Pyridostigmine
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Also known as Pyridostigmine cationPyridostigmine ionSID11111694SID90340824PYRIDOSTIGMINE BROMIDEMESTINONPyridostigmine Bromide (Mestinon)
Summary
Pyridostigmine (CHEMBL1115) is an approved small molecule (ATC N07AA02) targeting ACHE and BCHE; indicated across 7 conditions including myasthenia gravis and hiv infectious disease.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: N07AA02
- Targets: 2 (ACHE, BCHE)
- Indications: 7 conditions
- Clinical trials: 36
- Chemistry: 181.21 Da · C9H13N2O2+
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1115 |
| Name | Pyridostigmine |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 4991 |
| ATC | N07AA02 |
| Molecular formula | C9H13N2O2+ |
| Molecular weight | 181.21 |
| InChIKey | RVOLLAQWKVFTGE-UHFFFAOYSA-N |
SMILES: C[N+]1=CC=CC(=C1)OC(=O)N(C)C
IUPAC name: (1-methylpyridin-1-ium-3-yl) N,N-dimethylcarbamate
Also known as: Pyridostigmine, Pyridostigmine cation, Pyridostigmine ion, SID11111694, SID90340824, pyridostigmine, PYRIDOSTIGMINE, PYRIDOSTIGMINE BROMIDE, MESTINON, Pyridostigmine Bromide (Mestinon)
Parent form; salt/anhydrous children: CHEMBL812, CHEMBL4303667
Patent coverage: 3,984 distinct patent families (13,658 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 13,643 (100%) of the total. 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 |
|---|---|---|---|---|---|
| ACHE | acetylcholinesterase (Yt blood group) | Inhibition | 6.44 | 9.6% | P22303 |
| BCHE | butyrylcholinesterase | Inhibition | 6.05 | 0.1% | P06276 |
Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Inositol monophosphatase 1, Cholinesterase, Thyrotropin receptor, Acetylcholinesterase, Muscarinic acetylcholine receptor M1, Mitogen-activated protein kinase 1, Acetylcholinesterase, Acetylcholinesterase.
Bioactivity
ChEMBL activities: 6 potent at pChembl ≥ 5 of 11 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| P23795 | 7.3 | IC50 | 50 | nM | CHEMBL_ACT_678060 |
| P08482 | 7.2 | Potency | 63.1 | nM | CHEMBL_ACT_4811421 |
| P04058 | 7.09 | IC50 | 82 | nM | CHEMBL_ACT_14530704 |
| ACHE | 6.43 | AC50 | 372.6 | nM | CHEMBL_ACT_25142420 |
| ACHE | 5.92 | AC50 | 1200 | nM | CHEMBL_ACT_25142183 |
| BCHE | 5.46 | Ki | 3500 | nM | CHEMBL_ACT_499430 |
Target pathways
Aggregated over 2 target gene(s): ACHE, BCHE.
