Ozanimod

Summary

Ozanimod (CHEMBL3707247) is an approved small molecule (ATC L04AE02) targeting S1PR1, S1PR2, and S1PR3; indicated across 6 conditions including crohn disease and ulcerative colitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L04AE02
• Targets: 5 (S1PR1, S1PR2, S1PR3…)
• Indications: 6 conditions
• Clinical trials: 49
• Chemistry: 404.5 Da · C23H24N4O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3707247
Name	Ozanimod
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	52938427
ATC	L04AE02
Molecular formula	C23H24N4O3
Molecular weight	404.5
InChIKey	XRVDGNKRPOAQTN-FQEVSTJZSA-N

SMILES: CC(C)OC1=C(C=C(C=C1)C2=NC(=NO2)C3=C4CC[C@@H](C4=CC=C3)NCCO)C#N

IUPAC name: 5-[3-[(1S)-1-(2-hydroxyethylamino)-2,3-dihydro-1H-inden-4-yl]-1,2,4-oxadiazol-5-yl]-2-propan-2-yloxybenzonitrile

Also known as: Ozanimod, RPC-1063, RPC1063, OZANIMOD, ozanimod, C0088706

Parent form; salt/anhydrous children: CHEMBL3707246

Patent coverage: 625 distinct patent families (1,588 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 1,549 (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
S1PR1	S1P1 receptor	Agonist	9.48	0.2%	P21453
S1PR2	S1P2 receptor	Agonist	5	0%	O95136
S1PR3	S1P3 receptor	Agonist	5	0.2%	Q99500
S1PR4	S1P4 receptor	Agonist	5.1	0.2%	O95977
S1PR5	S1P5 receptor	Agonist	7.26	0%	Q9H228

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: Sphingosine 1-phosphate receptor 5, Sphingosine 1-phosphate receptor 2, Sphingosine 1-phosphate receptor 4, G-protein coupled receptor 183, Sphingosine 1-phosphate receptor 3, Sphingosine 1-phosphate receptor 1.

Bioactivity

ChEMBL activities: 12 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
S1PR1	9.8	EC50	0.16	nM	CHEMBL_ACT_16863706
S1PR1	9.8	EC50	0.16	nM	CHEMBL_ACT_18942832
S1PR1	9.8	EC50	0.16	nM	CHEMBL_ACT_19453556
S1PR1	9.4	EC50	0.4	nM	CHEMBL_ACT_25708615
S1PR1	9.39	EC50	0.41	nM	CHEMBL_ACT_18942833
S1PR1	8.47	EC50	3.36	nM	CHEMBL_ACT_25751474
S1PR5	8.37	EC50	4.3	nM	CHEMBL_ACT_19453557
S1PR5	7.26	EC50	55.2	nM	CHEMBL_ACT_16863926
S1PR4	5.38	EC50	4192	nM	CHEMBL_ACT_16863912
GPR183	5.36	IC50	4394	nM	CHEMBL_ACT_25751473
S1PR2	5.02	EC50	9559	nM	CHEMBL_ACT_16863884
S1PR3	5	EC50	9938	nM	CHEMBL_ACT_16863898

Target pathways

Aggregated over 5 target gene(s): S1PR1, S1PR2, S1PR3, S1PR4, S1PR5.

Top Reactome pathways

19 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	5	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
Signaling by GPCR	5	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
Class A/1 (Rhodopsin-like receptors)	5	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
Lysosphingolipid and LPA receptors	5	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
GPCR ligand binding	5	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
GPCR downstream signalling	4	S1PR2, S1PR3, S1PR4, S1PR5
G alpha (i) signalling events	4	S1PR2, S1PR3, S1PR4, S1PR5
Cytokine Signaling in Immune system	1	S1PR1
Disease	1	S1PR1
Immune System	1	S1PR1
Signaling by Interleukins	1	S1PR1
Infectious disease	1	S1PR1
Interleukin-4 and Interleukin-13 signaling	1	S1PR1
ESR-mediated signaling	1	S1PR3
Signaling by Nuclear Receptors	1	S1PR3
Extra-nuclear estrogen signaling	1	S1PR3
Potential therapeutics for SARS	1	S1PR1
SARS-CoV Infections	1	S1PR1
Viral Infection Pathways	1	S1PR1

