Lucerastat
drugOn this page
Also known as ACT-434964N-butyldeoxygalactonojirimycinN-n-butyl deoxygalactonojirimycin
Summary
Lucerastat (CHEMBL1086997) is a phase-3 clinical-stage small molecule; indicated across 1 condition including fabry disease.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Small molecule
- Indications: 1 condition
- Clinical trials: 6
- Chemistry: 219.28 Da · C10H21NO4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1086997 |
| Name | Lucerastat |
| Type | Small molecule |
| Max phase | 3 |
| FDA approved | no |
| PubChem CID | 501391 |
| Molecular formula | C10H21NO4 |
| Molecular weight | 219.28 |
| InChIKey | UQRORFVVSGFNRO-XFWSIPNHSA-N |
SMILES: CCCCN1C[C@@H]([C@H]([C@H]([C@H]1CO)O)O)O
IUPAC name: (2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Also known as: ACT-434964, Lucerastat, N-butyldeoxygalactonojirimycin, N-n-butyl deoxygalactonojirimycin, LUCERASTAT
Patent coverage: 27 distinct patent families (74 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
Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: Ceramide glucosyltransferase, Maltase-glucoamylase, Lysosomal alpha-glucosidase, Sucrase-isomaltase, intestinal, Beta-glucosidase, Glycogen debranching enzyme.
Bioactivity
ChEMBL activities: 8 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| GBA2 | 6.52 | IC50 | 300 | nM | CHEMBL_ACT_15096298 |
| GBA2 | 6.52 | IC50 | 300 | nM | CHEMBL_ACT_3204087 |
| SI | 6.34 | IC50 | 460 | nM | CHEMBL_ACT_3204036 |
| GAA | 5.82 | IC50 | 1500 | nM | CHEMBL_ACT_3204100 |
| UGCG | 5 | IC50 | 10000 | nM | CHEMBL_ACT_15096296 |
| UGCG | 5 | IC50 | 10000 | nM | CHEMBL_ACT_3204023 |
| MGAM | 5 | IC50 | 10000 | nM | CHEMBL_ACT_3204049 |
| AGL | 5 | IC50 | 10000 | nM | CHEMBL_ACT_3204112 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
1 indication (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 |
|---|---|---|---|
| Fabry disease | 3 | MONDO:0010526 | MONDO:0010526 |
Clinical trials
Total trials: 6.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE1 | 4 |
| PHASE3 | 2 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03737214 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease |
| NCT03425539 | PHASE3 | COMPLETED | Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease |
| NCT02930655 | PHASE1 | COMPLETED | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease |
| NCT02944474 | PHASE1 | COMPLETED | A Study to Assess the Safety, Tolerability and Pharmacokinetics of Lucerastat (CDP923) After Multiple Dosing in Healthy Subjects |
| NCT02944487 | PHASE1 | COMPLETED | A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects |
| NCT03380455 | PHASE1 | COMPLETED | Effect of Cimetidine on the Pharmacokinetics of Lucerastat in Healthy Subjects |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Diseases: Fabry disease