Top Reactome pathways
12 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Neurotransmitter clearance | 2 | ACHE, BCHE |
| Synthesis of PC | 2 | ACHE, BCHE |
| Synthesis, secretion, and deacylation of Ghrelin | 2 | ACHE, BCHE |
| Transmission across Chemical Synapses | 1 | ACHE |
| Neuronal System | 1 | ACHE |
| Metabolism | 1 | ACHE |
| Glycerophospholipid biosynthesis | 1 | ACHE |
| Phospholipid metabolism | 1 | ACHE |
| Peptide hormone metabolism | 1 | ACHE |
| Metabolism of proteins | 1 | ACHE |
| Metabolism of lipids | 1 | ACHE |
| Aspirin ADME | 1 | BCHE |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| acetylcholine catabolic process | 2 |
| acetylcholine catabolic process in synaptic cleft | 1 |
| regulation of receptor recycling | 1 |
| osteoblast development | 1 |
| cell adhesion | 1 |
| nervous system development | 1 |
| synapse assembly | 1 |
| receptor internalization | 1 |
| negative regulation of synaptic transmission, cholinergic | 1 |
| amyloid precursor protein metabolic process | 1 |
| positive regulation of protein secretion | 1 |
| retina development in camera-type eye | 1 |
| acetylcholine receptor signaling pathway | 1 |
| positive regulation of cold-induced thermogenesis | 1 |
| xenobiotic metabolic process | 1 |
Indications & clinical
Indications
7 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 |
|---|---|---|---|
| myasthenia gravis | 3 | MONDO:0009688 | EFO:0004991 |
| HIV infectious disease | 2 | MONDO:0005109 | EFO:0000764 |
| orthostatic hypotension | 2 | MONDO:0005469 | EFO:0005252 |
| fibromyalgia | 2 | MONDO:0005546 | EFO:0005687 |
| spinal muscular atrophy | 2 | MONDO:0001516 | Orphanet:83419 |
| diabetes mellitus | 1 | MONDO:0005015 | EFO:0000400 |
1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 36.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 15 |
| PHASE4 | 7 |
| PHASE1 | 3 |
| EARLY_PHASE1 | 3 |
| Not specified | 3 |
| PHASE2/PHASE3 | 2 |
| PHASE1/PHASE2 | 2 |
| PHASE3 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT06035757 | PHASE4 | RECRUITING | The Occurrence of Emergence Agitation in Pediatric Strabismus Surgery |
| NCT02171988 | PHASE4 | COMPLETED | Effect of Medical Treatment and Prognosis of Postural Orthostatic Tachycardia Syndrome (POTS) |
| NCT02308124 | PHASE4 | COMPLETED | Treatment and Prognosis of Neurogenic Orthostatic Hypotension : A Prospective Randomized Study |
| NCT03510546 | PHASE4 | UNKNOWN | Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis |
| NCT05110417 | PHASE4 | UNKNOWN | Reversal of Botulinum Neurotoxin Injection Related Dysphonia With Pyridostigmine |
| NCT05969119 | PHASE4 | COMPLETED | Addition of Pyridostigmine to Conventional Management of Postdural Puncture Headache |
| NCT06753071 | PHASE4 | COMPLETED | The Efficacy of Pyridostigmine Therapy After Transurethral Resection of Prostate in Cases with Underactive Urinary Bladder. |
| NCT05334485 | PHASE2/PHASE3 | RECRUITING | Pyridostigmine Efficacy and Safety for Treatment of Ileus After Colorectal Surgery |
| NCT04343963 | PHASE2/PHASE3 | UNKNOWN | Pyridostigmine in Severe SARS-CoV-2 Infection |
| NCT05919407 | PHASE3 | UNKNOWN | Pyridostigmine and Amifampridine for Myasthenia Gravis |
| NCT00409435 | PHASE2 | RECRUITING | A Study of Pyridostigmine in Postural Tachycardia Syndrome |
| NCT01370512 | PHASE2 | ENROLLING_BY_INVITATION | Droxidopa / Pyridostigmine in Orthostatic Hypotension |
