Rivipansel

Summary

Rivipansel (CHEMBL3707446) is a phase-3 clinical-stage small molecule targeting SELP; indicated across 3 conditions including sickle cell disease and liver disorder.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (SELP)
• Indications: 3 conditions
• Clinical trials: 9
• Chemistry: 1447.4 Da · C58H74N6O31S3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3707446
Name	Rivipansel
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	44232546
Molecular formula	C58H74N6O31S3
Molecular weight	1447.4
InChIKey	VXBNTHRZPJLRSS-PTCSXESPSA-N

SMILES: C[C@H]1[C@H]([C@H]([C@@H]([C@@H](O1)O[C@@H]2[C@H](C[C@H](C[C@H]2O[C@H]3[C@@H]([C@H]([C@H]([C@H](O3)CO)O)O[C@@H](CC4CCCCC4)C(=O)O)OC(=O)C5=CC=CC=C5)C(=O)NCCNC(=O)COCCOCC(=O)NC6=C7C(=CC(=C6)S(=O)(=O)O)C=C(C=C7S(=O)(=O)O)S(=O)(=O)O)NC(=O)C8=CC(=O)NC(=O)N8)O)O)O

IUPAC name: (2S)-2-[(2R,3R,4S,5S,6R)-3-benzoyloxy-2-[(1R,2R,3S,5R)-3-[(2,4-dioxo-1H-pyrimidine-6-carbonyl)amino]-5-[2-[[2-[2-[2-oxo-2-[(3,6,8-trisulfonaphthalen-1-yl)amino]ethoxy]ethoxy]acetyl]amino]ethylcarbamoyl]-2-[(2S,3S,4R,5S,6S)-3,4,5-trihydroxy-6-methyloxan-2-yl]oxycyclohexyl]oxy-5-hydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-3-cyclohexylpropanoic acid

Also known as: GMI-1070, PF-06460031, Rivipansel, RIVIPANSEL

Parent form; salt/anhydrous children: CHEMBL3833316

Patent coverage: 56 distinct patent families (145 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 107 (74%) 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
SELP	selectin P (CD62)	Binding	3.37		P16109

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: E-selectin.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
SELE	5.37	Kd	4300	nM	CHEMBL_ACT_26022435

Target pathways

Aggregated over 1 target gene(s): SELP.

Top Reactome pathways

5 total, by targets touching each:

Pathway	Targets	Genes
Hemostasis	1	SELP
Platelet degranulation	1	SELP
Cell surface interactions at the vascular wall	1	SELP
Platelet activation, signaling and aggregation	1	SELP
Response to elevated platelet cytosolic Ca2+	1	SELP

Dominant GO biological processes

GO term	Targets
positive regulation of leukocyte migration	1
inflammatory response	1
cell adhesion	1
heterophilic cell-cell adhesion	1
leukocyte cell-cell adhesion	1
positive regulation of platelet activation	1
calcium-dependent cell-cell adhesion	1
response to lipopolysaccharide	1
regulation of integrin activation	1
response to cytokine	1
defense response to Gram-negative bacterium	1
leukocyte tethering or rolling	1
positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction	1
cell-cell adhesion	1
positive regulation of leukocyte tethering or rolling	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
sickle cell disease	3	MONDO:0011382	MONDO:0011382
liver disorder	1	MONDO:0005154	EFO:0001421
kidney disorder	1	MONDO:0005240	EFO:0003086

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE1	5
PHASE3	2
PHASE1/PHASE2	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02187003	PHASE3	COMPLETED	Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
NCT02433158	PHASE3	TERMINATED	Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
NCT00911495	PHASE1/PHASE2	COMPLETED	Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
NCT01119833	PHASE2	COMPLETED	Study of GMI-1070 for the Treatment of Sickle Cell Pain Crisis
NCT01941511	PHASE1	COMPLETED	A Study to Evaluate the Effect of IV Doses of GMI-1070 on Qtc Intervals in Healthy African-American Adult Subjects
NCT02217917	PHASE1	COMPLETED	Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Subcutaneous Rivipansel With rHuPH20 in Healthy Subjects
NCT02813798	PHASE1	COMPLETED	A Study to Evaluate the Effect of IV Doses of Rivipansel on Subjects With Renal Impairment and in Healthy Subjects
NCT02871570	PHASE1	COMPLETED	A Study to Evaluate the Effect of IV Doses of Rivipansel in Subjects With Moderate Hepatic Impairment and in Healthy Subjects With Normal Hepatic Function
NCT02998099	PHASE1	COMPLETED	A Study to Evaluate the Effect of Age on the Pharmacokinetics, Safety and Tolerability of IV Rivipansel.

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.

2 molecules share ≥1 primary target. Top 2 by shared-target count:

Molecule	Source	Status	Shared targets
GALLIC ACID	ChEMBL + PubChem	Phase 2 (approved)	SELP
BIMOSIAMOSE	ChEMBL	Phase 2	SELP