Vatiquinone
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Also known as (r)-alpha-tocotrienol quinone.alpha.-tocotrienol quinoneAlpha tocotrienol quinoneAlpha-tocotrienol quinoneEPI-743PTC-743PTC743VatiquinonaUS11584728Compound Vatiquinone
Summary
Vatiquinone (CHEMBL1812161) is a phase-3 clinical-stage small molecule targeting ALOX15; indicated across 9 conditions including inborn mitochondrial metabolism disorder and friedreich ataxia.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Small molecule
- Targets: 1 (ALOX15)
- Indications: 9 conditions
- Clinical trials: 20
- Chemistry: 440.7 Da · C29H44O3
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1812161 |
| Name | Vatiquinone |
| Type | Small molecule |
| Max phase | 3 |
| FDA approved | no |
| PubChem CID | 46184405 |
| Molecular formula | C29H44O3 |
| Molecular weight | 440.7 |
| InChIKey | LNOVHERIIMJMDG-XZXLULOTSA-N |
SMILES: CC1=C(C(=O)C(=C(C1=O)C)CC[C@@](C)(CC/C=C(\C)/CC/C=C(\C)/CCC=C(C)C)O)C
IUPAC name: 2-[(3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl]-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione
Also known as: (r)-alpha-tocotrienol quinone, .alpha.-tocotrienol quinone, Alpha tocotrienol quinone, Alpha-tocotrienol quinone, EPI-743, PTC-743, PTC743, Vatiquinona, Vatiquinone, (R)-alpha-Tocotrienol quinone, VATIQUINONE, US11584728
Patent coverage: 66 distinct patent families (181 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| ALOX15 | 15-LOX-1 | Inhibition | 6.23 | 0.2% | P16050 |
Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Polyunsaturated fatty acid lipoxygenase ALOX15.
Bioactivity
ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| ALOX15 | 6.23 | IC50 | 590 | nM | CHEMBL_ACT_28947989 |
Target pathways
Aggregated over 1 target gene(s): ALOX15.
Top Reactome pathways
21 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Cytokine Signaling in Immune system | 1 | ALOX15 |
| Metabolism | 1 | ALOX15 |
| Immune System | 1 | ALOX15 |
| Synthesis of Leukotrienes (LT) and Eoxins (EX) | 1 | ALOX15 |
| Synthesis of 12-eicosatetraenoic acid derivatives | 1 | ALOX15 |
| Arachidonate metabolism | 1 | ALOX15 |
| Synthesis of 15-eicosatetraenoic acid derivatives | 1 | ALOX15 |
| Signaling by Interleukins | 1 | ALOX15 |
| Metabolism of lipids | 1 | ALOX15 |
| Interleukin-4 and Interleukin-13 signaling | 1 | ALOX15 |
| Fatty acid metabolism | 1 | ALOX15 |
| Biosynthesis of DHA-derived SPMs | 1 | ALOX15 |
| Biosynthesis of specialized proresolving mediators (SPMs) | 1 | ALOX15 |
| Biosynthesis of EPA-derived SPMs | 1 | ALOX15 |
| Biosynthesis of protectins | 1 | ALOX15 |
| Biosynthesis of DPA-derived SPMs | 1 | ALOX15 |
| Biosynthesis of E-series 18(S)-resolvins | 1 | ALOX15 |
| Biosynthesis of E-series 18(R)-resolvins | 1 | ALOX15 |
| Biosynthesis of DPAn-3 SPMs | 1 | ALOX15 |
| Biosynthesis of DPAn-6 SPMs | 1 | ALOX15 |
| Biosynthesis of DPAn-3-derived protectins and resolvins | 1 | ALOX15 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| ossification | 1 |
| negative regulation of adaptive immune response | 1 |
| lipid metabolic process | 1 |
| phosphatidylethanolamine biosynthetic process | 1 |
| inflammatory response | 1 |
| positive regulation of cell-substrate adhesion | 1 |
| arachidonate metabolic process | 1 |
| lipoxygenase pathway | 1 |