| NCT04353778 | PHASE1/PHASE2 | RECRUITING | Effects of Vagal Dysfunction on Gastrointestinal and Inflammatory Pathways in HIV |
| NCT05603715 | PHASE2 | RECRUITING | Pyridostigmine for the Treatment of Constipation in Parkinson Disease |
| NCT06366724 | PHASE2 | RECRUITING | LIFT: Life Improvement Trial |
| NCT00276406 | PHASE1/PHASE2 | COMPLETED | Use of Pyridostigmine for Constipation in Diabetics |
| NCT00518154 | PHASE2 | COMPLETED | Pilot Study of Pyridostigmine Upon Immune Activation in HIV-1 Patients Who Have an Inadequate Immune Response |
| NCT00535587 | PHASE2 | COMPLETED | Testing Mestinon and Exercise in Fibromyalgia |
| NCT01415921 | PHASE2 | COMPLETED | Safety Study of Pyridostigmine in Heart Failure |
| NCT01993680 | PHASE2 | COMPLETED | Orthostatic Dysregulation and Associated Gastrointestinal Dysfunction in Parkinson’s Disease -Treatment |
| NCT02227823 | PHASE2 | COMPLETED | Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 |
| NCT02307526 | PHASE2 | COMPLETED | Acetylcholinesterase Inhibition and Orthostatic Hypotension in SCI |
| NCT02893553 | PHASE2 | COMPLETED | The Effects of Normalizing Blood Pressure on Cerebral Blood Flow in Hypotensive Individuals With Spinal Cord Injury |
| NCT02941328 | PHASE2 | COMPLETED | SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 |
| NCT03312244 | PHASE2 | SUSPENDED | Pyridostigmine as Immunomodulator in People Living With HIV |
| NCT03674541 | PHASE2 | COMPLETED | The Exercise Response to Pharmacologic Cholinergic Stimulation in Myalgic Encephalomyelitis / Chronic Fatigue Syndrome |
| NCT06915181 | PHASE2 | WITHDRAWN | Clinical Usefulness and Influence on Esophageal Motility of Pyridostigmine for Dysphagia in Systemic Sclerosis |
| NCT03124355 | PHASE1 | ACTIVE_NOT_RECRUITING | Vagal Stimulation in POTS |
| NCT00223691 | PHASE1 | COMPLETED | Treatment of Orthostatic Hypotension in Autonomic Failure |
| NCT03063515 | PHASE1 | COMPLETED | AchE Inhibitor and Insulin |
| NCT02357225 | EARLY_PHASE1 | TERMINATED | A Pilot Study of Pyridostigmine in Pompe Disease |
| NCT02850276 | EARLY_PHASE1 | COMPLETED | Autonomic Neuropathy, GI Motility, and Inflammation in HIV |
| NCT03261570 | EARLY_PHASE1 | COMPLETED | Cardiovagal Baroreflex Deficits Impair Neurovascular Coupling and Cognition in POTS |
| NCT00953914 | Not specified | COMPLETED | Pyridostigmine and Its Effects on Autonomic Modulation in Diabetic Patients |
| NCT04768465 | Not specified | UNKNOWN | Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis |
| NCT06962007 | Not specified | COMPLETED | Sugammadex-Induced Clenching During Neuromuscular Blockade Reversal |
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.
155 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| BERBERINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| DONEPEZIL | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| ETHOPROPAZINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| GALANTAMINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| NEOSTIGMINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| NEOSTIGMINE METHYLSULFATE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| PHYSOSTIGMINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| PHYSOSTIGMINE SALICYLATE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| RASAGILINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| RESERPINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| RIVASTIGMINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| TACRINE | ChEMBL | Phase 4 (approved) | ACHE, BCHE |
