Pridopidine
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Also known as ACR-16ACR16FR-310826FR310826HuntexilPridopidina
Summary
Pridopidine (CHEMBL596802) is a phase-3 clinical-stage small molecule targeting SIGMAR1; indicated across 3 conditions including huntington disease and amyotrophic lateral sclerosis.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Small molecule
- Targets: 1 (SIGMAR1)
- Indications: 3 conditions
- Clinical trials: 13
- Chemistry: 281.4 Da · C15H23NO2S
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL596802 |
| Name | Pridopidine |
| Type | Small molecule |
| Max phase | 3 |
| FDA approved | no |
| PubChem CID | 9795739 |
| Molecular formula | C15H23NO2S |
| Molecular weight | 281.4 |
| InChIKey | YGKUEOZJFIXDGI-UHFFFAOYSA-N |
SMILES: CCCN1CCC(CC1)C2=CC(=CC=C2)S(=O)(=O)C
IUPAC name: 4-(3-methylsulfonylphenyl)-1-propylpiperidine
Also known as: ACR-16, ACR16, FR-310826, FR310826, Huntexil, Pridopidina, Pridopidine, PRIDOPIDINE
Parent form; salt/anhydrous children: CHEMBL2105691
Patent coverage: 195 distinct patent families (555 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 545 (98%) 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 |
|---|---|---|---|---|---|
| SIGMAR1 | sigma non-opioid intracellular receptor 1 | Agonist | 7.09 | 2.6% | Q99720 |
Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: D(2) dopamine receptor, Sigma non-opioid intracellular receptor 1.
Bioactivity
ChEMBL activities: 7 potent at pChembl ≥ 5 of 9 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| SIGMAR1 | 7.77 | Ki | 17 | nM | CHEMBL_ACT_24977773 |
| SIGMAR1 | 7.09 | Ki | 81.7 | nM | CHEMBL_ACT_22850783 |
| SIGMAR1 | 7.09 | Ki | 82 | nM | CHEMBL_ACT_25506946 |
| SIGMAR1 | 7.09 | Ki | 82 | nM | CHEMBL_ACT_25882780 |
| DRD2 | 5.12 | Ki | 7500 | nM | CHEMBL_ACT_22850847 |
| DRD2 | 5.12 | Ki | 7521 | nM | CHEMBL_ACT_3116204 |
| DRD2 | 5 | Ki | 10000 | nM | CHEMBL_ACT_24977780 |
Target pathways
Aggregated over 1 target gene(s): SIGMAR1.
Top Reactome pathways
5 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Disease | 1 | SIGMAR1 |
| Infectious disease | 1 | SIGMAR1 |
| Potential therapeutics for SARS | 1 | SIGMAR1 |
| SARS-CoV Infections | 1 | SIGMAR1 |
| Viral Infection Pathways | 1 | SIGMAR1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| lipid transport | 1 |
| nervous system development | 1 |
| regulation of neuron apoptotic process | 1 |
| protein homotrimerization | 1 |
| response to alcohol | 1 |
| regulation of postsynapse assembly | 1 |
| G protein-coupled opioid receptor signaling pathway | 1 |
Indications & clinical
Indications
3 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 |
|---|---|---|---|
| Huntington disease | 3 | MONDO:0007739 | MONDO:0007739 |
| amyotrophic lateral sclerosis | 2 | MONDO:0004976 | MONDO:0004976 |
| Parkinson disease | 2 | MONDO:0005180 | MONDO:0005180 |
Clinical trials
Total trials: 13.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 5 |
| PHASE3 | 4 |
| PHASE2/PHASE3 | 2 |
| PHASE1 | 1 |
| Not specified | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04297683 | PHASE2/PHASE3 | RECRUITING | HEALEY ALS Platform Trial - Master Protocol |
| NCT07322003 | PHASE3 | RECRUITING | Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS |
| NCT07609108 | PHASE3 | NOT_YET_RECRUITING | PRidopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease |
| NCT00665223 | PHASE3 | COMPLETED | A Study of Treatment With Pridopidine (ACR16) in Participants With Huntington’s Disease |
| NCT04556656 | PHASE3 | COMPLETED | PRidopidine’s Outcome On Function in Huntington Disease, PROOF- HD |
| NCT04615923 | PHASE2/PHASE3 | COMPLETED | HEALEY ALS Platform Trial - Regimen D Pridopidine |
| NCT00724048 | PHASE2 | COMPLETED | A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington’s Disease |
| NCT01306929 | PHASE2 | COMPLETED | Open-label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington Disease |
| NCT02006472 | PHASE2 | COMPLETED | A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington’s Disease |
| NCT02494778 | PHASE2 | TERMINATED | A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington’s Disease |
| NCT03922711 | PHASE2 | TERMINATED | A Study to Assess the Safety and Effectiveness of Pridopidine Compared to Placebo in the Treatment of Levodopa-Induced Dyskinesia in Patients With Parkinson’s Disease |
| NCT03019289 | PHASE1 | COMPLETED | A Study to Evaluate Sigma-1 and Dopamine-2 Receptor Occupancy by Pridopidine in the Human Brain of Healthy Volunteers and in Patients With Huntington’s Disease |
| NCT06069934 | Not specified | NO_LONGER_AVAILABLE | A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2) |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
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.
