Voclosporin

Summary

Voclosporin (CHEMBL5314379) is an approved protein (ATC L04AD03) targeting PPIA; indicated across 12 conditions including immune system disorder and lupus nephritis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: L04AD03
• Targets: 1 (PPIA)
• Indications: 12 conditions
• Clinical trials: 24
• Chemistry: 1214.6 Da · C63H111N11O12

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5314379
Name	Voclosporin
Type	Protein
Max phase	4
FDA approved	yes
PubChem CID	6918486
ATC	L04AD03
Molecular formula	C63H111N11O12
Molecular weight	1214.6
InChIKey	BICRTLVBTLFLRD-PTWUADNWSA-N

SMILES: CC[C@H]1C(=O)N(CC(=O)N([C@H](C(=O)N[C@H](C(=O)N([C@H](C(=O)N[C@H](C(=O)N[C@@H](C(=O)N([C@H](C(=O)N([C@H](C(=O)N([C@H](C(=O)N([C@H](C(=O)N1)[C@@H]([C@H](C)C/C=C/C=C)O)C)C(C)C)C)CC(C)C)C)CC(C)C)C)C)C)CC(C)C)C)C(C)C)CC(C)C)C)C

IUPAC name: (3S,6S,9S,12R,15S,18S,21S,24S,30S,33S)-30-ethyl-33-[(1R,2R,4E)-1-hydroxy-2-methylhepta-4,6-dienyl]-1,4,7,10,12,15,19,25,28-nonamethyl-6,9,18,24-tetrakis(2-methylpropyl)-3,21-di(propan-2-yl)-1,4,7,10,13,16,19,22,25,28,31-undecazacyclotritriacontane-2,5,8,11,14,17,20,23,26,29,32-undecone

Also known as: ISA-247, ISA247, ISATX-247, ISATX247, Lupkynis, LX-211, LX211, Voclosporin, Voclosporina, Voclosporine, VOCLOSPORIN

Patent coverage: 55 distinct patent families (172 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
PPIA	peptidylprolyl isomerase A	Binding	7.82	80%	P62937

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): PPIA.

Top Reactome pathways

15 total, by targets touching each:

Pathway	Targets	Genes
Platelet degranulation	1	PPIA
Uncoating of the HIV Virion	1	PPIA
Budding and maturation of HIV virion	1	PPIA
Integration of provirus	1	PPIA
Early Phase of HIV Life Cycle	1	PPIA
Minus-strand DNA synthesis	1	PPIA
Plus-strand DNA synthesis	1	PPIA
Binding and entry of HIV virion	1	PPIA
Assembly Of The HIV Virion	1	PPIA
APOBEC3G mediated resistance to HIV-1 infection	1	PPIA
Calcineurin activates NFAT	1	PPIA
Basigin interactions	1	PPIA
Neutrophil degranulation	1	PPIA
Gene and protein expression by JAK-STAT signaling after Interleukin-12 stimulation	1	PPIA
SARS-CoV-1 activates/modulates innate immune responses	1	PPIA

Dominant GO biological processes

GO term	Targets
protein peptidyl-prolyl isomerization	1
negative regulation of protein phosphorylation	1
positive regulation of protein phosphorylation	1
protein folding	1
negative regulation of protein kinase activity	1
apoptotic process	1
viral release from host cell	1
platelet activation	1
neuron differentiation	1
neutrophil chemotaxis	1
leukocyte chemotaxis	1
activation of protein kinase B activity	1
negative regulation of stress-activated MAPK cascade	1
lipid droplet organization	1
cellular response to oxidative stress	1

Indications & clinical

Indications

12 indications (2 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
immune system disorder	4	MONDO:0005046	EFO:0000540
lupus nephritis	4	MONDO:0005556	EFO:0005761
psoriasis	3	MONDO:0005083	EFO:0000676
uveitis	3	MONDO:0020283	EFO:1001231
intermediate uveitis	3	MONDO:0006806	EFO:1000986
posterior uveitis	3	MONDO:0006918	EFO:1001119
anterior uveitis	3	MONDO:0006651	EFO:1000811
panuveitis	3	MONDO:0017255	EFO:1001082
kidney disorder	2	MONDO:0005240	EFO:0003086
focal segmental glomerulosclerosis	2	MONDO:0100313	EFO:0004236
dry eye syndrome	2	MONDO:0006733	EFO:1000906
systemic lupus erythematosus	2	MONDO:0007915	MONDO:0007915

Clinical trials

Total trials: 24.