| BAMBUTEROL | ChEMBL | Phase 3 | ACHE, BCHE |
| EBSELEN | ChEMBL | Phase 3 | ACHE, BCHE |
| LATREPIRDINE | ChEMBL | Phase 3 | ACHE, BCHE |
| QUERCETIN | ChEMBL | Phase 3 | ACHE, BCHE |
| EPTASTIGMINE | ChEMBL | Phase 2 | ACHE, BCHE |
| ESERIDINE | ChEMBL | Phase 2 | ACHE, BCHE |
| FISETIN | ChEMBL | Phase 2 | ACHE, BCHE |
| GANSTIGMINE | ChEMBL | Phase 2 | ACHE, BCHE |
| HUPERZINE A | ChEMBL | Phase 2 | ACHE, BCHE |
| ICOPEZIL | ChEMBL | Phase 2 | ACHE, BCHE |
| NEOSTIGMINE BROMIDE | ChEMBL | Phase 2 | ACHE, BCHE |
| PHENOTHIAZINE | ChEMBL | Phase 2 | ACHE, BCHE |
| SANGUINARIUM | ChEMBL | Phase 2 | ACHE, BCHE |
| GENTIAN VIOLET | ChEMBL + PubChem | Phase 4 (approved) | ACHE |
| PAZOPANIB | ChEMBL + PubChem | Phase 4 (approved) | ACHE |
| TADALAFIL | ChEMBL + PubChem | Phase 4 (approved) | ACHE |
| ALFUZOSIN | ChEMBL | Phase 4 (approved) | ACHE |
| ARIPIPRAZOLE | ChEMBL | Phase 4 (approved) | ACHE |
| BOSUTINIB | ChEMBL | Phase 4 (approved) | ACHE |
| BROMOCRIPTINE | ChEMBL | Phase 4 (approved) | ACHE |
| BUTENAFINE | ChEMBL | Phase 4 (approved) | ACHE |
| CABERGOLINE | ChEMBL | Phase 4 (approved) | ACHE |
| CAFFEINE | ChEMBL | Phase 4 (approved) | ACHE |
| CANNABIDIOL | ChEMBL | Phase 4 (approved) | ACHE |
| CITALOPRAM | ChEMBL | Phase 4 (approved) | ACHE |
| CLOTRIMAZOLE | ChEMBL | Phase 4 (approved) | ACHE |
| DECAMETHONIUM | ChEMBL | Phase 4 (approved) | ACHE |
| DECAMETHONIUM BROMIDE | ChEMBL | Phase 4 (approved) | ACHE |
| DEQUALINIUM | ChEMBL | Phase 4 (approved) | ACHE |
| DIETHYLSTILBESTROL | ChEMBL | Phase 4 (approved) | ACHE |
| DISULFIRAM | ChEMBL | Phase 4 (approved) | ACHE |
| DOFETILIDE | ChEMBL | Phase 4 (approved) | ACHE |
| DYCLONINE | ChEMBL | Phase 4 (approved) | ACHE |
| EBASTINE | ChEMBL | Phase 4 (approved) | ACHE |
| ECONAZOLE | ChEMBL | Phase 4 (approved) | ACHE |
| EDROPHONIUM | ChEMBL | Phase 4 (approved) | ACHE |
| EDROPHONIUM CHLORIDE | ChEMBL | Phase 4 (approved) | ACHE |
| EPIRUBICIN | ChEMBL | Phase 4 (approved) | ACHE |
| ETHYLESTRENOL | ChEMBL | Phase 4 (approved) | ACHE |
| FENOFIBRATE | ChEMBL | Phase 4 (approved) | ACHE |
| FLAVOXATE | ChEMBL | Phase 4 (approved) | ACHE |
| FLUOXETINE | ChEMBL | Phase 4 (approved) | ACHE |
| GALLAMINE | ChEMBL | Phase 4 (approved) | ACHE |
| HEXACHLOROPHENE | ChEMBL | Phase 4 (approved) | ACHE |
| HEXAFLUORENIUM | ChEMBL | Phase 4 (approved) | ACHE |
| ILOPERIDONE | ChEMBL | Phase 4 (approved) | ACHE |
| IRINOTECAN | ChEMBL | Phase 4 (approved) | ACHE |
| ISOFLUROPHATE | ChEMBL | Phase 4 (approved) | ACHE |
Related Atlas pages
- Genes: ACHE, BCHE
- Diseases: myasthenia gravis
- Drugs: Berberine, Donepezil, Ethopropazine, Galantamine, Neostigmine, Physostigmine, Rasagiline, Reserpine, Rivastigmine, Tacrine, Bambuterol, Ebselen, Latrepirdine, Quercetin, Pazopanib, Tadalafil, Alfuzosin, Aripiprazole, Bosutinib, Bromocriptine, Butenafine, Cabergoline, Caffeine, Cannabidiol, Citalopram, Clotrimazole, Decamethonium, Dequalinium, Diethylstilbestrol, Disulfiram, Dofetilide, Dyclonine, Ebastine, Econazole, Edrophonium, Epirubicin, Ethylestrenol, Fenofibrate, Flavoxate, Fluoxetine, Gallamine, Hexachlorophene, Hexafluorenium, Iloperidone, Irinotecan, Isoflurophate