Uproleselan

Summary

Uproleselan (CHEMBL3989971) is a phase-3 clinical-stage small molecule targeting SELE; indicated across 4 conditions including acute myeloid leukemia and plasma cell myeloma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (SELE)
• Indications: 4 conditions
• Clinical trials: 17
• Chemistry: 1304.5 Da · C60H109N3O27

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989971
Name	Uproleselan
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	71600085
Molecular formula	C60H109N3O27
Molecular weight	1304.5
InChIKey	LYSYOXNOOPBOSC-NGSKMYNLSA-N

SMILES: CC[C@H]1C[C@H](C[C@H]([C@@H]1O[C@H]2[C@H]([C@@H]([C@@H]([C@@H](O2)C)O)O)O)O[C@H]3[C@@H]([C@H]([C@H]([C@H](O3)CO)O)O[C@@H](CC4CCCCC4)C(=O)O)NC(=O)C)C(=O)NCCNC(=O)CCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOC

IUPAC name: (2S)-2-[(2R,3R,4R,5R,6R)-3-acetamido-2-[(1R,2R,3S,5R)-3-ethyl-5-[2-[3-[2-[2-[2-[2-[2-[2-[2-[2-[2-[2-(2-methoxyethoxy)ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]ethoxy]propanoylamino]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-1271, GMI-1271 FREE ACID, Uproleselan, UPROLESELAN

Parent form; salt/anhydrous children: CHEMBL3989975

Patent coverage: 83 distinct patent families (287 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
SELE	selectin E (CD62E)	Antagonist	5.76	0.1%	P16581

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): SELE.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Hemostasis	1	SELE
Cell surface interactions at the vascular wall	1	SELE

Dominant GO biological processes

GO term	Targets
positive regulation of receptor internalization	1
leukocyte migration involved in inflammatory response	1
positive regulation of leukocyte migration	1
inflammatory response	1
heterophilic cell-cell adhesion	1
leukocyte cell-cell adhesion	1
phospholipase C-activating G protein-coupled receptor signaling pathway	1
calcium-mediated signaling	1
actin filament-based process	1
response to lipopolysaccharide	1
response to cytokine	1
response to tumor necrosis factor	1
regulation of inflammatory response	1
leukocyte tethering or rolling	1
response to interleukin-1	1

Indications & clinical

Indications

4 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
acute myeloid leukemia	3	MONDO:0018874	EFO:0000222
plasma cell myeloma	2	MONDO:0009693	EFO:0001378
severe acute respiratory syndrome	1	MONDO:0005091	MONDO:0100096

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 17.

Phase distribution

Phase	Trials
PHASE1	9
PHASE1/PHASE2	4
PHASE3	2
PHASE2/PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03701308	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Daunorubicin and Cytarabine With or Without Uproleselan in Treating Older Adult Patients With Acute Myeloid Leukemia Receiving Intensive Induction Chemotherapy
NCT03616470	PHASE3	TERMINATED	Study to Determine the Efficacy of Uproleselan (GMI-1271) in Combination With Chemotherapy to Treat Relapsed/Refractory Acute Myeloid Leukemia
NCT05054543	PHASE3	TERMINATED	Study to Evaluate the Efficacy of Uproleselan in Combination With Chemotherapy in Chinese Patients With R/R AML
NCT02306291	PHASE1/PHASE2	COMPLETED	Study to Determine Safety, Pharmacokinetics and Efficacy of GMI-1271 in Combination With Chemotherapy in AML
NCT02744833	PHASE1/PHASE2	TERMINATED	Study to Assess Safety, Tolerability, and Efficacy of GMI-1271 in Patients With Calf-level Deep Venous Thrombosis (DVT)
NCT04682405	PHASE2	COMPLETED	Uproleselan (GMI-1271) for GI Toxicity Prophylaxis During Melphalan-Conditioned Autologous Hematopoietic Cell Transplantation (Auto-HCT) for Multiple Myeloma (MM)
NCT04848974	PHASE1/PHASE2	COMPLETED	Uproleselan, Cladribine, and Low Dose Cytarabine for the Treatment of Patients With Treated Secondary Acute Myeloid Leukemia
NCT05057221	PHASE1/PHASE2	TERMINATED	Safety, Tolerability and Efficacy of Uproleselan (GMI-1271) in Patients With COVID-19 Pneumonia
NCT05146739	PHASE1	ACTIVE_NOT_RECRUITING	Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory That Expresses E-selectin Ligand on the Cell Membrane
NCT02168595	PHASE1	COMPLETED	Placebo-controlled Single Dose Study to Evaluate Safety and Pharmacokinetics of GMI-1271 in Healthy Volunteers
NCT02271113	PHASE1	COMPLETED	Phase I Study to Assess Safety and Pharmacokinetics of GMI-1271 in Healthy Adult Subjects
NCT02703051	PHASE1	COMPLETED	A Study to Evaluate the PK, Safety and Tolerability of GMI-1271 With and Without Filgrastim in Healthy Adult Subjects
NCT02811822	PHASE1	COMPLETED	A Study to Determine the Efficacy, Safety and Pharmacokinetics of GMI-1271 as Adjunct to Standard of Care for the Treatment of Multiple Myeloma
NCT03606447	PHASE1	COMPLETED	A Study in Healthy Volunteers to Investigate How Much Test Medicine [14C]-Uproleselan is Taken up by the Body When Administered Directly Into the Vein (IV)
NCT04839341	PHASE1	COMPLETED	Study to Evaluate PK and Safety With Uproleselan Combined With Chemotherapy to Treat Chinese R/R AML Patients
NCT04964505	PHASE1	TERMINATED	Uproleselan, Azacitidine, and Venetoclax for the Treatment of Treatment Naive Acute Myeloid Leukemia
NCT05569512	PHASE1	TERMINATED	Uproleselan With Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for AML

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
RIVIPANSEL	ChEMBL	Phase 3	SELE
Gallic Acid	PubChem	Approved	SELE