Phase distribution

Phase	Trials
PHASE3	12
PHASE2	5
Not specified	3
PHASE4	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07225387	PHASE4	RECRUITING	Safety and Efficacy of Combination Belimumab and Voclosporin in the Treatment of Proliferative Forms of Lupus Glomerulopathy: Synergy Trial
NCT07611214	PHASE4	RECRUITING	PRESERVE: LUPKYNIS in Combination With Belimumab, Obinutuzumab or Anifrolumab in Patients With Lupus Nephritis
NCT00244842	PHASE3	COMPLETED	Randomized Placebo-controlled Study of ISA247 in Plaque Psoriasis
NCT00258713	PHASE3	COMPLETED	A 36-Week Extension to Protocol ISA04-03
NCT00404612	PHASE3	COMPLETED	A Study of LX211 in Active Sight Threatening, Non-infectious Intermediate-, Anterior and Intermediate-, Posterior-, or Pan-Uveitis
NCT00404742	PHASE3	COMPLETED	A Study of LX211 in Clinically Quiescent Non-infectious Intermediate, Anterior and Intermediate, Posterior or Pan-Uveitis
NCT00404885	PHASE3	COMPLETED	A Multi-center Study to Assess the Efficacy and Safety of LX211 in Active Non-infectious Anterior Uveitis
NCT00408187	PHASE3	COMPLETED	Randomized, Placebo and Ciclosporin Controlled Study of ISA247 in Plaque Psoriasis Patients
NCT01243983	PHASE3	COMPLETED	Efficacy and Safety of Voclosporin to Treat Active Noninfectious Uveitis
NCT01586845	PHASE3	WITHDRAWN	Safety and Efficacy Study of Voclosporin and Tacrolimus in Transplantation
NCT03021499	PHASE3	COMPLETED	Aurinia Renal Response in Active Lupus With Voclosporin
NCT03597464	PHASE3	COMPLETED	Aurinia Renal Assessments 2: Aurinia Renal Response in Lupus With Voclosporin
NCT05288855	PHASE3	TERMINATED	Voclosporin in Adolescent and Pediatric Subjects With Lupus Nephritis
NCT05962788	PHASE3	TERMINATED	Long-Term Voclosporin Treatment in Adolescent and Pediatric Subjects With Lupus Nephritis
NCT00270634	PHASE2	COMPLETED	Study of ISA247 (Voclosporin) in De Novo Renal Transplantation
NCT02949973	PHASE2	COMPLETED	Aurinia Early Urinary Protein Reduction Predicts Response
NCT03598036	PHASE2	TERMINATED	Dose-Exploration Evaluating the Efficacy and Safety of Voclosporin in Subjects With Focal Segmental Glomerulosclerosis
NCT04701528	PHASE2	COMPLETED	Anti-Viral Effects of Voclosporin in COVID-19 Positive Kidney Transplant Recipients
NCT05306873	PHASE2	TERMINATED	Examining Distinct Immunophenotypes to Validate and Enhance Rational Treatment in Systemic Lupus
NCT02949999	PHASE1	COMPLETED	Voclosporin in Healthy Japanese Volunteers
NCT05306379	PHASE1	COMPLETED	Drug-Drug Interaction Study to Investigate Effects of Voclosporin on Pharmacokinetics of Simvastatin
NCT07053891	Not specified	RECRUITING	LUPKYNIS Drug-use Results Survey
NCT01236287	Not specified	NO_LONGER_AVAILABLE	Special Access for the Use of Voclosporin for Kidney Transplantation
NCT05337124	Not specified	COMPLETED	A Prospective Observational Registry of Patients Treated With LUPKYNIS® (Voclosporin) in the US

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

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.

7 molecules share ≥1 primary target. Top 7 by shared-target count:

Molecule	Source	Status	Shared targets
CYCLOSPORINE	ChEMBL + PubChem	Phase 4 (approved)	PPIA
LANSOPRAZOLE	ChEMBL	Phase 4 (approved)	PPIA
ALISPORIVIR	ChEMBL	Phase 3	PPIA
GECLOSPORIN	ChEMBL	Phase 2	PPIA
NIM811	ChEMBL	Phase 2	PPIA
SCY 635	ChEMBL	Phase 2	PPIA
Zidovudine	PubChem	Approved	PPIA