Dominant GO biological processes

GO term	Targets
sphingosine-1-phosphate receptor signaling pathway	5
G protein-coupled receptor signaling pathway	5
adenylate cyclase-activating G protein-coupled receptor signaling pathway	5
signal transduction	5
positive regulation of cell population proliferation	3
adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway	2
actin cytoskeleton organization	2
angiogenesis	1
blood vessel maturation	1
cardiac muscle tissue growth involved in heart morphogenesis	1
chemotaxis	1
cell adhesion	1
phospholipase C-activating G protein-coupled receptor signaling pathway	1
brain development	1
cell population proliferation	1

Indications & clinical

Indications

6 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
Crohn disease	3	MONDO:0005011	EFO:0000384
ulcerative colitis	3	MONDO:0005101	EFO:0000729
multiple sclerosis	3	MONDO:0005301	MONDO:0005301
relapsing-remitting multiple sclerosis	3	MONDO:0005314	EFO:0003929
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096
liver disorder	1	MONDO:0005154	EFO:0001421

Clinical trials

Total trials: 49.

Phase distribution

Phase	Trials
PHASE3	13
Not specified	13
PHASE1	11
PHASE4	5
PHASE2	4
PHASE2/PHASE3	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06529406	PHASE4	RECRUITING	Prospective Evaluation of Sequencing From antiCD-20 Therapies to Ozanimod
NCT05369832	PHASE4	TERMINATED	An Open-label Study of Ozanimod in Moderate to Severe Ulcerative Colitis in Clinical Practice
NCT05777902	PHASE4	TERMINATED	Multidimensional Integrated Assessment to Test the Efficacy and Response to Ozanimod in Multiple Sclerosis.
NCT06188637	PHASE4	WITHDRAWN	Ulcerative Colitis Leukocyte TRAfficking After Treatment With Zeposia: the ULTRAZ Study
NCT06334094	PHASE4	WITHDRAWN	Assessing the Cognitive Benefits of Ozanimod and Their Brain-Biomarkers in MS
NCT05076175	PHASE2/PHASE3	RECRUITING	A Study Investigating Oral Ozanimod (RPC1063) in Pediatric Participants With Moderate to Severe Active Ulcerative Colitis
NCT06408259	PHASE3	RECRUITING	Study to Evaluate the Effectiveness and Safety of Ozanimod Compared to Fingolimod in Children and Adolescents With Relapsing Remitting Multiple Sclerosis
NCT01628393	PHASE2/PHASE3	COMPLETED	Efficacy and Safety Study of Ozanimod (RPC1063) in Relapsing Multiple Sclerosis Patients
NCT02047734	PHASE3	COMPLETED	Efficacy and Safety Study of Ozanimod in Relapsing Multiple Sclerosis
NCT02294058	PHASE3	COMPLETED	Study of Ozanimod (RPC1063) in Relapsing Multiple Sclerosis (MS)
NCT02435992	PHASE3	COMPLETED	Safety and Efficacy Trial of RPC1063 for Moderate to Severe Ulcerative Colitis
NCT02531126	PHASE3	COMPLETED	An Extension Study of RPC1063 as Therapy for Moderate to Severe Ulcerative Colitis
NCT02576717	PHASE3	COMPLETED	A Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple Sclerosis
NCT03440372	PHASE3	TERMINATED	Induction Study #1 of Oral Ozanimod as Induction Therapy for Moderately to Severely Active Crohn’s Disease
NCT03440385	PHASE3	COMPLETED	Induction Study #2 of Oral Ozanimod as Induction Therapy for Moderately to Severely Active Crohn’s Disease
NCT03464097	PHASE3	TERMINATED	A Placebo-Controlled Study of Oral Ozanimod as Maintenance Therapy for Moderately to Severely Active Crohn’s Disease
NCT03467958	PHASE3	TERMINATED	An Extension Study of Oral Ozanimod for Moderately to Severely Active Crohn’s Disease
NCT03915769	PHASE3	COMPLETED	To Evaluate Efficacy and Long-term Safety of Ozanimod in Japanese Subjects With Moderately to Severely Active Ulcerative Colitis