| fatty acid oxidation | 1 |
| bone mineralization | 1 |
| positive regulation of actin filament polymerization | 1 |
| lipid oxidation | 1 |
| response to endoplasmic reticulum stress | 1 |
| regulation of peroxisome proliferator activated receptor signaling pathway | 1 |
| cellular response to interleukin-13 | 1 |
Indications & clinical
Indications
9 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| inborn mitochondrial metabolism disorder | 3 | MONDO:0004069 | MONDO:0044970 |
| Friedreich ataxia | 3 | MONDO:0100339 | MONDO:0100339 |
| autism spectrum disorder | 2 | MONDO:0005258 | EFO:0003756 |
| noise induced hearing loss | 2 | MONDO:0013098 | EFO:1001254 |
| Parkinson disease | 2 | MONDO:0005180 | MONDO:0005180 |
| Leigh syndrome | 2 | MONDO:0009723 | MONDO:0009723 |
| Rett syndrome | 2 | MONDO:0010726 | MONDO:0010726 |
| Tourette syndrome | 1 | MONDO:0007661 | EFO:0004895 |
| myopathy | 1 | MONDO:0005336 | EFO:0004145 |
Clinical trials
Total trials: 20.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 11 |
| Not specified | 3 |
| PHASE2/PHASE3 | 2 |
| PHASE3 | 2 |
| PHASE1/PHASE2 | 1 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05515536 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia |
| NCT04378075 | PHASE2/PHASE3 | TERMINATED | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy |
| NCT04577352 | PHASE2/PHASE3 | COMPLETED | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia |
| NCT05218655 | PHASE3 | COMPLETED | A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease |
| NCT01642056 | PHASE1/PHASE2 | COMPLETED | EPI-743 for Metabolism or Mitochondrial Disorders |
| NCT01721733 | PHASE2 | COMPLETED | Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome |
| NCT01728064 | PHASE2 | COMPLETED | Safety and Efficacy of EPI-743 in Patients With Friedreich’s Ataxia |
| NCT01793090 | PHASE2 | COMPLETED | EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment |
| NCT01822249 | PHASE2 | COMPLETED | Phase 2 Study of EPI-743 for Treatment of Rett Syndrome |
| NCT01923584 | PHASE2 | COMPLETED | A Phase 2A Trial of EPI-743 for Patients With Parkinson’s Disease |
| NCT01962363 | PHASE2 | COMPLETED | EPI-743 in Friedreich’s Ataxia Point Mutations |
| NCT02104336 | PHASE2 | TERMINATED | Phase 2 Study of EPI-743 in Children With Pearson Syndrome |
| NCT02226458 | PHASE2 | WITHDRAWN | An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder |
| NCT02257983 | PHASE2 | COMPLETED | Protective Effects of EPI-743 on Noise-Induced Hearing Loss |
| NCT02352896 | PHASE2 | COMPLETED | Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome |
| NCT05485987 | PHASE2 | COMPLETED | A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia |
| NCT01719523 | PHASE1 | COMPLETED | Open-Trial of EPI-743 for Adults With Tourette Syndrome |
| NCT01370447 | Not specified | NO_LONGER_AVAILABLE | EPI-743 for Mitochondrial Respiratory Chain Diseases |
| NCT02300753 | Not specified | NO_LONGER_AVAILABLE | Emergency Administration of EPI-743 to a Single Patient With Leber’s Hereditary Optic Neuropathy [LHON] |
| NCT07159139 | Not specified | NO_LONGER_AVAILABLE | Vatiquinone Expanded Access Protocol |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.
Related molecules
Related molecules
Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.