143 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| METHAMPHETAMINE | ChEMBL + PubChem | Phase 4 (approved) | SIGMAR1 |
| AMANTADINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| AMIODARONE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| AMITRIPTYLINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| ASTEMIZOLE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| AZELASTINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BENZTROPINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BETAXOLOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BREXPIPRAZOLE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BROMHEXINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BUFLOMEDIL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BUSPIRONE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| BUTENAFINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CARBETAPENTANE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CHLOROQUINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CHLORPROMAZINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CINACALCET | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CINNARIZINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CISAPRIDE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CITALOPRAM | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CLEMASTINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CLOMIPRAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| CLOZAPINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| COCAINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DEXCHLORPHENIRAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DEXFENFLURAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DEXTROMETHORPHAN | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DICYCLOMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DIHYDROERGOTAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DIMENHYDRINATE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DIPHENIDOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DOBUTAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DONEPEZIL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| DOXEPIN | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| ECONAZOLE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| FENTANYL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| FLUOXETINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| FLUPHENAZINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| FLUVOXAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| HALOPERIDOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| HYDROXYCHLOROQUINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| ILOPERIDONE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| KETANSERIN | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| LABETALOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| LASMIDITAN | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| LEVAMFETAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| LEVOBUNOLOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| LOPERAMIDE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| MAZINDOL | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| MEPAZINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| METHYSERGIDE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| MIFEPRISTONE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| NAFTIFINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| OXYBUTYNIN | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PAROXETINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PENTAZOCINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PERHEXILINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PHENTOLAMINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PIPAMAZINE | ChEMBL | Phase 4 (approved) | SIGMAR1 |
| PITOLISANT | ChEMBL | Phase 4 (approved) | SIGMAR1 |
Related Atlas pages
- Genes: SIGMAR1
- Diseases: Huntington disease
- Drugs: Methamphetamine, Amantadine, Amiodarone, Amitriptyline, Astemizole, Azelastine, Benztropine, Betaxolol, Brexpiprazole, Bromhexine, Buflomedil, Buspirone, Butenafine, Carbetapentane, Chloroquine, Chlorpromazine, Cinacalcet, Cinnarizine, Cisapride, Citalopram, Clemastine, Clomipramine, Clozapine, Cocaine, Dexchlorpheniramine, Dexfenfluramine, Dextromethorphan, Dicyclomine, Dihydroergotamine, Dimenhydrinate, Diphenidol, Dobutamine, Donepezil, Doxepin, Econazole, Fentanyl, Fluoxetine, Fluphenazine, Fluvoxamine, Haloperidol, Hydroxychloroquine, Iloperidone, Ketanserin, Labetalol, Lasmiditan, Levamfetamine, Levobunolol, Loperamide, Mazindol, Mepazine, Methysergide, Mifepristone, Naftifine, Oxybutynin, Paroxetine, Pentazocine, Perhexiline, Phentolamine, Pipamazine, Pitolisant