCT07125066·1 enrolled

Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.

Completed·FFondazione Telethon·Phase 2·NCT03734263·1 enrolled

Sources

References

[1]
ClinicalTrials.gov — 29 trial records for Sodium Benzoate. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Sodium Benzoate compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Sodium Benzoate. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Liminal BioSciences Ltd. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: LLiminal BioSciences Ltd.

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