170 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| CELECOXIB | ChEMBL + PubChem | Phase 4 (approved) | ALOX15 |
| HEXYLRESORCINOL | ChEMBL + PubChem | Phase 4 (approved) | ALOX15 |
| ACETYLCYSTEINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| AMINOCAPROIC ACID | ChEMBL | Phase 4 (approved) | ALOX15 |
| AMINOPYRINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| AMISULPRIDE | ChEMBL | Phase 4 (approved) | ALOX15 |
| AVOBENZONE | ChEMBL | Phase 4 (approved) | ALOX15 |
| BETAHISTINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| BITHIONOL | ChEMBL | Phase 4 (approved) | ALOX15 |
| BITHIONOLATE | ChEMBL | Phase 4 (approved) | ALOX15 |
| BRIMONIDINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| BUSULFAN | ChEMBL | Phase 4 (approved) | ALOX15 |
| CANDESARTAN CILEXETIL | ChEMBL | Phase 4 (approved) | ALOX15 |
| CAPSAICIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| CAPTOPRIL | ChEMBL | Phase 4 (approved) | ALOX15 |
| CARBIDOPA | ChEMBL | Phase 4 (approved) | ALOX15 |
| CARBOPLATIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| CHLORHEXIDINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| CHLOROXINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| CHOLECALCIFEROL | ChEMBL | Phase 4 (approved) | ALOX15 |
| CISPLATIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| CLIOQUINOL | ChEMBL | Phase 4 (approved) | ALOX15 |
| CLOTRIMAZOLE | ChEMBL | Phase 4 (approved) | ALOX15 |
| CYCLOBENZAPRINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| CYCLOPHOSPHAMIDE | ChEMBL | Phase 4 (approved) | ALOX15 |
| DACTINOMYCIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| DANTHRON | ChEMBL | Phase 4 (approved) | ALOX15 |
| DEQUALINIUM | ChEMBL | Phase 4 (approved) | ALOX15 |
| DIBUCAINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| DIENESTROL | ChEMBL | Phase 4 (approved) | ALOX15 |
| DIETHYLCARBAMAZINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| DIETHYLSTILBESTROL | ChEMBL | Phase 4 (approved) | ALOX15 |
| DISULFIRAM | ChEMBL | Phase 4 (approved) | ALOX15 |
| DOPAMINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| ENOXIMONE | ChEMBL | Phase 4 (approved) | ALOX15 |
| ESCITALOPRAM | ChEMBL | Phase 4 (approved) | ALOX15 |
| ETHOPROPAZINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| ETODOLAC | ChEMBL | Phase 4 (approved) | ALOX15 |
| FEXOFENADINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| FLUPIRTINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| FLUSPIRILENE | ChEMBL | Phase 4 (approved) | ALOX15 |
| FLUTAMIDE | ChEMBL | Phase 4 (approved) | ALOX15 |
| FURAZOLIDONE | ChEMBL | Phase 4 (approved) | ALOX15 |
| GALLAMINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| GATIFLOXACIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| GLUTAMINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| HALOPERIDOL | ChEMBL | Phase 4 (approved) | ALOX15 |
| HEXACHLOROPHENE | ChEMBL | Phase 4 (approved) | ALOX15 |
| HEXESTROL | ChEMBL | Phase 4 (approved) | ALOX15 |
| HYDROQUINONE | ChEMBL | Phase 4 (approved) | ALOX15 |
| HYDROXYZINE PAMOATE | ChEMBL | Phase 4 (approved) | ALOX15 |
| INAMRINONE | ChEMBL | Phase 4 (approved) | ALOX15 |
| INDIUM CHLORIDE | ChEMBL | Phase 4 (approved) | ALOX15 |
| ISRADIPINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| LANSOPRAZOLE | ChEMBL | Phase 4 (approved) | ALOX15 |
| LEVODOPA | ChEMBL | Phase 4 (approved) | ALOX15 |
| LEVONORDEFRIN | ChEMBL | Phase 4 (approved) | ALOX15 |
| LOPERAMIDE | ChEMBL | Phase 4 (approved) | ALOX15 |
| LORATADINE | ChEMBL | Phase 4 (approved) | ALOX15 |
| LOVASTATIN | ChEMBL | Phase 4 (approved) | ALOX15 |
Related Atlas pages
- Genes: ALOX15
- Diseases: inborn mitochondrial metabolism disorder, Friedreich ataxia
- Drugs: Celecoxib, Hexylresorcinol, Acetylcysteine, Aminocaproic Acid, Aminopyrine, Amisulpride, Avobenzone, Betahistine, Bithionol, Brimonidine, Busulfan, Candesartan Cilexetil, Capsaicin, Captopril, Carbidopa, Carboplatin, Chlorhexidine, Chloroxine, Cholecalciferol, Cisplatin, Clioquinol, Clotrimazole, Cyclobenzaprine, Cyclophosphamide, Dactinomycin, Danthron, Dequalinium, Dibucaine, Dienestrol, Diethylcarbamazine, Diethylstilbestrol, Disulfiram, Dopamine, Enoximone, Escitalopram, Ethopropazine, Etodolac, Fexofenadine, Flupirtine, Fluspirilene, Flutamide, Furazolidone, Gallamine, Gatifloxacin, Glutamine, Haloperidol, Hexachlorophene, Hexestrol, Hydroquinone, Hydroxyzine Pamoate, Inamrinone, Indium Chloride, Isradipine, Lansoprazole, Levodopa, Levonordefrin, Loperamide, Loratadine, Lovastatin