NCT04140305	PHASE3	TERMINATED	Study Describing Cognitive Processing Speed Changes in Relapsing Multiple Sclerosis Subjects Treated With Ozanimod (RPC-1063)
NCT05470985	PHASE2/PHASE3	TERMINATED	A Study to Evaluate the Efficacy, Safety, and Drug Levels of Oral Ozanimod in Pediatric Participants With Moderately to Severely Active Crohn’s Disease With an Inadequate Response to Conventional Therapy
NCT05644665	PHASE3	TERMINATED	A Study to Evaluate Efficacy and Long-term Safety of Oral Ozanimod in Chinese Participants With Moderately to Severely Active Ulcerative Colitis (UC)
NCT06862960	PHASE2	NOT_YET_RECRUITING	Ozanimod in Patients With Alzheimer’s Disease
NCT01647516	PHASE2	COMPLETED	Efficacy and Safety Study of Ozanimod in Ulcerative Colitis
NCT02531113	PHASE2	COMPLETED	Efficacy and Safety Trial of RPC1063 for Moderate to Severe Crohn’s Disease
NCT04405102	PHASE2	TERMINATED	COVID-19 Ozanimod Intervention Study
NCT02797015	PHASE1	COMPLETED	Pharmacokinetics and Pharmacodynamics Study of RPC1063 in RMS
NCT02994381	PHASE1	COMPLETED	A Single Dose Oral Excretion Balance Study of [14C]-RPC1063 in Healthy Male Adults
NCT03624959	PHASE1	COMPLETED	Drug-drug Interaction Study of Ozanimod With Inhibitor or Inducer of CYP2C8 and/or CYP3A
NCT03644576	PHASE1	COMPLETED	Drug-drug Interaction Study of Ozanimod With Pseudoephedrine to Evaluate the Effect on Blood Pressure and Heart Rate
NCT03694119	PHASE1	COMPLETED	Drug-drug Interaction Study of Ozanimod With Tyramine to Evaluate the Effect on Pressor Response
NCT04149678	PHASE1	COMPLETED	Drug Interaction Study of the Effect on Cyclosporine on Ozanimod and Major Active Metabolites
NCT04211558	PHASE1	COMPLETED	A Study to Evaluate the Pharmacokinetics of Single Oral Doses of Ozanimod in Healthy Adult Chinese Subjects
NCT04528290	PHASE1	COMPLETED	A Study to Evaluate the Relative Bioavailability of a Pediatric Granule Formulation of Ozanimod in Healthy Adult Subjects
NCT04639115	PHASE1	COMPLETED	A Phase 1, Multicenter, Open-Label Study to Evaluate the Effect of Mild or Moderate Hepatic Impairment on the Multiple-Dose Pharmacokinetics of Ozanimod
NCT04978298	PHASE1	COMPLETED	Study to Evaluate the Pressor Effect of Oral Tyramine During Ozanimod Treatment in Healthy Adult Participants
NCT05001152	PHASE1	COMPLETED	Taste Assessment of Ozanimod
NCT03500328	Not specified	ACTIVE_NOT_RECRUITING	Traditional Versus Early Aggressive Therapy for Multiple Sclerosis Trial
NCT04676204	Not specified	ENROLLING_BY_INVITATION	Relationship Between Oral DMT Burden and Adherence in MS
NCT05688436	Not specified	RECRUITING	A Study to Learn More About The Safety of Diroximel Fumarate (VUMERITY®) in Participants Who Took it During Pregnancy And About the Health of Their Babies
NCT06073873	Not specified	RECRUITING	A Post-Marketing Surveillance Study to Assess Safety of Ozanimod in Patients With Moderate to Severe Active UC in Korea

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.

10 molecules share ≥1 primary target. Top 10 by shared-target count:

Molecule	Source	Status	Shared targets
FINGOLIMOD	ChEMBL + PubChem	Phase 4 (approved)	S1PR1, S1PR2, S1PR3, S1PR4, S1PR5
ETRASIMOD	ChEMBL + PubChem	Phase 4 (approved)	S1PR1, S1PR2, S1PR4, S1PR5
SIPONIMOD	ChEMBL + PubChem	Phase 4 (approved)	S1PR1, S1PR3, S1PR4, S1PR5
PONESIMOD	ChEMBL	Phase 4 (approved)	S1PR1, S1PR3
CENERIMOD	ChEMBL	Phase 3	S1PR1
AMISELIMOD	ChEMBL	Phase 2	S1PR1
ICANBELIMOD	ChEMBL	Phase 2	S1PR1
NIGULDIPINE	ChEMBL	Phase 2	S1PR1
PINAFIDE	ChEMBL	Phase 2	S1PR1
Belzutifan	PubChem	Approved	S1PR3